Company Product Description Indication Status
Phase I
Aslan Pharmaceuticals Pte. Ltd., of Singapore ASLAN-004 Monoclonal antibody targeting IL-13 receptor alpha1 subunit Moderate to severe atopic dermatitis After at least 1 month of treatment at the lowest dose of 200 mg, 3 patients had reductions of 85%, 70% and 59% in their Eczema Area and Severity Index scores from baseline; data monitoring committee will review data in late December before opening second dosing cohort; study designed to test doses up to 600 mg followed by an expansion cohort of 12 patients on drug and 6 on placebo at the most efficacious dose
Biomx Inc., of Ness Ziona, Israel BX-001 Naturally occurring phage targeting Cutibacterium acnes Acne vulgaris Completed enrollment of 75 patients in study designed to measure the safety and tolerability of the drug; C. acnes levels will be measured as an exploratory endpoint; data expected in the first quarter of 2020
Inmune Bio Inc., of La Jolla, Calif. Xpro-1595 Targets soluble tumor necrosis factor Alzheimer's disease Treated first of up to 18 patients who express biomarkers of inflammation; 12-week study will measure safety and reduction of inflammation
Noxopharm Ltd., of Sydney Veyonda (NOX-66) Suppository of ecto-NOX disulfide-thiol exchanger type 2 inhibitor idronoxil Late-stage prostate cancer In 15 evaluable patients, Veyonda plus low-dose palliative radiotherapy produced a tumor response rate of 66%, including 1 partial response and 9 patients with stable disease; 5/16 patients had a >50% reduction in PSA; 10/16 patients had a >30% reduction in pain response; company plans to run a phase II/III study
Phase II
Akari Therapeutics plc, of London Nomacopan Complement C5 factor inhibitor; leukotriene BLT receptor antagonist Paroxysmal nocturnal hemoglobinuria In ongoing long-term study, all 6 participants from phase II who were transfusion dependent at entry are transfusion independent for minimum of 6 months with no reported drug-related serious adverse events
Concert Pharmaceuticals Inc., of Lexington, Mass. CTP-692  Deuterated form of D-serine, a co-agonist of the NMDA receptor Schizophrenia Started 300-patient study comparing 3 doses of the drug to placebo in patients who are stable on an antipsychotic medication; primary endpoint is the change in the Positive and Negative Syndrome Scale total score at 12 weeks compared to baseline; top-line data expected by the end of 2020
Knopp Biosciences LLC, of Pittsburgh Dexpramipexole  Reduces eosinophil levels Severe eosinophilic asthma In the first half of 2020, plans to start enrolling up to 100 patients with a history of exacerbation-prone asthma and elevated blood eosinophils in the 52-week study; primary endpoint is the annual rate of asthma exacerbations
Oragenics Inc., of Tampa, Fla. AG-013 (dapatifagene navolactibac) Trefoil factor-1 stimulator Oral mucositis Completed enrollment of 200 participants undergoing traditional chemoradiation to treat head and neck cancer to evaluate effectiveness of reducing OM; results expected early next year
Rafael Pharmaceuticals Inc., of Cranbury, N.J. CPI-613 (devimistat) Target the mitochondrial tricarboxylic acid cycle Relapsed or refractory Burkitt’s lymphoma/leukemia Massachusetts General Hospital added as a clinical study site
Sensorion SA, of Montpellier, France SENS-111 Histamine H4 receptor antagonist Acute unilateral vestibulopathy Study drug was safe and well-tolerated but missed primary endpoint of vertigo intensity; no additional development planned
Phase II
Akari Therapeutics plc, of London Nomacopan Complement C5 factor inhibitor; leukotriene BLT receptor antagonist Pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy Based on end-of-phase II meeting with FDA, single-arm responder-based pivotal study will be initiated, with treatment for up to 24 weeks; data from 7 participants in part A will be used to confirm dosing and endpoints for part B, which will begin recruiting following interim efficacy and safety readout from part A and meeting with FDA; primary endpoints are disease response defined by renal improvement and reduced transfusion dependence
Chugai Pharmaceutical Co. Ltd., of Tokyo Satralizumab IL-6 receptor antagonist Neuromyelitis optica spectrum disorders Findings from SakuraSky study, reported in May, were published online in The New England Journal of Medicine 
Eyegate Pharmaceuticals Inc., of Waltham, Mass. Ocular bandage gel (OBG; dexamethasone, iontophoresis delivery) Glucocorticoid receptor agonist Postoperative pain and inflammation Additional data from pivotal study in post-photorefractive keratectomy showed OBG prompted more rapid corneal wound closure than standard-of-care bandage contact lens (BCL); at day 2, average wound size for eyes treated with OBG was 3.61 mm2 vs. 6.66 mm2 for those treated with BCL
OSE Immunotherapeutics SA, of Nantes, France, and Haliodx SASU, of Marseille, France Tedopi (OSE-2101) Neoepitope combination Non-small-cell lung cancer In ongoing Atalante 1 trial, companies conducting translational investigation of immune biomarkers to define profile of responders
Pellepharm Inc., unit of Bridgebio Pharma Inc., of San Francisco Patidegib topical gel Hedgehog protein inhibitor Gorlin syndrome Fully enrolled pivotal trial
Phathom Pharmaceuticals Inc., of Buffalo Grove, Ill. Vonoprazan fumarate Potassium competitive acid blocker Erosive esophagitis Dosed first of expected 1,000 participants in pivotal Phalcon-EE trial assessing healing and maintenance of healing in EE and relief of heartburn
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. REGN-EB3 (atoltivimab + odesivimab + maftivimab) Viral structural protein inhibitor Ebola virus infection The New England Journal of Medicine published results, reported in August, from Palm trial in 681 patients during ongoing outbreak in Democratic Republic of the Congo showing triple-antibody cocktail outperformed Zmapp (Mapp Biopharmaceutical Inc., Defryus Inc.) control arm across multiple measures, including primary endpoint of mortality at day 28 (33.5% vs. 51.3%, p=0.002) and secondary endpoint of reduction in days until virus was undetectable in bloodstream

Notes

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

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