Full Press Release Details
BioBlast Pharma Reports Third Quarter
Tel Aviv, Israel November 17, 2015
- BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company, today announced
financial results for the third quarter ended September 30, 2015.
BioBlast ended the third quarter of
2015 with $23.1 million in cash and short-term bank deposits, total quarterly expenses were $3.6 million whereas total quarterly
expenses excluding stock based compensation were $3.0 million.
Colin Foster, President & CEO, said
"During the third quarter and more recently, we made significant progress with the announcement of positive interim safety
and efficacy data in our HOPEMD Phase 2 open label clinical study evaluating Cabaletta in oculopharyngeal muscular dystrophy
(OPMD). These data support our plans to initiate a Phase 3 clinical study in OPMD in 2016 following discussions with regulatory
authorities. Additionally, we made significant progress on the personnel front with the announcement of new U.S.-based senior executives
joining the BioBlast executive team. Previously, we announced the establishment of a U.S. headquarters in New Haven, Connecticut."
Third Quarter 2015 Financial Results:
Balance Sheet Highlights
Third Quarter and Recent Corporate Highlights:
These interim results additionally
give insight into the potential of Cabaletta for use in other protein aggregation-related diseases, such as spinocerebellar ataxia
type 3 (SCA3 or Machado Joseph disease), another devastating hereditary disease in which the Company is planning a pivotal Phase
3 study in the United States and European Union.
Dr. Wasiewski and Ms. Cherry
will succeed Dr. Dalia Megiddo, a Company co-founder, who until now, has been Chief Development Officer and head of manufacturing,
as well as Prof. Zohar Argov, who has been the Company's Chief Medical Officer. Going forward, Dr. Megiddo is expected to
serve as an advisor to the Company and will continue to serve as a member of the Company's board of directors. Prof. Argov
will become Special Medical Advisor to the CEO and will be a member of the Company's Scientific Advisory Board. Udi Gilboa,
also a co-founder of the Company, who is currently the Chief Financial Officer, will transition to
a special advisor role once a successor has been appointed. He will continue as a member of the Company's board of directors.
BioBlast Pharma Ltd.
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.
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, the clinical trial approval process, including discussions
with regulatory authorities, initiating future clinical trials and whether such trials will be considered pivotal or not, potential
uses of its product candidates for various indications, 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 as well as the plans to transition responsibilities to new executives and transition
BioBlast's co-founders positions into advisors. 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 | Nine Months Ended | |||||||||||||||
| September 30, | September 30, | |||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| (unaudited) | (unaudited) | (unaudited) | (unaudited) | |||||||||||||
| Research and development | $ | 1,713 | $ | 1,231 | $ | 5,807 | $ | 2,394 | ||||||||
| Pre Commercialization | 266 | - | 266 | - | ||||||||||||
| General and administrative | 1,608 | 899 | 4,570 | 1,489 | ||||||||||||
| Total operating expenses | ||||||||||||||||
| 3,587 | 2,130 | 10,643 | 3,883 | |||||||||||||
| Financial income, net | (39 | ) | (19 | ) | (115 | ) | (13 | ) | ||||||||
| Net comprehensive loss | 3,548 | 2,111 | 10,528 | 3,870 | ||||||||||||
| Taxes on income | 3 | - | 8 | - | ||||||||||||
| Net loss attributable to holders of Ordinary shares | $ | 3,551 | $ | 2,111 | $ | 10,536 | $ | 3,870 | ||||||||
| Net basic and diluted loss per share | $ | 0.25 | $ | 0.16 | $ | 0.74 | $ | 0.33 | ||||||||
| Weighted average number of Ordinary shares used in computing basic and diluted net loss per share | 14,230,480 | 12,999,711 | 14,230,480 | 11,585,732 |
Bio Blast Pharma Ltd
Consolidated Balance Sheet Data
| September 30, | December 31, | |||||||
| 2015 | 2014 | |||||||
| (unaudited) | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 4,947 | $ | 10,583 | ||||
| Short term bank deposits | 18,126 | 22,028 | ||||||
| Receivables and prepaid expenses | 734 | 274 | ||||||
| Total current assets | 23,807 | 32,885 | ||||||
| Long-term assets: | ||||||||
| Long-term deposit | 33 | 9 | ||||||
| Property and equipment, net | 81 | 60 | ||||||
| Total long-term assets | 114 | 69 | ||||||
| Total assets | $ | 23,921 | $ | 32,954 |
SHAREHOLDERS' EQUITY
| Current liabilities: | ||||||||
| Trade payables | $ | 807 | $ | 1,285 | ||||
| Other accounts payable | 955 | 995 | ||||||
| Total current liabilities | 1,762 | 2,280 | ||||||
| Long term liabilities: | ||||||||
| Accrued severance pay | 51 | - | ||||||
| Total long term liabilities | 51 | - | ||||||
| Shareholders' equity: | ||||||||
| Share capital | 39 | 39 | ||||||
| Additional paid-in capital | 41,027 | 39,057 | ||||||
| Accumulated deficit | (18,958 | ) | (8,422 | ) | ||||
| Total shareholders' equity | 22,108 | 30,674 | ||||||
| Total liabilities and shareholders' equity | $ | 23,921 | $ | 32,954 |
Consolidated Cash Flow Data
| Three Months Ended | Nine Months Ended | |||||||||||||||
| September 30, | September 30, | |||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| (unaudited) | (unaudited) | (unaudited) | (unaudited) | |||||||||||||
| Cash flow from operating activities: | ||||||||||||||||
| Net loss | $ | (3,551 | ) | $ | (2,111 | ) | $ | (10,536 | ) | $ | (3,870 | ) | ||||
| Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||||||||||
| Depreciation | 4 | 2 | 10 | 3 | ||||||||||||
| Stock based compensation | 528 | 291 | 1,970 | 305 | ||||||||||||
| Interest on short term deposit | (20 | ) | - | (98 | ) | - | ||||||||||
| Changes in operating assets and liabilities: | ||||||||||||||||
| Receivables and prepaid expenses | (149 | ) | (203 | ) | (460 | ) | (318 | ) | ||||||||
| Long-term deposit | (22 | ) | (8 | ) | (24 | ) | (5 | ) | ||||||||
| Trade payables | 46 | 366 | (478 | ) | 596 | |||||||||||
| Other accounts payable | (20 | ) | (6 | ) | (40 | ) | 348 | |||||||||
| Accrued severance pay | 19 | - | 51 | - | ||||||||||||
| Net cash used in operating activities | (3,165 | ) | (1,669 | ) | (9,605 | ) | (2,941 | ) | ||||||||
| Cash flow from investing activities: | ||||||||||||||||
| Withdrawal of (investment in) short term bank deposits | (1,000 | ) | - | 4,000 | - | |||||||||||
| Purchase of property and equipment | (10 | ) | (27 | ) | (31 | ) | (56 | ) | ||||||||
| Net cash provided by (used in) investing activities | (1,010 | ) | (27 | ) | 3,969 | (56 | ) | |||||||||
| Cash flow from financing activities: | ||||||||||||||||
| Issuance of shares, net | - | 31,581 | - | 36,855 | ||||||||||||
| Net cash provided by financing activities | - | 31,581 | - | 36,855 | ||||||||||||
| Increase (decrease) in cash and cash equivalents | (4,175 | ) | 29,885 | (5,636 | ) | 33,858 | ||||||||||
| Cash and cash equivalents at the beginning of the period | 9,122 | 4,243 | 10,583 | 270 | ||||||||||||
| Cash and cash equivalents at the end of the period | $ | 4,947 | $ | 34,128 | $ | 4,947 | $ | 34,128 | ||||||||
| 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 | $ | - | $ | - |