FDA Grants Fast Track Designation to Amolyt Pharma’s Eneboparatide to Treat Hypoparathyroidism

Image Credit: Adobe Stock Images/Lila Patel

Amolyt Pharma announced that the FDA has granted fast track designation to eneboparatide, a medication designed to treat hypoparathyroidism. The treatment, which is currently under evaluation in the Phase III Calypso clinical trial, has shown potential in earlier studies through improving key calcium levels and bone health. Results from the Phase III trial are expected at a date to be determined next year.

Calypso is a multicenter, randomized, placebo-controlled, double-blind study designed to evaluate the safety and efficacy of eneboparatide in patients with hypoparathyroidism. Investigators enrolled 165 patients, who were randomly assigned in a 2:1 ratio to receive eneboparatide or placebo. The primary endpoint is achievement of albumin-adjusted serum calcium within the normal range and independence from standard of care after 24 weeks of treatment, with secondary endpoints that include normalization of 24-hour urinary calcium in patients with hypercalciuria at baseline and assessment of patient-reported outcomes that reflect symptoms associated with physical and cognitive function, and impact on quality of life.¹

“We believe FDA’s granting of fast-track designation to eneboparatide reflects the agency’s recognition of the significant unmet needs that persist among patients suffering from hypoparathyroidism,” stated Thierry Abribat, PhD, founder, CEO, Amolyt Pharma, in a press release. “We look forward to maintaining a constructive dialog with the agency as we work to bring new hope to patients suffering from this rare but challenging endocrine disorder as efficiently as possible.”

Amolyt began the Calypso trial last May, with participants from the United States, Canada, Europe, and the United Kingdom planning to participate across 50 sites. A prior Phase II trial allowed for discontinuation of oral supplements in 93% of patients through the use of eneboparatide. Back in March, Amolyt was acquired by AstraZeneca for a total of $1.05 billion, including the rights to eneboparatide.^2-4

“We enthusiastically welcome the proposed acquisition of Amolyt by AstraZeneca, an organization that shares our dedication to delivering life-changing treatments to people living with rare diseases,” said Abribat, in a press release. “This agreement offers the opportunity to meaningfully advance our pipeline therapies. Strong Phase II data suggest eneboparatide has the potential to improve outcomes for patients and to shift the treatment paradigm for hypoparathyroidism, and we look forward to seeing the continued advancement of the Phase III trial.”

According to Medical News Today, hypoparathyroidism occurs when an individual’s parathyroid glands do not produce enough parathyroid hormone, resulting from injuries or surgical removal of parathyroid glands. In most cases, it will result in low blood calcium and high blood phosphate. It can also cause bone damage, kidney stones, and muscle spasms. Early symptoms include but aren’t limited to:

• Anxiety
• Muscle Pain
• Dry Skin
• Mood Changes
• Depression
• Yeast Infections⁵

“The current standard of care treatment, oral calcium and vitamin D supplementation seldom controls the life altering symptoms and complications of hypoparathyroidism, with many patients at risk of declining kidney function and diminished bone quality,” stated Mark Sumeray, MD, chief medical officer, Amolyt Pharma, in the press release. “In studies to date, eneboparatide has been shown to normalize mean serum calcium and mean urinary calcium excretion while restoring balanced bone turnover. Building upon findings from our successful Phase 2 clinical trial, we are working diligently to execute our ongoing Calypso Phase III study and look forward to topline data in 2025.”

References

1. Amolyt Pharma Granted FDA Fast Track Designation for Eneboparatide for the Treatment of Hypoparathyroidism. GlobeNewswire. May 2, 2024. Accessed May 2, 2024. https://www.globenewswire.com/news-release/2024/05/02/2874145/0/en/Amolyt-Pharma-Granted-FDA-Fast-Track-Designation-for-Eneboparatide-for-the-Treatment-of-Hypoparathyroidism.html

2. Amolyt Pharma Announces Phase 3 Clinical Trial of Eneboparatide for the Treatment of Hypoparathyroidism following Positive End of Phase 2 Meeting with FDA. Amolyt. May 2, 2023. Accessed May 2, 2024. https://amolytpharma.com/2023/05/02/amolyt-pharma-announces-phase-3-clinical-trial-of-eneboparatide-for-the-treatment-of-hypoparathyroidism-following-positive-end-of-phase-2-meeting-with-fda/

3. AstraZeneca Acquires Amolyt Pharma with the Aim of Developing Rare Endocrine Disease Treatments. PharmExec. March 14, 2024. Accessed May 2, 2024. https://www.pharmexec.com/view/astrazeneca-acquires-amolyt-pharma-with-the-aim-of-developing-rare-endocrine-disease-treatments

4. AstraZeneca to acquire Amolyt Pharma, expanding late-stage rare disease pipeline. AstraZeneca. March 14, 2024. Accessed May 2, 2024. https://www.astrazeneca.com/media-centre/press-releases/2024/astrazeneca-to-acquire-amolyt.html

5. What to know about hypoparathyroidism. MedicalNewsToday. Accessed May 2, 2024. https://www.medicalnewstoday.com/articles/hypoparathyroidism