On July 16, 2015 PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company focused on the discovery and development of innovative anti-cancer drugs based on PI3K and mTOR inhibition, reported the closing of an oversubscribed CHF 18 million (USD 19M) round of Series A2 financing (Press release, PIQUR Therapeutics, JUL 16, 2015, View Source [SID1234527274]). This round was backed by several existing investors, including Versant Ventures, as well as new, private investors.

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The new funding brings the total capital raised by PIQUR to date to over CHF 55 million (USD 60M). The company plans to use the funding to further strengthen its operational basis and to extend the financing horizon for additional Phase 2 studies for its lead compound PQR309, which has shown preclinical activity in different aggressive cancer cell lines inhibiting the PI3K/mTOR pathway, as well as clinical activity in phase 1. In addition, the new funding will support the preclinical safety and first clinical studies for one of its follow-up compounds.

"We greatly appreciate the continued support provided by our existing shareholders and welcome our new investors who bring valuable perspective, experience, and capital resources to the company," said Vladimir Cmiljanovic, CEO of PIQUR. Gaudenz von Capeller, PIQUR’s CFO, commented: "This financing strengthens our balance sheet as we work to achieve important milestones for our lead candidate as well as the follow-up compound."

Helping patients to survive cancer
PIQUR aims to help patients to survive cancer. Two out of three people are now living at least five years after their cancer has been diagnosed. Despite of significant medical innovations in the treatment of cancer, there remains a high unmet medical need for therapies that prolong patients’ survival and improve their quality of life. PIQUR targets both PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin), two key signaling molecules that are vital to several essential biological processes involved in malignant disease, such as cell growth, proliferation, survival and metastasis, making them attractive targets in cancer therapy.

About PQR309
PIQUR’s lead compound, PQR309, is a novel, oral, balanced pan- PI3K/mTOR inhibitor with excellent prospects to become a powerful anti-cancer drug. PQR309 compares favorably to current and clinically most advanced pan-PI3K/mTOR inhibitors with respect to the drug-like properties. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use to oncologic as well as hematologic malignant diseases involving the brain. PQR309 showed preclinical activity in different aggressive cancer cell lines inhibiting the PI3K/mTOR pathway, as well as clinical activity in phase 1.

On July 16, 2015 Cellectis reported the publication of a study in Cancer Research describing the applicability of TALEN-mediated genome editing to a scalable process, which enables the manufacturing of third-party CAR T-cell immunotherapies (Filing, 6-K, Cellectis, JUL 16, 2015, View Source [SID:1234506351]).

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Adoptive immunotherapy using autologous T-cells endowed with chimeric antigen receptors or CARs has emerged as a powerful means of treating cancer. However, a limitation of this approach is that autologous CAR T-cells must be generated on a custom-made basis.

To overcome the limitations of patient-derived CAR T-cell therapies, TALEN mediated gene inactivation can be used to generate non-alloreactive T-cells from third-party donors in a robust, scalable manufacturing process, thus allowing "off-the-shelf" CAR T-cell immunotherapies.

Laurent Poirot Ph.D. and his collaborators use this TALEN-mediated editing approach to develop a process for the large-scale manufacturing of T-cells deficient in expression of both their T-cell receptor (TCR) and CD52, a protein targeted by alemtuzumab, a chemotherapeutic agent. Functionally, T-cells manufactured with this process do not mediate graft-versus-host reactions, and are rendered resistant to destruction by alemtuzumab. These characteristics enable the administration of alemtuzumab concurrently or prior to engineered T-cells, supporting their engraftment.

Furthermore, endowing the TALEN-engineered cells with a CD19 CAR led to efficient destruction of CD19+ tumor targets even in the presence of the chemotherapeutic agent.

CAR T-cell immunotherapies can therefore be used in an "off-the-shelf" manner akin to other biological immunopharmaceuticals.

Laurent Poirot, Ph.D., Head of Early Discovery

Dr. Laurent Poirot studied physics and biology at the Ecole Polytechnique in France, before earning his Ph.D. at the Strasbourg University (France) and the Harvard Medical School in Boston. He then joined the Genomics Institute of the Novartis research foundation in San Diego as a postdoctoral fellow, where he studied the development of high throughput in vivo and in vitro approaches for the study of gene functions in immune cells. He joined Cellectis in 2009 as a Project Leader, and has been working as Head of Early Discovery since 2013.

Multiplex genome edited T-cell manufacturing platform for "off-the-shelf" adoptive T-cell immunotherapies

Laurent Poirot1, Brian Philip2, Cécile Schiffer Mannioui1, Diane Le Clerre1, Isabelle Chion-Sotinel1, Sophie Derniame1, Pierrick Potrel1, Cécile Bas1, Laetitia Lemaire1, Roman Galetto1, Céline Lebuhotel1, Justin Eyquem1,3, Gordon Weng-Kit Cheung2, Aymeric Duclert1, Agnès Gouble1, Sylvain Arnould1, Karl Peggs2, Martin Pule2, Andrew M. Scharenberg4 and Julianne Smith1

1 Cellectis, Paris, France
2 Department of Haematology, UCL Cancer Institute, University College London, London, UK
3 Current address: Memorial Sloan-Kettering Cancer Center, New York, NY
4 Current address: Department of Pediatrics, University of Washington, Seattle Children’s Research Institute, Seattle, WA

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On July 16, 2015 NanoString Technologies reported that the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for breast cancer have been updated to acknowledge that the PAM50 gene signature, commercialized as the Prosigna Breast Cancer Prognostic Gene Signature Assay, has been clinically validated for prediction of prognosis (Press release, NanoString Technologies, JUL 16, 2015, View Source [SID:1234506352]).

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"In line with our expectations, Prosigna is now recognized in the NCCN guidelines as providing clinically validated prediction of a woman’s risk of breast cancer recurrence," said Brad Gray, President and Chief Executive Officer of NanoString Technologies. "Discussion of Prosigna in the NCCN guidelines following our first submission is an important achievement, and we believe it establishes a solid foundation upon which we can continue to build the market for Prosigna."

The NCCN Guidelines are an authoritative source of information to help healthcare professionals make informed decisions about cancer care, and are often used by public and private payors to establish coverage policies. Prosigna’s acknowledgement in the NCCN Guidelines resulted from a review by a multidisciplinary panel of experts from NCCN member institutions, and is based on requests and clinical data submitted in June 2014. The newly updated guidelines (version 3.2015) appear on the NCCN website.

About the Prosigna Breast Cancer Prognostic Gene Signature Assay and nCounter Dx Analysis System

The Prosigna Assay provides a risk category and numerical score for assessment of the risk of distant recurrence of disease at 10 years in postmenopausal women with node-negative (Stage I or II) or node-positive (Stage II), hormone receptor-positive (HR+) breast cancer. Based on the PAM50 gene signature initially discovered by Charles Perou, Ph.D. and colleagues, the Prosigna Assay is an in vitro diagnostic tool that utilizes gene expression data weighted together with clinical variables to generate a risk category and numerical score to assess a patient’s risk of distant recurrence of disease. The Prosigna Assay measures gene expression levels of RNA extracted from formalin-fixed paraffin embedded (FFPE) breast tumor tissue previously diagnosed as invasive breast carcinoma.

The Prosigna Assay requires minimal hands-on time and runs on NanoString’s proprietary nCounter Dx Analysis System, which offers a reproducible and cost-effective way to profile many genes simultaneously with high sensitivity and precision.

The nCounter Dx Analysis System is a highly automated and easy-to-use platform that utilizes a novel digital barcoding chemistry to deliver high precision multiplexed assays. The system is available in the multi-mode FLEX configuration, which is designed to meet the needs of high-complexity clinical laboratories seeking a single platform with the flexibility to run the Prosigna Breast Cancer Assay and, when operated in the "Life Sciences" mode, process translational research experiments and multiplexed assays developed by the laboratory.

In the United States, the Prosigna Assay is available for diagnostic use when ordered by a physician. The Prosigna Assay has been CE-marked and is available for use by healthcare professionals in the European Union and other countries that recognize the CE Mark, as well as Canada, Israel, Australia, New Zealand, Turkey, South Africa and Hong Kong.

In the U.S., the Prosigna Assay is indicated in female breast cancer patients who have undergone surgery in conjunction with locoregional treatment consistent with standard of care, either as:

(1) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-negative, Stage I or II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors or (2) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-positive (1-3 nodes), Stage II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors. The device is not intended for patients with four or more positive nodes.

For more information, please visit www.prosigna.com.

On July 16, 2015 Navidea Biopharmaceuticals reported the results of voting at its 2015 Annual Meeting of Stockholders (the Annual Meeting) held July 16, 2015 (Press release, Navidea Biopharmaceuticals, JUL 16, 2015, View Source;p=RssLanding&cat=news&id=2068607 [SID:1234506354]). Approximately 80 percent of outstanding shares were represented at the meeting.

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At the Annual Meeting, Navidea’s stockholders:

Elected Gordon A. Troup to the Navidea Board of Directors to serve for a term of three years; and
Ratified the appointment of BDO USA, LLP to act as the Company’s independent registered public accounting firm for 2015.
The final results are subject to verification by the independent election inspectors and will be reported in a Form 8-K to be filed by Navidea with the Securities and Exchange Commission in the next few days.

Following the formal business portion of the Annual Meeting, Rick Gonzalez, Navidea President and CEO, made a brief presentation to stockholders in attendance at the Annual Meeting, including overviews on the following:

Lymphoseek (technetium Tc-99m tilmanocept) injection U.S. commercialization and life cycle management activities; and
Manocept CD206 targeting platform update, including a discussion of Macrophage Therapeutics development activities.

On July 15, 2015 Apogenix, a next generation immuno-oncology company, reported that it has entered into an exclusive licensing agreement with CANbridge Life Sciences, a biopharmaceutical company focused on developing Western drug candidates in China and North Asia, for the development and commercialization of lead immuno-oncology drug candidate APG101 in China, Macao, and Hong Kong (Press release, Apogenix, JUL 15, 2015, View Source [SID1234524582]). Under the terms of the agreement, Apogenix will receive upfront and milestone payments, as well as royalty payments at tiered, double-digit royalty rates following commercial launch of APG101 in China .

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APG101 is a CD95 ligand inhibitor which restores the immune response against tumors and inhibits invasive tumor cell growth. The drug candidate is being developed for the treatment of solid tumors and malignant hematological diseases. In a controlled phase II proof-of-concept trial in patients with recurrent glioblastoma, treatment with APG101 in combination with radiotherapy has demonstrated clinical superiority in all study endpoints compared to treatment with radiotherapy alone.

"The licensing agreement with CANbridge represents an important milestone in our goal to develop and commercialize our immuno-oncology compound APG101 as a new therapy for difficult-to-treat tumor indications on an international level," said Thomas Hoeger, Ph.D., Chief Executive Officer of Apogenix. "We are delighted to have found a strong and committed partner with a seasoned management team and extensive drug development expertise in these important Asian markets. We look forward to working with CANbridge to obtain approval for APG101 in China, Macao, and Hong Kong, so we can provide patients suffering from glioblastoma with a novel, much-needed therapeutic option as soon as possible."

In the phase II trial, glioblastoma patients expressing a certain biomarker associated with the CD95 ligand experienced the greatest benefit from treatment with APG101. The trial showed a statistically significant prolongation of overall survival in biomarker-positive patients treated with APG101, with a median overall survival of 16.1 months compared to 7.3 months in patients treated with radiotherapy alone. Apogenix is developing a companion diagnostic test based on this biomarker to identify those patients most likely to benefit from APG101.

"Development of this targeted therapeutic fits the CANbridge mission of bringing promising Western treatments to China and other Asian territories, where patients’ severe medical needs are going unmet," said James Xue, CANbridge Chairman and CEO. "The mortality rate of malignant glioma is one of the top ten among all cancers in China. With very limited treatment options, the outcomes for Chinese patients are even more grim than in the West. The potential to develop a targeted immuno-oncology product represents a tremendous advance for glioblastoma treatment in China."

"The CANbridge and Apogenix missions and cultures are perfectly aligned, which bodes well for the success of this partnership," said Henri Termeer, CANbridge’s Advisor and former Chairman and CEO of Genzyme Corporation. "Each company is dedicated to bringing forth treatments in their respective markets for patients with few options. Together, CANbridge and Apogenix can move this exciting program forward more effectively than either could alone in China."