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Orphan Drugs And Rare Diseases: Partnerships And Collaborations

May 16, 2013

The following discusses new partnerships and collaborations in the area of orphan drugs and rare diseases :

I – Siren Interactive

Parent Project Muscular Dystrophy (PPMD), a nonprofit focusing on Duchenne Muscular Dystrophy (DMD), launches in April 2013 the Duchenne Central mobile app in collaboration with Siren Interactive, a digital relationship marketing agency for rare disease therapies. The mobile app “is an easy-to-use, on-the-go tool that enables the Duchenne community to learn about and locate clinical trials and clinics.” The app is available for download for the iPhone and Android.

Liquid Grids, an all-in-one social marketing platform, announces on March 15, 2013, a partnership with Siren Interactive  to “find and engage chronic rare disease populations via social media.” According to the Press Release, the following are the three benefits :

•   Opportunity to effectively understand and identify the material focus of those in the chronic rare disease community

•   Capacity to identify and develop contextual messaging for the distinct personas of each group

•   Opportunity for scalable engagement outreach through social media that will guarantee impressions within the target chronic rare disease.

II – Ultragenyx And MPS7

Ultragenyx is collaborating with Dr. William Sly and colleagues at St. Louis University for the development of UX003 for MPS 7.

Ultragenyx, a California biotechnology company bringing new therapies for rare and ultra-rare genetic diseases, announces on May 15, 2013, a Phase I/II study of UX003 for Mucopolysaccharidosis Type 7 (MPS 7 or Sly Syndrome). UX003 is an Enzyme Replacement Therapy (ERT) for the treatment of patients with MPS 7. MPS 7 is a rare autosomal recessive lysosomal storage disorder with a deficiency of the  lysosomal enzyme β-glucuronidase and a severe multi-system disease. MPS 7 currently has no approved therapies.

III – Isis Pharmaceuticals

In April 2013, Roche and Isis Pharmaceuticals form an alliance to develop treatments for Huntington’s Disease (HD). The alliance will combine Isis Pharmaceuticals’ Antisense Oligonucleotide (ASO) technology and Roche’s expertise in developing neurodegenerative therapies. Under the agreement :

•   Roche makes an upfront payment of $30 million to Isis Pharmaceuticals
•   Total payments for license fee and pre- and post-licensing milestone payments can potentially reach $362 million, including up to $80 million in potential commercial milestone payments
•   Isis Pharmaceuticals to receive tiered royalties on sales of drugs
•   Roche has the option to license drugs from Isis Pharmaceuticals through completion of the first Phase I trial
•   Isis Pharmaceuticals, prior to the option exercise, is responsible for the discovery and the development of an antisense drug targeting the HTT (Huntingtin) protein responsible for HD
•   Both companies will work collaboratively on the discovery of an antisense drug using Roche’s “brain shuttle” program
•   If Roche exercises its option, the company is responsible for global development, regulatory, and commercialization for all drugs from the collaboration.

Isis Pharmaceuticals is also collaborating with Biogen Idec to develop and potentially commercialize an investigational compound, ISIS-SMNRx, to treat all types of Spinal Muscular Atrophy (SMA). Under the January 2012 agreement between the two companies, Isis Pharmaceuticals is responsible for global development and Biogen Idec has the option to license the compound until completion of the first successful Phase II/III study. Isis Pharmaceuticals anounces May 9, 2013, that it has earned a $3.5 million milestone payment from Biogen Idec as a result of the dosing of the first patient in a Phase II study of ISIS-SMNRx in infants with SMA.

IV – Others

As already mentioned in previous Orphan Druganaut Blog Posts, the following are two recent well publicized partnerships :

•   Shire and Atlas Venture to making early-stage investments in rare disease opportunities with Nimbus Discovery
•   Orphan drug accelerator Cydan receiving $16 million from Pfizer Venture Investments and New Enterprise Associates, to partner with rare disease stakeholders for the purpose of picking the most promising rare disease programs to start companies around the programs.

Please Note: Social Media Marketing by Paola Peralta (Own work) [CC-BY-SA-3.0] | Wikimedia Commons.

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

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