New and updated clinical data presented by biopharma firms at the Alzheimer's Association International Conference, including: Acumen, Alnylam, Athira, Biovie, Coya, Nkgen, Prothena, Taurx.
Responding to medical advances and new standards of care in Alzheimer’s, the U.S. Centers for Medicare & Medicaid Services (CMS) is proposing to end its 10-year-old coverage with evidence development policy that has limited Medicare reimbursement of amyloid PET scans to once in a lifetime for beneficiaries – and then only when they’re used in a CMS-approved trial.
Sangamo Therapeutics Inc. has signed an evaluation and option agreement with Prevail Therapeutics Inc., a wholly owned subsidiary of Eli Lilly & Co., through which Prevail has been granted rights to evaluate certain proprietary adeno-associated virus (AAV) capsids developed by Sangamo and may exercise certain options to license these capsids for multiple undisclosed neurological targets.
Shanghai Institute of Materia Medica of the Chinese Academy of Sciences have developed nitrogenous heterocyclic compounds described as as receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors.
Arrowhead Pharmaceuticals Inc. has filed an application in New Zealand for clearance to initiate a phase I/II trial of ARO-DUX4, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD).
Nysnobio Inc. has received a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to assist the advancement of the company’s lead parkin gene replacement therapy candidate, NB-001. The funding will support product manufacturing to enable IND safety studies in preparation for clinical trials evaluating NB-001 in Parkinson’s disease patients with biallelic loss-of-function mutations in the parkin gene, or parkin-PD.
Novartis AG just added muscle to its siRNA development effort by buying Dtx Pharma Inc. in an M&A deal that could total $1 billion. Novartis is getting a preclinical asset, DTx-1252, which just received the U.S. FDA’s orphan drug designation on June 8. Dtx is developing the siRNA candidate for treating the neuromuscular disease Charcot-Marie-Tooth disease type 1A (CMT1A). Basel, Switzerland-based Novartis will pay Dtx $500 million up front and make payments of up to $500 million in milestones completions.
Dealmaking proved alive and well, with Sangamo Biosciences Inc. disclosing a tie-up worth as much as $1.19 billion with a subsidiary of Eli Lilly and Co., while Mirum Pharmaceuticals Inc. pledged to acquire for $445 million the bile-acid product portfolio owned by Travere Therapeutics Inc.