On November 1, 2016 NewLink Genetics Corporation (NASDAQ:NLNK), a biopharmaceutical company focused on bringing novel immuno-oncology therapies to patients with cancer, reported consolidated financial results for the third quarter of 2016 and progress in its clinical and pipeline development programs (Press release, NewLink Genetics, NOV 1, 2016, View Source [SID1234516135]).

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"We continue to focus our clinical developmental efforts targeting the IDO pathway. We have two distinct IDO pathway inhibitors advancing in the clinic, GDC-0919 with our partner Genentech and indoximod, our proprietary IDO pathway inhibitor," said Charles J. Link, Jr. MD, Chairman, Chief Executive Officer and Chief Scientific Officer. "We are also encouraged by recent clinical data that increasingly validate the IDO pathway as an important target in immuno-oncology."

The Company hosted an investor day on October 25, outlining its vision and execution plans for the future. A webcast of the Company’s presentations can be found at View Source

"As described at our investor day, we believe 2017 will be an important year in the development of both GDC-0919 and indoximod and we look forward to providing further updates and results," added Nicholas N. Vahanian, MD, President and Chief Medical Officer.

The program featured leaders in the field of immuno-oncology and pioneers in the science of IDO: George C. Prendergast, PhD, President & CEO, Lankenau Institute for Medical Research (LIMR) and Editor in Chief, Cancer Research; David H. Munn, MD, Professor of Pediatric Hematology-Oncology, Medical College of Georgia, Augusta University; Montaser Shaheen MD, Associate Professor, University of New Mexico Cancer Center; and Ashkan Emadi, MD, PhD, Associate Professor, University of Maryland.

Key takeaways from the Investor Day included:

1. Validation of IDO as a Target. The IDO pathway can allow cancer to escape the immune system. Many cancers have developed the ability to employ IDO to evade immune attack. We believe clinical results are increasingly validating the IDO pathway as a target for cancer therapies. Just as scientists discovered the role of PD-1/PD-L1 expression and the usefulness of PD-1/PD-L1 blockade, there is an increasing body of research into the role of the IDO pathway in cancer.

2. NewLink’s Two IDO Pathway Inhibitor Clinical Candidates. NewLink Genetics is engaged in clinical trials for two IDO pathway inhibitor product candidates, each with its own distinct mechanism of action.

GDC-0919, a direct IDO enzymatic inhibitor, is being developed in partnership with Genentech. GDC-0919 is currently in a Phase 1b trial, in combination with atezolizumab in solid tumors. In October, 2014, NewLink and Genentech entered in to a license and collaboration agreement with an upfront payment of $150 million, more than $1 billion in potential milestones, and substantial royalties.
Indoximod, an IDO pathway inhibitor, is proprietary to NewLink Genetics. Indoximod is being tested in the clinic in multiple indications including melanoma, pancreatic cancer, malignant brain tumors, breast cancer, acute myeloid leukemia, and non-small cell lung cancer.
3. Indoximod Clinical Development. The Company reported that it will evaluate the data and report on several clinical trials underway in 2017. Furthermore, our clinical development strategy for indoximod includes formulation optimization intended to improve the candidate’s clinical and commercial potential.

4. Future R&D. NewLink also discussed its program targeting the PTEN pathway in regulatory T cells (Treg cells) as a central driver of tumor immunosuppression. NewLink Genetics is an early leader in the field of PTEN research, just as it was in developing IDO as a potential pathway for immune suppression in cancer.

Financial Results for the Three-Month Period Ended September 30, 2016

Cash Position: NewLink Genetics ended the quarter on September 30, 2016, with cash and equivalents totaling $148.3 million, compared to $197.8 million for the year ending December 31, 2015.

R&D Expenses: Research and development expenses in the third quarter of 2016 were $24.5 million, compared to $22.5 million during the comparable period in 2015. The increase was primarily due to a $3.2 million increase in contract manufacturing costs, a $340,000 increase in stock compensation expense, and a $400,000 increase in clinical trial expenses, offset by a decrease in equipment and supplies of $1.3 million, wages of $490,000 and a $160,000 decrease in consulting.

G&A Expenses: General and administrative expenses in the third quarter of 2016 were $7.7 million compared to $7.4 million during the comparable period in 2015. The increase was due primarily to an increase of $300,000 in consulting and personnel-related expenses.

Net Income/Loss: NewLink Genetics reported a net loss of $15.5 million, or a loss of $0.54 per diluted share, for the third quarter of 2016, compared to a net loss of $15.9 million, or a loss of $0.55 per diluted share, for the comparable period in 2015.

NewLink Genetics ended the quarter with 29,091,652 shares outstanding.

Financial Guidance and Upcoming Investor Meetings

NewLink Genetics expects to have approximately $132 million in cash and equivalents on December 31, 2016.

We have presented at seven investor meetings and conferences since the beginning of the year, including our own investor day held last week. We expect to present at three upcoming conferences in New York City, including the Global Mizuho Investor Conference on November 14, the Stifel 2016 Healthcare Conference on November 15, and the Piper Jaffray 28th Annual Healthcare Conference on November 29.

On November 1, 2016 Corcept Therapeutics Incorporated (NASDAQ: CORT), a pharmaceutical company engaged in the discovery, development and commercialization of drugs that treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of cortisol, reported its financial results for the quarter ended September 30, 2016 (Press release, Corcept Therapeutics, NOV 1, 2016, http://www.corcept.com/news_events/view/pr_1478031960 [SID1234516139]).

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Corcept reported revenue of $21.7 million and GAAP net income of $2.6 million for the third quarter of 2016, compared to revenue of $13.3 million and a GAAP net loss of $0.6 million for the third quarter of 2015. The company’s cash and cash equivalents were $47.9 million at September 30, 2016, an increase of $6.1 million from June 30, 2016.

The company expects that its revenue for 2016 will be $79-82 million, an increase from its original guidance of $76-81 million.

"The productivity of our expanded team of clinical specialists continues to improve," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "Because of their hard work, the number of patients with Cushing’s syndrome receiving Korlym grew again last quarter, as did the number of physicians prescribing the medication. We do not see a leveling off in this growth. Many endocrinologists have yet to prescribe Korlym and many patients who could benefit from the medication have yet to receive it."

"The success of our Korlym franchise is allowing us to build a development program of exciting breadth and depth," said Robert S. Fishman, MD, Corcept’s Chief Medical Officer. "Our proprietary, selective cortisol modulator CORT125134 – now in its Phase 2 trial – may offer Cushing’s syndrome patients Korlym’s powerful benefits, but without the side effects associated with Korlym’s affinity for the progesterone receptor. We look forward to those results next year.

"Our oncology program continues to progress," added Dr. Fishman. "We are enrolling patients in our Phase 1/2 open-label trial of CORT125134 as a treatment for solid-tumor cancers. Korlym’s efficacy is being investigated as a treatment for patients with triple-negative breast cancer and castration-resistant prostate cancer. Stacie Shepherd, MD, PhD, a senior oncology development executive from Abbvie, joined us last quarter to lead the program.

"Our pipeline will broaden significantly next year, when we expect to advance to the clinic additional selective cortisol modulators that have shown great promise in animal models of solid-tumor cancers and metabolic disorders, including fatty liver disease."

Financial Discussion

Corcept’s GAAP net income for the third quarter of 2016 was $2.6 million, compared to a GAAP net loss of $0.6 million for the third quarter of 2015. Excluding non-cash expenses related to stock-based compensation and accreted interest on the company’s capped royalty obligation (the "Royalty Financing"), Corcept generated $4.9 million of non-GAAP net income in the third quarter of 2016, compared to non-GAAP net income of $1.6 million in the third quarter of 2015. A reconciliation of GAAP to non-GAAP net operating results is set forth below.

Operating expenses for the third quarter of 2016 increased to $18.7 million, from $13.2 million in the third quarter of 2015, primarily due to: (i) increased employee compensation expense associated with expansion of the company’s commercial and clinical development teams; (ii) growth in patient support costs, distribution expenses and commissions resulting from higher sales volumes; and (iii) increased spending on the clinical development of CORT125134.

Corcept’s cash and cash equivalents totaled $47.9 million at September 30, 2016, compared to $41.8 million at June 30, 2016. These cash balances reflect scheduled payments made under the Royalty Financing of $4.0 million and $3.3 million in the third and second quarters of 2016, respectively. The company expects to make its final payment under the Royalty Financing in 2017.

Conference Call

Corcept will hold a conference call on November 1, 2016, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss this announcement. To participate, dial 1-800-446-1671 from the United States or 1-847-413-3362 internationally approximately ten minutes before the start of the call. The passcode will be 43632675. A replay will be available through November 15, 2016 at 1-888-843-7419 from the United States and 1-630-652-3042 internationally. The passcode for the replay will be 43632675.

About Cushing’s Syndrome

Endogenous Cushing’s syndrome is caused by prolonged exposure of the body’s tissues to high levels of the hormone cortisol and is generated by tumors that produce cortisol or ACTH. Cushing’s syndrome is an orphan indication that most commonly affects adults aged 20-50. An estimated 10-15 of every one million people are newly diagnosed with this syndrome each year, resulting in over 3,000 new patients annually in the United States. An estimated 20,000 patients in the United States have Cushing’s syndrome. Symptoms vary, but most people have one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Cushing’s syndrome can affect every organ system in the body and can be lethal if not treated effectively.

About Triple-Negative Breast Cancer

Triple-negative breast cancer is a form of the disease in which the three receptors that fuel most breast cancer growth – estrogen, progesterone and the HER-2/neu gene – are not present. Because the tumor cells lack the necessary receptors, treatments that target estrogen, progesterone and HER-2 receptors are ineffective. In 2013, approximately 40,000 women were diagnosed with triple-negative breast cancer. Corcept estimates that more than 75 percent of these women’s tumor cells express the GR receptor to which cortisol binds. There is no FDA-approved treatment and neither a targeted treatment nor an approved standard chemotherapy regimen for patients with relapsed triple-negative disease exists.

About Korlym

Korlym modulates the effect of cortisol at the glucocorticoid receptor, one of the two receptors to which cortisol binds, thereby inhibiting the effects of excess cortisol in patients with Cushing’s syndrome. Since 2012, Corcept has made Korlym available as a once-daily oral treatment of hyperglycemia secondary to endogenous Cushing’s syndrome in adult patients with glucose intolerance or diabetes mellitus type 2 who have failed surgery or are not candidates for surgery. Korlym was the first FDA-approved treatment for that illness. The FDA has designated it as an Orphan Drug for that indication.

About CORT125134

CORT125134 is the lead compound in Corcept’s portfolio of selective cortisol modulators. It is a non-steroidal competitive antagonist of the glucocorticoid receptor that does not bind to the body’s other hormone receptors, including the progesterone receptor. It is the affinity of Korlym for the progesterone receptor that results in termination of pregnancy and can cause endometrial thickening and irregular vaginal bleeding in some women. CORT125134 will not have these effects. Corcept is currently studying the compound in two clinical trials, one for the treatment of patients with Cushing’s syndrome and another for patients suffering from solid-tumor cancers. CORT125134 is proprietary to Corcept and is protected by composition of matter and method of use patents extending to 2033.

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.

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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."

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

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