On December 4, 2017 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported positive overall survival data from the long-term follow-up part of the Phase 2a trial of BL-8040 for the treatment of relapsed or refractory acute myeloid leukemia (r/r AML) (Press release, BioLineRx, DEC 4, 2017, View Source;p=RssLanding&cat=news&id=2320793 [SID1234522347]). The results demonstrate that the combination of BL-8040 with high-dose Ara-C (HiDAC) in this difficult-to-treat patient population significantly improved overall survival, compared with historical data of HiDAC monotherapy.

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The long-term survival results from the Phase 2a study derive from the expansion phase of the study, which included 16 patients (out of 42 total patients enrolled in the study). Participants were treated with BL-8040 as a monotherapy for two days, followed by a combination of BL-8040 and HiDAC for 5 days. The mean follow-up time was 338 days (29-853 days). BL-8040 in combination with HiDAC was found to be safe and well tolerated, and the response rate was 38% (6/16). Median overall survival was 11.1 (1-28) months, the estimated one-year survival rate was 37.5% and the estimated two-year survival rate was 28.5%, compared to historical data for patients treated only with HiDAC showing overall survival of approximately 6.1 months. Furthermore, the subset of patients exhibiting a response showed prolonged overall survival, with estimated one-year and two-year survival rates of 60%. Median overall survival for this group could not be calculated, since only two of these patients have relapsed.

The Phase 2a study assessed the efficacy of BL-8040, as a single agent and in combination with HiDAC, for the treatment of r/r AML. The majority of patients in the study were heavily pre-treated, and the treated patient population included patients who had relapsed following allogeneic stem-cell transplantation, as well as secondary AML patients – both conditions which represent difficult-to-treat populations with poor prognoses. Top-line results from the full study (n=42) were previously reported in March 2016, and showed an overall response rate (CR+CRi) of 38%, compared with historical data relating to HiDAC of approximately 20%.

Philip Serlin, Chief Executive Officer of BioLineRx, commented, "We are very pleased from the long-term follow-up results of this study, which continue to demonstrate the robust anti-leukemic activity of BL-8040 and show that combined treatment with HiDAC not only increases the response rate, but also increases overall survival time compared to historical data. We will continue to monitor the overall long-term survival of patients in this study, as we progress in the execution of our two other important studies in AML – our Phase 2a study in maintenance AML, which is part of our collaboration with Genentech, and our Phase 2b study in consolidation AML. The promising survival and response results we have seen in the r/r AML study, along with the future data readouts from the additional studies we’re currently running in this disease, have the potential to make BL-8040 a key player in the AML setting."

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 and immune-cells from the bone marrow, thereby sensitizing cancer cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing cell death (apoptosis) and mobilizing immune-cells. 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.

On December 4, 2017 Seattle Genetics, Inc. (NASDAQ:SGEN) reported that the company will webcast an investor and analyst event on Monday, December 11, 2017 during the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta, GA (Press release, Seattle Genetics, DEC 4, 2017, View Source;p=RssLanding&cat=news&id=2320818 [SID1234522352]). Industry experts will discuss the ADCETRIS (brentuximab vedotin) phase 3 ECHELON-1 data, and members of the Seattle Genetics management team will discuss other program highlights.

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The event will take place from approximately 8:30 p.m. to 9:30 p.m. Eastern Time. The webcast will be available live and for replay from Seattle Genetics’ website at www.seattlegenetics.com in the Investors section.

On December 4, 2017 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported updated clinical data from the larotrectinib pediatric Phase 1 SCOUT clinical trial (Press release, Loxo Oncology, DEC 4, 2017, View Source [SID1234522348]). These data are being presented today at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Pediatric Cancer Research in Atlanta.

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"Targeted therapy success stories in pediatric oncology are uncommon, and larotrectinib has invigorated the pediatric oncology community," said Brian Turpin, D.O., the presenting SCOUT principal investigator and assistant professor in the division of oncology at Cincinnati Children’s Hospital. "Larotrectinib’s near universal response rate and compelling durability of response in pediatric patients with TRK fusion cancers is likely to be practice changing. Furthermore, the first-ever TRK inhibitor response in a TRK fusion glioblastoma patient highlights the potential for larotrectinib in TRK fusion central nervous system tumors."

"We are grateful to the children and families who have enabled the development of larotrectinib through their participation in clinical trials," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology.

As of a July 17, 2017 data cut-off date, 24 pediatric patients were enrolled in the dose escalation portion of the Phase 1 trial, including 17 patients with TRK fusion cancers. TRK fusion patients carried primary diagnoses of infantile fibrosarcoma, thyroid cancer, and various soft tissue sarcomas.

TRK Fusion Patients (n=17)* Patients without TRK Fusions (n=7)
Independent Review Committee Assessed Response Investigator Assessed Response Investigator Assessed Response
Overall Response Rate
(ORR = PR+CR) 93%
(95% CI: 68% – 100%) 93%
(95% CI: 68% – 100%) 0%
Partial Response (PR) 80% 67%** 0%
Complete Response (CR) 13% 27% 0%
Stable Disease 7% 7% 0%
Progressive Disease 0% 0% 100%
* 2 patients not evaluable due to having non-measurable disease at baseline.
** Includes 2 patients with unconfirmed partial responses as of July 17, 2017, which were subsequently confirmed.

Among the 17 patients with TRK fusion cancers, 94% either remain on drug or received surgery with curative intent; four patients have been followed greater than one year and 12 have been followed greater than six months.

The larotrectinib adverse event profile is consistent with data previously presented publicly. The most common treatment-related adverse events at the Phase 2 dose included increased liver function tests, neutropenia, and nausea, all largely grade 1.

These data are being presented in a poster session on December 4, 2017 and an oral presentation on December 5, 2017. The poster and presentation will be available online at View Source at the time of their scheduled presentations.

About Larotrectinib (LOXO-101)
Larotrectinib is a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities involving the tropomyosin receptor kinases (TRKs). Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In an analysis of 55 RECIST-evaluable TRK fusion adult and pediatric patients, larotrectinib demonstrated a 75 percent independently-reviewed confirmed overall response rate (ORR) and an 80 percent investigator-assessed confirmed ORR, across many different types of solid tumors. Larotrectinib has been granted Breakthrough Therapy Designation Rare Pediatric Disease Designation and Orphan Drug Designation by the U.S. FDA. For additional information about the larotrectinib clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123 or visit www.loxooncologytrials.com.

In November 2017, Loxo Oncology and Bayer entered into an exclusive global collaboration for the development and commercialization of larotrectinib and LOXO-195, a next-generation TRK inhibitor. Loxo Oncology leads worldwide development and U.S. regulatory activities. Bayer leads ex-U.S. regulatory activities and worldwide commercial activities. In the U.S., Loxo Oncology and Bayer will co-promote the products.

About TRK Fusion Cancer
TRK fusions are chromosomal abnormalities that occur when one of the NTRK genes (NTRK1, NTRK2, NTRK3) becomes abnormally connected to another, unrelated gene (e.g. ETV6, LMNA, TPM3). This abnormality results in uncontrolled TRK signaling that can lead to cancer. TRK fusions occur rarely but broadly in various adult and pediatric solid tumors, including appendiceal cancer, breast cancer, cholangiocarcinoma, colorectal cancer, GIST, infantile fibrosarcoma, lung cancer, mammary analogue secretory carcinoma of the salivary gland, melanoma, pancreatic cancer, thyroid cancer, and various sarcomas. TRK fusions can be identified through various diagnostic tests, including targeted next-generation sequencing (NGS), immunohistochemistry (IHC), polymerase chain reaction (PCR), and fluorescent in situ hybridization (FISH). For more information, please visit www.TRKtesting.com.

On December 4, 2017 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology Company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, reported key presentations of clinical and scientific data related to its products at the 59th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), being held in Atlanta, Georgia, from December 9-12, 2017 (Press release, Spectrum Pharmaceuticals, DEC 4, 2017, View Source [SID1234522353]).

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For more information about the ASH (Free ASH Whitepaper) annual meeting and for a complete list of abstracts, please refer to the conference website at View Source

The following key PTCL-related abstracts and oral presentations will be presented at the ASH (Free ASH Whitepaper) meeting:

Abstract # Type Title Presenter
Date/Time
Location

342 Oral
The Role of Upfront Hematopoietic Stem Cell Transplantation (HSCT) in Peripheral T-Cell Lymphoma (PTCL) Patients in Complete Remission (CR) with a Special Focus on Nodal PTCL: Report from the Comprehensive Oncology Measures for Peripheral T-Cell Lymphoma Treatment (COMPLETE), a Prospective Multicenter Cohort Study
Park Sunday, Dec 10,
8:45 AM

Bldg B, Lvl 4, B401-402
818 Oral Pralatrexate in Combination with Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone (CHOP) in Previously Untreated Patients with Peripheral T-Cell Lymphoma (PTCL): A Phase 1 Dose-Escalation Study Shustov Monday, Dec 11,
4:45 PM

Bldg A, Lvl 4, Marcus Aud.
1455 Poster
North American Adult T Cell Leukemia Lymphoma (ATLL) Is Characterized By Distinct, Therapeutically Targetable Mutations in Epigenetic Modifiers
Shah Saturday, Dec 9,
5:30 PM-7:30 PM

Bldg A, Lvl 1, Hall A2
2743 Poster
Azacytidine and Decitabine Exhibit Differential Effects on Cytotoxicity and Methylation and Exhibit Class Synergy with Hdaci in Models of Ptcl
Scotto Sunday, Dec 10,
6:00 PM-8:00 PM

Bldg A, Lvl 1, Hall A2
380 Poster Direct to Drug Screening as a Route to Individualized Therapy in Multiple Myeloma Meurice Sunday, Dec 10,
6:00 PM-8:00 PM

Bldg A, Lvl 1, Hall A2
4012 Poster Histone Deacetylase Inhibitors Abolish HBZ Protein Expression and Induce Cell Death in HTLV-1 Related Adult T-Cell Leukemia-Lymphoma Ramos Monday, Dec 11,
6:00 PM-8:00 PM

Bldg A, Lvl 1, Hall A2
4020 Poster Selective Inhibition of HDAC1 and HDAC3 Inhibits Survival of Diffuse Large Cell Lymphoma Via Epigenetic Modulation of Death Associated Protein (DAPK) Gupta Monday, Dec 11
6:00 PM-8:00 PM

Bldg A, Lvl 1, Hall A2
The following key EVOMELA (melphalan) for injection-related abstracts will be presented at the ASH (Free ASH Whitepaper) meeting:

Abstract # Type
Title
Presenter
Date/Time
Location
Bldg A, Lvl 1, Hall A2

2023 Poster
Feasibility, Tolerability, and Patient-Reported Outcomes with Pharmacokinetic (PK)-Directed Dosing of Evomela (propylene glycol free melphalan) for Multiple Myeloma and AL Amyloidosis Patients Undergoing Autologous Hematopoietic Stem Cell Transplant (AHCT)
Shah Saturday, Dec 9,
5:30 PM-7:30 PM
3296 Poster Pharmacokinetics and Toxicities after Evomela (propylene glycol free melphalan) with Autologous Hematopoietic Stem Cell Transplant (AHCT) for Multiple Myeloma and AL Amyloidosis Shah Sunday, Dec 10,
6:00 PM-8:00 PM
The following key MARQIBO (vinCRIStine sulfate LIPOSOME injection)-related abstracts will be presented at the ASH (Free ASH Whitepaper) meeting:

Abstract # Type Title Presenter
Date/Time
Location
Bldg A, Lvl 1, Hall A2
1549 Poster Excellent Outcome of Elderly Patients with Favourable-Prognosis DLBCL Treated with 4 Cycles CHOP/Chlip-14 Plus 8 Applications of Rituximab and a PET-Based Intensification Strategy That Includes Involved-Site Radiotherapy (IS-RT): Results of the First 120 Patients of the OPTIMAL > 60 Trial of the Dshnhl Pfreundschuh Saturday, Dec 9,
5:30 PM-7:30 PM
1328 Poster Treatment of Relapsed or Refractory Acute Lymphoblastic Leukemia in Real-World US Practices LeBlanc Saturday, Dec 9,
5:30 PM-7:30 PM
2554 Poster Phase II Study of Hyper-Cmad with Liposomal Vincristine (Marqibo) for Patients with Newly Diagnosed Acute Lymphoblastic Leukemia (ALL) Sasaki Sunday, Dec 10,
6:00 PM-8:00 PM
The following key ZEVALIN (ibritumomab tiuxetan)-related abstracts will be presented at the ASH (Free ASH Whitepaper) meeting:

Abstract #

Type Title Presenter
Date/Time
Location
Bldg A, Lvl 1, Hall A2
1553 Poster A Phase II Intergroup Trial of PET-Directed Therapy for Limited Stage Diffuse Large B-Cell Lymphoma (DLBCL): SWOG Study S1001 (NCT01359592) Persky Saturday, Dec 9,
5:30 PM-7:30 PM
4559 Poster Long-Term Follow up of 90y-Ibritumomab Tiuxetan, Fluadarabine and TBI Based Non-Myeloablative Allogeneic Transplant Conditioning for Persistent High-Risk B-Cell Lymphoma Puronen Monday, Dec 11,
6:00 PM-8:00 PM
4066 Poster Efficacy of 90Y-Ibritumomab Tiuxetan in the Older Population with Non-Hodgkin Lymphoma Pre-Treated with Rituximab-Based Regimens: A Single-Institution Experience Agrawal Monday, Dec 11,
6:00 PM-8:00 PM

On December 4, 2017 Mylan N.V. (NASDAQ, TASE: MYL) and its partner, Aspen (JSE: APN), reported the U.S. launch of Myleran (busulfan) Injection, 60 mg/10 mL (6 mg/mL) Single-dose Vial, a generic version of Otsuka Pharmaceutical’s Busulfex Injection (Press release, Mylan, DEC 4, 2017, View Source [SID1234522349]). Aspen received final approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA) for this product, which is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia. (1)

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As part of its partnership with Aspen, Mylan will commercialize Busulfan Injection, 60 mg/10 mL (6 mg/mL) Single-dose Vial in the U.S. Mylan has one of the largest injectable portfolios in the industry and is also one of the largest suppliers of cancer medicines by volume in the U.S.

U.S. sales for Busulfan Injection, 60 mg/10 mL (6 mg/mL) Single-dose Vial were approximately $97 million for the 12 months ending Sept. 30, 2017, according to IQVIA.