Bioblast Pharma Announces Collaboration with Team Sanfilippo to Evaluate Trehalose for the Treatment of Sanfilippo Syndrome

Tel Aviv, Israel, Feb. 14, 2019 (GLOBE NEWSWIRE) -- Bioblast Pharma Ltd. (NasdaqCM: ORPN) (“Bioblast”), a clinical-stage, orphan disease-focused biotechnology company, and Team Sanfilippo (“TSF”), a nonprofit medical research foundation founded by parents of children with Sanfilippo Syndrome today announced a clinical collaboration to evaluate the safety and efficacy of Bioblast’s proprietary Trehalose solution for the treatment of patients with Sanfilippo syndrome.

Under the terms of the collaboration, TSF plans to conduct an open label study in up to 20 Sanfilippo patients, which will be followed for safety and efficacy. If successful, based on the results of such study, Bioblast may determine to continue the development of Trehalose towards a pivotal study for this extra rare disease while maintaining full commercial rights. TSF received FDA approval for this study was last week.

"We are excited to be working with TSF to explore the potential of Trehalose in this rare and devastating disease,” said Dalia Megiddo, M.D., interim Chief Executive Officer of Bioblast. "Based on the known mechanism of action of Trehalose on diseased lysosomes, several preclinical studies and case reports, we have high expectations from this proof of concept study. We have great respect and admiration for the brave parents of Sanfilippo children on their fighting spirit. This program is currently expected to remain in place following Bioblast’s previously announced business combination with Enlivex Therapeutics Ltd., which is expected to close in the first quarter of this year. The current shareholders of Bioblast will have the opportunity to benefit from the potential success of this program, as well as potentially other programs for the Trehalose technology after the transaction with Enlivex closes and pursuant to the previously announced CVR agreement with Enlivex. 

“We view Trehalose as a great promise to our children, families and community," commented Kathleen Buckley, President of the New York-based Team Sanfilippo. “We are eager to start the study and plan to do so in the very near future. We have been following the Trehalose potential and programs closely for several years and feel that it could indeed make a difference in the life of our loved ones.”

About Trehalose
Trehalose is a protein stabilizer that also activates autophagy and crosses the blood-brain-barrier. Trehalose is a low molecular weight disaccharide (.342 kDa) that protects against pathological processes in cells. It has been shown to penetrate muscle and cross the blood brain barrier. In animal models of several diseases associated with abnormal cellular-protein aggregation, it has been shown to reduce pathological aggregation of misfolded proteins as well as to activate autophagy pathways through the activation of Transcription Factor EB (“TFEB”), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material. Trehalose has been studied in prior phase 2 clinical studies in over 70 patients with two rare diseases and was found both safe and effective.

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.  Bioblast is traded on the Nasdaq under the symbol "ORPN". For more information, please visit our website:, the content of which is not incorporated herein by reference.

About Team Sanfilippo

TSF is a nonprofit medical research foundation founded in 2008 by parents of children with Sanfilippo Syndrome. TSF’s mission is to fund potential therapies that can be in clinical trials in the near future. Team Sanfilippo is dedicated to provide assistance to families to gain access to clinical trials, treatments and compassionate use. Team Sanfilippo remains dedicated to getting children of all ages access to clinical trials and treatments and assistance to families enrolled in clinical trials.

About Sanfilippo Syndrome:

Sanfilippo Syndrome, or mucopolysaccharidosis III (MPS-III), is a rare autosomal recessive lysosomal storage disease. It is caused by a deficiency in one of the enzymes needed to break down the glycosaminoglycan (GAG) heparan sulfate (which is found in the extra-cellular matrix and on cell surface glycoproteins).

Affected children generally do not show any signs or symptoms at birth. In early childhood, they begin to develop developmental disability and loss of previously learned skills. In later stages of the disorder, they may develop seizures and movement disorders. Patients with Sanfilippo Syndrome usually live into adolescence or early adulthood.

According to [source], the number of new instances of Sanfilippo Syndrome is estimated at 1 in 70,000 live births and overall prevalence estimates range from 1 to 9 in 1,000,000 people. The number of children suffering from this syndrome is  estimated at very few thousands world wide.

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. Forward-looking statements can be identified by words such as “will,” “would,” “anticipate,” “expect,” “believe,” “plan,” “hope,” “estimates,” “suggests,” “has the potential to,” “projects”, “assumes”, “goal”, “targets”, “likely”, “should” or “intend,” and other words and phrases of similar meanings, the negative of these terms, and similar references to anticipated or expected events, activities, trends, future periods or results. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed or implied in our forward-looking statements.  For example, Bioblast is using forward-looking statements when it discusses the plan to initiate a clinical study to treat Sanfilippo Syndrome, potential success thereof and next steps assuming such success, the expected closing of the merger with Enlivex, the potential of the CVR agreement with Enlivex and potential for benefit to the Bioblast shareholders from the Trehalose program. Because such statements deal with future events and are based on Bioblast’s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Bioblast 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’s Annual Report on Form 20-F filed with the Securities and Exchange Commission (“SEC”) on April 24, 2018 and Exhibit 99.2 to Bioblast’s Report on Form 6-K furnished to the SEC on December 6, 2018, and in any subsequent filings with the SEC. Except as otherwise required by law, Bioblast 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.


Bioblast Pharma, Ltd.

Dr. Dalia Megiddo, CEO


 Kathleen Buckley , President