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Marcio Souza

President and Chief Executive Officer, Praxis Precision Medicines, Inc.

Search every verified Marcio Souza interview, podcast appearance, and on-the-record quote — each transcript cross-checked by AI and human review to confirm speaker identity. Marcio Souza, President and CEO of Praxis Precision Medicines, has been active in rare epilepsy community events and discussions about the company's clinical programs. At the 2025 SCN8A Clinician, Researcher, and Family Gathering, he stated that the company was stopping the EMBOLD study due to "tremendous efficacy" in an interim data cut, with patients moving to open-label treatment with relugacine and the company working with the FDA on next steps for a new drug application. In a 2024 industry panel, he discussed clinical trial design, arguing that traditional phase classifications are a "disservice" in rare disease contexts and emphasizing the importance of understanding drug pharmacology, safety, and effectiveness over rigid trial phases. Souza has also spoken about Praxis's broader CNS portfolio and approach. In a 2024 podcast interview, he described the company's genetics-inspired strategy for developing treatments for brain diseases, highlighted the essential tremor program as potentially the first new drug in that indication in 60 years, and discussed the challenges of stigma around CNS disorders. In earlier presentations on PRAX-222 (elsunersen) for SCN2A-related epilepsy, he reported encouraging initial results, including dramatic seizure reductions after dosing, and emphasized the link between reducing seizure frequency and preventing sudden unexpected death in epilepsy. He has also discussed the company's decision to develop two drugs for the same SCN2A population, citing a commitment to the community and recognition that patients may respond differently to different treatments.

Recent appearances

  • 2025 SCN8A Clinician, Researcher, and Family Gathering

    December 27, 2025 · 📺 The Cute Syndrome

    The Cute Syndrome Foundation's 11th Annual SCN8A Gathering on December 5, 2025, in Atlanta, GA. Thank you to our Virtual Sponsor, Praxis Precision Medicines. 00:00 Welcome, Kacie Craig 10:30 SCN8A family presentation, Sheress Bowen 25:30 Utilizing Base Editing to Target SCN8A Epileptic Encephalopathy, Caeley Reever 44:20 SCN8A Italia update and grant announcement, Cinzia Scarcelli 46:25 Advancing Clinical Management of SCN8A-Related Disorder through Machine Learning, Joshua Hack 1:01:45 CRISPR Activation for Sodium Channel Disorders: Insights from SCN2A Haploinsufficiency, Perry Spratt 1:23:…

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  • Industry Panel: 2024 SCN8A Clinician, Researcher, and Family Gathering

    March 03, 2025 · 📺 The Cute Syndrome

    2024 SCN8A Gathering Industry Panel with Neurocrine Biosciences and Praxis Precision Medicines. This panel brought industry experts together to discuss clinical trials, drug approval, and the challenges of developing treatments for rare diseases. Panelists explained trial phases, placebo-controlled studies, and what families need to know about enrollment and drug development timelines. They also highlighted ways families could support research and advocacy efforts. Panelists: Hillary Savoie, The Cute Syndrome Foundation Kelley Del Real, Praxis Precision Medicines Marcio Souza, Praxis Precisi…

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  • 33 - Marcio Souza, CEO of Praxis Precision Medicines

    February 05, 2024 · 📺 Slice of Healthcare Network

    Join us on the latest episode! Our Guest: Marcio Souza, CEO of Praxis Precision Medicines. What you'll get out of this episode: Discussion on innovative CNS drug development on BioBreakthroughs podcast. Learn about Praxis' genetics-inspired approach to tackling complex brain diseases. Souza highlights the challenges and opportunities in CNS treatment, including the impactful EMBOLD and EMBRAVE studies. Discover Praxis Precision Medicines' future prospects and their commitment to revolutionizing patient care in the CNS field. To learn more about our guests and their companies: LinkedIn:   / m…

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  • Prax 222 (elsunersen) Phase II initial results.

    October 07, 2023 · 📺 SCN2A Australia

    Watch a recent presentation by Marcio Souza, CEO of Praxis Precision Medicines provides an update of the Prax 222 (elsunersen). This ASO program for Gain of Function presentation has been dosed in 5 patients now with initial results looking promising. #SCN2A #cureSCN2A #ASO #raredisease

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  • Exploring Breakthroughs: Initial PRAX-222 (elsunersen) Results in SCN2A Patients

    October 02, 2023 · 📺 FamilieSCN2A Foundation

    Join Shawn Egan, CSO of the FamilieSCN2A Foundation, as he hosts an interview with Praxis Precision Medicines CEO, Marcio Souza, CSO, Steve Petrou, and Chief of Staff Brian Spar. They discuss the promising initial clinical data readout for PRAX-222 (elsunersen), their ASO in development for SCN2A-related epilepsy, highlighted by encouraging seizure reduction and a favorable safety profile in both their initial cohort of 4 patients and in another patient dosed through emergency access. Additionally, learn about what's next for their clinical programs in SCN2A-related disorders. Learn more abou…

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  • Driving Mission Momentum at Praxis with WorkBoard

    March 31, 2022 · 📺 WorkBoard

    With mission urgency, Praxis dreams big and leans fearlessly into the red. Marcio Souza, President, and CEO of Praxis Precision Medicines shares how their organization aligns on outcomes and provides radical transparency to bring life-changing treatments to the patients who need them. Learn more about how Praxis drives mission momentum here - https://www.workboard.com/accelerate-...

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The summary and quote tags on this profile are produced with AI assistance from verified, first-person interview transcripts, then checked by our team to confirm the speaker's identity and the accuracy of every quote. See how we verify →