A leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the first quarter ended March 31, 2023, and provided a business update.
“Validation of a GMP stem cell gene therapy manufacturing process for mucopolysaccharidosis type II (MPS II) in preparation for an approved Phase 1/2 clinical trial”
“We look forward to building upon last year’s positive data and regulatory updates for our lead programs and demonstrating the potential of our HSC gene therapy approach in the year ahead,” said Erik Ostrowski, interim CEO and current CFO of AVROBIO. “We plan to initiate a global, registrational Phase 2/3 trial of our Gaucher disease type 3 (GD3) program later this year and are happy to report that our collaborator-sponsored Phase 1/2 trial for Hunter syndrome has recently commenced enrollment. Additionally, we plan to provide an update on our cystinosis program in mid-May at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.”
Program Highlights and Milestones
AVR-RD-02 for Gaucher disease:
- “The Guard1 clinical trial – A first in-human, Phase 1/2 study evaluating AVR-RD-02, an HSC gene therapy for Gaucher disease: Preliminary safety, pharmacodynamic and clinical efficacy results from the subjects observed for up to 24 months post-infusion” — AVROBIO presented safety and efficacy data at the 19th annual WORLDSymposium™, Feb. 22-26, 2023.
- Guard1 is recruiting individuals between the ages of 16 and 50 with Gaucher disease type 1 (GD1), including those who are treatment-naïve and who are stable on enzyme replacement therapy, with sites in Canada and the U.S.
- “Sustained improvement of clinical CNS and somatic features of GD3 after HSC gene therapy: A first-in-world report” — Clinical data from the first pediatric GD3 patient, dosed with investigational AVR-RD-02, was presented by one of the patient’s physicians from the University of Manchester (UoM), U.K. at WORLDSymposium™. New data included longer time points for peripheral blood glucocerebrosidase, chitotriosidase and albumin levels, all trending consistently with previously presented data. The 11-year-old GD3 patient was dosed at UoM on a named patient basis.
- Plan to initiate Guard3, a global registrational Phase 2/3 trial for GD3, in the second half of 2023, subject to regulatory alignment.
- AVR-RD-02 has been granted Rare Pediatric Disease Designation and Fast Track Designation by FDA, Orphan Drug Designation in the U.S. and U.K., and an Innovation Passport by MHRA under the Innovative Licensing and Access Pathway (ILAP).
AVR-RD-04 for cystinosis:
- “Phase 1/2 clinical trial of autologous hematopoietic stem and progenitor cell (HSPC) gene therapy for cystinosis” — Collaborators at the University of California, San Diego,1 presented updated data on the six patients dosed in the fully enrolled Phase 1/2 clinical trial at WORLDSymposium™, including additional vector copy number (VCN) data, as well as longer time points for leukocyte cystine levels and skin and GI mucosa cystine crystal data, for some patients. All clinical and safety data updates are trending consistently with the prior reported data as of the most recent safety data cut-off date of Jan. 9, 2023.
- Plan to provide a clinical and regulatory update on the cystinosis program at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in mid-May 2023.
- Plan to initiate activities for the Phase 1/2 clinical trial in the second half of 2023 subject to regulatory alignment.
- AVR-RD-04 has been granted Rare Pediatric Disease Designation and Fast Track Designation by FDA and Orphan Drug Designation in the U.S. and U.K.
AVR-RD-05 for neuronopathic mucopolysaccharidosis type II (MPS-II), or Hunter syndrome:
- Collaborator-sponsored Phase 1/2 clinical trial for neuronopathic mucopolysaccharidosis type II (MPS-II), or Hunter syndrome, initiated.
- “Validation of a GMP stem cell gene therapy manufacturing process for mucopolysaccharidosis type II (MPS II) in preparation for an approved Phase 1/2 clinical trial” — Collaborators at UoM highlighted data validating their manufacturing process in preparation for a Phase 1/2 clinical trial for Hunter syndrome anticipated to start later this year at WORLDSymposium.
- AVR-RD-05 has been granted Rare Pediatric Disease Designation and Orphan Drug Designation by FDA.
AVR-RD-03 for Pompe disease:
- AVR-RD-03 is currently being evaluated in a pre-clinical research program and the data to-date have shown significantly reduced toxic accumulation of glycogen in a mouse model of Pompe disease, including in cardiac and skeletal muscle as well as the central nervous system (CNS).
Organizational Update
On May 1, 2023, AVROBIO announced the appointment of Erik Ostrowski as interim CEO. Mr. Ostrowski, who will continue as AVROBIO’s CFO, succeeds founding president and CEO Geoff MacKay, who left to join an emerging early-stage company. The Board of Directors has begun a search to identify a permanent CEO.
First Quarter 2023 Financial Results
AVROBIO reported a net loss of $25.0 million for the first quarter of 2023 as compared to a net loss of $29.8 million for the comparable period in 2022.
Research and development expenses were $17.3 million for the first quarter of 2023 as compared to $19.3 million for the comparable period in 2022. This decrease was driven by a reduction in personnel-related costs, including non-cash stock-based compensation, and partially offset by an increase in program development expenses.
General and administrative expenses were $7.9 million for the first quarter of 2023 as compared to $10.2 million for the comparable period in 2022. This decrease was driven by a decrease in personnel-related costs, including non-cash stock-based compensation.
Other income (expense), net was $0.3 million for the first quarter of 2023 as compared to other (expense) income, net of ($0.4) million for the comparable period in 2022. This increase in other income is due to an increase in interest income which was partially offset by interest expense related to our term loan.
As of March 31, 2023, AVROBIO had $72.3 million in cash and cash equivalents, as compared to $92.6 million in cash and cash equivalents as of Dec. 31, 2022. Based on AVROBIO’s current operating plan, AVROBIO expects its cash and cash equivalents as of March 31, 2023, will enable AVROBIO to fund its operating expenses and capital expenditure requirements into the first quarter of 2024.
Source: https://www.businesswire.com/
