Horizon claims mid-stage victory in rare disease, lines up PhIII for new owners at Amgen

As Horizon ties up the loose ends for its $28 billion sale to Amgen, a drug it picked up from a previous acquisition of its own is delivering a mid-stage win.

Horizon had already declared Phase II success for dazodalibep among one group of patients with Sjögren’s syndrome back in September. Now, it is announcing that the drug also met the primary endpoint for the second patient population in the trial, setting up a 2023 launch for a Phase III program it will work with the FDA to design. That timeline is ahead of expectations, the company added.

A CD40 ligand antagonist that blocks T cell interaction with CD40-expressing B cells, dazodalibep came to Horizon by way of Viela Bio, which in turn got the drug from AstraZeneca alongside a slate of other rare disease programs that were being swept out in a reorg.

By the time Horizon bought Viela for close to $3 billion in 2021, Viela had already started a Phase II trial in Sjögren’s syndrome, a chronic autoimmune disease affecting salivary and tear glands but also multiple organs in severe cases.

It enrolled two different groups of patients: The first 74 patients had moderate-to-high systemic disease as defined by a gold standard activity index. The second group, with 109 patients, had only mild systemic disease activity based on that index, but were considered moderate-to-severe based on a different score of subjective symptoms.

Investigators tracked those symptoms, including dryness, pain and fatigue despite lacking additional organ issues, on a composite score dubbed ESSPRI.

“Participants in this study had been excluded from other recent trials, despite their substantial disease burden,” said Frederick Vivino, former director of the Penn Sjögren’s Center and chief, division of rheumatology at Penn Presbyterian Medical Center, in a prepared statement.

Elizabeth Thompson

At Day 169, patients treated with dazodalibep achieved a 1.8-point reduction in their ESSPRI scores compared to placebo-treated patients, who achieved a 0.53-point reduction. According to Horizon, that’s good for a least squares mean difference of 1.27 (p=0.0002).

Elizabeth Thompson, Horizon’s executive vice president of R&D, described it as a “significant separation in symptom intensity.”

In the first batch of Phase II results released on the first group of patients, the drug spurred a 6.3-point reduction on the ESSDAI score compared to 4.1 points on placebo, translating to a least squares mean difference of 2.2 points (p=0.017).

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