Telomir Pharmaceuticals Reports Telomir-1's Unique Profile Restores Mitochondrial Health While Improving Oxidative Stress Without Promoting Cell Proliferation in Diseased Human Cell Lines Highlighting Potential Relevance to Neurodegenerative Pathways in Alzheimer's and Parkinson's
Telomir Pharmaceuticals (NASDAQ:TELO) has announced promising preclinical results for its lead compound Telomir-1 in treating mitochondrial dysfunction. The study, conducted with Smart Assays Biotechnologies, demonstrated that Telomir-1 successfully restored mitochondrial function in Hutchinson-Gilford Progeria Syndrome (HGPS) cells without triggering oxidative stress or cell proliferation.
Key findings show that Telomir-1 increased mitochondrial energy production, reduced reactive oxygen species (ROS), and showed stronger effects in diseased cells compared to healthy controls. The compound's unique profile suggests potential applications in treating neurodegenerative conditions including Parkinson's, Alzheimer's, and ALS, where mitochondrial failure drives disease progression.
The company is currently finalizing IND-enabling work and evaluating potential clinical indications for Telomir-1's development pathway.
Telomir Pharmaceuticals (NASDAQ:TELO) ha annunciato risultati preclinici promettenti per il suo composto principale Telomir-1 nel trattamento della disfunzione mitocondriale. Lo studio, condotto in collaborazione con Smart Assays Biotechnologies, ha dimostrato che Telomir-1 è in grado di ripristinare con successo la funzione mitocondriale nelle cellule affette dalla Sindrome di Hutchinson-Gilford (HGPS), senza causare stress ossidativo o proliferazione cellulare.
I risultati chiave evidenziano che Telomir-1 ha aumentato la produzione di energia mitocondriale, ridotto le specie reattive dell'ossigeno (ROS) e mostrato effetti più marcati nelle cellule malate rispetto a quelle sane. Il profilo unico del composto suggerisce potenziali applicazioni nel trattamento di patologie neurodegenerative come Parkinson, Alzheimer e SLA, dove il malfunzionamento mitocondriale è un fattore chiave nella progressione della malattia.
L'azienda sta attualmente completando le attività necessarie per l'autorizzazione IND e valutando le possibili indicazioni cliniche per il percorso di sviluppo di Telomir-1.
Telomir Pharmaceuticals (NASDAQ:TELO) ha anunciado resultados preclínicos prometedores para su compuesto principal Telomir-1 en el tratamiento de la disfunción mitocondrial. El estudio, realizado en colaboración con Smart Assays Biotechnologies, demostró que Telomir-1 restauró con éxito la función mitocondrial en células con Síndrome de Hutchinson-Gilford (HGPS) sin provocar estrés oxidativo ni proliferación celular.
Los hallazgos clave muestran que Telomir-1 aumentó la producción de energía mitocondrial, redujo las especies reactivas de oxígeno (ROS) y presentó efectos más fuertes en células enfermas en comparación con células sanas. El perfil único del compuesto sugiere aplicaciones potenciales en el tratamiento de enfermedades neurodegenerativas como Parkinson, Alzheimer y ELA, donde la falla mitocondrial impulsa la progresión de la enfermedad.
La compañía está finalizando actualmente los trabajos para la habilitación IND y evaluando posibles indicaciones clínicas para la ruta de desarrollo de Telomir-1.
Telomir Pharmaceuticals (NASDAQ:TELO)는 미토콘드리아 기능 장애 치료를 위한 주력 화합물 Telomir-1의 유망한 전임상 결과를 발표했습니다. Smart Assays Biotechnologies와 함께 수행한 연구에서 Telomir-1은 허친슨-길포드 조로증(HGPS) 세포에서 산화 스트레스나 세포 증식을 유발하지 않고 미토콘드리아 기능을 성공적으로 복원하는 것으로 나타났습니다.
주요 결과는 Telomir-1이 미토콘드리아 에너지 생산을 증가시키고, 활성산소종(ROS)을 감소시켰으며, 건강한 세포보다 질병 세포에서 더 강한 효과를 보였음을 보여줍니다. 이 화합물의 독특한 특성은 미토콘드리아 기능 부전이 질병 진행을 촉진하는 파킨슨병, 알츠하이머, ALS 등 신경퇴행성 질환 치료에 잠재적 응용 가능성을 시사합니다.
회사는 현재 IND 승인 준비 작업을 마무리하고 Telomir-1 개발 경로에 대한 임상 적용 가능성을 평가 중입니다.
Telomir Pharmaceuticals (NASDAQ:TELO) a annoncé des résultats précliniques prometteurs pour son composé principal Telomir-1 dans le traitement des dysfonctionnements mitochondriaux. L'étude, réalisée en collaboration avec Smart Assays Biotechnologies, a démontré que Telomir-1 restaurait avec succès la fonction mitochondriale dans des cellules atteintes du syndrome de Hutchinson-Gilford (HGPS) sans provoquer de stress oxydatif ni de prolifération cellulaire.
Les résultats clés montrent que Telomir-1 a augmenté la production d'énergie mitochondriale, réduit les espèces réactives de l'oxygène (ROS) et a eu des effets plus marqués dans les cellules malades comparées aux cellules saines. Le profil unique du composé suggère des applications potentielles dans le traitement de maladies neurodégénératives telles que Parkinson, Alzheimer et SLA, où la défaillance mitochondriale est un moteur de la progression de la maladie.
L'entreprise finalise actuellement les travaux nécessaires à l'autorisation IND et évalue les indications cliniques potentielles pour la voie de développement de Telomir-1.
Telomir Pharmaceuticals (NASDAQ:TELO) hat vielversprechende präklinische Ergebnisse für seine Leitverbindung Telomir-1 bei der Behandlung mitochondrialer Dysfunktion bekannt gegeben. Die Studie, die in Zusammenarbeit mit Smart Assays Biotechnologies durchgeführt wurde, zeigte, dass Telomir-1 die mitochondriale Funktion in Zellen mit Hutchinson-Gilford-Progerie-Syndrom (HGPS) erfolgreich wiederherstellte, ohne oxidativen Stress oder Zellproliferation auszulösen.
Wesentliche Erkenntnisse zeigen, dass Telomir-1 die mitochondriale Energieproduktion steigerte, reaktive Sauerstoffspezies (ROS) reduzierte und in erkrankten Zellen stärkere Effekte zeigte als in gesunden Kontrollzellen. Das einzigartige Profil der Verbindung deutet auf potenzielle Anwendungen bei der Behandlung neurodegenerativer Erkrankungen wie Parkinson, Alzheimer und ALS hin, bei denen mitochondrialer Versagen den Krankheitsverlauf antreibt.
Das Unternehmen befindet sich derzeit in der Abschlussphase der IND-fähigen Arbeiten und bewertet potenzielle klinische Indikationen für den Entwicklungsweg von Telomir-1.
- Telomir-1 demonstrated successful restoration of mitochondrial function in diseased cells
- Compound showed selective effect in diseased tissue with reduced impact on healthy cells
- Successfully increased energy production without triggering harmful cell division
- Reduced reactive oxygen species (ROS) under stress conditions
- Potential applications across multiple major diseases including Parkinson's, Alzheimer's, and ALS
- Still in preclinical stage with no human trial data
- Clinical indication strategy not yet finalized
- Timeline to clinical trials not specified
Insights
Telomir-1 shows promising mitochondrial restoration in diseased cells without harmful side effects, potentially applicable to neurodegenerative disorders.
Telomir Pharmaceuticals has reported noteworthy preclinical results for their lead compound Telomir-1 that merit attention. The data demonstrates a unique therapeutic profile where the compound restores mitochondrial function in cells from Hutchinson-Gilford Progeria Syndrome (HGPS) patients without the typical drawbacks seen with mitochondrial-targeting agents.
What stands out most is the compound's triple-benefit mechanism: it increases mitochondrial energy production while simultaneously reducing reactive oxygen species (ROS) and avoiding unwanted cell proliferation. This is scientifically significant because most compounds that boost mitochondrial activity typically increase ROS production, which can damage cells further.
The selectivity profile is particularly interesting - Telomir-1 showed stronger effects in diseased Progeria cells compared to healthy controls. This suggests a disease-specific targeting mechanism that could minimize off-target effects, addressing a common challenge in drug development.
From a translational perspective, the mitochondrial dysfunction and oxidative stress observed in Progeria share pathological similarities with more common neurodegenerative conditions like Parkinson's and Alzheimer's. The company's strategic evaluation of potential indications suggests they're taking a data-driven approach to clinical development.
While these results are encouraging, it's important to note these remain preclinical findings - Telomir is still finalizing IND-enabling work and has not yet entered human trials. The transition from preclinical to clinical success is notoriously challenging, particularly for complex neurological conditions.
Promising preclinical results highlight Telomir-1's unique mechanism with potential applications across multiple high-value neurodegenerative markets.
Telomir's preclinical findings for Telomir-1 reveal a differentiated pharmaceutical profile that addresses a fundamental challenge in targeting mitochondrial dysfunction. The compound's ability to restore energy production without increasing oxidative stress or triggering unwanted cell proliferation represents a potential competitive advantage in the mitochondrial therapeutics landscape.
The company's strategic positioning is noteworthy. By demonstrating efficacy in a Progeria model while highlighting mechanistic relevance to larger market indications like Parkinson's, Alzheimer's, and ALS, Telomir is creating multiple potential commercialization pathways. These neurodegenerative markets represent substantial commercial opportunities due to high unmet need and limited effective treatments.
From a development perspective, Telomir appears to be approaching the IND stage, with the press release noting they are "finalizing IND-enabling work." This suggests they're transitioning from discovery to clinical development, a critical value inflection point for biotechnology companies.
The collaboration with Smart Assays Biotechnologies adds credibility to these findings. Their CEO's statement that Telomir-1 "stands out with one of the most distinctive profiles we've seen" provides third-party validation of the compound's potential differentiation.
While the news is positive, investors should recognize that Telomir has not yet disclosed their lead indication selection or specific clinical timeline. The statement that they are "evaluating several possible initial clinical indications" indicates strategic optionality but also suggests the clinical development path remains to be fully defined. As with all preclinical-stage biotechnology assets, significant development and regulatory hurdles remain before potential commercialization.
This unique cellular activity may help restore energy balance and improve essential cell functions such as protein synthesis and membrane stability in diseases like Parkinson's ALS Alzheimer's and Progeria where mitochondrial failure and oxidative stress drive progression
MIAMI, FL / ACCESS Newswire / July 24, 2025 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), or Telomir, a preclinical-stage biotechnology company developing therapies that target the root causes of biological aging and age-related diseases, today announced new preclinical results showing that its lead compound, Telomir-1, restored mitochondrial function without triggering oxidative stress or cell proliferation in human cells derived from a patient with Hutchinson-Gilford Progeria Syndrome (HGPS).
The study was conducted in collaboration with Smart Assays Biotechnologies Ltd., a leading preclinical contract research organization specializing in advanced cell-based models and drug mechanism profiling. Researchers used a Progeria human fibroblast cell line that models the mitochondrial dysfunction, oxidative stress, and premature aging associated with this rare genetic disorder.
"This cell line-specific metabolic response may reflect selective engagement of mitochondrial pathways-an encouraging signal for therapeutic programs targeting neurodegenerative diseases where mitochondrial dysfunction drives progression," said Dr. Raphael Mayer, CEO of Smart Assays. "For example, the compound's mode of action may be relevant to Parkinson's disease, where mitochondrial stress and redox imbalance directly contribute to dopaminergic neuron loss. We've worked with many companies developing mitochondrial-targeted therapies, and Telomir-1 stands out with one of the most distinctive profiles we've seen."
A Targeted Approach to Cellular Energy Restoration
Mitochondria are the "power plants" of the cell, responsible for generating the energy needed for survival, repair, and communication. In many serious diseases-including Progeria, Werner's disease, ALS, Parkinson's, and Alzheimer's-mitochondria lose function, and cells cannot produce the energy required to maintain health.
While restoring mitochondrial energy is a promising strategy, doing so without increasing reactive oxygen species (ROS) or causing genetically unstable cells to divide is critical. In this study, Telomir-1 demonstrated a unique activity profile: restoring energy selectively in diseased cells, without significantly affecting control fibroblasts.
In this Progeria model, Telomir-1:
Significantly increased mitochondrial energy production (WST-1 assay):
Indicating improved energy generation in metabolically compromised cells-a fundamental requirement for cellular repair and function.Did not increase the number of live cells (Calcein staining):
Suggesting Telomir-1 restored energy without triggering cell division, which may offer important safety advantages in fragile or genetically damaged cells.Reduced reactive oxygen species (ROS) under both basal and metal-induced stress conditions: ROS are damaging molecules linked to mitochondrial dysfunction and cellular decline. In this model, reducing ROS is especially important, as oxidative stress contributes to degeneration in genetically unstable cells such as those seen in Progeria and other mitochondrial-driven diseases.
Showed a stronger effect in Progeria cells than in healthy fibroblast controls:
Indicating a potentially selective effect in diseased tissue, with reduced risk of unwanted stimulation in healthy cells.
"In Progeria and other conditions where cells are genetically unstable, restoring function without forcing replication is an important safety consideration," said Dr. Itzchak Angel, CSA at Telomir. "Generally, increasing mitochondrial activity is associated with an increase in ROS production, which can be harmful to the cell.Telomir-1's ability to boost energy production, while reducing ROS and without triggering proliferation sets it apart from many compounds in this space."
Therapeutic Potential Across Mitochondrial-Driven Diseases
The mechanism observed in this study may have broader relevance across diseases where mitochondrial failure plays a central role, including:
Parkinson's disease: Mitochondrial dysfunction contributes to dopaminergic neuron loss and motor decline. Telomir-1 may support neuron survival by improving cellular energy.
ALS (Amyotrophic Lateral Sclerosis): Motor neurons rely on mitochondrial integrity. Restoring energy without overstimulating growth could help stabilize cell function.
Alzheimer's disease: Linked to early mitochondrial decline and oxidative stress, which precede memory loss and cognitive deterioration.
Progeria (HGPS): A pediatric aging disorder driven by oxidative and mitochondrial damage-offering a focused model for evaluating therapeutic impact.
"We continue to see Telomir-1 produce consistent, differentiated results across multiple models," said Erez Aminov, CEO of Telomir. "The ability to restore mitochondrial function without increasing ROS or activating cell replication may represent a fundamentally new approach to treating complex diseases rooted in energy failure."
Targeted Clinical Strategy Underway
Telomir has completed multiple preclinical studies in human cell models and in vivo systems and is finalizing IND-enabling work for Telomir-1. The company continues to evaluate several possible initial clinical indications, guided by data, unmet medical need, and regulatory strategy.
"We're now focused on selecting the right disease target that aligns with our mechanism and allows us to deliver the greatest clinical impact," added Aminov. "We look forward to sharing our next steps as we move toward the clinic."
Cautionary Note Regarding Forward-Looking Statements
This press release, statements of Telomir's management or advisors related thereto, and the statements contained in the news story linked in this release contain "forward-looking statements," which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These risks and uncertainties include, but are not limited to, the potential use of the data from our studies, our ability to develop and commercialize Telomir-1 for specific indications, and the safety of Telomir-1.
Any forward-looking statements in this press release are based on Telomir's current expectations, estimates and projections only as of the date of this release. These and other risks concerning Telomir's programs and operations are described in additional detail in its Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which are on file with the SEC and available at www.sec.gov. Telomir explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.
Contact Information
Helga Moya
info@telomirpharma.com
(786) 396-6723
SOURCE: Telomir Pharmaceuticals, Inc
View the original press release on ACCESS Newswire
FAQ
What are the key findings of Telomir-1's preclinical results for TELO stock?
How does Telomir-1 differ from other mitochondrial-targeted therapies?
What diseases could Telomir Pharmaceuticals (TELO) potentially treat with Telomir-1?
What is the current development stage of Telomir-1 by TELO?
How does Telomir-1 affect mitochondrial function in diseased cells?
