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|
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(State or other jurisdiction of incorporation)
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(Commission File Number)
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(I.R.S. Employer Identification No.)
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
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Title of each class
|
Trading
symbol(s)
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Name of each exchange
on which registered
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||
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Item 2.02 |
Results of Operations and Financial Condition.
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Item 9.01 |
Financial Statements and Exhibits.
|
Press release issued by AVROBIO, Inc., dated May 11, 2023.
|
|
104
|
The cover page from this Current Report on Form 8-K, formatted in Inline XBRL.
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AVROBIO, INC.
|
||
Date: May 11, 2023
|
By:
|
/s/ Erik Ostrowski
|
Erik Ostrowski
|
||
President, Interim Chief Executive Officer, Chief Financial Officer and Treasurer
|
• |
“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).
|
• |
“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.
|
• |
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 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).
|
March 31,
2023
|
December 31,
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
|
Three Months Ended
March 31,
|
||||||||
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
|