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4SC announces results for financial year 2016

On 29 March 2017 4SC AG (4SC, FSE Prime Standard: VSC) today published the financial results of the 4SC Group for the financial year ended 31 December 2016 that presents all material reporting period developments and provides an outlook for 2017 (Press release, 4SC, MAR 29, 2017, View Source [SID1234518598]). The full report is available at 4SC’s website.

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Jason Loveridge, Ph.D., CEO of 4SC, commented:

"Since I joined the company in September 2016, we have focused our resources on our lead clinical assets, resminostat and 4SC-202. We expanded our senior leadership team and are now well set-up to further advance our products in clinical development and toward market approval.

We look forward to 2017 as a year in which we can make significant progress at 4SC. We will continue patient enrollment in our pivotal study RESMAIN of resminostat in cutaneous T-cell lymphoma (CTCL) and are actively discussing the next steps for resminostat in liver cancer (hepatocellular carcinoma, HCC) following the interesting subgroup data generated in 2016. We also plan to initiate two Phase II studies of 4SC-202 in combination with checkpoint inhibitors and to initiate formal preclinical development of 4SC-208.

Throughout 2017, we expect to make significant progress and further enhance the value of 4SC through out-licensing deals of further non-core assets."

Key highlights of 2016 and 2017 YTD

Announced positive data from subgroup analysis of the Phase II study of resminostat in combination with sorafenib as first-line therapy in 170 Asian patients with advanced-stage HCC conducted by Yakult Honsha Co., Ltd., which showed a substantial increase in overall survival in patients with above the median platelet levels at study entry (50% of study population).
Received investigational new drug approval (IND) from the U.S. Food and Drug Administration (FDA) to conduct a clinical study of resminostat in combination with sorafenib in HCC.
Initiated patient enrollment in the pivotal study RESMAIN of resminostat as maintenance therapy in patients with advanced-stage CTCL conducted in 11 European countries at more than 50 study centers.
Announced strong preclinical data on 4SC-202’s potential in both monotherapy as well as in combination with checkpoint inhibitors.
Appointed Jason Loveridge, Ph.D., as Chief Executive Officer. Promoted Frank Hermann, M.D., to Chief Development Officer and Roland Baumgartner, Ph.D., to Chief Scientific Officer.
Established an international Scientific Expert Panel (iSEP) to provide 4SC with strategic counsel on research and clinical product development.
Sold operations of 4SC Discovery to BioNTech Small Molecules GmbH.
Sold the immunology portfolio to Immunic AG in exchange for a one-time upfront payment, milestone payments and royalties on sales.
Licensed 4SC-205 to Guangzhou LingSheng Pharma Tech Co., Ltd (Link Health) for development and marketing of 4SC-205 in China, Hong Kong, Taiwan and Macao in exchange for upfront and milestone payments totaling up to €76 million.

Business outlook for 2017

Continue enrolling patients in the pivotal study RESMAIN of resminostat in CTCL.
Define the optimal route forward for resminostat in HCC.
Initiate two Phase II studies of 4SC-202 in combination with checkpoint inhibitors.
Initiate formal preclinical development of 4SC-208.
Generate further out-licensing deals for non-core assets and continue evaluating potential partnering opportunities with pharmaceutical and biotech companies to progress the clinical development of 4SC’s core pipeline assets.

Development of cash balance in full year 2016 and financial forecast

4SC’s cash balance/funds amounted to €11,333 thousand as of 31 December 2016, compared with €22,794 thousand as of 31 December 2015. The decrease relates to an average monthly outflow of cash from operations of €827 thousand as well as to the repayment of a shareholder loan amounting to €1,500 thousand. 4SC currently estimates that the liquid funds earmarked for its financing will be sufficient for another twelve months.

NanoString Launches Multiple New Products at AACR 2017 to Advance Precision Oncology Research

On March 29, 2017 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported that it is releasing multiple new products at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Meeting (Press release, NanoString Technologies, MAR 29, 2017, View Source [SID1234518314]). The new products build on NanoString’s existing portfolio of products that leverage genomics and proteomics to advance precision oncology.

"We continue to enhance the capabilities and expand the content for our nCounter platform. Our goal is to deliver best-in-class solutions that address the complex challenges that are inherent in precision oncology," said Brad Gray, president and chief executive officer of NanoString Technologies.

New products released include the following:

The nCounter Low RNA Input Kit enables nCounter Gene Expression analysis to be performed with sample inputs ranging from 1-10 ng, depending on sample quality. The kit is compatible with NanoString’s panel products as well as custom offerings. It utilizes Multiplexed Target Enrichment (MTE) to linearly amplify mRNA sample transcripts and is compatible with a wide range of sample types including Formalin Fixed Parafin Embedded (FFPE) tissue.

The nCounter Vantage 3D RNA MAPK-PI3K Pathways Panel provides a curated gene set measuring transcriptional activity of the MAPK and PI3K pathways on the nCounter platform. Seminal publications focused on MAPK and PI3K research and signature development were collated and scored to develop this panel, which captures the diverse set of biological functions that the MAPK and PI3K pathways modulate. As part of the Vantage 3D portfolio, these panels pair with the Vantage 3D DNA SNV and Protein Solid Tumor Panels for integrated DNA, RNA, and protein quantification in solid tumor research.

The Intracellular Compatible Universal Cell Capture Kit enables the nCounter Vantage 3D RNA:Protein Immune Cell Signaling Assay with as few as 20,000 cells for expression analysis of 770 RNA and 26 key intracellular proteins, including cytokines, chemoattractants, and transcription factors. In combination with the new Low RNA Input Kit, both the Vantage 3D RNA:Protein Immune Cell Profiling and Immune Cell Signaling Assay provide in depth immune characterization from minimal sample.

The nCounter Myeloid Innate Immunity Panels provide researchers studying the role of myeloid innate immune response in cancer and other diseases a tool for rapid, comprehensive gene expression profiling of human or mouse RNA. Designed in partnership with Lisa Coussens, Ph.D., of Oregon Health and Sciences University, the panels enable quantitative evaluation of heterogeneous myeloid cell populations based on the differentiation and maturation status as well as functional activities.
NanoString will be exhibiting at AACR (Free AACR Whitepaper) (booth# 3115) and hosting a workshop entitled "Powering Precision Oncology Research with 3D Biology Technology: High Plex Multi-Analyte Profiling on FFPE with Spatial Resolution" at 10:00 AM on Monday April 3. NanoString is also featured in more than 50 presentations and posters that will be presented throughout the conference.

Preclinical Data for Kura Oncology Development Compounds to be Presented at AACR Annual Meeting 2017

On March 29, 2017 Kura Oncology, Inc. (Nasdaq:KURA), a clinical stage biopharmaceutical company focused on the development of precision medicines, reported that results from preclinical studies of tipifarnib, KO-947, its development candidate targeting ERK1/2 kinases, and KO-539, its development candidate targeting the menin-MLL interaction, will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017 (Press release, Kura Oncology, MAR 29, 2017, View Source [SID1234518313]). The AACR (Free AACR Whitepaper) Annual Meeting will be held at the Walter E. Washington Convention Center in Washington, D.C. from April 1-5, 2017.

The following posters will be presented during the conference. Abstracts are available at www.aacr.org.

Apr. 3, 2017, 1:00 p.m. – 5:00 p.m. EDT – Abstract 2063/20 (Poster): The farnesyltransferase inhibitor tipifarnib causes dramatic tumor regression and increases survival in murine HrasG12V driven aggressive thyroid cancers: Consequent adaptive and acquired resistance mechanisms inform combination treatments with improved responses. B. Untch et al., Memorial Sloan Kettering Cancer Center, New York, NY

Apr. 5, 2017, 8:00 a.m. – 12:00 p.m. EDT – Abstract 5168/11 (Poster): KO-947, a potent ERK inhibitor with robust preclinical single agent activity in MAPK pathway dysregulated tumors. F. Burrows et al., Kura Oncology, La Jolla, Calif.

Apr. 5, 2017, 8:00 a.m. – 12:00 p.m. EDT – Abstract 5077/22 (Poster): A novel small molecule menin-MLL inhibitor for potential treatment of MLL-rearranged leukemias. T. Wu et al., Wellspring Biosciences, La Jolla, Calif.

Epizyme to Present New Preclinical Data on Tazemetostat and Epigenetic Target Identification Efforts at AACR Annual Meeting

On March 29, 2017 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported that new preclinical data on tazemetostat, Epizyme’s lead product candidate and first-in-class EZH2 inhibitor, along with other epigenetic target identification efforts, will be presented in poster sessions at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017 taking place in Washington, D.C., April 1-5, 2017 (Press release, Epizyme, MAR 29, 2017, View Source [SID1234518311]).

"2017 continues to be a transformational year for Epizyme as we work to rewrite cancer treatment through novel epigenetic medicines," said Rob Bazemore, CEO of Epizyme. "We look forward to sharing these new data on tazemetostat and our innovative approach to advancing our preclinical pipeline with the oncology community at AACR (Free AACR Whitepaper)."

"As we evaluate these data, we are particularly encouraged by new research revealing the important role EZH2 plays in the proliferation of multiple myeloma, and preclinical activity of our first-in-class EZH2 inhibitor, tazemetostat, as both monotherapy and combination therapy in in vitro models of multiple myeloma," said Richard Chesworth, DPhil, senior vice president of research at Epizyme. "These findings reinforce the potential for tazemetostat as a treatment option across multiple B-cell malignancies."

Multiple myeloma is a cancer arising from terminally differentiated B-cell lymphocyte plasmablasts. Mounting evidence suggests that EZH2 is an important regulator of B-cell differentiation and may play an important role in clinical B-cell malignancies. Consistent with this role, inhibition of EZH2 alone has shown potent anti-proliferative effects in in vitro and in vivo preclinical models of multiple myeloma.

In a new study being presented at AACR (Free AACR Whitepaper), Epizyme evaluated the efficacy of tazemetostat, an EZH2 inhibitor, as monotherapy and in combination with standard of care agents in preclinical models of multiple myeloma. Tazemetostat selectively inhibited intracellular H3K27 methylation in multiple myeloma cell lines and elicited a robust anti-proliferative effect in 14-day assays. Synergistic activity was observed when tazemetostat was combined with commonly used multiple myeloma therapeutics (glucocorticoid receptor agonists and small molecule immune system modulators) when cells were treated with tazemetostat for seven days prior to the addition of the standard-of-care drugs. Based on these results, studies with selected therapeutic modalities were expanded into in vivo xenograft models to further evaluate monotherapy and combination activity of tazemetostat in multiple myeloma.

Data will also be presented from preclinical studies demonstrating synergistic activity following the addition of tazemetostat to current small molecule treatments for malignant rhabdoid tumors and atypical teratoid rhabdoid tumors, both rare and aggressive forms of cancer with high unmet medical need, typically affecting pediatric patients. In addition, Epizyme will present data demonstrating that tazemetostat induces potent and selective apoptosis in SMARCA2 and SMARCA4-deficient ovarian cell lines, which confirms SCCOHT (small cell carcinoma of the ovary hypercalcemic type) is also sensitive to CRISPR-mediated EZH2 gene ablation. These findings support Epizyme’s ongoing Phase 2 trial of tazemetostat in solid tumors, and may support future clinical evaluation in other tumor types such as lung cancers.

Details of the poster presentations are as follows:

Date & Time: Sunday, April 2, 1:00 – 5:00 p.m. ET
Title: CRISPR pooled screening of hundreds of cancer cell lines identifies differential dependencies on epigenetic pathways and synthetic lethal relationships
Abstract Number: 406/6
Location: Section 17

Date & Time: Monday, April 3, 8:00 a.m. – 12:00 p.m. ET
Title: Tazemetostat displays synergistic antiproliferative activity with backbone therapies in preclinical models of AT/RT and MRT
Abstract Number: 1944/16
Location: Section 42

Date & Time: Tuesday, April 4, 8:00 a.m. – 12:00 p.m. ET
Title: Selective killing of SMARCA2- and SMARCA4-deficient tumors by inhibition of EZH2: In vitro and in vivo preclinical models
Abstract Number: 3345/6
Location: Section 15

Date & Time: Wednesday, April 5, 8:00 a.m. – 12:00 p.m. ET
Title: Activity of the EZH2 inhibitor tazemetostat as a monotherapy and in combination with multiple myeloma therapies in preclinical models
Abstract Number: 5060/5
Location: Section 2

Cellectis to Present at the 2017 Annual Conference of the American Association for Cancer Research

On March 29, 2017 Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Alternext: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells (UCART), reported that data on its gene-edited allogeneic CAR T-cells will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Filing, 6-K, Cellectis, MAR 29, 2017, View Source [SID1234518310]). This includes both wholly-controlled Cellectis programs and Pfizer/Cellectis collaboration programs. The meeting will be held from April 1st to April 5th, 2017 in Washington, D.C., USA.

Cellectis presentation:

UCART22: An Allogeneic Adoptive Immunotherapy for Leukemia Targeting CD22 with CAR T-cells
Agnès Gouble1, Cécile Schiffer-Mannioui1, Séverine Thomas1, Anne-Sophie Gautron1, Laurent Poirot1, Julianne Smith2.
1Cellectis, Paris, France; 2Cellectis, New York, NY

Innate Effectors in Immunity to Cancer session – section 30
April 4, 2017, 8:00 AM – 12:00 PM Eastern Time

Pfizer/Cellectis presentations:

Allogeneic EGFRvIII Chimeric Antigen Receptor T Cells for Treatment of Glioblastoma
Oi Kwan Wong1, Mathilde Dusseaux2, Jing-Tyan Ma1, Melinda Au1, Sophie Leduc2, Joyce Chou1, Jessica M. Yu1, Marjorie Bateman1, Thomas Pertel1, Kevin C. Lindquist1, Julianne Smith3, Barbra Sasu1, Shu-Hui Liu1.
1Pfizer Inc., South San Francisco, CA; 2Cellectis, Paris, France; 3Cellectis, New York, USA

Innate Effectors in Immunity to Cancer session – section 30
April 4, 2017, 8:00 AM – 12:00 PM Eastern Time

Differential modulation of the PD-1 pathway impacts the anti-tumor activity of CAR T cells
Regina J. Lin1, Anne-Sophie Gautron2, Laurent Poirot2, Oi Kwan Wong1, Barbra Sasu1, Bijan Boldajipour1.
1Pfizer, South San Francisco, CA; 2Cellectis, Paris, France; 3Cellectis, New York, USA

Late-Breaking Research: Immunology session – section 35
April 4, 2017, 8:00 AM – 12:00 PM Eastern Time

Collaboration with Pfizer

In June 2014, Cellectis and Pfizer began collaborating on a joint clinical development program for UCAR-T therapies, in the field of oncology, directed at numerous targets selected by Pfizer and several targets selected by Cellectis.