@Pledge"
Poor management doesn't get unwritten by FDA approval. "
No. Poor management
doesn't deliver anything, much less an eventual FDA approval.
I'd say this management has delivered so much in the process of leading up to first-in-class approval that it would almost beggar belief i.e. in any reasonable person. Just the cost savings on the lifetime of clinical trials PI through PIII across
5indications, at least 2 of which are chronic diseases of blockbuster proportions, are staggering. The fact that Mesoblast Ltd IP ownership has been preserved throughout the development process is also tribute to astute management. IMO no further evidence is required.
It's a question of timing, that's all.
Hover, if you don't want to do the math on how much clinical trials cost, here's something to maybe take that foul and bitter taste out of your mouth, though I wouldn't bet on it.
Fact: The gold standard of recognition that the US FDA can provide to a biotech is a Regenerative Medicine Advanced Therapy (
RMAT) designation for a product/ indication. It grants all the benefits of
Breakthrough Therapy designation + all the benefits of Fast Track designation. These include:
- early interactions to discuss any potential surrogate or intermediate endpoints
- FDA statute addresses "
potential ways to support accelerated approval and satisfy post-approval requirements" (sound familiar?)
- priority review
- rolling review
- other actions to expedite development and review
- intensive guidance on efficient drug development, beginning as early as Phase 1
- FDA's organizational commitment, involving senior managers
Read this:
https://www.fda.gov/media/120267/downloadYou can't buy these benefits. Every biotech in the world would like to do that.
Fact: To get an RMAT any biotech in the world has to demonstrate to the FDA (who are not our friends) that a drug is a regenerative medicine therapy,
ANDthe drug is intended to treat, modify, reverse, or cure a serious condition,
AND demonstrate preliminary clinical evidence tht indicates that the drug has the potential to address unmet medical needs for such disease or condition.
That 'preliminary clinical evidence'has to be
Fact: In the world of cellular, gene & tissue (CGT) therapies, there have only ever been 101 RMATs issued by the FDA since the dawn of human history i.e. since the beginning of 2017.
Fact:
Mesoblast Limited holds 2 of those. And
Takeda holds another (for Alofisel under licence) i.e. for a product/ processes which it exploits under license from Mesoblast Ltd. Only one other company in the world holds or licenses more RMAT indications than Mesoblast Ltd i.e. 4 RMATs.
Fact: Mesoblast didn't get given its RMATs by its dad, or buy them from Osiris. And they're not for remestemcel-l. They're for rexlemestrocel-l, the product which was
only ever conceived and developed by Mesoblast Ltd management.
Fact:
Both Mesoblast's directly-held RMATs are before the US FDA
right now for pre-BLA consideration or have been there recently for confirmatory trial agreement and approval, & are not an IND sitting on someone's dusty shelf somewhere in the USA.
Anyone's proper research will prove these facts. That's good management in my books.
Tell you what. Let's discuss it at the 2024 AGM, where you can ask whoever is the CEO any questions you like.
GLTA(LT)H
(20min delay)
