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Oncology Highlights - December 2019

Stay informed on the latest news within oncology drug development. Subscribe to our newsletter to keep up to date with last month's science, industry and regulatory oncology highlights.

Enjoy reading! - The SMS-oncology team



News and publications

CAR-T cell therapy for relapsed/refractory mantle cell lymphoma: Primary results from the ZUMA-2 global, multicenter, single-arm Phase II trial assessed the safety and efficacy of the investigational CD19 chimeric antigen receptor (CAR) T cell therapy KTE-X19 (development supported by Kite, a Gilead company). Primary results reported a 93% objective response rate (ORR) and a 67% complete response rate after a single KTE-X19 infusion. These results, together with its acceptable safety profile, make KTE-19 a promising new therapy for patients with few other treatment options. Results were presented at the 61st Annual American Society of Hematology meeting in December 2019.

Pembrolizumab for pediatric patients with advanced cancers: While pembrolizumab is successfully used for treatment of adult patients with advanced cancers, pediatric patients still need effective treatment options. Currently, a Phase I/II open-label trial with pembrolizumab is ongoing in pediatric patients with advanced melanoma or programmed cell death ligand 1 (PD-L1) positive, advanced/relapsed/refractory solid tumors or lymphomas (KEYNOTE-051). The interim analysis revealed a 60% ORR in patients with relapsed/refractory non-Hodgkin lymphoma. These results, together with the acceptable safety profile, make pembrolizumab a promising therapy also for pediatric patients with selected tumors. Interim results were published in The Lancet Oncology, with final results expected in September 2022.


  • Astellas acquires Xyphos Biosciences, for $120 million. Xyphos’s focus on CAR NK and T cells to generate immunotherapies for oncology, with the first product candidate to be tested in a first-in-human trial in 2021, will diversify Astellas’ immuno-oncology drug portfolio.
  • Merck acquires ArQule, through an acquisition subsidiary, for $20 per share in cash for an approximate total equity value of $2.7 billion. ArQule’s focus on tyrosine kinase inhibitor (TKI) development, specifically its lead investigational candidate the novel Bruton’s TKI ARQ 531, will further diversify Merck’s oncology pipeline, with expansion into targeted therapies for hematological malignancies.
  • Sanofi acquires Synthorx for $2.5 billion ($68 a share in cash), considered approximately three times its market value, to build its portfolio of innovative therapies in the field of oncology. Synthorx’s lead immuno-oncology candidate THOR-707, an interleukin-2 variant currently in clinical development in multiple solid tumors as a single agent and combined with immune checkpoint inhibitors, is considered synergistic with Sanofi’s pipeline, thus enhancing its position in immuno-oncology.


The Committee for Medical Products for Human Use (CHMP) adopted a positive opinion for:

New generic medicines:

  • Azacitidine accord (Accord Healthcare), for treatment of adult patients ineligible for hematopoietic stem cell transplantation with myelodysplastic syndromes, chronic myelomonocytic leukemia or acute myeloid leukemia (AML) with 10-29% or, respectively 20-30% blasts and multi-lineage dysplasia, as well as for AML with >30% marrow blasts.

Extensions of indications:

  • Ramucirumab (Cyramza, Eli Lilly Nederland) in combination with erlotinib, for first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with activating epidermal growth factor receptor mutations.
  • Daratumumab (Darzalex, Janssen-Cilag International) in combination with bortezomib, thalidomide, and dexamethasone for treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplantation. 
  • Apalutamide (Erleada, Janssen-Cilag International) in combination with androgen deprivation therapy, for treatment of metastatic hormone-sensitive prostate cancer in adult men.


The FDA granted approval for:

  • Olaparib (Lynparza, AstraZeneca Pharmaceuticals) for maintenance treatment of adult patients with deleterious/suspected germline BRCA-mutated metastatic pancreatic adenocarcinoma, as detected by the BRACAnalysis CDx test. Approval was based on the POLO Phase III trial, whose results reported a median PFS of 7.4 months vs 3.8 months for patients who received olaparib versus placebo. The companion diagnostic test (Myriad Genetic Laboratories) was also approved for selection of patients with pancreatic cancer for treatment with olaparib.
  • Fam-trastuzumab deruxtecan-nxki (Enhertu, Daiichi Sankyo) for patients with unresectable or metastatic HER2-positive breast cancer who received ≥2 prior anti-HER2-based regimens in the metastatic setting. Approval was based on the multicenter DESTINY-Breast01 trial, whose results reported an ORR of 60.3% and a median response duration of 14.8 months in these patients.
  • Enfortumab vedotin-ejfv (Padcev, Seattle Genetics) for patients with locally advanced or metastatic urothelial cancer. Enfortumab vedotin-ejfv is the first FDA-approved treatment for these patients and the first-in-class antibody drug conjugate (ADC) directed against nectin-4, a protein highly expressed in urothelial cancer. The ADC is approved based on tumor response rate, with continued approval being contingent on verification and description of clinical benefit in confirmatory trials.
  • Enzalutamide (Xtandi, Pfizer and Astellas Pharma) for patients with metastatic castration-sensitive prostate cancer (mCSPC). The approval was based on the ARCHES Phase III trial, whose results reported that median radiographic PFS was not reached in patients with mCSPC receiving enzalutamide vs 19.4 months in patients receiving placebo.
  • Atezolizumab (Tecentriq, Roche) plus chemotherapy (nab-paclitaxel and carboplatin) for initial treatment of metastatic non-squamous non-small cell lung cancer. The approval was based on the Phase III IMpower130 trial showing a significantly longer median OS (18.6 vs 13.9 months, p=0.0384) in patients receiving the combination therapy vs chemotherapy alone.

The FDA also granted breakthrough therapy/device designation to several products to progress their development. These include:

  • Tucatinib (tucatinib, Seattle Genetics), combined with trastuzumab and capecitabine, for treatment of patients with locally advanced unresectable/metastatic HER2-positive breast cancer, including patients with brain metastases, who have been treated with trastuzumab, pertuzumab, and trastuzumab-emtansine.
  • Tibsovo (ivosidenib, Agios Pharmaceuticals), for treatment of adult patients with relapsed/refractory myelodysplastic syndrome with a susceptible isocitrate dehydrogenase 1 mutation.
  • JNJ-4528 (CAR-T cell therapy, Janssen), for treatment of relapsed/refractory multiple myeloma. The designation is supported by initial results of the Phase Ib/II CARTITUDE-1 open-label trial (NCT03548207), which reported a 91% response rate at data cut-off.
  • Orencia (abatacept, Bristol-Myers Squibb), for prevention of moderate to severe acute graft versus host disease (GVHD) in hematopoietic stem cell transplants from unrelated donors. Approved therapies for GVHD, a potentially life-threatening complication after stem cell transplant for treatment of hematological cancers, are currently lacking.