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AVROBIO Reports Fourth Quarter and Fiscal Year 2020 Financial Results and Provides Business Update
AVROBIO Reports Fourth Quarter and Fiscal Year 2020 Financial Results and Provides Business Update
100% kidney substrate reduction at 12 months post-gene therapy in the first patient dosed with the plato® gene therapy platform in FAB-GT1 trial for Fabry disease
One new patient dosed and three new patients enrolled in Fabry disease clinical trial since
Orphan drug designation received from
Balance sheet strengthened with a
“It has been a highly productive period for
Since January, the company has dosed a fifth patient and enrolled three additional patients in the FAB-GT trial for Fabry disease.
“In 2021, we’re focused on operational execution as we prepare for a number of important milestones, including meeting with the
Program Updates and Milestones
Received orphan drug designation (ODD) from the
-
Received ODD from the EC for AVR-RD-01 for Fabry disease in
November 2020 . -
All three
AVROBIO investigational gene therapies in the clinic have received orphan designation from FDA and EC.
Provided robust data updates in Fabry disease, cystinosis and Gaucher disease type 1 during WORLDSymposium™ in
- Announced 100% kidney substrate reduction at 12 months post-gene therapy in the first patient dosed with the plato® platform in the FAB-GT trial, together with additional data from multiple biomarkers and functional cardiac measurements across Phase 1 and Phase 2.
- For the first two patients dosed in the investigator-sponsored Phase 1/2 trial in cystinosis, showed improvement across multiple measures including substantial improvement in photophobia in the first patient at 12 months post-gene therapy.
- Presented early data from the Phase 1/2 clinical trial in Gaucher disease type 1 showing plasma chitotriosidase levels decreased 49% and toxic metabolite lyso-Gb1 levels decreased 44% in the first patient at six months post-gene therapy, compared to the patient’s pre-treatment levels while on enzyme replacement therapy (ERT).
- Additional details on the data presented during the 17th annual WORLDSymposium can be found here.
Expanded management team with appointment of Diana M. Escolar, M.D., FAAN, chief medical officer, bringing expertise and experience in rare diseases, with a focus on lysosomal and neurogenetic disorders, in
Provided updates on ongoing clinical trials and second wave of programs in leading lysosomal disorder gene therapy pipeline at first Virtual R&D Day in
- Shared positive data out as far as 3.5 years across a broad lysosomal disorder gene therapy pipeline.
- Presented data on Bu90-target concentration intervention (TCI), the company’s pioneering approach to personalized conditioning.
- Announced development plans for AVR-RD-06 in Gaucher disease type 3.
- Presented preclinical data from AVR-RD-05 for Hunter syndrome demonstrating normalization of multiple disease measures in a mouse model, including normalization of skeletal features such as the thickness of cheekbone and femur.
- Announced preclinical data for AVR-RD-03 for Pompe disease that showed the normalization of substrate levels in multiple hard-to-reach organs including brain and spinal cord.
- Additional details on the data presented at the Virtual R&D Day can be found here.
Strengthened balance sheet and extended anticipated cash runway into Q1 2023
-
Raised gross proceeds of
$75 million through a follow-on common stock offering inNovember 2020 . -
Based on the company’s current operating plan,
AVROBIO expects its cash and cash equivalents as ofDec. 31, 2020 , will enable the company to fund its operating expenses and capital expenditure requirements into Q1 2023.
Fourth Quarter and Year End 2020 Financial Results
Research and development expenses were
General and administrative expenses were
As of
_____________________________ |
1 Fabry Phase 2 trial, formerly known as FAB-201 |
About
Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease.
Forward-Looking Statements
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 words and phrases such as “aims,” “anticipates,” “believes,” “could,” “designed to,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our current and prospective product candidates, results of preclinical studies, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, the timing of patient recruitment and enrollment activities, and product approvals, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato® platform in our clinical trials and gene therapy programs, including the use of a personalized and ultra-precision busulfan conditioning regimen, and statements regarding our financial and cash position and expected cash runway. 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) |
||||||
|
|
|
||||
2020 |
|
2019 |
||||
Cash and cash equivalents |
$ |
259,682 |
$ |
187,043 |
||
Prepaid expenses and other current assets |
|
7,560 |
|
8,658 |
||
Property and equipment, net |
|
3,064 |
|
3,696 |
||
Other assets |
|
928 |
|
1,117 |
||
Total assets |
$ |
271,234 |
$ |
200,514 |
||
Accounts payable |
$ |
2,682 |
$ |
3,949 |
||
Accrued expenses and other current liabilities |
|
13,932 |
|
10,068 |
||
Deferred rent, net of current portion |
|
276 |
|
484 |
||
Total liabilities |
|
16,890 |
|
14,501 |
||
|
|
|
||||
Total stockholders’ equity |
|
254,344 |
|
186,013 |
||
Total liabilities and stockholders’ equity |
$ |
271,234 |
$ |
200,514 |
||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
||||||||||||||||
(In thousands, except per share data) |
||||||||||||||||
(Unaudited) |
||||||||||||||||
Three Months Ended
|
|
Twelve Months Ended
|
||||||||||||||
2020 |
|
2019 |
|
2020 |
|
2019 |
||||||||||
Operating expenses: |
|
|||||||||||||||
Research and development |
$ |
19,587 |
|
$ |
17,219 |
|
$ |
87,236 |
|
$ |
54,974 |
|
||||
General and administrative |
|
8,477 |
|
|
|
6,214 |
|
|
|
32,992 |
|
|
|
20,835 |
|
|
Total operating expenses |
|
28,064 |
|
|
|
23,433 |
|
|
|
120,228 |
|
|
|
75,809 |
|
|
|
|
|
|
|
|
|
||||||||||
Loss from operations |
|
(28,064 |
) |
|
|
(23,433 |
) |
|
|
(120,228 |
) |
|
|
(75,809 |
) |
|
Total other income (expense), net |
|
(67 |
) |
|
|
771 |
|
|
|
516 |
|
|
|
2,844 |
|
|
Net loss |
$ |
(28,131 |
) |
$ |
(22,662 |
) |
$ |
(119,712 |
) |
$ |
(72,965 |
) |
||||
|
||||||||||||||||
Net loss per share—basic and diluted |
$ |
(0.73 |
) |
$ |
(0.72 |
) |
$ |
(3.31 |
) |
$ |
(2.66 |
) |
||||
Weighted-average number of common shares outstanding—basic and diluted |
|
38,528 |
|
|
31,629 |
|
|
36,206 |
|
|
27,432 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20210318005244/en/
Investor Contact:
Westwicke, an
339-970-2843
chris.brinzey@westwicke.com
Media Contact:
Ten
617-581-9333
stephanie@tenbridgecommunications.com
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