Amolyt Pharma Gets FDA Clearance For Hypoparathyroidism Study

Amolyt Pharma is a drug development company based in France. The company, which focuses on creating therapeutic peptides for rare endocrine and related diseases, announced big news this month (March 2022). The US Food and Drug Administration (FDA) has cleared Amolyt Pharma’s Investigational New Drug (ING) application which will result in ongoing AZP-3601 clinical proof-of-concept trials in patients with hypoparathyroidism. AZP-3601 is the identification of the new drug under the trial. The trials are underway in a few European countries.

What They Had To Say

Founder and chief executive officer of Amolyt Pharma, Thierry Abribat, Ph. D., says, “We are pleased that the FDA has cleared our IND, allowing us to expand our AZP-3601 clinical program to the US, whereas many as 80-thousand people are diagnosed with hypoparathyroidism.” He went on to add, “Despite current treatment options, many patients continue to experience debilitating symptoms due to poorly regulated serum calcium levels and hypercalciuria, a key risk factor for chronic kidney disease, and they are in need of better therapeutic approaches.”

Senior vice president of clinical development and regulatory affairs of Amolyt Pharma, Soraya Allas, M. D., Ph. D., adds, “Based on data observed to date from our Phase 1 clinical trial in healthy volunteers, we believe AZP-3601 may provide significant clinical benefits, in particular, sustained 24-hour serum calcium and urinary calcium normalization.” Other data shows promise as well, “Bone biomarker data from this trial also leads us to believe that AZP-3601’s targeted mechanism of action may preserve bone integrity.” Allas says these are important benefits as 26-percent of patients with hypoparathyroidism have chronic kidney disease. Plus, another 17-percent have already developed osteopenia or osteoporosis.

What Got Them Here

It was back in October 2021 at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting when Amolyt Pharma became more than just a blip on the radar. It was at that meeting that the company revealed its positive Phase 1 data in healthy volunteers. That information provided a strong scientific rationale in support of the ongoing development of the drug.

The investigative therapeutic peptide identified as AZP-3601 is designed specifically to target a unique conformation of the parathyroid hormone (PTH) receptor. When it does this, it safely produces sustained and stable levels of calcium in the blood. While this happens, the symptoms of hypoparathyroidism are manageable. It also limits urine calcium excretion by restoring calcium reabsorption by the kidney. The goal of this is to prevent chronic kidney disease. AZP-3601 is also designed to possibly preserve bone integrity through a short half-life. Considering that most patients are either peri or postmenopausal women with a higher risk of developing osteoporosis, this is an important benefit.

More On Hypoparathyroidism

Hypoparathyroidism is a deficiency of parathyroid hormone (PTH) that leads to a decrease in calcium and an increase of phosphorus levels in the blood. Of the 80,000 people in the US and 110,000 people in Europe with hypoparathyroidism, 80-percent are women. Although there are treatments available to combat the condition, patients continue to suffer from persistent, life-changing symptoms. They also tend to develop complications and comorbidities that impact their quality of life. Some patients end up requiring targeted clinical requirements.

Hypoparathyroidism By The Numbers

Many tissues and organ systems are impacted by hypoparathyroidism with a particular focus on the kidneys and bones. Of the patients with hypoparathyroidism, 17-percent have either osteopenia or osteoporosis. A total of 53-percent of patients with hypoparathyroidism are either peri or postmenopausal women. Each in this category is at a higher risk of developing osteoporosis. Plus, 26-percent of patients with hypoparathyroidism have either kidney disease or failure. This fact alone emphasizes the need to reduce urinary calcium excretion as a primary goal of treatment.

Amolyt Pharma is a clinical-stage biotechnology company. The company is leveraging the established knowledge of its team in the development of new products. Amolyt Pharma has been working on therapeutic peptides that will provide patients suffering from rare endocrine and related diseases with life-altering treatments. The most promising of the products under development by Amolyt Pharma is AZP-3601. AZP-3601 is a long-acting parathyroid hormone (PTH) analog that could become a potential treatment for hypoparathyroidism.

Other products under development at Amolyt Pharma include AZP-3813. This is a peptide growth hormone receptor antagonist. It is being developed as a possible treatment of acromegaly. Acromegaly is a condition where the body creates an abundance of growth hormones which result in body tissues and bones growing faster than normal. In addition, the company is working on AZP-3404, which is currently undergoing indication selection work. Amolyt Pharma states they intend to expand and develop its portfolio through established partnerships. The global network the company has access to focuses on the field of endocrinology and has the support of investors from an international syndicate.

Final Thoughts

The numbers tell the tale. With the majority (about 80%) of the people in the United States and European Union suffering from hypoparathyroidism being women, and most of them are at a stage in their lives where they are at higher risk of developing bone diseases, it only makes sense that a drug would be developed to address these issues. Although there are treatments available, many patients find them to be ineffective in the long term. The USFDA must see potential in AZP-3601 in remedying the condition and that is why they have approved the application from Amolyt Pharma to move AZP-3601 to the next level – clinical trials.

The Investigative New Drug (ING) shows promise based on data the company shared during the 2021 Annual Meeting of the American Society for Bone and Mineral Research. Although AZP-3601 won’t appear on prescription orders anytime soon, the ball is rolling to get it to that stage so those patients who can benefit most from this new drug will finally have access to a treatment that will bring them some long-term relief. With the work completed to date by Amolyt Pharma and the blessing of the FDA, this could be a slam dunk for the development company from France.