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news.Israeli clinical stage biotechnology firm Vaxil Bio has received orphan drug designation from the European Medicines Agency (EMA) for ImMucin, to treat multiple myeloma (MM).
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The company’s lead drug candidate, ImMucin, targets the less studied Signal Peptide domain of the MUC1 tumor antigen, which harbours key advantages as an anti-MM modality.
ImMucin teaches the patient’s immune system to identify and destroy cells which display a short specific 21-mer portion from the cancer target MUC1.
MUC1 appears on 90% of all cancer cells but not in patients’ blood, a factor which can improve its potency.
Data from the company’s Phase I/II clinical trial with ImMucin on MM patients showed high safety profile, strong diversified T/B-cell immunity in all 15 patients across MHC repertoire and initial indications for clinical efficacy.
Under the trial, 11 out of the 15 patients showed stable disease or clinical improvement which did not require any further treatment.
Vaxil founder and CEO Dr Lior Carmon said: "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."
The company said that a follow-on study (still ongoing) on patients who responded clinically to ImMucin has shown some patients surviving more than four years without the need for further treatment.
Currently, ImMucin is also being evaluated in a Phase I/II trial to treat patients with metastatic breast cancer who are also receiving first line hormone therapy.
According to the company, a multi-center phase II clinical trial under IND is scheduled to be initiated in patients with multiple myeloma during 2015.