Company Product Description Indication Status
Phase I
AC Immune SA, of Lausanne, Switzerland ACI-12589 Positron emission tomography imaging tracer targeting alpha-synuclein protein Parkinson’s disease First patient scanned in the study testing the ability of ACI-12589 to detect and map deposits of pathological alpha-synuclein protein in the brain; data expected in the third quarter of 2021
Avrobio Inc., of Cambridge, Mass. AVR-RD-02 Ex vivo lentiviral gene therapy Gaucher disease type 1 At 6 months, the first treated patient in phase I/II trial had plasma chitotriosidase and lyso-Gb1 levels that were below the baseline level achieved while on enzyme replacement therapy
Avrobio Inc., of Cambridge, Mass. AVR-RD-04 Ex vivo lentiviral gene therapy Cystinosis  All 3 patients in phase I/II trial remain off cysteamine pills and eye drops at 16, 6 and 2 months post-gene therapy, respectively.
Covaxx, a subsidiary of United Biomedical Inc., both of Hauppauge, N.Y. UB-612 Multitope protein/synthetic peptide-based vaccine COVID-19 prophylaxis UB-612 induced neutralizing antibodies in all participants; the 100-ug dose produced anti-S1-RBD and viral neutralizing antibody responses that compared favorably to responses observed in hospitalized patients who had recovered from COVID-19
Cue Biopharma Inc., of Cambridge, Mass. CUE-101 Protein E7 modulator; T-cell receptor agonist; IL-2 receptor agonist First-line human papillomavirus-positive recurrent/metastatic head and neck squamous cell carcinoma First patient treated in the dose-escalation study testing CUE-101 plus Keytruda (pembrolizumab, Merck & Co. Inc.); primary endpoint is dose-limiting toxicity and pharmacokinetics; secondary endpoint is overall response rate
Dong-A ST, of Seoul, South Korea DA-1241 GPR119 agonist Type 2 diabetes In a mixed meal tolerance test, 8 weeks of treatment with the 100-mg dose produced an incremental AUE0-4h of plasma glucose of -13.8% compared to +10.5% for placebo and -9% for sitagliptin 
Freeline Therapeutics Holdings plc, of London FLT-180a  AAV-based gene therapy Hemophilia B After receiving feedback from the FDA on comparability of the product produced at commercial scale, company plans to confirm the dose in a phase I/II study instead of a phase IIb/III study; phase I/II is expected to start by the end of 2021 with data expected by the end of 2022; pivotal phase III expected to start by mid-2023 with data by the end of 2024
Jasper Therapeutics Inc., of Redwood City, Calif. JSP-191 Monoclonal antibody targeting CD117 Myelodysplastic syndromes or acute myeloid leukemia undergoing hematopoietic cell transplantation All 6 patients had successful engraftment; 5 of 6 had complete donor myeloid chimerism equal to or greater than 95% at 28 days; all 3 evaluable patients had total donor chimerism equal or greater than 95% at day 90; 3 of 5 evaluable patients had complete eradication of measurable residual disease (MRD) at day 28; the other 2 patients had substantial reductions in MRD
Magenta Therapeutics Inc., of Cambridge, Mass. MGTA-145 Stem cell mobilization therapy Healthy volunteers (eventually hematopoietic stem cell transplantation) MGTA-145 plus plerixafor mobilized high numbers of hematopoietic stem cells
Uniqure NV, of Amsterdam, the Netherlands  AMT-130 HTT gene inhibitor Huntington disease Based on 6-month safety data from first 2 enrolled participants and 90-day safety data from next 2 enrolled in phase I/II study, data safety monitoring board recommended full enrollment of 6 people in first cohort, expected by mid-2021
Phase II
Avrobio Inc., of Cambridge, Mass. AVR-RD-01 Ex vivo lentiviral gene therapy Fabry disease First patient in the study had complete clearance of Gb3 inclusions in a kidney biopsy 12 months after treatment
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. Peginterferon Lambda Type III interferon Mild to moderate COVID-19 Data from the ILIAD study published in Lancet Respiratory Medicine showed the drug accelerated clearance of SARS-CoV2 in newly diagnosed, non-hospitalized patients; 4 patients who received placebo and 1 patient who received drug required emergency room visits due to deteriorating respiratory symptoms
Magenta Therapeutics Inc., of Cambridge, Mass. MGTA-456 CD34 expanded cord blood product High-risk hematologic malignancies MGTA-456 produced neutrophil recovery in a median of 17 days compared to 23 days for matched controls; platelet recovery occurred in a median of 36 days for MGTA-456 and 59 days for the control group; 24% of patients had grade 2-4 acute graft-vs.-host disease compared to 46% of the control group
Veru Inc., of Miami VERU-111 Targets, crosslinks and disrupts alpha and beta tubulin subunits of microtubules Acute respiratory distress syndrome from COVID-19 Respiratory failure rate was 5.6% for patients treated with VERU-111 and 30% for those treated with placebo (p=0.05); for patients older than 60, respiratory failure rates were 9% for VERU-111 vs. 50% for placebo (p=0.046); for patients with WHO Score of Disease Severity ≥5 at baseline, respiratory failure rates were 11% for VERU-111 vs. 54% for placebo (p=0.04)
Phase III
Cassava Sciences Inc., of Austin, Texas Simufilam  Restores normal shape and function of altered filamin A Mild to moderate Alzheimer’s disease Following an end-of-phase-II meeting with the FDA, company plans to start its phase III program in the second half of 2021; 1 study will evaluate disease-modifying effects over 18 months; the other study will evaluate symptomatic improvement over 6 months
Erytech Pharma SA, of Lyon, France Eryaspase  L-asparaginase encapsulated inside donor-derived red blood cells Second-line pancreatic cancer Following a planned interim superiority analysis by the independent data monitoring committee, the TRYbeCA-1 study will continue without modification
Sinovac Biotech Ltd., of Beijing Coronavac SARS-CoV-2 vaccine COVID-19 prophylaxis Of the 12,396 health care workers in Brazil and Turkey in the study, efficacy rate against diseases caused by COVID-19 starting 14 days after the second dose was 50.65% for all cases, 83.7% for cases requiring medical treatment and 100% for hospitalized, severe and fatal cases; combined results of health care workers and the general population in Turkey showed efficacy rate for COVID-19 prevention was 91.25% after 14 days following the 2-dose vaccination


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