TransCode Therapeutics Announces Safety Review Committee Approval of Opening Third Cohort and Preliminary Results from First Cohort in Phase 1 TTX-MC138 Clinical Trial
TransCode Therapeutics (NASDAQ: RNAZ) announced the Safety Review Committee's unanimous approval to open the third cohort in its Phase 1 clinical trial of TTX-MC138, following favorable safety data from Cohort 2. The trial evaluates TTX-MC138, designed to inhibit microRNA-10b, which is important in metastatic cancer progression.
Key findings include: no significant safety or dose-limiting toxicities reported in Cohort 2; Cohort 3 will receive approximately double the dose of Cohort 2; pharmacokinetic and pharmacodynamic data from Cohort 1 align with preclinical and Phase 0 results, showing 66% inhibition of miR-10b at 24 hours post-infusion. The concentration of TTX-MC138 in human blood plasma exceeded levels achieved in nonclinical studies.
TransCode Therapeutics (NASDAQ: RNAZ) ha annunciato l'approvazione unanime del Comitato per la Revisione della Sicurezza per aprire la terza coorte del suo studio clinico di Fase 1 per TTX-MC138, dopo i favorevoli dati di sicurezza provenienti dalla Coorte 2. Lo studio valuta il TTX-MC138, progettato per inibire il microRNA-10b, importante nella progressione del cancro metastatico.
I risultati chiave includono: nessuna tossicità significativa o limitante la dose segnalata nella Coorte 2; la Coorte 3 riceverà circa il doppio della dose della Coorte 2; i dati farmacocinetici e farmacodinamici della Coorte 1 sono in linea con i risultati preclinici e della Fase 0, mostrando un'inibizione del 66% di miR-10b a 24 ore dopo l'infusione. La concentrazione di TTX-MC138 nel plasma umano ha superato i livelli raggiunti negli studi non clinici.
TransCode Therapeutics (NASDAQ: RNAZ) anunció la aprobación unánime del Comité de Revisión de Seguridad para abrir la tercera cohorte en su ensayo clínico de Fase 1 para TTX-MC138, tras los favorables datos de seguridad de la Cohorte 2. El ensayo evalúa TTX-MC138, diseñado para inhibir el microARN-10b, que es importante en la progresión del cáncer metastásico.
Los hallazgos clave incluyen: no se informaron toxicidades significativas o limitantes de dosis en la Cohorte 2; la Cohorte 3 recibirá aproximadamente el doble de la dosis de la Cohorte 2; los datos farmacocinéticos y farmacodinámicos de la Cohorte 1 están alineados con los resultados preclínicos y de la Fase 0, mostrando una inhibición del 66% de miR-10b a las 24 horas después de la infusión. La concentración de TTX-MC138 en plasma sanguíneo humano superó los niveles alcanzados en estudios no clínicos.
TransCode Therapeutics (NASDAQ: RNAZ)는 TTX-MC138의 1상 임상 시험에서 제3 코호트를 열기 위한 안전성 검토 위원회의 만장일치 승인을 발표했습니다. 이는 2코호트에서의 안전성 데이터가 호의적이었기 때문입니다. 이 임상 연구는 전이성 암의 진행에 중요한 마이크로RNA-10b를 억제하기 위해 설계된 TTX-MC138을 평가합니다.
주요 발견 사항은 다음과 같습니다: 2 코호트에서 유의미한 안전성 문제나 용량 제한 독성이 보고되지 않았습니다; 3코호트는 2코호트의 약 두 배의 용량을 받을 것입니다; 1코호트의 약물 동태 및 약리학적 데이터는 전임상 및 0단계 결과와 일치하며, 주입 후 24시간에 miR-10b의 66% 억제를 보여줍니다. 인간 혈장 내 TTX-MC138의 농도는 비임상 연구에서 달성된 수준을 초과했습니다.
TransCode Therapeutics (NASDAQ: RNAZ) a annoncé l'approbation unanime du Comité de Revue de Sécurité pour ouvrir la troisième cohorte de son essai clinique de Phase 1 sur TTX-MC138, suite aux données de sécurité favorables de la Cohorte 2. L'essai évalue TTX-MC138, conçu pour inhiber le microARN-10b, qui est important dans la progression des cancers métastatiques.
Les principales conclusions incluent : aucune toxicité significative ou limitant la dose n'a été signalée dans la Cohorte 2 ; la Cohorte 3 recevra environ le double de la dose de la Cohorte 2 ; les données pharmacocinétiques et pharmacodynamiques de la Cohorte 1 s'alignent sur les résultats précliniques et de Phase 0, montrant une inhibition de 66 % de miR-10b 24 heures après l'infusion. La concentration de TTX-MC138 dans le plasma sanguin humain a dépassé les niveaux atteints dans les études non cliniques.
TransCode Therapeutics (NASDAQ: RNAZ) gab die einstimmige Genehmigung des Sicherheitsexpertenausschusses bekannt, die dritte Kohorte in seiner klinischen Phase-1-Studie zu TTX-MC138 zu eröffnen, nachdem günstige Sicherheitsdaten aus Kohorte 2 vorlagen. Die Studie bewertet TTX-MC138, das zur Hemmung von MikroRNA-10b entwickelt wurde, welche für die fortschreitende Metastasierung von Krebs wichtig ist.
Wichtige Ergebnisse beinhalten: Keine signifikanten Sicherheits- oder dosisbegrenzenden Toxizitäten wurden in Kohorte 2 berichtet; Kohorte 3 wird etwa die doppelte Dosis von Kohorte 2 erhalten; die pharmakokinetischen und pharmakodynamischen Daten aus Kohorte 1 stimmen mit den präklinischen und Phase-0-Ergebnissen überein, wobei eine Hemmung von 66 % von miR-10b 24 Stunden nach der Infusion gezeigt wurde. Die Konzentration von TTX-MC138 im menschlichen Blutplasma überstieg die in nicht-klinischen Studien erreichten Werte.
- No safety or dose-limiting toxicities reported in Cohort 2
- Approval to proceed with higher dosing in Cohort 3
- 66% inhibition of miR-10b achieved at 24 hours post-infusion
- Higher blood plasma concentration in humans compared to nonclinical studies
- Multiple patients from first and second cohorts continue treatment
- None.
Insights
The positive safety data and preliminary PK/PD results from TransCode's Phase 1 trial of TTX-MC138 mark meaningful progress in RNA therapeutics for cancer treatment. The 66% inhibition of miR-10b at 24 hours post-infusion and higher plasma concentrations compared to preclinical studies suggest promising drug behavior. The successful completion of two cohorts without significant safety issues, coupled with the SRC's approval for dose escalation to Cohort 3, indicates a well-tolerated safety profile thus far. The retention of patients from earlier cohorts for continued treatment further supports the drug's tolerability. While early-stage, these results validate the delivery mechanism and provide initial evidence of TTX-MC138's potential in targeting metastatic cancers through miR-10b inhibition.
For a micro-cap biotech with a
Approval given after Safety Review Committee (SRC) review of safety data from the three patients comprising Cohort 2
- No significant safety or dose limiting toxicities reported in Cohort 2
- New patients currently being evaluated for eligibility in Cohort 3
- PK and PD data from Cohort 1 patients consistent with preclinical and Phase 0 trial results
Several patients in the first and second cohort remain on study for continued treatment. No significant safety or dose limiting toxicities have been reported. Analysis of Cohort 1 data for pharmacokinetic (PK) and pharmacodynamic (PD) activity is ongoing and to date suggests that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from the 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
"An SRC is a group of clinicians and experts that oversee patient safety during the conduct of a clinical trial. The SRC determines whether and how a study should proceed, including dose escalation and de-escalation decisions in accordance with the study design. The recommendations of the SRC are used to decide whether a clinical trial should be continued as designed, changed, or terminated," 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 patients may now be screened and scheduled in Cohort 3 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 microRNA 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 is developing 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 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.
For more information, please contact:
TransCode Therapeutics, Inc.
Tania Montgomery-Hammon, VP of Business Development
tania.montgomery@transcodetherapeutics.com
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SOURCE TransCode Therapeutics, Inc.
FAQ
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