Clene Provides Regulatory Update Following Constructive FDA Type-C Meeting on Neurofilament Biomarker Analysis Plan and Confirms Two Additional FDA Meetings
Clene (NASDAQ: CLNN) announced significant regulatory progress following a constructive FDA Type-C meeting regarding their neurofilament biomarker analysis plan. The company received supportive feedback on its statistical analysis plan (SAP) for comparing neurofilament light (NfL) biomarker data from its NIH-sponsored Expanded Access Protocol, which involves nearly 200 ALS patients treated with CNM-Au8®.
The company has scheduled two additional FDA meetings for Q3 2025: a Type C meeting to review long-term survival benefit data from CNM-Au8 treatment in ALS patients, and an End-of-Phase 2 Type B meeting to discuss MS program developments, particularly the Phase 2 VISIONARY-MS trial results and planned Phase 3 study.
Clene plans to conduct NfL analyses in early Q4 2025, with primary analysis at 9 months and supportive analysis at 6 months of treatment. If results show clinically meaningful NfL decline, the company aims to submit a new drug application (NDA) for ALS under the accelerated approval pathway by the end of 2025.
Clene (NASDAQ: CLNN) ha annunciato importanti progressi normativi dopo un costruttivo incontro di tipo C con la FDA riguardante il loro piano di analisi del biomarcatore neurofilamentare. L'azienda ha ricevuto feedback positivi sul piano di analisi statistica (SAP) per il confronto dei dati del biomarcatore neurofilamento light (NfL) provenienti dal Protocollo di Accesso Esteso sponsorizzato dal NIH, che coinvolge quasi 200 pazienti con SLA trattati con CNM-Au8®.
La società ha programmato due ulteriori incontri con la FDA per il terzo trimestre del 2025: un incontro di tipo C per esaminare i dati sul beneficio di sopravvivenza a lungo termine del trattamento con CNM-Au8 nei pazienti con SLA, e un incontro di tipo B di fine fase 2 per discutere gli sviluppi del programma sulla sclerosi multipla, in particolare i risultati dello studio di fase 2 VISIONARY-MS e lo studio di fase 3 pianificato.
Clene prevede di effettuare le analisi NfL all'inizio del quarto trimestre 2025, con un'analisi primaria a 9 mesi e un'analisi di supporto a 6 mesi di trattamento. Se i risultati dimostreranno un calo clinicamente significativo di NfL, l'azienda punta a presentare una nuova domanda di autorizzazione al farmaco (NDA) per la SLA tramite la procedura di approvazione accelerata entro la fine del 2025.
Clene (NASDAQ: CLNN) anunció un avance regulatorio significativo tras una reunión constructiva de tipo C con la FDA sobre su plan de análisis del biomarcador neurofilamento. La compañía recibió comentarios favorables sobre su plan de análisis estadístico (SAP) para comparar datos del biomarcador neurofilamento ligero (NfL) provenientes de su Protocolo de Acceso Ampliado patrocinado por el NIH, que incluye a casi 200 pacientes con ELA tratados con CNM-Au8®.
La empresa ha programado dos reuniones adicionales con la FDA para el tercer trimestre de 2025: una reunión de tipo C para revisar los datos de beneficio de supervivencia a largo plazo del tratamiento con CNM-Au8 en pacientes con ELA, y una reunión de tipo B de fin de fase 2 para discutir los avances del programa de esclerosis múltiple, en particular los resultados del ensayo de fase 2 VISIONARY-MS y el estudio de fase 3 planificado.
Clene planea realizar análisis de NfL a principios del cuarto trimestre de 2025, con un análisis principal a los 9 meses y un análisis complementario a los 6 meses de tratamiento. Si los resultados muestran una disminución clínicamente significativa de NfL, la compañía tiene como objetivo presentar una nueva solicitud de medicamento (NDA) para ELA bajo la vía de aprobación acelerada antes de finales de 2025.
Clene (NASDAQ: CLNN)는 신경섬유질 바이오마커 분석 계획과 관련하여 건설적인 FDA 유형 C 미팅 후 중요한 규제 진전을 발표했습니다. 이 회사는 NIH 후원 확장 접근 프로토콜에서 수집된 신경섬유질 라이트(NfL) 바이오마커 데이터를 비교하는 통계 분석 계획(SAP)에 대해 긍정적인 피드백을 받았으며, 이 프로토콜에는 CNM-Au8®로 치료받은 거의 200명의 ALS 환자가 포함되어 있습니다.
회사는 2025년 3분기에 두 차례의 추가 FDA 미팅을 예정하고 있습니다: ALS 환자에 대한 CNM-Au8 치료의 장기 생존 혜택 데이터를 검토하는 유형 C 미팅과, MS 프로그램 개발 사항을 논의하는 2상 종료 유형 B 미팅으로, 특히 2상 VISIONARY-MS 시험 결과 및 계획된 3상 연구에 관한 내용입니다.
Clene는 2025년 4분기 초에 NfL 분석을 실시할 계획이며, 9개월 시점에 주요 분석을, 6개월 시점에 보조 분석을 진행할 예정입니다. 결과가 임상적으로 의미 있는 NfL 감소를 보여주면, 회사는 2025년 말까지 가속 승인 절차를 통해 ALS에 대한 신약 신청서(NDA)를 제출하는 것을 목표로 하고 있습니다.
Clene (NASDAQ : CLNN) a annoncé des progrès réglementaires significatifs à la suite d’une réunion constructive de type C avec la FDA concernant leur plan d’analyse du biomarqueur neurofilament. La société a reçu des retours favorables sur son plan d’analyse statistique (SAP) pour comparer les données du biomarqueur neurofilament light (NfL) issues de son protocole d’accès élargi parrainé par le NIH, impliquant près de 200 patients atteints de SLA traités avec CNM-Au8®.
La société a prévu deux réunions supplémentaires avec la FDA pour le troisième trimestre 2025 : une réunion de type C pour examiner les données sur le bénéfice de survie à long terme du traitement CNM-Au8 chez les patients atteints de SLA, et une réunion de type B de fin de phase 2 pour discuter des développements du programme SEP, notamment les résultats de l’essai de phase 2 VISIONARY-MS et l’étude de phase 3 prévue.
Clene prévoit de réaliser les analyses NfL début du quatrième trimestre 2025, avec une analyse principale à 9 mois et une analyse complémentaire à 6 mois de traitement. Si les résultats montrent une baisse cliniquement significative du NfL, la société vise à soumettre une nouvelle demande d’autorisation de mise sur le marché (NDA) pour la SLA via la voie d’approbation accélérée d’ici la fin 2025.
Clene (NASDAQ: CLNN) gab bedeutende regulatorische Fortschritte nach einem konstruktiven FDA Type-C-Meeting bezüglich ihres Analyseplans für neurofilament Biomarker bekannt. Das Unternehmen erhielt unterstützendes Feedback zu seinem statistischen Analyseplan (SAP) zum Vergleich von Neurofilament Light (NfL) Biomarkerdaten aus seinem vom NIH geförderten Expanded Access Protocol, das nahezu 200 ALS-Patienten umfasst, die mit CNM-Au8® behandelt wurden.
Das Unternehmen hat zwei weitere FDA-Meetings für das dritte Quartal 2025 geplant: ein Type-C-Meeting zur Überprüfung von Langzeitüberlebensdaten der CNM-Au8-Behandlung bei ALS-Patienten und ein End-of-Phase-2 Type-B-Meeting zur Diskussion der Entwicklungen im MS-Programm, insbesondere der Phase-2 VISIONARY-MS-Studie und der geplanten Phase-3-Studie.
Clene plant, die NfL-Analysen Anfang des vierten Quartals 2025 durchzuführen, mit einer primären Analyse nach 9 Monaten und einer unterstützenden Analyse nach 6 Monaten Behandlung. Wenn die Ergebnisse einen klinisch bedeutsamen Rückgang der NfL zeigen, strebt das Unternehmen an, bis Ende 2025 einen neuen Zulassungsantrag (NDA) für ALS im beschleunigten Zulassungsverfahren einzureichen.
- FDA provided constructive feedback on neurofilament biomarker analysis plan
- Large patient pool of nearly 200 ALS patients in the Expanded Access Protocol
- Two additional FDA meetings secured for Q3 2025
- Potential NDA submission under accelerated approval pathway by end of 2025
- Multiple development programs advancing simultaneously in both ALS and MS
- NDA submission depends on demonstrating clinically meaningful decline in NfL biomarkers
- Final FDA acceptance of Statistical Analysis Plan still pending
Insights
Clene receives positive FDA feedback on ALS biomarker analysis plan, setting stage for potential accelerated approval submission in Q4 2025.
Clene has made significant regulatory progress with the FDA regarding its CNM-Au8® therapy for amyotrophic lateral sclerosis (ALS). The FDA provided constructive feedback on Clene's statistical analysis plan for neurofilament light (NfL) biomarker data from its NIH-sponsored Expanded Access Program. This biomarker is critical as decreased NfL levels could indicate reduced neuronal damage in ALS patients.
The company has already resubmitted its revised statistical analysis plan incorporating the FDA's requested changes, with formal acceptance expected this summer. The NfL analyses will be conducted early in Q4 2025, examining changes after both 9 months (primary) and 6 months (supportive) of treatment.
Importantly, the FDA has scheduled two additional meetings with Clene for Q3 2025: a Type C meeting to review long-term survival benefit data and an End-of-Phase 2 Type B meeting for its multiple sclerosis program. These meetings represent crucial milestones in Clene's regulatory strategy.
If the NfL findings demonstrate clinically meaningful improvement, Clene plans to submit a new drug application (NDA) under the FDA's accelerated approval pathway by the end of 2025. This pathway could significantly expedite CNM-Au8's time to market, allowing earlier access for ALS patients while Clene continues to gather confirmatory evidence of clinical benefit.
Clene advances toward potential accelerated approval filing with positive FDA interactions on ALS therapy and expanding MS program.
Clene's regulatory update reveals multiple catalysts that significantly de-risk its CNM-Au8® development program. The FDA's constructive feedback on the neurofilament light (NfL) biomarker analysis plan is particularly important, as NfL has emerged as a key pharmacodynamic marker for neurodegeneration. The biomarker's acceptance could provide Clene with a faster route to market through the accelerated approval pathway.
The company's strategy of leveraging data from its NIH-sponsored Expanded Access Program (supporting nearly 200 ALS patients) is methodologically sound. By comparing treated patients to matched controls, Clene can generate supportive evidence while providing compassionate access to its therapy.
The upcoming Q3 FDA meetings represent critical inflection points. The survival benefit meeting could provide the strongest possible efficacy signal for CNM-Au8, as survival is the ultimate endpoint in ALS trials. Meanwhile, the End-of-Phase 2 meeting for multiple sclerosis indicates Clene is diversifying its pipeline beyond ALS, focusing on cognitive improvement as an adjunct to standard MS therapies - addressing a significant unmet need.
With potential NDA submission by year-end 2025, Clene has established a clear regulatory timeline. If successful, CNM-Au8 could become one of the few disease-modifying treatments in a therapeutic area with limited options and substantial market potential.
- FDA provides supportive feedback on proposed statistical analysis plan for neurofilament biomarker analysis of Clene’s NIH-sponsored Expanded Access Program
- NfL EAP biomarker analyses to be conducted early in the 4th Quarter of 2025
- Clene confirms two additional FDA meetings scheduled for 3rd Quarter of 2025, to discuss ALS survival data and MS clinical development program
- Submission of new drug application for ALS under the accelerated approval pathway on track for potential submission in the 4th Quarter of 2025
SALT LAKE CITY, June 30, 2025 (GLOBE NEWSWIRE) -- Clene, Inc. (Nasdaq: CLNN) and its subsidiary, Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company dedicated to advancing therapies for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), today provided a regulatory update following a Type C meeting with the U.S. Food and Drug Administration (FDA), and announced two additional meetings scheduled with the FDA in the 3rd quarter of 2025.
In the recently concluded Type C meeting, Clene discussed its proposed statistical analysis plan (SAP) for comparing neurofilament light (NfL) biomarker data from its ongoing NIH-sponsored Expanded Access Protocol (EAP), supporting nearly 200 people living with ALS treated with compassionate use of CNM-Au8®, to matched ALS controls. The FDA provided constructive feedback on Clene’s proposed analysis methodology for assessing NfL change. NfL change will be analyzed following 9 months of treatment (primary NfL analysis) and after 6 months of treatment (supportive NfL analysis). These analyses are planned to provide supportive data of the NfL change demonstrated in the HEALEY ALS Platform Trial double-blind period following 6 months of treatment with CNM-Au8.
The Agency’s acceptance of the SAP, expected this summer, will establish an agreed upon framework for analyses of NfL change in EAP participants. Clene has already resubmitted its revised SAP incorporating FDA’s requested revisions. The NfL analyses will be conducted early in the 4th quarter of 2025. If the findings demonstrate a clinically meaningful decline in NfL, they may support a new drug application (NDA) submission under the accelerated approval pathway, planned for the end of 2025.
The FDA has also confirmed two additional meetings with Clene scheduled for the 3rd quarter of 2025:
- ALS Survival Data Type C Meeting: This meeting will review the long-term survival benefit from CNM-Au8 30 mg treatment compared to concurrently randomized controls from another Healey ALS Platform Trial Regimen, assessing whether these data support filing of an NDA under an accelerated approval pathway.
- End-of-Phase 2 Type B MS Program Meeting: This meeting will review results from the Phase 2 VISIONARY-MS trial and discuss the planned Phase 3 study focusing on cognition improvement as an adjunct to standard-of-care MS therapies, addressing a critical unmet medical need for people struggling with MS.
“We are encouraged by the FDA’s collaborative approach and their constructive feedback on our NfL biomarker analysis plan from the ongoing NIH-sponsored EAP program,” said Benjamin Greenberg, MD, Head of Medical at Clene. “With two additional FDA meetings scheduled to discuss long-term ALS survival results and the End-of-Phase 2 MS results, we are advancing our ALS and MS programs to deliver an innovative therapy for people living with neurodegenerative diseases.”
About Clene
Clene Inc., (Nasdaq: CLNN) (along with its subsidiaries, “Clene” and its wholly owned subsidiary Clene Nanomedicine, Inc.), is a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases, including amyotrophic lateral sclerosis, Parkinson’s disease, and multiple sclerosis. CNM-Au8® is an investigational first-in-class therapy that improves central nervous system cells’ survival and function via a mechanism that targets mitochondrial function and the NAD pathway while reducing oxidative stress. CNM-Au8® is a federally registered trademark of Clene Nanomedicine, Inc. The company is based in Salt Lake City, Utah, with R&D and manufacturing operations in Maryland. For more information, please visit www.clene.com or follow us on X (formerly Twitter) and LinkedIn.
About CNM-Au8®
CNM-Au8 is an oral suspension of gold nanocrystals developed to restore neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8® is a federally registered trademark of Clene Nanomedicine, Inc.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended, which are intended to be covered by the “safe harbor” provisions created by those laws. Clene’s forward-looking statements include, but are not limited to, statements regarding our or our management team’s expectations, hopes, beliefs, intentions or strategies regarding our future operations. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate,” “believe,” “contemplate,” “continue,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “will,” “would,” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements represent our views as of the date of this press release and involve a number of judgments, risks and uncertainties. We anticipate that subsequent events and developments will cause our views to change. We undertake no obligation to update forward-looking statements to reflect events or circumstances after the date they were made, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws. Accordingly, forward-looking statements should not be relied upon as representing our views as of any subsequent date. As a result of a number of known and unknown risks and uncertainties, our actual results or performance may be materially different from those expressed or implied by these forward-looking statements. Some factors that could cause actual results to differ include include general market conditions, whether clinical trials demonstrate the efficacy and safety of our drug candidates to the satisfaction of regulatory authorities, or do not otherwise produce positive results which may cause us to incur additional costs or experience delays in completing, or ultimately be unable to complete the development and commercialization of our drug candidates; the clinical results for our drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; our ability to achieve commercial success for our drug candidates, if approved; our limited operating history and our ability to obtain additional funding for operations and to complete the development and commercialization of our drug candidates; and other risks and uncertainties set forth in “Risk Factors” in our most recent Annual Report on Form 10-K and any subsequent Quarterly Reports on Form 10-Q. In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this press release, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain and you are cautioned not to rely unduly upon these statements. All information in this press release is as of the date of this press release. The information contained in any website referenced herein is not, and shall not be deemed to be, part of or incorporated into this press release.
| Media Contact Ignacio Guerrero-Ros, Ph.D., or David Schull Russo Partners, LLC Ignacio.guerrero-ros@russopartnersllc.com David.schull@russopartnersllc.com (858) 717-2310 | Investor Contact Kevin Gardner LifeSci Advisors kgardner@lifesciadvisors.com (617) 283-2856 |
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