Sapablursen

BFC Capital Market Update (March 8 - March 14) Market Overview Global Markets Mixed, U.S. Stocks Decline While China’s Healthcare Sector Outperforms Global equity markets saw mixed performances last week. In China, the Hong Kong Hang Seng Index declined by 1.1%, while Shanghai Composite and CSI 300 gained 1.4% and 1.46%, respectively. Meanwhile, U.S. markets extended their losses, with the Dow Jones Industrial Average dropping 3.1%, the NASDAQ Composite declining 2.4%, and the S&P 500 losing 2.3% over the week. In the healthcare sector, Hong Kong's Hang Seng Healthcare Index (HSHCI) outperformed, rising 1.8%. while the CITIC Healthcare Index advanced by 1.9%. In contrast, the Nasdaq Biotechnology Index (NBI) fell by 1.3%. Major Industry Updates Shares of Grand Pharmaceutical Group Surge on Strong Earnings Growth Last week, shares of Grand Pharmaceutical Group (HKEX: 0512, “the Group”) rose 14.0%, driven by its strong annual earnings report released on March 12. In 2024, the Group achieved revenue of HK$11.64 billion, representing a year-on-year increase of approximately 10.6%. Net profit attributable to shareholders reached approximately HK$2.47 billion, marking a year-on-year increase of approximately 31.3%. A standout performance came from the Group's radio medicine anti-tumor diagnosis and treatment segment, which generated revenue of approximately HK$589 million in 2024—an impressive 176.6% year-over-year growth. The Group highlighted the rapid market expansion of its core product, Yigan Tai® (Yttrium-90 [90Y] Microspheres Injection), as a key driver of this growth. Insilico Medicine Secures $110M Series E to Advance AI-Driven Drug Discovery Innovation On March 13, Insilico Medicine, a clinical-stage generative AI-driven biotech company, announced the completion of a $110 million Series E funding round. The investment was co-led by the private equity fund under Value Partners Group (HKG: 0806), one of Asia's largest independent asset management firms, with strong participation from industry- and technology-focused new investors, as well as continued support from global existing backers. The funds will be directed to enhance Insilico's AI platform, upgrade its state-of-the-art automated laboratory, and advance its drug pipeline, including its lead candidate for idiopathic pulmonary fibrosis (IPF). This investment is expected to accelerate innovation in biopharmaceutical R&D. Business Development Updates Last week featured several notable collaborations in the biopharma sector. Salipro Biotech and Boehringer Ingelheim entered a research and license agreement to accelerate drug discovery efforts targeting GPCRs, ion channels, and other membrane proteins, leveraging Salipro’s proprietary stabilization platform. MeiraGTx partnered with Hologen AI to apply AI-driven insights for the expedited Phase 3 development of AAV-GAD gene therapy for Parkinson’s disease. Roche and Zealand Pharma announced an exclusive collaboration to co-develop and co-commercialize petrelintide, an amylin analog for obesity, alongside Roche’s CT-388 incretin asset. Ono licensed sapablursen from Ionis Pharmaceuticals for polycythemia vera, strengthening its hematology portfolio. Finally, Johnson & Johnson teamed up with Nosis Bio to design AI-enabled RNA delivery vehicles targeting hard-to-reach cell types in chronic diseases. Collectively, these transactions underscore the industry’s ongoing push for precision therapies, AI-driven innovation, and expanded pipeline capabilities. Salipro Biotech and Boehringer Ingelheim Enter Research and License Agreement to Accelerate Multiple Boehringer Ingelheim Pipeline Programs March 13, 2025 – Salipro Biotech AB, a Swedish biotech company specialising in the stabilisation of challenging drug targets, announced a research and license agreement with Boehringer Ingelheim to accelerate the development of multiple drug targets. This collaboration aims to advance the discovery and development of new therapeutic solutions targeting G protein-coupled receptors (GPCRs), ion channels, transporters and other integral membrane proteins in therapeutic areas such as mental health and cardio-renal-metabolic diseases. The partnership leverages Salipro Biotech’s proprietary Salipro® platform technology, which stabilises membrane proteins in their native forms, enabling their use in drug discovery programs. Boehringer Ingelheim will conduct biophysical and structural studies, including cryo-electron microscopy (cryoEM), on Boehringer pipeline targets enabled by the Salipro®-technology to identify potential therapeutic candidates. MeiraGTx Enters into a Strategic Collaboration with Hologen AI to Expedite Phase 3 Development of AAV-GAD for Parkinson’s Disease and Industrialize MeiraGTx’s Proprietary Manufacturing Process March 13, 2025 – MeiraGTx Holdings plc, a vertically integrated, clinical stage genetic medicines company, announced a broad strategic collaboration with Hologen Limited, a world-leading developer of multi-modal generative AI foundation models of real-world clinical data for clinical medicine and pharmaceutical drug development. Roche enters into an exclusive collaboration & licensing agreement with Zealand Pharma to co-develop and co-commercialise petrelintide as a potential foundational therapy for people with overweight and obesity March 12, 2025 – Roche announced that it has entered into an exclusive collaboration and licensing agreement with Zealand Pharma (Nasdaq Copenhagen: ZEAL) (CVR-no. 20045078). Under the terms of this agreement, the two companies will collaborate to co-develop and co-commercialise petrelintide, Zealand Pharma’s amylin analog as a standalone therapy as well as a fixed-dose combination with Roche’s lead incretin asset CT-388. Ono Enters into License Agreement with Ionis Pharmaceuticals for Sapablursen for the Treatment of Polycythemia Vera March 12, 2025 – Ono Pharmaceutical Co., Ltd. announced that it has entered into a license agreement with Ionis Pharmaceuticals, Inc. (Carlsbad, CA,U.S.; CEO, Brett P. Monia, Ph.D.; “Ionis”) for sapablursen, an investigational RNA-targeted medicine for the treatment of polycythemia vera (PV). Sapablursen is currently being evaluated in adult patients with PV in the fully enrolled Phase 2 IMPRSSION study. Sapablursen was granted Fast Track designation in January 2024 and orphan drug designation in August 2024 by the U.S. Food and Drug Administration (FDA). Nosis Bio Enters into Strategic Collaboration with Johnson & Johnson for the Design and Development of Cell-Targeted Delivery Vehicles March 11, 2025 – Nosis Biosciences, an AI-enabled biotechnology company focused on the design and development of RNA medicines for challenging cell types, announced that it has entered into a strategic collaboration with Johnson & Johnson.* This collaboration combines the drug discovery and clinical development expertise from Johnson & Johnson with Nosis’ Connexa™ platform, an AI-powered drug design and delivery platform, to enable extra-hepatic therapeutic access to multiple and challenging cell types implicated in chronic disease. References： 1.https://staticpacific.blob.core.windows.net/press-releases-attachments/3536927/HKEX-EPS_20250312_11566861_0.PDF 2.https://www.prnewswire.com/news-releases/insilico-medicine-secures-110-million-series-e-financing-to-advance-ai-driven-drug-discovery-innovation-302401040.html

Takeda's narrowed focus on oncology, Sun Pharma's proposed acquisition of Checkpoint Therapeutics and Ono's antisense oligonucleotide deal with Ionis made our news this week. Takeda is sharpening its oncology focus amid an R&D revamp. Sun Pharma will acquire Checkpoint Therapeutics and an FDA-approved PD-L1 inhibitor. Japan's Ono Pharmaceutical bought an antisense oligonucleotide from Ionis Pharmaceuticals. Plus more.1. 'I don't feel we lost momentum': Takeda’s oncology execs sharpen focus after restructuringTakeda likely won’t do any cell therapy development deals in the near future, even though it’s still advancing an allogeneic gamma delta T-cell therapy in acute myeloid leukemia. Takeda’s oncology leaders Teresa Bitetti and P.K. Morrow, M.D., explained the company’s cancer drug strategy in an interview. In addition, the Japanese pharma is not counting out China as a source for partnering despite geopolitical risks. 2. On the heels of FDA nod for cancer drug, Sun Pharma buys Checkpoint Therapeutics for up to $416MSun Pharma has agreed to buy Checkpoint Therapeutics for $355 million upfront, plus a contingent value right that could bring the deal value to about $416 million. The acquisition bolsters Sun’s oncology-dermatology franchise with Unloxcyt, a PD-L1 inhibitor recently approved by the FDA for advanced cutaneous squamous cell carcinoma.3. Ono pays $280M upfront for Ionis' antisense oligonucleotide for rare blood cancerOno Pharmaceutical is paying $280 million upfront to acquire Ionis Pharmaceuticals’ antisense oligonucleotide sapablursen. The drug is designed to reduce the production of the TMPRSS6 protein and is currently in a phase 2 trial for the rare blood condition of polycythemia vera. The pact includes up to $660 million in potential milestones.4. J&J, Legend to invest $150M in Belgium cell therapy facility as new Carvykti rival loomsLegend Biotech and its partner Johnson & Johnson will invest $150 million to expand their so-called Tech Lane cell therapy facility in Belgium. The plant is slated to begin clinical production of Carvykti in a few weeks. The partners are further expanding manufacturing capacity even as Gilead Sciences and Arcellx’s rival BCMA CAR-T anito-cel is expected to enter the market in 2026.5. Illumina aims to cut $100M in costs following China import banAs China bans Illumina’s importation of DNA sequencers in retaliation to new U.S. tariffs, the company is looking to cut $100 million in expenses this year. Last year, Illumina counted about 7% of its sales, or about $300 million, from China. The cost reduction “includes optimizing stock-based compensation and non-labor spending and accelerating certain productivity measures,” Illumina said.6. Insilico Medicine raises $110M for new trials and robotic helping handsHong Kong-based Insilico Medicine has raised $110 million to advance its artificial-intelligence-powered drug design platforms. The series E round, led by Value Partners, will help refine Insilico’s AI models as well as support pivotal clinical trials of the company’s lead drug candidate, rentosertib (ISM001-055), in idiopathic pulmonary fibrosis.7. Frazier arms Callio with $187M series A to fly Hummingbird's HER2 dual payload ADC to clinicSingapore’s Hummingbird Bioscience has created a new company together with its investor Frazier Life Sciences and a few other venture capital funds. The new firm, called Callio Therapeutics, has launched with a $187 million series A and a phase 1-ready HER2-targeted dual-payload antibody-drug conjugate licensed from Hummingbird.Other News of Note:8. Daiichi taps Nosis to deliver RNA therapies beyond the liver9. Harbour BioMed launches new obesity biotech on mission to preserve muscle mass10. Kelun's TROP2 ADC picks up another approval in China11. Indian contractor Syngene throws down $37M for Emergent's Baltimore biologics plant12. Nasdaq stock market plans move to 24-hour trading in US13. Samsung backs Alzheimer’s blood test developer C2N Diagnostics with $10M14. Astellas, Yaskawa cement deal for robotic cell therapy production joint venture15. Otsuka debuts new kid-friendly resources about Alzheimer's to ease burden on 'sandwich generation' caregivers