One of our Fierce 15 honorees dove deep into the ocean for inspiration from the sea squirt to develop new human therapeutics. Another thought the adorable 13-lined ground squirrel, with its polka dots and hibernation habits, might provide a clue to new treatments.
And there’s the biotech that looked at the Human Genome Project and thought, what else can it do?
There’s a company looking at psychedelics in a whole new way, rising on the recent interest in repurposing the illicit substances for something better, like improving mental health.
And then, over in New Zealand, one tiny company is levying a fight against viral infections. It has been told its mission is outrageous, but we don’t think so.
Welcome to the 2024 Fierce 15. The Fierce Biotech editorial team is proud to present this class of the best in biotech. These 15 companies were carefully selected from hundreds of nominees.
This is the hardest project we do all year—to pick just 15 companies breaking barriers and defining the future of the industry. But it’s also the most fulfilling when we’re done and the most exciting to publish. So read on to find out who we picked this year.
Cour Pharmaceuticals
Retraining the culprits at the center of autoimmune diseases
Founded: 2013
Based: Skokie, Illinois
Clinical focus: Autoimmune diseases
What makes Cour fierce: Cour Pharmaceuticals hopes to bring more than four decades’ worth of research into the immune system to fruition with a novel type of reverse vaccine that aims to reprogram the body’s aggressive—if a touch overzealous—white blood cells and ultimately convince them to not attack otherwise healthy organs.
The startup’s goal is to build a targeted, platform-based approach that can address debilitating autoimmune diseases without blunting a patient’s ability to fight off an invading infection—and one that could potentially be tailored for a variety of different conditions.
With technology originally developed at the Northwestern University Feinberg School of Medicine in Chicago, Cour employs biodegradable nanoparticles that encapsulate certain antigens—specifically, whichever peptides or proteins are needed to match the ones responsible for triggering the chain reaction that drives T cells to destroy the body’s own tissues.
These nanoparticles hitch a ride on white blood cells known as monocytes as they travel to the spleen and liver. When those cells are recycled by the body, the antigens are released in a way that makes them look like normal dead cellular debris, which the immune system then learns to recognize.
Where a traditional vaccine would instruct these types of cells to attack a particular intruder, Cour’s strategy is to assure the immune system that they’re allowed to pass by unharmed.
“It was a 40-year endeavor trying to figure out how you get the immune system to be retrained so that it won’t attack the antigen of interest,” CEO John Puisis said in an interview with Fierce. “We are just very fortunate to hit the crossroads of a serendipitous scientific medical discovery.”
In the earliest stages of the company, the tactic was studied as a way to tamp down the dangerous inflammatory responses seen in people with severe peanut allergies. Today, Cour is focused solely on autoimmunity, with a pipeline that includes candidates targeting Type 1 diabetes, celiac disease and the muscle-weakening condition myasthenia gravis. And, so far, early results have been promising, according to the CEO.
“We can actually abrogate the disease,” Puisis said. “That’s what our data shows, which is really profound.”
In Type 1 diabetes, where T cells assault the insulin-producing islet cells of the pancreas, Cour plans to halt the progression of the disease.
The biotech’s CNP-103 program aims to present the body with 120 antigen epitopes by encapsulating four different recombinant proteins at once—and it’s slated to make a first-in-human debut next year through Cour’s phase 1b/2a trial.
The plan is to start the study with participants in the advanced stages of the disease—namely those with established losses of pancreatic beta cells who may be considering a tissue transplant, despite still having the autoimmunity—before developing it into a treatment for early diagnoses.
“Type 1 diabetes is still not terribly well managed today, despite improvements and continuous glucose monitoring,” said Chief Operating Officer Dannielle Appelhans. “But for addressing the underlying cause, there’s really nothing available.”
Meanwhile, Cour’s CNP-106 treatment for myasthenia gravis has cleared the proof-of-concept stage and is enrolling adults in a similar early to midphase study.
For celiac disease, the company has found a partner in Takeda, with a global licensing agreement inked in 2019 that includes a commitment of up to $420 million in potential milestone and royalty payments. That therapy—known as CNP-101 or TAK-101, depending on who you ask—smuggles in the gliadin proteins that serve as one of the two major components of gluten compounds.
In a previous first-in-human clinical trial of about 30 patients, the company demonstrated that within the weeks following two infusions, measures of inflammatory cytokine biomarkers as well as T-cell activation and infiltration into the small intestine were greatly diminished, even when study participants were subsequently given large doses of gluten.
“Doctors who diagnose patients with celiac disease give them a gluten challenge, and then they go look at the T cells,” Puisis said.
“What a few doctors told us was that, with our treatment, that person would no longer have been diagnosed. So take that for what it’s worth, but that’s a pretty profound change, and it was significant with a very small population,” he said. “I’m being cautiously exuberant.”
At the same time, Cour expects its products will be cheaper compared to gene therapies, despite falling under the same regulatory category as other cell-programming tech.
“What’s really interesting about what we’re doing is that we have this platform, but 98% of it is a polymer, and then the proteins are only 1% to 2%,” said Appelhans, who previously served as COO and CEO of cell therapy developer Rubius Therapeutics and as chief technical officer of Novartis’ gene therapy division. Appelhans will take over the CEO role from Puisis effective Sept. 1.
“So what really excites me is the potential to reach many patients across the world, because, while being an advanced modality, it doesn’t have the same cost structure associated with those.”
Investors: Lumira Ventures, Bristol Myers Squibb, Roche Venture Fund, Pfizer Breakthrough Growth Initiative, Alpha Wave Ventures