After two years of talks, privately held Ventus Therapeutics Inc. and Novo Nordisk A/S have signed an exclusive worldwide license deal to commercialize candidates from Ventus’ portfolio of peripherally restricted NLR pyrin domain-containing 3 (NLRP3) inhibitors. Along with Roche Holding AG’s September 2020 acquisition of Inflazome Ltd. and Novartis AG’s buyout of subsidiary IFM Tre in April 2019, this is another step by big players to get into the NLRP3 space.
A serious adverse event (SAE) in one participant has led the U.S. FDA to place a partial clinical hold on Avidity Biosciences Inc.’s lead program. The action is centered on the phase I/II Marina study of AOC-1001, an antibody oligonucleotide conjugate for treating myotonic dystrophy type 1, the most common form of muscular dystrophy in adults.
The race to become the first microbiome-based therapy to reach the market continues between two companies. A positive view by the U.S. FDA’s Vaccines and Related Biological Products Advisory Committee on Sept. 22 for RBX-2660 in preventing recurrent Clostridium difficile infection kept Ferring Pharmaceuticals Inc.’s microbiota-based live biotherapeutic moving forward.
Emphasizing April’s vote of concern about the safety of PI3K inhibitors, the U.S. FDA’s Oncologic Drugs Advisory Committee said privately held Secura Bio Inc.’s Copiktra (duvelisib) is more of a hindrance than a help. The adcom voted 8-4 on Sept. 23 that it sees a detriment in overall survival (OS) and some other safety issues that were revealed in newly updated data for the cancer treatment. Approved in 2018 for treating adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma after at least two prior therapies, Secura argued that its data continue to show a statistically significant and clinically meaningful benefit in OS and also in progression-free survival.
It took resolving issues from two complete response letters for Fennec Pharmaceuticals Inc. to finally land U.S. FDA approval of Pedmark. The injectable formulation of sodium thiosulfate is for reducing the risk of platinum-induced ototoxicity associated with cisplatin in pediatric patients 1 month and older with localized, non-metastatic solid tumors. Approval of the NDA arrived Sept. 20, three days before its Sept. 23 PDUFA date.
Virios Therapeutics Inc. said most likely COVID-19 had a hand in the phase IIb failure of IMC-1 (famciclovir + celecoxib), a dual COX-2/COX-1 inhibitor for treating fibromyalgia. The drug failed to hit statistical significance in dampening pain severity when compared to placebo (p=0.302).
What was once effective is now a non-starter. Newly updated guidelines from the World Health Organization (WHO) caution against using the COVID-19 treatments sotrovimab, from GSK plc and Vir Biotechnology Inc., and Regen-Cov (casirivimab + imdevimab), from Regeneron Pharmaceuticals Inc. Omicron, the group said, has rendered the monoclonal antibodies ineffective.
With four complete response letters behind it and seven months ahead of its April 2023 PDUFA date, Mallinckrodt plc’s Terlivaz (terlipressin) has been approved by the U.S. FDA for treating hepatorenal syndrome. Terlivaz had a decade-long series of obstacles before getting the long-awaited approval. The vasopressin analogue selective for V1 receptors was approved, in part, on results from the phase III CONFIRM trial of 300 patients, which met its primary endpoint of renal function improvement, avoidance of dialysis and short-term survival (p=0.012).
Rubius Therapeutics Inc. is taking a giant step back so that it can attempt taking a few steps forward. The company said it’s restructuring to focus on taking its Rubius Erythrocyte Design (RED) cell therapeutics platform from what it considers a good thing and turn it into a better one. Rubius engineers red blood cells to become cancer fighters but a lack of progress and the lure of similar but new technology could lead to bigger and better results.
Success in two pivotal studies of high dose Eylea (aflibercept) has significantly strengthened Regeneron Pharmaceuticals Inc.’s market position. But the company will face stiff competition from biosimilars and Roche Holding Group’s recently approved Vabysmo. After struggling for much of the year, the Tarrytown, N.Y.-based company’s stock (NASDAQ: REGN) rose about 20% this week. It closed 2% upward on Sept. 9 at $724.32 per share.
