In a recent news release, clinical-stage biotechnology company Amolyt Pharma (“Amolyt”) shared that positive data was available from a proof-of-concept study evaluating AZP-3601 for hypoparathyroidism. Within this study, researchers sought to understand the safety, efficacy, and tolerability of AZP-3601.
Patients within the trial were split into two separate cohorts. The available data comes from Cohort 1; altogether, 12 participants enrolled in this cohort. Patients were given AZP-3601 – with no calcium or vitamin D supplementation – over a 4-week period. Next, patients took AZP-3601 over a 2-month extension. Findings from the study include:
- AZP-3601 treatment helped normalize urinary calcium in those with hypoparathyroidism. Additionally, the treatment showed benefits for bone health. As hypoparathyroidism can cause low calcium levels in the bone, this finding shows promise.
- While treating with AZP-3601, many patients were able to reduce or discontinue calcium and vitamin D supplements without harming their health.
- Altogether, this treatment was found to be relatively safe and well-tolerated.
Amolyt will be sharing additional data from this cohort later this year. Additionally, the company hopes to see promising and positive data from Cohort 2 so that another clinical study can be launched in the future.
AZP-3601: An Overview
Right now, AZP-3601 is not approved for use; it is still considered to be an investigational therapy. This therapeutic peptide works by targeting the parathyroid hormone receptor. In doing so, the therapy works to heighten and stabilize blood calcium levels, which would reduce some of the health impacts of hypoparathyroidism. Since many patients with this condition experience kidney and bone problems, AZP-3601 is also designed to improve the reabsorption of calcium within the kidneys, and to protect overall bone health. Learn more about AZP-3601 here.
What is Hypoparathyroidism?
Hypoparathyroidism is a rare condition in which the parathyroid glands do not produce enough parathyroid hormone (PTH). Normally, PTH plays numerous roles within the body; one such role is balancing calcium and phosphorus. However, a lack of PTH causes imbalances, such as increased blood phosphorus and decreased blood and bone calcium levels. Hypoparathyroidism can result from autoimmune diseases, low blood magnesium levels, cancer treatment around the face and neck, neck surgery, or heredity. Symptoms can include:
- Fatigue and general weakness
- Paresthesias (tingling/burning in the toes, lips, and fingertips)
- Tetany (uncontrollable twitching and muscle spasms around the mouth, hands, arms, and throat)
- Muscle cramps in the legs, feet, face, and abdomen
- Dry, coarse skin
- Patchy hair loss
- Painful menstruation
- Brittle nails
- Hoarseness
- Anxiety and depression
- Tooth malformations
- Impaired kidney function
- Heart arrhythmias
- Cataracts
- Developmental delays
- Seizures
