Company Product Description Indication Status
Phase I
Neurana Pharmaceuticals Inc., of San Diego Tolperisone Muscle relaxant Muscle spasms Data from driving study published in the Journal of Clinical Pharmacy and Therapeutics showed participants on Tolperisone experienced no impact on driving performance vs. placebo; in contrast, those who received cyclobenzaprine showed significant impairment vs. placebo (p < 0.01); self-reported sleepiness, self-reported motivation and self-appraised driving performance showed no significant effects for tolperisone vs. placebo, while those on cyclobenzaprine reported increased sleepiness (day 1), decreased motivation (days 1 and 2) and worse driving performance (days 1 and 2)
Ocular Therapeutix Inc., of Bedford, Mass. OTX-CSI Cyclosporine intracanalicular insert Dry eye disease Dosed first patients in open-label study to enroll 5 subjects, followed for about 4 months; study will evaluate safety, tolerability and assess biological activity by measuring signs and symptoms of DED over the time period
Rocket Pharmaceuticals Inc., of New York RP-L201 Gene therapy Severe leukocyte adhesion deficiency Phase I/II data showed additional results from first patient; analyses of peripheral vector copy number (VCN) and CD18-expressing neutrophils were performed 6 months post treatment and patient demonstrated peripheral blood VCN levels of 1.3 and CD18-expression of 47%, which is sustained from the 45% expression observed 3 months post treatment; pretreatment CD18 expression was <1%; patient continues to display visible improvement of skin lesions; no safety or tolerability issues related to RP-L201 identified to date
Rocket Pharmaceuticals Inc., of New York RP-L102 Gene therapy Fanconi anemia 7 of 9 patients evaluable in phase I/II trial, with first 4 showing robust and durable engraftment, continued hematologic correction and blood count stabilization; hemoglobin levels for 2 patients have increased to healthy, normal range; 2 additional patients who have been followed for a year or more after treatment display early evidence of engraftment, as measured by increases in peripheral blood VCNs
Phase II
Marker Therapeutics Inc., of Houston MultiTAA-specific T-cell therapy Multi-antigen-targeted T-cell therapy Acute myeloid leukemia Due to COVID-19, initiation of trial expected to be delayed; Marker paused opening enrollment of first 3 patients, as the manufacturing facility it utilizes to supply study drug remains closed during the pandemic
Minoryx Therapeutics SA, of Barcelona, Spain Leriglitazone (MIN-102) Brain-penetrant, oral PPARγ agonist X-linked adrenoleukodystrophy Started registration-enabling Nexus study in pediatric patients with early stage cerebral ALD
Phase III
Galera Therapeutics Inc., of Malvern, Pa. Avasopasem manganese (GC-4419) Small-molecule dismutase mimetic Severe oral mucositis Plans to increase size of Roman trial in patients with locally advanced head and neck cancer, in response to the delay to initiation of phase IIa study in Europe, to ensure sufficient number of patients for NDA safety database; updated guidance expects top-line data in second half of 2021
Gamida Cell Ltd., of Boston Omidubicel Cell therapy Bone marrow transplant Top-line results showed median time to neutrophil engraftment was 12 days for patients randomized to omidubicel vs. 22 days for comparator group (p<0.001); rolling BLA submission planned for fourth quarter of 2020
Genfit SA, of Lille, France Elafibranor Dual PPAR-alpha/delta agonist Nonalcoholic steatohepatitis Interim analysis showed trial did not meet predefined primary endpoint of NASH resolution without worsening of fibrosis in intent-to-treat population of 1,070 patients; response rate in 717 patients enrolled on study drug was 19.2% for patients who received elafibranor 120 mg vs. 14.7% for patients in placebo arm; on the fibrosis key secondary endpoint, 24.5% of patients who received elafibranor 120 mg achieved fibrosis improvement of at least 1 stage vs. 22.4% in placebo arm
Glenmark Pharmaceuticals Ltd., of Mumbai, India Favipiravir Antiviral tablet COVID-19 Initiated trials in India; study completion estimated by July/August 2020
Sanofi SA, of Paris Sarclisa (isatuximab) Monoclonal antibody that binds to specific epitope on the CD38 receptor Relapsed multiple myeloma Ikema trial showed adding Sarclisa to carfilzomib and dexamethasone met primary endpoint at first planned interim analysis, demonstrating significantly prolonged progression-free survival vs. standard-of-care carfilzomib and dexamethasone alone; no new safety signals identified 
Phase IV
Galderma SA, of Lausanne, Switzerland Dysport (abobotulinumtoxinA) for injection  Botulinum toxin Glabellar lines Results of Dream study showed 95% of patients treated with Dysport achieved high levels of satisfaction with 2 treatments per year, and 97% of patients thought the result of the treatment looked natural

Notes

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