Roche, Blueprint Medicines Team Up Again in $775M RET Cancer Drug Pact

Xconomy Boston — 

A Blueprint Medicines cancer drug candidate currently under FDA review will have the marketing muscle of Roche behind it if it wins regulatory approval.

The two companies are partnering on the drug, pralsetinib, a targeted cancer therapy developed for lung and thyroid cancers characterized by abnormalities in a gene called RET. According to deal terms announced Tuesday, the companies will jointly commercialize the pill in the US. Roche gains rights to sell the drug globally, except for China. The Swiss pharmaceutical giant agreed to pay Cambridge, MA-based Blueprint (NASDAQ: BPMC) $675 million up front for those rights. It’s also making a $100 million equity investment in its partner at $96.57 per share, a nearly 26 percent premium to Monday’s closing stock price.

If the Blueprint drug achieves development, regulatory, and sales-based milestones, the biotech could earn up to $927 million more. Those milestones also cover any next-generation RET-targeting drug developed under the partnership. The collaboration “fundamentally changes the trajectory of both pralsetinib and Blueprint Medicines,” CEO Jeff Albers said on a Tuesday conference call.

“We’re now positioned to accelerate and expand the development of pralsetinib, and deliver to patients faster and more broadly on a global scale than we could alone,” he added.

The RET gene provides the genetic instructions for producing a protein involved in cell signaling. The growth of many types of cancers have been associated with fusions and mutations to the gene, and Blueprint designed pralsetinib to block these aberrations. The company developed the drug as a treatment for non-small cell lung cancer (NSCLC) characterized by fusions of the RET gene. It’s under priority review by the FDA, and a regulatory decision is expected by Nov. 23. The European Medicines Agency is also reviewing the drug for NSCLC.

In addition, Blueprint has also developed the drug for thyroid cancers. The first indication is thyroid cancer that’s positive for RET gene fusions. The second is medullary thyroid cancer (MTC), which stems from different cells than thyroid cancer, and is driven by RET mutations. The thyroid cancer applications of the drug are being reviewed under a pilot FDA program called “real-time oncology review,” which aims to more quickly evaluate new cancer drugs.

FDA approval of the first RET inhibitor was awarded in May to Eli Lilly (NYSE: LLY) drug selpercatinib (Retevmo). The decision for the twice-daily pill covers NSCLC, thyroid cancer, and medullary cancer driven by RET fusions or mutations. But before treatment, the genetic aberration must first be confirmed with a test. Lilly reached a deal last year with Thermo Fisher Scientific (NYSE: TMO) to develop a companion diagnostic for selpercatinib.

A comparable test for once-daily pralsetinib could come from Roche. Albers said on the call that Roche brings to Blueprint its precision medicine expertise, which will help the company more efficiently identify the patients eligible for treatment with the drug “through innovative application of both data and diagnostics.” Roche’s Flatiron Health subsidiary uses its software to analyze patient cancer data for drug research and identifying patients for clinical trials. Roche was a minority shareholder in Flatiron until 2018 when it paid $1.9 billion to acquire it outright.

Blueprint has one FDA-approved product. In January, the agency OK’d avapritinib (Ayvakit) as a treatment for gastrointestinal stromal tumor characterized by a particular genetic mutation. Albers said the commercialization activity around that drug combined with the ongoing work with pralsetinib drove the company to look for a partner, preferably one with global reach. According to Albers, some of the suitors focused on pralsetinib while others emphasized Blueprint’s preclinical pipeline.

Roche and Blueprint are already well-acquainted. In 2016, the companies struck up an alliance focused on developing small molecules for cancer immunotherapy. That pact now encompasses up to four programs. The only disclosed program targets MAP4K1, which the company says plays a role in T cell regulation. The progress of that alliance helped steer Blueprint toward Roche as a pralsetinib partner.

“That existing relationship and the trust that was established there that was one of the key drivers in proceeding with Roche here,” Albers said. “Any great collaboration is grounded in shared views around science and then shared goals and trust. That existing collaboration has been really fruitful and productive.”

Albers swatted down analyst suggestions that the alliance could set up Blueprint to be acquired by Roche. He said the structure of the deal ensures the financial independence of the biotech, providing it with the capital to invest in pralsetinib and the rest of the Blueprint drug pipeline, including fisogatinib, an experimental treatment in early-stage clinical development for hepatocellular carcinoma.

Blueprint believes that pralsetinib has potential applications in other cancers driven by RET gene alterations. The alliance with Roche calls for the companies to share in further development of pralsetinib in other solid tumors. The pact excludes China, Hong Kong, Macau, and Taiwan. CStone Pharmaceuticals holds development and commercialization rights to avapritinib, pralsetinib, and fisogatinib in those markets.

Image: iStock/Zolnierek