Juno Therapeutics’ CAR T Cell Product Candidate JCAR017 Continues to Demonstrate Encouraging Outcomes in Pediatric Patients With Relapsed/Refractory CD19 Positive Acute Lymphoblastic Leukemia

On April 21, 2015 Juno Therapeutics reported encouraging clinical responses in a Phase 1 study evaluating JCAR017 in pediatric patients with relapsed/refractory acute lymphoblastic leukemia (ALL) (Press release, Juno, APR 21, 2015, View Source [SID:1234503099]). The results of this trial to date demonstrated that 91% of patients achieved a complete remission as documented by flow cytometry. Adverse events were consistent with what has been previously reported. JCAR017 is a chimeric antigen receptor (CAR) T cell product candidate subject to a licensing arrangement with Seattle Children’s Research Institute. The results were presented in an oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2015 in Philadelphia, Pennsylvania.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The 91% remission rate in this Phase 1 study of JCAR017 is highly encouraging, particularly when considering these pediatric patients failed to respond to standard treatments," said Michael Jensen, M.D., Scientific Co-Founder, Juno Therapeutics, and Director of the Ben Towne Center for Childhood Cancer Research, Seattle Children’s Research Institute. "Based on these results we are eager to advance this study, and to continue advancing the use of cell therapies to change how we treat cancer and provide patients the opportunity for better treatment options."

Data from the ongoing, open-label, Phase 1 dose escalation study were presented by Dr. Jensen at AACR (Free AACR Whitepaper) today. The study is evaluating escalating doses of JCAR017 in pediatric patients with relapsed/refractory CD19 positive ALL. JCAR017, a defined cell product candidate, was successfully manufactured for all enrolled patients. The study was designed and conducted by Seattle Children’s Research Institute with patients from Seattle Children’s Research Institute.

In an intent-to-treat analysis, a complete remission documented by flow cytometry in 20 of 22 patients (91%) was measured. The complete remissions were observed at all doses evaluated in patients with prior CD19-directed therapy as well as in patients with infantile ALL. Severe cytokine release syndrome and/or severe neurotoxicity was observed in 8 patients. Four relapses have been observed to date, only one of which had CD19 positive disease.

Clinical data of Medigene’s dendritic cell (DC) vaccines presented at AACR conference

On April 20, 2015 Medigene AG (MDG1, Frankfurt, Prime Standard) reported that early clinical data of its dendritic cell (DC) vaccines were presented today at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Philadelphia, USA (Press release, MediGene, APR 20, 2015, View Source [SID:1234506589]). The clinical data were collected in an ongoing compassionate use program[1] conducted at the Department of Cellular Therapy at the Oslo University Hospital, Norway, under the responsibility of Prof. Gunnar Kvalheim. The poster presentation titled "A new generation of dendritic cells to improve cancer therapy shows prolonged progression free survival in patients with solid tumors" provides data from patients with various types of tumour which were included in this program.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In summary, one lung cancer patient, one prostate cancer patient, four glioblastoma patients and three acute myeloid leukaemia (AML) patients have started treatment with dendritic cells so far. The new generation of dendritic cells characterized by superior in-vitro functionality when compared to commonly used dendritic cells could be produced from cells of all patients, regardless of the type of malignancy. The included patients suffering from solid tumours clearly showed a longer progression free survival than could be expected according to the stage of their disease, except for the patient with prostate cancer, who due to personal reasons prematurely dropped out of the program. The three AML patients which were included in this dendritic cell compassionate use program have been showing a promising course of disease, however these cases are still too early for evaluation.

Conclusion of this evaluation by Prof. Gunnar Kvalheim, Head of Department of Cellular Therapy, Oslo University Hospital: "Solid tumour patients suffering from advanced disease treated with these DC vaccines have a prolonged progression free survival, showing that this immunotherapeutic approach will be a promising alternative to current standard therapies."

More detailed information can be found in the abstract under the following link: View Source;sKey=eae2d342-dd5a-41ba-9eb4-63990d3122b8&cKey=1ff23671-9f76-44fd-891b-50b8fbfdd1b6&mKey=19573a54-ae8f-4e00-9c23-bd6d62268424

The Oslo University Hospital has an agreement with Medigene for use of Medigene`s new generation DC vaccines for their ongoing academic clinical studies.

Prof. Dolores J. Schendel, Chief Scientific Officer of Medigene AG: "These positive results encourage us in pursuing our DC vaccine development program for which we have recently started our own clinical AML trial, complementing the ongoing academic clinical studies."

About Medigene’s DC vaccines: The platform for the development of new generation antigen-tailored DC vaccines is the most advanced platform of the three highly innovative and complementary immunotherapy platforms of Medigene Immunotherapies. The DC vaccines are currently being evaluated in a company-sponsored clinical trial in acute myeloid leukaemia (AML) as well as in two ongoing clinical investigator-initiated trials: a clinical phase I/II trial in AML at the Ludwig-Maximilian University Hospital Großhadern, Munich, and a clinical phase II trial in prostate cancer at the Oslo University Hospital. Moreover, a compassionate use program is being conducted at the Department of Cellular Therapy at the Oslo University Hospital.

Medigene’s dendritic cell product platform allows the design of new generation dendritic cell vaccines. Dendritic cells can take up antigens efficiently, process them and present them on their surface in a form that can induce antigen-specific T cells to proliferate and mature. This way T cells can recognize and eliminate antigen-bearing tumour cells. Dendritic cells can also induce natural killer cells (NK cells) to become active and attack tumour cells. Scientists of Medigene Immunotherapies have developed new, fast and efficient methods for preparing autologous (patient-specific) mature dendritic cells which have relevant characteristics to activate both T cells and NK cells. The dendritic cells can be loaded with various tumour antigens to treat different types of cancer and are designed for treatment of minimal residual disease or use in combination therapies.

Curadev Announces Research Collaboration and Licensing Agreement to Develop Cancer Immunotherapeutic

On April 20, 2015 Curadev Pharma reported that it has entered into a research collaboration and exclusive license agreement with Roche for the development and commercialization of IDO1 and TDO inhibitors (Press release, Curadev, APR 20, 2015, View Source [SID:1234503106]). The agreement covers the development of the lead preclinical immune tolerance inhibitor and a research collaboration with Roche’s research and early development organization to further explore the IDO and TDO pathways.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

IDO1 (indoleamine-2, 3-dioxygenase-1) and TDO (tryptophan-2, 3-dioxygenase) are enzymes that mediate cancer-induced immune suppression. This mechanism is exploited by tumor cells as well as certain type of immune cells, limiting the anti-tumor immune response.

Dual inhibition of the IDO1 and TDO pathways promises to maintain the immune response, prevent local tumor immune escape and potentially avoid resistance to other immunotherapies when used in combination, and could lead to new treatment options for cancer patients. Curadev’s preclinical lead-compound, a small-molecule that shows potent inhibition of the two rate-limiting enzymes in the tryptophan – to kynurenine metabolic pathways, has the potential for mono therapy as well as combination with Roche’s broad oncology pipeline and portfolio.

"We are very excited to be working with the global leader in oncology with their unrivalled expertise in clinical development," said Arjun Surya, PhD, Chief Scientific Officer, Curadev. "The collaboration acknowledges our focused research efforts on patient-critical drug targets that have yielded a drug candidate that could make a significant difference in the development of novel treatments for patients suffering from cancer."

Under the terms of agreement, which includes a research collaboration with Roche’s research and early development organization to further extend Curadev’s findings, Curadev will receive an upfront payment of $25 million and will be eligible to receive up to $530 million in milestone payments based on achievement of certain predetermined events and sales levels as well as escalating royalties potentially reaching double digits for the first product from the collaboration developed and commercialized by Roche. Curadev would also be eligible for milestones and royalties on any additional products resulting from the research collaboration. Roche will fund future research, development, manufacturing and commercialization costs and will also provide additional research funding to Curadev for support of the research collaboration.

FDA Accepts Supplemental BLA and Grants Priority Review for ADCETRIS® (Brentuximab Vedotin) in the AETHERA Setting for the Post-Transplant Consolidation Treatment of Hodgkin Lymphoma Patients at High Risk of Relapse

On April 20, 2015 Seattle Genetics reported that the U.S. Food and Drug Administration (FDA) has accepted for filing a supplemental Biologics License Application (BLA) for ADCETRIS (brentuximab vedotin) in the AETHERA setting for the post-transplant consolidation treatment of Hodgkin lymphoma (HL) patients at high risk of relapse or progression (Press release, Seattle Genetics, APR 20, 2015, View Source [SID:1234503088]). The FDA granted Priority Review for the application and the Prescription Drug User Fee Act (PDUFA) target action date is August 18, 2015. The submission of the supplemental BLA is based on positive results from a phase 3 clinical trial called AETHERA that was designed to determine if 16 cycles of ADCETRIS as consolidation therapy immediately following an autologous stem cell transplant (ASCT) could extend progression-free survival (PFS) in HL patients at high risk of relapse or progression. 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. ADCETRIS is approved in relapsed HL and sALCL but is currently not approved for consolidation therapy in HL patients immediately after ASCT.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 relapse or are refractory to frontline treatment. The standard for these patients is to proceed to an ASCT but approximately half of all HL patients who undergo an ASCT experience subsequent disease relapse.

"The FDA’s filing of our supplemental BLA and priority review designation for ADCETRIS as consolidation therapy represents a significant milestone towards our goal of making ADCETRIS available to high risk HL patients immediately following an autologous stem cell transplant who currently have no therapeutic options to prevent progression," said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. "The phase 3 AETHERA trial demonstrated that using ADCETRIS in this setting significantly improved progression-free survival with a manageable safety profile. We look forward to working with the FDA during their review of our application for approval of this additional indication for ADCETRIS."

The positive results from the phase 3 AETHERA trial were published in The Lancet in March 2015 and were presented at the 56th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2014. Results from the AETHERA trial in 329 HL patients at high risk of relapse following ASCT 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 and a p-value of 0.001. Median PFS was 43 months for patients who received ADCETRIS versus 24 months for patients who received placebo. The two-year PFS rate was 63 percent in the ADCETRIS arm compared to 51 percent in the placebo arm.

The PFS benefit was consistent across all pre-specified subgroups, including primary refractory patients, patients who relapsed within twelve months of frontline therapy and patients who relapsed after twelve months with extranodal disease.

ADCETRIS-treated patients received a median of 15 treatment cycles and 48 percent received the maximum of 16 cycles, indicating generally acceptable tolerability and a manageable adverse reaction profile.

The most common adverse events in the ADCETRIS arm were peripheral sensory neuropathy (56 percent), neutropenia (35 percent), upper respiratory tract infection (26 percent), fatigue (24 percent) and peripheral motor neuropathy (23 percent). The most common adverse events in the placebo arm were upper respiratory tract infection (23 percent), fatigue (18 percent) peripheral sensory neuropathy (16 percent), cough (16 percent) and neutropenia (12 percent). Eighty-five percent of patients with peripheral neuropathy on the ADCETRIS arm had resolution or improvement in symptoms with a median time to improvement of 23.4 weeks.

Submission of safety data from the AETHERA trial to the FDA is a post-marketing requirement.

About ADCETRIS

ADCETRIS is being evaluated broadly in more than 30 ongoing clinical trials, including four phase 3 studies, in earlier lines of its approved 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 (brentuximab vedotin) 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 received accelerated approval from the FDA and approval with conditions from Health Canada for two indications: (1) the treatment of patients with HL after failure of ASCT or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not ASCT candidates, and (2) the treatment of patients with sALCL after failure of at least one prior multi-agent chemotherapy regimen. The indications for ADCETRIS are approved under accelerated approval based on overall response rate. An improvement in patient-reported outcomes or survival has not been established. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

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 50 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 will be solely responsible for development costs.

About 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. HL is distinguished from other types of lymphoma by the presence of one characteristic type of cell, known as the Reed-Sternberg cell. 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.

Global Strategic Partners Merck KGaA, Darmstadt, Germany, and Pfizer Initiate Phase III Study with Avelumab* in Patients with Stage IIIb/IV Non-Small Cell Lung Cancer

On April 20, 2015 Merck KGaA and Pfizer reported the initiation and first patient treated in the international Phase III study (EMR 100070-004) designed to assess the efficacy and safety of the investigational cancer immunotherapy avelumab (MSB0010718C), compared with docetaxel, in patients with stage IIIb/IV non-small cell lung cancer (NSCLC) who have experienced disease progression after receiving a prior platinum-containing doublet therapy(Press release, Pfizer, APR 20, 2015, View Source [SID:1234503077]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Phase III study is an open-label, multicenter, 1:1 randomized clinical trial where patients with stage IIIb/IV NSCLC will receive either avelumab or docetaxel, regardless of PD-L1 status. Approximately 650 patients will participate across 290 sites in more than 30 countries in North America, South America, Asia, Africa and Europe. In North America, clinical trials on behalf of Merck KGaA, Darmstadt, Germany, will be conducted by EMD Serono, the company’s US and Canadian biopharmaceutical businesses. The study is part of the JAVELIN clinical trial program for avelumab.

The primary endpoint of the study is overall survival (OS) in patients with programmed death-ligand 1 positive (PD-L1+) stage IIIb/IV NSCLC who have experienced disease progression after receiving a prior platinum-containing doublet therapy. Secondary endpoints will be assessed across the entire study population regardless of PD-L1 status and include OS; overall response rate (ORR); progression-free survival (PFS); and patient-reported outcomes.

"New and innovative treatment strategies are urgently needed to improve overall survival for patients with NSCLC, and we are investigating avelumab as a potential treatment option for patients with this very difficult-to-treat disease," said Dr. Luciano Rossetti, Global Head of R&D of the biopharmaceutical business of Merck KGaA, Darmstadt, Germany. "The treatment of the first patient in the Phase III trial is an important milestone for our immuno-oncology alliance."

"This trial marks the first of several registration studies we are planning to initiate this year together, and underscores our commitment to accelerating the development of medications for patients with cancer," said Dr. Mace Rothenberg, Senior Vice President of Clinical Development and Medical Affairs and Chief Medical Officer for Pfizer Oncology. "Through this alliance, we will have the opportunity to combine the promising anti-PD-L1 antibody, avelumab, with our combined portfolios of approved and investigational oncology therapies, which may provide an exciting opportunity to potentially broaden the use of immunotherapy for patients with cancer."

The JAVELIN clinical trial program also includes an international Phase II trial to investigate avelumab in patients with metastatic Merkel cell carcinoma; an international Phase I trial to investigate avelumab in patients with metastatic or locally advanced solid tumors, and a Phase I trial to investigate avelumab in Japanese patients with metastatic or locally advanced solid tumors with an expansion part in Asian patients with gastric cancer. The Phase I program for avelumab includes more than 840 patients treated across multiple tumor types, including NSCLC, breast cancer, gastric cancer, ovarian cancer, bladder cancer, melanoma and mesothelioma.

*Avelumab is the proposed International Nonproprietary Name (INN) for the anti-PD-L1 monoclonal antibody (MSB0010718C)

About Non-Small Cell Lung Cancer

Globally, lung cancer is the most common cause of cancer-related deaths in men and the second most common in women, responsible for almost twice as many deaths as both breast and prostate cancer combined1. NSCLC is the most common type of lung cancer, accounting for 85 to 90 percent of all lung cancers2. Locally advanced and metastatic disease account for approximately 35 to 40 percent3 and 70 percent4 of patients, respectively with NSCLC.

Avelumab

Avelumab (also known as MSB0010718C) is an investigational fully human anti-PD-L1 IgG1 monoclonal antibody. By inhibiting PD-L1 interactions, avelumab is thought to enable the activation of T-cells and the adaptive immune system. By retaining a native Fc-region, avelumab is thought to engage the innate immune system and induce antibody-dependent cell-mediated cytotoxicity (ADCC). In November, 2014, Merck KGaA, Darmstadt, Germany, and Pfizer announced a strategic alliance to co-develop and co-commercialize avelumab.

JAVELIN Clinical Trial Program for Avelumab

JAVELIN is an expansive international clinical trial program exploring the use of PD-L1 inhibition with avelumab to treat multiple types of cancer. The JAVELIN clinical trial program includes a Phase III study designed to assess the efficacy and safety of avelumab compared with docetaxel in patients with stage IIIb/IV NSCLC who have experienced disease progression after receiving a prior platinum-containing doublet therapy; an international Phase II open-label multicenter trial to investigate the clinical activity and safety of avelumab in patients with metastatic Merkel cell carcinoma; an international Phase I open-label, multiple ascending dose trial to investigate the safety, tolerability, pharmacokinetics, biological and clinical activity in patients with metastatic or locally advanced solid tumors; and a Phase I trial to investigate the tolerability, safety, pharmacokinetics, biological, and clinical activity of avelumab in Japanese patients with metastatic or locally advanced solid tumors with an expansion part in Asian patients with gastric cancer.

Alliance between Merck KGaA, Darmstadt, Germany, and Pfizer Inc, New York, US

Immuno-oncology is a top priority for Merck KGaA, Darmstadt, Germany, and Pfizer Inc. The global strategic alliance between Merck KGaA, Darmstadt, Germany, and Pfizer Inc, New York, US, enables the companies to benefit from each other’s strengths and capabilities and further explore the therapeutic potential of avelumab, an investigational anti-PD-L1 antibody initially discovered and developed by Merck KGaA, Darmstadt, Germany. The immuno-oncology alliance will jointly develop and commercialize avelumab and advance Pfizer’s PD-1 antibody. The companies will collaborate on up to 20 high priority immuno-oncology clinical development programs, including combination trials, many of which are expected to commence in 2015.