On 28 April, Fosun Pharma ("the Company", stock code: 600196.SH; 02196.HK) reported its results for the first quarter of 2026 (the reporting period). During the reporting period, the Company achieved a total revenue of RMB 10,073 million, representing a year-on-year increase of 6.93%. Regarding R&D investment, the Company firmly implemented its innovation transformation strategy. During the reporting period, the R&D expenditure increased to RMB 897 million, increased by 1.59% YoY. While increasing the R&D intensity, the Company maintained a positive growth trend in profit. Net profit attributable to shareholders of the list company after deducting extraordinary gain or loss was RMB 501 million, up 21.96% year-on-year. Net profit attributable to shareholders of the listed company was RMB 871 million, increased by 13.87% YoY. Net cash flows generated from operating activities was RMB 1,149 million, up 8.8% YoY.

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In the first quarter of 2026, guided by the strategy of "Innovation Driven, Deep Globalization, and AI Embracement", Fosun Pharma firmly continued to advance its innovation transformation, promoted the deep synergy between innovation and R&D and global operations, and further enhanced the foundation for high-quality development. Driven by the continuous increase in R&D investment, the Company’s transformation of innovative achievements continues to materialize. During the reporting period, the NDA for 4 Innovative Drugs were accepted by the NMPA or the U.S. FDA, and 14 clinical trials for Innovative Drugs (calculated by approval) were approved by domestic and overseas regulatory authorities, covering core therapeutic areas such as oncology. In the Pharma R&D Annual Review 2026 white paper released by the world-known consulting firm Citeline, Fosun Pharma ranked among the global top 20, gained international professional recognition for its innovative R&D strength and pipeline quality.

Deepening Core Technology Platforms to Consolidate Advantages in Oncology and Other Fields

Fosun Pharma has established an open-ended R&D system that combines in-house R&D, co-development, licensing, fund incubation, and industrial investment, also focused on strengthening core technology platforms such as antibodies/ADC, small molecules, and cell therapy, continuously consolidating its innovation moat in core therapeutic areas including oncology.

In the field of antibody/ADC technology platform, Shanghai Henlius, a subsidiary of Fosun Pharma, has steadily advanced the approvals and clinical progress of several self-developed biological drugs. Notably, denosumab injection (HLX14) secured approval in Canada in March 2026, covering all indications of the reference product approved locally, achieving a global commercialization breakthrough in areas such as osteoporosis, bone metastasis from tumors, and giant cell tumor of bone. The NDA for bevacizumab injection (HLX04) was accepted in the U.S., marking a new milestone in the Company’s international registration capabilities for biosimilars. HLX43, an antibody-drug conjugate targeting PD-L1, is expected to create synergy with HLX07 or serplulimab injection for the treatment of advanced colorectal cancer and other solid tumors. HLX3901, a tetraspecific antibody of DLL3×DLL3×CD3×CD28, received approval for clinical trial, demonstrating significant potential in refractory solid tumors through multi-target synergistic activation of immune cells.

In the field of small molecule innovative drug platform, in January 2026, the NDA for foritinib succinate capsules (SAF-189), Fosun Pharma’s Class 1 innovative drug, was accepted by the NMPA, intended for the treatment of patients with anaplastic lymphoma kinase (ALK)-positive locally advanced or metastatic non-small cell lung cancer (NSCLC), expected to provide a new option for precision treatment of lung cancer. In February 2026, the NDA for the self-developed MEK 1/2 inhibitor luvometinib tablets (Trade name in Chinese mainland: Fu Mai Ning) for adult patients with neurofibromas type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas (PN) have been accepted and granted in the priority review by the NMPA. This marks a critical step toward full-age coverage following its approval for pediatric and adolescent NF1 patients in May 2025, also brings new hope to a large number of adult NF1 patients in China who lack effective drug treatments. Additionally, the NDA for methoxyetomidate hydrochloride injection (ET-26), a Class 1 innovative drug self-developed by the subsidiary Avanc Pharma for anesthesia induction and short-term surgical anesthesia, was also accepted.

In the field of prospective layout of early-stage pipeline, several new molecules have been approved for the clinical trials: FXB0871, a PD-1-targeted IL-2 fusion protein, was approved for clinical trials in locally advanced or metastatic solid tumors. Based on the ATTENUKINE platform, FXB0871 is expected to achieve high efficacy and low toxicity. FXS0683, a next-generation Bcl-2 inhibitor, was approved for Phase I clinical trials in hematological malignancies. Innovative molecule including HLX97 (a KAT6A/B small molecule inhibitor) and HLX3901 (tetra specific antibody of DLL3×DLL3×CD3×CD28) were approved for clinical trials, widely covering refractory solid tumors such as breast cancer and small cell lung cancer. Meanwhile, HLX22 (anti-HER2 monoclonal antibody) entered phase II clinical trials for the first-line treatment of HER2-positive recurrent or metastatic breast cancer in combination with HLX87. HLX43 (PD-L1 ADC) in combination with HLX07 or serplulimab injection, and HLX701 (CD47 fusion protein), among others, have entered Phase I clinical trials, further enhancing the Company’s layout in the fields of tumor immunology and targeted therapy.

Expanding Global Commercialization Network and Enhancing Academic Influence

During the reporting period, Fosun Pharma’s global registration and commercialization network continued to expand. The overseas registration of products like denosumab and bevacizumab progressed steadily. The Company reached deep collaborations with global partners such as Eisai and Abbott to accelerate the market coverage of innovative products in Japan, Asia-Pacific, Middle East, Africa, and Eastern Europe.

In the academic field, several innovation achievements were presented at top international conferences such as AACR (Free AACR Whitepaper) and ASCO (Free ASCO Whitepaper). The Phase III study data of luvometinib tablets for adult patients with neurofibromas type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas (PN) was selected for a rapid oral abstract session at 2026 ASCO (Free ASCO Whitepaper), the study results will be released for the first time during the meeting. At the 2026 AACR (Free AACR Whitepaper) Annual Meeting, Shanghai Henlius, a Fosun Pharma’s subsidiary, presented preclinical data for the novel trispecific T-cell engager HLX3902 and the tetraspecific antibody HLX3901, demonstrating best-in-class treatment potential, building a solid foundation for the future clinical development.

AI Embracement: Driving Digital Transformation via FoSTRAID

Fosun Pharma continues to deepen its digitalization and AI strategic layout, and systematically advances the platformisation, engineering and scaled implementation of AI capabilities focusing on core aspects such as new drug R&D, clinical research, products and services, and operation management. On this basis, the fully AI- embracing strategy centered on FoSTRAID (Fosun Pharma Strategic Transformation via AI & Data science) was further defined and steadily advanced. By integrating resources, a digital-intelligence architecture that promotes synergistic development across "foundation — platform — data — agent — scenario — mechanism" has been established.

The self-developed PharmAID Pharmaceutical Intelligence Platform deeply applies large language models, fine-tuned with professional medical corpora and R&D data. It integrates full-cycle of decision-making tools including competitor analysis, clinical competitive performance evaluation, NDA approval prediction, and peak sales estimation, comprehensively applied in key scenarios such as target discovery, molecular optimization, clinical writing, intelligence retrieval, and literature interpretation, providing systematic and data-driven support for drug R&D decision-making.

Looking ahead, Fosun Pharma will continue to adhere to the dual drivers of innovation and globalization, deepen its core technology platforms, promote the implementation of AI strategy and accelerate the transformation of its pipeline and global market access. The Company remains committed to providing high-quality healthcare products and services to patients worldwide, striving to become a leading global healthcare innovation integrator.

(Press release, Fosun Pharma, APR 28, 2026, View Source [SID1234664870])

On April 28, 2026 Veracyte, Inc. (Nasdaq: VCYT), a leading cancer diagnostics company, reported that more than 15 studies focused on the Decipher portfolio will be presented at the 2026 American Urological Association (AUA) Annual Meeting, taking place May 15-18 in Washington, D.C. The data showcases the growing impact of the Decipher Bladder and Prostate tests to inform more personalized patient care. The studies provide insights into molecular subtypes associated with patient outcomes in bladder cancer and feature national-scale real‑world data analyses in prostate cancer that demonstrate how Decipher Prostate informs treatment decisions. Additional real‑world data from the Decipher Genomics Research for Intelligent Discovery (GRID) research tool continues to fuel the flywheel of evidence in urologic cancers through ongoing research studies.

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"These studies underscore the power of Veracyte’s Diagnostics Platform to generate clinically meaningful evidence at scale," said Elai Davicioni, Ph.D., Veracyte’s medical director, Urology. "With more than a decade of data in our Decipher GRID tool, we are accelerating research to drive new discoveries that help clinicians better personalize care and improve outcomes for patients."

At this year’s meeting, during Bladder Cancer Awareness month in May, several studies will highlight the clinical relevance of molecular subtyping in bladder cancer using the Decipher Bladder test, including its role in guiding treatment decisions and predicting outcomes. Key presentations include:

Title:

Molecular Characterization and a Showcase Clinical Study on Treatment De-escalation for Luminal Favorable Muscle-Invasive Bladder Cancer

Presenter:

Melissa Moran, DO, Rutgers Robert Wood Johnson University Hospital

Format:

Poster

Abstract #:

IP35-15

Date/Time:

Saturday, May 16, 2026, 9:30 – 11:30 AM ET
Location:

147A

Title:

Non-luminal molecular subtypes demonstrate highest complete response rates after neoadjuvant chemo-immunotherapy for Muscle-Invasive Bladder Cancer (Biomarker analyses from NURE-combo & BLASST-01 phase 2 trials)

Presenter:

Joep J. de Jong, M.D., Erasmus MC Cancer Institute, Rotterdam, Netherlands

Format:

Poster

Abstract #:

IP47-06

Date/Time:

Saturday, May 16, 2026, 3:30 – 5:30 PM ET

Location:

147A

Title:

Non-Luminal Subtype is Associated with Worse Overall Survival in High-Risk Non-Muscle Invasive Bladder Cancer Patients – Biomarker Results from The Bladder Cancer Prognosis Programme

Presenter:

Joep J. de Jong, M.D., Erasmus MC Cancer Institute, Rotterdam, Netherlands

Format:

Poster

Abstract #:

IP47-07

Date/Time:

Saturday, May 16, 2026, 3:30 – 5:30 PM ET

Location:

147A

Title:

Examining the impact of TGF-β activity on fibroblast infiltration and immune exclusion in muscle-invasive bladder cancer
Presenter:

Shilpa Gupta, M.D., Cleveland Clinic Foundation

Format:

Podium presentation

Abstract #:

PD15-10

Date/Time:

Saturday, May 16, 2026, 4:42 – 4:50 PM ET

Location:

206

Together, these studies demonstrate how molecular subtyping can help refine risk stratification, identify patient subsets with differing clinical outcomes, and deepen understanding of tumor biology.

"These data suggest that molecular subtyping using Decipher Bladder may help identify patients who are more likely to achieve downstaging and complete responses to neoadjuvant chemo-immunotherapy," said Shilpa Gupta, M.D., GU Oncologist, Cleveland Clinic. "By better understanding tumor biology upfront, we have the potential to more precisely select therapies and, ultimately, improve outcomes while avoiding unnecessary treatment for patients with muscle-invasive bladder cancer."

Unlocking Insights with Real-World Evidence from Decipher Prostate and the GRID Database

Large‑scale analyses using Decipher genomic data alongside real‑world clinical datasets provide insights into how prostate cancer management is being shaped across diverse patient populations, helping clinicians more confidently tailor treatment decisions. These efforts also support ongoing research to deepen understanding of prostate cancer biology and inform personalized care. Key presentations include:

Title:

Association of Biopsy-Based Genomic Classifier and Initial Treatment for Prostate Cancer: Results from a National Clinical–Genomic Linkage

Presenter:

Michael Leapman, M.D., Department of Urology, Yale School of Medicine

Format:

Poster

Abstract #:

IP31-20

Date/Time:

Saturday, May 16, 2026, 9:30 – 11:30 AM ET

Location:

145AB

Title:

Genomic Classifier Results and Use of Post-Prostatectomy Treatment Among a National Cohort of Patients with Prostate Cancer

Presenter:

Michael Leapman, M.D., Department of Urology, Yale School of Medicine

Format:

Poster

Abstract #:

IP62-22

Date/Time:

Sunday, May 17, 2026, 1:00 – 3:00 PM ET

Location:

146A

Title:

Correlative analysis of the expression of two PET imaging targets, PSMA and ACP3, in a large radical prostatectomy cohort from the Decipher GRID registry

Presenter:

Mohammed Shahait, M.D., UCI Urology

Format:

Poster

Abstract #:

IP62-05

Date/Time:

Sunday, May 17, 2026, 1:00 – 3:00 PM ET

Location:

146A

"Decipher GRID enables analyses that were not previously possible," said Dr. Michael Leapman Associate Professor of Urology, Yale School of Medicine. "Our findings suggest that genomic information from Decipher Prostate may help refine treatment decision-making and move care toward more precise, individualized management of prostate cancer."

More information about Veracyte’s presence at AUA 2026 can be found at the company’s booth #3405 and on Veracyte’s website here.

About Decipher Prostate

The Decipher Prostate Genomic Classifier is a 22-gene test, developed using RNA whole-transcriptome analysis and machine learning, that helps inform treatment decisions for patients with prostate cancer. The test is performed on biopsy or surgically resected samples and provides an accurate risk of developing metastasis with standard treatment. Armed with this information, physicians can better personalize their patients’ care and may recommend less-intensive options for those at lower risk or earlier, more-intensive treatment for those at higher risk of metastasis. The Decipher Prostate test has been validated in many dozens of published studies involving more than 100,000 patients. It is the only gene expression test to achieve "Level 1B" evidence status and inclusion in the risk-stratification table in the most recent NCCN Guidelines* for prostate cancer. More information about the Decipher Prostate test can be found here.

About Decipher Bladder

The Decipher Bladder Genomic Classifier is a 219-gene test, developed using RNA whole-transcriptome analysis and machine learning, that is designed for use in patients following bladder cancer diagnosis who face questions regarding treatment intensity. The test classifies bladder tumors into five molecular subtypes, each having distinct tumor biology and potential clinical implications. This information can help physicians and their patients better understand the degree of benefit that would likely be gained from neoadjuvant chemotherapy and/or the likelihood of harboring non-organ-confined disease at time of surgery, respectively. More information about the Decipher Bladder test can be found here.

About Decipher GRID

The Decipher GRID database includes more than 200,000 whole-transcriptome profiles from patients with urologic cancers and is used by Veracyte and its partners to contribute to continued research and help advance understanding of prostate and other urologic cancers. GRID-derived information is available on a Research Use Only basis. More information about Decipher GRID can be found

(Press release, Veracyte, APR 28, 2026, View Source [SID1234664865])

On April 28, 2026 Theolytics, a clinical-stage biotechnology company developing next-generation oncolytic immunotherapies, reported that the first patient has been dosed in a Phase 1 clinical trial evaluating intraperitoneal (IP) administration of THEO-260 for the treatment of women with Platinum-Resistant Ovarian Cancer (PROC) (NCT07211659).

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The US trial, OCTOPOD-IP, will be the second clinical trial investigating THEO-260 in this indication, the first being the ongoing Phase 1/2a trial called OCTOPOD-IV in the UK, Spain and Canada (NCT06618235).

Positioned to tackle the complex, immune-suppressed nature of advanced solid tumours, THEO-260 is designed to target and eliminate cancer cells and cancer-associated fibroblasts (CAFs) while inducing immune activation. PROC represents a prototype of a broader category of stroma-rich solid tumours for which THEO-260 is being developed.

The US OCTOPOD-IP trial will assess the safety and tolerability of THEO-260 administered directly to the peritoneal cavity (IP delivery) in PROC patients and include extensive biomarker studies to showcase THEO-260’s differentiated mode of action.

The trial is sponsored by Theolytics and will be run under the guidance of principal investigator Amir Jazaeri, MD, Professor, Department of Gynecologic Oncology and Reproductive Medicine, Division of Surgery at The University of Texas MD Anderson Cancer Center.

"We are proud to announce the dosing of the first patient in this important study," said David Apelian MD, CEO of Theolytics. "The initiation of our US trial to evaluate the intraperitoneal delivery of THEO-260 in patients with PROC under the company’s first IND marks an important new milestone for Theolytics. THEO-260 is designed to be effective against ovarian cancer and other stroma-rich cancers by destroying cancer-associated fibroblasts within the tumour microenvironment as well as killing cancer cells directly, triggering immunogenic cell death and promoting T-cell activation. By advancing THEO-260 for the treatment of ovarian cancer, we hope to address the high unmet need for patients suffering with this disease and potentially for broader use in other difficult-to-treat, stroma-rich solid tumours."

(Press release, Theolytics, APR 28, 2026, View Source [SID1234664864])

On April 28, 2026 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that the Data Monitoring Committee completed the interim efficacy analysis of its pivotal Phase 3 FLASH2 (Fluorescent Light Activated Synthetic Hypericin 2) trial evaluating HyBryte (Synthetic Hypericin) in the treatment of cutaneous T-cell lymphoma (CTCL). Under the terms of the interim analysis, the study was recommended to halt for futility.

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"We are obviously very disappointed with the unanticipated outcome of the study," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Despite the fact that HyBryte demonstrated statistically significant reductions in CTCL lesions after 6 weeks treatment in the first FLASH study, a similar signal was not observed with 18 weeks of treatment in this study. Over the coming weeks, we will analyze the data to better determine why the study did not meet expectations. If there is any clarity gained from further analysis of the dataset, especially with respect to specific subsets of patients that may benefit from HyBryte therapy, then we intend to communicate our findings and explore follow-up discussions with the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA)."

Dr. Schaber continued, "With approximately $5.9 million of cash, we will evaluate all strategic options moving forward, including but not limited to merger and acquisition opportunities as well as the potential of advancing dusquetide for the treatment of Behçet’s Disease, which demonstrated promising biological efficacy in a Phase 2 study last year using the intravenous formulation and has received orphan drug designation most recently from the EMA."

About HyBryte

HyBryte (research name SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by safe, visible light approximately 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).

The published Phase 3 FLASH trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle (Cycle 1), 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the modified Composite Assessment of Index Lesion Severity [mCAILS] score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in this cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment responses also revealed a statistically significant improvement (p<0.0001) between the two timepoints, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a positive treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte’s mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant, and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With very limited systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

Following the first Phase 3 study of HyBryte for the treatment of CTCL, the FDA and the EMA indicated that they would require a second successful Phase 3 trial to support marketing approval. With agreement from the EMA on the key design components, the second, confirmatory study, called FLASH2, has demonstrated a good safety profile in the pre-specific safety evaluations by the Data Monitoring Committee. This study is a randomized, double-blind, placebo-controlled, multicenter study that replicates the double-blind, placebo-controlled design used in the first successful Phase 3 FLASH study that consisted of three 6-week treatment cycles (18 weeks total), with the primary efficacy assessment occurring at the end of the initial 6-week double-blind, placebo-controlled treatment cycle (Cycle 1). However, this second study extends the double-blind, placebo-controlled assessment to 18 weeks of continuous treatment (no "between-Cycle" treatment breaks) with the primary endpoint assessment occurring at the end of the 18-week timepoint. In the first Phase 3 study, a treatment response of 49% (p<0.0001 vs patients receiving placebo in Cycle 1) was observed in patients completing 18 weeks (3 cycles) of therapy. In this second study, all important clinical study design components remain the same as in the first FLASH study, including the primary endpoint and key inclusion-exclusion criteria.

In light of the interim analysis results, the Company will evaluate all available data and assess potential next steps, if any, with respect to the HyBryte development program.

Additional supportive studies have demonstrated the utility of longer treatment times (Study RW-HPN-MF-01), the lack of significant systemic exposure to hypericin after topical application (Study HPN-CTCL-02) and its relative efficacy and tolerability compared to Valchlor (Study HPN-CTCL-04).

In addition, the FDA awarded an Orphan Products Development grant to support the investigator-initiated study evaluation of HyBryte for expanded treatment in patients with early-stage CTCL, including in the home use setting. The grant, totaling $2.6 million over 4 years, was awarded to the University of Pennsylvania that was a leading enroller in the Phase 3 FLASH study.

About Cutaneous T-Cell Lymphoma (CTCL)

CTCL is a class of non-Hodgkin’s lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as patches and may progress to raised plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas.

CTCL constitutes a rare group of NHLs, occurring in about 4% of the more than 1.7 million individuals living with the disease in the U.S. and Europe (European Union and United Kingdom). It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects approximately 31,000 individuals in the U.S. (based on SEER [Surveillance, Epidemiology, and End Results] data, with approximately 3,200 new cases seen annually) and approximately 38,000 individuals in Europe (based on ECIS [European Cancer Information System] prevalence estimates, with approximately 3,800 new cases annually).

(Press release, Soligenix, APR 28, 2026, View Source [SID1234664863])

On April 28, 2026 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 high unmet need diseases, reported that new preclinical data from its PBGENE-DMD program have been accepted for an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2026 Annual Meeting, taking place May 11-15, 2026, in Boston, Massachusetts. The accepted abstract highlights new data demonstrating compelling efficacy observed in early-juvenile mice supporting the potential benefit of earlier intervention with PBGENE-DMD in younger patient populations. These data build on previously shared updates showing treatment with PBGENE-DMD leads to durable functional improvement in a humanized Duchenne muscular dystrophy (DMD) mouse model.

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Details of the presentation:

Abstract title: PBGENE-DMD gene editing drives safe, efficacious, and durable functional improvement in a humanized Duchenne muscular dystrophy mouse model

Session: Emerging molecular therapeutic strategies for muscular dystrophies
Presenter: Adam Michler Ph.D., DMD Research Lead
Presentation Type: Oral Presentation
Presentation Time: May 14, 2026 at 8:45 a.m. ET

About PBGENE-DMD, A Muscle-Targeted Excision Program

PBGENE-DMD is Precision’s development program for the treatment of Duchenne Muscular Dystrophy (DMD), a devastating genetic disease caused by mutations in the dystrophin gene that prevents production of the dystrophin protein, which is essential for maintaining muscle structural integrity and function. DMD affects approximately 15,000 patients in the U.S. alone, and there are currently no approved therapies capable of driving significant, durable functional improvements over time.

PBGENE-DMD is designed to durably improve function for approximately 60% of patients with DMD by employing two complementary ARCUS nucleases, delivered using a single AAV, to excise exons 45-55 of the dystrophin gene, restoring expression of a near full-length dystrophin protein. This protein more closely resembles normal dystrophin than synthetic, truncated microdystrophin approaches, which offer minimal functional benefit. Precision’s Phase 1/2 FUNCTION-DMD study is expected to enroll ambulatory DMD patients with mutations between exons 45 and 55, which impact approximately 60% of boys with DMD. The clinical trial will employ an appropriate immune modulation regimen and safety monitoring program to treat patients at world class specialized DMD clinical sites.

PBGENE-DMD was granted Orphan Drug Designation by the FDA in July 2025. The PBGENE-DMD program is eligible for a Priority Review Voucher (PRV) via the Rare Pediatric Disease Priority Review Voucher (PRV) program, which was signed into law on February 3, 2026, as part of the Consolidated Appropriations Act of 2026. PBGENE-DMD received Fast Track designation from the FDA in February 2026.

Further details on the trial can be found on Precision’s website and clinicaltrials.gov identifier NCT07429240.

(Press release, Precision Biosciences, APR 28, 2026, View Source [SID1234664862])