As general counsel for the clinical-stage biopharma company, Alex Nemiroff is guided by his experiences as the father of a child with a rare and incurable disease
When he was 36 years old, Alex Nemiroff's life changed forever.
That was the day his first son, Roger, was born with an extremely rare genetic epilepsy (SCN2A mutation) that currently has no cure. Roger started having seizures within hours of his birth, and he was diagnosed with a condition that carries every symptom imaginable and most often leads to death.
"He was our first child, so we really had no idea what to expect and certainly never expected this," Nemiroff says. "It was completely terrifying."
That was nine years ago. Within six months of Roger's birth, Nemiroff, an attorney, and his wife committed themselves to finding a treatment. At first, they focused only on learning enough to try to improve Roger's life.
"Doing the research was a knee-jerk reaction," says Nemiroff, now general counsel at Praxis Precision Medicines. "I thought learning more about the condition might open up some opportunity to potentially change my son's health trajectory. In retrospect, it is clear that we were trying to find a ‘fix’ so that we and our son would not have to deal with the realities of how sick he was."
With no experience in drug development, the lawyer-turned-biopharma-entrepreneur ventured out on a mission that he admits was way over his head, but he wasn’t deterred. Along with learning all he could about Roger’s condition, he began raising money and cofounded RogCon, a biotechnology company, to develop an antisense oligonucleotide to treat it.
Alex Nemiroff, general counsel for Praxis Precision Medicines. Photo courtesy Praxis Precision Medicines.
RogCon forged several partnerships on its way to developing a proof of concept. Then it enlisted the help of Praxis Precision Medicines.
"What was actually really helpful was the fact that we had no idea how challenging this was," Nemiroff says. “Throughout this process, people kept telling us we weren't going to be able to do this. ‘Go home and take care of your kids,’ they said. ‘They're very sick and need you at home. You're not going to cure this disease in their lifetime.’ But we continued to push, and here we are."
Nemiroff also began embracing spirituality, which included a deep, introspective dive into his own fears and insecurities. By creating grace, he says, he began functioning at a more wide-ranging, compassionate level, including maintaining a broader mindset around all children who are born with SCN2A.
"I really started to work on myself and to face a lot of the fears, issues and insecurities that have plagued me my entire life,” he says. “And for me, that meant diving into psychoanalysis and spirituality. What I discovered is that what holds so many of us back, whether it's being a leader, being a part of a team, driving a program, or a company, are our own issues. And so when I look back at the past with my son, I see so many things that held us up, that slowed progress down, and the way I dealt with challenges could have been a lot better. I have become more effective than ever before in confronting challenges through the personal work that I've been doing to transform these issues."
Nemiroff credits his shift in mindset to the successes along the road to development.
"Looking back, I realize how unlikely it was to end up where we ended up," he says.
“What I learned is that for any endeavor to succeed over the long term, you have to have a true, genuine, almost pure desire that is motivating and driving whatever you're doing so that it can really withstand all the attacks, challenges, and anything else that comes up along the way. I think about a time when this drug program started to slow down and get stalled, and it was incredibly frustrating and heartbreaking. It was around that time that I started to realize that trying to help my son is actually a very selfish desire. And I started to refocus my thoughts on the bigger picture, on how this will help all these other kids, and that's when things started to move."
"I was very worried about being seen as the parent of a sick child who was pushing this drug as fast as possible, and who has no appreciation for risk or drug development,” he says.
According to Nemiroff, Praxis President and CEO Marcio Souza disagreed, and he encouraged Nemiroff to be authentic.
"Your perspective is the strongest thing that you bring to the table,” Nemiroff says he was told. “You absolutely should push the urgency that you feel because otherwise, nobody is going to do it.”
“That completely changed my perspective, and transformed the way I looked at my role,” says Nemiroff, who now encourages parents and patients to speak up and add their perspective wherever they can.
“My role at the Praxis table goes beyond just general counsel, but also includes being a parent of a sick child, which informs my perspective,” he says. “And it really allows me to participate in discussions about things like drug development, science, and engaging with patients in a way I otherwise would not have been able to do. And what’s so fascinating to me is the interaction really spurs very interesting conversations and angles to look at these drugs and programs in a very different way.”
Praxis recently announced its plans for 2024. They include:
- Collecting topline results of PRAX-628 in a Phase 2a PPR study in the first quarter of 2024;
- The expected initiation of the Phase 2b study in focal epilepsy;
- Releasing results of the Phase 2 EMBOLD study of PRAX-562 in SCN2A and SCN8A developmental epilepsies, which should be available in the first half of 2024.
- Collecting topline results for both Phase 3 studies evaluating ulixacaltamide in essential tremor later in 2024;
- Completing regulatory interactions to advance elsunersen (PRAX-222) towards a pivotal study for the treatment of SCN2A gain-of-function (GOF) developmental epilepsies.
Late last year, Praxis posted a preliminary analysis of PRAX-222 Part 1 that showed 44% median reduction in seizures after three doses for SCN2A-gain-of-function pediatric patients.
Designed as a safety study only, the results were a surprise and a win.
“What we saw was completely shocking and incredible to us,” Nemiroff says. “If we had had even just 10% to 15% efficacy, just to demonstrate there’s a signal, that would have been phenomenal. To see 45% median seizure reduction and a significant increase in the number of seizure free days for these kids was just incredible for us to see.”
While these statistics are beyond expectations, Nemiroff has learned to be cautiously optimistic.
“There’s been so many setbacks and challenges along the way that, sure, a fear of the other shoe dropping builds up," he says. "Until this drug is really over the line and available for all these kids, it's going to be hard to actually let go, breathe and celebrate."
Robin Robinson is a contributing writer for HealthLeaders.
A small step toward trying to save his son transformed into a life-changing mindset reaching beyond the lines of drug development
As a young lawyer, Nemiroff was met with challenges beyond his personal and professional capabilities, but he rapidly learned there was no alternative but to embrace them
In 2024, Praxis plans to advance the four clinical-stage assets in its portfolio for movement disorders and both rare and broad epilepsies