Praxis Shares Details of Pipeline, Garners $100M in Financing

Praxis is currently focused on leveraging its research on the genetics of epilepsy. Using its insights, the company is currently working on a pipeline of treatments that address the genes that control the imbalance of excitation and inhibition of neuronal circuitry.

Massachusetts-based Praxis Precision Medicines officially launched on Monday with more than $100 million in financing. It was primarily funded by investors including Blackstone Life Sciences, Novo Holdings, Vida Ventures and Eventide. In addition, it is now sharing insight into its research projects, which are based on the development of new neurological drugs. Praxis already has two programs in mid-stage testing.

“Praxis aims to leverage the recent breakthroughs in genetics to develop innovative medicines that can improve the lives of the many patients who need them,” said Nicholas Galakatos, Ph.D., chairman of the Praxis board of directors and global head of Blackstone Life Sciences.

Praxis is currently focused on leveraging its research on the genetics of epilepsy. Using its insights, the company is currently working on a pipeline of treatments that address the genes that control the imbalance of excitation and inhibition of neuronal circuitry.

Praxis’ portfolio is led by PRAX-114, a GABAA positive allosteric modulator, and PRAX-944, a T-type calcium channel blocker. Both are in Phase 2 development, with PRAX-114 being examined for the treatment of major depressive disorder (MDD), and PRAX-944 being researched for the treatment of essential tremor. Over the next year, Praxis intends to begin its first trial for PRAX-114 for MDD subjects. It will also report proof-of-concept data for PRAX-944 and advance earlier stage programs into clinical development for rare epilepsies and other neurological disorders.

“As was achieved in oncology decades ago, recent genetic insights have presented meaningful opportunities to treat brain disorders in entirely different and targeted ways based on the specific genetically validated pathways driving a patient’s disease,” said Kiran Reddy, M.D., co-founder and member of Praxis’ board of directors. “We are reducing these insights to practice, to create novel medicines that could fundamentally alter the treatment path and outcomes for patients with brain disorders.”

In April, Coherent Market Insights estimated that the global anti-epileptic drugs market was valued at more than $7 million in 2019. In addition, it expects the market to surge in value, surpassing $11 million by the end of 2027.

As of late, researchers have also started discovering drugs that can potentially help epilepsy patients with symptoms, such as anxiety. For example, results from a study published in April showed that perampanel can potentially relieve insomnia in epilepsy patients by decreasing anxiety.

The cross-sectional study, conducted at Asan Medical Center, looked at 126 subjects with epilepsy and aimed to analyze the effect of perampanel on their insomnia, according to NeurologyLive. The conclusions were drawn by the researchers through the analysis of questionnaires given to the subjects.

In the past, perampanel has been tested extensively in patients with epilepsy. It has been approved by the U.S. Food and Drug Administration as monotherapy for partial-onset seizures with or without secondary generalized seizures in epilepsy patients age 12 and older.

In January 2020, Eisai released data that showed that perampanel was generally safe and well-tolerated in children with focal seizures with and without focal to bilateral tonic-clonic seizures and GTCS.

Eisai also presented data at the 73rd annual meeting of the American Epilepsy Society in December 2019 that showed perampanel had favorable retention rates and sustained efficacy for adolescents for during routine clinical care.

The most common side effects of perampanel, which is sometimes recognized under the brand name Fycompa, are dizziness, headache and irritability, according to the Epilepsy Foundation.

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