Advances lately in the genome-editing space include Beam Therapeutics Inc. publication in The CRISPR Journal details of its work with inlaid base editors, which the firm is applying in the BEAM-102 program for sickle cell disease. IBEs’ predictable, shifted editing window lets researchers go after disease-causing mutations that canonical base editors cannot reach, Beam said, and do the job with high efficiency and few off-target effects on the genome. The hottest news due in the near-term future from the sector will spill from Intellia Therapeutics Inc., of Cambridge, Mass., which is due to roll out first-in-human data with a systemic CRISPR-based genome editing therapy, NTLA-2001, in hereditary transthyretin amyloidosis.
Casualties continue to accrue in Huntington’s disease, but drug developers continue their work in the challenging, fatal genetic disorder that afflicts an estimated 2.71 per 100,000 people globally. In March, Basel, Switzerland-based Roche Holding AG pulled the plug on its phase III Generation HD1 study with the antisense therapy tominersen, licensed from Ionis Pharmaceuticals Inc., of Carlsbad, Calif., in a tie-up that dates back to the spring of 2013. Roche subsidiary Genentech Inc. said the move was based on an independent data monitoring committee's preplanned look at the drug's risk-benefit profile.
With phase III data due from Phathom Pharmaceuticals Inc. in the near term, investor eyes are turning to the ways that lead compound vonoprazan, a potassium-competitive acid blocker, might distinguish itself from proton pump inhibitors (PPIs) in gastroesophageal reflux disease (GERD).
Adverse events and criteria for determining remission in an otherwise positive early study by Syndax Pharmaceuticals Inc. with oral menin inhibitor SNDX-5613 apparently gave Wall Street pause, and shares (NASDAQ:SNDX) closed at $13.42, down $5.96, or 31%.
At the recent American Association for Cancer Research (AACR) meeting, Blueprint Medicines Corp. unveiled results from the registrational phase II Pathfinder study with Ayvakit (avapritinib) in systemic mastocytosis (SM), adding more promise to the KIT inhibitor class.
Heidelberg, Germany-based Affimed NV described its progress at the American Association for Cancer Research meeting and discussed the research in a conference call on 2020 financial results, adding fuel to investor enthusiasm for the firm’s natural killer (NK) cell approach, although the update did not come without some confusion.
With phase III data due from Phathom Pharmaceuticals Inc. in the near term, investor eyes are turning to the ways that lead compound vonoprazan, a potassium-competitive acid blocker, might distinguish itself from proton pump inhibitors (PPIs) in gastroesophageal reflux disease (GERD).
Plx Pharma Inc. is not letting out the details just yet about next quarter’s launch of liquid-filled aspirin capsule Vazalore, recently cleared by the FDA in 81-mg and 325-mg doses, but CEO Natasha Giordano said cardiologists are “essential to our strategy. We have developed very deep sales plans [that are] laser-focused.”
Companion diagnostics-focused Celcuity Inc. CEO Brian Sullivan said the deal with Pfizer Inc. for rights to pan-PI3K/mTOR inhibitor gedatolisib was “an organically developed opportunity, because of the research we had done on gedatolisib” in the course of investigating PI3K inhibitors. “We hadn’t shifted our strategy and said, ‘Oh, let’s start in-licensing drugs.’”
Cambridge, Mass.-based Agios Therapeutics Inc.’s encouraging phase III data from a pair of trials with allosteric activator mitapivat in pyruvate kinase deficiency brought more attention to the space, where Rocket Pharmaceuticals Inc. – at a much earlier stage – is trying a gene therapy called RP-L301.
