CAMBRIDGE, Mass.—While the upfront cash payment may “only” be $45 million, Blueprint Medicines Corp. is looking at a potential for $965 million in option fees and milestone payments, for a total of more than $1 billion if all goes according to its hopes and plans, in a deal inked in mid-March with Roche.
Blueprint touts itself as a” leader in discovering and developing highly selective kinase medicines for patients with genomically defined diseases” and notes that the deal calls for the discovery, development and commercialization of up to five small-molecule therapeutics targeting kinases that are believed to be important in cancer immunotherapy.
“By leveraging our proprietary drug discovery platform to selectively target immunokinases, we believe we may be able to enhance response rates and increase the potential for combination treatment approaches in cancer immunotherapy,” said Jeff Albers, CEO of Blueprint Medicines, in a March 15 conference call about the deal. “As we continue to focus on advancing our clincal-stage programs, we also intend to continue to invest in our discovery platform, advancing new discovery-stage programs each year.”
He noted that his company has a stated goal in 2016 to nominate two new discovery programs by the end of the year, and that Blueprint has been working on a number of targets, including immunokinase targets.
“By partnering with Roche we have accelerated our ability to advance multiple immunokinase programs this year while continuing to focus on additional kinase opportunities,” he added. “With respect to our business development efforts, the Roche alliance directly reflect our objective to work with partners that value our approach to combining our proprietary compound library with our genomics expertise.”
As for what will net Blueprint the bulk of the cash, the $965 million in contingent option fees and milestone payments are related to specified research, preclinical, clinical, regulatory and sales-based milestones across all five potential programs. Of the total contingent payments, around $215 million potentially is tied to option fees and milestone payments for research, preclinical and clinical development events prior to licensing across all five potential programs. In addition, the agreement provides for specified royalties and cost sharing.
Blueprint notes that so far, cancer immunotherapies have demonstrated important clinical benefits but that most have focused on antibodies or combinations with existing approved therapies. What they haven’t done is attempt to target immunokinases with small molecules, the company says, and Blueprint and Roche hope to change that by developing new mechanisms of modulating the tumor immune response by targeting immunokinases. One of the ultimate goals is to improve response rates and broaden the utility of using cancer immunotherapies to treat additional cancer types.
Immunokinases are intracellular targets known to regulate numerous aspects of immune response and, according to Blueprint, “represent an important opportunity for potentially innovative approaches to enhance the immune system’s ability to recognize and eradicate tumor cells.”
“We believe Blueprint Medicines’ proprietary drug discovery platform and expertise in immunokinases, combined with our proven ability to move quickly through drug discovery, is a perfect complement to Roche’s expertise with cancer immunotherapy biology and in developing and commercializing innovative therapies,” said Albers in the news release announcing the collaboration. “Under this collaboration, Blueprint Medicines will lead preclinical research and development through Phase 1 proof of concept for all five programs and retain U.S. commercial rights for two programs. We believe this highly collaborative relationship will enable us to accelerate our efforts in the emerging field of cancer immunotherapy and to continue building a leading biotechnology company.”
As FierceBiotech writer Damian Garde noted of the deal on March 15, “Most of today’s cancer immunotherapies, including Roche’s in-development atezolizumab, are antibody treatments that work by removing the brakes from cancer-fighting T cells. Roche is betting that Blueprint’s early-stage work could bring about a complementary force, dovetailing with the effects of so-called checkpoint inhibitors like atezolizumab to create a more powerful weapon against tumors.”
Now, for those who like the art of the deal and need to know more about specifics on the business side, here is the nitty-gritty of the deal structure, pretty much verbatim from Blueprint and Roche:
The collaboration provides for the worldwide development and commercialization of immunokinases in the field of cancer immunotherapy for up to five small-molecule drug candidates as single products or possibly in combination with Roche’s portfolio of therapeutics. Roche’s rights are structured as an option, triggered upon achievement of Phase 1 proof-of-concept studies, for an exclusive license to each drug candidate developed under the collaboration. Blueprint Medicines will be primarily responsible for preclinical research and conduct of clinical development for each program prior to any exercise of Roche’s option for such programs. If Roche exercises an option for a program, Roche will be responsible for subsequent global development for that program through registrational clinical trials. For up to three of the five programs, if Roche exercises its option, Roche will receive worldwide commercialization rights for the licensed product. For up to two of the five programs, if Roche exercises its option, Blueprint Medicines will retain commercialization rights in the United States for the licensed product, and Roche will receive commercialization rights outside of the United States for such licensed product. Blueprint Medicines will also retain worldwide rights to any drug candidates for which Roche elects not to exercise the applicable option.
For any licensed product for which Roche retains worldwide commercialization rights, Blueprint Medicines will be eligible to receive tiered royalties ranging from low-double-digits to high-teens on future net sales of the licensed product. For any licensed product for which Blueprint Medicines retains commercialization rights in the United States, Blueprint Medicines and Roche will be eligible to receive tiered royalties ranging from mid-single-digits to low-double-digits on future net sales in the other party’s respective territories in which it commercializes the licensed product. Blueprint Medicines and Roche will share the costs of Phase 1 development for each collaboration target. In addition, Roche will be responsible for post-Phase 1 development costs for each licensed product for which it retains global commercialization rights, and Blueprint Medicines and Roche will share post-Phase 1 development costs for each licensed product for which Blueprint Medicines retains commercialization rights in the United States.