On November 30, 2017 Mylan N.V. (NASDAQ, TASE: MYL) and Biocon Ltd. (BSE code: 532523, NSE: BIOCON) reported that the European Medicines Agency (EMA) has accepted for review Mylan’s Marketing Authorization Applications (MAA) for proposed biosimilar trastuzumab and proposed biosimilar pegfilgrastim (Press release, Mylan, NOV 30, 2017, View Source [SID1234522323]). Trastuzumab is used to treat certain HER2-positive breast and gastric cancers. Pegfilgrastim is used to reduce the duration of neutropenia (low count of neutrophils, a type of white blood cells) and the incidence of fever associated with neutropenia in adult patients treated with chemotherapy in certain types of cancer.

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EMA acceptance of the submissions follows an earlier withdrawal of both applications in response to an audit conducted by the European inspecting authority of Biocon’s drug product facility. Biocon has completed the Corrective and Preventive Actions (CAPAs) outlined as a result of the audit observations. The CAPAs will be confirmed during reinspection, which will be completed as part of the regulatory review process.

Good Manufacturing Practice (GMP) compliance certificates for Biocon’s two drug substance manufacturing facilities in Bangalore have been issued previously. Approval of these sites is key in the development and approval process as drug substance manufacture is core to the production of the actual biologic product in GMP compliance.

Mylan President Rajiv Malik commented: "We are extremely pleased with the acceptance of our MAAs. Having gone through initial reviews of the applications and after completing the CAPAs from the EMA audit, we are even more confident with the strength of our MAAs. Additionally, the Voluntary Action Indicated (VAI) designation we received from FDA gives us further confidence in the readiness of the manufacturing site. We now look forward to moving ahead with the rest of the regulatory review process in Europe and to bringing these important treatment options to cancer patients.

"These developments also demonstrate Mylan’s commitment to our partnership with Biocon, the strength of the collaboration’s scientific and manufacturing capabilities, and our relentless approach to increasing access to and affordability of important treatment options in Europe and around the world through the introduction of biosimilars."

Dr Arun Chandavarkar, CEO & Joint Managing Director, Biocon said: "EMA’s acceptance for review of the Marketing Authorization Applications (MAAs) for our proposed biosimilars of trastuzumab and pegfilgrastim is indeed a welcome development. These applications were resubmitted upon completion of the Corrective and Preventive Actions (CAPAs), including the modifications of our aseptic drug product facility. We expect these CAPAs to be verified during inspection as part of the review process. We continue to work closely with our partner Mylan in engaging with EMA to provide these high quality, affordable therapy options for cancer patients in Europe."

On November 30, 2017 GT Biopharma, Inc. (OTCQB: GTBP and Euronext Paris GTBP.PA) ("GT Biopharma" or the "Company") reported that Dr. Jeffrey Miller, Deputy Director of the Masonic Cancer Center, University of Minnesota, will be presenting data on its second-generation anti-CD16-IL-15-anti-CD33 TriKE (OXS-C3550); another first of its kind, single-chain, tri-specific NK cell engager (TriKE) (Press release, GT Biopharma , NOV 30, 2017, View Source [SID1234539536]).

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The TriKE platform technology, developed by Dr. Miller and his colleagues at the University of Minnesota, is designed to enhance the activity of Natural Killer (NK) cells. NK cells are a type of white blood cell which are an important component of the innate immune system and play a major role in the rejection of tumor and virally infected cells.

The original anti-CD16-IL-15-anti-CD33 TriKE (OXS-3550) utilizes the inclusion of a modified Interleukin-15 (IL-15), a peptide that activates NK cells, while the "engager" further increases NK cancer-cell killing capabilities and improves their function in the tumor microenvironment (Vallera et al, 2016). OXS-3550 is expected to enter human clinical trials in 2018. OXS-C3550, the second-generation anti-CD16-IL-15-anti-CD33 TriKE, utilizes a modified anti-CD16 component while incorporating the wild-type IL-15.

The OXS-C3550 TriKE was developed following continued research with the TriKE platform at the University of Minnesota Masonic Cancer Center. As demonstrated in non-clinical models, this targeted immunotherapy directs immune cells to kill cancer cells while diminishing drug-related toxicity.

The TriKE platform technology can be viewed as a protein version of CAR-T; but unlike traditional CAR-T platforms, it is anticipated that TriKEs could service a much larger part of the cancer population at a fraction of the cost. TriKEs are an antibody platform that could be tailored to treat any form of cancer, liquid or solid tumors.

OXS-C3550 will focus on acute myeloid leukemia (AML), the most common form of adult leukemia with 43,000 new cases each year. These patients will require frontline therapy, usually chemotherapy including cytarabine and an anthracycline, a therapy that has not changed in over 40 years. Also, about half of these patients are likely to have relapses and require alternative therapies. In addition, OXS-C3550 could be used to treat myelodysplastic syndrome (MDS), which has about 20,000 new cases diagnosed each year with minimal current treatment options (Siegel et al, 2014). At a minimum, OXS-3550 is expected to serve as a relatively safe, inexpensive, and easy to use therapy for resistant or relapsing AML. From a biologic standpoint, it could also be combined with chemotherapy as frontline therapy.

GT Biopharma Chief Medical officer (CMO) Dr. Raymond Urbanski said, "The data on our second-generation TriKE, which will be presented at the upcoming ASH (Free ASH Whitepaper) meeting, will be noticed by investigators in the field and will set the tone for all future discussions on the use of NK cells in the treatment of cancer. We believe the distinctions and potential benefits relative to other immunotherapies, including CAR-T, will become apparent."

GT Biopharma Chief Executive Officer (CEO) Dr. Kathleen Clarence-Smith said, "In collaboration with the experts from the University of Minnesota Masonic Cancer Center, GT Biopharma continues to advance the search for next generation anti-cancer treatments, especially on novel ways to enhance the cancer-killing capabilities of NK cells. In my view, the potential for the success of this immunotherapy approach is substantial and the possibility of extending it to solid tumors gives us even added hope."

GT Biopharma Executive Chairman Anthony J. Cataldo said, "This is another milestone achievement for the company as we continue to close out a very productive 2017."

On November 30, 2017 Foundation Medicine, Inc. (NASDAQ:FMI) reported that the U.S. Food and Drug Administration (FDA) approved FoundationOne CDx, the company’s comprehensive companion diagnostic test for solid tumors (Press release, Foundation Medicine, NOV 30, 2017, View Source [SID1234522325]). FoundationOne CDx is intended for use by health care professionals to help inform cancer treatment management in accordance with professional guidelines for patients with solid tumors. The first and only FDA-approved test of its kind for all solid tumors, FoundationOne CDx is a diagnostic test that acts as:

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a comprehensive companion diagnostic to identify patients who may benefit from treatment with specific FDA-approved targeted therapies;
a comprehensive genomic profiling (CGP) test that includes genomic biomarkers to help inform the use of other targeted oncology therapies, including immunotherapies;
a tool for physicians that identifies patient opportunities for clinical trial participation; and,
an FDA-approved platform for companion diagnostic development for biopharma companies developing precision therapeutics.
FoundationOne CDx assesses all classes of genomic alterations in 324 genes known to drive cancer growth, providing potentially actionable information to help guide treatment decisions. It is also indicated as a companion diagnostic for patients with certain types of non-small cell lung cancer (NSCLC), melanoma, colorectal cancer, ovarian cancer or breast cancer to identify those patients who may benefit from treatment with one of 17 on-label targeted therapies, including 12 therapies currently approved as first-line therapy for their respective indications. FoundationOne CDx also reports genomic biomarkers, such as microsatellite instability (MSI) and tumor mutational burden (TMB), that can help inform the use of immunotherapies; genomic alterations in other genes relevant to patient management; and, relevant clinical trial information.

Based on previous CGP testing conducted by Foundation Medicine, it is estimated that approximately 1 in 3 patients across five common advanced cancers are expected to match with an FDA approved therapy.1 The number of matched on-label therapies indicated on FoundationOne CDx is expected to increase over time as Foundation Medicine and its biopharma partners pursue FDA approval for additional companion diagnostics on the platform. Today, approximately 50% of new cancer drugs in development are projected to have a companion biomarker.2

Concurrent with FDA approval, the Centers for Medicare and Medicaid Services (CMS) issued a preliminary National Coverage Determination (NCD) for FoundationOne CDx. The draft NCD would provide coverage for FDA-approved companion diagnostic claims, as well as a pathway for additional coverage with evidence development in other solid tumor types. The final policy is expected to issue during the first quarter of 2018 following public comment of the preliminary NCD and an administrative period.

"Today we know that many people with cancer do not receive biomarker testing, let alone the comprehensive genomic testing they need to be efficiently matched to the best therapeutic option," said Andrea Ferris, President and CEO of LUNGevity Foundation. "This FDA approval means that, in one test, patients can access therapies where companion diagnostics have been established for their cancer while getting a broad tumor profile that can identify the therapies and clinical trials they could most benefit from. Along with the preliminary national coverage determination, this has the potential to democratize next-generation sequencing, lowering the barriers for patients treated in the community to access these biomarker-driven treatments."

"Comprehensive genomic profiling is the gateway to precision medicine. This decision from the FDA and CMS, which may lead to coverage for Medicare patients, represents an important step forward in improving patient and clinician access to precision medicine – both in setting a new quality standard for this type of testing and offering potentially improved healthcare coverage," said Ankur R. Parikh, DO, Medical Director of Precision Medicine, Cancer Treatment Centers of America. "Access to important genomic information is a critical step in being able to offer innovative and targeted treatment options."

FoundationOne CDx results are delivered in an integrated report that identifies alterations matched to FDA approved therapies, identifies additional alterations in genes known to drive cancer growth, furnishes information about genomic biomarkers, including MSI and TMB, provides relevant clinical trial information, and includes interpretive content developed in accordance with professional guidelines in oncology for patients with any solid tumor.

"Today’s historic parallel review decision from the FDA and CMS represents a major advancement in personalized cancer care," said Troy Cox, chief executive officer at Foundation Medicine. "Physicians will have an FDA-approved test for all solid tumors in their toolkit that can inform targeted and immunotherapy selection, as well as identify patient opportunities for clinical trial participation. Beyond its implications for patient care, we expect that FoundationOne CDx will provide biopharma companies with an FDA-approved platform that can help accelerate drug development and enable personalized oncology care. On behalf of the Foundation Medicine team, I’d like to thank FDA and CMS for their leadership and collaboration as we continue to work through the parallel review process with a shared mission of transforming cancer care."

FoundationOne CDx is the first solid tumor comprehensive genomic profiling test reviewed by the FDA and CMS in their Parallel Review program. FDA approval was based on analytic validation and concordance studies with FDA-approved assays. FoundationOne CDx is expected to be commercially available following finalization of the NCD from CMS.

Conference Call and Webcast Details
Foundation Medicine will conduct a conference call to discuss FDA approval and the draft national coverage determination from CMS for FoundationOne CDx on Friday, December 1 at 8:30 a.m. ET. To access the conference call via phone, dial 1-877-270-2148 from the United States and Canada, or dial 1-412-902-6510 internationally, and for either number reference Foundation Medicine. Dial in approximately ten minutes prior to the start of the call. The live, listen-only webcast of the conference call may be accessed by visiting the investors section of the company’s website at investors.foundationmedicine.com. A replay of the webcast will be available shortly after the conclusion of the call and will be archived on the company’s website for two weeks following the call.

About FoundationOne CDx
FoundationOne CDx is a next generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed paraffin embedded (FFPE) tumor tissue specimens. The test is intended as a companion diagnostic to identify patients who may benefit from treatment with the targeted therapies listed below in accordance with the approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. The FoundationOne CDx assay is a single-site assay performed at Foundation Medicine, Inc.

EGFR exon 19 deletions and EGFR exon 21 L858R alterations, which may indicate efficacy of erlotinib, afatinib or gefitinib in patients with NSCLC.
EGFR exon 20 T790M alterations which may indicate efficacy of osimertinib in NSCLC patients
ALK rearrangements which may indicate efficacy of crizotinib, alectinib or certinib in NSCLC patients
BRAF V600E which may indicate efficacy of dabrafenib in combination with trametinib in NSCLC patients
BRAF V600E which may indicate efficacy of vemurafenib or dabrafenib in melanoma patients
BRAF V600E and V600K which may indicate efficacy of trametinib or cobimetinib in combination with vemurafenib in melanoma patients
ERBB2 (HER2) amplification which may indicate efficacy of trastuzumab, pertuzumab orado-trastuzumab-emtansine in patients with breast cancer
KRAS wild-type (absence of mutations in codons 12 and 13) which may indicate efficacy of cetuximab in patients with colorectal cancer
KRAS wild-type (absence of mutations in exons 2, 3 and 4) and NRAS wild-type (absence of mutations in exons 2, 3 and 4) which may indicate efficacy of panitumumab in patients with colorectal cancer
BRCA1/2 alterations which may indicate efficacy of rucaparib in patients with ovarian cancer
For more information about FoundationOne CDx, visit View Source

On November 30, 2017 MorphoSys Increases Financial Guidance for 2017 Following Signature of Regional License Agreement for Antibody MOR202 with I-Mab (Press release, MorphoSys, NOV 30, 2017, View Source [SID1234556341])

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MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) and I-Mab announced today that they have entered into an exclusive regional licensing agreement to develop and commercialize MOR202 in China, Taiwan, Hong Kong and Macao. MOR202 is MorphoSys’s proprietary investigational antibody against CD38, for which recruitment of a European Phase 1/2a clinical study in relapsed/refractory multiple myeloma has been concluded.

Under the terms of the agreement, I-Mab Biopharma (a fully owned affiliate of I-Mab) will assume exclusive responsibility for all subsequent development and commercialization of MOR202 in the agreed territory. MorphoSys receives an immediate upfront payment of USD 20 million. MorphoSys will be entitled to receive additional success-based clinical and commercial milestone payments from I-Mab of up to approximately USD 100 million, as well as tiered, double-digit royalties on net sales of MOR202 in the territory.

In connection with the license agreement with I-Mab, MorphoSys has increased its financial guidance. For the year 2017, MorphoSys now expects revenues in the range from EUR 63 to 66 million (up from previously EUR 46 to 51 million) and earnings before interest and taxes (EBIT) of EUR -66 to -71 million (up from previously EUR -75 to -85 million). Guidance for revenues and EBIT includes royalty income on Tremfya(R) sales in Q3 2017, but does not include any royalty income on Tremfya(R) sales in Q4 2017. Following the partnering of MOR202, proprietary R&D expenses will be in the range from EUR 96 to 100 million (previously EUR 85 to 95 million).

On November 30, 2017 Eleven Biotherapeutics, Inc. (NASDAQ:EBIO), a late-stage clinical oncology company advancing novel product candidates based on its Targeted Protein Therapeutics (TPTs) platform, reported that Stephen Hurly, President and Chief Executive Officer, will present a company overview at the LD Micro Main Event 2017 in Los Angeles, CA on Thursday, December 7, 2017 at 8:30 a.m. PST (Press release, Eleven Biotherapeutics, NOV 30, 2017, View Source [SID1234522322]).

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A live webcast can be accessed from the Investors & Media section of Eleven’s website, www.elevenbio.com. An archived replay of the webcast will be available on the Company’s website for 90 days after the conference.