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Trump v. Drug Makers? And 6 More Burning Biopharma Questions for 2018

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than 42,000 Americans in 2016. In 2017, President Trump declare the crisis a public health emergency, but that did not come with new money to combat the addiction problem.

FDA commissioner Gottlieb is pressing his agency to do more, including greater emphasis on treating addiction with medication, including methadone. The National Institutes of Health has partnered with biopharma companies to speed up the development of new addiction medicines and non-addictive pain treatments. But NIH director Francis Collins has said that this initiative also needs government funding to really get off the ground. In 2018, we’ll see what, if any, actions government and the private sector will take as the death toll continues to mount.

Will Digital Health Provide More Alternatives to Pharmaceuticals?

Last September, Pear Therapeutics’ mobile app Reset received FDA clearance to treat substance abuse, a first for a so-called digital therapeutic. Evaluated as a medical device, Reset is an example of the growing overlap between non-pharmaceutical devices and drugs. 2017 also saw FDA approval of an Otsuka Pharmaceutical product that tracks whether patients have ingested the antipsychotic drug aripiprazole (Abilify) via a sensor in the pill and a wearable patch. It was the first approval of a digital system as a pharmaceutical product. Executives for Proteus Digital Health, the Redwood City, CA, company that developed the sensor, said the decision paves the way to digitally track other medicines.

Expect digital technology to continue blurring therapeutic lines. Akili Interactive Labs recently reported positive results for its video-game treatment for attention deficit hyperactivity disorder, and it will seek FDA approval in 2018. The company is also testing its approach in other neurological disorders such as Parkinson’s disease and traumatic brain injury.

To adapt to the technological changes, the FDA is working on new criteria for product evaluation. (Currently, the agency compares an experimental device to one already on the market—in some cases, to a product that’s decades old.) The FDA is accepting comments on its draft guidance.

What Will Patient-Centered Drug Development Look Like?

Passed with bipartisan huzzahs at the close of 2016, the omnibus 21st Century Cures Act, among many things, called for patients to have a stronger voice at the table during development and regulatory review of new drugs. As 2017 ended, the FDA was working to make it happen, with workshops on what it calls “patient-focused drug development” and guidance to companies detailing the kinds of patient experience data it wants—that is, how patients experience a disease and the treatments they’ve received—and how to collect those data.

These are not trivial changes. How will biopharma companies react to them? Patients groups have typically been involved at the clinical trial stage, helping recruit participants and providing input on trial endpoints. Now some companies are engaging with patients at earlier, preclinical stages of drug development. Many patient advocates say this is where the field is headed. But turning grand ideas into reality is rife with obstacles, and in this particular field, there is an extra layer of caution: When reviewing a drug, how should the FDA balance patient input with the drug’s clinical data, especially when faced with lackluster or negative efficacy data?

In 2018, as the FDA builds systems to incorporate patient data and input, it will need to find that balance, and drug companies could start to change the way they develop their products.

Is Microbiome Research Going to Help Us Anytime Soon?

The trillions of microbes in and on our bodies—especially in our intestines—are considered intrinsic to our health. The potential to exploit that knowledge and develop new medicines has resulted in a variety of startups, as well as internal programs and investments by pharmaceutical companies.

Efforts have yet to bear fruit, however, even in a disease where the cause-and-effect of an out-of-whack microbiome has been well established. People with stubborn gastrointestinal infections from the nasty C. difficile bacteria improve with a dose of someone else’s healthy poop. (Also known as a fecal microbiota transplant, or FMT.)

It was a shocker, then, when a pioneer in the sector, Seres Therapeutics (NASDAQ: MCRB), reported a failed Phase 2 pivotal study of its microbiome therapeutic for C. difficile that the company had previously touted as an improvement upon FMT. (Seres’s product, SER-109, was a mix of “good” bacterial spores from healthy donor feces that would take root in a patient’s gut and drive out the C. difficile.)

Meanwhile, another pioneer in the field, Second Genome, keeps shifting targets for its lead drug. First it was Crohn’s disease, then it was ulcerative colitis, and now it’s nonalcoholic steatohepatitis (NASH), the firm said just before the holiday break.

Seres and others, including Enterome, Finch Therapeutics, and Vedanta Biosciences, are pushing ahead with clinical tests using bacteria to treat C. diff and other gastrointestinal diseases. Beyond that horizon, academic and industry researchers are exploring more far-out links between the microbiome and conditions such as cancer and autism. If the therapeutic solutions that seem more obvious—those linking gut diseases and the microbiome—continue to prove elusive, perhaps the mysteries of our microbes and ourselves might be harder to solve than we think.

Here’s a fun bar bet to propose when you’re out one night at J.P. Morgan. Which will succeed first: An effort to use natural bacteria as a therapy? Or an effort to engineer bacteria into tiny drug-making factories?

Photo by Jeff Turner via a Creative Commons 2.0 license.

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