In Regeneron Pharmaceuticals Inc.’s phase I/II Chord study of 12 children with genetic hearing loss tied to the variants of the otoferlin gene, 10 out of 11 have showed notable improvements in their hearing. At 48 weeks after receiving Regeneron’s DB-OTO gene therapy, the hearing of the first treated patient had reached near normal. The data also showed from week 48 to week 72, the same child could identify words like mommy, cookies and airplane when they were spoken. Decibel Therapeutics Inc. agreed to be acquired in August 2023 by Regeneron in a deal valued at up to $213 million, including $109 million in equity, with additional payments via contingent value rights linked to clinical and regulatory milestones for DB-OTO, which was Decibel’s lead gene therapy program.

Bridgene’s signs $770M Takeda immunology, neurology deal 

Nearly four years after signing its first major partnership with Takeda Pharmaceutical Co. Ltd., Bridgene Biosciences Inc. returned for a second deal with Takeda – this time focused on using its chemoproteomics platform to discover novel small molecules against immunology and neurology targets. The earlier deal from March 2021, worth about $500 million in milestone payments, aimed to find and develop small molecules for neurodegenerative disorders. The newest deal involves $46 million in up-front and potential preclinical milestone payments, as well as clinical and commercial milestone payments bringing the total deal value to $770 million, plus tiered royalties. Bridgene’s platform, IMTAC – which stands for Isobaric Mass Tagged Affinity Characterization – identifies and measures proteins in live cells and tissues, with the goal of finding first-in-class small-molecule drugs against previously undruggable targets.

Qyuns’ QX-002N hits endpoints in ankylosing spondylitis trial  

Qyuns Therapeutics Co. Ltd.’s monoclonal antibody targeting IL-17A, QX-002N, met both primary and secondary endpoints in a phase III trial in ankylosing spondylitis (AS). Conducted at Peking Union Medical College Hospital, the multicenter, randomized, double-blinded, placebo-controlled study enrolled 641 patients with moderate to severe active AS. For the primary efficacy endpoint of Assessment of Spondyloarthritis International Society (ASAS)40 response rate at week 16, 40.4% of patients in the treatment group responded compared to 18.9% in the placebo group (p<0.0001). For the key secondary endpoint of ASAS20 response rate, the QX-002N group showed a 65.2% response at week 16 compared to 41.3% in the placebo group (p<0.0001).

Kyorin drafts deals for chronic cough DTx, loss of smell therapy

Kyorin Pharmaceutical Co. Ltd. struck two licensing deals recently, including one with Hyfe Inc. Feb. 25 to develop the world’s potential first prescription digital therapeutic (DTx) for chronic cough in Japan. Just a week prior to Hyfe, Kyorin announced an option and licensing deal with Cyrano Therapeutics Inc. for domestic rights to develop and commercialize Cyrano’s post-viral hyposmia, or loss of smell, therapy called CYR-064.

Results soon from Mineralys as others busy in aldosterone space

Mineralys Therapeutics Inc. should have data shortly with lorundrostat for uncontrolled or resistant hypertension, a compound that may have utility, too, in obstructive sleep apnea. The oral drug takes aim at dysregulated mineralcorticoid aldosterone as a selective aldosterone synthase inhibitor. Others in the mineralcorticoid/aldosterone space include Novo Nordisk A/S and Boehringer Ingelheim GmbH.

New admin toes the Biden line on Rx price negotiations

The Trump administration dashed hopes that it would temper the Medicare price negotiations mandated by the Inflation Reduction Act when it filed a brief last week in response to Novartis AG’s appeal to the U.S. Court of Appeals for the Third Circuit. Submitted by the Departments of Justice and Health and Human Services, the brief adheres to the position the Biden administration held in defending the negotiations, claiming that Novartis’ constitutional challenges are meritless. Meanwhile, the Third Circuit is scheduled to hear oral argument in April in a similar appeal brought by Novo Nordisk A/S.

BioWorld Insider Podcast: Mega money well spent? Mammoth mergers fall short of the dream

Billion-dollar M&As are commonplace now, but not too long ago they were a rarity. So many have occurred in the past few years, they’ve become the norm. But were all these multi-billion-dollar mega mergers worth the money? Karen Carey, BioWorld’s senior managing editor and chief analyst, crunched the numbers on 21 of the biggest M&As in a three-part BioWorld series and found very few have been, so far, good deals for the buyer. In this podcast, Carey sifts through the winners and losers and explains the analysis.

Also in the news

Alkeus, Altimmune, Arcutis, Auron, Axsome, Biocryst, Bristol Myers Squibb, Celltrion, Cytodyn, Ensho, Gilead, Glaukos, GSK, Idrx, Indivior, Innovent, Insmed, Invivyd, Johnson & Johnson, Ltz, Novo Nordisk, OSE, OS Therapies, Ottimo, Pepgen, Pfizer, Redx, Sanofi, Senti, Summit, Takeda, Terns, Teva