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New Novartis data at ASCO and EHA showcase momentum of pioneering portfolio with promising pipeline

On May 15, 2025 Novartis reported it will present data from 60 company or investigator sponsored abstracts that have the potential to change clinical practice, at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress (Press release, Novartis, MAY 15, 2025, View Source [SID1234653249]).

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"The breadth of our oncology and hematology portfolio – anchored by Kisqali, Pluvicto, Scemblix and Fabhalta – demonstrates our leadership in both solid tumors and hematologic diseases," said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. "At ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper), we will present new data on these priority medicines as well as updates from our pipeline and our industry-leading radioligand therapy research."

Novartis will also highlight its US partnerships with the National Football League (NFL), Alliance for Breast Cancer Policy, and ZERO Prostate Cancer, which encourage people to make proactive decisions about their health and advance patient-centered policy solutions to help improve outcomes.

"We’re witnessing a profound shift in how people move through their cancer journey, with cancer diagnoses occurring at younger ages and, simultaneously, older patients living longer and approaching aging with new vigor," said Victor Bultó, President, US, Novartis. "As a leader in driving medical advances in oncology, we have the responsibility to also make a difference in areas beyond treatment innovation. By partnering across the ecosystem, our goal is to advance the conversation around earlier detection and meet the evolving needs of this next generation of cancer patients."

Key highlights of data accepted by ASCO (Free ASCO Whitepaper) include:

Medicine Abstract Title Abstract Number/ Presentation Details
Kisqali
(ribociclib)* Efficacy and safety of ribociclib (RIB) + nonsteroidal aromatase inhibitor (NSAI) in NATALEE: Analysis across menopausal status and age Abstract #516
Rapid Oral
June 1, 8:00 – 9:30am CDT
Kisqali
(ribociclib) Real-world (RW) analysis of characteristics and risk of recurrence (ROR) in Black patients (pts) with HR+/HER2− early breast cancer (EBC) eligible for NATALEE Abstract #527
Poster Presentation
June 2, 9:00am – 12:00pm CDT
Kisqali
(ribociclib) Adjuvant WIDER: A phase 3b trial of ribociclib (RIB) + endocrine therapy (ET) as adjuvant treatment (tx) in a close-to-clinical-practice patient (pt) population with HR+/HER2− early breast cancer (EBC) Abstract #TPS617
Poster Presentation
June 2, 9:00am – 12:00pm CDT
Kisqali
(ribociclib) First-line (1L) ribociclib (RIB) + endocrine therapy (ET) vs combination chemotherapy (combo CT) in clinically aggressive hormone receptor (HR)+/HER2− advanced breast cancer (ABC): A subgroup analysis of patients (pts) with or without liver metastases (mets) from RIGHT Choice Abstract #1069
Poster Presentation
June 2, 9:00am – 12:00pm CDT
Scemblix
(asciminib) Efficacy and safety of asciminib (ASC) in patients (pts) with chronic-phase chronic myeloid leukemia (CML-CP) after 1 tyrosine kinase inhibitor (TKI): Interim analysis (IA) of the phase 2 ASC2ESCALATE trial Abstract #6516
Rapid Oral
May 30, 1:00 – 2:30pm CDT
Scemblix
(asciminib) Primary endpoint results of the phase 3b ASC4START trial of asciminib (ASC) vs nilotinib (NIL) in newly diagnosed chronic phase chronic myeloid leukemia (CML-CP): Time to treatment discontinuation due to adverse events (TTDAE) Abstract #6501
Oral Presentation
June 2, 3:00 – 6:00pm CDT
Pluvicto
(lutetium Lu 177 vipivotide tetraxetan) Clinical outcomes of prompt versus deferred 177Lu-PSMA-617 initiation for metastatic castration-resistant prostate cancer (mCRPC) based on prior androgen receptor pathway inhibitor (ARPI) and taxane chemotherapy exposure: a real-world PRostatE Cancer dISease observatION (PRECISION) data platform analysis Abstract #e17030
Online Publication
Pluvicto
(lutetium Lu 177 vipivotide tetraxetan) Real-world outcomes among patients with metastatic castration-resistant prostate cancer (mCRPC) receiving guideline-recommended therapies after treatment with 177Lu-PSMA-617: a real-world PRostatE Cancer dISease observatION (PRECISION) data platform analysis Abstract #e17035
Online Publication
Pluvicto
(lutetium Lu 177 vipivotide tetraxetan) PSMA-delay castration (DC): An open-label, multicenter, randomized phase 3 study of [177Lu]Lu-PSMA-617 versus observation in patients with metachronous PSMA-positive oligometastatic prostate cancer (OMPC) Abstract #TPS5127
Poster Presentation
June 2, 9:00am – 12:00pm CDT
Key highlights of data accepted by EHA (Free EHA Whitepaper) include:

Medicine Abstract Title Abstract Number/ Presentation Details
Fabhalta
(iptacopan) APPULSE-PNH: Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin (Hb) increases in patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH) and Hb ≥10 g/dL on anti-C5 therapy Abstract #S183
Oral Presentation
June 13, 5:00 – 6:15pm CEST

Fabhalta
(iptacopan) The 2-year safety and efficacy of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria (PNH) from APPLY- and APPOINT-PNH studies who entered the roll-over extension program (REP) Abstract #PF660
Poster Presentation
June 13, 6:30 – 7:30pm CEST

Scemblix
(asciminib) Asciminib (ASC) shows superior tolerability vs nilotinib (NIL) in newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP): Primary endpoint results of the phase (Ph) 3b ASC4START trial Abstract #S166
Oral Presentation
June 13, 5:00 – 6:25pm CEST

Scemblix
(asciminib) Improved patient-reported outcomes (PROs) with asciminib (ASC) vs investigator-selected tyrosine kinase inhibitors (IS-TKIs) in newly diagnosed chronic myeloid leukemia (CML): ASC4FIRST wk 48 analysis Abstract #PS1588
Poster Presentation
June 14, 6:30 – 7:30pm CEST

Scemblix
(asciminib)

Interim analysis (IA) results from ASC2ESCALATE support asciminib (ASC) as a treatment (Tx) option in chronic-phase chronic myeloid leukemia (CML-CP) after 1 tyrosine kinase inhibitor (TKI) Abstract #PF595
Poster Presentation
June 13, 6:30 – 7:30pm CEST

Pelabresib
(DAK539) Pelabresib in combination with ruxolitinib for janus kinase inhibitor-naive patients with myelofibrosis: 72-week follow-up with long-term efficacy outcomes of the phase III MANIFEST-2 study Abstract #S223
Oral Presentation
June 12, 5:00 – 6:15pm CEST

Ianalumab
(VAY736) A Phase 2 Study of Ianalumab in patients with primary immune thrombocytopenia previously treated with at least two lines of therapy (VAYHIT3) Abstract #S312
Oral Presentation
June 15, 11:00am – 12:15pm CEST

Rapcabtagene autoleucel
(YTB323) Rapcabtagene Autoleucel (YTB323) in patients with relapsed/refractory diffuse large B-cell lymphoma: A phase II trial clinical update Abstract #PF1152
Poster Presentation
June 13, 6:30 – 7:30pm CEST

Akari Therapeutics Reports First Quarter 2025 Financial Results and Provides Corporate Update

On May 15, 2025 Akari Therapeutics, Plc (Nasdaq: AKTX), a biotechnology company developing novel Antibody Drug Conjugates (ADCs) with immuno-oncology payloads for the treatment of cancer, reported its financial results for the first quarter ended March 31, 2025 and provided a corporate update (Press release, Akari Therapeutics, MAY 15, 2025, View Source [SID1234653207]).

"We remain laser focused on becoming a key player in the ADC space and advancing our novel ADC platform built around immuno-oncology payloads and our lead asset AKTX-101, an ADC targeting Trop2 with our immuno-oncology payload, PH1. We continue to develop and execute a clear path forward for our ADC pipeline and support these efforts with ongoing activities and building the team that we believe positions us for success in the near and long term," commented Abizer Gaslightwala, President and Chief Executive Officer of Akari. "In particular, we were pleased to recently welcome Mark Kubik, a seasoned leader in the Antibody Drug Conjugate space, as Head of Business Development, Oncology and believe his expertise will be invaluable as we continue to advance our novel ADC platform technology."

Leveraging its innovative payload platform, the Company is advancing a pipeline of potentially first-in-class, best-in-class ADC candidates across a wide range of cancer tumor targets. These initial candidates have shown significant tumor-killing activity in preclinical models with the ability to robustly activate the immune system to drive durable, and sustained outcomes.

Upcoming Expected Value-Driving Milestones

Novel ADC’s With Immuno-Oncology Payloads

Anticipate presenting preclinical data showing that a proof-of-concept ADC with PH1 payload exhibits robust immuno-oncology activity, at a scientific conference in second half of 2025.
Complete additional preclinical studies for novel PH1 payload exploring activity in prostate cancer cell lines.
Explore preclinical activity for AKTX-101 in different solid tumor indications including lung, as single agent and in combination with other approved agents.
Continue to focus on operational excellence and efficient capital allocation to advance novel payload ADC platform.
Ongoing efforts to seek strategic partners for research collaborations on PH1 immuno-oncology payload across customized tumor targets. Continued discussions with partners on advancing AKTX-101 ADC (Trop2/PH1 payload) through additional IND-enabling activities.
Non-Core Asset Out Licensing

Continue efforts to out-license non-core assets across inflammation, ophthalmology, and rare diseases as a source of non-dilutive capital to invest into ADC platform.
Summary of Financial Results for First Quarter 2025

The net loss from operations for the three months ended March 31, 2025 was approximately $3.7 million compared to $5.6 million for the same period in 2024.

The Company reported research and development expenses of $0.8 million for the three months ended March 31, 2025 compared to approximately $2.3 million for the same period in 2024. The decrease was primarily due to our decision to suspend our HSCT-TMA clinical stage program with nomacopan in May 2024.

General and administrative expenses were approximately $2.7 million for the three months ended March 31, 2025 compared to approximately $3.7 million for the same period in 2024. The decrease was primarily due to (i) decreases in legal and professional fees (primarily related to the Merger) and (ii) a decrease in directors’ and officers’ insurance.

As of March 31, 2025, the Company had cash of approximately $2.6 million. The net proceeds from the Company’s March 2025 offering, after deducting placement agent fees and other offering expenses, were approximately $6.0 million, of which $4.0 million was received in April 2025.

Tsingke Showcases at AACR 2025 — Advancing Global Cancer Research with Integrated Synthesis Solutions

On May 15, 2025 Tsingke Biotech reported that it has proudly participated in the AACR (Free AACR Whitepaper) Annual Meeting 2025, held this April in Chicago, USA (Press release, Tsingke Biotech, MAY 15, 2025, View Source;advancing-global-cancer-research-with-integrated-synthesis-solutions-302456314.html [SID1234653204]). As one of the world’s premier events in oncology research, AACR (Free AACR Whitepaper) gathered global leaders from academia and industry to explore the future of cancer diagnostics and therapeutics.

At Booth #3255, Tsingke presented its comprehensive synthesis and expression service portfolio, offering essential support from early-stage discovery to preclinical development. Our platform drew attention for its capabilities in gene synthesis, oligonucleotide production, mRNA technologies, and protein expression.

What Tsingke Offers for Oncology Research

High-Throughput Nucleic Acid Synthesis
Custom DNA/RNA oligos, siRNA, ASO
Complex modifications: LNA, phosphorothioate, dual-labeled probes
qPCR and NGS-grade primer/probe production
mRNA Synthesis & Raw Materials Supply
IVT template synthesis with 5’/3′ modifications and Poly(A) tails
High-purity enzymes, capping reagents, modified nucleotides, and buffers
Ready to support LNP formulation workflows
Protein Expression & Antibody Tools
Gene synthesis, vector construction, and expression validation
Fast-turnaround expression services in HEK293/CHO systems
Custom R&D Support for Novel Therapies
sgRNA libraries, gene editing tools, and RNAi constructs
Building a Global Synthesis Engine for Cancer Innovation

Tsingke is committed to enabling biotech companies, CDMOs, and academic researchers with a one-stop synthesis platform—from molecular design and synthesis to expression and functional validation. By supporting critical steps in target discovery, validation, and preclinical development, we help accelerate the path from lab to clinic.

FDA Grants Regenerative Medicine Advanced Therapy Designation for BrainChild Bio’s B7-H3 CAR T-cell Therapy for Incurable Pediatric Brain Tumors

On May 15, 2025 BrainChild Bio, Inc., a clinical-stage biotechnology company developing CAR T-cell therapies to treat tumors in the central nervous system (CNS), reported that the investigational B7-H3 targeting autologous CAR T-cell therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor (Press release, BrainChild Bio, MAY 15, 2025, View Source [SID1234653203]).

The use of a regenerative medicine, specifically a CAR T-cell therapy, offers the potential to overcome barriers for other drug modalities to be effective in addressing DIPG, including the precarious location of the DIPG tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains intact during tumor progression. BrainChild Bio has designed BCB-276 to be administered by locoregional delivery of targeted CAR T-cells directly into the cerebrospinal fluid, permitting infused CAR T-cells to directly access the tumor bed, using an indwelling reservoir-catheter device.

This approach to administering an autologous B7-H3 CAR T-cell therapy has been successfully implemented and resulted in the promising overall survival benefit in patients with brain tumors observed in the BrainChild-03 Phase 1 trial (NCT04185038), conducted by BrainChild Bio’s academic partner, Seattle Children’s Research Institute, and recently published in Nature Medicine.

"We are very pleased to now also receive RMAT designation, less than one month after being granted Breakthrough Therapy designation from FDA for our lead CAR T therapy, BCB-276, for the treatment of DIPG. Receiving designations from two independent reviews within FDA further validates the positive CAR-T clinical results achieved by our team to date and the urgent need for a treatment for DIPG," stated Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio. "Our team is keenly focused on initiating the pivotal Phase 2 trial by the end of this year and look forward to continuing to work with the FDA on an accelerated path forward to bring potential new CAR-T treatments for CNS brain tumors in children and adults."

FDA grants RMAT designation to investigational regenerative medicine therapies, including cell therapies, that are aimed at treating serious or life-threatening diseases have shown preliminary clinical evidence that the drug has the potential to address unmet medical needs for the disease. Investigational medicines with RMAT are provided intensive interactions with the FDA during the product candidate’s development process in addition to being eligible for rolling submission and priority review of the marketing application.

"It’s gratifying to see another important benchmark reached in our work to combat pediatric brain cancer," said Dr. Jeff Sperring, Chief Executive Officer at Seattle Children’s. "Our research is the foundation of progress to bring potential therapies to kids as fast as we can – and we’re excited about the possibilities afforded by this designation."

BrainChild Bio is preparing to advance BCB-276 in a Phase 2 multi-center, pivotal registration trial to support a potential Biologics License Application (BLA) to the FDA for the treatment of children and young adults with DIPG. This clinical plan is based on alignment between BrainChild Bio and FDA at a Type B meeting in late 2024.

About Diffuse Intrinsic Pontine Glioma (DIPG) and Application of CAR T-cell Therapies

Diffuse intrinsic pontine glioma (DIPG) is a primary high-grade brain tumor that arises in the pons and is uniformly fatal. DIPG affects approximately 300 children per year in the U.S. with the majority of diagnoses made in children between 5 and 10 years of age. Current standard-of-care treatment remains limited to palliative focal radiation therapy which results in a median overall survival of only about 11 months from diagnosis.1 Barriers to effective therapies for DIPG include the precarious location of the tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains relatively intact during tumor progression preventing therapies from gaining access to the cancer.

The barriers to effective therapies for DIPG can be effectively overcome by the locoregional delivery of appropriately targeted CAR T-cells directly into the cerebrospinal fluid via intracerebroventricular (ICV) dosing with an indwelling reservoir-catheter device. This enables the potential for extensive exposure of the pons to cerebrospinal fluid flow from the ventricular system, thus permitting infused CAR T-cells to directly access the tumor bed. This also allows for repetitive infusions of CAR T-cells to replenish the tumor bed, offering the potential for more durable and sustained efficacy. Additionally, with the blood brain barrier intact, this therapeutic approach can also minimize any on-target, off-tumor toxicities resulting from systemic exposure of CAR T-cells.

Rocket Pharmaceuticals Presents Preliminary Data from Phase 1 Clinical Trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy at 28th Annual Meeting of the American Society of Gene and Cell Therapy

On May 15, 2025 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported preliminary data from the Phase 1 clinical trial of RP-A601 for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM) (Press release, Rocket Pharmaceuticals, MAY 15, 2025, View Source [SID1234653202]). RP-A601 showed a well-tolerated safety profile with no dose-limiting toxicities, increased PKP2 protein expression, and preliminary indications of improvement or stabilization in arrhythmia burden, heart function, and quality of life in all three patients followed for up to 12 months. Results were presented today as a late-breaking oral presentation at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) and will be discussed on a company webinar today at 4:30 p.m. ET.

"Preliminary data from the Phase 1 study of RP-A601 for PKP2-ACM are highly encouraging, signaling potential clinical benefit along with a generally well-tolerated safety profile," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "These initial results represent the second gene therapy from our AAV cardiovascular portfolio to show positive clinical data, propelling us one step closer towards our mission of delivering potentially curative treatments to patients with rare and devastating heart conditions."

The preliminary safety and efficacy of RP-A601 were evaluated in a single-arm, open-label, multi-center Phase 1 study in three patients with PKP2-ACM who received a dose of 8×1013 GC/kg. Initial data from the Phase 1 study (safety cut-off May 6, 2025; efficacy cut-off April 2025) showed that RP-A601 was generally well-tolerated with no dose-limiting toxicities observed in all patients followed for up to 12 months. Most treatment emergent adverse events were mild/moderate in severity and self-limited with only one patient experiencing severe adverse events which resolved without clinical sequelae within two months post-treatment, believed to be associated with the immunomodulatory regimen.

Cardiac biopsies showed RP-A601 increased PKP2 protein expression in all three patients. In the patients with low baseline PKP2 expression (N=2), improvements in PKP2 protein expression relative to total cell protein were approximately 110% and 398%, respectively, from baseline to six months follow-up. In addition, RP-A601 increased protein expression and promoted desmosomal localization of PKP2, Desmocollin-2, and Cadherin-2 in all three patients.

Preliminary indications of improvement or stabilization were observed in arrhythmia burden, heart function, and quality of life. Patients in the Phase 1 trial also demonstrated:

Improvements/stabilization in right ventricular (RV) function.
All patients showed normal RV systolic function at most recent follow-up.
Improvements in quality-of-life as assessed through the Kansas City Cardiomyopathy Questionnaire (KCCQ) and New York Heart Association (NYHA) Class.
Clinical improvement in KCCQ-12 score of 34-41 points (≥5 point increases considered clinically meaningful in adults) and improved NYHA class (from Class II at baseline to Class I; NYHA Class I reflects the absence of clinical signs of heart failure) in both patients followed beyond six months.
Decreased/stabilized ventricular ectopy (premature ventricular contractions [PVC], non-sustained ventricular tachycardia [NSVT]) on rhythm monitoring.
Patients experienced a 9% to 63% reduction in PVCs from baseline evaluated six to 12 months post-treatment.
The only patient who had baseline NSVTs saw a decrease from five to zero NSVT episodes per 24-hour period at six months post-treatment.
Decreased/stabilized T-wave inversions on electrocardiogram (ECG).
One patient saw a reduction in ECG leads with T-wave inversions (precordial and inferior ECG) from six to two at six months post-treatment.
Investor Webcast Information
Company management will host a webinar today, May 15, 2025, at 4:30 p.m. ET. To join the investor webinar, please register at View Source The webcast is available under "Events" in the Investors section of the Company’s website at: View Source The webcast replay will be available on the Rocket website upon completion of the event.

About RP-A601
RP-A601 is an investigational gene therapy for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). RP-A601 consists of a recombinant adeno-associated serotype rh74 capsid containing a functional version of the human PKP2 transgene (AAVrh74.PKP2) which is administered as a single intravenous (IV) infusion. RP-A601 is being investigated as a one-time, potentially curative gene therapy treatment that may improve survival and quality of life for patients affected by PKP2-ACM. Rocket holds Fast Track designation in the U.S. and Orphan Drug designation in the U.S. and Europe for the program.

About PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
PKP2-ACM is an inherited heart disease caused by mutations in the PKP2 gene and characterized by life-threatening ventricular arrhythmias, cardiac structural abnormalities, and sudden cardiac death. PKP2-ACM affects approximately 50,000 adults and children in the U.S. and Europe. Patients living with PKP2-ACM have an urgent unmet medical need, as current medical, implantable cardioverter defibrillator (ICD), and ablation therapies do not consistently prevent disease progression or arrhythmia recurrence, are associated with significant morbidity including inappropriate shocks and device and procedure-related complications, and do not address the underlying pathophysiology or genetic mutation.