Weekly New-Drug Roundup: CDH6 ADC Shows Promise in Ovarian Cancer; Arvinas' Parkinson Degrader Data Disclosed

WuXi AppTec's weekly new-drug roundup: OnCusp's CDH6 ADC CUSP06 posted 62% ORR in platinum-resistant ovarian cancer; Arvinas' LRRK2 degrader ARV-102 showed dose-related oculomotor and biomarker signals in Parkinson's disease; Ractigen RAG-18, Korro KRRO-121, Crescent CR-002 and Medicovestor MC001 disclosed first-in-human or IND milestones.

This roundup highlights recently disclosed clinical and regulatory milestones across modalities — ADCs, targeted protein degraders, small activating RNA and oligonucleotide editors — signaling continued pipeline momentum.

OnCusp Therapeutics reported 1b dose-expansion data for CUSP06, a CDH6-directed ADC. The study enrolled 86 patients with platinum-resistant ovarian cancer (median three prior lines), with 81 evaluable. CUSP06 delivers exatecan (a topoisomerase I inhibitor) via an anti-CDH6 antibody and cleavable linker. In the 4.0 mg/kg cohort (n=21), the objective response rate (ORR) was 62%, including 3 complete responses; the 3.6 mg/kg cohort (n=20) showed 50% ORR. Among 30 evaluable patients previously treated with mirvetuximab soravtansine, 16 responded (ORR 53%). Principal adverse events involved hematology, gastrointestinal reactions and fatigue, with no ocular toxicity reported.

Arvinas' LRRK2 degrader ARV-102: exploratory Phase 1 data in Parkinson's

Arvinas disclosed Phase 1 data for ARV-102, an oral LRRK2-targeted protein degrader, in Parkinson's disease patients. The study enrolled 24 patients receiving 20–80 mg or placebo for 28 days. Compared with placebo, ARV-102 produced dose-related improvements in saccadic amplitude; reduced CSF LRRK2 protein levels correlated with oculomotor changes and biomarker shifts in related pathways. ARV-102 is designed to cross the blood–brain barrier and degrade LRRK2, modulating neurodegeneration-related biology. Endolysosomal and neuroinflammatory markers decreased while synapse-integrity and axon-guidance markers rose — supporting central target engagement — though the oculomotor analysis was exploratory and the study was not designed to demonstrate efficacy.

Multiple programs disclose first-in-human and IND progress

Ractigen Therapeutics reported first-in-human data for RAG-18, a lipid-conjugated small activating RNA targeting the UTRN promoter to raise utrophin expression and compensate for dystrophin loss. In the first cohort, three DMD boys received 15 mg IV monthly; day-113 muscle biopsies showed sarcolemmal utrophin signal density 3.5–5.3× baseline. Through day 169 there were no DLTs, SAEs or ≥grade-3 TEAEs; functional measures were inconsistent (6-minute walk changes +36.5 / −1 / −62.5 m; all three NSAA scores fell by 3), providing primarily mechanism and early safety signals.

Korro Bio dosed the first healthy-subject cohort (2 mg/kg SC) in the ANCHOR Phase 1/2 trial of KRRO-121, a GalNAc-conjugated oligonucleotide directing RNA editing to the glutamine synthetase transcript to generate a more stable enzyme for hyperammonemia; the Australian Phase 1 (up to 50 subjects) is ongoing, with the patient Phase 2 portion planned later.

Crescent Biopharma's PD-L1-targeted ADC CR-002 and Medicovestor's 'first-in-class' dimeric ADC chemo-immunotherapy MC001 each cleared FDA IND, advancing into the global Phase 1/2 APOLLO study (solid tumors) and an international Phase 1 (advanced solid tumors, pancreatic focus), respectively — both moving from preclinical to human study without yet disclosing efficacy or full safety data.

From ADCs and targeted degradation to RNA activation and oligonucleotide editing, multiple programs reported clinical and regulatory milestones this week, underscoring how frontier modalities keep advancing from concept toward clinical validation.