Pfizer Inc.’s voluntary recall of batches of Chantix (varenicline) in mid-July called attention to the smoking-cessation market, where the nicotinic acetylcholine receptor agonist holds lead position, with Glaxosmithkline plc’s Zyban (bupropion) – another oral prescription drug – also a contender. In line to potentially shake up the space with cytisinicline, a plant-based alkaloid, is Achieve Life Sciences Inc.
Fulcrum Therapeutics Inc. shares (NASDAQ:FULC) closed at $18.77, up $10.44 or 125%, on word of positive interim results from a phase I trial in healthy adult volunteers with oral FTX-6058 for sickle cell disease (SCD). The firm has “already achieved maximal target engagement [MTE] at all three doses,” said Christopher Morabito, the company’s chief medical officer. “I don’t think we’ll exceed that.”
The new case of myelodysplastic syndrome (MDS) that overshadowed Bluebird Bio Inc.’s earnings and resulted in a clinical hold by the FDA will be addressed similarly to an earlier hitch in the sickle cell disease program, said Andrew Obenshain, the company’s head of severe genetic diseases. “Essentially, we will try and do the same thing,” and exonerate the lentiviral vector, he said during a conference call with investors. “At this point, we don't have tumor cells or leukemia to analyze,” he noted.
While Aprea Therapeutics Inc.’s data disclosed July 21 from the phase II trial with eprenetapopt – also known as APR-246, a reactivator of mutant tumor suppressor protein p53 – plus azacitidine (AZA) whetted interest in taking aim at the “guardian of the genome,” the company’s fortune took an unexpected turn when the FDA smacked a partial clinical hold on work with the duo.
A little over a month after Versant Ventures-back Vividion Therapeutics Inc. filed to raise $100 million in an IPO, Bayer AG is paying $1.5 billion up front, with the promise of $500 million more in milestone payments, to take over the firm and its small-molecule precision oncology and immunology platform.
Publication this summer of phase II data with Spruce Biosciences Inc.’s CRF1 antagonist, tildacerfont, for classic congenital adrenal hyperplasia (CAH) in the Journal of Clinical Endocrinology and Metabolism (JCEM) highlighted the indication where a handful of players are known to be active.
Outlook Therapeutics Inc. CEO Russell Trenary said positive results from the phase III study called Norse Two represent “the final step we need” to proceed with the BLA in the first quarter of next year for an ophthalmic formulation of the VEGF binder bevacizumab to treat wet age-related macular degeneration (AMD).
Gene therapy’s one-and-done mindset proved “a boon and a bane,” said Avak Kahvejian, general partner at Flagship Pioneering. “You have one shot to get it right, is what that really means,” and his firm founded Ring Therapeutics Inc. to design redosable drugs in the space.
As amyotrophic lateral sclerosis continues to make headlines, candidates bearing varied approaches proliferate and the indication likely allows for multiple players, given the prospect of a combo regimen.
