ProQR Reports Initial Safety Data for AX-0810, Updates 2026 Milestones
ProQR Therapeutics (PRQR) announced encouraging initial safety and PK data from the first cohort of healthy volunteers in its ongoing Phase 1 trial of AX-0810 and provided an update on broader pipeline progress and anticipated 2026 milestones. "The initial human data from AX-0810 mark an important early milestone for ProQR, providing safety and pharmacokinetic observations in healthy volunteers," said Cristina Lopez Lopez, MD, PhD, Chief Medical Officer of ProQR. "These data support continued dosing and position us well for the upcoming target engagement readout in the first half of 2026." Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; Phase 1 enrollment and dosing in healthy volunteers ongoing with target engagement data expected in H1 2026, followed by inclusion of a patient cohort. Development Candidates selected for pipeline programs AX-2402 for Rett syndrome and AX-2911 for MASH. Strategic collaboration with Eli Lilly (LLY) achieved $4.5 million in milestones in 2025, contributing to strong financial position with runway into mid-2027
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- Significant Sales Growth: Eli Lilly's GLP-1 weight-loss drugs generated $7.4 billion in Q4 sales, a 110% increase, while Zepbound's revenue surged from $1.9 billion to $4.3 billion, indicating strong market demand and product acceptance.
- Overall Performance Exceeds Expectations: The company reported a 43% increase in total revenue to $19.29 billion for Q4, with adjusted earnings per share (EPS) jumping 42% to $7.54, significantly surpassing analyst expectations of $6.67, reflecting the company's competitive edge and profitability.
- Optimistic Future Outlook: Eli Lilly projects 2026 revenue between $80 billion and $83 billion, representing a 25% growth at the midpoint, and forecasts adjusted EPS ranging from $33.50 to $35, demonstrating confidence in future market demand.
- Huge Potential for New Drug: The upcoming oral GLP-1 drug orforglipron is expected to gain approval in Q2, and its convenient administration method positions Eli Lilly to further expand its market share in the GLP-1 drug sector, reinforcing the company's leadership in weight-loss medications.
- Acquisition Deal Size: Eli Lilly has agreed to acquire biotechnology firm Orna Therapeutics for up to $2.4 billion, including upfront payments and subsequent milestone-based payments, demonstrating its commitment to long-term innovation in genetic medicine.
- Platform Technology Advantage: Orna is developing a novel therapy using engineered circular RNA and lipid nanoparticles, enabling patients' bodies to produce cell-based treatments for underlying diseases, which could transform existing treatment paradigms.
- Clinical Trial Readiness: Orna's lead program, ORN-252, is a clinical-trial-ready in vivo CAR-T therapy targeting CD19, aimed at treating B-cell-driven autoimmune diseases, with early studies suggesting its therapy may offer longer-lasting effects than current RNA or cell therapy platforms.
- Positive Market Reaction: Following the acquisition announcement, Eli Lilly's shares rose 1.7% in premarket trading to $1,075, reflecting market optimism regarding the deal and confidence in the company's future growth potential.
- Acquisition Agreement: Eli Lilly announced its acquisition of biotechnology firm Orna Therapeutics for up to $2.4 billion, including upfront and milestone payments, which is expected to significantly enhance Lilly's therapeutic pipeline, particularly in cell therapies.
- Positive Stock Reaction: Following the acquisition announcement, Lilly's stock rose 1.6% in pre-market trading, reflecting market optimism about the deal, which could enhance the company's future profitability and competitive position.
- Innovative Technology Potential: This acquisition will provide Lilly with access to a new class of therapies based on engineered circular RNA and proprietary lipid nanoparticles, enabling the body to generate its own disease-fighting cell therapies, potentially offering new hope for patients with limited treatment options.
- Strategic Development Focus: Lilly executives noted that while cell therapies show promise for patients with autoimmune diseases, existing complexities and costs limit their widespread application; this acquisition will help develop entirely new genetic medicines and cell therapies, driving long-term growth in the biopharmaceutical sector.
- Escalating Legal Action: Novo Nordisk has filed a lawsuit seeking a permanent ban on Hims from selling compounded drugs that infringe its patents while also pursuing damages, demonstrating its commitment to defending its interests in the rapidly growing obesity drug market.
- Intensified Market Competition: Hims planned to offer its newly launched obesity pill at $49 per month, significantly lower than Novo's Wegovy, but has decided to halt sales after facing scrutiny from regulators and legal threats from the Danish drugmaker.
- Patient Safety Concerns: Novo accused Hims of deceiving patients by marketing unapproved drugs that lack FDA verification, highlighting the critical importance of regulatory standards to ensure drug safety and efficacy.
- FDA Investigation: The FDA has announced plans to take legal action against Hims, including restricting access to drug ingredients and referring the company to the Department of Justice for potential violations, further complicating Hims' legal challenges.
- Eli Lilly's Acquisition: Eli Lilly announced its agreement to acquire Orna Therapeutics for $2.4 billion in cash.
- Market Reaction: Following the announcement, Eli Lilly's shares experienced an increase.
- Tech Stock Decline: Concerns over AI have led to a decline in tech stocks over the past week, putting pressure on the market as investors await crucial economic data and hints from the Federal Reserve regarding potential rate cuts.
- Dow Jones Performance: The Dow Jones closed above 50,000 points for the first time on Friday, posting significant weekly gains, indicating a rotation of funds into other sectors and boosting investor confidence.
- Economic Data Focus: Key economic releases, including the nonfarm payrolls report and consumer price index, are set to be released this week, which could influence the Fed's rate decisions, with markets pricing in the first rate cut potentially in June.
- Stock Movements: Eli Lilly shares rose 2.1% in premarket trading, while Hims & Hers dropped 14% after canceling a weight-loss pill launch due to FDA legal threats, highlighting market sensitivity to regulatory risks.











