In mid-October, Amolyt Pharma (“Amolyt”) announced that the first participant was dosed in a Phase 1 clinical trial evaluating AZP-3601 as a potential treatment for patients with hypoparathyroidism. As of right now, the clinical trial is solely utilizing healthy participants. However, company leader Dr. Thierry Abribat, PhD, hopes to hold additional clinical trials on patients in the coming years. Altogether, AZP-3601, and Amolyt’s other therapeutic candidates, are designed to treat patients with rare endocrine and metabolic disorders who lack a wealth of therapeutic options.
AZP-3601
Currently, there are few treatments for patients with hypoparathyroidism. These include calcium supplements, alongside vitamin D supplements to aid with absorption. According to Dr. Abribat, around 20% of patients are able to control their condition using calcium supplements. However, this does not maintain stable calcium levels, but rather spikes levels throughout the day. Another potential therapeutic option is Natpara, developed to provide patients with PTH. However, Dr. Abribat explains that Natpara’s short half life still fails to provide normal calcium maintenance.
Amolyt created AZP-3601 to fulfill this unmet need and provide patients with a more effective and efficient option. If all goes well, the therapy, a PTH peptide analog, will help maintain blood calcium levels. Preclinical studies, conducted on animal models, showed AZP-3601 to be effective. In addition to maintaining calcium levels for over 24 hours, it also reduces the risk of osteoporosis and bone brittleness.
Hypoparathyroidism
There are a number of potential causes for hypoparathyroidism, a condition characterized by low parathyroid hormone (PTH) levels. Autoimmune and endocrine conditions, low magnesium in the blood, cancer treatment, genetics, and recent neck surgery all raise the risk of developing hypoparathyroidism. Normally, PTH regulates calcium and phosphorus levels throughout the body. However, PTH deficiency causes heightened phosphorus in the blood and low levels of calcium in the blood and bones. Hypoparathyroidism typically affects middle-aged females who are peri- or post-menopausal. However, the condition can also affect younger age groups.
Symptoms of hypoparathyroidism include:
- Brittle nails
- Patchy hair loss
- Dry skin
- Muscle weakness
- Fatigue
- Extremely painful menstrual cycles
- Depression
- Anxiety
- Fingertips, toes, and lip tingling or burning
- Muscle aches and spasms
Without treatment, dangerous and sometimes life-threatening complications can occur. These include:
- Tetany, or the involuntary contraction of muscles related to low calcium levels
- Kidney damage and disease
- Fainting
- Feeling of “pins and needles”
- Teeth malformations
- Poor intellectual development
- Cataracts
- Calcium deposits in the brain
- Pulmonary arrhythmias
- Heart failure
Learn more about hypoparathyroidism.
