Cytokinetics' Aficamten Shows Positive Results in MAPLE-HCM Trial for Hypertrophic Cardiomyopathy
• Aficamten, Cytokinetics' cardiac myosin inhibitor, met its primary endpoint in the Phase 3 MAPLE-HCM trial, demonstrating significant improvement in peak oxygen uptake in obstructive hypertrophic cardiomyopathy patients.
• The drug showed a favorable safety profile with statistically significant improvements across multiple secondary endpoints, including patient-reported outcomes and biomarkers of cardiac wall stress.
• These positive results position aficamten as a potential new treatment option for obstructive hypertrophic cardiomyopathy, a condition with limited therapeutic alternatives currently available.
Biopharma Q1 Updates: New Drug Launches and FDA Extension for Cytokinetics' Aficamten
• Neurocrine Biosciences, Madrigal Pharmaceuticals, and Vanda Therapeutics provided investors with key updates on their recent drug launches during Q1 earnings calls.
• Cytokinetics announced an FDA review extension for aficamten, their investigational cardiac myosin inhibitor, potentially impacting their timeline for market entry.
• The Q1 earnings season highlights significant catalysts across the biopharma sector as companies navigate regulatory pathways and initial commercialization challenges.
Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges
• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions.
• The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success.
• This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.
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.
Trump's Executive Order Delays Medicare Drug Price Negotiations, Sparking Industry and Policy Debate
• President Trump signed an executive order extending the exemption period for small-molecule drugs from Medicare price negotiations by four years, a move criticized by advocacy groups as favoring pharmaceutical industry interests.
• The order aims to address what the administration calls the "pill penalty" - the current policy where small-molecule drugs (90% of medications) face negotiations after 9 years while biologics have longer exemption periods.
• The Medicare Drug Price Negotiation Program, established under the Biden administration, had already achieved price reductions of 38-79% on 10 high-cost drugs with projected savings of $6 billion if applied in 2023.
Cytokinetics to Present New Aficamten Data at ACC 2025: Insights on Metabolism, Combination Therapy, and Long-Term Cardiac Effects
• Cytokinetics will present five studies on aficamten at the upcoming ACC Scientific Session, including new data on its metabolism pathways through multiple CYP enzymes and the safety of combination therapy with disopyramide.
• Long-term treatment with aficamten for up to 72 weeks demonstrated significant improvements in cardiac structure and function in patients with obstructive hypertrophic cardiomyopathy, including reduced left ventricular mass and wall thickness.
• A health economics analysis revealed significant racial disparities in non-obstructive HCM outcomes, with Black patients experiencing higher rates of adverse cardiovascular events and mortality compared to white, Asian, and Hispanic patients.
GSK's Blenrep Secures UK Approval for Multiple Myeloma Treatment in Combination Therapy
• The UK's medicines regulatory body has approved GSK's Blenrep (belantamab mafodotin) in combination with other drugs for multiple myeloma patients whose first treatment failed or caused severe side effects.
• This approval marks a significant comeback for Blenrep, which was withdrawn from markets in 2022 after failing to outperform existing treatments when used as monotherapy.
• Clinical trials demonstrated Blenrep's combination therapy extended progression-free survival and overall survival compared to standard care regimens, including those based on Darzalex (daratumumab).
Biogen Secures $250M from Royalty Pharma to Advance Lupus Antibody into Phase III Trials
• Biogen has entered into a strategic funding agreement with Royalty Pharma, securing $250 million over six quarters to advance litifilimab, a promising lupus treatment, into Phase III development.
• The BDCA2-targeting monoclonal antibody litifilimab showed significant reduction in joint pain for systemic lupus erythematosus patients in Phase II trials, with Phase III data expected between 2026-2027.
• The deal includes milestone payments and mid-single-digit royalties on worldwide sales, coming at a crucial time as Biogen restructures its pipeline and faces various business challenges.
Krystal Biotech and CRISPR Therapeutics Announce Key Updates in Gene Therapy Programs
• Krystal Biotech reported a 473% increase in full-year revenue, driven by VYJUVEK sales, and anticipates European and Japanese regulatory decisions in 2025.
• Krystal Biotech's KB407 for cystic fibrosis received full sanctioning for its Phase 1 CORAL-1 study, with interim molecular data expected mid-2025.
• CRISPR Therapeutics reported positive momentum in the CASGEVY launch, with over 50 activated treatment centers and patients initiating cell collection globally.
• CRISPR Therapeutics expects key clinical data updates in 2025 for CTX112 in oncology and autoimmune diseases, and for CTX310 and CTX320 in cardiovascular programs.
Cytokinetics Halts ALS Drug Trial and Faces FDA Rejection for Heart Failure Treatment
• Cytokinetics stopped the Phase 3 COURAGE-ALS trial of resedemtiv for amyotrophic lateral sclerosis (ALS) after a futility analysis showed no benefit over placebo.
• The FDA rejected Cytokinetics' omecamtiv mecarbil for heart failure, citing insufficient efficacy data, increasing the likelihood that the company will shelve the drug.
• Cytokinetics is now focusing on aficamten, a cardiac myosin inhibitor in Phase 3 development for hypertrophic cardiomyopathy (HCM), with results expected later in 2023.
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.
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