Today, Ampio Pharmaceuticals, the dynamic biopharma developer which has assembled an impressive portfolio of proprietary drugs for treating eye (diabetic macular edema – Optina™), kidney, and metabolic disease, as well as chronic inflammation (Ampion™), male sexual dysfunction (Zertane™), and central nervous system disease, reported reception of a notice of allowance for a patent from the United States Patent and Trademark Office covering Optina, also receiving a similar allowance from the Canadian Intellectual Property Office.
Diabetic macular edema (DME – fluid leaking into the area of the retina that provides sharp vision directly in front of you) and the diabetic retinopathy (the actual breaking down of tissues in the retina due to Type 1/2 diabetes that leads to) which causes it, are a serious problem resulting in vision loss and eventually blindness. There is no effective daily drug treatment and retinopathy occurs in almost all Type 1 cases (over 60% in Type 2) other than to regulate the conditional parameters (controlling blood sugar, blood lipid levels, hypertension, and the like), which exacerbate the fundamental problem.
Diabetic retinopathy is a massively underserved market that has exploded alongside the global diabetes epidemic, with the only options being unavailable in US/Canada and piecemeal at best (laser therapy and injection/implantation of drugs to/around the tissues).
So here is Optina, an ultra-low dose oral capsule form of danazol (the biphasic effect of which has been shown in endothelial cell permeability and f-actin cytoskeleton dynamics – Biochemical Biophysical Research Communications, Apr 2012), which basically tightens up the tissues that otherwise degrade. This biphasic effect of danazol (increasing or decreasing permeability at lower or higher concentrations) detailed in the BBRC publication (the first of what is to be a series), allows for modulated permeability. The mode of action then in Optina is essentially a modulation of the F-actin endoskeleton of the endothelial cells (which line the inside of blood vessels in the retina), resulting in better overall cohesion to the peripheral, or cortical actin, meaning the cell junctions are tight and leakage is reduced or eliminated.
AMPE has secured its footprint for Optina in North America with this move and has the requisite global patent ball rolling as well, with multiple additional patents pending worldwide. Since danazol (a derivative of the synthetic steroid ethisterone) was approved by the FDA back in the 1970s (for endometriosis, and more recently for chronic indications like fibrocystic disease of the breast, hereditary angioedema, and ITP), there is a very solid grounding for acceptance of the associated vascular dynamics.
CEO of AMPE, Michael Macaluso, emphasized the uncontested position of the drug, with no other oral drug being commercially available, or even known to exist for treatment of this condition. Macaluso cited the “unusually long” patent life spans granted (doesn’t expire until 2030), contrasting them with most other drugs at the same position on the timeline, just before a pivotal clinical trial, and staring at the very end of their patent life rapidly approaching like a freight train. Macaluso commented on ongoing discussions with potential partners to co-develop Optina, clearly excited about the DME potential and huge, underserved/unnerved market full of people who are at risk of going blind without such innovative products.
Chief Regulatory Officer at AMPE, Dr. Vaughan Clift, underscored the significance of the double masked, placebo controlled clinical study of Optina at Saint Michael Hospital in Toronto, which validated the in vitro findings regarding the extremely valuable function of a low dosage, as well as the inverse effect from a high dosage. The CRO is completely vetting the data from the Optina trial and AMPE intends to publish results via the proper venues after meeting with FDA to do a pre-IND run down of pivotal trials in the U.S. under 505(b)2 registration.
For more information on today’s patent announcement, or to stay up to date with the latest results out of this constantly innovating biopharma, please visit the Ampio Pharmaceuticals, Inc. website at: www.AmpioPharma.com
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