ASCENDIS PHARMA A (ASND)

Expected completion of clinical trial for TransCon CNP in Achondroplasia

Ascendis Pharma A/S expects to complete a clinical trial for TransCon CNP, which aims to evaluate the efficacy and safety of the drug in children with Achondroplasia, on August 1, 2025. The trial, identified as NCT05598320, involves 84 participants aged 2 to 11 years and compares once weekly subcutaneous doses of 100 µg CNP/kg against a placebo. It includes a double-blind, placebo-controlled phase followed by an open-label extension. The results could inform further development of TransCon CNP, which targets a significant unmet need in treating Achondroplasia, a genetic growth disorder.

• www.clinicaltrials.gov/study/NCT05598320

Expected Completion of TransCon TLR7/8 Agonist Clinical Trial

Ascendis Pharma's clinical trial for the TransCon TLR7/8 Agonist, designed for patients with advanced or metastatic solid tumors, is expected to complete on March 1, 2026. This trial evaluates the safety and efficacy of the agonist, with or without the checkpoint inhibitor Pembrolizumab. The TransCon TLR7/8 Agonist aims to deliver localized, prolonged concentrations of resiquimod, a potent TLR 7/8 agonist, thereby potentially enhancing anti-tumor immune responses while minimizing systemic exposure. The study is currently active but not recruiting, with an enrollment count of 188.

• www.clinicaltrials.gov/study/NCT04799054

Expected Completion of Clinical Trial for Navepegritide in Adolescents with Achondroplasia

Ascendis Pharma A/S expects to complete a clinical trial evaluating navepegritide, a drug for treating achondroplasia in adolescents, on January 1, 2027. The trial, designated NCT06732895, is currently recruiting participants aged 12 to 18. It aims to assess the efficacy and safety of once weekly subcutaneous doses of navepegritide at 100 μg/kg compared to a placebo over a 52-week period, with a primary measure of annualized growth velocity. The trial plans to enroll 24 participants in Phase 2 of development.

Expected Completion of Clinical Trial for TransCon CNP in Infants with Achondroplasia

Ascendis Pharma A/S is set to complete a Phase 2 clinical trial evaluating the safety and efficacy of TransCon CNP, a drug aimed at treating infants with Achondroplasia, by March 1, 2027. The trial, which is double-blind and randomized, will assess 100 μg CNP/kg administered once-weekly for 52 weeks to 72 participants under two years of age. The trial is currently in the recruiting phase, with a start date of January 23, 2024. TransCon CNP is designed to address the genetic condition of Achondroplasia, potentially advancing treatment options for affected infants.

Expected Completion of Phase 2 Clinical Trial for Navepegritide in Children with Achondroplasia

Ascendis Pharma Growth Disorders A/S is expected to complete a Phase 2 clinical trial for Navepegritide, in combination with Lonapegsomatropin, in children with Achondroplasia on September 1, 2027. This trial aims to assess the efficacy, safety, and tolerability of the combination treatment in children aged 2 to 11 years. The trial is currently active and not recruiting, with an enrollment of 22 participants. This proof-of-concept study may provide critical insights into potential therapeutic strategies for Achondroplasia, a genetic disorder affecting growth.

Expected Completion of Lonapegsomatropin Clinical Trial for Turner Syndrome

Ascendis Pharma A/S is set to complete its clinical trial for Lonapegsomatropin, a long-acting growth hormone product, on December 1, 2027. The trial, registered as NCT05690386, aims to compare various doses of Lonapegsomatropin with daily doses of Somatropin in prepubertal individuals with Turner Syndrome. The study involves 48 participants and consists of a 104-week dose-finding phase followed by an optional 78-week extension. The clinical trial is currently active but not recruiting new subjects, providing insights into the medication's efficacy and safety for this specific patient population. The outcome may impact future treatment protocols for Turner Syndrome, potentially affecting market dynamics within the endocrinology sector.

• www.clinicaltrials.gov/study/NCT05690386

Expected trial completion for TransCon IL-2 β/γ Clinical Trial

Ascendis Pharma's clinical trial of TransCon IL-2 β/γ, designed to enhance the safety and efficacy of IL-2 through a long-acting delivery system, is expected to complete on August 1, 2029. The trial investigates the safety and tolerability of this drug alone or in combination with Pembrolizumab and other anticancer therapies for participants with locally advanced or metastatic solid tumors. The trial is presently recruiting with an enrollment count of 345 participants.

• www.clinicaltrials.gov/study/NCT05081609

Completion of Lonapegsomatropin PASS Clinical Trial

Ascendis Pharma's Post-Authorization Safety Study (PASS) of lonapegsomatropin is expected to conclude on March 1, 2033. The trial, which began recruiting on March 20, 2023, aims to assess the long-term safety risks associated with lonapegsomatropin in a real-world context for approximately 500 patients. This study is essential as it helps in better understanding the potential safety profile of the drug in the post-marketing phase, which could impact its commercial viability and regulatory status.

• www.clinicaltrials.gov/study/NCT05775523

Expected Completion of SKYTROFA Clinical Trial

Ascendis Pharma A/S expects to complete the SKYTROFA clinical trial on March 1, 2033. This study, identified as NCT05820672, is a non-interventional effectiveness and safety trial involving 900 patients treated with SKYTROFA under routine clinical practice. The trial is currently recruiting participants, with the study beginning on March 20, 2023. The study aims to assess the effectiveness and safety of SKYTROFA, which is used in the treatment of conditions related to growth hormone deficiency.

• www.clinicaltrials.gov/study/NCT05820672

Expected Completion of Clinical Trial NCT05929807

Ascendis Pharma A/S is expected to complete its clinical trial (NCT05929807) on March 1, 2039, investigating the long-term safety, tolerability, and efficacy of weekly subcutaneous doses of TransCon CNP in children and adolescents with achondroplasia. The trial is in Phase 2/3 and currently enrolling participants who have previously completed related studies. TransCon CNP is being administered to help manage growth in patients with this condition, and treatment will continue until specified growth milestones are met. The trial aims to gather additional safety and efficacy data regarding TransCon CNP in this patient population.

• www.clinicaltrials.gov/study/NCT05929807