Innovating Treatments for Rare Neurological Disorders

[00:00:02] Calan Breckon: Today’s episode is sponsored by Kit, Formerly known as ConvertKit, the email marketing platform for creators. I’ve been using Kit for years because I found that it is the most efficient and easy to use out of all the email service providers. Kit simplifies your email marketing by combining powerful automations with an easy to use interface. I love the Visual Automations builder because I am a very visual person and it really helps me to organize all of my automations in a very simple and easy way. And let’s face it, Automations is a must have in order to succeed in any business. Today. Kit also integrates with all of your favorite e commerce platforms, lead generation services, and much more. The best part about Kit is that it runs on a sliding scale for payment so that you can get started for free while you learn all about Kit systems and how to grow your email list. So to get started today for free, head on over to calandbreckon.com/kit or just click the link in the show notes. Now let’s get on to today’s episode.

Welcome to the Business Gay Podcast where we talk about all things business, marketing and entrepreneurship. I’m your host Calan Breckon and on today’s episode I have Anmol Nagpal. Anmol is a neuroscientist turned Entrepreneur with over 10 years of experience in academia, healthcare and entrepreneurship with research published in top journals like Nature Medicine and Proceedings of the National Academy of Sciences, also known as PNAS. Since March 2023 he has been an Entrepreneur in Residence at District 3 Innovations, coaching over 50 early stage life science startups and managing a portfolio of 25 later stage biotech ventures, helping them accelerate their path to commercialization. Anmol holds a Master’s of Science in Neuroscience with a focus on Fragile X Syndrome and other genetic brain disorders. He also has a Bachelor of Arts in Science from McGill University as well as a graduate Diploma in Business from Queen’s University. I’m excited to dive in with Anmol to chat about all the amazing scientific things he’s been working on with Nospharma. So let’s jump in.

Hey Anmol, welcome to the show. How are you doing?

[00:02:16] Anmol Nagpal: I’m doing well Calan. Thanks for having me.

[00:02:19] Calan Breckon: Well, thank you. I’m super excited to jump in because we are getting sciency on today’s episode. I know usually for the listeners they get really entrepreneurship like nuts and bolts, but I also like to really highlight folks that are doing amazing just work in the world and you know that’s part of what you’re doing. So I’m excited to jump in.

[00:02:38] Anmol Nagpal: I’m looking forward to sharing a bit about what we do.

[00:02:40] Calan Breckon: Nice. So, first of all, I want to know a little bit about Nospharma. Can you tell me just a. Like, a little, like, you know, intro, little elevator pitch?

[00:02:53] Anmol Nagpal: Yeah, absolutely. So Nospharma is a biopharma company based out of Montreal, and we’re working on rare brain diseases, specifically a genetic condition called Fragile X syndrome, which is something I’ve been. I was studying in the lab for a number of years. A number of years, all through undergrad and through grad school.

So that’s kind of our core focus. We’re a pharma company, so we’re developing a pharmaceutical therapeutic for Fragile X.

[00:03:25] Calan Breckon: Okay. And what inspired the creation of Nospharma outside of just you studying it?

[00:03:32] Anmol Nagpal: Yeah, that’s a.

Well, look, I. So it actually came from two independent sources. So I was studying Fragile X, and then my co founder, who’s a professor at McGill, was also studying Fragile X, and we were doing it in different ways and really almost unrelated approaches to the science.

And I kind of left, like, I finished grad school in Covid, and I went about working, trying to pay rent for a couple of years. And I got a call from him at some point saying, hey, Anmol, I know this was your scientific background. Would you be willing to provide it to me for my project on Fragile X? And I said, you know, we can chat about it, but I’m not going to, like, I don’t want to do the science anymore part of it. So I’m happy to help out and help you think about things. Think. Think about the science, but not necessarily do it.

And he was like, well, you know what?

Better yet, if you don’t be the scientist who does the things, I’ll do that. Why don’t you help me commercialize it?

And his way of looking at the problem, Fragile X was kind of unique. He was really looking at it in a way that I had never considered before, and I thought that it had potential to actually bring therapeutic to patients with Fragile X. So I jumped in. That’s. That’s honestly how it came about.

[00:05:08] Calan Breckon: Okay. And we’re going to jump into that in a second because I do have questions about that side of things. But first, let’s track back a little bit. What exactly is Fragile X?

[00:05:16] Anmol Nagpal: Oh, that’s a good question. I can’t believe I didn’t tell you what it is.

So Fragile X is a. Is a rare genetic condition that affects about one in. Well, I won’t give you those numbers. About 80,000 people in the U.S. about, you know, six to 7,000 in Canada and about 80,000 in the big EU five countries.

It’s a genetic condition that basically means that when you have the condition, you can present with a range of symptoms that are lifelong. They can be severe or mild.

We’re really focused on the severe cases. And like a kid with Fragile X will present with like severe forms of intellectual disability, might have sleep problems, might have motor problems, they might have adhd. And all these things kind of coexist in parallel for the kid usually. And it means that they’ll require lifelong care for the severe cases.

They’ll require care from their family, from the healthcare system, pretty much forever. They will of course, get access to behavioral therapies and doctors will prescribe things like Ritalin to help them with focus, or they’ll prescribe them sleep aids to get them to sleep. But it’ll never actually treat Fragile X and remains an inefficient way to actually help kids with the condition.

[00:06:42] Calan Breckon: And they. I can assume that there’s like adults who just like have had this their whole life and had no idea. And it’s one of those things that there’s new sciences coming out about a bunch of different things where they’re like, oh, this is actually all related. And this is kind of where it’s related. People are going, oh, this explains so much about my life. Let’s circle back over to the part where you were about to dive into kind of like the unique perspectives or ideas you were having about how to treat these things. So how does Nospharma approach treating this rare neural neurological disorder differently from other companies?

[00:07:15] Anmol Nagpal: So a lot of companies have tried to treat Fragile X. The big pharma in the past have tried and they failed.

There’s other companies trying today. What’s key for us is we’re kind of targeting two core biological basis of Fragile X. So my co founder identified that there’s an impairment in brain cell communication in, in Fragile X people. And that was important, but people had been studying it for a long time. People knew there was an impairment in brain cell communication. They were looking at all different types of biology associated with it and making drugs to target it. Some were better than others. A lot of them, actually all of them failed. And today there’s a bunch of competitors still targeting brain cell communication, but in parallel, he noticed that there was an impairment in the ability of blood flow to be delivered while that brain cell communication is happening. And the delivery of blood flow in the brain is super important because it’s what carries all the nutrients and energy necessary for all those things that happen during brain cell communication.

So we discovered that by treating both in parallel, you’re actually able to get a more holistic rescue and treatment of the condition in laboratory models, of course, and that targeting brain cell communication alone on its own is not sufficient.

[00:08:44] Calan Breckon: It’s the multi pronged approach to like, this is working, but this is kind of working. But what, you know, if they come together, it’ll be even better, hopefully. Fingers crossed. Okay, so in that same kind of vein of thought, developing new medications can take a long time, like ridiculously long amounts of time. How are you doing things at Nus Pharma in terms of like working with health agencies to get treatments approved as quickly as possible?

[00:09:14] Anmol Nagpal: Yeah. So we took up a kind of an interesting strategy which was we didn’t make new drugs.

We understood the biology and so we said, well, there are drugs that exist to target both of those biological things. They’re used for other, other stuff, or one didn’t ever make it to market and sits on a shelf somewhere.

And our philosophy was we’d rather use something that already exists than create a new drug. And the simple reason for it was if we make a new drug, we have to make sure it’s safe. We’re going to spend a lot of money, a lot of time just doing safety stuff, making sure the drug actually works as the place we think it’s targeting.

So we said, let’s skip that and let’s just pull two drugs that we know actually target the biology that we want to target.

Instead of doing all this expensive work for no reason, we wanted to get the treatment to patients quickly. That was the philosophy behind it. As quick as possible. And then as a proxy of time and speed, it was also cheaper because we could leverage existing clinical data on the safety of each of these drugs to move forward through clinical trials. So I’ll give you a very tangible example. Like it would cost upwards of US$10 million to take a new molecule, even just to test it for the first time in a healthy patient population. To test it for safety, you’d spend probably $10 million just getting to that stage.

And that’s not running the clinical trial of the safety. For us, it costs about 2.

Doesn’t sound like much of 8 million. I mean, in pharma it doesn’t sound like much, but at the beginning of the journey it’s a huge sum of money. And you get to skip about two years of work in the lab and with patients. So that’s a huge value add in terms of both patent protection, but also it gets to patients much faster.

[00:11:29] Calan Breckon: So what stage would you say that nose pharm is at right now in terms of, like, developing treatments? Are you still very much in, like, research early modes with the medications that are existing, or are you moving into kind of patient testings? Like, where are you playing right now?

[00:11:46] Anmol Nagpal: Yeah, so our therapeutic is a combination therapeutic that basically we are combining into a single pill of these two molecules. The stage we’re at is what’s called IND enabling. It’s called investigational new drug. Now, what that means is just a fancy way of saying the FDA has now agreed to let us run the clinical trial. So we’re putting together the package of data that we would need to take to the FDA who will say, go for it, run your clinical trial. But it’s not just approval to run a clinical trial, it’s approval to run an inpatient clinical trial.

So one caveat here is that we get to skip the whole safety clinical trial portion of this work, which is called phase one, and move directly into patients at phase two.

[00:12:41] Calan Breckon: Gotcha. So with that and moving into patients. Now, I know you said that there’s a certain amount of people across Canada, so there’s already kind of a certain amount of existing folks. But what would happen if somebody wanted to get tested for this or if you wanted to test people to see if they could qualify or, you know, how does that work with doctors? Because a lot of the times doctors aren’t looking for these kind of obscure things at first. There’s so many other zones they go to before they kind of narrow down in this. And even then you have to have a doctor who’s kind of like, aware enough to test for that specific thing.

[00:13:18] Anmol Nagpal: Yeah. The way to test for fragile X is to do a genetic test.

It can be complicated to get access to a genetic test across geographies across Canada, across the us, across the eu. An example I’ll give you is, I think a couple months ago, the National Health Service in the UK released a report saying that a lot of young girls who have been diagnosed with, with autism are actually fragile X positive. And they’re recommending that when girls come to the clinic and get diagnosed with autism, that they’re automatically given a genetic test for fragile X.

So, you know, a single, like a healthcare system like ours, a single parent healthcare, healthcare system will kind of mandate physicians to do certain things in the presence of certain symptoms.

There’s also a component of women and girls being under diagnosed because their symptoms present a little differently than the men counterparts. And that’s why the NHS released such a statement saying, okay, physicians, you’ll have to genetically test young girls that you’ve previously diagnosed as autistic.

[00:14:35] Calan Breckon: That’s important because also just women in general in the medical system tend to get gaslit on so many things and so many issues of like, oh, you’re just being, you know, hormonal or this and that. And now we’re really looking at things and realizing, oh no, we’re totally gaslighting women into thinking that they don’t actually have what they have. And like well into their 30s, 40s, 50s, being diagnosed with things and being like, oh, okay, well, had you not gaslit me my whole life, I would have actually been able to start working towards this. And women specifically are under diagnosed autistic because it’s usually the guys or the rambunctious boys that get, you know, tested and you figure it out. Whereas, you know, girls or women tend to present a little bit more quieter or mask a lot heavier than men do and therefore it’s just not happening.

[00:15:27] Anmol Nagpal: Yeah. One of the things that was very important to both our co founder and I was that we, like, it’s a simple thing and it sounds simple, but we don’t do it in the medical sciences necessarily is that all our, like animal testing, our rodent models of fragile X include females, include female mice. And it was super important that we include it because we wanted the therapeutic to be that we could actually test it in women patients when we get to the clinic. And if we had no earlier data to support a rationale for it with the fda, they would say just, just use boys and men, like recruit boys and men.

So for us, like, it makes it of course more expensive because then you have to include two separate gender, well, in mice sexes, so it makes it more expensive. But for us, it was fundamental that we ensure that female mice are included in all the pre clinical laboratory work to make sure we have a good understanding of the similarities and also differences and the potential of the treatment in both groups.

[00:16:37] Calan Breckon: Good, I’m glad you’re doing that because it’s very important because, you know, a lot of medical history is based on just male and men and their stats and their data, and women kind of get like, okay, here’s the leftovers. So I’m glad that you’re taking those important steps. What do the next stages of research look like for Nospharma.

[00:16:58] Anmol Nagpal: So really we don’t do in house R and D. That’s the, I think key. We made a conscious decision not to set up like a laboratory and hire scientists.

It can make you very not nimble as a startup having rent to pay and massive overhead. So the next step for us in terms of our science is actually hiring what we call clinical research organizations. They are the people who have the techniques and the models to just execute what we need them to execute and send us the data. The other advantage of that is that they’re at arm’s length from us.

So we pay them to do it, we design it from them, but we don’t manage the data. They analyze the data. And that’s also kind of important to make sure that we’re looking at it in a little bit of a removed way.

[00:17:54] Calan Breckon: Objectively. Yeah, nice. Okay, so how can people, whether that be patients or families or supporters, get involved and learn more about Norse Pharma? Or if maybe they present in with certain autism or other things that, that you’ve talked about in this episode, maybe they want to get tested and like connect with you? How can somebody go about doing that?

Okay, start with general connection. How can people generally connect with you?

[00:18:26] Anmol Nagpal: Okay, so generally you can reach out to me on LinkedIn or, or shoot me an email. I think my email is on the website somewhere.

We like do talk to your physician if you’re, you know, presenting symptomatic in some way, like you’re concerned about your health. I’m not a physician, so I can’t, I can’t give you any medical advice.

But your, your doctor can recommend you for genetic testing of all sorts of things that might be relevant for you and.

[00:19:01] Calan Breckon: Yeah, no, no, that’s totally fine. Do you have any idea of like how that process happens in Canada? Because like you said, it didn’t seem like it’s something exactly that doctors do kind of right off the start. It’s one of those things that you have to go through the process, process of doing. And is it out of pocket in Canada? Is it included? Do you have any idea about that?

[00:19:22] Anmol Nagpal: I think it’s province specific. It’s typically included to some degree.

But I know that wait times to get access to genetic testing can be quite long. So if you’re not able to access it, if you can afford to do it privately, you should be able to access it pretty quickly.

But it is province specific. And I know like certain healthcare systems are more bogged down than others across Canada, so you might be waiting different lengths of time in Ontario or Quebec, and it’ll come at the recommendation of your family physician.

[00:20:03] Calan Breckon: Okay, cool. Awesome. Well, I’ll make sure to have the links to your LinkedIn and website and all that information in the show notes for anybody who is interested in you and the work that you are doing. I want to thank you so much for being on the show today. Anmol. This was a very different approach that we usually take to entrepreneurship, but I was excited to have you on and talk about the cool work that you were doing.

[00:20:25] Anmol Nagpal: Thanks, Kellen. Thanks for having me.

[00:20:27] Calan Breckon: Thanks for tuning in today. Don’t forget to hit that subscribe button. And if you really enjoyed today’s episode, I would love a star rating from you. The Business Gay Podcast is written, produced and edited by me, Calan Breckon. That’s it for today. Peace, love, rainbows.