Filed・Approved
As of Mar. 31, 2017
Approved in SG
Filed in HK, MY
◎ARQ 197
Tivantinib
Oral
c-Met Inhibitor
Hepatocellular Cancer
Phase III
in JP
Licensed from
ArQule
Phase Iin JP
Phase IIin US
Phase II in US
Phase II in JP and
KR
Updated since Jan 24th,
2017 （Area, Stage, Filed, Approved, etc.）
◎
R&D Pipeline
Aplastic Anemia
PhaseII/III
in JP and KR
Phase II, Phase III
Central
Nervous
System
KW-6002
Istradefylline
Oral
Adenosine A
2A
Receptor Antagonist
Parkinson’s Disease
Phase III
in US, CA, EU and
others
Phase IIin JP
Phase II
in US, EU and
others
KHK4827
Brodalumab
Injection
Anti-IL-17 Receptor
A Fully Human
Antibody
Psoriasis
Phase III in KR
Nephrology
Area
In-House
Human Antibody-Producing Technology
Jointly Developed with
Ultragenyx in US and EU
X-linked
Hypophosphatemia (XLH)
in pediatric patients
Phase III in US, CA,
EU, AU, JP, KR
Tumor Induced
Osteomalacia(TIO)/Epider
mal Nevus Syndrome
(ENS)
Kirin-Amgen
◎ASKP1240
Bleselumab
Injection
Anti-CD40 Fully
Human Antibody
Organ Transplant
Rejection
In-House
Human Antibody-Producing Technology
Jointly Developed with Astellas
◎KRN23
burosumab
Injection
Anti-FGF23 Fully
Human Antibody
X-linked
Hypophosphatemia(XLH)
in adult patients
Phase III in US, CA,
EU, JP and KR
In-House
◎ＫＷ-6356
Oral
Adenosine A
2A
Receptor Antagonist
Parkinson’s Disease
In-House
Oncology
Jointly Developed with
AstraZeneca/MedImmune
Chronic Obstructive
Pulmonary Disease(COPD)
Phase III in JP
Eosinophilic Chronic
Rhinosinusitis (ECRS)
Phase II in JP
In-House
POTELLIGENT

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Human Antibody-Producing Technology
◎KHK4563
Benralizumab
Injection
Anti-IL-5 Receptor
Humanized Antibody
Asthma
Phase III in JP and
KR
In-House
POTELLIGENT

Immunology
/Allergy
◎KHK4083
Injection
Anti-OX40 Fully
Human Antibody
Ulcerative colitis
KW-0761
Mogamulizumab
Injection
Anti-CCR4
Humanized Antibody
Adult T-cell
Leukemia/Lymphoma
Ph
ase
II
in US, EU and
others
In-House
POTELLIGENT

Cutaneous T-cell
Lymphoma
Phase III
in US, EU, JP and
others
In-House
or
Licensed
Remarks
Licensed from
Reata
◎KHK7580
Evocalcet
Oral
Calcium Receptor
Agonist
Secondary
Hyperparathyroidism
PhaseIII
in JP
Li
cense
d
f
rom
Mitsubishi
Tanabe
Pharma
◎RTA 402
Bardoxolone Methyl
Oral
Antioxidant
Inflammation
Modulator
CKD in Patients with Type
2 Diabetes
PhaseII
in JP
Code Name
Generic Name
Formulation
Mechanism of Action
Indication
Stage
Kirin-Amgen
◎KRN23
burosumab
Injection
Anti-FGF23 Fully
Human Antibody
X-linked
Hypophosphatemia (XLH)
Filed in EU
In-House
Human Antibody-Producing
Technology
Jointly Developed with Ultragenyx
in US and EU
Filed in BN
Licensed from
NPS
Other
AMG531
Romiplostim
Injection
Thrombopoietin
Receptor Agonist
Idiopathic (Immune)
Thrombocytopenic
Purpura
Approved in TH
Licensed from
Solasia Pharma
Z-206
Mesalazine
Oral
pH Dependent
Controlled
Release Tablet
Ulcerative Colitis
(Additional Dosage and
Administration)
Filed in JP
Licensed from
Zeria Pharma
Jointly Developed with Zeria
Pharma
NDA holder is Zeria Pharma
Oncology
Granisetron
Patch
５-HT
3
Serotonin
Receptor Antagonist
Chemotherapy induced
Nausea and Vomiting
Filed in MY
Area
Code Name
Generic Name
Formulation
Mechanism of Action
Indication
Myelodysplastic Syndromes
Kirin-Amgen
KRN321
Darbepoetin Alfa
Injection
Long-Acting
Erythropoiesis
Stimulating Agent
Stage
In-House
or
Licensed
Remarks
Nephrology
Renal Anemia (on Dialysis)
NDA in preparation
in CN
Renal Anemia
Filed in ID
KRN1493
Cinacalcet
Hydrochloride
Oral
Calcium Receptor
Agonist
Secondary
Hyperparathyroidism
Immunology
/Allergy
AMG531
Romiplostim
Injection
Thrombopoietin
Receptor Agonist
Kirin-Amgen
Idiopathic (Immune)
Thrombocytopenic
Purpura
Phase III in CN
Other
New Molecular Entity
Asthma
Filed in JP
In-House
POTELLIGENT

Jointly Developed with
AstraZeneca/MedImmune
NDA holder is AstraZeneca
KHK4827
Brodalumab
Injection
Anti-IL-17 Receptor
A Fully Human
Antibody
Psoriasis
Filed in TW
Kirin-Amgen
◎KHK4563
Benralizumab
Injection
Anti-IL-5 Receptor
Humanized Antibody
antibody
protein
small molecule
Ｐ
h
I
As of Mar.31, 2017
Combination with
Durvalumab/Tremelimumab
(Jointly Developed with AstraZeneca)
Combination with Docetaxel
Combination with
PF-05082566
(Jointly Developed with Pfizer)
Phase I/II
in US
Combination with Nivolumab
(Jointly Developed with Bristol-Myers
Squibb)
PhaseI
in JP
Combination with Nivolumab
(Jointly Developed with
Ono Pharmaceutical)
◎KHK4083
Injection
Anti-OX40 Fully
Human Antibody
Ulcerative colitis
Phase Iin JP
In-House
◎KHK4083
Injection
Anti-OX40 Fully
Human Antibody
Ulcerative colitis
Phase Iin JP
In-House
PhaseIin EU
PhaseIin JP
Updated since Jan 24th,
2017 （Area, Stage, Filed, Approved, etc.）
◎
Updated since Jan 24th,
2017 （Area, Stage, Filed, Approved, etc.）
Filed・Approved
Nephrology
KRN321
Darbepoetin Alfa
Injection
Long-Acting
Erythropoiesis
Stimulating Agent
Myelodysplastic Syndromes Approved in SG Kirin-Amgen
R&D Pipeline
Other
Kirin-Amgen
In-House
or
Licensed
Remarks
Area
Code Name
Generic Name
Formulation
Mechanism of Action
Indication
Stage
KHK4827
Brodalumab
Injection
Anti-IL-17 Receptor
A Fully Human
Antibody
Psoriasis
Filed in TW
AMG531
Romiplostim
Injection
Thrombopoietin
Receptor Agonist
Idiopathic (Immune)
Thrombocytopenic
Purpura
Approved in TH
Immunology
/Allergy
In-House
POTELLIGENT

Jointly Developed with
AstraZeneca/MedImmune
NDA holder is AstraZeneca
◎KHK4563
Benralizumab
Injection
Anti-IL-5 Receptor
Humanized Antibody
Asthma
Filed in JP
Kirin-Amgen
In-House
Others
KW-3357
Antithrombin
Gamma
Injection
Recombinant Human
Antithrombin
Disseminated Intravascular
Coagulation, Congenital
Antithrombin Deficiency
Phase Iin EU
Licensed from
Immunas
Pharma
Immunology/
Allergy
POTELLIGENT

Human Antibody-Producing Technology
POTELLIGENT

Human Antibody-Producing Technology
PhaseI
in US
Remarks
POTELLIGENT

Human Antibody-Producing Technology
KW-0761
Mogamulizumab
Injection
Anti-CCR4
Humanized Antibody
Solid Tumor
Phase I
in US
In-House
POTELLIGENT

◎KHK2823
Injection
Anti-CD123 Fully
Human Antibody
Cancer
Phase I
in UK
In-House
In-House
Combination with KW-0761
PhaseI
in JP
Syndax
領域
Code Name
Generic Name
Formulation
Mechanism of Action
Indication
Stage
In-House
or
Licensed
◎KHK2455
Oral
IDO 1 Inhibitor
Solid Tumor
New Molecular Entity
Oncology
◎KHK2375
Entinostat
Oral
HDAC Inhibitor
Breast Cancer
Central
Nervous
System
◎KHK6640
Injection
Anti–Amyloid Beta
Peptide Antibody
Alzheimer’s Disease
antibody
protein
small molecule

Alder Biopharmaceuticals has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Alder Biopharmaceuticals, 2017, APR 27, 2017, View Source [SID1234521703]).

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On April 17, 2017 PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, reported that
the European Medicines Agency (EMA) has granted orphan drug designation to PIQUR’s lead compound PQR309 for the treatment of patients with diffuse large B-cell lymphoma (DLBCL) (Press release, PIQUR Therapeutics, APR 27, 2017, View Source content/uploads/2017/04/PIQUR_MediaRelease_EMA_OrphanDrug_EN_20170427.pdf [SID1234527271]).

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"The EMA orphan drug designation for PQR309 in DLBCL is another important regulatory milestone, validating the potential therapeutic use of PQR309 in DLBCL," said Claudia Pluess, Senior Regulatory Affairs Manager at PIQUR. Dr. Vladimir Cmiljanovic, CEO of PIQUR, added, "PIQUR will continue to work with physicians and regulatory agencies to further define the clinical development strategy to bring a potential new treatment option to patients suffering
from this disease."

DLBCL is an aggressive form of lymphoma, and the most common type of non-Hodgkin lymphoma (NHL), accounting for about 30 percent of all NHL cases [1]. The disease occurs primarily in older individuals, though it can also occur in children and young adults in rare cases. 10 to 15 percent of DLBCL patients exhibit refractory disease and an additional 20 to 25 percent relapse after initial response to therapy [2].

In addition to this orphan drug designation by the EMA in DLBCL, PIQUR has also recently received orphan drug designation from the FDA for PQR309 for the treatment of primary CNS lymphoma (PCNSL).

The EMA orphan drug designation is a status assigned to a medicine intended for use against a rare condition (prevalence of the condition in the European Union must not be more than 5 in 10,000) and allows a pharmaceutical company to benefit from incentives offered by the EU to develop a medicine for the treatment, prevention or diagnosis of a disease that is life threatening or a chronically debilitating rare disease.

About PQR309
PIQUR’s lead compound, PQR309, is an oral, brain-penetrant, dual inhibitor of the PI3K/mTOR pathway, which is activated in 60 – 80% of human cancers. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use to malignant diseases involving the brain. PQR309 has shown both preclinical activity in various tumor models and clinical activity in Phase 1 and 2 studies.

PQR309 is currently being investigated in several Phase 1 and 2 clinical studies in advanced solid tumors (NCT02483858), relapsed or refractory lymphoma (NCT02249429), relapsed or refractory PCNSL (NCT02669511) and progressive glioblastoma multiforme (NCT02850744). In addition, the PIQHASSO Phase 1/2b study investigates PQR309 in combination with Eisai’s Eribulin in metastatic HER2-negative and triple-negative breast cancer (NCT02723877). Additional information regarding the PQR309 clinical trials is available on
www.clinicaltrials.gov.

On April 27, 2017 Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, reported the appointment of Roger Waltzman, M.D., M.B.A. as its Chief Medical Officer. Dr. Waltzman brings more than 20 years of experience in oncology across the entire new drug development and approval process, including serving in various executive roles at Novartis for nearly a decade (Press release, Rgenix, APR 27, 2017, View Source [SID1234523089]).

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In his role, he will oversee clinical development at Rgenix, including clinical trial design and the new drug application process with regulators. He will also support Rgenix’s goals of providing a meaningful response for cancer patients lacking effective therapies by overseeing the patient experience.

"Dr. Waltzman has an extraordinary background in oncology, which is our focus at Rgenix, and his expertise will be crucial as we progress with the clinical development of our lead therapy, RGX-104, and our other drug candidates in development," said Masoud Tavazoie, M.D., Ph.D., Chief Executive Officer and co-founder of Rgenix. "We look forward to his contributions to our work at Rgenix as we develop novel treatments for patients who suffer from cancer types with a high, unmet need."

"Rgenix has innovation at its core, and I’m pleased to be joining an organization that is looking to fill a gap in oncology that could benefit hundreds of thousands of patients. I look forward to collaborating with the Rgenix team on its therapies and clinical developments as it looks to drive positive change in the industry," said Dr. Waltzman.

Prior to Rgenix, Dr. Waltzman worked at Novartis in both Medical Affairs and Clinical Development roles. In oncology, he successfully filed an sNDA as the Clinical Development Head for Tasigna/Glivec and led the successful development and MAA/NDA filing of Jakavi/Jakafi, in collaboration with Incyte. His last position at Novartis was as Global Development Head for anti-malarials, where he continued Phase 2 development of two novel anti-malarials and facilitated a cost-saving Collaboration Agreement with the Bill and Melinda Gates Foundation and Medicines for Malaria Venture.

His most recent position was with Jaguar Animal Health and Napo Pharmaceuticals in San Francisco where he was the Chief Scientific Officer and Chief Medical Officer, leading the development of a first-in-class, FDA-approved botanical as a novel anti-diarrheal.

Dr. Waltzman received his medical degree from Brown University School of Medicine and his MBA from Columbia Business School. He completed his residency in Internal Medicine at Harvard’s Beth Israel Hospital, and his fellowship in hematology/oncology at Memorial Sloan-Kettering Cancer Center. He has been a member of the board of directors of GoDocGo and the Brown Medical Alumni Association since 2015.

MediciNova has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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