Danish biopharmaceutical company using its TransCon drug conjugate technology platform to develop new therapies in endocrinology, oncology and other rare disease areas. Listed on NASDAQ (ASND) since 2015.
Clinical Trials
27
12 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
2007
Active, not recruiting
12
44.4%
Completed
8
29.6%
Recruiting
6
22.2%
Terminated
1
3.7%
No approval data available
- Novo Nordisk has acquired three early-stage obesity programs from New York-based Kallyope, deepening a relationship that began with a 2024 research collaboration and single-asset license. - The lead asset, once-weekly peptide K-554, targets a previously undisclosed neural circuit component in feeding regulation and is distinct from GLP-1 and amylin biology. - The two remaining programs are small molecules against separate novel targets and are currently in lead optimization, with no financial terms disclosed. - The deal adds a neural-circuits discovery axis to a Novo obesity pipeline that already spans GLP-1, amylin, CB1 inverse agonist and multi-receptor agonist approaches.
- Canada's Drug Agency (CDA-AMC) has recommended conditional public reimbursement for Yorvipath (palopegteriparatide injection) in adults with chronic hypoparathyroidism inadequately controlled with conventional therapy. - Yorvipath is a parathyroid hormone (PTH) replacement therapy authorized by Health Canada on January 28, 2026, and supported by evidence from the Phase 3 PaTHway trial. - The recommendation marks an important milestone, though final listing decisions rest with participating federal, provincial, and territorial drug plans and hinge on price negotiation. - Clinicians and patient advocates highlight that PTH therapy addresses the underlying hormone deficiency, offering a distinct approach for patients whose disease remains uncontrolled despite calcium and active vitamin D therapy.
- BioMarin and Ascendis Pharma resolved all pending patent disputes, with Ascendis agreeing to pay royalties on sales of its achondroplasia drug Yuviwel (navepegritide). - Ascendis will pay BioMarin 20% of Yuviwel net sales in the U.S. and 18% in the EU, Brazil, and South Korea through May 2030. - The license covers all current and potential indications, including achondroplasia and hypochondroplasia, and use in combination with other medicines. - The settlement recognizes BioMarin's pioneering C-type natriuretic peptide (CNP) technology, including its development of VOXZOGO (vosoritide).
- Eyconis has dosed the first patients in OVERTURE, a Phase 1b/2a first-in-human trial of EYC-0305 in treatment-naïve wet age-related macular degeneration (AMD) patients. - EYC-0305 is a long-acting anti-VEGF antibody fragment using the TransCon platform, designed for six-month or longer dosing intervals via standard intravitreal injection without monthly loading doses. - The open-label, multiple ascending dose study will evaluate safety, tolerability, pharmacokinetics, and disease activity, with primary outcomes of ocular and systemic treatment-emergent adverse events measured at Week 72. - The milestone marks Eyconis' transition to a clinical-stage biotechnology company, with a study completion date of May 2028 reported on ClinicalTrials.gov.
- Ascendis Pharma reported positive clinical trial data for TransCon CNP, highlighting progress in its rare disease pipeline. - The company's revenue growth accelerated to $720 million in 2025, though it remains unprofitable with a net loss of $228 million. - Analyst consensus remains strongly bullish with 92% buy ratings and a $327 price target, reflecting optimism about product launches. - Key risks include persistent losses, high debt levels, and ongoing clinical trial dependencies.
- Ascendis Pharma announced 5-year Phase 2 PaTH Forward Trial data showing TransCon PTH (palopegteriparatide) sustained efficacy and safety in adults with hypoparathyroidism. - 82% of patients met the multi-component endpoint of normal serum calcium, no active vitamin D, and less than 600 mg/day calcium, with 95% completing the full five-year trial. - Significant improvements in kidney function were maintained, with a mean eGFR increase of 9.4 mL/min/1.73 m² from baseline, contrasting with expected age-related decline. - Health-related quality of life scores normalized rapidly and remained in the normative range through Week 266, with no new safety signals identified.
- Palopegteriparatide demonstrated sustained efficacy through 3 years, with 96% of patients achieving independence from conventional therapy and 84% maintaining normal serum calcium levels. - The treatment showed significant kidney benefits, with estimated glomerular filtration rate improving by 8.76 mL/min/1.73 m² overall and 13.98 mL/min/1.73 m² in patients with baseline eGFR below 60. - Mean 24-hour urine calcium excretion decreased to 162 mg per day at 3 years, reaching levels rarely seen in hypoparathyroidism patients. - Patients reported substantial improvements in quality of life and hypoparathyroidism-related symptoms that were maintained throughout the 3-year study period.
- Ascendis Pharma successfully transitioned from American Depositary Shares to direct listing of ordinary shares on Nasdaq Global Select Market on April 20, 2026. - All outstanding ADSs were exchanged on a one-to-one basis for ordinary shares, maintaining the same ASND ticker symbol under new CUSIP and ISIN identifiers. - The company expects the simplified listing structure to broaden global investment access and potentially facilitate inclusion in equity indexes for enhanced institutional ownership. - CFO Scott T. Smith indicated the direct listing aligns with Ascendis' transformation into a leading global biopharmaceutical company focused on TransCon technology platform.
- Rani Therapeutics appointed Dr. Sara Kenkare-Mitra as Strategic Advisor, bringing nearly three decades of biologics development experience and a track record of global regulatory approvals across multiple therapeutic areas. - The company also named Jesper Høiland as Head of Strategy, leveraging his 30+ years of pharmaceutical leadership experience and expertise in obesity and metabolic diseases. - Both appointments aim to advance the RaniPill® platform strategy and prioritize clinical programs with the greatest potential for meaningful impact, particularly following encouraging preclinical data with oral semaglutide. - The strategic hires position Rani to capitalize on the potential of oral biologic delivery across multiple therapeutic areas and support future partnering opportunities.
- BridgeBio's oral drug infigratinib demonstrated superior growth acceleration in children with achondroplasia, achieving 1.74-2.1 cm per year improvement over placebo in a pivotal trial of over 100 patients. - The drug showed best-in-indication efficacy compared to existing injectable treatments, with BioMarin's Voxzogo showing 1.57 cm per year and Ascendis' therapy showing 1.49-1.78 cm per year in their respective trials. - Infigratinib was well-tolerated with no serious adverse events or treatment discontinuations due to side effects, positioning it as a convenient oral alternative to current daily injection therapies. - BridgeBio plans to submit marketing applications in the second half of 2026 and accelerate development in hypochondroplasia, potentially capturing significant market share in the growing dwarfism treatment space.
