Hello ALL,
Update from Lodge Partners attached in regards to upcoming Adcom / ODAC process
In my opinion only, I give Lodge a fair bit of weight as the inaugural Brokers who IPO’d Mesoblast back in December 2004 and have continued to maintain their research and deep analysis into MSB.
Good Luck to ALL Holders !
Anjo-roch
>>>>>>>>>>>>>>>>>>>>>>>>>>>>>>>>>>>>
LODGE PARTNERS
RESEARCH UPDATE
Friday 31 July, 2020
< MesoblastLimited (ASX: MSB) >
RYONCILTMRoad to FDA Approval
Background: About a week ago, Mesoblast (MSB) announced a very important date in terms
of gaining approval for its first US product. The announcement was as expected, and the
precise date is relatively unimportant. The announcement and date, however, do set off a
relatively defined series of events that we believe give MSB about a 95% chance of receiving
US Food and Drug Administration (FDA) approval for their first product in that key territory
(you all know we never give anything a 100% chance of success). What follows is a
description of the events that are likely to occur, what you should be looking for and a few
traps you don’t want to fall into.
Announcement: RYONCIL™ Advisory Committee Date Set: Mesoblast’s RYONCIL™
(remestemcel-L) for paediatric steroid-resistance acute graft versus host disease (paediatric
SR-aGvHD) has had it advisory committee (AdCom) meeting date for the 13th of August 2020
by the Oncologic Drugs Advisory Committee (ODAC). Given the nature of MSB’s drug, it
would have been a much, much, much bigger worry had RYONCIL™ not been asked to
present at AdCom. The first of any new class medicines, no matter how overwhelming the
data behind it, is subjected to what is called an advisory committee meeting. If RYONCIL™
had been passed over for an AdCom, it would have almost certainly meant the FDA had
determined RYONCIL™ was “Not Approvable”. Not approvable is a nice way of saying your
product has been rejected, but you are free to do what we tell you and maybe we will
approve it the next time around.
ODAC is the FDA’s key player in the regulation of cancer drugs and it plays a very, very, very
big say on whether a cancer drug gets approved or not.
AdCom’s are straight forward. They run over a day (normally) and, generally, go like this:
1. MSB puts forward its argument as to why the drug, remestemcel-L, should be approved,
based on the data in the application MSB originally submitted (the Biologics Licence
Application (BLA)),
2. The ODA is then given the chance to say what they like and what they do not like about
the BLA,
3. Groups of interested parties are then give a chance to speak (often these interested
parties are those who form a support group for patients who might benefit from the
drug in question).
4. There are also 6 to 12 or so “Independent Experts”, who have been invited by ODAC to
review the BLA in question. They then get to have their say. The experts, we will call
them from here on, then get to talk about the results and ask questions of MSB and
ODAC. The big excitement of the meeting then follows. The experts must answer
questions ODAC has put to them prior to the meeting. Some of these questions are non-
voting questions, where the experts simply provide comments/opinions. Here ODAC is
collecting information for the remainder of the RYONCIL™ overall BLA review, they may
be trying to influence the experts one way or another on the review at hand, or for
information for to use when similar products come up before ODAC. Then come the BIG
questions. These are the yes/no questions on which the experts vote. The biggest
question that is almost always asked is:
“Has the sponsor(MSB) provided substantial evidence that the use of RYONCIL™ to
treat paediatricSR-aGvHD is both safe and efficacious?”
The results are then tabulated and displayed publicly. Obviously, the higher the number
of ‘yes” votes, the better for the sponsor. If a majority of the experts present at the
meeting vote yes, the entire vote is considered to be a “YES” vote, at least, in the eyes
of industry and the public. I imagine with this meeting, though, the experts will vote yes
in unison.
One thing everyone needs to understand is that the FDA will drop its BRIEFING NOTES for the RYONCIL™
AdCom no later than two (2) business days before the meeting (remember the Australian time difference).
These notes are extensive and will give you a very good idea about how things will go for RYONCIL™ during
(and after) the AdCom. They also contain the questions the experts will be asked to answer. The briefing
documents can likely be found here or here. I will try to download them and send them to everybody as soon
as I can, with a comment on them to follow shortly thereafter.
Basically, that is it until the Prescription Drug User Fee Act (PDUFA) date for RYONCIL™. The PDUFA date is
the date by which the FDA is mandated to have either “Approved” a drug or stated that the drug is “Not
Approvable” in its current form.
Note, while PDUFA dates are mandated, there enforcement is not strict. Based on everything RYONCIL™ has
going for it, I imagine the therapy will be approved before the PDUFA date. Having said that, with SARS-CoV-
2 floating round, anything could happen in terms of timing.
One thing to keep mind, the AdCom votes on exactly what is before it. It may not like one aspect of the drug in
question. If that aspect can be ameliorated by a simple labelling change, the change will be made, and the
drug allowed through, even if the AdCom originally voted “no”. The same can happen on the FDA’s end. The
point, being there is a lot that can happen between the AdCom vote and the FDA’s final decision. Therefore, it
is not uncommon for the FDA to approve drugs an AdCom has said no to and vice versa.
Conclusion: The path to approval for RYONCIL™ is now set. It just needs to get to the finish line.
We have no doubt it will get there. Strong Buy maintained.
(20min delay)
