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- TransCon CNP is Company’s Third Product Candidate in Clinic -
COPENHAGEN, Denmark, May 08, 2018 -- 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.
About Achondroplasia
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.
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 our 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) our ability to apply our prodrug technology to build a leading, fully integrated rare disease company, (ii) our expectations regarding when top-line data from our TransCon CNP phase 1 trial will be available, (iii) our expectations regarding our ability to create therapies with potential for best-in-class efficacy, safety and/or convenience and (iv) our product pipeline. We 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 we make, including the following: unforeseen safety or efficacy results in our TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development of TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs, general and administrative expenses, other research and development expenses and our business generally; delays in the development of TransCon Growth Hormone, 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; and our ability to obtain additional funding, if needed, to support our business activities. 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 our business in general, see our current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including our Annual Report on Form 20-F for the year ended December 31, 2017, which we filed with the SEC on March 28, 2018. Forward-looking statements do not reflect the potential impact of any future in-licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments we may enter into or make. We do not assume any obligation to update any forward-looking statements, except as required by law.
Internal contact: Scott T. Smith Chief Financial Officer (650) 352-8389 [email protected] Media contact: Ami Knoefler Head of Global Communications (650) 739-9952 [email protected] Investor contact: Patti Bank Westwicke Partners (415) 513-1284 [email protected]
