The research cooperation between Santhera and the Biozentrum of the University of Basel relates to the area of congenital muscular dystrophies (CMDs), as was outlined in a press release from the Basel-based pharmaceutical company. CMD is an inherited neuromuscular disease. Professor Markus Rüegg from the Biozentrum has pioneered a novel gene therapy approach for the treatment of LAMA2 MD, a particularly severe form of CMD. The approach will be explored further in a collaboration between the University of Basel and Santhera. This research is supported by public funding through a grant from Innosuisse, the Swiss Innovation Promotion Agency.
In this cooperation, Santhera intends to build on its experience with omigapilin “which we have recently studied in a Phase I clinical trial in patients with CMD in a collaboration with experts at the National Institutes of Health (USA),” said Santhera CEO Thomas Meier in the press release. “From our previous work we anticipate that this novel gene therapy approach and omigapil could act complementary.” This collaboration is also perfectly in line with Santhera’s new strategy “to focus on the business areas core to its long-term growth strategy by advancing its clinical-stage neuromuscular and pulmonary programs,” as was reported in another press release.
This emphasis is made possible by the agreement with the Chiesi Group. It is licensing the Santhera drug Raxone, which was developed for the treatment of Leber’s hereditary optic neuropathy (LHON). The Chiesi Group will make an upfront cash payment of 50 million Swiss francs and could pay up to 105 million francs in total for the worldwide rights apart from Canada and the USA. It also has the option to fully acquire Santhera’s Raxone business. “This license agreement for Raxone in LHON, our sole neuro-ophthalmology asset, will allow us to create significant value for our shareholders as it provides financial resources enabling us to focus on delivering innovation to patients with neuromuscular and pulmonary diseases of great unmet medical need,” commented Thomas Meier.
One focus will be the treatment of CMD. The pharmaceutical company also intends to fully exploit its strategic position in the development of treatments for Duchenne muscular dystrophy (DMD). Santhera further plans to submit a conditional marketing authorization application for Puldysa to the European Medicine Agency that addresses “the needs of advanced DMD patients experiencing respiratory dysfunction”.