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.

In Regeneron Pharmaceuticals Inc.’s phase I/II Chord study of 12 children with genetic hearing loss, 10 out of 11 have shown improvements after being treated with a gene therapy. “What is really remarkable about this type of therapeutic approach is that the first people who are going to see the impact are not actually the physicians – it’s the families,” Jonathon Whitton, vice president and Regeneron’s auditory global program head, told BioWorld.

Pepgen Inc. seems to have gained a leg up on competitors in early data with PGN-EDODM1 in myotonic dystrophy type 1 (DM1), and shares of the Boston-based firm (NASDAQ:PEPG) closed Feb. 24 at $2.29, up 92 cents, or about 67%. The company unveiled initial positive data from the 5- and 10-mg/kg dose cohorts in the ongoing Freedom-DM1 phase I study with PGN-EDODM1, which deploys Boston-based Pepgen’s Enhanced Delivery Oligonucleotide technology to deliver a therapeutic oligonucleotide that is designed to restore the normal function of MBNL1, a key RNA splicing protein.

Several Asia biotechs this week – including Innocare Pharma Ltd., Akeso Pharmaceuticals Inc., Sanbio Co. Ltd. and Ascletis Pharma Inc. – unveiled the start of new late-stage clinical trials or interim findings from early stage studies.

In response to the news of reported layoffs at the U.S. FDA over the weekend, Advanced Medical Association president and CEO Scott Whitaker said in press release that these “significant job cuts could have a very negative impact on patient care in this country.”

Radiance Biopharma Inc. bought its way into the ROR1 antibody-drug conjugate (ADC) space through a potential $1 billion-plus licensing deal, including a $15 million up-front payment, with CSPC Megalith Biopharmaceutical Co. Ltd. for rights to RB-164 (SYS-6005) in the U.S. and select countries.

The longstanding ambition of developing an inhaled gene therapy for cystic fibrosis has taken a step forward, with the start of a phase I/II trial of a product using a novel pseudotyped viral vector that it is hoped will circumvent problems encountered in previous studies with other vectors.

The phase IIb study results of Supernus Pharmaceuticals Inc.’s SPN-820 in treatment-resistant depression had pulled the company’s stock (NASDAQ:SUPN) down 15.6% on Feb. 19. The randomized, double-blind, placebo-controlled study of adults failed to show a statistically significant improvement on the primary endpoint of change from baseline on the Montgomery-Åsberg Depression rating scale. Shares closed at $33.52 apiece on Feb. 19.