Company Product Description Indication Status
Phase I
Alzecure Pharma AB, of Stockholm ACD-856 Stimulates neurotrophic signaling Healthy volunteers (eventually Alzheimer's disease, sleep disorders and traumatic brain injuries) Started study testing the half-life of the drug; data expected in the first half of 2020
Axovant Gene Therapies Ltd., of New York AXO-AAV-GM1 Gene therapy expressing GLB1 GM1 gangliosidosis First patient in the expanded access had clinically significant improvements based on neurological exam, the Vineland-3 scale, Clinical Global Impression assessments and nutritional status 6 months after treatment
Immutep Ltd., of Sydney Eftilagimod alpha (IMP-321) HLA class II antigen stimulator Advanced solid malignancies No dose limiting toxicities seen in the first 6 patients treated with 6 mg eftilagimod plus Bavencio (avelumab, Merck KGaA and Pfizer Inc.); started recruitment of second cohort of 6 patients to be treated with 30 mg eftilagimod plus Bavencio
Neurovive Pharmaceutical AB, of Lund, Sweden KL-1333 Modulates cellular levels of NAD+ Healthy volunteers (eventually primary mitochondrial disease) Completed recruitment of 5 cohorts of healthy volunteers who received multiple ascending doses
Noxxon Pharma NV, of Berlin NOX-A12 Targets C-X-C chemokine ligand 12 Brain cancer Data safety monitoring board reviewed 10-week data from first patient treated with NOX-A12 plus radiotherapy and recommended enrolling the rest of the cohort
Pioneer Project, of Marseille, France Monalizumab, MEDI-9447, AZD-6738 and AZD-9150 NKG2A inhibitor, CD73 inhibitor, ATR inhibitor and Stat3 inhibitor PD1(L1) inhibitor-resistant advanced non-small-cell lung cancer First patient treated in study testing each drug in combination with Imfinz (durvalumab, Astrazeneca plc); primary endpoint is the percentage of patients that achieve complete response, partial response or maintain a stable disease at 12 weeks
Phase II
Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. ARO-AAT RNAi targeting mutant alpha 1-antitrypsin Alpha-1 antitrypsin deficiency Treated first of approximately 12 patients in the open-label AROAAT2002 study testing changes in a novel histological activity scale at 6 months, 12 months, 18 months and 24 months
Concert Pharmaceuticals Inc., of Lexington, Mass. CTP-543 JAK1 and JAK2 inhibitor Moderate-to-severe alopecia areata Completed study comparing 8 mg twice-daily to 16 mg once-daily; results for 8 mg twice-daily arm were consistent with previous trials; data from study to be presented at a future medical meeting; plans to use the twice-daily regimen going forward
Phase III
Bone Therapeutics SA, of Gosselies, Belgium JTA-004 Noncellular viscosupplement Knee osteoarthritis Submitted CTA for the 676-patient study comparing the reduction of pain for JTA-004, placebo and Hylan G-F 20; enrollment expected to begin in early 2020 with results in the second quarter of 2021
Contrafect Corp., of Yonkers, N.Y. Exebacase Lysin Staph aureus bacteremia, including right-sided endocarditis Started 350-patient Disrupt study comparing exebacase to placebo with both groups getting standard antibiotics; primary endpoint is clinical response at day 14 for patients with methicillin-resistant Staph aureus
Lexicon Pharmaceuticals Inc., of The Woodlands, Texas Sotagliflozin Inhibits sodium-glucose co-transporter types 1 and 2 Type 2 diabetes with inadequate glycemic control on a dipeptidyl peptidase 4 inhibitor with or without metformin Sotagliflozin reduced A1C at week 26 compared to placebo; drug was noninferior to empagliflozin on A1C reduction from baseline at week 26; data to be presented at a future medical meeting
Poxel SA, of Lyon, France, and Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan Imeglimin Tetrahydrotriazine Type 2 diabetes In the Times 2 study, imeglimin reduced HbA1c over 52 weeks as a monotherapy (0.46%) and when added on to various diabetes treatments (0.12%-0.92%)
Stealth Biotherapeutics Inc., of Boston Elamipretide Mitochondrial-targeted compound Primary mitochondrial myopathy Mmpower-3 study did not meet its primary endpoints assessing changes in the six-minute walk test and Primary Mitochondrial Myopathy Symptom Assessment Total Fatigue Score


For more information about individual companies and/or products, see Cortellis.

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