Wall Street immediately set to weighing the long-term import of Cytokinetics Inc.’s regulatory delay with aficamten, but the short-term impact was evident, as shares (NASDAQ:CYTK) dipped to close May 2 at $37.35, a loss of $5.57 or 13%.
Lexeo Therapeutics Inc. produced more positive interim data from early stage studies of its gene therapy to treat Friedreich’s ataxia cardiomyopathy. The results have prompted the company to continue their ongoing dialogue with U.S. FDA regulators to finalize a registrational study protocol and launch that study by early 2026 with data the following year.
Top-line data from the second and third parts of Edgewise Therapeutics Inc.’s four-part phase II Cirrus-HCM study in hypertrophic cardiomyopathy (HCM) has produced more positive data. There was however less enthusiasm for the results than there had been in September for the first batch of data.
Affinia Therapeutics Inc. has nominated AFTX-201 as a development candidate for the treatment of BAG3 dilated cardiomyopathy. The gene therapy, using Affinia’s cardiotropic capsid, is given as a one-time intravenous injection.
Avidity Biosciences Inc. has announced two new precision cardiology development candidates targeting rare genetic cardiomyopathies. AOC-1086 targets phospholamban (PLN) cardiomyopathy and AOC-1072 targets protein kinase AMP-activated noncatalytic subunit γ2 (PRKAG2) syndrome.
The PLN-R14del mutation is often tied to dilated or arrhythmogenic cardiomyopathy, which can progress to end-stage heart failure. Cardiac phospholamban (PLN) plays a crucial role in calcium modulation in cardiomyocytes, which is disrupted by the PLN-R14del mutation.
Researchers from Affinia Therapeutics Inc. have described the development and preclinical evaluation of a new AAV-based gene therapy, designed using a novel cardiotropic capsid, for the potential treatment of MYBPC3-associated hypertrophic cardiomyopathy (HCM).
Potential implications for others in the space, including Pfizer Inc. and Bridgebio Pharma Inc., became the subject of buzz after Alnylam Pharmaceuticals Inc. provided details from the Helios-B phase III study with RNAi candidate vutrisiran, in the works for ATTR amyloidosis with cardiomyopathy.
Interim data from two early stage Friedreich’s ataxia (FA) cardiomyopathy studies from Lexeo Therapeutics Inc. hit the mark by reducing heart muscle thickness, a key cause of death among patients with the rare disease. The results came from the Sunrise-FA phase I/II study and an investigator-initiated phase Ia study of LX-2006, an adeno-associated virus-mediated gene therapy encoding the human frataxin gene. The drug is designed to improve frataxin protein expression to improve mitochondrial cell function.
Stealth Biotherapeutics Inc. has presented new data demonstrating cardioprotective effects of SBT-589 in preclinical models of Friedreich’s ataxia (FA). SBT-589 acts on mitochondrial pathways essential for cellular health and energy production that are impaired in FA cardiomyopathy.
