Clinical Catch-Up: January 24 – January 28
Clinical trial news definitely picked up this final week of January. Here’s a look.
Todos Medical announced a data lock for the interim data analysis of its Tollovir Phase II trial in hospitalized COVID-19 patients. The results were on January 27, 2022. Tollovir is a 3CL protease inhibitor and anti-cytokine therapeutic candidate.
Arcturus Therapeutics updated data from clinical development programs for ARCT-154 and ARCT-165, investigational, next-generation, self-amplifying mRNA vaccine candidates. Full cohort data from the ARCT-154 arm of the ongoing Phase I/II booster trial showed a 30-fold increase in neutralizing antibody geometric mean concentrations against SARS-CoV-2 at Day 15 post-boost and maintained a 28-fold increase at Day 29. ARCT-165 and ARCT-154 also demonstrated robust neutralizing antibody responses post-boost against several variants of concern and variants of interest. Arcturus also said that, when administered in low doses at least five months after initial vaccination with Comirnaty, the Pfizer-BioNTech vaccine, the candidates provided robust increases of 54- and 47-fold, respectively, in neutralizing antibody responses against the Omicron variant.
A Phase IV study out of Brazil and the University of Oxford evaluated the Chinese-made Sinovac COVID-19 vaccine plus booster shots from either Sinovac, AstraZeneca-Oxford, Pfizer-BioNTech or Johnson & Johnson. The study found that patients who received Sinovac first then received a different booster had significantly higher immune responses to variants. Mixed vaccines induced antibodies at “8-22-fold higher” levels than when older adults were given another shot of the Sinovac vaccine. And giving the boosters from other vaccines increased immunity against both Omicron and Delta variants.
Moderna launched the Phase II trial of its Omicron-specific booster vaccine candidate, dosing the first patient. The study will focus on adults 18 years and older and have two cohorts, people who previously received the two-dose Moderna series at least six months ago and people who received the two-dose primary series plus a 50-microgram booster dose of the original vaccine at least three months ago. The company expects to enroll about 300 people in each cohort.
Galecto published full results of a trial of its inhaled galectin-3 inhibitor, GB0139, in hospitalized patients with COVID-19 who required oxygen but not mechanical ventilation. It was compared to patients who received only standard of care. The drug hit the primary endpoint of safety with no treatment-related adverse events. The mean age of patients was 65 years, with a mean BMI of 32, multiple comorbidities and breathlessness, but they were all able to effectively inhale the drug and hit pharmacologically relevant plasma levels. Galectin-3 plays a central role in COVID-19-related acute lung injury, cytokine storm, T-cell exhaustion and organ micro-thrombosis. The drug also appeared to rapidly decrease markers of inflammation associated with cytokine storm, micro-thrombosis and short or long-term fibrosis.
BerGenBio and Oslo University Hospital will initiate a study of BerGenBio’s bemcentinib in hospitalized COVID-19 patients. The EU-SolidAct trial — European DisCoVeRy for Solidarity: An Adaptive Pandemic and Emerging Infection Platform Trial — is part of EU-RESPONSE, a pan-European research project to identify drugs against COVID-19. Bemcentinib, an AXL inhibitor, will be evaluated in up to 500 hospitalized COVID-19 patients.
TFF Pharmaceuticals completed enrollment in its Phase I trial of a dry powder formulation of niclosamide for COVID-19. Niclosamide is a potential antiviral treatment.
TScan Therapeutics received the green light from the FDA to run a Phase I trial of TSC-100 for patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation (HCT). TSC-100 targets the minor histocompatibility antigen HA-1.
Applied Pharmaceutical Science got the go-ahead from the FDA to initiate a Phase I trial of APS03118 for multiple cancers. The drug is a next-generation selective RET inhibitor.
Tango Therapeutics’ IND for a Phase I/II trial of TNG908 for cancers with MTAP deletions, including non-small cell lung cancer, mesothelioma, pancreatic cancer and cholangiocarcinoma, was cleared by the FDA. TNG908 is a synthetic lethal small molecule inhibitor of protein arginine methyltransferase 5 (PRMT5).
SQZ Biotech expects to initiate its Phase I/II COMMANDER-001 trial of SQZ-eAPC-HPV in HPV16+ solid tumors after the FDA approved its IND. The therapy was developed by delivering five different mRNAs into a patient’s monocytes, B cells, T cells and NK cells.
Lyell received the go-ahead from the FDA to launch a Phase I trial of LYL132 in solid tumors expressing New York esophageal squamous cell carcinoma 1 (NY-ESO-1). LYL132 is a T-cell receptor (TCR) therapy that incorporates Epi-R, the company’s epigenetic reprogramming technology.
Genentech published one-year results from four pivotal Phase III trials of faricimab in wet or neovascular, age-related macular degeneration (AMD) and diabetic macular edema (DME). All four studies hit their primary endpoints, demonstrating that patients receiving faricimab up to every four months achieved non-inferior vision gains compared to aflibercept, given every two months. Faricimab is a bispecific antibody designed for the eye that targets and inhibits two pathways linked to several vision-threatening retinal conditions by neutralizing Ang-2 and VEGF-A.
Lyra Therapeutics initiated the Phase III ENLIGHTEN trial of LYR-210 in adult, surgically naïve chronic rhinosinusitis patients. LYR-210 utilizes the company’s proprietary XTreo platform to enable six months of local, intra-nasal, anti-inflammatory therapy for a single treatment.
Connect Biopharma announced data from the Phase IIb trial of CBP-20-1 administered subcutaneously to adults with moderate-to-severe atopic dermatitis. CBP-201 is an antibody designed to target interleuekin-4 receptor alpha.
MediWound announced positive topline results from its Phase II trial of EscharEx for debridement of venous leg ulcers. It hit the primary endpoint, showing that patients receiving the treatment had a statistically significant higher incidence of complete debridement compared to the gel vehicle.
Erytech Pharma presented results of two trials of eryaspase in combination with mFOLFIRINOX as first-line treatment for advanced pancreatic cancer. Erypase (GRASPA) consists of L-asparaginase encapsulated inside donor-derived RBCs and targets the cancer cells’ altered asparagine and glutamine metabolism.
Mustang Bio announced the FDA had placed a clinical hold on its IND for the planned Phase II study of MB-207 for X-linked severe combined immunodeficiency (XSCID), also known as “bubble boy syndrome.” The hold is in place pending CMC clearance. MB-207 is a gene therapy.
Mind Medicine reported the FDA had approved its IND for a Phase IIb trial of MM-120 for generalized anxiety disorder (GAD). It was previously on hold. MM-120 is a pharmacologically optimized form of LSD.
Imara expects to launch a Phase II trial of tovinontrine for heart failure with preserved ejection fraction (JFpEF). Tovinontrine is a highly selective and potent small molecule inhibitor of phosphodiesterase-9 (PDE9).
Nanoscope received IND clearance from the FDA for a Phase II trial of MCO-010 ambient-light activatable optogenetic monotherapy in Stargardt patients. Stargardt is an inherited retinal disease. The therapy, MCO-010, is a gene therapy that reprograms healthy retinal cells to make them photosensitive.
SwanBio Therapeutics received the green light from the FDA for a Phase I/II trial of SBT101 for disease-causing ABCD1 mutation in adrenomyeloneuropathy. SBT101 is an AAV-based gene therapy.
Cerevance dosed the first patient in its Phase I trial of CVN766. CVN766 is a potent and highly selective small molecule antagonist of the orexin 1 receptor (Ox1R), which is expressed in the areas of the brain important for regulating fear, anxiety, reward and emotion. The company is developing the drug for panic disorder and schizophrenia.
Checkpoint Therapeutics announced positive topline results from its Phase III trial of cosibelimab in metastatic cutaneous squamous cell carcinoma (cSCC). The drug is an anti-PD-L1 checkpoint inhibitor. The trial hit the primary endpoint, demonstrating a confirmed objective response rate (ORR) of 47.4%.
Sierra Oncology announced positive topline data from the Phase III MOMENTUM trial of momelotinib in myelofibrosis patients who are symptomatic and anemic and previously treated with an approved JAK inhibitor. The trial hit all primary and key secondary endpoints. Momelotinib is a potent, selective and orally bioavailable JAK1, JAK2 and ACVR1/ALK2 inhibitor.
Vaccinex reported positive interim response data in its Phase Ib trial of its pepinemab with Merck’s Keytruda (pembrolizumab). Pepinemab is a humanized IgG4 monoclonal antibody. In the study, two patients with recurrent or metastatic head and neck squamous cell carcinoma showed complete responses.
Gilead Sciences reported the FDA placed a partial clinical hold on studies assessing the combination of magrolimab plus azacytidine over unexpected serious adverse events. Magrolimab is a potential, first-in-class monoclonal antibody against CD47 and a macrophage checkpoint inhibitor. Azacitidine is a chemotherapy agent marketed under the brand names Vidaza and Onureg, both manufactured by Bristol Myers Squibb.
4D Pharma announced additional clinical data from Part A of its Phase I/II study of MRx-4DP0004 for asthma. The drug is an orally delivered single strain Live Biotherapeutic. The study hit the primary endpoint and the safety profile was comparable to placebo.
Biogen and its partner Eisai Company released additional details about the Phase IV post-marketing study of Aduhelm (aducanumab) for Alzheimer’s disease. As part of the Phase IV trial, the companies plan to enroll 18% of U.S. participants from Black/African American and Latinx populations. The primary endpoint of the study will be measured via the Clinical Dementia Rating-Sum of Boxes (CDR-SB) at 18 months after the beginning of treatment. This is a validated measure of both cognition and function broadly used in clinical studies of early symptomatic Alzheimer’s and is consistent with the two Phase III EMERGE and ENGAGE trials. They also plan to increase enrollment from 1,300, which they had previously announced, to 1,500 patients with Mild Cognitive Impairment due to Alzheimer’s and mild Alzheimer’s disease, with confirmation of amyloid beta pathology. They expect to initiate patient screening in May 2022.
GeNeuro announced the completion of its ProTEct-MS Phase II trial of temelimab in patients whose multiple sclerosis progresses without relapsed as they have previously received chronic anti-CD20-Ab therapy with rituximab. They expect to report final results by the end of March.
Cognito Therapeutics and Providence Health Plan announced plans to run a Phase III trial of its optogenetic, disease-modifying treatment for patients with mild-to-moderate Alzheimer’s disease.
Moderna launched a Phase I clinical trial at George Washington University organized by IAVI to evaluate an mRNA vaccine to deliver HIV immunogens to patients. Funded by the Bill & Melinda Gates Foundation, it will evaluate 56 healthy, HIV-negative adults.
Excision BioTherapeutics initiated a Phase I study of EBT-101 to remove sections of the HIV genome from the human body. EBT-101 is a CRISPR-based technology.
Taysha Gene Therapies reported a patient death in otherwise positive interim data from their gene therapy for Sandhoff and Tay-Sachs diseases. TSHA-101 delivers both the HEXA and HEXB genes that make up the beta-hexosaminidase A enzyme. The patient death appears likely related to an initial COVID-19 infection, which the patient recovered from, but then contracted a secondary hospital-acquired methicillin-resistant staphylococcus aureus (MRSA) infection and died. It does not appear to be associated with the therapy.