Belharra Therapeutics Inc., an early stage firm which is pioneering a new approach to chemoproteomics, has already found an influential backer for the distinctive way it systematically probes the interactions between small-molecule ligands and the proteins to which they bind. The Genentech arm of Roche Holding AG is paying $80 million up front – and could hand over more than $2 billion in milestones – for a broadly based small-molecule drug discovery pact focused across multiple therapeutic areas, including oncology, immuno-oncology, autoimmune diseases and neurodegenerative diseases.
Through Lilly, Capsida prevails in a $740M deal
Eli Lilly and Co., through its wholly owned subsidiary Prevail Therapeutics Inc., is delving more deeply into gene therapies. In a new collaboration, Prevail will use privately held Capsida Biotherapeutics Inc.’s adeno-associated virus engineering platform, coupling the tech with Prevail’s gene therapies to target the causes of CNS diseases. Capsida is getting $55 million up front and the chance to bring in another $685 million in R&D and commercial milestones plus tiered royalties. Lilly bought Prevail in December 2020 for $880 million up front plus and an earn-out of up to $160 million if Lilly can gain regulatory approval for at least one of Prevail's drugs.
Vera hits phase IIb primary endpoint with atacicept in IgAN, but shares sink
Shares of Vera Therapeutics Inc. (NASDAQ:VERA) fell $11.13, or 60%, to trade midday at $7.17 on word of data from the phase IIb Origin trial with atacicept in patients with immunoglobulin A nephropathy (IgAN). The primary endpoint analysis – change in proteinuria as evaluated by urine protein creatinine ratio at week 24 of the pooled 75/150-mg dose groups – found that atacicept reached statistical significance, showing a 31% mean reduction vs. baseline (p=0.037 vs. placebo). Safety proved satisfactory, too, but Wall Street apparently expected better. Hopes were high in IgAN for the dual mechanism of atacicept, a fusion protein that blocks the cytokines APRIL and BlyS.
Geron rises on phase III MDS data, with US, EU filings ahead for imetelstat
Geron Corp. plans to submit for regulatory approval in the U.S. and Europe later this year following a positive readout from the phase III Imerge trial testing telomerase inhibitor imetelstat in patients with lower-risk myelodysplastic syndromes (MDS). Results met the primary and key secondary endpoints of transfusion-independence at eight and 24 weeks, respectively, across MDS subtypes, with no new safety signals observed. Shares of Geron (NASDAQ:GERN) were up 36%, or 88 cents, at midday.
Phathom down on FDA delay for vonoprazan
Phathom Pharmaceuticals Inc.’s shares sank 32% on news that the U.S. FDA will not take action on the company’s NDA for oral small-molecule potassium-competitive acid blocker vonoprazan by the Jan. 11 PDUFA date. The drug was under review for erosive esophagitis (EE), but on Aug. 2, 2022, Florham Park, N.J.-based Phathom revealed that it detected trace levels of a nitrosamine impurity, N-nitroso-vonoprazan (NVP), in commercial batches. While an acceptable daily intake limit for NVP was established by the FDA at 96 ng/day, the agency wants more stability data verifying the levels remain below the limit over the product’s shelf life.
Aiming for consistency, US FDA finalizes REMS guidance
After more than five years and extensive feedback, the U.S. FDA is finalizing its guidance on the format and content of risk evaluation and mitigation strategy (REMS) plans for prescription drugs and biologics associated with serious risks. Released Jan. 4, the final guidance is the FDA’s first for the new year. It can be used to draft a REMS for a single product or a shared system, and it includes recommendations for drafting a bifurcated REMS for when generics are launched.
Shionogi expands reach of COVID-19 antiviral, Xocova, to Korea and China
After receiving approval in Japan for its orally administered COVID-19 antiviral, 3CL protease inhibitor Xocova (ensitrelvir/S-217622) in late November, Shionogi & Co. Ltd. is gearing up for approvals in South Korea and China. The company’s South Korean partner, Ildong Pharmaceutical Co Ltd, which in-licensed the drug from Shionogi, has submitted a new drug application to South Korea’s Ministry of Food and Drug Safety and the application was accepted for review.
Also in the news
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