Greenwood Village, Colo., U.S.A. – Ampio Pharmaceuticals, a clinical stage biopharmaceutical company developing drugs for the treatment of inflammation, eye disease, kidney disease, CNS disease, metabolic disease and male sexual dysfunction have 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 31 2012 seeking guidance for a clinical trial path for Optina. A summary of the meeting is as follows:

• The meeting followed a letter from the FDA providing guidance for the path of approval of Optina in the USA.
• The FDA agreed to Ampio’s proposed indication for Optina 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.
• The proposed study, agreed by the FDA to be conducted by Ampio, will be based on similar end points as the intraocular injectable drug, Lucentis

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.

“We are pleased that the injectable drug Lucentis is likely to be approved and made available to the underserved DME patients. We believe Optina may provide additional advantages to these patients, as Optina is administered orally and is based on a very low dose of a previously approved drug, danazol. Extensive clinical experience with the drug has allowed the FDA to permit Ampio to use the 505(b)2 path for drug registration.

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. Complications such as eye infection, increased intra ocular pressure (IOP) and local eye irritation reported in the Lucentis studies are NOT a concern for Optina.”

Michael Macaluso, Ampio’s CEO, reflected on the achievement of this latest milestone, “One year ago we selected three 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.

These preparatory clinical trials took place in Europe (Zertane), Australia (Ampion) and Canada (Optina). With this latest meeting with the FDA, we have met our interim goal. We now have two drugs poised for pivotal trials (Zertane and Ampion) and Optina is approved for a 505 (b) 2 pathway in the USA.”

About the Canadian Clinical Trial

The Optina™ 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 Optina™ in adult patients with Diabetic Macular Edema (DME).  Three Optina™ doses were studied and administered orally twice daily.

Patients were randomized into one of the three Optina™ 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.

About Optina ™

Ampio is developing Optina™, an orally administered low dose of danazol, for the treatment of diabetic macular edema (DME).  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.

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.  Macular edema of the eyes is often an early form of diabetic retinopathy that causes significant vision loss.  Almost all Type 1 diabetics and more than 60% of Type 2 diabetics develop retinopathy.

There is no effective daily drug treatment for diabetic retinopathy other than general measures, such as controlling blood sugar, hypertension, blood lipids, etc.  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.

About Ampio

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.
Image Courtesy of  Public Citizen

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The Food and Drug Administration (FDA) approved a new drug, Horizant Extended Release Tablets, on Wednesday to treat moderate to severe primary Restless Legs Syndrome in adults.  Horizant is a patented new chemical entity discovered and developed by both GlaxoSmithKline and XenoPort.

Restless Legs Syndrome affects up to 10 percent of the U.S. population and is a disorder of the nervous system that causes an urge in the body to move one’s legs.  This disorder is classified as a sleeping disorder because it affects and interferes with a person’s sleeping habits.  The irresistible need to move one’s legs in attempt to relieve the sensations is often described as uncomfortable, “pins and needles,” or a “creepy crawly” feeling.  The sensations are often made worse when laying down, sitting or when legs are trying to rest.

“People with Restless Legs Syndrome can experience considerable distress from their symptoms,” Russell Katz, MD, director of the division of neurology products in the FDA’s Center for Drug Evaluation and Research, states in an FDA news release.  “Horizant provides significant help in treating these symptoms.”

The drug was approved based on two studies that were done in more than 2,300 adults.  These 12-week studies showed improvement in Restless Legs Syndrome patients trying Horizant that was considerably greater than those patients who were receiving a placebo.  Patients received oral daily doses in a range from 600 to 3,600 mg.

According to the full Prescribing Information provided on Horizant’s website, the recommended doze of this drug is 600 mg once daily taken with food.  The drug is not to be cut, crushed or chewed. Horizant is a prodrug of gabapentin and is described as an acetic acid.  It’s molecular formula is C16H27NO6.

This drug is administered orally and contains 600 mg of gabapentin enacarbil and the following other inactive ingredients: colloidal silicon dioxide, dibasic calcium phosphate dihydrate, glyceryl behenate, magnesium sterate, sodium lauryl sulfate, and talc.

The FDA states that Horizant “may cause drowsiness and dizziness and can impair a person’s ability to drive or operate complex machinery.”  Horizant’s website also confirms that this drug causes significant driving impairment.  It is an antiseizure medicine, and like all drugs of this type, Horizant holds a warning label about the risk of suicidal thoughts and actions in a small number of people.

According to a news release from GlaxoSmithKilne and XenoPort, do not take Horizant if you sleep during the daytime and remain awake at night.

This is a significant milestone in XenoPort and GlaxoSmithKline’s efforts to develop a non-dopaminergic therapy.  “We hope that we can continue to develop other important medicines that can provide meaningful treatment benefit to sufferers of central nervous system disorders,” Ronald Barett, CEO of XenoPort said.

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