Looking for novel cancer therapeutics

TARRYTOWN, N.Y. & CAMBRIDGE, Mass.—Regeneron Pharmaceuticals Inc. and bluebird bio Inc. on Aug. 6 announced a collaboration under which they will apply their respective technology platforms to the goal of discovering and developing novel immune cell therapies for cancer.

For bluebird bio’s part, the company brings its expertise in gene transfer and cell therapy, while Regeneron will more specifically leverage its VelociSuite platform technologies for the discovery and characterization of fully human antibodies and T cell receptors (TCRs) directed against tumor-specific proteins and peptides.

Just as they will both bring established technology and expertise, the two companies also will equally share the costs of research and development, up to the point of submitting an Investigational New Drug (IND) application. They have highlighted six targets initially as part of the collaboration deal.

If and when an IND is submitted for a potential cell therapy product, Regeneron will have the right to opt-in to a co-development/co-commercialization arrangement for certain collaboration targets, with 50/50 cost- and profit-sharing. If Regeneron does not opt-in, the company is eligible to receive milestone payments and royalties from bluebird bio on any potential resulting products.

In addition, Regeneron agreed to make a $100-million investment in bluebird bio common stock at a price of $238.10 per share, which represents a premium of 59 percent over the $150 closing price on Aug. 3. The premium will be credited against Regeneron’s initial 50-percent funding obligation for basic collaboration research.

“Like Regeneron, bluebird is a science-focused company looking to push the limits of what novel technologies can do in drug discovery and development,” said Dr. George D. Yancopoulos, president and CEO of Regeneron. “We believe that the tremendous synergies between Regeneron’s proven technologies and bluebird’s toolbox of advanced cell and gene therapy technologies create a promising opportunity to help people with cancer by developing innovative new treatments. This collaboration adds yet another dimension to our rapidly advancing portfolio of immuno-oncology candidates and combination approaches.”

Cell-based immunotherapies such as chimeric antigen receptor T cells (CAR-Ts) use human immune cells that are modified and returned to the patient to serve as therapeutic agents that specifically target and kill cancer cells. bluebird bio’s technologies use a customized lentiviral vector to modify T cells so that they can recognize tumor-specific proteins expressed by cancer cells and kill them upon engagement. Meanwhile, Regeneron’s VelociSuite technologies allow the creation of fully human antibodies and T cell receptors.

As the companies noted in a news release about the deal, “These complementary technologies have the potential to expand the types of tumors that modified T cells can safely and effectively target by enabling the T cells to reach both extracellular and intracellular tumor antigens.”

“The collaboration with Regeneron complements bluebird bio’s growing immuno-oncology development portfolio, which includes clinical and preclinical CAR-T and T cell receptor programs,” Dr. Philip Gregory, chief scientific officer of bluebird bio, commented in a statement. “With Regeneron’s proven targeting technologies, in combination with our deep expertise in cell biology and vector technology, as well as clinical experience with leading CAR-T cell drug products, we hope to rapidly advance novel cellular therapies with the potential to transform the lives of people with cancer.”

Reporting on the deal, Xconomy noted that Regeneron is not as far along as other major cancer immunotherapy players like Bristol-Myers Squibb and Merck & Co., but added that in partnership with Sanofi, Regeneron does have cemiplimab, which could soon be approved for certain skin cancers—as well as being tested by Regeneron and Sanofi in other cancer types.

As for bluebird, Xconomy noted that it “is closest to approval with a gene therapy—a one-time, long-lasting treatment—for the blood disease beta-thalassemia. But the company is developing cancer treatments as well. It has two cell therapies for multiple myeloma in human testing that are both part of a partnership with Celgene.” Thus, the deal with Regeneron gives bluebird a leg up to move on developing more potential cancer therapies.