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Clovis Oncology Receives Notification of PDUFA Extension for Rociletinib

On December 15, 2015 Clovis Oncology, Inc. (NASDAQ: CLVS) reported that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for Clovis’ New Drug Application (NDA) for rociletinib by the standard extension period of three months with the new goal date of June 28, 2016 (Press release, Clovis Oncology, DEC 15, 2015, View Source [SID:1234508577]).

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Rociletinib is an investigational therapy for the treatment of patients with mutant epidermal growth factor receptor (EGFR) non-small cell lung cancer (NSCLC) who have been previously treated with an EGFR-targeted therapy and have the EGFR T790M mutation.

Clovis submitted a Major Amendment on November 16, 2015 in response to the FDA’s request for additional clinical data for both the 500mg and 625mg BID dose patient groups for rociletinib. As expected, the FDA extended the PDUFA goal date to allow additional time for review of the new information requested by the Agency.

About Rociletinib

Rociletinib is the company’s novel, oral, targeted covalent (irreversible) mutant-selective inhibitor of EGFR in development for the treatment of NSCLC in patients with initial activating EGFR mutations, as well as the dominant resistance mutation T790M. Data from both the pivotal, single-arm TIGER-X and TIGER-2 clinical trials served as the basis for the U.S. and EU regulatory submissions for the treatment of advanced mutant EGFR T790M-positive lung cancer. Rociletinib was granted Breakthrough Therapy designation by the FDA in May 2014.

NewLink Genetics Corporation Completes Enrollment of Phase 3 PILLAR Trial Evaluating Algenpantucel-L for Patients With Locally Advanced Pancreatic Cancer

On December 15, 2015 NewLink Genetics Corporation (NASDAQ:NLNK), a biopharmaceutical company at the forefront of discovering, developing and commercializing novel immuno-oncology product candidates, including both cellular immunotherapy and checkpoint inhibitor platforms, to improve the lives of patients with cancer, reported the completion of enrollment in the Phase 3 PILLAR (Pancreatic Immunotherapy with algenpantucel-L for Locally Advanced non-Resectable cancer) clinical trial of algenpantucel-L for patients with borderline resectable or locally advanced unresectable pancreatic cancer (Press release, NewLink Genetics, DEC 15, 2015, View Source [SID:1234508574]). Total enrollment in the study exceeded 300 patients.

"I am delighted to be part of this important study, with the potential for helping patients with pancreatic cancer in clear need of pioneering immunotherapies like algenpantucel-L," said Harish Lavu, M.D., Associate Professor of Surgery at Thomas Jefferson University and a lead investigator for PILLAR.

"Completion of enrollment in this large phase 3 trial combined with our previous success enrolling 722 patients with resected pancreatic cancer in the IMPRESS registration trial demonstrates our ongoing commitment to patients with this disease," said Charles Link, Jr., M.D., Chairman and CEO of NewLink Genetics. "With the addition of our trial of indoximod, one of our IDO pathway inhibitors, in combination with chemotherapy for patients with metastatic pancreatic cancer, we now have clinical trials in all stages of this disease for which patients have very limited treatment options."

About the PILLAR Trial

PILLAR is a Phase 3, 1:1 randomized trial assessing overall survival for patients with borderline resectable or locally advanced unresectable pancreatic cancer treated with a regimen of FOLFIRINOX or gemcitabine/nab-paclitaxel in combination with algenpantucel-L cellular immunotherapy versus standard of care chemotherapy alone.

About HyperAcute Cellular Immunotherapy

NewLink Genetics’ HyperAcute Cellular Immunotherapy platform creates novel biologic products that are designed to stimulate the human immune system to recognize and attack cancer cells. HyperAcute Cellular Immunotherapy product candidates are composed of human cancer cells that are tumor specific, but not patient specific. These cells have been modified to express alpha-gal, a carbohydrate for which humans have pre-existing immunity. These alpha-gal-modified cells stimulate a rapid and powerful human immune response that educates the body’s natural defenses to seek out and destroy cancer cells. The objective of HyperAcute immunotherapies is to elicit an antitumor response by "educating" the immune system to attack a patient’s own cancer cells. HyperAcute immunotherapies do not require any tissue from individual patients and use intact whole cells rather than cell fragments or purified proteins. We believe these unique properties of HyperAcute Cellular Immunotherapy products result in the stimulation of a robust immune response.

NewLink’s lead product candidate, algenpantucel-L, is also being studied in a Phase 3 trial (IMPRESS: "Immunotherapy for Pancreatic Resectable cancer Survival Study") under a Special Protocol Assessment with the U.S. Food and Drug Administration. This trial involves 722 patients with surgically resected pancreatic cancer.

NewLink has several HyperAcute Cellular Immunotherapy product candidates focused on patients with multiple tumor types including lung cancer, melanoma and renal cell cancer.

ArQule Announces Orphan Drug Designation in Cholangiocarcinoma and Clinical Update for ARQ 087

On December 15, 2015 ArQule, Inc. (NASDAQ:ARQL) reported receipt of orphan drug designation from the Food and Drug Administration (FDA) for ARQ 087 in cholangiocarcinoma (Press release, ArQule, DEC 15, 2015, View Source [SID:1234508572]). ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth receptor (FGFR) family.

ArQule is currently conducting a biomarker-driven phase 2 trial for ARQ 087 in the U.S. and Italy in intrahepatic cholangiocarcinoma (iCCA) patients with FGFR2 fusions. The commencement of the phase 2 trial followed the observation of two confirmed partial responses in iCCA with FGFR2 fusions in the phase 1a portion of the trial. Subsequently, a minor response was observed in a third patient also harboring an FGFR2 fusion.

The phase 2 trial is designed to enroll up to 20 patients and potentially provide a clinical signal to be further explored in an expedited clinical strategy in this rare indication. The incidence rate for iCCA in the U.S. and Europe is approximately one in 100,000, respectively, while the incidence rate in Asia is believed to be higher.

In addition to the phase 2 trial for iCCA, ArQule is continuing to enroll the phase 1b portion of the trial with ARQ 087 in solid tumors harboring FGFR2 genetic alterations. A partial response was recently observed in a bladder cancer patient with an FGFR2 genetic alteration.

"This year we have already received orphan drug designation for ARQ 092 in Proteus syndrome. We are now pleased to have received our second orphan drug designation from the FDA for ARQ 087 in CCA. This is part of our continued pursuit of precision medicine," said Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "CCA is a rare cancer that lacks approved therapeutic options to specifically target FGFR2 fusions. We are very encouraged by the clinical efficacy so far observed in the phase 1 portion of the trial which validates the pre-clinical data recently presented at the AACR (Free AACR Whitepaper)-NCI-EORTC conference that demonstrated efficacy across a number of FGFR2-driven tumors in xenograft models."

About Intrahepatic Cholangiocarcinoma

Cholangiocarcinoma is the most common biliary malignancy and the second most common hepatic malignancy after hepatocellular carcinoma (HCC)1. Depending on the anatomic location, CCA is classified as intrahepatic (iCCA), perihilar (pCCA), and extrahepatic (eCCA.) iCCA originates from the intrahepatic biliary ductal system and forms an intrahepatic mass. The average age adjusted incidence rate for iCCA is approximately one in 100,000 per year in the United States and Europe2,3.

About FGFR and ARQ 087

ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor ("FGFR") family with demonstrated efficacy in FGFR2 translocations. The FGFR pathway is disrupted in several ways in human cancer, thus providing numerous therapeutic targets for an inhibitor of this pathway. ARQ 087 has demonstrated inhibition of tumor growth and downstream signaling in vivo in tumors whose growth is driven by these targets.

Signals of single agent activity with this compound were observed in phase 1a testing. Phase 1b expansion cohorts with ARQ 087 include patients with cholangiocarcinoma and adrenocortical tumors, as well as those with FGFR translocations, amplifications and mutations. Clinical development of ARQ 087 has advanced into phase 2 for intrahepatic cholangiocarcinoma (iCCA) following the observation of two confirmed responses in this patient population in the phase 1 portion of the program.

Bird Rock Bio Announces Corporate Name Change from RuiYi, Inc

On December 14, 2015 Bird Rock Bio, Inc., a clinical stage biopharmaceutical company, reported its corporate name change from RuiYi, Inc. to Bird Rock Bio (Press release, Bird Rock Bio, DEC 14, 2015, View Source [SID1234515802]).

"With the advancement of gerilimzumab into Phase 2 for rheumatoid arthritis (RA) and RYI-018 into Phase 1 for non-alcoholic fibrotic liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH) in 2016, our organization has shifted dramatically from discovery operations in Shanghai to global clinical development for novel antibody therapeutics targeting large unmet medical needs," said Paul Grayson, CEO. "We anticipate an exciting year of milestones for both of our highly differentiated antibodies and the new name reflects the shift and progress."

Backed by leading biotechnology venture investors, Bird Rock Bio’s strategy leverages biologic targets with substantial human proof of mechanism for the development of best in class molecules with strong clinical and commercial differentiation. RYI-018 is a first in class inverse agonist antibody to the CB1 receptor with anticipated clinical differentiation associated with an enhanced safety profile and gerilimzumab is a novel anti-IL-6 antibody with potential commercial differentiation as a disruptive, cost effective treatment.

Previous small molecule inverse agonists to the CB1 receptor demonstrated significant benefits in inflammation, metabolism and fibrosis (the principle contributors to NAFLD and NASH), however, these small molecule drugs had potential risk of severe CNS side effects. RYI-018 is the only known CB1 inverse agonist antibody and has demonstrated, pre-clinically, distribution to all peripheral tissues without penetration of the brain. As such, RYI-018 possesses differentiated and potentially compelling product potential for large unserved markets such as NASH. The Company is advancing RYI-018 through IND enablement and anticipates Phase 1 to initiate 2H 2016.

Gerilimzumab is a novel monoclonal antibody that is directed against the IL-6 cytokine, for the treatment of autoimmune disorders, including RA. Gerilimzumab has an ideal product profile, demonstrating in preclinical studies to have the highest known potency amongst the anti-IL-6 cytokine class and in a Phase 1 clinical trial in healthy volunteers to have an extended blood half-life, which is anticipated to support small volume dosing Bird Rock Bio once every eight weeks. This would enable high patient convenience and lowest pricing, which are compelling product features for a chronic therapy to increasingly cost-sensitive healthcare systems globally.

Bird Rock Bio believes gerilimzumab has the potential to effectively compete in the $35 billion global biologic market for rheumatoid arthritis by not only treating the large number of anti-TNF refractory patients but also becoming a front line treatment for moderate and severe patients. The Company expects to submit for its Phase 2 clinical trial in Brazil, a country with more than 1 million biologic naïve RA patients in 1H 2016

Molecular Basis For Development of Cyclacel’s CYC065 CDK2/9 Inhibitor In Triple-Negative Breast Cancer Presented At San Antonio Breast Cancer Symposium

On December 14, 2015 Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC) (Nasdaq:CYCCP) ("Cyclacel" or the "Company"), reported the presentation of preclinical data demonstrating the molecular basis for the development of CYC065 in triple negative breast cancer (TNBC), and in particular basal-like TNBC. CYC065 is a highly-selective, second-generation cyclin dependent kinase (CDK) inhibitor targeting CDK2/9 dependent tumors (Press release, Cyclacel, DEC 14, 2015, View Source [SID:1234508697]). The data were presented at the San Antonio Breast Cancer Symposium (SABCS), taking place December 8-12, 2015.

"We have recently reported that CYC065 can target malignancies driven by CDK2/9 dependent oncogenic and leukemogenic pathways such as MLL-rearranged leukemias and tumors overexpressing MYC", said Spiro Rombotis, Cyclacel’s President and Chief Executive Officer. "The data presented at SABCS show CYC065 could be active in breast cancer patients with a poor prognosis and that its mechanism induces breast cancer cell death by apoptosis. In addition, we have identified approved and investigational anticancer agents, including our own sapacitabine, which combine effectively with CYC065. A first-in-human, Phase 1 clinical trial with CYC065 has commenced and we look forward to reporting data from this study."

Data presented at SABCS (Program Number: P5-03-10, View Source) demonstrated in particular the mechanistic rationale for clinical development of CYC065 in basal-like TNBC, a cancer with poor prognosis frequently associated with BRCA1 mutations. Molecular characteristics of TNBC include amplification or overexpression of Cyclin E, the partner protein of CDK2, and MYC. CYC065 directs a pro-apoptotic mechanism in breast cancer cell lines, which includes transcriptional down regulation of key pro-survival and oncogenic regulators, including MCL-1 and MYC.

CYC065 was shown to rapidly induce cell death in breast cancer cell lines, while transiently inducing G1 cell cycle arrest in non-malignant breast lines. CYC065’s potent anticancer activity has been confirmed in breast cancer xenograft animal models. CYC065 effectively combined with Cyclacel’s sapacitabine in breast cancer cell lines, as was the case with seliciclib, Cyclacel’s first generation CDK2/9 inhibitor, when combined with sapacitabine.

An oral regimen of seliciclib and sapacitabine is being evaluated in an on-going Phase 1 all-comer study of patients with various advanced cancers. In previously reported initial data from this study, durable partial responses have been observed in breast, ovarian and pancreatic cancer patients with germline mutations in Homologous Recombination (HR) repair genes, specifically BRCA1 or BRCA2.

CYC065 is in a first-in-human, Phase 1 clinical trial.