Results The study (n=76) showed that 18% of patients receiving fostamatinib achieved a stable platelet response compared to none receiving placebo ( P =.0261).
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Patients who met the study’s primary endpoint had an increase in platelet counts to a level above 50,000/uL within the first weeks of treatment, offering early feedback as to whether it was a viable option for treating their ITP.
CEO Raul Rodriguez reminded listeners on a conference call that the FDA has asked for two controlled phase III trials and the second “is designed identically as the first”, so the company is “very confident” about duplicating the favorable outcome as the regulatory path is trod, “but we also would approach it differently if the results were different”. The study found fostamatinib to be generally well-tolerated, with gastrointestinal-related adverse events as the most frequent drug reactions. The product safety profile was consistent with previous clinical experience and that there were no fresh or unusual safety issues were discovered.
Some analysts say that fostamatinib could generate $360 million in annual sales for Rigel in case the approval limits its use to only patients who have failed treatment with existing therapies. Study subjects remained on treatment for up to 24 weeks.
Rigel’s shares had closed at $2.64 on Monday on the Nasdaq. This disease causes a patient’s own immune system to seek out and destroy blood platelets, which are critical for healing and clotting. That’s a relatively small patient population, so the FDA has already made a decision to give Fostamatinib Orphan drug status.
According to Rigel, about 50,000 to 60,000 adults in the United States live with primary chronic ITP. The company’s current clinical programs include fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, which is in Phase 3 clinical trials for immune thrombocytopenia (ITP); a Phase 2 clinical trial for autoimmune hemolytic anemia (AIHA); and a Phase 2 clinical trial for IgA nephropathy (IgAN). It has various product candidates in development: fostamatinib; R348; two oncology product candidates and two preclinical programs. Fostamatinib, which is taken orally, has the unique function of inhibiting SYK – the substance that causes platelet destruction in ITP patients.
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While I understand the market’s optimism given the clinical result, Rigel still has plenty of work ahead of it before Fostamatinib can start to actually produce revenue. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. “Importantly, we anticipate achieving substantial clinical milestones over the course of 2016 and 2017”.
