Company Update (NASDAQ:ORPN): Bio Blast Pharma Ltd Reports Second Quarter Financial Results


Bio Blast Pharma Ltd (NASDAQ: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-vivoproof-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:

  • Research and development (R&D) expenses for the second quarter of 2015 were $2.14 million, compared to $1.95 millionin the first quarter of 2015 and $0.72 million in the second quarter of 2014. The increase compared to the first quarter of 2015 was primarily due to a $0.37 million increase in clinical study and manufacturing expenses related to the Cabaletta platform, as well as an increase of $0.08 million in payroll expenses, offset by a $0.33 million decrease in pre-clinical study expenses related to SMA.
  • General and administrative (G&A) expenses for the second quarter of 2015 were $1.6 million, compared to $1.37 million in the first quarter of 2015 and $0.39 million in the second quarter of 2014. The increase compared to the first quarter was primarily due to $0.16 million of professional services expenses related to our U.S. operation and a $0.02 million increase in insurance and other office expenses.
  • Net loss for the second quarter of 2015 was $3.69 million, or $0.26 per share, compared to $3.3 million, or $0.23 per share, in the first quarter of 2015, and $1.11 million, or $0.10 per share, in the second quarter of 2014.

Balance Sheet Highlights:

  • Cash and cash equivalents (including short-term bank deposits) totaled $26.2 million as of June 30, 2015, compared to$32.6 million on December 31, 2014, and $4.24 million on June 30, 2014. The decrease compared to December 31, 2014, reflects the cash used in operations during the period.
  • Shareholders’ equity totaled $25.1 million as of June 30, 2015, compared to $30.7 million on December 31, 2014, and $3.8 million on June 30, 2014.

Second Quarter and Recent Corporate Highlights:

  • On April 9, 2015, the FDA granted Fast Track Designation to Cabaletta for the treatment of patients with OPMD.
  • On June 15, 2015, the Company announced that it was moving forward with a double blind placebo controlled Phase 3 pivotal study in the United States and Canada for Cabaletta in OPMD. The new pivotal study reflects positive signals reported from the current HOPEMD study in which recruitment is terminated at 25 patients who will continue to be followed through the remainder of the study.
  • On June 22, 2015, the Company announced positive preclinical in-vitro and in-vivo proof-of-concept study results of BBrm02 for SMA. BBrm02 is a proprietary, intrathecal formulation of azithromycin.
  • On June 25, 2015, the Company announced that it has been granted orphan drug designation from the European Commission for Cabaletta for the treatment of OPMD.
  • On June 29, 2015, the Company announced that it has been granted orphan drug designation from the European Commission for Cabaletta for the treatment of SCA. (Original Source)

Shares of Bio Blast Pharma Ltd closed yesterday at $5.23. ORPN has a 1-year high of $10 and a 1-year low of $4.50. The stock’s 50-day moving average is $6.37 and its 200-day moving average is $6.43.

On the ratings front, Roth Capital analyst Joseph Pantginis reiterated a Buy rating on ORPN, with a price target of $25, in a report issued on June 15. The current price target represents a potential upside of 378.0% from where the stock is currently trading. According to TipRanks.com, Pantginis has a total average return of -4.9%, a 34.2% success rate, and is ranked #3668 out of 3738 analysts.

Bio Blast Pharma Ltd is a development-stage biopharmaceutical company. It is engaged in the identification, licensing, acquisition, development and commercialization of drugs for rare and ultra-rare genetic and metabolic diseases.