Company Product Description Indication Status
Phase I
Kancera AB, of Stockholm KAND-567  Fractalkine ligand inhibitor Acute myocardial infarction Immunological analysis of blood samples from healthy participants in ongoing phase Ib program showed study drug blocked immune cells known to cause acute and chronic inflammatory diseases; effect on clinically relevant biomarkers provides additional support for potential cardiovascular protective effect
Lannett Co. Inc., of Philadelphia Insulin glargine biosimilar Insulin ligand; insulin receptor agonist Type 1 and 2 diabetes In 27 healthy volunteers, single subcutaneous dose matched reference biologic Lantus (Sanofi SA), meeting primary endpoints of pharmacokinetics and pharmacodynamics safety
Lixte Biotechnology Holdings Inc., of East Setauket, N.Y.  LB-100 Protein phosphatase 2A inhibitor Glioblastoma NCI enrolled first 2 of 8 planned participants in study assessing ability of LB-100 to enter brain and penetrate recurrent tumors when surgical removal of cancers is indicated
Moleculin Biotech Inc., of Houston Annamycin Lipophilic anthracycline derivative Acute myeloid leukemia Of 10 participants treated and evaluated at or above 120 mg/m2, interim results showed 1 complete response with incomplete recovery of white blood cells and/or platelets and 2 partial responses, where bone marrow blasts were reduced 50% to below 25%; 1 additional participant was bridged to bone marrow transplant
Phase II
Anavex Life Sciences Corp., of New York ANAVEX22-73 (blarcamesine) Sigma-1 receptor agonist Alzheimer's disease Interim 2-year data from phase IIa extension study, with participants followed for up to 5 years, showed high-dose cohort had lower Mini Mental State Examination decline (-1.1) compared to matched control cohort from Alzheimer’s Disease Neuroimaging Initiative study (-4.4) at week 104 (p<0.01)
Cerevance Inc., of Boston CVN-424 Non-dopaminergic protein modulator Parkinson's disease  Initiated trial expected to enroll 70 participants with motor fluctuations on levodopa; efficacy endpoints include reduction in off time and functional outcome measures
Ra Pharmaceuticals Inc., of Cambridge, Mass. Zilucoplan Complement C5 factor inhibitor Immune-mediated necrotizing myopathy Initiated dosing in trial expected to enroll about 24 participants positive for anti-3-hydroxy-3-methylglutaryl-coenzyme A reductase or anti-signal recognition particle autoantibodies; participants will be randomized 1-to-1 to daily subcutaneous doses of 0.3 mg/kg zilucoplan or placebo, with primary endpoint of change in creatine kinase from baseline to week 8
Phase III
Aveo Oncology, a unit of Aveo Pharmaceuticals Inc., of Cambridge, Mass. Tivozanib (Fotivda) VEGF receptor tyrosine kinase inhibitor Renal cell carcinoma Data from TIVO-3 study, reported in September, were published in The Lancet Oncology 
Omeros Corp., of Seattle Narsoplimab Complement cascade inhibitor; MASP-2 inhibitor Hematopoietic stem cell transplant-associated thrombotic microangiopathy In pivotal trial, 56% who received at least 1 dose achieved complete responder status, meeting FDA-agreed response criteria; 68% who received protocol-specified dosing of at least 4 weeks achieved complete responder status; 100-day survival following diagnosis was 65% for those who received at least 1 dose, 81% for those who received at least 4 weeks of dosing and 93% in complete responder group; statistically significant (p<0.01) improvements in platelet count, LDH and haptoglobin seen across all treated groups
Pluristem Therapeutics Inc., of Haifa, Israel PLX-PAD Allogeneic cells Muscle regeneration Completed enrollment of 50% of planned 240 patients; study treating muscle injury following arthroplasty for hip fracture, as compared to placebo
Rexgenero Ltd., of London REX-001 (rexmyelocel-T) Autologous bone marrow-derived mature mononuclear cell therapy Chronic limb ischemia First U.K. trial site opened in 2 Salamander trials evaluating 60 participants with CLI and rest pain and 78 with CLI and non-healing ischemic ulcers 
Soligenix Inc., of Princeton, N.J. SGX-301 Synthetic hypericin Cutaneous T-cell lymphoma Completed enrollment in Flash study; top-line results expected in first quarter of 2020
Zealand Pharma A/S, of Copenhagen Dasiglucagon Glucagon analogue Congenital hyperinsulinism Trial opened to evaluate drug in up to 12 children with CHI ages 7 days to 1 year; endpoints include reduction of intravenous glucose infusion rate, number of hypoglycemic events and total amount of carbohydrate administered
Zogenix Inc., of Emeryville, Calif. Fintepla (ZX-008, fenfluramine oral solution) Inhibits activity of glutamate N-methyl-D-aspartate receptors Dravet syndrome Results published in JAMA Neurology showed adding Fintepla treatment to patients experiencing high number of convulsive seizures despite antiepileptic regimens including stiripentol led to significant and clinically meaningful (> 50%) reduction in monthly convulsive seizure frequency


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