Alnylam Pharmaceuticals (ALNY) Investor update summary

Strategic pipeline and innovation overview

• RNAi platform enables highly specific, potent, and durable gene silencing, supporting infrequent dosing and broad disease targeting.

• Over 25 programs span rare and prevalent diseases, with a focus on high morbidity and mortality indications.

• Cardiovascular disease portfolio targets obesity, diabetes, hypercholesterolemia, and hypertension.

• Multiple pivotal studies and key data readouts are expected through 2028, driving clinical momentum.

Industry context and unmet need

• Hypertension affects over 1 billion people globally, with prevalence rising due to aging and obesity.

• Cardiovascular deaths attributable to high systolic blood pressure are projected to increase by 62% by 2050.

• Only about 20% of hypertensive patients achieve blood pressure control, with poor adherence and therapeutic inertia as major barriers.

• Uncontrolled hypertension drives significant morbidity, mortality, and economic burden, with annual U.S. costs estimated at $292B and projected to exceed $500B globally by 2050.

Limitations of current therapies

• Despite multiple oral antihypertensive classes, up to 80% of patients remain uncontrolled, largely due to poor adherence and daily dosing requirements.

• Visit-to-visit blood pressure variability and nocturnal hypertension contribute to ongoing cardiovascular risk, even when office BP appears controlled.

• Most high-risk patients spend a low proportion of time in target BP range, which is an independent predictor of major adverse cardiovascular events.