Company Product Description Indication Status
Phase I
Atara Biotherapeutics Inc., of South San Francisco ATA-188 Off-the-shelf, allogeneic EBV-specific T-cell immunotherapy Progressive multiple sclerosis Data from first part of ongoing study showed treatment well-tolerated across all 4 dose cohorts; patients who achieved sustained disability improvements at any timepoint maintained it at all future timepoints and a higher proportion of patients showed sustained disability improvements with increasing dose
Bicycle Therapeutics plc, of Cambridge, U.K. BT-8009 Second-generation Bicycle toxin conjugate Advanced solid tumors associated with Nectin-4 expression Dosed first patient in phase I dose-escalation portion of phase I/II trial
Biogen Inc., of Cambridge, Mass. BIIB-091 Bruton’s tyrosine kinase inhibitor Healthy volunteers (eventually multiple sclerosis) Safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses support continued development of the drug
Celltrion Group, of Incheon, Korea CT-P59 Monoclonal antibody COVID-19 Interim results from trial in healthy subjects demonstrated no significant drug-related adverse events and there were no adverse events from maximum tolerated dose cohort
Lyra Therapeutics Inc., of Watertown, Mass. LYR-210  Mometasone furoate in bioresorbable polymeric matrices Chronic rhinosinusitis Drug reduced IL-13, CCL26 and periostin (p<0.05); IL-5 and CCL18 showed a trend toward reduction; 4 cardinal symptoms score strongly correlated with sinonasal outcome (SNOT-22) scores and both improved over the 24-week study
Neurocrine Biosciences Inc., of San Diego, and Voyager Therapeutics Inc., of Cambridge, Mass. NBlb-1817 (VY-AADC) Gene therapy Parkinson’s disease Data from phase Ib PD-1101 3-year trial showed 1-time treatment resulted in sustained improvement in motor function including greater “on” time without dyskinesia, reduction in Unified Parkinson’s Disease Rating Scale Part III scores, and reduction in amount of medications; treatment reduced average “off” time by up to -1.91 hours and improved average “on” time by up to +2.23 hours; 14 of 15 patients continued to show improvement in disease staging after 3 years, as assessed by modified Hoehn & Yahr scale
Phase II
Anavex Life Sciences Corp., of New York Anavex 2-73 (blarcamesine) Oral, small-molecule activator of sigma-1 receptor Rett syndrome Completed study enrolling 31 patients; top-line data due in fourth quarter of 2020
Aura Biosciences Inc., of Cambridge, Mass. AU-011 (belzupacap sarotalocan) Viral-like particle bioconjugates activated with ophthalmic laser Primary choroidal melanoma Dosed first patient in study testing suprachoroidal administration
Clene Nanomedicine Inc., of Salt Lake City CNM-Au8 Nanocatalytic therapeutic Relapsing multiple sclerosis and Parkinson’s disease Interim results from 4 MS and 6 PD completers demonstrate significant CNS target engagement; data indicate catalytic bioenergetic improvements across important CNS bioenergetic metabolites, including total NAD levels, NAD+/NADH ratio and ATP levels, indicating homeostatic effect on brain bioenergetics
Clene Nanomedicine Inc., of Salt Lake City CNM-Au8 Nanocatalytic therapeutic Stable relapsing multiple sclerosis with chronic vision impairment Expanded interim results from Visionary-MS study showed, through week 36, patients on CNM-Au8 demonstrated notable median improvements in low contrast letter acuity (n=21, 42 eyes), as well as the 3 remaining modified Multiple Sclerosis Functional Composite subscales, including Symbol Digit Modalities Test (cognition), 9-Hole Peg Test (upper extremity function) and Timed 25-foot Walk (gait)
Galapagos NV, of Mechelen, Belgium Ziritaxestat (GLPG-1690) Autotaxin inhibitor Diffuse cutaneous systemic sclerosis Drug reduced the modified Rodnan Skin Score at week 24 by 8.3 points compared to a 5.7-point reduction for placebo (p=0.0411)
Immunic Inc., of New York IMU-838 Oral DHODH inhibitor Relapsing-remitting multiple sclerosis Unblinded data from Emphasis trial of subgroup analyses showed consistent effect for MRI lesion suppression across different populations, including a treatment effect independent of the presence of pre-study treatment; time course of MRI lesion suppression indicated the reduction of lesions was already present at the first time point of assessment in the trial (week 6), which is consistent with the rapid attainment of stable therapeutic drug levels; other secondary endpoints beyond MRI effect, such as relapse activity or changes in Expanded Disability Status Scale, showed trends toward an advantage for IMU-838 treatment groups vs. placebo regarding time-to-first relapse and annualized relapse rate, although study's duration was too short to provide a formal assessment; as previously reported, trial achieved all primary and secondary endpoints
Prothena Corp. plc, of Dunlin Prasinezumab  Monoclonal antibody targeting alpha-synuclein Parkinson's disease  Pasadena study didn't meet its primary endpoint of change in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) total score; pooled dose levels reduced the decline in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III by 35% compared to placebo; time to at least a 5-point progression on MDS-UPDRS Part III was slower for drug compared to placebo (HR=0.82)
Santhera Pharmaceuticals Holding AG, of Pratteln, Switzerland, and Reveragen Biopharma Inc., of Rockville, Md. Vamorolone Binds to same receptors as corticosteroids but modifies downstream activity of receptors Duchenne muscular dystrophy Reveragen completed enrollment in pivotal phase IIb Vision-DMD study; top-line data expected second quarter of 2021
Phase III
Biogen Inc., of Cambridge, Mass. Vumerity (diroximel fumarate) Monomethyl fumarate prodrug  Multiple sclerosis In the Evolve-MS-2 study, patients taking Vumerity reported a lower likelihood of experiencing gastrointestinal (GI) symptoms that interfered with daily activities or were associated with missed work, as well as less concomitant medication use to treat GI symptoms, compared to patients taking Tecfidera (dimethyl fumarate); in Evolve-MS-1, annual rate of brain volume change in patients treated with Vumerity for 2 years was similar to the rate observed in healthy individuals; approximately 90% of treated patients remained free from confirmed disability progression and around 84% were relapse-free at 2 years
Cancer Prevention Pharmaceuticals Inc., of Tucson, Ariz. CPP-1X/sul Ornithine decarboxylase inhibitor; cyclooxygenase inhibitor Familial adenomatous polyposis Data from FAP-310 study published in The New England Journal of Medicine showed trial did not demonstrate that overall disease progression was significantly lower compared to eflornithine or sulindac alone, though results showed that in a subgroup of patients with intact colons – representing the vast majority of young FAP patients – there was a 70% decreased risk of disease progression with CPP-1X/sul; based on data, company intends to pursue regulatory applications
Eli Lilly and Co., of Indianapolis Reyvow (lasmiditan) Serotonin (5-HT)1F receptor agonist Migraine Data from Centurion trial showed adults who took doses of 100 mg and 200 mg for migraine attacks had 3.8 and 4.6 times greater odds, respectively, of achieving pain freedom at 2 hours vs. those taking placebo (co-primary endpoint); drug showed superiority over placebo in all gated endpoints, including proportions of study participants who after treating their first migraine attack reported pain freedom at 1 hour (200 mg dose), pain relief at 1 hour and 2 hours (both doses), sustained pain freedom at 24 hours (both doses) and 48 hours (200 mg dose) and no disability at 2 hours (both doses)
Genentech, of South San Francisco, a member of the Roche Group Ocrevus (ocrelizumab) Monoclonal antibody targeting CD20-positive B cells Relapsing-remitting multiple sclerosis Data from 2-year phase IIIb Casting study showed drug is effective treatment option for patients who experienced suboptimal response to prior disease-modifying therapy (DMT); proportion of patients achieving no evidence of disease activity (NEDA) remained consistently high across all measured patient subgroups, including baseline MRI activity, relapse activity, disability level, age and the number of prior DMTs; 78% of patients treated with only 1 prior DMT vs. 70% treated with 2 prior DMTs achieved NEDA
GW Pharmaceuticals Ltd., of Carlsbad, Calif Nabiximols  Cannabinoids THC and CBD Multiple sclerosis-associated spasticity In the withdraw study, decrease in spasticity numeric rating scale from baseline was 46% for patients who stayed on drug compared to a 34% reduction for those who were switched to placebo (p=0.011); spasm frequency reduced by 44% for nabiximols compared to a reduction of 24% for placebo (p=0.006)
LFB SA, of Les Ulis, France  LFB IVIg 10%  Intravenous immunoglobulin  Chronic inflammatory demyelinating polyradiculoneuropathy  In the Prism study, 76.2% of patients responded, defined as an improvement of ≥1 point on the Inflammatory Neuropathy Cause and Treatment disability scale compared to a response rate of 33.3% for historical control group (p<0.0001)
Novartis AG, of Basel, Switzerland Mayzent (siponimod) Sphingosine 1-phosphate receptor modulator Progressing multiple sclerosis Analyses from phase IIIb Exchange and Expand trials showed treatment safe, with benefits in cognitive performance in reduction in risk of disability progression 
Partner Therapeutics Inc., of Lexington, Mass. Leukine (sargramostim, yeast derived rhu-GM-CSF) Recombinant yeast derived human granulocyte-macrophage growth factor Melanoma NCI-sponsored study testing combination with Yervoy (ipilimumab, Bristol Myers Squibb Co.) and Opdivo (nivolumab, BMS) in front-line treatment resumed enrollment; restart based on results of planned interim efficacy and safety analysis of survival data from first 250 patients enrolled
Phase IV
Biogen Inc., of Cambridge, Mass. Tysabri (natalizumab), Plegridy (peginterferon beta-1a) and Avonex (interferon beta-1a) Integrin alpha-4/beta-1 antagonist, interferons Multiple sclerosis In the MS Paths study, no significant differences in the rates of new T2 lesions, T2 lesion volumes and brain atrophy were seen between patients given Tysabri every 4 weeks compared to extended interval dosing; significant improvement was observed in 9 of 12 Quality of Life in Neurological Disorders domains in patients treated with Tysabri compared to 4 of 12 domains in patients treated with Ocrevus (ocrelizumab, Roche Holding AG); patients ages 60 or older treated with Plegridy or Avonex had improvements in processing speed test and contrast sensitivity test over 1 year


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