ALN-6400 Roundtable Frames Potential Non-TTR Blockbuster Opportunity

研报英文原文证据摘录

ALN-6400 Roundtable Frames Potential Non-TTR Blockbuster Opportunity

Flash Note Health Care | Biotechnology / SMID Cap

June 25, 2026

Alnylam Pharmaceuticals, Inc Cory Kasimov Adhi Sikand, PhD 917-733-5320 212-497-0813

ALNY | $294.06 cory.kasimov@evercoreisi.com adhi.sikand@evercoreisi.com

Outperform | Target Price/Base Case: $400.00 Noah Eisenberg Joshua Chazaro, MD

Company Update - Commentary 212-446-5622 917-750-5601

noah.eisenberg@evercoreisi.com joshua.chazaro@evercoreisi.com

ALNY investor frustration has been mounting as shares bounce around 52-wk lows. While most focus is still centered

on the TTR narrative (Amvuttra Rx, FY26 revenue, Cardio-TTRansform), the lack of confidence in the company’s non-

TTR pipeline is a key issue. To that end, the first of several RNAi R&D Roundtables today was a helpful attempt to

better inform investors on this front. The event focused on ALN-6400, a compelling candidate for two large, currently

untapped bleeding disorders. Overall, ALN-6400 has the potential to help ALNY meet its longer-term goal of 2+

non-TTR medicines with blockbuster potential but likely needs meaningful clinical momentum before investors

get more constructive.

• ALN-6400 is a liver-directed, PLG-lowering siRNA designed to reduce fibrinolysis/clot breakdown and provide

durable prophylactic bleed protection. The MoA makes sense, particularly because PLG lowering is meant

to stabilize clot without increasing thrombin generation, directly addressing a key hematologist concern

around thrombosis.

• Importantly, a single SC dose generated a deep anti-fibrinolytic signal out to ~3 mos vs hours for oral TXA,

supporting the potential for quarterly dosing. That durability matters, particularly in VWD, where current

prophylaxis can require 24-36 IV infusions per quarter.