Data from first lead-in patient cohort prompts Data Monitoring Com­mit­tee to rec­om­mend early con­tin­u­a­tion to ran­dom­ized placebo-controlled part 2 of trial

Tel Aviv, Israel (Press Release) – BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clin­i­cal-stage bio­pharma­ceu­tical com­pany focused on on­col­ogy and immunology, an­nounced to­day pos­i­tive re­sults from the lead-in period of the GENESIS trial, a double-blind, placebo-controlled Phase 3 trial com­par­ing BL-8040 in com­bi­na­tion with granulocyte colony-stimulating factor (G-CSF), to G-CSF alone, for the mobili­za­tion of hema­to­poietic stem cells (HSCs) used for au­tol­o­gous trans­plan­ta­tion in mul­ti­ple myeloma patients.

The open-label, single-arm, lead-in period of the study was de­signed to in­clude up to 30 patients, with Data Monitoring Com­mit­tee (DMC) re­view after com­ple­tion of approx­i­mately 10, 20 and 30 patients, in order to assess safety and ef­fi­cacy fol­low­ing treat­ment with BL-8040 plus G-CSF. Results of the first 11 patients show that BL-8040 in com­bi­na­tion with stan­dard G-CSF treat­ment is safe and tol­er­able. In addi­tion, the data show that 9/11 patients (82%) reached the pri­mary end­point threshold of ≥ 6x106 CD34 cells/kg with only one dose of BL-8040 and in up to 2 apheresis sessions. Fur­ther­more, 7/11 patients (64%) reached the threshold of ≥ 6x106 CD34 cells/kg in a single apheresis session only. These data dem­onstrate the poten­tial of BL-8040 treat­ment to re­duce the num­ber of admin­istra­tions and apheresis sessions, as well as hospi­tal­iza­tion costs, re­lated to the preparation of mul­ti­ple myeloma patients for au­tol­o­gous HSC trans­plan­ta­tion.

"Autologous HSC trans­plan­ta­tion fol­low­ing high-dose chemo­ther­apy has sig­nif­i­cantly im­proved out­comes for mul­ti­ple myeloma patients," said Dr. John F. DiPersio, Chief, Division of Oncology at the Washington Uni­ver­sity School of Medicine, and lead in­ves­ti­ga­tor of the study. "Current prac­tice in­volves mobilizing HSCs from the bone mar­row to the periph­eral blood, after which the cells are collected by apheresis. Results so far show that mobilizing HSCs with a single BL-8040 dose com­bined with G-CSF is not only safe and tol­er­able, but also dem­onstrates robust ef­fi­cacy re­gard­ing the num­ber of collected cells, and may re­duce the num­ber of re­quired apheresis sessions to a single session in the majority of patients. This is a very en­cour­ag­ing re­sult that, if corroborated in the placebo-controlled part of the trial, will be of great value to patients as well as to the med­i­cal com­munity. I am looking for­ward to par­tic­i­pat­ing in the trial and to poten­tially im­prov­ing the quality of treat­ment avail­able to mul­ti­ple myeloma patients."

"We are ex­treme­ly en­cour­aged by these re­sults. Based on the robust data re­ceived from the first 11 patients, the DMC issued a pos­i­tive recom­menda­tion to stop the lead-in part of the study and move im­medi­ately to the ran­dom­ized placebo-controlled part of the study," stated Philip Serlin, Chief Exec­u­tive Of­fi­cer of BioLineRx. "This is the first Phase 3 trial for our lead BL-8040 pro­gram, and as such, it is an im­por­tant mile­stone in BL-8040's com­pre­hen­sive devel­op­ment plan. We look for­ward to the top-line re­sults from the ran­dom­ized, double-blind, placebo-controlled part of the study, which are ex­pec­ted in 2020."

About the GENESIS Study

The GENESIS study is a Phase 3, ran­dom­ized, double-blind, placebo-controlled, multi­center study, eval­u­ating the safety, tol­er­a­bil­ity and ef­fi­cacy of BL-8040 in com­bi­na­tion with G-CSF, com­pared to placebo and G-CSF, for the mobilization of CD34 HSCs for au­tol­o­gous trans­plan­ta­tion in mul­ti­ple myeloma patients. The placebo-controlled part is de­signed to in­clude 177 patients in more than 25 centers. Treatment will in­clude 5 days of G-CSF, with a single dose of BL-8040 or placebo on day 4 with the op­tion to ex­pand treat­ment to up to 8 days of G-CSF and up to 2 days of BL-8040. Apheresis for collection of CD34 cells will be per­formed on day 5. An addi­tional 3 apheresis sessions may be con­ducted if needed in order to reach the goal of ≥ 6x106 mobilized CD34 cells/kg.

The pri­mary objective of the study is to dem­onstrate the superiority of a single dose of BL-8040 in com­bi­na­tion with G-CSF, over placebo and G-CSF, in the mobilization of ≥ 6x106 CD34 cells/kg in up to 2 apheresis sessions, in preparation for au­tol­o­gous stem cell trans­plan­ta­tion in mul­ti­ple myeloma patients. Secondary objectives in­clude time to engraftment of neu­tro­phils and plate­lets, durability of the engraftment, as well as safety and other ef­fi­cacy parameters.

About BL-8040

BL-8040 is a short syn­thet­ic peptide for stem cell mobilization and for treat­ment of hema­to­logical malig­nan­cies and solid tumors. It functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine re­cep­tor that is directly in­volved in the retention of stems cells in the bone mar­row, as well as tumor pro­gres­sion, angiogenesis, metastasis and cell sur­vival. CXCR4 is over-expressed in more than 70% of human can­cers and its ex­pres­sion often correlates with dis­ease se­ver­i­ty.

HSCs ex­press CXCR4 and are retained in the pro­tective bone mar­row niche via binding to CXCL12 (also known as SDF-1). Blocking of the CXCR4-SDF1 inter­action by BL-8040 leads to the mobilization of HSCs into the periph­eral blood. In a num­ber of clin­i­cal and pre-clinical stud­ies, BL-8040 has shown robust mobilization of HSCs.

Furthermore, BL-8040 induce mobilization of leukemic cells and im­mune-cells from the bone mar­row, thereby sensitizing leukemic cells to chemo- and bio-based anti-cancer ther­apy, as well as a direct anti-cancer effect by inducing cell death (apoptosis). BL-8040 was li­censed by BioLineRx from Biokine Thera­peutics and was pre­vi­ously devel­oped under the name BKT-140.

About BioLineRx

BioLineRx is a clin­i­cal-stage bio­pharma­ceu­tical com­pany focused on on­col­ogy and immunology. The Com­pany in-licenses novel com­­pounds, de­vel­ops them through pre-clinical and/or clin­i­cal stages, and then part­ners with pharma­ceu­tical com­pa­nies for ad­vanced clin­i­cal devel­op­ment and/or com­mer­cial­iza­tion.

BioLineRx's lead­ing thera­peutic can­di­dates are: BL-8040, a multi-therapy plat­form, which has suc­cess­fully com­pleted a Phase 2a study for re­lapsed/refractory AML, is in the midst of a Phase 2b study as an AML con­sol­i­da­tion treat­ment and has ini­ti­ated a Phase 3 study in stem cell mobilization for au­tol­o­gous trans­plan­ta­tion; and AGI-134, an immuno­therapy treat­ment in devel­op­ment for mul­ti­ple solid tumors, which has recently ini­ti­ated a Phase 1/2a study. In addi­tion, BioLineRx has a stra­te­gic col­lab­o­ra­tion with Novartis for the co-development of selected Israeli-sourced novel drug can­di­dates; a col­lab­o­ra­tion agree­ment with MSD (known as Merck in the US and Canada), on the basis of which the Com­pany is con­ducting a Phase 2a study in pancreatic can­cer using the com­bi­na­tion of BL-8040 and Merck's KEYTRUDA®; and a col­lab­o­ra­tion agree­ment with Genentech, a member of the Roche Group, to in­ves­ti­gate the com­bi­na­tion of BL-8040 and Genentech's atezo­lizu­mab in sev­er­al Phase 1b/2 stud­ies for mul­ti­ple solid tumor indi­ca­tions and AML.

For addi­tional in­­for­ma­tion on BioLineRx, please visit the Com­pany's website at www.biolinerx.com, where you can re­view the Com­pany's SEC filings, press re­leases, an­nouncements and events. BioLineRx in­dus­try up­dates are also reg­u­larly up­dated on Face­book, Twitter, and LinkedIn.

Cautions Concerning Forward-Looking State­ments

Various state­ments in this re­lease con­cern­ing BioLineRx's future ex­pec­ta­tions con­sti­tute "forward-looking state­ments" within the meaning of the Private Se­cu­ri­ties Lit­i­ga­tion Reform Act of 1995. These state­ments in­clude words such as "may," "expects," "antic­i­pates," "be­lieves," and "intends," and describe opinions about future events. These for­ward-looking state­ments in­volve known and un­known risks and un­cer­tain­ties that may cause the actual re­sults, per­for­mance or achieve­ments of BioLineRx to be ma­teri­ally dif­fer­en­t from any future re­sults, per­for­mance or achieve­ments ex­pressed or im­plied by such for­ward-looking state­ments. Some of these risks are: changes in rela­tion­ships with col­lab­o­rators; the im­pact of competitive prod­ucts and tech­no­log­i­cal changes; risks relating to the devel­op­ment of new prod­ucts; and the ability to implement tech­no­log­i­cal im­prove­ments. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual re­port on Form 20-F filed with the Se­cu­ri­ties and Ex­change Com­mis­sion on March 6, 2018. In addi­tion, any for­ward-looking state­ments rep­re­sent BioLineRx's views only as of the date of this re­lease and should not be relied upon as rep­re­senting its views as of any sub­se­quent date. BioLineRx does not assume any obli­ga­tion to up­date any for­ward-looking state­ments unless re­quired by law.

Source: BioLineRx Ltd.