Amorphical announces positive interim results from ongoing Crohn’s trial

Amorphical, developer of nano-amorphous mineral-based pharmacologic agents for health conditions, has announced positive interim results from its ongoing open-label clinical trial evaluating its investigational therapy for the treatment of moderate-to-severe Crohn’s disease.

The data suggests that the investigational therapy, when added to best available therapy, supports meaningful clinical improvement and remission.

The drug has been well tolerated, with no safety concerns observed to date.

Crohn’s disease is a chronic inflammatory bowel disease with significant unmet needs, particularly for patients who do not respond to or cannot tolerate existing therapies.

The investigational therapy, a proprietary nano amorphous calcium carbonate (ACC) agent, is designed to target the acidic microenvironment in the gut, offering a unique, non-immunosuppressive approach to supporting tissue repair and clinical improvement.

Dr Arik Segal is a lead investigator from the Department of Gastroenterology at Soroka University Medical Center.

The researcher said: “We are very encouraged by these interim results, which show a clear signal of clinical improvement and remission with this investigational therapy.

“The data supports our hypothesis that normalising the acidic microenvironment with amorphous calcium carbonate can be a valuable, non-immunosuppressive treatment strategy.

“We look forward to the continued progress of this trial and the unblinding of the double-blind phase.”

This trial is being conducted under the supervision of the local ethical committee in Israel.

Amorphical plans to leverage these results to inform a future FDA regulatory strategy.

The ongoing study has enrolled 22 patients, 17 of whom have open-label data available.

At baseline, 70 per cent of patients were already on at least one immunosuppressive or advanced therapy.

The interim data reflects varying follow-up durations:

• 12 out of 17 (71 per cent) patients experienced a clinical improvement of >100 CDAI (Crohn’s Disease Activity Index) points from baseline.
• 8 out of 17 (47 per cent) patients achieved clinical remission (CDAI <150).
• 4 out of 5 patients (80 per cent) with more than 10 months of follow-up in the open-label phase showed sustained remission.
• The mean CDAI score at baseline was 293±51, which improved to 176±94 during the open-label phase (p<0.001).

Eden Ben is chief executive officer of Amorphical.

Ben said: “This positive interim data in Crohn’s disease represents a significant milestone for our company and for the patients seeking new treatment options.

“The results strengthen our confidence in the broad potential of our nano-amorphous mineral technology to address conditions where underlying pH plays a key role.

“We are deeply committed to advancing this program and others, and bringing a new class of therapy to the market.”

The study remains blinded during the double-blind phase.

Additional insights are expected as the data becomes unblinded in November and long-term follow-up progresses.

With more than a decade of real-world exposure to its nano-amorphous mineral–based therapeutics, including use through early access and compassionate use programs in other conditions, Amorphical enters clinical development with valuable safety and tolerability insights to guide its regulatory pathway.

In addition to Crohn’s disease, the company is advancing clinical programs in pancreatic cancer, hypoparathyroidism, and osteoporotic fractures. Its platform is protected by more than 190 patents worldwide.