TEL AVIV, Israel, Aug. 25, 2016 (GLOBE NEWSWIRE) -- Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan
disease-focused biotechnology company, today announced financial results for the second quarter ended June 30, 2016.
The Company reported a net loss of $3.685 million for the period, $.22 per share, as compared to $3.686 million,
$.26 per share for the second quarter of 2015. The net loss for the first half of 2016 was $8.279 million, $.54 per share as
compared to $6.985 million, $.49 per share for the first half of 2015.
Cash and cash equivalents (including short-term bank deposits) totaled $15.7 million at June 30, 2016, compared
with $19.3 million at December 31, 2015.
Second Quarter 2016 and Recent Corporate Highlights:
- The Company announced its intention to initiate enrollment of more than 70 patients in a double-blind, placebo-controlled
Phase 2b clinical trial of trehalose 90mg/mL IV solution in patients with oculopharyngeal muscular dystrophy (OPMD) at up to 15
sites in the United States and Canada during the first quarter of 2017.
- The Company decided to concentrate internal development efforts exclusively on trehalose, and has therefore postponed work on
the mitochondrial replacement and read-through platforms.
- Fredric Price, Executive Chairman, assumed the role of Acting CEO, following the departure of the former CEO, Colin Foster,
who remains a member of the Board of Directors.
- On August 9, 2016, Ralf Rosskamp, MD, PhD, joined the Bioblast Board of Directors. Dr. Rosskamp is a pediatric
endocrinologist with over 15 years of experience in clinical medicine and almost 20 years of experience in the pharmaceutical
industry.
- Mr. Price and Warren Wasiewski, MD, the Company’s Chief Medical Officer, will be providing a business update at the Rodman &
Renshaw Annual Global Investment Conference on September 12, 2016 in New York City.
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 with a lead drug candidate, trehalose 90mg/mL solution, in Phase 2 development. Bioblast
was founded in 2012 and 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 it
discusses plans to conduct a Phase 2b clinical study, the design and timing of such study, the feasibility of additional programs
for trehalose IV solution, the timing of the payment of severance and related benefits in connection with the personnel adjustments
and expected savings. 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 "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 |
|
Six months ended |
| |
|
June 30, |
|
June 30, |
| |
|
|
2016 |
|
|
|
2015 |
|
|
|
2016 |
|
|
|
2015 |
|
| |
|
(unaudited) |
|
(unaudited) |
|
(unaudited) |
|
(unaudited) |
| |
|
|
|
|
|
|
|
|
| Research and development |
|
$ |
1,935 |
|
|
$ |
2,142 |
|
|
$ |
3,793 |
|
|
$ |
4,094 |
|
| Pre-commercialization |
|
|
64 |
|
|
|
- |
|
|
|
836 |
|
|
|
- |
|
| General and administrative |
|
|
1,723 |
|
|
|
1,596 |
|
|
|
3,585 |
|
|
|
2,962 |
|
| |
|
|
|
|
|
|
|
|
| Total operating expenses |
|
|
3,722 |
|
|
|
3,738 |
|
|
|
8,214 |
|
|
|
7,056 |
|
| |
|
|
|
|
|
|
|
|
| Financial income, net |
|
|
(14 |
) |
|
|
(55 |
) |
|
|
(41 |
) |
|
|
(76 |
) |
| |
|
|
|
|
|
|
|
|
| Loss before taxes on income |
|
|
3,708 |
|
|
|
3,683 |
|
|
|
8,173 |
|
|
|
6,980 |
|
| |
|
|
|
|
|
|
|
|
| Taxes on income (tax benefit) |
|
|
(23 |
) |
|
|
3 |
|
|
|
106 |
|
|
|
5 |
|
| |
|
|
|
|
|
|
|
|
| Net loss attributable to holders of Ordinary shares |
|
$ |
3,685 |
|
|
$ |
3,686 |
|
|
$ |
8,279 |
|
|
$ |
6,985 |
|
| Net basic and diluted loss per share |
|
$ |
0.22 |
|
|
$ |
0.26 |
|
|
$ |
0.54 |
|
|
$ |
0.49 |
|
| Weighted average number of Ordinary shares used in computing basic and diluted net
loss per share |
|
|
16,391,770 |
|
|
|
14,230,480 |
|
|
|
15,429,877 |
|
|
|
14,230,480 |
|
| |
|
|
|
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
| |
| Bioblast Pharma Ltd |
| Consolidated Balance Sheet Data |
| (U.S. dollars in thousands, except share and per share
amounts) |
| |
| |
|
June 30, |
|
December 31, |
| |
|
|
2016 |
|
|
|
2015 |
|
| |
|
(unaudited) |
|
|
| ASSETS |
|
|
|
|
| |
|
|
|
|
| CURRENT ASSETS: |
|
|
|
|
| Cash and cash equivalents |
|
$ |
8,714 |
|
|
$ |
7,286 |
|
| Short-term bank deposits |
|
|
7,004 |
|
|
|
12,046 |
|
| Receivables and prepaid expenses |
|
|
1,093 |
|
|
|
1,060 |
|
| |
|
|
|
|
| Total current
assets |
|
|
16,811 |
|
|
|
20,392 |
|
| |
|
|
|
|
| LONG-TERM ASSETS: |
|
|
|
|
| Long-term deposits |
|
|
56 |
|
|
|
33 |
|
| Property and equipment, net |
|
|
88 |
|
|
|
91 |
|
| |
|
|
|
|
| Total
long-term assets |
|
|
144 |
|
|
|
124 |
|
| |
|
|
|
|
| TOTAL ASSETS |
|
$ |
16,955 |
|
|
$ |
20,516 |
|
| |
|
|
|
|
| LIABILITIES AND SHAREHOLDERS' EQUITY |
|
|
|
|
| |
|
|
|
|
| CURRENT LIABILITIES: |
|
|
|
|
| Trade payables |
|
$ |
619 |
|
|
$ |
1,412 |
|
| Other accounts payable |
|
|
849 |
|
|
|
1,102 |
|
| |
|
|
|
|
| Total current
liabilities |
|
|
1,468 |
|
|
|
2,514 |
|
| |
|
|
|
|
| |
|
|
|
|
| LONG TERM LIABILITIES |
|
|
- |
|
|
|
70 |
|
| |
|
|
|
|
| SHAREHOLDERS' EQUITY: |
|
|
|
|
| Ordinary shares of NIS 0.01 par value - |
|
|
|
|
|
|
|
|
| 50,000,000 shares authorized at June 30, 2016 and December 31,
2015; |
|
|
|
|
|
|
|
|
| 16,391,770 issued and outstanding shares at June 30, 2016 and
14,230,480 issued and outstanding shares at December 31, 2015 |
|
|
45 |
|
|
|
39 |
|
| Additional paid-in capital |
|
|
47,508 |
|
|
|
41,680 |
|
| Accumulated deficit |
|
|
(32,066 |
) |
|
|
(23,787 |
) |
| |
|
|
|
|
| Total shareholders' equity |
|
|
15,487 |
|
|
|
17,932 |
|
| |
|
|
|
|
| TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY |
|
$ |
16,955 |
|
|
$ |
20,516 |
|
| |
|
|
|
|
|
|
|
|
| |
| Bioblast Pharma Ltd |
| Consolidated Cash Flow Data |
| (U.S. dollars in thousands) |
| |
| |
Three months
ended |
|
Six months
ended |
| |
June 30, |
|
June 30, |
| |
|
2016 |
|
|
|
2015 |
|
|
|
2016 |
|
|
|
2015 |
|
| |
(unaudited) |
|
(unaudited) |
|
(unaudited) |
|
(unaudited) |
| Cash flow from operating activities: |
|
|
|
|
|
|
|
| Net loss |
$ |
(3,685 |
) |
|
$ |
(3,686 |
) |
|
$ |
(8,279 |
) |
|
$ |
(6,985 |
) |
| Adjustments to reconcile net income to net cash used in operating activities: |
|
|
|
|
|
|
|
| Depreciation |
|
7 |
|
|
|
3 |
|
|
|
13 |
|
|
|
6 |
|
| Stock based compensation, net |
|
(1,007 |
) |
|
|
722 |
|
|
|
(256 |
) |
|
|
1,442 |
|
| Decrease (increase) in receivables and prepaid expenses |
|
(371 |
) |
|
|
91 |
|
|
|
(33 |
) |
|
|
(311 |
) |
| Decrease (increase) in interest on short term deposit |
|
76 |
|
|
|
(31 |
) |
|
|
42 |
|
|
|
(78 |
) |
| Decrease (increase) in long-term deposit |
|
2 |
|
|
|
- |
|
|
|
(23 |
) |
|
|
(2 |
) |
| Decrease in trade payables |
|
(287 |
) |
|
|
(46 |
) |
|
|
(793 |
) |
|
|
(524 |
) |
| Increase (decrease) in other accounts payable |
|
(314 |
) |
|
|
43 |
|
|
|
(253 |
) |
|
|
(20 |
) |
| Increase (decrease) in accrued severance pay |
|
(125 |
) |
|
|
19 |
|
|
|
(70 |
) |
|
|
32 |
|
| Net cash used in operating activities |
|
(5,704 |
) |
|
|
(2,885 |
) |
|
|
(9,652 |
) |
|
|
(6,440 |
) |
| |
|
|
|
|
|
|
|
| Cash flow from investing activities: |
|
|
|
|
|
|
|
| |
|
|
|
|
|
|
|
| Withdrawal of short-term bank deposits |
|
5,000 |
|
|
|
5,000 |
|
|
|
5,000 |
|
|
|
5,000 |
|
| Purchase of property and equipment |
|
(2 |
) |
|
|
(4 |
) |
|
|
(10 |
) |
|
|
(21 |
) |
| Net cash provided by investing activities |
|
4,998 |
|
|
|
4,996 |
|
|
|
4,990 |
|
|
|
4,979 |
|
| |
|
|
|
|
|
|
|
| Cash flow from financing activities: |
|
|
|
|
|
|
|
| |
|
|
|
|
|
|
|
| Issuance of Ordinary shares and warrants, net |
|
(106 |
) |
|
|
- |
|
|
|
6,090 |
|
|
|
- |
|
| Net cash provided by (used in) financing activities |
|
(106 |
) |
|
|
- |
|
|
|
6,090 |
|
|
|
- |
|
| |
|
|
|
|
|
|
|
| Increase (decrease) in cash and cash equivalents |
|
(812 |
) |
|
|
2,111 |
|
|
|
1,428 |
|
|
|
(1,461 |
) |
| |
|
|
|
|
|
|
|
| Cash and cash equivalents at the beginning of the period |
|
9,526 |
|
|
|
7,011 |
|
|
|
7,286 |
|
|
|
10,583 |
|
| |
|
|
|
|
|
|
|
| Cash and cash equivalents at the end of the period |
$ |
8,714 |
|
|
$ |
9,122 |
|
|
$ |
8,714 |
|
|
$ |
9,122 |
|
| |
|
|
|
|
|
|
|
INVESTOR CONTACT: Matthew P. Duffy Managing Director LifeSci Advisors, LLC Telephone: 212-915-0685
