Australian biotech progresses with orphan multiple myeloma candidate
This article was originally published in Scrip
Australian therapeutic antibody company Immune System Therapeutics (IST) is expanding the Phase II trial of its lead anticancer candidate MDX-1097 in Australia following encouraging responses from the first dosed patients and a positive review of safety and efficacy data by an independent data monitoring committee. The product has also received orphan drug designation from the US FDA, the company noted.
CEO Alan Liddle said the US news was "another positive step towards achieving our business goal of developing an effective treatment for patients suffering from multiple myeloma". IST is currently in discussions aimed at "putting in place a robust global licensing structure that will expedite the clinical and regulatory processes" needed to commercialise the treatment.
IST's lead candidate was derived from technology spun out from the University of Technology, Sydney (UTS) in Australia and developed for therapeutic use in multiple myeloma by IST. "The antibody was manufactured for clinical use by Medarex and is now wholly owned by IST," Mr Liddle told Scrip. MDX-1097 is a chimeric antibody (70% human, 30% mouse).
IST intends to have a partner for the next phase of development "which will most likely be global Phase IIb trial in multiple myeloma", Mr Liddle added.
"The current Phase II study was designed as a Simon two stage minimax study and based on statistical criteria, one response was required in the first 13 patients to expand the study to 27 patients. One very good partial response was observed in the first six patients and no adverse events were recorded therefore the data monitoring committee recommended that the study expand to 27 patients."
According to Mr Liddle, IST has enough funds to complete the ongoing Phase II study. "We expect to have licensed MDX-1097 by the end of this year and therefore will conduct a Phase IIb or Phase III trial with our pharmaceutical partner."
There are two types of multiple myeloma: kappa type (around 70% of patients) and lambda type (30% of patients). MDX-1097 targets the kappa-type.
"IST [also] has a fully human antibody in development to the lambda myeloma antigen which addresses the other 30% of multiple myeloma patients, and is developing a fully human antibody to the kappa myeloma antigen," said Mr Liddle.