The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation and fast track status to AGLE-102, a potential therapy for dystrophic epidermolysis bullosa (DEB) being developed by Aegle Therapeutics.
Rare pediatric disease designations are awarded investigative treatments for serious or life-threatening diseases of childhood affecting fewer than 200,000 people in the U.S. If AGLE-102 is ultimately approved, Aegle under this designation will get a voucher from the FDA that can be redeemed to obtain priority review of a subsequent investigational medication, or may sell it to another company.
Fast track status is intended to speed the development of investigational medications, open the treatment to priority review should it come up for approval, and provide additional opportunities for Aegle to consult with the regulatory agency.
“The FDA’s grant of Rare Pediatric Disease designation following its earlier grant of Fast Track Designation to AGLE-102 for DEB underscores the significant unmet medical need of children and adults living with this debilitating disease,” Shelley Hartman, Aegle’s CEO, said in a press release. “AGLE-102 has the potential to be the first multifaceted approach to treat this rare patient population.”
Mesenchymal stem cells (MSCs) are stem cells found in bone marrow that make and repair skeletal tissues. Like other stem cells, MSCs have the ability to differentiate into other cell types; they can also regulate the activity of nearby cells through the secretion of chemical messengers.
MSCs may promote other cells to make more type 7 collagen (COL7) — the protein that is defective in DEB due to mutations in the COL7A1 gene. As such, MSCs have been investigated as potential DEB therapies, with some promising results. However, donor-derived MSCs that can be used in therapy are costly to produce, difficult to obtain in quantity, and since MSCs are involved in many different biological processes, it can be hard to control their exact function once administered in the body.
AGLE-102 is a composite of MSC-derived extracellular vesicles isolated using a technology developed by Aegle. Extracellular vesicles are tiny sacks of cellular components — proteins, DNA, RNA, and other molecules — that are used to target cells.
The potential therapy uses these vesicles to restore production of normal COL7 in patients’ cells, without the “more complex issues” — like cell migration, differentiation and proliferation — “surrounding stem cell therapy,” the company reports.
Preclinical research has supported the potential of MSC-derived extracellular vesicles to stimulate COL7 production in cells.
“Aegle’s EV [extracellular vesicle] therapy is unique in that it delivers collagen 7 protein, COL7A1 [messenger RNA] and regenerative healing factors to potentially address the complex nature of DEB,” Evangelos Badiavas, MD, PhD, chief scientific officer at Aegle, said in a press release announcing the fast track status.
The company plans to open a Phase 1/2a trial (NCT04173650) in early 2021 assessing the safety and efficacy of AGLE-102, applied topically to lesions, against a placebo in people with DEB.
On top of a $4 million funding round announced early this year, Aegle recently finished a $6.5 million Series A financing round to advance its clinical pipeline. Tellus BioVentures was one of the investors.
“We are excited to support Aegle Therapeutics at this critical juncture in the company’s development. Aegle has built an excellent team with the capability of advancing AGLE-102, a unique combination of protein therapy, RNA therapy and regenerative medicine, into the forefront of cell-free therapy,” Lonnie Moulder, a Tellus founding partner, said in a related press release.
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