Scholar Rock Provides Corporate Update and Highlights Priorities for 2024
- Advancing industry-leading antimyostatin pipeline, comprised of multiple, novel assets with unparalleled selectivity, to treat spinal muscular atrophy (SMA) and cardiometabolic disorders
- Completed enrollment for apitegromab pivotal Phase 3 SAPPHIRE trial in patients with SMA; topline data anticipated in 4Q 2024
- Apitegromab Phase 2 proof-of-concept trial in obesity expected to commence in mid-2024
- Presenting new preclinical data on SRK-439, a novel investigational myostatin inhibitor for the treatment of obesity, at Keystone Symposia in February
- Presenting at the 42nd Annual
“We were pleased with our progress in 2023 as we advanced our industry leading antimyostatin pipeline with apitegromab and SRK-439 and have great momentum heading into 2024. With enrollment completed for our Phase 3 SAPPHIRE trial for patients with spinal muscular atrophy, we expect topline data in Q4 this year,” said
SPINAL MUSCULAR ATROPHY
Apitegromab is an investigational, fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle and is being developed as a potential first muscle-targeted therapy for the treatment of SMA. Apitegromab is the only muscle-targeted therapy to show clinical proof-of-concept in SMA.
- Planning to announce Phase 3 SAPPHIRE clinical trial topline data in 4Q 2024. SAPPHIRE is a randomized, double-blind, placebo-controlled clinical trial evaluating apitegromab in patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam. If the trial is successful and apitegromab is approved, the Company expects to initiate a commercial product launch in 2025.
- Continue to progress the ONYX long-term extension study for patients from both the TOPAZ and SAPPHIRE studies.
SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of obesity.
- Initiating a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in mid-2024. As part of the Company’s strategy to advance the development of SRK-439, it plans to initiate a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 RA, subject to IND clearance. Data from the clinical trial are expected in mid-2025 and will be used to guide clinical development of SRK-439. The Company plans to file an IND for SRK-439 for the treatment of obesity in 2025.
Presenting preclinical SRK-439 data in a poster presentation at Keystone Symposia at the Obesity: Causes and Consequences meeting on
February 5, 2024, in Vancouver, BC, Canada.
- Completed enrollment of apitegromab pivotal Phase 3 SAPPHIRE trial.
Presented TOPAZ 36-month extension trial data at the
Cure SMA Research & Clinical Care Meetingin June, and at the 28th Annual Congressof the World Muscle Societyin October, which showed long-term substantial and sustained improvements in motor function and patient-reported outcome measures in patients with nonambulatory Types 2 and 3 SMA receiving survival motor neuron (SMN) therapy.
- Initiated the ONYX trial, a long-term extension study for patients from both the TOPAZ and SAPPHIRE studies, which remains ongoing.
- Announced plans to expand into cardiometabolic disorders with SRK-439, starting with a Phase 2 proof-of-concept trial evaluating apitegromab in obesity to inform development of SRK-439.
- Presented SRK-181 Phase 1 DRAGON trial clinical and biomarker data at the SITC 38th Annual Meeting, which showed favorable tolerability and promising anti-tumor activity in heavily pretreated patients with clear cell renal cell carcinoma (ccRCC) resistant to anti-PD-1. The Company believes these data support proof-of-concept and completed enrollment of the DRAGON trial in December. The Company will provide additional clinical data updates as they become available in 2024.
Completed an equity financing of
$98 millionin October. As of December 31, 2023, Scholar Rockreported cash, cash equivalents, and marketable securities of approximately $280 million, which is projected to fund the Company’s operations into the second half of 2025.
“We are excited to enter 2024 with several important near-term milestones ahead of us. As the leader in selective myostatin inhibition, we believe we are well positioned to deliver significant value to patients living with spinal muscular atrophy and the millions of people suffering from the wide array of health challenges stemming from cardiometabolic and obesity disorders. We are highly encouraged by our potential to advance the standard of care where muscle-targeted therapies can play a role in addressing unmet patient needs,” said
J.P. Morgan Healthcare Conference Presentation and Webcast
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,
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, results and timing of its clinical trials for apitegromab and SRK-181 and its preclinical programs, including SRK-439, regulatory feedback including with respect to the IND submitted in connection with the planned Phase 2 trial of SRK-439 in combination with GLP-1 RAs in obesity, and indication selection and development timing, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, its expected cash and cash equivalents as of