Phase II melanoma data characterized by Iovance Biotherapeutics Inc. as positive failed to excite Wall Street, which took away 53.6% of the company’s share value (NASDAQ:IOVA), or $8.10, and pushed the closing price to $7.02 on May 27. The San Carlos, Calif.-based firm offered results from registrational cohort 4 (n=87) of the C-144-01 study testing lifileucel (LN-144, autologous tumor infiltrating lymphocytes) in advanced melanoma.
Nurix Therapeutics Inc. has started its first phase Ib expansion cohort for treating chronic lymphocytic leukemia with NX-2127 but it is facing the headwind of a drop in share price. Shares (NASDAQ:NRIX) of the San Francisco-based company closed 22.7% lower at $7.88 each on May 26. Share prices in the past 12 months have drifted lower since their crest of $26.69 each on Sept. 9, 2021.
Interim phase I data from 16 patients with refractory metastatic uveal melanoma provide a tantalizing hint that Ionctura SA’s phosphoinositide 3-kinase (PI3K) delta inhibitor IOA-244 PI3 may offer a survival benefit. Thirteen of the 16 patients –
81.25% – remained alive and on therapy after 12 months of treatment. All had received one or more prior therapies.
Solid phase III top-line results from a study in India for treating stroke with PMZ-1620 (sovateltide) have prodded Pharmazz Inc. to rethink its path to the clinic in the U.S. While the privately held company plans to apply for marketing authorization from the Indian Central Drugs Standard Control Organization, it also had planned to talk to the FDA about launching a phase II study. But since the new data are so solid, it may ask for an IND for a phase III study in the U.S., with the expectation that the number of participants would jump from 158 in the Indian study to as many as 400 to 500 participants in the U.S. and Europe, Anil Gulati, Pharmazz’s CEO and founder, told BioWorld.
The recent online publication of findings from the University of Southern California ataxia working group called Enigma served to fuel more interest in the simmering drug development space of Friederichs’s ataxia (FRDA), where a handful of gene therapies and other approaches, plus one promising small-molecule treatment, are in the works.
Springworks Therapeutics Inc. plans to file an NDA for its lead drug candidate, nirogacestat, for treating desmoid tumors later this year, as top-line data from a pivotal phase III trial indicate that it provided patients with a substantial progression-free survival benefit.
Just in case the U.S. FDA didn’t get the message from its advisory committee about drug applications based solely on clinical trial data from China, a trio of U.S. lawmakers wrote to FDA Commissioner Robert Califf to voice their concerns about the “current ‘East to West’ movement of clinical data” to support the approval of me-too drugs.
Concert Pharmaceuticals Inc. is on track for an NDA filing for CTP-543 in alopecia areata during the first half of 2023, following a positive read-out from the first of two phase III trials of the oral Janus kinase 1/2 inhibitor. The molecule hit the primary endpoint of the placebo-controlled Thrive-AA1 study, which was defined as the percentage of patients achieving a SALT (Severity of Alopecia Assessment Tool) score of 20 or less after 24 weeks of therapy.
Idorsia Ltd. looks on course to produce another marketed drug after supportive phase III results for its hypertension drug, aprocitentan. The company’s first U.S. FDA-approved drug, Quviviq (daridorexant), was launched in April and another product, Pivlaz (clazosentan), was approved and launched in April for cerebral vasospasm in Japan.
In the wake of a patient’s death, the U.S. FDA has placed a partial clinical hold on Foghorn Therapeutics Inc.’s phase I study of FHD-286 in treating relapsed and/or refractory acute myelogenous leukemia (r/r AML) and myelodysplastic syndrome.
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