It was another busy week for clinical trial news. Here’s a look.
It was another busy week for clinical trial news. Here’s a look.
COVID-19-Related
RedHill Biopharma announced plans to expand its Phase II/III trial of opaganib in severe COVID-19 in the U.S., based on FDA review of the Phase II trial. The expansion will help speed enrollment and expand the trial to eight additional countries and about 40 recruiting sites.
Moderna reported yesterday that it had developed a new form of its COVID-19 vaccine designed specifically to handle the South African variant. A sample of the vaccine has been delivered to the National Institutes of Health (NIH) for testing to determine if boosting people with this vaccine will stimulate a strong immune response.
Pfizer and BioNTech announced they initiated a study on the safety and immunogenicity of a third dose of their COVID-19 vaccine to better understand the effect of a booster on immunity against COVID-19 caused by the circulating variants as well as any potential new variants. It will draw on participants from the U.S.-based Phase I trial who will be offered the chance to receive a 30-microgram booster of the current vaccine six to 12 months after receiving their initial two-dose shots.
Also, the two companies are in discussions with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) over a registration-enabling clinical trial to evaluate a variant-specific vaccine with a modified mRNA sequence. It would be based on the South African variant and would allow the companies to update the current vaccine fast if necessary. They hope that they can follow the regulatory pathway that is used for flu vaccines, where modifications to the existing vaccines are made, testing quickly in animal models, then authorized.
Altimmune began enrollment in a Phase I trial of AdCOVID, a single-dose intranasal COVID-19 vaccine candidate. The vaccine is an adenovirus-vector vaccine designed to stimulate both systemic immunity and local immunity in the nasal cavity and respiratory tract.
Merck was told by the FDA that the agency needed additional data beyond the Phase III trial in order to authorize its experimental COVID-19 drug MK-7110. MK-7110 is an immune modulator being evaluated as a treatment for COVID-19 hospitalized patients with severe disease. Topline data from the Phase III trial indicated a 60% higher probability of improvement compared to placebo, as well as more than 50% reduction in the risk of death or respiratory failure.
AIVITA Biomedical completed the Phase I trial in Indonesia of its personalized vaccine candidate, AV-COVID-19. The 27-volunteer trial demonstrated the vaccine was well-tolerated with no serious adverse events and that it produced antibodies.
Non-COVID-19-Related
Otonomy announced that its Otividex to treat Meniere’s disease failed to hit the primary endpoint in its Phase III trial. Meniere’s disease is a condition of the inner ear that can cause vertigo. The primary endpoint was the count of definitive vertigo days (DVD) in Month 3 compared to placebo. The evaluation used the Negative Binomial Model. The data did not hit statistical significance for the per protocol (PP) population. Otividex is a sustained-exposure formulation of the steroid dexamethasone.
MatriSys Bioscience and the University of San Diego School of Medicine published research from its Phase I study of MSB-0221 in atopic dermatitis. MSB-0221 is a proprietary formulation containing Staphylococcus hominis (ShA9). The study demonstrated that skin diseases can be treated by modulating the populations of the skin microbiome. The trial met its primary endpoint.
vTv Therapeutics published the results of the SimpliciT-1 Study of TTP399 as an adjunct to insulin in adults with type 1 diabetes. TTP399 is a novel, hepatoselective glucokinase activator. The Phase II trial was run in two parts under a treat-to-target protocol in T1D patients over 12 weeks of daily dosing after a multi-week insulin optimization and placebo run-in period.
TG Therapeutics published final results from the Phase III GENUINE trial of ublituximab in r/r high-risk chronic lymphocytic leukemia (CLL). Ublituximab is an investigational glycoengineered anti-CD20 monoclonal antibody. It was evaluated in combination with ibrutinib. The combination significantly improved overall response rate, complete response rate and prolonged progression-free survival.
Nabriva Therapeutics published the results of a retrospective cohort study of 3,626 patients with a positive S. pneumoniae blood or respiratory culture and its macrolide resistance in the U.S. The study reinforced the need to combat the public health risks of antibiotic resistance. Essentially, they found high rates of anti-microlide resistance in S. pneumoniae infections.
bluebird bio and Bristol Myers Squibb published results from the pivotal Phase II KarMMa study of their B-cell maturation antigen (BCMA)-directed CAR T therapy, idecabtagene vicleucel (ide-cel; bb2121) in adults with r/r multiple myeloma who have received at least three previous therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. The trial hit its primary endpoint of overall response rate and key secondary endpoint of complete response rate.
Aimmune Therapeutics presented new clinical data from a pooled analysis of three Phase III trials and three open-label extension trials of Palforzia [peanut (Arachis hypogaea) Allergen Powder-denfp]. In patients with peanut allergy between ages 4 and 17 who had daily treatment for Palforzia for up to 3.5 years, adverse events were mostly mild, occurred early in the treatment, and declined over the course of treatment.
Genentech announced results from the dose finding Part 1 of the FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy (SMA). They were evaluating Evrysdi (risdiplam). The data demonstrated that Evrysdi at 12 months helped 90% of the infants survive without permanent ventilation and 33% sit without support for at least 5 seconds.
Karuna Therapeutics published data from the EMERGENT-1 Phase II trial of KarXT in schizophrenia. The drug demonstrated a statistically significant and clinically meaningful 11.6-point decrease in the Positive and Negative Syndrome Scale (PANSS) total score compared to placebo at Week 5. The drug is an oral modulator of muscarinic cholinergic receptors, which combines xanomeline, a novel muscarinic agonist, with trospium, an FDA-approved muscarinic antagonist that doesn’t cross the blood-brain barrier.
Kronos Bio dosed the first patient in the Phase I/II trial of KB-0742 in MYC-amplified solid tumors. The drug is a highly selective, orally bioavailable cyclin dependent kinase 9 (CDK9) inhibitor.
Clene Nanomedicine presented blinded interim data from its VISIONARY-MS study and updated interim data from its REPAIR-MS. Together, the Phase II studies evaluated the efficacy, safety, and CNS metabolic effects of CNM-Au8, a bioenergetic nanocatalyst in multiple sclerosis. Baseline scores for the participants with mild disease demonstrated less neurological impairment than those of the overall study population.
SpringWorks Therapeutics announced interim data from its Phase IIb ReNeu Trial of mirdemetinib for patients with NF1-PN. The trial is about 70% enrolled with full enrollment expected in the second half of this year. The data was on the first 20 patients enrolled, and as of the January 22 cutoff data, 50% had achieved an objective response.
Salarius Pharmaceuticals initiated the expansion stage of its ongoing Phase I/II trial of seclidemstat in r/r Ewing sarcoma and Ewing-related sarcomas, also known as FET-translocated sarcomas. Seclimdemstat is a novel, oral, reversible inhibitor of the lysine-specific histone demethylase 1 enzyme (LSDA), which plays a key role in the development and progression of certain cancers.
AlloSource enrolled the first patients in its clinical study of AlloWrap for two-level anterior cervical discectomy and fusion procedures. AlloWrap is an amniotic membrane designed to decrease post-operative soft tissue inflammation.
Elicio Therapeutics received the go-ahead from the FDA for its ELI-002 cancer vaccine trial. ELI-002 is an Amphiphile (AMP) KRAS vaccine containing AMP mKRAS peptides and a proprietary AMP CpG adjuvant, administered subcutaneously. The Phase I/II trial will identify patients with pancreatic ductal adenocarcinoma (PDAC) and other solid tumors who have undergone standard of care surgery and neoadjuvant/adjuvant chemotherapy that have persistent positive circulating tumor DNA.
