Mitotics · Molecular Oncology · Q2 2025

Emerging Therapies Across US Cancer

A comprehensive clinical reference covering the most consequential therapeutic advances across all major US cancer types — ADCs, CAR-T, bispecifics, KRAS inhibitors, radioligand therapy, and precision oncology at the frontier.

14Cancer Types
~2MNew US Cases/Yr
80+Emerging Agents
40+Pivotal Trials
12FDA Approvals 2024–25
Mitotics Clinical Intelligence · Pan-Cancer Emerging Therapies · Q2 2025 Update · United States

The 2025 oncology therapeutic landscape is undergoing a structural transformation. For the first time, the majority of FDA oncology approvals are mechanism-based precision agents rather than cytotoxic chemotherapy — driven by ADC engineering, bispecific antibody platforms, KRAS inhibitor breakthroughs, radioligand therapy, and next-generation CAR-T constructs. This reference synthesizes the most clinically consequential developments across all major US cancer types.

1

Pan-Cancer Therapeutic Modalities — 2025 Landscape


Breakthrough Class

Antibody-Drug Conjugates (ADCs)

ADCs have emerged as the most productive drug class in oncology. Trastuzumab deruxtecan (T-DXd, ENHERTU) has demonstrated activity across HER2-expressing tumors regardless of histology — establishing a pan-tumor HER2-directed paradigm. The bystander killing effect of topoisomerase-I payloads enables efficacy even in HER2-low tumors. >25 ADCs now in late-stage development across all major solid tumors.

Cellular Therapy

CAR-T & Cellular Therapies

Solid tumor CAR-T is progressing from proof-of-concept to early efficacy signals. Next-gen CAR-T designs incorporate multi-antigen targeting, armored/TEAM constructs (cytokine secretion), and allogeneic off-the-shelf platforms. Hematologic CAR-T (BCMA, CD19, CD22) is increasingly reaching curative intent. TIL therapy (lifileucel) became the first solid tumor cellular therapy FDA-approved in 2024.

Breakthrough Class

KRAS Inhibitors

Sotorasib and adagrasib validated the long-considered "undruggable" KRAS G12C target, now approved in NSCLC and CRC. Pan-KRAS inhibitors targeting G12D (the most prevalent KRAS mutation, dominant in PDAC) represent the next frontier — with RMC-9805, MRTX1133, and HRS-4642 advancing through Phase 1/2. KRAS G12C covers ~13% NSCLC, ~3% CRC, ~2% PDAC; G12D covers ~40% of PDAC.

FDA Approved

Radioligand Therapy (RLT)

Lutetium-177 PSMA (Lu-PSMA-617, Pluvicto) in mCRPC and Lu-177 DOTATATE (Lutathera) in NETs established RLT as a clinical pillar. Actinium-225 conjugates (alpha emitters) show dramatically higher linear energy transfer — PSMA-targeted Ac-225 demonstrating deep responses in heavily pre-treated mCRPC. RLT platforms are being extended to SSTR2 (lung NETs), FAPI (fibroblast activation), GRP78, and carbonic anhydrase IX targets.

Rapid Development

Bispecific Antibodies

T-cell engager bispecifics (TCEs) have achieved meaningful response rates in hematologic malignancies — blinatumomab (CD19×CD3), mosunetuzumab, epcoritamab, glofitamab (CD20×CD3) are approved. In solid tumors, tarlatamab (DLL3×CD3) for SCLC received accelerated FDA approval 2024 — the first TCE approved for a solid tumor. >60 bispecific constructs now in oncology pipelines.

Platform Class

Tumor-Agnostic & Pan-Tumor Strategies

TMB-high (pembrolizumab), MSI-H/dMMR (pembrolizumab), NTRK fusions (larotrectinib, entrectinib), RET fusions (selpercatinib), BRAF V600E (dabrafenib+trametinib), HER2 (T-DXd) — the pan-tumor approval pathway has matured. Basket trials now routinely identify histology-independent response signals. Liquid biopsy ctDNA screening is enabling population-level tumor-agnostic drug matching that was logistically impossible 5 years ago.

2

Highest-Impact Cancer Targets — 2025


Lung (NSCLC/SCLC)

~238,000 new cases/yr

KRAS G12C, EGFR exon 20, HER2, MET, RET, NTRK — most biomarker-driven of all solid tumors.

Breast Cancer

~310,000 new cases/yr

ADC revolution (T-DXd, SG), CDK4/6 combinations, PARP inhibitors, PI3K/AKT pathway.

Colorectal

~153,000 new cases/yr

KRAS G12C (sotorasib+cetuximab), HER2 amplification, BRAF V600E, RAS-WT EGFR therapy.

Prostate

~299,000 new cases/yr

PSMA radioligand therapy, PARP inhibitors (BRCA1/2), AR pathway evolution, BiTE.

Pancreatic

~66,000 new cases/yr

KRAS G12D inhibitors, PARP (BRCA-mutant), FOLFOX/NALIRIFOX, KRAS-targeted vaccines.

Melanoma

~100,000 new cases/yr

BRAF/MEK, nivolumab+relatlimab (LAG-3), TIL therapy (lifileucel), anti-PD-1 adjuvant.

Hematologic

~180,000 new cases/yr

CAR-T curative intent, bispecific TCEs, BTK degraders, BCL-2 inhibitors, menin inhibitors.

Bladder/UC

~82,000 new cases/yr

FGFR3 inhibitors, EV+pembrolizumab (1L mUC), ADC combinations, nectin-4 targeting.

The 2025 Oncology Inflection Point

The defining shift of the current era is the collapse of the traditional histology-first treatment paradigm. Molecular profiling — not organ of origin — increasingly determines treatment selection across virtually every solid tumor type. The approvals of trastuzumab deruxtecan across multiple HER2-expressing histologies, pembrolizumab in TMB-high and MSI-H tumors regardless of site, and larotrectinib/entrectinib in NTRK-fusion positive cancers have made pan-tumor biomarker testing not merely optional but standard-of-care. Comprehensive genomic profiling (CGP) at diagnosis is now supported by NCCN guidelines across most major cancer types.

Simultaneously, the ADC platform has achieved pharmacologic critical mass. Trastuzumab deruxtecan's 5.6-month PFS improvement over physician's choice in pretreated HER2-low breast cancer — a population previously considered HER2-negative — has expanded the targetable universe of HER2 therapy to an estimated 60% of all metastatic breast cancer patients. The same bystander-killing pharmacology is being leveraged against TROP2, HER3, Nectin-4, PTK7, CDH6, and ROR1 across multiple histologies.

Select a cancer type from the navigation above to see detailed emerging therapy breakdowns, key trial data, biomarker profiles, and 2025 treatment frameworks.

mitotics.com · Pan-Cancer Emerging Therapies · Q2 2025 · Molecular Oncology Series

Lung Cancer — NSCLC & SCLC

The most molecularly stratified solid tumor type. NSCLC has more actionable driver alterations than any other cancer, with targeted therapies available for EGFR, ALK, ROS1, BRAF V600E, MET, RET, NTRK, KRAS G12C, EGFR exon 20, and HER2.

~238,000 new US cases/yr ~127,000 deaths/yr #1 cancer mortality (US) ~75% diagnosed advanced stage
1

Emerging & Recently Approved Therapies — NSCLC


FDA Approved 2024

KRAS G12C Inhibitors

Sotorasib (CodeBreaK 200), Adagrasib (KRYSTAL-1)
  • Sotorasib: ORR 28%, mPFS 5.6 mo vs docetaxel (CodeBreaK 200)
  • Adagrasib: ORR 43% in KRYSTAL-1 Phase 2 (1L combo data emerging)
  • Combination with SHP2 inhibitors (RMC-4630, TNO155) — Phase 1/2 active
  • KRAS G12C prevalence: ~13% of NSCLC (mostly adenocarcinoma)
  • Next-gen: D-1553, GDC-6036 with improved CNS penetration in development
FDA Approved 2024

EGFR Exon 20 Inhibitors

Amivantamab-vmjw (Rybrevant), Mobocertinib
  • Amivantamab + lazertinib (MARIPOSA): PFS HR 0.70 vs osimertinib in 1L EGFR+
  • Amivantamab + chemo (PAPILLON): 11.4 vs 6.7 mo PFS in exon 20 insertions
  • Exon 20 insertions: ~2–3% NSCLC, historically no targeted options
  • PAPILLON established amivantamab-chemo as new 1L standard for exon 20
  • MARIPOSA-2: amivantamab + chemo post-osimertinib — active Phase 3
Phase 2/3 Readout

HER2-Directed ADC

Trastuzumab Deruxtecan (T-DXd, DESTINY-Lung)
  • DESTINY-Lung02: ORR 49.0% in HER2-mutant NSCLC (6.4 mg/kg dose)
  • HER2 mutations (~3% NSCLC): exon 20 insertions, A775_G776YVMA most common
  • FDA approved 2022; now exploring 1L setting with DESTINY-Lung08
  • ILD/pneumonitis: grade ≥3 in ~5% — requires vigilant monitoring
  • Trastuzumab + pertuzumab (HER2 amplified): DESTINY-Lung04 ongoing
Phase 1/2 Active

MET Exon 14 & Amplification

Tepotinib, Capmatinib, Telisotuzumab Vedotin
  • METex14 skipping: ~3% NSCLC — tepotinib/capmatinib both FDA approved
  • Telisotuzumab vedotin (Teliso-V): MET-overexpressing NSCLC, ORR 35% Phase 2
  • MET amplification (de novo): high copy number predicts amivantamab response
  • Osimertinib resistance via MET amplification (~5–10%): tepotinib + osi combination
  • SAVANNAH trial: osi + savolitinib in MET-amplified post-osi EGFR+
Paradigm Shift

Tarlatamab — SCLC DLL3 BiTE

Tarlatamab-dlle (DLL3×CD3 BiTE) — FDA Approved 2024
  • First T-cell engager approved for any solid tumor — SCLC 2L+
  • DeLLphi-301: ORR 40%, mDOR 9.7 months in heavily pretreated ES-SCLC
  • DLL3 expressed on >80% SCLC tumor cells; minimal normal tissue expression
  • CRS and neurotoxicity manageable with step-up dosing and first-dose monitoring
  • DeLLphi-304: 1L LS-SCLC + chemoradiotherapy — Phase 3 ongoing
Pipeline — Phase 1/2

TROP2 ADC in NSCLC

Sacituzumab Govitecan (SG), Dato-DXd (Dato-116)
  • Datopotamab deruxtecan (Dato-DXd): TROPION-Lung01 — ORR 26% vs docetaxel
  • TROPION-Lung08: Dato-DXd + pembrolizumab vs chemo-pembro 1L non-squamous
  • TROP2 expressed in ~64% NSCLC — potential broad applicability
  • ILD signal requires careful monitoring; payload overlap with T-DXd in dual-exposed pts
  • SG in squamous NSCLC: EVOKE-02 combination data 2025
2

Key Pivotal Trials — Lung 2025


MARIPOSA / PAPILLON Phase 3

Amivantamab Combinations Redefining EGFR NSCLC

MARIPOSA demonstrated amivantamab + lazertinib superiority over osimertinib monotherapy in treatment-naïve EGFR-mutant NSCLC. PFS HR 0.70 establishes the first combination challenge to osimertinib 1L dominance since FLAURA. PAPILLON independently validated amivantamab + chemo in EGFR exon 20 insertions, a historically difficult-to-treat population.

HR 0.70PFS vs Osi
23.7 momPFS Ami+Laz
16.6 momPFS Osi
DeLLphi-301 / DeLLphi-304 Pivotal / Ph3

Tarlatamab — First Solid Tumor BiTE Approved

DeLLphi-301 established tarlatamab as the standard second-line option for ES-SCLC. ORR 40% and mDOR 9.7 months in an otherwise chemorefractory setting represents a meaningful clinical advance. DeLLphi-304 is now testing 1L tarlatamab + chemoradiotherapy in limited-stage SCLC with curative intent — a landmark trial for SCLC.

40%ORR
9.7 momDOR
FDA ✓2024 Approved
CodeBreaK 200 Phase 3

Sotorasib vs Docetaxel — KRAS G12C 2L NSCLC

CodeBreaK 200 demonstrated PFS superiority for sotorasib over docetaxel in 2L KRAS G12C+ NSCLC. OS was non-significant partly due to crossover, raising questions about optimal sequencing. Combination strategies (sotorasib + pembrolizumab showing increased hepatotoxicity; sotorasib + EGFR inhibition) are being explored to deepen responses and address primary resistance.

5.6 momPFS Soto
4.5 momPFS Doce
28%ORR
TROPION-Lung01 Phase 2/3

Datopotamab Deruxtecan vs Docetaxel — Pretreated NSCLC

TROPION-Lung01 confirmed Dato-DXd as a viable docetaxel alternative in pretreated non-squamous NSCLC. PFS HR 0.75 and improved tolerability profile. TROPION-Lung08, combining Dato-DXd + pembrolizumab as 1L treatment, has the potential to establish a chemo-free backbone — awaiting OS readout.

HR 0.75PFS vs Doce
26%ORR

Lung Cancer — 2025 Bottom Line

NSCLC now has more FDA-approved targeted therapies than any other cancer type — yet the majority of KRAS-mutant, STK11-mutant, and KEAP1-mutant patients still lack effective precision options. The amivantamab+lazertinib data from MARIPOSA represents the first credible challenge to osimertinib monotherapy in 1L EGFR+ NSCLC — pending OS maturity, this combination may become a new standard of care. The unresolved question is whether adding VEGFR (ramucirumab), MET (savolitinib), or bispecific targeting to osimertinib backbone can delay or prevent the inevitable C797S/MET resistance mechanisms.

In SCLC, tarlatamab's FDA approval is the most significant advance since atezolizumab was added to first-line chemo in 2019. DeLLphi-304 — testing tarlatamab in 1L limited-stage SCLC — represents potentially the highest-impact SCLC trial currently enrolling.

mitotics.com · Lung Cancer Emerging Therapies · Q2 2025

Breast Cancer

The ADC revolution has redefined metastatic breast cancer treatment. HER2-low, TROP2-targeted, and PI3K/AKT pathway therapies are transforming outcomes across HR+, HER2+, and TNBC subtypes.

~310,000 new US cases/yr ~42,000 deaths/yr ~15% TNBC ~60% HER2-low (IHC 1+ or 2+/ISH–)
1

Emerging & Recently Approved Therapies — Breast


Paradigm-Defining

T-DXd — HER2-Low & HER2 Ultralow

Trastuzumab Deruxtecan (ENHERTU) — DESTINY-Breast04/06
  • DESTINY-Breast04: PFS HR 0.50, OS HR 0.64 vs physician's choice in HER2-low
  • Expanded HER2-low definition covers ~60% of all mBC patients
  • DESTINY-Breast06: HER2 ultralow (IHC 0 with incomplete staining) — PFS HR 0.63
  • ILD remains the critical AE — grade ≥3 ~5.9%; requires institutional monitoring protocols
  • DESTINY-Breast09: 1L mBC — T-DXd vs taxane+trastuzumab in HER2+ (active)
FDA Approved 2023

Sacituzumab Govitecan (SG) — TROP2 ADC

Trodelvy — ASCENT, TROPiCS-02
  • ASCENT: OS 12.1 vs 6.7 mo in mTNBC; ORR 35% (2L+)
  • TROPiCS-02: PFS HR 0.66 in HR+/HER2– after CDK4/6 + ET failure
  • FDA approved in both TNBC and HR+/HER2– mBC settings
  • SG + pembrolizumab (SACI-IO) — Phase 3 1L TNBC PD-L1+ results 2025
  • TROP2 expression: ~88% TNBC, ~80% HR+ — broad applicable population
FDA Approved 2023

Inavolisib + Palbociclib + Fulvestrant

INAVO120 — PIK3CA-mutated HR+/HER2– mBC
  • INAVO120: mPFS 15.0 vs 7.3 mo vs palbo+fulvestrant; HR 0.43
  • PIK3CA mutations: ~40% HR+/HER2– mBC — highly actionable target
  • Inavolisib uniquely degrades mutant PI3Kα selectively (vs alpelisib's class-I inhibition)
  • Improved hyperglycemia profile vs alpelisib — significant tolerability advantage
  • PI3K-AKT-mTOR pathway: now targetable with PIQRAY, KOSELUGO, everolimus combinations
Phase 3 Data 2025

Elacestrant — ESR1-Mutant mBC

Orserdu (EMERALD) — ESR1-Mutant HR+/HER2– mBC
  • EMERALD: mPFS 3.8 vs 1.9 mo in ESR1-mutant subgroup; HR 0.55
  • First oral SERD approved — addresses CDK4/6+AI acquired resistance via ESR1 mutation
  • ESR1 mutations: ~30–40% HR+ mBC after AI progression (liquid biopsy detected)
  • ELEVATE: elacestrant + abemaciclib Phase 3 — key combination trial
  • Competing oral SERDs: amcenestrant, camizestrant, giredestrant, imlunestrant in Ph3
Phase 2/3 Active

Capivasertib + Fulvestrant — AKT Inhibitor

CAPItello-291 — AKT pathway altered HR+/HER2– mBC
  • CAPItello-291: mPFS 7.3 vs 3.1 mo (AKT pathway altered); HR 0.50
  • AKT1/PIK3CA/PTEN alterations: ~50% HR+ mBC — large addressable population
  • FDA approved 2023 — use with ctDNA/tissue profiling to identify altered patients
  • Combination with CDK4/6 or T-DXd under exploration
  • Hyperglycemia + diarrhea principal AEs; on-target toxicity predictable
Phase 1/2 Pipeline

HER3-DXd, PTK7 ADC, B7-H3 ADC

Patritumab Deruxtecan, Cofetuzumab Pelidotin, DS-7300
  • HER3-DXd (patritumab DXd): ORR 30% in pretreated HER3+ mBC (Phase 2 DASH)
  • HER3 near-universally expressed in breast cancer — broad potential target
  • B7-H3 (DS-7300, ifinatamab deruxtecan): TNBC Phase 2 data emerging 2025
  • PTK7 ADC (cofetuzumab pelidotin): TNBC + HR+ studies active
  • Multiple ADC combinations under study — sequencing and cross-resistance key open questions

Breast Cancer — 2025 Bottom Line

The HER2-low paradigm established by DESTINY-Breast04 is arguably the most consequential breast cancer approval since trastuzumab. Redefining ~60% of all mBC patients as HER2-targetable changes the fundamental treatment architecture of the disease. DESTINY-Breast06 has extended this further to HER2 ultralow — a population that was previously classified as HER2-zero and thus ineligible. The pending question is whether 1L T-DXd (DESTINY-Breast09) can displace taxane+trastuzumab in HER2-positive disease.

In HR+/HER2– disease, the convergence of CDK4/6 inhibitors, oral SERDs (elacestrant and next-generation), PI3K/AKT inhibitors, and ADCs creates an unprecedented number of sequencing options after CDK4/6+AI failure — but also a growing challenge of evidence-based sequencing in clinical practice. ESR1 mutation testing via liquid biopsy at progression is now essential.

mitotics.com · Breast Cancer Emerging Therapies · Q2 2025

Colorectal Cancer (CRC)

CRC molecular profiling has matured significantly. KRAS G12C is now actionable, HER2 amplification is targetable, BRAF V600E has approved combinations, and MSI-H has checkpoint blockade as frontline standard.

~153,000 new US cases/yr ~52,000 deaths/yr ~40–45% KRAS-mutant ~5% MSI-H
1

Emerging & Recently Approved Therapies — CRC


FDA Approved 2023

Sotorasib + Cetuximab — KRAS G12C CRC

CodeBreaK 300 — KRAS G12C mCRC
  • CodeBreaK 300: ORR 26.4% (vs 0% TAS-102 or regorafenib); mPFS 5.6 mo
  • KRAS G12C CRC: ~3–4% mCRC — combination essential to overcome feedback signaling
  • Cetuximab required to block EGFR-mediated reactivation of KRAS pathway
  • Adagrasib + cetuximab (KRYSTAL-10): ORR 34%, mPFS 6.9 mo — comparably active
  • Pan-RAS inhibitor combinations (RMC-6236 + EGFR) in Phase 1 development
Standard of Care Shift

Pembrolizumab 1L — MSI-H mCRC

KEYNOTE-177 — dMMR/MSI-H mCRC
  • KEYNOTE-177: PFS HR 0.60, ORR 43.8% vs FOLFOX/FOLFIRI+bev/cet
  • dMMR/MSI-H: ~5% mCRC, but >20% right-sided colon cancers in certain populations
  • OS benefit confirmed — pembrolizumab is unambiguous 1L standard in MSI-H
  • Nivolumab + ipilimumab (CheckMate 142): ORR 55% in MSI-H — dual checkpoint active
  • MSI-H testing now universal standard at mCRC diagnosis per NCCN guidelines
Phase 2/3 Active

HER2-Directed Therapy in mCRC

T-DXd (DESTINY-CRC02), Tucatinib+Trastuzumab (MOUNTAINEER)
  • MOUNTAINEER: trastuzumab + tucatinib — ORR 38.1% in HER2+ RAS-WT mCRC
  • Tucatinib + trastuzumab FDA approved 2023 (1st HER2-targeted approval in CRC)
  • DESTINY-CRC02: T-DXd ORR 37–45% in HER2-overexpressing mCRC; Phase 2
  • HER2 amplification: ~2–3% mCRC (higher in RAS/RAF wild-type right-sided tumors)
  • MOUNTAINEER-03: tucatinib + trastuzumab + FOLFOX Phase 3 — 1L HER2+ mCRC
FDA Approved 2022

Encorafenib + Cetuximab — BRAF V600E

BEACON CRC — BRAF V600E mCRC (2L+)
  • BEACON CRC: OS 9.0 vs 5.4 mo (triplet vs control); ORR 26% doublet
  • BRAF V600E: ~8–12% mCRC — historically worst prognosis subtype
  • ANCHOR CRC: encorafenib+cetuximab+mFOLFOX6 1L Phase 2 — ORR 48%
  • BREAKWATER: 1L randomized Phase 3 vs FOLFOX/FOLFIRI — primary endpoint OS
  • BRAF V600E testing at mCRC diagnosis now standard — guides immediate 1L decision
Phase 1/2 Pipeline

Claudin18.2 ADC & Bispecifics

Zolbetuximab, AMG 910, IMAB362
  • CLDN18.2 expressed in ~25–30% CRC (highest in mucinous/signet ring histologies)
  • Zolbetuximab: SPOTLIGHT/GLOW established CLDN18.2 as actionable in gastric/GEJ (active CRC studies)
  • AMG 910 (CLDN18.2×CD3 bispecific): Phase 1 in GI tumors including CRC
  • CEA-CD3 bispecific (cibisatamab): Phase 1/2 in CEA+ mCRC
  • GUCY2C targeted therapy (indium-DOTA-5F3): Phase 1 colorectal
Phase 2 Emerging

Fruquintinib + Panitumumab (RAS-WT)

FRUITFUL — Later-line mCRC
  • Fruquintinib: selective VEGFR 1/2/3 inhibitor — FRESCO-2 confirmed OS benefit
  • Fruquintinib FDA approved 2023 as later-line option regardless of RAS status
  • FRUITFUL: fruquintinib + panitumumab — RAS-WT mCRC later lines
  • RAS/RAF wild-type population: residual EGFR sensitivity after prior EGFR exposure
  • Liquid biopsy RAS reversion monitoring enabling rechallenge strategies

Colorectal Cancer — 2025 Bottom Line

CRC has crossed a threshold where molecular profiling at diagnosis directly changes treatment selection for ≥30% of newly diagnosed metastatic patients — MSI-H (pembrolizumab 1L), BRAF V600E (encorafenib+cetuximab), KRAS G12C (sotorasib/adagrasib+cetuximab), HER2+ (tucatinib+trastuzumab). Universal comprehensive genomic profiling at mCRC diagnosis is no longer an academic consideration — it is required to implement standard of care.

The KRAS G12C breakthrough (CodeBreaK 300, KRYSTAL-10) is particularly consequential because it validates combination KRAS+EGFR co-targeting as a pharmacologic principle — demonstrating that KRAS inhibitor monotherapy is insufficient in CRC due to EGFR-mediated feedback reactivation. This principle is informing rational pan-RAS+EGFR combination design for G12D and G12V mutations.

mitotics.com · Colorectal Cancer Emerging Therapies · Q2 2025

Prostate Cancer

mCRPC therapy has been transformed by PSMA radioligand therapy, PARP inhibitors in HRR-deficient patients, and evolving AR-targeted approaches. The PSMA theranostic platform is the most impactful prostate development of the decade.

~299,000 new US cases/yr ~35,000 deaths/yr ~25% HRR-deficient (BRCA1/2, ATM) ~80% PSMA+ in mCRPC
1

Emerging & Recently Approved Therapies — Prostate


Theranostic Paradigm

Lu-177 PSMA-617 (Pluvicto)

VISION Trial — Post-ARPI, Post-Taxane mCRPC
  • VISION: OS 15.3 vs 11.3 mo; rPFS HR 0.40 — landmark FDA approval 2022
  • PSMAfore: Lu-PSMA vs ARPI switch in ARPI-experienced pre-taxane — rPFS HR 0.41
  • PSMA PET/CT (18F-DCFPyL, 68Ga-PSMA-11) required for patient selection
  • ~80% of mCRPC PSMA-positive on PET — broad eligible population
  • PSMAddition: 1L mHSPC + docetaxel — Phase 3 OS results 2025
Next-Gen RLT

Actinium-225 PSMA (Alpha Emitter)

Ac-225 PSMA-617 — Lu-PSMA Refractory
  • Retrospective data: ORR >60% in Lu-177-PSMA refractory patients
  • Alpha particles: ~60–80x higher LET than beta — more lethal per decay
  • TheraP-α: randomized Phase 2 Ac-225 vs Lu-177 in mCRPC — primary data 2025
  • Salivary gland toxicity (xerostomia) primary dose-limiting concern
  • PSMA-targeted bispecific (AMG 160, LNCB79): Phase 1 in PSMA+ mCRPC
FDA Approved 2023

PARP Inhibitors — HRR-Deficient mCRPC

Olaparib (PROfound), Rucaparib, Niraparib+Abiraterone
  • PROfound: olaparib vs enzalutamide/abiraterone — OS HR 0.69 in BRCA1/2
  • Olaparib + abiraterone (PROpel): rPFS HR 0.66 in unselected mCRPC (Phase 3)
  • Niraparib + abiraterone (MAGNITUDE): rPFS benefit in HRR+ patients
  • TALAPRO-2: talazoparib + enzalutamide regardless of HRR — Phase 3
  • Germline + somatic BRCA1/2/ATM/CDK12 testing now standard at mCRPC diagnosis
Phase 2/3 Active

AR Degraders & Next-Gen AR Pathway

ARV-110 (Bavdegalutamide), NVP-ABI-231, ODM-208
  • AR point mutations (F877L, T878A): ~20–30% enzalutamide/abiraterone resistance mechanism
  • ARV-110: PROTAC-based AR degrader — Phase 2 ORR 46% in AR T878x/H875Y mutants
  • ODM-208 (CYP11A1 inhibitor): blocks all steroid biosynthesis upstream of AR ligands
  • Bipolar androgen therapy (BAT): high-dose testosterone cycling — Phase 2 data intriguing
  • ARPI sequencing: enzalutamide → abiraterone (or vice versa) — minimal clinical benefit
Phase 1/2 Pipeline

PSMA-Directed ADC & Bispecific

PSMA-ADC (AMG 160, JNJ-63898081), FPI-2265
  • AMG 160 (PSMA×CD3 BiTE): PSA declines in heavily pre-treated mCRPC Phase 1
  • FPI-2265: Ac-225 PSMA — alpha-emitter Phase 1, strong PSA responses
  • PSMA-targeting expanding beyond prostate: bladder, salivary, neuroendocrine
  • STEAP1-ADC (STEAP1×AUR103): Phase 1 in mCRPC (STEAP1 highly prostate-expressed)
  • DLL3-targeted RLT: neuroendocrine prostate (NEPC) — emerging target in treatment-emergent NEPC
Phase 3 Active

Pembrolizumab Combinations — mCRPC

KEYNOTE-641, KEYNOTE-991 — Pembro + ARPI
  • KEYNOTE-991: pembrolizumab + enzalutamide vs enza alone in 1L mCRPC — Phase 3
  • CDK4/6 inhibitors (palbociclib) + ARPI: Phase 2 combinations (PALADIN trial)
  • MSI-H prostate (~3–5%): pembrolizumab highly active — universal NGS to identify
  • CDK12-mutant: biallelic loss ~7% mCRPC — ipilimumab+nivolumab showed signals
  • Cabozantinib: MET/VEGFR2/RET inhibitor — COMET-1 negative but combinations active

Prostate Cancer — 2025 Bottom Line

The PSMA theranostic platform — PET/CT for selection, lutetium for treatment — has become the defining advance in mCRPC management. PSMAfore's data establishing Lu-177-PSMA before docetaxel (rPFS HR 0.41) positions radioligand therapy as potentially a second-line standard before chemotherapy, restructuring the mCRPC treatment sequence. If PSMAddition's OS results demonstrate benefit in the mHSPC 1L setting, PSMA-directed therapy will move into the hormone-sensitive disease space.

The alpha emitter (Ac-225 PSMA) data in Lu-177-refractory patients represents the most active therapeutic signal in that population and may define a sequential RLT paradigm. Combining PSMA radioligand therapy with PARP inhibitors (for DNA damage repair synergy) and AR pathway agents is the most scientifically rational combination strategy and is actively being explored.

mitotics.com · Prostate Cancer Emerging Therapies · Q2 2025

Pancreatic Cancer (PDAC)

PDAC remains the hardest solid tumor to treat, with 5-year survival ~12%. KRAS is mutated in ~95% of cases. KRAS G12D inhibitors, PARP therapy for BRCA-mutant disease, and mRNA neoantigen vaccines represent the most promising emerging directions.

~66,000 new US cases/yr ~51,000 deaths/yr ~95% KRAS-mutant ~7–8% BRCA1/2 germline
1

Emerging Therapies — Pancreatic Cancer


Historic Breakthrough

KRAS G12D Inhibitors — MRTX1133, RMC-9805

Phase 1 — KRAS G12D PDAC (G12D = ~40% of all PDAC)
  • MRTX1133: first selective KRAS G12D inhibitor — Phase 1 enrollment active (first-in-class)
  • RMC-9805 (Revolution Medicines): covalent tri-complex KRAS G12D inhibitor — Phase 1 data 2025
  • HRS-4642: Phase 1 KRAS G12D-selective, oral — initial responses in PDAC reported
  • Pan-RAS inhibitor RMC-6236: active against G12D, G12V, G12R — Phase 1 solid tumors
  • KRAS inhibitor + mRNA vaccine (mRNA-5671) — personalized neoantigen combination in PDAC
Standard of Care

NALIRIFOX / FOLFIRINOX — 1L mPDAC

NAPOLI-3 — NALIRIFOX vs Gem+nab-PTX
  • NAPOLI-3: OS 11.1 vs 9.2 mo NALIRIFOX vs gem+nab-PTX — first OS improvement in >10 yrs
  • NALIRIFOX (nanoliposomal irinotecan + oxaliplatin + leucovorin + 5-FU) — new 1L preferred
  • FOLFIRINOX remains active alternative in fit patients where NALIRIFOX unavailable
  • Germline BRCA testing at diagnosis — affects maintenance olaparib eligibility (POLO)
  • Olaparib maintenance post-FOLFOX (POLO): PFS 7.4 vs 3.8 mo in BRCA1/2 germline+ PDAC
Immunotherapy Breakthrough

Autogene Cevumeran — mRNA KRAS Neoantigen Vaccine

BNT122 + atezolizumab + mFOLFIRINOX — Adjuvant PDAC
  • Phase 2 (Rojas et al. Nature 2023): 50% patients generated strong T-cell responses post-vaccine
  • T-cell responders: 18-month RFS rate significantly higher vs non-responders (HR 0.08)
  • Personalized vaccine targeting individual KRAS mutant neoepitopes — patient-specific manufacturing
  • Phase 3 adjuvant PDAC trial: BNT111 + atezolizumab vs atezolizumab alone now enrolling
  • If confirmed, first immunotherapy advance in adjuvant pancreatic cancer in decades
Phase 1/2 Active

Claudin18.2 ADC in PDAC

Zolbetuximab, CMG901, CLDN18.2-targeted strategies
  • CLDN18.2 expressed in ~40–60% PDAC — among highest solid tumor expression
  • Zolbetuximab (CLDN18.2 monoclonal): activity in CLDN18.2+ GI tumors including PDAC Phase 1
  • CMG901 (CLDN18.2 ADC): Phase 1 PDAC with ORRs in CLDN18.2+ patients
  • CLDN18.2×CD3 bispecific: AMG 910 Phase 1 including PDAC cohort
  • Selection by IHC CLDN18.2 ≥2+ in ≥40% cells — standardized cutoff under development
Preclinical / Phase 1

FAPI Radioligand Therapy

Lu-177-FAPI, Ac-225-FAPI — FAP-expressing Desmoplastic Tumors
  • FAP (fibroblast activation protein): highly expressed in PDAC desmoplastic stroma — >90%
  • Lu-177-FAPI-46: Phase 1/2 PDAC — FAPi PET identifies PDAC lesions with high sensitivity
  • Theranostic FAPi platform: same ligand for PET staging and therapeutic delivery
  • Stroma targeting addresses immunosuppressive TME unique challenge in PDAC
  • FAPI-based CAR-T: targeting FAP+ stromal cells to remodel TME — preclinical stage
Phase 2 Active

Anti-EGFR + KRAS Combination (RAS-Independent Targeting)

Pembrolizumab + KRAS G12C/D Inhibitor Combinations
  • RMC-6236 (pan-KRAS) + pembrolizumab: Phase 1 PDAC and other KRAS-mutant solid tumors
  • Rationale: KRAS inhibition enhances MHC-I antigen presentation — immunotherapy sensitizer
  • KRAS G12C inhibitor + anti-PD-1: CodeBreaK 101 includes PDAC sub-cohort
  • Sotorasib + AMG 510 in KRAS G12C PDAC (rare ~1–2%) — early activity seen
  • Combination with tumor microenvironment remodeling (anti-CCR2, anti-CXCL5) — preclinical

Pancreatic Cancer — 2025 Bottom Line

PDAC stands at a historic inflection point. KRAS has been mutated in ~95% of PDAC for 40 years and considered entirely undruggable — the validation of KRAS G12C inhibition in NSCLC and CRC, combined with first-in-human KRAS G12D inhibitor data now emerging from Phase 1, represents the most consequential mechanistic advance in pancreatic oncology since gemcitabine. KRAS G12D (~40% PDAC), G12V (~32%), and G12R (~16%) collectively cover ~88% of KRAS-mutant PDAC — the pipeline now targets all three.

The autogene cevumeran mRNA vaccine data — demonstrating that 50% of adjuvant PDAC patients mounted measurable T-cell immunity against their personal KRAS neoantigens, with the T-cell responders showing dramatically improved recurrence-free survival — is the strongest signal for immunotherapy in PDAC ever reported. The Phase 3 confirmatory trial is the most important PDAC trial currently enrolling.

mitotics.com · Pancreatic Cancer Emerging Therapies · Q2 2025

Melanoma

Melanoma led the immunotherapy revolution and continues to pioneer new checkpoints. LAG-3 combination, TIL therapy, and neoadjuvant immunotherapy with pathologic response as a surrogate endpoint are the defining 2025 advances.

~100,000 new US cases/yr ~8,000 deaths/yr ~45% BRAF V600E/K ~25% 5-yr OS stage IV (pre-IO era)
1

Emerging Therapies — Melanoma


LAG-3 Checkpoint

Nivolumab + Relatlimab (Opdualag)

RELATIVITY-047 — Untreated Advanced Melanoma
  • RELATIVITY-047: PFS 10.1 vs 4.6 mo; HR 0.75 vs nivolumab alone — FDA approved 2022
  • LAG-3 inhibition: third checkpoint (after PD-1 and CTLA-4) to achieve clinical validation
  • Activity regardless of PD-L1 expression — LAG-3 expression enriches for benefit
  • RELATIVITY-098: adjuvant nivolumab+relatlimab vs nivolumab — Phase 3 resected
  • LAG-3 combinations in CRC, NSCLC, hepatocellular actively enrolling
FDA Approved 2024

Lifileucel — TIL Therapy (Amtagvi)

C-144-01 — Ipilimumab and Anti-PD-1 Refractory Melanoma
  • C-144-01: ORR 31.5% (per independent review) in IO-refractory advanced melanoma
  • First TIL therapy FDA-approved for any cancer — landmark cellular therapy approval 2024
  • Manufacturing: tumor biopsy → ex vivo expansion → lymphodepletion → infusion (~22 days)
  • mDOR not reached at median 18.6 mo follow-up — durable responses in a fraction
  • IOV-3001: next-gen TIL product — Phase 2 earlier-line settings (pre-IO failure)
Standard of Care 2022

Pembrolizumab Adjuvant — Resected Stage IIB/C

KEYNOTE-716 — High-Risk Resected Melanoma
  • KEYNOTE-716: RFS HR 0.61 in stage IIB/C — expanded adjuvant indication 2022
  • Previously adjuvant immunotherapy reserved for stage III — now stage IIB/C eligible
  • Dabrafenib + trametinib adjuvant: COMBI-AD — RFS HR 0.49 in BRAF V600E/K stage III
  • NIBIT-MESO-2: neo/adjuvant tremelimumab+durvalumab — resectable melanoma
  • Neoadjuvant IO (SWOG S1801): pembro neoadjuvant > adjuvant in resectable stage IIIB–IV
mRNA Vaccine

mRNA-4157 + Pembrolizumab — Neoantigen Vaccine

KEYNOTE-942 — Adjuvant Resected High-Risk Melanoma
  • KEYNOTE-942: RFS HR 0.56 (pembro + mRNA-4157 vs pembro alone) — Phase 2
  • First mRNA therapeutic cancer vaccine to show randomized RFS benefit in any cancer
  • Individualized: 34 neoantigens selected per patient from tumor WES + RNA sequencing
  • KEYNOTE-942 Phase 3 (V940-001): 1,089-patient confirmatory trial — ongoing
  • Expanding to NSCLC, TNBC, renal cell — broad neoantigen vaccine platform development
Phase 1/2 Active

TIGIT & TIM-3 Checkpoint Combinations

Tiragolumab, Domvanalimab, Sabatolimab — Anti-PD-1 Combinations
  • TIGIT inhibitors: phase 3 failures in lung (SKYSCRAPER-01, CITYSCAPE) — mechanism intact
  • Domvanalimab (Fc-silent anti-TIGIT): STAR-121 Phase 3 melanoma with pembro ongoing
  • TIM-3 (sabatolimab + pembro): early response signals in solid tumors including melanoma
  • Melanoma as immunotherapy pioneer: LAG-3 now validated — TIGIT/TIM-3 credible next
  • Intratumoral IL-2 (PEGylated, mTOR-engineered): cis-delivery approaches in IO-refractory
Phase 2 Mucosal/Uveal

Rare Melanoma Subtypes — Unmet Needs

Uveal, Mucosal, Acral — Distinct Molecular Pathways
  • Uveal melanoma: GNAQ/GNA11 mutations (~90%) — tebentafusp (gp100×CD3 bispecific) OS benefit Phase 3
  • Tebentafusp: first OS benefit ever demonstrated in uveal melanoma — FDA approved 2022
  • Mucosal melanoma: KIT mutations (~30%), immunotherapy less effective than cutaneous
  • Imatinib in KIT-mutant mucosal: ORR ~20% — niche but validated signal
  • Acral melanoma: CDK4 amplification, lower TMB — less IO-responsive, targeted approaches needed

Melanoma — 2025 Bottom Line

Melanoma continues to be the proving ground for cancer immunotherapy. The mRNA-4157 neoantigen vaccine data from KEYNOTE-942 — a 44% reduction in recurrence risk when added to adjuvant pembrolizumab — is the strongest signal for therapeutic cancer vaccination ever reported in a randomized trial. If the Phase 3 confirmatory trial (V940-001) replicates this, individualized mRNA cancer vaccines will enter the oncology mainstream with immediate applicability to other cancer types including NSCLC and TNBC.

Lifileucel's FDA approval as the first TIL therapy in any cancer is a regulatory milestone, even with a modest 31.5% ORR in a heavily pre-treated population. The precedent — that expanded autologous tumor-infiltrating lymphocytes can achieve durable responses in IO-refractory patients — is directly enabling next-generation TIL engineering (PD-1 knockout, cytokine armoring, TCR-enhanced) across multiple solid tumors.

mitotics.com · Melanoma Emerging Therapies · Q2 2025

Hematologic Malignancies

CAR-T cell therapy, bispecific T-cell engagers, BTK degraders, BCL-2/BCL-XL inhibitors, and menin inhibitors are collectively redefining the treatment landscape across AML, ALL, multiple myeloma, NHL, and CLL.

~180,000 new US cases/yr ~57,000 deaths/yr Multiple CAR-T approvals 2021–25 ~6 bispecific TCE approvals 2022–25
1

Emerging Therapies — Hematologic Malignancies


CAR-T Standard of Care

BCMA CAR-T & Bispecifics — Multiple Myeloma

Ide-cel (bb2121), Cilta-cel (JNJ-4528), Teclistamab, Elranatamab
  • Cilta-cel (CARTITUDE-4): PFS HR 0.26 vs daratumumab+pomalidomide in 1–3 prior lines
  • CARTITUDE-4 data: 24-month PFS 60% vs 41% — CAR-T now entering earlier myeloma lines
  • Teclistamab (BCMA×CD3 BiTE): ORR 63% in heavily pretreated 5L+ myeloma
  • Elranatamab (BCMA×CD3): ORR 61% (MagnetisMM-3) — competitive data
  • Talquetamab (GPRC5D×CD3): ORR 70%+ — GPRC5D avoids BCMA-resistance mechanisms
Curative Intent AML

Menin Inhibitors — KMT2A/NPM1 AML

Revumenib (AUGMENT-101), Ziftomenib — R/R AML
  • Revumenib: ORR 63%, CR/CRh 23% in KMT2A-rearranged R/R AML — FDA approved 2024
  • KMT2A rearrangements: ~10% AML, ~30% infant ALL — historically poor prognosis
  • NPM1-mutant AML: second major menin inhibitor indication — ziftomenib Phase 2 active
  • Differentiation syndrome: ~25% — manageable with early recognition and steroids
  • Menin inhibitor + azacitidine/venetoclax combinations: Phase 1/2 intensive development
CLL/SLL Standard

BTK Inhibitors & Degraders — CLL

Zanubrutinib, Acalabrutinib, NX-2127 (BTK PROTAC)
  • ALPINE: zanubrutinib > ibrutinib (ORR 80% vs 73%, less A-fib) — preferred BTK inhibitor
  • Venetoclax + obinutuzumab (CLL14): MRD-guided fixed-duration — time-limited alternative
  • NX-2127 (BTK PROTAC/degrader): Phase 1 — degrades BTK C481S/C481F resistance mutations
  • BTK degraders designed to overcome ibrutinib/acalabrutinib C481 resistance (~50% BTKi failure)
  • Pirtobrutinib (non-covalent BTKi): ORR 67% post-covalent BTKi failure — approved 2023
Phase 2/3 Active

CD20 Bispecifics — Diffuse Large B-Cell Lymphoma

Epcoritamab, Glofitamab, Mosunetuzumab — R/R DLBCL
  • Epcoritamab + R-CHOP: EPCORE NHL-2 Phase 3 — 1L DLBCL (results 2025)
  • Glofitamab: 35% CR rate in R/R DLBCL — fixed 12-cycle duration FDA approved 2023
  • Mosunetuzumab: ORR 60% in R/R follicular lymphoma — FDA approved 2022
  • Bispecific before or after CAR-T: sequencing optimization actively being studied
  • Dual-target (CD20+CD19) bispecific approaches to prevent antigen escape — emerging pipeline
Phase 3 Active

Ivosidenib/Enasidenib — IDH-Mutant AML

AGILE, Phase 3 IDH1/2 Inhibitor Combinations
  • Ivosidenib + azacitidine (AGILE): OS 24.0 vs 7.9 mo in IDH1-mutant AML — frontline
  • IDH1 mutations: ~8% AML; IDH2 mutations: ~12% AML — significant populations
  • Enasidenib (IDH2 inhibitor): FDA approved R/R setting; combinations with AZA ongoing
  • Differentiation syndrome: class effect requiring vigilant monitoring
  • IDH inhibitor + venetoclax + AZA: triple combination Phase 1/2 highly active
Phase 1/2 Pipeline

BCL-XL Inhibitors & Navitoclax — AML/MF

Navitoclax, Pelabresib, BM-1197 — AML & Myelofibrosis
  • Navitoclax + ruxolitinib (TRANSFORM-1): SVR35 67% vs rux alone in MF — Phase 3
  • BCL-XL inhibition limited by thrombocytopenia (on-target, platelet-dependent killing)
  • Pelabresib (BET inhibitor) + ruxolitinib: MANIFEST-2 Phase 3 MF — data 2025
  • Imetelstat (telomerase inhibitor): OS benefit in low/int-1 MDS — FDA approved 2024
  • Magrolimab (anti-CD47 "don't-eat-me"): Phase 3 MDS+AZA vs AZA alone — results pending

Hematologic Malignancies — 2025 Bottom Line

CAR-T cell therapy has moved from salvage therapy to earlier lines of treatment in multiple myeloma — CARTITUDE-4's PFS HR of 0.26 in patients with only 1–3 prior lines essentially establishes ciltacabtagene autoleucel as a potentially curative-intent intervention in transplant-ineligible myeloma patients. The convergence of CAR-T (cidta-cel, ide-cel) and bispecific TCEs (teclistamab, elranatamab, talquetamab) has created a completely new treatment architecture for relapsed myeloma that renders older triplet regimens largely obsolete.

In AML, the menin inhibitor class (revumenib, ziftomenib) represents the most mechanistically novel advance in decades — directly reversing the transcriptional program driven by KMT2A rearrangements that has resisted targeted therapy for 30 years. Combination with venetoclax+azacitidine backbone is now the priority development direction.

mitotics.com · Hematologic Malignancies Emerging Therapies · Q2 2025

Bladder / Urothelial Cancer (UC)

The EV+pembrolizumab combination (EV-302) has become the new 1L mUC standard, ending cisplatin-based doublet dominance. FGFR3 inhibitors and antibody-drug conjugates dominate the pipeline.

~82,000 new US cases/yr ~17,000 deaths/yr ~20% FGFR3-altered (MIBC/mUC) >90% Nectin-4 expression
1

Emerging Therapies — Bladder / Urothelial Cancer


New 1L Standard

Enfortumab Vedotin + Pembrolizumab (EV-302)

EV-302/KEYNOTE-A39 — 1L Cisplatin-Eligible & Ineligible mUC
  • EV-302: OS HR 0.47 vs gem+platinum — most impactful mUC OS data in decades
  • mOS 31.5 vs 16.1 mo; mPFS 12.5 vs 6.3 mo; ORR 68% vs 44%
  • FDA approved December 2023 — regardless of cisplatin eligibility (eliminates fitness stratification)
  • Enfortumab: Nectin-4 ADC (auristatin payload); >90% UC Nectin-4 expression
  • EV-103: EV+pembro in cisplatin-ineligible — confirmed the landmark EV-302 findings
FDA Approved 2023

Erdafitinib — FGFR3-Altered mUC

THOR — Post-Immunotherapy FGFR3-Altered mUC
  • THOR: OS 12.1 vs 7.8 mo vs chemo in FGFR3-altered post-IO mUC — Phase 3 confirmed
  • FGFR3 mutations/fusions: ~20% mUC — pan-FGFR inhibitor erdafitinib approved 2019 (updated)
  • Futibatinib (FGFR1-4 irreversible): Phase 1/2 FGFR3-altered UC active
  • Derazantinib (FGFR1/2/3): Phase 2 FGFR3+ upper tract urothelial carcinoma
  • FGFR3 testing now standard at mUC diagnosis for 2L+ treatment selection
Phase 3 Active

Sacituzumab Govitecan — 2L mUC

TROPiCS-04 — Post-Platinum, Post-IO mUC
  • TROPiCS-04: SG vs taxane or vinflunine in 2L+ mUC — OS primary endpoint
  • Phase 2 data: ORR 29.1% — competitive with existing 2L options
  • TROP2 expressed in ~83% UC — broad applicable population
  • SG + pembrolizumab: Phase 1/2 combination data — ORR ~65% in small series
  • Post-EV-pembro failure population: significant unmet need emerging as 1L shifts
Phase 2 Active

HER2-Directed Therapy — HER2+ mUC

T-DXd (DESTINY-PanTumor02), Disitamab Vedotin
  • DESTINY-PanTumor02: T-DXd ORR 39.0% in HER2+ UC (IHC 3+ or 2+/ISH+)
  • HER2 overexpression: ~10–15% UC — underappreciated targetable population
  • Disitamab vedotin (RC48): HER2 ADC ORR 51% in HER2+ mUC Phase 2 — Chinese trial
  • RC48 global Phase 3 (DisitaMab Vedotin-022): vs chemo in HER2+ mUC enrolling
  • Trastuzumab + pertuzumab: modest activity; ADC approach clearly superior
Phase 2 Active — NMIBC

BCG-Unresponsive NMIBC — Novel Bladder-Directed

Pembrolizumab (KEYNOTE-057), Cretostimogene, N-803
  • KEYNOTE-057: pembro CR rate 40.9% in BCG-unresponsive CIS — FDA approved 2020
  • Cretostimogene + BCG: intravesical adenoviral CECT/BCG — Phase 3 (BOND-003) results 2025
  • N-803 (IL-15 superagonist) + BCG: QUILT-3.032 — CR 71% in BCG-unresponsive CIS
  • Intravesical delivery avoids systemic exposure — critical advantage in elderly population
  • TAR-200 (sustained-release intravesical gemcitabine): SunRISe-1 — Phase 2/3 NMIBC
Phase 1/2 Pipeline

CDH6 ADC, HER3 ADC — UC Pipeline

Patritumab DXd, CMG901, ifinatamab deruxtecan
  • HER3 expression: ~70% UC — patritumab deruxtecan (HER3-DXd) Phase 2 GU cohort active
  • B7-H3 (DS-7300): ~85% UC expression — Phase 1/2 urothelial cohort
  • Ifinatamab deruxtecan (I-DXd, B7-H3 ADC): Phase 2 HERTHENA-UC — ORR data 2025
  • Combination strategies: EV+SG payload overlap being explored cautiously
  • Post-EV + post-pembro failure: rapidly becoming the predominant clinical challenge as 1L shifts

Bladder / Urothelial Cancer — 2025 Bottom Line

EV-302 has fundamentally restructured mUC treatment. An OS HR of 0.47 versus gemcitabine-platinum — and an ORR of 68% in an all-comer population — makes EV+pembrolizumab one of the most impactful combination approvals in solid tumor oncology of the past decade. The fact that this combination works regardless of cisplatin eligibility eliminates the fitness-based treatment bifurcation that complicated mUC management for 20 years.

The critical emerging challenge is now defining optimal treatment after EV+pembrolizumab failure — a population that will grow substantially as 1L EV-pembro becomes universal. Sacituzumab govitecan, HER2-directed ADCs, and FGFR3 inhibitors (for the ~20% FGFR3-altered subpopulation) are the leading candidates for this emerging post-EV-pembro treatment space.

mitotics.com · Urothelial Cancer Emerging Therapies · Q2 2025

Ovarian Cancer

PARP inhibitors dominate frontline maintenance. ADC-based approaches targeting FRα and NaPi2b, mirvetuximab soravtansine approval, and immunotherapy combination trials define the 2025 pipeline.

~19,000 new US cases/yr ~13,000 deaths/yr ~25% BRCA1/2 germline ~30% HRD+ (non-BRCA)
1

Emerging Therapies — Ovarian Cancer


FDA Approved 2022

Mirvetuximab Soravtansine — FRα+ Ovarian

SORAYA — FRα-High Platinum-Resistant EOC
  • SORAYA: ORR 32.4%, mDOR 6.9 mo in FRα-high platinum-resistant — FDA approved 2022
  • MIRASOL: OS HR 0.67 vs investigator's choice — confirmatory Phase 3 2023
  • FRα (folate receptor alpha) high expression: ~35% platinum-resistant EOC
  • First ADC approval in ovarian cancer — validated FRα as a clinical target
  • FRα testing via VENTANA FOLR1 IHC assay — PS2+ in ≥75% tumor cells = high
Standard of Care

PARP Inhibitor Frontline Maintenance

Olaparib (SOLO-1), Niraparib (PRIMA), Bevacizumab+Olaparib (PAOLA-1)
  • SOLO-1: olaparib maintenance BRCA1/2-mutant — 7-yr OS benefit confirmed (HR 0.55)
  • PRIMA: niraparib HRD+ — PFS HR 0.43; HRD– modest benefit
  • PAOLA-1: bev+olaparib HRD+ — PFS HR 0.33; BRCA-mutant largest benefit
  • PARP inhibitor selection: BRCA1/2 (any PARP), HRD+ non-BRCA (niraparib/rucaparib), HRR-proficient (limited benefit)
  • MYRIAD myChoice CDx / Foundation Medicine HRD testing: required for patient selection
Phase 3 Active

Upifitamab Rilsodotin (UpRi) — NaPi2b ADC

UPLIFT — NaPi2b-Positive Platinum-Resistant EOC
  • UPLIFT Phase 3: UpRi vs paclitaxel in NaPi2b-positive PROC — primary OS readout 2025
  • Phase 2: ORR 43% in NaPi2b-high — highest ORR ever reported in PROC at time of publication
  • NaPi2b (SLC34A2): sodium-dependent phosphate transporter — ~75% high-grade serous EOC
  • Vedotin payload — auristatin microtubule disruption
  • Companion diagnostic: 22C3 IHC NaPi2b — cutoff validation ongoing
Phase 2/3 Active

Bevacizumab + PARP + IO Triplet

DUO-O, FIRST, AtTEnd — 1L Frontline Combinations
  • DUO-O: durvalumab+olaparib+bev → durvalumab+olaparib maintenance — HRD+ benefit
  • FIRST: pembrolizumab + chemo → pembro+niraparib maintenance — Phase 3
  • AtTEnd: atezolizumab + bev + chemo → atezo+bev maintenance — Phase 3
  • IO benefit in ovarian: greatest in HRD+ and BRCA-mutant — TMB plays less role
  • Neoadjuvant IO to improve cytoreduction: NEOPENELOPE — pembro neoadjuvant Phase 2
Phase 1/2 — Emerging

VEGFR3 Inhibitors & WEE1/ATR Inhibitors

Adavosertib, Ceralasertib, Berzosertib — DDR Targeted
  • WEE1 inhibitors (adavosertib): Phase 2 + olaparib in BRCA-WT PROC — ORR ~37%
  • ATR inhibitor (ceralasertib + olaparib): Phase 2 PARP-resistant BRCA-mutant EOC
  • DNA damage response (DDR) inhibitor combinations exploit replication stress in EOC
  • CCNE1 amplification (~20% EOC): PARP resistance mechanism — WEE1/CDK2 inhibition target
  • Niraparib + TSR-042 (anti-PD-1): ZEAL Phase 3 ongoing
Phase 2 — Rare Subtypes

Non-HGSOC: Clear Cell, Endometrioid, Mucinous

PIK3CA/ARID1A/HER2 Targeting, Pembrolizumab
  • Clear cell ovarian: ARID1A loss, PIK3CA mutations — PI3K inhibitors (alpelisib) Phase 2
  • HER2+ ovarian carcinoma (~8% clear cell): trastuzumab+pertuzumab Phase 2 signals
  • Endometrioid EOC: MSI-H subset (~10–15%) — pembrolizumab activity
  • Mucinous ovarian: KRAS mutations (~60%) — MEK inhibitor combinations under study
  • SMARCA4-mutant (ultra-rare aggressive): EZH2 inhibitor (tazemetostat) emerging data

Ovarian Cancer — 2025 Bottom Line

PARP inhibitor frontline maintenance has achieved its maximum impact in BRCA-mutant and HRD+ ovarian cancer — SOLO-1's 7-year OS data confirming HR 0.55 in BRCA-mutant patients represents the most durable improvement in high-grade serous EOC ever documented. The critical question now is managing PARP resistance and treating the ~50% HRD-negative population that derives limited PARP inhibitor benefit. Mirvetuximab soravtansine (MIRASOL OS benefit confirmed) is the first ovarian cancer ADC approval and validates FRα as a target — now being explored in earlier lines and non-HGSOC histologies.

UPLIFT's NaPi2b-ADC Phase 3 readout is the most anticipated ovarian trial of 2025. If ORR signals from Phase 2 (43%) translate to OS benefit over paclitaxel, UpRi will become the preferred second ADC option in platinum-resistant ovarian cancer — and will sharpen the question of FRα vs NaPi2b testing and sequencing.

mitotics.com · Ovarian Cancer Emerging Therapies · Q2 2025

Hepatocellular Carcinoma (HCC)

Atezolizumab+bevacizumab and durvalumab+tremelimumab have reshaped 1L HCC. Lenvatinib+pembrolizumab, HAIC combinations, and novel immunotherapy combinations define the active pipeline.

~41,000 new US cases/yr ~29,000 deaths/yr ~80% HCC in cirrhotic liver >50% advanced stage at diagnosis
1

Emerging Therapies — Hepatocellular Carcinoma


1L Standard of Care

Atezolizumab + Bevacizumab (IMbrave150)

IMbrave150 — 1L Advanced HCC
  • IMbrave150: OS HR 0.66 vs sorafenib — first IO combination to beat sorafenib in HCC
  • mOS 19.2 vs 13.4 mo; ORR 30% — FDA approved 2020, maintained 1L standard
  • Variceal bleeding risk: GI endoscopy required pre-treatment (bevacizumab anti-VEGF)
  • Child-Pugh A6 primarily; CPA5 acceptable; CPB excluded (atezo+bev safety not established)
  • TACE+atezo+bev (EMERALD-1): unresectable HCC + TACE — Phase 3 active
FDA Approved 2022

Tremelimumab + Durvalumab (HIMALAYA)

HIMALAYA — STRIDE Regimen, 1L Advanced HCC
  • HIMALAYA: OS HR 0.78 vs sorafenib with STRIDE regimen — FDA approved 2022
  • STRIDE: single priming dose tremelimumab (CTLA-4) + durvalumab maintenance (PD-L1)
  • Clinically relevant in patients with varices or contraindications to bevacizumab
  • OS benefit confirmed at 4-year follow-up: 25.2% vs 14.7% 4-yr OS rate
  • Durvalumab monotherapy: non-inferior to sorafenib with better tolerability — relevant option
Phase 3 Data 2025

Lenvatinib + Pembrolizumab (LEAP-002)

LEAP-002 — 1L Advanced HCC
  • LEAP-002: statistically non-significant OS trend (HR 0.83) despite encouraging mOS 21.2 mo
  • Lenvatinib+pembro not FDA-approved in HCC — mechanistic rationale remains strong
  • RENOBATE: regorafenib+nivolumab Phase 2 — ORR 31% in 2L+ HCC
  • Cabozantinib (CELESTIAL Phase 3) approved 2L; cabozantinib+atezo (COSMIC-312) — Phase 3 failed OS
  • Nivolumab+ipilimumab (CheckMate 040): ORR 32% in sorafenib-pretreated — accelerated approval
Phase 1/2 Pipeline

GPC3-Targeted CAR-T & ADC

GPC3-CAR-T, Cotunlimab, Codrituzumab
  • GPC3 (glypican-3): ~75% HCC overexpression — liver-specific surface antigen
  • GPC3-directed CAR-T: multiple Phase 1 trials (China/US); ORR ~25–35% early signals
  • Codrituzumab (anti-GPC3 ADC): Phase 2 HCC — modest ORR but target validated
  • Cotunlimab (GPC3×CD3 bispecific): Phase 1 HCC
  • Dual-target CAR-T (GPC3+AFP, GPC3+CD133): addressing tumor heterogeneity in HCC
Phase 2/3 Active

HAIC + Systemic Therapy Combinations

HAIC-FOLFOX + Atezo+Bev, SIR-Spheres + IO
  • HAIC (hepatic arterial infusion chemotherapy) + atezo+bev: Phase 3 (HAIC-SO) enrolling
  • HAIC-FOLFOX: randomized Phase 3 vs sorafenib in advanced HCC — superior OS in Chinese trial
  • TACE + durvalumab+bevacizumab (EMERALD-1): Phase 3 HCC — awaiting results
  • Y-90 SIRT (selective internal radiation) + systemic: Phase 2 combinations with IO
  • Thermal ablation + anti-PD-1: abscopal effect mechanism — Phase 2 combination studies
Phase 1/2 — Adjuvant

Adjuvant IO — Post-Resection & Ablation HCC

Nivolumab (CheckMate 9DX), Pembrolizumab (KEYNOTE-937)
  • CheckMate 9DX: adjuvant nivolumab post-resection/ablation — RFS HR 0.72 (Phase 3)
  • FDA approved nivolumab adjuvant HCC post-resection/ablation April 2024
  • KEYNOTE-937: adjuvant pembrolizumab — Phase 3 RFS data expected 2025
  • Recurrence rates post-curative resection: ~70% at 5 years — huge adjuvant unmet need
  • Sorafenib adjuvant (STORM): failed — IO likely superior given RFS data

HCC — 2025 Bottom Line

HCC has undergone a fundamental shift from a disease with essentially one active systemic agent (sorafenib, 2008–2018) to a disease with three competing 1L immunotherapy-based combinations. The adjuvant nivolumab approval post-resection/ablation (CheckMate 9DX, FDA April 2024) represents a new treatment paradigm for curative-intent HCC management — for a cancer where ~70% of patients recur within 5 years post-resection, adjuvant IO could be practice-changing if the DFS benefit is confirmed to translate to OS.

GPC3-directed cellular therapy and ADC development is the most distinctive HCC-specific pipeline — the high and specific GPC3 expression in ~75% of HCC tumors provides both selectivity and a biomarker-selectable patient population. CAR-T in HCC faces the core solid tumor obstacles (trafficking, TME immunosuppression, antigen heterogeneity) but the GPC3 target remains one of the cleanest solid tumor CAR-T opportunities in the field.

mitotics.com · Hepatocellular Carcinoma Emerging Therapies · Q2 2025

Gastric / Gastroesophageal Junction (GEJ) Cancer

Claudin18.2 and HER2 targeting are the defining precision advances in gastric cancer. CLDN18.2 is one of the most cancer-specific targets ever identified — high in gastric, markedly low in normal GI tissues outside the stomach.

~27,000 new US cases/yr ~11,000 deaths/yr ~12–15% HER2+ gastric ~35–40% CLDN18.2 2+ positive
1

Emerging Therapies — Gastric / GEJ Cancer


First CLDN18.2 Approval

Zolbetuximab + Chemo — CLDN18.2+ Gastric

SPOTLIGHT / GLOW — 1L CLDN18.2+/HER2– mGAC/GEJ
  • SPOTLIGHT: OS HR 0.75 (14.4 vs 12.2 mo) + mFOLFOX6 in CLDN18.2+ — FDA approved 2024
  • GLOW: OS HR 0.77 (8.69 vs 7.49 mo) + CAPOX — confirmed across chemo backbone
  • First CLDN18.2-targeted therapy FDA-approved — new biomarker testing paradigm in gastric
  • CLDN18.2 IHC testing (≥2+ in ≥75% tumor cells): now required prior to 1L mGAC treatment
  • CLDN18.2 ADC (CMG901, RC48-CLDN18.2): Phase 1/2 superior efficacy signals expected
Standard of Care

Trastuzumab + Pembrolizumab + Chemo — HER2+ Gastric

KEYNOTE-811 — 1L HER2+ mGAC/GEJ
  • KEYNOTE-811: OS HR 0.70 (20.0 vs 16.8 mo) — FDA approved 2023 for 1L HER2+ mGAC
  • PD-L1 CPS ≥1 appears to enrich for pembrolizumab benefit — test at diagnosis
  • T-DXd (DESTINY-Gastric02): ORR 41% in HER2+ 2L+ pretreated — established HER2+ 2L
  • Trastuzumab deruxtecan FDA approved for HER2+ gastric 2L (DESTINY-Gastric01/02)
  • Zanidatamab (HER2-biparatopic bispecific) + chemo Phase 3 (HERIZON-GEA-01) — 1L HER2+
Phase 2/3 Active

CLDN18.2 ADC — CMG901, RC48

CMG901, SYSA1801 — CLDN18.2-Directed ADC
  • CMG901 (CLDN18.2×vcMMAE ADC): Phase 2 ORR ~40% in CLDN18.2+ GC — highly active
  • SYSA1801: CLDN18.2-directed ADC Phase 1/2 — comparable activity signals
  • ADC > monoclonal antibody in CLDN18.2+ gastric: bystander killing + direct cytotoxicity
  • CLDN18.2×CD3 bispecific (AMG 910, BMF-219): Phase 1 GI tumor cohorts
  • CLDN18.2 CAR-T (CT041): Phase 1 — CR in small series in CLDN18.2-high GC
Phase 3 Active

Nivolumab + Ipilimumab — ESCC & GEJ 1L

CheckMate 648 — Esophageal Squamous Cell Carcinoma
  • CheckMate 648: nivo+ipi OS HR 0.78 vs chemo in ESCC — FDA approved 2021
  • Nivo+chemo also approved (CheckMate 648 chemo arm): OS HR 0.74 vs chemo alone
  • PD-L1 TPS ≥1 strongly enriches benefit: mOS 15.4 vs 9.1 mo in TPS ≥1
  • ESCORT-1st: camrelizumab + chemo — validated IO+chemo in Asian ESCC population
  • Adjuvant nivo (CheckMate 577): post-CRT resected ESCC — DFS HR 0.69, FDA approved 2021
Phase 2 Active

FGFR2b — Bemarituzumab

FIGHT — FGFR2b-Overexpressing 1L mGAC
  • FIGHT: bemarituzumab + mFOLFOX6 — PFS HR 0.68 in FGFR2b+ (Phase 2)
  • FGFR2b overexpression: ~30% of GC — IHC-selected population
  • Phase 3 randomized FIGHT-302: bemarituzumab + mFOLFOX6 vs chemo — ongoing
  • FGFR2b testing: VENTANA FGFR2 IHC companion diagnostic under development
  • FGFR2 amplification (subset): ~5% GC — erdafitinib/futibatinib alternatives
Phase 1/2 Pipeline

EBV+ Gastric Cancer — Immunotherapy Niche

Pembrolizumab, Nivolumab — EBV-Associated GC
  • EBV+ gastric cancer (~9% of all GC): highest PD-L1 CPS, highest TMB, MSI-H enriched
  • KEYNOTE-158: pembro ORR ~57% in EBV+ GC — among highest solid tumor IO ORR
  • EBV+ GC represents ideal IO monotherapy candidate — universal IHC EBER testing warranted
  • POLE/POLD1 mutations (~3% GC): ultra-high TMB, exceptional IO responses
  • MSI-H gastric (~10%): pembrolizumab 1L — CheckMate 649 and KEYNOTE-859 OS confirmed

Gastric / GEJ — 2025 Bottom Line

Zolbetuximab's FDA approval in 2024 marks the most significant paradigm shift in gastric cancer biomarker testing since HER2 in 2010. CLDN18.2 testing is now mandated at initial mGAC diagnosis — creating a new molecularly stratified treatment decision that was not possible before SPOTLIGHT/GLOW. The rapid pipeline evolution to CLDN18.2-directed ADCs (CMG901, SYSA1801) and bispecifics (AMG 910) suggests CLDN18.2 antibody monotherapy may be superseded by ADC-based approaches within 2–3 years, pending Phase 3 data.

HER2+ gastric has the most active pipeline of any gastric subtype — KEYNOTE-811 (pembro+trastuzumab+chemo), T-DXd (2L), and zanidatamab (HER2-biparatopic bispecific) collectively provide potentially curative-intent options in a previously 14-month median OS disease. EBV-positive gastric cancer remains the most under-recognized IO-exceptional subtype in GI oncology — ORR >50% with pembrolizumab monotherapy warrants universal EBER IHC testing at diagnosis.

mitotics.com · Gastric/GEJ Cancer Emerging Therapies · Q2 2025

Thyroid Cancer

RET, BRAF, NTRK, and RAS mutations define a highly actionable thyroid cancer molecular landscape. Selpercatinib and pralsetinib have transformed RET-mutant/fusion thyroid cancer, while BRAF V600E combinations target ATC.

~44,000 new US cases/yr ~2,200 deaths/yr ~60% BRAF V600E (PTC) ~10–20% RET fusion (PTC)
1

Emerging Therapies — Thyroid Cancer


Standard of Care

Selpercatinib & Pralsetinib — RET Fusion/Mutation

LIBRETTO-001, ARROW — RET-Altered Thyroid & Lung
  • LIBRETTO-001: selpercatinib ORR 79% in RET-mutant MTC; 64% in RET-fusion PTC
  • Pralsetinib: ORR 74% in RET-mutant MTC; 89% in RET-fusion PTC
  • Both FDA approved — selpercatinib also for lung and any solid tumor (pan-tumor)
  • LIBRETTO-531: selpercatinib vs cabozantinib/vandetanib in RET-mutant MTC — PFS HR 0.28
  • RET testing (RNA fusion + mutation): required at all thyroid cancer diagnosis per NCCN
Anaplastic ATC Standard

Dabrafenib + Trametinib — BRAF V600E ATC

ROAR Basket Trial — BRAF V600E Anaplastic Thyroid Cancer
  • ROAR: ORR 56% in BRAF V600E ATC — most active therapy ever reported in ATC
  • ATC: median OS previously 5–6 months — combination achieved 1-yr OS ~60%
  • FDA approved dabrafenib+trametinib for BRAF V600E ATC 2018 — remains standard
  • Pembrolizumab + dab+tram: Phase 2 combinations active in BRAF V600E ATC
  • BRAF V600E testing: required at ATC diagnosis (BRAF mutant in ~40% ATC)
Phase 2/3 Active

Radioactive Iodine (RAI) Redifferentiation

Selumetinib (ASTRA), MAPK pathway RAI resensitization
  • ASTRA: selumetinib (MEK inhibitor) → RAI restores iodine uptake in RAI-refractory DTC
  • Selumetinib FDA approved 2020 for pediatric NF1 — now in adult thyroid redifferentiation
  • Vemurafenib + RAI redifferentiation in BRAF V600E RAI-refractory PTC: Phase 2
  • Cabozantinib: COSMIC-311 — OS benefit in RAI-refractory DTC 2L, FDA approved 2021
  • Lenvatinib+everolimus: Phase 2 combination in lenvatinib-progressed DTC
Phase 1/2 Pipeline

NRAS/HRAS Mutant Thyroid — MEK/RAF Targeting

Binimetinib, Cobimetinib — RAS-Mutant Thyroid
  • RAS mutations (NRAS, HRAS): ~25% PTC, ~40% FTC — historically untargeted
  • Binimetinib + palbociclib: Phase 2 in NRAS-mutant thyroid (ARAF/NRAS combination)
  • Pan-RAS inhibitors (RMC-6236): NRAS/HRAS-mutant thyroid — Phase 1 data expected
  • Cobimetinib (MEK) + atezolizumab: Phase 2 in RAS-mutant thyroid — solid rationale
  • ALK fusions (~0.5–2% PTC): crizotinib/alectinib — tumor-agnostic approval applicable
Phase 1/2 — MTC

RET Resistance — Next-Gen Inhibitors

LOXO-260, BLU-701 — RET Solvent-Front Resistance
  • RET solvent-front mutations (G810X): primary selpercatinib/pralsetinib resistance mechanism
  • LOXO-260: designed against G810 mutations — Phase 1 data in RET inhibitor-resistant MTC/NSCLC
  • BLU-701: next-gen selective RET inhibitor with G810 coverage — Phase 1 active
  • Vandetanib + cabozantinib rechallenge: limited efficacy post-selpercatinib — sequential not recommended
  • Tepoxalin (novel RET/VEGFR inhibitor): preclinical activity in solvent-front resistant RET
Emerging — Pembrolizumab + TKI

Immunotherapy + Lenvatinib in DTC

KEYNOTE-B77, Study 205 — Lenvatinib + Pembro in DTC
  • Study 205 (lenvatinib + pembro): ORR 36% in RAI-refractory DTC; mPFS 22 mo — Phase 2
  • LEAP-007: lenvatinib+pembro failed in NSCLC but thyroid data distinct (different TME)
  • KEYNOTE-B77: randomized Phase 2/3 pembro+lenvatinib vs lenvatinib — enrolling
  • Thyroid TME: relatively immunosuppressive but IO response rates better than expected
  • MSI-H thyroid (<1%): pembrolizumab monotherapy — tumor-agnostic applicable

Thyroid Cancer — 2025 Bottom Line

Thyroid cancer has transformed from a disease managed almost entirely with RAI and surgery into one of the most molecularly actionable cancers in oncology. RET inhibitor therapy (selpercatinib, pralsetinib) has achieved ORRs of 60–89% in RET-altered thyroid cancer — response rates that rival the best TKI approvals in any cancer type. LIBRETTO-531's PFS HR of 0.28 versus the prior cabozantinib/vandetanib standard has made selpercatinib the unambiguous preferred treatment for RET-mutant MTC.

The outstanding challenge is anaplastic thyroid cancer in the ~60% without BRAF V600E — a population with essentially no effective systemic options and median OS under 6 months. Immune combinations, RAS-directed approaches, and anti-CTLA4 strategies are all under investigation, but ATC in the BRAF-WT population remains one of the most urgent unmet needs in all of oncology.

mitotics.com · Thyroid Cancer Emerging Therapies · Q2 2025

Pediatric Oncology

Pediatric cancers have distinct molecular landscapes from adult. NTRK fusions, ALK alterations, BRAF V600E in pHGG, RET fusions, and H3 K27M mutations are highly actionable. FDA pediatric oncology approvals have increased 10-fold since 2017 RACE Act.

~15,000 new US cases/yr (0–19) ~1,600 deaths/yr ~70%+ overall 5-yr survival (all peds) ~10–15% NTRK fusion rate (peds overall)
1

Emerging Therapies — Pediatric Oncology


FDA Approved 2022

Dabrafenib + Trametinib — BRAF V600E pHGG

FIREFLY-1 — BRAF V600E Pediatric High-Grade Glioma
  • FIREFLY-1: ORR 56%, mDOR 22.2 mo in BRAF V600E pHGG — FDA approved 2022
  • BRAF V600E: ~10–20% pediatric HGG, ~50–70% pediatric LGG (pilocytic astrocytoma)
  • Dab+tram also approved pediatric LGG (FIREFLY-2) — first-ever targeted approval in pLGG
  • BRAF+MEK combination superior to BRAF monotherapy: resistance prevention critical
  • CNS penetration adequate — vemurafenib (prior BRAF monotherapy) less penetrant
Standard of Care

Larotrectinib / Entrectinib — NTRK Fusion Pediatric

LOXO-TRK, STARTRK — Tumor-Agnostic NTRK Pediatric
  • Larotrectinib: ORR 75% in NTRK fusion+ pediatric tumors (infantile fibrosarcoma, NTRK+ CNS)
  • NTRK fusions: ~15% pediatric HGG (ETV6-NTRK3 infantile fibrosarcoma), ~10% secretory breast, rare in many pediatric histologies
  • Entrectinib: CNS-penetrant — preferred in NTRK+ CNS pediatric tumors
  • Both FDA-approved tumor-agnostically — NTRK testing universal recommendation in pediatric solid tumors
  • Resistance via NTRK solvent-front mutations (G595R, G623R): repotrectinib Phase 1/2
DIPG — Historic Unmet Need

H3 K27M-Mutant DIPG/DMG — ONC201

ONC201 (Dordaviprone) — H3 K27M DMG
  • ONC201: first drug to show OS benefit in H3 K27M-mutant DIPG/DMG in Phase 2
  • FDA granted accelerated approval 2024 — first DIPG drug approval ever
  • H3 K27M: ~80% DIPG, ~50% spinal DMG — nearly universal in DIPG
  • Mechanism: ClpP mitochondrial protease agonism → tumor cell death
  • ONC206, ONC212: next-gen dopamine receptor antagonist/mitochondrial agonists in development
Phase 2/3 Active

Anti-GD2 Therapy — Neuroblastoma

Dinutuximab (ANBL12P1), Anti-GD2 CAR-T, Naxitamab
  • Dinutuximab + immunotherapy (ANBL12P1): maintenance standard for high-risk NB — OS improved
  • Naxitamab (anti-GD2) + GM-CSF: ORR 34% in refractory/relapsed NB — FDA approved 2020
  • GD2-CAR-T: Phase 1 neuroblastoma — multiple constructs in pediatric trials
  • DFMO (difluoromethylornithine) maintenance: Phase 3 — polyamine pathway targeting in NB
  • I-131 MIBG theranostics: iobenguane I-131 — FDA approved refractory NB 2018
Phase 1/2 Pipeline

ALK Inhibitors — ALK-Positive ALCL & IMT

Crizotinib, Alectinib, Lorlatinib — ALK-Fusion Pediatric Tumors
  • ALK+ ALCL (anaplastic large cell lymphoma): ~85% ALK-fusion — crizotinib ORR ~90%
  • ALK+ inflammatory myofibroblastic tumor (IMT): ~50% ALK-fusion — crizotinib first-line
  • Lorlatinib: CNS-penetrant — preferred in ALK+ brain metastasis/CNS ALK-fusion tumors
  • ALK mutation testing at diagnosis (EML4-ALK, NPM-ALK) — tumor-agnostic lorlatinib applicable
  • Neuroblastoma ALK mutation (~10% NB): Phase 1/2 lorlatinib + chemotherapy combinations
Phase 1/2 Pipeline

EWS-FLI1 Inhibitors — Ewing Sarcoma

TK216, Lurbinectedin — EWS::FLI1 Targeting
  • EWS::FLI1 fusion: ~85% Ewing sarcoma — defining oncogenic driver, previously untargeted
  • TK216: first-in-class EWS-FLI1 inhibitor Phase 1/2 — pharmacodynamic target engagement confirmed
  • Lurbinectedin: RNA Pol II inhibitor — EWS-FLI1 transcriptional program disruption Phase 1/2
  • PRISM trial: high-throughput molecular profiling of refractory pediatric tumors — niche actionability
  • SMARCB1-deficient tumors (rhabdoid): EZH2 inhibitor (tazemetostat) Phase 1 pediatric

Pediatric Oncology — 2025 Bottom Line

The pediatric oncology molecular revolution was catalyzed by the RACE for Children Act (2017) — which mandated that adult oncology clinical trials include pediatric cohorts when biologically relevant. The result has been a 10-fold increase in pediatric oncology FDA approvals between 2017 and 2025, with NTRK inhibitors (larotrectinib, entrectinib), ALK inhibitors, and BRAF/MEK combinations leading the approvals.

The most profound advance is ONC201 (dordaviprone) in H3 K27M-mutant DIPG — a tumor that has killed children within 9 months of diagnosis for decades with no proven systemic therapy. FDA accelerated approval in 2024 marks the first regulatory approval specifically for DIPG in history. The H3 K27M mutation's near-universal presence in DIPG (~80%) makes this an unusually biomarker-homogeneous target population. ONC206 and next-generation compounds building on this mechanism are the immediate clinical priority.

mitotics.com · Pediatric Oncology Emerging Therapies · Q2 2025