Regeneron Pharmaceuticals (REGN) and bluebird bio, Inc. (BLUE) announced a collaboration to apply their respective technology platforms to the discovery, development, and commercialization of novel immune cell therapies for cancer.
The collaborators will specifically leverage Regeneron’s VelociSuite® platform technologies for the discovery of human antibodies as well as T cell receptors (TCRs) directed against tumor-specific proteins and peptides, and bluebird bio will contribute its field-leading expertise in gene transfer and cell therapy.
George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron said, “Like Regeneron, bluebird is a science-focused company looking to push the limits of what novel technologies can do in drug discovery and development. We believe that the tremendous synergies between Regeneron’s proven technologies and bluebird’s toolbox of the 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 the rapidly advancing immuno-oncology candidates and combination approaches.”
Philip Gregory, D.Phil., Chief Scientific Officer of bluebird bio stated: “The collaboration with Regeneron complements bluebird bio’s growing immuno-oncology development portfolio, which includes clinical and pre-clinical CAR T and T- cell receptor programs. 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.”
The two collaborating firms selected six initial targets.
Regarding the cost, Regeneron and Bluebird Bio will equally share the costs of research and development up to the point of submitting an Investigational New Drug (IND) application.
Additional targets may be selected over the five-year research collaboration term.
Following the IND submission 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, it will be eligible to receive milestone payments and royalties from bluebird bio on any potential resulting products.
Regeneron will make a $100 million investment in bluebird bio common stock at a price of $238.10 per share, which represents a premium of 59% over the $150 closing price on August 3, 2018.
The $37 million premium will be credited against Regeneron’s initial 50 percent funding obligation for basic collaboration research, after which the collaborators will fund ongoing research equally.
The transaction is subject to preclearance by the Federal Trade Commission under applicable antitrust laws.
Cell-based immunotherapies such as chimeric antigen receptor T cells (CAR Ts) use human immune cells (typically T cells derived from the patient with cancer), which are genetically engineered to be modified. They are then returned to the patient to serve as therapeutic agents that specifically target and kill cancer cells.
Experience with this approach demonstrated that modified T cells are highly active therapies for a variety of blood cancers even after other treatment approaches have failed, and there are existing FDA-approved medicines that utilize this approach.
Bluebird Bio Approach: This firm uses 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.
Regeneron’s Approach: VelociSuite® technologies, which include VelocImmune® and Veloci-T, that enable the creation of fully-human antibodies and T cell receptors. They enable the T cells to reach both extracellular and intracellular tumor antigens, which expands the types of tumors that modified T cells can safely and effectively target
Bluebird’s bio’s gene therapy clinical programs include investigational treatments for cerebral adrenoleukodystrophy, transfusion-dependent β-thalassemia, also known as β-thalassemia major, and severe sickle cell disease.
Bluebird bio’s oncology pipeline is built around the firm’s lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. Bluebird bio’s lead oncology programs are anti-BCMA CAR T programs partnered with The Top-Tier biotech firm Celgene (CELG).
Regeneron has six FDA-approved treatments and many product candidates in development, all of which were homegrown in its laboratories. Regeneron’s therapeutics and experimental products deal with eye disease, heart disease, allergic and inflammatory diseases, pain, cancer, infectious diseases and rare diseases.
Regeneron is improving the traditional drug development process through its proprietary technologies VelociSuite® , including VelocImmune® (a genetically-engineered mouse model that has a genetically-humanized B cell immune system that produces optimized fully-human antibodies) and Veloci-T (a genetically-engineered mouse model that has genetically-engineered T cell immunity). Regeneron is conducting ambitious research in it Regeneron Genetics Center®, which is said to conduct one of the largest genetics sequencing efforts in the world.
Another Gene Therapy Firm that we Picked and Continue To Admire
Another firm that we admire is RegenxBio (RGNX).
This Firm is focused on genetic engineering through its advanced Adeno-Associated viral vectors. Regenxbio shares have outperformed since we added this firm to our portfolio. We explained why is our enthusiasm in previous articles and in the Prohost Biotech Letters and articles.
We will add more information about this firm in the upcoming Week in Review.