On October 2, 2026 Eli Lilly and Company (NYSE: LLY) reported that the U.S. Food and Drug Administration (FDA) has approved an additional indication for Jaypirca (pirtobrutinib, 100 mg & 50 mg tablets), a non-covalent Bruton tyrosine kinase (BTK) inhibitor, for the treatment of adult patients with previously untreated chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) with no known 17p deletion. This approval allows for the use of Jaypirca as a first-line treatment for appropriate patients.

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"This approval is grounded in data from BRUIN CLL-313, which showed a significant delay in disease progression for pirtobrutinib compared to chemoimmunotherapy, along with safety and tolerability consistent with its established profile," said Jennifer A. Woyach, M.D., professor, hematologist-oncologist, and Director of the Division of Hematology at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute. "Doctors can now consider pirtobrutinib for appropriate patients when initial therapy is needed, not just later in a patient’s treatment journey. Given the efficacy and tolerability of modern targeted therapies – coupled with factors like age or comorbidity – many people diagnosed with CLL or SLL today may only receive one or two lines of therapy, making initial treatment choices critically important."

The labeling for Jaypirca contains warnings and precautions for infections, hemorrhage, cytopenias, cardiac arrhythmias, second primary malignancies, hepatotoxicity including drug-induced liver injury and embryo-fetal toxicity. See Important Safety Information below and full Prescribing Information for additional information, including dosing modifications.

Jaypirca, the first-and-only FDA-approved non-covalent BTK inhibitor, is a highly selective kinase inhibitor that utilizes a novel non-covalent binding mechanism to target the BTK pathway in patients with CLL/SLL.1,2

The FDA approval is based on results from the primary analysis of the BRUIN CLL-313 clinical trial, which were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2025 and published in The Journal of Clinical Oncology.3 BRUIN CLL-313 is the first prospective, randomized Phase 3 study to examine the efficacy and safety of a non-covalent BTK inhibitor in patients with previously untreated CLL/SLL without 17p deletion.

In BRUIN CLL-313, at a median follow-up of 28 months, the primary endpoint of Independent Review Committee (IRC)-assessed progression-free survival (PFS) was significantly improved with pirtobrutinib (n=141) compared to bendamustine plus rituximab (BR) (n=141) (HR=0.20 [95% CI, 0.11–0.37]; p<0.0001), and the median PFS was not yet reached for pirtobrutinib compared to 33.5 months for BR.4 IRC-assessed overall response rate (ORR) was 94% (95% CI, 89–98) in the pirtobrutinib arm (complete response [CR]=13%; partial response [PR]=81%) and 81% (95% CI, 73–87) in the BR arm (CR=21%; PR=60%). In the BRUIN CLL-313 trial, adverse reactions (ARs) led to dose reductions in 3.6% and permanent discontinuation of Jaypirca in 4.3% of patients. Serious ARs occurred in 28% of patients who received Jaypirca. Serious ARs occurring in ≥3% of patients included pneumonia (5%). The overall safety profile, including rates of atrial fibrillation or flutter, for patients treated with pirtobrutinib in the BRUIN CLL-313 trial was consistent with previously reported trials across treatment settings.

"This additional approval for Jaypirca, based on BRUIN CLL-313, marks a significant step forward, expanding its potential to reach more patients who may benefit – this time as an initial treatment for certain previously untreated patients with CLL or SLL," said Jacob Van Naarden, executive vice president and president of Lilly Oncology. "This milestone underscores Jaypirca’s versatility in the CLL continuum of care, from the first-line setting for appropriate patients to its valuable role in the relapsed or refractory post-covalent BTK inhibitor setting, reinforcing Jaypirca’s broad applicability as a meaningful treatment option for people with CLL or SLL."

Jaypirca is the first-and-only non-covalent BTK inhibitor recommended by the National Comprehensive Cancer Network (NCCN).1,5,6,7 Jaypirca is Category 2A recommended for treatment-naïve adult patients with CLL/SLL without del(17p), recommended for older patients with cardiac comorbidities who may only need one lifetime treatment for CLL/SLL.8 Jaypirca is a Category 1 preferred option for adult patients with relapsed or refractory CLL/SLL who have previously been treated with a covalent BTK inhibitor. Please see full NCCN guidelines for more information.

Lilly is studying Jaypirca in CLL/SLL in multiple Phase 3 studies. Details on the trials can be found by visiting clinicaltrials.gov.

See Important Safety Information below and full Prescribing Information for additional information.

Click here to view the CLL infographic.

About the BRUIN Clinical Development Program
The BRUIN clinical development program comprises four positive Phase 3 studies evaluating pirtobrutinib across multiple lines of CLL/SLL:

BRUIN CLL-313 is the first prospective, randomized Phase 3 study to examine the efficacy and safety of a non-covalent BTK inhibitor in patients with previously untreated CLL without 17p deletion. The BRUIN CLL-313 study is evaluating pirtobrutinib versus chemoimmunotherapy (BR) in patients with CLL/SLL without 17p deletion who have not been previously treated.
BRUIN CLL-314 is the first head-to-head Phase 3 CLL trial to compare covalent and non-covalent BTK inhibitors in a BTKi-naïve population, which included relapsed and refractory and treatment-naïve patients. The BRUIN CLL-314 study is evaluating pirtobrutinib versus ibrutinib in patients with CLL/SLL who were either treatment-naïve, or who were previously treated and were BTK inhibitor-naïve.
BRUIN CLL-321 is the first randomized Phase 3 study in CLL in which all patients were previously treated with a covalent BTK inhibitor. The BRUIN CLL-321 study is evaluating pirtobrutinib versus investigator’s choice of idelalisib plus rituximab (IdelaR) or BR in covalent BTK inhibitor pre-treated patients with relapsed and refractory CLL/SLL.
BRUIN CLL-322 is the first Phase 3 readout in CLL to utilize a venetoclax-containing control arm. The BRUIN CLL-322 study is evaluating time-limited pirtobrutinib plus venetoclax and rituximab versus venetoclax and rituximab in previously treated CLL/SLL patients.
About BRUIN CLL-313
BRUIN CLL-313 (NCT05023980) is a Phase 3, global, randomized, open-label study of Jaypirca (pirtobrutinib) versus chemoimmunotherapy (BR) in people with CLL/SLL without 17p deletion who have not been previously treated. The trial enrolled 282 patients who were randomized 1:1 to receive pirtobrutinib (200 mg orally, once daily until disease progression or unacceptable toxicity) or BR per labeled doses. BR is a chemoimmunotherapy regimen used in the treatment of CLL. The primary endpoint is PFS as assessed by blinded IRC. Secondary endpoints include investigator and IRC-assessed ORR and duration of response (DoR), investigator-assessed PFS, overall survival (OS), time to next treatment (TTNT), safety and tolerability and patient-reported outcomes (PRO).

About Jaypirca (pirtobrutinib)
Jaypirca (pirtobrutinib, formerly known as LOXO-305) (pronounced jay-pihr-kaa) is a highly selective (300 times more selective for BTK versus 98% of other kinases tested in preclinical studies), non-covalent inhibitor of the enzyme BTK, and is the first-and-only approved non-covalent BTK inhibitor binding to both wild-type and C481 mutated BTK.2 BTK is a validated molecular target found across numerous B-cell leukemias and lymphomas including mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL).9,10 Jaypirca is a U.S. FDA-approved oral prescription medicine, 100 mg or 50 mg tablets taken as a once-daily 200 mg dose with or without food until disease progression or unacceptable toxicity.

About Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
CLL and SLL are forms of slow-growing non-Hodgkin lymphoma that develop from white blood cells known as lymphocytes.11 CLL is one of the most common types of leukemia in adults.11 In the U.S., CLL accounts for about one-quarter of the new cases of leukemia and there will be approximately 22,760 new cases of CLL diagnosed this year.11,12 SLL is identical to CLL from a pathologic and immunophenotypic standpoint, with the main difference between them being the location of the cancer cells.11 In CLL, the cancer cells are present in the blood, and in SLL, the cancer cells are found in the lymph nodes.11

INDICATIONS FOR JAYPIRCA (pirtobrutinib)
Jaypirca is indicated for the treatment of:

Adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have previously been treated with a covalent BTK inhibitor.
Adult patients with previously untreated chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) with no known 17p deletion.
Adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a BTK inhibitor. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
IMPORTANT SAFETY INFORMATION FOR JAYPIRCA (pirtobrutinib)

Infections: Fatal and serious infections (including bacterial, viral, fungal) and opportunistic infections occurred in Jaypirca-treated patients. Across all clinical trials, Grade ≥3 infections occurred (23%), most commonly pneumonia (13%); fatal infections (4.3%), sepsis (4.8%), and febrile neutropenia (3.3%) occurred. In patients with CLL/SLL, Grade ≥3 infections occurred (24%), with fatal infections occurring in 5%. Opportunistic infections included Pneumocystis jirovecii pneumonia and fungal infection. Consider prophylaxis, including vaccinations and antimicrobial prophylaxis, in patients at increased risk for infection, including opportunistic infections. Monitor for signs and symptoms, evaluate, and treat. Based on severity, reduce dose, temporarily withhold, or permanently discontinue Jaypirca.

Hemorrhage: Fatal and serious hemorrhage has occurred with Jaypirca. Across all clinical trials, major hemorrhage (Grade ≥3 bleeding or any central nervous system bleeding) occurred (3.1%), including gastrointestinal hemorrhage; fatal hemorrhage occurred (0.3%). Bleeding of any grade, excluding bruising and petechiae, occurred (18%). Major hemorrhage occurred when taking Jaypirca with (1.3%) and without (1.8%) antithrombotic agents. Consider risks/benefits of coadministering antithrombotic agents with Jaypirca. Monitor for signs of bleeding. Based on severity, reduce dose, temporarily withhold, or permanently discontinue Jaypirca. Consider withholding Jaypirca 3-7 days pre- and post-surgery based on surgery type and bleeding risk.

Cytopenias: Jaypirca can cause cytopenias, including neutropenia, thrombocytopenia, and anemia. Across all clinical trials, Grade 3 or 4 cytopenias, including decreased neutrophils (26%), decreased platelets (9%), and decreased hemoglobin (9%), developed. Grade 4 decreased neutrophils (14%) and Grade 4 decreased platelets (4.3%) developed. Monitor complete blood counts regularly. Based on severity, reduce dose, temporarily withhold, or permanently discontinue Jaypirca.

Cardiac Arrhythmias: Cardiac arrhythmias occurred in patients taking Jaypirca. Across all clinical trials, atrial fibrillation or flutter were reported in 3.1% of Jaypirca-treated patients, with Grade 3 or 4 atrial fibrillation or flutter in 1.6%. Other serious cardiac arrhythmias such as supraventricular tachycardia and cardiac arrest occurred (0.3%). Cardiac risk factors such as hypertension or previous arrhythmias may increase risk. Monitor and manage signs and symptoms of arrhythmias (e.g., palpitations, dizziness, syncope, dyspnea). Based on severity, reduce dose, temporarily withhold, or permanently discontinue Jaypirca.

Second Primary Malignancies: Across all clinical trials, second primary malignancies, including non-skin carcinomas, developed in 10% of Jaypirca-treated patients, most frequently non-melanoma skin cancer (4.9%). Other second primary malignancies included solid tumors (including genitourinary and breast cancers) and melanoma. Advise patients to use sun protection and monitor for development of second primary malignancies.

Hepatotoxicity, Including Drug-Induced Liver Injury (DILI): Hepatotoxicity, including severe, life-threatening, and potentially fatal cases of DILI, has occurred in patients treated with BTK inhibitors, including Jaypirca. Evaluate bilirubin and transaminases at baseline and throughout Jaypirca treatment. For patients who develop abnormal liver tests after Jaypirca, monitor more frequently for liver test abnormalities and clinical signs and symptoms of hepatic toxicity. If DILI is suspected, withhold Jaypirca. If DILI is confirmed, discontinue Jaypirca.

Embryo-Fetal Toxicity: Jaypirca can cause fetal harm. Administration of pirtobrutinib to pregnant rats caused embryo-fetal toxicity, including embryo-fetal mortality and malformations at maternal exposures (AUC) approximately 3-times the recommended 200 mg once daily dose. Advise pregnant women of fetal risk and females of reproductive potential to use effective contraception during treatment and for one week after last dose.

Adverse Reactions (ARs) in Patients Who Received Jaypirca

The most common (≥30%) ARs in the pooled safety population of patients with hematologic malignancies (n=1153) were decreased neutrophil count (56%) and decreased hemoglobin (38%).

Mantle Cell Lymphoma

Serious ARs occurred in 38% of patients, with pneumonia (14%), COVID-19 (4.7%), musculoskeletal pain (3.9%), hemorrhage (2.3%), pleural effusion (2.3%), and sepsis (2.3%) occurring in ≥2% of patients. Fatal ARs within 28 days of last dose occurred in 7% of patients, most commonly due to infections (4.7%), including COVID-19 (3.1% of all patients).

Dose Modifications and Discontinuations Due to ARs: Dose reductions in 4.7%, treatment interruption in 32%, and permanent discontinuation of Jaypirca in 9% of patients. Permanent discontinuation in >1% of patients included pneumonia.

Most common ARs (≥15%) and Select Laboratory Abnormalities (≥10%) (all Grades %; Grade 3 or 4%): hemoglobin decreased (42; 9), platelet count decreased (39; 14), neutrophil count decreased (36; 16), lymphocyte count decreased (32; 15), creatinine increased (30; 1.6), fatigue (29; 1.6), musculoskeletal pain (27; 3.9), calcium decreased (19; 1.6), diarrhea (19; -), edema (18; 0.8), dyspnea (17; 2.3), AST increased (17; 1.6), pneumonia (16; 14), bruising (16; -), potassium decreased (13; 1.6), sodium decreased (13; -), lipase increased (12; 4.4), ALT increased (11; 1.6), potassium increased (11; 0.8), alkaline phosphatase increased (11; -). Grade 4 laboratory abnormalities in >5% of patients included neutrophils decreased (10), platelets decreased (7), lymphocytes decreased (6).

Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma from Single-Arm and Randomized Controlled Clinical Trials

Serious ARs occurred in 28-56% of patients across clinical trials. Serious ARs in ≥5% of patients in the single-arm trial were pneumonia (18%), COVID-19 (9%), sepsis (7%), febrile neutropenia (7%). Serious ARs in ≥3% of patients in the randomized controlled trials were pneumonia (5-21%), COVID-19 (5%), sepsis (3.4%). Across clinical trials, fatal ARs within 28-30 days of last Jaypirca dose occurred in 0.7-11% of patients, most commonly due to infections (7-10%), including sepsis (5%), COVID-19 (2.7-5%), pneumonia (3.4%) and septic shock (0.7%).

Dose Modifications and Discontinuations Due to ARs: Dose reductions in 3.6-10%, treatment interruption in 35-51%, and permanent discontinuation of Jaypirca in 4.3-17% of patients. Permanent discontinuation in >1% of patients included second primary malignancy, pneumonia, COVID-19, neutropenia, sepsis, anemia, and cardiac arrhythmias.

Most common ARs and Select Laboratory Abnormalities (≥20%) (all Grades %, Grade 3 or 4%)—across randomized controlled trials: neutrophil count decreased (48-54; 14-26), hemoglobin decreased (24-45; 6-10), platelet count decreased (37; 17), upper respiratory tract infections (21-27; 0.9-2.1), bilirubin increased (21-30; 0.9-5), pneumonia (28; 16), ALT increased (25-27; 1.8-2.9), creatinine increased (25; -), calcium decreased (23; 0.9), sodium decreased (22; 0.9), rash (22; 0.7), COVID-19 (21; 0.7), sodium increased (20; -); in a single-arm trial: neutrophil count decreased (63; 45), hemoglobin decreased (48; 19), calcium decreased (40; 2.8), fatigue (36; 2.7), bruising (36; -), cough (33; -), musculoskeletal pain (32; 0.9), platelet count decreased (30; 15), sodium decreased (30; -), COVID-19 (28; 7), pneumonia (27; 16), diarrhea (26; -), abdominal pain (25; 2.7), lymphocyte count decreased (23; 8), ALT increased (23; 2.8), AST increased (23; 1.9), creatinine increased (23; -), dyspnea (22; 2.7), hemorrhage (22; 2.7), lipase increased (21; 7), alkaline phosphatase increased (21; -), edema (21; -), nausea (21; -), pyrexia (20; 2.7), headache (20; 0.9). Grade 4 laboratory abnormalities in >5% of patients included neutrophils decreased (23).

Drug Interactions

Strong CYP3A Inhibitors: Concomitant use increased pirtobrutinib systemic exposure, which may increase risk of Jaypirca ARs. Avoid using strong CYP3A inhibitors with Jaypirca. If concomitant use is unavoidable, reduce Jaypirca dose according to approved labeling.

Strong or Moderate CYP3A Inducers: Concomitant use decreased pirtobrutinib systemic exposure, which may reduce Jaypirca efficacy. Avoid using Jaypirca with strong or moderate CYP3A inducers. If concomitant use with moderate CYP3A inducers is unavoidable, increase Jaypirca dose according to approved labeling.

Sensitive CYP2C8, CYP2C19, CYP3A, P-gp, or BCRP Substrates: Use with Jaypirca increased their plasma concentrations, which may increase risk of ARs related to these substrates for drugs sensitive to minimal concentration changes. Follow recommendations for these sensitive substrates in their approved labeling.

Use in Specific Populations

Pregnancy and Lactation: Due to potential for Jaypirca to cause fetal harm, verify pregnancy status in females of reproductive potential prior to starting Jaypirca. Presence of pirtobrutinib in human milk is unknown. Advise women to use effective contraception and to not breastfeed while taking Jaypirca and for one week after last dose.

Geriatric Use: In the pooled safety population of patients with hematologic malignancies, patients aged ≥65 years experienced higher rates of Grade ≥3 ARs and serious ARs compared to patients <65 years of age.

Renal Impairment: Because severe renal impairment increases pirtobrutinib exposure, reduce Jaypirca dose in these patients according to approved labeling.

PT HCP ISI MCL_CLL OCT2026

Please see Prescribing Information and Patient Information for Jaypirca.

Frequently Asked Questions

1. What is Jaypirca (pirtobrutinib)?

Jaypirca (pirtobrutinib) is a highly selective kinase inhibitor that is 300 times more selective for Bruton tyrosine kinase (BTK) versus 98% of other kinases tested in preclinical studies.2 Jaypirca is the first-and-only approved non-covalent BTK inhibitor binding to both wild-type and C481 mutated BTK.2 BTK is a validated molecular target found across numerous B-cell leukemias and lymphomas including mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL).9,10 The FDA approved Jaypirca as a monotherapy for the treatment of adult patients with previously untreated CLL or small lymphocytic lymphoma (SLL) with no known 17p deletion in October 2026.

2. What is chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL)?

CLL and SLL are forms of slow-growing non-Hodgkin lymphoma that develop from white blood cells called lymphocytes.11 CLL and SLL are considered different forms of the same disease and share the same pathologic and immunophenotypic characteristics.11 The primary difference between CLL and SLL is where the cancer cells are located.11 In CLL, the cancer cells are found primarily in the blood, while in SLL, the cancer cells are found primarily in the lymph nodes.11 CLL is one of the most common forms of leukemia in adults and accounts for approximately one-quarter of new leukemia diagnoses in the United States.11 An estimated 22,760 people in the U.S. will be diagnosed with CLL this year.12

3. What is chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) with no known 17p deletion?

For people with CLL or SLL, certain cytogenic factors may be associated with a higher risk of disease progression.11 One of these factors is a deletion in chromosome 17 [del(17p)] that is found in about 5% to 8% of patients with CLL/SLL when they are first diagnosed.11,13 Del(17p) typically affects the tumor protein p53 (TP53) tumor suppressor gene.11

4. As the basis of this approval, what are the clinical benefits of Jaypirca (pirtobrutinib) for adults with previously untreated chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) with no known 17p deletion?

In the Phase 3 BRUIN CLL-313 study, pirtobrutinib significantly improved Independent Review Committee (IRC)-assessed progression-free survival (PFS) compared to bendamustine plus rituximab (BR) (HR=0.20 [95% CI, 0.11–0.37]; p<0.0001) in patients with previously untreated CLL/SLL without 17p deletion, and the median PFS was not yet reached for pirtobrutinib compared to 33.5 months for BR.4 IRC-assessed overall response rate (ORR) was 94% (95% CI, 89–98) in the pirtobrutinib arm (complete response [CR]=13%; partial response [PR]=81%) and 81% (95% CI, 73–87) in the BR arm (CR=21%; PR=60%).

5. What are the safety results for Jaypirca (pirtobrutinib) from the BRUIN CLL-313 trial?

The overall safety profile, including rates of atrial fibrillation or flutter, for patients treated with pirtobrutinib in the BRUIN CLL-313 trial was consistent with previously reported trials across treatment settings. In the BRUIN CLL-313 trial, in patients with previously untreated CLL/SLL without 17p deletion, a low rate of all-grades atrial fibrillation or flutter was observed, occurring in 1.4% of patients taking pirtobrutinib. Median duration of treatment with pirtobrutinib was 32 months with 92% on treatment for greater than 24 months. Clinically relevant adverse reactions in <10% of patients who received pirtobrutinib include pneumonia and headache. Patients with significant cardiovascular disease (including uncontrolled or symptomatic arrhythmias) were excluded from the CLL-313 study, and the Prescribing Information for Jaypirca includes a Cardiac Arrhythmias warning/precaution. The all-grades adverse reactions ≥20% reported for patients treated with Jaypirca were upper respiratory tract infection (27%), rash (22%), COVID-19 including COVID-19 pneumonia (21%). Select all-grades laboratory abnormalities (≥20%) that worsened from baseline were neutrophil count decreased (48%), bilirubin increased (30%), ALT increased (27%), hemoglobin decreased (24%), and sodium increased (20%).

6. How does this indication for Jaypirca (pirtobrutinib) differ from the previously approved indication for patients with CLL in the U.S.?

In December 2025, the U.S. Food and Drug Administration (FDA) granted approval to Jaypirca (pirtobrutinib) for the treatment of adults with relapsed or refractory CLL or SLL who have previously been treated with a covalent BTK inhibitor. This prior FDA action expanded the Jaypirca label to include patients earlier in their treatment course and converted the December 2023 accelerated approval for later-line CLL/SLL to a traditional approval. This new approval in the U.S. authorizes Jaypirca for adult patients with previously untreated CLL/SLL with no known 17p deletion. This expanded indication brings Jaypirca to the first-line CLL/SLL setting for certain previously untreated patients.

7. Is Jaypirca (pirtobrutinib) approved for 1L CLL treatment?

Yes, Jaypirca (pirtobrutinib) is approved for adult patients with previously untreated CLL/SLL patients with no known 17p deletion. For appropriate patients starting treatment, physicians may consider Jaypirca in 1L CLL.

8. How is Jaypirca (pirtobrutinib) different than ibrutinib, acalabrutinib and zanubrutinib?

Jaypirca (pirtobrutinib) is the first-and-only approved non-covalent BTK inhibitor, representing a newer class of BTK inhibitor compared to ibrutinib, acalabrutinib, and zanubrutinib which are all covalent BTK inhibitors. Based on preclinical studies, pirtobrutinib is different in where and how it binds to the BTK protein in the adenosine triphosphate (ATP) pocket. There are no data from trials between Jaypirca and covalent BTK inhibitors comparing the clinical significance of their different binding mechanisms.

9. Is Jaypirca (pirtobrutinib) recommended in the NCCN Guidelines?

Jaypirca (pirtobrutinib) is the first-and-only non-covalent BTK inhibitor recommended by the National Comprehensive Cancer Network (NCCN).1,5,6,7 Jaypirca is Category 2A recommended for treatment-naïve adult patients with CLL/SLL without del(17p), recommended for older patients with cardiac comorbidities who may only need one lifetime treatment for CLL/SLL.8 Jaypirca is a Category 1 preferred option for adult patients with R/R CLL/SLL who have previously been treated with a covalent BTK inhibitor. Please see full NCCN guidelines for more information.

10. In which Phase 3 clinical trials is Jaypirca (pirtobrutinib) being studied in CLL and SLL?

Jaypirca (pirtobrutinib) is currently being studied in four Phase 3 clinical trials in CLL and SLL:

BRUIN CLL-321 (NCT04666038) is a Phase 3 open-label, randomized study of pirtobrutinib (LOXO-305) versus investigator’s choice of idelalisib plus rituximab or bendamustine plus rituximab in BTK inhibitor pretreated CLL/SLL. More information on the BRUIN CLL-321 study can be found on clinicaltrials.gov.
BRUIN CLL-322 (NCT04965493) is a Phase 3 open-label, randomized study of fixed duration pirtobrutinib plus venetoclax and rituximab versus venetoclax and rituximab in previously treated CLL/SLL. More information on the BRUIN CLL-322 study can be found on clinicaltrials.gov.
BRUIN CLL-313 (NCT05023980) is a Phase 3 open-label, randomized study of pirtobrutinib versus bendamustine plus rituximab in untreated patients with CLL. More information on the BRUIN CLL-313 study can be found on clinicaltrials.gov.
BRUIN CLL-314 (NCT05254743) is a Phase 3 open-label, randomized study of pirtobrutinib versus ibrutinib in patients with CLL/SLL. More information on the BRUIN CLL-314 study can be found on clinicaltrials.gov.
11. What makes BRUIN CLL-313 different from other CLL trials?

BRUIN CLL-313 (NCT05023980) is the first Phase 3 study to evaluate a non-covalent BTK inhibitor in patients with previously untreated CLL without 17p deletion.

12. With this approval, how many people living with CLL may now be eligible for treatment with Jaypirca (pirtobrutinib)?

There are roughly 23,000 new cases of CLL in the U.S. each year, and approximately 92% to 95% of patients diagnosed with CLL do not have a 17p deletion.12,13 The majority of treated patients with CLL will receive a BTK therapy at some point in their treatment, with many patients today receiving potentially only one or two lines of therapy throughout their entire treatment journey.

(Press release, Eli Lilly, OCT 2, 2026, View Source [SID1234671248])

On October 2, 2026 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical-stage biopharmaceutical company focused on developing multimodal immunotherapies to improve disease outcomes for patients with cancer, reported that the Company will present data from its aglatimagene besadenovec program in non-small cell lung cancer (NSCLC) in patients with progressive NSCLC despite immune checkpoint inhibitor (ICI) treatment, as well as clinical and mechanistic findings from its phase 1b clinical trial of linoserpaturev in recurrent glioblastoma, through two accepted poster presentations at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 41st Annual Meeting, taking place November 4-8, 2026 in Phoenix, Arizona.

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Aglatimagene besadenovec – NSCLC

Title: Multi-omics analyses reveal intratumoral and systemic immune activation to aglatimagene besadenovec+valacyclovir related to improved survival in patients with progressive NSCLC despite ICI treatment

Presenter: Caroline Duault, PharmD, PhD, Senior Research Scientist, Stanford University

Abstract Number: 603

Session Date: Friday, November 6, 2026

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Linoserpaturev – Recurrent Glioblastoma

Title: Combining linoserpaturev with Vascular Endothelial Growth Factor (VEGF) blockade to overcome hypoxia-associated immune suppression in glioblastoma

Presenter: Francesca Barone, MD, PhD, Chief Scientific Officer, Candel Therapeutics

Abstract Number: 468

Session Date: Saturday, November 7, 2026

Location: Phoenix Convention Center – North Building – Lower Level – Exhibition Halls 4, 5 and 6

About aglatimagene besadenovec

Aglatimagene besadenovec (aglatimagene), Candel’s most advanced multimodal biological immunotherapy candidate, is an investigational, off-the-shelf, replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to a patient’s tumor. After intratumoral administration, HSV-tk enzyme activity results in conversion of prodrug (valacyclovir) into deoxyribonucleic acid (DNA)-incorporating nucleotide analogs, leading to immunogenic cell death in cells exhibiting DNA damage and proliferating cells, with subsequent release of a variety of tumor (neo)antigens in the tumor microenvironment. At the same time, the adenoviral serotype 5 capsid proteins promote inflammation through the induction of expression of pro-inflammatory cytokines, chemokines, and adhesion molecules. Together, this regimen is designed to induce an individualized and specific CD8+ T cell-mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity, based on in situ immunization against a variety of tumor antigens. Aglatimagene has the potential to treat a broad range of solid tumors. Encouraging monotherapy activity as well as combination activity with standard of care radiotherapy, surgery, chemotherapy, and immune checkpoint inhibitors have previously been shown in several nonclinical and clinical settings. More than 1,000 patients have been dosed with aglatimagene in clinical trials with a favorable tolerability profile to date, supporting the potential for use with standard of care, when indicated. Aglatimagene is currently not approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority for any use.

About linoserpaturev

Linoserpaturev is a first-in-class, replication-competent, next-generation oncolytic herpes simplex virus-1 (HSV-1) immunotherapy candidate designed for dual activity for oncolysis and immune activation in a single therapeutic. In October 2023, the Company announced that Nature published results from the ongoing clinical trial where linoserpaturev was reported to be generally well-tolerated with no dose-limiting toxicity. In the clinical trial, the investigators observed improved median overall survival compared to historical controls after a single linoserpaturev injection in this therapy-resistant condition1. The Company and academic collaborators are currently supported by Break Through Cancer to evaluate the effects of repeated linoserpaturev injections in patients with recurrent glioblastoma in an expansion cohort from the phase 1b clinical trial. In October 2025, an original manuscript, published in Science Translational Medicine reported collected findings from a comprehensive analysis of 97 serial tumor biopsies collected from two patients treated with repeated administrations of linoserpaturev in arm C. Linoserpaturev previously received Fast Track Designation and Orphan Drug Designation for the treatment of recurrent high-grade glioma (rHGG) from the FDA.

(Press release, Candel Therapeutics, OCT 2, 2026, View Source [SID1234671247])

On October 2, 2026 Obsidian Therapeutics, Inc. ("Obsidian") (Nasdaq: OBX), a clinical-stage biopharmaceutical company harnessing novel protein-regulation technology to develop engineered tumor-infiltrating lymphocyte (TIL) cell therapies, reported a poster presentation of updated data in patients with immune-checkpoint inhibitor (ICI)-resistant advanced melanoma from the Phase 1 single-center first-in-human (FIH) study and the Phase 1/2 Agni-01 multicenter study of amsokigene autoleucel (amsoki-cel, formerly OBX-115), an engineered TIL cell therapy armored with pharmacologically regulatable membrane-bound IL15 (mbIL15), at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2026 Annual Meeting, taking place in Phoenix, AZ, November 4-8, 2026. The poster presentation will summarize pooled safety data across the FIH study and the Phase 1/2 Agni-01 multicenter study, and an efficacy update from Agni-01 patients treated at the recommended phase 2 dose (n=15).

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In addition, a trial-in-progress poster describing the phase 2 registration-enabling cohort of Agni-01 evaluating amsoki-cel in patients with ICI-resistant advanced melanoma will be presented.

Poster Presentation Details:

Title: OBX-115 TIL cell therapy in ICI-resistant advanced melanoma: Pooled safety and updated efficacy with minimally invasive tumor tissue procurement, low-dose lymphodepletion, and IL2-free regimen
Session Title: Poster Session
Abstract Number: 566
Date: Saturday, November 7, 2026
Location: PCC-North Building-Lower Level-Exhibition Halls 4-5-6
Lead Author: Rodabe N. Amaria, M.D., Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center

Title: Trial in progress: Agni-01 phase 2 cohort of OBX-115 tumor-infiltrating lymphocyte (TIL) cell therapy in patients with advanced melanoma
Session Title: Poster Session
Abstract Number: 570
Date: Saturday, November 7, 2026
Location: PCC-North Building-Lower Level-Exhibition Halls 4-5-6
Lead Author: Gino K. In, M.D., Division of Oncology, Norris Comprehensive Cancer Center, Keck School of Medicine, University of Southern California

(Press release, Obsidian Therapeutics, OCT 2, 2026, View Source [SID1234671246])

On October 2, 2026 Elicera Therapeutics AB (publ) reported that the company will participate in several industry, partnering and investor conferences in Sweden, Europe and the US during the fourth quarter of 2026. At these events, the company will present its business, the latest results from the Phase I part of the CARMA clinical study of the CAR T-cell therapy ELC-301, and its proprietary iTANK platform.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Elicera develops next-generation immuno-oncology treatments based on iTANK-armed CAR T cells and oncolytic viruses. The company’s CAR T-cell therapy ELC-301 is being evaluated in the Phase I/IIa clinical study CARMA for the treatment of patients with B-cell lymphoma who have no remaining treatment options. The Phase IIa part of the study has been approved by the Swedish Medical Products Agency, and patient recruitment is expected to begin in the fourth quarter of 2026.

During the fourth quarter of 2026, Elicera Therapeutics will participate in the following meetings and conferences:

Cell & Gene Meeting on the Mesa 2026
October 5-7, Phoenix, USA
Partnering meeting for the cell and gene therapy sector, organised by the Alliance for Regenerative Medicine (ARM). View Source

Symposium on the Future of Cell and Gene Therapy, hosted by CCRM
October 22, Gothenburg, Sweden
Elicera’s CEO Johan Liwing has been invited to take part in a panel discussion entitled "Bridging the competitiveness gap for ATMPs".

BioStock Life Science Summit 2026
November 4-5, Lund, Sweden
CEO Johan Liwing will present the company on 4 November at 13:45 CET.
View Source

Advanced Therapies USA 2026
November 4-5, Boston, USA
Congress and partnering meeting focused on the development and manufacturing of cell and gene therapies. Chair of the Board Margareth Jorvid will give a presentation on the topic "Effectively interacting with regulatory authorities" and take part in a panel discussion entitled "Navigating the Global Regulatory Landscape". View Source

BIO-Europe 2026
November 9-11, Cologne, Germany
Europe’s largest partnering conference for the pharmaceutical and biotech industries. https://informaconnect.com/bioeurope/

ATMP Sweden 2026 Conference
December 1-2, Campus Flemingsberg, Stockholm
Nordic conference on advanced therapy medicinal products (ATMPs), bringing together industry, academia and healthcare. View Source

Redeye Life Science Day 2026
December 2, Stockholm
CEO Johan Liwing will present the company in the morning. The presentation will be broadcast live on www.redeye.se and will be available on demand afterwards. View Source

Pharma Outsourcing 2026
December 2, Stockholm
CEO Johan Liwing has been invited to give a presentation entitled "Building for the Next Clinical Stage – How a Cell & Gene Therapy Biotech Needs to Evolve". View Source

"Backed by the positive results reported from the Phase I part of the CARMA study, we are entering a period with many opportunities to meet potential partners, investors and other players in the cell and gene therapy field. We look forward to presenting Elicera, ELC-301 and the iTANK platform, and to deepening our dialogue with both industry and the capital markets, in Sweden as well as internationally," says Johan Liwing, CEO of Elicera Therapeutics.

For further information please contact:
Johan Liwing, CEO, Elicera Therapeutics AB

Phone: +46 (0) 706 70 36 75
[email protected]

Certified Advisor
DNB Carnegie Investment Bank (publ)

About the CARMA Study
CARMA is a phase I/IIa clinical study evaluating the safety and efficacy of the CAR T-cell therapy ELC-301 in the treatment of patients with B-cell lymphoma. The study is divided into a dose-escalation phase (phase I) and a dose-expansion phase (phase IIa). Phase I primarily aims to establish the optimal dose and safety profile in up to 12 patients, while phase IIa will further evaluate the efficacy of the maximum tolerated dose in an additional six patients. Phase I is planned to include three cohorts (dosing groups), with three patients in the first and second cohorts, and six patients in the third cohort, who are expected to receive the maximum tolerated dose. The CARMA study is being conducted at Uppsala University Hospital and Karolinska University Hospital in Huddinge.

About ELC-301
ELC-301 is a fourth-generation CAR T-cell therapy targeting the CD20 antigen, armed with the company’s iTANK platform to activate a broader and more comprehensive parallel immune response against cancer. CAR T-cells are a form of cell therapy created by genetically modifying a patient’s T-cells to express a synthetic receptor (chimeric antigen receptor, CAR). This receptor is specifically designed to target a single tumor antigen—a molecule visible on the surface of cancer cells—and enables the T-cells to locate, bind to, and destroy the cancer cells.

About the iTANK-platform
The iTANK technology platform has been developed for arming and enhancing CAR T-cells to meet two of the major challenges CAR T-cell therapies face in the treatment of solid tumors: a very diverse set of tumor antigen targets and a very hostile tumor microenvironment. The technology is used to incorporate a transgene into CAR T-cells encoding a neutrophil activating bacterial protein (NAP). NAP secreted from the CAR(NAP) T-cells has been shown to be able to enhance the function of CAR T-cells and importantly activating a parallel bystander immune response against the cancer via CD8+ killer T-cells. This is expected to lead to a broad attack against most antigen targets on cancer cells. The iTANK platform is used to enhance the company’s own CAR T-cells but can also be universally applied to other CAR T-cell therapies under development. Proof-of-concept data was published in Nature Biomedical Engineering in April 2022. The publication, titled "CAR T cells expressing a bacterial virulence factor triggers potent bystander antitumor responses in solid cancers" (DOI number: 10.1038/s41551-022-00875-5) can be found here: View Source More information about iTANK platform is available here: View Source

(Press release, Elicera Therapeutics, OCT 2, 2026, View Source [SID1234671245])

On October 2, 2026 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported data from a new molecular profiling and tumor biomarker analysis of the Phase 2 RAMP 201 clinical trial of avutometinib plus defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC), as well as a new external control arm analysis comparing outcomes from RAMP 201 with conventional care. The analyses will be presented today at the International Gynecologic Cancer Society (IGCS) 2026 Annual Global Meeting in Montréal, Canada, October 1-3, 2026. Encore data from the Phase 2 RAMP 201J study in Japanese patients are also being featured in a rapid oral presentation.

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Rapid Orals: Molecular Profiling and Tumor Biomarker Analysis of RAMP 201, A Study Demonstrating Efficacy of Avutometinib in Combination with Defactinib in Recurrent Low-Grade Serous Ovarian Cancer

Date and Time: October 2, 2026, 10:30 – 11:30​ a.m. ET

Molecular profiling of baseline tumor samples from RAMP 201 was conducted to assess the activity of avutometinib plus defactinib across molecularly defined subgroups within the KRAS wild-type recurrent LGSOC population.

Among patients with KRAS wild-type LGSOC, 81% also had no NRAS and BRAF mutations. In this triple wild-type (KRAS/NRAS/BRAF) population, avutometinib plus defactinib demonstrated clinically meaningful activity, with a confirmed objective response rate (ORR) of 18% (7/39) and median progression-free survival (mPFS) of 13 months. Among patients with KRAS wild-type LGSOC with NRAS or BRAF mutations, the combination achieved a confirmed ORR of 22% (2/9).

"Patients whose tumors are wild-type for KRAS, NRAS, and/or BRAF have typically experienced response rates under 10% with trametinib, chemotherapy or endocrine therapy," said Professor Susana Banerjee, M.B.B.S., M.A., Ph.D., F.R.C.P, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust and Team Leader in Women’s Cancers at The Institute of Cancer Research, London and Global Lead Principal Investigator of ENGOTov60/GOG3052/NCRI/RAMP201. "This analysis provides additional insight into molecular biology of recurrent LGSOC and suggests that targeting RAF, MEK, and FAK with avutometinib plus defactinib may provide clinically meaningful benefit beyond tumors driven by KRAS, NRAS or BRAF mutations."

Poster Rounds with the Professor: Efficacy of Avutometinib and Defactinib vs Conventional Care in Low-Grade Serous Ovarian Cancer (LGSOC): An External Control Arm Analysis

Date and Time: October 2, 2026, 2:25 – 2:30 p.m. ET

The external control arm analysis compared outcomes among patients treated with avutometinib plus defactinib in RAMP 201 with a matched and reweighted cohort treated with conventional care, chemotherapy or anti-estrogen therapy, from the GOG 281 trial.

Among patients with KRAS-mutated LGSOC, weighted ORR was 38.7% with avutometinib plus defactinib versus 0% with conventional care and median weighted PFS was 22.1 months versus 6.5 months, respectively. Among patients with KRAS wild-type LGSOC, weighted ORR was 22.7% with avutometinib plus defactinib versus 7.9% with conventional care and median weighted PFS was 11.3 months versus 7.7 months, respectively.

"Taken together, the molecular profiling and external control arm analyses adds to the body of evidence of the potential of avutometinib plus defactinib to treat recurrent LGSOC in both KRAS-mutated and KRAS wild-type disease," said Michael Kauffman, M.D., Ph.D., president of development at Verastem Oncology. "The molecular profiling data show activity across biologically distinct subgroups of KRAS wild-type disease, including triple wild-type tumors, while the external control analysis shows higher response rates and statistically significant improvements in PFS over conventional care. Building on the established activity in KRAS-mutated recurrent LGSOC, these findings reinforce the approved use of avutometinib plus defactinib at first recurrence for patients with KRAS-mutated LGSOC, and the potential of combined RAF/MEK and FAK targeting to provide clinically meaningful benefit to a broader population of patients with KRAS wild-type recurrent LGSOC, many of whom have historically experienced poorer outcomes."

Rapid Oral Presentation: Avutometinib and Defactinib in Recurrent Low-Grade Serous Ovarian Cancer (LGSOC): A Phase II Study in Japanese Patients (RAMP 201J)

Date and Time: October 2, 2026, 10:30 – 11:30​ a.m. ET

This encore presentation includes results from RAMP 201J previously reported at the Annual Meeting of the Japanese Society of Gynecologic Oncology, held July 17–19, 2026, in Sapporo, Japan. As of May 29, 2026, 16 efficacy-evaluable patients with recurrent LGSOC received avutometinib plus defactinib, with a median follow-up of 12.4 months.

The combination achieved a 44% ORR and 94% disease control rate across all patients. ORRs were 71% in patients with KRAS-mutated tumors and 22% in those with KRAS wild-type tumors, with disease control rates of 100% and 89%, respectively. Overall, 94% of patients experienced tumor shrinkage, and 11 of 16 remained on treatment at the data cutoff.

​At the IGCS 2026 Annual Global Meeting, visit Verastem’s exhibition booth (#01) for more information on its approved therapy and ongoing cancer research. The full schedule and full presentation abstracts are available on the IGCS Annual Global Meeting web site.

About RAMP 201
RAMP 201 (ENGOTov60/GOG3052/NCRI) (NCT04625270) was an adaptive, two-part multicenter, parallel cohort, randomized, open-label Phase 2 registration-directed trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). The first part of the trial (Part A) determined the selection of the go-forward regimen, which was the combination of avutometinib and defactinib versus avutometinib alone, based on overall response rates. The expansion phases of the trial (Parts B and C) evaluated the safety and efficacy of the go-forward regimen of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily. The Part D portion of the trial evaluated a low dose of the combination to inform individualized dose reduction.

About Low-Grade Serous Ovarian Cancer (LGSOC)
LGSOC is a rare ovarian cancer that is insidious and persistent. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. Approximately 70 percent of LGSOC shows RAS pathway-associated mutations, and 30 percent of people with LGSOC have a KRAS mutation. The majority of patients report a negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life.

About AVMAPKI and FAKZYNJA Combination Therapy
AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.

The U.S. Food and Drug Administration (FDA) approved AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. In June 2026, Verastem provided updated results on the Phase 1/2 RAMP 205 trial (NCT05669482), which is evaluating avutometinib plus defactinib in combination with standard-of-care chemotherapy as a first-line treatment for patients with advanced pancreatic cancer. Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.

AVMAPKI FAKZYNJA CO-PACK U.S. Indication

Indication

AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.

This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Important Safety Information

Warnings and Precautions

Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.
Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.
Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.
Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.
Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.
Adverse Reactions

The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.

Drug Interactions

Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.
Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.
Use in Specific Populations

Lactation: Advise not to breastfeed.
Fertility: May impair fertility in males and females.
Click here for full Prescribing Information.

(Press release, Verastem, OCT 2, 2026, View Source [SID1234671244])