<SEC-DOCUMENT>0001144204-16-088498.txt : 20160316
<SEC-HEADER>0001144204-16-088498.hdr.sgml : 20160316
<ACCEPTANCE-DATETIME>20160316163048
ACCESSION NUMBER:		0001144204-16-088498
CONFORMED SUBMISSION TYPE:	6-K
PUBLIC DOCUMENT COUNT:		3
CONFORMED PERIOD OF REPORT:	20160316
FILED AS OF DATE:		20160316
DATE AS OF CHANGE:		20160316

FILER:

	COMPANY DATA:	
		COMPANY CONFORMED NAME:			BIO BLAST PHARMA LTD.
		CENTRAL INDEX KEY:			0001596812
		STANDARD INDUSTRIAL CLASSIFICATION:	PHARMACEUTICAL PREPARATIONS [2834]
		IRS NUMBER:				000000000
		STATE OF INCORPORATION:			L3
		FISCAL YEAR END:			1231

	FILING VALUES:
		FORM TYPE:		6-K
		SEC ACT:		1934 Act
		SEC FILE NUMBER:	001-36578
		FILM NUMBER:		161510066

	BUSINESS ADDRESS:	
		STREET 1:		37 DEREH MENACHEM BEGIN ST.
		STREET 2:		15TH FLOOR
		CITY:			TEL AVIV
		STATE:			L3
		ZIP:			6522042
		BUSINESS PHONE:		972 (0) 3 7326616

	MAIL ADDRESS:	
		STREET 1:		37 DEREH MENACHEM BEGIN ST.
		STREET 2:		15TH FLOOR
		CITY:			TEL AVIV
		STATE:			L3
		ZIP:			6522042
</SEC-HEADER>
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<TYPE>6-K
<SEQUENCE>1
<FILENAME>v434440_6k.htm
<DESCRIPTION>FORM 6-K
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<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; text-align: center; margin-bottom: 0pt">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">UNITED STATES</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">SECURITIES AND EXCHANGE COMMISSION</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">Washington, D.C. 20549</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">Form 6-K</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">Report of Foreign Private Issuer</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">Pursuant to Rule 13a-16 or 15d-16</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">under the Securities Exchange Act of 1934</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">For the month of: March 2016</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">Commission file number: 001-36578</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center"><U>BIOBLAST PHARMA LTD.</U></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">(Translation of registrant's name into English)</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">37 Dereh Menechem Begin St., 15<SUP>th</SUP>
Floor</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center"><U>Tel Aviv 6522042 Israel</U></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center">(Address of principal executive offices)</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">Indicate by check mark whether the registrant files or will
file annual reports under cover of Form 20-F or Form 40-F.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">Form 20-F &nbsp;<FONT STYLE="font-family: Wingdings">x
</FONT>Form 40-F <FONT STYLE="font-family: Wingdings">&uml;</FONT></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">Indicate by check mark if the registrant is submitting the Form
6-K in paper as permitted by Regulations S-T Rule 101(b)(1):_____</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">Indicate by check mark if the registrant is submitting the Form
6-K in paper as permitted by Regulations S-T Rule 101(b)(7):_____</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0"></P>

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<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0"><B>&nbsp;</B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: center"><B><U>CONTENTS</U></B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">This Report of Foreign Private Issuer on
Form 6-K of the Registrant consists of the press release issued by the Registrant on March 16, 2016, announcing final results from
its HOPEMD Phase 2 open-label clinical study of trehalose 90mg/mL IV solution in oculopharyngeal muscular dystrophy, which is attached
hereto as Exhibit 99.1.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">The first sentence of the first paragraph,
the second and third paragraphs, the fifth through the ninth paragraphs, the twelfth paragraph and the paragraph titled &ldquo;Forward
Looking Statements&rdquo; are incorporated by reference into the registration statement on Form F-3 (File No. 333-206032) and
the registration statement on Form S-8 (File No. 333-203114) of the Registrant, filed with the Securities and Exchange Commission,
to be a part thereof from the date on which this report is submitted, to the extent not superseded by documents or reports subsequently
filed or furnished.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0"><B>Exhibit No.</B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in">&nbsp;</P>

<TABLE CELLSPACING="0" CELLPADDING="0" STYLE="font: 10pt Times New Roman, Times, Serif; width: 100%">
<TR STYLE="vertical-align: top">
    <TD STYLE="width: 15%; font-size: 10pt"><FONT STYLE="font-family: Times New Roman, Times, Serif; font-size: 10pt">99.1</FONT></TD>
    <TD STYLE="width: 85%; font-size: 10pt"><FONT STYLE="font-family: Times New Roman, Times, Serif; font-size: 10pt">Press Release issued by BioBlast Pharma Ltd. on March 16, 2016, announcing final results from its HOPEMD Phase 2 open-label clinical study of trehalose 90mg/mL IV solution in oculopharyngeal muscular dystrophy.</FONT></TD></TR>
</TABLE>
<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in; background-color: white">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in; background-color: white"></P>

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<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-indent: 0.5in; background-color: white">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center"><B><U>SIGNATURES</U></B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">Pursuant to the requirements of the Securities Exchange Act
of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">&nbsp;</P>

<TABLE CELLSPACING="0" CELLPADDING="0" STYLE="font: 10pt Times New Roman, Times, Serif; width: 100%">
<TR STYLE="vertical-align: top">
    <TD STYLE="width: 30%">&nbsp;</TD>
    <TD STYLE="width: 10%">&nbsp;</TD>
    <TD STYLE="width: 60%">
        <P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0"><U>BioBlast Pharma Ltd. </U><BR>
(Registrant)</P>
        <P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0"><BR>
        <U>By /s/ Colin Foster </U></P></TD></TR>
<TR STYLE="vertical-align: top">
    <TD>&nbsp;</TD>
    <TD>&nbsp;</TD>
    <TD><FONT STYLE="font-family: Times New Roman, Times, Serif; font-size: 10pt">Name: Colin Foster</FONT></TD></TR>
<TR STYLE="vertical-align: top">
    <TD>&nbsp;</TD>
    <TD>&nbsp;</TD>
    <TD>&nbsp;</TD></TR>
<TR STYLE="vertical-align: top">
    <TD>&nbsp;</TD>
    <TD>&nbsp;</TD>
    <TD><FONT STYLE="font-family: Times New Roman, Times, Serif; font-size: 10pt">Chief Executive Officer and President</FONT></TD></TR>
</TABLE>
<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">Date: March 16, 2016</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0">&nbsp;</P>

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<DESCRIPTION>EXHIBIT 99.1
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<P STYLE="margin: 0; text-align: right"><B>Exhibit 99.1</B></P>

<P STYLE="margin: 0; text-align: right">&nbsp;</P>

<P STYLE="margin: 0; text-align: right"></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center"><IMG SRC="image_001.jpg" ALT=""></P>



<P STYLE="margin: 0; text-align: right"></P>

<P STYLE="margin: 0; text-align: right">&nbsp;</P>

<P STYLE="margin: 0; text-align: right"></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center"><B>BioBlast Pharma Announces Positive Final
Results from HOPEMD Phase 2 Open-Label Clinical Study of Trehalose 90mg/mL IV Solution in </B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: center; text-indent: 0.5in"><B>Oculopharyngeal Muscular
Dystrophy (OPMD)</B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: center"><B>&nbsp;</B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><B>New Haven, Connecticut &ndash; March
16, 2016</B> &ndash; 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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&ldquo;The final study results are consistent
with the interim data previously reported and continue to look promising,&rdquo; 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. &ldquo;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.&rdquo;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><B><I>HOPEMD Phase 2 Open-Label Clinical
Study - Results</I></B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><U>Safety and Tolerability</U> &ndash;
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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><U>Dysphagia Tests (swallowing difficulties)</U>
&ndash; 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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"></P>

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<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><U>Muscle Strength Tests </U>&ndash; 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
11.4% (n=22).</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><U>Muscle Function Tests</U> &ndash; 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).</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&ldquo;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,&rdquo; stated Warren Wasiewski M.D., Chief Medical Officer of BioBlast.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&ldquo;These positive safety and efficacy
signals give us confidence to continue the development of trehalose 90mg/mL IV solution in OPMD,&rdquo; said Colin Foster, President
and Chief Executive Officer of BioBlast. &ldquo;We look forward to the possibility of significantly helping patients afflicted
with this disease.&rdquo;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><B><I>HOPEMD Phase 2 Open-Label Clinical
Study - Design</I></B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">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&nbsp;at&nbsp;McGill University&nbsp;in Montreal,&nbsp;Canada,
and Hadassah-Hebrew University Medical Center&nbsp;in&nbsp;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.&nbsp; 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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"></P>

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<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><B><I>Scientific Symposia Information</I></B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">Final results of the HOPEMD Phase 2 open-label
study will be presented at two upcoming scientific neurology conferences:</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<TABLE CELLPADDING="0" CELLSPACING="0" WIDTH="100%" STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt"><TR STYLE="vertical-align: top">
<TD STYLE="width: 0.25in"></TD><TD STYLE="width: 0.25in">1.</TD><TD STYLE="text-align: justify">5<SUP>th</SUP> International Congress of Myology 2016, March 14-18, 2016 in Lyon, France</TD></TR></TABLE>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0 0pt 0.5in; text-align: justify">Plenary Session &ndash; Pharmacotherapy,
March 16, 2016 ~ 2:30pm-4:00pm</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0 0pt 0.5in; text-align: justify">Presenter: Prof. Zohar Argov
M.D. (Jerusalem, Israel)</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0 0pt 0.5in; text-align: justify">Title: Intravenous Trehalose
for Treatment of Dysphagia and Muscle Function in Oculopharyngeal Muscular Dystrophy (OPMD): Final Results of 24 Week Open-Label
Phase 2 Trial</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<TABLE CELLPADDING="0" CELLSPACING="0" WIDTH="100%" STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt"><TR STYLE="vertical-align: top">
<TD STYLE="width: 0.25in"></TD><TD STYLE="width: 0.25in">2.</TD><TD STYLE="text-align: justify">American Academy of Neurology 2016 Annual Meeting, April 15-21 in Vancouver, Canada</TD></TR></TABLE>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0 0pt 0.5in; text-align: justify">Presentations:</P>

<TABLE CELLPADDING="0" CELLSPACING="0" WIDTH="100%" STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt"><TR STYLE="vertical-align: top">
<TD STYLE="width: 0.75in"></TD><TD STYLE="width: 0.25in"><FONT STYLE="font-family: Symbol">&middot;</FONT></TD><TD STYLE="text-align: justify"><FONT STYLE="font-family: Times New Roman, Times, Serif">Integrated Neuroscience Session &ndash;
Advances in Acquired and Genetic Muscle Diseases, April 17, 2016 ~ 5:00pm </FONT></TD></TR></TABLE>

<TABLE CELLPADDING="0" CELLSPACING="0" WIDTH="100%" STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt"><TR STYLE="vertical-align: top">
<TD STYLE="width: 0.75in"></TD><TD STYLE="width: 0.25in"><FONT STYLE="font-family: Symbol">&middot;</FONT></TD><TD STYLE="text-align: justify"><FONT STYLE="font-family: Times New Roman, Times, Serif">Experimental Therapies in Neuromuscular
Diseases, April 19, 2016 ~ 7:15am</FONT></TD></TR></TABLE>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0 0pt 0.5in; text-align: justify">Presenter: Prof. Zohar Argov
M.D. (Jerusalem, Israel)</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0 0pt 0.5in; text-align: justify">Title: Intravenous Trehalose
for Treatment of Dysphagia and Muscle Function in Oculopharyngeal Muscular Dystrophy (OPMD): Final Results of 24 Week Open-Label
Phase 2 Trial</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><B><I>About Trehalose 90mg/mL IV Solution</I></B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><B><I>About Oculopharyngeal Muscular Dystrophy
(OPMD)</I></B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">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
(PABPN1) with an expanded polyalanine domain that aggregates within patient muscle cells. There is currently no approved pharmacologic
treatment for OPMD.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><B><I>About BioBlast Pharma Ltd. </I></B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">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
has a portfolio of product candidates with the potential to address unmet medical needs for incurable diseases. The BioBlast platforms
are based on a deep understanding of disease-causing biological processes, and potentially offer solutions for several diseases
that share the same biological pathology. BioBlast was founded in 2012 and is traded on the NASDAQ under the symbol &quot;ORPN&quot;.
For more information, please visit the Company's website, www.bioblast-pharma.com, the content of which is not incorporated herein
by reference.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"></P>

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<P STYLE="font: 10pt Times New Roman, Times, Serif; margin-top: 0pt; margin-bottom: 0pt; text-align: center"><IMG SRC="image_001.jpg" ALT="">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify"><B><I>Forward Looking Statements </I></B></P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">This press release contains forward-looking
statements within the meaning of the &quot;safe harbor&quot; provisions of the Private Securities Litigation Reform Act of 1995
and other Federal securities laws. For example, the Company is using forward looking statements when it discusses, confirming results
of its HOPEMD clinical study in a future clinical study, if such study is commenced and concluded at all, and the design thereof,
future efforts and plans to develop trehalose 90mg/mL IV solution, potential uses of its product candidates for various indications,
building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases, or
that BioBlast&rsquo;s 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 those historic results referred to in this press release would not be interpreted
differently in light of additional research and clinical and preclinical trial results. Because such statements deal with future
events and are based on BioBlast Pharma Ltd.'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 &quot;Risk Factors&quot; in BioBlast Pharma's Annual Report
on Form 20-F filed with the Securities and Exchange Commission (&quot;SEC&quot;) 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.</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">&nbsp;</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0"><B>INVESTOR CONTACT: </B><BR>
Matthew P. Duffy</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">Managing Director</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">LifeSci Advisors, LLC</P>

<P STYLE="font: 10pt Times New Roman, Times, Serif; margin: 0pt 0; text-align: justify">Telephone: +1 212.915.0685 direct</P>



<P STYLE="margin: 0; text-align: right">&nbsp;</P>

<P STYLE="margin: 0; text-align: right"></P>

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