Company Product Description Indication Status
Phase I
Gannex, of Shanghai, wholly owned unit of Ascletis Pharma Inc. ASC-42 Farnesoid X receptor agonist Nonalcoholic steatohepatitis First healthy participant dosed 
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. INO-4800 COVID19 spike glycoprotein modulator COVID-19 infection Data from first cohort of 40 participants, published in The Lancet's open-access Eclinicalmedicine, found candidate was immunogenic in all vaccinated subjects, generating immune response of humoral (including neutralizing antibodies) and/or cellular responses
Phase II
Algernon Pharmaceuticals Inc., of Vancouver, British  Columbia NP-120 (repurposed ifenprodil) NMDA receptor epsilon 2 subunit inhibitor COVID-19 infection Final participant from phase IIb portion of IIb/III study completed treatment and 2-week follow-up; final data expected by end of February 2021
Lysogene SA, of Paris LYS-SAF-302 AAVrh10-h.SGSH gene therapy  Mucopolysaccharidosis type IIIA Biomarker data from ongoing phase II/III AAVance trial showed reductions in concentration of heparan sulfate (HS) in cerebrospinal fluid of first 9 participants analyzed, at 6 and 12 months after treatment, relative to pre-treatment values, with average reductions highly statistically significant; no statistically significant changes in serum HS concentrations seen following treatment 
Phase III
Aim Immunotech Inc., of Ocala, Fla.  Ampligen (rintatolimod) TLR3 agonist immune system modulator COVID-19 infection Long hauler portion of active AMP-511 expanded access program protocol received IRB approval for public notification of potential enrollment of up to 20 participants with persistent post-infection symptoms
Aprea Therapeutics Inc., of Boston Eprenetapopt p53 tumor suppressor protein stimulator Myelodysplastic syndrome Trial assessing study drug with azacitidine (AZA) vs. AZA alone in TP53 mutant disease missed predefined primary endpoint of complete remission (CR) rate; in intent-to-treat population (n=154), CR rate for study drug arm was 33.3% (95% CI: 23.1%-44.9%) vs. 22.4% (95% CI: 13.6%-33.4%) for AZA alone (p=0.13)
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373 COVID19 spike glycoprotein modulator COVID-19 infection Pivotal Prevent-19 study initiated in U.S. and Mexico with goal of enrolling up to 30,000 volunteers 18 and older, with two-thirds to receive vaccine candidate; primary endpoint is prevention of PCR-confirmed symptomatic infection
Taiwan Liposome Co. (TLC), of Taipei, Taiwan  TLC-599 Bioseizer sustained-release formulation of dexamethasone sodium phosphate Knee osteoarthritis pain Pivotal Excellence study fully enrolled on schedule; top-line data expected in second half of 2021

Notes

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