StemCell Therapy

For biotech, 2019 ended like the penultimate episode of a prestige TV show. We got answers to some weighty questions, but mostly, the year left a breadcrumb trail to some major reveals.

The IPO window stayed open, helping scores of companies go public. Futuristic therapies proved their worth in clinical trials, pointing to a new era in medicine. And the markets ended the year on a high, buoyed by a Food and Drug Administration that seems ever more flexible when it comes to approving new drugs.

Now, with 2020, well get the more important answers. Sure, theres a lot of public biotech companies now, but what if thats a bad thing? Yes, cell and gene therapies look transformational, but what if they never make any money? And since when is everyone so confident they understand whats going on inside the FDA?

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Here are three trends to watch in biotech in 2020, a year that looks to be laden with opportunities and stumbling blocks for the drug industry.

While every biotech startup is undoubtedly special in the eyes of the venture capitalists quoted in its press releases, 2020 could be a year marked by fatigue for the outside public.

More than 140 biotech companies have gone public since 2017, according to the analysts at Evercore ISI, and now theres upward of 500 of them trading on the Nasdaq. Keeping tabs on them all is essentially impossible, and its become fairly commonplace for biotech types to see the name of a given company for the first time by reading about its implosion.

Thats arguably a good problem to have in societal terms. More biotech companies means more efforts to treat human disease. But it could be problematic for the herd. Drug development remains an expensive proposition, and the majority of the biotech companies that went public in the past three years have negligible or nonexistent revenue. That means theyre going to have to go back to the market with follow-on offerings, and they may not like what they find.

According to Cowens biotech thermometer, a regular update on Wall Street sentiment, investors are increasingly selective when it comes to equity offerings, spooked by slumping IPO returns and a glut of supply. If that trend continues into 2020, some of those 500-plus biotech companies might need to look for other means of keeping the doors open, including mergers that thin the flock.

Much of the conversation around cell and gene therapies has focused on how much they cost, and understandably so. Two million dollars is, objectively, a lot of dollars. But the anxiety in biotech circles is a bit different: Is anyone going to make money on these things?

Take, for instance, CAR-T cancer therapy. For some patients, a single dose erases any trace of aggressive, otherwise untreatable cancer. For every patient, a single dose costs about $400,000. That sounds like a lot, but churning out a genetically engineered immune cell is hardly akin to widget manufacture. CAR-T companies dont disclose their underlying costs, but these therapies are understood to be low-margin products.

Theyre also considered commercial disappointments. The first two approved CAR-Ts, Kymriah and Yescarta, have underperformed analyst expectations to date. And that has stoked concern that a coming wave of gene therapies could face similar commercial difficulties.

Like CAR-T, gene therapy is costly to make, can be administered only at certain sites, and has made headlines for its six- or seven-figure list prices. Biotech companies and their investors have staked billions of dollars on the idea that such one-time treatments can become lucrative products. If that assumption is incorrect and the industry cant figure out how to make money in therapy, there could be a painful knock-on effect for biotech.

Handily, theres a one-company test case to follow in 2020. Novartis (NVS) sells a CAR-T in the form of Kymriah and a gene therapy called Zolgensma. Furthermore, thanks to a recent $9.7 billion acquisition, it will likely soon sell an RNAi treatment for high cholesterol. Each endeavor is a bet that futuristic science can turn into money-making medicines. By the end of the year, well have a decent idea of whether its a wise one.

Remember 2015, when the FDA would approve or reject a drug, and people would form an opinion and move on? That all changed the following year when the agency approved eteplirsen, now called Exondys 51, which is a treatment for Duchenne muscular dystrophy from a company called Sarepta Therapeutics (SRPT).

Without relitigating the whole ordeal, its fair to say Sareptas case relied on scant, debatable evidence from a small trial. To some, the FDAs decision to approve eteplirsen anyway was a sign of forward-thinking regulation that put patients first. To others, it was a dereliction of duty that threatened to erode decades of pharmaceutical jurisprudence. And to a great many, it was reason to get on the internet and be churlish, conspiratorial, and even threatening.

On Twitter, the fight over eteplirsen has never really ended, just taken on different forms, like a biotech analog to Gamergate. Earlier this year, the debate over a heart drug made by Amarin (AMRN) quickly metastasized into eteplirsen redux, with name-calling, accusations of bad faith, and armchair psychoanalysis of FDA staff. There were smaller but similar fights over Axovant Sciences, Clovis Oncology (CLVS), and nearly every biotech company with a sizable short interest.

Its at least somewhat understandable why eteplirsen marked such a shift in biotech discourse. Where FDA past decisions seemed to come down from Mount Sinai with little in the way of transparency, the messy eteplirsen process made public internal infighting and clashing personalities at the agency. The FDAs top drug evaluator even considered Sareptas balance sheet while evaluating the drug, a departure from the agencys hands-off approach to the business of biopharma and evidence that approval decisions can be about more than benefits and risks.

Theres no evidence that the FDA was fundamentally changed by a single decision, as organizations that employ 17,000 people rarely are. But that peek behind the curtain was enough to give credence to seemingly any biotech bull case online. Where the FDA once appeared monolithic, now there were heroes and villains within, actors whose imagined biases could support any conspiracy theory. Formerly anonymous public servants became the topic of vicious debate among strangers with alphanumeric Twitter handles and pictures of dogs as online avatars. One even got called a cuck.

With all that as a backdrop, next year, Biogen (BIIB) is going to ask the FDA to approve aducanumab, a treatment for Alzheimers disease. The supporting data are confusing, drawn from a pair of terminated trials with divergent results. The agencys decision will have major implications for the drug industry, the health care system, and the more than 5 million Americans with Alzheimers.

And, on the fractious little planet that is biotech Twitter, aducanumab presents an opportunity to play out the eteplirsen debate on the grandest scale yet, with more kremlinology, more circular logic, and more vitriol. Be nice to one another out there.

Continue reading here:
3 trends in biotech to watch in 2020 - STAT

This entry was posted on December 30, 2019 at 3:58 am and is filed under Gene therapy. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |