2022 Operational and Financial Results
On March 22, 2023, BioLineRx Ltd. (NASDAQ:BLRX) reported 2022 operational and financial results in a press release concurrent with the filing of Form 20-F. A conference call and webcast were hosted later that morning. Key highlights since the previous earnings update in November include the report of data from the AGI-134 study in metastatic solid tumors, the appointment of a new Chief Medical Officer and a collaboration to use motixafortide in stem cell mobilization for gene therapy in sickle cell disease. Focus is on the upcoming target action date for motixafortide (Aphexda) on September 9th, 2023.
During the conference call the BioLineRx team and especially the President leading the US initiative, Holly May, inspired confidence that the launch of APHEXDA will be successful. The BioLineRx team has hired a number of professionals to lead up the commercialization effort including a head of US Sales and Marketing with previous experience commercializing plerixafor for stem cell mobilization at Sanofi and Genzyme. BioLineRx has initiated outreach activities visiting with leaders in the transplant community and attending Tandem, where notable stakeholders connect. Additional preparation efforts include pricing discovery, which has been augmented by recent pharmacoeconomic studies identifying a clear benefit from APHEXDA to prospects. The company’s strategy has prepared the commercialization team to launch shortly after regulatory approval is granted and is ready to begin early if the FDA announces before the planned target action date.
We provide a thorough discussion of BioLineRx’ plans to commercialize Aphexda in a previous report entitled BLRX: Self-Commercialization in the US. It discusses the company’s plans, reviews Aphexda’s mechanism of action and addresses the real-world benefits of an improved approach to stem cell mobilization.
Below we summarize financial results for the twelve-month period ended December 31, 2022, compared to the same period ending December 31, 2021:
➢ Research and development expenses totaled $17.6 million, down 9% from $19.5 million, on account of lower expenses related to the new drug application (NDA) supporting activities related to motixafortide and lower expenses associated with the completed motixafortide GENESIS clinical trial, offset by an increase in expenses associated with the AGI-134 study and in payroll and related expenses;
➢ Sales and marketing expenses were $6.5 million, up 540% from $1.0 million on account of initiation of pre-commercialization activities related to motixafortide and an increase in market research;
➢ General and administrative (G&A) expenses were $5.1 million, up 18% from $4.3 million due to a rise in share-based compensation and small increases across several G&A expense categories;
➢ Non-operating income was $5.7 million reflecting changes in fair-value adjustments of warrant liabilities on the balance sheet, offset by warrant offering expenses;
➢ Net financial expenses amounted to $1.5 million which was impacted by interest paid on loans, interest received from cash balances and strengthening of the US dollar;
➢ Net loss was ($25.0) million compared with ($27.1) million, or ($0.03) and ($0.04) per share respectively.
Cash, equivalents and short-term bank deposits as of December 31, 2022 totaled $51.1 million, down from year end 2021 balance of $57.1 million. BioLineRx raised a gross $55 million from debt and equity issuances in September increasing cash levels. Cash burn during 2022 amounted to ($23.2) million which was offset by a net $17.9 million contribution from financing cash flows.
Opportunities for Gene Therapy
In a March 6th press release, BioLineRx announced a collaboration with Washington University School of Medicine to evaluate motixafortide for CD34+ hematopoietic stem cell mobilization for gene therapy in sickle cell disease. The study opens up a door to enter into the gene therapy space which in many cases relies on the collection of stem cells for ex vivo modification of stem cells. Stem cell mobilization is important for gene therapy because it can increase the number of stem cells available for gene modification, which can improve gene therapy’s efficiency and effectiveness. In the case of sickle cell disease, the use of G-CSF is contraindicated which demands an alternative that can reliably generate sufficient cells in a safe manner.
The proposed study is a Phase I clinical trial that will evaluate the safety and feasibility of motixafortide to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies in sickle cell disease. Dr. John Dipersio, who we know from last year’s key opinion leader event, is the principal investigator for the trial and sees motixafortide as a candidate for a gene therapy stem cell mobilization agent. BioLineRx management sees other lentiviral gene therapy approaches, particularly those that are ex vivo as being well aligned with using motixafortide.
In late December, results from the Phase I/IIa study of AGI-134 in metastatic solid tumors were released. The study met its primary endpoint for safety and tolerability and AGI-134 demonstrated immune activity across multiple biomarkers. The product is classified as a biologic, which confers 12 years of market exclusivity upon approval by the FDA.
Total enrollment for the study was 38 subjects, 55% of which were male. Solid tumor types included melanoma (55%), colon, breast, squamous cell, sarcoma, cervical node, endometrial and synovial. A number of immune response biomarkers were identified including an increase in conventional dendritic cells (CD11c+, HLADR+), T helper cells (CD3+, CD4+), cytotoxic T cells (CD3+, CD+4) and macrophages (CD68+).
The study is comprised of two parts. The first is an accelerated dose-escalation stage to assess the safety and tolerability of intratumorally injected AGI-134 as a monotherapy, as well as to determine the maximum tolerated dose and the recommended dose for part 2 of the study. The second is a dose expansion stage at the recommended dose, designed to assess the safety, tolerability and anti-tumor activity of the drug as a monotherapy in a basket cohort of multiple solid tumor types.
The first part of the study was completed in September 2019, with AGI-134 being found to be safe and well tolerated, with no serious drug-related adverse events or dose-limiting toxicities reported. The maximum tolerated dose was not reached and the recommended dose for the second part of the study was identified. BioLineRx began the second part of the study in September 2019. Due to the pandemic, the clinical trial was suspended, but resumed in August 2020.
In January 2022, the AGI-134 trial completed enrollment and by year end results were announced. The study met its primary safety and tolerability endpoint. An immune response and markers of clinical efficacy were assessed as secondary endpoints. Most patients analyzed showed an increase in Alpha-Gal antibodies, indicating increased overall immune activity. Additionally, increases in antigen presenting cells were observed in most tissue samples analyzed. T cell and macrophage tumor infiltration was seen in approximately one-third of evaluable patients’ injected tumors and in approximately half of evaluable patients’ lesions not injected. Radiological assessments found that 29% (11/38) of patients in the trial achieved best overall response of stable disease. BioLineRx plans to seek publication of the data at a medical congress in 2023, and in consultation with its scientific advisory board, it will determine the next steps for the program in the 1H:23.
➢ Motixafortide, Phase II (Columbia) PDAC study data release - 2023
➢ Presentation of GENESIS data at medical meetings & conferences – 2023
➢ Initiate Phase II combination study of motixafortide with GenFleet – 2023
➢ Data published for Phase II PDAC trial – 2023
➢ Motixafortide, Phase I in Sickle Cell Disease - 2023
➢ Motixafortide in SCM target action (PDUFA) date – September 2023
➢ US launch of motixafortide in SCM – 3Q:23
➢ Potential initiation of randomized Phase 2 study of AGI-134 – 2023
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1. BioLineRx January 2023 Corporate Presentation. Note that the latest available version of BioLineRx’ pipeline does not include the most recent addition where motixafortide is in development to provide stem cell mobilization for a gene therapy approach for sickle cell anemia.