Today, a brief rundown of news announced ahead of the J.P. Morgan Healthcare Conference, including licensing deals from AbbVie and Novartis, earnings announcements from Vertex Pharmaceuticals and Alnylam Pharmaceuticals, and regulatory updates from Beam Therapeutics and Atara Biotherapeutics.
AbbVie is paying Yantai, China-based RemeGen $650 million up front, and potentially more than $5 billion overall, for a bispecific antibody drug targeting the proteins PD-1 and VEGF. The deal announced Monday hands AbbVie rights to develop, manufacture and commercialize RemeGen’s RC148 outside of China and nearby territories. The drug being evaluated in a Phase 1 lung cancer study and Phase 2 trials in other solid tumors and breast cancer. Some of those mid-stage studies are evaluating RC148 alongside an antibody-drug conjugate Pfizer gained rights to through a buyout of Seagen. That ADC is approved in China, as is an autoimmune drug RemeGen licensed to Vor Biopharma last year. — Jonathan Gardner
Ahead of a Monday presentation at the J.P. Morgan Healthcare Conference, Alnylam Pharmaceuticals on Sunday pre-announced fourth quarter results and issued revenue guidance for the coming year. Alnylam said its closely watched transthyretin amyloidosis drug Amvuttra generated $827 million between October and December, which missed consensus estimates by more than $20 million. But the $4.4 billion to $4.7 billion in projected 2026 net revenue from its TTR drugs — which include Amvuttra as well as an older medication, Onpattro — came in ahead of analysts’ projections. The numbers were issued alongside a new five-year strategy that involves hitting at least a 25% average annual growth rate through the end of 2030. Shares ticked down about 8%. — Ben Fidler
Vertex Pharmaceuticals also issued a business update that included insight into the recent sales performance for its pain drug Journavx. According to Vertex, more than 500,000 prescriptions have been filled since Journavx’s approval in January, and the company expects that number to more than triple in the coming year. Vertex has also secured coverage in acute pain with all of the large national pharmacy benefit managers. The updates suggest “steady growth” but imply fourth-quarter sales could come in slightly below consensus estimates of $33 million, wrote RBC Capital Markets analyst Brian Abrahams. Vertex also pre-reported $100 million in revenue for its gene editing treatment Casgevy. That number would come in slightly ahead of Wall Street projections, but the program “remains of low investor interest,” Abrahams wrote. — Ben Fidler
Beam Therapeutics, meanwhile, said that it's aligned with U.S. regulators on a potential accelerated approval submission for one of its base editing therapies. Beam is testing the therapy in people with the rare lung disease alpha-1 antitrypsin deficiency and, last year, delivered early results suggesting the treatment could correct the disease-causing mutation underlying the condition. On Sunday, Beam said that the Food and Drug Administration could clear the drug, BEAM-302, based on positive effects on surrogate markers over the course of a year. Beam expects to enroll about 50 more patients in an ongoing trial and treat them with the "optimal" dose, and will report updated results, as well as next steps for the program, by the end of the first quarter. Beam additionally anticipates seeking approval of a sickle cell therapy, risto-cel, later this year. Company shares climbed more than 25%. — Ben Fidler
Novartis is paying $165 million in upfront cash to license an experimental Alzheimer’s disease treatment from SciNeuro Pharmaceuticals. The drug is designed to remove toxic amyloid deposits from the brains of people with Alzheimer’s, but with the help of a “shuttle” technology that helps the medicine cross the blood-brain barrier. Mulitple other companies, including Roche, are evaluating similar approaches. SciNeuro, which recently raised $53 million in venture funding, could receive another $1.5 billion in the deal. — Jonathan Gardner
The FDA has again rejected a cell therapy from Atara Biotherapeutics, the company said Monday. A year ago, the agency cited manufacturing issues in turning back Atara’s request to approve Ebvallo for a type of post-transplant disease associated with Epstein Barr virus infections. Now, however, Atara said that the agency has reversed its previous position and claimed that the single-arm study supporting the company’s application is no longer sufficient to support an approval. Atara had previously aligned with FDA leaders on the study’s design through “multiple, documented meetings” over the last five years. Partner Pierre Fabre Pharmaceuticals, which holds commercial rights to Ebvallo, intends to meet with the FDA to find a path forward. Atara shares fell by more than 50%. — Delilah Alvarado