ImMucin Granted Orphan Drug Designation for Multiple Myeloma

An immunotherapeutic treatment known as ImMucin is getting moved up in the drug development pipeline. On June 22, 2015, the US Food and Drug Administration (FDA) granted an Orphan Drug Designation for the treatment of multiple myeloma (MM). The FDA approval follows similar recognition for ImMucin from the European Medicines Agency (EMA), and may mean this agent could be available in the not too distant future.

“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,” said Lior Carmon, PhD, MBA, who is the CEO and founder of Vaxil BioTherapeutics Ltd., Ness Ziona, Israel.

Dr. Carmon said based on preliminary studies by his company, ImMucin appears to produce a strong diversified immune response. 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 expression of MUC1. The expression of MUC1 appears on 90% of all cancer cells, but not in patient blood, a factor which can enhance its potency, according to Vaxil BioTherapeutics.

A recently completed phase I/II clinical study showed a high safety profile and strong diversified T/B-cell immunity in all 15 patients in the study across MHC repertoires, and initial indications of clinical efficacy.  The study showed that 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 4 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 first-line hormone therapy.

Recent introduction of autologous stem cell transplantation and the availability of immunomodulators and proteasome inhibitors have advanced the management of MM. However, for most MM patients it still remains as an incurable disease. MM is the second most common blood cancer, and it accounts for 1% of all cancers.

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 life-threatening diseases (fewer than 200,000 people in the US). Orphan Drug Designation grants the exclusivity to market in the US for 7 years from the moment the approval is provided.

References:

• Vaxil BioTherapeutics Ltd. (2015). Vaxil’s lead product ImMucin has been granted an orphan drug designation by the FDA for the treatment of multiple myeloma.