AVROBIO Reports Fourth Quarter and Fiscal Year 2021 Financial Results and Provides Business Update

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 fourth quarter and year ended Dec. 31, 2021, and provided a business update.

“After a busy 2021 with new patients dosed across three clinical trials, we acted in early January to strategically position AVROBIO for long-term success by reprioritizing our leading lysosomal disorder pipeline. Four weeks after, we reinforced that position by releasing strong interim data from the Phase 1/2 collaborator-sponsored clinical trial for cystinosis at the WORLDSymposium^TM 2022,” said Geoff MacKay, president and CEO of AVROBIO. “We look forward to sharing new data on the three patients dosed to date in our Gaucher disease type 1 program in 2022 and providing updates for our other pipeline programs following meetings with regulatory agencies planned for later this year. We potentially have many exciting catalysts ahead of us and believe that our strong balance sheet will support continued progress through anticipated milestones and into the first quarter of 2024.”

Program Updates

Presented interim data from Phase 1/2 clinical trial of AVR-RD-04 in cystinosis at the 18^th Annual WORLDSymposium™ 2022:

• First three patients dosed to date remain off oral cysteamine with follow up durations ranging between 12- and 26-months post-gene therapy infusion. Sustained engraftment has been observed in each of these patients, as evidenced by stable vector copy number (VCN) levels.
• Reduction in number of cystine crystals as measured in skin and intestinal mucosa biopsies observed across these three patients.
• A fourth patient was infused in November 2021.
• No adverse events (AEs) related to the drug product have been reported in the four patients infused to date. All AEs observed have been attributed to myeloablative conditioning, stem cell mobilization, underlying disease or pre-existing conditions.
• Clinical proof-of-concept in adult patients lays the groundwork for potential AVROBIO-sponsored trial planned to begin in 2023.
• The company hosted a conference call providing a full data update (see here).

Dosed a third patient in our Phase 1/2 GUARD1 clinical trial in Gaucher disease type 1

Deprioritized Fabry disease program in January 2022 to focus pipeline on Gaucher disease programs, cystinosis, Hunter syndrome and Pompe disease programs, extending cash runway into the first quarter of 2024

Presented updated safety data on first 14 patients treated across two AVROBIO clinical trials at the virtual 28^th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

• No AEs or serious adverse events related to drug product in 14 patients dosed in Phase 1 and 2 Fabry disease trials and Phase 1/2 Gaucher disease trial. All AEs observed have been attributed to myeloablative conditioning, stem cell mobilization, underlying disease or pre-existing conditions.
• Post-gene therapy administration follow-up out more than 4 ½ years for first patient infused.
• AVROBIO shared new industry-leading techniques designed to better elucidate the safety profile of investigational gene therapies at cellular level.
• Full data can be accessed here.

Business Updates

• Appointed Sean O’Bryan, who brings a wealth of experience in regulatory strategy and product development for cell and gene therapies, to chief regulatory officer in February 2022.
• Appointed Azadeh Golipour, Ph.D., who has filled multiple roles with increasing responsibility during her five-year career at AVROBIO, to chief technology officer in January 2022.
• Appointed Essra Ridha, M.D., MRCP, FFPM, who was previously clinical development lead at AVROBIO and has extensive experience in cell and gene therapy development gained at Sangamo Therapeutics and GlaxoSmithKline, to chief medical officer in October 2021.

Anticipated Milestones Over the Next 12 Months:

• AVR-RD-04 in cystinosis: Plan to engage with regulatory agencies to discuss clinical development and regulatory strategy with the intent of initiating a company-sponsored clinical trial in 2023, subject to regulatory clearance.
• Advancing our Gaucher disease franchise:
  • AVR-RD-02 in Gaucher disease type 1: Plan to provide an interim clinical data update in 2022.
  • AVR-RD-06 in Gaucher disease type 3: Plan to engage with regulatory agencies on a Phase 2/3 clinical development strategy.
• AVR-RD-05 in Hunter syndrome: Subject to regulatory clearance, collaborators at the University of Manchester plan to initiate a Phase 1/2 clinical trial in 2023.
• AVR-RD-03 in Pompe disease: Plan to engage with regulatory agencies on the clinical development strategy and plan to initiate a clinical trial in 2023, subject to regulatory clearance.

Fourth Quarter and Year End 2021 Financial Results

AVROBIO reported a net loss of $28.2 million for the fourth quarter of 2021, and a net loss of $119.1 million for the year ended 2021, as compared to a net loss of $28.1 million and a net loss of $119.7 million for the comparable periods in 2020, respectively.

Research and development expenses were $19.0 million for the fourth quarter of 2021, and $83.1 million for the year ended 2021, as compared to $19.6 million and $87.2 million for the comparable periods in 2020, respectively. These decreases were driven by decreased program development expenses, including $9.1 million in non-recurring license fees incurred in 2020, which included an $8.0 million expense related to a one-time, upfront fee paid as consideration for in-licensing the Hunter syndrome program, which were partially offset by an increase in personnel-related costs.

General and administrative expenses were $9.0 million for the fourth quarter of 2021, and $35.7 million for the year ended 2021, as compared to $8.5 million and $33.0 million for the comparable periods in 2020, respectively. These increases were primarily due to an increase in personnel-related costs and non-cash stock-based compensation, which were partially offset by a decrease in professional fees, legal fees and facilities costs.

As of Dec. 31, 2021, AVROBIO had $189.6 million in cash and cash equivalents, as compared to $259.7 million in cash and cash equivalents as of Dec. 31, 2020. Based on the company’s current operating plan, AVROBIO expects its cash and cash equivalents as of Dec. 31, 2021 will enable the company to fund its operating expenses and capital expenditure requirements into the first quarter of 2024.