On November 30, 2015 Boehringer Ingelheim reported insights from experts working on-the-ground in lung cancer, compiled by Boehringer Ingelheim, shed light on challenges faced by patients with advanced stage adenocarcinoma, a type of non-small cell lung cancer (NSCLC), in being tested for EGFR mutations, leaving some without access to the most appropriate treatment for them (Press release, Boehringer Ingelheim, NOV 29, 2015, View Source [SID:1234508356]).

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A new insights report sees healthcare professionals and patient group representatives reflect on existing gaps in nationwide EGFR testing, which should be conducted for eligible patients upon diagnosis according to guidelines.1

Dr Matthew Peters, Professor of Respiratory Medicine, Macquarie University Australia and Chair, Global Lung Cancer Coalition commented, "Whilst country-level insights within this report do vary, common barriers to achieving EGFR testing and personalised treatment for all eligible patients are clear. Education is key: for patients, healthcare professionals and the wider community. More is needed to bring us closer to helping each and every NSCLC patient receive the right treatment for them."

Country-level themes identified by experts from Western Europe and North America include:

Germany: insufficient reimbursement for mutation tests impacts EGFR testing rates for inpatients (majority of patients are diagnosed in an inpatient setting)

Italy: high number of patients are tested for EGFR mutations, but some patients and caregivers ask to start first-line treatment before receiving test results

Spain: many cancer centres are currently achieving high EGFR testing rates, but disparity exists as some centres are increasingly impacted by the growing economic crisis

UK: despite high levels of EGFR testing, some patients start treatment before results of this testing are available, so treatment is not personalised for their mutation

Canada: a healthcare system which is publically funded through each province and territory brings forth challenges in ensuring all eligible patients across the country are EGFR tested at diagnosis

USA: a substantial percentage of treatment decisions are not based on EGFR mutation subtype resulting in patients not receiving the most appropriate personalised treatment plans

Experts unite with a shared vision where all eligible NSCLC patients have the most appropriate diagnostic tests and access to personalised treatment. Common barriers holding back the realisation of this vision include difficulties in obtaining adequate tumour tissue samples to test, and delays in receiving test results. This is important as data have shown that EGFR targeted treatments significantly delay disease progression when compared to chemotherapy. For a specific targeted therapy, extended overall survival of patients with the most common type of mutation (del19) when compared to chemotherapy has also been observed.2

Dr Mehdi Shahidi, Medical Head, Solid Tumour Oncology, Boehringer Ingelheim added, "EGFR testing rates have improved dramatically over recent years, but there is still work to be done. Targeted therapies can benefit patients with EGFR mutation-positive lung cancer by delaying disease progression and for some, offering survival advantages compared to chemotherapy. This is why EGFR mutation testing and acting on the results of these tests is so important. Healthcare teams need to optimise the tools, resources and tests available to improve access to personalised treatment, for the ultimate benefit of patients."

On November 30, 2015 Novartis reported that it will demonstrate the strength of its research program and portfolio at the 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Novartis, NOV 29, 2015, View Source [SID:1234508354]). Presentations will highlight data across leukemias, lymphomas and myelomas as well as supportive care, including key findings in rare and difficult-to-treat patient populations, in addition to personalized cell therapies. The ASH (Free ASH Whitepaper) Annual Meeting will be held December 5-8 in Orlando, Florida.

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"We pride ourselves in our drive for new science and innovation, and look forward to bringing this promise to life at ASH (Free ASH Whitepaper) through the results of our latest hematology research," said Bruno Strigini, President, Novartis Oncology. "We will be presenting encouraging overall survival data for both investigational and approved products, underscoring our commitment to improve and extend people’s lives."

A key focus in hematology for Novartis is developing therapies for rare and difficult-to-treat patient populations. The acute myeloid leukemia (AML) community in particular is in need of new treatment options as the general therapeutic strategy has remained unchanged for more than 25 years[1],[2]. Further, about one-third of AML patients harbor a FLT3 mutation, which is associated with poorer prognoses than in those without the mutation[3],[4]. To this end, key data on PKC412 (midostaurin) from the following two studies will be presented:

The Multi-Kinase Inhibitor Midostaurin (M) Prolongs Survival Compared with Placebo (P) in Combination with Daunorubicin (D)/Cytarabine (C) Induction (ind), High-Dose C Consolidation (consol), and As Maintenance (maint) Therapy in Newly Diagnosed Acute Myeloid Leukemia (AML) Patients (pts) Age 18-60 with FLT3 Mutations (muts): An International Prospective Randomized (rand) P-Controlled Double-Blind Trial (CALGB 10603/RATIFY [Alliance]) (Abstract #6, Plenary Session, Sunday, December 6, 2:00 – 4:00 pm EST)

Midostaurin in Combination with Intensive Induction and As Single Agent Maintenance Therapy after Consolidation Therapy with Allogeneic Hematopoietic Stem Cell Transplantation or High-Dose Cytarabine (NCT01477606) (Abstract #322, Oral Presentation, Sunday, December 6, 5:15 pm EST)

Novartis and the University of Pennsylvania’s Perelman School of Medicine (Penn) have an exclusive global collaboration to research, develop and commercialize chimeric antigen receptor (CAR) T cell therapies for the investigational treatment of cancers. New data on investigational CART therapy CTL019, as well as cell processing technology will be presented at ASH (Free ASH Whitepaper) including:

Durable Remissions in Children with Relapsed/Refractory ALL Treated with T Cells Engineered with a CD19-Targeted Chimeric Antigen Receptor (CTL019) (Abstract #681, Oral Presentation, Monday, December 7, 3:15 pm EST)

Sustained Remissions Following Chimeric Antigen Receptor Modified T Cells Directed Against CD19 (CTL019) in Patients with Relapsed or Refractory CD19+ Lymphomas (Abstract #183, Oral Presentation, Sunday, December 6, 8:00 am EST)

Successful Translation of Chimeric Antigen Receptor (CAR) Targeting CD19 (CTL019) Cell Processing Technology from Academia to Industry (Abstract #3100, Poster Presentation, Sunday, December 6, 6:00 – 8:00 pm EST)

Novartis will share new research for pipeline compound ABL001, a small molecule designed to inhibit BCR-ABL. ABL001 is different from Glivec (imatinib)* and Tasigna (nilotinib) as it binds to a unique region of BCR-ABL, forcing a conformational change that disables the protein’s active site. As part of Novartis’ ongoing commitment to chronic myeloid leukemia (CML) research, ABL001 represents the company’s evolving science and is being investigated in Phase I trials:

ABL001, a Potent, Allosteric Inhibitor of BCR-ABL, Exhibits Safety and Promising Single- Agent Activity in a Phase I Study of Patients with CML with Failure of Prior TKI Therapy (Abstract #138, Oral Presentation, Saturday, December 5, 5:15 pm EST)
Novartis will also be presenting safety and efficacy data, including long-term studies, at ASH (Free ASH Whitepaper) from its approved hematological treatments:

Jakavi (ruxolitinib)**

Long-Term Efficacy and Safety in COMFORT-II, a Phase 3 Study Comparing Ruxolitinib with Best Available Therapy for the Treatment of Myelofibrosis: 5-Year Final Study Results (Abstract #59, Oral Presentation, Saturday, December 5, 10:30 am EST)
Safety and Efficacy of Ruxolitinib in an 1869-Patient Cohort of JUMP: An Open-Label, Multicenter, Single-Arm, Expanded-Access Study in Patients with Myelofibrosis (Abstract #2799, Poster Presentation, Sunday, December 6, 6:00 – 8:00 pm EST)
Demographics, Baseline (BL) Characteristics, and Disease Symptom Burden in RESPONSE-2: A Randomized, Phase 3 Study of Ruxolitinib in Polycythemia Vera Patients (pts) Who Are Resistant to or Intolerant of Hydroxyurea (HU) (Abstract #2807, Poster Presentation, Sunday, December 6, 6:00 – 8:00 pm EST)

Continued Treatment With Ruxolitinib Provides Additional Hematocrit Control and Spleen Volume Responses in Patients with PV Treated in the RESPONSE Study (Abstract #2804, Poster Presentation, Sunday, December 6, 6:00 – 8:00 pm EST)

Tasigna (nilotinib)

Dose-Optimized Nilotinib (NIL) in Patients (pts) with Newly Diagnosed Chronic Myeloid Leukemia in Chronic Phase (CML-CP): Final results from ENESTxtnd study (Abstract #344, Oral Presentation, Sunday, December 6, 4:45 pm EST)

Impact of Age on Efficacy and Toxicity of Nilotinib in Patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP): ENEST1st Sub-Analysis (Abstract #479, Oral Presentation, Monday, December 7, 8:00 am EST)

International Scale (IS)-Standardized BCR-ABL1 Digital Polymerase Chain Reaction (dPCR) Assays Using ABL1, BCR, and GUS Control Genes for Measuring Deep Molecular Response (MR) in Chronic Myeloid Leukemia (CML) (Abstract #136, Oral Presentation, Saturday, December 5, 4:45 pm EST)

Quantification of BCR-ABL with Digital PCR Results in a Significantly Lower Rate of Deep Molecular Response when Compared to RT-qPCR in CML Patients Treated in the ENEST1st Trial (Abstract #135, Oral Presentation, Saturday, December 5, 4:30 pm EST)
Farydak (panobinostat)

Final Analysis of Overall Survival from the Phase 3 Panorama 1 Trial of Panobinostat Plus Bortezomib and Dexamethasone Versus Placebo Plus Bortezomib and Dexamethasone in Patients with Relapsed or Relapsed and Refractory Multiple Myeloma (Abstract #3026, Poster Presentation, Sunday, December 6, 6:00 – 8:00 pm EST)
Analysis of Outcomes Based on Response in Patients with Relapsed or Relapsed and Refractory Multiple Myeloma Treated with Panobinostat or Placebo in Combination with Bortezomib and Dexamethasone in the Panorama 1 Trial: Updated Analysis Based on Prior Treatment (Abstract #4230, Poster Presentation, Monday, December 7, 6:00 – 8:00 pm EST)
In addition, Sandoz, a Novartis company and the global leader in biosimilars, will present data from PROTECT 2, one of their pivotal Phase III trials investigating use of their proposed pegfilgrastim biosimilar in patients with chemotherapy-induced neutropenia.

Proposed Biosimilar Pegfilgrastim (LA-EP2006) and Reference Pegfilgrastim for the Prevention of Neutropenia in Patients with Breast Cancer: A Randomized, Double-Blind Trial. PROTECT 2: Pegfilgrastim Randomized Oncology (supportive care) Trial to Evaluate Comparative Treatment Results (Abstract #632, Oral Presentation, Monday, December 7, 10:45 am EST)
Throughout ASH (Free ASH Whitepaper) 2015, Novartis Oncology will host dedicated content on the company website (View Source) that will feature unique insights and perspectives on emerging areas of cancer care and research.

Product Information
Approved indications for products vary by country and not all indications are available in every country. The product safety and efficacy profiles have not yet been established outside the approved indications. Because of the uncertainty of clinical trials, there is no guarantee that compounds will become commercially available with additional indications.

For full prescribing information, including approved indications and important safety information about marketed products, please visit
View Source

Because PKC412 (midostaurin), CTL019 and ABL001 are investigational compounds, the safety and efficacy profiles have not yet been fully established. Access to these investigational compounds is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the compound. Because of the uncertainty of clinical trials, there is no guarantee that PKC412 (midostaurin), CTL019 and ABL001 will ever be commercially available anywhere in the world.

On April 28, 2015 Co-D Therapeutics, Inc., a pre-clinical stage pharmaceutical company developing agents for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Co-D’s request for orphan drug designation of Triolimus for the treatment of the highly lethal malignancy, angiosarcoma (Press release, Co-D Therapeutics, 28 11, 2015, View Source [SID1234516124]).

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"We are pleased that Triolimus has been designated an orphan drug for the treatment of angiosarcoma," commented Dr. Kevin Kozak, co-founder and Chief Medical Officer of Co-D Therapeutics. "With few effective options, new agents for angiosarcoma treatment are urgently required. The orphan designation reflects both the FDA’s and Co-D’s commitment to addressing the unmet clinical needs of patients with rare diseases like angiosarcoma."

The Orphan Drug Act provides for economic incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the United States. Orphan drug designation will entitle Co-D to seven years of market exclusivity for Triolimus as a treatment for angiosarcoma following marketing approval by the FDA. Additional benefits include tax credits related to clinical trial expenses, potential exemption from the FDA-user fee, assistance in clinical trial design, and smaller trials required for new drug applications.

"The designation of Triolimus as an orphan-drug for angiosarcoma treatment is an early and critical milestone and will facilitate progress through the clinical and regulatory development process," said Abdalla Saad, co-founder and Chief Executive Officer of Co-D Therapeutics. "We find our preclinical data compelling and are thrilled the FDA judged Triolimus meritorious of orphan-drug designation."

Triolimus technology was developed by Prof. Glen Kwon at the University of Wisconsin-Madison. "We are excited to continue the enormously rewarding process of advancing Triolimus to the clinic," said co-founder and Chief Scientific Officer Prof. Kwon.

Triolimus’ intellectual property portfolio will be licensed from the Wisconsin Alumni Research Foundation (WARF). Funding from WARF’s Accelerator Program supported pre-clinical development of Triolimus.

About Angiosarcoma

Angiosarcoma is a rare, aggressive malignancy of endothelial cell differentiation that has a propensity for both local recurrence as well as regional and distant metastases. Long-term disease-free survival is achieved in only half of patients who present with apparently localized disease. Average survival in patients who present with distant disease is less than one year. This poor survival is, in large part, attributable to a dearth of currently available, effective treatment options.

About Triolimus

Triolimus is a novel "3-in-1" nanoparticle that contains three proven anti-cancer agents in a non-toxic carrier. Triolimus is designed to be superior to currently available chemotherapies through both the elimination of toxic excipients often required for intravenous use and through the delivery of three, complementary drugs to maximize anti-cancer effects.

On November 27, 2015 Can-Fite BioPharma Ltd. (NYSE MKT: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs being developed to treat inflammatory diseases, cancer and sexual dysfunction, reported financial results for the nine months ended September 30, 2015 and updates on its drug development programs (Filing, 6-K, Can-Fite BioPharma, NOV 27, 2015, View Source [SID:1234508352]).

Clinical Development Program and Corporate Highlights Include:

● Fortifies Balance Sheet with $13.8 Million Fund Raise
During September 2015, Can-Fite closed on approximately $9 million in funding from institutional investors. As of September 30, 2015 Can-Fite had cash and cash equivalents of $14.46 million. In October the Company raised an additional $4.8 million.

● CF102 – Receives Fast Track Designation in U.S. & Orphan Drug Designation in Europe; Reports Compelling Preclinical Data and Files Patent for NASH
During, and immediately following, the third quarter of 2015, three significant events moved CF102’s clinical development forward. 1) The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to CF102 as a second line treatment for hepatocellular carcinoma (HCC), the most common form of liver cancer. With Fast Track CF102 benefits from more frequent meetings and communications with the FDA to review the drug’s development plan to support approval, while also allowing the Company to submit parts of the New Drug Application (NDA) on a rolling basis for review as data becomes available. CF102 also has the FDA’s Orphan Drug Designation. 2) In October 2015, the European Medicines Agency (EMA) granted Orphan Drug Designation to CF102 for the indication of HCC, giving CF102 protocol assistances and a 10-year market exclusivity following market approval in 28 EU member states and 3 additional European Economic Area countries. Can-Fite continues to enroll and dose patients in its global Phase II liver cancer study. Approximately 78 patients are expected to be enrolled in the trial in the U.S., Europe, and Israel by June 30, 2016. 3) In November 2015, the Company reported data from a preclinical study of an animal model of non-alcoholic steatohepatitis (NASH), revealing CF102 had a statistically significant reduction in liver pathology. This data supports the development of CF102 for NASH, which represents a large and unmet medical need, with no U.S. FDA approved treatment currently available. The addressable market for the treatment of NASH in 2025 is estimated at $35-$40 billion by Deutsche Bank. Can-Fite filed a patent for CF102 in the treatment of NASH.

● CF602 – Reports Mechanism of Action Study Data for Upcoming IND Submission
In October 2015, Can-Fite reported new findings for its CF602 drug candidate showing a defined mechanism of action in erectile dysfunction similar to sildenafil (Viagra) in a rat model of diabetes mellitus. CF602 demonstrated effects on erection superior to that of Viagra in animal studies. Viagra, sold by Pfizer, generated global sales of $1.685 billion in 2014. Can-Fite plans to file an Investigational New Drug (IND) application with the FDA for CF602 in the third quarter of 2016.

● CF101 – Preparing for Phase III trials in Rheumatoid Arthritis & Psoriasis
Can-Fite is currently preparing the protocol for its Phase III trial of CF101 in the treatment of psoriasis. Protocol design is scheduled for completion by the end of 2015. Having already completed the Phase III protocol for CF101 in the treatment of rheumatoid arthritis, Can-Fite plans to submit this protocol to Institutional Review Boards (IRBs) during in the fourth quarter of 2015. Marking an important step for CF101 prior to coming to market, "piclidenoson" was accepted as the drug’s proposed generic by the World Health Organization.

● Enrolling Patients in Ongoing Phase II Glaucoma Study by Can-Fite Subsidiary OphthaliX
OphthaliX continues to enroll patients in a Phase II clinical study of CF101 for glaucoma and data release is expected during the first half of 2016.

"During and immediately following the third quarter, we achieved regulatory milestones for CF102 that we believe will significantly accelerate our liver cancer drug’s time to market. Given the lack of effective medications for liver cancer, we are pleased that Fast Track designation in the U.S. and Orphan Drug designation in Europe are designed to expedite CF102’s pathway through advanced clinical trials and into market approval. NASH, another large and unmet medical need, has just emerged as a potential new indication for CF102 based on compelling new preclinical data," stated Can-Fite CEO Dr. Pnina Fishman. "We also very encouraged by the new mechanism of action data we reported for CF602 in sexual dysfunction. These animal studies demonstrated that CF602 produced erectile effects superior to Viagra and therefore we believe it has the potential to offer value in the market. We are currently preparing CF602’s IND for the indication of sexual dysfunction."

"As we look ahead to 2016, we are preparing to commence Phase III trials for CF101 in both psoriasis and rheumatoid arthritis. With a portfolio of indications that are all advancing towards market, we were pleased to fortify our balance sheet with $13.8 million from institutional investors," Dr. Fishman added.

Research and development expenses for the nine months ended September 30, 2015 were NIS 9.58 million (U.S. $2.44 million) compared with NIS 12.44 million (U.S. $3.17 million) for the same period in 2014. Research and developments expenses for the nine months of 2015 comprised primarily of expenses associated with the Phase II study for CF102 as well as expenses for ongoing studies of CF101. The decrease is primarily due to the completion of the Phase II/III psoriasis study during the first quarter of 2015 and a decrease in the scope of the non-clinical expenses during the first nine months of 2015 as compared to the parallel period in 2014.

General and administrative expenses were NIS 6.79 million (U.S. $1.73 million) for the nine months ended September 30, 2015 compared to NIS 7.73 million (U.S. $1.97 million) for the same period in 2014. The decrease is primarily due to a reduction in salary and professional services expenses.

Financial expenses, net for the nine months ended September 30, 2015 aggregated NIS 4.70 million (U.S. $1.20 million) compared to financial income, net of NIS 3.28 million (U.S. $0.84 million) for the same period in 2014. The increase in financial expenses, net in the nine months of 2015 was mainly due to an increase in the fair value of warrants that are accounted as financial liability.

Can-Fite’s net loss for the nine months ended September 30, 2015 was NIS 20.53 million (U.S. $5.23 million) compared with a net loss of NIS 16.89 million (U.S. $4.31 million) for the same period in 2014. The increase in net loss for the nine months of 2015, was primarily attributable to an increase in finance expenses, net offset by decreases in operating expenses.

As of September 30, 2015, Can-Fite had cash and cash equivalents of NIS 56.73 million (U.S. $14.46 million) as compared to NIS 36.09 million (U.S. $9.20 million) at December 31, 2014. The increase in cash during the nine months ended September 30, 2015 is due to NIS 32.35 million ($8.25 million) received from issuance of shares and warrants, net of issuance expenses and NIS 5.14 million (U.S. $1.31 million) received from Cipher Pharmaceuticals as upfront payment for entering into the distribution agreement with Cipher, offset by operating expenses. An additional $4.3 million, net was raised in October 2015, following the end of the third quarter.

For the convenience of the reader, the reported NIS amounts have been translated into U.S. dollars, at the representative rate of exchange on September 30, 2015 (U.S. $ 1 = NIS 3.923).

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The Company’s consolidated financial results for the nine months ended September 30, 2015 are presented in accordance with International Financial Reporting Standards.