Neurogene focused on advancing Phase 1/2 trial for NGN-401, a differentiated clinical stage gene therapy to treat Rett syndrome using its EXACT technology; interim clinical data expected in 4Q24
Neurogene to merge with Neoleukin Therapeutics. (Credit: Rock Staar on Unsplash)
Neurogene , a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the closing of its merger with Neoleukin Therapeutics.
Neurogene shares are expected to begin trading on the NASDAQ Global Market under the ticker “NGNE” beginning today at the market open.
Concurrent with the closing of the merger, Neurogene closed an oversubscribed $95 million private financing, led by new and existing healthcare-dedicated specialist and mutual fund institutional investors, including participation from Great Point Partners, EcoR1 Capital, Redmile Group, Samsara BioCapital, Janus Henderson Investors, funds and accounts managed by Blackrock, Casdin Capital, Avidity Partners, Arrowmark Partners, Cormorant Asset Management, Alexandria Venture Investments, and a healthcare investment fund.
Neurogene’s cash, cash equivalents, and investments of approximately $200m, before payment of final transaction-related expenses, are expected to fund operations and multiple potentially value-creating milestones into the second half of 2026.
“This transformative transaction provides us with a strong cash position allowing us to demonstrate the best-in-class potential of our EXACT transgene regulation technology in treating Rett syndrome, a debilitating and complex neurological disease that cannot be treated with conventional gene therapy,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “We look forward to expanding our ongoing Phase 1/2 clinical trial in 2024 for pediatric patients with Rett syndrome beyond the first cohort of five patients, and presenting interim clinical data from this study in the fourth quarter of 2024, with additional data from an expanded number of patients expected in the second half of 2025.”
NGN-401 is an investigational adeno-associated virus (AAV9) gene therapy candidate for Rett syndrome purposefully designed and administered to maximize therapeutic activity while averting transgene overexpression toxicities. NGN-401 delivers the full-length human methyl cytosine binding protein 2 (MECP2) gene, providing an optimal gene replacement approach.
NGN-401 leverages Neurogene’s novel and proprietary EXACT transgene regulation technology, which provides a highly controlled and consistent MeCP2 expression on a cell by cell basis, and avoids overexpression-related toxicities associated with conventional gene therapy.
Neurogene recently announced the dosing of the first two patients with NGN-401 in the third and fourth quarters of 2023. Data from the ongoing Phase 1/2 clinical trial demonstrate that NGN-401 has been well tolerated, with no treatment-emergent serious adverse events or procedure-related events, and no signs of treatment-related overexpression toxicity.
NGN-401 has been granted Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation by the U.S. Food and Drug Administration (FDA).
Neurogene is also developing NGN-101 for the treatment of CLN5 Batten disease, with interim clinical data for NGN-101 expected in the second half of 2024, and is advancing multiple discovery-stage candidates leveraging its EXACT transgene regulation technology. Neurogene expects to initiate a clinical study of one product candidate from its discovery-stage portfolio in 2025.
Source: Company Press Release