Rigel's fostamatinib enters Phase 1 trial for sickle cell treatment

Published 22/01/2025, 14:14
Rigel's fostamatinib enters Phase 1 trial for sickle cell treatment

This article is based on a press release statement from Rigel Pharmaceuticals (NASDAQ:RIGL), Inc. The company maintains a healthy financial position with a current ratio of 1.96 and operates with a moderate debt level. For detailed financial analysis and expert insights on Rigel's growth prospects, investors can access the full research report available on InvestingPro, which offers comprehensive coverage of over 1,400 US stocks. The company maintains a healthy financial position with a current ratio of 1.96 and operates with a moderate debt level. For detailed financial analysis and expert insights on Rigel's growth prospects, investors can access the full research report available on InvestingPro, which offers comprehensive coverage of over 1,400 US stocks.

Today, the company announced the enrollment of the first patient in the trial. Fostamatinib is currently approved in the United States under the brand name TAVALISSE® for treating adult patients with chronic immune thrombocytopenia (ITP) who have not adequately responded to other treatments.

The open-label Phase 1 dose-escalation study aims to enroll approximately 20 patients with SCD. Participants will receive fostamatinib at a starting dose of 100 mg twice daily for 14 days, potentially increasing to 150 mg twice daily for an additional 28 days if tolerated. The primary focus is on the drug's safety and tolerability, with secondary objectives exploring its mechanism of action in SCD and its effects on red blood cell membrane integrity and other related biological processes. With a robust gross profit margin of 76% and positive analyst sentiment, as revealed in InvestingPro's comprehensive analysis (which includes 12+ additional key insights), the company appears well-positioned to advance its clinical programs.

Dr. Richard Childs, scientific director of the NHLBI, expressed optimism about the potential of SYK inhibition to mitigate complications associated with SCD, a condition that causes significant pain and life-threatening complications due to abnormal red blood cells that can block blood flow.

Rigel's President and CEO, Raul Rodriguez, also commented on the significance of the study for addressing an area of critical unmet need and advancing Rigel's mission to improve the lives of patients with hematologic disorders and cancer.

SCD is a genetic disorder affecting over 100,000 people in the United States and millions worldwide. It is characterized by the production of abnormal hemoglobin, leading to rigid, sickle-shaped red blood cells that can cause severe health complications.

Preclinical studies have indicated that the active metabolite of fostamatinib may reduce thrombo-inflammation, a key issue in SCD, without increasing bleeding risk. The study is conducted at the NIH Clinical Center in Bethesda, Maryland, with the NIH/NHLBI funding and sponsoring it and Rigel providing the study material.

This article is based on a press release statement from Rigel Pharmaceuticals, Inc.

In other recent news, Rigel Pharmaceuticals announced strong Q4 sales of approximately $57.6 million, marking a significant increase from the same period of 2023. The company's net product sales for the quarter are projected at $46.5 million, with TAVALISSE®, REZLIDHIA®, and GAVRETO® contributing to this figure. Rigel also anticipates total revenue for 2025 to reach between $200 to $210 million, suggesting confidence in continued growth.

Rigel's investigational compound R289 has been granted Orphan Drug designation by the FDA for the treatment of myelodysplastic syndromes and is currently undergoing a Phase 1b clinical study. In addition, Rigel's drug, R289, secured FDA Fast Track designation for MDS treatment, highlighting the potential of this drug to improve patient outcomes.

Citi has maintained a positive outlook on Rigel Pharmaceuticals, raising the stock's price target to $49.00 from the previous $40.00, influenced by the strong sales of Gavreto and the company's robust growth in quarterly revenue. Rigel Pharmaceuticals also announced a $10 million agreement with Kissei to develop Rezlidhia in Asia, with potential future payments reaching up to $152 million.

These are among recent developments, reflecting Rigel's focus on strategic growth and product pipeline development. The company's financial turnaround and strong sales growth, coupled with upcoming presentations and collaborations, are key highlights in Rigel's recent performance.

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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