Clinical Catch-Up: July 12-16
It was a moderately busy mid-summer week for clinical trial news. Here’s a look.
COVID-19-Related
ARCA biopharma provided an update on its Phase IIb ASPEN-COVID-19 trial of rNAPc2 for hospitalized COVID-19 patients. It has received approval from regulators in Argentina and Brazil to enroll patients in the trial. It is currently enrolling patients at seven sites in the U.S., and expects to enroll the first patients in South America in July. rNAPC2 is a small recombinant protein being developed for RNA virus-associated diseases. It is a selective inhibitor of tissue factor (TF).
ENA Respiratory initiated a Phase I trial of INNA-051 for the prevention of COVID-19. INNA-051 is a potent innate immune agonist that targets the receptor TLR2/6 and is being developed for intranasal delivery to target the primary entry site of viral respiratory infections.
NRx Pharmaceuticals and Quantum Leap Healthcare Collaborative have begun using inhaled Zyesamio (Aviptadil) to treat severely and critically ill COVID-19 patients in the I-SPY COVID-19 Phase II adaptive platform trial. The goal of the trial is to screen a range of promising compounds for severely and critically ill COVID-19 patients. Zyesami has shown a statistically significant increase in the probability of patients being alive and free of respiratory failure at 60 days. Zyesami is a synthetic form of Vasoactive Intestinal Polypeptide (VIP).
Mesoblast Limited presented clinical outcomes from the COVID-19 ARDS Trial of remestemcel-L in ventilator-dependent COVID_19 patients with moderate/severe acute respiratory distress syndrome (ARDS). The trial did not meet the primary endpoint of 43% reduction in overall mortality, although was reduced through 60 days in the prespecified subgroup analysis of 123 patients younger than 65.
Non-COVID-19-Related
Affinavax and Astellas Pharma reported data from the Phase II study of an experimental vaccine for Streptococcus pneumoniae. The vaccine, ASP3772, created an antibody response to each of the 24 polysaccharides; it also created an antibody response to the two conserved pneumococcal proteins. ASP3772 was developed using Affinavix’s proprietary MAPS (Multiple Antigen-Presenting System) technology to generate B-cell (antibody) and T-cell immune protection against S. pneumoniae.
Seattle Children’s Therapeutics published initial findings from the BrainChild-01 Phase I immunotherapy clinical trial of CAR-T therapies for children and young adults with recurrent or refractory brain and CNS tumors. The T-cells are dosed directly into the CNS space by infusing them into the fluid surrounding the brain and spinal cord or into the cavity after tumor resection.
Recce Pharmaceuticals dosed multiple patients with RECCE 327 in a topical Phase I/II trial for the treatment of infected burn wounds. RECCE 327 is a broad-spectrum synthetic polymer antibiotic developed for the treatment of blood infections and sepsis derived from E. coli and S. aureus bacteria.
Cidara Therapeutics announced new analyses from multiple trials of rezafungin. This included efficacy analysis from the Phase II STRIVE trial of the drug for Candidemia and/or Invasive Candidiasis, which demonstrated statistically significant differences in the time to negative blood culture between rezafungin and caspofugin in patients with more aggressive infection. Rezafungin is a novel once-weekly echinocandin for serious fungal infections.
Scynexis presented clinical data on ibrexafungerp in fungal infections, including Candida auris. Ibrexafungertp is from a structurally distinct glucan synthase inhibitor, triterpenoids, for fungal infections.
Enterome dosed the first patient in its Phase I/II SIDNEY trial of EO2463 for indolent non-Hodgkin lymphomas (iNHL). EO2463 is the company’s second OncoMimics vaccine. It is an off-the-shelf microbiome-peptide-based cancer vaccine that combines four microbiome-peptides of B lymphocyte-specific lineage markers.
CytoDyn announced its clinical trial with leronlimab in combination with leronlimab had advanced from Phase Ib to Phase II for metastatic triple-negative breast cancer. Leronlimab is a CCR5 antagonist.
Orizon Genomics received the go-ahead from the Spanish Drug Agency (AEMPS) to run a Phase IIb trial of vafidemstat in patients with schizophrenia. Vafidemstat is an oral, CNS optimized LSD1 inhibitor.
Hepion Pharmaceuticals announced positive topline results for its Phase IIa AMBITION trial of CRV431 for non-alcoholic steatohepatitis (NASH). CRV431 is a cyclophilin inhibitor. The primary outcome measure was incidence of safety and tolerability events compared to placebo. The drug was well tolerated with no serious adverse events.
Galecto resumed recruitment in its Phase IIb GALACTIC-1 trial of GB0139 for Idiopathic Pulmonary Fibrosis (IPF) under a revised protocol submitted to the FDA and other regulatory bodies. Under the new protocol, it is now recruiting additional patients who are not taking nintedanib or pirfenidone at screening. FB0139 is a small molecule galectin-3 inhibitor.
TFF Pharmaceuticals completed enrollment in the Phase I trial for Tacrolimus Inhalation Powder. The company’s Thin Film Freezing (TFF) platform improves the solubility and absorption of poorly water-soluble drugs and is especially well-suited to generate dry powder particles so they can be inhaled, especially to the deep lung.
Salarius Pharmaceuticals added Fox Chase Cancer Center in Philadelphia as an active trial site for the dose-expansion stage of its ongoing study of seclidemstat in patients with relapsed or refractory Ewing sarcoma and advanced FET-rearranged sarcomas. The addition expands the number of active sites to nine. Seclidemstat is a novel, oral, reversible inhibitor of lysine-specific histone demethylase 1 (LSD1).
Genocea Biosciences dosed the first patient in its TiTAN Phase I/IIa study of GEN-011 for solid tumors. TEN-011 is a next-generation solid tumor therapy comprised of NPTs CD4+ and CD8+, which are specific for up to 30 antigens to limit tumor escape.
Equillium plans to initiate a Phase III study of itolizumab for first-line treatment of acute graft-versus-host disease. Itolizumab is a first-in-class anti-CD6 monoclonal antibody that selectively targets the CD6-ALCAM pathway.
The University of Texas MD Anderson Cancer Center reported that a Phase II study of atezolizumab and bevacizumab was well-tolerated and demonstrated a 40% objective response rate (ORR) in patients with advanced malignant peritoneal mesothelioma, a rare cancer of the lining of the abdomen. This is one of the first trials for this cancer, which affects about 300 to 500 people in the U.S. each year.
Advaxis initiated a Phase I trial of ADXS-504 in biochemically recurrent prostate cancer. It will be run at Columbia University Irving Medical Center. ADXS-504 is the company’s off-the-shelf neoantigen immunotherapy.
Incyte published positive data from the Phase III REACH3 trial of Jakafi (ruxolitinib) showing the drug had significantly improved outcomes in patients with steroid-refractory or steroid-dependent chronic graft-versus-host disease (GVHD) compared to best available therapy. Jakafi is a first-in-class JAK1/JAK2 inhibitor.
Vir Biotechnology dosed the first patient in its Phase II MARCH trial of VIR-2218 with VIR-3434 for chronic hepatitis B virus (HBV) infection. VIR-2218 is a small interfering ribonucleic acid (siRNA) designed to inhibit the production of all HBV protines, including X, polymerase, S and core. VIR-3434 is an HBV-neutralizing monoclonal antibody that blocks entry of all 10 genotypes of HBV into liver cells (9hepatocytes), as well as decreasing the level of virons and subviral particles in the blood.
Alexion Pharmaceuticals announced positive topline data from its Phase III trial of Ultomiris (ravulizumab-cwvz) in adults with generalized myasthenia gravis (gMG). Ultomiris is a long-acting C5 complement inhibitor. Complement is part of the immune system. It is approved for adults and children with paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).
Eli Lilly and Banner Alzheimer’s Institute plan to collaborate on a Phase III trial of donanemab in Alzheimer’s disease, the TRAILBLAZER-ALZ3 trial. Donanemab is an antibody that targets a modified form of beta amyloid, N3pG.