Company Product Description Indication Status
Phase I
Apellis Pharmaceuticals Inc., of Waltham, Mass. Pegcetacoplan  C3 inhibitor Age-related macular degeneration Early data from the phase Ib APL2-103 study in patients with advanced geographic atrophy secondary to AMD found no cases of inflammation observed after 12 months and decrease in mean lesion growth in 9 patients with bilateral GA
Inovio Pharmaceuticals Inc., of Plymouth, Pa. INO-4800 DNA vaccine COVID-19 Trial fully enrolled with 40 healthy volunteers; targeting summer start for phase II/III trial
Phase II
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-701 (talabostat) Inhibitor of DPP4, DPP8, DPP9 and fibroblast activation protein Treatment-emergent neuroendocrine prostate cancer Started phase II portion of phase Ib/II trial in combination with Keytruda (pembrolizumab); interim data readout expected in fourth quarter of 2020
Generex Biotechnology Corp., of Miramar, Fla. AE37 +/- GM-CSF vaccine HER2 peptide vaccines Breast tumor Final results of phase IIb trial published in Breast Cancer Research & Treatment concluding that AE37 Ii-Key peptide vaccine is safe and associated with disease-free survival in subsets of breast cancer survivors after 10-year follow-up
Innate Pharma SA, of Marseille, France Avdoralimab Anti-C5aR antibody COVID-19 First patient dosed in placebo-controlled randomized, double-blind trial called Force; expects to enroll 108 patients
Innovent Biologics Inc., of Suzhou, China Parsaclisib  Phosphoinositide-3 kinase delta inhibitor Follicle center lymphoma; marginal zone B-cell lymphoma First patient dosed in pivotal China trial
Orchard Therapeutics plc, of London OTL-201 Ex vivo autologous hematopoietic stem cell gene therapy Mucopolysaccharidosis type IIIA First patient dosed in an open-label, proof-of-concept study
R-Pharm JSC, of Moscow, and Cromos Pharma LLC, of Portland, Ore. Olokizumab and RPH-104 IL-6 receptor antagonist and IL-1 beta ligand inhibitor COVID-19 Randomized first patients in phase II/III trial expected to recruit about 180 patients
Rafael Pharmaceuticals Inc., of Cranbury, N.J. CPI-613 (devimistat) Thioctoid Burkitt lymphoma Expanded trial in relapsed or refractory Burkitt lymphoma/leukemia
Spectrum Pharmaceuticals Inc., of Henderson, Nev. Poziotinib  Pan-HER inhibitor of EGFR Non-small-cell lung cancer Zenith20 trial amended to include new dosing regimens aimed at improving therapeutic index after results for cohort 1 missed primary endpoint
Phase III
Blueprint Medicines Corp., of Cambridge, Mass. Ayvakit (avapritinib) Kit tyrosine kinase inhibitor; PDGF receptor alpha antagonist Gastrointestinal stromal tumor Failed to meet primary endpoint of improving progression-free survival vs. regorafenib in patients with locally advanced unresectable or metastatic gastrointestinal stromal tumor
Genentech Inc., of South San Francisco, a unit of Roche Holding AG Risdiplam   SMN2 gene splicing modulator Spinal muscular atrophy Firefish Part 2 study met its primary endpoint by demonstrating a significant increase in motor milestones in infants with SMA type 1 ages 1-7 months after 12 months of treatment; following priority review, FDA review decision expected by Aug. 24, 2020
I-Mab Biopharma Co. Ltd., of Shanghai, and Morphosys AG, of Martinsried, Germany TJ-202/MOR-202 Monoclonal antibody targeting CD38 Multiple myeloma Dosed first patient in randomized, open-label, parallel-controlled mainland China part of trial evaluating efficacy when combined with lenalidomide/dexamethasone (len/dex) vs. len/dex alone
Ocular Therapeutix Inc., of Bedford, Mass. Dextenza (dexamethasone) Glucocorticoid receptor agonist Allergic conjunctivitis Treated subjects demonstrated a statistically significant (p<0.0001) change in ocular itching from baseline at all 3 pre-specified time points

Notes

For more information about individual companies and/or products, see Cortellis.

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