Dive Brief:
- Takeda Pharmaceutical Company Limited has formed a global alliance with genetic disease biotech Wave Life Sciences Ltd. to discover, develop and commercialize nucleic acid therapies for central nervous system disorders.
- The deal, potentially worth over $2 billion, includes Wave's programs in Huntington's disease, amyotrophic lateral sclerosis, frontotemporal dementia and spinocerebellar ataxia type 3, as well as a handful of preclinical CNS programs.
- Takeda will make an upfront payment of $110 million, buy $60 million of Wave's ordinary shares at $54.70 a share, and fund at least $60 million of research at Wave over four years, with an aim to advance a number of preclinical projects. These programs will be eligible for pre-commercial and commercial milestone payments, as well as high single-digit to mid-teen royalty payments on global sales.
Dive Insight:
Takeda has had a busy start to 2018. In January, it acquired Belgian stem cell company TiGenix NV for $625 million, set up a drug development deal with U.S. degenerative diseases biotech Denali Therapeutics Inc. for $150 million, and obtained a range of over-the-counter medicines and food supplements for sale in Russia from Unipharm Inc. And the spending spree has continued into February, with a collaboration with Fujifilm Corporation to develop regenerative medicine for heart failure using cardiomyocytes derived from iPSCs.
This is all happening in the wake of the $725 million R&D restructuring program announced 18 months ago, under the auspices of Takeda CEO Christophe Weber. The restructure promised a new focus on oncology, gastroenterology, and the central nervous system, greater globalization, and more of a focus on acquisition and research collaborations, and the deal with Wave sits neatly within this.
"At Takeda, we are focused on partnering with companies that share our research focus… Wave's expertise in optimizing oligonucleotides offers a complementary approach to programs that Takeda is currently pursuing for neurological disorders," said Daniel Curran, head of the Center for External Innovation at Takeda.
The deal with Wave has two components. In the first, Takeda gains the option to co-develop and co-commercialize three programs, once Wave has demonstrated clinical proof-of-mechanism. These are: WVE-120101 and WVE-120102, which target the mutated huntingtin (HTT) gene and are in Phase 1b/2a trials for Huntington's disease; WVE-3972-01, which targets the C9ORF72 gene and clinical trials in amyotrophic lateral sclerosis and frontotemporal dementia are planned for the fourth quarter of 2018; and a program targeting the ATXN3 gene for the treatment of spinocerebellar ataxia type 3.
In the second component of the deal, Takeda snags the right to license a number of preclinical programs in CNS disorders, including Alzheimer's disease and Parkinson's disease. Over the four years of the research funding from Takeda to Wave, the two companies can collaborate on up to six preclinical targets at any one time. The milestone payments for these could be worth more than $2 billion, with potential pre-commercial milestone payments of over $1 billion.
Jefferies analyst Eun K. Yang was upbeat about the deal, writing in a company note: "From the announced global deal with Takeda, Wave maintaining full control of clinical development is quite positive, along with 50/50 profit share. With proof-of-concept data flow in 2019 and potential first approval in [around] 2020, we view upside potential outweighs downside risks."
Wave also retains the rights to its neuromuscular disease programs, including its lead exon skipping Duchenne muscular dystrophy candidate, currently in Phase 1, and the collaboration will help fund its further development.
"This partnership provides additional resources to advance our clinical programs through multiple data readouts while continuing to expand our pipeline in neurology and other therapeutic areas," said Paul Bolno, president and CEO of Wave Life Sciences.
This is Wave's second big pharma collaboration. In 2016, the biotech signed a five-target collaboration, combining Wave's antisense and RNAi expertise and Pfizer’s hepatic targeting technology to develop therapeutics for metabolic diseases.