Company Product Description Indication Status
Phase I
Advaxis Inc., of Princeton, N.J. ADXS-503 Cancer-type specific immunotherapy Non-small-cell lung cancer Disease control rate of 67% in first evaluable patients who had progressed on Keytruda (pembrolizumab, Merck & Co. Inc.)
Allakos Inc., of Redwood City, Calif. Lirentelimab Non-fucosylated IgG1 monoclonal antibody targeting Siglec-8 Eosinophilic gastritis Subcutaneous formulation was safe and well-tolerated and demonstrated sustained eosinophil suppression supporting once-monthly dosing
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. ARQ-252 Topical small-molecule JAK1 inhibitor Chronic hand eczema Completed enrollment in phase I/IIb study; top-line data expected by mid-2021
Cingulate Therapeutics Inc., of Kansas City, Kan. CTx-1301 Dexmethylphenidate Attention deficit hyperactivity disorder Results from phase I/II study demonstrated the comparative bioavailability (bridging) between drug and Focalin XR (d-methylphenidate extended-release capsules), confirming the 505(b)(2) pathway
Exelixis Inc., of Alameda, Calif. XL-092 Oral tyrosine kinase inhibitor that targets VEGF receptors, MET, AXL, MER and other kinases Advanced solid tumors Enrollment of the first patient into the dose-escalation cohort of the combination arm pairing the drug with Cabometyx (atezolizumab, Roche Holding AG)
Galera Therapeutics Inc., of Malvern, Pa. Avasopasem manganese Selective small-molecule superoxide dismutase mimetic  Radiation-induced mucositis Interim data showed improvement in overall survival and multiple anticancer efficacy measures
Gmax Biopharm LLC, of Hangzhou, China GMA-301 Humanized monoclonal antibody (IgG4) against endothelin receptor type A  Pulmonary arterial hypertension First patient dosed
Hifibio Therapeutics Inc., of Cambridge, Mass. HFB-30132A SARS-CoV-2 neutralizing antibody  Treatment and prevention of COVID-19 First cohort completed
Mustang Bio Inc., of Worcester, Mass. MB-105 Prostate stem cell antigen chimeric antigen receptor T therapy Prostate cancer In 1 patient, drug demonstrated on day 28 a 94% reduction in prostate-specific antigen, near complete reduction of measurable soft tissue metastasis by computerized tomography, and improvement in bone metastases by magnetic resonance imaging
Nanobiotix SA, of Paris NBTXR-3 Radioenhancer Solid tumors Expansion part of the head and neck cancer study showed target lesion objective response rate of 83.9%, confirming data reported earlier in 2020; target lesion complete response rate of 67.7%, which was an increase over previously reported data (60%) with additional follow-up
NBE Therapeutics AG, of Basel, Switzerland NBE-002 Receptor tyrosine kinase-like orphan receptor 1 targeter Triple-negative breast cancer and other solid tumors Study commenced
Phase II
Ana Therapeutics Inc., of Foster City, Calif. Oral niclosamide (ANA-001) Antiviral; anti-inflammatory Moderate COVID-19 disease Started phase II/III trial in hospitalized patients (not requiring ventilators) to receive 7-day course in addition to standard of care
Atyr Pharma Inc., of San Diego ATYR-1923 Selective modulator of neuropilin-2 COVID-19 Completed enrollment of a total of 32 patients with severe respiratory complications; top-line data expected by end of 2020
Biophytis SA, of Paris Sarconeos (BIO-101) Small molecule COVID-19-related respiratory failure First patient in Brazil dosed in Cova phase II/III trial; objective is to prevent patients from being admitted to intensive care unit and requiring ventilation
Biosig Technologies Inc., of Westport, Conn., and subsidiary Viralclear Pharmaceuticals Inc. Merimepodib Inosine monophosphate dehydrogenase inhibitor Advanced COVID-19 Halted signal-finding study testing combination with intravenous remdesivir after unblinded safety monitoring committee detected imbalance in survival rates in grade 3 patients
Crinetics Pharmaceuticals Inc., of San Diego Paltusotine (CRN-00808) Oral nonpeptide biased agonist designed to be selective for somatostatin receptor subtype 2 Acromegaly Top-line results from Acrobat Edge study showed primary endpoint was achieved, with once-daily dosing maintaining IGF1 levels at week 13 in patients who were switched from injected somatostatin receptor ligand depot of either octreotide or lanreotide monotherapy (change in IGF-1 = -0.034  [-0.107, 0.107], median); data from Acrobat Evolve study, which had been halted early, did not allow for meaningful statistical comparisons due to reduced sample size
Forma Therapeutics Holdings Inc., of Watertown, Mass. Olutasidenib Small-molecule IDH1 inhibitor IDH1-mutant relapsed/refractory acute myeloid leukemia Data showed favorable tolerability profile as monotherapy; treatment achieved composite complete remission (CR) rate of 33.3% (30% CR and 3% CR with partial hematologic recovery), the primary efficacy endpoint; median duration of CR/CRh has not been reached, but sensitivity analysis indicates median duration to be 13.8 months
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda Mavrilimumab Fully human monoclonal antibody targeting GM-CSFRalpha Giant cell arteritis Study hit primary endpoint (time to first adjudicated GCA flare by week 26; p=0.-263) and secondary efficacy endpoint (sustained remission at week 26 in all treated patients; 83.2% vs. 49.9% for placebo, p=0.0038) 
Novartis AG, of Basel, Switzerland Iptacopan (LNP-023) Small-molecule, reversible inhibitor of factor B C3 glomerulopathy Interim results showed that after 12 weeks, drug significantly reduced proteinuria by 49% vs. baseline values, as measured by 24-hour urine protein/creatinine ratio (UPCR) assessment, in 12 patients (p=0.0005); strongly inhibited alternative complement pathway activity and improved plasma C3 levels, and also stabilized renal function as assessed by estimated glomerular filtration rate at week 12, with that effect maintained in 7 patients treated for a total of 6 months after rolling over into long-term extension study 
PDS Biotechnology Corp., of Florham Park, N.J. PDS-0101 Vaccine based on Versamune T-cell activating technology Locally advanced cervical cancer Study now open testing combination with standard-of-care chemoradiotherapy 
PTC Therapeutics Inc., of South Plainfield, N.J. Vatiquinone (PTC-743) Oral small molecule that inhibits 15-lipoxygenase Mitochondrial epilepsy Started registration-directed MIT-E phase II/III study, which will randomize about 60 patients with genetically confirmed mitochondrial disease and associated refractory epilepsy; primary endpoint is the reduction of observed motor seizure frequency in the placebo-controlled phase relative to the run-in phase
Reistone Biopharma Co. Ltd., of Shanghai SHR-0302 Selective JAK1 inhibitor Moderate to severe atopic dermatitis Quartz2 study met both primary and secondary endpoints; at week 12, 8-mg dose achieved an IGA score of "clear" and "almost clear" of  54.3% compared to 5.7% for placebo (p<0.001), while 4-mg dose achieved IGA success rate of 25.7% (p=0.022); achieved statistically significant differences vs. placebo for EASI75 (8 mg, 74.3% vs. placebo, 22.9%, p<0.001; 4 mg, 51.4% vs. placebo, p=0.013); study also demonstrated significant improvement in itch symptoms scores
Phase III
Allergy Therapeutics plc, of Worthing, U.K. Grass MATA MPI Short-course, aluminum-free allergen-specific immunotherapy Seasonal allergic rhinitis and/or rhinoconjunctivitis induced by grass pollen exposure Screened first patient in exploratory field study to evaluate efficacy of optimized phase III dose
Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl)  Apolipoprotein B antagonist; phospholipase A2 inhibitor Cardiovascular Reduce-IT Renal results from cardiovascular outcomes in patients with compromised renal function showed patients with decreased renal function prior to treatment had higher rates of cardiovascular events than the overall population studied in Reduce-IT; patients with decreased renal function prior to treatment showed similarly favorable relative risk reductions and numerically greater absolute risk reductions in cardiovascular events vs. overall patient population; consistent and robust benefit in cardiovascular outcomes seen in both prespecified and post hoc exploratory analyses across a broad range of baseline estimated Glomerular Filtration Rate categories
Biocardia Inc., of San Carlos, Calif. Cardiamp Cell therapy Heart failure Data from initial open-label roll-in cohort, published in the International Journal of Cardiology, show treatment is well-tolerated and demonstrated improved distance in 6-minute exercise testing at 6 months and a trend toward improvement at 1 year; the 10-patient cohort was the feasibility test for the currently enrolling CardiAMP HF phase III trial
Glycomimetics Inc., of Rockville, Md. Rivipansel Binds to E-, P- and L-selectin Vaso-occlusive crisis Post hoc analyses of Reset study showed pediatric patients treated within about 30 hours of onset of acute crisis pain experienced statistically significant improvements in primary efficacy endpoint of time to readiness for discharge (p=0.02) vs. placebo as well as in key secondary endpoints time-to-discharge (p=0.02)and time-to-discontinuation-of-I.V. opioids (p=0.045)
Rexgenero Ltd., of London REX-001 Suspension of immune and progenitor cells Critical limb ischemia Independent data monitoring committee agreed to continue trial unchanged after review of initial 22 patients
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Dupixent (dupilumab) IL-13 receptor antagonist; IL-4 receptor antagonist  Eosinophilic esophagitis Results from part A of pivotal trial in patients 12 and older showed rapid improvement in ability and comfort of swallowing: patients reported significant improvement on Dysphagia Symptom Questionnaire as early as 4 weeks and continued to improve through 24 weeks (p<0.05 and p<0.001, respectively); reduced esophageal eosinophil count below diagnostic disease threshold: 64% of patients achieved <15 eosinophils/high-power field vs. 8% for placebo at 24 weeks (p<0.001); peak esophageal eosinophil count was reduced by 71% with Dupixent vs. 3% with placebo from baseline (p<0.001)
VBI Vaccines Inc., of Cambridge, Mass. Sci-B-Vac 3-antigen prophylactic hepatitis B vaccine Hepatitis B Data from Protect and Constant trials showed vaccine safely elicited higher anti-HBs titers in all study subjects compared to Engerix-B (Glaxosmithkline plc), a single antigen HBV vaccine, regardless of age, gender or underlying co-morbidity; rapid onset of seroprotection rates (SPR) in young adults with 3-antigen HBV vaccine vs. single-antigen vaccine; in 4 studies, after 2 vaccinations, Sci-B-Vac achieved SPR rates of 87.2%-100% compared to 39%-89.4% for Engerix-B


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