Bio Blast Pharma Ltd. (ORPN) Stock | Positive Interim Results of Phase 2 Study


Bio Blast Pharma Ltd.  (NASDAQ: ORPN)


Bio Blast Pharma Ltd.  (ORPN), a developmental stage biopharmaceutical company yesterday announced positive interim results from HOPEMD phase 2 clinical study of Cabaletta. Cabaletta is used treat patients with oculopharyngeal muscular dystrophy. The study so far has shown statistical improvement versus the baseline.


Bio Blast Pharma Ltd. CEO Comments

“These results support our plan to move forward with a pivotal Phase 3 study in OPMD following pending meetings with regulatory authorities,” said Colin Foster, President and Chief Executive Officer, BioBlast. “We plan to share these interim data and the complete Phase 2 six month data set with the FDA. Following these interactions and based on the open IND, Orphan Drug designation, and Fast Track designation we have been granted for this program, our goal is to advance with a Phase 3 study in the U.S. and in Canada”.

“The interim results for OPMD, specifically with respect to dysphagia and muscle strength and function, give us 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 we are planning a pivotal Phase 3 study in the U.S. and E.U.,” Mr. Foster continued. “We will provide guidance in the future regarding the design and initiation of both the OPMD and SCA3 Phase 3 studies following discussions with regulatory authorities in each market.”  Globe Newswire


ORPN Technical Analysis


ORPN gapped up in price yesterday to $6.29, up from the prior day’s close of $5.04, which is a 25% increase in price based on favorable news. Taking a look at the daily chart, we can see the last time the stock traded above this price level was on September 21st, when it closed at $6.43. Taking a further look back on the chart, we can see the stock has been in an overall down trend dating back to June 24th when it was trading at $8.14. More recently, the stock has been in a tight trading range between $5.00-$5.60 for the last 3 weeks until this gap up in price. The stock does have an extremely low float of 4.09 million shares and is trading on over 67 times the normal daily trading volume. Both of these factors make the stock a prime candidate for the gap and go strategy. The stock did reach pre market highs of $6.60 but gave back $0.31 at the open, or equivalent to 5%. For trading purposes, my entry point would have been $6.40 looking for a run through pre market highs of $6.60. My stop loss would have been $6.25 fearing anything below that and the stock would start to fill in the gap up.


Company Profile

Bio Blast Pharma Ltd., a development stage biopharmaceutical company, focuses on the identification, licensing, acquisition, development, and commercialization of drugs for rare and ultra-rare genetic and metabolic diseases. The company develops Cabaletta, which is in Phase II/III clinical trials for the treatment of oculopharyngeal muscular dystrophy; and Phase II clinical trials for the treatment of spinocerebellar ataxia type 3, as well as in preclinical studies for the treatment of spinobulbar muscular atrophy. Its preclinical development stage product candidates comprise BBrm1 for spinal muscular atrophy; BB-FA for the treatment of friedrich’s ataxia; and BB-OTC for ornithine transcarbamylase deficiency. Bio Blast Pharma Ltd. was founded in 2012 and is headquartered in Tel Aviv, Israel.  Yahoo Finance