BioLineRx Ltd. is making strides with its stem cell mobilization program, particularly with APHEXDA (motixafortide), which received FDA approval in September for use in combination with G-CSF to mobilize hematopoietic stem cells for collection and subsequent autologous transplantation in patients with multiple myeloma. This approval marks a significant advancement in stem cell mobilization, addressing a critical unmet need for a more effective regimen.
Commercial Launch and Market Opportunity
BioLineRx has initiated the commercial launch of APHEXDA in the U.S., focusing on the approximately 18,000 multiple myeloma patients eligible for autologous stem cell transplantation annually. Of these, around 8,000 procedures are performed each year, a number that has nearly doubled since 2010. However, up to 47% of patients face challenges in collecting the target number of stem cells in one apheresis session due to factors like an aging patient population and the increased use of intensive induction therapies. APHEXDA aims to improve these outcomes, with data from the GENESIS Phase 3 trial showing that almost 90% of patients who proceeded to transplantation required only one apheresis session.
The company has priced APHEXDA at $[redacted] per vial, reflecting its value in the autologous stem cell transplant landscape. Despite the availability of lower-priced generic plerixafor, BioLineRx believes APHEXDA's differentiated clinical attributes will drive long-term adoption and establish it as the new standard of care.
Clinical Trial Updates and Expansion
Beyond multiple myeloma, BioLineRx is exploring the potential of motixafortide in other indications. A randomized Phase 2 combination clinical trial (CheMo4METPANC) is underway to evaluate motixafortide in first-line pancreatic cancer. Preliminary data from the pilot phase of the study showed a 64% partial response rate and a 91% disease control rate, compared to historical rates of 23% and 48%, respectively, with the current standard of care. The trial's primary endpoint is progression-free survival (PFS), with secondary objectives including safety, response rate, and overall survival. Enrollment is expected to begin in the coming months.
Additionally, BioLineRx is collaborating with Washington University School of Medicine to evaluate motixafortide in autologous hematopoietic stem cell-based gene therapy for sickle cell disease. A proof-of-concept trial is studying motixafortide as both a single agent and in combination with natalizumab to assess the safety and tolerability of these regimens as mobilization agents of CD34+ stem cells. Initial data is anticipated in the second half of 2024.
Partnership in Asia
BioLineRx has entered an exclusive license agreement with Gloria Biosciences for the development and commercialization of motixafortide in Asia, covering multiple indications. Gloria Biosciences plans to conduct a 30 to 50 patient bridging study in China to support approval and commercialization of APHEXDA for stem cell mobilization in multiple myeloma. In 2022, Asia reported over 51,000 cases of multiple myeloma, highlighting a significant unmet need in the region. The agreement includes a $15 million upfront payment to BioLineRx, with potential development and commercial milestones totaling up to $250 million, as well as royalties on net sales.
