On November 3, 2022 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a pre-commercial-stage biopharmaceutical company focused on oncology, reported that its full pharmacoeconomic study that indirectly evaluated the cost-effectiveness of using the investigational drug motixafortide as a primary stem cell mobilization (SCM) agent in combination with granulocyte colony stimulating factor (G-CSF), against plerixafor in combination with G-CSF, in multiple myeloma (MM) patients undergoing autologous stem cell transplantation, will be presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting held from December 10-13 in New Orleans, LA (Press release, BioLineRx, NOV 3, 2022, View Source [SID1234622932]). The analysis demonstrated significant net cost savings with motixafortide plus G-CSF and a greater proportion of patients achieving successful mobilization of optimal amounts of stem cells in a single apheresis day. The company has submitted a new drug application to the FDA for motixafortide in this indication.

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Collaborating investigators will also present a Phase 1 trial design of a novel mobilization regimen combining motixafortide and a VLA-4 inhibitor. The clinical trial will evaluate the regimen’s ability to safely produce the significant quantity of hematopoietic stem cells (HSC) required for the genetic manipulation processes used in gene therapy development in patients with sickle cell disease (SCD). Sickle cell disease is one of the most common inherited genetic diseases globally and HSC -based gene therapies now offer curative potential; however, effective HSC-based gene therapy depends on the collection of sufficient HSCs. Currently, the most widely used mobilization strategies include G-CSF and CXCR4 inhibition with plerixafor. However, G-CSF is contra-indicated in SCD and short-acting CXCR4 inhibition with plerixafor alone does not reliably yield optimal HSC numbers for SCD gene therapy applications. Developing novel HSC mobilization regimens to rapidly and reliably mobilize optimal CD34+ HSCs for gene therapy in SCD represents an unmet need.

"We are pleased to publish our full pharmacoeconomic data at ASH (Free ASH Whitepaper) which demonstrates that the combination of motixafortide plus G-CSF significantly lowers healthcare resource utilization in patients with multiple myeloma in the U.S. undergoing autologous stem cell transplantation," said Philip Serlin, Chief Executive Officer of BioLineRx. "Importantly, motixafortide plus G-CSF had significantly greater successful mobilization of the optimal number of cells following a single administration of motixafortide and in only one apheresis session, improving the patient journey. If approved, we believe motixafortide has the potential to become the new standard of care for all patients with multiple myeloma undergoing autologous stem cell transplantation. We are also excited to introduce the design of a Phase 1 study by investigators who are targeting unmet needs in both quantity and quality of cells harvested for cell engineering and administration. Our team is working diligently to develop motixafortide in different areas of stem cell mobilization with the objective of becoming the standard of care in these settings."

Major Findings of Cost-Effectiveness Analysis

The study was performed by the Global Health Economics and Outcomes Research (HEOR) team of IQVIA. For this study, an adjusted indirect comparison was undertaken, using data from the relevant phase 3 trials. This included finding and extracting efficacy data for both motixafortide (from GENESIS Phase 3 trial patient-level data) and plerixafor (aggregate data from literature), estimating plerixafor efficacy as if it had been one arm of the GENESIS trial (Bucher method), and implementing the results in the cost-effectiveness model.

Healthcare resource utilization (HRU) outcomes significantly favored motixafortide plus G-CSF during primary mobilization, including less frequent administration of G-CSF, lower G-CSF doses, and fewer apheresis sessions
The proportion of patients with successful mobilization of≥6×106 CD34+ cells/kg within 1 apheresis day was significantly greater with motixafortide plus G-CSF than with plerixafor plus G-CSF
Motixafortide plus G-CSF was dominant to plerixafor plus G-CSF over a lifetime horizon, resulting in additional QALYs and lower total costs as per base case deterministic results. The key cost drivers were the probability of successful transplantation, long-term maintenance costs, and the time to engraftment in poor mobilizers
PRESENTATIONS AT ASH (Free ASH Whitepaper)

Title: Cost-Effectiveness Analysis of Motixafortide Versus Plerixafor in Stem Cell Mobilization for Autologous Transplantation in Patients with Multiple Myeloma
Presenting Author: Mark Lamotte, MD, Global Health Economics & Outcomes Research, IQVIA
Date/Time: Poster 4848, Monday, December 12, 2022, 6:00 PM – 8:00 PM

Title: A Phase I Safety and Feasibility Study to Evaluate Motixafortide (CXCR4/SDF-1 Inhibition) and Natalizumab (VLA-4/VCAM-1 Inhibition) As a Novel Regimen to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapy in Sickle Cell Disease
Presenting Author: Zachary Crees, MD, Division of Oncology, Washington University School of Medicine, St. Louis, MO
Date/Time: Poster 4679, Monday, December 12, 2022, 6:00 PM – 8:00 PM

About the GENESIS Trial
The GENESIS trial (NCT03246529) was initiated in December 2017. GENESIS was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only one dose of motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize ≥ 6 million CD34+ cells in up to two apheresis sessions. A key secondary objective of the study was to demonstrate that only one dose of motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize ≥ 6 million CD34+ cells in only one apheresis session. In this regard, ~90% of patients in the GENESIS study went directly to transplantation after mobilizing the optimal number of stem cells following only one administration of motixafortide on top of G-CSF and in only one apheresis session, compared to less than 10% of those receiving G-CSF alone. Additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters.

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that affects some white blood cells called plasma cells, which are found in the bone marrow. When damaged, these plasma cells rapidly spread and replace normal cells in the bone marrow with tumors. In 2022, it is estimated that more than 34,000 people will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease in the U.S. While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.

About Autologous Stem Cell Transplantation
Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma. In the U.S., nearly 15,000 ASCTs are performed each year with the majority in patients with multiple myeloma. The current standard of care includes the administration of 5-8 daily doses of granulocyte colony stimulating factor (G-CSF), with or without 1-4 doses of plerixafor, and the performance of 1-4 apheresis sessions. For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy is carried out, consisting of 1-4 additional doses of plerixafor on top of G-CSF, and the performance of an additional number of apheresis sessions as necessary. In light of this, an agent with superior mobilization activity may significantly reduce the mobilization and harvesting burden and associated risks of the ASCT process and lead to significant clinical and resource benefits.