Praxis Precision Medicines (PRAX) Goldman Sachs 47th Annual Global Healthcare Conference 2026 summary

Market opportunity and strategy

• Essential tremor affects about 7 million in the U.S., with an initial addressable market of 2 million patients currently sub-optimally treated or untreated.

• Ulixacaltamide targets a large unmet need, with potential peak revenue estimated at $10 billion, considered conservative based on market size and drug efficacy.

• Pricing is expected between $50,000 and $100,000 annually, guided by payer feedback and analogs in similar movement disorder markets.

• Commercial launch readiness is targeted for early Q4, with a planned sales force of 300 to cover 13,000-15,000 accounts.

• Step-through requirements for propranolol are anticipated, but about half of patients are contraindicated, and payer restrictions may be less stringent than expected.

Clinical development and efficacy

• Ulixacaltamide demonstrated restoration of daily functions in essential tremor patients, with some regaining over 12 functions.

• The drug is under NDA review, with a PDUFA date in late January; breakthrough designation was granted after positive efficacy and safety results.

• Tolerability is generally good, with dizziness as the main side effect, mitigated by slower titration; no major organ safety concerns observed.

• Long-term persistence on therapy is high, with few discontinuations due to lack of efficacy.

• Label discussions with the FDA focus on titration and proper use, with no expected contraindications or REMS requirements.

Commercial execution and pipeline

• Launch strategy emphasizes upfront investment, broad physician education, and no patient left behind.

• Relutrigine is expected to launch in September for genetic DEE, targeting 5,000-10,000 U.S. patients and expanding with the EMERALD study.

• Relutrigine pricing may start between $450,000-$650,000 per patient annually, with flexibility for future adjustments.

• Vormatrigine's POWER1 study showed mixed results; future development will focus on patient subgroups with better response.

• The company is preparing for multiple launches over the next three quarters, leveraging a strong balance sheet.