AyuVis Research Inc., a TechFW client, has been granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its small molecule drug AVR-48 for the prevention of bronchopulmonary dysplasia or BPD.
AVR-48 works to modulate the immune system by decreasing the inflammatory response to MV and oxygen toxicity, thereby preventing the development of BPD, which should result in lives being saved, improvement in the quality of life of survivors, and reduced hospital costs, according to the company.
“We are very excited and encouraged that the FDA approved AVR-48 for a condition that has a huge medical need globally,” said Suchismita Acharya, Ph.D., the CEO of the company she founded in 2014.
“The recognition by the FDA is a validation of our science and will facilitate our strategic partnership discussions. The designation also can mark young companies as preferable to investors because it might be expected to have reduced competition. This is great motivation to our extraordinarily talented scientific, clinical, and business team to march toward our goal to the clinic and beyond.”
Bronchopulmonary dysplasia (BPD) is a chronic lung disease that develops in prematurely-born babies and can lead to long-term respiratory and psychological disabilities or death. These babies require mechanical ventilation (MV) or other positive pressure delivery of supplemental oxygen in order to survive, but the MV and high oxygen also damages their underdeveloped lungs and release inflammatory substances, causing inflammation and tissue damage. A damaged lung no longer can supply sufficient oxygen to other organs in the body, including the brain, and most of these babies live their adulthood with asthma-like systems and poor brain development.
Orphan Drug Designation is given to drugs and biologics that are defined for the treatment, prevention, or diagnosis of a rare disease or condition, one that affects fewer than 200,000 people in the U.S. Earning the designation creates financial incentives, including a partial tax credit for clinical trial expenditures, waiver of the new drug application fee (approximately $2.2 million), and eligibility for seven years of additional marketing exclusivity beyond the timeframe afforded by a strong patent position.
AyuVis has plans for its first-in-human Phase 1 clinical trial in 2021. To reach the marketplace, AyuVis will complete GMP manufacturing of AVR-48 for clinical trials, finish the remaining IND-enabling pre-clinical studies, and conduct Phases 1, 2a, and 2b clinical trials. With positive clinical results, there is the opportunity to receive Accelerated Approval from the FDA, which could preclude a Phase 3 trial.
Acharya, who spent 11 years in various leadership roles at Fort Worth’s Alcon Labs, started AyuVis because of her passion to find a solution to the problem of antibiotic resistance to drugs and to target the un-met clinical needs in serious and life-threatening indications. At Alcon Labs, she led projects from the early discovery stage to pre-clinical and clinical stages of development in different disease areas, including glaucoma, Age-related Macular Degeneration (AMD), and viral conjunctivitis.
David Riley, M.D., MBA, is the company’s Chief Medical Officer; Keith Bryant, MBA, is CBO; Dale Christensen, Ph.D., is Director of Early Development; Russell Bromley is Director of Operations; Stella Robertson, Ph.D., is Drug Development Advisor; Ranjan Misra is Business Advisor, and Darlene Boudreaux, former Executive Director of TechFW, is CFO.