January 29, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Cambridge, Mass.-based WAVE Life Sciences Ltd. annouced today that it plans to advance six clinical programs between now and 2018.
WAVE focuses on stereopure nucleic acid therapeutics that target the underlying causes of rare diseases. The company’s technology platform attempts to purify the useless “uncontrolled steroisomers” out of complex chemical mixtures. In Nov. 2015, the company raised $112 million in its initial public offering, bringing total capital by the end of the year to about $196 million.
“Over the next three years,” said Paul Bolno, president and chief executive officer of WAVE, in a statement, “WAVE plans to bring multiple novel therapies into the clinic, offering new hope to patients with rare diseases and their caregivers. We believe that we have the leading genetic medicines platform and continue to build both the team and the infrastructure to execute on our vision.”
In 2016, the company expects to file two Investigational New Drug (IND) applications with the U.S. Food and Drug Administration (FDA). Both INDs are allele-targeted antisense programs targeting the underlying causes of Huntington’s disease (HD).
It also plans to advance therapies across multiple modalities, including antisense, exon skipping and single-strand RNAi, noting that its pipeline holds promise of “targeting the root genetic cause of over a dozen gain-of-function orphan disorders uniquely suited to the company’s proprietary chemistry.”
WAVE also plans to expand its pipeline across rare diseases, which had over 20 discovery programs. The diseases involved include Duchenne muscular dystrophy (DMD), Epidermolysis bullosa simplex, spinal muscular atrophy type 1, amyotrophic lateral sclerosis, myotonic dystrophy type 1, and diseases of the liver, skin, eye and gastrointestinal tract.
The company also announced that it had appointed Wendy Erler as head of patient engagement. Previously, she was Region General Manager at Biogen . Peter Lasky will join as head of human resources. He was formerly with Sekisui Diagnostics, and before that, he was senior vice president head of global human resources at Shire Pharmaceuticals.
“WAVE’s stereopure drug candidates have the potential to transform the nucleic acid therapeutic class in much the same way as humanization optimized monoclonal antibodies,” said Peter Kolchinski, managing general partner of RA Capital and a director of WAVE, in a statement. “WAVE’s progress over the past 12 months has been remarkable. The company has the necessary cash runway to deliver on the promise inherent in its technology. Undoubtedly, WAVE is posed for tremendous growth.”
WAVE’s is currently trading for $14.97. Zacks Investment Report indicates the company on Jan. 16 had been given a consensus rating of 1.00 (Strong Buy) from four brokers, which all four gave it. It has a consensus price target of $24.67 for the next year. On Dec. 7, 2015, Leerink Swann gave it an “outperform” rating with a price target of $20. Jefferies Group on Dec. 7 gave WAVE a “buy” rating and a $28 price target. JMP Securities, also on Dec. 7, gave it an “outperform” rating with a surprisingly high target price of $52. And SunTrust, also Dec. 7, gave the company a “buy” rating and a $26 price target.
