On May 2, 2025 HAYA Therapeutics, SA, a biotechnology company pioneering precision RNA-guided regulatory genome targeting therapeutics that reprogram disease-driving cell states for rare, common, chronic and age-related diseases, reported the presentation of data at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in New Orleans from May 13-17, 2025 (Press release, Haya Therapeutics, MAY 2, 2025, View Source [SID1234652482]).

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The company will showcase its latest advances in an invited presentation focused on HAYA’s lead program, HTX-001, an antisense oligonucleotide designed to target the long non-coding RNA (lncRNA) Wisper, the master regulator of fibrosis in the heart, currently in Investigational New Drug-enabling studies and in development for the treatment of non-obstructive hypertrophic cardiomyopathy (nHCM). Additionally, HAYA will have a poster presentation on its innovative multimodal omics approach for profiling and designing antisense oligonucleotide-based therapeutics that enables a precise and potent silencing of novel lncRNA targets.

"Unlike traditional approaches that block single genes or proteins, we have built a platform to reprogram the fundamental biology that underlies disease," said Samir Ounzain, Ph.D., Co-founder and CEO of HAYA Therapeutics. "We are identifying and drugging novel regulatory genome-derived drivers of disease pathogenesis to reprogram sick cells into healthy ones."

"Over the past years, we’ve made significant progress on our lead program, HTX-001, targeting myocardial fibrosis and we anticipate initiating clinical trials in the near future. This forward momentum brings us closer to our ultimate mission: bringing disease-modifying medicines to patients," said Daniel Blessing, Ph.D., Co-founder and CTO of HAYA Therapeutics.

Poster Presentation:

Title: Harnessing Functional Genomics to Advance the Discovery and Development of Oligonucleotide-Based Therapies Targeting the Regulatory Genome
Presenter: Rudi Micheletti, HAYA’s Senior Director of Biology and Functional Genomics
Presentation Date/Time: Tuesday, May 13, 2025, 6:00 PM – 7:30 PM CDT
Location: Poster Hall I2
Abstract Number: 660

Scientific Symposium:

Title: A Wisper from the Heart: Targeting a Cardiac Myofibroblasts-specific LncRNA to Treat Myocardial Fibrosis
Presenter: Daniel Blessing, HAYA’s CTO and Co-founder
Session Title: Targeting Myocardium: To The Heart Of The Matter (Organized by the Cardiovascular CGT Committee)
Presentation Date/Time: Wednesday, May 14, 2025, 3:45 PM – 5:30 PM CDT
Location: Room 388-390

HAYA’s RNA-guided therapeutic platform represents a new approach to treating disease by targeting the root causes at the cellular level. At its core is HAYA’s Regulatory Genome Atlas, which maps the connection between noncoding regulatory genome elements, or lncRNAs, and disease-driving cell states by integrating multimodal functional genomics with a stack of proprietary computational tools and machine learning methodologies. This enables the development of genetic medicines that reprogram diseased cells back to healthy ones.

On May 2, 2025 Genmab A/S (Nasdaq: GMAB) reported its intention to submit in the first half of 2025 a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for subcutaneous epcoritamab, a bispecific antibody being investigated in combination with rituximab and lenalidomide (R2) for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL), following at least one prior systemic therapy (Press release, Genmab, MAY 2, 2025, View Source [SID1234652444]).

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The decision to submit the sBLA is supported by positive topline results from the Phase 3 EPCORE FL-1 trial evaluating epcoritamab plus R2 versus R2 alone in adult patients with R/R FL. Based on an interim analysis conducted by an Independent Data Monitoring Committee (IDMC) review, the study met one of its dual primary endpoints of ORR (Complete Response plus Partial Response, p-value < 0.0001). The safety profile of epcoritamab plus R2 in adult patients with R/R FL was consistent with the known safety profiles of the individual regimens (epcoritamab and R2) and as presented in the U.S. prescribing information for epcoritamab. No new safety signals were observed. The full results will be submitted later this year for presentation at an upcoming medical congress and discussed with global regulatory authorities.

"We are pleased with the strength of the data that allows us to submit a supplemental Biologics License Application in accordance with the U.S. FDA’s Project Frontrunner, which supports our commitment to advance novel medicines to patients who need them. The interim topline results demonstrate the potential of this investigational epcoritamab combination regimen to treat relapsed or refractory follicular lymphoma patients," said Jan van de Winkel, Ph.D., Chief Executive Officer, Genmab. "This milestone represents our commitment to the ongoing development of epcoritamab, with our partner AbbVie, and we look forward to seeing the full results from the study."

Use of epcoritamab plus R2 in R/R FL is not approved in the U.S., in the EU or in any other territory. The safety and efficacy of epcoritamab for use as a combination therapy in FL have not been established. Epcoritamab is currently approved by the FDA under Accelerated Approval as a monotherapy for the treatment of adults with R/R FL after two or more lines of systemic therapy.

About Follicular Lymphoma (FL)
FL is typically an indolent (or slow-growing) form of non-Hodgkin’s lymphoma (NHL) that arises from B-lymphocytes and is the second most common form of NHL accounting for 20-30 percent of all cases.i About 15,000 people develop FL each year in the U.S.ii and it is considered incurable with current standard of care therapies.iii Patients often relapse and, with each relapse the remission and time to next treatment is shorter.iv Over time, transformation to diffuse large B-cell lymphoma (DLBCL), an aggressive form of NHL associated with poor survival outcomes, can occur in more than 25 percent of FL patients.v

About the EPCORE FL-1 Trial
EPCORE FL-1 (NCT05409066) is a Phase 3 open-label interventional trial to evaluate the safety and efficacy of epcoritamab plus rituximab and lenalidomide (R2) versus R2 alone in patients with relapsed/refractory (R/R) follicular lymphoma (FL). The dual primary endpoints are ORR and progression-free survival assessed by independent review committee (IRC) per Lugano criteria.

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.vi

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 chemotherapy infusion 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 2, 2025 Zonhon Biopharma Institute reported the Investigational New Drug (IND) application for ZHB114 for Injection—a Class I innovative biologic drug independently developed by Zonhonbio with proprietary intellectual property rights—was officially approved by the U.S. Food and Drug Administration (FDA) (Press release, Zonhon Biopharma Institute, MAY 2, 2025, View Source [SID1234656085]). The newly approved indication covers advanced malignant tumors.

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On May 2, 2025 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, reported that its management team is scheduled to present at the BofA Securities 2025 Health Care Conference on Wednesday, May 14, 2025, at 8:00 am PT/11:00 am ET (Press release, Agios Pharmaceuticals, MAY 2, 2025, View Source [SID1234652475]).

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The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be archived on the company’s website for at least two weeks following the presentation.

On May 2, 2025 Eli Lilly and Company (NYSE:LLY) reported that it will participate in the Bank of America Securities 2025 Healthcare Conference, May 15, 2025. Lucas Montarce, Lilly executive vice president and chief financial officer, will take part in a fireside chat at 11:40 a.m., Eastern time (Press release, Eli Lilly, MAY 2, 2025, View Source [SID1234652476]).

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.