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


Bio Blast Pharma Ltd (NASDAQ: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:

  • Research and development (R&D) expenses for the third quarter of 2015 were $1.71 million, compared to $2.14 million in the second quarter of 2015. The decrease was primarily due to a $0.5 million decrease in clinical study and manufacturing-related expenses associated with the Cabaletta platform, as well as a decrease of $0.15 million in pre-clinical study expenses, offset against a $0.27 million increase in human resources related expenses.
  • Pre-commercialization expenses for the third quarter of 2015 were $0.27 million and consisted primarily of expense related to professional services, salaries, and related share-based compensation. No pre-commercialization expenses were incurred in previous quarters.
  • General and administrative (G&A) expenses for the third quarter of 2015 were $1.61 million, compared to $1.6 million in the second quarter of 2015. There was no significant change between the quarters. Within the G&A expenses, the main changes were an increase of $0.19 million in intellectual property and legal expenses, offset by a decrease of $0.24 millionin share-based compensation.
  • Net loss for the third quarter of 2015 was $3.6 million, or $0.25 per share, compared to $3.69 million, or $0.26 per share, in the second quarter of 2015. The net loss for the third quarter excluding stock based compensation was $3.0 million, or$0.21 per share, compared to $3.0 million, or $0.21 per share in the second quarter.

Balance Sheet Highlights

  • Cash and cash equivalents (including short-term bank deposits) totaled $23.1 million as of September 30, 2015, compared to $32.6 million on December 31, 2014, and $34.1 million on September 30, 2014. The decrease compared to December 31, 2014 reflects the cash used in operations during the period.
  • Shareholders’ equity totaled $22.1 million as of September 30, 2015, compared to $30.7 million on December 31, 2014, and$33.4 million on September 30, 2014.

Third Quarter and Recent Corporate Highlights:

  • On September 10, 2015, the Company announced that it had appointed Paul Firuta, Former NPS Pharmaceuticals, Inc. andViroPharma Inc. executive, to the newly-created position of Chief Commercial Officer. This position has global responsibility and is based in the U.S.A. Additionally, Mr. Firuta will be a member of the executive management team and will oversee, in particular, the commercial strategy for Cabaletta – the Company’s lead product candidate currently in Phase 2 clinical development in the United States and Canada.
  • On September 14, 2015, the Company announced that it was granted a patent from the U.S. Patent and Trademark Office for Cabaletta entitled “Compositions and Methods for Treating Spinocerebellar Ataxia”.
  • On September 16, 2015, the Company announced that Tom Dubin, Former Alexion Senior Vice President & Chief Legal Officer had joined its Board of Directors, effective immediately.
  • On October 9, 2015, the Company announced that it had appointed Theresa (Terri) Stevens, former Aptalis and Novartisexecutive, to the newly-created position of Chief Corporate Development Officer with global responsibility based in the U.S.A. Ms. Stevens will be a member of the executive management team and will oversee corporate strategy, M&A, and business development of marketed and development stage in-and out-licensing.
  • On October 27, 2015, the Company announced positive interim results from its HOPEMD Phase 2 open label clinical study with its lead compound, Cabaletta, in 25 patients with OPMD. In this interim analysis, Cabaletta was observed to be safe and well-tolerated with no drug-related serious adverse events. Statistically significant improvement or numerical improvement versus baseline was observed on multiple efficacy endpoints. These interim results support the Company’s plan to move forward with a pivotal Phase 3 study in OPMD following meetings with regulatory authorities. OPMD is a rare progressive muscle-wasting disease characterized by severe swallowing difficulties (dysphagia) leading to malnutrition, dehydration, and aspiration of food into the lungs, as well as more generalized, progressive muscle weakness.

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 theUnited States and European Union.

  • On November 16, 2015, the Company announced the appointment of three new senior executives to its leadership team: Warren Wasiewski, M.D. as Chief Medical Officer and Vice President, Research & Development, Leigh Cherry as Vice President Manufacturing, and Stacey Jurchison as Vice President, Investor Relations & Corporate Communications. All are U.S.-based positions with global responsibilities. This is consistent with our objective of significantly enhancing our global reach and specifically, U.S. presence.

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.(Original Source)

Shares of Bio Blast Pharma closed yesterday at $4.15. ORPN has a 1-year high of $8.85 and a 1-year low of $3.92. The stock’s 50-day moving average is $5.06 and its 200-day moving average is $5.84.

On the ratings front, Bio Blast has been the subject of a number of recent research reports. In a report released yesterday, Oppenheimer analyst Ling Wang reiterated a Buy rating on ORPN, with a price target of $32, which represents a potential upside of 671.1% from where the stock is currently trading. Separately, on August 21, Roth Capital’s Joseph Pantginis maintained a Buy rating on the stock and has a price target of $25.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Ling Wang and Joseph Pantginis have a total average return of -6.8% and -4.6% respectively. Wang has a success rate of 29.2% and is ranked #3602 out of 3847 analysts, while Pantginis has a success rate of 34.9% and is ranked #3765.

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.