PGN-EDODM1 for DM1 - PGN-EDODM1 targets the pathogenic CUGexp repeats in DM1, aiming to restore correct splicing by liberating MBNL1[29, 30, 31, 32, 33] - In DM1 patient cells, PGN-EDODM1 treatment resulted in a 54% reduction in toxic foci and a 69% correction of mis-splicing[36] - Preclinical studies showed that multiple doses of PGN-EDODM1 led to a 99% correction of myotonia, compared to a single dose[41] - In the FREEDOM Phase 1 trial, a single 10 mg/kg dose of PGN-EDODM1 produced a mean 29% splicing correction[57] PGN-EDO51 for DMD - In a healthy volunteer study, PGN-EDO51 demonstrated exon skipping, with levels reaching 1.4% at Day 10 and 2% at Day 28 in the 15 mg/kg cohort[76] - CONNECT1 study showed that PGN-EDO51 generated encouraging levels of muscle adjusted dystrophin production of 0.70% and total dystrophin production of 0.26% after just 3 months and 4 doses at 5 mg/kg[95] - CONNECT1 study also showed high levels of mean exon 51 skipping of 2.15% after just 3 months and 4 doses at 5 mg/kg[95] EDO Platform - PepGen's EDO platform is designed for nuclear delivery of oligonucleotide therapeutics, showing up to 25X higher nuclear uptake[7, 17, 19] - EDO technology has been shown to increase cellular uptake and endosomal escape up to 24-fold[21]

Company Presentation June 2025 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking sta ...

Corporate Presentation June 2025 Forward-Looking Statements and Topline Data Disclaimer The material in this presentation (this "Presentation") regarding Kezar Life Sciences, Inc. ("Kezar") is for informational purposes only. This Presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "should," "expect," "believe", "plan" and similar expressions (as well as other words or expressions referencing future events, ...

Financial Performance & Goals - In fiscal year 2021, The Toro Company (TTC) achieved net sales of $3.96 billion[19] - The company aims to exceed $5 billion in net sales by the end of fiscal year 2024 through organic growth[60] - The company also aims to exceed $750 million in annual adjusted operating earnings by the end of fiscal year 2024[60] - For the second quarter of fiscal year 2022, net sales reached $1.25 billion, an increase of 8.7% compared to the same period in fiscal year 2021[71] - For the first six months of fiscal year 2022, net sales were $2.182 billion, a 7.9% increase year-over-year[72] - The company anticipates net sales growth of 14% to 16% for fiscal year 2022[76] Segment Breakdown (Fiscal Year 2021) - Professional segment accounted for 74% of net sales, totaling $2.93 billion, with segment earnings at 17.3%[20] - Residential segment accounted for 26% of net sales, totaling $1.01 billion, with segment earnings at 12.0%[20] - In terms of geographic market, 79% of sales were from the United States, and 21% were international[20] Capital Allocation & Returns - The company targets a gross debt-to-EBITDA ratio of 1-2x[80] - The company's return on average invested capital for fiscal year 2021 was 20.8%[86] - The company's return on average equity for fiscal year 2021 was 32.7%[86]

Pipeline Programs - LP-10 (Liposomal tacrolimus) is positioned for market exclusivity in treating Hemorrhagic cystitis (HC), with a well-established mechanism and demonstrated safety in Phase 2a studies[11] - LP-310, an oral rinse formulation of LP-10, targets the Oral Lichen Planus (OLP) market, estimated at USD 980 million in 2028 and projected to reach USD 2.7 billion by 2034[14] - LP-410, an alternative oral rinse formulation of LP-10, is intended for the treatment of oral Graft-Versus-Host Disease (GVHD) and has received FDA Orphan Disease Designation[70] - LP-50 is an intravesical liposomal formulation of checkpoint Inhibitor for the treatment of bladder cancer[74] LP-310 Clinical Trial Results and Market Opportunity - Phase 2a multicenter dose escalation study completion of LP-310 for Oral Lichen Planus is anticipated in 2Q25[15] - LP-310 Phase 2a data shows safety with minimal systemic uptake and statistically significant improvement of efficacy[50] - Oral Lichen Planus (OLP) affects approximately 6 million Americans, presenting a substantial target market with no current FDA-approved pharmacotherapy[14, 19, 53, 86] - Projected annual revenue per customer for LP-310 is $8,000[53] LP-10 Market and Revenue Potential - Hemorrhagic cystitis affects approximately 60,000 individuals annually[13] - The company anticipates annual revenue of $20,000 per patient for LP-10 intravesical instillation[13, 91, 92] - Market penetration of 60,000 patients (45%) for LP-10 could yield $1.2 billion in annual revenue[13, 91, 92]

Corporate Overview - RedHill Biopharma is an emerging U S specialty biopharmaceutical company focused on gastrointestinal, infectious diseases, and oncology[9] - The company has a streamlined U S commercial organization and a robust development pipeline with multiple near-term milestones[9] - As of December 31, 2024, RedHill Biopharma had a cash balance of $4 8 million[18] - For FY24, RedHill Biopharma reported net revenues of $8 0 million and a gross margin of 60 3%[9, 18] - The company's market capitalization is approximately $4 4 million[18] Talicia® - Talicia® is the No 1 branded U S Rx for H pylori and is listed as a first-line option in the ACG guideline[9, 28, 44] - In a Phase 3 study, Talicia® demonstrated 84% eradication of H pylori in the ITT population versus 58% with the active comparator (p<0 0001)[59] - In the confirmed adherent population, Talicia® achieved a 90% eradication rate compared to 65% with the active comparator (p<0 0001)[59] - Talicia® has broad U S managed care coverage, covering 7 out of 10 commercial lives and 6 out of 10 government lives[48] Pipeline Products - Opaganib is under U S Government collaboration for evaluation as a potential medical countermeasure for GI-ARS[12, 75] - RHB-102 has positive results from a first Phase 3 U S study for Gastroenteritis[12] - RHB-104 has positive results from the first Phase 3 "MAP US" study for Crohn's disease[12]

Investment Highlights - The company is focused on a rare and orphan disease product pipeline[9] - The company is on track for First Netherton Syndrome Treatment Approval in 2026[9] - The global Netherton commercial opportunity is in excess of $1 billion[9] Product Pipeline - QRX003 is in pivotal clinical studies for Netherton Syndrome[12] - Clinical trial has been initiated for QRX003 for Peeling Skin Syndrome[12] - Clinical testing for QRX003 for Palmoplantar Keratoderma is expected to commence in the second half of 2025[12] Netherton Syndrome (NS) and QRX003 - Netherton Syndrome affects 6,000 – 8,000 newborns in the US and Europe combined, with up to 30,000 in Quoin Partnered Territories[21] - QRX003 is a topical lotion to be applied twice-daily to whole body surface[26] - Pilot study data showed marked improvements across all measured clinical endpoints with QRX003[31] - Pediatric whole body data showed that one patient experienced zero nightly sleep disturbances for the first time in her life after treatment with QRX003[40] Additional Rare Skin Disorders - Clinical testing in PPK to commence in 2025[50] - First Subject Peeling Skin Data showed the M-IASI decreased from 36 to 12 after 12 weeks[52]

Financial Performance & Growth - 2021年，Crocs 收入达到 23 亿美元，同比增长 67%，相对比2019年增长 88%[14]，调整后的营业利润率达到 30%[11,14] - Crocs 预计到 2026 年收入将达到 60 亿美元以上[11,74] - 2021年，数字化销售额同比增长 48%，相对比2019年增长 122%，占总销售额的 37%[14] - 第四季度，Crocs 收入为 5.87 亿美元，同比增长 43%[26]，调整后的营业利润率从 21% 增长到 29%[26] HEYDUDE Acquisition - Crocs 于 2022 年 2 月 17 日完成了对 HEYDUDE 的收购[53] - Crocs 预计 HEYDUDE 将在 2024 年成为一个超过 10 亿美元的品牌[56,57] - Crocs 预计 HEYDUDE 在收购后的 2022 年收入为 6.2 亿至 6.7 亿美元，如果包括收购前的时间，预计 HEYDUDE 2022 年的收入约为 7 亿至 7.5 亿美元[70,71] 2022 Outlook - Crocs 预计 2022 财年的收入约为 34 亿美元，同比增长 20% 以上[70] - Crocs 预计 2022 年调整后的营业利润率约为 26%[70] - Crocs 预计 2022 年调整后的摊薄后每股收益为 9.70 美元至 10.25 美元[70]

AVTX-009 and HS Market - AVTX-009, an anti-IL-1β mAb, is a lead compound with potential for best-in-class and best-in-disease profile in hidradenitis suppurativa (HS)[7] - The HS market is expected to grow to over $10 billion by 2035[8] - Avalo is enrolling patients in a Phase 2 LOTUS trial for HS, with topline data expected in mid-2026[8] - AVTX-009 has 15x higher affinity than lutikizumab, potentially predictive of higher efficacy and less frequent dosing[10] Financials and Strategy - Avalo expects its cash runway to extend into 2028[8] - As of March 31, 2025, Avalo had approximately $125 million in cash[70] - As of March 31, 2025, Avalo's adjusted market capitalization was $284.5 million, based on an adjusted common share count of 35.5 million and a stock price of $8.01[70] IL-1β and AVTX-009 Advantages - IL-1β gene expression is up to 100x increased in HS lesions compared to healthy skin[26] - AVTX-009 is designed to target the inflammatory driver of Hidradenitis Suppurativa (HS) to address significant unmet need[15] - AVTX-009 has a subcutaneous bioavailability of 73% and a half-life of 19 days[35]

TH104 - Opioid Overdose Prophylaxis - TH104 is a first-in-class buccal film delivery of nalmefene for military use and is NDA fileable[6, 84] - The FDA has confirmed that no additional clinical trials will be required prior to NDA submission for TH104 for its initial indication[7] - Current naloxone autoinjector provides approximately 2-hour protection versus 7-hour fentanyl exposure, necessitating frequent re-dosing[17] - Government acquired 788,000 Kaléo autoinjector units, indicating growing demand for synthetic opioid countermeasures[46] - At 24-hours, Groups A & B in Phase 1 Chronic Liver Disease (CLD) Study had mean declines of 30.7% & 35.2%, respectively in pruritus scores[106] TH023 - Oral Anti-TNFα for Autoimmune Diseases - TH023 is an oral anti-TNFα monoclonal antibody targeting autoimmune diseases and is Phase 1-ready[6, 84] - The oral anti-TNFα market opportunity has the potential to disrupt the $50B+ global autoimmune biologics market[6, 84] - The existing market size generated annually by TNFα inhibitors (e.g., Humira, Remicade) is approximately $47B[58] Strategic & Financial - The company anticipates a clear pathway to NDA submission for TH104 within 12 months of CMC initiation[34] - The global PBC treatment market is valued at approximately $2.5B and is expected to grow[96]