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 May 4, 2015

Alnylam Pharmaceuticals reported results of a phase II, open-label extension (OLE) study of patisiran for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropathy (FAP). The ongoing OLE study is an open-label, multicenter trial evaluating patisiran administration in FAP patients who previously were enrolled in a phase II study. Patisiran is being administered once every three weeks at a dose of 0.3mg/kg by intravenous infusion. Study results showed a mean 2.5 point decrease in modified Neuropathy Impairment Score (mNIS+7) at 12 months in patients who had reached the 12-month endpoint (N=20) at the time of data cutoff. This decrease in neuropathy progression compares favorably to the 13-to-18-point increase in mNIS+7 at 12 months that can be estimated from the literature in untreated FAP patients with similar baseline characteristics. In addition, patisiran treatment achieved a sustained mean serum TTR knockdown at the 80% target level for approximately 16 months, with an up to 88% mean knockdown achieved between doses. Patisiran also was found to be generally well-tolerated out to 17 months of drug administration, with no drug-related serious adverse events to date; all 27 patients enrolled in the study continue to receive patisiran.

Can-Fite BioPharma reported results of a phase II/III study of CF101 for moderate-to-severe plaque psoriasis. The double-blind, placebo-controlled study included 326 patients through 17 clinical centers in the U.S., Europe and Israel with a duration of 32 weeks. By 32 weeks of treatment with CF101, 33% of the patients achieved PASI 75, while the mean percent of improvement in PASI score was 57% (p<0.001). This was a statistically significant cumulative and linear improvement during weeks 16 to 32. Most significantly, by week 32 of the study, 20% of the study patients reached PASI 90, a result demonstrating a response rate of 90% clearing of skin lesions. Moreover, the PASI 90 subset analysis further suggests a higher and significant (p=0.026) CF101 response rate of 27% among patients previously untreated with systemic psoriasis therapy compared to patients pre-treated with systemic drugs. Can-Fite intends to continue the development of CF101 for the treatment of psoriasis and has initiated work on the design of the next advanced-stage clinical trial protocol.

Kamada issued results of a phase II/III study of inhaled alpha-1 antitrypsin (AAT) to treat AAT deficiency (AATD). For lung function, bronchial airflow measurements FEV1 (L) rose significantly in AAT treated patients and decreased in placebo treated patients (+15ml for AAT v. -27ml for placebo, a 42ml difference, p=0.0268). There was a trend toward better FEV1% predicted (0.54% for AAT v. -0.62% for placebo, a 1.16% difference, p=0.065). FEV1/SVC% rose significantly in AAT treated patients and decreased in placebo treated patients (0.62% for AAT vs. -0.87% for placebo, a 1.49% difference, p=0.0074). For lung function change at week 50 v. baseline, there was a trend toward reduced FEV1 (L) decline (-12ml for AAT v. -62ml for placebo, a 50ml difference, p=0.0956). There was a trend toward a reduced decline in FEV1% predicted (-0.1323% for AAT v. -1.6205% for placebo, a 1.4882% difference, p=0.1032). FEV1/SVC% rose significantly in AAT treated patients and decreased in placebo treated patients (0.61% for AAT v. -1.07% for placebo, a 1.68% difference, p=0.013). The company plans to submit the Marketing Authorization Application (MAA) to the EMA within the coming year. The company also plans to initiate discussions this year with the FDA on the regulatory pathway for registration of the product in the U.S.