TransCode Therapeutics Announces Completion of Cohort 3 Initial Dosing with Lead Candidate in Phase 1 Clinical Trial
TransCode Therapeutics (NASDAQ: RNAZ) has announced the completion of initial dosing in Cohort 3 of its Phase 1 clinical trial for TTX-MC138, its lead candidate. Three patients were enrolled and dosed in Cohort 3, with the dose being approximately double that of Cohort 2. The Safety Review Committee approved the third cohort after reviewing safety and pharmacokinetic (PK) data from Cohorts 1 and 2.
Notable findings include: no significant safety or dose limiting toxicities reported across all nine trial patients; several patients from Cohorts 1 and 2 continuing treatment in 28-day cycles; and PK/PD analyses showing consistency with preclinical and Phase 0 trial results. Cohort 1 demonstrated 66% inhibition of miR-10b at 24 hours post-infusion, matching Phase 0 observations. TTX-MC138 showed increased activity with dose escalation in Cohort 2, maintaining consistency across subsequent administrations.
TransCode Therapeutics (NASDAQ: RNAZ) ha annunciato il completamento della somministrazione iniziale nel Coorte 3 del suo studio clinico di Fase 1 per il TTX-MC138, il suo candidato principale. Tre pazienti sono stati arruolati e trattati nel Coorte 3, con una dose approssimativamente doppia rispetto al Coorte 2. Il Comitato per la Revisione della Sicurezza ha approvato il terzo coorte dopo aver esaminato i dati di sicurezza e farmacocinetica (PK) dei Coorti 1 e 2.
Tra i risultati significativi si segnalano: nessuna tossicità significativa o limitante per la dose riportata in tutti e nove i pazienti coinvolti nello studio; diversi pazienti dei Coorti 1 e 2 hanno continuato il trattamento in cicli di 28 giorni; e analisi PK/PD che mostrano coerenza con i risultati preclinici e della fase 0. Il Coorte 1 ha dimostrato un'inibizione del 66% del miR-10b dopo 24 ore dalla somministrazione, corrispondente alle osservazioni della fase 0. Il TTX-MC138 ha mostrato un'attività aumentata con l'incremento della dose nel Coorte 2, mantenendo coerenza tra le somministrazioni successive.
TransCode Therapeutics (NASDAQ: RNAZ) ha anunciado la finalización de la dosificación inicial en el Cohorte 3 de su ensayo clínico de Fase 1 para el TTX-MC138, su candidato principal. Tres pacientes fueron inscritos y tratados en el Cohorte 3, con una dosis aproximadamente el doble que la del Cohorte 2. El Comité de Revisión de Seguridad aprobó el tercer cohorte tras revisar los datos de seguridad y farmacocinética (PK) de los Cohortes 1 y 2.
Los hallazgos notables incluyen: sin toxicidades significativas o limitantes de dosis reportadas en los nueve pacientes del ensayo; varios pacientes de los Cohortes 1 y 2 continuando el tratamiento en ciclos de 28 días; y análisis PK/PD que muestran consistencia con los resultados preclínicos y de la fase 0. El Cohorte 1 demostró una inhibición del 66% del miR-10b a las 24 horas de la infusión, igualando las observaciones de la fase 0. El TTX-MC138 mostró una actividad aumentada con la escalación de dosis en el Cohorte 2, manteniendo consistencia en las administraciones posteriores.
TransCode Therapeutics (NASDAQ: RNAZ)는 TTX-MC138의 1상 임상 시험의 Coorte 3에서 초기 투약이 완료되었음을 발표했습니다, 이 제품이 주요 후보입니다. Coorte 3에는 세 명의 환자가 등록되고 투약되었으며, 투약량은 Coorte 2의 약 두 배입니다. 안전성 검토 위원회는 Coorte 1 및 2의 안전성 및 약리학적 데이터(PK)를 검토한 후 세 번째 코호트를 승인했습니다.
주목할 만한 발견사항은: 아홉 명의 환자 모두에서 보고된 유의미한 안전성 문제나 용량 제한 독성이 없음; Coorte 1 및 2의 여러 환자가 28일 주기로 치료를 계속하고 있습니다; 그리고 PK/PD 분석 결과가 전임상 및 0단계 시험 결과와 일관성을 보여줍니다. Coorte 1은 주입 후 24시간에 miR-10b의 66% 억제를 보여주며 0단계 관찰 결과와 일치합니다. TTX-MC138은 Coorte 2에서 용량 증가와 함께 활동이 증가하였으며, 이후 투여에서도 일관성을 유지합니다.
TransCode Therapeutics (NASDAQ: RNAZ) a annoncé l'achèvement de la dose initiale dans la Cohorte 3 de son essai clinique de phase 1 pour le TTX-MC138, son principal candidat. Trois patients ont été inscrits et dosés dans la Cohorte 3, avec une dose d'environ le double de celle de la Cohorte 2. Le Comité de Revue de Sécurité a approuvé la troisième cohorte après avoir examiné les données de sécurité et pharmacocinétique (PK) des Cohortes 1 et 2.
Les résultats notables incluent : aucune toxicité significative ou limitante de dose signalée chez les neuf patients de l'essai ; plusieurs patients des Cohortes 1 et 2 continuent le traitement dans des cycles de 28 jours ; et des analyses PK/PD montrant une cohérence avec les résultats précliniques et de phase 0. La Cohorte 1 a démontré une inhibition de 66 % du miR-10b 24 heures après l'infusion, correspondant aux observations de la phase 0. Le TTX-MC138 a montré une activité accrue avec l'escalade des doses dans la Cohorte 2, maintenant la cohérence au fil des administrations suivantes.
TransCode Therapeutics (NASDAQ: RNAZ) hat den Abschluss der initialen Dosierung in Kohorte 3 seiner Phase 1-Klinikstudie für TTX-MC138, seinen Hauptkandidaten, bekannt gegeben. Drei Patienten wurden in Kohorte 3 eingeschlossen und behandelt, wobei die Dosis ungefähr doppelt so hoch ist wie in Kohorte 2. Das Sicherheitsprüfungskomitee genehmigte die dritte Kohorte, nachdem es die Sicherheits- und pharmakokinetischen (PK) Daten von Kohorte 1 und 2 überprüft hatte.
Bemerkenswerte Ergebnisse sind: keine bedeutenden Sicherheits- oder dosenbegrenzenden Toxizitäten wurden bei allen neun Studienteilnehmern berichtet; mehrere Patienten aus Kohorte 1 und 2 setzen die Behandlung in 28-Tage-Zyklen fort; und PK/PD-Analysen zeigen Konsistenz mit präklinischen und Phase-0-Studienergebnissen. Kohorte 1 zeigte 66% Inhibition von miR-10b 24 Stunden nach der Infusion, was den 0-Phasen-Beobachtungen entspricht. TTX-MC138 zeigte eine erhöhte Aktivität mit Dosissteigerung in Kohorte 2 und behielt die Konsistenz bei nachfolgenden Verabreichungen.
- No safety concerns or dose limiting toxicities reported across all nine patients
- TTX-MC138 achieved 66% inhibition of miR-10b at 24 hours post-infusion
- Successful dose escalation with increased activity in Cohort 2
- PK/PD profile consistent with preclinical and Phase 0 results
- Multiple patients continuing treatment in 28-day cycles
- None.
Insights
The latest clinical trial results for TTX-MC138 demonstrate several important positive indicators that strengthen TransCode's position in the RNA therapeutics space. The absence of significant safety issues or dose-limiting toxicities across all three cohorts is particularly noteworthy, as safety concerns often derail early-stage RNA therapeutic candidates.
The pharmacokinetic data reveals two critical aspects: First, the 66% inhibition of miR-10b at 24 hours post-infusion in Cohort 1 validates the mechanism of action and confirms the Phase 0 findings. Second, the increased activity observed with dose escalation in Cohort 2, coupled with consistency in subsequent administrations, suggests a predictable and manageable therapeutic window - a important factor for potential regulatory approval.
The successful doubling of dosage in Cohort 3 and the continuation of treatment in 28-day cycles for earlier cohort patients are particularly significant because they indicate:
- Potential for dose optimization without hitting safety ceiling
- Sustained tolerability over multiple treatment cycles
- Possibility of establishing a reliable dosing regimen for future trials
While early-stage results are promising, the ongoing PK/PD analyses will be important for determining optimal dosing strategies and understanding the full therapeutic potential. The consistent inhibition of miR-10b across different dose levels suggests a robust therapeutic effect, which could differentiate TTX-MC138 in the competitive oncology landscape.
- No significant safety or dose limiting toxicities reported
- PK data from Cohorts 1 and 2 consistent with preclinical and Phase 0 trial results
Several patients in the first and second cohort remain on study for continued treatment, receiving additional doses of TTX-MC138 over cycles of approximately 28 days each. No significant safety or dose limiting toxicities have been reported in any of the trial's nine patients. Analyses of PK data and pharmacodynamic (PD) activity from Cohorts 1 and 2 is ongoing. To date, the analyses suggest that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode's previous Phase 0 clinical trial. Specifically, results from Cohort 1 confirmed the Phase 0 observation that TTX-MC138 shows evidence of pharmacodynamic activity in the presence of high baseline expression of miR-10b, reaching a
About TTX-MC138
TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. TransCode's 2023 Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.
About the Trial
TransCode's Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers. While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138. The trial comprises an initial dose-escalation stage followed by a dose-expansion stage. The primary objective of the dose-escalation stage is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion stage, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types selected based on preliminary results from the dose-escalation phase.
Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).
About TransCode Therapeutics
TransCode is a clinical-stage oncology company focused on treating metastatic disease. The company is committed to defeating cancer through the intelligent design and effective delivery of RNA therapeutics based on its proprietary TTX nanoparticle platform. The company's lead therapeutic candidate, TTX-MC138, is focused on treating metastatic tumors which overexpress microRNA-10b, a unique, well-documented biomarker of metastasis. In addition, TransCode has a portfolio of other first-in-class RNA therapeutic candidates designed to overcome the challenges of RNA delivery and thus unlock therapeutic access to a variety of novel genetic targets that could be relevant to treating a variety of cancers.
Forward-Looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, without limitation, statements concerning the timing, conduct and results of the Phase 1 clinical trial, statements about microRNAs and their involvement in cancer, and statements concerning the therapeutic potential of TransCode's TTX-MC138 and other therapeutic candidates. Any forward-looking statements in this press release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risks associated with drug discovery and development; the risk that the results of clinical trials will not be consistent with TransCode's preclinical studies or expectations or with results from previous clinical trials; risks associated with the conduct of clinical trials; risks associated with TransCode's financial condition and its need to obtain additional funding to support its business activities, including TransCode's ability to continue as a going concern; risks associated with the timing and outcome of TransCode's planned regulatory submissions; risks associated with obtaining, maintaining and protecting intellectual property; risks associated with TransCode's ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties; risks of competition from other companies developing products for similar uses; risks associated with TransCode's dependence on third parties; and risks associated with geopolitical events and pandemics, including the COVID-19 coronavirus and military actions. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause TransCode's actual results to differ from those contained in or implied by the forward-looking statements, see the section entitled "Risk Factors" in TransCode's Annual Report on Form 10-K for the year ended December 31, 2023, as well as discussions of potential risks, uncertainties and other important factors in any subsequent TransCode filings with the Securities and Exchange Commission. All information in this press release is as of the date of this release; TransCode undertakes no duty to update this information unless required by law.
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SOURCE TransCode Therapeutics, Inc.
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