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  • Lilly buys JAK inhibitor company Ajax for $2.3B & Intellia’s in-vivo CRISPR therapy clears Phase 3

Lilly buys JAK inhibitor company Ajax for $2.3B & Intellia’s in-vivo CRISPR therapy clears Phase 3

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Joachim Eeckhout & Eduardo Longoria
April 28, 2026

Good morning. It feels like the legal battle over COVID-19 vaccine royalties will never end. First, Bayer went after essentially all the makers of mRNA-based COVID-19 vaccines. Then BioNTech took aim at its rival Moderna. Now, CureVac, which became part of BioNTech after its acquisition last year, is pursuing Moderna as well. The lawyers must be rubbing their hands.

— Joachim E.

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SNAPSHOT

Lilly purchases Ajax for $2.3B to fix flaws in widely used cancer drugs

Eli Lilly is acquiring biotech startup Ajax Therapeutics for up to $2.3 billion to develop a next-generation drug aimed at improving JAK inhibitors used in blood cancers and immune diseases.

Why it matters: Current JAK inhibitors often lose effectiveness and carry safety risks, leaving patients with limited long-term options. A more durable, safer therapy could significantly improve treatment outcomes for diseases like myelofibrosis.

Backstory: JAK inhibitors are a standard treatment for certain cancers and autoimmune conditions, but they don’t alter disease progression and can stop working over time. Lilly previously attempted to develop its own JAK2 inhibitor without success.

Zoom in: Ajax’s lead drug (AJ1-11095) targets the JAK2 enzyme differently, binding to its inactive form rather than the active state targeted by existing drugs like Jakafi. This could help overcome resistance mechanisms that limit current therapies. The drug is in Phase 1 trials for myelofibrosis patients who have already been treated with other JAK inhibitors, with Lilly planning to advance dosing decisions later this year.

Big picture: The deal reflects Lilly’s aggressive acquisition strategy, having acquired 5 companies this year alone. Large pharma companies are increasingly buying early-stage assets with promising mechanisms before clinical data drives up valuations.

What’s next: Lilly will continue clinical development, aiming to validate whether Ajax’s approach delivers improved efficacy and durability.

SNAP AGAIN

Intellia’s CRISPR therapy clears the bar of Phase 3, edging toward first in-body gene editing approval

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Intellia Therapeutics’ gene editing drug, lonvo-z, cut hereditary angioedema (HAE) attacks by 87% in a Phase 3 trial, positioning it for potential U.S. approval and a 2027 launch.

Why it matters: If approved, lonvo-z could become the first “in vivo” CRISPR therapy, editing genes directly inside the body and potentially offering a one-time, long-lasting alternative to chronic treatments.

Backstory: Intellia has been a pioneer in CRISPR-based medicine but has faced years of setbacks. HAE already has multiple effective treatments, raising the bar for new entrants.

Zoom in: Over ~6 months, lonvo-z reduced monthly attack rates to 0.26 (≈3 per year) versus more than 2 per month on placebo. Additionally, 62% of patients had no attacks or need for other therapies, compared to 11% on placebo. Side effects (mainly infusion reactions, headache, and fatigue) were mild to moderate. Notably, no liver safety issues emerged, easing a key investor concern.

Yes, but: Future data will need to confirm long-term durability and safety to win over doctors and patients. Despite strong efficacy, analysts project modest peak sales (~$500M), reflecting skepticism about gene therapy uptake.

Big picture: The results mark a scientific milestone for gene editing, but also highlight a growing industry tension: breakthrough therapies don’t always translate into blockbuster sales, especially in crowded markets with established options.

SNIPPETS

What’s happening in biotech today?

⚖️ Trial tradeoff: Compass Therapeutics’ stock fell sharply after its bispecific antibody tovecimig failed to improve overall survival (OS) in a phase 2/3 trial for advanced biliary tract cancer. While the drug combined with chemotherapy showed a higher overall response rate (17.1% vs. 5.3%) and significantly improved progression-free survival (4.7 vs. 2.6 months), median OS was slightly worse than chemotherapy alone (8.9 vs. 9.4 months). The company attributed the result to high patient crossover, though its adjusted analysis was deemed unreliable. Despite mixed data, Compass plans to pursue FDA discussions, but analysts warn the OS miss may complicate approval prospects.

🧴 Psoriasis win: Oruka Therapeutics reported strong phase 2a results for its long-acting psoriasis drug ORKA-001, with 63.5% of treated patients achieving complete skin clearance at 16 weeks compared to 1 of 21 on placebo, meeting the primary endpoint and outperforming benchmarks associated with AbbVie’s Skyrizi. Analysts highlighted the drug’s potentially superior efficacy, clean safety profile, and extended half-life, enabling less frequent, possibly annual dosing, calling the data an “outright win” over Skyrizi and projecting blockbuster sales of $5 billion to $10 billion. The results boosted Oruka’s stock by 30%, and the company is now preparing for phase 3 trials while considering whether to include head-to-head comparisons with leading therapies.

✂️ Pipeline prune: Astellas Pharma is streamlining its pipeline by halting development of its gene therapy AT132 for X-linked myotubular myopathy and discontinuing two phase 1 candidates, citing strategic prioritization rather than safety issues for most cuts. AT132, previously linked to patient deaths and regulatory setbacks, will be replaced by a newer gene therapy, ASP2957, which shows improved muscle targeting and reduced liver exposure in preclinical studies and is now in early clinical testing. The shift will cost the company about $103 million.

 🩹 Lesion clear: Aicuris’s antiviral candidate pritelivir demonstrated strong efficacy and safety in a phase 3 trial for immunocompromised patients with refractory herpes simplex virus (HSV) infections, significantly outperforming existing treatments. In the PRIOH-1 study, pritelivir achieved higher rates of complete lesion healing compared to investigator’s choice therapy (62.7% vs. 34.0% at 28 days, rising to 82.4% vs. 42.0% at 48 days) and improved viral clearance, while also showing fewer adverse events and discontinuations. The oral, once-daily drug addresses a major unmet need in patients resistant to standard antivirals and is now under FDA priority review with a decision expected in late 2026, positioning it as a potentially transformative therapy.

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