Dive Brief:

• Alnylam Pharmaceuticals said Thursday it plans to submit its experimental drug vutrisiran for U.S. approval in early 2021, now that the company has in hand positive data from a key study.

• The study tested Alnylam’s drug in patients with nerve damage from hATTR amyloidosis, a rare, inherited disorder in which misfolded proteins bind together in various tissues and organs, often leading to major health complications and premature death. Results indicate that nine months into treatment, vutrisiran had significantly reduced neurological impairment in these patients, as compared to historical placebo data from APOLLO — the clinical trial which led to the approval of Alnylam’s first marketed drug for hATTR polyneuropathy, Onpattro.

• In addition to the neurological measures, Alnylam’s study explored whether vutrisiran could have an effect on the heart. Investigators have been monitoring patients for proteins produced when the heart is under stress, and, according to Alnylam, the drug appears to be providing some level of improvement. The company said full 9-month results will be presented at a medical conference in early 2021, while high-level 18-month results — including cardiac data — should be announced in late 2021.

  Dive Insight:

  Alnylam is one of the pioneering companies in RNA interference, a drugmaking technique that attempts to treat disease by silencing genes.

  The Cambridge, Massachusetts biotech spent 15 years developing Onpattro, which in 2018 became the first ever RNA interference drug approved by the FDA. Since then, it has ushered to market two more rare disease medicines, named Givlaari and Oxlumo. Over the first nine months of this year, Alnylam recorded about $250 million in net product revenue.

  The company hopes that, having succeeded in a late-stage study, vutrisiran will soon join its line of products.

  Paul Matteis, an analyst at Stifel, said the results presented Thursday look good, and that the drug would provide a sales bump for Alnylam even if it ends up with a label similar to Onpattro’s. He highlighted in a note to clients how vutrisiran’s administration would likely be seen by doctors and patients as more convenient, since it’s given every three months as a subcutaneous injection. Onpattro, meanwhile, is given every three weeks via an infusion.

  Yet, vutrisiran offers more than added an added revenue stream for Alnylam. If approved, the drug would help Alnylam deliver on its promise that RNA interference technology can be a repeatable drug platform.

  “Indeed, the vutrisiran results … now serve as a second example of the potential for RNAi therapeutics to have a meaningful impact for patients, showing the ability to halt and potentially even reverse polyneuropathy manifestations of the disease,” said John Maraganore, Alnylam’s CEO, in a Jan. 7 statement.

  In addition to its effects on neuropathy, Alnylam said vutrisiran showed an “encouraging safety profile.” Two patients in the study’s vutrisiran arm died during the nine month treatment period, but neither death was considered related to vutrisiran.

  There were also two serious adverse events — a urinary tract infection, and an unhealthy level of fat in the blood — deemed related to vutrisiran by the study’s investigator. Some of the more common treatment emergent adverse events included diarrhea, pain in the extremities and urinary tract infections, with each happening at a similar or lower rate compared to historical placebo data.

  Matteis wrote that these findings suggest there were “no major red flags safety-wise.”

  Alnylam shares were up 6% in late morning trading Thursday.