On November 1, 2016 Shire plc (Shire) (LSE: SHP, NASDAQ: SHPG) reported unaudited results for the three months ended September 30, 2016 (Press release, Shire, NOV 1, 2016, View Source [SID1234516161]).

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Financial Highlights Q3 2016(1) Growth(1) Non GAAP CER(1)(2)
Product sales $3,315 million +110% +111%
Product sales excluding Baxalta products $1,769 million +12% +13%
Total revenues $3,452 million +109% +109%
US GAAP operating loss from continuing operations ($406 million) (189%)
Non GAAP operating income(2) $1,254 million +73% +71%
US GAAP net income margin(3)(4) (11%) (38ppc)
Non GAAP EBITDA margin(2)(4) 38% (5ppc)
US GAAP net loss ($387 million) (185%)
Non GAAP net income(2) $962 million +50%
US GAAP diluted losses per ADS ($1.29) (156%)
Non GAAP diluted earnings per ADS(2) $3.17 (2%) (3%)
US GAAP net cash provided by operating activities $526 million (6%)
Non GAAP cash generation(2) $830 million +41%
Non GAAP free cash flow(2) $395 million (27%)

(1)Results include Baxalta Inc. (Baxalta) (acquired on June 3, 2016) and Dyax Corp. (Dyax) (acquired on January 22, 2016), unless otherwise noted. Percentages compare to equivalent 2015 period.
(2)The Non GAAP financial measures included within this release are explained on pages 27 – 28, and are reconciled to the most directly comparable financial measures prepared in accordance with US GAAP on pages 21 – 23.
(3)US GAAP net income as a percentage of total revenues.
(4) Percentage point change ("ppc").

Financial highlights

Very strong launch results for XIIDRA; 64,732 scripts written through October 21, with a market share of 16%.
Product sales growth of 110% in Q3 2016 to $3.3 billion, driven by record legacy Shire product sales and the inclusion of legacy Baxalta franchises.
Underlying growth in Q3 2016 hematology and immunology businesses largely in line with overall market trends; pro forma sales growth of legacy Baxalta products impacted by the timing of large orders.
Q3 2016 year to date growth for legacy Shire product sales was 15% (16% on a Non GAAP CER basis) and legacy Baxalta was 7% on a pro forma basis (8% on a Non GAAP CER pro forma basis), in line with our expectations.
Baxalta integration

Operating expense synergy initiatives ahead of schedule, including manufacturing footprint optimization review.
Sharp focus on transitioning legacy Baxalta products onto Shire’s commercial platform and operating execution.
Issued $12.1 billion aggregate principal debt at a weighted average interest rate of 2.6%; net proceeds used to fully repay bridge facility for financing Baxalta transaction.
Pipeline progress

Pipeline continuing to deliver with U.S. Food and Drug Administration (FDA) approval of CUVITRU, and European Commission (EC) Marketing Authorization for ONIVYDE accompanied by Orphan Drug Designation.
Fully enrolled Phase 3 study for SHP643 in prophylaxis of hereditary angioedema with results expected in the first half of 2017.
FDA resubmission of SHP465 for the treatment of ADHD on track to be made in Q4 2016.
Flemming Ornskov, M.D., M.P.H., Shire Chief Executive Officer, commented:

"During the third quarter, we made rapid progress integrating our new company while delivering record quarterly product sales growth and remaining on track to meet our full year Non GAAP guidance. The launch of XIIDRA is off to a very strong start, and we are using this momentum to build a leadership position in pharmaceutical ophthalmics. Commercial execution remains a top priority across the business. Also, our robust pipeline is continuing to advance, and we look forward to highlighting key programs during our upcoming Investor Day. The changes we are applying to the legacy Baxalta business are similar to the One Shire initiative we undertook in 2013-2014, which set off a period of strong growth and profitability. I am highly confident about Shire’s future growth prospects."

On November 1, 2016 Synthetic Biologics, Inc. (NYSE MKT: SYN), a late-stage clinical company developing therapeutics focused on the gut microbiome, reported an operational update and reported financial results for the three months ended September 30, 2016 (Press release, Synthetic Biologics, NOV 1, 2016, View Source [SID1234516162]).

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Synthetic Biologics, Inc. www.syntheticbiologics.com (PRNewsFoto/Synthetic Biologics, Inc.)
"Clinical progress for our two lead gut microbiome-focused drug candidates represent critical milestones for the company as we continue our evolution from an early-stage development company to a late-stage clinical development company focused on commercialization," said Jeffrey Riley, President and Chief Executive Officer of Synthetic Biologics. "During the third quarter, we completed enrollment in our Phase 2b proof-of-concept clinical trial to evaluate the ability of ribaxamase to protect the gut microbiome from the effects of certain commonly used intravenous beta-lactam antibiotics for the prevention of C. difficile infection (CDI), antibiotic-associated diarrhea (AAD) and the emergence of antibiotic-resistant organisms. We were also the only commercial company pursuing drug development to receive a government contract from the Centers for Disease Control and Prevention to investigate antimicrobial resistance. This grant will support our clinical research aimed at determining whether ribaxamase may prevent antibiotic-mediated microbial resistance in the gut microbiomes of participants in our Phase 2b study. We look forward to sharing top-line results from this trial during the first quarter of 2017."

Mr. Riley continued, "SYN-010, our therapeutic designed to reduce methane in the gut and treat the underlying cause of irritable bowel syndrome with constipation (IBS-C), continues its rapid clinical progress. We held a held an End of Phase 2 meeting with the FDA to determine the optimal clinical pathway to advance SYN-010 into pivotal trials. We continue to collaborate with the FDA and are developing a protocol for our Phase 2b/3 adaptive clinical study which we plan to initiate during the first quarter of 2017."

Clinical Program Progress

SYN-004 (ribaxamase): Prevention of CDI, AAD and the emergence of antibiotic-resistant organisms:

Completed enrollment in global Phase 2b placebo-controlled, proof-of-concept clinical trial intended to evaluate the ability of ribaxamase to prevent CDI, C. difficile-associated diarrhea (CDAD), AAD and the emergence of antibiotic-resistant organisms in patients hospitalized with a lower respiratory tract infection and receiving intravenous (IV) ceftriaxone
Enrolled 413 patients across global clinical sites
Anticipate announcing topline results from Phase 2b proof-of-concept clinical trial (1Q 2017)
Awarded government contract from the Centers for Disease Control and Prevention to determine SYN-004’s ability to prevent the emergence of antibiotic-resistant organisms in the gut microbiome of patients enrolled in the Company’s Phase 2b proof-of-concept clinical trial
SYN-010: Treatment of irritable bowel syndrome with constipation (IBS-C) – SYN-010:

Held End of Phase 2 meeting with FDA and received guidance for clinical study design and requirements for Phase 3 development
Submitted Phase 2b/3 adaptive study protocol and corresponding statistical analysis plan to FDA for first pivotal clinical trial (3Q 2016)
Plan to initiate Phase 2b/3 pivotal clinical trial (1Q 2017)
Third Quarter 2016 Financial Results

General and administrative expenses increased to $2.1 million for the third quarter of 2016, from $1.6 million for the third quarter of 2015. This increase is primarily the result of increased stock-based compensation, investor relations expenses and employee salaries and benefits costs offset by lower consulting and legal expenses. The charge related to stock-based compensation expense was $524,000 for the third quarter of 2016, compared to $387,000 for the third quarter of 2015.

Research and development expenses decreased to $7.0 million for the third quarter of 2016, from $10.0 million for the third quarter of 2015. This decrease is primarily the result of charges related to our Exclusive Channel Collaboration (ECC) agreement with Intrexon that we entered into in August 2015. In 2015, we issued 937,500 shares of our common stock to Intrexon as payment of the technology access fee that resulted in a non-cash charge of $3.0 million for the third quarter of 2015. Research and development expenses also include a charge related to non-cash stock-based compensation expense of $422,000 for the third quarter of 2016, compared to $259,000 for the same period last year.

Other income was $0.7 million for the third quarter of 2016, compared to other income of $4.1 million for the third quarter of 2015. Other income for the third quarter of 2016 is due to non-cash income of $0.7 million from the change in fair value of warrants. The decrease in the fair value of the warrants was due to the decrease in our stock price from the prior quarter. Non-cash income related to the decrease of fair value of warrants for the third quarter of 2015 was $4.1 million.

Cash and cash equivalents as of September 30, 2016 were $4.5 million.

On October 31, 2016 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported the presentation of data from the ENGOT-OV16/NOVA trial of niraparib at the 2016 International Gynecologic Cancer Society (IGCS) Biennial Meeting in Lisbon, Portugal (Press release, TESARO, OCT 31, 2016, View Source [SID1234516235]). These data were presented on Sunday, October 30 during the Best Oral session by Dr. Ursula Matulonis, M.D., Medical Director of the Gynecologic Oncology Program at Dana-Farber Cancer Institute and principal investigator on the ENGOT-OV16/NOVA trial, during the Best Oral session. The results were previously published in the New England Journal of Medicine on October 8, 2016 and presented at the ESMO (Free ESMO Whitepaper) 2016 Congress.

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"Many women with recurrent ovarian cancer experience a fear of recurrence in between regimens of platinum-based chemotherapy. The availability of an oral maintenance treatment that could lengthen the progression free survival interval between rounds of platinum-based chemotherapy with a tolerable side effect profile could be very empowering for patients," said Dr. Matulonis. "The data from ENGOT-OV16/NOVA are extremely encouraging and demonstrate the potential of niraparib to offer a meaningful benefit for our patients with ovarian cancer."

"We are grateful for the patients, their families, and the caregivers that participated in the ENGOT-OV16/NOVA study, and we would like to thank our partners at ENGOT for their diligence in executing this trial," said Mary Lynne Hedley, Ph.D., President and COO of TESARO. "We believe the results of this Phase 3 study demonstrated a meaningful benefit for women with platinum sensitive, recurrent ovarian cancer. We are pleased that the EMA recently accepted for review the MAA for niraparib, and we are on track to complete the rolling NDA submission imminently."

ENGOT-OV16/NOVA is a double-blind, placebo-controlled, international Phase 3 trial of niraparib that enrolled 553 patients with recurrent ovarian cancer who were in response to their most recent platinum-based chemotherapy. This trial was designed to assess progression free survival (PFS) in a broad population of patients who were assigned to one of two cohorts based upon germline BRCA mutation status. The ENGOT-OV16/NOVA trial successfully achieved its primary endpoint in both cohorts, demonstrating that niraparib treatment significantly prolonged PFS compared to control in patients who were germline BRCA mutation (gBRCAmut) carriers and in patients who were not germline BRCA mutation (non-gBRCAmut) carriers. A high proportion of patients in both treatment groups in both cohorts had received three or more prior lines of chemotherapy.

The most common (≥10%) treatment-emergent grade 3/4 adverse events in the niraparib arm were thrombocytopenia (33.8%), anemia (25.3%), and neutropenia (19.6%) with treatment discontinuation for these events of 3.3%, 1.4% and 1.9%, respectively. Thrombocytopenia was not associated with grade 3/4 bleeding events. The majority of these hematological laboratory abnormalities occurred within the first three cycles; following dose modifications the incidence of these lab abnormalities decreased and thrombocytopenia and neutropenia were infrequent beyond cycle 3. The rates of MDS/AML in the niraparib (1.4%) and control (1.1%) arms were similar. There were no deaths among patients during study treatment.

About the Phase 3 ENGOT-OV16/NOVA Clinical Trial of Niraparib
ENGOT-OV16/NOVA is a double-blind, placebo-controlled, international Phase 3 trial of niraparib that enrolled 553 patients with recurrent ovarian cancer who were in a response to their most recent platinum-based chemotherapy. Patients were enrolled into one of two independent cohorts based on germline BRCA mutation status. One cohort enrolled patients who were germline BRCA mutation carriers (gBRCAmut), and the second cohort enrolled patients who were not germline BRCA mutation carriers (non-gBRCAmut) and included patients with HRD-positive and HRD-negative tumors. Within each cohort, patients were randomized 2:1 to receive niraparib or placebo and were treated continuously with placebo or 300 milligrams of niraparib, dosed as three 100 milligram tablets once per day, until progression. The primary endpoint of this study was progression-free survival (PFS). Secondary endpoints include patient-reported outcomes, chemotherapy-free interval length, PFS 2, overall survival, and other measures of safety and tolerability. More information about this trial is available at View Source

About Niraparib
Niraparib is an oral, once-daily PARP inhibitor that is currently being evaluated in four ongoing pivotal trials. TESARO is building a robust niraparib franchise by assessing activity across multiple tumor types and by evaluating several potential combinations of niraparib with other therapeutics. The ongoing development program for niraparib includes a Phase 3 trial in patients with platinum-sensitive, recurrent ovarian cancer (the NOVA trial); a Phase 3 trial in patients with first-line ovarian cancer (the PRIMA trial); a registrational Phase 2 treatment trial in patients with ovarian cancer (the QUADRA trial); and a Phase 3 trial for the treatment of patients with BRCA-mutant breast cancer (the BRAVO trial). Several combination studies are also underway, including trials of niraparib plus pembrolizumab and niraparib plus bevacizumab. Janssen Biotech has licensed rights to develop and commercialize niraparib specifically for patients with prostate cancer worldwide, except in Japan.

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to niraparib for the treatment of patients with recurrent platinum-sensitive ovarian, fallopian tube, or primary peritoneal cancer. TESARO has initiated a rolling submission of a New Drug Application (NDA) for niraparib to the FDA, and intends to complete this submission during the fourth quarter. The Marketing Authorization Application (MAA) for niraparib has been submitted to and accepted for review by the European Medicines Agency (EMA) for the maintenance treatment of patients with platinum-sensitive, recurrent ovarian cancer who are in response to platinum-based chemotherapy.

Niraparib is an investigational agent and, as such, has not been approved by the U.S. FDA, the European Medicines Agency (EMA), or any other regulatory agencies.

About Ovarian Cancer
Approximately 22,000 women are diagnosed each year with ovarian cancer in the United States, and more than 65,000 women are diagnosed annually in Europe. Ovarian cancer is the fifth most frequent cause of cancer death among women. Despite high response rates to platinum-based chemotherapy in the second-line advanced treatment setting, approximately 85% of patients will experience recurrence within two years. If approved, niraparib may address the difficult "watchful waiting" periods experienced by patients with recurrent ovarian cancer in between cycles of platinum-based chemotherapy

Phase 1
Phase 2
Phase 3
Application
Therapeutic
area
Cardiovascular-
Metabolics
Oncology
Others

Edoxaban (JP)
(DU-176b / AF / FXa inhibitor)

Prasugrel (JP)
(CS-747 / Ischemic stroke / Anti-
platelet agent)

Esaxerenone (JP)
(CS-3150 / Hypertension /
MR antagonist)

Edoxaban (ASCA etc.)
(DU-176b / AF / FXa inhibitor)

Edoxaban (ASCA etc.)
(DU-176b / VTE / FXa inhibitor)

Tivantinib (US/EU)
(ARQ 197 / HCC / MET inhibitor)

Denosumab (JP)
(AMG 162 / Breast cancer adjuvant /
Anti-RANKL antibody)

Nimotuzumab (JP)
(DE-766 / Gastric cancer / Anti-EGFR
antibody)

Vemurafenib (US/EU)
(PLX4032 / Melanoma Adjuvant / BRAF
inhibitor)

Quizartinib (US/EU/Asia)
(AC220 / AML-2
nd
/ FLT3-ITD inhibitor)

Quizartinib (US/
EU/Asia
)
(AC220 / AML-1
st
/ FLT3-ITD inhibitor)

Pexidartinib (US/EU)
(PLX3397 / TGCT / CSF-1R/KIT/FLT3-ITD
inhibitor)

Laninamivir (US/EU)
(CS-8958 / Anti-influenza /
out-licensing with Biota)

Mirogabalin (US/EU)
(DS-5565 / Fibromyalgia / α2δ ligand)

Mirogabalin (JP/Asia)
(DS-5565 / DPNP/ α2δ ligand)

Mirogabalin (JP/Asia)
(DS-5565 / PHN / α2δ ligand)

Hydromorphone (JP)
(DS-7113 / Cancer pain / Opioid μ-
receptor regulator)

CHS-0214 (JP)
(Etanercept BS / Rheumatoid
arthritis / TNFα inhibitor)

VN-0105 (JP)
(DPT-IPV / Hib vaccine)

Esaxerenone (JP)
(CS-3150 / DM nephropathy / MR
antagonist)

DS-8500 (JP/US)
(Diabetes / GPR119 agonist)

Patritumab (EU)
(U3-1287 / Anti-HER3 antibody)

Pexidartinib (US)
(PLX3397 / CSF-1R/KIT/FLT3-ITD
inhibitor)

DS-1647 (JP)
(Glioblastoma / G47Δ virus)

DS-1040
(Acute ischemic stroke / TAFIa inhibitor)

DS-2330
(Hyperphosphatemia)

DS-9231/TS23
(Thrombosis / α2-PI inactivating antibody)

DS-9001
(Dyslipidemia / Anti-PCSK9 Anticalin-Albumod)

DS-3032 (US/JP)
(MDM2 inhibitor)

PLX7486 (US)
(FMS / TRK inhibitor)

PLX8394 (US)
(BRAF inhibitor)

DS-6051 (US/JP)
(NTRK/ROS1 inhibitor)

PLX9486 (US)
(KIT inhibitor)

DS-3201 (JP)
(EZH1/2 inhibitor)

PLX73086 (US)
(CSF-1R inhibitor)

PLX51107 (US)
(BRD4 inhibitor)

DS-1971
(Chronic pain)

DS-1501
(Osteoporosis / Anti-Siglec-15 antibody)

DS-7080 (US)
(AMD / Angiogenesis inhibitor)

DS-2969
(
Clostridium difficile
infection
/GyrB inhibitor)

DS-5141 (JP)
(DMD / ENA oligonucleotide)

VN-0102/JVC-001 (JP)
(MMR vaccine)

Hydromorphone (JP)
(DS-7113 / Cancer pain / Opioid μ-
receptor agonist)

CL-108 (US)
(Acute pain / Opioid μ-receptor
agonist)

Intradermal Seasonal
Influenza Vaccine (JP)
(VN-100 / prefilled i.d. vaccine for
seasonal flu)

VN-0107/MEDI3250 (JP)
(Nasal spray flu vaccine)

Denosumab (JP)
(AMG 162 / Rheumatoid arthritis /
Anti-RANKL antibody)
Major R&D Pipeline

DS-8895 (JP)
(Anti-EPHA2 antibody)

DS-8273 (US)
(Anti-DR5 antibody)

DS-5573 (JP)
(Anti-B7-H3 antibody)

DS-8201 (JP/
US
)
(Anti-HER2 ADC)

U3-1784 (EU)
(Anti-FGFR4 antibody)

DS-1123 (JP)
(Anti-FGFR2 antibody)

U3-1402 (JP)
(Anti-HER3 ADC)
As of October 2016
Red: Major changes after the FY2016 Q1 financial announcement on July 31, 2016
11. Major R&D Pipeline (Innovative pharmaceuticals
）
As of October 2016
◆
Filed
Class
Target indication
Opioid mu-receptor agonist
combination
Acute pain
Opioid mu-receptor agonist
Cancer pain
Denosumab
Anti-RANKL antibody
Rheumatoid arthritis
Intradermal influenza HA vaccine Prevention of seasonal Influenza
Live attenuated influenza vaccine Prevention of seasonal Influenza
Underline: change after FY2016 Q1 Financial Announcement in July 2016. Regarding edoxaban, the filing in Australia was withdrawn considering its business in total.
Edoxaban
Factor Xa inhibitor
Atrial fibrillation
（
AF
）
ASCA etc.
BR (14/6
*
), TH(15/7), CN(15/8), CA(15/8), TR(15/10)
* means June 2014, ditto
Venous thromboembolism (VTE)
ASCA etc.
BR (14/6), TH(15/7), CN(15/8), CA(15/8), TR(15/10)

The Vaccine is a pre-filled syringe type, intradermal influenza HA vaccine co-developed by four companies [Daiichi Sankyo, Terumo, Japan Vaccine and Kitasato Daiichi Sankyo Vaccine Co., Ltd.]. The intradermal injection device for this vaccine is
developed by Terumo. This device, which offers a more easy-to-use, surefire method to administer the vaccine than current methods. The device is also expected to ease patient hesitation to be injected and lower the risk of damaging peripheral
blood vessels and nerves within the subcutaneous tissue.
VN-0107/MEDI3250
JP
Submitted by Daiichi Sankyo in June 2016
Additional Indication, submitted in Sep 2016
Product Code Number/Generic Name
Region
Note, Filing year/month

Hydromorphone hydrochloride is an opiate, narcotic analgesic that has been available outside of Japan for over 80 years and it is the standard for pain management for cancer pain treatment according to WHO guidelines. Hydromorphone
hydrochloride is one of the agents publicly offered for development by the Review Committee on Unapproved Drugs and Indications with High Medical Needs. Daiichi Sankyo decided to develop the drug in 2012.
VN-100
JP
Submitted by Japan Vaccine in April 2015

The once daily oral anti coagulant (FXa inhibitor) discovered by Daiichi Sankyo. Edoxaban specifically, reversibly and directly inhibits the enzyme, Factor Xa, a clotting factor in the blood.
CL-108
US
NDA was submitted in March 2016 by Charleston
Laboratories, Inc., licensor and co-development partner

CL-108 is novel hydrocodone combination products being developed by Charleston Lab and Daiichi Sankyo for the treatment of moderate to severe pain while preventing or reducing Opioid-Induced Nausea and Vomiting (OINV). The product expects
to reduce the unwanted side effects of opioid-induced nausea and vomiting.
Hydromorphone
JP
NDA of oral formulation (extended-release and
immediate-release formulations) was submitted in March
2016 by Daiichi Sankyo Propharma
JP

Denosumab is fully human monoclonal antibody to target RANK Ligand, an essential mediator of osteoclast formation, in-licensed from Amgen Inc. in 2007. Daiichi Sankyo began sales in Japan of a 60 mg preparation of denosumab as a therapeutic
agent for osteoporosis under the product name PRALIA subcutaneous injection 60mg syringe in June 2013. In addition, from April 2012 Daiichi Sankyo began sales of a 120 mg preparation of denosumab as a therapeutic agent to treat bone
complications stemming from multiple myeloma and bone metastases from solid tumors under the product name RANMARK subcutaneous injection 120 mg, and from May 2014, as a therapeutic agent to treat giant cell tumor of bone under the
product name RANMARK subcutaneous injection 120 mg. Daiichi Sankyo is also participating in global phase 3 clinical trials of denosumab as adjuvant treatment for women with breast cancer.

The US brand name of this vaccine is FluMist Quadrivalent that is a live attenuated influenza vaccine which is administered as a nasal spray and contains four protective strains.
◆
Under development (Phase1-3)
Class
Target indication
target FY for
approval/launch
basically for P3
Remarks
Anti-platelet agent
Ischemic stroke
JP
P3
2017 Additional indication
Edoxaban
Factor Xa inhibitor
Elderly patients with non-valvular atrial fibrillation
JP
P3
2021
Additional dosage and administration for elderly patients
Anti-RANKL antibody
Breast cancer adjuvant
JP
P3
2020 Additional indication
MET inhibitor
Hepatocellular cancer
US/EU P3
2018
Anti-EGFR antibody
Gastric cancer
JP
P3
2020
BRAF inhibitor
Melanoma adjuvant
US/EU P3
–
Additional indication.
Licensee Roche is conducting the study.
Submission in 2016 is planned.
US/EU/Asia
P3
2018 Relapsed and refractory AML patients
US/EU/Asia
P3
2021- Newly diagnosed AML patients
JP
P1
–
Tenosynovial Giant Cell Tumor (TGCT)
US/EU P3
2019 Including pigmented villonodular synovitis
Solid tumor
Asia
P1
– Including TGCT
Glioblastoma
US
P2
–
Melanoma
US
P2
–
Melanoma, solid tumor
US
P1/2
–
Combination with pembrolizumab
in collaboration with Merck
Hypertension
JP
P3
2019
Diabetic nephropathy
JP
P2b
–
Fibromyalgia
US/EU P3
2019
Diabetic peripheral neuropathic pain
JP/Asia P3
2018
Postherpetic neuralgia
JP/Asia P3
2018
Hydromorphone
Opioid mu-receptor agonist
Cancer pain
JP
P3
2018 Injection formulation
TNF
a
inhibitor
Rheumatoid arthritis
JP
P3
2017 Etanercept biosimilar
DPT-IPV/Hib vaccine
Prevention of pertussis, diphtheria, tetanus, poliomyelitis
and Hib
JP
P3
2019 Co-develop with Sanofi K.K.and KDSV
Patritumab
Anti-HER3 antibody
Head & neck cancer
EU
P2
–
G47
Δ
oncolytic virus
Glioblastoma
JP
P2
–
SAKIGAKE Designation granted
GPR119 agonist
Diabetes
JP/US
P2
–
US/EU P2
– Out-licensing with Biota
JP
P1
– Nebulizer formulation
Acute myeloid leukemia
Generic Name / Project Code Number
Stage
Prasugrel
Denosumab
Tivantinib
Nimotuzumab
Vemurafenib
Quizartinib
FLT3-ITD inhibitor
DS-1647
Pexidartinib/PLX3397
CSF-1R/KIT/FLT3-ITD inhibitor
Esaxerenone/CS-3150
MR antagonist
Mirogabalin
α2δ ligand
CHS-0214
VN-0105
DS-8500
Laninamivir
Neuraminidase inhibitor
Influenza
Underline: change after FY2016 Q1 Financial Announcement in July 2016
◆
Under development (Phase1-3)
Class
Target indication
target FY for
approval/launch
basically for P3
Remarks
Solid cancer, lymphoma
US/JP
P1
–
Leukemia
US
P1
–
FMS/TRK inhibitor
Solid cancer
US
P1
–
Anti-EPHA2 antibody
Solid cancer
JP
P1
–
Anti-DR5 antibody
Solid cancer
US
P1
–
BRAF inhibitor
Solid cancer, leukemia
US
P1
–
DS-6051
NTRK/ROS1 inhibitor
Solid cancer
US/JP
P1
–
DS-5573
Anti-B7-H3 antibody
Solid cancer
JP
P1
–
PLX9486
KIT inhibitor
Solid cancer
US
P1
–
DS-8201
Anti-HER2 antibody drug conjugate
Solid cancer
JP/US
P1
–
Anti-FGFR4 antibody
Solid cancer
EU
P1
–
Anti-FGFR2 antibody
Solid cancer
JP
P1
–
DS-3201
EZH1/2 inhibitor
Non-Hodgkin’s lymphoma
JP
P1
–
PLX73086/AC708
CSF-1R inhibitor
Tenosynovial Giant Cell Tumor (TGCT)
US
P1
–
PLX51107
BRD4 inhibitor
Hematologic malignancies
US
P1
–
Anti-HER3 antibody drug conjugate
Solid cancer
JP
P1
–
TAFIa inhibitor
Acute ischemic stroke, Acute pulmonary embolism
–
P1
–
Hyperphosphatemia treatment
Hyperphosphatemia in chronic kidney disease (CKD) –
P1
–
α2 plasmin inhibitor-inactivating
antibody
Thrombosis (cardiovascular diseases, ischemic stroke) –
P1
– In-licensed from Translational Sciences Inc.
Anti-PCSK9 Anticalin-Albumod
Dyslipidemia
–
P1
–
Analgesic agent
Chronic pain
–
P1
–
Anti-Siglec-15 antibody
Osteoporosis
US
P1
–
DS-7080
Angiogenesis inhibitor
Neovascular age-related macular degeneration (AMD) US
P1
–
DS-2969
GyrB inhibitor
Clostridium difficile infection (CDI)
–
P1
–
DS-5141
ENA oligonucleotide
Duchenne muscular dystrophy
JP
P1/2
–
Measles-Mumps-Rubela vaccine Prevention of Measles, Mumps and Rubela
JP
P1/2
2022
Japan vaccine company is conducting the
phase 1/2 study
DS-1123
U3-1402
DS-1971
DS-1501
Generic Name / Project code number
Stage
DS-3032
MDM2 inhibitor
PLX7486
DS-8895
DS-8273
PLX8394
U3-1784
DS-1040
DS-2330
DS-9231/TS23
DS-9001
VN-0102/JVC-001
◆
Stage-up (major changes from the FY2016 Q1 Financial Announcement in July 2016)
Class
Target indication
Remarks
FXa inhibitor
Elderly patients with non-valvular atrial fibrillation
JP
P3
Started phase 3 study for new dosage and
administration
FLT3-ITD inhibitor
Acute myeloid leukemia
EU/Asia
P3
Newly diagnosed AML patients. Started
phase 3 study in EU and Asia in addition to
US
MR antagonist
Hypertension
JP
P3
Started phase 3 study
Anti-HER2 antibody drug conjugate Solid cancer
US
P1
Started phase 1 study in US in addition to
Japan
Anti-HER3 antibody drug conjugate
Solid cancer
JP
P1
Started phase 1 study
Current stage
Denosumab
Anti-RANKL antibody
Rheumatoid arthritis
JP
Submitted
Generic Name / Project code number
U3-1402
DS-8201
Additional Indication, submitted in Sep 2016
Edoxaban
Quizartinib
Esaxerenone/CS-3150
Oncology
[ Project after Phase 2 ]
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Tivantinib
ARQ 197
Oral
MET inhibitor
– Hepatocellular cancer
ArQule
TBD
・
A phase 3 clinical study for HCC with MET high patients was started in Jan 2013.
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Nimotuzumab
DE-766 Injection
Anti-EGFR antibody – Gastric cancer
InnoCIMAb
TBD
・
Phase 3 in Japan for Gastric cancer started in April 2013.
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Quizartinib
AC220
Oral
FLT3-ITD inhibitor – Acute myeloid leukemia
Daiichi Sankyo
(Ambit)
TBD
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Patritumab
U3-1287 Injection
Anti-HER3 antibody – Head and neck cancer
Daiichi Sankyo
(U3 Pharma)
TBD
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Vemurafenib
PLX4032
Oral
BRAF inhibitor
– Melanoma adjuvant
Daiichi Sankyo
（
Plexxikon
）
Zelboraf
Kinase inhibitor against a receptor-type tyrosine kinase, FLT3.
Therapeutic effect for patients with acute myeloid leukemia harboring FLT3-ITD mutation is expected.

The fully human monoclonal antibody to target HER3, one of the Epidermal Growth Factor Receptor (EGFR) family of proteins. HER 3 is overexpressed in many tumors of epithelial origin and HER2/HER3 dimers and EGFR/HER3
dimers are more potent to induce tumor cell proliferation than homodimers of HER2 or EGFR.

The molecular-targeted agent to inhibit HGF(hepatocyte growth factor) receptor, MET which has multiple roles in intracellular signal transductions such as cancer cell proliferation, angiogenesis, invasion, and apoptosis induction.

The humanized monoclonal antibody to target Epidermal Growth Factor Receptor(EGFR). This antibody is expected to be a best in class EGFR, safety against the skin toxicity and the efficacy comparable to the other antibodies.

The molecular-targeted agent to inhibit BRAF V600E mutation. Launched since 2011 as personalized treatment for patients with unrespectable or metastatic melanoma. NDA for combination therapy with MEK inhibitor cobimetinib
was approved in US on November 11, 2015. A phase 1 study of a combination with anti PD-L1 monoclonal antibody atezolizumab and cobimetinib is being investigated by Genentech.
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Pexidartinib
PLX3397
Oral
CSF-1R/KIT/FLT3-ITD
inhibitor
– Tenosynovial Giant Cell Tumor
– Glioblastoma
– Melanoma
Daiichi Sankyo
（
Plexxikon
）
TBD
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
G47
Δ
DS-1647 Injection
Oncolytic Virus – Glioblastoma
Prof. Todo
Institute of
Medical Science
Univ. of Tokyo
TBD
The molecular-targeted agent to inhibit CSF-1R, KIT and FLT3-ITD. This agent is expected to reduce tumor cell proliferation and expansion of metastases.

A triple-mutated, replication-conditional herpes simplex virus type 1 (the third generation oncolytic herpes simplex virus type 1), designed to replicate only in cancer cells. This oncolytic virus therapy is expected better safety and
efficacy profile compare to existing oncolytic virus. This product received SAKIGAKE Designation from MHLW on February 10th ,2016. Phase 2 Investigator Initiated Study for Glioblastoma is on-going in Japan.

Cardiovascular-Metabolics
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Edoxaban
DU-176b
Oral
Factor Xa inhibitor
– Atrial fibrillation (AF)
– Venous thromboembolism (VTE)
Daiichi Sankyo
Lixiana (JP)
LIXIANA (EU, Asia)
SAVAYSA (US)
・
Top line results of Hokusai-VTE (VTE) phase 3 study was presented at ESC 2013.
・
Top line results of ENGAGE AF-TIMI 48 (AF) phase 3 study was presented at AHA 2013.
・
AF/VTE indication: Following the launch in Japan in Sep 2014 (Dec 2014 for 60 mg tablet), edoxaban has already been marketed in more than 10 countries.
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Prasugrel
CS-747
Oral
Anti-platelet agent
– Ischemic stroke
Daiichi Sankyo
Ube Industries
Effient (US, Asia)
Efient (JPN, EU)
・
Co-development with Eli Lilly in the US and EU, development by Daiichi Sankyo in Japan
.
・
[JP] Approved in Mar 2014 for the ischemic heart disease undergoing PCI and launched in May 2014. Phase 3 studies for ischemic stroke are completed.
・
[US] Phase 3 study in pediatric sickle cell disease patients was conducted. The patent exclusivity in US has been extended for 180 days based on the pediatric study completion.
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Esaxerenone
CS-3150
Oral
MR antagonist
– Hypertension
– Diabetic nephropathy
Exelixis
TBD
・
On January 2015, phase 2b studies of HTN and DN started.
・
On November 2015, phase 2b study in essential hypertension in Japan was completed.
・
In September 2016, phase 3 of HTN started.
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
–
DS-8500
Oral
GPR119 agonist
– Diabetes
Daiichi Sankyo
TBD

The once daily oral anti coagulant (FXa inhibitor). Edoxaban specifically, reversibly and directly inhibits the enzyme, Factor Xa, a clotting factor in the blood. Launched in Japan in July 2011 as the prevention of venous
thromboembolism (VTE) in patients with total knee arthroplasty, total hip arthroplasty and hip fracture surgery.

The oral antiplatelet agent. Prasugrel helps to keep blood platelets from clumping together and developing a blockage in an artery.

The agent inhibits aldosterone binding to Mineralocorticoid Receptor(MR) which stimulate the sodium absorption into kidney. This agent is expected antihypertensive and organ-protective effect.

The agent agonistically acts on GPR119 which is expressed in small intestine and spleen cells, stimulates insulin secretion, and lowers blood sugar concentration.
Others
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Intradermal Seasonal
Influenza Vaccine
VN-100
Injection
(prefilled
syringe)
Seasonal flu vaccine – Prevention of seasonal influenza
Daiichi Sankyo
（
KDSV
）
Terumo
TBD

Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Hydromorphone
DS-7113
Oral/
Injection
opioid mu-receptor
regulator
– Cancer pain
–
TBD
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Denosumab
AMG 162 Injection
Anti-RANKL antibody
– Breast cancer adjuvant
– Rheumatoid arthritis
Amgen
SRE, GCTB: Ranmark(JP)
Osteoporosis: Pralia(JP)
・
July 2007 In-licensed from Amgen.
・
Phase 3 : Breast cancer adjuvant
・
September 2016 sNDA submission for rheumatoid arthritis (additional indication)
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
–
CHS-0214 Injection
TNFα inhibitor
– Rheumatoid arthritis
Coherus
TBD
・
Phase 3 study for patients with rheumatoid arthritis in Japan was completed. In preparation for submission.
The Vaccine is a pre-filled syringe type, intradermal influenza HA vaccine co-developed by four companies [Daiichi Sankyo, Terumo, Japan Vaccine and Kitasato Daiichi Sankyo Vaccine Co., Ltd.]. The intradermal injection device
for this vaccine is developed by Terumo. This device, which offers a more easy-to-use, surefire method to administer the vaccine than current methods. The device is also expected to ease patient hesitation to be injected and
lower the risk of damaging peripheral blood vessels and nerves within the subcutaneous tissue.
・
NDA submission in Apr 2015 by Japan Vaccine

The opiate, narcotic analgesic that has been available outside of Japan for over 80 years and recommended in WHO guideline as a standard analgesia for cancer pain. It is designated as unapproved drug by "Study Group on
Unapproved and Off-label Drugs of High Medical Need."
・
NDA of oral formulations (extended-release and immediate-release formulations) were submitted in Mar 2016 by Daiichi Saknyo Propharma. Phase 2/3 studies for injection formulation are ongoing.

The fully human monoclonal antibody to target RANK Ligand, an essential mediator of osteoclast formation. Launched in Japan in April 2012 as treatment for bone complications stemming from multiple myeloma and bone
metastases from solid tumors, in June 2013 as treatment for osteoporosis, and in May 2014 as treatment for giant cell tumor of bone.

Biosimilar product for etanercept
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
–
CL-108
Oral
opioid mu-receptor
regulator
– Acute pain
Charleston Laboratories
TBD
NDA submission in Mar 2016 by Charleston Laboratories
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
Mirogabalin
DS-5565
Oral
α2δ ligand
– Fibromyalgia
– Diabetic peripheral neuropathic pain
– Postherpetic neuralgia
Daiichi Sankyo
TBD
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
–
VN-0107
MEDI3250
Nasal
spray
Seasonal flu vaccine
– Prevention of seasonal influenza
AZ/MedImmune
TBD
Generic Name
Project
code Number
Dosage
Form
Class
Target Indication
Origin
Brand Name
–
VN-0105
Injection
DPT-IPV/Hib vaccine
– Prevention of pertussis, diphtheria, tetanus, poliomyelitis and Hib
Daiichi Sankyo
(KDSV)
Sanofi Pasteur
TBD
The pain therapy agent to reduce the neurotransmitter release from nerve terminals. This agent is expected to show the good balanced efficacy and safety profile.

Combination product of immediate-release promethazine, hydrocodone and acetaminophen. Reduction of Opioid-Induced Nausea and Vomiting is expected.

A combination vaccine reconstituting Hib with precipitated and purified pertussis-diphtheria-tetanus-inactivated polio vaccine (Salk vaccine) vaccine previously licensed and launched in Japan, as 1st in the class of pentavalent
vaccine (DPT-IPV/Hib
）
.
・
Phase 3 study in Japan started in Oct 2014.
・
Phase 2 studies for diabetic peripheral neuropathic pain were completed.
・
Phase 3 studies for diabetic peripheral neuropathic pain and postherpetic neuralgia are in execution in JP/Asia.
・
Phase 3 studies for fibromyalgia are in execution in US/EU.

The US brand name of this vaccine is FluMist Quadrivalent that is a live attenuated influenza vaccine which is administered as a nasal spray and contains four protective strains. Phase 3 safety and efficacy studies were conducted
for FluMist Quadrivalent in Japanese children over the 2014-2015 influenza season and NDA was submitted in Japan in June 2016

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

On October 31, 2016 Foundation Medicine, Inc. (NASDAQ:FMI) and Sarah Cannon Research Institute (Sarah Cannon) reported a collaboration focused on advancing personalized medicine utilizing molecular information for patients across Sarah Cannon’s cancer programs in the United States (Press release, Foundation Medicine, OCT 31, 2016, View Source [SID1234516142]). The organizations will gather results from Foundation Medicine’s full suite of comprehensive genomic profiling (CGP) assays to personalize treatment options for patients and to ultimately support improved outcomes.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Sarah Cannon is one of the world’s leading clinical research organizations conducting community-based clinical trials across a global network. Researchers can use Foundation Medicine’s CGP assays, FoundationOne for use with solid tumors, FoundationOne Heme for use with hematologic malignancies and FoundationACT for use as a liquid biopsy, to identify eligible patients for participation in Sarah Cannon’s clinical studies. Foundation Medicine’s CGP assays aid treating physicians and researchers to more effectively screen and match patients to early and late-phase clinical trials based on their genomic information. Sarah Cannon will also integrate Interactive Cancer Explorer, Foundation Medicine’s physician facing decision support portal, across its network to facilitate clinico-genomic knowledge among researchers and to enhance patient access to precision therapeutics.

Additionally, Sarah Cannon and Foundation Medicine have entered into a master research program agreement to collaborate on the development of research studies, as well as clinical programs designed to evaluate and establish the most appropriate use of Foundation Medicine’s assays into clinical care pathways.

"Our physicians are at the forefront of clinical research through our work with novel investigative therapies," said Howard A. "Skip" Burris, M.D., president, clinical operations and chief medical officer, Sarah Cannon. "Clinical trials, particularly those where patients can be molecularly matched to a study, are an integral part of effectively treating many types of cancers and accelerating patient access to novel therapies. We’re excited to collaborate with Foundation Medicine as we continue to enhance access to molecular information, which helps improve clinical care and inform research across our network."

Sarah Cannon and Foundation Medicine will also work together to develop training and educational programs that support the advancement of personalized medicine, including work with Sarah Cannon’s Nurse Navigator Program, a high-touch, personalized support program that enables nurses to comprehensively manage a patient’s experience with cancer.

"Precision medicine in cancer can be achieved by innovating new ideas that accelerate patient access to novel compounds in development," said Steven Kafka, president and chief operating officer for Foundation Medicine. "Together with Sarah Cannon, we believe we are in a unique position to eliminate roadblocks to patient access, to integrate genomics knowledge into clinical pathways and to extend that knowledge across the cancer care continuum to accelerate research and drive better outcomes for all patients."