Viela Bio, Inc.

The FDA on Thursday approved Amgen’s rare disease drug Uplizna in an autoimmune disorder, setting the stage for the therapy to gain a foothold in the rare disease market. Uplizna is now approved to treat IgG4-related disease, a condition in which the immune system causes the formation of tumor-like masses that lead to organ damage. It’s also the first FDA-approved treatment for IgG4-related disease. Uplizna’s average wholesale acquisition cost is $140,248.50 per dose, an Amgen spokesperson said. Patients will take two initial loading doses followed by infusions every six months, leading to an annual cost of $280,497 after the first year. The new approval helps Amgen as it positions Uplizna as a drug that can potentially treat a variety of related rare autoimmune conditions. In addition to IgG4-related disease and the rare eye disorder, called neuromyelitis optica spectrum disorder, Amgen is also planning to submit the drug to the FDA for myasthenia gravis by July. AstraZeneca, UCB and argenx already have approved treatments for myasthenia gravis. “We’re covering a spectrum of cells that are responsible for generating antibodies,” Amgen SVP of global development Narimon Honarpour said. “It’s really about maximizing the opportunity once you have seen that your medicine has worked in one area, and knowing about the mechanism of action in a way that enables you to anticipate potential benefits in other conditions.” In the Phase 3 study for IgG4-related disease, Uplizna reduced the risk of a disease-caused flare by 87% after one year compared to placebo. It also achieved all secondary endpoints. Uplizna’s peak sales in IgG4-related disease could reach up to $3 billion, TD Cowen analyst Yaron Werber wrote in a note last year. He estimates the US market is between 7,000 to 14,000 patients. Though other companies haven’t focused much on IgG4-related disease, Uplizna may eventually have to carve out a slice of the myasthenia gravis market. AstraZeneca’s Soliris and Ultomiris drugs are both approved in this indication, as well as argenx’s Vyvgart. UCB’s Rystiggo and Zilbrysq are also approved for myasthenia gravis. Additionally, Johnson & Johnson, Immunovant and Biohaven are all developing drugs for myasthenia gravis that could reach the market in the next few years. The myasthenia gravis market was estimated to be about $1.5 billion last year, according to GlobalData, and forecasts for the next five to 10 years range from $3 billion to $7 billion. Uplizna succeeded in a Phase 3 trial in myasthenia gravis last September, and Amgen shared additional positive data last month. Amgen acquired Uplizna as part of its 2022 acquisition of Horizon Therapeutics, which got the drug in a buyout of Viela Bio. Editor’s note: This article was updated to include Uplizna’s pricing information.

Amgen has shared new data from a late-stage trial of its B cell-depleting therapy Uplizna (inebilizumab-cdon) in adults with the rare autoimmune disorder generalised myasthenia gravis (gMG). The phase 3 MINT trial evaluated the efficacy and safety of the drug in both muscle-specific kinase autoantibody (MuSK)-positive and acetylcholine receptor autoantibody (AChR)-positive gMG patients, who were followed for 26 and 52 weeks, respectively. Amgen previously reported that the trial met its primary endpoint, with Uplizna demonstrating a statistically significant change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores compared with placebo at week 26 in the combined study population. New results from the study have shown durable and sustained efficacy of Uplizna in patients with AChR-positive gMG with two doses a year, following an initial loading dose. Among these patients 72.3% of those receiving Uplizna had at least a three point improvement in the MG-ADL score, compared to 45.2% in placebo. Up to 100,000 people in the US are affected by myasthenia gravis, with gMG accounting for approximately 85% of all cases. The disease impairs neuromuscular communication and can cause muscle weakness, trouble breathing, difficulty swallowing and impaired speech and vision. Uplizna is a humanised monoclonal antibody that works by reducing the number of B cells, which are central to the pathogenesis of gMG. The drug already holds approvals to treat certain adults with neuromyelitis optica spectrum disorder, a rare autoimmune disease that attacks the optic nerve, spinal cord, brain and brainstem, and is under priority review in the US for immunoglobulin G4-related disease. Regulatory filing activities for Uplizna in gMG are underway, with submissions expected to be complete in the first half of 2025, according to Amgen. Jay Bradner, executive vice president of research and development at Amgen, said: “The 52-week MINT trial results highlight the potential for a new standard of care in gMG, offering durable symptom relief with a simplified treatment regimen. “These findings reinforce Uplizna’s ability to provide sustained symptom relief with just two doses per year – an important advancement for patients living with gMG…”

Bing Yao’s ArriVent BioPharma has inked another licensing deal with a Chinese biopharma. The late-stage oncology biotech is picking up a new antibody-drug conjugate from Shanghai-based Lepu Biopharma, according to a Tuesday night disclosure. The deal — for $47 million upfront and up to a hefty $1.16 billion in biobucks — adds to a steady drumbeat of US drug developers acquiring the ex-China rights to therapies initially created or developed by Chinese biotechs. It marks at least the 11th such China-to-US biotech pact in the first three weeks of 2025, according to an Endpoints News tracker. The move is core to the operating model of ArriVent, which debuted in June 2021 with the goal of picking up drug candidates and medicines from Chinese biotechs and bringing them into the US and other geographies. The Pennsylvania biotech also has ADC collaborations with Alphamab and Aarvik Therapeutics. Lepu, meanwhile, has a joint venture with Keymed Biosciences called KYM Biosciences. AstraZeneca licensed an ADC from KYM in February 2023. Keymed has also done multiple China-to-US deals of its own in recent months. ArriVent’s pipeline is led by an oral, brain penetrant EGFR inhibitor known as furmonertinib. The medicine is approved in China, where it’s marketed by ArriVent partner Allist Pharmaceuticals. Global Phase 3 non-small cell lung cancer data are slated for this year, ArriVent said in its most recent quarterly update. With Lepu, ArriVent is staking a bet on MRG007, an experimental medicine for gastrointestinal cancers. ADCs like MRG007 are intended to target tumor cells with cytotoxic payloads and have become darlings among pharma’s oncology dealmaking teams in recent years. MRG007 will enter human trials later this year, the companies said in their announcement on Tuesday evening, or Wednesday morning in China . The move does not change ArriVent’s previously projected cash runway, which should take it “into 2026,” the biotech said. ArriVent said in November that it ended the third quarter with $282.9 million in cash and equivalents. The drug developer went public as $AVBP almost a year ago in a $175 million IPO, and it’s one of the few biotechs to have floated in the last few years that is still trading above its initial pricing. ArriVent is led by Yao, who previously took AstraZeneca spinout Viela Bio to an FDA approval for Uplizna in 2020 and then an M&A exit to Horizon Therapeutics a year later.