August 28, 2025 07:32 PM GMT Investor Presentation | Asia Pacific China BEST Conference: China's Emerging Frontiers – Biotech Ascent M Foundation Morgan Stanley Asia Limited+ Jack Lin Equity Analyst Jack.Lin@morganstanley.com +852 3963-3746 Laurence Tam Equity Analyst Laurence.Tam@morganstanley.com +852 2239-1753 Alexis Yan, CFA Equity Analyst Alexis.Yan@morganstanley.com +852 2239-7953 China Healthcare Asia Pacific Industry View Attractive Morgan Stanley does and seeks to do business with companies covered ...

Core Insights - The US FDA has approved Wayrilz (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who have not responded adequately to previous treatments, based on the successful LUNA 3 phase 3 study [1][3] - Wayrilz is a novel oral Bruton's tyrosine kinase (BTK) inhibitor that targets multiple immune pathways to address the underlying causes of ITP [2][11] - The approval highlights Sanofi's commitment to developing innovative therapies for rare and immunological diseases [3][11] Study Results - The LUNA 3 study involved 202 adult patients and demonstrated that 64% of patients on Wayrilz achieved a platelet count response at 12 weeks compared to 32% in the placebo group [3][10] - Patients on Wayrilz reported a 10.6-point improvement in health-related quality of life measures, while the placebo group showed a 2.3-point increase [4] - Statistically significant results included a durable platelet response at week 25 (23% in Wayrilz vs. 0% in placebo; p<0.0001) and a faster time to first platelet response (36 days in Wayrilz vs. not reached in placebo; p<0.0001) [7] Treatment Implications - Wayrilz offers a new treatment option for patients who have not responded to steroids or existing therapies, potentially improving management of ITP [5][11] - The drug has received Fast Track and Orphan Drug Designations from the FDA for ITP and is under regulatory review in the EU and China [8][12] - Sanofi's HemAssist program will provide support for patients undergoing treatment with Wayrilz, including assistance with access and insurance coverage [9] Company Overview - Sanofi is an R&D driven biopharma company focused on innovative therapies for various diseases, including rare and immunological conditions [13] - The company is committed to leveraging its understanding of the immune system to develop effective treatments and improve patient outcomes [13]

NOVATO, Calif., Aug. 29, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced participation in three upcoming investor conferences. Cantor Global Healthcare Conference 2025 (New York, NY) Thursday, September 4, 2025, Eric Crombez, M.D., Chief Medical Officer and Howard Horn, Chief Financial Officer, will participate in a fireside ...

This little company's scientific breakthrough opened up a whole new sliver of the drug market (although it's still leading the developmental race).Small biopharma story stocks just aren't paying off like they used to. Maybe there are just too many of them, with each one working on a medical breakthrough that's statistically unlikely to even come close to an approval.Every now and then, though, one of these companies defies the odds and gets a new drug on the market. CRISPR Therapeutics (CRSP -2.34%) is one ...

SAN DIEGO, Aug. 29, 2025 (GLOBE NEWSWIRE) -- Robbins LLP reminds stockholders that a class action was filed on behalf of investors who purchased or otherwise acquired Sarepta Therapeutics, Inc. (NASDAQ: SRPT) securities between June 22, 2023 and June 24, 2025. Sarepta is a commercial-stage biopharmaceutical company that focuses on RNA and gene therapies for the treatment of rare diseases. During the class period, Sarepta was engaged in the development of therapies to treat Duchenne muscular dystrophy (“Duch ...

Financial Data and Key Metrics Changes - The meeting reported the election of directors and the approval of an amendment to the stock option plan, indicating a stable governance structure and potential for future growth [7][8]. Business Line Data and Key Metrics Changes - No specific financial data or metrics related to individual business lines were provided during the meeting [9]. Market Data and Key Metrics Changes - The company is currently conducting a Phase I clinical trial with a total of 16 patients treated, indicating ongoing engagement in clinical research [10][11]. Company Strategy and Development Direction - The company is focused on advancing its clinical trials and plans to announce specific results in September, reflecting a commitment to transparency and progress in research [11]. Management Comments on Operating Environment and Future Outlook - Management acknowledged the diverse range of tumor types represented in the clinical trial, suggesting a broad market approach and adaptability in research [10]. - Future announcements regarding trial results are anticipated, which may impact investor sentiment and market perception [11]. Other Important Information - The meeting concluded with a focus on shareholder engagement and the importance of upcoming trial results, highlighting the company's commitment to its stakeholders [12]. Q&A Session Summary Question: How many patients are currently undergoing Transcode clinical studies? - The company reported that 16 patients have been treated in the Phase I clinical trial, with more details expected in September [10][11]. Question: How many have dropped out and for what reasons? Are there patients with demonstrated tumor regressions so far? - The company did not provide specific dropout rates or reasons but confirmed that the trial is open to a wide range of tumor types, indicating a comprehensive approach to patient selection [10].

Core Points - Pasithea Therapeutics Corp. is a clinical-stage biotechnology company focused on developing PAS-004, a next-generation macrocyclic MEK inhibitor for treating RASopathies and MAPK pathway-driven tumors [2] - The company will present at the H.C. Wainwright 27 Annual Global Investment Conference on September 8, 2025, at 5:00 PM ET [1][2] - PAS-004 is currently undergoing a Phase 1 clinical trial for advanced cancer patients and a Phase 1/1b clinical trial for adult patients with neurofibromatosis type 1-associated plexiform neurofibromas [2] Event Details - The presentation will take place at the Lotte New York Palace Hotel during the H.C. Wainwright 27 Annual Global Investment Conference [2] - Management will be available for one-on-one meetings throughout the event [2]

IRVINE, Calif., Aug. 29, 2025 (GLOBE NEWSWIRE) -- CG Oncology, Inc. (NASDAQ: CGON), a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients with bladder cancer, today announced that Arthur Kuan, Chairman & Chief Executive Officer, and Ambaw Bellete, President & Chief Operating Officer, are scheduled to participate in fireside chat presentations at the following investor conferences: Cantor Global Healthcare Confe ...

Core Insights - Alumis Inc. is a late-stage biopharma company focused on developing next-generation targeted therapies for immune-mediated diseases [2] - The company is leveraging a proprietary data analytics platform to create a pipeline of oral tyrosine kinase 2 inhibitors and other therapies [2] - Upcoming investor conferences will feature Alumis, providing opportunities for engagement with investors [1][4] Company Overview - Alumis is developing therapies aimed at improving health outcomes for patients with immune-mediated diseases, including moderate-to-severe plaque psoriasis and systemic lupus erythematosus [2] - The pipeline includes envudeucitinib (formerly ESK-001) and A-005 for various systemic and neuroinflammatory conditions, as well as lonigutamab for thyroid eye disease [2] - The company has several preclinical programs identified through its precision approach [2] Upcoming Events - Alumis will participate in multiple investor conferences in September 2025, including: - Cantor Global Healthcare Conference on September 3 at 9:45 am ET [4] - Wells Fargo 2025 Healthcare Conference on September 5 at 9:30 am ET [4] - Morgan Stanley 23rd Annual Global Healthcare Conference on September 8 at 4:50 pm ET [4] - H.C. Wainwright 27th Annual Global Investment Conference on September 9 at 10:30 am ET [4] - Baird 2025 Global Healthcare Conference on September 10 at 2:35 pm ET [4] - Stifel 2025 Virtual Immunology & Inflammation Forum on September 16 at 12:30 pm ET [4]

Core Viewpoint - OSE Immunotherapeutics is reaffirming its strategic priorities and addressing shareholder concerns ahead of its Annual General Meeting (AGM) scheduled for September 30, 2025, amidst shareholder activism and misinformation campaigns [1][3][4] Company Strategy - The company aims to leverage its two main programs, Tedopi® and Lusvertikimab, through strategic partnerships and financing, positioning itself as a leading European biotech in immunotherapy for oncology and inflammation [2][5] - OSE's strategy focuses on maintaining strategic optionality for its lead assets while balancing near-term financing and value creation to avoid premature dilution of shareholder value [5][6] Shareholder Activism - OSE has faced significant shareholder activism, including a misinformation campaign by minority shareholders, which has created confusion ahead of the AGM [3][8] - The company has initiated legal actions to protect all shareholders and ensure a fair debate, as the activist group has allegedly acted against sound governance principles [8][10] AGM and Legal Proceedings - The AGM has been postponed to September 30, 2025, to ensure fair voting conditions based on accurate information regarding the intentions of the activist shareholders [9][11] - Legal proceedings have been initiated to investigate the actions of minority shareholders, with potential implications for their voting rights if irregularities are confirmed [10][11] Financial and Development Plans - Tedopi® is in a pivotal Phase 3 clinical trial for non-small cell lung cancer, with enrollment completion expected in the second half of 2026 and initial readouts anticipated by the end of 2027 [14] - Lusvertikimab's development includes a Phase 2b trial in ulcerative colitis, expected to start in 2026, with readouts in 2027 and 2028 [14] - The company has clarified that the expected cost for the Lusvertikimab trial is in the tens of millions of euros, significantly lower than misleading figures circulated by activist shareholders [14] Governance and Communication - OSE emphasizes its commitment to transparent governance and has published a Shareholder Q&A to counter misinformation and provide clear information on its strategy and financial trajectory [7][12] - The company is dedicated to maintaining open dialogue with shareholders and encourages them to review the Q&A document for accurate information [13]