Announces Positive Interim Results from HOPEMD Phase 2 Clinical Study of Cabaletta in Oculopharyngeal Muscular

Announces Positive Interim Results from HOPEMD Phase 2 Clinical Study of Cabaletta in Oculopharyngeal Muscular

Conference Call and Webcast, 8:30 a.m.

New Haven, CT and Tel Aviv, Israel -

October 27, 2015 - BioBlast Pharma Ltd., (NasdaqGM: ORPN), a clinical-stage biotechnology company developing meaningful

therapies for patients with rare and ultra-rare genetic diseases, announced positive interim results from a Phase 2 open label

clinical study of its lead compound, Cabaletta (IV trehalose), in 25 patients with oculopharyngeal muscular

dystrophy (OPMD), a rare progressive muscle-wasting disease characterized by severe swallowing difficulties (dysphagia) leading

to malnutrition, dehydration, and aspiration of food into the lungs, as well as more generalized, progressive muscle weakness.

Aspiration pneumonia and severe emaciation are frequently the cause of death.

The HOPEMD study was designed as a proof-of-concept

open-label clinical study in 74 patients for 24 weeks, following which all patients would be randomized into a treatment arm or

non-treatment control group, and followed for an additional 12 months in a continuation study. The primary objective was to assess

the safety and tolerability of Cabaletta. Secondary endpoints were to determine if Cabaletta improves or prevents worsening of

OPMD disease markers. As previously reported, based on the positive signals seen in the first 25 patients enrolled in Canada and

Israel, further recruitment has been terminated, with the aim of beginning a Phase 3 study. These 25 patients remain in the Phase

2 study. To date, 22 patients have completed 24 weeks of treatment. Of these patients, 19 have now been randomized into the continuation

study. Although unplanned, an analysis was done on clinical data accumulated as of September 1, 2015 on these 25 patients.

this analysis, Cabaletta was observed to be safe and well-tolerated with no drug-related serious adverse events. Statistically

significant improvement or numerical improvement versus baseline, was observed on multiple efficacy endpoints. Ten out of 12 patients

(83.3%) were observed to stabilize or improve on an efficacy endpoint related to dysphagia, the Penetration Aspiration Score as

measured by Video Fluoroscopy (VFS-PAS). There was a statistically significant improvement of 35.3% (p<0.0001, n=20)

from baseline in the timed drinking test, also a key dysphagia

measure. In addition, patients demonstrated a statistically significant improvement of 12.1% (p=0.0448, n=21) in their swallowing

quality of life symptom score as measured by the SWAL-QOL questionnaire. Notably, there was a statistically significant correlation

between the timed drinking test and the SWAL-QOL percent change in symptom score (p=0.0479, n=20). With respect to muscle-related

efficacy endpoints, patients showed a statistically significant improvement of 13.4% (p=0.0059, n=19) in their lower extremities

muscle strength versus baseline, and showed numerical improvement in other muscle strength and function tests.

"The detailed analysis of the interim

results of the Phase 2 Cabaletta therapy trial for OPMD are very encouraging," stated Bernard Brais, MD, M.Phil., PhD, FRCP(C),

Professor, Department 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. "Since OPMD

is a chronically progressive muscular dystrophy, we felt it was optimistic to expect more than a stabilization of symptoms.

In fact, the data suggest that most participants improved across multiple clinical endpoints with significant swallowing improvements,

suggesting that the drug may lead to early benefits. These results are quite promising and I look forward to the confirmation

of the results in the planned placebo controlled Phase 3 study which could, for the first time, show rapid and chronic improvement

in a late-onset muscular dystrophy."

HOPEMD Study - Interim Safety

and Efficacy Results

The interim safety and efficacy results

of the Phase 2 open label study showed that, at up to 24 weeks of treatment (300mL of IV trehalose 9% solution once weekly), statistically

significant improvements or numerical improvements were observed in the following endpoints, including those that BioBlast plans

to incorporate into the Phase 3 protocol. All evaluable data were included in the statistical analysis of this interim report.

Safety and Tolerability

- Cabaletta was well tolerated with no drug related serious adverse events identified, a finding that is consistent with

BioBlast's previous report at the American Academy of Neurology meeting in April 2015. In particular there were no significant

changes in glucose, insulin homeostasis or patient weight. Cabaletta was well tolerated and no patients discontinued the study.

Penetration Aspiration Score

as Measured by Video Fluoroscopy (VFS-PAS) - Out of 12 patients whose scores were evaluated by VFS-PAS, a well-validated

radiographic technique to determine the severity of swallowing difficulties and risk of aspiration, 10 patients (83.3%) showed

stabilization (4 out of the 10) or improvement (6 out of the 10) in their VFS-PAS scores. Seven patients enrolled in Canada had

a baseline and 24-week VFS-PAS score. Five of the 7 patients had stable (3 out of the 7) or improved (2 out of the 7) VFS-PAS scores

while 2 patients deteriorated. Five patients enrolled in Israel who did not have a scheduled baseline score had an unscheduled

VFS-PAS evaluation performed after being on treatment for varying amounts of time ranging from one to four months. The results

of this unscheduled evaluation were compared to the results obtained after 24 weeks of therapy. All 5 patients enrolled in Israel

were stable (2 out of 5) or improved (3 out of 5). Seven baseline VFS-PAS measurements recorded in Israel were uninterpretable

due to faulty radiological procedure and were excluded from the interim data set.

Timed Drinking Test -

There was a statistically significant 35.3% improvement versus baseline (p<0.0001) in the timed drinking test (80mL cold water),

a frequently used and validated clinical test of swallowing dysfunction. The improvement in the timed drinking test correlated

with patient reported severity of their symptoms, further attesting to the clinical meaningfulness of the result (correlation=-0.4431,

p=0.0504 for absolute change, and correlation=-0.4474, p=0.0479 for % change in symptom severity, n=20).

Swallowing Quality of Life

(SWAL-QOL) - The well-validated SWAL-QOL questionnaire, specifically developed for patients suffering from problems

in swallowing, demonstrated a statistically significant improvement in the mean reported symptom severity score from baseline through

to 24 weeks. Total mean symptom severity score improved from 54.0 points at baseline to 61.4 points at 24 weeks (12.1% improvement),

Muscle Strength Tests

- The composite score of three lower extremity muscle strength tests showed a statistically significant improvement over

baseline of 13.4% (p=0.0059, n=19). This composite reflects a statistically significant improvement of 13.9% in knee extension

(p=0.0058), and 24.3% in foot extension (p=0.0381). Hip flexion did not achieve statistical significance but had a numerical improvement

The composite score of two upper extremity muscle strength tests (comprised of shoulder abduction and arm flexion)

showed a numerical improvement of 12.9% versus baseline, but did not achieve statistical significance (p=0.0836, n=19).

Functional Muscle Tests

- The 30 second arm-lift test showed a statistically significant improvement of 17.6% (p=0.0191, n=18) while the 30

second sit-to-stand test showed a numerical improvement of 14.1% versus baseline (p=0.0682, n=18). Notably, this latter test showed

a statistically significant improvement in absolute numbers with a p=0.0160. In the standard 4-stair climbing test, importantly,

no deterioration was observed but there was no statistically significant improvement.

"We are encouraged by these interim

results of the HOPEMD open label Phase 2 study," stated Dalia Megiddo, MD, BioBlast's Chief Development Officer. "Of

particular note, statistically significant improvements or positive changes were observed in disease features that lead to the

major disabilities of OPMD (swallowing and muscle weakness). The two tests performed to evaluate dysphagia (difficulties with swallowing),

both showed meaningful improvements over baseline and are supported by patient reported severity of their symptoms. Together with

the improvements and positive changes in muscle strength and muscle function tests, these results indicate the potential efficacy

of Cabaletta in changing the course of this inexorable and debilitating disease.

"The totality of the data, with endpoints

showing numerical and/or statistical improvement from baseline, gives us confidence that the proof of concept for Cabaletta in

OPMD patients has been demonstrated," Dr. Megiddo concluded.

"The OPMD community currently has

very limited treatment options and there is a critical unmet need for patients with this rare disorder," stated Peter L.

Saltonstall, the President and CEO of the National Organization for Rare Disorders (NORD). "We are grateful to BioBlast

and to the physicians and patients who have committed their time and resources to this important clinical trial. New

treatments, combined with further understanding and awareness of OPMD, can bring much needed hope to those treating and living

with this devastating condition."

"These results support our plan to

move forward with a pivotal Phase 3 study in OPMD following pending meetings with regulatory authorities," said Colin Foster,

President and Chief Executive Officer, BioBlast. "We plan to share these interim data and the complete Phase 2 six month

data set with the FDA. Following these interactions and based on the open IND, Orphan Drug designation, and Fast Track designation

we have been granted for this program, our goal is to advance with a Phase 3 study in the U.S. and in Canada.

"The interim results for OPMD, specifically

with respect to dysphagia and muscle strength and function, give us insight into the potential of Cabaletta for use in other protein

aggregation-related diseases, such as spinocerebellar ataxia type 3 (SCA3 or Machado Joseph disease), another devastating hereditary