Core Insights - BOOST Pharma has presented new long-term data from the BOOSTB4 phase 1/2 trial for its cell therapy BT-101, targeting Osteogenesis imperfecta, at the 15th International Conference on Osteogenesis imperfecta in Hong Kong [1][2] Company Overview - BOOST Pharma is a clinical-stage biopharmaceutical company focused on developing BT-101 as a stem cell therapy for infants with Osteogenesis imperfecta, a condition leading to fragile bones and frequent fractures [2] - Karolinska Development AB holds a 14% ownership stake in BOOST Pharma, indicating its investment in innovative medical solutions [4] Clinical Trial Results - Long-term results from the BOOSTB4 trial show that over 50% of treated patients did not experience any bone fractures in the second year after the last dose [3] - The previously reported 70% reduction in fractures during the first year was maintained and improved, leading to an overall reduction of nearly 78% compared to the pre-treatment period [3] Therapeutic Potential - The results from BOOST Pharma suggest that BT-101 has a robust therapeutic effect and potential as a disease-modifying treatment in a field with significant medical need and no adequate existing treatments [4]

Core Viewpoint - Iovance Biotherapeutics' stock has seen a significant decline, down approximately 75% from its peak in December, leading to mixed opinions among analysts regarding its valuation and future potential [1][2][3]. Group 1: Analyst Ratings and Price Targets - Chardan Capital maintains a buy rating with a price target of $25, suggesting a potential gain of about 720% from the current price of $3.05 [2]. - The consensus price target for Iovance is $10 per share, indicating a potential average gain of 228% [3]. Group 2: Product Approval and Market Potential - The FDA approved Iovance's first product, Amtagvi, in February 2024, which is a cell-based treatment for advanced melanoma [5]. - In clinical trials, Amtagvi demonstrated a tumor shrinkage response in 31.5% of patients who had previously failed PD-1 blocking therapies [6]. - Among 41 patients evaluated post-approval, 20 showed smaller tumors or no tumors, with a response rate of 60.9% in those with limited prior treatments [7]. Group 3: Challenges Facing Iovance - Goldman Sachs downgraded Iovance to sell, citing concerns over a slower-than-expected launch of Amtagvi [8]. - The launch faces challenges including the FDA's approval based on tumor shrinkage rather than overall survival benefits, a small sales team, and the complex administration of the treatment [9][10]. - Sales for Amtagvi reached $43.6 million in Q1 2025, which is below expectations for a blockbuster product [11]. Group 4: Financial Outlook and Market Valuation - Iovance's market cap is approximately $1.05 billion, which is considered low for a biotech company with a new product [12]. - Management projects sales between $250 million and $300 million for the year, with biotech stocks typically trading at mid-to-high single-digit multiples of trailing sales [12]. - The resignation of the CFO raises concerns about future sales guidance, as such changes are often associated with disappointing forecasts [14].