AVROBIO Reports First Quarter 2023 Financial Results and Provides Business Update
AVROBIO Reports First Quarter 2023 Financial Results and Provides Business Update
On track to initiate registrational global Phase 2/3 clinical trial for Gaucher disease type 3 (GD3) in second half 2023, subject to regulatory alignment
Plan to provide clinical and regulatory updates on cystinosis program in conjunction with ASGCT annual meeting in
Collaborator-sponsored Phase 1/2 clinical trial for mucopolysaccharidosis type II (MPS-II), or Hunter syndrome, initiated
Announced appointment of current CFO
“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
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 theU.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. andU.K. , and an Innovation Passport by MHRA under theInnovative 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 ofJan. 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 inmid-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. andU.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
First Quarter 2023 Financial Results
Research and development expenses were
General and administrative expenses were
Other income (expense), net was
As of
About
Our vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into patients’ own hematopoietic stem cells (HSCs), with the goal of durably expressing the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for Gaucher disease, cystinosis and Hunter syndrome, as well as a preclinical program for Pompe disease. Our proprietary plato® gene therapy platform is scalable for planned global commercialization. We are headquartered in
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 forward-looking terminology such as “aims,” “anticipates,” “believes,” “continue,” “could,” “designed to,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “predicts,” “projects,” “seeks,” “strives,” “should,” “will,” and similar expressions or the negative of these terms. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our current and prospective preclinical and clinical 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, preclinical, compassionate use or clinical trial results, product approvals and regulatory pathways, the timing of patient recruitment and enrollment activities, our expectations with respect to our plans with collaborators, 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, statements regarding a leadership transition including the appointment of an interim CEO and our search to identify a permanent CEO, 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.
Any forward-looking statements in this press release are based on AVROBIO’s current expectations, estimates and projections about our industry as well as management’s current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIO’s product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of
CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands) (Unaudited) |
||||||
|
|
|||||
2023 |
2022 |
|||||
|
||||||
Cash and cash equivalents |
$ |
72,326 |
$ |
92,563 |
||
Prepaid expenses and other current assets |
|
4,925 |
|
7,112 |
||
Property and equipment, net |
|
2,574 |
|
2,894 |
||
Operating lease assets |
|
2,857 |
|
1,057 |
||
Other assets |
|
323 |
|
323 |
||
Total assets |
$ |
83,005 |
$ |
103,949 |
||
|
|
|
||||
Accounts payable |
$ |
591 |
$ |
384 |
||
Accrued expenses and other current liabilities |
|
11,081 |
|
11,732 |
||
Note payable, net of discount |
|
15,356 |
|
15,276 |
||
Operating lease liabilities |
|
2,979 |
|
1,187 |
||
Total liabilities |
|
30,007 |
|
28,579 |
||
|
|
|
||||
Total stockholders’ equity |
|
52,998 |
|
75,370 |
||
Total liabilities and stockholders’ equity |
$ |
83,005 |
$ |
103,949 |
||
|
|
|||||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In thousands, except per share data) (Unaudited) |
||||||
|
Three Months Ended
|
|||||
|
2023 |
2022 |
||||
|
|
|
||||
Operating expenses: |
|
|
||||
Research and development |
$ |
17,333 |
$ |
19,253 |
||
General and administrative |
|
7,887 |
|
10,165 |
||
Total operating expenses |
|
25,220 |
|
29,418 |
||
|
|
|
||||
Loss from operations |
|
(25,220) |
|
(29,418) |
||
Other income (expense), net |
|
263 |
|
(415) |
||
Net loss |
$ |
(24,957) |
$ |
(29,833) |
||
Net loss per share — basic and diluted |
$ |
(0.57) |
$ |
(0.68) |
||
Weighted-average number of common shares outstanding — basic and diluted |
|
44,037 |
|
43,695 |
||
____________________________
1 Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to
View source version on businesswire.com: https://www.businesswire.com/news/home/20230511005197/en/
Investor Contact:
Westwicke, an
339-970-2843
chris.brinzey@westwicke.com
Media Contact:
Ten
617-999-9620
krodophele@tenbridgecommunications.com
Source: