GlobeNewswire Inc | Nov 11, 2020 04:01PM EST

November 11, 2020

European Medicines Agency (EMA) validatedthe Marketing Authorisation Application (MAA)for TransConTM hGH (lonapegsomatropin)in pediatric growth hormone deficiency (GHD)

Submitted regulatory filings to enable initiation ofEuropean and Canadian sites for phase 3 PaTHway Trial for TransCon PTH

In collaboration with VISEN Pharmaceuticals, filed an Investigational New Drug Application (IND) to initiate the phase 2 ACcomplisH China Trial of TransCon CNP

On track for remaining 2020 corporate milestone of filing an IND or similar for TransCon TLR7/8 Agonist by year-end

Conference call today at 4:30 p.m. Eastern Time

COPENHAGEN, Denmark, Nov. 11, 2020 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, today announced financial results for the quarter ended September 30, 2020.

As Ascendis progresses toward closing out 2020, I am proud that we are on track or ahead of schedule for all 2020 corporate goals and to achieve Vision 3x3, moving us one step closer to becoming a fully integrated, global biopharmaceutical company, said Jan Mikkelsen, President and CEO. As a company, we are driven by our core values to achieve our long-term strategic Vision 3x3, using the power of the TransCon technology. We push ourselves to continually deliver not only in endocrinology but also in oncology and to bring these products to patients as efficiently and safely as possible.

Corporate Highlights & Progress

-- TransCon hGH (lonapegsomatropin) Submitted MAA to the EMA, which has now validated the application. Filed a Clinical Trial Notification with the Pharmaceuticals and Medical Devices Agency in Japan to initiate the companys phase3 riGHt Trial of lonapegsomatropin for the treatment of pediatric GHD. Announced the U.S. Food and Drug Administration (FDA) accepted the companys Biologics License Application and set a PDUFA date for June 25, 2021. -- TransCon PTH Submitted regulatory filings to enable initiation of European and Canadian sites for phase 3 PaTHway Trial. Received orphan designation by the European Commission (EC) for the treatment of hypoparathyroidism (HP). Submitted an amendment to its IND with FDA for the PaTHway phase 3 clinical trial evaluating the safety, tolerability and efficacy of TransCon PTH in adults with HP. Announced preliminary six-month results from the open-label extension portion of PaTH Forward, a global phase 2 trial evaluating the safety, tolerability and efficacy of TransCon PTH in adult subjects with HP. The preliminary results support potential use of TransCon PTH as a hormone replacement therapy for HP and demonstrated normalization of quality of life. -- TransCon CNP In collaboration with VISEN Pharmaceuticals, filed an IND application to initiate the phase 2 ACcomplisH China Trial. Received orphan designation by the EC. -- Oncology On track for filing IND or similar for TransCon TLR7/8 Agonist by year-end. TransCon TLR7/8 Agonist is a long-acting prodrug of resiquimod. Administered as an intratumoral injection, TransCon TLR7/8 Agonist is designed to provide sustained release of unmodified resiquimod directly to the tumor. -- Announced Mark A. Bach, M.D., Ph.D., as Senior Vice President of Clinical Development and Medical Affairs for Endocrinology Rare Diseases. Dr. Bach has 30 years of experience in clinical research and development. -- Ended the third quarter 2020 with cash, cash equivalents and marketable securities totaling 957.5 million.

Third Quarter 2020 Financial Results

For the third quarter, Ascendis Pharma reported a net loss of 121.7 million, or 2.31 per share (basic and diluted) compared to a net loss of 25.1 million, or 0.53 per share (basic and diluted) for the same period in 2019.

Revenue for the third quarter was 2.8 million compared to 2.2 million in the same quarter of 2019. The increase was due to a higher sale of clinical supply and services to VISEN, partly offset by a lower amount of license revenue being recognized.

Research and development (R&D) costs for the third quarter were 64.1 million compared to 46.3 million during the same period in 2019. Higher R&D costs in 2020 reflect an increase in personnel-related costs, expansion of R&D facilities and the continued progress in development of the companys product candidates.

Selling, general and administrative (SG&A) expenses for the third quarter were 17.5 million compared to 10.0 million during the same period in 2019. The increase is primarily due to additional personnel-related costs, higher IT and other infrastructure costs and continued build of the companys commercial capabilities.

As of September 30, 2020, Ascendis Pharma had cash, cash equivalents and marketable securities totaling 957.5 million compared to 471.6 million as of June 30, 2020. As of September 30, 2020, Ascendis Pharma had 53,416,091 ordinary shares outstanding.

Conference Call and Webcast information

Ascendis Pharma will host a conference call and webcast today at 4:30 p.m. Eastern Time (ET) to discuss its third quarter 2020 financial results. Details include:

Date November 11, 2020Time 4:30 p.m. ETDial In (U.S.) 844-290-3904Dial In (International) 574-990-1036Access Code 8955314

A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will also be available on this website shortly after conclusion of the event for 30 days.

About Ascendis Pharmas Pipeline

Ascendis Pharma currently has three product candidates in clinical development in rare endocrine diseases:

-- TransCon hGH (lonapegsomatropin), an investigational long-acting prodrug of somatropin (human growth hormone or hGH) that releases somatropin with the identical amino acid sequence and size as daily growth hormone, is designed as a once-weekly treatment for GHD and is currently under regulatory review by the U.S. Food and Drug Administration and the European Medicines Agency. -- TransCon PTH, an investigational long-acting prodrug of parathyroid hormone (PTH) in phase 3 development as a once-daily replacement therapy for hypoparathyroidism (HP) designed to replace PTH at physiologic levels for 24 hours every day, and address both short-term symptoms and long-term complications of the disease. -- TransCon CNP, an investigational long-acting prodrug of C-type natriuretic peptide (CNP) in phase 2 development as a therapy for children with achondroplasia (ACH), the most common form of dwarfism, for which there is no FDA-approved treatment. TransCon CNP is designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose.

Additionally, the company has established oncology as its second therapeutic area of focus and plans to submit an IND or similar by year-end for TransCon TLR7/8 Agonist, the companys first oncology product candidate.

AboutAscendis Pharma A/S

Ascendis Pharmais applying its innovative TransCon technologies to build a leading, fully integrated biopharmaceutical company focused on making a meaningful difference in patients lives. Guided by its core values of patients, science and passion, the company utilizes its TransCon technologies to create new and potentially best-in-class therapies.

Ascendis Pharmacurrently has a pipeline of three independent endocrinology rare disease product candidates in clinical development and is advancing oncology as its second therapeutic area of focus. The company continues to expand into additional therapeutic areas to address unmet patient needs. Ascendis is headquartered inCopenhagen, Denmark, with additional offices in Heidelberg andBerlin, Germany, and inPalo AltoandRedwood City, California.

For more information, please visit www.ascendispharma.com.

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 2020 corporate goals and Vision 3x3, (ii) Ascendis PDUFA date for its Biologics License Application for lonapegsomatropin, (iii) Ascendis planned IND filing for TransCon TLR7/8 Agonist by year-end, (iv) Ascendis ability to apply its platform technology to build a leading, fully integrated biopharma company, (v) Ascendis product pipeline and expansion into additional therapeutic areas and (vi) Ascendis expectations regarding its ability to utilize 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: unforeseen safety or efficacy results in its oncology programs, lonapegsomatropin, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development and potential commercialization of its oncology programs, lonapegsomatropin, 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, lonapegsomatropin, 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 of 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 prospectus supplement filed onJuly 9, 2020and Ascendis current and future reports filed with, or submitted to, theU.S. Securities and Exchange Commission(SEC), including its Annual Report on Form 20-F filed with theSEConApril 3, 2020. Forward-looking statements do not reflect the potential impact of any future in-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, theAscendis Pharmalogo, the company logo and TransCon are trademarks owned by theAscendis Pharma Group. November 2020Ascendis Pharma A/S.

FINANCIAL TABLES FOLLOW

Ascendis Pharma A/SConsolidated Statements of Profit or Loss and Other Comprehensive Income /(loss)(In EUR'000s, except share and per share data) Three Months Ended Nine Months Ended September September 30, 30, 2020 2019 2020 2019 Revenue 2,757 2,243 6,418 10,868 Research and (64,059 ) (46,258 ) (185,152 ) (141,343 )development costsSelling, general andadministrative (17,523 ) (10,000 ) (56,243 ) (31,396 )expensesOperating profit / (78,825 ) (54,015 ) (234,977 ) (161,871 )(loss) Share of profit / (3,101 ) (1,338 ) (6,501 ) (5,452 )(loss) of associateFinance income 136 30,547 1,677 30,285 Finance expenses (39,970 ) (368 ) (40,391 ) (812 )Profit / (loss) (121,760 ) (25,174 ) (280,192 ) (137,850 )before tax Tax on profit / 19 61 202 196 (loss) for the periodNet profit / (loss) (121,741 ) (25,113 ) (279,990 ) (137,654 )for the period Attributable to (121,741 ) (25,113 ) (279,990 ) (137,654 )owners of the Company Basic and dilutedearnings / (loss) per ? (2.31 ) ? (0.53 ) ? (5.64 ) ? (2.99 )share Number of shares usedfor calculation 52,715,204 47,590,837 49,647,471 46,066,493 (basic and diluted) Net profit / (loss) (121,741 ) (25,113 ) (279,990 ) (137,654 )for the periodOther comprehensive income / (loss)Items that may bereclassified subsequently toprofit or loss:Exchange differenceson translating (75 ) 37 (136 ) 2 foreign operationsOther comprehensiveincome / (loss) for (75 ) 37 (136 ) 2 the period, net oftax Total comprehensiveincome / (loss) for (121,816 ) (25,076 ) (280,126 ) (137,652 )the period, net oftax Attributable to (121,816 ) (25,076 ) (280,126 ) (137,652 )owners of the Company

Ascendis Pharma A/SConsolidated Statements of Financial Position(In EUR'000s) September 30, December 31, 2020 2019Assets Non-current assets Intangible assets 4,653 3,495 Property, plant and equipment 47,325 45,069 Investment in associate 11,574 15,538 Deposits 1,243 1,463 Marketable securities 27,728 - 92,523 65,565 Current assets Trade receivables 2,722 804 Other receivables 4,511 3,136 Prepayments 15,266 7,648 Income taxes receivable 1,305 1,473 Marketable securities 166,343 - Cash and cash equivalents 763,454 598,106 953,601 611,167 Total assets 1,046,124 676,732 Equity and liabilities Equity Share capital 7,172 6,443 Distributable equity 944,413 590,671 Total equity 951,585 597,114 Non-current liabilities Lease liabilities 26,952 30,720 Other payables 3,160 908 30,112 31,628 Current liabilities Lease liabilities 6,328 5,899 Contract liabilities 223 858 Trade payables and accrued expenses 38,108 27,765 Other payables 19,549 13,349 Income taxes payable 219 119 64,427 47,990 Total liabilities 94,539 79,618 Total equity and liabilities 1,046,124 676,732

Investor contacts: Media contact:Tim Lee Ron RogersAscendis Pharma Ascendis Pharma(650) 374-6343 (650) 507-5208tle@ascendispharma.com rrs@ascendispharma.com Patti Bank Westwicke Partners (415) 513-1284 patti.bank@westwicke.com ir@ascendispharma.com