Avidity, BMS sign $2.3B deal for cardiovascular disease

In a deal that could bring Avidity Biosciences Inc. $2.3 billion if all milestones are met, Bristol Myers Squibb Co. gained global licensing and research rights to Avidity’s antibody oligonucleotide conjugates platform to advance up to five cardiovascular targets. Avidity’s technology, which combines monoclonal antibodies with oligonucleotide therapies, aims to address diseases that are untreatable with RNA therapeutics. The deal includes $100 million up front, a potential $1.35 billion in R&D milestone payments, and up to about $825 million in commercial milestone payments, as well as tiered royalties up to low double-digits. This is an expansion to Avidity’s 2021 partnership with BMS’s wholly owned subsidiary, Myokardia Inc.

Springworks wins first FDA nod for gamma-secretase inhibitor in desmoid tumors

Springworks Therapeutics Inc.’s nirogacestat became the first drug indicated specifically for desmoid tumors, following U.S. FDA approval on the anticipated PDUFA date of Nov. 27. Branded Ogsiveo, nirogacestat has breakthrough therapy, fast track and orphan designations. It was cleared for use in adults with progressing desmoid tumors who require systemic treatment and comes with a broad label that Springworks CEO Saqib Islam said sets up the therapy to become a “practice-changing medicine.” Shares of Springworks (NASDAQ:SWTX) were trading up 25% at midday.

Argenx sinks on phase III miss with Vyvgart Hytrulo in ITP

Argenx SE disclosed top-line results from the Advance-SC phase III study evaluating Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in adults with primary immune thrombocytopenia (ITP). The study fell short of the primary endpoint of a sustained platelet count response in chronic ITP patients. Shares of the firm (NASDAQ:ARGX) were trading midday at $438.30, down $57.47, or 11%. The drug is a neonatal Fc receptor blocker and endoglycosidase combo cleared by the U.S. FDA for generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive.

Acelyrin: CRO’s vendor incorrectly programmed study protocol

Acelyrin Inc. said a vendor used by Fortrea Inc., a CRO the company used in one of its clinical trials, incorrectly programmed a study’s protocol and created a dose sequencing error. The phase IIb/III study of the company’s interleukin-17A inhibitor, izokibep, for treating psoriatic arthritis is ongoing. Acelyrin said the sequencing error caused some patients in one of the dosing arms to randomly receive placebo and active treatment instead of the intended alternating pattern. The company said there is no risk to patient safety, and the dosing sequence has been corrected. Acelyrin stock (NASDAQ:SLRN) was suffering at midday, as the share price had dropped 30% to about $6 each.

CRUK warns UK government of shortfall in funding for cancer research

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.

Biden to expand HHS authorities to address US drug shortages

With drug shortages becoming a fact of life, U.S. President Joe Biden said yesterday 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. Meanwhile, administration officials met with manufacturers of RSV vaccines and prophylactics to underscore the urgency needed to meet the demand for the winter season and to prepare for next year.

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