On November 13, 2024 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported new and updated data from its hematology pipeline will be shared across 23 abstracts at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2024 Annual Meeting, taking place from December 7-10 in San Diego, CA (Press release, Regeneron, NOV 13, 2024, View Source [SID1234648286]). The latest research advances demonstrate the potential of Regeneron’s diverse pipeline, which aims to address unmet needs in ten types of blood cancers and disorders. These innovative and differentiated approaches include CD3 bispecific antibodies, costimulatory bispecific antibodies, and a pioneering combination of a monoclonal antibody and small interfering RNA (siRNA).

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"Our presentations at ASH (Free ASH Whitepaper) demonstrate the progress we are making toward transforming care for a range of blood cancers and disorders where advancements are desperately needed," said L. Andres Sirulnik, M.D., Ph.D., Senior Vice President and Hematology Clinical Development Unit Head at Regeneron. "These include new head-to-head data exploring a novel combination that aims to maximize disease control in paroxysmal nocturnal hemoglobinuria compared to a standard-of-care treatment, as well as initial results from our confirmatory odronextamab trial in first-line follicular lymphoma. Together, our presentations underscore our commitment to work towards translating scientific breakthroughs to differentiated medicines for hematology patients."

At ASH (Free ASH Whitepaper), abstracts in blood disorders include an oral presentation from an exploratory cohort of a Phase 3 trial investigating a novel combination of pozelimab with cemdisiran compared to ravulizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were naïve to complement inhibition at baseline. Notably, the presentation will detail interim results from patients who were initially randomized to ravulizumab but crossed over to the combination in an open label extension portion of the trial.

Progress on the odronextamab development program will be featured in twelve abstracts. Among them are initial results in patients with previously untreated follicular lymphoma (FL) from Part 1 of the Phase 3 OLYMPIA-1 confirmatory trial, which consists of a non-randomized safety run-in (Part 1) followed by a randomized efficacy portion (Part 2) comparing odronextamab monotherapy to rituximab plus standard-of-care chemotherapies. Two oral presentations on the ELM-1 and pivotal ELM-2 trials will feature new analyses in settings with significant unmet needs: one in patients with diffuse large B-cell lymphoma (DLBCL) who progressed after CAR-T therapy and the other in relapsed/refractory (R/R) marginal zone lymphoma (MZL).

Other notable abstracts include the latest linvoseltamab efficacy and safety results from the pivotal LINKER-MM1 study in R/R multiple myeloma (MM), preclinical data evaluating a CD38xCD28 costimulatory bispecific antibody in combination with linvoseltamab, and preclinical data on TMPRSS6 inhibition in beta-thalassemia.

The full list of Regeneron presentations at ASH (Free ASH Whitepaper) includes:

Abstract Title Abstract Presenter Session Date/Time (PT)
Odronextamab
Efficacy and Safety of Odronextamab Monotherapy in Patients (Pts) with Diffuse Large B-Cell Lymphoma (DLBCL) Progressing after CAR T-Cell Therapy: Primary Analysis from the ELM-1 Study Abstract #866
Oral Presentation
Session

Matthew Matasar Monday, December 9, at 2:45-4:15 pm

Marriott Marquis San Diego Marina, Pacific Ballroom Salons 15-17
Efficacy and Safety of Odronextamab in Relapsed/Refractory Marginal Zone Lymphoma (R/R MZL): Data from the R/R MZL Cohort in the ELM-2 Study Abstract #862
Oral Presentation
Session

Tae Min Kim Monday, December 9, at 2:45-4:15 pm

Marriott Marquis San Diego Marina, Marriott Grand Ballroom 11-13
Odronextamab Monotherapy in Previously Untreated Patients with High-Risk Follicular Lymphoma (FL): Results of the Safety Lead-in of the Phase 3 OLYMPIA-1 Study Abstract #4411
Poster Presentation
Session
Elizabeth Brem

Monday, December 9, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
Efficacy and Safety of Odronextamab in Rare Subtypes of Relapsed/Refractory Aggressive B-Cell Non-Hodgkin Lymphoma (B-NHL): Data from a Dedicated Cohort of Other B-NHLs in the ELM-2 Study Abstract #4502
Poster Presentation
Session Emmanuel Bachy Monday, December 9, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
Treatment Duration and Risk of Fatal Infections in Patients with B-Cell Non-Hodgkin Lymphoma Achieving Complete Response with Odronextamab Abstract #3080
Poster Presentation Gottfried von Keudel

Monday, December 9, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
Dynamics of Complete Responses in Patients with Relapsed or Refractory Diffuse Large B Cell Lymphoma Treated with Odronextamab in the ELM-2 Study Abstract #4486
Poster Presentation
Session
Sabarish Ayyappan

Monday, December 9, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
Long-Term Efficacy and Safety of Odronextamab in Relapsed/Refractory Diffuse Large B-Cell Lymphoma (DLBCL): Pooled Analysis from ELM-1 and ELM-2 Studies Abstract #3118
Poster Presentation
Session
John N. Allan Sunday, December 8, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
Evaluation of CAR-HEMATOTOX Scoring as a Predictor of Infection Risk following Treatment with Odronextamab (a CD20×CD3 Bispecific Antibody) in Relapsed/Refractory Diffuse Large B-Cell Lymphoma Abstract #3076
Poster Presentation
Session
Mathew Matasar Sunday, December 8, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
Evaluation of Baseline CAR-HEMATOTOX Scores to Predict Increased Severe Infection Risk in Patients with Relapsed/Refractory Follicular Lymphoma Treated with Odronextamab Abstract #1650
Poster Presentation
Session
Matthew Matasar Saturday, December 7, at 5:30-7:30 pm

San Diego Convention Center, Halls G-H
Dynamics of Complete Responses in Patients with Relapsed or Refractory Follicular Lymphoma Treated with Odronextamab in the ELM-2 Study Abstract #1628
Poster Presentation
Session
Stefano Luminari Saturday, December 7, at 5:30-7:30 pm

San Diego Convention Center, Halls G-H
Trial in Progress: Odronextamab for the Treatment of Patients with Relapsed/Refractory Mantle Cell Lymphoma following Prior BTK Inhibitor Therapy – A Cohort of the ELM-2 Study Abstract Publication Only Srikanth Ambati N/A
Matching-Adjusted Indirect Comparisons (MAICs) of Odronextamab Versus Mosunetuzumab and Epcoritamab for the Treatment of Patients with Relapsed/Refractory Follicular Lymphoma (R/R FL) after Two or More Lines of Systemic Therapy Abstract Publication Only

Deepa Jagadeesh N/A
Linvoseltamab
Linvoseltamab in Patients with Relapsed/Refractory Multiple Myeloma: Longer Follow-Up and Selected High-Risk Subgroup Analyses of the LINKER-MM1 Study Abstract #3369
Poster Presentation Mansi R. Shah Sunday, December 8, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
Soluble BCMA Dynamics in Patients with Relapsed/Refractory Multiple Myeloma (RRMM) Treated with Linvoseltamab in LINKER-MM1 Abstract #3310
Poster Presentation Anasuya Hazra Sunday, December 8, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
Characterization of Linvoseltamab’s BCMA Binding Epitope and Efficacy Against BCMA Mutations in Relapsed/Refractory Multiple Myeloma Abstract #3265
Poster Presentation Ken Lee & Yi Zhou Sunday, December 8, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
A CD38xCD28 Costimulatory Bispecific Antibody Demonstrates Potent Preclinical Combinatorial Activity with a BCMAxCD3 T Cell-Engager Abstract #3283
Poster Presentation Kara Olson & David J. DiLillo Sunday, December 8, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
Reducing Time Toxicity for Anti-B-Cell Maturation Antigen (BCMA) Bispecific Treatment: Evidence from Pivotal Single-Arm Trial Data on Teclistamab, Elranatamab, and Linvoseltamab for Triple-Class Exposed (TCE) Relapsed/Refractory Multiple Myeloma (RRMM)
Abstract #2271
Poster Presentation Session
Sikander Ailawadh Saturday, December 7, at 5:30-7:30 pm

San Diego Convention, Halls G-H
Exposure-Response Analyses of Various Efficacy and Safety Endpoints in Support of Registrational Dose Selection of Linvoseltamab in Patients with Relapsed/Refractory Multiple Myeloma Abstract Publication Only Oleg Milberg N/A
Comparative Effectiveness of Linvoseltamab Versus Current Real-World Standard-of-Care Therapies in Triple-Class Exposed Relapsed/Refractory Multiple Myeloma Treated at IMWG Sites Abstract Publication Only

Shaji Kumar N/A
Comparative Effectiveness of Linvoseltamab Versus Current Real-World (RW) Standard-of-Care (SOC) Therapies in Triple-Class Exposed Relapsed/Refractory Multiple Myeloma (RRMM): Key Subgroups Analysis Abstract Publication Only

Shaji Kumar N/A
Additional Presentations
Efficacy and Safety of Pozelimab Plus Cemdisiran vs Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Naïve to Complement Inhibition* Abstract #306
Oral Presentation Session Christopher Patriquin Saturday, December 7, at 4 – 5:30 pm

San Diego Convention Center, Room 11
TMPRSS6 Inhibition Rapidly Reverses Liver Iron Overload and Prevents an Increase of Splenic Pro-Inflammatory Macrophages in a Mouse Model of Beta-Thalassemia Abstract #2473
Poster Presentation Session

Heinrich E. Lob Sunday, December 8, at 6:00-8:00 pm

San Diego Convention Center, Halls G-H
The Reality of Beta Thalassemia and Iron Chelation Therapy – A Qualitative Study Unveiling the Patient Burden Abstract Publication Only

Chris Hartford
*Agreement with Alnylam Pharmaceuticals, Inc.

Linvoseltamab as well as the combination of pozelimab and cemdisiran are investigational, and the potential uses of odronextamab in R/R MZL and rare subtypes of R/R aggressive B-cell non-Hodgkin lymphoma are also investigational and have not been approved by any regulatory authority. Odronextamab is approved in the European Union as Ordspono to treat R/R FL or DLBCL after two or more lines of systemic therapy, but the safety and efficacy of odronextamab have not been fully evaluated by any other regulatory authority.

About Regeneron in Hematology
At Regeneron, we’re applying more than three decades of biology expertise with our proprietary VelociSuite technologies to develop medicines for patients with diverse blood cancers and rare blood disorders.

Our blood cancer research is focused on bispecific antibodies that are being investigated both as monotherapies and in various combinations and emerging therapeutic modalities. Together, they provide us with unique combinatorial flexibility to develop customized and potentially synergistic cancer treatments.

Our research and collaborations to develop potential treatments for rare blood disorders include explorations in antibody medicine, gene editing and gene-knockout technologies, and investigational RNA-approaches focused on depleting abnormal proteins or blocking disease-causing cellular signaling.

About Regeneron’s VelocImmune Technology
Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron’s Co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985.They were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes REGEN-COV (casirivimab and imdevimab), Dupixent (dupilumab), Libtayo, Praluent (alirocumab), Kevzara (sarilumab), Evkeeza (evinacumab-dgnb), Inmazeb (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz (pozelimab-bbfg).

On November 13, 2024 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases, reported an update on the company’s development pipeline and announced financial results for the third quarter ended September 30, 2024 (Press release, Quince Therapeutics, NOV 13, 2024, View Source [SID1234648285]).

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Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, said, "We are pleased to report accelerating enrollment of our pivotal Phase 3 NEAT clinical trial in Ataxia-Telangiectasia (A-T). As of today, we have enrolled 32 patients with A-T across clinical sites in the U.S., U.K., and European Union. Additionally, the majority of planned NEAT study sites are now activated and open for enrollment. We expect enrollment momentum to continue as we work toward our commitment to complete enrollment in the second quarter of 2025 and report topline results in the fourth quarter of 2025."

Pivotal Phase 3 NEAT Clinical Trial

Enrolled 32 participants to date in the company’s Phase 3 NEAT (Neurologic Effects of EryDex on Subjects with A-T; IEDAT-04-2022/NCT06193200) clinical trial to evaluate the neurological effects of EryDex in patients with A-T.
Quince plans to enroll approximately 86 patients with A-T ages six to nine years old (primary analysis population) and approximately 20 patients with A-T ages 10 years or older.
Participants who complete the full treatment period, complete study assessments, and provide informed consent will be eligible to transition to an open label extension study, which will begin in the fourth quarter of 2024.
Pivotal Phase 3 NEAT clinical trial is being conducted under a Special Protocol Assessment agreement with the U.S. Food and Drug Administration (FDA).
Expect to report Phase 3 NEAT topline results in the fourth quarter of 2025 with a New Drug Application (NDA) submission to the FDA and a Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in 2026, assuming positive study results.
NEAT is an international, multi-center, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of the company’s lead asset, EryDex (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells), in patients with A-T.
Participants will be randomized (1:1) between EryDex or placebo and treatment will consist of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last visit completion in a rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo as per the SPA agreement with the FDA.
Pipeline and Corporate Updates

Participation at scientific congresses, including a poster presentation of safety data from the previously completed ATTeST study at the 53rd Child Neurology Society Annual Meeting. Quince is also sponsoring and participating at the 2024 International Congress for Ataxia Research in November 2024 with two poster presentations, including growth and bone mineral density in patients with A-T treated with EryDex, and an analysis of the International Cooperative Ataxia Rating Scale (ICARS) subcomponent scores in patients with A-T.
Generating Phase 2 clinical trial study designed to evaluate EryDex for the potential treatment of patients with Duchenne muscular dystrophy (DMD), including those with corticosteroid intolerance, who represent the majority of the DMD population. Quince plans to initiate a DMD Phase 2 study in 2025, which the company expects to conduct utilizing capital efficient study approaches.
Completed evaluation process of other potential rare disease indications beyond A-T and Duchenne muscular dystrophy for EryDex where chronic corticosteroid treatment is – or has the potential to become – a standard of care, if there were not corticosteroid-related safety concerns. The prioritized list of other potential rare disease targets under consideration includes: 1) autoimmune hepatitis, 2) dermatomyositis, 3) pemphigus vulgaris, 4) Hashimoto’s encephalopathy, 5) Becker muscular dystrophy, 6) pediatric lupus, 7) juvenile idiopathic arthritis, 8) myasthenia gravis, 9) limb-girdle muscular dystrophy, 10) chronic inflammatory demyelinating polyradiculoneuropathy, and 11) pulmonary sarcoidosis.
Third Quarter and Year-to-Date 2024 Financial Results

Reported cash, cash equivalents, and short-term investments of $47.8 million for the third quarter ended September 30, 2024. Quince expects its existing cash runway to be sufficient to fund the company’s capital efficient development plan through Phase 3 NEAT topline results and into 2026.
Expect strong cash position to fully fund lead asset, EryDex, through Phase 3 NEAT topline results in the fourth quarter of 2025 and prepare for NDA and MAA submissions in 2026, assuming positive study results. This includes approximately $20 million for the NEAT clinical trial and approximately $15 million in direct trial costs for an open label extension study.
Reported research and development (R&D) expenses of $4.9 million for the third quarter ended September 30, 2024. R&D expenses during the quarter primarily included costs related to ongoing Phase 3 NEAT clinical trial activities and related manufacturing costs.
Reported general and administrative (G&A) expenses of $3.6 million for the third quarter ended September 30, 2024. G&A expenses for the quarter primarily included personnel-related and stock-based compensation expenses, commercial planning and new product planning expenses, and other professional administrative costs.
Reported a net loss of $5.5 million, or a net loss of $0.13 per basic and diluted share, for the third quarter ended September 30, 2024. Weighted average shares outstanding for the quarter were 43.2 million.
Reported net cash used in operating activities of $24.4 million for the nine months ended September 30, 2024, which included a net loss of $44.4 million for the period, adjusted for $22.4 million of non-cash items, including $17.1 million goodwill impairment charge, $2.1 million change in the fair value of contingent consideration liabilities, $3.6 million in stock-based compensation, a net increase in operating assets of $2.5 million, and a net increase in accounts payable, accrued expenses, and other current liabilities of $0.1 million. Additionally, Quince made a cash milestone payment of $5 million to EryDel shareholders in the third quarter of 2024 following the achievement of the first patient enrolled in the NEAT study in the second quarter of 2024.

On November 13, 2024 Pulmatrix ("Pulmatrix") (Nasdaq: PULM), a clinical-stage biopharmaceutical company, reported a merger agreement with Cullgen Inc. ("Cullgen"), a privately-held, clinical-stage biopharmaceutical company applying its proprietary targeted protein degradation uSMITE platform to discover and advance therapeutics for the treatment of cancer and other diseases (Press release, Pulmatrix, NOV 13, 2024, View Source [SID1234648284]). Cullgen utilizes its proprietary technology platform, uSMITE, featuring novel E3 ligands, to build the next generation of targeted protein degraders and degrader-antibody conjugates ("DACs").

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In 2023 Cullgen completed a Series C financing led by AstraZeneca-CICC Venture Capital Partnership, and also announced a strategic partnership with Astellas Pharma Inc.

About the Proposed Transaction

Subject to the terms and conditions of the merger agreement, and upon the closing of the merger, pre-merger Pulmatrix stockholders are expected to own approximately 3.6% of the combined company, and pre-merger Cullgen stockholders are expected to own approximately 96.4% of the combined company which will operate under the name Cullgen Inc., be headquartered in San Diego, CA and trade on The Nasdaq Capital Market (Nasdaq). Pulmatrix stockholders will also receive a special cash dividend to the extent that Pulmatrix’s net cash at closing exceeds $2.5 million, subject to certain adjustments.

The transaction is expected to close by the end of March 2025, subject to obtaining stockholder and CSRC approval.

Management and Board of Directors

At the effective time of the merger, the executive officers of the combined company will be led by Ying Luo, Ph.D. Cullgen’s Chairman and CEO. The Cullgen Board of Directors will be supplemented by one representative of Pulmatrix.

Ying Luo, Ph.D., commented, "I’m delighted to announce this planned merger with Pulmatrix, which comes at a pivotal moment in the evolution of our company as we advance our pipeline of targeted protein degraders into clinical development for cancer and other diseases. Listing on Nasdaq will help fuel our growth and enable us to unlock the full potential of our technology platform, including our plans to develop and advance degrader-antibody conjugates and additional targeted protein degraders into the clinic."

Peter Ludlum, Interim Chief Executive Officer of Pulmatrix, added, "Following a thorough evaluation of strategic alternatives, the Pulmatrix board of directors and management team believe that this anticipated transaction represents an opportunity to deliver value to our stockholders. We anticipate that this merger will allow our stockholders to participate in Cullgen’s promising research and development activities and also provide a benefit in the form of a potential dividend component immediately prior to the close of the merger. Prior to transaction closing, we will attempt to increase cash available for the special dividend by divesting corporate assets including PUR1800, PUR3100, and the patent portfolio for iSPERSE."

Cullgen Pipeline Overview and Development Milestones

Cullgen currently has three degrader programs that are in or about to initiate Phase 1 clinical testing. CG001419 is a first-in-class, selective, clinically active, oral pan-TRK degrader that is being studied in two separate clinical trials—one for solid tumors, and the other for the treatment of acute and chronic pain. With respect to the cancer trial, Cullgen has dosed ten patients thus far with no observed dose-limiting toxicity, treatment-related serious adverse events or grade ≥ 3 treatment related adverse events. Regarding the pain trial, Cullgen recently received HREC (Australia) approval to begin enrolling patients to evaluate the safety and pharmacokinetic characteristics of CG001419 in healthy volunteers. Cullgen anticipates enrolling the first patient in early 2025. This program aims to provide a new non-opioid non-NSAID analgesic which can fulfill unmet medical needs in the field of pain.

Cullgen is also evaluating CG009301, a GSPT1 degrader for the treatment of blood cancers including relapsed/refractory acute myeloid leukemia (AML), higher-risk myelodysplastic syndrome (HR-MDS), and acute lymphoblastic leukemia (ALL). Cullgen has received IND allowance from the China CDE for this product candidate and Cullgen anticipates dosing the first patient in the first quarter of 2025. CG009301 also has the potential to be used for the treatment of solid tumors harboring MYC amplification.

In addition to these three clinical programs, Cullgen is also advancing several other targeted protein degraders and DACs, predominantly for the treatment of cancer and autoimmune disease, including a program targeting a cell cycle protein that has been partnered with Astellas Pharma Inc.

Webcast Information

Management will host an informational webcast that will be made available to access at 12:00 pm ET today. A link to the webcast can be found in the Investor Events and Presentations section of the Pulmatrix website at and in the News & Events section of Cullgen’s website at .

On November 13, 2024 Mural Oncology plc (Nasdaq: MURA), a clinical-stage immuno-oncology company developing novel, investigational engineered cytokine therapies designed to address areas of unmet need for patients with a variety of cancers, reported financial results for the third quarter of 2024 and provided an update on pipeline progress (Press release, Mural Oncology, NOV 13, 2024, View Source [SID1234648283]).

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"Mural launched as a stand-alone company one year ago with a mission to shepherd in the second wave of immuno-oncology for patients—IO 2.0—and we believe we are well on the path to realize that vision. With our deep expertise of protein engineering and cancer biology, we are working to address key limitations with cytokine therapies and unleash their full potential. We are now focused on clinical execution, with major readouts of our two potentially registrational studies of nemvaleukin in the first half of next year, and commercial readiness. We are also deepening our pipeline with candidate nominations for our IL-18 and IL-12 programs expected by the end of this year," said Caroline Loew, Ph.D., CEO of Mural Oncology.

Clinical Progress & Upcoming Catalysts:

Mural’s late-stage trials of nemvaleukin alfa (nemvaleukin), an engineered fusion protein designed to leverage Interleukin-2’s (IL-2) antitumor effects while mitigating its hallmark toxicities, remain on track. The company shared new information related to study design, statistical assumptions, and study execution at a virtual Investor Day in September 2024, including:

Completion of enrollment in ARTISTRY-7, the company’s potentially registrational phase 3 trial in platinum-resistant ovarian cancer (PROC), with a total of 456 patients enrolled. The study is comparing the combination of nemvaleukin and pembrolizumab versus investigator’s choice single agent chemotherapy. Mural expects to report interim overall survival (OS) results in late Q1 or early Q2 based on an analysis performed at approximately 75% of OS events. If the hazard ratio meets the bar for success (0.727, or a 27.3% reduction in the risk of death assuming exactly 215 OS events), the company plans to file a Biologics License Application (BLA) in 2025 subject to discussions with the U.S. Food and Drug Administration (FDA). The company expects to report final results in the second quarter of 2026.
Completion of enrollment in ARTISTRY-6, Cohort 2, the company’s potentially registrational phase 2 study of single agent nemvaleukin in patients with unresectable or metastatic mucosal melanoma, with 92 patients enrolled. Mural anticipates reporting top-line results from cohort 2 of ARTISTRY-6 in Q2 of 2025. The target response rate is 25%. Mural believes that in this rare and highly aggressive tumor with poor outcomes even in the first line setting, demonstrating durable responses with a response rate of 20-25% would be meaningful for patients, and would support a discussion with the FDA regarding a BLA submission and potential accelerated approval.
For the full updates from Mural’s Investor Day, please visit the webcast on our Events & Presentations page.

Mural is also evaluating a less-frequent intravenous (LFIV) dose of nemvaleukin in patients with cutaneous melanoma in ARTISTRY-6, Cohort 3 (monotherapy) and Cohort 4 (combination with pembrolizumab). The company continues to expect preliminary data readouts in the monotherapy cohort in the first half of 2025, and in the combination cohort in the second half of 2025.

Preclinical Program Updates:

Mural plans to nominate development candidates for its IL-18 and IL-12 programs by the end of 2024. The company expects to submit an Investigational New Drug (IND) Application for its IL-18 program to the FDA in Q4 2025.

Other Recent Corporate Highlights:

In September, Mural announced the appointment of Sachiyo Minegishi to its board of directors and chair of the Audit Committee. Ms. Minegishi brings over two decades of biopharma experience, with a focus on corporate strategy, finance, development, and commercialization. She is currently the Chief Operating Officer at Rectify Pharmaceuticals, driving corporate and financing strategy to advance its lead program from discovery to clinical stage. Prior to Rectify, she was Chief Financial Officer at Akouos, Inc., where she led corporate finance and business development strategy and played a key role in the acquisition of the company by Eli Lilly.

In October, Mural continued to prepare for potential launch readiness by creating a new commercial division in the company. Brandon Kotaniemi, SVP of Commercial, will drive Mural’s commercial strategy as the company prepares for the potential BLA submission and launch of nemvaleukin. He will also partner closely with Mural’s development team as the company looks to move its IL-18 program into the clinic.

In November, Mural presented clinical and preclinical data at the 39th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper). The three poster presentations included tumor microenvironment pharmacodynamic data from the phase 1/2 ARTISTRY-3 study of nemvaleukin as well as data from Mural’s two preclinical research programs in IL-18 and IL-12.

Financial Results for the Quarter Ended September 30, 2024:

Cash Position: As of September 30, 2024, cash, cash equivalents, and marketable securities were $175.5 million.

R&D Expenses: Research and development expenses were $27.6 million for the third quarter of 2024 compared to $40.4 million for the third quarter of 2023. This decrease in R&D expenses was primarily due to different team composition compared to the personnel previously allocated to us by Alkermes plc (Alkermes), our former parent prior to the separation, as well as decreased spend on the ARTISTRY-1 and ARTISTRY-2 trials as activities related to these trials wound down in 2023, and decreased spend on the ARTISTRY-7 trial due to the timing of patient enrollment.

G&A Expenses: General and administrative expenses were $6.5 million for the third quarter of 2024 compared to $6.0 million for the third quarter of 2023. This increase in G&A expenses was primarily due to costs associated with operating as a standalone company after the separation. This includes professional fees as well as differences in costs of insurance and taxes compared to amounts previously allocated to us by Alkermes prior to the separation.

Net Loss: Net loss was $31.8 million for the third quarter of 2024 compared to $51.3 million for the third quarter of 2023.

Financial Guidance:
The company reaffirms guidance that its cash, cash equivalents, and marketable securities as of September 30, 2024 are expected to fund its operations into the fourth quarter of 2025.

As noted previously, management forecasts lower operating expenses in 2025 versus 2024 due to the timing of clinical trial expenses.

On November 13, 2024 Mersana Therapeutics, Inc. (NASDAQ: MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported a business update and announced financial results for the third quarter ended September 30, 2024 (Press release, Mersana Therapeutics, NOV 13, 2024, View Source [SID1234648282]).

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"During the third quarter of 2024, our team continued its strong execution as we advanced the dose escalation portions of our Phase 1 clinical trials of XMT-1660 and XMT-2056, made further progress in our collaborations and maintained the strength of our balance sheet," said Martin Huber, M.D., President and Chief Executive Officer of Mersana Therapeutics. "In multiple presentations over the course of the past year, we shared data demonstrating the potential for Dolasynthen ADCs to generate anti-tumor activity and avoid many of the toxicities that have limited other ADC platforms. We are looking forward to presenting initial XMT-1660 clinical data later this year. We also are finalizing plans to begin the expansion portion of our trial, with an initial focus on patients with triple-negative breast cancer who have previously been treated with at least one topoisomerase-1 ADC."

Recent Accomplishments, Strategic Priorities and Expected Milestones

XMT-1660: Mersana continues to advance its Phase 1 clinical trial of XMT-1660, the company’s lead Dolasynthen ADC candidate targeting B7-H4. The dose escalation portion of the trial is ongoing, with the company having recently escalated to a dose of 115 milligrams per meter squared administered every four weeks. A maximum tolerated dose has not yet been established. Approximately 75 percent of the patients that have been enrolled in the trial to date have triple-negative breast cancer (TNBC) or hormone receptor-positive breast cancer (HR+BC). Among the enrolled patients with TNBC, approximately 90 percent have received a prior topoisomerase-1 (topo-1) ADC, and among the enrolled patients with HR+BC, approximately half have received a prior topo-1 ADC. Last week at World ADC 2024, Mersana presented new preclinical data demonstrating XMT-1660’s anti-tumor activity following topo-1 treatment. By the end of 2024, Mersana plans both to share initial safety, tolerability, efficacy and biomarker data from its Phase 1 dose escalation and backfill cohorts at a company event and to initiate the expansion portion of the trial in patients with TNBC who have previously been treated with at least one topo-1 ADC.

XMT-2056: Mersana continues to escalate dosing in its Phase 1 clinical trial of XMT-2056, the company’s lead Immunosynthen ADC candidate targeting a novel HER2 epitope. GSK plc has an exclusive global license option to co-develop and commercialize XMT-2056. At the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2024 Annual Meeting last week, Mersana presented new preclinical data demonstrating XMT-2056’s ability to activate STING signaling and inhibit tumor growth at very low doses.

Collaborations: Mersana continues to advance its Johnson & Johnson and Merck KGaA, Darmstadt, Germany collaborations. The collaboration with Merck KGaA, Darmstadt, Germany focuses on discovering novel Immunosynthen ADCs for up to two targets. The collaboration with Johnson & Johnson focuses on discovering novel Dolasynthen ADCs for up to three targets. In the third quarter of 2024, Mersana achieved and received payment for an $8 million development milestone under the Johnson & Johnson collaboration and achieved a $1 million development milestone under the Merck KGaA, Darmstadt, Germany collaboration, for which payment was received in the fourth quarter of 2024.

Third Quarter 2024 Financial Results

Cash, cash equivalents and marketable securities as of September 30, 2024, were $155.2 million. Mersana continues to expect that its capital resources will be sufficient to support its current operating plan commitments into 2026.
Net cash used in operating activities for the third quarter of 2024 was $8.6 million, which reflects the impact of the aforementioned $8 million milestone payment and a $3.5 million payment for manufacturing activities from Johnson & Johnson.
Collaboration revenue for the third quarter of 2024 was $12.6 million, compared to $7.7 million for the same period in 2023. The year-over-year change was primarily related to an increase in revenue recognized under Mersana’s collaboration and license agreements with Johnson & Johnson and Merck KGaA, Darmstadt, Germany.
Research and development (R&D) expenses for the third quarter of 2024 were $14.8 million, compared to $30.5 million for the same period in 2023. Included in the third quarter of 2024 R&D expenses were $2.3 million in non-cash stock-based compensation expenses. The year-over-year decline in R&D expenses was primarily related to reduced costs associated with manufacturing and clinical development activities for UpRi, a discontinued ADC candidate, and reduced employee compensation expense following the company’s restructuring in 2023.
General and administrative (G&A) expenses for the third quarter of 2024 were $9.9 million, compared to $12.9 million during the same period in 2023. Included in the third quarter of 2024 G&A expenses were $1.7 million in non-cash stock-based compensation expenses. The year-over-year decline in G&A expenses was primarily related to reduced consulting and professional services fees and reduced employee compensation expense following the aforementioned restructuring.
Net loss for the third quarter of 2024 was $11.5 million, or $0.09 per share, compared to a net loss of $41.7 million, or $0.35 per share, for the same period in 2023.
Conference Call Reminder
Mersana will host a conference call today at 8:00 a.m. ET to discuss business updates and its financial results for the third quarter of 2024. To access the call, please dial 833-255-2826 (domestic) or 412-317-0689 (international). A live webcast of the presentation will be available on the Investors & Media section of the Mersana website at www.mersana.com, and a replay of the webcast will be available in the same location following the conference call for approximately 90 days.