Phase I

Aerpio Pharmaceuticals Inc., of Cincinnati


Inhibits vascular endothelial protein tyrosine phosphatase

Primary open-angle glaucoma

Interim phase Ib data from first 3 cohorts showed topical ocular administration was well-tolerated over 7 days at all dose levels; time and dose-dependent reduction in intraocular pressure that, at highest QD dose, peaked at 4 hours post-dose (-1.47 mmHg; p=0.041)/-10.64%; p=0.027) and was sustained through 8 hours on day 7, returning to baseline levels at 24 hours post-dose; based on data, protocol amended to include patients with ocular hypertension and POAG; full results expected in first quarter of 2020

Alder Biopharma-ceuticals Inc., of Bothell, Wash.


Monoclonal antibody that inhibits pituitary adenylate cyclase-activating polypeptide


Dosed first patient in study as a preventive treatment; about 100 patients will be enrolled, with initial results expected in second half of 2020

Medivir AB, of Stockholm


Synthetic small molecule peptidomimetic of SMAC and inhibitor of IAP

Recurrent head and neck squamous cell carcinoma

First patient enrolled in study sponsored and financed as part of National Cancer Institute’s Cancer Therapy Evaluation Program; primary goals are to test safety of drug in combination with radiation therapy and to establish maximum tolerated dose

Proqr Therapeutics NV, of Leiden, the Netherlands

Sepofarsen (QR-110)

RNA-based oligonucleotide

Leber’s congenital amaurosis 10 due to the p.Cys998x mutation in the CEP290 gene

Top-line results from PQ-110-001 phase I/II study showed target dose (80 mcg with a 160-mcg loading dose) was associated with clinically meaningful and statistically significant improvement in vision, with favorable benefit/risk profile; phase II/III pivotal study ongoing

Phase II

Akari Therapeutics plc, of New York and London


C5 complement inhibitor

Bullous pemphigoid

Data showed daily subcutaneous doses were well-tolerated in 6 elderly patients; 4 of 6, who were classified as upper limit of moderate BP and only showed minor improvement on mometasone prior to initiation, saw mean 41% and 63% decline in Bullous Pemphigoid Disease Area Index (BPDAI) score at day 21 and 42, respectively, after treatment with nomacopan, and 59% and 68% decline in blister score at day 21 and 42, respectively; of 2 mild patients, 1 showed 100% decline in blisters by day 21, sustained to day 42, and decline in BPDAI score of 55% and 45% by day 21 and 42, respectively

Arcutis Biotherapeutics Inc., of Westlake Village, Calif.


Topical cream formulation of PDE4 inhibitor roflumilast

Atopic dermatitis

Completed enrollment of phase IIa trial of 136 adolescents, 12 and older, and adults; primary endpoint is change from baseline in Eczema Area and Severity Index Total Score at week 4

Frequency Therapeutics Inc., of Woburn, Mass.


Combination of small-molecule drugs designed to activate inner ear progenitor cells to induce hair cell regeneration

Sensorineural hearing loss

Dosing started in phase IIa trial to enroll about 96 adults with stable SNHL; key endpoints are word recognition, words-in-noise and standard pure tone audiometry; top-line data expected in second half of 2020

MGB Biopharma Ltd., of Glasgow, Scotland


Bactericidal antibiotic

Clostridium difficile-associated disease

First cohort of patients in phase IIa trial completed treatment at lowest dose, with results indicating high efficacy and good tolerability; safety committee reported no concerns, and recruitment of next cohort is progressing; headline results from all 3 cohorts expected in early 2020

Orasis Pharmaceuticals Ltd., of Herzliya, Israel

CSF-1 eye drop

Colony-stimulating factor 1


Met primary endpoint in phase IIb study, showing statistically significant improvement in distance-corrected near visual acuity of 3-line or greater gain; also showed safety and tolerability profile

Principia Biopharma Inc., of South San Francisco


Oral, small-molecule BTK inhibitor


Preliminary data from part B of study, testing 6 months of therapy, showed 6 of 15 patients (40%) reached complete response thus far, with 4 remaining on treatment who have potential to still reach CR; to date, 9 of 15 (60%) achieved a Pemphigus Disease Area Index score of 1 or 0

Phase III

Bryn Pharma LLC, of Raleigh, N.C.


Intranasal epinephrine spray


Completed dosing in pivotal study

Galderma SA, of Lausanne, Switzerland


IL-31-blocking monoclonal antibody

Moderate to severe atopic dermatitis

Enrolled first patient in study

Seattle Genetics Inc., of Bothell, Wash.


Oral, small-molecule selective HER2 inhibitor

Locally advanced or metastatic HER2-positive breast cancer

Dosed first patient in HER2Climb-02 trial testing tucatinib vs. placebo, in combination with standard-of-care Kadcyla (ado-trastuzumab emtansine, Roche Holding AG); primary endpoint is progression-free survival; about 460 patients to be enrolled


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

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