Exciting news for Frosties: A Phase 2 clinical study is underway for a new Raynaud’s drug that, if successful, will be the first FDA-approved drug specifically for Raynaud’s.
Aisa Pharma, Inc. (https://aisapharma.com), a Boston based biopharmaceutical company, has launched trials for Profervia®, an oral drug that will combine cilnidipine, a calcium channel blocker (CCB) approved in a few countries outside the U.S. and tadalafil (marketed as Cialis®), a phosphodiesterase (PDE) inhibitor used throughout the world for erectile dysfunction and pulmonary hypertension. Both cilnidipine and tadalafil increase blood flow to peripheral organs and tissues.
CCBs and PDE inhibitors are used to treat patients with Raynaud’s alone and the 95% of systemic sclerosis (scleroderma) patients who have Raynaud’s. However, cilnidipine, which has a unique efficacy and tolerability profile, has never been tested alone or in combination with a PDE inhibitor in these diseases.
Aisa Pharma’s founder and CEO Andrew Sternlicht, M.D., an Assistant Professor of Anesthesiology at Tufts Medical Center, says the study is focused on patients who have secondary Raynaud’s disease associated with scleroderma, but the drug is expected to benefit Raynaud’s sufferers with both the primary and secondary forms of the disease.
Dr. Sternlicht, who has Raynaud’s himself, said the new treatment may offer hope to the millions of patients with Raynaud’s who often have difficulty carrying out their normal activities, particularly during the colder months. “Aisa is taking a novel approach, leveraging our proprietary drug combination, which maximizes the potency of two well-established classes of medicine with demonstrated safety and efficacy in these patients,” he said.
The double-blind study currently underway includes 76 patients and is being conducted in Australia. A larger, multi-center study in the United States is set to follow in the second quarter of 2022.
Calcium channel blockers and PDE inhibitors are currently used to treat patients with Raynaud’s and the connective tissue disease scleroderma, but cilnidipine has never been tested alone or in combination with a PDE inhibitor for these disorders, Dr. Sternlicht told us. Although tadalafil and other PDE inhibitors are used primarily for the treatment of erectile dysfunction and pulmonary hypertension, many Frosties report they find relief from painful Raynaud’s episodes when taking the drugs. Current treatment protocol recommends the combination of these two classes of drugs as being the most effective regimen for patients with unresponsive or severe disease.
For scleroderma patients with Raynaud’s the benefits of Profervia may also extend beyond improvement of Raynaud’s symptoms into underlying disease pathological contributors. Dr. Sternlicht said, “Our panel of expert physicians who treat and conduct research for scleroderma patients are excited about our program and believe the drug combination may modify the course of the disease.”
We’re also excited to learn that medical researchers are starting to recognize the huge market potential for new therapies geared to Raynaud’s patients. Too often we hear Raynaud’s referred to as “rare,” but an estimated 5-10% of Americans have it. That makes it more prevalent than multiple sclerosis, Parkinson’s disease and leukemia combined!
A primary reason we don’t have the media exposure of well recognized diseases is because producers of “off label” drugs used to treat the condition are prohibited from funding Raynaud’s education programs. We must depend on member donations, small grants, and sponsorships from manufacturers of warming products who support our cause.
We will keep our Raynaud’s community apprised as clinical studies get underway for this new Raynaud’s drug at centers across the U.S.
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