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3 Things Alnylam's Management Wants You to Know

By Brian Orelli, PhD - Updated Apr 15, 2019 at 5:13PM

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The biotech gave investors reasons to cheer at the J.P. Morgan Healthcare Conference

Check out the latest Alnylam Pharmaceuticals earnings call transcript.

Like most drug companies, Alnylam Pharmaceuticals (ALNY -3.10%) presented at the J.P. Morgan Healthcare Conference. The company detailed the progress it's made on the launch of its first product, hereditary transthyretin-mediated (hATTR) amyloidosis drug Onpattro, as well as plans for the development of its pipeline, which includes a whopping six drugs in phase 3 development.

Between the main presentation and the breakout session, CEO John Maraganore and his crew of executives spent about 40 minutes laying out the biotech's story. Here are the top three takeaways from the presentations.

Person in scrubs talking to patient in an exam room

Image source: Getty Images.

1. Onpattro launch is on track

Alnylam's third-quarter results, which included just seven weeks of sales for Onpattro, disappointed investors, but the launch has since picked up the pace.


Q3 2018

Q4 2019 (Unaudited)


$0.46 million

$11 million-$12 million

Patients on commercial drug


Over 200

Cumulative start forms



Start forms from early access program (EAP)

Around 60%

Approximately 50%

Data source: Alnylam. N/D = Not disclosed.

While the sales increase is impressive, it's the start forms and the patient numbers that investors should be paying attention to since they are the measures that help predict sales in future quarters. Doctors submit start forms to enter patients into Alnylam's assistance program, which helps patients receive insurance reimbursements and get set up for their infusions. Many patients on commercial drug at the end of the fourth quarter likely started in the middle or end of the quarter, so they didn't account for a full quarter of sales, but will register a full three months in future quarters since Onpattro isn't a cure.

Patients in the EAP were getting free drug, so switching them to commercial access is important, and investors ultimately want to see the percentage of start forms coming from patients in the EAP going down, because it shows how well Alnylam is capturing hATTR patients it didn't already know about.

2. Value-based agreements are increasing access

"The proactive approach we took to market access is paying off. You're not hearing about payer headwinds like you typically hear in orphan launches," Alnylam's COO Barry Greene said.

Alnylam currently has three value-based agreements: with Harvard Pilgrim HealthCare, Humana, and an unnamed top-five payer. The company is also in discussions with 15 additional payers for value-based agreements, which, in aggregate, would cover over 90% of insured lives.

These value-based agreements allow Alnylam and the insurance companies to share the risk; insurers are more likely to cover a drug -- and less likely to argue about the price -- if Alnylam agrees to get paid less when Onpattro doesn't help the patient.

3. An approval per year

While the near-term focus is clearly on Onpattro, Alnylam has a well-stocked pipeline and could end up getting an approval a year for the foreseeable future.

Data from a phase 3 study testing givosiran in patients with acute hepatic porphyrias, conditions caused by the buildup of neurotoxic intermediates, is expected in March. If the company can hit its goal of filing a marketing application in June or early July, it could see an approval before the end of this year, assuming the Food and Drug Administration gives the drug a priority review.

Later this year, an ongoing phase 3 study for lumasiran, which treats a kidney disease called primary hyperoxaluria type 1, is expected to read out, putting a potential approval for the drug in 2020. And, depending on timing of data and filing of a marketing application, an FDA approval for inclisiran, a cholesterol-lowering drug being developed with The Medicines Company (MDCO), could come in late 2020 or early 2021.

Looking further ahead

Alnylam has its hands full launching Onpattro, but the potential market for the drug could get substantially larger with an expanded approval to treat patients with the version of TTR that affects the heart. At the J.P. Morgan conference, Alnylam announced plans to start a phase 3 study called Apollo B in the middle of this year. It will test the drug in TTR patients with cardiomyopathy, putting a potential expanded approval in the 2021-22 time frame.

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