Wave Life Raring To Go The Distance

wave sept19

Shares of Wave Life Sciences Ltd. (WVE) are down over 55% from their 52-week high of $56 recorded on September 24, 2018.

Wave Life Sciences is a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases like Duchenne muscular dystrophy, Huntington's disease and inherited retinal diseases.

The Company's lead drug candidate is Suvodirsen, a stereopure oligonucleotide, for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping.

Duchenne muscular dystrophy is a fatal, genetic neuromuscular disorder characterized by progressive, irreversible loss of muscle function, including heart and lung. DMD usually affects boys, and its prevalence is roughly 1 in every 5,000 newborn boys every year. Children affected by DMD lose their ability to walk and they will be confined to wheelchairs by late childhood.

About 13% of DMD patients are amenable to Exon 51 skipping. There is one FDA-approved drug for patients with Duchenne who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping, and that is Sarepta Therapeutics' Exondys 51. Approved in September 2016, the drug carries a price tag of $892,000 per year.

An open-label extension (OLE) study of Suvodirsen in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping is underway. The study was initiated in August 2018 with patients from the Phase 1 clinical trial. An interim analysis of dystrophin expression from muscle biopsies in boys receiving Suvodirsen in this study is expected in the fourth quarter of 2019.

If all goes well as planned, the Company expects to file for an accelerated approval of Suvodirsen in the United States in the second half of 2020.

Suvodirsen is also under a phase 2/3 trial, dubbed DYSTANCE 51. Initiated in June of 2019, this trial is expected to enroll approximately 150 boys who are between 5 and 12 years of age (inclusive) with a genetically confirmed diagnosis of DMD amenable to exon 51 skipping therapy.

Results of the DYSTANCE 51 trial are intended to serve as a basis of regulatory submissions globally.

The other clinical drug candidates in the pipeline are WVE-120101 and WVE-120102 proposed for the treatment of Huntington's disease, under a phase 1b/2a program, dubbed PRECISION-HD.

PRECISION-HD is a global clinical program consisting of the PRECISION-HD1 trial evaluating WVE-120101 targeting SNP1 and the PRECISION-HD2 trial evaluating WVE-120102 targeting SNP2. Approximately two-thirds of all HD patients are expected to carry SNP1, SNP2, or both, in association with the HD gene. SNPs are a common type of genetic variation.

Huntington's disease is a fatal, autosomal dominant disease, characterized by cognitive decline, psychiatric illness, and chorea, for which there are no approved disease-modifying therapies. There are about 30,000 people with Huntington's disease in the US, with another 200,000 at risk of developing the condition.

The Company expects to present the first topline results from the PRECISION-HD clinical program by this year-end.

Also in the pipeline are a number of preclinical candidates say, WVE-N531 to treat DMD in boys amenable to exon 53 skipping, WVE-C092 for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, and Stereopure oligonucleotides for the potential treatment of inherited retinal diseases.

Wave's Deals with Pfizer and Takeda

Wave Life Sciences has a Research, License and Option Agreement with Pfizer that was signed in May 2016, and amended in November 2017. The agreement is related to the discovery, development and commercialization of stereopure oligonucleotide therapeutics for up to five programs, each directed at a genetically-defined hepatic target selected by Pfizer.

Last February, Wave Life Sciences inked a global strategic collaboration with Takeda Pharmaceutical Company Limited to advance therapies for disorders of the central nervous system.

Takeda has the option to co-develop and co-commercialize Wave Life Sciences' programs in Huntington's disease (HD), amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and spinocerebellar ataxia type 3 (SCA3). In addition, Takeda also has the right to license multiple preclinical programs targeting CNS disorders, including Alzheimer's disease and Parkinson's disease.

Key Numbers:

Since inception, the Company has incurred significant operating losses, and as of June 30, 2019, had an accumulated deficit of $425.8 million. Revenue is generated under the Pfizer Collaboration Agreement.

In the first half of 2019, Wave Life reported revenue of $10.65 million compared to $6.3 million in the year-ago period.

The Company ended June 30, 2019, with cash of $252.9 million.

WVE has traded in a range of $18.16 to $56.00 in the last 1 year. The stock closed Wednesday's trading at $25.65, down 2.77%.

For comments and feedback contact: editorial@rttnews.com

Business News

Follow RTT