Specialised Therapeutics Asia Pte Ltd.

Strategic roadmap to achieve blockbuster status for multiple products and expand the Company’s engine for future innovation COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030. Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42nd Annual J.P. Morgan Healthcare Conference. “With unwavering focus on our values of Patients, Science, and Passion, we remain on track to fulfill Vision 3x3, with the approval already of two Endocrinology Rare Disease products and value leadership in the U.S. growth hormone market for SKYTROFA,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Today we are introducing Vision 2030, our strategic roadmap to attain blockbuster status for multiple products and expand our engine for future innovation.” Selected Key Updates TransCon™ hGH (lonapegsomatropin, approved as SKYTROFA® in the U.S. and EU) Following positive topline results from Phase 3 foresiGHt Trial in adult growth hormone deficiency (GHD), plan to submit a supplemental Biologics License Application to FDA in the second quarter of 2024.Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.Attained U.S. market value leadership in 2023.SKYTROFA preliminary unaudited fourth quarter 2023 revenue is expected to be approximately €64 million. Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates). TransCon PTH (palopegteriparatide, approved as YORVIPATH® in the EU) First launch planned in Germany in January 2024, leveraging the Company’s existing commercial infrastructure.FDA regulatory review for the treatment of adult patients with hypoparathyroidism continues, with a Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024. TransCon CNP (navepegritide) Treatment with TransCon CNP at 100 mg/kg/week for one year in all 57 children with achondroplasia (age 2 to 10 years) in ACcomplisH, demonstrated growth consistent with results from the blinded period and achieved improvements in health-related quality of life and disease impacts assessed with SF-10 and Achondroplasia Child Experience Measure.During the third quarter of 2023, submitted Clinical Trial Application (CTA) for infant trial (age 0-2 years).Topline results from the pivotal ApproaCH Trial expected in the fourth quarter of 2024.During the fourth quarter of 2024, plan to submit a New Drug Application to FDA for children with achondroplasia (age 2-11 years).Week 26 topline data from the COACH Trial (TransCon hGH/TransCon CNP combination) expected in the fourth quarter of 2024.During the fourth quarter of 2024, plan to submit an Investigational New Drug application or similar in adults with achondroplasia. Global commercial presence in Endocrinology Rare Disease During the fourth quarter of 2023, entered into an exclusive license agreement with Teijin Limited for TransCon hGH, TransCon PTH, and TransCon CNP in Japan. Phase 3 PaTHway Japan Trial of TransCon PTH completed, and Phase 3 riGHt Trial of TransCon hGH fully enrolled.VISEN’s TransCon hGH Phase 3 and TransCon CNP Phase 2 trials in China completed.Expanding global reach through exclusive sales & distribution agreements with geographic market leaders in International Markets, with three regional agreements signed to date: Specialised Therapeutics Asia Pte Ltd, Er-Kim, and Vector Pharma FZCO. Oncology During the fourth quarter of 2024, expect to complete enrollment in BelieveIT-201, a Phase 2 trial in advanced head and neck squamous cell carcinoma (HNSCC).During the fourth quarter of 2024, plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts in the IL-Believe Trial. Ophthalmology Creation of Ophthalmology NewCo, financed by institutional investors, expected during the first quarter of 2024. Financial Update and Outlook Based on Current Plans Preliminary unaudited December 31, 2023 cash, cash equivalents, and marketable securities of ~€400 million.Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates).Expect to provide YORVIPATH revenue update during 2024.Expect total operating expenses (SG&A and R&D) of approximately €600 million for 2024.Expect to be operating cash flow breakeven on a quarterly basis by the end of 2024. Vision 2030: Ascendis Pharma’s 2025–2030 Strategic Roadmap Achieve blockbuster status for multiple products and expand our engine for future innovation. Be the Leading Endocrinology Rare Disease Company Achieve blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercializationBe the leader in growth disorders and hypoparathyroidism, pursuing clinical conditions, innovative life cycle management, and complementary patient offeringsExpand pipeline with Endocrinology Rare Disease blockbuster product opportunities Create Value in Additional Therapeutic Areas through Innovative Business Models Obtain accelerated approval in oncology with registrational trials ongoingPursue TransCon product opportunities in >$5B indicationsMaximize value creation of these product opportunities through collaboration with therapeutic area market leaders Differentiate with Ascendis Fundamentals Outperform industry drug development benchmarks with Ascendis’ product innovation algorithmRemain independent as a profitable biopharma through lean and flexible ways of workingLet our values Patients, Science, Passion drive our decisions to success Presentation at J.P. Morgan Healthcare Conference on Monday, January 8th A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com. The presentation will begin at 12:00 p.m. Eastern Time / 9:00 am Pacific Time. A webcast replay will be available for 30 days. The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at https://investors.ascendispharma.com. 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 Germany and the United States. Please visit 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’ ability to achieve blockbuster status for multiple products and expand its engine for future innovation, (ii) Ascendis’ ability to achieve its 2024 corporate milestones and Vision 2030, (iii) Ascendis’ ability to fulfill Vision 3x3, (iv) Ascendis’ plan to submit a supplemental Biologics License Application for TransCon hGH in the second quarter of 2024, (v) the timing of topline results from the Phase 2 trial in Turner syndrome, (vi) Ascendis’ expectations with respect to SKYTROFA revenue for 2023 and 2024, (vii) Ascendis’ plan to launch TransCon PTH in Germany in January 2024, (viii) TransCon PTH’s PDUFA date of May 14, 2024, (ix) Ascendis’ plans to launch TransCon PTH in the U.S. in the third quarter of 2024, if approved, (x) the timing of topline results from the ApproaCH Trial, (xi) Ascendis’ plan to submit a New Drug Application for TransCon CNP for children with achondroplasia, (xii) Ascendis’ plan to submit an Investigational New Drug application or similar for TransCon CNP in adults with achondroplasia; (xiii) the timing of topline annualized height velocity data from the COACH Trial, (xiv) Ascendis’ ability to expand its global reach through exclusive sales & distribution agreements with geographic market leaders, (xv) Ascendis’ expectations with regard to the completion of enrollment in BelieveIT-201, (xvi) Ascendis’ plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts in the IL-Believe Trial, (xvii) Ascendis’ expectations regarding its financial results and performance in 2023 and 2024, (xviii) Ascendis’ Vision 2030, (xix) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xx) 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, SKYTROFA®, and YORVIPATH® are trademarks owned by the Ascendis Pharma group. © January 2024 Ascendis Pharma A/S. Investor Contacts: Media Contact:Tim Lee Melinda BakerAscendis Pharma Ascendis Pharma+1 (650) 374-6343 +1 (650) 709-8875ir@ascendispharma.com media@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com

Agreement between Ascendis Pharma A/S and ST is for the exclusive distribution and commercialisation of three Ascendis Pharma endocrinology therapies Two of the therapies are already internationally approved, the third is in development following successful Phase 2 data ST's exclusive distribution agreement covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand and Vietnam SINGAPORE, Jan. 7, 2024 /PRNewswire/ -- Independent biopharmaceutical company Specialised Therapeutics Asia Pte Ltd (ST) has added three new endocrinology therapies to its specialist portfolio, following an exclusive distribution agreement with Danish company Ascendis Pharma A/S (NASDAQ: ASND). Under the terms of the agreement, ST will commercialise Ascendis Pharma's weekly injectable paediatric human growth hormone treatment SKYTROFATM (lonapegsomatropin), hypoparathyroidism treatment YORVIPATHTM (palopegteriparatide) and investigational achondroplasia therapy TransCon™ CNP (navepegritide). The agreement spans ST's key regions of Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. Two of the products included in this agreement are already internationally approved: Once-weekly SKYTROFA is a human growth hormone (hGH) approved in the United States for the treatment of paediatric patients aged >1 years weighing >11.5 kg with growth failure due to inadequate secretion of endogenous growth hormone (GH)1 and in the European Union for growth failure in children and adolescents aged from 3 to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD]).2 YORVIPATH is a first-in-class parathyroid hormone (PTH) replacement therapy to treat chronic hypoparathyroidism, a rare and potentially serious condition where the body produces no or abnormally low levels of PTH. It is approved in the European Union for the treatment of adults with chronic hypoparathyroidism.3 The third product – TransCon CNP – is in development by Ascendis Pharma for the treatment of achondroplasia (ACH), the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, following successful Phase 2 trial results.4 Australian endocrinologist Dr Veronica Preda noted that YORVIPATH would be the first specialist therapeutic option for Australian patients living with hypoparathyroidism. "Hypoparathyroidism can seriously impact quality of life and has potentially life-threatening consequences," Dr Preda said. "To have an option that is able to treat the underlying cause of the disease, moving beyond standard oral calcium and active Vitamin D, is a great step forward." Announcing the partnership, ST Chief Executive Officer Carlo Montagner said this agreement was an important company milestone, signalling ST's expansion into both endocrinology and paediatric medicine. Mr Montagner commented: "We are delighted to have been selected as Ascendis Pharma's exclusive partner for commercialising their portfolio in Oceania and these South-East Asia countries and look forward to launching these critical endocrinology products in our regions as soon as possible. "All three products are valuable inclusions to our broad therapeutic pipeline and our international business, as we continue to leverage our substantial experience commercialising specialist medicines across multiple regions. "We look forward to working with endocrinologists across our territories to make these endocrine therapies available to all eligible patients who may benefit." Ascendis Pharma Executive Vice President and Chief Commercial Officer Camilla Harder Hartvig said ST had been selected to launch the endocrinology portfolio in these countries based on its strong track record commercialising specialist products in multiple regions. "We are delighted to partner with Specialised Therapeutics to broaden the reach of our endocrinology rare disease portfolio, contributing to our shared goal of making a meaningful difference for patients facing unmet medical needs," she said. About Specialised Therapeutics Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in Australia, New Zealand and across Southeast Asia. Headquartered in Singapore, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases. ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions. The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas. ST is a member of the World Orphan Drug Alliance (WODA). Additional information: About Ascendis Pharma 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, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Please visit to learn more. About SKYTROFA (lonapegsomatropin) SKYTROFA™ (lonapegsomatropin, developed as TransCon™ hGH) is a prodrug of somatropin, designed to provide sustained release of unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the same 191 amino acid sequence and size as endogenous GH.5 TransCon hGH is approved and marketed as SKYTROFA (lonapegsomatropin-tcgd) in the United States1 and as SKYTROFA (lonapegsomatropin) in the European Union2 as a once-weekly treatment for children and adolescents with GHD. SKYTROFA single-use, prefilled cartridges are manufactured in nine dosage strengths, allowing for convenient dosing flexibility. They are designed for use only with the SKYTROFA Auto-Injector and may be stored at room temperature for up to six months. The recommended dose of SKYTROFA for treatment-naïve patients and patients switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly.1,2 The dose may be adjusted based on the child's weight and insulin-like growth factor-1 standard deviation score (IGF-1 SDS).2 TransCon hGH was studied in over 300 children with GHD across the Phase 3 program, which consisted of the Height Trial5 (for treatment-naïve patients), the Flight Trial6 (for treatment-experienced patients), and the Enlighten Trial7 (a long-term extension trial). Patients who completed the Height or Flight Trials were able to continue in Enlighten, with some on lonapegsomatropin treatment for over four years. Ascendis Pharma is also conducting the ongoing global Phase 3 Foresight Trial of TransCon hGH in adults with GHD.8 About Paediatric Growth Hormone Deficiency Paediatric GHD is a serious orphan disease caused when the pituitary gland does not produce enough GH. Children with GHD are not only characterised by short stature; they also may experience metabolic abnormalities, psychosocial challenges, and an overall poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of hGH to improve growth and overall endocrine health. About YORVIPATH (palopegteriparatide) YORVIPATH™ (palopegteriparatide, developed as TransCon™ PTH) is a once-daily prodrug with sustained release of active PTH approved by the European Union as a PTH replacement therapy for the treatment of adults with chronic hypoparathyroidism. Treatment should be initiated and monitored by physicians or qualified healthcare professionals experienced in the diagnosis and management of patients with hypoparathyroidism.3 TransCon PTH met all primary and key secondary endpoints in the Phase 3 Pathway Trial, demonstrating a response rate of 78.7% compared to 4.8% for control (p-value <0.0001) for the primary composite endpoint, and statistically significant improvements compared to control on all key secondary endpoints, which included measures evaluating patient-reported disease symptoms and impacts.9 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. 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.9 About TransCon CNP (navepegritide) TransCon™ CNP (navepegritide) is an investigational long-acting prodrug of C-type natriuretic peptide (CNP), designed to provide continuous exposure of CNP at safe, therapeutic levels, via a single, weekly subcutaneous dose, for the treatment of children with ACH.4 The Phase 2 Accomplish Trial, a randomised, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in prepubertal children with ACH aged 2 to 10 years old, met its primary objectives, and demonstrated that TransCon CNP at 100 µg/kg/week was superior to placebo for the primary efficacy endpoint of annualised growth velocity (AGV) at 52 weeks4. All 57 randomised children completed the blinded portion of Accomplish and are currently continuing in the open label extension at the 100 μg/kg/week dose4. Ascendis Pharma recently confirmed that these 57 clinical trial patients have all completed one year of treatment with TransCon CNP at 100 µg/kg/week, and announced that TransCon CNP is the first investigational product to demonstrate improvements in health-related quality of life and disease impacts in children with ACH.10 About Achondroplasia Achondroplasia is the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, caused by a genetic mutation in the fibroblast growth factor receptor 3 (FGFR3). This leads to an imbalance between the stimulatory and inhibitory signaling pathways involved in regulating bone growth. People living with ACH may experience serious complications and comorbidities due to inhibited skeletal development. Complications may include sleep apnoea and respiratory problems, chronic back and leg pain from lower spine impingement, and sudden infant death from compression of the brain stem. Chronic ear infections due to eustachian tube problems can lead to hearing loss and speech delay. Children with ACH may also experience social and emotional challenges. References SKYTROFA (lonapegsomatropin-tcgd) US Prescribing Information. SKYTROFA (lonapegsomatropin) EU Summary of Product Characteristics. YORVIPATH (palopegteriparatide) EU Summary of Product Characteristics. Savarirayan, R, Hoemschemeyer DG, Ljungberg M, et al., Lancet. 2023;65:1–10. Thornton PS, Maniatis AK, Aghajanova E, et al., J Clin Endocrinol Metab. 2021;106(11):3184–3195. Maniatis AK, Nadgir U, Saenger P, et al., Horm Res Paediatr. 2022;95(3):233–243. Maniatis AK, Casella SJ, Nadgir UM, et al. J Clin Endocrinol Metab. 2022;107(7): e2680–e2689. Foresight Trial (NCT05171855), . Khan AA, Rubin MR, Schwarz P, et al. J Bone Miner Res. 2023;38(1):14–25. Ascendis Pharma A/S, Significant Health and Quality of Life Improvements Achieved in Children with Achondroplasia Treated for One Year with TransCon™ CNP (Navepegritide) at 100 µg/kg/week, Press Release, 20 December 2023, . SOURCE Specialised Therapeutics

YONDELIS® (trabectedin) now PBS listed for Australian patients Listing described as "wonderful news" for patients living with rare lipo and leiomyo sarcomas YONDELISÒ (trabectedin) demonstrates 45% reduction in risk of disease progression or death versus dacarbazine1 SINGAPORE, July 31, 2023 /PRNewswire/ -- AUSTRALIAN cancer patients who have been diagnosed with rare soft tissue sarcomas will now have affordable access to a global therapy shown to improve survival, following its listing on the Pharmaceutical Benefits Scheme (PBS). The therapy YONDELIS (trabectedin) is a novel anti-tumour agent originally derived from the sea squirt and will be available to eligible patients on the PBS from August 1. It is used extensively around the world and has been shown to improve progression-free survival for patients with liposarcoma and leiomyosarcoma when used after anthracycline-based therapy.1 Until today, some patients have paid up to $50,000 to access YONDELIS treatment. News of the PBS listing is being welcomed by oncologists and the Australian sarcoma community, who say it will alleviate cost of treatment pressures for those patients whose disease has progressed. Medical oncologist and Scientific Advisory Committee member and Lead of the ANZSA National Sarcoma Database Dr Susie Bae, said YONDELIS has been available in Europe since 2007 for patients with advanced soft tissue sarcoma, and Australian patients had waited many years for reimbursed access. "This milestone means patients don't need to worry about not being able to afford or miss out on an active drug that can potentially buy precious time with their loved ones, by providing disease control and keeping symptoms at bay for longer," Dr Bae said. Melbourne patient advocate and mother of two Karen Lurati – herself diagnosed with liposarcoma six years ago – said this listing provided new hope for other patients. "A PBS listing for YONDELIS is so exciting for those people who may not have been able to afford the treatment before," she said. "Rare cancers don't often get (Government) funding or attention. To now have this therapy on the PBS is great progress. "Patients often feel that they have to go overseas and spend enormous amounts of money on treatments that may not be available in Australia. This can be frustrating and financially crippling. So, for patients to have access to a global therapy in their own country is wonderful news." And Rare Cancers Australia (RCA) Chief Executive Richard Vines said he was "delighted with this outcome", describing the listing as "great news" for patients living with an L-sarcoma. "For too long, sarcoma patients have been unable to access all therapies which may provide benefit," he said. Today's announcement means they can access a PBS funded medicine instead of having to try and find tens of thousands of dollars – if not more – to self-fund a treatment that may give them more time." YONDELIS is marketed in Australia by independent pharmaceutical company Specialised Therapeutics, under an exclusive license arrangement with international partner PharmaMar. ST Chief Executive Officer Carlo Montagner said the PBS listing was a significant milestone for the company. He commented: "We acquired the YONDELIS rights in 2019 following requests from key oncology groups and doctors, who had been importing the product at great cost and with complex logistics for those patients diagnosed with these rare cancers. "This PBS listing is the culmination of a substantive effort by our team together with the oncology community to achieve full regulatory approval and a PBS listing. "We look forward to continuing our work with the sarcoma community." Ends. About Specialised Therapeutics Asia Headquartered in Singapore, Specialised Therapeutics (ST) is an international biopharmaceutical company established to commercialise new therapies and technologies to patients in Australia, New Zealand and across South-East Asia. ST and its regional affiliates collaborate with leading global pharmaceutical and diagnostic companies to bring novel, innovative and life-changing healthcare solutions to patients affected by a range of diseases. Our mission is to provide therapies that would otherwise not be available to communities in our regions. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, neurology, ophthalmology and supportive care. Additional information can be found at About YONDELIS® (trabectedin) YONDELIS® (trabectedin) is a novel, multimodal, synthetically produced antitumor agent, originally derived from the sea squirt, Ecteinascidia turbinata. The anti-cancer medicine works by preventing tumor cells from multiplying and is approved in 76 countries in North America, Europe, South America and Asia for the treatment of advanced soft-tissue sarcomas as a single-agent, and in 69 countries for relapsed ovarian in combination with doxorubicin HCl liposome injection. The approval was based on the results of a pivotal phase 3, randomised, open-label controlled study which evaluated YONDELIS versus dacarbazine in over 500 patients with unresectable or metastatic liposarcoma (LPS) or leiomyosarcoma (LMS) previously treated with an anthracycline and at least one additional chemotherapy regimen. LPS and LMS are subtypes of soft tissue sarcoma (STS) and represent more than 35% of all STS cases.3 The median progression-free survival (PFS) among the YONDELIS treatment group was 4.2 months compared to 1.5 months in the dacarbazine treatment group, representing a 45% reduction in the risk of disease progression or death with YONDELIS (HR=0.55; 95% CI: 0.44 - 0.70; p<0.001).1 Among the 340 patients who received YONDELIS and were included in the safety analysis in the randomised trial, the most common (≥20%) adverse reactions were nausea (73%), fatigue (67%), vomiting (44%), constipation (36%), decreased appetite (34%), diarrhoea (34%), dyspnoea (25%), peripheral oedema (24%) and headache (23%). The most common (≥20%) laboratory abnormalities were neutropenia (49%), increased alanine transaminase (ALT) (45%), anaemia (39%), increased aspartate aminotransferase (AST) (35%), thrombocytopaenia (30%) and increased blood alkaline phosphatase (20%).1 About Soft Tissue Sarcoma Soft tissue sarcoma is a rare type of cancer that forms as a painless lump (tumour) in any one of the soft tissues connecting all the organs and body structures – including fat, muscle, nerves, deep skin tissue, blood vessels and the tissue surrounding joints (synovial tissue). Soft tissue sarcomas commonly develop in the thigh, shoulder and pelvis and may sometimes develop in the abdomen or chest.6 Metastatic or locally advanced STS is generally considered incurable, with the mainstay of treatment being systemic chemotherapy. For some patients with limited disease burden however, long-term remission can be achieved through a multimodality approach involving medical, surgical and radiation therapy.4 About PharmaMar PharmaMar is a biopharmaceutical company focused on the research and development of new oncology treatments, whose mission is to improve the healthcare outcomes of patients afflicted by serious diseases with our innovative medicines. The Company is inspired by the sea, driven by science, and motivated by patients with serious diseases to improve their lives by delivering novel medicines to them. PharmaMar intends to continue to be the world leader in marine medicinal discovery, development and innovation. PharmaMar has developed and now commercializes Yondelis® in Europe by itself, as well as Zepzelca® (lurbinectedin), in the US; and Aplidin® (plitidepsin), in Australia, with different partners. In addition, it has a pipeline of drug candidates and a robust R&D oncology program. PharmaMar has other clinical-stage programs under development for several types of solid cancers: lurbinectedin, ecubectedin, PM534 and PM54. It also has a preclinical and clinical program in virology. Headquartered in Madrid (Spain), PharmaMar has subsidiaries in Germany, France, Italy, Belgium, Austria, Switzerland and The United States. PharmaMar also wholly owns Sylentis, a company dedicated to researching therapeutic applications of gene silencing (RNAi). To learn more about PharmaMar, please visit us at . SOURCE Specialised Therapeutics