Praxis Precision Medicines Inc. (PRAX): Next potential mid-cap growth story in biotech, led by >$10B market opportunity in essential tremor; Initiate at Buy, $447 PT

Research evidence excerpt

Praxis Precision Medicines Inc. (PRAX): Next potential mid-cap growth story in biotech, led by >$10B market opportunity in essential tremor; Initiate at Buy, $447 PT

Goldman Sachs Praxis Precision Medicines Inc. (PRAX)

Exhibit 1: Summary of key pipeline

Development Stage Catalyst

Peak

Program Trial PoS Indication PC IND Ph1 Ph2 Ph3 Approved Event Timing

Sales ($M)

January

Ulixacaltamide NDA $4,780 90% Essential tremor PDUFA

29th, 2027

December

NDA $470 90% SCN2A/SCN8A DEE PDUFA

27th, 2026

Relutrigine

Ph3 $2,580 50% Broader DEEs Topline data 4Q26

Elsunersen Ph3 $530 65% SCN2A DEE Topline data 2027

Vormatrigine - NM Focal epilepsy

Total Opportunity $8,400

Source: Company data, Goldman Sachs Global Investment Research

Company overview

Praxis Precision Medicines, Inc. (PRAX) is a biopharmaceutical company leveraging the

genetics of epilepsy in pursuit of CNS (central nervous system) drug discovery and

development. PRAX has two drug discovery platforms - Cerebrum and Solidus - aimed

at discovering small molecules (Cerebrum) and antisense oligonucleotides or ASOs

(Solidus), which have produced 4 clinical candidates to-date (3 small molecules and 1

ASO). The company has recently transitioned toward commercial readiness, with two

PDUFA dates in the next several months.

Clinical pipeline assets

n Ulixacaltamide in essential tremor. PRAX’s lead program is ulixacaltamide, a

selective T-type calcium channel inhibitor, in essential tremor (ET), with

breakthrough designation (BTD) and a PDUFA date of January 29, 2027. The NDA

filing was based on Ph3 ESSENTIAL3 data, which was toplined in October 2025 and

presented at the American Academy of Neurology (AAN) meeting in April 2026. The

data showed a statistically significant 4.3 point improvement in the modified