Company Product Description Indication Status
Phase I
Abfero Pharmaceuticals Inc., of Boston SP-420 Iron chelator Myelodysplastic syndromes and myelofibrosis Started study in patients with transfusional iron overload
Ampio Pharmaceuticals Inc., of Englewood, Colo. Ampion (AP-014) Aryl hydrocarbon receptor agonist; stem cell antigen-1 inhibitor COVID-19 Added 2 new study sites to ongoing trial testing inhalation treatment
Arrys Therapeutics Inc., of Cambridge, Mass. Grapiprant (ARY-007) EP4 receptor antagonist Advanced or metastatic post-PD-1/L1 non-small-cell lung cancer adenocarcinoma  Phase I/II study testing combination with Keytruda (pembrolizumab, Merck & Co. Inc.) terminated, based on totality of generated combined safety and efficacy data in interim period
Frequency Therapeutics Inc., of Woburn, Mass. FX-322 Stem cell stimulator Acquired sensorineural hearing loss Phase I/II data published in Otology & Neurotology showed statistically significant increases in word recognition (WR) and words-in-noise (WIN) scores; individuals with stable SNHL that received a single dose showed improvements in the number of words recognized in quiet from baseline to day 90 in the WR test (p=0.029) and in the WIN test (p=0.012), while there were no meaningful changes in WR and WIN scores of the placebo group
LG Chem Life Sciences Co., of Seoul, Korea LG-00303174 (formerly TT-01025) SSAO/VAP-1 inhibitor Nonalcoholic steatohepatitis First of about 64 healthy adults dosed
Matrisys Bioscience Inc., of La Jolla, Calif. MSB-0221 Allogenic targeted microbiome transplant containing Staphylococcus hominis A9 Atopic dermatitis Data from trial in 54 adults that met primary safety endpoint and showed decreased colonization by S. aureus (p<0.001) published in Nature Medicine 
Targovax ASA, of Oslo, Norway ONCOS-102 GM-CSF receptor agonist Mesothelioma At 21-month follow-up, phase I/II trial of study drug + standard-of-care chemotherapy showed half of participants in first-line treated group remained alive, with median overall survival not reached
Union Therapeutics A/S, of Hellerup, Denmark UNI-91103 (intranasal niclosamide)  SKP2 inhibitor COVID-19 infection Agent selected for assessment of efficacy as prophylaxis in Protect-V trial at University of Cambridge, which recruited first of about 1,500 people with kidney disease
Phase II
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. Avexitide GLP-1 antagonist  Post-bariatric hypoglycemia Prevent trial results published in Journal of Clinical Endocrinology & Metabolism; primary endpoint was met with statistical significance in both 30-mg and 60-mg regimens
Redhill Biopharma Ltd., of Tel Aviv Opaganib Sphingosine kinase-2 inhibitor  COVID-19 infection Ongoing phase II/III study in people with severe infection to expand to U.S. following FDA review of U.S. phase II data and recommendations
Revance Therapeutics Inc., of Nashville, Tenn. DaxibotulinumtoxinA Acetylcholine receptor antagonist; botulinum toxin A stimulator Upper limb spasticity In Juniper trial that enrolled 83 adults with moderate to severe spasticity, all 3 doses (250 units, 375 units, 500 units) were numerically higher than placebo for improvement in MAS score and 500-unit dose achieved statistically significant reduction from baseline in muscle tone vs. placebo (p=0.0488); all doses showed numerical improvement vs. placebo on PGIC assessment but missed statistical significance 
Stealth Biotherapeutics Corp., of Boston Elamipretide Mitochondria-targeted compound Dry age-related macular degeneration with geographic atrophy Reclaim-2 trial completed enrollment with 176 participants; top-line data on primary endpoint of low-luminance BCVA expected in first half of 2022
Valbiotis SA, of La Rochelle, France TOTUM-070 Food plant extract-derived substance Hypercholesterolemia First of 120 participants had first clinic visit in Heart study assessing effectiveness of 5 g daily on blood LDL-cholesterol levels as primary endpoint
Vtv Therapeutics Inc., of High Point, N.C. TTP-399 Glucokinase activator Type 1 diabetes Simplicit-1 study data, published in Diabetes Care, showed treatment at 800-mg dose produced statistically significant reductions in HbA1c and resulted in clinically relevant (~40%) reduction in frequency of severe or symptomatic hypoglycemia vs. placebo
Phase III
Octapharma AG, OF Lachen, Switzerland Nuwiq (simoctocog alfa) Factor VIII agonist Hemophilia A Final results from NuProtect study on agent's immunogenicity in 105 previously untreated people with severe disease, published in Thrombosis and Haemostasis, showed 16.2% (n=17) developed high-titer inhibitors; 10.5% (n=11) developed low-titer inhibitors, including 5 that were transient; and 26.7% (n=28) developed any inhibitor
Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Global Rare Diseases, unit of Chiesi Farmaceutici SpA, of Parma, Italy Pegunigalsidase alfa (PRX-102) Alpha-galactosidase stimulator Fabry disease Top-line data from phase III Bright switch-over trial showed 2 mg/kg of study drug every 4 weeks maintained stable clinical presentation in adults and no new participants developed treatment-induced anti-drug antibodies following switch 
TG Therapeutics Inc., of New York Ublituximab Glycoengineered anti-CD20 monoclonal antibody Chronic lymphocytic leukemia Final results from Genuine combination trial with ibrutinib (Imbruvica, Johnson & Johnson/Abbvie Inc.) in 126 people with relapsed/refractory high-risk disease, published in The Lancet Haematology, showed overall response rate 90% (53 of 59) for combination vs. 69% (40 of 58) for ibrutinib alone (p=0.0060), with complete response/complete response with incomplete hematologic recovery rate of 20% (12 of 59) and 5% (3 of 58), respectively (p=0.024)


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