Praxis Precision Medicines (PRAX) investor relations material

Pipeline progress and upcoming milestones

• Essential tremor program achieved successful readouts in both studies, with NDA filing anticipated early next year.

• Multiple epilepsy assets advancing, including relutrigine for rare SCN2A and SCN8A indications, with interim analysis ongoing and larger Emerald study readout expected next year.

• Two potential NDA filings and two clinical readouts are expected within the next six months.

• Additional readout for focal epilepsy is also anticipated.

Essential tremor study insights and regulatory alignment

• Early interim analysis by DMC suggested futility due to small sample size and high variability, but final results were clearly positive.

• Decision to continue the study was based on low opportunity cost and high potential upside.

• FDA alignment includes agreement on two prospective studies, study design, safety database, and use of mADL 11 as primary endpoint.

• Minor protocol changes, such as shifting primary analysis from week 12 to week 8, were made before unblinding and communicated to the FDA.

• Study succeeded at all time points and on both ITT and MITT analysis sets.

Commercial strategy and market opportunity

• Essential tremor market in the US includes 7 million patients, with 2–3 million actively seeking treatment.

• Recruitment efforts built a database of over 200,000 patients, providing insights for launch planning.

• Launch strategy targets 13,000–14,000 neurologists, requiring about 300 sales reps and a robust DTC campaign.

• Peak sales for the essential tremor drug are estimated at $8–10 billion.