- An experimental drug from Alnylam Pharmaceuticals significantly lowered levels of a tissue-damaging chemical in patients with a rare kidney disease, detailed data released by the company Sunday at a virtual medical meeting showed.
- The treatment, known as lumasiran, is now under review at the Food and Drug Administration, which has set a Dec. 3 deadline for making a decision on approval. If cleared, lumasiran would become Alnylam's third marketed drug after Onpattro and Givlaari.
- The company estimates between 3,000 and 5,000 patients have primary hyperoxaluria type 1, the disease lumasiran treats. While the results could set up Alnylam for an approval, it's not the only company developing a treatment; Dicerna Pharmaceuticals has a competing drug now in late-stage trials.
Alnylam is one of the leaders in a field of medicine that uses interfering nucleic acids to disrupt cellular instructions sent by misfiring genes.
In the case of primary hyperoxaluria type 1, lumasiran helps to lower production of oxalate, elevated levels of which can lead to kidney stones and, over the long term, can damage and destroy kidneys.
In Alnylam's study, called ILLUMINATE-A, six months of treatment with lumasiran led to oxalate levels 65% lower than when patients began the trial, the company said. Patients who received a placebo saw their oxalate levels drop by only 12%.
Alnylam said last December the trial had succeeded, but didn't release detailed data then.
While the trial hasn't shown that use of lumasiran will avert kidney failure, the FDA frequently approves drugs in rare, genetically driven diseases based on biomarker data like reduced oxalate. Detecting a difference in outcomes like organ failure often takes years and requires many patients — ILLUMINATE-A enrolled just 39 patients.
On one endpoint analyzed in this trial, the occurrence of kidney stones, lumasiran treatment didn't result in any improvement relative to placebo.
Five of the 26 patients taking the drug had kidney stones, and two of the 13 on placebo did. With small numbers enrolled in the trial and the short six-month time frame, that endpoint can only be considered "exploratory," meaning any comparison is not statistically strong.
In a note to clients, Cantor Fitzgerald analyst Alethia Young wrote that "the full dataset reinforced our confidence in the strong efficacy" of lumasiran. "We do not expect any controversy around the profile of this drug," she added.
With an FDA decision on lumasiran due by December, Alnylam could have a decent head start on Dicerna, which has yet to complete enrollment in its trial of drug nedosiran, which has a similar design to ILLUMINATE-A.
One commercial advantage nedosiran could have, however, is that it treats all three types of primary hyperoxaluria.
Alnylam's two marketed drugs, Onpattro and Givlaari, treat conditions where patients number in the thousands and often go undiagnosed for years, making patient awareness a key component of any commercial campaign. The same may be the case with lumasiran, as Alnylam is building an awareness campaign around patients with kidney stones.
Young said she expects the launch will be "slow and steady." Onpattro and Givlaari had sales of $67 million and $5 million, respectively, in the first quarter of 2020.