Scholar Rock announces the publication of data from a phase 1 clinical trial evaluating apitegromab in healthy volunteers in the Journal Advances in Therapy


CAMBRIDGE, Mass .– (BUSINESS WIRE) –Scholar rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious conditions in which protein growth factors play a fundamental role, today announced that the results of its Phase 1 safety trial and the pharmacological profile of apitemograb in healthy volunteers were published in the peer-reviewed journal Advances in therapy. Apitegromab is a selective inhibitor of myostatin activation in development for the treatment of spinal muscular atrophy (SMA).

“The data from Phase 1 provided an important basis to support the advancement of the development of apitegromab and we are delighted to share these detailed results through publication in this peer-reviewed journal,” said Yung Chyung, MD, medical director of Scholar Rock. “We recently announced the first positive results from the Phase 2 TOPAZ trial, demonstrating the transformative potential of apitegromab to improve persistent motor function disorders in patients with SMA type 2 and type 3 and we look forward to advancing the development of apitegromab in ADS. ”

The objectives of the phase 1, double-blind, placebo-controlled study were to assess the safety, immunogenicity, pharmacokinetics (PK) and pharmacodynamics (PD measured by serum latent myostatin levels) of ‘apitegromab to support future clinical studies. A total of 66 healthy volunteers were recruited and randomized to receive either intravenous (IV) apitegromab or placebo at doses between 1 mg / kg and 30 mg / kg. Main findings shared in Advances in therapy include:

  • Single and multiple ascending doses of apitegromab were safe and well tolerated at IV doses up to 30 mg / kg. No anti-drug antibodies were detected in subjects treated with apitegromab.

  • Apitegromab demonstrated a dose-proportional linear pharmacokinetic profile and a half-life of 24 to 31 days for the doses tested.

  • Apitegromab has demonstrated dose-dependent increases in latent serum myostatin concentrations as well as sustained target saturation.

    • Mean serum latent myostatin levels increased and peaked between days 14 and 28 after single doses up to 10 mg / kg and between days 42 and 56 for doses of 20 mg / kg and 30 mg / kg. This contrasts with the mean latent serum myostatin levels remaining at or near baseline after placebo.

    • For subjects who received multiple doses of apitegromab (every 2 weeks for a total of three doses), the mean latent serum myostatin concentrations remained elevated until the third dose and began to decrease on day 56 for the doses. of 10 mg / kg and 20 mg / kg. and day 84 for the dose of 30 mg / kg.

Data from this Phase 1 study, as well as preclinical data, supported advancing the development of apitegromab into the Phase 2 TOPAZ trial, which assessed the potential of apitegromab to improve motor function in patients. patients with SMA type 2 and type 3. Recently Scholar Rock ad Positive 12-month baseline data from TOPAZ trial demonstrating transformative therapeutic potential of apitegromab in patients with SMA type 2 and type 3. A phase 3 registration trial is expected to start by completion from 2021.

About Apitegromab

Apitegromab is a selective inhibitor of myostatin activation and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ growth factor superfamily, is primarily expressed by skeletal muscle cells, and the absence of its gene is associated with increased muscle mass and strength in several animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote clinically meaningful improvement in motor function in patients with ADS. The United States Food and Drug Administration (FDA) granted the Orphan Drug Designation (ODD) and the Rare Pediatric Disease (RPD) designation, and the European Medicines Agency (EMA) granted the priority drug designation (PRIME) and the orphan drug product designation to apitegromab for the treatment of SMA. The efficacy and safety of apitegromab have not been established and apitegromab has not been approved for use by the FDA or any other regulatory body.

About SMA

Spinal muscular atrophy (SMA) is a rare and often fatal genetic disorder that typically occurs in young children. It is estimated that 30,000 to 35,000 patients have ADS in the United States and Europe. It is characterized by loss of motor neurons, atrophy of voluntary muscles in the limbs and trunk, and progressive muscle weakness. The underlying pathology of SMA is caused by insufficient production of the motor neuron survival (SMN) protein, essential for motor neuron survival, and is encoded by two genes, SMN1 and SMN2. Although there has been progress in the development of therapeutics that treat the underlying genetic defect of SMA, via MNS-dependent pathways, there is still a high unmet need for therapeutics that directly treat SMA. muscle atrophy.

About Scholar Rock

Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative drugs for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of new product candidates with the potential to transform the lives of patients with a wide range of serious illnesses, including neuromuscular disorders, cancer, fibrosis and anemia. Scholar Rock’s newly elucidated understanding of the molecular mechanisms of growth factor activation has enabled him 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 that the 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/).

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 the potential of SRK-181, Scholar’s future expectations, plans and prospects. Rock, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of clinical trials, potential of its proprietary platform, and intellectual property protection. The use of words such as “may”, “could”, “will”, “should”, “expect”, “plan”, “anticipate”, “believe”, “estimate”, “plan”, ” intend ”,“ future ”,“ potential ”or“ continue ”and other similar expressions are intended to identify such forward-looking statements. All of these forward-looking statements are based on management’s current expectations with regard to future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those stated. or implied by these forward-looking statements. These risks and uncertainties include the possibility that the data from the TOPAZ clinical trial may be incompatible with the data observed in subsequent clinical trials, competition from third parties who develop products for similar uses, the ability of Scholar Rock to obtain, maintain and protect its intellectual property, Scholar Rock’s dependence on third parties for the development and manufacture of product candidates, including to provide clinical trials, Scholar Rock’s ability to manage expenses and obtain additional funding as necessary to support its business operations and establish and maintain strategic business alliances and new business initiatives, and the impacts of public health pandemics such as COVID-19 on business operations, including its clinical trials, as well as these risks more fully discussed in the section entitled “Risk Factors” of the Scholar Rock’s annual report on Form 10-K for the year ended December 31, 2020 as well as discussions of potential risks, uncertainties and other material factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Forward-looking statements represent the views of Scholar Rock only to date and should not be taken as representative of its views at a later date. All information in this press release is as of the date of the press release, and Scholar Rock assumes no obligation to update such information except as required by law.




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