AVROBIO Reports Third Quarter 2022 Financial Results and

Provides Business Update

Patient dosing completed in collaborator-sponsored Phase 1/2 clinical trial for cystinosis

Rare pediatric disease designations received for Gaucher disease and cystinosis investigational gene therapies

Comprehensive Gaucher disease program update planned for Wednesday, Dec. 7

“We had a steady cadence of news this quarter highlighting our steadfast efforts to bring our gene therapies to patients,” said Geoff MacKay, president and CEO of AVROBIO. “We look forward to providing a comprehensive update next month on our HSC gene therapy targeting Gaucher disease, the most common lysosomal disorder, impacting an estimated 30,000 patients worldwide. This update will contain interim clinical data from our Gaucher disease type 1 Phase 1/2 trial, including data out over two years. Notably, we plan to provide an update on our regulatory interactions for a planned Gaucher disease type 3 clinical study, as well as our overall Gaucher clinical development strategy.”

Program Updates

•	Received rare pediatric disease designation (RPDD) from the U.S. Food and Drug Administration (FDA). Read full press release here

•	Received Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). Read full press release here

•	AVR-RD-02 previously has been granted Fast Track status from FDA and orphan drug designation (ODD) in the U.S. and EU

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Patient dosing completed in collaborator-sponsored Phase 1/2 trial. Interim efficacy data from first five patients show systemic gene therapy effect across multiple tissues evaluated, including peripheral blood leukocytes, eyes, skin, gastrointestinal mucosa and neurocognitive system. Read full press release here

•	Received RPDD for AVR-RD-04 from FDA. Read full press release here

•	AVR-RD-04 previously has been granted ODD in the U.S. and EU

•	Submitted a meeting request to the MHRA to discuss a company-sponsored clinical trial for AVR-RD-04 and expect a scientific advice meeting with MHRA in Q1 2023; planning to engage with other regulatory authorities as we expand this anticipated global trial

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Clinical trial application (CTA) approved by the MHRA, Research Ethics Committee and Health Research Authority for a Phase 1/2 clinical trial in infants diagnosed with neuronopathic mucopolysaccharidosis type II (nMPS-II) or Hunter syndrome. AVROBIO’s collaborators at the University of Manchester, U.K., expect to dose the first patient in the trial in 1H 2023. Read full press release here

•	AVR-RD-05 previously has been granted RPDD and ODD in the U.S.

•	Read the full press release here

Gaucher disease program update planned for Dec. 7, 2022

Third Quarter 2022 Financial Results

AVROBIO reported a net loss of $ 23.0 million for the third quarter of 2022 as compared to a net loss of $32.6 million for the comparable period in 2021. This decrease was driven by a reduction in research and development expenses.

Research and development expenses were $15.9 million for the third quarter of 2022 as compared to $23.0 million for the comparable period in 2021. This decrease was driven by a reduction in program development expenses and personnel-related costs, including non-cash stock-based compensation.

General and administrative expenses were $7.1 million for the third quarter of 2022 as compared to $9.6 million for the comparable period in 2021. This decrease was attributable to a decrease in personnel-related costs, including non-cash stock-based compensation, and a decrease in other expenses, primarily related to facilities costs and professional fees.

Other income (expense), net was less than $0.1 million in income for the third quarter of 2022 as well as for the comparable period in 2021.

As of September 30, 2022, AVROBIO had $116.0 million in cash and cash equivalents, as compared to $189.6 million in cash and cash equivalents as of December 31, 2021. Based on AVROBIO’s current operating plan, AVROBIO expects its cash and cash equivalents as of September 30, 2022, will enable AVROBIO to fund its operating expenses and capital expenditure requirements into the first quarter of 2024.

About AVROBIO

 

Forward-Looking Statement

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as “aims,” “anticipates,” “believes,” “could,” “designed to,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our current and prospective product candidates, the expected safety profile of our investigational gene therapies, results of preclinical studies, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, the timing of patient recruitment and enrollment activities, our plans and expectations with respect to interactions with regulatory agencies and the timing and likelihood of success thereof, the expected benefits and results of our implementation of the plato^® platform in our clinical trials and gene therapy programs and its potential impact on our manufacturing and commercialization activities, and statements regarding our financial and cash position and expected cash runway, including impact on anticipated milestones. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

CONDENSED CONSOLIDATED BALANCE SHEETS

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CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

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