Substantially Improved Monotherapy ORR at 100 mg QD
Confirmed ORR for the 100 mg once-daily casdatifan cohort increased from 35% (Aug data cut) to 45% (Jan 30 data cut) — an absolute increase of 10 percentage points (≈28.6% relative increase). Pooled confirmed ORR improved from 31% to 35% (4 percentage points, ≈12.9% relative).
Strong and Durable PFS Results
100 mg cohort median PFS of 15.1 months (median follow-up 17.8 months). Pooled median PFS 12.2 months. These PFS outcomes are ~2–3x longer than the 5.6 months reported for belzutifan in the same late-line setting (15.1 vs 5.6 months ≈2.7×).
Low Primary Progression Rates Supporting TKI‑Sparing Frontline Strategy
CAS shows consistently low primary progression across cohorts (example: CAS + zim cohort primary progression 9% for first 23/30 patients). By contrast, belzutifan monotherapy has a 35% primary progression rate in its Phase III trial — an absolute reduction of ~26 percentage points (≈74% relative reduction) vs belzutifan.
ARC‑20 Platform Enables Rapid Development and Dose Optimization
ARC‑20 provided 121 late‑line monotherapy patient data supporting 100 mg QD as the optimal dose, rapid addition of ~90 patients across new cohorts, and ~30 active sites in 4 countries to accelerate combo cohorts and support Phase III starts (PEAK‑1 and frontline cohorts).
PEAK‑1 Phase III Enrolling; Fast‑to‑Market Design
PEAK‑1 (casdatifan + cabozantinib vs cabozantinib) is actively enrolling with a sole primary endpoint of PFS to shorten time to readout; company expects to complete enrollment by year‑end and views this as a high‑confidence, fast‑to‑market strategy in IO‑experienced ccRCC (~21,000 patients in major markets targeted for PEAK‑1).
Significant Commercial Opportunity Quantified
Management estimates casdatifan peak sales of ~$2.5B in the IO‑experienced (PEAK‑1) setting alone and ~$3B+ in first line, totaling a potential >$5B opportunity in 1st/2nd line RCC; current marketed belzutifan run‑rate ~ $1B validates class opportunity.
Robust Cash Position and Runway
Cash of $1.0 billion at end of Q4 vs $841 million at end of Q3 (+$159M, ≈18.9% increase), bolstered by $288M financing in November. Company expects cash and investments to fund operations until at least the second half of 2028.
Q4 Financials and 2026 Guidance
Q4 GAAP revenue of $33M vs $26M in Q3 (+$7M, ≈26.9% QoQ). Q4 R&D expense decreased to $121M from $141M in Q3 (≈‑14.2% QoQ). Company guides 2026 GAAP revenue of $45M–$55M and expects operating expenses to decrease meaningfully in 2026.
Advancing Immunology Pipeline with Near‑Term Clinic Entry
Three advanced I&I programs highlighted: MRGPRX2 antagonist expected to enter clinic later this year (Phase I healthy volunteers then proof‑of‑concept in chronic inducible urticaria with potential PoC in 9–12 months); selective TNF receptor‑1 inhibitor expected in clinic late 2026/early 2027 — both positioned for differentiation vs biologics.