Full Press Release Details
Bio Blast Pharma Reports Third Quarter
Tel Aviv, Israel - November 21, 2014 - Bio
Blast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage biotechnology company committed to developing clinically meaningful therapies
for patients with rare and ultra-rare genetic diseases, today announced financial results for the third quarter ended September
Third Quarter 2014 Financial Results:
Balance Sheet Highlights
Third Quarter and Recent Corporate Highlights:
37 Menachem Begin Rd, Rubinshtein Tower,
15th Floor, Tel Aviv 6522042
Tel: +972 2409060 Fax: +972 3 7313340
Management Commentary
"Year-to-date we have made significant progress in executing
our business strategy and advancing our clinical activities across our pipeline of drug candidates," said Dr. Dalia Megiddo,
Chief Executive Officer of Bio Blast. "Our successful IPO has strengthened our balance sheet, giving us the resources to
advance our clinical programs and take the company to the next level. Of note, our phase 2/3 clinical trial for our lead clinical
program for OPMD is making good progress as we continued to see high patient recruitment rates and remain on track to meet our
goal of completing patient enrollment in the next several months."
About Bio Blast Pharma Ltd.
Bio Blast 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 Bio Blast
platforms are based on deep understanding of the disease-causing biological processes, and potentially offer solutions for several
diseases that share the same biological pathology. Bio Blast 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
Forward Looking Statements
This press release may contain 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 advancement of our clinical programs, 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 that 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 Bio Blast Pharma Ltd.'s current expectations, they are subject to various risks and uncertainties and actual results,
performance or achievements of Bio Blast 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 Bio Blast Pharma's final IPO prospectus
filed with the Securities and Exchange Commission ("SEC") and in any subsequent filings with the SEC. Except as otherwise
required by law, Bio Blast 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
37 Menachem Begin Rd, Rubinshtein Tower, 15th Floor,
Tel: +972 2409060 Fax: +972 3 7313340
Bio Blast Pharma Ltd.
Statement of Operation
(U.S. dollars in thousands,
except per share amounts)
| Three Months Ended | Nine Months Ended | |||||||||||||||
| September 30, | September 30, | |||||||||||||||
| 2014 | 2013 | 2014 | 2013 | |||||||||||||
| (unaudited) | (unaudited) | (unaudited) | (unaudited) | |||||||||||||
| Research and development | $ | 1,231 | $ | 137 | $ | 2,394 | $ | 203 | ||||||||
| General and administrative | 899 | 55 | 1,489 | 161 | ||||||||||||
| Total operating expenses | 2,130 | 192 | 3,883 | 364 | ||||||||||||
| Financial expenses (Income), net | (19 | ) | 3 | (13 | ) | 4 | ||||||||||
| Net loss attributable to holders of Ordinary shares | $ | 2,111 | $ | 195 | $ | 3,870 | $ | 368 | ||||||||
| Net basic and diluted loss per share | $ | 0.16 | $ | 0.02 | $ | 0.33 | $ | 0.05 | ||||||||
| Weighted average number of Ordinary shares used in computing basic and diluted net loss per share | 12,999,711 | 8,534,581 | 11,585,732 | 8,094,816 |
37 Menachem Begin Rd, Rubinshtein Tower,
15th Floor, Tel Aviv 6522042
Tel: +972 2409060 Fax: +972 3 7313340
Bio Blast Pharma Ltd.
| September 30, | December 31, | |||||||
| 2014 | 2013 | |||||||
| (unaudited) | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 34,128 | $ | 270 | ||||
| Receivables and prepaid expenses | 347 | 29 | ||||||
| Total current assets | 34,475 | 299 | ||||||
| Long-term assets: | ||||||||
| Long-term deposit | 10 | 5 | ||||||
| Property and equipment, net | 55 | 2 | ||||||
| Total long-term assets | 65 | 7 | ||||||
| Total assets | $ | 34,540 | $ | 306 |
SHAREHOLDERS' EQUITY
| Current liabilities: | ||||||||
| Trade payables | $ | 713 | $ | 46 | ||||
| Other accounts payable | 433 | 85 | ||||||
| Total current liabilities | 1,146 | 131 | ||||||
| Shareholders' equity: | ||||||||
| Share capital | 39 | 24 | ||||||
| Additional paid-in capital | 38,626 | 1,551 | ||||||
| Accumulated deficit | (5,271 | ) | (1,400 | ) | ||||
| Total shareholders' equity | 33,394 | 175 | ||||||
| Total liabilities and shareholders' equity | $ | 34,540 | $ | 306 |
37 Menachem Begin Rd, Rubinshtein Tower,
15th Floor, Tel Aviv 6522042
Tel: +972 2409060 Fax: +972 3 7313340
Bio Blast Pharma Ltd.
| Three Months Ended | Nine Months Ended | |||||||||||||||
| September 30, | September 30, | |||||||||||||||
| 2014 | 2013 | 2014 | 2013 | |||||||||||||
| (unaudited) | (unaudited) | (unaudited) | (unaudited) | |||||||||||||
| Cash flow from operating activities: | ||||||||||||||||
| Net loss | $ | (2,111 | ) | $ | (195 | ) | $ | (3,870 | ) | $ | (368 | ) | ||||
| Adjustments to reconcile net income to net cash used in operating activities: | ||||||||||||||||
| Depreciation | 2 | - | 3 | - | ||||||||||||
| Stock based compensation | 291 | 9 | 305 | 69 | ||||||||||||
| Change in operating assets and liabilities: | ||||||||||||||||
| Receivables and prepaid expenses | (203 | ) | (25 | ) | (318 | ) | (21 | ) | ||||||||
| Long term deposit | (8 | ) | (4 | ) | (5 | ) | (4 | ) | ||||||||
| Trade payables | 366 | 10 | 596 | (17 | ) | |||||||||||
| Other accounts payable | (6 | ) | 33 | 348 | 40 | |||||||||||
| Net cash used in operating activities | (1,669 | ) | (172 | ) | (2,941 | ) | (301 | ) | ||||||||
| Cash flow from investing activities: | ||||||||||||||||
| Purchase of property and equipment | (27 | ) | (2 | ) | (56 | ) | (2 | ) | ||||||||
| Net cash used in investing activities | (27 | ) | (2 | ) | (56 | ) | (2 | ) | ||||||||
| Cash flow from financing activities: | ||||||||||||||||
| Issuance of shares, net | 31,581 | 992 | 36,855 | 992 | ||||||||||||
| Net cash provided by financing activities | 31,581 | 992 | 36,855 | 992 | ||||||||||||
| Increase in cash and cash equivalents | 29,885 | 818 | 33,858 | 689 | ||||||||||||
| Cash and cash equivalents at the beginning of the period | 4,243 | 17 | 270 | 146 | ||||||||||||
| Cash and cash equivalents at the end of the period | $ | 34,128 | $ | 835 | $ | 34,128 | $ | 835 | ||||||||
| Supplemental disclosure of non-cash investing and financing activities: | ||||||||||||||||
| Issuance expenses yet to be paid as of September 30, 2014 | $ | 70 | $ | - | $ | 70 | $ | - | ||||||||
| Classification of Deferred issuance expenses to equity | $ | 97 | $ | - | $ | - | $ | - |
37 Menachem Begin Rd, Rubinshtein Tower,
15th Floor, Tel Aviv 6522042
Tel: +972 2409060 Fax: +972 3 7313340