Enalare Therapeutics Receives Rare Pediatric Disease Designation for Lead Compound ENA-001 for the Treatment of Apnea of Prematurity

 Enalare Therapeutics Inc., a biopharmaceutical company dedicated to developing novel therapies for patients suffering from life threatening critical care conditions, announced today that the Food and Drug Administration (FDA) has granted it's lead compound, ENA-001, rare pediatric disease designation for the treatment of Apnea of Prematurity. Enalare is planning to develop this unique agnostic respiratory stimulant for a multitude of indications related to acute respiratory depression, including for neonates experiencing Apnea of Prematurity.

Under the FDA's rare pediatric disease designation program, the FDA may grant a priority review voucher 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 18 years of age. Subject to FDA approval of ENA-001 for the treatment of Apnea of Prematurity, Enalare would be eligible to receive a priority review voucher that may be applied either to a subsequent product's marketing application or sold/transferred to another company.

"We are very excited to have received rare pediatric disease designation for ENA-001 for the treatment of Apnea of Prematurity," stated Joseph Pergolizzi MD, Chief Research and Development Officer of Enalare. "We believe our unique 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."

About ENA-001

Enalare's lead compound, ENA-001, is a one-of-a-kind new chemical entity designed as an agnostic respiratory stimulant. The compound has a unique mechanism of action that affects ventilation via the peripheral chemoreceptor pathways in the carotid body. The compound utilizes the body's own ventilation control system to beneficially influence breathing and has been shown to be effective and well-tolerated in four previous human studies.

About Apnea of Prematurity

Apnea of Prematurity is a development 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 birth weight, including nearly 100 percent in neonates with a gestational age of less than 29 weeks or birth weight less 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 acute respiratory and critical care conditions, including drug overdose, post-surgery respiratory depression, and apnea of prematurity. The Company is planning to initiate additional clinical studies for ENA-001 across multiple indications in the coming year.


Morgan Wagner
Enalare Investor Relations

Source: Enalare Therapeutics Inc.