tracked it down
22 March, 2016
Transcription of Discussion Given by Mesoblast Limited (ASX:MSB) Managing Director & CEO, Professor Silviu Itescu
Mesoblast Limited (ASX:MSB) is a biomedical company; we’re developing late stage therapies based on proprietary cell therapy, that we’re developing. Cells called mesenchymal lineage cells that are present in all of us that we extract and we can industrially scale up, for a range of very important unmet medical needs.
I think there are several aspects that make Mesoblast unique. Firstly, we are using a very interesting and proprietary technology, the mesenchymal lineage platform. These cells are unique in that they can be expanded into large numbers, from a very small start in population and that they do not activate the immune system. So therefore, they can be used from one donor to literally treat thousands of unrelated people. And that gives us a business model that is scalable and industrial, with products that look and feel like typical drugs and biologics, with potency assays with batch-to-batch consistency and reproducibility.
And that allows us to target large unmet needs, including chronic heart failure, including severe chronic back pain and including severe immune-mediated diseases. Our cells have certain receptors on them that can respond to signals that are present at high levels, in patients with immune-mediated diseases like rheumatoid arthritis. And in those patients, our cells are able to respond by switching off multiple arms of the immune system. That’s particularly relevant to a disease like rheumatoid arthritis, especially in patients where 30 per cent of the time, there’re people who don’t respond to standard immune-mediated therapies, because the disease is multifactorial.
So in this target population, biological refractory patients, we are currently in the midst of a 48 patient Phase 2 trial. Randomised placebo controlled that is currently being evaluated in the US, testing a single low dose injection and a single high dose injection versus placebo, in these unfortunate patients. And data that we recently reported top line results on, suggested that in the first cohort, who received a single low dose of our off-the-shelf allogeneic cells, at three months out there was quite a substantial achievement of the primary end point, the ECR 20 improvement. And in addition to that, a number of patients achieved what looked to be sustainable, durable remissions.
So we were very excited by these initial data. Graft Versus Host disease is a devastating complication in children and adults, who’ve undergone treatment for leukaemia with chemotherapy. And then in order to build up their immune systems, they receive an unrelated donor by marrow transplant. In that scenario, 50 per cent of children and adults develop Graft Versus Host disease, where the graft attacks their body. In those children and adults who have the more severe forms of the disease, involving liver and gut, the probability of mortality is as high as 80 to 85 per cent, in the first several months. And in this disease, we’re developing a product that we call MSC-100-IV that aims to turn off the immune system and induce a complete remission. And therefore, improve survival in these unfortunate cases.
Our partner JCR Pharmaceuticals Co Limited is our distributor for this product, for Graft Versus Host disease in Japan. They’re also the manufacturer of the product. And we will be the recipient of royalties and certain payments, based on achievable milestones. We said, we explained to the market that we intended to reduce our cash burn on a quarter-on-quarter basis, by 20 to 25 per cent. And we’ve achieved that in the last quarter. We expect to continue to maintain that over this coming period. And as I’ve just mentioned, that we intend to bring in additional corporate partnerships into the company. Which if were successful, will mean additional cash and certainly extension of our runway, through to at least a potential for a first product filing with the FDA.
There are a number of important catalysts. We’re now a mature company; we’ve got multiple products in Phase 3. We expect certain interim readouts from some of our Phase 3 trials, and we expect additional partnerships to unveil themselves. And I think you’re going to see that Mesoblast will continue to execute on what we have laid out, in terms of crystallising events. We’re a global company, we have multiple products, we have a unique and patented technology that encompasses multiple jurisdictions. So we’re very excited about the near-term crystallising of events for the company.
source:
http://www.finnewsnetwork.com.au/archives/finance_news_network122843.html
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