Ascendis Pharma A/S
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2006-01-01
- Employees
- 14
- Market Cap
- $7.9B
- Introduction
Ascendant Resources Inc is a mining company. The company is engaged in evaluating resource opportunities. The Company’s sole investment and exploration activities are in the Lagoa Salgada Project in Portugal. It is focused on its own producing El Mochito zinc, silver and lead mine. The El Mochito mine is located in northwestern Honduras. The underground operation takes advantage of selective and bulk underground mining methods to mine multiple flat-lying manto deposits and high-grade vertical chimneys, producing zinc and lead concentrates with silver credits.
TransCon PTH Shows Sustained 4-Year Efficacy in Hypoparathyroidism Patients
• New data from Ascendis Pharma's Phase 2 PaTH Forward Trial demonstrates that TransCon PTH (palopegteriparatide) provides durable response in adults with hypoparathyroidism through 214 weeks of treatment.
• At Week 214, 98% of patients maintained normal serum calcium levels and 93% remained independent from conventional therapy, with 67.8% showing clinically meaningful improvements in kidney function.
• The treatment demonstrated a favorable safety profile with no new safety signals identified, while also showing sustained normalization of skeletal dynamics with bone mineral density remaining within normal ranges.
Ascendis Pharma Submits NDA for Novel Achondroplasia Treatment with Multiple Clinical Benefits
• Ascendis Pharma has submitted a New Drug Application to the FDA for TransCon CNP (navepegritide), a once-weekly treatment for children with achondroplasia that demonstrated benefits beyond linear growth.
• The application is supported by robust clinical data from three randomized, double-blind, placebo-controlled trials with up to three years of open-label extension data showing improved muscle function and reduced leg bowing.
• Achondroplasia affects over 250,000 people globally with complications including spinal deformities, muscle weakness, and respiratory issues, highlighting the significant unmet medical need this therapy aims to address.
Bristol Myers Squibb Acquires 2seventy Bio for $102 Million as Gene Therapy Sector Faces Valuation Challenges
• Bristol Myers Squibb has acquired 2seventy Bio for $102 million net, just weeks after Bluebird Bio, 2seventy's parent company, was sold for a mere $30 million, highlighting significant valuation challenges in the gene therapy sector.
• The low acquisition prices for both companies underscore the broader business challenges facing gene therapy developers, despite creating treatments that deliver life-changing value to patients.
• Despite recent setbacks and commercial hurdles related to complex manufacturing and high pricing, biopharmaceutical companies continue to invest in gene therapy research, indicating ongoing confidence in the field's scientific potential.
Ascendis Pharma A/S Announces Key Updates and Achievements in 2024
Ascendis Pharma A/S has provided a comprehensive update on its business and strategic roadmap, including significant milestones for 2025, participation in the 43rd Annual J.P. Morgan Healthcare Conference, and the commercial availability of YORVIPATH® in the United States. The company also announced positive results from the New InsiGHTS trial and the FDA's acceptance of its supplemental Biologics License Application for TransCon™ hGH.
TransCon hGH Meets Primary Endpoint in Turner Syndrome Trial
• Ascendis Pharma's New InsiGHTS Phase 2 trial demonstrated that TransCon hGH achieved its primary objective in children with Turner syndrome at Week 26.
• The study showed comparable safety and tolerability between once-weekly TransCon hGH and daily somatropin, with no discontinuations related to the study drug.
• Annualized height velocity in children treated with TransCon hGH was similar to that observed in those treated with daily somatropin.
• Ascendis Pharma plans a basket trial in 2025 to support label expansion for TransCon hGH based on these positive results.
FDA Accepts Ascendis Pharma's TransCon hGH Application for Adult Growth Hormone Deficiency
• The FDA has accepted Ascendis Pharma's sBLA for TransCon hGH to treat adult growth hormone deficiency, setting a PDUFA date for July 27, 2025.
• Phase 3 foresiGHt trial data demonstrated TransCon hGH's superiority in reducing trunk fat and increasing lean body mass compared to placebo.
• TransCon hGH showed a strong safety profile, with tolerability comparable to daily hGH treatment and no discontinuations related to the study drug.
• The potential approval could address a significant unmet need, as only 5-10% of adults with GHD currently receive treatment.
Ascendis Pharma's Yorvipath Commercially Available in the US, Offering New Hope for Hypoparathyroidism
• Ascendis Pharma has launched Yorvipath (palopegteriparatide) in the US, the first FDA-approved treatment for hypoparathyroidism in adults.
• Yorvipath is a once-daily prodrug of parathyroid hormone, designed to provide continuous exposure to active PTH over 24 hours.
• Ascendis has established a dedicated support program to assist patients with clinical education, access, and injection training.
• Clinical trials demonstrated that 93% of YORVIPATH-treated patients achieved independence from both active vitamin D and therapeutic doses of elemental calcium.
Ascendis Pharma Announces Progress Across Endocrinology and Oncology Programs in Q3 2024
• Ascendis Pharma's YORVIPATH is set to launch in the U.S. by mid-January 2025, with significant revenue growth reported ex-U.S. in Q3 2024.
• The company plans to submit an NDA for TransCon CNP to the FDA in Q1 2025 for treating achondroplasia in children, followed by an MAA submission to the EMA in Q3 2025.
• SKYTROFA revenue reached €47.2 million in Q3 2024, with full-year revenue expected to be between €200-€220 million, excluding prior sales deductions.
• A collaboration with Novo Nordisk was established to develop treatments for metabolic and cardiovascular diseases, including a once-monthly GLP-1 receptor agonist.
Novo Nordisk Enters Agreement with Ascendis Pharma for Monthly GLP-1 Agonist
• Novo Nordisk secures exclusive global rights to Ascendis Pharma's TransCon technology for developing treatments for metabolic diseases, including obesity and type 2 diabetes.
• The lead project focuses on a once-monthly GLP-1 receptor agonist, initially targeting obesity and type 2 diabetes, with potential expansion into other therapeutic areas.
• Ascendis Pharma will receive up to $285 million in upfront, development, and regulatory milestone payments for the lead program, plus sales-based milestones and royalties.
• Novo Nordisk will fund clinical development, manufacturing, and commercialization, while Ascendis leads early-stage development; the deal awaits regulatory approval.
TransCon PTH Demonstrates Sustained Skeletal Benefits in Hypoparathyroidism Patients: 3-Year Data
• Ascendis Pharma's TransCon PTH (palopegteriparatide) normalized bone remodeling in adults with hypoparathyroidism after 162 weeks of treatment.
• The Phase 2 PaTH Forward Trial results suggest long-term palopegteriparatide treatment promotes skeletal health, mirroring that of parathyroid sufficiency.
• The study involved 57 adults and showed TransCon PTH, a once-daily prodrug, effectively provides continuous PTH exposure over 24 hours.
TransCon IL-2 β/γ Shows Promise in Platinum-Resistant Ovarian Cancer
• Ascendis Pharma's TransCon IL-2 β/γ demonstrated anti-tumor activity in 29% of efficacy-evaluable patients with platinum-resistant ovarian cancer (PROC).
• The Phase 1/2 IL-Believe trial data suggests clinical benefit in heavily pre-treated PROC patients, including those who progressed on Elahere.
• The combination of TransCon IL-2 β/γ and chemotherapy was generally well-tolerated, with mostly grade 1 or 2 treatment-emergent adverse events.
• These findings, presented at ESMO 2024, indicate potential efficacy across multiple cancer types, building on previous results in melanoma.
Ascendis Pharma Unveils Vision 2030 Strategic Roadmap
Ascendis Pharma A/S introduces Vision 2030, a strategic roadmap aiming for blockbuster status for multiple products and expanding future innovation. Key updates include progress on TransCon™ hGH, TransCon PTH, and TransCon CNP, alongside plans for global expansion and new therapeutic areas.
Ascendis Pharma A/S Updates on Vision 3x3 Strategy and 2022 Milestones
Ascendis Pharma A/S provided an update on its Vision 3x3 strategic roadmap and key milestones for 2022 at the 40th Annual J.P. Morgan Healthcare Conference. The company highlighted its progress in 2021, including the U.S. launch of SKYTROFA®, and outlined its plans for clinical data readouts and regulatory submissions across its endocrinology rare disease and oncology portfolios in 2022.
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