Ascendis Pharma A/S Announces Dosing of First Subjects in Phase 1 Trial of TransCon CNP
- Category: Proteins and Peptides
- Published on Tuesday, 08 May 2018 15:31
- Hits: 615
- TransCon CNP is Company’s Third Product Candidate in Clinic -
COPENHAGEN, Denmark I May 08, 2018 I Ascendis Pharma A/S (Nasdaq:ASND), a biopharmaceutical company that utilizes its innovative TransCon technology to address significant unmet medical needs, announced today that it has dosed the first subjects in a first-in-human phase 1 trial of TransCon CNP, a long-acting prodrug of C-type natriuretic peptide (CNP) in development as a therapeutic option for achondroplasia and potentially for other fibroblast growth factor receptor (FGFR)-related skeletal disorders. The double-blind, randomized, placebo-controlled phase 1 trial will evaluate single ascending doses of TransCon CNP in healthy adult subjects to assess safety, tolerability and pharmacokinetics.
“Achondroplasia is a serious condition resulting in extensive comorbidities. Given the lack of any FDA-approved therapy, it represents a significant unmet medical need,” said Jonathan Leff, M.D., Ascendis Pharma’s Chief Medical Officer. “TransCon CNP has been designed to provide continuous CNP exposure to optimize efficacy without cardiovascular risk in a convenient once-weekly dose. Data from this trial will help validate our target product profile, once again translating our promising preclinical results into clinical data.”
TransCon CNP is the third product candidate in Ascendis Pharma’s rare disease endocrinology pipeline to advance into clinic, developed using its innovative TransCon technology platform. The company anticipates top-line data from the phase 1 trial to be available in the fourth quarter of 2018.
Achondroplasia is the most common form of dwarfism, affecting approximately 250,000 people worldwide. Achondroplasia results in severe skeletal complications and comorbidities, including narrowing of the foramen magnum, sleep apnea, and chronic ear infections. Individuals with achondroplasia often face multiple surgeries and procedures to alleviate its many complications.
The condition is caused by an autosomal dominant activating mutation in fibroblast growth factor receptor 3 (FGFR3) gene that leads to an imbalance in the effects of the FGFR3 and C-type natriuretic peptide (CNP) signaling pathways. Preclinical and clinical data show that the CNP pathway stimulates growth; increased CNP counteracts the effects of the FGFR3 mutation downstream, thus promoting bone growth.
About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative prodrug technology to build a leading, fully integrated rare disease company focused on making a meaningful difference in patients’ lives. The company utilizes its TransCon technology with clinically validated parent drugs to create new therapies with potential for best-in-class efficacy, safety and/or convenience.
Ascendis Pharma has a wholly-owned pipeline of three rare disease endocrinology product candidates in clinical development. These include once-weekly TransCon Growth Hormone, which is being evaluated in a phase 3 program for children with growth hormone deficiency (GHD), TransCon PTH, a long-acting prodrug of parathyroid hormone for hypoparathyroidism currently in phase 1, and TransCon CNP, a long-acting prodrug of C-type natriuretic peptide, which is also in phase 1 development for achondroplasia and other FGFR-related skeletal disorders.
Additionally, Ascendis Pharma has multi-product collaborations with Sanofi in diabetes and Genentech in the field of ophthalmology.
For more information, please visit www.ascendispharma.com.
SOURCE: Ascendis Pharma