Blueprint Medicines (NASDAQ:BPMC) has released positive study results for Ayvakit, which is a therapy for treating advanced systemic mastocytosis or SM patients.
Blueprint to file supplemental New Drug application for Ayvakit
The company has indicated that encouraging Phase 1 and Phase 2 clinical study results will help it file supplemental New Drug Application with the FDA for Ayvakit in Q4 2020. Advanced systemic mastocytosis is a rare fatal disease whereby an uncontrolled growth of a specific white blood cell affects different organs. MS can result in anemia, bone fractures, and even organ damage. Blueprint studied 85 patients with advanced SM, and results showed that Ayvakit can extend median overall survival.
Already Ayvakit, which is a kinase inhibitor that blocks specific proteins in cells, has FDA approval in treating adults with metastatic gastrointestinal stromal tumors (GIST). Despite the approval, the company still has very few sales, and losses have been widening in the last six years. However, this could change this year, considering the company entered a partnership in July with Roche’s division.
Blueprint boosted through a partnership with Roche
Following the agreement, Roche received a commercialization license of the company’s cancer drug Pralsetinib outside China and the US. On the other hand, Blueprint will receive an equity investment of $100 million on its stock and cash of $675 million, which will push it into the black. Additionally, Blueprint will be eligible for potential milestone payments of $927 million. Recently Roche and Blueprint received FDA approval for using pralsetinib in treating non-small cell lung cancer adult patients.
JMP Securities analyst Reni Benjamin stated that Ayvakit approval is great news for Blueprint in what has been a spectacular year. The safety profile of the therapy looks great, which is very important. JMP approximates the market opportunity for advanced SM to be around $670 million per year in the US and EU. The analysts approximate the market for non-advanced SM at around $3.5 billion annually in Japan, the US, and the EU. The company is seeking approval for Ayvakit in treating non-advanced SM.