Three months after reporting a win in a phase 3 trial of its kidney transplant drug imlifidase—which is likely to pave the way for a first-time approval in the U.S.—Hansa Biopharma has stumbled in a late-stage study of the treatment in a different indication.
The Swedish drugmaker has come up short in its effort to push imlifidase into the treatment of the autoimmune condition anti-glomerular basement membrane (anti-GBM) disease. In the company's GOOD-IDES-02 trial, approximately 60% of patients who received imlifidase followed by standard care treatment did not require dialysis at 6 months.
While that figure represented a “substantial improvement and clinical benefit" compared to control group results in previous anti-GBM trials, it did not show an improvement against the control arm in GOOD-IDES-02, Hansa explained in a Dec. 16 press release. Patients who received standard care alone in the study had a “similar response" to those in the treatment arm, Hansa wrote.
In GOOD-IDES-02, standard care consisted of intense plasma exchange (PLEX) combined with cyclophosphamide (CYC) and glucocorticoids.
“We are disappointed not to be able to provide a new treatment option for this patient group, who to date have experienced poor outcomes,” Hansa CEO Renée Aguiar-Lucander said in a release. “Despite the deep and rapid reduction of anti-GBM antibodies following imlifidase treatment, it did not result in a statistically significant outcome in this setting."
Nevertheless, Hansa is pleased with the 60% of imlifidase patients who no longer required dialysis, with Aguiar-Lucander calling those results "almost three-fold that of typically reported outcomes for this disease."
In historical control groups in the indication, only 20% to 25% of patients were typically dialysis-free at six months. The traditionally poor results helped inform Hansa's decision to undertake the study, the company said Tuesday.
“This suggests standard of care has improved significantly since these earlier studies,” investment bank Redeye - Nordic Growth said in a research note, adding that the outcome was a “moderate setback” for Hansa. Redeye had previously estimated peak sales for imflifidase in anti-GBM at $130 million versus $450 million in the drug's kidney transplant niche.
Anti-GBM disease, which is also referred to as Goodpasture syndrome, is an autoimmune disorder that affects around 1.6 people per one million annually, with a majority of patients eventually losing kidney function. The condition causes antibodies to attack the body's own glomerular basement membrane (GBM) in the kidneys and the alveolar basement membrane (ABM) in the lungs, causing inflammation, bleeding, and potential kidney or respiratory failure.
In 2020, the European Union granted approval to imlifidase for desensitization in transplant patients who have difficulty finding a kidney match because their bodies produce high levels of antibodies that are meant to attack foreign tissues. The commercial name for the treatment is Idefirix.
The medication is also approved in Australia, the United Kingdom and Switzerland. Hansa still plans to file an application for FDA approval in its established kidney transplant indication by the end of this year, Aguiar-Lucander said in Tuesday's announcement.
The anti-GBM stumble should not hinder Hansa’s efforts to gain U.S. approval in the kidney setting, according to analysts at Jefferies.
“It is unclear as to why aggressive [Standard of Care] appears to have performed unusually well,” Jefferies wrote. “Adding imlifidase may have overlapping mechanisms with PLEX," the Jefferies team speculated, adding that "depending on sequencing and timing, the incremental benefit could be muted when SoC is delivered very early and aggressively.”