Clinical Catch-Up: July 27-31
As usual, there was plenty of clinical trial news last week. Here’s a look.
COVID-19-Related
Moderna launched its Phase III COVE (Coronavirus Efficacy) trial in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID) and BARDA. It is expected to include about 30,000 participants in the U.S., evaluating 100-microgram doses of the vaccine in a randomized, placebo-controlled study. The primary endpoint will be prevention of symptomatic COVID-19 disease. Key secondary endpoints include prevention of severe COVID-19, defined as need for hospitalization, and prevention of SARS-CoV-2 infection regardless of symptoms.
Pfizer and BioNTech picked their COVID-19 vaccine candidate and plan to begin a global Phase II/III safety and efficacy clinical trial. The company had four different vaccine candidates and after reviewing preclinical and clinical data from Phase I/II clinical trials and having discussions with the U.S. Food and Drug Administration (FDA) and other countries’ regulators, selected BNT162b2 to take into larger studies. The vaccine will be dosed at a 30-microgram level with a two-dose regimen. The vaccine is an mRNA product, with the mRNA encoding for the SARS-CoV-2 full length spike glycoprotein (S).
AI Therapeutics launched a Phase II trial in newly diagnosed COVID-19 patients in collaboration with Yale University. The trial will evaluate up to 142 outpatients with LAM-002A to determine if it can reduce viral load in confirmed COVID-19 patients
Can-Fite BioPharma submitted an IND to the FDA for a Phase II trial of piclidenoson for COVID-19. Piclidenoson has been dosed in more than 1,000 patients in previous trials as well as two ongoing Phase III studies for rheumatoid arthritis and psoriasis.
NEOIMMUNETECH received FDA approval for a second study of NT-17 (efineptakin alfa) for COVID-19. The investigator-initiated Phase I study is being run by Jian Li Campian of Washington University School of Medicine, St. Louis.
Genentech’s Actemra did not hit the primary and secondary endpoints in a Phase III trial of hospitalized patients with severe COVID-19 associated pneumonia. It did not hit a secondary endpoint of reduced patient mortality. Actemra is approved for rheumatoid arthritis.
Celltrion Group received approval from the UK regulatory agency for a Phase I trial of CT-P59, a COVID-19 antiviral antibody treatment candidate, in patients with mild symptoms of COVID-19. The company will also begin full-scale commercial production of the drug in September.
Merck announced it is moving its COVID-19 vaccine candidate into Phase I clinical trials in the third quarter. The vaccine is V591, which it picked up when it acquired Austria-based Themis in May. The vaccine uses a measles virus vector platform based on a vector developed by researchers at the Institut Pasteur.
Non-COVID-19-Related
Solid Biosciences received FDA notification that the IGNITE DMD Phase I/II clinical trial remains on clinical hold. The original hold was announced in November 2019 after a serious adverse event (SAE) in the sixth patient to receive the therapy. SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy for Duchenne muscular dystrophy (DMD).
Rigel Pharmaceuticals published post-hoc analysis of a Phase III clinical program of Tavalisse (fostramatinib disodium hexahydrate) in adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The data indicated a higher response rate and decrease in bleeding incidents in ITP patients receiving Tavalisse as a second-line therapy.
Regeneron Pharmaceuticals announced positive two-year data from the Phase III PANORAMA trial of Eylea (aflibercept) Injection 2 mg in patients with moderately severe to severe non-proliferative diabetic retinopathy (NPDR). The data demonstrate that untreated moderately severe and severe NPDR can lead to vision-threatening events, and that more than half of patients in the untreated sham arm developed a vision-threatening complication or center-involved diabetic macular edema within two years of entering the trial, while Eylea treatment decreased the likelihood of these happening by at least 75%.
AstraZeneca reported successful results from its Phase III DAPA-CKD clinical trial of Farxiga (dapagliflozin) in end stage kidney disease (ESKD). The trial demonstrated a statistically significant and clinically meaningful effect on a composite of worsening of renal function or risk of death, onset of end stage kidney disease (ESKD) or cardiovascular (CV) or renal death in adults with chronic kidney disease. The trial also hit all secondary endpoints in CKD patients with or without type 2 diabetes.
Aimmune Therapeutics published complete results from its pivotal Phase III European ARTEMIS trial of Palforzia for peanut allergy. Palforzia is an oral immunotherapy to mitigate allergic reactions, including anaphylaxis, that might occur with accidental exposure to peanut. It is a formulation of peanut allergen powder that is used to desensitize patients to peanuts who are otherwise allergic.
AbbVie’s Phase III ADVNCE trial of atogepant for migraines hit its primary endpoint of statistically significantly greater reduction in mean monthly migraine days. The drug is an oral calcitonin gene-related peptide (CGRP) receptor antagonist.
Boehringer Ingelheim and Eli Lilly and Company announced that the EMPEROR-Reduced Phase III trial in adults with heart failure with reduced ejection fraction, with and without diabetes, met the primary endpoint. Jardiance (empagliflozin) was superior to placebo in decreasing the risk of a composite of cardiovascular death or hospitalization from heart failure when added to standard of care.
Supernus Pharmaceuticals published the results of a pivotal Phase III trial of SPN-812 (viloxazine extended-release capsules) for ADHD. The trial hit its primary endpoint, with the drug significantly reducing ADHD symptoms in children.
Genocea Biosciences presented initial clinical data on the first five patients from Part B of the ongoing Phase I/IIa trial to explore the combination of the company’s neoantigen vaccine (GEN-009) and checkpoint inhibitor-based regimens in advanced solid tumors. The preliminary data show an acceleration of shrinkage beyond that of the CPI regimen.
AstraZeneca and Sanofi published data from their Phase IIb trial of nirsevimab, which demonstrated a significant decrease in medically attended lower respiratory tract infections, mainly bronchiolitis and pneumonia, and hospitalizations caused by respiratory syncytial virus (RSV) in healthy preterm infants. Nirsevimab is an extended half-life RSV monoclonal antibody.
Enterome SA initiated a clinical trial of EO2401 in combination with an immune checkpoint inhibitor for progressive or first recurrent glioblastoma multiforme (GBM). EO2401 is an off-the-shelf immuno-oncology candidate derived from the company’s OncoMimic platform. OncoMimics are microbiome-derived peptide antigens that mimic antigens expressed by tumor cells.
BioArctic and Eisai presented data from the open-label extension of the Phase IIb study of BAN2401 in early Alzheimer’s disease. The data showed a continuous decrease in amyloid levels in the brain measured at three, six and 12 months in the patients receiving the drug compared to those who had received placebo. BAN2401 is a humanized IgG1 version of the mouse monoclonal antibody mAb158, which binds to beta amyloid protofibrils.