AVROBIO, Inc. Reports Third Quarter 2018 Financial Results and Provides Business Update
During the quarter, announced updated Fabry clinical data, acceptance of Clinical Trial Application for Gaucher, and continued platform optimization
“The third quarter has been productive for AVROBIO,” commented
Third Quarter and Recent Business Highlights
- Announced clinical data and patient updates from the investigator-sponsored Phase 1 study and the
AVROBIO-sponsored Phase 2 clinical trial (FAB-201) in Fabry disease. In October 2018, AVROBIOprovided updated clinical data on two patients in the Phase 1 study and the first patient enrolled in the FAB‑201 clinical trial for AVR-RD-01 in Fabry disease. As of the cut-off date, three out of three patients with results reported after treatment with AVROBIO’s gene therapy continued to express α‑galactosidase A (the enzyme that is deficient in Fabry disease) at levels above the diagnostic range for males with classic Fabry disease. After the 18-month follow-up visit for the first patient, the clinical investigators received approval, and the patient consented, to discontinue regular bi-weekly treatment with current standard of care enzyme replacement therapy (ERT). Thus far, the gene therapy has been well tolerated with no serious adverse events (SAEs) related to AVR‑RD‑01 (as of the safety data cut-off date of August 24, 2018). The Company plans to provide further updates in the first quarter of 2019.
- Continued enrollment in both the investigator-sponsored Phase 1 study and the FAB-201 clinical trial in 2019. The Phase 1 trial is designed to assess the safety of AVR-RD-01 in up to six patients with Fabry disease who have been treated with ERT for at least six months. As of
October 1, 2018, three patients have been treated in the Phase 1 study and investigators will continue to enroll up to an additional two patients with Fabry disease in the study. The Phase 2 clinical trial (FAB-201) is an open-label, single-arm clinical trial evaluating the safety and efficacy of AVR-RD-01 in 8 to 12 ERT-naive male patients. As of October 1, 2018, one patient has been treated in the Phase 2 trial. The Company plans to continue dosing patients in FAB-201 and to provide an update in the first quarter of 2019.
- Received no objection to clinical trial application (CTA) from
Health Canadafor AVR-RD-02 in Gaucher disease. AVR-RD-02, the next gene therapy in AVROBIO’s pipeline, is being developed as a potential cure for patients with Gaucher disease. The Company continues to anticipate the initiation of the Phase 1/2 clinical trial of AVR-RD-02 in patients with Type 1 Gaucher disease and to begin dosing patients in 2019.
- Continued preparation for the initial use of optimized platform in patients in 2019. Key to AVROBIO’s strategy is to continuously advance its technology and production process to improve the performance of its gene therapies. The company continues its ongoing work to transition to LV2, AVROBIO’s four-plasmid lentiviral vector, to automate a closed manufacturing system, and to optimize the conditioning regimen. In vitro comparability testing of LV2 is in progress, and regulatory filing and potential approval for use in ongoing and future clinical trials is expected in 2019.
- AVR-RD-03 (Pompe disease) and AVR-RD-04 (cystinosis) continue to advance as planned.
AVROBIOcontinues to make progress with AVR-RD-03, a gene therapy candidate being investigated for Pompe disease currently in early preclinical development. AVR-RD-04, a gene therapy candidate being investigated for the treatment of patients with cystinosis, remains on track to enroll the first patient in 2019 in an investigator-sponsored Phase 1/2 study by the University of California, San Diego.
Third Quarter Financial Results
Research and development expenses for the third quarter 2018 were
General and administrative expenses were
Based on its current operating plan,
Various express or implied statements in this release concerning AVROBIO’s future expectations, plans and prospects, including without limitation, its expectations regarding the development and the continued progress of AVROBIO’s programs, including the commencement of clinical trials, expansion of trial sites and timing of data release, the therapeutic potential of its product candidates, AVROBIO’s planned platform optimization efforts and the intended benefits thereof, and statements regarding the Company’s cash position and expected runway, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Any forward-looking statements in this press release are based on management’s current 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 of AVROBIO’s ongoing or planned clinical trials, the risk that
CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share data)
|September 30,||December 31,|
|Cash and cash equivalents||$||138,577||$||5,963|
|Prepaid expenses and other current assets||1,501||345|
|Property and equipment, net||2,306||349|
|Accrued expenses and other current liabilities||3,845||2,098|
|Warrant to purchase redeemable convertible preferred stock||-||35|
|Deferred rent, net of current portion||736||126|
|Other long-term liability||-||500|
|Redeemable convertible preferred stock||-||26,500|
|Total stockholders’ equity (deficit)||137,109||(23,135||)|
|Total liabilities, redeemable convertible preferred stock
and stockholders’ equity (deficit)
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(In thousands, except share and per share data)
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Research and development||$||9,232||$||5,411||$||22,286||$||8,726|
|General and administrative||3,000||959||7,281||2,230|
|Total operating expenses||12,232||6,370||29,567||10,956|
|Loss from operations||(12,232||)||(6,370||)||(29,567||)||(10,956||)|
|Total other income (expense), net||641||(71||)||(773||)||(96||)|
|Net loss per share attributable to common
stockholders — basic and diluted
|Weighted-average number of common shares used in computing net loss per share attributable to common stockholders—basic and diluted||23,747,141||2,263,195||9,945,538||2,216,180|
Katina Dorton AVROBIO, Inc.617-914-8413 firstname.lastname@example.org Christopher F. Brinzey Westwicke Partners339-970-2843 email@example.com Media Contact: Kathryn MorrisThe Yates Network 914-204-6412 firstname.lastname@example.org
Source: AVROBIO, Inc.