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AVROBIO Reports 1Q 2020 Financial Results and Provides Business Update
AVROBIO Reports 1Q 2020 Financial Results and Provides Business Update
Three oral presentations accepted on new clinical and preclinical data at the
Completed two collaboration agreements to reinforce technological leadership in lentiviral gene therapy
Raised
“Our strong progress has continued, as evidenced by recently announced collaborations with
New Collaboration Agreements
Announced collaboration with
-
New development and commercialization agreement with
Saladax Biomedical focused on developing and validating a fully automated nanoparticle immunoassay kit designed to simplify, streamline and optimize therapeutic drug monitoring (TDM) for patients treated with the conditioning agent busulfan. - High-speed assay kit has the potential to reduce from hours to minutes the time it takes to evaluate how quickly a patient metabolizes busulfan and enable many more hospitals and clinics to become TDM-capable sites.
Announced collaboration with Magenta Therapeutics to study novel antibody-drug conjugate conditioning regimen as part of strategic focus on maintaining technology leadership in gene therapy
- New research and clinical collaboration agreement with Magenta Therapeutics will evaluate the potential utility of MGTA-117, Magenta’s novel antibody-drug conjugate for conditioning patients before they receive one of AVROBIO’s investigational lentiviral gene therapies.
Program Updates
AVR-RD-01 clinical trials in Fabry disease:
-
Ongoing data collection has continued for dosed patients in both trials, though certain data collection has been temporarily delayed. Patient identification activities for the Phase 2 FAB-201 trial continue for our trial sites in
Australia ,Canada and theU.S. Dosing of new patients at our clinical sites, which has been paused as a result of the COVID-19 pandemic, is expected to resume as our clinical sites allow. -
New data from the
AVROBIO -sponsored Phase 2 trial of AVR-RD-01 for Fabry disease will be presented during an oral presentation at ASGCT, byBirgitte Volck , M.D., Ph.D., AVROBIO’s president of R&D, onWednesday, May 13, 2020 , from5:00-5:15 p.m. ET .
AVR-RD-04 Phase 1/2 trial in cystinosis:
AVROBIO’s investigational gene therapy for cystinosis (AVR-RD-04) is being evaluated in a single-arm, Phase 1/2 investigational trial sponsored by the
-
The
U.S. Food and Drug Administration (FDA) granted orphan drug designation for AVR-RD-04 for the treatment of cystinosis inMarch 2020 . - Patient identification activities are continuing. Ongoing data collection has continued for the first dosed patient in the trial, though certain data collection has been temporarily delayed.
-
A second patient has been enrolled in the study and has completed apheresis. Cryopreserved drug product for that patient has been manufactured and dosing is expected to occur as soon as
UCSD allows. -
New data from the collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 in cystinosis will be presented during an oral presentation at ASGCT, by
Stephanie Cherqui , Ph.D., principal investigator, associate professor of pediatrics atUCSD School of Medicine and former chair of the ASGCT Gene andCell Therapy ofGenetic and Metabolic Diseases Committee , onWednesday, May 13, 2020 , from4:30-4:45 p.m. ET .
AVR-RD-02 Phase 1/2 trial in Gaucher disease:
- The first patient in the trial has been enrolled and has completed apheresis. Cryopreserved drug product for that patient has been manufactured and dosing is currently anticipated for Q2, but is dependent on when the clinical site allows.
-
Subsequent new patient enrollment timelines have been impacted by the COVID-19 pandemic. Patient identification activities continue for trial sites in
Australia andCanada . -
New trial sites are expected to open in the
U.S. andIsrael in 4Q 2020.
AVR-RD-03 preclinical program in Pompe disease:
New data from AVROBIO’s preclinical research program for a gene therapy for Pompe disease (AVR-RD-03) will be presented during an oral presentation at ASGCT, by
First Quarter 2020 Financial Results
Research and development expenses were
General and administrative expenses were
As of
About
Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in 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 prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, 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, the potential and expected benefits of Saladax’s immunoassay kits, including the ability to improve, simplify and streamline therapeutic drug monitoring for patients treated with the conditioning agent busulfan, the potential and expected benefits of MGTA-117, Magenta’s investigational antibody-drug conjugate, including MGTA-117’s potential application to AVROBIO’s investigational gene therapies as a conditioning agent, the potential impact of the COVID-19 outbreak on our clinical trial programs and business generally, as well as our plans and expectations with respect to the timing and resumption of any development activities that may be temporarily paused as a result of the COVID-19 outbreak, 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 |
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(In thousands) |
||||||
(Unaudited) |
||||||
|
|
|
||||
|
|
|
||||
2020 |
|
2019 |
||||
Cash and cash equivalents |
$ |
257,695 |
$ |
187,043 |
||
Prepaid expenses and other current assets |
|
7,652 |
|
8,658 |
||
Property and equipment, net |
|
3,407 |
|
3,696 |
||
Other assets |
|
974 |
|
1,117 |
||
Total assets |
$ |
269,728 |
$ |
200,514 |
||
Accounts payable |
$ |
2,135 |
$ |
3,949 |
||
Accrued expenses and other current liabilities |
|
10,638 |
|
10,068 |
||
Deferred rent, net of current portion |
|
430 |
|
484 |
||
Total liabilities |
|
13,203 |
|
14,501 |
||
|
|
|
||||
Total stockholders’ equity |
|
256,525 |
|
186,013 |
||
Total liabilities and stockholders’ equity |
$ |
269,728 |
$ |
200,514 |
||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
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(In thousands, except share and per share data) |
||||||
(Unaudited) |
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|
Three Months |
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|
2020 |
|
2019 |
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|
||||||
Operating expenses: |
|
|||||
Research and development |
|
$ |
18,274 |
$ |
12,446 |
|
General and administrative |
|
|
8,315 |
|
|
5,254 |
Total operating expenses |
|
|
26,589 |
|
|
17,700 |
|
|
|
|
|||
Loss from operations |
|
|
(26,589) |
|
|
(17,700) |
|
|
|
|
|
||
Total other income (expense), net |
|
|
616 |
|
|
597 |
|
|
|
|
|
||
|
|
|
|
|
||
Net loss |
|
$ |
(25,973) |
$ |
(17,103) |
|
|
|
|
|
|
||
Net loss attributable to common stockholders – basic and diluted |
|
$ |
(25,973) |
|
$ |
(17,103) |
|
|
|||||
|
||||||
Net loss per share attributable to common stockholders — basic and diluted |
|
$ |
(0.77) |
$ |
(0.72) |
|
|
||||||
Weighted-average number of common shares used in computing net loss per share attributable to common stockholders—basic and diluted |
|
|
33,666,801 |
|
23,893,696 |
|
1 Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to
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Investor Contact:
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339-970-2843
chris.brinzey@westwicke.com
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857-559-3397
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