Company Product Description Indication Status
Phase I
Adaptimmune Therapeutics plc, of Oxfordshire, U.K. ADP-A2M4CD8 SPEAR T-cell therapy incorporating an affinity optimized TCR targeting MAGE-A4 and a CD8α co-receptor Advanced cancers expressing MAGE-A4 In the Surpass study, there was 1 partial response in the 5 treated patients; other 4 patients had stable disease
Avalyn Pharma Inc., of Seattle AP-01  Inhaled pirfenidone Idiopathic pulmonary fibrosis Last patient completed the 24-week treatment
Respira Therapeutics Inc., of Albuquerque, N.M. RT234-PAH (vardenafil) Phosphodiesterase type 5 inhibitor Healthy volunteers (pulmonary arterial hypertension) In the multiple ascending dose portion of the study, 2.4-mg dose every 4 hours for up to 4 doses per day for 7 consecutive days was well-tolerated with no dose-limiting toxicity observed and low incidence of cough and local airway irritation
Rocket Pharmaceuticals Inc., of New York RP-L201 Ex vivo lentiviral gene therapy Leukocyte adhesion deficiency-I One patient had durable CD18 expression of 40% with visible signs of improvement in existing skin lesions and no new infections at 12 months post-treatment; second patient had CD18 expression of 28% at 4 months post-treatment
Phase II
Amylyx Inc., of Cambridge, Mass. AMX-0035  Targets endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways Amyotrophic lateral sclerosis Data from the open-label extension of the Centaur study published in Muscle & Nerve showed patients who received the drug at baseline had a 44% lower risk of death compared to the group who originally received placebo (p=0.023)
Arch Biopartners Inc., of Toronto Metablok LSALT peptide Moderate to severe COVID-19 First patient treated in the study testing whether the drug can reduce acute lung injury, acute kidney injury and other complications caused by inflammation
Faron Pharmaceuticals Oy, of Turku, Finland, and Merck KGaA, of Darmstadt, Germany Traumakine (intravenous interferon beta-1a), Rebif (subcutaneous interferon beta-1a) Interferons COVID-19 In the Solidarity study, overall in-hospital morality was 12.9% for the 1,412 patients who received interferon beta-1a and 11% for the 2,050 patients in the control group; most patients in the interferon beta-1a group received Rebif; unclear how many patients received Traumakine
Galapagos NV, of Mechelen, Belgium, and Les Laboratoires Servier SAS, of Paris GLPG-1972/S-201086 Targets ADAMTS-5 Knee osteoarthritis Change from baseline to week 52 in cartilage thickness was -0.116 mm for the placebo group and -0.068 mm, -0.097 mm and -0.085 mm, for the low, medium and high doses of the drug, respectively
Imara Inc., of Boston IMR-687 PDE9 inhibitor Beta-thalassemia Treated first of approximately 120 patients measuring the reduction of days between red blood cell transfusions and change in iron load rate as a result of transfusions for transfusion dependent patients; for non-transfusion dependent patients, effects on fetal hemoglobin and anemia will be measured; interim analyses after 30 patients have reached 24 weeks of treatment and when 30 patients have reached 36 weeks of treatment
Lipella Pharmaceuticals Inc., of Pittsburgh LP-10 (liposomal tacrolimus) Inhibits calcineurin Hemorrhagic cystitis Started recruiting patients for the study
Pharmamar SA, of Madrid, Spain Aplidin (plitidepsin) Compound extracted from ascidian Aplidium albicans Hospitalized COVID-19 Average reduction of the viral load was 50% on day 7 and 70% on day 15; 80.7% of patients discharged on or before the 15th day of hospitalization; plans to start a phase III study
Respira Therapeutics Inc., of Albuquerque, N.M. RT234-PAH (vardenafil) Phosphodiesterase type 5 inhibitor Pulmonary arterial hypertension In the dose-escalating study, pulmonary vascular resistance was reduced within 5 minutes of administration of RT234-PAH; drug produced acute improvements in exercise tolerance
Y-mabs Therapeutics Inc., of New York Naxitamab  Monoclonal antibody targeting GD2 Relapsed/refractory high-risk neuroblastoma In the pivotal 201 study of 22 patients, overall response rate was 68%, including 59% complete response; bone marrow clearance plus complete response was seen in 7 of 9 patients with positive bone marrow at start of study
Y-mabs Therapeutics Inc., of New York Omburtamab  Monoclonal antibody targeting B7-H3 CNS/leptomeningeal metastasis from neuroblastoma In the pivotal 101 study of 17 patients, 12-month overall survival was 87%, compared to approximately 30% for historical controls
Zynerba Pharmaceuticals, Inc. Zygel  CBD transdermal gel Fragile X syndrome In a pre-planned ad hoc analysis of patients having at least 90% methylation of the FMR1 gene, patients taking Zygel had a 40% improvement in their Social Avoidance subscale of the ABC-CFXS compared to a 21.1% improvement for placebo (p=0.020) 
Phase III
Amarin Corp., of Dublin Vascepa (icosapent ethyl) Apolipoprotein B antagonist; phospholipase A2 inhibitor Hypertriglyceridemia Post hoc analysis of patient in the Reduce-it study who had undergone a percutaneous coronary intervention showed time to first 5-point major adverse cardiovascular event (MACE) was reduced by 34% with Vacepa vs. placebo (p<0.0001); total (first and subsequent) events were reduced by 39% (p<0.0001); for 3-point MACE, time to first event was reduced by 34% (p<0.0001)


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