Amorphical has completed patient enrolment in a Phase 2 trial evaluating its investigational AMOR-1 therapy in adults with hypoparathyroidism, with results expected in the fourth quarter of 2026.

The multinational, multicentre, double-blind, randomised study is evaluating whether AMOR-1 can help patients maintain safe serum calcium levels while reducing their intake of elemental calcium compared with conventional calcium supplementation.

The trial has enrolled 50 adults with hypocalcaemia associated with hypoparathyroidism, a rare endocrine disorder caused by insufficient production of parathyroid hormone.

Amorphical is developing AMOR-1 using its nano-amorphous calcium carbonate (ACC) technology, which the company says is designed to improve calcium absorption and bioavailability compared with conventional crystalline calcium formulations.

The company expects final primary completion and a data readout from the Phase 2 study in Q4 2026.

Phase 2 trial tests 50% calcium dose reduction

The Phase 2 study, identified as NCT06547151, has randomised participants in a 2:1 ratio to receive either AMOR-1 or a control consisting of conventional crystalline calcium carbonate supplements.

During an initial two-to-four-week replacement phase, participants gradually replace their existing calcium supplements with the assigned treatment.

Patients receiving AMOR-1 have their total elemental calcium intake reduced by 50% compared with their baseline intake, while participants in the control group continue receiving 100% of their initial elemental calcium intake.

Participants then enter a 10-to-12-week maintenance period during which they are monitored for safety and efficacy measures, including blood and urine calcium levels.

The study is designed to assess whether AMOR-1 can maintain appropriate serum calcium levels despite the reduction in elemental calcium intake.

The potential benefit of the approach is based on Amorphical’s nano-amorphous calcium carbonate technology, which the company says provides greater calcium bioavailability than conventional crystalline calcium.

Eden Ben, chief executive officer of Amorphical, said: “Achieving full enrollment in our Phase 2 FDA trial is a major milestone for Amorphical and brings us one step closer to redefining the standard of care for hypoparathyroidism.”

Ben added that the company expects to analyse the unblinded data later this year as it continues to progress the programme.

AMOR-1 targets calcium management in hypoparathyroidism

Hypoparathyroidism occurs when the body produces insufficient parathyroid hormone, resulting in low blood calcium levels that can cause a range of symptoms and complications.

Treatment commonly involves calcium supplementation and active vitamin D, while newer approaches include hormone replacement therapies.

Amorphical is developing AMOR-1 as a potential treatment that could allow patients to maintain calcium levels while taking less elemental calcium.

The company’s approach is based on its nano-amorphous mineral technology, which is intended to improve the absorption of calcium compared with conventional crystalline formulations.

Prof. Ilan Shimon, director of the Institute of Endocrinology and Metabolism at Beilinson Hospital, Rabin Medical Center, and lead investigator of the Phase 2 study, said: “The ability of AMOR-1’s nano-amorphous platform to deliver targeted, highly bioavailable calcium means we may finally be able to maintain desired serum calcium levels with half the amount of elemental calcium compared to conventional therapies.”

The statement reflects the hypothesis being tested in the Phase 2 study, rather than a clinical outcome demonstrated by the trial.

The company said patients with hypoparathyroidism can face challenges in maintaining appropriate calcium levels, with treatment requiring careful management of calcium supplementation and vitamin D.

Amorphical’s clinical development programme has been supported by more than a decade of real-world exposure through early access and compassionate use programmes, according to the company.

The biotechnology company is also developing programmes involving its technology in pancreatic cancer, Crohn’s disease and osteoporotic fractures.

The next major milestone for AMOR-1 will be the expected Phase 2 data readout in Q4 2026. Results from the trial will help determine whether the nano-amorphous calcium formulation can maintain calcium levels while reducing patients’ overall elemental calcium intake.