Cellectis Reaches Milestone in Servier Collaboration

On July 8, 2015 Cellectis reported the achievement of a significant milestone under the Company’s collaboration agreement with Servier, in the preclinical development of two next-generation product candidates in solid tumors (Press release, Cellectis, JUL 8, 2015, View Source [SID:1234506188]).

Schedule your 30 min Free 1stOncology Demo!
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!

Under the terms of the Company’s collaboration agreement with Servier, Cellectis is eligible to an undisclosed payment.

The collaboration announced in February 2014, is focused on research, development, and potentially commercialization of five product candidates targeting leukemia and solid tumors.

"We are very pleased with the productivity of this alliance enabling us to accelerate our development in the field of solid tumors", commented Mathieu Simon, MD, EVP Chief Operating Officer of Cellectis.

"We believe that immunotherapy will dramatically change the management of metastatic cancers. Our goal at Servier is to make these new technologies available for the largest number of cancer patients", commented Jean-Pierre Abastado, MD, Director Oncology Innovation Therapeutic Pole of Servier.

UC San Diego and GSK Collaborate to Eradicate Cancer Stem Cells, Treat Leukemia

On July 08, 2015

Researchers at the University of California, San Diego School of Medicine and Moores Cancer Center are working with GSK on a bench-to-bedside project to treat leukemia and other diseases by eliminating cancer stem cells. The collaboration is part of GSK’s Discovery Partnerships with Academia (DPAc) program, where academic partners become core members of drug-hunting teams. Catriona Jamieson, MD, PhD, associate professor of medicine and chief of the Division of Regenerative Medicine, will lead UC San Diego’s effort in the new DPAc team.

Schedule your 30 min Free 1stOncology Demo!
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!

The UC San Diego-GSK DPAc team will collaborate to discover and develop a new therapeutic compound that inhibits RNA editing, a process cells use to enhance RNA diversity prior to translating genetic information into proteins. Jamieson’s group has shown that uncontrolled RNA editing helps cancer stem cells self-renew, making more malignant cells, in chronic myeloid leukemia and other blood cancers. Cancer stem cells can become dormant and evade chemotherapy and then activate again later, causing relapse of leukemia and other cancers and allowing tumors to resist treatment.

"The problem with leukemia is that in many cases while we can control the symptoms of disease, we can’t completely eradicate it because current therapies don’t block cancer stem cell self-renewal. Enhanced RNA editing is like a cancer stem cell engine, and with this collaboration we want to turn that engine off," said Jamieson, who is also deputy director of the Sanford Stem Cell Clinical Center and director of stem cell research at UC San Diego Moores Cancer Center. "We’re very fortunate to have this opportunity to work completely in step with a highly adept, dynamic company to take the RNA editing target idea from design to delivery of a new therapy that may prevent cancer relapse."

GSK launched DPAc as an independent unit in 2010. DPAc’s goal is to expedite promising basic research into drug discovery and development, with the potential to bring innovative new treatments to patients. Each selected project works as a joint team, with the academic and GSK scientists sharing data and working toward common goals. Through this arrangement, academic researchers benefit from GSK’s capabilities in medicinal chemistry and resources, such as state-of-the-art high-throughput screening technology — the expertise and automated equipment required to quickly and accurately screen millions of potential drug compounds for activity against a selected cellular target.

"We are honored to be selected to collaborate with GSK," said David A. Brenner, MD, vice chancellor for health sciences and dean of UC San Diego School of Medicine. "Paired with GSK’s expertise, our collaborative cancer stem cell research infrastructure, bolstered by the California Institute for Regenerative Medicine, National Cancer Institute and philanthropist T. Denny Sanford, places our DPAc team in the best possible position for success."

To be considered for DPAc, academic research proposals must have a clear therapeutic hypothesis, a defined target that can likely be addressed with a drug-like molecule and be led by researchers with deep target and disease expertise, access to model systems and clinical samples to progress the target.

"The GSK DPAc program is highly selective and works to identify the best academics with the best targets for partnership," said Carolyn Buser-Doepner, PhD, vice present and global head of DPAc at GSK. "The UC San Diego collaboration represents several firsts for the DPAc team at GSK — it is our first target in the emerging area of RNA editing and it is our first bench-to-bedside DPAc collaboration with a California university. Furthermore, Dr. Jamieson is an ideal collaborator with deep target and disease expertise, a highly productive preclinical research team with access to clinical samples and the demonstrated ability to translate studies into the clinic."

"Academic researchers do science well, but we often need industry partners to help translate those discoveries into new therapeutics that will make a difference to patient lives," said Ida Deichaite, PhD, director of industry relations at Moores Cancer Center. "This DPAc arrangement is a first for the University of California and will hopefully provide a model for future collaborations."

In addition to a strong collaborative infrastructure, Jamieson credits major support from School of Medicine and Moores Cancer Center leadership, including Brenner, Lawrence Goldstein, PhD, director of the Stem Cell Program, Wolfgang Dillmann, MD, chair of the Department of Medicine, and Scott Lippman, MD, director of the Moores Cancer Center.

"This is a wonderful example of academia-industry collaboration to accelerate drug development and clinical impact," said Lippman, "and opens the door for cancer stem cell targeting from a completely new angle — inhibition of aberrant RNA editing."

"Groundbreaking research on the basic behavior of stem cells — during human development and in the pathology of disease — has long been one of our strengths at UC San Diego," said Goldstein, who is also scientific director of the Sanford Consortium for Regenerative Medicine and director of the Sanford Stem Cell Clinical Center. "Now, those fundamental studies are paying off, as we begin to apply that information in the development of new therapies."

CELLSEARCH® System chosen as platform for new research on PD-L1 and Circulating Tumor Cells

On July 8, 2015 Janssen Diagnostics reported that its CELLSEARCH System technology was used in new research demonstrating the ability to detect PD-L1 expression on circulating tumor cells (CTCs) (Press release, Johnson & Johnson, JUL 8, 2015, View Source [SID:1234506187]). The results of this research appear in the article, "Frequent Expression of PD-L1 on Circulating Breast Cancer Cells," published online in Molecular Oncology, and demonstrate that PD-L1 positive CTCs were found in 11 out of 16 (68.8%) patients with circulating tumor cells.

Schedule your 30 min Free 1stOncology Demo!
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!

This particular application of the CELLSEARCH System is for research purposes only and has not been cleared by the US Food and Drug Administration. This publication potentially adds a new important application to the research use of the CELLSEARCH System to characterize CTCs for PD-L1. In performing their research, the investigators selected the CELLSEARCH System as an automated technical platform that has demonstrated consistent, reproducible results.

The importance of immune checkpoint regulators in oncology is becoming more widely understood and has given rise to the development of immunotherapies for cancer treatment. PD-L1 is an immune checkpoint regulator targeted by a number of approved and developmental oncology therapies. These results indicate that CTC analysis for PD-L1 expression is feasible and when confirmed, could open up the opportunity to predict response to certain therapies in future, larger studies using a blood test rather than tissue biopsy. In a research setting, the CELLSEARCH System offers the ability to capture CTCs via a routine blood test.

"I believe we have demonstrated that CTC characterization for PD-L1 expression is feasible using a CTC platform. This paves the way for the utilization of a CTC/PD-L1 assay in future clinical trials to explore whether it can stratify patients according to response, and potentially predict the efficacy of immune checkpoint blockade," comments study investigator Catherine Alix-Panabières, Ph.D., Director of the Laboratory of Rare Human Circulating Cells, University Medical Centre of Montpellier, University of Montpellier EA2415, Montpellier, France.

CTCs have the potential to allow real-time, dynamic monitoring of tumor characteristics without the need for repeated invasive biopsies. It was demonstrated earlier that CTC expression of key biomarkers such as HER21, IGFR2, c-MET3 and other proteins can be monitored successfully. This publication in Molecular Oncology underscores the potential of CELLSEARCH CTC testing to also become an important tool in cancer immunotherapy.

About CELLSEARCHSystem Technology

The CELLSEARCH System was honored with a Prix Galien USA Award in 2009 for Best Medical Technology on the basis of the innovative nature of its development, applicability and future biomedical potential.

For further information, please refer to the CELLSEARCH CXC Kit Instructions for Use at www.cellsearchruo.com. The application of the CELLSEARCH System described in this study is for research use only, and not for use in diagnostic procedures. The performance characteristics, safety, and effectiveness for this application have not been established and are not cleared or approved by the FDA.

CureVac Announces Phase I/IIa Clinical Study Data of its mRNA
Cancer Immunotherapy in Prostate Cancer Published in the Journal
for ImmunoTherapy of Cancer

On July 7, 2015 CureVac, a clinical-stage biopharmaceutical company pioneering the field of mRNA-based technology, reported that a Phase I/IIa study of the company’s mRNA cancer immunotherapy (CV9103) in advanced castration-resistant prostate cancer was published in the peer-reviewed Journal for ImmunoTherapy of Cancer ((Press release, CureVac, JUL 7, 2015, View Source [SID1234518778]). CV9103 is a self-adjuvanted, sequence-optimized, chemically unmodified mRNA immunotherapy targeting four antigens: prostate-specific antigen (PSA), prostate-specific membrane antigen (PSMA), prostate stem cell antigen (PSCA), and six- transmembrane epithelial antigen of the prostate 1 (STEAP1). The research article, titled "Self-adjuvanted mRNA vaccination in advanced prostate cancer patients: a first-in-man phase I/IIa study," describes CureVac’s clinical study of CV9103 in 44 patients with advanced castration-resistant prostate cancer. The related data signify the first Phase IIa clinical study in which an mRNA therapy has demonstrated antigen- specific immune responses in the majority of patients. Based on the favorable data, CureVac is currently conducting a randomized, placebo-controlled Phase IIb study in 197 prostate cancer patients with the follower vaccine CV9104 targeting six antigens. Ingmar Hoerr, CEO of CureVac, commented, "We are very pleased that the results of this Phase I/IIa clinical study of our RNActive technology were published in such an esteemed peer-reviewed journal as it validates our leadership position in mRNA therapeutics and highlights the continued advancement of our clinical pipeline. Prostate cancer remains our most advanced program, with the Phase IIb clinical trial progressing as planned, but CureVac also possesses a deep and diverse mRNA-driven pipeline spanning six clinical trials with more than 300 individuals treated so far and about 15 programs targeting multiple treatment opportunities and disease indications." As described in the article, the Phase I part of the study was designed to investigate the safety and recommended dosage of CV9103, with 12 patients up to five intradermal injections of 256 (n = 3), 640 (n = 3), or 1280 μg (n = 6) mRNA. In the Phase IIa part, 32 patients were enrolled to receive the recommended dose of 1280 μg mRNA defined in Phase I. The primary endpoint of the study was safety and tolerability, and the secondary endpoint was induction of antigen specific immune responses monitored at baseline and at weeks 5, 9 and 17. Data indicated that CV9103 was well tolerated, with the majority of related adverse events being of mild to moderate intensity. The most frequent treatment-related side effects were injection site erythema and injection site reaction in 27 (61%) and 21 (48%) patients, respectively. A quantitative analysis of ELISpot, ICS, and tetramer staining assays revealed that CV9103 was able to induce both CD4 and CD8 T cell responses. Of the 33 evaluable patients treated at 1280 μg, a cellular immune response could be detected in 25 (76%). Importantly immune responses against all four antigens could be induced indicating the versatility of the platform. Arnulf Stenzl, Medical Director of the Department of Urology, University of Tübingen Medical School, and senior author of the paper, stated, "The data generated by this Phase I/IIa clinical study demonstrate that CV9103 mRNA was well tolerated and immunogenic. Furthermore a trend towards longer survival time was also observed in immune responders that was strongest in patients responding to multiple antigens CV9103. Based on these results, it is evident that this mRNA technology warrants further clinical investigation." Just recently, CureVac published promising data of its RNArt technology platform in Molecular Therapy that demonstrated for the first time that sequence-optimized, chemically unmodified mRNAs raised relevant protein levels in non-human primates, indicating that mRNA achieves meaningful biological effects in large animals with body weight close to humans.

Schedule your 30 min Free 1stOncology Demo!
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!


Trials Show Promise for Locoregional Treatment for Hepatocellular Carcinoma and Liver Metastases

On July 7, 2015 Provectus Pharmaceuticals reported that varied locoregional treatments for hepatocellular carcinoma and metastatic colorectal cancer liver metastases are producing promising clinical results, according to a study presented here at the 2015 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer (ESMO-GI) (Press release, Provectus Pharmaceuticals, JUL 7, 2015, View Source [SID:1234506186]).

Schedule your 30 min Free 1stOncology Demo!
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!

"We have seen interesting data on locoregional therapies for hepatocellular carcinoma and colon cancer," stated Chris Verslype, MD, University Hospital, Leuven, Belgium, on July 4. "These therapies are gaining interest due to the fact that the liver is the predominant site of disease for most of these tumours."

Dr. Verslype commented on trials across several phases presented at the meeting.

Guy van Hazel, MD, University of Western Australia, Perth, Australia, updated and extended results, on July 4, of SIRFLOX, a phase 3 trial of first-line FOLFOX (folinic acid, 5-fluorouracil, and oxaliplatin)-based chemotherapy with or without selective internal radiation therapy (SIRT) in metastatic colorectal cancer patients with liver-dominant metastases. Improvement in progression-free survival, the primary endpoint, was not achieved in patients receiving SIRT. Disease progression in the liver, however, was reduced by 31% in patients receiving SIRT (SIR-Spheres; delivered as yttrium-90 microspheres via a hepatic artery injection).

Dr. van Hazel’s new SIRFLOX data pertained to 318 patients (159 FOLFOX plus bevacizumab; 159 FOLFOX plus bevacizumab plus SIRT) who had metastases only in the liver. Among them, progression-free survival with SIRT was extended by 8.7 months (FOLFOX plus bevacizumab, 12.4 months; FOLFOX plus bevacizumab plus SIRT, 21.1 months; hazard ratio [HR], 0.64; 95% confidence interval [CI], 0.48-0.86; P = .003). Among patients who had liver and extrahepatic metastases, however, the improvement in progression-free survival with SIRT was not significant (16.7 vs 12.6 months; P = .147). Dr. van Hazel noted that cumulative incidence of liver progression was superior for SIRT in patients with or without bevacizumab (P = .018 and .028, respectively).

"We saw that first-line radioembolisation in combination with bevacizumab and chemotherapy is able to retard progression in the liver, and it does seem to be safe," commented Dr. Verslype. "We feel, at our institution in Leuven, that we should restrict this to patients with very limited extrahepatic disease, where we can probably make a difference in the long run for our patients."

The phase 2 EORTC (European Organisation for Research and Treatment of Cancer) 40004/CLOCC trial, the first study prospectively investigating the efficacy of radiofrequency ablation added to standard systemic treatments in patients with nonresectable colorectal liver metastases, included 152 patients randomised to 6 months of systemic treatment with or without radiofrequency ablation. In ~75% of the patients, the systemic treatment was FOLFOX (with bevacizumab in ~17%). Prior reports showed the primary objective of overall survival of >38% at 30 months to have been met with 61.7% overall survival in the group receiving systemic treatment plus radiofrequency ablation and with 57.6% overall survival among those receiving systemic treatment only (NS), said Theo Ruers, MD, The Netherlands Cancer Institute, Antoni Van Leeuwenhoek Ziekenhuis, Amsterdam, The Netherlands, on July 3. Median progression-free survival, however, at a median follow-up of 4.4 years, was significantly longer for the radiofrequency group (16.8 vs 9.9 months; HR, 0.63; P = .025).

Progression-free survival at a median follow-up of 9.7 years was 2.0% for those receiving systemic therapy and 22.3% for systemic therapy plus radiofrequency ablation (HR, 0.57: 95% CI, 12.7-33.7; P = .005). Eight-year overall survival was 8.8% for systemic therapy alone and 35.9% for radiofrequency ablation (HR, 0.58, 95%; 95% CI, 0.38-0.88; P = .01). Progressive disease was the main cause of death (56.7% systemic plus radiofrequency ablation, 81.4% systemic only).

The findings, despite the small sample size, "encourage the use of ablative techniques as a treatment modality in patients with nonresectable colorectal liver metastases," said Dr. Ruer.

A third study, a phase 1 study of locoregional chemoablation with PV-10 (10% rose bengal solution), included patients with liver tumours (nonresectable hepatocellular carcinoma or liver metastases) of ≥1 cm.

The study demonstrated safety and activity, said Paul Goldfarb, MD, Sharp Clinical Oncology Research, San Diego, California, on July 2. PV-10 has shown high complete response rates and durable local control in phase 2 testing in metastatic melanoma and is currently in phase 3 testing.

This first presentation of 6 patients with evaluable liver data revealed only injection-site and photosensitivity reactions, with stable disease in all tumours and some partial responses. An expansion cohort includes 24 patients.

PV-10 is injected directly into lesions and has been shown to have effects on noninjected and distant lesions.

"We may expect some immunological effects with this kind of treatment, so locoregional treatment doesn’t necessarily mean that the effect of the therapy remains limited to the tumour itself in the liver. There may be effects on distant tumour sites," Dr. Verslype concluded.