Company Product Description Indication Status
Phase I
Abeona Therapeutics Inc., of New York ABO-102 (rebisufligene etisparvovec) SGSH gene stimulator Sanfilippo syndrome type A (MPS IIIA) Updated phase I/II Transpher A data showed neurocognitive development was preserved within normal range of non-afflicted child for 2.5 to 3 years (latest time point measured) following treatment in 3 children in high-dose cohort 3 (3x1013 vg/kg)
Abeona Therapeutics Inc., of New York ABO-101 Alpha N acetylglucosaminidase stimulator Sanfilippo syndrome type B (MPS IIIB) Phase I/II Transpher B study showed treatment was associated with dose-dependent and sustained improvement in CNS and systemic biomarkers
Adagio Therapeutics Inc., of Waltham, Mass. ADG-20 COVID-19 spike glycoprotein modulator COVID-19 infection U.S. trial initiated in healthy adults; in addition to primary endpoints of safety and tolerability, secondary endpoints include pharmacokinetics and immunogenicity, with study exploring serum neutralizing activity ex vivo against SARS-CoV-2
Bluebird Bio Inc., of Cambridge Mass. BB-1111 (betibeglogene autotemcel) HBB gene stimulator Sickle cell disease Phase I/II and phase III studies temporarily suspended due to reported suspected unexpected serious adverse reaction of acute myeloid leukemia
Diffusion Pharmaceuticals Inc., of Charlottesville, Va. Trans sodium crocetinate Vitamin A analogue COVID-19 infection Safety monitoring committee confirmed that no dose-limiting toxicities or serious adverse events were seen in final (1.5-mg/kg dose) cohort of phase Ib trial in hospitalized people with confirmed hypoxemia, completing trial and meeting primary safety endpoint; secondary endpoint data expected in second quarter of 2021
Equillium Inc., of La Jolla, Calif. Itolizumab T-cell differentiation antigen CD6 inhibitor Graft-vs.-host disease Interim phase Ib/II Equate data showed dose-dependent reduction of CD6 expression on CD4+ and CD8+ T cells, consistent with proposed mechanism of action; in higher-dose cohorts (0.8 and 1.6 mg/kg), overall response rate was 100% (n=6) at day 29, including 5 complete responses and 1 very good partial response; reduction in baseline corticosteroid use at day 29 was about 40% to 80%
Gensight Biologics SA, of Paris Lumevoq (lenadogene nolparvovec) MT-ND4 gene stimulator Leber hereditary optic neuropathy  BioDrugs published phase I/IIa Reveal trial data that confirmed safety and determined dose subsequently tested in phase III Rescue and Reverse trials
Histogen Inc., of San Diego HST-001 Hair stimulating complex Androgenic alopecia At week 26 in phase Ib/IIa trial, men treated with study drug showed statistically significant change from baseline in total hairs (terminal and vellus) in target area
Interna Technologies BV, of Utrecht, the Netherlands INT-1B3 MicroRNA mimic Solid tumors First participant in first cohort dosed; dose-escalation portion will enroll about 30 people with advanced disease; dose-expansion part will enroll up to 50 people with hepatocellular carcinoma or triple-negative breast cancer, with top-line data from this part expected by year-end 2021
Kodiak Sciences Inc., of Palo Alto, Calif. KSI-301 Anti-VEGF antibody biopolymer conjugate Wet age-related macular degeneration; diabetic macular edema; retinal vein occlusion 1-year data from ongoing phase Ib study showed two-thirds of participants in each disease cohort achieved 6-month or longer treatment-free interval at 1 year, including 66% for wet AMD, 69% for DME and 66% for RVO; 78% of wet AMD, 84% of DME and 75% of RVO participants were on 4-month or longer interval at year 1
Metacrine Inc., San Diego MET-409 Farnesoid X receptor agonist Nonalcoholic steatohepatitis Phase Ib study results, published in the Journal of Hepatology, showed study drug, dosed at 50 mg, achieved about 38% mean relative liver fat reduction and was associated with 16% overall pruritus rate, with no discontinuations due to pruritus, and 7% LDL-cholesterol increase
Neuren Pharmaceuticals Ltd., of Melbourne, Australia NNZ-2591 Synthetic analogue of molecule derived from IGF-1 Phelan-McDermid syndrome; Angelman syndrome; Pitt Hopkins syndrome Twice daily oral dosing for 7 days was safe and well-tolerated in healthy volunteers
Noxxon Pharma NV, of Berlin NOX-A12 (olaptesed pegol) Stromal cell-derived factor 1 ligand inhibitor Brain cancer First participant dosed in third, high-dose cohort (600 mg/week) of phase I/II combination trial with external-beam radiotherapy
Realta Life Sciences Inc., of Norfolk, Va. RLS-0071 Complement C1s subcomponent inhibitor COVID-19-associated acute lung injury First healthy volunteer dosed
Recce Pharmaceuticals Ltd., of Sydney RECCE-327 Bactericidal permeability protein inhibitor; outer membrane protein inhibitor Chronic burn wounds Formalized agreement with Fiona Stanley Hospital to conduct phase I/II trial set to enroll up to 30 participants with gram-positive and gram-negative bacterial burn wound infections; dosing expected to begin in first quarter of 2021
Regenxbio Inc., of Rockville, Md. RGX-314  VEGF receptor antagonist Wet age-related macular degeneration Interim data from cohorts 4 (n=12) and 5 (n=12) of phase I/IIa trial showed stable visual acuity, with mean BCVA change of +1 letters and -1 letters from baseline, respectively, and decreased central retinal thickness, with mean change of -46 µm and -93 µm, respectively; long-term follow-up study (n=6) showed mean BCVA improvement of +12 letters from baseline at 3 years, with stable retinal anatomy compared to 2-year time point
Phase II
Clene Inc., of Salt Lake City CNM-ZnAg Clean surface nanotherapeutic COVID-19 infection Wholly owned subsidiary Clene Nanomedicine Inc. initiated study in Brazil expected to enroll about 276 acutely symptomatic, non-hospitalized people; primary endpoint is rate of decreased hospitalizations at day 28, with secondary endpoints assessing time to symptom resolution
Gyroscope Therapeutics Ltd., of London GT-005 Complement Factor I (CFI) gene stimulator Geographic atrophy Interim data from phase I/II Focus trial in people with disease secondary to age-related macular degeneration showed 9/10 treated had increases in CFI levels, averaging 146% compared to baseline (p=0.02); of 9 with increased CFI, 8 showed sustained increases at week 24 and beyond, including 1 sustained increase at 84 weeks; increases in CFI levels seen in those with and without rare gene variants
Lysogene SA, of Paris, and Sarepta Therapeutics Inc., of Cambridge, Mass. LYS-SAF302 Gene therapy  Mucopolysaccharidosis type IIIA In the AAVance study, concentration of heparan sulfate-derived oligosaccharides in the cerebrospinal fluid (CSF) was reduced by 27% and 30% at 6 months and 12 months, respectively (p<0.005 and p<0.05, respectively); GM2 ganglioside in CSF was reduced by 7% and 40% at 6 months and 12 months, respectively (p<0.005 at 12 months)
Monopar Therapeutics Inc., of Wilmette, Ill. Validive Mucobuccal tablet formulation of clonidine Chemoradiotherapy-induced severe oral mucositis in patients with oropharyngeal cancer Treated first patient in the phase IIb/III Voice study; primary endpoint is occurrence of severe oral mucositis
Neoimmunetech Inc., of Rockville, Md. NT-I7 (efineptakin alfa) Long-acting human interleukin-7 Previously treated advanced or metastatic gastric, gastroesophageal junction or esophageal adenocarcinoma First patient treated in the phase II study comparing NT-17 plus Opdivo (nivolumab) to Opdivo alone; primary endpoints are objective response rate and overall survival
Sagent Pharmaceuticals Inc., of Schaumburg, Ill. Camostat mesilate Serine protease inhibitor COVID-19 outpatient Drug is being added to the phase II/III ACTIV-2 adaptive platform treatment trial run by the NIH and the AIDS Clinical Trials Group
Synairgen plc, of Southampton, U.K. SNG-001 Inhaled interferon-beta formulation Mild to moderate COVID-19 symptoms not yet requiring hospitalization First patient treated with SNG-001 in the phase II/III ACTIV-2 study
Phase III
Polypid Ltd., of Petah, Tikva, Israel D-PLEX100 Prolonged-release broad-spectrum antibiotic doxycycline  Post-abdominal surgery incisional infections Enrolled 100th patient in the Shield I study; adaptive study is expected to enroll between 616 and 900 patients; top-line data expected by the end of 2021

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