[May 25, 2016] |
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Chugai's Bispecific Antibody "ACE910/Emicizumab" Phase l Data in Patients with Hemophilia A Published in The New England Journal of Medicine Online
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 (News - Alert) 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
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Outline of the study
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Dosing group
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Number of patients
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Dose
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Patients with
inhibitors
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Patients without
inhibitors
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Cohort 1
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4
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2
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1*, 0.3** mg/kg
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Cohort 2
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4
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2
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3*, 1** mg/kg
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Cohort 3
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3
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3
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3 mg/kg
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*Initial dose, **Second and subsequent doses
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Study results SAFETY All
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 BENEFIT A 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:
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Median ABR
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Dosing group
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Median ABR (events/year)
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Six months before
study entry
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During emicizumab
administration
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Cohort 1
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32.5
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4.4
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Cohort 2
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18.3
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0.0
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Cohort 3
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15.2
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0.0
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About Chugai Chugai 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20160525006575/en/
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