On October 16, 2017 Kura Oncology, Inc. (Nasdaq:KURA), a clinical stage biopharmaceutical company focused on the development of precision medicines for oncology, reported presentations at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), taking place October 26-30, 2017 in Philadelphia (Press release, Kura Oncology, OCT 16, 2017, View Source;p=RssLanding&cat=news&id=2308717 [SID1234520946]).

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A late-breaking poster will be presented on Saturday, October 28, by Dr. Alan Ho of Memorial Sloan Kettering Cancer Center and will feature data from Kura’s recently-announced positive Phase 2 proof-of-concept trial in head and neck squamous cell carcinoma (HNSCC) with HRAS mutations. In addition, the poster has been selected as the subject of a short, oral presentation during the Spotlight on Proffered Papers Session 1 on Friday, October 27.

A second late-breaking poster will be presented on Saturday, October 28, and will feature preclinical data for KO-539, Kura’s potent and selective inhibitor of the menin-MLL interaction, supporting the potential clinical utility of KO-539 in NPM1- and DNMT3A-mutant AML.

The schedule and locations for the late-breaking oral presentation and poster presentations is as follows:

Oral Presentation:

Title of Presentation: Preliminary results from a Phase 2 proof-of-concept trial of tipifarnib in tumors with HRAS mutations
Date & Time: Friday, October 27, 2017, 10:50 a.m. – 12:20 p.m. EDT
Presenter: Alan L. Ho, M.D., Ph.D., Memorial Sloan Kettering Cancer Center
Session: Spotlight on Proffered Papers Session 1
Location: Terrace Ballroom, 400 Level, Pennsylvania Convention Center

Poster Presentations:

Title of Poster: Preliminary results from a Phase 2 proof-of-concept trial of tipifarnib in tumors with HRAS mutations
Date & Time: Saturday, October 28, 12:30 p.m. – 4:00 p.m. EDT
Presenter: Alan L. Ho, M.D., Ph.D., Memorial Sloan Kettering Cancer Center
Session: Late-Breaking Poster Session A (Clinical Trials)
Abstract Number: LB-A10
Location: Hall E, Pennsylvania Convention Center

Title of Poster: A novel small molecule menin-MLL inhibitor for potential treatment of MLL-rearranged leukemias and NPM1/DNMT3A-mutant AML
Date & Time: Saturday, October 28, 12:30 p.m. – 4:00 p.m. EDT
Presenter: Francis Burrows, Ph.D., Kura Oncology
Late-Breaking Poster Session A (Epigenetic Targets)
Abstract Number: LB-A27
Location: Hall E, Pennsylvania Convention Center

On October 16, 2017 Atossa Genetics (NASDAQ:ATOS), a clinical-stage pharmaceutical company developing novel therapeutics and delivery methods for breast cancer and other breast conditions, reported that the Ernest Mario School of Pharmacy at Rutgers, The State University at New Jersey, plans to conduct a study utilizing Atossa’s intraductal microcatheter technology (Press release, Atossa Genetics, OCT 16, 2017, View Source [SID1234520936]).

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The Rutgers researchers believe that directly administering drugs into the breast duct where breast cancer grows, by inserting microcatheters into the nipple, is a better alternative than systemic administration, because the drugs will be directly delivered to the tissue. The Rutgers program uses a unique directed delivery system comprised of nanoscale pharmaceutical carriers loaded with single drugs. The long-term goal of the research program is to develop a locally administered drug delivery system that selectively targets and delivers pathway-specific targeting therapeutics to eliminate breast cancer cells and cancer stem-like cells while sparing normal breast cells.

"We are encouraged that a leading research institution like Rutgers recognizes the potential merit of our microcatheter technology. Atossa fully supports additional research utilizing our patented microcatheter technology," stated Steven Quay, MD, PhD, Atossa CEO and President.

The Rutgers program is in the research and development phase and has not been approved by the FDA or any other regulatory body. Studies demonstrating safety and efficacy, among other things, and regulatory approvals will be required before commercialization.

On 16 October, 2017 Ipsen (Euronext: IPN; ADR: IPSEY) and its partner Exelixis (NASDAQ: EXEL) reported that its global phase 3 CELESTIAL trial met its primary endpoint of overall survival (OS), with cabozantinib providing a statistically significant and clinically meaningful improvement in median OS compared to placebo in patients with advanced hepatocellular carcinoma (HCC) (Press release, Ipsen, OCT 16, 2017, View Source [SID1234520945]). The independent data monitoring committee for the study recommended that the trial should be stopped for efficacy following review of the second planned interim analysis. CELESTIAL is a randomized, global phase 3 trial of cabozantinib versus placebo in patients with advanced HCC who have been previously treated with sorafenib. The safety data in the study were consistent with the established profile of cabozantinib.

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In line with and in collaboration with our partner Exelixis, Ipsen expects to file in the first half of 2018 a variation of the initial application to the EMA and other relevant regulatory agencies and to evaluate potential next steps in the development strategy for cabozantinib outside the United States and Japan as a treatment for advanced HCC in patients who have been previously treated. Detailed results from CELESTIAL will be submitted for presentation at a future medical conference.

Alexandre Lebeaut, MD, Executive Vice-President, R&D, Chief Scientific Officer, Ipsen, said: "Liver cancer is one of the leading causes of cancer deaths worldwide and more effective treatment options are urgently needed. We are pleased to report that in the CELESTIAL clinical study cabozantinib has been shown to provide a survival benefit and therefore has the potential to bring a new oral systemic treatment to previously treated patients with advanced liver cancer. "

About the CELESTIAL Study

CELESTIAL is a randomized, double-blind, placebo-controlled study of cabozantinib in patients with advanced HCC conducted at more than 100 sites globally in 19 countries. The trial was designed to enroll 760 patients with advanced HCC who previously received sorafenib and may have received up to two prior systemic cancer therapies for HCC and had adequate liver function. Enrollment of the trial was completed in September 2017, and 773 patients were ultimately randomized. Patients were randomized 2:1 to receive 60 mg of cabozantinib once daily or placebo and were stratified based on etiology of the disease (hepatitis C, hepatitis B or other), geographic region (Asia versus other regions) and presence of extrahepatic spread and/or macrovascular invasion (yes or no). No cross-over was allowed between the study arms.

The primary endpoint for the trial is OS, and secondary endpoints include objective response rate and progression-free survival. Exploratory endpoints include patient-reported outcomes, biomarkers and safety.

Based on available clinical trial data from various published trials conducted in the second-line setting of advanced HCC, the CELESTIAL trial statistics for the primary endpoint of OS assumed a median OS of 8.2 months for the placebo arm. A total of 621 events provide the study with 90 percent power to detect a 32 percent increase in median OS (HR = 0.76) at the final analysis. Two interim analyses were planned and conducted at 50 percent and 75 percent of the planned 621 events.

About HCC

Hepatocellular Carcinoma (HCC) is the most common form of liver cancer in adults.1 The disease originates in cells called hepatocytes found in the liver. With approximately 800’000 new cases diagnosed each year, HCC is the sixth most common cancer and the second-leading cause of cancer deaths worldwide.2,3 According to the GLOBOCAN data, it is estimated that across the European Union (EU-28) nearly 60’000 new patients will be diagnosed with liver cancer in 2020.4 Without treatment, patients with the disease in advanced stage usually survive between 4 and 8 months.5

About CABOMETYX (cabozantinib)

Cabometyx is an oral small molecule inhibitor of receptors, including VEGFR, MET, AXL and RET. In preclinical models, cabozantinib has been shown to inhibit the activity of these receptors, which are involved in normal cellular function and pathologic processes such as tumor angiogenesis, invasiveness, metastasis and drug resistance.

In February of 2016, Exelixis and Ipsen jointly announced an exclusive licensing agreement for the commercialization and further development of cabozantinib indications outside of the United States, Canada and Japan. This agreement was amended in December of 2016 to include commercialization rights for Ipsen in Canada. On April 25, 2016, the FDA approved Cabometyx tablets for the treatment of patients with advanced RCC who have received prior anti-angiogenic therapy and on September 9, 2016, the European Commission approved Cabometyx tablets for the treatment of advanced RCC in adults who have received prior vascular endothelial growth factor (VEGF)-targeted therapy in the European Union, Norway and Iceland. Cabometyx is available in 20 mg, 40 mg or 60 mg doses. The recommended dose is 60 mg orally, once daily.

Ipsen also submitted to European Medicines Agency (EMA) the regulatory dossier for cabozantinib as a treatment for first-line advanced RCC in the European Union on August 28, 2017; on September 8, 2017, Ipsen announced that the EMA validated the application.

Cabozantinib is not approved for the treatment of advanced hepatocellular carcinoma.

On October 13, 2017 Impact Biomedicines ("Impact") reported its launch to pioneer the development of life-changing treatments for patients with myeloproliferative neoplasms and other cancers (Press release, Impact Biomedicines, OCT 13, 2017, View Source [SID1234520974]). The Company’s pipeline is centered around fedratinib, a potent and highly selective oral small molecule JAK2 kinase inhibitor that is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV). In conjunction with this launch, Impact is pleased to share that the U.S. Food and Drug Administration (FDA) has removed the clinical hold placed on fedratinib and that the company has received $22.5 million from Medicxi through a Series A financing.

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Impact was formed in 2016 after acquisition of Sanofi’s full rights for the global development and commercialization of fedratinib. The majority equity holders in Impact include the co-founders and Medicxi, with Sanofi retaining a minority stake.

In 2013, the development of fedratinib was discontinued by Sanofi after the FDA issued a clinical hold subsequent to reports of a few potential cases of Wernicke’s encephalopathy (WE), an acute neurological condition usually indicative of a vitamin B deficiency, in patients participating in fedratinib clinical trials. Following a Type A meeting and review of additional data, the FDA concluded that Impact provided the necessary documentation to support lifting the clinical hold – opening the path for Impact’s continued development of fedratinib.

The clinical package for fedratinib includes data from a total of 18 studies completed in 877 subjects. In JAKARTA-1, a completed international Phase 3 pivotal trial for the treatment of myelofibrosis, fedratinib met its primary and secondary endpoints by reducing spleen size in 47% of patients by ≥35% at 24 weeks (p<0.0001) and improving symptom score in 36% of patients by ≥50% at 24 weeks (p< 0.0001). Comparable responses were seen in patients with normal or low platelet counts and thrombocytopenia was similar between placebo and the target dose of 400mg. In JAKARTA-2, a follow-on study in patients who were unresponsive to all other available therapies, including patients who were either Jakafi (ruxolitinib) resistant or intolerant, fedratinib showed similar activity. In that study, 55% of patients who had failed or were intolerant to ruxolitinib experienced a spleen size reduction of ≥35% with fedratinib. Notably, responses were noted in 63% of patients intolerant to ruxolitinib and 61% of patients who had lost ruxolitinib response. Currently, ruxolitinib is the only drug approved by the FDA to treat patients with MF and PV. The most common adverse events for fedratinib were hematological (anemia) and gastrointestinal (nausea, diarrhea and vomiting). The results of these trials have been published in leading peer-reviewed journals.

"The decision to discontinue the development of fedratinib in 2013 was heartbreaking for the patients who were experiencing positive responses while in clinical trials. There are very limited therapeutic options for these patients and fedratinib was active in most patients when nothing else had worked. Because of the very high unmet medical need in MF, the Impact team completed a thorough review of the available data, including careful due diligence into the potential cases of WE and I am glad to report that as a result of this effort, the FDA has lifted the clinical hold," said John Hood, Ph.D., Chief Executive Officer of Impact. "In addition to MF, Impact intends to pursue multiple clinical indications for fedratinib to realize its potential as a best-in-class JAK2 kinase inhibitor."

To support the Company’s drug development and manufacturing efforts and the build out of the management team, Impact closed a $22 million Series A financing with Medicxi.

"We’ve made it a point to double down and invest more in maturing life science companies who are delivering innovation where it’s needed most. In line with our ‘asset-centric’ approach, we believe Impact is doing just that by reviving fedratinib with a thoughtful development approach and due diligence," said Kevin Johnson, Impact board member and Co-Founder of Medixci. "We are confident that the dedicated team at Impact has the vision and skill to create a sustainable long-term business around fedratinib."

The development of fedratinib is being led by a highly skilled and devoted team, including members of the original TargeGen team, where fedratinib was first developed prior to its acquisition by Sanofi.

Dr. Hood serves as Chief Executive Officer of Impact Biomedicines. Prior to founding Impact, he was co-founder and Chief Scientific Officer of Samumed, a pharmaceutical platform company focused on advancing regenerative medicine and oncology applications. Prior to that, Dr. Hood was Director of Research and co-inventor of fedratinib at TargeGen, Inc., (subsequently acquired by Sanofi SA), where he led a team identifying small molecule kinase inhibitors for the treatment of eye diseases and cancer. He is an inventor on 100+ patents and author on 50+ scientific articles. Dr. Hood obtained a Ph.D. in medical physiology and B.S. in biochemistry from Texas A&M University.

Dr. Catriona Jamieson serves as Interim Chief Medical Officer of Impact Biomedicines, and co-founder. Concurrently, Dr. Jamieson is a Professor of Medicine and Chief of Regenerative Medicine, Deputy Director of the Sanford Stem Cell Clinical Center, Co-leader of the Hematologic Malignancies Program, and Director of Stem Cell Research at the Moores UC San Diego Cancer Center. She specializes in myeloproliferative neoplasms and leukemia, and was the principal investigator on several fedratinib trials. Dr. Jamieson obtained an M.D., a Ph.D. in microbiology and a B.S. in Genetics from the University of British Columbia prior to completing fellowships in hematology and bone marrow transplantation at Stanford University and being recruited to UC San Diego.

About Myeloproliferative Neoplasms
Myeloproliferative Neoplasms (MPN) are a closely-related group of blood cancers in which the body, specifically the bone marrow, makes too many red blood cells, platelets, or certain white blood cells. There are three types of blood cancers that comprise MPNs: Myelofibrosis, Polycythemia Vera and Essential Thrombocythemia. MPNs are characterized by the mutations of JAK2, MPL o

On October 13, 2017 Impact Biomedicines ("Impact") reported its launch to pioneer the development of life-changing treatments for patients with myeloproliferative neoplasms and other cancers (Press release, Impact Biomedicines, OCT 13, 2017, View Source [SID1234522384]). The Company’s pipeline is centered around fedratinib, a potent and highly selective oral small molecule JAK2 kinase inhibitor that is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV). In conjunction with this launch, Impact is pleased to share that the U.S. Food and Drug Administration (FDA) has removed the clinical hold placed on fedratinib and that the company has received $22.5 million from Medicxi through a Series A financing.

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Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Impact was formed in 2016 after acquisition of Sanofi’s full rights for the global development and commercialization of fedratinib. The majority equity holders in Impact include the co-founders and Medicxi, with Sanofi retaining a minority stake.

In 2013, the development of fedratinib was discontinued by Sanofi after the FDA issued a clinical hold subsequent to reports of a few potential cases of Wernicke’s encephalopathy (WE), an acute neurological condition usually indicative of a vitamin B deficiency, in patients participating in fedratinib clinical trials. Following a Type A meeting and review of additional data, the FDA concluded that Impact provided the necessary documentation to support lifting the clinical hold – opening the path for Impact’s continued development of fedratinib.

The clinical package for fedratinib includes data from a total of 18 studies completed in 877 subjects. In JAKARTA-1, a completed international Phase 3 pivotal trial for the treatment of myelofibrosis, fedratinib met its primary and secondary endpoints by reducing spleen size in 47% of patients by >35% at 24 weeks (p<0.0001) and improving symptom score in 36% of patients by >50% at 24 weeks (p< 0.0001). Comparable responses were seen in patients with normal or low platelet counts and thrombocytopenia was similar between placebo and the target dose of 400mg. In JAKARTA-2, a follow-on study in patients who were unresponsive to all other available therapies, including patients who were either Jakafi (ruxolitinib) resistant or intolerant, fedratinib showed similar activity. In that study, 55% of patients who had failed or were intolerant to ruxolitinib experienced a spleen size reduction of >35% with fedratinib. Notably, responses were noted in 63% of patients intolerant to ruxolitinib and 61% of patients who had lost ruxolitinib response. Currently, ruxolitinib is the only drug approved by the FDA to treat patients with MF and PV. The most common adverse events for fedratinib were hematological (anemia) and gastrointestinal (nausea, diarrhea and vomiting). The results of these trials have been published in leading peerreviewed journals.

"The decision to discontinue the development of fedratinib in 2013 was heartbreaking for the patients who were experiencing positive responses while in clinical trials. There are very limited therapeutic options for these patients and fedratinib was active in most patients when nothing else had worked. Because of the very high unmet medical need in MF, the Impact team completed a thorough review of the available data, including careful due diligence into the potential cases of WE and I am glad to report that as a result of this effort, the FDA has lifted the clinical hold," said John Hood, Ph.D., Chief Executive Officer of Impact. "In addition to MF, Impact intends to pursue multiple clinical indications for fedratinib to realize its potential as a best-in-class JAK2 kinase inhibitor."

To support the Company’s drug development and manufacturing efforts and the build out of the management team, Impact closed a $22 million Series A financing with Medicxi.

"We’ve made it a point to double down and invest more in maturing life science companies who are delivering innovation where it’s needed most. In line with our ‘asset-centric’ approach, we believe Impact is doing just that by reviving fedratinib with a thoughtful development approach and due diligence," said Kevin Johnson, Impact board member and Co-Founder of Medixci. "We are confident that the dedicated team at Impact has the vision and skill to create a sustainable long-term business around fedratinib."

The development of fedratinib is being led by a highly skilled and devoted team, including members of the original TargeGen team, where fedratinib was first developed prior to its acquisition by Sanofi.

Dr. Hood serves as Chief Executive Officer of Impact Biomedicines. Prior to founding Impact, he was co-founder and Chief Scientific Officer of Samumed, a pharmaceutical platform company focused on advancing regenerative medicine and oncology applications. Prior to that, Dr. Hood was Director of Research and co-inventor of fedratinib at TargeGen, Inc., (subsequently acquired by Sanofi SA), where he led a team identifying small molecule kinase inhibitors for the treatment of eye diseases and cancer. He is an inventor on 100+ patents and author on 50+ scientific articles. Dr. Hood obtained a Ph.D. in medical physiology and B.S. in biochemistry from Texas A&M University.

Dr. Catriona Jamieson serves as Interim Chief Medical Officer of Impact Biomedicines, and co-founder. Concurrently, Dr. Jamieson is a Professor of Medicine and Chief of Regenerative Medicine, Deputy Director of the Sanford Stem Cell Clinical Center, Co-leader of the Hematologic Malignancies Program, and Director of Stem Cell Research at the Moores UC San Diego Cancer Center. She specializes in myeloproliferative neoplasms and leukemia, and was the principal investigator on several fedratinib trials. Dr. Jamieson obtained an M.D., a Ph.D. in microbiology and a B.S. in Genetics from the University of British Columbia prior to completing fellowships in hematology and bone marrow transplantation at Stanford University and being recruited to UC San Diego.