On April 10, 2018 Loxo Oncology (Nasdaq:LOXO) and Illumina, Inc. (Nasdaq:ILMN) reported a global strategic partnership to develop and commercialize a multi-gene panel for broad tumor profiling, resulting in a distributable, next-generation sequencing (NGS) based companion diagnostic (CDx) with a pan-cancer indication (Press release, Loxo Oncology, APR 10, 2018, View Source [SID1234525372]). The co-development partnership will seek approval for a version of the Illumina TruSight Tumor 170 as a companion diagnostic (CDx) for Loxo Oncology’s larotrectinib, which targets NTRK gene fusions, and LOXO-292, which targets RET gene alterations, across tumor types.

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TruSight Tumor 170 is a comprehensive, state-of-the-art, next-generation sequencing test that interrogates point mutations, fusions, amplifications and splice variants in 170 genes associated with common solid tumors. The CDx version of TruSight Tumor 170 will allow local laboratories to provide referring physicians with comprehensive genomic information, so that patients can be matched to the most appropriate therapeutic options. This version of TruSight Tumor 170 will run on the NextSeq 550Dx platform.

"We are leveraging our leadership in next-generation sequencing to deliver in-vitro diagnostic solutions to improve the management of cancer patients in the clinic," said Garret Hampton, Ph.D., executive vice president of clinical genomics at Illumina. "To this end, we are partnering with leading biotechnology companies, such as Loxo Oncology, to develop companion diagnostics for best-in-class therapeutics. Distributable diagnostic solutions, such as a CDx version of TruSight Tumor 170, in combination with the NextSeq 550Dx platform, will enable labs to perform precision medicine testing in-house."

Under the partnership, the companies will collaborate to validate a CDx version of TruSight Tumor 170 for NTRK fusions and RET fusions/mutations as a Class III FDA-approved diagnostic in conjunction with larotrectinib and LOXO-292, respectively. The companies are also planning to broaden the clinical utility of the full panel by obtaining regulatory approval for the other assay content, to be marketed as a tumor profiling test. Illumina will lead regulatory activities related to the Class III plans for NTRK and RET, the Class II plans for the tumor profiling content, and CE marking.

"We are very excited to announce this collaboration with Illumina, the world’s leader in NGS technology," said Jacob Van Naarden, chief business officer of Loxo Oncology. "We have piloted numerous NGS assays, and the Illumina TruSight Tumor 170 assay has consistently demonstrated robust performance with its assessment of both DNA and RNA, including highly sensitive gene fusion detection. The broad 170-gene assay content has the potential to deliver meaningful insights from a single tumor specimen, identifying patients with NTRK fusions, RET fusions, RET mutations, and many other actionable tumor alterations. Furthermore, we believe that this collaboration will improve patient access to high-quality NGS testing because pathologists will be able to run TruSight Tumor 170 locally and receive reimbursement."

About TruSight Tumor 170
TruSight Tumor 170 currently serves as the foundation for a comprehensive research use oncology menu, including:
170 unique genes informed by partnering pharmaceutical companies, academic community leaders, and industry guidance enable broad tumor profiling
Integrated workflow allowing more comprehensive testing while preserving precious samples by evaluating DNA and RNA in one integrated protocol with only 40ng from FFPE samples
Underlying assay method to serve as a standard for oncology testing and will be deployed across a variety of applications including Immuno-Oncology and liquid biopsy

About Larotrectinib (LOXO-101)
Larotrectinib is a potent, oral and highly selective tropomyosin receptor kinase (TRK) inhibitor. The investigational new drug is in clinical development for the treatment of patients with cancers that harbor a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In clinical trials, larotrectinib demonstrated marked and durable anti-tumor activity in TRK fusion cancer regardless of patient age or tumor type. In an analysis of 55 RECIST-evaluable adult and pediatric patients with NTRK gene fusions, larotrectinib demonstrated an 80 percent investigator-assessed confirmed overall response rate (ORR) and a 75 percent centrally-assessed confirmed ORR, across many different types of solid tumors. Larotrectinib was well tolerated; the majority of all adverse events were grade 1 or 2. There were no treatment-related grade 4 or 5 events, and no treatment-related grade 3 adverse events occurred in more than 5% of patients.
Larotrectinib has been granted Breakthrough Therapy Designation, Rare Pediatric Disease Designation and Orphan Drug Designation by the U.S. FDA.

In November 2017, Loxo Oncology and Bayer entered into an exclusive global collaboration for the development and commercialization of larotrectinib and LOXO-195, a next-generation TRK inhibitor. Bayer and Loxo Oncology will jointly develop the two products with Loxo Oncology leading the ongoing clinical studies as well as the filing in the U.S., and Bayer leading ex-U.S. regulatory activities and worldwide commercial activities. In the U.S., Loxo Oncology and Bayer will co-promote the products.

For additional information about the larotrectinib clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123 or visit www.loxooncologytrials.com.

About LOXO-292
LOXO-292 is a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase. RET fusions have been identified in approximately 2% of non-small cell lung cancer, 10-20% of papillary thyroid cancer, and a subset of colon and other cancers. RET point mutations account for approximately 60% of medullary thyroid cancer. Both RET fusion and select RET mutated cancers are primarily dependent on this single activated kinase for their proliferation and survival. This dependency, often referred to as "oncogene addiction," renders such tumors highly susceptible to small molecule inhibitors targeting RET. LOXO-292 was designed to inhibit native RET signaling as well as anticipated acquired resistance mechanisms that could otherwise limit the activity of this therapeutic approach. LOXO-292 is currently being studied in a Phase 1 trial. Interested patients and physicians can contact the Loxo Oncology Physician and Patient RET Clinical Trial Hotline at 1-855-RET-4-292 or email [email protected].

About Illumina
Illumina is improving human health by unlocking the power of the genome. Our focus on innovation has established us as the global leader in DNA sequencing and array-based technologies, serving customers in the research, clinical, and applied markets. Our products are used for applications in the life sciences, oncology, reproductive health, agriculture, and other emerging segments. To learn more, visit www.illumina.com and follow @illumina.

On April 10, 2018 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the Company’s lead, oral Selective Inhibitor of Nuclear Export (SINE) compound selinexor for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy (Press release, Karyopharm, APR 10, 2018, View Source [SID1234525246]). The FDA’s statement, consistent with the design of Karyopharm’s Phase 2b STORM study, noted that the three prior lines of therapy include regimens comprised of an alkylating agent, a glucocorticoid, Velcade (bortezomib), Kyprolis (carfilzomib), Revlimid (lenalidomide), Pomalyst (pomalidomide) and Darzalex (daratumumab). In addition, the patient’s disease must be refractory to at least one proteasome inhibitor (Velcade or Kyprolis), one immunomodulatory agent (Revlimid or Pomalyst), glucocorticoids and to Darzalex, as well as to the most recent therapy. The Company expects to report top-line data from the STORM study at the end of April 2018.

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The FDA’s Fast Track program facilitates the development of drugs intended to treat serious conditions and that have the potential to address unmet medical needs. A drug program with Fast Track status is afforded greater access to the FDA for the purpose of expediting the drug’s development, review and potential approval. In addition, the Fast Track program allows for eligibility for Accelerated Approval and Priority Review, if relevant criteria are met, as well as for Rolling Review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be submitted for review.

"The designation of Fast Track for selinexor represents important recognition by the FDA of the potential of this anti-cancer agent to address the significant unmet need in the treatment of patients with penta-refractory myeloma that has continued to progress despite available therapies," said Sharon Shacham, PhD, MBA, Founder, President and Chief Scientific Officer of Karyopharm. "We are fully committed to working closely with the FDA as we continue development of this potential new, orally-administered treatment for patients who currently have no other treatment options of proven benefit."

About the Phase 2b STORM Study

In the multi-center, single-arm Phase 2b STORM (Selinexor Treatment of Refractory Myeloma) study, approximately 122 patients with heavily pretreated, penta-refractory myeloma receive 80mg oral selinexor twice weekly in combination with 20mg low-dose dexamethasone, also dosed orally twice weekly. Patients with penta-refractory disease are those who have previously received an alkylating agent, a glucocorticoid, two immunomodulatory drugs (IMiDs) (Revlimid (lenalidomide) and Pomalyst (pomalidomide)), two proteasome inhibitors (PIs) (Velcade (bortezomib) and Kyprolis (carfilzomib)), and the anti-CD38 monoclonal antibody Darzalex (daratumumab), and their disease is refractory to at least one PI, at least one IMiD, Darzalex, glucocorticoids and their most recent anti-myeloma therapy. Overall response rate is the primary endpoint of the study, with duration of response and clinical benefit rate being secondary endpoints. All responses will be adjudicated by an Independent Review Committee (IRC).

About Selinexor

Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. To date, over 2,300 patients have been treated with selinexor, and it is currently being evaluated in several mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade (bortezomib) and low-dose dexamethasone (BOSTON), in combination with low-dose dexamethasone (STORM) and as a potential backbone therapy in combination with approved therapies (STOMP), and in diffuse large B-cell lymphoma (SADAL), and liposarcoma (SEAL), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with one or more approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

On April 10, 2018 Oncolytics Biotech Inc. (TSX: ONC) (OTCQX: ONCYF), currently developing REOLYSIN (pelareorep), an intravenously delivered immuno-oncolytic virus creating an inflamed phenotype, reported that a poster highlighting the effectiveness of pelareorep in combination with Keytruda and/or an anti-CD73 immunotherapy in prostate cancer cell lines was presented at the 11th International Oncolytic Virus Conference (IOVC) (Press release, Oncolytics Biotech, OCT 10, 2018, View Source [SID1234525554]). The conference takes place at Oxford University, April 9-12, 2018, in Oxford, UK.

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"This poster adds to the critical mass of validating data that demonstrates the positive outcomes seen when using pelareorep in combination with other immuno-oncology drugs," said Dr. Matt Coffey, President and CEO of Oncolytics Biotech. "Prostate cancer is generally considered to be a ‘cold’ tumor, and this non-inflamed phenotype is thought to be largely responsible for the lack of sensitivity of these patients to immune checkpoint blockade. We continue to believe that the use of oncolytic viruses can overcome pre-existing mechanisms of resistance to immunotherapy in many cancers by transforming these ‘cold’ tumors into ‘hot,’ immune cell infiltrated tumors."

Data presented in the poster demonstrated that:

treatment of subcutaneous TRAMP-C2 prostate tumors with a combination of pelareorep and anti-PD-1 (Keytruda) or anti-CD73 antibody significantly enhanced survival of mice compared to pelareorep or antibody therapy alone;

immune profiling of pelareorep treated and untreated tumors confirmed the ability of pelareorep to increase tumour immune cell infiltration;

pelareorep infection of tumours is needed before a therapeutic effect of anti-immune inhibitory/suppressive antibodies is seen;

pelareorep-initiated antitumor immunity protects against subsequent tumour challenge; and

after the study of negative regulators, only B and T lymphocyte attenuator (BTLA) and PD-L1 were significantly upregulated in the pelareorep treated TRAMP-C2 tumors compared to untreated tumour.

The poster presentation by Dr. Guy Simpson, Department of Clinical and Experimental Medicine, University of Surrey, is now available on the Posters, Presentations & Publications page of the company’s website: www.oncolyticsbiotech.com/technology/posters-publications.

About REOLYSIN/Pelareorep
REOLYSIN, also known as pelareorep, is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers.

On April 10, 2018 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, reported that updated poziotinib Phase 2 data in MD Anderson’s EGFR Exon 20 Mutant Non-Small Cell Lung Cancer study are available, based on longer follow-up (Press release, Spectrum Pharmaceuticals, APR 10, 2018, View Source [SID1234525373]).

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"I am pleased to observe the preliminary confirmed objective response rate and potential progression-free survival (PFS) benefit in EGFR Exon 20 Mutant Non-Small Cell Lung Cancer patients," said John Heymach, M.D., Ph.D., Chairman and Professor, Department of Thoracic/Head and Neck Medical Oncology, The University of Texas MD Anderson Cancer Center. "In the first 11 patients, the confirmed objective response rate was 64%. This is very exciting because we were initially hoping to get response rates between 20% to 30%. I am encouraged to see that in these 11 patients, the median PFS has not been reached after a median follow up of 6.5 months. In addition, the two most common adverse events observed in the study to date are skin rash and diarrhea, which are known EGFR inhibitor-related toxicities. We are looking forward to presenting comprehensive data from this study at a major medical meeting later this year."

"The updated data from MD Anderson provides additional insight into just how meaningful poziotinib may be in this area of high unmet need," said Joe Turgeon, President and CEO of Spectrum Pharmaceuticals. "At each turn, the possibility of this drug as an option for EGFR Exon 20 mutant NSCLC patients is becoming more clear."

"Our study at MD Anderson has far exceeded our enrollment expectations," said Xiuning Le, M.D., Assistant Professor, Department of Thoracic/Head and Neck Medical Oncology, The University of Texas MD Anderson Cancer Center. "At this point, the original cohort of 30 EGFR patients is fully enrolled and the expanded cohort of 20 patients is nearing the completion of enrollment. As enrollment in our study nears completion, we will soon begin enrolling patients in Spectrum’s ongoing multicenter Phase 2 study."

"These early data from MD Anderson suggest poziotinib may have a meaningful impact on outcomes for patients who have limited treatment options," said David Chu, M.D., New York Cancer and Blood Specialists. "As one of the initial sites on the east coast for Spectrum’s ongoing multi-center Phase 2 study, we have patients seeking us out from around the world. I am excited about this potential option for these patients."

On April 10, 2018 VBL Therapeutics (Nasdaq:VBLT), a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class treatments for cancer, reported that the Company will present new data on MOSPD2, a novel target for Bi-specific Ab mediated killing of tumor cells, at the 2018 Annual American Association for Cancer Research (AACR) (Free AACR Whitepaper) Meeting, to be held April 14-18, 2018 at McCormick Place North/South in Chicago, Illinois (Press release, VBL Therapeutics, APR 10, 2018, View Source [SID1234525248]).

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Poster Presentation Details:

Late-Breaking Research: Immunology 1
Date: Monday, April 16, 2018
Time: 8:00 am – 12:00 noon
Session Location: Poster Section 45