Alnylam defends RNAi drugs after AstraZeneca
Alnylam defends its heart disease RNAi drugs after AstraZeneca's Wainua failed a trial. The company says its drugs work even with stabilizer therapies.
Alnylam Pharmaceuticals is defending its class of heart disease drugs after the failure of a rival therapy from AstraZeneca. AstraZeneca reported on Friday that its drug Wainua did not succeed in a clinical trial for the heart condition ATTR-CM, a form of transthyretin amyloidosis.
Some analysts saw the result as a negative signal for all 'silencer' drugs, the type of medicine Alnylam sells. Their concern is that silencers may offer no added benefit if patients are already taking a different class of treatment called 'stabilizers.' This interpretation suggests stabilizers might be the superior approach. Alnylam is now publicly countering that view.
According to Alnylam, the failure of AstraZeneca's candidate does not reflect on the value of its own products. The company's response has direct implications for its currently marketed drug, Amvuttra, and for its next-generation treatment, nucresiran. Both are silencer-type therapies.
The contested mechanism of action
The debate centers on two distinct drug mechanisms for treating ATTR-CM. Stabilizer drugs, like Pfizer's Vyndaqel, work by keeping a problematic protein from misfolding. Silencer drugs, which use RNA interference (RNAi) technology, aim to reduce the production of that protein in the first place. Alnylam argues these mechanisms are complementary, not mutually exclusive.
Some analysts had speculated that the stabilizer mechanism might be fundamentally more effective. Alnylam rejects this. The company contends that its silencer drugs can provide significant benefit regardless of a patient's background therapy.
Alnylam's case for its products
Alnylam's defense rests on data from its own clinical programs. The company points to evidence supporting Amvuttra and its pipeline drug nucresiran. It maintains that the AstraZeneca outcome is specific to that company's drug and trial design.
Crucially, Alnylam asserts that the analyst interpretation is incorrect. The failure of one silencer does not condemn the entire class, the company says. This position is vital for investor confidence in Alnylam's future.
The company's marketed drug, Amvuttra, is already approved for a related liver form of ATTR. Its success in the cardiac form is a major commercial goal. The next-generation nucresiran is designed for less frequent dosing, a potential advantage.
The commercial stakes
The financial implications are substantial. The market for ATTR-CM treatments is valuable and competitive. Pfizer's stabilizer drug Vyndaqel has established a strong position. Alnylam's RNAi drugs aim to capture a significant share.
The analyst concerns threatened to undermine that ambition. If doctors believe stabilizers alone are sufficient, they may not prescribe add-on silencer therapies. Alnylam's public rebuttal is an attempt to protect its market opportunity.
The story was reported by STAT+, a specialist publication. Alnylam's arguments are now part of the ongoing scientific and commercial discussion. The company's next steps will involve generating more data to support its case.
