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 February 13, 2017

Biofrontera reported results of a phase III trial of BF-200 ALA (Ameluz) in combination with daylight photodynamic therapy (PDT). The intra-individual, randomized, observer-blind, multicenter study was performed with 52 patients at seven centers in Spain and Germany. Every patient had three to nine mild to moderate AK lesions in each of two comparable treatment areas on the face and/or scalp. For an intra-patient comparison of the treatments, each patient received daylight-PDT with BF-200 ALA on one side, and MAL on the other side. The study will be followed by assessments of lesion recurrence six and 12 months after daylight PDT. The study met its primary endpoint, resulting after a single daylight PDT in 79.8% total lesion clearance rate per patient’s side in the areas treated with BF-200 ALA and daylight PDT, compared to 76.5% total lesion clearance in areas treated with MAL and daylight PDT. For comparison, the much larger study ALA-AK-CT002, which compared the two products and demonstrated superiority of BF-200 ALA over MAL, resulted in lesion complete clearance rates after the first PDT with narrow-spectrum lamps of 77.1% with BF-200 ALA and 73.0% with MAL, respectively. These results will be employed for the filing of the EU label extension, which Biofrontera plans to submit in the second quarter of 2017.

Bioverativ and Swedish Orphan Biovitrum issued results of a phase III study of ALPROLIX in previously-treated children with severe hemophilia B. Kids B-LONG was a global, open-label, multicenter study involving 30 boys under the age of 12 with severe hemophilia B (factor IX activity equal to or less than 2 IU per dL, or 2%) with at least 50 prior exposure days to factor IX therapies. To date, Kids B-LONG is the largest study to evaluate extended half-life factor IX therapy in children with hemophilia B. The study was conducted at 16 hemophilia treatment centers in Australia, Hong Kong, Ireland, the Netherlands, South Africa, the U.K. and the U.S. Overall, 27 participants (90%) completed the study. The median time participants spent in the study was 49.4 weeks, and 24 participants received ALPROLIX infusions on at least 50 separate days (exposure days). In this study, no participants developed inhibitors to ALPROLIX. ALPROLIX was well-tolerated and adverse events (AEs) observed were typical of the pediatric hemophilia B population. The most common AEs were common cold (n=7, 23%) and fall (n=6, 20%). Four participants experienced serious AEs during the study, all of which were assessed as unrelated to ALPROLIX by the investigators. In the study, there were no reports of anaphylaxis or serious hypersensitivity reactions to ALPROLIX, no vascular thrombotic events and no deaths. Children (n=30) treated prophylactically with ALPROLIX had a median ABR of 2.0 overall and zero spontaneous joint bleeds. Of all patients treated, 10 of 30 (33%) experienced no bleeding episodes, and 19 of 30 (63%) reported no joint bleeding on-study. Overall, 92% of bleeding episodes were controlled by one or two infusions of ALPROLIX. Following a switch to ALPROLIX therapy, 80% of children extended their dosing interval compared to previous treatment, and nearly all remained on once-weekly prophylactic dosing throughout the study. 

Envisia Therapeutics released results of a phase II study of ENV515 (travoprost XR) in patients with glaucoma. The ongoing second cohort of the phase II trial is a 12-month safety and efficacy evaluation that enrolled five glaucoma patients at sites within the U.S. The pre-washout baseline for all patients in this cohort, treated with LUMIGAN or XALATAN prior to enrollment, was 19.7mmHg, with a post-washout baseline of 26.1mmHg for eight AM intraocular pressure (IOP). A single low dose of ENV515 decreased the mean + SD 8 AM IOP by 6.7 ± 3.7 mmHg or 25% over 11 months (mean of all 8 AM IOPs over eleven months). The mean 8 AM IOP after a single low dose of ENV515 was 19.5 mmHg over the 11-month period. ENV515 also demonstrated an IOP lowering effect comparable to prestudy topical prostaglandin analogs (XALATAN and LUMIGAN) and in-study topical timolol maleate 0.5% ophthalmic solution (daily eye drops). There were no serious adverse events and the most common adverse event was early-onset transient hyperemia, or eye redness, related to the dosing procedure.