NCIC Clinical Trials Group

With Monkeypox, COVID-19, and subsequent Long COVID symptoms in addition to persistent diseases like cancer and Alzheimer's, the world is dealing with a lot right now. Luckily, the pharmaceutical world has clinical trials underway to address all of them. SIGA Technologies' product TPOXX (tecovirimat), an antiviral approved in the U.S. for smallpox, is being used under certain conditions for monkeypox, but only under “compassionate use” in the U.S. The NIH is now involved in developing a U.S.-based randomized clinical trial to evaluate the drug for the treatment of monkeypox disease, which will be conducted by the AIDS Clinical Trials Group. SyneuRx International announced results from its Phase II trial of SNB01 (pentarlandir), a novel COVID-19 oral antiviral candidate. The trial evaluated 89 patients who had breakthrough or unvaccinated cases of COVID-19 and were given high-dose, low-dose or placebo. Pantarlandir decreased the inflammation caused by the disease and improved overall health. Pantarlandir blocks coronavirus replication. CymaBay Therapeutics completed enrollment for its Phase III RESPONSE study of seladelpar for Primary Biliary Cholangitis (PBC). The study is evaluating the safety and efficacy of the drug for patients with PBC who have been using ursodeoxycholic acid (UDCA or ursodial) but have not achieved the recommended treatment goal, or who can’t tolerate UDCA. Seladelpar is a first-in-class oral, selective PPAR delta agonist. Eliem Therapeutics reported results from its Phase IIa trial of ETX-810 for lumbosacral radicular pain (LSRP). ETX-810 is a new entity prodrug of the bioactive lipid palmitoylethanolamide (PEA). It is also being evaluated in diabetic peripheral neuropathic pain (DPNP). The study did not achieve statistically signification separation from placebo on the study’s primary endpoint, which was changed from baseline to week 4 in the weekly average of the daily pain score measured with the Pain Intensity Numerical Rating Scale (PI-NRS). This was consistent with the Phase IIa study of the drug in DPNP. As a result, they are discontinuing the development of the drug. Immutep presented new data from its Phase II TACTI-002 trial of eftilagimod alpha for first-line treatment of non-small cell lung cancer (NSCLC). The study hit the primary objective, with an overall response rate (ORR) of 38.6% to the combination of the drug with Merck’s Keytruda (pembrolizumab) and favorable anti-tumor activity. There were also improved secondary endpoints, including Disease Control Rate (DCR) and interim median Progression Free Survival (PFS), across all PD-L1 expression levels. The drug is a soluble LAG-3 fusion protein. Orca Bio announced its Phase III Precision-T trial is open, enrolling and treating patients. It expects to enroll about 174 patients at more than 20 transplant centers across the U.S. It is studying the safety and efficacy of Orca-T compared to standard-of-care allogeneic hematopoietic stem cell transplant. Orca-T is an investigational high-precision cell therapy made up of infusions containing regulatory T-cells, conventional T-cells and CD34+ stem cells derived from peripheral blood from either related or unrelated matched donors. Halozyme Therapeutics announced Roche’s Phase III IMscin001 trial of a subcutaneous formulation of Tecentriq (atezolizumab) with Halozyme’s ENHANZE technology met its co-primary endpoints. The study demonstrated non-inferior levels of Tecentriq in the blood when injected SC compared with IV infusion in cancer immunotherapy-naïve patients with locally advanced or metastatic NSCLC where previous platinum therapy failed. Landos Biopharma announced positive topline data from its Phase Ib trial of NX-13 for ulcerative colitis. The drug is a novel, oral, NLRX1 agonist, dosed once a day for four weeks. The drug was well tolerated. The company plans to initiate a Phase II trial to evaluate the safety, efficacy, and optimal dosing. Athira Pharma presented the first results from its Phase II proof-of-concept trial of fosgonimeton in mild-to-moderate Alzheimer’s. The drug is a small-molecule positive modulator of the HGF/MET neurotrophic factor system. The primary outcome analysis (mITT, MMRM) did not meet statistical significance. However, the company indicates the data gave meaningful insights into the drug’s potential effects, including showing its potential activity, which included a decrease in plasma levels of the fluid biomarker, NfL. Novavax initiated a Phase IIb/III Hummingbird trial to evaluate two doses of its COVID-19 vaccine in younger children ages 6 months through 11 years, followed by a booster at 6 months after the primary vaccination series. The study is expected to enroll 3,600 children in the U.S., Mexico, Colombia, Argentina, Spain, UK, South Africa, the Philippines and Brazil.