Company Product Description Indication Status
Phase I
Aligos Therapeutics Inc., of South San Francisco ALG-000184 Capsid assembly modulator Chronic hepatitis B Began dosing the first cohort
Chinook Therapeutics Inc., of Seattle BION-1301 Anti-APRIL monoclonal antibody IgA nephropathy Significantly reduced Gd-IgA1 levels in healthy volunteers
Ideaya Biosciences Inc., of South San Francisco IDE-397 Methionine adenosyltransferase 2a inhibitor Cancer harboring methylthioadenosine phosphorylase deletion First patient in
Longeveron Inc., of Miami Lomecel-B Allogeneic, bone marrow-derived medicinal signaling cell Influenza Study completed testing immune response to vaccine in subjects with aging frailty, with top-line results expected in the third quarter of 2021
Noxxon Pharma NV, of Berlin NOX-A12 CXCL12 inhibitor Brain cancer Completed patient recruitment
Panbela Therapeutics Inc., of Minneapolis SBP-101 Polyamine metabolic inhibitor Metastatic pancreatic ductal adenocarcinoma Partial clinical hold lifted
Qlaris Bio Inc., of Wellesley, Mass. QLS-101 Adenosine triphosphate-sensitive potassium channel modulator  Glaucoma First patient enrolled
Recce Pharmaceuticals Ltd., of Sydney RECCE-327 Bactericidal permeability protein inhibitor; outer membrane protein inhibitor Topical burns The phase I/II study was registered in the Australian New Zealand Clinical Trial Registry (ANZCTR); study will enroll 10 patients who will receive RECCE-327 daily for 14 days and 20 patients who will receive the drug 3 times per week for 2 weeks
Regenxbio Inc., of Rockville, Md. RGX-121 Gene therapy expressing iduronate-2-sulfatase Mucopolysaccharidosis type II Dosed first patient in cohort 3 with 2.0x10^11 genome copies per gram of brain mass, the highest planned dose in the ongoing phase I/II study
Regulus Therapeutics Inc., of La Jolla, Calif. RGLS-4326 Oligonucleotide designed to inhibit miR-17 and to preferentially target the kidney Autosomal dominant polycystic kidney disease First cohort completed dosing
Phase II
Abivax SA, of Paris ABX-464 Binds to the cap binding complex Moderate to severe ulcerative colitis Completed 16 weeks of induction treatment with different doses or placebo 
Afyx Therapeutics SA, of Copenhagen Rivelin Clobetasol Mucoadhesive patch designed to deliver clobetasol to lesions on wet tissue surfaces Oral lichen planus   Demonstrated clinically significant results at 20-µg dose
Anji Pharmaceuticals Inc., of Cambridge, Mass. Pradigastat  Inhibitor of diacylglycerol acyl transferase 1  Constipation Clinical sites added in the U.S. and China
Biolinerx Ltd., of Tel Aviv, Israel Motixafortide  CXCR4 inhibitor Pancreatic adenocarcinoma Substantial improvement observed across all study endpoints, including overall survival, progression-free survival and overall response rate, in the most challenging patients
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Vosoritide Analogue of C-type natriuretic peptide  Achondroplasia The mean (±SD) increase in annualized growth velocity observed over 60 months of treatment was 1.35 (±1.07) cm/year; there was an overall mean (±SD) increase in height Z-score (which measures the height deficit in standard deviations relative to the mean for age and gender-matched average stature children) at 60 months of 0.78 (±0.70) using the CDC standards for average stature children
Immunic Inc., of New York IMU-838 Orally available, next-generation selective immune modulator that inhibits the intracellular metabolism of activated immune cells by blocking the enzyme dihydroorotate dehydrogenase  Relapsing/remitting multiple sclerosis Data, along with previously published data from cohort 1, show 30 mg once daily is the most appropriate dose for future trials 
Inflarx NV, of Jena, Germany Vilobelimab Monoclonal antibody targeting complement factor C5a Pyoderma gangraenosum Reached target enrollment of 18 patients across the 3 dose groups; interim results expected by the end of 2021 with final results expected in 2022
Merck & Co. Inc., of Kenilworth, N.J., and Ridgeback Biotherapeutics LP, of Miami Molnupiravir (MK-4482/EIDD-2801) Ribonucleoside analogue Hospitalized COVID-19 An interim analysis of data from the phase II portion of the phase II/III Move-In trial concluded that the study was unlikely to demonstrate a clinical benefit; study is being discontinued
Merck & Co. Inc., of Kenilworth, N.J., and Ridgeback Biotherapeutics LP, of Miami Molnupiravir (MK-4482/EIDD-2801) Ribonucleoside analogue Outpatient COVID-19 In the phase II portion of the phase II/III Move-Out study, the percentage of patients given molnupiravir who were hospitalized and/or died was lower than the percentage of patients given placebo; on recommendation of the data monitoring committee, enrollment criteria will be changed to symptom duration of 5 or fewer days before enrollment and participants will have at least 1 risk factor for progression to severe disease; plans to start enrolling patients in the phase III portion of the study by late April or early May
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373 Recombinant protein vaccine COVID-19 prophylaxis Vaccine was added to the investigator-initiated Com-COV2 study of 1,050 adults 50 or older measuring immune system responses for 2 doses of the same vaccine compared to vaccination with 2 different vaccines
Roche Holding AG, of Basel, Switzerland Evrysdi (risdiplam) Survival of motor neuron 2 splicing modifier Spinal muscular atrophy More than twice as many babies (61% vs. 29%) were able to sit without support for at least 5 seconds after 24 months compared to 12 months of treatment
Phase III
Adlai Nortye Biopharma Co. Ltd., of Hangzhou, China Buparlisib (AN-2025) Pan-PI3K inhibitor Recurrent or metastatic head and neck squamous cell carcinoma Treated first of approximately 500 patients in the Buran study testing buparlisib plus paclitaxel; primary endpoint is overall survival
Covis Pharma BV, of Luxembourg Alvesco (ciclesonide) Glucocorticoid receptor agonist Non-hospitalized COVID-19 In the 400-patient study, 70.6% of patients taking Alvesco and 63.5% of patients taking placebo had an improved time to alleviation of COVID-19 related symptoms (p=0.5502); Alvesco produced a 70% reduction in subsequent emergency department visits or hospital admissions for reasons attributable to COVID-19 by day 30 compared to a 30% reduction for placebo (p=0.0301)
SK Life Science Inc., of Paramus, N.J. Cenobamate Inhibits voltage-gated sodium currents Uncontrolled partial-onset seizures In the C021 study, 33.9% of the 177 patients who remained on cenobamate had sustained seizure freedom for at least 12 months as of their last clinic visit; 46.9% of patients were seizure-free for at least 1 year at any time during the follow-up; 25% of patients discontinued all of their concomitant anti-seizure medications
Urovant Sciences Inc., a unit of Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan Gemtesa (vibegron) Beta-3 adrenergic agonist Overactive bladder Data from the Empowur trial and its extension study published in the Journal of Urology showed the least squares mean change from baseline to week 52 in micturitions was ?2.4 for Gemtesa compared to ?2 for tolterodine; urge urinary incontinence episodes was ?2.2 for Gemtesa compared to ?1.7 for tolterodine (p <0.05); incontinence episodes was ?2.5 for Gemtesa compared to ?1.9 for tolterodine (p <0.05)


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