Cortexa Therapeutics, Inc., a biotechnology company based in Phoenix, Arizona, has announced progress in its scientific validation process as it advances through Phase 1b and Phase 2 studies. These studies are being conducted with the support of a contract research organization partner in Scotland.
The current research phases aim to validate Cortexa’s biological target and reinforce the scientific foundation for its proprietary therapeutic approach. The company expects both phases to conclude early next month, with data to follow soon after.
During Phase 1a, Cortexa encountered issues with the degradation of housekeeping genes used for qPCR normalization. In response, the company shifted its methodology to RNA sequencing for Phase 1b. This change allows for unbiased transcriptomic profiling and eliminates reliance on unstable genes. It also improves statistical robustness and increases the credibility of the dataset.
Phase 2 moves beyond RNA analysis by focusing on protein quantification. Researchers are measuring receptor protein levels and subunit composition, particularly examining ratios between calcium-permeable and calcium-impermeable receptor populations (AMPA vs. NMDA). Quantifying these receptor imbalances is central to Cortexa’s mechanistic framework.
“If receptor imbalance can be quantified, it can be rationally targeted,” said Nathan Cole, Founder of Cortexa Therapeutics. “Our current work is about building that quantifiable foundation.”
The company also highlighted recent scientific literature that underscores the role of sex biology in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS). A review published in Ageing Research Reviews (Zamani et al., 2024) notes that sex differences affect ALS incidence and progression, with hormones like estrogen and testosterone contributing to disease variability. The review emphasizes that sex-specific biology should inform both research design and therapy development.
Following completion of Phases 1b and 2, Cortexa plans to integrate transcriptomic and protein data sets, refine its disease model, prepare materials for publication, expand investor outreach efforts, and move toward compound validation phases.
“The foundation is being built now,” Cole added. “We look forward to sharing results soon.”
Cortexa Therapeutics focuses on advancing mechanistic insights and developing rational therapies for neurodegenerative diseases.
