MSB CC Q&A 29 Aug 24

Q & A Section of Mesoblast Conference Call 29 August, 2024

Enjoy



Operator

Thank you. [Operator Instructions]

And today's first question comes from Louise Chen with Cantor. Please go ahead.


Unidentified Analyst


Hi. Good evening, everyone. This is Karby [ph] on for Louise from Cantor. Thank you for taking your questions. First, can you discuss any potential partnerships or collaboration that could accelerate your commercialization effort?


And second, how are you planning on tackling reimbursement challenges, assuming you get approval for RYONCIL? Thank you so much.


Silviu Itescu


Thank you. Well, so RYONCIL for pediatric and adult GVHD, we have a go-to-market strategy on our own, because we've already put a lot of effort into doing so. The patient population is relatively targeted and the transplant centers that perform these transplants are relatively small in number. So highly manageable commercialization process.


We are in discussions with payers, and we have a very good sense of what the reimbursement is likely to be in this space, based on two important considerations. One is that the recent approvals of CAR T therapies, particularly in children with leukemia, we represent a similar population as the children we're targeting with GVHD, provides at least a level of basis for comparison.


Secondly, we have five-year outcomes which we've talked to and have provided to the FDA last year. Five-year outcomes from our Phase 3 trial demonstrating a 50% overall survival for between four to five years, meaning that at least 50% of the children are cured of this disease. And that puts the treatment paradigm into the realm of genetic diseases, where again, intense therapy at the front end provides a substantial number of patients with curative outcomes. And so, when one thinks of reimbursement, given the orphan size of the population and given the precedence in both CAR T therapies and gene therapy, we think that the reimbursement is going to fall somewhere between those parameters.


The second question I think you asked me was on partnering. And so, it really depends on which products we talk about. For our back pain product, we already have a commercialization partner in Europe, that's Grunenthal, which is Europe's number one pharmaceutical company in the pain space. The relationship with Grunenthal is robust. On successful completion of this Phase 3 trial, they will take on the responsibility of market access, pricing and distribution, and then we will be eligible for a variety of milestone payments.


In the US, we will be seeking a similar relationship with a commercialization partner, leveraging the existing sales, marketing, distribution channels, rather than seeking to build those ourselves. And similarly with cardiovascular disease, we have now a pathway to potential approval, both on a first pediatric indication and secondly, the end stage heart disease indication. Those are fully manageable by us as a company, given the small patient populations.


I think, in parallel, as we move the product through the FDA for potential approval in either of those two populations, we will be engaging with, and are engaging with currently potential commercialization partners who will take on the -- again, the potential commercial channels for the adult patients with class two to class four heart failure.


Unidentified Analyst


Awesome. Thank you so much and congrats on the progress.


Silviu Itescu


Thank you.


Operator


Thank you. [Operator Instructions]


Our next question today comes from Edward Tenthoff with Piper Sandler. Please go ahead.


Edward Tenthoff


Great. Thank you very much and congrats on all the update. I'm looking forward to the review for RYONCIL in acute GVHD. I'm wondering, when should we expect an update with respect to REVASCOR and kind of the regulatory filing plans. And how long do you think the Phase 3 for the spinal product will take? Thank you.


Silviu Itescu


Sure. Let me take the spinal product question first. The Phase 3 now is up and running. You know how enrollment works, where you first of all recruit. A lot of the centers contract them, they start to screen their patients, et cetera, and you have a hockey stick kind of enrolment period. And so, we're in that early phase right now and we expect that it will ramp up over the coming three months or so.


Our projection is that it'll take about 12 months to fully enroll, and then the primary endpoint is a 12-month outcome in terms of pain reduction. So, we will be updating the market as that program continues to move forward.


Edward Tenthoff


I'm sorry, Silviu. How many months to enroll? I apologize.


Silviu Itescu


About 12 months.


Edward Tenthoff


Yep. Thanks.


Silviu Itescu


With respect to the regulatory interactions on REVASCOR in cardiac disease, we have two potential early pathways to approvals. One is for the pediatric congenital heart disease and the other is for end stage patients on LVADs.


The immediate plan for discussion with the agency in the second half of the year is going to be on the pediatric indication because we have a pediatric rare disease voucher designation. And it is important if you know that the first approval is the one that is linked to a pediatric voucher. So that is the fact that we need clarity with the FDA whether the randomized controlled trial that has already completed and that was the basis of the voucher designation can support a filing for approval process.


After that discussion, we will be then also meeting with the agency, given the support they've given us, to an accelerated approval for the adults based on the totality of the LVAD study and the prior DREAM study. We will be meeting with them to understand exactly what clinical data needs to go into a filing for that patient population.


The confirmatory study that will be required as part of any accelerated approval will be a confirmatory study of the DREAM population in ischemic heart failure, patients with class three, four heart failure. So that's a trial that is being designed as we speak and will be obviously presented to the FDA when we meet on a pre-BLA basis.


Edward Tenthoff


Excellent. Thank you.


Operator


Thank you. And this concludes our question-and-answer session. I'd like to turn the conference back over to Dr. Itescu for closing remarks.


End of Q&A
Silviu Itescu


Great. Well, I'm very excited about the progress we've made in the last six months, and we look forward to maintaining the momentum and to updating the market in short order on FDA interactions and the potential approval of RYONSIL for our 1st commercial launch. Thank you everybody.


Operator


Thank you. That does conclude our conference for today. Thank you for participating. You may now disconnect.