Фармкомпания Argo Biopharmaceutical показала эффективность новой терапии при наследственном ангионевротическом отеке

Argo Biopharmaceutical has debuted new data from a Phase II study on its preventative therapy, BW-20805 in hereditary angioedema (HAE), that the company says highlights its "differentiated clinical profile" in this condition. Through a multi-national, open-label trial (NCT06846398), Argo set out to establish how BW-20805 acts in the body, while evaluating its potential effectiveness in reducing swelling episodes in 25 patients with HAE. According to updated data from the study, which was presented at the ongoing Berlin-held Bradykinin Symposium, 300mg of BW-20805 dosed once every 24 weeks was able to reduce the average monthly rate of HAE attacks by 96%. This positive impact on HAE episodes was also seen at all other tested doses, as patients given a 600mg dose every 24 weeks or a 300mg dose every 12 weeks experienced an 83% and 93% drop in attack rates, respectively. BW-20805 also markedly reduced the frequency and severity of HAE attacks, with between 50% and 75% of patients remaining attack-free between day 29 and day 169 depending on dosage. On top of its promising signs of early efficacy, researchers uncovered BW-20805's favourable pharmacodynamic activity, with the onset of the drug's activity being rapid and sustained over the study period. Patients also tolerated BW-20805 well, with the majority of treatment-emergent adverse events (TEAEs) being mild in severity, with many being injection site reactions. No TEAEs led to discontinuation or withdrawal from treatment. HAE is a genetic disorder that causes unpredictable, recurring episodes of severe swelling in the body's tissues. It affects roughly one in every 50,000 people globally, and most commonly impacts the hands, feet, face, genitals and the intestinal tract. BW-20805 is a silent interfering RNA (siRNA)-based therapy designed to target the mRNA behind the plasma prekallikrein protein (PKK), which plays a key role in driving the uncontrolled inflammatory response – and thus swelling attacks. By durably disabling PKK's mRNA, Argo claims that BW-20805 could address the root cause of the condition with less frequent dosing. Cracking the competitive HAE market If approved, BW-20805 would enter an increasingly crowded HAE market, which has recently seen several preventative and acute targeted therapeutics. According to GlobalData's Pharmaceutical Intelligence Center, the current best seller in HAE is Takeda's blockbuster preventative therapy, Takhzyro (lanadelumab), which was one of the centrepieces of the Japanese pharma's $62bn takeover bid for Irish pharma company, Shire Therapeutics, back in 2019.