Positive Results from VISEN’s Phase 3 Trial of Once-Weekly TransCon hGH in China Consistent with Ascendis Pharma’s Phase 3 Height Trial
Ascendis Pharma A/S announced positive results from VISEN Pharmaceuticals' Phase 3 trial of TransCon™ hGH for pediatric growth hormone deficiency (GHD) in China. The trial met its primary endpoint, demonstrating significantly greater annualized height velocity at 52 weeks (p=0.0010) compared to daily growth hormone treatment. This result aligns with previous multinational trial outcomes, confirming the safety and efficacy of TransCon hGH. The treatment is now approved in the U.S. and EU. The CEO praised VISEN for its achievement, underscoring TransCon technology's global applicability.
- Phase 3 trial of TransCon hGH met primary endpoint with p=0.0010, indicating significant improvement in height velocity.
- Results consistent with prior multinational trials, reinforcing TransCon hGH's efficacy and safety.
- TransCon hGH is now approved in both the U.S. and EU for pediatric GHD.
- None.
- VISEN’s Phase 3 Trial achieved primary endpoint; pediatric growth hormone deficiency (GHD) patients treated with TransCon™ hGH demonstrated greater annualized height velocity at 52-weeks (p=0.0010) compared to patients treated with daily growth hormone
- These results demonstrate the ability of TransCon technology to deliver consistent and reproducible results for patients across a broad range of geographies and populations
COPENHAGEN, Denmark, May 23, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) announced today that results from VISEN Pharmaceuticals’ Phase 3 trial of once-weekly TransCon hGH in children with GHD in China demonstrated results that were consistent with the Ascendis Pharma’s earlier multi-national Phase 3 trial. VISEN Pharmaceuticals’ Phase 3 trial achieved its primary endpoint, with pediatric GHD patients treated with once-weekly TransCon hGH demonstrating greater annualized height velocity at 52-weeks (p=0.0010) compared to patients treated with daily growth hormone. In both Ascendis Pharma’s and VISEN Pharmaceuticals’ Phase 3 trials, TransCon hGH – now approved in both the U.S. and EU as a once-weekly treatment for pediatric GHD – demonstrated statistical non-inferiority and superiority on the primary endpoint with comparable safety and tolerability to daily growth hormone.
“Ascendis Pharma congratulates VISEN Pharmaceuticals for successfully completing its first Phase 3 clinical trial of a TransCon product candidate in China,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “These results demonstrate the ability of TransCon technology to deliver consistent and reproducible results for patients across a broad range of geographies and populations.”
Results of the Phase 3 trial of TransCon hGH in China were reported by VISEN Pharmaceuticals, whose press release can be viewed on www.visenpharma.com.
About Pediatric Growth Hormone Deficiency
Pediatric GHD is a serious orphan disease characterized by short stature and metabolic abnormalities that affect overall physical and mental health. In GHD, the pituitary gland does not produce sufficient growth hormone, which is important not only for height but also for optimal bone, heart, muscle, and brain development.
About TransCon™ Technology
TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent 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 clearance. When injected into the body, physiologic pH and temperature conditions initiate the release of the active, unmodified parent drug in a predictable release manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and can be used systemically or locally.
About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Heidelberg and Berlin, Germany; Palo Alto and Redwood City, California; and Princeton, New Jersey. Please visit www.ascendispharma.com to learn more.
Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ ability to apply its platform technology to build a leading, fully integrated biopharma company, and (ii) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers and distributors to supply TransCon hGH, the SKYTROFA® Auto-Injector and other study drug for commercial sales in the U.S. and clinical studies; unforeseen safety or efficacy results in its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to commercialization of lonapegsomatropin-tcgd in the U.S., the co-pay program, and the further development of TransCon hGH, expenses related to the development and potential commercialization of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs, selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; dependence on third party manufacturers to supply study drug for planned clinical studies; Ascendis’ ability to obtain additional funding, if needed, to support its business activities and the effects on its business from the worldwide COVID-19 pandemic. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on March 2, 2022 and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and SKYTROFA are trademarks owned by the Ascendis Pharma Group. © May 2022 Ascendis Pharma A/S.
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