Company Product Description Indication Status
Phase I
Aeglea Biotherapeutics Inc., of Austin, Texas Pegzilarginase Arginase-I stimulator Arginase 1 deficiency A 56-week analysis showed a statistically significant reduction in plasma arginine from baseline was observed with a single dose, with continued improvement through the 20- and 56-week analyses, and all 13 patients achieved plasma arginine levels within the target range; the most common treatment-related serious adverse events were hypersensitivity and hyperammonemia, both of which were infrequent and expected, and no treatment-related serious adverse events led to patient discontinuation
Atara Biotherapeutics Inc., of South San Francisco ATA-188 Allogeneic T-cell activated therapy Progressive forms of multiple sclerosis Data from an ongoing study demonstrated it was well-tolerated across all 4 dose cohorts; there was a higher proportion of patients showing sustained disability improvements with increasing dose, and sustained disability improvements seen at 6 months were maintained at 12 months in all 3 cohorts that have reached the 12-month time point
Durect Corp., of Cupertino, Calif. DUR-928 Sulfated oxysterol Nonalcoholic steatohepatitis In top-line results from its phase Ib study, both the 50-mg and 600-mg dose groups showed a statistically significant median reduction at day 28 from baseline of serum alanine aminotransferase levels at -16% and -17%, respectively; the 600-mg dose group also showed statistically significant median reductions at day 28 from baseline of serum aspartate aminotransferase (-18%) and gamma-glutamyl transferase (-8%); DUR-928 was well-tolerated at all 3 doses, and there were no serious adverse events reported
Glaxosmithkline plc, of London GSK-3858279 Small inducible cytokine A17 ligand inhibitor  Pain Recruitment activities in study testing battery of evoked pain tests in healthy volunteers temporarily suspended due to COVID-19
Immunocore Ltd., of Oxfordshire, U.K. IMC-F106C Bispecific developed using Immtac technology platform that targets tumors that express PRAME PRAME-expressing cancers Started a first-in-human trial, designed to study its safety and preliminary activity as a monotherapy and in combination with a checkpoint inhibitor 
Innovent Biologics Inc., of Suzhou, China IBI-939 Anti-T-cell immunoreceptor with Ig and ITIM domains (TIGIT) recombinant fully human monoclonal antibody Advanced malignancies The first patient was dosed in a trial in China
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373 SARS-CoV-2 recombinant spike protein nanoparticle vaccine COVID-19 infection The first participants were enrolled in phase I portion of a phase I/II trial; preliminary immunogenicity and safety results from the phase I portion are expected in July 2020
Oncolytics Biotech Inc., of San Diego Pelareorep Oncolytic retrovirus Breast cancer Data from the Aware-1 study showed that all patients treated with pelareorep demonstrated an increase in CD8+ T cells as confirmed in tumor biopsies (range of 1.6-fold to 11.2-fold increase) and experienced an increase in the number of PD-L1-positive cells in their tumors in as early as 3 weeks after beginning treatment (range of 1.3-fold to 11-fold increase); 4 of 6 evaluated patients exhibited an increase in CelTIL, which is associated with favorable clinical response, the study's primary endpoint
Phase II
Arena Pharmaceuticals Inc., of San Diego Etrasimod Once-daily, oral S1P receptor modulator Moderate to severe atopic dermatitis Completed full enrollment of 140 patients in phase IIb Advise study; top-line data expected by year-end
Clene Nanomedicine Inc., of Salt Lake City CNM-Au8 Nanocatalytic therapy Amyotrophic lateral sclerosis Australian subsidiary completed more than 50% of participant randomization in Rescue-ALS study, with 26 of a planned 42 enrolled to date; on track to complete enrollment in third quarter of 2020
Dermavant Sciences, of Long Beach, Calif., and Basel, Switzerland, a subsidiary of Roivant Sciences Ltd. Tapinarof (DMVT-505) Therapeutic aryl hydrocarbon receptor modulating agent topical cream Plaque psoriasis Phase IIb data published in The Journal of the American Academy of Dermatology showed treatment at 1% QD demonstrated improvement in PASI75 as early as week 2, which was statistically significant starting at week 8 and maintained for 4 weeks after treatment discontinuation (through week 16) vs. vehicle QD (n=20) (p<0.001 at week 12); PASI90 response also showed statistically significant efficacy at week 12 and maintenance of effect at week 16 vs. vehicle QD (n=20) (p=0.001 at week 12); significantly higher proportion of patients treated with tapinarof 1% QD reported very or moderately improved psoriasis symptoms (88%) (p<0.001) and psoriasis-related pruritus (76%) (p=0.006) compared with those receiving vehicle QD (35%) at week 12
Synairgen plc, of Southampton, U.K. SNG-001 Inhaled formulation of interferon-beta-1a COVID-19 Launched expansion of SG016 trial to dose patients in home setting, aiming to dose those earlier in the illness, within 3 days of symptoms developing
Phase III
Acadia Pharmaceuticals Inc., of San Diego Pimavanserin Selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors Major depressive disorder Plans to combine Clarity-2 and Clarity-3 studies testing drug as adjunctive treatment into 1 study with prespecified statistical analysis plan; as a result, no new patients will be enrolled in the 2 identically designed trials, each of which will be concluded with slightly more than 50% enrollment; top-line data expected in third quarter of 2020
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Onpattro (patisiran) RNAi therapeutic Polyneuropathy of hereditary ATTR amyloidosis New results from open-label extension (OLE) study showed reductions in serum TTR levels were maintained in patisiran-treated patients; patients on treatment for 42 months demonstrated sustained improvement in neuropathy impairment and quality of life relative to Apollo study baseline, as shown by mean negative changes in modified Neuropathy Impairment Score + 7 (mNIS+7) and Norfolk Quality of Life – Diabetic Neuropathy scores; patients on treatment from the phase II OLE population also demonstrated improvement in mNIS+7 score over 48 months
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Onpattro (patisiran) RNAi therapeutic hATTR amyloidosis with disease progression after orthotopic liver transplant Interim phase IIIb data from 23 patients who showed disease progression post-OLT showed, after 3 weeks of patisiran treatment, mean reduction from baseline in serum TTR levels was 81.9%
Argenx NV, of Breda, the Netherlands Efgartigimod Antibody fragment designed to reduce disease-causing IgG antibodies and block the IgG recycling process Myasthenia gravis Top-line data showed pivotal Adapt trial met primary endpoint defined as percentage of responders on the Myasthenia Gravis Activities of Daily Living score among acetylcholine receptor-antibody positive generalized MG patients; Argenx plans to submit BLA to FDA by the end of 2020
Eli Lilly and Co., of Indianapolis Emgality (galcanezumab-gnlm) CGRP-targeting monoclonal antibody Migraine Treatment significantly improved work productivity and reduced interictal burden, defined as health and well-being between migraine attacks, in an analysis of 3-month double-blind period of Conquer study
Glenmark Pharmaceuticals Ltd., of Mumbai, India Favipiravir and umifenovir Antiviral drugs COVID-19 Started open-label Faith study to test combination treatment in hospitalized patients with moderate COVID-19 infection
Janssen Research & Development LLC, a unit of New Brunswick, N.J.-based Johnson & Johnson Selexipag  Oral prostacyclin receptor agonist Pulmonary arterial hypertension Decision made April 1, 2020, to suspend screening in study of children with PAH due to COVID-19
Mesoblast Ltd., of Melbourne, Australia Ryoncil (remestemcel-L) Allogeneic mesenchymal stem cells Steroid-refractory acute graft-vs.-host disease Results from 3 trials in children and adults published in May issue of Biology of Blood and Marrow Transplantation included data from Study 275 in 241 children, showing day 28 overall response (OR) in 65% of subjects; study GVHD001/002 in 55 children showed day 28 OR achieved in 70%, statistically significant compared to prespecified control value of 45% (p=0.0003); study 280 showed, among high-risk children and adults, day 28 OR was significantly greater in Ryoncil group (58% vs. 37%, p=0.03)
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Dupixent (dupilumab) Fully human monoclonal antibody targeting interleukin-4 (IL-4) and IL-13 proteins Eosinophilic esophagitis Results from part A of pivotal trial in patients 12 and older met both co-primary endpoints, as well as all key secondary endpoints; an ongoing part B portion of the study is evaluating additional dosing regimen
Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel Ajovy (fremanezumab) CGRP-targeting monoclonal antibody Migraine Pooled analyses of Focus, Halo-EM and Halo-CM trials in patients 60 or older with episodic migraine or chronic migraine showed reductions from baseline in monthly migraine days, headache days of at least moderate severity, and days with acute headache medication use over 12 weeks were significantly greater with quarterly and monthly fremanezumab vs. placebo (all p≤0.0103)


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