Gilead Sciences Inc. was looking to get into oncology in a big way. Arcus Biosciences Inc. had a pipeline of cancer drugs it didn't want to break up. While a little unusual, the landmark 10-year pact the companies made last year just made sense, company executives explained during a session at Biocom California's Global Life Science Partnering Conference.
The 2017 FDA approval of Luxturna (voretigene neparvovec-rzyl, Roche Holding AG) spurred a race to create the next gene therapy for the eye. The organ is very amenable to gene therapy given that it's a confined space with post-mitotic cells that has immune privilege and requires substantially smaller amounts of viral vector compared to systemic treatments.
With the FDA approval of Keytruda (pembrolizumab, Merck & Co. Inc.) in the front-line setting of metastatic or unresectable, recurrent head and neck cancer squamous cell carcinoma, the space has become a race to develop drugs using a variety of mechanisms of action to improve the efficacy of the anti-PD-1 monoclonal antibody.
The 2020 World Conference on Lung Cancer, which was scheduled to take place in Singapore last August, is set to kick off virtually later this week. The postponement gave companies time to generate additional data as they battle to treat patients with their targeted therapies.
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.