Lilly to present initial clinical data for first-in-class type II JAK2 inhibitor in patients with previously treated myelofibrosis at the 2026 EHA Annual Meeting
Rhea-AI Summary
Lilly (NYSE: LLY) reported initial Phase 1 AJX-101 data for its first-in-class type II JAK2 inhibitor AJ1-11095 in previously treated myelofibrosis, to be presented at the 2026 EHA Annual Meeting.
Results show high rates of spleen volume reduction, symptom improvement, VAF decreases, and a generally manageable safety profile.
AI-generated analysis. Not financial advice.
Positive
- SVR35 spleen response achieved as best response in 70% of patients
- TSS50 symptom improvement at week 12 seen in 70% of patients
- Driver mutation VAF reductions observed in 21 of 23 patients
- 59% of 17 patients at week 24 had ≥20% VAF reduction; 35% had ≥50%
- No dose-limiting toxicities reported; 78% of patients remain on study
- Asset added to Lilly pipeline following completed acquisition of Ajax Therapeutics
Negative
- Data come from a small, early Phase 1 dose-escalation trial
- Common treatment-emergent adverse events included anemia and decreased platelet count
- Additional adverse events reported: dysgeusia and increased alanine aminotransferase
Key Figures
Market Reality Check
Peers on Argus
LLY is down 2.41% while close peers show mixed, mostly small moves: ABBV +0.01%, JNJ +0.36%, AZN -2.06%, NVS -0.56%, NVO -0.14%. No peers appeared in the momentum scanner, supporting a stock-specific move.
Previous Clinical trial Reports
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| May 21 | Obesity Phase 3 data | Positive | +2.2% | Retatrutide delivered up to 30.3% weight loss in pivotal obesity trial. |
| Apr 16 | CV outcomes trial | Positive | -0.1% | Foundayo met MACE-4 non-inferiority with 57% lower all-cause death. |
| Mar 19 | Diabetes Phase 3 data | Positive | -0.1% | Retatrutide achieved up to 2.0% A1C reduction and 16.8% weight loss. |
| Mar 16 | Dermatology Phase 3 | Positive | -0.2% | EBGLYSS delivered strong pediatric atopic dermatitis responses with favorable safety. |
| Dec 18 | Weight maintenance trial | Positive | +1.4% | Orforglipron helped maintain weight loss after injectable incretin therapy. |
Recent clinical trial announcements have mostly been positive yet produced modest average moves of 0.66%, with more mild negative than positive next-day reactions.
Over the past six months, Lilly’s news flow has been dominated by positive clinical trial readouts across obesity, type 2 diabetes, atopic dermatitis, and weight maintenance. Events like retatrutide’s strong Phase 3 obesity data and Foundayo cardiovascular and glycemic outcomes showed substantial efficacy and generally reassuring safety. Market reactions, however, have often been muted or even slightly negative. Today’s myelofibrosis Phase 1 data continue this pattern of strong scientific progress not always matching short-term share performance.
Historical Comparison
In the last five clinical-trial updates, LLY moved an average of 0.66%. Today’s -2.41% reaction to early myelofibrosis data is weaker than prior typical moves on similar catalysts.
Clinical updates span obesity, type 2 diabetes, atopic dermatitis, and weight maintenance, with today’s myelofibrosis data extending Lilly’s pipeline into hematologic malignancies.
Market Pulse Summary
This announcement highlights promising first-in-class Phase 1 results for AJ1-11095 in previously treated myelofibrosis, with notable spleen, symptom, and mutation burden improvements in a small, heavily pretreated cohort of 23 patients. It extends Lilly’s track record of positive clinical data across multiple disease areas. Investors may monitor durability of responses, expansion cohort outcomes, and how this hematology asset complements Lilly’s existing metabolic and immunology franchises in future updates.
Key Terms
phase 1 medical
type ii jak2 inhibitor medical
myelofibrosis medical
variant allele frequency (vaf) medical
treatment-emergent adverse events medical
dose-limiting toxicities medical
expansion cohort medical
clinicaltrials.gov technical
AI-generated analysis. Not financial advice.
These data will be highlighted in an oral presentation at the 2026 European Hematology Association (EHA) Annual Meeting taking place in
"Patients with myelofibrosis who have been previously treated with an existing type I JAK2 inhibitor face very limited treatment options, highlighting an urgent need for new therapies," said John Mascarenhas, MD, professor of medicine, Icahn School of Medicine at Mount Sinai and principal investigator of the AJX-101 study. "These early clinical findings suggest that selective targeting of the type II conformation of JAK2 may provide a differentiated approach. With an encouraging safety profile, meaningful spleen size reduction, symptom improvement, and decrease in underlying mutant disease burden, these data, while early, point to the potential to meaningfully impact treatment options for people with certain myeloproliferative neoplasms."
AJX-101 is the first clinical trial to evaluate a type II selective JAK2 inhibitor in patients with myelofibrosis. The trial enrolled 23 patients across five dose levels (25, 50, 75, 100, and 125 mg once daily) in its dose escalation phase. Patients had received a median of two prior therapies, and all had previously received a type I JAK2 inhibitor. The trial enrolled patients across all major myelofibrosis subtypes and driver mutations.
AJ1-11095 demonstrated responses across the standard efficacy endpoints of spleen volume reduction and symptom improvement.1 The SVR35 rate, a reduction in spleen volume of at least
The overall safety profile for the medicine was generally manageable. No dose-limiting toxicities were observed, and most patients enrolled in the dose escalation phase remain on study (
"The depth of response seen across spleen, symptoms, and VAF from these early phase results is in excess of what has been seen historically in this disease setting," said Jacob Van Naarden, executive vice president and president of Lilly Oncology. "These data provide clear proof of concept for what this selective type II JAK2 inhibitor could mean for patients with myelofibrosis and shed light on the conviction we brought to the acquisition of Ajax. With this program now officially part of Lilly's pipeline, we are committed to rapidly advancing it through clinical development and further exploring its potential to meaningfully improve outcomes for people with myeloproliferative neoplasms across a range of disease settings."
AJ1-11095 is currently being evaluated in an expansion cohort in second-line myelofibrosis, with plans to investigate in patients with high-risk polycythemia vera and those with myelofibrosis who have not yet received a JAK2 inhibitor. Details on the AJX-101 trial can be found by visiting clinicaltrials.gov.
About AJ1-11095
AJ1-11095 is an investigational, oral, first-in-class type II JAK2 inhibitor. AJ1-11095 is designed to bind JAK2 in its inactive conformation — an approach intended to more completely suppress the aberrant signaling that drives myelofibrosis, in contrast to currently approved JAK2 inhibitors that bind JAK2 in its active state. AJ1-11095 demonstrated superior activity compared to ruxolitinib in preclinical models of myelofibrosis. AJ1-11095 is currently being studied in AJX-101, a global, open-label, multicenter, Phase 1 study in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis who have previously been treated with a type I JAK2 inhibitor, NCT06343805.
About Lilly
Lilly is a medicine company turning science into healing to make life better for people around the world. We've been pioneering life-changing discoveries for 150 years, and today our medicines help tens of millions of people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world's most significant health challenges: redefining diabetes care; treating obesity and curtailing its most devastating long-term effects; advancing the fight against Alzheimer's disease; providing solutions to some of the most debilitating immune system disorders; and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we're motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable. To learn more, visit Lilly.com and Lilly.com/news, or follow us on Facebook, Instagram, and LinkedIn. P-LLY
© Lilly USA, LLC 2026. ALL RIGHTS RESERVED.
Trademarks and Trade Names
All trademarks or trade names referred to in this press release are the property of the company, or, to the extent trademarks or trade names belonging to other companies are referenced in this press release, the property of their respective owners. Solely for convenience, the trademarks and trade names in this press release are referred to without the ® and ™ symbols, but such references should not be construed as any indicator that the company or, to the extent applicable, their respective owners will not assert, to the fullest extent under applicable law, the company's or their rights thereto. We do not intend the use or display of other companies' trademarks and trade names to imply a relationship with, or endorsement or sponsorship of us by, any other companies.
Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about AJ1-11095 as a potential treatment for adults with myelofibrosis and other myeloproliferative neoplasms, and the timeline for future studies, regulatory submissions, presentations, and other milestones relating to AJ1-11095 and the AJX-101 clinical program, and reflects Lilly's current beliefs and expectations. However, as with any pharmaceutical product, there are substantial risks and uncertainties in the process of drug research, development, and commercialization. Among other things, there is no guarantee that planned or ongoing studies will be completed as planned or that future study results will be consistent with study results to date. For further discussion of these and other risks and uncertainties that could cause actual results to differ from Lilly's expectations, see Lilly's Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission. Except as required by law, Lilly undertakes no duty to update forward-looking statements to reflect events after the date of this release.
Refer to: | Megan Talon; megan.talon@lilly.com (Media) |
Michael Czapar; czapar_michael_c@lilly.com (Investors) |
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SOURCE Eli Lilly and Company