Dr. David Solomon is the CEO of Pharnext, an advanced clinical-stage biopharmaceutical company using artificial intelligence to create therapies for rare and orphan diseases. Their first drug for Charcot-Marie-Tooth Disease (CMT1A) is in it's second pivotal Phase 3 trial. CMT1A is a rare genetic disorder of the nervous system with no current satisfactory treatment available. Dr. Solomon is committed to moving Pharnext's work on the CMT1A drug forward to bring solutions to the patients and families affected by the disease.

EPISODE HIGHLIGHTS

Share some background on your career and Pharnext.

I started my career in academia as a faculty member at Columbia University School of Medicine in Neurology Pharmacology and Biological Sciences. I'm now the CEO of Pharnext which excites me because of the strength in data of PXT3003, our lead medicine to treat Charcot-Marie-Tooth Disease, a rare genetic neurological disorder that causes sever impairment. Pharnext was established in 2007 and we're dedicated to getting PXT3003 approved.

What innovative approach led to finding a treatment for CMT1A?

The insight came from thinking about the genetics of the disease. CMT1A results from a defect and duplication of a gene called PMP22. The insight we had was to down-regulate PMP22 using a novel approach, maybe we could repair the defect and alleviate clinical disabilities experienced. We looked at the genetics of the disease, looking at a range of biochemical pathways that could be tweaked with medicine. PXT3003 is a fixed-dose combination of three medicines: baclofen, naltrexone and sorbitol. Together, these medicines bring unexpected and unanticipated results that help patients. 

Explain how artificial intelligence and big data is being used. 

We form a disease network around a specific disease. We look at the implicated genes of the disease, all the biochemical pathways affected and use a combination of genetic data and big data to analyze all the pathways and find which medicines in combination can improve outcomes of disease. 

What are the strengths and weaknesses in using artificial intelligence for rare disease?

AI and big data are predictive tools, but they're only as good as the input into the systems. It's beginning to be a standard tool that can be used as another approach, but it's the novelty of ideas, innovation and determination of scientists and biotech companies that brings results. There's no substitute for great science. 

Do you seek out patient registries and natural history studies with an abundance of patients involved and their data documented?

We first pick a disease where we think our platform can bring solutions. Then we look at the current therapies and if there are none, that's exciting for us. Finally we look at potential organized patient groups and reach out to them to see if they want to partner with us to provide access to patients or co-investing in early studies. 

What are you most excited about for Pharnext?

I'm excited that we're launching our study and by launching we're going to get our medicine into patients and closer to an approval. In the future, we're interested to see if our platform can perform for a range of other diseases where there are no other therapies.

LINKS AND RESOURCES MENTIONED

The Disorder Channel

https://www.thedisordercollection.com/

Pharnext

https://pharnext.com/en

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