On April 29, 2026 Laguna Biotherapeutics, Inc. (Laguna), a clinical-stage biotechnology company focused on novel live bacterial therapeutics, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application, granting a "safe to proceed" for its lead clinical candidate from the QUAIL platform, LGNA-100.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

LGNA-100 is a first-in-class, attenuated live bacterial immunotherapy designed to safely harness our immune system’s evolved response to Listeria; robustly and durably expanding and activating endogenous γδ T cells that can directly kill cancer cells while also improving existing immunotherapies. The Phase 1 first-in-human study will validate the QUAIL platform and evaluate LGNA-100 in patients with high-risk leukemia following hematopoietic stem cell transplantation (HSCT) to prevent leukemic relapse.

"The IND clearance of our first clinical study is a defining moment and transformative milestone for Laguna as we transition into a clinical-stage company," said Jonathan Kotula, Ph.D., CEO of Laguna. "Our goal is to create systems-level therapies for complex diseases. With LGNA-100, and the QUAIL platform we are taking a fundamentally new approach to selectively stimulate innate T cells to improve long-term outcomes for pediatric patients with high-risk leukemia."

The clinical rationale for the QUAIL platform builds directly upon decades of research into the human γδ T cell response to Listeria, and the protective role of γδ T cells against leukemia recurrence following HSCT. A presentation covering our clinical rationale will be presented at the ISCT 2026 Annual Meeting on May 6 in Dublin, Ireland.

"In the setting of αβ-depleted HSCT, γδ T cells are critical effectors that provide potent graft-versus-leukemia activity without driving graft-versus-host disease," said Dr. Alice Bertaina, MD, PhD, Co-Director of the Bass Center for Childhood Cancer and Blood Diseases, Lucile Packard Children’s Hospital at Stanford University and lead Clinical Advisor for Laguna. "While early pharmacologic activators like zoledronic acid (ZOL) showed the clinical potential of this approach, intense ZOL stimulation pushes these cells into a more mature, terminally differentiated and exhausted state. Our comprehensive preclinical evaluations demonstrate that LGNA-100 drives a distinct, multifunctional γδ T cell response with improved kinetic, phenotypic, and functional features compared to ZOL, supporting more durable activation without hyperactivation or early exhaustion. I am very excited to see this translated into the clinic for these high-risk leukemia patients."

"Pediatric AML remains one of the most challenging frontiers in oncology, demanding novel modalities that can detect and kill the disease without compounding toxicities," said Bill Newell, former CEO of Sutro Biopharma and Strategic Advisor to Laguna. "Having spent years evaluating platforms to tackle these exact malignancies, I believe Laguna’s approach using γδ T cells to potentially solve the problems associated with high-risk leukemia is a massive leap forward. Securing this IND is a testament to the rigor of their science and positions LGNA-100 as a highly differentiated asset in the cancer immunotherapy space."

The Phase 1 clinical study is a company-sponsored open-label, first-in-human, single ascending dose study designed to assess safety and tolerability, and support the proof of LGNA-100’s mechanism of action. The study will evaluate LGNA-100 administered via intravenous (IV) infusion in pediatric and young adult participants with high-risk acute leukemias and MDS who have received an αβ-depleted HSCT.

About LGNA-100

LGNA-100 also known as QUAIL-100 is an investigational cancer immunotherapeutic agent derived from Listeria monocytogenes (Lm), developed from the QUAIL platform, designed to activate and expand a patient’s endogenous γδ T cells.

(Press release, Laguna Bio, APR 29, 2026, View Source [SID1234664922])

On April 29, 2026 Spherix reported breadth of physician-driven research underscores emerging trends in oncology as three abstracts are accepted at ASCO (Free ASCO Whitepaper) 2026:

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Operational and clinical drivers of subcutaneous PD-1/PD-L1 inhibitor adoption: A mixed-methods analysis of U.S. oncology practice patterns.
Tumor-specific drivers of PD-1/PD-L1 inhibitor brand preference in NSCLC, melanoma, and renal cell carcinoma: A U.S. oncologist perspective.
Treatment decision-making after CAR-T and bispecific antibody therapy in relapsed/refractory multiple myeloma: Real-world perspectives from U.S. hematologist/oncologists.
Spherix Global Insights continues to expand its presence in oncology market intelligence with the release of new findings from its RealTime Dynamix service focused on PD-1 inhibition in solid tumors, alongside the company’s acceptance of three abstracts at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, including one poster presentation, coauthored with Dr. Kimberly Ku of Illinois CancerCare. Together, these milestones underscore both the breadth of Spherix’s oncology research portfolio and the growing impact of its physician-derived insights on the broader oncology community.

The latest RealTime Dynamix research, based on a survey of more than 100 U.S. oncologists, highlights a rapidly evolving PD-1 landscape marked by increasing overall utilization, meaningful shifts in route of administration, and emerging complexities in real-world treatment decision-making.

Across tumor types, oncologists report that use of PD-1 inhibitor regimens is expected to grow over the next six months, reinforcing the class’s continued central role in solid tumor management. At the same time, the data reveal a notable transition from traditional intravenous administration toward subcutaneous options. While intravenous regimens remain foundational – often serving as the preferred starting point – physicians increasingly signal a willingness to adopt subcutaneous alternatives, particularly in maintenance settings where convenience and efficiency are more highly valued.

Importantly, this shift is not without friction. Spherix findings suggest that while uptake of subcutaneous PD-1 inhibitors is accelerating, particularly in monotherapy settings, utilization may be more limited in combination regimens due to logistical challenges and access considerations encountered in real-world practice. These dynamics highlight a nuanced adoption curve, where clinical enthusiasm is tempered by operational realities.

The competitive landscape remains highly differentiated by tumor type. Pembrolizumab (Keytruda, Merck) continues to lead across multiple non-small cell lung cancer (NSCLC) segments and renal cell carcinoma (RCC), while nivolumab (Opdivo, Bristol Myers Squibb) maintains a strong position in melanoma. However, the introduction of subcutaneous formulations is reshaping share dynamics within brands, with meaningful internal shifts from intravenous to subcutaneous use projected across key indications in the coming months.

Beyond branded therapies, Spherix research points to a growing awareness of PD-1 biosimilars in development, though physician visibility into actual product selection remains limited. Oncologists largely recognize the value of biosimilars – particularly in high-volume settings such as NSCLC – yet report that treatment decisions are often dictated at the institutional or payer level, reducing direct physician control over brand choice.

Collectively, these findings illustrate a market in transition, where innovation in formulation, evolving treatment strategies, and cost considerations are converging to shape the next phase of PD-1 utilization.

Spherix’s upcoming presence at ASCO (Free ASCO Whitepaper) 2026 builds on this momentum. The company’s three accepted abstracts, including a poster presentation, reflect the depth and rigor of its oncology research and mark a significant milestone as Spherix brings its real-world evidence and physician insights to one of the field’s most prominent global stages for the first time.

The accepted research spans key areas of oncology, further reinforcing Spherix’s commitment to delivering timely, actionable insights that inform strategic decision-making across the healthcare ecosystem. By pairing robust primary research with deep therapeutic expertise, Spherix continues to provide a uniquely comprehensive view of rapidly evolving markets.

As the oncology landscape grows increasingly complex, Spherix remains focused on illuminating the intersection of clinical innovation and real-world practice – equipping stakeholders with the clarity needed to navigate change and anticipate what comes next.

About RealTime Dynamix

RealTime Dynamix is an independent service providing strategic guidance through quarterly or semiannual reports, which include market trending and a fresh infusion of event-driven and variable content with each wave. The reports provide an unbiased view of the competitive landscape within rapidly evolving specialty markets, fueled by robust HCP primary research and our in-house team of experts.

(Press release, Spherix, APR 29, 2026, View Source [SID1234664921])

On April 29, 2026 Telix Pharmaceuticals Limited (ASX: TLX, NASDAQ: TLX, "Telix") reported dosimetry results from the randomized Phase 2 OPTIMAL-PSMA trial of TLX597-Tx in metastatic castration-resistant prostate cancer (mCRPC), presented at the 2026 International Prostate Cancer Symposium (IPCS 2026) held in Lugano, Switzerland. These findings support TLX597-Tx’s potential to deliver a treatment that overcomes quality-of-life challenges that currently limit the clinical utility of existing RLTs in earlier-stage metastatic prostate cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

TLX597-Tx (177Lu-DOTA-HYNIC-panPSMA) is a novel PSMA-targeting small molecule RLT candidate with a highly favorable dosimetry profile. Significantly reduced radiation exposure to healthy organs, including the salivary glands and kidneys5, may lower the incidence of xerostomia (dry mouth) and renal toxicity and support better tolerability for patients. This dosimetry profile combined with higher tumor uptake compared to existing PSMA RLTs may deliver a wider therapeutic window and enable dose intensification to maximize tumor control while preserving patient quality-of-life.

OPTIMAL-PSMA is an open-label, multi-center, randomized, Phase 2 investigator-initiated trial (IIT) led by Professor Louise Emmett at St Vincent’s Hospital in Sydney, Australia. The study is evaluating the safety, dosimetry, and efficacy of an intensified dosing regimen of TLX597-Tx compared with a standard dose schedule in 120 men with advanced mCRPC, randomized on a 2:1 basis. The novel dose-intensification regimen delivers higher activity per cycle (8.5 GBq), delivered on day 1, day 3 and day 15, followed by 10-weekly dosing for three further cycles. The study aims to confirm that a dose intensified TLX597-Tx regimen will maximize the radiation dose to cancerous lesions when they are most vulnerable to damage and therefore improve overall response to treatment.

Telix believes these dosimetry data support further evaluation of TLX597-Tx in earlier-stage disease and is initiating OPTIMAL-E, a Phase 2 study in androgen pathway-sensitive prostate cancer (mHSPC).

Principal Investigator for OPTIMAL-PSMA, Professor Louise Emmett, commented, "The goal of OPTIMAL-PSMA is to identify a dose regimen for TLX597-Tx that leads to deeper and longer responses without increasing toxicity for men with metastatic prostate cancer. We look forward to starting the Phase 2 OPTIMAL-E trial soon."

Dr. David N. Cade, Group Chief Medical Officer at Telix, added, "These encouraging dosimetry results from OPTIMAL-PSMA, combined with earlier exploratory work6, are very promising and highlight TLX597-Tx’s potential to substantially increase the tumor dose while minimizing radiation to sensitive organs. For people living with earlier-stage metastatic disease, preserving quality-of-life alongside effective cancer control is mandatory. These findings support further study in mHSPC and reinforce Telix’s strategy to develop differentiated PSMA-targeting therapies, so clinicians may be able to tailor treatment choice to the patient’s disease stage and individual condition."

TLX597-Tx is being developed alongside TLX591-Tx (lutetium-177 (177Lu) rosopatamab tetraxetan), Telix’s lead antibody-based prostate cancer therapy candidate, currently the subject of the Phase 3 ProstACT Global7 trial in mCRPC, which is actively dosing patients in jurisdictions with regulatory approval and recently reported data from a safety and dosimetry lead-in8. TLX591-Tx and TLX597-Tx exhibit complementary modes-of-action, suggesting the potential for distinct applications in mCRPC and mHSPC settings as part of Telix’s portfolio approach to treating prostate cancer. TLX591-Tx and TLX597-Tx have not received marketing authorization in any jurisdiction.

(Press release, Telix Pharmaceuticals, APR 29, 2026, View Source [SID1234664920])

On April 29, 2026 Pfizer Inc. (NYSE: PFE) reported positive topline results from the Phase 3 MagnetisMM-5 study evaluating ELREXFIO (elranatamab) as monotherapy in adults with relapsed or refractory multiple myeloma (RRMM) who received at least one prior line of treatment. The study demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of progression‑free survival (PFS), as assessed by blinded independent central review (BICR), versus standard-of-care daratumumab plus pomalidomide and dexamethasone (DPd). The safety and tolerability of ELREXFIO was consistent with its known safety profile.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The PFS results exceeded the pre-specified interim analysis target hazard ratio for efficacy, with most ELREXFIO-treated patients remaining progression-free. The trial remains ongoing to assess overall survival, a key secondary endpoint, which was not yet mature at the time of this interim analysis. These data will be discussed with global health authorities, and detailed results from MagnetisMM-5 will be submitted for presentation at a future medical congress.

Multiple myeloma is an aggressive and currently incurable blood cancer that affects plasma cells made in the bone marrow:

It is the second most common type of blood cancer worldwide, with over 36,000 new cases each year in the United States and over 187,000 globally.1,2,3
Despite treatment advances, most patients relapse and develop relapsed or refractory disease, often requiring multiple lines of therapy, with approximately 40% not surviving beyond five years.2,4
Multiple myeloma can significantly impact quality of life, with immune suppression increasing susceptibility to infection, and symptoms such as fatigue, bone pain, and psychological distress making everyday activities more difficult.5-7
"Effective intervention earlier in the course of disease represents a critical opportunity to improve outcomes for people living with multiple myeloma," said Jeff Legos, Chief Oncology Officer, Pfizer. "ELREXFIO has already helped address a significant unmet need in heavily pre‑treated patients, delivering deep, durable, and clinically meaningful responses. The MagnetisMM-5 results reinforce our confidence in ELREXFIO’s potential to benefit patients earlier in their treatment journey and support our comprehensive strategy to evaluate ELREXFIO both as monotherapy and as part of combination approaches across multiple lines of therapy."

ELREXFIO is approved in more than 35 countries worldwide, including in the United States where it received accelerated approval for use in adults with RRMM who have received at least four prior lines of therapy, and in the European Union where it was granted conditional marketing authorization for adults with RRMM who have received at least three prior therapies and have demonstrated disease progression on the last therapy.

MagnetisMM-5 is part of a comprehensive development program in double-class exposed MM patients. The program includes the fully recruited Phase 3 MagnetisMM-32 study, which is designed to evaluate ELREXFIO in patients who have previously received daratumumab as part of standard-of-care front-line MM treatment.

About Multiple Myeloma
Multiple myeloma is an aggressive and currently incurable blood cancer that affects plasma cells made in the bone marrow. Healthy plasma cells make antibodies that help the body fight infection.6 While disease trajectory varies for each person, relapses are nearly inevitable.9 The goal of therapy for people with relapsing or refractory multiple myeloma is to achieve disease control with acceptable toxicity and improved quality of life.10

About MagnetisMM-5
MagnetisMM-5 is an open-label, multicenter, randomized Phase 3 study to evaluate the efficacy and safety of ELREXFIO versus standard-of-care daratumumab plus oral pomalidomide and dexamethasone in patients with RRMM. The study enrolled 497 patients across 26 countries who have received at least one line of previous treatment, including lenalidomide and a proteasome inhibitor (PI). Participants received subcutaneous ELREXFIO as two step-up priming doses followed by a weekly 76 mg injection, with frequency adjusted to every two weeks after 24 weeks if a partial response was achieved for more than two months, and again to every four weeks for all patients after 48 weeks on treatment. The primary endpoint is progression-free survival (PFS), as assessed by blinded independent central review (BICR). A key secondary endpoint is overall survival.

Pfizer continues to evaluate ELREXFIO both as a monotherapy and as a combination regimen as part of the MagnetisMM clinical trial program.

About ELREXFIO (elranatamab)
ELREXFIO is a subcutaneously delivered B-cell maturation antigen (BCMA)-cluster of differentiation CD3-directed bispecific antibody immunotherapy that binds to BCMA on myeloma cells and CD3 on T cells, activating the T cells to kill myeloma cells.

U.S. Important Safety Information and Indication

ELREXFIO may cause side effects that are serious, life-threatening, or can lead to death, including cytokine release syndrome (CRS) and neurologic problems. CRS is common during treatment with ELREXFIO.

Tell your healthcare provider or get medical help right away if you develop any signs or symptoms of CRS or neurologic problems, including:

fever of 100.4°F (38°C) or higher
trouble breathing
chills
dizziness or light-headedness
fast heartbeat
headache
increased liver enzymes in your blood
agitation, trouble staying awake, confusion or disorientation, or seeing or hearing things that are not real (hallucinations)
trouble speaking, thinking, remembering things, paying attention, or understanding things
problems walking, muscle weakness, shaking (tremors), loss of balance, or muscle spasms
numbness and tingling (feeling like "pins and needles")
burning, throbbing, or stabbing pain
changes in your handwriting
Due to the risk of CRS, you will receive ELREXFIO on a "step-up" dosing schedule and should be hospitalized for 48 hours after the first "step-up" dose and for 24 hours after the second "step-up" dose of ELREXFIO.

For your first dose, you will receive a smaller "step-up" dose of ELREXFIO on day 1
For your second dose, you will receive a larger "step-up" dose of ELREXFIO, which is usually given on day 4 of treatment
For your third dose, you will receive the first "treatment" dose of ELREXFIO, which is usually given on day 8
If your dose of ELREXFIO is delayed for any reason, you may need to repeat step-up dosing. Before each dose of ELREXFIO during the step-up dosing schedule, you will receive medicines to help reduce your risk of CRS. Your healthcare provider will decide if you need to receive medicines to help reduce your risk of CRS with future doses.

ELREXFIO is available only through the ELREXFIO Risk Evaluation and Mitigation Strategy (REMS) Program due to the risk of CRS and neurologic problems. You will receive an ELREXFIO Patient Wallet Card from your healthcare provider. Carry the ELREXFIO Patient Wallet Card with you at all times and show it to all of your healthcare providers. The ELREXFIO Patient Wallet Card lists signs and symptoms of CRS and neurologic problems. Get medical help right away if you develop any of the signs and symptoms listed on the ELREXFIO Patient Wallet Card. You may need to be treated in a hospital.

Before taking ELREXFIO, tell your healthcare provider about all of your medical conditions, including if you:

have an infection
are pregnant or plan to become pregnant. ELREXFIO may harm your unborn baby. Females who are able to become pregnant should do a pregnancy test before starting treatment with ELREXFIO and should use effective birth control during treatment and for four months after your last dose of ELREXFIO. Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with ELREXFIO
are breastfeeding or plan to breastfeed. It is not known if ELREXFIO passes into your breast milk. Do not breastfeed during treatment and for four months after your last dose of ELREXFIO
Tell your healthcare provider about all of the medications you take, including prescription and over-the-counter medications, vitamins, and herbal supplements.

Do not drive, operate heavy or potentially dangerous machinery, or do other dangerous activities during treatment with ELREXFIO:

for 48 hours after completing each of the 2 doses of ELREXFIO that are part of the "step-up dosing schedule" and your first full treatment dose, and
at any time during treatment with ELREXFIO if you develop any new neurologic symptoms, such as dizziness, confusion, shaking (tremors), sleepiness, or any other symptom that impairs consciousness, until the symptoms go away
Infections: Upper respiratory tract infection and pneumonia are common during treatment with ELREXFIO. ELREXFIO can cause bacterial and viral infections that are severe, life-threatening, or that may lead to death.

Your healthcare provider may prescribe medications for you to help prevent infections and treat you as needed if you develop an infection during treatment with ELREXFIO
Tell your healthcare provider right away if you develop any signs or symptoms of an infection during treatment with ELREXFIO, including: fever of 100.4°F (38°C) or higher, chills, cough, shortness of breath, chest pain, sore throat, pain during urination, or feeling weak or generally unwell
People with active infections should not start ELREXFIO
Decreased white blood cell counts: Decreased white blood cell counts are common during treatment with ELREXFIO and can also be severe. A fever can occur with low white blood cell counts and may be a sign that you have an infection. Your healthcare provider will treat you as needed.

Liver problems: ELREXFIO can cause increased liver enzymes and bilirubin in your blood. These increases can happen with or without you also having CRS. Tell your healthcare provider if you develop any of the following signs or symptoms of a liver problem, including:

tiredness
loss of appetite
pain in your right upper stomach-area
dark urine
yellowing of your skin or the white part of your eyes
The most common side effects of ELREXFIO include:

tiredness
injection site reaction, such as redness, itching, pain, bruising, rash, swelling, and tenderness
diarrhea
muscle and bone pain
decreased appetite
rash
cough
nausea
fever
The most common severe abnormal lab test results with ELREXFIO include decreased white blood cells, red blood cells, and platelets.

Your healthcare provider may temporarily or permanently stop ELREXFIO if you have any of the side effects listed and they are severe. These are not all of the possible side effects of ELREXFIO.

Call your healthcare provider for medical advice about side effects. You may report side effects to the U.S. Food and Drug Administration (FDA) at 1-800-FDA-1088.

What is ELREXFIO?

ELREXFIO is a prescription medication used to treat adults with multiple myeloma who:

have already received at least 4 treatment regimens, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody, to treat their multiple myeloma, and
their cancer has come back or did not respond to prior treatment
ELREXFIO was approved based on patient responses and durability of response. There are ongoing studies to confirm its clinical benefit. It is not known if ELREXFIO is safe and effective in children.

Please read full Prescribing Information, including BOXED WARNING, for ELREXFIO.

(Press release, Pfizer, APR 29, 2026, View Source [SID1234664918])

On April 29, 2026 Immunome, Inc. (Nasdaq: IMNM), a biotechnology company committed to developing first-in-class and best-in-class targeted cancer therapies, reported the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for varegacestat, an investigational, oral, once-daily gamma secretase inhibitor (GSI), for the treatment of adults with desmoid tumors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"The varegacestat NDA submission marks an important milestone for Immunome. It reflects the strength of the RINGSIDE dataset and the commitment of the team advancing this program," said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Immunome. "We believe varegacestat has the potential to provide adults with progressing desmoid tumors with a meaningful new oral treatment option, and we are grateful to the patients, families, investigators and study site teams whose participation made this submission possible."

The NDA is supported by positive results from the global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial of varegacestat in patients with progressing desmoid tumors.

The trial met its primary endpoint of improving progression-free survival, demonstrating a statistically significant and clinically meaningful improvement vs. placebo, with an 84% reduction in the risk of disease progression or death (hazard ratio (HR) = 0.16, 95% CI: 0.071, 0.375; p<0.0001). The confirmed objective response rate (ORR) based on RECIST v1.1 was 56% with varegacestat vs. 9% with placebo (p<0.0001), as assessed by blinded independent central review.

In an exploratory analysis, varegacestat demonstrated a median best change in tumor volume of -83% vs. +11% with placebo, as assessed by blinded independent central review. In addition, the trial met all key secondary endpoints, with varegacestat achieving statistically significant improvements vs. placebo in landmark tumor volume reduction and worst pain intensity.

Varegacestat was generally well tolerated, with a manageable safety profile consistent with the GSI class. The most common adverse events for participants in the treatment arm were diarrhea (82%), fatigue (44%), rash (43%), nausea (35%) and cough (34%). Most events were grade 1 or 2.

Immunome previously announced that data from RINGSIDE has been selected for presentation in an oral abstract session at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place May 29-June 2, 2026 in Chicago.

About the RINGSIDE Trial

The global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial (NCT04871282) evaluated the efficacy and safety of varegacestat in patients with progressing desmoid tumors. A total of 156 patients were randomized to receive varegacestat 1.2 mg daily or placebo until disease progression or death, representing the largest randomized study in this population. The primary endpoint of the trial was progression-free survival as assessed by blinded independent central review. Statistically controlled secondary endpoints were confirmed ORR using RECIST v1.1 and change in tumor volume at week 24, both determined by blinded independent central review, as well as change in pain intensity at week 12 as determined using a patient reported outcome instrument. Additional secondary endpoints included duration of response, best reduction in tumor volume, patient-reported outcomes, and safety and tolerability. RINGSIDE includes an open-label extension phase, which is ongoing.

About Desmoid Tumors

Desmoid tumors (also known as aggressive fibromatosis or desmoid-type fibromatosis) are aggressive non-metastatic soft tissue tumors that are prone to recurrence. Approximately 1,000-1,650 people are diagnosed with desmoid tumors each year in the United States, and there are approximately 10,000-11,000 actively managed patients. Those affected face debilitating pain, deformity and, in some cases, life-threatening organ damage. The chronic pain and physical limitations associated with desmoid tumors lead to a high clinical burden and impaired quality of life. Although desmoid tumors are not considered cancerous, they often require systemic treatment to prevent permanent disability and alleviate disease burden.

About Varegacestat

Varegacestat (formerly AL102) is an investigational, oral, once-daily gamma secretase inhibitor. In December 2025, Immunome reported positive topline results for the Phase 3 RINGSIDE trial of varegacestat in adults with progressing desmoid tumors. Data from RINGSIDE have been selected for oral presentation at the 2026 ASCO (Free ASCO Whitepaper) Annual Meeting.

(Press release, Immunome, APR 29, 2026, View Source [SID1234664917])