On May 14, 2025 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for diseases with high unmet need, reported the strategic prioritization and acceleration of PBGENE-DMD, the Company’s first-in-class in vivo gene editing approach for Duchenne Muscular Dystrophy (DMD) and highlighted a PBGENE-DMD poster presentation at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting being held May 13-17, 2025, in New Orleans, Louisiana (Press release, Precision Biosciences, MAY 14, 2025, View Source [SID1234653078]).

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"Nomination and acceleration of PBGENE-DMD as our second wholly owned program is a result of the compelling preclinical evidence we have generated to date," said Michael Amoroso, President and Chief Executive Officer of Precision BioSciences. "Currently, there are no approved treatments or treatments in development that significantly improve muscle function over time to beneficially alter the long-term prognosis of this devastating disease. PBGENE-DMD is the first in vivo gene editing program that has the potential to transform the treatment paradigm and deliver durable functional improvement for most patients, as up to 60% of those afflicted carry mutations in the ‘hot spot’ region between exons 45-55. Based on these data, the significant unmet need in DMD and the clear regulatory guidance established for new therapeutics in DMD, we are committed to advancing PBGENE-DMD to the next stage of development. We look forward to rapidly advancing this program toward the clinic as our second wholly owned program after PBGENE-HBV and further establishing the therapeutic potential of ARCUS in vivo gene editing."

DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the U.S. alone. There are currently no approved therapies that can drive significant and durable functional muscle improvements. PBGENE-DMD employs two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring the body’s natural production of a functional dystrophin protein.

"PBGENE-DMD has the potential to provide a one-time, durable intervention that could allow for lifelong benefits in muscle regeneration and function. Preclinical models have shown that PBGENE-DMD results in significant and sustained improvement of maximum force output by restoring the human body’s production of a functional, near full-length dystrophin protein," added Cassie Gorsuch, Ph.D., Chief Scientific Officer at Precision BioSciences. "Approximately one in every 3,500–5,000 males in the United States is afflicted with DMD, and these patients have limited treatment options. Our prioritization of PBGENE-DMD in conjunction with long term, preclinical functional improvement reinforces our belief in the program and its potential to address significant unmet need for patients living with DMD."

In preclinical data being presented at ASGCT (Free ASGCT Whitepaper), PBGENE-DMD demonstrated significant and durable functional improvement in a humanized DMD mouse model. Following AAV delivery, PBGENE-DMD restored the body’s ability to produce a functional dystrophin protein broadly across multiple muscles, including cardiac and skeletal muscles. Over the course of 9 months, mice treated with PBGENE-DMD showed increased dystrophin protein expression resulting in substantial and sustained functional muscle improvement. In addition, PBGENE-DMD-edited dystrophin mRNA transcript in muscle satellite stem cells, which are progenitor cells for new muscle cells, supports the potential for long-term durability.

"Precision’s ARCUS gene excision approach targeting exons 45-55 represents a novel approach to addressing the underlying disease for the majority of patients with Duchenne muscular dystrophy," said Debra Miller, founder and CEO of CureDuchenne. "We are excited by the potential of this approach and look forward to Precision educating the DMD community about it at our upcoming FUTURES National Conference on May 24." CureDuchenne is a nonprofit organization recognized as the global leader in research, patient care and innovation for improving and extending the lives of those with Duchenne muscular dystrophy.

Precision is working diligently and targeting to file an IND and/or CTA in 2025 with clinical data anticipated in 2026. The Company believes that its current cash runway will be sufficient to progress both PBGENE-HBV, its current Phase 1 clinical program, and PBGENE-DMD through Phase 1 clinical readouts.

In order to accelerate development of PBGENE-DMD and maintain operational capability to pursue PBGENE-HBV and PBGENE-DMD through Phase 1 clinical results, Precision plans to pause development of PBGENE-3243, its potential treatment for m.3243-associated mitochondrial disease, and will stage future development alone or with partners following completion of the Phase 1 ELIMINATE-B trial and after the PBGENE-DMD program enters the clinic. Precision has completed pre-IND discussions with regulators for PBGENE-3243 and the final clinical candidate is ready to commence toxicology studies. "We remain excited about the potential for PBGENE-3243 to help people living with m.3243 mitochondrial diseases and remain committed to those afflicted with m.3243 associated mitochondrial disease in the future, alone or through partnerships," added Mr. Amoroso.

Conference Call Information

Precision BioSciences will host a conference call on Thursday, May 15 at 8:00 am ET. To access the live conference call, participants may register here. The live audio webcast of the call will be available in the Investors section under Events & Presentations at investor.precisionbiosciences.com. An archived replay of the webcast will be available for approximately 30 days following the event.

PBGENE-DMD Presentation Details:

Title: ARCUS-Mediated Gene Editing Excision of Exons 45-55 of the Human Dystrophin Gene using PBGENE-DMD Leads to Functional Dystrophin Protein and Durable Restoration of Skeletal Muscle-Function In Vivo for the Treatment of Duchenne Muscular Dystrophy

Session: Poster Reception

Date and Time: Wednesday, May 14, 2025, 5:30 PM – 7:00 PM CT

Location: Poster Hall I2

On May 14, 2025 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs), reported data presentations from the LOTIS-7 Phase 1b clinical trial evaluating ZYNLONTA (loncastuximab tesirine-lpyl) in combination with glofitamab in patients with relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL) (Press release, ADC Therapeutics, MAY 14, 2025, View Source [SID1234653097]). Updated results will be shared at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress (EHA2025) taking place in Milan, Italy with an oral encore presentation at the 18th International Conference on Malignant Lymphoma (ICML) in Lugano, Switzerland. Updated LOTIS-5 safety run-in data evaluating the combination of ZYNLONTA plus rituximab (Lonca-R) will also be featured at EHA (Free EHA Whitepaper)2025.

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"We are excited to present the latest LOTIS-7 data from a larger subset of patients with longer follow-up at EHA (Free EHA Whitepaper) and ICML," said Mohamed Zaki, MD, PhD, Chief Medical Officer of ADC Therapeutics. "The robust efficacy and manageable safety seen to date with the combination of ZYNLONTA and glofitamab, two potent anti-cancer agents with different mechanisms of action, reinforce the potential for this regimen to change the treatment paradigm for patients with aggressive lymphoma."

The LOTIS-7 abstract provides data as of the January 17, 2025, cutoff, in which 31 patients received ≥1 ZYNLONTA dose and were safety evaluable, with 22 patients efficacy evaluable. Four of these patients (2 each at 120µg/kg and 150 µg/kg) converted to complete response (CR) within 3 weeks after the data cutoff and are included as CRs. Updated data will be presented during EHA (Free EHA Whitepaper)2025.

Key highlights in the LOTIS-7 abstract are as follows:

In the efficacy evaluable population, overall response rate (ORR) was 95.5% (21/22), complete response (CR) rate was 90.9% (20/22), and median duration of response (DOR) was not reached.
Among 31 patients treated, the combination demonstrated a manageable safety profile.
Adverse events were consistent with known profiles of the individual agents, with neutropenia (32.3%) being the most common Grade ≥3 treatment-emergent adverse event (TEAEs). Grades 3/4 TEAEs of interest included generalized edema, pericardial effusion, photosensitivity reaction, rash, sepsis and pneumonia (each 3.2%). Grade 1/2 AE of CRS (29.0%/9.7%) and ICANS (0%/6.5%) were observed with no Grade ≥3 at the time of data cut off.
Details of the EHA (Free EHA Whitepaper)2025 poster presentations are as follows:
Title: Initial Results From LOTIS-7: A Phase 1b Study of Loncastuximab Tesirine Plus
Glofitamab in Patients With Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL)
Session: Poster Session 2
Session Date and Time: Saturday, June 14; 12:30-1:30 p.m. ET / 18:30 -19:30 CEST
Location: Poster Hall, Allianz MiCo, Milano Convention Centre
Presenting Author: Juan Pablo Alderuccio, MD, Associate Professor of Medicine and Hematologist at Sylvester Comprehensive Cancer Center, University of Miami
Abstract: PS1911

Title: Updated Safety Run-In Results From LOTIS-5: A Phase 3, Randomized Trial of Loncastuximab Tesirine With Rituximab Versus Immunochemotherapy in Patients With R/R DLBCL/HGBL
Session: Poster Session 2
Session Date and Time: Saturday, June 14; 12:30-1:30 p.m. ET / 18:30 -19:30 CEST
Location: Poster Hall, Allianz MiCo, Milano Convention Centre
Presenting Author: Carmelo Carlo-Stella, MD, PhD, section chief of Lymphoid Malignancies and Cancer Therapeutics at Humanitas Clinical and Research Center (IRCCS)
Abstract: PS1957

Details of the ICML oral encore presentation are as follows:
Title: Initial Results From LOTIS-7: A Phase 1b Study of Loncastuximab Tesirine Plus Glofitamab in Patients With Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL)
Session: 13 – Aggressive B-Cell Lymphomas
Session Date and Time: Friday, June 20; 8:00 a.m.-9:30 a.m. ET / 14:00-15:30 CEST
Location: Room A, broadcast in Cinema Corso, Lugano Convention Centre, Palazzo dei Congressi
Presenting Author: Juan Pablo Alderuccio, MD, Associate Professor of Medicine and Hematologist at Sylvester Comprehensive Cancer Center, University of Miami
Abstract: 078

Additionally, a poster entitled, "Updated analysis of a phase 2 multicenter study of the loncastuximab in relapsed/refractory marginal zone lymphoma demonstrates high rate of complete responses" will be presented at ICML. This single-arm, open-label investigator-initiated study is being conducted at the Sylvester Comprehensive Cancer Center at University of Miami and City of Hope, and led by Izidore Lossos, MD, Professor, Director, Lymphoma Program at the Sylvester Comprehensive Cancer Center, University of Miami.

About LOTIS-7
LOTIS-7 is a Phase 1b global multicenter, multi-arm study in patients with relapsed or refractory B-cell non-Hodgkin lymphoma (B-NHL) including Part 1 (dose escalation) and Part 2 (dose expansion). The three dosing arms include ZYNLONTA plus polatuzumab vedotin, ZYNLONTA plus glofitamab, and ZYNLONTA plus mosunetuzumab T-cell-engaging bispecific monoclonal antibodies (BsAbs). Enrollment in LOTIS-7 includes Part 1 of the study with a 3+3 dose escalation in 3L/3L+ heavily pre-treated patients with ZYNLONTA doses starting at 90 µg/kg and then proceeding to 120 µg/kg and 150 µg/kg. Part 2 includes dose expansion in 2L/2L+ large B-cell lymphoma in the ZYNLONTA plus glofitamab arm at dose levels determined from Part 1 (120 µg/kg and 150 µg/kg of ZYNLONTA plus the approved dosing of glofitamab). Primary endpoints of the study include safety and tolerability. Secondary efficacy endpoints include ORR, DOR, CRR, PFS, RFS, and OS as well as pharmacokinetics and immunogenicity.

For more information about the LOTIS-7 trial, visit clinicaltrials.gov (NCT04970901).

About ZYNLONTA
ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval and in the European Union under conditional approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Please see full prescribing information including important safety information about ZYNLONTA at www.ZYNLONTA.com.

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.

On May 14, 2025 Circio Holding ASA (OSE: CRNA), a biotechnology company developing powerful circular RNA technology for next generation nucleic acid medicine, today announces the presentation of new and strengthened in vivo data at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) annual meeting 2025 in New Orleans, USA (Press release, Circio, MAY 14, 2025, View Source [SID1234652961]).

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In the poster presentation, Circio is showcasing its latest in vitro and in vivo results for the circVec platform. circVec continues to demonstrate broad capability to significantly boost protein expression level and durability for both viral and non-viral gene and cell therapy.

Importantly, new results show that both viral and non-viral circVec vectors display tissue patterns that are distinct from equivalent mRNA-vectors. The pattern is hallmarked by increased protein expression in muscle, heart and spleen, and consistently low in liver. These surprising observations demonstrate a fundamental biological difference between circRNA and mRNA-based expression and show that the circVec platform has advantages that can open new therapeutic opportunities where current gene therapy approaches fall short.

"Circio is rapidly expanding the in vivo circVec data package, with several recent intriguing advances. It has become evident that circVec not only offers increased protein expression level and durability in general, but also that this effect is associated with a distinct tissue profile. These results provide a valuable roadmap to direct our R&D activities and develop a therapeutic strategy focused on areas that are not well served by existing gene therapy approaches." said Dr. Thomas B. Hansen, CTO of Circio. "We selected the prestigious ASGCT (Free ASGCT Whitepaper) meeting to present our latest results as it provided a great opportunity to showcase our technology to a broad life science audience and potential partners."

In addition, Circio is continuing to progress its circVec-AAV gene therapy development for muscular dystrophies and cardiomyopathies. Longitudinal in vivo data up to six months show a general overall advantage of circVec expression at low dose levels. Enhanced target tissue expression and reduced liver accumulation is also observable with circVec-AAV vectors, indicating that the circVec advantage is more pronounced in specific tissues and therapeutic settings. Follow-up analyses and testing of novel circVec-AAV variants are currently ongoing to further explore these observations and identify the most favorable opportunities for circVec-AAV gene therapy development.

Title of presentation:
CircVec: a powerful circular RNA expression platform to enhance viral and non-viral gene and cell therapies

Time and poster number:
13 May 2025, 18:00-19:30hrs CDT, poster #655

Location:
Ernest N. Morial Convention Center, New Orleans, Louisiana, USA

The poster is attached to this press release and is available on Circio´s webpage

On May 14, 2025 Genmab A/S (Nasdaq: GMAB) reported that it will present 14 abstracts evaluating epcoritamab, a T-cell engaging bispecific antibody administered subcutaneously, as a monotherapy and in combination across disease settings in patients with diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL) at the 30th European Hematology Association (EHA) (Free EHA Whitepaper) Congress, being held in Milan, Italy, and virtually, June 12-15, 2025 (Press release, Genmab, MAY 14, 2025, View Source [SID1234653059]).

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Two oral presentations will feature data from the Phase 1/2 EPCORE NHL-2 trial evaluating epcoritamab plus rituximab and ifosfamide-carboplatin-etoposide (R-ICE) in patients with relapsed/refractory (R/R) DLBCL eligible for autologous stem cell transplantation, and the Phase 1/2 EPCORE NHL-5 trial evaluating epcoritamab plus polatuzumab vedotin, rituximab, cyclophosphamide, doxorubicin, and prednisone (pola-R-CHP) in previously-untreated patients with DLBCL. Additionally, results from the Phase 1/2 EPCORE NHL-1 and NHL-3 trials, including three years of follow-up in patients with R/R DLBCL and FL treated with epcoritamab monotherapy, will be presented as a poster.

All abstracts accepted for presentation have been published and may be accessed online via the EHA (Free EHA Whitepaper) Open Access Library.

"Together with AbbVie, we have made tremendous progress advancing our broad epcoritamab development program and we are pleased to share important results at EHA (Free EHA Whitepaper) 2025 evaluating epcoritamab in a variety of treatment settings and patient populations," said Dr. Judith Klimovsky, Executive Vice President and Chief Development Officer of Genmab. "The data presented at EHA (Free EHA Whitepaper) further reinforce our commitment to epcoritamab and its potential to become a core therapy across B-cell malignancies."

Several abstracts evaluating epcoritamab will also be presented at the 18th International Conference on Malignant Lymphoma (ICML), taking place June 17-21, 2025, in Lugano, Switzerland.

Abstracts accepted for presentation at EHA (Free EHA Whitepaper) include:

Abstract Number Abstract Title Type of Presentation Date/Time of Presentation
S245 First Disclosure of Epcoritamab + R-ICE in Patients with Relapsed/Refractory Diffuse Large B-cell Lymphoma (R/R DLBCL) Eligible for Autologous Stem Cell Transplantation (ASCT): EPCORE NHL-2 Oral Sunday, June 15
11:00-12:15 CEST
S247 Durable Efficacy with Fixed-Duration Epcoritamab + Polatuzumab Vedotin, Rituximab, Cyclophosphamide, Doxorubicin, and Prednisone (Pola-R-CHP) for 1L Diffuse Large B-cell Lymphoma (EPCORE NHL-5) Oral Sunday, June 15
11:00-12:15 CEST
PF881 Epcoritamab Monotherapy Demonstrates Deep and Durable Responses at Three-Year follow-up in Patients with Relapsed/Refractory Follicular Lymphoma Poster Friday, June 13 18:30-19:30 CEST

PF885 Epcoritamab Plus Lenalidomide and Rituximab Achieves High Response Rates and Survival Benefits Compared with Usual Care in Relapsed/Refractory Follicular Lymphoma: A Comparative Analysis Poster Friday, June 13 18:30-19:30 CEST
PF920 Sustained Remission in R/R DLBCL with Epcoritamab Monotherapy: EPCORE NHL-1 3y Results and Novel Subgroup Analyses in Patients with Complete Response at 2y Poster Friday, June 13 18:30-19:30 CEST

PS1886 Matching-Adjusted Indirect Comparison of Epcoritamab with Rituximab + Lenalidomide vs Tafasitamab with Rituximab + Lenalidomide in Second-Line+ Follicular Lymphoma Poster Saturday, June 14 18:30-19:30 CEST

PS1898 Patient-Reported Outcomes in Patients with Relapsed/Refractory Follicular Lymphoma Treated with Epcoritamab within the Entire Study Cohort and in Patients with Symptoms at Baseline Poster Saturday, June 14 18:30-19:30 CEST

PS1968 Match-Adjusted Comparative Analysis of Epcoritamab + R-DHAX/C or R-ICE vs R-DHAX/C or R-ICE In 2L+ Transplant-Eligible Patients with Diffuse Large B-Cell Lymphoma Poster Saturday, June 14 18:30-19:30 CEST

PS1942 Match-Adjusted Comparative Analysis of the Efficacy Of Epcoritamab + R-Mini-CHOP vs R-Mini-CHOP in Previously Untreated Diffuse Large B-Cell Lymphoma Poster Saturday, June 14 18:30-19:30 CEST

PS1932 Treatment Outcomes in Newly Diagnosed Diffuse Large B-Cell Lymphoma Patients with High Cardiovascular Risk, and Treated with Non-Anthracycline Containing Regimens Poster Saturday, June 14 18:30-19:30 CEST

PS1944 Patient Preferences for Attributes of Bispecific Antibodies Indicated for the Treatment of Relapsed/Refractory Diffuse Large B-Cell Lymphoma in the United States Poster Saturday, June 14 18:30-19:30 CEST

PS1912 Circulating Tumour DNA-Directed Intervention with Epcoritamab Alone or in Combination with Lenalidomide and Rituximab is Feasible in the Early Post-CAR-T Population at High Risk of Relapse: Preliminary Data from EpLCART Poster Saturday, June 14 18:30-19:30 CEST

PS1979 Epcoritamab with Gemcitabine, Dexamethasone, and Cisplatin (Epco-GDP) in Relapsed, Refractory Large B-cell Lymphoma – An Interim Analysis of Phase II Multicenter Investigator-initiated Trial Poster Saturday, June 14
15:30-16:00 CEST
PS1892 Phase II Investigator-initiated Trial of Epcoritamab-Lenalidomide in Treatment Naïve Follicular Lymphoma Poster Saturday, June 14
18:30-19:30 CEST
The safety and efficacy of these investigational uses have not been established.

About Epcoritamab
Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology and administered subcutaneously. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.i

Epcoritamab (approved under the brand name EPKINLY in the U.S. and Japan, and TEPKINLY in the EU) has received regulatory approval in certain lymphoma indications in several territories. Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication.

Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes five ongoing Phase 3, open-label, randomized trials including a trial evaluating epcoritamab as a monotherapy in patients with R/R DLBCL compared to investigators choice chemotherapy (NCT04628494), a trial evaluating epcoritamab in combination with R-CHOP in adult patients with newly diagnosed DLBCL (NCT05578976), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) in patients with R/R FL (NCT05409066), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) compared to chemoimmunotherapy in patients with previously untreated FL (NCT06191744), and a trial evaluating epcoritamab in combination with lenalidomide compared to chemoimmunotherapy in patients with R/R DLBCL (NCT06508658). The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit www.clinicaltrials.gov for more information.

On May 14, 2025 Revolution Medicines, Inc. (Nasdaq: RVMD), a late-stage clinical oncology company developing targeted therapies for patients with RAS-addicted cancers, reported the first patient has been dosed in RASolve 301, a global, randomized, open-label Phase 3 clinical trial. RASolve 301 will evaluate the safety and efficacy of daraxonrasib (RMC-6236), a RAS(ON) multi-selective inhibitor, in patients with previously treated, locally advanced or metastatic RAS mutant non-small cell lung cancer (NSCLC) compared to docetaxel chemotherapy (Press release, Revolution Medicines, MAY 14, 2025, View Source [SID1234653079]).

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RASolve 301 is anticipated to enroll approximately 420 patients with NSCLC worldwide who have received one to two prior lines of therapy for the treatment of advanced disease including an anti-PD-1/anti-PD(L)-1 agent and platinum-based chemotherapy. The pivotal clinical trial is designed to include a core population of patients with NSCLC carrying RAS mutations at position G12 (G12X), and an expanded population that also includes patients with tumors carrying other specific RAS mutations. The dual primary endpoints are progression-free survival (PFS) and overall survival (OS) in the core patient population. Key secondary endpoints include PFS, OS and objective response rate (ORR) in the expanded population.

"We are pleased that dosing is underway in the RASolve 301 Phase 3 clinical trial, an important step in developing daraxonrasib, a highly innovative compound that targets a diverse array of RAS mutations that drive tumor growth in 30% of NSCLC cases," said Mark A. Goldsmith M.D., Ph.D., chief executive officer and chairman of Revolution Medicines. "For the vast majority of these RAS cancer drivers, there are currently no approved targeted drugs that can be used in place of chemotherapy. In this trial we are collaborating with physicians globally to evaluate the potential of daraxonrasib as a new therapy for people living with RAS mutant lung cancer."

The company’s decision to evaluate daraxonrasib as a monotherapy in this NSCLC setting was informed by early evidence from a single-arm trial showing the compound had an acceptable safety profile and encouraging antitumor activity. Daraxonrasib is also being evaluated in the RASolute 302 clinical trial, which is a global, randomized Phase 3 trial evaluating daraxonrasib versus standard of care chemotherapy in second-line patients with previously treated metastatic pancreatic ductal adenocarcinoma (PDAC).

About Non-Small Cell Lung Cancer and RAS Mutations
More than 197,000 people are diagnosed with non-small cell lung cancer (NSCLC) in the U.S. each year.1 Despite treatment advancements, NSCLC remains a leading cause of cancer-related mortality worldwide, primarily due to its late-stage diagnosis and limited response to conventional therapies. RAS mutations are among the most common oncogenic drivers in NSCLC, occurring in approximately 30% of cases.2 A significant challenge in treating NSCLC is its genetic diversity, with different mutations including RAS G12X, G13X and Q61X each playing a crucial role in the development and progression of NSCLC in this patient population.

About Daraxonrasib
Daraxonrasib (RMC-6236) is an oral, direct RAS(ON) multi-selective inhibitor with the potential to help address a wide range of cancers driven by oncogenic RAS mutations. Daraxonrasib suppresses RAS signaling by blocking the interaction of RAS(ON) with its downstream effectors. It does so by targeting oncogenic RAS mutations G12X, G13X and Q61X that are common drivers of major cancers including pancreatic ductal adenocarcinoma (PDAC), non-small cell lung cancer (NSCLC) and colorectal cancer (CRC).