AM-Pharma receives FDA fast track designation for recAP in Acute Kidney Injury, and reports completion of first stage in Phase II trial

BUNNIK, Netherlands–(BUSINESS WIRE)–AM-Pharma B.V., a biopharmaceutical company focused on the development
of recAP (recombinant human Alkaline Phosphatase) for inflammatory
diseases, announces that it has received fast track designation from the
U.S. Food and Drug Administration (FDA) for recAP to treat Acute Kidney
Injury (AKI). Additionally, the Company reports that the interim
analysis on the first stage of the adaptive Phase II trial in AKI has
been successfully completed.
The fast track designation helps facilitate the development and expedite
the review process of drugs designed to treat severe conditions and
fulfill an unmet medical need.¹ This status also enables a
continuous dialogue with the FDA on the clinical and regulatory
development of recAP.
Within the Phase II study, an independent data monitoring committee has
completed its interim analysis of the first 120 patients recruited to
the trial. The outcome is the selection of the best of the three doses
that were investigated in the first stage of the study. In the second
stage of the clinical trial, an additional 170 patients will be
recruited in two arms of 85 patients each, where patients will receive
either the selected recAP dose or placebo.
The study is the largest to date investigating treatment of AKI, and
will recruit a minimum of 290 patients in more than 70 sites across
Western Europe and North America. Results from the study are expected in
2017.
“Both the fast track designation and the completion of the interim
analysis indicate the positive progress of recAP and potentially shorten
the time to bring the product to patients,” said Erik van den Berg, CEO
of AM-Pharma. “We look forward to continuing our interactions with the
regulatory agencies for fast development of recAP to address this unmet
medical need in Acute Kidney Injury.”
¹ U.S. Food and Drug Administration; available at https://www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm
Notes to Editors
About AM-Pharma www.am-pharma.com
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). Based on strong results from 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 in Phase II development for sepsis-associated AKI. In May
2015, AM-Pharma signed a deal with Pfizer, which made an upfront payment
of $87.5 million for a minority equity interest, and exclusive option to
acquire the Company, with additional potential payments of up to $512.5
million upon option exercise and potential launch of any product that
may result from the agreement.
About Acute Kidney Injury
Acute Kidney Injury (AKI) involves inflammatory processes in the kidney
which can lead to complete loss of renal function. Hospital-acquired AKI
affects annually around 3 million patients in Europe, the US and Japan,
and is associated with mortality in roughly 700,000 patients. It occurs
in as many as 4% of hospital admissions and 40% of critical care
admissions. Depending on the severity and cause of renal injury,
mortality ranges from 10% to as high as 70%. In the US alone, hospitals
spend around $10 billion each year on managing this major medical
problem. The most important causes of AKI are sepsis, cardiovascular
surgery, exposure to nephrotoxic drugs and trauma. AKI patients that
need dialysis have the worst prognosis. Currently the only treatment
option is dialysis and supportive care. No drugs are approved to treat
this condition. Typically these patients are treated in Intensive Care,
often with support of nephrologists.^1,2,3
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 in Phase II development for the potential
treatment of AKI, with the potential to be developed for HPP. An oral
formulation has been developed for the treatment of UC. The enzyme is
produced by cGMP manufacture for preclinical and clinical trial supply
and commercialization.
¹ Murugan R. and Kellum J.A., (2011) Nat Rev Nephrol. Vol 7:
209-217
² Heung M. and Chawla L., (2014) Nephron Clin Pract. Vol 127:
30-34
³ Chertow et al., (2005) J Am Soc Nephrol. Vol 16: 3365-3370
Soc Nephrol. Vol 16: 3365-3370

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