In big news for Northern Ireland-headquartered drug discovery group Almac, US regulators have awarded orphan drug status to the firm’s experimental therapy for ovarian cancer.
ALM201 is a first-in-class therapeutic peptide developed to mimic some of the properties of the naturally occurring protein FKBPL, known to have an effect on a number of important tumour biology processes, including cancer stem cells and angiogenesis.
According to the company, the peptide has “extremely potent anti-angiogenic activity, inhibiting migration, tubule formation and microvessel formation in vitro and in vivo”, and is very effective in mouse xenograft models at low doses without affecting proliferation.
Ovarian cancer ranks among the top ten diagnosed and top five deadliest cancers in most countries, but around 80 percent of patients present with advanced disease.
Being assigned Orphan Statue “will allow for valuable assistance from the FDA during clinical development for either ourselves or our partners for this difficult to treat patient population,” said Dr Stephen Barr, managing director and president of the group.