Agilis Biotherapeutics Announces Orphan Product Designation Approval in Europe for the Treatment of Friedreich Ataxia

First Gene Therapy Treatment Candidate to Receive Orphan Designation in EU and USA

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Agilis Biotherapeutics, Inc. (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the central nervous system (CNS), announced today that the European Commission (EC) has granted Orphan Medicinal Product (OMP) designation in the European Union (EU) to the Company's gene therapy product candidate, AGIL-FA, being developed for the treatment of Friedreich ataxia (FA), an inherited degenerative neuromuscular disorder resulting in loss of motor coordination and strength, hearing, vision, speech and often premature death. The EC's approval follows a positive opinion in July 2017 from the European Medicine Agency's (EMA) Committee for Orphan Medicinal Products (COMP). This follows the Orphan Drug Designation for AGIL-FA granted by the U.S. Food and Drug Administration (FDA) last year. The Company's gene therapies for AADC deficiency and Angelman syndrome have previously received orphan status in both the EU and US.

"Receiving the first orphan designations for a gene therapy product candidate from the FDA and now the EU for the treatment of FA is an honor," said Mark Pykett, President and CEO of Agilis. "The orphan designation is another step on our path to bring this important new therapy to patients who currently lack treatment options."

AGIL-FA is a gene-therapy product consisting of a unique gene construct developed in partnership with Intrexon Corporation (NYSE: XON) delivered with adeno-associated virus technology. The Company has completed extensive characterization of AGIL-FA, including analyses in inducible pluripotent stem cell systems, a genetic model of FA, and multiple large animal studies demonstrating reproducible transduction of the FXN gene and expression of frataxin protein in target CNS cells integral to the neurological manifestations in FA in support of human clinical studies. The Company has completed a Pre-IND meeting with the FDA and is on track to open an IND in 2018.

"We are extremely pleased to receive this designation, as we move our FA development program forward," said Kirsten Gruis, MD, Agilis' Chief Medical Officer. "With each of our IND and clinical stage pipeline candidates having now received Orphan Designation in two major markets, our programs are well positioned to advance innovative therapeutics for patients with rare genetic diseases affecting the CNS."

Friedreich ataxia (FA) is a rare and life-shortening neurodegenerative disease caused by a defect in the FXN gene that reduces production of the frataxin protein. AGIL-FA is focused on delivering corrective DNA to specific CNS cells to restore frataxin protein levels. Agilis has worked closely with the Friedreich's Ataxia Research Alliance (FARA) to focus the development program on patient needs. "FARA is delighted to continue our support of Agilis and their innovative approach to the treatment of FA," said Jennifer Farmer, MS, Executive Director of FARA. "We look forward to continuing our partnership to advance this important potential therapy, as well as supporting research to identify biomarkers and clinical outcome measures, which will advance the development of the product candidate into clinical trials."

The EU orphan designation provides Agilis with development and commercial incentives, including 10 years of market exclusivity, prioritized consultation by EMA on the development of the drug, including clinical studies, and certain exemptions from or reductions in regulatory fees in Europe. Orphan designation is granted to drugs that are intended for the treatment of life threatening or chronically debilitating rare diseases where no therapeutic options either exist or are satisfactory. Rare diseases are those defined as having a prevalence of less than five in 10,000 people in the European Union.

About Friedreich ataxia
Friedreich ataxia (FA) is an inherited, monogenetic ataxia syndrome caused by a genetic defect in the FXN gene that leads to reduced production of frataxin, a key mitochondrial protein for ATP production. FA is the most common hereditary ataxia, with an estimated 5,000 to 10,000 patients in the U.S. (i.e., one in every 50,000 people). Onset of FA is typically in childhood with symptoms of progressive ataxia with impaired coordination of volitional muscle movements, slurred speech, and loss of ambulation after 10-15 years resulting in severe disability. Patients also develop scoliosis (which often requires surgical intervention), diabetes mellitus, hearing and vision impairments, and a serious cardiomyopathy that results in premature death in early adulthood. Current FA therapies are primarily focused on symptomatic relief, and there are no FDA-approved drugs to treat the cause of FA.

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About Agilis Biotherapeutics, Inc.
Agilis is advancing innovative gene therapies designed to provide long-term efficacy for patients with debilitating, often fatal, rare genetic diseases that affect the central nervous system. Agilis' gene therapies are engineered to impart sustainable clinical benefits by inducing persistent expression of a therapeutic gene through precise targeting and restoration of lost gene function to achieve long-term efficacy. Agilis' rare disease programs are focused on gene therapy for AADC deficiency, Friedreich's ataxia, and Angelman syndrome, all rare genetic diseases that include neurological deficits and result in physically debilitating conditions.

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Safe Harbor Statement
Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.


Agilis Biotherapeutics, Inc.
Dr. Jodi Cook, 339-440-5818
Chief Operating Officer


Source: Agilis Biotherapeutics, Inc.

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