-
SEG101 reduced annual rate of
sickle cell-related pain crises (SCPC) by 45.3% compared to placebo
in patients with or without hydroxyurea therapy
-
SEG101 is a potential new
disease-modifying, preventive treatment option for patients with
SCPC; first in nearly 20 years
-
Data being highlighted in ASH
2016 media briefing, presented at Plenary Scientific Session and
published simultaneously in The New England Journal of
Medicine
Basel, December 3, 2016 -
Results from the Phase II SUSTAIN study show that SEG101
(crizanlizumab, formerly SelG1), an anti-P-selectin antibody,
reduced the median annual rate of sickle cell-related pain crises
(SCPC) by 45.3% compared to placebo (1.63 vs 2.98, p=0.010) in
patients with or without hydroxyurea therapy[1]. Novartis today
announced that the data are being featured in the official press
briefing at the 58th American
Society of Hematology (ASH) Annual Meeting and presented during the
Plenary Scientific Session tomorrow (Abstract #1, 2:00 - 4:00 p.m.
PST). The results also are being published simultaneously in
The New England Journal of Medicine.
"Acute painful episodes, commonly referred to
as vaso-occlusive crises, are a substantial cause of morbidity
in sickle cell disease with limited treatment options," said
Kenneth I. Ataga, M.D., Division of Hematology/Oncology, University
of North Carolina, Chapel Hill. "These findings show that
crizanlizumab significantly reduces the frequency of painful
crises and represents a potentially novel disease-modifying
therapeutic option."
In the SUSTAIN study, patients were assigned to
high-dose (5.0 mg/kg), low-dose (2.5 mg/kg) and placebo arms. The
study met its primary endpoint, reduction of the annual rate of
SCPC in the high-dose arm by 45.3% vs. placebo (medians of 1.63 vs.
2.98, p=0.010). In the low- dose arm, the annual rate of SCPC was
reduced by 32.6% vs. placebo (medians of 2.01 vs. 3.0, p = 0.180).
For patients in the high dose arm, time to first SCPC vs. placebo
was 2.9 times longer (medians of 4.07 vs. 1.38 months, p = 0.001)
and time to second SCPC was 2.0 times longer than placebo (medians
of 10.32 vs. 5.09 months, p = 0.022)[1].
"Patients have long been in need of a new therapy
for treatment of SCPC, the most common and debilitating
complication of sickle cell disease," said Bruno Strigini, CEO of
Novartis Oncology. "We are pleased that data from the SUSTAIN study
show SEG101 may have the potential to become the first new option
for patients dealing with SCPC since hydroxyurea was approved for
use in sickle cell anemia about 20 years ago[2]."
Despite its availability, hydroxyurea often is not
utilized primarily due to concerns about patient compliance and
potential adverse events[3],[4].
About the SUSTAIN
trial
The SUSTAIN trial was a multicenter, multinational, randomized,
placebo-controlled, double-blind, 12-month study to assess safety
and efficacy of the anti-P-selectin antibody SEG101 with or without
hydroxyurea therapy in sickle cell disease patients with sickle
cell-related pain crises. Patients included in the study had a
history of 2 to 10 pain crises in the previous 12 months. Patients
receiving hydroxyurea or erythropoietin were included if prescribed
for the preceding 6 months and dose was stable for at least 3
months. The trial randomized 198 patients age 16 to 65 to receive
high dose SEG101, low dose SEG101 or placebo[1].
Adverse events that occurred in 5% or more of
patients in an active dose group and were elevated over placebo by
at least 2-fold were arthralgia, pruritus, vomiting, chest pain,
diarrhea, road traffic accident, fatigue, myalgia, musculoskeletal
chest pain, abdominal pain, influenza and oropharyngeal pain. There
were no apparent increases in infections with SeG101 treatment.
Five deaths occurred during the study, 2 at 5.0 mg/kg, 1 at 2.5
mg/kg and 2 in placebo; no deaths were deemed related to the study
drug[1].
About SEG101 (crizanlizumab)
SEG101 (crizanlizumab, formerly SelG1) is a humanized
anti-P-selectin monoclonal antibody that binds a molecule called
P-selectin on the surface of endothelial cells and platelets in the
blood vessels, causing a blockade of P-selectin[1],[5]. P-selectin
drives the vaso-occlusive process[1],[6]. Vaso-occlusive
crises, also known as SCPC, occur episodically when sickle-shaped
red blood cells block blood flow through blood vessels[7]. The
therapeutic blockade of P-selectin can prevent painful
vaso-occlusion in small blood vessels and maintain blood
flow[1],[7].
Disclaimer
The foregoing release contains forward-looking statements that can
be identified by words such as "potential," "potentially," "may,"
or similar terms, or by express or implied discussions regarding
potential marketing approvals for SEG101, or regarding potential
future revenues from SEG101. You should not place undue reliance on
these statements. Such forward-looking statements are based on the
current beliefs and expectations of management regarding future
events, and are subject to significant known and unknown risks and
uncertainties. Should one or more of these risks or uncertainties
materialize, or should underlying assumptions prove incorrect,
actual results may vary materially from those set forth in the
forward-looking statements. There can be no guarantee that SEG101
will be submitted or approved for sale in any market, or at any
particular time. Nor can there be any guarantee that SEG101 will be
commercially successful in the future. In particular, management's
expectations regarding SEG101 could be affected by, among other
things, the uncertainties inherent in research and development,
including unexpected clinical trial results and additional analysis
of existing clinical data; unexpected regulatory actions or delays
or government regulation generally; the company's ability to obtain
or maintain proprietary intellectual property protection; general
economic and industry conditions; competition in general; global
trends toward health care cost containment, including ongoing
pricing pressures; unexpected manufacturing, safety or quality
issues, and other risks and factors referred to in Novartis AG's
current Form 20-F on file with the US Securities and Exchange
Commission. Novartis is providing the information in this press
release as of this date and does not undertake any obligation to
update any forward-looking statements contained in this press
release as a result of new information, future events or
otherwise.
About Novartis
Novartis provides innovative healthcare solutions that address the
evolving needs of patients and societies. Headquartered in Basel,
Switzerland, Novartis offers a diversified portfolio to best meet
these needs: innovative medicines, eye care and cost-saving generic
pharmaceuticals. Novartis is the only global company with leading
positions in these areas. In 2015, the Group achieved net sales of
USD 49.4 billion, while R&D throughout the Group amounted to
approximately USD 8.9 billion (USD 8.7 billion excluding impairment
and amortization charges). Novartis Group companies employ
approximately 118,000 full-time-equivalent associates. Novartis
products are available in more than 180 countries around the world.
For more information, please visit http://www.novartis.com.
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References
[1] Ataga KI, et al. SUSTAIN: A Multicenter,
Randomized, Placebo-Controlled, Double-Blind, 12-Month Study to
Assess Safety and Efficacy of SelG1 with or without Hydroxyurea
Therapy in Sickle Cell Disease Patients with Sickle Cell-Related
Pain Crises. Abstract #1. 2016 58th American
Society of Hematology (ASH) Annual Meeting, San Diego,
California.
[2] Segal JB, Strouse JJ, et al. Evidence Reports/Technology
Assessments, No. 165. Rockville, MD: Agency for Healthcare
Research and Quality (US); 2008 Feb.
[3] Zumberg MS, Reddy S, et al. Hydroxyurea therapy for sickle cell
disease in community-based practices: a survey of Florida and North
Carolina hematologists/oncologists. Am J Hematol. 2005
Jun;79(2):107-113.
[4] Miller ST, Kim HY, et al. for Sickle Cell Disease Clinical
Research Network (SCDCRN). Inpatient management of sickle cell
pain: A 'snapshot' of current practice. Am J Hematol. 2012
Mar;87(3):333-336.
[5] Novartis Pharmaceuticals Corporation. Data on file.
2016.
[6] Manwani D. Frenette PS. Vaso-occlusion in sickle cell disease:
pathophysiology and novel targeted therapies. Blood. 2013;
122(24):3892-3898.
[7] Quinn CT. Anti-adhesive therapy for sickle cell disease. The
Hematologist. 2014; 11(6):15.
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