Ampio Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing drugs for the treatment of inflammation, eye disease, kidney disease, CNS disease, metabolic disease and male sexual dysfunction today announced that a pre-IND meeting with the FDA's ophthalmology division of the Center for Drug Evaluation and Research (CDER) took place on July 31st 2012 seeking guidance for a clinical trial path for Optinaâ„¢.Â A summary of the meeting is as follows:
The meeting followed a letter from the FDA providing guidance for the path of approval of Optinaâ„¢ in the USA.
The FDA agreed to Ampio's proposed indication for Optinaâ„¢ as a drug for the treatment of Diabetic Macular Edema (DME) in adults.
The FDA agreed that the approval process through the 505(b)2 pathway is acceptable.
The FDA raised no safety or CMC concerns.
The FDA agreed that no additional non-clinical studies are necessary and that guidance for the dose and inclusion/exclusion criteria for this larger trial may be drawn from the recently completed successful Canadian trial.Â
The proposed study, agreed by the FDA to be conducted by Ampio, will be based on similar end points as the intraocular injectable drug, Lucentisâ„¢
Dr. Vaughan Clift, Ampio's Chief Regulatory Officer, noted: "We are now able to launch a larger study using visual acuity rather than Optical Coherence Tomography (OCT) as the measure of efficacy and we are able to adjust the dosage to body mass index (BMI) to achieve the optimal benefit.Â We are pleased that the injectable drug Lucentisâ„¢ is likely to be approved and made available to the underserved DME patients.Â We believe Optinaâ„¢ may provide additional advantages to these patients, as Optinaâ„¢ is administered orally and is based on a very low dose of a previously approved drug, danazol. Â Extensive clinical experience with the drug has allowed the FDA to permit Ampio to use the 505(b)2 path for drug registration.Â The 505(b)2 is a significantly shorter and less expensive path than those of new chemical entities. There is an obvious advantage to an oral drug vs. one that requires injection directly into the eye.Â Complications such as eye infection, increased intra ocular pressure (IOP) and local eye irritation reported in the Lucentisâ„¢ studies are NOT a concern for Optinaâ„¢."
Michael Macaluso, Ampio's CEO, reflected on the achievement of this latest milestone, "One year ago we selected three (3) drugs from our extensive pipeline and set as our goal the advancement of each drug through carefully managed clinical paths into late stage pivotal trials that are within our means to finance, are relatively short in duration and in full compliance with FDA guidance. Â These preparatory clinical trials took place in Europe (Zertaneâ„¢), Australia (Ampionâ„¢) and Canada (Optinaâ„¢). With this latest meeting with the FDA, we have met our interim goal.Â We now have two drugs poised for pivotal trials (Zertaneâ„¢ and Ampionâ„¢) and Optinaâ„¢ is approved for a 505 (b) 2 pathway in the USA."
About the Canadian Clinical Trial
The Optinaâ„¢ trial, conducted at St Michael Hospital in Canada, was a phase II, placebo-controlled, double-masked study to evaluate the efficacy and safety of a 12-week treatment with oral Optinaâ„¢ in adult patients with Diabetic Macular Edema (DME).Â Three Optinaâ„¢ doses were studied and administered orally twice daily. Patients were randomized into one of the three Optinaâ„¢ arms, or placebo. The primary endpoint was change from baseline to week twelve of treatment in retinal thickness as measured by Optical Coherence Tomography (OCT). Secondary endpoints were 1) change from baseline to week twelve in retinal volume and 2) change from baseline to week twelve in Early Treatment. The study results indicate a clinically meaningful reduction in edema at the appropriate dosage and a parallel improvement in visual acuity.Â
Ampio is developing Optinaâ„¢, an orally administered low dose of danazol, for the treatment of diabetic macular edema (DME).Â Ampio's Chief Scientific Officer discovered an unexpected activity in his investigation of the mechanism of action of danazol: very low doses reversed inflammation induced increases in the permeability of blood vessels, thus reducing vascular leakage.Â Dr. Bar-Or subsequently managed In Vitro studies that suggested this effect may reduce the vasogenic edema produced from multiple diseases, including diabetic macular edema which can impair vision and cause blindness (Diabetic Macula Edema, DME). The specific Optina dosages are proprietary and subject to multiple patent filings.
Danazol, a derivative of the synthetic steroid ethisterone, was approved by the FDA in the 1970s for endometriosis and, more recently, for other chronic indications, including hereditary angioedema.
About Diabetic Retinopathy & Diabetic Macular Edema:
Diabetic retinopathy is damage to the retina of the eyes caused by complications of diabetes mellitus and resulting in loss of vision.Â Macular edema of the eyes is often an early form of diabetic retinopathy that causes significant vision loss.Â Almost all Type 1 diabetics and more than 60% of Type 2 diabetics develop retinopathy.Â There is no effective daily drug treatment for diabetic retinopathy other than general measures, such as controlling blood sugar, hypertension, blood lipids, etc.Â Due to the global diabetes epidemic, diabetic retinopathy is recognized as a high, unmet medical need that would benefit from having oral therapeutic options available.
Ampio Pharmaceuticals, Inc. develops innovative proprietary drugs for inflammation, eye disease, kidney disease, CNS disease, metabolic disease and male sexual dysfunction. The product pipeline includes new uses for previously approved drugs and new molecular entities ("NMEs"). By concentrating on development of new uses for previously approved drugs, approval timelines, costs and risk of clinical failure are reduced because these drugs have strong potential to be safe and effective while their shorter development times can significantly increase near term value. A key strategy includes actively exploring partnership, licensing and other collaboration opportunities to maximize Ampio's product development programs. For more information about Ampio, please visit our website, www.ampiopharma.com.
Ampio's statements in this press release that are not historical fact and that relate to future plans or events are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by use of words such as "believe," "expect," "plan," "anticipate," and similar expressions. These forward-looking statements include risks associated with clinical trials, expected results, regulatory approvals, successful commercialization and marketing of Zertaneâ„¢ and the combination drug in Korea, and changes in business conditions and similar events. The risks and uncertainties involved include those detailed from time to time in Ampio's filings with the Securities and Exchange Commission, including Ampio's Annual Report on Form 10-K and Quarterly Reports on Form 10-Q.