The Therapy Acceleration Program® (TAP) identifies and funds innovative projects related to therapies that have the potential to change the standard of care for patients with blood cancer, especially in areas of high unmet medical need.
TAP funding assists both clinical investigators and companies in gaining critical clinical proof of concept data that better enables them to obtain the resources they need or a partner to complete the testing, registration and marketing of new treatments for leukemia, lymphoma and myeloma.
TAP funding is different from the traditional grant at LLS. The TAP review process is separate from the grant process and each approved project is closely monitored by TAP staff.
For information on how to apply for TAP, please click here for more information. Click here for biotech and here for academic application information.
Latest News from Current and Former TAP Partners
2019 click here for 2018 click here for 2017 click here for 2016
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X4 Pharmaceuticals, Inc. (Nasdaq:XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, and The Leukemia & Lymphoma Society (LLS) announced a collaboration to accelerate the development of X4’s lead product candidate, mavorixafor (X4P-001) for the treatment of Waldenström’s macroglobulinemia (WM), a rare form of non-Hodgkin lymphoma.
Mavorixafor was selected for LLS’s Therapy Acceleration Program® (TAP), a strategic initiative where LLS builds business alliances and collaborations with biotechnology companies and academic researchers to speed the development of new therapies for blood cancers. Under the collaboration, X4 will conduct a multi-national Phase 1/2 clinical trial to evaluate the safety and assess the preliminary anti-tumor activity of mavorixafor in combination with ibrutinib in WM patients. The trial is planned to commence this year. Lee Greenberger, Ph.D., chief scientific officer of LLS, will also serve as a member of an advisory board to X4, providing important strategy and partnership guidance throughout the trial.
- Selvita (WSE:SLV), a clinical stage company engaged in the research and development of novel cancer therapies as well as provision of drug discovery and development services, announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to conduct a Phase 1 study of selective CDK8 inhibitor SEL120 in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (HR-MDS). The open-label, dose-escalation study is designed to evaluate safety and tolerability of SEL120 as well as to establish the recommended dose of SEL120 for subsequent clinical development.
The Leukemia & Lymphoma Society (LLS) is a proud partner of Selvita and is providing up to $3.25 million in co-funding over four years, through its Therapy Acceleration Program® (TAP), in order to complete SEL120 IND-enabling studies and a phase I trial in AML.
- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s investigational new drug (IND) application for KO-539, a potent and selective small molecule inhibitor of the menin-mixed lineage leukemia (menin-MLL) protein-protein interaction. The Company anticipates initiating a Phase 1 clinical trial of KO-539 in relapsed or refractory acute myeloid leukemia (AML) in the second quarter of 2019. In December 2014, Kura Oncology entered into an exclusive license agreement with the University of Michigan to access intellectual property and technology related to a class of small molecule inhibitors targeting menin-MLL that have potential utility as a treatment of acute leukemias and other cancers. LLS funded the University of Michigan through the Therapy Acceleration Program® (TAP).
Dr. Grembecka at the University of Michigan first received funding from LLS in 2009 through Translational Research Program (TRP) grant, which helps advance discoveries from the lab into clinical trials. She is also a recipient of an LLS Career Development Program (CDP) award. LLS has continuously supported this work since the awarding of the first grant, and in 2010, in light of the tremendous promise of some of the small molecule compounds she was working on, transferred the project into its TAP program. TAP is designed to help research projects advance into the drug development pipeline. One of the goals of TAP is to capitalize on LLS's robust portfolio of grant-supported academic projects - particularly those that show promise of near-term clinical benefit for patients with blood cancers. Since 2009, LLS has provided Dr. Grembecka's lab more than $8 million in funding through its TRP, CDP and TAP programs. (click here for LLS press release from March 30 2015)
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The FDA has approved tagraxofusp-erzs (SL-401; Elzonris) infusion for the treatment of adult and pediatric patients, aged ≥2 years, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). The approval is based on data from 2 cohorts of a multicenter, open-label, nonrandomized single-arm trial. In the first cohort, 7 of 13 patients (54%) treated with tagraxofusp achieved a complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc). In the second cohort, which comprised 15 patients, 1 patient achieved CR and 1 experienced a CRc. The study (NCT02113982) was sponsored by Stemline Therapeutics, Inc. (Nasdaq: STML) and is supported in part by The Leukemia & Lymphoma Society's Therapy Acceleration Program® (TAP). This is the third approval for a TAP supported project.
“Prior to today’s approval, there had been no FDA approved therapies for BPDCN,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, acting director of the Office of Hematology and Oncology Products, in the FDA’s Center for Drug Evaluation and Research. “The standard of care has been intensive chemotherapy followed by bone marrow transplantation. Many patients with BPDCN are unable to tolerate this intensive therapy, so there is an urgent need for alternative treatment options.” -
Verastem, Inc. (Nasdaq: VSTM) announced a collaboration with The Leukemia & Lymphoma Society® (LLS), to accelerate the development of duvelisib for the treatment of patients with peripheral T-cell lymphoma (PTCL), an aggressive type of non-Hodgkin lymphoma (NHL). Verastem Oncology’s duvelisib was selected for the LLS’s Therapy Acceleration Program® (TAP) which provides additional resources to support the development of therapies for patients with blood cancers. The Company plans to use the funds to conduct certain translational and clinical activities relating to the development of duvelisib for the treatment of PTCL. LLS and Verastem Oncology will share the cost of the development program, portions of which will be conducted in collaboration with Memorial Sloan Kettering Cancer Center, The Dana-Farber Cancer Institute, The Washington Universityin St. Louis and Stanford University.
Verastem Oncology is currently conducting an open-label, multicenter, Phase 2 clinical trial (the PRIMO study) evaluating the efficacy and safety of duvelisib monotherapy in adult patients with histologically confirmed relapsed or refractory PTCL. This study is expected to enroll approximately 120 patients. In addition, funds from the TAP will be used to support the expansion of an investigator-sponsored study being conducted by Steven Horwitz, MD, Medical Oncologist, Memorial Sloan Kettering Cancer Center and NYC Health + Hospitals/Bellevue. This study is evaluating the combination of duvelisib with romidepsin, an HDAC inhibitor, with a goal to create deeper and more durable responses for patients with relapsed or refractory PTCL. Initial data on the combination as presented at the American Society of Hematology (ASH) 2017 Annual Meeting, demonstrated a 64% overall response rate including 34% complete responses.
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Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company focused on discovering and developing highly targeted cancer immunotherapies that harness the power of innate and adaptive immunity (NK and T cells), announced that it has entered into a strategic collaboration agreement with Genentech, a member of the Roche Group, to develop and commercialize novel NK cell engager-based immunotherapeutics to treat multiple cancers. Affimed will apply its proprietary Redirected Optimized Cell Killing (ROCK®) platform, which enables the generation of both NK cell and T cell-engaging antibodies, to discover and advance innate immune cell engager-based immunotherapeutics of interest to Genentech. The collaboration includes candidate products generated from Affimed’s ROCK® platform and multiple undisclosed solid and hematologic tumor targets. Affimed and Genentech will collaborate on the discovery, early research and late-stage research phases. Genentech will be responsible for clinical development and commercialization worldwide.
Under the terms of the agreement, Affimed will receive $96 million in an initial upfront payment and other near-term committed funding. Affimed may be eligible to receive up to an additional $5.0 billion over time, including payments upon achievement of specified development, regulatory and commercial milestones, and royalties on sales. The agreement is subject to customary closing conditions and closing is expected to occur in the third quarter of 2018.
LLS has funded Affimed for the development of AFM13 since 2013 through its Therapy Acceleration Program® (TAP). AFM13 is a first-in-class immunotherapy drug designed to treat HL patients and patients with CD30-positive malignancies. LLS has committed to investing up to $4.4 million to support multiple clinical projects.
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Jazz Pharmaceuticals plc (Nasdaq : JAZZ) announced that the European Commission approved Vyxeos® 44 mg/100 mg powder for concentrate for solution for infusion for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Vyxeos is an advanced liposomal formulation that delivers a synergistic molar ratio of daunorubicin and cytarabine. The European Commission approval extends to all European Union Member States, as well as Iceland, Norway and Liechtenstein.
The Marketing Authorisation Application (MAA) for Vyxeos included clinical data from five studies, including the pivotal Phase 3 study. Data from the Phase 3 study was published in the Journal of Clinical Oncology in July 2018. The study evaluated the efficacy and safety of Vyxeos compared to 7+3 chemotherapy in 309 patients 60 to 75 years of age with newly diagnosed t-AML or AML-MRC, a rapidly progressing and life-threatening blood cancer. The study met its primary endpoint as Vyxeos demonstrated a superior improvement in overall survival compared to the 7+3 treatment regimen. The median overall survival for the Vyxeos treatment group was 9.6 months compared with 5.9 months for the 7+3 treatment group. Vyxeos was also associated with a significantly higher remission rate than 7+3 with a complete response rate of 37% versus 26%. In addition, the overall rate of hematopoietic stem cell transplant (HSCT) was 34% in the Vyxeos arm and 25% in the 7+3 arm. The reported adverse reactions with Vyxeos were generally consistent with the known safety profile of cytarabine and daunorubicin therapy.
LLS has funded the development of Vyxeos since 2009 through its Therapy Acceleration Program® (TAP). Under the TAP initiative, LLS partners directly with biotechnology companies to accelerate the development of promising therapies for unmet medical needs, many of which otherwise might not receive support. LLS partnered with Celator Pharmaceuticals, the developer of Vyxeos, through TAP; Celator was acquired by Jazz Pharmaceuticals in 2016.
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Kite, a Gilead Company (Nasdaq: GILD), announced that the European Commission (EC) has granted Marketing Authorization for Yescarta® (axicabtagene ciloleucel) as a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy. The Marketing Authorization approves axicabtagene ciloleucel for use in the 28 countries of the European Union, Norway, Iceland and Liechtenstein. Axicabtagene ciloleucel is a chimeric antigen receptor T cell (CAR T) therapy, which harnesses a patient’s own immune system to fight certain types of blood cancer. The cell therapy has been proven to induce complete response (no detectable cancer) in a proportion of patients with relapsed or refractory DLBCL and PMBCL, which are aggressive forms of non-Hodgkin lymphoma (NHL).
The Marketing Authorization Application (MAA) is supported by data from the ZUMA-1 trial of axicabtagene ciloleucel in adult patients with refractory aggressive NHL. In the single-arm trial, 72 percent of patients (n=73/101) who received a single infusion of axicabtagene ciloleucel responded to therapy, with 51 percent (n=52/101) achieving a complete response (as assessed by an independent review committee, median follow-up of 15.1 months). At one year following infusion, 60 percent of patients were alive and the median overall survival (OS) had not been reached.
LLS has supported the ZUMA-1 trial leading to the approval of Yescarta since 2015 through its Therapy Acceleration Program® (TAP). Through TAP, LLS partners directly with biotechnology companies, including the developer of Yescarta, Kite, a Gilead Company, to help accelerate the development of promising treatments.
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Stemline Therapeutics, Inc. (Nasdaq: STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s Biologics License Application (BLA) for ELZONRIS (tagraxofusp; SL-401) for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). The FDA also granted Priority Review for the BLA and has set a target action date of February 21, 2019, under the Prescription Drug User Fee Act (PDUFA). The FDA grants Priority Review to product applications that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
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Sutro Biopharma, Inc., and The Leukemia & Lymphoma Society® , or LLS, are partnering to develop STRO-001, Sutro’s CD74-targeting antibody-drug conjugate, to treat relapsed and/or refractory multiple myeloma and non-Hodgkin’s lymphoma. CD74 is a protein highly expressed in B-cell malignancies such as myeloma and lymphoma. LLS has agreed to contribute clinical development funding for STRO-001 — Sutro’s first internally-developed product candidate — through its Therapy Acceleration Program®, which forges collaborations with biotechnology companies to help bring innovative therapies to patients faster. Sutro intends to use the funding for a Phase 1 study, initiated in April of this year, to evaluate STRO-001 for treating multiple myeloma, diffuse large B-cell lymphoma, mantle cell lymphoma and indolent lymphomas, such as follicular lymphoma.
The clinical trial is currently open and enrolling patients at City of Hope Comprehensive Cancer Center, Duarte, CA; Medical College of Wisconsin, Milwaukee; Texas Oncology, Austin; Rocky Mountain Cancer Centers, Aurora, CO; and Virginia Cancer Specialists in Fairfax. Sutro plans to add more sites later this year.
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OncoPep, Inc. announced the initiation of a Phase 1b clinical trial evaluating the safety and tolerability of PVX-410, an investigational multi-peptide cancer vaccine, for patients with smoldering multiple myeloma (SMM). Smoldering multiple myeloma is an early precursor to a rare blood cancer known as multiple myeloma, which affects plasma cells. The open-label investigator-sponsored study, led by Noopur Raje, M.D. at Massachusetts General Hospital, will assess two different combinations of the study drugs; a combination of PVX-410 along with an investigational histone deacetylase (HDAC 6) inhibitor, citarinostat (CC-96241, Celgene) and a triple combination of the PVX-410 vaccine, citarinostat, and lenalidomide. The study is expected to enroll approximately 20 patients at multiple trial sites, including Massachusetts General Hospital. More information on the trial can be found at clinicaltrials.gov, identifier number NCT02886065.
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miRagen Therapeutics, Inc. (NASDAQ: MGEN), a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, and The Leukemia & Lymphoma Society® (LLS) announced that they entered into an agreement providing for collaboration and funding intended to support the development of miRagen’s cobomarsen (also known as MRG-106), a microRNA-155 inhibitor, for the treatment of patients with the mycosis fungoides (MF) form of cutaneous T-cell lymphoma (CTCL). There are approximately 16,000-20,000 people in the U.S. with mycosis fungoides.
LLS will provide up to $5 million through the purchase of miRagen common stock to help support the SOLAR trial. This includes a $1 million investment upon signing of the agreement and potential additional stock purchases upon the achievement of certain milestones during the SOLAR trial. Funding from LLS is being provided under its Therapy Acceleration Program® (TAP), a strategic initiative through which LLS partners directly with biotechnology companies and academic institutions to help accelerate the development of promising therapies.
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Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, announced that it has completed submission of a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for ELZONRIS (tagraxofusp; SL-401), a potential treatment for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a CD123+ malignancy of unmet medical need for which the agent was awarded Breakthrough Therapy Designation (BTD). The study (NCT02113982) is sponsored in part by The Leukemia & Lymphoma Society's Therapy Acceleration Program® (TAP).
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Johns Hopkins University spinout WindMIL grabbed $32.5M to fuel its work on a next-gen approach to cell therapy. The Baltimore-based startup is tapping memory T cells residing in the bone marrow — and their innate ability to recognize tumors — to make what they call a novel class of cancer therapy. Founded out of the labs of Johns Hopkins University professors Ivan Borrello and Kim Noonan, WindMIL got its name, in part, from the result of reactivating and expanding these memory T cells extracted from the patient’s body: marrow infiltrating lymphocytes, or MILs. WindMIL Therapeutics’ lead program is currently enrolling patients in a Phase 2b clinical trial for patients in high-risk myeloma (clinicaltrials.gov ID# NCT01858558). LLS has supported this project through its Therapy Acceleration Program® (TAP) with Johns Hopkins University since July 2013. LLS committed to support this project with up to $3.5 million over five years. The project evolved from Borrello's previous work supported by LLS through a Translational Research Program grant.
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Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company focused on discovering and developing highly targeted cancer immunotherapies, today presented new interim data from the Phase 1b dose escalation study evaluating AFM13, its lead NK cell engager candidate, at the European Hematology Association (EHA) 23rd Congress in Stockholm. Assessment of 18 patients treated at the highest AFM13 dose showed best overall response rate (ORR) of 89% (16/18 patients) and complete metabolic response rate (CmR) of 28% (5/18 patients). The ORR and CmR for these 18 patients compare favorably to those of anti-PD-1 monotherapy in similar patient populations. Responders included three patients who were either primary refractory to or had relapsed during front-line therapy and were refractory to all subsequent lines of therapy. The combination of AFM13 and pembrolizumab was well tolerated, with most adverse events mild to moderate in nature and manageable with standard of care measures.
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Forty Seven, Inc., a clinical-stage, immuno-oncology company focused on developing therapies to activate macrophages in the fight against cancer, announced proof-of-concept data from an ongoing Phase 1b/2 trial evaluating 5F9 in combination with rituximab in patients with relapsed/refractory non-Hodgkin’s lymphoma (r/r NHL). 5F9 is a monoclonal antibody against CD47, which is designed to block the “don’t eat me” signal used by cancer cells to avoid being ingested by macrophages. The data was presented in an oral presentation at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Illinois on June 3, 2018.
As of the data cutoff of April 2018, 22 patients had been treated across all dose groups in the Phase 1b portion of the trial, including 15 patients with DLBCL and seven patients with FL. Before dosing, 95% of patients were considered refractory to a prior rituximab regimen and the median number of prior therapies was four (ranging from two to 10). 5F9 was observed to be generally well-tolerated at all doses and the maximum tolerated dose was not defined with 5F9 dosing up to 30 mg/kg. The majority of adverse events (AEs) reported by investigators were Grade 1 or 2 and the most common treatment-related AEs were expected CD47-mechansim-based effects on red blood cells (RBC), which led to a temporary and reversible anemia. Across all 22 evaluable patients, the data showed an objective response rate (ORR) of 50% and a complete response rate (CR) of 36%. In DLBCL, the ORR was 40%, with 33% of patients achieving a CR. In FL, the ORR was 71%, with 43% of patients achieving a CR.
LLS is supporting this clinical trial in collaboration with Forty Seven through its Therapy Acceleration Program® (TAP), a strategic initiative to partner directly with biotechnology companies to help accelerate the development of promising therapies.
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Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company focused on discovering and developing highly targeted cancer immunotherapies, reported additional preliminary patient data from two separate clinical studies of its lead NK cell engager candidate AFM13. The data demonstrate that AFM13 was well-tolerated and showed promising therapeutic efficacy both in combination with the anti-PD-1 antibody Keytruda® (pembrolizumab) in Hodgkin lymphoma (HL) and as monotherapy in CD30-positive lymphoma.
AFM13 in combination with Keytruda® in relapsed/refractory HL - Best response preliminary assessment data from 9 patients treated at the highest AFM13 dose level (7 mg/kg) as reported by central read, showed an objective response rate (ORR) of 89% (8/9), including complete metabolic responses (CmR) in 44% (4/9) and partial metabolic responses (PmRs) in 44% (4/9) of patients. One patient experienced stable disease (SD). This ORR of 89% compared favorably to the historical ORR of Keytruda (58-63%) as monotherapy in a similar patient population. Namely, these patients were R/R HL and post autologous stem cell transplantation (ASCT) or ineligible for ASCT and had failed brentuximab vedotin (BV). Importantly, the reported CR rate of 44% represents a doubled CR rate compared to previously reported anti-PD1 studies (9-22%). The combination was well-tolerated with most of the adverse events observed mild to moderate in nature and manageable with standard of care. In total, the extension cohort includes 21 patients and enrollment has recently been completed.
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Kiadis Pharma N.V. (“Kiadis Pharma”) (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing innovative T-cell therapy products aiming to make bone marrow transplantations safer and more effective for patients, announced that the last patient in the Phase II CR-AIR-008 (‘008’) trial has received a single dose of ATIR101™. This exploratory Phase II trial (clinicaltrials.gov identifier: NCT02500550) was designed to evaluate the safety and efficacy of two doses of ATIR101™ in patients with a hematologic malignancy who received a hematopoietic stem cell transplantation from a haploidentical (half-matched) donor. A total of 15 patients were recruited into the trial. Six of these patients received two doses of ATIR101™ before the independent data monitoring committee (IDMC) recommended that the trial should continue treating patients with one dose of ATIR101™ (announced on December 21, 2016). The remaining nine patients on the trial have now been treated with a single dose of ATIR101™. Of these nine patients, five patients are 1-year post treatment and results are consistent with the previously conducted 23-patient CR-AIR-007 single dose Phase II trial.
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Cancer ‘vaccine’ eliminates tumors in mice - Activating T cells in tumors eliminated even distant metastases in mice, Stanford researchers found. The researchers believe the local application of very small amounts of the agents could serve as a rapid and relatively inexpensive cancer therapy that is unlikely to cause the adverse side effects often seen with bodywide immune stimulation. “When we use these two agents together, we see the elimination of tumors all over the body,” said Ronald Levy, MD, professor of oncology. “This approach bypasses the need to identify tumor-specific immune targets and doesn’t require wholesale activation of the immune system or customization of a patient’s immune cells.” One agent is currently already approved for use in humans; the other has been tested for human use in several unrelated clinical trials. A clinical trial was launched in January to test the effect of the treatment in patients with lymphoma. Levy, who holds the Robert K. and Helen K. Summy Professorship in the School of Medicine, is the senior author of the study, which was published Jan. 31 in Science Translational Medicine. Instructor of medicine Idit Sagiv-Barfi, PhD, is the lead author. The research was supported by the National Institutes of Health (grant CA188005), The Leukemia & Lymphoma Society (TAP-120921), the Boaz and Varda Dotan Foundation and the Phil N. Allen Foundation.
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Kiadis Pharma N.V. (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing innovative cell-therapy products to make bone marrow transplantations safer and more effective for patients, announced that the first patient has been enrolled in the HATCY Phase 3 clinical trial for ATIR101™. The multinational trial will evaluate the safety and efficacy of ATIR101™ as an adjunctive treatment to blood stem cell transplantation from a half-matched (haploidentical) family donor compared to post-transplant cyclophosphamide (PTCy or ‘Baltimore’ protocol) in adult patients with blood cancer.
This Phase 3 trial (ClinicalTrial.gov identifier: NCT02999854; EudraCT number: 2016-004672-21; Kiadis number: CR-AIR-009; Kiadis name: HATCY) will enroll 195 patients in Europe, US and Canada. The primary objective of this randomized, open label, parallel arm Phase 3 trial is to compare safety and efficacy of a haploidentical (half-matched family donor) T-cell depleted hematopoietic stem-cell transplant (HSCT) and adjunctive treatment with ATIR101™ versus a haploidentical T-cell replete HSCT with post-transplant administration of high dose cyclophosphamide in adult patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) or myelodysplastic syndrome (MDS). The primary composite endpoint is graft-versus-host disease free and relapse-free survival (GRFS), secondary endpoints include overall survival (OS). An additional objective of the study is to compare the effect of the two treatments on quality of life and health economics.
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Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, announced that the pivotal Phase 2 trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) has met its primary endpoint. Based on feedback from the U.S. Food and Drug Administration (FDA), Stemline remains on track to begin submission of its Biologics License Application (BLA) in the 4Q17-1Q18 timeframe.
Across all stages, lines, and doses in the trial (n=45 BPDCN patients; 32 first-line, 13 relapsed/refractory), the CR rate was 60% (27/45) and the ORR was 82% (37/45). In first-line patients who received SL-401 at all tested doses (n=32 patients), the CR rate was 69% (22/32) and the ORR was 88% (28/32). 41% (13/32) of first-line patients who received SL-401 at all tested doses have been bridged to SCT following remission on SL-401.
SL-401 has been granted Breakthrough Therapy Designation (BTD) by the FDA for the treatment of BPDCN, and Orphan Drug Designation (ODD) by the FDA and EU for the treatment of patients with BPDCN and acute myeloid leukemia (AML). The study (NCT02113982) is sponsored in part by The Leukemia & Lymphoma Society's Therapy Acceleration Program®.
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The Leukemia & Lymphoma Society (LLS) hails the U.S. Food and Drug Administration (FDA) approval of a new, personalized cell therapy that supercharges the patient’s immune system to find and kill cancer cells. The therapy, axicabtagene ciloleucel (Yescarta™), is the second of this new method of treatment, known as CAR (chimeric antigen receptor) T-cell immunotherapy, to receive FDA approval for blood cancer patients in the past six weeks, and the first to treat patients with lymphoma.
LLS has supported the clinical trial leading to the approval of Yescarta since 2015 through its Therapy Acceleration Program® (TAP). Through TAP, LLS partners directly with biotechnology companies, including the developer of Yescarta, Kite, a Gilead Company, to help accelerate the development of promising treatments.
The FDA’s approval of Yescarta is for treatment of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), the most common type of non-Hodgkin lymphoma (NHL), and several more rare types including primary mediastinal large B-cell lymphoma (PMBCL), high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma (transformed follicular lymphoma or tFL).
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NexImmune and The Leukemia & Lymphoma Society (LLS) have entered a partnership to advance the Company’s novel cellular immunotherapy into Phase 1 clinical testing for patients with high risk AML who have relapsed after an allogeneic hematopoietic stem cell transplant (HSCT). NexImmune is advancing immunotherapy products based on the Company’s proprietary Artificial Immune Modulatory (AIM) nanotechnology platform. The AIM technology enables simultaneous enrichment, expansion and activation of cytotoxic T cells directed at multiple tumor-relevant antigen targets (Tumor Associated Antigens or neo-antigens) across a broad range of both solid and hematologic malignancies. The Company’s lead product candidate, AIM ACT, is designed to generate T cells targeted against multiple tumor antigens associated with AML. The Company has completed Pre-IND discussions with the FDA, and expects to submit an IND in 1HQ2018.
The partnership is through LLS's Therapy Acceleration Program® (TAP), through which LLS forges collaborations with early stage biotechnology companies to help bring innovative therapies to patients faster. LLS has made an undisclosed investment in NexImmune to help support the overall clinical development of the product.
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Selvita S.A. (WSE: SLV) and The Leukemia & Lymphoma Society (LLS) announced a partnership to co-fund further preclinical and clinical development of a targeted therapy to treat patients with acute myeloid leukemia (AML). Selvita has discovered and is developing SEL120, a therapy that targets the cyclin-dependent kinase 8 (CDK8) protein, which plays a unique and critical role in gene regulation. SEL120 has shown efficacy in treating AML cells both in vitro and in vivo. Under the terms of the agreement, LLS will provide up to $3.25 million funding over four years, through its Therapy Acceleration Program® (TAP), in order to help fund further SEL120 IND-enabling studies and a phase I trial in AML.
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The Leukemia & Lymphoma Society (LLS) applauds the U.S. Food and Drug Administration (FDA) approval of CPX-351 (Vyxeos), an innovative combination of two chemotherapy drugs being used to treat two high-risk subgroups of acute myeloid leukemia (AML) patients. After more than four decades of little progress in treating AML, this approval marks the third drug approval for AML patients in just the past three months. LLS contributed sustained and significant support to advance this therapy.
LLS has funded the development of Vyxeos since 2009 through its Therapy Acceleration Program® (TAP). Under the TAP initiative, LLS partners directly with biotechnology companies to accelerate the development of promising therapies for unmet medical needs, many of which otherwise might not receive support. LLS partnered with Celator Pharmaceuticals, the developer of Vyxeos, through TAP; Celator was acquired by Jazz Pharmaceuticals last year.
The FDA approved Vyxeos to treat newly diagnosed adult AML patients who acquire AML after being treated for a previous cancer, a complication that occurs in approximately 8-10 percent of patients treated for cancer. Vyxeos also is approved for patients whose AML developed after progressing from a diagnosis of myelodysplastic syndrome, another form of blood cancer.
“The vision for our innovative Therapy Acceleration Program® has become a reality with this approval, which will help patients with high-risk AML who are in desperate need of more options,” said Louis J. DeGennaro, LLS president and CEO. “From the start, LLS recognized the potential of Vyxeos, and we are very gratified by this opportunity to deliver better treatments and outcomes for patients with high-risk AML. In its 10th year, TAP is demonstrating its effectiveness at accelerating cancer treatments, as Vyxeos is the first TAP-supported therapy approved by the FDA, a significant achievement for LLS’s venture philanthropy model. More LLS-supported therapies are currently under FDA review.”
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Kite Pharma, Inc. (Nasdaq:KITE), announced that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for axicabtagene ciloleucel as a treatment for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) who are ineligible for autologous stem cell transplant. This application represents the first chimeric antigen receptor (CAR) T-cell therapy submitted to the EMA. Axicabtagene ciloleucel is currently under review by the U.S. Food and Drug Administration (FDA), and the FDA has set a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2017.
The MAA for axicabtagene ciloleucel is supported by data from the ZUMA-1 trial, which met the primary endpoint of objective response rate (ORR), with 82 percent (p < 0.0001) of patients achieving a response after a single infusion of axicabtagene ciloleucel. At a median follow-up of 8.7 months, 44 percent of patients were in ongoing response, which included 39 percent of patients in complete response (CR). The most common Grade 3 or higher adverse events included cytokine release syndrome and neurologic events, which were generally reversible.
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Kite Pharma, Inc. (Nasdaq:KITE), highlighted the recent online publication of results in Molecular Therapy from a National Cancer Institute (NCI) study of anti-CD19 chimeric antigen receptor (CAR) T-cell therapy in patients with aggressive NHL including diffuse large B-cell lymphoma (DLBCL). Nine patients with chemorefractory aggressive NHL were treated with a single dose of anti-CD19 CAR T cells with a CD28 co-stimulatory domain. Seven of the 9 patients were evaluable for response. Complete remissions (CR) were observed in 5 of the 7 evaluable patients. Four of the 5 CRs are ongoing from 38 to 56+ months after treatment. There were no chronic toxicities attributable to CAR T cells except B-cell aplasia and hypogammaglobulinemia. Importantly, 3 of 4 patients in ongoing CR had recovery of normal polyclonal B cells, showing that durable CRs can be maintained in the absence of continued activity of anti-CD19 CAR T cells. The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program® supported by funding in part Kite Pharma's ZUMA-1 Phase 2 clinical trial which was based on the inital trial performed pursuant to a Cooperative Research and Development Agreement (CRADA) between NCI and Kite.
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Jazz Pharmaceuticals plc (Nasdaq: JAZZ) announced that the U.S. Food and Drug Administration (FDA) has accepted for filing with Priority Review its recently submitted New Drug Application (NDA) for VYXEOS™ (cytarabine and daunorubicin) liposome injection, an investigational treatment for acute myeloid leukemia (AML), a rapidly progressing and life-threatening blood cancer.
Priority Review status is designated for drugs that may offer major advances in treatment or provide a treatment where no adequate therapy exists. The granting of Priority Review for the VYXEOS NDA accelerates the timing of the FDA review of the application compared to a standard review. The NDA submission includes clinical data from five studies, including the pivotal Phase 3 study.
The study was conducted in partnership with The Leukemia & Lymphoma Society (LLS) through its Therapy Acceleration Program® (TAP), which has supported the clinical development of VYXEOS beginning in Phase 2.
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Kite Pharma, Inc., (Nasdaq:KITE) announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for axicabtagene ciloleucel. The submission follows positive data demonstrated with a single infusion of axicabtagene ciloleucel in the ZUMA-1 Phase 2 trial in patients with refractory aggressive non-Hodgkin lymphoma (NHL). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of November 29, 2017. The filing acceptance is supported by data from the ZUMA-1 Phase 2 trial which met the primary endpoint of objective response rate (ORR) recorded after a single infusion of axicabtagene ciloleucel with 82 percent (p < 0.0001). At a median follow-up of 8.7 months, 44 percent of patients were in ongoing response, which included 39 percent of patients in complete response (CR). In December 2015, axicabtagene ciloleucel received Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for DLBCL, TFL, and PMBCL. The company expects to submit its Market Authorization Application (MAA) of axicabtagene ciloleucel with the European Medicines Agency (EMA) in the third quarter of 2017. ZUMA-1 is supported in part by funding from The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program®.
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Kiadis Pharma N.V. (“Kiadis Pharma”) (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing innovative products to make bone marrow transplantations for patients suffering from blood cancers and inherited blood disorders safer and more effective, today announces it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its lead product, ATIR101™. Kiadis Pharma is seeking marketing approval in the European Union for ATIR101™ as an adjunctive treatment in haematopoietic stem cell transplantation (HSCT) for malignant disease.
The filing is based on Kiadis Pharma’s existing clinical data and follows positive interactions with the EMA Rapporteur and Co-Rapporteur which indicated support for the filing using the Company’s single dose Phase II trial with ATIR101™ as the pivotal study. Kiadis Pharma submitted the application under the European Union’s centralized procedure, which permits the agency to issue a single marketing authorization that is valid across all EU countries.
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The Leukemia & Lymphoma Society (LLS) continues to advance the promising field of immunotherapy research, harnessing the body’s own immune system to fight cancer, with a $4 million funding commitment in an investigational therapy being developed by Forty Seven Inc. for lymphoma patients. The novel drug will be tested in combination with the FDA-approved rituximab, already part of standard treatment for several types of NHL. The therapy is directed against CD47, a protein that provides a “don’t eat me” signal to the immune system and blocks the ability of immune cells called macrophages to devour those cancer cells. The combination Hu5F9-G4 and rituximab displayed synergy in preclinical animal models of NHL. Forty Seven was founded in 2015 by Stanford University researchers Irv Weissman, M.D., and Ravi Majeti, M.D., Ph.D., both of whom have been recipients of LLS grants supporting their early work targeting CD47. Forty Seven has licensed the therapy from Stanford.
LLS is supporting the collaboration through its Therapy Acceleration Program ® (TAP), a strategic initiative to partner directly with biotechnology companies to help accelerate the development of promising therapies. There are currently 16 projects supported through LLS’s TAP portfolio.
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Kite Pharma, Inc., (Nasdaq:KITE) announced two plenary presentations of positive data from the primary analysis of ZUMA-1 for its lead CAR-T candidate, axicabtagene ciloleucel, in patients with refractory aggressive B-cell non-Hodgkin lymphoma (NHL) at the 2017 American Association of Cancer Research Annual Meeting in Washington, D.C. Both presentations were given by Frederick L. Locke, M.D., the ZUMA-1 Co-Lead Investigator, and Director of Research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida. The study met the primary endpoint of objective response rate (ORR) recorded after a single infusion of axicabtagene ciloleucel, with 82 percent (p < 0.0001). These results demonstrate the treatment effect of axicabtagene ciloleucel in diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL), which are types of aggressive NHL.
The following table shows response data including the month 6 ORR and CR as well as ongoing response rates at the primary analysis data cut-off.
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DLBCL (n=77) |
TFL/PMBCL (n=24) |
Combined (n=101) | |||
| ORR (%) | CR (%) | ORR (%) | CR (%) | ORR (%) | CR (%) | |
| ORR | 82 | 49 | 83 | 71 | 82 | 54 |
| Month 6 | 36 | 31 | 54 | 50 | 41 | 36 |
| Ongoing | 36 | 31 | 67 | 63 | 44 | 39 |
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Jazz Pharmaceuticals plc (Nasdaq: JAZZ) announced the completion of a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of Vyxeos™ (cytarabine and daunorubicin) liposome for injection, an investigational treatment for acute myeloid leukemia (AML), a rapidly progressing and life-threatening blood cancer. The company has requested a priority review for the Vyxeos NDA, which, if granted, would accelerate the expected timing of the FDA's review. The NDA submission includes clinical data from five studies, including the pivotal Phase 3 study. Data from the Phase 3 study, which met its primary endpoint, were previously presented at the American Society of Clinical Oncology Annual Meeting in June 2016. The study was conducted in partnership with The Leukemia & Lymphoma Society® (LLS) through its Therapy Acceleration Program® (TAP), which has supported the clinical development of VYXEOS beginning in Phase 2.
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Kite Pharma, Inc. (Nasdaq:KITE) announced that it has completed the rolling submission with the U.S. Food and Drug Administration (FDA) of the Biologics License Application (BLA) for axicabtagene ciloleucel (previously known as KTE-C19) as a treatment for patients with relapsed or refractory aggressive non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant (ASCT). In December 2015 axicabtagene ciloleucel received Breakthrough Therapy Designation (BTD) by the FDA for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL). If approved, Kite plans to commercially launch axicabtagene ciloleucel in 2017. Kite is also planning a regulatory submission to the European Medicines Agency (EMA) for axicabtagene ciloleucel in 2017.
"The Leukemia & Lymphoma Society (LLS) applauds Kite for achieving this significant milestone and bringing this promising therapy closer to patients with lymphoma who desperately need new options," said Louis J. DeGennaro, Ph.D., LLS President and Chief Executive Officer. "LLS has supported companies that are working to dramatically change cancer treatment through the development of immunotherapy for the past two decades, and we immediately recognized the great opportunity to support Kite's CAR-T program in 2015 through our Therapy Acceleration Program ® (TAP). Partnerships created through TAP, now in its tenth year, have the potential to bring several breakthrough therapies, such as axicabtagene ciloleucel, to patients in the coming year."
The ZUMA-1 pivotal trial for axicabtagene ciloleucel for the treatment of patients with aggressive NHL was supported in part by funding from LLS' TAP.
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Kiadis Pharma N.V. (Euronext Amsterdam and Brussels: KDS) announced that the Pediatric Committee (PDCO) of the European Medicines Agency (EMA) has accepted the Company’s Pediatric Investigation Plan (PIP) for ATIR101™ for the adjunctive treatment in hematopoietic stem cell transplantation (HSCT) for a malignant disease. In addition, the PDCO has agreed that the Company may defer conducting the studies defined in the PIP until after it files a Marketing Authorization Application (MAA) in Europe for the use of ATIR101™ for the treatment of blood cancers. The PIP provides for a Phase II trial to evaluate the safety and efficacy of ATIR101™ as an adjunctive treatment on top of an HSCT in pediatric patients up to 18 years of age with a hematologic malignancy, who are eligible for an HSCT but without the availability of a fully matched donor. Patients will receive either an HSCT from a partially matched (haploidentical) related family donor with the adjuvant infusion of ATIR101™, or an HSCT using umbilical cord blood stem cells from an unrelated donor. The primary endpoint of the trial will be Graft versus Host-Disease-free, relapse-free survival (GRFS).
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Stemline Therapeutics, Inc. (Nasdaq:STML) announced that the enrollment of Stage 3 in the SL-401 pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN) has been completed. Stage 3 enrolled 13 first-line BPDCN patients; statistical analysis will be based on evaluable first-line patients. The registration pathway was previously agreed upon with the U.S. Food and Drug Adminstration (FDA). Depending on the data from the trial, Stemline plans to use the results generated to support the potential filing of a Biologics License Application (BLA) for full approval in first-line BPDCN, and is targeting possible BLA filing in the second half of this year. This multicenter, pivotal trial has enrolled 47 BPDCN patients at seven centers in the U.S. The 47 BPDCN patients were comprised of 32 first-line patients (29 dosed at 12 ug/kg/day) and 15 relapsed/refractory patients dosed at 12 ug/kg/day. Stemline plans to provide a clinical update on patients enrolled in Stages 1 and 2 at a medical conference around mid-year, with top-line data from Stage 3 expected in the second half of 2017. To ensure ongoing patient access to SL-401, Stemline plans to continue to enroll both first-line and relapsed/refractory BPDCN patients under the current protocol. SL-401 received Breakthrough Therapy Designation (BTD) by the FDA in August 2016.
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Kiadis Pharma N.V. (Euronext Amsterdam and Brussels: KDS) provided a progress update on the clinical and regulatory status of ATIR101™, the Company’s lead product to address the key risks and limitations of hematopoietic stem cell transplantation (HSCT) in blood cancer. Following regulatory approval from the national authority in Canada (Health Canada), the company announced the initiation of its randomized, controlled, transatlantic Phase III trial with ATIR101™ (CR-AIR-009). Approximately 195 patients with acute leukemia will be enrolled in total in the Phase III trial and randomized 1:1 to receive a haploidentical allogeneic HSCT using either the Kiadis Pharma approach with a single dose of ATIR101™ or the post-transplant cyclophosphamide approach (also known as the Baltimore Protocol). The trial protocol has also been submitted for approval and is being evaluated by the United States FDA (Food and Drug Administration) as well as several European regulatory authorities. Following approval the trial will be rolled out to additional study centers in the United States and Europe.
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Stemline Therapeutics, Inc. (Nasdaq:STML) announced an agreement with the U.S. Food and Drug Administration (FDA) on the registration pathway for SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN). To support the filing of a Biologics License Application (BLA) for full approval in first-line BPDCN, Stemline is currently enrolling an additional small cohort, planned for 8-12 first-line BPDCN patients, into its ongoing Phase 2 trial. To date, approximately half of these new patients are enrolled into the study, with full enrollment expected this quarter. Stemline intends to file a BLA in 2H17, which is anticipated to undergo an expedited review given SL-401's Breakthrough Therapy Designation. If successful, Stemline projects a commercial launch of SL-401 in 2018.
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Kite Pharma, Inc. (Nasdaq:KITE) presented results from the ZUMA-1 trial of axicabtagene ciloleucel (KTE-C19) in patients with chemorefractory aggressive non-Hodgkin lymphoma (NHL) in two oral presentations at the American Society of Hematology (ASH) 58th Annual Meeting in San Diego, California.
ZUMA-1 enrolled 111 patients with diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL), or transformed follicular lymphoma (TFL). Patients were required to have chemorefractory disease, defined as progressive or stable disease as best response to last line of therapy, or disease progression ≤12 months after autologous stem cell transplant. Manufacturing was successful for 110 patients, and 101 patients were treated. The pre-specified interim analysis was triggered when 51 patients with DLBCL had a minimum of three months of follow-up. At the time of interim analysis, 11 patients with PMBCL/TFL had been followed for three months. An additional 31 patients with one month of follow-up were included in the late breaker presentation.
ZUMA-1 met the primary endpoint of objective response rate (ORR), p < 0.0001. Response rates by disease subtype are shown in the table below. Responses were observed across key subgroups, including 75 percent CR in patients who relapsed in ≤12 months after autologous stem cell transplant and 47 percent CR in patients refractory to second line or later chemotherapy. At the month three assessment, 39 percent of patients were in CR.
| Subgroup | N | ORR | CR |
| DLBCL | 51 | 76% | 47% |
| TFL/PMBCL | 11 | 91% | 73% |
| Total | 62 | 79% | 52% |
The primary analysis of ZUMA-1 will include a minimum of 6 months of follow-up. Kite intends to seek regulatory approval of axicabtagene ciloleucel in refractory aggressive NHL and plans to complete its rolling submission of the Biologics License Application (BLA) in the first quarter of 2017.
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Stemline Therapeutics, Inc. (Nasdaq:STML) announced the oral presentation of positive clinical data from its ongoing SL-401 Phase 2 potentially pivotal clinical trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN) at the 2016 American Society of Hematology (ASH) Annual Meeting, being held at the San Diego Convention Center in San Diego, CA.
The Phase 2 BPDCN data presented at ASH cover 32 evaluable adult BPDCN patients treated with SL-401. Results demonstrated that SL-401 produced a 100% (16/16) overall response rate (ORR), including an 81% (13/16) complete response (CR) rate in first-line BPDCN patients treated at the recommended dose of 12 ug/kg/day and a 95% (18/19) ORR in first-line patients treated at the recommended dose or lower. In relapsed/refractory patients, the ORR was 69% (9/13) with a CR rate of 31% (4/13).
| Line of Therapy | First-line | First-line | R/R | All lines |
| Dose Group | All Doses | 12 ug/kg | 12 ug/kg | All Doses |
| n (evaluable/total) | 19 | 16 | 13 | 32 |
| ORR | 18/19 (95%) | 16/16 (100%) | 9/13 (69%) | 27/32 (84%) |
| CR*, n (rate) | 14 (74%) | 13 (81%) | 4 (31%) | 18 (56%) |
| Bridged to SCT, n | 6 | 6 | 1 | 7 (22%) |
| (allo+auto) | (3+3) | (3+3) | (1+0) | (4+3) |
Response duration data continue to appear promising, with 69% (11/16) first-line BPDCN patients treated at 12 ug/kg/day remaining relapse-free (range: 1+ to 20+ months, ongoing). In the relapsed/refractory setting, 46% (6/13) patients are relapse-free (range: 1+ to 7+ months). The median progression-free and overall survival for first-line has not been reached and for relapsed/refractory it is currently 8.5 months.
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Kiadis Pharma N.V. (Euronext Amsterdam and Brussels: KDS) presented positive one-year data with its lead product ATIR101™ from the single dose Phase II trial (NCT01794299/EudraCT 2012-004461-41) at the 58th Annual Meeting and Exposition of the American Society of Hematology (ASH) in San Diego, United States of America.
The data presented at ASH confirms that ATIR101™ can be safely infused and shows a significant reduction in Transplant Related Mortality (TRM) (primary endpoint) and a significant improvement in Overall Survival (OS) (secondary endpoint) in comparison to an observational control group of patients undergoing a T-cell depleted haploidentical donor transplantation only. Combined with the lack of severe Graft-versus-Host-Disease (GVHD) and limited relapse, this translates into favorable GVHD-free, relapse-free survival (GRFS) one-year post-transplantation. Based on the positive results from this Phase II trial, the Company is proceeding with the development of ATIR101™ by initiating a Phase III trial in which patients with acute leukemia will be randomized to receive a haploidentical HSCT according to either the PTCy approach or the Kiadis Pharma approach with a single dose of ATIR101™. This Phase III trial has been submitted to regulatory authorities and is currently under review for approval.
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Kite Pharma, Inc. (Nasdaq:KITE) announced that it has initiated the rolling submission with the U.S. Food and Drug Administration (FDA) of the Biologics License Application (BLA) for KTE-C19 as a treatment for patients with relapsed/refractory aggressive B-cell non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant (ASCT). The pivotal ZUMA-1 study supporting this submission enrolled patients with chemorefractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL), three subtypes of aggressive NHL. The company expects to complete its BLA submission by the end of the first quarter of 2017.
Kite also announced that the United States Adopted Name, or USAN, for KTE-C19 will be axicabtagene ciloleucel. Axicabtagene ciloleucel (KTE-C19) received Breakthrough Therapy Designation (BTD) by the FDA in December 2015. If approved, Kite plans to commercially launch KTE-C19 in 2017.
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Acetylon Pharmaceuticals announced that it has entered into an agreement to be acquired by Celgene Corporation. The acquisition will provide Celgene with, among other things, worldwide rights to Acetylon’s selective HDAC6 inhibitor programs and intellectual property in oncology, neurodegeneration, and autoimmune disease, including its lead drug candidates citarinostat (ACY-241) and ricolinostat (ACY-1215).
Since May 2011, The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program® has provided funding to help support Acetylon’s oral selective HDAC6 inhibitor drug candidates, ACY-1215 and ACY-241, in multiple myeloma.
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Oncopep Inc. announced the initiation of a Phase 1b clinical trial evaluating PVX-410, a multi-peptide therapeutic cancer vaccine, in patients with moderate or high-risk for progression smoldering multiple myeloma (SMM), an asymptomatic precursor to multiple myeloma, which is a cancer of the plasma cells. The study, led by Noopur Raje, M.D. at Massachusetts General Hospital, will assess the safety and tolerability of PVX-410 in combination with durvalumab with or without lenalidomide. The trial initiation follows the successful completion of a Phase 1/2a dose escalation study of PVX-410 in patients with SMM, the results of which will be presented at the 58th Annual Meeting of the American Society of Hematology (ASH), being held from December 3-6, 2016 in San Diego, California.
In August of 2014, The Leukemia & Lymphoma Society (LLS) participated in the Series B financing which included participation from angel groups, family foundations and individuals. LLS’ decision to participate in the financing round resulted from a review of OncoPep’s application for funding from LLS' Therapy Acceleration Program®.
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The University of Texas MD Anderson Cancer Center’s Institute for Applied Cancer Science (IACS) has initiated the first clinical study of a novel drug designed to starve cancer cells, IACS-10759. The study will enroll patients with acute myeloid leukemia (AML) and is supported by a $3.5 million investment from The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program®.
“LLS is going on the offensive against AML, a disease that has seen little change in the standard of care in more than 40 years,” said Lee Greenberger, Ph.D., LLS’s chief scientific officer. “Through our Beat AML initiative and other promising projects, such as this very innovative approach being developed by the MD Anderson team, we are hoping to change the paradigm of treatment for this deadly disease.”
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Kite Pharma, Inc., (Nasdaq:KITE) announced positive topline results from a pre-planned interim analysis of ZUMA-1 for its lead product candidate, KTE-C19, in patients with chemorefractory diffuse large B-cell lymphoma (DLBCL). KTE-C19 met the primary endpoint of objective response rate (ORR), p < 0.0001, with ORR of 76 percent, including 47 percent complete remissions (CR). ZUMA-1 enrolled patients with chemorefractory aggressive NHL into two cohorts. Cohort 1 included patients with DLBCL, and Cohort 2 enrolled patients with transformed follicular lymphoma (TFL) and primary mediastinal B-cell lymphoma (PMBCL). Kite's intent is to seek regulatory approval of KTE-C19 in DLBCL, TFL and PMBCL based upon the combined data of both cohorts.
"ZUMA-1 enrolled patients with chemorefractory aggressive NHL, a disease that is very difficult to treat. The combined CR rate of 39 percent at three months is very exciting as it represents nearly a five-fold increase from the CR rate of 8 percent seen in the SCHOLAR-1 study in a similar patient population," said Jeff Wiezorek, M.D., Senior Vice President of Clinical Development. "ZUMA-1 is the largest CAR-T study reported in NHL. We were able to manufacture KTE-C19 for 99 percent of patients enrolled in the study, and successfully handle the study logistics and adverse event management at over 20 sites, most of which had no prior experience in CAR-T therapy."
Additional data from this interim analysis will be submitted for presentation at an upcoming scientific meeting. The primary analysis of 101 patients with chemorefractory aggressive NHL (DLBCL, TFL and PMBCL) will include approximately six months of follow-up and is expected in the first quarter of 2017. ZUMA-1 is supported in part by funding from The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program®.
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Kiadis Pharma N.V. (Euronext Amsterdam and Brussels: KDS) announced that its partnership with The Leukemia & Lymphoma Society (LLS) has been strengthened with a second equity investment by LLS into the development of Kiadis Pharma’s lead product ATIR101™. LLS initially invested approximately US $1 million in February 2016 through an equity investment via its Therapy Acceleration Program®, a strategic initiative to partner directly with biotechnology companies to help accelerate the development of promising therapies. This second investment will now also be used to finance Kiadis Pharma’s second ongoing Phase II trial in leukemia patients which the Company then intends to continue into a randomized controlled Phase III pivotal study on track to start later this year. The ongoing Phase II trial is investigating the repeated dosing of ATIR101™ as an adjunctive treatment to a T-cell depleted haploidentical hematopoietic stem cell transplantation (HSCT) (donor cells from a half-matched related donor) in adult patients with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL). The trial (CR-AIR-008; NCT02500550 / EudraCT 2015-002821-20) is conducted under an IND of the United States Food and Drug Administration and is set up to enroll patients in the United States as well as other countries, including Canada, Belgium and the United Kingdom.
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Kite Pharma, Inc. (Nasdaq: KITE) announced that it has completed enrollment of 72 patients in DLBCL cohort in the Phase 2 portion of ZUMA-1. Kite looks forward to announcing top-line data from the first 50 DLBCL patients in the ZUMA-1 study, and, subject to these results, plan on submitting a Biologics License Application (BLA) with the U.S. Food and Drug Administration by the end of 2016.
The Leukemia & Lymphoma Society (LLS) and Kite Pharma entered into a partnership in July 2015 to enhance the development of Kite's lead product candidate, KTE-C19, for the treatment of patients with refractory aggressive non-Hodgkin lymphoma (NHL). LLS will launch a broad scope educational program focusing on CAR T-cell therapy for the treatment of blood cancers, as well as support outreach for clinical trial enrollment. LLS also will contribute up to $2.5 million, through the Therapy Acceleration Program®, to help fund Kite's ongoing Phase 1/2 clinical study of KTE-C19 (ZUMA-1; NCT02348216), which is designed to evaluate safety and efficacy in the treatment of patients with refractory diffuse large B cell lymphoma (DLBCL), as well as two rare lymphomas - primary mediastinal B cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL).
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Affimed N.V. (Nasdaq:AFMD) has reported that the Company’s IND application for a Phase1b AFM13/ KEYTRUDA® (pembrolizumab) combination study in HL patients relapsed or refractory to chemotherapy, including Adcetris™ (NCT02665650), has recently been accepted by the FDA and is now active. The first sites have been opened and the study is recruiting. The Company anticipates providing a first update on the study by the end of 2016 or in the first quarter of 2017.
The Leukemia & Lymphoma Society (LLS) and Affimed N.V. entered into a partnership to co-fund a phase 2 trial with the Recruit-TandAb AFM13, a novel tetravalent bispecific antibody directed against human CD30 and CD16A in Hodgkin Lymphoma (HL) patients for whom currently available treatments have failed. AFM13 is a first-in-class immunotherapy drug designed to treat HL patients and patients with CD30-positive malignancies. LLS has committed to investing up to $4.4 million over 2 years to support the project.
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Kite Pharma, Inc. (Nasdaq:KITE) ("Kite") presented via a poster presentation at the 2016 American Society of Clinical Oncology (ASCO) annual meeting the updated durability of complete responses in the Phase 1 portion of the ZUMA-1 trial. "Three reported complete remissions in patients with chemorefractory DLBCL after a single treatment with CAR T-cell therapy are still ongoing at nine months. This is remarkable given that single-digit complete response rates are historically observed in patients who do not respond to chemotherapy," said Sattva S. Neelapu, Associate Professor and Director of Translational Research, Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center. "These results are extremely important as CAR engineered T-cells have the potential to transform the treatment landscape for chemorefractory DLBCL."
The Phase 1 portion of ZUMA-1 treated a total of 7 patients with chemorefractory DLBCL. The results showed that treatment with KTE-C19 achieved rapid and durable responses in patients with chemorefractory disease (objective response rate 71%, complete response rate 57%). Ongoing complete responses were observed in 3 patients after nine months of follow-up. KTE-C19 related adverse events consisted predominantly of cytokine release syndrome (CRS) and neurotoxicity, which were generally reversible. Grade 3 or higher CRS was observed in 14% and neurotoxicity in 57%; all were reversible except in one patient with dose-limiting toxicity.
The Leukemia & Lymphoma Society (LLS) and Kite Pharma, Inc. entered into a partnership to enhance the development of Kite's lead product candidate, KTE-C19, for the treatment of patients with refractory aggressive non-Hodgkin lymphoma (NHL). Under the collaboration, LLS will launch a broad scope educational program focusing on CAR T-cell therapy for the treatment of blood cancers, as well as support outreach for clinical trial enrollment. LLS also will contribute up to $2.5 million, through the Therapy Acceleration Program®, to help fund Kite's ongoing Phase 1/2 clinical study of KTE-C19 (NCT02348216).
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Stemline Therapeutics, Inc. (Nasdaq:STML) presented via an oral presentation at the 2016 American Society of Clinical Oncology (ASCO) annual meeting the positive clinical data from its ongoing SL-401 Phase 2 potentially pivotal clinical trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN). Results demonstrate that SL-401 produced an 89% (17/19) overall response rate (ORR) in BPDCN, with a 100% (12/12) ORR in first-line patients and a 71% (5/7) ORR in relapsed/refractory patients. In 12 evaluable first-line patients (all doses), there were 9 complete responses (CR) and 2 clinical complete responses (CRc). CRc is defined as a CR in non-skin affected organs with marked gross clearance of skin lesions and residual microscopic skin disease. In the 10 evaluable first-line patients treated at 12 ug/kg/day, the CR/CRc rate was 100% (8 CR and 2 CRc). In the 7 evaluable relapsed/refractory BPDCN patients, including one treated on a compassionate use basis, the ORR rate was 71%, which included 1 CR and 1 CRc (29% CR/CRc rate) and 3 partial responses (PR). The study (NCT02113982) is sponsored in part by The Leukemia & Lymphoma Society's Therapy Acceleration Program®. Please click here for a video to hear from Dr. Naveen Pemmaraju, where he discusses early results from an ongoing trial of SL-401 in patients with the rare, aggressive malignancy BPDCN.
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Kiadis Pharma N.V. (Euronext Amsterdam and Brussels: KDS) announced a regulatory strategy update that, based on positive Phase II data, it has taken the decision to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its lead product ATIR101™ for use in blood cancers to reduce relapse rates, Transplant Related Mortality (TRM) and Graft-versus-Host-Disease (GVHD) in the context of a hematopoietic stem cell transplantation using a haploidentical donor. The Company will now start compiling an MAA document and anticipates submitting the application to EMA in Q1, 2017.
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Kite Pharma, Inc. (Nasdaq:KITE) announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) has granted access to its newly established Priority Medicines (PRIME) regulatory initiative for KTE-C19 in the treatment of patients with refractory diffuse large B-cell lymphoma (DLBCL). PRIME provides early and enhanced regulatory support to optimize regulatory applications and speed up the review of medicines that address a high unmet need. Access to the Priority Medicines initiative is granted by the EMA to support the development and accelerate the review of new therapies to treat patients with unmet medical need. The criteria for the Priority Medicines initiative require early clinical evidence that the therapy offers a therapeutic advantage over existing treatments or benefits patients without treatment options. This designation provides appointment of a rapporteur, early dialogue and scientific advice at key development milestones, and the potential to qualify products for accelerated review earlier in the application process. The U.S. Food and Drug Administration granted Breakthrough Therapy Designation to KTE-C19 for the treatment of patients with DLBCL, primary mediastinal B-cell lymphoma (PMBCL), and transformed follicular lymphoma (TFL) in December 2015.
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Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Celator Pharmaceuticals, Inc. (Nasdaq: CPXX) announced on May 31 2016 that they have entered into a definitive agreement for Jazz Pharmaceuticals to acquire Celator for $30.25 per share in cash, or approximately $1.5 billion. The transaction is expected to close in the third quarter of 2016.
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Celator Pharmaceuticals, Inc. announced that the United States Food and Drug Administration (FDA) granted Breakthrough Therapy designation to VYXEOS (also known as CPX-351). VYXEOS is an investigational product in development as a treatment for AML and other blood cancers. The Breakthrough Therapy designation is primarily based upon the positive results from the pivotal Phase 3 clinical trial in older patients with previously untreated high-risk (secondary) AML. The designation is for the treatment of adults with therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC). This designation includes the patient populations enrolled in the Phase 3 clinical trial. The Phase 3 trial met its primary endpoint demonstrating a statistically significant improvement in overall survival. Data will be presented at the American Society of Clinical Oncology (ASCO) 2016 Annual Meeting on Saturday, June 4th. FDA awards Breakthrough Therapy designation in order to expedite the development and review of new medicines that are intended to treat serious or life-threatening diseases when the therapy has demonstrated substantial improvement over available therapies on at least one clinically significant endpoint or when there is significant unmet medical need. Celator plans to submit a New Drug Application (NDA) to the FDA by the end of the third quarter of 2016.
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Kiadis Pharma N.V. announced positive results on the primary endpoint of its single dose Phase II trial (NCT01794299/EudraCT 2012-004461-41) with its lead product ATIR101™ at the 42nd Annual Meeting of the European Society of Blood and Marrow Transplantation (EBMT) in Valencia, Spain. The data confirms that ATIR101™ can be safely infused, does not cause grade III-IV Graft-versus-Host-Disease (GVHD) and shows a significant reduction in Transplant Related Mortality (TRM) and a significant improvement in Overall Survival (OS) in comparison to a historical control group of patients undergoing a T-cell depleted haploidentical donor transplantation only. Based on the positive results from this Phase II trial, the Company will proceed with the development of ATIR101™ as an adjunctive immuno-therapeutic treatment to a haploidentical HSCT for patients with acute leukemia, initiating a randomised Phase III trial in the second half of 2016.
Kiadis previously entered into a partnership with The Leukemia & Lymphoma Society (LLS) under which LLS is investing via its Therapy Acceleration Program® (TAP) through an equity investment of approximately $1 million for the Phase II development investigating the repeated dosing of ATIR101™. This ongoing Phase II trial (CR-AIR-008; NCT02500550) is set up to enroll patients in the United States as well as other countries, including Canada, Belgium and the United Kingdom.
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Celator Pharmaceuticals, Inc. announced positive results from the Phase 3 trial of VYXEOS™ (cytarabine: daunorubicin) Liposome for Injection (also known as CPX-351) in patients with high-risk (secondary) acute myeloid leukemia (AML) compared to the standard of care regimen of cytarabine and daunorubicin known as 7+3. The trial met its primary endpoint demonstrating a statistically significant improvement in overall survival. The median overall survival for patients treated with VYXEOS in the study was 9.56 months compared to 5.95 months for patients receiving 7+3, representing a 3.61 month improvement in favor of VYXEOS. The hazard ratio (HR) was 0.69 (p=0.005) which represents a 31 percent reduction in the risk of death versus 7+3. The percentage of patients alive 12 months after randomization was 41.5% on the VYXEOS arm compared to 27.6% on the 7+3 arm. The percentage of patients alive 24 months after randomization was 31.1% on the VYXEOS arm compared to 12.3% on the 7+3 arm. Based on these results the company expects to submit a New Drug Application (NDA) for VYXEOS with the U.S. Food and Drug Administration (FDA) later this year and submit a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in the first quarter of 2017. The study was conducted in partnership with The Leukemia & Lymphoma Society® (LLS) through its Therapy Acceleration Program® (TAP), which has supported the clinical development of VYXEOS beginning in Phase 2.
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Valor Biotherapeutics, LLC announced that the first patient was dosed in a phase 1 clinical study of its lead product candidate, IGN002, a novel investigational treatment for non-Hodgkin lymphoma (NHL). Valor, a joint venture between ImmunGene and Caliber Biotherapeutics, has partnered with The Leukemia & Lymphoma Society (LLS) on the pre-clinical development, manufacturing, and initial proof-of-concept clinical study of IGN002. Data from pre-clinical studies of IGN002 were presented at the American Society of Hematology meeting in December 2015.
Clinical Trials in Partnership with LLS
Both TAP and LLS research grant fundings are critical in supporting many active clinical trials at any given time. The ClinicalTrials.gov website provides an updated listing of clinical trials where LLS is listed as a collaborator. ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world. More information about the clinical trials in partnership with LLS TAP can also be found within each TAP division found below.
How TAP Works
The program is comprised of two divisions, each with designated strategies, to speed the development of blood cancer treatments:
