On September 19, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported that it has completed the planned enrollment of 380 patients in the global Toca 5 pivotal Phase 3 trial approximately three months ahead of schedule (Press release, Tocagen, SEP 19, 2018, View Source;p=RssLanding&cat=news&id=2368044 [SID1234529590]). Toca 5 is a randomized, multi-center study evaluating the safety and efficacy of Toca 511 & Toca FC compared to standard of care in patients undergoing resection for recurrent high grade glioma (HGG). The principal investigator is Timothy Cloughesy, M.D., director of the University of California, Los Angeles Neuro-Oncology Program.

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"Achieving our enrollment goal ahead of schedule is a testament to the enthusiasm and dedication of our investigators and study coordinators as well as the participating patients, families and patient advocates," said Asha Das, M.D., senior vice president and chief medical officer of Tocagen. "Achieving this enrollment goal takes us one step closer towards a potentially transformative new treatment option for patients with brain cancer."

"Completing enrollment in Toca 5 is the latest example of our recent progress as we continue to execute against our goals," said Marty Duvall, chief executive officer of Tocagen. "This important milestone also triggers the next milestone payment of $2 million from ApolloBio, our licensor of Toca 511 & Toca FC within the greater China region."

On August 23rd, Tocagen announced the Toca 5 study would continue without modification after an Independent Data Monitoring Committee completed the first interim analysis. Tocagen estimates the second interim analysis in the first half of 2019 and the final planned safety and efficacy analyses by the end of 2019. More information about Toca 5 can be found on ClinicalTrials.gov using the clinical trial identifier NCT02414165.

About High Grade Glioblastoma

Recurrent HGG is among the most common and aggressive primary brain cancers and often strikes in the prime of life. The two most common forms of HGGs are glioblastoma and anaplastic astrocytoma. The total number of new diagnoses of HGG expected in 2018 is about 188,000 worldwide. Unfortunately, HGG recurs in most patients after frontline treatment, and standard of care treatment typically offers a median survival of only seven to nine months.

About Toca 511 & Toca FC

Tocagen’s lead product candidate is a two-part cancer-selective immunotherapy comprised of an investigational biologic, Toca 511 and an investigational small molecule, Toca FC. Toca 511 (vocimagene amiretrorepvec) is a retroviral replicating vector (RRV) that selectively infects cancer cells and delivers a gene for the enzyme, cytosine deaminase (CD). Through this targeted delivery, infected cancer cells carry the CD gene and produce CD. Toca FC is an orally administered, extended-release formulation of the prodrug, 5-fluorocytosine (5-FC), which is converted into an anti-cancer drug, 5-fluorouracil (5-FU), when it encounters CD. 5-FU kills cancer cells and immune-suppressive myeloid cells in the tumor microenvironment resulting in anti-cancer immune activation and subsequent tumor killing.

1stOncology™, the leading oncology drug development surveillance and analysis platform from BioSeeker Group, now fully supports the latest release of the GLOBOCAN database (September 2018) which includes estimates of the incidence, mortality and prevalence from 36 types of cancer and for all cancers combined in 185 countries of the world. The estimates are presented for 2018, separately for each sex and for the 18 traditional age-groups.

The online tool allows the production of tables and visual descriptions of the current cancer burden by country or region, and permits projections regarding future cancer burden over the next 20 years.

Log-in here to access GLOBOCAN 2018 Epidemiology Data in 1stOncology™ or, if you are not a current user, request a free demo here to learn more.

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On September 18, 2018 Bristol-Myers Squibb Company (NYSE: BMY) reported that the European Medicines Agency (EMA) has validated the Company’s type II variation application for Empliciti (elotuzumab) in combination with pomalidomide and low-dose dexamethasone (EPd) for the treatment of adult patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI), and have demonstrated disease progression on the last therapy (Press release, Bristol-Myers Squibb, SEP 18, 2018, View Source [SID1234529478]). Validation of the application confirms the submission is complete and begins the EMA’s centralized review process.

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"Given the need for new treatment options for patients with multiple myeloma, we look forward to working closely with the EMA as they review this application," said Fouad Namouni, M.D., head, oncology development, Bristol-Myers Squibb. "It is our hope that this new Empliciti-based combination will soon become available for patients in the European Union with multiple myeloma, whose disease progressed on lenalidomide and a PI."

The application is based on data from ELOQUENT-3, a randomized Phase 2 study evaluating the EPd combination versus pomalidomide and dexamethasone (Pd) alone in patients with relapsed or refractory multiple myeloma (RRMM). Data from this study were presented at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June.

Bristol-Myers Squibb and AbbVie are co-developing Empliciti, with Bristol-Myers Squibb solely responsible for commercial activities.

About ELOQUENT-3

The Phase 2 ELOQUENT-3 trial randomized 117 patients with RRMM who received two or more prior therapies and were either refractory or relapsed and refractory to lenalidomide and a PI. Patients were randomized 1:1 to receive either EPd (n=60) or Pd (n=57) in 28-day cycles until disease progression or unacceptable toxicity. Patients in both the EPd and Pd arms received 4 mg of pomalidomide for days 1-21 of each cycle, and the weekly equivalent of 40 mg or 20 mg dexamethasone for patients ≤75 years or >75 years, respectively. In the EPd arm, Empliciti was administered at the dose of 10 mg/kg IV weekly for the first 2 cycles and 20 mg/kg monthly starting from cycle 3. Patients randomized to EPd experienced a 46% reduction in risk of disease progression (HR 0.54; 95% CI: 0.34 to 0.86, p=0.0078) compared with patients randomized to Pd alone, with median PFS, the study’s primary endpoint, of 10.3 months (95% CI: 5.6 to not estimable) compared with 4.7 months (95% CI: 2.8 to 7.2) in Pd patients. The PFS benefit experienced among patients randomized to EPd was consistent among patients who had received two to three prior lines of therapy (HR 0.55; 95% CI: 0.31 to 0.98) and four or more prior lines of therapy (HR 0.51; CI 95%: 0.24 to 1.08).

Rates of treatment-related hematologic adverse events (AEs) were comparable between EPd and Pd groups (38% and 42%, respectively). The most commonly occurring hematologic AEs among patients receiving EPd were neutropenia (13%), anemia (10%), thrombocytopenia (8%) and lymphopenia (8%). AEs led to discontinuation in 18% of patients in the EPd arm, compared with 24% of patients in the Pd arm.

On September 18, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company developing highly selective medicines for patients with genomically defined cancers, reported that abstracts from its LOXO-292 and larotrectinib programs have been accepted for oral presentations at the 88th Annual Meeting of the American Thyroid Association to be held October 3-7, 2018, in Washington, DC (Press release, Loxo Oncology, SEP 18, 2018, View Source [SID1234529642]).

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The LOXO-292 oral presentation will provide an updated analysis of patients with RET mutant medullary thyroid cancer and RET fusion thyroid cancers enrolled in the dose escalation cohorts of the ongoing LIBRETTO-001 Phase 1/2 clinical trial. The larotrectinib oral presentation will provide an analysis of patients with TRK fusion thyroid cancer enrolled to the larotrectinib clinical program.

The schedule for the presentations is as follows:

LOXO-292 Oral Presentation Session Date & Time: October 6, 2018, 9:05 a.m.-9:20 a.m. ET
Title: Clinical Activity of LOXO-292, a Highly Selective RET Inhibitor, in Patients with RET-Altered Thyroid Cancers
Session Title: Clinical Short Call Oral
Presenter: Lori J. Wirth, M.D.

Larotrectinib Oral Presentation Session Date & Time: October 4, 2018, 1:50 p.m.-2:05 p.m. ET
Title: Activity of Larotrectinib in Patients with Advanced TRK Fusion Thyroid Cancer
Session Title: Thursday Clinical Oral Abstracts
Presenter: Marcia S. Brose, M.D., Ph.D.

About LOXO-292
LOXO-292 is an 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 and mutations occur across multiple tumor types with varying frequency. 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 has been granted Breakthrough Therapy Designation by the U.S. FDA.

LOXO-292 is currently being studied in the global LIBRETTO-001 Phase 1/2 trial. For additional information about the LOXO-292 clinical trial, please refer to www.clinicaltrials.gov. 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 RET-Altered Cancers
Genomic alterations in the RET kinase, which include fusions and activating point mutations, lead to overactive RET signaling and uncontrolled cell growth. RET fusions have been identified in approximately 2% of non-small cell lung cancer, 10-20% of papillary and other thyroid cancers, and a subset of other cancers. Activating RET point mutations account for approximately 60% of medullary thyroid cancer (MTC). Both RET fusion cancers and RET-mutant MTC 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.

About Larotrectinib
Larotrectinib is an oral and selective investigational tropomyosin receptor kinase (TRK) inhibitor 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 anti-tumor activity in patients with tumors harboring NTRK gene fusions, regardless of patient age or tumor type. In an analysis of 55 RECIST-evaluable adult and pediatric patients with NTRK gene fusions, using a July 17, 2017 data cutoff, larotrectinib demonstrated a 75 percent centrally-assessed confirmed overall response rate (ORR) and an 80 percent investigator-assessed confirmed ORR, across many different types of solid tumors. The majority (93 percent) of all adverse events were grade 1 or 2.

Larotrectinib has been granted Priority Review, 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 are jointly developing 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/trk-trials.

About TRK Fusion Cancer
TRK fusion cancer occurs when a neurotrophic tyrosine receptor kinase (NTRK) gene fuses with another unrelated gene, producing an altered tropomyosin receptor kinase (TRK) protein. The altered protein, or TRK fusion protein, is constantly active, triggering a permanent signal cascade. These proteins become the primary driver of the spread and growth of tumors in patients with TRK fusion cancer. TRK fusion cancer is not limited to certain types of cells or tissues and can occur in any part of the body. NTRK gene fusions occur in various adult and pediatric solid tumors with varying prevalence, including appendiceal cancer, breast cancer, cholangiocarcinoma, colorectal cancer, GIST, infantile fibrosarcoma, lung cancer, mammary analogue secretory carcinoma of the salivary gland, melanoma, pancreatic cancer, thyroid cancer, and various sarcomas. Only sensitive and specific tests can reliably detect TRK fusion cancer. Next-generation sequencing (NGS) can provide a comprehensive view of genomic alterations across a large number of genes. Fluorescence in situ hybridization (FISH) can also be used to test for TRK fusion cancer, and immunohistochemistry (IHC) can be used to detect the presence of TRK protein.

On SEptember 18, 2018 NewLink Genetics Corporation (NASDAQ:NLNK) reported that the Company will present at the 2018 Cantor Global Healthcare Conference on Wednesday, October 3, 2018, at 4:00PM ET in New York, New York (Press release, NewLink Genetics, SEP 18, 2018, View Source [SID1234530679]).

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A live webcast of the presentation will be available on the Company’s website at www.newlinkgenetics.com in the "Investors & Media" section under "Events and Presentations." An archived edition of the presentation will be available on NewLink Genetics’ website later that day.