On November 14, 2024 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the third quarter ended September 30, 2024, and provided a business update (Press release, Ascendis Pharma, NOV 14, 2024, View Source [SID1234649389]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2024 has been another transformative year for Ascendis. Now, all three of our three endocrinology rare disease programs have delivered clinically differentiated pivotal data, each demonstrating potential ability to address major unmet medical needs and the potential for each to achieve blockbuster status. We are ready and very excited about launching YORVIPATH in the U.S. with product availability expected in mid-January of 2025," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "In addition, our new partnership with Novo Nordisk highlights our ability to extend the success of our TransCon platform and positions Ascendis to benefit patients and capture significant value in this large, high volume therapeutic areas."

Select Highlights & Anticipated 2024 Milestones

TransCon hGH:
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA revenue for the third quarter of 2024 totaled €47.2 million compared to €47.0 million during the same period in 2023. Volume growth was offset by higher sales deductions and a negative adjustment to prior period sales deductions of €2.5 million.
SKYTROFA revenue for the first nine months of 2024 totaled €138.5 million, a 21% year-over-year increase compared to €114.4 million during the same period of 2023. Volume growth was partially offset by higher sales deductions. In addition, sales deductions attributable to periods prior to January 1, 2024 totaled €9.3 million.
Submitted U.S. Food & Drug Administration (FDA) supplemental Biologics License Application for TransCon hGH for the treatment of adults with growth hormone deficiency.
Topline results from Phase 2 New InsiGHTS Trial in Turner syndrome expected in the fourth quarter of 2024.
Expect to initiate a basket trial evaluating SKYTROFA in other established daily growth hormone indications in the first half of 2025.
TransCon PTH:
(palopegteriparatide, marketed as YORVIPATH)
YORVIPATH launching in U.S. with our expanded U.S. field teams engaging with health care providers, our Ascendis Signature Access Program accepting prescriptions and enrolling patients starting in December in preparation for product availability in mid-January 2025
Third quarter YORVIPATH revenue outside the U.S. totaled €8.5 million, a sequential quarter-over-quarter revenue increase of more than 60%, reflecting growing physician and patient demand with now over 600 patients on treatment, partially offset by accruals reflecting the end of the free pricing period in the third quarter. Final pricing in Germany is expected to be completed next year.
TransCon CNP
(navepegritide)
Announced positive topline data from pivotal ApproaCH Trial with children with achondroplasia (ages 2-11 years) treated with once-weekly TransCon CNP.
Plan to submit New Drug Application (NDA) to the FDA for TransCon CNP for the treatment of children with achondroplasia during the first quarter of 2025 and a Marketing Authorisation Application (MAA) for the treatment of children with achondroplasia to the European Medicines Agency during the third quarter of 2025.
Expect topline Week 26 data from COACH, the combination TransCon hGH and TransCon CNP trial of children with achondroplasia (ages 2-11 years) in the second quarter of 2025.
Oncology Programs
Presented first results from platinum-resistant ovarian cancer (PROC) cohort of the Phase 1/2 IL-Believe Trial at ESMO (Free ESMO Whitepaper) 2024. Initial data suggest clinical activity in heavily pre-treated PROC patients and that TransCon IL-2 β/γ in combination with chemotherapy was generally well-tolerated.
Recently, we closed enrollment to dose expansion cohorts involving TransCon TLR7/8 Agonist in the transcendIT-101 and IL Believe trials to prioritize our efforts on TransCon IL-2 β⁄γ.
Strategic Collaboration
In November, granted Novo Nordisk A/S an exclusive, multi-product, worldwide license to the TransCon technology platform to develop, manufacture and commercialize Novo Nordisk proprietary products in metabolic diseases and a product-by-product exclusive license in cardiovascular diseases. The lead program in the collaboration is a once-monthly GLP-1 receptor agonist product candidate that will initially target obesity and type 2 diabetes.
For the lead program, Ascendis will be eligible to receive total payments of up to $285 million in upfront, development, and regulatory milestone payments, plus sales-based milestone payments and escalating tiered, mid-single digit royalties on global net sales.
Novo Nordisk will be responsible for Ascendis’ early development costs as well as for clinical development, regulatory, commercial manufacturing, and commercialization activities.
Financial Update and Outlook Based on Current Plans
As of September 30, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities, totaling €626 million, compared to €399 million as of December 31, 2023.
Full year 2024 SKYTROFA revenue excluding sales deductions related to prior years expected to be €200 – €220 million.
Expect total operating expenses (SG&A and R&D) to be approximately €600 million for 2024.
Third Quarter 2024 Financial Results
Total revenue for the third quarter of 2024 was €57.8 million, compared to €48.0 million during the same period for 2023. The year-over-year increase in total revenue was primarily attributable to revenue contribution of €8.5 million from YORVIPATH following commercial launch in the first quarter of 2024. Non-product revenue was €2.1 million in the third quarter of 2024, compared to €1.1 million for the same period for 2023.