Company Product Description Indication Status
Phase I
Azafaros BV, of Leiden, the Netherlands AZ-3102 Glucosylceramide synthase inhibitor GM1 and GM2 gangliosidoses First cohort of healthy participants dosed in 2-part study
Dynavax Technologies Corp., of Emeryville, Calif., and Valneva SE, of Saint-Herblain, France VLA-2001 Inactivated viral vaccine with CpG 1018 adjuvant COVID-19 infection Part A of phase I/II trial in 153 healthy adults ages 18 to 55 showed 100% seroconversion rate for S-protein binding IgG antibodies in high-dose group and neutralizing antibody titers at or above levels seen in convalescent sera; pivotal phase III expected to begin by end of April 2021, subject to regulatory approval
I-Mab Biopharma Co. Ltd., of Shanghai, and ABL Biotechnologies Inc., of Seongnam, South Korea TJ-L14B/ABL-503 Bispecific CDw137 agonist/PD-L1 inhibitor Advanced solid tumors First participant dosed in study assessing preliminary antitumor activity, maximum tolerated dose and/or recommended phase II dose
Opiant Pharmaceuticals Inc., of Santa Monica, Calif. OPNT-003 (nasal nalmefene) Opioid antagonist Opiate dependence First healthy volunteers dosed in head-to-head study vs. nasal naloxone to reverse respiratory depression produced by synthetic opioid, remifentanil; top-line data expected in fourth quarter of 2021
Pinteon Therapeutics Inc., of Cambridge, Mass. PNT-001 Tau antibody Traumatic brain injury First of 64 participants with acute TBI dosed in phase Ib study
Turning Point Therapeutics Inc., of San Diego TPX-0046 RET inhibitor Non-small-cell lung cancer and medullary thyroid carcinoma In the Sword-1 study, of the 5 RET tyrosine kinase inhibitor (TKI)-naive patients, 4 had tumor regressions with 2 patients at the highest dose tested reaching partial responses; of 9 TKI-pretreated patients, 3 achieved tumor regressions; plans to continue dose finding before moving to dose-expansion cohorts of up to 75 patients at the recommended phase II dose
Union Therapeutics A/S, of Hellerup, Denmark Niclosamide Inhaled (UNI-91103) and intranasal (UNI-91104) formulations of the antihelminth  COVID-19 Data published in The Lancet Regional Health – Europe showed the drug was well-tolerated in 34 healthy volunteers; drug had dose-proportional pharmacokinetic; no signs of systemic accumulation
Phase II
Aptinyx Inc., of Evanston, Ill. NYX-458 NMDA receptor modulator Cognitive impairment and mild dementia associated with Parkinson’s disease and dementia with Lewy bodies Resumed patient screening following a pause due to the COVID-19 pandemic; data expected in the second half of 2022
Azurrx Biopharma Inc., of Delray Beach, Fla. Niclosamide (FW-1022) Antihelminth  COVID-19-related gastrointestinal infections Started the Reservoir study; primary efficacy endpoint is the rate of fecal SARS-CoV-2 virus clearance; top-line data expected in the first quarter of 2022
Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. Enhertu (trastuzumab deruxtecan) HER2-directed antibody-drug conjugate HER2-overexpressing locally advanced, unresectable or metastatic colorectal cancer after progression following standard-of-care chemotherapy First patient treated in the Destiny-CRC02 study testing 2 dose levels of the drug; primary endpoint is confirmed objective response rate by blinded independent central review; secondary endpoints include duration of response, disease control rate, clinical benefit ratio, investigator-assessed ORR, progression-free survival, overall survival, pharmacokinetics, patient-reported outcomes and safety
Dilafor AB, of Stockholm, a Karolinska Development AB portfolio company Tafoxiparin  Heparan sulfate mimetic Initiate labor Completed enrollment of 170 patients in the phase IIb study; data expected in the first half of 2021
Enzychem Lifesciences Corp., of Englewood Cliffs, N.J. EC-18 CCL26 gene inhibitor; TLR modulator Chemoradiation induced oral mucositis Completed enrollment of 80 patients in stage 2 of the phase II study; database lock is expected near the end of the second quarter of 2021
Molecular Partners AG, of Zurich, Switzerland, and Novartis AG, of Basel, Switzerland Ensovibep Binds to the SARS-CoV-2 spike protein at 3 distinct locations COVID-19 First of up to 40 patients treated in the study measuring the dynamics of viral clearance, pharmacokinetics and tolerability of ensovibep
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373  Protein-based vaccine COVID-19 prophylaxis Started crossover (placebo to vaccine or vaccine to placebo) for the phase IIb study in South Africa
Scholar Rock Inc., of Cambridge, Mass. Apitegromab (SRK-015) Monoclonal antibody targeting promyostatin  Type 2 and type 3 spinal muscular atrophy In ambulatory type 3 patients 5-21 years of age, mean change from baseline in Revised Hammersmith Scale (RHS) was a 0.3-point decline after 12 months; 57% of the 23 patients improved their RHS score; in type 2 and non-ambulatory type 3 patients 5-21 years of age, mean change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) of a 0.6-point improvement; 64% of the 14 patients had a >1-point increase in HFMSE; in type 2 patients who were 2 years or older and less than 5 at the start of treatment, mean change from baseline in HFMSE was 7.1-point and 5.3-point improvements for the  20-mg/kg and 2-mg/kg doses, respectively
Phase III
Neurorx Inc., of Radnor, Pa., and Relief Therapeutics Holding AG, of Geneva Zyesami (aviptadil) VIP receptor agonist COVID-19 Aviptadil plus Veklury (remdesivir, Gilead Sciences Inc.) will be tested in the 640-patient Tesico study run by National Institute of Allergy and Infectious Diseases; primary endpoint is participant recovery from respiratory failure over 90 days
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373  Protein-based vaccine COVID-19 prophylaxis Started crossover (placebo to vaccine or vaccine to placebo) for the phase III study in the U.K. 
Phase IV
Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson Uptravi (selexipag) Prostacyclin receptor agonist Pulmonary arterial hypertension Data from the Sphere registry study published in the Journal of Heart and Lung Transplantation showed 56% of patients treated with Uptravi maintained their 1-year mortality risk score and 20% of patients reduced their score


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