Company Product Description Indication Status
Phase I
Ampio Pharmaceuticals Inc., of Englewood, Colo. Ampion (intranasal) Stem cell antigen-1 inhibitor COVID-19 infection Dosing initiated in second group following clearance by safety monitoring committee
Applied Genetic Technologies Corp. (AGTC), of Gainesville, Fla. rAAV2tYF-GRK1-RPGR X-linked retinitis pigmentosa GTPase regulator X-linked retinitis pigmentosa Based on data from all 28 participants across 6 dose groups in ongoing phase I/II program, at 12-month time point for 9 centrally dosed in groups 2 and 4, measurable improvements in visual sensitivity seen in 2/8 evaluable, with 8/9 showing stable or improving visual acuity; at 6-month time point for 11 centrally dosed in groups 5 and 6, measurable improvements in visual sensitivity seen in 5/11, with 9/11 showing stable or improving visual acuity
Deciphera Pharmaceuticals Inc., of Waltham, Mass. DCC-3014 CSF1R inhibitor Tenosynovial giant cell tumor Of 22 participants evaluable for efficacy at data cutoff of Oct. 5, 2020, with central assessment available for 21/22, objective response rate was 41% (9/22), including 1 complete response
Edgewise Therapeutics Inc., of Boulder, Colo. EDG-5506 Myosin 2 inhibitor Becker muscular dystrophy Single and multiple ascending-dose trial initiated in healthy adults and those with Becker MD
Lineage Cell Therapeutics Inc., of Carlsbad, Calif. Opregen Human embryonic stem cell-derived retinal pigmented epithelial cells Dry age-related macular degeneration Phase I/IIa study fully enrolled with 24 participants; updated interim results expected in Nov. 2020
Oncternal Therapeutics Inc., of San Diego TK-216 ETS transcription factor inhibitor Ewing sarcoma Interim data for 50 evaluable participants from ongoing trial, including 23 treated at recommended phase II dose (RP2D) at efficacy cutoff date of Oct. 16, 2020, showed 2/23 treated at RP2D (9%) achieved complete response (CR), including 1 surgical CR, and both with CRs remain on treatment with no evidence of disease; best objective response rate was 9%; 8 additional people treated at RP2D had stable disease; median progression-free survival for those treated at RP2D was 1.8 months
Reven Holdings Inc., of Golden, Colo. Rejuveinix  Intravenous formulation of pharmaceutical composition with antioxidant and anti-inflammatory ingredients COVID-19 infection As reported in Frontiers in Pharmacology, Section: Respiratory Pharmacology, no clinically significant or serious side effects seen in ascending dose-escalation study in 76 healthy volunteers
Shanghai Henlius Biotech Inc., of Shanghai HLX-14 (denosumab biosimilar) Anti-RANK-L monoclonal antibody Osteoporosis 2-part study initiated in healthy males to compare pharmacokinetics, pharmacodynamics, safety, tolerability and immunogenicity with reference drug Prolia (denosumab, Amgen Inc.)
Phase II
Applied Genetic Technologies Corp. (AGTC), of Gainesville, Fla. rAAV2tYF-GRK1-RPGR X-linked retinitis pigmentosa GTPase regulator X-linked retinitis pigmentosa Phase II/III Vista trial expected to include about 60 people randomized across low-dose group (1.2E+11 vg/mL, group 2 dose from ongoing phase I/II trial), high-dose group (1.1E+12 vg/mL, group 5 dose from ongoing trial) and untreated control group; primary endpoint is visual sensitivity, defined as having at least 7 decibel improvement in visual sensitivity in at least 5 prespecified loci at month 12; trial expected to open enrollment in first quarter of 2021 and to provide 6-month interim analysis results in third quarter of 2022
Blueberry Therapeutics Ltd., of Macclesfield, U.K. BB-2603 Nanoformulated, topical form of terbinafine Onychomycosis Study paused due to COVID-19 pandemic impact
Five Prime Therapeutics Inc., of South San Francisco Bemarituzumab  Monoclonal antibody targeting fibroblast growth factor receptor 2b (FGFR2b) FGFR2b+ and non-HER2+ front-line advanced gastric or gastroesophageal junction cancer In the Flight study, median progression-free survival was 9.5 months for bemarituzumab plus mFOLFOX6 chemotherapy, compared to 7.4 months for chemotherapy alone (p=0.073); median overall survival hasn't yet been reached for the combination compared to 12.9 months for chemotherapy (p=0.027); combination improved overall response rate by 13.1% (p=0.106)
Gyroscope Therapeutics Ltd., of London GT-005   Complement factor stimulator  Geographic atrophy secondary to dry age-related macular degeneration Started the 180-patient Horizon study comparing 2 dose levels to untreated patients; primary endpoint is progression of geographic atrophy over 48 weeks
Lumos Pharma Inc., of Austin, Texas LUM-201 Oral growth hormone Pediatric growth hormone deficiency Started the OraGrowtH210 phase IIb study testing 3 dose levels of the drug compared to standard-of-care injectable growth hormone therapy; primary efficacy endpoint is annualized growth height velocity over 6 months
Prilenia Therapeutics BV, of Naarden, the Netherlands Pridopidine  S1R agonist Levodopa-induced dyskinesia in Parkinson’s disease Study terminated due to COVID-19
Sagent Pharmaceuticals Inc., of Schaumburg, Ill. Camostat mesilate Serine protease inhibitor COVID-19 in high-risk outpatients Started the 300-patient Camelot study; primary endpoint is disease progression at day 28; secondary endpoints include survival rate, time to fever resolution, time to disease progression, and others
Phase III
Asieris Pharmaceuticals Co. Ltd., of Shanghai APL-1702 (Cevira) Hexaminolevulinate photodynamic drug-device therapy High-grade squamous intraepithelial lesions First patient treated in the study
Cynata Therapeutics Ltd., of Melbourne, Australia CYP-004 Cymerus mesenchymal stem cell Osteoarthritis of the knee Started the 440-patient Sculptor study comparing the treatment to placebo on clinical outcomes and knee joint structure over a 2-year period
Mesoblast Ltd., of New York Remestemcel-L Culture-expanded mesenchymal stem cells from donors Moderate to severe acute respiratory distress syndrome due to COVID-19 After review of data from first 135 patients, the independent data safety monitoring board recommended the study continue; final interim analysis after 180 of 300 expected patients have completed 30 days of follow-up
Pfizer Inc., of New York Abrocitinib Janus kinase 1 inhibitor Moderate to severe atopic dermatitis In the Jade Regimen withdraw study, 81.1% of patients who remained on the 200-mg dose and 57.4% of patients who switched to the 100-mg dose didn't experience a loss of response requiring rescue treatment compared 19.1% of those who were switched to placebo (p<0.0001 for both doses)
Russian Direct Investment Fund, of Moscow Sputnik V COVID-19 vaccine COVID-19 prophylaxis At the first interim look after 20 confirmed cases of COVID-19, the vaccine had an efficacy rate of 92%
Xbrane Biopharma AB, of Solna, Sweden Xlucane Ranibizumab biosimilar Wet age-related macular degeneration Enrolled all planned 580 participants in Xplore pivotal study; top-line data expected in mid-2021


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