Ammonett Pharma Receives Positive Opinion from European Orphan Medicinal Products Committee for MK-0677 for Treatment of Pediatric Growth Hormone Deficiency

MIDLOTHIAN, Va., May 23, 2017 -- Ammonett Pharma LLC (the Company or Ammonett), a privately-held biotechnology company developing a novel, daily, orally-administered mini-pill Growth Hormone (GH) secretagogue, today announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion recommending the Company’s MK-0677 (Oratrope™) for designation as an orphan medicinal product for the treatment of Growth Hormone Deficiency (GHD).

Pediatric Growth Hormone Deficiency (PGHD) in children and GHD in adults are conditions caused by insufficient amounts of GH in the body. Children with GHD have abnormally short stature with normal body proportions. GHD can be present at birth (congenital) or develop later (acquired). Current standard treatment of PGHD and GHD is through daily injections of recombinant growth hormone (rhGH).

Oratrope is Ammonett’s novel drug, ibutamoren, a small molecule, which mimics the effects of Growth Hormone release inducing hormone (Ghrelin), a natural hormone found in the stomach, which controls the release of GH from the pituitary gland. Oratrope is intended to be administered orally as a daily mini-pill, allowing the body to produce its own natural GH in a physiologically pulsatile manner over 24 hours, unlike the single daily bolus of exogenous GH delivered at present by daily injection.

“Receiving a positive opinion for Orphan Designation for Oratrope in the European Union represents a significant milestone for our global regulatory strategy for the clinical development of Oratrope,” said Michael Thorner, MB, BS, DSc, FRCP, MACP, Ammonett’s Founder and Chief Scientific Officer. “Based on previously generated clinical data, we believe Oratrope could be a potentially new effective treatment option for children with PGHD and we look forward to initiating our planned Phase 2b trial in this indication.”

About European Orphan Drug Designation

Orphan drug designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan status in the European Union (EU), the medicine must be for the treatment of a life-threatening or chronically debilitating condition that affects no more than five in 10,000 people in the EU and for which no satisfactory treatments exist or, where they do exist, the medicine will be of significant benefit to those affected by that condition.

Applications for orphan designation are evaluated by the EMA’s COMP, which provides its opinion on whether or not the medicine qualifies as an orphan medicine for the treatment, prevention or diagnosis of a rare disease. If the COMP issues a positive opinion, the European Commission will subsequently provide a formal decision which grants the medicine orphan status.

An orphan designation allows a pharmaceutical company to benefit from incentives from the EU to develop a medicine for a rare disease, such as reduced fees, regulatory support during the product development phase, access to the centralized authorization procedure (a single application for all EU countries), and 10 years of market exclusivity once the medicine is approved.

About Ammonett Pharma LLC

Ammonett Pharma LLC, is a privately held biopharmaceutical company focused on the development of novel therapies restoring normal growth and metabolism. Oratrope is Ammonett’s lead product, targeting children with PGHD who have an intact hypothalamic pituitary GH axis.

Contacts

Kevin P Tully CPA, CGA
Chief Executive Officer
[email protected]

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Andrew Amiel
VP Business Development
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