Top Advances in Efficacy Demonstrated in Oncology Clinical Trials in 2017

2017 was a particularly busy and productive year in oncology, particularly around clinical efficacy. Some of the most exciting efficacy data achieved in decades have been achieved by CAR T-cells this year.  Although it’s too early to confidently be using the word “cure” for large groups of patients, complete remission rates appear excellent for some hard-to-treat hematology-oncology patient cohorts. Currently marketed CAR T-cell therapies—Kymriah and Yescarta—are autologous, CD19-targeting CAR T-cells which the FDA approved for patients who have exhausted other treatment options. However, both therapies are associated with severe, life-threatening, and potentially fatal side effects. Encouragingly, future development of CAR T-cells are evaluating better tolerated, more controlled, and logistically simpler (allogeneic) versions of this exciting class. Newer CAR T-cell therapies, for example Juno Therapeutics’ lisocabtagene maraleucel (a CD-19 targeted product) and Bluebird Bio/Celgene’s BCMA targeted strategy have demonstrated impressive efficacy in other clinical trials. We are continuing to watch the class with great interest in 2018.

We also saw some exciting clinical data out of ASCO this year, particularly in breast cancer:

  • The highly anticipated results of the APHINITY trial, which showed that when Perjeta is added to Herceptin and chemotherapy it demonstrates an improved invasive disease-free survival compared with Herceptin and chemotherapy alone.
  • Data from the MONARCH-2 trial which showed that at an interim analysis, Verzenio improved the median progression-free survival of locally-advanced or metastatic HR-positive/HER2-negative patients compared with fulvestrant alone.
  • Phase IIII data from the OlympiAD trial which showed that, at an interim analysis, previously-treated metastatic breast cancer patients with germline BRCA1/2 mutations receiving Lynparza achieved a progression-free survival of 7 months compared with 4.2 months for those receiving chemotherapy. Lynparza also reduced the chance of disease progression by 42%, thus becoming the first targeted agent to show a significant clinical benefit in the metastatic germline BRCA1/2-mutated population.


Promising clinical data was also revealed for non-small cell lung cancer (NSCLC) in 2017:

  • AstraZeneca’s Tagrisso demonstrated progression-free survival benefits over standard of care therapy in first-line EGFR-positive disease in the Phase III FLAURA These results support the label expansion of Tagrisso into the first-line EGFR-positive setting and will significantly expand the eligible patient population.
  • In May 2017, Keytruda received the second first-line label expansion in combination with chemotherapy. After approving Keytruda for first-line, PD-L1-positive NSCLC, the FDA granted it a label expansion in the first-line setting in combination with pemetrexed and carboplatin. The combination is indicated for nonsquamous patients irrespective of PD-L1 status and it received accelerated approval based on statistically significant progression-free survival and overall response rate benefits compared with chemotherapy alone in the Phase II KEYNOTE-021 This approval significantly expands the number of treatment opportunities for Keytruda by allowing its use in patients with low PD-L1 or PD-L1-negative tumors, a significantly larger patient population. Notably, Keytruda is the first immune checkpoint inhibitor to receive approval in combination with chemotherapy for any oncology indication.
  • ASCO 2017 highlighted the impressive progression-free survival improvements for Alecensa compared with Xalkori in the pivotal Phase III ALEX trial in first-line ALK-translocation-positive patients. Alecensa received a label expansion into the first-line setting in November and we expect it to rapidly displace Xalkori for ALK-translocation-positive patients. Novartis received first-line approval Zykadia six months before Roche, but the use of chemotherapy as the comparator in the pivotal trial is likely to limit Zykadia’s uptake.


2017 was a particularly busy and productive year. Not only here at Decision Resources Group (the oncology team launched two new data and insight solutions) but also within the oncology pharmaceutical and regulatory sectors.

The pace of clinical development, the number of revolutionary new treatments approved, and the sheer volume of clinical data has been overwhelming. While significant progress in innovation was demonstrated in 2017, we also witnessed approvals of several ‘me-too’ and same-in-class drugs in oncology.

Want to learn more? Read our list of the top 5 trends that we think made 2017 a year to remember for oncology.



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Contributors: Andrew Merron: Ph.D., Executive Director, Oncology and Biosimilars, Biopharma Insights
Published on: 05 January, 2018