NEW YORK – Ellipses Pharma this week said the US Food and Drug Administration has granted orphan drug designation to its dual FLT3 and Aurora kinase inhibitor EP0042, which it is developing for patients with treatment-resistant acute myeloid leukemia. The agency earlier this year approved the London-based firm's investigational new drug application, allowing for the expansion of a Phase I/II trial of EP0042 into the US. The firm plans to enroll 63 AML patients, including 20 patients with FLT3 internal tandem duplication-mutant AML and 10 patients with FLT3 wild-type AML. The FDA grants orphan designation to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval.
Perspective Therapeutics subsidiary Viewpoint Molecular Targeting this week inked a deal with the contract development and manufacturing organization PharmaLogic to develop and produce the radiopharmaceutical candidates VMT-01 and VMT-α-NET. Viewpoint is developing the targeted alpha therapies for certain patients with metastatic melanoma and neuroendocrine tumors, respectively. Under the terms of the agreement, PharmaLogic will produce and supply doses of both VMT-01 and VMT-α-NET for early-phase clinical trials.
Exscientia this week unveiled two development candidates in preclinical studies: EXS74539, an LSD1 inhibitor, and EXS73565, a MALT1 protease inhibitor. The firm said investigational new drug (IND) applications-enabling studies are ongoing for both candidates, and it plans to provide an update in clinical development plans in both hematological cancers and solid tumors in the second half of the year. Both candidates were funded through a 2019 collaboration with Celgene, which was taken over by Bristol Myers Squibb in the acquisition. However, BMS's options to the candidates have now lapsed, and Exscientia maintains all worldwide rights to both compounds.
Deciphera Pharmaceuticals said this week that the US Food and Drug Administration has granted breakthrough therapy designation to the investigational switch-control inhibitor Qinlock (ripretinib) as a treatment for patients with advanced gastrointestinal stromal tumors (GIST) whose cancers harbor a KIT exon 11 mutation and co-occurring KIT exon 17 and/or 18 mutations. The FDA based the designation on the results of the INTRIGUE Phase III study, in which Qinlock clinically benefited this biomarker-defined second-line GIST patient population. The FDA grants breakthrough therapy designation to expedite the development and review of drugs intended to treat serious conditions when early evidence suggests a clinical benefit over available therapies on a significant endpoint.
Servier said this week that the Phase III INDIGO trial of vorasidenib in patients with IDH1- or IDH2- mutant low-grade glioma met its primary endpoint of progression-free survival and its secondary endpoint of time to next intervention. These results were statistically significant and clinically meaningful. The company said it is now working on regulatory filing timelines and vorasidenib supply capacity.
Leucid Bio said this week that it signed an agreement with Great Ormond Street Hospital NHS Foundation Trust to manufacture its lead CAR T-Cell candidate LEU011 for use in human studies. LEU011 targets NKG2D ligands. Leucid plans to file a clinical trial application with the intention to begin a trial in the second half of 2023 in patients with solid tumors and hematological malignancies.
The Lung Cancer Research Foundation, in collaboration with Daiichi Sankyo and AstraZeneca, said this week that they will fund up to three grants supporting research on antibody-drug conjugates (ADCs). The grants will back researchers exploring the mechanism of action for TROP2-directed ADCs in lung cancer and HER2-directed ADCs in non-small cell lung cancer, as well as primary and acquired resistance mechanisms to both categories of ADCs. LCRF is also interested in research on the discovery and validation of predictive biomarkers of TROP2-directed or HER2-directed ADC efficacy in lung cancer; the prevalence of prognostic or predictive biomarkers from lung cancer cells or blood; the co-expression of biomarkers with other known biomarkers; and the evolution of biomarkers before, during, and after standard treatment.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Oncology News.