Showcased 20 presentations at the 27th American
Society of Gene & Cell Therapy (ASGCT) Annual Meeting
demonstrating progression of neurology-focused preclinical
pipeline, including additional data from novel delivery capsid,
STAC-BBB, that demonstrated industry-leading blood-brain barrier
(BBB) penetration in non-human primates (NHPs) following
intravenous administration.
Announced discovery of novel next-generation
modular integrase (MINT) platform that allows targeting of a serine
recombinase engineered to enable large-scale genome editing.
Completed dosing in Phase 1/2 STAAR study of
isaralgagene civaparvovec for Fabry disease and engaged in active
discussions with potential Fabry collaboration partners.
Pfizer anticipates biologics license
application (BLA) and marketing authorization application (MAA)
submissions for Hemophilia A collaboration in early 2025 if
mid-2024 pivotal readout is supportive.
Raised approximately $24 million in gross
proceeds from a registered direct offering with institutional
investors.
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines
company, today reported business highlights and first quarter 2024
financial results.
“Sangamo continues to progress its neurology-focused genomic
medicine pipeline as most recently showcased at the 27th ASGCT
Annual Meeting, with foundational advances across our epigenetic
regulation programs and capsid engineering platform, supported by
recent discovery of the neurotropic capsid STAC-BBB, and our novel
next-generation integrase technology,” said Sandy Macrae, Chief
Executive Officer of Sangamo. “We are pleased with the progress
being made in business development discussions across our
portfolio, including our Fabry disease program. We are building on
the momentum achieved in the first quarter and look forward to
achieving meaningful potential milestones throughout the rest of
2024 and beyond.”
Recent Business Highlights
Corporate Updates
- Raised approximately $24 million in gross proceeds from a
registered direct offering with institutional investors.
Prioritized Neurology Pipeline
Neurology Epigenetic Regulation Programs
- Investigational new drug (IND) enabling toxicology studies are
nearing completion in the Nav1.7 program to treat chronic
neuropathic pain. An IND submission is expected in the fourth
quarter of 2024.
- Clinical trial authorization (CTA) enabling activities are
advancing for Sangamo’s program to treat prion disease, leveraging
the novel STAC-BBB capsid. A CTA submission is expected in the
fourth quarter of 2025.
- Intend to resume development of previously paused zinc finger
repressor program addressing tauopathies, including Alzheimer’s
disease, which leverages STAC-BBB. IND submission could occur as
early as the fourth quarter of 2025.
- Showcased 10 poster presentations at the ASGCT Annual Meeting
demonstrating advances in epigenetic regulation for the treatment
of various neurological diseases, including prion disease and
tauopathies.
Novel Adeno-Associated Virus (AAV) Capsid Delivery
Technology
- Announced preclinical data from novel AAV capsid, STAC-BBB,
that demonstrated industry-leading BBB penetration in NHPs
following intravenous administration, with capsid-enabled delivery
of zinc finger payloads targeting prion disease and tauopathies,
resulting in potent and widespread repression of target genes.
- Presented two platform presentations and four poster
presentations at ASGCT outlining developments in AAV capsid
delivery capabilities developed through Sangamo’s SIFTER capsid
engineering platform.
- Engaged in ongoing business development discussions with
potential collaborators for STAC-BBB.
Next-Generation Genome Engineering
- Presented one platform and three poster presentations at ASGCT
showcasing Sangamo’s next-generation genome engineering
capabilities, including the discovery of the Modular Integrase
(MINT) platform, a versatile, protein-guided genome editing method
designed to integrate large sequences of DNA into the genome to
potentially treat – with a single medicine – patients who have
unique mutations in the same gene.
- Published a manuscript in bioRxiv titled, “Systematic
Development of Reprogrammed Modular Integrases Enables Precise
Genomic Integration of Large DNA Sequences” further detailing how
the MINT platform builds on the strength of Sangamo’s structural
protein-DNA engineering capabilities derived from its zinc finger
platform to target Bxb1, a serine recombinase. This technology is
intended to integrate entire genes into the genome, to avoid
double-stranded DNA breaks and the need for assistance from
ancillary genome editing or DNA-repair modulating cargo.
- The MINT platform could be deployed internally for
neurology-focused indications, and could provide potential
partnering opportunities, both for human disease and in
agricultural biotech settings.
- Engaged in ongoing business development discussions for
Sangamo’s modular integrase capabilities with potential
collaborators.
Other Programs
Fabry Disease
- Dosed the final patient in the Phase 1/2 STAAR study of
isaralgagene civaparvovec, an investigational gene therapy for the
treatment of Fabry disease, resulting in a total of 33 patients
dosed in the study. Screening, enrollment and dosing are now
complete.
- One additional patient was withdrawn from enzyme replacement
therapy (ERT), resulting in a total of 14 patients withdrawn from
ERT to date. The four patients dosed since February 2024 who began
the study on ERT have plans in place to withdraw ERT treatment at
the appropriate time.
- Presented updated clinical data in February 2024 at the 20th
Annual WORLDSymposium in San Diego, CA, showing sustained benefit
and differentiated safety profile for isaralgagene
civaparvovec.
- Aligned with U.S. Food and Drug Administration (FDA) on an
abbreviated pathway to potential approval. Granted PRIME
eligibility from the European Medicines Agency (EMA) and Innovative
Licensing and Access Pathway (ILAP) from U.K. Medicines and
Healthcare products Regulatory Agency (MHRA) for isaralgagene
civaparvovec.
- Engaged in active discussions with potential collaboration
partners for the Fabry disease program. Deferring additional
investments, including planning for a potential registrational
trial, until a collaboration partnership or financing is
secured.
Hemophilia A
- Pivotal readout expected in mid-2024 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.
- Pfizer anticipates BLA and MAA submissions in early 2025 if the
pivotal readout is supportive.
- Eligible to earn from Pfizer up to $220.0 million in milestone
payments upon the achievement of certain regulatory and commercial
milestones for giroctocogene fitelparvovec and product sales
royalties of 14% - 20% if giroctocogene fitelparvovec is approved
and commercialized, subject to certain reductions.
First Quarter 2024 Financial Results
Consolidated net loss for the first quarter ended March 31, 2024
was $49.1 million, compared to a net income of $21.1 million, for
the same period in 2023.
Basic and diluted net loss per share for the first quarter ended
March 31, 2024 was $0.27, compared to basic net income per share of
$0.13 per share and diluted net income per share of $ 0.12 per
share for the same period in 2023.
Revenues
Revenues for the first quarter ended March 31, 2024 were $0.5
million, compared to $158.0 million for the same period in
2023.
The decrease of $157.5 million in revenues was primarily
attributed to decreases of $132.3 million and $9.8 million in
revenues relating to our collaboration agreements with Biogen and
Novartis, respectively, due to the termination of collaboration
agreements in June 2023, a decrease of $12.3 million in revenue
relating to our collaboration agreement with Kite which expired
pursuant to its terms in April 2024, and a decrease of $3.1 million
in revenue relating to our license agreements with Sigma-Aldrich
Corporation and Ligand Pharmaceuticals Inc.
GAAP and Non-GAAP Operating Expenses
Three Months Ended March 31, (In millions)
2024
2023
Research and development
$
35.9
$
63.2
General and administrative
11.8
18.1
Impairment of long-lived assets
4.3
20.4
Impairment of goodwill
-
38.1
Total operating expenses
52.0
139.8
Impairment of long-lived assets
(4.3
)
(20.4
)
Impairment of goodwill
-
(38.1
)
Depreciation and amortization
(1.4
)
(3.5
)
Stock-based compensation expense
(2.7
)
(8.3
)
Non-GAAP operating expenses
$
43.6
$
69.5
Total operating expenses on a GAAP basis for the first quarter
ended March 31, 2024 were $52.0 million compared to $139.8 million
for the same period in 2023. Non-GAAP operating expenses, which
exclude impairment charges, depreciation and amortization, and
stock-based compensation expense, for the first quarter ended March
31, 2024 were $43.6 million, compared to $69.5 million for the same
period in 2023.
The decrease in total operating expenses on a non-GAAP basis was
primarily attributable to lower compensation and other personnel
costs mainly due to a reduction in the bonus expense and lower
headcount as a result of restructuring of operations and a
corresponding reduction in workforce announced during 2023 and the
first quarter of 2024, and decrease in preclinical and clinical
expenses due to termination of collaboration agreements, and
deferral and reprioritization of certain research and development
programs.
Cash and Cash Equivalents
Cash and cash equivalents as of March 31, 2024 were $54.4
million, compared to cash, cash equivalents and marketable
securities of $81.0 million as of December 31, 2023. In March, we
completed a registered direct offering of common stock, for net
proceeds of approximately $21.8 million after deducting placement
agents’ fees and estimated offering expenses payable by us. We
believe that our available cash and cash equivalents as of March
31, 2024, in combination with the cost savings expected from the
restructurings, workforce reduction and other potential cost
reductions, will be sufficient to fund our planned operations into
the third quarter of 2024.
Financial Guidance for 2024 Reiterated (initial guidance
provided on March 13, 2024)
- On a GAAP basis, we continue to expect total operating expenses
in the range of approximately $145 million to $165 million in 2024,
which includes non-cash stock-based compensation expense and
depreciation and amortization, subject to additional funding.
- We continue to expect non-GAAP total operating expenses,
excluding estimated non-cash stock-based compensation expense of
approximately $13 million, and depreciation and amortization of
approximately $7 million, in the range of approximately $125
million to $145 million in 2024, subject to additional
funding.
Upcoming Events
Sangamo plans to participate in the following events:
Investor Conferences:
- RBC Capital Markets Global Healthcare Conference, May 14-15,
2024
- Jefferies Global Healthcare Conference, June 5-6, 2024
- H.C. Wainwright 5th Annual Neuro Perspectives Virtual
Conference, week of June 24, 2024
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
The Sangamo management team will hold a corporate call to
further discuss program advancements and financial updates on
Thursday, May 9 at 4:30pm 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 genomic medicine company dedicated to
translating ground-breaking science into medicines that transform
the lives of patients and families afflicted with serious
neurological diseases who do not have adequate or any treatment
options. Sangamo believes that its zinc finger epigenetic
regulators are ideally suited to potentially address devastating
neurological disorders and that its capsid discovery platform can
expand delivery beyond currently available intrathecal delivery
capsids, including in the central nervous system. Sangamo’s
pipeline also includes multiple partnered programs and programs
with opportunities for partnership and investment. To learn more,
visit www.sangamo.com and connect with us on LinkedIn and
Twitter/X.
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 Sangamo’s product candidates and its
engineered capsids and the potential of its next generation genome
engineering technology, the anticipated plans and timelines of
Sangamo and its collaborators in conducting our ongoing and
potential future clinical trials and presenting clinical data from
such clinical trials, the anticipated advancement of Sangamo’s
product candidates to late-stage development, including plans to
seek a potential partner or additional financing to proceed with
potential future Phase 3 trials of isaralgagene civaparvovec and
the design and timing thereof, the timeline to present data from
the Phase 3 AFFINE trial and to make BLA and MAA submissions for
giroctocogene fitelparvovec, the potential to earn milestone
payments and receive product sales royalties if giroctocogene
fitelparvovec is approved and commercialized, expectations
regarding advancement of Sangamo’s preclinical neurology programs,
including announcement of data from, and anticipated IND and CTA
submissions related to, such programs, the potential of the MINT
platform to enable large-scale genomic engineering, Sangamo’s
expected cash runway, Sangamo’s 2024 financial guidance related to
GAAP and non-GAAP total operating expenses, impairments and
stock-based compensation, plans to participate in industry and
investor conferences, efforts to secure additional funding,
including plans to seek partners for certain of Sangamo’s programs
and the discussions related thereto, 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 lack of capital resources to fully develop,
obtain regulatory approval for and commercialize its product
candidates and/or technologies, including the ability to secure the
funding or partnerships required to advance its preclinical and
clinical programs; Sangamo’s ability to execute its restructurings
as currently contemplated; Sangamo’s need for substantial
additional funding to execute its operating plan and to continue to
operate as a going concern, including the risk that Sangamo will be
unable to obtain the funding necessary to advance its preclinical
and clinical programs and to otherwise operate as a going concern,
in which case Sangamo may be required to cease operations entirely,
liquidate all or a portion of its assets and/or seek protection
under the U.S. Bankruptcy Code; the uncertain and costly research
and development process, including the risk that preclinical
results may not be indicative of results in any future clinical
trials; 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 its
collaborators, including the initiation and operation of clinical
trials; 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 registrational trial of Sangamo’s product candidates; the
potential for technological developments that obviate technologies
used by Sangamo; Sangamo’s reliance on collaborators and its
potential inability to secure additional collaborations, and
Sangamo’s ability to achieve expected future operating results.
All forward-looking statements about our future plans and
expectations, including our financial guidance, are subject to our
ability to secure adequate additional funding. There can be no
assurance that Sangamo and its 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 its collaborators. These risks and
uncertainties are described more fully in Sangamo’s Securities and
Exchange Commission, or SEC, filings and reports, including in
Sangamo’s Annual Report on Form 10-K for the year ended December
31, 2023, as supplemented by Sangamo’s Quarterly Report on Form
10-Q for the quarter ended March 31, 2024, and subsequent 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 Sangamo undertakes 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 depreciation and amortization, 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 depreciation and amortization, and
stock-based compensation expense because they are non-cash expenses
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 March 31,
2024
2023
Revenues
$
481
$
157,957
Operating expenses: Research and development
35,891
63,216
General and administrative
11,767
18,136
Impairment of long-lived assets
4,349
20,433
Impairment of goodwill
-
38,138
Total operating expenses
52,007
139,923
(Loss) income from operations
(51,526
)
18,034
Interest and other income, net
2,535
3,293
(Loss) income before income taxes
(48,991
)
21,327
Income tax expense
98
194
Net (loss) income
$
(49,089
)
$
21,133
Net (loss) income per share
-
-
Basic
$
(0.27
)
$
0.13
Diluted
$
(0.27
)
$
0.12
Shares used in computing net (loss) income per share
-
-
Basic
180,342
168,533
Diluted
180,342
169,181
Selected Balance Sheet Data: March
31, 2024 December 31, 2023 Cash and cash
equivalents
$
54,417
$
81,002
Total assets
$
128,961
$
165,320
Total stockholders' equity
$
56,697
$
82,887
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version on businesswire.com: https://www.businesswire.com/news/home/20240509815876/en/
Investor Relations & Media Inquiries Louise Wilkie
ir@sangamo.com media@sangamo.com
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