FDA Grants Orphan Drug Designation to Allena Pharmaceuticals’ Investigational Therapy for the Treatment of Primary Hyperoxa...
July 13 2017 - 7:05AM
Business Wire
Allena Pharmaceuticals, Inc., a specialty biopharmaceutical
company dedicated to bringing first in class, specific,
non-absorbed, oral protein therapeutics to patients with serious
renal, urologic and orphan diseases, announced today that the US
Food and Drug Administration (FDA) has granted orphan drug
designation to Allena’s investigational product ALLN-177, an oral
formulation of oxalate decarboxylase, for the treatment of primary
hyperoxaluria (PH).
Allena’s lead compound ALLN-177, is being developed to treat
patients with severe hyperoxaluria, or patients with markedly
elevated urinary oxalate excretion. PH, a type of severe
hyperoxaluria, is a rare genetic disorder caused by endogenous
overproduction of oxalate by the liver that can result in kidney
stone disease, kidney damage, and kidney failure, which may lead to
death. PH has three main types, PH1, PH2 and PH3, and is estimated
to affect approximately 1 in 58,000 based on population analysis.1
The most severe and most common type among genetically
characterized patients is PH1. These patients typically develop
recurrent kidney stones with progressive nephrocalcinosis and end
stage renal disease by 20-30 years of age.1 There are no FDA
approved therapies for PH, and the most severe patients may be
treated with liver and/or kidney transplant.
ALLN-177 is a first-in-class therapeutic being developed to
treat patients with severe hyperoxaluria using an oral,
non-absorbed enzyme that works in the gastrointestinal (GI) tract,
where it can degrade both dietary and endogenously produced
oxalate. GI elimination of oxalate may help alleviate the chronic
systemic oxalate burden on PH patients.
“Primary Hyperoxaluria is a devastating disease for patients and
their families. We desperately need better therapeutic options to
treat this disease,” said Craig B. Langman, M.D., the Issac A. Abt
M.D. Professor of Kidney Diseases at Feinberg School of Medicine,
Northwestern University and Head, Kidney Diseases at Lurie
Children’s Hospital Chicago.
The preclinical data supporting the FDA orphan designation
demonstrated oxalate decarboxylase significantly reduced urinary
and plasma oxalate in multiple animal models including a rodent
disease model of PH and a porcine model with urine oxalate in the
range seen in PH.
The Orphan Drug Designation Program is administered by the FDA's
Office of Orphan Products Development, which grants orphan status
to drugs intended to treat rare diseases that affect fewer than
200,000 people in the U.S. The program provides incentives for
sponsors and has enabled the development and marketing of more than
400 products for rare diseases since 1983.
“This is an important regulatory designation to advance the
development of ALLN-177 for patients who could benefit from novel
therapeutic options that directly address excess oxalate,” said
Louis Brenner, M.D., President and Chief Operating Officer of
Allena Pharmaceuticals. “We are committed to the development of
ALLN-177 for the treatment of patients with severe hyperoxaluria
disorders.”
About Hyperoxaluria and ALLN-177
Hyperoxaluria is a metabolic disorder resulting from high
oxalate levels in the urine due to either hyper-absorption of
oxalate from the diet (secondary) or from overproduction of oxalate
by the liver due to a genetic defect (primary). Kidney stones are
typically the first sign of hyperoxaluria, are often painful, and
may require interventional procedures. Severe hyperoxaluria in
settings of enteric and primary hyperoxaluria may also lead to
kidney damage (nephrocalcinosis), chronic kidney disease and
end-stage renal disease, which may lead to death.
ALLN-177 is an orally-administered, recombinant
oxalate-degrading enzyme in development for the treatment of
severe hyperoxaluria. ALLN-177 targets oxalate in the GI tract in
an effort to reduce the burden of both dietary and
endogenously produced oxalate. ALLN-177 has the potential to
decrease the oxalate available systemically for deposition as
calcium oxalate crystals or stones in the kidneys, as well as
reduce long-term kidney complications.
About Allena Pharmaceuticals
Allena Pharmaceuticals, Inc. is a specialty biopharmaceutical
company dedicated to bringing first in class, specific,
non-absorbed, oral protein therapeutics to patients with serious
renal, urologic and orphan diseases. Allena is completing a Phase 2
program in secondary hyperoxaluria. The company’s technological
approach enables the design and development of oral protein
therapies that remain in the gastrointestinal (GI) tract, where the
protein exerts its therapeutic effect by degrading certain
nephrotoxic metabolites, without being absorbed into the
bloodstream. Led by a proven management team with deep expertise in
protein therapeutic design, development, and commercialization,
Allena is committed to bringing breakthrough new treatments to
patients with unmet medical needs. Based in Newton, MA, the company
is supported by a top-tier investor syndicate including Frazier
Healthcare, Third Rock Ventures, Bessemer Venture Partners, HBM
Partners, Pharmstandard International S.A., Partner Fund
Management, Fidelity Management & Research Company, and other
investors. For more information, please visit www.allenapharma.com
(http://www.allenapharma.com).
References:
1. Hopp, Katharina et al. “Phenotype-Genotype
Correlations and Estimated Carrier Frequencies of Primary
Hyperoxaluria.” J Am Soc Nephrol. 26.10 (2015): 2559-2570.
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Allena Pharmaceuticals, Inc.Janet Giroux, 617-467-4577Manager of
Corporate Operationsjgiroux@allenapharma.com