Praxis Precision Medicines Leads Way as Three More Biotechs Prep IPOs

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Praxis Precision Medicines kept mostly quiet about its research on central nervous system disorder (CNS) drugs until May, when it emerged from stealth and revealed $100 million in cumulative financing. Now the company is aiming to raise about that amount in an IPO that will fund clinical development of its drug pipeline.

Cambridge, MA-based Praxis is developing drugs intended to address the misfiring neuronal circuitry at the root of some neurological disorders. The company’s drugs are based on research into genetic mutations that regulate neuronal circuits in the brain whose dysregulation leads to the seizures in rare, inherited forms of epilepsy. But the company says in its IPO filing that additional research found that these genes also play roles in other neurologic and psychiatric disorders. Praxis is building on that research to develop drugs for a range of neurological conditions.

The most advanced Praxis drug candidate, PRAX-114, is a small molecule designed to target GABA, a neurotransmitter in the brain, as a way of treating major depressive disorder (MDD) and perimenopausal depression. While benzodiazepenes, a class of tranquilizing drugs, already target this neurotransmitter, these medicines come with side effects and aren’t currently approved for treating depression.

Praxis contends its tablet, a positive allosteric modulator of GABA that binds to a different site on GABA than benzodiazapenes, an approach that could offer a potent effect on depression and anxiety without the sedative effects associated with benzodiazepenes. The company points to another potential advantage compared to medicines currently in development for depression. For example, Sage Therapeutics (NASDAQ: SAGE) drug candidate zuranolone, which also targets GABA to treat MDD, has results in a published patent application that show its effect increased when taken after a meal, Praxis says in its filing. The company says it believes this food effect has led Sage to carry out clinical development with the requirement that the drug be taken with a high-fat meal.

“We believe that the ability to administer PRAX-114 with or without food is key for clinical and commercial success in MDD, as many patients struggle with adherence to medication and forcing a dietary regimen would impose further complications in this vulnerable population,” Praxis says in its filing.

In an ongoing Phase 2a study in Australia, Praxis says MDD patients treated with PRAX-114 showed “marked improvement” in depression scores within two weeks of treatment. The company plans to start a Phase 2/3 clinical trial in MDD in the fourth quarter of this year; preliminary results are expected in the second half of 2021. A Phase 2a study in perimenopausal depression is underway in Australia; preliminary data are expected in the second half of next year.

Another small molecule, PRAX-944, is in development for treating essential tremor. Praxis notes that the only FDA-approved essential tremor drug, the beta blocker propranolol, is poorly tolerated by patients. Another drug, the anti-convulsant primidone, is used off-label but can take six to eight weeks to find the right dose for maximum benefit. Furthermore, the drug brings sedative effects, balance problems, and an acceleration of osteoporosis with long-term use.

The Praxis essential tremor drug Praxis drug, which is currently in Phase 2a testing, is designed to block T-type calcium channels that research shows are a driver of the movement disorder. It’s the same target that Jazz Pharmaceuticals (NASDAQ: JAZZ) is aiming for with JZP-385, a drug that came via the acquisition of Cavion last year. That drug is currently in mid-stage testing. Praxis expects preliminary clinical data for its essential tremor drug will be ready in the first half of next year.

Praxis’s lead rare disease drug candidate, PRAX-562, is being developed for a range of rare CNS disorders, including pediatric epilepsy and adult cephalgia, a syndrome of cluster headaches. The drug is designed block sodium channels that modulate neurons, an approach also taken by other drugs. But Praxis says its drug is more selective, offering the potential for fewer side effects. The company expects to report Phase 1 data in the fourth quarter of this year.

Praxis was founded was founded in 2015 based on the epilepsy research of founders Kiran Reddy, David Goldstein, and Steven Petrou. According to the prospectus, the company has raised more than $282 million, most recently a $110 million Series C-1 financing in July. Blackstone is the company’s largest shareholder, with a 31.8 percent pre-IPO stake, according to the filing. Eventide Asset Management owns 12.9 percent; Vida Ventures owns 10.1 percent.

Praxis plans to use the IPO cash for Phase 2/3 clinical development of PRAX-114 in MDD, as well as mid-stage development of the drug in perimenopausal depression. The cash will also support Phase 2/3 testing of PRAX-944 in essential tremor. Additional funds will be spent on early-stage development of PRX-562. Praxis has applied for a Nasdaq listing under the stock symbol “PRAX.”

Two more biotechs joined the IPO queue. Here’s a look at their drugs and their clinical trial plans:

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