It’s just a discussion next week of Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG’s sickle cell disease (SCD) therapy, with no vote expected from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. The FDA isn’t questioning the drug’s efficacy as much as it is concerned about the effects of off-target editing.
ABVC Biopharma Inc. entered a multiyear, global licensing agreement for its CNS drugs to treat major depressive disorder and attention deficit hyperactivity disorder. The license deal with Aibtl Biopharma Inc. would cover clinical trial, registration, manufacturing, supply and distribution rights. The products were valued at $667 million by a third party, according to ABVC.
Four other companies want to replicate Biomarin Pharmaceuticals Inc.’s recent success as they seek approvals to treat the rare but most prevalent genetic form of dwarfism, achondroplasia. The U.S. FDA approved the sNDA for injectable Voxzogo (vosoritide), fibroblast growth factor receptor 3 (FGFR3) inhibitor from Biomarin on Oct. 20.
Novartis AG’s radioligand therapy, Pluvicto, a bestseller for the company that has been restructuring itself, continues to show strong growth in the third quarter of 2023, according to its most recent financial results. Sales were up 217% from the same quarter in 2022 at $256 million. Analysts are predicting blockbuster sales. Adding to the momentum were new data from the phase III PSMAfore study of Pluvicto (lutetium [177Lu] vipivotide tetraxetan), in second-line cancer, hitting its primary endpoint of radiographic progression-free survival in treating prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer after treatment with androgen receptor pathway inhibitor therapy.
A new deal between privately held Hummingbird Bioscience Pte. Ltd. and Endeavor Biomedicines Inc. is just one of three antibody-drug conjugate (ADC) agreements reached in the past week, marking a fourth-quarter surge for the therapy.
For a whopping $7.1 billion up front, Roche AG is buying Telavant Holdings Inc., a firm that is owned by Roivant Sciences Ltd. and Pfizer Inc. The deal also includes $150 million on the back end, and Roche gains rights to develop, manufacture and commercialize a fully human monoclonal antibody targeting tumor necrosis factor-like ligand 1A, RVT-3101, for treating inflammatory bowel disease (IBD) and possibly other diseases in the U.S. and Japan. Pfizer retains the rights for the rest of the world.
A new deal between privately held Hummingbird Bioscience Pte. Ltd. and Endeavor Biomedicines Inc. is just one of three antibody-drug conjugate (ADC) agreements reached in the past week, marking a fourth-quarter surge for the therapy. Endeavor has acquired the exclusive, worldwide rights to Hummingbird’s HMBD-501, a HER3-targeted ADC with an exatecan payload. Hummingbird could receive up-front and milestone payments of up to $430 million, along with royalties from net sales.
Nkarta Inc. is looking to return to the heady heights it hit three years ago when its shares were going for around $70 each. The company is reinventing itself as a cancer fighter and branching out as an autoimmune specialist. Nkarta joins at least four other companies looking at expanding their cell therapies into other autoimmune indications.
Monte Rosa Therapeutics Inc. has cut a deal with Roche Holding AG that brings the molecular glue degrader-based medicines developer an up-front $50 million and the possibility of more than $2 billion in milestone payments. The Boston-based company coupled the deal by releasing positive interim data from the phase I dose-escalation portion of its phase I/II open-label, multisite study of MRT-2359 in Myc-driven solid tumors.
Pfizer Inc. may have a blockbuster on its hands with the U.S. FDA’s approval of Velsipity (etrasimod), a selective sphingosine-1-phosphate (S1P) receptor modulator for adults with moderately to severely active ulcerative colitis (UC).
