What You May Have Missed in Biotech: Eye Drug Fails, Ear Drug Flops, and Herpes Drug Succeeds

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Biotech was full of news this week on clinical trials for the eyes, the ears, and other body parts people don’t usually talk about.

Catch up on your headlines here:

Eleven Biotherapeutics Fails in Dry Eye Clinical Trial
Cambridge, Mass.-based Eleven Biotherapeutics lost 75% of its value, ending the week at $3 a share, after the company said its experimental drug for dry eye failed in its first pivotal trial for moderate-to-severe dry eye disease. This study was not cheap, as it enrolled 669 patients and compared the new drug with a placebo. Eleven said it still has $59 million in the bank, and plans to advance the same drug into another pivotal clinical trial, this time for allergic conjunctivitis, an inflammatory condition of the eye.

Otonomy Whiffs in Ear Drug Study
San Diego-based Otonomy has been closely watched for some time because of its experimental drug for Meniere’s disease, which causes vertigo, hearing loss, and steady ringing in the ears (tinnitus). Unfortunately, the study, which randomly assigned 154 patients to get the new drug or a placebo, didn’t show any statistically significant advantage for the new drug. Even so, the company said it ‘narrowly’ missed its threshold for success, and it plans to discuss a bigger, pivotal phase trial plan with the FDA. Investors were skeptical, sending Otonomy’s shares down 20% on Friday.

Genocea Biosciences Passes Trial In Genital Herpes
Cambridge, Mass.-based Genocea Biosciences reported success this week with a mid-stage clinical trial of its experimental treatment for genital herpes. The study of 310 patients with genital herpes showed that the Genocea drug was able to significantly reduce viral shedding within 28 days, the main goal. A reduction in viral shedding is important not only because it’s a quantifiable measurement of anti-viral activity, but also because it reduces the risk of transmitting the disease to sexual partners. Equally important, patients in the study reported improvement in their genital lesions. Given the prevalence of genital herpes—which affects 400 million people worldwide—investors were pleased with Genocea’s opportunity in further confirmatory trials. Genocea stock climbed 16% this week.

Sarepta Therapeutics Soars on FDA Filing News
Sarepta Therapeutics saw a huge gain of 72% this week, according to FactSet, based on what amounts to investor speculation that the company has patched up a rough relationship with the Food and Drug Administration. The Cambridge, Mass.-based company said this week that it has begun submitting a New Drug Application for its experimental drug for Duchenne Muscular Dystrophy, and that it hopes to complete its submission by mid-2015. Of course, when the FDA allows a new drug application to be filed, that doesn’t mean it’s automatically clearing the drug for sale in the U.S. Sarepta watchers will just have to stay tuned for many more months to see if that happens.

Bluebird, Intercept Unveil Clinical Plans
Bluebird Bio and Intercept Pharmaceuticals both spoke out this week about their latest strategic talks with the FDA, and got different reactions. Cambridge, Mass.-based Bluebird Bio said that upon reviewing more encouraging follow-up data with its gene therapy for beta-thalassemia (see Adam Feuerstein’s coverage at TheStreet.com), regulators in the U.S. and European Union have agreed to Bluebird’s plans to run two pivotal studies which will each enroll 15 patients. If successful, those small studies will lay the groundwork for what would be a historic gene therapy approval. Investors cheered, sending the stock up 15.5% this week to close at $178.38 a share.

New York-based Intercept, a trailblazer in a different field, wasn’t so warmly received. The company is developing a drug for a fatty liver condition called non-alcoholic steatohepatitis (NASH). It agreed on the basic framework for its pivotal study with the FDA. This study will have to be much larger, however, with 2,500 patients, because NASH is a much more common condition. The study will take a long time (72 weeks of patient follow-up, plus longer-term measurements on liver function). It will surely cost a bundle. While investors drove down Intercept stock 14.6% this week, others in the industry weren’t so discouraged. Rosanna Kapeller, chief scientific officer of Nimbus Discovery, wrote an interesting blog about how Intercept is establishing a road map with the FDA which other companies will be able to follow.

Regeneron, Sanofi Pass Trial with Rheumatoid Arthritis Drug
Tarrytown, NY-based Regeneron Pharmaceuticals and its partner, Paris-based Sanofi, said this week that their experimental antibody drug for rheumatoid arthritis passed a pivotal clinical trial in 546 patients who didn’t respond well or couldn’t tolerate the standard drugs known as TNF-inhibitors. The Regeneron drug, which is made to bind with a different inflammatory molecule called IL-6, was able to reduce disease symptoms by 20% or more in 61% of patients who got the highest dose of the new drug. Infections and injection site reactions were the most commonly reported side effects. Regeneron stock just keeps on chugging, ending the week at $513.88.

23andMe’s Plan to Turn Into a Drug Discovery Company
Mountain View, Calif.-based 23andMe gets a lot of attention for its work in consumer-oriented genetic testing, but it has turned heads lately with its stated plan to use its deep database of genetic and patient-reported data to help it discover drugs. Two big-name hires from Genentech, Richard Scheller and Robert Gentleman, recently joined the company to see what they can do with the data. Gentleman offered some interesting comments this week in an interview with Bio-IT World.

Pfizer R&D Exec Quits to Join Synthetic Biology Startup
Jose-Carlos Gutierrez-Ramos, a former senior vice president of research and development at Pfizer, made news this week when he said he was joining a fledgling startup in Cambridge, Mass. called Synlogic. The idea, which I wrote about at Timmerman Report (Subscription Required), is to genetically program in specific circuits into live bacteria that can sense the environment they are in, and deliver specific doses of a drug a patient may need, like more insulin. It might sound like science fiction, but Gutierrez-Ramos, a well-known executive, said the company is on track to file its first applications to start clinical trials in 2016.

Amgen Dumps Autoimmune Drug
Biotech giant Amgen said Friday that it was dropping out of a collaboration with AstraZeneca to co-develop brodalumab, an experimental drug designed to inhibit an inflammatory protein called IL-17. The drug has been designed to treat chronic conditions like psoriasis, psoriatic arthritis, and ankylosing spondylitis. Amgen walked away after it saw “events of suicidal ideation and behavior” in patients in clinical trials. That could be a show-stopper, especially for a drug that would be taken on a chronic basis.

Luke Timmerman is the founder and editor of Timmerman Report, a subscription publication for biotech insiders.

Source: Forbes