Chugai's Bispecific Antibody "ACE910/Emicizumab" Phase l Data in Patients with Hemophilia A Published in The New England Jour...
May 25 2016 - 10:30PM
Business Wire
-- First NEJM Publication of Chugai’s Drug
Candidate --
Chugai Pharmaceutical Co., Ltd. (TOKYO:4519) announced today
that the data from hemophilia A patients who participated in a
Phase I study of the bispecific antibody emicizumab (ACE001JP
study) was published online in The New England Journal of Medicine
(NEJM) on May 25, 2016 (EST). Emicizumab is currently under
development for hemophilia A. In this first-in-patient Phase I
study, a once-weekly subcutaneous injection of emicizumab
demonstrated a clinically acceptable safety profile and a potential
benefit for preventing bleeding in hemophilia A patients, both with
and without factor VIII (FVIII)
inhibitors.http://www.nejm.org/doi/full/10.1056/NEJMoa1511769
The patient part of ACE001JP study enrolled 18 Japanese
hemophilia A patients, both with and without FVIII inhibitors, to
investigate the safety and to explore the potential benefit of
emicizumab for preventing bleeding with regular injection. In this
open-label study, patients were treated with once-weekly
subcutaneous injection of emicizumab across three dosing cohorts
for 12 successive weeks. The study results had been presented at
the 56th American Society of Hematology (ASH) Annual Meeting held
in San Francisco, CA on December 8, 2014.
“A great challenge with the current treatments for hemophilia A
is that these patients need frequent intravenous injections and
face the possibility of developing inhibitors to FVIII,” said
Chugai’s Director and Executive Vice President, Dr. Yutaka Tanaka.
“The study results indicated a potential for benefit from
emicizumab in treating hemophilia A patients with inhibitors with
once-weekly subcutaneous injection. With these findings, we expect
that emicizumab may be a new treatment option to fulfill unmet
medical needs of hemophilia A patients.”
Emicizumab was designated as a Breakthrough Therapy by the US
Food and Drug Administration in September 2015. Currently, a Phase
III global study in hemophilia A patients with FVIII inhibitors is
being conducted in collaboration with Roche, Chugai’s strategic
alliance partner. Phase III global studies in patients without
FVIII inhibitors and pediatric patients are also planned for
initiation in 2016.
The data of the healthy subject parts of ACE001JP study was
published in Blood online, the journal published by ASH, on
December 1, 2015.http://dx.doi.org/10.1182/blood-2015-06-650226
Outline of the
study
Dosing group
Number of patients
Dose
Patients with
inhibitors
Patients without
inhibitors
Cohort 1 4 2 1*, 0.3** mg/kg Cohort 2 4 2 3*, 1** mg/kg Cohort 3
3 3
3 mg/kg
*Initial dose, **Second and subsequent
doses
Study resultsSAFETYAll
adverse effects (AEs) observed during the 12-week course of
emicizumab administration were of mild intensity, except for upper
respiratory tract infection and headache of moderate intensity in 2
patients. There was no evidence of clinically relevant coagulation
abnormalities as indicated by clinical findings or laboratory
tests. No thromboembolic AEs were observed, even when a FVIII
product or bypassing agent as hemostatic therapy for bleeding
events was given during the course of emicizumab administration.
One patient discontinued emicizumab administration due to injection
site erythema of mild intensity. No anti-emicizumab antibodies
developed during the 12 weeks.
POTENTIAL BENEFITA potential benefit for the prevention of
bleeding with emicizumab was demonstrated during the 12-week course
of administration in all cohorts irrespective of the presence of
inhibitors. There was no bleeding in 13 patients during the course
of emicizumab administration. The median ABRs (annualized bleeding
rates) before and during emicizumab administration in each cohort
were as follows:
Median ABR
Dosing group Median ABR (events/year) Six
months before
study entry
During emicizumab
administration
Cohort 1 32.5 4.4 Cohort 2 18.3 0.0 Cohort 3
15.2 0.0
About ChugaiChugai Pharmaceutical is one of Japan’s
leading research-based pharmaceutical companies with strengths in
biotechnology products. Chugai, based in Tokyo, specializes in
prescription pharmaceuticals and is listed on the 1st section of
the Tokyo Stock Exchange. As an important member of the Roche
Group, Chugai is actively involved in R&D activities in Japan
and abroad. Specifically, Chugai is working to develop innovative
products which may satisfy the unmet medical needs, mainly focusing
on the oncology area.In Japan, Chugai’s research facilities in
Gotemba and Kamakura are collaborating to develop new
pharmaceuticals and laboratories in Ukima are conducting research
for technology development for industrial production. Overseas,
Chugai Pharmabody Research based in Singapore is engaged in
research focusing on the generation of novel antibody drugs by
utilizing Chugai’s proprietary innovative antibody engineering
technologies. Chugai Pharma USA and Chugai Pharma Europe are
engaged in clinical development activities in the United States and
Europe.The consolidated revenue in 2015 of Chugai totalled 498.8
billion yen and the operating income was 90.7 billion yen (IFRS
Core basis).Additional information is available on the internet at
http://www.chugai-pharm.co.jp/english.
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For MediaChugai Pharmaceutical Co.,
Ltd.Media Relations Group, Corporate Communications Dept.,Koki
HaradaTel: +81-3-3273-0881E-mail:
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