Phase I

Adverum Biotechnologies Inc., of Menlo Park, Calif.


Gene therapy

Wet age-related macular degeneration

24-week data from first cohort (n=6) treated with 1-time intravitreal dose in Optic trial showed patients achieved vision maintenance and improvements in retinal anatomy, with 0 anti-VEGF rescue injections required

Freeline Therapeutics Ltd., of London


Liver-directed AAV gene therapy

Fabry disease

Dosed first patient in Marvel1 phase I/II trial in adult males; in addition to safety, study will assess endpoints including clearance of Gb3 and LysoGb3 from plasma, changes in renal and skin biopsies, renal and cardiac function, GLA immune response, viral shedding and quality of life

Gracell Biotechnologies Co. Ltd., of Suzhou, China


FasT CAR-19 cell gene therapy

Relapsed/refractory B-cell acute lymphoblastic leukemia

Treatment assessed in 24 patients over 28 days of follow-up showed 23 (95.8%) achieved complete remission, with or without complete blood count recovery; 21 (87.5%) achieved undetectable minimal residual disease

GT Biopharma Inc., of Tampa, Fl.


Tri-specific NK cell Engager

Acute myelogenous leukemia

Started enrollment in phase I/II trial; open-label, dose-escalation phase I portion will evaluate drug in patients with CD33-expressing, high risk myelodysplastic syndromes, r/r AML or advanced systemic mastocytosis

Moderna Inc., of Cambridge, Mass.


Composed of 2 mRNAs encoding heavy and light chains of anti-chikungunya antibody with lipid nanoparticle technology

Chikungunya virus

Data from first analysis in healthy adults showed all 3 dose levels led to detectable levels of CHKV-24 antibody in all subjects, ranging from 1 mcg/mL to 14 mcg/mL; all participants also showed circulating neutralizing antibody activity against chikungunya virus replication in NT50 assay

Moderna Inc., of Cambridge, Mass.


Vaccine combining 6 mRNAs in single vial

Primary cytomegalovirus infection

Data from 3-month interim analysis showed, in both seronegative and seropositive subjects, a dose-related increase in neutralizing antibody titers in both epithelial cell and fibroblast assays; in seronegative patients, after second vaccination, neutralizing antibody titers in epithelial cells were 3 to 5 times higher than CMV-seropositive baseline titers at 90 mcg and 180 mcg doses, while, in fibroblasts, neutralizing antibody titers were equivalent to CMV-seropositive titers at both doses; in seronegative patients, second vaccination boosted neutralizing antibody titers in epithelial cells to a level of 10-fold to 19-fold baseline titers in all groups, and boost neutralizing antibody titers in fibroblasts to level of 2-fold to 4-fold baseline titers in all groups

Navitor Pharmaceuticals Inc., of Cambridge, Mass.


Oral, small-molecule activator of brain mTORC1

Treatment-resistant depression

Completed 3 studies (001, 002 and 003) in healthy volunteers and patients; in 001, signals of efficacy were observed on core symptoms of depression as measured by site-rated 6-item Hamilton Depression Scale and centrally rated 6- and 8-item Montgomery-Asberg Depression Rating Scale; in 002, a single, oral dose showed rapid exposure in both plasma and cerebrospinal fluid; 003 data showed statistically significant quantitative electroencephalogram signals of neural activation vs. placebo related to specific differentiated functional effects in the brain

Noxxon Pharma NV, of Berlin


CXCL12 inhibitor

Newly diagnosed brain cancer

Started recruitment in phase I/II study testing combination with radiotherapy; main objective is safety and tolerability, while secondary endpoints include activity of therapy, assessed through monitoring of tumor vascularization by MRI scans, progression-free survival, overall survival and rates of response

Phase II

Cellular Biomedicine Group Inc., of New York and Shanghai


Allogenic human adipose-derived mesenchymal progenitor cell therapy

Knee osteoarthritis

First phase II study plans to recruit 108 patients with Kellgren and Lawrence grade II-III KOA at 6 hospitals in China

Oncopeptides AB, of Stockholm


Lipophilic peptide-conjugated alkylator

Multiple myeloma

Interim data from pivotal Horizon trial showed activity in patients with relapsed/refractory disease, many of whom also have extramedullary disease; overall response rate was 28% and clinical benefit rate was 40%; 86% of evaluated patients achieved disease stabilization or better

Phase III

Chugai Pharmaceutical Co. Ltd., of Tokyo, and Genentech Inc., of South San Francisco, part of the Roche Group


Anti-IL-6 receptor humanized recycling antibody

Neuromyelitis optica spectrum disorder

In Sakurastar study, monotherapy reduced risk of relapse by 55% (p=0.0184) in overall population, including AQP4-IgG seropositive and seronegative patients, achieving primary endpoint of time to first protocol-defined relapse in the double-blind period

Genentech Inc., of South San Francisco, a unit of the Roche Group

Tecentriq (atezolizumab)

PD-L1-targeting antibody

Advanced nonsquamous and squamous non-small-cell lung cancer

Data showed Impower110 study testing Tecentriq as first-line monotherapy vs. cisplatin or carboplatin and pemetrexed or gemcitabine in patients without ALK or EGFR mutations met primary endpoint in interim analysis, demonstrating statistically significant overall survival benefit in patients with high PD-L1 expression vs. chemotherapy alone; study will continue to final analysis for people with lower levels of PD-L1 expression

Ovid Therapeutics Inc., of New York

OV-101 (gaboxadol)

Delta-selective GABAA receptor agonist

Angelman syndrome

First patient randomized in pivotal Neptune study; about 50 pediatric patients, ages 4 to 12, will be enrolled; primary endpoint is change in overall score on Clinical Global Impression-Improvement-Angelman syndrome scale; top-line data expected in mid-2020

Strekin AG, of Basel, Switzerland


Intratympanic thermogel and oral formulation of PPAR agonist pioglitazone

Sensorineural hearing loss

Completed enrollment of the target of 165 patients in Restore study; top-line results anticipated in early 2020

Tocagen Inc., of San Diego

Toca-511 and Toca-FC

Vocimagene amiretrorepvec and extended-release formulation of 5-fluorocytosine


Missed the primary endpoint of overall survival vs. standard of care treatment (11.1 months median vs. 12.2 months, HR=1.06, p=0.6154); missed secondary endpoints, too

Phase IV

Biogen Inc., of Cambridge, Mass.

Tysabri (natalizumab)

Integrin alpha-4/beta-1 antagonist

Multiple sclerosis

Data from observational, open-label, single-arm Strive study found 70.1% of certain patients with early relapsing MS achieved no evidence of disease activity in first 2 to 4 years of treatment


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


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