Ascendis Pharma A/S Announces Two Oral Presentations Highlighting the Potential for TransCon™ PTH to be a Replacement Therapy for Adult Hypoparathyroidism at ENDO 2022

Rhea-AI Summary

Ascendis Pharma A/S (Nasdaq: ASND) announced significant advancements in its investigational product candidate TransCon PTH for adult hypoparathyroidism, showcasing Phase 2 and Phase 3 trial data at ENDO 2022. Dr. Aliya Khan reported that the PaTHway Trial highlighted a majority of treated patients achieving disease control, including normalized serum calcium and independence from conventional therapy. The treatment was well-tolerated. These presentations aim to reshape the treatment paradigm for hypoparathyroidism and underline Ascendis' commitment to innovative therapies.

Positive

• Successful Phase 3 PaTHway Trial for TransCon PTH demonstrated the majority of patients achieved normalization of serum calcium.
• TransCon PTH-treated patients exhibited mean decrease in 24-hour urine calcium excretion to normal levels.
• No discontinuations related to TransCon PTH during the trial, indicating high tolerability.

Negative

• None.

–  Late-breaker oral presentation of TransCon PTH Phase 3 PaTHway Trial data in adult hypoparathyroidism on Tuesday, June 14

–  Oral presentation of TransCon PTH Phase 2 PaTH Forward Trial Open-Label Extension Data in adult hypoparathyroidism on Monday, June 13

–  PaTHway Trial results represent the second consecutive successful pivotal Phase 3 trial of a TransCon product candidate

COPENHAGEN, Denmark, June 07, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that its presentations at ENDO 2022 will include oral presentations of Phase 2 and Phase 3 data for its investigational product candidate TransCon PTH in adult hypoparathyroidism (HP). During the meeting, which will be held in-person and virtually June 11-14 in Atlanta, Dr. Aliya Khan, M.D., Clinical Professor of Medicine at McMaster University and Director of the Calcium Disorders Clinic at McMaster University Medical Center, will give a late-breaker oral presentation of Phase 3 PaTHway Trial data for TransCon PTH in adult HP.

“This is the first Phase 3 trial in adult hypoparathyroidism in which the majority of treated patients achieved real control of their disease – that is, normalization of serum calcium, independence from conventional therapy, and a more normal patient-reported quality of life,” said Dr. Khan. “Additionally, TransCon PTH was generally well-tolerated with no discontinuations related to study drug, and TransCon PTH-treated patients showed a mean decrease in 24-hour urine calcium excretion into the normal range. I am hopeful that, with these encouraging results, we are closer to changing the treatment paradigm for patients living with this under-recognized and often debilitating disease.”

An additional Ascendis oral presentation at ENDO 2022 will include a review of Phase 2 TransCon PTH long-term data by Dr. Bart Clarke, Professor of Endocrinology at Mayo Clinic, showing durable benefit for adult hypoparathyroidism patients treated with TransCon PTH through Week 84 of the Pathway Trial. Additionally, Ascendis posters will present new data showing the continued safety and efficacy of once-weekly TransCon hGH in children with growth hormone deficiency treated for 2.5 years in the EnliGHten Trial, and, for adults with growth hormone deficiency, new research showing low treatment rates, increased medical risks, and higher healthcare costs.

Ascendis will also host a Medical Affairs booth (#1515) at ENDO 2022, as well as a separate booth (#1415) and events showcasing SKYTROFA^® (lonapegsomatropin-tcgd), the company’s once-weekly treatment for pediatric growth hormone deficiency.

“ENDO is one of the largest endocrine health symposiums, and we are especially pleased this year to be providing a comprehensive review of data from our second consecutive successful Phase 3 trial,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Ascendis is deeply committed to developing products that deliver value for patients, payers and society, and we are honored to be able to partner with patients, physicians, and other specialists in our work to advance understanding and treatment of rare endocrine diseases.”

Ascendis Pharma presentations and educational sessions at ENDO 2022 will include:

	Hypoparathyroidism
	Tuesday, June 14 9:45 – 11:15am		Late-Breaker Phase 3 PaTHway Trial: Participants Treated with TransCon PTH Achieved Independence from Conventional Therapy While Maintaining Normal Serum Calcium Presented by: Aliya Khan, M.D.
	Monday, June 13 11:30am – 1:00pm		Oral Presentation The PaTH Forward Trial: Efficacy and Safety of TransCon PTH Through Week 84 for Adults with Hypoparathyroidism Presented by: Bart Clarke, M.D.
	Saturday, June 11 1:00 – 2:00pm Product Theater #2		Education Managing Patients with Hypoparathyroidism: A Healthcare Professional and Patient Partnership Speaker: John P. Bilezikian, M.D.
	Growth Hormone Deficiency
	Monday, June 13 12:30 – 2:30pm On-site and on-demand for virtual attendees		Rapid Fire e-Poster Safety and Efficacy of Treatment with Lonapegsomatropin in Children with Growth Hormone Deficiency at Week 130 in the EnliGHten Trial Presented by: Paul Saenger, M.D.
	On-site and on-demand for virtual attendees		Poster Plus Economic Burden of Growth Hormone Deficiency in a U.S. Adult Population Authored by: Alden Smith, Janna Manjelievskaia, et al
	Saturday, June 11 10:15 – 11:15am Product Theater #3		Education SKYTROFA (Lonapegsomatropin-tcgd): The First FDA-Approved Once-Weekly Treatment for Pediatric Growth Hormone Deficiency Speaker: Aristides Maniatis, M.D.

About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative platform technology 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, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Heidelberg and Berlin, Germany; Palo Alto and Redwood City, California; and Princeton, New Jersey. Please visit www.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) Ascendis’ expectations regarding the potential for TransCon PTH to be a replacement therapy for adult hypoparathyroidism, (ii) Ascendis’ ability to apply its platform technology to build a leading, fully integrated biopharma company, and (iii) 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 and distributors to supply TransCon hGH, the SKYTROFA^® Auto-Injector and other study drug for commercial sales in the U.S. and clinical studies; unforeseen safety or efficacy results in its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to commercialization of lonapegsomatropin-tcgd in the U.S., the co-pay program, and the further development of TransCon hGH, expenses related to the development and potential commercialization of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs, selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; dependence on third party manufacturers to supply study drug for planned clinical studies; Ascendis’ ability to obtain additional funding, if needed, to support its business activities and the effects on its business from the worldwide COVID-19 pandemic. 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 March 2, 2022 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 SKYTROFA are trademarks owned by the Ascendis Pharma Group. © June 2022 Ascendis Pharma A/S.

Investor Contacts: Tim Lee  Ascendis Pharma  +1 (650) 374-6343  tle@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 ir@ascendispharma.com

FAQ

What were the results of the Phase 3 PaTHway Trial conducted by Ascendis Pharma?

The Phase 3 PaTHway Trial showed that the majority of adult patients with hypoparathyroidism achieved normalized serum calcium levels and independence from conventional therapy.

When were the presentations for TransCon PTH conducted at ENDO 2022?

Oral presentations for TransCon PTH were held on June 13 and June 14, 2022, during the ENDO 2022 conference.

What is TransCon PTH and its significance in adult hypoparathyroidism?

TransCon PTH is an investigational product candidate aimed at treating adult hypoparathyroidism, and the recent trials indicate it may change the treatment paradigm for patients.

Who presented the findings of the PaTHway Trial at ENDO 2022?

Dr. Aliya Khan from McMaster University presented the late-breaker findings of the PaTHway Trial at ENDO 2022.

What does the successful Phase 3 trial mean for Ascendis Pharma's future?

The success of the Phase 3 trial may position Ascendis Pharma as a leader in treating hypoparathyroidism, indicating potential market growth and heightened investor interest.