Regional Business Director - Ttr

Alnylam plans bigger study of RNA drug for Huntington's disease, despite failures in the field. Senior science correspondent. As with many of the most feared neurological conditions, Huntington's disease has proven to be a minefield for drug developers. Promising biomarker data Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.

Alnylam says Huntington's drug shows promise in early study. Gene silencing therapy is latest in a series of intriguing candidates for the fatal disease. Oct. 23, 2026 An experimental Huntington's drug from Alnylam Pharmaceuticals showed promise in a small study, the company said Friday, suggesting it can partially silence the mutant gene that drives the devastating neurological disorder. Patients who received the highest dose of the drug, called brezosiran, saw the levels of mutant huntingtin in their spinal fluid fall by 60% or 64% on average, depending on the type of assay used. The company described the safety profile as "encouraging" and said it planned to start a placebo-controlled study next year to prove the drug can actually slow the disease. "We're getting high levels of knockdown with good safety and tolerability, and so this is all very, very encouraging," said Pushkal Garg, head of research and development at Alnylam, which presented the data Friday at the European Huntington's Disease Network Clinical Research Congress. Your morning rundown of the science, politics, and money driving biotech today

Alnylam is now solidly profitable. Why are investors treating it like a broken biotech? This beaten-down biotech has a better story than its stock chart suggests. Key points. * Alnylam's stock has plunged more than 40% this year, but the company's financials look great. * Investors have been spooked by a guidance cut and a rival drug's clinical setback. * However, Wall Street remains bullish about Alnylam - for good reason. * Motley Fool Issues First-Ever 9X Buy Alert" It's been a horrible, lousy, rotten year so far for Alnylam Pharmaceuticals (ALNY -2.43%) investors. Feel free to add any other similar adjectives you'd like. Alnylam's share price has plunged more than 40% year to date. Only one other large-cap stock in the entire healthcare sector has performed worse. Alnylam's impressive 69% gain in 2025 has been almost completely erased. The drugmaker's financials look great. It's now solidly profitable. But investors are clearly treating Alnylam like it's a broken biotech stock. Just how profitable is Alnylam? Not long ago, Alnylam was hemorrhaging cash. The company posted net losses throughout 2024. As recently as the second quarter of last year, Alnylam's bottom line reflected a net loss of $16.2 million based on generally accepted accounting principles (GAAP). However, Alnylam has experienced a dramatic transformation in profitability since then. It recorded impressive earnings of $251 million in the third quarter of 2025. This total was significantly boosted by funds received from its collaboration with Roche (RHHBY +0.00%). But Alnylam remained profitable going forward, relying less on collaboration revenue. Sales skyrocketed for Amvuttra as it gained momentum in the transthyretin amyloid cardiomyopathy (ATTR-CM) market. Missed Nvidia in 2009? (This rare signal is flashing again). When the Total Conviction signal flashed for Nvidia in 2009, most people ignored it. The ones who acted turned $5,000 into $3,105,454*. Now, for the first time in years, that same "Total Conviction" signal is flashing for a company 1/100th the size of Nvidia. It's a key player in the $1.8 trillion space race, and with the stock recently sitting 20% off its highs, the window to get in early is closing fast. *Returns as of October 6, 2026. In the second quarter of 2026, Alnylam's GAAP net income totaled $164.5 million. Its adjusted earnings were much higher at $251.8 million. Meanwhile, the company's revenue soared 67% year over year to $1.29 billion. As icing on the cake, Alnylam's cash position (including cash, cash equivalents, and marketable securities) stood at $3.3 billion at the end of the quarter. ALNY - Alnylam Pharmaceuticals. $225.10 -$5.61 (-2.43%) NASDAQ · As of 4:00 PM ET 10/21/26 ALNY · YTD -43.75% Key data points. Market cap $30.87B Day's range $224.88 - $230.84 52wk range $197.81 - $485.36 Volume 1.2M Avg volume 1.7M Gross margin 79.65% Why Alnylam is being treated like a broken biotech. If you only looked at Alnylam's financial statements, you'd probably conclude that everything is sunshine and roses for the drugmaker. So why is Alnylam being treated like a broken biotech? There are two main reasons - and I think each of them is overstated. First, in Q2, Alnylam cut its 2026 full-year revenue guidance for Amvuttra and Onpattra. Management had projected sales of $4.4 billion to $4.7 billion but lowered the outlook to $4.2 billion to $4.5 billion. CEO Yvonne Greenstreet said that the change was due to demand normalization after an initial spike of previously untreated ATTR-CM patients switching to Amvuttra. Importantly, Amvuttra's sales continue to grow robustly. However, Alnylam's management team simply underestimated how the initial pent-up demand would turbocharge sales before stabilizing. The second reason behind Alnylam's sinking share price this year is concern about disappointing Phase 3 results for Wainua (eplontersen) in treating ATTR-CM. Wainua isn't Alnylam's drug, by the way: It's marketed by AstraZeneca (AZN -0.22%) and Ionis Pharmaceuticals (IONS -2.49%). However, some investors worried that Wainua's setback in ATTR-CM could lower the chances of success for Alnylam's nucresiran, which is currently in late-stage testing as a treatment for ATTR-CM and hereditary ATTR amyloidosis with polyneuropathy (ATTR-PN). To be sure, nucresiran might run into similar problems as Wainua. However, the two drugs use different mechanisms. Their clinical trials were designed differently. If Alnylam sees something in the details of the Wainua ATTR-CM study, it could adjust its nucresiran study, if appropriate. A better story than the stock chart suggests. While many investors view Alnylam as broken, Wall Street doesn't. Of the 23 analysts who have rated the stock over the past three months, 19 recommended buying it. The average 12-month price target for Alnylam reflects a potential upside of 54%. Piper Sandler's (PIPR +0.39%) Edward Tenthodd even thinks the stock could more than double over the next 12 months. Alnylam remains strong financially. Its products, particularly Amvuttra, continue to generate robust growth. The company's pipeline is promising. All things considered, I think Alnylam has a much better story than its stock chart suggests. Is Alnylam Pharmaceuticals a smart long-term play? Before you buy stock in Alnylam Pharmaceuticals, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now... and Alnylam Pharmaceuticals wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of its recommendation, you'd have $370,440!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of its recommendation, you'd have $1,330,110!* Now, it's worth noting Stock Advisor's total average return is 924% - a market-crushing outperformance compared to 212% for the S&P 500. Don't miss the latest top 10 list, available with Stock Advisor, and join an investing community built by individual investors for individual investors. *Stock Advisor returns as of October 21, 2026.