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Latest From Ipsen SA
Rocket's all-stock acquisition of Renovacor is valued at about $53m and adds a preclinical portfolio targeting genetically driven cardiovascular disease.
Focused on rare liver diseases such as acute-on-chronic liver failure following its 2020 disappointment in NASH, Genfit looks to become sector leader in ACLF and other indications such as hepatic encephalopathy.
A planned Phase II trial for PLK1 inhibitor onvansertib in KRAS-mutant colorectal cancer may support accelerated approval, but results are two years off and the company cut its prostate cancer plans to conserve cash.
Rare disease treatments have been increasingly attractive targets for investment, but clinical trials of these agents are laden with unique challenges, not least of which is that there are by definition small and possibly disparately located patient populations. However, by cultivating relationships with patient organizations and carefully designing studies, companies in the rare disease space are proving they can overcome logistical hurdles.
- OTC, Consumer
- Specialty Pharmaceuticals
- Other Names / Subsidiaries
- Clementia Pharmaceuticals, Inc.
- Epizyme, Inc.
- Ipsen Bioscience, Inc.
- Tercica, Inc.
- Syntaxin Ltd.
- OctreoPharm Sciences GmbH
- Octagen Corporation