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Pipeline Review Check

Phase 1
Phase 2
Phase 3
Application
Therapeutic
area
Cardiovascular-
Metabolics
Oncology
Others
n
Prasugrel
(JP)
(CS-747 / Ischemic stroke / Anti-
platelet agent)
n
Edoxaban (ASCA
etc.)
(DU-176b / AF / oral factor Xa inhibitor)
n
Edoxaban
(ASCA etc.)
(DU-176b / VTE / oral factor Xa inhibitor)
n
Tivantinib (US/EU)
(ARQ 197 / HCC / MET inhibitor)
n
Denosumab (JP)
(AMG 162 / Breast cancer adjuvant /
Anti-RANKL antibody)
n
Nimotuzumab (JP)
(DE-766 / Gastric cancer / Anti-EGFR
antibody)
n
Vemurafenib (US/EU)
(PLX4032 / Melanoma Adjuvant / BRAF
inhibitor)
n
Quizartinib (US/EU/Asia)
(AC220 / AML-2
nd
/ FLT3-ITD inhibitor)
n
Quizartinib (US)
(AC220 / AML-1
st
/ FLT3-ITD inhibitor)
n
Pexidartinib
(US/EU)
(PLX3397 / TGCT / CSF-1R/KIT/FLT3-ITD
inhibitor)
n
Laninamivir
(US/EU)
(CS-8958 / Anti-influenza /
out-licensing with Biota)
n
Mirogabalin (US/EU)
(DS-5565 / Fibromyalgia /
α
2
δ
ligand)
n
Mirogabalin
(JP/Asia)
(DS-5565 / DPNP/
α
2
δ
ligand)
n
Mirogabalin
(JP/Asia)
(DS-5565 / PHN /
α
2
δ
ligand)
n
Denosumab (JP)
(AMG 162 / Rheumatoid arthritis /
Anti-RANKL antibody)
n
Hydromorphone (JP)
(DS-7113 / Cancer pain / Opioid
μ
–
receptor regulator)
n
CHS-0214 (JP)
(Etanercept BS / Rheumatoid
arthritis / TNF
α
inhibitor)
n
VN-0105 (JP)
(DPT-IPV /
Hib
vaccine)
n
VN-0107/MEDI3250
(JP)
(Nasal spray flu vaccine
vaccine)
n
CS-3150 (JP)
(Hypertension
・
DM
nephropathy /
MR antagonist)
n
DS-8500
(JP/US)
(Diabetes / GPR119 agonist)
n
Patritumab (US/EU)
(U3-1287 / Anti-HER3 antibody)
n
Pexidartinib (US)
(PLX3397
/ CSF-1R/KIT/FLT3-ITD
inhibitor)
n
DS-1040
(Acute ischemic stroke / TAFIa inhibitor)
n
DS-2330
(
Hyperphosphatemia
)
n
DS-9231/TS23
(Thrombosis /
α
2-PI
inactivating antibody)
n
DS-9001
(Dyslipidemia / Anti-PCSK9 Anticalin-Albumod)
n
DS-3032 (US/JP)
(MDM2
inhibitor)
n
PLX7486 (US)
(FMS / TRK inhibitor)
n
PLX8394 (US)
(BRAF inhibitor)
n
DS-6051 (US/
JP
)
(NTRK/ROS1 inhibitor)
n
PLX9486 (US)
(KIT inhibitor)
n
DS-3201 (JP)
(EZH1/2 inhibitor)
n
PLX73086 (US)
(CSF-1R
inhibitor)
n
PLX51107 (US)
(BRD4 inhibitor)
n
DS-1971
(Chronic pain)
n
DS-1501
(Osteoporosis / Anti-Siglec-15 antibody)
n
DS-7080 (US)
(AMD / Angiogenesis inhibitor)
n
DS-2969
(
Clostridium
difficile
infection
/
GyrB
inhibitor)
n
DS-5141 (JP)
(DMD / ENA
oligonucleotide)
n
VN-0102/JVC-001 (JP)
(MMR
vaccine)
n
Hydromorphone (JP)
(DS-7113 / Cancer pain / Opioid
μ
–
receptor agonist)
n
CL-108 (US)
(Acute pain / Opioid
μ
-receptor
agonist)
n
Intradermal Seasonal
Influenza Vaccine (JP)
(VN-100 / prefilled
i.d.
vaccine for
seasonal flu)
Major R&D Pipeline
n
DS-8895 (JP)
(Anti-EPHA2 antibody)
n
DS-8273 (US)
(Anti-DR5 antibody)
n
DS-5573
(JP)
(Anti-B7-H3 antibody)
n
DS-8201 (JP)
(Anti-HER2 ADC)
n
U3-1784 (EU)
(Anti-FGFR4 antibody)
n
DS-1123 (JP)
(Anti-FGFR2 antibody)
Red
:
Major changes after the FY2015
Q3
financial announcement
on January 29, 2016
As of May 2016
2
12. Major R&D Pipeline (Innovative pharmaceuticals
）
As of May 2016
◆
Launched/Approved
Generic Name
Class
Indication
Region
Status
Remarks
Edoxaban
Factor Xa inhibitor
Atrial fibrillation
（
AF
）
ASCA
etc.
Launched
S. Korea (16/2
*
)
* means Feb 2016, ditto
Approved
Taiwan (16/2)
Venous thromboembolism (VTE)
ASCA
etc.
Launched
S. Korea (16/2)
Approved
Taiwan (16/2)

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. 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.
Additional indications for AF/VTE was approved in September 2014 and 60 mg tablet was launched in December 2014. Launched in US, Switzerland, UK, Germany, Ireland and
Netherlands in February, May, July, August, September and November 2015, respectively. Approved in South Korea in August 2015.
VN-101
Cell-culture H5N1 flu vaccine
Prevention of H5N1 influenza
JP
Approved in March 2016

This vaccine is a cell-culture H5N1 flu vaccine that has developed by Daiichi Sankyo and
Kitasato
Daiichi Sankyo Vaccine(KDSV).
KDSV was submitted a supplemental application for optimization of pediatric use.
Underline: change after FY2015 Q3 Financial Announcement in Jan 2016

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8-K – Current report

On May 12, 2016 AmpliPhi Biosciences Corporation (NYSEMKT: APHB), a global leader in the development of bacteriophage-based antibacterial therapies to treat drug-resistant infections, reported its financial results for the first quarter ended March 31, 2016 (Filing, Q1, AmpliPhi Biosciences, 2016 , MAY 12, 2016, View Source [SID:1234512411]).

"We have made significant progress in 2016, effectively executing on our clinical development and business strategies," said M. Scott Salka, CEO of AmpliPhi Biosciences. "We acquired additional bacteriophage assets, presented in vitro data for AB-PA01 for Pseudomonas aeruginosa, dosed our first patient and successfully completed the first cohort in our Phase I clinical trial of AB-SA01 for the treatment of Staphylococcus aureus infections in patients with chronic rhinosinusitis. We also saw our outstanding Series B Preferred stock be converted to Common, thereby streamlining our capital structure."

Highlights Demonstrate Progress in R&D, Governance, and Finance
· Acquired key bacteriophage assets from Novolytics in January 2016, broadening AmpliPhi’s IP portfolio and accelerating the development of our phage-based therapies
· AmpliPhi’s collaboration partner, the Westmead Institute’s Centre for Infectious Diseases and Microbiology, received an AUS $860,000 grant from the Australian Government to isolate and develop phages targeting E. Coli and Klebsiella. AmpliPhi will participate in the project by providing its proprietary expertise in bacteriophage isolation, characterization and manufacturing scale-up
· Appointed Steve Martin as Chief Financial Officer in January 2016, strengthening AmpliPhi’s financial and business management expertise
· Dosed the first patient in AmpliPhi’s Phase I clinical trial of AB-SA01 for the treatment of Staphylococcus aureus infections in patients with chronic rhinosinusitis
· The outstanding Series B Preferred stock was converted into common stock, streamlining AmpliPhi’s capital structure
· Presented in vitro data demonstrating that AB-PA01, AmpliPhi’s proprietary, investigational phage mix, was capable of effectively infecting and killing Pseudomonas aeruginosa (P. aeruginosa) clinical isolates from a global population of patients with and without cystic fibrosis, including multi-drug resistant strains of P. aeruginosa. AB-PA01 also demonstrated activity similar to meropenem in a murine model of acute lung infection
· Completed dosing of the first cohort in the Phase I clinical trial of AB-SA01 in patients with chronic rhinosinusitis. Three patients each received AB-SA01 twice daily for seven days; treatment was well tolerated and there were no apparent drug-related adverse events. The first patient in the second cohort has been dosed and will receive AB-SA01 twice daily for 14 days. We expect to report results from this study in the second half of 2016.

First Quarter 2016 Financial Results:
· Cash and cash equivalents as of March 31, 2016 totaled $6.2 million. AmpliPhi anticipates that its current financial resources will provide sufficient cash to fund operations into the third quarter of 2016
· Revenues related to sublicensing agreements from AmpliPhi’s former gene therapy program were $0.1 million for the quarter ended March 31, 2016 and for the same period in 2015
· Research and development expenses for the quarter ended March 31, 2016 totaled $2.0 million compared to $1.0 in the same period of 2015. The increase was primarily related to $0.2 million in higher compensation costs, $0.1 million of professional recruiting fees, and $0.4 million for the fair value from the assets acquired from Novolytics
· General and administrative expenses for the quarter ended March 31, 2016 were $2.6 million compared to $1.4 million for the same period of 2015. The increase was primarily attributable to an increase by $0.8 million in non-cash stock-based compensation related to two new executives, and $0.3 million in incremental legal, accounting and recruitment fees
· Loss from operations was $4.5 million during the three months ended March 31, 2016 which included $0.9 million of non-cash stock-based compensation, depreciation and amortization expense during the quarter
· There are currently 8.2 million shares of common stock outstanding
· AmpliPhi expects to file its Quarterly Report on Form 10-Q on May 12, 2016

8-K – Current report

On May 12, 2016 GlobeImmune, Inc. (NASDAQ: GBIM) reported an update on the Company’s business and clinical programs and announced financial results for the first quarter 2016 (Filing, Q1, GlobeImmune, 2016, MAY 12, 2016, View Source [SID:1234512345]).

The Company continues to seek potential strategic transactions. Cantor Fitzgerald & Co. has been retained by the Company to assist in reviewing ways to maximize stockholder value. There is not a defined timeline for the strategic review process and the review may not result in any specific action or transaction.

The Company has three ongoing clinical trials being conducted by the Company’s corporate collaborators, Gilead Sciences, Inc. and Celgene Corporation. GS-US-330-1401, the GS-4774 Phase 2 clinical trial in patients with chronic HBV infection who are currently not receiving treatment, is fully enrolled and the 48-week results are projected to be available in the second half of 2016. The 24-week results for this trial did not show a statistical difference between control and treatment arms. The results from a GI-6207 Phase 2 trial in subjects with medullary thyroid cancer are projected to be available in the second half of 2016. A Phase 2 clinical trial designed to investigate the safety and efficacy of evaluating GI-6301 in combination with radiation therapy in patients with chordoma is still enrolling patients.

Financial Results – First Quarter Ended March 31, 2016

GlobeImmune reported a net loss of $0.9 million for the three months ended March 31, 2016 compared to $1.6 million for the same period in 2015. The decrease in net loss for 2016 was due to lower compensation expense due to layoffs in research and development and general and administrative expenses. GlobeImmune reported a loss applicable to common stockholders of $0.15 per share, for three months ended March 31, 2016 compared to loss applicable to common stockholders of $0.27 per share for the same period in 2015.

Research and development for proprietary programs expense for the three months ended March 31, 2016 was $0.3 million compared to $0.4 million for the same period in 2015, a decrease of $0.1 million. The decrease was primarily due to a decrease in salary expense due to layoffs in 2015. Costs of manufacturing services for the three months ended March 31, 2016 were $0 compared to $0.2 million for the same period in 2015. The decrease was primarily due to a decrease in expenses relating to manufacturing services for Gilead for the Phase 2 HBV trial. Costs of collaboration license and services for the three months ended March 31, 2016 was $0.5 million compared to $0.9 million for the same period in 2015, a decrease of $0.4 million. The decrease was primarily due to a decrease in expenses related to the clinical trial for GS-4774 and reduction in salary expense due to layoffs in 2015. General and administrative expense for the three months ended March 31, 2016 was $0.9 million compared to $1.2 million for the same period in 2015, a decrease of $0.3 million. The decrease was due to a reduction in salary expense due to layoffs in 2015.

At March 31, 2016, GlobeImmune had cash and equivalents of $8.7 million. The Company believes it has sufficient cash to operate the company as a going concern through the middle of 2017 as it continues to evaluate strategic alternatives. If a strategic alternative is not found in the near future, we could decide to wind down the operations of the Company which will consume cash faster than currently planned as a going concern.

Mirna Therapeutics Reports First Quarter 2016 Financial Results and Program Updates

On May 12, 2016 Mirna Therapeutics, Inc. (Nasdaq:MIRN), a clinical stage biopharmaceutical company developing a broad pipeline of microRNA-based oncology therapeutics, reported financial results for the first quarter of 2016 and provided an update on recent developments (Press release, Mirna Therapeutics, MAY 12, 2016, View Source [SID:1234512339]).

"During the first quarter we continued to make progress advancing our lead product candidate MRX34, the first microRNA therapeutic in clinical development in cancer," commented Paul Lammers, M.D., M.Sc., Mirna’s President and CEO. "To date, MRX34 has produced clinically significant responses in patients with various types of late-stage cancers, demonstrating its ability to affect multiple pathways involved in cancer growth and immune evasion. We are continuing to advance MRX34 toward Phase 2 in late 2016 and also pushing ahead with our preclinical program to study its potential in combination with other cancer drugs."

Dr. Lammers continued, "On the corporate front, we were pleased to welcome Peter Greenleaf, Chief Executive Officer of Sucampo Pharmaceuticals, to our Board of Directors, and Dr. Vincent J. O’Neill as our new Chief Medical Officer. We look forward to their unique contributions as we advance our clinical development strategy in the promising new field of microRNA therapeutics."

FIRST QUARTER AND MRX34 PROGRAM UPDATES

Update from MRX34 Phase 1 trial planned at ASCO (Free ASCO Whitepaper). The Company expects to present additional clinical data from the ongoing Phase 1 trial at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in early June. To date, MRX34 has demonstrated compelling clinical results as a single agent therapy, including confirmed partial responses in patients with renal cell carcinoma, acral melanoma, and hepatocellular carcinoma. Additionally, several patients with solid tumors have achieved long-term stable disease during treatment with MRX34. These responses were observed in cancer patients with advanced Stage 4 metastatic disease, whose cancer had progressed after previously receiving multiple rounds of therapy. Top-line data from this study are expected in 2017.
Phase 1b translational medicine trial on track to begin in late 2016. This study will include serial tumor biopsies and is intended to develop deeper insights into the mechanism of action of MRX34 in melanoma patients and identify potential biomarkers of drug activity and treatment response.
MRX34 expected to begin Phase 2 by end of 2016. Studies in renal cell carcinoma and melanoma patients are being planned based on the responses observed to date in the ongoing Phase 1 trial, and on the high unmet medical need despite the availability of new therapies.
Preclinical studies ongoing of combination regimens with potential to enhance effectiveness of standard cancer therapies. At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in April, Mirna researchers presented in vitro findings that demonstrated the synergistic anticancer effects between MRX34 and platinum and other commonly used cytotoxic chemotherapy drugs across a range of non-small cell lung cancer (NSCLC) cell lines. Synergistic anticancer effects were also shown between MRX34 and tyrosine kinase inhibitors. These results suggest a broad potential for combination of MRX34 with other standard of care drug classes.

Preclinical studies are also ongoing to support selection of a second microRNA product candidate from the Company’s pipeline, with an Investigational New Drug (IND) application planned in late 2017.
CORPORATE UPDATES

Further strengthened management with the appointments of Peter Greenleaf to the Company’s Board of Directors and Dr. Vincent J. O’Neill to the role of Chief Medical Officer. Mr. Greenleaf, an industry veteran with over 20 years of experience in drug development and commercialization, including three years as President of Medimmune, currently serves as the Chief Executive Officer of Sucampo Pharmaceuticals. Dr. O’Neill is a medical oncologist with 15 years of therapeutic and diagnostic product development experience, and has held senior leadership roles at several global pharmaceutical companies, including Sanofi, Genentech and GlaxoSmithKline.
FIRST QUARTER 2016 FINANCIAL RESULTS

Cash Position and Guidance: Cash, cash equivalents, and marketable securities totaled $80.6 million as of March 31, 2016, compared to $89.7 million as of December 31, 2015. The Company has no debt. Based on the current operating plan, the Company expects that current cash resources will be sufficient to meet operating requirements into 2018.
Research and Development Expenses: Research and development expenses were approximately $4.5 million for the three months ended March 31, 2016 as compared to $3.4 million for the same period in 2015. The increase was primarily attributable to increased employee compensation expense due to a higher headcount to support the advancement of the Company’s clinical and preclinical development programs, as well as increases in manufacturing, clinical, and intellectual property costs.
General and Administrative Expenses: General and administrative expenses were approximately $2.1 million for the three months ended March 31, 2016 as compared to approximately $0.9 million for the same period in 2015. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to a higher headcount and higher outside professional and consulting costs, the majority of which were costs to comply with public company operating and reporting requirements.
Net Loss: Net loss was approximately $6.6 million for the first quarter of 2016 compared to $4.3 million in the comparable period in 2015. The results included non-cash, stock-based compensation charges of approximately $447,000 in the three months ended March 31, 2016 and approximately $134,000 in the same period in 2015.

Medigene reports results of first quarter 2016

On May 12, 2016 Medigene AG (MDG1, Frankfurt, Prime Standard), a clinical stage immuno-oncology company focusing on the development of T-cell immunotherapies for the treatment of cancer, reported financial results and corporate updates for the first quarter of 2016 (Press release, MediGene, MAY 12, 2016, View Source [SID:1234512338]).

Major events since the beginning of 2016:
Immunotherapies:

Phase II of Phase I/II trial with DC vaccine for the treatment of acute myeloid leukaemia (AML) initiated following positive recommendation by DSMB
Early IIT clinical data for DC vaccines presented at AACR (Free AACR Whitepaper) by academic partner Oslo University
DC patent portfolio strengthened
Additional viral vector production capacities secured for clinical TCR studies
Research collaboration started with University of Lausanne for TCRs
Company:

Management change: CSO Prof Dolores Schendel appointed CEO/CSO, Dave Lemus takes over as COO
Strengthened management team with appointment of three Senior Vice Presidents
Key figures in the first quarter of 2016:

Increase in total revenue by 132% to €3,909 k (3M 2015: €1,686 k)
Increase in R&D expenses for immunotherapies by 64% to €1,907 k (3M 2015: €1,166 k)
EBITDA loss reduced by 54% to €939 k (3M 2015: €2,042 k)
Cash and cash equivalents and time deposits of €46,310 k as at 31 March 2016 (31 December 2015: €46,759 k)
Confirmation of financial guidance 2016
Prof. Dr Dolores Schendel, Chief Executive Officer of Medigene AG, comments: "In the first quarter of 2016, the clinical development of our immunotherapy programmes progressed further. We are making extensive efforts now towards putting into practice our first clinical studies with TCRs. As Medigene’s new CEO, I’m delighted about these important examples of progress."

Dave Lemus, Chief Operating Officer of Medigene AG, adds: "Medigene’s transformation into a leading immuno-oncology company begins increasingly to take shape. Accordingly, the planned increase in expenses for immunotherapy-driven research and development in the first quarter of 2016 evidences this change, and is enabled by the company’s positive financial condition."

Key figures in Q1 2016:

IN € K Q1 2016

Q1 2015

Change

Results of operations
Total revenue 3,909 1,686 132%
Veregen revenue 624 714 -13%
Other operating income 3,285 972 >200%
thereof gain on sale of intangible assets, net (EndoTAG) 2,365 0 -
Gross profit 3,594 1,418 153%
Selling and general administrative expenses -2,312 -1,740 33%
Research and development expenses -2,395 -1,932 24%
Operating result -1,113 -2,254 -51%
Net profit/loss for the period -1,387 -3,672 -62%
EBITDA -939 -2,042 -54%
Earnings per share (€) -0.07 -0.26 -73%
Personnel expenses -2,452 -1,758 39%

Cash flows
Net cash used in operating activities -1,064 -1,777 -40%
Net cash provided by/used in investing activities 626 -53 >-200%
Net cash used in financing activities -11 0 -

Balance sheet data as at 31 March 2016 and 31 December 2015
Cash and cash equivalents and time deposits 46,310 46,759 -1%
Total assets 110,095 113,531 -3%
Current liabilities 9,013 9,664 -7%
Non-current liabilities 12,970 13,879 -7%
Shareholders’ equity 88,112 89,988 -2%
Equity ratio (%) 80 79 1%

Employees as at 31 March 81 69 17%
FTE as at 31 March 75 62 20%

Medigene share as at 31 March
Total number of shares outstanding 19,688,202 13,956,417 41%
The Company generates its revenue and other operating income from its non-core business. Total revenue of the Company increased by 132% to €3,909 k in the reporting period (3M 2015: €1,686 k) on account of extraordinary effects related to the sale of EndoTAG agreed in December 2015.

Medigene increased research and development expenses by 24% in the first three months of 2016 to €2,395 k (3M 2015: €1,932 k). The increase in these expenses is mainly due to the intended increase in expenditure for preclinical and clinical trials for Medigene’s immunotherapies, which increased significantly by 64% to €1,907 k in the first three months of 2016 (3M 2015: €1,166 k).

In spite of higher development expenses for its immunotherapy programmes, Medigene reduced its EBITDA loss in the first three months of 2016 by 54% to €939 k (3M 2015: EBITDA loss of €2,042 k). Medigene’s EBITDA is derived from the net profit/loss for the period; it does not include any taxes, financial result, foreign exchange gains or losses, share of result of associates, or depreciation or amortisation.

Medigene reduced its net loss in the first three months of 2016 by 62% to €1,387 k (3M 2015: €3,672 k).

Medigene reduced its net cash used in operating activities significantly in the first three months of 2016 to €1,064 k (3M 2015: €1,777 k). This represents an average monthly cash outflow of €0.4 m in the first three months of 2016 (3M 2015: €0.6 m). The major part of the cash used was directed at research and development as well as sales and administration.

The cash and cash equivalents and time deposits of the Company amounted to €46,310 k as at the end of the reporting period (31/12/2015: €46,759 k).

Financial forecast 2016:
Medigene confirms its financial guidance for 2016 published in the 2015 annual report. In 2016, Medigene is planning to expand its clinical development programs, which will significantly increase R&D expenses in the field of immunotherapies to €9 – 11 m (2015: €5.5 m). The EBITDA loss is estimated to be €10 -12 m (2015: €9.5 m).

Medigene expects total Veregen revenue of €3 – 4 m (2015: €3.1 m), and stable or increasing total revenue for 2016 (2015: €6.8 m). Since this revenue is not generated in the Company’s core business of immunotherapies, these figures are not indicative of the same. This financial guidance does not include any possible proceeds from potential new partnership agreements, or any exchange rate fluctuation.

The detailed Q1 statement 2016 is available online at:
www.annualreport2015.medigene.com View Source

Press and analysts’ conference call: A press and analysts conference call (in English) will be held today at 3:00 pm CEST / 9:00 am EDT (USA) and will be webcast live. Please access the synchronized presentation slides and a recording via Medigene’s website, www.medigene.com.