Chugai's Emicizumab Showed Continued Benefits in Patients with Hemophilia A
－Update from Japanese P I/II study presented at WFH 2016－
Chugai Pharmaceutical Co., Ltd. (TOKYO:4519) announced today that latest data from an ongoing Japanese Phase
I/II study (ACE002JP) of emicizumab was presented at the World Federation of Hemophilia 2016 World Congress in Orlando, Florida,
United States. Emicizumab is a bispecific antibody for subcutaneous injection under development for hemophilia A.
ACE002JP is an extension study of the patient part of a Phase I study (ACE001JP), which was conducted to investigate safety and
exploratory prophylactic efficacy profiles of emicizumab in Japanese hemophilia A patients both with and without FVIII inhibitors.
The latest data analysis continued to show a promising profile of once-weekly subcutaneous injection of emicizumab in terms of
safety and prophylactic efficacy, regardless of the presence of factor VIII inhibitors. The mean follow-up period were 32.6, 27.0
and 21.4 months in the 0.3, 1 and 3 mg/kg groups respectively.
“This new long-term follow-up data supports emicizumab’s continued prophylactic efficacy in hemophilia A patients both with and
without inhibitors,” said Chugai’s Vice President, Head of Project & Lifecycle Management Unit, Dr. Yasushi Ito. “We anticipate
that emicizumab will bring innovation to the current treatment for hemophilia A and deliver significant value to patients and their
families.”
Emicizumab was designated as a Breakthrough Therapy by the US Food and Drug Administration in September 2015. Currently, two
Phase III global studies in adolescents/adults and children with hemophilia A who acquired FVIII inhibitors are ongoing
respectively in collaboration with Roche, Chugai’s strategic alliance partner. A Phase III global study in patients without FVIII
inhibitors is also planned to start later this year. Furthermore, a Phase III global study investigating administration once every
four weeks is also planned.
The data of the patient part of ACE001JP was published in The New England Journal of Medicine in May 2016.
http://www.nejm.org/doi/full/10.1056/NEJMoa1511769
| |
|
[Outline of the study]
|
| Cohort |
|
|
Number of patients |
|
|
Dose |
|
|
Patients with inhibitors |
|
|
Patients without inhibitors |
|
|
| C-1 |
|
|
4 |
|
|
2 |
|
|
1*, 0.3** mg/kg |
| C-2 |
|
|
4 i) |
|
|
2 |
|
|
3*, 1** mg/kg |
| C-3 |
|
|
3 |
|
|
3 ii) |
|
|
3 mg/kg |
|
*Initial dose, **Second and subsequent doses
|
|
i) One patient discontinued emicizumab due to injection site erythema of mild intensity during Phase
I study
|
|
ii) One patient did not transit to extension study since prior treatment was sufficiently
efficacious.
|
| |
[Study results]
SAFETY
- 18 patients all experienced adverse events. Those adverse events were of mild or moderate intensity,
except for 2 severe cases (appendicitis and mesenteric hematoma).
- No thromboembolic adverse events or clinically significant abnormality of coagulation tests were
observed.
- 7 of 18 patients experienced local injection site reactions which were manageable.
- No neutralizing ADA (anti-drug antibodies) were observed.
EFFICACY
- ABR (Annualized Bleeding Rate) remained low and 8 of 18 patients had experienced zero bleeds.
- Breakthrough bleeds were successfully treated with standard episodic treatment, FVIII products or
bypassing agents.
- The median ABR and AJBR (Annualized Joint Bleeding Rate) at pre- and post-administration of
emicizumab in each cohort are as follows:
| |
| Cohort |
|
|
Median ABR
(times)
|
|
|
Median AJBR
(times)
|
|
|
Median [range]
follow-up (months)
|
|
|
Pre-emicizumab |
|
|
Post-emicizumab |
|
|
Pre-emicizumab |
|
|
Post-emicizumab |
|
|
|
C-1
(N=6)
|
|
|
32.5 |
|
|
1.4 |
|
|
27.4 |
|
|
1.1 |
|
|
32.6
[32.2-33.3]
|
|
C-2
(N=6)
|
|
|
18.3 |
|
|
0.2 |
|
|
15.2 |
|
|
0.2 |
|
|
27.0
[8.2-28.5]
|
|
C-3
(N=6)
|
|
|
15.2 |
|
|
0.0 |
|
|
9.1 |
|
|
0.0 |
|
|
21.4
[11.1-22.5]
|
| |
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 totaled 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.
For Media
Chugai Pharmaceutical Co., Ltd.
Media Relations Group, Corporate Communications Dept.,
Koki Harada
Tel: +81-3-3273-0881
E-mail: pr@chugai-pharm.co.jp
***
For US media
Chugai Pharma USA Inc.
Casey Astringer
Tel: +1-908-516-1350
E-mail: pr@chugai-pharm.com
***
For European media
Chugai Pharma France SAS
Nathalie Leroy
Tel: +33-1-56-37-05-21
E-mail: pr@chugai.eu
***
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Chugai Pharma Taiwan Ltd.
Susan Chou, Osamu Kagawa
Tel: +886-2-2715-2000
E-mail: pr@chugai.com.tw
***
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Investor Relations Group, Corporate Communications Dept.,
Toshiya Sasai
Tel: +81-3-3273-0554
E-mail: ir@chugai-pharm.co.jp
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