In conversation with Praxis SCI Incubate cohort member AXONIS Therapeutics

AXONIS Therapeutics CEO & Co-Founder Joanna Stanicka, PhD, talks with us about restoring movement in paralyzed patients after spinal cord injury (SCI) through tissue reactivation and neuron regeneration.

 

What lead you here in terms of education, experience, and people you met along the way?

My journey into medical sciences, research and commercialization began when I was 10 years old in Poland. My cousin Ania got very sick with an aggressive form of rheumatoid arthritis. We were told she’d likely be in a wheelchair for the rest of her life. She was signed up for clinical trials with a new disease-modifying biological therapy, and within a few months the progression of her disease completely stopped. It was remarkable. Today Ania is a mother, runs her own house and – most importantly – is an independent person. To see a healthy person become completely dependent and in pain within a few months, then see them relieved so quickly with the right treatment was life-changing. I knew at that moment I wanted to pursue a career in science.

When I was 19 years old, I left Poland on a 40-hour bus trip to Ireland and went onto complete my PhD in cancer research at University College Cork. My focus was how genes and proteins impact the healthy and disease states of cells and applying this knowledge to developing treatments – particularly for cancer. While in Ireland, I was headhunted by Dr. Zhigang He’s laboratory at Boston Children’s Hospital/Harvard Medical School. What I was seeing in cancer cells, he was seeing for neuro-regeneration. I joined his lab to carry out this regeneration research and led a genome-wide CRISPR screening, which was a huge project involving many people.

In 2019, as we were coming to the end of the screening, Corey Goodman was looking for someone to lead a start-up company called Axonis, spun out from Dr. He’s research. Dr. He asked me if I wanted to lead Axonis, and after some thought, I decided to accept the position.

Joanna, Corey (current Axonis Executive Chairman and Co-Founder) and Zhigang celebrating Zhigang’s Reeve-Irvine Research Medal for his scientific contribution to the SCI field in 2019. It was a long night of discussions on Axonis and how to start the KCC2 program.

It’s been a transformative experience. I love everyday. It doesn’t matter how many hours I work. I am so privileged to be around such inspiring, hard-working and successful people, and we are all driven by our united vision to make a real difference for SCI patients, their families, and the global healthcare system.

How are your customers and potential customers challenged in their current situation?

Despite the fact that SCI impacts thousands of people worldwide every year, we still do not have any approved drugs to treat paralysis. This mainly stems from the complexity of neurotrauma.

Meanwhile the impact to those who sustain a SCI is not just paralysis – it is also pain and spasticity, which is the term used to describe when muscles stiffen and cannot be controlled. To further put this into context, there are about 300,000 chronically injured SCI patients in US, and about 70 to 80 percent of them experience either pain, spasticity or both. Therapies to relieve these chronic patients from paralysis, as well as pain and spasticity, are desperately needed.

What specific solution is your company providing?

Axonis is developing an oral drug to restore motor function, as well as reduce pain and spasticity, after an incomplete SCI.

The drug works by rebalancing excitation and inhibition of neurons in the spared tissue. In a typical nervous system, our neurons have a correct balance between being excited and inhibited. But after an SCI, there is an imbalance. Neurons may get excited or inhibited at the wrong moments. Through this oral drug, we are working to reactivate the signals to allow the correct balance to occur. The drug acts on paralysis, as well as pain and spasticity.

The approach we are using was first discovered in Dr. He’s lab in 2018. His research demonstrated that the spared tissue in mice with an incomplete SCI could be reactivated through restoring expression of a protein called KCC2, allowing them to recover their stepping ability. We have tested this in rats and we are starting safety and toxicology studies this year. The first-in-human clinical trial is planned for 2023. 

What differentiates your company from the competition?

There are two main ways our company is different. First, as far as I know, we are the only company on the market that is developing an oral drug for all three debilitating co-morbidities: paralysis, pain and spasticity. Oral treatments are generally more accessible than other interventions, such as surgery. They are also easy to administer and can be scaled up, depending on the level of pain or spasticity a patient is experiencing.

The other way our company stands out is two of our founders and leaders live with an SCI. Our Co-Founder and President is Bob Yant, who himself became quadriplegic almost 40 years ago. Our CFO, Mark Pinney, also lives with an SCI as a result of a car accident years ago. Having their perspective at the leadership level means we are genuinely motivated to ensure the drug development brings about meaningful changes to those with spinal cord injury, including the necessity of investigating pain and spasticity. We know that early interactions with consumers and listening to their needs is key to achieving success. Even though we are still at the preclinical stage, we are already guided by input from our consumers.

What do you hope to get out of SCI Incubate?

When Arushi Raina, the Director of Commercialization at Praxis, interviewed me, she smiled and said: “Why do you want to join the SCI Incubate program? You have everything you need.” But I am a cell neurobiologist and scientist who has worked primarily with rodents for SCI. I can design a study on mice or rats without a problem, but that is very different than running clinical trials with people.

Praxis helps connect me with consumers, and also provides mentorship within the SCI field on the mistakes that have occurred before for SCI clinical trials.  For example, Praxis highlighted the need to consult with consumers early on to learn what outcomes they want to see through our SCI clinical trials. Is it their ability to use their hands? Or the ability to walk? What is their main issue, and how can we help them get there? Praxis also pointed out the importance of tackling spasticity and pain. I had initially focused on the KKC2 enhancer to cure paralysis, but now I have learned that helping with pain and spasticity is as important as motor function.

The support from Praxis has been truly far-reaching. Praxis also supported our fundraising efforts and is helping to build out our regulatory, clinical and IP strategies. We are incredibly grateful for this mentorship and coaching.

Looking to the future, I believe that through strong collaborations, the next decade will bring breakthrough drugs and devices to the SCI community. This builds on all the exciting progress in SCI basic research that we have seen in recent years on regeneration, neuroprotection and neuromodulation. At Axonis, we want to be part of that future through delivering the first oral neuromodulating treatment.

Axonis team in COVID times. Despite the pandemic, we communicate with each other daily to ensure productivity. This photo was taken during one of our happy hours on Friday evening.

Learn more about the Praxis SCI Incubate Program.