Company Product Description Indication Status
Phase I
Agenus Inc., of Lexington, Mass. AgenT-797 Allogeneic iNKT cell therapy Hematologic cancers and solid tumors Treated first patient in the study that is starting with hematologic cancers and expects to expand into solid tumors shortly; endpoints include safety, tolerability and preliminary clinical activity of agenT-797; initial data expected in the fourth quarter of 2021
Aligos Therapeutics Inc., of South San Francisco ALG-000184 Class II capsid assembly modulator Chronic hepatitis B infection Started dosing the first cohort of patients in the ALG-000184-201 study testing the safety, pharmacokinetics and antiviral activity of up to 28 daily oral doses of ALG-000184
Auris Medical Holding Ltd., of Hamilton, Bermuda AM-301 Nasal spray gel Allergic rhinitis Study met the primary endpoint of noninferiority for Total Nasal Symptom Score relative to the marketed comparator product during a pollen challenge
Galectin Therapeutics Inc., of Norcross, Ga. Belapectin Galectin-3 Inhibitor Advanced metastatic melanoma and head and neck squamous cell carcinoma Data published in the Journal for ImmunoTherapy of Cancer showed belapectin plus Keytruda (pembrolizumab, Merck & Co. Inc.) produced an objective response rate of 50% in 14 patients with metastatic melanoma and 33% in 6 patients with head and neck squamous cell carcinoma
Gannex, a unit of Shanghai-based Ascletis Pharma Inc. ASC-41 Thyroid hormone receptor beta agonist Nonalcoholic fatty liver disease Started dosing in the first cohort that will test the effect of itraconazole and phenytoin on the pharmacokinetics of ASC-41 in healthy volunteers; second cohort will evaluate the pharmacokinetics, safety and tolerability of ASC-41 in patients
Moderna Inc., of Cambridge, Mass. mRNA-1345 Vaccine Respiratory syncytial virus Interim data show that through 1-month post-vaccination, of the younger adult cohorts, a single vaccination of 50 μg or 100 μg was generally well-tolerated in younger adults ages 18-49; vaccine was also shown to increase RSV neutralizing antibodies in seropositive younger adults
Nervgen Pharma Corp., of Vancouver, British Columbia NVG-291 Protein tyrosine phosphatase sigma inhibitor Diseases related to nerve damage Bellberry Human Research Ethics Committee in Australia approved the study in healthy volunteers; plans to dose first subject in the second quarter of 2021
Proqr Therapeutics NV, of Leiden, the Netherlands Sepofarsen CEP290 mRNA splicing modulator CEP290-mediated Leber congenital amaurosis 10 A case report published in Nature Medicine showed visual acuity, light sensitivity and visual fields reached a substantial efficacy peak near 3 months after injection and remained better than baseline at 15 months
Rocket Pharmaceuticals Inc., of Cranbury, N.J. RP-L201 Lentiviral-based gene therapy Severe leukocyte adhesion deficiency-I A patient followed for 18 months had durable CD18 expression of approximately 40% and resolution of skin lesions with no new lesions reported; a second patient followed for 9 months had CD18 expression of approximately 28%; 2 patients followed for 3 months had CD18 expression of approximately 51% and approximately 70%, respectively
Synthetic Biologics Inc., of Rockville, Md. SYN-004 (ribaxamase) Degrades certain I.V. beta-lactam antibiotics Allogeneic hematopoietic cell transplant First patient treated in the study testing the safety, tolerability and potential absorption into the systemic circulation of oral SYN-004 in patients given intravenous beta-lactam antibiotic to treat fever
Phase II
Achieve Life Sciences Inc., of Seattle Cytisinicline Plant-based alkaloid that binds to the nicotinic acetylcholine receptor Nicotine addiction Data published in Nicotine and Tobacco Research showed patients taking the 3-mg dose 3 times daily were 5 times more likely to quit smoking than patients who took placebo (p<0.001)
Moderna Inc., of Cambridge, Mass. mRNA-1647 Vaccine Cytomegalovirus 7-month interim data showed the 50-μg, 100-μg and 150-μg dose levels were generally well-tolerated
Orphomed Inc., of San Francisco ORP-101 Opioid receptor kappa antagonist/mu partial agonist Irritable bowel syndrome with diarrhea Passed the second interim futility analysis triggered by enrollment of 260 patients; top-line data expected in the second half of 2021
Phase III
Ardelyx Inc., of Fremont, Calif., and Kyowa Kirin Co. Ltd., of Tokyo Tenapanor Sodium hydrogen exchanger 3 inhibitor Hyperphosphatemia Started 4 studies in Japan: a placebo-controlled, parallel-group comparative study; a phosphate binder-combination parallel-group comparative study; an open-label, single-arm study in hyperphosphatemia patients on peritoneal dialysis; and a long-term study evaluating serum phosphorus in patients who switch from 1 or more phosphate binders to tenapanor
Astrazeneca plc, of Cambridge, U.K. and Veracyte Inc., of South San Francisco Calquence (acalabrutinib) BTK inhibitor Untreated diffuse large B-cell lymphoma First of up to 600 patients enrolled in the study using Veracyte’s Lymphmark lymphoma subtyping test to identify patients who may benefit from Calquence plus R-CHOP
Endo International plc, of Dublin Qwo (collagenase clostridium histolyticum-aaes) Combination of bacterial collagenases Moderate to severe cellulite in the buttocks Data from the Release-1 and -2 studies published in Dermatologic Surgery showed the drug met the primary endpoint of a composite 2-level response on a 5-point cellulite severity scale; over half of the women treated with Qwo in both studies also met the secondary endpoint, a 1-level improvement in the patient-reported assessment
Innocoll Biotherapeutics plc, of Athlone, Ireland Xaracoll (bupivacaine hydrochloride) Collagen drug-device Abdominoplasty and open soft-tissue surgeries Started the INN-CB-024 placebo-controlled study in patients undergoing abdominoplasty and the INN-CB-025 open-label study in patients undergoing open ventral hernia repair, abdominoplasty, open abdominal hysterectomy, laparoscopic-assisted colectomy or reduction mammoplasty
Romark Laboratories LC, of Tampa, Fla. NT-300 (nitazoxanide extended-release tablets) Inhibits viral replication Mild or moderate COVID-19 In the overall population, median time to sustained response was approximately 13 days for both patients treated with NT-300 and those treated with placebo; in the predefined subgroup with mild disease, median time to sustained response was 10.3 days for NT-300 and 13.4 days for placebo; 0.5% of NT-300-treated patients vs. 3.6% of patients treated with placebo progressed to severe illness

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