Acelyrin eye drug heads to late-stage testing after trial win

A young biotechnology company plans to move an experimental medicine for a rare eye disease into late-stage testing after results from a small study signaled that it is generally safe and works as intended.

On Wednesday, Los Angeles-based Acelyrin said a Phase 2b/3 trial will begin in the back half of this year — the first of two studies the company hopes will serve as the foundation for an approval filing with the Food and Drug Administration. Acelyrin has been testing its medicine, known scientifically as lonigutamab, in people with thyroid eye disease, an inflammatory condition that progressively damages tissues around the eye.

In that smaller trial, participants were divided into two groups. “Cohort 1” was placebo-controlled, with two patients in the control arm and six patients given 40 mg of lonigutamab every three weeks over the course of six weeks. “Cohort 2,” meanwhile, was open-label and used a dosing schedule of one 50 mg “loading dose” followed by once-weekly 25 mg injections. Acelyrin said six-week data were available from six patients in this cohort.

According to the company, there were “clinically meaningful responses” across each of the main ways the trial measured the effectiveness of lonigutamab. Among the drug-treated participants in Cohort 1, one quarter improved by at least one point on a double vision scale; one-half improved by at least two points on an eyeball protrusion scale; and all improved by at least two points on a “clinical activity score” — which evaluates symptoms of thyroid eye disease.

Neither of the two placebo-treated patients showed such improvements.

In Cohort 2, two, four and five of the six participants had responses across those three measurements, respectively.

Acelyrin noted that patients tolerated its drug well. So far, researchers have not reported any serious adverse events or cases of hearing loss or high blood sugar. An antibody drug, lonigutamab is designed to block a protein tied to insulin-like growth factor-1, a hormone that affects insulin sensitivity as well as the growth of bones and tissues.

An already approved drug for thyroid eye disease, Tepezza, targets the same protein. But unlike Acelyrin’s medicine, which is given via an under-the-skin injection, it’s administered as an intravenous infusion over the course of 60 to 90 minutes.

Genmab and Roche originally investigated Tepezza as a potential cancer treatment, before a small biotech named River Vision began developing it in eye disease. Horizon Therapeutics bought River Vision in 2017 for an upfront cash payment of $145 million. Then, six years later, Amgen acquired Horizon for nearly $28 billion.

Within Horizon’s portfolio of therapies for rare and immune system-related diseases, Tepezza stood out as especially lucrative. First approved in January 2020, the drug has reached blockbuster status in each of the last three years. In 2022, net sales were nearly $2 billion. Amgen, which completed the Horizon acquisition on Oct. 6, recorded $448 million worth of Tepezza sales between then and Dec. 31.

Acelyrin’s drug, should it ultimately secure approval, would compete with Tepezza and may even hold an advantage since it’s given as a quick injection.

“It is important to note that this is preliminary data in a small group, however, the positive results are highly promising,” said Shoaib Ugradar, a private practice ophthalmology specialist, in a statement from Acelyrin.

“Given the growing body of evidence that suggests thyroid eye disease may have long-term sequelae, the convenience of a subcutaneous administered medication with a potentially favorable side effect profile becomes critical,” Ugradar added.

But Acelyrin isn’t alone. Viridian Therapeutics is advancing a similar type of medicine. One intravenous version is already in Phase 3 testing, while another in the form of a subcutaneous injection could follow later this year. Though it’s tough to compare drugs across trials, overall Viridian’s “looks better at this time,” wrote Jefferies analyst Michael Yee.

The results “suggest … lonigutamab is an active drug,” but “with significant caveats around all of the data that we haven’t seen,” added Leerink Partners analyst Thomas Smith, in a note to clients. 

Acelyrin said further data will be presented at future scientific meetings.

Shares of the company were up about 6% by late Tuesday morning, to trade at $8.42 apiece.

Acelyrin launched in late 2020 with backing from the venture capital firm Westlake Village Partners. It had raised $550 million before going public in May 2023, which provided another $540 million in funding.

The company’s most advanced drug, called izokibep, is in late-stage testing for various immune disorders that affect the skin, eyes and joints. Acelyrin shares lost more than half of their value last year, after izokibep failed one of those trials in September. 

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