Five ‘Big Idea’ Biotech Trends to Watch in 2017

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Different types of stories cross my radar as a biotech journalist every day.

There are partnerships between established drugmakers that need new products, and the little aspiring companies that need cash to develop their thing. Stories about clinical trial results that move markets. Regulatory actions that make or break companies and investors.

But for me, there’s something especially fun about talking with scientific entrepreneurs who raised their first big wad of investment cash. They’re latching onto some kernel of insight from a discovery. Or maybe they have a vision for stitching together a set of technologies. Either way, they are venturing into a great unknown to see if they can make a big step ahead in medicine. They often have insights, big ideas, that put them years ahead of the lumbering drug giants.

The odds are stacked against the creators. But even in failure, startups can sometimes offer clues on where medicine is heading. Sometimes outside groups will see what’s wrong and fix it. And yes, sometimes the startups will succeed, going public or getting acquired.

Here were a few trends I observed from covering startups which raised issues that will remain relevant in the year ahead.

New ideas for the treatment of Alzheimer’s

Alzheimer’s disease is a demographic iceberg. As the baby boomers get older, the national healthcare bills for dealing with it are astronomical—with one analysis estimating Alzheimer’s will gobble up one-fourth of Medicare spending in 2040. The pharmaceutical industry has had no effective response yet. Billions of dollars have been invested in targeted antibody drugs that are supposed to help clear out the buildup of amyloid-beta protein plaques that are thought to gum up memory and cognition as we age. But we’re beginning to see some signs of support for alternative scientific approaches. Cambridge, Mass.-based Yumanity Therapeutics raised $45 million to create a new drug discovery engine for Alzheimer’s and other neurodegenerative diseases. South San Francisco-based Annexon Biosciences, a Stanford University spinout, raised $44 million to build on research that suggests you can fight Alzheimer’s by stopping the immune system from from removing synapses we need for neuronal functioning. And earlier this month, a bootstrap startup I wrote about in Timmerman Report, EIP Pharma, showed that an old anti-inflammatory drug was able to improve cognition and memory in a small number of patients with mild Alzheimer’s. Given the urgent need, the enormous market, and the string of failures with anti-amyloid antibodies like Eli Lilly’s solanezumab, I suspect we’ll see pharma spread its eggs around into multiple baskets in the year ahead.

Scientists At The Movies

Much of drug discovery is based on static images of a molecular target. Chemists want to make compounds that can bind with the specific target. But many of these targets are fluid in real life, and their shape-shifting dynamics make them especially hard targets. What if instead of looking at a still photograph, you could watch a series of images, a “movie” to see how the protein targets folds in different configurations? Could you gather deeper insight into the particular toeholds that a drug could bind with? Two companies in the Boston area, Relay Therapeutics and Morphic Therapeutic, each raised more than $50 million in their Series A venture financings to see if they can better capture the fluidity of certain protein targets and use that knowledge to discover drugs. This is a big idea that big organizations are watching closely – Morphic is backed by Pfizer, GSK and AbbVie, while Relay Therapeutics was started by Third Rock Ventures and D.E. Shaw Research.

Solving the Problem, Not Just Pushing Drugs

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