On June 12, 2025 Schrödinger, Inc. (Nasdaq: SDGR) reported encouraging initial clinical data from its ongoing Phase 1, open-label, dose-escalation study of SGR-1505 in patients with relapsed/refractory B-cell malignancies (Press release, Schrodinger, JUN 12, 2025, View Source [SID1234653869]). SGR-1505 was observed to be safe, well tolerated, and clinically active, with responses observed in multiple histologies, including in patients with chronic lymphocytic leukemia (CLL) and Waldenström macroglobulinemia (WM). These data are being presented in a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress.

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"We are very encouraged by the initial results from our Phase 1 study in patients with relapsed/refractory B-cell malignancies. The data presented today, coupled with the differentiated preclinical and safety profiles observed in our previously completed study in healthy volunteers, further increases our conviction about the potential for SGR-1505 to be a best-in-class therapy," said Margaret Dugan, M.D., chief medical officer at Schrödinger. "Dose escalation is complete, and we look forward to discussing these results and our proposed recommended Phase 2 dose with the FDA later this year."

"Despite recent advances in the treatment of B-cell malignancies, resistance to currently available therapies eventually results in treatment failure and disease progression for many patients," said Stephen Spurgeon, M.D., Associate Professor of Medicine, Oregon Health and Science University, and an investigator for the clinical study. "We know that MALT1 plays a critical role in key signaling pathways that drive cancer cell survival and proliferation, making it a promising target for a broad range of B-cell malignancies. Although these data are from an early-phase study, they suggest SGR-1505 demonstrates on-target activity resulting in potential clinical benefit. I look forward to seeing additional data as the study progresses, including response data in patients with aggressive histologies."

"The positive data reported today represent a key milestone for Schrödinger and follow the clinical successes of programs advanced by collaboration partners and companies we have co-founded," said Karen Akinsanya, Ph.D., president, head of therapeutics R&D and chief strategy officer, partnerships at Schrödinger. "These data reinforce the power of Schrödinger’s platform to enable the rapid design of differentiated molecules and the impact that our computational approach can have on a drug discovery and development program."

Major Takeaways from the Study

As of the data cut-off date, May 13, 2025, 49 patients were enrolled and evaluable for safety, including 18 patients with CLL/SLL, nine with diffuse large B-cell lymphoma (DLBCL), six with Waldenström macroglobulinemia (WM), and five with marginal zone lymphoma (MZL).
Patients had a median of four (range two-nine) prior lines of therapy, with the most common being Bruton’s tyrosine kinase (BTK) inhibitors (55.1%), BCL-2 inhibitors (18.4%) and BTK+BCL-2 inhibitors (18.4%).
SGR-1505 was well-tolerated with no dose-limiting toxicities or deaths due to treatment-emergent adverse events (TEAEs). Forty three percent of patients (n=21) experienced ≥ 1 treatment-related adverse event (TRAE), with the most common (≥ 10%) being rash (12%) and fatigue (12%). Ten patients (20%) experienced treatment-emergent serious adverse events (SAEs); one was treatment-related. All blood bilirubin increased TEAEs were asymptomatic, reported in patients with UGT1A1 polymorphisms and none were Grade 4.
Inhibition of IL-2 is a pharmacodynamic biomarker for target engagement and an exploratory endpoint in the study. Preliminary data indicated that SGR-1505 inhibits T-cell derived IL-2 upon ex vivo stimulation achieving the PD target of ~90% inhibition in the majority of PD-evaluable participants treated at ≥ 150 mg QD and all Q12H doses at steady state.
Preliminary efficacy data indicated SGR-1505 was clinically active as a monotherapy in a number of relapsed/refractory B-cell malignancies. Of the 49 participants, 45 patients had at least one follow-up disease assessment or disease progression and were evaluable for preliminary efficacy. The overall response rate (ORR) across all dose levels was 22% (n = 10/45). Thirteen of 49 patients had been on treatment for ≥120 days.
Among patients with indolent disease, 3/17 CLL/SLL, 5/5 WM, and 1/5 MZL patients responded. The responses of the three CLL responders were independently reviewed and confirmed, and two had a partial response (PR) with lymphocytosis (PR-L). Two of three CLL patients with partial responses were double-exposed to BTK and BCL-2 inhibitors, and all WM patients were exposed to BTK inhibitors.
The study recently began enrolling patients with aggressive lymphomas into the 300 mg QD and 100 mg Q12H cohorts. A PR was reported in one of four ABC-DLBCL patients.
Study Design
The Phase 1 dose-escalation study (NCT05544019) assessed SGR-1505 as a monotherapy treatment in patients with relapsed/refractory B-cell malignancies. The primary endpoint is the incidence and severity of adverse events and dose-limiting toxicities. Secondary endpoints include pharmacokinetic and pharmacodynamic measurements as well as objective response rate, duration of response and disease control rate.

EHA Poster Presentation Details
The full abstract (#PS1569) can be found online at www.ehaweb.org.

Poster Title: A Phase 1 study of SGR-1505, an oral, potent, MALT1 inhibitor for relapsed/refractory (R/R) B-cell malignancies, including chronic lymphocytic leukemia/small lymphocytic leukemia (CLL/SLL)
Presentation Date and Time: Saturday, June 14, 2025, 6:30-7:30PM CST (12:30-1:30PM ET)
Location: Poster Session 2

About SGR-1505
SGR-1505 is an oral investigational MALT1 inhibitor being evaluated for the treatment of relapsed/refractory B-cell malignancies. MALT1 plays a central role in key signaling pathways that drive cancer cell survival and proliferation, making its location downstream of BTK in the NF-κB signaling pathway an attractive target for the development of novel therapeutics for a potentially broad range of B-cell malignancies. In preclinical studies, SGR-1505 was observed to be highly potent and selective, and has demonstrated anti-tumor activity in preclinical models both as a monotherapy and in combination with BTK and BCL-2 inhibitors. There is also emerging therapeutic rationale supporting MALT1 inhibition as a potential treatment for inflammatory and autoimmune disorders.

SGR-1505 was designed using Schrödinger’s computational platform at scale and was discovered approximately 10 months after the company started its MALT1 program. Schrödinger believes that SGR-1505 is currently the most advanced MALT1 inhibitor known to be in clinical development and has both first-in-class and best-in-class potential. A Phase 1 study in patients with relapsed/refractory B-cell malignancies is ongoing (NCT05544019).

Webcast and Conference Call Information
Schrödinger will host a conference call on Thursday, June 12, 2025, at 8:00 a.m. ET to review the clinical opportunity for SGR-1505 and review the Phase 1 data presented at EHA (Free EHA Whitepaper). The live webcast can be accessed under "Events & Presentations" in the investors section of Schrödinger’s website, View Source To participate in the live call, please register for the call here. It is recommended that participants register at least 15 minutes in advance of the call. The archived webcast will be available on Schrödinger’s website for approximately 90 days following the event.

On June 12, 2025 BioNTech SE (Nasdaq: BNTX, "BioNTech") and CureVac N.V. (Nasdaq: CVAC, "CureVac") reported that they have entered into a definitive Purchase Agreement pursuant to which BioNTech intends to acquire all of the shares of CureVac, a clinical-stage biotech company developing a novel class of transformative medicines in oncology and infectious diseases based on messenger ribonucleic acid ("mRNA") (Press release, BioNTech, JUN 12, 2025, View Source [SID1234653838]). The all-stock transaction will bring together two highly complementary companies based in Germany and will build on BioNTech’s proven track record and established position in the global mRNA industry.

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With the acquisition, BioNTech aims to strengthen the research, development, manufacturing, and commercialization of investigational mRNA-based cancer immunotherapy. The strategic transaction will complement BioNTech’s capabilities and proprietary technologies in mRNA design, delivery formulations, and mRNA manufacturing. For BioNTech, this transaction marks the next milestone in the execution of its oncology strategy which focuses on two pan-tumor programs, mRNA-based cancer immunotherapy candidates, and BNT327, a PD-L1xVEGF-A bispecific antibody candidate.

Under the terms of the Purchase Agreement, each CureVac share will be exchanged for approx. $5.46 in BioNTech ADSs, resulting in an implied aggregate equity value for CureVac of approx. $1.25 billion (subject to the adjustments described below). The consideration is subject to a collar mechanism, such that if the 10-day volume weighted average price of the BioNTech ADSs ending on the fifth business day prior to the closing of the offer ("VWAP") exceeds $126.55, the exchange ratio would be 0.04318, and if the VWAP is lower than $84.37, the exchange ratio would be 0.06476. Upon closing of the transaction, CureVac shareholders are expected to own between 4% and 6% of BioNTech.

"This transaction is another building block in BioNTech’s oncology strategy and an investment in the future of cancer medicine," said Prof. Ugur Sahin, M.D., CEO and Co-Founder of BioNTech. "We intend to bring together complementary capabilities and leverage technologies with the goal of advancing the development of innovative and transformative cancer treatments and establishing new standards of care for various types of cancer in the coming years."

"To me, this transaction is more than a business decision, it represents a shared commitment to leverage the full potential of mRNA as a disruptive technology to develop transformative therapies with greater scale and speed," said Dr. Alexander Zehnder, CEO of CureVac. "For more than two decades, both companies have operated with related ambitions, often tackling challenges from different angles. This transaction aims at combining complementary scientific capabilities, proprietary technologies, and manufacturing expertise in the mRNA field under one roof."

BioNTech will start preparing an integration plan in alignment with BioNTech’s ongoing group-wide transformation. Following the closing of the transaction, CureVac’s operating subsidiary will become a wholly owned subsidiary of BioNTech. As part of this plan, BioNTech will integrate CureVac’s state-of-the-art research and manufacturing site in Tübingen.

BioNTech’s all-stock acquisition of CureVac is expected to create long-term value for shareholders of both companies, building on BioNTech’s proven track record in mRNA research, development, manufacturing, and commercialization, in particular the COVID-19 vaccine, which was developed in collaboration with Pfizer Inc. and marked the first approved mRNA product in the history of medicine. Based on BioNTech’s strong financial position with €15.9 billion in cash, cash equivalents and security investments as of March 31, 2025, its global presence, late-stage clinical pipeline, and sustained investment in mRNA research across a broad range of solid tumor types, the acquisition positions the company to accelerate and broaden the development of mRNA-based medicines for patients in need.

Following the closing of the exchange offer BioNTech and CureVac will effectuate a corporate reorganization of CureVac and its subsidiaries, resulting in BioNTech owning 100% of CureVac’s business and interests in CureVac and its subsidiaries. As part of this corporate reorganization, CureVac shareholders who do not tender their shares in the exchange offer will receive the same consideration received for each CureVac share tendered in the exchange offer (without interest and subject to applicable withholding taxes). An extraordinary general meeting of CureVac’s shareholders will be convened in connection with the exchange offer to adopt, among other things, certain resolutions relating to the transaction.

The transaction was unanimously approved by both BioNTech’s and CureVac’s management and supervisory boards. The transaction, which is expected to close in 2025, is subject to the satisfaction of customary closing conditions, including a minimum acceptance threshold of at least 80% of CureVac’s shares (which threshold may be reduced to 75% unilaterally by BioNTech under certain circumstances) and required regulatory approvals.

Certain shareholders of CureVac representing 36.76% of CureVac’s shares, including dievini Hopp BioTech holding GmbH & Co. KG and certain of its affiliates and all members of CureVac’s management and supervisory boards, have entered into tender and support agreements, pursuant to which they have agreed, among other things, and subject to the terms and conditions of such agreements, to tender their shares in the exchange offer and to vote in favor of the resolutions relating to the transaction at the CureVac extraordinary general meeting to be held in connection with the transaction. In addition, the German Federal government has confirmed to generally have a positive view on the transaction. BioNTech therefore assumes that Kreditanstalt für Wiederaufbau – which holds 13.32% of the shares in CureVac on behalf of the Federal Republic of Germany – will support the transaction by tendering its shares in CureVac. As a result, BioNTech expects to have contractual commitments to support the transaction from shareholders of CureVac representing a total of 50.08% of CureVac shares towards the 80% minimum condition required under the exchange offer.

Covington & Burling LLP, Hengeler Mueller Partnerschaft von Rechtsanwälten mbB and Loyens & Loeff N.V. served as legal counsel to BioNTech. Skadden, Arps, Slate, Meagher & Flom LLP and NautaDutilh N.V. served as legal counsel to CureVac. PJT Partners served as exclusive financial advisor to BioNTech. Goldman Sachs Bank Europe SE served as exclusive financial advisor to CureVac.

On June 12, 2025 BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported that it will present new clinical data from three cornerstone hematology assets at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress (Press release, BeOne Medicines, JUN 12, 2025, View Sourcenews/beone-medicines-showcases-breakthrough-data-in-cll-and-mcl-at-eha-2025/b755b055-956b-40e5-8a80-763881ca3f12" target="_blank" title="View Sourcenews/beone-medicines-showcases-breakthrough-data-in-cll-and-mcl-at-eha-2025/b755b055-956b-40e5-8a80-763881ca3f12" rel="nofollow">View Source [SID1234653854]). Four oral presentations highlight the promising clinical activity of BeOne’s next-generation BCL2 inhibitor sonrotoclax, BTK protein degrader BGB-16673, and the backbone of our hematology franchise, BTK inhibitor BRUKINSA (zanubrutinib), which has the broadest label globally of any approved BTK inhibitor. These data reinforce the company’s strategic vision to redefine the standard of care for B-cell malignancies.

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"The data presented at EHA (Free EHA Whitepaper) 2025 underscore the strength of BeOne’s comprehensive hematology pipeline, built on the success of BRUKINSA, the only BTK inhibitor to demonstrate superior progression-free survival over ibrutinib in a Phase 3 trial,1" said Lai Wang, Ph.D. Global Head of R&D at BeOne. "With our potentially best-in-class BCL2 inhibitor, sonrotoclax, and first-in-class BTK degrader, BGB-16673, we are advancing innovative therapies aimed at addressing resistance mechanisms and improving outcomes for patients with B-cell malignancies."

The data presented at EHA (Free EHA Whitepaper) 2025 support the ongoing advancement of sonrotoclax and BGB-16673 into Phase 3 studies and lay the groundwork for BeOne’s first regulatory submissions for these programs. The company’s integrated development approach—anchored in differentiated mechanisms and translational science—positions its programs to address key areas of unmet need in hematologic oncology.

"While existing therapies have improved outcomes in CLL and related malignancies, many patients still relapse or develop resistance and continue to face limited options," said Stephan Stilgenbauer, Professor of Medicine and Medical Director of the Comprehensive Cancer Center Ulm (CCCU), Head of the Early Clinical Trials Unit (ECTU), and Head of the Division of CLL Dept. of Internal Medicine III at Ulm University. "The updated data presented at EHA (Free EHA Whitepaper) underscore the potential of novel approaches, including BTK degradation and BCL2-based combinations, to overcome known mechanisms of resistance and expand treatment options for patients."

Sonrotoclax + BRUKINSA Demonstrates Deep Responses in CLL and MCL

BeOne’s data will highlight the emerging potential of its next-generation assets to address the unmet needs of patients with B-cell malignancies.

Updated results from Phase 1 studies evaluating sonrotoclax in combination with BRUKINSA in patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) and with R/R mantle cell lymphoma (MCL) demonstrated consistent, deep responses and a manageable safety profile. Sonrotoclax is well-positioned to improve key aspects of the BCL2 inhibitor class and has demonstrated robust and durable antitumor activity and a tolerable safety profile across all dose levels.

R/R CLL/SLL (Oral Presentation S159):
Overall response rate (ORR): 96%; complete response (CR): 52% across all dose levels
Undetectable minimal residual disease (uMRD) was achieved in 82% of patients overall, with responses deepening over time
No tumor lysis syndrome (TLS) or febrile neutropenia observed
R/R MCL (Oral Presentation S234):
ORR: 79%; CR rate: 66%, with 84% of responders in ongoing response at data cutoff
No TLS or atrial fibrillation/flutter reported; the most common grade ≥3 adverse event was neutropenia (19.6%)
Robust Early Results in CLL and WM with Potentially First-in-Class BTK Degrader

As the most clinically advanced BTK degrader, BGB-16673 continues to show potential in patients with various hematological malignancies. Updated data from the ongoing Phase 1 CaDAnCe-101 study of BGB-16673 in R/R CLL/SLL and R/R Waldenström macroglobulinemia (WM) showed substantial antitumor activity and a tolerable safety profile across heavily pretreated populations.

R/R CLL/SLL (Oral Presentation S158):
ORR: 84.8% across all dose levels and 93.8% at the recommended phase 2 dose of 200mg
Patients were heavily pretreated, with most previously treated with BTK and BCL2 inhibitors. The median number of prior therapies was four. Responses deepened over time
Grade ≥3 adverse events included neutropenia (24%) and pneumonia (11%); no reported deaths were attributed to study drug
R/R Waldenström Macroglobulinemia (Oral Presentation S231):
ORR: 84.4%; major response rate: 75.0%; very good partial response rate (VGPR): 31.3%
Rapid onset of response (median: 1.0 month), with continued deepening over time
Efficacy observed across genetic backgrounds and prior BTKi exposure
Most common grade ≥3 TEAE was neutropenia/neutrophil count decreased (31%); no reported atrial fibrillation or febrile neutropenia.
BRUKINSA Monotherapy Showed Sustained OS and PFS Benefit

Data from Arm D of the SEQUOIA Phase 3 trial will also be presented at the meeting (Abstract PS1566), demonstrating that treatment with BRUKINSA plus venetoclax has the potential to drive progression-free survival and overall deep and durable responses across the frontline CLL patient spectrum, including patients with high-risk mutational status. SEQUOIA Arm D investigated BRUKINSA plus venetoclax in 114 patients with treatment-naïve (TN) CLL / SLL with or without del(17p) and/or TP53 high-risk mutations. At a median follow-up of 31.2 months, the combination induced a high 24-month progression-free survival (PFS) rate of 92% (95% CI, 85-96%) and an impressive overall response rate (ORR) of 97%. The 24-month overall survival (OS) rate was 96% (95% CI, 90%-98%). Notably, these benefits were observed regardless of del(17p)/TP53 mutational status. The safety profile of BRUKINSA was consistent with the results of prior studies with no new safety signals identified.

Arm C of the SEQUOIA study investigated BRUKINSA monotherapy in patients with TN CLL / SLL and del(17p) mutations, representing the largest prospective cohort of CLL/SLL patients with del(17p), will be presented at EHA (Free EHA Whitepaper) (Abstract: PS1565). At a median follow-up of over 5.5 years (65.8 months), most patients remained progression-free. Notably, at 60 months, 72.2% of patients who received BRUKINSA remained progression-free (95% CI, 62.4, 79.8). When adjusted for the impact of the COVID-19 pandemic, 73.0% of patients in the cohort remained progression-free (95% CI, 63.3, 80.6) at 60 months. The 60-month OS rate was 85.1% (95% CI, 76.9, 90.6) and 87.0% (95% CI, 79.0, 92.1) when adjusted for COVID-19. At the time of data cut-off, the ORR was 97.3%, and 62.2% of patients were still receiving treatment with BRUKINSA. The safety profile of BRUKINSA was consistent with the results of prior studies with no new safety signals identified.

BeOne will host an investor R&D Day on June 26 at 8:30 am ET covering our deep and broad global innovation pipeline and platforms, as well as the Company’s vision, differentiated capabilities, and value creation drivers. The live webcast can be accessed from the investors section of BeOne’s website at View Source, View Source, or View Source An archived replay will be available to investors for 90 days following the event.

About Sonrotoclax (BGB-11417)

Sonrotoclax is designed to block the B-cell lymphoma 2 (BCL2) protein, which is one of several proteins that help cancer cells survive. It is part of a group of drugs called BH3 mimetics, which mimic natural cell death signals. Studies in the lab and during early drug development have shown that sonrotoclax is a potent and specific inhibitor of BCL2 with a short half-life and no accumulation. Sonrotoclax has shown promising clinical activity across a range of B-cell malignancies, and more than 1,900 patients have been enrolled to date across the global development program. The U.S. Food and Drug Administration (FDA) granted sonrotoclax Fast Track Designation for the treatment of adult patients with mantle cell lymphoma (MCL) and Waldenström macroglobulinemia (WM).

About BGB-16673

BGB-16673 is an orally available Bruton’s tyrosine kinase (BTK) targeting protein degrader from BeOne’s chimeric degradation activation compound (CDAC) platform. BGB-16673 is designed to promote the degradation, or breakdown, of both wildtype and mutant forms of BTK, including those that commonly result in resistance to BTK inhibitors in patients who experience progressive disease. BGB-16673 is the most advanced BTK protein degrader in the clinic, with an extensive global clinical development program. The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to BGB-16673 for the treatment of adult patients with relapsed or refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL), and adult patients with R/R mantle cell lymphoma (MCL).

About BRUKINSA (zanubrutinib)

BRUKINSA is an orally available, small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared with other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues.

BRUKINSA has the broadest label globally of any BTK inhibitor and is the only BTK inhibitor to provide the flexibility of once or twice daily dosing. Additionally, BRUKINSA is also the only BTK inhibitor to demonstrate PFS superiority to a first-generation BTK inhibitor in a Phase 3 study.

The global BRUKINSA clinical development program includes about 7,100 patients enrolled in 30 countries and regions across more than 35 trials. BRUKINSA is approved for at least one indication in more than 75 markets, and more than 200,000 patients have been treated globally.

Select Important Safety Information

Serious adverse reactions, including fatal events, have occurred with BRUKINSA, including hemorrhage, infections, cytopenias, second primary malignancies, cardiac arrhythmias, and hepatotoxicity (including drug-induced liver injury).

In the pooled safety population (N=1729), the most common adverse reactions (≥30%), including laboratory abnormalities, in patients who received BRUKINSA were neutrophil count decreased (51%), platelet count decreased (41%), upper respiratory tract infection (38%), hemorrhage (32%), and musculoskeletal pain (31%).

Please see full U.S. Prescribing Information including U.S. Patient Information.

The information provided in this press release is intended for a global audience. Product indications vary by region.

On June 12, 2025 Samsung Bioepis Co., Ltd. ("Samsung Bioepis") reported the long-term safety data of EPYSQLI (eculizumab; SB12), a biosimilar to Soliris1, in paroxysmal nocturnal hemoglobinuria (PNH) at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress 2025 held at Milan, Italy from June 12 to 15 (Press release, Samsung Bioepis, JUN 12, 2025, View Source [SID1234653870]).

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EPYSQLI was approved by the European Commission (EC) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in May 2023 and March 2024, respectively2.

The study assessed the long-term safety of EPYSQLI in PNH patients by evaluating the consistency of safety outcomes, particularly serious adverse events (SAEs) between the initial 52-week Phase 3 study period and the extended treatment (ET) period, spanning from 52 weeks to up to 158 weeks. The same maintenance dose of 900 mg EPYSQLI was administered every 2 weeks. A total of 46 patients from the Phase 3 study received ET. During ET, seven patients (15.2%) experienced a total of 14 SAEs with no occurrence of fatal cases, and all patients fully recovered without permanently discontinuing the treatment. The exposure-adjusted event rate (EAER) was comparable between initial 52-week period and ET period (EAER were 0.13 and 0.17, respectively), and there was no statistically difference between initial 52-week and ET period in EAER (p-value = 0.76). The study is consistent with the findings of the Phase 3 study with no newly identified safety signals and no fatal cases occurred throughout the entire treatment period with all SAEs resolving completely.

Details of the abstract are as follows3:

Abstract title: Long-Term Safety of SB12 in Paroxysmal Nocturnal Hemoglobinuria: Up to 2-year Extension Treatment Safety Data
Abstract number: PB2811
Type: Publication Only
Session title: Bone marrow failure syndromes incl. PNH – Clinical
Author(s): Jun Ho Jang, Siook Baek, Yumin Baek, Jinah Jung, Ciprian Tomuleasa, Hanna Oliynyk, Theerin Lanamtieng, Soo Min Lim
Besides approval by the EC, EPYSQLI is also approved by the U.S. Food and Drug Administration (FDA) and Korea’s Ministry of Food and Drug Safety (MFDS) as a biosimilar to Soliris. In countries where EPYSQLI is approved and available, EPYSQLI may not be prescribed and/or dispensed for its unapproved other indications for which Soliris is approved.

About EPYSQLI (Eculizumab Biosimilar) in the European Union (EU)
EPYSQLI is indicated in adults and children for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). EPYSQLI is not approved for and should not be used for the treatment of generalised myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). EPYSQLI must be administered by a healthcare professional and under the supervision of a physician experienced in the management of patients with hematological disorders or renal disorders.

EPYSQLI EU Important Safety Information
The EU Summary of Product Characteristics for EPYSQLI includes the following Special warning and Precautions:

Meningococcal Infection
Due to its mechanism of action, the use of eculizumab increases the patient’s susceptibility to meningococcal infection (Neisseria meningitidis). Meningococcal disease due to any serogroup may occur. To reduce the risk of infection, all patients must be vaccinated at least 2 weeks prior to receiving eculizumab unless the risk of delaying eculizumab therapy outweighs the risks of developing a meningococcal infection. Patients who initiate eculizumab treatment less than 2 weeks after receiving a tetravalent meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Vaccines against serogroups A, C, Y, W 135 are recommended in preventing the commonly pathogenic meningococcal serogroups. Vaccine against serogroup B where available is also recommended. Patients must receive vaccination according to current national vaccination guidelines for vaccination use.

Vaccination may further activate complement. As a result, patients with complement-mediated diseases, including PNH and aHUS, may experience increased signs and symptoms of their underlying disease, such as haemolysis (PNH) or TMA (aHUS). Therefore, patients should be closely monitored for disease symptoms after recommended vaccination.

Vaccination may not be sufficient to prevent meningococcal infection. Consideration should be given to official guidance on the appropriate use of antibacterial agents. Cases of serious or fatal meningococcal infections have been reported in eculizumab-treated patients. Sepsis is a common presentation of meningococcal infections in patients treated with eculizumab. All patients should be monitored for early signs of meningococcal infection, evaluated immediately if infection is suspected, and treated with appropriate antibiotics if necessary. Patients should be informed of these signs and symptoms and steps taken to seek medical care immediately.

Other Systemic Infections
Patients may have increased susceptibility to other type of serious infections, especially with Neisseria and encapsulated bacteria.

Infusion Reactions
Administration of eculizumab may result in infusion reactions or immunogenicity that could cause allergic or hypersensitivity reactions (including anaphylaxis). Eculizumab administration should be interrupted in all patients experiencing severe infusion reactions and appropriate medical therapy administered.

Anticoagulant therapy
Treatment with eculizumab should not alter anticoagulant management.

PNH Laboratory Monitoring
PNH patients should be monitored for signs and symptoms of intravascular haemolysis, including serum lactate dehydrogenase (LDH) levels, and may require dose adjustment within the recommended 14±2 day dosing schedule during the maintenance phase (up to every 12 days).

aHUS Laboratory Monitoring
aHUS patients receiving eculizumab therapy should be monitored for thrombotic microangiopathy by measuring platelet counts, serum LDH and serum creatinine, and may require dose adjustment within the recommended 14±2 day dosing schedule during the maintenance phase (up to every 12 days).

Treatment Discontinuation for PNH
If patients discontinue treatment with eculizumab they should be closely monitored for signs and symptoms of serious intravascular haemolysis.

Treatment Discontinuation for aHUS
If aHUS patients discontinue treatment with eculizumab, they should be monitored closely for signs and symptoms of severe thrombotic microangiopathy complications.

The most common adverse reaction observed with eculizumab treatment in clinical trials was headache, (occurred mostly in the initial phase of dosing), and the most serious adverse reaction was found to be meningococcal infection.

Refer to the Summary of Product Characteristics for EPYSQLI’s full safety information.

On June 12, 2025 GO2 for Lung Cancer (GO2) and the Addario Lung Cancer Medical Institute (ALCMI) reported to have launched SUCCEED, a landmark, remote-participation, observational clinical study in small cell lung cancer (SCLC), one of the deadliest and most under-researched cancers (Press release, Bonnie J Addario Lung Cancer Foundation, JUN 12, 2025, View Source [SID1234653839]).

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Despite comprising up to 15% of all lung cancer cases, SCLC has seen minimal treatment advancements in nearly four decades. Even with the introduction of immunotherapy to standard chemotherapy treatment, survival gains have been modest, extending median overall survival by just two months. With a 5-year survival rate of just 6%, the need for deeper biological understanding and new treatment approaches has never been more urgent.

One of the key innovations of the SUCCEED study is its remote, decentralized design, allowing patients with SCLC across the United States to enroll and participate from home. This inclusive approach is critical, as many SCLC patients are older, live farther from the center where they receive treatment, and face multiple co-morbidities. These reasons, and the fact that SCLC progresses quickly, have historically limited their participation in clinical research.

"The SUCCEED study is an important step forward for people with small cell lung cancer, who have long faced limited treatment options and barriers to participating in research," said GO2 for Lung Cancer President and CEO, Laurie Ambrose. "By bringing the study directly to patients, SUCCEED makes it easier for them to get involved, have their voices heard, and contribute to research that could lead to better treatments and, ultimately, more time with their loved ones."

The SUCCEED study will test whether this remote, direct-to-patient approach works well with this population while collecting blood samples from people with SCLC. The samples will be bio-banked and could potentially support future studies aimed at finding new treatments.

"In order to best serve patients with small cell lung cancer, the SUCCEED study will seek to learn about patient preferences and goals," said Principal Investigator Christine Lovly, MD, PhD, from Vanderbilt University Medical Center. "We will do so in a unique way, bringing the study to the patient so that patients can join while being close to home, hopefully making it easier for them to take part."

"SCLC research has long been left behind," said ALCMI Board Member and Chief Operating Officer Richard Erwin. "The SUCCEED study is designed to change that by harnessing real-world data and samples from patients often excluded from traditional clinical trials."

For information on study eligibility and participation, visit here.