RNAi Digest - August 2020

RNAi Digest - August 2020

Blackstone and Alnylam closed a $150 million R&D financing agreement for cardiovascular diseases

17 Aug 2020

Vutrisiran (siRNA targeting TTR mRNA) and ALN-AGT (siRNA targeting Angiotensinogen)

  • Alnylam will receive up to $150 million from Blackstone Life Sciences for the development of Alnylam’s cardiometabolic disease programs vutrisiran and ALN-AGT
  • Includes up to $70 million to support the ongoing HELIOS-B Phase 3 study of vutrisiran in ATTR amyloidosis patients with cardiomyopathy, and up to $80 million to support Phase 2 and Phase 3 trials of ALN-AGT for the treatment of hypertension
  • Backing their interest in the positive top line results in a Phase 1 (for N=184) in hypertension: N=48 demonstrated 90% mean knockdown of angiotensinogen; Expected PCD ‘ Dec’21

Blackstone & Alnylam closed $150 million R&D funding part of $2 billion deal

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CI Scientists Remarks: 

  • This $150 million is a part of the $2 billion strategic financing collaboration between Blackstone & Alnylam to accelerate the advancement of RNAi therapeutics
    • $70 million for HELIOS-B Phase 3 study of vutrisiran in ATTR amyloidosis patients with cardiomyopathy; N=600, PCD-Jun’24
    • $80 million to support Phase 2 and Phase 3 development of ALN-AGT in development for the treatment of hypertension
  • Other parts of deal include:
    • $1 billion to acquire 50% of Anylam’s royalties and commercial milestones for “Inclisiran” (first and only siRNA cholesterol-lowering treatment, filed in US & EU) (will be paid by Novartis)
    • $750 million in a first lien senior secured term loan led by GSO
    • $100 million purchase of Alnylam common stock
  • This unique non-dilutive financing deal from Blackstone will aid in Anylam’s clinical and commercial efforts
  • Alnylam has two approved products: ONPATTRO (patisiran), approved for Hereditary transthyretin (hATTR) Amyloidosis in the US, EU, Canada, Japan, Switzerland and Brazil, and GIVLAARI (givosiran), approved for Acute Hepatic Porphyria in the US, EU and Brazil

– Tarun Raisinghani, CI Scientists

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