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Duchenne Muscular Dystrophy: Business Developments (08.07.13)

August 7, 2013

Duchenne Muscular Dystrophy (DMD) has attracted a lot of interest from both investors and the media recently. This is the second Blog Post that discusses  and reviews recent business developments for DMD (first Blog Post).

I – Prosensa

Prosensa, a Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases, announces on August 1, 2013, that the company is awarded a prestigious Framework Programme 7 (FP7) research grant from the European Union (EU). The FP7 research grant will support the ongoing clinical study of Prosensa’s novel development candidate, PRO045, for the treatment of DMD. The grant is approximately EUR 6 million (approximately US $7.9 million). The project, “Consortium for Products Across Europe in Duchenne Muscular Dystrophy (SCOPE-DMD)”, will run for three years. The study will evaluate PRO045 in an innovatively designed clinical proof-of-concept study in boys with DMD. Hans Schikan, CEO of Prosensa says that:

“FP7 grants are awarded on the basis of a highly competitive, two-stage, peer-review process, therefore this award serves as validation of our exon-skipping technology platform. We very much look forward to working with this esteemed consortium on PRO045 and are grateful to the EU for its support of important clinical research initiatives …”.  

PRO045 is currently in a Phase I/IIa dose-escalating safety study to assess its safety and efficacy. The compound induces exon 45 skipping in the dystrophin gene and could be suitable for an estimated 8% of all DMD patients. PRO045 has orphan drug status in the EU and the US.

Prosensa currently has 6 exon-skipping compounds in development for treating DMD. The FDA grants ODD to Prosensa’s 4 preclinical compounds PrO045, PRO052, PRO053, and PRO055 DMD in January 2013. Prosensa’s 2 other Exon-specific compounds, PRO044 and PRO051 (Drisapersen), receive ODD prior to 2013.  PRO051 (Drisapersen), is being developed in collaboration with GlaxoSmithKline.  Drisapersen receives in June 2013, the FDA Breakthrough Therapy Designation.

The chart below is generated from the FDA’s Orphan Drug Product Designation Database Application.

Generic Name Designation Date Indication
Exon 44 specific   phosphorothioate oligonucleotide 11.05.09 DMD
Exon 45 specific phosphorothioate   oligonucleotide 01.23.13 DMD
Exon 51 specific   phosphorothioate oligonucleotide 08.25.09 DMD
Exon 52 specific phosphorothioate   oligonucleotide 01.23.13 DMD
Exon 53 specific phosphorothioate   oligonucleotide 01.23.13 DMD
Exon 55 specific phosphorothioate   oligonucleotide 01.23.13 DMD

II – Summit plc

Summit plc, a drug discovery and development company focusing on DMD, announces on July 18, 2013, that the company has entered a funding agreement with the Australian DMD foundation, Save Our Sons (‘S.O.S.’). The agreement is worth up to AUD 1.25 million (approximately US $1.12 million). The funding will support the development of SMT C1100, the company’s utrophin modulator drug for DMD.

Just two weeks later, Summit plc announces on July 31, that the company is awarded a grant of £2.4 million (approximately US $3.7 million) from the UK Biomedical Catalyst Fund. The grant will support the clinical development of SMT C1100 – Phase Ib and Phase II clinical trials, long-term toxicology studies, and the development of novel biomarkers for use in the Phase II clinical trial. The UK Biomedical Catalyst Fund is a “program of public funding designed to deliver growth to the UK life sciences.”

Please Note: “AZT” by John Crawford (Photographer); NIH, US Federal Gov’t [Public Domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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