FDA grants breakthrough therapy status to BeOne’s lymphoma drug

Published 13/10/2025, 11:06
FDA grants breakthrough therapy status to BeOne’s lymphoma drug

SAN CARLOS, Calif. - The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to sonrotoclax, an investigational BCL2 inhibitor being developed by BeOne Medicines Ltd. (NASDAQ:ONC) for treating adults with relapsed or refractory mantle cell lymphoma. The company, currently valued at $35.24 billion, has shown strong market performance with a 73.3% gain year-to-date, according to InvestingPro data.

The FDA also accepted BeOne’s request to participate in Project Orbis, an initiative that enables concurrent review of oncology products among global health authorities, potentially accelerating international access to the treatment.

The designation was based on early results from a Phase 1/2 study evaluating sonrotoclax in patients who had previously received Bruton’s tyrosine kinase inhibitor and anti-CD20 therapy. BeOne plans to present complete data from the study at an upcoming medical conference.

Mantle cell lymphoma is a rare, aggressive form of non-Hodgkin lymphoma that accounts for approximately 5% of all NHL cases globally. The five-year survival rate is about 50%, according to information provided in the company’s announcement.

The Phase 1/2 study enrolled 125 adult patients with relapsed or refractory mantle cell lymphoma. In Part 1, 22 patients received daily treatment with either 160 mg or 320 mg of sonrotoclax, while in Part 2, 103 patients received the recommended daily dose of 320 mg.

This marks the first Breakthrough Therapy Designation for sonrotoclax and the second for BeOne’s hematology program. The FDA has previously granted the drug Fast Track Designation for mantle cell lymphoma and Waldenström macroglobulinemia, as well as Orphan Drug Designation for several blood cancers.

A Phase 3 confirmatory study called CELESTIAL-RRMCL is currently underway, according to the company’s press release statement. While currently operating at a loss, analysts tracked by InvestingPro expect BeOne to achieve profitability this year, with multiple analysts recently revising their earnings forecasts upward. Subscribers can access 8 additional exclusive ProTips and detailed financial metrics for BeOne Medicines.

In other recent news, BeOne Medicines has seen a series of significant developments. The company announced positive results from a Phase 1/2 study of sonrotoclax in patients with relapsed/refractory mantle cell lymphoma, achieving its primary endpoint of overall response rate. Barclays has initiated coverage on BeOne Medicines with an Overweight rating, setting a price target of $385.00, highlighting potential growth from upcoming pivotal data readouts. Bernstein SocGen Group raised its price target for BeOne Medicines to $362.00, up from $259.00, while maintaining a Market Perform rating, citing increased estimates for its B-cell products. Citizens JMP reiterated its Market Outperform rating with a price target of $348.00, noting key value drivers in its analysis. Meanwhile, Oncolytics Biotech has unveiled the design for its pancreatic cancer registration trial, which will feature a three-arm design and is set to discuss with the FDA in mid-November 2025. These developments reflect ongoing strategic advancements and potential growth opportunities for both companies.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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