Carlsbad-based Isis Pharmaceuticals Inc. said it has entered into agreement with biotechnology giant Biogen Idec of Weston, Mass., to develop and commercialize an investigational drug for a rare genetic disorder known as spinal muscular atrophy, which is often deadly in infants and young children.
Publicly held Isis could receive as much as $299 million plus royalties from Biogen. That total includes $29 million upfront plus $45 million in development milestones. If Biogen eventually licenses the drug, it will pay up to $225 million more in fees and payments tied to regulatory and sales milestones.
“We believe that, together with Biogen Idec, we will be able to expeditiously develop this investigational drug in hopes of bringing to market an effective and desperately needed treatment to improve the lives of children with SMA,” Stanley T. Crooke, Isis chairman and CEO, said in a statement.
Isis in December launched phase 1 clinical trials of its drug candidate ISIS-SMNRx, which has already won fast-track status and an orphan drug designation from the Food and Drug Administration. The orphan program is designed to encourage drug development for diseases that affect fewer than 200,000 people in the U.S.
Spinal muscular atrophy, or SMA, easily falls into the “orphan” category, affecting approximately 30,000 to 35,000 patients in the U.S., Europe and Japan combined, Isis said.
While it’s a rare condition, SMA is the most common genetically caused death in infants and toddlers, according to the New York-based Spinal Muscular Atrophy Foundation. The disease leads to a progressive loss of muscle control and movement, and increasing weakness due to the loss of motor neurons in the spinal cord.
“SMA is a heartbreaking disease — it can kill children before their second birthday and there are currently no therapies to treat the disease,” said Biogen CEO George A. Scangos.
ISIS-SMNRx is a disease-modifying therapy that’s designed to compensate for the underlying genetic defect causing SMA. Scangos said that the compound has the potential to be a highly effective, first-to-market drug for the deadly disease.
Under terms of the collaboration agreement, Isis will continue to develop the drug through mid-to-late stage clinical trials, with Biogen providing advice on trial design and regulatory strategy. At the end of those trials, Biogen will decide whether to exercise its option to license the drug, which would give the company control over global development, regulatory approvals and commercialization.
Stock analyst Jim McCamant of the Berkeley-based Medical Technology Stock Newsletter said he suspects Biogen might not have been the only company looking to make a deal with Isis.
“Isis usually waits until after proof-of-concept before partnering programs, suggesting there may have been competition to get a hold of a promising drug for a potentially lucrative orphan indication,” McCamant said in his most recent report.
The investigational SMA drug ISIS-SMNRx is among more than two dozen products in Isis’ pipeline that have been discovered using the company’s proprietary antisense platform. Antisense drugs work by binding to RNA molecules, which are made up of four building blocks called nucleotides.
Isis says that antisense drugs have a higher level of specificity than traditional small-molecule drugs and can be created for conditions that are normally considered “un-druggable.”
Ranked 20th on the San Diego Business Journal’s list of Largest Public Companies with market capitalization of more than $1 billion as of January 2011, Isis has drug candidates targeting cancers, metabolic disorders, inflammation and other conditions.
McCamant said that 2012 “may finally be the year that Wall Street and the world wake up to the potential of antisense therapeutics.”
While the Biogen-Isis deal is notable in size and scope, the most significant piece of antisense news this year could be the pending FDA approval for Isis’ lead drug candidate Kynamro, McCamant said.
The drug is designed to treat patients with very high cholesterol and who are at risk of heart disease; these patients are often diagnosed as having familial hypercholesterolemia, a genetic disease.
Isis licensed the drug in 2008 to Genzyme, which was acquired by France’s Sanofi-Aventis SA in February 2011. Isis and Genzyme already submitted the drug to European Union regulators, and expects to submit a New Drug Application to the FDA in the first quarter of this year.