TEL AVIV, Israel, Feb. 24, 2017 (GLOBE NEWSWIRE) -- Bioblast Pharma Ltd. (Nasdaq: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:
- Completed clinical trials: As previously announced on January 18, 2017, the Company reported the results of
a six-month open label Phase 2a clinical trial that also included an additional six-month follow-up period investigating
trehalose in patients with Spinocerebellar Ataxia Type 3 (SCA3).
- Departure of members of the Board of Directors: Messrs. Udi Gilboa and Gili Cohen resigned from our Board of
Directors effective January 2, 2017 and January 15, 2017, respectively, for personal reasons. Following the resignations, the
Board of Directors currently consists of nine members.
Fourth Quarter and Year End 2016 Financial results and Cash Guidance
- R&D Expenses: Research and development expenses were $1.8 million for the quarter ended December 31,
2016, compared to $1.9 million for the same period in 2015. The research and development expenses for the fourth quarter of 2016
included clinical development activities with respect to completed clinical studies as well as initiation activities with respect
to an upcoming Phase 2b double-blind placebo-controlled clinical trial of our trehalose 90mg/mL IV solution (“Trehalose IV”) in
oculopharyngeal muscular dystrophy (“OPMD”) patients (the “Phase 2b Trial”).
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.
- Pre-commercialization Expenses: Pre-commercialization expenses were $0.2 million for the quarter ended
December 31, 2016, compared to $0.6 million for the same period in 2015. The decrease was primarily related to the decision to
halt spending in this area until our Chief Commercial Officer, who joined in the middle of the quarter, had an opportunity to
assess activities in this area.
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.
- G&A Expenses: General and administrative expenses were $1.1 million for the quarter ended December 31,
2016, compared to $2.4 million for the same period in 2015. The decrease was primarily related to downsizing the management team,
which took effect primarily in the second quarter of 2016.
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.
- Net Loss: For the quarter ended December 31, 2016, net loss attributable to holders of ordinary shares was
($3.1) million, or ($0.19) per basic and diluted share, as compared to a net loss of ($4.8) million, or ($0.34) per share, for
the same period in 2015.
- Cash Position: Cash, cash equivalents and short-term bank deposits as of December 31, 2016 were $9.9
million, compared to $12.9 million as of September 30, 2016, primarily reflecting the Company’s fourth quarter operating
expenditures. Additional funding beyond our existing cash resources will be required to entirely cover the cost of the Phase 2b
Trial and the underlying expenses of our operations while the study is ongoing. Should we be unable to obtain the additional
funding required to complete our clinical activity, we may need to reduce our activities until we have sufficient resources.
About Bioblast
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 |
|
|
|
| |
|
|
|
|
|
|
INVESTOR CONTACT Matthew P. Duffy Managing Director LifeSci Advisors LLC Matthew@lifesciadvisors.com Phone: 212-915-0685
