Edgewise Therapeutics
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2017-01-01
- Employees
- 88
- Market Cap
- -
- Website
- http://www.edgewisetx.com
- Introduction
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.
Haya Therapeutics Secures $65M to Develop Drugs Targeting the 'Dark Genome'
• 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 Secures Future of Exparel with Strategic Patent Settlement Agreement
• 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's EDG-7500 Shows Promise in Phase 2 HCM Trial with Improved Safety Profile
• Edgewise Therapeutics' novel cardiac sarcomere modulator EDG-7500 demonstrated significant reductions in left ventricular outflow tract gradient in obstructive HCM patients without meaningful changes in ejection fraction.
• The CIRRUS-HCM trial showed dose-dependent improvements in both obstructive and nonobstructive HCM, with the 100mg dose reducing NT-proBNP biomarker levels by 62% and 42% respectively.
• EDG-7500's mechanism as a sarcomere modulator may offer advantages over existing cardiac myosin inhibitors like Bristol Myers Squibb's Camzyos, with promising efficacy and safety data after four weeks of treatment.
FDA Misses Deadline for Full Approval Decision on Novavax's COVID-19 Vaccine
• The U.S. Food and Drug Administration (FDA) has missed its April 1 deadline to decide on granting full approval to Novavax's protein-based COVID-19 vaccine, which currently has Emergency Use Authorization.
• The delay comes shortly after the resignation of Peter Marks, the FDA's top vaccine regulator who played a key role in COVID-19 vaccine approvals during the pandemic.
• According to reports, FDA senior leaders have indicated that Novavax's application needs more data, making approval unlikely in the near term, though the company maintains it has responded to all information requests.
Edgewise Therapeutics Announces 2025 Priorities Following Positive Phase 2 Results
• Edgewise Therapeutics is prioritizing the completion of recruitment for the GRAND CANYON trial for Becker muscular dystrophy in early 2025.
• The company plans to seek FDA feedback on Phase 2 CANYON trial results for sevasemten in Becker muscular dystrophy during the first half of 2025.
• Initial positive results from the Phase 2 CIRRUS-HCM trial of EDG-7500 in obstructive hypertrophic cardiomyopathy were reported, with more data expected in 2025.
• Edgewise strengthened its financial position with $232 million raised, supporting ongoing muscular dystrophy and cardiovascular programs.
UNITY Biotechnology Appoints Federico Grossi as Chief Medical Officer
• UNITY Biotechnology appointed Federico Grossi as Chief Medical Officer, enhancing its leadership with his extensive experience in ophthalmology and drug development.
• Dr. Grossi previously led the development of SYFOVRE for geographic atrophy at Apellis Pharmaceuticals, bringing a proven track record to UNITY.
• His appointment is timely as UNITY anticipates Phase 2b ASPIRE trial results for UBX1325 in diabetic macular edema (DME).
• UNITY aims to leverage Dr. Grossi's expertise to advance its pipeline of senolytic treatments for age-related retinal diseases.
FDA Gears Up for Critical Decisions on Alzheimer's, Breast Cancer, and Neurological Therapies in Early 2025
• The FDA is set to decide on Biogen and Eisai's Leqembi for monthly intravenous maintenance in early Alzheimer's disease, potentially improving patient convenience.
• AstraZeneca and Daiichi Sankyo await a decision on Dato-DXd for metastatic HR-positive, HER2-negative breast cancer, offering a new antibody-drug conjugate approach.
• Vertex's suzetrigine, a non-opioid analgesic for moderate-to-severe acute pain, anticipates FDA verdict, representing a novel drug class for pain management.
• SpringWorks' mirdametinib is under priority review for neurofibromatosis type 1-associated plexiform neurofibromas, addressing a significant unmet need.
Key Advances in Neuromuscular and Movement Disorder Treatments Highlighted in 2024
• AbbVie's Vyalev, a 24-hour subcutaneous levodopa infusion, gained FDA approval for managing motor fluctuations in advanced Parkinson's, offering a novel therapeutic approach.
• Intellia's CRISPR therapy, NTLA-2001, demonstrated safe redosing in ATTR amyloidosis patients, achieving additive pharmacodynamic effects on the target protein.
• A phase 3 study revealed that buntanetap is a safe and well-tolerated drug which improves motor, nonmotor, and cognitive symptoms of Parkinson's disease.
• The FDA supported using αSyn-SAA biomarker in Parkinson's clinical trials, enhancing therapeutic development through improved diagnostic precision.
Tenaya Therapeutics' TN-201 Shows Promise in Early Hypertrophic Cardiomyopathy Trial
• Tenaya Therapeutics reported initial data from its MyPEAK-1 trial, evaluating TN-201 gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM).
• The Phase 1b study showed TN-201 was generally well-tolerated in the first cohort, with manageable liver enzyme elevations and no cardiac toxicities reported.
• Early data suggest successful cardiac transduction and increased expression of TN-201 RNA and MyBP-C protein levels in treated patients.
• Tenaya plans to report additional data from Cohort 1 in the first half of 2025 and initial data from Cohort 2 in the second half of 2025.
FDA Halts PepGen's DMD Trial, Sanofi's MS Drug Tolebrutinib Gains Breakthrough Status, and Sevasemten Shows Promise in Becker Muscular Dystrophy
• The FDA has placed a clinical hold on PepGen's Phase 2 CONNECT2-EDO51 trial for PGN-EDO51 in Duchenne muscular dystrophy (DMD) due to regulatory questions.
• Sanofi's tolebrutinib, a BTK inhibitor, received breakthrough therapy designation from the FDA for non-relapsing secondary progressive multiple sclerosis (nrSPMS).
• Edgewise Therapeutics' sevasemten met its primary endpoint in a Phase 2 trial for Becker muscular dystrophy, showing a significant decrease in creatine kinase.
Edgewise Therapeutics' Sevasemten Shows Promise in Phase 2 Trial for Becker Muscular Dystrophy
• Edgewise Therapeutics' sevasemten met the primary endpoint in its Phase 2 CANYON trial, significantly reducing creatine kinase levels in Becker muscular dystrophy patients.
• The trial also demonstrated stabilization of the North Star Ambulatory Assessment (NSAA) scores in sevasemten-treated patients, indicating a potential for functional stabilization.
• Sevasemten was well-tolerated in both adult and adolescent populations, with no new safety concerns identified during the 12-month treatment period.
• Edgewise plans to engage with the FDA and EMA to discuss marketing authorization strategies and is on track to complete enrollment for the GRAND CANYON pivotal cohort by Q1 2025.
Edgewise Therapeutics Poised for Growth with Novel Muscle Disorder Therapies
• Edgewise Therapeutics is advancing EDG-7500 for HCM, with a readout expected in September, showing potential to reduce titration needs.
• Sevasemten, targeting BMD, has demonstrated encouraging biomarker changes in the DUNE study, estimating a $1 billion market opportunity.
• A comprehensive update on the DMD program is anticipated in H2 2024, with a pivotal trial planned for H1 2025, expanding the pipeline.
• Analysts view Edgewise favorably, citing novel mechanisms and potential for significant market penetration if clinical trials yield positive results.
Cytokinetics' Aficamten Advances in Regulatory Review for Obstructive Hypertrophic Cardiomyopathy
• The EMA has validated Cytokinetics' Marketing Authorization Application (MAA) for aficamten, a cardiac myosin inhibitor, for treating obstructive hypertrophic cardiomyopathy (HCM).
• The FDA has accepted the New Drug Application (NDA) for aficamten with a PDUFA target action date of September 26, 2025, and no advisory committee meeting is planned.
• Aficamten significantly improved exercise capacity and clinical outcomes in the SEQUOIA-HCM Phase 3 trial, supporting regulatory submissions in the U.S., Europe, and China.
DMD Pipeline Shows Promise with Over 75 Therapies in Development
• Over 75 drugs are in development for Duchenne Muscular Dystrophy (DMD), targeting various mechanisms and routes of administration.
• REGENXBIO initiated a Phase I/II trial of RGX-202 in young boys with DMD to assess safety and efficacy.
• Emerging therapies focus on gene modulation, dystrophin replacement, and anti-inflammatory approaches to combat DMD.
• Key companies like Sarepta Therapeutics, Italfarmaco, and FibroGen are advancing DMD therapeutics through clinical trials.
Duchenne Muscular Dystrophy: Biotech Firms Advance Novel Therapies
• Wave Life Sciences reported positive interim results for WVE-N531, an exon-skipping oligonucleotide, showing promising dystrophin expression in patients with Duchenne muscular dystrophy.
• Sarepta Therapeutics expanded the label for its gene therapy Elevidys to include all patients aged four and older with mutations in the dystrophin gene.
• Italfarmaco's Duvyzat (givinostat), the first nonsteroidal treatment for all genetic variants of Duchenne, received FDA approval based on phase 3 trial results.
• Avidity Biosciences' delpacibart zotadirsen (del-zota) demonstrated a significant increase in dystrophin production and reduction of creatine kinase levels in a phase 1/2 trial.
Novo Nordisk's $1bn Oral Weight Loss Drug Shows Promise but Raises Questions in Phase IIa Trial
• Novo Nordisk's monlunabant, an oral cannabinoid receptor 1 inverse agonist acquired for $1bn, demonstrated statistically significant weight loss in a Phase IIa trial but raised concerns about optimal dosing.
• The 10mg dose achieved a 7.1kg weight reduction compared to 0.7kg with placebo, but higher doses showed limited additional benefit while increasing dose-dependent gastrointestinal and neuropsychiatric side effects.
• Investor confidence wavered with Novo Nordisk's stock dropping over 6% following the announcement, as the company plans a Phase IIb trial next year to better determine the optimal efficacy-to-safety ratio.
FDA Cancels Advisory Committee Meeting for Applied Therapeutics' Govorestat, Boosting Approval Prospects
• The FDA has canceled the advisory committee meeting for govorestat, Applied Therapeutics' drug for classic galactosemia, signaling potential approval by the November 28 PDUFA date.
• Analysts are optimistic about govorestat's approval and commercial potential, with Leerink Partners raising the probability of success (PoS) for both galactosemia and SORD deficiency.
• Govorestat, a CNS penetrant aldose reductase inhibitor, targets rare neurological diseases by blocking glucose conversion to sorbitol, with peak sales estimated at $870 million for galactosemia alone.
• Applied Therapeutics' stock price surged following the announcement, reflecting investor confidence in govorestat's prospects and the company's broader pipeline.
Edgewise Therapeutics' EDG-7500 Shows Positive Results in Hypertrophic Cardiomyopathy Trials
• Edgewise Therapeutics announced positive top-line data for EDG-7500 in Phase 1 and Phase 2 trials, demonstrating its potential in treating obstructive hypertrophic cardiomyopathy (HCM).
• The Phase 1 trial showed EDG-7500 was well-tolerated in healthy subjects, with no clinically meaningful changes in left ventricle ejection fraction (LVEF).
• In the Phase 2 CIRRUS-HCM trial, single doses of EDG-7500 led to significant reductions in left ventricular outflow tract (LVOT) gradient without impacting LVEF.
• A 64% mean reduction in NT-proBNP, a key heart failure biomarker, was observed, highlighting EDG-7500's potential in diastolic dysfunction diseases.
ESC Congress 2024: Novel Insights in Heart Failure, Hypertension, and Risk Assessment
• The ESC Congress 2024 highlighted personalized care in atrial fibrillation and innovative hypertension therapies, emphasizing single-pill combinations to improve adherence.
• The FINEARTS-HF trial revealed that finerenone significantly reduces heart failure escalation and hospitalizations, especially when combined with SGLT2 inhibitors.
• Trials on transcatheter mitral valve repair showed it's as effective as surgery, offering a less invasive option for secondary mitral regurgitation patients.
• Research indicates current cardiovascular risk assessments are inadequate, advocating for direct disease evaluation over traditional risk factor surrogates.
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