January 2014 Edition Vol.8, Issue 1

Phase 3 Hits & Misses of 2013

Phase 3 Hits & Misses of 2013 

By Nancy Ciancaglini

Here they are—the phase 3 trials and therapies that either shone or disappointed investors and analysts in 2013. We’ve compiled a list of hits and misses of clinical trials from the past year below.  


1) Amgen announced that the phase 3 head-to-head ASPECCT (‘763) trial, testing Vectibix® (panitumumab) versus Erbitux® (cetuximab) as a single agent in chemorefractory metastatic colorectal cancer (mCRC) patients with wild-type KRAS tumors, met its primary endpoint of non-inferiority for overall survival (OS) (May 7, 2013).   

2) Sanofi’s pivotal phase 3 JAKARTA study of selective JAK2 inhibitor fedratinib (SAR302503) in myelofibrosis met its primary endpoint in both dose groups. The primary endpoint assessed the proportion of patients with a reduction in spleen volume ≥35% after 24 weeks of treatment (May 17, 2013).

3) GlaxoSmithKline and Genmab announced that a phase 3 study of Arzerra®  (ofatumumab) in combination with chlorambucil in patients with previously untreated chronic lymphocytic leukemia (CLL) had met its primary endpoint of PFS (May 29, 2013).

4) Pfizer’s Xalkori® (crizotinib) significantly delayed the worsening of lung cancer in ALK-positive patients compared with traditional chemotherapy in a phase 3 study, but didn’t prolong OS in the same trial (Jun. 1, 2013).

5) In a phase 3 study, Bayer and Onyx Pharmaceuticals said that Nexavar® (sorafenib) helped to nearly double the number of months that patients with differentiated thyroid cancer lived without their disease worsening—but the drug made no difference in OS (Jun. 2, 2013).

6) In a phase 3 trial, adding Roche’s Avastin to standard chemotherapy extended survival for women with metastatic or relapsed cervical cancer. This is the first time a biologic significantly prolonged life in this setting (Jun. 2, 2013).

7) Novartis’ Afinitor® (everolimus) tablets significantly extended time without disease progression in heavily pretreated women with HER2-positive advanced breast cancer when combined with Herceptin® (trastuzumab) and vinorelbine in the pivotal phase 3 BOLERO-3 trial (Jun. 2, 2013).

8) Eli Lilly & Co. announced that the phase 3 SQUIRE trial of necitumumab (IMC-11F8), in combination with gemcitabine (Gemzar) and cisplatin, met its primary endpoint of increasing OS in patients with advanced squamous non-small cell lung cancer as a first-line treatment (Aug. 13, 2013).

9) Eli Lilly reported that the phase 3 RAINBOW study of ramucirumab in combination with paclitaxel met its primary endpoint of improved OS in patients with advanced gastric cancer; a secondary endpoint of improved PFS was also met (Sept. 26, 2013).

10) In the phase 3 TH3RESA study, Roche’s Kadcyla® (trastuzumab emtansine) significantly extended the time people with advanced HER2-positive breast cancer lived without their disease worsening (PFS, a co-primary endpoint) compared with people who received a treatment of their physician’s choice (Sept. 28, 2013).

11) Gilead Sciences stopped a pivotal phase 3 study (Study 116) early of idelalisib in previously-treated chronic lymphocytic leukemia (CLL) patients after interim results demonstrated significant efficacy for the trial’s primary endpoint of PFS (Oct. 9, 2013).

12) Medivation and partner Astellas stopped the phase 3 PREVAIL trial of Xtandi® (enzalutamide) early when the large trial met both co-primary endpoints of OS and PFS in chemotherapy-naïve patients with advanced prostate cancer (Oct. 22, 2013).

13) Data from Merck & Co.’s pivotal phase 3 efficacy study (Protocol 001) demonstrated that the investigational HPV vaccine V503 appeared to offer broader protection against the cancer-causing virus than the company's Gardasil® vaccine (Nov. 3, 2013).

14) In the phase 3 CLL11 study, people with previously untreated chronic lymphocytic leukemia (CLL) and co-existing medical conditions taking Gazyva® (obinutuzumab) in combination with chlorambucil lived about a year longer without their disease worsening compared with patients taking Roche’s Rituxan® (rituximab) in combination with chlorambucil  (Nov. 7, 2013).

15) In a pivotal phase 3 trial, patients receiving Amgen’s experimental melanoma drug talimogene laherparepvec lived about four months longer than patients receiving an older treatment (Nov. 18, 2013).

16) Oncolytics Biotech said that its drug Reolysin® slowed the progression of head and neck cancers in a late-stage clinical trial (Nov. 21, 2013).

17) Novartis’ investigational compound LBH589 (panobinostat) significantly extended time without disease progression in the phase 3 PANORAMA-1 study of patients with relapsed or refractory multiple myeloma (Dec. 6, 2013).

18) Patients with myelofibrosis taking Novartis’ drug Jakavi® (ruxolitinib) lived longer than those receiving placebo or conventional therapy, based on several analyses from the phase 3 COMFORT-I and COMFORT-II studies (Dec. 9, 2013).

19) At ASH, Celgene presented the initial phase 3 results of the FIRST trial, evaluating lenalidomide plus low–dose dexamethasone (Rd) versus standard thalidomide in newly diagnosed multiple myeloma patients ineligible for stem cell transplantation.  After a median follow-up period of 37 months, the study met its primary endpoint (PFS), demonstrating that patients treated with continuous Rd were 28% less likely to experience disease progression or death than those patients treated with the thalidomide combination (Dec. 8, 2013).      

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