Setting the stage for upcoming U.S. FDA decisions, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. scored conditional marketing approval from the U.K.’s Medicines and Healthcare products Regulatory Agency for Casgevy (exagamglogene autotemcel [better known as exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). It’s the first go-ahead for a CRISPR-based therapy anywhere in the world. The PDUFA date for the compound is Dec. 8 for SCD and March 30, 2024, for TDT.
Augtyro US FDA-approved for ROS1+ non-small-cell lung cancer
More than a week earlier than its PDUFA date, Bristol Myers Squibb Co. (BMS) received U.S. FDA approval of its next-generation ROS1 tyrosine kinase inhibitor repotrectinib for adults with ROS1-positive locally advanced or metastatic non-small-cell lung cancer. The approval validates the BMS acquisition of Turning Point Therapeutics Inc. for $4.1 billion in 2022. At the time of the proposed buyout, SVB Leerink analyst Andrew Berens said the market potential was about $1.4 billion as a first-line treatment and about $455 million as a second-line treatment. Branded Augtyro, the drug, also formerly known as TPX-0005 and BMS-986472, is expected to be available to patients before the end of 2023.
Withdrawal off the ODAC table for outlier lymphoma drugs
Changes in ownership are no excuse for delays in the completion of confirmatory trials for drugs granted accelerated approval, U.S. FDA officials told the Oncologic Drugs Advisory Committee (ODAC) today. They explained that in acquiring a drug with accelerated approval, a company also is acquiring the postmarket requirements (PMRs) the former owner agreed to. Ownership change is one of the reasons Acrotech Biopharma Inc. gave for the years-long delay in completing confirmatory trials for the two peripheral T-cell lymphoma drugs, Folotyn (pralatrexate) and Beleodaq (belinostat), it acquired in 2019 from Spectrum Pharmaceuticals Inc. But other than removing a drug from the market, the FDA has no tools to hold sponsors to the agreed-upon PMR timetables. Although Folotyn and Beleodaq are outliers in terms of delayed confirmatory trials, the FDA made it clear that today’s meeting was not about whether the drugs should remain on the market. Rather, the ODAC is being asked to discuss approaches to confirm the clinical benefit of the drugs.
Pyramid returns MSP1 inhibitor to Voronoi, ending $846M deal
South Korean pharmaceutical Voronoi Inc. said that New Jersey-based Pyramid Biosciences Inc. terminated its previous $846 million deal for Voronoi’s solid and breast cancer drug candidate, VRN-08, in a letter dated Oct. 24, 2023. To BioWorld, Voronoi noted that the deal termination could be related to Pyramid shifting its strategic focus to antibody-drug conjugate (ADC) drug development, including Pyramid’s flagship trophoblast cell surface antigen 2-targeting ADC called PBI-410.
Etcembly aims at cancer with AI-driven, rapid TCR engineering technology
Etcembly emerged from stealth mode in August with something it regards as seemingly impossible: A machine learning platform that has the ability to predict and engineer – at never-seen-before scale and speed – T-cell receptors (TCR) that enable bispecific T-cell engager (BiTE) antibodies targeting cancer cells to be produced. The artificial intelligence (AI) engine, Emly, scans hundreds of millions of patient-derived TCR sequences to create T cell-directed immunotherapies in a way that has enabled its own lead, a BiTE for triple negative breast cancer, to demonstrate single digit picomolar affinity – a millionfold higher affinity than the native receptor, according to the firm – with no apparent cross-reactivity.
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