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September 24, 2019 Bioscience Notebook

Biohaven drug to be tested in unique ALS trial led by Mass. General

Contributed | Biohaven Pharmaceuticals
By Natalie Missakian

An experimental ALS drug being developed in the Elm City will be tested next year in a first-of-its-kind clinical trial led by Massachusetts General Hospital.

Biohaven Pharmaceuticals’ verdiperstat was one of five drugs selected from a pool of roughly 30 potential new treatments for amyotrophic lateral sclerosis, the deadly neurodegenerative condition also known as Lou Gehrig’s Disease.

About 30,000 people in the U.S. suffer from the affliction, which causes progressive muscle weakness and atrophy and is usually fatal within three years.

Set to begin in early 2020, the nationwide trial will be the first to test multiple ALS therapies at once, according to Mass. General’s Sean M. Healey & AMG Center for ALS. 

The so-called platform trial will use specialized statistical tools to evaluate the drugs simultaneously.

The model, which has already been successful in the cancer field, is being advanced by the U.S. Food & Drug Administration and other proponents as a speedier, less expensive way to bring life-changing drugs for serious diseases to patients. 

“We are excited to accelerate how therapies are developed for people with ALS,” said Healey Center Director Merit Cudkowicz, MD, in a statement. “We are in a new era for ALS therapy development and we must think differently and boldly about how to find effective treatments.”

Cudkowicz said the approach can cut in half the amount of time it takes to find out if new drugs are effective and can lower trial costs by about a third.

Enrolled patients also have a better chance of getting an actual drug instead of a placebo, since data can be pooled from participants in all placebo groups, she said.

New drugs can be added to the study as they become available, and the center said it plans to keep the trial open until a successful treatment is found.  

“This study would not be possible on these timelines without the support and collaboration of the Healey ALS Platform Trial,” Biohaven Vice President Irfan Qureshi, MD, development lead for verdiperstat, said in a statement.

Biohaven competed for a spot in the trial with nearly 30 applications from drugmakers in 10 different countries. An expert panel selected the five potential treatments it considered most ready to participate in the study. 

The company’s proposed treatment is an MPO inhibitor, which controls the enzyme myeloperoxidase (MPO), believed to play a role in ALS because it increases stress oxidation and inflammation levels in the brain. 

Biohaven licensed the drug from AstraZeneca in 2018. It has already been tested for safety in 250 healthy volunteers in Phase 1 and 2 trials.

The Healey Center is providing partial financial backing for the latest trial, which will be conducted at 54 sites around the U.S.

Other drugs selected were zilucopan, a C5 inhibitor developed by Ra Pharmaceuticals Inc.; CNM-Au8, a liquid suspension of pure gold nanocrystals that aims to boost impaired motor neurons, developed by Clene Nanomedicine Inc.; Pridopidine, a highly selective S1R agonist, developed by Prilenia Therapeutics; and IC14, an immunotherapy drug developed by Implicit Bioscience Ltd. 

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Growing Branford biotech IsoPlexis Corp. said it has inked a deal with Tokyo firm BioStream Co. Ltd. to sell its products in Japan. Terms of the agreement were not disclosed.

The company said last May it planned to expand its reach globally after raising $25 million in a Series C funding round. 

IsoPlexis specializes in single-cell detection systems that allow researchers to characterize cells based on the proteins they secrete. 

The data can be used to develop highly targeted, personalized treatments for cancer patients, and predict how patients may respond to therapy, according to officials. 

“[W]e look forward to our systems generating impactful data at leading Japanese pharmaceutical companies, research laboratories and academic institutions,” CEO Sean Mackay said in a statement.

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Guilford-based Bioasis Technologies Inc. on Monday announced plans to raise about $4 million through a brokered private placement financing of units.

The company, which in 2018 moved its headquarters to Guilford from British Columbia, trades on Canada’s TSX Venture Exchange under the symbol BTI and on the OTCQB under the symbol BIOAF.

Boustead Securities LLC is acting as the placement agent for U.S. investors. 

Each unit will consist of one common share of the company and one warrant to purchase one common share offered at 22.5 cents in Canadian dollars (17 cents in U.S. dollars based on current exchange rates), Bioasis said.  

The biotech said it will use the proceeds for manufacturing, IND (investigational new drug) enabling studies, legal expenses and working capital. The non-public offering is expected to close on Sept. 27.

“This financing will provide additional support to Bioasis as we progress xB³-001 as a treatment for HER2+ breast cancer and brain metastases,” said Deborah Rathjen, CEO and executive board chair, in a statement.  “It follows the positive response we received from the FDA to our recent pre-IND submission and recognizes the strength of the company’s intellectual property covering xB³-001.” 

The company has developed technology to transport drugs across the brain’s filtering mechanism, the blood-brain barrier. Many drugs that work elsewhere in the body, such as the breast-cancer treatment Herceptin, are ineffective on brain metastases because they cannot fully penetrate the barrier.

Contact Natalie Missakian at news@newhavenbiz.com

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