Alnylam launches new clinical trial exploring TTR-mediated amyloidosis

Alnylam Pharmaceuticals has begun dosing in its Phase I clinical trial with ALN-TTRsc in a bid to explore treatments for TTR-mediated amyloidosis (ATTR)

ATTR is caused by mutations in the TTR gene, which leads to abnormal amyloid protein deposits accumulating in a number of tissues, such as the peripheral nerves and heart, which can lead to neuropathy or cardiomypathy.

The condition needs to be treated as soon as possible, as it leads to substantial morbidity and mortality, while familial amyloidotic cariomypathy impacts a minimum of 40,000 people across the world.

On the other hand, familial amyloidotic polyneuropathy affects at least 100,000 individuals globally, meaning laboratory work could make a big difference to people's health.

The ALN-TTRsc drug is a RNAi therapeutic that includes a Sirna conjugated to a GaINAc ligand, enabling receptor-mediated delivery to the liver.

The solution is the first GaINAc-siRNA and the first subcutaneously delivered, systemic RNAi therapeutic to be put into clinical development.

ALN-TTRs's Phase I trial is being carried out in Britain as a randomised, double-blind, placebo-controlled study, with the primary objective being to review the safety and tolerability of single and multiple doses of the drug.

Akshay Vaishnaw, executive vice president and chief medical officer of Alnylam, said: "RNAi therapeutics hold great promise for the treatment of ATTR since they have demonstrated rapid, potent, and durable knockdown of TTR, the disease-causing protein.

"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."

Philip Hawkins, professor of medicine at University College London Medical School, also commented on the study, noting that he is encouraged by the clinical and pre-clinical data recorded already in the ALN-TTR program.

Pre-clinical work has shown that subcutaneous administration of ALN-TTRsc has led to potent and sustained suppression of TTR.

Posted by Neil Clark