Edgewise Therapeutics, Inc. is a clinical-stage biopharmaceutical company. It focuses on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is significant unmet medical need. Its platform utilizes custom-built throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue, initially focused on addressing rare neuromuscular and cardiac diseases. The company was founded by Badreddin Edris, Alan Russel and Peter A. Thompson in 2017 and is headquartered in Boulder, CO.
相关临床试验
21
5 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2017
进行中（未招募）
4
19.1%
已完成
12
57.1%
Enrolling By Invitation
1
4.8%
招募中
4
19.1%
暂无批准数据
- Nitza Thomasson joined Servier in March 2025 as Executive Director and Global Head of R&D Neurology to build the company's rare neurology therapeutic area from scratch. - Servier currently has three neurology drugs in its pipeline, targeting rare refractory epilepsies, movement disorders, neuromuscular disorders, and other conditions. - The company recently strengthened its portfolio through the acquisition of Edgewise Therapeutics' neuromuscular dystrophy pipeline. - Thomasson emphasizes that success in rare neurology requires embracing scientific uncertainty, adapting approaches to each disease, and building a team culture grounded in trust and high expectations.
- Immutrin has appointed Dr. Robert Blaustein, former Edgewise and Merck executive, as Chief Medical Officer to guide clinical strategy for its novel amyloidosis therapy. - Dr. Blaustein brings over 15 years of cardiovascular drug development experience, including leading the successful Phase 3 VICTORIA heart failure trial for vericiguat. - The company is preparing to advance its lead antibody therapy toward first-in-human studies for ATTR cardiomyopathy treatment. - Immutrin has established a US subsidiary to support planned clinical activities in North America as it transitions to a clinical-stage organization.
- Edgewise Therapeutics has expanded enrollment in its pivotal GRAND CANYON Phase 3 trial of sevasemten from 120 to 175 patients, increasing statistical power to approximately 98% to detect a 1.8-point improvement in North Star Ambulatory Assessment. - The company remains on track for Phase 3 readout in Q4 2024 and targets potential regulatory approval by end-Q4 2027, with first full-year revenue projected for 2028 if results are positive. - For its hypertrophic cardiomyopathy program, Edgewise has revised EDG-7500 screening protocols to address earlier atrial fibrillation concerns and plans to disclose Part D results covering both obstructive and non-obstructive HCM before initiating Phase 3 in Q4. - The company reports no atrial fibrillation burden or correlation between plasma concentration and left ventricular ejection fraction following implementation of continuous patch monitoring and enhanced screening measures.
- Edgewise Therapeutics reported positive updates from its ongoing CIRRUS-HCM Phase 2 trial of EDG-7500, an oral cardiac sarcomere modulator for hypertrophic cardiomyopathy. - The 25 mg dose cohort demonstrated clinical activity across key HCM disease markers while maintaining a favorable safety profile with no clinically meaningful changes in left ventricular ejection fraction. - The company remains on track to deliver comprehensive efficacy and safety data from Part D in Q2 2026 and plans Phase 3 trial initiation by end of 2026. - EDG-7500's differentiated safety profile relative to cardiac myosin inhibitors supports its potential use in diverse HCM patient populations.
- The Duchenne Muscular Dystrophy market reached approximately $2.15 billion in 2023 and is expected to grow significantly due to increased drug uptake and anticipated gene therapy launches. - Over 75 companies are actively developing pipeline therapies for DMD, with recent FDA designations including Atossa Therapeutics' (Z)-Endoxifen receiving Rare Pediatric Disease designation. - Capricor Therapeutics announced positive Phase 3 HOPE-3 trial results for Deramiocel, while the FDA accepted their BLA for review with Priority Review designation. - Current approved treatments include EMFLAZA, VYONDYS 53, EXONDYS 51, AMONDYS 45, VILTEPSO, and gene therapy ELEVIDYS in the US, with AGAMREE launched in Germany in 2024.
- Edgewise Therapeutics appointed Christopher Martin, former Chief Commercial Officer of Verona Pharma, to its Board of Directors to support upcoming commercial launches. - Martin brings extensive experience in product launches, having led commercial strategies for multiple biotech companies including Verona Pharma's Ohtuvayre and SK Life Science's XCOPRI. - The appointment comes as Edgewise prepares for its first commercial launch of sevasemten in Becker muscular dystrophy and advances its cardiovascular asset EDG-7500 to Phase 3 trials. - Edgewise's pipeline includes three novel therapeutics targeting muscle diseases: sevasemten for muscular dystrophies, EDG-7500 for hypertrophic cardiomyopathy, and EDG-15400 for heart failure.
- The FDA declined to consider Edgewise Therapeutics' sevasemten for accelerated approval in Becker muscular dystrophy, citing insufficient data from the Phase II CANYON trial. - The agency affirmed that North Star Ambulatory Assessment scores can serve as a clinically meaningful endpoint for traditional approval, providing a clear regulatory pathway forward. - Edgewise must now rely on its ongoing Phase III GRAND CANYON trial, which is expected to read out in Q4 2026 and is designed to demonstrate statistical significance versus placebo. - The company's stock dropped 10% following the announcement, though analysts noted the decision was not unexpected given the FDA's tightened stance on accelerated approval requirements.
• Haya Therapeutics has raised $65 million in Series A funding to develop therapeutics targeting long non-coding RNAs in the previously dismissed "junk DNA" regions of the human genome. • The company's lead candidate, HTX-100, targets a long non-coding RNA called "Wisper" that plays a central role in non-obstructive hypertrophic cardiomyopathy, with clinical trials expected to begin soon. • Unlike competitors' approaches that focus on reducing heart contractions, Haya's technology aims to address the underlying disease mechanisms by suppressing fibrosis and reprogramming disease-driving cell states.
• Pacira BioSciences has reached a settlement agreement with Fresenius, Jiangsu Hengrui Pharmaceuticals, and eVenus Pharmaceuticals regarding patents for its pain management drug Exparel, protecting market exclusivity until 2030. • Under the agreement, Fresenius will begin limited-volume sales of generic bupivacaine liposome injectable suspension in early 2030, with gradual market share increases reaching the high thirties percent by the final years of the agreement. • The settlement strategically preserves Pacira's market dominance for its lead drug Exparel for nearly a decade before allowing controlled generic entry, significantly extending protection beyond potential patent challenges.
- Edgewise Therapeutics received an FDA determination that mid-stage study data for its muscular dystrophy drug was insufficient for accelerated approval, though the agency validated the physical ability endpoint for traditional approval. - The company disclosed safety concerns in a separate heart disease trial for EDG-7500, including two cases of dangerous atrial fibrillation that prompted analyst downgrades. - Scotiabank downgraded the stock from Sector Outperform to Sector Perform, cutting the price target from $50 to $14, citing safety issues that undermine the drug's competitive positioning. - Despite setbacks, J.P.Morgan analysts remain positive on the company's cardiovascular assets as the primary value driver in the mid-term.
