Alnylam Pharmaceuticals Continues Onpattro Launch With More to Come

Alnylam Pharmaceuticals (ALNY 0.63%) continued its launch of Onpattro in the first quarter, doubling the number of patients taking the drug from the end of the year to the end of the first quarter.

Alnylam Pharmaceuticals results: The raw numbers

Data source: Alnylam Pharmaceuticals.

What happened with Alnylam Pharmaceuticals this quarter?

• Sales of Onpattro, which treats hereditary transthyretin-mediated amyloidosis (ATTR), came in at $26.3 million, more than double the $12.1 million in the fourth quarter.
• In the first quarter, 90% of the start forms received came from newly identified patients who hadn't been treated in the expanded access program, suggesting the sales force is getting the word out about the new medication.
• Collaboration revenue fell year over year due to a one-time milestone payment from Sanofi in the year-ago quarter.
• In March, Alnylam reported positive phase 3 clinical trial data for givosiran, which treats a nervous system disease called acute hepatic porphyria.
• With a pipeline of drug candidates to fund, Alnylam won't be profitable anytime soon, but the company has a substantial cash runway with $1.29 billion in the bank, after raising $382 million in a secondary offering in January.
• Alnylam will also get another $800 million as part of a recently announced drug development deal with Regeneron Pharmaceuticals that is scheduled to close this quarter.

Image source: Getty Images.

What management had to say

Alnylam's president Barry Greene laid out some additional details on the prescribers, which could be helpful as the company tries to expand the approval of Onpatro into ATTR patients with heart complications: "Of note, 55% of start forms in Q1 came from cardiologists, which we believe reflects the mixed phenotype patient population and continued recognition of polyneuropathy as a major disease manifestation even in patients who may initially present with cardiac manifestations, and therefore be under the care of a cardiologist for their disease. We're also very encouraged by the number of new prescribing physicians and the number of physicians who have submitted multiple prescriptions."

On the patient side, Greene noted that Onpattro is competing well with Pfizer's (PFE 0.42%) tafamidis, which will likely be approved in the U.S. in the near future after posting solid phase 3 data last year. "In addition to naive patients, we are observing patients and physicians switching to Onpatrro from other products, including tafamidis, which is approved in the EU and reimbursed in many countries," Greene said.

Looking forward

Management didn't give revenue guidance, which is fair given the dynamic nature of a drug launch.

On the pipeline front, Alnylam expects to submit marketing applications for givosiran to regulators in the U.S. and EU in the middle of this year, setting up approvals later this year or early next year.

In the third quarter, investors should expect results from three phase 3 clinical trials testing inclisiran, the cholesterol lowering drug Alnylam is developing with The Medicines Company (MDCO). Also this year, Alnylam plans to present phase 3 data for lumasiran, which treats a kidney disease called primary hyperoxaluria type 1.