Nov. 20 Quick Takes: Mixed myelofibrosis results from MorphoSys’ pelabresib

MorphoSys AG (NASDAQ:MOR; Xetra:MOR) is readying regulatory submissions in the U.S. and Europe for pelabresib to treat JAK inhibitor-naive patients with myelofibrosis despite Chief Research and Development Officer Tim Demuth’s assertion in June that “positive p-value” for both spleen volume reduction and symptom score reduction would likely be needed for regulatory approval — something pelabresib failed to show. The BET inhibitor met the primary endpoint of spleen volume reduction in a Phase III study, with the 66% of patients in the pelabresib plus ruxolitinib group achieving at least a 35% reduction in spleen volume at week 24 vs. 35% in the placebo plus ruxolitinib group. However, the combination missed the trial’s two key secondary endpoints measuring symptom reduction. MorphoSys noted in a press release that the secondary endpoints showed a trend favoring the pelabresib-ruxolitinib combination, and that statistical significance was reached in a subgroup of intermediate-risk patients. In June, CEO Jean-Paul Kress described to BioCentury how pelabresib fits into his efforts to transition MorphoSys from a platform company to a product company. 

Bristol Myers Squibb Co. (NYSE:BMY) and 2seventy bio Inc. (NASDAQ:TSVT) said FDA will miss the Dec. 16 PDUFA date on an sBLA seeking approval of anti-BCMA CAR T Abecma idecabtagene vicleucel for earlier-line treatment of relapsed or refractory multiple myeloma. The partners now expect the agency to hold an advisory committee meeting on a date to be confirmed to discuss overall survival data from the Phase III KarMMa-3 study, which will be presented at the 2023 American Society of Hematology (ASH) Annual Meeting and Exposition on Dec. 11. Abecma is already approved for patients who have received four or more prior lines. In the KarMMa-3 trial, Abecma met the primary endpoint of progression-free survival vs. standard regimens in patients who had received fewer two to four prior lines (13.3 months vs. 4.4 months; HR:0.49; p<0.0001). ...