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 AVROBIO-sponsored clinical
trials in 2019
CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO,
Inc. (NASDAQ: AVRO) (the “Company”), a Phase 2 clinical-stage gene
therapy company, today reported financial results for the fourth quarter
and fiscal year ended December 31, 2018 and provided a business update.
“2018 was a very productive year for AVROBIO, as we built a solid
financial foundation with our IPO and made significant progress
advancing our pipeline of gene therapies for Fabry and other lysosomal
storage diseases,” commented Geoff
MacKay, President and Chief Executive Officer of AVROBIO. “We enter
2019 with strong momentum. Our recently-presented preliminary clinical
data from the Fabry program builds upon a growing body of evidence for
the therapeutic potential of AVR-RD-01 as a gene therapy for patients
with this debilitating disease. In addition, we look forward to
expanding AVROBIO’s clinical activities, with trials for Gaucher disease
and cystinosis expected to start in 2019. Finally, after three and a
half years of development, we were excited to introduce the plato™
platform, which includes a proprietary vector system, cell manufacturing
solution and conditioning regimen with therapeutic drug monitoring. We
believe that plato provides the foundation for the potential worldwide
commercialization of our gene therapies. We plan to incorporate plato
into our Fabry and Gaucher clinical trials in the second half of 2019.”
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. and Canada in 2019. In addition, in December 2018, AVROBIO
received orphan drug designation for AVR-RD-01 from the U.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) from Health 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 from Health 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. In December 2018, the
FDA 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 by Stephanie Cherqui, PhD,
Associate Professor of Pediatrics at the University 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. -
Large scale vector production: The Company expects to have
-
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; and Josie 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
AVROBIO reported a net loss of $16.0 million for the fourth quarter of
2018, and a net loss of $46.4 million for the year ended December 31,
2018, as compared to a net loss of $7.6 million and a net loss of $18.6
million for the comparable periods in 2017, respectively. These
increases were due to increased research and development expenses, as
well as increased general and administrative expenses.
Research and development expenses were $12.8 million for the fourth
quarter of 2018, and $35.1 million for the year ended December 31, 2018,
as compared to $6.5 million and $15.2 million for the comparable periods
in 2017, respectively. These increases were driven by increased
preclinical and clinical development activities related to the
advancement of the Company’s pipeline, as well as increased
personnel-related costs resulting from an increase in employee headcount.
General and administrative expenses were $3.9 million for the fourth
quarter of 2018, and $11.1 million for the year ended December 31, 2018,
as compared to $1.0 million and $3.2 million for the comparable periods
in 2017, respectively. These increases were primarily due to an increase
in employee headcount, consulting and professional fees related to the
support of ongoing business operations as a publicly traded company, and
the impact of stock-based compensation.
As of December 31, 2018, AVROBIO had $126.3 million in cash and cash
equivalents, as compared to $6.0 million in cash and cash equivalents as
of December 31, 2017. This increase was primarily the result of the
completion of the Company’s initial public offering completed in June
2018, which raised net proceeds of $104.0 million, and the Company’s
Series B financing completed in January 2018, which generated net
proceeds of $58.3 million. Based on the Company’s current operating
plan, AVROBIO expects its cash and cash equivalents as of December 31,
2018 will enable the Company to fund its operating expenses and capital
expenditure requirements into the second half of 2020.
About AVROBIO, Inc.
AVROBIO,
Inc., is a Phase 2 clinical-stage gene therapy company developing
gene therapies to potentially cure rare diseases with a single dose.
AVROBIO’s lentiviral-based
gene therapies employ hematopoietic stem cells that are collected
from the patient and then modified with a lentiviral vector to insert
functional copies of the gene that is defective in the target disease.
AVROBIO is focused on the development of its gene therapy, AVR RD 01, in Fabry
disease, as well as additional gene therapy programs in other lysosomal
storage disorders including Gaucher
disease, cystinosis
and Pompe
disease. The Company’s plato platform is a proprietary vector system
and automated, closed cell manufacturing solution designed to support
worldwide commercialization. AVROBIO is headquartered in Cambridge, MA
and has offices in Toronto, ON. For additional information, visit www.avrobio.com.
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 AVROBIO or our
collaborators, the risk that AVROBIO may not realize the intended
benefits of our gene therapy platform, including the features of our
plato platform, the risk that our product candidates or procedures in
connection with the administration thereof will not have the safety or
efficacy profile that we anticipate, the risk that prior results, such
as signals of safety, activity or durability of effect, observed from
preclinical or clinical trials, will not be replicated or will not
continue in ongoing or future studies or trials involving AVROBIO’s
product candidates, the risk that we will be unable to obtain and
maintain regulatory approval for our product candidates, the risk that
the size and growth potential of the market for our product candidates
will not materialize as expected, risks associated with our dependence
on third-party suppliers and manufacturers, risks regarding the accuracy
of our estimates of expenses and future revenue, risks relating to our
capital requirements and needs for additional financing, and risks
relating to our ability to obtain and maintain intellectual property
protection for our product candidates. For a discussion of these and
other risks and uncertainties, and other important factors, any of which
could cause AVROBIO’s actual results to differ materially and adversely
from those contained in the forward-looking statements, see the section
entitled “Risk Factors” in AVROBIO’s Quarterly Report on Form 10-Q for
the quarter ended September 30, 2018, as well as discussions of
potential risks, uncertainties and other important factors in AVROBIO’s
subsequent filings with the Securities and Exchange Commission. AVROBIO
explicitly disclaims any obligation to update any forward-looking
statements except to the extent required by law.
| * 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 | ||||||||||||||||
Contacts
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