Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced
new 24-week data from patients treated with the 900 mg dose of
voxelotor in the ongoing HOPE-KIDS 1 Study, a Phase 2a open-label
study in adolescents ages 6 to 17 years with sickle cell disease
(SCD). Results demonstrated sustained and durable improvements in
hemoglobin levels and a reduction in clinical measures of hemolysis
with voxelotor in adolescents with SCD. The data will be presented
today at the 23rd European Hematology Association (EHA) Congress in
Stockholm.
“We continue to be encouraged by the results of the ongoing
HOPE-KIDS 1 study, which are consistent with inhibition of HbS
polymerization by voxelotor and support its ongoing clinical
evaluation as a potential disease-modifying therapy for both adults
and adolescents with SCD,” said Ted W. Love, M.D., president and
chief executive officer of GBT. “Results to date support our
ongoing development of voxelotor in a broad range of patients,
including in our Phase 3 HOPE Study, which is also evaluating
voxelotor at doses of 900 mg and 1500 mg per day in adolescents and
adults. We continue to expect to announce top-line clinical data
from Part A of the HOPE Study by the end of this quarter.”
Results from a Phase 2a Study (GBT440-007) Evaluating
Adolescents with Sickle Cell Disease Treated with Multiple Doses of
Voxelotor (GBT440), a HbS Polymerization Inhibitor
(abstract #PF709)The HOPE-KIDS 1 Study (GBT440-007), an open-label,
single- and multiple-dose study, is evaluating the safety,
tolerability, pharmacokinetics and exploratory treatment effect of
voxelotor in a pediatric population (ages 6 to 17) with SCD. Part A
of the study evaluated a single 600 mg dose of voxelotor while Part
B is exploring the safety of voxelotor at doses of 900 mg and 1500
mg per day administered to patients ages 12 to 17.
The EHA presentation analyzed 22 patients who received voxelotor
900 mg/day for 24 weeks in Part B. Data showed the following
results achieved with voxelotor treatment:
- Increased hemoglobin levels and improved clinical measures of
hemolysis at 24 weeks, as evaluated by changes from baseline in
hemoglobin, percent of reticulocytes, and percent of unconjugated
bilirubin.
- Specifically, 43 percent of patients (9 of 21) achieved a
hemoglobin response >1 g/dL at 24 weeks with a median hemoglobin
change from baseline of 0.7 g/dL.
- Reduced daily symptoms at 24 weeks as assessed by total symptom
scores (TSS), which improved in 13 of 21 patients.
- 55 percent of patients (11 of 20) had a numerical decrease in
transcranial doppler (TCD) flow at 24 weeks; among hemoglobin
responders (>1 g/dL), 88 percent (7 of 8) had a numerical
decrease in TCD at 24 weeks.
- Favorable tolerability profile in adolescents, consistent with
results in adults. Drug-related adverse events related to voxelotor
were grade 1 or 2, except one grade 3 urticaria that did not recur
with continued dosing. The most common drug-related adverse events
(occurring in two or more patients) were nausea (12 percent),
vomiting (8 percent), headache (8 percent) and rash (8 percent).
There were no drug-related discontinuations due to adverse
events.
Pharmacokinetics (PK) of Voxelotor (GBT440) Using
Population Pharmacokinetic (PPK)
and Physiologically Based Pharmacokinetic
(PBPK) Modeling in Pediatric Subjects with Sickle Cell
Disease (abstract #PF713)A separate poster will be
presented today at EHA summarizing the single-dose pharmacokinetics
(PK) of voxelotor in children and the multiple-dose PK of voxelotor
in adolescents. The analysis was based on data from approximately 6
children (ages 6 to 11) who have completed Part A of the HOPE-KIDS
1 study and 25 adolescents (ages 12 to 17) who have completed Part
B.
Results showed that voxelotor PK exposures in children following
a single oral 600 mg dose were higher than in adolescents or
adults, suggesting that lower doses (on a weight or surface area
basis) should be evaluated for future clinical trials in children.
Additionally, voxelotor PK exposures in adolescents following
multiple 900 mg doses were similar to those observed in adults,
suggesting similar doses may be administered in adolescents.
Further, physiologically-based PK modeling confirmed the need for
lower doses in children younger than age 12.
Three additional posters will be presented at EHA that support
GBT’s voxelotor SCD program, including one on the development,
translation and cultural adaptation of GBT’s Sickle Cell Disease
Severity Measure (SCDSM). This nine-item electronic patient
reported outcome (PRO) instrument was designed by GBT with guidance
from the U.S. Food and Drug Administration (FDA) to evaluate
changes in SCD symptom burden in the Phase 3
Hemoglobin Oxygen Affinity
Modulation to Inhibit Sickle Hemoglobin
PolymErization (HOPE) Study. Two
encore presentations – one on the novel design of the HOPE Study,
and another on findings from seven adult SCD patients with severe
anemia and multiple co-morbidities who received voxelotor through
compassionate access – also will be presented.
About Sickle Cell Disease (SCD)SCD is a
lifelong inherited blood disorder caused by a genetic mutation in
the beta-chain of hemoglobin, which leads to the formation of
abnormal hemoglobin known as sickle hemoglobin (HbS). In its
deoxygenated state, HbS has a propensity to polymerize, or bind
together, forming long, rigid rods within a red blood cell (RBC).
The polymer rods deform RBCs to assume a sickled shape and to
become inflexible, which can cause blockage in capillaries and
small blood vessels. Beginning in childhood, SCD patients suffer
unpredictable and recurrent episodes or crises of severe pain due
to blocked blood flow to organs, which often lead to psychosocial
and physical disabilities. This blocked blood flow, combined with
hemolytic anemia (the destruction of RBCs), can eventually lead to
multi-organ damage and early death.
About Voxelotor in Sickle Cell Disease
Voxelotor (previously called GBT440) is being developed as an oral,
once-daily therapy for patients with SCD. Voxelotor works by
increasing hemoglobin's affinity for oxygen. Since oxygenated
sickle hemoglobin does not polymerize, GBT believes voxelotor
blocks polymerization and the resultant sickling of red blood
cells. With the potential to restore normal hemoglobin function and
improve oxygen delivery, GBT believes that voxelotor may
potentially modify the course of SCD. In recognition of the
critical need for new SCD treatments, the U.S. Food and Drug
Administration (FDA) has granted voxelotor Breakthrough Therapy,
Fast Track, Orphan Drug and Rare Pediatric Disease designations for
the treatment of patients with SCD. The European Medicines Agency
(EMA) has included voxelotor in its Priority Medicines (PRIME)
program, and the European Commission (EC) has designated voxelotor
as an orphan medicinal product for the treatment of patients with
SCD.
GBT is currently evaluating voxelotor in the HOPE (Hemoglobin
Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a
Phase 3 clinical study in patients age 12 and older with SCD.
Additionally, voxelotor is being studied in the ongoing Phase 2a
HOPE-KIDS 1 Study, an open-label, single- and multiple-dose study
in pediatric patients (age 6 to 17) with SCD. HOPE-KIDS 1 is
assessing the safety, tolerability, pharmacokinetics and
exploratory treatment effect of voxelotor.
About GBTGBT is a clinical-stage
biopharmaceutical company determined to discover, develop and
deliver innovative treatments that provide hope to underserved
patient communities. GBT is developing its lead product candidate,
voxelotor, as an oral, once-daily therapy for sickle cell disease.
To learn more, please visit www.gbt.com and follow the company on
Twitter @GBT_news.
Forward-Looking Statements Statements we make
in this press release may include statements that are not
historical facts and are considered forward-looking within the
meaning of Section 27A of the Securities Act of 1933, as amended
and Section 21E of the Securities Exchange Act of 1934, as amended.
We intend these forward-looking statements, including statements
regarding the therapeutic potential and safety profile of voxelotor
including in pediatric and adolescent patients, our ability to
implement and complete our clinical development plans for
voxelotor, our ability to generate and report data from our ongoing
and potential future studies of voxelotor (including our ongoing
Phase 3 HOPE Study and our ongoing Phase 2a HOPE-KIDS 1 Study),
regulatory review and actions relating to voxelotor, and the timing
of these events, to be covered by the safe harbor provisions for
forward-looking statements contained in Section 27A of the
Securities Act and Section 21E of the Securities Exchange Act and
are making this statement for purposes of complying with those safe
harbor provisions. These forward-looking statements reflect our
current views about our plans, intentions, expectations, strategies
and prospects, which are based on the information currently
available to us and on assumptions we have made. We can give no
assurance that the plans, intentions, expectations or strategies
will be attained or achieved, and furthermore, actual results may
differ materially from those described in the forward-looking
statements and will be affected by a variety of risks and factors
that are beyond our control including, without limitation, the
risks that our clinical and preclinical development activities may
be delayed or terminated for a variety of reasons, that results of
clinical trials may be subject to differing interpretations, that
regulatory authorities may disagree with our clinical development
plans or require additional studies or data to support further
clinical investigation of our product candidates, that drug-related
adverse events may be observed in clinical development, and that
data and results may not meet regulatory requirements or otherwise
be sufficient for further development, regulatory review or
approval, along with those risks set forth in our Annual Report on
Form 10-K for the fiscal year ended December 31, 2017, and our
Quarterly Report on Form 10-Q for the quarter ended March 31, 2018,
as well as discussions of potential risks, uncertainties and other
important factors in our subsequent filings with the U.S.
Securities and Exchange Commission. Except as required by law, we
assume no obligation to update publicly any forward-looking
statements, whether as a result of new information, future events
or otherwise.
Contact Information: Myesha Lacy
(investors)GBT650-351-4730investor@gbt.com
Julie Normart (media)W2O pure415-946-1087media@gbt.com
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