After a long lull, investments are pouring back into brain drug research. In 2023, M&A deals centering on central nervous system prospects were second only to oncology, according to an IQVIA report.
But while enthusiasm is high in the push to develop new drugs for neurological disorders like epilepsy, Alzheimer’s and Parkinson’s disease, the vast majority of investigational medications still don’t make it far — and 92% fail when they reach human testing.
“Neuro has one of the highest failure rates and one of the highest investment rates and that means something is wrong,” said Alif Saleh, CEO of AxoSim, a Louisiana-based biotech with a brain-on-a-chip platform.
Not only are the scientific hurdles in brain research higher than other areas but most early-stage testing for toxicity and efficacy is conducted in animals whose brains are no match for the complexity of the human organ, he said.
The FDA Modernization Act 2.0 has enabled a shift away from animal models by eliminating the requirement for these preclinical tests to gain drug approval. Now, the door has been swung open to computer modeling, AI-driven tests and organ-on-a-chip options like AxoSim. Other companies in this growing preclinical testing space include Hesperos and Emulate, which use organ-on-a-chip technology, and Quris AI, which has a bio-AI clinical prediction platform.
A brain-on-a-chip
Saleh joined AxoSim in June to help commercialize its brain-on-a-chip technology which leverages 3D human brain organoids to screen drugs for safety and efficacy.
In 2023, AxoSim acquired Vyant Bio subsidiary StemoniX’s microBrain drug discovery technology platform for neurological disorders. The $2.25 million deal added to the company’s suite of technologies, including AxoSim's NerveSim and BrainSim platforms, giving AxoSim broad applications for central and peripheral nervous system-based conditions, Saleh said.
“It's all about driving pharma adoption [and] standardizing the tools around the platform so it’s easy to use."
Alif Saleh
CEO, AxoSim
The company’s specialization in brain-related technology gives them a strategic advantage over the competition, he said.
“The majority of companies are going after multiple organs –– lung, heart and brain,” Saleh said. “What I’ve learned in my 15-year career bringing tools and platforms to pharma is that you need to be focused on one disease area.”
He said the company’s platform may also give it advantages over those using computer modeling because it offers an actual measurement instead of a prediction. A study of AxoSim’s technology found it attained better specificity than animal models (93% versus 30%), although the animal model performed better on sensitivity (75% versus 53%).
Getting pharma on board
AxoSim leaders are now focused on rolling out their technology to more companies.
“It's all about driving pharma adoption, standardizing the tools around the platform so it's easy to use, can ramp up quickly, can scale quickly, and then getting more and more customers to use it,” Saleh said.
The company has already reeled in some big fish in the industry to use the platform, although AxoSim has yet to disclose any partners.
“Pharma always likes to dabble but we’ve gotten out of the dabbling phase with a number of them,” he said.
While the AxoSim platform currently only tests for toxicity and efficacy, Saleh said the plan is to also measure the effects of drugs on other brain functions, such as memory and learning.
“If you can solve that you're in a pretty good spot commercially,” he said.
