GDF11

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today provided recent corporate updates and highlighted upcoming priorities for 2025. “ We are excited about the potential for apitegromab to help those living with SMA as the first and only muscle-targeted therapy for the treatment of this condition and are making excellent progress in finalizing our regulatory applications and preparing for commercial launch,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “ We remain on track for BLA and MAA submission in the first quarter of 2025 and are laser focused on bringing apitegromab to people with SMA as soon as possible. Building on the success of our leading, highly selective anti-myostatin approach in SMA, we plan to expand development of apitegromab into additional rare neuromuscular disorders to serve more people with unmet medical needs and as the foundation of a growing neuromuscular franchise.” Dr. Backstrom continued, “ Additionally, we are making excellent progress to deliver value across our pipeline, including the next wave of innovation in cardiometabolic disorders. We look forward to reporting topline data from our Phase 2 EMBRAZE proof-of-concept trial of apitegromab in combination with a GLP-1 receptor agonist in obesity and submitting an IND for SRK-439, our highly selective myostatin inhibitor specifically designed for obesity and cardiometabolic disorders.” 2025 Strategic Priorities and Upcoming Milestones: Scholar Rock plans to focus on three strategic priorities in 2025 as it transitions into a commercial-stage biopharmaceutical company, creating sustainable long-term value opportunities: Commercialize apitegromab in SMA. Expand the potential benefit of apitegromab. Advance Scholar Rock’s anti-myostatin program in cardiometabolic disorders. Commercialize apitegromab in SMA On track to submit a biologics license application (BLA) to the FDA and a marketing authorisation application (MAA) to the European Medicines Agency in 1Q 2025 for apitegromab in SMA. Preparing for U.S. commercial launch in 4Q 2025, and European launch to follow. Presenting detailed results from the pivotal Phase 3 SAPPHIRE trial at upcoming medical meetings in 2025. Expand the potential benefit of apitegromab Initiating Phase 2 OPAL clinical trial in SMA patients under two years of age in mid-2025 . The trial will evaluate apitegromab in patients under two years of age who have been or are continuing to be treated with any currently approved SMN therapy, including onasemnogene abeparvovec. Building upon the positive Phase 3 SAPPHIRE trial, the Company is exploring development of apitegromab in other rare neuromuscular disorders such as Duchenne muscular dystrophy, Becker muscular dystrophy, Facioscapulohumeral muscular dystrophy and amyotrophic lateral sclerosis to build a growing neuromuscular franchise. Advance the anti-myostatin program in cardiometabolic disorders Topline data expected in the second quarter of 2025 from the Phase 2 EMBRAZE proof-of-concept trial, a randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of apitegromab, a highly selective investigational myostatin inhibitor, to preserve muscle mass in overweight and obese adults who are taking a GLP-1 receptor agonist (GLP-1 RA). Trial outcomes will be used to guide clinical development of SRK-439. Plan to file an IND for SRK-439 for the treatment of obesity on the background of standard of care GLP-1 RAs in 3Q 2025. Scholar Rock disclosed new preclinical data which showed that SRK-439 protected against tirzepatide-induced muscle loss in DIO mice. Lean mass preservation was seen with doses of SRK-439 as low as 0.3 mg/kg and lean mass gain was observed at higher doses. The addition of SRK-439 with tirzepatide improved overall body composition with dose-dependent enhancement of fat mass loss. Scholar Rock believes that its existing cash, cash equivalents and marketable securities will be sufficient to fund the Company’s anticipated operating expenses and capital expenditure requirements into the fourth quarter of 2026. “ Scholar Rock is funded to scale up and drive forward our key priorities in 2025, including the commercialization of apitegromab, the EMBRAZE readout, the initiation of clinical development for SRK-439, and growing our pipeline,” said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. “ We believe in the transformative potential of our highly selective muscle-targeted approach and will advance our programs as we prepare to transition into a commercial company.” 2024 Highlights and Accomplishments: Reported positive topline data from the pivotal SAPPHIRE trial in October 2024. The trial achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement for apitegromab versus placebo in motor function as measured by the Hammersmith Functional Motor Scale – Expanded (HFMSE) in SMA patients on chronic dosing of standard of care therapies (either nusinersen or risdiplam). Continued high participation in the ONYX open-label extension study evaluating the long-term safety and efficacy of apitegromab in patients who completed the TOPAZ or SAPPHIRE trials. More than 90 percent of patients on combination therapy in the TOPAZ trial have completed 4 years of apitegromab treatment and enrolled into ONYX. Following trial completion, 98 percent of SAPPHIRE patients (185/188) enrolled in the ongoing ONYX open-label expansion study. Initiated Phase 2 EMBRAZE proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in May and completed enrollment in September. Presented new SRK-439 preclinical data at multiple conferences: ObesityWeek, American Diabetes Association’s 84th Scientific Sessions (ADA) and Keystone Symposia’s Obesity: Causes and Consequences meeting. Successfully completed upsized $345 million public offering in October to fund planned commercial launch in SMA and continue advancement of priority programs. J.P. Morgan Healthcare Conference Presentation and Webcast Scholar Rock management will highlight these updates in a corporate presentation at the 43 rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2025, at 9:45 a.m. PT (12:45 p.m. ET). 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 . An archived replay of the webcast will be available on the Company’s website for approximately 90 days following the presentation. About Apitegromab 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. Scholar Rock believes that its highly selective targeting of pro- and latent forms of myostatin with apitegromab may lead to a clinically meaningful improvement in motor function in patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. Apitegromab has not been approved for any use by the FDA or any other regulatory agency. About the Phase 3 SAPPHIRE Trial SAPPHIRE was a randomized, double-blind, placebo-controlled Phase 3 clinical trial that evaluated the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are receiving current standard of care (either nusinersen or risdiplam). SAPPHIRE enrolled 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 enrolled 32 patients aged 13-21 years old was also evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo. About EMBRAZE EMBRAZE is a randomized, double-blind, placebo-controlled, Phase 2 proof-of-concept trial evaluating the efficacy, safety and pharmacokinetics of apitegromab in adults with a body mass index (BMI) of >27 (overweight) or a BMI of >30 (obese) and taking a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of EMBRAZE is 100 subjects aged 18-65 who are overweight or obese without diabetes. As part of the study design, the treatment period is 24 weeks, and all subjects will receive a GLP-1 RA. In addition, all subjects will be randomized 1:1 to receive either apitegromab or placebo by intravenous (IV) infusion every four weeks during the 24-week treatment period. The primary endpoint is change from baseline at Week 24 in lean mass assessed by dual-energy X-ray absorptiometry. Secondary endpoints include additional weight loss measures, safety and tolerability, and pharmacokinetic outcomes. Exploratory endpoints at Weeks 24 and 32 include cardiometabolic parameters (e.g. HbA1c), body composition, and physical function. About SRK-439 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 cardiometabolic disorders, including obesity. Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass during weight loss. The efficacy and safety of SRK-439 have not been established and SRK-439 has not been approved for any use by the FDA or any other regulatory agency. About Scholar Rock Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role. This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at ScholarRock.com and follow @ScholarRock and on LinkedIn. The efficacy and safety of apitegromab, SRK-181, and SRK-439 have not been established and apitegromab, SRK-181, and SRK-439 have not been approved for any use by the FDA or any other regulatory agency. Scholar Rock ® is a registered trademark of Scholar Rock, Inc. 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. 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, results, its product candidate selection and development timing, including timing for the initiation of and reporting results from its preclinical studies and clinical trials for apitegromab, SRK-439, linavonkibart 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 3 trial of apitegromab or Part A or Part B of the Phase 1 trial of linavonkibart, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidate; 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, including from the EMBRAZE clinical trial; 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; its ability to establish or maintain strategic business alliances; its ability to receive priority or expedited regulatory review or to obtain regulatory approval of apitegromab; its ability to expand globally and the anticipated commercial launch in the United States of apitegromab in the fourth quarter of 2025; as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Form 10-K for the year ended December 31, 2023, and Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, 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.

KAWASAKI, Japan--( BUSINESS WIRE )--PeptiDream Inc., a public Kanagawa, Japan-based biopharmaceutical company (President: Patrick C. Reid, hereinafter “PeptiDream”)(Tokyo: 4587) today announced the highly promising results from the preclinical testing of the company’s oral myostatin inhibitors, administered in combination with semaglutide (a GLP-1 receptor agonist; a class of molecules known for the ability to aid in weight loss by modulating appetite and promoting satiety) in a mice model of obesity. PeptiDream has discovered a series of potent macrocyclic and bridged-macrocyclic peptide inhibitors of Myostatin. Myostatin (also known as growth differentiation factor 8, or GDF8), along with GDF11 and Activin, are members of the transforming growth factor-beta (TGFbeta) superfamily, and function in a complex process that regulates muscle growth and function. Numerous preclinical and clinical studies have suggested that myostatin inhibitors can increase lean muscle mass, improve physical strength, reduce visceral fat, and improve metabolic dysfunction, such as insulin-mediated glucose disposal, providing growing evidence that myostatin may be an important therapeutic target for the treatment of a variety of muscular dystrophies, such as Spinal muscular atrophy “SMA”, Facioscapulohumeral muscular dystrophy “FSHD”, Duchene muscular dystrophy “DMD” and other muscle wasting diseases, as well as more recently the potential treatment for obesity, metabolic syndrome, and type 2 diabetes mellitus. In preclinical DMD mice models, PeptiDream previously reported weekly administration of its peptide myostatin inhibitors, via subcutaneous or oral administration, resulted in both strong suppression of myostatin signaling and high exposure in muscle, yielding significant improvements in four-limb grip strength in treated animals. These extremely promising findings strongly supported their continued development for potential use in DMD, and given the results, PeptiDream initiated additional studies to investigate the use of its oral myostatin peptide inhibitors in obesity, where there is growing evidence that myostatin inhibitors can preserve lean body mass in individuals living with obesity and taking a GLP-1 receptor agonist (such as semaglutide). To this end, peptides from this series were tested in a diet-induced obesity (“DIO”) model where mice were given either a high-fat (60%) diet plus semaglutide (0.12 mg/kg, daily injection), or a high-fat diet (60%) plus semaglutide (0.12 mg/kg, daily injection) in combination with PeptiDream’s peptides orally administered (0.5, 1.5, 4.5 mg/kg; daily dose or 3, 10, 30 mg/kg; weekly dose). Body weight of the animals was measured every 2 days and Echo MRI was utilized to analyzes changes in both Fat Mass and Lean Body Mass at 14 and 28 days of treatment. Key Findings: Significant weight loss : Mice receiving the combination of oral peptide myostatin inhibitor with semaglutide showed a significant reduction in body weight compared to controls, with weight loss maintained over the study period. Lean mass preservation : Unlike many traditional weight-loss therapies that lead to a loss of both fat and lean muscle mass, both daily and weekly administration of PeptiDream’s oral peptide myostatin inhibitor successfully preserved lean body mass when administered in combination with semaglutide, highlighting its potential for improving body composition. Enhanced therapeutic potential : The results suggest that the synergistic effects of myostatin inhibition and semaglutide could be an effective strategy for patients with obesity, offering a novel approach to weight management that avoids muscle loss, a common drawback of many current obesity treatments. “We are very excited to share these preclinical results. Our myostatin inhibitors showed significant lean body mass preservation, with both daily and weekly oral administration, effectively counter-acting the lean mass loss associated with semaglutide-induced weight loss. These findings provide compelling evidence of the first-in-class potential of our oral peptide myostatin inhibitors to be used in combination with other weight loss agents to transform the management of healthy weight loss, as well as providing more convenient and patient-friendly oral treatment options to patients.” said Patrick C. Reid PhD, President & CEO of PeptiDream . PeptiDream presented a portion of these preclinical results at its R&D Day event on December 12, 2024 ( R&D Day presentation materials are available on the PeptiDream website ). PeptiDream is continuing preclinical development activities while exploring clinical development options for this exciting program, including discussions with potential partners interested in licensing/partnering the program. About PeptiDream Inc. PeptiDream Inc. (Tokyo Stock Exchange Prime Market 4587) is leading the translation of macrocyclic peptides into a whole new class of innovative medicines to address unmet medical needs and improve the quality of life of patients worldwide. In its radiopharmaceutical business, through its wholly-owned subsidiary PDRadiopharma, PeptiDream markets and sells a number of approved radiopharmaceuticals and radiodiagnostics in Japan, as well as leveraging its proprietary Peptide Discovery Platform System (PDPS) technology to discover and develop a deep pipeline of innovative targeted radiotherapeutics and radiodiagnostics, spanning both wholly-owned internal programs and globally partnered programs. In its non-radiopharmaceutical business, PeptiDream is similarly leveraging PDPS to discover and develop a broad and diverse pipeline of investigational peptide therapeutics, peptide drug conjugates (PDC) and multi-functional peptide conjugates (MPC) across an extensive global network of discovery and development partners. PeptiDream is headquartered in Kawasaki, Japan. For more information about our company, science and pipeline, please visit www.peptidream.com/en/ .

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today reported financial results and corporate updates for the third quarter ended September 30, 2024. “ We were thrilled to announce positive and statistically significant topline data from the pivotal Phase 3 SAPPHIRE trial, a major achievement that underscores apitegromab’s potential to redefine the standard of care for patients with SMA,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “ The positive SAPPHIRE trial validates our approach to targeting pro-and latent myostatin and demonstrates apitegromab’s potentially transformative benefit. Based on these results, we plan to submit a BLA and MAA in the first quarter of 2025 and are working diligently to prepare for the commercial launch of apitegromab, and if approved, to deliver the potentially transformative benefits of apitegromab to children and adults living with SMA.” Dr. Backstrom continued, “ The robust results from SAPPHIRE also reinforce the broader potential of our selective myostatin inhibition strategy across our pipeline, with potential readthrough to our cardiometabolic program as we evaluate apitegromab in obesity in our Phase 2 EMBRAZE proof-of-concept trial. We were pleased to announce in September that the EMBRAZE trial is fully enrolled, and we expect to report initial data in the second quarter of next year.” Company Highlights and Upcoming Milestones SMA Program 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 first muscle-targeted therapy to show clinical proof-of-concept in SMA. Reported positive topline data from Phase 3 SAPPHIRE clinical trial . The study achieved its primary endpoint demonstrating a statistically significant and clinically meaningful improvement for apitegromab versus placebo in motor function as measured by the gold standard HFMSE in SMA patients on chronic dosing of standard of care therapies (either nusinersen or risdiplam). In the main efficacy population (ages 2-12), the mean difference in change from baseline in HFMSE was 1.8 points (p=0.0192) for all patients receiving apitegromab 10 mg/kg and 20 mg/kg (n=106) compared to placebo (n=50). Patients receiving 20 mg/kg of apitegromab (n=53) showed a 1.4 point mean difference compared to placebo (p=0.1149). The prespecified analysis of the 10 mg/kg dose showed that patients receiving 10 mg/kg of apitegromab (n=53) showed a mean difference in change from baseline in HFMSE of 2.2 points compared to placebo (nominal p=0.0121). Based upon PK/PD data from the SAPPHIRE trial, similar levels of target engagement were observed for the 10 mg/kg and 20 mg/kg dose groups. Scholar Rock plans to submit a U.S. BLA and a European Union MAA in 1Q 2025. Presented preliminary baseline characteristics from SAPPHIRE during a poster presentation at the 29 th Annual Congress of the World Muscle Society in October in Prague, Czech Republic. Analyses of the full Phase 3 SAPPHIRE data are ongoing, and Scholar Rock plans to present detailed results at an upcoming medical conference in early 2025. The ONYX open-label, multi-center extension study remains ongoing. The study is evaluating the long-term safety and efficacy of apitegromab in patients who completed the TOPAZ or SAPPHIRE trials. More than 90 percent of patients on combination therapy in the TOPAZ trial have completed 4 years of apitegromab treatment and enrolled into ONYX. Following trial completion, 98 percent of SAPPHIRE patients (185/188) enrolled in the ongoing ONYX open-label expansion study. On track to initiate OPAL clinical trial in SMA patients under two years old in mid-2025. Cardiometabolic Program SRK-439 is a novel, preclinical, investigational myostatin inhibitor that binds to pro- and latent myostatin with high affinity and is selective for myostatin (i.e., no GDF11 or Activin A binding), and is initially being developed for the treatment of obesity. Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass during weight loss. Completed enrollment of Phase 2 EMBRAZE proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in September. The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of apitegromab, a highly selective investigational myostatin inhibitor, to preserve muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. Data are expected in the second quarter of 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. Immuno-Oncology Program SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy, such as anti-PD-(L)1 antibodies, in patients with advanced cancer. New SRK-181 data from the Phase 1 DRAGON proof-of-concept trial presented at the SITC 39 th Annual Meeting in November. The presentation, “ DRAGON Trial: Durable remission rate with the latent TGFβ1 inhibitor linavonkibart (SRK-181) and pembrolizumab in patients with immune checkpoint inhibitor resistant advanced cancers,” highlighted new clinical and biomarker results. Highlights since the Company’s last update on the DRAGON trial include one additional complete response, seen in the clear cell renal cell (ccRCC) cohort (ORR=23.3%) and durable responses in multiple cohorts. Additionally, biomarker results continue to support proof of mechanism; new data shows suppressed granulocytic myeloid-derived suppressor cells (gMDSC) levels in the tumor microenvironment of responders. Notably new biomarker data in the ccRCC cohort supports a potential patient selection strategy with positive correlations between clinical response and baseline CD8+ infiltration status, elevated baseline Treg levels, and elevated baseline TGFβ1 levels in the tumor compartment. Enrollment of the DRAGON trial was completed in December 2023, and patients who remain on the study continue to be treated. Other Corporate Updates Appointed Beth Shafer, Ph.D., to the newly created role of Chief Business Officer. Ms. Shafer brings over 20 years of biopharmaceutical industry leadership and expertise to Scholar Rock, where she will drive the Company’s long-term corporate and business development strategy. Raised $345 million in upsized follow-on offering. In October, the Company completed an upsized follow-on offering raising aggregate gross proceeds of $345 million. Third Quarter 2024 Financial Results For the quarter ended September 30, 2024, net loss was $64.5 million or $0.66 per share compared to a net loss of $42.4 million or $0.53 per share for the quarter ended September 30, 2023. The Company did not record any revenue for the quarter ended September 30, 2024, or for the quarter ended September 30, 2023. Research and development expense was $48.7 million for the quarter ended September 30, 2024, compared to $30.3 million for the quarter ended September 30, 2023. The increase was primarily attributable to costs associated with the ONYX and EMBRAZE trials, and the development of SRK-439. General and administrative expenses were $16.1 million for the quarter ended September 30, 2024, compared to $13.3 million for the quarter ended September 30, 2023. The increase was due to employee-related costs. As of September 30, 2024, Scholar Rock had cash, cash equivalents and marketable securities of approximately $139.1 million ($463.5 million, on a pro forma basis, including the approximately $324.4 million raised in the October 2024 equity offering). Along with cash available to the Company, runway is extended into 4Q 2026. “ Supported by our recent upsized public offering on the heels of successful SAPPHIRE data, Scholar Rock is advancing our mission from a position of strength as we move forward with preparations for our first commercial launch in the fourth quarter of 2025,” said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. “ We are working tirelessly to obtain approval for apitegromab and deliver it to patients with SMA as soon as possible, as well as continuing to advance our portfolio of highly selective myostatin inhibition programs.” Conference Call Information Management will provide an update on the Company and discuss third quarter 2024 results via conference call on Tuesday, November 12 at 8:15 am ET. To access the live conference call, participants may register here . The live audio webcast of the call will be available under “Events and Presentations” in the Investor Relations section of the Scholar Rock website at http://investors.scholarrock.com . To participate via telephone, please register in advance here . Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. An archived replay of the webcast will be available on the Company’s website for approximately 90 days. About Scholar Rock Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily of cell proteins and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role. This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. The efficacy and safety of apitegromab, SRK-181, and SRK-439 have not been established and apitegromab, SRK-181, and SRK-439 have not been approved for any use by the FDA or any other regulatory agency. Scholar Rock ® is a registered trademark of Scholar Rock, Inc. 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 we post on our website, Twitter or LinkedIn could be deemed material information. As a result, we encourage investors, the media and others interested to review the information we post there regularly. 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. 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 and SRK-181 and its preclinical programs, including SRK-439, and indication selection and development timing, including the timing of any regulatory submissions, the therapeutic potential, clinical benefits and safety of any product candidates, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, its cash runway, expectations relating to commercial launch in the US in the fourth quarter of 2025, expectations regarding the achievement of important milestones, 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 3 SAPPHIRE trial will be sufficient to support regulatory approval, that the full results from the Phase 3 SAPPHIRE trial may differ from the topline data, that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, or Part A or Part B 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 or ongoing clinical trials of the same product candidates; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials; 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; and 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 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, 2024, 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. Scholar Rock Holding Corporation Condensed Consolidated Statements of Operations (unaudited) (in thousands, except share and per share data) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Operating expenses Research and development $ 48,719 $ 30,337 $ 134,185 $ 86,939 General and administrative 16,061 13,335 48,512 36,324 Total operating expenses 64,780 43,672 182,697 123,263 Loss from operations (64,780 ) (43,672 ) (182,697 ) (123,263 ) Other income (expense), net 301 1,313 2,857 3,600 Net loss $ (64,479 ) $ (42,359 ) $ (179,840 ) $ (119,663 ) Net loss per share, basic and diluted $ (0,66 ) $ (0,53 ) $ (1,86 ) $ (1,49 ) Weighted average common shares outstanding, basic and diluted 97,050,637 80,606,438 96,587,149 80,115,143 Scholar Rock Holding Corporation Condensed Consolidated Balance Sheets (unaudited) (in thousands) September 30, 2024 December 31, 2023 Assets Cash, cash equivalents and marketable securities $ 139,065 $ 279,938 Other current assets 13,862 8,256 Total current assets 152,927 288,194 Other assets 26,206 22,841 Total assets $ 179,133 $ 311,035 Liabilities and Stockholders' Equity Current liabilities $ 39,372 $ 32,741 Long-term liabilities 60,565 53,076 Total liabilities 99,937 85,817 Total stockholders' equity 79,196 225,218 Total liabilities and stockholders' equity $ 179,133 $ 311,035