Novartis farms out rare disease drug to LifeMax Labs

Netherton syndrome is caused by mutations in the SPINK5 gene, which codes for LEKT1, a protein that inhibits the activity of certain enzymes called serine peptidases. (Novartis)

Novartis is licensing a treatment for Netherton syndrome, a rare genetic disorder, to LifeMax Laboratories, a biotech based in the San Francisco area looking to address diseases that have limited treatment options. 

LifeMax Labs, a subsidiary of LifeMax Healthcare, picks up the global rights to develop, manufacture and market BPR277, a topical treatment in the clinic for atopic dermatitis and Netherton syndrome. In return, Novartis will receive an upfront payment, development and regulatory milestones and royalties on net sales. Financial details were not disclosed. 

Netherton syndrome is caused by mutations in the SPINK5 gene, which codes for LEKT1, a protein that inhibits the activity of certain enzymes called serine peptidases. Patients with Netherton make a form of LEKT1 that cannot control serine peptidase activity, which causes various symptoms including short, brittle hair, a predisposition to allergy problems such as asthma and eczema and inflamed, red, scaly skin. There are no FDA-approved therapies specifically for Netherton. Treatment focuses on managing symptoms and avoiding skin infections and other complications. 

“We are very excited to enter into this licensing agreement with Novartis and are committed to continuing the development of BPR277 which fits well with our corporate strategy of developing drugs for diseases with few or no therapeutic options," said LifeMax CEO Larry Hsu in a statement. Hsu co-founded LifeMax and previously founded Impax Laboratories.

RELATED: BridgeBio Pharma gains $135M for genetic disease work 

The company could potentially develop BPR277 for other diseases “with skin barrier impairment,” Hsu said.

LifeMax isn't alone in trying to address Netherton syndrome. Pittsburgh-based Krystal Biotech filed in August 2017 to raise about $35 million in its IPO, funds that were earmarked to advance its lead asset, a gene therapy for dystrophic epidermolysis bullosa, but that would also push a gene therapy for Netherton into the IND stage by the second half of this year. And BridgeBio Pharma reeled in $135 million last September to support a clutch of early-stage programs, including transthyretin amyloidosis, pantothenate kinase-associated neurodegeneration, dystrophic epidermolysis bullosa and various cancers in addition to Netherton.

The LifeMax deal comes after Novartis out-licensed a handful of cancer drugs. These include the failed multi-kinase inhibitor dovitinib and a pair of oral pan-Akt kinase inhibitors picked up in its asset swap with GlaxoSmithKline back in 2014. The Swiss pharma licensed dovitinib—which did not beat Bayer’s Nexavar in phase 3—to Oncology Venture in another deal of undisclosed size. It handed off GSK-developed afuresertib (ASB183) and uprosertib (UPB795), which had entered the clinic for a variety of cancers, to Shanghai-based Laekna. 


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