- Announced additional progress towards strategic transformation
into neurology-focused genomic medicine company.
- Focusing resources on proprietary epigenetic regulation
therapies treating neurological diseases and novel AAV capsid
delivery technologies.
- Dosed a total of 25 patients in Phase 1/2 STAAR study in Fabry
disease, with promising clinical data continuing to emerge.
Deferring additional investments in Phase 3 planning until
collaboration partner or Phase 3 trial funding secured.
- Actively seeking collaboration partners or direct investors in
CAR-Treg cell therapy programs. Deferring new investments until
collaboration partner or external investment is secured.
- Announced planned shutdown of Brisbane headquarters,
restructuring of operations, and US workforce reduction of
approximately 40%.
- Cost savings expected from the restructuring, workforce
reduction and other potential cost reductions anticipated to reduce
annual operating expenses by approximately 50%.
- Conference call and webcast scheduled for Thursday, November 2,
8:30 a.m. Eastern Time.
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines
company, today announced recent business highlights, including
progress on its strategic transformation and a corresponding
restructuring of operations and workforce reduction, and reported
third quarter 2023 financial results.
“In 2020, we shared our refreshed company strategy which aims to
both maximize the potential of our proprietary genomic editing and
delivery technology, and to focus on areas where we believe we can
apply that technology to be either first-in-class or best-in-class.
The process of streamlining Sangamo’s pipeline has been accelerated
within today’s challenging economic environment and we have had to
make difficult decisions to defer further investments and seek
collaboration partners or direct investment in both our Fabry gene
therapy and CAR-Treg cell therapy programs,” said Sandy Macrae,
Chief Executive Officer of Sangamo. “As we work to unlock value in
our clinical programs that is not currently reflected as part of
Sangamo, we plan to do everything in our power to get these
important assets into the hands of parties with the means to
advance them towards patients. At the same time, we will continue
to progress our promising epigenetic regulation programs for
neurological diseases and hope to soon share a breakthrough in our
capsid delivery capabilities, which we believe could open the door
for many other high-value and unmet diseases to be addressed with
our editing capabilities. We continue to seek ways to raise
additional capital to strengthen our financial foundation.”
The restructuring announced today represents a further step
towards simplifying the Sangamo organization and focusing on our
epigenetic regulation therapies treating neurological diseases and
our novel AAV capsid delivery technologies. Sangamo is deferring
new investments in its Fabry and CAR-Treg programs beyond what is
currently committed and is actively seeking collaboration partners
or direct investors in both. In addition, Sangamo expects to close
its Brisbane, California facility in early 2024 to conserve cash
resources, and will transition its headquarters to its Richmond,
California facility as of January 1, 2024. As a result of this
restructuring, Sangamo is reducing its US workforce by
approximately 40%, or approximately 162 roles.
In connection with the restructuring, D. Mark McClung, Executive
Vice President, Chief Operating Officer and Jason Fontenot, Senior
Vice President, Chief Scientific Officer will be leaving the
company on January 2, 2024. In the context of a streamlined and
more focused organization, we are eliminating their roles. Until
his departure, Mark will continue to lead our search for partners
and investors in our Fabry and CAR-Treg programs. Amy Pooler,
currently serving as Vice President, Neuroscience and Gregory
Davis, currently serving as Vice President, Genome Engineering
Design and Technology, have been appointed as Head of Research and
Head of Technology, respectively, effective November 17, 2023.
The restructuring and workforce reductions, in combination with
other potential cost reductions, are anticipated to reduce our
non-GAAP annual operating expenses from approximately $240
million-$260 million in 2023 to approximately $115 million-$135
million in 2024, a decrease of approximately 50%. Sangamo expects
to incur approximately $8 million-$10 million in one-time
restructuring costs in the fourth quarter of 2023. Sangamo believes
its cash, cash equivalents, and marketable securities as of
September 30, 2023, in combination with the cost savings expected
from the restructuring, workforce reduction and other potential
cost reductions, will be sufficient to fund its planned operations
into the third quarter of 2024.
Dr. Macrae continued: “I am grateful to all our employees for
their commitment to Sangamo’s mission and the patients we seek to
serve, and have special appreciation to all those who are leaving
for their important contributions. Additionally, I would like to
personally thank Mark for the wisdom, candor and leadership he has
brought to Sangamo. He has been a trusted colleague and will be
greatly missed. I would also like to thank Jason for his
dedication, passion and leadership. He leaves a strong scientific
legacy for which we will always be grateful.”
Recent Business Highlights
Program Highlights
Neurology Epigenetic Regulation Programs – Progressed
IND-enabling activities for Nav1.7; presented updated preclinical
data at Prion 2023; presented preclinical data on zinc finger
activators at the European Society of Gene and Cell Therapy
(ESGCT); made significant progress in identifying new, potentially
transformative AAV delivery capsids.
- Progressed IND enabling activities for the Nav1.7 program to
treat chronic neuropathic pain. Continue to expect an IND
submission for this program in 2024.
- Presented data from the prion disease program at the Prion 2023
Conference in October 2023, showing in animal models that Sangamo’s
zinc finger repressors significantly reduce expression of the prion
protein in the brain, extend lifespan and limit the formation of
toxic prion aggregates.
- Presented an oral presentation at ESGCT in October 2023,
showing that our zinc finger activators can be designed to restore
normal gene and protein expression of SCN2A in vitro and in vivo to
potentially address neurodevelopmental disorders such as autism
spectrum disorder and intellectual disability.
- Presented data on Shank3 gene activation mediated by zinc
finger transcriptional activators as a potential therapeutic
approach for Phelan-McDermid Syndrome at ESGCT.
- Continued to advance identification and selection of engineered
AAV capsids for enhanced central nervous system delivery through
both intrathecal and intravenous delivery. Anticipate sharing
nonhuman primate data from our capsid development efforts in early
2024.
Fabry Disease – Dosed total of 25 patients in Phase 1/2
STAAR study; all patients dosed to date continue to demonstrate
sustained, elevated α-Gal A levels for up to three years for the
longest treated patient; received Regenerative Medicine Advanced
Therapy (RMAT) Designation from U.S. FDA; enrolled sufficient
patients in the Phase 1/2 study believed to provide a preliminary
assessment of safety and efficacy; deferring Phase 3 planning
investments and actively seeking partners and investment.
- Dosed three additional patients in the dose expansion phase of
the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our
wholly owned gene therapy product for the treatment of Fabry
disease, for a total of 25 patients dosed to date, including 14 at
the planned Phase 3 dose of 5x1013 vg/kg.
- All patients dosed to date continue to demonstrate sustained,
elevated α-Gal A levels, with 12 patients having achieved at least
one year of follow-up and the longest treated patient having
achieved three years of follow-up.
- All 11 patients who were withdrawn from enzyme replacement
therapy (ERT) remain off ERT, for up to 24 months for the longest
withdrawn patient.
- Treated patients continue to report improvements in their
quality of life, some even over and above the benefits they were
experiencing on ERT.
- Received U.S. FDA RMAT designation for isaralgagene
civaparvovec, which aims to expedite the review of new therapeutics
that are intended to address an unmet need in patients with serious
conditions. The U.S. FDA has previously granted isaralgagene
civaparvovec both Orphan Drug and Fast Track Designations.
- Stopping further screening and enrollment in the Phase 1/2
STAAR study, after successfully enrolling sufficient patients
believed to provide a preliminary assessment of efficacy and safety
in the Phase 1/2 study.
- Expect to complete dosing of the remaining enrolled patients in
the first half of 2024.
- Anticipate presenting updated Phase 1/2 clinical data at a
medical meeting in early 2024.
- Deferring additional investments in Phase 3 planning until
collaboration partnership or Phase 3 trial financing is
secured.
CAR-Tregs – Received approval for accelerated dosing
protocol for Phase 1/2 STEADFAST study from European regulatory
authorities; dosed the first patient in the second dose cohort;
successfully manufactured product candidate for third and new
fourth dose levels; presented updated preclinical data at ESGCT;
seeking a collaboration partner or direct investment in
CAR-Tregs.
- Dosed the first patient in the second cohort of the Phase 1/2
STEADFAST study evaluating TX200, our wholly owned autologous
CAR-Treg cell therapy treating patients receiving an HLA-A2
mismatched kidney from a living donor.
- The product candidate continues to be generally well tolerated
in all four patients dosed to date.
- Received all necessary regulatory and ethics approvals for an
accelerated dose escalation protocol from European regulatory
authorities that could allow dosing to advance more quickly through
the cohorts and which allows for a new and highest fourth dose
cohort, compared to the three cohorts in the previously approved
study protocol. The new, fourth cohort dose will be 18-fold higher
than the first cohort starting dose.
- Completed manufacturing of the dose for the patient in the
third cohort, who recently received a kidney transplant. Dosing of
this fifth patient is expected in the fourth quarter of 2023,
pending approval from the Safety Monitoring Committee.
- Completed manufacturing of the dose for the first patient in
the fourth and highest dose cohort, who recently received a kidney
transplant. Dosing of this sixth patient is expected in January
2024, pending approval from the Safety Monitoring Committee – which
would accelerate dosing plans by 18 months compared to the
previously approved study protocol.
- Presented preclinical data at ESGCT demonstrating the potential
of autologous MOG-CAR-Tregs to provide a long-lasting treatment
option for multiple sclerosis and updated animal model data
demonstrating the promise of IL23R-CAR-Tregs in the potential
treatment of Crohn’s disease.
- Actively seeking a potential collaboration partner or direct
external investment in the CAR-Treg cell therapy programs. Expect
to provide an update on these efforts in the first quarter of
2024.
- Deferring new investments until a collaboration partner or
external investment is secured.
Hemophilia A (Pfizer) – dosing complete in Phase 3 AFFINE
trial; pivotal data read-out expected in mid-2024; BLA and MAA
submissions anticipated in second half of 2024.
- Pfizer has advised us that dosing is complete in the Phase 3
AFFINE trial of giroctocogene fitelparvovec, an investigational
gene therapy we are developing with Pfizer for patients with
moderately severe to severe hemophilia A.
- A pivotal readout is expected in mid-2024, with Pfizer
anticipating BLA and MAA submissions in the second half of 2024 if
the pivotal readout is supportive.
- Expect to present updated data with Pfizer from the Phase 1/2
ALTA study of giroctocogene fitelparvovec in an oral presentation
at the 65th American Society for Hematology Annual Meeting and
Exposition on December 11, 2023.
Third Quarter 2023 Financial Results
Consolidated net loss for the third quarter ended September 30,
2023 was $104.2 million, or $0.59 per share, compared to a net loss
of $53.2 million, or $0.34 per share, for the same period in 2022,
primarily due to a non-cash charge relating to impairment of
long-lived asset of $44.8 million, which was a result of continued
decline in our stock price and related market capitalization,
initiation of actions to seek external financing and reprioritize
certain research and development programs, and continued decline in
equity values in the biotechnology industry.
Revenues
Revenues for the third quarter ended September 30, 2023 were
$9.4 million, compared to $26.5 million for the same period in
2022.
The decrease of $17.1 million in revenues was primarily
attributed to a decrease of $9.6 million and $9.1 million in
revenue relating to our collaboration agreements with Novartis and
Biogen, respectively, due to the termination of these collaboration
agreements in June 2023, and a decrease of $1.4 million in revenue
relating to our collaboration agreement with Kite, reflecting a
reduction in collaboration activities during the quarter. These
decreases were partially offset by an increase of $3.0 million in
revenue relating to our other license agreements.
GAAP and Non-GAAP Operating Expenses
Three Months Ended
Nine Months Ended
September 30,
September 30,
(In millions)
2023
2022
2023
2022
Research and development
$
57.1
$
65.1
$
183.4
$
183.7
General and administrative
13.9
16.2
48.1
46.2
Impairment of goodwill and indefinite-lived intangible assets
-
-
89.5
-
Impairment of long-lived assets
44.8
-
65.2
-
Total operating expenses
115.8
81.3
386.2
229.9
Impairment of goodwill and indefinite-lived intangible assets
-
-
(89.5
)
-
Impairment of long-lived assets
(44.8
)
-
(65.2
)
-
Stock-based compensation expense
(6.2
)
(7.8
)
(21.3
)
(23.4
)
Non-GAAP operating expenses
$
64.8
$
73.5
$
210.2
$
206.5
Total operating expenses on a GAAP basis for the third quarter
ended September 30, 2023 were $115.8 million, compared to $81.3
million for the same period in 2022. GAAP operating expenses for
the third quarter ended September 30, 2023 included a non-cash
charge relating to impairment of long-lived asset of $44.8 million,
as described above. Non-GAAP operating expenses, which exclude
impairment charges and stock-based compensation expense, for the
third quarter ended September 30, 2023 were $64.8 million, compared
to $73.5 million for the same period in 2022.
The decrease in total operating expenses on a non-GAAP basis was
primarily attributable to lower compensation and other personnel
costs mainly due to lower headcount as a result of restructuring of
operations and corresponding reduction in workforce announced in
April 2023, and decrease in manufacturing and lab supply expenses
due to deferral and reprioritization of certain research and
development programs. These decreases were partially offset by
higher facilities and infrastructure related costs, and higher
external expenses as we advance our clinical and preclinical
pipeline.
Cash, Cash Equivalents and Marketable Securities
Cash, cash equivalents and marketable securities as of September
30, 2023 were $132.1 million, compared to $307.5 million as of
December 31, 2022. As of September 30, 2023, we have raised
approximately $15.1 million in net proceeds under our at-the-market
offering program since January 1, 2023. We believe that our
available cash, cash equivalents and marketable securities as of
September 30, 2023, in combination with the cost savings expected
from the restructuring, workforce reduction and other potential
cost reductions, will be sufficient to fund our planned operations
into the third quarter of 2024.
Updated Financial Guidance for 2023
- GAAP operating expenses, including impairment of goodwill,
indefinite-lived intangible assets, and long-lived assets, and
stock-based compensation expense, for the full year 2023 are now
estimated to be in the range of approximately $422 million to $442
million, reflecting the additional non-cash impairment charges
recorded in the third quarter. The previous GAAP operating expenses
guidance provided on August 8, 2023 was in the range of
approximately $378 million to $398 million.
- We continue to estimate non-GAAP operating expenses to be in
the range of approximately $240 million to $260 million, which
remains unchanged from the last update on April 26, 2023. Estimated
non-GAAP operating expenses exclude impairment of goodwill of $38.1
million, impairment of indefinite-lived intangible assets of $51.3
million, impairment of long-lived assets of $65.2 million, and
stock-based compensation expense of $28.0 million.
Upcoming Events
Sangamo plans to participate in the following events:
Investor Conferences
- Truist Securities BioPharma Symposium, November 8-9, 2023
- Jefferies London Healthcare Conference, November 14-16,
2023
- EvercoreISI HealthCONx, November 28-30, 2023
Access links for available webcasts for these investor
conferences will be available on the Sangamo website in the
Investors and Media section under Events. Available materials will
be found on the Sangamo website after the event under
Presentations.
Conference Call to Discuss Third Quarter 2023 Results
The Sangamo management team will discuss these results on a
conference call tomorrow, Thursday, November 2, 2023, at 8:30 a.m.
Eastern Time.
Participants should register for, and access, the call using
this link. While not required, it is recommended you join 10
minutes prior to the event start. Once registered, participants
will be given the option to either dial into the call with the
number and unique passcode provided or to use the dial-out option
to connect their phone instantly.
An updated corporate presentation is available in the Investors
and Media section under Presentations.
The link to access the live webcast can also be found on the
Sangamo website in the Investors and Media section under Events. A
replay will be available following the conference call, accessible
at the same link.
About Sangamo Therapeutics
Sangamo Therapeutics is a clinical-stage biopharmaceutical
company with a robust genomic medicines pipeline. Using
ground-breaking science, including our proprietary zinc finger
genome engineering technology and manufacturing expertise, Sangamo
aims to create new genomic medicines for patients suffering from
diseases for which existing treatment options are inadequate or
currently don’t exist. To learn more, visit www.sangamo.com and
connect with us on LinkedIn and Twitter.
Forward-Looking Statements
This press release contains forward-looking statements regarding
our current expectations. These forward-looking statements include,
without limitation, statements relating to: the therapeutic and
commercial potential of our product candidates, the anticipated
plans and timelines of Sangamo and our collaborators for screening,
enrolling and dosing patients in and conducting our ongoing and
potential future clinical trials and presenting clinical data from
our clinical trials, including expectations regarding the
conclusion of dosing in our Phase 1/2 STAAR study, preparations and
plans for patient dosing in the STEADFAST study, the potential for
acceleration of the study timeline and the availability of data
therefrom, the anticipated advancement of our product candidates to
late-stage development, including Sangamo’s plans to seek a
potential partner or additional financing to proceed with potential
future Phase 3 trials of isaralgagene civaparvovec and the timing
thereof, the timeline to present data from the Phase 3 AFFINE trial
and to make BLA and MAA submissions for giroctocogene
fitelparvovec, expectations regarding advancement of our
preclinical neurology programs, including announcement of data
from, and anticipated IND submissions related to, such programs,
plans to seek a partner for or investor in our CAR-Treg program,
expectations concerning our strategic prioritization and
restructuring, including plans to close our Brisbane facility and
the expected charges and cost savings associated with such
restructuring, future potential cost reductions, our expected cash
runway, our 2023 financial guidance related to GAAP and non-GAAP
total operating expenses, impairments and stock-based compensation,
our plans to participate in industry and investor conferences, and
other statements that are not historical fact. These statements are
not guarantees of future performance and are subject to certain
risks and uncertainties that are difficult to predict. Factors that
could cause actual results to differ include, but are not limited
to, risks and uncertainties related to Sangamo’s ability to execute
its strategic prioritization and restructuring as currently
contemplated; the actual charges associated with the restructuring
being higher than anticipated or changes to the assumptions on
which the estimated charges associated with the restructuring are
based; Sangamo’s ability to achieve projected cost savings in
connection with the restructuring and to further reduce operating
expenses; unintended consequences from the restructuring that
impact Sangamo’s business; our lack of capital resources to fully
develop, obtain regulatory approval for and commercialize our
product candidates, including our ability to secure the funding
required to initiate a potential Phase 3 trial of isaralgagene
civaparvovec in a timely manner or at all; our need for substantial
additional funding to execute our operating plan and to continue to
operate as a going concern; the potential of our preclinical
programs utilizing zinc finger technology to address neurological
health disorders; the effects of macroeconomic factors or financial
challenges, including as a result of the ongoing overseas conflict,
current or potential future bank failures, inflation and rising
interest rates, on the global business environment, healthcare
systems and business and operations of Sangamo and our
collaborators, including the initiation and operation of clinical
trials; the research and development process, including the
enrollment, operation and results of clinical trials and the
presentation of clinical data; the impacts of clinical trial
delays, pauses and holds on clinical trial timelines and
commercialization of product candidates; the uncertain timing and
unpredictable nature of clinical trial results, including the risk
that therapeutic effects in the Phase 3 AFFINE trial will not be
durable in patients as well as the risk that the therapeutic
effects observed in the latest preliminary clinical data from the
Phase 1/2 STAAR study will not be durable in patients and that
final clinical trial data from the study will not validate the
safety and efficacy of isaralgagene civaparvovec, and that the
patients withdrawn from ERT will remain off ERT; the unpredictable
regulatory approval process for product candidates across multiple
regulatory authorities; reliance on results of early clinical
trials, which results are not necessarily predictive of future
clinical trial results, including the results of any Phase 3 trial
of our product candidates; the potential for technological
developments that obviate technologies used by Sangamo; our
reliance on collaborators and our potential inability to secure
additional collaborations, and our ability to achieve expected
future financial performance.
There can be no assurance that we and our collaborators will be
able to develop commercially viable products. Actual results may
differ materially from those projected in these forward-looking
statements due to the risks and uncertainties described above and
other risks and uncertainties that exist in the operations and
business environments of Sangamo and our collaborators. These risks
and uncertainties are described more fully in our Securities and
Exchange Commission, or SEC, filings and reports, including in our
Annual Report on Form 10-K for the year ended December 31, 2022, as
supplemented by our Quarterly Report on Form 10-Q for the quarter
ended September 30, 2023 to be filed with the SEC, and future
filings and reports that Sangamo makes from time to time with the
SEC. Forward-looking statements contained in this announcement are
made as of this date, and we undertake no duty to update such
information except as required under applicable law.
Non-GAAP Financial Measures
To supplement our financial results and guidance presented in
accordance with GAAP, we present non-GAAP operating expenses, which
excludes stock-based compensation expense and impairment of
goodwill, indefinite-lived intangible assets and long-lived assets
from GAAP operating expenses. We believe that this non-GAAP
financial measure, when considered together with our financial
information prepared in accordance with GAAP, can enhance
investors’ and analysts’ ability to meaningfully compare our
results from period to period and to our forward-looking guidance,
and to identify operating trends in our business. We have excluded
stock-based compensation expense because it is a non-cash expense
that may vary significantly from period to period as a result of
changes not directly or immediately related to the operational
performance for the periods presented, and we have excluded
impairment of goodwill, indefinite-lived intangible assets and
long-lived assets to facilitate a more meaningful evaluation of our
current operating performance and comparisons to our operating
performance in other periods. This non-GAAP financial measure is in
addition to, not a substitute for, or superior to, measures of
financial performance prepared in accordance with GAAP. We
encourage investors to carefully consider our results under GAAP,
as well as our supplemental non-GAAP financial information, to more
fully understand our business.
SELECTED CONSOLIDATED FINANCIAL DATA (unaudited; in
thousands, except per share data)
Statement of
Operations Data:
Three months ended
Nine months ended
September 30,
September 30,
2023
2022
2023
2022
Revenues
$
9,398
$
26,460
$
174,190
$
84,069
Operating expenses: Research and development
57,089
65,116
183,351
183,719
General and administrative
13,918
16,238
48,068
46,239
Impairment of goodwill and indefinite-lived intangible assets
-
-
89,485
-
Impairment of long-lived assets
44,799
-
65,232
-
Total operating expenses
115,806
81,354
386,136
229,958
Loss from operations
(106,408
)
(54,894
)
(211,946
)
(145,889
)
Interest and other income, net
3,515
1,769
9,610
5,754
Loss before income taxes
(102,893
)
(53,125
)
(202,336
)
(140,135
)
Income tax expense (benefit)
1,270
30
(4,800
)
170
Net loss
$
(104,163
)
$
(53,155
)
$
(197,536
)
$
(140,305
)
Basic and diluted net loss per share
$
(0.59
)
$
(0.34
)
$
(1.14
)
$
(0.93
)
Shares used in computing basic and diluted net loss per share
177,171
158,042
173,375
150,850
Selected Balance Sheet Data: September 30, 2023 December 31, 2022 Cash, cash
equivalents, and marketable securities
$
132,111
$
307,477
Total assets
$
219,697
$
562,509
Total stockholders' equity
$
134,922
$
294,958
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231101893355/en/
Investor Relations & Media
Inquiries Louise Wilkie ir@sangamo.com media@sangamo.com
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