The FDA approval follows similar recognition for ImMucin from the EMA. This approval dem­onstrates the FDA's acknowledgement of an existing medical need for treat­ment of multiple myeloma and ImMucin's poten­tial in treating this type of cancer.

Ness Ziona, Israel (Press Release) – Vaxil Bio, (TASE: VAXL) a com­pany specializing in the devel­op­ment of immuno­therapy-based drugs, reports today that its lead prod­uct, ImMucin, has been granted an "orphan drug" desig­na­tion by the US Food and Drug Admin­istra­tion (FDA), for the treat­ment of multiple myeloma (MM).

ImMucin is an immuno­therapeutic treat­ment which educates the MM patient's immune system, particularly T-cells and anti­bodies, to attack MM cancer cells via a specific domain, termed signal peptide, of the tumor marker MUC1. This domain bears sig­nif­i­cant advantages as an immuno­therapeutic modality. Immunotherapy as a cancer treat­ment modality has become an important field of medicine during the last two years. It has been the subject of great interest, as drugs that utilize the mech­a­nisms of the immune system have been shown to produce results with far-reaching consequences, and have turned out to be "game changers" in the treat­ment of this disease.

"Orphan drug" desig­na­tion is granted by the FDA in order to promote the clin­i­cal devel­op­ment of drugs and treat­ments with sig­nif­i­cant treat­ment poten­tial against rare (fewer than 200,000 people in the US) life-threatening diseases. "Orphan drug" desig­na­tion grants the exclusivity to market in the US for 7 years from the moment the approval is provided.

Dr. Lior Carmon, CEO and founder of Vaxil: "Despite notable recent ad­vances in the treat­ment of MM, there are still no options resulting in full re­cov­ery from the disease. This is why there is a need for drugs that can delay the recurrence of the disease. ImMucin is intended to meet this need in MM patients with "minimal residual disease", turning their cancer into a chronic man­ageable disease. In light of the clin­i­cal results achieved thus far with ImMucin, manifested by a strong di­vers­i­fied immune response, we believe that ImMucin has great thera­peutic and economic poten­tial.

We have witnessed the growth of the immuno­therapy sector and the great interest pharma­ceu­tical giants have expressed in the promising clin­i­cal results of drugs that activate the immune system. Therefore, the FDA approval for ImMucin as an orphan drug, as well as similar approval by the European EMA, is an important mile­stone in the devel­op­ment of this unique treat­ment, both on regu­la­tory and business levels."

About ImMucin

Vaxil's lead prod­uct, ImMucin, trains the patient's immune system to identify and destroy cells which display a short specific 21-mer portion (signal peptide domain) of the cancer-associated (marker) ex­pres­sion of MUC1, which appears on 90% of all cancer cells but not in patient blood, a factor which can en­hance its potency. Vaxil com­pleted a Phase I/II clin­i­cal study with ImMucin in MM patients, which showed a high safety profile, strong di­vers­i­fied T/B-cell immunity in all 15 patients across MHC repertoires and initial indi­ca­tions of clin­i­cal efficacy; 11 out of the 15 treated patients dem­onstrated stable disease or clin­i­cal im­prove­ment which did not require any further treat­ment. An ongoing follow-up study in patients who responded clin­i­cally to ImMucin has shown that some patients haven't required any further treat­ment for their disease in the four years since ImMucin treat­ment. ImMucin is also being eval­u­ated in a Phase I/II study for the treat­ment of patients with metastatic breast cancer who are also receiving 1st first-line hormone ther­apy.

About MM

MM is the second-most common blood cancer, in which plasma cells 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 ad­vanced the man­agement of MM. However, for most patients it still remains as an incurable disease.

About Vaxil

Vaxil is an Israel-based com­pany specializing in the devel­op­ment of immuno­therapy prod­ucts for the treat­ment of cancer and infectious diseases by activating the patient's immune system. The com­pany's prod­ucts are based on the pro­pri­e­tary VaxHit tech­nology, which enables the identi­fi­ca­tion of sequences (antigens) in a unique domain of the proteins called the signal peptide that is capable of triggering a unique and specific reac­tion, among T -cells and anti­bodies (B-cells) in the immune system of most patients.

Recently, Vaxil has reported that by using the VaxHit tech­nology, one can identify targets within the same specific domain in proteins, and thus produce im­proved recombinant anti­bodies that can serve to diagnose and poten­tially treat cancer and infectious diseases.

Vaxil BioTherapeutics Ltd. was founded in 2006 by Dr. Lior Carmon, an expert in immuno­therapy of cancer and doctor of tumor immunology from the Weizmann Institute of Science.

Link to film about the com­pany's tech­nology: https://www.youtube.com/watch?v=XhEwj6Ue13s

Source: Vaxil Bio.