CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (NASDAQ: AVRO), a Phase 2 clinical-stage gene therapy company, today reported financial results for the first quarter ended March 31, 2019 and provided a business update.

“2019 is off to a strong start as we advance our pipeline of gene therapies for lysosomal storage diseases on multiple fronts. We expect to exit this year with three gene therapy programs in clinical trials – for Fabry, Gaucher and cystinosis,” commented Geoff MacKay, President and Chief Executive Officer of AVROBIO. “Underpinning this progress is plato™, AVROBIO’s proprietary platform for developing, manufacturing, and commercializing our gene therapy programs globally, which we plan to utilize initially in our Company-sponsored clinical trials in Fabry and Gaucher. Our highest priority in early 2019 was to secure regulatory clearances that enable the use of plato in multiple trials in multiple countries, namely in Australia, Canada and the U.S. We believe that the plato platform, which is designed to enable the treatment of thousands of patients, if our gene therapies are approved, represents a significant advance toward commercial readiness.”

Business Update and Program Milestones

• AVROBIO’s plato platform: plato is the Company’s commercial-scale platform for anticipated future worldwide commercialization and pipeline expansion activities. Three important upgrades are expected to be incorporated in 2019 – a proprietary state-of-the-art four-plasmid vector system, automation of a closed cell manufacturing process and a conditioning regimen that utilizes therapeutic drug monitoring (TDM). The plato platform is designed to enhance the potential potency, safety, efficacy, and long-term durability of AVROBIO’s gene therapies, and may additionally provide the capability to address central nervous system manifestations that accompany many lysosomal storage diseases.
• Fabry disease: AVR-RD-01 is AVROBIO’s investigational gene therapy candidate for the treatment of Fabry disease. During the quarter, the Company:
  • Completed enrollment in the investigator-sponsored FACTs1 Phase 1 study, and continued recruitment in AVROBIO’s FAB-2012 Phase 2 trial. A total of seven patients have now been dosed across these two studies.
  • Achieved U.S. Food and Drug Administration (FDA) clearance of AVROBIO’s Investigational New Drug (IND) application for AVR-RD-01. This allows AVROBIO to move forward on two key program initiatives anticipated for the second half of 2019: the incorporation of our plato platform into the FAB-201 Phase 2 trial and the opening of our first clinical site in the U.S.
  • Presented clinical data in February 2019 that continued to support the potential of AVR-RD-01 as a gene therapy for Fabry disease. All patients reporting data exhibited elevated AGA enzyme activity, with the first patient in the Phase 1 study exhibiting elevated AGA enzyme activity at 22 months after being treated with AVR-RD-01. An impact of gene therapy on substrate and metabolite levels was observed both in patients who have discontinued enzyme replacement therapy (ERT) as well as in treatment-naïve patients. Lyso-Gb3 levels of the first patient in the Phase 1 study were lower at 22 months while on AVR-RD-01 alone than while on ERT alone. An 85 percent reduction in lyso-Gb3 activity was observed for the first patient in the Phase 2 clinical trial, who is ERT-naïve, at the six-month timepoint.
• Gaucher disease: AVR-RD-02 is AVROBIO’s investigational gene therapy candidate for the treatment of Gaucher disease. The Company received clearance for its clinical trial application (CTA) utilizing the plato platform from Health Canada and plans to initiate its GAU-201 Phase 1/2 clinical trial in the second half of 2019. We expect the trial to enroll 8 to 16 patients with Type 1 Gaucher disease and to include both treatment-naïve patients as well as patients stable on ERT.
• Cystinosis: AVR-RD-04 is AVROBIO’s investigational gene therapy candidate for the treatment of patients with cystinosis. The FDA cleared the IND application for the investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04. The trial, which is designed to enroll up to 6 patients who are currently being treated with cysteamine, will be conducted at the University of California, San Diego (UCSD) and is expected to start in the second half of 2019.
• AVROBIO highlighted its manufacturing expertise and preclinical data at the 2019 ASGCT Annual Meeting. AVROBIO’s senior management and academic collaborators made presentations at the American Society of Cell and Gene Therapy (ASGCT), including:
  • Kim Warren, Ph.D., Head of Operations for AVROBIO, who presented on the potential of AVROBIO’s plato platform to address gene therapy manufacturing and commercialization challenges.
  • Azadeh Golipour, Ph.D., AVROBIO’s Senior Director of Manufacturing Operations, who presented on advances in the development of plato’s automated gene modified cell production process, from concept to process verification.
  • Stefan Karlsson, MD, Ph.D., Professor in the Division of Molecular Medicine and Gene Therapy at Lund University in Sweden, who presented preclinical data from the Company’s programs in Gaucher disease.
  • Stephanie Cherqui, Ph.D. and her UCSD team members, who gave several oral presentations on preclinical cystinosis data.

First Quarter 2019 Financial Results

AVROBIO reported a net loss of $17.1 million for the first quarter of 2019 as compared to a net loss of $8.2 million for the comparable period in 2018. This increase was due to increased research and development expenses, as well as increased general and administrative expenses.

Research and development expenses were $12.4 million for the first quarter of 2019 as compared to $5.6 million for the comparable period in 2018. This increase was driven by increased preclinical, clinical development and manufacturing activities related to the advancement of the Company’s pipeline, as well as increased personnel-related costs including non-cash stock-based compensation expense resulting from an increase in employee headcount.

General and administrative expenses were $5.3 million for the first quarter of 2019 as compared to $2.1 million for the comparable period in 2018. This increase was primarily due to an increase in employee headcount, legal, consulting and professional fees related to the support of ongoing business operations as a publicly traded company, as well as the impact of non-cash stock-based compensation.

As of March 31, 2019, AVROBIO had $108.5 million in cash and cash equivalents, as compared to $126.3 million in cash and cash equivalents as of December 31, 2018. Based on the Company’s current operating plan, AVROBIO expects its cash and cash equivalents as of March 31, 2019 will enable the Company to fund its operating expenses and capital expenditure requirements into the second half of 2020.

About AVROBIO, Inc.

AVROBIO, Inc., is a Phase 2 clinical-stage gene therapy company. AVROBIO is focused on the development of its gene therapy candidate, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. The Company’s plato™ platform includes a proprietary vector system, automated cell manufacturing solution and refined conditioning regimen with therapeutic drug monitoring. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com.