Mining clues from libraries of brain tissue samples, Cerevance takes a precision approach to central nervous system diseases

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focus: Craig Thompson, CEO of Celebance

Cerevance’s unique advantages: The quest to unlock the mysteries of neurodegenerative disease has long centered on studying the human brain, and UK-based Celebance is leveraging that approach with what CEO Craig Thompson believes will be one of the biotech industry’s largest collections of in-house donated brain tissue samples.

Celebance, which was founded in 2016 with funding from Takeda Pharmaceutical and a license agreement for the company’s NETSseq drug discovery platform, has built a library of 16,000 brain samples from donors aged between 8 and 104 at its Cambridge lab. What’s unique about this growing “brain bank” is that it contains both samples from people who have died from central nervous system diseases like Alzheimer’s, as well as samples from donors who died from other causes, Thompson says, which allows it to build a more detailed picture of disease progression.

“We see insights that others don’t see,” Thompson said.

To gain these insights, the company identified about 12,000 genes for each cell type in the tissue samples and sequenced more than 60 trillion genetic base pairs. Using machine learning and AI tools, Celebance stitched together data from “different donors at different stages of the disease,” Thompson said.

By analyzing the gene expression profiles of different cell types and determining which cells are up- or down-regulated at different stages, Cerevance was able to zero in on new therapeutic targets.

Why is this important: While the dopamine pathway has been a mainstay for most drug developers in the Parkinson’s field, Serevance is taking a different approach with its lead candidate, CVN424. The drug, in mid-stage trials as both an adjunct and monotherapy, targets the G protein-coupled receptor GPR6.

“GPR6 is only expressed in one cell in the striatopallidal pathway, which is important because it allows us to give higher doses and know the effect is restricted to that cell type,” Thompson said.

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“Our focus is to bring precision medicine to neuroscience and explore new ways to treat patients.”

Craig Thompson

Celebance CEO

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If approved, CVN424 could be administered orally, giving it an advantage over potential competitors that require injections. But the key will be for it to show better efficacy with fewer side effects than other oral medications, Thompson said.

“What we’re seeing is close to the effects that would only be achieved with an infusion product, but with this drug the side effects are minor,” Thompson said.

Maintaining fundraising momentum: Celebance Rise $47 million in new funding The company advanced its clinical research through an extended round of Series B-1 funding last April. This funding injection will enable the company to advance CVN424 into Phase 3 trials as an adjuvant therapy, where the drug has proven safe and effective in alleviating symptoms. Phase 2 Study Thompson thinks this is a category with the potential to be a big hit.

“Sales (as an adjuvant therapy) could be in the $1 billion to $3 billion range with moderate market penetration,” he said. “Any penetration rate above 25 percent would get you above that level.”

The company’s pipeline also includes earlier-stage drugs targeting multiple psychiatric disorders, including schizophrenia, as well as Phase 1 candidates for Alzheimer’s disease and ALS that target a potassium channel called KCNK13.

Thompson said Celebance has been “successful in raising capital” in recent years despite a “tough” investment market. Recent successes in the central nervous system, such as Eli Lilly’s Alzheimer’s drug, are Kishunra’s Approval This month, it demonstrated market potential in a field long hampered by failed clinical trials.

“New investors are becoming interested in our story,” Thompson said.

With clinical results for CVN424 as a monotherapy for Parkinson’s disease expected later this year, Celebance is considering a possible IPO, but that decision may be subject to market conditions.

“We don’t want to see another biotech go public and then trade at 25% of its offering price,” Thompson said. “We want to look at the broader market and see where we stand. But bankers are telling us there is more investor interest given their later-stage portfolios.”

Meanwhile, Celebance announced in May that it had achieved the first milestone in an ongoing project. Research collaboration with Merck The deal is to find new targets for Alzheimer’s. Celebance didn’t disclose how much it was paid for fulfilling the contract, or how many targets the collaboration is looking for, but Thompson said the collaboration fits into a broader strategy to use the brain bank and the NETSseq platform to find new ways to attack neurodegenerative diseases.