GAINESVILLE, Fla., Aug. 27, 2015 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, today announced it had met a patient enrollment milestone in the X-Linked Retinoschisis (XLRS) clinical study, which will earn AGTC a $5 million payment from Biogen.
AGTC filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) in March to conduct a Phase I/II clinical trial of the company’s gene therapy product candidate for the treatment of XLRS. Utilizing technology licensed from the University of Florida, AGTC’s XLRS product candidate uses an AAV capsid with surface residues that have been specifically engineered for better penetration to the back of the eye. The primary clinical endpoint is safety and the secondary endpoints include assessments of visual acuity, visual field, ERG and optical coherence tomography. The study is expected to enroll a total of 27 patients at four sites and preliminary data from the first cohorts of patients are expected by the end of 2015.
This marks the first milestone since AGTC and Biogen announced in July a broad collaboration agreement focused on the development of gene-based therapies for two orphan diseases of the retina and three options for early stage discovery programs in two ophthalmic and one non-ophthalmic condition. Biogen has completed the $124 million equity purchase and up-front payment, following the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and the satisfaction of other customary closing conditions.
“We are pleased to announce this important milestone under our recent partnership with Biogen,” said Sue Washer, Chief Executive Officer of AGTC. “This continues to be an encouraging time of growth for the Company and we look forward to advancing this collaboration in an effort to accelerate the development and commercialization of novel gene-based therapies for treating complex inherited diseases with no currently available treatments.”
AGTC has a robust product pipeline, including five named ophthalmology development programs across four targets (XLRS, XLRP, achromatopsia and wet age-related macular degeneration), one non-ophthalmology program (alpha-1 antitrypsin deficiency) and proof-of-concept data in multiple additional indications. The Company employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. The Company has a significant intellectual property portfolio and expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation and physical delivery of gene therapy products.
AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases in ophthalmology. AGTC’s lead product candidates focus on X-linked retinoschisis, achromatopsia and X-linked retinitis pigmentosa, which are inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC is also using its gene therapy expertise to expand into disease indications with large market opportunity such as wet AMD.
Forward Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: uncertainty inherent in the regulatory review process; uncertainty regarding the ability to achieve the expected benefits from the proposed collaboration, including as a result of risks and uncertainties associated with drug development and commercialization, reliance on third parties over which AGTC may not always have full control and other risks associated with collaborations; and other risks and uncertainties described under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2014, as filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.
CONTACT: David Carey (IR) or Danielle Lewis (PR) Lazar Partners Ltd. T: (212) 867-1768 or (212) 843-0211 email@example.com or firstname.lastname@example.org Corporate Contact: Larry Bullock Chief Financial Officer Applied Genetic Technologies Corporation T: (386) 462-2204 email@example.com