FDA approves Blueprint’s pralsetinib in Metastatic RET Fusion-Positive NSCLC

GAVRETO is a once-daily oral RET-targeted therapy developed by Blueprint Medicines. It is designed to selectively and potently inhibit RET alterations that drive many cancer types, including approximately 1 to 2 percent of patients with NSCLC. Currently, RET is one of seven NSCLC biomarkers that can be targeted with an FDA-approved therapy.

GAVRETO was granted accelerated approval by the FDA, and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. In 87 patients previously treated with platinum-based chemotherapy, the overall response rate (ORR) was 57 percent (95% CI: 46%, 68%) with a 5.7 percent complete response (CR) rate, and the median duration of response (DOR) was not estimable (95% CI: 15.2 months, not estimable). In 27 treatment-naïve patients who were ineligible for platinum-based chemotherapy per the study protocol, the ORR was 70 percent (95% CI: 50%, 86%) with an 11 percent CR rate, and the median DOR was 9.0 months (95% CI: 6.3 months, not estimable). GAVRETO has warnings and precautions of interstitial lung disease/pneumonitis, hypertension, hepatotoxicity, hemorrhagic events, risk of impaired wound healing and risk of embryo-fetal toxicity.

GAVRETO is the second breakthrough therapy discovered by Blueprint Medicines that has received FDA approval in 2020, less than 10 years since the company started operations.

Blueprint Medicines and Genentech plan to make GAVRETO available in the U.S. within one week. GAVRETO will be available in a 100 mg dose strength, and the recommended starting dose is 400 mg once daily. 

DSMB recommends continuation of ph 3 trial of Meso’s remestemcel-L in ARDS

The multi-center study includes three interim analyses for stopping accrual early for efficacy or futility when 30%, 45% and 60% of the total target of randomized patients have reached the primary endpoint. Up to 300 patients are planned to be randomized 1:1 in the double-blinded Phase 3 trial to receive either two intravenous infusions of remestemcel-L within five days, or placebo, on top of maximal care. The primary endpoint is all-cause mortality within 30 days of randomization. The key secondary endpoint is days alive off mechanical ventilatory support within 60 days of randomization. The trial is expected to complete recruitment during 4Q-2020.

Mesoblast’s lead allogeneic cell therapy product candidate, remestemcel-L, is an investigational therapy comprising culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. Remestemcel-L is believed to have immunomodulatory properties to counteract the cytokine storms that are implicated in various inflammatory conditions by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.