Ascendis Pharma offers positive surprise as its tech not just equals, but improves outcomes in key study

Technology developed by Denmark’s Ascendis Pharma to release a particular drug within the body in a predictable manner at regular intervals without the need for multiple separate administrations has been shown to work well in a late-stage trial in pediatric patients with growth hormone deficiency — validating the company’s platform that is used across its portfolio.

The technology, called TransCon or transient conjugation, comprises an unmodified approved drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from being released, but once the product is injected into the body, physiologic pH and temperature conditions allow for the predictable dissemination of the active drug — this can be done daily or even half yearly. The tech can be used for systemic administration or locally in areas that are difficult to treat with conventional therapy.

The 161 treatment-naïve-patient-trial, called heiGHt, compared the once-weekly TransCon growth hormone (hGH) against a daily growth hormone — Pfizer’s Genotropin — in children with growth hormone deficiency, an orphan disease caused by the insufficient production of growth hormone by the pituitary gland that is characterized by short stature, metabolic abnormalities, psychosocial challenges and cognitive gaps.

TransCon hGH is designed to deliver unmodified hGH — the same growth hormone used in daily therapies — at a predictable rate over one week. Currently in the United States and Europe, patients must contend with daily hGH injections resulting in thousands of injections over time, which often leads to poor patient compliance, the company said.

Trial data showed that Ascendis’ product not only met the main goal of non-inferiority in annualized height velocity (a measure of speed of growth) at 52 weeks, but actually proved superior to daily hGH. These results could constitute a home run, considering various analysts were expecting just the non-inferiority benchmark to be met.

Joseph Schwartz

In the primary analysis of the intent-to-treat population, TransCon hGH demonstrated a significantly greater annualized height velocity of 11.2 cm/year compared to 10.3 cm/year for the daily hGH — the treatment difference amounted to 0.86 cm/year, hitting a p-value of 0.0088. The experimental treatment began to surpass the comparator at a statistically significant rate from week 26 onward.

Ascendis’ shares $ASND skyrocketed more than 64.6% in morning trading to $114.10.

“The stock is not cheap, but we do see room for significant upside upon positive pivotal data that may be used for registration of the first long-acting growth hormone treatment, which has been a prized goal for many companies over several decades,” Leerink’s Joseph Schwartz wrote in a note in late January.

The company, which has partnered with Roche’s Genentech and Sanofi to evaluate its technology for ophthalmology and diabetes drugs respectively, plans to submit a marketing application for its pediatric GHD product in the first half of 2020.

“Ascendis’ management continues to deliver with this best-case outcome for TransCon hGH in a Ph3 trial, further validating the technology platform and with positive read-through to other clinical programs and pipeline potential. We also see the positive Ph3 raising the company’s profile as a potential acquisition target, as Ascendis offers balanced clinical pipeline with fairly de-risked programs, potential best-in-class assets, an emerging new therapeutics vertical and plenty of optionality within the TransCon technology platform,” Credit Suisse analysts wrote in a note.

Top-line data for the fliGHt Trial, evaluating TransCon hGH in subjects who switch from daily hGH, are expected in the second quarter. The company is also evaluating its technology for use in hypoparathyroidism and achondroplasia.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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