Company Product Description Indication Status
Phase I
Creative Medical Technology Holdings Inc., of Phoenix, Ariz. Jadicells Umbilical cord-derived stem cells COVID-19 infection  At 28 days, 91% of Jadicell-treated patients survived; only 42% of patients in the placebo group survived; data published in Stem Cells Translational Medicine
Erasca Inc., of San Diego ERAS-601 SHP2 inhibitor Advanced solid tumors First patient dosed
Millendo Therapeutics Inc., of Ann Arbor, Mich. MLE-301 Neurokinin 3 receptor antagonist Menopausal vasomotor symptoms Compound generally well-tolerated but data do not support moving forward with its development 
Seres Therapeutics Inc., of Cambridge, Mass. SER-287 Oral, biologically derived microbiome therapeutic candidate Mild to moderate ulcerative colitis Results published in Gastroenterology demonstrate an impact on clinical remission and endoscopic improvement with a favorable safety profile 
Vir Biotechnology Inc., of San Francisco VIR-1111 T-cell vaccine HIV First patient dosed
Zentalis Pharmaceuticals Inc., of New York ZN-c5 Oral selective estrogen receptor degrader ER+/HER2- advanced or metastatic breast cancer Trial begun in combination with Verzenio (abemaciclib, Eli Lilly and Co.)
Zentalis Pharmaceuticals Inc., of New York ZN-c5 Oral selective estrogen receptor degrader Advanced ovarian cancer Trial begun in combination with chemotherapy
Zentalis Pharmaceuticals Inc., of New York ZN-c5 Oral selective estrogen receptor degrader Acute myeloid leukemia and non-Hodgkin lymphoma Trial begun
Phase II
Imara Inc., of Boston IMR-687 Small-molecule inhibitor of PDE9 Sickle cell disease Phase IIa results showed monotherapy and combination with hydroxyurea (HU) well-tolerated at all dose levels; monotherapy A1 substudy showed 25% lower rate of vaso-occlusive crises/sickle cell-related pain crises (VOCs/SCPCs) in treatment group vs. placebo; 58% of patients (7 of 12, 9 events total) experienced at least 1 VOC/SCPC in treatment group vs. 83% (5 of 6, 14 events total) in placebo population; rate of VOC-related hospitalizations was lower in treatment group, with 33% of patients (4 of 12) experiencing 1 VOC-related hospitalization in treatment group vs. 66% (4 of 6) on placebo; 1 of 7 evaluable patients (14%) in A1 recorded absolute increase in HbF percentage from baseline of greater than 1% (increase of 3.2%); in combination B1, data showed no meaningful differences in VOCs/SCPCs or VOC-related hospitalizations vs. HU+placebo groups; biomarker results showed an overall increase in F-cells and HbF levels from baseline to week 24, while Hb levels did not meaningfully change; 3 of 8 evaluable subjects (33%) had absolute increases in HbF percentage of greater than 1%, with a mean absolute increase in HbF percentage of 4.3% in that subset 
Infinity Pharmaceuticals Inc., of Cambridge, Mass. Eganelisib Inhibits PI3K-gamma Metastatic urothelial cancer Independent data monitoring committee (IDMC) for Mario-275 trial testing combination with Opdivo (nivolumab, Bristol Myers Squibb Co.) in platinum-refractory, immuno-oncology-naïve advanced disease determined there was a favorable risk/benefit for patients after implementation of dose reduction from 40 mg QD to 30 mg QD to reduce reversible liver enzyme elevations reported after first scheduled IDMC meeting; evaluation of 49 patients enrolled in study showed encouraging safety, response rate and progression-free survival, including in patients with low levels of PD-L1 expression
Inovio Inc., of Plymouth Meeting, Pa. VGX-3100 HPV E6 protein modulator Human papillomavirus-16/HPV-18-associated vulvar dysplasia Results from open-label study showed a 25% or greater reduction observed for 63% of participants (12 of 19) at 6 months post-treatment; 3 of 20 participants with histology data (15%) resolved their vulvar HSIL and had no HPV-16/18 virus detectable in the healed area; company intends to pursue phase III development
Windtree Therapeutics Inc., of Warrington, Pa. Lucinactant  Synthetic KL4 surfactant COVID-19-associated lung injury and acute respiratory distress syndrome Dosed first patient; study will evaluate changes in physiological parameters in COVID-19 patients who are intubated and mechanically ventilated
Phase III
Mycovia Pharmaceuticals Inc., of Durham, N.C. Oteseconazole  Antifungal Recurrent vulvovaginal candidiasis Top-line results showed Ultraviolet trial met all primary and secondary endpoints; recurrence rate was 5.1% with Oteseconazole vs. 42.2% for fluconazole to placebo recurrence rate (p<0.001); drug was noninferior to fluconazole in the resolution of signs and symptoms at day 14; recurrence rate of 3.8% vs. 41.1% for fluconazole to placebo (p<0.001); prevented a recurring episode in 95% of women for about 1 year
Ocuphire Pharma Inc., of Farmington Hills, Mich. Nyxol  Preservative-free, stable eye drop formulation of phentolamine mesylate Reverse pharmacologically induced mydriasis Completed enrollment earlier than expected in Mira-2 registration study
Regenxbio Inc., of Rockville, Md. RGX-314 Gene therapy Wet age-related macular degeneration First of 2 pivotal studies, Atmosphere, is active; patient screening is ongoing in study to enroll about 300 across 2 dose arms vs. ranibizumab; primary endpoint is noninferiority to ranibizumab based on change in baseline in Best Corrected Visual Acuity at 1 year


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