Blueprint Medicines Announces Strong Q1 2024 Results, Raises AYVAKIT®/AYVAKYT® Revenue Guidance

Blueprint Medicines Corporation, based in Cambridge, Massachusetts, has announced its financial results for the first quarter of 2024, along with a business update and revised financial guidance. The company reported strong performance in the launch of AYVAKIT for indolent systemic mastocytosis (ISM), achieving $92.5 million in net product revenues for the quarter, reflecting a 135% year-over-year growth. Consequently, Blueprint Medicines has raised its full-year AYVAKIT revenue guidance to between $390 million and $410 million.

CEO Kate Haviland expressed satisfaction with the company's progress, stating that the initial quarters of AYVAKIT's launch have set a strong sales trajectory, positioning the product to potentially exceed $2 billion in peak sales for systemic mastocytosis. The increased revenues also allow the company to invest in other pipeline opportunities, particularly in the fields of allergy and inflammation, as they prepare to move BLU-808, a wild-type KIT inhibitor, into clinical trials in the latter half of 2024.

During the first quarter, significant advancements were made in mast cell disorders. Blueprint Medicines presented long-term data from the PIONEER trial for AYVAKIT in ISM, which demonstrated durable symptom relief and a favorable safety profile. Additionally, preclinical data for BLU-808 showed promising results in chronic urticaria and other mast cell disorders, leading to plans for an Investigational New Drug (IND) filing with the FDA in Q2 2024 to begin Phase 1 studies.

In the HR+/HER2- breast cancer segment, the company progressed with the development of BLU-222, a CDK2 inhibitor. Plans are in place to present positive combination safety data with ribociclib and fulvestrant at the 2024 ASCO Annual Meeting, indicating early clinical activity.

Corporate developments included an agreement with Rigel Pharmaceuticals, Inc., granting Rigel the U.S. rights to research, develop, and market GAVRETO (pralsetinib), ensuring continued patient access. Blueprint Medicines also published its third annual Sustainability Report, detailing progress on environmental, social, and governance (ESG) initiatives and focusing on key ESG topics for stakeholders.

Financially, Blueprint Medicines reported total revenues of $96.1 million for Q1 2024, including $92.5 million from AYVAKIT and $3.6 million from collaboration revenues. This marks an increase from the $63.3 million recorded in Q1 2023. The cost of sales remained flat at $3.2 million, while research and development expenses decreased to $88.2 million from $112.1 million the previous year, due to operational efficiencies and timing of clinical trial material manufacturing. Selling, general, and administrative expenses rose to $83.6 million from $71.0 million, driven by commercialization activities for AYVAKIT.

Net income for the quarter was $89.1 million, compared to a net loss of $129.6 million in Q1 2023. This turnaround was largely due to a one-time non-cash debt extinguishment gain of $173.7 million from the Royalty Pharma termination agreement. As of March 31, 2024, the company's cash, cash equivalents, and investments totaled $735.6 million, ensuring a strong capital position.

Blueprint Medicines aims to achieve several milestones in the first half of 2024, including the submission of an IND for BLU-808 and presenting data for BLU-222 at the ASCO Annual Meeting. The company is also engaging in ongoing strategic business development discussions.

Overall, Blueprint Medicines is well-positioned for sustained growth and innovation, driven by its robust pipeline and strategic investments in key therapeutic areas.