Aegle Therapeutics has raised $4 million to fund the first clinical trial evaluating AGLE-102, a therapy candidate for people with dystrophic epidermolysis bullosa (DEB) based on the company’s extracellular vesicle therapy.
The Phase 1/2 trial (NCT04173650) is anticipated to begin in the first half of this year after receiving clearance from the U.S. Food and Drug Administration. The study, which is not yet recruiting, will be conducted in the U.S. and is expected to include 30 participants.
“We are very excited to close this first institutional financing round with such knowledgeable, high-caliber biotech investors,” Shelley Hartman, Aegle’s CEO, said in a press release. “The new funding validates our business plan and allows us to advance AGLE-102 into the clinic as well as expand the capabilities and opportunities of this cutting-edge platform.”
Mesenchymal stem cells (MSCs) can be isolated from several tissues and have the ability to develop into a variety of cell types. Donor-derived MSCs have been explored as a treatment for DEB, but the effects of these cells when placed in living tissues are hard to control and their production is costly.
To overcome these limitations, Aegle has turned to extracellular vesicles, which are small vesicles released by MSCs. Extracellular vesicles can transport and deliver wound healing “messages” in the form of proteins, genetic material (e.g., RNA, DNA), and other molecules to target cells.
The vesicles are collected from a donor and administered to patients. They are released by bone marrow-derived MSCs from the donor, and isolated using a technology developed by Aegle that is meant to preserve vesicles’ quality.
According to the company, this opens up the possibility of harnessing the healing power of MSCs in a cell-free manner while enabling higher production and lower handling costs.
AGLE-102 uses these extracellular vesicles to restore production of type VII collagen (COL7) — the defective protein in patients with DEB — and accelerate wound healing.
In preclinical studies, these vesicles demonstrated a similar regenerative capacity as donor-derived MSCs and succeeded in making affected cells start producing their own functional COL7.
“We are very excited to participate in advancing this pioneering research into the clinic. We expect that in addition to DEB, there are many other potential applications for this platform technology,” said David Schimmel, a member of New World Angels who will join Aegle’s board, together with Lonnie Moulder at Tellus BioVentures and Elona Baum of DEFTA Healthcare Technologies.
Aegle was one of the three companies recently recognized for its efforts to treat DEB at the 21st Annual Debra of America Benefit, held recently in New York City. The others were Amryt Pharma and Lenus Therapeutics.