Conference call and webcast scheduled for
Tuesday, August 8, 4:30 p.m. Eastern Time.
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines
company, today reported recent business highlights and second
quarter 2023 financial results.
“I am extremely proud of our significant progress this quarter.
We are pleased to have executed several business development deals,
demonstrating the excitement in Sangamo’s science and platform,”
said Sandy Macrae, Chief Executive Officer of Sangamo. “We continue
to make progress in our clinical programs, with productive written
feedback received from the FDA on our Fabry Phase 3 strategy, and
initial approvals received on our amended TX200 protocol to
accelerate dose escalation. The impressive data we presented this
quarter on our preclinical neurology and AAV capsid delivery
capabilities reinforces our scientific capabilities, which continue
to lay the foundation for the company’s future.”
Recent Business Highlights
Corporate Updates
- Announced research evaluation and option agreement with Prevail
Therapeutics, a wholly owned subsidiary of Eli Lilly and Company,
for novel engineered cerebrospinal fluid-administered capsids with
enhanced nervous system delivery. We have granted Prevail rights to
evaluate our proprietary adeno-associated virus (AAV) capsids
developed through our AAV capsid engineering platform, SIFTER, in
exchange for an upfront payment. If Prevail exercises the option to
license the capsids, then we will become eligible to earn exercise
fees and potential development and commercial milestones of up to
$1.2 billion and tiered royalties on potential sales of products
using the capsids.
- Announced research evaluation and option agreement with Chroma
Medicine to explore zinc finger proteins (ZFPs) for epigenetic
editing. We have granted Chroma rights to evaluate our novel ZFPs
in specified targets outside of the central nervous system in
exchange for an upfront payment. If Chroma exercises the option to
license the ZFPs, then we will become eligible to earn exercise
fees, potential development and commercial milestone payments and
tiered royalties on potential sales of products using the
ZFPs.
Program Highlights
Fabry Disease – Dosed total of 22 patients in Phase 1/2
STAAR study; received Fast Track Designation from U.S. FDA;
received productive written FDA feedback on proposed Phase 3 trial
strategy; expect to submit proposed Phase 3 protocol to the FDA as
early as the end of 2023.
- Dosed 2 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 22 patients dosed to date.
- Received U.S. FDA Fast Track Designation for isaralgagene
civaparvovec, which aims to expedite the review of new therapeutics
that are intended to treat serious or life-threatening
conditions.
- Received productive written feedback from U.S. FDA on proposed
Phase 3 trial strategy that seeks to maximize the potential patient
population for ST-920 across two studies. Based on feedback
received, we do not expect a head-to-head comparison with enzyme
replacement therapy to be required in the naïve and pseudo-naïve
patient study. Requesting specific additional clarifications before
submitting a proposed Phase 3 protocol to the FDA, which we expect
to be as early as the end of 2023.
Renal Transplant Rejection – Safety Monitoring Committee
endorsed moving to the second dose level based on encouraging
safety data to date; successfully manufactured dose for the first
patient in the second dose cohort; received first approvals from
regulatory authorities for protocol amendment to accelerate dose
escalation.
- A total of six study sites across four countries are now open
and enrolling patients.
- Based on encouraging safety data, the Safety Monitoring
Committee endorsed moving to cohort 2 in 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 three patients dosed to date.
- Completed manufacturing of dose for the first patient in the
second cohort, who recently received a kidney transplant. Dosing of
this fourth patient is expected in the third quarter of 2023.
- Received first full country approval for amended protocol to
accelerate dose escalation.
- Intend to share initial data by the end of 2023.
Neurology Epigenetic Regulation Programs – Presented
first preclinical data evaluating zinc finger transcriptional
repressors targeting Nav1.7; presented updated animal model data in
prion disease; presented additional data describing advances being
made in AAV capsid delivery.
- Presented comprehensive preclinical data package evaluating
zinc finger repressors (ZF-Rs) targeting the Nav1.7 gene at the
American Society for Cell and Gene Therapy (ASGCT) 26th Annual
Meeting. The data demonstrated potent and specific repression of
Nav1.7 expression, without impacting other sodium channels, and
that the ZF-Rs are well tolerated in non-human primates. Continue
to expect an IND submission in 2024.
- Presented updated animal model data at ASGCT showing that our
ZF-Rs significantly reduce expression of the prion protein in the
brain, extend lifespan, and limit formation of toxic prion
aggregates. Continue to expect an IND submission in 2025.
- Entered into a license agreement with Voyager Therapeutics for
Voyager’s intravenous-administered capsid, which has been shown in
animal models to achieve the specific central nervous system
coverage we believe to be required for our prion disease
program.
- Continued to advance identification and selection of engineered
AAV capsids for enhanced central nervous system delivery, with
additional data presented at ASGCT describing the identification of
multiple novel AAV capsids exhibiting characteristics consistent
with enhanced blood brain barrier transit.
Hemophilia A (Pfizer) – Phase 3 AFFINE trial continues to
progress; pivotal data read-out expected in mid-2024; BLA and MAA
submissions anticipated in second half of 2024.
- 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, continues
to progress following the dosing of all patients required to
support primary analysis.
- A pivotal readout is expected in mid-2024, with Pfizer
anticipating BLA and MAA submissions in the second half of
2024.
Second Quarter 2023 Financial Results
Consolidated net loss for the second quarter ended June 30, 2023
was $114.5 million, or $0.66 per share, compared to a net loss of
$43.2 million, or $0.29 per share, for the same period in 2022.
This was primarily due to higher non-cash charges relating to
impairment of indefinite-lived intangible assets of $51.3 million,
which was a result of continued decline in our market
capitalization reflecting the decline in equity values in the
biotechnology industry, and a decrease in revenues of $22.5 million
as a result of termination of certain collaboration agreements.
Revenues
Revenues for the second quarter ended June 30, 2023 were $6.8
million, compared to $29.4 million for the same period in 2022.
The decrease of $22.5 million in revenues was primarily
attributed to decreases of $8.5 million, $7.8 million, and $1.8
million of revenues relating to our collaboration agreements with
Novartis, Biogen and Sanofi, respectively, due to the termination
of these collaboration agreements. Additionally, revenues relating
to our collaboration agreement with Kite decreased by $5.1 million,
reflecting a reduction in collaboration activities during the
quarter.
GAAP and Non-GAAP Operating Expenses
Three Months Ended Six Months Ended June 30,
June 30, (In millions)
2023
2022
2023
2022
Research and development
$
63.0
$
60.0
$
126.3
$
118.6
General and administrative
16.1
15.1
34.1
30.0
Impairment of goodwill and indefinite-lived intangible assets
51.3
-
89.5
-
Impairment of long-lived assets
-
-
20.4
-
Total operating expenses
130.4
75.1
270.3
148.6
Impairment of goodwill and indefinite-lived intangible assets
(51.3
)
-
(89.5
)
-
Impairment of long-lived assets
-
-
(20.4
)
-
Stock-based compensation expense
(6.8
)
(7.9
)
(15.1
)
(15.6
)
Non-GAAP operating expenses
$
72.3
$
67.2
$
145.3
$
133.0
Total operating expenses on a GAAP basis for the second quarter
ended June 30, 2023 were $130.4 million, compared to $75.1 million
for the same period in 2022. GAAP operating expenses for the second
quarter ended June 30, 2023 include a full write-down of
indefinite-lived intangible assets of $51.3 million, which was a
result of continued decline in our market capitalization reflecting
the decline in equity values in the biotechnology industry.
Non-GAAP operating expenses, which exclude impairment charges and
stock-based compensation expense, for the second quarter ended June
30, 2023 were $72.3 million, compared to $67.2 million for the same
period in 2022.
The increase in total operating expenses on a non-GAAP basis was
primarily attributable to higher compensation and other personnel
costs mainly due to restructuring-related charges, higher
facilities, infrastructure-related costs, and professional services
as we advance our preclinical and clinical pipeline. These
increases were partially offset by reimbursement of certain
research and development expenses by Sanofi under the termination
and transition agreement.
Cash, Cash Equivalents and Marketable Securities
Cash, cash equivalents and marketable securities as of June 30,
2023 were $182.1 million, compared to $307.5 million as of December
31, 2022. 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 June 30, 2023, in combination with
other potential cost reductions, will be sufficient to fund our
planned operations for at least the next 12 months.
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, are now estimated to be in the
range of approximately $378 million to $398 million, reflecting the
additional non-cash impairment charges. The previous GAAP operating
expenses guidance provided on April 26, 2023 was in the range of
approximately $315 million to $335 million.
- We continue to estimate non-GAAP operating expenses to remain
unchanged from the last update on April 26, 2023 and to be in the
range of approximately $240 million to $260 million. Estimated
non-GAAP operating expenses exclude estimated impairment of
goodwill of $38.1 million, impairment of indefinite-lived
intangible assets of $51.3 million, impairment of long-lived assets
of $20.4 million and stock-based compensation expense of $28
million.
Upcoming Events
Sangamo plans to participate in the following events:
Investor Conferences
- 2023 Wells Fargo Healthcare Conference, September 6-8,
2023
- Truist Securities BioPharma Symposium, November 8-9, 2023
- Jefferies London Healthcare Conference, November 14-16,
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 Second Quarter 2023
Results
The Sangamo management team will discuss these results on a
conference call today, Tuesday, August 8, 2023, at 4:30 p.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 potential future Phase 3 trials
of isaralgagene civaparvovec and the timing thereof, the
anticipated submission to the FDA of a proposed Phase 3 trial
protocol, the expectation that a head-to-head comparison with
existing enzyme replacement therapy will not be required in the
naïve and pseudo-naïve patient study, the timeline to present data
from the Phase 3 AFFINE trial and 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,
the potential for Chroma and Prevail to exercise their options and
for Sangamo to receive exercise fees, development and commercial
milestone payments and royalties under the Chroma and Prevail
agreements, our expected cash runway, potential cost reductions,
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 the effects of macroeconomic
factors or financial challenges, including as a result of the
ongoing conflict between Russia and Ukraine 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, including data from
kidney biopsies, 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; our limited experience manufacturing
biopharmaceutical products, including the risks that we may be
unable to maintain compliant manufacturing facilities, build
additional facilities and manufacture our product candidates as
intended; the potential for Chroma, Prevail or Voyager to breach or
terminate their agreements with Sangamo; the potential for Sangamo
to fail to realize its expected benefits from the Chroma, Prevail
or Voyager agreements; the potential for technological developments
that obviate technologies used by Sangamo; 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; 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 June 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 total operating expenses,
which excludes stock-based compensation expense and impairment of
goodwill, indefinite-lived intangible assets and long-lived assets
from GAAP total 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 Six months
ended June 30, June 30,
2023
2022
2023
2022
Revenues
$
6,835
$
29,378
$
164,792
$
57,609
Operating expenses: Research and development
63,046
60,019
126,262
118,603
General and administrative
16,014
15,093
34,150
30,001
Impairment of goodwill and indefinite-lived intangible assets
51,347
-
89,485
-
Impairment of long-lived assets
-
-
20,433
-
Total operating expenses
130,407
75,112
270,330
148,604
Loss from operations
(123,572
)
(45,734
)
(105,538
)
(90,995
)
Interest and other income, net
2,802
2,643
6,095
3,985
Loss before income taxes
(120,770
)
(43,091
)
(99,443
)
(87,010
)
Income tax (benefit) expense
(6,264
)
82
(6,070
)
140
Net loss
$
(114,506
)
$
(43,173
)
$
(93,373
)
$
(87,150
)
Basic and diluted net loss per share
$
(0.66
)
$
(0.29
)
$
(0.54
)
$
(0.59
)
Shares used in computing basic and diluted net loss per share
174,325
148,158
171,445
147,194
Selected Balance Sheet Data: June 30, 2023 December 31, 2022 Cash, cash
equivalents, and marketable securities
$
182,138
$
307,477
Total assets
$
325,074
$
562,509
Total stockholders' equity
$
233,642
$
294,958
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version on businesswire.com: https://www.businesswire.com/news/home/20230808479265/en/
Investor Relations & Media
Inquiries Louise Wilkie ir@sangamo.com media@sangamo.com
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