Will Ascendis End The Year On A High Note?

Parathyroid hormone plays a critical role in controlling calcium, phosphate and calcitriol (active vitamin D) levels in the blood and bone. Hypoparathyroidism is a rare endocrine disorder characterized by deficient or absent parathyroid hormone and it affects over 200,000 people worldwide. People living with hypoparathyroidism experience a diverse range of physical, mental, cognitive and emotional symptoms.

Ascendis Pharma A/S (ASND) has developed TransCon PTH to treat adult patients with hypoparathyroidism.

TransCon PTH is a once-daily hormone replacement therapy designed to restore physiologic levels of parathyroid hormone 24 hours a day.

In a global phase III trial, dubbed PaTHway, on which the company’s New Drug Application for TransCon PTH was based, the primary and all key secondary endpoints were met.

TransCon PTH is under FDA review in the U.S., with a decision expected on April 30, 2023.

Ascendis also plans to seek approval for TransCon PTH in adult patients with hypoparathyroidism in Europe during this quarter, based on the results of the PaTHway trial.

A phase III trial of TransCon PTH in Japan, dubbed Pathway Japan, is underway, with results expected this quarter. A phase II trial of TransCon PTH in hypoparathyroidism is ongoing in Greater China through VISEN Pharmaceuticals.

TransCon CNP for pediatric achondroplasia; TransCon hGH for pediatric growth hormone deficiency and adult growth hormone deficiency; TransCon TLR7/8 agonist, designed to promote potent anti-tumoral responses while reducing systemic drug exposure and related adverse events; and TransCon IL-2 ß/? for treatment of locally advanced or metastatic solid tumors are the other investigational therapies in the pipeline.

— TransCon CNP is under a phase II clinical trial in children ages 2-10 years with achondroplasia, dubbed ACcomplisH. Topline and initial open-label extension data from this study are expected during this quarter.

Achondroplasia is a skeletal disease and the most common form of dwarfism. Biomarin Pharmaceutical Inc.’s (BMRN) Voxzogo is the first approved drug for children ages 5 and older with achondroplasia. It secured FDA approval last November.

— TransCon hGH is under a global phase III trial in adult growth hormone deficiency, dubbed foresiGHt. Enrollment in this study is expected to be completed this quarter.

TransCon hGH received approval in the U.S. last August for the replacement of growth hormone in children 1 year old or older with growth hormone deficiency and is marketed under the brand name Skytrofa. In Europe, this medicine, received approval in January of this year and is intended for children and adolescents from 3 up to 18 years of age.

The U.S. revenue of Skytrofa more than doubled quarter-to-quarter to €12.3 million in the third quarter of 2022. The drug is expected to be launched in Germany in mid-2023.

— TransCon TLR7/8 Agonist is under a phase I/II trial in participants with locally advanced or metastatic solid tumor malignancies is ongoing. This compound is being tested alone or in combination with Merck’s Keytruda in the trial.

— TransCon IL-2 ß/? as monotherapy in patients with locally advanced or metastatic solid tumors is under a phase I/II dose escalation and dose expansion trial. Topline data from monotherapy dose escalation part of the trial is expected during this quarter.

Cash position:

As of September 30, 2022, Ascendis Pharma’s cash, cash equivalents, and marketable securities totaled €935.1 million.

Ascendis Pharma’s American Depositary Shares began trading on The NASDAQ Global Select Market on January 28, 2015, under the ticker symbol “ASND”, priced at $18.00 per ADS.

ASND has traded in a range of $61.58 to $164.66 in the last 1 year. The stock closed Wednesday’s trading at $113.94, down 2.37%.

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