Alnylam Pharmaceuticals has started the Phase I / II clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) to treat AAT deficiency-associated liver disease (alpha-1 liver disease).

ALN-AAT is a subcutaneously administered investigational RNAi, which uses the firm’s ESC-GalNAc-siRNA conjugate delivery technology.

Initially, the study will be carried out in normal healthy volunteers and then in patients with alpha-1 liver disease. The trial is being conducted based on pre-clinical data presented during Digestive Disease Week (DDW).

Alnylam Pharmaceuticals R&D executive president and chief medical officer Dr Akshay Vaishnaw said: "We believe ALN-AAT has the potential to be a transformative therapy for patients with alpha-1 liver disease, an increasingly recognised clinical manifestation of alpha-1 antitrypsin deficiency where liver transplantation is the only available treatment option."

"We believe ALN-AAT has the potential to be a transformative therapy for patients with alpha-1 liver disease."

The trial is a randomised, single-blind and placebo-controlled study that is being carried out in three parts, including Part A, B and C.

Parts A and B are single-dose and multi-dose and dose-escalation studies that will enrol up to a total of 48 healthy adult volunteers.

GlobalData Strategic Intelligence

US Tariffs are shifting - will you react or anticipate?

Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.

By GlobalData

Part C will be a multi-dose study that will enrol up to a total of 24 adults with alpha-1 liver disease and mild-to-moderate liver fibrosis.

According to the firm, the primary objective of the study is to assess safety and tolerability of single and multiple subcutaneous doses of ALN-AAT and secondary objectives comprise evaluation of pharmacokinetics and clinical activity for ALN-AAT as measured by knockdown of serum AAT.

In January 2014, Alnylam partnered with Sanofi firm Genzyme to enhance the development and commercialisation of RNAi therapeutics worldwide.

Clinical Trials Arena Excellence Awards - Have you nominated?

Nominations are now open for the prestigious Clinical Trials Arena Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact. This is your chance to showcase your achievements, highlight industry advancements, and gain global recognition. Don't miss the opportunity to be honoured among the best - submit your nomination today!

Nominate Now