Clinical Catch-Up: Pfizer’s Paxlovid, Immunome Set and Gilead’s Trodelvy

It was another busy week for clinical trial announcements and news. Here’s a look.

COVID-19-Related

Pfizer launched a Phase II/III trial of Paxlovid in non-hospitalized symptomatic pediatric patients at risk for progression to severe COVID-19. Paxlovid is an oral antiviral. The company also reported that the COVID-19 vaccine it developed with BioNTech is moderately effective against the Omicron variant in children ages 5-15, reducing the risk of infection by 31% in children 5-11 and 59% in children 12-15.

Innovation Pharma reported findings from its Brilacidin Phase II COVID-19 study data review and compassionate use of Brilacidin in critically-ill COVID-19 patients. The drug showed promising treatment effects in NEWS2 clinical improvement scores and in patients with the most elevated biomarker levels. Brilacidin is an antiviral drug.

Immunome announced the FDA had lifted the clinical hold on its Investigational New Drug (IND) application for IMM-BCP-01 for COVID-19. The FDA had placed a clinical hold on it due to a request for further data related to the preparation and administration of the antibody cocktail at clinical sites. It is a three-antibody cocktail targeting non-overlapping regions of the Spike protein of SARS-CoV-2.

Non-COVID-19-Related

Gilead Sciences reported its Phase III TROPiCS-02 trial of Trodelvy (sacituzumab govitecan-hziy) hit the primary endpoint in patients with HR+/HER2- metastatic breast cancer who had received previous endocrine therapy, CDK4/6 inhibitors and two to four lines of chemotherapy. It showed statistically significant improvement in progression-free survival (PFS) versus physician’s choice of chemotherapy. Trodelvy is a first-in-class antibody and topoisomerase inhibitor conjugate directed to the Trop-2 receptor.

Alpine Immune Sciences announced the FDA had placed a partial clinical trial on its NEON-2 study of davoceticept in combination with Merck’s Keytruda (pembrolizumab) in adults with choroidal melanoma, a cancer of the eye. The company reported one patient who had received a single dose of each drug had died, which is being attributed to cardiogenic shock.

Astellas Pharma announced results from the Phase III SKYLIGHT 4 trial of fezolinetant for hot flashes and night sweats associated with menopause. The drug is an oral, nonhormonal selective neurokinin-3 receptor agonist. The primary endpoint was an assessment of endometrial health, which the study hit.

OptiNose announced its Phase III ReOpen1 trial hit both its co-primary endpoints. OptiNose XHANCE (fluticasone propionate) Exhalation Delivery System is being developed for chronic sinusitis. The first endpoint was a composite score of nasal congestion and facial pressure sensation with nasal discharge; the second was ethmoid and maxillary sinus volume occupied by inflammation or disease, which was determined via CT scan.

Kintor Pharma completed patient enrollment in its Phase II study of KX-826 (pyrilutamide) in China for androgenetic alopecia in female patients. The drug is an androgen receptor antagonist and a potential first-in-class topical drug for androgenetic alopecia and acne vulgaris.

Evaxion Biotech completed recruitment for its Phase I/IIa study of EVX-02 in adjuvant melanoma patients. The company’s proprietary PIONEER AI technology identified a sufficient number of mutations to design a personalized treatment.

LAVA Therapeutics presented initial clinical data of LAVA-051. The data showed encouraging initial clinical safety, no dose-limiting toxicity or cytokine release syndrome. The drug is a Gammabody designed to activate Vgamma9 Vdelta 2 T cells and type 2 NKT cells to kill CD1d-expressing tumor cells.

Valneva announced positive Phase III results for its chikungunya vaccine candidate. The data showed 98.9% seroprotection one month after inoculation, with 96.3% six months after inoculation.

Eucure Biopharma, a subsidiary of Biocytogen, announced the first patient dosed for a Phase I study of YH002 (anti-OX40 monoclonal antibody) in combination with YH001 (anti-CTLA-4 monoclonal antibody) in Australia. The trial will run in Australia and China.

AIM ImmunoTech published positive data from a single-center named patient program treating advanced and metastatic pancreatic cancer patients. Ampligen (rintatolimod) is being evaluated as a monotherapy in an Early Access Program in the Netherlands. Ampligen is a TLR-3 agonist.

Ananda Scientific and NYU Grossman School of Medicine enrolled the first patient in a clinical trial of Nantheia A1002N5S for Opioid Sparing in patients with Radiculopathic Pain Syndromes. The drug is an investigational compound of cannabidiol in the company’s Liquid Structure delivery technology.

Akeso’s IND application for a Phase II trial of Cadonilimab in combination with docetaxel was approved by China’s regulators. The drug, a PD-1/CTLA-4 bispecific antibody is being tested in combination with docetaxel in advanced non-small cell lung cancer (NSCLC) that had progressed on combination therapy of a PD-1/L1 inhibitor and platinum-based doublet chemotherapy.

TC BioPharm announced positive interim data from its Phase Ia/IIb trial of TCB-002, OmnImmune for r/r acute myeloid leukemia (AML). The drug is an allogeneic unmodified gamma delta t-cell product.

CoapTech received an Investigational Device Exemption (IDE) approval from the FDA to initiate its PUMA-G Peds System study. The device is designed to provide a safer and more efficient way to place feeding tubes in children.

Apex Labs received a No Objection Letter (NOL) from Health Canada over its application for a Phase IIa trial of APEX-002-A01-01. This study will evaluate psilocybin in Veterans with post-traumatic stress disorder using the drug product APEX-002-A01. The drug is a low-dose synthetic psilocybin product in development for PTSD and treatment resistant depression.

AlzeCure Pharma announced new data from its ongoing clinical Phase I MAD trial of ACD856. The drug passes the blood-brain barrier and reaches the brain for the treatment of Alzheimer’s disease. The drug is designed to stimulate several important signaling systems in the brain with the idea that this will lead to improved cognition.

Sanofi and Swedish Orphan Biovitrum AB shared positive topline data from the ongoing Phase III trial of efanesoctocog alfa in severe hemophilia A. The drug hit the primary endpoint of clinically meaningful bleed prevention in people 12 years and older with severe hemophilia. It also hit the secondary endpoint, superiority to prophylactic factor VIII replacement therapy and significantly reduced the ABR in intra-patient comparisons.

Enzychem Lifesciences announced it had completed the final Clinical Study Report (CSR) for their Phase II trial of EC-18 in Chemoradiation-induced Oral Mucositis (CRIOM). The primary efficacy endpoint demonstrated the median duration of SOM from baseline through short-term follow-up period was 0.0 days in the EC-18 group versus 13.5 days in the placebo group. EC-18 acts as an immunomodulator, facilitating the resolution of inflammation and early return to homeostasis.

Denali Therapeutics initiated dosing in a Phase I/II trial of DNL593 for frontotemporal dementia (FTD) caused by mutations in the granulin gene (GRN). DNL593 is an investigational brain-penetrant, recombinant PGRN replacement therapy enabled by the company’s Protein Transport Vehicle.

Oncolytics Biotech announced Phase Ib data demonstrating clinical proof-of-concept for pelareorep-proteasome inhibitor combination therapy in multiple myeloma. The data showed the combination was well-tolerated and led to prolonged PFS of over three years in a subset of patients.

Freeline Therapeutics dosed the first patient in its Phase I/II B-LIEVE trial of FLT180a for hemophilia B. FLT180a uses an AAVS3 capsid to deliver the functioning gene of a variant of human factor IX to restore normal blood clot formation.

Biomind Labs received the go-ahead in Brazil for its second Phase II trial of N, N-dimethyltryptamine (DMT) for treatment-resistant depression. The first Phase II trial was for an intramuscular formulation of DMT for treatment-resistant depression. This is for an inhaled formulation.

Venatorx Pharmaceuticals announced that its Phase III trial of cefepime-taniborbactam for severe bacterial infections, specifically complicated urinary tract infections in adults, had positive outcomes. The drug is a fourth-generation cephalosporin against many gram-negative and gram-positive bacteria and taniborbactam, a beta-lactamase inhibitor.

ExCellThera dosed the first patient in its Phase I/II trial of ECT-001 for pediatric and young adults with high risk myeloid malignancies. ECT-001 is a cell therapy generated from the expansion of cord blood units using the UM171 small molecule and an optimized culture system.

AbbVie announced its Phase III PROGRESS trial of atogepant (Qulipta) for prevention of chronic migraine hit its primary endpoint of statistically significant reduction from baseline in mean monthly migraine days compared to placebo. The drug is an oral calcitonin gene-related peptide (CGRP) receptor antagonist.

Pipeline Therapeutics reported positive clinical data from its Phase I trial of PIPE-307 in healthy volunteers. The drug is an oral, highly selective antagonist of the muscarinic M1 receptor being developed for multiple sclerosis.

CoCrystal Pharma dosed the first patient in a Phase I trial of CC-42344, an oral antiviral, for pandemic and seasonal influenza A.

Confo Therapeutics dosed the first patients in the Phase I trial of CFTX-1554 for non-opioid treatment of neuropathic pain. The drug is a novel inhibitor of the angiotensin II type 2 receptor.

Emergent BioSolutions dosed the first participant in its Phase I trial of stabilized isoamyl nitrite (SIAN). The drug is being developed for known or suspected acute cyanide poisoning. It is being developed as a single-use intranasal spray intended for use by first responders and medical personnel after a cyanide incident.