Clinical Catch-up for July 15-19
Last week was the Alzheimer’s Association International Conference (AAIC), which presented a lot of research, but not necessarily the results of clinical trials. One ongoing clinical study by Wake Forest University School of Medicine is the EXERT study, which is attempting to determine if exercise can protect from memory and cognition problems associated with Alzheimer’s. The trial takes 300 people at high risk for Alzheimer’s and randomly splits them into two groups for 18 months. Half perform aerobic exercise, the other half stretching and flexibility exercises. They participate in memory and cognition tests, as well as brain blood flow, atrophy and protein testing. The study is still looking for another 100 people between the ages of 65 and 89 who don’t regularly exercise who have mild cognitive impairment.
Otherwise, here’s a look at the top clinical trial news from last week.
Neon Therapeutics announced top-line results in its Phase Ib clinical trial of NEO-PV-01, its personal neoantigen vaccine candidate. NEO-PV-01 was being evaluated in combination with Bristol-Myers Squibb’s Opdivo (nivolumab) in advanced or metastatic melanoma, smoking-associated non-small cell lung cancer (NSCLC) and bladder cancer.
In all three cancer types, data showed prolonged and consistent improvements in progression-free survival (PFS) that is similar to that seen with checkpoint inhibitor monotherapy. In 27 patients with metastatic NSCLC, median PFS was 5.6 months. In 21 patients with metastatic bladder cancer, median PFS was 5.6 months. At 13.4-months median follow-up in 34 patients with metastatic melanoma, median PFS hadn’t been reached yet.
GlaxoSmithKline announced that its Zejula (niraparib) hit its primary endpoint in the Phase III PRIMA trial as maintenance therapy in first-line ovarian cancer following platinum-based chemotherapy. The primary endpoint was a statistically significant improvement in progression-free survival for women regardless of their biomarker status. PRIMA is a double-blind, randomized Phase III trial to evaluate Zejula compared to placebo in first-line Stage III or IV ovarian cancer patients.
Biohaven Pharma published data from its Phase III clinical trial of Zydis (rimegepant) for acute migraine in The Lancet. The results showed that the drug compared to placebo experienced rapid and sustained clinical benefits. The trial compared rimegepant to placebo in 1,375 patients, 1,351 who were evaluable. The trial included numerous endpoints, with the drug observed to be superior on 21 prespecified endpoints with statistical significance shown on the co-primary endpoints of pain freedom and freedom from the most bothersome symptom at two hours after dosing.
Mallinckrodt Pharmaceuticals halted its Phase IIb PENNANT clinical trial of Acthar Gel (repository corticotropin injection) for amyotrophic lateral sclerosis (ALS). An Independent Data and Safety Monitoring Board recommended the trial be stopped over concerns about patients developing pneumonia. The IDSMB found a higher rate of patients who received the therapy developed pneumonia than those on placebo. There was also no conclusive evidence the treatment was going to hit its 36-week primary endpoint. The company indicated it had permanently halted the trial and patients would be tapered off the treatment before discontinuing use. Acthar Gel has been approved in the U.S. for 19 different indications, including symptomatic sarcoidosis, an inflammatory disease, and acute exacerbations of multiple sclerosis.
CTI BioPharma met with the FDA over its myelofibrosis candidate, pacritinib. CTI now plans to evaluate 200 mg of the drug twice daily in 180 patients with myelofibrosis and severe thrombocytopenia. It expects to launch the Phase III PACIFIC trial in the third quarter of this year. The Phase II PAC203 dose-finding trial was developed to evaluate the safety and efficacy of three doses of the drug in 150 patients with myelofibrosis. CTI expects topline data from the Phase II part of the trial in the third quarter of this year and is hoping to present results at a scientific conference before the end of the year.
Rafael Pharmaceuticals is expanding its Phase III clinical trial (AVENGER 500) of its CPI-631 (devimistat) in combination with FOLFIRINOX (mFFX) in metastatic adenocarcinoma of the pancreas into Israel. The trial is currently being run in multiple sites in the U.S., and plans to open eight sites in Israel. In Israel, Talia Golan, head of Sheba Pancreatic Cancer Center at Tel Hashomer Hospital, will lead the trials in Israel.
Research presented at the AAIC meeting reported finding higher rates of subjective cognitive decline among lesbian, gay, bisexual and/or transgender (LGBT) Americans compared to cisgender heterosexual individuals. The trial used self-reported data on subjective cognitive decline (SCD). The research presented by Jason Flatt from UCSF found that 14% of participants reported subjective cognitive decline compared to 10% among the cisgender heterosexual participants. Even after adjusting for income, age and race, the LGBT group was 29% more likely to report subjective cognitive decline. The connection is undetermined, although the researchers speculate that it is related to higher rates of depression, stress, inability to work and a lack of regular healthcare access in the LGBT population.
Another group reported on the effectiveness of a first-of-its-kind Alzheimer’s intervention for LGBT older individuals with dementia. They identified unique risk factors of LGBT older adults with dementia, including more likely to live alone (almost 60%), not partnered or married (65%), no children (72%) and no caregiver (59%) compared to older non-LGBT adults with dementia. The intervention study looked at individualized programs of exercise, and behavioral and coping strategies to improve physical function, independence and quality of life.