This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.

Start Date30 Sep 2025

Sponsor / Collaborator

Inova Health Care Services

NCT06763055

/ RecruitingPhase 3IIT

The Fifth INTEnsive pReventing Secondary Injury in Acute Cerebral Haemorrhage Trial Within ACT-GLOBAL

This is a domain within the ACT-GLOBAL platform trial to compare the effectiveness of early and appropriate pharmacological interventions in acute intracerebral hemorrhage (ICH) to control secondary brain injury. Up to 2000 patients with presumed spontaneous supratentorial intracerebral hemorrhage (ICH) will be followed for 6 months (or death, if prior to 6 months).
Adaptive interim analyses will be used, with statistical triggers to determine if any of the interventions are superior to control. The end of the trial is defined as the date that all participants have completed their 6-month assessment.
A large amount of preclinical data indicates that the outcome from ICH is linked to the detrimental effects of breakdown substances from brain bleeds. However, there remains a lack of compelling evidence supporting the effectiveness of any pharmacological intervention that can mitigate the secondary cerebral injury. The INTERACT domain aims to assess the effectiveness of intravenous deferoxamine and low-dose oral colchicine, both individually and in combination, to standard of care alone, on improving functional outcome in patients with spontaneous supratentorial ICH.
Those patients who meet eligibility criteria will be randomized to receive one of four interventions:
1. No deferoxamine mesylate and no colchicine (labeled as control)
2. Deferoxamine mesylate only: deferoxamine mesylate at a dose of 32mg/kg/day via intravenous infusion immediately (within 1 hour) post-randomization and continue for the following 2 consecutive days.
3. Colchicine only: 0.5mg of oral colchicine daily for 30 consecutive days.
4. Both deferoxamine mesylate and colchicine: deferoxamine mesylate at a dose of 32mg/kg/day via intravenous infusion immediately (within 1 hour) post-randomization and continue for the following 2 consecutive days; plus 0.5mg of oral colchicine daily for 30 consecutive days.

Start Date27 Feb 2025

Sponsor / Collaborator

The George Institute for Global Health

[+1]

NCT06352632

/ RecruitingPhase 3IIT

A Multi-faCtorial, mulTi-arm, Multi-staGe, Randomised, gLOBal Adaptive pLatform Trial for Stroke (ACT-GLOBAL)

Stroke is causing 6.6 million deaths and is a major cause of disability worldwide in 2019. There remains an urgent need for interventions that improve outcomes which can be implemented with wide applicability for stroke. ACT-GLOBAL is a multi-factorial, multi-arm, multi-stage, randomised, global adaptive platform trial for stroke, aiming to identify the treatment/s associated with the highest chance of improving outcome in stroke patients. In ACT-GLOBAL multiple questions will be evaluated simultaneously and sequentially as data accrues and can evaluate interactions between different treatment options.

Start Date26 Sep 2024

Sponsor / Collaborator

The George Institute for Global Health

[+2]

100 Clinical Results associated with Deferoxamine Mesylate

100 Translational Medicine associated with Deferoxamine Mesylate

100 Patents (Medical) associated with Deferoxamine Mesylate

11,422

Literatures (Medical) associated with Deferoxamine Mesylate

01 Dec 2025·CANCER CHEMOTHERAPY AND PHARMACOLOGY

Sulforaphane inhibits multiple myeloma cell-induced osteoclast differentiation and macrophage proliferation by elevating ferroportin1

Article

Author: Zhang, Chang ; Cao, Deliang ; Hu, Wei ; Xia, Jiliang ; Li, Junjun ; Zhao, Hongyan ; He, Fen ; Sun, Jingqi ; Luo, Cong ; Zeng, Xi ; Lu, Zhongwei ; Sun, Weichu

PURPOSE:

Osteolysis is a common complication in patients with multiple myeloma (MM). Our previous studies have demonstrated that MM cells can promote osteoclast differentiation of macrophages. In this study, we explored the effect of sulforaphane (SFN), a natural NRF2 activator found in broccoli, on MM cell-induced osteoclast differentiation.

METHODS:

Conditional medium (CM) derived from MM cells was used to induce osteoclast differentiation, and TRAP staining was performed to examine osteoclast. Gene expression was detected by western blotting or real-time PCR. Cell counting and EdU staining were performed to test macrophage proliferation.

RESULTS:

We showed that the CM of MM cells downregulated the expression of ferroportin1 (Fpn1), the only known iron exporter in vertebrate cells, thereby increasing cellular iron levels in murine macrophage cells RAW264.7. Deferoxamine (DFO), an iron chelator, effectively blocked MM cell CM-induced osteoclast differentiation and macrophage proliferation, suggesting that iron overload played a key role in these cellular events. Subsequent mechanistic investigations revealed that MM cell CM induced osteoclast differentiation and macrophage proliferation by activating the JNK/AP-1/NFATC1 pathway and PI3K/AKT pathway. SFN was found to increase Fpn1 expression, leading to decreased cellular iron levels in RAW264.7 cells activated by MM cell CM. Importantly, the osteoclast differentiation and macrophage proliferation induced by MM cell CM were significantly inhibited by SFN.

CONCLUSION:

Altogether, our findings indicated that SFN inhibits MM cell-induced osteoclast differentiation and macrophage proliferation by elevating FPN1 levels. SFN could be a promising therapeutic strategy for MM-associated osteolysis.

01 Dec 2025·TALANTA

Azo dye-based on-site assay paper kit for rapid and selective naked-eye and smartphone-assisted detection of deferoxamine drug

Article

Author: Koç, Ömer Kaan

Ferric ion {Fe(III)} is an integral part of many structural and functional components in the body and plays critical roles in physiological and pathological processes. High Fe(III) concentrations damage many systems and organs in the body. The most common method used to prevent excessive accumulation of Fe(III) ions is chelation therapy and the most commonly used drug is deferoxamine (DFO). In this study, an azo dye-based test-paper probe was developed for the colorimetric determination of DFO. The bi-dental azo-dye, named DAZS (diazosalicylaldehyde), was synthesized with a 58 % yield as due to the coupling reaction of 4,4'-diaminobibenzyl and salicylaldehyde. The surface of cellulose-based chromatographic papers immersed in DAZS solution was coated with DAZS and peraped DAZS-coated paper (DAZS-paper) was used as a sensor kit. The structure of DAZS, DAZS-paper, and its interaction mechanism with Fe(III) and DFO were elucidated by FTIR, ¹H NMR, ¹³C NMR, STEM, EDX, and AFM techniques. One arm of DAZS, a symmetrical molecule, interacts with the cellulose-based chromatographic paper, while the other arm remains exposed. This exposed arm interacts with the Fe(III), interrupting the intramolecular charge transfer (ICT) mechanism present in the DAZS molecule, and the color of the DAZS-paper becomes lighter. This interaction, which forms the Fe(III) sensing strategy, is disrupted in the presence of DFO. As a result of its strong chelating feature, DFO forms a chelate complex with Fe(III), enabling the separation of Fe(III) from DAZS. With the separation of the Fe(III), the ICT mechanism is activated again in the free part of DAZS, providing the coloration of DAZS-paper. Although the observed coloration is directly proportional to the concentration of DFO, color analysis was performed with a smartphone via the "colorimeter" application. By analyzing the test paper and solution medium with a smartphone, the detection limits (LOD) of DFO were found to be 95.0 nmol L^-1 and 82.0 nmol L^-1, respectively. In the selectivity study conducted with various Fe(III) chelating agents, DFO was determined with high selectivity. It was found that common metal cations and anions did not significantly affect the developed probe. The developed method, which was verified by a chromatographic reference method, can also be easily applied to the commercial drug Desferal and fetal bovine serum containing DFO.

01 Dec 2025·Toxicology reports

Targeting mitochondrial damage and ER stress to inhibit ferroptosis in cadmium-induced nephrotoxicity

Review

Author: Mammadova, Khayala ; Arshad, Muhammad ; Raza, Muhammad ; Fatima, Anmol ; Yousaf, Saba

This review highlights the pivotal roles of autophagy, ferroptosis, and endoplasmic reticulum (ER) stress in mediating cadmium (Cd)-induced nephrotoxicity. Cadmium exposure results in ER stress, which in turn activates major UPR pathways such as IRE1, ATF6, and PERK. By encouraging lipid peroxidation and suppressing cellular antioxidant defence, these mechanisms worsen ferroptosis and produce a feedback mechanism that increases cellular damage. There are two roles of autophagy in Cd-induced ferroptosis, which include its action in reducing cadmium-induced cytotoxicity by breaking down damaged components, and excessive autophagy, namely ferritinophagy, which promotes ferroptosis by iron dysregulation. The rise of mitochondrial ROS (MitoROS) caused by Cd-induced mitochondrial malfunction aids ferroptosis. This, in turn, causes ER stress and autophagy. This implies that focusing on mitochondrial health could be a useful treatment strategy. Effective treatment approaches include autophagy inhibitors like chloroquine, which have been shown to effectively reduce Cd-induced ferroptosis, and promising medicines that suppress ER stress, such as TUDCA. Desferrioxamine and other iron chelators effectively lower lipid peroxidation and iron dysregulation, therefore preventing ferroptotic cell death. Additionally, a multi-targeted treatment plan is suggested that targets iron metabolism, ER stress, and autophagy. In order to create tailored treatments for Cd-induced nephrotoxicity, this review emphasizes the need for additional study into the molecular pathways of Cd-induced ferroptosis, namely the ER stress-autophagy axis. The goal of future research should be to apply these mechanistic insights to clinical settings to enhance public health outcomes and create efficient therapies for renal failure brought on by cadmium toxicity.

News (Medical) associated with Deferoxamine Mesylate

13 Aug 2025

·www.globenewswire.com

BiomX Reports Second Quarter 2025 Financial Results and Provides Program Updates

Positive Phase 2 results for BX211 demonstrated >40% wound size reduction vs. placebo in diabetic foot osteomyelitis patients; Planning underway for potential registrational study Nature Communications publication of new BX004 Phase 1b/2a data demonstrated further ~500-fold (2.7 log₁₀)2 bacterial reduction versus placebo with no detectable emergence of resistance observed, highlighting strength of BiomX platform capabilitiesPhase 2b trial of BX004 in Cystic Fibrosis (CF) has successfully commenced patient dosing; FDA feedback on real-world evidence strategy expected in H2 2025; Company expects to report topline results in Q1 2026 BiomX will host a conference call and webcast today at 8:00 AM ET NESS ZIONA, Israel, Aug. 13, 2025 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX” or the “Company”), a clinical-stage company advancing novel natural and engineered phage therapies targeting specific pathogenic bacteria, today announced financial results for the second quarter ended June 30, 2025, and provided recent clinical and corporate updates. “In the past few months, BiomX achieved several significant milestones that advance our pipeline and further validate our phage therapy platform,” said Jonathan Solomon, Chief Executive Officer of BiomX. “Positive Phase 2 results for BX211 in diabetic foot osteomyelitis demonstrated sustained, statistically significant improvements in wound size reduction, with over 40% reduction in ulcer size compared to placebo, and a favorable safety profile. In parallel, new peer-reviewed data for BX004, published in Nature Communications, reported approximately 500-fold (2.7 log₁₀)2 further bacterial reduction versus placebo and no detectable emergence of resistance, critical evidence supports the strength of our candidate BX004. With BX004’s Phase 2b trial now underway, FDA feedback on our real-world evidence plan expected later this year, and planning for a potential registrational BX211 study in progress, BiomX is positioned for multiple value-driving catalysts over the next 12 months.” Clinical Program Updates BX211 – Phage for the treatment of patients with diabetic foot infections and osteomyelitis (DFI/DFO) associated with Staphylococcus aureus (S. aureus) BiomX reported positive topline results from the Phase 2 trial of BX211 in March 2025 just prior to the start of the second quarter, demonstrating that BX211 was safe and well-tolerated and produced sustained and statistically significant1 percentage area reduction (PAR) of ulcer size (p = 0.046 at week 12; p=0.052 at week 13) with separation from placebo starting at week 7 and a difference greater than 40% by week 10.The Phase 2 trial results also showed that BX211 produced statistically significant improvements in both ulcer depth at week 13 in patients with ulcer depth defined as bone exposure at baseline (p=0.048) and in reducing the expansion of ulcer area (p=0.017) compared to placebo, with all patients receiving standard of care including systemic antibiotic therapy as appropriate over the 12-week treatment period. Planning for a potential registrational study of BX211 is underway, pending feedback from the U.S. Food and Drug Administration (FDA).Following strong Phase 2 results, the Company hosted a Virtual Key Opinion Leader event during the second quarter featuring leading clinical experts in diabetic foot infections and osteomyelitis. The discussion highlighted the clinical significance of the BX211 data, its potential role alongside standard of care, and perspectives on the path to registrational development. A replay of the webcast is available at the following Link, the content of which does not form a part of this press release.The Company is in continued discussions with the U.S. Defense Health Agency regarding next steps for the BX211 program following the positive Phase 2 results. BX004 – Fixed phage cocktail for chronic Pseudomonas aeruginosa (P. aeruginosa) in patients with CF In July 2025, BiomX successfully initiated dosing of patients in its Phase 2b trial of BX004. The Phase 2b trial is a randomized, double-blind, placebo-controlled, multicenter study in approximately 60 CF patients with chronic P. aeruginosa infections. Patients are randomized 2:1 to receive either BX004 or placebo via inhalation twice daily for 8 weeks. The trial is designed to measure multiple efficacy endpoints, including reduction in bacterial burden, improvements in lung function, and enhanced quality of life as measured by validated patient questionnaires.The Company expects to report topline results from the Phase 2b study in the first quarter of 2026.In the second half of 2025, BiomX expects to receive feedback from the FDA regarding the potential investigation and use of real-world evidence linking bacterial reduction to clinical outcomes. Successful alignment could potentially streamline approval pathway.Earlier in July, Nature Communications published new findings from the Company’s Phase 1b/2a trial, showcasing previously unreported antimicrobial efficacy data showing that BX004 achieved a substantially greater improvement of approximately 500-fold (2.7 log₁₀)2 in bacterial reduction compared with placebo in CF patients. The data also highlight that no bacterial resistance to BX004 was detected during the trial, and also detailed in the article are BiomX’s innovative approach to large-scale data analysis in order to optimize bacteriophage cocktails. Second Quarter 2025 Financial Results Cash balance and restricted cash as of June 30, 2025, were $15.2 million, compared to $18 million as of December 31, 2024. The decrease was primarily due to net cash used in operating activities. BiomX estimates its cash, cash equivalents and restricted cash are sufficient to fund its operations into the first quarter of 2026. Research and development expenses, net were $5.0 million for the second quarter of 2025, compared to $6.9 million for the second quarter of 2024. The decrease was primarily driven by: reduced salary expenses from workforce reductions; lower rent expenses following a right-of-use asset impairment in 2024; and increased grant funding from the Medical Technology Enterprise Consortium (under the DHA) and the Israel Innovation Authority. This was partially offset by higher expenses from initiating the Phase 2b clinical trial of the Company’s CF product candidate, BX004. General and administrative expenses were $2.4 million for the second quarter of 2025, compared to $2.8 million for the second quarter of 2024. The decrease was primarily attributed to a reduction in legal and other professional service fees, partially offset by increased share-based compensation expenses. Net loss was $6.0 million for the second quarter of 2025, compared to income of $4.5 million for the second quarter of 2024. The decrease was mainly due to the change in the fair value of warrants issued as part of the Company’s March 2024 financing. Net cash used in operating activities for the six months ended June 30, 2025, was $14.8 million, compared to $22.6 million for the same period in 2024. Conference Call and Webcast Details BiomX will host a conference call and webcast on August 13, 2025, at 8 AM ET to discuss its second quarter 2025 financial results and to provide a corporate update. Conference Callhttps://register-conf.media-server.com/register/BI35d84b315cb147c78290b6b8f1fde487 Webcast Link https://edge.media-server.com/mmc/p/89dfoxpe/ About BX211BX211 is a phage treatment for the treatment of DFO associated with S. aureus. DFO is a bacterial infection of the bone that usually develops from an infected foot ulcer and is a leading cause of amputation in patients with diabetes. In March 2025, BiomX announced positive topline results from the Phase 2 trial in which BX211 was demonstrated to be safe and well-tolerated and patients receiving BX211 exhibited statistically significant1 and sustained reduction of ulcer size (PAR)(p=0.046 at week 12; p=0.052 at week 13), with a separation from placebo starting at week 7 and a difference greater than 40% by week 10. In addition, BX211 also produced statistically significant1 improvements in both ulcer depth at week 13 (in patients with ulcer depth defined as bone at baseline, ulcer depth was classified according to deepest tissue involved as measured by swab) (p=0.048), and in reducing the expansion of ulcer area (p=0.017). Over the 12-week treatment period, all patients (treatment and placebo) were treated in accordance with standard of care, including with systemic antibiotic therapy as appropriate. BiomX is currently planning a potential registrational study pending discussions and feedback from the FDA, and availability of cash resources. About BX004BiomX is developing BX004, a fixed multi-phage cocktail, for the treatment of CF patients with chronic pulmonary infections caused by P. aeruginosa, a main contributor to morbidity and mortality in patients with CF. In February 2023, BiomX announced positive results from Part 1 of the Phase 1b/2a study, showing safety, tolerability, and microbiologic activity. In November 2023, BiomX announced positive topline results from Part 2 of the Phase 1b/2a trial, in which BX004 demonstrated improvement in pulmonary function associated with a reduction in P. aeruginosa burden compared to placebo in a predefined subgroup of patients with reduced lung function (baseline FEV1<70%). BiomX is now enrolling up to approximately 60 patients in a randomized, double blind, placebo-controlled, multi center Phase 2b trial of BX004 in CF patients with chronic P. aeruginosa lung infections. The 8-week study will assess lung function, bacterial load, and quality of life metrics. BX004 has received FDA Fast Track and Orphan Drug Designations. About BiomXBiomX is a clinical-stage company leading the development of natural and engineered phage cocktails and personalized phage treatments designed to target and destroy harmful bacteria for the treatment of chronic diseases with substantial unmet needs. BiomX discovers and validates proprietary bacterial targets and applies its BOLT (“BacteriOphage Lead to Treatment”) platform to customize phage compositions against these targets. For more information, please visit www.biomx.com, the content of which does not form a part of this press release. Safe HarborThis press release contains express or implied “forward-looking statements” within the meaning of the “safe harbor” provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “target,” “believe,” “expect,” “will,” “may,” “anticipate,” “estimate,” “would,” “positioned,” “future,” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. For example, when BiomX refers to its anticipated timing for reporting results for its clinical assets as well as the design of clinical trials thereof, future catalysts, potential timing and outcome of expected feedback from the FDA and discussions with the U.S. Defense Health Agency, the potential safety, efficacy and toleration of BX004 and BX211, the potential benefits of BX004 and BX211, future clinical development of BX004 and BX211, including conducting a registrational trial in BX211, the potential of its candidates to address the substantial unmet needs of patients, and the estimates of the sufficiency of its cash, cash equivalents and short-term deposits, it is using forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on BiomX management’s current beliefs, expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of BiomX’s control. These risks and uncertainties include, but are not limited to, changes in applicable laws or regulations; the possibility that BiomX may be adversely affected by other economic, business, and/or competitive factors, including risks inherent in pharmaceutical research and development, such as: adverse results in BiomX’s drug discovery, preclinical and clinical development activities, the risk that the results of preclinical studies and early clinical trials may not be replicated in later clinical trials, BiomX’s ability to enroll patients in its clinical trials, and the risk that any of its clinical trials may not commence, continue or be completed on time, or at all; decisions made by the FDA, and other regulatory authorities; decisions made by investigational review boards at clinical trial sites and publication review bodies with respect to our development candidates; BiomX’s ability to obtain, maintain and enforce intellectual property rights for its platform and development candidates; its potential dependence on collaboration partners; competition; uncertainties as to the sufficiency of BiomX’s cash resources to fund its planned activities for the periods anticipated and BiomX’s ability to manage unplanned cash requirements; and general economic and market conditions. Therefore, investors should not rely on any of these forward-looking statements and should review the risks and uncertainties described under the caption “Risk Factors” in BiomX’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (the “SEC”) on March 25, 2025, and additional disclosures BiomX makes in its other filings with the SEC, which are available on the SEC’s website at www.sec.gov. Forward-looking statements are made as of the date of this press release, and except as provided by law BiomX expressly disclaims any obligation or undertaking to update forward-looking statements. Contacts:BiomX, Inc.Ben CohenHead Corporate Communicationsbenc@biomx.com 1 All p-values described in this release are non-adjusted2 A 2.7 log₁₀ reduction represents a 10^2.7 = ~500-fold reduction in bacterial load, which equates to approximately 99.8% reduction. BIOMX INC.CONDENSED CONSOLIDATED BALANCE SHEETS(USD in thousands, except share and per share data)(unaudited) As of June 30, 2025 December 31, 2024 ASSETS Current assets Cash and cash equivalents 14,046 16,856 Restricted cash 979 958 Other current assets 1,610 2,706 Total current assets 16,635 20,520 Non-current assets Non-current restricted cash 161 161 Operating lease right-of-use assets 4,959 5,457 Property and equipment, net 4,245 5,045 In-process Research and development asset 12,050 12,050 Total non-current assets 21,415 22,713 38,050 43,233 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities Trade accounts payable 2,178 1,882Current portion of lease liabilities 1,233 1,130Other accounts payable 2,851 5,255Total current liabilities 6,262 8,267 Non-current liabilities Operating lease liabilities, net of current portion 8,143 8,454Other liabilities 80 77Warrants 4,405 2,287Total non-current liabilities 12,628 10,818 Commitments and Contingencies (Note 6) Stockholders’ equity Preferred Stock, $0.0001 par value; Authorized – 1,000,000 shares as of June 30, 2025 and December 31, 2024. Issued and outstanding- 147,735 as of June 30, 2025 and December 31, 2024. 18,645 18,645Common Stock, $0.0001 par value; Authorized – 750,000,000 shares as of June 30, 2025 and December 31, 2024. Issued and outstanding- 26,443,257 shares as of June 30, 2025 and 18,176,661 shares as of December 31, 2024. 7 6 Additional paid in capital 194,901 186,194Accumulated deficit 194,393)) (180,697Total stockholders’ equity 19,160 24,148 38,050 43,233 BIOMX INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(USD in thousands, except share and per share data)(unaudited) Three Months EndedJune 30, Six Months EndedJune 30, 2025 2024 20252024 Research and development (“R&D”) expenses, net 5,014 6,897 10,26411,002General and administrative expenses 2,419 2,828 4,9255,508 Operating loss 7,433 9,725 15,18916,510 Other expenses (income) 70 (2,017) 76(2,105)Interest expenses 5 13 10863Income from change in fair value of warrants 1,498)( (11,868) (2,412)(3,858)Finance expense (income), net 25 (329) 8301,436Loss (income) before tax 6,035 (4,476) 13,69312,846 Tax expenses 2 5 310 Net loss (income) 6,037 (4,471) 13,69612,856 Basic loss (earnings) per share of Common Stock 0.19 (0.14) 0.501.95Diluted loss per share of Common Stock 0.19 0.69 0.501.95 Weighted average number of shares used in computing basic loss (earnings) pershare of Common Stock 31,308,396 6,980,943 27,250,021 6,605,952Weighted average number of shares used in computing diluted loss per share ofCommon Stock 31,308,396 10,750,194 27,250,021 6,605,952

Clinical ResultPhase 2Fast TrackOrphan DrugFinancial Statement

13 Aug 2025

·ir.biomx.com

BiomX Reports Second Quarter 2025 Financial Results and Provides Program Updates

Positive Phase 2 results for BX211 demonstrated >40% wound size reduction vs. placebo in diabetic foot osteomyelitis patients; Planning underway for potential registrational study Nature Communications publication of new BX004 Phase 1b/2a data demonstrated further ~500-fold (2.7 log₁₀)2 bacterial reduction versus placebo with no detectable emergence of resistance observed, highlighting strength of BiomX platform capabilities Phase 2b trial of BX004 in Cystic Fibrosis (CF) has successfully commenced patient dosing; FDA feedback on real-world evidence strategy expected in H2 2025; Company expects to report topline results in Q1 2026 BiomX will host a conference call and webcast today at 8:00 AM ET NESS ZIONA, Israel, Aug. 13, 2025 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX” or the “Company”), a clinical-stage company advancing novel natural and engineered phage therapies targeting specific pathogenic bacteria, today announced financial results for the second quarter ended June 30, 2025, and provided recent clinical and corporate updates. “In the past few months, BiomX achieved several significant milestones that advance our pipeline and further validate our phage therapy platform,” said Jonathan Solomon, Chief Executive Officer of BiomX. “Positive Phase 2 results for BX211 in diabetic foot osteomyelitis demonstrated sustained, statistically significant improvements in wound size reduction, with over 40% reduction in ulcer size compared to placebo, and a favorable safety profile. In parallel, new peer-reviewed data for BX004, published in Nature Communications, reported approximately 500-fold (2.7 log₁₀)2 further bacterial reduction versus placebo and no detectable emergence of resistance, critical evidence supports the strength of our candidate BX004. With BX004’s Phase 2b trial now underway, FDA feedback on our real-world evidence plan expected later this year, and planning for a potential registrational BX211 study in progress, BiomX is positioned for multiple value-driving catalysts over the next 12 months.” Clinical Program Updates BX211 – Phage for the treatment of patients with diabetic foot infections and osteomyelitis (DFI/DFO) associated with Staphylococcus aureus (S. aureus) BiomX reported positive topline results from the Phase 2 trial of BX211 in March 2025 just prior to the start of the second quarter, demonstrating that BX211 was safe and well-tolerated and produced sustained and statistically significant1 percentage area reduction (PAR) of ulcer size (p = 0.046 at week 12; p=0.052 at week 13) with separation from placebo starting at week 7 and a difference greater than 40% by week 10. The Phase 2 trial results also showed that BX211 produced statistically significant improvements in both ulcer depth at week 13 in patients with ulcer depth defined as bone exposure at baseline (p=0.048) and in reducing the expansion of ulcer area (p=0.017) compared to placebo, with all patients receiving standard of care including systemic antibiotic therapy as appropriate over the 12-week treatment period. Planning for a potential registrational study of BX211 is underway, pending feedback from the U.S. Food and Drug Administration (FDA). Following strong Phase 2 results, the Company hosted a Virtual Key Opinion Leader event during the second quarter featuring leading clinical experts in diabetic foot infections and osteomyelitis. The discussion highlighted the clinical significance of the BX211 data, its potential role alongside standard of care, and perspectives on the path to registrational development. A replay of the webcast is available at the following Link, the content of which does not form a part of this press release. The Company is in continued discussions with the U.S. Defense Health Agency regarding next steps for the BX211 program following the positive Phase 2 results. BX004 – Fixed phage cocktail for chronic Pseudomonas aeruginosa (P. aeruginosa) in patients with CF In July 2025, BiomX successfully initiated dosing of patients in its Phase 2b trial of BX004. The Phase 2b trial is a randomized, double-blind, placebo-controlled, multicenter study in approximately 60 CF patients with chronic P. aeruginosa infections. Patients are randomized 2:1 to receive either BX004 or placebo via inhalation twice daily for 8 weeks. The trial is designed to measure multiple efficacy endpoints, including reduction in bacterial burden, improvements in lung function, and enhanced quality of life as measured by validated patient questionnaires. The Company expects to report topline results from the Phase 2b study in the first quarter of 2026. In the second half of 2025, BiomX expects to receive feedback from the FDA regarding the potential investigation and use of real-world evidence linking bacterial reduction to clinical outcomes. Successful alignment could potentially streamline approval pathway. Earlier in July, Nature Communications published new findings from the Company’s Phase 1b/2a trial, showcasing previously unreported antimicrobial efficacy data showing that BX004 achieved a substantially greater improvement of approximately 500-fold (2.7 log₁₀)2 in bacterial reduction compared with placebo in CF patients. The data also highlight that no bacterial resistance to BX004 was detected during the trial, and also detailed in the article are BiomX’s innovative approach to large-scale data analysis in order to optimize bacteriophage cocktails. Second Quarter 2025 Financial Results Cash balance and restricted cash as of June 30, 2025, were $15.2 million, compared to $18 million as of December 31, 2024. The decrease was primarily due to net cash used in operating activities. BiomX estimates its cash, cash equivalents and restricted cash are sufficient to fund its operations into the first quarter of 2026. Research and development expenses, net were $5.0 million for the second quarter of 2025, compared to $6.9 million for the second quarter of 2024. The decrease was primarily driven by: reduced salary expenses from workforce reductions; lower rent expenses following a right-of-use asset impairment in 2024; and increased grant funding from the Medical Technology Enterprise Consortium (under the DHA) and the Israel Innovation Authority. This was partially offset by higher expenses from initiating the Phase 2b clinical trial of the Company’s CF product candidate, BX004. General and administrative expenses were $2.4 million for the second quarter of 2025, compared to $2.8 million for the second quarter of 2024. The decrease was primarily attributed to a reduction in legal and other professional service fees, partially offset by increased share-based compensation expenses. Net loss was $6.0 million for the second quarter of 2025, compared to income of $4.5 million for the second quarter of 2024. The decrease was mainly due to the change in the fair value of warrants issued as part of the Company’s March 2024 financing. Net cash used in operating activities for the six months ended June 30, 2025, was $14.8 million, compared to $22.6 million for the same period in 2024. Conference Call and Webcast Details BiomX will host a conference call and webcast on August 13, 2025, at 8 AM ET to discuss its second quarter 2025 financial results and to provide a corporate update. Conference Call https://register-conf.media-server.com/register/BI35d84b315cb147c78290b6b8f1fde487 Webcast Link https://edge.media-server.com/mmc/p/89dfoxpe/ About BX211 BX211 is a phage treatment for the treatment of DFO associated with S. aureus. DFO is a bacterial infection of the bone that usually develops from an infected foot ulcer and is a leading cause of amputation in patients with diabetes. In March 2025, BiomX announced positive topline results from the Phase 2 trial in which BX211 was demonstrated to be safe and well-tolerated and patients receiving BX211 exhibited statistically significant1 and sustained reduction of ulcer size (PAR)(p=0.046 at week 12; p=0.052 at week 13), with a separation from placebo starting at week 7 and a difference greater than 40% by week 10. In addition, BX211 also produced statistically significant1 improvements in both ulcer depth at week 13 (in patients with ulcer depth defined as bone at baseline, ulcer depth was classified according to deepest tissue involved as measured by swab) (p=0.048), and in reducing the expansion of ulcer area (p=0.017). Over the 12-week treatment period, all patients (treatment and placebo) were treated in accordance with standard of care, including with systemic antibiotic therapy as appropriate. BiomX is currently planning a potential registrational study pending discussions and feedback from the FDA, and availability of cash resources. About BX004 BiomX is developing BX004, a fixed multi-phage cocktail, for the treatment of CF patients with chronic pulmonary infections caused by P. aeruginosa, a main contributor to morbidity and mortality in patients with CF. In February 2023, BiomX announced positive results from Part 1 of the Phase 1b/2a study, showing safety, tolerability, and microbiologic activity. In November 2023, BiomX announced positive topline results from Part 2 of the Phase 1b/2a trial, in which BX004 demonstrated improvement in pulmonary function associated with a reduction in P. aeruginosa burden compared to placebo in a predefined subgroup of patients with reduced lung function (baseline FEV1<70%). BiomX is now enrolling up to approximately 60 patients in a randomized, double blind, placebo-controlled, multi center Phase 2b trial of BX004 in CF patients with chronic P. aeruginosa lung infections. The 8-week study will assess lung function, bacterial load, and quality of life metrics. BX004 has received FDA Fast Track and Orphan Drug Designations. About BiomX BiomX is a clinical-stage company leading the development of natural and engineered phage cocktails and personalized phage treatments designed to target and destroy harmful bacteria for the treatment of chronic diseases with substantial unmet needs. BiomX discovers and validates proprietary bacterial targets and applies its BOLT (“BacteriOphage Lead to Treatment”) platform to customize phage compositions against these targets. For more information, please visit www.biomx.com, the content of which does not form a part of this press release. Safe Harbor This press release contains express or implied “forward-looking statements” within the meaning of the “safe harbor” provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “target,” “believe,” “expect,” “will,” “may,” “anticipate,” “estimate,” “would,” “positioned,” “future,” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. For example, when BiomX refers to its anticipated timing for reporting results for its clinical assets as well as the design of clinical trials thereof, future catalysts, potential timing and outcome of expected feedback from the FDA and discussions with the U.S. Defense Health Agency, the potential safety, efficacy and toleration of BX004 and BX211, the potential benefits of BX004 and BX211, future clinical development of BX004 and BX211, including conducting a registrational trial in BX211, the potential of its candidates to address the substantial unmet needs of patients, and the estimates of the sufficiency of its cash, cash equivalents and short-term deposits, it is using forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on BiomX management’s current beliefs, expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of BiomX’s control. These risks and uncertainties include, but are not limited to, changes in applicable laws or regulations; the possibility that BiomX may be adversely affected by other economic, business, and/or competitive factors, including risks inherent in pharmaceutical research and development, such as: adverse results in BiomX’s drug discovery, preclinical and clinical development activities, the risk that the results of preclinical studies and early clinical trials may not be replicated in later clinical trials, BiomX’s ability to enroll patients in its clinical trials, and the risk that any of its clinical trials may not commence, continue or be completed on time, or at all; decisions made by the FDA, and other regulatory authorities; decisions made by investigational review boards at clinical trial sites and publication review bodies with respect to our development candidates; BiomX’s ability to obtain, maintain and enforce intellectual property rights for its platform and development candidates; its potential dependence on collaboration partners; competition; uncertainties as to the sufficiency of BiomX’s cash resources to fund its planned activities for the periods anticipated and BiomX’s ability to manage unplanned cash requirements; and general economic and market conditions. Therefore, investors should not rely on any of these forward-looking statements and should review the risks and uncertainties described under the caption “Risk Factors” in BiomX’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (the “SEC”) on March 25, 2025, and additional disclosures BiomX makes in its other filings with the SEC, which are available on the SEC’s website at www.sec.gov. Forward-looking statements are made as of the date of this press release, and except as provided by law BiomX expressly disclaims any obligation or undertaking to update forward-looking statements. Contacts: BiomX, Inc. Ben Cohen Head Corporate Communications benc@biomx.com 1 All p-values described in this release are non-adjusted 2 A 2.7 log₁₀ reduction represents a 10^2.7 = ~500-fold reduction in bacterial load, which equates to approximately 99.8% reduction. BIOMX INC. CONDENSED CONSOLIDATED BALANCE SHEETS (USD in thousands, except share and per share data) (unaudited) BIOMX INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (USD in thousands, except share and per share data) (unaudited) Released August 13, 2025

Clinical ResultPhase 2Fast TrackOrphan DrugFinancial Statement

15 May 2025

·ir.biomx.com

BiomX Reports First Quarter 2025 Financial Results and Provides Business and Program Updates

In March 2025, BiomX announced positive topline results of the phase 2 trial evaluating BX211 for the treatment of diabetic foot osteomyelitis (DFO) BX004 Phase 2b study in Cystic Fibrosis (CF) on track to report topline results in Q1 2026 In April 2025, shareholders approved exercise of warrants issued in $12 million in financings announced in February 2025; funds expected to provide runway through topline Phase 2b results for BX004 trial The Company will host a conference call and webcast today at 2:00 PM ET NESS ZIONA, Israel, May 15, 2025 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX” or the “Company”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced financial results for the first quarter ended March 31, 2025, and provided program and business updates. “The first quarter of 2025 was a period of exceptional progress for BiomX, both on the corporate front and across our clinical pipeline,” said Jonathan Solomon, Chief Executive Officer of BiomX. “The strength of the positive Phase 2 results from our BX211 program in diabetic foot osteomyelitis exceeded our expectations and received resounding endorsement from key opinion leaders and industry experts. These positive results represent a significant milestone for the field of bacteriophage therapy and further reinforce our platform’s potential to address serious, resistant infections. Notably, the BX211 program has been supported to date by approximately $40 million in non-dilutive funding from the U.S. Defense Health Agency (DHA), which has identified an urgent need for new treatments to combat antibiotic-resistant infections in current and future conflict environments. The work received DHA and Department of Navy funding under an Other Transaction Authority (OTA) award through the Medical Technology Enterprise Consortium (MTEC) and managed by the Naval Medical Research Command (NMRC) – Naval Advanced Medical Development (NAMD). We are currently in discussions regarding potential next steps in partnership with the DHA to further advance this program. Looking ahead, we remain focused on our next major catalyst, which is the anticipated Phase 2b readout of BX004 in cystic fibrosis in the first quarter of next year, supported by the recent financings we secured to advance this program and extend our operational runway.” Clinical Program Updates BX211 – phage for the treatment of DFO associated with Staphylococcus aureus (S. aureus) In March 2025, BiomX announced positive results from the Company’s DFO Adaptive Novel Care Evaluation (DANCE™) Phase 2 trial evaluating its BX211 phage treatment for DFO associated with Staphylococcus aureus (S. aureus). Key highlights from the Phase 2 trial results included: BX211 was found to be safe and well-tolerated. BX211 produced sustained and statistically significant1 Percentage Area Reduction (PAR) of ulcer size (p = 0.046 at week 12; p=0.052 at week 13), with a separation from placebo (standard of care) starting at week 7 and a difference greater than 40% by week 10. Compared to placebo, BX211 also produced statistically significant1 improvements in both ulcer depth at week 13 (in patients with ulcer depth defined as bone at baseline) (p=0.048), and in reducing the expansion of ulcer area (p=0.017), compared to placebo. BX211 demonstrated favorable trends compared to placebo across several additional clinical parameters, including: proportion of visits with no clinical evidence of infection; evidence of resolving DFO by MRI/X-ray at week 12; proportion of patients with abnormal C-Reactive Protein at baseline that achieved a reduction of CRP of at least 50% at any point in the study; and greater Wagner scale improvement2. Through week 13, BX211 demonstrated comparable efficacy against both Methicillin-susceptible and resistant strains, as well as against high and low biofilm producers—consistent with the orthogonal mechanism of phage therapy to antibiotics and its inherent anti-biofilm capabilities. A key opinion leader (KOL) event discussing the Phase 2 results took place on April 3, 2025. A replay of the webcast is available here, the content of which does not form a part of this press release. Pending feedback from the FDA, BiomX is planning for a Phase 2/3 clinical trial of BX211. BX211 was found to be safe and well-tolerated. BX211 produced sustained and statistically significant1 Percentage Area Reduction (PAR) of ulcer size (p = 0.046 at week 12; p=0.052 at week 13), with a separation from placebo (standard of care) starting at week 7 and a difference greater than 40% by week 10. Compared to placebo, BX211 also produced statistically significant1 improvements in both ulcer depth at week 13 (in patients with ulcer depth defined as bone at baseline) (p=0.048), and in reducing the expansion of ulcer area (p=0.017), compared to placebo. BX211 demonstrated favorable trends compared to placebo across several additional clinical parameters, including: proportion of visits with no clinical evidence of infection; evidence of resolving DFO by MRI/X-ray at week 12; proportion of patients with abnormal C-Reactive Protein at baseline that achieved a reduction of CRP of at least 50% at any point in the study; and greater Wagner scale improvement2. Through week 13, BX211 demonstrated comparable efficacy against both Methicillin-susceptible and resistant strains, as well as against high and low biofilm producers—consistent with the orthogonal mechanism of phage therapy to antibiotics and its inherent anti-biofilm capabilities. A key opinion leader (KOL) event discussing the Phase 2 results took place on April 3, 2025. A replay of the webcast is available here, the content of which does not form a part of this press release. BX004 – fixed phage cocktail for the treatment of CF in patients with chronic pulmonary infections caused by Pseudomonas aeruginosa (P. aeruginosa) During the first quarter, BiomX initiated the exploration and analysis of real-world evidence in people with CF on the relationship between P. aeruginosa reduction and clinical outcomes. Regulatory discussions with the U.S. Food and Drug Administration (“FDA”) and other regulatory authorities are expected in the second half of 2025, during which we intend to present our plans to analyze real-world evidence and seek endorsement that supports potential future regulatory filings. Business Update On April 21, 2025, a BiomX Special Meeting of stockholders took place during which shareholders approved the exercise of certain warrants issued as part of the Company’s $12 million financings announced in February 2025. The completed financings will provide runway expected to support the Company’s activities into the first quarter of 2026, when the Company anticipates the readout of topline Phase 2b results for BX004 for the treatment of CF patients with chronic pulmonary infections caused by P. aeruginosa. First Quarter 2025 Financial Results Cash balance and restricted cash as of March 31, 2025, were $21.2 million, compared to $18.0 million as of December 31, 2024. The increase was primarily due to funds raised in the February 2025 financings, partly offset by net cash used in operating activities. BiomX estimates its cash, cash equivalents and restricted cash are sufficient to fund its operations into the first quarter of 2026. Research and development expenses, net were $5.3 million for the first quarter of 2025, compared to $4.1 million for the first quarter of 2024. The increase was primarily due to the following factors: preparations for the Phase 2b clinical trial of the Company’s CF product candidate, BX004; an increase in expenses relating to the Phase 2 clinical trial of the Company’s DFO product candidate, BX211; and an increase in rent and related expenses following the March 2024 acquisition of Adaptive Phage Therapeutics (APT). This increase was partly offset by higher grants BiomX received. General and administrative expenses were $2.5 million for the first quarter of 2025, compared to $2.7 million for the first quarter of 2024. The decrease is primarily attributed to expenses incurred during 2024 in connection with the acquisition of APT completed in March 2024, partially offset by increased salaries and share-based compensation expenses. Net loss was $7.7 million for the first quarter of 2025, compared to $17.3 million for the first quarter of 2024. The decrease is mainly due to the change in the fair value of the warrants issued as part of the Company’s financing completed in March 2024. Net cash used in operating activities for the three months ended March 31, 2025, was $8.7 million, compared to $11.4 million for the same period in 2024. Conference Call and Webcast Details BiomX will host a conference call and webcast on May 15, 2025, at 2:00 PM ET to discuss its first quarter 2025 financial results and to provide a corporate update. Participant Dial-In Number: +1 877-407-0724 Participant International Dial-In: +1 201-389-0898 Webcast Link Link About BX211 BX211 is a phage treatment for the treatment of DFO associated with S. aureus. DFO is a bacterial infection of the bone that usually develops from an infected foot ulcer and is a leading cause of amputation in patients with diabetes. In March 2025, BiomX announced positive topline results from the Phase 2 trial in which BX211 was demonstrated to be safe and well-tolerated and patients receiving BX211 exhibited statistically significant1 and sustained reduction of ulcer size (PAR)(p = 0.046 at week 12; p=0.052 at week 13), with a separation from placebo starting at week 7 and a difference greater than 40% by week 10. In addition, BX211 also produced statistically significant1 improvements in both ulcer depth at week 13 (in patients with ulcer depth defined as bone at baseline, ulcer depth was classified according to deepest tissue involved as measured by swab) (p=0.048), and in reducing the expansion of ulcer area (p=0.017). Over the 12-week treatment period, all patients (treatment and placebo) were treated in accordance with standard of care, including with systemic antibiotic therapy as appropriate. BiomX is currently planning a Phase 2/3 trial, pending discussions and feedback from the FDA. About BX004 BiomX is developing BX004, a fixed multi-phage cocktail, for the treatment of CF patients with chronic pulmonary infections caused by P. aeruginosa, a main contributor to morbidity and mortality in patients with CF. In November 2023, BiomX announced positive topline results from Part 2 of the Phase 1b/2a trial where BX004 demonstrated improvement in pulmonary function associated with a reduction in P. aeruginosa burden compared to placebo in a predefined subgroup of patients with reduced lung function (baseline FEV1<70%). BiomX expects to initiate a randomized, double blind, placebo-controlled, multi-center Phase 2b trial in CF patients with chronic P. aeruginosa pulmonary infections. The trial is designed to enroll approximately 60 patients randomized at a 2:1 ratio to BX004 or placebo. Treatment is expected to be administered via inhalation twice daily for a duration of 8 weeks. The trial is designed to monitor the safety and tolerability of BX004 and is designed to demonstrate improvement in microbiological reduction of P. aeruginosa burden and evaluation of effects on clinical parameters such as lung function measured by FEV1 and patient reported outcomes. Pending progress of the trial, topline results are expected in the first quarter of 2026. The FDA has granted BX004 Fast Track designation and Orphan Drug Designation. About BiomX BiomX is a clinical-stage company leading the development of natural and engineered phage cocktails and personalized phage treatments designed to target and destroy harmful bacteria for the treatment of chronic diseases with substantial unmet needs. BiomX discovers and validates proprietary bacterial targets and applies its BOLT (“BacteriOphage Lead to Treatment”) platform to customize phage compositions against these targets. For more information, please visit www.biomx.com, the content of which does not form a part of this press release. Safe Harbor This press release contains express or implied “forward-looking statements” within the meaning of the “safe harbor” provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “target,” “believe,” “expect,” “will,” “may,” “anticipate,” “estimate,” “would,” “positioned,” “future,” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. For example, when BiomX refers its anticipated timing for reporting results for its clinical assets as well as the design thereof, expected discussions with the FDA and other regulatory authorities and the DHA and results thereof, the potential of its candidates to address the substantial unmet needs of patients with intractable infections, and the estimates of the sufficiency of its cash, cash equivalents and restricted cash, it is using forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on BiomX management’s current beliefs, expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of BiomX’s control. These risks and uncertainties include, but are not limited to, changes in applicable laws or regulations; the possibility that BiomX may be adversely affected by other economic, business, and/or competitive factors, including risks inherent in pharmaceutical research and development, such as: adverse results in BiomX’s drug discovery, preclinical and clinical development activities, the risk that the results of preclinical studies and early clinical trials may not be replicated in later clinical trials, BiomX’s ability to enroll patients in its clinical trials, and the risk that any of its clinical trials may not commence, continue or be completed on time, or at all; decisions made by the FDA, DHA and other regulatory authorities; decisions made by investigational review boards at clinical trial sites and publication review bodies with respect to our development candidates; BiomX’s ability to obtain, maintain and enforce intellectual property rights for its platform and development candidates; its potential dependence on collaboration partners; competition; uncertainties as to the sufficiency of BiomX’s cash resources to fund its planned activities for the periods anticipated and BiomX’s ability to manage unplanned cash requirements; and general economic and market conditions. Therefore, investors should not rely on any of these forward-looking statements and should review the risks and uncertainties described under the caption “Risk Factors” in BiomX’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (the “SEC”) on March 25, 2025, and additional disclosures BiomX makes in its other filings with the SEC, which are available on the SEC’s website at www.sec.gov. Forward-looking statements are made as of the date of this press release, and except as provided by law BiomX expressly disclaims any obligation or undertaking to update forward-looking statements. Contacts: BiomX, Inc. Ben Cohen Head Corporate Communications benc@biomx.com 1 All p-values described in this release are non-adjusted 2 The Wagner Scale is a clinical grading system used to classify the severity of diabetic foot ulcers, ranging from 0 (intact skin) to 5 (extensive gangrene). Released May 15, 2025

Clinical ResultPhase 2Fast TrackFinancial StatementOrphan Drug

100 Deals associated with Deferoxamine Mesylate

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KEGG	Wiki	ATC	Drug Bank
D01186	Deferoxamine Mesylate	V03AC01	DB00746

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Chronic iron overload	United States	Novartis Pharmaceuticals Corp.	01 Apr 1968
Chronic iron overload	United States	Mitem Pharma SAS	01 Apr 1968
Iron Overload	United States	Novartis Pharmaceuticals Corp.	01 Apr 1968
Iron Overload	United States	Mitem Pharma SAS	01 Apr 1968

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Chronic ulcer	Phase 2	United States	Tautona Group	21 Jul 2020
Skin Ulcer	Phase 2	United States	Tautona Group	21 Jul 2020
Beta-Thalassemia	Phase 2	Greece	Novartis Pharmaceuticals Corp.	01 Jan 2011
Transfusion-dependent Beta Thalassemia	Phase 2	Greece	Novartis Pharmaceuticals Corp.	01 Jan 2011
Anemia, Sickle Cell	Phase 2	United States	Novartis Pharmaceuticals Corp.	01 May 2003
Hemosiderosis	Phase 2	United States	Novartis Pharmaceuticals Corp.	01 May 2003
Metastatic Malignant Neoplasm to the Leptomeninges	Phase 1	United States	Memorial Sloan Kettering Cancer Center	22 Dec 2021
Muscular Atrophy	Preclinical	South Korea	Suwon University	20 Jun 2023
Parkinson Disease	Preclinical	-	-	04 Jun 2017
Spinal Cord Injuries	Preclinical	European Union	SCT-Spinal Cord Therapeutics GmbH	-

Clinical Result

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Phase

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05184816 (Phase 1a analysis, ASCO2025)	Phase 1	Metastatic Malignant Neoplasm to the Leptomeninges lipocalin-2/SLC22A17	8	Intrathecal deferoxamine (IT-DFO) 10 mg	girugkbijw(addpvezwec) = median overall survival for the evaluable cohort (n = 7) was 10.0 months (95% CI, 6.5 - NA) shhndaocwu (vhvuqdyubr ) View more	Positive	30 May 2025
				Intrathecal deferoxamine (IT-DFO) 30 mg
NCT00901199 (CTgov)	Phase 2	Transfusion-dependent Thalassemia \| Thalassemia \| Iron Overload	22	Deferasirox+Deferoxamine	nmdakatuxp(yvdsaftrhg) = bdszvkfwjv zrtbmbsrfy (avigorxfti, ofsoihxhpq - wcwgtihgvf) View more	-	12 Aug 2021
NCT01254227 (HYPERION, CTgov)	Phase 2	Iron Overload	60	DFX+Deferasirox (DFX)+Deferoxamine	ojiwdtogza(wzhwqdeufl) = iotwiemnsh nxwaavetpu (jvnzgtxomt, aogdmzhduy - pjkpjtuivh) View more	-	24 Jun 2021
NCT01459718 (CTgov)	Phase 2	Beta-Thalassemia \| Iron Overload \| Transfusion-dependent Beta Thalassemia	32	Deferasirox+Deferoxamine (DFO)	swrzdocswi = ifjwzsksqg tketptfanu (twzzrmbkuq, cwfordxdpa - gktcgcdiam) View more	-	23 Oct 2019
NCT01662895 (HIDEF \| Hi-Def, CTgov)	Phase 2	Cerebral Hemorrhage	42	Deferoxamine (Deferoxamine)	ayodxsoboc = qqdtmciojo durmplyggu (lisfzwoiwt, hxkdqnsvjv - wrgyqcyqtx) View more	-	30 May 2019
				Normal saline (Normal Saline)	ayodxsoboc = cvrdxhnhkx durmplyggu (lisfzwoiwt, gdwfaklttj - ionumvtxyn) View more
NCT02175225 (CTgov)	Phase 2	Cerebral Hemorrhage	294	Deferoxamine Mesylate (Deferoxamine Mesylate)	byankilvhs = pbjvbwonen yzxmnrxljh (lhzwpdmwdq, bovtosoryc - qixxxboici) View more	-	30 May 2019
				Placebo (for Deferoxamine Mesylate) (Normal Saline)	byankilvhs = kvykgvhkht yzxmnrxljh (lhzwpdmwdq, dopejpbbkn - xzcywsfsan) View more
NCT02175225 (Pubmed) Manual	Phase 2	Cerebral Hemorrhage	291	Deferoxamine mesylate	jgheyrrmcs(lwypojgfut) = itqprvcybi apfafzlihn (exlzwufiry ) View more	Negative	01 May 2019
				Placebo	jgheyrrmcs(lwypojgfut) = nbqxgulgqa apfafzlihn (exlzwufiry ) View more
NCT00749515 (CTgov)	Phase 4	Beta-Thalassemia \| Transfusion-dependent Thalassemia \| Anemia, Sickle Cell	15	deferasirox (Inadequate Responders)	jqmasisvwt(hepvjlldjp) = atsxziurom jrztqnwgbm (bixmqbgugc, 259.42) View more	-	06 Feb 2019
				deferasirox (Adequate Responders (Control))	jqmasisvwt(hepvjlldjp) = kfvwrtwoqt jrztqnwgbm (bixmqbgugc, 63.4) View more
NCT01254227 (HYPERION, Pubmed \| Circulation \| Blood) Manual	Phase 2	Iron Overload	60	deferasirox+deferoxamin	ikqqzbdtez(nuymgqweht) = 7.7 ms vs 7.2 ms (baseline) dcznttqnrh (ukjnyzwfwn ) View more	Positive	18 Jun 2015
NCT00600938 (CORDELIA, Pubmed \| Am J Hematol)	Phase 2	Beta-Thalassemia \| Iron Overload	197	Deferasirox	nswtqdpbky(pgdecqoecd) = serious drug-related AEs were reported in 6.8% of deferasirox and 6.9% of DFO patients mbbojybpqa (mbkvzzytqq ) View more	-	01 Feb 2015
				Deferoxamine (DFO)

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