On November 2, 2023 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the third quarter ended September 30, 2023 and reviewed recent business highlights (Press release, Alnylam, NOV 2, 2023, View Source [SID1234636728]).

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"We continued to deliver strong commercial results in the third quarter, with the successful launch of AMVUTTRA contributing to 35% year-over-year growth in net product revenues," said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. "While we were disappointed by the recent Complete Response Letter for ONPATTRO for the treatment of the cardiomyopathy of ATTR amyloidosis, we remain confident in the HELIOS-B Phase 3 study of vutrisiran, which is on track to deliver topline results in early 2024. We’re also excited about advancements across our broad pipeline of RNAi therapeutics, including recent positive topline results from the KARDIA-1 Phase 2 study of zilebesiran, as well as upcoming Phase 1 results for ALN-TTRsc04 and ALN-KHK by year-end. With this continued execution we remain on track to meet our Alnylam P5x25 goals of becoming a top-tier biotech company delivering sustained innovation and exceptional financial results."

Third Quarter 2023 and Recent Significant Corporate Highlights

Commercial Performance

Total TTR: ONPATTRO (patisiran) & AMVUTTRA (vutrisiran)

Achieved global net product revenues for ONPATTRO and AMVUTTRA for the third quarter of $82 million and $149 million, respectively, representing 35% total TTR reported year-over-year growth compared to Q3 2022.
Attained over 3,790 hATTR amyloidosis patients with polyneuropathy worldwide on commercial treatment with ONPATTRO or AMVUTTRA as of September 30, 2023.
Total Ultra-Rare: GIVLAARI (givosiran) & OXLUMO (lumasiran)

Achieved global net product revenues for GIVLAARI and OXLUMO for the third quarter of $54 million and $29 million, respectively, representing 33% total Ultra-Rare reported year-over-year growth compared to Q3 2022.
Attained over 625 patients worldwide on commercial GIVLAARI treatment as of September 30, 2023.
Attained over 375 patients worldwide on commercial OXLUMO treatment as of September 30, 2023.
Leqvio (inclisiran)

Leqvio launch in the U.S. and other markets is ongoing, with focus on patient on-boarding, removing access hurdles and enhancing medical education. In the U.S., Leqvio is covered at or near label for 76% of patients. More than 55% of Leqvio source of business in the U.S. is now through "Buy and Bill" acquisition mode. FDA expanded the label to include primary hyperlipidemia (patients at increased risk of ASCVD) and the removal of four adverse reactions from the safety section as well as Limitations of Use. In Q3 2023, Leqvio was approved in China and in Japan and is now approved in 93 countries.
R&D Highlights

Reported positive topline results from the KARDIA-1 Phase 2 dose-ranging study of zilebesiran, an investigational RNAi therapeutic in development to treat hypertension in patients at high cardiovascular risk.

Published results from Phase 1 study of zilebesiran in the New England Journal of Medicine.

Reported updated positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP in patients with early-onset Alzheimer’s disease at the 2023 Alzheimer’s Association International Conference and at the 16th Clinical Trials on Alzheimer’s Disease conference.

Based on the achievement of specified clinical proof-of-principle criteria for the Phase 1 ALN-APP program, Alnylam received a $100 million milestone payment from Regeneron.
Presented new 24-month results from an interim analysis of the open-label extension period of the APOLLO-B Phase 3 study of patisiran in patients with the cardiomyopathy of transthyretin-mediated amyloidosis at the Heart Failure Society of America Annual Scientific Meeting 2023.

Published results from Phase 3 APOLLO-B study of patisiran in the New England Journal of Medicine.

Sanofi reported positive data for fitusiran, in development for the treatment of hemophilia A or B, with or without inhibitors, from the Phase 3 open-label extension study (ATLAS-OLE).

Sanofi is currently in discussions with the FDA regarding filing in 2024.
Additional Business Updates

Entered into a global strategic collaboration with Roche for the co-development and co-commercialization of zilebesiran.
Recognized by Science magazine as a Top Employer for the fifth consecutive year.
Upcoming Events

Alnylam announces today that it will present a review of its RNAi platform and pipeline progress at a virtual R&D Day on December 13, 2023.

Alnylam announces today that results from the KARDIA-1 Phase 2 dose-ranging study of zilebesiran will be presented as a Late-Breaker at the American Heart Association Scientific Sessions 2023 on November 11, 2023, in Philadelphia, Pennsylvania.

In addition, in late 2023:

Alnylam intends to report topline results from the Phase 1 study of ALN-TTRsc04.
Alnylam intends to report topline results from the Phase 1 study of ALN-KHK.
Vir is conducting multiple trials evaluating the potential for ALN-HBV02 (VIR-2218) and VIR-3434 to achieve a functional cure for chronic hepatitis B. Phase 2 data readouts are on track for Q4 2023.
Vir also announced that initial Phase 2 data readouts for the SOLSTICE trial evaluating ALN-HBV02 (VIR-2218) and VIR-3434 as monotherapy and in combination for the treatment of people living with chronic hepatitis delta, the most aggressive form of viral hepatitis, are expected in Q4 2023.