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Alnylam Data Puts a Nobel Discovery One Step Closer to Approved Drug

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patisiran—meant to be administered once every three weeks, for life—will have a “six-figure price per year” if approved, which would be in line with the going rate for so-called orphan drugs for rare diseases with no other treatments. The first treatment for spinal muscular atrophy, Biogen’s (NASDAQ: BIIB) nusinersen (Spinraza), for instance, costs $750,000 for the first year and $375,000 each year thereafter. Sarepta Therapeutics’s (NASDAQ: SRPT) Duchenne muscular dystrophy drug eteplirsen (Exondys 51) costs an average of $300,000 per year.

Alnylam spent $382.4 million on R&D in 2016 and $276.5 million in 2015. The company declined to disclose how much it spent over the years developing patisiran specifically, however.

[Updated with comments from Greene] Pending approval, Alnylam expects to begin selling the drug in mid-2018, president Barry Greene said on the conference call. Alnylam believes the drug can help the estimated 20,000 to 30,000 patients around the world with TTR amyloidosis—including those with some degree of heart problems.

Rare disease drug developers have had varying levels of success convincing payers to cover the increasingly high prices of orphan drugs. Greene said the company has been talking with payers and expects “real receptivity to patisiran,” given the data Alnylam has accrued.

“We are working with the payer community to design programs so that when we have an approved therapy, we can help patients achieve rapid and affordable access,” Greene said.

The looming commercial battle aside, today’s patisiran data are a scientific victory for a field that has been waiting for years to deliver on its initial promise.

RNAi is a method cells use to mute a gene with strands of RNA before it can produce a protein. It was first discovered in 1998 by a team led by scientists Andrew Fire and Craig Mello. As Mello, who has no ties to Alnylam, explained to Xconomy recently, the two weren’t directly studying gene silencing, just trying to use it as a tool when they  “noticed there were really surprising things going on.” Eight years later Fire and Mello won a Nobel Prize for their work.

Drug makers found RNAi particularly intriguing because it offered the potential to create synthetic versions of the protein-blocking RNA: a new class of drugs to aim at diseases that other drug making methods—like monoclonal antibodies or small molecule drugs—couldn’t touch.

Firms like Alnylam, Sirna Therapeutics, RXi Pharmaceuticals and more formed in the early 2000s to try to harness the technology. But as is often the case with new science, it has taken years of ups and downs to get RNAi even this close to market.

Significant hype was followed by the hard smack of biological reality. Delivering relatively large RNA molecules into the right cells, without causing unintended problems, proved a daunting task that even Mello recently said the field “underestimated.”

Across the first decade of the 2000s, clinical studies ended in failure. Large pharmaceutical firms like Roche, Abbott Laboratories, Pfizer, and Novartis fell in and out of love with RNAi. Other drug development methods and tools, such as gene therapy and CRISPR-Cas9 gene editing, have since garnered more headlines and hype.

Alnylam survived through dark days and became the RNAi field’s largest company, thanks to several early partnerships that raked in a pile of cash. It tailored its ambitions exclusively to diseases it could target by delivering the molecules to the liver. It focused largely on rare diseases with no available treatments, hoping for quicker development paths.

There have been recent bumps along the way for Alnylam. Last year, it dumped one drug in Phase 3, revusiran, and another in early-stage testing, ALN-AAT. Both moves were due to safety problems, as was the company’s recent decision to suspend all trials of an experimental hemophilia drug, fitusiran. But Alnylam was bullish enough to open a commercial hub in the U.K. in September 2016 while the Apollo trial was ongoing.

Patisiran’s success today makes that bet a good one and marks a turning point both for Alnylam and, at long last, RNAi.

As Mello said earlier this month, before the data were released: “It’s a whole new ballgame now. The hard work has been done, or a lot of it. It’s time to start making drugs.”

Alnylam splits rights to patisiran with Sanofi, part of a wide-ranging alliance. Alnylam has rights in the U.S. and Western Europe, Sanofi everywhere else.

Here’s more, meanwhile, on the long saga of RNAi, and Alnylam’s place in it.

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