Lab Notes: Helius launches flagship product in U.S.; Virpax reports positive preclinical results for seizure drug

This week's Philadelphia-area life sciences industry news includes a Bucks County medical device company's launch of its flagship product in the United States, a Chester County firm's progress on an experimental cannabidiol seizure medicine, a Montgomery County pharmaceutical company's pursuit of a second approval for its lead product, and more.

Here's the roundup:

Helius Medical Technologies

The Bucks County neurotech company's flagship product hit the market in the United States this week.

The portable neuromodulation stimulator, or PoNS, received Food and Drug Administration marketing clearance last year to help patients with multiple sclerosis with gait deficit improve their walking ability.

“Approximately 40% of individuals with MS will need walking assistance within 15 years of the onset of the disease," said Dr. Antonella Favit-Van Pelt, the company's chief medical officer. "PoNS therapy provides a unique opportunity for these patients to improve gait functionality and mobility”

The device, approved in Canada since 2019, 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.

The FDA approval specifies that the PoNS is to be used as an adjunct to a supervised therapeutic exercise program in patients 22 years of age and older. It is available by prescription only. The company said it fulfilled its first PoNS prescription on April 27.

Newtown-based Helius (NASDAQ: HSDT) is also studying the use of the technology to treat gait and balance deficits resulting from a stroke.

Founded in 2014, Helius generated revenue of $552,000 and posted a net loss of $18 million in 2021. The company has 19 employees.

Virpax Pharmaceuticals

The Berwyn pharmaceutical company said it successfully completed its initial preclinical studies for VRP324, its experimental treatment for the management of seizures in children and adults.

In Virpax's preclinical pharmacokinetics study, VRP324 was able to deliver cannabidiol to the brain after nasal administration in a rodent model. Nasal delivery of VRP324 provided high concentrations of CBD in the brain, and, the company said, the preclinical studies confirm that there were higher levels of CBD in the brain versus the plasma.

VRP324 uses a preassembled device and cartridge to propel the MET-CBD powder formulation into the nose via the olfactory nerve.

“The results from this preclinical study met our expectations," said Anthony P. Mack, founding chairman and CEO of Virpax (NASDAQ: VRPX). "We are encouraged that our molecular envelope technology may offer a more effective and efficient delivery method than the approved oral formulation, especially for children who suffer from seizure disorders."

Mack said the company is preparing a pre-investigation new drug application briefing document for the FDA as the first step toward seeking the federal agency's approval to study the new drug candidate in human patients.

The product candidate has been formulated to potentially treat seizures associated with tuberous sclerosis complex, Lennox-Gastaut syndrome and Dravet syndrome in patients 1 year of age and older.

Harmony Biosciences

The Plymouth Meeting pharmaceutical company began late-stage clinical testing of pitolisant in adult patients with idiopathic hypersomnia.

Pitolisant is the active ingredient in Wakix, Harmony's treatment for excessive daytime sleepiness associated with narcolepsy that was approved by the FDA in 2019.

Idiopathic hypersomnia is a rare and chronic neurological disease that is characterized by excessive daytime sleepiness despite sufficient or even long sleep time.

Dr. Jeffrey Dayno, Harmony's chief medical officer, said both the patient community and health care professionals have expressed interest in looking at pitolisant as a treatment for idiopathic hypersomnia because of its novel mechanism. He said pitolisant is designed to increase the synthesis and release of histamine, an important wake-promoting neurotransmitter in the brain.

John C. Jacobs, CEO of Harmony (NASDAQ: HRMY), said if the Phase 3 study is successful the company will seek an additional regulatory approval for Wakix.

iEcure

The Philadelphia gene editing company focused initially on treating liver disorders expanded its research team with the appointment of Dr. George A. Diaz as vice president and therapeutic area lead for urea cycle disorders.

Urea cycle disorders are conditions characterized by inborn errors of metabolism that result from defects in one of the enzymes or transporter molecules involved in the liver's removal of ammonia from the bloodstream.

“George is a key hire as we solidify the clinical strategies for our gene editing programs," said Joseph Truitt, iEcure's CEO.

Diaz has more than 25 years of experience in genetics and pediatrics, particularly in the areas of metabolic diseases and monogenic disorders. He most recently was chief of the division of medical genetics, and a professor in the departments of genetics & genomic sciences and pediatrics, at Icahn School of Medicine at Mount Sinai in New York.

Founded in 2020, iEcure is collaborating with the University of Pennsylvania’s gene therapy program.