Evaluation of the effectiveness of trehalose on inflammatory factors, cardiac biomarkers and clinical outcomes after coronary artery bypass graft (CABG) surgery: A double-blind randomized controlled clinical trial

Start Date06 Aug 2025

Sponsor / Collaborator

Isfahan University of Medical Sciences

IRCT20250317065107N1

/ RecruitingPhase 2IIT

To assess the effect of trehalose on cognitive abillity in patients with Lewy body dementia and evaluation of key proteins in the protein interaction network of LBD

Start Date22 May 2025

Sponsor / Collaborator

Mashhad University of Medical Sciences

100 Clinical Results associated with Trehalose

100 Translational Medicine associated with Trehalose

100 Patents (Medical) associated with Trehalose

15,936

Literatures (Medical) associated with Trehalose

01 Dec 2026·JOURNAL OF MOLECULAR MEDICINE-JMM

Traumatic brain injury-related ferroptosis: current perspectives

Review

Author: Min, Yucong ; Zhang, JianKang ; Liu, Fashun ; Wang, Hao ; Pan, Jizhou ; Zhu, Rongkun

Traumatic brain injury (TBI) is a leading cause of mortality and long-term disability worldwide, with secondary injuries amplifying neuronal damage through mechanisms such as ferroptosis-a regulated iron-dependent cell death driven by lipid peroxidation. This review synthesizes current evidence on ferroptosis in TBI, highlighting its core pathways: (1) iron metabolism dysregulation (e.g., TfR1 upregulation, ferritin suppression), (2) lipid peroxidation (mediated by ACSL4/LPCAT3/PUFA oxidation), and (3) glutathione depletion (via System X_c^-/GSH/GPX4 axis disruption). Ferroptosis intersects with mitochondrial dysfunction, ER stress, and neuroinflammation, exacerbating neuronal death. The hippocampus, vulnerable to post-TBI damage, shows elevated ferroptosis markers (e.g., Tyro3, NRF2/p53 pathways). Iron chelators (e.g., deferoxamine), antioxidants (e.g., ferrostatin-1, liproxstatin-1), and natural compounds (e.g., melatonin, trehalose) demonstrate neuroprotection by mitigating oxidative stress and restoring metabolic balance. Emerging approaches include ncRNA therapies (e.g., miR-212-5p) and stem cell interventions targeting ferroptosis-related genes. Despite progress, key gaps persist in understanding ferroptosis crosstalk with apoptosis/pyroptosis, optimizing drug delivery (e.g., nanoparticle carriers), and validating biomarkers for clinical translation. Targeting ferroptosis offers a promising avenue for TBI treatment, but further research is needed to refine therapeutic specificity and integrate these strategies into clinical practice.

01 Apr 2026·CARBOHYDRATE RESEARCH

Oligosaccharides from Scorzonera yildirimlii and S. zorkunensis, and their potential antimicrobial, enzyme inhibition, cytotoxic effect, and in silico study

Article

Author: Yıldırmış, Sermet ; Bozdal, Gozde ; Şahin, Büşra ; Bozdeveci, Arif ; Coşkunçelebi, Kamil ; Seyhan, Gökçe ; Aksel, Ahmet Buğra ; Arıcı, Şeyma ; Ali, Yasemin Altun ; Yaylı, Nurettin ; Korkmaz, Büşra ; Makbul, Serdar

In this study, the phytochemical investigation of Scorzonera yildirimlii and S. zorkunensis led to the isolation and identification of eight new oligosaccharide constituents (1-8). Their structures were elucidated through comprehensive spectroscopic analysis (NMR, FT-IR, and mass spectrometry) and comparison with literature data. The structure of isolated compounds were O-α-D-Glcp-(1 → 2)-O-β-D-Fruf-(3 → 1)-O-myo-inositol (1), O-α-D-Glcp-(1 → 2)-O-β-D-Fruf-(3 → 1)-O-epi-inositol (2), O-β-D-Fruf-(2 → 6)-O-α-D-Glcp-(1 → 1)-O-β-D-Glcp (3), O-β-D-Fruf-(2 → 6)-O-α-D-Glcp-(1 → 1)-O-β-D-Galp (4), O-β-D-Fruf-(2 → 6)-O-β-D-Fruf-(2 → 6)-O-α-D-Glcp-(1 → 1)-O-β-D-Glcp (5), O-β-D-Fruf-(2 → 6)-[O-β-D-Glcp-(1 → 4)]-O-α-D-Glcp-(1 → 1)-O-β-D-Glcp (6), O-β-D-Fruf-(2 → 6)-[O-β-D-Glcp-(1 → 4)]-3-OCH₃-O-α-D-Glcp-(1 → 1)-O-β-D-Glcp (7), and O-β-D-Fruf-(2 → 6)-[O-β-D-Glcp-(1 → 4)]-O-α-D-Glcp-(1 → 4)-O-α-D-Glcp-(1 → 2)-O-β-D-Fruf (8). The antimicrobial activities of all the isolated compounds (1-8) were investigated against six microorganisms. Compounds 2 and 3 were effective against Mycobacterium smegmatis at minimum inhibitory concentrations (MICs) of 3.13 μg/mL and 3.44 μg/mL, respectively, compared with streptomycin (MIC = 4.0 μg/mL). The enzyme-inhibitory properties of the isolated compounds (1-8) were evaluated against tyrosinase and α-glucosidase. Among the tested compounds, 4 (39.78 ± 10.29 %) exhibited inhibitory activity against tyrosinase, while compound 2 (68.04 ± 4.68 %) exhibited inhibitory activity against α-glucosidase. In silico studies of 1-8 showed the best predicted binding affinities for the Trehalose-LpqY complex in the range of -11.088 kcal/mol to -14.297 kcal/mol, compared to streptomycin (-6.638 kcal/mol). Compounds 1-8 exhibited almost no cytotoxic activity against L-929 cells, further supporting their potential for therapeutic use.

01 Mar 2026·FOOD CHEMISTRY

Trehalose substitution for sucrose improves freeze-thaw stability of Penaeus vannamei surimi: Focus on molecular interactions and cross-linking properties

Article

Author: Shi, Wenzheng ; Zeng, Wei ; Shi, Jian ; Liu, Sixi ; Xu, Tong ; Zhang, Hansong

This study investigated the substitution of sucrose with trehalose (2 %, 3 %, 4 %) to enhance the freeze-thaw stability of Penaeus vannamei surimi. Results exhibited a dose-dependent improvement in freeze-thaw stability with increasing trehalose concentration. Following five freeze-thaw cycles, 4 % trehalose outperformed 4 % sucrose in protecting myofibrillar protein, reflected in 5.3 % higher salt solubility and 7.8 % higher total sulfhydryl content. The improved protein stability enhanced gel cross-linking, evidenced by a 15.1 % higher gel strength and a denser microstructure. Molecular docking revealed that trehalose formed seven bonds within the myosin pocket, whereas sucrose formed only four superficial bonds and a steric hindrance. The stronger trehalose-protein interactions contributed to cryoprotection by both protecting protein structure and inhibiting ice crystal growth, thereby facilitating the formation of a superior gel network. This study provides a foundation for developing high-quality frozen shrimp surimi.

News (Medical) associated with Trehalose

19 Jan 2026

·www.biospace.com

JPM26: Beam, Cabaletta, More Zero In on Regulatory Alignment After Tumultuous Year

iStock, wildpixel As biotechs faced investors at the J.P. Morgan Healthcare Conference this week, they emphasized agreement with the FDA on clinical trial design and regulatory pathways to approval. Atara, meanwhile, lamented the agency’s “complete reversal of position” after its therapy for a rare surgical complication was rejected. Last year, a clutch of biotechs experienced regulatory whiplash as previous FDA guidance was seemingly reversed, leading to rejections or delayed timelines. This trend continued into 2026, as Atara Biotherapeutics on Monday announced an FDA rejection it described as “a complete reversal of position.” Amid this regulatory uncertainty, several biotechs wanted to make it crystal clear during the J.P. Morgan Healthcare Conference this week that their alignment with the regulator was rock solid. Beam Therapeutics, Cabaletta Bio, PTC Therapeutics and BridgeBio all communicated alignment with the FDA on regulatory pathways or next steps for their programs, according to analyst notes dispersed throughout the week as companies provided updates at JPM26. H.C. Wainwright analysts highlighted regulatory alignment as a key theme throughout the meeting. “Regulatory alignment is compressing timelines and improving visibility to value inflections,” the firm wrote in a Wednesday morning note. Nevertheless, Atara’s Monday news demonstrates that biotech is not out of the woods of FDA uncertainty just yet. In its complete response letter, for the proposed use of Ebvallo in treating a serious post-transplant complication related to the Epstein-Barr virus, the FDA said Atara’s single-arm ALLELE study was “no longer considered to be adequate to provide evidence of effectiveness for accelerated approval,” according to Atara’s announcement. The company further noted that the agency’s “new position is contrary to the FDA’s prior guidance to Atara, the FDA’s alignment with Atara on the clinical trial data set, and the acceptance of the trial design as a single arm study as relevant for this patient population at BLA submission.” The chief executives of Replimune and Capricor shared similar sentiments after rejections last year. Speaking on a BioSpace webinar in November, BMO Capital Markets analyst Evan Seigerman spoke to this trend, specifically pointing to Replimune, which in July was hit with a surprise rejection for its advanced melanoma candidate RP1. “Today, they had one set of guidance from FDA with regard to clinical trial design, or how they could have a unique trial design . . . and then all of a sudden, there’s a 180,” Seigerman said of the company’s experience, “and it throws everything into a tailspin.” FDA FDA Reversals Send UniQure, Biohaven, Capricor, More Into a ‘Tailspin’ Since July, several biotechs have been forced to pivot as previous agreements with the FDA around evidence required for approval were reversed, a phenomenon that, according to experts, could portend a more restrictive regulator. November 24, 2025 · 7 min read · Heather McKenzie Beam, Cabaletta, PTC and BridgeBio are hoping for better luck. Here is how their regulatory pathways are lining up as they embark on 2026. Beaming Up to an Accelerated Pathway With AATD Alignment Beam Therapeutics, which is taking key steps this year to transition to a commercial-ready company, announced that it has aligned with the FDA on a potential accelerated approval pathway for BEAM-302 in alpha-1 antitrypsin deficiency (AATD). The investigational gene editor showed positive early results in a Phase I/II trial in March 2025, demonstrating the first-ever genetic correction in the rare disease, which is characterized by no or insufficient expression of the AAT protein. The accelerated pathway for BEAM-302 will be based on AAT biomarkers evaluated over 12 months, Beam said in a press release issued Jan. 11, two days before the company’s presentation at JPM26. The company plans to enroll around 50 additional patients who will be treated with the optimal biologic dose in an expansion of the Phase I/II study. “We believe this defined regulatory path for BEAM-302 significantly de-risks development,” Wainwright analysts wrote on Wednesday. Beam’s autologous cell therapy for sickle cell disease (SCD), risto-cel, is even closer to the regulatory finish line, with the company expecting to biologics license application by the end of 2026. Cabaletta Reaches Consensus With FDA on Registrational Lupus Cohorts Meanwhile, Cabaletta Bio has reached alignment with the FDA on new registrational cohort designs for its CD19 CAR T cell therapy rese-cel in Phase I/II trials in systemic lupus erythematosus (SLE) and lupus nephritis (LN). However, the plan involves the use of single-arm cohorts, which have been a point of contention in some of the FDA’s seemingly inconsistent guidance. Under the agreed-upon design, rese-cel will be evaluated in two independent, single-arm cohorts, one comprising patients with non-renal SLE and the other made up of patients with LN, according to Cabaletta’s Jan. 12 press release . Each cohort will include approximately 25 patients. Cabaletta is even further along with rese-cel in dermatomyositis (DM) and antisynthetase syndrome (ASyS), as the biotech launched these “FDA-aligned” registrational cohorts in December. “In our view, Cabaletta continues to execute on advancing rese-cel in multiple indications, as highlighted by the initiation” of this registrational trial, William Blair analysts said in a Monday note. And Cabaletta is expecting complete Phase I/II data from rese-cel in SLE, systemic sclerosis (SSc) and myasthenia gravis (MG) in the first half of 2026, according to its announcement. “While we believe the company has several opportunities in 2026 for value creation, the most critical will likely be potential regulatory alignment in SSc and MG and updated clinical data from its trials without preconditioning,” William Blair said on Monday. PTC Gives Positive Huntington’s Update After Mixed Phase II Reaction PTC Therapeutics had a mixed 2025 from a regulatory standpoint. In July, the rare disease–focused biotech won FDA approval for Sephience for children and adults with phenylketonuria (PKU). However, in August the FDA rejected the company’s Friedreich’s ataxia candidate, vatiquinone, saying in its CRL that “substantial evidence of efficacy was not demonstrated.” In a press release issued ahead of CEO Matthew Klein’s JPM presentation on Jan. 12, Klein lauded the approval and “strong start to the global launch” of Sephience. Earlier in the pipeline, PTC is developing votoplam for Huntington’s disease. On Monday, the company announced it had aligned with the FDA on design of a global Phase III trial, which it plans to initiate in the first half of this year. During the end-of-Phase II meeting, held during the fourth quarter last year, the FDA also confirmed its openness to a potential accelerated approval pathway for the asset, PTC said, given the significant unmet need in Huntington’s. Neuroscience Deep Dive: Huntington’s at a Crossroads Recent breakthroughs and three decades of progress in treating Huntington’s disease January 6, 2026 · 2 min read · Heather McKenzie PTC518, a small molecule that includes a premature stop codon in the huntingtin (HTT) mRNA, lowered blood HTT protein levels by 23% at the lowest dose in the Phase II PIVOT-HD trial, PTC reported in May 2025. Earlier-stage patients also saw functional improvements. While the trial met its primary endpoint, analysts were less than impressed. Willliam Blair at the time noted that patients with later-stage disease did not show any functional benefit on PTC518 and protein reduction in cerebrospinal fluid was less robust in these patients. In a note to investors on Monday, William Blair analysts said they “view alignment with the FDA on the trial design for votoplam in Hungtinton’s [sic] disease as positive, and look forward to the company sharing additional details regarding the trial’s design and what endpoints could be used to support accelerated approval.” BridgeBio’s Muscular Dystrophy Drug on Track for Traditional Approval Everything is coming up roses for BridgeBio, which delivered what Truist Securities in a Monday note called a “ surprise beat ” for transthyretin amyloid cardiomyopathy (ATTR-CM) drug Attruby in Q4, 2025. The $146 million in revenue, reported during the company’s JPM presentation on Jan. 12, represented a growth of 35% quarter-on-quarter, according to Truist. On the regulatory front, BridgeBio reported that two end-of-Phase II meetings with the FDA had taken place, for encaleret in chronic hypoparathyroidism and ribitol in limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). For ribitol, BridgeBio suggested that the FDA indicated alignment on pursuing traditional full approval in LGMD2I/R9 based on positive functional data at the trial’s interim analysis, according to Truist. The company plans to submit new drug applications for both assets to the FDA in the first half of 2026. .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to ClinicaSpace! 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Clinical ResultPhase 3Phase 2Drug Approval

10 Nov 2025

·www.globenewswire.com

Cabaletta Bio Reports Third Quarter 2025 Financial Results and Provides Business Update

Rese-cel data presented at multiple medical meetings demonstrated potentially transformative, drug-free clinical responses with a favorable safety profile for autoimmune patients supporting outpatient use All myositis patients in the Phase 1/2 DM/ASyS cohort with sufficient follow-up who would have met key criteria for the registrational cohort met the registrational, 16-week primary endpoint Planned BLA submission for rese-cel in 2027 based on 14-patient, single-arm DM/ASyS registrational cohort initiating enrollment this quarter within the RESET-Myositis™ trial FDA alignment on additional registrational cohort designs for RESET-SSc™ and RESET-SLE™ anticipated by year-end 2025 PHILADELPHIA, Nov. 10, 2025 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today reported financial results for the third quarter ended September 30, 2025, and provided a business update. “Our team continued to execute with discipline and precision to extend our leadership through the RESET™ clinical development program. Rapid enrollment has resulted in multiple clinical data presentations highlighting rese-cel’s ability to deliver drug-free, transformative clinical responses for patients across multiple autoimmune diseases,” said Steven Nichtberger, M.D., Chief Executive Officer of Cabaletta. “In addition, the early no preconditioning data from our initial dose cohort support our plan to evaluate the efficacy and durability of rese-cel in lupus and other autoimmune patients using a single, weight-based dose without preconditioning.” Recent Operational Highlights and Upcoming Anticipated Milestones Rese-cel: Rese-cel (resecabtagene autoleucel, formerly CABA-201) is an investigational, autologous CAR T cell therapy engineered with a fully human CD19 binder and a 4-1BB co-stimulatory domain, designed specifically for the treatment of autoimmune diseases. Administered as a single, weight-based infusion, rese-cel is intended to transiently and deeply deplete CD19-positive cells, with the goal of resetting the immune system and achieving durable clinical responses without the need for chronic therapy. Cabaletta is evaluating rese-cel in the RESET (REstoring SElf-Tolerance) clinical development program, which includes multiple ongoing company-sponsored trials across a diverse and growing range of autoimmune diseases in rheumatology, neurology and dermatology. Clinical Development Presented positive new and longer-term clinical data from 32 patients across four autoimmune trials: At multiple medical meetings in October 2025, Cabaletta shared encouraging results from patients treated with rese-cel in myositis, systemic sclerosis, lupus, and myasthenia gravis, including full Phase 1/2 data from RESET-Myositis and preliminary data from RESET-SSc, RESET-SLE, and RESET-MG™ trials. These findings highlight rese-cel’s potential to deliver transformative, drug-free responses for patients with a favorable safety profile that can support outpatient use.Initial, low-dose no preconditioning data presented and expansion of approach into lupus: At the 2025 European Society of Gene & Cell Therapy Annual Congress, Cabaletta presented initial data from the RESET-PV™ trial evaluating a low dose of rese-cel without preconditioning in patients with pemphigus vulgaris, demonstrating complete B cell depletion in 2 of 3 patients along with early clinical responses with a generally well-tolerated profile. Based on these data, Cabaletta is expanding patient enrollment in the RESET-PV trial at the current dose, with the potential to evaluate higher doses, as warranted. In addition, Cabaletta is incorporating a new dose-escalation cohort in the RESET-SLE trial to evaluate rese-cel without preconditioning given the clinical responses observed in lupus following complete B cell depletion after administration of rese-cel with preconditioning.Initiation of myositis registrational DM/ASyS cohort anticipated by year-end 2025: Consistent with the previously announced alignment with the U.S. Food and Drug Administration (FDA) on registrational cohort design, which is based on several interactions, including direct review and feedback on the registrational trial protocol in August 2025, we are aligned on the dermatomyositis (DM) and antisynthetase syndrome (ASyS) registrational cohort with a 16-week primary endpoint of moderate or major total improvement score response while off immunomodulators and on no or low-dose steroids. The planned size of 14 patients for the registrational cohort is based on the assumed treatment effect of rese-cel in DM/ASyS patients and an estimated background rate. The estimated background rate will be determined from an external myositis patient registry, as aligned with the FDA, and will include patients with similar inclusion criteria as those in the registrational DM/ASyS cohort. In the Phase 1/2 DM/ASyS cohort, all patients with sufficient follow-up who would have met key criteria for the registrational cohort met the registrational primary endpoint. If successful, data from the DM/ASyS cohort will be used in part to support the Company’s first projected Biologics License Application (BLA) submission for rese-cel in myositis in 2027.Additional clinical data from RESET trials expected throughout 2026: On December 6, 2025, Cabaletta will present a poster titled “Mechanistic basis of the acute safety profile of rese-cel, an autologous CD19-CAR T, in patients with autoimmune disease treated in four ongoing phase 1/2 clinical trials” at the 67th American Society of Hematology Annual Meeting and Exposition, taking place in Orlando, FL. In addition, Cabaletta plans to present complete Phase 1/2 data from the RESET-SSc and RESET-SLE trials in the first half of 2026 and from the RESET-MG trial in the second half of 2026. The Company also anticipates clinical data on rese-cel without preconditioning from the initial RESET-SLE dose-escalation cohort and additional dosing from the RESET-PV trial in 2026. Regulatory New PRIME, RMAT and Fast Track regulatory designations granted by EMA and FDA: The Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) granted PRIME scheme access for rese-cel for the treatment of myositis during its September 2025 meeting. Through PRIME, the EMA offers early and proactive support to medicine developers to optimize the generation of robust data on a medicine’s benefits and risks and enable accelerated assessment of medicines applications. In addition, the FDA has granted new regulatory designations to rese-cel, including Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of systemic lupus erythematosus and lupus nephritis and Fast Track Designation for generalized myasthenia gravis.FDA alignment on additional registrational cohort designs expected through 2026: Cabaletta expects to align with the FDA on key registrational design elements for the RESET-SSc and RESET-SLE trials by year-end 2025 and for the RESET-MG trial in the first half of 2026. Subject to clinical data and regulatory alignment on registrational cohort design elements for these trials, the Company expects to initiate enrollment in registrational cohorts in 2026. Corporate Updates Appointment of Chief Commercial Officer: In October 2025, Steve Gavel was appointed Chief Commercial Officer. Mr. Gavel brings highly relevant CAR T experience from Legend Biotech where he led the launch and commercialization of CARVYKTI® from 2018 until 2025. In his role at Cabaletta, he will lead all aspects of global commercial strategy and execution for rese-cel as well as potential future pipeline opportunities. Third Quarter 2025 Financial Results Research and development expenses were $39.8 million for the three months ended September 30, 2025, compared to $26.3 million for the same period in 2024.General and administrative expenses were $6.8 million for the three months ended September 30, 2025, compared to $6.8 million for the same period in 2024.As of September 30, 2025, Cabaletta had cash, cash equivalents and short-term investments of $159.9 million, compared to $164.0 million as of December 31, 2024. The Company expects that its cash position as of September 30, 2025, will enable it to fund its operating plan into the second half of 2026. About Cabaletta BioCabaletta Bio (Nasdaq: CABA) is a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases. The CABA™ platform encompasses two complementary strategies which aim to advance the discovery and development of engineered T cell therapies with the potential to become deep and durable, perhaps curative, treatments for a broad range of autoimmune diseases. The lead CARTA (Chimeric Antigen Receptor T cells for Autoimmunity) strategy is prioritizing the development of rese-cel, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy. Rese-cel is currently being evaluated in the RESET™ (REstoring SElf-Tolerance) clinical development program spanning multiple therapeutic areas, including rheumatology, neurology and dermatology. Cabaletta Bio’s headquarters and labs are located in Philadelphia, PA. For more information, please visit www.cabalettabio.com and connect with us on LinkedIn. Forward-Looking StatementsThis press release contains “forward-looking statements” of Cabaletta Bio within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including without limitation, express or implied statements regarding: Cabaletta’s business plans and objectives as a whole; Cabaletta’s ability to realize its vision of launching the first curative targeted cell therapy designed specifically for patients with autoimmune diseases; Cabaletta’s ability to successfully complete research and further development and commercialization of its drug candidates in current or future indications, including the timing and results of Cabaletta’s clinical trials and its ability to conduct and complete clinical trials; expectation that clinical results will support rese-cel’s safety and activity profile; statements regarding the timing of interactions with regulatory authorities, including such authorities’ review of safety information from Cabaletta’s ongoing clinical trials and alignment with regulatory authorities on potential registrational pathway for rese-cel; Cabaletta’s ability to leverage its emerging clinical data and its efficient development strategy; Cabaletta’s belief that its new data demonstrates rese-cel’s ability to deliver drug-free, transformative clinical responses for patients across multiple autoimmune diseases and that the early no preconditioning data from its initial dose cohort has opened new opportunities to potentially treat autoimmune patients using a single weight-based dose of rese-cel without preconditioning; Cabaletta’s expectations that the milestones it has achieved underscores the potential of rese-cel to redefine treatment paradigms and bring drug-free remission to patients with high unmet need and also that its findings highlight rese-cel’s potential to deliver transformative, drug-free responses for patients with a favorable safety profile that can support outpatient use; Cabaletta’s ability to capitalize on and potential benefits resulting from its research and translational insights; the clinical significance of the clinical data read-out at upcoming scientific meetings and timing thereof; Cabaletta’s expectations around the potential success and therapeutic benefits of rese-cel, including its belief that rese-cel has the potential to reset the immune system and achieve durable clinical responses without the need for chronic therapy; the Company’s advancement of separate Phase 1/2 clinical trials of rese-cel in patients with SLE, myositis, SSc, gMG and PV and advancement RESET-MS trial, including updates related to status, enrollment, safety data, efficiency of clinical trial design and timing of data read-outs or otherwise; Cabaletta’s ability to initiate the myositis registrational trial and timing thereof; Cabaletta’s plans to expand patient enrollment in the RESET-PV trial at the current dose, with the potential to evaluate higher doses; Cabaletta’s expectations regarding the new dose-escalation cohort in the RESET-SLE trial to evaluate rese-cel without preconditioning based on the clinical responses observed in lupus; Cabaletta’s plans to initiate enrollment in the registrational DM/ASyS cohort in 2025; Cabaletta’s plans to announce additional clinical data from RESET trials throughout 2026; Cabaletta’s expectations around alignment with FDA on key registrational design elements for the RESET-SSc and RESET-SLE trials by year-end 2025 and for the RESET-MG trial in the first half of 2026; Cabaletta’s expectations to initiate enrollment in registrational cohorts in 2026; Cabaletta’s plans to submit a BLA for rese-cel in myositis in 2027 and obtain regulatory approval from the FDA and other regulatory authorities and Cabaletta’s use of capital, expense and other financial results in the future and its ability to fund operations into the second half of 2026. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to regulatory filings and potential clearance; the risk that signs of biologic activity or persistence may not inform long-term results; Cabaletta’s ability to demonstrate sufficient evidence of safety, efficacy and tolerability in its preclinical studies and clinical trials of rese-cel; the risk that the results observed with the similarly-designed construct employed in academic publications, including due to the dosing regimen, are not indicative of the results we seek to achieve with rese-cel; risks that results from one program may not translate to results for another program; risks that modifications to trial design or approach may not have the intended benefits and that the trial design may need to be further modified; risks related to clinical trial site activation, delays in enrollment generally or enrollment rates that are lower than expected; delays related to assessment of clinical trial results; risks related to unexpected safety or efficacy data observed during clinical studies; risks related to volatile market and economic conditions and public health crises; Cabaletta’s ability to retain and recognize the intended incentives conferred by Orphan Drug Designation and Fast Track Designation or other designations for its product candidates, as applicable; risks related to Cabaletta’s ability to protect and maintain its intellectual property position; risks related to fostering and maintaining successful relationships with Cabaletta’s collaboration and manufacturing partners; uncertainties related to the initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Cabaletta’s product candidates will not be successfully developed and/or commercialized; and the risk that the initial or interim results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Cabaletta’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Cabaletta’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Cabaletta’s other subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Cabaletta undertakes no duty to update this information unless required by law. CABALETTA BIO, INC.SELECTED FINANCIAL DATA(unaudited; in thousands, except share and per share data)Statements of Operations Three Months EndedSeptember 30, Nine Months EndedSeptember 30, 2025 2024 2025 2024 unauditedunauditedOperating expenses: Research and development $39,824 $26,290 $106,480 $71,671 General and administrative 6,764 6,756 23,150 19,685 Total operating expenses 46,588 33,046 129,630 91,356 Loss from operations (46,588) (33,046) (129,630) (91,356)Other income: Interest income 1,808 2,417 4,705 8,078 Interest expense (584) − (1,449) − Other income, net 498 − 437 − Net loss (44,866) (30,629) (125,937) (83,278)Net loss per share of voting and non-voting common stock, basic and diluted $(0.44) $(0.62) $(1.75) $(1.69) Selected Balance Sheet Data September 30,2025 December 31,2024 (unaudited) Cash, cash equivalents and investments $ 159,931 $163,962Total assets 189,759 185,046Total liabilities 50,293 32,711Total stockholders’ equity 139,466 152,335 Contacts:Anup MardaChief Financial Officerinvestors@cabalettabio.com

Fast TrackClinical StudyASHExecutive ChangeCell Therapy

03 Jun 2025

·pipelinereview.com

SELLAS Presents Preclinical Efficacy of SLS009 in ASXL1 Mutated Colorectal Cancer at 2025 ASCO Annual Meeting

– ASCO Presentation Supports SLS009 as a Potential Targeted Therapy for ASXL1 Mutated Colorectal Cancer – – 22,500 New Cases of Colorectal Cancer with High Microsatellite Instability per Year in the US: 55% ASXL1m Frequency – NEW YORK, NY, USA I June 02, 2025 I SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS” or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced preclinical efficacy of SLS009 (tambiciclib) in ASXL1 mutated colorectal cancer lines. The data are featured in a presentation, entitled “ In vitro efficacy of CDK9 inhibitor tambiciclib (SLS009) in ASXL1 mutated colorectal cancer cell lines ” at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 30- June 3, 2025, in Chicago, Illinois. In a panel of cell lines, SLS009 demonstrated potent anti-proliferative activity: “These results provide strong rationale for continued advancement of SLS009 as a potential treatment for ASXL1-mutated cancers,” said Dr. Dragan Cicic, Senior Vice President, Chief Development Officer at SELLAS. “The ability to selectively target ASXL1-driven tumors at concentrations well below the known safety threshold opens the door for tolerable and effective therapy. Based on the findings, we believe that ASXL1 mutation status could serve as a potential biomarker for response to SLS009 inhibition, which may allow us to further refine patient selection and improve outcomes. We look forward to presenting these results at ASCO.” SLS009 is currently being investigated in a Phase 2 open-label, single-arm, multi-center study designed to evaluate the safety, tolerability, and efficacy of SLS009 in combination with venetoclax and azacitidine including AML patients with ASXL1 mutations. Initial clinical safety and efficacy data are available. In addition, the study aims to identify biomarkers for the target patient population and enrichment for further trials. For more information on the study, visit clinicaltrial.gov identifier NCT04588922 . About SELLAS Life Sciences Group, Inc. SELLAS is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS’ lead product candidate, GPS, is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has the potential as a monotherapy and combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing SLS009 (tambiciclib) – potentially the first and best-in-class differentiated small molecule CDK9 inhibitor with reduced toxicity and increased potency compared to other CDK9 inhibitors. Data suggests that SLS009 demonstrated a high response rate in AML patients with unfavorable prognostic factors including ASXL1 mutation, commonly associated with poor prognosis in various myeloid diseases. For more information on SELLAS, please visit www.sellaslifesciences.com. SOURCE: Sellas Life Sciences

Phase 2ASCOClinical Result

100 Deals associated with Trehalose

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KEGG	Wiki	ATC	Drug Bank
-	Trehalose	-	DB12310

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Machado-Joseph Disease	Phase 3	United States	Seelos Therapeutics, Inc.	30 Aug 2022
Machado-Joseph Disease	Phase 3	United States	Fresenius Kabi Norge AS	30 Aug 2022
Machado-Joseph Disease	Phase 3	Australia	Seelos Therapeutics, Inc.	30 Aug 2022
Machado-Joseph Disease	Phase 3	Australia	Fresenius Kabi Norge AS	30 Aug 2022
Machado-Joseph Disease	Phase 3	Brazil	Fresenius Kabi Norge AS	30 Aug 2022
Machado-Joseph Disease	Phase 3	Brazil	Seelos Therapeutics, Inc.	30 Aug 2022
Machado-Joseph Disease	Phase 3	Germany	Fresenius Kabi Norge AS	30 Aug 2022
Machado-Joseph Disease	Phase 3	Germany	Seelos Therapeutics, Inc.	30 Aug 2022
Machado-Joseph Disease	Phase 3	Portugal	Seelos Therapeutics, Inc.	30 Aug 2022
Machado-Joseph Disease	Phase 3	Portugal	Fresenius Kabi Norge AS	30 Aug 2022

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05136885 (CTgov)	Phase 2/3	Amyotrophic Lateral Sclerosis	161	SLS-005 (SLS-005)	ydbwdecbrx(kjboknzylu) = omhqnpzgif btbpclvlzr (dsrplduinf, 0.090) View more	-	28 Aug 2024
NCT05136885 (CTgov)	Phase 2/3	Amyotrophic Lateral Sclerosis	161	Matching Placebo (Matching Placebo)	ydbwdecbrx(kjboknzylu) = jfzehawmuh btbpclvlzr (dsrplduinf, 0.091) View more	-	28 Aug 2024
NCT05136885 (Biospace) Manual	Phase 2/3	Amyotrophic Lateral Sclerosis	-	SLS-005 (IV Trehalose)SLS-005 (IV Trehalose)	idvtcnpgvo(jewnzudcan) = dstqfaxman xginfdgbmn (aezqhlqpru ) View more	Negative	19 Mar 2024
NCT05136885 (Biospace) Manual	Phase 2/3	Amyotrophic Lateral Sclerosis	-	Placebo	idvtcnpgvo(jewnzudcan) = batxdpnddk xginfdgbmn (aezqhlqpru ) View more	Negative	19 Mar 2024
AAN2016	Phase 2	Muscular Dystrophy, Oculopharyngeal	25	Cabaletta (trehalose 9% solution)	csaccpjgck(areljjnmkh) = improvement trend which did not reach significance nodvmkgwzr (dxqswjmpzl ) View more	-	05 Apr 2016
AAN2016	Phase 2	Muscular Dystrophy, Oculopharyngeal	25	Cabaletta (trehalose 9% solution)	rzplkhlkos(ctodreolhq) = qxaqfzscrf myfcxljrhl (amxfruptdv )	-	05 Apr 2016
AAN2015	Phase 2	Muscular Dystrophy, Oculopharyngeal	-	High dose IV trehalose (Cabaletta)	atqbthupjy(wjtyqyifhe) = No drug-related adverse effects were noted, in particular no cardiovascular changes were observed tlxvgdfkjv (zgllacstnd ) View more	Positive	06 Apr 2015

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