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DRUG CLASS:

GDF15 inhibitor

21d
Regulation of ferroptosis in colorectal cancer through therapeutic modulation and miRNA targeting. (PubMed, Biochem Biophys Rep)
Interestingly, we also found that medications such as prasterone, tazemetostat, isoxyl, gemcitabine, ponsegromab, scx-2023, and nicotinamide could potentially be used in combination with the identified miRNAs to target ferroptosis in CRC. To further validate the stability and reliability of the predicted protein-ligand interactions, molecular dynamics (MD) simulations and MM-PBSA analyses were performed on selected top-ranking complexes, which confirmed their stable and favorable binding and supported the robustness of our docking results. These findings suggest that targeting these miRNAs and their associated genes, along with using the identified drugs, could be a promising strategy for CRC treatment, leveraging the potential of ferroptosis-inducing therapies.
Journal • PARP Biomarker
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PARP1 (Poly(ADP-Ribose) Polymerase 1) • RRM2 (Ribonucleotide Reductase Regulatory Subunit M2) • SLC7A11 (Solute Carrier Family 7 Member 11) • MIR16 (MicroRNA 16) • MIR15A (MicroRNA 15a) • MIR423 (MicroRNA 423) • MIR93 (MicroRNA 93)
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gemcitabine • Tazverik (tazemetostat) • ponsegromab (PF-06946860)
29d
GDFATHER-01: First-in-Human Study of the GDF-15 Neutralizing Antibody Visugromab (CTL-002) in Patients With Advanced Cancer (GDFATHER) (clinicaltrials.gov)
P1/2, N=263, Active, not recruiting, CatalYm GmbH | Trial completion date: Oct 2027 --> Apr 2030 | Trial primary completion date: Oct 2025 --> Apr 2028
Trial completion date • Trial primary completion date • Checkpoint inhibition • First-in-human
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visugromab (CTL-002)
1m
A Dose Escalation Study of AV-380 in Cancer Patients With Cachexia (clinicaltrials.gov)
P1, N=30, Recruiting, AVEO Pharmaceuticals, Inc. | Trial completion date: Jun 2026 --> Dec 2026 | Trial primary completion date: Dec 2025 --> Dec 2026
Trial completion date • Trial primary completion date
2ms
VINCIT: Visugromab in Cachexia International Trial (clinicaltrials.gov)
P2/3, N=518, Recruiting, CatalYm GmbH | Not yet recruiting --> Recruiting
Enrollment open
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visugromab (CTL-002)
2ms
The evolving landscape of cancer cachexia prevention: A review of metronomic chemotherapy and drug repurposing strategies. (PubMed, Med Oncol)
A good number of preclinical and early clinical studies have shown evidence for both approaches, particularly for Ponsegromab, a Growth Differentiation Factor-15 (GDF-15) inhibitor. Due to the multifactorial nature of cachexia, a multimodal, integrated intervention is a good substitute for the maximum tolerated dose, which can be effective against it. Future directions in cancer therapy should emphasize the development of robust clinical trial designs with cachexia-specific endpoints to advance precision prevention strategies.
Review • Journal
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GDF15 (Growth differentiation factor 15)
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ponsegromab (PF-06946860)
3ms
New P2 trial
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Opdivo (nivolumab) • docetaxel • visugromab (CTL-002)
4ms
Enrollment open
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ponsegromab (PF-06946860)
4ms
Design of Bicyclic Peptide Tandems Mimicking the Homodimeric GDF15 Protein to Inhibit GDF15-GFRaL-RET Complex Cell Signaling. (PubMed, J Med Chem)
These dimerized protein mimetics inhibited cell signaling in a functional assay and showed improved pharmacokinetic properties compared with their monomeric counterparts. This is the first example of a homodimeric Bicycle molecule inhibiting receptor complex formation, thereby antagonizing the intracellular signaling response.
Journal
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GDF15 (Growth differentiation factor 15)
5ms
Latest Updates on Sarcopenia and Cachexia: Insights from the 17th Sarcopenia, Cachexia, and Wasting Disorders Conference. (PubMed, J Bone Metab)
The conference also highlighted promising clinical advancements, including the HIPGEN trial on placental-expanded stromal cells for muscle regeneration in hip fracture patients and the ponsegromab study targeting growth/differentiation factor-15 inhibition to mitigate cancer cachexia-associated muscle wasting. This review highlights the integration of basic science, innovative diagnostics, and clinical applications as a promising framework for addressing the complex challenges posed by muscle-wasting disorders. As the field progresses, these insights offer hope for improving the quality of life and survival of affected patients.
Journal
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GDF15 (Growth differentiation factor 15)
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ponsegromab (PF-06946860)
5ms
Enrollment open
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Keytruda (pembrolizumab) • carboplatin • pemetrexed • visugromab (CTL-002)