TransCode Therapeutics Reports Further Progress on Phase 1a Clinical Trial with No Dose Limiting Toxicities Reported in Patients with Metastatic Cancer
TransCode Therapeutics (NASDAQ: RNAZ) reported positive progress in its Phase 1a clinical trial of TTX-MC138, a first-in-class therapeutic targeting miR-10b for metastatic cancer treatment. Key findings include:
- 13 patients received treatment across 4 dose levels (0.8 mg/kg to 4.8 mg/kg)
- No significant safety or dose-limiting toxicities reported
- Two patients maintained stable disease for ~7 months
- 8 patients continue treatment in 28-day cycles
- PK/PD analysis shows promising results with miR-10b target engagement in 5 out of 6 analyzed patients
The positive tolerability profile and PK/PD results support advancement to the Phase 1b expansion portion of the trial.
TransCode Therapeutics (NASDAQ: RNAZ) ha riportato progressi positivi nella sua sperimentazione clinica di Fase 1a di TTX-MC138, un trattamento innovativo che mira a miR-10b per la cura del cancro metastatico. I principali risultati includono:
- 13 pazienti trattati con 4 livelli di dosaggio (da 0,8 mg/kg a 4,8 mg/kg)
- Nessuna tossicità significativa o dose-limitante segnalata
- Due pazienti hanno mantenuto una malattia stabile per circa 7 mesi
- 8 pazienti continuano il trattamento con cicli di 28 giorni
- L'analisi PK/PD mostra risultati promettenti con il coinvolgimento del target miR-10b in 5 pazienti su 6 analizzati
Il profilo di tollerabilità positivo e i risultati PK/PD supportano il passaggio alla fase 1b di espansione dello studio.
TransCode Therapeutics (NASDAQ: RNAZ) informó avances positivos en su ensayo clínico de Fase 1a con TTX-MC138, un tratamiento innovador dirigido a miR-10b para el tratamiento del cáncer metastásico. Los hallazgos clave incluyen:
- 13 pacientes recibieron tratamiento en 4 niveles de dosis (de 0,8 mg/kg a 4,8 mg/kg)
- No se reportaron toxicidades significativas ni limitantes de dosis
- Dos pacientes mantuvieron la enfermedad estable durante aproximadamente 7 meses
- 8 pacientes continúan el tratamiento en ciclos de 28 días
- El análisis PK/PD muestra resultados prometedores con la interacción del objetivo miR-10b en 5 de 6 pacientes analizados
El perfil positivo de tolerabilidad y los resultados PK/PD respaldan avanzar a la fase 1b de expansión del ensayo.
TransCode Therapeutics (NASDAQ: RNAZ)는 전이성 암 치료를 위한 miR-10b 표적의 혁신적 치료제인 TTX-MC138의 1a상 임상시험에서 긍정적인 진전을 보고했습니다. 주요 결과는 다음과 같습니다:
- 13명의 환자가 4가지 용량 수준(0.8 mg/kg에서 4.8 mg/kg)에서 치료를 받음
- 중대한 안전 문제나 용량 제한 독성 없음
- 2명의 환자가 약 7개월간 안정된 질병 상태 유지
- 8명의 환자가 28일 주기 치료 계속 진행 중
- PK/PD 분석 결과 6명 중 5명에서 miR-10b 타깃 결합이 유망하게 나타남
우수한 내약성 프로필과 PK/PD 결과는 1b상 확장 시험으로의 진행을 뒷받침합니다.
TransCode Therapeutics (NASDAQ : RNAZ) a annoncé des progrès positifs dans son essai clinique de phase 1a de TTX-MC138, un traitement innovant ciblant le miR-10b pour le traitement du cancer métastatique. Les résultats clés comprennent :
- 13 patients traités à 4 niveaux de dose (de 0,8 mg/kg à 4,8 mg/kg)
- Aucune toxicité significative ou limitant la dose rapportée
- Deux patients ont maintenu une maladie stable pendant environ 7 mois
- 8 patients poursuivent le traitement en cycles de 28 jours
- L’analyse PK/PD montre des résultats prometteurs avec l’engagement de la cible miR-10b chez 5 des 6 patients analysés
Le profil de tolérance positif et les résultats PK/PD soutiennent la progression vers la phase 1b d’expansion de l’essai.
TransCode Therapeutics (NASDAQ: RNAZ) meldete positive Fortschritte in seiner Phase-1a-Studie zu TTX-MC138, einem neuartigen Therapeutikum, das miR-10b für die Behandlung von metastasiertem Krebs gezielt angreift. Wichtige Erkenntnisse umfassen:
- 13 Patienten wurden in 4 Dosierungsstufen behandelt (0,8 mg/kg bis 4,8 mg/kg)
- Keine signifikanten Sicherheits- oder dosisbegrenzenden Toxizitäten berichtet
- Zwei Patienten zeigten etwa 7 Monate lang stabile Erkrankung
- 8 Patienten setzen die Behandlung in 28-Tage-Zyklen fort
- PK/PD-Analyse zeigt vielversprechende Ergebnisse mit miR-10b-Zielbindung bei 5 von 6 analysierten Patienten
Das positive Verträglichkeitsprofil und die PK/PD-Ergebnisse unterstützen den Übergang zur Phase-1b-Erweiterung der Studie.
- No significant safety or dose-limiting toxicities reported in any of the 13 patients
- Two patients demonstrated stable disease over seven months of treatment
- Successful miR-10b target engagement shown in 5 out of 6 analyzed patients
- PK data shows predictable dose-response relationship consistent with preclinical results
- Trial advancing to Phase 1b expansion based on positive safety profile
- None.
Insights
Phase 1a trial of TTX-MC138 shows clean safety profile across all dose levels with target engagement and stable disease in two patients.
TransCode's Phase 1a trial for TTX-MC138 demonstrates a favorable safety profile across four escalating dose levels (0.8 mg/kg to 4.8 mg/kg) in 13 metastatic cancer patients. The absence of dose-limiting toxicities, even at the highest dose, is particularly encouraging for an RNA therapeutic targeting microRNA-10b, as delivery and safety challenges have historically limited RNA drug development.
The pharmacodynamic data showing target engagement at 24 hours post-infusion in 5 of 6 analyzed patients from the lower dose cohorts provides molecular evidence that TTX-MC138 is reaching its intended target. This target engagement data, coupled with the predictable dose-response pharmacokinetic profile, suggests the drug's mechanism is functioning as designed.
While Phase 1a trials primarily assess safety, the observation that two patients have maintained stable disease for approximately seven months provides an initial signal of potential biological activity. For patients with metastatic cancer, stable disease over this duration represents a potentially meaningful clinical finding, though the small sample size necessitates caution in interpretation.
The consistent safety profile and preliminary signs of biological activity appropriately support the planned advancement to Phase 1b expansion cohorts, where the therapeutic potential of TTX-MC138 can be more thoroughly evaluated in a larger patient population.
Early clinical data validates TransCode's RNA delivery platform with clean safety profile and target engagement, derisking their lead program.
TransCode's clinical update represents an important derisking event for their lead program TTX-MC138. The clean safety profile across multiple dose levels addresses one of the primary investor concerns with novel RNA therapeutics – whether the drug can be safely administered at potentially therapeutic doses.
The pharmacokinetic/pharmacodynamic data showing predictable dose-response relationships and target engagement in the majority of analyzed patients provides preliminary validation of the company's RNA delivery technology. This technical validation extends beyond just TTX-MC138, potentially supporting the broader platform value proposition.
The stable disease observed in two patients over approximately seven months, while preliminary, offers an encouraging signal that warrants further investigation in the planned Phase 1b portion of the trial. For an RNA therapeutic targeting microRNA-10b, which is believed to play a critical role in cancer metastasis, these early clinical signals align with the proposed mechanism of action.
The advancement to Phase 1b represents a material milestone that will generate more robust efficacy data and potentially create additional value-inflection points. With eight patients continuing treatment and expanded enrollment underway, forthcoming data should provide greater clarity on TTX-MC138's therapeutic potential in metastatic cancer.
- A total of 13 patients treated with four escalating doses of TTX-MC138
- No significant safety or dose limiting toxicities reported
- Two patients have so far maintained stable disease on treatment for at least seven months
- PD analysis at 24 hours post-dosing provides evidence of miR-10b target engagement
Eight patients remain on study for continued treatment, receiving an additional dose of TTX-MC138 during each 28 day treatment cycle and may remain on study absent any significant safety observations or disease progression. To date, the two patients who have remained on study the longest have received seven doses of TTX-MC138 over the course of approximately seven months and have demonstrated stable disease. No significant safety or dose limiting toxicities have been reported to date in any of the trial's 13 patients. Ongoing analyses of PK activity from Cohorts 1, 2 and 3 suggest that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode's Phase 0 clinical trial. Specifically, the preliminary PK data follow a predictable dose-response relationship. Analysis of PD activity from cycle 1 treatments in Cohorts 1 and 2, treated with a dose of 0.8 mg/kg and 1.6 mg/kg respectively, demonstrates miR-10b target engagement at 24 hours post-infusion in 5 out of the 6 patients analyzed to date.
The observed tolerability profile and the available PK/PD results thus far supports 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. At the highest dose administered, TTX-MC138 was well tolerated with no significant toxicities noted.
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 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.