Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial
- Topline data from Phase 3 trial expected in Q4 2024
- Company to present encore 36-month efficacy, safety, and patient-reported outcomes data from the Phase 2 TOPAZ trial extension at the 2023 World Muscle Society (WMS) Annual Congress
- Apitegromab is the only muscle-targeted therapy candidate with clinical data showing proof of concept in spinal muscular atrophy
“Completing SAPPHIRE enrollment is a critical step on the path to bringing apitegromab, a potentially transformative therapy, to individuals living with SMA. SAPPHIRE was designed to build on the positive TOPAZ results, and we look forward to reporting the topline results from this registrational study in Q4 next year,” said
Details of the poster presentations are as follows:
Title: Effect of apitegromab on PEDI-CAT and PROMIS-fatigue questionnaire at 36 months in patients with spinal muscular atrophy
Presentation type: Poster presentation
Date and time:
Title: Effect of apitegromab on motor function at 36 months in patients with nonambulatory spinal muscular atrophy aged 2 – 12 years old
Presentation type: Poster presentation
Date and time:
For conference information, visit https://www.wms2023.com/.
The presentations will be made available in the Publications & Posters section of Scholar Rock’s website following the presentation.
About the Phase 2 TOPAZ Trial
The TOPAZ trial is an ongoing proof-of-concept, open-label Phase 2 trial evaluating the safety and efficacy of apitegromab in patients with Types 2 and 3 SMA. In the main treatment period, patients were dosed intravenously every four weeks as monotherapy or with nusinersen, an approved SMN-targeted therapy. The trial enrolled 58 patients in the
About the Phase 3 SAPPHIRE Trial
SAPPHIRE is an ongoing randomized, double-blind, placebo-controlled, phase 3 clinical trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are receiving SMN-targeted therapy (either nusinersen or risdiplam). SAPPHIRE targeted enrolling approximately 156 patients aged 2-12 years old in the main efficacy population. These patients were randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An exploratory population that targeted enrolling up to 48 patients aged 13-21 years old will also separately be evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo. For more information about SAPPHIRE, visit www.clinicaltrials.gov.
Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate to demonstrate clinical proof of concept in spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans.
Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease that afflicts an estimated 30,000 to 35,000 people in
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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, any development plans, strategy and progress for apitegromab, the timing of its clinical trials, anticipated clinical data, and therapeutic benefits for apitegromab, and other product candidates and indication selection and development timing, the ability of any product candidate to perf,orm in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of apitegromab and its other product candidates and its 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, are not predictive of, may be inconsistent with, or more favorable than, data generated from future clinical trials of the same product candidates, including, without limitation, the Phase 3 clinical trial of apitegromab in SMA, 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 SAPPHIRE Phase 3 clinical trial, information provided or decisions made by regulatory authorities, Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials, , as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Annual Report on Form 10-Q for the quarter ended