BioBlast Pharma Announces Preclinical and Clinical Pharmacokinetic Results with Cabaletta, Interim Safety Results from Phase 2/3 HOPEMD Study in OPMD Interim Data on First 11 Patients in the Ongoing

BioBlast Pharma Announces Preclinical

and Clinical Pharmacokinetic Results with Cabaletta, Interim Safety Results from Phase 2/3 HOPEMD Study in OPMD

TEL AVIV, Israel, September 17, 2014 - BioBlast

Pharma Ltd (NasdaqGM: ORPN), a clinical-stage biotechnology company committed to developing clinically meaningful therapies for

patients with rare and ultra-rare genetic diseases, announced today positive preclinical and clinical pharmacokinetic (PK) data

on Cabaletta, its drug candidate for patients suffering from oculopharyngeal muscular dystrophy (OPMD). The Company also announced

interim safety results from the ongoing Phase 2/3 HOPEMD study in OPMD.

"We are encouraged with results of our pharmacokinetic

and safety studies," commented Dr. Dalia Megiddo, Chief Executive Officer of BioBlast. "We believe that these results

validate the effectiveness of our Cabaletta formulation and method of administration in delivering our drug into muscle cells for

prolonged periods of time. The human PK results are consistent with the preclinical data and altogether deepen our understanding

of the pharmacokinetics of Cabaletta and our ability to direct it to human tissues. The lack of any drug related adverse events

in the patients analyzed from the Phase 2/3 HOPEMD study is yet an additional reassurance as we move forward with our clinical

Cabaletta, BioBlast's clinical therapeutic platform is

based on Trehalose, a disaccharide known as an effective protein stabilizer and enhancer of autophagy. Prior to the development

of the Cabaletta unique formulation, there was no way to get Trehalose into patients' cells due to the natural enzymatic

processes that broke the disaccharide into glucose. The specific enzymes that catabolized Trehalose are abundant in the intestine,

blood, liver and kidneys.

BioBlast performed a study in SD (Sprague- Dawley) rats comparing

the pharmacokinetics of Trehalose after oral and IV administrations. All animals were treated either with 1g/kg of oral or IV trehalose.

The mean plasma exposure of Trehalose was 182 times higher after IV administration compared to oral administration. Cmax

(the highest measured drug concentration) after IV administration was 1379 microgram/ml compared to 4.28 microgram/ml after

oral administration. The half-life (T1 2) after IV administration was 2.07

hours compared to 0.74 hours for oral administration. The AUC (a measure of total drug exposure over time) after IV

administration was 781 microgramh/ml versus 4.52 microgramh/ml after oral administration. Importantly Trehalose was detected

in muscles in significant concentrations: AUC in muscles was 107 microgram*h/ml after IV administration. No trehalose was detected

in muscles after oral administration. Trehalose significant concentrations in muscles were detected for 48 hours with T1 2

Following the rat study BioBlast performed a pharmacokinetic

study in patients receiving Cabaletta as part of its multicenter international study for OPMD. The results of the pharmacokinetic

study in humans were found to be consistent to those found in rats and similar plasma exposure required for trehalose penetration

after IV administration to humans was 1.97 hours, similar to 2.07 observed in animals. The highest measured drug concentration

(Cmax) was 1579 microgram/ml - similar to 1379 microgram/ml found in animals.

AUC after IV administration to humans was 4863 hmicrogram/ml compared to 781 hmicrogram/ml found in animals.

Additionally, BioBlast released the results of its initial interim

safety and tolerability analysis of Cabaletta from the first 11 patients in the ongoing Phase 2/3 HOPEMD study. All patients

in this analysis received an average of 7 weekly infusion of the full study dose. There were no drug related adverse events and

all safety parameters including vital signs, blood biochemistry hematology and immunology and ECG were unchanged after close to

2 months of therapy. Specifically, glucose and insulin blood levels remained within normal ranges following Cabaletta administration.

An increased level of glucose in the urine (Glycosuria) was detected in all patients immediately following Cabaletta administration

signifying the effective clearance of trehalose by the kidneys.

One serious, non-drug related, adverse event was reported, a

urinary tract infection in a patient who had undergone a prior operation for kidney cyst. The patient fully recovered following

treatment with antibiotics. The patient continues to participate in the trial.

Cabaletta is 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 and other PolyA/PolyQ diseases.

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.

These last two are often the cause of death. The disease is caused by genetic mutation responsible for the creation of a mutant

unstable protein (PABPN1) that aggregates within patient's cell.

About BioBlast Pharma

BioBlast Pharma is a publicly traded, 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.

Forward-Looking Statements

This press release may contain 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 the potential of Cabaletta formulation

and delivery method to be effective, that we will move forward with our clinical program, or that our platforms potentially offer

solutions for several genetic diseases. Because such statements deal with future events and are based on Bio Blast Pharma Ltd.'s

current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Bio

Blast 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 Bio Blast Pharma's registration statement on Form F-1 filed with

the Securities and Exchange Commission ("SEC") and in any subsequent filings with the SEC. 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 referred to in this press release would be interpreted

differently in light of additional research and clinical and preclinical trials results. Except as otherwise required by law, Bio

Blast 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.