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Rare disease-focused Prosensa brings in US investor in new €23 million round

This article was originally published in Scrip

Prosensa, the Dutch firm focused on RNA-modulating therapies for rare diseases, has raised €23 million in new venture capital funding. The round was led by new investor New Enterprise Associates and included existing investors Abingworth, Life Sciences Partners, Gimv, Idinvest Partners and MedSciences Capital.

"Prosensa is our first biopharma investment in Europe and signals our openness to selectively consider investing…in Europe as well as in our traditional geographies in the US, China and India," said David Mott, general partner at NEA.

"Bringing a US investor on board has an important strategic impact," Prosensa's chief business officer Luc Dochez told Scrip. "It is another validation of the company's progress, next to the validation of its top-notch European investors as well as its pharmaceutical partner GlaxoSmithKline. NEA has a large network and in-depth experience of the US market which will be helpful and beneficial to realise the company's ambition to become a global rare disease player."

Mr Dochez said that the new cash, which will fund the company "well into 2014", will be used to further advance the development of its R&D pipeline. "Thanks to this financing, more compounds targeting additional, but distinct, subgroups of Duchenne muscular dystrophy patients will be advanced into the clinic," he explained. "The first fully unencumbered, non-partnered compound will enter into a clinical study in the second half of this year and Prosensa has now raised the necessary funds to bring this forward to patients completely by itself. The funds also enable the further development into other diseases, such as Myotonic Dystrophy and Huntington's disease. Both are also rare diseases with a significant unmet medical need, and we plan to further progress the development of selected compounds here as well."

Prosensa has two compounds in the clinical trials in partnership with GSK (PRO051/GSK2402968 and PRO044) and four more compounds in preclinical development, as well as preclinical compounds for Myotonic Dystrophy and Huntington's disease.

Prosensa's Duchenne muscular dystrophy compounds are based on its exon-skipping technology that uses oligonucleotides to restore expression of a functional dystrophin protein.

"The collaboration between GSK and Prosensa is going very well," said Mr Dochez. "Beyond PRO051/GSK2402968, which is currently in Phase III and PRO044 in Phase I/II, GSK has option rights to two additional exon-skipping programmes in Duchenne muscular dystrophy. In one programme we expect to initiate a clinical study in the second half of 2012. The other programme we anticipate the identification of a clinical candidate. Development into the clinic is expected then to follow in the course of 2013."

All of Prosensa's other exon-skipping programmes fall outside the GSK collaboration, he highlighted. "Prosensa has unencumbered rights to these programmes. The first un-partnered asset will enter clinical development in the second half of 2012, whereas the second one is expected to follow in the course of 2013."

Asked about Prosensa's partnering plans, Mr Dochez said: "We will explore additional partnering opportunities for our other pipeline programmes. Our RNA modulation platform is broadly applicable to both rare and common diseases and we believe that a significant part of the value is yet untapped. The company has grown over the years thanks to a very active partnering strategy, partnering with academic groups, patient organisations as well a large pharmaceutical company and we will continue to do so in the next years."

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