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AVROBIO, Inc. Reports Fourth Quarter and Fiscal Year 2018 Financial Results and Provides Business Update
AVROBIO, Inc. Reports Fourth Quarter and Fiscal Year 2018 Financial Results and Provides Business Update
Recruitment completed in investigator-sponsored Phase 1 Fabry study
Phase 1 and Phase 2 clinical data updates for AVR-RD-01 in Fabry disease reported at WORLDSymposium 2019
Clinical trials in Gaucher and cystinosis expected to start in 2019
Introduced plato™ platform for worldwide gene therapy
commercialization, to be incorporated in
“2018 was a very productive year for
Fourth Quarter and Recent Business Highlights
- Clinical progress in Fabry disease. Recruitment was recently
completed for the investigator-sponsored Phase 1 study conducted by
the FACTs* team, with the dosing of the fifth patient in
February 2019 .AVROBIO continues enrollment of FAB-201, its Phase 2 clinical trial of AVR-RD-01 in Fabry disease. Two patients have been dosed to date, and the Company intends to open additional trial sites in the U.S. andCanada in 2019. In addition, inDecember 2018 ,AVROBIO received orphan drug designation for AVR-RD-01 from theU.S. Food and Drug Administration (FDA ). - Preliminary clinical data updates on AVR-RD-01 gene therapy for
Fabry disease at WORLDSymposium 2019. Data presented at
WORLDSymposium and at an
AVROBIO -sponsored investor event supported the potential of AVR-RD-01 as a gene therapy for Fabry disease. A total of seven patients have been treated to date across the Phase 1 FACTs and FAB-201 investigational studies. Four of those patients have at least three months post-gene therapy follow-up. Their data showed α-galactosidase A (AGA) plasma enzyme activity above the range for males with classical Fabry disease at all timepoints measured, including at 22 months post-treatment in Patient 1 in the Phase 1 study. Reductions in substrate and metabolite levels were observed both in patients who discontinued enzyme replacement therapy (ERT) and who were treatment-naïve. This included an 85% reduction in lyso-Gb3 metabolite levels at 6 months in the first patient dosed in the FAB-201 clinical trial. The AVR-RD-01 investigational gene therapy has been generally well tolerated with no serious adverse events (SAEs) related to the study drug, at up to 22- and 6-months follow-up in the Phase 1 and Phase 2 trials, respectively. - On track to enter the clinic in 2019 with two additional gene
therapy programs for lysosomal storage diseases. In
October 2018 ,AVROBIO received a no objection letter (NOL) fromHealth Canada to the clinical trial application (CTA) for GAU-201, its Phase 1/2 clinical trial of AVR-RD-02 in Gaucher disease. The Company subsequently received two additional NOLs fromHealth Canada to CTA amendments that incorporate elements of the plato platform into the trial. Dosing is expected to begin in the second half of 2019. InDecember 2018 , theFDA accepted the Investigational New Drug (IND) application for an investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 in cystinosis. This trial will be led byStephanie Cherqui , PhD, Associate Professor of Pediatrics at theUniversity of California, San Diego , and is expected to begin in the second half of 2019.
- Introduced AVROBIO’s plato platform for worldwide
commercialization.
Plato consists of a 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).Plato was designed to enhance the potency, safety, efficacy, and long-term durability of AVROBIO’s gene therapies, and may additionally provide the capability to address CNS manifestations that accompany many lysosomal storage diseases.Plato also has the potential to overcome historical gene therapy manufacturing bottlenecks, such as scale and capacity. Anticipated manufacturing and other benefits of plato include:- Large scale vector production: The Company expects to have manufacturing capabilities at 200-liter bioreactor scale in the second half of 2019, with vector production capable of treating a substantial number of patients per year;
- Cost-effective manufacturing with global reach: The automated, closed cell manufacturing system is intended to improve quality and consistency between batches. In addition, its portability may allow for global production using lower-level clean rooms that are cost-effective and in ample supply around the world. We believe this is the first use of this approach in CD34+ gene therapy;
- Convenience for patients: The gene therapy product is cryopreserved to simplify logistics and allow convenient scheduling for patients.
The Company intends to start using plato in its FAB-201 and GAU-201 clinical trials in 2019.
- Expanded and strengthened leadership team with four senior
management hires. In
December 2018 ,AVROBIO strengthened its senior management team with the appointments of four senior management hires:Birgitte Volck , MD, PhD, as President of Research and Development to oversee medical, clinical, regulatory, pre-clinical, research and manufacturing;Erik Ostrowski as Chief Financial Officer;Steven Avruch as General Counsel; andJosie Yang , PhD as head of Regulatory Affairs. These new leaders augment the senior management team and help prepare the Company for its next stage of growth.
Fourth Quarter and Fiscal Year 2018 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, the intended incentives conferred by orphan drug designation, potential regulatory approvals and the timing thereof, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, 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 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
* FACTs = Fabry disease Clinical research and Therapeutics in Canada |
CONDENSED CONSOLIDATED BALANCE SHEETS |
||||||||||
December 31, | December 31, | |||||||||
2018 | 2017 | |||||||||
Cash and cash equivalents | $ | 126,302 | $ | 5,963 | ||||||
Prepaid expenses and other current assets | 3,718 | 345 | ||||||||
Property and equipment, net | 2,634 | 349 | ||||||||
Other assets | 825 | 365 | ||||||||
Total assets | $ | 133,479 | $ | 7,022 | ||||||
Accounts payable | $ | 2,784 | $ | 527 | ||||||
Accrued expenses and other current liabilities | 7,822 | 2,098 | ||||||||
Warrant to purchase redeemable convertible preferred stock | - | 35 | ||||||||
Derivative liability | - | 371 | ||||||||
Deferred rent, net of current portion | 689 | 126 | ||||||||
Other long-term liability | - | 500 | ||||||||
Total liabilities | 11,295 | 3,657 | ||||||||
Redeemable convertible preferred stock | - | 26,500 | ||||||||
Total stockholders’ equity (deficit) | 122,184 | (23,135 | ) | |||||||
Total liabilities, redeemable convertible preferred stock
and stockholders’ equity (deficit) |
$ | 133,479 | $ | 7,022 | ||||||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
||||||||||||||||||||
Three Months Ended December 31, | Twelve Months Ended December 31, | |||||||||||||||||||
2018 | 2017 | 2018 | 2017 | |||||||||||||||||
Operating expenses: | ||||||||||||||||||||
Research and development | $ | 12,809 | $ | 6,465 | $ | 35,095 | $ | 15,191 | ||||||||||||
General and administrative | 3,867 | 965 | 11,148 | 3,195 | ||||||||||||||||
Total operating expenses | 16,676 | 7,430 | 46,243 | 18,386 | ||||||||||||||||
Loss from operations | (16,676 | ) | (7,430 | ) | (46,243 | ) | (18,386 | ) | ||||||||||||
Total other income (expense), net | 655 | (166 | ) | (118 | ) | (262 | ) | |||||||||||||
Net loss | $ | (16,021 | ) | $ | (7,596 | ) | $ | (46,361 | ) | $ | (18,648 | ) | ||||||||
Reconciliation of net loss to net loss attributed to common
stockholders:
Net loss |
$ | (16,021 | ) | $ | (7,596 | ) | $ | (46,361 | ) | $ | (18,648 | ) | ||||||||
Accretion of issuance costs on convertible preferred stock | 37 | (2,243 | ) | (85 | ) | |||||||||||||||
Net loss attributable to common stockholders – basic and diluted | $ | (16,021 | ) | $ | (7,559 | ) | $ | (48,604 | ) | $ | (18,733 | ) | ||||||||
Net loss per share attributable to common stockholders — basic and diluted | $ | (0.67 | ) | $ | (3.29 | ) | $ | (3.62 | ) | $ | (8.38 | ) | ||||||||
Weighted-average number of common shares used in computing net loss per share attributable to common stockholders—basic and diluted | 23,791,495 | 2,294,280 | 13,435,478 | 2,235,865 | ||||||||||||||||
View source version on businesswire.com: https://www.businesswire.com/news/home/20190325005290/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