Company Product Description Indication Status
Phase I
Inhibikase Therapeutics Inc., of Atlanta IkT-148009 Abelson tyrosine kinase inhibitor Healthy volunteers (eventually Parkinson’s disease) Treated first subject in the study designed to determine the maximum tolerated dose and the pharmacokinetic profile of single and multiple ascending doses of IkT-148009 
Mesoblast Ltd., of New York Remestemcel-L Mesenchymal stromal cells Multisystem inflammatory syndrome associated with COVID-19 Data published in Pediatrics from the first 2 patients treated under an expanded access program; after treatment with remestemcel-L, patients had rapid normalization of left ventricular ejection fraction, reductions in biomarkers of systemic and cardiac inflammation and improved clinical status
Mind Medicine Inc., of Basel, Switzerland Ketanserin  LSD neutralizer LSD trip  Starting a 2-period crossover clinical trial testing the effect of ketanserin on the acute response to LSD in healthy subjects after LSD administration; study expected to be completed by the end of 2021
Salarius Pharmaceuticals Inc., of Houston Seclidemstat  Reversible inhibitor of the lysine-specific histone demethylase 1 enzyme Relapsed/refractory Ewing sarcoma Established the phase II dose in the phase I/II study; treatment at the recommended phase II dose achieved plasma concentrations above level shown to produce preclinical activity; data to be presented at an upcoming medical meeting
Selecta Biosciences Inc., of Watertown, Mass., and Asklepios Biopharmaceutical Inc., of Research Triangle Park, N.C. SEL-399 Adeno-associated viral serotype 8 empty vector capsid containing no DNA combined with ImmTOR Healthy volunteers (eventually gene therapy treatment) Started a dose-escalation trial to determine the optimal dose of ImmTOR to mitigate formation of antibodies to adeno-associated viral serotype 8
Silence Therapeutics plc., of London                                                          SLN-360 RNAi targeting LPA Elevated lipoprotein (a) levels Started dosing patients in the single-ascending and multiple-ascending dose study; data from the single-ascending dose portion of the study expected in the second half of 2021
Phase II
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia Ifenprodil  Noncompetitive NMDA antagonist  COVID-19 Source data audit for the phase IIb portion of the phase IIb/III study from all sites and for all patients is complete; database will be locked for analysis on 3/5/21
Biophytis SA, of Paris Sarconeos (BIO-101) Proto-oncogene Mas agonist Acute respiratory failure associated with COVID-19 Starting recruiting in France and Belgium for the ongoing part 2 of the COVA study; interim analysis of part 1 of the study expected in the first quarter of 2021; results from full study expected in 2Q2021
Cidara Therapeutics Inc., of San Diego Rezafungin Echinocandin  Candidemia and/or invasive candidiasis Investigator-assessed clinical cure and mycological response at day 14 was 75% in 12 high-risk immunocompromised patients treated with rezafungin compared to 66.7% in 9 patients treated with caspofungin; safety and efficacy of rezafungin were not adversely affected by renal impairment
Evgen Pharma plc, of Cheshire, U.K. SFX-01 Up-regulates Nrf2 pathway Acute respiratory distress syndrome in patients with suspected COVID-19 Star trial has recruited and randomized 102 patients; initial data for the interim analysis of first 100 patients expected in the 2Q2021
Immunic Inc., of New York IMU-838 DHODH inhibitor Moderate COVID-19 In the Calvid-1 study, rate of invasive ventilation for hospitalized patients with moderate COVID-19 was less than 1%, complicating analysis of primary endpoint; secondary endpoints of 28-day mortality, survival without respiratory failure, and probability of use of intensive care unit were similarly low; 18.5% of 15 patients treated with IMU-838 reached clinical recovery at day 7 compared to 12.8% of 10 patients treated with placebo
Kintara Therapeutics Inc., of San Diego VAL-083 Bifunctional alkylating agent Glioblastoma multiforme who have been pre-treated with temozolomide prior to disease recurrence Enrolled final patient in the phase II study measuring overall survival
Moebius Medical Ltd., of Tel Aviv, Israel MM-I Non-opioid intra-articular injection Pain in knee osteoarthritis First of approximately 312 patients treated in the phase IIb study; primary endpoint is reduction in pain compared to baseline as measured according to Western Ontario and McMaster Universities' Osteoarthritis Index A pain score at 12 weeks; top-line data expected to be available in mid-2022
Pfizer Inc., of New York Elranatamab (PF-06863135) Bispecific antibody targeting B-cell maturation antigen and CD3 Relapsed/refractory multiple myeloma First patient treated in the study that will enroll 90 patients who have prior treatment with a BCMA-directed ADC or CAR-T therapy and 60 patients who haven't; primary endpoint is objective response rate as assessed by blinded independent central review; secondary endpoints include duration of response, progression-free survival, minimal residual disease negativity rate, overall survival and safety; estimated primary completion date is June 2022
PTC Therapeutics, Inc., of South Plainfield, N.J. PTC-299 Targets dihydroorotate dehydrogenase COVID-19 Started the second stage of the Fite19 study, which will enroll an additional 340 patients
Redhill Biopharma Ltd., of Tel Aviv, Israel RHB-107 (upamostat) Serine protease inhibitor COVID-19 outpatient Started dosing of the first part of the phase II/III study; primary endpoint is time to sustained recovery from illness
Scisparc Ltd., of Tel Aviv, Israel SCI-110 Combination of dronabinol and palmitoylethanolamide  Alzheimer's disease Signed an agreement with The Israeli Medical Center for Alzheimer's to conduct a phase IIa study; primary endpoint is safety; secondary endpoint is ability of the drug to ameliorate agitation and other behavioral disturbances
Phase III
Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. Lynparza (olaparib) Poly ADP-ribose polymerase inhibitor Germline BRCA-mutated, high-risk HER2-negative early-stage breast cancer At the interim analysis, the independent data monitoring committee for the Olympia study determined that the trial crossed the superiority boundary for the primary endpoint of invasive disease-free survival
Eli Lilly and Co., of Indianapolis Tirzepatide  Dual glucose-dependent insulinotropic polypeptide and glucagon-like peptide-1 receptor agonist Type 2 diabetes At 52 weeks in the Surpass-3 study, treatment with 5 mg, 10 mg and 15 mg of tirzepatide reduced A1C by 1.93%, 2.20% and 2.37%, respectively, compared to a reduction of 1.34% for insulin degludec; weight loss for tirzepatide was also statistically significant compared to insulin degludec; at 40 weeks in the Surpass-5 study, treatment with 5 mg, 10 mg and 15 mg of tirzepatide reduced A1C by 2.23%, 2.59% and 2.59%, respectively, compared to a reduction of 0.93% for placebo; weight loss for tirzepatide was also statistically significant in the study

Notes

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