Company Product Description Indication Status
Phase I
Alligator Bioscience AB, of Lund, Sweden ATOR-1015 Bispecific CTLA4/OX40 antibody Melanoma Start of phase Ib trial delayed for amendments to adjust for grade 3 infusion reactions at dose of 750 mg (12.5 mg/kg) in ongoing phase I study
Ampio Pharmaceuticals Inc., of Englewood, Colo. Ampion Aryl hydrocarbon receptor agonist; stem cell antigen-1 inhibitor COVID-19 infection Study assessing intravenous formulation met primary endpoint of safety and tolerability; treatment group improved and, at hospital discharge, had stronger clinical improvement than standard-of-care group, measured by WHO clinical improvement scale and National Early Warning Score
Astrazeneca plc, of Cambridge, U.K., and The Leukemia & Lymphoma Society, of Rye Brook, N.Y. Calquence (acalabrutinib) Bruton’s tyrosine kinase inhibitor COVID-19 in patients with acute myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndromes and aplastic anemia Launched phase I/II study testing the drug in approximately 60 patients
Brain Neurotherapy Bio Inc., of Columbus, Ohio GDNF gene therapy  AAV2 gene therapy expressing glial cell line-derived neurotrophic factor Early to moderate stages of Parkinson’s disease  Treated first patient in the phase Ib study
Enterome SA, of Paris EO-2401  Microbiome-antigen based cancer immunotherapy Adrenocortical carcinoma and malignant pheochromocytoma/paraganglioma  Started the phase I/II Spencer study testing the safety, tolerability, immunogenicity and preliminary efficacy of EO-2401 in up to 72 patients
Flexion Therapeutics Inc., of Burlington, Mass. FX-201 IL-1Ra gene therapy  Osteoarthritis First patient treated in the second cohort of the dose-escalation study; data from study expected in 2021
Scholar Rock Inc., of Cambridge, Mass. SRK-181 Inhibitor of latent TGFbeta1 activation Locally advanced or metastatic solid tumors First patient treated in part A2 of the Dragon study testing SRK-181 in combination with anti-PD-(L)1 therapy; part A1, testing SRK-181 monotherapy has progressed through 800 mg and continues to advance dose escalation
Sinovac Biotech Ltd., of Beijing Coronavac Vaccine COVID-19 Data from phase I/II studies in China show good safety and immunogenicity in healthy adults ages 60 and older, which is comparable to result seen in healthy adults ages 18-59 in earlier studies; both seroconversion rate and GMT level for elderly volunteers were comparable to younger adult group (98% and 42.2 vs. 97.4% and 44.1); the medium dose was selected to enter into phase III testing 
Trillium Therapeutics Inc., of Cambridge, Mass. TTI-622  SIRPa-IgG4 Fc fusion protein  Advanced relapsed or refractory lymphoma or multiple myeloma Objective response rate was 33% in 18 evaluable patients; 3 of 6 patients treated at the highest level had a response
Trillium Therapeutics Inc., of Cambridge, Mass. TTI-621  SIRPa-IgG1 Fc fusion protein  Advanced relapsed or refractory hematologic malignancies  At the 1-mg/kg dose, there was 1 partial response and 1 skin complete response in 6 evaluable patients; study is enrolling 2-mg/kg dose level
Viking Therapeutics Inc., of San Diego VK-0214 Thyroid receptor beta agonist Healthy subjects (eventually X-linked adrenoleukodystrophy) Started enrollment in study measuring safety and tolerability of single and multiple oral doses of VK-0214; study will also identify VK-0214 doses for further clinical development in patients
Phase II
Applied Genetic Technologies Corp., of Gainesville, Fla. AAV-RPGR gene therapy  Gene therapy expressing RPGR X-linked retinitis pigmentosa  Plans to start a study in the first quarter of 2021; study will compare 2 doses to an untreated control group; primary endpoint is response rate; defined as having at least 7 decibel improvement in visual sensitivity in at least 5 loci at month 12
Aztherapies Inc., of Boston ALZT-OP1a  Inhaled formulation of cromolyn Mild- to moderate-stage amyotrophic lateral sclerosis Treated first of 80 patients in the study measuring plasma biomarkers and functional changes using the ALS Functioning Rating Scale-Revised over a 12-week treatment period
Cellectar Biosciences Inc., of Florham Park, N.J. CLR-131  Phospholipid-drug conjugate delivering iodine-131 Triple class refractory multiple myeloma In the Clover-1 study, overall response rate was 40% for 15 patients who received a total administered dose of 60 mCi or greater
Intensity Therapeutics Inc., of Westport, Conn. INT230-6  Cisplatin and vinblastine with a penetration enhancer molecule Colon (non-MSI high), pancreatic and bile duct cancers and squamous cell carcinoma Treated first patient in the expansion cohorts of the Keynote A10 study testing INT230-6 plus Keytruda (pembrolizumab, Merck & Co. Inc.)
Lipac Oncology LLC, of Menlo Park, Calif. Lipax (paclitaxel)  Proliposomal paclitaxel formulation   Non-muscle invasive bladder cancer  Response rate of 56% in patients treated with Lipax
Myokardia Inc., of Brisbane, Calif. Danicamtiv (MYK-491) Cardiac myosin activator  Primary dilated cardiomyopathy Treated first of up to 24 patients; primary endpoint is safety and tolerability; secondary endpoints include cardiac pharmacodynamic parameters as measured by echocardiography; top-line data expected in second half of 2021
Neurosense Therapeutics, of Herzliya, Israel NST-002  Undisclosed Amyotrophic lateral sclerosis After 6 months of the 15-patient phase IIa study, there were trends toward slowing of disease progression based on both ALS Functioning Rating Scale-Revised and vital capacity; trends were seen in last 3 months of the study, which will continue for an additional 6 months
Regenxbio Inc., of Rockville, Md., and Clearside Biomedical Inc., of Alpharetta, Ga. RGX-314  AAV-based gene therapy using Clearside’s SCS Microinjector Wet age-related macular degeneration Treated first patient in the study
Phase III
Amryt Pharma plc, of Dublin Filsuvez (AP-101/Oleogel-S10) Keratinocyte modulator  Dystrophic and junctional epidermolysis bullosa  The Ease study met its primary endpoint showing the drug improved the proportion of patients with complete closure of the target wound within 45 days of treatment compared to control gel (p=0.013); results to be presented at an upcoming scientific symposium
Astrazeneca plc, of Cambridge, U.K. AZD-1222 Vaccine COVID-19 Study paused due to an unexplained illness
Astrazeneca plc, of Cambridge, U.K. Dapagliflozin SGLT2 inhibitor Type 2 diabetes mellitus Study in Asian patients with inadequate glycemic control on metformin/saxagliptin terminated; sponsor decided to stop commercialization of Qternmet/Qtrilmet (dapagliflozin/saxagliptin/metformin) and to stop all related ongoing activities/studies for business reasons
Intra-Cellular Therapies Inc., of New York Lumateperone Serotonin 5-HT2A receptor antagonist Major depressive episodes associated with bipolar I or bipolar II disorder  In Study 402, lumateperone 42 mg produced a least squares mean reduction from baseline on the MADRS total score of 16.9 points compared to 14.5 points for placebo (p=0.0206); drug produced a 0.31 effect size on the CGI-BP-S Depression Score (p=0.0082)
Merck & Co. Inc., of Kenilworth, N.J. V-114 15-valent pneumococcal conjugate vaccine  Pneumococcal disease prophylaxis In the Pneu-Age study, V-114 was noninferior to the currently available 13-valent pneumococcal conjugate vaccine (PCV13) for the 13 serotypes targeted by both vaccines and superior for serotypes 22F and 33F only targeted by V114; in the Pneu-True study, V-114 met its primary immunogenicity objective demonstrating equivalent immune response across all 15 serotypes for 3 different lots of the vaccine
Roche Holding AG, Basel, Switzerland Fenebrutinib Bruton’s tyrosine kinase inhibitor Multiple sclerosis Started 3 studies testing drug in relapsing MS (Fenhance 1 and Fenhance 2) and primary progressive MS (Fentrepid); all studies have primary endpoint of 12-week composite confirmed disability progression; relapsing MS studies have co-primary endpoint of annualized relapse rate
Roche Holding AG, Basel, Switzerland Ocrevus (ocrelizumab) Monoclonal antibody targeting CD20-positive B cells Multiple sclerosis Starting phase IIIb Musette study in relapsing MS and Gavotte study in primary progressive MS comparing the currently approved 600-mg dose to a higher dose
Phase IV
Octapharma USA Inc., of Paramus, N.J. Octagam 10% Intravenous immunoglobulin COVID-19 pneumonia  Of the 14 patients treated with Octagam, 2 progressed to mechanical ventilation compared to 7 of 12 patients treated with standard of care (SOC) (p=0.038); median hospital stay was 11 days for those treated with Octagam compared to 19 days for SOC (p=0.01); median change in oxygenation (PaO2/FiO2) from baseline to day 7 was +131 for Octagam and +44.5 for SOC (p=0.01)
Roche Holding AG, Basel, Switzerland Ocrevus (ocrelizumab) Monoclonal antibody targeting CD20-positive B cells Multiple sclerosis Started Chimes study in African-Americans and Hispanic- and Latinx-Americans with relapsing MS


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