The 39th Annual J.P. Morgan Healthcare Conference was sans Celgene Corp.'s annual tradition of kicking off the conference with preliminary revenue and earnings from the previous year, but plenty of other companies stepped up and offered preliminary results of their own. Unfortunately, some companies continued to face headwinds selling drugs during the pandemic as patients avoided their doctors' offices.
Money is flowing into gene therapy as investors have started looking favorably on the risk-reward prospects of the space. "I believe the risk has really substantially decreased as regulatory path has become clearer. There are more projects with lower risk," said Sarah Bhagat, a partner at Sofinnova Investments.
Prevail Therapeutics Inc. won't be prevailing on its own. Eli Lilly and Co. is acquiring the gene therapy company for $880 million up front plus an earn-out of up to $160 million if Lilly can gain regulatory approval for at least one of Prevail's drugs.
Atara Biotherapeutics Inc. licensed a pair of mesothelin-directed CAR T treatments, ATA-2271 and ATA-3271, to Bayer AG for $60 million up front with the potential for $610 million in development, regulatory and commercialization milestone payments. Atara is also eligible for tiered royalties that peak in the low double-digit percentage of net sales of the two drugs. South San Francisco-based Atara will provide translational and clinical manufacturing services for the two drugs that will be reimbursed by Bayer.
There will be plenty of data from clinical trials testing treatments for various lymphomas at the 62nd American Society of Hematology (ASH) annual meeting this weekend. While CAR T cells have changed the landscape in the relapsed and refractory space for large B-cell lymphoma patients with two FDA approved medications, Kymriah (tisagenlecleucel, Novartis AG) and Yescarta (axicabtagene ciloleucel, Gilead Sciences), "the uptake has been good but not dominant. There's still plenty of space. There's still plenty of need," Jason Westin, leader of the diffuse large B-cell lymphoma research team at the University of Texas MD Anderson Cancer Center in the Department of Lymphoma and Myeloma, said on a call with clients from Raymond James.
Multiple companies have had their FDA reviews put on hold because coronavirus-related travel restrictions at the FDA has kept their manufacturing plants from being inspected.
Wellcome Leap has launched its first program, dedicating $50 million to help develop human tissues, organoids and full organs. The Human Organs, Physiology and Engineering (HOPE) program is looking to bring biologists and engineers together to develop both therapeutic organs as well as organs that can be used in vitro to help discover and develop new medications.
Intellia Therapeutics Inc. is looking to disrupt the transthyretin (TTR) amyloidosis (ATTR) market with NTLA-2001, its CRISPR-based treatment designed to be a potential cure for the disease. The drug, which is delivered via a lipid nanoparticle, edits the patient's DNA in vivo to create a stop codon and eliminate the expression of TTR, the protein that aggregates in ATTR patients' nervous systems and hearts, disrupting their functions.
Be Biopharma Inc. is looking to develop engineered B cells to treat a wide range of diseases. The new category of cellular medicine is based on the work of David Rawlings and Richard James, researchers at Seattle Children’s Research Institute and the University of Washington.
Crispr Therapeutics AG's first stab at developing an allogeneic CAR T-cell therapy, CTX-110, looks promising, but the efficacy data were overshadowed by a death in the study.
