Shots:

• Thierry spoke about the IND clearance of its lead candidate in hypoparathyroidism and the company’s clinical trial plans for rare endocrine and related diseases
• Thierry also communicated about the closing of funding Series B equity financing round
• The interview gives a deeper understanding of Hypoparathyroidism and its treatment options

Smriti: What did this Investigational New Drug (“IND”) application acceptance mean for the company and the hypoparathyroidism community?

Thierry: The acceptance of our IND is a meaningful milestone as we continue to advance our lead asset, AZP-3601, as a potential treatment for people living with hypoparathyroidism. A great unmet need exists for better therapeutic options since even when taking conventional therapy, many patients continue to experience debilitating symptoms due to poorly regulated serum calcium levels and hypercalciuria, a key risk factor for chronic kidney disease.

Smriti: What are the company’s clinical trial plans to bring this treatment to patients?

Thierry: The U.S. Food and Drug Administration (FDA) cleared our IND application for the ongoing AZP-3601 clinical proof-of-concept trial in patients with hypoparathyroidism that is currently being conducted in several European countries. We plan to announce safety and efficacy data from the trial in the second half of 2022.

Smriti: How does AZP-3601 work? How is it different from other therapies available on the market?

Thierry: AZP-3601 is an investigational therapeutic peptide designed to target a specific conformation of the parathyroid hormone (PTH) receptor. Its differentiated mechanism of action is expected to safely produce sustained and stable levels of calcium in the blood and limit urine calcium excretion.

 In addition to its differentiated receptor profile, AZP-3601 is also designed to have a short half-life to potentially preserve bone integrity, an important benefit, since the majority of patients are peri- and postmenopausal women at increased risk of developing osteoporosis.

Smriti: Tell us more about hypoparathyroidism. Discuss its epidemiology, symptoms, and other treatments.

Thierry: Hypoparathyroidism is a rare endocrine disease that is characterized by a deficiency of human parathyroid hormone (“PTH”). PTH is produced by four small glands that are located on the thyroid. A deficiency or absence of PTH results in decreased calcium and elevated phosphorus levels in the blood and affects the overall calcium metabolism. Loss of PTH most commonly occurs due to damage or removal of the parathyroid glands during thyroid or neck surgery.

Individuals living with hypoparathyroidism may experience cognitive and neuromuscular symptoms, including brain fog or inability to focus, muscle pain, cramping, fatigue, and tingling of the hands or feet.

There are approximately 80,000 and 110,000 people with hypoparathyroidism in the U.S. and E.U., respectively. The sizeable patient population can be separated into segments with different clinical needs. These include patients who experience persistent symptoms, patients with kidney disease, and patients with bone disease.

Even while on conventional treatment, patients within each segment continue to experience a high burden of illness. It is estimated that about 25% of people with hypoparathyroidism have chronic kidney disease. Additionally, more than 50% of people with hypoparathyroidism are peri- and post-menopaused women who are at an increased risk of developing osteoporosis, and close to 20% have been diagnosed with osteopenia or osteoporosis. 

Smriti: Do you expect/ need more funding to advance AZP-3601?

Thierry: In September of 2021, we closed an $80 million Series B equity financing round. The financing was co-led by Sectoral Asset Management and Andera Partners. Those funds are allocated to advancing our pipeline of potential therapeutics for rare endocrine and related diseases, including our lead candidate, AZP-3601, for hypoparathyroidism.

Smriti: What are the company’s other efforts in rare endocrine and related disease candidates?

Thierry: In addition to AZP-3601 for the potential treatment of hypoparathyroidism, our pipeline includes AZP-3813, a product candidate for the potential treatment of acromegaly that is currently undergoing IND enabling studies. We are committed to building an expansive pipeline of therapeutic approaches for the treatment of rare endocrine and related diseases and are actively reviewing additional potential assets.

Source: Canva

About Author:

Thierry Abribat is the Founder and Chief Executive Officer at Amolyt Pharma. He holds a Doctorate of Veterinary Medicine and a Ph.D. from the National Polytechnic Institute of Toulouse.

Related Post: PharmaShots Interview: Amolyt Pharma’s Thierry Abribat Shares Insight on the Data of AZP-3601 for Hypoparathyroidism