AVROBIO Reports First Quarter 2022 Financial Results and Provides Business Update

AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today reported financial results for the first quarter ended March 31, 2022 and provided a business update.

“This is an important year for AVROBIO with key milestones that we believe will elevate the company profile and drive significant value for stockholders,” said Geoff MacKay, president and CEO of AVROBIO. “Earlier in the year, we made the strategic decision to reprioritize our pipeline activities and streamline our organization, which not only strengthened our cash position, but also focused our efforts to further advance our cystinosis and Gaucher disease type 3 programs into late-stage trials.

“Collectively, our pipeline targets more than 40,000 patients. Within each indication there is a significant unmet medical need and a high annual cost associated with the standard of care, which we believe translates into a multi-billion-dollar market opportunity across our pipeline,” he said. “These programs either currently, or once we shift to company-sponsored clinical trials will, leverage our plato® gene therapy platform, which has been designed to enable our manufacturing, analytics and delivery to be Phase 3-ready and we believe this will allow us to bring these therapies to patients efficiently and expeditiously.

“At the WORLDSymposium™ in February 2022, we highlighted new interim clinical data that we believe support clinical proof-of-concept for AVR-RD-04 in adults with cystinosis and lays the groundwork for the initiation of a company-sponsored trial planned for 2023. We plan to show additional data from the collaborator-sponsored Phase 1/2 clinical trial, which is now fully enrolled, at the upcoming ASGCT meeting. Patient enrollment continues to progress in our GUARD1 Phase 1/2 trial for Gaucher disease type 1, and we look forward to providing a broad program update later in the year that will include new GUARD1 clinical data, as well as outline our development strategy for our Gaucher disease type 3 program. Lastly, we also anticipate regulatory interactions for our Hunter syndrome and Pompe disease programs later this year, both of which we’re planning to advance into the clinic in 2023,” said MacKay. “We’re driven every day by the potential of our proven platform to dramatically improve the lives of thousands of patients with debilitating lysosomal disorders. The team is working with urgency to advance our pipeline and we believe the multiple clinical and regulatory milestones anticipated over the coming months will help crystallize our efforts to bring our gene therapies to new patients. We look forward to sharing our progress over the course of the year.”

Program Updates

Dosed a fifth patient and enrolled a sixth patient in the Phase 1/2 collaborator-sponsored trial for cystinosis:

• Clinical trial for the first and only gene therapy for cystinosis is now fully enrolled
• First-of-its-kind collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to University of California San Diego (UCSD) from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH)

Enrolled two additional patients in the Phase 1/2 GUARD1 clinical trial for Gaucher disease type 1:

• Clinical protocol calls for 8 to 16 patients between the ages of 18 and 50 with Gaucher disease type 1 to be enrolled, including both those who are treatment-naïve and those who are stable on enzyme replacement therapy

Data updates on two programs to be provided at American Society of Gene and Cell Therapy (ASGCT) 25^th annual meeting being held May 16-19, 2022, in Washington, D.C.:

• New data from the ongoing, collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 (CTNS-RD-04) in cystinosis will be presented on May 16, 2022 at 3 p.m., ET by Stephanie Cherqui, Ph.D., lead investigator of the clinical trial and associate professor of Pediatrics at UCSD
• Pre-clinical data demonstrating long-term efficacy and safety from the company’s Pompe disease program will be presented on May 18, 2022 at 5:30 p.m., ET by Niek van Til, Ph.D., consultant for AVROBIO

Anticipated Milestones Over the Next 12 Months:

• AVR-RD-04 in cystinosis: Plan to engage in 2022 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 alignment
• 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 with aim to initiate a trial in 2023, subject to regulatory alignment
• AVR-RD-05 in Hunter syndrome: Collaborators at the University of Manchester plan to initiate a Phase 1/2 clinical trial in 2023, subject to regulatory alignment
• AVR-RD-03 in Pompe disease: Plan to engage with regulatory agencies on clinical development strategy and plan to initiate a clinical trial in 2023, subject to regulatory alignment

First Quarter 2022 Financial Results
AVROBIO reported a net loss of $29.8 million for the first quarter of 2022 as compared to a net loss of $26.9 million for the comparable period in 2021. This increase was driven by increased research and development expenses as well as increased general and administrative expenses.

Research and development expenses were $19.3 million for the first quarter of 2022 as compared to $18.5 million for the comparable period in 2021. This increase was driven by an increase in clinical trial consulting expenses which was partially offset by a decrease in personnel-related costs, including non-cash stock-based compensation.

General and administrative expenses were $10.2 million for the first quarter of 2022 as compared to $8.4 million for the comparable period in 2021. This increase was attributable to an increase in personnel-related costs driven by severance costs related to the January 2022 workforce reduction and an increase in other expenses, primarily related to facilities costs, professional fees and legal fees.

Other (expense) income, net was $0.4 million in expense for the first quarter of 2022 as compared to other (expense) income, net of $0.02 million in expense for the comparable period in 2021. This increase was driven by interest expense related to our loan and security agreement with Silicon Valley Bank which we entered into during the fourth quarter of 2021.

As of March 31, 2022, AVROBIO had $161.7 million in cash and cash equivalents, as compared to $189.6 million in cash and cash equivalents as of December 31, 2021. Based on the company’s current operating plan, AVROBIO expects its cash and cash equivalents as of March 31, 2022 will enable the company to fund its operating expenses and capital expenditure requirements into the first quarter of 2024.