Ann: FDA Notifies Clinical Data Sufficient for Refiling aGVHD BLA, page-725

Initial BLA submission- Pre end of the world covid pandemic- Company share price circa $3.00 536,000,000 shares on issue 1.6 Billion valuation

We are close to the 2020 valuation, another 20 cents to go

Talk about being knocked around by Covid and a couple of Complete Response Letters ( fair enough).

The significance of directors buying on market recently should not be underestimated- nil purchased 2019.

Straight after the initial BLA submission we get told it works in Chronic - off label sales anyone?

	20 February 2020	Positive Outcomes Using Remestemcel-L in Chronic GVHD

Medical professionals vote that Ryoncil is safe and efficacious

	14 August 2020	ODAC Votes in Favor of Remestemcel-L for GvHD

Professor Kurtzberg for the win , linking in vivo biomarkers to good old survival outcomes

	07 December 2020	Remestemcel-L Reduces Biomarkers for Mortality in SR-aGVHD

Pipeline product durable three year pain reduction results

	12 January 2022	36-Month Results of Phase 3 Trial in Chronic Low Back Pain

Novartis would not have been impressed with this one

	23 November 2022	Long-Term Survival for Acute GvHD Treatment with Remestemcel

Pipeline for back pain gets a leg up

	09 February 2023	FDA Grants RMAT Designation for Rexlemestrocel-L in CLBP

CHF results published

	28 February 2023	DREAM HF Trial Published in JACC

Notified no form 483 issued so Ryoncil manufacturing is signed off by the FDA - how is the price lower than 2020???????????????????????

	26 May 2023	MSB Reports Q3 Financial Results and Operational Highlights

Possible 100 million dollar voucher - that will be nice to pay for stock manufacturing

	19 January 2024	FDA Grants Rare Pediatric Disease Designation for Revascor

100% successful- small trial- but we are definitely on the FDA radar

	15 February 2024	FDA Grants Orphan Drug Designation for Revascor

Probably the biggest highlight on this page- pathway to Global Partnership- my view

	11 March 2024	FDA Supports Accelerated Approval Pathway for Heart Failure

Back to refiling the BLA

	26 March 2024	FDA Notifies Clinical Data Sufficient for Refiling aGVHD BLA

After 4 years of setbacks we are back on track.

For the newbies, the pipeline has not increased in valuation- Ryoncil has been substantially derisked with manufacturing sign off, survival results and apparent agreement on potency related issues to allow for refiling of the SR aGvHD pediatric BLA.

The hype was either parabolic back in Feb 2020 or the market is pricing it wrong today.

I'm biased as am a holder. Still, I review, recheck, drink and sleep.

My mind is running a repetitive loop.

Phil Krause saying trial showed efficacy - need to prove potency, @JB1975 saying issues with small n and too many confounding variables like different medical professionals administering different treatments ( sorry jb my understanding- not direct quotes), Si saying we couldn't do a new trial until potency assays approved. New trial being partnered with CIBMTR, advised funded etc. Then the company meets with the FDA and reverts to original plan. Asefficacy was signalled in pediatric, then yes, it is unethical to run another pediatric trial.

To stop the loop

My mind says - if efficacy is proven, then JB's points re small n and confounding variables are no longer valid , in terms of getting approved.
If the adult trial is on hold, then there is agreement that the 001 pediatric data may be enough to get approval.

Potency must be proven,imo, in the 001 trial product- as no further trials ( review process) were to occur until agreement or else why would the review be back on?

The announcement to refile specifically said after additional consideration of the data from the 001 study- not any other part of the BLA submission.


Happy to have a conversation re my loopiness- please point out faults in my supposed logic- might have a few bucks on the line here


How are we under Feb 2020 valuation?



Reg (The Bemused)