Syros Pharmaceuticals Inc. (NASDAQ:SYRS) experienced a significant setback as its Phase III trial, Select-MDS-1, evaluating tamibarotene in patients with myelodysplastic syndrome (MDS) who have RARA gene overexpression, failed to meet its primary endpoint. The trial's outcome led to an 86% plunge in the company's stock value.
The Select-MDS-1 trial assessed the efficacy of the oral, selective, retinoic acid receptor alpha agonist, tamibarotene, in combination with azacitidine. Among the first 190 patients enrolled, the intent-to-treat analysis revealed a complete response (CR) rate of 23.8% in the tamibarotene plus azacitidine arm, compared to 18.8% in the placebo plus azacitidine arm. The difference was not statistically significant, yielding a p-value of 0.2084.
"We are sifting the data and evaluating next steps," said a Syros spokesperson. The company, based in Cambridge, Mass., is now undertaking a thorough review of the trial data to determine the future direction of the tamibarotene program in MDS.
Adaptimmune's Lete-cel Shows Promise in Sarcomas
Adaptimmune Therapeutics plc reported positive results from a pivotal study for its second candidate, lete-cel, with plans for a rolling BLA filing in 2025. The primary analysis from 64 patients with synovial sarcoma or myxoid/round cell liposarcoma who received previous anthracycline-based therapy showed treatment with lete-cel produced RECISTv1.1 responses in 42% of patients, with six complete responses and 21 partial responses. Lete-cel, or letetresgene autoleucel, is an engineered TCR T-cell therapy targeting the solid tumor antigen NY-ESO-1 and comes from the same platform that produced Tecelra (afamitresgene autoleucel), a T-cell therapy targeting MAGE-A4 that won accelerated approval in August for treating advanced synovial sarcoma.
Daiichi Sankyo and Astrazeneca Update BLA Submission for Trop2 Lung Cancer ADC
Daiichi Sankyo Co. Ltd. and Astrazeneca plc have submitted a new BLA to the U.S. FDA for accelerated approval for datopotamab deruxtecan (dato-dxd) for treating locally advanced or metastatic epidermal growth factor receptor-mutated (EGFR-mutated) non-small-cell lung cancer (NSCLC) patients who have received prior systemic therapies. The companies have voluntarily withdrawn their dato-dxd BLA in the U.S. for patients with advanced or metastatic nonsquamous NSCLC based on the Tropion-Lung01 phase III trial. The decision to submit a new BLA for EGFR-mutated NSCLC and withdraw the previously submitted BLA for nonsquamous NSCLC was informed by feedback from the FDA, the companies said.
Apollo and Sunshine Lake Pharma Ink Deal for FGF21/GLP-1 Agonist
Apollo Therapeutics Group Ltd. and Sunshine Lake Pharma Co. Ltd. have entered into a licensing agreement potentially worth $938 million for APL-18881 (HEC-88473), a dual fibroblast growth factor 21 (FGF21)/glucagon-like peptide-1 (GLP-1) receptor agonist developed by Sunshine Lake Pharma. The drug is currently in Phase II clinical trials for type 2 diabetes. Apollo will gain global rights, excluding China, to develop and commercialize APL-18881.
