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
FDA; AVROBIO plans to open U.S. clinical trial sites for its ongoing
multi-country FAB-201 trial

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

(NASDAQ: AVRO) (the “Company”), a Phase 2 clinical-stage gene
therapy company, today reported financial results for the first quarter
ended March 31, 2019 and provided a business update.

“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 Geoff
, President and Chief Executive Officer of AVROBIO.
“Underpinning this progress is plato™, AVROBIO’s proprietary platform
for developing, manufacturing, and commercializing our gene therapy
programs globally, which we plan to utilize initially in our
Company-sponsored clinical trials in Fabry and Gaucher. Our highest
priority in early 2019 was to secure regulatory clearances that enable
the use of plato in multiple trials in multiple countries, namely in
Australia, Canada and the U.S. We believe that the plato platform, which
is designed to enable the treatment of thousands of patients, if our
gene therapies are approved, represents a significant advance toward
commercial readiness.”

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
    . 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 AVROBIO to 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 2019 that 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
    . The Company received clearance for its clinical trial
    application (CTA) utilizing the plato platform from Health Canada and
    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.
    The FDA cleared 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.
  • AVROBIO highlighted 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 Therapy at Lund University in Sweden, who
      presented preclinical data from the Company’s programs in Gaucher
    • Stephanie Cherqui, Ph.D. and her UCSD team members, who gave
      several oral presentations on preclinical cystinosis data.

First Quarter 2019 Financial Results

AVROBIO reported a net loss of $17.1 million for the first quarter of
2019 as compared to a net loss of $8.2 million for the comparable period
in 2018. This increase was due to increased research and development
expenses, as well as increased general and administrative expenses.

Research and development expenses were $12.4 million for the first
quarter of 2019 as compared to $5.6 million for the comparable period in
2018. This increase was driven by increased preclinical, clinical
development and manufacturing activities related to the advancement of
the Company’s pipeline, as well as increased personnel-related costs
including non-cash stock-based compensation expense resulting from an
increase in employee headcount.

General and administrative expenses were $5.3 million for the first
quarter of 2019 as compared to $2.1 million for the comparable period in
2018. This increase was primarily due to an increase in employee
headcount, legal, consulting and professional fees related to the
support of ongoing business operations as a publicly traded company, as
well as the impact of non-cash stock-based compensation.

As of March 31, 2019, AVROBIO had $108.5 million in cash and cash
equivalents, as compared to $126.3 million in cash and cash equivalents
as of December 31, 2018. Based on the Company’s current operating plan,
AVROBIO expects its cash and cash equivalents as of March 31, 2019 will
enable the Company to fund its operating expenses and capital
expenditure requirements into the second half of 2020.

About AVROBIO, Inc.

is a Phase 2 clinical-stage gene therapy company. AVROBIO is
focused on the development of its gene therapy candidate, AVR-RD-01, in Fabry
, as well as additional gene therapy programs in other
lysosomal storage disorders including Gaucher
, cystinosis
and Pompe
. The Company’s plato™ platform includes a proprietary vector
system, automated cell manufacturing solution and refined conditioning
regimen with therapeutic drug monitoring. 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,
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
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 Annual Report on
Form 10-K for the fiscal year ended December 31, 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.

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


(In thousands)

March 31, December 31,
2019 2018
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
Other assets   825   825
Total assets $ 117,368 $ 133,479
Accounts payable $ 3,458 $ 2,784
Accrued expenses and other current liabilities 6,477 7,822
Deferred rent, net of current portion   645   689
Total liabilities 10,580 11,295
Total stockholders’ equity   106,788   122,184
Total liabilities and stockholders’ equity $ 117,368 $ 133,479

thousands, except share and per share data)


Three Months Ended March 31,
2019   2018
Operating expenses:
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)
Net loss $ (17,103) $ (8,242)

Reconciliation of net loss to net loss attributed to common



Net loss





Accretion of issuance costs on convertible preferred stock


Net loss attributable to common stockholders – basic and diluted

$ (17,103) $ (10,485)
Net loss per share attributable to common stockholders — basic and
$ (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


Investor Contact:
Christopher F. Brinzey
Westwicke, an
ICR Company

Media Contact:
Kathryn Morris
The Yates Network

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