Alnylam Doses First Patient in Phase 1 Study of ALN-AGT, an Investigational RNAi Therapeutic for the Treatment of Hypertension

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the first patient has been dosed in the Company’s Phase 1 study of ALN-AGT, an investigational RNAi therapeutic targeting angiotensinogen (AGT) for the treatment of hypertension in high unmet need populations, including patients with resistant or refractory hypertension, chronic kidney disease or heart failure. The Company expects to report initial results in late 2019.

“We are pleased to have initiated dosing in this Phase 1 study with the goal of evaluating the safety and preliminary pharmacokinetic and pharmacodynamic activity of ALN-AGT in patients with hypertension,” said Lauren Melton, Senior Director, Program Leader, ALN-AGT program at Alnylam. “We believe RNAi-mediated angiotensinogen silencing represents a novel and targeted approach with the potential to offer robust and highly durable control of blood pressure for patients with resistant or refractory hypertension.”

The Phase 1 study is a multi-center, randomized, double-blind, placebo-controlled trial designed to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of subcutaneously administered ALN-AGT in patients with essential hypertension. The study will be conducted in four parts: Part A: single ascending dose phase in hypertensive patients; Part B: single dose in hypertensive patients with controlled salt intake; Part C: multi-dose phase in hypertensive patients; and Part D: multi-dose phase in hypertensive patients who are obese. In Parts C and D, once daily oral doses of irbesartan (angiotensin II receptor blocker) will be used as the active comparator. Patients will be randomized 2:1 ALN-AGT to placebo or ALN-AGT to irbesartan. The planned enrollment for this study, including optional cohorts, is up to 168 patients.

About ALN-AGT
ALN-AGT is an investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen (AGT) in development for the treatment of hypertension in high unmet need populations. ALN-AGT utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables subcutaneous dosing with increased selectivity and a wide therapeutic index. The safety and efficacy of ALN-AGT have not been evaluated by the FDA, EMA or any other health authority.