AISA Pharma Receives FDA Orphan Drug Designation for AISA-021 for Treatment of Systemic Sclerosis

Company also provides updates on its Phase 2 RECONNOITER Study in Raynaud's Phenomenon and Systemic Sclerosis and other corporate activities

BOSTON, Sept. 9, 2024 /PRNewswire/ -- Aisa Pharma, Inc. a private, clinical-stage biopharmaceutical company announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AISA-021(cilnidipine), a fourth-generation calcium channel antagonist.  Aisa is developing AISA-021 for the treatment of Systemic Sclerosis (SSc), a rare disease, which impacts approximately 175,000 Americans and has the highest mortality rate of any autoimmune disease.

"To our knowledge, this is the first time the FDA has granted orphan drug designation to a calcium channel antagonist for an autoimmune illness," said Andrew Sternlicht, MD, CEO and Founder of Aisa Pharma. "We hope this designation will accelerate our development program for AISA-021, which is designed to provide a once-daily, well-tolerated, and economical treatment that we hope can improve the lives of patients with SSc. We are actively seeking a development partner and investors to support bringing this much-needed treatment to patients."

Under the FDA's Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval. To be granted the orphan drug designation, FDA concludes that it is medically plausible that the treatment may improve the disease.

Aisa submitted information from the ongoing Phase 2 study demonstrating improvement on several endpoints versus placebo, including SSc symptoms of overall disease severity, disease-related pain, gastrointestinal dysfunction, skin ulcers, disability, and breathing symptoms, as well as Raynaud's symptoms. In a preliminary review by an independent data safety committee, the ongoing study of 60 patients demonstrated a much lower incidence and severity of side effects as well as improved efficacy for preventing Raynaud's attacks, when compared to published reviews of current therapies. 

Aisa also provided the following corporate updates:

• A Pre-IND meeting with the FDA for AISA-021 in the treatment of Systemic Sclerosis and Secondary Raynaud's Phenomenon is scheduled for September 2024.
  
  
• The initial drug screening performed by the NIH as part of the Preclinical Pain Screening Platform to evaluate the suitability of AISA-021 as a non-opioid analgesic, demonstrated no abuse liability or addictive potential for the drug. Additional results are pending.
  
  
• Two abstracts on AISA-021 will be presented at the ACR Convergence Scientific Meeting in Washington DC on 11/16/2024:

AISA 021, a Novel Calcium Channel Antagonist in Development for Raynaud's & Systemic Sclerosis, Has Activity at Sodium Channel Targets for Pain Relief and Treats Scleroderma Pain Better Than Current Calcium Channel Blockers in a Phase 2A Study

Preliminary Results from the RECONNOITER Trial, a Phase 2 Study of AISA 021 in the Treatment of Secondary Raynaud's, Primarily Due to Systemic Sclerosis

About AISA-021:  AISA-021 is a form of cilnidipine, a fourth-generation calcium channel blocker (CCB), distinguished from other dihydropyridine CCBs by its increased selectivity for the N-type calcium channel. Cilnidipine is approved in certain Asian countries and widely used to treat hypertension there, but was never approved in the US, Canada, EU and UK. Cilnidipine causes vasodilation and further improves function in the heart, kidney and vasculature. In robust studies of hypertension treatment, cilnidipine is better tolerated with a wider therapeutic window than other approved CCBs, which are used off-label for Raynaud's.  Unlike other approved CCBs, AISA-021 may also have a role in treating pain.

About Systemic Sclerosis and Raynaud's Phenomenon in Systemic Sclerosis:  Systemic Sclerosis (SSc) is the most fatal of autoimmune illnesses and affects approximately 175,000 patients in the United States. Approximately half of patients die of the disease within 12 years from initial diagnosis and 95% of SSc patients experience Raynaud's symptoms, which they describe as their most debilitating symptom. Raynaud's Phenomenon is characterized by decreased blood flow which can cause severe pain and tingling in the hands and fingers. Cardiac, renal and vascular disease, which have been shown to improve in patients treated for hypertension, contribute to morbidity and mortality in patients with SSc.  No drug has been approved anywhere in the world for the treatment of Raynaud's.

About Aisa Pharma Inc. Aisa is a private biopharmaceutical company in Boston, MA, founded in 2019. For additional information: [email protected]; http://www.aisapharma.com

SOURCE Aisa Pharma