Oncology Update: RGT-61159 Orphan Drug, CRB-701 Fast Track, PDS0101 Survival Data & More
From FDA orphan drug and fast-track designations to breakthrough survival data and novel subcutaneous immunotherapies—this roundup spans rare adenoid cystic carcinoma, head and neck, non-small cell lung, and hepatocellular cancers. It highlights RNA splicing modulation, antibody-drug conjugates, bispecific checkpoint inhibitors, and microbiome-based therapies driving oncology innovation and competitive dynamics forward.
In Today’s Newsletter
🧬 FDA Orphan Drug Designation for RGT-61159 in ACC [1] [US • 17 Sep 2025]
Context: Oral MYB inhibitor via RNA splicing; Phase 1a/b in relapsed/refractory ACC or CRC (NCT06462183).
Key point: FDA granted ODD with incentives incl. fee relief, tax credits, 7-year exclusivity if approved.
Implication: Introduces competition that may affect pricing and formulary access.
🧪 CRB-701 earns FDA Fast Track in HNSCC [2] [US • 16 Sep 2025]
Context: Nectin-4 ADC (SYS6002) with DAR2 MMAE; Phase 1/2 dose-optimization data due at ESMO 2025.
Key point: Fast Track for recurrent/metastatic HNSCC post platinum + anti-PD(L)-1; second Fast Track for CRB-701.
Implication: May influence prescriber choice and payer reviews pending full data.
📈 PDS0101+pembrolizumab extends survival in CPS 1–19 HNSCC [3] [US • 18 Sep 2025]
Context: VERSATILE-002 Phase 2; 60% of pts had CPS 1–19; n=32 sub-analysis. Literature comparators only.
Key point: Reported mOS 29.5 mo vs published ~10.8 mo (pembro) and ~12.3 mo (pembro+chemo).
Implication: May influence prescriber choice and payer reviews pending full data.
💡 FDA pre-IND alignment on FG001 (glioma surgery imaging) [4] [US • 16 Sep 2025]
Context: FG001 uPAR imaging agent; IND submission planned Q4 2025 for Phase 2 HGG trial.
Key point: FDA aligned on US Phase 2 design and Phase 3 program elements.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🫁 Japan approves IBTROZI™ (taletrectinib) for ROS1+ NSCLC [5] [JP • 19 Sep 2025]
Context: Based on TRUST Phase 2 program; AmoyDx® PLC Panel companion diagnostic approved; Nippon Kayaku commercializes.
Key point: Builds on prior approvals in US and China; reimbursement price in Japan expected Q4 2025.
Implication: Introduces competition that may affect pricing and formulary access.
🧿 Cadonilimab global registrational trial first patient dosed in HCC [6] [HK • 15 Sep 2025]
https://akesobio.com/en/media/akeso-news/250915/
Context: COMPASSION-36/AK104-225 Phase 2 registrational; cadonilimab (PD-1/CTLA-4) + lenvatinib vs lenvatinib in IO-resistant HCC.
Key point: First global registrational trial of cadonilimab beyond China; addresses post-atezo/bev setting.
Implication: Signals pipeline investment and modality expansion.
🌸 Raludotatug deruxtecan (R-DXd) gets FDA Breakthrough Therapy [7] [US • 15 Sep 2025]
https://www.daiichisankyo.com/files/news/pressrelease/pdf/202509/20250915_E.pdf
Context: CDH6-targeted ADC from Daiichi Sankyo/Merck; designation supported by Phase 1 and REJOICE-Ovarian01 Phase 2/3.
Key point: First BTD for R-DXd; second BTD in Daiichi–Merck alliance; fifteenth oncology BTD for Daiichi.
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 Microbiotica completes MELODY-1 enrollment (MB097+pembrolizumab) [8] [UK • 17 Sep 2025]
Context: Phase 1b, 41 melanoma pts across UK/FR/IT/ES; randomized to ± vancomycin before MB097+pembrolizumab. Results 1H 2026.
Key point: First-in-human precision microbiome consortium (9 bacterial strains) fully recruited in anti-PD-1-resistant melanoma.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🦴 ROMVIMZA™ (vimseltinib) approved in EU for TGCT [9] [EU • 17 Sep 2025]
https://www.deciphera.com/news/deciphera-receives-european-commission-approval-for-romvimza
Context: CSF1R inhibitor; supported by Phase 3 MOTION and Phase 1/2; improvements in ROM, pain, stiffness, QoL.
Key point: First EU-approved therapy for TGCT; Deciphera (Ono affiliate) to commercialize.
Implication: Introduces competition that may affect pricing and formulary access.
💉 FDA approves KEYTRUDA QLEX™ (SC pembrolizumab+berahyaluronidase) [10] [US • 19 Sep 2025]
Context: Phase 3 D77 in 1L metastatic NSCLC; SC dosing showed comparable exposure and ORR to IV pembrolizumab.
Key point: First SC checkpoint inhibitor across broad indications; 1–2 min administration by HCP.
Implication: Could streamline initiation and adherence via remote prescribing and logistics.
Why it matters
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- Rare tumor incentives (ODD, BTD) accelerate RNA modulators and ADCs in underserved cancers.
- Subcutaneous KEYTRUDA QLEX offers site-of-care and time-saving advantages for patients and providers.
- Japan and EU regulatory actions expand global reach of IBTROZI (NSCLC) and ROMVIMZA (TGCT).
- PDS0101 and MB097 show promise in checkpoint-resistant populations, highlighting combo and microbiome strategies.
- Akeso’s cadonilimab push reflects intensifying bispecific competition in IO-resistant HCC.
📢 Stay Ahead in Oncology Research!
✅ Contact LucidQuest at info@lqventures.com for strategic guidance on oncology innovations and clinical research.
FAQ
What is RGT-61159?
An oral MYB splicing modulator from Rgenta; FDA granted Orphan Drug Designation in ACC, with trial ongoing in ACC/CRC [1].
What’s special about CRB-701?
A site-specific Nectin-4 ADC with DAR2 MMAE design; now has Fast Track for HNSCC post platinum + anti-PD(L)-1 [2].
How meaningful are PDS0101 survival data?
Reported 29.5-mo mOS in CPS 1–19 subgroup is from trial sub-analysis; comparator pembrolizumab data are from published literature, not head-to-head [3].
How does FG001 fit into surgery?
FG001 lights up glioma tissue intraoperatively; FDA pre-IND alignment supports Phase 2/3 trial designs [4].
Where is taletrectinib approved now?
IBTROZI is approved in US, China, and now Japan (MHLW) for ROS1+ NSCLC, with companion diagnostic cleared [5].
What is cadonilimab’s trial COMPASSION-36?
A global Phase 2 registrational trial of cadonilimab+lenvatinib vs lenvatinib in IO-resistant HCC; first patient dosed [6].
What did FDA grant to R-DXd?
Breakthrough Therapy Designation for CDH6+ platinum-resistant ovarian cancer, based on Phase 1 and REJOICE-Ovarian01 [7].
What is MB097?
A nine-strain live biotherapeutic consortium to enhance anti-PD-1 efficacy; fully recruited MELODY-1 Phase 1b trial [8].
What is ROMVIMZA?
Vimseltinib, a CSF1R inhibitor, became first EU-approved TGCT therapy, supported by MOTION Phase 3 trial [9].
What is KEYTRUDA QLEX?
A subcutaneous pembrolizumab+berahyaluronidase combo, FDA approved across most solid tumor indications with ~1–2 min administration [10].
Entities / Keywords
Rgenta Therapeutics; RGT-61159 (MYB splicing modulator) | Corbus Pharmaceuticals; CRB-701/SYS6002 (Nectin-4 ADC) | PDS Biotechnology; PDS0101 (Versamune® HPV) | FluoGuide; FG001 (uPAR imaging) | Nuvation Bio; IBTROZI™ (taletrectinib) | Nippon Kayaku | Akeso; cadonilimab (PD-1/CTLA-4) | Daiichi Sankyo & Merck; raludotatug deruxtecan (R-DXd; CDH6 ADC) | Microbiotica; MB097 (LBP) | Deciphera / Ono; ROMVIMZA™ (vimseltinib; CSF1R) | Merck; KEYTRUDA QLEX™ (SC pembrolizumab+berahyaluronidase).
References
- https://www.globenewswire.com/news-release/2025/09/17/3151594/0/en/Rgenta-Therapeutics-Receives-Orphan-Drug-Designation-from-the-U-S-FDA-for-RGT-61159-for-the-Treatment-of-Adenoid-Cystic-Carcinoma-ACC.html
- https://ir.corbuspharma.com/news-events/press-releases/detail/448/fda-grants-fast-track-designation-to-corbus-pharmaceuticals-nectin-4-targeting-adc-crb-701-in-head-and-neck-squamous-cell-carcinoma
- https://www.pdsbiotech.com/index.php/investors/news-center/press-releases/press-releases1/132-2025-news/1018-pds-biotech-sets-significant-benchmark-in-head-and-neck-canc2025-09-18-055003
- https://fluoguide.com/mfn_news/fluoguide-receives-positive-fda-feedback-on-fg001-in-high-grade-glioma/
- https://investors.nuvationbio.com/news/news-details/2025/Nuvation-Bio-Receives-Approval-from-Japans-Ministry-of-Health-Labour-and-Welfare-for-IBTROZITM-for-Patients-with-Advanced-ROS1-positive-Non-Small-Cell-Lung-Cancer/default.aspx
- https://akesobio.com/en/media/akeso-news/250915/
- https://www.daiichisankyo.com/files/news/pressrelease/pdf/202509/20250915_E.pdf
- https://microbiotica.com/microbiotica-announces-completion-of-recruitment-in-its-international-phase-1b-trial-melody-1-of-mb097-a-precision-microbiome-co-therapy-in-advanced-melanoma/
- https://www.deciphera.com/news/deciphera-receives-european-commission-approval-for-romvimza
- https://www.merck.com/news/fda-approves-mercks-keytruda-qlex-pembrolizumab-and-berahyaluronidase-alfa-pmph-injection-for-subcutaneous-use-in-adults-across-most-solid-tumor-indications-for-keytruda-pem/