Telomir Demonstrates Telomir-1 Reverses Epigenetic Gene Silencing of STAT1, Restoring Tumor Suppressor in Human Prostate Cancer Cells, Outperforming Chemotherapy and Rapamycin
Telomir Pharmaceuticals (NASDAQ:TELO) has announced breakthrough preclinical findings for its Telomir-1 drug candidate in treating aggressive prostate cancer. The study demonstrated that Telomir-1 successfully reversed epigenetic gene silencing of STAT1, a crucial tumor suppressor, outperforming both Paclitaxel and Rapamycin in PC3 xenograft models.
The drug showed superior efficacy in a 21-day oral administration trial, fully reversing STAT1 hypermethylation in a dose-dependent manner. Additionally, Telomir-1 reduced hypermethylation of TMS1, another key tumor suppressor, while maintaining a favorable safety profile by not elongating telomeres in cancerous cells.
These findings build upon previous March 2025 results where Telomir-1 reduced tumor volume by approximately 50% and eliminated chemotherapy-induced mortality when combined with Paclitaxel.
Telomir Pharmaceuticals (NASDAQ:TELO) ha annunciato risultati preclinici rivoluzionari per il suo candidato farmaco Telomir-1 nel trattamento del cancro alla prostata aggressivo. Lo studio ha dimostrato che Telomir-1 ha invertito con successo la silenziazione epigenetica del gene STAT1, un importante soppressore tumorale, superando sia Paclitaxel che Rapamicina nei modelli xenotrapianti PC3.
Il farmaco ha mostrato un’efficacia superiore in una prova di somministrazione orale di 21 giorni, invertendo completamente l’ipermetilazione di STAT1 in modo dose-dipendente. Inoltre, Telomir-1 ha ridotto l’ipermetilazione di TMS1, un altro soppressore tumorale chiave, mantenendo un profilo di sicurezza favorevole senza allungare i telomeri nelle cellule tumorali.
Questi risultati si basano su precedenti dati di marzo 2025 in cui Telomir-1 ha ridotto il volume tumorale di circa il 50% ed eliminato la mortalità indotta dalla chemioterapia quando combinato con Paclitaxel.
Telomir Pharmaceuticals (NASDAQ:TELO) ha anunciado hallazgos preclínicos innovadores para su candidato a fármaco Telomir-1 en el tratamiento del cáncer de próstata agresivo. El estudio demostró que Telomir-1 revirtió con éxito el silenciamiento epigenético del gen STAT1, un supresor tumoral crucial, superando tanto a Paclitaxel como a Rapamicina en modelos xenoinjertados PC3.
El fármaco mostró una eficacia superior en un ensayo de administración oral de 21 días, revirtiendo completamente la hipermetilación de STAT1 de manera dependiente de la dosis. Además, Telomir-1 redujo la hipermetilación de TMS1, otro supresor tumoral clave, manteniendo un perfil de seguridad favorable sin alargar los telómeros en células cancerosas.
Estos hallazgos se basan en resultados previos de marzo de 2025 donde Telomir-1 redujo el volumen tumoral aproximadamente en un 50% y eliminó la mortalidad inducida por la quimioterapia cuando se combinó con Paclitaxel.
Telomir Pharmaceuticals (NASDAQ:TELO)는 공격적인 전립선암 치료를 위한 후보 약물 Telomir-1의 획기적인 전임상 결과를 발표했습니다. 연구 결과 Telomir-1은 중요한 종양 억제자인 STAT1의 후생유전학적 유전자 침묵을 성공적으로 역전시켰으며, PC3 이종이식 모델에서 Paclitaxel과 Rapamycin보다 뛰어난 효능을 보였습니다.
이 약물은 21일간 경구 투여 시험에서 우수한 효능을 나타내며, 용량 의존적으로 STAT1 과메틸화를 완전히 역전시켰습니다. 또한 Telomir-1은 또 다른 핵심 종양 억제자인 TMS1의 과메틸화를 감소시키면서 암세포의 텔로미어를 연장하지 않아 안전성 프로파일도 우수했습니다.
이 결과는 2025년 3월 이전 연구에서 Telomir-1이 종양 부피를 약 50% 감소시키고 Paclitaxel과 병용 시 화학요법 유발 사망률을 제거한 것을 기반으로 합니다.
Telomir Pharmaceuticals (NASDAQ:TELO) a annoncé des résultats précliniques révolutionnaires pour son candidat-médicament Telomir-1 dans le traitement du cancer de la prostate agressif. L’étude a démontré que Telomir-1 a réussi à inverser le silence épigénétique du gène STAT1, un suppresseur de tumeur crucial, surpassant à la fois le Paclitaxel et la Rapamycine dans des modèles xénogreffes PC3.
Le médicament a montré une efficacité supérieure lors d’un essai d’administration orale de 21 jours, inversant complètement l’hyperméthylation de STAT1 de manière dépendante de la dose. De plus, Telomir-1 a réduit l’hyperméthylation de TMS1, un autre suppresseur tumoral clé, tout en maintenant un profil de sécurité favorable en n’allongeant pas les télomères dans les cellules cancéreuses.
Ces résultats s’appuient sur des données antérieures de mars 2025 où Telomir-1 avait réduit le volume tumoral d’environ 50% et éliminé la mortalité induite par la chimiothérapie lorsqu’il était combiné avec le Paclitaxel.
Telomir Pharmaceuticals (NASDAQ:TELO) hat bahnbrechende präklinische Ergebnisse für seinen Wirkstoffkandidaten Telomir-1 bei der Behandlung von aggressivem Prostatakrebs bekannt gegeben. Die Studie zeigte, dass Telomir-1 die epigenetische Gen-Silenzierung von STAT1, einem wichtigen Tumorsuppressor, erfolgreich umkehrte und dabei sowohl Paclitaxel als auch Rapamycin in PC3-Xenotransplantatmodellen übertraf.
Das Medikament zeigte eine überlegene Wirksamkeit in einer 21-tägigen oralen Verabreichungsstudie und kehrte die STAT1-Hypermethylierung dosisabhängig vollständig um. Zusätzlich reduzierte Telomir-1 die Hypermethylierung von TMS1, einem weiteren wichtigen Tumorsuppressor, und behielt dabei ein günstiges Sicherheitsprofil bei, indem es die Telomere in Krebszellen nicht verlängerte.
Diese Ergebnisse bauen auf früheren Resultaten vom März 2025 auf, bei denen Telomir-1 das Tumorvolumen um etwa 50% reduzierte und die durch Chemotherapie induzierte Mortalität in Kombination mit Paclitaxel eliminierte.
- Demonstrated superior efficacy over Paclitaxel and Rapamycin in reversing STAT1 gene silencing
- Achieved approximately 50% tumor volume reduction in previous studies
- Eliminated chemotherapy-induced mortality when combined with Paclitaxel
- Showed dual action on both STAT1 and TMS1 tumor suppressors
- Demonstrated favorable safety profile without elongating telomeres in cancer cells
- Still in preclinical stage with no defined timeline for clinical trials
- Initial IND indication yet to be announced
- Multiple competing therapeutic areas could delay focus on specific indications
Insights
Telomir-1 shows remarkable ability to reverse cancer-specific epigenetic silencing, potentially reactivating immune surveillance mechanisms while maintaining safety profile.
The newly reported preclinical results for Telomir-1 represent a significant breakthrough in cancer epigenetics. The drug's ability to fully reverse hypermethylation of the STAT1 gene promoter in aggressive PC3 prostate cancer cells is particularly noteworthy. STAT1 functions as a master regulator of immune surveillance and programmed cell death—when silenced through hypermethylation, cancer cells effectively become invisible to the immune system. That Telomir-1 outperformed both
The dual action on both STAT1 and TMS1 (also known as ASC/PYCARD) is mechanistically significant. While other compounds affected TMS1 methylation, only Telomir-1 demonstrated this dual modulatory effect. This is critical because these genes control complementary anti-cancer pathways: STAT1 regulates immune response while TMS1 governs inflammasome activation and apoptosis. By potentially reactivating both pathways simultaneously, Telomir-1 could address two fundamental mechanisms of cancer progression—immune evasion and resistance to programmed cell death.
Perhaps most important from a safety perspective is the finding that Telomir-1 did not elongate telomeres in the PC3 cancer cells. This selective action addresses a key concern in cancer therapeutics: while telomere elongation might be beneficial in treating degenerative conditions, it could potentially accelerate cancer growth. The fact that Telomir-1 exerted its anti-tumor effects without lengthening cancer cell telomeres suggests a favorable safety profile and mechanistic selectivity.
The approximately
The new preclinical data on Telomir-1 reveals a distinct mechanism of action that differentiates it from standard cancer treatments. The drug's ability to reverse epigenetic silencing of STAT1—a critical immune regulator—in aggressive prostate cancer cells is scientifically significant and potentially valuable from a commercial perspective.
What stands out is Telomir-1's demonstrated superiority over established compounds. Outperforming both Paclitaxel (a widely-used chemotherapy) and Rapamycin (an established mTOR inhibitor) in restoring STAT1 function suggests a novel approach to cancer treatment. This mechanism could prove particularly synergistic with the rapidly growing immunotherapy market, where overcoming immune evasion mechanisms remains a significant challenge.
The dual action on both STAT1 and TMS1 gene silencing represents a potential competitive advantage, as it targets complementary pathways involved in immune function and cell death. The selective action—affecting these pathways without elongating cancer cell telomeres—addresses safety concerns that might otherwise limit development.
Beyond oncology, Telomir is pursuing multiple therapeutic applications including Wilson's disease, age-related macular degeneration, autism spectrum disorder, and dysphonia. This diversified pipeline strategy reduces clinical development risk while potentially expanding the market opportunity.
The company's preclinical stage status means substantial development risks remain. No IND (Investigational New Drug) application has been filed yet, and the transition from preclinical to clinical studies represents a significant hurdle. Additionally, the company hasn't specified which indication will be its initial clinical focus, suggesting the development strategy is still evolving. While these preclinical results are promising, investors should recognize that many promising preclinical compounds fail to demonstrate comparable efficacy and safety in human trials.
New data shows Telomir-1 fully reverses STAT1 gene silencing by DNA methylation - a key immune regulator suppressed in cancer - delivering stronger epigenetic effects than Paclitaxel or Rapamycin in aggressive PC3 tumor models.
MIAMI, FL / ACCESS Newswire / July 18, 2025 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), a preclinical-stage biotechnology company developing therapies that target the root causes of biological aging and age-related diseases, today announced compelling new preclinical findings highlighting Telomir-1's ability to reverse epigenetic gene silencing in aggressive human prostate cancer cells-achieving greater efficacy than Paclitaxel and Rapamycin in restoring the STAT1 tumor suppressor.
STAT1, a master regulator of immune surveillance and programmed cell death, is frequently silenced in advanced cancers through promoter hypermethylation. In this state, cancer cells effectively disable one of the body's key defense mechanisms-shutting down immune detection and blocking signals that would normally trigger their death. In this newly reported study, Telomir-1 administered orally over 21 days in a mouse model implanted with aggressive human prostate cancer cells (PC3 xenograft) fully reversed this hypermethylation in a dose-dependent fashion. In contrast, Paclitaxel (PTX) showed no effect, and Rapamycin achieved only partial reduction of hypermethylation of STAT1.
By restoring STAT1 activity, Telomir-1 may be reawakening the body's built-in tumor suppressor system-allowing immune cells to once again recognize, target, and eliminate cancer. This mechanism offers a potential explanation for the tumor shrinkage observed by Telomir-1 and highlights a biological pathway not addressed by standard chemotherapy.
"This study provides novel and direct molecular evidence of Telomir-1's ability to reprogram cancer epigenetics," said Erez Aminov, CEO of Telomir. "By potentially restoring the function of key tumor suppressor genes like STAT1, we're not just slowing tumor growth-we're turning the immune system back on. These results highlight Telomir-1's potential as a powerful new approach to treating aggressive cancers as combination therapy with cancer immunomodulators."
In addition to STAT1, Telomir-1 also reduced hypermethylation of TMS1 (also known as ASC or PYCARD), a pro-apoptotic tumor suppressor commonly silenced in prostate cancer. While PTX and Rapamycin showed comparable or greater effects on TMS1 methylation, Telomir-1 is unique in its ability to modulate both STAT1 and TMS1-two genes that together regulate immune response and apoptosis.
Importantly, TMS1 also plays a central role in inflammasome activation, which not only contributes to tumor cell death but also supports the immune system's ability to detect and clear abnormal cells. When TMS1 is hypermethylated, this immune signaling pathway is disrupted-reducing caspase-1 activation and inflammatory cytokine release. This can impair immune surveillance and allow cancer cells to persist undetected. By reducing TMS1 hypermethylation, Telomir-1 may help restore immune recognition and enhance the tumor's sensitivity to both chemotherapy and immunotherapy.
"PC3 cells are notoriously aggressive and resistant to treatment," said Dr. Itzchak Angel, Chief Scientific Advisor at Telomir. "The potential simultaneous reactivation of STAT1 and TMS1 by inhibition of the regulatory hypermethylation suggests that Telomir-1 may help overcome two of the biggest hurdles in cancer biology-immune evasion and apoptosis resistance. This dual action could help explain the tumor suppression observed in our earlier studies. It may also offer a potential novel horizon for Telomir-1 as an associated therapy with immunosuppressors and chemotherapies."
Selective Action on Telomeres Reinforces Safety
Importantly, in this study we have also found that Telomir-1 did not elongate telomeres in the cancerous prostate cells (PC3)-a critical finding that reinforces its cancer safety profile. While telomere elongation is a therapeutic goal in degenerative and aging-related diseases, in cancer cells, longer telomeres can fuel unchecked growth. PC3 cells already maintain short but stable telomeres via telomerase-a hallmark of malignancy. The fact that Telomir-1 exerted strong biological effects without increasing telomere length supports its selectivity in targeting telomere maintenance where appropriate.
Distinct and unique Mechanism
These new insights build upon findings previously announced on March 19, 2025, where Telomir-1 was shown to reduce tumor volume by approximately
Advancing Toward the Clinic
Telomir is conducting ongoing research to evaluate Telomir-1 across multiple therapeutic areas, including oncology, Wilson's disease, age-related macular degeneration (AMD), autism spectrum disorder, and dysphonia. The company is continuing preclinical development across these programs and plans to announce its initial IND indication at a future date.
"We remain encouraged by the preclinical data and continue to explore Telomir-1's potential across several disease areas," added Aminov. "Our team is committed to advancing this program thoughtfully and strategically as we move toward clinical development."
For more information, visit www.telomirpharma.com.
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
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