AVROBIO, Inc. Reports First Quarter 2019 Financial Results and Provides Business Update
Regulatory clearances achieved that enable integration of the plato™ platform into Fabry FAB-201 and Gaucher GAU-201 clinical trials in the second half of 2019
IND for AVR-RD-01 for the treatment of Fabry disease cleared by the
Next interim Fabry clinical data update anticipated during summer 2019
Investigator-sponsored cystinosis clinical trial on track to start in the second half of 2019
“2019 is off to a strong start as we advance our pipeline of gene
therapies for lysosomal storage diseases on multiple fronts. We expect
to exit this year with three gene therapy programs in clinical trials –
for Fabry, Gaucher and cystinosis,” commented
Business Update and Program Milestones
- AVROBIO’s plato platform: plato is the Company’s commercial-scale platform for anticipated future worldwide commercialization and pipeline expansion activities. Three important upgrades are expected to be incorporated in 2019 – a proprietary state-of-the-art four-plasmid vector system, automation of a closed cell manufacturing process and a conditioning regimen that utilizes therapeutic drug monitoring (TDM). The plato platform is designed to enhance the potential potency, safety, efficacy, and long-term durability of AVROBIO’s gene therapies, and may additionally provide the capability to address central nervous system manifestations that accompany many lysosomal storage diseases.
- Fabry disease: AVR-RD-01 is AVROBIO’s investigational gene
therapy candidate for the treatment of Fabry
disease. During the quarter, the Company:
- Completed enrollment in the investigator-sponsored FACTs1 Phase 1 study, and continued recruitment in AVROBIO’s FAB-2012 Phase 2 trial. A total of seven patients have now been dosed across these two studies.
Achieved U.S. Food and Drug Administration( FDA) clearance of AVROBIO’s Investigational New Drug (IND) application for AVR-RD-01. This allows AVROBIOto move forward on two key program initiatives anticipated for the second half of 2019: the incorporation of our plato platform into the FAB-201 Phase 2 trial and the opening of our first clinical site in the U.S.
Presented clinical data in
February 2019that continued to support the potential of AVR-RD-01 as a gene therapy for Fabry disease. All patients reporting data exhibited elevated AGA enzyme activity, with the first patient in the Phase 1 study exhibiting elevated AGA enzyme activity at 22 months after being treated with AVR-RD-01. An impact of gene therapy on substrate and metabolite levels was observed both in patients who have discontinued enzyme replacement therapy (ERT) as well as in treatment-naïve patients. Lyso-Gb3 levels of the first patient in the Phase 1 study were lower at 22 months while on AVR-RD-01 alone than while on ERT alone. An 85 percent reduction in lyso-Gb3 activity was observed for the first patient in the Phase 2 clinical trial, who is ERT-naïve, at the six-month timepoint.
- Gaucher disease: AVR-RD-02 is AVROBIO’s investigational gene
therapy candidate for the treatment of Gaucher
disease. The Company received clearance for its clinical trial
application (CTA) utilizing the plato platform from
Health Canadaand plans to initiate its GAU-201 Phase 1/2 clinical trial in the second half of 2019. We expect the trial to enroll 8 to 16 patients with Type 1 Gaucher disease and to include both treatment-naïve patients as well as patients stable on ERT.
- Cystinosis: AVR-RD-04 is AVROBIO’s investigational gene therapy
candidate for the treatment of patients with cystinosis.
FDAcleared the IND application for the investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04. The trial, which is designed to enroll up to 6 patients who are currently being treated with cysteamine, will be conducted at the University of California, San Diego(UCSD) and is expected to start in the second half of 2019. AVROBIOhighlighted its manufacturing expertise and preclinical data at the 2019 ASGCT Annual Meeting. AVROBIO’s senior management and academic collaborators made presentations at the American Society of Cell and Gene Therapy(ASGCT), including: Kim Warren, Ph.D., Head of Operations for AVROBIO, who presented on the potential of AVROBIO’s plato platform to address gene therapy manufacturing and commercialization challenges.
- Azadeh Golipour, Ph.D., AVROBIO’s Senior Director of Manufacturing Operations, who presented on advances in the development of plato’s automated gene modified cell production process, from concept to process verification.
Stefan Karlsson, MD, Ph.D., Professor in the Division of Molecular Medicine and Gene Therapyat Lund Universityin Sweden, who presented preclinical data from the Company’s programs in Gaucher disease. Stephanie Cherqui, Ph.D. and her UCSD team members, who gave several oral presentations on preclinical cystinosis data.
First Quarter 2019 Financial Results
Research and development expenses were
General and administrative expenses were
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, anticipated benefits of our gene therapy platform including potential impact on our commercialization and pipeline expansion activities, 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, timing and likelihood of success, 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 the date of this release 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
1 FACTs = Fabry disease Clinical research and Therapeutics in
2 The official name of the ‘FAB-201 Study’ is AVRO-RD-01-201, which is a Phase 2 trial of AVROBIO’s investigational gene therapy, AVR-RD-01, in Fabry disease. FAB-201 is designed to evaluate the safety and efficacy of AVR-RD-01 in 8 to 12 treatment- naïve male Fabry patients.
CONDENSED CONSOLIDATED BALANCE SHEETS
|March 31,||December 31,|
|Cash and cash equivalents||$||108,476||$||126,302|
|Prepaid expenses and other current assets||5,203||3,718|
|Property and equipment, net||2,864||2,634|
|Accrued expenses and other current liabilities||6,477||7,822|
|Deferred rent, net of current portion||645||689|
|Total stockholders’ equity||106,788||122,184|
|Total liabilities and stockholders’ equity||$||117,368||$||133,479|
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
|Three Months Ended March 31,|
|Research and development||$||12,446||$||5,647|
|General and administrative||5,254||2,141|
|Total operating expenses||17,700||7,788|
|Loss from operations||(17,700)||(7,788)|
|Total other income (expense), net||597||(454)|
Reconciliation of net loss to net loss attributed to common stockholders:
Accretion of issuance costs on convertible preferred stock
Net loss attributable to common stockholders – basic and diluted
|Net loss per share attributable to common stockholders — basic and diluted||$||(0.72)||$||(4.51)|
|Weighted-average number of common shares used in computing net loss per share attributable to common stockholders—basic and diluted||23,893,696||2,324,790|
Christopher F. Brinzey
Westwicke, an ICR Company
The Yates Network