Aficamten

Cytokinetics updates on cardiac myosin modulation programs, including NDA submission for aficamten to FDA in Q3, and plans to begin COMET-HF and AMBER-HFpEF trials in Q4. Global commercial launch preparations for aficamten underway, with focus on specialty cardiology franchise.

Cytokinetics launches 'HCM Beyond the Heart' campaign to educate HCPs on hypertrophic cardiomyopathy's personal burdens, featuring patient stories. The campaign aims to support holistic patient care beyond pharmacotherapeutic treatment, with aficamten expected to be FDA-approved and launched by 2025. The campaign's formal launch is rescheduled for the American Heart Association convention in November.

September 2024 saw significant developments in cardiology, including the ESC Congress 2024 and FDA approvals like plozasiran for familial chylomicronemia syndrome. Edgewise Therapeutics reported positive data for EDG-7500 in hypertrophic cardiomyopathy. ESC Congress 2024 featured 4,400 abstracts and 32,000 attendees, highlighting new data on finerenone, milvexian, and aficamten. The Family Heart Global Summit in Dallas also provided insights on lipoprotein(a) therapies. The APAC Recap series, a collaboration with the Academy of Physician Associates in Cardiology, shared key takeaways from medical meetings.

Novo Nordisk shares fell 6% after mixed Phase 2 weight-loss drug results; Sanofi presented data supporting tolebrutinib for MS; Lasker Awards given to scientists for GLP-1 weight-loss drug research; Edgewise Therapeutics' heart failure drug shows promising early results; FDA declines to approve Vanda Pharmaceuticals' gastroparesis drug; House Energy and Commerce Committee supports extending pediatric rare disease voucher program.

Aficamten improved exercise capacity by changing cardiac function in a study led by Mass General Brigham researchers, published in JAMA Cardiology.

Aficamten, a cardiac myosin inhibitor, improved exercise tolerance in patients with hypertrophic obstructive cardiomyopathy in the SEQUIOA-HCM trial, enhancing peak oxygen uptake and cardiac function, as published in JAMA Cardiology.

Aficamten treatment improves exercise performance in obstructive HCM, significantly enhancing multiple exercise measures in a 24-week trial, with correlations to clinical responses.

New data on aficamten's safety and long-term use presented at the European Society of Cardiology Congress 2024, showing it well-tolerated with a profile similar to placebo. Analysis from FOREST-HCM demonstrates successful withdrawal of standard of care medications in patients with obstructive HCM treated with aficamten. Company to host conference call and webcast on September 3rd.

Additional data from SEQUOIA-HCM trial show aficamten improves cardiac remodeling, structure, function, symptoms, and biomarkers in patients with obstructive hypertrophic cardiomyopathy (HCM). Presentations at the European Society of Cardiology Congress 2024 were accompanied by four simultaneous journal publications. Cytokinetics to host a conference call on September 3rd to discuss these findings.

The biotech industry, valued at $1.2 trillion, faces challenges post-pandemic with reduced revenues, regulatory pressures, and decreased FDA approvals. Funding has significantly dropped, impacting innovation. In 2023, biotech stocks like Jazz Pharmaceuticals, Incyte Corporation, and Ultragenyx Pharmaceutical are among the worst performers, with significant YTD share price declines.