- The first two patients treated with the 3e13 vg/kg dose
achieved stable Factor VIII (FVIII) levels demonstrating durability
in the normal range through 44 and 37 weeks, respectively - All
five patients in the 3e13 vg/kg dose cohort achieved normal range
FVIII levels within 5-7 weeks following treatment, with no bleeding
events with up to 44 weeks of follow-up - Lower-dose cohorts
indicated durable FVIII activity with up to 52 weeks of follow-up -
The Companies have progressed SB-525 into a Phase III
registrational program led by Pfizer
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, and Pfizer, Inc. (NYSE: PFE), today announced updated
follow-up results from the Phase 1/2 Alta study evaluating
investigational SB-525 gene therapy in patients with severe
hemophilia A. The data showed that SB-525 was generally well
tolerated and demonstrated sustained increased Factor VIII (FVIII)
levels following treatment with SB-525 through to 44 weeks, the
extent of follow-up for the longest treated patient in the 3e13
vg/kg dose cohort. Data from 11 patients treated with SB-525 will
be featured in a poster presentation today, December 7, 2019, at
the 61st Annual Meeting of the American Society of Hematology (ASH)
in Orlando, FL. The SB-525 ASH poster, which includes the full set
of data, is available on Sangamo’s website in the Investors and
Media section under Events and Presentations.
This press release features multimedia. View
the full release here:
https://www.businesswire.com/news/home/20191207005019/en/
“I am pleased that all five patients in the high dose (3e13
vg/kg) cohort rapidly achieved normal levels of Factor VIII, and
that Factor VIII levels have been stable and durable in the normal
range for the first two patients up to 44 and 37 weeks following
treatment respectively, with no bleeding events or factor usage up
to a follow up of 44 weeks in the longest treated patient,” said
Barbara Konkle, M.D., Bloodworks Northwest, Professor of Medicine
at University of Washington and a Principal Investigator of the
Alta study. “It is important to continue to follow these patients
to determine whether these results are sustained in the longer term
as the combination of a favorable safety profile coupled with
sustained expression at a level that prevents bleeding and allows
normal activity will be the hallmark of a successful gene therapy
for hemophilia A.”
Alta study data presented at ASH included 11 patients treated
across four ascending dose cohorts: 9e11 vg/kg (2 patients), 2e12
vg/kg (2 patients), 1e13 vg/kg (2 patients) and 3e13 vg/kg (5
patients). The data cutoff date was October 17, 2019.
An analysis of plasma FVIII antigen was assessed by ELISA and
demonstrated antigen concentrations consistent with the FVIII
activity measured by the chromogenic assay. Dose dependent
increases in FVIII activity over baseline were observed across the
dose cohorts. The lower-dose cohorts indicate durable FVIII
activity with up to 52 weeks of follow-up.
In the 3e13 vg/kg dose cohort, patients achieved normal range
FVIII activity within 5-7 weeks of treatment with SB-525. The first
two patients treated in this cohort (Patients 7 and 8) have
achieved stable FVIII levels, demonstrating durability in the
normal range through 44 and 37 weeks, respectively, as measured by
the chromogenic assay. The two patients most recently treated in
this cohort (Patients 10 and 11), with 22 and 12 weeks of
follow-up, respectively, demonstrated a similar pattern of FVIII
expression. The FVIII expression pattern observed in Patient 9
differed from that of other patients in the cohort. Seven weeks
following treatment, Patient 9 achieved normal range FVIII levels.
Beginning at week 13, FVIII levels in that patient fluctuated in a
range below normal, but still well above the level needed to
prevent spontaneous bleeding. At week 18, FVIII levels in Patient 9
began to increase, and as of the latest measurement at week 24,
continued to rise. No patient in the 3e13 vg/kg dose cohort has
experienced bleeding events up to 44 weeks of follow-up, and no
patient in this dose cohort required factor replacement following
initial use of prophylactic factor.
SB-525 was generally well tolerated across all dose cohorts. The
treatment-related adverse events include: alanine aminotransferase
(ALT) elevation (36.4%, n=4), pyrexia (27.3%, n=3), increased
aspartate aminotransferase (18.2%, n=2), tachycardia (18.2% n=2),
fatigue (9.1%, n=1), hypotension (9.1%, n=1) and myalgia (9.1%,
n=1). Treatment-related serious adverse events (SAEs) of
hypotension (grade 3) and fever (grade two) occurred in one patient
in the 3e13 vg/kg cohort six hours following dosing with SB-525
that fully resolved within 24 hours. No similar events were
reported in the other patients dosed in that cohort. No patients
treated with SB-525 experienced an ALT elevation associated with
loss of Factor VIII expression. In the 3e13 vg/kg dose cohort, four
patients experienced transient low grade ALT elevations (>1.5 x
baseline) that were managed with a tapering course of oral
steroids. The study does not use corticosteroids prophylactically,
initiating them only in the event of an ALT elevation that is
greater than 1.5x baseline.
“The updated results from the Alta study suggest that SB-525 may
represent a differentiated gene therapy for patients with severe
hemophilia A,” said Bettina Cockroft, M.D., Chief Medical Officer
of Sangamo. “The results continue to suggest that if sustained over
a longer duration, SB-525 has the potential to be a predictable,
reliable, and safe treatment that may bring clinical benefits to
patients with severe hemophilia A.”
Sangamo has completed the manufacturing technology transfer and
initiated the transfer of the Investigational New Drug (IND)
Application to Pfizer, which is expected to be completed in the
first quarter 2020. Pfizer is enrolling patients in the Phase 3
lead-in study, the data from which is expected to provide a
baseline for patients who are subsequently enrolled into the Phase
3 study (ClinicalTrials.gov Identifier: NCT03587116).
“We are pleased with the progress that we have made in
progressing SB-525 gene therapy toward a Phase 3 registrational
study, including enrolling the first patient in the 6-month lead-in
study. We expect to dose the first patient in the Phase 3
registrational study in 2020,” said Seng Cheng, Senior Vice
President and Chief Scientific Officer of Pfizer’s Rare Diseases
Research Unit. “We continue to believe that if the observed safety
and efficacy results are sustained, this gene therapy has the
potential to transform the treatment paradigm of severe hemophilia
A.”
About the Alta study
The Phase 1/2 Alta study is an open-label, dose-ranging,
multicenter clinical trial designed to assess the safety and
tolerability of SB-525 in patients with severe hemophilia A. The
mean age of the 11 patients assessed is 30 years (range 18-47
years). All 11 patients are male. The U.S. Food and Drug
Administration 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. SB-525 is being developed as
part of a global collaboration between Sangamo and Pfizer.
About SB-525 Gene Therapy
SB-525 comprises a recombinant adeno-associated virus serotype 6
vector (AAV6) encoding the complementary deoxyribonucleic acid for
B domain deleted human FVIII. The SB-525 vector cassette was
designed to optimize both the vector manufacturing yield and
liver-specific FVIII protein expression. The SB-525 transcriptional
cassette incorporates multi-factorial modifications to the
liver-specific promoter module, FVIII transgene, synthetic
polyadenylation signal and vector backbone sequence.
About Sangamo Therapeutics
Sangamo Therapeutics is committed to translating ground-breaking
science into genomic medicines with the potential to transform
patients’ lives using gene therapy, ex vivo gene-edited cell
therapy, and in vivo genome editing and gene regulation. For more
information about Sangamo, visit www.sangamo.com.
Pfizer Inc: Working together for a healthier world®
At Pfizer, we apply science and our global resources to bring
therapies to people that extend and significantly improve their
lives. We strive to set the standard for quality, safety and value
in the discovery, development and manufacture of health care
products, including innovative medicines and vaccines. Every day,
Pfizer colleagues work across developed and emerging markets to
advance wellness, prevention, treatments and cures that challenge
the most feared diseases of our time. Consistent with our
responsibility as one of the world's premier innovative
biopharmaceutical companies, we collaborate with health care
providers, governments and local communities to support and expand
access to reliable, affordable health care around the world. For
more than 150 years, we have worked to make a difference for all
who rely on us. We routinely post information that may be important
to investors on our website at www.pfizer.com. In addition, to
learn more, please visit us on www.pfizer.com and follow us on
Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us
on Facebook at Facebook.com/Pfizer.
Sangamo Forward Looking Statements
This press release contains forward-looking statements regarding
Sangamo's current expectations. These forward-looking statements
include, without limitation, statements relating to the
investigational hemophilia A gene therapy, SB-525, including its
potential therapeutic benefits; the potential long-term durability
of SB-525 gene therapy; SB-525 having the potential to be a
predictable and reliable treatment that may bring clinical benefit
to patients with hemophilia A and to potentially represent a
transformative treatment paradigm; plans to advance SB-525 into a
potential registrational study; the potential benefits of the RMAT
and Orphan medicine designation for SB-525; and other statements
that are not historical fact. These statements are not guarantees
of future performance and are subject to risks and assumptions 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 research and development process; additional data,
including the risk that the data reported from the Alta to date may
not be indicative of the final results from the Alta study or that
such final results may not validate and support the safety and
efficacy of SB-525; the completion of the Alta study; the
possibility of unfavorable new clinical data from the Alta study
and further analyses of existing clinical data from the study that
may material change clinical outcomes; the risk that clinical trial
data are subject to differing interpretations and assessments by
regulatory authorities; whether regulatory authorities will be
satisfied with the design of and results from the clincal studies
relating to SB-525, any potential registrational studies or any
other clinical studies of SB-525; whether Sangamo will be able to
maintain or receive the benefits associated with RMAT, Orphan Drug,
Fast Track and Orphan Medicinal Product designations for SB-525;
Sangamo's reliance on Pfizer and other third-parties to meet their
clinical and manufacturing obligations; Sangamo’s ability to
maintain strategic partnerships; and the potential for
technological developments by Sangamo's competitors that will
obviate Sangamo's gene therapy technology. Further, there can be no
assurance that the necessary regulatory approvals will be obtained
for SB-525 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.
Pfizer Disclosure Notice: The information contained in this
release is as of December 7, 2019. Pfizer assumes no obligation to
update forward-looking statements contained in this release as the
result of new information or future events or developments.
This release contains forward-looking information about an
investigational hemophilia A agent, SB-525, including its potential
benefits, that involves substantial risks and uncertainties that
could cause actual results to differ materially from those
expressed or implied by such statements. Risks and uncertainties
include, among other things, the uncertainties inherent in research
and development, including the ability to meet anticipated clinical
endpoints, commencement and/or completion dates for our clinical
trials, regulatory submission dates, regulatory approval dates
and/or launch dates, as well as the possibility of unfavorable new
clinical data and further analyses of existing clinical data; risks
associated with interim data; the risk that clinical trial data are
subject to differing interpretations and assessments by regulatory
authorities; whether regulatory authorities will be satisfied with
the design of and results from our clinical studies; whether and
when drug applications for any potential indications for SB-525 may
be filed in any jurisdictions; whether and when regulatory
authorities in any jurisdictions may approve any such applications,
which will depend on myriad factors, including making a
determination as to whether the product's benefits outweigh its
known risks and determination of the product's efficacy and, if
approved, whether SB-525 will be commercially successful; decisions
by regulatory authorities impacting labeling, manufacturing
processes, safety and/or other matters that could affect the
availability or commercial potential of SB-525; and competitive
developments.
A further description of risks and uncertainties can be found in
Pfizer's Annual Report on Form 10-K for the fiscal year ended
December 31, 2018 and in its subsequent reports on Form 10-Q,
including in the sections thereof captioned "Risk Factors" and
"Forward-Looking Information and Factors That May Affect Future
Results", as well as in its subsequent reports on Form 8-K, all of
which are filed with the U.S. Securities and Exchange Commission
and available at www.sec.gov and www.pfizer.com.
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Sangamo
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
Pfizer
Media Contact: Steve Danehy +1 (978) 273-3946
Steve.Danehy@pfizer.com
Investor Contact: Bryan Dunn +1 (212) 733-8917
Bryan.Dunn@pfizer.com
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