Ascendis Pharma(ASND) - 2021 Q4 - Annual Report

Executive Summary & Business Update Company Introduction & Vision Ascendis Pharma aims to become a viable, sustainable, and profitable biopharmaceutical company, targeting a US$10 billion global market potential in endocrinology rare diseases - Ascendis Pharma's CEO, Jan Mikkelsen, stated that 2021 was an extraordinary year and 2022 is expected to be even stronger, attributing this to the regulatory approvals of TransCon hGH in the U.S. and Europe[1] - The company is progressing towards its Vision 3x3, aiming to become a viable, sustainable, and profitable biopharmaceutical company[2] - Ascendis Pharma estimates its first therapeutic area of endocrinology rare disease alone represents a combined US$10 billion global market potential[2] Corporate 2021 Highlights & Anticipated 2022 Milestones Ascendis Pharma achieved significant 2021 milestones and outlined key 2022 expectations across its TransCon pipeline, including commercial launches, regulatory authorizations, and clinical trial advancements TransCon hGH (SKYTROFA®) SKYTROFA® launched commercially in the U.S. in Fall 2021 and received EU marketing authorization in January 2022, with a Turner Syndrome protocol submission planned for Q2 2022 - SKYTROFA® (lonapegsomatropin-tcgd) was commercially launched in the U.S. in Fall 2021 as the only FDA-approved once-weekly treatment for pediatric growth hormone deficiency[3] - SKYTROFA® Prescriptions (as of Feb 28, 2022) | Metric | Value | | :-------------------------------------- | :---- | | SKYTROFA® Prescriptions Written | 708 | | Number of Prescribers | ~263 | | Prescribers writing for multiple patients | 44% | - In January 2022, the Company received marketing authorization for Lonapegsomatropin Ascendis Pharma in the European Union for the treatment of children and adolescents with growth failure due to insufficient endogenous growth hormone[3] - Ascendis Pharma plans to submit a protocol to the FDA in Q2 2022 to evaluate TransCon hGH for Turner Syndrome, expecting to evaluate higher doses compared to pediatric or adult GHD[4] - The ongoing conflict in Ukraine and Russia may affect timelines for the foresiGHt Trial (Phase 3 trial in adult growth hormone deficiency), but currently has no material business impact[4] TransCon PTH TransCon PTH demonstrated stable serum and urinary calcium levels in Phase 2, with Phase 3 top-line results expected in Q1 2022, leading to planned NDA and MAA submissions - Week-84 data from the Phase 2 PaTH Forward Trial showed subjects treated with TransCon PTH maintained stable and normal mean serum calcium levels and urinary calcium excretion, with all subjects free from active vitamin D and 93% taking less than 600 mg/day of calcium supplements[5] - Top-line results from PaTHway, a Phase 3 randomized, double-blind placebo-controlled clinical trial for TransCon PTH in adults with HP, are expected in the first quarter of 2022[5] - If Phase 3 trial results are positive, Ascendis Pharma plans to submit a New Drug Application (NDA) to the FDA in Q3 2022 and a Marketing Authorisation Application (MAA) to the European Medicines Agency in Q4 2022[6] - Top-line results from PaTHway Japan, a single-arm Phase 3 trial, are expected in Q3 2022, and initiation of a pediatric HP program is planned for Q4 2022[6] TransCon CNP Enrollment for the Phase 2 ACcomplisH Trial was completed in December 2021, with top-line data anticipated in Q4 2022, and an IND application for infants planned for Q2 2022 - Enrollment in the Phase 2 ACcomplisH Trial for TransCon CNP in achondroplasia (ages 2–10) was completed in December 2021, with 42% of enrolled subjects between 2 and 5 years old[6] - Top-line data from the ACcomplisH Trial are expected in the fourth quarter of 2022[6] - The Company plans to file an Investigational New Drug (IND) application or similar for the ACcomplisH Infants Trial (ages 0–2) during the second quarter of 2022[7] Oncology Pipeline (TransCon TLR7/8 Agonist & TransCon IL-2 ß/g) Ascendis Pharma's oncology pipeline showed early clinical activity, with top-line data for monotherapy and combo-therapy dose escalation expected in Q3 and Q4 2022, and a Phase 2 cohort expansion IND planned for Q4 2022 - Initial first-in-human results from transcendIT-101 (Phase 1/2 study of TransCon TLR7/8 Agonist) demonstrated early signs of clinical activity in three out of three efficacy-evaluable cancer patients treated as monotherapy or in combination with pembrolizumab[7] - Top-line data from monotherapy and combo-therapy dose escalation for TransCon TLR7/8 Agonist are expected in the third quarter of 2022[8] - The IL-ßeliege Trial (Phase 1/2 clinical trial for TransCon IL-2 ß/g in advanced cancer) was initiated in Q3 2021, with top-line monotherapy data expected in Q4 2022[8] - Ascendis Pharma plans to submit an IND or similar for Phase 2 cohort expansion for the combination of TransCon TLR7/8 Agonist and TransCon IL-2 ß/g during the fourth quarter of 2022[8] Corporate Governance & Financial Position Update Rafaèle Tordjman was elected to the board in November 2021, and the company concluded 2021 with a strong cash position of €789.6 million - Rafaèle Tordjman, Founder & CEO of Jeito Capital, was elected to Ascendis Pharma's board in November 2021[8] - Cash Position (as of Dec 31, 2021) | Metric | Amount | | :---------------------------------------- | :------------- | | Cash, cash equivalents, and marketable securities | €789.6 million | Full Year 2021 Financial Performance Overview of Financial Results Ascendis Pharma reported a net loss of €383.6 million for 2021, an improvement from 2020, with increased revenue driven by SKYTROFA sales and higher R&D and SG&A expenses - Full Year Financial Highlights (EUR millions) | Financial Metric | 2021 | 2020 | Change (YoY) | | :--------------- | :------ | :------ | :----------- | | Total Revenue | 7.8 | 7.0 | +11.4% | | R&D Costs | 295.9 | 260.9 | +13.4% | | SG&A Expenses | 160.2 | 76.7 | +108.9% | | Net Profit / (Loss) of Associate | 12.0 | (9.5) | N/A | | Net Finance Income / (Expense) | 55.8 | (79.0) | N/A | | Net Loss | (383.6) | (419.0) | -8.4% | | EPS (Basic & Diluted) | (7.00) | (8.28) | -15.5% | | Cash, Cash Equivalents, & Marketable Securities (Dec 31) | 789.6 | 834.1 | -5.3% | - Revenues for 2021 include U.S. SKYTROFA sales, and license, clinical supply and services provided to third parties, primarily VISEN Pharmaceuticals[10] - Higher R&D costs in 2021 reflect continued advancement of development programs and an increase in personnel-related costs to support them[10] - Higher SG&A expenses were primarily due to an increase in commercial and administrative personnel as well as an increase in commercial and IT costs[10] - The net profit of associate for 2021 included a non-cash gain of €42.3 million as a result of a financing round in VISEN[11] Consolidated Statements of Profit or Loss and Comprehensive Income / (loss) The consolidated statement shows a net loss of €383.6 million for 2021, an improvement from €419.0 million in 2020, despite increased operating loss, due to positive finance income and associate profit - Consolidated Statements of Profit or Loss and Comprehensive Income / (loss) (EUR'000s) | Metric | Year Ended Dec 31, 2021 | Year Ended Dec 31, 2020 | | :------------------------------------------------------ | :---------------------- | :---------------------- | | Revenue | 7,778 | 6,953 | | Cost of sales | 3,523 | - | | Gross profit | 4,255 | 6,953 | | Research and development costs | 295,867 | 260,904 | | Selling, general and administrative expenses | 160,180 | 76,669 | | Operating profit / (loss) | (451,792) | (330,620) | | Share of profit / (loss) of associate | 12,041 | (9,524) | | Finance income | 59,718 | 1,812 | | Finance expenses | 3,911 | 80,842 | | Profit / (loss) before tax | (383,944) | (419,174) | | Tax on profit / (loss) for the year | 367 | 219 | | Net profit / (loss) for the year | (383,577) | (418,955) | | Attributable to owners of the Company | (383,577) | (418,955) | | Other comprehensive income / (loss) (net of tax) | 3,855 | (42) | | Total comprehensive income / (loss) for the year, net of tax | (379,722) | (418,997) | Consolidated Statements of Financial Position Total assets increased to €1,084.9 million in 2021 from €979.8 million in 2020, driven by growth in property, plant and equipment, investment in associate, inventories, and marketable securities - Consolidated Statements of Financial Position (EUR'000s) | Metric | Dec 31, 2021 | Dec 31, 2020 | | :------------------------------------------ | :------------ | :------------ | | Assets | | | | Non-current assets | 279,035 | 239,660 | | Intangible assets | 5,272 | 5,717 | | Property, plant and equipment | 126,049 | 108,112 | | Investment in associate | 38,345 | 9,176 | | Other receivables (non-current) | 1,808 | 1,375 | | Marketable securities (non-current) | 107,561 | 115,280 | | Current assets | 805,886 | 740,133 | | Inventories | 75,405 | - | | Trade receivables | 2,200 | 387 | | Income tax receivable | 893 | - | | Other receivables (current) | 20,093 | 6,957 | | Prepayments | 25,231 | 13,994 | | Marketable securities (current) | 235,797 | 134,278 | | Cash and cash equivalents | 446,267 | 584,517 | | Total assets | 1,084,921 | 979,793 | | Equity and liabilities | | | | Equity | 883,635 | 838,711 | | Share capital | 7,646 | 7,217 | | Distributable equity | 875,989 | 831,494 | | Non-current liabilities | 100,930 | 88,278 | | Lease liabilities (non-current) | 97,966 | 85,116 | | Contract liabilities (non-current) | 2,964 | - | | Other liabilities (non-current) | - | 3,162 | | Current liabilities | 100,356 | 52,804 | | Lease liabilities (current) | 6,995 | 6,859 | | Contract liabilities (current) | 2,601 | 363 | | Trade payables and accrued expenses | 59,417 | 21,897 | | Other liabilities (current) | 29,952 | 23,384 | | Income tax payables | 198 | 301 | | Provisions | 1,193 | - | | Total liabilities | 201,286 | 141,082 | | Total equity and liabilities | 1,084,921 | 979,793 | Additional Information Conference Call and Webcast Information Ascendis Pharma hosted a conference call and webcast on March 2, 2022, to discuss its full year 2021 financial results, with dial-in and webcast access details provided - Conference Call Details | Detail | Information | | :-------------------------- | :----------------------------------------- | | Date | Wednesday, March 2, 2022 | | Time | 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time | | Dial In (U.S.) | 844-290-3904 | | Dial In (International) | 574-990-1036 | | Access Code | 2009938 | | Webcast Link | www.ascendispharma.com (Investors and News section) | About Ascendis Pharma A/S Ascendis Pharma A/S is a global biopharmaceutical company leveraging its innovative TransCon platform technology to develop potentially best-in-class therapies - Ascendis Pharma applies its innovative platform technology to build a leading, fully integrated, global biopharmaceutical company focused on making a meaningful difference in patients' lives[14] - The company uses its TransCon technologies to create new and potentially best-in-class therapies, guided by its core values of patients, science and passion[14] - Ascendis is headquartered in Copenhagen, Denmark, with additional facilities in Heidelberg and Berlin, Germany; Palo Alto and Redwood City, California; and Princeton, New Jersey[14] Forward-Looking Statements This section contains forward-looking statements regarding Ascendis Pharma's future operations, plans, and objectives, which involve substantial risks and uncertainties - The press release contains forward-looking statements that involve substantial risks and uncertainties regarding Ascendis' future operations, plans, and objectives[15] - Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements due to various important factors[15] - Key risks include dependence on third-party manufacturers and distributors, unforeseen safety or efficacy results in development programs, unforeseen expenses related to commercialization and development, delays in development programs, uncertainty regarding market-size estimates and profitability, ability to obtain additional funding, and the effects of the worldwide COVID-19 pandemic[15][16] - Ascendis does not assume any obligation to update any forward-looking statements, except as required by law[16] Contact Information Contact details for investor relations and media inquiries are provided for Ascendis Pharma, including specific personnel and their contact information - Investor and Media Contacts | Role | Name | Contact Information | | :--------------- | :------------ | :-------------------------- | | Investor Contact | Tim Lee | +1 (650) 374-6343, tle@ascendispharma.com | | Media Contact | Melinda Baker | +1 (650) 709-8875, media@ascendispharma.com | | Media Contact | Patti Bank | +1 (415) 513-1284, patti.bank@westwicke.com | | General Investor Relations | N/A | ir@ascendispharma.com |