普通外文研报

Impressive Ph1 RESTORE-FA Data Supports Pivotal & Validates GeneTAC Platform

发布日期: 2026-05-19研究机构: Jefferies公司 / 股票: DSGN.OQ报告页数: 12原文语言: 英语证据页码: 1

研报英文原文证据摘录

USA | Biotechnology

Design Therapeutics EquityMayResearch19, 2026

TARGET | ESTIMATE CHANGEImpressive Ph1 RESTORE-FA Data Supports

RATING BUYPivotal & Validates GeneTAC Platform

PRICE $14.37^

RESTORE-FA 4-wk data came ahead of 2H26 timelines and looks unequivocally

positive in our view. Key q into this update was whether DT-216P2 could PRICE TARGET | % TO PT $21.00 ($15.00) | +46%

produce durable FXN expression. In this update we got that and more, with the 52W HIGH-LOW $17.25 - $3.31

1mg/kg (n=4) showing improvement in key clinical endpoints mFARS / USS. FLOAT (%) | ADV MM (USD) 59.3% | 7.62

Level of benefit unprecedented in FA per our analysis. Focus now shifts to MARKET CAP $898.1M

pivotal trial planning, which we expect to hear more on later this yr. TICKER DSGN ^Prior trading day's closing price unless otherwise

noted.

For background see initiation: DSGN's platform is 'GeneTAC' molecules targeting gene expression

in monogenic disease. DT-216P2 is DSGN's 2nd attempt in Friedreich's ataxia w/ improved

FY (Dec) CHANGE TO JEFe JEF vs CONS

formulation after the first (P1) had insufficient PK.

2026 2027 2026 2027

Key focus is whether DT-216P2's greater exposure leads to durable FXN + clinical benefit + path REV NA NA NA NA

to registration EPS NA -4% NA NA

In our view, the Ph1 RESTORE-FA 4wk data are a best-case outcome... albeit early/limited, the level 2026 ($) Q1A Q2 Q3 Q4 FY

of clinical benefit + correlation to FXN expression are impressive, positioning DT-216 as a potential EPS (0.29) (0.32) (0.35) (0.39) (1.34)

game-changer for FA and validating the platform PREV

Investor q's are mostly "why is this not positively received?" w/ thinking it could be related to:

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