US-based biotechnology company Achillion Pharmaceuticals has commenced dosing patients in a Phase II clinical trial of ACH-4471 for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).

ACH-4471 is an orally administered, small-molecule inhibitor of factor D with potential to address intravascular and extravascular hemolysis by blocking the amplification loop of the complement alternative pathway.

The proof-of-concept Phase II trial is designed to evaluate the safety, efficacy and pharmacokinetics of ACH-4471 in untreated patients.

Achillion president and chief executive officer Milind Deshpande said: “We are pleased to have begun this trial in PNH patients to evaluate ACH-4471, the first orally administered factor D inhibitor to have demonstrated complement alternative pathway (AP) inhibition in humans, which represents a potentially novel and unique mechanistic approach to treating this life-threatening disease."

"The proof-of-concept Phase II trial is designed to evaluate the safety, efficacy and pharmacokinetics of ACH-4471 in untreated patients."

The trial design includes intra-patient dose-escalation from an initial 100mg administered thrice daily.

The primary objective of the trial is measure of the change in the levels of intravascular hemolysis' sensitive biomarker known as serum lactate dehydrogenase (LDH), through 28 days of dosing.

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The trial will also examine secondary endpoints such as changes in levels of haemoglobin and red blood cells, and complement pathway biomarkers such as Bb and factor D.

It is expected that the trial's interim results will be available in the second quarter of this year.

Achillion plans to further investigate ACH-4471 in a Phase II trial to treat C3 glomerulopathy patients, alongside the study of extended release formulations to minimise the dosing frequency.