On May 7, 2019 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, reported its financial results for the first quarter ended March 31, 2019 and provided an update on the company’s recent achievements and anticipated upcoming milestones (Press release, Audentes Therapeutics, MAY 7, 2019, View Source [SID1234535866]).

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"We are excited by the continued strong momentum across our business," stated Matthew R. Patterson, Chairman and Chief Executive Officer. "We recently presented new positive data from ASPIRO, the Phase 1/2 study of AT132 for the treatment of XLMTM, at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper). These data further support the compelling profile of this important product candidate and help further our progress toward the goal of making AT132 globally available to patients living with XLMTM as rapidly as possible."

Mr. Patterson continued, "In our Pompe disease program, this quarter we successfully completed an initial NHP safety study of our product candidate AT845 and the encouraging results give us added confidence as we complete GLP toxicology and dose-ranging studies to support a third quarter IND submission. Finally, we are thrilled to have recently announced the expansion of our technology platform and development pipeline with the addition of programs utilizing vectorized antisense to address Duchenne muscular dystrophy and myotonic dystrophy, two devastating neuromuscular diseases."

Recent Achievements & Upcoming Key Events

AT132 for X-Linked Myotubular Myopathy (XLMTM):

Presented new positive data from ASPIRO at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper).
The ASGCT (Free ASGCT Whitepaper) data set included safety and efficacy assessments out to 48 weeks of follow-up for six treated patients in dose Cohort 1 (1×1014 vg/kg) and 24 weeks for three treated patients in dose Cohort 2 (3×1014 vg/kg).
Patients receiving AT132 achieved reductions in ventilator dependence not previously observed in chronically ventilated patients with neuromuscular disorders, including four patients achieving ventilator independence and all other treated patients demonstrating sustained and clinically meaningful reductions in ventilator use.
The data also demonstrated sustained improvements in neuromuscular function in both dose cohorts with corresponding achievement of clinically meaningful motor milestones. Additionally, muscle biopsy data continued to show robust tissue transduction, protein expression, and histological improvements in 24 and 48-week muscle biopsies in all patients, with evidence of more rapid pathological improvement by week 24 in the Cohort 2 biopsy samples.
AT132 has been generally well-tolerated and has shown a manageable safety profile across both dose cohorts with no clinically meaningful safety differences between doses to date.
Enrollment of an additional three to five patients in Cohort 2 (3×1014 vg/kg) of ASPIRO is ongoing.
On track to select optimal dose of AT132 in the second quarter of 2019. Plan to provide an updated data package to FDA and on track to provide update on the license application submission plans for AT132 in the third quarter of 2019.
Next clinical data presentation planned at the 24th International Annual Congress of the World Muscle Society (WMS) in Copenhagen, Denmark, October 1-5, 2019.
AT845 for Pompe Disease:

Successfully completed an initial non-GLP NHP study, which showed a clean safety profile.
Previously planned GLP toxicology and dose-ranging studies in progress.
On track to file IND in the third quarter of 2019.
AT702/AT751/AT753 for Duchenne Muscular Dystrophy (DMD):

Announced exclusive license agreement with the Research Institute at Nationwide Children’s Hospital to develop AT702, an AAV-antisense candidate designed to induce exon 2 skipping for DMD caused by duplications of exon 2 and mutations in exons 1-5 of the dystrophin gene. Conducting additional preclinical work of AT702 and plan to commence a Phase 1/2 study at Nationwide Children’s in the fourth quarter of 2019.
Separate from the Nationwide Children’s collaboration, conducting preclinical work to advance AT751 and AT753, additional vectorized exon skipping candidates designed to treat DMD patients with genotypes amenable to exon 51 and exon 53 skipping. Both AT751 and AT753 utilize the same vector construct backbone as AT702, enabling a potentially accelerated path into clinical development.
Initial programs target more than 25% of patients with DMD. Plan to leverage vectorized exon skipping platform to develop further product candidates to address up to 80% of DMD patients over time.
AT466 for Myotonic Dystrophy (DM1):

Collaboration announced with Nationwide Children’s to evaluate vectorized RNA knockdown and vectorized exon skipping for DM1.
Preclinical studies are underway, and IND planned for 2020.
AT342 for Crigler-Najjar Syndrome and AT307 for CASQ2-CPVT:

Completed a strategic review of our product candidates and plan to focus on those programs that have the potential to create the greatest long-term value for patients and shareholders.
Plan to explore outlicensing opportunities to continue to advance these important and promising gene therapy programs.
Manufacturing:

Announced new internal cGMP plasmid manufacturing facility, further establishing the company’s leadership in AAV manufacturing and enabling the rapid advancement of neuromuscular programs from preclinical development to commercialization.
Made continued progress on chemistry, manufacturing, and controls (CMC) BLA and MAA-readiness efforts for AT132 based on FDA and EMA feedback on the company’s preliminary filing strategy.
Corporate:

Expanded leadership team with the additions of Mark Meltz, Senior Vice President, General Counsel, to lead the legal, compliance, and corporate governance functions and Sarah Spencer, Vice President, Corporate Communications, to lead internal and external communications to build global awareness of the company, its mission, culture, and innovative product portfolio.
First Quarter 2019 Financial Results

Cash Position: At March 31, 2019, Audentes had cash, cash equivalents and marketable securities of $375.0 million, which is expected to fund operations into 2021.
Research and Development Expenses: Research and development expenses were $39.8 million for the first quarter of 2019 compared to $19.9 million for the same period in 2018, an increase of $19.9 million. The increase in research and development expense was primarily attributable to increased R&D headcount and related facility costs, a one-time licensing fee of $7.0 million related to the AT702 program, increased internal manufacturing costs, increased clinical trial expenses for our AT132 program, increased pre-clinical and clinical trial planning costs for our AT845 program, and an increase in stock compensation expense.
General and Administrative Expenses: General and administrative expenses were $12.0 million for the first quarter of 2019 compared to $6.5 million for the same period in 2018, an increase of $5.5 million. The increase in general and administrative expenses was primarily attributable to increased headcount and related facility costs, increased professional service and consulting fees, higher costs related to public company regulatory compliance and increase in stock compensation expense.
Net Loss: Net loss was $49.4 million for the first quarter of 2019 compared to $25.6 million for the same period in 2018.
Conference Call
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its first quarter 2019 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investors + Media section of the Audentes website at www.audentestx.com. Alternatively, please call (833) 659-8620 (U.S.) or (409) 767-9247 (international) and dial the conference ID# 6839198 to access the call.

A replay of the webcast will be available on the Audentes website for approximately 30 days.