Business

Areas of Disease and Pipeline

Areas of disease expected as possible indications for regeneration-inducing medicine

Because regeneration-inducing medicine does not act directly on the affected part of the disease, but instead acts on stem cells present in areas such as the bone marrow or blood, it may overcome many issues associated with conventional regenerative medicine/cell therapy, and can target treatment for diseases that have been difficult to treat with conventional therapies. If our regeneration-inducing medicine is put into practice, it is expected to develop a broad range of clinical applications.

Overview of development pipeline

Our research and development pipeline is show below, together with the current state of progress. The pipeline is divided into the following five projects (PJ1 to PJ5). The main target markets for each pipeline are Japan, the US and Europe.

Development
code
Details Indication Organization
in charge
of development
Development stage Out-license
contract partner
Exploration Non-
clinical
Phase I
study
Phase II
study
Phase III
study
PJ1HMGB1
peptides
-01 HMGB1 bone marrow mesenchymal stem cell mobilization domain peptides Epidermolysis
bullosa
Osaka University
Planned to receive a marketing approval after the end of phase II Shionogi & Co., Ltd.
(S-005151)
-02 same as above Cerebral infarction Shionogi & Co., Ltd.
 
-03 same as above Cardiomyopathy (ischemic cardiomyopathy/dilated cardiomyopathy) Osaka University
Phase II study
in preparation
 
-04 same as above Osteoarthritis of the knee Hirosaki University
 
-05 same as above Chronic liver disease Niigata University
 
PJ2 -01 RIM3 Ulcerative colitis
Atopic dermatitis
In-house
(partnership is planned)
     
-02 Regeneration-inducing medicine novel peptides A Multiple tissue
damage diseases
In-house
(partnership is planned)
     
-03 Regeneration-inducing medicine novel peptides B Multiple tissue
damage diseases
In-house
(partnership is planned)
     
PJ3 -01 Biologic regeneration-inducing proteins Multiple tissue
damage diseases
In-house
(partnership is planned)
     
PJ4 -01 Autologous cell sampling devices for medical treatment Intractable ulcerative osteochondral disease In-house
(partnership is planned)
ND
PJ5 -01 Stem cell gene therapy Epidermolysis
bullosa
In-house
(partnership is planned)
Phase I/II study non

In the case of PJ1 -01, the number of target patients with epidermolysis bullosa dystrophica, is around 200 in Japan, with about 15 new cases expected each year, and therefore it is not feasible to plan a large-scale phase III study. In addition, epidermolysis bullosa dystrophica is a rare intractable disease, and there is currently no effective therapy. Accordingly, we expect to apply for a marketing approval for the drug based on the results of the phase II study.

In the case of PJ4 -01, we are currently coordinating development plans, and do not currently expect to conduct studies after phase I, but as no final decision has been made, this is marked ND.