BRISBANE, Calif. and
NEW YORK, April 2, 2019 /PRNewswire/
-- Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic
medicine company, and Pfizer, Inc. (NYSE: PFE) today announced
interim data from the Phase 1/2 Alta study evaluating
investigational SB-525 gene therapy for severe hemophilia A. Data
indicate that SB-525 was generally well-tolerated and demonstrated
a dose-dependent increase in Factor VIII (FVIII) levels across the
four dosage cohorts. Eight patients total were dosed. Based on
these results, the Safety Monitoring Committee (SMC) recommended
cohort expansion at the 3e13 vg/kg dose. Further details will be
disclosed during Sangamo's conference call and webcast scheduled
for 8:00 a.m. ET today, which can be
accessed on the Sangamo website.
"The interim data from the first eight patients with hemophilia
A treated with SB-525 gene therapy in the Alta study are
encouraging and demonstrate a dose-dependent relationship, evidence
of sustained factor levels, and low variability, both within each
patient and within each cohort," said Edward Conner, MD, Chief Medical Officer of
Sangamo. "These interim results suggest that SB-525 may be
well-tolerated and may prove to have the predictability and
sustained treatment effect that can bring clinical benefit in
patients with hemophilia A. We need to continue observing how the
data mature and how additional patients in the expansion cohort
respond to SB-525. We look forward to working with Pfizer to
potentially advance SB-525 into a registrational study."
The Phase 1/2 interim data include eight patients treated across
four ascending dosage cohorts (9e11 vg/kg, 2e12 vg/kg, 1e13 vg/kg
and 3e13 vg/kg, with two patients per cohort). Patients
demonstrated a dose-dependent increase in FVIII levels, achieving
clinically relevant increases in FVIII activity in the higher
dosage cohorts and normal FVIII levels in the 3e13 vg/kg dose
cohort (normal range: 50-150%). At week 6 post infusion, the two
fourth dose cohort patients reached 140% and 94% of normal (as
measured by one-stage clotting assay) and 93% and 65% (as measured
by chromogenic assay). A dose-dependent reduction in the use of
Factor VIII replacement therapy was also observed, with patients in
the highest dose cohort not requiring factor replacement therapy
after initial use of prophylactic factor and experiencing no
bleeding events to date. SB-525 was generally well-tolerated, with
one patient (treated with the 3e13 vg/kg dose) reporting a
treatment-related serious adverse event of hypotension and fever,
which occurred following vector infusion and resolved with
treatment within 24 hours of completion of vector
infusion.
"The interim results with SB-525 gene therapy for patients with
severe hemophilia A are early but very promising," said
Barbara Konkle, MD, Bloodworks
Northwest and Professor of Medicine at University of Washington and investigator of the
Alta study. "It will be important to observe additional patients
and for a longer follow-up duration to determine whether these
positive interim findings are recapitulated and sustained."
Patients in the Alta study were not treated with prophylactic
steroids. No treatment-related serious adverse events and no ALT
elevations requiring more than seven days of corticosteroid
treatment were observed in the first three cohorts. One patient in
the fourth cohort experienced an ALT elevation (>1.5x ULN) at
week four that required a tapering course of oral steroids. The
patient did not have any associated loss of Factor VIII activity or
ALT elevations seven weeks following initiation of the steroid
therapy (five weeks post vector infusion). The same
patient in the fourth cohort experienced the aforementioned serious
adverse event.
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.
"We are encouraged by the early clinical data suggesting
tolerability of the recombinant AAV6 vector and potential for
normalization of Factor VIII levels. We look forward to the
opportunity to expanding the cohort administered a 3e13 vg/kg dose
and subsequent planning for the pivotal study," said Seng Cheng, SVP and Chief Scientific Officer of
Pfizer's Rare Diseases Research Unit. "As the development and
commercial partner for SB-525, we are encouraged by the possibility
that SB-525 may one day transform the treatment landscape for
patients with hemophilia A."
Longer-term follow-up data will be presented at an upcoming
scientific meeting. Per the SMC recommendation and study protocol,
the fourth cohort will be expanded by up to five patients. Patient
enrollment is underway.
In addition to the partnership for the development and
commercialization of gene therapies for hemophilia A, Sangamo and
Pfizer are also collaborating on the development of gene therapies
for amyotrophic lateral sclerosis (ALS) and frontotemporal lobar
degeneration (FTLD) using Sangamo's proprietary zinc finger protein
transcription-factor technology.
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 in up to 20 adult patients with
severe hemophilia A. The U.S. Food and Drug Administration has
granted Orphan Drug and Fast Track 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 Sangamo Therapeutics
Sangamo
Therapeutics, Inc. is focused on translating ground-breaking
science into genomic medicines with the potential to transform
patients' lives. Our capabilities in gene therapy, cell therapy,
genome editing, and gene regulation allow us to apply the
appropriate therapeutic approach to the underlying genetic cause of
the disease. 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. Our global portfolio includes
medicines and vaccines as well as many of the world's best-known
consumer health care products. 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.
Forward-Looking Statements
Pfizer Disclosure Notice
The information contained in this release is as of
April 2, 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.
Sangamo Disclosure Notice
This press release contains forward-looking statements based
on Sangamo's current expectations. These forward-looking statements
include, without limitation, statements relating to an
investigational hemophilia A gene therapy, SB-525, including its
potential benefits, plans for the ongoing development of SB-525 and
plans to present additional data, and plans to collaborate with
Pfizer on other product candidates, as well as other
statements that are not historical fact. 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, risks and uncertainties related to uncertainties
inherent in research and development, including risks
associated with interim data; the possibility of
unfavorable new clinical data and further analyses of
existing clinical 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 SB-525'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; competitive developments; and
Sangamo's reliance on partners and other third-parties to further
develop product candidates. These risks and uncertainties and other
risks 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. Forward-looking
statements contained in this press release 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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SOURCE Sangamo Therapeutics, Inc.