CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today reported financial results for the third quarter ended Sept. 30, 2020 and provided a business update.

“We continue to progress our clinical programs and have now dosed 12 patients across three different lysosomal disorders. We’re also excited by the strategic expansion of our pipeline with a new program for Hunter syndrome. This devastating lysosomal disorder ravages both the brain and body, causing young boys to progressively lose cognitive and motor function. The current standard of care does not halt this tragic disease progression. We aim to halt, prevent or reverse disease with a lentiviral gene therapy designed to deliver active protein from head to toe, including throughout the central nervous system,” said Geoff MacKay, president and CEO of AVROBIO. “We look forward to providing additional details on our new Hunter syndrome program and sharing new data on our three clinical programs, including initial clinical data from our Gaucher disease program, at our upcoming Virtual R&D Day.”

Expanded lentiviral gene therapy pipeline with program for Hunter syndrome (AVR-RD-05)

• The addition of AVR-RD-05 represents a strong strategic fit that expands AVROBIO’s leading lysosomal disorder gene therapy pipeline. The technology was in-licensed from The University of Manchester, UK (“UoM”).
• AVR-RD-05 involves ex vivo transduction of the patient’s own hematopoietic stem cells with a lentiviral vector to integrate a therapeutic gene designed to express the functional enzyme the patient needs to maintain cellular health, coupled to a proprietary peptide tag that is designed to improve stability of the enzyme in the bloodstream and facilitate uptake in the central nervous system.
• Hunter syndrome represents a significant unmet need, affecting an estimated one in 100,000 to 170,000 males worldwide with a high mortality rate and devastating complications throughout the body and brain, including severe cardiac and respiratory dysfunction, skeletal malformations and hearing impairment.
• An investigator-sponsored Phase 1/2 clinical trial of AVR-RD-05 is expected to commence in the second half of 2021.

Received orphan drug designation (ODD) from the European Commission for AVR-RD-01, an investigational gene therapy for Fabry disease, and AVR-RD-02, an investigational gene therapy for Gaucher disease type 1

• ODD is given to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than five in 10,000 patients in the European Union.
• AVR-RD-01 and AVR-RD-02 also have received orphan drug designation from the U.S. Food and Drug Administration.

Broad program update planned at upcoming Virtual R&D Day on Nov. 17, 2020

• Provide initial data on the first patient dosed in the Phase 1/2 trial for Gaucher disease, new data on the first two patients dosed in the investigator-sponsored Phase 1/2 trial for cystinosis, as well as updated patient data from the Phase 1 and Phase 2 clinical trials for Fabry disease.
• In-depth review of the company’s continued optimization of plato®, its proprietary platform designed to bring gene therapies to patients worldwide. The company will also discuss the advantages of busulfan as a conditioning agent for lentiviral gene therapy and the company’s pioneering approach to personalized conditioning with precision dosing.
• Strategic update on the company’s path to market strategy and next wave of lentiviral gene therapy programs, including the recently announced program in Hunter syndrome.

Updated statement on COVID-19 impact

Data collection for dosed clinical trial patients is ongoing, with any delays related to COVID-19 for certain measures in our Fabry disease and cystinosis trials currently being remediated. Patient recruitment and screening activities continue across our three actively enrolling clinical trials for Fabry disease, cystinosis and Gaucher disease. The company expects to provide an update on enrollment progress across all trials at R&D day on Nov. 17, 2020.

Third Quarter 2020 Financial Results

AVROBIO reported a net loss of $36.8 million for the third quarter of 2020 as compared to a net loss of $17.1 million for the comparable period in 2019. This increase was due to increased research and development expenses, as well as increased general and administrative expenses.

Research and development expenses were $28.5 million for the third quarter of 2020 as compared to $13.0 million for the comparable period in 2019. This increase was driven by increased program development activities related to the advancement of the company’s pipeline, including an $8.0 million expense related to a one-time, upfront fee we agreed to pay UoM as consideration for in-licensing the Hunter syndrome program, as well as increased personnel-related costs resulting from an increase in employee headcount, which includes the impact of non-cash stock-based compensation.

General and administrative expenses were $8.2 million for the third quarter of 2020 as compared to $5.0 million for the comparable period in 2019. This increase was primarily due to an increase in employee headcount, which includes the impact of non-cash stock-based compensation, as well as professional fees and consulting costs.

As of Sept. 30, 2020, AVROBIO had $219.5 million in cash and cash equivalents, as compared to $187.0 million in cash and cash equivalents as of Dec. 31, 2019. Based on the company’s current operating plan, AVROBIO expects its cash and cash equivalents as of Sept. 30, 2020 will enable the company to fund its operating expenses and capital expenditure requirements into the second half of 2022.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt and/or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing preclinical programs in Hunter syndrome and Pompe disease. AVROBIO is powered by the plato® gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.