BioBlast Receives Notice of Allowance From USPTO Covering Cabaletta for the Treatment of OPMD

Tel Aviv, ISRAEL

TEL AVIV, Israel, Feb. 24, 2015 (GLOBE NEWSWIRE) -- BioBlast Pharma Ltd (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced today it has received a notice of allowance from the United States Patent and Trademark Office (USPTO) on a composition and method for the treatment of oculopharyngeal muscular dystrophy (OPMD) with Cabaletta. The Company has demonstrated efficacy in preclinical cell and animal models with Cabaletta, its lead therapeutic program and it is currently the subject of human clinical trials pursuing two indications.

"We are pleased to have received this notice of allowance which covers the significant innovations we have made in the stabilization of protein and prevention of protein aggregates within cells," stated Colin Foster, President & Chief Executive Officer of BioBlast. "Cabaletta is our most advanced platform, and is currently in Phase 2/3 for OPMD and Phase 2 for spinocerebellar ataxia type 3 (SCA3).  We have developed this platform based upon a unique formula of trehalose, a well-known and safe disaccharide. We look forward to making further progress toward providing a therapy to help OPMD patients in need of treatment."

OPMD is an inherited myopathy characterized by dysphagia (difficulty in swallowing) and the loss of muscular strength and weakness in multiple parts of the body. As the dysphagia becomes more severe, patients become malnourished, lose significant weight, become dehydrated and suffer from repeated incidents of aspiration pneumonia. Dehydration and aspiration pneumonia are often the cause of death. The disease is caused by a genetic mutation responsible for the creation of a mutant unstable protein (PABPN1) that aggregates within patient's muscle cells.

About Cabaletta

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 as well as acting as an autophagy enhancer. Cabaletta has been documented as demonstrating significant efficacy in preclinical animal models of OPMD and other PolyA/PolyQ diseases.

BioBlast's current HOPEMD Phase 2/3 clinical trial is aimed at testing the efficacy of Cabaletta to treat OPMD patients ( identifier: NCT02015481)

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. Founded in 2012, the company is rapidly building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable genetic 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.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. For example, we are using forward looking statements when we discuss making further progress toward providing a therapy to help OPMD patients, that our platforms potentially offer solutions for several diseases that share the same biological pathology, or when we may imply that the notice of allowance referred to above shall result in notice of issuance of a patent in the foreseeable future. In addition, historic results of scientific research and clinical and preclinical trials do not guarantee that the conclusions of future research or trials would not suggest different conclusions or that historic results would not be interpreted differently in light of additional research and clinical and preclinical trials results. Because such statements deal with future events and are based on BioBlast Pharma Ltd.'s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of BioBlast Pharma could differ materially from those described in or implied by the statements in this press release, including those discussed under the heading "Risk Factors" in BioBlast Pharma's prospectus dated July 30, 2014 filed with the Securities and Exchange Commission ("SEC") and in any subsequent filings with the SEC. Except as otherwise required by law, BioBlast Pharma disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.


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