RICHMOND, Calif., Oct. 26, 2016 /PRNewswire/ -- Sangamo
BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome
editing, today reported its third quarter 2016 financial results
and provided an update on recent events and development
timelines for its therapeutic programs.
"The third quarter of 2016 has been a pivotal time for Sangamo,
as we worked to focus our efforts and execute on our prioritized
therapeutic programs in hemophilia B, hemophilia A, MPS I and MPS
II," said Sandy Macrae, M.B., Ch.B.,
Ph.D., Sangamo's president and chief executive officer. "I am
pleased to announce that the Phase 1/2 clinical trial for SB-FIX,
our in vivo genome editing program for hemophilia B, is
open. We are also on track to file an IND application for our AAV
cDNA Factor 8 gene therapy program for hemophilia A by the end of
this year. In addition, we submitted the additional data package
for our MPS I and MPS II programs to the FDA in September, and I am
pleased to report that the FDA has cleared these programs for
clinical development. Preparations are now underway to initiate
Phase 1/2 clinical trials for these indications in early 2017."
Dr. Macrae continued, "We also made a number of organizational
changes, including several key hires in our clinical and technical
operations teams and instituted new procedures in order to position
the company for clinical success. I am very encouraged by the
commitment of our entire team and the progress we have made in the
third quarter to drive these activities forward. I remain confident
that we can demonstrate the value and therapeutic potential of our
genome editing and gene therapy platforms and with reliable steps,
make sensible progress and realize our vision of transforming
Sangamo into a patient-focused therapeutics company."
Recent Highlights
- Initiation of FIXtendz (SB-FIX-1501) Phase 1/2 clinical
trial designed to assess safety, tolerability and potential
efficacy of SB-FIX in adults with hemophilia B. In October,
Sangamo opened the first clinical study of an in vivo genome
editing therapeutic, its Phase 1/2 clinical trial (FIXtendz,
SB-FIX-1501). SB-FIX-1501 is an open-label, dose-escalation study
in male subjects over eighteen years of age, with severe hemophilia
B, who do not have inhibitors or hypersensitivity to recombinant
Factor IX protein (rFIX). The study will enroll up to nine subjects
in three dosing cohorts of two subjects per cohort, with additional
subjects to be enrolled at the optimal therapeutic dose, and will
evaluate the safety and potential efficacy of a single
administration of SB-FIX.
- U.S. Food and Drug Administration (FDA) grants orphan drug
designation to SB-FIX, the first in vivo genome editing
therapeutic in development. In September, Sangamo announced
that the FDA granted Orphan Drug Designation (ODD) to SB-FIX, the
company's zinc finger nuclease (ZFN)-mediated in vivo genome
editing therapeutic candidate for hemophilia B. Orphan drug
designation is granted to investigational drugs and biologics that
are intended to treat rare diseases that affect fewer than 200,000
people in the U.S. This designation helps facilitate drug
development by providing several benefits to drug developers,
including assistance with clinical study design and drug
development, tax credits for qualified clinical trial costs,
exemption from certain FDA application fees and seven years of
market exclusivity upon regulatory product approval.
- FDA clearance to initiate Phase 1/2 clinical trials for
SB-318 (MPS I) and SB-913 (MPS II) therapeutic programs.
Sangamo submitted the additional in vitro studies requested
by the FDA in September and recently received clearance to initiate
Phase 1/2 clinical trials for the Mucopolysaccharidosis Type I (MPS
I, Hurler syndrome) and Mucopolysaccharidosis Type II (MPS II,
Hunter syndrome) programs based on its ZFN-mediated in vivo
genome editing therapeutic platform. The company expects to
initiate the clinical studies in early 2017.
- Appointment of new head of technical operations. In
August, Sangamo appointed Mohammad
El-Kalay, Ph.D., as Vice President, Technical Operations.
Dr. El-Kalay brings over 25 years of operational management
experience in the life sciences field, including expertise in
process development and cGMP manufacturing operations at clinical
scale with hematopoietic stem cells, T-cells and various other cell
types. Dr. El-Kalay is responsible for process development and
manufacturing of all biotherapeutics for Sangamo.
Third Quarter 2016 Results
For the third quarter ended
September 30, 2016, Sangamo reported
a consolidated net loss of $19.0
million, or $0.27 per share,
compared to a net loss of $9.2
million, or $0.13 per share,
for the same period in 2015. As of September
30, 2016, the Company had cash, cash equivalents, marketable
securities and interest receivable of $155.4
million.
Revenues for the third quarter of 2016 were $2.8 million, compared to $8.6 million for the same period in 2015. Third
quarter 2016 revenues were generated from the Company's
collaboration agreements with Biogen and Shire International GmbH
(Shire), enabling technology agreements and research grants. The
revenues recognized for the third quarter of 2016 consisted of
$2.7 million from collaboration
agreements and $0.1 million from
research grants, compared to $8.4
million and $0.2 million,
respectively, for the same period in 2015. The decrease in
collaboration agreement revenues was a result of an amendment to
the Company's collaboration and license agreement with Shire in the
third quarter of 2015, which returned the rights to the hemophilia
programs to Sangamo, as well as a decrease in revenues from the
Biogen agreement as the initial research phase of these programs
has matured and activities during this quarter were largely
internal.
In the third quarter of 2016, Sangamo recognized $1.2 million of revenues related to research
services performed under the collaboration agreement with Biogen,
and $0.2 million of revenues related
to research services performed under the collaboration agreement
with Shire. In addition, Sangamo received upfront payments of
$13.0 million and $20.0 million pursuant to the agreements entered
into with Shire in 2012 and Biogen in 2014, respectively. The Shire
payment is being recognized as revenue on a straight-line basis
over the initial six-year research term. Beginning in January 2016, the Biogen payment is being
recognized over approximately 42 months which reflects the revised
service period related to Sangamo's deliverables under the Biogen
agreement. The Company recognized $0.5
million of the Shire upfront payment and $0.6 million of the Biogen upfront payment as
revenue for the third quarter of 2016.
Research and development expenses were $17.0 million for the third quarter of 2016,
compared to $16.7 million for the
same period in 2015. General and administrative expenses were
$5.0 million for the third quarter of
2016, compared to $4.6 million for
the same period in 2015.
Total operating expenses for the third quarter of 2016 were
$22.0 million, compared to
$21.3 million for the same period in
2015.
Nine Months Results
For the nine months ended
September 30, 2016, the consolidated
net loss was $62.0 million, or
$0.88 per share, compared to a
consolidated net loss of $26.7
million, or $0.38 per share,
for the nine months ended September 30,
2015. Revenues were $10.5
million for the nine months ended September 30, 2016, compared to $30.4 million for the same period in 2015. The
decrease in revenues was primarily related to the amendment of our
collaboration and license agreement with Shire, as well as a
decrease in revenues related to our agreements with Sigma and
Biogen. Total operating expenses were $73.2
million for the nine months ended September 30, 2016, compared to $61.6 million for the same period in 2015 and
reflect increased expenses related to salaries and benefits,
including stock-based compensation expense, as well as professional
fees, consulting services and other corporate costs.
Financial Guidance for 2016
The Company reiterates
guidance as follows:
- Cash and Investments: Sangamo expects that its cash,
cash equivalents and marketable securities will be at least
$140 million at the end of 2016,
inclusive of research funding from existing collaborators but
exclusive of funds arising from any additional new collaborations
or partnerships, equity financings or other new sources.
- Revenues: Sangamo expects that revenues will be in the
range of $12 million to $17 million
in 2016, inclusive of research funding from existing
collaborations.
- Operating Expenses: Sangamo expects that operating
expenses will be in the range of $85 million
to $95 million for 2016.
Conference Call
Sangamo will host a conference call
today, October 26, 2016, at
5:00 p.m. ET, which will be open to
the public. The call will be webcast live and can be accessed via a
link on the Sangamo BioSciences website in the Investor Relations
section under Events and Presentations. A replay of the webcast
will be available for two weeks after the call. During the
conference call, the Company will review these results, discuss
other business matters and provide guidance with respect to the
remainder of 2016.
The conference call dial-in numbers are (877) 377-7553 for
domestic callers and (678) 894-3968 for international callers. The
conference ID number for the call is 96289087. A conference
call replay will be available for one week following the conference
call, from approximately 8:00 p.m. ET
on October 26, 2016 to 11:59 p.m. ET on November
2, 2016. The conference call replay numbers for domestic and
international callers are (855) 859-2056 and (404) 537-3406,
respectively. The conference ID number for the replay is
96289087.
About Sangamo
Sangamo BioSciences, Inc. is focused on
Engineering Genetic Cures® for monogenic and infectious
diseases by deploying its novel zinc finger DNA-binding protein
technology, in therapeutic genome editing and gene regulation, and
AAV-based gene therapy platforms. The Company's proprietary
ZFN-mediated in vivo genome editing approach is focused on
monogenic diseases, including hemophilia and lysosomal storage
disorders, including MPS I and MPS II. Sangamo has initiated a
Phase 1/2 clinical trial for hemophilia B, the first in vivo
genome editing application cleared by the FDA. In addition, Sangamo
has Phase 1/2 and Phase 2 clinical programs in HIV/AIDS (SB-728).
The Company has also formed a strategic collaboration with Biogen
Inc. for hemoglobinopathies, including sickle cell disease and
beta-thalassemia, and with Shire International GmbH to develop
therapeutics for Huntington's disease. It has established strategic
partnerships with companies in non-therapeutic applications of its
technology, including Dow AgroSciences and Sigma-Aldrich
Corporation. For more information about Sangamo, visit the
Company's website at www.sangamo.com.
ZFP Therapeutic® is a registered trademark of
Sangamo BioSciences, Inc.
This press release contains forward-looking statements
regarding Sangamo's current expectations. These forward looking
statements include, without limitation, references to expected
timing of initiating clinical trials, presentation of clinical
trial data and submission of INDs, anticipated cash and investment
balance, operating expenses, revenue and achievement of milestone
and royalty payments under Sangamo's collaboration agreements,
changes in internal organization, the research and development of
ZFNs and ZFP TFs, and the focus on clinical trials and therapeutic
applications of Sangamo's gene therapy and ZFP technology
platforms. These statements are not guarantees of future
performance and success and are subject to certain risks,
uncertainties and assumptions that are difficult to predict.
Factors that could cause actual results to differ include, but are
not limited to, the early stage of ZFP Therapeutic development, the
lengthy and uncertain regulatory approval process, uncertainties
related to the timing of initiation and completion of clinical
trials, whether clinical trial results will validate and support
the safety and efficacy of ZFP Therapeutics, and the ability to
maintain and establish strategic partnerships. Further, there
can be no assurance that the necessary regulatory approvals will be
obtained or that Sangamo and its partners will be able to develop
commercially viable gene-based therapeutics. Actual results may
differ from those projected in forward-looking statements due to
risks and uncertainties that exist in Sangamo's operations and
business environments. These risks and uncertainties are described
more fully in Sangamo's Annual Reports on Form 10-K and Quarterly
Reports on Form 10-Q as filed with the Securities and Exchange
Commission. Forward-looking statements contained in this
announcement are made as of this date, and Sangamo undertakes no
duty to update such information except as required under applicable
law.
SELECTED
CONSOLIDATED FINANCIAL DATA
|
(unaudited; in
thousands, except per share data)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended
|
|
Nine Months
Ended
|
|
September
30,
|
|
September
30,
|
|
2016
|
|
2015
|
|
2016
|
|
2015
|
Statement of
Operations Data:
|
|
|
|
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
Collaboration
agreements
|
$
2,728
|
|
$
8,406
|
|
$
10,031
|
|
$
28,878
|
Research
grants
|
95
|
|
163
|
|
436
|
|
1,540
|
Total
revenues
|
2,823
|
|
8,569
|
|
10,467
|
|
30,418
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
17,008
|
|
16,694
|
|
51,728
|
|
47,292
|
General and
administrative
|
5,021
|
|
4,560
|
|
21,468
|
|
14,309
|
Total operating
expenses
|
22,029
|
|
21,254
|
|
73,196
|
|
61,601
|
Loss from
operations
|
(19,206)
|
|
(12,685)
|
|
(62,729)
|
|
(31,183)
|
Interest and other
income, net
|
238
|
|
101
|
|
668
|
|
406
|
Loss before
taxes
|
(18,968)
|
|
(12,584)
|
|
(62,061)
|
|
(30,777)
|
Benefit from income
taxes
|
3
|
|
3,339
|
|
27
|
|
4,087
|
Net loss
|
$
(18,965)
|
|
$
(9,245)
|
|
$(62,034)
|
|
$(26,690)
|
Basic and diluted net
loss per common share
|
$
(0.27)
|
|
$
(0.13)
|
|
$
(0.88)
|
|
$
(0.38)
|
|
|
|
|
|
|
|
|
Shares used in
computing basic and diluted net loss per common share
|
70,618
|
|
69,892
|
|
70,493
|
|
69,622
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
SELECTED BALANCE
SHEET DATA
|
|
|
|
|
|
|
|
|
September 30,
2016
|
|
December 31,
2015
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents, marketable securities and interest
receivable
|
$
155,398
|
|
$
209,307
|
|
|
|
|
Total
assets
|
164,978
|
|
217,235
|
|
|
|
|
Total stockholders'
equity
|
143,836
|
|
192,439
|
|
|
|
|
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SOURCE Sangamo BioSciences, Inc.