A small biotech wants to disrupt billion-dollar injectable drug markets with a single pill (PTGX)

Many of the powerful drugs used to treat autoimmune diseases have to be injected.
Protagonist Therapeutics wants to change that, instead of using the same approach but in a pill.
The potentially game-changing approach could disrupt billion-dollar markets.

Protagonist Therapeutics, a biotech company based in the Bay Area, is trying to develop pill versions of injected drugs. If it succeeds, it could one day disrupt billion-dollar drug markets.

Protagonist is working on treatments for inflammatory bowel disease, debilitating conditions that involve inflammation in the digestive system. Right now, the handful of powerful treatments available to patients are given as injections, and Protagonist wants to essentially make the same drugs but in the form of a pill. The approach uses drugs called "oral peptides," a field that has seen its fair share of setbacks and challenges.

But in May, the company got a big vote of confidence when Johnson & Johnson's pharmaceutical unit committed to giving Protagonist $50 million in a deal that could potentially pay out $990 million as one of Protagonist's drugs makes it through clinical trials.

While the treatments are still in early days, if the data reads out, it could have big implications for how auto-immune diseases like IBD are treated.

The elusiveness of oral peptides

Generally, peptides are structures made of amino acids that are smaller than proteins. They're found all over in the human body. Insulin, which is critical to managing blood sugar levels in the body, is a peptide. As such, our bodies are really good at digesting them, so it's hard to make a pill made out of them.

"Oral peptides are like the holy grail in the field of peptides," Protagonist CEO Dinesh Patel said.

That meant navigating the acidic environment, the gut microbiome, avoiding enzymes that want to break down peptides. Protagonist would subject the drugs to those challenges, and if the compound could stay stable without becoming less effective, it could be a viable candidate.

"In some instances, we had to take a step back to take two steps forward," Patel said.

So far, the team has been able to do that with a few different drugs, one of which is now in clinical trials.

Protagonist isn't the only company that's developing oral peptides. There are a few are already on the market like Linzess and Trulance, drugs that treat irritable bowel syndrome and chronic idiopathic constipation respectively.

What's different about Protagonist is that they're going after the same targets as drugs that are currently only available as biologic drugs that need to be injected. The first and farthest along is PTG-100, an alpha-4 beta-7 antagonist, which is the same target as Takeda's vedolizumab, a drug that made more than $1 billion in global sales 2016. The second, PTG-200, goes after the same target as J&J's blockbuster drug Stelara.

A long way to go

To be sure, these drugs aren't ready for prime time yet. It likely will be years until they're far enough in development to be ready for approval.

That's especially true for Protagonist's almost-$1 billion partnership with J&J for PTG-200, the one that's still in pre-clinical development that goes after the same target as J&J's Stelara. As part of the deal, J&J will pay out up to $990 million as Protagonist hits certain milestones in PTG-200's development. "The impressive point here is how early JNJ swooped in to lock in the drug," Ronny Gal an analyst at Bernstein said in a note after the deal was announced.

But if the data reads out, it could drastically change the way we use drugs to treat autoimmune diseases. Logistically, taking a pill is much easier and far less painful than an injection. Plus, while oral peptides are more expensive to make than your average pill, the drugs will likely still come in at a much lower cost than the biologic drugs available today.

From a treatment perspective, Patel said it might be possible to combine drugs with different targets to better treat the condition. Plus, by making the drugs simple pills, people might be able to start using them earlier, rather than trying other treatments first until the disease progresses.

And if this oral peptide platform pans out, they could go against really general drugs as well, like Humira. But for now, focusing closely on IBD targets.