AsciepiX Therapeutics Inc., based in Baltimore, Maryland, has announced that the first patient has been dosed in the Phase 1/2a “CONGO” clinical trial to evaluate the safety and bioactivity of AXT107 in patients with Diabetic Macular Edema (DME). The lead candidate, AXT107, inhibits VEGF-A and VEGF-C and activates Tie2 for several of retinal disorders and the study (NCT04697758) is aimed to enrol up 18 participants across ten sites in the US.
AsclepiX uses computational biology to identify peptide regulators of vascular homeostasis to focus on retinal and oncologic diseases. The company is aimed to building clinical candidate peptides discovered by mining the human proteome for peptide sequences to identify novel anti-angiogenic peptides. According to the researchers in the company, anti-angiogenic peptide sequences use a wide array of protein types to target drug candidates including structural proteins, pro-angiogenic proteins, chemokines, metalloproteases, and growth hormones. Several peptides are currently aimed at testing in vitro assays to show inhibited neovascularization in ocular models and the researcher’s goal is to progress their pipeline studies into clinical trial research. The lead drug uses AXT107 as a synthetic 20-mer peptide derived from non-collagenous sequences of the collagen IV protein, normally released during the wound healing process to turn off angiogenesis. Anti-VEGF approvals for several ocular treatments have shown clear therapeutic benefit and this class of treatments has now spurned new multiple commercial opportunities.
According to the company, the U.S. Food and Drug Administration (FDA) accepted an Investigational New Drug application (IND) for AXT107 in December 2020. Novel mechanisms of action propose that AXT107 has the potential to be a standard of care monotherapy. AXT107, if approved, can provide patients with greater clinical benefit coupled with convenient once-a-year dosing, which could transform the treatment of retinal diseases. Commenting on the achievement, Dr. Jeffrey Heier, MD, a Principal Investigator and a Director of Retina Research at Ophthalmic Consultants of Boston stated that, “as diabetes is on the rise globally, we have an increasing number of DME patients. The current standard of care is frequent injections with anti-VEGF therapies, but significant challenges for physicians and patients remain. We are hopeful that AXT107’s unique mechanisms of action, as a peptide and intravitreal self-forming gel depot, will improve outcomes and decrease patient and caregiver burden for our DME patients.”