Company Product Description Indication Status
Phase I
Blackthorn Therapeutics Inc., of San Francisco BTRX-335140 Kappa opioid receptor antagonist Depression and other central nervous system disorders Shown to engage receptors in the brain at levels that are projected to be effective in treating relevant symptoms
Faron Pharmaceuticals Oy, of Turku, Finland Clevegen Cancer immunotherapy targeting Clever-1 positive tumor associated macrophages  Metastatic or inoperable solid tumors Shown to down-regulate a range of major immuno-oncology checkpoints
Eicosis LLC, of Davis, Calif. EC-5026 Inhibits soluble epoxide hydrolase Pain management Dosed first patient
Exicure Inc., of Skokie, Ill. AST-008 TLR-9 agonist Solid tumors Preliminary data from ongoing phase Ib/II trial showed study drug, alone and in combination with pembrolizumab (Keytruda, Merck & Co. Inc.), produced cytokine and chemokine expression and immune cell activation in patient blood; of 4 participants with Merkel cell carcinoma, 1 who previously progressed on anti-PD-1 therapy has confirmed stable disease with decreased target lesion diameters for > 12 weeks and 1 had target lesion complete response and confirmed overall partial response > 24 weeks; 9 participants had progressive disease, 2 had not been evaluated and 1 was not evaluable
Hepion Pharmaceuticals Inc., of Edison, N.J. CRV-431 Hepatitis B structural protein inhibitor; peptidyl-prolyl cis-trans isomerase A inhibitor Nonalcoholic steatohepatitis Multiple ascending-dose trial escalated to next dosing level of 150 mg daily for 28 days
Oncternal Therapeutics Inc., of San Diego TK-216 ETS transcription factor inhibitor Ewing sarcoma Opened phase Ib expansion cohort of ongoing trial to evaluate recommended phase II dose regimen (200 mg/m2/day for 14 days) in combination with vincristine in relapsed/refractory disease
Proqr Therapeutics NV, of Leiden, the Netherlands QR-1123 RHO gene modulator Retinitis pigmentosa First of about 35 adults with autosomal dominant disease due to P23H mutation in rhodopsin gene dosed in phase I/II Aurora trial; improvement of visual function and retinal structure will be assessed through visual acuity, visual field and optical coherence tomography; initial data expected in 2021
Synlogic Inc., of Cambridge, Mass. SYNB-1618 Synthetic biotic Phenylketonuria Data from bridging study in healthy volunteers identified maximum tolerated dose of 2 x 1012 live cells (5.3 x 1011 colony forming units) and showed that dose ramp improved tolerability and that pH buffering was required for maximum Phe-consuming activity of the strain
Y-Mabs Therapeutics Inc., of New York Naxitamab Anti-GD2 3F8 monoclonal antibody Neuroblastoma and other solid tumors Investigator-sponsored front-line study of 34 participants with high-risk stage 4 neuroblastoma in first complete remission showed 72% event-free survival at 24 months compared to 63% reported at 24 months for population of 89 individuals treated with competing antibody; 20% of those treated with study drug received bone marrow transplant compared with all treated with competing anti-GD2 antibody; overall survival for study drug was 86% at 24 months compared to 84% for competing antibody
Phase II
Astrazeneca plc, of Cambridge, U.K., and Daiichi Sankyo Co. Ltd., of Tokyo [Fam]-trastuzumab deruxtecan (DS-8201) HER2-targeting antibody-drug conjugate Metastatic breast cancer Pivotal Destiny-Breast01 monotherapy (5.4 mg/kg) trial in 184 individuals with HER2-positive disease achieved objective response rate, the primary endpoint, of 60.9%; disease control rate was 97.3% with median duration of response of 14.8 months (range: 13.8 - 16.9) and median progression-free survival of 16.4 months (range: 12.7 - not reached); median overall survival was not reached, with estimated survival rate of 86% at 1 year; data published in The New England Journal of Medicine 
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-701 (talabostat) Dual dipeptidyl peptidase VIII/IX inhibitor; seprase inhibitor Advanced solid tumors Open-label basket trial will evaluate study drug in combination with pembrolizumab (Keytruda, Merck & Co. Inc.); efficacy measures include progression-free survival, overall survival and duration of response
Gemvax & Kael Co. Ltd., of Seoul, South Korea GV-1001 (tertomotide) Telomerase modulator Alzheimer's disease Combination study with donepezil in participants with moderate to severe AD met primary endpoint, measured by Severe Impairment Battery score, which decreased by 0.12 compared to decrease of 7.23 for donepezil-only control group
Viela Bio Inc., of Gaithersburg, Md. VIB-4920 CD40 ligand receptor antagonist Sjögren’s syndrome First of 174 participants dosed in phase IIb trial; primary outcome measures are change from baseline in European League Against Rheumatism (EULAR) Sjogren's syndrome disease activity index (ESSDAI) at day 169 in population 1 (moderate to high systemic disease activity defined by EULAR ESSDAI of >= 5) and in population 2 (moderate to severe subjective symptoms defined by EULAR Sjogren's Syndrome Patient Reported Index score >/= 5 and residual stimulated salivary flow but mild systemic disease activity defined by ESSDAI score < 5)
Xeris Pharmaceuticals Inc., of Chicago Glucagon Glucagon ligand Hypoglycemia In-clinic stage of study assessing ready-to-use (RTU) glucagon for postprandial episodes following bariatric surgery found that all individuals with successful meal challenge were able to self-administer minidose of study drug as directed during declining blood glucose to restore or maintain normal blood glucose levels within 15 minutes; effect was maintained at 30 minutes and hyperglycemia was not observed; incidence of follow-on episode of hypoglycemia requiring oral glucose for rescue was less with RTU glucagon than placebo
Phase III
Cstone Pharmaceuticals Ltd., of Suzhou, China, and Blueprint Medicines Corp., of Cambridge, Mass. Avapritinib Oral selective inhibitor of KIT and PDGFRA Advanced gastrointestinal stromal tumors Completed target patient enrollment in China and reached Voyager trial enrollment target globally; study testing drug as third- or fourth-line treatment vs. regorafenib
Diffusion Pharmaceuticals Inc., of Charlottesville, Va. TSC Trans sodium crocetinate Glioblastoma multiforme Data showed increased survival in inoperable patients enrolled in 19-patient lead-in portion of Intact study in combination with standard of care
Eli Lilly and Co., of Indianapolis Selpercatinib (LOXO-292) RET tyrosine kinase receptor inhibitor Non-small-cell lung cancer Opened Libretto-431 study, expected to enroll 400 individuals with advanced or metastatic treatment-naïve RET fusion-positive disease, randomized 1-to-1 to study drug or platinum-based and pemetrexed therapy with or without pembrolizumab (Keytruda, Merck & Co. Inc.); primary endpoint is progression-free survival and secondary endpoints include overall survival, overall response rate, duration of response and intracranial ORR
Iterum Therapeutics plc, of Dublin Sulopenem  Oral and I.V. penem antibiotic  Complicated intra-abdominal infections Top-line results from Sure 3 trial showed the difference in clinical response at day 28 in micro-modified intent-to-treat population was 4.7% with a 95% confidence interval of -10.3% to 1%, just missing the noninferiority requirement set by the FDA that the lower limit of the difference in the outcome rates be >-10% 
Leo Pharma A/S, of Ballerup, Denmark Tralokinumab Monoclonal antibody targeting IL-13 Moderate to severe atopic dermatitis Met all primary and secondary endpoints in 3 pivotal Ecztra studies in adults; marketing applications planned for 2020
Macrogenics Inc., of Rockville, Md. Margetuximab Monoclonal antibody targeting HER2 oncoprotein HER2-positive metastatic breast cancer Updated Sophia results showed overall survival data favored margetuximab in combination with chemotherapy vs. trastuzumab/chemotherapy in patients previously treated with HER2-targeted therapies in intention-to-treat population; however, those data did not reach statistical significance at second interim analysis as of September 2019 cutoff after 270 events (median OS=21.6 months vs. 19.8 months; p=0.326); final prespecified OS analysis planned after 385 events have accrued, which is projected in second half of 2020
Roche Holding AG, of Basel, Switzerland Perjeta (pertuzumab) HER2-targeting antibody HER2-positive early breast cancer Data from second interim overall survival analysis of Aphinity study in combination with Herceptin (trastuzumab) and chemotherapy, conducted after median follow-up of about 74 months, showed Perjeta-based regimen reduced risk of recurrence or death by 24% vs. Herceptin/chemotherapy/placebo; at 6 years, 90.6% of patients in Perjeta arm have not seen their cancer return vs. 87.8% in placebo arm, for an absolute benefit of 2.8%
Seattle Genetics Inc., of Bothell, Wash. Tucatinib Tyrosine kinase inhibitor selective for HER2 without significant EGFR inhibition  HER2-positive metastatic breast cancer Data from pivotal HER2Climb study, published in The New England Journal of Medicine, showed addition of tucatinib to trastuzumab and capecitabine improved overall survival, reducing risk of death by 34% compared to trastuzumab and capecitabine alone; primary endpoint of progression-free survival showed addition of tucatinib was superior to trastuzumab and capecitabine alone, with a 46% reduction in risk of disease progression or death (p<0.00001); estimated PFS at 1 year was 33% in tucatinib arm vs. 12% in trastuzumab/capecitabine-only arm, and median PFS was 7.8 months and 5.6 months, respectively 


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