Goldman Sachs: Praxis Precision Medicines (PRAX.US) — Next Potential Mid-Cap Biotech Growth Story; Initiate at Buy, $447 PT
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Key Views
Ulixacaltamide targets a substantial ET market—approximately 7 million U.S. prevalent cases, yet only about 1 million receiving treatment with no new therapies approved in over 50 years. In the Phase 3 ESSENTIAL3 trial, it met the primary endpoint with an mADL11 improvement of 4.3 versus placebo at 1.7 (p<0.001; n=432). Goldman models approximately $5 billion in unadjusted 2035 U.S. sales (90% probability of success), potentially conservative within the estimated $10 billion-plus ET market. Launch trajectory analysis suggests consensus estimates have upside of +5%/+10%/+9% for 2027/2028/2029.
Relutrigine showed seizure reductions of 46–53% in EMBOLD studies. The broader EMERALD readout in 4Q26 could expand the treatable population from roughly 5,000 to over 150,000, with an estimated $2.6 billion unadjusted peak sales (50% success probability).
Outlook
Key catalysts include relutrigine's PDUFA date of December 27, 2026, EMERALD data in 4Q26, and ulixacaltamide's PDUFA date of January 29, 2027. Discontinuation rates (~30% in Phase 3) represent a risk, though titration modifications may mitigate this.
Recommended Securities
Praxis Precision Medicines (PRAX.US) — Buy, 12-month PT $447.
Key exhibits
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