Alnylam Announces Positive Early Results on Clinical Outcome Measures from ILLUMINATE-A Phase 3 Study of OXLUMO (lumasiran) - Seite 2

Results

Among 24 patients in ILLUMINATE-A who had been treated with lumasiran for 12 months and had valid renal ultrasounds at baseline, 46 percent (11/24) of patients experienced improvement in nephrocalcinosis grade relative to baseline, 17 percent (4/24) remained stable, and 13 percent (3/24) experienced worsening; 25 percent (6/24) did not have ultrasounds available at 12 months. Of 14 patients with baseline nephrocalcinosis and available ultrasounds, 79 percent (11/14) showed improvement relative to baseline, and of those who improved, 73 percent (8/11) improved in both kidneys. Additional data were presented regarding effects on estimated glomerular filtration rate (eGFR) and kidney stone events.

To view the full results presented at the virtual ASPN congress, please visit www.alnylam.com/capella.

IMPORTANT SAFETY INFORMATION

Adverse Reactions

The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%). Symptoms included erythema, pain, pruritus, and swelling.

Pregnancy and Lactation

No data are available on the use of OXLUMO in pregnant women. No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production. Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for OXLUMO and any potential adverse effects on the breastfed child from OXLUMO or the underlying maternal condition.

For additional information about OXLUMO, please see the full Prescribing Information.

About OXLUMO (lumasiran)

OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. OXLUMO works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. Injection site reactions (ISRs) were the most common drug-related adverse reaction. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent with that observed in ILLUMINATE-A. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology designed to increase potency and durability. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly thereafter at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. OXLUMO should be administered by a healthcare professional. For more information about OXLUMO, visit OXLUMO.com.