NEW HAVEN, Conn. and TEL AVIV, Israel, Oct. 26, 2015 (GLOBE NEWSWIRE) -- BioBlast Pharma Ltd., (Nasdaq:ORPN), a clinical-stage biotechnology company developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced that it will report the interim Phase 2 clinical study results of Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) on Tuesday, October 27th. Company management will host a conference call & webcast at 8:30am Eastern Time.
|Conference Call and Webcast Information|
|Tuesday, October 27 at 8:30a.m. Eastern Time|
|Replays available through November 10, 2015:|
A replay of the webcast can be accessed by visiting the "Investors" page of the company's website at http://bioblast-pharma.com/.
Cabaletta is a chemical chaperone that protects against pathological processes in cells. It has been shown to reduce 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, SCA3 and other PolyA/PolyQ diseases.
In OPMD, Cabaletta is being developed to prevent the aggregation of the pathological protein (PABPN1) in muscle cells, the hallmark of the disease, by stabilizing the protein, reducing the formation of protein aggregations, and promoting their clearance from cells through autophagy, thus preventing muscle cell death.
About Oculopharyngeal Muscular Dystrophy (OPMD)
OPMD is an inherited myopathy characterized by dysphagia (difficulty in swallowing) and the loss of muscular strength and weakness in multiple muscles of the body. Symptoms generally appear in mid-life and get worse over time. As the dysphagia becomes more severe, patients become malnourished, lose significant weight, become dehydrated and suffer from repeated incidents of aspiration pneumonia. Aspiration pneumonia and severe emaciation are frequently 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. There is currently no approved therapeutic treatment for OPMD.
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 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 understandings 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.
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 that our diverse portfolio of product candidates has the potential to address unmet medical needs for incurable genetic orphan diseases, or that our platforms potentially offer solutions for several diseases that share the same biological pathology. 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'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 Annual Report on Form 20-F filed with the Securities and Exchange Commission ("SEC") on March 31, 2015, 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.
CONTACT: U.S. Investor Contact: Matthew Duffy Managing Director LifeSci Advisors, LLC email@example.com (212) 915-0685 Media Relations Contact: Deanne Eagle Planet Communications firstname.lastname@example.org (917) 837-5866 Colin Foster President & CEO BioBlast Pharma Inc. email@example.com (475) 655-3032
Source: BioBlast Pharma