There are only so many Advils a migraineur can pop before the ulcers and GI bleeds start setting in. They may be effective pain drugs, but NSAIDs are notorious for causing gastrointestinal distress – particularly when taken in the high doses needed to treat migraines.
North Carolina specialty pharma startup Achelios Therapeutics has built a platform that, in the form of a topical gel, allows NSAIDs to be absorbed through the skin – largely avoiding entry into the bloodstream and irritation of the GI tract. When placed on the face, near the ear and above the trigeminal nerve, the medicine – called Topofen – can be directed straight to the region that causes the migraine.
The company just announced Phase 2a results of Topofen among 40 patients, finding it to be rather effective in curbing migraines – 77 percent of participants felt pain relief, and 45 percent felt it for two to 24 hours.
CEO Crist Frangakis explained that Achelios’s drug isn’t meant to replace the current best-in-show migraine therapy, a class of drugs called triptans, but rather to complement them. Triptans cause vasoconstriction and can’t be used in folks with cardiovascular issues – up to 30 percent of the migraine population, Frangakis said.
Since its 2012 launch, Achelios has received about $7 million in angel funding, Frangakis said. It’s aiming to raise $30 million now, looking to venture capital or pharma partnerships to get it to the next step.
Its ask: $12 million would go toward two more Phase 2 trials – one to test the company’s delivery system prophylactically, and the other to flesh out the Phase 2a trial as a migraine treatment. About $2 million of that would be geared toward toxicology studies in animal models. It’d want an second, $18 million tranche to conduct Phase 3 trials – because, as Frangakis said, most big pharma isn’t interested in partnering out on specialty pharma like Achelios’ until it’s up to Phase 3.
The company’s exploring ways to use the pain drug delivery platform in other conditions, such as in orphan pain conditions like Reflex Sympathetic Dystrophy and arthritis.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
The market’s fairly lush for migraine therapy – a recent report projected it’d grow modestly from $2.5 billion in 2012 to $3.7 billion in 2023. Some 36 million Americans get migraines.
