Alnylam Pharmaceuticals has commenced a Phase I study of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).

ALN-TTRsc, a siRNA conjugated to a GalNAc ligand that enables receptor-mediated delivery to the liver, is the company’s first subcutaneously delivered systemic RNAi therapeutic to enter clinical development.

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Alnylam executive vice president and chief medical officer Dr Akshay Vaishnaw said RNAi therapeutics demonstrated rapid, potent and durable knockdown of disease-causing the protein TTR.

"ALN-TTRsc, a siRNA conjugated to a GalNAc ligand that enables receptor-mediated delivery to the liver, is the company’s first subcutaneously delivered systemic RNAi therapeutic to enter clinical development."

"We are advancing what we believe to be the industry leading effort in ATTR; this includes ALN-TTRsc for the treatment of FAC and ALN-TTR02 for the treatment of FAP which is currently enrolling patients in a Phase II trial," Vaishnaw said.

The randomised, single and multi-dose, dose-escalation study that is being conducted in the UK will enrol 40 healthy volunteer subjects.

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The evaluation of the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc is the primary objective of the double-blind study.

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Assessment of clinical activity of the drug as measured by serum TTR levels are the secondary objectives.

University College London Medical School, Centre for Amyloidosis and Acute Phase Proteins head Professor Philip Hawkins said; "RNAi therapeutics are a novel and compelling approach for the treatment of ATTR, as this novel modality has been shown to achieve robust knockdown of serum levels of both wild-type and mutant TTR."

Alnylam expects to report data from the placebo-controlled trial in mid-2013, subsequent to which a Phase II study of ALN-TTRsc in FAC patients is scheduled for commencement by the end of 2013.

Upon obtaining a positive outcome, a pivotal trial for ALN-TTRsc in FAC patients will be conducted in 2014.