Ascendis Pharma A/S Provides Pipeline Update and Reviews Progress Towards Vision 3x3 at 38th Annual J.P. Morgan Healthcare Conference
“2019 was a transformative year for
Pipeline Updates and 2020 Outlook
- TransCon hGH: TransCon hGH is an investigational long-acting prodrug of human growth hormone (hGH) in phase 3 development as a once-weekly treatment for growth hormone deficiency (GHD). TransCon hGH releases unmodified somatropin and has demonstrated a statistically significant increase in height velocity compared to a daily hGH in the phase 3 heiGHt Trial:
Ascendis Pharma recently held two pre-BLA meetings with the U.S. Food and Drug Adminstration (FDA ) to review its Chemistry, Manufacturing and Controls (CMC), and clinical/non-clinical packages for TransCon hGH as a potential treatment for pediatric GHD. The company is on track to file a Biologics License Application with theFDA in the second quarter. A Marketing Authorisation Application to theEuropean Medicines Agency is planned to follow in the fourth quarter.- Long-term data presented from the ongoing enliGHten Trial (long-term extension) continued to demonstrate statistically superior growth of subjects treated with once-weekly TransCon hGH in the heiGHt Trial who continued into enliGHten, compared to those who started treatment with daily Genotropin® and switched to TransCon hGH after one year. The adverse event profile of TransCon hGH, which was comparable to Genotropin in the phase 3 heiGHt Trial, was consistent across the phase 3 clinical trials.
- The company plans to submit regulatory filings to initiate a global, phase 3 clinical trial in adult GHD during the first quarter, and to initiate a trial in pediatric GHD in
Japan during the fourth quarter.
- TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a once-daily replacement therapy for hypoparathyroidism (HP) designed to replace PTH at physiologic levels for 24 hours each day and fully address all aspects of the disease:
- Following completion of screening of subjects in the recently expanded phase 2 PaTH Forward Trial, Ascendis intends to enroll approximately 55 subjects in the trial. The company expects to report top-line results from the trial around the end of
March 2020 , with six-month data from the open-label extension phase expected in the third quarter. - Preliminary data presented from the first eight subjects who completed four weeks of follow-up in the open-label extension portion of the phase 2 PaTH Forward Trial reinforce the company’s target product profile for TransCon PTH as a promising new potential therapy for HP in the absence of standard of care.
- The company plans to submit regulatory filings to initiate a global, phase 3 clinical trial in adults with HP during the fourth quarter.
- Following completion of screening of subjects in the recently expanded phase 2 PaTH Forward Trial, Ascendis intends to enroll approximately 55 subjects in the trial. The company expects to report top-line results from the trial around the end of
- TransCon CNP: TransCon CNP is an investigational long-acting prodrug of CNP in development as a therapy for children with achondroplasia, the most common form of dwarfism, for which there is no
FDA -approved treatment. TransCon CNP is designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose:
- Ascendis is conducting the phase 2 ACcomplisH Trial of TransCon CNP in children (ages 2-10 years) with achondroplasia and plans to escalate sequential dose cohorts throughout 2020.
- The company is expanding the TransCon CNP program in
China through its strategic investment inVISEN Pharmaceuticals , with initiation of a second phase 2 trial in children with achondroplasia during the fourth quarter.
- Oncology: Ascendis continues to advance a pipeline of multiple pre-clinical programs in oncology by applying both systemic and sustained localized TransCon technologies for clinically validated pathways:
- Additional data from non-human primate studies demonstrated that a single dose of TransCon IL-2 b/g provided biased receptor binding and prolonged enhancement of lymphocyte counts, suggesting feasibility of every three week dosing and reduced risk of toxicity.
- Ascendis Pharma’s innovative TransCon technology for sustained localized release intratumorally (IT) was accepted to participate in the FDA’s Emerging Technology Program. The program provides for enhanced interactions and dialogue with the
FDA to discuss, identify and resolve potential technical and CMC regulatory questions related to the TransCon sustained IT programs prior to filing regulatory submissions. - The company plans to file an IND or equivalent for its first oncology program in 2020, furthering the goal to create best-in-class oncology therapeutics.
Presentation at
Live webcasts of the J.P. Morgan presentation and associated Question & Answer session will be available in the Investors and News section of the
The presentation will begin at
The company’s corporate investor presentation and slides from the J.P. Morgan presentation are also available in the Investors and News section.
About
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™ technologies to create new and potentially best-in-class therapies.
Ascendis is headquartered in
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 expectation to file a Biologics License Application with the U.S.
Ascendis,
Internal contact:Scott T. Smith Chief Financial Officer (650) 352-8389 ir@ascendispharma.com Media contact: Ami Knoefler Head ofGlobal Communications (650) 739-9952 ack@ascendispharma.com Investor contact:Patti Bank Westwicke Partners (415) 513-1284 patti.bank@westwicke.com
Source: Ascendis Pharma