Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine
company, today announced that hemophilia A gene therapy clinical
data and hemoglobinopathies ex vivo gene-edited cell therapy data
will be featured in poster presentations at the 61st Annual Meeting
of the American Society of Hematology (ASH). The ASH abstracts,
which were submitted on August 3, 2019, were released online this
morning. The conference will take place in Orlando, FL, from
December 7-10, 2019.
Gene Therapy
- Abstract #2060: “Updated Follow-up of the Alta Study, a Phase
1/2, Open Label, Adaptive, Dose-Ranging Study to Assess the Safety
and Tolerability of SB-525 Gene Therapy in Adult Patients with
Severe Hemophilia A” Presenter: Barbara Konkle, M.D., Bloodworks
Northwest, Professor of Medicine at University of Washington
December 7th, 2019, 5:30-7:30pm Eastern Time
The SB-525 poster will show updated Alta study data including
durability of Factor VIII (FVIII) levels, bleeding rate, factor
usage, and safety, for all five patients in the high dose cohort of
3e13 vg/kg, with approximately 4 months to 11 months of follow-up
after treatment with SB-525.
As of the abstract submission date, four patients in the 3e13
vg/kg cohort achieved FVIII levels within the normal range with no
bleeding events reported up to 24 weeks post-administration. These
patients did not require FVIII replacement therapy following the
initial prophylactic period of up to approximately 3 weeks
post-SB-525 administration. The fifth patient in the 3e13 vg/kg
cohort had only recently undergone treatment with SB-525 at the
time of the abstract submission. As previously reported, one
patient had treatment-related serious adverse events (SAEs) of
hypotension and fever, which occurred approximately 6 hours after
completion of the vector infusion and resolved with treatment
within 24 hours, with no loss of FVIII expression. SB-525 is being
developed as part of a global collaboration between Sangamo and
Pfizer.
“The rapid kinetics of Factor VIII expression, durability of
response, and the relatively low intra-cohort variability in the
context of a complete cessation of bleeding events and elimination
of exogenous Factor VIII usage continues to suggest SB-525 is a
differentiated hemophilia A gene therapy,” said Bettina Cockroft,
M.D., M.B.A., Chief Medical Officer of Sangamo, commenting on the
published abstract. “We are pleased with the progress of the
program toward a registrational Phase 3 study led by Pfizer, who
announced it has enrolled its first patient in the 6-month Phase 3
lead-in study. We have recently completed the manufacturing
technology transfer to Pfizer and initiated the transfer of the
IND.”
Ex Vivo Gene-Edited Cell Therapy
- Abstract #3544: “Preliminary Results of a Phase 1/2 Clinical
Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in
Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta
Thalassemia” Presenter: Angela Smith, MD, Associate Professor in
the Division of Pediatric Blood and Marrow Transplantation at the
University of Minnesota December 9th, 2019, 6:00-8:00pm Eastern
Time
- Abstract #974: “Zinc Finger Nuclease-Mediated Disruption of the
BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing,
Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells
Derived from Sickle Cell Disease Patients” Presenter: Samuel
Lessard, Ph.D., Scientist, Sanofi
The ST-400 beta thalassemia poster will show preliminary results
from the first three patients enrolled in the Phase 1/2 THALES
study. In this study, hematopoietic stem progenitor cells (HSPCs)
are apheresed from the patient, edited to knock out the erythroid
specific enhancer of the BCL11A gene, and cryopreserved prior to
infusion back into the patient following myeloablative conditioning
with busulfan. The first three patients all have severe beta
thalassemia genotypes: β0/β0, homozygous for the severe β+ IVS-I-5
(G>C) mutation, and β0/β+ genotype including the severe
IVS-II-654 (C>T) mutation, respectively.
As of the abstract submission date, Patient 1 and Patient 2 had
experienced prompt hematopoietic reconstitution. Patient 1 had
increasing fetal hemoglobin (HbF) fraction that contributed to a
stable total hemoglobin. After being free from packed red blood
cell (PRBC) transfusions for 6 weeks, the patient subsequently
required intermittent transfusions. Patient 2 had rising HbF levels
observed through 90 days post-infusion. For both patients, as of
the most recent follow-up reported in the abstract, on-target
insertions and deletions (indels) were present in circulating white
blood cells. Patient 3 had just completed ST-400 manufacturing at
the time of abstract submission. As previously disclosed, Patient 1
experienced an SAE of hypersensitivity during ST-400 infusion
considered by the investigator to be related to the product
cryoprotectant, DSMO, and which resolved by the end of the
infusion. No other SAEs related to ST-400 have been reported and
all other AEs have been consistent with myeloablation. No clonal
hematopoiesis has been observed. Longer follow-up will be required
to assess the clinical significance of these early results. ST-400
is being developed as part of a global collaboration between
Sangamo and Sanofi, along with support through a grant from the
California Institute for Regenerative Medicine (CIRM).
“The first three patients enrolled in the THALES study all have
severe beta thalassemia genotypes that result in almost no
endogenous beta globin production. The increases in fetal
hemoglobin and presence of on-target indels in circulating blood
cells suggests successful editing using zinc finger nucleases. The
results are preliminary and will require additional patients and
longer-term follow-up to assess their clinical significance,” said
Adrian Woolfson, BM., B.Ch., Ph.D., Head of Research and
Development. “It is important to note that myeloablative
hematopoietic stem cell transplantation reboots the hematopoietic
system, and that sufficient time is required for the stem cells to
fully repopulate the marrow and for new blood cells to form. In
other myeloablative conditioning studies in a similar patient
population, full manifestation of the effects of gene modification
in the red blood cell compartment has taken as long as 12 months or
more to become evident.”
Sanofi’s in vitro sickle cell disease poster details a similar
approach to ST-400, using mobilized HSPCs from normal donors and
SCD patients and utilizing the same zinc finger nuclease for gene
editing, delivered as transient non-viral RNA, and designed to
disrupt the erythroid specific enhancer of the BCL11A gene, which
represses the expression of the gamma globin genes, thereby
switching off HbF synthesis. Results from ex vivo studies
demonstrated enriched biallelic editing, increased HbF, and reduced
sickling in erythroid cells derived from non-treated sickle cell
disease patients. Sanofi has initiated a Phase 1/2 trial evaluating
BIVV003, an ex vivo gene-edited cell therapy using ZFN gene editing
technology to modify autologous hematopoietic stem cells using
fetal hemoglobin to produce functional red blood cells with higher
BhF content that are resistant to sickling in patients with severe
sickle cell disease. Recruitment is ongoing.
About the Alta study
The Phase 1/2 Alta study is an open-label, dose-ranging clinical
trial designed to assess the safety and tolerability of SB-525 gene
therapy in patients with severe hemophilia A. SB-525 was
administered to 11 patients in 4 cohorts of 2 patients each across
4 ascending doses (9e11 vg/kg, 2e12 vg/kg, 1e13vg/kg and 3e13vg/kg)
with expansion of the highest dose cohort by 3 additional patients.
The U.S. Food and Drug Administration (FDA) has granted Orphan
Drug, Fast Track, and regenerative medicine advanced therapy (RMAT)
designations to SB-525, which also received Orphan Medicinal
Product designation from the European Medicines Agency.
About the THALES study
The Phase 1/2 THALES study is a single-arm, multi-site study to
assess the safety, tolerability, and efficacy of ST-400 autologous
hematopoietic stem cell transplant in 6 patients with
transfusion-dependent beta thalassemia (TDT). ST-400 is
manufactured by ex vivo gene editing of a patient's own
(autologous) hematopoietic stem cells using non-viral delivery of
zinc finger nuclease technology. The THALES study inclusion
criteria include all patients with TDT (β0/β0 or non- β0/β0) who
have received at least 8 packed red blood cell transfusions per
year for the two years before enrollment in the study. The FDA has
granted Orphan Drug status to ST-400.
About Sangamo Therapeutics
Sangamo Therapeutics, Inc. is focused on translating
ground-breaking science into genomic medicines with the potential
to transform patients' lives using gene therapy, ex vivo
gene-edited cell therapy, in vivo genome editing, and gene
regulation. For more information about Sangamo, visit
www.sangamo.com.
Forward-Looking Statements
This press release contains forward-looking statements regarding
Sangamo's current expectations. These forward-looking statements
include, without limitation, statements regarding the Company's
ability to develop and commercialize product candidates to address
genetic diseases with the Company's proprietary technologies, as
well as the timing of commencement of clinical programs and the
anticipated benefits therefrom. These statements are not guarantees
of future performance and are subject to certain risks,
uncertainties and assumptions that are difficult to predict.
Factors that could cause actual results to differ include, but are
not limited to, the outcomes of clinical trials, the uncertain
regulatory approval process, uncertainties related to the execution
of clinical trials, Sangamo's reliance on partners and other
third-parties to meet their clinical and manufacturing obligations,
and the ability to maintain strategic partnerships. Further, there
can be no assurance that the necessary regulatory approvals will be
obtained or that Sangamo and its partners will be able to develop
commercially viable product candidates. Actual results may differ
from those projected in forward-looking statements due to risks and
uncertainties that exist in Sangamo's operations and business
environments. These risks and uncertainties are described more
fully in Sangamo's Annual Report on Form 10-K for the year ended
December 31, 2018 as filed with the Securities and Exchange
Commission and Sangamo's most recent Quarterly Report on Form 10-Q.
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.
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Investor Relations – Global McDavid
Stilwell 510-970-6000, x219 mstilwell@sangamo.com
Media Inquiries – Global Aron
Feingold 510-970-6000, x421 afeingold@sangamo.com
Investor Relations and Media Inquiries –
European Union & United Kingdom Caroline Courme 33 4 97
21 27 27 ccourme@sangamo.com
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