Mesoblast, a biotech company based in Melbourne, Australia, is making determined strides toward securing FDA approval for its off-the-shelf therapy, remestemcel-L, as a treatment for steroid-refractory acute graft-versus-host disease (aGVHD). Despite two prior unsuccessful attempts, the biotech is now embarking on a new strategy following a meeting with the FDA that has left it convinced of a viable path to market.
Previously, Mesoblast faced rejection when seeking approval for pediatric use in 2020 and a subsequent attempt this year without conducting a new trial. The FDA’s complete response letter last month prompted Mesoblast to outline fresh plans, expressing optimism about gaining approval through additional potency assay data or new clinical evidence in adults.
After its meeting with the FDA, Mesoblast now has a clear roadmap to follow. Two crucial tasks lie ahead. Firstly, Mesoblast intends to generate new potency assay data to demonstrate the standardization of the product’s identity, strength, quality, purity, and dosage form, all tested in the pediatric phase 3 trial. Potency assays have been a persistent concern, with Mesoblast recognizing the need for refinement following its initial rejection. While improvements were acknowledged in the second rejection, these tests have remained a hurdle to approval. Mesoblast asserts that resolving the potency issue is essential for the FDA to consider the pediatric phase 3 trial as a suitable basis for approval.
The second imperative task is conducting a clinical trial involving adults. Three years after the FDA’s request for another study, Mesoblast has outlined plans for a single-arm trial encompassing adults and children aged 12 and above. This trial will enroll patients who have not responded to both steroids and a second-line agent, such as ruxolitinib. The trial will be supported by an appropriate potency assay, according to Mesoblast.
While an agreement with the FDA regarding the trial specifics has not been reached yet, Mesoblast notes the agency’s willingness to consider the proposed trial design, contingent upon the suitability of the potency assay. The biotech believes it can fund the trial costs through cost-cutting measures implemented last month, including a 30% reduction in the CEO’s pay.
Investors have responded positively to these developments, pushing Mesoblast’s shares up by 6% to $1.39 in premarket trading. However, it’s important to note that the stock still remains significantly below its value of over $4 prior to the second FDA rejection. Mesoblast remains committed to addressing these challenges and advancing remestemcel-L toward potential approval.