GLOBAL RESEARCH ARCHIVE
IRSF Field Report 2: '401 Drives Progressive Hand & Gross Motor Skill Gains
Research evidence excerpt
IRSF Field Report 2: '401 Drives Progressive Hand & Gross Motor Skill Gains
Biotechnology
Neurogene
TD SECURITIES (USA) LLC QUICK TAKE: CONFERENCE TAKEAWAY
June 30, 2026
Price: $32.28 (06/29/2026) IRSF Field Report 2: '401 Drives Progressive
Price Target: NA Hand & Gross Motor Skill Gains
BUY (1)
Ritu Baral THE TD COWEN INSIGHT
646 562 1379
NGNE presented add'l sub analyses from the updated IRSF Ph1/2 '401 dataset highlighting
ritu.baral@tdsecurities.com
gains in hand & gross motor function. '401 led to clinically meaningful improvements in
Athena Chin RSHFS-assessed hand function, w/ acquisition of advanced skills appearing dependent on
646 562 1468 baseline motor ability. RSGMS (gross-motor) data also showed hierarchical, developmentally
athena.chin@tdsecurities.com
progressive gains linked to baseline function.
Key Data
Symbol NASDAQ: NGNE Today at IRSF 2026 (June 29–July 1; Prior Lake, MN), NGNE presented additional subanalyses
Market Cap $510.1MM from the updated 12+ month Ph1/2 NGN-401 dataset. Presentations focused on changes
in hand function and gross motor function following '401 treatment, as well as caregiverClick here for the full company update from
perspectives on developmental milestone gains observed to date. We highlight the keyour latest quarterly report.
findings below.
NGN-401 Showed Clinically Meaningful Improvements In Hand Function, With Emerging
Evidence Of Progressive Acquisition Of More Complex Manual Skills
In the Ph1/2 '401 Rett study, hand function was assessed using both i) developmental
milestones included in the registrational Phase 3 EMBOLDEN trial and ii) the Rett Syndrome
Hand Function Scale (RSHFS), a clinically validated tool that measures hand function in 8
levels of increasing sequential complexity.
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