Phase I

Auris Medical Holding Ltd., of Hamilton, Bermuda


Betahistine hydrochloride

Weight gain; drowsiness

Interim results showed good safety and tolerability over the 4-week treatment period and relevant reductions in olanzapine-induced weight gain and daytime sleepiness

Eyevensys SAS, of Paris, France


TNFRSF1A gene stimulator; TNF alpha ligand inhibitor


Completed part 1 of safety study, finding safety similar to other intraocular treatments; 3 of 9 non-infectious uveitis patients treated showed clinical improvements lasting for 6 months after 1 administration of the treatment

Kangpu Biopharmaceuticals Ltd., of Shanghai


Ubiquitin ligase modulator

Systemic lupus erythematosus

Found no safety or tolerability concerns and showed that dosing fed does not have significant influence on exposure vs. fasted

Panoptica Inc., of Mount Arlington, N.J.


VEGF receptor antagonist

Wet age-related macular degeneration

Of patients receiving PAN-90806 for 12 weeks without needing rescue with anti-VEGF intraocular injection, 88% experienced either clinical improvement or stability of their disease, with no serious or severe AEs related to drug

Phase II

Harbour Biomed Therapeutics Ltd., of Shanghai

HBM-9036 (tanfanercept)

TNF alpha ligand inhibitor; TNF binding agent


Completed monotherapy trial showing significant improvements in signs as measured by Corneal Staining Score, paving way for phase III trials in China in early 2020

Immunic Inc., of San Diego


Blocks dihydroorotate dehydrogenase

Relapsing-remitting multiple sclerosis

Final patient enrolled and randomized in Emphasis trial; top-line data expected during third quarter of 2020

Jazz Pharmaceuticals plc, of Dublin


Defibrotide sodium

Prevention of neurotoxicity in relapsed or refractory diffuse large B-cell lymphoma treated with CAR T therapy

First patient enrolled in exploratory study evaluating prevention of CAR T cell-associated neurotoxicity in patients receiving Yescarta (axicabtagene ciloleucel, Gilead Sciences Inc.)

Menlo Therapeutics Inc., of Redwood City, Calif.


Small-molecule selective NK1 receptor antagonist

Pruritus in psoriasis patients

Results showed statistically significant reduction in pruritus vs. placebo, with 33% of serlopitant patients achieving 4-point or greater improvement on worst-itch numeric rating scale at 8 weeks compared to baseline vs. 21% for placebo patients (p=0.028); 4-point responder rate at week 8 was higher in serlopitant-treated patients vs. placebo group when evaluating groups segmented by baseline characteristics: age, gender, weight, percentage of body surface area affected by psoriasis lesions, itch severity or severity of psoriasis

Phase III

Amag Pharmaceuticals Inc., of Waltham, Mass.

Vyleesi (bremelanotide injection)

Believed to act via melanocortin receptor agonism

Hypoactive sexual desire disorder

Pivotal data published in Obstetrics & Gynecology showed both trials met prespecified co-primary endpoints of improvement in desire and reduction in associated distress as measured by validated patient-reported outcome instruments

Brainstorm Cell Therapeutics Inc., of New York


Autologous MSC-NTF cells

Amyotrophic lateral sclerosis

Fully enrolled the 200-patient trial testing repeat intrathecal administration; expect all trial participants to complete visits and assessments by October 2020

Geron Corp., of Menlo Park, Calif.


Telomerase inhibitor

Lower-risk myelodysplastic syndromes

Dosed first of about 170 patients in Imerge study in transfusion-dependent patients who are relapsed after or refractory to erythropoiesis-stimulating agents; primary endpoint is 8-week transfusion-independence rate; top-line results expected by midyear 2022

Gilead Sciences Inc., of Foster City, Calif., and Galapagos NV, of Mechelen, Belgium


Oral JAK1 inhibitor

Moderately to severely active rheumatoid arthritis

Week 52 data from Finch 1 and Finch 3 studies consistent with and support efficacy and safety profiles in weeks 12 and 24 analyses

Horizon Therapeutics plc, of Dublin


Monoclonal antibody targeting IGF-1R

Active thyroid eye disease

Data from Optic confirmatory trial showed significant benefit vs. placebo on diplopia (68% vs. 29 % on placebo, p=0.001), quality of life (mean change of 13.79 on Graves' Ophthalmopathy Quality of Life vs. 4.43 for placebo, p<0.001) and clinical activity score (59% vs. 21% for placebo, p<0.001)

Kodiak Sciences Inc., of Palo Alto, Calif.


Anti-VEGF antibody biopolymer conjugate

Wet age-related macular degeneration

First patents treated in Dazzle pivotal study to test individualized dosing regimens, as infrequently as every 5 months and no more often than every 3 months, vs. Eylea (aflibercept, Regeneron Pharmaceuticals Inc.) every 8 weeks

Redhill Biopharma Ltd., of Tel Aviv, Israel


Oral antibiotic combination therapy

Crohn's disease

Full 52 week results from blinded treatment showed Map US study continued to meet primary endpoint of clinical remission vs. placebo (CDAI < 150) at week 26 (36.7% vs. 22.4%, p=0.0048), key secondary endpoints of maintenance of remission at weeks 16 and 52 (25.9% vs. 12.1%, p=0.0016) and durable clinical remission on all visits, week 16 through 52 (18.7% vs. 8.5%, p=0.0077)


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


No Comments