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Date: 30-Jul-2021

Alnylam And PeptiDream Enter Into Collaboration Agreement To Discover And Develop Peptide-siRNA Conjugates For Targeted Delivery Of RNAi Therapeutics

CAMBRIDGE, Mass. & KAWASAKI CITY, Kanagawa Prefecture, Japan--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and PeptiDream, Inc. (Tokyo Stock Exchange: 4587), the leading peptide-based drug discovery company with its proprietary Peptide Discovery Platform System (PDPS), announced today a license and collaboration agreement to discover and develop peptide-siRNA conjugates to create multiple opportunities to deliver RNAi therapeutics to tissues outside the liver. Through this collaboration, the companies will collaborate to select and optimize peptides for targeted delivery of small interfering RNA (siRNA) molecules to a wide range of cell types and tissues via specific interactions with receptors expressed on the target cells.

Under the terms of the alliance, Alnylam will select a set of receptors for PeptiDream’s peptide discovery platform. PeptiDream will select, optimize, and synthesize peptides for each receptor. Alnylam will then generate peptide-siRNA conjugates and perform in vitro and in vivo studies to support final peptide selection. The collaboration has the potential to yield multiple treatment opportunities by targeting disease causing mRNA transcripts in a wide variety of tissue types.

“Having solved delivery of RNAi therapeutics to the liver and made substantial preclinical progress on in vivo delivery to the CNS, eye, and lung, we are now adding to our suite of delivery technologies that have the potential to enable efficient delivery of siRNA to even broader tissue types throughout the human body,” said Kevin Fitzgerald, Ph.D., Chief Scientific Officer of Alnylam. “We are excited to enter into this collaborative research agreement with PeptiDream, an industry leader in the discovery and optimization of peptide ligands against a wide variety of receptors. Through this collaboration, we hope to identify robust ligand-receptor pairs for extrahepatic tissue delivery, similar to the GalNAc-ASGPR pair that we have pioneered for effective liver delivery.”

“We look forward to partnering with Alnylam to leverage our PDPS technology and peptide discovery capabilities to identify novel ligands against Alnylam selected target receptors that can facilitate delivery of drug payloads to a broad range of extrahepatic tissues,” said Patrick C. Reid Ph.D., President and Representative Director of PeptiDream. “This deal further expands on our strategy of using peptides to deliver a variety of therapeutic payloads (as peptide drug-conjugates) to disease cells/tissues in a targeted fashion. We hope this partnership will enable the creation of innovative peptide-siRNA conjugates in a variety of diseases and unlock many new therapeutic opportunities.”

Under the terms of the agreement, PeptiDream will receive an upfront payment from Alnylam as well as R&D funding over the term of the research collaboration, as provided in the agreement. PeptiDream may also receive payments based on the achievement of specified development, regulatory, and commercial milestones potentially totaling up to $2.2 billion (¥244 billion*1). In addition, PeptiDream is eligible to receive low-to-mid single digit royalties on sales on any such Alnylam products.

*1: USD/ JPY currency conversion rate: 110.9

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and Leqvio® (inclisiran) being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam, on LinkedIn, or on Instagram.