Ascendis Pharma A/S Announces Positive Preliminary Phase 1 Data for TransCon CNP
- TransCon CNP delivered continuous exposure of CNP at target levels for seven days with a single subcutaneous administration -
- TransCon CNP phase 1 results reinforce preclinical data and support target product profile -
A presentation of the phase 1 results may be viewed on the
“The TransCon CNP data presented for the first time today indicate that our product candidate delivered continuous exposure of CNP at target levels over seven days, supporting once-weekly administration,” said
In this phase 1, double-blind, randomized, placebo-controlled trial, 45 healthy adult subjects were enrolled. Five doses of TransCon CNP were tested sequentially, beginning with the lowest dose: 3.0, 10, 25, 75 and 150 microgram/kg. Up to 10 subjects in each dose cohort were randomized to receive TransCon CNP or placebo in a 4:1 ratio. After each cohort completed dosing, a Data Safety Monitoring Board (DSMB) reviewed the blinded data to approve escalation to the next higher dose. The primary endpoint was frequency of adverse events after administration of TransCon CNP. Secondary endpoints included additional safety parameters, tolerability and pharmacokinetics.
TransCon CNP is a long-acting prodrug of CNP comprised of an unmodified parent drug transiently bound to an inert carrier molecule via a proprietary linker. Following subcutaneous administration, TransCon CNP is designed to allow the body’s physiologic pH and temperature to release and activate CNP in a controlled manner over the course of one week. The carrier and linker are then cleared through the kidneys. Because the parent CNP drug is unmodified, its mode of action is expected to be maintained. TransCon CNP is in development as a treatment for achondroplasia and has potential as a treatment for other growth disorders, including fibroblast growth factor receptor (FGFR)-related skeletal disorders.
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 the fibroblast growth factor receptor 3 (FGFR3) gene that leads to an imbalance in the effects of the FGFR3 and 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.
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 utilizes its TransCon™ technology to create new and potentially best-in-class therapies.
Ascendis is headquartered in
For more information, please visit www.ascendispharma.com.
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 plans to initiate our phase 2 TransCon CNP trial in pediatric subjects with achondroplasia in mid-2019, (ii) our ability to apply our platform technology to build a leading, fully integrated biopharma company, (iii) our expectations regarding our ability to create potentially best-in-class therapies 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 hGH, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development of TransCon hGH, 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 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; 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 (
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Source: Ascendis Pharma A/S