Globe Newswire recently published an article announcing positive results from Amolyt Pharma’s Phase IIa proof of concept clinical trial of AZP-3601. Amolyt Pharma, Cambridge, Massachusetts, and Lyon, France, is a global company focusing on the development of therapeutic peptides for patients with rare endocrine and other related diseases.
The company is developing AZP-3601 to treat hypoparathyroidism which is defined as a deficiency of the parathyroid hormone. The result is a decrease in calcium and elevated phosphorus levels in the blood.
Approximately eighty percent of the 80,000 people in the United States with the disorder are women. In Europe, the number of women with hypoparathyroidism is estimated at 110,000.
The disease impacts various organ systems and tissues, especially bones and kidneys. It is estimated that about twenty-six percent of patients have kidney disease or kidney failure. Therefore, a key goal would be a reduction of urinary calcium excretion.
Amolyt’s announcement heralded positive results from a second patient group in a Phase IIa clinical proof of concept trial. Proof of concept is a milestone in drug development and a crucial step toward marketing a human therapeutic.
AZP-3601 is a PTH1 receptor agonist that is expressed in bone and kidney. It maintains calcium ion homeostasis which regulates the flow of calcium into and out of bones.
Data from the Phase IIa study indicated that AZP-3601 was well tolerated. There was no indication of severe adverse events. Any moderate or mild adverse events were consistent with previous studies. The starting dose of (10 μg) was increased to (20 μg) within a few weeks of the onset of the trial.
Within three months, ninety-three percent of patients were able to discontinue their standard therapy of calcium and vitamin D supplementation. Serum calcium stayed within range and bone mineral density remained stable.
Urinary calcium excretion was normalized with a resumption of bone turnover (replacement). Approximately ten percent of the skeleton is replaced each year.
About the Trial
During a four-week period, sixteen patients diagnosed with hypoparathyroidism were administered daily injections of AZP-3601. A treatment extension of eight weeks followed during which vitamin D and calcium supplementation were gradually removed.
Furthermore, individual doses of AZP-3601 up to 80 μg daily could be administered within the extension period. The increase in dose allowance occurred in order to support an evaluation for a Phase 3 trial the following year.
Dr. Thierry Abribat CEO and founder of Amolyt Pharma commented on the encouraging data for AZP-3601. Dr. Thierry states that the normalization of urinary calcium secretion plus resumption of bone regrowth gives rise to AZP’s meeting its treatment goals.
