US-based Ra Pharmaceuticals has begun dosing patients in the Phase II clinical programme of its RA101495 for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).

RA101495 is being developed as a subcutaneous complement component 5 (C5) inhibitor to control complement-induced hemolysis.

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The Phase II programme is currently being conducted to assess the safety, tolerability, preliminary efficacy, pharmacokinetics and pharmacodynamics of RA101495, and includes two open-label Phase II trials.

One trial is being performed in the US and will include up to six to eight patients who are treated with eculizumab but showed an inadequate response.

The programme's other Phase II trial is being conducted outside the US and is designed to evaluate between eight and 12 eculizumab-naive patients, as well as six to eight patients who will switch from eculizumab to RA101495.

"RA101495 is a convenient, once-daily, self-administered subcutaneous dose and represents a significant advancement in the treatment of PNH."

Ra Pharma president and chief executive officer Doug Treco said: “Commencement of dosing in our Phase II clinical programme for RA101495 in PNH is an important step in bringing a potential new therapy to patients with this rare and life-threatening blood disorder.

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"RA101495 is a convenient, once-daily, self-administered subcutaneous dose and represents a significant advancement in the treatment of PNH."

Both trials will include the administration of 0.3mg/kg RA101495 SC loading dose and later daily 0.1mg/kg SC.

Their primary endpoint is a change in lactate dehydrogenase (LDH) from baseline.

It is expected that the initial data from the Phase II programme will be available by mid-year and additional data by year-end.