Yet another busy week for clinical trial news. Here’s a look.
Yet another busy week for clinical trial news. Here’s a look.
COVID-19-Related
CytoDyn dosed the first patient in its Phase III trial in Brazil for critically ill COVID-19 patients. The study is evaluating leronlimab, a CCR5 antagonist.
Axcella Therapeutics announced it is initiating a new clinical program to study AXA1125 in patients with Long Covid. Long Covid is also called Post COVID-19 Condition and Post-Acute Sequelae of COVID-19 (PASC). The trial will be conducted in the UK. AXA1125 is a composite of six amino acids and derivatives that targets multiple metabolic pathways.
Ocugen has submitted an Investigational New Drug (IND) to the U.S. Food and Drug Administration (FDA) to run a Phase III trial of India’s BBV152 (Covaxin), a vaccine against COVID-19. The vaccine, Covaxin, is a whole-virion inactivated COVID-19 product. The vaccine was jointly developed by India’s Bharat Biotech and the Indian Council of Medical Research (ICMR). Ocugen has a deal with Bharat to develop the vaccine in the U.S. and Canada. It has been distributed under emergency use authorizations in 17 countries.
Relief Therapeutics announced its wholly-owned subsidiary APR Applied Pharma Research (APR) reported positive interim results from its trial of nasal spray Sentinox in COVID-19. It also confirmed its safety and tolerability. Sentinox is an acid-oxidizing solution containing hypochlorous acid at 0.005% and is certified in Europe as a Class III Medical Device.
TFF Pharmaceuticals received approval from Health Canada to launch a Phase I trial of a dry powder formulation of niclosamide. The drug is being tested as an antiviral treatment for COVID-19.
ARCA biopharma announced the Data and Safety Monitoring Committee (DSMC) completed a pre-specified interim analysis of its ASPEN-COVID-19 Phase IIb study of rNAPc2 in hospitalized to severe COVID-19. The DSMC recommended completion o the study with no modifications. rNAPc2 is a small recombinant protein, a potent, selective inhibitor of tissue factor (TF).
Non-COVID-19-Related
Novartis announced the CANOPY-1 Phase III trial of canakinumab failed to meet its primary endpoints when combined with Merck’s checkpoint inhibitor Keytruda (pembrolizumab) plus platinum-based doublet chemotherapy in non-small cell lung cancer (NSCLC). Specifically, the trial did not meet the primary endpoints of overall survival (OS) and progression-free survival (PFS) in patients receiving the combination therapy compared to the placebo cohort that received Keytruda plus platinum-based doublet chemotherapy in patients with previously untreated locally advanced or metastatic NSCLC.
The study failed to meet statistical significance for those endpoints, but the company did say it showed potentially clinically meaningful improvements in both PFS and OS in pre-specified subgroups based on the baseline inflammatory biomarker, he-CRP, as well as other biomarker-defined subgroups.
Aquestive Therapeutics announced positive topline data from its Phase I PK study of AQST-109 sublingual film for delivery of epinephrine in emergency treatment of allergic reactions including anaphylaxis. The safety, tolerability, PK and pharmacodynamics all fell within the target range of standard of care autoinjectors.
Horizon Therapeutics announced positive topline results showing its Krystexxa with methotrexate had a significant increase in efficacy compared to the response rate of Krystexxa with placebo in people with chronic gout refractory to conventional therapies. The MIRROR study demonstrated that 71% of patients who received Krystexxa with methotrexate compared to 40% of the Krystexxa-placebo group hit the primary endpoint.
Applied Therapeutics reported biomarker data from a pilot trial of AT-007 in eight patients with Sorbitol Dehydrogenase Deficiency (SORD Deficiency). AT-007 is a novel CNS penetrant Aldose Reductase Inhibitor (ARI) for CNS rare metabolic diseases, including Galactosemia, SORD Deficiency and PMM2-CDG.
Finch Therapeutics presented clinical data supporting CP101 for prevention of recurrent C. difficile (C. diff) infection. The data was from the Phase II PRISM3 trial. CP101 delivers a complete microbial community in a one-time oral administration, without the need for bowel preparation.
TCR2 Therapeutics announced a clinical trial collaboration with Bristol Myers Squibb to study gavo-cel in combination with Opdivo (nivolumab) and Yervoy (ipilimumab) in a Phase II study of refractory mesothelin-expressing solid tumors. This includes non-small cell lung cancer (NSCLC), ovarian cancer, malignant pleural/peritoneal mesothelioma (MPM) and cholangiocarcinoma.
Evaxion Biotech announced a clinical trial collaboration with Merck & Co. to evaluate Evaxion’s EVX-01 with Merck’s Keytruda (pembrolizumab) in a Phase IIb trial in melanoma. EVX-01 is a novel personalized cancer neoepitope immunotherapy.
Phathom Pharmaceuticals presented data from the PHALCON-HP Phase III trial of vanoprazon in H. pylori infections. The study showed that both vonoprazan in combination with amoxicillin and clarithromycin and vonoprazan in combination with amoxicillin showed superior eradication rates in all patient s compared to standard of care therapy consisting of lansoprazole, in combination with amoxicillin and clarithromycin.
Turning Point Therapeutics presented early clinical data from the ongoing Phase I/II CARE trial in pediatric and young adults with advanced solid tumors harboring ALK, ROS1 or NTRK alterations. Repotrectinib demonstrated encouraging clinical activity. The drug is a next-generation kinase inhibitor targeting the ROS1 and TRK oncogenic drivers of NSCLC and advanced solid tumors.
Privo Technologies announced positive results from the Phase I/II trial of PRV111 in early-stage head and neck squamous cell carcinoma (HNSCC). PRV11 is a transmucosal delivery system that can deliver cisplatin topically and directly to the tumor site.
Ultimovacs announced a new Phase II trial of its UV1 universal cancer vaccine in combination with Merck’s Keytruda (pembrolizumab) in non-small cell lung cancer (NSCLC). They expect the first patient to be treated in the first half of 2022.
Krystal Biotech announced the last participant in the Phase III GEM-3 trial had completed the 26-week dosing period and 30-day safety follow-up visit. The study is evaluating beremagene geperpavec (B-VEC) as a topical gene therapy for dystrophic epidermolysis bullosa (DEB).
BioVie announced the FDA had authorized its Phase II trial of NE3107 in Parkinson’s disease. NE3107 inhibits inflammatory activation of ERK and NFB that leads to neuroinflammation and insulin resistance, but not their homeostatic functions, such as insulin signaling and neuron growth and survival.
Recce Pharmaceuticals received approval from the Human Research Ethics Committee (HREC) to start its Phase I trial of RECCE 327 in Australia. R327 is a new class of broad-spectrum anti-infectives being evaluated for treatment and prevention of sepsis.
Cybrexa Therapeutics announced favorable early Phase I data for CBX-12 (alphalex-exatecan). The drug is being developed for advanced or metastatic solid tumors. The company’s alphalex technology platform allows antigen-independent targeting of tumors and intracellular delivery of potent anticancer drugs.
EsoCap dosed the first patient in the Phase II ACESO trial of ESO-101 for active eosinophilic esophagitis (EoE). The EsoCap technology is made up of a capsule containing a thin mucoadhesive film loaded with an active pharmaceutical ingredient, in this case, the anti-inflammatory corticosteroid, mometasone furoate.
4D Molecular Therapeutics announced interim clinical data from the Phase I/II trial of 4D-310 in patients with Fabry disease. 4D-310 is a targeted and evolved C102 vector-based product candidate.
AnHeart Therapeutics dosed the first patient in the Phase II trial of taletrectinib in ROS1 fusion-positive lung cancer. The drug is a next-generation tyrosine kinase inhibitor (TKI) designed to target ROS1 and NTRK fusion mutations.
Bio-Path received FDA clearance to initiate a Phase I/Ib study of prexigebersen-A (liposomal Grb2-A or BP1001-A) in patients with solid tumors, including ovarian, endometrial, pancreatic and triple negative breast cancer. The drug is a modified drug product with the same drug substance prexigebersen but includes formulation enhancements to produce smaller drug nanoparticles.
Context Therapeutics and Wisconsin Oncology Network (WON) dosed the first patient in the Phase II trial of Onapristone in Combination with Fulvestrant for Patients with ER-positive and HER2-negative Metastatic Breast Cancer after Progression on Endocrine therapy and CDK 4/6 Inhibitors (The SMILE Study). Onapristone is a progesterone receptor antagonist.
InflaRx announced positive data from the third cohort in its Phase IIa open-label study of vilobelimab in Pyoderma Gangraenosum. Vilobelimab is a first-in-class monoclonal anti-human complement factor C5a antibody.
Adagene, the National University Cancer Institute, Singapore at the National University Hospital in Singapore, National Cancer Centre Singapore, and the Singapore Translational Cancer Consortium initiated a Phase Ib/II trial of ADG106 in combination with Bristol Myers Squibb’s checkpoint inhibitor Opdivo (nivolumab). ADG106 is an anti-CD137 agonist antibody. It is being developed for advanced solid tumors and non-Hodgkin’s lymphoma. This study will look at NSCLC patients who have progressed on previous therapies.
Nurix Therapeutics announced initial data demonstrating clinically meaningful degradation of Bruton’s tyrosine kinase (BTK) in patients with r/r B-cell malignancies, including CLL patients with significant mutations in the BTK gene associated with resistance to standard of care BTK inhibitors. The data is based on NX-2127, an oral degrader of BTK with immunomodulatory drug activity.
SpringWorks Therapeutics provided an update on its ongoing clinical collaboration with GlaxoSmithKline in its Phase II trial of nirogacestat, SpringWorks’ gamma-secretase inhibitor, in combination with GSK’s Blenrep (belantamab mafodotin-blmf) in r/r multiple myeloma. It is being tested in combination as a sub-study of GSK’s ongoing DREAMM-5 platform trial. The first combination dose level has been expanded based on encouraging preliminary data. The expanded Phase II cohort is continuing to explore the safety and efficacy profile compared to the higher dose monotherapy control arm.
Gilead Sciences and Merck initiated a Phase II trial of islatravir and lenacapavir in HIV patients who are virologically suppressed on antiretroviral therapy. The combination is a once-weekly oral regimen.
Vifor Pharma and Angion Biomedica reported the Phase III trial of ANG-3777 did not show a statistically significant difference from placebo on the primary endpoint, eGFR at 12 months, in the population of deceased donor kidney transplant patients at risk for developing DGF. This data and CSA-AKI data expected later in the year will inform their clinical strategy for the drug.
AbbVie’s ABBV-951 hit the primary endpoint in a 12-week Phase III trial in advanced Parkinson’s disease. The patients who received daily doses of the drug demonstrated statistically significant increases in “On” time without dyskinesia. ABBV-951 was compared to oral levodopa/carbidopa.
Revolo Biotherapeutics activated clinical trial sites and opened enrollment in a Phase II study of ‘1104 in patients with eosinophilic esophagitis (EoE). ‘1104 is a first-in-class peptide for allergic diseases.
Centogene enrolled the first patient in the EFRONT Study, an observational study to understand the prevalence of genetic mutations in patients with frontotemporal dementia (FTD). The trial plans to enroll and complete data-rich genetic testing for more than 3,000 FTD patients at centers in Belgium, Germany, Greece, Italy, Portugal, Spain and Turkey.
Synthekine is advancing STK-012 into a Phase Ia/Ib trial. The drug is an IL-2 partial agonist in development as a monotherapy and in combination with checkpoint inhibitors in advanced solid tumors.
Inari Medical announced positive acute and long-term interim data from the first 500 pulmonary embolism patients in the FlowTriever Outcomes Registry. At 48 hours after the procedure, the major adverse event rate and mortality rate were 1.4% and 0.2%, respectively. The FlowTriever system is 510(k)-cleared by FDA and CE marked for nonsurgical removal of clot from peripheral blood vessels.
Rafael Pharmaceuticals announced the AVENGER 500 Phase III trial of CPI-613 (devimistat) in combination with modified FOLFIRINOX as a first-line therapy in metastatic adenocarcinoma of the pancreas failed to hit the primary endpoint of overall survival. CPI-613 is a first-in-class compound that targets enzymes involved in cancer cell energy metabolism located in the mitochondria of cancer cells.
Candel Therapeutics presented data on patient-reported tolerability assessment of intraprostatic injections from the ongoing Phase III trial of CAN-2409 in localized prostate cancer. CAN-2409 is a replication-deficient adenovirus that delivers the herpes simplex virus thymidine kinase (HSV-tk) gene to cancer cells.
HCW Biologics got the greenlight to launch a Phase Ib trial of HCW9218 in advanced pancreatic cancer. HCW9218 is an injectable, fusion protein complex that drives bifunctional, anti-tumor activity by activating desired immune responses to attack cancer cells while blocking unwanted immunosuppressive activities.
OptiNose completed recruitment in the second of two Phase IIIb trials of XHANCE (fluticasone propionate) nasal spray for chronic sinusitis. EXHANCE is a drug-device combination that uses an Optinose Exhalation Delivery System to deliver a topical anti-inflammatory.
Provectus Biopharmaceuticals presented updated data from its Phase Ib trial of PV-10 (rose Bengal disodium) in combination with Merck’s Keytruda (pembrolizumab) for advanced cutaneous melanoma in patients refractory to immune checkpoint blockade. PV-10 induces tumor-specific reactivity in circulating T cells.
Hutchmed initiated a Phase III trial in China of HMPL-523 in adults with primary immune thrombocytopenia. HMPL-523 is a novel, investigational spleen tyrosine kinase inhibitor.
Corvus Pharmaceuticals’ partner Angel Pharmaceuticals received approval in China to initiate a Phase I/Ib trial of Corvus’ ITK inhibitor CPI-818 for r/r T-cell lymphomas. Corvus cofounded Angel Pharma to develop its pipeline in China.
Alnylam reported 18-month data from its Phase III HELIOS-A study of vutrisiran in polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis. The study hit all secondary endpoints at 18 months. It demonstrated statistically significant improvements in progression of neuropathy and improved quality of life, gait speed and overall disability compared to placebo.
Gilead Sciences announced interim data from the ongoing BICSTaR study of Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide) in HIV. It also collected patient-reported outcomes in routine clinical practice. The data demonstrated a consistent efficacy profile in a real-world setting.
Talaris Therapeutics initiated the Phase II FREEDOM-2 trial of FCR001 in delayed tolerance induction. The trial will evaluate FCR001, an allogeneic cell therapy, and its ability to induce immune tolerance to a transplanted kidney in patients who received the transplant from a living donor up to a year before receiving the drug.
