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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. Continue Reading "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." AISA Pharma receives FDA orphan drug designation for their new drug treatment for Systemic Sclerosis Post this 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 StudyPreliminary 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]; SOURCE Aisa Pharma

In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading to severe cardiac, neurological, and psychiatric symptoms as well as physical differences such as webbed fingers and toes. In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading to severe cardiac, neurological, and psychiatric symptoms as well as physical differences such as webbed fingers and toes. The treatment restored typical cellular function in 3D structures created from cells of people with Timothy syndrome, known as organoids, which can mimic the function of cells in the body. These results could serve as the foundation for new treatment approaches for the disorder. The study, supported by the National Institutes of Health (NIH), appears in the journal Nature. "Not only do these findings offer a potential road map to treat Timothy syndrome, but research into this condition also offers broader insights into other rare genetic conditions and mental disorders," said Joshua A. Gordon, M.D., Ph.D., director of the National Institute of Mental Health, part of NIH. Sergiu Pasca, M.D., and colleagues at Stanford University, Stanford, California, collected cells from three people with Timothy syndrome and three people without Timothy syndrome and examined a specific region of a gene known as CACNA1C that harbors a mutation that causes Timothy syndrome. They tested whether they could use small pieces of genetic material that bind to gene products and promote the production of a protein not carrying the mutation, known as antisense oligonucleotides (ASOs), to restore cellular deficits underlying the syndrome. In the lab, researchers applied the ASOs to human brain tissue structures grown from human cells, known as organoids, and tissue structures formed through the integration of multiple cell types, known as assembloids. They also analyzed organoids transplanted into the brains of rats. All of the methods were created using cells from people with Timothy syndrome. Applying the ASOs restored normal functioning in the cells, and the therapy's effects were dose-dependent and lasted at least 90 days. "Our study showed that we can correct cellular deficits associated with Timothy syndrome," said Dr. Pasca. "We are now actively working towards translating these findings into the clinic, bringing hope that one day we may have an effective treatment for this devastating neurodevelopmental disorder. The genetic mutation that causes Timothy syndrome affects the exon 8A region of the CACNA1C gene. The gene contains instructions for controlling calcium channels -- pores in the cell critical for cellular communication. The CACNA1C gene in humans also contains another region (exon 8) that controls calcium channels but is not impacted in Timothy syndrome type 1. The ASOs tested in this study decreased the use of the mutated exon 8A and increased reliance on the nonaffected exon 8, restoring normal calcium channel functioning.