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First Patient Dosed in Phase 3 MORPHO Trial Evaluating Gilteritinib as Maintenance Therapy Following Hematopoietic Stem Cell Transplant in Patients with FLT3 Mutation-positive Acute Myeloid Leukemia

On August 22, 2017 Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, "Astellas") reported that the first patient was dosed in the registrational Phase 3 MORPHO trial of gilteritinib, the fourth Phase 3 trial underway in the gilteritinib clinical development program (Press release, Astellas, AUG 22, 2017, View Source [SID1234520296]). The MORPHO trial is a randomized, double-blind, placebo-controlled, multi-center trial that compares gilteritinib to placebo as maintenance therapy over a period of two years following hematopoietic stem cell transplant (HCT) in patients with FLT3 internal tandem duplication (ITD) mutation-positive (FLT3/ITD+) acute myeloid leukemia (AML) and in remission after induction therapy. The primary endpoint is relapse-free survival (RFS), and the study is being conducted in collaboration with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN).

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"We know that FLT3+ AML patients face potentially worse outcomes than those with other mutations, and while some patients may experience remission following a stem cell transplant, many unfortunately relapse," said trial investigator and BMT CTN Study co-chair Mark J. Levis, M.D., Ph.D., of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University. "Given this reality, it is exciting to study gilteritinib in patients following a stem cell transplant."

Gilteritinib has demonstrated inhibitory activity against FLT3 ITD as well as FLT3 tyrosine kinase domain (TKD), two common types of FLT3 mutations that are seen in approximately one third of patients with AML. Further, gilteritinib has also demonstrated inhibition of AXL, which is reported to be associated with therapeutic resistance.

AML is a cancer that impacts the blood and bone marrow and is most commonly experienced in older adults. According to the American Cancer Society, in 2016 there were an estimated 21,000 new cases of AML diagnosed in the United States and about 10,600 cases resulted in death.

"The initiation of the MORPHO trial is another significant milestone for Astellas and for patients as therapeutic options can be very limited for this FLT3+ AML population," said Steven Benner, M.D., senior vice president and global therapeutic area head, oncology development, Astellas. "We are committed to patients with FLT3+ AML and currently have underway four Phase 3 trials to explore the potential benefit of gilteritinib for patients suffering from such an aggressive form of blood cancer characterized by both genetic and resistance mutations."

Astellas is currently investigating gilteritinib in various AML patient populations through several Phase 3 trials, including the registrational ADMIRAL trial in relapsed/refractory FLT3+ AML.

The safety and efficacy of the agent discussed herein are under investigation and have not been established. There is no guarantee that the agent will receive regulatory approval and become commercially available for the uses being investigated. Information about pharmaceutical products (including products currently in development) which is included in this press release are not intended to constitute an advertisement or medical advice.

Blueprint Medicines to Present Updated Data from Ongoing Phase 1 Clinical Trial of BLU-554 in Patients with Advanced Hepatocellular Carcinoma at ESMO 2017 Congress and 11th ILCA Annual Conference

On August 21, 2017 Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, reported that updated data from its ongoing Phase 1 clinical trial evaluating BLU-554 in patients with advanced hepatocellular carcinoma (HCC) will be presented in oral presentations at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress on September 10, 2017 in Madrid, Spain and at the 11th International Liver Cancer Association (ILCA) Annual Conference on September 17, 2017 in Seoul, South Korea (Press release, Blueprint Medicines, AUG 21, 2017, View Source;p=irol-newsArticle&ID=2294896 [SID1234520303]). BLU-554 is a potent and highly selective inhibitor of FGFR4.

At ESMO (Free ESMO Whitepaper) and ILCA, Blueprint Medicines anticipates presenting updated safety and clinical activity data from its ongoing Phase 1 clinical trial of BLU-554. As of the most recent data cutoff date of August 18, 2017, 38 patients with FGFR4 pathway activation, as indicated by FGF19 overexpression, were evaluable for clinical activity, and an objective response rate of 16 percent (95 percent confidence interval 6-31 percent) was observed in this population.

"The preliminary BLU-554 data announced today continue to provide support for selective FGFR4 inhibition as a novel targeted treatment approach in biomarker-selected patients with hepatocellular carcinoma," said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. "As we evaluate the results for BLU-554 in the context of historical data for currently approved therapies showing response rates of less than 10 percent, we are encouraged that radiographic tumor reductions were observed in a refractory population with progressive disease and few or no remaining therapeutic options. We look forward to sharing a comprehensive update on the ongoing BLU-554 clinical trial, as well as the path forward for further development, in September."

Details of the presentations are as follows:

2017 ESMO (Free ESMO Whitepaper) Congress

Presentation Title: Phase 1 Safety and Clinical Activity of BLU-554 in Advanced Hepatocellular Carcinoma (HCC)
Session Title: Developmental Therapeutics
Date & Time: Sunday, September 10, 2017 from 5:30 – 5:45 p.m. CET (11:30 – 11:45 a.m. ET)
Abstract ID: 365O
Location: Cordoba Auditorium, IFEMA, Feria de Madrid

11th ILCA Annual Conference

Presentation Title: Clinical Activity Of BLU-554, A Potent, Highly-Selective FGFR4 Inhibitor In Advanced Hepatocellular Carcinoma (HCC) With FGFR4 Pathway Activation
Session Title: General Session 5
Date & Time: Sunday, September 17, 2017 from 11:30 a.m. – 1:00 p.m. KST (Saturday, September 16, 2017 from 10:30 p.m. – 12:00 a.m. ET)
Abstract No: O-032
Location: Grand Hyatt Seoul

Syros Receives FDA Orphan Drug Designation for SY-1425 for Treatment of AML

On August 21, 2017 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of disease-driving genes, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SY-1425 for the treatment of acute myeloid leukemia (AML) (Press release, Syros Pharmaceuticals, AUG 21, 2017, View Source [SID1234520301]). SY-1425, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently in a Phase 2 clinical trial in genomically defined subsets of patients with AML and myelodysplastic syndrome (MDS).

"Treatment of AML remains a significant unmet medical need, with many patients lacking adequate therapeutic options," said David A. Roth, M.D., Syros’ Chief Medical Officer. "We believe that SY-1425 may provide a meaningful benefit for subsets of AML patients whose disease is driven by abnormally high expression of the RARA or IRF8 genes. Receiving orphan drug designation is an important regulatory milestone in the development of SY-1425. We’re pleased with the continued progress of the ongoing Phase 2 clinical trial, and we look forward to presenting initial clinical data in the fourth quarter of this year."

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

Using its gene control platform, Syros discovered subsets of AML and MDS patients with super-enhancers associated with RARA or IRF8. Syros identified proprietary biomarkers related to these super-enhancers. These super-enhancers are believed to drive overexpression of the RARA or IRF8 genes, locking cells in an immature, undifferentiated and proliferative state, leading to disease. In preclinical studies, SY-1425 promoted differentiation of AML cells with high RARA or IRF8 expression and inhibited tumor growth and prolonged survival in patient-derived xenograft models of AML with high RARA expression. Syros estimates that about one-third of AML and MDS patients have either the RARA or IRF8 biomarker, or both.

The ongoing Phase 2 clinical trial of SY-1425 is assessing the safety and efficacy of SY-1425 as a single agent in four AML and MDS patient populations, as well as in combination with azacitidine, a standard-of-care therapy, in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy. All patients in the trial are prospectively selected using biomarkers for high expression of RARA or IRF8. Additional details about the trial can be found using the identifier NCT02807558 at www.clinicaltrials.gov.

Argos Therapeutics Announces Interim Analysis of Phase 3 ADAPT Trial to be Presented at ESMO 2017 Congress

On August 21, 2017 Argos Therapeutics Inc. (NASDAQ:ARGS), an immuno-oncology company focused on the development and commercialization of individualized immunotherapies based on the Arcelis precision immunotherapy technology platform, reported that an interim analysis of data from the ongoing Phase 3 ADAPT clinical trial evaluating Rocapuldencel-T for the treatment of metastatic renal cell carcinoma (mRCC) will be presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress to be held Sept. 8 — 12 in Madrid, Spain (Press release, Argos Therapeutics, AUG 21, 2017, View Source [SID1234520295]).

The oral presentation, entitled, "Interim Analysis of the Phase 3 ADAPT Trial Evaluating Rocapuldencel-T (AGS-003), an Individualized Immunotherapy for the Treatment of Newly-Diagnosed Patients with Metastatic Renal Cell Carcinoma (mRCC)," will be given by Robert Figlin, MD, Professor and Chairman, Division of Hematology and Oncology at Cedars Sinai Medical Center and co-principal investigator for the ADAPT trial, on Monday, Sept. 11th at 12:00pm CEST. The presentation will provide an overview of the study data set as of the time of the interim analysis that was conducted by an independent data monitoring committee (IDMC) in February 2017, including previously disclosed data as well as additional subsequent analyses based upon the data set. Dr. Figlin will also discuss the rationale for continuing the ADAPT trial.

For more information, please visit View Source

BioLineRx Announces Regulatory Submission of Phase 3 Registrational Study for BL-8040 in Stem Cell Mobilization

On August 21, 2017 BioLineRx Ltd. (NASDAQ/TASE:BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported the filing of regulatory submissions required to commence a randomized, controlled Phase 3 registrational trial of BL-8040 for the mobilization of hematopoietic stem cells for autologous transplantation in patients with multiple myeloma (Press release, BioLineRx, AUG 21, 2017, View Source [SID1234520291]). The trial, named GENESIS, is expected to commence by the end of 2017, following receipt of regulatory approvals.

The Phase 3 GENESIS trial is aimed at evaluating the safety, tolerability and efficacy of the combination treatment of BL-8040 and granulocyte colony-stimulating factor (G-CSF), as compared to the control arm of placebo and G-CSF. The trial will be conducted in two parts: The first part, designed to validate the optimal dosing of BL-8040, is a lead-in, open-label, multi-center study that will include 10-30 patients, in order to assess the efficacy and safety of treatment with BL-8040 and G-CSF. This part will be followed by a randomized, placebo-controlled, multi-center study in approximately 180 patients. The primary endpoint will be the proportion of subjects mobilizing ≥6.0 x 106 CD34+ cells/kg with up to 2 apheresis sessions in preparation for autologous transplantation after a single administration of BL-8040 and G-CSF, as compared to placebo and G-CSF.

Philip Serlin, Chief Executive Officer of BioLineRx, stated, "We are excited to move forward with BL-8040 into a Phase 3 registration study. We have previously reported positive results supporting BL-8040 as a one-day dosing and up-to-two-day collection regimen for rapid mobilization of stem cells. This represents a significant improvement over the current treatment, which requires four-to-eight daily injections of G-CSF and one-to-four apheresis sessions. We therefore hope that this Phase 3 trial will further support these results and help improve the standard of care for multiple myeloma patients."

"In parallel, we are continuing to expand the potential of our robust BL-8040 oncology platform, by advancing multiple clinical studies for additional indications that are ongoing or expected to commence during 2017. These include a large, randomized, controlled Phase 2b study in AML, as well as several Phase 2 combination studies with immune checkpoint inhibitors in solid tumors and hematological malignancies," added Mr. Serlin.

About BL-8040
BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and stem cell mobilization. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells from the bone marrow, thereby sensitizing these cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing cell death (apoptosis). In addition, BL-8040 has also demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, T, B and NK cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Stem Cell Mobilization
High-dose chemotherapy followed by stem cell transplantation has become an established treatment modality for a variety of hematologic malignancies, including multiple myeloma, as well as various forms of lymphoma and leukemia. Stem cells are mobilized from the bone marrow using granulocyte colony-stimulating factor (G-CSF), harvested from the peripheral blood by apheresis, and infused to the patient after chemotherapy. This type of treatment often replaces the use of traditional bone marrow transplantation, because the stem cells are easier to collect and the treatment allows for a quicker recovery time and fewer complications.