Company Product Description Indication Status
Phase I
Apellis Pharmaceuticals Inc., of Waltham, Mass.,  APL-9  Targeted C3 therapy Severe COVID-19 Preliminary open-label safety data from 6 patients in a phase I/II study support advancement; results showed key markers of inflammation were within or near normal range at the end of the APL-9 treatment period
Castle Creek Biosciences Inc., of Exton, Pa. FCX-013 Gene therapy Moderate to severe localized scleroderma First adult patient dosed in phase I/II trial
Kalvista Pharmaceuticals Inc., of Cambridge, Mass. KVD-824  Oral plasma kallikrein inhibitor Healthy volunteers (eventually hereditary angioedema prophylaxis) Achieved targeted exposure levels expected to be required for prophylaxis; plans to file an IND for a phase II study in the first quarter of 2021
Oryzon Genomics SA, of Madrid, Spain Iadademstat (ORY-1001)  Selective KDM1A inhibitor Acute myeloid leukemia Phase I/IIa data published in the Journal of Clinical Oncology showed treatment well-tolerated, with signs of clinical and biological activity as single agent; complete remission with uncomplete hematological recovery in 1 patient was also reported
Regulus Therapeutics Inc., of La Jolla, Calif. RGLS-4326 Oligonucleotide designed to inhibit miR-17 Autosomal dominant polycystic kidney disease Initiated dosing in phase Ib trial; adaptive-design, open-label, multiple-dose study will involve up to 3 cohorts and test safety, pharmacokinetics and changes in levels of polycystin 1 and polycystin 2 levels
Phase II
Anavex Life Sciences Corp., of New York ANAVEX2-73 (blarcamesine) Sigma-1 receptor activator Parkinson’s disease dementia In the ANAVEX2-73-PDD-001 study, there were clinically meaningful, dose-dependent and statistically significant improvements in the Cognitive Drug Research computerized assessment system analysis; data to be presented at an upcoming medical meeting
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. Peginterferon Lambda Type III interferon Mild to moderate COVID-19 In the ILIAD study, patients treated with drug were 4.1-fold more likely to clear their viral load by day 7 (p=0.029); in patients with a baseline viral load > 6 log copies/mL, 79% of the 19 patients treated with drug and 38% of the 16 patients treated with placebo had a negative test for virus at day 7 (p=0.038)
GNT Pharma Inc., of Yongin, South Korea Nelonemdaz  NR2B-selective N-methyl d-aspartate receptor antagonist Acute ischemic stroke In the 209-patient Sonic study, 60.0%, 64.6% and 51% of patients taking 500-mg nelonemdaz, 750-mg nelonemdaz and placebo, respectively, had a score of 0-2 on the modified Rankin Scale on day 90
Ionis Pharmaceuticals Inc., of Carlsbad, Calif. IONIS-PKK-LRx Antisense targeting prekallikrein  Respiratory complications in COVID-19 Investigator-initiated study plans to enroll up to 110 patients at 25 hospitals in Brazil; primary endpoint is the number of days alive and free of oxygen support up to 15 days
Kalvista Pharmaceuticals Inc., of Cambridge, Mass. KVD-900 Plasma kallikrein inhibitor Hereditary angioedema Recruitment complete; data expected before the end of 2020
Lysogene SA, of Paris, and Sarepta Therapeutics Inc., of Cambridge, Mass. LYS-SAF302 Gene therapy  Mucopolysaccharidosis type IIIA A patient in the phase II/III AAVance study died; immediate cause of death is currently unknown, but there is no evidence that the death is linked to the treatment with LYS-SAF302
Vertex Pharmaceuticals Inc., of Boston VX-814  Corrects misfolding of AAT Alpha-1 antitrypsin deficiency Elevated liver enzymes (AST/ALT) were observed in several patients; company is discontinuing development of the drug
Vertex Pharmaceuticals Inc., of Boston VX-864 Corrects misfolding of AAT Alpha-1 antitrypsin deficiency Enrollment continues; data expected in first half of 2021
Phase III
Astex Pharmaceuticals Inc., of Pleasanton, Calif., a unit of Tokyo-based Otsuka Pharmaceutical Co. Ltd. Guadecitabine (SGI-110) DNA hypomethylating agent Previously treated acute myeloid leukemia and previously treated myelodysplastic syndromes or chronic myelomonocytic leukemia Drug didn't improve overall survival compared to physicians’ choice of alternative therapy in the Astral-2 and Astral-3 studies; full data to be presented at upcoming scientific meetings
Janssen Pharmaceutical Co., of Spring House, Pa., a unit of New Brunswick, N.J.-based Johnson & Johnson Tremfya (guselkumab) Monoclonal antibody targeting the p19 subunit of interleukin-23 Moderate to severe plaque psoriasis In the Voyage 1 study, at week 252, 84% of patients taking the drug achieved a Psoriasis Area Severity Index 90 and 82.4% achieved an Investigator's Global Assessment score of 0 or 1
Regenerx Biopharmaceuticals Inc., of Rockville, Md. RGN-259  Thymosin beta 4 ligand  Dry eye syndrome Enrollment in the 700-patient Arise-3 study is expected to be completed in November 2020; final collection of data for the primary and secondary endpoints will be in December 2020
Sage Therapeutics Inc., of Cambridge, Mass. Zuranolone  Neuroactive steroid GABAA receptor positive allosteric modulator Major depressive disorder  Interim results from the Shoreline study showed patients who had a 50% or greater decrease in the 17-item Hamilton Rating Scale for Depression took a mean of 1.9 treatments per year of zuranolone 30 mg


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

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