格隆汇10月13日丨恒瑞医药(600276.SH)公布，公司子公司广东恒瑞医药有限公司收到国家药品监督管 理局核准签发关于SHR-1905注射液的《药物临床试验批准通知书》，将于近期开展临床试验。SHR- 1905注射液是胸腺基质淋巴细胞生成素（TSLP）单克隆抗体，可以阻断炎症细胞因子的释放，抑制下 游炎症信号的传导，最终改善炎症状态并控制疾病进展。 ...

Financing and Capital Structure - Karyopharm Therapeutics Inc. announced comprehensive financing and capital structure transactions expected to provide $100 million of financial flexibility, extending the cash runway into Q2 2026 [1] - The company is exploring potential financing and strategic alternatives to enhance liquidity and maximize value, with Centerview Partners acting as the financial advisor [2] - A private placement of approximately $8.75 million will involve the sale of 1.49 million shares and warrants to purchase 1.32 million shares at an exercise price of $6.64 per share [2] Revenue Expectations - The company expects total revenue, including license and royalty revenue from partners, to be between $42 million and $44 million, with U.S. XPOVIO net product revenue anticipated to be approximately $32 million for Q3 2025 [3] Clinical Trials - Enrollment continues in the Phase 3 XPORT-EC-042 trial evaluating selinexor as a maintenance-only therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer, with topline data expected in mid-2026 [4] - The company completed enrollment in the Phase 3 SENTRY trial, which evaluates selinexor in combination with ruxolitinib in JAKi-naïve myelofibrosis patients, with topline data anticipated in March 2026 [4][5] Stock Performance - Karyopharm stock is down 12.74% at $5.03 at the time of publication [5]

Core Insights - AstraZeneca's drug baxdrostat has achieved its primary goal in a late-stage clinical trial for treatment-resistant hypertension, significantly lowering blood pressure [1] - The company plans to submit baxdrostat for approval by the end of the year and anticipates annual sales could exceed $5 billion [1] - Additional indications for baxdrostat under clinical research include chronic kidney disease and heart failure prevention [1]

SAN FRANCISCO, Sept. 30, 2025 (GLOBE NEWSWIRE) -- Investors in MoonLake Immunotherapeutics (NASDAQ: MLTX) saw the price of their shares crater $55.75, or about 90%, after the company announced disastrous VELA-2 trial results for sonelokimab, its highly anticipated treatment for patients with skin disease (hidradenitis suppurative or “HS”). The development and severe market reaction has prompted national shareholders rights firm Hagens Berman to open an investigation into whether MoonLake may have misled inv ...

Core Viewpoint - Paradigm Biopharma has initiated its final-stage Phase 3 trial for injectable pentosan polysulfate sodium (iPPS) aimed at treating osteoarthritis in knee joints, with participants being dosed in both Australia and the U.S. [1] Group 1: Trial Progress and Recruitment - A larger-scale recruitment campaign is underway, with current participants considered early patients, and the company is expanding the number of trial sites to maximize recruitment [3] - The company has launched a trial-specific website named 'Hope4OA' to support the recruitment efforts [4] - Nearly all trial sites are expected to be activated and recruiting by the end of October, positioning the company to meet its enrollment targets and deliver an interim analysis by mid-2026 [6] Group 2: Regulatory Feedback and Screening - Following feedback from the FDA, the company has improved its collective screening failure rate for the current Phase 3 trial iteration, named PARA_OA_O12 [4] - The early site start-up focused on comprehensive site initiation and imaging certification to ensure effective screening and enrollment at scale [5]

Summary of MoonLake Immunotherapeutics 2025 R&D Day Company Overview - **Company**: MoonLake Immunotherapeutics - **Focus**: Development of sonelokimab for the treatment of hidradenitis suppurativa (HS) Key Points from the Call Industry and Product Insights - **Trials**: The VELA-1 and VELA-2 trials are part of the VELA program testing sonelokimab in adults with moderate to severe HS, with primary endpoint readout at week 16 [3][4] - **Results**: VELA-1 met all primary and secondary endpoints with a 17% delta to placebo for I-score 75 at week 16, while VELA-2 faced challenges due to a higher-than-expected placebo response [4][5] - **Regulatory Perspective**: The company believes the trials have merit to proceed in clinical development despite VELA-2's issues, emphasizing the importance of pain scores and quality of life improvements [5][6] Clinical Data and Analysis - **Patient Cohort**: A total of 838 patients participated across both trials, with balanced patient characteristics [6][8] - **Statistical Analysis**: The company utilized composite and treatment policy strategies for data analysis, both required by regulators to test robustness [11][12] - **Efficacy**: VELA-1 showed high statistical significance (P < 0.001) for primary endpoints, while VELA-2 had a borderline P-value of 0.053, driven by a single patient [15][17] - **Response Over Time**: Sonelokimab demonstrated a fast onset of response, with significant differences from placebo observed as early as week 4 [19][20] Patient-Reported Outcomes - **Pain Reduction**: Significant pain reduction was observed with sonelokimab compared to placebo, with similar results across both trials [25][26] - **Quality of Life**: The HS-specific quality of life score showed substantial improvement in patients treated with sonelokimab, indicating a meaningful impact on daily living [26][27] - **Safety Profile**: The combined VELA trials indicated a favorable safety profile with no new safety signals, differentiating it from competitors [30][44] Future Directions - **Regulatory Engagement**: The company plans to engage with regulators to clarify the path forward for approval, believing that the data supports the efficacy of sonelokimab [49][50] - **Market Competition**: Confidence in competing against existing therapies is high, with the belief that sonelokimab has a differentiated profile in terms of efficacy, safety, and convenience [51][60] - **Upcoming Presentations**: The company will present VELA data at a scientific conference in Nashville at the end of October [52] Additional Considerations - **Placebo Response**: The unusually high placebo response in VELA-2 is acknowledged, with ongoing investigations to understand its causes [54][55] - **Financial Position**: The company reported a cash position of $425 million, indicating a stable financial outlook despite challenges [63] Conclusion MoonLake Immunotherapeutics is optimistic about the future of sonelokimab in treating HS, with strong clinical data supporting its efficacy and safety. The company is preparing for regulatory discussions and aims to clarify the path to approval while maintaining confidence in its competitive positioning within the market.

Group 1 - The company, Iwubio (300357.SZ), has completed a Phase I clinical study on its developed product "Fumigatus Puncture Solution" to explore its safety and efficacy in Chinese volunteers [1] - The study reported good safety within the set dosage range (55DU/mL-495000DU/mL), with adverse events being classified as Grade 1 or 2, and no serious adverse reactions or events leading to withdrawal [1] - A certain proportion of delayed reactions were observed among subjects, potentially related to the fungal nature of Fumigatus [1] Group 2 - The specificity of the product at all dosages was above 95%, with no dose-related effects observed; however, sensitivity showed an increasing trend with higher dosages, reaching 95% at a dosage of 55000DU/mL [1] - Researchers recommend advancing the product into further clinical studies using dosages of 55000DU/mL and above, while also suggesting exploration of better strategies to reduce delayed reactions from a pharmaceutical perspective before subsequent clinical trials [1]

Group 1 - The company has completed a Phase I clinical study of "Fumigatus Point Injection" in Chinese volunteers, focusing on its safety and efficacy [1] - The clinical trial report indicates that within the set dosage range (55 DU/mL - 495,000 DU/mL), the product demonstrated good safety, with no serious adverse reactions or events leading to withdrawal [1] - A certain percentage of delayed reactions were observed among participants, with sensitivity increasing with dosage, reaching 95% at a dosage of 55,000 DU/mL [1]

Core Viewpoint - The company announced that its subsidiary, Guangzhou Baiyunshan Guanghua Pharmaceutical Co., Ltd., has registered the Phase II clinical trial information for its pediatric Xiao Chai Hu granules on the National Medical Products Administration's clinical trial registration and information disclosure platform [1] Group 1 - The clinical trial is designed to evaluate the efficacy and safety of pediatric Xiao Chai Hu granules in treating pediatric gastrointestinal colds with Shaoyang syndrome through a randomized, double-blind, parallel-controlled, multi-center Phase II clinical study [1] - The common title of the trial is "Phase II Clinical Trial of Pediatric Xiao Chai Hu Granules for Treating Pediatric Gastrointestinal Colds with Shaoyang Syndrome" [1]

Core Viewpoint - Baiyunshan Pharmaceutical Group's BYS10 tablets, a selective RET small molecule inhibitor, have received feedback from the National Medical Products Administration (NMPA) for future clinical trials and potential market application [1] Group 1: Product Development - BYS10 tablets are developed for treating advanced solid tumors, including non-small cell lung cancer (NSCLC) and medullary thyroid carcinoma (MTC) [1] - The formulation specifications for BYS10 tablets are 25mg and 100mg [1] - The clinical trial approval for BYS10 was granted in January 2022, with the first patient signing the informed consent in January 2023 [1] Group 2: Clinical Trial Results - Phase I clinical trial data indicate that BYS10 tablets exhibit strong and durable anti-tumor activity in patients with RET gene mutations across various solid tumors [1] - The drug has shown good clinical activity and tolerability in patients with advanced NSCLC, thyroid cancer, and MTC [1] - Based on the safety and efficacy data from Phase I trials, the NMPA has agreed to the application for a Phase II single-arm clinical trial for BYS10 tablets [1]