A Roundup of Some of This Week’s Clinical Trial Successes!


This week had a few disappointing clinical trials, as do most weeks, but it also had a number of significant successes. Here’s a look at some of the clinical trials that met their primary endpoints this week.

Gemphire Therapeutics. Based in Livonia, Michigan, Gemphire announced on June 28 that its gemcabene met its primary endpoint in a Phase IIb INDIGO-1 clinical trial in severe hypertriglyceridemia (SHTG)—severe high triglycerides. Patients receiving the drug had triglycerides reduced by 47 percent compared to 27 percent in patients receiving placebo. Statistically significant secondary endpoints included lower LDL-C, non-HDL-C, VLDL-C, apoB, ApoE, ApoCIII and SAA levels, mostly various forms of cholesterol.

“We are pleased to reach this milestone of meeting both primary and multiple secondary endpoints and look forward to advancing gemcabene into Phase III trials,” said Steven Gullans, Gemphire’s chief executive officer, in a statement.

Spectrum Pharmaceuticals. Located in Henderson, Nevada, Spectrum announced on June 29 that its ADVANCE Phase III clinical trial of Rolontis in neutropenia met its primary endpoint. Patients receiving the drug versus receiving pegfilgrastim in Cycle 1 had an absolute risk reduction of severe neutropenia of 8.5 percent. It also announced that the RECOVER study, the second Phase III Rolontis trial, met the primary efficacy endpoint of non-inferiority in duration of severe neutropenia (DSN) between Rolontis and pegfilgrastim.

“We are pleased that both Rolontis Phase III studies, which studied more than 600 patients combined, demonstrated non-inferiority and similar safety profiles to the current standard of care,” said Joe Turgeon, Spectrum’s president and chief executive officer, in a statement. “The emerging Rolontis efficacy and safety profile appears to be strong and we are excited to potentially provide a novel therapy to the patients and physicians managing chemotherapy-induced neutropenia.”

Astellas Pharma and Pfizer. Tokyo-based Astellas Pharma and U.S.-based Pfizer announced results from the pivotal Phase III PROSPER trial, which evaluated enzalutamide and androgen deprivation therapy (ADT) versus placebo plus ADT in patients with non-metastatic castration-resistant prostate cancer (CRPC). The results were published in the New England Journal of Medicine. Enzalutamide plus ADT significantly cut the risk of developing metastases or death compared to ADT alone.

I’m pleased with the PROSPER trial results, which confirm that men with non-metastatic CRPC receiving enzalutamide plus androgen deprivation therapy (ADT) had an almost two-year delay in appearance of prostate cancer metastasis or death as compared to those taking ADT,” said Maha Hussain, Genevieve Teuton Professor of Medicine, Robert H. Lurie Comprehensive Cancer Center of Northwestern University, and lead study investigator, in a statement.

Medivation inked a deal with Astellas in October 2009 to develop and commercialize enzalutamide. Pfizer since acquired Medivation.

Roche. On June 25, Roche announced that its Phase III IMpower 133 clinical trial of Tecentriq (atezolizumab) plus carboplatin and etoposide chemotherapy in extensive-stage small cell lung cancer (ES-SCLC) met its co-primary endpoints. This is yet another win for the company’s hopes of bringing the first immunotherapy combination built-in Tecentriq for lung cancer.

The interim data analysis showed that people who received the Tecentriq-chemotherapy combination lived significantly longer than patients that receive just the chemotherapy alone. The Tecentriq-combination also cut the risk of disease worsening or death (PFS) compared to the chemo-only group.

Merck and AstraZeneca. On June 27, AstraZeneca and Merck announced positive results from their Phase III SOLO-1 clinical trial of Lynparza (olaparib) in BRCA-mutated (BRCAm) advanced ovarian cancer.

The trial, evaluating the drug in this population as a first-line maintenance therapy, had a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to those receiving placebo. Safety and tolerability were consistent with other clinical trials. The two companies expect to begin talks with regulatory agencies.

Global Blood Therapeutics (GBT). Also on June 27, GBT, based in South San Francisco, reported the results of a review of Part A of its Phase III HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study of voxelotor for sickle cell disease.

The trial met its primary endpoint, the proportion of patients with greater than 1 g/dL increase in hemoglobin versus baseline, which was a statistically significant increase at both the 1500 mg and 900 mg doses after 12 weeks compared to placebo. Of the patients receiving the 1500 mg dose, 58 percent had a greater than 1 g/dL increase in hemoglobin at 12 weeks compared to 9 percent of patients taking a placebo. Of the patients receiving the 900 mg dose, they had a 38 percent increase. The company stated, “This compares favorably to the hemoglobin increase assumption agreed to with the U.S. Food and Drug Administration (FDA) in the HOPE Study protocol of a 35 percent response.”

Eli Lilly. On June 29, Eli Lilly and Company announced the results of its COAST-W Phase III clinical trial of Taltz (ixekizumab) to treat Ankylosing Spondylitis (AS). The trial met both primary and major secondary endpoints.

AS is a type of arthritis that primarily affects the spine, although it can affect other joints as well. It causes inflammation of the vertebra and can lead to severe and chronic pain and discomfort. In its most advanced cases, the inflammation can lead to ankylosis, new bone formation in the spine, which causes segments of the spine to fuse in a fixed, immobile position. It can also cause inflammation, pain and stiffness in the shoulders, hips, ribs, heels and small joints of the hands and feet. Sometimes the eyes are involved and in rare cases, the heart or lungs. Patients typically have symptoms before the age of 30.

Taltz showed a statistically significant improvement in the symptoms of AS. It was measured by the proportion of patients who achieved Assessment of Spondyloarthritis International Society 40 (ASAS40) response at 16 weeks compared to placebo. The COAST-W program is the first to use ASAS40 across the program as the primary endpoint to define treatment success, instead of the traditional endpoint of ASAS20.

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