Company Product Description Indication Status
Phase I
Xenon Pharmaceuticals Inc. of Burnaby, British Columbia, and Neurocrine Biosciences Inc., of San Diego XEN-901 Nav1.6 sodium channel inhibitor SCN8A-related epilepsy Study in healthy adults complete; developing a pediatric-specific, granule formulation with an IND expected in the middle of 2020
Phase II
Algernon Pharmaceuticals, of Vancouver, British Columbia NP-120 (Ifenprodil) N-methyl-d-aspartate receptor glutamate receptor antagonist Idiopathic pulmonary fibrosis and chronic cough Plans to announce the contract research organization and the principal investigator for the study shortly
Alkahest Inc., of San Carlos, Calif. GRF-6019  Plasma fraction Mild to moderate Alzheimer’s disease In the ALK6019-201 phase IIa study, patients taking drug for 6 months had no clinically detectable decline in 11-item AD assessment scale-cognitive subscale and the Mini-Mental State Examination and a small decline in AD Cooperative Study Activities of Daily Living scale 23-item version and the Clinical Dementia Rating scale Sum-of-Boxes scores
Bristol-Myers Squibb Co., of New York, and Bluebird Bio Inc., of Cambridge, Mass. Idecabtagene vicleucel (ide-cel; bb2121) CAR T targeting B-cell maturation antigen Relapsed and refractory multiple myeloma In the 140-patient Karmma study, overall response rate was 73.4% for the 128 patients treated with dose levels of 150-450 x 10^6 CAR+ T cells, including 31.3% of patients with complete responses; median progression-free survival was 8.6 months
Galectin Therapeutics Inc., of Norcross, Ga. Belapectin (GR-MD-02) Galectin-3 inhibitor Nonalcoholic steatohepatitis with cirrhosis and portal hypertension Data published in Gastroenterology showed treatment of 162 patients didn’t reduce HVPG or fibrosis, compared with placebo; in patients with esophageal varices, 2-mg/kg dose reduced HVPG and development of varices
Imaginab Inc., of Los Angeles 89Zr-Df-IAB22M2C CD8 tracer  Advanced and metastatic cancer patients Started study comparing tracer to standard-of-care scans and immunohistochemistry analysis of CD8 in biopsied tissues before and after immuno-oncology therapies
Kalvista Pharmaceuticals Inc., of Cambridge, Mass. KVD-001  Plasma kallikrein inhibitor Diabetic macular edema The 6-μg dose produced a +2.6 letters improvement in best corrected visual acuity vs. sham (p=0.223); 3-μg dose was +1.5 letters (p=0.465); no significant differences observed for central subfield thickness or the diabetic retinopathy severity scale
Marinus Pharmaceuticals Inc., of Radnor, Pa. Ganaxolone  Positive allosteric modulator of GABA-A Refractory status epilepticus All 6 patients met the primary endpoint of preventing progression to I.V. anesthetics within 24 hours of treatment initiation
Marinus Pharmaceuticals Inc., of Radnor, Pa. Ganaxolone  Positive allosteric modulator of GABA-A Seizures associated with tuberous sclerosis complex Plans to start an open-label study of 20-40 patients treated for 12 weeks after a 4-week baseline observation; primary endpoint will be seizure frequency
Phase III
Can-Fite Biopharma Ltd., of Petach Tikva, Israel Piclidenoson A3 adenosine receptor agonist Moderate to severe plaque psoriasis Comfort study enrolled more than 50% of the 407 planned patients
Greenwich Biosciences Inc., of Carlsbad, Calif, a subsidiary of GW Pharmaceuticals plc Epidiolex (cannabidiol) Duel cannabinoid CB1/CB2 receptor modulator Seizures associated with tuberous sclerosis complex Treatment with 25 mg/kg/day and 50 mg/kg/day reduced seizures by 49% and 48%, respectively, compared to a 27% reduction for placebo (p=0.0009 and p=0.0018, respectively); 36% and 40% of patients, respectively, had a 50% or greater reduction in seizures compared to 22% of patients taking placebo (p=0.0692 and p=0.0245, respectively)
Obseva SA, of Geneva Linzagolix  GnRH receptor antagonist Heavy menstrual bleeding due to uterine fibroids. In the 535-patient Primrose 2 study, responder rate was 93.9% for the 200-mg dose with hormonal add-back therapy (ABT) 56.7% for the 100 mg without ABT, compared to 29.4% for the placebo group (p<0.001 for both); both doses also met secondary endpoints of amenorrhea (p<0.001), reduction in pain (p<0.001), improvement in quality of life (p<0.001) and improvement in hemoglobin levels (p<0.002)
Ocugen Inc., of Malvern, Pa. OCU-300 Ophthalmic nanoemulsion of brimonidine tartrate Ocular graft-vs.-host disease Study reached 50% enrollment of the 60 planned patients; top-line results expected in the second half of 2020

Notes

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

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