Full Press Release Details
BioBlast Pharma Reports Second Quarter
Tel Aviv, Israel August 21, 2015
- BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company, today announced
financial results for the second quarter ended June 30, 2015.
BioBlast ended the second quarter of
2015 with $26.2 million in cash and short-term bank deposits, having spent $3.7 million in total expenses during the quarter.
Colin Foster, President & CEO, said
"During the second quarter we made significant progress in advancing our clinical and regulatory activities in the USA,
Canada, and Israel across our pipeline of drug candidates. Of note, recently the U.S. Food and Drug Administration (FDA) granted
Fast Track Designation and clearance to proceed with an Investigational New Drug Application (IND) for the development of Cabaletta
to treat patients with oculopharyngeal muscular dystrophy (OPMD). Additionally, we have been granted an orphan drug designation
from the European Commission for Cabaletta for the treatment of OPMD and Spinocerebellar Ataxia (SCA). Both of these events will
help to expedite the continued development of our lead drug candidate in the United States and Europe. Moreover, we have announced
positive preclinical in-vitro and in-vivo proof-of-concept study results of our novel read-through drug candidate
(BBrm02) for Spinal Muscular Atrophy (SMA). This recent development will accelerate the BBrm2 platform into the clinical stage."
Second Quarter 2015 Financial Results:
Balance Sheet Highlights:
Second Quarter and Recent Corporate
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 a 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 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 the timing and
advancement of its product candidates, including the clinical trial and approval processes, 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 those 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 Annual Report on Form 20-F filed with the Securities and Exchange
Commission ("SEC") on March 31, 2015, 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
Bio Blast Pharma Ltd
Consolidated Statement of Operation
(U.S. dollars in thousands,
except per share amounts)
| Three Months Ended | Six Months Ended | |||||||||||||||
| June 30, | June 30, | |||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| (unaudited) | (unaudited) | (unaudited) | (unaudited) | |||||||||||||
| Research and development | $ | 2,142 | $ | 721 | $ | 4,094 | $ | 1,163 | ||||||||
| General and administrative | 1,596 | 391 | 2,962 | 590 | ||||||||||||
| Total operating expenses | ||||||||||||||||
| 3,738 | 1,112 | 7,056 | 1,753 | |||||||||||||
| Financial expenses (income), net | (55 | ) | 2 | (76 | ) | 6 | ||||||||||
| Net comprehensive loss | 3,683 | 1,114 | 6,980 | 1,759 | ||||||||||||
| Taxes on income | 3 | - | 5 | - | ||||||||||||
| Net loss attributable to holders of Ordinary shares | $ | 3,686 | $ | 1,114 | $ | 6,985 | $ | 1,759 | ||||||||
| Net basic and diluted loss per share | $ | 0.26 | $ | 0.10 | $ | 0.49 | $ | 0.16 | ||||||||
| Weighted average number of Ordinary shares used in computing basic and diluted net loss per share | 14,230,480 | 11,030,480 | 14,230,480 | 10,873,973 |
Bio Blast Pharma Ltd
Consolidated Balance Sheet Data
| June 30, | December 31, | |||||||
| 2015 | 2014 | |||||||
| (unaudited) | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 9,122 | $ | 10,583 | ||||
| Short term bank deposits | 17,106 | 22,028 | ||||||
| Receivables and prepaid expenses | 585 | 274 | ||||||
| Total current assets | 26,813 | 32,885 | ||||||
| Long-term assets: | ||||||||
| Long-term deposit | 11 | 9 | ||||||
| Property and equipment, net | 75 | 60 | ||||||
| Total long-term assets | 86 | 69 | ||||||
| Total assets | $ | 26,899 | $ | 32,954 |
SHAREHOLDERS' EQUITY
| Current liabilities: | ||||||||
| Trade payables | $ | 761 | $ | 1,285 | ||||
| Other accounts payable | 975 | 995 | ||||||
| Total current liabilities | 1,736 | 2,280 | ||||||
| Long term liabilities: | ||||||||
| Accrued severance pay | 32 | - | ||||||
| Total long term liabilities | 32 | - | ||||||
| Shareholders' equity: | ||||||||
| Share capital | 39 | 39 | ||||||
| Additional paid-in capital | 40,499 | 39,057 | ||||||
| Accumulated deficit | (15,407 | ) | (8,422 | ) | ||||
| Total shareholders' equity | 25,131 | 30,674 | ||||||
| Total liabilities and shareholders' equity | $ | 26,899 | $ | 32,954 |
Consolidated Cash Flow Data
| Three Months Ended | Six Months Ended | |||||||||||||||
| June 30, | June 30, | |||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| (unaudited) | (unaudited) | (unaudited) | (unaudited) | |||||||||||||
| Cash flow from operating activities: | ||||||||||||||||
| Net loss | $ | (3,686 | ) | $ | (1,114 | ) | $ | (6,985 | ) | $ | (1,759 | ) | ||||
| Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||||||||||
| Depreciation | 3 | 1 | 6 | 1 | ||||||||||||
| Stock based compensation | 722 | 5 | 1,442 | 14 | ||||||||||||
| Interest on short term deposit | (31 | ) | - | (78 | ) | - | ||||||||||
| Changes in operating assets and liabilities: | ||||||||||||||||
| Receivables and prepaid expenses | 91 | (78 | ) | (311 | ) | (115 | ) | |||||||||
| Long-term deposit | - | - | (2 | ) | 3 | |||||||||||
| Trade payables | (46 | ) | 270 | (524 | ) | 231 | ||||||||||
| Other accounts payable | 43 | 252 | (20 | ) | 353 | |||||||||||
| Accrued severance pay | 19 | - | 32 | - | ||||||||||||
| Net cash used in operating activities | (2,885 | ) | (664 | ) | (6,440 | ) | (1,272 | ) | ||||||||
| Cash flow from investing activities: | ||||||||||||||||
| Investment in short term bank deposits | 5,000 | - | 5,000 | - | ||||||||||||
| Purchase of property and equipment | (4 | ) | (28 | ) | (21 | ) | (29 | ) | ||||||||
| Net cash provided by (used in) investing activities | 4,996 | (28 | ) | 4,979 | (29 | ) | ||||||||||
| Cash flow from financing activities: | ||||||||||||||||
| Issuance of shares, net | - | - | - | 5,371 | ||||||||||||
| Deferred issuance expenses | - | (20 | ) | - | (97 | ) | ||||||||||
| Net cash provided by (used in) financing activities | - | (20 | ) | - | 5,274 | |||||||||||
| Increase (decrease) in cash and cash equivalents | 2,111 | (712 | ) | (1,461 | ) | 3,973 | ||||||||||
| Cash and cash equivalents at the beginning of the period | 7,011 | 4,955 | 10,583 | 270 | ||||||||||||
| Cash and cash equivalents at the end of the period | $ | 9,122 | $ | 4,243 | $ | 9,122 | $ | 4,243 |