On April 3, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the phase III ALUR study met its primary endpoint, demonstrating that Alecensa (alectinib) significantly improved progression-free survival (PFS) in people with anaplastic lymphoma kinase (ALK)-positive advanced (metastatic) non-small cell lung cancer (NSCLC) who had progressed following treatment with one prior line of both platinum-based chemotherapy and crizotinib, compared with chemotherapy (Press release, Hoffmann-La Roche, APR 3, 2017, View Source [SID1234518398]).1 The peer-reviewed data will be published later this year.

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"We are pleased to announce that the results of the phase III ALUR trial further support the use of Alecensa as a treatment for people with ALK-positive lung cancer who, after having progressed on both chemotherapy and crizotinib, are in need of new treatment options", said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development. "The results of this trial will support our access discussions with global health authorities as we seek to bring Alecensa to patients faster."
Alecensa received conditional marketing authorisation from the European Commission in people with ALK-positive NSCLC previously treated with crizotinib on 16 February 2017,2 based on the results of the phase I/II NP286733 and NP287614 studies. Alecensa is also approved as a monotherapy (alone) for people with ALK-positive NSCLC who have progressed on or are intolerant to crizotinib in the US and nine other countries globally.5 In addition, Alecensa is approved in Japan for people whose tumours were advanced, recurrent or could not be removed completely through surgery (unresectable).6
Alecensa is also being explored as a first-line treatment option with the phase III ALEX study comparing Alecensa to crizotinib in ALK-positive NSCLC.7 The ALEX study is expected to report data in the first half of 2017.
Approximately 75,000 people globally are diagnosed with ALK-positive NSCLC every year.8,9,10 It is a distinct form of lung cancer commonly diagnosed in younger people (median age 52),11 and in women (approximately 54% of cases).12 ALK-positive NSCLC is also generally found in those with a light or non-smoking history. 13
About the ALUR study1

ALUR (NCT02604342) is a randomised, multi-centre, open-label phase III study evaluating the efficacy and safety of Alecensa versus chemotherapy (pemetrexed or docetaxel) in people with ALK-positive NSCLC previously treated with one prior line of both platinum-based chemotherapy and crizotinib. People were randomised (2:1) to receive either Alecensa or chemotherapy. The primary endpoint of the ALUR study is PFS and secondary endpoints include: overall survival (OS), central nervous system (CNS) objective response rate (ORR) in people with measurable brain metastases at baseline and median time to CNS progression. The multicentre study was conducted in 119 people across 15 countries.
About the NP28673 and NP28761 studies

NP28673 is a phase I/II global, single arm, open-label, multicentre trial evaluating the safety and efficacy of Alecensa in 138 people with ALK-positive NSCLC whose disease progressed on crizotinib.3 NP28761 is a phase I/II North American, single arm, open-label, multicentre trial evaluating the safety and efficacy of Alecensa in 87 people with ALK-positive NSCLC whose disease progressed on crizotinib.4 A pooled analysis from the NP28673 and NP28761 studies showed that Alecensa shrank tumours in 52.2% (95% CI: 39.7%, 64.6%) of people with advanced ALK-positive NSCLC whose disease had progressed following treatment with crizotinib (overall response rate; ORR).3,4 The NP28673 study also showed that Alecensa extended the time that people lived without their disease worsening or death (PFS) by a median of 8.9 months3 (95% CI: 5.6, 12.8), while the NP28761 study demonstrated a median PFS benefit of 8.2 months14 (95% CI: 6.3, 12.6) with Alecensa.
In addition, a pooled analysis of the two studies showed that Alecensa shrank CNS tumours that were measurable in 64% of people (95% CI: 49.2%, 77.1%), and 22% (n=-29) achieved a complete response of their measurable and non-measurable CNS tumours.15
About Alecensa

Alecensa (RG7853/AF-802/RO5424802/CH5424802) is an oral medicine created at Chugai Kamakura Research Laboratories and is being developed for people with NSCLC whose tumours are identified as ALK-positive. ALK-positive NSCLC is often found in younger people who have a light or non-smoking history.13 It is almost always found in people with a specific type of NSCLC called adenocarcinoma.13 Alecensa is currently approved in the United States, Europe, Kuwait, Israel, Hong Kong, Canada, South Korea, Switzerland, India, Australia and Taiwan for the treatment of advanced (metastatic) ALK-positive NSCLC whose disease has worsened after, or who could not tolerate treatment with, crizotinib and in Japan for people whose tumours were advanced, recurrent or could not be removed completely through surgery (unresectable).5,6
In a pooled analysis of CNS endpoints from studies NP28673 and NP28761, Alecensa demonstrated activity in brain metastases, indicating that the drug may be taken up in the brain.14 The brain is protected by the blood-brain barrier, a network of tightly joined cells that line the inside of the blood vessels in the brain and spinal cord.16 One of the ways the blood-brain barrier prevents molecules from affecting the brain is to actively eject them from the barrier through a process known as ‘active efflux’.17 The active efflux system does not recognise Alecensa, which means that it may travel into and throughout brain tissue.3,4
The global phase III ALEX study of Alecensa includes a companion test developed by Roche Diagnostics. Alecensa is marketed in Japan by Chugai Pharmaceutical, a member of the Roche Group.
About Roche in lung cancer

Lung cancer is a major area of focus and investment for Roche, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have four approved medicines to treat certain kinds of lung cancer and more than ten medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.

On April 2, 2017 Precision-Biologics Inc. reported an abstract reporting data on the company’s neo-antigen program has been accepted for an oral presentation at the upcoming 110th Annual Meeting of the American Association of Clinical Research (AACR) (Free AACR Whitepaper) in Washington D.C., April 1-5, 2017 (Press release, Precision Biologics, APR 2, 2017, View Source [SID1234518432]).

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"Precision Biologics continues to make progress with our preclinical and clinical programs," said Philip M. Arlen, President and CEO of Precision Biologics. "As this study demonstrates, our novel, humanized IgG1 monoclonal antibody, NEO-201, specifically targets a neoantigen expressed on a broad range of solid tumors. Tumor cell killing, antibody-dependent, cell-mediated cytotoxicity (ADCC), was significantly enhanced when NEO-201 was combined with an allogenic source of natural killer cells provided by NantKwest (Nasdaq:NK)."

Patrick Soon-Shiong, MD, Chairman and CEO of NantCell added, "These results highlight the potential to synergistically improve the response of antibody therapy when used in combination with NantKwest’s off-the-shelf natural killer cells." Dr. Soon-Shiong continued," Neoantigen targeting is an attractive, emerging therapeutic approach to next generation cancer care and NEO-201, with specificity to tumor tissues and not normal tissues, offers the potential to further improve response rates and look to transitioning this NEO-201/NK cell combination into human clinical trials targeting ovarian, GI and other cancer types as quickly as possible."

Presentation Summary
Preclinical characterization of a novel monoclonal antibody targeting a neo-antigen expressed in ovarian and GI malignancies

Abstract #3025,View Source!/4292/presentation/5981
Presenter: Kristen Zeligs, MD Walter Reed National Military Medical Center, Bethesda, MD
Monday, April 3, 2017, 4:05-4:20PM, Ballroom A-B

This oral presentation will present preclinical data demonstrating the potential benefit of targeting the NEO-201 neoantigen in both cancer cell lines as well as patient’s tumors. NEO-201 monoclonal antibody was shown to mediate ADCC activity, which was further enhanced when used with allogenic natural killer cells in tumor cell lines expressing the NEO-201 neoantigen. The NEO-201 antigen is expressed on numerous solid tumors with minimal expression on healthy tissue. The company plans to move this program into human clinical trials in the near future.
In October 2015, NantCell Inc., a subsidiary of NantWorks, LLC, acquired a majority stake in Precision Biologics with a $50 million investment. NantCell made the investment to further its efforts to activate the human immune system to treat the biology of cancer and avoid the toxic effects of standard chemotherapy. With Precision Biologics proprietary library of cancer vaccines, NantCell is working on developing compounds that could change the way cancer is detected and treated.

On April 2, 2017 BeiGene, Ltd. (NASDAQ:BGNE), a clinical-stage biopharmaceutical company developing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported data from the Phase IB dose expansion study of RAF dimer inhibitor BGB-283 in patients with B-RAF or K-RAS/N-RAS mutated solid tumors in an oral presentation during a Clinical Trials Plenary Session at the 2017 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Washington, DC (Press release, BeiGene, APR 2, 2017, View Source [SID1234518415]). BGB-283 is a novel inhibitor of RAF, in both its monomeric and dimeric forms, and has demonstrated activity in both B-RAF and K-RAS-mutated tumors in preclinical studies and in the Phase IA dose escalation portion of this Phase I study. In the Phase IB portion, BGB-283 has generally been well-tolerated at a dose of 30 mg once a day (QD) and has continued to show antitumor activity not only in subjects with B-RAF V600-mutated solid tumors, but also in subjects with K-RAS-mutated solid tumors.

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"BGB-283 appears to have a desirable risk-benefit profile in the combined efficacy and safety data from the Phase IA and IB parts of this study. Clinical activity has been observed for BGB-283 not only in B-RAF V600-mutated melanoma, thyroid cancer, and ovarian cancer, but also in K-RAS-mutated non-small cell lung cancer and endometrial carcinoma. These observations warrant further investigation of BGB-283 in the clinic," commented Jayesh Desai, MD, FRACP, a medical oncologist at The Royal Melbourne Hospital and Peter MacCallum Cancer Centre in Melbourne, Australia, and coordinating principal investigator of the study.

"Data from the Phase IB trial of BGB-283 further supported the anti-tumor activity in both RAF- and RAS-mutated tumors that we observed in the Phase IA study. We look forward to continuing clinical development of BGB-283 as either a single agent or in combination with other targeted or immuno-oncology agents," commented Amy Peterson, MD, Chief Medical Officer, Immuno-oncology at BeiGene.

Summary of Results

The multicenter, open-label Phase I trial of BGB-283 conducted in Australia and New Zealand is comprised of two parts – Phase IA (dose-escalation) and Phase IB (dose-expansion) in patients with B-RAF or K-RAS/N-RAS mutated solid tumors or patients with pancreatic cancer. The dose-expansion portion of the trial was designed to evaluate the safety and efficacy of BGB-283 at the recommended Phase II dose of 30 mg QD established in the Phase IA part of the trial. The expansion cohorts include patients with B-RAF V600-mutated cancers including melanoma, either naïve or having developed resistance to existing B-RAF or MEK inhibitors, colorectal cancer (CRC), non-small cell lung cancer (NSCLC), and papillary thyroid cancer (PTC), as well as patients with other B-RAF-mutated solid tumors. They also include patients with K-RAS/N-RAS-mutated endometrial cancer, NSCLC, and CRC, as well as patients with other K-RAS/N-RAS-mutated solid tumors and patients with pancreatic cancer.

The safety analysis, which included 96 patients as of the September 12, 2016 cut-off, suggested that BGB-283 was generally well-tolerated at 30 mg QD, with most drug-related adverse events (AEs) being grades 1 or 2 in severity. The most frequent drug-related AEs (≥10%) of any grade were fatigue (38.5%), dysphonia (26.0%), decreased appetite (21.9%), palmar-plantar erythrodysaesthesia syndrome (21.9%), thrombocytopenia (19.8%), dermatitis acneiform (17.7%), diarrhea (16.7%), rash (16.7%), nausea (15.6%), hypertension (11.5%), and glossodynia (10.4%). The most frequent drug-related grade 3-4 AEs (≥2%, 2 patients or more) included fatigue (7.3%), hypertension (6.3%), thrombocytopenia (6.3%), pyrexia (3.1%), hyponatremia (2.1%), anemia (2.1%), neutropenia (2.1%), febrile neutropenia (2.1%), decreased platelet count (2.1%), increased alanine aminotransferase (2.1%), increased gamma-glutamyltransferase (2.1%), and sepsis (2.1%).

The cut-off for the efficacy analysis was September 17, 2016. In seven patients with B-RAF V600-mutated melanoma (including one V600K and one V600R) who were naïve to B-RAF or MEK inhibitors, there were three partial responses (PRs) and three cases of stable disease (SD). In three patients with B-RAF V600-mutated PTC, there was one PR and two cases of SD. In six patients with K-RAS-mutated NSCLC, there was one PR and two cases of SD. In ten patients with solid tumors with B-RAF non-V600 mutations or solid tumors with B-RAF V600 mutations that are not included in other cohorts, there were two PRs, in one patient with BRAF V600E-mutated melanoma and one with BRAF V600E-mutated ovarian cancer, and three cases of SD. In two patients with B-RAF V600-mutated NSCLC, there was one unconfirmed PR and one case of SD.

Additional cases of SD were observed in four of six melanoma patients with B-RAF V600-mutated melanoma who had responses to but developed resistance against B-RAF or MEK inhibitors, nine of 13 patients with B-RAF V600-mutated CRC, five of five patients with K-RAS-mutated endometrial cancer, 12 of 20 patients with K-RAS/N-RAS-mutated CRC, and 10 of 21 patients with other K-RAS/N-RAS-mutated solid tumors or pancreatic cancer.

In the Phase IA portion of the study, confirmed objective responses included a complete response in a patient with B-RAF V600E-mutated melanoma, and two PRs, one in a patient with B-RAF V600E-mutated thyroid cancer and one in a patient with K-RAS-mutated endometrial cancer.

About BGB-283

Discovered by BeiGene scientists, BGB-283 is a novel RAF inhibitor with unique RAF dimer and EGFR inhibition activities. BGB-283 has shown antitumor activities in preclinical models and in cancer patients not only in tumors with BRAF V600E mutations but also those with non-V600E BRAF mutations and KRAS/NRAS mutations.

On April 2, 2017 Verastem, Inc. (NASDAQ: VSTM), focused on discovering and developing drugs to treat cancer, reported the oral presentation of data for its lead focal adhesion kinase (FAK) inhibitor, defactinib, by the Company’s scientific collaborator David G. DeNardo, PhD, Assistant Professor of Medicine, Division of Oncology, Department of Immunology, Washington University School of Medicine in St. Louis, at the 2017 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in Washington, DC (Press release, Verastem, APR 2, 2017, View Source [SID1234518406]).

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In an oral presentation titled, "Reprogramming the tumor microenvironment to improve responses to therapy," Dr. DeNardo described data demonstrating that FAK inhibition can enable efficacy of PD-1 inhibition in preclinical models of pancreatic cancer that, like the clinical disease, are otherwise refractory to checkpoint inhibition. As described in Dr. DeNardo’s presentation, a clinical trial is in progress, which combines Verastem’s FAK inhibitor defactinib with Merck’s PD-1 inhibitor pembrolizumab together with gemcitabine in patients with advanced pancreatic ductal adenocarcinoma (PDAC). Initial analysis of immune biomarkers from matched pairs of metastatic biopsies, taken either pre or post treatment, from patients with PDAC has been conducted in this study.

"Immunotherapeutic agents have shown little clinical benefit in pancreatic cancer and this is likely due, at least in part, to the presence of an immunosuppressive tumor microenvironment including a dense stroma which may prevent T cell entry into, or function within, tumor tissues," said Dr. DeNardo. "Our initial biomarker data, indicating an increase in activated proliferating cytotoxic T cells together with a reduction in tumor-associated macrophages (TAMs) appear promising. We are extremely interested to see whether these immune changes that may shift the balance from immunosuppressive to immunoreactive tumors, may translate into a clinical benefit for pancreatic cancer patients who have few effective treatment options."

Jonathan Pachter, PhD, Verastem’s Chief Scientific Officer, added, "Pancreatic cancer has the highest mortality rate of all major cancers, and the disease is still considered largely incurable. The data presented today by Dr. DeNardo at AACR (Free AACR Whitepaper) 2017 provide important support and rationale for the ongoing clinical study evaluating Verastem’s lead FAK inhibitor defactinib in combination with pembrolizumab and gemcitabine in patients with pancreatic cancer."
The ongoing Phase 1 clinical trial is being conducted at the Washington University School of Medicine in St. Louis under the direction of Andrea Wang-Gillam, MD, PhD, Clinical Director of the Gastrointestinal Oncology Program with financial support from the Precision Medicine Research Associates and the BJH Foundation. The trial, which is expected to enroll approximately 50 patients, is currently completing its dose-escalation portion.

Details for the oral symposium presentation at AACR (Free AACR Whitepaper) 2017 are:
Title: Reprogramming the Tumor Microenvironment to Facilitate Responses to Immunotherapy
Session: Major Symposium; SY14 – Myelomonocytic Cells and Stroma as Therapeutic Targets
Location: Room 206 – Level 2 Washington Convention Center
Date and time: Sunday, April 2, 2017 from 2:05-2:30pm ET
A copy of the oral presentation slides will be available here following the conclusion of Dr. DeNardo’s presentation and a webcast will be available from AACR (Free AACR Whitepaper) beginning on April 26, 2017.

About the Tumor Microenvironment
The tumor microenvironment encompasses multiple tumor and non-tumor cell populations and an extracellular matrix that support cancer cell survival. This includes immunosuppressive regulatory T cells, myeloid-derived suppressor cells, tumor-associated macrophages, cancer-associated fibroblasts and extracellular matrix proteins that can hamper the entry and therapeutic benefit of cytotoxic T cells and anti-cancer drugs. In addition to targeting the proliferative and survival signaling of cancer cells, Verastem’s product candidates, including duvelisib and defactinib, also target the tumor microenvironment to potentially improve a patient’s response to therapy.

About Defactinib
Defactinib is an investigational inhibitor of FAK, a non-receptor tyrosine kinase encoded by the PTK-2 gene that mediates oncogenic signaling in response to cellular adhesion and growth factors.1 Based on the multi-faceted roles of FAK, defactinib is used to treat cancer through modulation of the tumor microenvironment, enhancement of anti-tumor immunity, and reduction of cancer stem cells.2,3 Defactinib is currently being evaluated in combination with immunotherapy for the treatment of pancreatic cancer, ovarian cancer, non-small cell lung cancer, and mesothelioma, in three combination clinical trials with pembrolizumab or avelumab from Merck & Co. and Pfizer/Merck KGaA, respectively.4,5,6 Information about these and additional clinical trials evaluating the safety and efficacy of defactinib can be found on www.clinicaltrials.gov.

On April 2, 2017 Kite Pharma, Inc., (Nasdaq:KITE) reported two plenary presentations of positive data from the primary analysis of ZUMA-1 for its lead CAR-T candidate, axicabtagene ciloleucel, in patients with refractory aggressive B-cell non-Hodgkin lymphoma (NHL) at the 2017 American Association of Cancer Research Annual Meeting in Washington, D.C (Press release, Kite Pharma, APR 2, 2017, View Source [SID1234518399]). Both presentations were given by Frederick L. Locke, M.D., the ZUMA-1 Co-Lead Investigator, and Director of Research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida.

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The study met the primary endpoint of objective response rate (ORR) recorded after a single infusion of axicabtagene ciloleucel, with 82 percent (p < 0.0001). These results demonstrate the treatment effect of axicabtagene ciloleucel in diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL), which are types of aggressive NHL.

ZUMA-1 enrolled 111 patients of whom 101 were successfully treated with axicabtagene ciloleucel. ZUMA-1 patients were heavily pretreated and representative of those in the SCHOLAR-1 pooled analysis of refractory aggressive NHL. The key ZUMA-1 patient characteristics are below:

Stage III/IV disease (85 percent)
Refractory to chemotherapy, no prior autologous stem cell transplant (ASCT) (79 percent)
Relapsed within 12 months of ASCT (21 percent)
Received three or more lines of prior therapy (69 percent)
Refractory to two consecutive lines of prior therapy (54 percent)
The following table shows response data including the month 6 ORR and CR as well as ongoing response rates at the primary analysis data cut-off.

DLBCL (n=77) TFL/PMBCL (n=24) Combined (n=101)
ORR (%) CR (%) ORR (%) CR (%) ORR (%) CR (%)
ORR 82 49 83 71 82 54
Month 6 36 31 54 50 41 36
Ongoing 36 31 67 63 44 39

ORR was generally consistent in key subgroups. ORR in patients who are refractory to second or greater line of therapy was 83 percent and 76 percent in patients who relapsed within 12 month of ASCT.

With a median follow-up of 8.7 months, the median overall survival (OS) has not yet been reached. The overall duration of response (DOR) was 8.2 months and has not yet been reached for patients with a CR. In the SCHOLAR-1 pooled analysis, the median OS was estimated to be 6.6 months with only 8 percent achieving CR with currently available therapies.

As previously reported, the most common grade 3 or higher adverse events included anemia (43 percent), neutropenia (39 percent), decreased neutrophil count (32 percent), febrile neutropenia (31 percent), decreased white blood cell count (29 percent), thrombocytopenia (24 percent), encephalopathy (21 percent) and decreased lymphocyte count (20 percent). As compared to the interim analysis, grade 3 or higher cytokine release syndrome (CRS) decreased from 18 percent to 13 percent and neurologic events decreased from 34 percent to 28 percent. There were three deaths throughout the course of the trial not due to disease progression. Two events, one hemophagocytic lymphohistiocytosis (HLH) and one cardiac arrest in the setting of CRS, were deemed related to axicabtagene ciloleucel. The third case, a pulmonary embolism, was deemed unrelated. There were no cases of cerebral edema.

The accompanying plenary presentation reviewed the product characteristics and biomarker analysis from ZUMA-1. Axicabtagene ciloleucel was successfully manufactured (99 percent) from heavily pretreated patients with a broad range of baseline T cell numbers. Objective responses were observed across a wide range of product CD4:CD8 ratios, and were associated with higher levels of anti-CD19 CAR T cells in the blood. Immune signatures of CRS and neurological events were identified.

"We are excited to present the data from the primary analysis of ZUMA-1 at AACR (Free AACR Whitepaper). Close to half of patients treated remained in response, which represents a remarkable result in this heavily treated population with refractory aggressive NHL who previously faced a dismal outlook," said Jeff Wiezorek, M.D., Senior Vice President of Clinical Development. "We believe the rates of CRS and neurologic events decreased over the course of the study as clinicians gained experience in the management of adverse events. Biomarker analysis advances our understanding of product characteristics and immune signatures associated with clinical outcomes and provides us further opportunity to potentially improve the safety profile."

Kite recently announced completion of its rolling submission of the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA). Kite plans to submit a marketing authorization application (MAA) for axicabtagene ciloleucel for the treatment of relapsed or refractory DLBCL, PMBCL and TFL with the European Medicines Agency (EMA) in 2017.

Sattva S. Neelapu, M.D., Department of Lymphoma/Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center served as a co-lead investigator in the ZUMA-1 trial.

ZUMA-1 is supported in part by funding from The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program.

About axicabtagene ciloleucel

Kite’s lead product candidate, axicabtagene ciloleucel, is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. Axicabtagene ciloleucel has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.