Mesoblast (NASDAQ:MESO) is an Australian biotech company that the Wall Street Transcript has been keeping in touch with for over 8 years.
Our latest interview with CEO Silviu Itescu, MBBS, FRACP, demonstrates the perseverance and intelligence needed for a novel medical treatment to succeed.
Silviu Itescu, CEO and Managing Director Mesoblast (NASDAQ:MESO)
Dr. Itescu has served on Mesoblast’s board of directors since the company’s founding in 2004, was Executive Director from 2007, and became Chief Executive Officer and Managing Director in 2011.
Prior to founding Mesoblast in 2004, he established an international reputation as a physician scientist in the fields of stem cell biology, autoimmune diseases, organ transplantation, and heart failure.
Dr. Itescu has been a faculty member of Columbia University in New York, and the University of Melbourne and Monash University in Australia.
In 2013, Dr. Itescu received the inaugural Key Innovator Award from the Vatican’s Pontifical Council for Culture for his leadership in translational science and clinical medicine in relation to adult stem cell therapy.
The Mesoblast (NASDAQ:MESO) CEO had this to say recently:
“These are off-the-shelf therapeutics that are frozen, cryopreserved at minus 140, and shipped in that way finally to the hospitals.
And we’ve done the appropriate Phase II, Phase III clinical trials that support regulatory approvals across various jurisdictions, including the FDA.
That’s the snapshot of the company.”
The Mesoblast CEO goes on to explain:
“…When you’ve got this mega inflammatory condition that results in inflammatory cytokines that destroy your organs, like your intestinal tract and liver, those same cytokines activate our cells through surface receptors.
And when our cells get activated in this way, they release other factors that turn off the very cells that made those inflammatory cytokines.
So, they’re kind of orchestrators of an anti-inflammatory response that really brings down that severe inflammatory process and protects the end organs.
Ultimately, GVHD is a disease where the bone marrow transplant attacks the body through these cytokines.
And if you can tone down the cytokines in the immune cells of the bone marrow, then you’re protecting the patient. And really, you’re protecting the gut, you’re protecting the liver.
And ultimately, the objective is to change a disease that is fatal 70% or 90% of the time to a disease that can be cured.
That’s what we do.
…We’ve successfully completed the Phase III trial.
We met the primary endpoint.
The FDA now is reviewing the file for a potential approval.
They’ve already inspected our manufacturing site, so the expectation is that we will get a decision on an approval no later than the PDUFA date, perhaps in advance of that.
And we have to prepare and are preparing for a successful commercial launch.
We have built our resources in-house in terms of the Head of Commercial and Medical Affairs and are building out a very small, targeted commercial team that will be in a position to engage with the hospitals that do all these transplants.
Our product is already being used under compassionate care, under expanded access, and we’ve treated several hundred children already in the major centers across the U.S. So we are well known.”
Mesoblast (NASDAQ:MESO) has been developing this drug for years. In a May, 2016 interview, Dr. Itescu stated that:
“The nearest product to commercialization is our orphan drug indication for graft versus host disease, which is a major complication of bone-marrow transplants.
We have a Phase III trial actively recruiting in children, where already in data for 260 children we have shown that we can significantly impact both response rates and, more importantly, survival at day 100.
This is a devastating complication in children, where the mortality rate can approach 80%.
So if you can have an impact on survival, you’ve really got a tremendous drug on your hands.
This Phase III trial is expected to complete recruitment toward the end of this year, and we expect by the end of 2017 to have an application with the FDA for approval.
So that’s the shortest timeline of our products for commercialization.
That same product was already approved very recently in Japan and has been launched in Japan in the last month or so, evidence that a first-world regulatory body has looked favorably on our products and that we’ve received the kind of reimbursement that one would expect for an orphan drug.
That’s pretty exciting for a company in the stem cell space.”
In a March 2019 interview, Mesoblast (NASDAQ:MESO) CEO Silviu Itescu stated that:
“Our first product approved globally is being marketed in Japan as it has been licensed to JCR Pharmaceuticals, which is an orphan drugs specialist in Japan.
The approval was the first fully approved cell therapy product of any type in Japan.
It occurred about two and a half to three years ago after an approval for use in children and adults with steroid-refractory graft versus host disease.
Graft versus host is a disease that is a devastating complication of an allogeneic bone marrow transplant.
It occurs in about 50% of people who get an allogeneic bone marrow transplant, and typically, 90%-plus of people who undergo these transplants have had high-dose chemotherapy for their underlying leukemia.
So the transplants serve to rebuild their bone marrows after chemotherapy.
You can imagine seeking a cure for the underlying leukemia and then getting this devastating complication of graft versus host disease, which has a very high mortality in its most severe forms.
It is a terrible complication.
There is nothing that is approved other than steroids in the first instance.
Also, 50% of people will fail steroids.
After steroids, nothing else is approved anywhere in the world other than our mesenchymal lineage product in Japan.
So we’ve been very pleased that this product was approved.
It has received very good reimbursement.
We believe that since the launch of the product, the adoption rates by clinicians approximates about 50% of our expected addressable market.
So we are very pleased by the uptake and the way it has performed.
We are aware of no safety issues.
It gives us great insight into how we would expect a similar drug to perform once it has launched in the United States.
We will be filing with the FDA for the same product very shortly.
We have been using it now in the United States for several years under an expanded access program, so over 240 children have already received this in the United States.
We have performed a very rigorous Phase III trial that demonstrated similar data to what we had generated in the earlier EAP program.
In the Phase III trial, we were able to establish a very significant improvement in overall response rates at day 28 and a very significant association with improvement in survival at three months and at least through to six months.
So the results that we have achieved clearly demonstrate that this product, if approved, will be a first-line therapy after steroids have failed.
The Japanese experience informs us and gives us great confidence as to how it’s going to perform in the U.S.”
Mesoblast (NASDAQ:MESO) also interviewed with the Wall Street Transcript in 2021 at the tail end of the COVID epidemic. In this interview, CEO Silviu Itescu stated:
“We achieved the pre-specified primary endpoint of Day 28 overall response and achieved a significant outcome around survival benefit in children with steroid-refractory GVHD.
For children under 12, there is no approved therapy.
In these children, mortality rate approaches somewhere between 70% to 90%.
So it’s a real bad disease, a real unmet need.
We’ve demonstrated through the submission process and with a 9-to-1 positive vote by the ODAC panel — there was agreement that we demonstrated both safety and efficacy for these children for whom there is no approved therapy.
Our focus is on demonstrating to a sufficiently high standard to the FDA that if approved, the product maintains lot to lot consistency every time we make it.
Obviously, in the Phase 3 trial, the product demonstrated consistency because the results speak for themselves.
We’ve continued to make the product.
And we have established new assays that I think continue to even further underline that we understand the mechanism of action and that go to the heart of the mechanism that the cells go through in order to improve the outcomes in these steroid-refractory GVHD patients.
And so we think that we will have alignment with the FDA because the potency assays now are very much in line with the disease process, how we think the cells turn off the disease process.
And we think that it’ll allow us to be very confident, and for the agency to be confident, that as the product is hopefully in the marketplace, it will demonstrate that every lot behaves exactly the same way as the lot before.
That’s really what the FDA wants.
They want to see that we’ve got a handle on the mechanism, that we have a handle on the production, that there’s no differences in production from every product that goes out the door.”
“Well, over the next 12 months, we expect to see some pretty substantial inflection points, including first product approval and launch in the U.S. market.
I think we have enough cash to get us through that period.
We’ve got most recently $138 million in cash.
We’re in the process of refinancing and extending our debt facility.
And that will strengthen our balance sheet.
And we expect to enter into several strategic partnerships, which will also, I think, strengthen our balance sheet.
So we’re pretty confident around our cash position.
And as long as we’re judicious in the use of capital, which we are, and invest primarily in the areas that are focused on commercialization — i.e., the commercial footprint and manufacturing inventory, which is what we’re focusing on very much on the first logical product — I think we’re well cashed up at this point.”
Read all the interviews in their entirety with the Mesoblast (NASDAQ:MESO) CEO Silviu Itescu to get an in-depth look at how biotech entrepreneurs develop their cutting edge products and bring them to market globally.
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