CHAPEL HILL, N.C., Dec. 21, 2015 -- Achelios Therapeutics announced today it will present the results of a Phase IIa placebo-controlled clinical trial in moderate and severe migraine sufferers treated with TOPOFEN™, the company’s proprietary topical anti-migraine therapy. The data demonstrate that the simple application of a well-known non-steroidal anti-inflammatory drug (NSAID), using the company’s proprietary formulation on the skin, over the trigeminal nerve branches in the temple and neck area of the face, can be a safe and effective alternative treatment for patients suffering from acute migraine.
William R. Bauer M.D., Ph.D., FAAN, a neurologist and migraine headache specialist and advisor to Achelios, says, “Migraine remains a significant global cause of disability and disruption of activities of daily living. TOPOFEN™ results show promise to provide a safe and effective treatment that will positively impact the disabling effects of such a condition.”
The results of the Phase IIa clinical trial will be presented at 9:15 a.m. on Jan. 11 at Biotech Showcase 2016, an investor and networking conference, in San Francisco. The study was conducted by scientists from Achelios and the Michigan Headache & Neurological Institute.
Wolfgang Liedtke, M.D., Ph.D., of Duke University is a member of the team that conducted the research and a paid advisor to Achelios and an associate professor of neurology and attending physician in the Duke University Pain Clinics, says, “The results of the study are encouraging, and those of us who treat migraine think they may lead to a meaningful alternative treatment for a substantial number of migraineurs. This study showed that it may be possible to affect severe migraine, which can be a debilitating neurological pain condition, with a topical application to facial trigeminal nerve endings, which was unexpected.”
Crist Frangakis, Ph.D., president and CEO of Achelios Therapeutics, the study sponsor, says, “The positive results of this study provide preliminary evidence that migraines can be effectively treated topically without the safety and side effects challenges that often accompany systemic oral medications. Serious side effects of NSAIDs, such as stomach bleeding and ulcers, result in more than 100,000 hospitalizations and thousands of deaths each year in the U.S. With TOPOFEN™ physicians and patients, especially those with cardiovascular risks, will have a new treatment option.”
The randomized, crossover, double-blind, placebo-controlled study involved 48 screened adults with a history of episodic migraine with and without aura (the warning signs that can precede headache pain in about 20 percent of sufferers). Patients were instructed to treat five moderate to severe migraines by applying the TOPOFEN™ gel on the skin, over the three branches of the trigeminal nerve, and to record their symptoms on an electronic diary.
Compared with placebo, TOPOFEN™ resulted in greater improvement in pain assessments after study drug application, a faster time to pain response, a reduction of migraine-associated symptoms such as nausea, light and sound hypersensitivity, and greater suppression of pain over a 24-hour period.
Of the severe migraine patients, 45 percent had sustained pain relief from 2 to 24 hours compared to 15 percent of placebo. Also 50 percent of patients who treated their severe pain with TOPOFEN™ were pain free at 24 hours compared to 25 percent of placebo-treated patients. Patients whose severe headaches were treated with TOPOFEN™ were at least three times as likely to experience complete relief of associated symptoms (nausea and photophobia) versus placebo. Based on the duration of action and safety demonstrated in this study, TOPOFEN™ may also be an excellent candidate for use in migraine prevention.
TOPOFEN™, also known as ELS-M11, is a proprietary gel formulation consisting of 5 percent ketoprofen, a potent NSAID. The gel formulation was developed to permeate human skin rapidly and efficiently.
At least 37 million Americans suffer from migraine, according to the National Headache Foundation. The socio-economic cost burden is estimated to exceed $25 billion annually.
Migraine is believed to occur when brain cells trigger the trigeminal nerve to release neuropeptides that increase inflammation and make the dura mater – a membrane surrounding the brain – hypersensitive. The trigeminal nerve is a major pain pathway that supplies all sensory innervation for the head, face and all adjacent sentient structures such as teeth, eyeball, sinuses, and, importantly for headaches such as migraine. All of these structures can activate pain-sensing trigeminal nerve endings. Migraine pain is typically felt on one side of the face, around the eye or temple.
Achelios Therapeutics, established in 2012 in Chapel Hill, N.C., is a privately held, pharmaceutical-development company focused on developing topical drugs for the treatment of migraine and peripheral pain. The company’s name (Achelios) is derived from the mythological Greek moon goddess “Achelois,” which loosely translates as “she who washes away pain.”
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