Pfizer will partner with WAVE Life Sciences to develop nucleic acid therapies for metabolic diseases, under a collaboration that could generate up to $911 million-plus for WAVE.

The collaboration, disclosed today by WAVE, will combine that company’s proprietary stereopure drug development platform, which allows for designing therapeutics across antisense, RNA interference (RNAi), and other modalities, along with N-acetylgalactosamine (GalNAc) and Pfizer’s hepatic targeting technology aimed at enhanced delivery to the liver.

WAVE agreed to advance up to five programs from discovery through to the selection of clinical candidates. At that point, Pfizer may exercise its option to license the programs exclusively and undertake further development and any commercialization.

WAVE said it has already agreed with Pfizer on two of the targets—one of which will be WAVE’s apolipoprotein C-III program—with the remaining targets to be declared within 18 months.

Under the collaboration, WAVE has received rights to Pfizer's hepatic targeting technology, which WAVE may use for hepatic programs beyond the collaboration. Should WAVE use Pfizer’s technology, the pharma giant is eligible for unspecified payments from WAVE tied to achieving development and commercial milestones, as well as tiered royalties on sales of any products that include Pfizer’s hepatic targeting technology.

Pfizer agreed to pay WAVE $40 million upfront, of which $30 million will consist of an equity investment in WAVE at a price of $16 per share—9% above the biotech’s $14.63 closing share price yesterday on NASDAQ.

Should five products be successfully developed and commercialized as planned, WAVE said it may earn up to $871 million in potential research, development, and commercial milestone payments from Pfizer, plus royalties, tiered up to low double-digit percentages, on product sales.

Headquartered in Cambridge, MA, WAVE has committed to launching six clinical programs by 2018, including three anticipated IND filings by year’s end and early 2017. The company’s pipeline consists of more than 20 discovery programs in therapeutic areas that include the central nervous system, muscle, eye, skin, gastrointestinal, and liver, the area addressed by the collaboration with Pfizer.

WAVE raised a total of approximately $196 million in capital last year, led by a $112 million initial public offering, and including a $66 million Series B financing completed in August 2015, and $18 million Series A financing in January 2015.

“We expect that our existing cash together with the upfront payment will enable us to fund our operating expenses and capital expenditure requirements into 2019,” WAVE President and CEO Paul Bolno, M.D., MBA, said in a statement. “This alliance is consistent with WAVE’s strategy to build and advance a portfolio of medicines for neurological and neuromuscular diseases, while working with partners with deep expertise in other important therapeutic areas.”

Cardiovascular and metabolic diseases comprise one of Pfizer’s six therapeutic areas. The other five are immunology and inflammation, neuroscience and pain, oncology, rare diseases, and vaccines.

“By working together to develop unique, proprietary technologies emerging from both companies, we will explore new liver-targeted approaches to address the cause of genetically defined diseases and interrupt the progression of complex, metabolic disorders,” added Morris Birnbaum, M.D., Ph.D., svp and CSO with Pfizer’s Cardiovascular & Metabolic Research Unit.








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