Nicox Partner Fera Pharmaceuticals Obtains Orphan Drug
March 2n/a2022 – release at 7:30 CET
Nicox S.A. (Euronext Paris: FR0013018124, COX), an international society of ophthalmology, and Fera Pharmaceuticals, a private US specialty pharmaceutical company, today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to naproxcinod for the treatment of sickle cell disease, which affects approximately 100,000 Americans. Naproxcinod is a nitric oxide (NO)-donating naproxen combining the cyclooxygenase (COX) inhibitory activity of naproxen with that of nitric oxide developed by Nicox and licensed exclusively to Fera in the United States. Nicox has tested naproxcinod in more than 2,700 patients with osteoarthritis, generating a significant body of clinical safety data that is available to support Fera’s development of naproxcinod and ultimately a New Drug Application submission for sickle cell disease.
“We congratulate Fera on obtaining Orphan Drug Designation for naproxcinod, which is a very important step to be able to develop this molecule as a potential treatment for sickle cell disease. Fera has already carried out pre-clinical development work on naproxcinod in sickle cell disease models, and the extensive clinical package already developed by Nicox positions the molecule, the first to go into clinical development by our company – for accelerated development.” noted Michele Garufi, CEO and President of Nicox.
“Wwe are extremely happy this the FDA granted orphan drug status to naproxcinod because it now allows us to continue our development for sickle cell disease with the benefits that come with that designation. noted Frank DellaFera, founder and CEO of Fera Pharmaceuticals.
Aon orphan drug designation
The FDA’s Orphan Drug Designation program grants orphan drug status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. Benefits of orphan drug designation in the United States include seven years of market exclusivity after FDA approval, waiver or partial payment of application fees, and tax credits for trial-related expenses. qualified clinics conducted after receipt of orphan designation.
Role of nitric oxide in sickle cell disease
Sickle cell disease is an inherited condition that affects red blood cells, with a faulty version of hemoglobin causing normally oval-shaped red blood cells to take on a sickle shape, leading to symptoms such as pain, frequent infections and anemia. Rupture of these cells into the bloodstream can lead to inflammation, reduced NO and subsequent thickening of the endothelial cell wall, as well as platelet activation. In this inflamed and reduced-volume environment, sickle-shaped red blood cells, leukocytes (white blood cells) and activated platelets aggregate to create a “vascular jam” (vaso-occlusion), leading to a painful vaso-occlusive crisis.
Naproxcinod, a cyclooxygenase inhibitor (CINOD) nitric oxide (NO) donating naproxen, is a nonsteroidal anti-inflammatory product candidate designed to release NO and naproxen, originally discovered and developed by Nicox. Nicox and Fera entered into an agreement in December 2015, amended in September 2018 and December 2020, which granted Fera exclusive rights to develop and commercialize naproxcinod for the US market. Nicox is eligible to potentially receive a one-time $40 million sales-based milestone if naproxcinod reaches $1 billion in annual sales (for any indication) in the United States as well as 7% royalties on future net sales of naproxcinod in the United States. Fera is responsible for all clinical development, manufacturing, regulatory, and commercialization activities in the United States.
Nicox retains all rights to naproxcinod outside of the United States, subject to payment of royalties to Fera, if intellectual property developed under the agreement is used outside of the United States
Nicox is headquartered in Sophia Antipolis, France, is listed on Euronext Paris (Compartment C: Mid Caps; Ticker: COX) and is part of the CAC Healthcare, CAC Pharma & Bio and Next 150 indices.
For more information about Nicox, its products or its pipeline, please visit: www.nicox.com.
The risk factors likely to have a significant effect on Nicox’s business are presented in the 3rd chapter of ‘Universal registration document, annual financial report and management report 2020‘ filed with the French Financial Markets Authority (AMF) on March 1, 2021 and in the 2n/a chapter of the amendment to the “Universal Registration Document, annual financial report and management report 2020» filed with the AMF on December 9, 2021 which are available on the Nicox website (www.nicox.com).