APEPTICO: New orphan drug status for therapeutic peptides

The Committee for Orphan Medicinal Products of the European Medicines Agency grants orphan drug designation to APEPTICO’s development compounds AP301 and AP318 for treatment of for treatment of Pseudohypoaldosteronism Type 1B.

APEPTICO Forschung und Entwicklung GmbH, a biotechnology company developing novel peptide-based drugs, today announced that its development compounds AP301 (INN: Solnatide) and AP318 have been granted orphan drug designation by the Committee for Orphan Medicinal Products of the European Medicines Agency for treatment of Pseudohypoaldosteronism Type 1B.

Pseudohypoaldosteronism type 1B (PHA 1B) is an autosomal recessive disorder caused by loss-of-function mutations in the epithelial sodium ion channel (ENaC). This life-threatening condition usually presents in the first weeks of life with severe dehydration, salt wasting and failure to thrive, symptoms which persist into adulthood. Patients often suffer from respiratory infections and may die from potassium overload and cardiac arrest. Currently, no satisfactory method of treatment exists.

This was the first time that a development compound has been granted orphan medicinal product designation for this life-threatening, chronic condition by the European Medicines Agency. APEPTICO’s request was based on results from cell-based studies making use of heterologous expression of mutant versions of the human ENaC. The majority of non-clinical data have been generated during scientific collaborations of APEPTICO with Professor Rosa Lemmens-Gruber’s team from the Department of Pharmacology & Toxicology of the University Vienna (Vienna, Austria). Genetic manipulation, recombinant expression and electrophysiological techniques were used to demonstrate that AP301 and AP318 restored the sodium transport through mutant, loss-of-function ENaC to normal levels and higher.

Dr. Bernhard Fischer, CEO of APEPTICO commented: “I am very pleased that the European Medicines Agency has approved our application for orphan drug designations for solnatide (laboratory code AP301) and AP318 for treatment of pseudohypoaldosteronism type 1B. Until today there exists no approved therapy for this chronic and life-threatening condition.” Dr. Fischer added, “We are very proud to expand the clinical uses of our therapeutic peptides into chronic conditions while we further substantiate the clinical application of AP301 and related synthetic peptides in phase III clinical trials for treatment of the pulmonary permeability oedema in patients with acute respiratory distress syndrome and for treatment of primary graft dysfunction in patients following lung transplantation, both acute life-threatening conditions.”

About APEPTICO Forschung und Entwicklung GmbH

APEPTICO is a privately-held biotechnology company based in Austria, developing peptide-based products targeting chronic and life-threatening diseases. The peptide molecules correspond to validated, pharmacodynamic active structures and domains of well-known proteins and biopharmaceuticals. By concentrating on synthetically produced protein structures APEPTICO avoids general risks associated with gene- and cell-technologies. APEPTICO makes use of its technology platforms PEPBASE(TM) and PEPSCREEN(TM) to significantly reduce cost and to shorten time to market.

About the APEPTICO’s therapeutic protein structures

APEPTICO’s proprietary therapeutic molecules, such as AP301 (INN: solnatide) and AP318 are synthetically manufactured structural equivalents to domains of the human proteins. Liquid and dry powder formulations of such protein structures can be administered into the lung by inhalation of aerosol particles with diameter 5 micrometres or less. Most recently, APEPTICO has successfully completed two Phase II clinical trials with orally inhaled peptides for treatment of patients with pulmonary permeability oedema and ARDS (acute respiratory distress syndrome) and for treatment of patients with primary graft dysfunction following lung transplantation. For both acute and life-threatening conditions no specific drug-based treatments exist so far.

About pseudohypoaldosteronism type 1B (PHA 1B)

Pseudohypoaldosteronism type 1B (PHA 1B) or autosomal recessive pseudohypoaldosteronism type I, is characterized by salt wasting from the kidney, colon, and sweat and salivary glands leading to high concentrations of sodium in sweat, stool, and saliva. The disorder involves multiple organ systems and is especially threatening in the neonatal period. Laboratory evaluation shows hyponatremia, hyperkalemia, and increased plasma renin activity with high serum aldosterone concentrations. Sweat and salivary glands, the distal renal tubule, and colonic mucosa are unresponsive to mineralocorticoids. In addition to severe dehydration, vomiting and failure to thrive occurring in the first weeks of life, the clinical picture may be complicated by cardiac dysrhythmias, collapse, shock or cardiac arrest. An increase in the volume of airway surface liquid leads to frequent respiratory tract manifestations and respiratory tract infections are common in affected children. PHA 1B is severe: no remission has been reported and patients suffer from recurrent life-threatening episodes of salt loss requiring salt supplements and control of hyperkalemia to ensure survival. PHA 1B is transmitted in an autosomal recessive manner and is caused by mutations in the genes coding for the subunits of the amiloride-sensitive sodium channel, ENaC resulting in the expression of mutant, loss-of-function ENaC.

About Orphan Drugs

An orphan drug is a pharmaceutical agent that specifically treats a rare medical condition, the condition itself being referred to as an orphan disease. Orphan drug legislation aim to encourage pharmaceutical companies to develop drugs for rare diseases. Under the law, companies that develop such a drug for an orphan disorder gain marketing exclusivity for 10 years (EU) and 7 years (USA) after marketing approval.

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