Positive interim results from clinical study of Cabaletta in OPMD

On October 27, 2015 BioBlast Pharma Ltd. announced positive interim results from a Phase 2 open label clinical study of Cabaletta®, in 25 patients with oculopharyngeal muscular dystrophy (OPMD).

OPMD

OPMD is a rare progressive muscle-wasting disease characterized by severe swallowing difficulties leading to malnutrition, dehydration, and aspiration of food into the lungs, as well as more generalized, progressive muscle weakness. Aspiration pneumonia and severe emaciation are frequently the cause of death.

Study

The study was designed as a proof-of-concept open-label clinical study in 74 patients for 24 weeks, following which all patients would be randomized into a treatment arm or non-treatment control group, and followed for an additional 12 months in a continuation study. The primary objective was to assess the safety and tolerability of Cabaletta. Secondary endpoints were to determine if Cabaletta improves or prevents worsening of OPMD disease markers.

As previously reported, based on the positive signals seen in the first 25 patients enrolled in Canada and Israel, further recruitment has been terminated, with the aim of beginning a Phase 3 study.

Although unplanned, an analysis was done on clinical data accumulated as of September 1, 2015 on these 25 patients.

• Cabaletta was observed to be safe and well-tolerated with no drug-related serious adverse events. 
• Statistically significant improvement or numerical improvement versus baseline, was observed on multiple efficacy endpoints related to dysphagia.
• Patients showed a statistically significant improvement in their lower extremities muscle strength versus baseline, and showed numerical improvement in other muscle strength and function tests.

"The detailed analysis of the interim results of the Phase 2 Cabaletta therapy trial for OPMD are very encouraging," stated Bernard Brais, MD, M.Phil., PhD, FRCP(C), Professor, Department of Neurology and Neurosurgery and Human Genetics, Faculty of Medicine, McGill University, Co-director Rare Neurological Diseases Group at the Montreal Neurological Institute, and Principal Investigator in the study. "Since OPMD is a chronically progressive muscular dystrophy, we felt it was optimistic to expect more than a stabilization of symptoms. In fact, the data suggest that most participants improved across multiple clinical endpoints with significant swallowing improvements, suggesting that the drug may lead to early benefits. These results are quite promising and I look forward to the confirmation of the results in the planned placebo controlled Phase 3 study which could, for the first time, show rapid and chronic improvement in a late-onset muscular dystrophy."

Cabaletta and SCA3

"The interim results for OPMD, specifically with respect to dysphagia and muscle strength and function, give us insight into the potential of Cabaletta for use in other protein aggregation-related diseases, such as spinocerebellar ataxia type 3 (SCA3 or Machado Joseph disease), another devastating hereditary disease in which we are planning a pivotal Phase 3 study in the U.S. and E.U.," Mr. Foster continued.   

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 Press release BioBlast.       

BioBlast Pharma receives orphan drug designation for the treatment of SCA3

Press release of 19 november 2014



BioBlast Pharma announced today that it has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) for Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (SCA3, Machado Joseph disease).


Cabaletta is a chemical chaperone that protects against pathological processes in cells. It has been shown to prevent pathological aggregation of proteins within cells in several diseases associated with abnormal cellular-protein aggregation. Cabaletta has demonstrated efficacy in preclinical cells and animal models of SCA3 and other PolyA/PolyQ diseases, including Oculopharyngeal Muscular Dystrophy (OPMD) and Spino bulbar muscular atrophy. BioBlast plans to make clinical progress in each of these indications in 2015.


"The orphan drug designation is yet another step on BioBlast’s route to create and capture value from our research and clinical work,” stated Dalia Megiddo, MD MBA, Chief Executive Officer of BioBlast. “We currently are conducting a phase 2 clinical trial to test the efficacy of Cabaletta in SCA3 and plan to start our SCA3 pivotal study in 2015.”

About Orphan Drug Designation

Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waives the Prescription Drug User Fee Act (PDUFA) filing fees.

About BioBlast Pharma

BioBlast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. Founded in 2012, the company is rapidly building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable 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.