January 2, 2019
Bioscience Notebook

FDA fast-tracks BioXcel drug for agitation; Yale researchers make promising Alzheimer's discovery

BioXcel CEO Vimal Mehta

BioXcel Therapeutics' experimental drug to treat acute agitation has been awarded federal "Fast Track" status — a designation likely to accelerate the regulatory review process.

The news follows the U.S. Food & Drug Administration's decision last month to greenlight the New Haven company's investigational new drug application (IND) for BXCL501, an under-the-tongue version of an already approved intravenous drug dexmedetomidine (Dex).

BioXcel is seeking approval of the drug to treat agitation in dementia, schizophrenia and bipolar disorder patients.

"This regulatory milestone will help facilitate clinical development and expedite regulatory review of BXCL501, bringing us closer to potentially delivering an optimal therapeutic solution effective for both patients and caregivers," said BioXcel CEO Vimal Mehta.

The FDA awards "Fast Track" status to new drugs with the potential to treat serious or life-threatening conditions and address unmet needs. Company officials say current IV treatments for acute agitation are invasive and come with serious side effects.

BioXcel, which trades on the Nasdaq exchange under the symbol BTAI, is conducting a Phase 1 study of the drug's bioavailability and safety in healthy adults. It expects to announce initial findings within the next six months.

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New Haven biotech Cybrexa Therapeutics said it plans to begin the federal review process for its first tumor-targeting cancer drug by the end of the year.

Cybrexa said Wednesday it has chosen CBX-11 as its lead drug candidate to treat patients with various solid tumors. It expects to file an investigational new drug application (IND) with the FDA in the fourth quarter of 2019 and start human trials in early 2020.

The nearly two-year-old company, led by serial biotech entrepreneur Per Hellsund, is developing anti-cancer agents that target tumors while sparing normal tissue.

Cybrexa says its alphalex platform, based on Yale research, can deliver small molecule cancer-fighting agents directly into tumor cells, resulting in fewer side effects.

CBX-11 combines that technology with an already-approved anti-cancer agent known as a PARP inhibitor, which stops cancer cells from being able to repair themselves. The drug will be studied both alone and in combination with chemotherapy, Cybrexa said.

"Selection of our lead clinical candidate is the first step in establishing the human proof-of-concept data for our alphalex technology and marks a major milestone for Cybrexa," President and CEO Hellsund said in a statement.

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Yale researchers have identified a "drinkable cocktail" of designer molecules they believe could potentially slow the progression of Alzheimer's disease.

Consisting of a polymer derived from a decomposed antibiotic, the cocktail repaired brain synapses and restored memories in mice engineered to have a condition that mimics Alzheimer's, according to research published today in the journal Cell Reports.

It works by interfering with the binding of amyloid beta peptides to prion proteins — which triggers events such as the buildup of plaque that lead to the progression of Alzheimer's.

A collaborating team at Dartmouth University reported positive results when it delivered the same cocktail to cells modeled to have Creutzfeldt-Jakob Disease, another dementia-causing neurological condition.

The study was led by Stephen Strittmatter, a professor of neurology and neuroscience and director of Yale Alzheimer's Disease Research Center, and Yale research scientist Erik Gunther.

Yale said the next step is to ensure the molecules are safe for clinical trials for Alzheimer's disease.

Contact Natalie Missakian at news@newhavenbiz.com

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