SAN FRANCISCO, Jan. 7, 2019 /PRNewswire/ -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today provided an update on recent progress and the company's strategic priorities for 2019.

"2018 was an outstanding year for Audentes," stated Matthew R. Patterson, Chairman and Chief Executive Officer. "We are particularly encouraged by the progress in the ASPIRO Phase 1/2 study and the promising clinical profile of AT132 for the treatment of X-linked Myotubular Myopathy. We received RMAT and PRIME designations in the U.S. and EU respectively, and recently held productive initial interactions with both the FDA and EMA to discuss potential registration pathways for AT132. We look forward to sharing further details from those interactions in early 2019."

Mr. Patterson continued, "We are preparing for multiple milestones and inflection points across our portfolio of product candidates in the coming year, including the continuation of BLA and commercial readiness activities in our XLMTM program. To support this effort, we recently expanded our leadership team with the addition of Eric Mosbrooker, a seasoned executive with a demonstrated track-record of building global commercial organizations and successfully launching rare disease products, to lead the development and execution of our global commercial strategy. In our Pompe disease program, we are encouraged by the progress of our ongoing preclinical studies and are planning to file an IND for AT845 in the third quarter of 2019. We are also advancing our new product candidate, AT720, a novel AAV-based therapeutic being developed to treat a large neuromuscular disease with significant unmet medical need, and plan to disclose the therapeutic target and provide a program overview in the second quarter of 2019. Finally, our ability to meet these important milestones is supported by a strong balance sheet. With over two years of cash runway, we are well-positioned to make meaningful progress toward our goal of providing transformative therapies to patients living with devastating rare diseases."

Mr. Patterson will provide a corporate update and 2019 outlook in a presentation at the 37th Annual J.P. Morgan Healthcare Conference on Tuesday, January 8, 2019, at 3:30 pm PST. To access the live webcast of the presentation, please visit the Events & Presentations page within the Investors & News section of the Audentes website. Replays of the live webcast will be available on the Audentes website for approximately 30 days following the conference.

Key 2018 Accomplishments and 2019 Milestones

AT132 for XLMTM

• Reported promising safety, efficacy, and muscle biopsy data from ASPIRO. The latest ASPIRO data set (presented October 2018) demonstrated all six treated patients in Cohort 1 (1x1014 vector genomes per kilogram (vg/kg) and the sentinel patient in Cohort 2 (3x1014 vg/kg) showed meaningful improvements in neuromuscular and respiratory function with results ranging from 4 to 48 weeks. Three patients achieved ventilator independence, and all other patients showed significant reductions in ventilator use. Additionally, week 24 muscle biopsy data for the first four treated patients showed robust tissue transduction, protein expression, and histological improvement.
• Completed dosing of full Cohort 2 (three treated patients and one delayed-treatment control), and plan to dose an additional three to five patients as part of a Cohort 2 expansion arm in early 2019. Next clinical data update planned at the 2019 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in May.
• Awarded Regenerative Medicine Advanced Therapy (RMAT) and Priority Medicines (PRIME) designations by the FDA and the EMA, respectively. Completed productive initial interactions with both agencies to discuss potential registration pathways for AT132. Plan to provide additional meeting details in Q1 2019.

AT845 for Pompe Disease

• Conducted encouraging preclinical studies of AT845 for the treatment of Pompe Disease, and plan to file IND in Q3 2019.

AT342 for Crigler-Najjar Syndrome

• Established initial proof of concept based on 12-week data from first patient enrolled in the VALENS Phase 1/2 study at a dose of 1.5x1012 vg/kg. Next program update planned in Q1 2019.
• Awarded Rare Pediatric Disease and Fast Track designations for AT342 by the FDA.

Pipeline Expansion

• Advancing our new product candidate, AT720, a novel AAV-based therapeutic being developed to treat a large neuromuscular disease with significant unmet medical need.
• Plan to disclose the therapeutic target and provide a program overview in Q2 2019.

Manufacturing

• Commenced BLA readiness and process validation activities for AT132. Chemistry, manufacturing, and controls (CMC) approach is consistent with recent FDA gene therapy guidance. Since program inception, clinical material has been manufactured utilizing substantially the same process, scale, and facility intended to supply the commercial market.

Financials

• Strengthened balance sheet with two follow-on financings, resulting in aggregate net proceeds of approximately $380 million after the deduction of underwriting discounts, commissions, and estimated offering expenses.
• On a proforma basis at September 30, 2018, Audentes had cash, cash equivalents and short-term investments totaling nearly $450 million, which includes net proceeds of $162.5 million from its follow-on financing that closed in October 2018. These resources are expected to fund the company into 2021.

About Audentes Therapeutics, Inc.Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases. We are currently conducting Phase 1/2 clinical studies of our lead product candidates, AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM), and AT342 for the treatment of Crigler-Najjar syndrome. We have two additional product candidates in development, including AT845 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT). We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.

For more information regarding Audentes, please visit www.audentestx.com.

Forward Looking StatementsThis press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: anticipated clinical milestones, potential pipeline expansion, the timing and nature of clinical development activities, the timing of the release of data from ongoing or upcoming clinical trials, the nature and timing of the release of information related to regulatory communications, the timing of regulatory filings, the expected safety profile of the company's product candidates, the expected benefits of the company's product candidates, the expected capacity of the company's internal manufacturing facility, and the use and adequacy of cash reserves. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates, obtain regulatory approval of and ultimately commercialize its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, establish and scale-up manufacturing processes that comply with regulatory requirements, protect intellectual property and other risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

Audentes Contacts:

Investor Contact:Andrew Chang415.818.1033[email protected]

Media Contact:Katie Hogan415.951.3398[email protected]

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