Jefferies Global Healthcare Conference June 5, 2025 Forward-looking Statements and Other Important Information This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding Karyopharm's beliefs about the market opportunity and annual peak revenue opportunities for selinexor; expectations with respect to commercialization efforts; the ability of selinexor to treat patients with multiple ...

Data featured in oral presentation and poster session at 48th European Cystic Fibrosis Conference WALTHAM, Mass., June 06, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced the presentation of preclinical data that de ...

Sionna Therapeutics Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or other events to be materially different from any future results, performance or other events expressed or implied by the forward-looking statements. Given these uncertainties, you should not place undue reliance on forward-looking statements. Our actual future results, performance or other events may be materially different from what we expect. Exc ...

SION-719 and SION-451 were generally well tolerated and achieved desired pharmacokinetic targets that reinforce their potential as either an add-on to standard of care or in a Sionna dual combination Positive Phase 1 Results for SION-719 and SION-451 The Phase 1 randomized, double-blind, placebo-controlled clinical trials evaluated the safety, tolerability, and PK profiles of single ascending doses (SAD) and multiple ascending doses (MAD) of SION-719 and SION-451 in healthy volunteers. The effect of food on ...

Core Viewpoint - Gyre Therapeutics has announced a public offering of 2,222,222 shares at a price of $9.00 per share, aiming to raise approximately $20 million to support its clinical trials and operational needs [1][3]. Group 1: Offering Details - The public offering consists of 2,222,222 shares priced at $9.00 each, with an option for underwriters to purchase an additional 333,333 shares [1]. - The gross proceeds from the offering are expected to be around $20 million before deducting underwriting discounts and commissions [1]. - The offering is anticipated to close on or about May 27, 2025, pending customary closing conditions [1]. Group 2: Use of Proceeds - Gyre plans to utilize the net proceeds from the offering, along with existing cash and cash flows, to advance its Phase 2 clinical trial of F351 for liver fibrosis associated with metabolic dysfunction [3]. - The funds will also be allocated for research and development, manufacturing scale-up, working capital, and general corporate purposes [3]. Group 3: Company Background - Gyre Therapeutics is a biotechnology company based in San Diego, focusing on organ fibrosis, particularly liver fibrosis related to metabolic dysfunction [6]. - The company is advancing a pipeline in China through its indirect controlling interest in Gyre Pharmaceuticals, which includes various therapeutic developments [6].

Core Insights - Gyre Therapeutics announced that its lead compound, Hydronidone (F351), successfully met the primary endpoint in a pivotal Phase 3 trial for treating liver fibrosis in patients with chronic hepatitis B in China [1][2][3] Trial Details - The Phase 3 trial was a 52-week, multicenter, double-blind, placebo-controlled study involving 248 patients with CHB fibrosis across 39 hospitals in China [2][12] - Patients were randomized 1:1 to receive either Hydronidone (270 mg/day) or placebo, alongside entecavir antiviral therapy [2][12] - The trial achieved a statistically significant proportion of patients showing a ≥1-stage regression in liver fibrosis with Hydronidone compared to placebo (52.85% vs. 29.84%, P=0.0002) [6][7] Efficacy and Safety Results - Hydronidone demonstrated a statistically significant regression in liver fibrosis and improvement in liver inflammation compared to placebo [5][6] - Key secondary endpoint results showed a ≥1-grade improvement in inflammation (49.57% for Hydronidone vs. 34.82% for placebo, P=0.0246) [6] - The safety profile of Hydronidone was favorable, with serious adverse events occurring in 4.88% of patients compared to 6.45% in the placebo group, and no discontinuations due to adverse events [7][9] Future Plans - Gyre plans to submit a New Drug Application (NDA) to China's NMPA in Q3 2025 for accelerated approval of Hydronidone [7][11] - A Phase 2 trial in the U.S. for MASH-associated liver fibrosis is expected to begin in the second half of 2025 [7][11] Market Opportunity - Approximately 75 million people in China are chronically infected with hepatitis B, with an estimated 2.6 million diagnosed with compensated F2-F4 CHB fibrosis, representing a significant addressable market for Hydronidone [13][14] - Currently, there are no approved anti-fibrotic therapies for CHB fibrosis in China, highlighting the urgent medical need that Hydronidone aims to address [13][14] About Hydronidone - Hydronidone is a structural analogue of Pirfenidone, exhibiting enhanced potency in inhibiting key drivers of liver fibrosis [15][16] - The compound has shown robust anti-fibrotic activity in multiple preclinical models and is positioned to transform the treatment landscape for liver fibrosis [16] Company Overview - Gyre Therapeutics is a biopharmaceutical company focused on developing and commercializing Hydronidone for liver fibrosis, including MASH in the U.S. [18] - The company is advancing a broad pipeline through its indirect controlling interest in Gyre Pharmaceuticals, including therapeutic expansions and development programs [18]

WALTHAM, Mass., May 22, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that preclinical data assessing combinations of Sionna’s nucleotide-binding domain 1 (NBD1) stabilizers, SION-451 and SION-719, with complement ...

Summary of Karyopharm Therapeutics (KPTI) Conference Call Company Overview - **Company**: Karyopharm Therapeutics (KPTI) - **Key Products**: Selinexor (XPOVIO) - **Focus Areas**: Oncology, specifically multiple myeloma and myelofibrosis Key Points Commercial Performance and Product Dynamics - **Q1 Demand Growth**: KPTI reported a 5% year-over-year demand growth for XPOVIO in Q1, despite a one-time impact of $5 million from atypical returns related to high-dose selinexor [3][4] - **Real-World Utilization**: The company is focusing on increasing the utilization of selinexor in both community and academic settings, with a particular emphasis on lower doses [4][5] - **Community Treatment**: Selinexor is primarily used in the community setting for multiple myeloma patients, positioned in the second to fourth line of treatment [5][6] Growth Drivers and Future Outlook - **T Cell Engaging Therapies**: KPTI is generating data to support the use of selinexor in conjunction with T cell engaging therapies, which are becoming more prevalent in earlier treatment lines [9][10] - **Myelofibrosis Opportunity**: The ongoing SENTRI trial is evaluating selinexor in combination with ruxolitinib, with promising early data showing a 79% SVR 35 rate at week 24 compared to 30-35% for ruxolitinib alone [12][13][15] - **Durability of Response**: The company reported a 100% durability of response for patients achieving SVR 35 or TSS 50, indicating sustained benefits from treatment [17] Clinical Trials and Data - **Phase III Trials**: KPTI is focused on executing its Phase III trials for both myelofibrosis and endometrial cancer, with the latter leveraging a biomarker (p53 wild type) for patient selection [45][46] - **Endpoint Changes**: The transition from TSS 50 to absolute TSS is seen as a more sensitive measure for symptom improvement, gaining positive feedback from investigators and regulators [28][29] Safety and Tolerability - **Antiemetic Use**: KPTI has incorporated dual antiemetics in its Phase III trials to mitigate nausea and vomiting, achieving over 85% compliance and lower rates of these side effects [36][38] - **Safety Profile**: The safety profile of selinexor is evolving positively, with improvements noted in gastrointestinal side effects [17][38] Commercial Strategy - **Market Positioning**: KPTI aims to target all frontline myelofibrosis patients, leveraging existing commercial infrastructure to facilitate rapid uptake among physicians [41][42] - **Unmet Need**: Research indicates a significant unmet need in the myelofibrosis market, with 75% of physicians willing to adopt therapies that address key disease hallmarks [42] Financial Discipline - **Operational Focus**: KPTI is maintaining a disciplined approach to operational expenditures, concentrating resources on Phase III readouts and shutting down non-essential programs [48][49] Additional Insights - **Endometrial Cancer Landscape**: The company anticipates that selinexor will address a sizable unmet need in patients with p53 wild type tumors, with enrollment for the Phase III trial progressing well [46][47] - **Future Readouts**: KPTI is positioned for significant upcoming data readouts that could transform treatment paradigms in both myelofibrosis and endometrial cancer [49]

WALTHAM, Mass., April 30, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that management will present at The Citizens Life Sciences Conference on Wednesday, May 7, 2025 at 12:30 p.m. ET. A live webcast of the prese ...

WALTHAM, Mass., April 30, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that management will present at The Citizens Life Sciences Conference on Wednesday, May 7, 2025 at 12:30 p.m. ET. A live webcast of the prese ...