BPMC - Blueprint raised to Buy at Jefferies on market potential of rare disease therapy

Jefferies has upgraded Blueprint Medicines (NASDAQ:BPMC) to Buy from Hold as the commercial-stage biotech prepares to report topline data for registration-enabling PIONEER trial for Ayvakit in non-advanced (Indolent) systemic mastocytosis (ISM) in late summer 2022. A rare genetically driven condition, systemic mastocytosis impacts patients’ quality of life, with many suffering from the non-advanced form of the disease. Ayvakit, known as avapritinib in generic terms, is already approved in the U.S. for advanced systemic mastocytosis. Blueprint (BPMC) expects to seek FDA label expansion for Ayvakit in 2H 2022, targeting an additional indication in ISM. Citing a U.S. patient survey, Jefferies analysts led by Eun K. Yang point out that ISM patient population eligible for the treatment stands ~50% lower than the  company estimates. In reaction, the team trims their U.S. Ayvakit sales estimates by ~10-20% for 2023 – 25 and cuts the price target for Blueprint (BPMC) to $78 from $88 per

For further details see:

Blueprint raised to Buy at Jefferies on market potential of rare disease therapy