Developing treatments as a parent

Episode 3: Parents on a mission

Having a child with SCN2A or other genetic epilepsy is challenging and the future is uncertain. When Alex Nemiroff’s son Roger was born, he was determined to work towards finding treatments for SCN2A. Alex is now the CEO of RogCon, which has a research program (RC-222) developing an antisense oligonucleotide (ASO) treatment for gain-of-function epilepsies.  

Hosted by Kris Pierce and David Cunnington, parents of Will, who has SCN2A.

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Guest interview:

Alex Nemiroff is the father of Roger, who has SCN2A, and a co-founder and CEO of RogCon. After Roger was born and found to have SCN2A, Alex was determined to work to find treatments for SCN2A. He joined forces with Kelley Dalby, a like-minded SCN2A parent, and together with Prof Steven Petrou, formed RogCon. RogCon has partnered with Praxis Precision Medicines to help develop a treatment for SCN2A.

Regular Hosts:

Ms Kris Pierce RN MHSc MWellness, is a rare disease advocate and mother to Will who has SCN2A. Kris has held a range of board, project management, advocate and consumer representative roles and has been instrumental in working with local, state and federal governments to secure funding for multi-million dollar projects. Kris is highly skilled in building teams to work together collaboratively and is a co-founder of Genetic Epilepsy Team Australia (GETA) and SCN2A Australia, and a RARE Global Advocacy Leadership Council member.

Follow Kris on LinkedIn or Twitter.

Dr David Cunnington is a sleep physician and father to Will who has SCN2A. He is director of Melbourne Sleep Disorders Centre, and co-founder and contributor to SleepHub. David trained in sleep medicine both in Australia and at Harvard Medical School in the United States. David’s clinical practice covers all areas of sleep medicine and he is actively involved in training health professionals in sleep. David is a regular commentator on sleep, both in traditional and social media.
Follow David’s posts on sleep on Facebook or Twitter


Intro: Welcome to SCN2A Insights bringing you the latest research and clinical updates on SCN2A and genetic epilepsy from around the world.

David Cunnington: Hi. Welcome to this episode of SCN2A Insights. I’m David Cunnington.

Kris Pierce: And I’m Kris Pierce. In this episode, we speak to Alex Nemiroff who is Chief Executive Officer and Founder of RogCon, a small biotech company focusing on drug development in SCN2A. He is also the parent of Roger who is affected by SCN2A.

David Cunnington: So thanks very much, Alex, for joining us on the podcast.

Alex Nemiroff: Happy to be here.

David Cunnington: What’s your relationship with SCN2A and genetic epilepsy?

Alex Nemiroff: On the personal side which is certainly first and foremost, I have a son, Roger, who was born with SNC2A mutation that in his case caused seizures a couple of hours after birth. And in retrospect, we were pretty sure he was having seizures before he was born.

And so, when he was born and we were dealing with his situation along with taking care of him, we began to kind of explore the prospects of facilitating a way towards a novel treatment that years later now has led to a company that I’m a co-founder of along with Kelley Dalby and Steven Petrou called RogCon. And the goal of that company is really to discover and develop novel therapeutics for mutations in SCN2A and the patients that suffer from them.

David Cunnington: So I really take my hat off to you having been through that same journey as you and for us having Will with seizures at his first couple of days of life, I reckon’ we were pretty much scrambling for the first five years or so and he is 17 now and we are still scrambling to some extent. How did you find that vision and time to say, “All right, I’m going to do something about it and this is what I’m going to do?”

Alex Nemiroff: In retrospect, I think if I’m being totally honest, it was in many ways, a coping mechanism for myself, sort of the idea of rather than maybe dealing with the pain and the trauma directly to shifting into this mode of how can I help fix this? And I hate to use the word “fix” because of what it implies. And so essentially what I mean though is I just kind of very quickly looked at this as objectively as I could in that situation to say, “Here’s this challenge. Is there anything that I can do to help address it?”

When Roger was born and he had seizures right away and we transferred him from Miami where we lived in the US to Boston Children’s. I remembered distinctly having a conversation with his doctors there when we got the diagnosis and they were explaining SCN2A to us and they said, “This is severe and the outcome is going to be poor or likely poor but it’s a bit uncertain because nobody has really studied this disease extensively.”

That statement really stuck with probably starting that night when we were just up like probably you guys, I mean all the other parents when we were just reading everything we can online and trying to wrap our heads around this. But I remember thinking OK, if it hasn’t been studied extensively then we haven’t sort of exhausted or the community hasn’t exhausted the prospects for some sort of a novel treatment.

So when we left and we were discharged, that kind of became my obsession and focus was is there anything or what can I be doing to help accelerate efforts to better study this disease. And once we do that, can we take those learnings and translate them into some type of novel therapeutic approach?

David Cunnington: That was your vision and where you were at. How did you then link up with Kelley?

Alex Nemiroff: After we’ve left the hospital and we were back home, I connected with a lot of different parents. I think everyone ultimately has the same objective. We all want to find what we were calling then a cure. I met Kelley through some conversations with a number of parents. And what stood out with respect to her was that she seemed to have this very narrow focus like I did on everything that we do and every effort that we put towards this disease should really be around accelerating toward development.

For me, it kind of fit in that her personality of sort of being a little bit more aggressive on that front was really a nice complement to mine which is a bit more of a universal perspective on what are the pieces that we need to bring together. So what we decided to do together was start to sponsor some basic research. We asked around enough doctors and we were directed to Australia and to the lab of Steve Petrou. And from the first conversation with Steve, we knew that this was the right person to work with. And so together, we began the journey there and started studying these mutations.

What had happened at the time, back – this was in 2014, there was a little bit of a misunderstanding – a misdirected understanding of this disease. It had really been studied largely in this small subpopulation of inherited cases where seizures just like in my son’s case and in yours was seizure onset first day of life to be seizures through the first year but then in inherited cases, the seizures were remitting and the prognosis for the most part was good. So with that construct, it didn’t really make all that much sense for many people to be focusing on this and soon they are trying to find a novel treatment because the symptoms were largely remitting.

When we spoke to Steve the first time, he said, “Look, what we really need to do right away is really study and understand the biophysical consequences of these de novo, spontaneous mutations. And then depending upon what we see, we could go a few different routes.”

And so the three of us together began doing that.

David Cunnington: So it can’t all be plain sailing. You are trying to look after Roger at home. You are trying to manage developing this. You’ve literally got skin in the game. There’s emotion riding on the outcome. Has everything gone positively or have you had some setbacks?

Alex Nemiroff: By this point, we had just started down the road of studying this. And everyone that we had spoken to was very, very receptive to talking to parents who seem to be motivated towards understanding what the children were dealing with. So at this point, it was certainly something that we hadn’t faced resistance on it. And like I said, I mean it was kind of feeding this escape from actually dealing with the situation in front of us. And ironically enough years later now, I find myself more dealing with the pain and the trauma that I avoided all these years just focusing on this new treatment. Back then at that point, we hadn’t faced resistance.

David Cunnington: So reached out to Steve Petrou and he suggested looking at the biology of SCN2A. How did things progress from there?

Alex Nemiroff: So we started to see what was Steve had anticipated that was increased excitability. The natural progression from that point in terms of a therapeutic would be something that would effectively modulate that excitability. And Steve suggested that there was a company in the United States in California with a very interesting technology. The company’s name at the time was Isis. It’s now Ionis. He said, “Why don’t we reach out to them and see if they feel like their technology would be a fit with what we are seeing in the lab and then maybe we could begin to collaborate together?”

At that point, we kind of had to make a decision on, do we want to continue down this path and Kelley had started a foundation prior to she and I meeting and we were sort of operating in that space initially but what we felt was that the best that we could do in this process would be to kind of marshal it forward and continue to push and to bring parties together. And we felt like the posture, the optimal posture to do that would be from a company’s perspective as opposed to a nonprofit.

What was really sort of serendipitous is that originally there was some question as to the commercial viability of this disease and over the first couple of years working on it, diagnostics improved, we had a better understanding of the population, orphan incentives made developing  drugs for rare diseases very desirable. And so, we were able to justify shifting into a company platform and trying to move this forward in the company context, which like I said, I always felt like it would be much better way to get this moving and moving fast than if we were trying to do it from a nonprofit vantage point.

So we went to Ionis early on and we basically pitched them on the idea of working with us and seeing if their technology which is antisense oligonucleotides would be effective in this disease and in the model systems that we have created.

David Cunnington: And it takes money to develop these type of products. I’m sure you’ve had to make pitches and get outside your comfort zone as a parent. How do you go with that process?

Alex Nemiroff: That has definitely been one of the most emotionally challenging parts of this whole thing. I think that pitching to people on investment is challenging enough on its own. When you are pitching on something that ultimately if successful going to help your kid and a bunch of others. So the success or failure of your pitch has a lot more riding other than just economics. It has been really tough.

We faced a ton of rejection. I mean it’s rejection that at times makes you want to just sort of quit and stop. But of course, we are coming home every night to our kids and talking to all these other families and seeing what’s at stake here. So within that, you have kind of the strength to continue to push on and we did in those early years and we just took meeting after meeting and we found our way to get the funding needed to move this forward.

And yeah, I mean looking back, that was possibly the hardest part of all this. And it continues to kind of be as we move forward because I think it’s easy to kind of overlook the fact that essentially your entire day when you are working on these programs and all of the various aspects related to them, you are coming home to your child at night who is potentially going to be directly impacted by the success or failure of your efforts. So that has emotionally been tough. But fortunately I things are moving forward.

David Cunnington: Along the way, you formed partnerships with Ionis and now Praxis Precision Medicines. And what are those partnerships allow you to do?

Alex Nemiroff: Another kind of I think core philosophy that we had at RogCon that we never felt like we should be building out a company ourselves essentially like hiring people to perform each of the tasks that are needed to develop a new drug. We felt like that would not be a smart way to do this. But rather, what we decided to do was really try to leverage via partnerships the best in the world as we saw it for each of the various parts of this process. And so, early on as I mentioned, we began working with Steve Petrou and his lab at the Florey Institute doing all the target biology work. We then began a collaboration with Ionis Pharmaceuticals around their antisense technology. That collaboration which now is continuing all the way through the stage we are right now in this program is going to provide us with effective and the ultimate drug that we – assuming all goes well, we will be bringing to market.

And then on the commercial side, we began talking to Kiran Reddy, the CEO of Praxis, years back and developed the relationship with him when he was assisting almost in a mentorship type role our early years at RogCon. And I think the goal was always if there would be a way to work together, we thought that he was someone we would want to work with and who we would trust with a program like this. Fortunately, over those years that we were doing a lot of the early stage where he started Praxis and the timing aligned such that we recently formalized the relationship the Praxis wherein they are serving as the development and commercial partner on this program. So basically, they will be the ones that are taking this drug and do a lot of the safety work and engaging with the regulators and bringing this to market.

And so we felt like and now feel like there are really sort of the best people and best group that are working on each aspect of this program. So ultimately, we kind of feel very confident that we have an optimal structure.

David Cunnington: And that’s also positioned you well ahead of a number of other genes or groups looking at other SCN2A treatments because no one else has got that commercial partnerships and structure to actually bring that drug development right through the clinical trials and right through to then bringing a drug to market. So that’s just fantastic that you’ve been able to bring that together.

Alex Nemiroff: Well, I think that when that one big aspect of all of this that kind of really led us to where we are is that everyone seems to share a similar vision and priority which is to accelerate this to market as quickly as possible while of course feeling like we are bringing something optimal in terms of safety and likelihood for success to the patient community, and that’s certainly something that we felt with Ionis and without question that’s something that we feel very confident in Praxis, their desire to bring quality epilepsy drugs to market.

David Cunnington: And it must have been a proud moment for you at AES last year when Steve Petrou presented that poster about the ASO that’s in development. How did that make you and Kelley feel in terms of the work that you’ve been doing?

Alex Nemiroff: That was a fantastic milestone. There had been milestones along the way that had been incredible to see. But the tough thing about working in this space and being persistent and a person connected to it is that you quickly hear stories about many of the kids that are facing so many challenges, including your own, some of them who pass away, you are experiencing how long this drug development taks and how challenging this process is and all the reasons why this drug should ultimately fail. And so, it just gets sobering very quick. I don’t think that we’ve actually celebrated any really – of these milestones more than for a minutes and then it’s time to what do we need to do next and how can we kind of mitigate risks of the next stage of this program as we move forward.

David Cunnington: Thanks very much for the work that you’re doing because, very humble in the way you are responding, because from the outside when we as parents look at the work you’re doing and some of the work coming out of Steve’s lab as part of your overall project. It’s fantastic work and really gives us a hope that things are moving forward.

Alex Nemiroff: Things are definitely moving forward. The goal as we stated on the first day we’ve set out because to work on this stuff would be not stopping until there’s a treatment for every kid that’s suffering from this disease. And as the years have kind of progressed, we have seen a bunch of different subpopulations and ultimately that means different treatments for different subpopulations.

So I think everybody understands it’s a very challenging space to be in but the good thing is that in this case the programs that we are working on, we are very optimistic based on the data that we are seeing that we are going to be able deliver something to the patients that will meaningfully impact the disease.

David Cunnington: Thanks very much for your help, Alex, and the work that you are doing.

Alex Nemiroff: Absolutely.

Kris Pierce: It was great to hear from Alex and just to see the personal drive behind why he and Kelley set up RogCon, their insight and drive to find treatments and cures as a parent has had its challenges and they’ve worked through those and are bringing something to our community. As parents, we all want to do something and to be honest, totally inspired by Alex and Kelley and the work they have done with RogCon and we look forward to where that goes.

David Cunnington: So you can keep up with the latest updates by subscribing to this podcast or get regular updates on SCN2A through SCN2A Australia’s Facebook or Twitter @SCN2AAustralia.

Outro: This podcast is not intended as a substitute for your own independent health professional’s advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider within your country or place of residency with any questions you may have regarding a medical condition.