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BioBlast Pharma Ltd (NASDAQ:ORPN) Blasts Off

BioBlast Pharma Ltd (NASDAQ:ORPN) Blasts Off
Written by
Alex Carlson
Published on
March 22, 2016
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Shares of BioBlast Pharma Ltd (NASDAQ:ORPN) exploded up the charts after the company's Cabaletta treatment showed positive effects in patients with rare muscle-wasting disease who have difficulty swallowing. While shares did come back down after the company did an equity raise, we think shares are consolidating before heading back towards the $8 number the stock hit on the news.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 platforms are based on a deep understanding of disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology.Final results from a six-month open-label proof-of-concept Phase 2 study, HOPEMD, assessing BioBlast Pharma's Cabaletta (IV trehalose) in patients with oculopharyngeal muscular dystrophy (OPMD) showed a positive treatment benefit in addition to being safe and well-tolerated. The primary objective of the trial was to evaluate the safety and tolerability of trehalose 90 mg/mL IV solution. Secondary endpoints explored its effect on OPMD disease markers, including difficulty swallowing (dysphagia) and muscle weakness, although the study was not powered to demonstrate statistically significant efficacy.Trehalose 90 mg/mL was safe and well-tolerated with no drug-related serious adverse events. No patients withdrew from the study due to treatment-related issues. There was one death, due to aspiration pneumonia, not considered drug-related. The drug's effect on dysphagia was assessed several ways: a timed cold water drinking test (80 mL), nectar and honey (80 mL) timed drinking tests and Penetration Aspiration Score as measured by video fluoroscopy (VFS-PAS), a technique to assess the severity of swallowing difficulties and risk of aspiration. A patient-reported questionnaire, SWAL-QOL, was used to assess the degree to which patients felt their swallowing improved with treatment.Participants (n=23) experienced a mean reduction of 31.8% in the time to complete the cold water drinking test. The average reductions in the nectar and honey drinking test times were 43.8% and 46.6%, respectively (n=11). In the VFS-PAS analysis (n=11), six patients improved, two were stable and three deteriorated. There was a 12.7% (n=24) improvement versus baseline in mean SWAL-QOL score.Measurements of the change in muscle strength and function also showed a positive treatment effect. The company plans to initiate a Phase 2b study in OPMD to beef up its data package and to guide the design of a Phase 3 trial, expected to commence in 2017.Sort of taking the shine off this news was the announcement that BioBlast entered into definitive securities purchase agreements with two healthcare focused institutional investors providing for the issuance of approximately 2.16 million ordinary shares at a purchase price per share of $3.10 in a registered direct offering and warrants to purchase up to approximately 1.08 million ordinary shares with an exercise price of $4.50 with gross proceeds of approximately $6.7 million. The warrants are initially exercisable six months following issuance and have a term of five and one-half years following issuance.BioBlast finished 2015 with cash and cash equivalents (including short-term bank deposits) totaling $19.3 million. We feel that the company jumped the gun too quickly to raise money and would have been better off raising money later at a higher valuation. Our guess is that the company already had this offering in the works already and had no choice but to go through with it.Currently trading at a $39 million market cap, ORPN has proven that it has runner in its blood and can explode higher up the charts. We feel that shares will steadily trend higher from here on out. We will be updating Insider Financial as soon as we know more. For continuing coverage on ORPN, sign up for our free newsletter today and get our next hot stock pick!Disclosure: We have no position in ORPN and have not been compensated for this article.

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