Irinotecan Hydrochloride Lioposome

Positive data from a Pfizer-partnered antibody-drug conjugate, Roche's new investment in China and Hengrui's upcoming dual listing in Hong Kong made our news this week. Pfizer and RemeGen's HER2 antibody-drug conjugate delivered positive phase 3 results in a bladder cancer trial in China. Roche plans to invest nearly $300 million to build a new Chinese plant to produce Vabysmo. China's Hengrui Pharma is looking to raise up to $1.27 billion in its Hong Kong IPO. And more.1. Pfizer partner RemeGen says HER2 ADC prolongs life in newly diagnosed bladder cancerRemeGen’s Pfizer-partnered HER2 antibody-drug conjugate, disitamab vedotin, was successful in a phase 3 study among Chinese patients with first-line HER2-expressing bladder cancer. The ADC, used alongside Junshi Biosciences’ PD-1 inhibitor toripalimab, outperformed chemotherapy regardless of patients' eligibility for cisplatin or HER2 expression levels. The trial win bodes well for Pfizer’s global DV-001 study, also called Keynote-D74.2. Roche pledges nearly $300M to establish new Vabysmo production plant in ChinaRoche is investing about 2 billion Chinese yuan ($282 million) to build a new biologics plant in Shanghai. The plant will occupy about 25,000 square meters (269,000 square feet) of real estate and will mainly be used for domestic production of the eye med Vabysmo. Construction is expected to finish in 2029, and production could kick off by 2031, according to Roche.3. Hengrui looks to raise up to $1.27B in Hong Kong IPO (Morningstar)Jiangsu Hengrui Pharmaceutical, already listed in Shanghai, is planning to raise up to HK$9.89 billion ($1.27 billion) in a Hong Kong IPO. The plan involves selling 224.52 million shares at a price range of HK$41.45 to HK$44.05 each. The Chinese pharma giant expects to finalize pricing by May 21 and start trading from May 23.4. Qilu Pharmaceutical pays $38M upfront for greater China rights to Minghui's ADCThe B7-H3 target has yielded another deal. Qilu Pharmaceutical is paying 280 million Chinese yuan ($38 million) upfront for greater China rights to Minghui Pharmaceutical’s B7-H3 ADC, MHB088C. Also in the B7-H3 ADC field, Merck & Co. has licensed ifinatamab deruxtecan from Daiichi Sankyo, GSK got a candidate from Hansoh and BioNTech partnered with Duality Bio.5. CSPC licenses Onivyde copycat to Cipla despite Ipsen patent dispute (release, PDF)Despite a pending patent infringement lawsuit from Ipsen, CSPC Pharmaceutical has penned a deal to license its irinotecan liposome injection in the U.S. to Cipla for $15 million upfront. Milestone payments from the pact could exceed $1 billion. In a lawsuit filed in August 2024, Ipsen alleges that the CSPC candidate infringes a patent for the French pharma’s irinotecan liposome product Onivyde.6. Novartis cancer drugs Kisqali, Scemblix win China approvals (releases, Chinese)In two approvals for Novartis’ business in China, local authorities approved the company’s Kisqali for the adjuvant treatment of early-stage HR-positive, HER2-negative breast cancer, as well as Scemblix for newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia. Both indications have been cleared in the U.S. and are considered major growth drivers for the Swiss pharma.Other News of Note:7. Merck KGaA drops China surcharge on orders after US-China tariff deal (Reuters)8. Sino-American siRNA biotech Sirius Therapeutics raises $50M (release)

FY 2024 total sales growth of 9.9% at CER1, or 8.7% as reported, with growth driven by strong performance across all therapeutic areas, including a 67.4% increase in the Rare Diseases portfolio, 9.2% in Neuroscience, and 7.3% in Oncology; Somatuline® (lanreotide) sales grew by 5.6%, while all other products, excluding Somatuline, achieved double-digit sales growth at 12.2% FY 2024 core operating income of €1,109m, growing 10.8% as reported, with core operating margin of 32.6% of total sales Continued pipeline expansion in 2024, with significant regulatory approvals, addition of several preclinical therapies with global rights and innovative modalities, and a late-stage asset Four key regulatory and clinical milestones expected in 2025, including the Proof-of-Concept data readout for the Long-Acting Neurotoxin (LANT) Financial guidance2 for 2025 including total sales growth greater than 5.0%3 at CER, and core operating margin greater than 30.0% of total sales, based on accelerated sales growth of the ex-Somatuline portfolio and assuming negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe PARIS, FRANCE, 13 February 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its financial results for the full year 2024. “Ipsen delivered solid results and advanced its pipeline in 2024, laying a strong foundation for sustained growth,” said David Loew, Chief Executive Officer, Ipsen. “With the successful global rollout of Iqirvo and Bylvay, and the U.S. launch of Onivyde, alongside multiple business development deals adding several innovative assets, we are well positioned to execute our strategic roadmap. This year, we look forward to achieving key milestones, including the first data readout for the Long-Acting Neurotoxin (LANT), and further expand and progress our pipeline across all three therapeutic areas to bring promising new medicines to patients.” Pipeline Progress Significant regulatory milestones were achieved in 2024, including FDA approval of Onivyde® (irinotecan) for first-line pancreatic ductal adenocarcinoma (PDAC), along with accelerated U.S. approval and European approval for Iqirvo® (elafibranor), respectively. Additionally, Kayfanda® (odevixibat) was approved for Alagille syndrome (ALGS) in the E.U. The company also opted-in for the CABINET Phase III study of Cabometyx® (cabozantinib) in patients with advanced neuroendocrine tumors (NETs), with study results presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in the New England Journal of Medicine. An IND application was filed for IPN01194, an ERK inhibitor, advancing the potential medicine into clinical development with a Phase I/IIa trial in advanced solid tumors. Ipsen improved further the depth and breadth of its pipeline by adding five preclinical innovative therapies with global rights and new modalities, and an ex-U.S. licensing agreement with DayOne Biopharmaceuticals for the late-stage oncology asset tovorafenib, an oral RAF inhibitor for pediatric low-grade glioma. Two global licensing agreements for Antibody Drug Conjugate (ADC) in oncology with Sutro Biopharma and Foreseen Biotechnology were signed. An extension of the oncology partnership with Marengo Therapeutics to include TriSTAR, a next-generation precision T-cell engager was completed, as well as more recently, in the fourth quarter, a global licensing agreement with Biomunex for a preclinical novel T-cell engager (TCE). A collaboration with Skyhawk Therapeutics to develop RNA-modulating small molecules for rare neurological diseases was also signed during the year. Ipsen executed several divestments in 2024, including the sale of Increlex® (mecasermin injection) to Eton Pharmaceuticals and the sale of its rare pediatric disease Priority Review Voucher. Environmental, Social and Governance Ipsen took important steps in 2024 in delivering its ambitious sustainability strategy. The company continued to integrate sustainability across its operations. From reducing its environmental footprint to advancing patient access and fostering a strong workplace culture, the company increased its commitment to driving progress for patients, employees, communities, and the planet. Our sustainability efforts were recognized across multiple environment initiatives. The company achieved a 45% reduction in Scopes 1 & 2 greenhouse gas emissions and a 25% reduction in Scope 3, fully in line with its 2030 targets (versus 2019 baseline). Significant efforts were made to engage suppliers and third parties in Ipsen’s sustainability roadmap including the first-ever “Ipsen Supplier Sustainability Day”. Following an intensive transformation project, 99.8% of Ipsen’s global electricity now comes from renewable sources. Through the Fleet for Future project, the company continues to advance sustainable transportation, with 43% of its total company’s fleet now electric vehicles as of 2024. We remain committed to gender balance in leadership, with women now representing 55% of the Global Leadership Team. 2025 Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in Europe for advanced neuroendocrine tumors (NETs), including pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors Tovorafenib (FIREFLY-1 trial) – Regulatory submission in Europe for pediatric low-grade glioma Fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP) LANT88(LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics These milestones reinforce Ipsen’s commitment to advancing innovative therapies and expanding treatment options for patients worldwide. 2025 Financial Guidance Ipsen has set for FY 2025 the following financial guidance, which excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in January 2025, a favorable effect on total sales of around 1% from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance on total sales and core operating margin is based on accelerated sales growth of the ex-Somatuline portfolio and assumes negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. Consolidated financial statements The Board of Directors approved the consolidated financial statements on 12 February 2025. The consolidated financial statements have been audited and the Statutory Auditors’ report is in the process of being published. Ipsen’s comprehensive audited financial statements will be available in due course on ipsen.com (regulated-information section). Conference call A conference call and webcast for investors and analysts will begin today at 1pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its Q1 2025 sales on April 16th, 2025. Notes All financial figures are in € millions (€m), unless otherwise noted. The performance shown in this announcement covers the twelve-month period to 31 December 2024 (FY 2024) and the three-month period to 31 December 2024 (Q4 2024), compared to the twelve-month period to 31 December 2023 (FY 2023) and the three-month period to 31 December 2023 (Q4 2023), respectively, unless stated otherwise. The commentary is based on the performance in FY 2024, unless stated otherwise. About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com. Ipsen contacts Investors Media 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions.3 Based on the average level of exchange rates in Jan 2024, a favorable effect on total sales of about 1% from currencies is expected. 4 Extract of consolidated results. The Company’s auditors performed a limited review of the condensed consolidated financial statements.5 Including an impairment loss of €279m (or €2,33 /share) related to Sohonos, reflecting lower revised sales following lower patient uptake.6 Dividend related to the current financial year to be paid the following year.7 Decided by the Ipsen S.A. Board of Directors and to be proposed at the annual shareholders’ meeting on 21 May 2025. 8 Long-acting neurotoxin Attachment Ipsen PR_FY 2024 – Results announcement_13022025 FY 2024 total sales growth of 9.9% at CER1, or 8.7% as reported, with growth driven by strong performance across all therapeutic areas, including a 67.4% increase in the Rare Diseases portfolio, 9.2% in Neuroscience, and 7.3% in Oncology; Somatuline® (lanreotide) sales grew by 5.6%, while all other products, excluding Somatuline, achieved double-digit sales growth at 12.2% FY 2024 core operating income of €1,109m, growing 10.8% as reported, with core operating margin of 32.6% of total sales Continued pipeline expansion in 2024, with significant regulatory approvals, addition of several preclinical therapies with global rights and innovative modalities, and a late-stage asset Four key regulatory and clinical milestones expected in 2025, including the Proof-of-Concept data readout for the Long-Acting Neurotoxin (LANT) Financial guidance2 for 2025 including total sales growth greater than 5.0%3 at CER, and core operating margin greater than 30.0% of total sales, based on accelerated sales growth of the ex-Somatuline portfolio and assuming negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe PARIS, FRANCE, 13 February 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its financial results for the full year 2024. “Ipsen delivered solid results and advanced its pipeline in 2024, laying a strong foundation for sustained growth,” said David Loew, Chief Executive Officer, Ipsen. “With the successful global rollout of Iqirvo and Bylvay, and the U.S. launch of Onivyde, alongside multiple business development deals adding several innovative assets, we are well positioned to execute our strategic roadmap. This year, we look forward to achieving key milestones, including the first data readout for the Long-Acting Neurotoxin (LANT), and further expand and progress our pipeline across all three therapeutic areas to bring promising new medicines to patients.” Pipeline Progress Significant regulatory milestones were achieved in 2024, including FDA approval of Onivyde® (irinotecan) for first-line pancreatic ductal adenocarcinoma (PDAC), along with accelerated U.S. approval and European approval for Iqirvo® (elafibranor), respectively. Additionally, Kayfanda® (odevixibat) was approved for Alagille syndrome (ALGS) in the E.U. The company also opted-in for the CABINET Phase III study of Cabometyx® (cabozantinib) in patients with advanced neuroendocrine tumors (NETs), with study results presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in the New England Journal of Medicine. An IND application was filed for IPN01194, an ERK inhibitor, advancing the potential medicine into clinical development with a Phase I/IIa trial in advanced solid tumors. Ipsen improved further the depth and breadth of its pipeline by adding five preclinical innovative therapies with global rights and new modalities, and an ex-U.S. licensing agreement with DayOne Biopharmaceuticals for the late-stage oncology asset tovorafenib, an oral RAF inhibitor for pediatric low-grade glioma. Two global licensing agreements for Antibody Drug Conjugate (ADC) in oncology with Sutro Biopharma and Foreseen Biotechnology were signed. An extension of the oncology partnership with Marengo Therapeutics to include TriSTAR, a next-generation precision T-cell engager was completed, as well as more recently, in the fourth quarter, a global licensing agreement with Biomunex for a preclinical novel T-cell engager (TCE). A collaboration with Skyhawk Therapeutics to develop RNA-modulating small molecules for rare neurological diseases was also signed during the year. Ipsen executed several divestments in 2024, including the sale of Increlex® (mecasermin injection) to Eton Pharmaceuticals and the sale of its rare pediatric disease Priority Review Voucher. Environmental, Social and Governance Ipsen took important steps in 2024 in delivering its ambitious sustainability strategy. The company continued to integrate sustainability across its operations. From reducing its environmental footprint to advancing patient access and fostering a strong workplace culture, the company increased its commitment to driving progress for patients, employees, communities, and the planet. Our sustainability efforts were recognized across multiple environment initiatives. The company achieved a 45% reduction in Scopes 1 & 2 greenhouse gas emissions and a 25% reduction in Scope 3, fully in line with its 2030 targets (versus 2019 baseline). Significant efforts were made to engage suppliers and third parties in Ipsen’s sustainability roadmap including the first-ever “Ipsen Supplier Sustainability Day”. Following an intensive transformation project, 99.8% of Ipsen’s global electricity now comes from renewable sources. Through the Fleet for Future project, the company continues to advance sustainable transportation, with 43% of its total company’s fleet now electric vehicles as of 2024. We remain committed to gender balance in leadership, with women now representing 55% of the Global Leadership Team. 2025 Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in Europe for advanced neuroendocrine tumors (NETs), including pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors Tovorafenib (FIREFLY-1 trial) – Regulatory submission in Europe for pediatric low-grade glioma Fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP) LANT88(LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics These milestones reinforce Ipsen’s commitment to advancing innovative therapies and expanding treatment options for patients worldwide. 2025 Financial Guidance Ipsen has set for FY 2025 the following financial guidance, which excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in January 2025, a favorable effect on total sales of around 1% from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance on total sales and core operating margin is based on accelerated sales growth of the ex-Somatuline portfolio and assumes negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. Consolidated financial statements The Board of Directors approved the consolidated financial statements on 12 February 2025. The consolidated financial statements have been audited and the Statutory Auditors’ report is in the process of being published. Ipsen’s comprehensive audited financial statements will be available in due course on ipsen.com (regulated-information section). Conference call A conference call and webcast for investors and analysts will begin today at 1pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its Q1 2025 sales on April 16th, 2025. Notes All financial figures are in € millions (€m), unless otherwise noted. The performance shown in this announcement covers the twelve-month period to 31 December 2024 (FY 2024) and the three-month period to 31 December 2024 (Q4 2024), compared to the twelve-month period to 31 December 2023 (FY 2023) and the three-month period to 31 December 2023 (Q4 2023), respectively, unless stated otherwise. The commentary is based on the performance in FY 2024, unless stated otherwise. About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com. Ipsen contacts Investors Media 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions.3 Based on the average level of exchange rates in Jan 2024, a favorable effect on total sales of about 1% from currencies is expected. 4 Extract of consolidated results. The Company’s auditors performed a limited review of the condensed consolidated financial statements.5 Including an impairment loss of €279m (or €2,33 /share) related to Sohonos, reflecting lower revised sales following lower patient uptake.6 Dividend related to the current financial year to be paid the following year.7 Decided by the Ipsen S.A. Board of Directors and to be proposed at the annual shareholders’ meeting on 21 May 2025. 8 Long-acting neurotoxin Attachment Ipsen PR_FY 2024 – Results announcement_13022025