Ann: Quarterly Activities/Appendix 4C Cash Flow Report, page-178

I was listening to the Sarepta conference call live tonight. They had downgraded revenue guidance owing to a number of factors such as the worst flu season for 15 years. One patient died after receiving ELEVIDYS. They had elevated liver enzymes after receiving their gene therapy. After this tragic adverse event, treatment cycles were extended by a further one and a half months while physicians considered the safety implications of this major adverse event. During the Q&A, Serepta were asked about the impact of the recent FDA redundancy programme and they said that they had seen nochange in regulatory response to any of their applications in recent weeks and they were working well with Nicole Verdun…(just like Mesoblast) !
When you look into the areas targeted for FDA cutbacks I have seen no mention of any RMAT designated programmes , although they were appearing to want to penalise some Columbia University research and some woke research projects for political reasons.

There is an awful lot of noise incoming. Trump continues to make puzzling statements. He is sounding off about tariffs , increases in inspection fees on overseas manufacturing facilities and the strategic need to promote onshoring. There is even a suggestion about trying to reduce Pharma prices to the same costs as other developed nations. Despite the fact that Ryoncil is based in Singapore which has a FTA (free trade agreement ) with the USA, there is always the risk that he will interfere by introducing some new rules on imports to into the USA . The Pharma industry wields massive power in the USA and orphan designation therapies have managed to be protected by a “carve out exemption” for first line approvals from the recent Bidens Inflation Reduction Act.
Mesoblast has been evaluating manufacturing options in the USA for some time..it has the Lonza site in Houston as a backup solution, but alternatively, all they need to do is find an approved GMP cell manufacturing site in the USA which has already completed an recent inspection.

The just anointed new head of CBER, is known to want to see rigorous controlled clinical trials which will put obstacles in the way of many of our competitors seeking to gain approval based around Phase 2 trials using flimsy surrogate or non mortality endpoints . Mesoblast already has full approval for Ryoncil so filing label extensions represent a much easier route to market for additional indications.

I suspect sales of Ryoncil for GVHD are now starting to build as payer coverage expands.. Achieving a weekly patient treated run rate of two per week by the end of the first twelve months looks sensible and achievable …this number should double the following year to represent over 200 Sr aGHVD patients plus adult and off label . If these numbers are realistic , the shares should attract a very high pe ratio as a first in class therapy…
It is now a question of time before the market wakes up to progress …Silviu needs to get out and communicate the amazing Ryoncil progress with investors. In the meantime the shorters have sold an average almost 800k of MSB stock everyworking day for the last threemonths . At some stage MSB’s share price will have to be upgraded massively based on core revenues. OP



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