Wave Life Sciences Ltd. announced that Pfizer recently nominated the fourth and fifth final hepatic targets under the collaboration agreement between the two companies to develop genetically targeted therapies for the treatment of metabolic hepatic diseases, such as nonalcoholic steatohepatitis. Upon payment of nomination fees, Pfizer had the right to select a maximum of five targets to advance from discovery through candidate selection with Wave.
Over the past two years, Wave and Pfizer have been collaborating to advance genetically defined targets for the treatment of metabolic hepatic diseases, bringing together Wave’s stereochemistry platform across antisense and single-stranded RNAi modalities, along with GalNAc and Pfizer’s hepatic targeting technology for delivery to the liver. Two targets, including apolipoprotein C-III, were declared upon commencement of the collaboration in May 2016. In August 2016, Pfizer nominated its third target. Once candidates have been developed, Pfizer may elect to exclusively license the programs and undertake further development and potential commercialization.
“The designation of these final two targets marks another milestone in a highly productive collaboration in which we have further established the benefits and versatility of our stereochemistry platform while making meaningful progress toward potentially addressing metabolic diseases of the liver,” said Paul Bolno, MD, MBA, Chief Executive Officer and President of Wave Life Sciences. “We look forward to continuing to advance all five programs and delivering differentiated stereopure candidates to our partners at Pfizer.”
Morris Birnbaum, Chief Scientific Officer, Internal Medicine Research Unit at Pfizer, said, “Based on encouraging preclinical in vivo data generated through this collaboration, we believe that Wave’s unique stereochemistry platform can augment our targeting technology and enhance the profile of potentially therapeutic oligonucleotides. We are pleased to have identified five important and druggable hepatic targets and fully expect to maintain this momentum as we collaborate with Wave on candidate selection.”
Through the collaboration, Wave has been able to further explore the therapeutic potential of single-stranded RNAi. Wave has demonstrated improved stability and pharmacology in vitro and in vivo, and produced comparable or greater potency than double-stranded RNAi formats that have historically been used to treat hepatic diseases. This progress is expected to extend RNAi mediated silencing to tissues beyond the liver. Wave has also made advancements in GalNAc-conjugated stereopure antisense oligonucleotides, with in vivo data demonstrating significant activity of stereopure GalNAc-conjugated antisense oligonucleotides over stereorandom GalNAc-conjugated oligonucleotides.