Latest New Medical Therapy Trial Results

Following are the most recent results for new drug therapies currently in clinical trials worldwide. Results are also searchable by therapeutic area beginning with the most recent updates.

Week of April 13, 2015

GENFIT reported results of a phase IIb trial of GFT505 for nonalcoholic steatohepatitis (NASH). The 52-week trial enrolled 274 subjects (double-blind, placebo-controlled; three arms: placebo, 80mg, 120mg) with centrally-read, liver biopsy proven NASH. Treatment with GFT505 provided a significant beneficial effect on the primary endpoint (GFT505 120mg v. placebo, p=0.016, RR=2.03) in the global randomized population (n=274, full analysis set), in which patients without an end-of-treatment biopsy were considered as non-responders. The primary endpoint also was achieved in the evaluable population of patients who underwent both baseline and end-of-study liver biopsies (n=237, ITT; p=0.027 v. placebo; RR=1.94). In the evaluable patient population, GFT505 120mg also had a beneficial effect of a decrease of NAS-score =2 (p=0.04 v. placebo). The data provide the basis for upcoming phase III trials.

Ohr Pharmaceutical issued results of a phase II trial of OHR-102 (0.2% squalamine lactate ophthalmic solution) combination therapy for the treatment of the wet form of age-related macular degeneration (wet-AMD). The study was a nine-month trial enrolling 142 subjects with two treatment arms—OHR-102 drops administered twice daily plus Lucentis PRN v. placebo eye drops administered twice daily plus Lucentis PRN. In the intent-to-treat population with classic containing choroidal neovascularization (CNV) (OHR-102 n=38, Lucentis monotherapy n=32), 42% of the patients receiving OHR-102 achieved a =3 line gain at nine months, as compared to 28% in the Lucentis monotherapy group. Less of a benefit was seen in the overall population (classic containing and occult only CNV lesions). In patients with classic CNV, mean gains in visual acuity were +10.5 letters for the OHR- 102 combination arm and +5.4 letters with Lucentis monotherapy, a clinically meaningful benefit of +5.1 letters. The mean number of injections between the treatment arms, the primary endpoint of the study, was not meaningfully different. OHR-102 was generally well-tolerated, with only two treatment-related discontinuations in the study. These analyses will help guide and optimize the design of the phase III trials of OHR-102 in wet-AMD.

Portola Pharmaceuticals reported results from the second part of the phase III study of andexanet alfa with the Factor Xa inhibitor Eliquis (apixaban) in healthy volunteers. Andexanet alfa, a FDA-designated Breakthrough Therapy, was administered as an intravenous (IV) bolus followed by a continuous two-hour infusion to sustain the reversal of anti-coagulation activity. The randomized, double-blind, placebo-controlled study enrolled 31 healthy volunteers given apixaban 5mg twice daily for four days and then randomized in a 3:1 ratio to andexanet alfa administered as a 400mg IV bolus followed by a continuous infusion of 4mg/min for 120 minutes (n=23) or to placebo (n=8). Two to five minutes after completion of a bolus dose of andexanet alfa, the anticoagulant activity of apixaban was reversed by approximately 94% (p<0.0001) compared with placebo as measured by anti-Factor Xa activity. Every subject treated with andexanet alfa had between 90% and 96% reversal of the anticoagulant activity of apixaban. No serious adverse events, thrombotic events or antibodies to Factor X or Xa were reported. Portola plans to submit data from the study, and initial data from a phase IV study, as part of its BLA to the FDA under an Accelerated Approval pathway by the end of 2015.

RegeneRx Biopharmaceuticals reported results of a physician-initiated, phase II, randomized, placebo controlled study of RGN-259 in patients with severe dry eye. At day 56, the RGN-259-treated group had 35.1% reduction of ocular discomfort compared with vehicle control (P=0.0141), and 59.1% reduction of total corneal fluorescein staining compared with vehicle control (P = 0.0108). Other improvements seen in the RGN-259-treated patients included tear film breakup time and increased tear volume production. RGN-259 has been designated an orphan drug for the treatment of neurotrophic keratopathy (NK). RegeneRx recently was allowed by the FDA to move into phase III clinical trials with RGN-259 for the treatment of patients with NK. The drug candidate also is being studied in patients with dry eye syndrome in the U.S. and Asia.