Ascendis Pharma Launches 2nd TransCon™ Product: YORVIPATH® Now Available in Germany and Austria for Adults with Chronic Hypoparathyroidism

Rhea-AI Summary

Ascendis Pharma A/S (Nasdaq: ASND) has announced the availability of YORVIPATH® (palopegteriparatide, developed as TransCon PTH), a parathyroid hormone (PTH) replacement therapy, in Germany and Austria. This marks the second product developed with Ascendis Pharma’s TransCon technology platform to be launched commercially. The company aims to achieve regulatory approvals for three Endocrinology Rare Disease products by 2025 and aims for each to achieve blockbuster status by 2030. With an expanding commercial infrastructure, Ascendis Pharma is well positioned to meet the needs of physicians and patients worldwide.

Positive

• YORVIPATH is now available in Germany and Austria
• Second product developed with TransCon technology platform to be launched commercially
• Company aims to achieve regulatory approvals for three Endocrinology Rare Disease products by 2025
• Company aims for each product to achieve blockbuster status by 2030
• Expanding commercial infrastructure in place

Negative

• None.

Pharmaceutical Market Analyst

The commercial launch of YORVIPATH in Germany and Austria represents a significant strategic move for Ascendis Pharma, particularly in the context of its broader Vision 3x3 and Vision 2030 goals. The product's approval and subsequent availability in these markets not only serve to meet a significant unmet medical need within the hypoparathyroidism community but also signal the company's commitment to expanding its portfolio of endocrinology rare disease treatments.

From a market perspective, the launch could potentially increase Ascendis Pharma's market share in the hormone replacement therapy sector. Given that YORVIPATH is the second product developed using the company's proprietary TransCon technology, its performance will also be indicative of the platform's commercial viability. The success of YORVIPATH could encourage further investment in TransCon technology, potentially leading to a diversified product pipeline that may offer long-term revenue streams.

However, the commercial success of YORVIPATH will depend on various factors including market acceptance, pricing strategy, reimbursement policies and the effectiveness of the commercial infrastructure in place. Additionally, the pursuit of 'blockbuster' status by 2030 sets high expectations for revenue generation, which will require careful monitoring of the product's market penetration and competitive positioning.

Medical Research Analyst

YORVIPATH (palopegteriparatide) is a notable development in the treatment of chronic hypoparathyroidism, a condition characterized by insufficient levels of parathyroid hormone (PTH), which is critical for calcium and phosphorus metabolism. Current treatment options are limited and often involve high doses of calcium supplements and active vitamin D, which can lead to complications and do not address the underlying PTH deficiency.

The availability of a PTH replacement therapy such as YORVIPATH offers a more physiological treatment approach, potentially improving patient outcomes and quality of life. This could lead to increased demand and a shift in treatment standards within the endocrinology field. It's important to note that the clinical benefits of YORVIPATH will be scrutinized by the medical community and long-term studies will be essential to validate its efficacy and safety profile.

For stakeholders, the implications are twofold: there is the potential for improved patient management and for Ascendis Pharma, a stronger foothold in the rare disease market. However, the long-term impact will hinge on sustained clinical success and the ability to navigate the complex reimbursement landscape in different countries.

Healthcare Economist

The introduction of YORVIPATH in the European market is a key event to observe from a health economics standpoint. Chronic hypoparathyroidism, while rare, can lead to substantial economic burden due to its chronic nature and the need for ongoing management. The direct costs associated with traditional treatments, as well as the indirect costs stemming from patient morbidity, can be significant.

YORVIPATH's ability to provide a more targeted PTH replacement therapy could potentially reduce these costs by minimizing complications and the need for multiple medications. Moreover, if YORVIPATH can demonstrate cost-effectiveness by reducing long-term healthcare resource utilization, it could gain favor among payers and healthcare providers, which is crucial in securing market access and favorable reimbursement.

However, the pricing strategy will be a critical factor in determining its economic impact. High costs could limit access and create barriers to adoption, particularly if the incremental benefits do not justify the price differential over existing therapies. It will be essential to balance the need for a return on investment with the broader goal of ensuring patient access to innovative treatments.

COPENHAGEN, Denmark, Jan. 31, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that YORVIPATH^® (palopegteriparatide, developed as TransCon PTH), a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism, is now available in Germany and Austria. YORVIPATH is the second product developed with Ascendis Pharma’s TransCon technology platform to be launched commercially.

“We are pleased to initiate the launch of YORVIPATH to address the needs expressed within the hypoparathyroidism community for a new PTH-based treatment option,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “In addition to addressing a significant unmet medical need, this launch of our second TransCon product represents a key milestone in our goal to launch the three Endocrinology Rare Disease products for which, under Vision 3x3 we target achieving regulatory approvals by 2025 and for each of which, under Vision 2030, we aim to achieve blockbuster status by 2030. With an increasingly broad commercial infrastructure in place, we are well positioned to accomplish this by expanding the availability of YORVIPATH and our other approved products to meet the needs of physicians and patients around the world.”

About YORVIPATH
YORVIPATH (palopegteriparatide, developed as TransCon PTH) is a prodrug of parathyroid hormone (PTH 1-34) administered once daily, designed to provide parathyroid hormone levels within the normal physiological range across the 24-hour dosing period. YORVIPATH was granted marketing authorization by the European Commission (EC) in November 2023 as a PTH replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. A decision on YORVIPATH in the United Kingdom is expected from the Medicines & Healthcare Products Regulatory Agency (MHRA) during the first quarter of 2024. In the United States, the U.S. Food & Drug Administration (FDA) has set a PDUFA date of May 14, 2024 to complete their review of Ascendis Pharma’s New Drug Application for TransCon PTH for adults with chronic hypoparathyroidism. TransCon PTH is also in development in Japan through Teijin Ltd. and China through VISEN Pharmaceuticals. Internationally, Ascendis is working to expand the global reach of its Endocrinology Rare Disease portfolio, including YORVIPATH, through exclusive distribution agreements, three of which have been established to date.

About Hypoparathyroidism
Hypoparathyroidism is an endocrine disease caused by insufficient levels of PTH, the primary regulator of calcium/phosphate balance in the body, acting directly on bone and kidneys and indirectly on intestines. Hypoparathyroidism is considered chronic if it persists >6 months following surgery per the 2016 Endocrine Society Guidelines, 2019 Canadian and International Consensus Statement, and 2022 European Society of Endocrinology Consensus Statement. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%); other etiologies include autoimmune disorders, familial disorders, and idiopathic causes.

About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit https://ascendispharma.com to learn more.

Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) YORVIPATH’s ability to address the need for a new PTH-based treatment option, (ii) Ascendis’ goal of obtaining approval for, launching and achieving blockbuster status for three Endocrinology Rare Disease products by 2030, (iii) Ascendis’ ability to expand the availability of YORVIPATH and its other approved products, (iv) the timing of the MHRA’s decision on YORVIPATH in the United Kingdom, (v) the FDA’s PDUFA date for TransCon PTH, (vi) Ascendis’ plan to expand the global reach of its Endocrinology Rare Disease portfolio, including YORVIPATH, through exclusive distribution agreements, (vii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (viii) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including inflation, the effects on its business from the worldwide COVID-19 pandemic and ongoing conflicts such as that in the region surrounding Ukraine and Russia. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 16, 2023 and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.

Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and YORVIPATH^® are trademarks owned by the Ascendis Pharma group. © January 2024 Ascendis Pharma A/S.

Investor Contacts: Tim Lee Ascendis Pharma +1 (650) 374-6343 tle@ascendispharma.com ir@ascendispharma.com	Media Contact: Melinda Baker Ascendis Pharma +1 (650) 709-8875 media@ascendispharma.com
Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com

What is the name of the parathyroid hormone replacement therapy announced by Ascendis Pharma A/S?

The parathyroid hormone replacement therapy announced by Ascendis Pharma A/S is YORVIPATH® (palopegteriparatide, developed as TransCon PTH).

Where is YORVIPATH now available?

YORVIPATH is now available in Germany and Austria.

What is the ticker symbol for Ascendis Pharma A/S?

The ticker symbol for Ascendis Pharma A/S is ASND.

What are the company's goals for regulatory approvals and blockbuster status for its products?

The company aims to achieve regulatory approvals for three Endocrinology Rare Disease products by 2025 and aims for each to achieve blockbuster status by 2030.

What infrastructure is Ascendis Pharma A/S expanding?

Ascendis Pharma A/S is expanding its commercial infrastructure.