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1 US FDA Approval of MIMRYLOTM (rusfertide) for Treatment of Erythrocytosis in Adults with Polycythemia Vera (PV) Dinesh V. Patel, Ph.D. President & CEO Conference Call Monday August 31, 2026 5:30 am PDT / 8:30 am EDT
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Legal Disclaimer This presentation and any accompanying oral presentation contain forward -looking statements within the meaning of the Private Securities Litigation Reform Act. All statements other than statements of historical facts contained in this presentation are forward-looking statements, including statements regarding: our potential receipt of milestone and royalty payments from Takeda and J&J; market potential for MIMRYLOTM (rusfertide) and ICOTYDETM (icotrokinra); actions of the U.S. Food and Drug Administration (“FDA”) and foreign regulatory agencies; timing and results related to our pre-clinical and clinical product candidates (including PN-881, PN-477, PN-458, and PN-8047, among others); and the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates. In some cases, you can identify forward-looking statements by terminology such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potentially,” “predict,” “sh ould,” “will,” or the negative of these terms or other similar expressions. Forward-looking statements are subject to risks and uncertainties, including those discussed in our filings with the Securities and Exchange Commission, including in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Oper ations” sections of our most recently filed periodic reports on Form 10-K and Form 10 -Q and subsequent filings and in the documents incorporated by reference therein. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quant ified and some of which are beyond our control, you should not rely on these forward-looking statements as predictions of future events. The event s and circumstances reflected in our forward-looking statements may not be achieved or occur and actual results could differ materially from those p rojected in the forward- looking statements. The information included in these materials is provided as of the date specified on the cover page of thi s presentation, unless specified elsewhere herein, and is qualified as such. Except as required by applicable law, we undertake no obligation to upd ate any forward-looking statements or other information contained herein, whether as a result of any new information, future events, changed circumst ances or otherwise. This presentation includes products under clinical investigation which have not yet been approved for marketing by the FDA. T hey are currently limited by Federal law to investigational use. No representation is made as to their safety or effectiveness for the purposes for whi ch they are being investigated. All copyrights and trademarks included herein are the property of their respective owners. 2
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Now FDA Approved: MIMRYLO 3 First and Only Hepcidin Mimetic MIMRYLO and the MIMRYLO logo are trademarks of Takeda Pharmaceuticals U.S.A., Inc. Indication • Indicated for treatment of erythrocytosis in adults with polycythemia vera (PV) Polycythemia Vera • Blood cancer characterized by excessive production of red blood cells1 – Elevated hematocrit (Hct)2 – Primary treatment goal: maintain Hct <45%3,4 1. NORD Rare Disease Database, Polycythemia Vera. https://rarediseases.org/rare-diseases/polycythemia-vera/; 2. Spivak JL. Curr Treat Options Oncol. 2018;19(2):12; 3. Marchioli R, et al. N Engl J Med. 2013; 368(1):22-33; 4. Barbui, T, et al. Leukemia. 2018;32(5):1057-69.
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MIMRYLO (Rusfertide, PTG-300): A Hepcidin Mimetic Journey from ‘Concept to Commercial’ US FDA approval Aug ‘26NDA submitted Dec ‘25 Ph3 VERIFY met 1°/ all four key 2° endpoints Mar ‘25 Ph3 VERIFY study (PV) initiated Jan ‘22 Partnership Jan’24 Peptide Platform ‘Scaffold Hopping’ 2012 Ph2 REVIVE study initiated Oct ‘19 Ph2 PACIFIC study initiated Jan ’21 Ph2 REVIVE study met 1° endpoint Mar ‘23 ASCO’25 VERIFY plenary presentation Jun ‘25 NEJM REVIVE publication Feb ‘24 4 Selected for development in PV 2017
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Ongoing Phase 3 VERIFY Study: PV1,2 5 Sustained Hct Control With Fewer PHLs vs. Placebo; Addresses Major Unmet Need in PV Hct, hematocrit; PHL, phlebotomy; PV, polycythemia vera. 1. Kuykendall AT, et al. J Clin Oncol. 2025;43(17_suppl):LBA3; 2. Kuykendall AT, et al. Blood.2025;146(Suppl 1):81. • Primary endpoint: Wks 20-32 1. Response: rusfertide vs placebo (p<0.0001) ✓ • Key 2° endpoints: Wks 0-32 1. Average number of PHLs (p<0.0001) ✓ 2. Proportion of patients with Hct <45% (p<0.0001) ✓ 3. PROMIS Fatigue SF-8a Score ✓ 4. MFSAF Total Symptom Score ✓ VERIFY featured in Plenary Presentation at ASCO’25 *Ongoing therapy could include therapeutic phlebotomy and/or cytoreductive therapy. Inclusion Criteria ≥3 PHL (28 wks prior) OR ≥5 PHL (1 year prior) Part 1B: Open-Label Durability of Response (Wks 32-52) Placebo + ongoing therapy* Part 1A: Double-Blind Rusfertide + ongoing therapy* Rusfertide + ongoing therapy* Dose titration Wks 0-20 1° endpoint Wks 20-32
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MIMRYLO : Highlights of Prescribing Information (Link)1 6 • Efficacy: 76.9% of patients achieved response between Weeks 20-32 – MIMRYLO maintains hematocrit control and reduces phlebotomy burden • Patient reported outcomes (PRO): MIMRYLO improves fatigue in adults with PV – Improvement in fatigue measured by the PROMIS Fatigue Short Form 8a • Safety: no black box warning – Carcinogenicity: Rusfertide was not carcinogenic in a 6-month transgenic mouse study at subcutaneous doses up to 25 mg/kg/week or in a 2-year rat carcinogenicity study at doses up to 3 mg/kg/week. – Warnings and Precautions: New or worsening thrombocytosis, injection-site reactions, and embryo-fetal toxicity. – Adverse reactions: The most common (>15%) adverse reactions were injection site reactions (56%) and anemia (16%). – Discontinuations: Adverse reactions which resulted in permanent discontinuation of MIMRYLO included injection site reactions in 2 (0.7%) patients, thrombocytosis in 2 (0.7%) patients, and anemia in 1 (0.4%) patient. 1. MIMRYLO (rusfertide). Prescribing information. Takeda Pharmaceuticals America, Inc.; 2026. Accessed on 28 August 2026. https://assets- dam.takeda.com/image/upload/v1787954309/Oncology/News/approval-mimrylo/MIMRYLO_NDA_Approved_Labels.pdf
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MIMRYLO : Erythrocytosis-Specific Treatment Option for Adults With PV 7 Peak Revenue Potential of $1-2 Billion4 ~155k prevalence1 ~78k treated patients with PV in US2 ~41k Phlebotomy (PHL) ~26k Hydroxyurea (HU) ~6k Ruxolitinib ~3k Ropeg- interferon And/Or And/Or And/Or 1. Mehta J, et al. Leuk Lymphoma. 2014;55(3):595-600. 2. Komodo Health closed claims dataset (2016-2023). 3. Verstovsek S, et al. Ann Hematol. 2023;102(3):571-81. 4. Takeda 44th Annual J.P. Morgan Healthcare Conference corporate presentation 2026. Treatment Paradigm Patients will cycle through various treatments2 • Unmet need exists at each step of the treatment landscape – ~78% with uncontrolled Hct>45% despite current treatments3 • MIMRYLO expected to be used at each step4 MIMRYLO maintains hematocrit control, reduces phlebotomy burden and improves fatigue
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ICOTYDE (icotrokinra); Oral IL-23R Antagonist; Partner: J&J $387.5M Payments earned $580M Potential future milestones 6–10% Tiered worldwide royalties >$10B3 ICOTYDE peak sales potential FDA Approved Mar ’26 (Mod to Sev Plaque PsO) · PsA Ph3 · UC Ph3 · CD Ph2/3 · EMA (CHMP) positive opinion: Jul ’26 · China (NMPA) Approved Aug ‘26 7.25% weighted-avg royalty at $4B; 10% for annual sales ≥$4B MIMRYLO (rusfertide); SC Hepcidin Mimetic; Partner: Takeda $800M Payments earned $875M Potential future milestones 14–29%1 Tiered worldwide royalties $1–2B2 Takeda-guided peak sales FDA Approved Aug ‘26 (PV) · Ph3 VERIFY PV study met primary + all 4 key secondary endpoints 21% weighted-avg royalty at $1.5B; 29% for annual sales ≥$1.5B Takeda and J&J Partnership Economics 8 1. 1% royalty payable to Zealand Pharma; 2. Takeda 44th Annual J.P. Morgan Healthcare Conference corporate presentation 2026; 3. HSBC Dec 2025 Consensus Forecast (across all indications). ICOTYDE is a trademark of Johnson & Johnson and its affiliates. ~$1.2B in upfront, milestone, and opt-out payments earned through August 2026
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Strong Financial Position and Anticipated Future Cash Flows 9 Supports Aggressive Development of our R&D Portfolio and Potential Capital Return $849.5M CASH, CASH EQUIVALENTS & MARKETABLE SECURITIES as of June 30, 2026 $275M ADDITIONAL PAYMENTS TRIGGERED UPON MIMRYLO APPROVAL $200M remaining opt-out payment and $75M FDA approval milestone POTENTIAL FUTURE CASH INFLOWS FROM PARTNERSHIPS ~$1.5B Future milestones + 6-10% royalties on ICOTYDE + 14-29% royalties on MIMRYLO
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Revenue Engine Drives Pipeline Development 10 Targeting Combined Addressable Markets >$100 Billion Commercial & Partnered Inflammation & Immunology Metabolic ICOTYDE (icotrokinra) Oral IL-23R Antagonist • Partner: J&J FDA Approved: Mar ’26 EMA (CHMP) Positive Opinion: Jul ’26 China (NMPA) Approved: Aug ‘26 Moderate-to-Severe Plaque Psoriasis 6–10% royalties + milestone payments >$10B peak potential 1 MIMRYLO (rusfertide) SC Hepcidin Mimetic • Partner: Takeda FDA Approved: Aug ’26 Polycythemia Vera 14–29% royalties + milestone payments $1-2B peak sales2 Oral PN-881 · IL-17A/F Ph1 completed Ph2b initiation in psoriasis: Q1 ’27 Potential Indications: PsO, PsA, HS, SpA Oral IL-4Rα Antagonist Lead optimization Potential Indications: AD, Asthma Oral & SC PN-477 · GLP/GIP/GCG SC Ph1 ongoing; Oral Ph1: 1H ’27 Oral PN-458 · GLP/GIP IND enabling studies Amylin Mono/Poly-Agonists Lead optimization Revenue Engine Wholly-Owned Pipeline Oral PN-8047 · Hepcidin Mimetic Ph1: Q1 ’27 Potential Indication: Polycythemia Vera Hematology 1. HSBC Dec 2025 Consensus Forecast (across all indications); 2. Takeda 44th Annual J.P. Morgan Healthcare Conference corporate presentation 2026.
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Thank you 11