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Ascendis Progresses With Hypoparathyroidism Drug TransCon PTH

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Ascendis Pharma A/S (ASND - Free Report) announced positive preliminary six-month results from the open-label extension (“OLE”) portion of phase II PaTH Forward study evaluating its prodrug candidate, TransCon PTH in adult patients with hypoparathyroidism (“HP”). The company also filed an amendment to its investigational new drug ("IND") application to initiate a phase III study — PaTHway — on the candidate in the United States.

The company is developing the candidate as a potential replacement therapy for HP, administered once-daily. TransCon PTH works by restoring the parathyroid hormone to physiologic levels in the body for 24 hours, every day, which currently available therapies are unable to do.

Data from PaTH Forward study demonstrated that patients in the extension part of the study receiving maintenance dose of TransCon PTH continued independent of pill burden and consistently improved their quality of life. The patients also achieved improvement in 24-hour urine calcium excretion and serum phosphate. Among patients who were treated with TransCon PTH, 91% were off active vitamin D and were taking less than 500 mg per day of calcium supplements, current standard of care. Moreover, 76% patients eliminated all supplements and 71% patients had serum calcium in the normal range and their 24-hour urine calcium was in the normal range or was reduced by 50% from baseline.

The PaTH Forward study evaluated three doses of TransCon PTH — 15, 18 or 21 µg/day — followed by maintenance dose ranging from 6 to 30 µg in the OLE part. All doses of the candidate were well tolerated.

The company filed its IND amendment for PaTHway study following discussions with the FDA and European regulatory authorities. It plans to file a clinical trial application to initiate study in Europe later this year. The late-stage study will evaluate TransCon PTH in adults with HP for a primary composite endpoint. The composite endpoint is — proportion of patients with serum calcium in the normal range, independence from active vitamin D, and taking less than 600 mg/day of calcium supplements — at week 26.

Shares of Ascendis have increased 9.2% year to date compared with the industry’s growth of 2.2%.

Ascendis Pharma has two other candidates built on the proprietary TransCon platform, which are in different stages of development. The most advanced candidate in its pipeline is TransCon hGH (lonapegsomatropin). The company has filed a biologics license application to the FDA seeking approval for TransConhGH for the treatment of pediatric growth hormone deficiency in June. However, this field is dominated by Novo Nordisk, Pfizer (PFE - Free Report) , Eli Lilly, Sandoz, Merck KGaA and Roche (RHHBY - Free Report) .

A phase II study is evaluating the other candidate, TransCon CNP, for treating achondroplasia, the most common form of dwarfism. We remind investors that BioMarin Pharmaceuticals (BMRN - Free Report) is also developing its pipeline candidate vosoritide for treating achondroplasia, a disease for which no drug is approved yet.

The company is also planning to diversify its pipeline to include oncology candidates and file IND applications to initiate a clinical study on its pre-clinical oncology candidate in the fourth quarter of 2020 and in 2021.

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Ascendis currently carries a Zacks Rank #4 (Sell).

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