Enalare Therapeutics Receives Orphan Drug Designation From the U.S. FDA on ENA-001 for the Treatment of Apnea of Prematurity (AoP)

ENA-001, a New Chemical Entity (NCE) with a novel mechanism of action as an agnostic respiratory stimulant, has previously been granted Rare Pediatric Disease designation for the Treatment of AoP by the FDA, with eligibility for a priority review voucher (PRV). The compound is also being developed by Enalare for the treatment of post-operative respiratory depression and community drug overdose.

Enalare Therapeutics Inc., a biopharmaceutical company dedicated to developing novel therapies for patients suffering from life threatening critical care conditions and community based health emergencies, announced today that the U.S. Food and Drug Administration (FDA) has granted its lead compound, ENA-001, Orphan Drug Designation for the treatment of Apnea of Prematurity (AoP). AoP is a common developmental disorder within pre-term births attributed to immaturity of the pulmonary system characterized by either cessation of breathing for more than 20 seconds or cessation of breathing that lasts less than 20 seconds but is accompanied by either bradycardia or hypoxemia. The condition affects approximately 25% of all preterm infants and is inversely correlated with gestational age and birth weight, including nearly 100 percent in neonates with a gestational age of less than twenty-nine weeks or birth weight less than 1,000 grams. AoP exposes these delicate neonates to repeated episodes of hypoxemia which is shown to increase risk of mortality in the NICU as well as short and long-term impaired neurological development.

Orphan Drug Designation is granted to drugs or biological products for the treatment of rare diseases or conditions that impact fewer than 200,000 people in the United States. Incentives that come with the designation include eligibility for federal grants, orphan drug tax credits, waiver of filing fees, and the potential for a seven-year marketing exclusivity period. The designation does not alter the standard regulatory requirements and process for obtaining marketing approval.

"We are very excited to have received Orphan Drug Designation for ENA-001 for the treatment of Apnea of Prematurity," stated Tom Miller, PhD, Vice President of Clinical Development at Enalare. "We believe our novel agnostic respiratory stimulant has the potential to significantly improve the standard of care for neonates experiencing this life-threatening and debilitating respiratory condition, and look forward to pursuing its development accordingly."

"Receiving both Orphan Drug Designation and Rare Pediatric Disease designation from the FDA on ENA-001 for the treatment of AoP is an important achievement for our Company," stated Alfred Schweikert, PhD, RAC, Senior Vice President of Regulatory Affairs and Quality Assurance at Enalare. "We look forward to working closely with the FDA and the neonatology community in developing this potential new therapeutic treatment for these delicate patients."

With regard to the previously assigned Rare Pediatric Disease designation on ENA-001 for the treatment of AoP, the FDA may grant a priority review voucher ("PRV") to a sponsor who receives a product approval for a rare pediatric disease. A rare pediatric disease is defined as a serious or life-threatening condition which affects less than 200,000 individuals in the U.S. per year and who are primarily less than eighteen years of age. Subject to FDA approval of ENA- 001 for the treatment of Apnea of Prematurity, Enalare would be eligible to receive a PRV that may be applied either to a subsequent product's marketing application or sold/transferred to another company.

ENA-001 is also being developed in an Intramuscular ("IM") formulation in partnership with the Biomedical Advanced Research and Development Authority ("BARDA"), part of the Administration for Strategic Preparedness and Response in the U.S. Department of Health and Human Services (contract number 75A50122C00072). The funding is provided by BARDA to support the advanced research and development of medical countermeasures (MCM) for chemical, biological, radiological and nuclear (CBRN) agents, pandemic influenza, and emerging infectious diseases that threaten the U.S. civilian population.

The development of ENA-001 is also supported by the National Institute on Drug Abuse ("NIDA") of the National Institutes of Health ("NIH") under award number R44DA057133. The content of this document is the responsibility of its authors and does not necessarily represent the official views of the National Institutes of Health.

About ENA-001

Enalare's lead compound, ENA-001, is a one-of-a-kind new chemical entity (NCE) designed as an agnostic respiratory stimulant. The compound has a novel mechanism of action that affects ventilation via the peripheral chemoreceptor pathways in the carotid body. It utilizes the body's own ventilation control system to beneficially influence breathing and has been shown to be effective and well tolerated in five human studies to date. With its novel mechanism of action and based on findings to date, it could potentially improve the lives of those impacted by several life-threatening conditions, including community drug overdose, post-operative respiratory depression, and apnea of prematurity. ENA-001 is an investigational compound and is not approved for use by the FDA.

About Apnea of Prematurity

Apnea of Prematurity (AoP) is a developmental disorder attributed to immaturity of the pulmonary system characterized by either cessation of breathing for more than twenty seconds or cessation of breathing which lasts less than twenty seconds but is accompanied by either bradycardia or hypoxemia. Apnea of Prematurity affects approximately twenty-five percent of all preterm infants and is inversely correlated with gestational age and birthweight, including nearly 100 percent in neonates with a gestational age of less than twenty-nine weeks or birth weightless than 1,000 grams. Apnea of prematurity exposes these delicate neonates to repeated episodes of hypoxemia which is shown to increase risk of mortality in the NICU as well as short and long-term impaired neurological development.

About Enalare Therapeutics Inc.

Enalare Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for patients suffering from life threatening critical care conditions and community based health emergencies, includingpost-operative respiratory depression, community drug overdose, and apnea of prematurity. Enalare plans to initiate additional clinical studies for ENA-001 across multiple indications in the near-term.

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Source: Enalare Therapeutics

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Categories: Pharmaceuticals and Biotech

Tags: Apnea of Prematurity, Enalare Therapeutics, Orphan Drug Designation


About Enalare Therapeutics

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Enalare Therapeutics Inc. is a clinical stage biopharmaceutical company dedicated to developing and commercializing novel therapies for patients suffering from acute respiratory conditions.

Morgan Wagner
Investor Relations
Enalare Therapeutics
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