Announced global epigenetic regulation and
capsid delivery license agreement with Genentech to develop novel
genomic medicines for neurodegenerative diseases. Expect to receive
$50 million in near-term upfront license fees and milestone
payments and eligible to earn up to $1.9 billion in milestone
payments, plus tiered royalties on net sales.
Reported Pfizer’s announcement of positive
topline results from Phase 3 AFFINE trial evaluating giroctocogene
fitelparvovec, an investigational Hemophilia A gene therapy that
Sangamo is co-developing with and licensing to Pfizer.
Continue to amass encouraging safety and
efficacy data from the Phase 1/2 STAAR study of isaralgagene
civaparvovec for Fabry disease, including preliminary evidence of
improved kidney function and withdrawal from enzyme replacement
therapy (ERT) by seventeen of eighteen patients.
Announced data 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.
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, today reported business highlights and second quarter 2024
financial results.
“Sangamo has demonstrated important progress over the last few
months, with the announcement of a significant business development
agreement with Genentech earlier today; positive topline results
from the Phase 3 AFFINE trial in Hemophilia A we are developing
with Pfizer; and highly encouraging Phase 1/2 data continuing to
accumulate from our wholly-owned Fabry disease program – all of
which have the potential to meaningfully extend our cash runway as
we continue to advance our wholly owned neurology epigenetic
regulation pipeline,” said Sandy Macrae, Chief Executive Officer of
Sangamo. “Our license agreement with Genentech – the first in what
we hope could be multiple capsid collaborations to come with other
potential partners – reinforces the potential of our gene editing
and STAC-BBB capsid delivery platforms. Our core, and highly
focused, neurology pipeline continues to advance as planned and we
look forward to providing further updates.”
Recent Business Highlights
Corporate Updates
Genentech License Agreement
- Announced global epigenetic regulation and capsid delivery
license agreement with Genentech, a member of the Roche Group, to
develop novel genomic medicines for neurodegenerative
diseases.
- Granted an exclusive license to Genentech for Sangamo’s
proprietary zinc finger repressors that are directed to the tau
gene, which is critically involved in Alzheimer’s disease and other
tauopathies, as well as a second undisclosed neurology target.
Agreed to also exclusively license STAC-BBB to Genentech for tau
and the second neurology target.
- Expect to receive from Genentech $50.0 million in near-term
upfront license fees and milestone payments.
- Eligible to earn up to $1.9 billion in development and
commercial milestones spread across multiple potential products
under the agreement and tiered royalties on net sales of such
products, subject to certain specified reductions.
Clinical Programs
Hemophilia A
- Pfizer announced positive topline results from the Phase 3
AFFINE trial evaluating giroctocogene fitelparvovec, an
investigational gene therapy that Sangamo is co-developing with and
licensing to Pfizer for the treatment of adults with moderately
severe to severe hemophilia A.
- Sangamo is 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.
- Pfizer reported that the AFFINE trial achieved its primary
objective of non-inferiority, as well as superiority, of total
annualized bleeding rate (ABR) from Week 12 through at least 15
months of follow up post-infusion compared with routine Factor VIII
(FVIII) replacement prophylaxis treatment. Following a single 3e13
vg/kg dose, giroctocogene fitelparvovec demonstrated a
statistically significant reduction in mean total ABR compared to
the pre-infusion period.
- Key secondary endpoints as defined by the trial protocol were
met and also demonstrated superiority compared to prophylaxis. 84%
of participants maintained FVIII activity >5% at 15 months
post-infusion with the majority of participants having FVIII
activity ≥15%. The mean treated ABR showed a statistically
significant 98.3% reduction post-infusion.
- In the AFFINE trial, giroctocogene fitelparvovec was generally
well tolerated.
- Pfizer reported that analyses of the full Phase 3 dataset from
the AFFINE trial are ongoing and additional data will be presented
at upcoming medical meetings.
- Pfizer reported that it will discuss these data with regulatory
authorities in the coming months.
Fabry Disease
- Dosing is complete in the Phase 1/2 STAAR study of isaralgagene
civaparvovec, an investigational gene therapy for the treatment of
Fabry disease, with a total of 33 patients dosed.
- Three additional patients were withdrawn from ERT, resulting in
a total of 17 patients withdrawn from ERT to date. All 17 patients
remain off ERT as of August 6. The one remaining patient dosed
since February 2024 who began the study on ERT has plans in place
to withdraw ERT treatment at the appropriate time.
- With the longest-treated patient having achieved four years of
follow-up, Sangamo continues to amass encouraging clinical data,
including evidence of improvements in kidney function. In the 18
patients treated for more than one year, a statistically
significant rise in both mean and median eGFR levels was observed
in male and female treated patients, based on preliminary findings.
Anticipate sharing updated clinical data in the coming months.
- Held a productive meeting in June 2024 with the European
Medicines Agency (EMA) on proposed pathway to potential approval
for isaralgagene civaparvovec in Europe, with members of the U.S.
Food and Drug Administration (FDA) in attendance.
- Engaged in ongoing business development discussions with
potential collaborators for the Fabry disease program.
Prioritized Neurology Pipeline
Neurology Epigenetic Regulation Programs
- Investigational new drug (IND) enabling activities continue to
advance in the Nav1.7 program to treat chronic neuropathic
pain.
- Clinical trial authorization (CTA) enabling activities continue
to advance for Sangamo’s program to treat prion disease, leveraging
the novel STAC-BBB capsid.
- Showcased 10 poster presentations at the 27th ASGCT Annual
Meeting demonstrating advances in epigenetic regulation for the
treatment of various neurological diseases, including prion disease
and tauopathies.
- Engaged in ongoing business development discussions with
potential collaborators for zinc finger neurology epigenetic
regulation programs.
Novel Adeno-Associated Virus (AAV) Capsid Delivery
Technology
- Continued to advance work on STAC-BBB, our novel neurotropic
AAV capsid 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.
- Presented promising initial findings for a possible mechanism
supporting how STAC-BBB may cross the blood-brain barrier and
continued to successfully advance STAC-BBB manufacturing
activities.
- Engaged in ongoing business development discussions with new
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.
- Presented MINT platform developments in June 2024 at the
Federation of American Societies for Experimental Biology (FASEB),
Genome Engineering: Research and Applications Conference.
- 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.
Second Quarter 2024 Financial Results
Consolidated net loss for the second quarter ended June 30, 2024
was $36.1 million, or $0.18 per share, compared to a net loss of
$114.5 million, or $0.66 per share, for the same period in
2023.
Revenues
Revenues for the second quarter ended June 30, 2024 were $0.3
million, compared to $6.8 million for the same period in 2023.
The decrease of $6.5 million in revenues was primarily
attributed to decreases of $2.4 million and $2.2 million in
revenues relating to our prior collaboration agreements with Biogen
and Novartis, respectively, due to the termination of those
collaboration agreements in June 2023, a decrease of $1.2 million
in revenue relating to our collaboration agreement with Kite which
expired pursuant to its terms in April 2024, and a decrease of $0.5
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 Six Months Ended June 30,
June 30, (In millions)
2024
2023
2024
2023
Research and development
$
24.2
$
63.0
$
60.1
$
126.3
General and administrative
12.0
16.1
23.8
34.1
Impairment of long-lived assets
1.2
-
5.5
20.4
Impairment of goodwill and indefinite-lived intangible assets
-
51.3
-
89.5
Total operating expenses
37.4
130.4
89.4
270.3
Impairment of long-lived assets
(1.2
)
-
(5.5
)
(20.4
)
Impairment of goodwill and indefinite-lived intangible assets
-
(51.3
)
-
(89.5
)
Depreciation and amortization
(1.2
)
(4.2
)
(2.6
)
(7.7
)
Stock-based compensation expense
(3.1
)
(6.8
)
(5.8
)
(15.1
)
Non-GAAP operating expenses
$
31.9
$
68.1
$
75.5
$
137.6
Total operating expenses on a GAAP basis for the second quarter
ended June 30, 2024 were $37.4 million compared to $130.4 million
for the same period in 2023. Non-GAAP operating expenses, which
exclude impairment charges, depreciation and amortization, and
stock-based compensation expense as shown in the reconciliation
table above, for the second quarter ended June 30, 2024 were $31.9
million, compared to $68.1 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 lower headcount as a result of restructuring of
operations and a corresponding reductions in workforce announced
during 2023, a decrease due to restructuring expenses recorded in
the second quarter of 2023 related to a reduction in workforce
announced in April 2023, a decrease in preclinical and clinical
expenses due to termination of collaboration agreements, and
deferral and reprioritization of certain research and development
programs, a decrease in external professional services, and a
decrease in facilities and infrastructure related expenses. These
decreases were partially offset by an increase relating to a
terminated manufacturing-related supplier arrangement for costs
that will be incurred without economic benefit to Sangamo.
Cash and Cash Equivalents
Cash and cash equivalents as of June 30, 2024 were $27.8
million, compared to cash, cash equivalents and marketable
securities of $81.0 million as of December 31, 2023. On August 2,
2024, we entered into a global epigenetic regulation and capsid
delivery license agreement with Genentech, a member of the Roche
Group, under which we expect to receive an aggregate of $50.0
million in near-term upfront license fees and milestone payments.
We believe that our available cash and cash equivalents as of June
30, 2024, in combination with the $50.0 million in expected
near-term license fees and milestone payments from Genentech, will
be sufficient to fund our planned operations into the first quarter
of 2025.
Financial Guidance for 2024
- On a GAAP basis, we expect total operating expenses in the
range of approximately $150 million to $170 million in 2024, which
includes non-cash stock-based compensation expense, impairment
expense, and depreciation and amortization.
- We expect non-GAAP total operating expenses, excluding
estimated non-cash stock-based compensation expense of
approximately $13 million, impairment expense of approximately $6
million, and depreciation and amortization of approximately $7
million, in the range of approximately $125 million to $145 million
in 2024.
Upcoming Events
Sangamo plans to participate in the following events:
- Wells Fargo Healthcare Conference, September 4-6, 2024
- H.C. Wainwright 26th Annual Global Investment Conference,
September 9-11, 2024
Access links for available webcasts for 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
Tuesday, August 6, 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 impact of business
development and clinical advancements on Sangamo’s cash runway and
ability to continue to operate as a going concern, the therapeutic
and commercial potential of Sangamo’s product candidates, including
the durability of therapeutic effects, the therapeutic and
commercial potential of technologies used by Sangamo in its product
candidates, including its gene therapy and cell therapy
technologies and , zinc finger platform, the potential of its
adeno-associated virus capsid delivery platform and its novel
next-generation Modular Integrase (MINT) platform, Sangamo’s
ability to realize the expected benefits of the license agreement
with Genentech, including but not limited to the receipt and timing
of the upfront license fee and Sangamo’s completion of the
requisite technology transfer in order to receive an expected
near-term milestone payment, the potential for Genentech to
complete clinical development, regulatory interactions,
manufacturing and global commercialization of any resulting
products, the potential for Sangamo to receive development and
commercial milestone payments and royalties, Sangamo’s ability to
establish and maintain collaborations and strategic partnerships
and realize the expected benefits of such arrangements, including
its ability to find a collaboration partner for its Fabry disease
gene therapy program and additional collaborations with respect to
Sangamo’s STAC-BBB capsid delivery platform and epigenetic
regulation capabilities, and Pfizer’s continued advancements of the
giroctocogene fitelparvovec program, including the potential for
Pfizer to complete clinical development, regulatory interactions,
manufacturing and global commercialization of any resulting
products, anticipated revenues from existing and new collaborations
and the timing thereof, 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, the timeline to
present data from the Phase 3 AFFINE trial and Pfizer’s discussions
of data with applicable regulatory authorities, advancement of
Sangamo’s preclinical neurology programs, including announcement of
data from, and anticipated IND and CTA submissions. the potential
of the MINT platform to enable large-scale genomic engineering,
Sangamo’s estimates regarding the sufficiency of its cash resources
and its expenses, capital requirements and need for substantial
additional financing, 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, Sangamo’s efforts and ability 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 the potential for Genentech to
breach or terminate its agreement with Sangamo; and the potential
for Sangamo to fail to realize its expected benefits from the
Genentech agreement, including but not limited to further
validating the importance of the zinc finger platform to support
the development of therapeutics for neurodegenerative diseases;
Sangamo’s lack of capital resources and 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 funding or partnerships or additional
collaboration partners 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; Sangamo’s ability to execute its
restructurings as currently contemplated; 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
conflicts, current or potential future bank failures, inflation and
high 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 or that Sangamo will earn any
milestone or royalty payments under the Genentech agreement or
obtain regulatory approvals for product candidates arising from
this agreement. 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 Reports on Form 10-Q for the quarters ended
March 31, 2024 and June 30, 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
Six months ended June 30,
June 30,
2024
2023
2024
2023
Revenues
$
356
$
6,835
$
837
$
164,792
Operating expenses: Research and development
24,223
63,046
60,114
126,262
General and administrative
12,045
16,014
23,812
34,150
Impairment of long-lived assets
1,172
-
5,521
20,433
Impairment of goodwill and indefinite-lived intangible assets
-
51,347
-
89,485
Total operating expenses
37,440
130,407
89,447
270,330
Loss from operations
(37,084
)
(123,572
)
(88,610
)
(105,538
)
Interest and other income, net
1,030
2,802
3,565
6,095
Loss before income taxes
(36,054
)
(120,770
)
(85,045
)
(99,443
)
Income tax expense (benefit)
74
(6,264
)
172
(6,070
)
Net loss
$
(36,128
)
$
(114,506
)
$
(85,217
)
$
(93,373
)
Basic and diluted net loss per share
$
(0.18
)
$
(0.66
)
$
(0.44
)
$
(0.54
)
Shares used in computing basic and diluted net loss per share
203,946
174,325
194,049
171,445
Selected Balance Sheet Data:
June 30, 2024 December 31, 2023 Cash and cash
equivalents
$
27,786
$
81,002
Total assets
$
93,014
$
165,320
Total stockholders' equity
$
23,690
$
82,887
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240806551275/en/
Investor Relations & Media Inquiries Louise Wilkie
ir@sangamo.com media@sangamo.com
Sangamo Therapeutics (NASDAQ:SGMO)
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