NEW YORK / Mar 18, 2024 / 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 fourth quarter and full year 2023 financial results and highlighted recent corporate updates.
“We started the year with strong execution in our Phase 1/2 NGN-401 gene therapy trial for female pediatric patients with Rett syndrome, meeting our goal to expand the trial and add a high-dose cohort. We believe this expanded dataset will support future regulatory discussions to align on the design of a potential registrational study,” said Rachel McMinn, Ph.D., Chief Executive Officer and Founder of Neurogene. “NGN-401 has the potential to be a best-in-class treatment option with a targeted route of administration to deliver full-length, functional copies of the MECP2 gene. We are pleased that NGN-401 has been well-tolerated in all three patients dosed, with no signs of overexpression-related toxicity, demonstrating the potential of our EXACT transgene regulation technology to constrain MECP2 levels within a narrow and therapeutically relevant range. As we look ahead, we remain on track for interim clinical data from the NGN-401 trial in the fourth quarter of this year.”
Continued Dr. McMinn, “Our decision to execute a reverse merger, along with the concurrent private financing, provides us with cash runway into the second half of 2026, beyond key inflection points in our NGN-401 program and our NGN-101 program for CLN5 Batten disease. We look forward to sharing data from both programs later this year and continuing to advance our pipeline to enable an additional development candidate using EXACT in 2025.”
Fourth Quarter 2023 and Recent Highlights, and Anticipated Milestones
Phase 1/2 Trial of NGN-401 Gene Therapy for Treatment of Rett Syndrome
Phase 1/2 Trial of NGN-101 Gene Therapy for Treatment of CLN5 Batten Disease
Additional Corporate Updates
Fourth Quarter and Full Year 2023 Financial Results
Upcoming Events
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 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 www.neurogene.com.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability profile of NGN-401; trial designs, clinical development plans and timing for NGN-401, including anticipated timing of enrollment in and clinical trial results from the Company’s NGN-401 Phase 1/2 trial for Rett syndrome; effects of the removal of staggered dosing in Cohort 1; initiation of new clinical sites for NGN-401 in the UK; expected interactions with the FDA regarding NGN-101; nomination of additional preclinical product candidates; and our expected cash resources and liquidity. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “on track,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: risks related to the timing and success of enrolling patients in the expanded cohort of our Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the expected timing and results of dosing of patients in our clinical trials, including NGN-401; the potential that we may not be able to expand our Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome into the UK based on a variety of factors, including but not limited to any decisions of regulatory authorities, costs of expanding the trial in the UK, the availability of suitable clinical test sites, and the ability to enroll patients in the UK or other reasons; the potential for negative impacts to patients resulting from using a higher dose of NGN-401 in Cohort 2 of the Phase 1/2 clinical trial for the treatment of Rett syndrome; the risk that we may not be able to report our data on the predicted timeline; our limited operating history; the risk that we may not be able to raise adequate additional capital to finance our operations, complete our clinical trials and commercialize our products; risks related to our ability to obtain regulatory approval for, and ultimately commercialize, our product candidates, including NGN-401; risks related to the outcome of non-clinical testing and early clinical trials for our product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; risks related to our limited experience in designing clinical trials and lack of experience in conducting clinical trials; and other risks and uncertainties identified under the heading "Risk Factors" included in our Annual Report on Form 10-K for the year ended December 31, 2023, filed with the Securities and Exchange Commission (“SEC”) on March 18, 2024, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication.
- Financial Tables Follow -
Neurogene Inc. | |||||
| December 31, |
| December 31, | ||
Assets |
|
|
| ||
Cash and cash equivalents | $ | 148,210 |
| $ | 82,021 |
Other current assets |
| 52,138 |
|
| 2,698 |
Non-current assets |
| 22,225 |
|
| 24,546 |
Total assets | $ | 222,573 |
| $ | 109,265 |
Liabilities |
|
|
| ||
Current liabilities | $ | 22,973 |
| $ | 6,651 |
Non-current liabilities |
| 13,576 |
|
| 3,987 |
Total liabilities |
| 36,549 |
|
| 10,638 |
Total convertible preferred stock |
| — |
|
| 244,366 |
Stockholders' equity (deficit) |
| 186,024 |
|
| (145,739) |
Total liabilities, convertible preferred stock and stockholders' equity (deficit) | $ | 222,573 |
| $ | 109,265 |
Neurogene Inc. | |||||
| Year Ended December 31, | ||||
| 2023 |
| 2022 | ||
Operating expenses: |
|
|
| ||
Research and development | $ | 44,394 |
| $ | 47,505 |
General and administrative |
| 11,189 |
|
| 9,012 |
Total operating expenses |
| 55,583 |
|
| 56,517 |
Loss from operations |
| (55,583) |
|
| (56,517) |
Other income, net |
| 2,911 |
|
| 1,328 |
Bargain purchase gain |
| 16,355 |
|
| — |
Net loss | $ | (36,317) |
| $ | (55,189) |
| Pre-Merger |
| Post- |
| Pre-Merger | |||||||||
Per share information (1): | (a) |
| (b) |
| (c) |
| (a) |
| (b) | |||||
Net income (loss) per share, basic | $ | (117.28) |
| $ | — |
| $ | 27.76 |
| $ | (139.88) |
| $ | — |
Weighted-average shares outstanding used in computing net income (loss) per share, basic |
| 426,097 |
|
| — |
|
| 491,867 |
|
| 394,533 |
|
| — |
Net income (loss) per share, diluted | $ | (117.28) |
| $ | — |
| $ | 2.93 |
| $ | (139.88) |
| $ | — |
Weighted-average shares outstanding used in computing net income (loss) per share, diluted |
| 426,097 |
|
| — |
|
| 4,656,947 |
|
| 394,533 |
|
| — |
(1) | On December 18, 2023, the Company completed its reverse merger, which among other things, resulted in Neurogene OpCo merging with and into a wholly owned subsidiary of Neoleukin Therapeutics, Inc. As the earnings per share information for the pre-merger period is not comparable to the earnings per share information for the post-merger period, the earnings per share information is being presented separately for these periods. See Note 3, Net Income (Loss) Per share, for additional information. | |||
(a) | Presents information for the pre-merger period for Class A common stock. The pre-merger period is January 1, 2023 through December 17, 2023 for the year ended December 31, 2023 and the full fiscal year ended December 31, 2022. | |||
| (b) | Presents information for the pre-merger period for Class B common stock. The pre-merger period is January 1, 2023 through December 17, 2023 for the year ended December 31, 2023 and the full fiscal year ended December 31, 2022. | ||
| (c) | Presents information for the post-merger period for common stock. The post-merger period is December 18, 2023 through December 31, 2023. |
Last Trade: | US$22.50 |
Daily Change: | -0.14 -0.62 |
Daily Volume: | 184,159 |
Market Cap: | US$334.120M |
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