Phase I

Annexon Biosciences Inc., of South San Francisco


Monoclonal antibody targeting C1q

Guillain-Barré syndrome

Treatment in phase Ib study produced classical cascade inhibition, measured in the blood and cerebrospinal fluid; neurofilament light chain levels were reduced; patients had improved muscle strength

Checkmate Pharmaceuticals Inc., of Cambridge, Mass.


CpG-A Toll-like receptor 9 agonist encapsulated in a virus-like particle

Squamous cell cancer of the head and neck

First patient treated with CMP-001 plus anti-PD-L1 Bavencio (avelumab) in the phase Ib/II Javelin Medley study sponsored by Pfizer Inc. in collaboration with Merck KGaA; the study is also testing triplet combinations, adding 4-1BB agonist utomilumab or OX40 agonist PF-04518600 to CMP-001 and Bavencio

Actinogen Medical ASX, of Sydney


Blocks 11beta-HSD1

Healthy elderly subjects (Alzheimer's disease)

In the XanaHES study, 12 weeks of treatment reduced serum cortisol levels from baseline (p<0.001); treatment improved 3 of 6 domains of the Cogstate Cognitive Test Battery: One Back Test (p<0.01, effect size=0.83), Identification Test (p=0.05, effect size=0.67) and Detection Test (p=0.09, effect size=0.76)

Immunicum AB, of Stockholm


Activated allogeneic dendritic cells

Head and neck squamous cell carcinoma, non-small-cell lung cancer and gastric and gastroesophageal junction adenocarcinoma

There were no serious adverse events in the 3 patients treated with 2 intratumoral injections of 3M cells of ilixadencel when combined with Keytruda (pembrolizumab, Merck & Co. Inc.) in phase Ib/II trial; plans to test the next dose level of 10M cells

Themis Bioscience GmbH, of Vienna,

Second-generation Zika vaccine

Vaccine using a measles vector platform

Zika infection prophylaxis

Started study testing the safety and immunogenicity of the vaccine in 48 healthy volunteers

Phase II

Anaptysbio Inc.,of San Diego


Interleukin 1-like receptor 2 inhibitor

Generalized pustular psoriasis

Interim analysis of Gallop study in first 2 participants with moderate to severe disease to complete 16-week monotherapy showed both achieved primary endpoint of disease score improvement at day 29 and day 113 without requiring rescue therapy; both showed modified Japanese Dermatology Association score improvement, with reduction of 58% at day 8 and 63% at day 113; complete clearance of skin pustules was achieved by day 8 and through day 113, and serum C-reactive protein decreased to near normal levels in both participants

Ixaltis SAS, of Toulouse, France


Dual 5-HT receptor agonist/antagonist

Urinary incontinence

2 studies conducted in Europe, Canada and U.S. that enrolled about 300 participants showed clinically meaningful effect

Minerva Neurosciences Inc., of Waltham, Mass.


5-HT reuptake inhibitor; 5-HT1a receptor antagonist; 5-HT2a receptor modulator

Major depressive disorder

Screening completed in phase IIb trial in those with moderate to severe disease; top-line data expected in fourth quarter 2019

Minerva Neurosciences Inc., of Waltham, Mass.


Orexin 2 receptor antagonist

Major depressive disorder

Study drug showed quantitative advantage in primary endpoint of all-cause discontinuation at 6 months, at 41% vs. 47% for comparator quetiapine XR (Seroquel, Luye Pharma Group Ltd.) but did not achieve statistical separation; seltorexant 20 mg produced greater improvement at week 24 (-22.7 points) in mood improvement measured by MADRS total score than 40-mg dose (-7.9), quetiapine 150-mg dose (-17) and quetiapine 300 mg (-14.8)

Rottapharm Biotech Srl, of Monza, Italy


Imidazoline-2 receptor ligand

Chronic pain associated with osteoarthritis

Data published in Osteoarthritis and Cartilage showed the drug appears to be effective on pain characterizing the metabolic OA phenotype

Phase III

Catabasis Pharmaceuticals Inc., of Cambridge, Mass.


Nuclear factor kappa B inhibitor

Duchenne muscular dystrophy

PolarisDMD trial exceeded target enrollment of 125 boys; top-line data due in fourth quarter 2019

CTI Biopharma Corp., of Seattle


CSF-1 antagonist; FLT3/IRAK-1/JAK2 kinase inhibitor

Myelofibrosis; thrombocytopenia

Pivotal Pacifica trial opened enrollment; 180 adults with myelofibrosis and severe thrombocytopenia will be randomized (2-to-1) to 200 mg of drug twice daily or physician's choice; primary endpoint is percentage who achieve at least 35% reduction in spleen volume at 24 weeks

Minerva Neurosciences Inc., of Waltham, Mass.

Roluperidone (MIN-101)

5-HT 2a receptor antagonist; opioid receptor sigma antagonist 2


384 of target 501 participants enrolled into ongoing trial to treat negative symptoms in schizophrenia; top-line data expected in first half of 2020

Sanofi SA, of Paris

Jevtana (cabazitaxel)

Taxane chemotherapy

Metastatic castration-resistant prostate cancer

Data published in The New England Journal of Medicine showed Card study met primary endpoint of improved radiographic progression-free survival with drug (N=129) compared to abiraterone (Zytiga, Johnson & Johnson) or enzalutamide (Xtandi, Pfizer Inc./Astellas Pharma Inc.) (n=126; median 8 vs. 3.7 months; p<0.0001); in key secondary endpoint of overall survival (median 13.6 vs. 11 months; p=0.0078), Jevtana reduced risk of death from any cause by 36% vs. comparators

Taiwan Liposome Co. Ltd., of Taiwan


Liposomal formulation of dexamethasone sodium phosphate


Placebo- and active comparator-controlled pivotal Excellence trial initiated to evaluate single and repeated doses in 500 participants with osteoarthritis of the knee


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


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