In a deal that could bring Avidity Biosciences Inc. $2.3 billion if all milestones are met, Bristol Myers Squibb Co. gained global rights to Avidity’s antibody oligonucleotide conjugates platform to advance up to five genetic cardiovascular targets. Avidity’s technology, which combines the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, aims to address the root cause of diseases that are untreatable with current RNA therapeutics. Its lead internal programs are based on the targeted delivery of RNA into muscle. Read More
Springworks Therapeutics Inc.’s nirogacestat became the first drug indicated specifically for desmoid tumors, as well as the first gamma-secretase inhibitor to win marketing approval, winning a U.S. FDA nod on the anticipated PDUFA date of Nov. 27. Branded Ogsiveo, nirogacestat has breakthrough therapy, fast track and orphan designations. Read More
Argenx SE’s surprise phase III blowup with subcutaneous Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in the platelet disorder primary immune thrombocytopenia (ITP) – blamed on a higher-than-expected placebo response – had investors speculating about possible read-throughs to other indications. Read More
Acelyrin Inc. said a vendor used by Fortrea Inc., a CRO the company used for its phase IIb/III study testing interleukin-17A inhibitor izokibep in psoriatic arthritis, incorrectly programmed the study’s protocol and created a dose sequencing error, resulting in some patients in two of the four dosing arms to randomly receive placebo and active treatment instead of the intended alternating pattern. The discovery has prompted a review of other studies involving the CRO, including a study that failed to reach statistical significance in September. Read More
Cancer Research UK (CRUK) is worried that rising prices and its total dependence on public donations mean its funding model is becoming unsustainable. The charity is calling on the U.K. government to step in and plug a £1 billion (US$1.23 billion) shortfall it said will open up over the next decade, in order to maintain investment at 2019 levels in real terms. Read More
With drug shortages becoming a fact of life, U.S. President Joe Biden said he plans to issue a presidential determination to broaden the Department of Health and Human Services’ (HHS) authorities under the Defense Production Act to enable investment in the domestic manufacturing of essential medicines, medical countermeasures and other critical inputs that the president deems essential to the national defense. Read More
As expected, Aldeyra Therapeutics Inc. received a complete response letter (CRL) from the U.S. FDA regarding its NDA for reproxalap in dry eye disease, with the agency requiring “at least one” additional study to prove efficacy of the reactive aldehyde species, or RASP, modulator. Read More
Reports of T-cell malignancies, including lymphoma, have the U.S. FDA investigating the risks for patients who received certain autologous CAR T-cell immunotherapies. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: AAX, Accutar, Anaptysbio, Biomarin, Candel, Centessa, DMK, Emergent, Ensysce, Evommune, Fusion, Karuna, Oblique, Oncozenge, Sutro, US Worldmeds, Vaxcyte, Vivus. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Abbvie, Abeona, Arcturus, Everest, Genelux, Genfleet, Gri, Gracell, Inmune, Innovent, Krystal, Mesoblast, Neumora, Outlook, Priothera, Redhill, Sernova, TC, Valneva, Vertex. Read More
