Company Product Description Indication Status
Phase I
4D Molecular Therapeutics Inc., of Emeryville, Calif. 4D-110 AAV-based gene therapy designed to deliver functional copy of CHM gene Choroideremia Dosed first patient testing candidate delivered by single intravitreal injection
Daré Bioscience Inc., of San Diego DARE-HRT1 Delivers bio-identical 17β-estradiol and progesterone continuously over 28-day period Vasomotor symptoms and menopause Started trial testing potential option for hormone therapy to treat vasomotor symptoms and prevent bone loss and fracture associated with menopause
Opthea Ltd., of Melbourne, Australia OPT-302 VEGF-C/VEGF-D ligand inhibitor Diabetic macular edema  Updated data from phase Ib/IIa trial in subset who received at least 3 consecutive aflibercept injections on regular basis before enrollment (n=35) showed mean improvement in BCVA of +6.6 letters (n=22) from baseline to week 12 in study drug + aflibercept combination vs. +3.4 letters (n=13) for those continuing on aflibercept monotherapy; proportion of those gaining ≥10 letters from baseline to week 12 was 27.3% for combination vs. 0% for aflibercept monotherapy and proportion gaining ≥15 letters to week 12 was 9.1% for combination vs. 0% for monotherapy; improved anatomical changes were consistent with functional visual acuity outcomes
Promab Biotechnology Inc., of Richmond, Calif. CAR-T/NK cells CD19-targeted CAR T cells Relapsed/refractory CD19-positive B-cell malignant hematological tumors Completed investigator-initiated trial showing, among 10 cases, 7 patients were in continuous remission, with longest continuous remission of 9 months; cytokine release syndrome side effects were significantly reduced
TCR2 Therapeutics Inc., of Cambridge, Mass. TC-210 Mesothelin modulator Solid tumors Interim data showed first 5 participants treated in phase I portion of phase I/II trial in mesothelin-expressing disease had tumor regression, including 2 RECIST unconfirmed partial responses and 2 with stable disease through 6 months
Phase II
Aerie Pharmaceuticals Inc., of Durham, N.C. AR-1105 (dexamethasone intravitreal implant) Glucocorticoid receptor agonist Macular edema Sustained effects with both clinical formulations (CF-1 and CF-2) were shown by increases in BCVA and reductions in macular edema; peak efficacy was observed earlier with CF-1 while CF-2 showed longer duration of effect of up to 6 months
AI Therapeutics Inc., of Guilford, Conn. LAM-002A (apilimod dimesylate) PIKfyve kinase inhibitor COVID-19 infection Trial initiated and expected to enroll 142 outpatients; primary efficacy endpoint is reduction of viral load in those with confirmed infection; additional efficacy measures include death, hospitalization and oxygen saturation
Destiny Pharma plc, of Brighton, U.K. XF-73 (exeporfinium chloride nasal gel) Porphyrin-based compound Staphylococcus aureus infection FDA agreed with protocol amendment to reduce phase IIb study size due to impact of COVID-19 pandemic on enrollment; study two-thirds enrolled, with recruitment now expected to complete by year-end 2020; planned interim safety review expected to report in August 2020
Immunic Inc., of New York IMU-838 DHODH inhibitor COVID-19 infection First of about 120 hospitalized participants enrolled in investigator-sponsored phase IIb Ionic combination trial with oseltamivir (Tamiflu, Roche Holding AG) in adults with moderate to severe infection
Jcyte Inc., of Newport Beach, Calif. Jcell Human retinal progenitor cells Retinitis pigmentosa Phase IIb study randomized 84 participants, with 74 meeting criteria for protocol analysis; mean changes in BCVA from baseline to month 12 were +2.81, +2.96, and +7.43 letters in sham (n=26), 3x106 hRPC (n=25) and 6x106 hRPC (n=23) treatment arms, respectively; in post hoc exploratory analysis of target subgroup (n=37), mean changes in BCVA from baseline to month 12 were +1.85, -0.15, and +16.27 letters in sham (n=13), 3x106 hRPC (n=13) and 6x106 hRPC (n=11; p=0.003) treatment arms, respectively
NGM Biopharmaceuticals Inc., of South San Francisco NGM-621 Complement C3 inhibitor Geographic atrophy Catalina study initiated and expected to enroll 240 individuals with GA secondary to age-related macular degeneration in 1 or both eyes; primary efficacy endpoint is change from baseline in square root of GA lesion area at 48 weeks vs. sham
Puma Biotechnology Inc., of Los Angeles Nerlynx (neratinib) EGFR antagonist; Erbb2/Erbb4 tyrosine kinase receptor inhibitor Cervical cancer Data from Summit trial in HER2-mutant, metastatic disease, originally reported in March 1019, published online in Gynecologic Oncology
Rezolute Inc., of Redwood City, Calif. RZ-358 Insulin receptor antagonist Congenital hyperinsulinism Enrollment in phase IIb Rise study, paused due to COVID-19 pandemic, expected to resume by end of third quarter of 2020; study expected to complete in second half of 2021
Phase III
Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl) Apolipoprotein B antagonist; phospholipase A2 inhibitor Hypertriglyceridemia Revascularization analysis from Reduce-It trial showed first and total event reductions of 34% (p<0.0001)and 36% (p<0.0001), respectively, vs. placebo; early coronary revascularization benefit signal shown in prespecified exploratory analysis, with sustained statistical significance attained by 11 months; times to first revascularization events also were reduced vs. placebo across subtypes of intervention, including elective, urgent and emergent
Dalcor Pharmaceuticals Inc., of Montreal Dalcetrapib  Cholesterol ester transfer protein inhibitor Acute coronary syndrome Independent data safety monitoring board recommended that pivotal Dal-GenE trial in cardiovascular patients with ADCY9 AA genotype continue as planned with no modifications; results expected in first half of 2021
Eyepoint Pharmaceuticals Inc., of Watertown, Mass. Yutiq (fluocinolone acetonide intravitreal implant) Glucocorticoid receptor agonist Uveitis Post-hoc analysis of data that examined imputed recurrences driven by use of confounding systemic medications through 36 months found 48% reported imputed recurrence in 10 treated participants, with 40% due to adjunctive medicine, 31% to systemic treatments and 9% to local injections; 71% of sham eyes had reported imputed recurrence, with 64% due to adjunctive medicine, 21% to systemic treatments and 43% to local injections
Moderna Inc., of Cambridge, Mass. mRNA-1273 COVID19 Spike glycoprotein modulator COVID-19 infection First of about 30,000 participants dosed in U.S. Cove study; primary endpoint is prevention of symptomatic disease; analysis will be event-driven based on number of participants with symptomatic disease
Rigel Pharmaceuticals Inc., of South San Francisco Tavalisse (fostamatinib disodium) Spleen tyrosine kinase inhibitor Immune thrombocytopenia Post-hoc analysis published in British Journal of Haematology showed study drug as second-line therapy produced higher response rate and decrease in bleeding incidents in in adults with chronic disease


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