BioBlast Pharma Announces Positive Final Results from HOPEMD Phase 2 Open-Label Clinical Study of Trehalose 90mg/mL IV Solution in Oculopharyngeal Muscular Dystrophy (OPMD) New Haven, Connecticut

BioBlast Pharma Announces Positive Final

Results from HOPEMD Phase 2 Open-Label Clinical Study of Trehalose 90mg/mL IV Solution in

Oculopharyngeal Muscular

New Haven, Connecticut - March

16, 2016 - BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced

positive results from its HOPEMD Phase 2 six-month open-label clinical study in patients with oculopharyngeal muscular dystrophy

(OPMD), a rare progressive muscle-wasting disease characterized by swallowing difficulties (dysphagia), leading to the risk of

aspiration of food into the lungs, weight loss, and generalized progressive muscle weakness. These results will be presented today

by Prof. Zohar Argov M.D., Senior Medical Advisor to BioBlast, during the plenary session at Myology 2016, a leading muscle conference

in Lyon, France, and, in April, 2016, during two general sessions at the American Academy of Neurology in Vancouver, Canada. See

information in this release regarding these scientific symposia.

The primary objective of the HOPEMD Phase

2 open-label study was to assess the safety and tolerability of trehalose 90mg/mL IV solution. Although not powered for efficacy,

secondary endpoints were included to explore if trehalose 90mg/mL IV solution could improve or prevent worsening of OPMD disease

markers. The study enrolled 25 patients with clinical dysphagia and muscle weakness at two centers in Canada and Israel. All patients

have now completed 24 weeks of weekly treatment with trehalose 90mg/mL IV solution and these are the data presented today.

Trehalose 90mg/mL IV solution was observed

to be safe and well-tolerated with no drug-related serious adverse events. Improvements versus baseline were observed in multiple

secondary efficacy endpoints related to dysphagia and muscle strength and function as further discussed below.

"The final study results are consistent

with the interim data previously reported and continue to look promising," stated Bernard Brais, MD, M.Phil., PhD, FRCP(C),

Professor, Departments 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. "With no pharmacotherapy

available to treat OPMD, trehalose 90mg/mL IV solution may be a step forward in caring for these patients. We look forward to confirming

these results in the planned Phase 2b double blind placebo controlled study."

HOPEMD Phase 2 Open-Label Clinical

Safety and Tolerability -

Trehalose 90mg/mL IV solution was observed to be safe and well tolerated with no drug-related serious adverse events reported.

There were no significant changes in lab safety data including chemistry, hematology, and ECG tests. There was one death due to

aspiration pneumonia that was not considered drug-related but instead, related to the underlying disease. No patients chose to

discontinue the study for reasons related to safety or side effects.

Dysphagia Tests (swallowing difficulties)

- The dysphagia endpoints were the timed cold water drinking test (80mL) for all sites, the nectar (80mL) and honey (80mL)

timed drinking tests at the Canadian site and the Penetration Aspiration Score as measured by video fluoroscopy (VFS-PAS), a radiographic

technique to determine the severity of swallowing difficulties and risk of aspiration. A patient reported swallowing quality of

life questionnaire (SWAL-QOL) specifically developed for patients suffering from swallowing problems was employed to assess the

degree to which patients felt that their swallowing capability improved with treatment.

There was a mean reduction in time to complete

the cold water drinking test of 31.8% versus baseline (n=23). In the nectar and honey timed drinking tests, time to complete was

reduced by 43.8% and 46.6% respectively (n=11). Out of the 11 patients in Canada whose scores were evaluated in the per protocol

analysis of the VFS-PAS, six patients improved (54.5%), two patients showed stabilization (18.2%), and three patients deteriorated

(27.2%). As previously reported, due to deviations from protocol and deficient radiological procedures, the VFS-PAS tests from

the Israel cohort were excluded from the final analysis. With respect to the SWAL-QOL questionnaire, there was a 12.7% (n=24) improvement

versus baseline with the mean total symptom severity score increasing from 43.2 to 48.7.

Muscle Strength Tests - As

measured quantitatively by a digital hand-held dynamometer, there was a mean increase in lower body muscle strength compared to

baseline in knee extension of 15.0% (n=22) and foot dorsiflexion of 22.4% (n=22). Hip flexion did not materially change (1.3% deterioration,

n=21). For the upper extremity strength tests, arm (bicep) flexion increased on average 17.9% (n=22), and shoulder abduction by

Muscle Function Tests - The

30 second arm-lift test showed a 16.0% increase in the number of completed tasks (n=20) at 24 weeks of treatment versus baseline

while the 30 second sit-to-stand test showed a 16.6% increase (n=21). The standard 4-stair climbing test did not materially change

(1.5% deterioration, n=21).

"We are encouraged by the safety

profile observed and the early efficacy signals noted in this relatively small study of OPMD patients. With this knowledge, we

can proceed to develop our double blind placebo controlled study to confirm these results and progress our clinical program to

registration," stated Warren Wasiewski M.D., Chief Medical Officer of BioBlast.

"These positive safety and efficacy

signals give us confidence to continue the development of trehalose 90mg/mL IV solution in OPMD," said Colin Foster, President

and Chief Executive Officer of BioBlast. "We look forward to the possibility of significantly helping patients afflicted

HOPEMD Phase 2 Open-Label Clinical

The HOPEMD study was designed as an initial

proof-of-concept open-label clinical study in 74 patients for 24 weeks, following which it was intended that all patients would

be randomized into a treatment arm or non-treatment control group, and followed for an additional 12 months in a separate continuation

study. The study was conducted at two centers - Montreal Neurological Institute at McGill University in Montreal, Canada,

and Hadassah-Hebrew University Medical Center in Jerusalem, Israel. The primary objective was to assess the safety and

tolerability of trehalose 90mg/mL IV solution in patients suffering from OPMD. Although not powered for efficacy, secondary endpoints

were included to explore if trehalose 90mg/mL IV solution could improve or prevent worsening of OPMD disease markers, notably those

related to dysphagia (difficulty in swallowing) and upper and lower muscle weakness. As previously reported, based on the interim

efficacy signals seen in the first 25 patients enrolled in Canada (11 patients) and Israel (14 patients), recruitment was terminated

in mid-2015. Now that all patients have received at least 24 weeks of treatment, BioBlast intends to discontinue the ongoing

continuation study and offer patients access to the drug in an expanded access program.

Scientific Symposia Information

Final results of the HOPEMD Phase 2 open-label

study will be presented at two upcoming scientific neurology conferences:

Plenary Session - Pharmacotherapy,

March 16, 2016 ~ 2:30pm-4:00pm

Presenter: Prof. Zohar Argov

M.D. (Jerusalem, Israel)

Title: Intravenous Trehalose

for Treatment of Dysphagia and Muscle Function in Oculopharyngeal Muscular Dystrophy (OPMD): Final Results of 24 Week Open-Label

Presenter: Prof. Zohar Argov

M.D. (Jerusalem, Israel)

Title: Intravenous Trehalose

for Treatment of Dysphagia and Muscle Function in Oculopharyngeal Muscular Dystrophy (OPMD): Final Results of 24 Week Open-Label

About Trehalose 90mg/mL IV Solution

Trehalose 90mg/mL IV solution 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.

Trehalose 90mg/mL IV solution has been documented as demonstrating significant promise in preclinical animal models of OPMD and

other PolyA/PolyQ diseases.

In OPMD, trehalose 90mg/mL IV solution

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 is an inherited myopathy characterized

by dysphagia (difficulty in swallowing), eyelid drooping (ptosis), the loss of muscle 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, and may suffer from repeated incidents of aspiration pneumonia. Aspiration pneumonia and

severe emaciation may result in death. The disease is caused by a genetic mutation responsible for the creation of a mutant protein