Full Press Release Details
Bioblast Pharma Reports First Quarter
2016 Financial Results
New Haven, Connecticut - May 25,
2016 - Bioblast Pharma Ltd. (NasdaqGM: 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
First Quarter 2016 Financial Results:
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
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.
LifeSci Advisors, LLC
Telephone: 212-915-0685
Consolidated Statements of Operations
(U.S. dollars in thousands, except per
| 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 |
Consolidated Balance Sheet Data
dollars in thousands)
| 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 |
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 |
Consolidated Statements of Cash Flow
| 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 | - |