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On August 17, 2015 BioLineRx Ltd. (NASDAQ: BLRX; TASE: BLRX), a clinical-stage biopharmaceutical company dedicated to identifying, in-licensing and developing promising therapeutic candidates, reported the initiation of a Phase 2b trial for BL-8040 as a novel consolidation treatment for acute myeloid leukemia (AML) (Filing, 6-K, BioLineRx, AUG 17, 2015, View Source [SID:1234507267]). The Phase 2b study will examine BL-8040 as part of a second stage treatment, termed consolidation therapy, to improve outcomes for AML patients who have achieved remission after the standard initial treatment regimen, known as induction therapy. The consolidation therapy is aimed at eliminating the minimal residual disease left in the bone marrow after induction therapy that can lead to relapse. This study is the first of three clinical studies in additional indications for BL-8040 which BioLineRx plans to commence during 2015, thus significantly expanding its unique BL-8040 oncology platform.

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Dr. Kinneret Savitsky, Chief Executive Officer of BioLineRx, stated, "The majority of high-risk AML patients achieving first complete remission relapse within one year, despite the current standard consolidation therapy. Patients with AML relapse have a poor prognosis despite further therapy, and less than 10% of these patients are cured by conventional therapy. Leukemic stem cells that are dormant in the bone marrow are presumed to be a major reason for AML relapse. Based on the pre-clinical and clinical data accumulated to date, BL-8040 is anticipated to boost the efficacy of consolidation therapy due to its dual mechanism of action. Firstly, BL-8040 induces mobilization of leukemic cells from the bone marrow, which enhances the cytotoxic effects of chemotherapy, and secondly, it possesses anti-leukemic pro-apoptotic properties that help eliminate AML cells directly. Based on positive results from our ongoing phase 2 clinical trial for BL-8040, which show substantial mobilization of AML cells from the bone marrow to the peripheral blood, as well as robust induction of AML cell apoptosis, we believe BL-8040 will be a promising addition to consolidation therapy for AML patients."

Dr. Savitsky added, "In addition to initiation of the Phase 2b AML consolidation study, we are eagerly looking forward to the top-line results from our ongoing Phase 2 study of BL-8040 for treating relapsed and refractory AML patients, which we expect in the fourth quarter of 2015. We also look forward to the next stages of development for BL-8040 as a novel stem cell mobilization treatment, after its recent successful completion of a Phase 1 trial. Finally, we are also excited about the anticipated initiation of clinical studies for BL-8040 in two additional indications over the next few months, thus further expanding and enhancing the potential of our oncology platform."

The Phase 2b trial, which is conducted in collaboration with the University of Halle as sponsor and with the participation of two large leukemia study groups in Germany, is a double-blind, placebo-controlled, randomized, multi-center study aimed at assessing the efficacy of BL-8040 in addition to standard consolidation therapy in AML patients. The primary endpoint of the study is to compare the relapse free survival (RFS) time in AML subjects in their first remission during a minimum follow-up time of 18 months after randomization. In addition, pharmacodynamic measurements will be conducted in order to assess the minimal residual disease, and biomarker analyses will be performed to identify predictors of BL-8040 response. The study will enroll up to 194 patients at up to 25 sites in Germany. AML patients between 18 and 75 years of age with documented first remission will be randomized in a 1:1 ratio to receive high dose Cytarabine, either with BL-8040 or with a matching placebo, as consolidation therapy.

About BL-8040
BL-8040 is a clinical-stage drug candidate for the treatment of acute myeloid leukemia, as well as other hematological indications. It is a short peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis (growth of new blood vessels in the tumor), metastasis (spread of the disease to other organs or organ parts) and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a Phase 1/2, open-label, dose escalation, safety and efficacy clinical trial in 18 multiple myeloma patients, BL-8040, when combined with G-CSF, demonstrated an excellent safety profile at all doses tested and was highly effective in the mobilization of hematopoietic stem cells and white blood cells from the bone marrow to the peripheral blood. Additionally, in a Phase 1 stem-cell mobilization study in healthy volunteers, BL-8040 as a single agent was safe and well tolerated at all doses tested and resulted in efficient stem-cell mobilization and collection in all study participants. Importantly, the results of this study support the use of BL-8040 as one-day, single-dose collection regimen, which is a significant improvement upon the current standard of care.

BL-8040 also mobilizes cancer cells from the bone marrow and may therefore sensitize these cells to chemo- and bio-based anti-cancer therapy. Importantly, BL-8040 has also demonstrated a direct anti-cancer effect by inducing apoptosis. Pre-clinical studies show that BL-8040 inhibits the growth of various tumor types including multiple myeloma, non-Hodgkin’s lymphoma, leukemia, non-small cell lung carcinoma, neuroblastoma and melanoma. BL-8040 significantly and preferentially stimulated apoptotic cell death of malignant cells (multiple myeloma, non-Hodgkin’s lymphoma and leukemia). Significant synergistic and/or additive tumor cell killing activity has been observed in-vitro and in-vivo when tumor cells were treated with BL-8040 together with Rituximab, Bortezomib, Imatinib, Cytarabine and the FLT-3 inhibitor AC-220 (in NHL, MM, CML, AML, and AML-FLT3-ITD models, respectively). In addition, the current Phase 2 clinical trial in AML patients has demonstrated robust mobilization and apoptosis of cancer cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Acute Myeloid Leukemia (AML)
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow and is the most common type of acute leukemia in adults. According to the American Cancer Society, approximately 19,000 new cases of AML were diagnosed in the United States in 2014, and the median age of AML patients was 67 years old. The first treatment line for patients with AML includes a combination of chemotherapy drugs and is called induction treatment. The median survival for AML patients receiving induction chemotherapy is less than two years, with shorter survival for patients over the age of 60 or for those with certain gene or chromosome aberrations. Due to relapsed or refractory disease (where the disease is not responsive to standard treatments), the overall five-year survival rate for AML is between 10 and 40 percent.

Seattle Genetics Announces FDA Regular Approval of ADCETRIS® (Brentuximab Vedotin) for Classical Hodgkin Lymphoma Patients at High Risk of Relapse or Progression as Post-Autologous Hematopoietic Stem Cell Transplantation Consolidation

On August 17, 2015 Seattle Genetics, Inc. (Nasdaq: SGEN) reported that the U.S. Food and Drug Administration (FDA) has approved ADCETRIS (brentuximab vedotin) for the treatment of patients with classical Hodgkin lymphoma (HL) at high risk of relapse or progression as post-autologous hematopoietic stem cell transplantation (auto-HSCT) consolidation (Press release, Seattle Genetics, AUG 17, 2015, View Source [SID:1234507285]).

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The approval is based on a phase 3 clinical trial called AETHERA that was designed to compare up to 16 cycles (approximately one year) of ADCETRIS therapy administered every three weeks following auto-HSCT to placebo. The primary endpoint was met with a significant improvement in median progression-free survival (PFS) of 42.9 months (95% CI: 30.4, 42.9) for patients who received ADCETRIS versus 24.1 months (95% CI: 11.5, not estimable) for patients who received placebo, an improvement of 18.8 months (hazard ratio=0.57 [95% CI: 0.40, 0.81]; p-value=0.001). In addition, data from the AETHERA trial converted the U.S. accelerated approval of the relapsed classical HL indication to regular approval. ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, which is expressed in classical HL and systemic anaplastic large cell lymphoma (sALCL), as well as other lymphoma subtypes. This is the third indication for ADCETRIS, which was granted accelerated FDA approval in August 2011 for two other indications: (1) treatment of Hodgkin lymphoma patients who fail autologous transplant or who fail at least two prior multi-agent chemotherapy regimens and are not autologous transplant candidates, and (2) treatment of systemic ALCL patients who fail at least one prior multi-agent chemotherapy regimen. The sALCL indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

This Smart News Release features multimedia. View the full release here: View Source

ADCETRIS Product Photo (Photo: Business Wire)
ADCETRIS Product Photo (Photo: Business Wire)

Globally, there are more than 65,000 cases of HL diagnosed each year. Although frontline combination chemotherapy can result in durable responses, up to 30 percent of these patients fail frontline treatment. The standard for these patients is salvage therapy, followed by auto-HSCT; approximately half of all HL patients who undergo an auto-HSCT experience subsequent disease relapse.

"With this FDA approval, ADCETRIS is the first and only consolidation treatment option available to high risk classical Hodgkin lymphoma patients who undergo a transplant to preserve their post-auto-HSCT remissions," said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. "This represents a meaningful advance for cancer patients and an important milestone for the ADCETRIS development program. Together with our three ongoing phase 3 trials and more than 30 additional clinical trials, this approval supports our goal to broadly establish ADCETRIS as the foundation of therapy for classical Hodgkin lymphoma and CD30-expressing malignancies."

"The FDA approval of brentuximab vedotin for post-autologous hematopoietic transplantation consolidation treatment in classical Hodgkin lymphoma patients with high risk of relapse or progression is a significant milestone for patients and physicians," said Craig Moskowitz, M.D., lead investigator on the trial and Clinical Director, Division of Hematologic Oncology, Memorial Sloan Kettering Cancer Center. "Approximately half of all Hodgkin lymphoma patients who undergo an autologous hematopoietic stem cell transplant will relapse, representing a significant need for additional treatment options to improve progression free survival."

AETHERA Phase 3 Trial Demonstrates Significant Progression-Free Survival Benefit with ADCETRIS as Post-Transplant Consolidation in Hodgkin Lymphoma

The positive results from the phase 3 AETHERA trial were published in The Lancet in March 2015 and presented at the 56th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2014. A total of 329 HL patients at risk of relapse or progression were enrolled, including 165 on the ADCETRIS arm and 164 on the placebo arm. Patients eligible for enrollment in the AETHERA trial must have had a history of primary refractory HL, have relapsed within one year from receiving frontline chemotherapy and/or have had disease outside of the lymph nodes at the time of pre-auto-HSCT relapse. Results included:

The trial achieved its primary endpoint and demonstrated a significant increase in PFS per independent review facility, with a hazard ratio of 0.57 (95% CI: 0.40, 0.81) and a p-value of 0.001. Median PFS was 43 months (95% CI: 30.4, 42.9) for patients who received ADCETRIS versus 24 months (95% CI: 11.5, not estimable) for patients who received placebo.

The most common adverse events (≥20 percent), of any grade and regardless of causality, in the ADCETRIS arm were neutropenia (78 percent), peripheral sensory neuropathy (56 percent), thrombocytopenia (41 percent), anemia (27 percent), upper respiratory tract infection (26 percent), fatigue (24 percent), peripheral motor neuropathy (23 percent), nausea (22 percent), cough (21 percent) and diarrhea (20 percent). The most common adverse events (≥20 percent), of any grade and regardless of causality, in the placebo arm were neutropenia (34 percent), upper respiratory tract infection (23 percent) and thrombocytopenia (20 percent). Sixty-seven percent of patients on the ADCETRIS arm experienced peripheral neuropathy. Of those patients, 85 percent had resolution (59 percent) or partial improvement (26 percent) in symptoms at the time of their last evaluation, with a median time to improvement of 23 weeks (range 0.1-138).

There are no post-marketing requirements as part of the U.S. approval. The AETHERA data have also been submitted to both the European Medicines Agency (EMA) and Health Canada requesting marketing authorization.

As part of a broad clinical development program, ADCETRIS is also being evaluated in three additional phase 3 clinical trials, referred to as ECHELON-1, ECHELON-2 and ALCANZA. Enrollment is expected to be completed in ECHELON-1 and ALCANZA during 2015 and in ECHELON-2 during 2016.

About ADCETRIS

ADCETRIS is being evaluated broadly in more than 30 ongoing clinical trials, including three additional phase 3 studies, in earlier lines of its approved classical HL and sALCL indications as well as in many additional types of CD30-positive malignancies, including cutaneous T-cell lymphoma, B-cell lymphomas and mature T-cell lymphomas.

ADCETRIS is an ADC comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics’ proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing tumor cells.

ADCETRIS for intravenous injection has received approval from the FDA for three indications: (1) regular approval for the treatment of patients with classical HL after failure of auto-HSCT or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not auto-HSCT candidates, (2) regular approval for the treatment of classical HL patients at high risk of relapse or progression as post-auto-HSCT consolidation, and (3) accelerated approval for the treatment of patients with sALCL after failure of at least one prior multi-agent chemotherapy regimen. The sALCL indication is approved under accelerated approval based on overall response rate. Continued approval for the sALCL indication may be contingent upon verification and description of clinical benefit in confirmatory trials. Health Canada granted ADCETRIS approval with conditions for relapsed or refractory HL and sALCL.

ADCETRIS was granted conditional marketing authorization by the European Commission in October 2012 for two indications: (1) for the treatment of adult patients with relapsed or refractory CD30-positive HL following ASCT, or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, and (2) the treatment of adult patients with relapsed or refractory sALCL. ADCETRIS has received marketing authorization by regulatory authorities in more than 55 countries. See important safety information below.

Seattle Genetics and Takeda are jointly developing ADCETRIS. Under the terms of the collaboration agreement, Seattle Genetics has U.S. and Canadian commercialization rights and Takeda has rights to commercialize ADCETRIS in the rest of the world. Seattle Genetics and Takeda are funding joint development costs for ADCETRIS on a 50:50 basis, except in Japan where Takeda is solely responsible for development costs.

ADCETRIS (brentuximab vedotin) U.S. Important Safety Information

BOXED WARNING

Progressive multifocal leukoencephalopathy (PML): JC virus infection resulting in PML and death can occur in patients receiving ADCETRIS (brentuximab vedotin).

Contraindication

ADCETRIS is contraindicated with concomitant bleomycin due to pulmonary toxicity (e.g., interstitial infiltration and/or inflammation).

Warnings and Precautions

Peripheral neuropathy: ADCETRIS treatment causes a peripheral neuropathy that is predominantly sensory. Cases of peripheral motor neuropathy have also been reported. ADCETRIS-induced peripheral neuropathy is cumulative. Monitor patients for symptoms of neuropathy, such as hypoesthesia, hyperesthesia, paresthesia, discomfort, a burning sensation, neuropathic pain or weakness and institute dose modifications accordingly.

Anaphylaxis and infusion reactions: Infusion-related reactions, including anaphylaxis, have occurred with ADCETRIS. Monitor patients during infusion. If an infusion-related reaction occurs, interrupt the infusion and institute appropriate medical management. If anaphylaxis occurs, immediately and permanently discontinue the infusion and administer appropriate medical therapy.

Hematologic toxicities: Prolonged (≥1 week) severe neutropenia and Grade 3 or 4 thrombocytopenia or anemia can occur with ADCETRIS. Febrile neutropenia has been reported with ADCETRIS. Monitor complete blood counts prior to each dose of ADCETRIS and consider more frequent monitoring for patients with Grade 3 or 4 neutropenia. Monitor patients for fever. If Grade 3 or 4 neutropenia develops, consider dose delays, reductions, discontinuation, or G-CSF prophylaxis with subsequent doses.

Serious infections and opportunistic infections: Infections such as pneumonia, bacteremia, and sepsis or septic shock (including fatal outcomes) have been reported in patients treated with ADCETRIS. Closely monitor patients during treatment for the emergence of possible bacterial, fungal or viral infections.

Tumor lysis syndrome: Closely monitor patients with rapidly proliferating tumor and high tumor burden.
Increased toxicity in the presence of severe renal impairment: The frequency of ≥Grade 3 adverse reactions and deaths was greater in patients with severe renal impairment compared to patients with normal renal function. Avoid the use of ADCETRIS in patients with severe renal impairment.

Increased toxicity in the presence of moderate or severe hepatic impairment: The frequency of ≥Grade 3 adverse reactions and deaths was greater in patients with moderate or severe hepatic impairment compared to patients with normal hepatic function. Avoid the use of ADCETRIS in patients with moderate or severe hepatic impairment.

Hepatotoxicity: Serious cases of hepatotoxicity, including fatal outcomes, have occurred with ADCETRIS. Cases were consistent with hepatocellular injury, including elevations of transaminases and/or bilirubin, and occurred after the first dose of ADCETRIS or rechallenge. Preexisting liver disease, elevated baseline liver enzymes, and concomitant medications may also increase the risk. Monitor liver enzymes and bilirubin. Patients experiencing new, worsening, or recurrent hepatotoxicity may require a delay, change in dose, or discontinuation of ADCETRIS.

Progressive multifocal leukoencephalopathy (PML): JC virus infection resulting in PML and death has been reported in ADCETRIS-treated patients. First onset of symptoms occurred at various times from initiation of ADCETRIS therapy, with some cases occurring within 3 months of initial exposure. In addition to ADCETRIS therapy, other possible contributory factors include prior therapies and underlying disease that may cause immunosuppression. Consider the diagnosis of PML in any patient presenting with new-onset signs and symptoms of central nervous system abnormalities. Hold ADCETRIS if PML is suspected and discontinue ADCETRIS if PML is confirmed.

Pulmonary Toxicity: Events of noninfectious pulmonary toxicity including pneumonitis, interstitial lung disease, and acute respiratory distress syndrome, some with fatal outcomes, have been reported. Monitor patients for signs and symptoms of pulmonary toxicity, including cough and dyspnea. In the event of new or worsening pulmonary symptoms, hold ADCETRIS dosing during evaluation and until symptomatic improvement.

Serious dermatologic reactions: Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN), including fatal outcomes, have been reported with ADCETRIS. If SJS or TEN occurs, discontinue ADCETRIS and administer appropriate medical therapy.
Embryo-fetal toxicity: Fetal harm can occur. Advise pregnant women of the potential hazard to the fetus.

Most Common Adverse Reactions:

ADCETRIS was studied as monotherapy in 160 patients with relapsed classical HL and sALCL in two uncontrolled single-arm trials. Across both trials, the most common adverse reactions (≥20%), regardless of causality, were neutropenia, peripheral sensory neuropathy, fatigue, nausea, anemia, upper respiratory tract infection, diarrhea, pyrexia, rash, thrombocytopenia, cough and vomiting.

ADCETRIS was studied in 329 patients with classical HL at high risk of relapse or progression post-auto-HSCT in a placebo-controlled randomized trial. The most common adverse reactions (≥20%) in the ADCETRIS-treatment arm (167 patients), regardless of causality, were neutropenia, peripheral sensory neuropathy, thrombocytopenia, anemia, upper respiratory tract infection, fatigue, peripheral motor neuropathy, nausea, cough, and diarrhea.

Drug Interactions:

Concomitant use of strong CYP3A4 inhibitors or inducers, or P-gp inhibitors, has the potential to affect the exposure to monomethyl auristatin E (MMAE).

Use in Specific Populations:

MMAE exposure and adverse reactions are increased in patients with moderate or severe hepatic impairment or severe renal impairment. Avoid use.

For additional Important Safety Information, including Boxed WARNING, please see the full Prescribing Information for ADCETRIS at View Source

About Classical Hodgkin Lymphoma

Lymphoma is a general term for a group of cancers that originate in the lymphatic system and is the most common type of blood cancer. There are two major categories of lymphoma: HL and non-Hodgkin lymphoma. Classical HL is distinguished from other lymphomas by the characteristic presence of CD30-positive Reed-Sternberg cells. The Reed-Sternberg cell generally expresses CD30.

According to the American Cancer Society, approximately 9,050 cases of HL will be diagnosed in the United States during 2015 and more than 1,150 will die from the disease.

Spectrum Pharmaceuticals Announces Agreement with FDA on the Special Protocol Assessment (SPA) for the Upcoming Apaziquone Phase 3 Trial in Patients with Non-Muscle Invasive Bladder Cancer (NMIBC)

On August 17, 2015 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations and a primary focus in Hematology and Oncology, reported that the company has reached agreement with the U.S. Food and Drug Administration (FDA) on the Special Protocol Assessment (SPA) of the planned Phase 3 clinical trial of its novel, potent pro-drug, apaziquone (Press release, Spectrum Pharmaceuticals, AUG 17, 2015, View Source [SID:1234507284]). This trial will further evaluate the intravesical use of apaziquone for the treatment of patients with non-muscle invasive bladder cancer (NMIBC) as one or two instillations, immediately following transurethral resection of bladder tumor (TURBT).

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"Spectrum’s agreement with the FDA on the SPA represents a significant milestone for bladder cancer patients," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "The learnings from previous Phase 3 studies and comments from the FDA have been incorporated in the new protocol to improve the chances of success. We look forward to initiating this trial and filing the apaziquone NDA by year-end. This NDA is based on data from the previously completed program that included two Phase 3 studies with a total of 1,615 patients. We believe there continues to be a significant unmet need for these patients, as no drugs have been approved in the U.S. for more than 40 years for the treatment of low-grade NMIBC. Due to the high rate of recurrence, the overall cost of the treatment of bladder cancer in the U.S. is a staggering $3.4 billion annually, most of which is related to direct treatment of the disease. We endeavor to bring this much needed therapy for patients and help reduce overall medical costs at the same time."

In accordance with the SPA, the Phase 3 trial will be a randomized, double-blind, placebo-controlled, multicenter trial that will enroll patients with Ta G1 or G2 NMIBC. The patients will be randomized to receive either one instillation of apaziquone, two instillations of apaziquone, or placebo. The primary endpoint is Time to Recurrence. Since apaziquone is known to be inactivated in presence of blood, the new protocol includes a 30-60 minute waiting period post-TURBT, before apaziquone instillation. Patients that receive two instillations of apaziquone, will receive the second dose approximately two weeks after surgery minimizing the potential for drug inactivation due to bleeding. Further, it is recommended that patients with significant post-operative bleeding not receive apaziquone.

Apaziquone is an anticancer pro-drug that is activated by bio-reductive enzymes that are over-expressed in bladder cancer cells, rendering it into a highly cytotoxic alkylating agent. Spectrum has conducted two multi-center, international Phase 3 trials of a single intravesical instillation of apaziquone (4 mg) into the bladder in the immediate post-operative period after surgical resection of low-grade NMIBC. Pooled data from the two studies (n=1,615) showed a statistically significant treatment effect for the primary study endpoint, 2-Year Recurrence Rates, in favor of apaziquone (p-value = 0.0218) and in a key secondary endpoint, Time to Recurrence (p-value = 0.0096).

About Bladder Cancer

According to the National Cancer Institute, bladder cancer is the fifth most common malignancy in the US with 74,000 new cases of bladder cancer expected in 2015, and currently over 500,000 patients living with the disease. Due to high recurrence rates, intensive surveillance strategies, and expensive annual treatment costs, bladder cancer has the highest per patient costs, and an overall cost estimated at around $3.4 billion. Non-muscle invasive bladder cancer (NMIBC) is a form of bladder cancer that is localized in the surface layers of the bladder and has not invaded or spread to the deeper muscle layer. Approximately 70% of all patients newly diagnosed with bladder cancer have NMIBC. Urologists treat the disease predominantly by transurethral resection of the bladder tumor(s) (TURBT); in the U.S., there are approximately 300,000 TURBT procedures every year to treat bladder cancer. Because of the high recurrence rates, both professional urology associations and NCCN Guidelines recommend instillation of a cytotoxic agent following TURBT for NMIBC, although in the U.S., there are no FDA-approved agents for this indication.

Kite Pharma Provides Update on KTE-C19 Clinical Trial

On August 17, 2015 Kite Pharma, Inc. (Kite) (Nasdaq:KITE) treported an update from the Company’s ongoing Phase 1/2 clinical trial of KTE-C19 in patients with refractory aggressive non-Hodgkin’s lymphoma (NHL) who have failed prior chemotherapy treatments and have a poor prognosis (Press release, Kite Pharma, AUG 17, 2015, View Source [SID:1234507281]). KTE-C19 is an investigational therapy in which a patient’s T cells are genetically modified to express a Chimeric Antigen Receptor (CAR) designed to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias.

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In May, Kite announced that the first patient was treated with KTE-C19 in the Phase 1 portion of the trial and we have since treated multiple patients. Complete responses have been observed by investigators. The responses happened shortly after treatment was administered and Kite is monitoring these patients to determine durability of treatment. To date, toxicities associated with treatment have been similar to those observed in the National Cancer Institute’s study of anti-CD19 CAR T cell therapy. There was one patient death early in the study, which was determined to be unrelated to KTE-C19 by the study investigator. After appropriate discussions with the U.S. Food and Drug Administration (FDA), Kite continued to enroll and treat patients in its study and the study was never placed on clinical hold. Kite has submitted an abstract and plans to present top-line data from the Phase 1 portion of the trial at the upcoming 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, to take place in Orlando, FL, December 5-8, 2015.

"We are encouraged by the progress of the KTE-C19 clinical trial and excited by the responses we have seen so far. We believe the KTE-C19 clinical findings are in line with previous results demonstrating the potential of this promising therapeutic approach," said Arie Belldegrun, M.D., FACS, Chairman, President and Chief Executive Officer of Kite. "In agreement with ASH (Free ASH Whitepaper), we have taken this exceptional step of providing an update on the trial in order to address recent misinformation in the market related to our clinical program. We are on track to transition to the Phase 2 portion of the trial and plan to present Phase 1 data at ASH (Free ASH Whitepaper) later this year."

Kite’s Phase 1/2 clinical trial of KTE-C19 is a single arm, open-label, multi-center study, designed to determine the safety and efficacy of KTE-C19 in patients with refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL), or transformed follicular lymphoma (TFL). Upon completion of the Phase 1 portion of the study, Kite expects to proceed with the Phase 2 portion that will include a total of approximately 112 patients. Additional information about Kite’s Phase 1/2 study may be found at ClinicalTrials.gov, using Identifier NCT: 02348216.

DelMar Pharmaceuticals Receives Additional Non-Dilutive Funding from NRC-IRAP to Support Expanded Research Program with Lead Product Candidate VAL-083

On August 17, 2015 DelMar Pharmaceuticals, Inc. (OTCQX: DMPI) ("DelMar" and the "Company"), a biopharmaceutical company focused on developing and commercializing proven cancer therapies in new orphan drug indications, reported an increase in funding of up to CDN$287,000 from the National Research Council of Canada Industrial Research Assistance Program (NRC-IRAP) to support an ongoing research project (Press release, DelMar Pharmaceuticals, AUG 17, 2015, View Source [SID:1234507280]).

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With the increase, NRC-IRAP’s total non-refundable financial contributions to DelMar total CDN$420,000 to date.

"We are very pleased with the continued support to accelerate and expand our non-clinical research to further establish the competitive differentiation of our lead product candidate VAL-083," said Jeffrey Bacha, president & CEO of DelMar Pharmaceuticals. "We have made tremendous progress this year executing on our clinical development strategy with VAL-083. These additional funds, along with support from the technological expertise and advisory services provided by NRC-IRAP, will be invaluable as we continue to advance VAL-083 in refractory glioblastoma multiforme (GBM) and expand its utility in other tumor types."

This most recent funding builds upon previous non-repayable contributions from NRC-IRAP totaling CDN$133,000 that supported research conducted in collaboration with University of British Columbia, the Vancouver Prostate Centre and the B.C. Cancer Agency. The increase in funding reflects the success of this research program to date. The results of the collaborative project, funded in part by the NRC-IRAP, support differentiation of the anti-cancer mechanism of VAL-083 and its potential as a treatment for other tumor types beyond its current Phase II GBM clinical trial into non-small cell lung cancer (NSCLC) and other solid tumors.

DelMar’s drug development program leverages numerous preclinical and clinical Phase I/Phase II historical research studies in which VAL-083 demonstrated activity, safety and efficacy in treating a wide range of tumor types including lung, brain, cervical and ovarian cancers. The Company intends to expand its clinical development program for VAL-083 into NSCLC and other solid tumors to target specific unmet medical needs in major cancer indications.

DelMar recently presented interim data of its ongoing Phase I/II clinical trial in patients with recurrent GBM at the American Association of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual meeting. The Company confirmed the completion of the Phase I dose-escalation portion of the trial and presented data supporting a dose response trend: Patients receiving a dose greater than or equal to 30mg/m2 had a median survival of 9.0 months vs. 4.4 months at doses less than 10mg/m2. DelMar also confirmed the initiation of a 14-patient Phase II expansion cohort at a dose of 40mg/m2. The purpose of the Phase II expansion cohort is to gain additional information about the safety and efficacy of VAL-083 at the 40mg/m2 dose prior to advancement into registration-directed Phase II/III clinical trials.

About VAL-083
VAL-083 is a "first-in-class", small-molecule chemotherapeutic. In more than 40 Phase 1 and 2 clinical studies sponsored by the U.S. National Cancer Institute, VAL-083 demonstrated safety and efficacy in treating a number of cancers including lung, brain, cervical, ovarian tumors and leukemia. VAL-083 is approved in China for the treatment of chronic myelogenous leukemia and lung cancer and has received orphan drug designation in Europe and the U.S. for the treatment of gliomas.

DelMar is currently studying VAL-083 in a multi-center Phase I/II clinical trial for patients with refractory glioblastoma multiforme (GBM) in accordance with the protocol that has been filed with the U.S. Food and Drug Administration (FDA). As a potential treatment for glioblastoma, VAL-083’s mechanism of action appears to be unaffected by the expression of MGMT, a DNA repair enzyme that causes chemotherapy resistance to front-line treatment with Temodar (temozolomide).