Ascendis Pharma Announces Positive Preliminary Phase 1 Data for TransCon CNP

Ascendis Pharma (Nasdaq:ASND), a biopharmaceutical company that utilizes its innovative TransCon^™ technology to address significant unmet medical needs, today announced positive preliminary results from a phase 1 trial of TransCon CNP.

As quoted in the press release:

“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 Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “This is an important step forward for patients. We believe that TransCon CNP is well-suited to address the challenges of people living with achondroplasia, and offers a potential new therapeutic option that looks beyond height to address the associated comorbidities. We look forward to advancing this program into clinical trials for children living with achondroplasia next year.”

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.

Click here to read the full press release.