- Alnylam Pharmaceuticals said its genetic medicine for a type of kidney condition succeeded in a Phase 2 trial, reporting Thursday that the experimental drug reduced signs of disease after 32 weeks of treatment when used on top of regular therapy.
- The data will help the company develop its plans for a Phase 3 trial, in collaboration with partner Regeneron. The Food and Drug Administration last year gave accelerated approval to a drug for the same condition, called IgA nephropathy, based on a bigger trial but using the same measurement of urinary protein that Alnylam’s Phase 2 trial did.
- If successful, Alnylam could find itself competing with up to three other drugs in IgA nephropathy, as two experimental pills are currently in late-stage studies. Wall Street analysts point to another Alnylam trial, which could substantially expand use of its marketed product Onpattro, as a bigger event this year. Results are expected imminently.
Alnylam has achieved something few biotechs manage to do: launching not just one but four products, all while managing to retain its independence. But in spite of its clinical and commercial success, the company still loses money, amounting to $852 million in 2021 and $240 million in the first three months of 2022.
That picture could change with growing sales and success from other research projects, on which its spent nearly $800 million in 2021. A drug called cemdisiran is a part of that outlook, treating a kidney condition that affects an estimated 130,000 people in the U.S.
The Phase 2 trial of cemdisiran selected 31 patients to either receive the drug or an inactive placebo, with a primary goal of measuring effects on urinary protein, a marker of disease progression, after eight months of treatment. Patients who received cemdisiran experienced, on average, a 37% reduction in urinary protein, though, notably, the trial wasn't structured to statistically compare how patients taking drug fared versus those who got placebo.
Urinary protein reduction was the trial goal Swedish biotech Calliditas Therapeutics used last year to win accelerated approval, in the U.S., of its steroid pill Tarpeyo. That drug reduced urinary protein 31% when compared to a placebo and is now the first and, currently, only marketed treatment for IGA nephropathy proven to do so.
Two other drugs — Travere Therapeutics’ sparsentan and Chinook Therapeutics’ atrasentan — are in late-stage testing for IgA nephropathy. Like Tarpeyo, those are drugs taken orally every day, while cemdisiran, which blocks production of an immunological protein by “silencing” RNA, is an injection taken once every four weeks.
With competition for cemdisiran is likely to be sharp, Alnylam investors are looking more closely at an upcoming trial readout for its marketed drug Onpattro, which treats the nervous system complications of an inherited disease called transthyretin amyloidosis.
That trial, called APOLLO-B, could allow Alnylam to expand into patients suffering from cardiac complications, which only Pfizer’s drugs Vyndamax and Vyndaqel treat. Those two drugs earned Pfizer more than $2 billion in 2021, compared with the $475 million in Onpattro revenue that Alnylam recorded.