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AVROBIO Reports First Quarter 2021 Financial Results and Provides Business Update
AVROBIO Reports First Quarter 2021 Financial Results and Provides Business Update
Held meeting with the
Dosing completion in ongoing FAB-GT Phase 2 trial reaches 50%, with three additional patients dosed in three months
“During the first quarter, we met with the
Program Updates and Milestones
Held meeting with FDA to discuss the regulatory path for AVR-RD-01 for Fabry disease in the first quarter of 2021
-
Based on a recent
U.S. regulatory development for Fabry disease therapies,AVROBIO intends to discuss with FDA a potential registration trial with a proposed primary efficacy surrogate endpoint of clearance of Gb3 inclusions in biopsied renal peritubular capillaries as the basis for potential full approval. See full update here. -
Subject to FDA feedback,
AVROBIO intends to pursue full approval for investigational AVR-RD-01 as a first-line therapy for Fabry disease by conducting a single, head-to-head registration trial versus Fabrazyme® (agalsidase beta)1 using a kidney biopsy surrogate endpoint similar to the FAB-GT Phase 2 trial, where the company has seen 100% and 87% substrate reductions at one year post-gene therapy in the two patients with evaluable kidney biopsies. The company anticipates proposing to FDA a registration trial design with a scope, size and duration comparable to other gene therapy trials. -
Although FDA guidance provides that a surrogate endpoint that was the basis for approval in a particular clinical development program should not be assumed to be appropriate for use in a different program,
AVROBIO believes Fabrazyme’s full approval based on a similar kidney biopsy surrogate endpoint could potentially support the use of such an endpoint in a registration trial of AVR-RD-01.
Three additional patients dosed in three months and plans to amend FAB-GT trial protocol
- Three patients have now been dosed over the past three months in the FAB-GT Phase 2 trial. Seven patients of the up to 14 planned trial participants have been dosed to date.
-
AVROBIO expects to amend the FAB-GT trial protocol in the second quarter of 2021 to help support the potential use of AVR-RD-01 in a broad Fabry disease population. The company plans to enroll female participants, eliminate antibody-status exclusions and add the collection of data on additional parameters that are recognized to be limitations of enzyme replacement therapy (ERT), such as endpoints to assess the gene therapy’s potential ability to address cardiovascular and central nervous system manifestations.
Four data abstracts accepted for presentation at the 24th virtual Annual Meeting of the
- New data updates from an investigator-sponsored Phase 1/2 trial2in cystinosis and from AVROBIO’s preclinical program in Pompe disease, as well as encore data from AVROBIO’s clinical program in Gaucher disease type 1, have been presented.
-
Encore data from AVROBIO’s clinical program in Fabry disease will be presented on
Friday, May 14, 2021 . - See more information here.
Provided robust data updates for 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 both the Phase 1 and Phase 2 Fabry trials.
- Showed improvement across multiple measures for first two patients dosed in the investigator-sponsored Phase 1/2 trial in cystinosis, and 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 ERT.
- Additional details on the data presented during the 17th annual WORLDSymposium™ can be found here.
First Quarter 2021 Financial Results
Research and development expenses were
General and administrative expenses were
Other (expense) income, net was
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, including timing and design of our potential registration trial, the intended use of such trial as our registration trial for this product candidate, anticipated interactions with regulatory agencies and the planned use of surrogate endpoints in future development of AVR-RD-01, 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 |
|||||||
(In thousands) |
|||||||
(Unaudited) |
|||||||
|
|
||||||
2021 |
2020 |
||||||
Cash and cash equivalents |
$ |
233,027 |
$ |
259,682 |
|||
Prepaid expenses and other current assets |
|
9,037 |
|
7,560 |
|||
Property and equipment, net |
|
2,930 |
|
3,064 |
|||
Other assets |
|
918 |
|
928 |
|||
Total assets |
$ |
245,912 |
$ |
271,234 |
|||
Accounts payable |
$ |
2,698 |
$ |
2,682 |
|||
Accrued expenses and other current liabilities |
|
10,070 |
|
13,932 |
|||
Deferred rent, net of current portion |
|
214 |
|
276 |
|||
Total liabilities |
$ |
12,982 |
$ |
16,890 |
|||
|
|
|
|
||||
Total stockholders’ equity |
|
232,930 |
|
254,344 |
|||
Total liabilities and stockholders’ equity |
$ |
245,912 |
$ |
271,234 |
|||
|
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
||||||||
(In thousands, except per share data) |
||||||||
(Unaudited) |
||||||||
Three Months Ended
|
||||||||
2021 |
2020 |
|||||||
Operating expenses: |
||||||||
Research and development |
$ |
18,527 |
|
$ |
18,274 |
|
||
General and administrative |
|
8,357 |
|
|
|
8,315 |
|
|
Total operating expenses |
|
26,884 |
|
|
|
26,589 |
|
|
|
|
|
|
|||||
Loss from operations |
|
(26,884 |
) |
|
|
(26,589 |
) |
|
Other (expense) income, net |
|
(15 |
) |
|
|
616 |
|
|
Net loss |
$ |
(26,899 |
) |
$ |
(25,973 |
) |
||
Net loss per share — basic and diluted |
$ |
(0.65 |
) |
$ |
(0.77 |
) |
||
Weighted-average number of common shares
|
|
41,618 |
|
|
33,667 |
|
1 Fabrazyme® (agalsidase beta) is a registered trademark owned by Sanofi Genzyme.
2 Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to
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