Company Product Description Indication Status Phase I Dicerna Pharmaceuticals Inc., of Lexington, Mass. RG-6346 RNAi targeting hepatitis B virus mRNA Chronic hepatitis B virus infection In group C, 9 of 10 participants who received RG-6346 and a nucleoside analogue and completed the treatment period achieved 1 log10 IU/mL reduction in hepatitis B surface antigen (HBsAg) at day 112; mean reduction in HBsAg at day 112 was 1.39 log10 IU/mL for the 1.5-mg/kg dose, 1.80 log10 IU/mL for the 3-mg/kg dose and 1.84 log10 IU/mL for the 6-mg/kg dose Dicerna Pharmaceuticals Inc., of Lexington, Mass. Nedosiran RNAi targeting lactate dehydrogenase Primary hyperoxaluria 1, 2 and 3 (PH1, PH2 and PH3) In the Phyox3 open-label extension, 8 of 8 patients with PH1 achieved normalization or near-normalization urinary oxalate levels (Uox) at day 120 with a mean Uox of 0.404 mmol/1.73m2 BSA/24 hr; 1 of 3 patients with PH2 achieved normalization of Uox Personalized Stem Cells Inc., of Poway, Calif. PSC-01 Adipose-derived stromal vascular fraction cells Knee osteoarthritis Completed enrollment in the study; data expected by the end of 2020 Precigen Triple-Gene, a unit of Precigen Inc., of Germantown, Md. INXN-4001 Nonviral plasmid expressing SDF-1alpha, VEGF165 and S100A1 Chronic heart failure Half of the 10 patients had a 5-point improvement in their total KCCQ score 6 months after treatment; 4 of 8 evaluable patients had a >50-meter improvement in their 6-minute walk time at 6 months Tessa Therapeutics Pte Ltd., of Singapore CD30 CAR T Autologous cell therapy Relapsed/refractory Hodgkin lymphoma For the 41 patients, 1-year survival was 94% in phase I/II study; of patients with complete responses, 61% had no evidence of recurrence after 1 year Phase II Bioaegis Therapeutics Inc., of Morristown, N.J. rhu-pGSN Recombinant human plasma gelsolin Hospitalized patients with severe COVID-19 pneumonia Enrolled first patient in study testing 3 doses of the drug; primary endpoint is survival without organ failure and without mechanical ventilation, vasopressors or dialysis on day 14 Idorsia Pharmaceuticals Japan, unit of Idorsia Ltd, of Allschwil, Switzerland Daridorexant Dual orexin receptor antagonist Insomnia In dose-confirmation study in 47 adults in Japan, study drug showed dose-dependent decrease in wake after sleep onset (WASO) at day 1 and 2 and decreased latency to persistent sleep in dose-dependent manner; improvements seen on measures such as subjective WASO, subjective latency to sleep onset and subjective total sleep time Medicinova Inc., of La Jolla, Calif. Ibudilast (MN-166) Phosphodiesterases 4 and 10 inhibitor Alcohol use disorder Drug reduced the number of heavy drinking days compared to placebo (p=0.03) and attenuated alcohol cue-elicited activation in the bilateral ventral striatum (p=0.02) Nordic Nanovector ASA, of Oslo, Norway Betalutin CD37-targeting antibody-radionuclide conjugate Relapsed/refractory follicular lymphoma Independent review committee recommended company focus on the 15-MBq/kg Betalutin dosage arm and discontinue the 20-MBq/kg dose Phase III Boehringer Ingelheim International GmbH, of Ingelheim, Germany Ofev (nintedanib) Tyrosine kinase inhibitor Chronic fibrosing interstitial lung diseases with a progressive phenotype In the Inbuild study, at 52 weeks, 43.4% of patents taking Ofev had a >5% absolute decline in forced vital capacity (FVC), compared to 55% of patients taking placebo; >10% FVC was seen in 28.3% and 36.6% of patient taking Ofev and placebo, respectively Levo Therapeutics Inc., of Chicago LV-101 (intranasal carbetocin) Oxytocin-receptor agonist Prader-Willi syndrome Primary endpoint for the 9.6-mg dose of LV-10 was not met; 3.2-mg dose improved the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score (p=0.016); HQ-CT scores for combined doses produced a p-value of 0.055; 3.2-mg dose also improved clinical global impression of change (p=0.027) and the PWS Anxiety and Distress Behaviors Questionnaire (p=0.027) Novartis AG, of Basel, Switzerland Ofatumumab Monoclonal antibody targeting CD20 Relapsing forms of multiple sclerosis Data published in The New England Journal of Medicine showed drug reduced annualized relapse rate by 51% and 58% compared to teriflunomide in the Asclepios I and II studies, respectively (p<0.001 for both); drug reduced risk of confirmed disability worsening at 3 months by 34% (p=0.002) and at 6 months by 32% (p=0.01) Rafael Pharmaceuticals Inc., of Cranbury, N.J. CPI-613 (devimistat) Targets the mitochondrial tricarboxylic acid cycle Metastatic pancreatic cancer Completed target enrollment of 500 patients in the Avenger 500 study
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