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Rare Diseases: BioBlast Pharma IPO

July 16, 2014

BioBlast Pharma, a Tel Aviv, Israel-based clinical-stage biotech founded in 2012, is developing a platform of products for rare and ultra-rare genetic diseases. The company files an F-1 Form in February 2014, with the US Securities and Exchange Commission (SEC), to raise up to $37.5 million in an IPO. On July 7th, BioBlast Pharma files an amended F-1 Form with an increase in the amount the company is attempting to raise – up to $46 million. BioBlast Pharma plans to list on the NASDAQ with the symbol “ORPN”. Oppenheimer and Roth Capital are the joint bookrunners.

BioBlast Pharma’s most advanced product Cabaletta, a mutant protein stabilizing platform, is in a Phase II clinical trial (HOPEMD) for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD). Cabaletta is an IV formulation of Trehalose, which the company says enables the drug to reach muscles. Trehalose prevents aggregation of proteins in cells. OPMD is a rare genetic condition and form of muscular dystrophy that begins usually after age 40. It effects the muscles of the eyelid first, resulting usually in droopy eyelids (ptosis), followed by problems with swallowing (dysphagia). Patients with OPMD can also have weakness in the muscles near the center of the body (proximal muscles).

Other Cabaletta Preclinical Rare Disease Development Programs

•   Spinocerebellar Ataxia Type 3 (SCA3) or Machado Joseph Disease

•   Spino Bulbar Cerebellar Ataxia (SBMA) or Kennedy’s Disease.

SCA3 is a genetic disease where patients have memory deficits, difficulty with swallowing and speech, and other muscular disorders. SCA3 begins in early adolescence and progresses over time. It is an incurable disease with no currently approved treatment.

SBMA results in the degeneration and loss of lower motor neurons in the brainstem and spinal cord. There is currently no approved treatment.

Cabaletta FDA Orphan Drug Designation Database Record

Generic   Name: Trehalose
Trade   Name: Cabaletta
Date   Designated: 10-25-2013
Orphan   Designation: Occulopharyngeal Muscular Dystrophy
Orphan   Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan   Indication
Sponsor:   BIOBLAST PHARMA LTD. 35 Achad Haam Tel Aviv ISRAEL

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Other BioBlast Pharma Preclinical Rare Disease Pipeline Candidates

•   Spinal Muscular Atrophy (SMA)

•   Friedrich’s Ataxia (FA)

•   Ornithine Transcarbamylase Deficiency (OTCD).

Use of Some Net Proceeds From IPO

•   $6 million for completing clinical program for OPMD

•   $3.5 million for completing Phase II and pivotal clinical study for SCA3

•   $4.2 million for initiating and completing Phase II/III clinical study for SBMA

•   $4.5 million for completing pre-clinical program for FA and Phase I clinical study

•   $7.8 million for completing pre-clinical program for OTCD and initiation and completion of Phase I and IIA/B clinical studies

•   $2.2 million for initiating and completing Phase I/II clinical program for SMA.

Please Note: “Wall Street & Broadway” by Fletcher6 (Own work) [CC-BY-SA-3.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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