mediCAD® 8 Revolutionizes Orthopedic and Trauma Planning with Intelligent AI Integration




mediCAD® 8 Revolutionizes Orthopedic and Trauma Planning with Intelligent AI Integration

LANDSHUT, Germany, Oct. 26, 2025 (GLOBE NEWSWIRE) — The latest release of mediCAD® 8 by mediCAD Hectec GmbH marks a new era in intelligent orthopedic and trauma planning. Combining artificial intelligence, automation, and with a completely new user experience, mediCAD® 8 transforms how surgeons prepare and evaluate orthopedic and traumatological procedures – making planning faster, more accurate, and more consistent than ever before. The integrated mediCAD AI technology, represents a significant advancement in digital surgical planning, delivering measurable improvements in precision and efficiency. 

At the heart of the new release lies mediCAD AI, a framework that uses advanced algorithms to automatically recognize anatomical landmarks and interpret medical images with remarkable precision. The system detects key reference points across the hip, knee, and lower limb, supporting surgeons with data-driven accuracy from the first step of the planning process. Comparative studies show that landmark detection in mediCAD® 8 achieves an average deviation of less than two millimeters, ensuring precise alignment and reducing the need for manual corrections frequently required in earlier versions. 

The benefits of this automation extend beyond accuracy. In practice, segmentation and implant placement now only take about one minute, compared with two and a half minutes in the previous version, a reduction of roughly sixty percent. Notably, this performance can be achieved even by less experienced users, while version 7 requires expert-level familiarity to reach comparable results.  The AI-driven workflow supports the user in providing quick, accurate results, saving human time for medical evaluation.   

In addition to landmark recognition, mediCAD® 8 automatically detects image parameters such as orientation and scale, displaying only the relevant tools for each case. The system’s intelligent logic minimizes setup time and ensures that the appropriate planning modules are immediately available. The new 2D Trauma module introduces advanced features, including anatomy mirroring for comparison with the non-pathological side, integrated segmentation and fracture management with color-coded visualization, user-oriented interface for efficient fracture reduction. Expanded measurement options now cover the entire body, supporting comprehensive trauma analysis. The upcoming 3D Trauma module extends these capabilities into a three-dimensional environment, offering greater spatial understanding and precision. 

By combining AI-driven automation, accuracy, and advanced trauma functionality, mediCAD® 8 defines a new benchmark for intelligent digital surgery planning – empowering surgeons to plan faster, act with confidence, and enhance patient outcomes. 

Email: info@mediCAD.eu, phone number: +49 871 330 203 0

A video accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/47f0bf4d-9432-4b6a-9618-5d60088c29cb

Avacta presents first preclinical data from dual payload pre|CISION® medicines at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets




Avacta presents first preclinical data from dual payload pre|CISION® medicines at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets

First-in-class dual payload peptide drug conjugate delivers two complementary therapies from a single molecule with potential to overcome resistance and maximize tumor control

LONDON and PHILADELPHIA, Oct. 25, 2025 (GLOBE NEWSWIRE) — Avacta Therapeutics (AIM: AVCT, ‘Avacta’, ‘the Company’), a clinical stage biopharmaceutical company developing pre|CISION^®, a tumor-activated oncology delivery platform, today announced the presentation of preclinical data demonstrating its novel first-in-class dual payload pre|CISION^® technology (AVA6207) at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston, Massachusetts.

The data presented showcase the Company’s innovative approach to delivering two distinct therapeutic payloads simultaneously to the tumor microenvironment (TME) through a single fibroblast activation protein (FAP)-mediated cleavage event. Avacta is the first company to develop dual payload peptide drug conjugates (PDCs), circumventing resistance mechanisms that cancer cells develop against single-drug therapies while maximizing therapeutic effect through targeted combination delivery. This approach has the potential to address several critical challenges in cancer therapy.

The Company’s dual payload pipeline currently comprises two strategic approaches: combination of microtubule inhibition and topoisomerase I inhibition (MMAE and exatecan), representing two distinct anti-cancer mechanisms with established clinical activity; and DNA damage response (DDR) agents (ATR or PARP inhibitors) combined with exatecan, where inhibition of DNA repair potentiates the cytotoxic effect of exatecan.

Christina Coughlin, M.D., Ph.D., Chief Executive Officer of Avacta, commented:

“The first dual payload peptide drug conjugate marks an important step forward in oncology therapy, significantly extending the potential of our pre|CISION^® platform by implementing combination cancer therapy in a single small molecule medicine. The synergistic enhancement in anti-tumor activity observed with our exatecan-DDR inhibitor combinations highlights the potential of targeting the tumor with a potent cytotoxic drug while attacking the known resistance mechanisms. We believe this technology has the potential to markedly improve outcomes for cancer patients, particularly those with highly-resistant tumors.

“The pre|CISION^® platform has been validated by compelling clinical data with faridoxorubicin (AVA6000, FAP-Dox), demonstrating a tumor-to-plasma payload concentration of 100:1 and a significant reduction in off-target toxicities despite dosing up to approximately 4x the dose of conventional doxorubicin. The new dual payload intellectual property extends the platform to release two drugs from one pre|CISION^® molecule, targeting highly-resistant cancers by addressing key resistance mechanisms.”

Key Preclinical Findings:

The pre|CISION^® dual payload technology demonstrated robust FAP-selective delivery and potent anti-tumor activity across multiple complementary payload combinations:

• Validated dual payload release mechanism: Biologic and biochemical analyses confirmed simultaneous release of two independent payloads from a single FAP cleavage event. Modifications to the self-immolative linkers enabled tunable payload delivery kinetics, providing flexibility to optimize therapeutic profiles for different payload combinations. Strategic modifications of linker and capping group structures enabled optimization of compound activity.
• FAP-selective tumor cell killing with maintained potency: Dual payload compounds achieved potent cytotoxic activity comparable to free payloads in the presence of FAP (IC50 values of 2-9 nM), while demonstrating minimal activity without FAP (IC50 >100 nM), confirming excellent tumor selectivity in 2D and 3D tumor spheroid models over the course of seven days.
• Confirmed dual mechanism biomarker modulation: Target-specific biomarkers for both payloads were modulated only in the presence of FAP. FAP-Exd/MMAE compounds induced decreased TOP1 levels, DNA damage markers (γH2AX, pCHK1), tubulin depolymerization, M-phase arrest, and characteristic S-phase and G2/M cell cycle arrest patterns, confirming both mechanisms of action were operational.
• Enhanced synergistic activity addressing resistance mechanisms: FAP-Exd/PARPi and FAP-Exd/ATRi compounds demonstrated 4-5 fold greater FAP-dependent tumor cell killing compared to exatecan alone, directly addressing the known DDR-mediated resistance pathway to topoisomerase I inhibitors. FAP-dependent biomarker modulation confirmed payload release (reduced TOP1, PAR, and pCHK1 levels) alongside elevated DNA damage and apoptosis markers (γH2AX, cleaved PARP), with synergistically increased γH2AX demonstrating enhanced therapeutic effect.
• Validated bystander mechanism in physiologically relevant 3D models: Tumor-fibroblast co-culture 3D spheroid studies confirmed that FAP-positive cancer-associated fibroblasts mediate payload release, resulting in concentration of both therapeutics in the TME and effective killing of FAP-negative tumor cells. Activity was dependent on FAP presence, with minimal effect in monocultures or upon FAP inhibition, validating the bystander mechanism of action.

Dual payload cancer therapies represent a highly novel approach in oncology with the first dual payload antibody drug conjugates (ADCs) emerging in recent years. The pre|CISION^® platform has four key advantages over traditional ADC delivery, including tumor-specific payload release that avoids the toxicities associated with nonspecific release, small molecule manufacturing, better tumor penetration and bystander effect, along with a large addressable market of 90% of solid tumors.

The poster presentation (Abstract #C123) titled “Discovery and characterization of novel pre|CISION^® technology compounds delivering complementary dual payloads to the tumor microenvironment following FAP cleavage” is available on the Company’s website at https://avacta.com/.

The Avacta team will deliver a presentation via Investor Meet Company to review the published data upon their return from the Congress at 16:00 GMT on Wednesday October 29, 2025.  Details for the webinar will be posted on the Avacta home page and available through the Investor Meet Company platform.

For further information from Avacta, please contact:

About pre|CISION^®

The key aspect of pre|CISION^® is its peptide drug conjugates (PDC) technology. The combination of the cancer drug and the proprietary cleavable peptide (the PDC) is inert and incapable of entering cells and killing them until the peptide is specifically released within the tumor. The active payload in the pre|CISION^® PDC is released when the PDC comes into contact with the common tumor-associated protein, known as fibroblast activation protein (FAP), in the tumor. The release of the payload from the pre|CISION^® product directly in the tumor results in higher concentration of the drug at the tumor and lower blood and healthy tissue levels than standard systemic administration, offering the potential to improve efficacy and patient tolerability.

About Avacta –https://avacta.com/

Avacta is a clinical stage life sciences company developing an innovative proprietary drug delivery peptide drug conjugate (PDC) platform, pre|CISION^®. The pre|CISION^® platform uniquely enables the repurposing of a range of oncology drugs as PDC payloads with the goal to significantly reduce toxicity and side effects for patients by concentrating the drug directly in the tumor.

Preclinical Data Presented at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics Support Potential of BBO-10203, a First-in-Class RAS:PI3Kα Breaker That Inhibits KRAS-Mutant Tumor Growth without Inducing Hyperglycemia




Preclinical Data Presented at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics Support Potential of BBO-10203, a First-in-Class RAS:PI3Kα Breaker That Inhibits KRAS-Mutant Tumor Growth without Inducing Hyperglycemia

• Data demonstrate BBO-10203 blocks RAS-mediated activation of PI3Kα and strongly inhibits pAKT signaling in tumor cells without affecting glucose metabolism
• Robust monotherapy activity, as well as combination activity with BBOT’s KRAS^G12C ON/OFF inhibitor, BBO-8520, and panKRAS inhibitor, BBO-11818, was observed at well-tolerated dose levels in a panel of KRAS-mutant models
• The combination of a KRAS inhibitor with a PI3Kα pathway inhibitor may maximize the response rate and reduce the development of adaptive resistance mechanisms due to full inhibition of both MAPK and PI3Kα signaling
• BBOT-10203 is currently being evaluated in the Phase 1 BREAKER-101 trial for patients with HER2+ amplified or HR+/HER2- breast cancer, and KRAS mutant colorectal or non-small cell lung cancer with initial Phase 1 clinical data expected in the first half of 2026

SOUTH SAN FRANCISCO, Calif., Oct. 25, 2025 (GLOBE NEWSWIRE) — BridgeBio Oncology Therapeutics, Inc. (“BBOT”) (Nasdaq: BBOT), a clinical-stage biopharmaceutical company focused on RAS-pathway malignancies, today announced new preclinical data showing BBO-10203 selectively and specifically blocks the physical interaction between RAS and PI3Kα, resulting in the inhibition of RAS-driven PI3Kα-AKT signaling in tumors without the risk of hyperglycemia. In addition, combination activity with BBOT’s KRAS^G12C ON/OFF inhibitor, BBO-8520, and panKRAS inhibitor, BBO-11818, was observed at well tolerated dose levels in a panel of KRAS mutant models. The data were presented at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics.

“Aberrant activation of the PI3Kα pathway is among the most common oncogenic drivers across human cancers and leads to promotion of tumor growth, survival, and resistance to standard therapies,” said Pedro Beltran, PhD, Chief Scientific Officer of BBOT. “Current PI3Kα inhibitors are hindered by dose-limiting toxicities like hyperglycemia, which restrict target coverage, limit the number of eligible patients, and shorten the duration of treatment – leaving a significant unmet medical need. We’ve designed BBO-10203 to break the interaction between RAS and PI3Kα and inhibit RAS-mediated activation of the PI3Kα pathway. These preclinical data demonstrate BBO-10203 can accomplish this in in vivo studies without affecting glucose metabolism and achieve robust anti-tumor activity both as a monotherapy and in combination with our KRAS inhibitors, BBO-8520 and BBO-11818.”

These preclinical findings demonstrate BBO-10203 covalently binds PI3Kα on cysteine 242 in the RAS binding domain, breaking the protein-protein interaction between RAS and PI3Kα. Monotherapy results show achievement of complete cellular target engagement at low nanomolar concentrations and oral bioavailability with robust dose- and time-dependent inhibition of pAKT across diverse human cancer cell lines with KRAS mutations. Importantly, BBO-10203 does not induce hyperglycemia or hyperinsulinemia during an oral glucose tolerance test. In a panel of cell-line derived xenograft (CDX), patient-derived xenograft (PDX), and genetically engineered mouse (GEM) models, treatment with BBO-10203, both as a monotherapy and in combination with BBO-8520, BBOT’s direct inhibitor of KRAS^G12C in both the ON and OFF states, and with BBO-11818, the company’s panKRAS inhibitor targeting mutant KRAS in both the ON and OFF states with strong potency against KRAS^G12D and KRAS^G12V mutants, show robust anti-tumor activity. Importantly, the combination of BBO-10203 + BBO-8520 and BBO-10203 + BBO-11818 induces deep tumor regressions through direct effects on tumor cell proliferation and apoptosis and are well-tolerated.

BBO-10203 is currently being evaluated in the Phase 1 BREAKER-101 trial for patients with HER2+ amplified or HR+/HER2- breast cancer, and KRAS mutant colorectal or non-small cell lung cancer as a monotherapy and in combination with standard of care treatment, and will be evaluated in combination with KRAS inhibitors.

“We are pleased to share these preclinical data on BBO-10203’s potential as a RAS:PI3Kα breaker,” said Eli Wallace, PhD, Chief Executive Officer of BBOT. “By breaking the interaction between RAS and PI3Kα while preserving normal insulin signaling, these results further support our belief that BBO-10203 represents a truly differentiated approach with significant biological and therapeutic potential. We continue to enroll patients in our Phase 1 BREAKER-101 trial and look forward to expanding into combination studies, including with our own KRAS inhibitors.”

A copy of the poster titled “BBO-10203, a first-in-class, orally bioavailable, selective breaker of the RAS:PI3Kα interaction inhibits tumor growth alone and in combination with KRAS inhibitors in KRAS mutant models without inducing hyperglycemia” will be available on the “Publications” page of the BBOT website following the conference.

About BBO-10203
BBO-10203 is a first-in-class small molecule which breaks the protein-protein interaction between RAS and PI3Kα and inhibits RAS-mediated activation of the PI3Kα pathway. It selectively disrupts oncogenic RAS-PI3Kα signaling while sparing insulin-mediated glucose uptake, potentially maintaining efficacy with reduced risk of hyperglycemia or hyperinsulinemia. BBO-10203 is currently being evaluated in the Phase 1 BREAKER-101 trial for patients with locally advanced or metastatic HER2+ breast cancer, HR+/HER2- breast cancer, KRAS mutant colorectal cancer, and KRAS mutant non-small cell lung cancer. Initial Phase 1 clinical data are expected in the first half of 2026.

About BBOT
BBOT is a clinical-stage biopharmaceutical company advancing a next-generation pipeline of novel small molecule therapeutics targeting RAS and PI3Kα malignancies. BBOT has the goal of improving outcomes for patients with cancers driven by the two most prevalent oncogenes in human tumors. For more information, please visit www.bbotx.com and follow us on LinkedIn.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements in this press release that are not historical facts may be deemed forward-looking statements,  which generally are accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends. These statements are based on various assumptions, whether or not identified in this press release, and are the current expectations of BBOT’s management and are not predictions of actual performance. Many actual events and circumstances are beyond the control of BBOT. These forward-looking statements are subject to a number of risks and uncertainties, including changes in domestic and foreign business, market, financial, political, and legal conditions; risks relating to the uncertainty of the projected financial information with respect to BBOT; risks related to the approval of BBOT’s product candidates and the timing of expected regulatory and business milestones, including the progress of enrollment in clinical trials and availability of data from ongoing and planned clinical trials; the impact of competitive products; risks relating to BBOT’s ability to obtain sufficient supply of materials; and those factors discussed in documents BBOT has filed or will file with the U.S. Securities and Exchange Commission.

In addition, forward-looking statements reflect BBOT’s expectations, plans, or forecasts of future events and views as of the date of this press release and are qualified in their entirety by reference to the cautionary statements herein. BBOT anticipates that subsequent events and developments will cause BBOT’s assessments to change. These forward-looking statements should not be relied upon as any guarantee, assurance, prediction or definitive statement of fact or probability or as representing BBOT’s assessments as of any date subsequent to the date of this press release. Neither BBOT, nor any of its affiliates undertake any obligation to update these forward-looking statements, except as required by law.

BBOT Contacts:

Investor Contact:
Heather Armstrong, Head of Investor Relations
BBOT
Investors@BBOTx.com

Media Contact:
Jake Robison
Inizio Evoke Comms
Jake.robison@inizioevoke.com

Data presented at ACR Convergence 2025 highlight Sobi’s commitment to advancing care across rare and underserved inflammatory conditions




Data presented at ACR Convergence 2025 highlight Sobi’s commitment to advancing care across rare and underserved inflammatory conditions

NASP data highlight reduction in disease burden among people living with uncontrolled gout

Gamifant (emapalumab) data showed response rates in patients with MAS in Still’s disease across MAS subtypes, including in those with recurrent disease

Discussion of the first ever international, randomized, multicentre, double-blind, placebo-controlled phase 2 dose finding trial of Vonjo® (pacritinib) in adult patients with VEXAS syndrome

WALTHAM, Mass., Oct. 25, 2025 (GLOBE NEWSWIRE) — Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced the presentation of data across its immunology portfolio highlighting the company’s continued leadership in addressing rare and underserved inflammatory conditions. A total of 15 scientific abstracts, including six oral presentations featuring new data from completed and ongoing studies of Nanoecapsulated Sirolimus plus Pegadricase (NASP, formerly SEL-212), Vonjo® (pacritinib), and Gamifant® (emapalumab-lzsg), are being presented at the annual American College of Rheumatology (ACR) Convergence 2025 meeting in Chicago, October 24-29, 2025.
                                                                          
“Uncontrolled gout is a distinct and severe form of gout, marked by intense pain and an increased risk of mortality. Yet, despite the urgent need, there have been no new FDA-approved treatment options in over a decade,” said Laura Saltonstall, MD, Vice President of Immunology, Medical Affairs at Sobi North America. “We’re encouraged by the new data presented for our novel, investigational therapy, NASP, in uncontrolled gout, including the observed effect on painful flares and disease burden.”

Presentations of post hoc analysis of the DISSOLVE Trials investigating NASP in uncontrolled gout

DISSOLVE I and II were Phase III replicate, double-blind, placebo-controlled studies evaluating the efficacy and safety of NASP over 24 weeks. DISSOLVE I included a blinded extension phase for an additional 24 weeks.

Patients were randomized 1:1:1 to receive high-dose (HD) NASP: sequential infusions, 0.15 mg/kg NAS and 0.2 mg/kg pegadricase; low dose (LD) NASP: sequential infusions, 0.10 mg/kg NAS and 0.2 mg/kg pegadricase or placebo every four weeks. In the DISSOLVE I extension phase, NASP was generally well tolerated with low discontinuation rates. No new safety signals were reported.

In two oral presentations, data presented from post-hoc analyses evaluated serum uric acid (sUA) levels, joint exam findings, health-related quality-of-life (HRQOL) outcomes, and gout flares in the subgroup of patients with uncontrolled gout who received six doses of NASP or placebo.

The U.S. FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 27, 2026, following the acceptance of the BLA submission on September 10, 2025.

Nanoencapsulated Sirolimus Plus Pegadricase Reduced Disease Burden in Patients With Uncontrolled Gout: Results from the Phase 3 DISSOLVE Trials (Abstract Number: 2587)

• In the NASP arms, reductions in sUA levels were noted immediately after the first dose, and mean sUA generally remained ≤2.0 mg/dL throughout the study, with the placebo arm staying generally >6 mg/dL.
• From baseline to Week 24, median sUA level reduced by 97% and 98% in HD and LD NASP, respectively, and increased by 1.5% in placebo; ~2 fold reductions in the number of tender and swollen joints were observed with HD NASP and LD NASP compared to placebo.
• NASP treatment was associated with improvements in SF-36 PCS Score and HAQ-DI Pain Score from baseline to Week 24 that were ≥ 2-fold higher than changes in patients treated with placebo.

Nanoencapsulated Sirolimus plus Pegadricase (NASP) Demonstrates a Reduction in Gout Flares: Results from the Phase 3 DISSOLVE Studies (Abstract Number: 2588)

• During the first month of treatment (weeks 1-4), the proportion of patients experiencing a gout flare was similar for NASP and placebo, 23.8%, 28.6% and 20.9% in patients receiving HD NASP, LD NASP and placebo, respectively.
• During the trial, the number of flares continued to decrease in patients treated with NASP, while an increase in the number of gout flares was observed in patients receiving placebo.
• During weeks 13–24, the average number of gout flares per patient was 2.3 and 5.7 times fewer on HD and LD, respectively, vs PBO. This translates to 12 flares in 42 patients and 4 in 35 patients in the HD NASP and LD NASP, respectively vs and 44 in 67 pts in placebo.
• Across all treatment arms, most flares ranged from mild to moderate intensity.

Additional key data from select NASP poster sessions highlighted:

Reduction in Tophi Observed in Patients with Uncontrolled Gout Treated with NASP: Results from Phase 3 DISSOLVE Studies (Abstract Number: 1998)

• Tophus outcomes from the pooled Phase III studies: Observed complete resolution of tophus at Week 24 was 31% of patients receiving HD NASP and 48% receiving LD NASP and 5% with placebo.

“We have new hope for patients suffering from gout, where there is an unmet need due to disease burden. The reduction in uric acid levels, gout flares, and size of tophi highlight the potential of NASP in optimizing the treatment of uncontrolled gout,” said Puja P Khanna, MD, MPH, Professor of Medicine, University of Michigan Medical School.

Presentations on Gamifant (emapalumab-lzsg) in hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in Still’s disease

• Data were pooled from two open-label, single-arm interventional studies (NI-0501-06 [NCT03311854] and NI-0501-14 [EMERALD; NCT05001737]) in 39 patients with MAS in Still’s disease who had an inadequate response to high-dose GCs.
• The primary efficacy endpoint was complete response (CR) at Week 8 (resolution of clinical signs according to investigator assessment [visual analog scale (VAS) ≤1/10 cm]) and normalization of seven MAS-related laboratory parameters. Partial response (PR) was defined as VAS <4 cm and normalization of ≥3 abnormal baseline laboratory parameters included in the composite primary endpoint. Clinical remission was defined as VAS ≤1 cm.
• The most common adverse reactions (≥10%) for Gamifant included viral infection (44%), rash (21%), anemia (18%), leukopenia (15%), thrombosis (15%), bacterial infections (13%), headache (13%), hyperglycemia (13%), infusion-related reactions (13%), abdominal pain (10%), hypertension (10%), pyrexia (10%), and thrombocytopenia (10%).
• The U.S. Food and Drug Administration approved Gamifant as the first-ever treatment for adults and children with Macrophage Activation Syndrome in Still’s disease in June 2025.

Emapalumab Treatment for Patients with Differing Presentations of Macrophage Activation Syndrome (MAS) Secondary to Still’s Disease: Results from a Pooled Analysis of Two Prospective Trials (Abstract Number: 1671)

• In this post hoc analysis, the authors concluded that Gamifant demonstrated efficacy across diverse MAS presentations, including in recurrent cases.
  • Across all MAS presentations, Gamifant was associated with response rates consistent with the established efficacy profile.
  • Glucocorticoid doses tapering were similar in patients across MAS presentations.

Baseline Pharmacodynamic Markers and Response to Emapalumab in Children and Adults with Macrophage Activation Syndrome (MAS) in Still’s Disease: Results from a Pooled Analysis of Two Prospective Trials (Abstract Number: 0780)

• Gamifant treatment was associated with reductions in key PD markers of IFNγ activity, including CXCL9 and ferritin.
  • CXCL9 reduced by -99% and ferritin by -92% at week 8 in the subgroup of patients with complete or partial response.

Presentations on Vonjo (pacritinib) and VEXAS Syndrome
PAXIS: A Randomized, Double-Blind, Placebo-Controlled, Dose Finding Phase 2 Study (Part 1) Followed by an Open-Label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients with VEXAS Syndrome

• An oral presentation on the design of a randomized, double-blind, placebo-controlled, dose-finding Phase 2 study of pacritinib in patients with VEXAS syndrome.
  • PAXIS is the first randomized, double-blind, placebo-controlled pharmacotherapy trial for this complex, refractory, and sometimes fatal condition.

Development of a Consensus Definition of VEXAS Flare for Use in Clinical Research (Abstract Number: 0260)

• Data presented reviewed the development of consensus criteria to establish a standardized definition of “flare” in VEXAS for clinical research and patient care.
  • The consensus was developed by a 9-member international expert panel using modified Delphi methodology and was based on literature review and iterative consensus rounds.
  • This consensus definition provides a uniform framework for identifying disease activity and guiding future research for a disease with significant morbidity and mortality.

Development of a Disease Activity Index for the Assessment of VEXAS Syndrome (VEXAS-DAI) (Abstract Number: 0777)

• Additional data presented in a poster session highlighted the development of a new, comprehensive and standardized disease activity index (DAI) for measuring disease activity in VEXAS syndrome.
  • The VEXAS-DAI was developed based on input from 24 expert physicians worldwide to measure active inflammation in patients with VEXAS syndrome.
  • Work is ongoing to validate the VEXAS-DAI instrument in the context of a clinical trial and evaluate its potential in advancing the development of therapies in this area of high unmet medical need.

Contacts 
For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. 

GAMIFANT (emapalumab)

INDICATIONS

Gamifant (emapalumab-lzsg) is an interferon gamma (IFNγ)-neutralizing antibody indicated for the treatment of adult and pediatric (newborn and older) patients with:

• Primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.
• HLH/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.

IMPORTANT SAFETY INFORMATION

Infections

Gamifant may increase the risk of fatal and serious infections with pathogens including mycobacteria, herpes zoster virus, and histoplasma capsulatum. Do not administer Gamifant in patients with these infections until appropriate treatment has been initiated.

In patients with primary HLH receiving Gamifant in clinical trials, serious infections such as sepsis, pneumonia, bacteremia, disseminated histoplasmosis, necrotizing fasciitis, viral infections, and perforated appendicitis were observed in 32% of patients.

In patients with HLH/MAS in Still’s disease receiving Gamifant in clinical trials, serious infections such as pneumonia, cytomegalovirus infection, cytomegalovirus infection reactivation, and sepsis were observed in 13% of patients.

Evaluate patients for tuberculosis risk factors and test for latent infection prior to initiating Gamifant. Administer tuberculosis prophylaxis to patients at risk for tuberculosis or known to have a positive purified protein derivative (PPD) test result.

Consider prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infection while receiving Gamifant. Employ surveillance testing during treatment with Gamifant.

Closely monitor patients receiving Gamifant for signs or symptoms of infection, promptly initiate a complete diagnostic workup appropriate for an immunocompromised patient, and initiate appropriate antimicrobial therapy.

Increased Risk of Infection With Use of Live Vaccines

Do not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied.

Infusion-Related Reactions

Infusion-related reactions in patients with primary HLH, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion.

Infusion-related reactions in patients with HLH/MAS in Still’s disease, including pyrexia, headache, paresthesia, bone pain, pruritic rash, and peripheral coldness, were reported with Gamifant treatment in 13% of patients. Infusion-related reactions were reported as mild in 8% of patients and as moderate in 5% of patients.

Monitor patients for infusion-related reactions, which can be severe. Interrupt the infusion for infusion reactions and institute appropriate medical management before continuing infusion at a slower rate.

Adverse Reactions

Primary HLH

Serious adverse reactions were reported in 53% of patients. The most common serious adverse reactions (≥3%) included infections, gastrointestinal hemorrhage, and multiple organ dysfunction. Fatal adverse reactions occurred in 2 (6%) of patients and included septic shock and gastrointestinal hemorrhage.

The most common adverse reactions were (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%).

HLH/MAS

Serious adverse reactions were reported in 12 patients (31%), with the most common serious adverse reaction being pneumonia (5%). Fatal adverse reactions occurred in two patients (5%) and included multiple organ dysfunction and circulatory shock.

The most common adverse reactions (≥10%) for Gamifant included viral infection (44%), rash (21%), anemia (18%), leukopenia (15%), thrombosis (15%), bacterial infections (13%), headache (13%), hyperglycemia (13%), infusion-related reactions (13%), abdominal pain (10%), hypertension (10%), pyrexia (10%), and thrombocytopenia (10%).

Please see Full Prescribing Information for Gamifant.

VONJO (pacritinib)

Indication
VONJO is a kinase inhibitor indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 10⁹ /L (1). This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Important Safety Information

Hemorrhage
Avoid use in patients with active bleeding and hold VONJO prior to any planned surgical procedures. May require dose interruption, dose reduction or permanent discontinuation depending on severity.

Diarrhea
Manage significant diarrhea with anti-diarrheals, dose reduction, or dose interruption.

Thrombocytopenia
Manage by dose reduction or interruption.

Prolonged QT Interval
Avoid use in patients with baseline QTc >480 msec. Interrupt and reduce VONJO dosage in patients who have a QTcF >500 msec. Correct hypokalemia prior to and during VONJO administration.

Major Adverse Cardiac Events (MACE)
Risk may be increased in current/past smokers and patients with other cardiovascular risk factors. Monitor for signs, evaluate and treat promptly.

Thrombosis
Including deep venous thrombosis, pulmonary embolism, and arterial thrombosis may occur. Monitor for signs, evaluate and treat promptly.

Secondary Malignancies
Lymphoma and other malignancies may occur. Past/current smokers may be at increased risk.

Risk of Infection
Delay starting VONJO until active serious infections have resolved. Observe for signs and symptoms of infection and manage promptly.

Adverse Reactions
The most common (≥20% of patients) adverse reactions are diarrhea, thrombocytopenia, nausea, anemia, and peripheral edema.

Please see Full Prescribing Information for Vonjo.

About NASP, formerly SEL-212
NASP is a novel investigational medicine designed to reduce serum uric acid (sUA) levels in people living with uncontrolled gout, potentially reducing harmful tissue urate deposits which, when left untreated, can lead to debilitating gout flares and joint deformity. NASP is administered every 4 weeks as a sequential, two-component, infusion therapy consisting of tolerogenic nanoencapsulated sirolimus (NAS) which mitigates the formation of anti-drug antibodies (ADAs) and a uricase, pegadricase (P), which reduces serum uric acid. ADAs develop due to unwanted immune responses to biologic medicines, reducing their efficacy and tolerability, which remains an issue across multiple therapeutic modalities and disease states including uncontrolled gout.

About Uncontrolled Gout
Uncontrolled gout is the most common form of inflammatory arthritis with more than 8.3 million people in the US having been diagnosed. Gout is caused by high levels of uric acid in the body that accumulate around the joints and other tissues and can result in flares that cause intense pain. Approximately 200,000 people in the United States suffer from uncontrolled gout, with serum uric acid (sUA) levels above 6 mg/dL despite treatment with oral urate lowering therapies. This leads to debilitating flares and tophi. Elevated sUA levels have also been associated with diseases of the heart, vascular system, metabolism, kidney and joints.

About Gamifant® (emapalumab-lzsg)
Gamifant (emapalumab-lzsg) is the only approved anti-interferon gamma (IFNy) monoclonal antibody. Gamifant works by binding to and neutralizing interferon gamma (IFNy). When interferon gamma (IFNy) is secreted in an uncontrolled manner, hyperinflammation occurs within the body. Gamifant is indicated for administration through intravenous infusion over one hour.

Gamifant is approved in the US for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Gamifant is also approved in the US for the treatment of adult and pediatric (newborn and older) patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still’s disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.

About macrophage activation syndrome (MAS)
Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently in Still’s disease including systemic juvenile idiopathic arthritis (sJIA) – a rare systemic disorder of auto-inflammatory nature with common clinical manifestations such as daily spiking fever, typical transient cutaneous rash, arthritis, lymphadenopathy, hepatosplenomegaly and serositis. MAS is characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly progressing to multiple organ failure and death. MAS is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH).

About Vonjo® (pacritinib)
Vonjo is a kinase inhibitor that is indicated in the US for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 10⁹/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

About VEXAS
VEXAS syndrome is a disease that causes inflammatory and hematologic (blood) manifestations. The syndrome is caused by mutations in the UBA1 gene of blood cells and acquired later in life. The condition is not genetically inherited.

About Sobi® 
Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn.

Cullinan Therapeutics Shares Additional Preclinical B Cell Depletion Data for CLN-978, Supporting Clinical Development Across Multiple Autoimmune Diseases, at ACR Convergence 2025




Cullinan Therapeutics Shares Additional Preclinical B Cell Depletion Data for CLN-978, Supporting Clinical Development Across Multiple Autoimmune Diseases, at ACR Convergence 2025

CLN-978 led to rapid and deep B cell depletion in vitro and in vivo in multiple autoimmune diseases

CAMBRIDGE, Mass., Oct. 25, 2025 (GLOBE NEWSWIRE) — Cullinan Therapeutics, Inc. (Nasdaq: CGEM), a clinical-stage biopharmaceutical company accelerating potential first- or best-in-class, high-impact therapies in autoimmune diseases and cancer, will present new preclinical data for CLN-978, its novel investigational CD19xCD3 bispecific T cell engager. These data will be presented at the American College of Rheumatology (ACR) Convergence 2025, being held in Chicago, Illinois, October 24-29, in a poster presentation session on October 28, 10:30 a.m.-12:30 p.m. CT (Poster Session C, Poster Number 2293). Cullinan will also have a Booth (#1074) in the Exhibit Hall.

New in vitro preclinical data show CLN-978 robustly and specifically depleted target B cells while activating T cells in human peripheral blood mononuclear cells (PBMC) derived from patients with rheumatoid arthritis (RA), Sjögren’s disease (SjD), or systemic lupus erythematosus (SLE). These effects were similar to those observed with PBMCs from healthy donors.

Dose-dependent B cell depletion was observed following subcutaneous administration of CLN-978 in nonhuman primates (NHPs). Doses of CLN-978 considered well tolerated in the NHPs achieved deep and sustained B cell depletion in blood and multiple tissues including bone marrow and lymph nodes, suggesting the potential to achieve meaningful B cell depletion in patients.

In a murine model of SLE, CLN-978 treatment led to a reduction in circulating B cells, levels of anti-dsDNA IgG, and IgG deposition in the kidney, indicating a disease-modifying effect in both peripheral blood and affected disease tissues.

“We continue to generate preclinical data that reinforce the potential of CLN-978 as a highly potent T cell engager designed to deplete B cells deeply across multiple autoimmune diseases,” said Jeffrey Jones, MD, MBA, Chief Medical Officer, Cullinan Therapeutics. “In parallel, we are advancing our global clinical programs in rheumatoid arthritis, Sjögren’s disease, and systemic lupus erythematosus, recognizing the significant unmet needs for people living with these diseases. Current therapies are typically limited to addressing disease symptoms whereas CLN-978 has the potential to modify the underlying pathophysiology of the disease itself.”

Cullinan is advancing the global clinical development of CLN-978 through its OUTRACE studies across RA (NCT06994143), SjD (NCT07041099), and SLE (NCT06613360), with active trials now underway across all three indications and in multiple countries.

About CLN-978
CLN-978 is a novel, differentiated and highly potent CD19xCD3 bispecific T cell engager. CLN-978 triggers redirected lysis of CD19-expressing target cells in vitro and in vivo. CLN-978 is engineered to achieve very high affinity binding to CD19 to efficiently target B cells, including those with very low CD19 levels. Small in molecular size (65 kDa), CLN-978 contains two single-chain variable fragments, one binding with very high affinity to the CD19 target and the other binding to CD3 on T cells, and a single-domain antibody binding to human serum albumin to extend serum half-life. CLN-978 was developed by an internal Cullinan team and is a wholly owned asset. CLN-978 has the potential to offer a convenient, off-the-shelf, subcutaneously delivered therapeutic option for patients living with autoimmune diseases and is being studied in Cullinan’s OUTRACE studies for patients with rheumatoid arthritis, Sjögren’s disease, and systemic lupus erythematosus. CLN-978 is investigational and has not been approved by any health authority.

About Cullinan Therapeutics   
Cullinan Therapeutics, Inc. (Nasdaq: CGEM) is a biopharmaceutical company developing potential first- or best-in-class, high-impact therapies for autoimmune diseases and cancer. Cullinan pursues promising therapeutic targets while leveraging core expertise in T cell engagers, which are established in oncology and are now advancing into autoimmune diseases. With a clinical-stage pipeline built on a rigorous scientific approach and purposeful innovation, Cullinan is advancing its mission to deliver new standards of care for patients. Learn more about Cullinan at https://cullinantherapeutics.com/, and follow Cullinan on LinkedIn and X. 

Forward Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding the company’s beliefs and expectations regarding: future data presentations, our preclinical and clinical developments plans and timelines for CLN-978, the clinical and therapeutic potential of CLN-978, and other statements that are not historical facts. The words “believe,” “continue,” “could,” “estimate,” “expect,” “intends,” “may,” “plan,” “potential,” “project,” “pursue,” “will,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to known and unknown risks and uncertainties that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These risks include, but are not limited to, the following: uncertainty regarding the timing and results of regulatory submissions; the risk that any INDs or other global regulatory submissions we may file with the United States Food and Drug Administration or other global regulatory agencies are not cleared on our expected timelines, or at all; the success of our clinical trials and preclinical studies; the risks related to our ability to protect and maintain our intellectual property position; the risks related to manufacturing, supply, and distribution of our product candidates; the risk that any one or more of our product candidates, including those that are co-developed, will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the success of any collaboration, partnership, license or similar agreements. These and other important risks and uncertainties discussed in our filings with the Securities and Exchange Commission, including under the caption “Risk Factors” in our most recent Annual Report on Form 10-K and subsequent filings with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change, except to the extent required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release. Moreover, except as required by law, neither the company nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made.

Contacts:

Investors
Nick Smith
+1 401.241.3516
Nsmith@cullinantx.com

Media
Rose Weldon
+1 215.801.7644
Rweldon@cullinantx.com

Kyverna Therapeutics Highlights Potential of KYV-101 in Rheumatoid Arthritis with Phase 1 Data from Investigator-Initiated Trial Presented at ACR Convergence 2025




Kyverna Therapeutics Highlights Potential of KYV-101 in Rheumatoid Arthritis with Phase 1 Data from Investigator-Initiated Trial Presented at ACR Convergence 2025

KYV-101 resulted in a profound reduction in disease-associated autoantibodies and impact on disease activity in patients with difficult-to-treat rheumatoid arthritis (RA)

KYV-101 continues to demonstrate a well-tolerated profile, consistent with observations from 100 patients treated with KYV-101 to date¹

Emerging IIT data in RA reinforce broad potential for KYV-101 in rheumatology indications

EMERYVILLE, Calif., Oct. 25, 2025 (GLOBE NEWSWIRE) — Kyverna Therapeutics, Inc. (Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced the presentation of data from the Phase 1 portion of an investigator-initiated Phase 1/2 trial (IIT) evaluating KYV-101 in patients with active and treatment-refractory rheumatoid arthritis (RA). The data will be featured in a poster presentation from Charité, University of Berlin, at the American College of Rheumatology (ACR) Convergence 2025, taking place in Chicago, Illinois from October 24-29, 2025.

“We are very encouraged by these results, in which KYV-101 continues to provide robust CAR T cell expansion and B-cell depletion with a well-tolerated profile, driving compelling outcomes in patients with difficult-to-treat autoimmune disease,” said Warner Biddle, Chief Executive Officer of Kyverna Therapeutics. “Alongside IIT data recently presented in multiple sclerosis, these promising results in rheumatoid arthritis reinforce Kyverna’s potential to address serious autoimmune diseases beyond our core neuroimmunology CAR T programs, further demonstrating our leadership in this space.”

Charité – University of Berlin Poster Presentation

The COMPARE trial is an open-label, randomized, controlled Phase 1/2 study evaluating KYV-101 against the anti-CD20 monoclonal antibody rituximab in patients with anti-citrullinated protein antibody (ACPA)-positive, treatment-refractory RA with moderate to high disease activity.

All six patients enrolled in the Phase 1 portion of the study displayed highly refractory disease and had failed a mean of 5.8 prior biologic and targeted synthetic disease-modifying anti-rheumatic drugs (DMARDs) before entering the study and receiving a single infusion of 1×10⁸ KYV-101 CD19 CAR T cells with follow-up ranging from 28-175 days. The primary endpoint for the Phase 1 study was safety and tolerability with patients additionally evaluated for efficacy and key biomarkers of RA.

Key highlights are outlined below:

• Safety: KYV-101 was well-tolerated with no high-grade Cytokine Release Syndrome (CRS), and no instances of Immune Cell Associated Neurotoxicity Syndrome (ICANS).
• Biological Activity: CAR T-cells expanded rapidly, peaking between 14 and 21 days, and B-cell depletion occurred in all patients. Profound reductions in pathogenic ACPA, and in Rheumatoid Factor – Immunoglobulin M (RF-IgM) titers were also observed.
• Efficacy: With follow up ranging from 28 to 175 days, four out of six patients met the American College of Rheumatology 20% improvement criteria (ACR20) response, with two of these patients additionally achieving an ACR50 response (meeting 50% improvement thresholds).

“These are encouraging results in patients with long-standing, treatment-resistant rheumatoid arthritis for whom KYV-101 could offer profound relief from this debilitating disease,” said David Simon, M.D., Ph.D., Head of the Clinical Trial Unit in the Department of Rheumatology and Clinical Immunology at Charité, University of Berlin and Principal Investigator of the COMPARE trial. “These data highlight the safety and potency of KYV-101, with a rapid decline in key biomarkers and promising clinical response that are especially meaningful since all treated patients had failed multiple prior therapies. We believe these observations warrant further study of KYV-101 in RA as we progress into the Phase 2 portion of the study.”

These results supported the initiation of the randomized Phase 2 portion of the study, which is currently ongoing with patient enrollment completed.

Presentation Details

Title: An Open-label, Randomized, Controlled Phase 1/2 Study to Assess the Safety and Efficacy of KYV-101 Anti-CD19 CAR-T Cell Therapy in Active and Difficult-to-treat Rheumatoid Arthritis: Preliminary Results of the COMPARE Trial
Presenter: Dr. Ioanna Minopoulou, M.D., MSc, Charité, University of Berlin
Session: Rheumatoid Arthritis – Treatment Poster I, Poster Session A
Date and Time: Sunday, October 26, 2025, 10:30 AM – 12:30 PM CT

About KYV-101
KYV-101 is a fully human, autologous, CD19 CAR T-cell therapy with CD28 co-stimulation, designed for potency and tolerability, which is under investigation for B-cell-driven autoimmune diseases. With a single administration, KYV-101 has potential to achieve deep B-cell depletion and immune system reset to deliver durable drug-free, disease-free remission in autoimmune diseases.

About Rheumatoid Arthritis
Rheumatoid arthritis is a chronic and systemic autoimmune disease in which the immune system attacks the lining of the joints, causing persistent inflammation that leads to pain, swelling, disability and stiffness of multiple joints. Over time, ongoing immune activity can erode cartilage and bone, resulting in progressive joint damage and deformity. RA can also cause inflammation in other organs, including blood vessels, the lungs and heart, contributing to fatigue and overall reduced quality of life. Autoantibodies produced by B cells, most notably rheumatoid factor (RF) and anti-citrullinated protein antibodies (ACPAs), represent a hallmark of RA and play a key role in driving disease. While current therapies, including biologic and targeted synthetic agents, aim to manage symptoms and slow or prevent joint damage, many patients continue to experience persistent disease activity or lose response over time.

About Kyverna Therapeutics
Kyverna Therapeutics, Inc. (Nasdaq: KYTX) is a clinical-stage biopharmaceutical company focused on liberating patients through the curative potential of cell therapy. Kyverna’s lead CAR T-cell therapy candidate, KYV-101, is advancing through late-stage clinical development with registrational trials for stiff person syndrome and myasthenia gravis, and two ongoing multi-center Phase 1/2 trials for patients with lupus nephritis. The Company is also harnessing other KYSA trials and investigator-initiated trials, including in multiple sclerosis and rheumatoid arthritis, to inform the next priority indications for the Company to advance into late-stage development. Additionally, its pipeline includes next-generation CAR T-cell therapies in both autologous and allogeneic formats, including efficiently expanding into broader autoimmune indications and the potential to increase patient reach with KYV-102 using its proprietary whole blood rapid manufacturing process. For more information, please visit https://kyvernatx.com.

Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” The words, without limitation, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these or similar identifying words. Forward-looking statements in this press release include, without limitation, those related to: the topics to be discussed at the ACR Convergence 2025 meeting; KYV-101’s potential to deliver durable drug-free, disease-free remission with a single dose; KYV-101’s potential to continue to demonstrate a consistent and well-tolerated profile and its potential to offer profound relief from RA; Kyverna’s potential to address serious autoimmune diseases beyond its core neuroimmunology CAR T programs; Kyverna’s engagement with regulators; and Kyverna’s clinical trials, investigator initiated trials and named-patient access data. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties related to market conditions, the possibility that results from prior clinical trials, named-patient access activities and preclinical studies may not necessarily be predictive of future results; intellectual property rights; and other factors discussed in the “Risk Factors” section of Kyverna’s most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q that Kyverna has filed or may subsequently file with the U.S. Securities and Exchange Commission. Any forward-looking statements contained in this press release are based on the current expectations of Kyverna’s management team and speak only as of the date hereof, and Kyverna specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Investors: InvestorRelations@kyvernatx.com
Media: media@kyvernatx.com

¹ Includes patients treated in KYSA clinical trials, investigator-initiated trials, and “IH” or “Individueller Heilversuch,” also known as “named-patient basis access”. Similar to expanded access or compassionate use in the United States, IH is a regulatory mechanism in Germany that allows for the supply of a treatment that has not received marketing authorization for an individual patient in response to a request by the treating physician on behalf of the named patient. This option can be pursued for the expected benefit of a patient who has exhausted all available treatment options, under the discretion of the treating physician with the patient’s consent. The use of KYV-101 in the IH setting is not a substitute for, nor intended to replace, Kyverna’s clinical trials. The goal is not to assess the effectiveness of a potential therapy, but rather to provide an individual patient with a possible efficacious approach when all other treatment options have failed, as determined by the patient’s physician.

Novartis showcases significant immunology advancements in ACR congress with new data in complex autoimmune diseases




Novartis showcases significant immunology advancements in ACR congress with new data in complex autoimmune diseases

• Late-breaking positive Phase III data from ianalumab NEPTUNUS-1 and NEPTUNUS-2 trials in Sjögren’s disease to be presented
• Biomarker data informing use of investigational CAR-T cell therapy rapcabtagene autoleucel (YTB323) in systemic lupus erythematosus also to be presented
• Data underscore Novartis commitment to advance innovative medicines for complex, difficult-to-treat autoimmune diseases with high unmet need
• Novartis to hold virtual investor event following ACR highlighting immunology pipeline progress

Basel, October 25, 2025 – Novartis announced today plans to present data from 27 company- or investigator-sponsored abstracts across its Immunology portfolio and pipeline at the 2025 American College of Rheumatology (ACR) Convergence. Data to be presented include late-breaking pivotal Phase III results from the replicate NEPTUNUS-1 and NEPTUNUS-2 trials evaluating ianalumab in Sjögren’s disease¹. New biomarker data from an ongoing Phase 1/2 study of rapcabtagene autoleucel in severe refractory systemic lupus erythematosus will also be presented, along with Cosentyx data in multiple rheumatology indications^2,3.

“Our data at this year’s ACR demonstrate that Novartis is at the forefront of scientific innovation and is developing medicines for some of the most challenging autoimmune diseases, such as Sjögren’s,” said Angelika Jahreis, Global Head, Development, Immunology, Novartis. “Autoimmune diseases are often devastating and life-limiting. We are committed to developing new therapies with the potential to transform the standard of care for the millions who continue to suffer from rheumatic diseases.”

Ianalumab is an investigational medicine that has the potential to become the first targeted therapy for Sjögren’s disease, an area of high unmet need with no FDA-approved treatments^4,5. Sjögren’s disease affects millions of people globally and is the second most prevalent rheumatic disease⁶.

Additional presentations include data for rapcabtagene autoleucel, a novel one-time investigational CAR-T cell therapy being evaluated across several refractory autoimmune disease for its potential to induce an immune reset^7-9. Further presentations will feature real-world data on Cosentyx^® (secukinumab) in psoriatic arthritis, and new insights into the dual mode of action of ianalumab.

Investor call on Novartis Immunology pipeline 
Following the conclusion of ACR, Novartis will host a conference call for investors to provide updates on the company’s Immunology pipeline on Thursday, October 30, 2025, at 11:30 a.m. ET. Details can be found here.

Key abstracts accepted by ACR include:

About Novartis Immunology 
At Novartis, we’re advancing bold science for autoimmune diseases, where meaningful therapeutic progress has long stalled.

With a growing legacy of first-in-class innovation across Rheumatology, Dermatology and Allergy, and a diverse industry-leading pipeline, we’re committed to shaping what’s next in Immunology. From small molecules to biologics and CAR-T cell therapy, our innovation is powered by cutting-edge science, focused on where we can have the greatest impact on patient outcomes and supported by strong collaboration across the healthcare ecosystem. 

We’re not just treating autoimmune diseases. We’re reimagining medicine, together.

Product information
For full prescribing information, including approved indications and important safety information about marketed products, please visit https://www.novartis.com/about/products

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis 
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.

Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram.

References

1. Novartis. Data on file.
2. Morand E, et al. Biomarker Data From an Open-Label, Phase 1/2 Study for YTB323 (Rapcabtagene Autoleucel, a Rapidly Manufactured CD19 CAR-T Therapy) Suggest Reset of the B Cell Compartment in Severe Refractory SLE. Abstract presented at ACR Convergence 2025. Accessed September 19, 2025. https://acrabstracts.org/abstract/biomarker-data-from-an-open-label-phase-1-2-study-for-ytb323-rapcabtagene-autoleucel-a-rapidly-manufactured-cd19-car-t-therapy-suggest-reset-of-the-b-cell-compartment-in-severe-refractory-sle/
3. Armstrong A, et al. Comparison of Incidence of Psoriatic Arthritis in Patients With Psoriasis Treated With Interleukin-17 Inhibitors vs Interleukin-23 Inhibitors, Interleukin-12/23 Inhibitors, and Tumor Necrosis Factor Inhibitors in Real-World Practice: A Retrospective Study. Abstract presented at ACR Convergence 2025. Accessed September 19, 2025. https://acrabstracts.org/abstract/comparison-of-incidence-of-psoriatic-arthritis-in-patients-with-psoriasis-treated-with-interleukin-17-inhibitors-vs-interleukin-23-inhibitors-interleukin-12-23-inhibitors-and-tumor-necrosis-factor-i/
4. Dorner T, et al. Safety and Efficacy of ianalumab in patients with Sjogren’s disease: 52-week results from a randomized, placebo-controlled, phase 2b dose-ranging study. Arthritis and Rheumatology. 2025; 77(5):560-570
5. Negrini S, et al. Sjogren’s syndrome: a systemic autoimmune disease, Clin Exp Med. 2022; 22(1): 9-25
6. National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Health Care Services; Committee on Selected Immune Disorders and Disability. Sjogren’s Disease/Syndrome. Accessed September 11, 2025. https://www.ncbi.nlm.nih.gov/books/NBK584486/ 
7. ClinicalTrials.gov NCT05798117 [Last accessed: September 2025]
8. ClinicalTrials.gov NCT06665256 [Last accessed: September 2025]
9. ClinicalTrials.gov NCT06655896 [Last accessed: September 2025]

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ITM Announces Analyses from Phase 3 COMPETE Data Showing Higher Objective Response Rates with n.c.a. 177Lu-edotreotide (ITM-11) vs. Everolimus Across Subgroups of Patients with GEP-NETs at NANETS 2025 Annual Symposium




ITM Announces Analyses from Phase 3 COMPETE Data Showing Higher Objective Response Rates with n.c.a. 177Lu-edotreotide (ITM-11) vs. Everolimus Across Subgroups of Patients with GEP-NETs at NANETS 2025 Annual Symposium

Austin, Texas, October 25, 2025 – ITM Isotope Technologies Munich SE (ITM), a leading radiopharmaceutical biotech company, announced analyses data from its Phase 3 COMPETE trial in patients with Grade 1 or Grade 2 somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Results showed consistently higher objective response rates (ORR) and longer progression-free survival (PFS) across subgroups in patients treated with n.c.a. ¹⁷⁷Lu-edotreotide (also known as ITM-11 or ¹⁷⁷Lu-edotreotide) compared to everolimus, reinforcing its previously reported efficacy profile. Data were shared by study investigator, Jaume Capdevila, MD, PhD, in both oral and poster presentations at the 2025 North American Neuroendocrine Tumor Society (NANETS) Annual Multidisciplinary NET Medical Symposium, held October 23-25, 2025, in Austin, Texas.

As previously announced at ENETS 2025, the COMPETE trial, which included a total of 309 patients randomized to either ¹⁷⁷Lu-edotreotide (n=207) or everolimus (n=102), met its primary endpoint of progression-free survival, or PFS, (23.9 vs. 14.1 months; p=0.022; HR 0.67, 95% CI [0.48, 0.95]). At ESMO 2025, ITM announced that the COMPETE trial also met a key secondary endpoint of objective response rate (ORR) (21.9% vs 4.2%, p<0.0001).

In this analysis of the COMPETE trial presented at NANETS 2025, the key findings showed:

• ORR was higher in patients in the ¹⁷⁷Lu-edotreotide arm vs. everolimus across subgroups, including those with pancreatic NETs, Grade 1 tumors, Grade 2 tumors, and those who had received prior therapy (2^nd line); post-hoc analysis
• Although data is still maturing, there was a preliminary trend of longer median overall survival (OS) in the ¹⁷⁷Lu-edotreotide arm vs. everolimus in most patient subgroups

   

“We are encouraged by the positive trends we see in PFS extension and higher ORR for ¹⁷⁷Lu-edotreotide compared to everolimus in these patient subgroups. These results strengthen the existing data set for Lu-edotreotide’s potential as a new therapeutic option for people living with inoperable GEP-NETs,” said Dr. Capdevila, senior medical oncologist at Vall d’Hebron University Hospital, Barcelona. 

ITM also announced real-world clinical and meta-analysis data in patients with a range of neuroendocrine tumors at NANETS.

In early October, ITM presented dosimetry data from the COMPETE trial at the European Association of Nuclear Medicine (EANM) Annual Congress in Barcelona, Spain. The data showed that ¹⁷⁷Lu-edotreotide delivered targeted radiation to tumors while minimizing exposure to healthy tissue, supporting its efficacy and safety profile.

“These additional subgroup data reinforce and extend evidence from the COMPETE Phase 3 trial, supporting ¹⁷⁷Lu-edotreotide’s potential as a promising radiopharmaceutical therapy for neuroendocrine tumors if approved,” said Dr. Andrew Cavey, chief executive officer of ITM. 

Oral Presentation Details
Title: Efficacy of ¹⁷⁷Lu-edotreotide vs everolimus in patients with grade 1 or grade 2 GEP-NETs: Phase 3 COMPETE trial (post hoc subgroup analyses)
Date and Time: October 24, 2025, 3:35-4:50 pm Central Time
Session: Part II Featured Abstracts| Access to New Treatments – How can we bring innovative treatments to patients more quickly and effectively?
Presenter: Jaume Capdevila, MD, PhD, senior researcher, department of Medical Oncology, Vall d’Hebron University Hospital & Vall d’Hebron Institute of Oncology

Poster Presentation Details:
Title: Efficacy of ¹⁷⁷Lu-edotreotide vs everolimus in patients with grade 1 or grade 2 GEP-NETs: Phase 3 COMPETE trial (post hoc subgroup analyses)
Date and Time: Friday, October 24, 5:15-6:30 pm Central Time
Session: NANETS Poster Tour
Presenter: Dr. Jaume Capdevila, Vall d’Hebron University Hospital, Barcelona.

Title: First-line Treatment with ¹⁷⁷Lu-edotreotide ([¹⁷⁷Lu]Lu-DOTATOC) in patients with NETs: a Swiss NET Registry Analysis
Date and Time: Friday, October 24, 5:15-6:30 pm Central Time
Session: NANETS Poster Tour 
Presenter: Dr. Guillaume Nicolas, University Hospital Basel

Title: Efficacy and safety of ¹⁷⁷Lu-edotreotide ([¹⁷⁷Lu]Lu-DOTATOC) for the treatment of neuroendocrine tumors (NETs) – a systematic literature review (SLR) and meta-analysis
Date and Time: Friday, October 24, 5:15-6:30 pm Central Time
Session: NANETS Poster Tour
Presenter: Dr. Julia G Fricke, University Hospital Basel

About the COMPETE Trial
The COMPETE trial (NCT03049189) evaluated ¹⁷⁷Lu-edotreotide (ITM-11), a proprietary, synthetic, targeted radiotherapeutic investigational agent compared to everolimus, a targeted molecular therapy, in patients with inoperable, progressive Grade 1 or Grade 2 gastroenteropancreatic neuroendocrine tumors (GEP-NETs). This trial met its primary endpoint, with ¹⁷⁷Lu-edotreotide demonstrating clinically and statistically significant improvement in progression-free survival (PFS) compared to everolimus. ¹⁷⁷Lu-edotreotide is also being evaluated in COMPOSE, a Phase 3 study in patients with well-differentiated, aggressive Grade 2 or Grade 3, SSTR-positive GEP-NET tumors.

About ITM Isotope Technologies Munich SE 
ITM, a leading radiopharmaceutical biotech company, is dedicated to providing a new generation of radiopharmaceutical therapeutics and diagnostics for hard-to-treat tumors. We aim to meet the needs of cancer patients, clinicians, and our partners through excellence in development, production, and global supply of medical radioisotopes. With improved patient benefit as the driving principle for all we do, ITM advances a broad precision oncology pipeline, including multiple phase 3 studies, combining the company’s high-quality radioisotopes with a range of targeting molecules. By leveraging our two decades of pioneering radiopharma expertise, central industry position and established global network, ITM strives to provide patients with more effective targeted treatment to improve clinical outcome and quality of life. www.itm-radiopharma.com

ITM Contacts:
Corporate Communications
Kathleen Noonan/Julia Westermeir
Phone: +49 89 329 8986 1500
Email: communications@itm-radiopharma.com

Investor Relations
Ben Orzelek
Phone: +49 89 329 8986 1009
Email: investors@itm-radiopharma.com

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Aletheia Capital Endeavours Launches RepIntel, a Campaign-Grade Platform to Accelerate Global Access to Therapies




Aletheia Capital Endeavours Launches RepIntel, a Campaign-Grade Platform to Accelerate Global Access to Therapies

New York, New York, USA, Oct. 24, 2025 (GLOBE NEWSWIRE) — Aletheia Capital Endeavours (ACE) today announced the launch of RepIntel, a precision field-intelligence platform designed to close one of healthcare’s most persistent gaps: the failure of lifesaving therapies to reach the patients they were created for.

The company was founded by Gavin Perri, the New York impresario and Executive Chairman of Aletheia Capital Endeavours, and Filomena Di Luise, Senior Executive Manager at a Fortune 100 technology firm, who will serve as President and Chief Operating Officer for RepIntel. Di Luise brings enterprise-scale infrastructure expertise to the venture’s leadership team.

Drawing on frameworks that redefined political mobilization, RepIntel translates those same principles of precision targeting, coordinated field orchestration, and real-time intelligence into the final mile of healthcare delivery.

“Every year, scientific breakthroughs transform what medicine can do. Yet millions still go without the treatments that could change or save their lives,” said Gavin Perri, Executive Chairman of Aletheia Capital Endeavours. “RepIntel is designed to solve that systemic failure. By transforming field operations into an adaptive intelligence network, we’re building the connective tissue that allows innovation to translate into real-world impact.”

RepIntel unites vast healthcare datasets with ground-level decision-making, enabling pharmaceutical teams to identify unmet needs, prioritize underserved populations, and orchestrate outreach with surgical precision. Its first deployments target highly regulated markets, where the speed, accuracy, and equity of distribution directly determine patient outcomes.

The launch marks ACE’s first flagship technology venture and reflects a broader thesis: that the most resilient and equitable healthcare systems will be those that harness distributed intelligence, empowering those closest to patients to guide the flow of treatments and resources in real time. In doing so, RepIntel establishes the operational layer needed to ensure that scientific progress becomes societal progress.

About Aletheia Capital Endeavours

Aletheia Capital Endeavours (ACE) is a New York-based venture studio and applied think tank architecting the cultural, technological, and intellectual systems that shape the future. Its work spans entertainment ventures and large-scale philanthropic initiatives, often structured as thesis-oriented coalitions of high-net-worth partners that align social impact with strategic tax efficiency and legacy-oriented wealth planning.

About RepIntel

RepIntel is a precision field-intelligence platform incubated by ACE, designed as a critical layer in the delivery architecture of modern healthcare. It enables pharmaceutical and healthcare institutions to deploy campaign-grade targeting, adaptive outreach, and distributed decision-making to bridge the last-mile gap, ensuring that medical breakthroughs reach patients with the speed, precision, and equity the 21st century demands.

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CONTACT: ACE Press Assistant
press@aletheiacapital.org

Prenetics Global Limited Announces Proposed Public Offering




Prenetics Global Limited Announces Proposed Public Offering

CHARLOTTE, N.C., Oct. 24, 2025 (GLOBE NEWSWIRE) — Prenetics Global Limited (“Prenetics” or the “Company”), a leading health sciences company, today announced that it has commenced a public offering of shares of its Class A Ordinary Shares (or prefunded warrants in lieu thereof) and common warrants (the “Offering”). The Company will allocate proceeds from the offering toward the global expansion of IM8, driven by its explosive growth, and the strategic accumulation of Bitcoin (BTC) as part of its pioneering dual-pronged health and wealth strategy.

The Offering is subject to market conditions, and there can be no assurance as to whether or when the Offering may be completed, or as to the actual size or terms of the Offering.

Dominari Securities LLC is acting as the sole placement agent for the Offering

This Offering is being made pursuant to an effective shelf registration statement on Form F-3, as amended (No. 333-288824), declared effective by the U.S. Securities and Exchange Commission (the “SEC”) on September 11, 2025. A preliminary prospectus supplement and accompanying shelf prospectus (“Shelf Prospectus”) describing the terms of the Offering will be filed with the SEC and will be available on the SEC’s website located at https://www.sec.gov. Electronic copies of the preliminary prospectus supplement (together with the final prospectus supplement for this Offering, the “Prospectus Supplement”) and the accompanying shelf prospectus may be obtained, when available, by contacting Dominari Securities LLC, Attention: Syndicate Department, 725 5th Ave 23 Floor, New York, NY 10022, by email at info@dominarisecurities.com, or by telephone at (212) 393-4500.

Before investing in this Offering, interested parties should read, in their entirety, the prospectus supplement and the Shelf Prospectus and the other documents that the Company has filed with the SEC pertaining to the Offering and that are incorporated by reference in the Prospectus Supplement and the Shelf Prospectus, which provide more information about the Company and such Offering.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About Prenetics

Prenetics (NASDAQ: PRE) is a leading health sciences company redefining the future of health and longevity through IM8, its flagship consumer brand co-founded with David Beckham and now one of the world’s fastest-growing supplement brands.

As the first consumer health company to establish a Bitcoin Treasury, Prenetics continues to pioneer at the intersection of health innovation and digital assets — purchasing 1 Bitcoin per day, now totaling 272 BTC as of October 24, 2025.

Forward-Looking Statements

This press release contains forward-looking statements. These statements are made under the “safe harbor” provisions of the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements can be identified by terminology such as “will,” “expects,” “anticipates,” “future,” “intends,” “plans,” “believes,” “estimates,” and similar statements. Prenetics may also make written or oral forward-looking statements in its periodic reports to the U.S. Securities and Exchange Commission, in its annual report to shareholders, in press releases and other written materials, and in oral statements made by its officers, directors, or employees to third parties. Statements that are not historical facts, including statements about Prenetics’ beliefs and expectations, are forward-looking statements. Forward-looking statements involve inherent risks and uncertainties. A number of factors could cause actual results to differ materially from those contained in any forward-looking statement, including but not limited to the following: the Company’s ability to complete the offering, the Company’s ability to execute its new Bitcoin treasury strategy; the volatility of Bitcoin; the Company’s ability to manage its growth and expansion; the Company’s ability to compete in the highly competitive consumer health market; and other risks and uncertainties. Further information regarding these and other risks is included in Prenetics’ filings with the U.S. Securities and Exchange Commission. All information provided in this press release is as of the date of this press release, and Prenetics does not undertake any obligation to update any forward-looking statement, except as required under applicable law.

Investor Relations Contact:
investors@prenetics.com 
PRE@mzgroup.us 

Angela Cheung
Investor Relations / Corporate Finance 
angela.hm.cheung@prenetics.com