- UCB Pharma has signed two deals aimed at building out its gene therapy pipeline, announcing Thursday that it will buy Belgium-based Handl Therapeutics and collaborate with University of Florida-spinout Lacerta Therapeutics. UCB did not disclose how much it is paying in either transaction.
- Handl and Lacerta both aim to treat neurological disorders using corrective genes delivered by adeno-associated virus, or AAV, vectors. If successful, their research would help augment UCB's existing portfolio in central nervous system disorders, which includes the anti-seizure drugs Vimpat and Keppra.
- UCB faces the loss of market exclusivity of its biggest seller, the rheumatoid arthritis drug Cimzia, beginning in 2021 in Europe and 2024 in the U.S. Vimpat, its only other blockbuster, loses U.S. patent protection in 2022. To make up for expected loss of revenue, the company will need its pipeline assets, currently led by psoriasis drug bimekizumab, to succeed.
Cimzia had sales of about $2 billion and Vimpat added another $1.6 billion more in 2019, accounting for close to two-thirds of UCB's total sales. Those sales will decline following patent expirations, requiring the company to come up with new sources of revenue.
With four active late-stage experimental drugs, UCB is trying to position itself for the post-Cimzia era. However, its lead asset, bimekizumab, will be entering a crowded psoriasis space, and the way it works is similar to more recently approved drugs like Taltz from Eli Lilly and Cosentyx from Novartis.
Other experimental medicines, however, focus on diseases with fewer therapeutic options, including myasthenia gravis, lupus and active seizures. Gene therapy capabilities would also help UCB build up its portfolio in rare neurological disorders, which the two transactions announced Thursday are meant to do.
UCB started working in gene therapy when it acquired Duke University spinout Element Genomics. With the buyout of Handl today, the Belgian pharma gains an AAV technology platform with a neurodegenerative focus, and two research programs. Handl's platform has been built from technology licensed from four universities in Europe, the U.K. and Chile.
Under the collaboration with Lacerta, the gene therapy startup will conduct research, preclinical activities and the early manufacturing process development, while UCB will do studies to prepare for an Investigational New Drug application, along with manufacturing and clinical development.
Lacerta launched in 2017 and got a boost with a $30 million investment from Sarepta Therapeutics in 2018, which gave the Massachusetts biotech rights to gene therapy programs focusing on the central nervous system.