Wave gains FDA orphan designation for Huntington’s candidate


Fresh from a major $911 million deal with Pfizer ($PFE) just last month, Wave Life Sciences ($WVE) has seen the FDA grant it an orphan drug designation for its lead candidate WVE-120101.

The experimental drug, which targets rs362307--a single nucleotide polymorphism that is associated with the disease-causing mutation in the huntingtin gene--is seeking an approval to treat the underlying causes of Huntington’s disease.

The orphan status gives it 7 years of exclusivity in the U.S., and other boosts, including tax credits related to clinical trial expenses, an exemption from the FDA user fee, and FDA assistance in clinical trial design.

The big idea at Wave, which also captured Pfizer’s attention earlier this year when it signed a near $1 billion R&D pact with the biotech, is that they can use new technology to create simple, effective nucleic acid therapies that modulate RNA to treat genetically defined rare diseases. Cambridge, MA-based Wave went public last November, raising just over $100 million.

Wave’s approach with WVE-120101 enables selective silencing of the disease-causing HTT allele, while leaving the healthy HTT allele to produce normally functioning protein. The biotech has two INDs planned for the drug to come at the end of the year.  

“We are pleased to receive orphan drug designation for our first Huntington’s disease therapy and to advance what may be the first allele-targeted therapy into clinical trials, particularly as there are no approved disease-modifying treatments for HD,” said Paul Bolno, president and CEO of Wave.

“WVE-120101 is the first of our two lead allele-specific antisense programs for HD, and we are on track to file investigational new drug applications for both in late 2016. Each program distinctly targets the underlying genetic cause of HD and between these two programs, a substantial majority of HD patients would be covered. Our team at Wave is driven by a sense of urgency and purpose as we work to advance disease-modifying treatments for HD patients.”

The National Institutes of Health estimates that around 30,000 people in the U.S. have HD. In addition to symptomatic patients, more than 200,000 also carry the gene and are at risk of inheriting the disease.

Just last month, the FDA turned down Teva’s ($TEVA) HD candidate deutetrabenazine (SD-809), with a CRL telling the Israeli company to carry out further testing on certain metabolites seen after dosing with the drug.

Currently, Lundbeck's Xenazine is the only approved drug for the condition, and made around $330 million last year. Teva's drug is a 'deuterated' form of VMAT-2 inhibitor tetrabenazine--the active ingredient in Xenazine product--with a modified structure that seeks to improve the safety and efficacy of the parent molecule. Neither however treat the underlying causes of the disease, as Wave is attempting to do.

The company also has an in-house program for an exon 51-skipping Duchenne drug aimed at increasing levels of dystrophin.

The biotech was founded by two world-renowned scientific leaders Gregory Verdine (from Harvard University) and Takeshi Wada (from the Tokyo University of Science).

Wave closed 6% up yesterday before the news was announced.

- check out the release

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