Company Product Description Indication Status
Phase I
Arbutus Biopharma Corp., of Warminster, Pa. AB-729 GalNAc-delivered RNAi compound Chronic hepatitis B virus infection Data from ongoing AB-729-001 phase Ia/Ib trial showed repeat dosing of 60 mg every 4 and 8 weeks resulted in comparable declines in mean HBsAg through week 16; data support advancing into phase IIa studies in 2021
Bellicum Pharmaceuticals Inc., of Houston BPX-603 Dual switch GoCAR T product Tumors expressing HER2 Enrollment and apheresis of first patient in phase I/II trial testing administration with rimiducid
Fusion Pharmaceuticals Inc., of Hamilton, Ontario FPI-1434 Radioimmunoconjugate connecting antibody targeting IGF-1R with alpha-emitting isotope actinium-225 Advanced solid tumors Dosed first patient in multidose portion of trial
Pharming Group NV, of Leiden, the Netherlands Ruconest  Recombinant human C1 inhibitor COVID-19 Enrolled first patient in pilot study involving up to 120 hospitalized subjects with confirmed COVID-19 treated for the prevention of severe SARS-CoV-2 infections
Stalicla SA, of Geneva STP-1 Combination of pan-PDE inhibitor and modulator of NKCC1 Autism spectrum disorder phenotype 1 subgroup Enrolled first patient in phase Ib trial
Ultimovacs ASA, of Oslo, Norway UV-1 Telomerase inhibitor Metastatic malignant melanoma 5-year data from open-label trial testing combination with Yervoy (ipilimumab, Bristol Myers Squibb Co.) showed 50% overall survival
Phase II
Clene Nanomedicine Inc., of Salt Lake City CNM-Au8 Concentrated, aqueous suspension of clean-surfaced faceted gold nanocrystals Amyotrophic lateral sclerosis Blinded interim results from Rescue-ALS trial showed, as of Oct. 27, 2020, data cutoff, more than 40% of patients with completed week 12 data experienced improvements in motor function assessed by MUNIX; completed, unblinded results expected in second half of 2021
Immutep Ltd., of Sydney, and EOC Pharma Group, of Shanghai Eftilagimod alpha Soluble LAG-3-Ig fusion protein Metastatic breast cancer EOC launched study in up to 152 patients in China
Inovio Inc., of Plymouth Meeting, Pa., and Advaccine Biopharmaceuticals Co. Ltd., of Suzhou, China INO-4800 DNA vaccine COVID-19 Dosed first subject in China; study to enroll about 640 patients, 18 and older
Kintor Pharmaceutical Ltd., of Shanghai GT-90001 ALK-1 antibody Advanced hepatocellular carcinoma Data from studies testing combination with Opdivo (nivolumab, Bristol Myers Squibb Co.) showed, among 20 evaluable patients, 8 (40%) showed partial remission
Moderna Inc., of Cambridge, Mass. mRNA-1273 mRNA vaccine COVID-19 First adolescent participants dosed in phase II/III study in patients ages 12-18; conducted in collaboration with U.S. Biomedical Advanced Research and Development Authority
Puretech Health plc, of Boston LYT-100 (deupirfenidone) TGF beta receptor antagonist; p38 MAP kinase inhibitor; PDGF receptor antagonist Breast cancer-related, upper limb secondary lymphedema Initiated phase IIa proof-of-concept trial; results expected in fourth quarter of 2021
Regenxbio Inc., of Rockville, Md. RGX-314 NAV AAV8- based gene therapy encoding antibody fragment to inhibit VEGF Diabetic retinopathy Dosed first patient in Altitude study testing suprachoroidal delivery using SCS Microinjector; initial data expected in 2021
Spero Therapeutics Inc., of Cambridge, Mass. SPR-720 Oral antimicrobial agent Nontuberculous mycobacterial pulmonary disease Initiated dosing in dose-ranging phase IIa trial; top-line results expected in first half of 2022
Sunovion Pharmaceuticals Inc., of Marlborough, Mass.  SEP-4119 5-HT 7 receptor agonist Bipolar depression Primary analysis showed numerical improvement in Montgomery-Åsberg Depression Rating Scale (MADRS) total score after 6 weeks of treatment (200 mg: -19.5 vs. -16.2, 400 mg: -19.3 vs. -16.2 respectively, both dose groups vs. placebo, p=0.054; 200-mg group effect size [ES] = -0.31 and 400 mg group ES = -0.29); in full intent-to-treat analysis, including those enrolled in Japan, the least squares (LS) mean reduction from baseline at week 6 in MADRS total score showed improvement vs. placebo for both 200- and 400-mg doses (-3.68 [p=0.016] and -3.38 [p=0.024], respectively)
Tracon Pharmaceuticals Inc., of San Diego Envafolimab Subcutaneously administered single-domain antibody against PD-L1 Sarcoma Dosed first patient in Envasarc registration trial expected to enroll 160 patients; primary endpoint of objective response rate with duration of response as key secondary endpoint; top-line data expected in mid-2021
Phase III
Abbvie Inc., of North Chicago Rinvoq (upadacitinib) JAK1 inhibitor Atopic dermatitis In phase IIIb Heads Up study, Rinvoq 30 mg once daily achieved superiority to Dupixent (dupilumab, Sanofi SA/Regeneron Pharmaceuticals Inc.) 300 mg every other week, meeting primary endpoint of proportion with at least 75% improvement in EASI 75 at week 16 (71% vs. 61%, respectively, p=0.006) in moderate to severe disease; study drug also showed superiority vs. Dupixent for ranked secondary endpoints, including additional measures of skin clearance and itch reduction
Abivax SA, of Paris ABX-464 Rev protein modulator Ulcerative colitis; COVID-19 infection; Crohn's disease Pivotal UC program will begin in 2021 following completion of fully enrolled phase IIb study and 4 additional phase I studies in healthy volunteers, including study in healthy Japanese volunteers to confirm drug's pharmacokinetic profile, in agreement with FDA and EMA; also in 2021, ongoing phase IIb/III study in COVID-19 expected to complete and planned pivotal phase IIb/III study in Crohn's to initiate; manufacturing scale-up and process optimization underway
Apellis Pharmaceuticals Inc., of Waltham, Mass., and Swedish Orphan Biovitrum AB (Sobi), of Stockholm Pegcetacoplan Complement C3 inhibitor; complement cascade inhibitor Paroxysmal nocturnal hemoglobinuria  Sustained improvement in hemoglobin seen at week 48 in Pegasus study; all (n=77) who completed 16-week randomized period entered extension; mean increase from baseline of 2.7 g/dL equaled increase seen at week 16 in treated participants; sustained improvements also seen in transfusion avoidance, reticulocyte count, lactate dehydrogenase level and FACIT-fatigue score
Gensight Biologics SA, of Paris Lumevoq (GS-010) MT-ND4 gene stimulator Leber hereditary optic atrophy Data from pivotal Reverse trial, reported in May 2019, published in Science Translational Medicine documenting sustained and clinically meaningful bilateral improvement in visual outcomes from unilateral injection
Geron Corp., of Foster City, Calif. Imetelstat Telomerase inhibitor Myelodysplastic syndromes Registration-enabling Imerge trial in lower-risk disease reached 50% enrollment; full enrollment expected in second quarter of 2021, with top-line results projected in second half of 2022
Obseva SA, of Geneva Yselty (linzagolix) GNRH antagonist Uterine fibroids Top-line 52-week Primrose 1 results showed sustained efficacy for primary endpoint of reduced heavy menstrual bleeding across all doses, in line with earlier findings in Primrose 2; pooled week 52 results from the studies showed 56.4% of women on 100 mg met primary endpoint and responder rate was 89.3% on 200 mg+add-back therapy
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162b2 COVID-19 spike glycoprotein modulator COVID-19 infection Safety and final efficacy results from pivotal trial confirming 95% effectiveness against COVID-19 published in The New England Journal of Medicine; 170 cases observed with onset at least 7 days after second dose, including 8 in vaccine recipients and 162 in placebo
Savara Inc., of Austin, Texas Aerovanc (vancomycin hydrochloride dry-powder, inhaled) Peptidoglycan recognition protein inhibitor Methicillin-resistant Staphylococcus aureus  Avail trial in people with cystic fibrosis missed primary endpoint of percent predicted mean absolute change from baseline in FEV1 (vs. placebo) of 1.4 at week 4 (end of cycle 1; p=0.33), 1.3 at week 12 (end of cycle 2; p=0.33) and 3 at week 20 (end of cycle 3; p=0.07); exacerbation rate per year was 2.3 for study drug and placebo; development halted
TG Therapeutics Inc., of New York Ublituximab B-lymphocyte antigen CD20 modulator Multiple sclerosis Ultimate I and II monotherapy trials met primary endpoint of significantly reducing annualized relapse rate (ARR), at p<0.005 in each study, with ublituximab showing ARR of <0.10 in each; relative reductions of approximately 60% and 50% in ARR over Aubagio (teriflunomide, Sanofi SA) observed in respective studies


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