Bioblast Pharma

Hope should not be rare

Bioblast Pharma Reports First Quarter 2016 Financial Results

Wed, 2016-05-25 00:00 -- Bioblast Pharma

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,, 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)
      March 31,   December 31,
      2016   2015
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  
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       -  


Matthew P. Duffy
Managing Director
LifeSci Advisors, LLC
Telephone:  212-915-0685