Locations@DocMcstuffins I only looked up the 180day participants...

Locations

@DocMcstuffins

I only looked up the 180day participants in GVHD001 but the list of original participating hospitals is pretty impressive from the Phase 3 trial . Virtually all the main teaching hospitals accounting for 50% of all cases in paediatric sr aGVHD are very familiar with Ryoncil and should be covered in the first wave of the rollout . As acute steroid refractory GVHD is an unmet need I am struggling to understand what choice doctors have right now for the under 12s... In one of the Reach trials I recall Rux had only 4 patients treated in the under 18 category. Anyone with a brain cell can read up on Reach 1 &2 and see that Ruxolitinib struggles to show any real efficacy in the severe grades of C/D disease . I don't suppose Doc you noticed the percentage of patients that also had to discontinue Rux during the Reach trials owing to toxicity, side effects, etc.

The elephant in the room is that apart from Jakafi , most therapies for GVHD disease rely on blanket immunosuppression…which leads to very poor overall survival outcomes . There may be a case for still using Jakafi in adult for Grade B disease where the response is reasonable and maybe ECP for skin is another alternative, but I believe we trounce all comers in GI/Liver and multi organ disease categories.
Mesoblast has already guided that it will implementing a “stepped roll out”for Ryoncil …but the inventory is already there …and I think treating 241 patients in the EAP, shows that it is not that difficult to find treatment refractory patients when other therapies halve failed.
The Company has plenty of stock ready for distribution…and I think they will be a prime candidate for a Phase 4 trial in adult…it would be plausible to think they may seek accelerated approval for adult whilst committing to a 70-80 patient trial …alternatively they may need do a head to head RCT against Rux with a 180 day endpoint (28 day surrogates are less reliable) After all,seldom are companies approved for a paediatric therapy then prevented by the FDA from being used off label in adults. I am sure you appreciate from J Kurtzberg et al, that paediatric patients with this disease struggle to take pills (Rux is taken by mouth) as opposed to intravenously (Ryoncil) because of extreme fatigue and vomiting …Mesoblast knows from recent clinical data it has similar efficacy in adult patients too. I think your “concerns” are pretty lame !

It’s pretty obvious that many shorters are preparing to change tack in anticipation of a successful BLA application. Eric Rose has already suggested that GVHD will ultimately (post adult approval which is 4X the size) be a $1bn market opportunity…but to be confident we just need confirmation of the reimbursement charges …Nothing comes close to Ryoncil for long term durability of response in severe categories. Failure to prescribe would be akin to incompetence in my opinion. I expect off label sales in adult to build up quite quickly . OP


https://classic.clinicaltrials.gov/ct2/show/NCT02652130
Participating Hospitals in GVHD001

United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
CHOC Children's Hospital of Orange County
Orange, California, United States, 92868
UCSF Benioff Children's Hospital
San Francisco, California, United States, 94143
United States, Colorado
Children's Hospital Colorado Center for Cancer/Blood Disorders
Aurora, Colorado, United States, 80045
United States, Delaware
Alfred I. duPont Hospital for Children of the Nemours Foundation
Wilmington, Delaware, United States, 19803
United States, Florida
Miami Children's Research Institute
Miami, Florida, United States, 33155
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Michigan
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
United States, Missouri
Washington University
Saint Louis, Missouri, United States, 63110
United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10174
The Children's Hospital at Montefiore
New York, New York, United States, 10467
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27705
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
United States, Texas
Texas Transplant Institute
San Antonio, Texas, United States, 78229
United States, Virginia
Virginia Commonwealth University
Richmond, Virginia, United States, 23284
United States, Washington
Fred Hutchinson Cancer Center
Seattle, Washington, United States, 98109
United States, Wisconsin
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226

Please do not rely on the opinions or facts contained in the above post when making an investment decision.