Clinical Trial Results - The SIGNAL-AA Phase 2a trial studied bempikibart in patients with severe or very severe Alopecia Areata [1] - The study included patients with SALT scores between 50 and 100 [15] - At Week 24, patients with baseline SALT 50-100 showed a mean SALT score change of 163% with bempikibart treatment [20] - In patients with baseline SALT 50-95, the mean SALT score change at Week 24 was 245% with bempikibart [20] - Some patients experienced continued response even 7 months post last dose [23, 24] Safety and Tolerability - In the bempikibart group, 70% of participants experienced at least one treatment-emergent adverse event (TEAE) [27] - In the placebo group, 38% of participants experienced at least one TEAE [27] - No Grade 3 or higher related adverse events were reported in the bempikibart group [27] Study Design and Demographics - The study randomized patients in a 3:1 ratio to bempikibart (n=33) or placebo (n=81) [15] - The mean baseline SALT score in the bempikibart group was 749, while in the placebo group it was 819 [16]

NEW YORK, July 04, 2025 (GLOBE NEWSWIRE) -- WHY: Rosen Law Firm, a global investor rights law firm, announces the filing of a class action lawsuit on behalf of purchasers of securities of Sarepta Therapeutics, Inc. (NASDAQ: SRPT) between June 22, 2023 and June 24, 2025, both dates inclusive (the “Class Period”). A class action lawsuit has already been filed. If you wish to serve as lead plaintiff, you must move the Court no later than August 25, 2025. SO WHAT: If you purchased Sarepta securities during the ...

Is it worth it to buy a stock after it experiences significant market gains? The answer is, it depends. Some companies don't have much growth fuel left in the tank after a nice run, while others still boast significant upside potential after juicy gains. It's best to stay away from the former, but the investing wisdom according to which we should "buy low" still applies to the latter.Let's consider two companies that have performed exceptionally well recently but still have excellent long-term prospects: Su ...

Corporate Presentation March 2024 Forward-Looking Statements This presentation contains statements about our future expectations, plans and prospects that constitute forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including risks relating to: both our and our collaborators' ability to successfull ...

Corporate Presentation June 2025 JENNIFER Living with SCA3 JENNIFER Living with SCA3 Participant in the Troriluzole Clinical Study © 2025 Biohaven, Ltd. All rights reserved. Forward-Looking Statement This presentation includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements about Biohaven Ltd. (the "Company") and our planned and ongoing trials for our troriluzole, taldefgrobep alfa, BHV-7000, BHV-2100, BHV-8000, BHV-1300, BHV-1400, ...

CROSSING BARRIERS AND DEFEATING DEGENERATION MAY 2025 ©2025 Denali Therapeutics Inc. DISCLAIMERS Forward-Looking Statements. This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements do not relate strictly to historical or current facts and they may be accompanied by such words as "anticipate," "believe," "could," "estimate," "expected," "forecast," "intend," "may," "plan," "potential," "possible," "futu ...

Corporate Presentation JULY 2023 Forward Looking Statements This presentation (together with any other statements or information that we may make in connection herewith) contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 with respect to Kiniksa Pharmaceuticals, Ltd. (and its consolidated subsidiaries, collectively, unless context otherwise requires, "Kiniksa," "we," "us" or "our"). In some cases, you can identify forward looking statements by terms ...

Financial Performance & Growth - Total revenue for Q1 2025 reached $125 million, representing a 15% growth [6] - Galafold revenue in Q1 2025 was $1042 million, with a 14% patient demand growth [23] - Pombiliti + Opfolda revenue in Q1 2025 was $21 million, showing a 92% growth at CER [50] - The company expects to surpass $1 billion in total revenue in FY 2028 [6] Strategic Priorities & Guidance - The company aims for a total revenue growth of 15-22% at CER in FY 2025 [7, 77] - Galafold revenue is projected to grow by 10-15% at CER in FY 2025 [6, 7] - Pombiliti + Opfolda revenue is expected to increase by 50-65% at CER in FY 2025 [6, 7, 53, 77] - The company anticipates positive GAAP net income during H2 2025 [7, 77] Product & Market Overview - The global Fabry market is projected to reach approximately $3 billion by 2029 [14, 15] - The global Pompe market was approximately $15 billion in 2024 and is expected to exceed $2 billion by 2029 [42, 43] - DMX-200, a Phase 3 program for FSGS, addresses a market affecting over 40000 people in the US [67]

Clinical Programs & Data - CT-0508 monotherapy showed stable disease (SD) in 29% (4/14) of patients, with 44% SD in HER2 3+ patients [13] - 75% (6/8) of HER2 3+ patients exhibited a decrease in ctDNA, with reductions up to 93% [17] - CT-0525, a CAR-Monocyte therapy, has a ~2,000-fold increased exposure compared to CT-0508 in preclinical models [44] - The first patient was treated with CT-0525 in 2Q 2024, with initial data expected in 4Q 2024 [43] In Vivo CAR-M Collaboration with Moderna - The collaboration includes up to 12 targets, with 7 nominated [64] - Carisma received an $80 million upfront payment and is eligible for over $3 billion in potential milestones and royalties [64] - Nomination of the first development candidate targeting GPC3 for HCC triggered a $2 million milestone payment [6, 65] Financial Status - As of June 30, 2024, Carisma had $40.4 million in cash and cash equivalents [100] - The company's cash runway is expected to last into 3Q 2025 [6, 100] Fibrosis Program - Preclinical data demonstrates that engineered macrophages can reduce liver fibrosis, with Relaxin-IL10 macrophages showing >100% reduction in collagen in one model [84] - Nomination of a development candidate for liver fibrosis is expected in 1Q 2025 [8, 93]

BDTX-1535 Clinical Development - BDTX-1535 is a MasterKey therapy targeting families of oncogenic mutations, designed to expand the addressable patient population [4,5,8] - BDTX-1535 is in Phase 2 clinical trials for 2L/3L NSCLC with final data expected in H1 2026, and 1L NSCLC with initial data expected in Q4 2025 [9] - BDTX-1535 is also in Phase 0/1 clinical trials for GBM with initial data in newly diagnosed patients expected in H1 2026 [9] - Preliminary Phase 2 data shows a 42% ORR in recurrent NSCLC patients with PACC-NCM and/or C797S mutations [58] - The company anticipates FDA feedback on the approval pathway for BDTX-1535 in Q4 2025 [84] EGFR Mutations and Treatment Landscape - 23-30% of newly diagnosed EGFRm NSCLC patients carry non-classical mutations (NCMs) [14,17,28] - Real-world data shows that approximately 60% of 1L patients with EGFR-NCM NSCLC receive chemotherapy [21,22,28] - Median time on treatment for 1L NCM NSCLC is less than 8 months [23,28] - BDTX-1535 demonstrates high potency across the spectrum of NCMs and maintains activity where osimertinib loses potency [26,28,32,34] Financial Status - The company ended Q1 2025 with $152.4 million, providing a cash runway into Q4 2027 [5]