TOPAZ

Scholar Rock to Present New 24-Month Data from Phase 2 TOPAZ Study at Two International Conferences: World Muscle Society Congress and International Scientific Congress on Spinal Muscular Atrophy

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Tuesday, October 4, 2022
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Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).

Key Points: 
  • Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may result in a clinically meaningful improvement in motor function in patients with SMA.
  • Spinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children.
  • It is characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness.

Scholar Rock to Present New Apitegromab Data Including 24-Month Efficacy and Safety Data from TOPAZ Phase 2 Trial at the 2022 Annual Cure SMA Conference

Retrieved on: 
Thursday, June 9, 2022
Health, Genetics, Clinical Trials, General Health, Pharmaceutical, Biotechnology, FTD, Company, Conference, RPD, Review, COVID-19, Research, European Medicines Agency, Neuromuscular Disorders, Neurology, Cancer, U.S. Securities and Exchange Commission, SMA, Intellectual property, Rock, Food, CEO, Investor, ODD, Quality of life, Social media, Securities Act of 1933, Human, EMA, Johns Hopkins University, Webcast, Security (finance), Myostatin, Doctor of Pharmacy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Risk, ID, Patient, Fast Track, Spinal muscular atrophy, Private Securities Litigation Reform Act, Fibrosis, LLC, Drug Quality and Security Act, FAQ, LinkedIn, Disease, TOPAZ, FDA, Growth, NASDAQ, Twitter, File, Clinical trial, Detail, Orphan drug, Safety, Pharmaceutical industry, Management, Gongshi, Pediatrics

Details of the podium presentations are as follows:

Key Points: 
  • Details of the podium presentations are as follows:
    Presenter: Hemal Shah, PharmD, an independent HEOR consultant for Scholar Rock, and CEO of Value Matters, LLC.
  • To participate in the call, please dial 833-519-1308 (domestic) or 914-800-3874 (international) and refer to conference ID: 6495684.
  • A webcast of the call will also be available on the Investors & Media section of the Scholar Rock website at http://investors.scholarrock.com .
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.

Scholar Rock Reports First Quarter 2022 Financial Results and Updates Strategic Priorities

Retrieved on: 
Monday, May 16, 2022
Biotechnology, Pharmaceutical, Health, Clinical Trials, SMA, DRAGON, U.S. Securities and Exchange Commission, UC, Muscular Dystrophy Association, MDA, Research, Review, Episcopal conference, MDSC, Rock, Conference, Energy Independence and Security Act of 2007, Private Securities Litigation Reform Act, Twitter, Clear cell renal cell carcinoma, Cancer, Risk, MEL, Social media, Neuromuscular Disorders, Squamous cell carcinoma, Clinical trial, COVID-19, NSCLC, Securities Act of 1933, Large-cell lung carcinoma, CMO, Clinical data management, Growth, Part, FAQ, Company, Investor, Cure, Global health, Fibrosis, Gongshi, Human, LinkedIn, SAPPHIRE, Security (finance), American Academy, Population, Ageing, Leadership, File, NASDAQ, Neurology, Workforce, Intellectual property, Safety, TOPAZ, Completion, Pharmaceutical industry, Management, Patient

“We are highly confident in the transformative potential of apitegromab based upon the Phase 2 TOPAZ data we have released to date. We look forward to presenting two-year extension trial data in the coming weeks,” said Nagesh Mahanthappa, Ph.D., Founding Chief Executive Officer of Scholar Rock. “Based upon the strength of the data released to date and our commitment to the SMA community, this narrower focus and associated headcount reduction is necessary to execute our core mission -- completing the Phase 3 SAPPHIRE trial to bring apitegromab, our highly innovative therapeutic candidate, to patients suffering with this devastating disease.”

Key Points: 
  • In conjunction with the pipeline prioritization, these changes are expected to extend the cash runway into the fourth quarter of 2023.
  • We look forward to presenting two-year extension trial data in the coming weeks, said Nagesh Mahanthappa, Ph.D., Founding Chief Executive Officer of Scholar Rock.
  • The Company expects research expenses to decline beginning in the third quarter of 2022 due to the portfolio updates and workforce reduction announced today.
  • As of March 31, 2022, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $210 million.

Scholar Rock Presents Data Analysis of Multiple Efficacy Endpoints from the Apitegromab TOPAZ Phase 2 Trial at the 2022 European Paediatric Neurology Society Congress

Retrieved on: 
Thursday, April 21, 2022
Oncology, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, EMA, RPD, File, U.S. Securities and Exchange Commission, COVID-19, Pathology, Security (finance), NASDAQ, Muscle weakness, Risk, Patient, LinkedIn, Neuromuscular Disorders, Review, European Medicines Agency, Ground Floor, Hammersmith, FDA, Securities Act of 1933, Myostatin, Food, Human, Private Securities Litigation Reform Act, Motor neuron, SMA, Social media, Company, Gene, Orphan drug, Congress, SMN1, Twitter, Spinal muscular atrophy, Investor, Genetic disorder, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Drug Quality and Security Act, Intellectual property, Growth, SEC, Safety, Gongshi, Fast Track, Therapy, Cancer, Survival, ODD, FTD, Fibrosis, Rock, Clinical trial, EPNS, TOPAZ, Atrophy, FAQ, SMN2, SMN, Pharmaceutical industry

Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.

Key Points: 
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.
  • The efficacy and safety of apitegromab have not been established and apitegromab has not been approved for any use by the FDA or any other regulatory agency.
  • Spinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children.
  • Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path.

Scholar Rock Reports Full Year 2021 Financial Results and Highlights Business Progress

Retrieved on: 
Monday, March 7, 2022
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Private Securities Litigation Reform Act, Neuromuscular Disorders, File, SAPPHIRE, Ageing, Research, Social media, SMN, Fibrosis, Population, CFO, TOPAZ, Gongshi, MEL, Twitter, SMA, Security (finance), Safety, U.S. Securities and Exchange Commission, Interim, LinkedIn, MDSC, Growth, Large-cell lung carcinoma, FAQ, Drug development, Part, Company, UC, COVID-19, Squamous cell carcinoma, Clear cell renal cell carcinoma, Cancer, Securities Act of 1933, Human, Rock, COO, Clinical trial, Risk, Drug Quality and Security Act, Review, Intellectual property, DRAGON, Investor, NSCLC, Publication, NASDAQ, Pharmaceutical industry, Airline, Patient

The randomized, double-blind, placebo-controlled Phase 3 clinical trial is evaluating apitegromab as add-on therapy for patients on either nusinersen or risdiplam.

Key Points: 
  • The randomized, double-blind, placebo-controlled Phase 3 clinical trial is evaluating apitegromab as add-on therapy for patients on either nusinersen or risdiplam.
  • As of December 31, 2021, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $253.0 million, which is expected to fund the Companys operations into mid-2023.
  • Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path.
  • Scholar Rock is a registered trademark of Scholar Rock, Inc.

Scholar Rock Provides Corporate Update and Highlights Priorities for 2022

Retrieved on: 
Monday, January 10, 2022
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, ATM, Clinical trial, Safety, Neuromuscular Disorders, Silicon Valley Bank, Extracellular matrix, Growth, Ageing, LinkedIn, Risk, Security (finance), Antibody, Fibrosis, Cancer, CSO, Rock, Business operations, Immune system, NASDAQ, Interim, U.S. Securities and Exchange Commission, SAPPHIRE, DRAGON, Partnership, Research, Cell, Private Securities Litigation Reform Act, Company, Human, Investor, SMN, Exercise, Gilead Sciences, COVID-19, Therapy, Gilead, Population, CFO, Intellectual property, TOPAZ, SMA, Pharmaceutical industry, Medicine, Vaccine, Part, Gongshi, Patient

The novel anti-fibrotic antibodies discovered during this collaboration demonstrate the unique capabilities of the discovery platform we have built at Scholar Rock, said Gregory Carven, CSO of Scholar Rock.

Key Points: 
  • The novel anti-fibrotic antibodies discovered during this collaboration demonstrate the unique capabilities of the discovery platform we have built at Scholar Rock, said Gregory Carven, CSO of Scholar Rock.
  • We made great progress across our portfolio in 2021 and were carrying that momentum into 2022, said Ted Myles, CFO and Head of Business Operations of Scholar Rock.
  • Scholar Rock is a registered trademark of Scholar Rock, Inc.
  • 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 Reports Third Quarter 2021 Financial Results and Highlights Business Progress

Retrieved on: 
Tuesday, November 9, 2021
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Phase 3 Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients Remains on Track to Initiate by Year-End 2021.

Key Points: 
  • Phase 3 Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients Remains on Track to Initiate by Year-End 2021.
  • Scholar Rock is preparing to announce the design of the Phase 3 pivotal study later this month.
  • As we approach the end of 2021, I am incredibly proud of the execution by the entire Scholar Rock team this year.
  • Scholar Rock is a registered trademark of Scholar Rock, Inc.

Scholar Rock to Present Apitegromab TOPAZ Phase 2 Pharmacologic Data at the 2021 World Congress of Neurology

Retrieved on: 
Sunday, October 3, 2021
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Gongshi, Food, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Spinal muscular atrophy, Clinical trial, ODD, SMA, Serum, Myostatin, Motor neuron, Muscle weakness, SMN, Rock, Risk, Congress, Security (finance), Therapy, European Medicines Agency, Private Securities Litigation Reform Act, NASDAQ, U.S. Securities and Exchange Commission, FDA, Cancer, Fast Track, Patient, Pathology, LinkedIn, RPD, Gene, Genetic disorder, WCN, Investor, Company, Growth, COVID-19, SMN2, Human, SMN1, TOPAZ, Atrophy, FTD, Safety, Survival, Fibrosis, Orphan drug, EMA, Neuromuscular Disorders, Pharmaceutical industry, Apitegromab, SMA, Scholar Rock, APITEGROMAB, SMA, SCHOLAR ROCK

Time: Virtual e-posters are available for viewing for the duration of the congress (October 3-7, 2021) and will be available on demand for 3 months post conference.

Key Points: 
  • Time: Virtual e-posters are available for viewing for the duration of the congress (October 3-7, 2021) and will be available on demand for 3 months post conference.
  • Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.
  • Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path.

 Scholar Rock Presents Additional Data Analyses from the Apitegromab TOPAZ Phase 2 Trial at the World Muscle Society 2021 Virtual Congress

Retrieved on: 
Thursday, September 23, 2021
FDA, Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, Gongshi, Food, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Spinal muscular atrophy, Scoliosis, Clinical trial, SMA, Myostatin, Motor neuron, Muscle weakness, ODD, SMN, Rock, Risk, Security (finance), Therapy, European Medicines Agency, NASDAQ, FDA, Cancer, BST, Fast Track, Patient, Pathology, Poster, LinkedIn, RPD, Gene, Genetic disorder, WMS, Private Securities Litigation Reform Act, Investor, Company, Growth, COVID-19, SMN2, Human, TOPAZ, SMN1, Atrophy, FTD, Safety, Survival, Fibrosis, Orphan drug, EMA, Neuromuscular Disorders, Pharmaceutical industry, Medicine, Vaccine, Medical imaging, Apitegromab, SMA, Scholar Rock, APITEGROMAB, SMA, SCHOLAR ROCK

Greater increases in HFMSE (non-ambulatory) and Revised Hammersmith Scale (ambulatory) scores were seen in patients who were not limited by scoliosis or joint contractures.

Key Points: 
  • Greater increases in HFMSE (non-ambulatory) and Revised Hammersmith Scale (ambulatory) scores were seen in patients who were not limited by scoliosis or joint contractures.
  • Virtual poster presentations (LBP.10 and EP.277) on September 23, 2021, at 16:30-18:30 BST
    E-Posters available to view on demand and exhibition area open starting Monday, September 20, 2021.
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.
  • Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path.

Scholar Rock Reports Second Quarter 2021 Financial Results and Highlights Business Progress

Retrieved on: 
Tuesday, August 10, 2021
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Gongshi, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, Business operations, Food, Conference, Cancer, Private Securities Litigation Reform Act, Spinal muscular atrophy, TOPAZ, Growth, Type, European Medicines Agency, BLA, PRIME, Company, U.S. Securities and Exchange Commission, Fast Track, CFO, Risk, WHO, Nusinersen, Squamous cell carcinoma, Interim, Pharmacokinetics, Population, Myostatin, NASDAQ, Research, Large-cell lung carcinoma, LinkedIn, Review, Fibrosis, Cure, Human, Security (finance), Part, Investor, FDA, Rock, DRAGON, World Health Organization, Neuromuscular Disorders, Johns Hopkins School of Medicine, Clinical trial, COVID-19, Biologics license application, Pharmaceutical industry, Lithium

Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today reported financial results for the second quarter ended June 30, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs.

Key Points: 
  • Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today reported financial results for the second quarter ended June 30, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs.
  • Research and development expense was $25.6 million for the quarter ended June 30, 2021 compared to $17.0 million for the quarter ended June 30, 2020.
  • General and administrative expense was $9.3 million for the quarter ended June 30, 2021 compared to $6.4 million for the quarter ended June 30, 2020.
  • Scholar Rock is a registered trademark of Scholar Rock, Inc.