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AVROBIO Reports Second Quarter 2019 Financial Results and Provides Business Update
AVROBIO Reports Second Quarter 2019 Financial Results and Provides Business Update
Reported positive interim results from clinical trials of AVR-RD-01 investigational gene therapy in Fabry disease, including 87% substrate reduction in first kidney biopsy at one year post-treatment
On track to dose first patients in Phase 1/2 clinical trials for Gaucher and cystinosis this year
Raised
“We have made substantial progress across our entire pipeline, especially in our clinical program for AVR-RD-01 in Fabry disease. We believe the compelling interim data from the Phase 2 and Phase 1 trials announced in July serves to both validate our approach to treating patients with Fabry disease, as well as support our pipeline of investigational gene therapies for other lysosomal storage disorders,” commented
Program Updates and Milestones
- The Company announced positive data from the Phase 2 and Phase 1 clinical trials of AVR-RD-01 in Fabry disease, including the following highlights:
- Substantial Gb3 substrate reduction in kidney biopsy. The kidney biopsy for the first treatment-naïve patient dosed in the Company’s FAB-2011 Phase 2 trial, as reviewed by two independent examiners, showed a reduction from an average of 3.55 Gb3 inclusions per peritubular capillary (PTC)2 at baseline to an average of 0.47 inclusions per PTC one year after administration of the Company’s AVR-RD-01 investigational gene therapy, representing an 87% reduction.
- Sustained plasma lyso-Gb3 reductions. The first Phase 2 patient had an 87% reduction in plasma lyso-Gb3 at one year. The first four Phase 1 patients had their plasma lyso-Gb3 levels reduced between 33% and 41% versus their enzyme replacement therapy (ERT) pre-treatment levels. In particular, the 41% reduction level has stabilized at more than two years for the first Phase 1 patient.
- Durability data for AVR-RD-01 continues to show sustained results across multiple parameters. All six patients across the Phase 1 and Phase 2 trials for whom data are reported at six months or longer post-treatment with AVR-RD-01 show sustained AGA enzyme activity in leukocytes and plasma and exhibit consistent vector copy number (VCN) trends, with VCN levels for the first Phase 1 patient stable at more than two years post-treatment.
- Kidney and cardiac functions stable. Kidney and cardiac functions, as measured by GFR3 and cardiac MRI Left Ventricular Mass parameters, were stable in a normal range in the first Phase 2 patient at one year.
- Phase 1 patients who have discontinued ERT remain off ERT. The two patients in the Phase 1 trial who discontinued ERT post-AVR-RD-01 treatment remain off ERT to date. A third patient in the Phase 1 trial recently discontinued ERT treatment.
- No unexpected trends or safety events were identified. Serious adverse events (SAEs) and adverse events (AEs) reported were generally consistent with myeloablative conditioning, underlying disease or pre-existing conditions.
Recruitment continues to progress in FAB-201 in
- Phase 1/2 trial of AVR-RD-02 in Gaucher disease (GAU-201) remains on track with expected dosing of first patient, incorporating the plato platform, this year.
AVROBIO received a ‘no objection letter’ for its GAU-201 clinical trial application (CTA) fromHealth Canada and intends also to open clinical sites inAustralia and additional countries next year, pending regulatory clearances. Manufacturing infrastructure is in place to support clinical trials in the U.S.,Canada andAustralia . The Phase 1/2 trial, which is currently recruiting patients inCanada , is designed to enroll 8 to 16 patients between the ages of 16 and 35 with Type 1 Gaucher disease and will include both treatment-naïve patients as well as patients stable on ERT. Patients will receive a single treatment with AVR-RD-02 and will be followed for one year to measure safety and efficacy. Efficacy endpoints include measures of liver and spleen volumes, hemoglobin, platelet counts, bone pain and bone density measures, along with other blood markers used in Gaucher disease clinical research. In the second quarter,AVROBIO announced new pre-clinical data at the Annual Meeting of theAmerican Society of Gene and Cell Therapy (ASGCT) highlighting, in a mouse model for Type 1 Gaucher disease, the impact AVR-RD-02 had on bone pathology, an important aspect of the target product profile and a significant unmet need for Gaucher patients, including for those currently being treated by ERT. - Phase 1/2 clinical trial of AVR-RD-04 in cystinosis remains on track with expected dosing of first patient this year. The Phase 1/2 clinical trial evaluating AVR-RD-04 in cystinosis, an investigator-sponsored trial led by Dr.
Stephanie Cherqui at theUniversity of California, San Diego (UCSD), is expected to dose its first patient this year. The clinical trial is designed to enroll 6 patients with cystinosis currently being treated with cysteamine to examine clinical parameters such as kidney function, muscle strength, bone density, and endocrine function. InJune 2019 , the UCSD cystinosis study group announced that it had been awarded a$12.0 million grant from theCalifornia Institute for Regenerative Medicine (CIRM) to directly fund this Phase 1/2 trial. - Strengthened balance sheet and extended anticipated cash runway into the second half of 2021. In
July 2019 , the Company raised net proceeds of approximately$129.5 million through a follow-on equity offering. The Company expects that these net proceeds, along with AVROBIO’s cash and cash equivalents as ofJune 30, 2019 , will enable the Company to fund its operating expenses and capital expenditure requirements into the second half of 2021.
Second Quarter 2019 Financial Results
Research and development expenses were
General and administrative expenses were
As of
About
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 such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy, prospective products and goals, the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, potential regulatory approvals and the timing thereof, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, the expected safety profile of our product candidates, timing and likelihood of success of our current or future product candidates, plans and objectives of management for future operations, future results of anticipated products, and the market opportunity for our product candidates, and statements regarding the Company’s 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
1 The official name of ‘FAB-201’ is AVRO-RD-01-201, which is a Phase 2 trial of AVROBIO’s investigational gene therapy, AVR-RD-01, in Fabry disease.
2 Peritubular capillaries (PTCs), also referred to as kidney interstitial capillaries (KICs), convey blood after filtration in the glomeruli, enabling it to eventually exit the kidneys and return to the circulatory system.
3 Glomerular Filtration Rate (GFR) includes estimated GFR (eGFR) determined using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula, and measured GFR (mGFR) determined using plasma clearance of iohexol.
CONDENSED CONSOLIDATED BALANCE SHEETS
|
|||||||||
June 30, |
December 31, |
||||||||
|
2019 |
|
2018 |
||||||
Cash and cash equivalents |
$ |
90,348 |
$ |
126,302 |
|||||
Prepaid expenses and other current assets |
|
7,840 |
|
3,718 |
|||||
Property and equipment, net |
|
2,741 |
|
2,634 |
|||||
Other assets |
|
1,060 |
|
825 |
|||||
Total assets |
$ |
101,989 |
$ |
133,479 |
|||||
Accounts payable |
$ |
2,572 |
$ |
2,784 |
|||||
Accrued expenses and other current liabilities |
|
6,193 |
|
7,822 |
|||||
Deferred rent, net of current portion |
|
590 |
|
|
689 |
||||
Total liabilities |
|
9,355 |
|
11,295 |
|||||
Total stockholders’ equity |
|
92,634 |
|
122,184 |
|||||
Total liabilities and stockholders’ equity |
$ |
101,989 |
$ |
133,479 |
|||||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
|
||||||||||||||||
Three Months Ended June 30, |
Six Months Ended June 30, |
|||||||||||||||
|
2019 |
|
|
2018 |
|
|
2019 |
|
|
2018 |
|
|||||
Operating expenses: |
|
|||||||||||||||
Research and development |
$ |
12,267 |
|
$ |
7,407 |
|
$ |
24,713 |
|
$ |
13,054 |
|
||||
General and administrative |
|
4,345 |
|
|
|
2,140 |
|
|
|
9,599 |
|
|
|
4,281 |
|
|
Total operating expenses |
|
16,612 |
|
|
|
9,547 |
|
|
|
34,312 |
|
|
|
17,335 |
|
|
|
|
|
|
|
|
|
||||||||||
Loss from operations |
|
(16,612 |
) |
|
|
(9,547 |
) |
|
|
(34,312 |
) |
|
|
(17,335 |
) |
|
Total other income (expense), net |
|
557 |
|
|
|
(960 |
) |
|
|
1,154 |
|
|
|
(1,414 |
) |
|
Net loss |
$ |
(16,055 |
) |
$ |
(10,507 |
) |
$ |
(33,158 |
) |
$ |
(18,749 |
) |
||||
|
|
|
|
|
|
|
|
|||||||||
Reconciliation of net loss to net loss attributed to common stockholders: Net loss |
$ |
(16,055 |
) |
|
$ |
(10,507 |
) |
|
$ |
(33,158 |
) |
|
$ |
(18,749 |
) |
|
Accretion of issuance costs on convertible preferred stock |
|
- |
|
|
|
- |
|
|
|
- |
|
|
|
(2,243 |
) |
|
Net loss attributable to common stockholders – basic and diluted |
$ |
(16,055 |
) |
|
$ |
(10,507 |
) |
|
$ |
(33,158 |
) |
|
$ |
(20,992 |
) |
|
|
||||||||||||||||
Net loss per share attributable to common stockholders — basic and diluted |
$ |
(0.67 |
) |
$ |
(2.98 |
) |
$ |
(1.38 |
) |
$ |
(7.16 |
) |
||||
Weighted-average number of common shares used in computing net loss per share attributable to common stockholders—basic and diluted |
|
24,046,262 |
|
|
3,529,269 |
|
|
23,985,717 |
|
|
2,930,358 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20190808005181/en/
Source:
Investor Contact:
Christopher F. Brinzey
Westwicke, an ICR Company
339-970-2843
chris.brinzey@westwicke.com
Media Contact:
Kathryn Morris
The Yates Network
914-204-6412
kathryn@theyatesnetwork.com