Clinical Catch-Up: August 10-14

Clinical Research

It was a very busy week for clinical trial updates. Here’s a look.

COVID-19-Related

Omeros Corporation reported data from a compassionate-use study of narsoplimab in COVID-19 patients with Acute Respiratory Distress Syndrome (ARDS). The drug is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-12 (MASP-2). All patients initially required mechanical ventilation and all recovered and survived with narsoplimab treatment.

Pharming Group enrolled the first patient in a randomized, controlled, investigator-initiated trial in up to 150 patients with confirmed COVID-19 to be treated with Ruconest (recombinant human C1 inhibitor). In April of this year, the company reported encouraging data from a passionate-use program at the University Hospital Basel, Switzerland in five patients with COVID-19 and related severe pneumonia.

Romarck initiated a Phase III trial of NT-300 (nitazoxanide extended-release tablets) for mild or moderate COVID-19. It will study up to 800 people 12 years and older with COVID-19. In cell cultures, nitazoxanide inhibits replication of a broad range of respiratory viruses, including SARS-CoV-2.

Arcturus Therapeutics announced all subjects in the first cohort in its Phase I/II trial have been dosed with ARCT-021, its investigational vaccine for COVID-19. It is being conducted with CTI Clinical Trial and Consulting Services and in collaboration with Duke-NUS Medical School in Singapore.

Mayo Clinic published preliminary results from a three-month study in more than 35,000 patients of the use of convalescent plasma on COVID-19 patients. Patients who received transfusions of convalescent plasma within three days of diagnosis demonstrated a seven-day death rate of 8.7%. Patient who received plasma after four or more days had a mortality rate of 11.9%. This was deemed statistically significant.

However, critics are struggling to fully analyze the data, because there was no placebo group to compare the data to.

China National Pharmaceutical Group (Sinopharm) reported that its COVID-19 vaccine appeared to be safe and triggered antibody immune responses in early and mid-stage trials. The trial had already moved to a Phase III trial in the United Arab Emirates and is expected to supply the vaccine to Pakistan as part of a trial agreement. There were no serious side effects. The trials were based on data form 320 healthy adults in Phase I and II clinical trials.

Non-Covid-19-Related

Seres Therapeutics announced positive topline data from the pivotal Phase III ECOSPOR III trial of its oral microbiome therapy SER-109 for recurrent C. difficile infection (CDI). The drug demonstrated a highly statistically significant absolute decrease of 30.2% in the percentage of patients who had a CDI recurrence within eight weeks of receiving the drug compared to placebo. SER-109 is an investigational, oral, biologically-derived microbiome therapeutic. It is made up of a “highly-purified consortia of spore-based commensal bacteria and designed to be manufactured in accordance with Good Manufacturing Practice conditions,” Seres describes.

Genentech, a Roche company, announced topline data from its Phase III program of etrolizumab in moderately to severely active ulcerative colitis. The data were mixed, with data from both studies analyzing the drug as a maintenance therapy did not meet the primary endpoints. In the HIBISCUS I induction study, people without prior anti-tumor necrosis factor (anti-TNF) treatment, the drug met the primary endpoint. Etrolizumab is a dual anti-integrin that targets inflammatory bowel diseases (IBD) by selectively inhibiting alpha4ß7 and alphaEß7.

Precigen ActoBio announced that the Phase Ib monotherapy portion of the ongoing Phase Ib/IIa trial of AG019 ActaBiotics for early-onset type 1 diabetes. AG019 is an oral capsule consisting of engineered Lactococcus lactis modified to deliver autoantigen human proinsulin (hPINS) and the tolerance-enhancing cytokine human interleukin-10 to the mucosal lining of the gastrointestinal tissues.

Kadimastem announced that AstroRx showed a positive efficacy signal and a good safety profile in Cohort B of its Phase I/IIa clinical trial in amyotrophic lateral sclerosis (ALS). AstroRx is a cell therapy product that contains functional, healthy astrocytes, which are nervous system support cells, derived from human Embryonic Stem Cells (hESC). They are designed to protect diseased motor neurons through several mechanisms of action.

Bristol Myers Squibb announced positive data from two separate Phase III clinical trials of its checkpoint inhibitor Opdivo (nivolumab).

First, in CheckMate -649, Opdivo was evaluated with chemotherapy compared to chemotherapy alone as first-line treatment for metastatic gastric cancer, gastroesophageal junction (GEJ) cancer or esophageal adenocarcinoma. It met the two primary endpoints, overall survival (OS) and progression-free survival (PFS), with the OS identified at a pre-specified interim analysis and PFS at a final analysis, both in patients whose tumors express PD-L1 with a combined positive score greater than or equal to five. The OS benefit was also seen in the all-randomized population.

The second trial is the Phase III CheckMate -577 trial of Opdivo as an adjuvant therapy for patients with resected esophageal or gastroesophageal junction (GEJ) cancer. This trial also met its primary endpoint of disease-free survival (DFS) at a pre-specified interim analysis. Treatment with Opdivo after neoadjuvant chemoradiation therapy (CRT) and complete surgical resection showed a statistically significant improvement in DFS compared to placebo in the all-randomized population.

City of Hope plans to initiate a clinical trial to test the feasibility of delivering chemotherapy via aerosols. It is PIPAC: HIPEC, a heated chemotherapy procedure that calls for surgical removal of tumors prior to flooding the abdominal cavity with scalding chemotherapy. In PIPAC, surgeons use trocars to inflate the abdomen, then liquid chemotherapy is turned into a fine spray that is blown into the patient, then after 30 minutes of soaking, the droplets are vacuumed out.

Tyme Technologies announced a positive outcome of an interim futility review for the HopES Sarcoma Phase II trial of SM-88 in Ewing’s Sarcoma and other high-risk sarcomas. SM-88 is an oral modified tyrosine derivative. The company plans to proceed with the current trial design as planned.

Frequency Therapeutics announced it expected to complete enrollment in its Phase IIa study in the fourth quarter of FX-322 for stable sensorineural hearing loss. FX-322 is designed to activate progenitor cells in the ear to regenerate damaged auditory cells and improve hearing.

Eloxx Pharmaceuticals resumed enrollment in its Phase II trial for ELX-02 in cystic fibrosis, after being paused during the COVID-19 pandemic. ELX-02 is a small molecule designed to restore production of full-length functional proteins.

American Gene Technologies received approval from the FDA for the first Phase I human trial of its lead HIV program. It will study AGT103-T, a single-dose, lentiviral vector-based gene therapy developed to eliminate HIV.

Corbus Pharmaceuticals completed enrollment in DETERMINE, a Phase III trial of lenabasum for dermatomyositis. The trial has enrolled 176 subjects. Lenabasum is an oral, small molecule that selectively binds as an agonist to the cannabinoid receptor type 2 (CB2).

Gyroscope Therapeutics initiated its Phase II program of GT005 for geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). GT005 is a gene therapy designed to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein.

The University of Texas MD Anderson Cancer Center published the results of the Phase III VIALE-A trial of venetoclax and azacytidine compared to azacytidine alone in acute myeloid leukemia (AML). The addition of venetoclax, an inhibitor of the BCL-1, demonstrated a median overall survival of 14.7 months compared to 9.6 months in patients receiving azacytidine alone. The trial was supported by AbbVie and Genentech.

Boehringer Ingelheim enrolled the first patient in InPedILD, a global Phase III trial of Ofev (nintedanib) in children and adolescents between six and 17 years of age with clinically significant fibrosing interstitial lung disease (ILD). Ofev is indicated in more than 80 countries for idiopathic pulmonary fibrosis (IPF), as well as in more than 40 countries to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated ILD (SSc-ILD).

Kineta received approval in the Netherlands to initiate a first in-human clinical trial of KCP506 for chronic neuropathic pain. KCP506 is a first-in-class alpha9apha10 nicotinic acetylcholinereceptor (nAChR) antagonist.

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