Processing Your Payment

Please do not leave this page until complete. This can take a few moments.

September 10, 2019 Bioscience Notebook

Alexion inks licensing deal with CA biotech; BioXcel drug earns ‘orphan’ designation

PHOTO | Contributed Alexion Pharmaceuticals' research facility at 100 College St.
By Natalie Missakian

Alexion Pharmaceuticals Inc. has signed a licensing deal worth at least $50 million with a subsidiary of California biotech BridgeBio Pharma for the right to develop and commercialize its drug AG10 in Japan.

Under the deal, Alexion said it will pay BridgeBio subsidiary Eidos Therapeutics $25 million upfront along with a $25 million equity investment. There is also potential for additional milestone and royalty-dependent payments, Alexion said. 

AG10 is an orally administered small-molecule drug that stabilizes a protein known as TTR. The drug treats the root cause of transthyretin amyloidosis (destabilzed and “misfolded” TTR in the blood).

Edios is currently evaluating the drug in a Phase 3 study in the U.S. and Europe for ATTR cardiomyopathy (ATTR-CM), a progressive, fatal heart disease. It also plans a Phase 3 study to treat ATTR polyneuropathy, a progressive, fatal disease of the peripheral nervous system.

* * * 

New Haven artificial intelligence biotech BioXcel Therapeutics Inc. (BTI) said its immuno-oncology drug BXCL701 has been granted FDA “orphan drug” status for the treatment of Acute Myeloid Leukemia.

The U.S. Food & Drug Administration awards the designation to experimental drugs that show promise in treating diseases affecting fewer than 200,000 Americans.

In addition to tax credits, the designation allows BTI to qualify for a waiver of some FDA fees and seven years of market exclusivity if the drug is approved for the condition.

BTI has already been developing the drug for the treatment of pancreatic cancer and a rare form of prostate cancer. The company said it added AML as a target after pre-clinical studies showed it had potential against the aggressive blood cancer.

“BXCL701 has been observed to directly attack and kill AML cells in multiple preclinical studies, corroborating our belief that BXCL701 presents an opportunity to address this rare and deadly disease,” said BTI Senior Vice President and Chief Medical Officer Vincent O’Neill, MD, in a statement.

BTI, which uses artificial intelligence to repurpose older drugs and discover new ones, plans to study BXCL701 alone and in combination with other cancer therapies.

* * *

New Haven cancer diagnostics firm Precipio Inc. said it has partnered with the University of Pennsylvania’s Perelman School of Medicine to provide oncology diagnostic expertise to its customers.

“We are delighted that through this collaboration, our expertise will further benefit physicians and their patients worldwide,” said David Roth, MD, chairman of the university’s department of pathology. 

Precipio said the move bolsters its strategy to “combine leading academic sub-specialized expertise with the highest quality laboratory processes to eradicate the problem of disease misdiagnosis.”

In addition to its diagnostic tools, Precipio offers expert oncology pathology services to hospitals and health-care providers outside the U.S. through its academic partners, which include Yale School of Medicine and the Dana Farber Cancer Institute. 

CEO Ilan Danieli said that adding Penn will allow the company to scale up the business and “comfortably handle in excess of $25 million revenue per year in pathology services.”

* * *

Oncology biotech Cybrexa Therapeutics has tapped Arvinas CEO John Houston to serve on its board of directors.

The appointment comes as Cybrexa prepares to start human trials on its lead cancer drug, CBX-11, early next year. 

New Haven-based Cybrexa is counting on Houston’s expertise to help advance the company to the next level. At Arvinas, he oversaw the company’s successful initial public offering in 2018 as well as the launch of Phase 1 clinical trials on drugs for prostate and breast cancer this year. He previously served as a senior vice president at Bristol-Myers Squibb.

“John’s experience in oncology and guiding companies through the transition from preclinical to clinical-stage and private to public will be extremely valuable as Cybrexa continues to grow,” Cybrexa President and CEO Per Hellsund said in a statement.

* * *

New Haven-born antibiotics maker Melinta Therapeutics Inc. has appointed Jennifer Sanfilippo interim CEO and director.

She replaces John H. Johnson, who resigned last month after nine months in the position.

Sanfilippo previously served as Melinta’s senior vice president and general counsel. Her appointment, effective Aug. 28, was “the result of a thoughtful succession planning process undertaken by the board of directors,” Board Chairman David Gill said in a statement. 

Prior to joining Melinta, Sanfilippo held a series of leadership roles at the Medicines Co.

Melinta is now headquartered in Morristown, N.J. after closing its New Haven office in March. 

* * *

Biohaven Pharmaceuticals Holding Co. Ltd. said Tuesday it had completed enrollment of a pivotal Phase 2-3 trial of its second cutting-edge migraine drug, vazegepant, the first of its kind to be administered in a nasal spray.

Topline data is expected to be announced by the end of this year, the New Haven-based biotech said.

The study, which enrolled more than 2,100 patients, will measure the drug’s efficacy versus a placebo at doses of 5, 10 and 20 mgs. 

Part of Biohaven’s “nojection” migraine platform, the drug aims to provide fast relief that can be easily self-administered during an attack, Biohaven said.

Vazegepant is part of a new class of migraine drugs known as CGRP receptor antagonists, which work by blocking a brain chemical that transmits pain. 

It is Biohaven’s second CGRP receptor-targeting compound to enter the clinic. An FDA decision on its flagship migraine drug rimegepant is expected in early 2020.

Contact Natalie Missakian at news@newhavenbiz.com

Sign up for Enews

Most Recent

Most Recent

0 Comments

Order a PDF