The focus is on how news of a securities fraud investigation involving Alnylam Pharmaceuticals, together with downward revisions to the company's earnings outlook, will affect investors.
Alnylam Pharmaceuticals may continue to create new treatments built on its RNAi platform and broad rare disease portfolio, supported by the stability of its TTR franchise. In the short term, the market will focus on the company's execution of late-stage clinical programs such as zilebesiran and nucresiran. These programs are viewed as factors that could affect the future of AMVUTTRA and the ATTR earnings base.
The largest business risk, however, is the concentration in the TTR franchise. The downward revision to the 2026 earnings forecast and the securities fraud investigation have drawn attention to the scale and predictability of earnings from ATTR. Some believe that heightened payer pressure could test the high P/E multiple.
Nucresiran targets ATTR cardiomyopathy in the large TRITON CM trial and is said to have reduced the median level by approximately 95%. If data supporting broad use are obtained, it could become a successor to AMVUTTRA, and investors are placing considerable importance on this possibility.
Current analyst forecasts call for approximately $8.8 billion in revenue and approximately $1.7 billion in profit in 2029, with annual growth and expanding profitability expected. At the same time, some believe that the wide divergence in fair value estimates means uncertainty remains around the share price.
From another perspective, some investors have produced estimates showing revenue in 2029 far below that level, prompting calls for a multifaceted assessment. The profitability of the TTR portfolio could be significantly affected by the success or failure of the nucresiran launch.