TransCode Therapeutics Announces Safety Review Committee Approval to Open Fourth Cohort in Phase I/II Clinical Trial
TransCode Therapeutics (NASDAQ: RNAZ) has received approval from the Safety Review Committee (SRC) to proceed with Cohort 4 in its Phase I/II clinical trial for TTX-MC138, a first-in-class RNA therapeutic for metastatic cancers. The approval follows favorable safety data from Cohort 3, with no significant safety issues or dose-limiting toxicities reported across all three previous cohorts.
Key highlights:
- 6 out of 9 treated patients remain on study, with one patient receiving 7 doses over approximately 7 months
- Cohort 4 will receive a dose approximately 50% higher than Cohort 3
- SRC approved additional patient enrollment in Cohort 3
- Pharmacokinetic and pharmacodynamic data from Cohorts 1 and 2 align with preclinical and Phase 0 trial results
- Preliminary analysis suggests dose levels of 0.8-1.6 mg/kg could represent an efficacious range
TransCode Therapeutics (NASDAQ: RNAZ) ha ricevuto l'approvazione dal Safety Review Committee (SRC) per procedere con il Coorte 4 nel suo studio clinico di Fase I/II per TTX-MC138, una terapia RNA innovativa per i tumori metastatici. L'approvazione è stata concessa dopo dati di sicurezza favorevoli provenienti dal Coorte 3, senza problemi di sicurezza significativi o tossicità limitanti alla dose segnalati in tutti e tre i coorti precedenti.
Principali punti salienti:
- 6 dei 9 pazienti trattati rimangono nello studio, con un paziente che ha ricevuto 7 dosi in circa 7 mesi
- Il Coorte 4 riceverà una dose circa il 50% più alta rispetto al Coorte 3
- Il SRC ha approvato l'arruolamento di ulteriori pazienti nel Coorte 3
- I dati farmacocinetici e farmacodinamici dei Coorti 1 e 2 sono in linea con i risultati preclinici e dello studio di Fase 0
- L'analisi preliminare suggerisce che i livelli di dose di 0.8-1.6 mg/kg potrebbero rappresentare un intervallo efficace
TransCode Therapeutics (NASDAQ: RNAZ) ha recibido la aprobación del Safety Review Committee (SRC) para proceder con el Cohorte 4 en su ensayo clínico de Fase I/II para TTX-MC138, una terapia de RNA innovadora para cánceres metastásicos. La aprobación sigue a datos de seguridad favorables del Cohorte 3, sin problemas de seguridad significativos ni toxicidades limitantes por dosis reportadas en los tres cohortes anteriores.
Puntos destacados:
- 6 de los 9 pacientes tratados permanecen en el estudio, con un paciente recibiendo 7 dosis durante aproximadamente 7 meses
- El Cohorte 4 recibirá una dosis aproximadamente un 50% más alta que el Cohorte 3
- El SRC aprobó la inclusión de más pacientes en el Cohorte 3
- Los datos farmacocinéticos y farmacodinámicos de los Cohortes 1 y 2 están alineados con los resultados preclínicos y del ensayo de Fase 0
- El análisis preliminar sugiere que los niveles de dosis de 0.8-1.6 mg/kg podrían representar un rango eficaz
TransCode Therapeutics (NASDAQ: RNAZ)는 전이성 암을 위한 첫 번째 RNA 치료제인 TTX-MC138의 임상 시험 1/2단계에서 Cohort 4를 진행할 수 있도록 Safety Review Committee (SRC)의 승인을 받았습니다. 이 승인은 Cohort 3에서의 안전성 데이터가 긍정적이었고, 이전의 세 개 코호트에서 중대한 안전성 문제나 용량 제한 독성이 보고되지 않았기 때문에 이루어졌습니다.
주요 하이라이트:
- 치료받은 9명의 환자 중 6명이 연구에 남아 있으며, 한 환자는 약 7개월 동안 7회의 투여를 받았습니다.
- Cohort 4는 Cohort 3보다 약 50% 높은 용량을 받을 것입니다.
- SRC는 Cohort 3에 추가 환자 등록을 승인했습니다.
- Cohort 1 및 2의 약리학적 데이터는 전임상 및 0단계 시험 결과와 일치합니다.
- 예비 분석에 따르면 0.8-1.6 mg/kg의 용량 수준이 효과적인 범위를 나타낼 수 있습니다.
TransCode Therapeutics (NASDAQ: RNAZ) a reçu l'approbation du Safety Review Committee (SRC) pour procéder avec la Cohorte 4 de son essai clinique de Phase I/II pour TTX-MC138, une thérapie par ARN de première classe pour les cancers métastatiques. L'approbation fait suite à des données de sécurité favorables provenant de la Cohorte 3, sans problèmes de sécurité significatifs ni toxicités limitantes de dose signalés dans les trois cohorte précédents.
Points clés :
- 6 des 9 patients traités restent dans l'étude, avec un patient recevant 7 doses sur environ 7 mois
- La Cohorte 4 recevra une dose environ 50 % plus élevée que celle de la Cohorte 3
- Le SRC a approuvé l'inscription de patients supplémentaires dans la Cohorte 3
- Les données pharmacocinétiques et pharmacodynamiques des Cohortes 1 et 2 sont en accord avec les résultats précliniques et de l'essai de Phase 0
- Une analyse préliminaire suggère que des niveaux de dose de 0,8 à 1,6 mg/kg pourraient représenter une plage efficace
TransCode Therapeutics (NASDAQ: RNAZ) hat die Genehmigung des Safety Review Committee (SRC) erhalten, um mit Kohorte 4 in seiner klinischen Studie der Phase I/II für TTX-MC138, eine neuartige RNA-Therapie für metastasierende Krebserkrankungen, fortzufahren. Die Genehmigung folgt auf positive Sicherheitsdaten aus Kohorte 3, bei denen keine signifikanten Sicherheitsprobleme oder dosislimitierende Toxizitäten in den drei vorherigen Kohorten berichtet wurden.
Wichtige Höhepunkte:
- 6 der 9 behandelten Patienten bleiben in der Studie, wobei ein Patient über einen Zeitraum von etwa 7 Monaten 7 Dosen erhalten hat
- Kohorte 4 wird eine Dosis erhalten, die etwa 50% höher ist als die von Kohorte 3
- SRC genehmigte die Aufnahme zusätzlicher Patienten in Kohorte 3
- Pharmakokinetische und pharmakodynamische Daten aus Kohorte 1 und 2 stimmen mit den präklinischen und Phase-0-Studienergebnissen überein
- Vorläufige Analysen deuten darauf hin, dass Dosen von 0,8-1,6 mg/kg einen wirksamen Bereich darstellen könnten
- No safety issues or dose-limiting toxicities reported in first three cohorts
- Strong patient retention with 6 out of 9 patients continuing treatment
- Pharmacokinetic/pharmacodynamic data consistent with preclinical and Phase 0 results
- Evidence of drug activity in patient blood confirmed
- Potential efficacious dose range identified (0.8-1.6 mg/kg)
- None.
Insights
TransCode's advancement to Cohort 4 in their Phase I/II trial represents a standard but necessary milestone in the clinical development of TTX-MC138. The absence of dose-limiting toxicities across all three previous cohorts is encouraging for an RNA therapeutic targeting metastatic cancer, allowing for the planned 50% dose increase in Cohort 4.
The retention of 6 out of 9 patients from earlier cohorts for continued treatment signals favorable tolerability. The longest-treated patient having received 7 monthly doses without safety issues or disease progression provides preliminary evidence of acceptable safety profile at current dose levels.
The pharmacokinetic/pharmacodynamic (PK/PD) data consistency between the clinical trial and both preclinical studies and Phase 0 results is scientifically relevant. This consistency suggests the drug is behaving as expected in humans, with evidence of activity in patient blood. The preliminary suggestion that doses of 0.8-1.6 mg/kg could represent an efficacious range provides direction for future dosing decisions.
The parallel strategy of opening Cohort 4 while expanding enrollment in Cohort 3 is a smart approach to gather more comprehensive data. However, it's important to recognize that these are safety findings only. While necessary for development, efficacy evidence remains pending and will be the critical determinant of clinical value.
TransCode's progression to the fourth dosing cohort represents a typical risk-reduction event for a small-cap biotech with a
The absence of safety concerns across three dosing cohorts derisks the near-term development path for TTX-MC138, their first-in-class RNA therapeutic using antisense technology. The company's ability to dose-escalate by
The consistency between clinical PK/PD data and both preclinical and Phase 0 results validates their delivery technology approach, which is critical for RNA therapeutics where delivery remains a key challenge. However, the suggestion that doses of 0.8-1.6 mg/kg "could represent an efficacious range" should be viewed cautiously as it's based on preliminary PK analysis, not confirmed efficacy signals.
For investors, this represents continued execution rather than a transformative catalyst. While the safety profile appears favorable, the true value-creating data will come from efficacy signals in later development stages. The expansion of patient enrollment indicates confidence in the program's progress but does not yet provide the efficacy validation needed for significant value reassessment.
- Clinical trial Safety Review Committee (SRC) approved opening of Cohort 4 based on favorable safety data from the three patients comprising Cohort 3
- No significant safety or dose limiting toxicities reported in Cohorts 1, 2, or 3
- Patients currently being evaluated for Cohort 4 eligibility
- PK and PD data from patients in Cohort 1 and Cohort 2 consistent with preclinical results and results from Phase 0 clinical trial
Under the clinical protocol, patients may remain on study absent safety events or disease progression. Out of 9 patients treated with TTX-MC138 in the first three cohorts, 6 remain on study for continued treatment since there have been no dose limiting toxicities or disease progression with these patients. The patient that has remained on study the longest has, to date, received 7 doses each approximately 28 days apart over the approximately 7 months that this patient has been on study. In addition to approving opening the fourth cohort, the SRC approved enrollment of additional patients in Cohort 3 to build upon the safety profile of TTX-MC138. Further, Cohort 1 and 2 data analysis for both pharmacokinetic (PK) and pharmacodynamic (PD) activity 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. Preliminary results from Cohort 2 confirmed observations from the Phase 0 trial and from Cohort 1 that the drug candidate shows evidence of activity in patient blood. Preliminary PK analysis suggests that dose levels 0.8-1.6 mg/kg, the levels administered to Cohorts 1 and 2, could represent an efficacious range.
"SRC approval to open the fourth cohort and expand enrollment in Cohort 3 is an important advancement for the clinical trial. It will provide an opportunity to obtain additional safety and PK/PD data, inform the dose expansion stage of the clinical trial and may allow us to obtain initial evidence of clinical activity" commented Sue Duggan, TransCode's Senior Vice President of Operations. Duggan added, "Enrollment into the study continues based on the cumulative safety data review. Eligible subjects may now be screened and scheduled in Cohort 4 for treatment with the next dose level of 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 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 stage.
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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