Neurogene Merge with Neoleukin Therapeutics

NEW YORK–(BUSINESS WIRE)–Neurogene Inc. (NASDAQ: NGNE), 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, Inc. 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 $200 million, 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.

Transaction Details

To ensure the combined company’s compliance with the minimum bid price requirement of $4.00 per share for initial listing on The Nasdaq Global Market, Neoleukin implemented a reverse split of its common stock at a ratio of 1-for-4 shares. In the reverse stock split, every four shares of Neoleukin common stock outstanding were combined and reclassified into one share of Neoleukin common stock. Immediately thereafter, and pursuant to the terms of the previously announced merger agreement, Neurogene became a wholly owned subsidiary of Neoleukin upon completion of the merger, and each outstanding share of Neurogene common stock was converted into 0.0756 shares of Neoleukin common stock. Following the closing of the merger, there are approximately 16,887,060 shares of the combined company’s common stock outstanding (assuming the exercise in full of all pre-funded warrants), with prior Neurogene stockholders, including investors in the private placement, owning approximately 84% and prior Neoleukin stockholders owning approximately 16% of the combined company’s outstanding securities. The combined company will be led by Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene, and other members of the Neurogene management team.

TD Cowen served as exclusive financial advisor to Neurogene. TD Cowen and Stifel served as placement agents for Neurogene’s concurrent private financing. Gibson Dunn & Crutcher LLP served as legal counsel to Neurogene and Cooley LLP served as legal counsel to the placement agents. Leerink Partners served as the exclusive financial advisor to Neoleukin. Fenwick & West LLP served as legal counsel to Neoleukin.

About Neurogene

The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and by designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit