Company Product Description Indication Status
Phase I
4D pharma plc, of Leeds, U.K.
Single strain live biotherapeutic product Advanced solid tumors Additional interim data from first 6 participants in part A of ongoing phase I/II combination trial with anti-PD-1 Keytruda (pembrolizumab, Merck & Co. Inc.) showed 2 partial responses with evidence of tumor shrinkage who remain on study (1 for > 10 months) and 1 with stable disease who remains on study for > 8 months; evidence of increased tumor-infiltrating lymphocytes seen following therapy
Arch Biopartners Inc., of Toronto Metablok LSALT peptide Prevention of organ inflammation Completed dosing of final 8 healthy volunteers, which received highest daily dose tested for 3 consecutive days; met primary endpoints of safety and tolerability 
Eton Pharmaceuticals Inc., of Deer Park, Ill. ET-101 (topiramate oral solution) GABA receptor agonist; NMDA receptor antagonist; sodium channel inhibitor Partial-onset/primary generalized tonic-clonic seizures Bioequivalence study in healthy males under fasting conditions showed 90% confidence intervals for pharmacokinetic parameters were within 80% to 125% of criteria vs. FDA-approved oral solid comparator product; food effect study conducted under fed conditions showed bioavailability unaffected
Inxmed (Shanghai) Co. Ltd., of Shanghai IN-10018 Focal adhesion kinase inhibitor Metastatic uveal, NRAS-mutant melanoma First participant dosed in phase Ib trial as monotherapy and in combination with MEK inhibitor cobimetinib (Roche Holding AG) that will include 6 sites in U.S. and 3 in Australia
Lattice Biologics Ltd., of Belgrade, Mont. Amnioboost Amniotic fluid-derived stem cell therapy Severe acute respiratory syndrome First of 10 participants with laboratory confirmed COVID-19 infection and evidence of lung involvement requiring supplemental oxygen or mechanical ventilation enrolled in placebo-controlled study in Seattle; therapy involves ~ 5M Amnioboost stem cells on day 1 of enrollment and another ~ 5 million on day 2; daily scores based on scale of clinical outcomes for treatment and placebo groups will be assessed on day 7 and study halted if no evidence of treatment difference
Recce Pharmaceuticals Ltd., of Sydney RECCE-327  Bactericidal permeability/outer membrane protein inhibitor Bacterial infection Execution of agreement for independent intravascular safety assessment at Australian-based trial facility expected to be finalized in first quarter of 2020; discussions underway with Australian teaching hospital to initiate independent phase I/II topical efficacy study in bacterial wound, burn and skin infections
Surface Oncology Inc., of Cambridge, Mass. SRF-617 CD39 antagonist Advanced solid tumors Phase I/Ib dose-escalation study initiated with combination arms of gemcitabine and Abraxane, anti-PD-1 or A2A/A2B small-molecule inhibitor AB-928 (Arcus Biosciences Inc.); planned cohorts will focus on pancreatic and gastric cancer and tumors showing resistance to anti-PD-1 therapy; biopsy expansion cohort designed to provide data on changes in tumor tissue CD39 enzymatic activity related to treatment; initial clinical update expected by year-end 2020
Phase II
Blueprint Medicines Corp., of Cambridge, Mass. Ayvakit (avapritinib) KIT tyrosine kinase inhibitor; PDGF receptor alpha antagonist Indolent systemic mastocytosis Part 1 of Pioneer trial in 39 participants showed statistically significant mean decline of ~30% in total symptom score at 16 weeks, measured by Indolent SM Symptom Assessment Form, across all dose cohorts (25 mg, 50 mg and 100 mg) vs. ~3% in placebo cohort (p=0.001); reductions in scores deepened over time and, at cutoff date, 37 participants remained on study with median follow-up of 18 weeks; declines also seen in serum tryptase, bone marrow mast cells and KIT D816V allele burden and improvements in patient-reported quality of life; 25 mg once daily selected as part 2 dose
Inventiva SA, of Daix, France Lanifibranor Oral small molecule that actives all 3 PPAR isoforms Nonalcoholic steatohepatitis Last visit of last patient out of 247 enrolled in phase IIb trial; publication of headline results expected in June 2020
Phase III
Abeona Therapeutics Inc., of New York EB-101 COL7A1 gene stimulator Recessive dystrophic epidermolysis bullosa First of up to 15 participants treated in pivotal Viital study assessing effectiveness in about 30 large, chronic wound sites; primary outcome measure is wound healing vs. untreated wound sites on same patients; secondary endpoints include assessments of pain and patient-reported outcomes
Adial Pharmaceuticals Inc., of Charlottesville, Va. AD-04 (repurposed ondansetron) 5-HT 3 receptor antagonist Alcohol use disorder Pivotal trial in genetically targeted patients modified to protect participants during COVID-19 pandemic, including reducing number of in-person visits to clinical sites, replacing some visits with telemedicine behavioral treatments and assessments, removing non-essential elements from in-person assessments to reduce visit duration
Astrazeneca plc, of Cambridge, U.K. Imfinzi (durvalumab) PD-L1 inhibitor Small-cell lung cancer Top-line data from final analysis of Caspian combination trial with choice of standard-of-care (SoC) chemotherapies showed sustained overall survival (OS) for extensive-stage disease in first-line setting; second arm testing anti-CTLA4 monoclonal antibody tremelimumab with Imfinzi and SoC did not meet primary endpoint of statistically significant OS improvement 
Merck & Co. Inc., of Kenilworth, N.J. Gefapixant (MK-7264) Selective P2X3 receptor antagonist Refractory or unexplained chronic cough  Top-line results from 2 trials showed primary efficacy endpoints were met for the gefapixant 45-mg twice-daily treatment arms, demonstrating statistically significant decrease in 24-hour coughs per hour vs. placebo at 12 and 24 weeks; 15-mg twice-daily treatment arms did not meet primary efficacy endpoint in either study
Oncosec Medical Inc., of San Diego Tavo (tavokinogene telseplasmid) Plasmid-based interleukin-12 delivered via electroporation gene delivery system Metastatic melanoma  Data published in the Annals of Oncology showed the objective overall response rate was 35.7% in the main study, with a complete response rate of 17.9%; the median progression-free survival in the main study was 3.7 months while the median overall survival was not reached at a median follow-up of 29.7 months
Provention Bio Inc., of Oldwick, N.J. PRV-031 (teplizumab) CD3 antagonist Type 1 diabetes Paused Protect study due to COVID-19 pandemic


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