FDA Grants Orphan Drug Status to AISA-021 for Systemic Sclerosis

Aisa Pharma, Inc., a clinical-stage biopharmaceutical company based in Boston, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its drug AISA-021 (cilnidipine), a fourth-generation calcium channel antagonist. AISA-021 is being developed for the treatment of Systemic Sclerosis (SSc), a rare and severe autoimmune disease affecting about 175,000 individuals in the United States, and which has the highest mortality rate among autoimmune diseases.

Andrew Sternlicht, MD, CEO and Founder of Aisa Pharma, emphasized that this is the first instance where a calcium channel antagonist has been granted orphan drug designation for an autoimmune illness by the FDA. Dr. Sternlicht expressed hope that this designation will expedite the development of AISA-021, which aims to offer a once-daily, well-tolerated, and cost-effective treatment option to improve the quality of life for patients suffering from SSc. The company is actively looking for development partners and investors to help bring this essential treatment to patients.

The orphan drug designation under the FDA’s Orphan Drug Act provides various benefits, including tax credits, grants, waiver of specific administrative fees for clinical trials, and seven years of market exclusivity following the drug's approval. To qualify for this designation, the FDA must determine that it is medically plausible that the treatment could improve the condition it is intended to treat.

Aisa Pharma presented data from their ongoing Phase 2 study, which showed significant improvement in several endpoints compared to a placebo. These endpoints include overall disease severity, pain associated with the disease, gastrointestinal issues, skin ulcers, disability, breathing symptoms, and symptoms of Raynaud's Phenomenon. According to a preliminary review by an independent data safety committee, the study involving 60 patients revealed a lower incidence and severity of side effects and improved efficacy in preventing Raynaud's attacks compared to current therapies.

Additionally, Aisa Pharma shared several corporate updates. A Pre-IND meeting with the FDA regarding AISA-021's use in treating Systemic Sclerosis and Secondary Raynaud's Phenomenon is scheduled for September 2024. Initial drug screening by the NIH as part of the Preclinical Pain Screening Platform indicated that AISA-021 has no abuse liability or addictive potential, with additional results still pending.

Two abstracts about AISA-021 are set to be presented at the ACR Convergence Scientific Meeting in Washington, DC, on November 16, 2024. The abstracts will cover the drug's activity at sodium channel targets for pain relief and its superior efficacy in treating scleroderma pain compared to current calcium channel blockers observed in a Phase 2A study, as well as preliminary results from the RECONNOITER trial, a Phase 2 study of AISA-021 in treating Secondary Raynaud's primarily due to SSc.

AISA-021, a form of cilnidipine, is a fourth-generation calcium channel blocker with increased selectivity for the N-type calcium channel. Cilnidipine is approved in several Asian countries and widely used to treat hypertension, although it has never been approved in the US, Canada, EU, and UK. The drug causes vasodilation and improves heart, kidney, and vascular function. Studies have shown that cilnidipine is better tolerated and has a broader therapeutic window than other approved calcium channel blockers, which are sometimes used off-label for Raynaud's. Unlike other calcium channel blockers, AISA-021 may also have potential in treating pain.

Systemic Sclerosis, the most fatal autoimmune disease, affects approximately 175,000 patients in the U.S. and has a high mortality rate, with about half of patients dying within 12 years of diagnosis. Raynaud's Phenomenon, characterized by decreased blood flow causing severe pain and tingling in the hands and fingers, affects 95% of SSc patients and is considered their most debilitating symptom. No drug has yet been approved globally for the treatment of Raynaud's.

Aisa Pharma Inc., founded in 2019 and based in Boston, MA, continues its work to bring innovative treatments to those suffering from severe and rare diseases.

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