A biotech that's using artificial intelligence to identify neuroscience and immuno-oncology medicines received FDA approval for an investigational new drug application this week, and Benzinga connected with its CEO for more insight on the company's innovation efforts.
New Haven, Connecticut-based BioXcel Pharmaceuticals announced Monday that its IND was accepted by the FDA to initiate a clinical trial for the triple immuno-oncology combination of BXCL701, bempegaldesleukin and avelumab for the treatment of pancreatic cancer as a second-line therapy.
BioXcel is collaborating with partners including Nektar Therapeutics NKTR and Pfizer Inc. PFE for the trial.
In addition, BioXcel announced that the United Kingdom's Medicines and Healthcare products Regulatory Agency granted a clinical trial authorization for a Phase 1b/2 trial evaluating the combination of BXCL701 and pembrolizumab in treatment emergent neuroendocrine prostate cancer.
BioXcel Pharmaceuticals CEO Dr. Vimal Mehta updated Benzinga on the company's pipeline in a Q&A session, and his answers have been edited for length and clarity.
Benzinga: BioXcel is an AI-driven biotech, and your company is the first to use AI to identify compounds and bring them into the clinic. Can you give us some insight on the industry and the difference than AI can make?
Mehta: AI has a different definition for different people, but primary AI in our opinion is when we look at how it augments the intelligence of our drug development team. So we use AI machine learning to understand millions of data points in the publication. There are 5,000 applications being published per day. And it is very difficult for the human mind to be able to absorb all that information.
We use machine learning, and once we have defined a problem, we try to identify compounds that have the potential to meet medical needs. BXLC-501 has received fast track designation from the FDA. It's focused on the treatment of acute agitation.
Agitation is a large health care burden in the hundreds of millions of dollars. There aren't many treatment options for these patients.
It is a pretty large social problem, and we are trying to address that by developing a BXLC-501 supplement that is put under the tongue. It's minty in taste and will start working very rapidly.
The diseases we are targeting for this product [include] agitation and dementia from opium withdrawal symptoms. That's a national crisis, and patients need to get off opium.
Benzinga: The two lead programs that the company is working on using AI are neurology and immuno-oncology. What are some of the latest Phase 1 results from the neurology BXCL-501 program? Any readthrough from these results?
Mehta: The data shows a high level of confidence that drug is being delivered in order to provide benefits for the patient, and we have initiated a Phase 2 trial.
We will be dialing in the dose needed for schizophrenia. Once we have the data, it will allow us to move this program into the registration trial in schizophrenia and agitation as well as bipolar. We expect to have it by the end of 2019.
The trial will be completed in the first half of 2020, and we expect we will be able to file our first new drug application with the FDA in the second half of 2020. We continue our focus on AI technologies to identify patients.
Benzinga: What are some of the next steps for clinical development moving into Phase 2 and on to Phase 3? What are some of BXCL-501's strengths?
Mehta: Phase 2 is designed to make sure that we understand what the response rates are and how effective it is with different doses. We have an understanding of the highest dose and the lowest effective dose and are utilizing that information to power our Phase 3 trial so we can get a significant response in our Phase 3 trial.
In Phase 3, it is expected to be about 300-350 patients for schizophrenia and similarly about 300-350 for bipolar. So it will be a total of 600-700 patients. We are very confident based on what we have already seen, and we have announced this data previously with the current form of the drug.
With a small trial population of 14 patients — 10 on treatment and four on placebo — we saw a 90-percent response rate.
Benzinga: What's the latest news on the immuno-oncology BXCL-701 program? What can we expect in the near future?
Also: the largest oncology meeting in the world, ASCO, is around the corner — is BioXcel planning on attending?
Mehta: We are focusing on the immunity of the body. There are two types of immunity — one is innate. When you get an infection with anything in your body, it immediately reacts. The second is adaptive, where you have a response kicking in later on. So we are primarily focusing on immunity.
We are developing a product for neuro prostate cancer a product for pancreatic cancer. For the second, we have a partnership with Pfizer and Merck Serono in Europe. Four companies are working together to crack the problem of pancreatic cancer. We are using three different agents and sharing the cost of development.
In terms of neuro prostate cancer, it will come out at the end of this year. We have an ongoing trial where we are combining BXCL-701 with Keytruda.
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