Clene and APST Enter Into an Agreement to Support FDA’s Recommendation for Long-Term NfL Biomarker Analyses of CNM-Au8®’s Impact on NfL Reduction
Clene Inc. (NASDAQ: CLNN) has entered into an agreement with APST Research GmbH to utilize APST's extensive neurofilament light chain (NfL) database to support FDA-recommended analyses of CNM-Au8®'s effect on NfL decline in ALS patients. APST maintains one of the largest ALS repositories with data from over 4,300 patients, including serum NfL and self-reported ALSFRS-R scores.
The collaboration will enable Clene to compare NfL changes observed in participants of NIH-sponsored Expanded Access Protocols (EAPs) to matched controls from the APST dataset. This analysis aims to demonstrate that the rate of NfL change is associated with survival in ALS patients.
Clene has supported nearly 500 ALS patients through three EAP programs to collect Real-World Evidence of CNM-Au8's effects. The company plans to submit a statistical analysis plan to the FDA soon and, if results are positive, will support a New Drug Application (NDA) for potential Accelerated Approval of CNM-Au8 in ALS, planned for the second half of 2025.
Across over 800 participant years of treatment with CNM-Au8, no significant safety concerns or trends have been identified, with no serious adverse events related to the treatment reported to date.
Clene Inc. (NASDAQ: CLNN) ha stipulato un accordo con APST Research GmbH per utilizzare l'estesa banca dati sulla catena leggera di neurofilamenti (NfL) di APST, al fine di supportare le analisi raccomandate dalla FDA riguardanti l'effetto di CNM-Au8® sulla diminuzione dei NfL nei pazienti affetti da SLA. APST gestisce uno dei più grandi archivi di SLA con dati provenienti da oltre 4.300 pazienti, inclusi i livelli di NfL nel siero e i punteggi autovalutati dell'ALSFRS-R.
Questa collaborazione consentirà a Clene di confrontare i cambiamenti di NfL osservati nei partecipanti ai protocolli di accesso espanso (EAP) sponsorizzati dal NIH con i controlli abbinati del dataset di APST. Questa analisi mira a dimostrare che il tasso di cambiamento dei NfL è associato alla sopravvivenza nei pazienti affetti da SLA.
Clene ha supportato quasi 500 pazienti affetti da SLA attraverso tre programmi EAP per raccogliere evidenze dal mondo reale sugli effetti di CNM-Au8. L'azienda prevede di presentare a breve un piano di analisi statistica alla FDA e, se i risultati saranno positivi, sosterrà una domanda di approvazione di nuovo farmaco (NDA) per l'approvazione accelerata di CNM-Au8 nella SLA, prevista per la seconda metà del 2025.
In oltre 800 anni partecipanti di trattamento con CNM-Au8, non sono stati identificati problemi o tendenze di sicurezza significativi, con nessun evento avverso grave relativo al trattamento riportato fino ad oggi.
Clene Inc. (NASDAQ: CLNN) ha firmado un acuerdo con APST Research GmbH para utilizar la extensa base de datos de la cadena ligera de neurofilamentos (NfL) de APST, con el fin de apoyar los análisis recomendados por la FDA sobre el efecto de CNM-Au8® en la disminución de NfL en pacientes con ELA. APST mantiene uno de los mayores repositorios de ELA con datos de más de 4,300 pacientes, incluidos los niveles de NfL en suero y las puntuaciones autoinformadas del ALSFRS-R.
Esta colaboración permitirá a Clene comparar los cambios en NfL observados en los participantes de los Protocolos de Acceso Ampliado (EAP) patrocinados por el NIH con controles emparejados del conjunto de datos de APST. Este análisis tiene como objetivo demostrar que la tasa de cambio de NfL está asociada con la supervivencia en pacientes con ELA.
Clene ha apoyado a casi 500 pacientes con ELA a través de tres programas EAP para recopilar evidencia del mundo real sobre los efectos de CNM-Au8. La empresa planea presentar pronto un plan de análisis estadístico a la FDA y, si los resultados son positivos, apoyará una Solicitud de Nuevo Medicamento (NDA) para la posible Aprobación Acelerada de CNM-Au8 en ELA, prevista para la segunda mitad de 2025.
En más de 800 años de tratamiento participativo con CNM-Au8, no se han identificado preocupaciones o tendencias de seguridad significativas, sin informes de eventos adversos graves relacionados con el tratamiento hasta la fecha.
클렌 Inc. (NASDAQ: CLNN)은 APST Research GmbH와 협약을 체결하여 APST의 방대한 신경섬유 경량 사슬(NfL) 데이터베이스를 활용하여 ALS 환자에서 NfL 감소에 대한 CNM-Au8®의 영향을 분석하는 FDA 권장 분석을 지원합니다. APST는 4,300명 이상의 환자로부터 수집된 데이터가 포함된 가장 큰 ALS 저장소 중 하나를 유지하고 있으며, 여기에는 혈청 NfL과 자가 보고된 ALSFRS-R 점수가 포함됩니다.
이번 협업을 통해 클렌은 NIH가 후원하는 확장 접근 프로토콜(EAP) 참가자에서 관찰된 NfL 변화를 APST 데이터 세트의 매칭된 대조군과 비교할 수 있게 됩니다. 이 분석은 NfL 변화율이 ALS 환자의 생존과 관련이 있음을 입증하는 것을 목표로 합니다.
클렌은 CNM-Au8의 효과에 대한 실제 증거를 수집하기 위해 세 개의 EAP 프로그램을 통해 거의 500명의 ALS 환자를 지원했습니다. 회사는 곧 FDA에 통계 분석 계획을 제출할 예정이며, 결과가 긍정적일 경우 2025년 하반기에 ALS에서 CNM-Au8의 가속 승인을 위한 신약 신청(NDA)을 지원할 것입니다.
CNM-Au8로 치료한 800년 이상의 참가자 연수 동안, 치료와 관련된 심각한 부작용은 보고되지 않았으며, 안전성 문제나 경향도 발견되지 않았습니다.
Clene Inc. (NASDAQ: CLNN) a conclu un accord avec APST Research GmbH pour utiliser la vaste base de données sur la chaîne légère de neurofilaments (NfL) d'APST afin de soutenir les analyses recommandées par la FDA concernant l'effet de CNM-Au8® sur la diminution des NfL chez les patients atteints de SLA. APST maintient l'un des plus grands dépôts de données sur la SLA avec des informations provenant de plus de 4 300 patients, y compris les niveaux de NfL dans le sérum et les scores ALSFRS-R auto-évalués.
Cette collaboration permettra à Clene de comparer les changements de NfL observés chez les participants aux Protocoles d'Accès Élargi (EAP) sponsorisés par le NIH avec des témoins appariés du jeu de données d'APST. Cette analyse vise à démontrer que le taux de changement de NfL est associé à la survie des patients atteints de SLA.
Clene a soutenu près de 500 patients atteints de SLA à travers trois programmes EAP pour collecter des preuves du monde réel sur les effets de CNM-Au8. L'entreprise prévoit de soumettre prochainement un plan d'analyse statistique à la FDA et, si les résultats sont positifs, elle soutiendra une demande de nouveau médicament (NDA) pour une approbation accélérée de CNM-Au8 dans la SLA, prévue pour la seconde moitié de 2025.
Au cours de plus de 800 années de traitement avec CNM-Au8, aucune préoccupation ou tendance de sécurité significative n'a été identifiée, et aucun événement indésirable grave lié au traitement n'a été signalé à ce jour.
Clene Inc. (NASDAQ: CLNN) hat eine Vereinbarung mit APST Research GmbH getroffen, um die umfangreiche Datenbank zur leichten Neurofilamentkette (NfL) von APST zu nutzen, um die von der FDA empfohlenen Analysen der Auswirkungen von CNM-Au8® auf den NfL-Rückgang bei ALS-Patienten zu unterstützen. APST betreibt eines der größten ALS-Archive mit Daten von über 4.300 Patienten, einschließlich Serum-NfL und selbstberichteten ALSFRS-R-Werten.
Die Zusammenarbeit ermöglicht es Clene, die NfL-Veränderungen, die bei Teilnehmern an NIH-gesponserten erweiterten Zugangsprotokollen (EAPs) beobachtet wurden, mit den passenden Kontrollen aus dem APST-Datensatz zu vergleichen. Diese Analyse soll zeigen, dass die Änderungsrate des NfL mit der Überlebensrate bei ALS-Patienten verbunden ist.
Clene hat fast 500 ALS-Patienten durch drei EAP-Programme unterstützt, um Real-World-Evidenz über die Wirkungen von CNM-Au8 zu sammeln. Das Unternehmen plant, bald einen statistischen Analyseplan bei der FDA einzureichen und wird, sofern die Ergebnisse positiv sind, einen Antrag auf beschleunigte Zulassung (NDA) für CNM-Au8 bei ALS unterstützen, der für die zweite Hälfte des Jahres 2025 geplant ist.
In über 800 Teilnehmerjahren der Behandlung mit CNM-Au8 wurden keine signifikanten Sicherheitsbedenken oder -tendenzen festgestellt, und bis heute wurden keine schweren unerwünschten Ereignisse im Zusammenhang mit der Behandlung gemeldet.
- Agreement with APST provides access to one of the largest ALS NfL datasets to support FDA requirements
- Plans to submit NDA for CNM-Au8 in ALS in second half of 2025
- CNM-Au8 showed no significant safety concerns across 800+ participant treatment years
- No serious adverse events related to CNM-Au8 reported to date
- Real-World Evidence from nearly 500 ALS patients being collected through EAP programs
- FDA requiring additional supportive evidence before approval consideration
- NDA submission not planned until second half of 2025
- Accelerated Approval pathway still contingent on positive NfL reduction data
Insights
Clene's Strategic Partnership with APST Research Strengthens Regulatory Path for CNM-Au8 in ALS
Clene's new agreement with APST Research represents a critical strategic move to address specific FDA requirements for their lead ALS candidate. This partnership gives Clene access to one of the world's largest ALS neurofilament light chain (NfL) datasets - a important biomarker that measures neuronal damage and has become increasingly important in ALS drug development.
The FDA's specific request for additional NfL analyses signals that regulators see potential in CNM-Au8 but require more robust evidence of its effect on this biomarker. This agreement directly addresses this regulatory hurdle by providing matched control data from over 4,300 ALS patients to compare against Clene's Expanded Access Program participants.
The timing is particularly significant as Clene aims to submit their NDA in H2 2025 via the Accelerated Approval pathway. This regulatory strategy hinges on demonstrating that NfL reduction correlates with meaningful clinical outcomes like survival - precisely what this APST dataset will help establish.
CNM-Au8's clean safety profile across 800+ participant years of treatment with no drug-related serious adverse events is particularly noteworthy for a neurological treatment. This safety record, combined with previously observed survival benefits and NfL reductions in the HEALEY trial, forms the foundation of Clene's regulatory case.
For investors, this agreement represents more than just additional data - it's a critical risk-reduction measure for Clene's regulatory strategy. By strengthening the evidence package for their planned NDA submission, Clene is addressing a specific FDA concern that could otherwise become a significant obstacle to approval.
The ALS treatment landscape remains severely underserved, with only treatment options that provide modest benefits. CNM-Au8's unique nanotherapeutic approach targeting cellular energetics represents a novel mechanism compared to existing treatments. If approved, CNM-Au8 would enter a market with approximately 30,000 ALS patients in the US alone and competition.
While this agreement strengthens Clene's regulatory position, investors should note that the company still faces the inherent challenges of neurological drug development, including the historical difficulty of translating biomarker improvements into functional benefits that satisfy regulators. The coming months will be critical as Clene finalizes their statistical analysis plan and prepares for their NDA submission.
- Longitudinal ALS patient data from APST Research will enable a more robust analysis of the effect of CNM-Au8 on NfL biomarkers
- APST Research data includes thousands of people living with ALS including clinical, survival, and longitudinal NfL data
- Clene plans to analyze the APST ALS NfL biomarker and clinical dataset to compare NfL change from its ongoing NIH-sponsored EAP to address the FDA’s request for supportive evidence of CNM-Au8’s effect on NfL reduction observed in the HEALEY ALS Platform Trial
- Clene plans to submit a New Drug Application (NDA) in second-half 2025
SALT LAKE CITY, Feb. 25, 2025 (GLOBE NEWSWIRE) -- Clene Inc. (Nasdaq: CLNN) (along with its subsidiaries, “Clene”) and its wholly owned subsidiary Clene Nanomedicine Inc., a late clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), today announced that it has entered into an agreement with German-based APST Research GmbH (APST) to utilize its extensive NfL database to support the FDA-recommended analyses of CNM-Au8®’s effect on NfL decline in participants in ongoing Expanded Access Protocols (EAPs).
APST maintains one of the largest comprehensive ALS repositories of people living with ALS, including demographic data, clinical data, ALS motor phenotypes and biomarker data, specifically, serum neurofilament light chain (sNfL). The database in the repository comprises extensive sNfL data from over 4,300 ALS patients as well as self-reported ALSFRS-R. The APST platform offers digitized data on thousands of individuals living with ALS to comprehensively view the disease's progression by gathering information on ALS phenotypes, biomarker data, and patient-reported outcomes. This effort utilized cutting-edge data collection and analytics to provide a comprehensive long-term natural history of NfL change. Comparisons to the APST NfL dataset will be a crucial element of the FDA-recommended statistical analysis plan for NfL evaluations of NIH-sponsored EAP participants.
The NfL dataset being analyzed by Clene includes data from more than 1625 ALS patients, aligning the biomarker data of NfL to clinical ALSFRS-R assessments, slow vital capacity (SVC), and clinical events such as ventilation support and nutrition intervention. The objective of the planned analyses is to compare NfL change observed in the NIH-sponsored EAP participants to matched controls using the APST dataset, and to demonstrate that the rate of NfL change is associated with survival in people living with ALS.
Clene has supported the treatment of nearly 500 people living with ALS through its three (3) ALS EAP programs to collect Real-World Evidence (RWE) of the effects of CNM-Au8 in ALS. RWE evidence collected from EAP participants may be supportive of the observed survival improvement and NfL decline observed in the HEALEY ALS Platform Trial with CNM-Au8 30 mg treatment. Clene plans to submit the statistical analysis plan for the NIH-sponsored EAP NfL analyses to the FDA shortly. Analyses of NIH-sponsored EAP NfL reduction, if positive, will support the planned NDA submission for potential Accelerated Approval of CNM-Au8 in ALS, planned in the second half of 2025.
Across over 800 participant years of treatment with CNM-Au8, no significant safety concerns or safety trends have been identified. No serious adverse events (SAEs) have been identified as related to CNM-Au8 treatment by any investigator to date.
“We are excited to enter into this endeavor with one of the world’s largest ALS NfL datasets in order to supplement available biomarker NfL data from our NIH-sponsored EAP, and to support the existing clinical study data for the potential review of an application for approval of CNM-Au8 in ALS via an accelerated regulatory pathway,” said Clene’s CEO, Rob Etherington. “We remain dedicated to the ALS community and are honored to continue our efforts to help critically ill patients and their families.”
“Our extensive and robust data collection empowers pharmaceutical companies to advance their clinical research and trials, driving us toward a deeper understanding of ALS disease progression” said APST's founder and CEO, Thomas Meyer, a renowned ALS key opinion leader.
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 APST Research
APST is a digital research platform and service provider for clinical research in ALS. Collaborating with leading ALS centers in Germany and Europe, the APST platform generates clinical, phenotypic and biomarker data, patient-reported outcomes and real-world experiences of innovative medicines and technologies in ALS. APST's mobile “ALS App” directly connects people with ALS, providing trusted ways of generating patient-centric data.
APST is on a mission to meet the needs of pharmaceutical companies pioneering urgently needed ALS medicines with high-quality patient data and clinical knowledge.
For more information on how APST's ALS repository supports ALS clinical research, visit https://apstresearch.com/en/ or follow us on 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 Clene’s expectations regarding the availability of an accelerated approval regulatory pathway, the timing of clinical trials and the submission of an NDA, Clene’s intention to follow the FDA’s recommendation to provide data from the ongoing EAPs and address the FDA’s requests, and that Clene can provide the additional evidence to meet the FDA’s data requests. In addition, any statements that refer to 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. Some factors that could cause actual results to differ include general market conditions; whether clinical trials of our drug candidates fail to demonstrate safety and efficacy 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; Clene’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its 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; Clene’s ability to achieve commercial success for its drug candidates, if approved; Clene’s limited operating history and its ability to obtain additional funding for operations and to complete the development and commercialization of its 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 |
FAQ
What is the purpose of Clene's agreement with APST Research for CNM-Au8 development?
How many ALS patients are included in the APST Research database that CLNN will access?
When does Clene (CLNN) plan to submit its New Drug Application for CNM-Au8 in ALS?
What safety profile has CNM-Au8 demonstrated in CLNN's ALS treatment programs?
How many ALS patients has Clene (CLNN) treated through its Expanded Access Protocol programs?
What biomarker is Clene focusing on for its CNM-Au8 ALS treatment approval pathway?
