Clinical Catch-Up: February 24-28


The month of February ended with a flurry of clinical trial announcements. Here’s a look.

NGN Biopharmaceuticals announced positive preliminary topline results from the 24-week Cohort 4 of its Phase II trial of aldafermin in biopsy-confirmed non-alcoholic steatohepatitis (NASH) with stage 2 or 3 liver fibrosis. Aldafermin is an engineered variant of the human hormone FGF19 developed as a once-daily treatment for NASH. Cohort 4 was powered to show the effect of aldafermin compared to placebo on the primary endpoint of change in absolute liver fat content (LFC), which hit statistical significance.

Galera Therapeutics presented final data from the two-year tumor outcomes follow-up of avasopasem manganese for severe oral mucositis (SOM) in its Phase IIb clinical trial. Avasopasem manganese is a highly selective small molecule superoxide dismutase (SOD) mimetic.

Allecra Therapeutics indicated its Exblifep (cefepime-enmetazobactam) hit pre-specified primary endpoints in its Phase III ALLIUM clinical trial in complicated urinary tract infections (cUTI), including acute pyelonephritis (AP). It also demonstrated superiority over piperacillin-tazobactam. Exblifep is a combination of a novel extended-spectrum beta-lactamase inhibitor, enmetazobactam, and the fourth-generation cephalosporin cefepime.

Overall success was 79.1% for patients receiving Exblifep compared to 58.9% of piperacillin-tazobactam. Patients who stopped taking the drugs were comparable, with 5.2% in the Exblifep cohort and 4.0% in the piperacillin-tazobactam cohort. Exblifep was well tolerated, with 4.3% of patients reporting serious adverse events compared to 3.7% in the piperacillin-tazobactam group.

Spring Bank Pharmaceuticals announced a new clinical collaboration with Roche to test SB 11285, Spring Bank’s IV-administered STING agonist with Roche’s checkpoint inhibitor Tecentriq (atezolizumab) in advanced solid tumors. The company dosed the first patient in the monotherapy dose escalation component of the Phase Ia trial in the fourth quarter of 2019. This will now be followed by a combination dose escalation with Tecentriq and with Bristol-Myers Squibb’s checkpoint inhibitor Opdivo (nivolumab).

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Acer Therapeutics completed and announced the final data from Part B of its pivotal trial of ACER-001. ACER-001 is a proprietary, taste-masked formulation of sodium phenylbutyrate for Urea Cycle Disorders (UCDs). The trial evaluated 36 healthy adults in a single-center, single-blind, randomized, single-dose crossover study to equate bioequivalence of ACER-001 compared to Buphenyl. Consistent with Part A, data from Part B showed ACER-001 to be bioequivalent to Buphenyl.

Amgen, Cytokinetics and Servier announced that the Data Monitoring Committee for GALACTIC-HF completed the second and final planned interim analysis and recommended the Phase III trial of omecamtiv mecarbil continue without changes. Omecamtiv mecarbil is a novel, selective cardiac myosin activator that binds to the catalytic domain of myosin. It is being evaluated in heart failure in more than 8,200 patients in 35 countries who were either hospitalized at the time of enrollment for heart failure or had a hospitalization or admission to an emergency room for heart failure within one year before screening.

Seelos Therapeutics announced additional interim data from its Phase I pharmacokinetic/pharmacodynamic (PK/PD) studies of intranasal Racemic Ketamine (SLS-002) for Major Depressive Disorder (MDD). It included all 42 patients from SLS-002-101 and 38 of 62 patients from SLS-002-102. Both studies showed that the four doses of the drug are generally safe and well-tolerated.

Menlo Therapeutics announced results from its Phase II trial of daily oral serlopitant for chronic pruritus (itch) of unknown origin (CPUO). The trail of 233 patients failed to meet its primary endpoint. There were no meaningful differences between serlopitant and placebo groups.

Fulcrum Therapeutics completed patient enrollment in ReDUX4, its Phase IIb trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD). An open-label extension for participants in ReDUX4 who completed 24 weeks of dosing was also launched. FSHD is a progressive disease-causing skeletal muscle loss, initially showing weakness in muscle in the face, shoulders, arms and trunk, and progessing throughout the lower body. Losmapimod is a selective p38alpha/beta mitogen-activated protein kinase (MAPK) inhibitor in-licensed from GlaxoSmithKline.

Gilead Sciences announced it will launch two Phase III clinical trials of its investigational antiviral drug remdesivir in adults diagnosed with COVID-19, the disease caused by the novel coronavirus. Gilead’s trials will evaluate two dosing durations of the drug, which is given intravenously. The randomized, open-label, multicenter studies will enroll about 1,000 patients mostly in Asia, as well as in countries that have had high numbers of diagnosed cases. The trials are planned to start in March.

These trials are on top of two clinical trials in China’s Hubei province led by the China-Japan Friendship Hospital and a recently launched trial in the U.S. led by the National Institute of Allergy and Infectious Diseases (NIAID). Gilead donated the drug and provided scientific expertise for those trials. The China trial data is expected in April.

Remdesivir is a nucleotide analog with broad-spectrum antiviral activity in in vitro studies and in in vivo studies in animals against Ebola, Marburg, MERS and SARS. MERS and SARS are both caused by coronaviruses that have at least some similarities to the coronavirus causing COVID-19.

BrainStorm Cell Therapeutics announced that Partners MS Center at Brigham and Women’s Hospital has contracted with the company as a clinical trial site for its Phase II trial of NurOwn in progressive multiple sclerosis (MS). NurOwn, autologous MSC-NTF cells, are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo.

Advaxis updated results from the combination arms of its Phase I/II trial of ADXS-503 in non-small cell lung cancer (NSCLC). The trial is studying ADXS-503, part of the company’s ADXS-HOT immunotherapy program using its Lm technology to target hotspot mutations in specific cancer types alone and in combination with Merck’s checkpoint inhibitor Keytruda (pembrolizumab). The trial showed substantial tumor shrinkage in the first two patients in the combination arm, Part B, with one patient hitting a partial response and the other achieving stable disease with 25% reduction in a target lesion.

MapKure, BeiGene and SpringWorks Therapeutics dosed the first patient in Australia in a Phase I trial of BGB-3245 in advanced or refractory solid tumors. The FDA has also approved the IND for it, allowing study expansion in the U.S. BGB-3245 is a next-generation B-RAF inhibitor.

Abivax dosed the first patient in a U.S. Phase I/II trial of ABX196 for hepatocellular carcinoma (HCC), the most common type of liver cancer. ABX196 is an invariant Natural Killer T-cell (iNKT) agonist. It is administered with Bristol-Myers Squibb’s checkpoint inhibitor Opdivo (nivolumab).

Oramed Pharmaceuticals announced positive topline data from the second and final cohort of its Phase IIb trial of ORMD-0801, its lead oral insulin candidate. The trial evaluated a 90-day regimen of different doses of the drug in patients with inadequate glycemic control on oral antihyperglycemic agents, evaluating the change in A1C from baseline to week 12.

Bayer published data showing Vitrakvi (larotrectinib) efficacy and safety in adult and pediatric patients with TRK fusion cancer. The ORR was 73% in adults, 92% in pediatric patients, and 75% for brain metastases/CNS. The median duration of response was almost three years with median progression free survival (mPFS) of 28.3 months.

Mereo BioPharma announced the successful completion of a Type B End-of-Phase II meeting with the FDA to discuss setrusumab for osteogenesis imperfecta (OI). The FDA agreed on the design of a Phase III pediatric study in OI to be completed before the submission of a BLA. Setrusumab is a fully humanized monoclonal antibody that inhibits sclerostin, a protein that inhibits the activity of bone-forming cells.

BioDelivery Sciences International presented the results of a study of its Phase I trial of buprenorphine buccal film and oral oxycodone hydrochloride. Belbuca (buprenorphine buccal film) is indicated for management of pain severe enough to require around-the-clock, long-term opioid treatment. The study showed that Belbuca’s effect on respiratory drive was comparable to placebo at all doses.

Galera Therapeutics announced full tumor outcomes results from the two-year follow-up of patients with head and neck cancer treated with GC4419 (avasopasem manganses). GC4419 is the company’s lead product candidate for severe oral mucositis (SOM). It was evaluated in a Phase IIb clinical trial. The drug is a highly selective small molecule superoxide dismutase (SOD) mimetic. The drug maintained anti-cancer benefit of chemoradiation while reducing SOM.

Incysus initiated a Phase I trial of Drug Resistant Immunotherapy (DRI) for newly-diagnosed glioblastoma. The company’s DRI approach combines conventional chemotherapies with a γδ T-cell-based immunotherapy to modify the tumor microenvironment and stimulate the immune system. γδ T-cells are a type of white blood cells that helps the immune system recognize and kill cancer cells.

Antibe Therapeutics enrolled the final patients in its Phase IIb trial of ATB-346 in osteoarthritis pain. The drug is a hydrogen sulfide-releasing derivative of naproxen. The final patients will be treated for two weeks, followed by a two-week monitoring period and final data analysis of the primary endpoint.

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