Amorphical, developer of nano-amorphous mineral-based pharmacologic agents, has announced positive interim results from its ongoing open-label clinical trial evaluating a novel therapy for moderate-to-severe Crohn’s disease. The data suggests that, when added to best available therapy, the investigational treatment supports meaningful clinical improvement and remission, with no safety concerns observed to date.

The therapy, a proprietary nano-amorphous calcium carbonate (ACC) agent, is designed to target the acidic microenvironment in the gut, offering a non-immunosuppressive approach to tissue repair and clinical improvement. Crohn’s disease remains a chronic inflammatory bowel condition with significant unmet needs, particularly for patients who do not respond to or cannot tolerate existing therapies.

Interim data from 17 patients with open-label results shows that 12 (71%) experienced a clinical improvement of more than 100 points on the Crohn’s Disease Activity Index (CDAI) from baseline, while 8 (47%) achieved clinical remission (CDAI <150). Among 5 patients with over 10 months of follow-up, 4 (80%) showed sustained remission. The mean CDAI score improved from 293±51 at baseline to 176±94 during the open-label phase (p<0.001).

Dr Arik Segal, lead investigator at Soroka University Medical Center, said the findings support the hypothesis that normalising the acidic microenvironment with amorphous calcium carbonate can be a valuable treatment strategy. “We are very encouraged by these interim results, which show a clear signal of clinical improvement and remission,” he added, noting anticipation for the unblinding of the double-blind phase.

Eden Ben, chief executive officer of Amorphical, described the data as a significant milestone. He said it strengthens confidence in the broad potential of the company’s nano-amorphous mineral technology to address conditions where underlying pH plays a key role, and underscores the company’s commitment to advancing this program and others, including pancreatic cancer, hypoparathyroidism, and osteoporotic fractures.

The study remains blinded during the double-blind phase, with additional insights expected in November as unblinding and long-term follow-up progress. With over a decade of real-world exposure to its nano-amorphous mineral therapeutics through early access and compassionate use programs, Amorphical enters clinical development with substantial safety and tolerability data and a regulatory pathway guided by more than 190 worldwide patents.