#United States #San Diego #Rigel Pharmaceuticals #R289 #ASH 2024

Rigel Pharmaceuticals Showcases Initial Data on R289

Rigel Pharmaceuticals, Inc. (NASDAQ: RIGL), a pioneering player in the biotechnology arena focused on hematologic disorders and cancer therapies, has shared exciting preliminary results from their ongoing Phase 1b clinical trial of R289. This trial focuses on patients suffering from relapsed or refractory lower-risk myelodysplastic syndrome (LR-MDS), a condition that currently lacks comprehensive treatment options.

During the 66th American Society of Hematology (ASH) Annual Meeting, held from December 7-10, 2024, in San Diego, the company exhibited significant data suggesting that R289 has shown a favorable safety profile and potential therapeutic efficacy. The presentation was delivered by Dr. Guillermo Garcia-Manero, shedding light on crucial insights into the treatment of elderly patients burdened with this serious blood disorder.

Highlights of the Study

Elderly patients, particularly those who are transfusion-dependent with LR-MDS, often find themselves with limited treatment avenues. Dr. Lisa Rojkjaer, Rigel's Chief Medical Officer, expressed optimism regarding the findings, noting that R289 was well tolerated among study participants, and initial clinical responses, such as transfusion independence, have been observed.

The ongoing Phase 1b study primarily aims to measure the safety and tolerability of R289, alongside secondary goals that evaluate its preliminary efficacy and pharmacokinetics. As of October 25, 2024, a total of 22 patients have been enrolled, with a median age of 76 and an average of three prior therapies experienced by participants.

Key observations include:

• - Among the enrolled patients, 73% had undergone treatment with a hypomethylating agent, while 77% had been treated with luspatercept.
• - Reported adverse events were minimal, with the most prevalent being Grade 1/2 diarrhea and fatigue, affecting 27% of patients. Higher severity events, Grade 3/4, including anemia and pneumonia, were less common, reflecting R289's manageable safety profile.
• - The median duration of therapy participation was 4.6 months, indicating a commitment to ongoing patient monitoring and evaluation.

Encouraging Efficacy Data

The efficacy evaluations revealed that 40% of participants classified as transfusion-dependent showed response rates after dosing R289 at or above 500 mg once daily. Among these patients, Red Blood Cell (RBC) transfusion independence was achieved for a duration ranging from 12.7 to 51.9 weeks, showcasing the drug's potential to significantly impact patient quality of life. Furthermore, one patient even presented a minor improvement in hematologic parameters, highlighting R289's promising profile as a treatment choice.

Rigel's commitment to bringing R289 to market has received an additional boost from the FDA's Fast Track designation, affirming its potential to meet an urgent medical need for patients affected by previously treated transfusion-dependent LR-MDS.

Conclusion

With R289 now entering more advanced stages of clinical evaluation, Rigel Pharmaceuticals is focused on continuing to explore the full potential of this innovative therapy as a new treatment pathway for a population of patients who currently have limited options. As the landscape of cancer therapies continues to evolve, the insights gleaned from this Phase 1b trial may pave the way for enhanced treatment paradigms.

For more updates and detailed information about Rigel Pharmaceuticals and R289, visit Rigel's official website.