Key Takeaways Shares of Krystal Biotech (KRYS) gained 3.9% yesterday after the company announced that it had dosed the first patient in a phase I/II study evaluating its eye drop KB801 for the treatment of neurotrophic keratitis (NK), a rare, progressive corneal disease. KB801 is an investigational, redosable eye drop gene therapy developed to offer a novel treatment approach for NK. The therapy is designed to promote the sustained, localized production and secretion of nerve growth factor (NGF) by epitheli ...

Core Insights - Iovance Biotherapeutics Inc. is undergoing a leadership transition with the resignation of CFO Jean-Marc Bellemin, effective July 10, 2025, amid commercial challenges and a class-action lawsuit [1][2][3] Leadership Transition - CFO Jean-Marc Bellemin's resignation aligns with his executive employment agreement and comes at a critical time for the company, which has not yet named a successor [2] - Bellemin has been in the CFO role since November 2020 [1] Commercial Challenges - Iovance's flagship drug, Amtagvi, has faced significant commercial setbacks, with first-quarter sales falling short of Wall Street expectations, leading to a revision of full-year revenue projections [3][6] - Following the disappointing sales figures and revenue guidance, Iovance's stock price dropped over 44%, from $3.17 to $1.75 per share within a day [7] Legal Issues - The company is defending against a class-action securities fraud lawsuit, which alleges misleading statements regarding the operational readiness of its authorized treatment centers (ATCs) crucial for Amtagvi's administration [4][5] - The lawsuit claims that ATCs have not been able to initiate treatments in a timely manner, leading to high patient drop-off rates [5] Market Reaction - The market reacted negatively to the company's underwhelming performance and the subsequent legal challenges, indicating investor concerns about Iovance's commercial execution and transparency [6][7] - Hagens Berman, a law firm, is investigating the allegations and the accuracy of Iovance's public statements regarding Amtagvi [8][9]

Key Takeaways Shares of Rhythm Pharmaceuticals (RYTM) were up 36.6% yesterday after it reported positive top-line data from a phase II study evaluating the investigational oral melanocortin-4 receptor (MC4R) agonist, bivamelagon (formerly LB54640), in patients with acquired hypothalamic obesity. Acquired hypothalamic obesity is a severe and debilitating condition caused by hypothalamic damage, often resulting from brain tumors, their treatment or other injuries. This leads to rapid weight gain, uncontrollab ...

Key Takeaways Shares of KalVista Pharmaceuticals (KALV) have soared 36% in the past week, all thanks to the FDA's approval of its lead pipeline drug for the hereditary angioedema (HAE) indication. Earlier this week, the FDA approved the company's novel plasma kallikrein inhibitor, sebetralstat, for the treatment of acute (sudden) HAE attacks in individuals aged 12 years and older. The drug will be marketed under the brand name Ekterly. This latest approval makes Ekterly the first and only oral on-demand the ...

Ultragenyx Pharmaceutical Inc. RARE and Mereo BioPharma Group plc MREO stocks are plunging on Thursday.What Happened?The companies on Wednesday announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is progressing toward a final analysis around the end of the year, consistent with the original plan.OI, also known as brittle bone disease, is a group of genetic disorders that mak ...

Core Viewpoint - Ascentage Pharma's novel Bcl-2 selective inhibitor, lisaftoclax (APG-2575), has received conditional approval from China's National Medical Products Administration (NMPA) for treating adult patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) who have previously undergone at least one systemic therapy, marking it as the first Bcl-2 inhibitor approved for this indication in China and the second globally [1][3][7] Company Overview - Ascentage Pharma is a global biopharmaceutical company focused on addressing unmet medical needs in cancer treatment, with a rich pipeline of innovative drug candidates, including inhibitors targeting key proteins in the apoptotic pathway [10][11] - The approval of lisaftoclax represents a significant milestone in the company's development efforts, following its first commercialized drug, olverembatinib [2][10] Product Details - Lisaftoclax is an orally administered small-molecule Bcl-2 selective inhibitor designed to restore normal apoptosis in cancer cells by blocking the antiapoptotic protein Bcl-2 [2][5] - The drug has demonstrated broad therapeutic potential in various hematologic malignancies and solid tumors, particularly in CLL/SLL, both as a monotherapy and in combination therapies [2][3] Clinical Study Insights - The approval was based on a pivotal Phase II study (APG2575CC201) that evaluated the efficacy and safety of lisaftoclax in patients with relapsed or refractory CLL/SLL, achieving a compelling overall response rate (ORR) that met the prespecified endpoint [3][4] - Lisaftoclax exhibited a favorable safety profile, with no occurrences of tumor-lysis syndrome (TLS) and manageable hematologic and non-hematologic toxicities [3][4] Market Context - CLL/SLL is a common hematologic malignancy, particularly affecting older populations, with over 100,000 new diagnoses globally each year [4] - In China, the incidence of CLL/SLL is rising, necessitating safer and more effective treatment options beyond the current primary therapies, such as BTK inhibitors, which have limitations including high relapse rates and associated toxicities [4][5][6] Future Development Plans - Ascentage Pharma is conducting four global registrational Phase III trials for lisaftoclax, including studies in combination with BTK inhibitors and in various hematologic malignancies [7][13] - The company aims to accelerate the global development of lisaftoclax to address unmet clinical needs and expand its therapeutic applications [7][13]

Illumina was also recognized as one of the Best Companies to Work For byU.S. News & World ReportSAN DIEGO, July 10, 2025 /PRNewswire/ -- Illumina Inc. (NASDAQ: ILMN) today announced two awards recognizing the company's leadership in sustainability practices and as an employer of choice. TIME named Illumina to its World's Most Sustainable Companies list for the second year in a row, and U.S. News & World Report named Illumina to its Best Companies to Work For list. The awards highlight the company's continue ...

Core Insights - NeOnc Technologies Holdings, Inc. has received formal approval from its Board of Directors for a strategic transaction with Quazar Investment, marking a significant step towards closing the deal [1][2][3] Company Overview - NeOnc Technologies is a clinical-stage biotechnology company focused on developing treatments for brain and central nervous system cancers, utilizing its NEO™ drug development platform [5] - The company’s proprietary chemotherapy agents, including NEO100™ and NEO212™, are currently in Phase II clinical trials and have received FDA Fast-Track and Investigational New Drug (IND) status [5] Strategic Partnership - The Board's unanimous approval allows NeOnc to participate in a $50 million equity investment and expansion into the MENA region under a non-binding term sheet with Quazar [2][3] - Quazar plans to lead a capital formation round priced at $25 per share, with 70% of proceeds allocated to acquiring NeOnc common stock and 30% for clinical trials and infrastructure development in the UAE and MENA region [3] Transaction Milestones - The transaction requires NeOnc to meet five milestones, with the current Board approval being the first of these [2][4] - NeOnc has 120 days to satisfy the remaining conditions necessary for closing the transaction [4] Future Developments - The strategic partnership aims to establish a strong operational presence in the MENA region and accelerate the clinical development of NeOnc's core assets [3] - Key future actions include the legal formation of NuroMENA and NuroCure in Abu Dhabi, execution of a Sub-License Agreement, finalization of offering documents, and approval of a comprehensive business plan [8]

NEW YORK, July 10, 2025 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT).Shareholders who purchased shares of RCKT during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any recovery.CONTACT US HERE:https://securitiesclasslaw.com/securities/rocket-pharmaceuticals-inc-loss-submission-form/?id=156038&from=4CLASS PERIO ...

Core Viewpoint - Oncolytics Biotech Inc. is hosting a KOL webinar on July 22, 2025, to discuss the immunotherapeutic agent pelareorep in the context of metastatic pancreatic ductal adenocarcinoma and other gastrointestinal cancers [1][2]. Company Overview - Oncolytics Biotech Inc. is a clinical-stage biotechnology company focused on developing pelareorep, an intravenously delivered immunotherapeutic agent that has shown promising results in clinical studies for metastatic breast cancer and pancreatic cancer [10][11]. - Pelareorep works by inducing anti-cancer immune responses and transforming "cold" tumors into "hot" tumors, enhancing the effectiveness of various cancer treatments [10]. Webinar Details - The webinar will feature key opinion leaders (KOLs) including Dirk Arnold, Alexander Eggermont, Sanjay Goel, and Devalingam Mahalingam, who will discuss existing clinical data and the potential of pelareorep in treating mPDAC and other gastrointestinal cancers [2][4][5][6][8]. - A live Q&A session will follow the formal presentation and roundtable discussion [3]. KOL Profiles - Dirk Arnold, M.D., Ph.D., is a prominent figure in gastrointestinal cancers and immunotherapy, with extensive experience in clinical trials [4]. - Alexander Eggermont, M.D., Ph.D., has a strong background in clinical and translational immunotherapy, having authored over 900 peer-reviewed papers [5]. - Sanjay Goel, M.D., M.S., FASCO, focuses on drug development and health disparities, with over 150 research publications [6][7]. - Devalingam Mahalingam, M.D., Ph.D., specializes in early-phase clinical studies and has been involved in numerous clinical trials [8][9].