BioBlast sets sights on PhIII after efficacy prompts early halt of mid-stage study

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October 29, 2015 | By Nick Paul Taylor

http://www.fiercebiotech.com/story/bioblast-sets-sights-phiii-after-efficacy-prompts-early-halt-mid-stage-stud/2015-10-29
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BioBlast Pharma ($ORPN) has posted a glimpse at the data that prompted it to wrap up a Phase II trial after enrolling just one-third of the targeted number of patients. And while there is very little data available publicly to support BioBlast’s optimism, the company thinks it has a strong enough hand to start discussing a Phase III trial with the FDA.

BioBlast CEO Colin Foster
BioBlast CEO Colin Foster

BioBlast also reported improvements in timed drinking tests, swallowing quality of life symptom scores and muscle strength, leading it to conclude that the ethical thing to do would be to wrap up the study early and power toward Phase III. However, in doing so, BioBlast has left itself with a set of data that has some limitations. While BioBlast enrolled 25 patients before halting recruitment, it only has Penetration Aspiration Scores for 12 participants because of failings at the Israeli trial site. The failings means BioBlast lacks Penetration Aspiration Scores for seven patients enrolled in Israel.Tel Aviv, Israel-based BioBlast stopped the open-label trial early on the basis of an interim analysis of 25 patients with oculopharyngeal muscular dystrophy (OPMD). The genetic disorder causes muscle wasting that ultimately renders people unable to swallow. To gauge whether its drug, Cabaletta, can help treat these symptoms, BioBlast x-rayed the mouths and throats of participants to see how deep material passed before and after they received the drug. Of the 12 patients assessed, 6 improved and 4 stabilized on the Penetration Aspiration Score 24 weeks after treatment with Cabaletta.

BioBlast also reported improvements in timed drinking tests, swallowing quality of life symptom scores and muscle strength, leading it to conclude that the ethical thing to do would be to wrap up the study early and power toward Phase III. However, in doing so, BioBlast has left itself with a set of data that has some limitations. While BioBlast enrolled 25 patients before halting recruitment, it only has Penetration Aspiration Scores for 12 participants because of failings at the Israeli trial site. The failings means BioBlast lacks Penetration Aspiration Scores for seven patients enrolled in Israel.