Equipped with fresh $100M, CAMP4 heads up trail to the clinic, hoping partners will soon tag along

CAMP4 Therapeutics is bound for the clinical wilderness saddled with an additional $100 million, sealing a series B aimed at expanding the company’s platform and taking a lead asset into phase 1 trials. For now, the company is backpacking solo, but partners may soon join the journey. 

The latest round announced Wednesday was led by Enavate Sciences, an offshoot of Patient Square Capital, along with Gaingels, a venture capital firm that prioritizes investments in companies with diverse leadership. It comes just over a year after CAMP4 tacked on $45 million in additional funds, but, amid a market reset, no cash cushion is too soft.

In an interview with Fierce Biotech, CEO Josh Mandel-Brehm said his relationship with Jim Momtazee, managing partner at Patient Square, began shortly after the June 2021 raise, at which point Mandel-Brehm said all eyes were on the clinic. 

“I think that resonated with that group, not only because of the potential of the science, but he had taken the time to get to know our team and how we operate and that we can execute,” he said. 

The platform focuses on regulatory RNA (shortened to regRNA), a largely untapped sibling of the RNA family because it's been thought that it doesn’t work with portions of the genome that translate to proteins. But CAMP4 says that’s changing, with new science indicating regRNAs control expression of nearby protein-bearing genes—and the company’s RNA Actuating Platform was built to find them all. Where regRNA is found to play a role in gene expression that’s related to protein production, the company is designing antisense therapeutics to upregulate expression.

Mandel-Brehm and Chief Scientific Officer David Bumcrot, Ph.D., said that much of the new capital will funnel toward designing those drugs and bolstering development of what’s already in the clinic. 

CAMP4 has yet to lock in any collaborations with other pharmas since partnerships with Alnylam and Biogen ended, but that may soon change. Mandel-Brehm says in the last month, business development conversations have advanced with the goal of nailing one or two “big partnerships.” 

“We don't want a lot of little small relationships, because, unfortunately, those are not so efficient,” he said. “So it'll probably be one or two big ones.”

So far, the company has five programs targeting the central nervous system and liver that the latest investment will help fund including two focused on urea cycle disorders. But CAMP4’s priority is its lead asset for Dravet syndrome, which spurs severe seizures. The company expects to land in the clinic with the med midway through 2023. Additionally, Bumcrot says the aim is to get the company's treatment for urea cycle disorders of all enzyme deficiencies into the clinic by the end of next year. As for future development, Bumcrot feels the platform is likely amenable to finding targets in the cardiac and skeletal muscles. 

In preparation for its clinical aspirations, CAMP4 recently hired Ann Barbier, M.D., Ph.D., as chief medical officer, plucking her from the same role at Translate Bio. Barbier brings relevant experience, having overseen the clinical development of Translate’s RNA-based cystic fibrosis treatment. The company was ultimately bought by Sanofi in August 2021.