Company Product Description Indication Status
Phase I
Arecor Ltd., of London AT-247 Insulin ligand; insulin receptor agonist Type 1 diabetes Following dosing, insulin detected in blood 12 minutes (p=0.0004) earlier than following injection with Novorapid (insulin aspart, Novo Nordisk A/S) and 2 minutes (p=0.0003) earlier than injection with Fiasp (insulin aspart, ultra-fast acting, Novo); onset of glucose lowering action was 23 minutes (p=0.0004) earlier for study drug than Novorapid and 9 minutes (p=0.0006) earlier than Fiasp, and early (within 60 minutes) glucose lowering action after dosing was 4-fold (p=0.0004) greater than with Novorapid and 2-fold (p=0.0009) greater than with Fiasp
Bionic Sight LLC, of New York BS-01 (optogenetic gene therapy) Channel rhodopsin stimulator Retinitis pigmentosa First participant dosed in phase I/II trial assessing effectiveness to detect light, motion and shape/object recognition; application of therapy to subsequent participants postponed due to COVID-19 pandemic, with enrollment set to resume in second half of 2020
Enb Therapeutics Inc., of New York ENB-003 Endothelin ET-B receptor antagonist Solid tumors Dosing completed in first cohort in dose-escalation combination trial with pembrolizumab (Keytruda, Merck and Co. Inc.); safety review committee approved escalation to next dose
Exicure Inc., of Chicago Cavrotolimod (AST-008) TLR-9 agonist Merkel cell and cutaneous squamous cell carcinoma First participant dosed in dose-expansion phase of phase I/II trial in advanced or metastatic disease assessing antitumor response rate of study drug plus checkpoint inhibitor treatment
Forma Therapeutics Inc., of Watertown, Mass. FT-4202 Selective RBC pyruvate kinase-R activator  Sickle cell disease  Results from SCD patient arm of ongoing study demonstrated favorable tolerability and pharmacokinetic/pharmacodynamic effects
Immunicum AB, of Stockholm Ilixadencel Allogeneic dendritic cell vaccine Gastrointestinal stromal tumors Findings from phase I/II trial, which met primary endpoint of safety, showed favorable profile in combination with tyrosine kinase inhibitors and provided initial signals of clinical benefit in 2 of 6 participants, published in Cancer Immunology, Immunotherapy 
Renibus Therapeutics Inc., of Dallas RBT-6  Stannous protoporphyrin Chronic kidney disease Findings in 24 participants with moderate to severe disease and elevated plasma HO-1, ferritin, p21 and NQO1, published in Clinical Journal of American Society of Nephrology, showed the 4 proteins manifested statistically significant dose- and time-dependent elevations following infusion, suggesting agent's ability to assess antioxidant defense mechanisms and reserves
Sarepta Therapeutics Inc., of Cambridge, Mass. SRP-9001 DMD gene stimulator Duchenne muscular dystrophy 1-year data on 4 participants, published in JAMA Neurology, confirmed maintenance of vector transduction and functional improvements
Phase II
Addex Therapeutics Ltd., of Geneva JNJ-40411813 (ADX-71149) Metabotropic glutamate receptor 2 agonist Epilepsy Licensee Janssen Pharmaceuticals Inc., unit of Johnson & Johnson, of New Brunswick, N.J., set to advance candidate into phase IIa proof-of-concept study in individuals with focal onset seizures with suboptimal response to levetiracetam; primary objective is efficacy using time-to-event endpoint
Anavex Life Sciences Corp., of New York ANAVEX-2-73 (blarcamesine) Sigma-1 receptor agonist Rett syndrome Original enrollment target exceeded by 50%; top-line results expected in second half of 2020
Dermavant Sciences, unit of Roivant Sciences Ltd., of Basel, Switzerland Tapinarof Aryl hydrocarbon receptor modulator Atopic dermatitis Phase IIb secondary efficacy and patient-reported outcomes, published in The Journal of the American Academy of Dermatology, showed statistically significant improvement in EASI75 (51%; n=41) vs. vehicle (25%; n=40) at week 12 (p=0.016) and statistically significantly greater reductions from baseline in mean percentage change in BSA (-48%; n=41) vs. vehicle (-5%; n=40) at week 12 (p=0.006); participant rating of overall symptom severity as very or moderately improved (80%; n=41) achieved statistical significance vs. vehicle (43%; n=40) at week 12 (p<0.001)
Oncopeptides AB, of Stockholm Melflufen Peptide-drug conjugate delivering alkylating payload into tumor cells Relapsed/refractory multiple myeloma Top-line results from pivotal Horizon study testing combination with dexamethasone showed overall response rate of 29% in intent-to-treat population, 26% in triple class refractory patients and 24% in extra medullary disease patients; median progression-free survival was 4.2 months, 3.9 months and 2.9 months, respectively, while median overall survival was 11.6 months, 11.2 months and 6.5 months, respectively; company on track to submit NDA to FDA by end of second quarter of 2020
PDS Biotechnology Corp., of Florham Park, N.J. PDS-0101 Cationic lipid nanoparticle vaccine Human papillomavirus-associated cancer First participant dosed in NCI-led combination study with bintrafusp alfa (M-7824, Merck KGaA/Glaxosmithkline plc) and NHS-IL12 (M-9241, Merck Serono/NCI) in advanced disease; primary endpoint is objective response rate
Provention Bio Inc., of Oldwick, N.J. Teplizumab (PRV-031) CD3 antagonist Type 1 diabetes Extended follow-up showed median time to clinical diagnosis following single 14-day course of treatment was about 5 years, improvement of 1 year from previously published data, vs. about 2 years for placebo, unchanged from previous data; treatment was associated with greater on-study C-peptide (p=0.009) vs. placebo
Vyome Therapeutics Inc., of Princeton, N.J. VB-1953 TLR-MD2 inhibitor  Acne In 471 people with moderate to severe inflammatory disease, trial showed statistically significant difference in primary endpoint of mean absolute change in inflammatory lesions at week 12 vs. combined vehicle group (p<0.012); in comparison between treatment arm and vehicle, mean absolute change in inflammatory lesions at week 12 was 20.4 and 17.8, respectively, in intent-to-treat group (p<0.003) and 20.4 and 16.6, respectively, in per protocol group (p<0.001)
Phase III
Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl) Fish oil Cardiovascular disease Vascepa produced 23% reductions in both first and total primary composite major adverse cardiovascular events (5-point MACE) compared to placebo in a subset analysis of patients with diabetes in the Reduce-It study; drug reduced first and total hard 3-point MACE by 30% and 29%, respectively, compared to placebo
Eli Lilly and Co., of Indianapolis Verzenio (abemaciclib) Inhibitor of cyclin-dependent kinases 4 & 6 High-risk, hormone receptor-positive, HER2-negative early breast cancer In the MonarchE study, Verzenio plus adjuvant endocrine therapy (ET) improved invasive disease-free survival compared to ET alone based on a interim analysis; data to be presented at a medical meeting later in 2020
Humanigen Inc., of Burlingame, Calif. Lenzilumab Monoclonal antibody targeting granulocyte macrophage-colony stimulating factor COVID-19 Median time to improvement and recovery in 12 patients was 5 days for lenzilumab, compared to 10-11 days for matched patients who received remdesivir (Gilead Sciences Inc.)
Janssen Pharmaceutical Co. of New Brunswick, N.J.-based Johnson & Johnson Daratumumab Monoclonal antibody targeting CD38 Newly diagnosed light chain amyloidosis Daratumumab plus cyclophosphamide, bortezomib and dexamethasone (CyBorD) produced a hematologic complete response in 53% of patients, compared to 18% of patients taking CyBorD (p<0.0001)
Merck & Co. Inc., of Kenilworth, N.J., and Pfizer Inc., of New York Steglatro (ertugliflozin) Oral SGLT2 inhibitor Type 2 diabetes and atherosclerotic cardiovascular disease Results showed Vertis CV cardiovascular outcomes trial testing Steglatro when added to background standard-of-care treatment in more than 8,200 patients met primary endpoint of noninferiority on major adverse CV events vs. placebo
Neuren Pharmaceuticals Ltd., of Melbourne, Australia, and Acadia Pharmaceuticals Inc., of San Diego Trofinetide Synthetic analogue of the amino-terminal tripeptide of IGF-1 Rett syndrome Restarted enrollment in study after a pause due to COVID-19
Sanofi SA, of Paris Avalglucosidase alfa Enzyme replacement therapy  Late-onset Pompe disease Avalglucosidase alfa was noninferior to alglucosidase alfa with avalglucosidase alfa producing a 2.4-point greater improvement in percent-predicted forced vital capacity compared to alglucosidase alfa (p=0.0074); the result wasn't statistically significant for superiority 


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