RICHMOND, Calif., Aug. 3, 2016 /PRNewswire/ -- Sangamo
BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome
editing, today reported its second quarter 2016 financial results
and updated on recent events and development timelines for its
preclinical and clinical therapeutic programs.
"My mandate as CEO of Sangamo is to lead the Company in its
transition from a research-focused organization to a clinical-stage
product development company capable of building a versatile
portfolio of therapeutics based on both our proprietary zinc finger
nuclease (ZFN) and gene therapy platforms, and I am excited to
be part of that evolution," said Sandy
Macrae, M.B., Ch.B., Ph.D., Sangamo's president and chief
executive officer.
Dr. Macrae continued, "In my first few months I have been
reviewing programs and working with the team to understand how
best to use our resources to position the Company for success. As
part of this process, we are re-prioritizing some of our efforts.
In 2016, our intention remains to file an investigational new drug
(IND) application for our hemophilia A program which uses an
adeno-associated virus (AAV) cDNA gene therapy approach and to
begin a Phase 1/2 clinical trial of our in vivo genome
editing program in hemophilia B. However, the initiation of
our Phase 1/2 trials in MPS I and MPS II is delayed until 2017 and
we expect that the timing of milestones related to some of our
other programs will change as we refocus our efforts.
"I am confident that this review and future focusing of our
efforts will be beneficial to the efficiency and performance of the
organization in the long run. I am pleased to be leading Sangamo to
a new stage in its development and excited to be a part of building
a world class organization capable of developing innovative
medicines for patients and creating value for its
shareholders."
Summary of Therapeutic Development Program
Updates
Sangamo has eight programs in therapeutic
development in addition to ongoing clinical trials to evaluate
its ZFN-mediated genome editing approach for HIV/AIDS in T-cells as
well as hematopoietic stem and progenitor cells (HSPCs). As part of
its review and evaluation process the Company is prioritizing
efforts to enable development of its therapeutics and
execution of it trials to the highest standards.
The current expected status of Sangamo's programs is summarized
below. Additional detail will be provided during this afternoon's
call.
- Sangamo expects to file an investigational new drug (IND)
application for its AAV cDNA human Factor 8 (hF8) gene therapy
program for the treatment of hemophilia A and to initiate the Phase
1/2 clinical trial of its in vivo genome editing approach
for hemophilia B in 2016.
- The Company's proposed Phase 1/2 clinical trials for both
Mucopolysaccharidosis Type I (MPS I) and MPS II are expected to
begin in 2017 after Sangamo completes certain in vitro
preclinical studies and discussions with the U.S. Food and Drug
Administration (FDA).
- IND applications for Sangamo's collaborative programs with
Biogen in beta-thalassemia and sickle cell disease are anticipated
to be filed in 2017 after some additional preclinical work to
optimize the programs. Sangamo will provide more information as it
becomes available.
- Sangamo is evaluating the preclinical development plans for its
in vivo genome editing approach for Fabry disease and
Gaucher disease to inform the optimal path forward to IND
application.
- The Company expects to present data in 2017 from its ongoing
Phase 2 clinical trial in T-cells (SB-728-1101 Cohort 3*) and
investigator-sponsored Phase 1/2 clinical study (SB-728mR-HSPC) in
HSPCs, both of which are designed to evaluate Sangamo's
ZFN-mediated genome-editing approach for HIV/AIDS.
Recent Events
- Appointment of Sandy Macrae
as new president and CEO. In June, Sangamo announced the
appointment of Alexander 'Sandy' Macrae, M.B., Ch.B., Ph.D., MRCP,
as president and chief executive officer to succeed founder and
former president and CEO, Edward
Lanphier. Dr. Macrae, a physician scientist, joins Sangamo
with deep experience in clinical development and global business
strategy, most recently serving as global medical officer of Takeda
Pharmaceuticals. Mr. Lanphier stepped down from his chief executive
leadership role and, following the Company's 2016 annual meeting of
stockholders on June 14, 2016,
assumed chairmanship of Sangamo's Board of Directors.
- Announcement of new gene therapy clinical development
program for the treatment of hemophilia A. Following the
presentation of preclinical data at the World Federation of
Hemophilia 2016 World Congress demonstrating supraphysiological
levels of human factor VIII (hFVIII) expression from Sangamo's new
proprietary AAV cDNA therapeutic, the Company announced that it is
advancing the clinical development of this AAV cDNA approach
(SB-525) for the treatment of hemophilia A. Sangamo also
announced the goal of filing an IND application with the FDA in
2016 and, pending FDA clearance, initiating a Phase 1/2 clinical
trial in adults with hemophilia A.
- Presentation of new data and recent developments from
multiple research and ZFP Therapeutic® programs at 2016
Annual Meeting of the American Society of Gene & Cell Therapy
(ASGCT). At the 2016 ASGCT Annual Meeting, Sangamo presented
new preclinical data from its proprietary ZFN-mediated in
vivo genome editing approach for MPS I, providing histologic
evidence of clearance of toxic glycosaminoglycans (GAGs) in various
tissues after a single systemic treatment of the Company's
therapeutic candidate. Data were also presented from Sangamo
scientists and their collaborators highlighting technological
advances in process development and therapeutic applications.
Specifically:
- Data demonstrating the capabilities of ZFNs as tools in the
development of allogeneic T-cell therapies, including simultaneous
high efficiency biallelic knock out of the T-cell receptor (TCR)
and HLA Class I protein as well as highly efficient targeted
integration into these sites.
- Improved multipotency and long-term engraftment of ZFN-modified
HSPCs after treatment with Valproic acid (VPA), a small-molecule
epigenetic modifier.
- The use of serum-free conditions to increase ZFN-mediated
targeted integration efficiency in isolated T-cells.
Second Quarter 2016 Results
For the second quarter
ended June 30, 2016, Sangamo reported
a consolidated net loss of $26.6
million, or $0.38 per share,
compared to a net loss of $12.1
million, or $0.17 per share,
for the same period in 2015. As of June 30,
2016, the Company had cash, cash equivalents, marketable
securities and interest receivable of $172.6
million.
Revenues for the second quarter of 2016 were $3.7 million, compared to $8.4 million for the same period in 2015. Second
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 second quarter of 2016 consisted of
$3.6 million from collaboration
agreements and $0.1 million from
research grants, compared to $7.8
million and $0.6 million,
respectively, for the same period in 2015. The decrease in
collaboration agreement revenues was primarily 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.
In the second quarter of 2016, Sangamo recognized $1.9 million of revenues related to research
services performed under the collaboration agreement with Biogen,
and $0.4 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 second quarter of 2016.
Research and development expenses were $19.5 million for the second quarter of 2016,
compared to $15.6 million for the
same period in 2015. The increase was primarily due to increases in
manufacturing and clinical trial expenses, consulting and
personnel-related expenses.
General and administrative expenses were $11.1 million for the second quarter of 2016,
compared to $5.0 million for the same
period in 2015. The increase was primarily due to corporate costs
and separation expenses associated with the CEO transition, the
majority of which was non-cash, stock-based compensation
expense.
Total operating expenses for the second quarter of 2016 were
$30.5 million, compared to
$20.6 million for the same period in
2015.
Six Months Results
For the six months ended
June 30, 2016, the consolidated net
loss was $43.1 million, or
$0.61 per share, compared to a
consolidated net loss of $17.4
million, or $0.25 per share,
for the six months ended June 30,
2015. Revenues were $7.6
million for the first half of 2016, compared to $21.8 million for the same period in 2015. Total
operating expenses were $51.2 million
for the first half of 2016, compared to $40.3 million for the first half of 2015.
Financial Guidance for 2016
The Company updates
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.
The Company reiterates guidance as follows:
- 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, August 3, 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 52450915. A conference
call replay will be available for one week following the conference
call, from approximately 8:00 p.m. ET
on August 3, 2016 to 11:59 p.m. ET on August
10, 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
52450915.
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 In Vivo
Protein Replacement Platform™ (IVPRP) approach is focused on
monogenic diseases, including hemophilia and lysosomal storage
disorders. Based on its proprietary IVPRP approach, Sangamo is
initiating a Phase 1/2 clinical trial for hemophilia B, the first
in vivo genome editing application cleared by the FDA. In
addition, Sangamo has a Phase 2 clinical program to evaluate the
safety and efficacy of novel ZFP Therapeutics® for the
treatment of HIV/AIDS (SB-728). The Company has also formed a
strategic collaboration with Biogen Inc. for hemoglobinopathies,
such as 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 potential milestone and
royalty payments under Sangamo's agreements with Shire and Biogen,
the research and development of ZFNs and ZFP TFs, clinical trials
and therapeutic applications of Sangamo's ZFP technology platform
and achievement of research milestones and objectives under
collaboration agreements with Shire and Biogen. These statements
are not guarantees of future performance 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
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
|
|
Six Months
Ended
|
|
June
30,
|
|
June
30,
|
|
2016
|
|
2015
|
|
2016
|
|
2015
|
Statement of
Operations Data:
|
|
|
|
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
Collaboration
agreements
|
$
3,592
|
|
$
7,801
|
|
$
7,303
|
|
$
20,472
|
Research
grants
|
110
|
|
557
|
|
341
|
|
1,377
|
Total
revenues
|
3,702
|
|
8,358
|
|
7,644
|
|
21,849
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
19,454
|
|
15,618
|
|
34,720
|
|
30,598
|
General and
administrative
|
11,090
|
|
5,017
|
|
16,447
|
|
9,749
|
Total operating
expenses
|
30,544
|
|
20,635
|
|
51,167
|
|
40,347
|
Loss from
operations
|
(26,842)
|
|
(12,277)
|
|
(43,523)
|
|
(18,498)
|
Interest and other
income, net
|
243
|
|
151
|
|
430
|
|
305
|
Loss before
taxes
|
(26,599)
|
|
(12,126)
|
|
(43,093)
|
|
(18,193)
|
Benefit from income
taxes
|
24
|
|
-
|
|
24
|
|
748
|
Net loss
|
$
(26,575)
|
|
$
(12,126)
|
|
$(43,069)
|
|
$(17,445)
|
Basic and diluted net
loss per common share
|
$
(0.38)
|
|
$
(0.17)
|
|
$
(0.61)
|
|
$
(0.25)
|
|
|
|
|
|
|
|
|
Shares used in
computing basic and diluted net loss per common share
|
70,487
|
|
69,684
|
|
70,430
|
|
69,485
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
SELECTED BALANCE
SHEET DATA
|
|
|
|
|
|
|
|
|
June 30,
2016
|
|
December 31,
2015
|
|
|
|
|
|
(Unaudited)
|
|
|
|
|
|
|
Cash, cash
equivalents, marketable securities and interest
receivable
|
$
172,575
|
|
$
209,307
|
|
|
|
|
Total
assets
|
180,906
|
|
217,235
|
|
|
|
|
Total stockholders'
equity
|
160,023
|
|
192,439
|
|
|
|
|
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SOURCE Sangamo BioSciences, Inc.