AM-Pharma Receives FDA and EMA Orphan Drug Designation to Treat Hypophosphatasia

BUNNIK, The Netherlands--AM-Pharma B.V., a biopharmaceutical company focused on the development of recAP (recombinant human Alkaline Phosphatase), today announces that the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have both granted orphan designation status to recAP for the treatment of hypophosphatasia.

recAP is currently in Phase II development for testing the potential treatment of acute kidney injury, with the potential to be developed for hypophosphatasia.

The genetic disorder hypophosphatasia (HPP) is a mineralisation disorder that results in soft bones (rickets or osteomalacia) and in defects of teeth and periodontal tissues. A recent paper published in the journal Bone1 showed that recAP increased the mineralisation of bone and teeth, prevented seizures, and avoided potential side effects of craniosynostosis (premature ossification of the skull), and ectopic calcification (bone deposits in soft tissues) in a mouse model of the disease.

"We are delighted with the FDA's and EMA's decisions to grant recAP orphan drug status for hypophosphatasia, a devastating and potentially fatal disease," said Erik van den Berg, CEO of AM-Pharma. "Whilst pursuing the primary indication for recAP in acute kidney injury, we are exploring the product's potential in other indications. These new orphan designations help provide a strong grounding for recAP in hypophosphatasia."

About AM-Pharma

AM-Pharma is a biopharmaceutical company focused on the preclinical and clinical development of recAP (recombinant Human Alkaline Phosphatase) as a treatment of Acute Kidney Injury (AKI), Ulcerative Colitis (UC), and Hypophosphatasia (HPP). In April 2015, Pfizer acquired a minority equity position in AM-Pharma and an exclusive option to acquire the remaining equity in the company. Pfizer's option becomes exercisable on completion of a Phase II trial of recAP in sepsis-associated Acute Kidney Injury. Based on the strong results of earlier Phase II trials with bovine Alkaline Phosphatase in AKI and UC, AM-Pharma developed an innovative recombinant form of human Alkaline Phosphatase (recAP), which is currently being investigated in clinical trials. Since inception, the company has raised €67 million from a syndicate of international investors including Inventages, Forbion Capital Partners, Gilde Healthcare, Ysios Capital, Kurma biofund, IDInvest Partners, BB Biotech Ventures, Abbvie and Shire. The most recent financing round in September 2014 was €12.2 million, for the completion of a Phase II study of recAP in AKI patients, as well as development of an oral formulation of recAP for UC patients.

About Hypophosphatasia

Hypophosphatasia is a rare potentially fatal inherited metabolic bone disease. It results from ALPL gene mutations, which lead to a deficiency of Alkaline Phosphatase (AP) and bone mineralisation disorders, including soft bones (rickets or osteomalacia) and defects in teeth and periodontal tissues. Currently, there is no cure for hypophosphatasia and treatment is directed towards preventing or correcting the symptoms and complications. Some medications are under evaluation, although no proven medical therapy has yet been approved. recAP therapy in hypophosphatasia might be an effective enzyme replacement approach, avoiding antibody formation, vascular calcification and craniosynostosis (premature fusing of the skull plates), whilst improving skeletal strength.

About recAP

AM-Pharma's therapeutic candidate, recAP (recombinant Alkaline Phosphatase), is a proprietary recombinant human AP constructed from two naturally occurring human isoforms of the AP enzyme, which is highly stable and active. It is under development for testing the potential treatment of AKI, with the potential to be developed for hypophosphatasia. An oral formulation may be developed for the treatment of ulcerative colitis. The enzyme is produced by cGMP manufacture for preclinical and clinical trial supply and commercialization.

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