Special Report: Biotech company Exopharm has begun its second human clinical trial using extracellular vesicles isolated from human platelets, solidifying its leadership position in the field of exosome therapeutics.

Up to 15 patients will receive the exosomes in the PLEXOVAL II study, which is testing the exosomes for safety and benefits in wound healing.

The patients will receive Exopharm’s Plexaris exosome product following skin punch biopsies, primarily to make sure the treatment is safe. Wound closure and scarring activity will also be followed.

Plexaris is part of Exopharm’s naïve extracellular vesicle (NEV) program. Exosomes are excreted by stem cells and Exopharm says they show great promise as regenerative medicine across a range of age-related medical conditions including mobility problems such as osteoarthritis and sensory functions such as hearing loss.

Exopharm was granted Human Research Ethics Committee approval to begin the healing study under the Australian Clinical Trials Notification scheme in September.

“EV medicines are new, not only for clinicians, but for regulators as well. We have a very clear, step-wise clinical pathway for validating our manufacturing and addressing any safety issues of EV products,” says Dr Angus Tester, Exopharm’s head of product evaluation.

“With a successful outcome from PLEXOVAL II, Exopharm will be well-positioned to initiate Phase 2 studies with off-the-shelf Plexaris and Phase 1 studies with our mesenchymal stem cell EV product Cevaris.”

A world-first

Alison Mew, director of manufacturing and development at Exopharm, says that all the exosome doses for the PLEXOVAL II study came from a pool of donor platelets.

“As far as we know, this is the largest scale clinical grade manufacture of an EV product performed anywhere,” Mew said.

“It is further evidence of the manufacturing capability of Exopharm.”

Dosing for the randomised, double-blinded, placebo-controlled phase 1 study will continue in October and November, and the study is on track for completion this calendar year.

Exopharm has decided to terminate recruitment into its PLEXOVAL I study, which was suspended earlier this year due to COVID-19 restrictions. The PLEXOVAL II study is more advanced and uses off-the-shelf exosomes rather than those derived from the same patient.

$2.1 million R&D tax refund received

Exopharm also announced it has received a $2,110,891 cash refund from the Australian Tax Office under the Federal Government’s Research and Development Tax Incentive scheme.

“This is a substantial increase on the previous year’s refund and is indicative of the levels of innovation occurring at Exopharm,” said Dr Chris Baldwin, Exopharm’s chief commercial officer.

“With the R&D Rebate and the proceeds of the recent $10m capital raise coming in, Exopharm’s cash position is now much improved and we see a runway through to the end of CY 2021 in our present budget.”

This article was developed in collaboration with Exopharm, a Stockhead advertiser at the time of publishing.  

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.