Alexion inks genomics pacts to continue R&D reboot 

Alexion has teamed up with GNS Healthcare and Sema4 to advance its rare disease R&D agenda. The partnerships give Alexion access to modeling and simulation software used by Celgene and a partner to support its push into genomics.

GNS is providing the modeling and simulation software, REFS. The platform, an acronym of reverse engineering and forward simulation, is intended to enable scientists to mine clinical, sequencing and omics data to generate models. These models are designed to improve understanding of the causes of rare diseases by enabling researchers to query them in search of insights into mechanisms and possible drug targets.  

Alexion will use REFS in its collaboration with Sema4. That alliance will see Alexion work on its tool for highlighting suspected rare-disease genes, SmartPanel, with the Mount Sinai Health System spinout. Alexion thinks it can improve SmartPanel by tapping into Sema4’s sequencing and genome interpretation capabilities.

REFS’ role in the Sema4 collaboration relates to a phenomenon Alexion has dubbed “genomic shields.” This term refers to the mechanisms that enable patients that have a mutation known to cause disease to resist manifesting the symptoms of the condition. If Alexion can understand these disconnects between genotype and phenotype, it may identify ways to treat patients in whom rare diseases are symptomatic.   

The Sema4 agreement comes two months after Alexion unveiled another pact built upon its SmartPanel. That earlier alliance saw Rady Children’s Institute for Genomic Medicine sign up to assess the tool’s ability to cut the time it takes to diagnose critically ill newborns with rare genetic conditions. 

If SmartPanel can accelerate diagnosis, it could lead to improved outcomes for newborns. Alexion would also realize boosts to its business. The rare disease business model is built on identifying the small numbers of patients with a particular condition and then selling expensive drugs to them for the rest of their lives. SmartPanel could identify users of Alexion drugs at the earliest possible opportunity, saving its team from searching for patients while maximizing sales. 

Alexion has entered into the three agreements against a backdrop of internal upheaval. Investigations into the sales practices that made Soliris a big seller cost the CEO and CFO their jobs late last year. And the new broom at the top, ex-Baxalta chief Ludwig Hantson, has started his reign by terminating deals with Moderna Therapeutics, Blueprint Medicines and Arbutus Biopharma and putting some in-house programs on ice.

The retooled R&D organization is going back to Alexion’s roots in complement biology. And, as the recent partnerships show, leaning on others to bolster its ability to identify patients with rare diseases and understand the genetics of their conditions.