FDA ODAC Meeting Material discussion analysis, page-431

Who’s going to be in bed early and up at dawn to check how MESO went on the NASDAQ and what the outcome was of the ODAC / FDA discussion?

4 things stood out for me in the 127 page remestemcel-L ODAC / FDA briefing doc:

1. IS IT SAFE? Yes. In summary, remestemcel-L was well tolerated with a favorable safety profile. Safety of 1,100 patients across the development program was investigated (source p.60 https://www.fda.gov/media/140996/download)
2. DOES IT WORK? Yes. In summary, the remestemcel-L development program demonstrated significant and clinically meaningful efficacy for the treatment of paediatric patients with SR-aGVHD (source p.51 https://www.fda.gov/media/140996/download)
3. WHAT IS THE BENEFIT / RISK? Children with SR-aGVHD represent a highly vulnerable patient population who desperately need an effective treatment option with a favorable safety profile. SR-aGVHD has a high risk of treatment failure and mortality, particularly in those with severe GVHD. Currently, there are no approved treatment options for children under 12 years of age. For children 12 and older, currently available treatments have limited efficacy, particularly in the most severe patients with Grades C and D disease, and these treatments are associated with significant toxicities. In contrast to currently used treatments, remestemcel-L has shown consistent benefit across disease severity, including severe disease, with a favorable safety profile. In the pivotal Phase 3 study, Study GVHD001, there was a high OR at Day 28 of 70% and a consistently high OR in patients with Grades C and D disease of 73%. Importantly, survival to Day 180 was 69%. The efficacy and safety of remestemcel-L for patients with SR-aGVHD are supported by results from EAP 275, which showed clinically meaningful efficacy in a highly refractory real-world population, including patients with severe Grade C or D disease who failed to respond to multiple lines of standard of care. Regarding risk, remestemcel-L has a well-tolerated safety profile that is highly favorable compared to standard therapies, including current treatment and recently approved therapy. The most commonly reported AEs in remestemcel-L studies were expected in the pediatric SR-aGVHD immunocompromised population and were not considered to be associated with remestemcel-L in the majority of cases. In conclusion, the totality of evidence demonstrates remestemcel-L’s highly favorable benefit- risk profile, supporting approval of remestemcel-L for the treatment of pediatric patients with SR-aGVHD. (source p.63 https://www.fda.gov/media/140996/download)
4. CAN YOU MAKE IT SAFELY AND EFFECTIVELY? Yes. The remestemcel-L manufacturing process has been developed to produce a safe, effective, and consistent product. Over development relevant quality attributes have been established that are representative of product characteristics for identity, purity, safety, and efficacy. In conclusion, remestemcel-L has a well-established and robust manufacturing process which produces a product that consistently meets product quality attributes. (source p.35 https://www.fda.gov/media/140996/download)

GLTAH - See you tomorrow

Moose