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Full Year 2021 Operational and Financial Results
On March 16, 2022, BioLineRx Ltd. (NASDAQ:BLRX) reported 2021 operational and financial results in a press release concurrent with the filing of Form 20-F. A conference call and webcast was hosted later that morning. Some of the key highlights shared include the results of a study that estimated US stem cell mobilization market value at $360 million in 2021, pharmacoeconomic studies demonstrating significant cost savings from Motixafortide use and anticipated mid-2022 submission of an NDA for Motixafortide to the FDA as mobilization agent for multiple myeloma patients undergoing autologous stem cell transplantation.
Achievements for the fourth quarter ending December 31, 2021 and to-date include:
➢ Positive results from pharmacoeconomic study - October 2021
➢ Formation of immuno-oncology (IO) scientific advisory board (SAB) - December 2021
➢ Presentations at ASH Annual Meeting - December 2021
➢ Pre-NDA meeting with FDA held for BL-8040 - January 2022
➢ Phase I/IIa AGI-134 enrollment completed - January 2022
➢ Additional positive results from pharmacoeconomic study - March 2022
Upcoming milestones include:
➢ AGI-134 Phase I/IIa initial results - 2H:22
➢ Presentation of GENESIS data at medical meetings & conferences - 2022
➢ BL-8040 NDA submission - mid-2022
➢ Phase II study of AGI-134 - 2023
➢ Potential FDA approval of Motixafortide - 2023
➢ US launch of motixafortide in SCM - 2023
Below we summarize financial results for the year ended December 31, 2021, compared to the same ending December 31, 2020:
➢ Research and development expenses totaled $19.5 million, increasing 7.1% from $18.2 million, resulting primarily from an increase in expenses associated with the AGI-134 phase 1/2a study, as well as an increase in payroll and related-expenses from the company-wide salary reduction in response to COVID-19, offset by lower expenses associated with the completed motixafortide GENESIS and COMBAT clinical trials;
➢ Sales and marketing expenses were $1.0 million, up 19% from $0.8 million resulting from an increase in consultancy services for motixafortide;
➢ General and administrative expenses were $4.3 million, increasing 10% from $3.9 million. The increase resulted primarily from an increase in directors' and officers' insurance expenses;
➢ Net financial expenses amounted to $0.4 million compared to $1.4 million, primarily relating to interest paid on loans, offset by investment income earned on bank deposits;
➢ Net loss was ($27.0) million compared with ($30.0) million, or ($0.04) and ($0.12), respectively.
Cash, equivalents and short term bank deposits as of December 31, 2021 totaled $57.1 million. BioLineRx has only current maturities of long-term loans amounting to $2.8 million. Cash consumed during 2021 amounted to $23.7 million. Net financing cashflows, driven primarily by share and warrant activity, generated $57.7 million.
Positive results from pharmacoeconomic study
As an adjunct to the Phase III trial, a pharmaco-economic study was initiated to evaluate the potential cost savings from using Motixafortide. The aim of the study, performed by IQVIA's Global Health Economics and Outcomes Research team, was to demonstrate the cost benefit derived from a reduction in doses of G-CSF and apheresis sessions required, a reduction in rescue therapies required, higher rates of transplantation, and quality-of-life benefits in Motixafortide-treated patients. The pharmacoeconomic study measured health resource utilization (HRU) during BioLineRX' GENESIS trial. Included in the HRU data collected were the following:
➢ Motixafortide and G-CSF doses;
➢ Apheresis sessions performed, in primary mobilization;
➢ Percentage of patients needing rescue mobilization due to poor primary mobilization, including apheresis sessions needed; and
➢ G-CSF and plerixafor doses required and hospitalization costs related to conditioning and transplantation, including length of
stay.
Quality-adjusted-life-years (QALYs) gained were also added to the model. Motixafortide plus G-CSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to standard-of-care G-CSF alone.
Data from the study was shared in an October 2021 release that highlighted a significant benefit for the use of Motixafortide. The cost benefits for using Motixafortide in combination with G-CSF in multiple myeloma patients were estimated at ~$17,000 (later updated to ~$19,000 in the FY:21 annual results to reflect inflation), excluding the cost of Motixafortide, when compared to the cost of G-CSF alone. The benefits arise from the ability of Motixafortide to increase the success of apheresis sessions, to HRU during the autologous stem cell transplantation process and produce QALY benefits. There are also additional safety and time benefits as well due to the fewer number of administrations and apheresis sessions needed.
On March 3, 2022, BioLineRx provided additional analysis from a follow-on pharmacoeconomic study based on data from BioLineRx' Phase III GENESIS trial. This study evaluated cost effectiveness of Motixafortide in combination with G-CSF compared with plerixafor with G-CSF in multiple myeloma patients undergoing autologous stem cell transplant. The supplemental analysis was performed by the same team at IQVIA which had generated the original findings reported in October 2021. Plerixafor data was sourced from literature as if it were an arm of the GENESIS trial (Bucher method), and these results were implemented in the cost-effectiveness model. Compared with plerixafor with G-CSF, motixafortide with G-CSF demonstrated a statistically significant decrease in HRU, improvement in QALY benefits, and net cost savings of ~$30,000 not including the cost of motixafortide, further supporting motixafortide and G-CSF as new standard of care in mobilization for autologous transplant.
Commercialization
During the call, management provided additional detail on commercialization plans for motixafortide. Pre-commercialization activities have started and include preparations related to medical affairs, commercial packaging and design, market access activities and brand name selection. The company is open to either internal or external commercialization and has identified about 80 transplant centers in the US that represent over 80% of stem cell transplant volume. A sales force of about 15-20 sales personnel would likely be appropriate for this number of touch points.
Formation of immuno-oncology (IO) scientific advisory board (SAB)
On December 15, 2021, BioLineRx shared its formation of an immuno-oncology Scientific Advisory Board (SAB). The SAB's role is to provide insight and guidance on BioLineRx' activities in the field of immuno-oncology and includes recognized leaders in cancer immunology, intra-tumoral injections and clinical development.
The founding SAB members include:
➢ Ronald Levy, MD, the Robert K. and Helen K. Summy Professor and Director of the Lymphoma Program at Stanford University School of Medicine, Palo Alto, CA;
➢ Aurélien Marabelle, MD, PhD, Clinical Director, Cancer Immunotherapy Program, Gustave Roussy, Paris, France and Director, Translational Research Laboratory in Immunotherapy, INSERM, Paris, France;
➢ Ignacio Melero MD, PhD, Professor of Immunology at the Academic Hospital of Navarra, Spain and at the Center for Applied Medical Research (CIMA) of the University of Navarra, Spain; and
➢ Jon Wigginton, MD, Chair of the SAB and Senior Advisor at Cullinan Oncology, former Chief Medical Officer of MacroGenics, and former Therapeutic Area Head, Immuno-Oncology, Early Clinical Research at Bristol-Myers Squibb.
Presentations at ASH Annual Meeting
BioLineRx presented three posters at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition, which was held December 11-14, 2021. The oral presentation was given by Dr. Zachary Crees from the Washington University School of Medicine in St. Louis and was entitled: "Motixafortide (BL-8040) and G-CSF Versus Placebo and G-CSF to Mobilize Hematopoietic Stem Cells for Autologous Stem Cell Transplantation in Patients with Multiple Myeloma: The GENESIS Trial." The presentation elaborated on the successful results of GENESIS Phase III pivotal trial of combination Motixafortide and G-CSF for the mobilization of stem cells in multiple myeloma patients.
Pre-NDA meeting with FDA held for BL-8040
On January 18, 2022, BioLineRx announced that it had a successful pre-NDA meeting with the FDA. Last year, top-line results for GENESIS were reported in May, following last-patient last-follow-up. GENESIS was launched in December 2017, and was a randomized, placebo-controlled, multi-center, Phase III registrational trial for the mobilization of hematopoietic stem cells for autologous transplantation in patients with multiple myeloma (MM). The trial began with an open-label, lead-in period for dose confirmation targeting 10-30 patients to evaluate safety and efficacy of Motixafortide with G-CSF. Positive results from the initial lead-in period with 11 patients prompted the Data Monitoring Committee to give the go-ahead to advance the full study, which targeted inclusion of 177 patients across more than 25 centers. In August 2020, an interim analysis for the full study, based on approximately 65% of the intended study sample size, was developed as patient dropout had been significantly lower than expected. Interim results were positive and were announced in October 2020. Equipped with statistically significant results, the DMC recommended a halt to patient enrollment. Topline results were made available in May 2021. On January 18, 2022, BioLineRx announced that it had a successful pre-NDA meeting with the FDA. The purpose of the meeting was to reach an agreement with the FDA regarding the content of motixafortide's NDA and to confirm that GENESIS provided sufficient clinical data. The FDA agreed that the proposed submission was sufficient. BioLineRx anticipates the NDA submission will occur in mid-2022.
Enrollment completed, Phase I/IIa (Part 2) AGI-134
On January 24, 2022, BioLineRx announced that enrollment for its Phase I/IIa was completed for AGI-134. AGI-134 is BioLineRx' intratumoral cancer vaccine candidate. This first-in-man study (NCT03593226) is designed to assess the safety and biological activity of AGI-134 in unresectable metastatic solid tumors. The Phase 1/2a study is a multicenter, open-label affair, which recruited 38 patients in the UK, Spain and Israel in two parts. Part 1 was completed in five subjects, and determined the recommended dose for part 2. Part 2 is a dose expansion study at the recommended dose in 33 patients, designed to evaluate the safety and tolerability of AGI-134, and to validate AGI-134's mechanism of action using a wide array of biomarkers. Results from the trial are expected to be announced in 2H:22.
Initiating Coverage
On January 13, 2022, we initiated on BioLineRx. In our initiation, we provide a scientific background on cytokines and their various receptors, including CXCR4, which has been implicated in the mobilization of hematopoietic stem cells. Background on chemokine agonists available in the market and in advanced studies is offered. We discuss stem cell transplantation and its relevant statistics. The report then moves into a review of BioLineRx' clinical candidates: Motixafortide and AGI-134. Mechanism of action is detailed and clinical trials both completed and active are examined for each indication. Epidemiology and standard of care treatment are presented for MM, AML along with additional details for pancreatic cancer. A review of the company's intellectual property is followed by a discussion on the competitive environment and profiles for management team members. We conclude with a summary of recent financial and operational events, risks and our valuation assumptions.
Summary
On March 16, 2022, BioLineRx reported fourth quarter and fiscal year 2021 financial and operational results. Highlights for the quarter and to date include positive results from BioLineRx' pharmacoeconomic study of motixafortide in stem cell mobilization, formation of the IO SAB, presentations at ASH Annual Meeting, a successful pre-NDA meeting with the FDA, and enrollment completion for AGI-134's Phase I/IIa (Part 2) trial. BioLineRx anticipates sharing initial results for AGI-134 in 2H:22, the presentation of GENESIS data at conferences in 2022, and NDA submission for BL-8040 mid-2022. Approval is anticipated in 2023 with US commercial launch shortly thereafter.
BioLineRx' lead candidate, Motixafortide, has completed its pivotal trial in MM and we expect an NDA to be submitted mid-2022. Following an expected year-long review process, we see commercialization beginning mid-2023. The market for Motixafortide is sizable with approximately 23,000 stem cell transplants taking place per year in the United States. About 59% of these are autologous and 90% are in MM and lymphomas with about 65% of the autologous MM and lymphoma patients appropriate for the therapy. BioLineRx' second indication for Motixafortide is in pancreatic cancer which is being developed in Phase II trials. Our timeline anticipates that a Phase II/III or Phase III trial will be launched in the next year producing top-line in 2026, followed by submission to regulatory agencies in the US, Europe and China. We also model development and eventual commercialization of AGI-134 in the United States.
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1. BioLineRX management stated that there were about 212 stem cell transplant centers in the United States, of which 79% comprise more than 80% of all procedures.
