New Pivotal Study Reflects Positive Signals Reported From the Current HOPEMD Study, Which Will Be Closed Out at 25 Patients
TEL AVIV, Israel I June 15, 2015 I BioBlast Pharma Ltd. (ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today the start of a Phase 3 pivotal study in the USA and Canada for Cabaletta (trehalose) in Oculopharyngeal Muscular Dystrophy (OPMD).
Colin Foster, BioBlast’s President and CEO said, “We see positive signals in a consequential number of the 25 treated patients currently enrolled in our open label HOPEMD study, with respect to various muscle and other performance tests. These positive signals lead us to believe that the clinical study duration required to demonstrate efficacy may be considerably shorter than we initially predicted.
“Based upon various in vivo and cellular models of disease, we had initially expected to slow the deterioration of the disease over a period of 18 months in the HOPEMD study. Instead, we have seen evidence of improvement in a number of the functional measures within approximately the first six months.
“While we had expected that we would see a reduction in the rate of decline for these patients, we were very pleased to note in several muscle group strength tests, functional muscle tests, and the drinking test as well as several patient reported outcome measures, there were significant improvements over baseline levels. These are interim results and a randomized open label study should be cautiously interpreted. Nevertheless we are encouraged by these findings and hope to see that this positive signal continues as the study progresses.
“As a result, we have made the decision to initiate a 12 month, placebo-controlled, multi-center Phase 3 study in the next quarter that will begin enrolling approximately 60 OPMD patients in the USA and Canada into this new trial. We have stopped enrollment in the HOPEMD trial at twenty-five patients — we had originally intended to enroll 70 patients. New OPMD patients will now be enrolled in the Phase 3 pivotal study. We anticipate that this Phase 3 study will be completed by the end of 2016, and because of its shorter duration, will provide results at the same time as the HOPEMD study.
“Consequently, we believe this new Phase 3 study will expedite our OPMD program towards a regulatory filing.”
About Oculopharyngeal Muscular Dystrophy (OPMD)
OPMD is a rare inherited myopathy characterized by dysphagia (difficulty in swallowing), the loss of muscle strength and weakness in multiple parts of the body. Patients typically suffer from severe dysphagia, ptosis (eye lid drooping), tongue atrophy, proximal lower limb weakness, dysphonia (altered and weak voice), limitation in looking upward, facial muscle weakness and proximal upper limb weakness. The disease is most often diagnosed in the fifth-sixth decade of life and progresses throughout the patient’s life. As the dysphagia becomes more severe, patients become malnourished, lose significant weight, become dehydrated and suffer from repeated incidents of aspiration pneumonia. These last two are often the cause of death.
There is no medical treatment or, to our knowledge, potential cure for OPMD. Current therapeutic strategies are confined to surgical interventions that have limited efficacy and need to be repeated.
About BioBlast
BioBlast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. The company has a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable genetic orphan diseases.
The BioBlast platforms are based on deep understanding of the disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology. For more information please visit the Company’s website, www.bioblast-pharma.com, the content of which is not incorporated herein by reference.
SOURCE: BioBlast
Post Views: 203
New Pivotal Study Reflects Positive Signals Reported From the Current HOPEMD Study, Which Will Be Closed Out at 25 Patients
TEL AVIV, Israel I June 15, 2015 I BioBlast Pharma Ltd. (ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today the start of a Phase 3 pivotal study in the USA and Canada for Cabaletta (trehalose) in Oculopharyngeal Muscular Dystrophy (OPMD).
Colin Foster, BioBlast’s President and CEO said, “We see positive signals in a consequential number of the 25 treated patients currently enrolled in our open label HOPEMD study, with respect to various muscle and other performance tests. These positive signals lead us to believe that the clinical study duration required to demonstrate efficacy may be considerably shorter than we initially predicted.
“Based upon various in vivo and cellular models of disease, we had initially expected to slow the deterioration of the disease over a period of 18 months in the HOPEMD study. Instead, we have seen evidence of improvement in a number of the functional measures within approximately the first six months.
“While we had expected that we would see a reduction in the rate of decline for these patients, we were very pleased to note in several muscle group strength tests, functional muscle tests, and the drinking test as well as several patient reported outcome measures, there were significant improvements over baseline levels. These are interim results and a randomized open label study should be cautiously interpreted. Nevertheless we are encouraged by these findings and hope to see that this positive signal continues as the study progresses.
“As a result, we have made the decision to initiate a 12 month, placebo-controlled, multi-center Phase 3 study in the next quarter that will begin enrolling approximately 60 OPMD patients in the USA and Canada into this new trial. We have stopped enrollment in the HOPEMD trial at twenty-five patients — we had originally intended to enroll 70 patients. New OPMD patients will now be enrolled in the Phase 3 pivotal study. We anticipate that this Phase 3 study will be completed by the end of 2016, and because of its shorter duration, will provide results at the same time as the HOPEMD study.
“Consequently, we believe this new Phase 3 study will expedite our OPMD program towards a regulatory filing.”
About Oculopharyngeal Muscular Dystrophy (OPMD)
OPMD is a rare inherited myopathy characterized by dysphagia (difficulty in swallowing), the loss of muscle strength and weakness in multiple parts of the body. Patients typically suffer from severe dysphagia, ptosis (eye lid drooping), tongue atrophy, proximal lower limb weakness, dysphonia (altered and weak voice), limitation in looking upward, facial muscle weakness and proximal upper limb weakness. The disease is most often diagnosed in the fifth-sixth decade of life and progresses throughout the patient’s life. As the dysphagia becomes more severe, patients become malnourished, lose significant weight, become dehydrated and suffer from repeated incidents of aspiration pneumonia. These last two are often the cause of death.
There is no medical treatment or, to our knowledge, potential cure for OPMD. Current therapeutic strategies are confined to surgical interventions that have limited efficacy and need to be repeated.
About BioBlast
BioBlast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. The company has a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable genetic orphan diseases.
The BioBlast platforms are based on deep understanding of the disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology. For more information please visit the Company’s website, www.bioblast-pharma.com, the content of which is not incorporated herein by reference.
SOURCE: BioBlast
Post Views: 203
