TransCode Therapeutics Successfully Completes Initial Dosing of Patients in Cohort 4 of Phase 1a Clinical Trial; No Dose Limiting Toxicities Reported
TransCode Therapeutics (NASDAQ: RNAZ) has announced successful completion of initial dosing in Cohort 4 of its Phase 1a clinical trial for TTX-MC138. Key highlights include:
- 15 patients treated across 4 dose levels (0.8 mg/kg to 4.8 mg/kg)
- No significant safety or dose-limiting toxicities reported
- 10 patients remain on study with stable disease
- Two patients received 7 doses over ~7 months showing stable disease
- PK/PD data shows predictable dose-response relationship
- Evidence of miR-10b target engagement at 24 hours post-infusion in Cohorts 1 and 2
- Safety Review Committee approved expanded enrollment in Cohort 3
The positive results support advancement to the Phase 1b dose expansion portion of the trial.
TransCode Therapeutics (NASDAQ: RNAZ) ha annunciato il completamento con successo della somministrazione iniziale nel Gruppo 4 del suo trial clinico di Fase 1a per TTX-MC138. I punti salienti includono:
- 15 pazienti trattati su 4 livelli di dose (da 0,8 mg/kg a 4,8 mg/kg)
- Nessuna tossicità significativa o limitante a livello di dose riportata
- 10 pazienti ancora in studio con malattia stabile
- Due pazienti hanno ricevuto 7 dosi in circa 7 mesi mostrando malattia stabile
- I dati PK/PD mostrano una relazione dose-risposta prevedibile
- Evidenza di coinvolgimento del target miR-10b a 24 ore dall'infusione nei Gruppi 1 e 2
- Il Comitato di Revisione della Sicurezza ha approvato l'ampliamento dell'arruolamento nel Gruppo 3
I risultati positivi supportano il passaggio alla fase di espansione del dosaggio della Fase 1b dello studio.
TransCode Therapeutics (NASDAQ: RNAZ) ha anunciado la finalización exitosa de la dosificación inicial en la Cohorte 4 de su ensayo clínico de Fase 1a para TTX-MC138. Los puntos clave incluyen:
- 15 pacientes tratados en 4 niveles de dosis (de 0,8 mg/kg a 4,8 mg/kg)
- No se reportaron toxicidades significativas ni limitantes de dosis
- 10 pacientes permanecen en el estudio con enfermedad estable
- Dos pacientes recibieron 7 dosis durante aproximadamente 7 meses mostrando enfermedad estable
- Los datos PK/PD muestran una relación dosis-respuesta predecible
- Evidencia de compromiso del objetivo miR-10b a las 24 horas post-infusión en las Cohortes 1 y 2
- El Comité de Revisión de Seguridad aprobó la ampliación del reclutamiento en la Cohorte 3
Los resultados positivos apoyan el avance a la fase de expansión de dosis de la Fase 1b del ensayo.
TransCode Therapeutics (NASDAQ: RNAZ)가 TTX-MC138에 대한 1a상 임상시험 4번째 코호트의 초기 투여를 성공적으로 완료했다고 발표했습니다. 주요 내용은 다음과 같습니다:
- 4가지 용량 수준(0.8 mg/kg부터 4.8 mg/kg까지)에서 15명의 환자 치료
- 중대한 안전 문제나 용량 제한 독성 없음 보고
- 10명의 환자가 안정된 질환 상태로 연구에 계속 참여 중
- 2명의 환자가 약 7개월 동안 7회 투여를 받아 안정된 질환 상태 유지
- PK/PD 데이터가 예측 가능한 용량-반응 관계를 나타냄
- 1, 2 코호트에서 투여 24시간 후 miR-10b 표적 관여 증거 확인
- 안전성 검토 위원회가 3코호트의 모집 확대 승인
이러한 긍정적인 결과는 1b상 용량 확장 단계로의 진행을 뒷받침합니다.
TransCode Therapeutics (NASDAQ : RNAZ) a annoncé la réussite de la première administration dans la cohorte 4 de son essai clinique de phase 1a pour TTX-MC138. Les points clés sont :
- 15 patients traités à travers 4 niveaux de dose (de 0,8 mg/kg à 4,8 mg/kg)
- Aucune toxicité significative ou limitante liée à la dose rapportée
- 10 patients restent dans l'étude avec une maladie stable
- Deux patients ont reçu 7 doses sur environ 7 mois, montrant une maladie stable
- Les données PK/PD montrent une relation dose-réponse prévisible
- Preuve d'engagement de la cible miR-10b à 24 heures post-infusion dans les cohortes 1 et 2
- Le comité de revue de sécurité a approuvé l'élargissement du recrutement dans la cohorte 3
Ces résultats positifs soutiennent la progression vers la phase d'expansion de dose de la phase 1b de l'essai.
TransCode Therapeutics (NASDAQ: RNAZ) hat den erfolgreichen Abschluss der Erstgabe in Kohorte 4 seiner Phase-1a-Studie für TTX-MC138 bekannt gegeben. Wichtige Highlights sind:
- 15 Patienten wurden in 4 Dosierungsstufen behandelt (0,8 mg/kg bis 4,8 mg/kg)
- Keine signifikanten Sicherheitsprobleme oder dosislimitierenden Toxizitäten berichtet
- 10 Patienten verbleiben mit stabiler Erkrankung in der Studie
- Zwei Patienten erhielten über ca. 7 Monate 7 Dosen und zeigten stabile Erkrankung
- PK/PD-Daten zeigen eine vorhersehbare Dosis-Wirkungs-Beziehung
- Nachweis einer miR-10b-Zielbindung 24 Stunden nach Infusion in Kohorten 1 und 2
- Das Sicherheitsüberprüfungskomitee genehmigte eine erweiterte Einschreibung in Kohorte 3
Die positiven Ergebnisse unterstützen den Übergang zur Dosisexpansionsphase der Phase 1b-Studie.
- No significant safety or dose-limiting toxicities reported across all 15 patients
- 10 patients showing stable disease with no progression
- Two longest-treated patients maintained stable disease after 7 doses over 7 months
- PK/PD data demonstrates predictable dose-response relationship consistent with preclinical results
- Successful miR-10b target engagement observed at 24 hours post-infusion
- None.
Insights
TransCode reports positive safety data and target engagement in Phase 1a trial of TTX-MC138, advancing to Phase 1b with stable disease in longest-treated patients.
The completion of initial dosing across all four cohorts in TransCode's Phase 1a trial for TTX-MC138 represents meaningful progress for this RNA therapeutic. The clean safety profile is particularly noteworthy - no dose-limiting toxicities observed across 15 patients treated at escalating doses ranging from 0.8 mg/kg to 4.8 mg/kg. This favorable tolerability is significant for RNA therapeutics, which historically face delivery and toxicity challenges.
The pharmacodynamic data provides crucial molecular evidence of drug activity. The demonstration of miR-10b target engagement at 24 hours post-infusion even at the lower dose cohorts (0.8 mg/kg and 1.6 mg/kg) suggests the drug is successfully reaching its intended target. MicroRNA-10b is implicated in cancer metastasis, making this target engagement an important signal for the drug's mechanism of action.
While Phase 1a trials primarily assess safety rather than efficacy, the clinical observations provide early signals worth monitoring. Ten patients remain on treatment with no evidence of disease progression. The two longest-treated patients have received seven doses over approximately seven months with stable disease. Stable disease in this context means tumors neither growing nor shrinking significantly - a meaningful outcome in cancer treatment, though not the same as tumor regression.
The Safety Review Committee's approval to both advance to Cohort 4 and expand enrollment in Cohort 3 reflects confidence in the safety profile while appropriately gathering additional data. The planned advancement to the Phase 1b expansion portion indicates the company has sufficient evidence to justify continued development.
These results collectively represent an important de-risking milestone for TransCode's RNA delivery platform. The combination of clean safety data, evidence of target engagement, and stable disease in long-term patients provides a foundation for the upcoming Phase 1b portion, which will further evaluate both safety and preliminary anti-tumor activity in a larger patient population.
- 15 patients treated across four escalating dose levels of TTX-MC138
- No significant safety or dose limiting toxicities reported
- 10 patients remain on study with no evidence of disease progression
- PD analysis at 24 hours post-dosing provides evidence of miR-10b target engagement
Ten patients remain on study for continued treatment, receiving an additional dose of TTX-MC138 during each treatment cycle every 28 days, and may remain on study absent any significant safety observations or disease progression. Two patients who have remained on study the longest have received so far seven doses of TTX-MC138 over approximately seven months and have demonstrated stable disease. The 10 patients currently on study have shown no disease progression. Further, no significant safety or dose limiting toxicities have been reported in any of the trial's 15 patients. Analysis of PK 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. Specifically, preliminary PK and pharmacodynamic (PD) data follow a predictable dose-response relationship. Analysis of PD activity from cycle 1 treatments in Cohorts 1 and 2, treated with doses of 0.8 mg/kg and 1.6 mg/kg, respectively, demonstrates miR-10b target engagement at 24 hours post-infusion.
The observed tolerability profile and the available PK/PD results thus far support advancement of the clinical trial to further evaluate safety and potential anti-tumor activity of TTX-MC138 in the planned dose expansion (Phase 1b) portion of the trial.
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 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 and at a certain dose level 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 TransCode's 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, 2024, 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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