NEW HAVEN, Conn., May 25, 2016 (GLOBE NEWSWIRE) -- Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan
disease-focused biotechnology company, today announced financial results for the first quarter ended March 31, 2016.
“During the first quarter, we announced positive final results of our HOPEMD open label Phase 2a study of
trehalose 90mg/mL IV solution in patients with oculopharyngeal muscular dystrophy (OPMD),” said Colin Foster, Bioblast’s President
and CEO. “Trehalose 90mg/mL IV solution was observed to be safe and well tolerated, with no drug-related serious adverse
events. Additionally, improvements versus baseline were observed in multiple efficacy endpoints related to dysphagia and
muscle strength and function.”
“We ended the quarter with $21.6 million in cash and equivalents,” Mr. Foster, continued, “and are working on
plans to reduce expenses going forward, such that we will be able to meet our operating objectives.”
First Quarter 2016 and Recent Corporate Highlights:
- The Company announced positive final results from the HOPEMD open label Phase 2a study of trehalose 90mg/mL IV solution in
OPMD.
- The Company presented the HOPEMD open label Phase 2a final study results at Myology 2016 and the American Academy of
Neurology (AAN) 2016 Annual Meeting.
- The Company raised approximately $6.7 million in gross proceeds in a public offering from two health-care focused
institutional investors.
First Quarter 2016 Financial Results:
- Cash and cash equivalents (including short-term bank deposits) totaled $21.6 million at March 31, 2016, compared with $19.3
million at December 31, 2015.
- Research and development (R&D) expenses for the first quarter of 2016 amounted to $1.9 million, compared with $2.0
million in the first quarter of 2015. The decrease is primarily attributed to lower regulatory consulting expenses and to
reduced activity with respect to the Company’s read-through (BBrm02) program, partially offset by higher staffing expenses and
higher expenses incurred related to the trehalose IV solution open label Phase 2a clinical study in OPMD.
- General and administrative (G&A) expenses for the first quarter of 2016 were $1.9 million, compared with $1.4 million in
the first quarter of 2015. The increase in G&A is primarily attributed to salary and travel expenses related to a
higher level of staffing in the U.S. and to higher legal and patent maintenance expenses, partially offset by a lower stock
compensation expense.
- Pre-commercialization expenses for the first quarter of 2016 were $0.8 million, compared with no pre-commercialization
expenses in the first quarter of 2015.
- Net loss for the first quarter of 2016 was $4.6 million, or $0.32 per share, compared with a net loss of $3.3 million, or
$0.23 per share, in the first quarter of 2015.
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 has a portfolio of product candidates with the potential to address unmet medical
needs for incurable diseases. The Bioblast product portfolio is based on a deep understanding of disease-causing biological
processes, and potentially offers solutions to 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.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, the Company is using forward looking statements when
it discusses plans to reduce expenses and meeting operating objectives, building a diverse portfolio of product candidates with the
potential to address unmet medical needs for incurable diseases, or that Bioblast’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 "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 Statements of Operations
(U.S. dollars in thousands, except per share amounts) |
| |
| |
Three Months Ended |
| |
March 31, |
| |
|
2016 |
|
|
|
2015 |
|
| |
(unaudited) |
|
(unaudited) |
| |
|
|
|
| Research and development |
$ |
1,858 |
|
|
$ |
1,952 |
|
| Pre-commercialization |
|
772 |
|
|
|
- |
|
| General and administrative |
|
1,862 |
|
|
|
1,366 |
|
| |
|
|
|
| Operating loss |
|
4,492 |
|
|
|
3,318 |
|
| |
|
|
|
| Financial expense, net |
|
(27 |
) |
|
|
(21 |
) |
| |
|
|
|
| Loss before taxes on income |
|
4,465 |
|
|
|
3,297 |
|
| |
|
|
|
| Taxes on income |
|
129 |
|
|
|
2 |
|
| |
|
|
|
| Net loss |
$ |
4,594 |
|
|
$ |
3,299 |
|
| Net basic and diluted loss per share |
$ |
0.32 |
|
|
$ |
0.23 |
|
| Weighted average number of Ordinary shares used in computing basic and diluted net
loss per share |
|
14,467,984 |
|
|
|
14,230,480 |
|
| |
|
|
|
Bioblast Pharma Ltd
Consolidated Balance Sheet Data
(U.S. dollars in thousands) |
| |
| ASSETS |
| |
| |
|
|
March 31, |
|
December 31, |
| |
|
|
2016 |
|
2015 |
| |
|
|
(unaudited) |
|
|
| Current assets: |
|
|
|
|
|
| Cash and cash equivalents |
|
|
$ |
9,526 |
|
|
$ |
7,286 |
|
| Short term bank deposits |
|
|
|
12,080 |
|
|
|
12,046 |
|
| Receivables and prepaid expenses |
|
|
|
722 |
|
|
|
1,060 |
|
| |
| Total current assets |
|
|
|
22,328 |
|
|
|
20,392 |
|
| |
|
|
|
|
|
| Long-term assets: |
|
|
|
|
|
| Long-term deposit |
|
|
|
58 |
|
|
|
33 |
|
| Property and equipment, net |
|
|
|
93 |
|
|
|
91 |
|
| Total long-term assets |
|
|
|
151 |
|
|
|
124 |
|
| |
|
|
|
|
|
| Total assets |
|
|
$ |
22,479 |
|
|
$ |
20,516 |
|
| |
LIABILITIES AND
SHAREHOLDERS’ EQUITY |
| |
|
|
|
|
|
| Current liabilities: |
|
|
|
|
|
| Trade payables |
|
|
$ |
983 |
|
|
$ |
1,412 |
|
| Other accounts payable |
|
|
|
1,192 |
|
|
|
1,102 |
|
| |
|
|
|
|
|
|
|
|
|
| Total current liabilities |
|
|
|
2,175 |
|
|
|
2,514 |
|
| |
|
|
|
|
|
| Long term liabilities: |
|
|
|
|
|
| Accrued severance pay |
|
|
|
125 |
|
|
|
70 |
|
| |
|
|
|
|
|
|
|
|
|
| Total long term liabilities |
|
|
|
125 |
|
|
|
70 |
|
| |
|
|
|
|
|
| Shareholders’ equity: |
|
|
|
|
|
| Share capital |
|
|
|
45 |
|
|
|
39 |
|
| Additional paid-in capital |
|
|
|
48,515 |
|
|
|
41,680 |
|
| Accumulated deficit |
|
|
|
(28,381 |
) |
|
|
(23,787 |
) |
| |
|
|
|
|
|
| Total shareholders’ equity |
|
|
|
20,179 |
|
|
|
17,932 |
|
| |
|
|
|
|
|
| Total liabilities and shareholders’ equity |
|
|
$ |
22,479 |
|
|
$ |
20,516 |
|
| |
|
|
|
|
|
|
|
|
|
Bioblast Pharma Ltd
Consolidated Statements of Cash Flow Data
(U.S. dollars in thousands) |
| |
| |
Three Months Ended |
| |
March 31, |
| |
|
2016 |
|
|
|
2015 |
|
| |
(unaudited) |
|
(unaudited) |
| Cash flow from operating activities: |
|
|
|
| Net loss |
$ |
(4,594 |
) |
|
$ |
(3,299 |
) |
| Adjustments to reconcile net loss to net cash used in operating activities: |
|
|
|
| Depreciation |
|
6 |
|
|
|
3 |
|
| Stock based compensation |
|
751 |
|
|
|
720 |
|
| Interest on short term deposit |
|
(34 |
) |
|
|
(47 |
) |
| Change in operating assets and liabilities: |
|
|
|
| Receivables and prepaid expenses |
|
338 |
|
|
|
(402 |
) |
| Long term deposit |
|
(25 |
) |
|
|
(2 |
) |
| Trade payables |
|
(506 |
) |
|
|
(478 |
) |
| Other accounts payable |
|
61 |
|
|
|
(63 |
) |
| Accrued severance pay |
|
55 |
|
|
|
13 |
|
| Net cash used in operating activities |
|
(3,948 |
) |
|
|
(3,555 |
) |
| |
|
|
|
| Cash flow from investing activities: |
|
|
|
| |
|
|
|
| Purchase of property and equipment |
|
(8 |
) |
|
|
(17 |
) |
| |
|
|
|
|
|
|
|
| Net cash used in investing activities |
|
(8 |
) |
|
|
(17 |
) |
| |
|
|
|
| Cash flow from financing activities: |
|
|
|
| |
|
|
|
| Issuance of shares, net |
|
6,196 |
|
|
|
- |
|
| |
|
|
|
|
|
|
|
| Net cash provided by financing activities |
|
6,196 |
|
|
|
- |
|
| |
|
|
|
| Increase (decrease) in cash and cash equivalents |
|
2,240 |
|
|
|
(3,572 |
) |
| |
|
|
|
| Cash and cash equivalents at the beginning of the period |
|
7,286 |
|
|
|
10,583 |
|
| |
|
|
|
| Cash and cash equivalents at the end of the period |
$ |
9,526 |
|
|
$ |
7,011 |
|
| |
|
|
|
| Non-cash
transaction: |
|
|
|
| Issuance expenses yet to be paid as of March 31, 2016 |
$ |
107 |
|
|
|
- |
|
| |
|
|
|
INVESTOR CONTACT: Matthew P. Duffy Managing Director LifeSci Advisors, LLC Telephone: 212-915-0685
