Second Positive Phase 2 Trial Boosts Confidence in Alnylam’s RNAi Blood Pressure Med, Supporting Roche’s $2.8 Billion Biobucks Bet

1. A second successful Phase 2 trial of Alnylam's RNAi blood pressure medication, zilebesiran, has bolstered Roche's confidence in their $310 million investment and the potential for up to $2.8 billion in milestone payments.
2. The KARDIA-2 study involved 672 adults with mild to moderate hypertension receiving zilebesiran alongside existing hypertension drugs like Pfizer's Norvasc, Daiichi Sankyo's Benicar, or indapamide.
3. Patients experienced a clinically and statistically significant reduction in systolic blood pressure using 24-hour ambulatory blood pressure monitoring at a three-month assessment.
4. Safety and tolerability profiles of zilebesiran appear encouraging, according to Alnylam.
5. Full results of the KARDIA-2 study are scheduled to be presented at a scientific conference next month.
6. Zilebesiran is a subcutaneously administered RNAi therapeutic targeting liver-expressed angiotensinogen.
7. Roche has the option to spearhead the development of zilebesiran for future indications beyond hypertension.
8. A new Phase 2 study, KARDIA-3, has been initiated to assess zilebesiran in patients with high cardiovascular risk and uncontrolled hypertension despite treatment with multiple approved antihypertensive medications.
9. Zilebesiran previously demonstrated success in the midstage KARDIA-1 study, with both 300-mg and 600-mg doses producing a more than 15-mmHg mean reduction in 24-hour systolic blood pressure after three months compared to placebo.
10. Investors are also focused on Alnylam's vutrisiran, an RNAi treatment in the phase 3 HELIOS-B trial for hereditary transthyretin-mediated (hATTR) amyloidosis, with results expected in June or July.