IPS HEART Granted Orphan Drug Designation in the EU for Becker Muscular Dystrophy
While we are grateful to the dozens of BMD patients who have emailed us directly for information on our upcoming clinical trials, to date we are initially focused on advancing GIVI-MPCs clinically in a Phase I/II DMD clinical trial until we can secure additional partners to help us advance clinically in BMD. IPS HEART is currently under formal CDA with certain large pharmaceutical companies for a possible partnership that will also allow us to advance GIVI-MPCs clinically in BMD as we have already successfully produced GIVI-MPCs via large scale suspension based bioreactor manufacturing which can supply the large patient population in the future.
About IPS HEART/GIVI-MPCs
GIVI-MPCs derived from pluripotent stem cells form new skeletal muscle with 100% full-length human dystrophin for DMD and BMD. IPS HEART has also developed ISX-9-CPCs, a human iPSC-derived cardiac therapy for treatment of Duchenne cardiomyopathy and heart failure that develops new heart muscle.
About Becker & Duchenne Muscular Dystrophy
BMD is a muscular disease and DMD is a fatal neuromuscular disease.
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Because we understand that the skin we are in shapes our lives, we are advancing dermatology for every skin story. For more information: Appendices Appendix 1: H1 2025 net sales by product category and geography Appendix 2: Q2 2025 net sales by product category and geography Appendix 3: Reconciliation of H1 2025 P&L from IFRS to Core reporting In million USD IFRS - as reported Exceptional & transformation related items Impairments Amortization Depreciation Core reporting % Net Sales based on Core reporting Net Sales 2,448 - - - - 2,448 Other revenue 18 - - - - 18 Cost of goods sold (761) - 5 105 11 (641) Gross profit 1,705 - 5 105 11 1,826 74.6% Research and development (104) - - - 1 (103) 4.2% Sales and marketing (818) - - - 7 (811) 33.1% General and administrative (276) - 4 17 16 (238) 9.7% Medical and regulatory (55) - - - - (55) 2.2% Distribution (64) - - - 1 (64) 2.6% Other income / (expenses) (29) 29 - - - - - Operating profit as reported 358 Total adjustments 29 9 122 36 Core EBITDA 555 Expand Appendix 4: Reconciliation of H1 2025 of Core EBITDA to IFRS Net Income Appendix 5: Reconciliation of H1 2025 from IFRS Net Income to Core Net Income 12 In million USD H1 2024 H1 2025 Net income / (loss) 47 194 Total EBITDA adjustments 11 59 38 VCB financing revaluation (28) - Amortization 112 122 Foreign exchange loss on financing activities 30 1 Income taxes on above items (10) (25) Core Net Income 12 210 329 Core EPS in USD 13 0.89 1.39 Expand Appendix 6: H1 2025 Total Net Indebtedness In million USD December 31 2024 June 30 2025 Total Indebtedness 14 2,813 2,715 Cash and Cash Equivalents (457) (458) Total Net Indebtedness 2,356 2,257 Expand Appendix 7: Additional modeling metrics Notes and references Note: Due to rounding numbers presented may not add up precisely to the totals provided and percentages may not precisely reflect the absolute figures. All ratios, subtotals and variances are calculated using the underlying amount rather than the presented rounded amount. NICE. Nemolizumab for treating atopic dermatitis - technology appraisal guidance. Available online. Accessed June 2025 Silverberg, JI, et al. Nemolizumab long-term safety and efficacy up to 104 weeks in the ARCADIA open-label extension study in adolescents and adults with moderate-to-severe atopic dermatitis. Presented at Revolutionizing Atopic Dermatitis Conference 2025; June 6-7; Nashville, United States. Silverberg J, et al. Nemolizumab with concomitant topical therapy in adolescents and adults with moderate-to-severe atopic dermatitis (ARCADIA 1 & 2): results from two replicate double-blinded, randomised controlled phase 3 trials. Lancet. 2024;404(10451):445-460. doi: 10.1016/S0140-6736(24)01203-0 Ständer S, et a. Nemolizumab long-term efficacy and safety up to 100 weeks in the OLYMPIA open-label extension study in patients with prurigo nodularis: An interim analysis. Presented at International Congress of Dermatology; June 18-21, 2025; Rome, Italy. A Study to Assess the Efficacy and Safety of Nemolizumab (CD14152) in Participants With Prurigo Nodularis (PN) (NCT04501679). Available online. Accessed May 2025 Study to Assess the Efficacy and Safety of Nemolizumab (CD14152) in Participants With Prurigo Nodularis (PN) (NCT04501666). Available online. Accessed May 2025 Jimenez SA, Mendoza FA, Piera-Velasquez S. A review of recent studies on the pathogenesis of Systemic Sclerosis: focus on fibrosis pathways. Front Immunol. 2025;16: 1551911. doi: 10.3389/fimmu.2025.1551911 Truchetet ME, et al. Current Concepts on the Pathogenesis of Systemic Sclerosis. Clin Rev Allergy Immunol. 2021;64(3): 262–283. doi: 10.1007/s12016-021-08889-8 Teresa J, et al. Therapeutics in chronic pruritus of unknown origin. Itch. 2023;8(1): pe64. doi: 10.1097/itx.0000000000000064 Andrade E, et al. Interventions for chronic pruritus of unknown origin. CDSR. 2020;1(1): CD013128. doi: 10.1002/ H1 2024 adjustments include 48 M USD for IPO related incentive plans, 5 M USD for platform transformation costs, 4 M USD for VCB bonus, 2 M USD for IPO. H1 2025 adjustments include 4 M USD litigation, 6 M USD onerous items, 2 M USD M&A, 9 M USD impairments, 4 M USD restructuring, 13 M USD for operating FX Core Net Income is defined as net income / (loss) from continuing operations adjusted for the same items that are treated as exceptional for purposes of defining Core EBITDA, as well as amortization of intangible assets, foreign exchange gains and losses on financing activities. Taxes on the adjustments between IFRS net income and Core Net Income take into account, for each individual item included in the adjustment, the tax rate that will finally be applicable to the item based on the jurisdiction where the adjustment will finally have a tax impact Core EPS is calculated as Core net income divided by the weighted average number of outstanding shares Indebtedness includes financial debt and lease liabilities Includes assumptions for other income and expenses related to tangible asset impairments, ongoing litigation and onerous items, restructuring charges and others, excluding M&A fees On reported profit before tax Includes interest income and interest expense, excluding FX impact Of reported net income based on prior year results, subject to Board and AGM approval Includes 13 M USD of Operating FX from H1 2025 Forward-looking statements Certain statements in this announcement are forward-looking statements. Forward-looking statements are statements that are not historical facts and may be identified by words such as "plans", "targets", "aims", " believes", "expects", "anticipates", "intends", "estimates", "will", "may", "continues", "should" and similar expressions. These forward-looking statements reflect, at the time, Galderma's beliefs, intentions and current targets/ aims concerning, among other things, Galderma's results of operations, financial condition, industry, liquidity, prospects, growth and strategies and are subject to change. The estimated financial information is based on management's current expectations and is subject to change. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial consequences of the plans and events described herein. Actual results may differ from those set forth in the forward-looking statements as a result of various factors (including, but not limited to, future global economic conditions, changed market conditions, intense competition in the markets in which Galderma operates, costs of compliance with applicable laws, regulations and standards, diverse political, legal, economic and other conditions affecting Galderma's markets, and other factors beyond the control of Galderma). Neither Galderma nor any of their respective shareholders (as applicable), directors, officers, employees, advisors, or any other person is under any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. You should not place undue reliance on forward-looking statements, which speak of the date of this announcement. Statements contained in this announcement regarding past trends or events should not be taken as a representation that such trends or events will continue in the future. Some of the information presented herein is based on statements by third parties, and no representation or warranty, express or implied, is made as to, and no reliance should be placed on, the fairness, reasonableness, accuracy, completeness or correctness of this information or any other information or opinions contained herein, for any purpose whatsoever. Except as required by applicable law, Galderma has no intention or obligation to update, keep updated or revise this announcement or any parts thereof.
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