Vaximm AG, an OSR Company, Announces Results from Phase 2a Trial of VXM01 and Avelumab Combination Therapy in Glioblastoma
Vaximm AG, an OSR Holdings subsidiary, has announced final data from its Phase 2a clinical trial evaluating VXM01 in combination with avelumab for recurrent glioblastoma (GBM) treatment. The trial demonstrated:
- Good safety profile with mostly mild to moderate events
- 12.0% objective response rate in non-resected patients
- Median overall survival of 11.1 months
- Median time to progression of 2.7 months
The study (n=25) showed promising results compared to typical GBM prognosis. No serious adverse events were attributed to VXM01, while 81.8% were related to the disease itself. In resected patients, overall survival ranged from 2.2 to 46.5 months. The trial identified potential biomarkers for VXM01-mediated tumor response, supporting further investigation of the treatment combination.
Vaximm AG, una filiale di OSR Holdings, ha annunciato i dati finali del suo studio clinico di Fase 2a che valuta VXM01 in combinazione con avelumab per il trattamento del glioblastoma ricorrente (GBM). Lo studio ha dimostrato:
- Un buon profilo di sicurezza con eventi per lo più lievi o moderati
- Un tasso di risposta obiettiva del 12,0% nei pazienti non resezionati
- Una sopravvivenza mediana di 11,1 mesi
- Un tempo mediano fino alla progressione di 2,7 mesi
Lo studio (n=25) ha mostrato risultati promettenti rispetto alla prognosi tipica del GBM. Nessun evento avverso grave è stato attribuito a VXM01, mentre l'81,8% era correlato alla malattia stessa. Nei pazienti resezionati, la sopravvivenza complessiva variava da 2,2 a 46,5 mesi. Lo studio ha identificato potenziali biomarcatori per la risposta tumorale mediata da VXM01, sostenendo ulteriori indagini sulla combinazione di trattamenti.
Vaximm AG, una subsidiaria de OSR Holdings, ha anunciado los datos finales de su ensayo clínico de Fase 2a que evalúa VXM01 en combinación con avelumab para el tratamiento del glioblastoma recurrente (GBM). El ensayo demostró:
- Un buen perfil de seguridad con eventos mayormente leves a moderados
- Una tasa de respuesta objetiva del 12,0% en pacientes no reseccionados
- Una supervivencia media de 11,1 meses
- Un tiempo medio hasta la progresión de 2,7 meses
El estudio (n=25) mostró resultados prometedores en comparación con el pronóstico típico del GBM. No se atribuyeron eventos adversos graves a VXM01, mientras que el 81,8% estaban relacionados con la enfermedad en sí. En pacientes reseccionados, la supervivencia general osciló entre 2,2 y 46,5 meses. El ensayo identificó biomarcadores potenciales para la respuesta tumoral mediada por VXM01, apoyando una investigación adicional de la combinación de tratamientos.
Vaximm AG는 OSR Holdings의 자회사로, 재발성 교모세포종(GBM) 치료를 위한 avelumab과의 병용요법에서 VXM01를 평가한 2a상 임상시험의 최종 데이터를 발표했습니다. 이 시험은 다음과 같은 결과를 보여주었습니다:
- 대부분 경미하거나 중간 정도의 사건으로 좋은 안전성 프로필
- 절제되지 않은 환자에서 12.0%의 객관적 반응률
- 중앙 전체 생존 기간 11.1개월
- 진행까지의 중앙 시간 2.7개월
연구(n=25)는 전형적인 GBM 예후에 비해 유망한 결과를 보여주었습니다. VXM01에 기인한 심각한 부작용은 없었고, 81.8%는 질병 자체와 관련이 있었습니다. 절제된 환자에서 전체 생존 기간은 2.2개월에서 46.5개월 사이였습니다. 이 시험은 VXM01에 의한 종양 반응을 위한 잠재적 바이오마커를 식별하였으며, 치료 조합에 대한 추가 조사를 지원합니다.
Vaximm AG, une filiale d'OSR Holdings, a annoncé les données finales de son essai clinique de Phase 2a évaluant VXM01 en combinaison avec avelumab pour le traitement du glioblastome récurrent (GBM). L'essai a démontré :
- Un bon profil de sécurité avec principalement des événements légers à modérés
- Un taux de réponse objective de 12,0% chez les patients non réséqués
- Une survie médiane de 11,1 mois
- Un temps médian jusqu'à la progression de 2,7 mois
L'étude (n=25) a montré des résultats prometteurs par rapport au pronostic typique du GBM. Aucun événement indésirable grave n'a été attribué à VXM01, tandis que 81,8% étaient liés à la maladie elle-même. Chez les patients réséqués, la survie globale variait de 2,2 à 46,5 mois. L'essai a identifié des biomarqueurs potentiels pour la réponse tumorale médiée par VXM01, soutenant des investigations supplémentaires de la combinaison de traitements.
Vaximm AG, eine Tochtergesellschaft von OSR Holdings, hat die endgültigen Daten aus ihrer Phase-2a-Studie veröffentlicht, die VXM01 in Kombination mit Avelumab zur Behandlung von rezidivierenden Glioblastomen (GBM) bewertet. Die Studie zeigte:
- Gutes Sicherheitsprofil mit überwiegend milden bis moderaten Ereignissen
- 12,0% objektive Ansprechrate bei nicht resezierten Patienten
- Medianes Gesamtüberleben von 11,1 Monaten
- Medianzeit bis zur Progression von 2,7 Monaten
Die Studie (n=25) zeigte vielversprechende Ergebnisse im Vergleich zur typischen GBM-Prognose. Es wurden keine schweren unerwünschten Ereignisse VXM01 zugeschrieben, während 81,8% mit der Erkrankung selbst in Zusammenhang standen. Bei resezierten Patienten lag das Gesamtüberleben zwischen 2,2 und 46,5 Monaten. Die Studie identifizierte potenzielle Biomarker für die durch VXM01 vermittelte Tumorantwort und unterstützt weitere Untersuchungen der Behandlungs-Kombination.
- Favorable safety profile with no serious adverse events attributed to VXM01
- 12.0% objective response rate in non-resected patients
- Median OS of 11.1 months exceeds typical 2-9 months prognosis
- Treatment showed effectiveness regardless of tumor size
- Small trial size (n=25) limits statistical significance
- High variability in patient responses (OS range 2.2-46.5 months)
- Additional studies needed to determine optimal treatment regimens
Insights
Vaximm's Phase 2a results for VXM01-avelumab combination therapy in recurrent glioblastoma represent a meaningful development in a notoriously difficult-to-treat cancer. Glioblastoma remains one of the most aggressive CNS malignancies with dismal survival rates, particularly after recurrence, where median overall survival typically ranges from 2-9 months.
The safety profile is particularly encouraging—no serious adverse events attributed to VXM01 is significant in this context, as most available treatments carry substantial toxicity burdens. The fact that 81.8% of SAEs were related to the underlying disease rather than the treatment indicates a manageable safety profile in these fragile patients.
From an efficacy perspective, while preliminary, the median overall survival of 11.1 months exceeds historical benchmarks, and the 12.0% objective response rate in non-resected patients deserves attention. Though modest in absolute terms, any measurable response in recurrent GBM represents clinical benefit given the disease's aggressive nature and resistance to therapy.
The observation that tumor shrinkage occurred regardless of baseline tumor size suggests potential applications across various disease stages. The identification of potential biomarkers indicates progress toward a precision medicine approach, which could optimize patient selection in future trials.
As a Phase 2a study with a small sample size (n=25), these results warrant cautious interpretation but justify continued development of VXM01 as a treatment option for this devastating disease with few effective therapies.
The completion of Vaximm's Phase 2a trial represents a positive development for OSR Holdings' subsidiary in the competitive immuno-oncology space. The dual-mechanism approach—combining an oral anti-VEGFR-2 vaccine (VXM01) with a PD-L1 inhibitor (avelumab)—attacks glioblastoma through complementary pathways, potentially offering advantages over single-agent approaches.
Several elements stand out from an investment perspective: First, the collaboration with Merck KGaA provides external validation and resource support, typical of partnerships that help biotechs navigate expensive late-stage trials. Second, the oral delivery format of VXM01 represents a potential competitive advantage in a field dominated by injectable immunotherapies.
The biomarker identification is particularly significant as it could enable patient stratification in future trials, potentially improving response rates and creating a precision medicine approach—factors that significantly enhance regulatory and commercial prospects.
While focusing on glioblastoma presents challenges due to the blood-brain barrier and the tumor's immunosuppressive environment, successful therapies in this indication can command premium pricing due to the severe unmet need.
The modest sample size (n=25) limits definitive conclusions, but the safety profile and preliminary efficacy signals provide a foundation for advancement to larger trials. For OSR Holdings, these results potentially enhance the value of their Vaximm subsidiary and justify continued investment in this therapeutic approach.
Results from Phase 2a Trial demonstrate a good safety and tolerability profile of VXM01 and Avelumab Combination Therapy in Glioblastoma, supporting further investigation of VXM01
Key results and observations:
- The VXM01-avelumab combination therapy was generally well-tolerated, with the majority of safety events being mild to moderate in severity. These safety and tolerability data are in-line with previously reported data on avelumab alone with no additional safety signals for the combination of VXM01 and avelumab.
- No serious adverse events (SAEs) were attributed to VXM01, while 9 of 11 (
81.8% ) were related to the target disease, underscoring the well manageable safety profile of this combination therapy in a frail patient population. - The non-resected patient cohort showed a
12.0% objective response rate (ORR).12.0% of these patients showed a partial remission and4.0% had stable disease. This suggests that, with further investigation, VXM01 in combination with PD-L1 inhibition (e.g. avelumab) could offer meaningful clinical benefit for this challenging patient population. In resected patients, the overall survival (OS) ranged from 2.2 to 46.5 months, highlighting the variability in response and the need for additional studies to determine optimal treatment regimens for specific subgroups of GBM patients. - Despite the small size of this open-label trial (n=25), the observed median time to progression of 2.7 months (
95% CI: 2.7 – 2.7, range 0.3 - 22.1 months), and median OS of 11.1 months (95% CI: 8.5 – 16.3, range 3.8- 38.2 months), are encouraging initial results in the context of prognosis for patients with recurrent glioblastoma, reported to have a median PFS of 1.5 to 6 months and median OS of 2 to 9 months.(Birzu et al. 2020) - Decreased tumor size was observed in responding patients independent of tumor size at baseline, supporting the expectation that VXM01 vaccine treatment may be effective in patients with larger sized tumors as well as patients with early-stage cancer or very small tumors.
- Exploratory biomarker investigations identified potential predictive and pharmacodynamic biomarkers of a VXM01-mediated tumor response
Moving Forward:
The reported safety and tolerability data, together with early indications for the potential relevance of a VXM01dependent, VEGFR-2 specific immune response in GBM therapy warrant further study.
"The completion of this Phase 2a study is a significant milestone for Vaximm AG, as it provides strong evidence that VXM01, in combination with avelumab is generally well-tolerated in patients with recurrent glioblastoma," said Dr. Constance Hoefer, CEO of Vaximm AG. "We are encouraged by these early results and the potential to improve outcomes for patients with this aggressive cancer. We remain committed to advancing VXM01 as a key therapeutic candidate for the treatment of glioblastoma, other cancers and other diseases where VXM01 may have positive impact on treatment outcomes"
About VXM01:
VXM01 is an oral T-cell immunotherapy that is designed to activate T-cells to attack the tumor vasculature and, in several tumor types, attack cancer cells directly. It is based on a live attenuated, safe, orally available bacterial vaccine strain, which is modified to carry vascular endothelial growth factor receptor-2 (VEGFR2) as the target gene. VXM01 stimulates the patient's immune system to activate VEGFR2-specific, cytotoxic T-cells (so-called killer cells). These immune killer cells then actively destroy cells in the tumor vasculature, leading to an increased infiltration of various immune cells into the tumor. In several tumor types, including brain cancer, VEGFR2 is highly over-expressed on the cancer cells themselves. In preclinical studies, a murine analog VXM01 vaccine showed broad anti-tumor activity in different tumor types. This activity was linked to a VEGFR2-specific T-cell response and was accompanied by the destruction of the tumor vasculature and increased immune cell infiltration. In a Phase I double-blind, randomized, placebo-controlled study in 71 patients with advanced pancreatic cancer, VXM01 appeared to be safe and well tolerated and led to the activation of VEGFR2-specific cytotoxic T-cells, which was associated with significantly improved patient survival. Clinical activity in terms of objective responses and survival has been observed in recurrent glioblastoma.
About Vaximm AG:
Vaximm AG is a pioneering biotechnology company focused on developing innovative immunotherapies. Through innovative approaches, Vaximm aims to unlock the potential of cancer vaccines and immune-oncology therapies to address the unmet needs of patients suffering from various types of cancer. For more information about Vaximm, please visit www.vaximm.com.
About OSR Holdings Inc:
OSR Holdings, Inc. (NASDAQ: OSRH) is a global healthcare company dedicated to advancing healthcare outcomes and improving the quality of life for people and their families. OSR aims to build and develop a robust portfolio of innovative and potentially transformative therapies and healthcare solutions. Its current operating businesses (through three wholly-owned subsidiaries) include (i) developing oral immunotherapies for the treatment of cancer ("Vaximm"), (ii) developing design-augmented biologics for age-related and other degenerative diseases ("Darnatein") and (iii) neurovascular intervention medical device and systems distribution in
Contact:
Vaximm AG
Email: ir@osr-holdings.com
Website: www.vaximm.com
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SOURCE OSR Holdings Inc.
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