As Bridgebio Pharma Inc. awaits word from the U.S. FDA regarding the small-molecule transthyretin (TTR) stabilizer acoramidis for TTR-mediated amyloidosis cardiomyopathy, the firm closely followed this week’s headline about another spinout with news that the Palo Alto, Calif.-based company has signed a joint research pact with Hitgen Ltd., of Chengdu, China.
Despite the U.S. FDA’s tentative approval of Liquidia Corp.’s Yutrepia (treprostinil) for treating pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease, the company is stuck in the starting gate. Liquidia said it disagrees with the agency’s stance of simultaneously granting regulatory exclusivity in both indications to United Therapeutics Corp.’s powdered formulation of treprostinil, branded Tyvaso, until May 23, 2025. That means full approval for the inhalation powder won’t come until after that date and neither will a Yutrepia launch.
With a move into Lilly Gateway Labs in Boston’s Seaport District, privately held Tevard Biosciences Inc. is ramping up development of its transfer RNA (tRNA)-based therapies to cure everything from Dravet syndrome and other neurological conditions to cardiology indications and muscular dystrophies.
Novo Nordisk A/S presented a mixed bag of R&D results for its cardiometabolic assets in the first half of 2024 as it axed three drug candidates but advanced one to a late-stage study. In the search for greener pastures beyond a crowding obesity market, Novo announced Aug. 7 that it advanced ziltivekimab into a phase III study to assess the subcutaneous therapy’s cardiovascular outcomes in acute myocardial infarction patients.
Draupnir Bio is poised to advance a new approach to targeted protein degradation by engaging the sortilin receptor on lysosomes to promote the destruction of extracellular and membrane-bound disease proteins.
In one of the largest venture rounds for biopharma in 2024, Cardurion Pharmaceuticals Inc. closed a $260 million series B financing, with funds slated to advance and expand its pipeline for potentially first-in-class drugs targeting cardiovascular disease, including two programs in phase II development.
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.
Just seven months after in-licensing ocedurenone (KBP-5074) from KBP Biosciences Pte. Ltd. in a $1.3 billion deal, Novo Nordisk A/S halted its late-stage study after ocedurenone failed to significantly control hypertension in chronic kidney disease patients.
In reviving another case involving drug label carveouts, the U.S. Court of Appeals for the Federal Circuit insisted that its June 25 decision in Amarin Pharma Inc. v. Hikma Pharmaceuticals plc will not kill so-called skinny labels that allow generics to come to market when some of the brand’s indications still have patent protection.
Any skepticism that might have been lingering in the wake of Alnylam Pharmaceuticals Inc.’s February decision to tweak the analysis plan for the Helios-B trial testing vutrisiran in transthyretin amyloidosis with cardiomyopathy appeared to dissipate as the company reported top-line data showing the study met the primary and all secondary endpoints.
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