• Recognition of the unmet medical need and high therapeutic potential of ImMucin in Multiple Myeloma
• A multi-center phase II clinical study under IND is planned to start in Multiple Myeloma patients during 2015.

Ness Ziona, Israel (Press Release) – Vaxil Bio (TASE: VAXL), a leading developer of immuno­therapeutic prod­ucts to treat cancer and infectious dis­eases, reports today that its lead drug can­di­date, ImMucin has received orphan drug desig­na­tion from the European Medicines Agency (EMA) of the European Com­mis­sion (EC) for the treat­ment of Multiple Myeloma (MM), a blood cancer. ImMucin targets the less studied Signal Peptide domain of the MUC1 tumor an­ti­gen, which harbors key advantages as an anti-MM modality.

Orphan desig­na­tion by the EMA is granted to promote the clin­i­cal devel­op­ment of drugs that target rare life-threatening or debilitating con­di­tions and which are ex­pec­ted to provide sig­nif­i­cant thera­peutic advantage over existing treat­ments. Orphan desig­na­tion provides sig­nif­i­cant benefits, in­­clud­ing ten years of market exclusivity fol­low­ing market­ing approval, reductions in the fees and costs of the regu­la­tory process and sci­en­tific assistance from the EMA in clin­i­cal devel­op­ment.

Dr. Lior Carmon, Vaxil's Founder and CEO comments: "Obtaining orphan drug desig­na­tion for ImMucin in the European Union is a highly important mile­stone in the devel­op­ment of this novel treat­ment. There are still limited treat­ment options for those MM patients with "minimal residual dis­ease" i.e. in a state where the dis­ease is in remission or near remising but with residual tumor cells in the bone marrow, which are known today to lead to inferior over­all survival. These patients are looking for a main­te­nance treat­ment that can man­age their dis­ease and stop or at least delay relapse. We are excited about ImMucin's prospects to po­ten­tially treat these patients as val­i­dated by the EMA's de­ci­sion. This will obviously be further eval­u­ated in our upcoming clin­i­cal trial due to start this year."

About ImMucin

Vaxil's lead prod­uct, ImMucin, teaches the patient's immune system to identify and destroy cells which display a short specific 21-mer portion (Signal peptide domain) from the cancer target (marker) MUC1, which appears on 90% of all cancers cells but not in patients' blood, a factor which can en­hance its potency. In 2013, Vaxil com­pleted a Phase I/II clin­i­cal study with ImMucin on MM patients which showed high safety profile, strong di­vers­i­fied T/B-cell immunity in all 15 patients across MHC repertoire and initial indi­ca­tions for clin­i­cal efficacy; 11 out of the 15 patients demonstrating stable dis­ease or clin­i­cal im­prove­ment which did not require any further treat­ment. A follow-on study (still ongoing) on patients who responded clin­i­cally to ImMucin has shown that some patients are more than 4 years without requiring any further treat­ment for their dis­ease. ImMucin is also being eval­u­ated in a Phase I/II study to treat patients with metastatic breast cancer who are also receiving 1st first line hormone ther­apy.

About MM

MM is the second most common blood cancer; where plasma cells (the tumor) accumulate in bone marrow leading to bone destruction and marrow failure. MM accounts for 1% of all cancers and <10% of all hema­to­log­i­cal malig­nan­cies. Recent introduction of au­tol­o­gous stem-cell trans­plan­ta­tion and the avail­a­bil­ity of agents such as immunomodulators and pro­te­a­some inhibitors have changed the man­agement of MM. However, for most patients it still remains an incurable dis­ease.

Source: Vaxil Bio.