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New Haven Biz-April 2023

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14 n e w h a v e n B I Z | A p r i l 2 0 2 3 | n e w h a v e n b i z . c o m AI Advantage: BioXcel uses artificial intelligence to speed up drug discovery By Liese Klein D eveloping new drugs is rarely as simple as spending an amount of money and coming out with an effective and marketable product on a set schedule. Artificial intelligence can help — siing through millions of molecular combinations and existing drugs to find the few that can be repurposed or redesigned to cure. Now, innovations in artificial intelligence like OpenAI's ChatGPT chatbot are highlighting AI's potential — and New Haven biotech BioXcel erapeutics is ready to capitalize on those breakthroughs. "ere are a number of things out there that are showing really good promise," said Frank Yocca, chief scientific officer at BioXcel. "It shows the power of using AI when you can use it in the right way, when it can really benefit." ChatGPT, launched publicly in November, harnesses the power of an AI platform that can process huge quantities of data to produce human- like responses to queries. Some of the chatbot's responses have proven to be inaccurate or inappropriate, but the AI behind the technology is constantly learning and improving — and bioscientists are taking note. "We look at all of the different and various new things that are applied in AI that could make our system work more effectively," Yocca said. e spotlight on the evolution of AI comes at a busy time for BioXcel, Yocca said. e company commercially launched its first drug late last year — Igalmi, an under-the-tongue treatment for the agitation associated with schizophrenia or bipolar disorder in adults. BioXcel's AI technology identified the compound that became Igalmi, a sedative previously approved for use in anesthesia called dexmedetomidine. A second AI-discovered candidate, BXCL701, performed well in a phase two trial against a form of prostate cancer with no currently approved treatments, the company announced in January. In combination with immunotherapy drug pembrolizumab, BXCL701 helped patients with small cell neuroendocrine metastatic castration-resistant prostate cancer, an aggressive form of the disease. BioXcel's candidate has the potential "to redefine the standard of care for patients with advanced disease," Dr. Rahul Aggarwal, associate director for clinical sciences at the University of California San Francisco Helen Diller Family Comprehensive Cancer Center, said in February. "is drug now will enter a bigger phase 2B study, and hopefully head for approval," Yocca said. "So, we're very excited about that as well." As part of efforts to develop cancer drugs like BXCL701, BioXcel formed a new subsidiary last year called OnkosXcel. Growth targets e company's recent developments led BioXcel executives to New York on Feb. 21, where they rang the Nasdaq closing bell. A public company traded on the Nasdaq, BioXcel plans to ramp up sales of Igalmi this year; it expanded its salesforce to 70 as of December, the company said in March when announcing its financials. BioXcel missed its fourth-quarter revenue targets for 2022 but had $194 million in cash on hand as of Dec. 31, according to a statement. e company said it was seeking strategic financing from Oaktree and Qatar Investment Authority. Analyst Francisco Javier Garcia, of Seeking Alpha, said BioXcel should see stronger sales of Igalmi as the drug rolls out beyond hospital settings. Smaller biotechs have been impacted by market instability due to recent bank failures impacting revenue targets, he added. "Success in the commercial launch of a drug is determined by good revenue growth in the first few quarters, and I really think we'll see very good growth in the next few quarters," Garcia said in March. At the heart of BioXcel's development in recent years is its AI platform, which sis through terabytes of scientific data in journal articles and other sources to create knowledge graphs, which link facts and concepts and puts them in context. e networked findings are then analyzed by data scientists and neuroscientists to identify new drug compounds, or existing drugs that can be used in new ways. e innovations on view in ChatGPT, by contrast, center around BioXcel executives ring the Nasdaq closing bell in New York on Feb. 21. PHOTO | NASDAQ Frank Yocca

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