Biotech Blazer Blooms On Long Wharf

Twelve stories above New Haven Harbor, a burgeoning biotech company is using Big Data technology to mine Big Pharma chemistry to create drugs that treat pancreatic cancer and symptoms of Alzheimer’s Disease, schizophrenia, and delirium.

The company is BioXcel Therapeutics. It is, a publicly-traded spin-off of the Branford-based data analytics company BioXcel Corporation, which specializes in using Artificial Intelligence (AI) to create new medicines in the realms of neuroscience and immuno-oncology.

BioXcel’s first two drugs, BXCL501 and BXCL701, both of which are still under development, arose from the sifting through of millions upon millions of pages of pharmaceutical research and clinical studies to find existing drugs that can be reconfigured and repurposed to address underserved medical needs.

“BioXcel Therapeutics is the first public company that is utilizing artificial intelligence platforms in developing innovative medicines,” BioXcel Therapeutics CEO Vimal Mehta said with pride during an interview with the Independent. ​“AI is in our DNA.” (Click on the video below for an overview of one of the AI-technologies that BioXcel Therapeutics uses.)

Since its founding in March 2017, the company has raised $60 million and grown from five to over 100 employees.

Just a few weeks ago, the company finished a $200,000 renovation of its new twelfth-floor office space at the Maritime Center at 555 Long Wharf Dr., where 20 of the company’s employees work on AI and drug development. The rest of the company’s staff works at a manufacturing facility in Philadelphia.

“We wanted to be easily accessible to a lot of people who are investors, analysts, partners who travel between New York City and Boston,” Mehta said about basing his company out of 555 Long Wharf.

Opening a headquarters in New Haven was significantly cheaper than doing so in Boston or New York.

“That was also a very big factor,” he said. ​“Developing a biotech company here in terms of the infrastructure was very economical. I would recommend that to every CEO who is building a company, to build it in New Haven.”

Proximity to Yale University and Yale-New Haven Hospital didn’t hurt in attracting world-class scientists, he said. ​“We did not have any problems in the recruitment of the talent.”

The Daily Grind

Here’s how the company works: Every day, according to Mehta and BioXcel Corporation co-founder Krishnan Nandabalan (who is quoted in the above Microsoft-produced video about the AI technology BioXcel Therapeutics uses), over 5,000 papers in the medical field are published. Plus, Mehta said, there are over 50 million such scientific papers already in existence.

“There’s so much generation of new information,” Mehta said, ​“but there’s no accumulation and no means to be able to digest this information and put it in a format that allows you to identify new insights about drugs.”

That’s where BioXcel Therapeutic’s AI computers and proprietary algorithms come in.

These computer programs pour through all that data, looking for drugs that are already in at least Phase 2 of clinical development but, per BioXcel Therapeutics’s analysis, are not currently realizing their full biological potential. (In the world of clinical trials for new medication, Phase 1 refers to testing a potential drug to see if it is safe for patients to take. Phase 2 refers to testing a safe drug among a patient population to see if it actually works.)

By focusing on Phase 2 drugs, Mehta said, his company cuts in half the necessary risk and requisite investment that comes with developing new drugs. That’s because another company has already spent the time and money on discovery, clinical manufacturing, and Phase 1 safety tests.

Thanks to the ever-rapidly expanding scientific understanding of which biological mechanisms can be used to address which medical needs, there are likely thousands of drugs at late stages of development or already in the marketplace that could be reconfigured and repurposed to address something different than what they had initially been designed to do.

Agitation Creation

Take BXCL501, for example.

Mehta said that his company discovered that Precedex, an injectible sedative used during surgery that was developed by Pfizer over 20 years ago, has the biological makeup necessary to treat agitation experienced by people suffering from Alzheimer’s, schizophrenia, bipolar disorder, opiate withdrawal, and delirium.

BioXcel Therapeutics then adapted the biological mechanism underlying Precedex into a new drug in the former of a sublingual film that, when tweaked, greatly reduces physical and mental agitation experienced by people suffering from a variety of illnesses. The new drug just recently received Fastrack approval by the fedearl Food and Drug Administration (FDA), meaning that Mehta hopes to have all studies done and the drug certified and available for purchase and use by early 2020. Based on Precedex’s current price, he said, he anticipates that the new drug will cost around $145 per dose.

“Agitation currently is a $100 million healthcare burden in the United States alone,” Mehta said. ​“In order to give relief to the patient, you need to help them with a symptoms-based approach.”

The company discovered its second drug, BXCL701, through a similar process of mining existing biomedical studies and research. But in this case, they found a drug that, when combined with existing drugs made by Pfizer, Merck, and Nektar, could be used to treat pancreatic cancer and neuroendocrine prostate cancer. In particular, this repurposed drug, which BioXcel Pharmaceuticals acquired in 2016, activates the body’s innate immunity to break down biological barriers surrounding a tumor, which the Pfizer, Merck, and Nektar cocktail can penetrate and reduce.

Mehta said he estimates that 3,000 drugs have cleared Phase 1 safety tests but, due to an insufficient prior understanding of the drugs’ full potential biological impact, are currently underutilized.

“Our development cycle is four to five years rather than 10 to 12 years,” he said, thanks to their AI-boosted analysis of existing medical needs and of existing medicines that can be remodeled and repurposed. “We like to say that we are changing the R&D economics.”