Choosing to exercise its options with a group of research and development partners, the pharma giant will accelerate a promising new C.F. gene therapy.
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Germany’s Boehringer Ingelheim is taking its commitment to patients with cystic fibrosis to the next level. Choosing to exercise its options with a group of research and development partners, the pharma giant will accelerate a promising new C.F. gene therapy.
In a perfect union of academia, pharma, and biotech, Boehringer Ingelheim will snag intellectual property from I.P. Group and the U.K. Cystic Fibrosis Gene Therapy Consortium (GTC) while utilizing Oxford BioMedica’s lentiviral vector technology to bring BI 3720931 through clinical trials to approval and market.
Per the deal announced in August 2018, Boehringer Ingelheim will dish out an option exercise fee of about $4.8 million to Oxford Biomedica, with up to nearly $40 million in milestone payments on the line.
BI 3720931 is an inhaled formulation that introduces a healthy CFTR gene into target cells. Mutations to this gene are what cause cystic fibrosis. Currently, about 70,000 people are living with the rare, life-threatening disease worldwide.
It is estimated that two million people in the U.K. carry the faulty gene without realizing it. If two carriers have a child, there is a 25% chance the child will have C.F. The mutation causes the CFTR protein to malfunction, leading to a buildup of thick mucus that causes lung infections, pancreas destruction, and other organ complications. The average lifespan with today’s treatments is mid-forties.
Professor Eric Alton, the coordinator of the GTC, said, “The novel lung-targeting technology we have developed has demonstrated high gene transfer efficiency in pre-clinical models and offers the possibility of repeated administration to maintain a therapeutic effect, a benefit that other viral-based gene therapies may not be able to provide.
“Our novel therapy has the potential to improve CFTR function and modify disease in all C.F. patients, independent of the more than 2,000 different known gene mutations. The immediate target is those patients who are not eligible for CFTR modulators.”
Boehringer Ingelheim is also making waves in the biosimilar market. Its adalimumab biosimilar Cyltezo, approved in August 2017, is among the first Humira biosimilars to launch in July 2023. In 2019, Humira was one of the top-selling pharmaceuticals in the U.S. market. In 2020, the drug generated $19.83 billion in revenue.
Six biosimilars are coming onto the market in 2023, but Boehringer Ingelheim has a leg up on the competition with its interchangeable designation granted by the FDA. The approval means pharmacists can substitute Cyltezo for Humira without consulting the prescribing doctor. Cyltezo will be interchangeable across all Humira’s indications.
