UNITED
STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event Reported): January 9, 2023
(Exact Name of Registrant as Specified in Charter)
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(State or Other Jurisdiction of Incorporation)
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(Commission File Number)
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(I.R.S. Employer Identification Number)
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(Address of Principal Executive Offices) (Zip Code)
(857 ) 259-3860
(Registrant’s telephone number, including area code)
(Registrant’s telephone number, including area code)
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Securities registered pursuant to Section 12(b) of the Act:
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Title of each class
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Trading Symbol(s)
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Name of each exchange on which registered
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). Emerging growth company
☒
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange
Act. ☒
Item 7.01. Regulation FD Disclosure.
On January 3, 2022, Scholar Rock Holding Corporation (the “Company”) announced that management will present at the 41st Annual J.P. Morgan Healthcare
Conference on Tuesday, January 10, 2023 at 1:30 p.m. PT (4:30 p.m. ET). A copy of the presentation slide deck that will be presented is being furnished as Exhibit 99.1 to this report on Form 8-K. A live webcast of the presentation may be accessed
by visiting the Investors & Media section of the Scholar Rock website at http://investors.scholarrock.com.
The information in this Item 7.01 and Exhibit 99.1 attached hereto is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of
the Securities Exchange Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section. It may only be incorporated by
reference in another filing under the Exchange Act or the Securities Act of 1933, as amended, if such subsequent filing specifically references the information furnished pursuant to Item 7.01 and Exhibit 99.1 of this Current Report on Form 8-K.
Item 8.01. Other Events.
On January 9, 2023, the Company issued a press release announcing a corporate update and highlighting priorities for 2023. A copy of this press release is
being filed herewith as Exhibit 99.2 to this Current Report on Form 8-K.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
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Exhibit
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Description
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104
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Cover Page Interactive Data File (embedded within the Inline XBRL document)
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SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the
undersigned hereunto duly authorized.
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Scholar Rock Holding Corporation
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Date: January 9, 2023
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By:
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/s/ Junlin Ho
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Junlin Ho
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General Counsel & Corporate Secretary
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| Exhibit 99.1 |
Deep InsightsAdvancingImpactful Medicines January 2023
Disclaimers Various statements in this presentation concerning the future expectations, plans and
prospects of Scholar Rock, Inc. (“Scholar Rock”), including without limitation, Scholar Rock’s expectations regarding its strategy, its product candidate selection and development timing, including timing for the initiation of and reporting
results from its clinical trials for apitegromab, SRK-181, and other product candidates and indication selection and development timing, its cash runway, the ability of any product candidate to perform in humans in a manner consistent with
earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as “may,” “could,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,”
“estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements for the purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and
adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data, including the results from the Phase 2 trial of apitegromab or
Part A of the Phase 1 trial of SRK-181, are not predictive of, may be inconsistent with, or more favorable than, data generated from future clinical trials of the same product candidate, including the Phase 3 clinical trial of apitegromab in
SMA and Part B of the Phase 1 clinical trial of SRK-181, respectively, Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline, the data
generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials, information provided or decisions made by regulatory authorities, competition from third parties that are developing products for similar uses, Scholar
Rock’s ability to obtain, maintain and protect its intellectual property, the success of Scholar Rock’s current and potential future collaborations, Scholar Rock’s dependence on third parties for development and manufacture of product
candidates including, without limitation, to supply any clinical trials, Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business
alliances and new business initiatives, and the impacts of current macroeconomic and geopolitical events, including changing conditions from the COVID-19 pandemic, hostilities in Ukraine, increasing rates of inflation and rising interest
rates, on business operations and expectations, as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, as well as
discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and
should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by
law. Apitegromab and SRK-181 are investigational drug candidates under evaluation. Apitegromab and SRK-181 have not been approved for any use by the FDA or any other regulatory agency and the safety and efficacy of apitegromab and SRK-181
have not been established. © Scholar Rock, Inc. All rights reserved.
Scholar Rock: Transforming Patient Lives, Targeting High Unmet Medical Need 3 Global leader in TGFB
superfamily biology Targeting the latent forms of growth factors Exquisite selectivity to deliver differentiated therapeutic profiles Revolutionary Platform Rich preclinical pipeline focused on high unmet patient needs Phase 3
SAPPHIRE study underway, data readoutexpected in 2024 Phase 1 POC DRAGON study underway in immuno-oncology Neuromuscular and Beyond Compelling proof-of-concept TOPAZ data informed Phase 3 SAPPHIRE study design Seasoned leadership team
with track record of clinical and commercial success Anticipated cash runway into 2025 Positioned for Success Commercial planning underway for apitegromab (SMA) in US and Europe Broad platform, including promising early-stage assets,
provides opportunities to advance alone or in partnership Strategic Optionality
Revolutionary Approach to Regulating TGFβ Superfamily Implicated in Devastating Diseases 4 Scholar
Rock’s R&D Platform Transforming Medical Practice Selectively target the latent form of growth factors in the microenvironment of cells and tissues with uniquely designed antibodies Overcome the challenges that plague traditional
approaches that target the “mature” growth factor, which are difficult to differentiate and lead to unintended negative effects Scholar Rock’s TargetLatent Growth Factor Traditional Target“mature” growth factor TGFβ Superfamily: Highly
Sought-After Targets Dysregulation plays a role in devastating diseases that have a high unmet need including: Neuromuscular disorders Fibrosis Oncology Recognized by the industry as important targets given their fundamental roles in
regulating a variety of cellular processes
DISCOVERY/PRECLINICAL PHASE 1 PHASE 2 PHASE 3 2023 MILESTONES SPINAL MUSCULAR ATROPHYApitegromab
(selective anti-latent myostatin) 36-month TOPAZ data SAPPHIRE: LPI IMMUNO-ONCOLOGYSRK-181 (Selective context-independent, anti-latent TGFβ-1) Rolling clinical data updates ANEMIASelective anti-RGMc IND-enabling
studies FIBROSIS Selective context-dependent (LTBP1 & LTBP3) anti-latent TGFβ-1 IND-enabling studies Robust Pipeline of Novel Product Candidates 5 Potential to transform the lives of patients suffering from a wide range of serious
diseases, including neuromuscular disorders, oncology, and fibrosis
Leadership Team: Experienced in Drug Development and Commercialization 6 Jay Backstrom, MD,
MPH President & CEO 30 years of clinical R&D experience, leading multiple successful regulatory approvals Ted Myles, MBA Chief Operating Officer & CFO 25 years of progressive experience in clinical and commercial-stage
companies 25 years of experience leading HR, culture transformation, leadership development, DEI, and talent management Caryn Parlavecchio Chief Human Resources Officer 15 years of experience leading and advising life sciences companies
in areas of legal and compliance Junlin Ho, JD General Counsel &Corporate Secretary Mo Qatanani, PhD SVP, Research 15 years of industry experience on the strategic and operational sides of research & development Jing Marantz,
MD, PhD, MBA Chief Medical Officer 20 years of industry expertise across clinical pharmacology, neurology, hematology/oncology, and rare diseases
Apitegromab: The Next Potential Transformative Therapy for Patients with Spinal Muscular Atrophy (SMA)
Apitegromab: Potential Muscle-Directed Therapy for SMA 8 * Based on Animal Model Data; 1. Adapted
from: SMA Foundation Overview. http://www.smafoundation.org/wp-content/uploads/2012/03/SMA-Overview.pdf.; Accessed April 18, 2021; 2. Long KK, et al. Hum Mol Genet. 2019;28(7):1077-1088; 3. Pirruccello-Straub M, et al. Sci Reports.
2018;8(1):2292. doi:10.1038/s41598-018-20524-9 Apitegromab is a MUSCLE-DIRECTED APPROACH aimed at improving motor function*2,3 Myostatin is a negative regulator of skeletal muscle growth Apitegromab is a fully human, mAb that
specifically binds to proforms of myostatin and inhibits myostatin activation leading to increased muscle mass and muscle function Strong evidence indicates upstream targeting of structurally differentiated latent myostatin avoids
undesirable off-target effects Apitegromab Muscle fiber atrophy SMN-directed therapiesPREVENT FURTHER DEGENERATION of motor neurons1 ...but do not directly address muscle atrophy Motor neuron degeneration
9 *TOPAZ Phase 2 trial evaluated patients with Type 2 and 3 SMA (did not include Type 1) 1. Lally et
al, Orphanet Journal of Rare Diseases, 2017; 2. SMA Europe. SMATracker. About SMA. Accessed January 24, 2022. https://smatracker.eu/what-is-spinal-muscular-atrophy; 3. National Organization for Rare Disorders. Spinal muscular atrophy.
Accessed January 24, 2022. https://rarediseases.org/rare-diseases/spinal-muscular-atrophy/. 4. Cure SMA. Care Series Booklet. Accessed September 19, 2021. 2020.
https://www.curesma.org/wpcontent/uploads/2020/08/08262020_Understanding_SMA_vWeb.pdf. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any
use by the US FDA or any other health authority, and its safety and efficacy have not been established. Severe, progressive disabilities and unable to walk independently Significant, progressive motor function impairment; many lose
ambulation Infantile onset; unable to sit up independently Type 3 35% Type 2 51% Type 1 14% TOPAZ* 12-month results showed transformative potential in non-ambulatory Types 2 and 3 patients >2/3 of overall patient population KBV
Research and secondary Research Analysis. Global Spinal Muscular Atrophy Market Analysis (2022-2028). November 2022, p. 42 Global SMA Treatment Market expected to reach $11.4B by 2028 Spinal Muscular Atrophy Motor neuron impairment and
loss due to SMN genetic deficiency, leading to muscle atrophy and weakness GLOBAL DISEASE: 30,000-35,000 affected in US and Europe alone1, 2, 3,4
Patients continue to experience major functional impairments despite utilization of SMN-directed
therapies Potential to Pioneer a New Treatment Era: Opportunity for Muscle-Directed Therapy to Complement SMN-Directed Therapies 10 PHASE 3 TRIAL DESIGN Type 1, 2 ,3 1 day -12 years of age (Non-ambulatory recruited) Type 1, 2, 3 1
month – 25 years of age (Ambulatory and Non-ambulatory recruited) Type 1 up to 6 months of age (Non-ambulatory recruited) PRIMARY ENDPOINT Mean change from baseline in HFMSE at 15 months Mean change from baseline in MFM-32 at 12
months Ability to sit independently and event-free survival INITIAL INDICATION† Spinal Muscular Atrophy (SMA) in pediatric and adult patients Spinal Muscular Atrophy (SMA) in pediatric and adult patients Spinal Muscular Atrophy (SMA) in
pediatric patients less than 2 years CURRENT MARKET PENETRATION Patients treated WW: >11,000* Revenues (LTM): $1.7+ billion Patients treated WW: >7000** Revenues (YTD’0922): ~CHF 793 million Patients treated WW: >2500***
Revenues (LTM): $ 1.4+ billion *As of Biogen SPINRAZA website and 3Q22 financial update on 10/25/22; includes patients treated worldwide in post-marketing setting, expanded access program, and clinical trials. **As of Roche YTD Sep’2022
financial update on 10/18/22; includes patients treated worldwide between clinical trials, commercial, and compassionate use program. ***As of Novartis 3Q22 financial update on 10/25/22; commercially, via managed access programs and in
clinical trials HFMSE = Hammersmith Functional Motor Scale Expanded; MFM-32 = Motor Function Measure – 32 items †Refer to most current USPI
Apitegromab Offers Potential to Address Unmet Patient Need 11 HFMSE=Hammersmith Functional Motor
Scale-Expanded 1. Mercuri E et al.; N Engl J Med 2018; 378:625-635; DOI: 10.1056/NEJMoa1710504; cherish trial results. This third-party information is provided for background only and is not intended to convey or imply a comparison to the
TOPAZ clinical trial results. Mean improvement in HFMSE experienced by patients with non-ambulatory Types 2/3 SMA in nusinersen Phase 3 CHERISH trial 3.9-point increase in HFMSE from nusinersen(4.9 point increase relative to sham
control) HFMSE Score at Month 15 Total Possible HFMSE Score of 66 Unmet need remains substantial
Phase 2 TOPAZ Trial: Safety and Efficacy Data from First Muscle-directed Treatment Candidate in SMA
TOPAZ Age 2-12 Analysis* in Pooled Non-Ambulatory Cohorts (20mg/kg) Transformative Potential as Add-On
for Apitegromab1,2 13 Mean HFMSE Increase OF 4.4 POINTS with majority experiencing ≥ 3-point increases on top of background SMN therapy HFMSE Gains Also Notable in subset of individuals in this analysis who had started background
nusinersen at age ≥ 5: 75% (6/8) with ≥ 1-point increase 50% (4/8) with ≥ 3-point increase Non-Ambulatory Types 2/3 SMA (Apitegromab 20 mg/kg; Intent-to-Treat Population) Age 2-12 years (n=16†) Mean HFMSE change from baseline, (95%
CI) +4.4 (1.3, 7.4) Patients with ≥ 1-pt increase in HFMSE, n (%) 13 (81%) Patients with ≥ 3-pt increase in HFMSE, n (%) 9 (56%) TOPAZ results showed HFMSE improvement from baseline or RHS stabilization across all three pre-specified
cohorts.1 *Exploratory, post hoc analysis; †For 12-month endpoint, if patients skipped three consecutive doses due to site restrictions caused by COVID-19, records after dose skipping were excluded from analysis. The last observation carry
forward was used for other missing data; 1.Crawford T et al. TOPAZ topline results; Presented at CureSMA, 2021 Virtual SMA Research & Clinical Care Meeting; June 9-11, 2021. 2. Scholar Rock Inc. Corporate Presentations, August 2022 at
Deep Insights, Impactful Medicines (scholarrock.com) Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other
health authority, and its safety and efficacy have not been established. No safety signals for apitegromab were identified to date; the five most frequently reported treatment-emergent adverse events were headache, pyrexia, upper respiratory
tract infection, cough, and nasopharyngitis
Sizable, Sustained Increases in HFMSE Observed Over 24 Months of ApitegromabPooled Non-Ambulatory
Patients Excluding Data Post Scoliosis Surgery (all dose groups) 14 For the 24-month evaluation, an observed case analysis was conducted, which pooled all the non-ambulatory patients (Cohorts 2 and 3) and was based upon the available data
for given timepoints. This analysis population included patients receiving either low dose (2 mg/kg) or high dose (20 mg/kg) apitegromab (inclusive of patients in Cohort 3 who switched from 2 mg/kg to 20 mg/kg in Year 2). This analysis
excludes from the observed case analysis any HFMSE data following scoliosis surgery in TOPAZ. Of the three non-ambulatory patients who had scoliosis surgery, data from one was excluded and the other two did not have valid HFMSE assessments.
Error bars represent SEM. Values in parentheticals represent 95% confidence interval. Crawford T et al. TOPAZ EXTENSION: 24-MONTH EFFICACY AND SAFETY OF APITEGROMAB IN PATIENTS WITH LATER-ONSET SPINAL MUSCULAR ATROPHY (TYPE 2 AND TYPE 3 SMA)
Podium Presentation Presented at CureSMA; June 2022. Data on File. Scholar Rock, Inc. Cambridge, MA. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been
approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. Age 2-21 Years Age 2-12 Years n= 35 29 32 28 n= 29 23 26 23 Mean Change from Baseline in HFMSE (95% CI)
Continued Increase in RULM Observed at 24 Months of Apitegromab Pooled Non-Ambulatory Patients
Excluding Data Post Scoliosis Surgery (all dose groups) 15 For the 24-month evaluation, an observed case analysis was conducted, which pooled all the non-ambulatory patients (Cohorts 2 and 3) and was based upon the available data for given
timepoints. This analysis population included patients receiving either low dose (2 mg/kg) or high dose (20 mg/kg) apitegromab (inclusive of patients in Cohort 3 who switched from 2 mg/kg to 20 mg/kg in Year 2). This analysis excludes data
from 3 non-ambulatory patients after their scoliosis surgery during TOPAZ from the Observed Case Analysis. Error bars represent SEM. Values in parentheticals represent 95% confidence interval. Crawford T et al. TOPAZ EXTENSION: 24-MONTH
EFFICACY AND SAFETY OF APITEGROMAB IN PATIENTS WITH LATER-ONSET SPINAL MUSCULAR ATROPHY (TYPE 2 AND TYPE 3 SMA) Podium Presentation Presented at CureSMA; June 2022. Data on File. Scholar Rock, Inc. Cambridge, MA. Apitegromab is an
investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been
established. n= 34 28 31 30 n= 28 22 25 25 Mean Change from Baseline in RULM (95% CI) Age 2-21 Years Age 2-12 Years
No Serious Safety Risks IdentifiedOver 24 Months of Apitegromab Treatment 16 Treatment-Emergent
Adverse Events (TEAEs)* 2 mg/kg dose (N=10) n (%) 20 mg/kg dose (N=48) n (%) Total (N=58) n (%) Any TEAE 10 (100) 45 (93.8) 55 (94.8) Any Serious TEAE 3 (30) 11 (22.9) 14 (24.1) Any TEAE leading to study drug
discontinuation 0 (0.0) 1 (2.1) 1 (1.7) Any Grade 3 (severe) or higher TEAE 2 (20) 9 (18.8) 11 (19) Crawford T et al. TOPAZ EXTENSION: 24-MONTH EFFICACY AND SAFETY OF APITEGROMAB IN PATIENTS WITH LATER-ONSET SPINAL MUSCULAR ATROPHY
(TYPE 2 AND TYPE 3 SMA) Podium Presentation Presented at CureSMA; June 2022 *Notes: % = 100 x n/N (n=incidence) **51/57 patients Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that start after the first dose
of study drug or start prior to the administration of study drug and worsen in severity/grade or relationship to investigational medication after the administration of study drug. Data is for safety events collected over the 24-month period
and includes patients who switched from 2 mg/kg to 20 mg/kg. Data on file, extracted on April 7, 2022. Scholar Rock, Inc. Cambridge, MA. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular
atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. Incidence and types of TEAEs were consistent with the underlying disease or nusinersen
therapy Five most frequently reported TEAEs were headache, pyrexia, upper respiratory tract infection, cough, and nasopharyngitis No deaths or Suspected Unexpected Serious Adverse Reactions (SUSARs) reported Adverse events reported as
mostly mild to moderate in severity No identified serious risks as of 4/7/2022 approximately 90% remain on apitegromab as of 12/31/2022**
Sapphire Phase 3 Pivotal Trial
Ongoing SAPPHIRE Phase 3 Trial Overview 18 Randomized, double-blind, placebo-controlled, parallel arm
design (n=204) Enrolling patients on SMN-directed therapy (nusinersen or risdiplam) Anticipate completing enrollment in 2023 TREATMENT (52 weeks) Apitegromab (20 mg/kg IV q4w) + SMN-directed therapy Apitegromab (10 mg/kg IV q4w) +
SMN-directed therapy Placebo (IV q4w) + SMN-directed therapy SCREENING MAIN POPULATION (n=156) Ages 2-12 With non-ambulatory Types 2 and 3 SMA N=52 N=52 N=52 R Stratified randomization to ensure balanced allocation: Age at SMN
therapy initiation(age < 5 vs age ≥ 5) SMN therapy (nusinersen vs. risdiplam) ENDPOINTS Primary Efficacy: Mean HFMSE change from baseline at 12 months Additional Efficacy Measures: RULM, WHO, other outcome measures Safety, PK/PD,
ADA Additional Data Opportunities Exploratory population (age 13-21), in patients using SMN therapy Focused upon safety & exploratory efficacy (n=48; 2:1 randomization between apitegromab 20 mg/kg vs placebo) Separate open-label
extension study (after patients complete 12-month treatment period) Focused upon safety & exploratory long-term efficacy ClinicalTrials.gov Identifier: NCT05156320 R=Randomization
2023 enrollment 2024 data 2025 launch Executing on the Promise: Apitegromab SMA Trials 19 WHERE
WE WERE Phase 2 12-Month COMPLETE 24-Month EXTENSION COMPLETE WHERE WE AREPhase 3 WHERE WE PLAN TO GO 36-Month EXTENSION IN PROCESS 12-Month IN PROCESS Ambulatory patients Type 1 patients (including those treated with gene
therapy, under 2 years of age) Long-term EXTENSION
SRK-181: Potential Transformative Backbone for a New Era ofCancer Immunotherapy
Resistance to Checkpoint Inhibitor (CPI) Therapies Remains a Significant Clinical Challenge 21 1.
Source: Gores, M. (2022). In the eye of the storm: PD-(L)1 inhibitors weathering turbulence [White paper]. IQVIA. https://www.iqvia.com/library/white-papers/in-the-eye-of-the-storm-pd-l-1-inhibitors-weathering-turbulence 2. Source:
Carretero-Gonzalez et al. (2018) Oncotarget 9:8706-8715 Meta-analysis of twelve randomized trials with control arm or adequate safety profile (includes nivolumab, pembrolizumab, and atezolizumab) Clinically derived rationale points to
significant opportunity to increase checkpoint therapy responses by targeting TGFβ-1 7.9-10.41MILLION US patients on CPI therapies First-lineCPI therapy Second-line(or later) CPI therapy DID NOT respond 63% 2 78% 2
Strong Scientific Rationale for the Role of TGFβ Inhibition in Immuno-Oncology 22 “Bristol Myers
Squibb Enters Agreement to Acquire Forbius TGF-beta Program” August 2022. “Merck to Acquire Tilos Therapeutics: Merck Gains Portfolio of Investigational Antibodies Modulating TGFβ” $773 million total potential deal value June
2019. Selective inhibition of TGFβ-1 activation overcomes primary resistance to checkpoint blockade therapy by altering tumor immune landscape Science Translational Medicine, March 25, 2020. Constance J. Martin, et al. Vol 12, Issue 536.
DOI: 10.1126/scitranslmed.aay8456 Nature (online), February 14, 2018. TGFβ attenuates tumour response to PD-L1 blockade by contributing to exclusion of T cells Sanjeev Mariathasan1*, Shannon J. Turley1*, Dorothee Nickles1*, Alessandra
Castiglioni1, Kobe Yuen1, Yulei Wang1, Edward E. Kadel III1, Hartmut Koeppen1, Jillian L. Astarita1, Rafael Cubas1, Suchit Jhunjhunwala1, Romain Banchereau1, Yagai Yang1, Yinghui Guan1, Cecile Chalouni1, James Ziai1, Yasin Şenbabaoğlu1,
Stephen Santoro1, Daniel Sheinson1, Jeffrey Hung1, Jennifer M. Giltnane1, Andrew A. Pierce1, Kathryn Mesh1, Steve Lianoglou1, Johannes Riegler1, Richard A. D. Carano1, Pontus Eriksson2, Mattias Höglund2, Loan Somarriba3, Daniel L.
Halligan3, Michiel S. van der Heijden4, Yohann Loriot5, Jonathan E. Rosenberg6, Lawrence Fong7, Ira Mellman1, Daniel S. Chen1, Marjorie Green1, Christina Derleth1, Gregg D. Fine1, Priti S. Hegde1, Richard Bourgon1 & Thomas
Powles8 Willy Hugo, Jesse M. Zaretsky, Lu Sun, Douglas B. Johnson, Antoni Ribas, Roger S. Lo Volume 165, Issue 1, 24 March 2016, Pages 35-44 Genomic and Transcriptomic Features of Response to Anti-PD-1 Therapy in Metastatic Melanoma July
24, 2020: https://doi.org/10.1038/ s41571-020-0403-1 Nature Reviews , July 24, 2020 NATURE REVIEWS | CLINICAL ONCOLOGY TGFβ: biology in cancer progression and immunotherapy Rik Derynck1,2,3, Shannon J. Turley4 and Rosemary J.
Akhurst2,3
SRK-181: Unique Latent TGFβ-1 Selective Approach to Overcoming Checkpoint Inhibitor
Resistance 23 Traditional Target“Mature” growth factor 1. Wakefield LM, Winokur TS, Hollands RS, Christopherson K, Levinson AD, Sporn MB. Recombinant latent transforming growth factor beta 1 has a longer plasma half-life in rats than
active transforming growth factor beta 1, and a different tissue distribution. J Clin Invest. 1990 Dec;86(6):1976-84. doi: 10.1172/JCI114932. PMID: 2254455; PMCID: PMC329834. SRK-181: Latent TGFβ-1 Inhibitor Targets TGFβ-1 Potential to
overcome CPI resistance SRK-181 inhibits the TGFβ-1 implicated in check point inhibitor resistance Selective to β-1 isoform Highly selective to β-1 isoform vs. 2 and 3 Increases therapeutic window and potentially avoids toxicities
associated with non-selective TGFβ inhibition Other programs target multiple isoforms of TGFβ Context-independent Inhibits all sources of TGFβ-1 SRK-181 targets all TGFβ-1 sources (LRRC33, GARP and LTBP1 and 3) Some programs only target
one source Targets the latent form of TGFβ-1 Selectively targeting the latent form shuts off the growth factor before activation Increases opportunity to inhibit TGFβ-1 Most other programs target the mature form of TGFβ-1
SRK-181-mIgG1 + Anti-PD1 Overcomes Immune Exclusion 24 Anti-PD1/ SRK-181-mIgG1 Anti-PD1 Overcoming
immune exclusion Tumor micro-environment Preclinical data published in Science Translational Medicine. Martin CJ, et al. Sci Transl Med. 2020 Mar 25;12(536):eaay8456. https://scholarrock.com/platform/publications. Data from MBT-2 syngeneic
tumor model. Dose 10mg/kg QW for 4 weeks. Overcome Exclusion SRK-181-mIgG1 combination therapy led to influx and amplication of cytotoxic CD8+ cells in preclinical bladder tumor model Immune Exclusion
DRAGON Phase 1 POC Trial to Evaluate SRK-181’s Ability to Overcome Primary Resistance to Checkpoint
Inhibitors 25 * A cohort of 2000 mg Q2W (n=3) was also evaluated. **The clear cell RCC cohort will also explore the effects of SRK-181 in patients with relapsed response after anti-PD-(L)1 treatment. 1. NCT04291079 on
www.clinicaltrials.gov. PART B SRK-181 + anti-PD-(L)1; non-responders to prior anti-PD-(L)1 SRK-181 + pembrolizumab Non-small cell lung cancer SRK-181 + pembrolizumab Urothelial carcinoma SRK-181 + pembrolizumab Cutaneous
melanoma SRK-181 + pembrolizumab Clear cell renal cell carcinoma** SRK-181 + pembrolizumab Head and neck small cell carcinoma COHORT TREATMENT PART A SRK-181 80 mg (n=1) SRK-181 240 mg (n=1) SRK-181 800 mg (n=3) SRK-181 1600 mg
(n=3) SRK-181 2400 mg (n=3) SRK-181 3000 mg (n=3)* SRK-181 all-comers A1 A2 SRK-181+anti-PD-(L)1; non-responders to prior anti-PD-(L)1 SRK-181 240 mg (n=3) SRK-181 800 mg (n=3) SRK-181 1600 mg (n=3) SRK-181 2400 mg (n=3)
DRAGON Part A: Safety 26 PART A1 Treatment-Emergent AEs Related to SRK-181, All Grades >10%
PART A2 Treatment-Emergent AEs Related to SRK-181 or Anti-PD(L)1, All Grades >10% Dose (MG) 80N=1 240N=1 800N=3 1600N=4 2400N=3 3000N=3 2000N=4 AllN=19 Fatigue 0 1 0 0 1 0 1 3 (15.8%) Decreased
Appetite 1 0 1 0 0 0 0 2 (10.5%) Nausea 1 0 0 0 0 0 1 2 (10.5%) Dose (MG) 240N=3 800N=3 1600N=6 2400N=3 AllN=19 Pruritis 1 0 1 1 3 (20.0%) Rash 0 1 0 2 3 (20.0%) Rash maculo-papular 1 0 1 1 3
(20.0%) Diarrhea 0 0 2 0 2 (13.3%) Yap T et al. SRK-181, a latent TGFβ1 inhibitor: safety, efficacy, and biomarker results from the dose escalation portion of a phase I trial (DRAGON trial) in patients with advanced solid tumors
(Poster 780); Presented at SITC; Nov. 10-11, 2022. Clinical cutoff date: August 29, 2022. All dose levels were administered q3w except 2000 mg, which was administered q2w. SRK-181 is an investigational drug candidate that is being evaluated
for the treatment of cancer. SRK-181 has not been approved by the US FDA or any other health authority, and its safety and efficacy have not been established. Treatment-related Grade 3 AEs: Alanine aminotransferase increased (1
patient) Treatment-related SAE were elevated troponin I (1 patient, at 2000 mg q2w) No DLTs were observed up to 3000 mg q3w and 2000 mg q2w No Grade 4 or 5 treatment-related AEs occurred Treatment-related Grade 3 AEs: Puritus (2
patients), blister, immune-mediated lung disease, rash and rash maculo-popular(1 patient each) Treatment-related SAEs: Blister, pruritus, and rash (all in 1 patient) and immune-mediated lung disease (1 patient) No DLTs were observed up to
2400 mg q3w No Grade 4 or 5 treatment-related AEs occurred
X X X X X X X X X X X X X X X X X X X Ovarian Ovarian Ovarian Pancreatic Pancreatic Colorectal Colorectal Colorectal Colorectal Colorectal Colorectal Testicular Prostate Prostate Prostate Prostate Primary
Unknown Primary Unknown Head and Neck Months 0 1 2 3 4 5 6 7 8 9 10 11 12 X X X X X X X X X X X X X X Head and Neck Head and Neck RCC Melanoma Squamous Cell Skin Carcinoma RCC Melanoma TNCB Head and
Neck Liver NSCLC RCC RCC Uveal Melanoma Primary Unknown Progressed on Prior Anti-PD-(L)1 Duration of Treatment DRAGON Part A: Preliminary Efficacy Data* Presented at SITC November 2022 27 Part A1 (n=19) Part A2 (n=15) 8 patients
had a best response of stable disease (SD) All 3 patients with ovarian cancer were stable beyond the 16-week cutoff Yap T et al. SRK-181, a latent TGFβ1 inhibitor: safety, efficacy, and biomarker results from the dose escalation portion
of a phase I trial (DRAGON trial) in patients with advanced solid tumors (Poster 780); Presented at SITC; Nov. 10-11, 2022. *Preliminary anti-tumor effects were assessed using RECIST1.1 and reported based upon local investigator reads: as of
August 29, 2022. SRK-181 is an investigational drug candidate that is being evaluated for the treatment of cancer. SRK-181 has not been approved by the US FDA or any other health authority, and its safety and efficacy have not been
established. At 800 mg q3w, 1 partial response (PR) was observed in patient with anti-PD-1-resistant clear cell renal cell carcinoma (ccRCC) 9 patients had best response of SD 6 patients (green highlight) were stable beyond the 16-week
cutoff 1 ongoing patient with head and neck cancer had a 29.4% tumor reduction Clinical cutoff date: 29AUG2022 Best Response of PR Best Response of SD Best Response of PD Not Evaluated Partial Response Progressive
Disease Ongoing Discontinued X Assessments: 2400 mg q3w 2400 mg q3w 240 mg q3w 2000 mg q2w 2000 mg q2w 2400 mg q3w 2000 mg q2w 800 mg q3w 1600 mg q3w 2400 mg q3w 3000 mg q3w 1600 mg q3w 3000 mg q3w 80 mg q3w 1600 mg
q3w 800 mg q3w 3000 mg q3w 1600 mg q3w 2000 mg q2w 240 mg q3w 2400 mg q3w 800 mg q3w 1600 mg q3w 2400 mg q3w 1600 mg q3w 800 mg q3w 1600 mg q3w 1600 mg q3w 800 mg q3w 240 mg q3w 2400 mg q3w 1600 mg q3w 240 mg q3w 1600 mg
q3w
Preliminary Efficacy Data in Combination with Pembrolizumab: Best Response in Target Lesions 28 Best
Response in Target Lesions Best Response of PR Best Response of SD Best Response of PD Ongoing Percent Change from Baseline 800 mg q3w 800 mg q3w 1600 mg q3w 1600 mg q3w 1600 mg q3w 1600 mg q3w 2400 mg q3w 2400 mg q3w 2400 mg
q3w 240 mg q3w 240 mg q3w 800 mg q3w Head and Neck Head and Neck Head and Neck Uveal Melanoma NSCLC Melanoma Melanoma Liver RCC RCC TNBC Squamous Cell Skin Carcinoma Part A2 Yap T et al. SRK-181, a latent TGFβ1 inhibitor:
safety, efficacy, and biomarker results from the dose escalation portion of a phase I trial (DRAGON trial) in patients with advanced solid tumors (Poster 780); Presented at SITC; Nov. 10-11, 2022. *Clinical cutoff date: August 29,
2022. Response is assessed using RECIST v1.1 by PI; the scan is performed during screening, 6 weeks after first dose, every 9 weeks for the next 6 months of treatment, and every 12 weeks thereafter. SRK-181 is an investigational drug
candidate that is being evaluated for the treatment of cancer. SRK-181 has not been approved by the US FDA or any other health authority, and its safety and efficacy have not been established. Part B (as of 8/29/22) 14 patients
enrolled One additional confirmed PR ongoing patient with anti-PD-1 resistant clear cell renal cell carcinoma All dose levels were generally well tolerated including recommended SRK-181 dose of 1500 mg q3w or 1000 mg q2w in combination
with anti‑PD-(L)1 for Part B
SRK-181: Encouraging Early Clinical Data Consistent with Hypothesis 29 TGFβ pathway evaluation
(PD) Target engagement (blood) TGFβ-1 signaling (tumor p-SMAD2 & RNAseq) Immunophenotyping, including immune exclusion status Tumor immune contexture (e.g., tumor CD8+ T cells) Immune cell contexture (tumor & blood
MDSC’s) Immune response markers (e.g., IO gene signature) Therapeutically relevant dose Drug exposure needed for efficacy Objective response Anti-tumor response and survival benefits TGFβ pathway evaluation (PD) Target engagement
(blood) TGFβ-1 signaling (tumor p-SMAD2 & RNAseq) Immunophenotyping, including immune exclusion status Tumor immune contexture (e.g., tumor CD8+ T cells) Immune cell contexture (tumor & blood MDSC’s) Immune response markers
(e.g., IO gene signature) Therapeutically relevant dose Dosing regimens achieved target steady state levels Objective response Anti-tumor response observed (partial responses) Preclinical Data Phase 1 DRAGON proof-of-concept trial
30 *(PD-1/PD-L1) First in class monoclonal antibody targeting latent and context-independent binding
to TGFβ1 Differentiated from other TGFβ inhibitors by its novel selectivity Offers potential to avoid toxicity and dose-limiting challenges of non-selective TGFβ inhibition approaches Differentiation Emerging evidence implicates TGFβ1 as
driving resistance to checkpoint inhibitor therapies Potent and selective inhibitor of latent TGFβ1 activation in preclinical studies Strong safety and preclinical efficacy data Strong Scientific Rationale DRAGON Part A demonstrated
ability to escalate to high doses of SRK-181 at levels exceeding the anticipated efficacious drug exposure level Advanced to DRAGON Part B: Evaluating SRK-181 in 5 parallel tumor-specific cohorts, with efficient path towards early POC for
each Early efficacy signals have been observed Clear Clinical Pathway PD-(L)1* becoming a standard of care therapy in many tumor types; the market for synergistic combination product would be vast SRK-181 could potentially be used in
many tumor types, potentially both in patients resistant to PD-(L)1 and in CPI naïve patients, as well as other therapeutic applications High Unmet Medical Need & Large Commercial Opportunity SRK-181Summary
Next Horizon: Fibrosis
TGFβ is Established as Key Driver of Fibrosis Across Multiple Diseases 32 Nature Reviews , April 25,
2016 NATURE REVIEWS | NEPHROLOGY TGF-β: the master regulator of fibrosis Xiao-ming Meng1, David J. Nikolic-Paterson2 and Hui Yao Lan3 Nature Reviews. August 19, 2014 NATURE REVIEWS | RHEUMATOLOGY Transforming growth factor β―at the
centre of systemic sclerosis Robert Lafyatis Int. J. Mol. Sci. August 27, 2018 Targeting TGF-β Signaling in Kidney Fibrosis Yoshitaka Isaka J. Am. Soc. Nephrol. December 3, 2017 Targeting Anti-TGF-β Therapy to Fibrotic Kidneys with a
Dual Specificity Antibody Approach Steve McGaraughty,* Rachel A. Davis-Taber,† Chang Z. Zhu,* Todd B. Cole,* Arthur L. Nikkel,* Meha Chhaya,† Kelly J. Doyle,* Lauren M. Olson,* Gregory M. Preston,† Chrisine M. Grinnell,† Katherine M.
Salte,* Anthony M. Giamis,* Yanping Luo,* Victor Sun,† Andrew D. Goodearl,† Murali Gopalakrishnan,* and Susan E. Lacy† J Pathol, July 25, 2021 TGF-β as a driver of fibrosis: physiological roles and therapeutic opportunities Erine H
Budi1, Johanna R Schaub1, Martin Decaris1, Scott Turner1, Rik Derynck2 J Receptors Sign Trans, Feb 13, 2020 Inevitable role of TGF-β in progression of nonalcoholic fatty liver disease Bhagyalakshmi Nair and Lekshmi R. Nath PNAS, February
24, 1986 Transforming growth factor type β: Rapid induction of fibrosis and angiogenesis in vivo and stimulation of collagen formation in vitro ANITA B. ROBERTS* MICHAEL B. SPORN*, RICHARD K. ASSOIAN*, JOSEPH M. SMITH*, NANETTE S. ROCHE*,
LALAGE M. WAKEFIELD*, URSULA I. HEINE*, LANCE A. LIOTTA*, VINCENT FALANGA†, JOHN H. KEHRL‡, AND ANTHONY S. FAUCI‡ Proc Am Thorac Soc, July 3, 2006 Transforming Growth Factor β A Central Modulator of Pulmonary and Airway Inflammation and
Fibrosis Dean Sheppard
GARP Presents TGFβ on Tregs LRRC33 Presents TGFβ on Macrophages LTBP1 & 3 Present TGFβ in
Connective Tissue Targeting Presenting Molecule/TGFβ-1 Complexes Provides Context Specificity Targeting Latent TGFβ-1 Complexes Creates Multiple “Handles” For Selectivity 33 Latent TGF-β GARP Treg Integrin expressing
cell avb8 Integrin Latent TGF-β LRRC33 Macrophage Integrin expressing cell avb8 Integrin Latent TGF-β binding protein (LTBP) Extracellular matrix Fibrillin Latent TGF-β Epithelial Cell avb6 Integrin Latent TGFβ-1
LTBP-49247 Reduces TGFβ Signaling and Fibrosis in Preclinical Models of Kidney Fibrosis 34 LTBP-49247
reduces a TGFβ PD biomarker in kidneys of Col4a3 KO mice (Alport Syndrome model) LTBP-49247 reduces fibrosis in kidneys of Alport model ** p < 0.01 One way ANOVA vs. IgG HYP=hydroxyproline Efficacy also seen in rat model of kidney
fibrosis No observed toxicity in mouse 13-week non-GLP repeat dose study Favorable PK in cynomolgus monkeys (t1/2 ~28 days) suggests LTBP-49247 is amenable to clinical subcutaneous dosing with promising developability profile
Significant Opportunities to Address High Unmet Need Across Multiple Fibrotic Indications 35 Alport
Syndrome (AS) Focal Segmental Glomerulosclerosis (FSGS) IgA Nephropathy (IgAN) Primary Sclerosing Cholangitis (PSC) Diffuse Cutaneous Systemic Sclerosis (dcSSc) Idiopathic Pulmonary Fibrosis (IPF) Collectively, significant commercial
potential given large patient population with clear high unmet need given poor outcomes and lack of effective therapeutics Significant impact to delay or stop progression to end-stage disease and organ transplant Expansion opportunities
via other indications given shared etiologies
Next Horizon: Iron-Restricted Anemia
BMP6/RGMc Pathway is a Well Validated Regulator of Systemic Iron Homeostasis Fig: Muckenthaler, M.U.,
Rivella, S., Hentze, M.W. and Galy, B. (2017) A Red Carpet for Iron Metaboism. Cell, 168(3): 344-361 1: Kuns-Hashimoto R, et al. (2008) Selective binding of RGMc/hemojuvelin, a key protein in systemic iron metabolism, to BMP-2 and neogenin.
Am J Physiol Cell Physiol 294(4):C994-C1003 2: Constante M, et al.. (2007) Repression of repulsive guidance molecule C during inflammation is independent of Hfe and involves tumor necrosis factor-alpha. Am J Pathol 170(2):497-504 3: Core
A.B., et al. (2014) Hemojuvelin and bone morphogenetic protein (BMP) signaling in iron homeostasis. Front Pharmacol. 5:104. 4. Wang CY and Babitt JL. (2016) Hepcidin Regulation in the Anemia of Inflammation. Curr Opin Hematol 23(3):
189-197. Elevation proinflammatory cytokines drives increased hepcidin expression and results in anemia due to functional iron deficiency4 Anemia of Inflammation/ Chronic Disease Hepcidin Serum iron Untreated Hepcidin Serum iron Ab
treated Human mutations in HJV/RGMc establish it as a central player in hepcidin regulation1 Knockout phenotypes and tissue-specific expression pattern demonstrate that its predominant role is in iron homeostasis2 Member of repulsive
guidance molecule (RGM) family (RGMa, RGMb, RGMc/HJV) that act as BMP co-receptors to modulate BMP signaling3 HJV/RGMc is a key player in the regulation of hepcidin expression 37
HJV-35202: A High-Affinity Antibody Demonstrating Selective Inhibition of HJV/RGMc and Robust PK/PD in
Cynos 38 Potent in vitro binding affinity (KD=3.9E-11) Highly specific to RGMc/HJV with well understood mechanism Sustained PD effect in single dose Cyno study Specific to RGMc over other RGM family members Nicholls S.B., et al. Poster:
RGMc-selective antibodies modulate iron homeostasis in vivo, 12th International BMP Conference, Tokyo, October 2018 Scholar Rock, Data on File High-affinity antibody Specific to RGMc, with mechanism of specificity
understood Cross-reactive to human, mouse, rat and cyno Sustained PD observed in healthy rats and cynos, with clear PK/PD relationship Highly manufacturable framework with no sequence liabilities Formulatable into a subcutaneous format
(150 mg/mL) HJV-35202 (mg/mL) Serum Iron and UIBC (mg/dL) PK: HJV-35202 (10mg/kg, IV) PD: UIBC: HJV-35202 (10mg/kg, IV) PDF: Serum Iron: HJV-35202 (10mg/kg, IV) Octet Response Units RGMa RGMb RGMc/HJV RGMa RGMb RGMc/HJV Key
Attributes of HJV-35202:1,2
Significant Opportunities to Target Iron-Restricted Anemias Across Multiple Indications 39 Chronic
Kidney Disease (CKD) Anemia of Chronic Inflammation (AI) Myelofibrosis (MF) Targeting RGMc/HJV for anemia is well validated and relatively de-risked High levels of hepcidin, the main regulator of systemic iron metabolism, is associated
with anemia across various diseases Safe and convenient RGMc inhibitor has promise of improving patient outcomes across multiple indications as stand alone or in combination with SoC Significant and clear unmet need given lack of approved
treatments or severe limitations of current treatments Well defined patient population Collectively, sizeable commercial opportunity given relatively large population Potential for rapid with clear regulatory path Opportunity to build an
anemia franchise with initial POC and indication expansion in the future
Scholar Rock Summary
41 Key Investment Highlights Revolutionary Platform Overcome the Challenges targeting the latent
forms of growth factors Discover and Develop monoclonal antibodies with extraordinary selectivity Positioned For Success Upcoming Data Readouts for both clinical programs $205M financing in June 2022 Year end cash balance of $315M,
anticipated runway into 2025 Robust Clinical Pipeline SRK-181 (Phase 1) Potential to shift current treatment landscape for cancer patients with CPI resistance Apitegromab (Phase 3) Potential first-in-class Significant market opportunity
Program on track Clear path to approval Discovery-stage Pipeline Fibrosis and iron-restricted anemia Strategic optionality
Appendix
Plateau of HFMSE increases observed following initial treatment effect of risdiplam, although longer
timeframes currently under investigation HFMSE=Hammersmith Functional Motor Scale-Expanded. Mercuri E, et al. Presented at: World Muscle Society Congress 2020, P. 257 Oskoui M, et al. Presented at: 2021 Muscular Dystrophy Association
Clinical & Scientific Conference; March 15-18, 2021. Poster 80. * Overall population 2-25 years old. This third-party information is provided for background only and is not intended to convey or imply a comparison to the TOPAZ clinical
trial results. Plateauing of HFMSE increases observed following initial treatment effects for nusinersen Motor Gains in Patients with Types 2 and 3 SMA on SMN Therapies Appear to Plateau After Initial
Gains 43 Risdiplam2* Nusinersen1 Mean (+SE) Change in HFMSE Total Score From Baseline Analysis Visit, days Initial Treatment CHERISH Chronic Maintenance Phase SHINE 1 92 169 253 350 450 690 930 1170 1410 1650 Mean Change
From Baseline in HFMSE Total Score Visit, months 0 4 8 12 18 24 Risdiplam Placebo <1-point increase in HFMSE after ~ first year of treatment 3.9 Points 4.6 Points Unmet Need for Motor Function Gains
1. Mercuri E, et.al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J
Med. 2018;378:625-635. 2. Efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA) Roche/PTC Therapeutics This third-party information is provided for background only and is not
intended to convey or imply a comparison to the TOPAZ clinical trial results Nusinersen CHERISH Trial in Later-Onset SMA1 In patients with later-onset SMA who were age >5 at screening: Primary benefit of nusinersen: stabilization of
motor function Majority of patients do not experience HFMSE increases Non-Ambulatory Type 2/3 SMA: Majority of Patients Started on SMN Therapy After Age 5 Do Not Experience Motor Function Increases 44 Risdiplam SUNFISH Trial in
Later-Onset SMA2 Low percentage of patients over the age of 5 achieved ≥3-point increase on MFM32 scale, even with risdiplam treatment HFMSE secondary endpoint showed a mean 0.58-point improvement over placebo (not statistically
significant) Change From Baseline to 15 Months in HFMSE Score Age, year 2 4 6 8 Nusinersen (N=66) Control (N=34) % of patients with > 3 change in MFM32 total at Month 12 Change in MFM32 total score 2-5 Years 6-11
Years 12-17 Years 18-25 Years Risdiplam Placebo
SAPPHIRE Phase 3 Design is Optimized by Insights from TOPAZ 45 Largest HFMSE gains observed in the
non-ambulatory Type 2/3 SMA cohorts Exploratory age 2-12 analysis in non-ambulatory Type 2/3 showed transformative potential HFMSE gains evident by 12 months of treatment Dose response seen (greater effect observed with 20 mg/kg over 2
mg/kg) Study population: Non-ambulatory Type 2/3 SMA Primary efficacy endpoint: HFMSE Age 2-12 main efficacy population 12-month treatment duration 20 mg/kg apitegromab dose Also evaluating 10 mg/kg arm (to explore potential that dose
between 2 and 20 mg/kg may be comparable to 20 mg/kg) TOPAZ Learnings SAPPHIRE Design Elements
TOPAZ Subject Disposition, Demographics and Baseline Characteristics1,2 46 AMBULATORY
PATIENTS NON-AMBULATORY PATIENTS COHORT 1 COHORT 2 COHORT 3 20 mg/kg monotherapy 20 mg/kg + nusinersen 20 mg/kg + nusinersen 2 mg/kg + nusinersen 20 mg/kg + nusinersen N (dosed) 11 12 15 10 10 Mean age at screening (min,
max) 12.1 (7, 19) 13.1 (7, 21) 11.7 (8, 19) 4.1 (2, 6) 3.8 (2, 6) Mean age at SMA diagnosis (min, max) 5.9 (2, 15) 4.5 (2, 15) 3.1 (1, 16) 1.2 (1, 2) 1.2 (1, 3) Female (%) 73% 58% 53% 30% 50% SMN2 Gene Copy* (#, %) 2 1
(9%) 0 (0%) 1 (10%) 1 (10%) 3 4 (36%) 9 (75%) 11 (73%) 8 (80%) 8 (80%) 4 4 (36%) 1 (8%) 2 (13%) 1 (10%) 0 (0%) # of maintenance doses of nusinersen at baseline (min, max) N/A 5.6 (2, 8) 5.1 (2, 9) 5.5 (2, 9) 5.4 (3,
8) Discontinuation(s) 0 2† 1† 0 0 Scoliosis (#, %) 7 (63.6) 4 (33.3) 11 (73.3) 4 (40%) 3 (30%) Contracture(s) (#, %) 6 (54.5) 7 (58.3) 13 (86.7) 8 (80%) 4 (40%) Mean RHS score (min, max) 47.6 (26, 63) 51.3 (43, 62) Mean
HFMSE score (min, max) 22.7 (13, 39) 26.1 (12, 44) 23.5 (14, 42) *1 patient answered 3-4, 1 patient answered >4 , both patients are in Cohort 1 treated with 20 mg/kg + nusinersen; data not available for all patients. †1 cohort 1
patient discontinued study in 12M Treatment Period, 1 cohort 1 patient and 1 cohort 2 patient discontinued during 24M Extension Period A. All discontinuations were for reasons unrelated to study drug. HFMSE=Hammersmith Functional Motor
Scale Expanded; RHS=Revised Hammersmith Scale. Crawford T et al. Presented at: 2022 Annual SMA Conference; June 16-19, 2022; Anaheim, CA. 2. Data on file; Scholar Rock. 2022.
Non-Ambulatory Type 2 High Dose Cohort: Initiated nusinersen age <51,2 47 Sizable increases in
HFMSE observed in patients already treated with chronic maintenance nusinersen Improved: 88% (7/8) ≥ 5-point increase: 63% (5/8) > 10-point increase: 38% (3/8) Continuous and durableimprovements observed through12-months of
treatment Apitegromab (20 mg/kg) + nusinersen n=8* Mean change from baseline in HFMSE (95% CI) +7.1 (1.8, 12.5) # (%) patients achieving: ≥ 1-pt increase in HFMSE 7/8 (88%) ≥ 3-pt increase in HFMSE 5/8 (63%) ≥ 5-pt increase in
HFMSE 5/8 (63%) Baseline characteristics: mean (min, max) n=10 Age 3.8 (2, 6) HFMSE score 23.5 (14, 42) # of nusinersen maintenance doses 5.4 (3, 8) *This was a primary intent-to-treat (ITT) analysis that, as prespecified, excluded
2 patients who missed 3 doses due to COVID-19 related site access restrictions. An all-patients sensitivity analysis that included those 2 patients had similar results as this primary ITT analysis. Crawford T et al. TOPAZ topline results;
Presented at CureSMA, 2021 Virtual SMA Research & Clinical Care Meeting; June 9-11, 2021. 2. Data on file; Scholar Rock. 2022. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy.
Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established.
Non-Ambulatory Type 2/3 Cohort: Initiated nusinersen age ≥51,2 48 Majority of patients improved in
HFMSE (despite initiating background nusinersen age ≥ 5) ≥ 1-point increase: ~67% ≥ 3-point increase: ~30% Durability of effect observed through 12-months of treatment *Intent-to-treat analysis excluded 1 patient (per prespecified
approach) who missed 3 doses due to COVID-19 related site access restrictions; 1 patient who had inadvertently been enrolled who was receiving (and continued to receive) an acetylcholinesterase inhibitor was removed, which is not permitted
per the trial protocol; 1. Crawford T et al. TOPAZ topline results; Presented at CureSMA, 2021 Virtual SMA Research & Clinical Care Meeting; June 9-11, 2021. 2. Data on file. Scholar Rock, Inc. Apitegromab is an investigational drug
candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. Apitegromab (20 mg/kg)
+ nusinersen Per Protocol Population* (n=13) Intent-to-Treat Population (n=14) Mean change from baseline in HFMSE (95% CI) +1.2 (-0.5, 2.9) +0.6 (-1.4, 2.7) # (%) patients achieving: ≥ 1-pt increase in HFMSE 9/13 (69%) 9/14
(64%) ≥ 3-pt increase in HFMSE 4/13 (31%) 4/14 (29%) ≥ 5-pt increase in HFMSE 2/13 (15%) 2/14 (14%) Baseline characteristics: mean (min, max) n=15 Age 11.7 (8, 19) HFMSE score 22.7 (13, 39) # of nusinersen maintenance doses 5.1
(2, 9)
TOPAZ Topline 12-Month Data Showed Apitegromab’s Transformative Potential in Patients with Type 2/3
SMA 49 Crawford T et al. TOPAZ topline results; Presented at CureSMA, 2021 Virtual SMA Research & Clinical Care Meeting; June 9-11, 2021 * Pooled cohorts of non-ambulatory patients treated with apitegromab 20 mg/kg and 2
mg/kg **Non-ambulatory patients who initiated background nusinersen at a young age of <5 years and treated with apitegromab 20 mg/kg dose. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal
muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. HFMSE change from baseline Majority of non-ambulatory patients* experienced
HFMSE increases from apitegromabduring chronic maintenance phase of SMN therapy Apitegromab led to HFMSE improvements in both non-ambulatory cohortsincluding patients started on nusinersen at age ≥ 5 Initiated background nusinersen Age
< 5** Age ≥ 5 Mean HFMSE Increase +7.1 points +0.6 points ≥ 1-point Increase % (n/N) 88% (7/8) 64% (9/14) ≥ 3-point Increase% (n/N) 63% (5/8) 29% (4/14)
*Pooled cohorts of non-ambulatory patients treated with apitegromab 20 mg/kg and 2 mg/kg; excludes 4
patients who each missed 3 doses of apitegromab due to COVID-19-related site access restrictions and were not included in the primary (intent-to-treat) analysis. Data on file. Scholar Rock, Inc. Cambridge, MA. Apitegromab is an
investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been
established. HFMSE change from baseline Youngest (2 years) Oldest (19 years) Age of patient at baseline in TOPAZ HFMSE Improvements Observed Across Age Range of Non-Ambulatory Patients with Relatively Larger Gains from Earlier
Treatment 50
WHO Motor Development Milestone Achievements Further Support Apitegromab’s Potential to Improve Motor
Function1 51 Non-ambulatory Type 2/3 Patients # of patients gaining ≥ 1 WHO motor milestone(s) Pooled 7/35 Initiated nusinersen age < 5 4/20 Initiated nusinersen age ≥ 5 3/15 WHO motor milestone analysis included all patients
who completed the 12-month treatment period, including 4 patients who missed 3 doses of apitegromab due to COVID-19-related site access restrictions. Median baseline score for both non-ambulatory cohorts was 1.0. Pictures are not of
patients with SMA and are not meant to be representative of patients with SMA. 1. Crawford T et al. TOPAZ topline results; Presented at CureSMA, 2021 Virtual SMA Research & Clinical Care Meeting; June 9-11, 2021 1 patient (initiated
nusinersen age ≥5) gained 2 new motor milestones 1 patient (initiated nusinersen age <5, 20 mg/kg) gained 3 new motor milestones Sitting without support Standing with assistance Walking with assistance Walking alone Following 12
months of apitegromab treatment... Hands & knees crawling Standing alone
Significance of Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Limb Module
(RULM) 52 HFMSE Assesses the physical abilities of patients with Types 2/3 SMA ABLE TO: Touch Head Above Ear Level whilst maintaining stable trunk and head Roll From Supine to Prone over the right side without pulling/ pushing on
hands 33 Items Graded on scale 0 to 2 0 = unable 1 = performed with modification or adaptation 2 = without modification or adaptation Item scores are summed to give a total score The higher the total score, the greater the patient’s
motor function Maximum score: 66 Examples of items: One hand to head in sitting Rolls supine to prone Lying to sitting Four–point kneeling Supported standing Stepping Ascends 4 stairs with railing RULM Evaluates Motor Performance
in Upper Limbs ABLE TO: Bring Token to Cup placed vertically at shoulder height Bring Weight at Eye Level using two hands 19 Items Graded on scale 0 to 2(Except for 1 activity with a binary score) 0 = unable 1 = able with
modification 2 = able with no difficulty Evaluated upper limb tasks correspond to ability to perform everyday activities Maximum score: 37 Examples of items: Putting a coin into a cup Elevating a cup to mouth Picking up a
coin Bringing hand to shoulder Lifting up weighted objects Opening a zip lock bag Drawing a line on paper O’Hagen et al. 2007; Glanzman et al. 2011; Hammersmith Functional Motor Scale Expanded for SMA (HFMSE) Manual, 2019 Mazzone et
al. 2017; Pierzchlewicz et al. 2021; Revised Upper Limb Module for SMA Manual, 2014
TOPAZ Extension Period: 24-Month Patient Disposition 53 Cohort 1 Ambulatory Non–Ambulatory
Cohort 2 Cohort 3 Total # Non-Ambulatory Patients (2-21) 15 20 35 # Non-Ambulatory Patients (2-12) 9 20 29 Dropped Out (0-12 M) 1 0 0 0 Dropped Out (12-24 M) 1 1 0 1 Not Having Valid HFMSE testing at Month
24 Not applicable 5* 1** 6 Not Having RULM at Month 24 Not applicable 2*** 1**** 3 # of patients who received scoliosis surgery 1 2***** 1 3 * Includes 1 patient who withdrew from study; 1 patient off schedule due to scheduled
surgery; 1 patient who had hip pain; 1 patient with femur fracture; and 1 patient who refused to be in supine position. ** Patient with bilateral lower extremity cast *** Includes 1 patient withdrew from study, and 1 patient off schedule
due to planned surgery. **** Patient was too young for RULM at baseline and RULM was not conducted at following visit. ***** Patients did not have valid HFMSE test at 24 months.
SRK-015 20 mg/kg (N=10) SRK-015 2 mg/kg (N=10) Strong Evidence of Dose Response Observed Over 24
Months Further Supported by Data from Low Dose to High Dose Switch in Non-Ambulatory Patients 54 20 mg/kg throughout Switched from 2 mg/kg to 20 mg/kg Dose-switching period Crawford T et al. TOPAZ EXTENSION: 24-MONTH EFFICACY AND
SAFETY OF APITEGROMAB IN PATIENTS WITH LATER-ONSET SPINAL MUSCULAR ATROPHY (TYPE 2 AND TYPE 3 SMA) Podium Presentation Presented at CureSMA; June 2022 This analysis excludes from the Observed Case Analysis the HFMSE data attained
post-scoliosis surgery during TOPAZ. Error bars represent SEM. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any
other health authority, and its safety and efficacy have not been established. Mean Change from Baseline in HFSME Scores Over Time (Cohort 3) Excludes data after scoliosis surgery Change from Baseline of HFSE Score Time
(Weeks) 10 8 4 2 0 6 0 16 36 52 68 84 104 8 24 40 1/9 patients switched from 2 to 20 mg/kg 9/10 patients switched from 2 to 20 mg/kg All patients switched from 2 to 20 mg/kg Year 2 Year 1 Sample Size at Each
Visit Cohort 3 SRK 2 mg/kg 10 10 10 7 8 9 9 9 10 10 Cohort 3 SRK 20 mg/kg 10 10 10 8 8 10 8 10 10 8
Reported Impact of Scoliosis Surgery on Motor Abilities in SMA 55 Dunaway Young et al.
2020 Scoliosis Surgery HFMSE Total Score 3-month post-surgery assessment 14/17 Lost >3 points on the HFMSE (mean change = - 12.1, SD = 8.9) Functionally meaningful change 13/17 Minimal HFMSE changes within ± 2 points (mean change
= - 0.7) No change or stability 0/17 Improvement > 2 points post-surgery Post-Surgery HFMSE scores Type 2/3 SMApeer-reviewed study
Sizable, Sustained Increases in HFMSE Observed At 24 Months of ApitegromabPooled Non-Ambulatory
Patients 56 Observed Case Analysis is based upon data available for a given timepoint, and this analysis population includes patients treated with the lower dose 2 mg/kg and does not exclude any patients who missed apitegromab doses due to
COVID-19 site access restrictions. Error bars represent standard error of the mean (SEM). Values in parentheticals represent 95% confidence interval. Data on File. Scholar Rock, Inc. Cambridge, MA. Apitegromab is an investigational drug
candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. Mean Change from
Baseline in HFMSE (95% CI) Age 2-21 Years Age 2-12 Years n= 35 29 32 29 n= 29 23 26 24
Strong Evidence of Dose Response Observed Over 24 MonthsFurther Supported by Data from Low Dose to High
Dose Switch in Non-Ambulatory Patients 57 Most patients in Cohort 3 who switched from 2 mg/kg to 20 mg/kg continued to show HFMSE improvement Study Day Patient with scoliosis surgery Apitegromab is an investigational drug candidate
being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. . Change from Baseline of HFSE
Score 20 mg/kg Throughout 2 mg/kg → 20 mg/kg Study day is relative to the date of the first dose of 20 mg/kg Change from Baseline of HFSE Score Benefit maintained through 24-month timepoint Rapid Increase Continued improvement after
switch Gradual increases of HFMSE at 2 mg/kg Switch to 20 mg/kg
Continued Increase in RULM Observed at 24 Months of Apitegromab Pooled Non-Ambulatory Patients
58 Observed Case Analysis is based upon data available for a given timepoint, and this analysis population includes patients treated with the lower dose 2 mg/kg and does not exclude any patients who missed apitegromab doses due to COVID-19
site access restrictions. Data on File. Scholar Rock, Inc. Cambridge, MA. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US
FDA or any other health authority, and its safety and efficacy have not been established. Age 2-21 Years Age 2-12 Years n= 34 28 31 33 n= 28 22 25 28 Mean Change from Baseline in RULM (95% CI)
Cohort 3: Mean RULM Score Change Over TimeRULM Trended Up in Low Dose Arm Patients After Switch to High
Dose 59 Observed Case Analysis is based upon data available for a given timepoint, and this analysis population includes patients treated with the lower dose 2 mg/kg and does not exclude any patients who missed apitegromab doses due to
COVID-19 site access restrictions. Error bars represent standard error of the mean (SEM). Error bars represent SEM. Crawford T et al. TOPAZ EXTENSION: 24-MONTH EFFICACY AND SAFETY OF APITEGROMAB IN PATIENTS WITH LATER-ONSET SPINAL MUSCULAR
ATROPHY (TYPE 2 AND TYPE 3 SMA) Podium Presentation Presented at CureSMA; June 2022. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use
by the US FDA or any other health authority, and its safety and efficacy have not been established. Cohort 3 SRK 2 mg/kg 10 10 10 7 8 9 9 9 10 10 Cohort 3 SRK 20 mg/kg 10 10 10 8 8 10 8 10 10 8 Sample Size at Each
Visit Change from Baseline of RULM Score Time (Weeks) 0 16 36 52 68 84 104 4 3 1 0 -1 2 8 24 40 Mean Change from Baseline in RULM Scores Over Time (Cohort 3) Excludes data after scoliosis surgery SRK-015 20 mg/kg
(N=10) SRK-015 2 mg/kg (N=10) 1 of 9 patients in Cohort 3 switched from 2 mg/kg to 20 mg/kg 9 of 10 patients in Cohort 3 switched from to 2 mg/kg to 20 mg/kg All patients in Cohort 3 switched from 2 mg/kg to 20 mg/kg
Correlation of HFMSE to RULM Increased Over 24 MonthsPooled Non-Ambulatory Patients 60 Crawford T et
al. TOPAZ EXTENSION: 24-MONTH EFFICACY AND SAFETY OF APITEGROMAB IN PATIENTS WITH LATER-ONSET SPINAL MUSCULAR ATROPHY (TYPE 2 AND TYPE 3 SMA) Podium Presentation Presented at CureSMA; June 2022. This analysis is based on the Observed Case
Analysis population. The 12-month graph displays all patients who had a valid measurement at visit 15 (Day 364) and the 24-month graph displays all patients who had a valid measurement at extension visit 14 (Day 728).; Apitegromab is an
investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. -
Basil Darras, MD Associate Neurologist-in-Chief, Boston Children’s Hospital; Professor of Neurology, Harvard Medical School; TOPAZ trial Investigator “The observation that the majority of patients in this analysis experienced gains in both
the HFMSE and RULM over 24 months further supports the therapeutic potential of apitegromab.” r = 0.28 p = 0.1216 n= 31 RULM and HFMSE Change from Baseline at 12 Months Observed Cases RULM Change from Baseline at V15/Day 364 HFMSE
Change from Baseline at V15/Day 364 95% Confidence Limits Regression r = 0.58 p = 0.0011 n=28 RULM and HFMSE Change from Baseline at 24 Months Observed Cases RULM Change from Baseline at EXT14/Day 728 HFMSE Change from Baseline at
EXT14/Day 728 95% Confidence Limits Regression
PEDI-CAT: Measure of activities of daily living PROMIS (Fatigue): Measure of Patient Fatigue ESBBT
(Fatigability): Measure of how fast a patient fatigues 1 2 3 PRO measurement tool4 Muscle endurance measurement tool6 4-point scale (1=unable to 4=easy) assessment of various activities, higher scores reflect improved
abilities1,2 PEDI-CAT has been validated in SMA, but alone cannot identify small changes in function across all types of SMA3 Measures mild subjective feelings of tiredness to debilitating and sustained feelings of exhaustion, with lower
scores reflecting less fatigue4,5 Has been utilized to assess fatigue and fatigability in the Cure SMA database, but has not been fully validated in SMA5 Part of a series of endurance shuttle tests that include: nine-hole peg test, box and
block test, and walk test (ESNHPT, ESBBT, and ESWT)6 Patients are asked to move blocks individually from one box to another in one minute, with higher numbers of blocks suggesting higher muscle endurance6 The endurance shuttle tests have
been validated for use in patients with SMA7 Measures pediatric abilities through 3 functional domains, daily activities, mobility, and social cognitive1 Activities of Daily Living and Fatigue: Assessed by Three Measures 61 ADL,
activities of daily living; ESBBT, endurance shuttle box and block test; ESNHPT, endurance shuttle nine-hole peg test; ESWT, endurance shuttle walk test; PEDI-CAT, pediatric evaluation of disability inventory computer adaptive test; PROMIS,
patient-reported outcomes measurement information system; PRO(s), patient-reported outcome(s); SMA, spinal muscular atrophy. 1. Cre Care. PEDI-CAT. Accessed April 26, 2022. https://www.pedicat.com/. 2. Data on file; Scholar Rock. 2022. 3.
Pasternak A, et al. Muscle Nerve. 2016;54(6):1097-1107. 4. NIH. PROMIS. Accessed April 26, 2022. https://commonfund.nih.gov/promis/index. 5. Belter L, et al. Orphanet Journal of Rare Diseases. 2020;15:217. 6. Cure SMA. Best Practices for
Physical Therapists and Clinical Evaluators in Spinal Muscular Atrophy (SMA). 2021. Available at: https://www.curesma.org/wp-content/uploads/2021/09/Clinical-Evaluators-Best-Practices-13-August-2021.pdf. 7. Bartels B, et al. Orphanet Journal
of Rare Diseases. 2020;15:75. PEDI-CAT, PROMIS, and ESBBT Used to assess: ADL Fatigue Muscle Endurance
Non-Ambulatory Patients Showed Continuous Improvements in ADL and Fatigue Measures Over 24 Months of
Apitegromab Treatment1,2 62 Tertiary Endpoints: Improvements in ADL and Fatigue Apitegromab treatment in non-ambulatory type 2 resulted in patients improving in patient-reported outcomes related to self-sufficiency and
fatigue Non-Ambulatory Type 2 ≥2 years nusinersen initiated before 5 years of age(95% CI) 12 MONTH 24 MONTH ADL, activities of daily living; PEDI-CAT, the Pediatric Evaluation of Disability Inventory computer adaptive test; PROMIS,
Patient-Reported Outcome Measurement Information System. 1. Crawford T et al. P.102. Apitegromab in SMA: An analysis of multiple efficacy endpoints in the TOPAZ extension study Neuromuscular Disorders.2022; 32 (SUPPLEMENT 1): S86-S87; 2.
Crawford T et al. P.102. Apitegromab in SMA: An analysis of multiple efficacy endpoints in the TOPAZ extension study; Poster Presented and Poster Highlights Podium Presentation presented WMS October 2022. Apitegromab is an investigational
drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been
established. Improvement PARENT FATIGUEMean change in PROMIS parent DECREASE: –5.5 (–10.1, –0.9) (n=4) DECREASE: –5.0 (–8.9, –1.1) (n=10) MOBILITY Mean change in PEDI-CAT mobility INCREASE: 0.4 (–2.4, 3.2) (n=11) INCREASE:
2.6 (0.4, 4.7) (n=14) ADL Mean change in PEDI-CAT activities INCREASE: 1.9 (–0.3, 4.1) (n=11) INCREASE: 3.0 (1.8, 4.1) (n=14)
Apitegromab treatment in non-ambulatory types 2 and 3 resulted in patients improving in
patient-reported outcomes related to self-sufficiency and fatigue Non-Ambulatory Patients Showed Stabilization or Improvements in ADL and Fatigue Measures over 24 Months of Apitegromab Treatment1,2 63 Tertiary Endpoints: Improvements in
ADL and Fatigue Improvement Non-AmbulatoryType 2/3 5-21 years nusinersen initiated after 5 years of age(95% CI) 12 MONTH 24 MONTH ADULT FATIGUEMean change in PROMIS adult DECREASE: –3.5 (–35.3, 28.3) (n=2) DECREASE: –3.5
(–9.9, 2.9) (n=2) PARENT FATIGUEMean change in PROMIS parent DECREASE: -0.6 (–5.9, 4.7) (n=10) DECREASE: -1.3 (-6.7, 4.0) (n=9) ADL Mean change in PEDI-CAT activities INCREASE: 0.9 (–1.4, 3.2) (n=12) INCREASE: 0.7
(-1.8, 3.2) (n=8) ADL, activities of daily living; PEDI-CAT, the Pediatric Evaluation of Disability Inventory computer adaptive test; PROMIS, Patient-Reported Outcome Measurement Information System. 1. Crawford T et al.
P.102. Apitegromab in SMA: An analysis of multiple efficacy endpoints in the TOPAZ extension study Neuromuscular Disorders.2022; 32 (SUPPLEMENT 1): S86-S87; 2. Crawford T et al. P.102. Apitegromab in SMA: An analysis of multiple efficacy
endpoints in the TOPAZ extension study; Poster Presented and Poster Highlights Podium Presentation presented WMS October 2022. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy.
Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established.
Non-Ambulatory Patients Continued to Improve in Fatigability and Endurance Measures, Which May be
Complementary to Upper Limb Function Improvements over 24 Months1 64 The ESBBT is the first validated and sensitive fatigability test for proximal arm function in SMA and may be complementary to outcome measures that focus on arm motor
function such as the RULM, by adding the dimension of endurance2 Trends of improvements with ESBBT correlate with RULM over 24 months ESBBT, endurance shuttle box and block test; SMA, spinal muscular atrophy; SMN, survival motor neuron;
QoL, quality of life. 1.Darras BT, et al. Apitegromab in SMA (TOPAZ trial): Efficacy, Safety, and PK/PD Assessments From 24- Month Data; Podium Presentation presented at SMAEU; October 2022 . 2. Mazzone ES, et al. RULM for SMA: development of
a new module. Muscle Nerve. 2017;55(6):869–74. Baseline is defined as the last measurement prior to the first dose of study drug. Subject visits after an intercurrent event of 3 consecutive missed doses during the Extension A period, or after
taking nusinersen for SMN up-regulator therapy if in Cohort1, are excluded from the Efficacy Eligible Set. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been
approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. Apitegromab treatment in non-ambulatory types 2 and 3 (cohort 2) resulted in patients improving in QoL assessments
related to self-sufficiency and endurance Tertiary Endpoints: Improvements in Fatigability Improvement Non-AmbulatoryType 2/3 5-21 years nusinersen initiated before5 years of age(95% CI) 12 MONTH 24 MONTH ENDURANCE Mean change in
ESBBT activities INCREASE: 281 (–22.7, 584.2)1 (n=8) INCREASE: 147.9 (0.6, 295.2)1 (n=8)
Therapeutic Potential of Apitegromab Observed in the Ambulatory Type 3 SMA Cohort at 24
Months1-4 65 Ambulatory Patients (Revised Hammersmith Scale; RHS) 24-Month Analysis COHORT 1 20 mg/kg pooled1 (n=21) 20 mg/kg Monotherapy4 (n=11) 20 mg/kg + nusinersen2,3 (n=10) Mean change from baseline, (95% CI) -1.8 (-4.7,
1.1) -2.8 (-8.4, 2.8) -0.7 (-3.1, 1.7) Patients achieving ≥ 1-pt increase, n (%) 9/21 (42.9%) 5/11 (45.5%) 4/10 (40%) Patients achieving ≥ 3-pt increase, n (%) 5/21 (23.8%) 3/11 (27.3%) 2/10 (20%) Observed Case Analysis includes
all patients who had a valid measurement at E14 (Day 728). Inclusive of data from 3 patients in apitegromab monotherapy who lost ability to ambulate. 1. Crawford T et al. TOPAZ EXTENSION: 24-MONTH EFFICACY AND SAFETY OF APITEGROMAB IN
PATIENTS WITH LATER-ONSET SPINAL MUSCULAR ATROPHY (TYPE 2 AND TYPE 3 SMA) Podium Presentation Presented at CureSMA; June 2022. 2. Crawford T et al. P.102. Apitegromab in SMA: An analysis of multiple efficacy endpoints in the TOPAZ extension
study Neuromuscular Disorders.2022; 32 (SUPPLEMENT 1): S86-S87. 3. Crawford T et al. P.102. Apitegromab in SMA: An analysis of multiple efficacy endpoints in the TOPAZ extension study; Poster Presented and Poster Highlights Podium
Presentation presented WMS October 2022. 4. Data on File, Scholar Rock Inc. 2022. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by
the US FDA or any other health authority, and its safety and efficacy have not been established.
Fourteen patients experienced a serious TEAE, all assessed by the respective trial investigator as
unrelated to apitegromab: One patient treated with 2 mg/kg dose (Cohort 3) was hospitalized due to adenoidal hypertrophy and tonsillar hypertrophy to perform scheduled adenotonsillectomy (Grade 2). Events resolved without sequelae. Two
patients treated with 20 mg/kg dose (both Cohort 1) presented with gait inability considered a significant disability (both Grade 3). Events remain ongoing. One patient treated with 20 mg/kg dose (Cohort 1) was hospitalized with post lumbar
puncture syndrome (Grade 2). Event resolved without sequelae. One patient treated with 20 mg/kg dose (Cohort 1) was hospitalized due to viral upper respiratory tract infection (Grade 2). Event resolved without sequelae. Five patients
treated with 20 mg/kg dose (one from Cohort 1, three from Cohort 2, and one from Cohort 3) were hospitalized for spinal fusion surgery/ scoliosis/ scoliosis surgery (all Grade 3). All events resolved without sequelae. One patient treated
with 20 mg/kg dose (Cohort 1) was hospitalized due to bilateral developmental hip dysplasia and left hip dislocation (both Grade 3). Events resolved without sequelae. One patient treated with 2 mg/kg dose (Cohort 3) was hospitalized due to
hip dislocation (Grade 3). Event resolved with sequelae (anxiety and post-operative pain). One patient treated with 20 mg/kg dose (Cohort 3) was hospitalized due to respiratory syncytial virus infection (Grade 2). Events resolved without
sequelae. One patient treated with 2 mg/kg dose (Cohort 3) was hospitalized due to vomiting and pneumonia (Grade 3). Events resolved without sequelae. Overall Safety and Tolerability Profile Over 24 Months of Treatment: Serious
TEAEs 66 Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy
have not been established.
Four patients presented with non-serious Grade 3 events, all assessed by the respective trial
investigator as unrelated to apitegromab: One patient treated with 20 mg/kg dose (Cohort 1) presented with post lumbar puncture syndrome. Event resolved without sequelae. One patient treated with 20 mg/kg dose (Cohort 2) presented with
worsening of scoliosis. Event resolved (with surgery, reported as serious, above) without sequelae. One patient treated with 20 mg/kg dose (Cohort 2) presented with osteopenia. Events remains ongoing. One patient treated with 2 mg/kg
(Cohort 3) presented with two instances of hypoglycemia and one instance of metabolic acidosis. All events resolved without sequelae. One patient (Cohort 1) discontinued from the trial due to Grade 2 muscle fatigue that started prior to
initiation of dosing with study drug; assessed by the trial investigator as unrelated to apitegromab. Overall Safety and Tolerability Profile Over 24 Months of Treatment: Non-Serious Grade 3 Events 67 Apitegromab is an investigational drug
candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established.
SRK-181: Transformative Potential as the Backbone For Next Era of Cancer Therapy 68 CPI
Combination (solid tumors) Other Combinations (solid tumors) & Hematologic Other immunotherapies Chemotherapy XRT Otherapplications Myelofibrosis Liquid tumors 1st line immunotherapy First in class monoclonal antibody
targeting latent and context-independent binding to TGFB-1 CURRENT FOCUS Primary and AcquiredCPI resistance Investigation in primary (pre-existing) CPI resistance offers path to early clinical POC for SRK-181
Regulatory T cell Tumor Associated Macrophage Cancer Associated Fibroblasts (Stromal cells) Tumor
cells SRK-181 Targets latent TGFβ-1 Inhibits activation of latent TGFβ-1 across ALL compartments Turns it off at the source Context-Independent: SRK-181 Inhibits Latent TGFβ-1 Across All Compartments of the Tumor
Microenvironment 69 Latent TGFβ-1 Key driver of tumor resistance to CPIs Present in multiple compartments of the tumor microenvironment TGFβ Latent growth factor TGFβ source presentation LRRC33 GARP LTBPs LTBPs LRRC33:
leucine-rich-repeat-containing protein family member 33 | LTBPs: latent transforming growth factor β binding proteins | GARP: glycoprotein A repetitions predominant
SRK-181 Therapeutic Hypothesis: Potential Advantages of Latent TGFβ-1
Inhibitor 70 SRK-181* Bifunctional TGFβ/CPI ALK5 Inhibitor Nonselective TGFβ antibody Selectivity for TGFβ-1: potential for wider therapeutic window and improved safety Ability to combine with any anti-PD-(L)1 Ability to
optimize dosing of each component of combination therapy Activity spatially distinct from anti-PD-(L)1 in tissue Inhibits all sources of TGFβ-1 contributing to CPI resistance (Context independent) Target latent form (Blocks TGFβ-1
activation) *SRK-181 is an investigational product candidate currently being evaluated in DRAGON phase 1 clinical trial. The efficacy and safety of SRK-181 have not been established. X X X X X X X X X X
TGFβ-1 Blockade with SRK-181-mIgG1 Rendered Preclinical Tumor Models Susceptible to Anti-PD1
Therapy 71 Days after treatment initiation Tumor volume (mm3) Anti-PD1/ SRK-181-mIgG1(3 mg/kg QW) 4/14 Anti-PD1/ SRK-181-mIgG1(10 mg/kg QW) 8/14 Preclinical data published in Science Translational Medicine. Martin CJ, et al. Sci
Transl Med. 2020 Mar 25;12(536):eaay8456. https://scholarrock.com/platform/publications/. *SRK-181-mIgG1 is the murine version of SRK-181; responder defined as tumor size <25% endpoint volume at study end. Bladder Cancer Breast Cancer
(TGFβ-1/3 co-expressing) 0/13 Anti-PD1 (10 mg/kg BIW) 0/10 SRK-181-mIgG1* (10 mg/kg QW) Anti-PD1/ SRK-181-mIgG1(10 mg/kg QW) 5/10 Responders 0/9 Control 0/9 Anti-PD1 (10 mg/kg BIW) SRK-181-mIgG1* (10 mg/kg QW) Responders
0/12 MBT-2 and EMT6 Models
SRK-181-mIgG1 Combination Treatment Led to Melanoma Tumor Regression and Survival Benefit
72 *P<0.01. †P<0.05 Log-rank (Mantel-Cox test) vs anti-PD1. * * † Days after treatment initiation Tumor Regression Survival Benefit Anti-PD1 + SRK-181-mlgG1 (30 mg/Kg/wk) Anti-PD1 + SRK-181-mlgG1 (3 mg/Kg/wk) Anti-PD1 +
SRK-181-mlgG1 (10 mg/Kg/wk) Anti-PD1 (10 mg/Kg/2xwk) SRK-181-mlgG1 (30 mg/Kg/wk) Control Melanoma (Cloudman S91) model Preclinical data published in Science Translational Medicine. Martin CJ, et al. Sci Transl Med. 2020 Mar
25;12(536):eaay8456. https://scholarrock.com/platform/publications. Days after treatment initiation Monotherapy 3/12 Anti-PD1 (10 mg/kg BIW) Tumor volume (mm3) SRK-181-mIgG1 (30 mg/kg QW) Responders 0/12 Combination Therapy Led to
tumor regression and survival benefit Anti-PD1/SRK-181-mIgG1 (30 mg/kg QW) 8/11 Days after treatment initiation Anti-PD1/SRK-181-mIgG1 (10 mg/kg QW) Tumor volume (mm3) 4/9
Selectivity of SRK-181 Offers Potential to Overcome Toxicity and Dose-limiting Challenges of
Non-selective TGFβ Pathway Approaches 73 Preclinical data published in Science Translational Medicine. Martin CJ, et al. Sci Transl Med 2020 Mar 25;12(536): eaay8456. *Source: Anderton MJ, et al. Induction of heart valve lesions by
small-molecule ALK5 inhibitors. Toxicol Pathol. 2011;39: 916-924.; and Stauber AJ, et al. Nonclinical safety evaluation of a transforming growth factor β Receptor I kinase inhibitor in Fischer 344 rats and beagle dogs. J Clin Pract. 2014:
4:3. Microscopic Observations in Heart Valvulopathy Atrium—Mixed cell infiltrate Myocardium—Degeneration/necrosis Myocardium—Hemorrhage Myocardium—Mixed cell infiltrate, base Coronary artery—Necrosis with
inflammation Cardiomyocyte—Necrosis/inflammatory cell infiltrate CONTROL Vehicle iv, qwk x 4 LY2109761 300 mg/kg po, qd x 8 PanTGFβAb 30 mg/kg Iv, 1 dose 10 mg/kg iv, qwk x 4 30 mg/kg iv, qwk x 4 100 mg/kg iv, qwk x
4 SRK-181 Selective TGFβ-1 Toxicity: Minimal Non-selective TGFβ Toxicity: Minimal, slight and moderate Unremarkable Minimal Slight Moderate Toxicology: Repeat Dose Pilot Toxicology Study Adult female Sprague Dawley rats Cardiac
findings were exhibited in animals dosed with pan-TGFβ antibody or LY2109761 (inhibitor of ALK5, common TGFβ receptor kinase) as expected based on published data† NO CARDIOTOXICITIES (valvulopathy)were noted with SRK-181 NOAEL for
SRK-181: 100 mg/kg QW(highest dose evaluated) 4-week GLP toxicology studies RATS NOAEL for SRK-181: 200 mg/kg QW (highest dose evaluated) NON-HUMAN PRIMATES NOAEL for SRK-181: 300 mg/kg(highest dose evaluated) Not test article related
Bladder urothelial carcinoma Had & neck squamous carcinoma Kidney clear cell carcinoma Kidney
papillary cell carcinoma Liver hepatocellular carcinoma Lung adenocarcinoma Skin cutaneous melanoma Stomach adenocarcinoma Acute myeloid leukemia Adrenocortical cancer Brain lower grade glioma Breast invasive carcinoma Cervical &
endocervical cancer Cholangiocarcinoma Colon adenocarcinoma Diffuse large B-cell carcinoma Esophageal carcinoma Glioblastoma multiforme Kidney chromophobe Lung squamous cell carcinoma Mesothelioma Ovarian serous
cystadenocarcinoma Pancreatic adenocarcinoma Pheochromocytoma & paraganglioma Prostate adenocarcinoma Emerging Evidence Implicates TGFβ-1 as Driving Primary Resistance to Checkpoint Inhibitors 74 Human Tumor Analyses Reveal TGFβ-1
as Most Likely Driver of TGFβ Signaling Pathway in Cancers †Priti H, et al. Top 10 challenges in cancer immunotherapy. Immunity. 2020 Jan 14:52(1):17-35. https://doi.org/10.1016/j.immuni.2019.12.011. *Source: National Cancer Institute -
Cancer Genome Atlas Program. NSCLC TMB Low High Substantial % of Solid Tumors Exhibit Immune Exclusion Immune Excluded Immune Phenotype Inflamed Immune Desert Tumor Type Melanoma RCC UBC TNBC Gastric CRC
MSS Pancreatic SCLC HR+BC Prostate Immune Excluded Inflamed Immune Desert Cancer Genome Atlas RNAseq Analysis of >10,000 Samples Spanning 33 Tumor Types* TGFB1 TGFB2 TGFB3 Scale % of patient samples (+) for TGF-β
isoform 100 80 60 40 20 0
Biomarker Strategies Employed in DRAGON Trial 75 Multiple tissue-based and circulating biomarker
analyses to be evaluated in DRAGON study Higher resolution histochemical characterization of tumor immune contexture (e.g. CD8+) Classification of inflamed, excluded or immune desert tumors and tumor nests Ability of SRK-181 to overcome
tumor immune exclusion Analysis of immune response markers (e.g. PD-L1) Changes to intra-tumoral and/or circulating immune cell contexture (MDSC) Show evidence of the SRK-181 target engagement e.g. circulating TGFβ-1 levels TGFβ pathway
modulation: e.g. Histochemical analysis of pSMAD e.g. RNA-based TGFβ gene signatures and pathway analyses Paired biopsies from the head and neck cohort allow for a potential to accelerate the development path Immunophenotyping Assessment
of immune landscape TGFβ-1 pathway evaluation Assessment of signaling pathway
Clear Evidence of Target EngagementPharmacodynamic Biomarker Results for Part A: Circulatory
TGFβ-1 76 Yap T et al. SRK-181, a latent TGFβ1 inhibitor: safety, efficacy, and biomarker results from the dose escalation portion of a phase I trial (DRAGON trial) in patients with advanced solid tumors (Poster 780); Presented at SITC;
Nov. 10-11, 2022. Circulatory TGFβ-1 and PF4 levels were quantitated by using validated ELISA kits from R&D System.12 Because platelet activation during sample processing can lead to elevated TGFβ-1 levels, samples with elevated PF4, a
platelet activation biomarker, were excluded from the analysis based on a preliminary cutoff value. Pre-infusion. SRK-181 is an investigational drug candidate that is being evaluated for the treatment of cancer. SRK-181 has not been
approved by the US FDA or any other health authority, and its safety and efficacy have not been established. Binding to latent TGFβ-1 delays maturity state allowing TGFβ-1 to accumulate in system Combination treatment with pembrolizumab did
not appear to impact circulatory TGFβ-1 levels C1D2 C1D8 C1D15 C2D1* C3D1* C3D2 C3D8 C3D15 C4D1* C5D1* Baseline Circulatory TGFβ-1 (Fold-Change from Baseline) 2 3 4 6 5 Median Circulatory TGFβ-1 Increased Post-treatment
with SRK-181 (Q3W, All Patients)
Preclinical Data Provide Scientific Rationale to Evaluate Peripheral Samples for Evidence of SRK-181
Activity 77 MBT-2 bladder tumor model IgG, anti-PD-1 and SRK-181-mIgG1 dosed d1, d7 Analysis on day 10 Circulating MDSC Levels Correlate with Tumor Volume Both tumoral and circulatory MDSC are being evaluated in the DRAGON
study Immunophenotyping Assessment of immune landscape Measurement of MDSCs in circulation may provide indirect evidence of drug action on the tumor Myeloid-derived suppressor cells (MDSCs) have immune suppressive functions SRK-181 plus
anti-PD1 combination drive MDSC levels down significantly in the tumor microenvironment Reductions in circulating MDSC levels correlate with reduced tumor volume following SRK-181 and anti-PD1 treatment in MBT-2 tumor
model Anti-PD-1+ SRK-181 1 mpk 3 mpk 10 mpk IgG Ctrl SRK-181 Anti-PD-1
| Exhibit 99.2 |
Scholar Rock Provides Corporate Update and Highlights Priorities for 2023
- Pivotal Phase 3 SAPPHIRE trial enrollment completion expected in 2023
- Phase 1 DRAGON trial of SRK-181 continues to advance with presented data that showed early indications of efficacy; ongoing clinical data updates planned in 2023
- Anticipated cash runway into 2025
CAMBRIDGE, Mass.--(BUSINESS WIRE)--January 9, 2023--Scholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today provided recent corporate updates and highlighted upcoming priorities for its pipeline programs in 2023.
“In 2022, Scholar Rock made significant progress in advancing its clinical programs, notably with the 24-month extension data from the Phase 2 TOPAZ trial, which reinforces our conviction behind apitegromab and the Phase 3 SAPPHIRE trial, and with early data readouts from the Phase 1 DRAGON trial. The company also strengthened its financials in June 2022 with a substantial equity raise. We are excited by the potential coming from our highly differentiated platform targeting growth factors like TGFβ, as we advance our spinal muscular atrophy and oncology programs to address critical unmet needs for patients,” said Dr. Jay Backstrom, M.D., M.P.H., President & CEO of Scholar Rock. “In 2023, we see continued momentum for our growing pipeline, including completing enrollment of our pivotal SAPPHIRE trial, disclosing 36-month extension data from the Phase 2 TOPAZ trial and clinical and biomarker updates from the SRK-181 Phase 1 DRAGON trial, and advancing two preclinical assets towards IND-enabling studies in fibrosis and iron-restricted anemia.”
2023 Priorities:
Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-targeted therapy for the treatment of spinal muscular atrophy (SMA).
- Complete enrollment of Phase 3 SAPPHIRE clinical trial in 2023. SAPPHIRE is a randomized, double-blind, placebo-controlled clinical trial evaluating apitegromab for patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam. The last patient is expected to be enrolled in SAPPHIRE in 2023, with the top-line data readout expected in 2024. If successful, the company expects to initiate a commercial product launch in 2025.
- Progress TOPAZ long-term extension to 36-month data readout. The company expects to report 36-month extension data in the first half of 2023. As of December 31, 2022, approximately 90 percent of patients (51/57) remained enrolled in the trial’s long-term extension period.
SRK-181 is an investigational selective inhibitor of latent TGFβ-1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer.
- Advance Progress in DRAGON Phase 1 trial. Scholar Rock is expecting to provide biomarker and clinical updates from the DRAGON Phase 1 trial in 2023.
Preclinical fibrosis and iron-restricted anemia assets
- Advance the fibrosis program towards IND-enabling studies. Scholar Rock plans to advance a highly potent, anti-latent TGFβ-1 antibody that selectively inhibits TGFβ1 activation within the extracellular matrix by targeting latent TGFβ-1 associated with latent TGFβ-binding proteins (LTBPs), thus enabling specific inhibition of TGFβ-1 in fibrotic tissue.
- Advance the iron-restricted anemia program towards IND-enabling studies. Scholar Rock plans to advance a highly selective, RGMc/HJV antibody that targets the signaling of BMP6, a key regulator of iron availability in the body. Utilizing Scholar Rock’s unique structural biology insights into BMP6 and its co-receptors and leveraging its novel antibody discovery and optimization platform, the company generated an anti-RGMc antibody that can modulate iron release and has the potential to address anemia.
2022 Highlights:
- TOPAZ 24-month extension trial data showed sizeable and sustained gains in Hammersmith Functional Motor Scale Expanded (HFMSE), increased Revised Upper Limb Module (RULM), and positive trends in quality-of-life data for nonambulatory patients with Types 2 and 3 SMA receiving an SMN-targeted therapy.
- Completed equity financing of $205 million in June. As of December 31, 2022, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $315 million, which is expected to fund the company’s operations into 2025.
- Phase 1 DRAGON trial data presented at the Society for Immunotherapy of Cancer's Annual Meeting in November showed that SRK-181 continued to be generally well tolerated with early indications of efficacy (as of the data cut-off date of August 29, 2022).
- Presented new data on LTBP showing reduction of TGFβ-1 signaling and fibrosis in relevant in vivo preclinical models. The findings were presented at the 2022 FASEB Science Research Conference in July and the American College of Toxicology Annual Meeting in November.
- Announced Jay Backstrom, M.D., M.P.H. was appointed President & CEO in October, bringing an exceptional range of research and development, regulatory, and leadership experience spanning several decades in the biopharmaceutical industry.
- Announced Jing L. Marantz, M.D., Ph.D., M.B.A., was appointed Chief Medical Officer in November. Dr. Marantz is an accomplished biopharmaceutical executive with over 20 years of industry experience spanning multiple specialties, including neurology, hematology/oncology, and rare diseases.
“With our strong balance sheet and two well established clinical programs, both of which we expect to generate data in 2023, Scholar Rock is uniquely positioned to bring differentiated therapies to patients suffering from serious diseases in which protein growth factors play a fundamental role,” said Ted Myles, Chief Operating Officer and Chief Financial Officer.
About Scholar Rock
Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, and fibrosis. Scholar Rock’s approach to targeting the molecular mechanisms of growth factor activation enabled it to develop a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level. By developing product candidates that act in the disease microenvironment, the Company intends to avoid the historical challenges associated with inhibiting growth factors for therapeutic effect. Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path. For more information, please visit www.ScholarRock.com or follow Scholar Rock on Twitter (@ScholarRock) and LinkedIn (https://www.linkedin.com/company/scholar-rock/).
Availability of Other Information About Scholar Rock
Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on Twitter and LinkedIn. The information that we post on our website or on Twitter or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
Scholar Rock® is a registered trademark of Scholar Rock, Inc.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab, SRK-181, and other product candidates and indication selection and development timing, its cash runway, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data, including the results from the Phase 2 clinical trial of apitegromab, or Part A of the Phase 1 clinical trial of SRK-181, are not predictive of, may be inconsistent with, or more favorable than, data generated from future clinical trials of the same product candidate, including, without limitation, the Phase 3 clinical trial of apitegromab in SMA or Part B of the Phase 1 clinical trial of SRK-181, Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline, the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials, information provided or decisions made by regulatory authorities, competition from third parties that are developing products for similar uses, Scholar Rock’s ability to obtain, maintain and protect its intellectual property, Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials, Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, and the impacts of current macroeconomic and geopolitical events, including changing conditions from the COVID-19 pandemic, hostilities in Ukraine, increasing rates of inflation and rising interest rates, on business operations and expectations, as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.
Contacts
Scholar Rock:
Investors
Rushmie Nofsinger
Scholar Rock
rnofsinger@scholarrock.com
ir@scholarrock.com
857-259-5573
Media
Ariane Lovell
Finn Partners
ariane.lovell@finnpartners.com
media@scholarrock.com
917-565-2204