Alnylam Pharmaceuticals Reports Third Quarter 2016 Financial Results And Highlights Recent Period Activity

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter 2016, and highlighted recent progress in advancing its pipeline.

"We continue to advance a broad pipeline of investigational RNAi therapeutics - including 8 programs in clinical development - across a wide range of disease indications with high unmet need. Despite a disappointing outcome in our revusiran program, we remain committed to serving the needs of the ATTR amyloidosis community with patisiran and to developing RNAi therapeutics with our ESC-GalNAc conjugate platform as a new class of innovative medicines. Based on a recent recommendation from the APOLLO Data Monitoring Committee for patisiran and a comprehensive review of data from over 800 human subjects treated with RNAi therapeutics - excluding revusiran - for up to nearly three years, we are encouraged by the overall safety profile for our platform," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We're pleased with new positive clinical results we presented in the third quarter for patisiran for hATTR-PN, fitusiran for hemophilia, ALN-AS1 for porphyria, and ALN-GO1 for primary hyperoxaluria. Now and through the end of the year, we are anticipating a very data rich period - including key clinical data presentations for five distinct programs at the AHA and ASH meetings and at our R&D Day in December - and we look forward to sharing our progress."

Third Quarter 2016 and Recent Significant Corporate Highlights
  • Advanced patisiran for the treatment of hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloidotic polyneuropathy (FAP).
    • The Company announced today that it has initiated its patisiran Expanded Access Program (EAP) for patients with hATTR-PN. The patisiran EAP provides an opportunity for eligible hATTR-PN patients, who are unable to participate in an ongoing clinical trial or whose treatment options are otherwise limited, to receive patisiran therapy until the drug becomes commercially available, should it receive approval.
    • Announced that the patisiran APOLLO Data Monitoring Committee (DMC) met at the Company's request following its decision to discontinue development of revusiran, and recommended continuation of the APOLLO Phase 3 trial without modification.
    • Reported positive initial 24-month data from ongoing Phase 2 open-label extension (OLE) study with patisiran for the treatment of hATTR-PN. Results showed that patisiran can potentially halt or improve neuropathy progression. Patisiran administration was also found to be generally well tolerated in hATTR-PN patients out to 25 months, with no drug-related serious adverse events (SAEs) reported through the data transfer date.
  • Announced decision to discontinue development of revusiran, an investigational RNAi therapeutic that was being developed for the treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM).
    • The decision was made following the recommendation by the DMC for the ENDEAVOUR Phase 3 trial of revusiran to suspend dosing and the observation of an imbalance in mortality in revusiran-treated patients as compared to those on placebo.
    • The Company has initiated a comprehensive evaluation of revusiran data.
    • The decision to discontinue development of revusiran does not affect patisiran or any other Alnylam investigational RNAi therapeutic program in development.
  • Advanced fitusiran for the treatment of hemophilia and rare bleeding disorders (RBD).
    • Reported positive interim clinical results from Phase 1 study of fitusiran. Fitusiran achieved a median estimated annualized bleeding rate (ABR) of zero in hemophilia patients without inhibitors. In the initial low-dose cohort of patients with inhibitors, fitusiran achieved antithrombin lowering, increased thrombin generation, and preliminary evidence for reduced bleeding. Fitusiran administration was generally well tolerated in patients with and without inhibitors through the data transfer date.
    • Continued dosing hemophilia patients in the ongoing Phase 1/2 OLE study, with up to 16 months of dosing.
    • In July, the Company also updated its guidance for Phase 3 initiation, and currently remains on track to start studies in early 2017.
  • Reported positive interim clinical results from Phase 1 study of ALN-AS1 for the treatment of acute hepatic porphyrias.
    • In asymptomatic high excreter (ASHE) porphyria subjects, single and multiple doses of ALN-AS1 achieved rapid, dose-dependent, and durable lowering of aminolevulinic acid (ALA) and porphobilinogen (PBG) - the toxic heme synthesis intermediates that mediate porphyria attacks - of up to 95%, with effects sustained for over ten months after a single dose. ALN-AS1 was generally well tolerated following single and multiple doses through the data transfer date.
    • In addition, both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) granted orphan drug designation to ALN-AS1 for the treatment of acute hepatic porphyrias.
  • Reported positive initial clinical results from Phase 1/2 study of ALN-GO1 for the treatment of primary hyperoxaluria type 1 (PH1).
    • ALN-GO1 achieved human proof of concept with statistically significant increases in glycolate, a biomarker of effective glycolate oxidase knockdown, in healthy adult volunteers. Single doses of ALN-GO1 were found to be generally well tolerated through the data transfer date.
    • In addition, the Company published pre-clinical data with ALN-GO1 in the Journal of the American Society of Nephrology (Liebow  et al., J Am Soc Nephrol, 2016;  doi:10.1681/ASN.2016030338).
  • Reported initial clinical results from Phase 1/2 study of ALN-AAT for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease and provided program update.
    • Results showed that ALN-AAT administration provided potent, dose-dependent, and durable knockdown of serum AAT of up to 88.9%.
    • Three instances of asymptomatic, transient elevations of liver enzymes were detected in the highest dose groups. As a result, the Company plans to advance a new Development Candidate, ALN-AAT02.
  • Alnylam's partner, The Medicines Company, announced positive top-line results from the Day 90 interim analysis in the ongoing ORION-1 Phase 2 study of ALN-PCSsc (also known as PCSK9si), an investigational RNAi therapeutic for the treatment of hypercholesterolemia.
    • ALN-PCSsc achieved significant and durable LDL-C reduction up to Day 90 and was generally well tolerated, including no drug-related liver enzyme elevations, neuropathy signals, or renal function changes.
  • Initiated Phase 1/2 clinical trial with ALN-HBV for the treatment of Hepatitis B Virus (HBV) infection.
  • Expanded Alnylam's global footprint with the opening of a new development and commercial hub in Maidenhead, United Kingdom. The Maidenhead office joins Alnylam's Zug, Switzerland office - its European headquarters - in allowing the Company to support planned growth in Europe.
  • Expanded Management Team with appointments of Antoine Barouky, General Manager, France; Emmanuel Dulac, Ph.D., Senior Vice President, Chief Commercial Officer; Arianna Greco, Vice President, Legal - Europe and Canada; Yvonne Greenstreet, MBChB, Executive Vice President, Chief Operating Officer; Thomas Hoock, Ph.D., Vice President, Program Lead; and Siva Sakhamuri, Ph.D., Vice President, Manufacturing.

Upcoming Events in Late 2016
  • Alnylam's partner The Medicines Company plans to present initial results from the ORION-1 Phase 2 study of ALN-PCSsc in a Late-Breaking Clinical Trial Session at the American Heart Association (AHA) Scientific Sessions 2016, on November 15, 2016.
  • Alnylam announces today that it plans to present clinical data from multiple pipeline programs at the 58 th American Society of Hematology (ASH) Annual Meeting, being held December 3 - 6, 2016 in San Diego, California, including:
    • Additional data from the Phase 1 study of fitusiran in patients with hemophilia, including initial data from the Phase 1/2 OLE study, in poster presentations on:
      • Saturday, December 3, at 5:30 pm PT (patients with inhibitors);
      • Sunday, December 4, at 6:00 pm PT (patients without inhibitors).
    • Initial data from Part C of the Phase 1/2 study of ALN-AS1 in acute intermittent porphyria patients with recurrent porphyria attacks in a poster presentation on Saturday, December 3, at 5:30 pm PT.
    • Longer-term follow-up data from the Phase 1/2 study of ALN-CC5, an investigational RNAi therapeutic for the treatment of complement-mediated diseases, in patients with paroxysmal nocturnal hemoglobinuria (PNH) receiving concomitant eculizumab in a poster presentation on Monday, December 5, at 6:00 pm PT.
  • The Company also announces today that it plans to present initial clinical results from the Phase 1 study of ALN-TTRsc02, an investigational RNAi therapeutic for the treatment of ATTR amyloidosis, at the Company's R&D Day, which will be held the morning of Friday, December 16, 2016 in New York City and will be webcast.

Financials

"Alnylam continues to maintain a strong balance sheet, ending the third quarter of 2016 with approximately $1.2 billion in cash, including restricted investments," said Michael Mason, Vice President, Finance and Treasurer. "Our financial strength allows us to continue to invest in a broad pipeline of investigational RNAi therapeutics, aligned with achievement of our 'Alnylam 2020' goals. As for financial guidance this year, we remain on track to end 2016 with greater than $1.0 billion in cash, including $150.0 million in restricted investments."

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