Clinical Catch-Up: May 4-8

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It’s the first time in several weeks that the number of non-COVID-19-related clinical trial stories outnumber the COVID-19-related stories. That is likely because the COVID-19 studies were recently initiated. It’s also a hopeful sign that some of the rest of the clinical trials are getting back on track. Here’s a look.

COVID-19-Related

CytoDyn reported that it expects to complete enrollment for its 75-patient, Phase II trial of leronlimab for COVID-19. The FDA has approved 54 emergency INDs for leronlimab treatment of COVID-19, and the company has requested compassionate use authorization from the FDA for COVID-19 patients not eligible for participation in two ongoing U.S. clinical trials.

ioTech International indicated that three of its products, ioRinse Oral Rinse, ioCleanse Hand Cleanser and ioMist Nasal Spray, based on its non-staining formulations of molecular iodine, are being used in a clinical trial at St. Joseph’s Hospital University Medical Center in Patterson, New Jersey. The endpoint of the six-week study is reduction in COVID-19 symptoms from healthcare workers using the products who are treating COVID-19 patients.

Adaptive Biotechnologies and Microsoft began enrolling a virtual clinical study, ImmuneRACE, to rapidly map and measure the immune response to the COVID-19 virus in order to improve diagnostics. The study calls for 1,000 participants in various U.S. metro areas impacted by COVID-19. De-identified data will be made freely available to public health authorities, academia and industry.

Moderna received approval for a Phase II trial of its mRNA vaccine for COVID-19, mRNA-1273. It expects to begin the study shortly and is already finalizing a Phase III protocol, that it hopes to begin in early summer of 2020.

Evelo Biosciences, Rutgers University and Robert Wood Johnson University Hospital announced they had submitted an IND for an Evelo-sponsored Phase II trial of EDP1815 for hospitalized patients with newly diagnosed COVID-19. The trial will evaluate 60 patients. EDP1815, in a Phase Ib trial in psoriasis, blunted the production of multiple inflammatory cytokines, including IL-6, IL-8, TNF, and IL-1b.

CalciMedica announced the Independent Safety Review Committee (ISRC) recommended its open-label Phase II trial of CM4620-IE in patients with severe COVID-19 pneumonia continue as planned. CM4620-IE is a potent and selective small molecule CRAC channel inhibitor that prevents CRAC channel overactivation. The trial aims to enroll 120 patients with COVID-19 pneumonia on oxygen therapy.

Yet another clinical study, this one analyzing the use of malaria drug hydroxychloroquine at New York-Presbyterian Hospital, suggests the drug is not helpful in treating COVID-19. The research was published in the New England Journal of Medicine on Thursday, May 7. The latest study looked at the link between using the drug and intubation or death at New York-Presbyterian Hospital. The primary endpoint was composite of intubation or death in a time-to-event analysis. The authors evaluated 1,446 patients. Of them, 70 were intubated, died, or discharged within 24 hours after presentation and so were excluded from the analysis. Of the remaining 1,376, in a median follow-up of five days, 45.8% of patients were treated within 24 hours after arriving in the emergency room, and 85.9% were treated within 48 hours. The study found no significant link between use of hydroxychloroquine and intubation or death.

RedHill Biopharma received the go-ahead from the FDA for its Phase IIa trial of Yeliva (opaganib) in moderate-to-severe COVID-19. A total of 139 patients have been dosed to date in the completed Phase I and Phase II trials of the drug in cancer. This Phase IIa will enroll up to 40 patients. The drug is a proprietary, first-in-class, oral spingosine kinase-2 (SK2) selective inhibitor with anticancer, anti-inflammatory and anti-viral activities.

Novant Health initiated a Phase IIb/III clinical trial of CytoDyn’s leronlimab for severely and critically ill COVID-19 patients. Leronlimb is a CCR5 antagonist developed for multiple therapeutic indications, primarily in cancer, but in earlier studies has shown effectiveness in treating COVID-19.

Non-COVID-19-Related

Newron Pharmaceuticals announced topline results from its STARS clinical trial of sarizotan in Rett syndrome. The drug failed to hit the primary or secondary efficacy endpoints. As a result, the company is terminating the program. Rett syndrome is a severe neurodevelopment disorder. It mostly affects females, about one in 10,000. There are no approved treatments.

CEL-SCI Corporation hit the targeted threshold of 298 deaths required to conduct the data evaluation for its pivotal Phase III head and neck cancer study of Multikine (Leukocyte Interleukin, Inj.) immunotherapy. The database is being prepared for database lock. Once locked, the final analysis of the trial can be performed. CEL-SCI will stay reminded to the study results throughout the process. In the study, patients who were recently diagnosed with advanced primary squamous cell carcinoma of the head and neck were treated with Multikine before they received surgery, radiation and/or chemotherapy. Multikine is designed to help the immune system “see” the tumor when the immune system is still relatively intact and better able to mount an attack on the cancer.

Nabriva Therapeutics published results from a post-hoc analysis of clinical data from patients receiving hospital treatment from the pivotal Lefamulin Evaluation Against Pneumonia (LEAP) 1 and LEAP 2 Phase III clinical trials. The results indicate Xenleta (lefamulin) results in a rapid and similar time to clinical response compared to moxifloxacin. Xenleta is a first-in-class pleuromutilin antibiotic approved by the FDA for community-acquired bacterial pneumonia.

Galecto announced new data describing both preclinical and clinical evidence for anti-fibrotic activity for GB-139. It is being developed for most forms of lung fibrosis with initial focus on idiopathic pulmonary fibrosis (IPF). The clinical data was from its Phase IIb trial, with a readout expected in 2022. To date, the data showed the drug is highly effective in the bleomycin model, including pharmacodynamic evidence with reduction of galectin-3.

Galecto also recruited 150 patients in its Phase IIb GALACTIC-1 trial of GB-139 in idiopathic pulmonary fibrosis (IPF). GB0139 is a potent inhaled small molecule inhibitor of galectin-3, a protein that plays a central role in various organ fibrosis. The study is recruiting from more than 100 centers in the U.S., Europe and Canada with a goal of enrolling 450 patients.

Akebia Therapeutics announced positive topline data from INNO2VATE, the first of two global Phase III cardiovascular outcomes programs. The two INNO2VATE trials studied the efficacy and safety of vadadustat, the company’s oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) compared to darbepoetin alfa for anemia caused by chronic kidney disease (CKD) in adults on dialysis. The drug hit the primary and key secondary efficacy endpoints, snowing non-inferiority to darbepoetin alfa.

TG Therapeutics announced positive topline results from its UNITY-CLL Phase III trial studying the combination of umbralisib plus ublituximab (U2) compared to obinutuzumab plus chlorambucil in patients with previously untreated and relapsed/refractory chronic lymphocytic leukemia (CLL). The trial met the primary endpoint at a prespecified interim analysis showing a statistically significant improvement in progression-free survival. As a result, the trial will be halted early for superior efficacy.

Angion Biomedica published results from its Phase II trial of ANG-3777 in transplant-associated acute kidney injury. The data describes the results of ANG-3777 in patients who underwent deceased-donor kidney transplantation and had signs of kidney injury. ANG-3777 is a small molecule designed to mimic the biological activity of hepatocyte growth factor (HGF), which activates the c-Met cascade of pathways involved in tissue repair and organ recovery.

Sanofi and Regeneron Pharmaceuticals announced topline data for their pivotal Phase II trial of Libtayo (cemiplimab) in advanced basal cell carcinoma (BCC) patients that had progressed on or could not tolerate previous hedgehog pathway inhibitor (HHI) therapies. Libtayo is a PD-1 checkpoint inhibitor. The drug showed clinically meaningful and durable responses in the patients. The companies expect to make regulatory submissions this year.

Rebiotix and Ferring Pharmaceuticals announced positive preliminary results from their ongoing pivotal Phase III trial of RBX2660. The therapy is being developed to decrease C. difficile (C. diff) infection recurrences. RBX2660 is an investigational formulation of a first-in-class microbiota-based therapy. RBX2660 is derived from fecal samples, frozen, and delivered to patients via an enema. The 270-patient placebo-controlled trial met the primary goal, the percentage of patients receiving the therapy to prevent recurrent C. diff in eight weeks compared to those receiving the placebo.

Researchers with City of Hope and Leiden University Medical Center in the Netherlands, published Phase I trial data from a vaccine against type 1 diabetes. Nine participants were monitored for six months after receiving the injections. No signs of systemic immune suppression or major adverse reactions were seen.

SCYNEXIS published six abstracts for the 30th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID) abstract book detailing Phase III results and other testing of its ibrexafungerp for treating invasive fungal infections, including Candida auris. Ibrexafungerp is an investigational antifungal agent and the first representative of a novel class of structurally distinct glucan synthase inhibitors, the fungerps.

NuCana re-commenced enrollment of new patients in NuTide:121, its ongoing global Phase III trial of Acelerin plus cisplatin in biliary tract cancer. The re-opening started in specific areas, including Australia, Canada, South Korea, Taiwan, Ukraine and the UK. The trial will enroll up to 828 patients in about 120 sites. Acelerin is a new chemical entity derived from the nucleoside analog gemcitabine.

Genentech/Roche presented two abstracts at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting on their Phase II trial of faricimab for wet AMD and Phase III results of satralizumab for neuromyelitis optica spectrum disorder (NMOD). The FDA has accepted Genentech’s BLA for satralizumab for NMOSD in October 2019 with a PDUFA date later this year.

Axcella announced positive topline data from AXA1125-003 clinical trial evaluating AXA1125 and AXA1957 in non-alcoholic fatty liver disease (NAFLD). In the non-IND study, 102 adults with NAFLD presumed to have NASH were split 2:2:2:1 to receive AXA1125, one of two AXA1957 doses, or a placebo twice daily for 16 weeks. The two drugs were well-tolerated, demonstrating sustained decreases in key biomarkers of metabolism, inflammation and fibrosis.

Rubius Therapeutics dosed the first patient in its Phase I/II trial of RTX-240 for relapsed/refractory or locally advanced solid tumors. RTX-240 is an allogeneic off-the-shelf Red Cell Therapeutic engineered to mimic the human immune system by stimulating adaptive and innate immunity. It is engineered to express a co-stimulatory molecule, F-1BB ligand, and a cytokine, IL-15TP, on the cell’s surface that activates and expands both natural killer (NK) cells and T-cells.

Ovid Therapeutics announced positive topline results from the Phase II ROCKET trial of OV101 for Fragile X syndrome. The drug, gaboxadol, is a novel delta-selective GABAA receptor agonist. The primary endpoint was the safety and tolerability of the drug over 12 weeks in three different active dose arms, which it met for all three doses. It also showed a statistically significant effect on secondary behavioral endpoints, Aberrant Behavior Checklist-Community for Fragile X syndrome, and Anxiety, Depression and Mood Scale (ADAMS).

Burning Rock initiated its 14,000-patient PREDICT trial to validate its pan-cancer early detection technology in China. It will collaborate with Shanghai Zhongshan Hospital and other Chinese medical centers to test the blood-based circulating cell-free DNA (cfDNA) test for pan-cancer early detection on its ELSA-seq technology for the detection of methylation profiles.

Quantum Genomics announced interim analysis QGC001/1QG4, its trial of firibastat in end-stage renal failure (ESRD) patients. The study showed that, because of a slower elimination, exposure to firibastat and its main metabolites is relatively more important in ESRD patients compared to healthy volunteers, without a significant increase in peak concentration.

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