Company Product Description Indication Status
Phase I
Acer Therapeutics Inc., of Newton, Mass. ACER-001 (repurposed sodium phenylbutyrate) BC ketoacid dehydrogenase kinase inhibitor Urea cycle disorders Final data from part B of pivotal trial in 36 healthy adults showed bioavailability and bioequivalence to Buphenyl (sodium phenylbutyrate)
Herantis Pharma plc, of Helsinki, Finland CDNF Neuroprotective and neurorestorative factor Parkinson's disease Top-line analysis confirms positive safety and tolerability 
Ico Therapeutics Inc., of Vancouver, British Columbia Oral amphotericin B Antifungal  Vulvovaginal candidiasis All repeat doses were well-tolerated, with no serious adverse events
Reneuron Group plc, of London Human retinal progenitor cell stem cell therapy Human retinal progenitor cell stem cell therapy Retinitis pigmentosa Long-term efficacy data from the study continue to show a meaningful clinical effect from the therapy at all time points out to 12 months post-treatment
Phase II
IMV Inc., of Dartmouth, Nova Scotia DPX-Survivac HLA-restricted survivin peptides  Advanced recurrent ovarian cancer 79% of evaluable patients achieved disease control on target lesions; 53% experienced tumor regressions; 37% achieved durable clinical benefit lasting ≥ 6 months; including 4 partial regressions so far, overall response rate not yet reached as 6 patients remain on treatment; treatment well-tolerated with limited side effects observed
IO Biotech ApS, of Copenhagen IO-102 Cancer therapeutic vaccine Non-small-cell lung cancer Safety monitoring committee has recommended to continue the trial without modifications as the futility boundary was successfully passed
Theralase Technologies Inc., of Toronto TLD-1433 Light-activated photodynamic compound Non-muscle invasive bladder cancer Nova Scotia Health Authority Research Ethics Board approved the commencement of the study
Phase III
Allecra Therapeutics GmbH, of Saint-Louis, France Exblifep (cefepime-enmetazobactam) Combination of extended-spectrum beta-lactamase inhibitor and fourth-generation cephalosporin Complicated urinary tract infections, including acute pyelonephritis Allium trial met FDA and EMA prespecified primary endpoints and demonstrated superiority over piperacillin-tazobactam; overall success was 79.1% for treatment vs. 58.9% for piperacillin-tazobactam
Marinus Pharmaceuticals Inc., of Radnor, Pa. Ganaxolone Positive allosteric modulator of GABAA receptors CDKL5 deficiency disorder Reached 100-patient enrollment target for pivotal Marigold study in children and young adults; top-line results expected in third quarter of 2020


For more information about individual companies and/or products, see Cortellis.

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