After hearing two conflicting presentations of the safety and efficacy of Geron Corp.’s imetelstat, the U.S. FDA’s Oncology Drugs Advisory Committee (ODAC) voted 12-2 March 14 that the drug’s benefit outweighed its risks as a treatment for transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes in patients who have failed or no longer respond to erythropoiesis stimulating agents (ESAs), or who are not eligible for ESA treatment.
Although Geron Corp.’s imetelstat met its primary and key secondary endpoints in a phase III study, the U.S. FDA is questioning the magnitude and durability of the effect of the first-in-class telomerase inhibitor as a second-line treatment of transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes. The agency’s concerns resulted in more than an 12% stock tumble March 12 after the FDA released its briefing document two days ahead of an Oncology Drugs Advisory Committee meeting, in which the panel will be asked to vote on whether imetelstat’s benefits outweigh its risks.
Halia Therapeutics Inc. has, its CEO confidently asserts, taken an atypical road to building its infrastructure and financing. The Lehi, Utah-based company just completed a $30 million series C financing to further develop its lead asset, a selective and orally bioavailable first-in-class NLRP3/NEK7 inflammasome inhibitor.
Remix Therapeutics Inc. has closed a $60 million financing to advance its lead program, REM-422, into the clinic and for further advancement of a pipeline of RNA processing targeted therapeutics.
Wall Street will be watching closely for such adverse effects as anemia that foiled Gilead Sciences Inc.’s CD47-binding magrolimab earlier this year, but so far Shattuck Labs Inc.’s SL-172154 looks strong in combination with azacitidine to treat front-line higher-risk myelodysplastic syndrome (HR-MDS) and TP53-mutant (TP53m) acute myeloid leukemia (AML).
I-Mab Biopharma Co. Ltd. has regained full rights to its CD47 antibody program from Abbvie Inc., including lemzoparlimab, the most advanced candidate. The move, disclosed in a Sept. 22 U.S. SEC filing, eliminates the potential $1.295 billion in milestones associated with the amended collaboration deal signed in 2022.
I-Mab Biopharma Co. Ltd. has regained full rights to its CD47 antibody program from Abbvie Inc., including lemzoparlimab, the most advanced candidate. The move, disclosed in a Sept. 22 U.S. SEC filing, eliminates the potential $1.295 billion in milestones associated with the amended collaboration deal signed in 2022.
The U.S. FDA has awarded Bristol Myers Squibb Co. (BMS) with its third approval for treating anemia with Reblozyl (luspatercept-aamt). Specifically, the approval is for treating anemia without previous erythropoiesis stimulating agent use in adults with very low- to intermediate-risk myelodysplastic syndromes who may require regular red blood cell transfusions.