Press Release
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AVROBIO Reports Second Quarter 2021 Financial Results and Provides Business Update
AVROBIO Reports Second Quarter 2021 Financial Results and Provides Business Update
Company is planning to initiate multiple registration trials in 2022
Multiple data and regulatory updates anticipated over next 12 months
“Data from the patients dosed to date across three indications continue to support our first-in-class, one-time investigational gene therapies as potentially transformative treatments for the more than 50,000 people worldwide living with the life-limiting lysosomal disorders we are researching,” said
Multiple program milestones anticipated over the next 12 months
AVR-RD-01 in Fabry disease:
-
Two additional patients have been dosed in the FAB-GT trial since
June 2021 . -
AVROBIO intends to amend the FAB-GT Phase 2 trial protocol inAugust 2021 to include female participants and eliminate the antibody status exclusion as well as collect additional central nervous system (CNS) and cardiovascular data. -
Company plans to engage the
U.S. Food and Drug Administration (FDA) to discuss a revised regulatory approach with the goal of initiating a registration trial in mid-2022. -
AVROBIO expects to present updated safety and tolerability data on all nine FAB-GT Phase 2 patients dosed to date in the fourth quarter of 2021 and updated efficacy and durability data for all dosed patients at the 18th Annual WORLDSymposium inFebruary 2022 .
AVR-RD-04 in cystinosis:
-
FDA granted Fast Track Designation for AVR-RD-04 and cleared the Investigational New Drug (IND) application for the
AVROBIO long-term follow up trial for patients dosed in the investigator-sponsored Phase 1/2 clinical trial1 of AVR-RD-04 (CTNS-RD-04) led by our collaboration partner atUniversity of California San Diego (UCSD). -
Stephanie Cherqui , Ph.D., principal investigator of the investigator-sponsored Phase 1/2 clinical study, provided an update on the patients dosed to date in the Phase 1/2 trial at the virtual 24th Annual Meeting of theAmerican Society of Gene & Cell Therapy (ASGCT),May 11-14, 2021 . -
AVROBIO expects to provide a clinical and regulatory update in the first quarter of 2022 and is planning to initiate a company-sponsored clinical trial in the second half of 2022, which it believes could potentially serve as a registration trial, subject to regulatory clearance.
Gaucher disease programs:
-
AVR-RD-02 in Gaucher disease type 1:
-
The first patient dosed has completed the 48-week Phase 1/2 Guard1 trial and has enrolled in the long-term follow up trial, where patients will be followed for 14 years after the initial trial period.
AVROBIO is currently enrolling additional patients in the Phase 1/2 trial. -
AVROBIO expects to present updated safety data on Patient 1 in the fourth quarter of 2021 and a full program update in the first half of 2022.
-
The first patient dosed has completed the 48-week Phase 1/2 Guard1 trial and has enrolled in the long-term follow up trial, where patients will be followed for 14 years after the initial trial period.
-
AVR-RD-06 in Gaucher disease type 3:
-
AVROBIO plans to engage regulatory agencies to discuss the regulatory strategy for AVR-RD-06, the company’s program for Gaucher disease type 3, a form of Gaucher disease associated with severe neurological symptoms. - The company is planning to initiate a potential registration trial of AVR-RD-06 in patients with Gaucher disease type 3 in the second half of 2022, subject to regulatory clearance.
-
Second Quarter 2021 Financial Results
Research and development expenses were
General and administrative expenses were
As of
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 product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, our plans and expectations with respect to the development of AVR-RD-01, AVR-RD-04 and AVR-RD-06, including timing and design of potential registration trials for such product candidates, the intended use of such trials as our registration trials for these product candidates, and anticipated interactions with regulatory agencies, the timing of new clinical and regulatory updates, 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, the expected safety profile of our investigational gene therapies, 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
|
|||||
|
|
||||
Cash and cash equivalents |
$ |
226,438 |
$ |
259,682 |
|
Prepaid expenses and other current assets |
|
7,081 |
|
7,560 |
|
Property and equipment, net |
|
3,990 |
|
3,064 |
|
Other assets |
|
586 |
|
928 |
|
Total assets |
$ |
238,095 |
$ |
271,234 |
|
Accounts payable |
$ |
2,029 |
$ |
2,682 |
|
Accrued expenses and other current liabilities |
|
14,924 |
|
13,932 |
|
Deferred rent, net of current portion |
|
150 |
|
276 |
|
Total liabilities |
|
17,103 |
|
16,890 |
|
Total stockholders’ equity |
|
220,992 |
|
254,344 |
|
Total liabilities and stockholders’ equity |
$ |
238,095 |
$ |
271,234 |
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
|
|||||||||||||||
Three Months Ended |
Six Months Ended |
||||||||||||||
2021 |
2020 |
2021 |
2020 |
||||||||||||
Operating expenses: |
|
||||||||||||||
Research and development |
$ |
22,544 |
|
$ |
20,866 |
|
$ |
41,024 |
|
$ |
39,140 |
|
|||
General and administrative |
|
8,831 |
|
|
7,991 |
|
|
17,235 |
|
|
16,306 |
|
|||
Total operating expenses |
|
31,375 |
|
|
28,857 |
|
|
58,259 |
|
|
55,446 |
|
|||
Loss from operations |
|
(31,375 |
) |
|
(28,857 |
) |
|
(58,259 |
) |
|
(55,446 |
) |
|||
Total other (expense) income, net |
|
(12 |
) |
|
29 |
|
|
(27 |
) |
|
645 |
|
|||
Net loss |
($ |
31,387 |
) |
($ |
28,828 |
) |
($ |
58,286 |
) |
($ |
54,801 |
) |
|||
Net loss per share — basic and diluted |
($ |
0.74 |
) |
($ |
0.80 |
) |
($ |
1.39 |
) |
($ |
1.57 |
) |
|||
Weighted-average number of common shares outstanding — basic and diluted |
|
42,510 |
|
|
36,105 |
|
|
42,067 |
|
|
34,886 |
|
1 Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to
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