Think about the orphans

Synageva BioPharma enters R&D partnership with Mitsubishi Tanabe Pharma to develop protein therapeutic for orphan disease target

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LEXINGTON, Mass.—Synageva BioPharmaCorp. recently announced that it has formed a research anddevelopment partnership with Osaka, Japan-based Mitsubishi TanabePharma Corp. to develop a novel therapeutic for an undisclosed orphandisease.
Under the terms of the deal, MitsubishiTanabe Pharma will make an upfront payment of $3 million and willcontribute additional research funds, getting in return Synageva'sexpertise in the rare disease space, its product developmentcapabilities and the benefits of its proprietary protein expressionplatform, according to Sanj K. Patel, Synageva's president and CEO.

Partnering is a particular focus ofSynageva's efforts on both the business and science sides of thecorporate equation, with the company noting on its website that it is"committed to becoming the leading innovator of therapies forpatients with rare diseases. We intend to further our mission toreach these patients by establishing partnerships with organizationsto license assets to partners or bring new assets into Synageva fordevelopment and commercialization."

That means everything fromout-licensing to in-licensing and technology licensing to productdevelopment collaborations like this one with Mitsubishi Tanabe.Looking to such development collaborations, the company notes that"Synageva's senior team has been involved in the development andcommercialization of nearly all of the novel protein therapeuticsmarketed to date for rare disease markets."

Although neither company would commenton the current deal, Synageva's Patel noted very specifically inthe news release that "this agreement illustrates Synageva'sstrategy to help fund our internal product development by selectivelyforming partnerships to create therapeutics for rare diseases nottargeted by our pipeline products." This suggests that the targetisn't related to Synageva's current pipeline, and the only otherthing Patel will say about the target is that the partnership "shouldresult in a new treatment for a devastating condition."

It remains to be seen whether that"devastating condition" is one of Mitsubishi Tanabe's currentpipeline targets, which domestically in Japan focuses on immunologyand inflammatory diseases, diabetes and lipid metabolism disordersand hepatic diseases, and in Europe and the United States is"centered on two products that have advanced to the developmentstage in the field of renal diseases," according to MitsubishiTanabe's website.

Synageva is a clinical-stagebiopharmaceutical company focused on the discovery, development andcommercialization of therapeutic products for patients withlife-threatening rare diseases and unmet medical need, with its majorproduct being SBC-102, which has been granted orphan designations bythe U.S. Food and Drug Administration (FDA) and the EuropeanMedicines Agency, and fast-track designation by the FDA.

SBC-102 is a recombinant humanlysosomal acid lipase currently under clinical investigation for thetreatment of patients with early-onset and late-onset lysosomal acidlipase deficiency—this progressive and often fatal lysosomalstorage disorder is also known as Wolman disease and cholesterylester storage disease.

In addition to SBC-102, Synageva hasother protein therapeutic programs targeting lysosomal storagedisorders and other rare diseases that are in various stages ofpreclinical development.

Synageva welcomes 'leading clinicianand rare disease pioneer'

LEXINGTON, Mass.—In other recentnews, Synageva BioPharma Corp. in early September announced theappointment of Dr. Mark Goldberg, to the position of senior vicepresident of product development, which the company maintains is "akey leadership position focused on progressing the company'sclinical-stage enzyme replacement therapy, SBC-102, towardsregulatory approval and advancing the company's other rare diseaseprograms."

At Genzyme, where he had previouslyserved as senior vice president of clinical development and globaltherapeutic group head of oncology and personalized genetic health,Goldberg was reportedly instrumental in the development and approvalof Fabrazyme, Aldurazyme, Myozyme and Lumizyme.

"Dr. Goldberg has had atransformational impact on the development of new treatments fordevastating rare diseases and we are thrilled to have him join ourworld-class leadership team," said Sanj K. Patel, president and CEOof Synageva BioPharma. "He paved the path to approval for many ofthe enzyme replacement therapies for lysosomal storage diseases andhe will bring that creativity and leadership to Synageva. Dr.Goldberg has already made an indelible mark on the company throughhis service on the board of directors and will now be able to applyhis wealth of experience to the success of our product pipeline."

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