Core Insights - Esperion Therapeutics (ESPR) has nominated ESP-2001, a specific allosteric ATP citrate lyase (ACLY) inhibitor, as a new preclinical development candidate for treating primary sclerosing cholangitis (PSC), a rare autoimmune liver disease with no approved treatments [1][7] - The company plans to initiate IND-enabling studies for ESP-2001 and submit an IND application to the FDA, aiming to start clinical studies in 2026 [2] - ESP-2001 has shown potential in reducing liver and bile duct injury, inflammation, and fibrosis in preclinical studies, indicating its ability to impact PSC progression [3] Company Developments - Esperion's stock rose by 5.2% following the announcement of ESP-2001 [2] - The collaboration with Evotec (EVO) was crucial in discovering ESP-2001, combining Esperion's expertise in ACLY therapy with Evotec's drug discovery platform [2][3] - The nomination of ESP-2001 triggered an undisclosed payment to Evotec [3] Financial Performance - Esperion's net product sales of Nexletol and Nexlizet in the U.S. grew by 42% in the first half of 2025, reaching $75.2 million, driven by increased prescription volumes [10] - Esperion aims to diversify its portfolio beyond cardiovascular diseases into the liver disorder market, targeting a potential market opportunity exceeding $1 billion annually with ESP-2001 [10] Market Position - Esperion currently holds a Zacks Rank of 3 (Hold) [11] - The company has two FDA-approved drugs, Nexletol and Nexlizet, which are marketed under different names in ex-U.S. markets [8][9]

Core Viewpoint - Cellectar Biosciences is preparing to submit a Conditional Marketing Authorization (CMA) application for iopofosine I 131 as a treatment for refractory Waldenstrom macroglobulinemia (WM), with potential European approval and commercial launch expected in 2027 [1][4]. Company Overview - Cellectar Biosciences is a late-stage clinical biopharmaceutical company focused on developing drugs for cancer treatment, utilizing its proprietary Phospholipid Drug Conjugate™ (PDC) delivery platform [14][15]. Product Details - Iopofosine I 131 is a novel cancer-targeting agent designed for post-BTKi refractory WM patients, having received PRIME designation from the EMA for patients who have undergone at least two prior lines of therapy [2][11]. - The drug has demonstrated an overall response rate (ORR) of 83.6% and a major response rate (MRR) of 58.2% in the CLOVER WaM Phase 2 study [4][6]. Regulatory Pathway - The decision to file for CMA follows advice from the Scientific Advice Working Party (SAWP) of the EMA, which indicated that the data supports the eligibility for CMA submission for the specified patient population [3][8]. - CMA allows for earlier access to medicines addressing unmet medical needs, permitting approval based on less comprehensive clinical data [8]. Market Opportunity - There is a significant unmet medical need for WM treatment in Europe, affecting an estimated 35,000 to 45,000 patients [3][12]. - The company believes that iopofosine I 131 presents a substantial market opportunity due to its compelling patient outcomes and regulatory designations [5][12]. Future Plans - The company plans to pursue worldwide approval, including a New Drug Application (NDA) with the U.S. FDA under an accelerated approval pathway, contingent upon obtaining additional funding for a confirmatory study [4][6]. - The U.S. NDA will be supported by data from the Phase 2b CLOVER WaM clinical trial, which includes FDA-requested follow-up results [6][7].