Alexion Pharmaceuticals, which earlier this week confirmed axing 7% of its workforce, negotiated a license to use Arbutus Biopharma’s lipid nanoparticle (LNP) delivery technology for one of its rare disease messenger RNA (mRNA) therapeutic candidates. Arbutus will earn $7.5 million upfront from Alexion and potentially another $75 million in development, regulatory, and commercial milestones, plus single-digit sales royalties. The firm will also carry out technology development and provide manufacturing and regulatory support for the Alexion mRNA candidate.

“This agreement validates our leadership position in LNP technology and underscores the value of this platform in enabling RNA therapeutics,” commented Mark J. Murray, Ph.D., Arbutus’ president and CEO. “We are excited to be working with Alexion to advance a treatment for rare disease. …This transaction with Alexion illustrates the value of our LNP platform, which has broad potential to deliver mRNA and gene-editing therapeutics.”

Arbutus is focused on developing a pipeline of antiviral and immune stimulation candidates for hepatitis B virus (HBV) therapy. The firm’s lead LNP-delivered RNA interference (RNAi) candidate ARB-1467 (formerly TKM-HBV) is undergoing a Phase II multidose study in chronically infected HBV patients receiving stable nucleos(t)ide therapy. Positive data from the ongoing study were reported in December 2016. Arbutus' core protein/capsid assembly inhibitor AB-423 is in preclinical development. 

The firm is, in addition, developing a pipeline of non-HBV assets, including the Polo-like kinase 1 (PLK1)-targeting candidate TKM-PLK1, which has been evaluated in a Phase /II clinical trial in patients with hepatocellular carcinoma, gastrointestinal neuroendocrine tumors (GI-NET), and adrenocortical carcinoma.

Amid board reshufflings, restructuring, and job cuts, Alexion also reported earlier this month that the FDA had accepted for review a supplemental Biologics License Application (sBLA) to extend the indication for its flagship product Soliris® (eculizumab) as a treatment for patients with refractory generalized myasthenia gravis (gMG) who are antiacetylcholine receptor (AChR) antibody positive. A PDUFA date of October 23rd has been set. A marketing authorization application for the gMG indication has also been filed in the EU.

Soliris is approved for treating paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The drug is, in addition, undergoing clinical development for treating relapsing neuromyelitis optica spectrum disorder and antibody-mediated rejection. Alexion’s other marketed products include Strensiq® (asfotase alfa), an enzyme replacement therapy for treating prenatal, infantile-, and juvenile-onset hypophosphatasia (HPP). Kanuma® (sebelipase alfa) is an enzyme replacement therapy for treating lysosomal acid lipase deficiency (LAL-D).

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