Arrowhead Pharmaceuticals has dosed the first participants in two Phase I/IIa clinical trials of pulmonary candidates, ARO-MUC5AC and ARO-RAGE, to potentially treat various muco-obstructive and inflammatory pulmonary diseases, respectively.

ARO-MUC5AC and ARO-RAGE are two investigational ribonucleic acid interference (RNAi) therapeutics intended to lower the production of mucin 5AC (MUC5AC) and the receptor for advanced glycation end products (RAGE).

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One of the trials is named AROMUC5AC-1001. This double-blinded, randomised, placebo-controlled trial will enrol up to 42 normal healthy volunteers (NHV) and up to 16 moderate-to-severe asthma patients.

The single ascending dose portion of the trial will comprise three sequentially enrolled NHV cohorts with escalating single-dose levels. 

Three NHV cohorts and two asthma patient cohorts will comprise the multiple ascending dose portion of the trial.

Analysing the safety and tolerability, pharmacokinetics and pharmacodynamics of ARO-MUC5AC in NHVs and asthma patients are the goals of the trial.

The other study is a Phase I/IIa double-blinded, placebo-controlled, randomised ARORAGE-1001 trial enrolling up to 64 healthy subjects and up to 16 mild-to-moderate asthma patients. 

The single ascending dose portion of this trial will have four sequentially enrolled NHV cohorts as well as an optional cohort receiving escalating single-dose levels. 

Its multiple ascending dose portion will include four NHV cohorts and two asthma patient cohorts. 

Evaluating the safety and tolerability, pharmacokinetics and pharmacodynamics of ARO-RAGE in NHVs and asthma patients are the objectives of this trial. 

Arrowhead Pharmaceuticals president and CEO Chris Anzalone said: “Initiating clinical studies for ARO-MUC5AC and ARO-RAGE, Arrowhead’s next-generation pulmonary targeted RNAi therapeutic candidates, is a significant milestone for the company. 

“These are potentially important new medicines that address targets that have been difficult to drug using other modalities and are designed to treat muco-obstructive and inflammatory lung diseases in fundamentally new ways.”

In February this year, the company concluded the participant enrolment as planned in the Phase IIb trial of ARO-ANG3 for treating mixed dyslipidemia patients.