Company Product Description Indication Status
Phase I
Curis Inc., of Lexington, Mass. CA-4948 IRAK4 kinase inhibitor Relapsed or refractory non-Hodgkin lymphoma, including patients with diffuse large B-cell lymphoma, Waldenström's macroglobulinemia and oncogenic MYD88 mutations 5 of the 6 patients evaluable for anticancer activity at the 2 highest dose levels of CA-4948 have experienced reduced tumor burden
Curis Inc., of Lexington, Mass. Fimepinostat Small-molecule dual inhibitor of PI3K/HDAC Relapsed or refractory diffuse large B-cell lymphoma, including patients with double-hit/double-expressor lymphoma Study testing the drug with venetoclax (Venclexta, Abbvie Inc./Roche Holding AG) has enrolled 11 patients, 6 patients in the first cohort and 5 patients in the second cohort; favorable safety profile so far
Innovent Biologics Inc., of Suzhou, China IBI-110 Anti-lymphocyte activation gene 3 recombinant fully human monoclonal antibody Advanced malignancies First patient dosed
Sunesis Pharmaceuticals Inc., of South San Francisco Vecabrutinib BTK inhibitor Relapsed/refractory chronic lymphocytic leukemia and other B-cell malignancies Clinical benefit observed in covalent BTK inhibitor-resistant patients with poor prognostic features and in both wild-type and C481-mutated BTK disease; in the 300-mg dose group (cohort 5), stable disease was observed in 3 of 5 patients, and 2 remain on treatment in cycle 5 and cycle 6, including a wild-type BTK patient with -40% change in tumor burden at first scan
Phase II
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. ARQ-154 PDE4 inhibitor Seborrheic dermatitis First of about 150 participants enrolled; primary endpoint is achievement of Investigator Global Assessment Scale score of clear or almost clear plus 2-grade improvement from baseline at week 8
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Troriluzole (riluzole prodrug) Glutamate modulator Alzheimer's disease Phase II/III T2 Protect AD study of individuals with mild to moderate AD to continue unchanged after completing preplanned interim futility analysis of first 100 participants following 6 months of treatment
Cassava Sciences Inc. (formerly Pain Therapeutics Inc.), of Austin, Texas PTI-125 Filamin A modulator Alzheimer's disease Additional data from phase IIa study showed improvements in biomarkers of AD in plasma and lymphocytes and consistency across biomarker improvements in CSF, plasma and lymphocytes; reductions (p<0.01) were seen in nitrated and phosphorylated forms of tau protein
EIP Pharma Inc., of Boston Neflamapimod p38 MAP kinase alpha inhibitor Early stage Alzheimer's disease No difference seen in Reverse-SD study between neflamapimod and placebo groups in primary efficacy endpoint of combined change from baseline to week 24 in z-scores of Hopkins Verbal Learning Test total and delayed recall; statistically significant effects of study drug vs. placebo seen in change from baseline to week 24 in CSF protein levels of p-tau181 (p=0.012 vs. placebo; mean difference=- 2 pg/mL, 95% CI: -3.6, -0.5) and total tau (p=0.03; mean difference=-18.9 pg/mL, 95% CI: -36.0, -1.8)
Galapagos NV, of Mechelen, Belgium GLPG-1690 Autotaxin inhibitor Diffuse cutaneous systemic sclerosis Novesa trial completed enrollment of 33 participants; top-line results expected in second half of 2020
Galera Therapeutics Inc., of Malvern, Pa. GC-4419 (avasopasem manganese) Superoxide dismutase stimulator Oral mucositis  Results from phase IIb trial in 223 participants with advanced head and neck cancer, reported in June 2018, published in Journal of Clinical Oncology 
Rocket Pharmaceuticals Inc., of New York RP-L102 FANCA gene stimulator Fanconi anemia First of 12 expected participants dosed in registration-enabling "Process B" study; primary endpoint and potential surrogate endpoint for accelerated approval is improved mitomycin-C resistance in bone marrow colony forming cells 
Phase III
Fergene LLC, subsidiary of Ferring Pharmaceuticals SA, of Saint-Prex, Switzerland Nadofaragene firadenovec
IFNA2 gene stimulator
Bladder cancer Study in 157 participants with high-grade non-muscle invasive disease met primary endpoint with 53% achieving complete response (CR) at 3 months and 24% continuing to show CR at 12 months; additional data showed 73% high-grade recurrence-free (HGRF) survival in those with papillary disease at 3 months and 44% HGRF survival at 12 months
Intercept Pharmaceuticals Inc., of New York Obeticholic acid Farnesoid X receptor agonist Fibrosis Results from interim analysis of ongoing Regenerate study in participants with nonalcoholic steatohepatitis, reported in February, published in The Lancet; data served as basis for NDA, accepted by FDA in November
Novartis AG, of Basel, Switzerland QMF-148 Once-daily, fixed-dose combination of indacaterol acetate and mometasone furoate Asthma Data from 52-week Palladium trial showed superiority to mometasone furoate (MF) at medium and high doses in improving lung function, meeting primary endpoint; statistically significant superiority vs. MF alone also demonstrated in key secondary endpoint of improvement in asthma control; other secondary analyses showed improvements in lung function when comparing IND/MF to a LABA/ICS standard of care (salmeterol xinafoate/fluticasone propionate)
UCB SA, of Brussels, Belgium Bimekizumab IL-17A and IL-17F inhibitor Moderate to severe plaque psoriasis Results from Be Sure study met co-primary endpoints at week 16, demonstrating superiority to adalimumab in achieving at least 90% improvement in Psoriasis Area and Severity Index (PASI 90) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1); met all ranked secondary endpoints with statistical significance, including superior total skin clearance at weeks 16 and 24, as measured by PASI 100, vs. adalimumab; bimekizumab was statistically superior to adalimumab in achieving rapid response, defined as PASI 75 at week 4


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