Ascendis Pharma Outlines Growth Strategy and Pipeline Progress at 38th Annual J.P. Morgan Healthcare Conference
January 12, 2020 – Copenhagen, Denmark
Ascendis Pharma A/S, a biopharmaceutical company leveraging its proprietary TransCon™ technology platform to target significant unmet medical needs, presented an optimistic outlook for 2020 alongside a review of its Vision 3×3 strategic roadmap extending through 2025. The company shared these details during the 38th Annual J.P. Morgan Healthcare Conference in San Francisco, emphasizing its focus on sustainable growth and advancing transformational therapies.
A Milestone Year and Future Ambitions
Jan Mikkelsen, President and CEO of Ascendis Pharma, reflected on 2019 as a pivotal period in the company’s history. Ascendis celebrated its first phase 3 success with the TransCon hGH candidate for treating growth hormone deficiency (GHD), in addition to advancing two other endocrine disorder programs into phase 2 trials globally. The firm also established oncology as a key therapeutic area, expanding its pipeline with innovative candidates built on the TransCon platform.
Mikkelsen emphasized that these achievements mark only the beginning in building a globally integrated biopharmaceutical leader capable of delivering multiple sustainable growth engines. The company remains committed to its Vision 3×3 goals, dedicated to improving patient outcomes worldwide.
Advances and Forward-Looking Plans in the Pipeline
Ascendis Pharma maintains a robust pipeline centered on rare endocrine diseases, with oncology becoming an emerging focus. Key program updates and upcoming plans for 2020 include:
TransCon hGH: Long-Acting Growth Hormone Therapy
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Clinical Progress: TransCon hGH is a weekly, long-acting prodrug of human growth hormone, designed to provide unmodified somatropin for effective growth promotion in GHD patients. The pivotal phase 3 heiGHt trial demonstrated a statistically significant improvement in height velocity compared to daily hGH treatment.
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Regulatory Milestones: The company held two pre-Biologics License Application (BLA) meetings with the U.S. Food and Drug Administration (FDA) to discuss the chemistry, manufacturing, controls (CMC), and clinical data supporting TransCon hGH for pediatric GHD. Ascendis targets filing its BLA in the second quarter of 2020 and intends to submit a Marketing Authorization Application to the European Medicines Agency (EMA) by year-end.
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Extended Data: Ongoing long-term findings from the enliGHten trial, an extension of heiGHt, reveal continued superior growth with weekly TransCon hGH versus patients initially on daily Genotropin® then switched to TransCon hGH. The safety profile remains consistent between treatments.
- Global Expansion: Plans are underway to initiate a global phase 3 trial for adult GHD in early 2020 and to begin a pediatric trial in Japan by the fourth quarter.
TransCon PTH: Novel Therapy for Hypoparathyroidism
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Clinical Development: TransCon PTH is a once-daily parathyroid hormone prodrug designed to maintain physiological PTH levels over 24 hours, targeting comprehensive management of hypoparathyroidism.
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Trial Updates: The phase 2 PaTH Forward trial has completed screening and aims to enroll approximately 55 subjects. The company anticipates reporting top-line results around March 2020, with open-label extension six-month data expected in the third quarter.
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Promising Early Data: Preliminary findings from initial participants reinforce TransCon PTH’s potential as a valuable therapeutic option, addressing the limitations of current standard care.
- Next Steps: Regulatory submissions to launch a global phase 3 trial in adults with hypoparathyroidism are planned for late 2020.
TransCon CNP: Addressing Achondroplasia in Children
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Therapeutic Focus: TransCon CNP is an investigational weekly long-acting prodrug of C-type natriuretic peptide, intended to treat achondroplasia — the most prevalent form of dwarfism currently with no FDA-approved treatments.
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Clinical Program: The ongoing phase 2 ACcomplisH trial enrolls children ages 2 to 10 with achondroplasia, with sequential dose escalation planned throughout the year.
- Expansion in Asia: Through its strategic investment in VISEN Pharmaceuticals, Ascendis plans to initiate a second phase 2 trial in China during the fourth quarter, further extending the reach of TransCon CNP.
Oncology Pipeline Growth
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Innovative Development: Ascendis is applying its TransCon sustained-release technology to oncology, exploring systemic and localized treatment options.
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Preclinical Progress: Non-human primate studies showed that a single dose of TransCon IL-2 b/g achieves biased receptor binding and prolonged lymphocyte stimulation, supporting the possibility of dosing every three weeks with reduced toxicity.
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Regulatory Engagement: The company’s localized intratumoral (IT) TransCon technology has been accepted into the FDA’s Emerging Technology Program, facilitating enhanced collaboration on technical and regulatory topics before submission.
- Clinical Advancement: Ascendis plans to file an Investigational New Drug (IND) application or equivalent for its initial oncology candidate in 2020, reinforcing its commitment to developing best-in-class cancer therapies.
Upcoming Investor Presentation
Ascendis Pharma’s presentation at the J.P. Morgan Healthcare Conference took place on January 13, 2020, featuring a live session followed by a Q&A. The company’s corporate investor presentation and related materials were made accessible on its website, offering comprehensive insights into its strategy and development programs.
About Ascendis Pharma
Headquartered in Copenhagen, Denmark, with offices in Heidelberg, Germany, and Palo Alto, California, Ascendis Pharma embodies a mission-driven approach to biopharmaceutical innovation. It harnesses its proprietary TransCon™ technology platform to develop potentially best-in-class therapies targeting rare endocrine diseases while expanding into oncology and other therapeutic areas with significant unmet needs. Guided by core values emphasizing patients, science, and passion, Ascendis aims to create meaningful impacts on patients’ lives globally.
Forward-Looking Statements
This summary contains forward-looking statements reflecting Ascendis Pharma’s expectations about future operations, clinical developments, regulatory filings, and business plans. Actual results could vary due to numerous risks including clinical trial outcomes, regulatory processes, manufacturing challenges, financial uncertainties, and other factors. Investors are advised to review company filings for detailed discussions of such risks. Ascendis undertakes no obligation to update these statements except as required by law.
Trademarks: Ascendis, Ascendis Pharma, and TransCon are trademarks owned by the Ascendis Pharma group. © January 2020 Ascendis Pharma A/S.