Neurogene (NGNE) investor relations material

Executive summary

• NGN-401 gene therapy for Rett syndrome is the primary focus, with the Embolden registrational trial nearing full dosing—approximately 90% of participants dosed and completion expected in Q2 2026; positive interim data for the pediatric cohort reported as of October 2025.

• NGN-401 at the 1E15 vg dose has been generally well-tolerated, with no HLH cases reported as of May 11, 2026.

• Received Breakthrough Therapy, RMAT, PRIME, Fast Track, Orphan Drug, and Rare Pediatric Disease designations for NGN-401, expediting regulatory pathways.

• Discontinued NGN-101 for CLN5 Batten disease after FDA denied RMAT designation; evaluating options for the program.

• Strong cash position projected to fund operations through at least Q1 2028.

Financial highlights

• Net loss of $30.9 million for Q1 2026, up from $22.6 million in Q1 2025, driven by increased R&D expenses.

• Research and development expenses rose to $25.2 million (from $17.8 million), mainly due to higher clinical trial and manufacturing costs for NGN-401.

• General and administrative expenses were $8.2 million for Q1 2026, flat year-over-year, with higher pre-commercial and professional fees offset by lower stock-based compensation.

• Cash, cash equivalents, and short-term investments totaled $243.2 million as of March 31, 2026.

• Accumulated deficit reached $383.6 million.

Outlook and guidance

• Current cash resources expected to fund operations through at least Q1 2028.

• Completion of dosing in the Embolden trial expected in Q2 2026.

• Updated interim safety and efficacy data from Phase 1/2 trial, including 12-month follow-up, anticipated mid-2026.

• Plans to initiate commercial manufacturing scale-up and Process Performance Qualification campaign for NGN-401 in mid-2026.

• Anticipates continued increase in R&D and G&A expenses as clinical programs advance and pipeline expands.