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.
Adverum Biotechnologies Inc. CEO Laurent Fischer said the firm chose the more prudent route in scrapping development of gene therapy ADVM-022 (AAV.7m8-aflibercept) for diabetic macular edema (DME) as a result of dose-limiting toxicity in the phase II Infinity trial.
Differences between Seres Therapeutics Inc.’s next-generation microbiome therapy and SER-287 were highlighted in the wake of the phase IIb failure with the latter in the study called Eco-Reset, which missed its primary endpoint of improving clinical remission rates in ulcerative colitis (UC) compared to placebo.
Amylyx Pharmaceuticals Inc.’s oversubscribed, $135 million series C financing led by Viking Global Investors will help lay the groundwork for commercializing in Canada and Europe, as well as for a second phase III study in the U.S. with lead candidate AMX-0035 for amyotrophic lateral sclerosis.
Cytokinetics Inc. CEO Robert Blum declined to remark upon what one analyst called “the elephant in the room” as the company popped the lid off positive top-line data from the first and second cohorts of the phase II study with CK-3773274 (known by the shorthand CK-274) in hypertrophic cardiomyopathy (HCM).
A sometimes contentious policy roundtable on how to handle Alzheimer’s disease therapies followed the July 15 meeting of the California Technology Assessment Forum, an independent evidence appraisal committee of the Institute for Clinical and Economic Review (ICER). CTAF’s 15 panelists voted unanimously that the evidence was not adequate to prove Biogen Inc.’s recently approved Aduhelm (aducanumab) superior to supportive care alone.
Members of the California Technology Assessment Forum (CTAF), an independent appraisal committee of the Institute for Clinical and Economic Review (ICER), discussed – and voted on – the safety vs. efficacy and other aspects of Biogen Inc.’s embattled Aduhelm (aducanumab), the amyloid beta-targeting therapy recently approved for Alzheimer’s disease. It didn’t go well for Biogen.
Approval could come next year for Furoscix from Scpharmaceuticals Inc. (Scpharma), a solution of the standard-of-care heart failure diuretic furosemide formulated to a neutral pH and designed for outpatient use.
Prime Medicine Inc. (PM) likely has “a lock on prime editing technology for therapeutic uses,” CEO Keith Gottesdiener told BioWorld, though research labs are continuing to refine the approach. Cambridge, Mass.-based PM has $315 million in the bank that will help advance the platform, which behaves like a DNA word processor to search and replace disease-causing genetic sequences at their exact location in the genome.
