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Ascendis Pivotal Growth Hormone Deficiency Study Data Positive

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Ascendis Pharma A/S (ASND - Free Report) announced positive top-line data from its pivotal phase III study evaluating TransCon Growth Hormone (hGH) for treating growth hormone deficiency (“GHD”) in children. Data from the study showed that hGH significantly increased annualized height velocity (“AVH”) compared to Pfizer's (PFE - Free Report) Genotropin and has a better comparable safety and tolerability profile with the drug.

Ascendis’ shares have gained 90.1% so far this year compared with the industry’s increase of 9.3%.

The pivotal heiGHt study evaluated a once-weekly subcutaneous administration of TransCon hGH for a period of 52 weeks compared to the daily subcutaneous administration of Genotropin. Patients treated with TransCon hGH achieved an AVH of 11.3 cm per year whereas Genotropin had a AVH of 10.3 cm per year. The data were presented at the Endocrine Society’s annual meeting, ENDO 2019.

TransCon hGH is a sustained release formulation of unmodified hGH, which is used in currently approved daily therapies. It helps patients to manage deficiency in growth hormone with once-weekly administration instead of daily intake. Hence, the candidate has significant potential to grab market share, if approved.

Another study – fliGHt – is evaluating TransCom hGH in patients who switch from daily growth hormone therapies to its weekly administration. Data from this study is expected in the second quarter of 2019. The company plans to submit a Biologics License Application for TransCom hGH for treating pediatric GHD in the first half of 2020.

The press release states that GHD in pediatric patients is a serious orphan disease characterized by short height and metabolic abnormalities, which can affect overall physical and mental health. Growth hormone deficiency in pediatric and adult patients represents a market opportunity of more than $3 billion.

Ascendis is a Denmark-based clinical-stage biopharmaceutical company developing several candidates for multiple indications. The company has three pipeline candidates, which are being developed for rare endocrinology diseases. One of them is being developed for treating achondroplasia and was granted orphan drug designation last month.

Ascendis is also focused on developing treatment for oncology indications. Moreover, the company has collaborations with Sanofi (SNY - Free Report) in diabetes and Genentech, a subsidiary of Roche (RHHBY - Free Report) in the field of ophthalmology.

With no approved product, the company needs to successfully commercialize its pipeline candidate for growth. However, the company’s resources are likely to support operations for more than a couple of years at the current rate of its operating expenses.

 

Zacks Rank

Ascendis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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