Ascendis Pharma Stock Price, News & Analysis

Ascendis Pharma has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency for TransCon PTH, a potential first treatment for hypoparathyroidism. This condition affects an estimated 85,000-223,000 patients in Europe and currently lacks effective hormonal replacement therapy. The MAA is based on data from the Phase 3 PaTHway Trial, where 77 out of 79 patients continued treatment, indicating strong efficacy and tolerability. The FDA is currently reviewing TransCon PTH, with a target action date of April 30, 2023.

Ascendis Pharma announced positive topline results from the ACcomplisH Trial, a Phase 2 study evaluating the efficacy of once-weekly TransCon CNP in children with achondroplasia. The trial demonstrated a statistically significant improvement in annualized height velocity (AHV) at 52 weeks for the 100 µg/kg/week dose compared to placebo (p=0.0218). Remarkably, all 57 participants completed the trial, indicating robust tolerability. These findings support further development of TransCon CNP as a treatment option for achondroplasia. Ascendis will discuss these results in a conference call on November 14, 2022.

Ascendis Pharma (Nasdaq: ASND) has provided new data from the Phase 1/2 transcendIT-101 trial of TransCon TLR7/8 Agonist for advanced solid tumors. Results show early signs of clinical activity when used alone or with pembrolizumab, along with no systemic toxicity. The treatment demonstrated sustained immune system engagement after one injection. The recommended Phase 2 dose was established at 0.5 mg/lesion. Ongoing trials will further investigate its efficacy against various cancer types. Results were presented at the Society for Immunotherapy of Cancer annual meeting.

Repertoire Immune Medicines has appointed Torben Straight Nissen as the new interim CEO, shifting focus to its DECODE™ Platform aimed at developing immune medicines for cancer, autoimmune disorders, and infectious diseases. This transition follows the discontinuation of Phase 1 clinical studies for certain T cell therapeutic candidates. The company aims to leverage insights from previous studies while reducing operational needs, including a staff reduction. The DECODE platform enables the identification of key immune interactions, emphasizing its potential in innovative treatment solutions.

Ascendis Pharma (ASND) reported third-quarter 2022 revenue of €15.3 million, a significant rise from €1.1 million year-over-year, driven by SKYTROFA® sales of €12.3 million. R&D expenses rose to €97.4 million, reflecting a strategic focus on upcoming product launches, particularly TransCon PTH, which is under FDA Priority Review with a PDUFA date of April 30, 2023. The net loss for the quarter was €169.0 million or €3.03 per share. Despite increased operational costs, Ascendis ended the quarter with a robust cash position of €935.1 million, supporting ongoing development initiatives.

Ascendis Pharma A/S (Nasdaq: ASND) announced that the FDA has accepted its New Drug Application for TransCon PTH, targeting adults with hypoparathyroidism, under Priority Review. The decision has a PDUFA target action date of April 30, 2023. Ascendis also plans to submit a Marketing Authorisation Application to the EMA this quarter. Hypoparathyroidism affects approximately 200,000 patients in the U.S., Europe, and Japan, with conventional therapies being inadequate. The company aims to make TransCon PTH the first hormone replacement therapy for this condition.

Ascendis Pharma A/S (Nasdaq: ASND) will conduct a conference call and live webcast on November 2, 2022, at 4:30 p.m. ET to discuss its third quarter 2022 financial results and provide a business update. Interested participants can access the webcast through the provided links on the company's website, where a replay will be available for 30 days. Ascendis Pharma focuses on utilizing its innovative TransCon™ platform to develop new therapies aimed at making a positive impact on patients' lives.

Frazier Life Sciences has appointed Jan Møller Mikkelsen as a Senior Advisor, enhancing its leadership team. Mikkelsen, with over 30 years in the biopharmaceutical sector, is the founder and CEO of Ascendis Pharma (NASDAQ: ASND) and has held senior positions at several notable companies. Frazier Life Sciences, managing over $3.3 billion in capital, focuses on innovative biopharmaceutical investments, boasting 62 portfolio companies that have achieved IPOs or M&As since 2005.

Ascendis Pharma announced positive early results from the Phase 1/2 transcendIT-101 trial for TransCon TLR7/8 Agonist, showing it is well-tolerated with signs of clinical activity in advanced solid tumors. The recommended Phase 2 dose will be evaluated across four specific cancer cohorts, including melanoma and head and neck squamous-cell carcinoma. An abstract of the topline data will be presented at the SITC 2022 meeting in Boston on November 8-12. The trial aims to leverage the body’s immune system to improve cancer therapy outcomes.