BALTIMORE, MD, January 5, 2021 – AsclepiX Therapeutics, Inc., a biopharmaceutical company using computational biology to identify potent peptide regulators of vascular and cellular homeostasis for the treatment of retinal and oncologic diseases, today 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).
“Dosing the first patient with AXT107 shortly after the acceptance of its IND is a significant milestone for AsclepiX, as it marks the firstclinical stage product and trial for our company,” said Steven Altschuler, MD, Chairman, AsclepiX Therapeutics, Inc. “AXT107 is the culmination of the vision, determination and hard work of the entire AsclepiX team; it’s an exciting time at AsclepiX as we advance our development plans.”
In December 2020, the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug application (IND) for AXT107 for the treatment of retinal diseases including DME, neovascular age-related macular degeneration (nAMD) and macular edema following retinal vein occlusion (RVO). AXT107 is an investigational drug candidate that inhibits VEGF-A and VEGF-C and activates Tie2. With its novel mechanisms of action, 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.
“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,” stated Jeffrey Heier, MD, Principal Investigator and Director of the Vitreoretinal Service and Director of Retina Research at Ophthalmic Consultants of Boston. “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.”
“With AXT107, patients may benefit from achieving sustained vision gains and extended duration of action, thereby reducing patient treatment burden,” said Theresa Heah, MD, MBA, Chief Medical Officer and Executive Vice President, Operations, AsclepiX Therapeutics, Inc. “We are excited to partner with leading retinal specialists to advance the AXT107 trials, and appreciate their dedication to continue to treat patients and participate in trials despite the ongoing pandemic.”
Existing therapies that inhibit VEGF have revolutionized the management of retinal vascular diseases, but are indicated for repeated intravitreal injections every 1-3 months. This creates a significant treatment burden, with many patients ultimately receiving fewer than the recommended number of injections, resulting in suboptimal visual outcomes. More importantly, a contingent of eyes fail to attain optimal visual outcomes even when receiving anti-VEGF therapy at recommended intervals. Due to its long half-life and unique intravitreal self-assembling gel depot formation, AXT107 can potentially be dosed as a yearly intravitreal injection, which could dramatically reduce the treatment burden associated with standard therapies.
About the CONGO Phase 1/2a Trial in DME
Congo is an open-label, dose-escalating, 48-week trial assessing the safety, tolerability, bioactivity and duration of action of a single intravitreal injection of 0.1mg (low dose), 0.25mg (mid-dose), or 0.5 mg (high-dose) of AXT107 in approximately 18 patients with DME.
AXT107 emerged from AsclepiX’s computational biology-based discovery platform and is derived from a cryptic peptide within collagen IV that works by activating naturally existing, homeostatic mechanisms of angiogenesis. The synergy of AXT107’s effect, via two validated pathways and three mechanisms of action, may offer improvement over anti-VEGF therapy alone.
Due to its long half-life and unique intravitreal self-assembling gel depot formation, AXT107 can potentially be dosed as a yearly intravitreal injection, which could dramatically reduce the treatment burden associated with standard therapies. The Tie2 effects complement those of the anti-VEGF action, potentially offering greater improvement in vision gains as well as reduction of vascular leakage and suppression of inflammation. AXT107 has demonstrated superiority to, and greater durability, than Eylea in animal models and has demonstrated safety in animal and toxicology studies for 15 months. The IND for AXT107 was submitted in November 2020 and was cleared by the FDA in December 2020.
About AsclepiX Therapeutics, Inc.
AsclepiX Therapeutics, Inc. is a biopharmaceutical company using computational biology to identify potent peptide regulators of vascular homeostasis that is disrupted in ocular and oncologic diseases. The novel clinical candidate peptides discovered by AsclepiX tap into these naturally existing self-regulating mechanisms, evolved over millions of years, that the body uses to maintain homeostasis and thus restore and maintain health. AXT107, an investigational product candidate and the lead clinical candidate, has a unique mechanism of action that targets two clinically proven pathways of diseases of the retina. For more information, please visit: www.asclepix.com
Diabetic macular edema (DME) is caused by a complication of diabetes called diabetic retinopathy. DME is a build-up of fluid in the macula, resulting in swelling and thickening which distorts vision near or in the center of your field of vision. It is estimated that 7.7 million Americans have diabetic retinopathy, and of those, about 750,000 also have DME.
Anti-VEGF intravitreal injections are the standard of care for DME, which many patients are indicated for monthly injections due to the chronic progression of diabetes and DME. This creates a significant treatment burden, with many patients ultimately receiving fewer than the recommended number of injections, resulting in suboptimal visual outcomes.
This press release contains “forward-looking statements” concerning the development of AsclepiX Therapeutics, Inc. products, the potential benefits and attributes of such products, and the company’s expectations regarding its prospects. Forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual future events or results to differ materially from such statements. These statements are made as of the date of this press release. Actual results may vary. AsclepiX Therapeutics, Inc. undertakes no obligation to update any forward-looking statements for any reason.
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SOURCE: AsclepiX Therapeutics, Inc