Development of a mimetic multimodal peptide for the treatment of macular edema

Period of Performance: 09/01/2015 - 08/31/2016


Phase 1 SBIR

Recipient Firm

Asclepix Therapeutics, LLC
Lutherville, MD 21093
Principal Investigator


DESCRIPTION (provided by applicant): Macular edema (ME) is a common disease secondary to retinal vein occlusion and both Type I and Type 2 Diabetes. It is the leading cause of blindness in people between the ages of 20-74. Lucentis and Eylea have been approved for the treatment of ME. Although effective, they do not increase the visual acuity for about half the patients suffering from ME. We have developed ACX107, a 20-mer peptide with remarkable activity against a host of pro-angiogenic growth factors, which we believe will improve vision more effectively and in more patients because of its broad anti-angiogenic activity. We have found that ACX107 inhibits choroidal neovascularization, retinal neovascularization, causes neovascular regression, inhibits retinal detachment, and dramatically inhibits VEGF induced vascular leakage in mouse and rabbit models. Remarkably the peptide appears to work even 1 month after a single intravitreal injection. These results suggest that ACX107 could be the next generation drug for the treatment of ME. Here we propose to complete a dose ranging study as well as a duration of activity study, to develop an analytical method to quantify ACX107, and do IND enabling toxicology studies. What we have proposed here is the portion of the full development work that we can accomplish on the budget and time confines of the Phase I SBIR mechanism. The rest of the development work will require significantly more funding which we could accomplish with a Phase II SBIR or other investment like that of a strategic partnership.