Aankondiging • Nov 12

Neurogene Reports Positive Interim Efficacy Data from First Four Low-Dose Pediatric Participants in Ngn-401 Gene Therapy Clinical Trial for Rett Syndrome

Neurogene Inc. announced positive interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase 1/2 open-label trial designed to evaluate NGN-401 gene therapy for the treatment of female pediatric patients with Rett syndrome. Low-dose NGN-401 has demonstrated a favorable safety profile. Interim Clinical Data as of Data Cut-Off Date of October 17, 2024. Interim Safety Data (N=7). Low-dose (1E15 vg) and high-dose (3E15 vg) NGN-401 have been well-tolerated with a favorable safety profile in the first seven pediatric participants (N=5 low-dose; N=2 high-dose): No treatment-related serious adverse events (SAEs). No signs or symptoms indicative of MeCP2 overexpression toxicity. Most treatment-related adverse events are known potential risks of adeno-associated virus (AAV), have been responsive to steroids, and are resolved or are resolving. No intracerebroventricular (ICV)-related AEs. No seizures for any participants following NGN-401 treatment. Neurogene became aware of an emerging treatment-related SAE consistent with known risks of AAV gene therapy in the third high-dose participant who was recently dosed. Low-Dose Interim Efficacy Data (N=4). The first four participants (age range 4-7 years old, efficacy assessments at 15, 12, 9, and 3 months post-dosing) in low-dose Cohort 1 showed consistent, concordant and durable improvements across key Rett syndrome assessments: All participants achieved a rating of “much improved,” or a score of 2, on the clinician-rated Clinical Global Impression Scale of Improvement (CGI-I) from baseline; a score of < 3 is considered clinically meaningful. All participants improved in the caregiver-completed Rett Syndrome Behavior Questionnaire (RSBQ), ranging from 28% to 52% improvement from baseline. All participants acquired skills and/or developmental milestones in one or more core clinical domains of Rett syndrome - hand function/fine motor, language/communication and ambulation/gross motor. These improvements include complex skills that are rarely learned in this population and skills that are rarely relearned after developmental regression when compared to the NIH-sponsored Rett syndrome natural history. New skills and milestones have increased and deepened over time. Initiation of Adolescent/Adult Cohort in NGN-401 Clinical Trial, Neurogene announced that it has initiated an adolescent/adult Cohort 3 to gain initial data on the potential of NGN-401 to treat a broader patient population. This cohort is designed to enroll three participants ages 16 and above at the high dose. FDA Alignment on CMC Requirements to Initiate Future Registrational Trial and Support Potential Product Launch, Neurogene also announced that it has gained alignment with the FDA on its potency assay strategy for NGN-401, which is necessary to have in place prior to initiating a registrational trial. In addition, the FDA is aligned with Neurogene’s manufacturing scale-up plans for NGN-401, which is important to support a future commercial product launch. Completed and Upcoming Milestones for the NGN-401 Program, Expect to complete enrollment in the low-dose pediatric Cohort 1 (N=8) in the fourth quarter of 2024. Plans to provide an update of registrational trial design in the first half of 2025. Plans to announce additional interim Phase 1/2 clinical data in the second half of 2025. Neurogene announced that the Company does not expect to move forward with the NGN-101 CLN5 Batten disease gene therapy program at this time. Given the rarity of the disease, continued investment in the program was predicated on alignment on a streamlined registrational pathway with FDA. To support a streamlined pathway, Neurogene submitted a Regenerative Medicine Advance Therapy application to the FDA. Despite the Company’s belief that the application met the standard of preliminary clinical evidence required to obtain an RMAT designation, the RMAT application was denied. Neurogene is currently evaluating options for the program.

Aankondiging • Dec 20

Neurogene Inc. (NasdaqCM:NGNE) completed the acquisition of Neoleukin Therapeutics, Inc. (NasdaqCM:NLTX) from a group of shareholders for approximately $120 million in a reverse merger transaction.

Neurogene Inc. entered into a term sheet to acquire Neoleukin Therapeutics, Inc. (NasdaqCM:NLTX) from a group of shareholders in a reverse merger transaction on June 2, 2023. Neurogene Inc. entered into a definitive merger agreement to acquire Neoleukin Therapeutics, Inc. from a group of shareholders for approximately $180 million in a reverse merger transaction on July 17, 2023. Subject to the terms of the merger agreement, each outstanding share of Neurogene capital stock will be converted solely into the right to receive a number of shares of Neoleukin common stock, equal to the exchange ratio, estimated to be approximately 1.7374 shares of Neoleukin common stock for each share of Neurogene’s common stock. Post merger Neoleukin stockholders are expected to own approximately 16% of the combined company and pre-merger Neurogene stockholders (including those purchasing Neurogene shares in the concurrent private financing) are expected to own approximately 84% of the combined company. Upon completion of the merger the combined company is expected to operate under the name Neurogene Inc. and trade on the Nasdaq Capital Market under the ticker symbol “NGNE”. Under specified circumstances, Neoleukin may be required to pay Neurogene a termination fee of $3.04 million and Neurogene may be required to pay Neoleukin a termination fee of $12 million. The combined company will be led by Rachel McMinn, Founder and Chief Executive Officer of Neurogene, and other members of the Neurogene management team. The combined company’s Board of Directors will be comprised of five board members selected by Neurogene and two members selected by Neoleukin.The transaction is subject to approval by Neurogene and Neoleukin stockholders, the effectiveness of a registration statement, Nasdaq’s approval of the listing of the shares of Neoleukin common stock to be issued in connection with the merger, Neurogene’s receipt (or receipt simultaneous with the Closing of the merger) of cash proceeds from its private placement of not less than $75 million, Neoleukin net cash, as finally determined in accordance with the Merger Agreement, equaling an amount not less than $60 million and the satisfaction of customary closing conditions. The transaction has been unanimously approved by the Board of Directors of both Neurogene and Neoleukin. Neoleukin’s board of directors unanimously recommends that Neoleukin stockholders vote for the merger. The merger is expected to close in the fourth quarter of 2023. TD Cowen is serving as exclusive financial advisor to Neurogene. TD Cowen and Stifel are serving as placement agents on Neurogene’s planned concurrent private financing. Ryan A. Murr and Branden C. Berns of Gibson Dunn & Crutcher LLP are serving as legal counsel and legal due diligence provider to Neurogene and Cooley LLP is serving as legal counsel to the placement agents. Leerink Partners LLC acted as the exclusive financial advisor and fairness opinion provider to Neoleukin Board. David K. Michaels and Jeremy R. Delman of Fenwick & West LLP are serving as legal counsel and legal due diligence provider to Neoleukin. Equiniti Trust Company, LLC acted as transfer agent for Neoleukin and American Stock Transfer & Trust Company, LLC acted as transfer agent for Neurogene. Morrow Sodali LLC acted as proxy solicitor to Neoleukin for a fee of $20,000, plus reimbursement of out-of-pocket expenses. In connection with Leerink Partners’ services as financial advisor to Neoleukin, Neoleukin has agreed to pay Leerink Partners an aggregate fee of $2.5 million, $500,000 of which became payable upon the rendering of its opinion and the remainder of which is payable contingent upon consummation of the merger.Neurogene Inc. (NasdaqCM:NGNE) completed the acquisition of Neoleukin Therapeutics, Inc. (NasdaqCM:NLTX) from a group of shareholders for approximately $120 million in a reverse merger transaction on December 18, 2023. As a result of and upon the effective time of the Merger (as defined herein), among other things, (a) each then-issued and outstanding share of Class A Common Stock, par value $0.0001 per share, of Neurogene (“Neurogene Class A Common Stock ”) converted automatically into 0.0756 shares of common stock, par value $0.000001 per share, of Neoleukin (the “Company Common Stock” and prior to the effective time of the Merger, the “Neoleukin Common Stock”), (b) each then-issued and outstanding share of Class B Common Stock, par value $0.0001 per share, of Neurogene (“Neurogene Class B Common Stock” and, together with Neurogene Class A Common Stock, “Neurogene Common Stock”), converted automatically into 0.0756 shares of Company Common Stock, (c) each then-issued and outstanding share of Series A-1 Preferred Stock, par value $0.0001 per share, of Neurogene (“ Neurogene Series A-1 Preferred Stock ”) converted automatically into 0.0756 shares of Company Common Stock, (d) each then-issued and outstanding share of Series A-2 Preferred Stock, par value $0.0001 per share, of Neurogene (“Neurogene Series A-2 Preferred Stock”) converted automatically into 0.0756 shares of Company Common Stock, (e) each then-issued and outstanding share of Series B Preferred Stock, par value $0.0001 per share (“Neurogene Series B Preferred Stock” and together with the Neurogene Common Stock, the Neurogene Series A-1 Preferred Stock and the Neurogene Series A-2 Preferred Stock, the “Neurogene Capital Stock”) converted automatically into 0.0756 shares of Company Common Stock, and (f) each then-issued and outstanding pre-funded warrant of Neurogene, each representing a right to acquire one share of Neurogene Common Stock for $1.559999 per pre-funded warrant (each, a “Neurogene Pre-Funded Warrant”), converted automatically, on a one-for-one basis, into a pre-funded warrant of the Company (each, a “ Company Pre-Funded Warrant”) that represents a right to acquire 0.0756 shares of Company Common Stock at an exercise price of $0.000001 per share. In connection with the completion of the Merger, Neoleukin changed its name from “Neoleukin Therapeutics, Inc.” to “Neurogene Inc.” (the “Company Name Change”). As of December 19, 2023, Neurogene shares are expected to begin trading on the NASDAQ Global Market under the ticker “NGNE” beginning today at the market open. The Board has determined that Robert Baffi, Cory Freedland, Sarah Noonberg, Rohan Palekar and Robert Keith Woods (“ Keith Woods ”), each of whom is a current member of the Board, qualify as “independent directors” as defined by the Nasdaq Listing Rules. The Neoleukin board of directors previously determined that former directors Martin Babler, M. Cantey Boyd, Erin Lavelle, Todd Simpson and Rusty Williams were “independent” under the Nasdaq Listing Rules. Immediately after the effective time the Merger on December 18, 2023, the Board appointed Rachel McMinn as the Company’s Chief Executive Officer and Christine Mikail as the Company’s President and Chief Financial Officer, each to serve at the discretion of the Board. Immediately after the effective time the Merger on December 18, 2023, the Board appointed the following four individuals to the Board: Rachel McMinn, Robert Baffi, Cory Freedland and Keith Woods. Rachel McMinn was also appointed as Chair of the Board. 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. Neoleukin convened and adjourned the Special Meeting on December 13, 2023. At the Special Meeting, Neoleukin’s stockholders approved, among other matters, amendments to the amended and restated certificate of incorporation of Neoleukin to (i) increase the number of authorized shares of Company Common Stock from 20,000,000 shares (after giving effect to the 1-for-5 reverse stock split that was effected on September 25, 2023) to 500,000,000 (the “Authorized Share Increase”) and (ii) effect the Reverse Stock Split. Following the Special Meeting, the Neoleukin board of directors approved the Reverse Stock Split at a ratio of 1:4.

Sneeuwvlok Score
Waardering	4/6
Toekomstige groei	0/6
Prestaties in het verleden	0/6
Financiële gezondheid	6/6
Dividenden	0/6

Historische aandelenkoersen
Huidige aandelenkoers	US$26.87
52 Week Hoogtepunt	US$37.27
52 Week Laag	US$14.65
Bèta	2.08
1 maand verandering	-0.67%
3 maanden verandering	36.53%
1 Jaar Verandering	52.15%
3 jaar verandering	n/a
5 jaar verandering	n/a
Verandering sinds IPO	77.95%

	NGNE	US Biotechs	US Markt
7D	-0.3%	1.0%	1.3%
1Y	52.2%	34.2%	27.7%

NGNE volatility
NGNE Average Weekly Movement	11.3%
Biotechs Industry Average Movement	10.9%
Market Average Movement	7.2%
10% most volatile stocks in US Market	16.6%
10% least volatile stocks in US Market	3.0%

NGNE fundamentele statistieken
Marktkapitalisatie	US$428.23m
Inkomsten(TTM)	-US$98.64m
Inkomsten(TTM)	n/a

NGNE resultatenrekening (TTM)
Inkomsten	US$0
Kosten van inkomsten	US$82.28m
Brutowinst	-US$82.28m
Overige uitgaven	US$16.36m
Inkomsten	-US$98.64m

Winst per aandeel (EPS)	-6.24
Brutomarge	0.00%
Nettowinstmarge	0.00%
Schuld/Eigen Vermogen Verhouding	0%

Gegevens	Laatst bijgewerkt (UTC-tijd)
Bedrijfsanalyse	2026/05/27 02:10
Aandelenkoers aan het einde van de dag	2026/05/27 00:00
Inkomsten	2026/03/31
Jaarlijkse inkomsten	2025/12/31

Pakket	Gegevens	Tijdframe	Voorbeeld Amerikaanse bron ^*
Financiële gegevens bedrijf	10 jaar	Resultatenrekening Kasstroomoverzicht Balans	SEC-formulier 10-K SEC-formulier 10-Q
Consensus schattingen analisten	+3 jaar	Financiële prognoses Koersdoelen analisten	Onderzoeksrapporten van analisten Blue Matrix
Marktprijzen	30 jaar	Aandelenprijzen Dividenden, splitsingen en acties	ICE Marktgegevens SEC-formulier S-1
Eigendom	10 jaar	Top aandeelhouders Handel met voorkennis	SEC-formulier 4 SEC-formulier 13D
Beheer	10 jaar	Leiderschapsteam Raad van bestuur	SEC-formulier 10-K SEC-formulier DEF 14A
Belangrijkste ontwikkelingen	10 jaar	Bedrijfsaankondigingen	SEC-formulier 8-K

Analist	Instelling
Whitney Ijem	Canaccord Genuity
Gum-Ming Lowe	Craig-Hallum Capital Group LLC
Debjit Chattopadhyay	Guggenheim Securities, LLC

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No independent directors

Neurogene Inc. (NasdaqCM:NGNE) completed the acquisition of Neoleukin Therapeutics, Inc. (NasdaqCM:NLTX) from a group of shareholders for approximately $120 million in a reverse merger transaction.

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