On November 10, 2022 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a pre-commercial-stage biopharmaceutical company focused on oncology, reported that the U.S. Food and Drug Administration (FDA) has accepted for review and filed the Company’s New Drug Application (NDA) for APHEXDA (motixafortide) in stem cell mobilization for autologous transplantation in multiple myeloma patients (Press release, BioLineRx, NOV 10, 2022, View Source [SID1234623742]). The FDA has assigned the NDA a Prescription Drug User Fee Act (PDUFA) target action date of September 9, 2023.

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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. With the trend toward more aggressive induction treatment protocols, there is a clear need amongst patients with multiple myeloma to be able to reliably and rapidly secure the necessary amount of stem cells to continue their treatment programs.

"APHEXDA has the potential to significantly improve outcomes and treatment experiences for patients with multiple myeloma, and the acceptance of our NDA brings us closer to this important goal," said Philip Serlin, Chief Executive Officer of BioLineRx. "The clinical outcomes demonstrated by our GENESIS Phase 3 study showed that nearly 90 percent of patients collected an optimal number of cells for transplantation following a single administration of APHEXDA and in only one apheresis session. We believe APHEXDA can become the standard of care in the multiple myeloma transplant setting, while also substantially decreasing healthcare resource utilization across a number of important areas. The Company is actively engaged in launch preparedness and excited about the potential of bringing this important therapeutic candidate to patients."

The NDA is supported by the results from the GENESIS Phase 3 trial of motixafortide on top of G-CSF (versus placebo on top of G-CSF) in stem cell mobilization for autologous transplantation in multiple myeloma patients. The study met all primary and secondary endpoints with a very high degree of statistical significance (p<0.0001). The combination was also found to be safe and well tolerated.

About the GENESIS Trial

The GENESIS trial (NCT03246529) was initiated in December 2017. GENESIS is 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.