Company Product Description Indication Status
Phase I
Aim Immunotech Inc., of Ocala, Fla. Ampligen  Ribonuclease stimulator; 2,5-oligoadenylate synthetase stimulator; TLR-3 agonist; polymerase co-factor VP35 inhibitor  COVID-19 and other respiratory viral diseases Completed dosing of 10 healthy subjects in cohort 2 who received 200 μg of Ampligen; proceeding to enrollment of cohort 3 at a dose of 500 μg
Ampio Pharmaceuticals, of Englewood, Colo. Ampion  Stem cell antigen-1 inhibitor COVID-19 Mortality rate was 5% for patients treated with Ampion plus standard of care (SOC) compared to 24% for SOC alone; average hospitalization time was 4 days less for the Ampion group
Discgenics, Inc., of Salt Lake City IDCT Allogeneic discogenic cell therapy Lumbar disc degeneration Independent data monitoring committee cleared the drug in the first of 2 planned midtrial safety reviews
Logicbio Therapeutics Inc., of Lexington, Mass. LB-001 MUT gene stimulator Methylmalonic acidemia First 2 patients screened in the Sunrise study didn’t meet all of the enrollment criteria; expected to enroll the first patient by midyear 2021
Phase II
Alkermes plc, of Dublin Nemvaleukin alfa Interleukin-2 variant Melanoma previously treated with anti-PD-(L)1 therapy Started the Artistry-6 study; primary endpoint is overall response rate; secondary endpoints include safety and tolerability, duration of response, progression-free survival, disease control rate and time to response
Lumos Pharma Inc., of Austin, Texas LUM-201 (ibutamoren) Promotes the secretion of growth hormone from the pituitary gland Pediatric growth hormone deficiency LUM-201 increased the pulsatile release of growth hormone (GH) for 24 hours in 2 patients positive for predictive enrichment marker (PEM), resulting in sizable increases in height velocity after 6 months of treatment; there was no increase in GH and no increase in height velocity in 1 PEM-negative patient
Noxopharm Ltd., of Sydney Veyonda  Topoisomerase inhibitor and radiopharmaceutical Moderate COVID-19 Interim data from the Noxcovid study suggests Veyonda provides protection against progression of the severe inflammation associated with a worsening of the disease; biomarker data expected within the next few weeks
Protagonist Therapeutics Inc., of Newark, Calif. Rusfertide (PTG-300) Hepcidin mimetic Polycythemia vera Completed enrollment in the study; interim data expected to be presented at a major medical meeting in the second quarter of 2021
Reithera Srl, of Rome GRAd-COV2 Vaccine COVID-19 prophylaxis Completed enrollment of 900 participants
Sifi SpA, of Cantania, Italy, and Glia LLC, of Boston Pro-ocular Progesterone-based topical gel Dry eye disease Enrolled first of 105 patients in the ProGIFT study comparing 2 dose levels of Pro-ocular to placebo; primary endpoints are corneal fluorescein staining and sum of frequency and intensity of dryness/irritation patient feeling assessed by SANDE questionnaire
Verona Pharma pl., of London Ensifentrine PDE3/PDE4 inhibitor Chronic obstructive pulmonary disease Data published in the International Journal of Chronic Obstructive Pulmonary Disease showed ensifentrine produced a placebo-adjusted peak forced expiratory volume in 1 second of 77.5 and 124.2 for the 0.375-mg and 3-mg doses, respectively (p=0.037 and p< 0.001, respectively)
Phase III
Aldeyra Therapeutics Inc., of Lexington, Mass. Reproxalap  Inhibitor of reactive aldehyde species Allergic conjunctivitis Compared to placebo, reproxalap produced a reduction in ocular itching score over all 11 prespecified primary endpoint comparisons from 110 to 210 minutes in the allergen chamber (p<0.0001 for each comparison); drug improved investigator-assessed ocular redness over the duration of the allergen chamber (p<0.0001), change from baseline in patient-reported ocular tearing score on a 0‑3 point scale over the duration of the allergen chamber (p<0.0001) and change from baseline in total ocular severity score over the duration of the allergen chamber (p<0.0001)
Aravive Inc., of Houston AVB-500 Fusion protein targeting GAS6 Platinum-resistant ovarian cancer Treated first of 300-400 patients in the study testing AVB-500 plus paclitaxel; primary endpoint is progression-free survival
Brickell Biotech Inc., of Boulder, Colo. Sofpironium bromide gel Blocks acetylcholine Primary axillary hyperhidrosis Completed enrollment in the Cardigan I study; Cardigan II study is more than 50% enrolled; top-line results from both studies expected in the fourth quarter of 2021
Obseva SA, of Geneva Yselty (linzagolix) GNRH receptor antagonist Uterine fibroids Started the Primrose 3 long-term follow-up study to the Primrose 1 and 2 studies to measure bone mineral density at 12, 18 and 24 months
Revance Therapeutics Inc., of Nashville, Tenn., and Shanghai Fosun Pharmaceutical Group Co. Ltd., of Shanghai DaxibotulinumtoxinA  Botulinum toxin Glabellar lines and cervical dystonia Started studies testing the drug in both indications


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