Words that count from the individual with the most skin in the gameDespite a first product approved for adults with SR-aGVHD two years ago, survival outcomes for children or adults with the most
severe forms of SR-aGVHD have not improved over the past two decades and remain poor. The lack of any approved treatments
for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes in children.
Over the past twelve months, the Company has generated substantial new information on clinical and potency assay items
identified in the Complete Response Letter (CRL) received from FDA in September 2020 to the BLA for remestemcel-L in the
treatment of children with SR-aGVHD. Since receiving the CRL, we have maintained an active dialog with the FDA.
Toward the end of last year, we held a meeting with the FDA’s Office of Tissues and Advanced Therapies (OTAT) to address potency
assay and chemistry, manufacturing and controls (CMC) items identified in the CRL where OTAT indicated that Mesoblast’s
approach to address the outstanding CMC items is reasonable.
Most recently, as guided by FDA, Mesoblast submitted to the
Investigational New Drug (IND) file for remestemcel-L in the treatment of children with SR-aGVHD substantial new information
it has generated in response to the CRL.This represents a major milestone in the Company’s complete response to the FDA. The
submission summarizes controlled clinical data providing further evidence of remestemcel-L as a potential therapy in SR-aGVHD.
Additionally, the improved process controls we have put in place to assure robust and consistent commercial product, together
with a potency assay that predicts consistent survival outcomes, makes a compelling case for remestemcel-L as a viable
treatment for these children.
The 12-month update on survival outcomes from the randomized controlled trial of remestemcel-L in ventilator-dependent COVID19 patients with moderate/severe acute respiratory distress syndrome (ARDS) were released showing the early survival outcomes in the remestemcel-L group relative to controls were maintained at later timepoints in those under age 65, with a 42% reduction in mortality through 12 months and with continued
observed synergy with dexamethasone. Mesoblast is working under a Memorandum of Understanding (MOU) with Vanderbilt
University Medical Center (VUMC) to collaborate on the design and execution of a second COVID-19 trial for remestemcel-L.
VUMC coordinates and works closely with a clinical trial network of investigators at over 40 sites across the US focused on
studying ARDS and other critical illnesses, and will be jointly developing with us a trial protocol to confirm the observed
reduction in mortality in COVID-19 ARDS patients under 65 years of age in the earlier study.
Lastly on the remestemcel-L platform, an investigator-initiated randomized, controlled study is underway in patients with
medically refractory ulcerative colitis or Crohn’s colitis, with delivery of remestemcel-L made directly to areas of inflammation
via an endoscope.
The first 12-patient cohort in this study showed rapid mucosal healing and disease remission compared
to placebo in refractory patients at high risk of progression to surgery and we look forward to receiving outcomes from
additional patients in the study.
From the bloke above's BossToday we announced long-term survival results for our lead product remestemcel-L in children with severe steroid-refractory acute graft-versus-host disease (SR-aGVHD), a devastating disease with high mortality. The results showed durable survival through 4 years of follow-up reaffirming the
potential significance of remestemcel-L as new life-saving treatment for children with SR-aGVHD where to date there are no other approved therapies. These new long-term survival data are a key component of the Company’s BLA resubmission to the United States Food and Drug Administration
(FDA).
These long-term survival results follow our filing with FDA in October of substantial new information on clinical and potency assay items which they requested in their Complete Response Letter from 2020,and will be formally submitted to FDA.
CMC items identified in the CRL appear to have been covered in the October 2022 update to the IND file. That announcement was price sensitive and I imagine contained all the goodies which widen our moat to any competition.
Further proof of product efficacy, as required by the CRL, was made available from Real World 4-year survival data.
Now , back to the first bloke
"I am of the firm belief that the work we have put into the BLA resubmission will significantly raise the bar in terms of industry standards and ultimately be a competitive advantage for the Company. Even more importantly,
our hard work has served to further enhance the understanding of the mechanism of our cell therapy, to generate additional clinical data further demonstrating the relationship between potency and survival in the most severe cases in children who have the highest mortality rates from this dreadful disease, and set the Company up for the best chance of success in gaining our first and subsequent product approvals".
What I love about our CEO is that he openly admits he and his team are learning as they go, that is a far cry from serial protagonists on these threads who presume to know it all.
If this is approved then our pipeline across Chronic Heart Failure, Chronic Lower Back Pain, All Cause Ards and multiple inflammatory diseases put us squarely in the sights of every major pharmaceutical company out there. Check out
@Pharmagang's recent post if you missed it.
Something monumental about to happen, shortly
Reg
(20min delay)