Hello and welcome to Pathfinders podcast series from RBC Capital Markets, where we uncover the key trends and catalysts shaping the fast moving world of biotech and pharma. I'm your host, Joe Coletti. We're here at RBC Global Healthcare Conference in New York, and in this episode will be focusing on the treatment of chronic and life threatening mast and stem cell diseases.
Speaking with special guest Ronald Martel, Ronald is CEO of Jasper Therapeutics, a clinical stage biotech focused on the development of reclaimed or BRIC, a novel antibody therapy targeting kit to treat patients with mast cell and stem cell driven diseases. Ron is a veteran biopharma executive and serial entrepreneur. Having founded five companies and served on the boards of directors of several others.
Ron, thanks for joining us today. CHO Thank you very much. And on behalf of all of my colleagues at Jasper, really appreciate you and RBC for the opportunity that's great to hear. And we're so interested in this conversation. We can't wait to to hear a little bit more and share the audience in particular a little bit more of what you guys are doing.
Maybe we could start by just talking a little bit more about Jasper's mission and in particular, your lead program, BRIC. Sure. So our mission at Jasper is to bring safer drugs to patients who have a high unmet medical need and to really focus on areas where drugs can have curative intent. There there aren't a lot of opportunities in medicine where we can truly cure diseases.
And unfortunately, in those places where we we know we can. Oftentimes they're not available to patients because the drug itself may be too toxic and or the regimen that they're taking might be too toxic. And so if we can improve on that and we think that that's a significant advancement in the field. And so with that were our lead program is is an antibody called a MAB or BRIC, as you stated.
It's an antibody that targets a receptor on the surface of mast cells. In stem cells, that receptor is called C kit. C kit has been known in the field for a couple of decades. However, it's been a very difficult target to drug, especially with drugs that are small molecules. And the safety profile for those small molecules have been unacceptable to this point.
And that's why we're focusing on an antibody with a monoclonal antibody. You can have very high precision to make sure you're hitting just that receptor on the surface of those cells. That's great. Can you talk maybe a little bit more about your differentiated approach to the treatment, maybe expand a little bit more for our audience? Sure. So particular Mab as an antibody, when it binds to this receptor on the surface of a cell, it's the it shuts down all of the signaling.
And so you can think of it sort of as the lock and key or the key to start your car. So the ignition on the surface of the cell is C kit, and there's a circulating ligand in the body called SC for stem cell factor that naturally binds to this receptor and that creates the energy to go into the cell first to power the machinery inside of of the cell.
And with particular MAB, we bind in the same place that this normal ligand does. And so when we bind there, it's like turning the engine off so it shuts down. All of that signals so that energy goes away. And when that energy goes away, then the cells undergo what's called apoptosis or organized cell death. And so they don't break apart and cause unwanted effects, but they're just organized, sort of like enveloped, and then phagocytosis removes them from the body.
And so it's a really clean and efficient way. And, you know, you know, in medicine, when you're starting to get into the deep end of the pool, when it takes you a long time to be able to explain how your drug works. And that's one of the beauties about antibodies and about this target is it's it's really simple to understand.
It's an effector to target. It's an E to T of one. There are a number of other approaches that are being pursued that are out there. Most of them are small molecules. And the problem with the small molecule is to get the specificity because they have to get inside the cell and largely they will get inside of every cell in the body instead of being very specific to like the mast cell as an example.
And oftentimes then you end up with these unwanted side effects that I alluded to earlier that mean that a lot of patients can't take these drugs because the side effect profile is is too toxic. So you, Jasper, maintains full worldwide rights to develop and commercialize BRIC in all indications. Can you give us a little bit of an overview of how your development pipeline meets the market opportunity for mass cell diseases and and the franchise potential of these clinical programs?
Sure. And yes, indeed, we are fortunate at this stage of our company to still retain full worldwide rights, for all indications. We've explored the stem cell transplant conditioning space and have a number of ongoing clinical trials there as well, including some ultra orphan diseases like severe combined immunodeficiency or skid or the Bubble boy disease or Fanconi anemia, where we think we can help those patients who so desperately need it.
But I think the bigger picture is that particular MAB is really the definition of a portfolio within a product. So we have seven clinical trials ongoing, including those in the mass cell side of the business. And we're currently conducting a study in chronic spontaneous urticaria and another one in chronic Inducible urticaria. And we announced on Monday that we'll be starting yet another clinical trial in a mass cell driven disease, specifically asthma.
And so we're we're really excited about the potential here across mass cell diseases. So let's talk about a little bit how you see sort of the broader competitive landscape in this space right now and how you see it evolving, but also how. Jasper, I mean, I think you've alluded to a little bit in what you've already said, but why you guys are so well positioned, do you think, to succeed in the future?
Well, first, as a patient advocate and as a drug developer, I love seeing a lot of drugs in development. We're all better when there's competition, when patients have options. So I'm I'm a big fan of a lot of the different drug development that's going on. I think not to be too overly reductive, but our drug and our nearest competitor or cell docs with their antibody, we are the only programs in development that are depleting the mast cells.
All of the other drugs that are in development are designed to inhibit the mast cell. So all of these diseases have some trigger, maybe itchy. You know, there are a number of different triggers. And so they're attempting to block that receptor that with that trigger. But that would likely mean that maybe you only work in those patients. And then how do you identify the patients who only have IgG is a trigger.
The other downside in a lot of situations, oral therapies are really good and bad and have an advantage. But in this case what you need to do is have constant 24 seven 365 inhibition of the signal if you're taking an oral. And so if you're a patient in that oral is twice a day, it means you have to remember to take 700 pills a year.
And we all know that patients are that compliant. And if you take away that inhibition, maybe even for a day or so, then the symptoms are going to come right back. So I hope somebody is able to unwind that Gordian knot. But at the moment, we certainly think we have a superior approach. So I want to zoom out just a little bit on the sector, maybe think a little bit more broadly about some of the key thematics or other trends.
Obviously, there's a lot happening in the macro environment right now which sometimes is more impactful, sometimes is not in the space, which we know is very unique in a lot of different ways. Can you talk about things that you're really thinking about and watching closely, you know, particularly in the medium and long term as you sort of plan for the future?
Well, I think one thing that in this conference is evidenced by it is that in spite of the market the last couple of years, there still is very robust support from the investment community in this space. So I think that that's really reassuring that the capital is there. And so if you if you have a company that's worthy of financing, you're able to finance.
I think on the broader market, we'll look to see what some of the the federal regulations do or do not and what plays out over some of the things that have been proposed over the last few years or in the works. I think as a as a company that works in biologics and my entire career has really been in biologics, that the playing space there still looks a little bit more friendly than in some of the other spaces, but certainly we want to keep an eye on that.
Now, we've talked about A.I. and advances in AI a little bit at the conference. I know it may not necessarily be one of the big things that you're focused on right now, but I'm curious if you have any broader observations around A.I. and how advancements may be thought about or impacting the sector at large as we sort of look forward?
Well, I think I'll skip probably some of the other themes that you've heard from others that are more experts in the space talking about, you know, how to use A.I. to either better understand the biology or drug drug interactions or how can you use AI to identify and design additional molecules and different structures. One of the things I'm really interested in from an AI standpoint is in the conduct of clinical trials.
I think A.I. has the ability or the opportunity here for us to design better clinical trials. Unfortunately, we generally we design clinical trials that we enroll a lot more patients than what we probably need to because we're not really able to use, you know, the word we all use is in rich, right? How can we really identify the patients who have the best opportunity of responding to your drug?
And I think that's better for patients. It's certainly better for the industry. Drug development could probably happen faster. We could also probably eliminate drugs faster. It's better for patients so they don't go on trials where they may not have a benefit at all and ultimately from the industry. There should be significant cost savings because we're not running larger clinical trials for longer.
So I'm really interested to see how that could play out over the coming years. You know, you mentioned clinical trials. I want to ask your take on this. We've heard a lot from some folks in this conference about some of the challenges and especially in phase one and communicating to investors about the opportunity, given what you can or can't say during that phase.
Just curious, through your experience, do you find that that continues to be a challenge in some ways? Have you had experiences, whether at Jasper or before, of anything that you've done that you think has been, you know, more successful in terms of communicating, you know, more effectively, particularly when you're in that phase one? Yeah, I think maybe sort of back to the top of the story, the more easily explained or the more you can tell a story around your mechanism of action, then that translates to the investors and ultimately that translates to the patient.
Because generally what I've seen is that when investors are having a hard time getting their head around it, what that really means is they're already thinking, How am I going to explain this to a patient? How am I going to convince them to come in to a clinical trial? And I think that's a space where we need to get better at as an industry is how do we make certain that we can not just do what we have to do, which is informed consent, but really explain, you know, why we're doing this clinical trial?
You know what this drug might do to enhance involvement in clinical trials. One last question. This goes to sort of bigger picture could be about Jasper. Are there some things in the industry? Could be trends, could be themes? Anything else that you think, particularly investors, should be paying a bit more attention to or should be watching more closely in the weeks, months, sort of years ahead?
Yeah, I think we're living right now in the golden age of of tools for drug development. I'm reminded back to when I worked at Genentech and I was the program lead for trastuzumab or Herceptin and it was really the first targeted drug in oncology. And we were only dreaming of some of the tools that we might be able to have for how we could better understand what's going on inside of of a cell or something like that.
And and we now have those tools and I mean, look at CRISPR, what we're able to do, but we're able to, you know, really think about our drug development and how to design drugs in a better fashion. And I'm really excited that there's there's real opportunity to to take that in, translate that into something. Having said that, I think the caution with that also is we don't get too enamored with those tools and lose focus on, you know, tinkering instead of getting to the work of drug development.
I think that's a great way to end, Ronald. Thank you so much for being on the podcast and I hope you enjoyed the conference. It was a fantastic conference and thank you for having us. That's all for this episode. Thanks again for listening to Pathfinders and BioPharma brought to you by RBC Capital Markets. Please remember to subscribe to get more great content and be alerted about future episodes.
This episode was recorded on May 14th, 2024. If you'd like to learn more or continue the conversation, please visit our bookmarked forward slash biopharma. See you next time. This content is based on information available at the time it was recorded and is for informational purposes only. It is not an offer to buy or sell or a solicitation, and no recommendations are implied.
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