This week's life sciences industry news includes a Main Line company's progress with its lead new drug candidate, the expansion of clinical testing for a Bucks County company's medical device in stroke patients, the launch of a new life sciences consulting firm, and more.
Here's the roundup:
Larimar Therapeutics
The Radnor biotechnology company has received Food and Drug Administration clearance to advance a Phase 2 clinical trial for its lead drug candidate CTI-1601, an experimental treatment for Friedreich's ataxia.
The FDA had placed a partial clinical hold on the study in 2021 after Larimar informed the agency of mortalities which occurred at the highest dose levels in a 26-week non-human primate toxicology study designed to support extended dosing of patients. At the time of the initial notice, Larimar had no interventional clinical trials with patients enrolled or enrolling at the time. The hold was lifted in September.
Larimar (NASDAQ: LRMR) said the four-week study will involve patients receiving a daily dose of 50 milligrams of CTI-1601 daily for the first two weeks, then be dosed every other day for the final two weeks.
The company's open label extension trial, where patients will receive 25 milligrams of CTI-1601 daily, was also cleared for initiation by the FDA.
“Gaining clearance to advance our Phase 2 trial and initiate the [open label expansion] trial are crucial steps in CTI-1601’s development as potentially the first therapy to increase frataxin levels in patients with Friedreich's ataxia,” said Dr. Carole Ben-Maimon, president, and CEO of Larimar. “Given the inability of current treatments to address the frataxin deficiency underlying Friedreich's ataxia, we believe CTI-1601 has the potential to improve the treatment paradigm for this devastating disease."
Friedreich's Ataxia is an inherited disease that causes progressive damage to the nervous system and movement difficulties. An estimated 15,000 people in the U.S. and Europe are afflicted with the disorder.
Helius Medical Technologies
The Bucks County neurotech company is expanded its previously announced trial to test its flagship PoNS therapy as a potential stroke treatment to 60 patients from 12.
The investigator-initiated study, led by Dr. Steven Kautz at the Medical University of South Carolina, is evaluating the effects of cranial-nerve non-invasive neuromodulation — delivered using PoNS therapy — on gait and dynamic balance in chronic stroke survivors.
The company's PoNS device, an acronym for portable neuromodulation stimulator, is a portable, non-implantable device that delivers mild electrical stimulation to the dorsal surface of the patient’s tongue. The device consists of a controller that goes around the neck and a mouthpiece that rests on the front of the tongue — with the electrodes facing the tongue. The electrodes send mild electrical impulses to the tongue to stimulate two cranial nerves that have direct connections into the brain through the brainstem.
After receiving FDA marketing clearance in 2021, Helius launched the device last year in the U.S. to help patients with multiple sclerosis with gait deficit improve their walking ability. PoNS is also approved in Canada for use in both multiple sclerosis and stroke patients.
Dr. Antonella Favit-Van Pelt, the chief medical officer at Newtown-based Helius (NASDAQ: HJSDT), said the company has welcomed the opportunity to help support and expand Dr. Kautz’s stroke study. “This is a significant step toward our efforts to leverage the breakthrough designation granted in August 2021 and pursue approval for stroke in the U.S. in the near future,” she said.
Patient enrollment is expected to begin next month and be completed by the end of the year.
Helius, which has 26 employees, generated revenue of $787,000 and posted a net loss of $15.5 million in 2022.
NexGenLab
A consulting firm specializing in laboratory expansion and operations has opened, virtually, in the Philadelphia region.
NextGenLab is being led by co-founders Clifford Brechner and Richard Higby, two industry veterans with more than 60 years of combined experience.
“Our primary objective is to liberate the principal investigator and entrepreneur from the day-to-day challenges of laboratory relocation and expansion," Brechner said.
Brechner said the services NextGenLab will provide — such as staffing, training, site selection and advisory services — are designed to give entrepreneurs the freedom to concentrate on their scientific innovations, work with investors and "contribute to a healthier tomorrow.”
Higby noted Philadelphia is a growth hub for cell and gene therapy companies. "It is exciting to be located in the area where we can participate in the excellent growth and investment in human health," he said, adding the firm is also in active discussions with entrepreneurs in San Diego, San Francisco, Boston, and other life sciences growth centers.
Quick hits
Wilmington-based Incyte (NASDAQ: INCY) and Syndax Pharmaceuticals (NASDAQ: SNDX) of Waltham, Massachusetts, reported positive clinical trial results for their experimental treatment of chronic graft-versus-host disease in adult and pediatric patients. Based on the results, and a pending agreement from the FDA, Syndax and Incyte intend to submit a biologics license application for the new drug candidate, called axatilimab, to the FDA by the end of the year. … Harmony Biosciences Holdings (NASDAQ: HRMY) of Plymouth Meeting said it has entered into a new $185 million senior secured term loan deal with a bank syndicate. Harmony intends to use the net proceeds from the term loan and existing cash to repay the company's existing debt of $196.5 million and for related fees and expenses.
