On January 22, 2018 Nymox Pharmaceutical Corporation (NASDAQ:NYMX) reported today top-line 5-year results from Nymox’s U.S. Study NX03-0040. Study NX03-0040 was undertaken starting in 2012 at 44 investigational sites across the U.S. comprising a highly representative sample of 146 men with the biopsy confirmed diagnosis of T1c prostate cancer, which is the most common type of low grade localized prostate cancer (Press release, Nymox, JAN 22, 2018, View Source [SID1234523418]). After 5 years, the study has now shown that high dose Fexapotide 15mg single dosage treatment resulted in 80% less surgery or radiotherapy associated with Gleason grade progression (p=.0003), and that both doses of Fexapotide (15mg and 2.5mg) were consistently effective (p=.0003). There were 4.4% patients in the entire Fexapotide group who showed increase in their Gleason primary pattern grade in the 5-year study, compared to controls where the incidence of grade 4 or higher primary pattern was 23.5%, a reduction of 81.3% (p=.0061).

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Paul Averback MD, CEO of Nymox, said, "These major new results show the beneficial long-term effect of a single injection of Fexapotide Triflutate. The results are expected to be even better with regimens of additional or multiple treatment administrations if required."

In the studies Fexapotide triflutate was administered by a single painless injection directly into the prostate requiring several minutes or less in an office procedure guided by routine ultrasound. The drug was injected into the area of the prostate where the cancer was previously detected prior to enrollment in NX03-0040; and repeated biopsies every 18 months, serial PSA measurements and long-term follow-up were performed on all consenting treated patients and controls. After 5 years of study, high dose Fexapotide 15mg single treatment resulted in 80% less surgery or radiotherapy associated with Gleason grade progression (p=.0003), and both doses of Fexapotide (15mg and 2.5mg) were consistently effective (p=.0003). There were 4.4% patients in the entire Fexapotide group who showed increase in their Gleason primary pattern grade in the 5-year study, compared to controls where the incidence of grade 4 or higher primary pattern was 23.5%, a reduction of 81.3% (p=.0061). The new study results also indicated that after 5 years of study, all recorded instances of surgery or radiotherapy, including elective cases without Gleason upgrades, were decreased by 69.8% (p=.0002) in Fexapotide 15mg treated patients compared to the randomized control group.

Dr. Averback added, "Eight years of other related U.S. long-term Phase 3 BPH studies of Fexapotide have shown reduction in new prostate cancer incidence to 1.2%, compared to previous large BPH studies of earlier drugs where the incidence of prostate cancer is in the 10-20% range. There are therefore 2 different long-term Fexapotide programs which have now each independently shown that Fexapotide has a significant and highly beneficial effect for men with prostate cancer."

"These strong results clearly support Management’s ongoing efforts to advance both of the Company’s 2 major projects towards marketing goals. Nymox expects to report further on its U.S. development plans for registration trials for low grade prostate cancer. There is a global unmet medical need for more effective prostate treatments without the undesirable side effects of current treatments," he said.

One of the major problems with the main current prostate treatments for localized prostate cancer (radical prostatectomy, external beam radiation, brachytherapy) is the relatively high incidence of serious sexual problems post-treatment. In 9 studies, Fexapotide treatment has been shown to have a negligible significant adverse effect post-treatment on sexual function or testosterone levels.

Prostate cancer is the most commonly diagnosed cancer in men, other than skin cancer, and is the second leading cause of cancer death for men. Approximately 50% of prostate cancers are initially considered low risk.

Fexapotide has shown significant long-term benefit for prostate enlargement (benign prostatic hyperplasia, BPH). The recent results of Phase 3 studies of Fexapotide for BPH were communicated in podium and symposium presentations to the American Urological Association at four sectional Annual Meetings in 2017 in Scottsdale (North Central AUA November 15, 2017), Havana, (New York AUA November 6, 2017), Naples (South Central AUA November 27, 2017), and Savannah (Northeastern AUA October 12, 2017). The Company has filed for approval for Fexapotide in Europe for BPH for prostate enlargement in 2017, and the filing was validated in September 2017.

For more information please contact [email protected] or 800-936-9669.

On January 21, 2018 Laminar Pharma, a pioneering clinical stage biopharmaceutical company developing a new generation of products modulating metabolism of membrane lipids based on the groundbreaking MLT platform, reported that the European Commission has awarded a 6,15M€ grant to the CLINGLIO consortium, lead by Laminar Pharma, to execute the project entitled "A Clinical Phase IIB trial with 2OHOA in patients with newly-diagnosed malignant glioma" (Press release, Laminar Pharma, JAN 21, 2018, View Source [SID1234562095]).

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The CLINGLIO project was evaluated as a Research and Innovation Action (RIA) within the call H2020-SC1-2017-Two-Stage-RTD, Topic SC1-PM-08-2017 (New therapies for rare diseases), part of the Health, Demographic Change and Well-being Work Programme of the H2020. Total budget available for this H2020-SC1-2017-Two-Stage-RTD call was 173M€, of which 65M€ were assigned to topic SC1-PM-08-2017. Overall, 668 proposals were submitted to this call, of which 37 were pre-selected for funding across the four topics of the call (5,5% of the initial proposals). In the New Therapies for Rare Diseases topic, the EC will select for funding up to 11 proposals with an average budget per project of around 6M€.

The grant has been awarded to a multinational, well balanced consortium formed by 5 leading clinical research institutions in the UK (Royal Marsden Hospital and Northern Institute for Cancer Research, University ofNewcastle upon Tyne), France (Institut Gustave Roussy), Italy (Istituto Neurologico Carlo Besta) and Israel (Hadassah Medical Organization), two universities in Spain (Universitat de les Illes Balears, UIB) and Italy (Universita degli Studi di Salerno) and 4 specialized SMEs from The Netherlands (SMS Oncology, clinical CRO), Hungary (Lipodom Kft, lipidomic analysis), USA (LMBRI, pharmacoeconomics and market access) and Spain (Praxis Pharmaceutical, Drug product manufacturing and commercialization, and Laminar Pharma, coordinator and sponsor of the clinical trial).

The main objective of the CLINGLIO project is to execute a randomised, double-blind, placebo-controlled adjuvant trial in primary newly diagnosed glioblastoma patients to assess the efficacy and safety of 2OHOA in combination with radiotherapy and temozolomide. This study is a phase IIB adaptive trial with interim dose selection, sample size reassessment and biomarker threshold/omics signature determination. It is anticipated that around 15 clinical research hospitals across Europe and Israel will recruit 150 patients in the first part of the study, distributed in three arms: 1) control, with Standard of Care (SoC) plus placebo, 2) SoC plus 2OHOA "low dose" and 3) SoC plus 2OHOA "high dose". An interim analysis will take place after 75 events (Disease Progression) occurs and depending on the results of this interim analysis 60 to 120 additional patients will be enrolled in the second part of the study. The primary endpoint will be Progression Free Survival, according to RANO criteria, and Overall Survival will be a key secondary endpoint.

If the results of this clinical trial are positive, Laminar Pharma plans to apply for a conditional approval of 2OHOA in Europe for the treatment of newly diagnosed GBM patients, in combination with radiotherapy and temozolomide

On January 21, 2018 SymBio Pharmaceuticals Limited (Headquarters: Tokyo, "SymBio") (JASDAQ: 4582) reported that it has initiated a Phase 1 study in Japan for oral TREAKISYM in patients with progressive solid tumors (Press release, SymBio Pharmaceuticals, JAN 21, 2018, View Source [SID1234523465]).

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SymBio holds approval for TREAKISYM injectables which are already used for the treatment of three indications of malignant lymphoma (first-line and relapsed/refractory low-grade B-cell non-Hodgkin’s lymphoma and mantle cell lymphoma, and chronic lymphocytic leukemia). The purpose of the Phase 1 study is to evaluate the recommended dose, dosage regimen, the tolerability1 and the safety of oral TREAKISYM, as it is a new formulation, and to identify types of solid tumors that show promise for treatment.

Based on the efficacy and safety data related to TREAKISYM injectables that were demonstrated in the treatment of malignant lymphoma, the purpose of this study is also to provide a new treatment option for patients by developing the new oral formulation leveraging superior traits and fewer adverse events, including alopecia, compared with existing chemo therapy. Furthermore, SymBio will evaluate safer dosage regimes with no adverse effect on efficacy by leveraging the pharmacokinetic traits of the oral formulation, specifically, lowering Cmax and administration in lower doses during the treatment period. Oral formulation drugs can also be taken at home, eliminating the need for the patient to visit the hospital for intravenous infusion and reducing the treatment burden on the patient.

The development of oral TREAKISYM is part of SymBio’s strategy to develop a "TREAKISYM platform." For TREAKISYM injectables, the Phase III study for the indication of relapsed/refractory diffuse large B-cell lymphoma is underway. Although DLBCL accounts for the largest segment of malignant lymphoma in terms of patient numbers, currently only multiple drug therapies are available for r/r DLBCL. In addition, SymBio is deploying a sustainable growth strategy and will maximize the value of TREAKISYM by significantly extending the product life through the development of TREAKISYM liquid formulations (TREAKISYM Ready-to-dilute and TREAKISYM Rapid Infusion).2

1. Tolerability refers to the degree to which overt adverse effects of a drug can be tolerated by a human subject.
2. Please see SymBio’s press release of September 21, 2017: "Eagle Pharmaceuticals Licenses Japanese Rights for Bendamustine Hydrochloride Ready-to-dilute and Rapid Infusion Injection Products to SymBio Pharmaceuticals Limited."

About TREAKISYM

TREAKISYM (non-proprietary name: bendamustine hydrochloride), a cytocide anti-cancer drug first used in Germany in the 1970s, is now widely used in more than 50 countries with indications for low-grade non-Hodgkin’s lymphoma, mantle cell lymphoma, and chronic lymphocytic leukemia.

Bendamustine is a unique compound having chemical properties of both an alkylating agent3 and a metabolic antagonist4, and a mode of action different from other anti-cancer drugs. It is expected that bendamustine, given its unique properties, could be effective for the treatment of solid tumors as well as malignant lymphoma. A number of clinical studies of bendamustine injectables have been conducted outside of Japan to explore this potential, with clinical efficacy reported for certain solid tumors, including breast cancer, small-cell lung cancer, and soft tissue sarcoma. Furthermore, clinical studies of oral bendamustine for multiple myeloma, low-grade non-Hodgkin’s lymphoma, and chronic lymphocytic leukemia have indicated favorable results with respect to both safety and tolerability3 of oral formulation.

TREAKISYM Intravenous Infusion 100 mg was approved in October, 2010 for manufacturing and marketing for the indication of relapsed/refractory low-grade B-cell non-Hodgkin’s lymphoma and mantle cell lymphoma in Japan.
TREAKISYM was approved for the additional indication of chronic lymphocytic leukemia in Japan in August, 2016.
TREAKISYM Intravenous Infusion 25 mg, a standard low-dose product, was approved for manufacturing and marketing in Japan in September, 2016.
TREAKISYM was approved for the additional indication of first-line treatment of low-grade B-cell non-Hodgkin’s lymphoma and mantle cell lymphoma in Japan in December, 2016.
TREAKISYM has been marketed through Eisai Co., Ltd. since December, 2010.

3. An alkylating agent is a type of cytotoxic anti-cancer drug. Alkylating agents inhibit DNA replication by attaching alkyl group sites to the DNA chain.
4. A metabolic antagonist is a type of cytotoxic anti-cancer drug. Metabolic antagonists prevent DNA replication and the growth and division of tumor cells by interfering with the utilization of substances produced in the metabolic process.

On January 20, 2018 Array BioPharma Inc. (Nasdaq: ARRY) and Pierre Fabre reported updated results from the 30 patient safety lead-in of the Phase 3 BEACON CRC trial evaluating the triplet combination of encorafenib, a BRAF inhibitor, binimetinib, a MEK inhibitor and cetuximab, an anti-EGFR antibody, in patients with BRAF-mutant metastatic colorectal cancer (CRC) whose disease has progressed after one or two prior regimens (Press release, Array BioPharma, JAN 20, 2018, View Source;p=RssLanding&cat=news&id=2327568 [SID1234523363]). The data were presented at the ASCO (Free ASCO Whitepaper) 2018 Gastrointestinal Cancers Symposium in San Francisco, California.

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In patients with the BRAFV600E mutation, the estimated median progression-free survival (mPFS) at the time of analysis was 8 months. The confirmed overall response rate (ORR)* in patients with the BRAFV600E mutation was 48%, and 3 patients achieved complete responses (CR). Further, the ORR was 62% in the 16 patients (10/16) who received only one prior line of therapy. These data represent substantial improvements compared to several separate historical published standard of care benchmarks for this population.

"We are very excited about these safety lead-in results, which show both an unprecedented progression-free survival and overall response rate in patients with BRAFV600-mutant colorectal cancer," said Scott Kopetz, M.D., Ph.D., FACP, Associate Professor, Department of Gastrointestinal Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center. "To put these data in context, the observed median progression-free survival of 8 months exceeds historical benchmarks of approximately 2 months for median progression-free survival, and 4 to 6 months for median overall survival, with current standards of care in this patient population. These results demonstrate the potential of the triplet combination to benefit this population of patients who currently have very limited effective treatment options."

In the safety lead-in, the triplet combination was generally well-tolerated. Two patients discontinued treatment due to adverse events (AEs) with only one of these considered related to treatment. The most common grade 3 or 4 AEs seen in at least 10% of patients were fatigue (4/30), urinary tract infection (3/30), increased aspartate aminotransferase (AST; 3/30) and increased blood creatine kinase (CK; 3/30).

All patients with elevated baseline levels of the tumor markers CEA and CA19-9 had a reduction from baseline, with similar and substantial (median 83% – 96%) reductions across both markers in patients with objective responses and those with stable disease.

The enrollment in the randomized portion of the BEACON CRC trial is ongoing. Patients interested in participating in this trial may talk to their doctor to have their tumor tested for the BRAF mutation for eligibility to enroll in this new and important trial. Further details on the trial are available at clinicaltrials.gov (NCT02928224).

A PDF of the ASCO (Free ASCO Whitepaper) 2018 Gastrointestinal Cancers Symposium presentation can be found on Array’s website: View Source

*Overall response rate (ORR) = Complete response (CR) + Partial response (PR)

About BEACON CRC
BEACON CRC is a randomized, open-label, global trial evaluating the efficacy and safety of encorafenib, binimetinib and cetuximab in patients with BRAF-mutant metastatic CRC whose disease has progressed after one or two prior regimens. Thirty patients were treated in the safety lead-in and received the triplet combination (encorafenib 300 mg daily, binimetinib 45 mg twice daily and cetuximab per label). Of the 30 patients, 29 had a BRAFV600E mutation. Microsatellite instability-high (MSI-H), resulting from defective DNA mismatch repair, was detected in only 1 patient. As previously announced, the triplet combination demonstrated good tolerability, supporting initiation of the randomized portion of the trial.

The randomized portion of the BEACON CRC trial is designed to assess the efficacy of encorafenib in combination with cetuximab with or without binimetinib compared to cetuximab and irinotecan-based therapy. Approximately 615 patients are expected to be randomized 1:1:1 to receive triplet combination, doublet combination (encorafenib and cetuximab) or the control arm (irinotecan-based therapy and cetuximab). The primary endpoint of the trial is overall survival of the triplet combination compared to the control arm. Secondary endpoints address efficacy of the doublet combination compared to the control arm, and the triplet combination compared to the doublet therapy. Other secondary endpoints include PFS, ORR, duration of response, safety and tolerability. Health related quality of life data will also be assessed. The trial will be conducted at over 250 investigational sites in North America, South America, Europe and the Asia Pacific region. Patient enrollment is expected to be completed in 2018.

BEACON CRC is the first and only Phase 3 trial designed to test a BRAF/MEK combo targeted therapy in BRAF-mutant advanced CRC. Phase 2 trial results were presented at the 2016 ASCO (Free ASCO Whitepaper) annual meeting. [1] In the doublet arm of encorafenib and cetuximab, median overall survival (mOS) exceeded one year, which is more than double several separate historical published standard of care benchmarks for this population. [1-7] Further, the ORR was 22% and the mPFS was 4.2 months. [1] Historical published ORR and mPFS benchmarks in this patient population using standard of care regimens range between 4% to 8% and 1.8 and 2.5 months, respectively. [5-8]

About Colorectal Cancer
Worldwide, colorectal cancer is the third most common type of cancer in men and the second most common in women, with approximately 1.4 million new diagnoses in 2012. Of these, nearly 750,000 were diagnosed in men, and 614,000 in women. Globally in 2012, approximately 694,000 deaths were attributed to colorectal cancer. In the U.S. alone, an estimated 140,250 patients will be diagnosed with cancer of the colon or rectum in 2018, and approximately 50,000 are estimated to die of their disease. [9] In the U.S., BRAF mutations are estimated to occur in 10% to 15% of patients with colorectal cancer and represent a poor prognosis for these patients. [3, 4, 10, 11] Based on recent prospective historical data, the prevalence of MSI-H in tumors from patients with metastatic BRAF-mutant CRC ranged from 14% in a recent Phase 1b/2 trial (NCT01719380) (Array, data on file) to 18% in a recent Southwestern Oncology Group (SWOG) randomized phase 2 trial. [7]

About Encorafenib and Binimetinib
BRAF and MEK are key protein kinases in the MAPK signaling pathway (RAS-RAF-MEK-ERK). Research has shown this pathway regulates several key cellular activities including proliferation, differentiation, survival and angiogenesis. Inappropriate activation of proteins in this pathway has been shown to occur in many cancers including melanoma and colorectal cancer. Encorafenib is a late-stage small molecule BRAF inhibitor and binimetinib is a late-stage small molecule MEK inhibitor, both of which target key enzymes in this pathway. Encorafenib and binimetinib are being studied in clinical trials in advanced cancer patients, including the Phase 3 BEACON CRC trial and the Phase 3 COLUMBUS trial.

The U.S. Food and Drug Administration (FDA) is currently reviewing the New Drug Applications (NDAs) to support use of the combination of encorafenib and binimetinib for the treatment of patients with BRAF-mutant advanced, unresectable or metastatic melanoma. The FDA set a target action date under the Prescription Drug User Fee Act (PDUFA) of June 30, 2018 for both applications. In addition, the European Medicines Agency (EMA) is reviewing the Marketing Authorization Applications for encorafenib and binimetinib.

Encorafenib and binimetinib are investigational medicines and are not currently approved in any country.

Array BioPharma has exclusive rights to encorafenib and binimetinib in the U.S. and Canada. Array has granted Ono Pharmaceutical exclusive rights to commercialize both products in Japan and South Korea and Pierre Fabre exclusive rights to commercialize both products in all other countries, including Europe, Asia and Latin America. The BEACON CRC trial is being conducted with support from Pierre Fabre and Merck KGaA, Darmstadt, Germany (support is for sites outside of North America).

On January 20, 2017 BiVictriX Therapeutics ("BVX") has reported the completion of a successful fundraise to develop a new class of targeted cancer drugs (Press release, BiVictriX Therapeutics, JAN 20, 2018, View Source [SID1234526189]). The company’s ‘Dual Targeting Approach’ is designed to give superior selectivity towards blood cancers.

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Approximately £0.5m has been raised from a syndicate comprising Alderley Park Ventures, Finance Wales and high net worth individuals;

Funds to be used to establish Proof of Principle on the Company’s technology in two lead programmes;

Acceleris Capital Ltd (Manchester UK) provided corporate finance advice to the Company.

Founded in 2016 by serial life sciences entrepreneur Dr Peter Jackson and BVX CEO Tiffany Thorn, the company is focused on developing and licensing novel Antibody Drug Conjugates ("ADCs"), which have been coined as the new ‘Magic Bullet’ therapeutics in the field of oncology. The Company has developed a novel strategy, ‘The Dual Targeting Approach’ that improves ADC selectivity towards cancer cells. The lead and back-up candidate are in the areas of Acute Myeloid Leukaemia ("AML") and a specific type of agressive Non-Hodgkin Lymphoma, respectively. These areas have been targeted due to a combination of unmet clinical need, market size and the specificity of the targeted antigens.

BVX will focus on developing a platform technology to produce next generation bispecific ADCs with superior tumour selectivity. By exploiting its novel therapeutic strategy, the ‘Dual Targeting Approach’, the Company expects to significantly reduce off-target side effects on healthy tissues. This approach differentiates BVX from mainstream ADC developers by improving selectivity and minimising side effects, which can be significant hurdles in the development of these drugs.

The new investors include Alderley Park Ventures and Finance Wales who both look to back innovative, high-technology companies. The principal use of funds will be to initiate early scientific research to demonstrate Proof of Principle for our approach ahead of establishing technical Proof of Concept and selection of a lead therapeutic candidate.

Commenting on the fundraise, BVX’s CEO Tiffany Thorn said, "We are delighted to close our fundraising with a very strong syndicate of investors who are able to support BVX as we commence operations and look to grow. The Company is now looking forward to demonstrating Proof of Principle for our approach over the next 12 months and have selected a strong panel of service providers to assist in our development now that we have the necessary funds to grow."

David Youngman, Corporate Finance Director at Acceleris Capital and Non-executive director of BVX, said:

"Acceleris Capital has been delighted to work with BVX to pull together the funding plan and syndicate to allow BVX to commence operations. We are very excited by the potential applications of the ‘Dual Targeting Approach’ in this exciting area of oncology therapeutics."

Finance Wales Investment Executive Carmine Circelli, said:

"This is an exciting company with strong growth potential. Tiffany and the team have an innovative proposition and we look forward to seeing them commercialize successfully. We’re also pleased to be working with an experienced team of fellow co-investors to support BVX’s expansion in Wales."

The DWF Corporate team in Manchester acted as legal advisers to the Company in the transaction.