Cash, cash equivalents and investments as of September 30, were $139.0M. The Company continues to expect current cash, cash equivalents and marketable securities, together with an estimated $189.5M in net proceeds from the private placement, to fund operations into the second half of 2027. “The recently announced interim data from participants dosed with 1E15 vg in the NGN-401 clinical trial showed consistent improvements across multiple domains that define Rett syndrome, in contrast to what is expected based on the natural history of the disease,” stated Rachel McMinn, CEO of Neurogene (NGNE). “We have observed girls gaining complex skills rarely ever learned in this population, as well as skills that were once present and lost during the phase of developmental regression. The favorable safety and efficacy data for NGN-401 at the 1E15 vg dose demonstrate its potential to have a meaningful impact on the disease course of Rett syndrome. We will continue to engage with the FDA on the planning for a future registrational clinical trial and anticipate providing an update on the trial design in the first half of 2025.”
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Read More on NGNE:
- Neurogene Adjusts NGN-401 Trial After Adverse Event
- Neurogene provides update on NGN-401 clinical trial for Rett syndrome
- Neurogene Reports Promising Interim Results for NGN-401 Trial
- Neurogene price target raised to $60 from $44 at Stifel
- Neurogene price target lowered to $60 from $65 at BMO Capital