Zilbrysq autoinjector found safe, effective in trials for use in gMG

An investigational autoinjector device for administering Zilbrysq (zilucoplan), an approved therapy for generalized myasthenia gravis (gMG), is a safe and effective option for individuals now using the currently available prefilled syringe, according to new data from two clinical trials.

Users were “highly satisfied” with the Zilbrysq autoinjector and generally preferred it over the prefilled syringe, the researchers noted, calling the device’s use “an effective alternative … for patients with gMG.”

These findings were presented at this year’s annual meeting of the American Academy of Neurology (AAN), in a poster titled “Bioequivalence and effectiveness of auto-injector-delivered, self-administered zilucoplan compared with pre-filled syringe.” The work was funded by UCB, the company that markets Zilbrysq.

“At UCB, our approach to rare disease goes beyond developing effective medicines; it’s about improving overall experience and outcome for patients,” Kimberly Moran, PhD, UCB’s senior vice president and head of U.S. rare disease, said in a company press release detailing the trial findings.

Omar Sinno, MD, UCB’s U.S. medical strategy lead for rare disease, added: “While symptom control is of course essential, successfully treating gMG should go beyond symptom management and consider tailored therapies that align with a patient’s needs, lifestyle and quality-of-life goals.”

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gMG is an autoimmune disorder in which self-targeting antibodies impair nerve-muscle communication, leading to symptoms such as fatigue and muscle weakness.

2 trials tested autoinjector device for safey, effectiveness

Zilbrysq is approved in the U.S. and elsewhere as a treatment for people with gMG who are positive for the most common type of MG-causing antibody targeting the acetylcholine receptor (AChR) protein. The therapy works by blocking activation of a group of immune proteins that play a key role in the antibody-mediated attack.

It’s administered once daily via injection under the skin, and for now is available in prefilled syringes. UCB has been developing a button-free autoinjector for Zilbrysq, which allows the therapy to be given via a simple movement known as push-on-skin.

UCB conducted two clinical trials to test the autoinjector. One study, dubbed DV0012 (NCT06511076), enrolled a small number of healthy adults who received one daily Zilbrysq injection using either the prefilled syringe or the autoinjector, and another injection the next day using the opposite method.

The goal was to see if both devices delivered equivalent amounts of the therapy. The results were positive, with pharmacological data showing no meaningful differences between the two.

The second trial, DV0013 (NCT06471361), enrolled 31 people with AChR-related gMG who had previously been taking Zilbrysq using the prefilled syringe. These participants switched to the autoinjector for two weeks, with the main goal of demonstrating that patients could properly self-administer the therapy as assessed by their doctor.

These results showed that virtually all Zilbrysq doses were administered correctly with the autoinjector. Importantly, the researchers noted, patients generally reported better satisfaction with the new device.

[Zilbrysq] had a high complete dose delivery rate with the auto-injector, with patients highly satisfied and most preferring the auto-injector over the [prefilled syringe].

Data from both trials showed no unexpected safety-related issues.

Zilbrysq “had a high complete dose delivery rate with the auto-injector, with patients highly satisfied and most preferring the auto-injector over the [prefilled syringe]; it was also well tolerated,” the researchers wrote in the poster.

“These data suggest that [Zilbrysq] auto-injector is an effective alternative to [prefilled syringe] for patients with gMG,” the team noted.

Zilbrysq’s approvals were based mainly on data from the Phase 3 RAISE trial (NCT04115293), which demonstrated that the therapy was better than a placebo at easing symptoms in AChR-related gMG patients.

Participants in RAISE and a previous Zilbrysq Phase 2 study (NCT03315130) could then enter an ongoing extension study, called RAISE-XT (NCT04225871), which is tracking long-term outcomes with Zilbrysq.

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Zilbrysq also shown to help reduce symptoms long term

In a separate poster titled “Sustained minimal symptom expression in generalized myasthenia gravis: A 120-week post hoc analysis of RAISE-XT,” researchers presented a new analysis of pooled data from the original trials and subsequent RAISE-XT. This analysis specifically looked at the number of patients who attained minimum symptom expression (MSE), or little to no impact of the disease in their daily life.

The results showed that, over the course of more than two years of follow-up, 63% of Zilbrysq-treated patients achieved MSE. This was achieved a median of about 8.5 months after starting treatment, with some individuals achieving MSE in the first week.

Also, MSE responses were maintained for a median of 80.8% of the follow-up time, the data showed.

“Patients receiving [Zilbrysq] experienced sustained MSE over long-term treatment,” the researchers wrote.

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Drug developer working outside the lab to help patients

In addition to developing treatments, UCB is also a key partner on a recently launched initiative from the Myasthenia Gravis Foundation of America (MGFA) called MGFA Food Support Program. It aims to provide eligible MG patients easy access to nutritious, already prepared meals delivered right to their doors.

“Members of the MG community tell us that mealtime is stressful because the disease impacts a patient’s ability to prepare nutritious meals for their families or consume the right combination of nutritional foods to help maintain a high quality of life,” Samantha Masterson, MGFA’s president and CEO, said in a foundation press release announcing the initiative.

Aprill Lane, UCB’s US advocacy lead, rare disease, said the partnership works hand-in-hand with the work the company is doing in its labs.

“If we want to support improving health outcomes, we have to address the barriers that exist outside of the healthcare setting,” Lane said. “Our collaboration with MGFA is an example of how we can lead and partner differently by co-creating programs that support the whole person not just the diagnosis.”