Company Product Description Indication Status
Phase I
Biogen Inc., of Cambridge, Mass. BIIB-067 (tofersen) Antisense oligonucleotide Motor neuron disease Healthy volunteer study testing microdose of radiolabeled BIIB-067 (99mTc-MAG3-BIIB067) co-administered with BIIB-067 on hold due to COVID-19
Curis Inc., of Lexington, Mass. CA-4948 IRAK4 inhibitor Relapsed or refractory non-Hodgkin lymphoma Due to COVID-19, certain sites remain open for enrollment while others have temporarily halted enrollment of new patients; company expects to report updated efficacy data from study and declare recommended phase II dose in 2020
Gossamer Bio Inc., of San Diego GB-002 Inhaled PDGFR inhibitor Pulmonary arterial hypertension Given COVID-19-related delays in enrollment, company now anticipates reporting initial results from phase Ib trial in second half of 2020
Gossamer Bio Inc., of San Diego GB-004 Oral HIF-1α stabilizer Ulcerative colitis Reported top-line data from phase Ib trial in mild to moderate UC, showing tolerability and gut-targeted pharmacokinetic profile with rapid clearance from systemic circulation and multifold higher concentrations of drug in the gut as compared to the plasma after 8 hours of dosing; though not powered for differences in clinical outcomes, encouraging trends observed at day 28, including mucosal healing observed in 4 of 23 patients (17%) vs. 0 of 11 in placebo arm, histologic remission in either sigmoid or rectum (10/23 [43%] vs. 2/11 [18%]), clinical response (6/20 [30%] vs. 2/11 [18%]) and improvement in rectal bleeding subscore (13/21 [62%] vs. 5/11 [45%]); 1 patient in GB-004 arm achieved clinical remission
I-Mab Biopharma Co. Ltd., of Shanghai TJD-5 (TJ-004309) CD73 antibody Advanced solid tumors First patient dosed in phase I/II trial in China
Medday Pharmaceuticals SA, of Paris MD-1003 (biotin) Dual acetyl CoA carboxylase-1/2 stimulator Multiple sclerosis Studies comparing pharmacokinetic parameters in renal impaired/hepatic impaired patients and healthy subjects terminated following phase III study failure to meet primary endpoint
Novadip Biosciences SA, of Mont-Saint Guibert, Belgium NVD-003 Autologous cell-based osteogenic product Non-healing fracture of lower limb Analysis of phase I/IIa trial in 9 adults showed grafting surgery completed successfully in all, without deviating from standard medical practice; at median of 12 months post-treatment, no related safety signals reported; exploratory analysis performed on data from first 5 patients to complete 6-month follow-up showed strong positive trend in radiological healing, with confirmed bone formation for all patients and radiologically confirmed union for 3
Phase II
Actelion Ltd., of Allschwil, Switzerland Uptravi (selexipag) Prostacyclin receptor agonist Pulmonary arterial hypertension  Screening in study to confirm doses in children with PAH on hold due to COVID-19
Alexion Pharmaceuticals Inc., of Boston ALXN-1830 FcRn inhibitor Warm autoimmune hemolytic anemia Study stopped due to COVID-19, as WAIHA patients may be at higher risk of contracting disease based on standard-of-care treatments
Avrobio Inc., of Cambridge, Mass. AVR-RD-01  GLA gene stimulator Fabry disease All 3 participants from trial who discontinued enzyme replacement therapy (ERT) after receiving study therapy remained off ERT at data cutoff of April 27, 2020
Avrobio Inc., of Cambridge, Mass. AVR-RD-04 CTNS gene stimulator Cystinosis New data from investigator-sponsored phase I/II trial showed that, 6 months following administration of therapy, first participant's estimated glomerular filtration rate improved from baseline of 38 mL/mi/1.73m2 to 52 mL/mi/1.73m2 and serum creatinine fell from 2.2 mg/dL at baseline to 1.6 mg/dL at 6 months
Avrobio Inc., of Cambridge, Mass. AVR-RD-01  GLA gene stimulator Fabry disease Interim data from ongoing Fab-201 trial showed first participant continued to show increased leukocyte and plasma alpha-galactosidase enzyme activity up to 22 months post-treatment; third had sustained decrease in plasma lyso-Gb3 and total Gb3 levels 9 months post-dosing; fourth, who was first dosed with plato gene therapy platform, had 43% reduction in plasma lyso-Gb3 at 1 month and, at 3 months, leukocyte enzyme and plasma enzyme activity levels ~3X higher than mean activity level of first 3 participants at same time point
Capricor Therapeutics Inc., of Los Angeles CAP-1002 Cardiosphere-derived allogenic stem cell therapy Duchenne muscular dystrophy 1-year data from Hope-2 trial showed improved performance of upper limb, with mean change of 2.4 points over placebo (p=0.05); data also showed global improvements in cardiac function measured by ejection fraction (p=0.004) and indexed volumes (LVESV, p=0.01, LVEDV p=0.07)
Fulcrum Therapeutics Inc., of Cambridge, Mass. Losmapimod p38alpha/beta mitogen activated protein kinase inhibitor Facioscapulohumeral dystrophy Due to impact of COVID-19 pandemic, amended protocol of fully enrolled phase IIb Redux4 trial, extending from 24 to 48 weeks, adding 36-week biopsy for those unable to receive 16-week biopsy and including interim analysis for those who completed 16-week biopsy per original protocol, with interim data expected in third quarter of 2020; also initiated open-label extension
Opiant Pharmaceuticals Inc., of Santa Monica, Calif. OPNT-002 (naltrexone hydrochloride, intranasal) Opioid receptor antagonist Alcohol use disorder Paused initiation of recruitment due to COVID-19 pandemic
Roivant Sciences Ltd., of New York and Basel, Switzerland Gimsilumab Fully human monoclonal antibody targeting GM-CSF COVID-19 Independent data monitoring committee for pivotal Breathe study in patients with lung injury or acute respiratory distress syndrome unanimously recommended trial continue after prespecified safety assessment from first 10% of randomized subjects after 6 days follow-up; company recently amended protocol to permit use of investigational antivirals as well as treatment with convalescent plasma prior to enrollment; 56 patients enrolled to date
Phase III
Actelion Ltd., of Allschwil, Switzerland Uptravi (selexipag) Prostacyclin receptor agonist Chronic thromboembolic pulmonary hypertension Screening temporarily halted in Select study due to COVID-19
Novartis AG, of Basel, Switzerland QAW-039 (fevipiprant) DP2 receptor antagonist Asthma Study in patients whose disease is inadequately controlled by standard of care terminated
Novo Nordisk A/S, of Bagsvaerd, Denmark Semaglutide GLP-1 receptor agonist Weight management Headline results from phase IIIa Step 4 study showed, after 20-week run-in period, the 803 people reaching target dose of 2.4 mg had reduced mean body weight from 107.2 kg to 96.1 kg and were randomized to continued treatment with either once-weekly semaglutide 2.4 mg or placebo for 48 weeks; trial achieved primary endpoint, demonstrating continued treatment resulted in additional mean weight loss of 7.9%, from a mean baseline body weight at randomization of 96.1 kg, while those on placebo regained 6.9% of body weight; people who received semaglutide 2.4 mg for 68 weeks achieved total weight loss of 17.4%

Notes

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