When it comes to drug development and medical research, particularly within the rare disease sphere, it is incredibly important for patients to feel involved in the process. After all, patients want to feel heard and understood, and to know that the company creating a therapy has their best interest in mind. When I spoke with Dr. Mark Sumeray, MD, MS, FRCS about his work at Amolyt Pharma, he explained the company’s focus on patient-centricity and how important it is to facilitate those connections.

In October 2022, Amolyt Pharma shared positive data from a Phase 2a study on AZP-3601 for patients with hypoparathyroidism. The study sought to understand how AZP-3601 could not only reduce symptoms and negative health effects related to hypoparathyroidism, but how it could address the needs of patients living with it. For example, AZP-3601 helped to reduce urinary calcium excretion and control blood calcium levels which (in turn) reduced the need for calcium and vitamin D supplementation.

In our interview for Patient Worthy, Dr. Sumeray and I spoke about what hypoparathyroidism is, the process of developing and testing AZP-3601, new findings from the study, the challenges and rewards of drug development in rare disease, and where the company hopes to expand in the future.

About Mark Sumeray, MD, MS, FRCS

Dr. Mark Sumeray is the Chief Medical Officer (CMO) of Amolyt Pharma (“Amolyt”). He has over 23 years in the biotech and pharmaceutical industries with a focus on drug development, business development, and commercialization.

Dr. Sumeray received his BSc (Honors) in physiology from University College London (UCL), as well as his MS BS in Medicine. He also earned his FRCS in Surgery from the Royal College of Surgeons.

“As you might expect, I have a background as a physician. During my clinical practice, I focused on cardiovascular surgery. At that time in my career, that was what I thought I wanted to do. However, I later became interested in drug development, biotech, and regulatory affairs. Since I became more involved in the industry, I’ve spent about half of my time working in drug development for patients with rare or orphan diseases for which there are no true treatment options.” – Dr. Sumeray

Prior to joining Amolyt, Dr. Sumeray previously worked at Amryt Pharmaceuticals, Aegerion Pharmaceuticals, and Bristol-Myers Squibb, among others.

Hypoparathyroidism: An Overview

Hypoparathyroidism is a rare condition in which the parathyroid glands produce abnormally low levels of parathyroid hormone (PTH). It affects an estimated 80,000 people within the United States and 120,000 in Europe. Dr. Sumeray explains:

“Hypoparathyroidism is usually caused by removal of the thyroid gland for thyroid cancer or other reasons. During the removal, the parathyroid glands are often removed or damaged at the same time, though not deliberately. When this happens, patients are unable to make parathyroid hormone, which is important for a number of functions such as controlling calcium levels in the blood by acting on the skeleton to influence the uptake and release of calcium. It also acts on the kidney where calcium is filtered from the blood into the urine. Normally, it’s reabsorbed. But with hypoparathyroidism, patients don’t have the normal ability to reabsorb calcium. This can lead to progressive kidney failure and bone issues.”

PTH plays a role in bone metabolism. Our bone is in a constant process of breaking down old bone and replacing it with new bone to ensure strength and function. Without enough PTH, some individuals may experience a number of negative health effects.

Outside of surgical thyroid removal, hypoparathyroidism may also result from low levels of blood magnesium, extensive cancer treatment around the face and neck, inheritance (in the case of the hereditary form), or certain autoimmune conditions.

Symptoms and Treatments

Symptoms associated with hypoparathyroidism can (but do not always) include:

• Paresthesias (tingling and burning in the fingertips, lips, and toes)
• Muscle aches and cramps in the legs, feet, face, and abdomen
• Painful muscle spasms
• Dry, coarse skin
• Brittle nails
• Patchy hair loss
• Painful menstruation
• Fatigue
• Seizures
• Teeth malformations
• Fainting
• Arrhythmias
• Impaired kidney function

Please note that this is not an exhaustive list of symptoms.

Currently, there are certain treatment options available for hypoparathyroidism, including vitamin D and calcium supplements, as well as Natpara. However, these treatment options have room for improvement. According to Dr. Sumeray:

One of the current standards-of-care is to give patients calcium supplements to replace the calcium they’re losing through their urine, along with vitamin D to increase calcium absorption from the diet. While this can increase calcium levels, it doesn’t help the problem of calcium loss through urine. You’re putting calcium in the body but losing it too easily.

Over the past few years, the medical field has been working to improve the standard-of-care by providing the missing hormone to patients. While this sounds obvious, it’s not easy. As Dr. Sumeray explains, PTH is not released into the bloodstream in a simple way.

However, Dr. Sumeray and the Amolyt team are working to address this need through the development of a novel investigational peptide hormone called AZP-3601.

Join us in Part 2 as we discuss more about AZP-3601, Phase 2a study findings, the challenges and rewards of rare disease drug development, and Amolyt’s future research plans.