Alnylam Pharmaceuticals Reports Third Quarter 2020 Financial Results

11/5/20

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter 2020 and reviewed recent business highlights.

“We are extremely pleased with the performance of ONPATTRO and GIVLAARI in the third quarter, reflecting strong commercial execution and improving market conditions following the more challenging COVID-19 pandemic phase experienced in the second quarter. We’re also excited about the recent positive CHMP opinions for OXLUMO and LEQVIO, moving these potentially transformative investigational RNAi therapeutics closer to approval. We believe that this positions Alnylam to potentially exit 2020 with four revenue-generating products bolstering our sustained growth,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “In addition, our robust late-stage pipeline of investigational medicines continued to advance in the quarter. Notably, we achieved positive results from the ILLUMINATE-B study – demonstrating efficacy and safety for our investigational RNAi therapeutic lumasiran in children under the age of six, including infants. We also achieved continued enrollment in our APOLLO-B and HELIOS-B Phase 3 studies of patisiran and vutrisiran, respectively, in development for the treatment of ATTR amyloidosis with cardiomyopathy. With our recent progress, we now expect to exceed our Alnylam 2020 vision and goals of building a multi-product, global biopharma company with a deep clinical pipeline and a robust, organic product engine to drive future sustainable innovation and value creation. Finally, we look forward to highlighting further progress and future plans at our upcoming virtual R&D Day event in December.”

Third Quarter 2020 and Recent Significant Corporate Highlights

Commercial Performance

ONPATTRO®

  • Achieved global net product revenue for the third quarter of 2020 of $82.5 million, representing 24% quarterly growth globally and 21% quarterly growth in the U.S. alone.
  • Attained over 1,150 patients worldwide on commercial ONPATTRO treatment as of September 30, 2020.
  • Continued progress with market access efforts across the CEMEA region (Canada, Europe, Middle East, and Africa), with a recent launch in Portugal, conclusion of price negotiations in France, and completion of initial access agreement in Canada.
  • Continued global expansion with achievement of regulatory approval in Israel.
  • Received the prestigious 2020 Prix Galien USA Award for Best Biotechnology Product.

GIVLAARI®

  • Achieved global net product revenue for the third quarter of 2020 of $16.7 million.
  • Attained over 150 patients worldwide on commercial GIVLAARI treatment as of September 30, 2020.
  • Continued strong market access progress in the U.S., with 10 VBAs finalized to date with commercial payers and confirmed access for over 90% of covered U.S. lives.
  • Continued progress with market access efforts across the CEMEA region, with ongoing launch in Germany, cohort Temporary Authorization for Use (ATU) supply in France, and named patient sales in other countries.
    • Received an Improvement of Medical Benefit (ASMR) score of II in France, concluding that GIVLAARI offers significant additional therapeutic value. In 2019, only two new commercial medicines received a similar ASMR score.
    • In addition, obtained a “Considerable Benefit” rating in Germany and secured a strong health technology assessment (HTA) rating in Italy.
  • Continued global expansion with approval in Canada and submission of a new drug application in Japan.
  • Received the NORD 2020 Industry Innovation Award.

R&D Highlights

  • Advanced patisiran (the non-proprietary name for ONPATTRO), in development for the treatment of the cardiomyopathy of both hereditary and wild-type ATTR amyloidosis.
    • Continued enrollment in the APOLLO-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy.
    • Presented additional clinical data with patisiran – including 24-month results from the global open-label extension (OLE) study and results from an open-label study in patients with hATTR amyloidosis post-orthotopic liver transplant – and published findings from an evaluation of patisiran with concomitant or prior use of TTR stabilizers.
  • Advanced vutrisiran, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
    • Continued treating patients in the fully enrolled HELIOS-A Phase 3 study of vutrisiran in hATTR amyloidosis patients with polyneuropathy, and remain on track to report topline results in early 2021.
    • Continued enrollment in the HELIOS-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy.
    • Announced potential for a biannual dosing regimen option for vutrisiran, providing support for further product differentiation as a potential best-in-class agent.
  • Presented new interim data from the Phase 1/2 open-label extension (OLE) study of givosiran (the non-proprietary name for GIVLAARI) in acute hepatic porphyria (AHP).
  • Advanced lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1).
    • Received a positive CHMP opinion from EMA recommending approval of lumasiran for the treatment of PH1 in patients of all ages. If approved, lumasiran will be marketed in Europe under the brand name OXLUMO™.
    • Received a positive scientific opinion from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) through the Early Access to Medicines Scheme (EAMS).
    • Presented positive complete results from ILLUMINATE-B, a global Phase 3 pediatric study of lumasiran in PH1 patients less than six years of age, including infants, with preserved renal function.
    • Continued enrollment in the ILLUMINATE-C Phase 3 study of lumasiran for the treatment of advanced PH1 in patients of all ages.
  • Alnylam’s partner, Novartis, continued advancing inclisiran, potentially the first and only RNAi therapeutic cholesterol-lowering treatment. Inclisiran is undergoing regulatory review in the U.S. and EU.
    • Received a positive CHMP opinion from EMA recommending approval of inclisiran for the treatment of adults with hypercholesterolemia or mixed dyslipidemia. If approved, inclisiran will be marketed under the brand name LEQVIO®.
  • Alnylam’s partner, Sanofi, continued advancement of the ATLAS Phase 3 program for fitusiran in patients with hemophilia A or B with and without inhibitors.
  • Advanced early- and mid-stage RNAi therapeutic pipeline programs.
    • Continued dosing in the Phase 1 study of ALN-AGT in hypertension.
    • In collaboration with Regeneron, advanced cemdisiran, an investigational RNAi therapeutic for the treatment of complement-mediated diseases.
      • Continued enrollment in a Phase 2 clinical trial of cemdisiran monotherapy in patients with IgA nephropathy, with topline results expected in 2021.
    • Alnylam’s partner Vir Biotechnology presented new data on VIR-2218 (ALN-HBV02) at the European Association for the Study of the Liver Digital International Liver Congress.
      • In addition, Vir initiated a Phase 2 combination trial of VIR-2218 with pegylated interferon-alpha (PEG-IFN-?), with initial clinical data anticipated in 2021.
    • The Company is announcing today that it has initiated dosing in a Phase 1 study of ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 in development for the treatment of nonalcoholic steatohepatitis (NASH). ALN-HSD is being advanced in collaboration with Regeneron.
    • The Company is announcing today that it will delay its planned filing of an IND for ALN-COV in order to obtain additional pre-clinical efficacy data in models of COVID-19 infection.
      • ALN-COV is part of a multi-target pre-clinical collaboration with Vir Biotechnology.
    • Continued progress with investigational RNAi therapeutics for CNS and ocular diseases, including advancement of ALN-APP, in development for the treatment of hereditary cerebral amyloid angiopathy (hCAA) and autosomal dominant Alzheimer’s Disease (ADAD), which remains on track for a CTA filing in mid-2021.
      • Regeneron exercised its co-development/co-commercialization option on the ALN-APP program, which Alnylam will lead.

Additional Business Updates

  • Barry Greene, former President of Alnylam, transitioned from Alnylam at the end of the third quarter. Yvonne Greenstreet assumed an expanded role as President and Chief Operating Officer on October 1, 2020.
  • Closed $150 million R&D funding component of the previously announced $2 billion strategic financing collaboration with Blackstone to accelerate the advancement of RNAi therapeutics.
  • Expanded global reach of commercialization activities with new third-party distribution agreement with taiba Middle East.
  • Received recognition from Science magazine as a top employer.

Upcoming Events

  • Alnylam announced today that it intends to present interim results from the Phase 1 trial of ALN-AGT at the American Heart Association Scientific Sessions 2020 on Friday, November 13.
  • The Company also announced today that it plans to present a review of its R&D and commercial activities at its upcoming R&D Day event being held virtually December 15 and 16.

In addition, in late 2020, Alnylam intends to:

  • Achieve regulatory approval for lumasiran in the EU and an FDA action for lumasiran in late 2020.
  • Alnylam’s partner Novartis expects regulatory approval for inclisiran in the EU and an FDA action for inclisiran in late 2020.
  • Achieve regulatory approval for ONPATTRO in Taiwan.
  • Alnylam’s partner Regeneron plans to initiate a Phase 1 study of cemdisiran in combination with pozelimab.

Financial Results for the Quarter Ended September 30, 2020

“The third quarter was very strong, with a rebound in ONPATTRO sales growth, particularly in the U.S., and continued impressive early performance with GIVLAARI. While the pandemic headwinds haven’t completely receded, we believe our teams have been very effective in meeting the needs of patients. As a result of this continued strength and our cautious optimism for the fourth quarter, we are again revising our full-year revenue guidance for ONPATTRO, increasing the range from $280-$300 million to $295-$310 million,” said Jeff Poulton, Chief Financial Officer of Alnylam. “Beyond the commercial business, we also closed the $150 million R&D funding component of the Blackstone strategic financing collaboration, finalizing the last piece of a broad relationship that we believe secures our path to self-sustainability without the need for future equity offerings. Looking ahead, we believe we are well positioned to continue executing on our current commercial portfolio, with potential for two additional RNAi therapeutics coming to market, and advancing our broad pipeline of investigational programs to drive future growth.”

About ONPATTRO® (patisiran)

ONPATTRO is an RNAi therapeutic that was approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the polyneuropathy associated with the disease. For more information about ONPATTRO, visit ONPATTRO.com.