MorphoSys Receives Clinical Milestone for Start of Phase 1 Trial with Novel Cancer Antibody

On October 20, 2016 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) reported that it has received a milestone payment from Novartis in connection with the initiation of a phase 1 clinical trial with a novel HuCAL antibody. The antibody will be tested in the field of cancer. Financial details were not disclosed (Press release, MorphoSys, OCT 19, 2016, View Source [SID1234515930]).

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"This is the 13th therapeutic antibody based on MorphoSys’s technologies that Novartis is evaluating in clinical trials, thus making our collaboration one of the most successful antibody alliances in our industry" commented Dr. Marlies Sproll, Chief Scientific Officer of MorphoSys AG. "During the course of this year, the MorphoSys pipeline has further matured and currently includes more programs in clinical trials than ever before. This reflects the great value of both MorphoSys’s technology and the resulting products, in our partnered as well as proprietary pipeline."

MorphoSys’s clinical pipeline now comprises 28 clinical programs. Two of them are in phase 3, 15 in phase 2 and 11 in phase 1. Novartis has currently 13 HuCAL antibodies in clinical development, of which six are in phase 2 and seven in phase 1.

Celgene Corporation and Sage Bionetworks Announce Technology Collaboration to Develop Observational Study Using the Apple ResearchKit Framework

On October 18, 2016 Celgene Corporation (NASDAQ: CELG) and Sage Bionetworks reported a collaboration to develop an iPhone application utilizing the Apple ResearchKit framework to improve the understanding of the burden of disease for patients living with chronic anemia due to myelodysplastic syndromes (MDS) or beta-thalassemia (Press release, Celgene, OCT 18, 2016, View Source [SID1234515895]).

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Smartphone-based apps like Sage Bionetworks’ mPower for Parkinson’s disease are increasingly being utilized as a tool by clinical study researchers to collect and analyze increasing volumes of patient-reported data in order to better capture and understand disease burden better and to improve therapeutic developments. Smartphone clinical study apps also enable the return communication of important study information to the patient participants.

"We stand at a point where technology is unlocking the ability to capture patient reported outcomes," said Michael Pehl, President, Hematology & Oncology for Celgene. "Through our collaboration with Sage Bionetworks and the evolving capability of smartphones and wearables as robust data collection devices, we believe we will be able to provide important new insights for patients with MDS and beta-thalassemia. We are pleased to be working alongside Sage Bionetworks and the patient community on this important project."

Celgene and Sage Bionetworks have chosen to address chronic anemia caused by myelodysplastic syndromes and beta-thalassemia. These diseases impose a great burden on affected individuals that is difficult to understand and quantify, and typically have clinical endpoints outside traditional measures. The collaboration seeks to move to a paradigm where this and other information like physiological testing are collected on a multi-dimensional and regular basis.

Already a leader in MDS, Celgene is currently developing three assets in the clinic across myelodysplastic syndromes and beta-thalassemia (CC-486, luspatercept, and enasidenib (AG-221/CC-90007)).

In addition to helping collect difficult to quantify data, the new mobile study will collect neurological assessments of patients using cognitive testing software from BrainBaseline, a leading technology for the self-assessment of cognitive performance. The app will also be an important channel for two-way communication and support for patients living with their disease – allowing them to understand their physical functioning and other symptoms of anemia.

Dr. Lara Mangravite, President of Sage Bionetworks stated, "We are thrilled to partner with Celgene to explore the use of sensor-based technologies to quantify the daily burden of disease in patients with chronic anemia. This is a first of its kind exploration from which we hope to gain insights that can be used to understand the impact of chronic anemia."

Celgene and Sage Bionetworks are working closely with the MDS Foundation (www.mds-foundation.org) and Cooleys Anemia Foundation (www.thalassemai.org) in defining the right elements for capture in the application to ensure patient relevance and applicability.

NanoString Enhances Solid Tumor Profiling with 3D Biology Capabilities for Formalin-Fixed, Paraffin-Embedded (FFPE) Tissue Samples

On October 18, 2016 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported the commercial launch of two new translational research assays in the company’s Vantage 3D product line at the American Society of Human Genetics (ASHG) Annual Meeting in Vancouver Canada:

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nCounter Vantage 3D DNA (SNV) Solid Tumor Panel
nCounter Vantage 3D Protein Solid Tumor Signaling Pathways Panel

The two new panels can be combined with one another as well as with the nCounter Vantage RNA Panels to enable more comprehensive profiling of formalin-fixed, paraffin-embedded (FFPE) tumor tissue (Press release, NanoString Technologies, OCT 18, 2016, View Source [SID1234515900]). The nCounter Vantage 3D Solid Tumor portfolio provides a deeper understanding of genotype and phenotype in various solid tumor cancers by enabling multiplexed, digital analysis of combinations of DNA, RNA, fusion genes, proteins and phospho-proteins simultaneously in a single experiment. Researchers now have the ability to extract more biological information from the limited tissue available from each sample using just two FFPE slides. Designed for flexibility, the nCounter Vantage 3D Solid Tumor portfolio provides new ways for cancer researchers to apply 3D Biology technology.

The nCounter Vantage 3D Protein Solid Tumor Signaling Pathways Panel enables the identification of activation state through the measurement of total proteins and phospho-proteins from a single slice of FFPE tissue or from as little as 250 ng of protein from cell or tissue lysate. The protein solid tumor panel can be used in combination with nCounter Vantage 3D DNA Panels and nCounter Vantage 3D RNA Panels, or in combination with the 770 gene PanCancer Pathways Panel to provide comprehensive solid tumor profiling.

"Simultaneous measurement of DNA, RNA and proteins could change how we assess and treat cancer," said Gordon B. Mills M.D. Ph.D., Professor and Chair of Systems Biology at MD Anderson Cancer Center. "This new 3D Biology platform allows for an efficient approach to overcome this hurdle, and the identification of new biomarkers and signatures from a single platform could provide key clinical insights that can lead to a more personalized approach to medicine. Importantly, this platform could rapidly move to the clinic, directly benefitting patients."

"Identifying clinically actionable mutations that can be correlated with unique signatures and cell response is key to our biomarker discovery and signature development," said E. Aubrey Thompson, Ph.D., Professor of Cancer Biology, Mayo Clinic. "In the past we would have had to run multiple assays to answer our research question. Now we can run a single analysis, saving time and precious FFPE samples."

Many experiments involve measurements of DNA, RNA, and protein to determine the biological impact and correlation of genotypic and phenotypic changes. Traditionally, the analysis of all three biomolecules typically requires three separate platforms. This traditional approach has two important limitations: inefficient use of small samples and data compatibility challenges from using multiple platforms which creates uncertainty in determining whether discrepancies are due to biological factors or errors in measurement. The nCounter Vantage 3D Solid Tumor portfolio enables the simultaneous measurement of DNA, RNA and protein, addressing previous limitations.

The new products expand the portfolio of 3D Biology panels for analyzing solid tumors and FFPE tissue. The nCounter Vantage 3D DNA (SNV) Solid Tumor Panel is designed for highly multiplexed profiling of known cancer mutations, as well as insertions and deletions (INDELs) from as little as 5 nanograms of DNA.

NanoString will host a workshop at ASH (Free ASH Whitepaper)G where collaborators from MD Anderson Cancer Center and Fred Hutchinson Cancer Research Center will present data that demonstrate the robustness and sensitivity of the assay across a wide range of FFPE samples. The presentation, "3D Biology: Simultaneous Single-Molecule Quantification of DNA (SNVs), mRNA, Fusion Genes and Proteins Using Molecular Barcodes," will be held in Room 10 of the East Building, on Wednesday, October 19th from 1:00-2:30pm Pacific Time.

These new product launches demonstrate NanoString’s continued commitment to expanding the boundaries of biological research through 3D Biology. At the 2016 ASH (Free ASH Whitepaper)G Annual Meeting in Vancouver, British Columbia, NanoString will showcase nCounter Vantage 3D Solid Tumor capabilities at booth #512.

LabCorp Subsidiary Sequenom, Inc. Announces Expiration of and Final Tender Results for Tender Offers for 5% Convertible Senior Notes Due 2017 and 5% Convertible Senior Exchange Notes Due 2018

On October 18, 2016 Sequenom, Inc. ("Sequenom"), a wholly-owned subsidiary of Laboratory Corporation of America Holdings (LabCorp) (NYSE: LH), reported the final results, as of 5:00 p.m., New York City time, on Monday, October 17, 2016 (the "Expiration Date"), of the cash tender offers (the "Tender Offers") for any and all of the outstanding 5% Convertible Senior Notes Due 2017 (CUSIP No. 817337 AB4, the "2017 Notes") and 5% Convertible Senior Exchange Notes Due 2018 (CUSIP No. 817337 AC2, the "2018 Notes" and, together with the 2017 Notes, the "Notes") issued by Sequenom (Press release, LabCorp, OCT 18, 2016, View Source;p=RssLanding&cat=news&id=2212557 [SID1234515901]).

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The depositary for the tender offers has advised Sequenom that, as of the Expiration Date, (i) a total of $44,871,000 aggregate principal amount of the outstanding 2017 Notes, representing approximately 99.713% of the outstanding 2017 Notes, were validly tendered (and not validly withdrawn), and (ii) a total of $85,000,000 aggregate principal amount of the outstanding 2018 Notes, representing 100.00% of the outstanding 2018 Notes, were validly tendered (and not validly withdrawn) in the Tender Offers.

Sequenom has accepted for purchase all 2017 Notes and 2018 Notes properly tendered and not validly withdrawn in the Tender Offers. Holders whose 2017 Notes were validly tendered on or before 5:00 p.m., New York City time, on September 29, 2016 (the "Early Tender and Consent Payment Deadline"), and not withdrawn, will receive, in respect of each $1,000 principal amount of 2017 Notes, the "2017 Total Consideration" of $1,037.50 plus Accrued Interest (such price being rounded to the nearest $0.01 per $1,000 principal outstanding amount of Notes) on October 20, 2016 (the "Settlement Date"). Holders whose 2018 Notes were validly tendered on or before the Early Tender and Consent Payment Deadline, and not withdrawn, will receive, in respect of each $1,000 principal amount of 2018 Notes, the "2018 Total Consideration" of $1,046.25 plus Accrued Interest (such price being rounded to the nearest $0.01 per $1,000 principal outstanding amount of Notes) on the Settlement Date. Holders whose 2017 Notes were validly tendered after the Early Tender and Consent Payment Deadline but on or prior to the Expiration Date, and not withdrawn, will receive, in respect of each $1,000 principal amount of 2017 Notes tendered, the "Purchase Price" of $1,000.00, plus accrued interest to but excluding the Settlement Date. No tenders of Notes will be valid if submitted after the Expiration Date.

Barclays acted as the dealer manager and solicitation agent for the Tender Offers.

Sirenas and Calibr to Form Drug Discovery Collaboration

On October 18, 2016 Sirenas, LLC and the California Institute for Biomedical Research (Calibr) reported that they are collaborating in an effort to discover and develop small molecules in oncology, neuroscience, regenerative medicine and neglected disease (Press release, Sirenas, OCT 18, 2016, View Source [SID1234517478]). Sirenas will provide Calibr with access to its marine-derived chemical diversity, AtlantisTM data mining technology, and synthetic chemistry expertise. Calibr will evaluate compounds in a series of novel biological assays and preclinical disease models.

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"Sirenas has combined the rich chemical diversity found in marine organisms with enabling analytics and informatics to create a powerful platform that we believe could be valuable in targeting some of the most intractable disease phenotypes," said Pete Schultz, President and CEO of Calibr. "We are delighted to pair the tools and expertise we have developed at Calibr with Sirenas’ technology to tackle unmet public health needs."

"Marine organisms have evolved over hundreds of millions of years in highly competitive environments, which has allowed them to evolve very diverse, potent chemistry," said Eduardo Esquenazi, Ph.D., founder and CEO of Sirenas. "In the past, the challenges of identifying and translating those compounds has hindered their pursuit, but we have focused on building an approach that can quickly and cost effectively bring forth these novel compounds into new avenues of drug discovery research. The collaboration with Calibr provides significant biological capabilities to better understand how our compounds work against a number of disease targets."

Financial terms of the collaboration were not disclosed, but the parties plan to co-develop promising leads through definitive preclinical and early clinical studies.