# MyPathway

Source: https://onco.cc/trials/mypathway/  
OnCo record `mypathway` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

MyPathway was Roche's basket trial: six of its approved drugs given to people whose tumours carried the matching target but in a cancer the drug was never approved for. Its clearest win was trastuzumab plus pertuzumab in HER2-amplified bowel cancer, which became a guideline option.

## Summary

MyPathway opened in April 2014 as an open-label, multi-basket phase 2 run by Genentech at community and academic sites in the United States. Patients whose tumours carried a HER2 alteration, an EGFR mutation, a BRAF mutation, a Hedgehog pathway alteration, an ALK rearrangement or, later, high tumour mutational burden or microsatellite instability received the matching approved drug outside its licensed indication, with objective response as the endpoint in each drug-by-tumour cohort. It enrolled 673 patients before completing.

The first report, by John Hainsworth and colleagues in the Journal of Clinical Oncology in 2018, covered 230 treated patients and found responses in 23 percent overall, with the strongest signals for trastuzumab plus pertuzumab in HER2-amplified colorectal cancer and for vemurafenib in BRAF V600-mutant non-melanoma cancers. The updated colorectal cohort, in the Lancet Oncology in 2019, reported a response rate of 32 percent in 57 patients with HER2-amplified, treatment-refractory disease, most durable in RAS wild-type tumours; that result put trastuzumab plus pertuzumab into United States guidelines for HER2-amplified colorectal cancer. The atezolizumab cohort, in Cancer Discovery in 2022, showed that response to single-agent PD-L1 blockade in unselected tumours rose with tumour mutational burden and was concentrated in the most highly mutated tumours.

MyPathway is the industry counterpart to NCI-MATCH and TAPUR, and the model for Roche's later TAPISTRY platform. It showed a company could generate label-adjacent evidence cheaply from a basket, and it produced two of the more reliable tumour-agnostic signals (HER2 amplification for dual HER2 blockade and BRAF V600 for BRAF inhibition) alongside many cohorts with little activity.

## Fields

- Kind: Trial
- Status: completed
- Last checked: 2026-09-17
- Also known as: My Pathway; MyPathway basket
- Registry id: NCT02091141
- Phase: 2
- Setting: Advanced solid tumours with alterations in HER2, EGFR, BRAF, the Hedgehog pathway, ALK or high tumour mutational burden, outside the cancers for which the matching Roche drug is approved: trastuzumab with pertuzumab, erlotinib, vemurafenib with cobimetinib, vismodegib, alectinib or atezolizumab
- Sponsor: Genentech
- Enrolled: 673
- Result: Response in 23 percent of the first 230 patients; trastuzumab plus pertuzumab gave a 32 percent response rate in HER2-amplified colorectal cancer, now a guideline option; atezolizumab activity rose with tumour mutational burden.
- Outcomes: Objective response, trastuzumab plus pertuzumab in HER2-amplified colorectal cancer: Treated patients 32%; Objective response, trastuzumab plus pertuzumab in HER2-amplified or overexpressing metastatic biliary tract cancer (investigator-assessed): Treated patients 23%

## Notes

- Biliary tract cancer cohort (Javle et al., Lancet Oncology 2021): 39 previously treated patients with HER2 amplification, overexpression or both from 23 US sites received pertuzumab 840 mg then 420 mg with trastuzumab 8 then 6 mg/kg every 3 weeks; 9 achieved a partial response (23 percent) at a median follow-up of 8.1 months, the first prospective evidence for dual HER2 antibody therapy in biliary cancer and the basis of the UK DETERMINE trastuzumab-pertuzumab arm open to HER2-amplified gallbladder cancer. The proportion with gallbladder cancer is in the paper's tables, not its abstract.

## Sources

- ClinicalTrials.gov NCT02091141: https://clinicaltrials.gov/study/NCT02091141
- Hainsworth et al., Journal of Clinical Oncology 2018: targeted therapy for advanced solid tumors on the basis of molecular profiles, results from MyPathway: https://doi.org/10.1200/JCO.2017.75.3780
- Meric-Bernstam et al., Lancet Oncology 2019: pertuzumab plus trastuzumab for HER2-amplified metastatic colorectal cancer (MyPathway): https://doi.org/10.1016/S1470-2045(18)30904-5
- Friedman et al., Cancer Discovery 2022: atezolizumab treatment of tumors with high tumor mutational burden from MyPathway: https://doi.org/10.1158/2159-8290.CD-21-0450

## Connected records

- cancers: [Biliary tract cancer (all types)](https://onco.cc/cancers/biliary-tract-cancer/), [Biliary tract cancer (cholangiocarcinoma)](https://onco.cc/cancers/cholangiocarcinoma/), [Colorectal cancer](https://onco.cc/cancers/colorectal/), [Gallbladder cancer](https://onco.cc/cancers/gallbladder/), [HER2-amplified colorectal cancer](https://onco.cc/cancers/her2-amplified-colorectal/)
- terms: [Basket, umbrella, and platform trials](https://onco.cc/terms/basket-umbrella-platform/), [Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups)](https://onco.cc/terms/gallbladder-cancer-in-biliary-trials/), [Master protocol (platform, basket and umbrella trials)](https://onco.cc/terms/master-protocol/), [Rare cancers](https://onco.cc/terms/rare-cancers/), [Tumour mutational burden (TMB)](https://onco.cc/terms/tmb/), [Tumour-agnostic (tissue-agnostic) approval](https://onco.cc/terms/tumour-agnostic/)
- technologies: [Comprehensive genomic profiling](https://onco.cc/technologies/cgp/), [Immune checkpoint inhibitors](https://onco.cc/technologies/checkpoint-inhibitor/), [Monoclonal antibodies](https://onco.cc/technologies/monoclonal-antibody/), [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- drugs: [Alectinib](https://onco.cc/drugs/alectinib/), [Atezolizumab](https://onco.cc/drugs/atezolizumab/), [Cobimetinib](https://onco.cc/drugs/cobimetinib/), [Erlotinib](https://onco.cc/drugs/erlotinib/), [Pertuzumab](https://onco.cc/drugs/pertuzumab/), [Trastuzumab](https://onco.cc/drugs/trastuzumab/), [Vemurafenib](https://onco.cc/drugs/vemurafenib/), [Vismodegib](https://onco.cc/drugs/vismodegib/)
- companies: [Roche / Genentech](https://onco.cc/companies/roche-genentech/)
- trials: [TRIUMPH](https://onco.cc/trials/triumph/), [Tumor-agnostic Precision Immuno-oncology and Somatic Targeting Rational for You (TAPISTRY) Platform Study](https://onco.cc/trials/nct04589845/)
- people: [Funda Meric-Bernstam](https://onco.cc/people/funda-meric-bernstam/)
- key papers: [Atezolizumab Treatment of Tumors with High Tumor Mutational Burden from MyPathway, a Multicenter, Open-Label, Phase IIa Multiple Basket Study](https://onco.cc/key-papers/paper-friedman-cancer-discov/), [Pertuzumab and trastuzumab for HER2-positive, metastatic biliary tract cancer (MyPathway): a multicentre, open-label, phase 2a, multiple basket study](https://onco.cc/key-papers/paper-javle-mypathway-her2-biliary-lancet-oncol-2021/), [Pertuzumab plus trastuzumab for HER2-amplified metastatic colorectal cancer (MyPathway): an updated report from a multicentre, open-label, phase 2a, multiple basket study](https://onco.cc/key-papers/paper-mypathway-lancet-oncol-2019/), [Targeted Therapy for Advanced Solid Tumors on the Basis of Molecular Profiles: Results From MyPathway, an Open-Label, Phase IIa Multiple Basket Study](https://onco.cc/key-papers/paper-hainsworth-j-clin-oncol/)

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