Company Product Description Indication Status
Phase I
Epicentrx Inc., of La Jolla, Calif. RRx-001 Small-molecule immunotherapy that targets the CD47-SIRPα axis Newly diagnosed glioblastoma RRx-001 combination therapy (radiotherapy + temozolomide) led to overall survival of 21.9 months, compared with historical OS data of 15-20 months; the RRx-001 combo also led to progression-free survival of 13 months, compared with historical PFS data of 6-7 months
Oyster Point Pharma Inc., of Princeton, N.J. Varenicline Nicotinic acetylcholine receptor agonist Dry eye disease Top-line results indicate the relative bioavailability (systemic exposure as defined by adjusted geometric mean AUC0-inf) was 13 times lower for single dose of highest strength of OC-01 nasal spray as compared to single dose of highest strength Chantix (varenicline, Pfizer Inc.) tablet (7.46 vs. 99.67 h*ng/ml)
TFF Pharmaceuticals Inc., of Austin, Texas Voriconazole inhalation powder Triazole antifungal Pulmonary aspergillosis Trial begun
Ziopharm Oncology Inc., of Phoenix Ad-RTS-hIL-12 plus veledimex Targets IL-12 Recurrent or progressive glioblastoma multiforme Interim analysis from ongoing substudy in combination with low-dose dexamethasone showed decrease in tumor from baseline, resulting in patient’s lesion being too small to measure, assessed as partial response, with follow-up ongoing; data from ongoing substudy in combination with anti-PD-1 drug Opdivo (nivolumab, Bristol-Myers Squibb Co.) showed decrease by 64% in patient’s tumor from baseline, resulting in partial response, with follow-up ongoing
Phase II
Agios Pharmaceuticals Inc., of Cambridge, Mass. Vorasidenib and Tibsovo (ivosidenib) Oral inhibitor of mutant IDH1 and IDH2 enzymes and IDH1 inhibitor Low-grade glioma Data from both single-agent cohorts of perioperative study show median postoperative treatment duration of all doses was 5.42 (0.9–13.5) months for vorasidenib and 6.93 (1–13.2) months for Tibsovo; both demonstrated brain penetrance, with mean brain:plasma ratios of 3.16 (vorasidenib 10 mg), 1.74 (vorasidenib 50 mg), 0.13 (Tibsovo 250 mg) and 0.10 (Tibsovo 500 mg); mean percent reduction in 2-HG was 92.6% and 91.1%, for vorasidenib 50 mg and Tibsovo 500 mg, respectively, relative to untreated samples; among those postoperatively treated with 50 mg vorasidenib or 500 mg Tibsovo, 4 of each group (31% for both) achieved objective tumor responses
Beigene Ltd., of Beijing Tislelizumab Anti-PD-1 antibody Gastric/gastroesophageal junction adenocarcinoma or esophageal squamous cell carcinoma Updated results in combination with chemotherapy showed, at cutoff, 7 (46.7%) with G/GEJ adenocarcinoma achieved confirmed partial response, and objective response rate was 46.7%; in ESCC cohort, 7 (46.7%) achieved confirmed PR and ORR was 46.7%; median duration of response was not mature in G/GEJ cohort and was estimated as 12.8 months in ESCC cohort; median progression-free survival was 6.1 months and 10.4 months, respectively
Cymabay Therapeutics Inc., of Newark, Calif. Seladelpar PPAR-delta agonist Nonalcoholic steatohepatitis Terminating phase IIb study after planned, blinded histological assessments of first tranche of liver biopsies revealed atypical histological findings, including histology characterized as interface hepatitis presentation, with or without biliary injury; also halted study in primary sclerosing cholangitis
Delmar Pharmaceuticals Inc., of San Diego VAL-083 Small-molecule chemotherapy Glioblastoma multiforme For the 22 patients who completed at least 1 cycle of treatment as of Nov. 2, median progression-free survival was 9.9 months; for 18 patients initially receiving intended treatment dose (30 mg/m2/day on days 1, 2 and 3 of a 21-day cycle) median PFS was 10.4 months
Dnatrix Inc., of Houston DNX-2401 (tasadenoturev) Oncolytic adenovirus Recurrent glioblastoma A total of 48 patients treated with DNX-2401, followed by pembrolizumab (Keytruda, Merck & Co. Inc.), with majority experiencing clinical benefit, including durable complete and partial responses; interim median overall survival of 12.3 months
Emergent Biosolutions Inc., of Gaithersburg, Md. Chikungunya vaccine  Virus-like particle vaccine  Chikungunya virus Interim analysis showed after first dose, up to 98% of participants produced neutralizing antibody response against the virus within 7 days after vaccination
Erytech SA, of Lyon, France Eryaspase L-asparaginase encapsulated inside donor-derived red blood cells Metastatic pancreatic cancer Phase IIb data published in the European Journal of Cancer showed combination with chemotherapy significantly prolonged both overall survival and progression-free survival in entire patient population, with a 40% reduction in risk of death (OS p=0.008) and a 44% reduction in risk of disease progression on average over time (PFS p=0.005)
Immunovant Inc., of New York IMVT-1401 Monoclonal antibody targeting the neonatal Fc receptor Moderate to severe active Graves’ ophthalmopathy Started 77-patient Ascend-GO 2 study comparing 3 doses of the drug to placebo; primary efficacy endpoint is the proptosis responder rate measured at week 13, defined as the percentage of patients with a > 2 mm reduction in proptosis in the study eye without deterioration in the fellow eye; top-line data expected in early 2021
Kazia Therapeutics Ltd., of Sydney GDC-0084  PI3K inhibitor  Glioblastoma First 8 evaluable patients had a median progression-free survival of 8.4 months; 75% of evaluable patients are alive, so median overall survival can’t be calculated; maximum tolerated dose of 60 mg
La Jolla Pharmaceutical Co., of San Diego LJPC-401 (synthetic human hepcidin)  Peptide hormone Iron overload in beta-thalassemia LJ401-BT01 study is being stopped after an interim analysis of half of the targeted 100 patients found no significant differences between drug and the control in the primary or key secondary endpoints
La Jolla Pharmaceutical Co., of San Diego LJPC-401 (synthetic human hepcidin)  Peptide hormone Hereditary hemochromatosis In the LJ401-HH01 study, patients taking drug had a mean reduction in TSAT of 33% at 16 weeks compared a mean reduction of 3% for placebo (p<0.0001); LJPC-401-treated patients had 0.10 phlebotomies per month compared to 0.50 phlebotomies per month for placebo (p<0.0001)
Vaccitech Ltd., of Oxford, U.K. VTP-100 Universal influenza A vaccine Influenza prophylaxis Completed recruitment and vaccination for the FLU009 and FLU010 studies; interim analysis of FLU009 and final data from FLU010 expected in early 2020
Phase III
Akcea Therapeutics Inc., of Boston, and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. AKCEA-TTR-Lrx Antisense targeting transthyretin  Polyneuropathy caused by hereditary TTR amyloidosis Started Neuro-TTRansform study of 140 patients; 20 patients will start on Tegsedi (inotersen) for 35 weeks; the co-primary efficacy endpoints at week 66, percent change from baseline in serum TTR concentration, change from baseline in the modified Neuropathy Impairment Score +7 and change from baseline in Norfolk Quality of Life Questionnaire-Diabetic Neuropathy, will be compared to placebo group from the NEURO-TTR study of Tegsedi
Avadel Pharmaceuticals plc, of Dublin FT-218 Controlled-release sodium oxybate Excessive daytime sleepiness and cataplexy in patients with narcolepsy Reached target enrollment of 205 patients in the Rest-On study; top-line data expected in the second quarter of 2020
Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. Edaravone (MT-1186) Oral suspension formulation of neuroprotective agent Amyotrophic lateral sclerosis Started study testing long-term safety and tolerability
Pharmazz Inc., of Willowbrook, Ill. Sovateltide  Endothelin-B receptor agonist Acute cerebral ischemic stroke Study has enrolled 7 of approximately 110 patients; top-line data expected by the middle of 2020
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan TAK-003  Dengue vaccine Dengue prophylaxis Updated 18-month data from the Tides study showed overall vaccine efficacy was 73.3% compared to 80.2% in the primary endpoint analysis at 12 months
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Alunbrig (brigatinib) ALK inhibitor Advanced anaplastic lymphoma kinase-positive non-small-cell lung cancer After more than 2 years of follow-up in the Alta-1L study, Alunbrig reduced the risk of disease progression or death by 76% compared to Xalkori (crizotinib, Pfizer Inc.) in newly diagnosed patients whose disease had spread to the brain at time of enrollment; Alunbrig reduced the risk of disease progression or death in all patients by 57%
Tocagen Inc., of San Diego Toca 511 & Toca FC Vocimagene amiretrorepvec and extended-release formulation of 5-fluorocytosine Recurrent high-grade glioma Study didn’t meet its primary or secondary endpoints; in a preplanned subgroup analysis of 60 patients with second recurrence, Toca 511 and Toca FC produced a 57% risk reduction for death compared to standard of care (lomustine, temozolomide or bevacizumab) with a median overall survival of 21.82 months compared to 11.14 months


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