Phase 1 Data Promising for TransCon PTH to Potentially Treat Hypoparathyroidism

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by Diogo Pinto |

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TransCon PTH, an investigational therapy for hypoparathyroidism (HP) being developed by Ascendis Pharma, showed positive results in healthy volunteers, according to initial data from a Phase 1 clinical trial.

The company presented the trial data recently at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco, California.

“We look forward to significant progress during 2018 for our rare disease endocrinology pipeline,” Jan Mikkelsen, Ascendis’ president and CEO, said in a press release. “Our initial phase 1 data for TransCon PTH support our target product profile of TransCon PTH as a true [parathyroid hormone] replacement therapy in HP. We are making tremendous progress as we work to bring new therapies to patients with rare diseases.”

Using TransCon technology, TransCon PTH is designed to restore parathyroid hormone levels, normalizing blood and urinary calcium levels, serum phosphate levels, and bone turnover.

Ascendis’ innovative TransCon technology is intended to extend the duration of a treatment’s action on the body and maximize its overall benefit by releasing the unmodified active parent drug at predefined rates, controlled by the body’s pH and temperature. In this way, the company believes the therapies retain their original mode of action, improving their chances of success.

In the Phase 1 trial, TransCon PTH was proven to last more than 60 hours in the circulation of healthy volunteers, supporting a once-daily administration.

Single ascending doses of TransCon PTH also led to dose-dependent increases of serum calcium, linked to parathyroid hormone levels in the blood, sustained for more than 72 hours with low variability between individuals. TransCon PTH also demonstrated the expected effects on renal calcium levels.

Based on this promising data, the company plans to move the therapy directly into Phase 3 clinical trials in the first quarter of 2019.

Along with TransCon PTH, the company also presented updates on two other pipeline products at the conference: TransCon Growth Hormone, currently in a Phase 3 trial (NCT03305016) for pediatric growth hormone deficiency with results anticipated in the first quarter of 2019; and TransCon CNP, for which a Phase 1 trial in healthy individuals is expected to begin in the second quarter of 2018, as a potential treatment for achondroplasia — a bone growth disorder with no approved treatment yet by the U.S. Food and Drug Administration.