Ascendis Pharma A/S ASND released topline data on Monday from a trial that included 84 children with achondroplasia, a genetic disorder that causes dwarfism and disproportionate short stature.TransCon CNP (navepegritide), an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to provide sustained release and continuous exposure of active CNP.CNP regulates blood flow, blood pressure, and the reactivity of platelets and leukocytes.Also Read: Ascendis Pharma Faces ...

–   U.S. FDA Orphan Drug exclusivity provides seven years of market exclusivity for YORVIPATH in the United States for the treatment of hypoparathyroidism in adults COPENHAGEN, Denmark, Sept. 11, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the United States Food & Drug Administration (FDA) has granted Orphan Drug exclusivity to YORVIPATH® (palopegteriparatide, developed as TransCon PTH), providing seven years of market exclusivity for YORVIPATH in the United States for t ...

magical_light/iStock via Getty Images Ascendis Pharma (ASND) shocked investors with a big Q2 revenue miss, driven by a large net price cut on Skytrofa that management says was necessary to ensure broader access to the drug. The company did not share the exact price cut, but we can calculate from Q2 demand and adjusted net sales numbers that it is approximately 35%. I am not sure how much of the price cut Ascendis can make up with increased patient numbers, but I am sure that the loss of peak sales on Sk ...

Tuesday, Ascendis Pharma A/S ASND reported second-quarter sales of $38.76 million (35.9 million euros), missing the consensus of $92.7 million, down from 47.4 million euros reported a year ago.Cantor Fitzgerald writes that the Skytrofa update probably overshadowed another key update from the call: the U.S. pricing for Yorvipath, which came out at $285,000/patient/year, much higher than our expectation of $160-190K.The analyst notes that due to the lower Skytrofa guidance, the company’s cash flow breakeven t ...

A bottom-line whiff and reduced guidance made the company an outlier for the wrong reasons on Hump Day.An earnings miss and sharply reduced guidance for its ONE commercialized product doomed Ascendis Pharma (ASND -11.27%) to a bad Wednesday on the stock market. The biotech's shares fell by more than 11% in price after it divulged its latest set of quarterly results. By comparison, the bellwether S&P 500 index suffered a relatively modest drop of 0.2%.Declines and misses in the second quarterAscendis, a biot ...

Tuesday, Ascendis Pharma A/S ASND reported second-quarter sales of $38.76 million (35.9 million euros), missing the consensus of $92.7 million, down from 47.4 million euros reported a year ago.Results in the quarter were primarily impacted by a negative adjustment to prior periods' estimates and assumptions for sales deductions of 27.1 million euros, where 19.5 million euros and 7.6 million euros were attributable to the three months ended March 31, 2024, and periods before January 1, 2024, respectively.Als ...

Financial Data and Key Metrics Changes - In Q2 2024, SKYTROFA volume more than doubled compared to Q2 2023, but reported revenue decreased by 27% year-over-year to EUR 26.2 million due to higher sales deductions and adjustments to prior sales accruals [22][24] - Total revenue for Q2 2024 was EUR 36 million, including EUR 4.6 million from services and license revenue [24] - R&D costs decreased by 21% to EUR 83.5 million in Q2 2024 compared to EUR 105 million in Q2 2023, while SG&A expenses increased to EUR 74.3 million from EUR 70.3 million [25] - Net finance income increased to EUR 29.4 million in Q2 2024 from EUR 26.4 million in Q2 2023 [26] Business Line Data and Key Metrics Changes - YORVIPATH revenue for Q2 2024 was EUR 5.2 million, reflecting the first full quarter of commercial revenue in Germany and Austria, up from EUR 1.5 million in Q1 2024 [24] - SKYTROFA revenue for the first half of 2024 totaled EUR 91.2 million, a 35% year-over-year increase compared to EUR 67.4 million in the same period in 2023 [23] - The number of patients on YORVIPATH treatment in Germany and Austria increased to over 250, with a retention rate of 98% [14][42] Market Data and Key Metrics Changes - The US growth hormone market is estimated to be around $1.4 billion, with SKYTROFA positioned as a premium product with a net value per patient approximately three times that of daily growth hormone [30] - By the end of Q2 2024, SKYTROFA penetration in the US pediatric growth hormone deficiency treated patient population was 18% [23] Company Strategy and Development Direction - The company aims to achieve approval for all three endocrinology rare disease product candidates by the end of 2025, with a focus on expanding into larger therapeutic areas such as oncology and obesity [10][11] - The strategy for SKYTROFA includes simplifying market access and expanding the product label to include adult growth hormone deficiency [15][17] Management Comments on Operating Environment and Future Outlook - Management expressed optimism about the transformative impact of YORVIPATH for patients with hypoparathyroidism and highlighted the strong retention rates for the product [21][42] - The company expects to achieve operating cash flow breakeven on a quarterly basis in 2024 or 2025, depending on the launch timing of YORVIPATH in the US [27] Other Important Information - The company announced a new capped synthetic royalty funding agreement with Royalty Pharma for $150 million in exchange for a 3% royalty on net sales of YORVIPATH in the US [26] - The company plans to submit a supplemental BLA for adult growth hormone deficiency for SKYTROFA in Q3 2024 [17] Q&A Session Summary Question: How does the company project getting SKYTROFA to a blockbuster in the US? - Management indicated that the US growth hormone market is about $1.4 billion and SKYTROFA has a net value per patient three times that of daily growth hormone, with plans for label expansion [30][31] Question: What are the competitive dynamics regarding SKYTROFA's pricing? - Management acknowledged that there are two other long-acting products in the US market but emphasized SKYTROFA's best-in-class potential and strong market position [34][35] Question: What feedback has been received from payers regarding YORVIPATH pricing? - Management reported no negative feedback on the responsible pricing strategy for YORVIPATH, which aims to support all patients with hypoparathyroidism [39] Question: How is the penetration of YORVIPATH tracking in Germany and Austria? - Management noted that the uptake is strong, with around 125 prescribers and a retention rate of 98%, indicating a positive trajectory for the product [41][42] Question: What assumptions led to the lower guidance for SKYTROFA? - Management explained that the guidance was based on observed growth in volume and the reset of market access, with expectations for continued growth in the second half of the year [48][50] Question: What is the expected run rate for SKYTROFA moving into Q3? - Management indicated that the reported net sales for Q2, adjusted for true-ups, would provide a good basis for expectations in Q3 and Q4 [81]

COPENHAGEN, Denmark and NEW YORK, Sept. 03, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) and Royalty Pharma plc (Nasdaq: RPRX) today announced that Ascendis Pharma Bone Diseases A/S, a wholly-owned subsidiary of Ascendis Pharma A/S, has entered into a $150 million capped synthetic royalty funding agreement with Royalty Pharma based on U.S. net sales of YORVIPATH. “We are pleased to again partner with Royalty Pharma, a leading funder of innovation across the biopharma industry, as we launch YO ...

Core Insights - Ascendis Pharma has received FDA approval for YORVIPATH, the first and only treatment for hypoparathyroidism in adults, highlighting the company's commitment to addressing unmet medical needs [2][4] - The company reported a second quarter revenue of €36 million, a decrease from €47.4 million in the same period last year, primarily due to negative adjustments in sales deductions [9][10] - SKYTROFA revenue for Q2 2024 was €26 million, reflecting a 27% year-over-year decrease, although there was a 134% increase in volume [3][9] - The company anticipates full-year 2024 SKYTROFA revenue to be between €220 million and €240 million [8] Financial Performance - Total revenue for Q2 2024 was €36 million, down from €47.4 million in Q2 2023, impacted by a negative adjustment of €27.1 million in sales deductions [9][10] - YORVIPATH generated €5.2 million in revenue during its first full quarter of commercial launch in Germany and Austria [4] - Research and development costs for Q2 2024 were €83.5 million, a decrease from €105 million in Q2 2023, attributed to lower external development costs [11][12] - The net loss for Q2 2024 was €109.4 million, or €1.91 per share, compared to a net loss of €121.4 million, or €2.16 per share, in Q2 2023 [12][19] Product Development and Pipeline - The company is preparing for the U.S. launch of YORVIPATH and expects initial supply to be available in Q1 2025, with potential earlier introduction in Q4 2024 [4][8] - Topline results from the pivotal ApproaCH Trial for TransCon CNP are expected in the coming weeks, with plans to submit a New Drug Application to the FDA for children with achondroplasia in Q1 2025 [5][6] - Ascendis Pharma is on track to submit a supplemental Biologics License Application for adult growth hormone deficiency for SKYTROFA in Q3 2024 [3][8] Market Position and Strategy - Ascendis Pharma's focus on rare endocrine diseases is underscored by the approval of two out of three of its TransCon product candidates, positioning the company for potential blockbuster status in the U.S. market [2][4] - The company has entered into a $150 million capped synthetic royalty funding agreement with Royalty Pharma related to YORVIPATH sales in the U.S. [8]

COPENHAGEN, Denmark, Aug. 28, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it will report second quarter 2024 financial results and provide a business update on Tuesday, September 3, 2024, after the close of the U.S. financial markets. Ascendis Pharma will also host a conference call and live webcast on September 3, 2024, at 4:30 p.m. Eastern Time (ET) to discuss its second quarter 2024 financial results. Those who would like to participate may access the live webcast here, or ...