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Achelios Gets FDA Guidance on Approval Path for Migraine Drug

By Barry Teater, NCBiotech Writer

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Achelios Therapeutics of Chapel Hill has completed a meeting with the U.S. Food and Drug Administration about a path to approval for its leading drug candidate, TOPOFEN, to treat acute and chronic migraine, a condition known for its debilitating pain.

"This new FDA guidance confirms our previous interactions with the agency, provides for a streamlined development path to approval, and is consistent with our previous guidance and with the FDA regulations for a 505(b) 2 New Drug Application (NDA)," John G. Fort, M.D., MBA, the company's chief medical officer, said in a news release. 

"Based on the streamlined clinical trial requirements, Achelios will be able to achieve an NDA filing in a relatively quick and cost-effective manner compared to what is typically required for a new chemical entity (NCE),” Fort said.

In the so-called “Type C” meeting with the FDA, Achelios sought confirmation of the requirements for Phase 3 and NDA submissions for TOPOFEN. 

A recently completed Phase 2a study demonstrated that the topical application of TOPOFEN to facial areas with trigeminal nerve branches may be a safe and effective alternative treatment for patients suffering from acute migraine, the company said. The trigeminal nerve is a major pain pathway that supplies all sensory innervation for the head, face and adjacent structures such as teeth, eyeball and sinuses.

"The results of the study are encouraging, and those of us who treat migraine think it may lead to a meaningful alternative treatment for a substantial number of migraineurs,” said Wolfgang Liedtke, M.D., Ph.D., a tenured professor of neurology, anesthesiology and neurobiology and attending physician at Duke University. Liedtke was a member of the team that conducted the research and is a paid advisor to Achelios.

NCBiotech funding helped drive development

The Phase 2a study was supported in part by a $250,000 Small Business Research Loan from the North Carolina Biotechnology Center. Liedtke also received a $100,000 Collaborative Funding Grant from the Center for earlier work on TOPOFEN at Duke.

TOPOFEN is a proprietary gel formulation of ketoprofen, a well-known non-steroidal anti-inflammatory drug (NSAID), a widely used class of drugs that relieve pain, reduce fever and stem inflammation. The gel formulation was developed to permeate human skin rapidly and efficiently.

The ability to treat migraine topically would avoid the safety and side effects challenges that often accompany systemic oral NSAIDS. Serious side effects of NSAIDs, such as stomach bleeding and ulcers, cause more than 100,000 hospitalizations and thousands of deaths each year in the United States.

Currently there are no approved topical NSAIDs indicated for the treatment of migraine.

Migraine is believed to occur when brain cells trigger the trigeminal nerve to release neuropeptides that increase inflammation, dilate blood vessels and make the dura mater – a membrane surrounding the brain – hypersensitive. Migraine headaches are typically on one side of the head, felt around the eye or temple.

Migraine affects at least 37 million Americans

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.

"TOPOFEN represents a novel approach for the acute treatment and prophylaxis in chronic migraine, with unique benefits for the patient and importantly, given the intellectual property that has been both filed and granted, a patent-protected opportunity for a potential partner,” said Crist Frangakis, president and CEO of Achelios.  “We believe TOPOFEN provides great value given its low required development costs and accelerated time to market relative to other potential new therapies.”

In addition to migraine treatment, TOPOFEN has potential use in a variety of pain disorders including temporomandibular joint disease, post-operative inflammatory pain, chemotherapy-induced neuropathic pain and joint arthritis pain, Frangakis said.

Achelios, established in 2012, is a privately held, pharmaceutical-development company focused on developing innovative topical drug-delivery technologies.

The company’s name is derived from the mythological Greek moon goddess Achelois, which loosely translates as “she who washes away pain.”

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