What one analyst called an “intriguing” overall survival signal in phase III results has Karyopharm Therapeutics Inc. planning to meet with the U.S. FDA about a would-be sNDA filing for Xpovio (selinexor) in myelofibrosis (MF).
In a competitive and still-needy space where Apogee Therapeutics Inc. CEO Michael Henderson noted that “even modestly differentiated products are quickly becoming blockbusters,” his firm’s IL-13 antibody zumilokibart (zumi, APG-777) has turned up satisfying phase II data in part A of the phase II atopic dermatitis (AD) experiment called Apex in moderate to severe disease. “We’re still digesting the data,” Henderson added, pointing out that the results proved better than Apogee expected at both time intervals tested.
Attacking attention deficit hyperactivity disorder (ADHD) from two different angles, thanks to a new asset just brought aboard, should help Collegium Pharmaceutical Inc. protect its revenue stream in the pesky and widespread condition. Stoughton, Mass.-based Collegium disclosed its pact March 19 with Corium Therapeutics Holdings LLC for the former to buy the latter’s Azstarys (serdexmethylphenidate and dexmethylphenidate).
Further weight loss details are yet to come this year, but Eli Lilly and Co. scored positive top-line results in Transcend-T2D-1, a phase III trial evaluating the efficacy and safety of retatrutide, an investigational first-in-class GIP, GLP-1 and glucagon triple hormone receptor agonist, as an adjunct to diet and exercise in type 2 diabetes (T2D).
Awaiting the potential U.S. FDA approval of a second product this year, Protagonist Therapeutics Inc. with partner Johnson & Johnson won the go-ahead for oral peptide Icotyde (icotrokinra), an IL-23 receptor antagonist, to treat moderate to severe plaque psoriasis in adults and pediatric patients 12 and older who weigh at least 40 kg and who are candidates for systemic therapy or phototherapy.
Low expectations for Rhythm Pharmaceuticals Inc.’s phase III study dubbed Emanate with Imcivree (setmelanotide) in a handful of rare genetic obesities meant hardly any share price consequence, and the stock (NASDAQ:RYTM) closed March 17 at $87.68, down $2.83. “We’re not going to file [for approval] on any of these,” CEO David Meeker said, but the latest findings together represent “a solid building block going forward,” he added.
With top-line pivotal data with gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) due in the next quarter, Regenxbio Inc. rolled out positive interim data from the phase I/II Affinity trial at the Muscular Dystrophy Association Clinical and Scientific Conference (MDA) in Orlando, Fla., where Bridgebio Pharma Inc., Capricor Therapeutics Inc., and Solid Biosciences Inc. also had clinical findings to talk about.
The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal to consider PTC Therapeutics Inc.’s Translarna (ataluren) for the treatment of nonsense mutation disease.
Fast on the heels of the U.S. FDA’s go-ahead regarding the expanded use of Wellcovorin (leucovorin) tablets for cerebral folate deficiency in adult and pediatric patients with a confirmed variant in the folate receptor 1 gene – some of whom bear “autistic features” as part of their condition – the American Academy of Pediatrics advised against giving the drug to children with autism spectrum disorder.
As soon as Dianthus Therapeutics Inc. disclosed its early “go” decision on claseprubart in chronic inflammatory demyelinating polyneuropathy (CIDP), Wall Street set about comparing the phase III monoclonal antibody (mAb) to other prospects in the space, with particular focus on Sanofi SA’s riliprubart (SAR-455088).
