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Clinical Catch-Up: April 27 – May 1

There was a lot of clinical trial news, much of it related to COVID-19, including promising data for Gilead Sciences’ remdesivir to treat the disease. There were also quite a few non-COVID-19-related trial updates as the industry continues working despite the global pandemic, presenting abstracts and posters at virtual conferences, and analyzing data from trials.

COVID-19-Related

Gilead Sciences reported encouraging results from its clinical trial of remdesivir in COVID-19. The company announced topline results from the open-label, Phase III SIMPLE trial looking at 5-day and 10-day dosing of the drug in hospitalized patients with severe disease. It also reported positive data from the National Institute of Allergy and Infectious Diseases’ (NIAID) trial of the drug in COVID-19.

Regeneron and Sanofi’s rheumatoid arthritis drug Kevzara showed some promise in treating the most severe cases of COVID-19, but did not show benefit in less-advanced disease. As a result, the companies have halted the trial for that group of patients.

Mateon Therapeutics submitted an IND to the FDA for its OT-101 for COVID-19. OT-101 is a potent inhibitor of SAR-CoV-2 replication with efficacy and safety similar to Gilead’s remdesivir, but OT-101 targets both viral replication and the pneumonia and fibrosis. It is a first-in-class TGF-beta antisense therapeutic that has broad efficacy against solid tumors. Mateon also published several peer-reviewed articles related to its R&D efforts against COVID-19, including a special report in Annals of Pulmonary and Critical Care Medicine of its Phase II trial of OT-101 in COVID-19 patients with hypoxemic respiratory failure.

Northwell Health has begun testing famotidine, the active ingredient in OTC heartburn drug Pepcid, in COVID-19 patients. It is being given intravenously at nine times the heartburn dose. The drug seems to bind to a key enzyme in COVID-19.

PharmaMar began the APLICOV-PC clinical trial in COVID-19 with Aplidin (plitidepsin) with authorization from the Spanish Medicines and Healthcare Products Agency (AEMPS). It will be conducted in three hospitals in Madrid, Spain. Plitidepsin blocks the protein eEF1A, which is used by SARS-CoV-2 to reproduce and infect other cells.

Pfizer and Germany-based BioNTech are collaborating on a messenger RNA vaccine against SARS-CoV-2, the novel coronavirus that causes COVID-19. The two companies have already begun Phase I/II clinical testing in Germany. The companies now plan to start testing in the U.S. once the FDA gives approval.

Capricor Therapeutics announced new data showing 100% survival in critical COVID-19 patients treated with its allogeneic cardiac cell therapy CAP-1002 at Cedars-Sinai Medical Center in six compassionate care cases. As a result, the FDA approved its expanded access protocol to treat up to 20 more COVID-19 patients. CAP-1002 shows immunomodulatory properties.

CytoDyn provided updates on 49 COVID-19 patients who received leronlimab under the FDA’s eIND program. Eleven patients in a New York hospital with severe disease from acute respiratory failure showed positive results. They also detailed 23 patients in a southern California hospital, three in a Georgia Hospital, one in another New York hospital and one in a Northern California hospital. The results are typically positive.

Mesoblast Limited initiated enrollment of its Phase II/III clinical trial of remestemcel-L in 300 patients with COVID-19 ARDs. Remestemcel-L is being developed for inflammatory conditions, including acute graft versus host disease.

CalciMedica announced the addition of a study to investigate the use of CM4620-IE in patients with critical COVID-19 pneumonia on high-flow nasal cannula oxygen therapy. CM4620-IE is a potent and selective small molecule CRAC channel inhibitor that prevents CRAC channel overactivation, which can cause pulmonary endothelial damage and cytokine storm.

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Non-COVID-19-Related

Axsome Therapeutics announced that its AXS-05 met the primary endpoint in the ADVANCE-1 Phase II/III clinical trial for agitation in patients with Alzheimer’s disease.

AXS-05 is a dextromethorphan/bupropion modulated delivery tablet, an oral NMDA receptor antagonist with multimodal activity on other neurotransmitter systems, including sigma-1, serotonin, norepinephrine, and dopamine.

The primary endpoint was a statistically significant mean reduction in the Cohen Mansfield Agitation Inventory (CMAI) total score compared to placebo at Week 5. This is a 29-item caregiver-rated scale that evaluates the frequency of agitation-related behaviors. It includes behavior like pacing and restlessness, verbal aggression like screaming and shouting, and physical aggression like grabbing, pushing and hitting.

The patients taking AXS-05 had a mean decrease from baseline of 15.4 points compared to 11.5 points for placebo, which was 48% for AXS-05 compared to 38% for placebo. AXS-05 was also superior to bupropion on the CMAI total score, improving agitation symptoms.

Avadel Pharma announced positive topline data from its pivotal Phase III REST-ON clinical trial of FT218 for excessive daytime sleepiness and cataplexy in patients with narcolepsy. The trial, under a Special Protocol Assessment, met all three co-primary endpoints at all three doses, 9g, 7.5g, and 6g. The endpoints were Maintenance of Wakefulness Test (MWT), Clinical Global Impression-Improvement (CG-I) and mean weekly cataplexy attacks. Cataplexy is a sudden and uncontrollable muscle weakness or paralysis that is sometimes triggered by a strong emotion, such as laughter. FT218 is an investigational, once-nightly formulation of Micropump controlled-release sodium oxybate.

Aptose Biosciences presented preliminary clinical data on CG-806 at the AACR Virtual Annual Meeting 2020. They were preliminary reports from a Phase Ia/b trial in patients with relapsed or refractory chronic lymphocytic leukemia (CLL), small lymphocytic leukemia (SLL) or non-Hodgkin lymphoma (NHL) who have failed or been intolerant to two lines of established therapy. CG-806 is an oral, first-in-class FLT3/BTK cluster selective kinase inhibitor.

iTeos Therapeutics presented data from first-in-human study of its A2A receptor antagonist, EOS-850, in a Phase I trial. The data was presented in a poster at the AACR Virtual Annual Meeting 2020. There was a favorable tolerability trial. The drug is being developed for the treatment of solid tumors.

Orchard Therapeutics dosed the first patient in an open-label, proof-of-concept study of OTLL-201, a gene therapy for mucopolysaccharidosis type IIIA (MPS-IIIA). MPS-IIIA is also called Sanfilippo syndrome type A, which is a rare, inherited neurometabolic disorder that leads to the buildup of sugar molecules in the body. It results in progressive intellectual disability and loss of motor function. OTL-201 is an ex vivo autologous hematopoietic stem cell (HSC) gene therapy.

Harpoon Therapeutics dosed the first patient with HPN217 in a Phase I/II trial of relapsed, refractory multiple myeloma (RRMM). HPN217 is being developed under a global license and option agreement with AbbVie. HPN217 targets B-cell maturation antigen (BCMA).

Genentech, a Roche company, presented one-year data from FIREFISH Part 2, its pivotal trial of risdiplam in infants one to seven months old with symptomatic Type 1 spinal muscular atrophy (SMA). The trial met the primary endpoint with 29% of the babies sitting without support for five seconds by month 12 as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III).

Rafael Pharmaceuticals indicated MD Anderson Cancer Center will begin enrolling patients in its expanded Phase II trial of CPI-613 (devimistat) for relapsed or refractory Burkitt’s lymphoma/leukemia. The trial is led by Ariela Noy at Memorial Sloan Kettering Cancer Center, with enrollment also at City of Hope in Duarte, California and Massachusetts General Hospital in Boston. Devimistat is a first-in-class therapy that targets the mitochondrial tricarboxylic acid (TCA) cycle.

The University of Texas MD Anderson Cancer Center indicated that data from the Phase III EMBRACA trial found that the PARP inhibitor talazoparib did not show a statistically significant overall survival (OS) benefit in metastatic HER2-negative breast cancer and mutations in the BRCA1/2 genes. Talazoparib is marketed as Talzenna by Pfizer for adults with deleterious or suspected deleterious germline breast cancer susceptibility gene (BRCA)-mutated HER2-negative locally advanced or metastatic breast cancer.

Corbus Pharmaceuticals published results from its Phase II trial of lenabasum in 42 patients with diffuse cutaneous systemic sclerosis. Data showed improvements across the primary endpoint, the American College of Rheumatology Combined Response Index. Lenabasum is an oral, small molecule that selectively binds as an agonist to the cannabinoid receptor type 2 (CB2) to resolve inflammation, limit fibrosis and support tissue repair.

VIVEX Biologics published initial clinical trial data from its VAST trial evaluating VIA Disc for patients with one or two level symptomatic degenerative disc disease (DDD). VIA Disc is a non-surgical, injectable treatment for patients with chronic lower back pain resulting from DDD.

BioCardia accepted and implemented FDA-recommended modifications to the primary endpoint for its CardiAMP Cell Therapy Heart Failure Trial and associated statistical analysis plan. Going forward, the primary endpoint will be an outcomes composite score based on three-tiered Finkelstein-Schoenfeld (FS) hierarchical analysis. CardiAMP is a cell therapy, an autologous bone marrow-derived mononuclear cell formulation that stimulates the body’s natural healing response.

Flexion Therapeutics published data from two new analyses of patients treated with Zilretta (triamcinolone acetonide injectable suspension), including an analysis of 305 patients in the Phase III and Phase IIIb trials. The drug showed no negative impact on joint structure, while a separate analysis showed greater mobility and walking more steps per day after treatment.

TG Therapeutics published results from the multicenter Phase II trial of ublituximab in relapsing multiple sclerosis (RMS). The publication includes safety and efficacy data from 48 patients, which was generally well tolerated and had a 100% responder rate. Ublituximab is a novel glycoengineered monoclonal antibody that targets a specific and unique epitope on the CD20 antigen found on mature B-lymphocytes.

Oncocyte Corporation published two abstracts, one on new data on DetermaRx and the other describing the IRENE clinical study of DetermaDx. Both were accepted originally as oral presentations at the American Thoracic Society (ATS) 2020 International Conference. DetermaRx is a treatment stratification test that allows the identification of early-stage lung cancer patients at high risk for recurrence post-resection. DetermaDx is a liquid biopsy test to clarify if a patients’ lung nodules are benign.

Orion Biotechnology Canada successfully completed the participant follow-up in their Phase I trial of OB-002H. OB-002H is an investigational microbicide. The Phase I study was an open-label single-dose and multi-dose first-in-man trial to determine the safety, acceptability, and pharmacokinetic profile of the drug for HIV prevention.

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