Puretech Health(PRTC) - 2024 Q4 - Annual Report

Executive Summary & Company Highlights PureTech Health achieved significant clinical, regulatory, and financial milestones in 2024, securing FDA approval and maintaining a strong balance sheet Overview of 2024 Achievements PureTech Health achieved significant clinical, regulatory, and financial milestones in 2024, including positive Phase 2b results for LYT-100, FDA approval of Cobenfy™, and rapid growth of Seaport Therapeutics, maintaining a robust balance sheet into at least 2027 - PureTech's innovation engine drove meaningful clinical, regulatory, and financial milestones in 2024[2] - Key achievements include positive Phase 2b results for wholly-owned deupirfenidone (LYT-100) in IPF and compelling Phase 1b data for wholly-owned LYT-200 in AML and solid tumors[2] - FDA approval of PureTech-invented Cobenfy™ for schizophrenia and rapid growth of Founded Entity Seaport Therapeutics, which raised over $325 million, were also significant[2] Cash, Cash Equivalents, and Short-Term Investments | Metric | As of Dec 31, 2024 | As of Mar 31, 2025 | | :------------------------------------------------ | :------------------- | :------------------- | | PureTech level cash, cash equivalents, and short-term investments | $366.8 million | $339.1 million | | Consolidated cash, cash equivalents, and short-term investments | $367.3 million | $339.5 million | - The company has an operational runway into at least 2027[2][26] Webcast and Conference Call Information PureTech Health plc will host a webcast and conference call on April 30, 2025, at 9:00 am EDT / 2:00 pm BST to discuss its annual results - A webcast and conference call will be held on April 30, 2025, at 9:00 am EDT / 2:00 pm BST to discuss the results[4] - Live webcast and presentation slides are available on PureTech's investor relations website under 'Events and Presentations'[4] - Dial-in numbers for the UK and US, along with a global access code (018948), are provided for phone participation[4] Operational Highlights PureTech's wholly-owned programs and Founded Entities achieved significant clinical, regulatory, and financial milestones in 2024 and early 2025 Wholly-Owned Programs PureTech's wholly-owned programs, deupirfenidone (LYT-100) and LYT-200, demonstrated significant clinical progress in 2024 and early 2025 - PureTech's wholly-owned programs, deupirfenidone (LYT-100) and LYT-200, delivered significant clinical and regulatory milestones[10] Deupirfenidone (LYT-100) Deupirfenidone (LYT-100) achieved positive Phase 2b results in IPF, showing potential to stabilize lung function, with plans for a Phase 3 trial by year-end 2025 - Positive topline results from the ELEVATE IPF Phase 2b clinical trial were announced in December 2024, meeting primary and key secondary endpoints[10] - The higher dose (825 mg TID) demonstrated the potential to stabilize lung function over 26 weeks, with an effect size 50% greater than pirfenidone[10] - Preliminary data from the ongoing open label extension (OLE) study indicate sustained slowing of lung function decline through 52 weeks[10] - PureTech plans to meet with the FDA before the end of Q3 2025 to discuss Phase 2b results and aims to initiate a Phase 3 trial by the end of 2025[10] Gallop Oncology (LYT-200) LYT-200, advanced by Gallop Oncology, showed promising clinical activity and a favorable safety profile in AML/MDS and head and neck cancers, receiving FDA Fast Track and Orphan Drug designations - LYT-200 (anti-galectin-9 mAb) is being advanced for hematological malignancies (AML, MDS) and solid tumors (head and neck cancers)[16] - In an ongoing Phase 1b trial for relapsed/refractory AML and MDS, LYT-200 showed a favorable safety profile and promising clinical efficacy, including complete and partial responses[16] - The Phase 1b trial for solid tumors (head and neck cancers) was completed, demonstrating a favorable safety profile, disease control, and initial efficacy signals, including a complete response lasting over two years[16] - LYT-200 received FDA Fast Track designation for AML (January 2025) and recurrent/metastatic head and neck cancer (March 2024), and Orphan Drug designation for AML (February 2024)[16] Founded Entities PureTech's Founded Entities achieved significant milestones, including FDA approval of Cobenfy™ for schizophrenia, triggering $29 million in milestone payments and future royalties - PureTech's Founded Entities continued to advance their programs, securing regulatory approvals, significant financings, and clinical progress[5] Karuna Therapeutics (Cobenfy™) Cobenfy™ (formerly KarXT) received FDA approval in September 2024 for schizophrenia, triggering $29 million in milestone payments and future royalties - Cobenfy™ (formerly KarXT) received FDA approval in September 2024 for the treatment of schizophrenia in adults[13] - The FDA approval triggered $29 million in milestone payments to PureTech[13] - PureTech is entitled to potential future milestone payments and approximately 2% royalties on net annual sales over $2 billion[13] Seaport Therapeutics Seaport Therapeutics launched with a $100 million Series A financing, followed by a $226 million Series B, totaling $326 million raised since April 2024 - PureTech launched Seaport Therapeutics with a $100 million oversubscribed Series A financing[14] - This was followed by a $226 million oversubscribed Series B financing, bringing the total capital raised by Seaport to $326 million since April 2024[14] - Seaport is advancing novel neuropsychiatric medicines powered by the Glyph platform[14] Vedanta Biosciences Vedanta Biosciences initiated a pivotal Phase 3 study for VE303 and anticipates Phase 2b results for VE202 in 2025 - Vedanta enrolled the first patient in the pivotal Phase 3 RESTORATiVE303 study of VE303 for the prevention of recurrent C. difficile infection (rCDI) in May 2024[17] - Additional results from the VE303 Phase 2 CONSORTIUM clinical trial were published in Nature Medicine in January 2025[17] - Vedanta anticipates topline results from its Phase 2b clinical trial of VE202 in ulcerative colitis in 2025[17] Vor Biopharma Vor Biopharma continued clinical progress with trem-cel and VCAR33ALLO, receiving Fast Track and Orphan Drug designations - Vor continued to progress its Phase 1/2 VBP101 study of trem-cel in AML and MDS, showing durable engraftment and shielding from Mylotarg toxicity[15] - Vor received supportive feedback from the FDA regarding a registrational clinical trial design for trem-cel[15] - In 2024, Vor dosed the first patient in VBP301, a Phase 1/2 study of VCAR33ALLO (CAR-T cell therapy) in relapsed/refractory AML, which received Fast Track and Orphan Drug designations[18] Company Information This section provides an overview of PureTech Health, its forward-looking statements, and contact information About PureTech Health PureTech Health is a clinical-stage biotherapeutics company focused on developing new classes of medicine for devastating diseases, leveraging a hub-and-spoke R&D model - PureTech is a clinical-stage biotherapeutics company dedicated to developing new medicines for devastating diseases[26] - Its R&D engine has developed 29 therapeutic candidates, with three having received FDA approval[27] - Programs are advanced both internally and through Founded Entities, utilizing a hub-and-spoke model[26] [Cautionary Note Regarding Forward-Looking Statements](index=5&type=section&id=Cautionary%20Note%20Regarding%20Forward-Looking%20