CEO Arthur Lahr licht toe bij de presentatie van de halfjaarcijfers 2020 op 30 September 2020:
To give you an overview of our company, I would love to tell you a bit more about the platform and our stage of development. We have a truly unique immunotherapy platform based on hyperfunctional NK cells, derived from optimal donors and then expanded with PM21 platform.
We can engineer our cells and we do so where needed, but we don’t have to. Our base NK cell platform already has the appropriate persistence and potency to be effective in patients. The platform in addition is off the shelf and truly industrial and that really was what attracted our partners Sanofi and the U.S. Department of Defense.
We also have a lot of clinical data in severely ill immunocompromised patients with cancer, and in those patients, we’ve seen remarkable remissions and the strong improvement in outcomes, as well as strong anti-effective efficacy. We’ve never had any safety events and we’ve been able to give the product repeatedly to patients and see the cells persist in those patients.
So what do I mean when I say that our cells have a very bold applicability. If you look at the NK cells in nature, they have a multiple different ways to kill target cells. And in our NK cells, we have upregulated all those different mechanisms and have thereby made them truly hyperfunctional.
With that, the NK cells really have an unique role across the different diseases and across even heterogeneous disease an escape.
In addition, the platform is truly scalable. With our platform, we can actually continue to expand the cells weeks and weeks without any loss of functionality. And in addition, we can cryopreserve the cells without loss of potency and efficacy. And because of those unique features, we can take material from a single donor and expand it into more than $1 million prescription. That gives us a unique ability to scale up and to drive down cost of goods.
With our platform we’ve now created a very rich pipeline with four active programs and a number of additional programs that are under preparation. Our most advanced program is in transplants, where we’ve gotten approval to start a Phase 2, which we intend to activate and start enrolling end of the year.
Our AML program has generated very strong proof-of-concept data in 21 patients and we’re currently enrolling a Phase 1 in the United States. Our preclinical multiple myeloma program has been partnered with Sanofi. It is an engineered NK cell where we have knocked out the CD38. We partnered this program a couple of months ago and received an upfront payment of €17.5 million. We’ve recently launched an influenza COVID program where we anticipate to move into Phase 1 as soon as well and we have an IND approved for this Phase 1 and the program is fully funded by the U.S. Government.
To give a very high level overview of the partnership with Sanofi. What we partnered is our CD38 knock out NK cell. This knock out NK cell works synergistically with Sanofi’s recently approved anti-CD38 antibody.
The problem, as you may know, with anti-CD38 antibodies is that they hamper their own efficacy by not only killing multiple myeloma cells but also killing the patient’s own NK cells. By knocking out CD38 ex-vivo, we can infuse new fresh NK cells and thereby bolster the potency of these anti-CD38 antibodies.
And as I mentioned, the upfront was for this preclinical program was €17.5 million, but there’s many more milestones linked to preclinical, clinical and regulatory milestones, as well as up to a low double-digit royalties.
I showed you a rich pipeline that we’ve created over the last few years. But this is only just to start. This slide just gives a couple of the expansion opportunities that we have with our platform. The programs involved are the ones that we currently have running. But if you take for example, blood cancers, we have now programs in acute myeloid leukemia and multiple myeloma, which of course, has the opportunity to move beyond those programs, CML, long Hodgkin, etcetera.
In solid tumors, we have not yet launched a program, but we have a lot of preclinical data confirming the synergy between our NK cells and multiple approved antibodies, as well as for example, PARP inhibitors. And of course, we will expand into those programs and build a pipeline also in solid tumors.
In infectious disease, we launched a program in flu and COVID, but the clinical data that we already generated, that shows a dramatic reduction or dramatic effect on a serious microbial hospital infections, and of course, we want to expand and explore those opportunities further.
And then lastly transplants, the data that we have so far is in haplo transplants. But the impact of our cells both on residual tumor cells and infections, of course, makes this platform suitable to expand in other types of transplants.
We often get asked how we are different from other NK companies and there’s a couple of key parameters that clearly differentiate us and set us apart to some of the well known names, especially in the United States.
To start with clinical proof-of-concept data. We have more clinical data proof-of-concepts or proof-of-efficacy data in patients then any other party in the field. We have data in 45 patients with very long readout times, two years and beyond, which compares very favorably to the other parties.
We’re also clinically more advanced with a Phase 2 starting, where as most parties are still preclinical in Phase 1. We use very different source material. We start with mature fully licensed NK cells from healthy donors and we thereby pick the most optimal donors for specific indications, taking an ideal profile NK cell as a starting point for expansion. That is very different from a number of other parties who start with stem cells, which still needs to be fully maturated into NK cells like a product.
We also use a very different manufacturing platform. In our manufacturing platform we don’t use life impact cancer cells as the primary reagents to expand, but instead we use PM21, which is derived from these cancer cells, but it’s fully purified and does not contain any residual tumor cells and tumor DNA. And this gives us a huge regulatory and safety benefits over the other platforms.
Finally on engineering. All other parties engineer their cells because they have to, to obtain the right potency and persistence of their platforms. We’ve shown in our clinical trials, as well as preclinically, that even the non-engineered cells have the adequate potency and persistence. We can engineer, and the deal with Sanofi, of course clearly supports that, but we don’t have to. We only do it if there’s additional benefit.
With respect to partnerships, we have had not only a large pharma partner evaluate and thereby validate our platform but also the U.S. Government.
So to summarize our position in the field across the key dimensions and requirements for cell therapy. Having strong and potent cells that have a broad mechanism of action which are able to treat many different diseases. A truly industrial platform manufacturing system that allows reaching a large patient population.
I believe we truly have a great ideal solution to make NK cells for the future of immunotherapy.