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BioBlast Pharma Reports Fourth Quarter Financial Results Tel Aviv, Israel - February 17, 201 5 - BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage biotechnology company committed to developing clinically meaningful

Key Takeaway: BioBlast Pharma Reports Fourth Quarter Tel Aviv, Israel - February 17, 2015 - 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, today ann

Full Press Release Details

BioBlast Pharma Reports Fourth Quarter
Tel Aviv, Israel - February 17, 2015 - 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, today announced financial results for the fourth
quarter ended December 31, 2014.
Fourth Quarter 2014 Financial Results:
Balance Sheet Highlights
Fourth Quarter and Recent Corporate Highlights:
Management Commentary
"Year-to-date we have made significant progress in executing
our business strategy and advancing our clinical activities across our pipeline of drug candidates," said Colin Foster, Chief
Executive Officer of BioBlast. "Our successful IPO has strengthened our balance sheet, giving us the resources to advance
our clinical programs and taken the company to the next level. Of note, we continue to see high patient recruitment rates in our
phase 2/3 clinical trial for our lead clinical program for OPMD and remain on track to meet our goal of completing patient enrollment
in the next several months."
About BioBlast Pharma Ltd.
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 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. BioBlast was founded in 2012 and is traded on the NASDAQ under the symbol "ORPN".
For more information please visit the Company's website, www.bioblast-pharma.com, the content of which is not incorporated herein
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 advancement and timing of our clinical programs,
building a diverse portfolio of products candidates with the potential to address unmet medical needs for incurable diseases, or
that our 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 that historic results referred to in this press release would not be interpreted
differently in light of additional research and clinical and preclinical trials 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 "Risk Factors" in BioBlast Pharma's final IPO prospectus
filed with the Securities and Exchange Commission ("SEC") 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.
U.S. Investor Contact:
LifeSci Advisors, LLC
BioBlast Pharma Ltd.
Statement of Operation
(U.S. dollars in thousands,
except per share amounts)
Three Months Ended Year Ended
December 31, December 31,
2014 2013 2014 2013
(unaudited) (unaudited) (unaudited) (unaudited)
Research and development $ 2,047 $ 529 $ 4,441 $ 732
General and administrative 1,150 255 2,639 416
Total operating expenses
3,197 784 7,080 1,148
Financial Income, net (45 ) (7 ) (58 ) (3 )
Net loss attributable to holders of Ordinary shares $ 3,152 $ 777 $ 7,022 $ 1,145
Net basic and diluted loss per share $ 0.22 $ 0.08 $ 0.57 $ 0.14
Weighted average number of Ordinary shares used in computing basic and diluted net loss per share 14,230,480 9,182,867 12,259,600 8,423,018
BioBlast Pharma Ltd.
December 31, December 31,
2014 2013
(unaudited)
Current assets:
Cash and cash equivalents $ 10,583 $ 270
Short term bank deposits 22,028 -
Receivables and prepaid expenses 274 29
Total current assets 32,885 299
Long-term assets:
Long-term deposit 9 5
Property and equipment, net 60 2
Total long-term assets 69 7
Total assets $ 32,954 $ 306
SHAREHOLDERS' EQUITY
Current liabilities:
Trade payables $ 1,285 $ 46
Other accounts payable 995 85
Total current liabilities 2,280 131
Shareholders' equity:
Share capital 39 24
Additional paid-in capital 39,057 1,551
Accumulated deficit (8,422 ) (1,400 )
Total shareholders' equity 30,674 175
Total liabilities and shareholders' equity $ 32,954 $ 306
BioBlast Pharma Ltd.
Three Months Ended Year Ended
December 31, December 31,
2014 2013 2014 2013
(unaudited) (unaudited) (unaudited) (unaudited)
Cash flow from operating activities:
Net loss $ (3,152 ) $ (777 ) $ (7,022 ) $ (1,145 )
Adjustments to reconcile net income to net cash used in operating activities:
Depreciation 2 -*) 5 -*)
Stock based compensation 431 185 736 254
Interest on short term deposit (28 ) - (28 ) -
Change in operating assets and liabilities:
Receivables and prepaid expenses 73 2 (245 ) (19 )
Long term deposit 1 (1 ) (4 ) (5 )
Trade payables 643 (11 ) 1,239 (28 )
Other accounts payable 562 37 910 78
Net cash used in operating activities (1,468 ) (565 ) (4,409 ) (865 )
Cash flow from investing activities:
Short term bank deposits (22,000 ) - (22,000 ) -
Purchase of property and equipment (7 ) - (63 ) (2 )
Net cash used in investing activities (22,007 ) - (22,063 ) (2 )
Cash flow from financing activities:
Issuance of shares, net (70 ) - 36,785 991
Net cash provided by financing activities (70 ) - 36,785 991
Increase (decrease) in cash and cash equivalents (23,545 ) (565 ) 10,313 124
Cash and cash equivalents at the beginning of the period 34,128 835 270 146
Cash and cash equivalents at the end of the period $ 10,583 $ 270 $ 10,583 $ 270
*) Represents an amount lower than $1.
Last updated: Feb 17, 2015