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INVESTOR CONTACT Matthew P. Duffy Managing Director LifeSci Advisors LLC Matthew@lifesciadvisors.com Phone: 212-915-0685 Bioblast Pharma Reports Fourth Quarter and Year End 2016 Financial Results Tel Aviv, Israel

Key Takeaway: LifeSci Advisors LLC Matthew@lifesciadvisors.com Bioblast Pharma Reports Fourth Quarter and Year End 2016 Financial Results Tel Aviv, Israel, - February 24, 2017 - Bioblast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company, today an

Full Press Release Details

LifeSci Advisors LLC
Bioblast Pharma Reports Fourth Quarter
and Year End 2016 Financial Results
Tel Aviv, Israel, - February 24, 2017
- Bioblast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company, today announced
financial results for the fourth quarter ended December 31, 2016 and other recent developments.
Key Corporate Developments:
Fourth Quarter and Year End
2016 Financial results and Cash Guidance
For the year ended December
31, 2016, research and development expenses totaled $8.9 million compared to $7.7 million in the prior year. The increase was primarily
due to increased salaries and related expenses including share-based compensation expenses, as well as initiation activities with
respect to the Phase 2b Trial.
For the year ended December
31, 2016, pre-commercialization expenses totaled $1.1 million, compared to $0.8 million in the prior year. The increase was primarily
due to increased market research activities in the first half of the year directed at assessing the commercial opportunity presented
by our Trehalose IV solution for the treatment of OPMD and SCA3 patients, which was offset by the reversal of previously recognized
share-based compensation due to the forfeiture of options previously granted to departing employees.
For the year ended December
31, 2016, general and administrative expenses totaled $5.9 million, compared to $7.0 million in the prior year. The general and
administrative costs during 2016 included termination related payments to departing employees. Such termination related payments
were partially offset by a reversal of previously recognized share-based compensation due to the forfeiture of options previously
granted to departing employees.
Bioblast Pharma is a clinical-stage
biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases.
Bioblast is traded on the NASDAQ under the symbol "ORPN". For more information, please visit our website: www.BioblastPharma.com,
the content of which is not incorporated herein by reference.
Forward Looking Statements
This press release contains
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 future
clinical studies, the design and timing of such studies, and the Company's expectation for its cash, cash equivalents and
short-term bank deposits need to raise additional funds and sufficiency of financial resources. In addition, historic results
of scientific research and clinical and preclinical studies do not guarantee that the conclusions of future research or studies
will suggest identical or similar conclusions or that historic results referred to in this press release be interpreted differently
in light of additional research and clinical and preclinical study results. Any forward-looking statements in this press release
are based on management's current expectations of future events and are subject to a number of risks and uncertainties that
could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements.
Because such statements deal with future events and are based on Bioblast Pharma'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 Annual Report on Form 20-F filed with the Securities and Exchange Commission ("SEC") on March 29, 2016,
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.
Bioblast Pharma Ltd.
Consolidated Statement of Operations
(U.S. dollars in thousands, except share
and per share amounts)
Three months ended December 31, Year ended December 31,
2016 2015 2016 2015
U.S. dollars in thousands, except share and per share data
Research and development $ 1,791 $ 1,887 $ 8,881 $ 7,694
Pre-commercialization 210 563 1,085 829
General and administrative 1,073 2,383 5,900 6,953
Total operating expenses 3,074 4,833 15,866 15,476
Loss from operations (3,074 ) (4,833 ) (15,866 ) (15,476 )
Financial income, net 11 20 60 135
Loss before taxes on income (3,063 ) (4,813 ) (15,806 ) (15,341 )
Taxes on income (10 ) (16 ) (216 ) (24 )
Net loss $ (3,073 ) $ (4,829 ) $ (16,022 ) $ (15,365 )
Net loss attributable to Ordinary shareholders $ (3,073 ) $ (4,829 ) $ (16,022 ) $ (15,365 )
Net loss per share attributable to Ordinary shareholders - basic and diluted $ (0.19 ) $ (0.34 ) $ (1.01 ) $ (1.08 )
Weighted average number of Ordinary shares outstanding - basic and diluted 16,391,770 14,230,480 15,906,220 14,230,480
Bioblast Pharma Ltd.
Consolidated Balance Sheet Data
December 31,
2016 2015
U.S. dollars in thousands, except share data
ASSETS
CURRENT ASSETS:
Cash and cash equivalents $ 6,871 $ 7,286
Short-term bank deposits 3,007 12,046
Receivables and prepaid expenses 663 1,060
Total current assets 10,541 20,392
LONG-TERM ASSETS:
Long-term assets 18 33
Property and equipment, net 71 91
Total long-term assets 89 124
TOTAL ASSETS $ 10,630 $ 20,516
LIABILITIES AND SHAREHOLDERS' EQUITY
CURRENT LIABILITIES:
Trade payables $ 700 $ 1,412
Other accounts payable 1,231 1,102
Total current liabilities 1,931 2,514
LONG-TERM LIABILITIES - 70
SHAREHOLDERS' EQUITY:
Ordinary shares of NIS 0.01 par value - 50,000,000 shares authorized at December 31, 2016 and 2015; 16,391,770 and 14,230,480 issued and outstanding shares at December 31, 2016 and 2015, respectively 45 39
Additional paid-in capital 48,463 41,680
Accumulated deficit (39,809 ) (23,787 )
Total stockholders' equity 8,699 17,932
TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY $ 10,630 $ 20,516
Bioblast Pharma Ltd.
Consolidated Cash Flow Data
Year ended December 31,
2016 2015
U.S. dollars in thousands
Cash flows from operating activities
Net loss $ (16,022 ) $ (15,365 )
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and disposal of property and equipment 38 17
Share based compensation 700 2,623
Interest on short-term deposit 39 (18 )
Changes in operating assets and liabilities:
Decrease (increase) in receivables and prepaid expenses 399 (786 )
Decrease (increase) in long-term assets 13 (24 )
Increase (decrease) in trade payables (712 ) 127
Increase in other accounts payable 129 107
Increase (decrease) in long-term liabilities (70 ) 70
Net cash used in operating activities (15,486 ) (13,249 )
Cash flow from investing activities
Withdrawal of (investment in) short-term bank deposits 9,000 10,000
Purchase of property and equipment (18 ) (48 )
Net cash provided by (used in) investing activities 8,982 9,952
Cash flow from financing activities
Issuance of shares and warrants, net 6,089 -
Net cash provided by financing activities 6,089 -
Increase (decrease) in cash and cash equivalents (415 ) (3,297 )
Cash and cash equivalents, beginning of the year 7,286 10,583
Cash and cash equivalents, end of the year $ 6,871 $ 7,286
Supplemental disclosures of cash flow information:
Cash paid for taxes $ 210 $ 4
Cash received for interest $ 120 $ 162
Last updated: Feb 24, 2017