TransCode Therapeutics Announces Initial Dosing in Fourth Cohort of Phase 1 Clinical Trial with TTX-MC138
TransCode Therapeutics (NASDAQ: RNAZ) has announced significant progress in its Phase 1 clinical trial of TTX-MC138, their lead therapeutic candidate designed to inhibit microRNA-10b in metastatic cancer. The first patient in Cohort 4 has received initial dosing, with two additional patients scheduled for treatment.
Key highlights include:
- Ten patients treated across four cohorts at escalating dose levels
- No significant safety or dose-limiting toxicities reported
- Seven patients remain on study for continued treatment
- Cohort 4 dosing is approximately 50% higher than Cohort 3
- Ongoing data analysis from Cohorts 1-3 shows pharmacokinetic and pharmacodynamic profiles consistent with preclinical results and Phase 0 trial
The Safety Review Committee has approved additional patient enrollment in Cohort 3 to further establish the safety profile of TTX-MC138.
TransCode Therapeutics (NASDAQ: RNAZ) ha annunciato progressi significativi nel suo studio clinico di Fase 1 per TTX-MC138, il loro principale candidato terapeutico progettato per inibire il microRNA-10b nel cancro metastatico. Il primo paziente nel Cohorte 4 ha ricevuto la somministrazione iniziale, con altri due pazienti programmati per il trattamento.
I punti salienti includono:
- Dieci pazienti trattati in quattro coorti a livelli di dose crescenti
- Nessuna tossicità significativa per la sicurezza o limitante per la dose riportata
- Sette pazienti rimangono nello studio per un trattamento continuato
- La dose della Cohorte 4 è circa il 50% superiore a quella della Cohorte 3
- L'analisi dei dati in corso delle Coorti 1-3 mostra profili farmacocinetici e farmacodinamici coerenti con i risultati preclinici e lo studio di Fase 0
Il Comitato di Revisione della Sicurezza ha approvato l'arruolamento di ulteriori pazienti nella Cohorte 3 per stabilire ulteriormente il profilo di sicurezza di TTX-MC138.
TransCode Therapeutics (NASDAQ: RNAZ) ha anunciado avances significativos en su ensayo clínico de Fase 1 para TTX-MC138, su principal candidato terapéutico diseñado para inhibir el microARN-10b en cáncer metastásico. El primer paciente en Cohorte 4 ha recibido la dosis inicial, con otros dos pacientes programados para el tratamiento.
Los aspectos destacados incluyen:
- Diez pacientes tratados en cuatro cohortes a niveles de dosis en aumento
- No se han reportado toxicidades significativas para la seguridad o limitantes de dosis
- Siete pacientes permanecen en el estudio para tratamiento continuado
- La dosis de la Cohorte 4 es aproximadamente un 50% más alta que la de la Cohorte 3
- El análisis de datos en curso de las Cohortes 1-3 muestra perfiles farmacocinéticos y farmacodinámicos consistentes con los resultados preclínicos y el ensayo de Fase 0
El Comité de Revisión de Seguridad ha aprobado la inclusión de pacientes adicionales en la Cohorte 3 para establecer más el perfil de seguridad de TTX-MC138.
TransCode Therapeutics (NASDAQ: RNAZ)는 전이성 암에서 microRNA-10b를 억제하도록 설계된 주요 치료 후보인 TTX-MC138의 1상 임상 시험에서 중요한 진전을 발표했습니다. 코호트 4의 첫 번째 환자가 초기 투약을 받았으며, 추가로 두 명의 환자가 치료를 받을 예정입니다.
주요 하이라이트는 다음과 같습니다:
- 증가하는 용량 수준에서 네 개의 코호트에서 치료된 10명의 환자
- 보고된 안전성 또는 용량 제한 독성이 없음
- 7명의 환자가 지속적인 치료를 위해 연구에 남아 있음
- 코호트 4의 투여량은 코호트 3보다 약 50% 더 높음
- 코호트 1-3의 지속적인 데이터 분석은 전임상 결과 및 0상 시험과 일치하는 약리학적 동태 및 약리학적 동작 프로필을 보여줌
안전성 검토 위원회는 TTX-MC138의 안전성 프로필을 더욱 확립하기 위해 코호트 3에 추가 환자 등록을 승인했습니다.
TransCode Therapeutics (NASDAQ: RNAZ) a annoncé des progrès significatifs dans son essai clinique de phase 1 pour TTX-MC138, leur principal candidat thérapeutique conçu pour inhiber le microARN-10b dans le cancer métastatique. Le premier patient de la Cohorte 4 a reçu une première dose, avec deux patients supplémentaires prévus pour le traitement.
Les points clés incluent:
- Dix patients traités dans quatre cohortes à des niveaux de dose croissants
- Aucune toxicité significative pour la sécurité ou limitante pour la dose signalée
- Sept patients restent dans l'étude pour un traitement continu
- La dose de la Cohorte 4 est environ 50 % plus élevée que celle de la Cohorte 3
- L'analyse des données en cours des Cohortes 1-3 montre des profils pharmacocinétiques et pharmacodynamiques cohérents avec les résultats précliniques et l'essai de phase 0
Le Comité de Révision de la Sécurité a approuvé l'inscription de patients supplémentaires dans la Cohorte 3 pour établir davantage le profil de sécurité de TTX-MC138.
TransCode Therapeutics (NASDAQ: RNAZ) hat bedeutende Fortschritte in seiner Phase-1-Studie zu TTX-MC138 bekannt gegeben, dem führenden therapeutischen Kandidaten, der darauf abzielt, das microRNA-10b bei metastasierendem Krebs zu hemmen. Der erste Patient in Kohorte 4 hat die erste Dosis erhalten, zwei weitere Patienten sind für die Behandlung eingeplant.
Wichtige Highlights sind:
- Zehn Patienten wurden in vier Kohorten mit steigenden Dosen behandelt
- Keine signifikanten Sicherheits- oder dosislimitierenden Toxizitäten berichtet
- Sieben Patienten bleiben für eine fortgesetzte Behandlung in der Studie
- Die Dosis in Kohorte 4 ist etwa 50 % höher als in Kohorte 3
- Die laufende Datenanalyse der Kohorten 1-3 zeigt pharmakokinetische und pharmakodynamische Profile, die mit den präklinischen Ergebnissen und der Phase-0-Studie übereinstimmen
Das Sicherheitsprüfungskomitee hat die zusätzliche Patientenaufnahme in Kohorte 3 genehmigt, um das Sicherheitsprofil von TTX-MC138 weiter zu etablieren.
- No safety issues or dose-limiting toxicities reported across all cohorts
- 70% patient retention rate with 7 out of 10 patients continuing treatment
- Successful dose escalation to 50% higher levels in Cohort 4
- Pharmacokinetic/pharmacodynamic profiles align with preclinical expectations
- None.
Insights
TransCode's Phase I trial progression for TTX-MC138 represents a significant milestone in the clinical development pathway. The successful dosing of the first patient in Cohort 4 with a 50% higher dose than Cohort 3 demonstrates orderly dose escalation without encountering safety barriers. The absence of dose-limiting toxicities across ten treated patients is particularly noteworthy in RNA therapeutic development, where safety concerns often emerge during dose escalation.
The retention of seven out of ten patients continuing treatment suggests both tolerable safety and absence of disease progression, though at this Phase I stage, efficacy conclusions would be premature. The Safety Review Committee's dual approval to both advance to Cohort 4 and expand enrollment in Cohort 3 indicates confidence in the emerging safety profile.
The preliminary pharmacokinetic and pharmacodynamic data consistency with preclinical and Phase 0 results is technically significant as it suggests the drug is behaving predictably in humans. For an RNA therapeutic targeting microRNA-10b, which plays a critical role in metastasis pathways, establishing this PK/PD relationship is an essential foundation before efficacy assessment.
This progression maintains TransCode's timeline toward potential dose expansion where initial efficacy signals might emerge. However, as characteristic of Phase I trials, the primary objective remains safety characterization rather than efficacy demonstration.
- First patient in Cohort four has been treated with TTX-MC138
- Ten patients have been treated with TTX-MC138 at escalating dose levels
- Additional patients being evaluated for eligibility for expanded enrollment
- No significant safety or dose limiting toxicities have been reported
Under the clinical protocol, patients may remain on study absent safety events or disease progression. Of the ten patients treated with TTX-MC138 in the first four cohorts, seven remain on study for continued treatment as there have been no dose limiting toxicities or disease progression in these patients. In addition to approving opening the fourth cohort, the Safety Review Committee approved enrollment of additional patients in Cohort 3 to build upon the safety profile of TTX-MC138. Further, data analysis of both pharmacokinetic (PK) and pharmacodynamic (PD) activity from Cohorts 1, 2 and 3 is ongoing and suggests that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode's Phase 0 clinical trial.
"Commencement of treatment in Cohort 4 has been our vision for evaluating the potential of TTX-MC138 at multiple dose levels. Cohort 4 builds upon our safety, PK/PD and exploratory data and is an important element of our clinical development strategy. We believe this milestone will inform the dose expansion stage of the clinical trial and may allow us to obtain initial evidence of clinical activity as the program continues to advance," commented Sue Duggan, TransCode's Senior Vice President of Operations. Duggan added, "Enrollment into the study continues based on the cumulative safety data review. Additional Cohort 4 patients are scheduled for treatment in Cohort 4 for treatment with TTX-MC138 while preliminary data analysis continues."
About TTX-MC138
TTX-MC138 is a first-in-class therapeutic candidate that targets microRNA-10b, a micro-RNA widely believed to be a driver of metastatic disease. 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 phase followed by a dose-expansion phase. The primary objective of the dose-escalation phase is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion phase, 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. 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 pre-clinical 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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