On January 4, 2016 Kura Oncology, Inc. (NASDAQ:KURA), a clinical stage biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) accepted the company’s Investigational New Drug (IND) application to begin Phase 1 clinical testing of KO-947, its small molecule inhibitor of extracellular-signal-regulated kinases 1 and 2 (ERK1/2) as a treatment for cancers in which the mitogen activated protein kinase (MAPK) pathway is dysregulated (Press release, Kura Oncology, JAN 4, 2017, View Source [SID1234517257]). Additionally, Kura announced nomination of KO-539, an orally-available small molecule inhibitor of the menin-MLL interaction, as a development candidate for the treatment of mixed lineage leukemias, a genetically-defined subset of the two most common forms of acute leukemia, acute myeloid leukemia and acute lymphoblastic leukemia. Schedule your 30 min Free 1stOncology Demo! "We are delighted to reach these important milestones in the development of our pipeline programs. KO-947, our ERK inhibitor, has shown compelling and differentiated anti-tumor activity in preclinical models of cancers in which the MAPK pathway is dysregulated, and we look forward to advancing it into human clinical testing," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We are also pleased to nominate KO-539, an inhibitor of the menin-MLL interaction, as a development candidate, which we are advancing as a potential treatment for patients with particularly aggressive forms of leukemia. Along with the progress we have made on tipifarnib, our lead program, these achievements are a testament to our team’s ability to execute on our stated goal of simultaneously advancing multiple programs aimed at addressing the urgent needs of cancer patients facing a poor prognosis and limited treatment options."
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Kura expects to initiate a Phase 1 clinical trial of KO-947 in patients with a variety of solid tumors with dysregulation of the MAPK pathway in the first half of 2017. The company’s goal for its menin-MLL inhibitor program is to initiate a Phase 1 clinical trial in 2018.
Author: [email protected]
Regorafenib from Bayer Granted Priority Review in the U.S. for Second-Line Treatment of Liver Cancer (for specialized target groups only)
On January 4, 2017 Bayer reported that the U.S. Food and Drug Administration (FDA) has granted priority review designation to the supplemental New Drug Application (sNDA) for regorafenib for the second-line treatment of patients with unresectable hepatocellular carcinoma (uHCC) in the U.S (Press release, Bayer, JAN 4, 2017, View Source [SID1234517256]). Schedule your 30 min Free 1stOncology Demo! "Liver cancer is one of the leading cancer-related causes of death world-wide and more than 30,000 cases of liver cancer are diagnosed in the U.S each year. As the first and only approved systemic treatment for HCC, Nexavar was a significant step in addressing the unmet need in this field, but effective second-line treatment options are urgently needed for patients", said Dr. Joerg Moeller, member of the Executive Committee of Bayer AG’s Pharmaceutical Division and Head of Development. "The priority review for regorafenib in the U.S. is great news for patients as it supports our efforts to make this treatment available as early as possible in the second-line setting for HCC in the U.S."
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The FDA grants priority review to medicines that if approved, would be significant improvements in the safety or effectiveness of the treatment for serious conditions. Under the Prescription Drug User Fee Act (PDUFA), the FDA aims to complete its review within six months (compared to 10 months under standard review).
Regorafenib is already approved under the brand name Stivarga in many countries, including the U.S., to treat metastatic colorectal cancer and unresectable and/or metastatic gastrointestinal stromal tumors.
The regulatory submission for regorafenib is based on data from the international, multicenter, placebo-controlled Phase III RESORCE [REgorafenib after SORafenib in patients with hepatoCEllular carcinoma] trial. The trial investigated regorafenib in patients with unresectable hepatocellular carcinoma (HCC) whose disease had progressed during treatment with sorafenib (Nexavar) tablets. Results showed that regorafenib significantly improved overall survival (OS) compared to placebo (HR 0.63; 95% CI 0.50-0.79; p<0.001), which over the trial period represents a 37 percent reduction in the risk of death for patients who received regorafenib plus best supportive care (BSC) compared to patients treated with placebo plus BSC. The median OS was 10.6 months in patients treated with regorafenib, compared to 7.8 months in patients who received placebo plus BSC. The safety and tolerability was generally consistent with the known profile of regorafenib, with no clinically meaningful differences in health-related quality of life (HRQoL) between the regorafenib and placebo plus BSC groups. Data from the study were first presented at the 18th World Congress on Gastrointestinal Cancer (WCGC) in June 2016 and published online on December 5, 2016 in the peer-reviewed journal The Lancet.
Regorafenib has also been submitted to regulatory authorities in Japan and the EU for the treatment of second-line HCC and submissions in additional countries are in progress.
About the RESORCE trial
The Phase III RESORCE [REgorafenib after SORafenib in patients with hepatoCEllular carcinoma] clinical trial enrolled 573 patients whose disease had progressed during treatment with sorafenib. Patients were randomized in a 2:1 ratio to receive either regorafenib or placebo plus best supportive care.
Patients received 160 mg regorafenib once daily or placebo, for 3 weeks on/1week off, with 28 days constituting one full treatment cycle. The primary endpoint of the study was overall survival, and secondary endpoints were time to progression, progression-free survival, objective tumor response rate and disease control rate. Health-related quality of life was assessed by the FACT-Hep and EQ-5D questionnaires. Safety and tolerability were also continuously monitored.
About Hepatocellular Carcinoma
Hepatocellular carcinoma (HCC) is the most common form of liver cancer and represents approximately 70-85 percent of liver cancer worldwide. Liver cancer is the sixth most common cancer in the world and the second leading cause of cancer-related deaths globally. More than 780,000 cases of liver cancer are diagnosed worldwide each year (52,000 in the European Union, 501,000 in the Western Pacific region and 30,000 in the United States) and the incidence rate is increasing. In 2012, approximately 746,000 people died of liver cancer including approximately 48,000 in the European Union, 477,000 in the Western Pacific region and 24,000 in the United States.
About Regorafenib (Stivarga)
Regorafenib is an oral multi-kinase inhibitor that potently blocks multiple protein kinases involved in tumor angiogenesis (VEGFR1, -2, -3, TIE2), oncogenesis (KIT, RET, RAF-1, BRAF), metastasis (VEGFR3, PDGFR, FGFR) and tumor immunity (CSF1R).
Regorafenib is approved under the brand name Stivarga in more than 90 countries worldwide, including the U.S., countries of the EU and Japan for the treatment of metastatic colorectal cancer (mCRC). The product is also approved in over 80 countries, including the U.S., countries of the EU and Japan, for the treatment of metastatic gastrointestinal stromal tumors (GIST). In the EU, Stivarga is indicated for the treatment of adult patients with mCRC who have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidine-based chemotherapy, an anti-VEGF therapy and an anti-EGFR therapy, as well as for the treatment of adult patients with unresectable or metastatic GIST who progressed on or are intolerant to prior treatment with imatinib and sunitinib.
Regorafenib is a compound developed by Bayer. In 2011, Bayer entered into an agreement with Onyx, now an Amgen subsidiary, under which Onyx receives a royalty on all global net sales of regorafenib in oncology.
Flamel Technologies Completes Cross-Border Merger and Becomes Avadel Pharmaceuticals plc
On January 3, 2016 Flamel Technologies SA, reported that it has completed its previously announced cross-border merger with and into its wholly-owned Irish subsidiary, Avadel Pharmaceuticals plc (NASDAQ: AVDL) (Avadel), effective January 1, 2017, with Avadel surviving the merger as the public holding company (Filing, 8-K, Flamel Technologies, JAN 3, 2017, View Source [SID1234517292]). As a result of the merger, all of Flamel’s outstanding ordinary and American Depository Shares (ADSs) were canceled and exchanged on a one-for-one basis for Avadel ordinary shares and ADSs, respectively. Avadel ADSs will begin trading on the NASDAQ Global Market under trading symbol "AVDL" on January 3, 2017.
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Michael Anderson, Avadel’s Chief Executive Officer, remarked, "We are excited to enter 2017 as Avadel. The completion of the cross-border merger from France to Ireland serves as a way to unify our subsidiaries under a shared corporate identity, and provides the company with a new set of corporate governance policies that will allow us greater flexibility as we continue to grow our business and commercialize products."
Mr. Anderson continued, "Our new name, Avadel, which stands for ‘advanced delivery,’ was born out of our company’s strong history in drug delivery and serves as a constant reminder of a key piece of our company’s growth strategy – to develop differentiated pharmaceutical products utilizing our proprietary and innovative technologies."
"We are excited to begin 2017 with a new name, an ongoing Phase III trial and a strong financial position," finished Mr. Anderson.
Marina Biotech Granted European Claims Covering Bacteria Mediated Gene Silencing
On January 3, 2017 Marina Biotech, Inc. (OTCQB: MRNA), a biopharmaceutical company focused on the development and commercialization of innovative therapeutics for disease intersections of arthritis, hypertension, and cancer, reported that the European Patent Office intends to grant the company a patent for Bacteria Mediated Gene Silencing (EP 08768475.9, European Patent 2173875) (Filing, 8-K, Marina Biotech, JAN 3, 2017, View Source [SID1234517265]).
The granted claims relate to the company’s tkRNAi technology being utilized in its CEQ508 program that is being developed to treat familial adenomatous polyposis (FAP). Marina’s patent portfolio around tkRNAi and its CEQ508 program includes 14 issued patents worldwide. The claims are broad, and cover a prokaryotic vector comprising a promoter for generating siRNAs. Targets for the siRNA include beta-catenin. The vector can include an invasion factor, and a lysis regulator. An invasive bacterium including the vector is also encompassed. Marina Biotech will continue to expand its development of tkRNAi to cover other therapeutic targets as permitted by the granted patent- including Ras, APC, HER-2, MDR-I, MDR-2, FATP4, SGLUT-1, GLUT-2, GLUT-5, apobec-1, MTP, IL-6, IL-6R, IL-7, IL-12, IL-13, Ra-I, IL-18, p38/JNK MAP kinase, p65/NK-kB, CCL20, Claudin-2, Chitinase 3-like 1, apoA-IV, MHC class I and MHC class II.
"We are excited by the recent positive developments from the CEQ-508 program and looking forward to expand bacteria mediated gene silencing to other therapeutic areas protected by our patent portfolio, including oncology, diabetes, and hyperlipidemia", stated Dr. Vuong Trieu, Chairman of the Board at Marina Biotech. "This unique ability to orally deliver oligo therapeutics across different species and kingdoms will open up applications not possible with any other delivery platforms".
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Flamel Technologies Completes Cross-Border Merger and Becomes Avadel Pharmaceuticals plc
On August 12, 2016 Flamel Technologies (NASDAQ: FLML) reported that it received shareholder approval to reincorporate its country of domicile to Ireland from France via a cross-border merger (Press release, Avadel Pharmaceuticals, AUG 12, 2016, View Source;item=o8hHt16027g9XhJTr8+weNRYaV9bFc2rMd0Q/AXw4zvZ/USJmYQCtLIQG2aEdNsmFxHqxmm39PDt+LydB4tmozzAmW9nBdXvb3mR+p/DbTIWkJzh8LD8LGxkQnP3b9aywxrvUjWJ9oubudnn9pG8ZA==&cb=636204461836502476 [SID1234521917]). Shareholders voted in favor of the reincorporation by proxy at the Company’s Extraordinary General Meeting, held at the Company’s headquarters in Lyon, France on August 10, 2016.
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Under the terms of the Company’s reincorporation, Flamel will merge with and into its wholly owned subsidiary, Avadel Pharmaceuticals Limited. In connection with the merger, Avadel Pharmaceuticals Limited will re-register as an Irish public limited company and will be known as "Avadel Pharmaceuticals plc," which will be the name under which the Company will conduct its business from and after the merger, effective on January 1, 2017.
Mike Anderson, Chief Executive Officer of Flamel, remarked, "Flamel’s vision has transformed over the last few years from a pure play drug delivery company, to that of a diversified specialty pharmaceutical company capable of independently developing and marketing its own proprietary products. Ireland is an ideal location to execute this vision as it is quickly becoming a global pharma hub, and offers corporate governance policies more akin to those in the U.S. Our board and senior management both feel strongly that this move will allow the Company to operate in a manner that will return maximum value to shareholders once we begin to launch products utilizing our proprietary technologies, such as Micropump sodium oxybate, for which we expect to initiate a Phase III pivotal trial imminently."
More information regarding the Company’s reincorporation to Ireland can be found in its proxy to shareholders, filed with the Securities and Exchange Commission on July 5, 2016.
About Flamel Technologies:
Flamel Technologies SA (NASDAQ: FLML) is a specialty pharmaceutical company utilizing its core competencies in formulation development and drug delivery to develop safer and more efficacious pharmaceutical products, addressing unmet medical needs and/or reducing overall healthcare costs. Flamel currently markets three previously Unapproved Marketed Drugs ("UMDs") in the United States, Bloxiverz (neostigmine methylsulfate injection), Vazculep (phenylephrine hydrochloride injection), and Akovaz (ephedrine sulfate injection). The Company also develops products utilizing its proprietary drug delivery platforms, Micropump (oral sustained release microparticles platform), along with its tangent technologies, LiquiTime (a Micropump-derivative platform for liquid oral products) and Trigger Lock (a Micropump-derivative platform for abuse-resistant opioids). Additionally, the Company has developed a long acting injectable platform, Medusa, a hydrogel depot technology, particularly suited to the development of subcutaneously administered formulations. Current applications of Flamel’s drug delivery products include sodium oxybate (Micropump), extended-release of liquid medicines such as ibuprofen and guaifenesin (LiquiTime, through a license arrangement with Elan Pharma International Limited for the U.S. Over-the-Counter market) and a current study of the delivery of exenatide utilizing the Medusa technology. In February 2016, Flamel acquired FSC Pediatrics, a company that markets three pediatric pharmaceutical products – Cefaclor for oral suspension, indicated for infection, Karbinal ER, indicated for allergic rhinitis and AcipHex Sprinkle (rabeprazole sodium) indicated for the treatment of gastroesophageal disease (GERD). FSC also received 510(k) clearance from the FDA in October 2014 for Flexichamber, a collapsible holding chamber for used in the administration of aerosolized medication using pressurized Metered Dose Inhalers (pMDIs) for the treatment of asthma. The Company is headquartered in Lyon, France and has operations in Dublin, Ireland and St. Louis, Missouri.