Latest news with #CTCL
Business Wire
23-05-2025
- Business
- Business Wire
Innate Pharma Highlights Durable Responses to Lacutamab in Sezary Syndrome and Mycosis Fungoides
MARSEILLE, France--(BUSINESS WIRE)--Regulatory News: Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (' Innate ' or the ' Company ') today announced the presentation of long-term follow-up data from the Phase 2 TELLOMAK clinical trial evaluating lacutamab, an anti-KIR3DL2 monoclonal antibody, in patients with Sézary syndrome (SS) and mycosis fungoides (MF), two rare and aggressive forms of cutaneous T-cell lymphoma (CTCL). The results will be presented at the American Society of Clinical Oncology (ASCO) 2025 Annual Meeting, in Chicago, Illinois. Lacutamab was recently granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of Sézary syndrome, underscoring its potential to address critical needs in advanced CTCL. As of October 17, 2024, data cut-off, lacutamab demonstrated compelling and sustained clinical activity in heavily pretreated patients, with a global ORR of 42.9% for SS and 19.6% for MF. With longer follow-up, we observed improved median duration of response of 25.6 months in SS and 13.8 months in MF, highlighting the durability of responses in these challenging indications 1. In addition, lacutamab was very well tolerated supporting the strong rationale for further investigations in combination beyond CTCL, especially in combination with other anti-lymphoma agents in peripheral T-cell lymphomas (PTCL). ' Patients with advanced mycosis fungoides and Sézary syndrome often face a poor prognosis and limited treatment options after multiple prior lines of therapy,' said Prof. Pierluigi Porcu, Director, Division of Hematologic Malignancies and Hematopoietic Stem Cell Transplantation, Sidney Kimmel Cancer Center, Jefferson Health, Philadelphia and principal investigator of the TELLOMAK trial. ' The durability and depth of responses observed with lacutamab in this study are highly promising and represent a significant advancement for this patient population.' ' The long-term follow-up data from the TELLOMAK clinical study confirms lacutamab's meaningful clinical benefit in Sézary syndrome and mycosis fungoides and were the basis of the FDA Breakthrough Therapy Designation. We are encouraged by these results and are actively preparing a Phase 3 trial in collaboration with health authorities to bring this promising therapy to patients as swiftly as possible,' added Dr Sonia Quaratino, Chief Medical Officer of Innate Pharma. 1 Compared to results previously presented at ASH 2023 and ASCO 2024. Efficacy results in SS patients (Data cut-off: OCT 17, 2024) Efficacy results in MF patients (Data cut-off: OCT 17, 2024) Best Response All MF N=107 KIR3DL2 ≥1% N=48 KIR3DL2 <1% N=59 CR (complete response), N (%) 3 (2.8) 3 (6.3) 0 (0.0) PR (partial response), N (%) 18 (16.8) 7 (14.6) 11 (18.6) SD (stable disease), N (%) 71 (66.4) 30 (62.5) 41 (69.5) PD (progressive disease), N (%) 13 (12.1) 6 (12.5) 7 (11.9) ORR (Objective Response Rate), % [95%CI] Olsen 2011 19.6 [13.2, 28.1] 20.8 [11.7, 34.3] 18.6 [10.7, 30.4] ORR, % [95%CI] Olsen 2022 24.3 [17.2, 33.2] 29.2 [18.2, 43.2] 20.3 [12.0, 32.3] Time to response, months, median (range) 2.8 (1-37) 1.0 (1-5) 2.8 (1-37) DoR, months, median [95% CI] 13.8 [7.4, NE] 13.8 [4.6, NE] 15.7 [5.1, NE] PFS, months, median [95% CI] 10.2 [8.0, 15.4] 11.8 [5.6, 16.8] 9.5 [6.5, 16.6] Expand Abstract details: Abstract: 2522 Abstract Title: Lacutamab in patients with relapsed and refractory Sézary syndrome: Long term follow-up from the TELLOMAK phase 2 trial Session Type: Poster Session Session Title: Developmental Therapeutics—Immunotherapy Session Date and Time: Monday June 2, 2025 – 1:30 – 4:30 PM CDT Abstract: 2523 Abstract Title: Lacutamab in patients with relapsed and/or refractory mycosis fungoides: Long-term follow-up and translational data from the TELLOMAK phase 2 trial Session Type: Poster Session Session Title: Developmental Therapeutics—Immunotherapy Session Date and Time: Monday June 2, 2025 – 1:30 – 4:30 PM CDT About Lacutamab Lacutamab is a first-in-class anti-KIR3DL2 humanized cytotoxicity-inducing antibody that is currently in clinical trials for treatment of cutaneous T-cell lymphoma (CTCL), an orphan disease, and peripheral T cell lymphoma (PTCL). Rare cutaneous lymphoma of T lymphocytes have a poor prognosis with few efficacious and safe therapeutic options at advanced stages. KIR3DL2 is an inhibitory receptor of the KIR family, expressed by approximately 65% of patients across all CTCL subtypes and expressed by up to 90% of patients with certain aggressive CTCL subtypes, in particular, Sézary syndrome. KIR3DL2 is expressed in up to 50% of patients with mycosis fungoides and peripheral T-cell lymphoma (PTCL). It has a restricted expression on normal tissues. Lacutamab has been granted European Medicines Agency (EMA) PRIME designation, and the US Food and Drug Administration (FDA) granted Fast Track designation for the treatment of patients with relapsed or refractory Sézary syndrome who have received at least two prior systemic therapies. Lacutamab is granted orphan drug status in the European Union and the United States for the treatment of CTCL. Lacutamab has received Breakthrough Therapy Designation from the FDA. About TELLOMAK TELLOMAK (NCT03902184) is a global, open-label, multi-cohort Phase 2 clinical trial in patients with Sézary syndrome and mycosis fungoides (MF) in the United States and Europe. Specifically: Cohort 1: lacutamab being evaluated as a single agent in approximately 60 patients with Sézary syndrome who have received at least two prior systemic therapies, including mogamulizumab. The Sézary syndrome cohort of the study could enable the registration of lacutamab in this indication. Cohort 2: lacutamab being evaluated as a single agent in patients with MF that express KIR3DL2, as determined at baseline with a Simon 2-stage design. Cohort 3: lacutamab being evaluated as a single agent in patients with MF that do not express KIR3DL2, as determined at baseline, with a Simon-2 stage design. All comers: lacutamab being evaluated as a single agent in patients with both KIR3DL2 expressing and non-expressing MF to explore the correlation between the level of KIR3DL2 expression and treatment outcomes utilizing a formalin-fixed paraffin embedded (FFPE) assay under development as a companion diagnostic. The trial is fully enrolled. The primary endpoint of the trial is objective global response rate. Key secondary endpoints are progression-free survival, duration of response, overall survival, quality of life, pharmacokinetics and immunogenicity and adverse events. About Innate Pharma Innate Pharma S.A. is a global, clinical-stage biotechnology company developing immunotherapies for cancer patients. Its innovative approach aims to harness the innate immune system through three therapeutic approaches: multi-specific NK Cell Engagers via its ANKET ® (Antibody-based NK cell Engager Therapeutics) proprietary platform and Antibody Drug Conjugates (ADC) and monoclonal antibodies (mAbs). Innate's portfolio includes several ANKET ® drug candidates to address multiple tumor types as well as IPH4502, a differentiated ADC in development in solid tumors. In addition, anti-KIR3DL2 mAb lacutamab is developed in advanced form of cutaneous T cell lymphomas and peripheral T cell lymphomas, and anti-NKG2A mAb monalizumab is developed with AstraZeneca in non-small cell lung cancer. Innate Pharma is a trusted partner to biopharmaceutical companies such as Sanofi and AstraZeneca, as well as leading research institutions, to accelerate innovation, research and development for the benefit of patients. Headquartered in Marseille, France with a US office in Rockville, MD, Innate Pharma is listed on Euronext Paris and Nasdaq in the US. Learn more about Innate Pharma at Follow us on LinkedIn and X. Information about Innate Pharma shares Disclaimer on forward-looking information and risk factors This press release contains certain forward-looking statements, including those within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. The use of certain words, including 'anticipate,' 'believe,' 'can,' 'could,' 'estimate,' 'expect,' 'may,' 'might,' 'potential,' 'should,' 'will,' or the negative of these and similar expressions, is intended to identify forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, these forward-looking statements are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those anticipated. These risks and uncertainties include, among other things, the uncertainties inherent in research and development, including related to safety, progression of and results from its ongoing and planned clinical trials and preclinical studies, review and approvals by regulatory authorities of its product candidates, the Company's reliance on third parties to manufacture its product candidates, the Company's commercialization efforts and the Company's continued ability to raise capital to fund its development. For an additional discussion of risks and uncertainties, which could cause the Company's actual results, financial condition, performance or achievements to differ from those contained in the forward-looking statements, please refer to the Risk Factors ('Facteurs de Risque") section of the Universal Registration Document filed with the French Financial Markets Authority ('AMF'), which is available on the AMF website or on Innate Pharma's website, and public filings and reports filed with the U.S. Securities and Exchange Commission ('SEC'), including the Company's Annual Report on Form 20-F for the year ended December 31, 2024, and subsequent filings and reports filed with the AMF or SEC, or otherwise made public by the Company. References to the Company's website and the AMF website are included for information only and the content contained therein, or that can be accessed through them, are not incorporated by reference into, and do not constitute a part of, this press release. In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by the Company or any other person that the Company will achieve its objectives and plans in any specified time frame or at all. The Company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. This press release and the information contained herein do not constitute an offer to sell or a solicitation of an offer to buy or subscribe to shares in Innate Pharma in any country.
Yahoo
14-05-2025
- Business
- Yahoo
BioInvent Presents Additional CTCL Phase 2a Data for BI-1808 Monotherapy at EHA 2025
Data from the cutaneous T-cell lymphoma (CTCL) cohort demonstrate promising clinical activity that correlates with strong immune activation One complete response (CR), three partial responses (PR), and four stable diseases (SD) achieved as of Feb 2025 in eight evaluable patients Study currently enrolling patients for signal-seeking. Dose optimization phase to follow LUND, SE / / May 14, 2025 / BioInvent International AB ("BioInvent") (Nasdaq Stockholm:BINV), a biotech company focused on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, today announced that additional data from the ongoing Phase 2a dose expansion study of BI-1808 monotherapy in Cutaneous T-cell Lymphoma (CTCL) will be presented in a poster at the European Hematology Association (EHA) 2025 congress to take place June 12-15 in Milan, Italy. The submitted abstract data from the CTCL cohort include results from ten patients treated with BI-1808 with eight deemed evaluable. As of the cut-off date February 20, 2025, one patient had achieved a complete response (CR), three achieved partial response (PR), and four demonstrated stable disease (SD). The treatment was well tolerated with primarily mild to moderate adverse events with no grade 3 or higher events reported. Notably, immune activation was observed in the early weeks of treatment, specifically depletion of regulatory T cells and an influx of CD8+ T cells into the skin, suggesting that BI-1808 effectively stimulates the targeted response in CTCL. The upcoming poster presentation will include more detailed and recent data, including findings in patients with PTCL (Peripheral T-cell lymphoma). "We look forward to presenting these positive data at EHA, which illustrate BI-1808's potential as an immunomodulatory treatment for CTCL patients, said Martin Welschof, Chief Executive Officer of BioInvent. "The emerging clinical responses, along with immune activation reflect the meaningful biological activity of BI-1808 and support further development in hematologic malignancies and solid tumor settings. We believe that BI-1808 could become a novel treatment option for CTCL patients, where innovative approaches are urgently needed - a belief further supported by the recent Fast Track and Orphan Drug Designation granted by the FDA." Details of the presentation are below: Title: Robust Single Agent Activity of BI-1808, a Tumor Necrosis Factor Receptor 2 (TNFR2) Blocker/Depleter, in Cutaneous T Cell Lymphoma (CTCL) PatientsSession Date and Time: June 13, 2025, 6:30-7:30 pm CESTSession Title: Poster Session 1Lead Author: Stefan K. Barta, University of Pennsylvania Hospital, Philadelphia, PA, USAAbstract Number: PF961 The poster will be posted to the Scientific Publications section of the company website after the e-poster disclosure ( on June 12 at 9:00 am CEST. The safety and preliminary efficacy of BI-1808 monotherapy are currently being evaluated in Part A of the ongoing Phase 2a (NCT04752826) study in patients with T-cell lymphomas, including CTCL. The trial is expected to enroll 20 patients at a signal-seeking dose, after which a dose optimization phase will be initiated. About BI-1808 The anti-TNFR2 antibody BI-1808 is part of BioInvent's tumor-associated regulatory T cells (Treg)-targeting program. TNFR2 is particularly upregulated on Tregs of the tumor microenvironment and has been shown to be important for tumor expansion and survival, representing a new and promising target for cancer immunotherapy. BI-1808 is a first-in-class drug candidate in clinical development for the treatment of solid tumors and blood cancer. BI-1808 has shown single agent activity and excellent tolerability in an ongoing Phase 2a study and signs of efficacy and favorable safety profile in combination with pembrolizumab in the ongoing Part B of the Phase 1/2a study (ASCO 2024). About BioInvent BioInvent International AB (Nasdaq Stockholm: BINV) is a clinical-stage biotech company that discovers and develops novel and first-in-class immuno-modulatory antibodies for cancer therapy, with currently five drug candidates in six ongoing clinical programs in Phase 1/2 trials for the treatment of hematological cancer and solid tumors. The Company's validated, proprietary F.I.R.S.T™ technology platform identifies both targets and the antibodies that bind to them, generating many promising new immune-modulatory candidates to fuel the Company's own clinical development pipeline and providing licensing and partnering opportunities. The Company generates revenues from research collaborations and license agreements with multiple top-tier pharmaceutical companies, as well as from producing antibodies for third parties in the Company's fully integrated manufacturing unit. More information is available at For further information, please contact: Cecilia Hofvander, VP Investor RelationsPhone: +46 (0)46 286 85 50Email: International AB (publ)Co. Reg. No.: 556537-7263Visiting address: Ideongatan 1Mailing address: 223 70 LUNDPhone: +46 (0)46 286 85 The press release contains statements about the future, consisting of subjective assumptions and forecasts for future scenarios. Predictions for the future only apply as the date they are made and are, by their very nature, in the same way as research and development work in the biotech segment, associated with risk and uncertainty. With this in mind, the actual outcome may deviate significantly from the scenarios described in this press release. This information is information that BioInvent International is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact persons set out above, at 2025-05-14 15:30 CEST. Attachments BioInvent Presents Additional CTCL Phase 2a Data for BI-1808 Monotherapy at EHA 2025 SOURCE: BioInvent International View the original press release on ACCESS Newswire Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
14-05-2025
- Business
- Yahoo
BioInvent Presents Additional CTCL Phase 2a Data for BI-1808 Monotherapy at EHA 2025
Data from the cutaneous T-cell lymphoma (CTCL) cohort demonstrate promising clinical activity that correlates with strong immune activation One complete response (CR), three partial responses (PR), and four stable diseases (SD) achieved as of Feb 2025 in eight evaluable patients Study currently enrolling patients for signal-seeking. Dose optimization phase to follow LUND, SE / / May 14, 2025 / BioInvent International AB ("BioInvent") (Nasdaq Stockholm:BINV), a biotech company focused on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, today announced that additional data from the ongoing Phase 2a dose expansion study of BI-1808 monotherapy in Cutaneous T-cell Lymphoma (CTCL) will be presented in a poster at the European Hematology Association (EHA) 2025 congress to take place June 12-15 in Milan, Italy. The submitted abstract data from the CTCL cohort include results from ten patients treated with BI-1808 with eight deemed evaluable. As of the cut-off date February 20, 2025, one patient had achieved a complete response (CR), three achieved partial response (PR), and four demonstrated stable disease (SD). The treatment was well tolerated with primarily mild to moderate adverse events with no grade 3 or higher events reported. Notably, immune activation was observed in the early weeks of treatment, specifically depletion of regulatory T cells and an influx of CD8+ T cells into the skin, suggesting that BI-1808 effectively stimulates the targeted response in CTCL. The upcoming poster presentation will include more detailed and recent data, including findings in patients with PTCL (Peripheral T-cell lymphoma). "We look forward to presenting these positive data at EHA, which illustrate BI-1808's potential as an immunomodulatory treatment for CTCL patients, said Martin Welschof, Chief Executive Officer of BioInvent. "The emerging clinical responses, along with immune activation reflect the meaningful biological activity of BI-1808 and support further development in hematologic malignancies and solid tumor settings. We believe that BI-1808 could become a novel treatment option for CTCL patients, where innovative approaches are urgently needed - a belief further supported by the recent Fast Track and Orphan Drug Designation granted by the FDA." Details of the presentation are below: Title: Robust Single Agent Activity of BI-1808, a Tumor Necrosis Factor Receptor 2 (TNFR2) Blocker/Depleter, in Cutaneous T Cell Lymphoma (CTCL) PatientsSession Date and Time: June 13, 2025, 6:30-7:30 pm CESTSession Title: Poster Session 1Lead Author: Stefan K. Barta, University of Pennsylvania Hospital, Philadelphia, PA, USAAbstract Number: PF961 The poster will be posted to the Scientific Publications section of the company website after the e-poster disclosure ( on June 12 at 9:00 am CEST. The safety and preliminary efficacy of BI-1808 monotherapy are currently being evaluated in Part A of the ongoing Phase 2a (NCT04752826) study in patients with T-cell lymphomas, including CTCL. The trial is expected to enroll 20 patients at a signal-seeking dose, after which a dose optimization phase will be initiated. About BI-1808 The anti-TNFR2 antibody BI-1808 is part of BioInvent's tumor-associated regulatory T cells (Treg)-targeting program. TNFR2 is particularly upregulated on Tregs of the tumor microenvironment and has been shown to be important for tumor expansion and survival, representing a new and promising target for cancer immunotherapy. BI-1808 is a first-in-class drug candidate in clinical development for the treatment of solid tumors and blood cancer. BI-1808 has shown single agent activity and excellent tolerability in an ongoing Phase 2a study and signs of efficacy and favorable safety profile in combination with pembrolizumab in the ongoing Part B of the Phase 1/2a study (ASCO 2024). About BioInvent BioInvent International AB (Nasdaq Stockholm: BINV) is a clinical-stage biotech company that discovers and develops novel and first-in-class immuno-modulatory antibodies for cancer therapy, with currently five drug candidates in six ongoing clinical programs in Phase 1/2 trials for the treatment of hematological cancer and solid tumors. The Company's validated, proprietary F.I.R.S.T™ technology platform identifies both targets and the antibodies that bind to them, generating many promising new immune-modulatory candidates to fuel the Company's own clinical development pipeline and providing licensing and partnering opportunities. The Company generates revenues from research collaborations and license agreements with multiple top-tier pharmaceutical companies, as well as from producing antibodies for third parties in the Company's fully integrated manufacturing unit. More information is available at For further information, please contact: Cecilia Hofvander, VP Investor RelationsPhone: +46 (0)46 286 85 50Email: International AB (publ)Co. Reg. No.: 556537-7263Visiting address: Ideongatan 1Mailing address: 223 70 LUNDPhone: +46 (0)46 286 85 The press release contains statements about the future, consisting of subjective assumptions and forecasts for future scenarios. Predictions for the future only apply as the date they are made and are, by their very nature, in the same way as research and development work in the biotech segment, associated with risk and uncertainty. With this in mind, the actual outcome may deviate significantly from the scenarios described in this press release. This information is information that BioInvent International is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact persons set out above, at 2025-05-14 15:30 CEST. Attachments BioInvent Presents Additional CTCL Phase 2a Data for BI-1808 Monotherapy at EHA 2025 SOURCE: BioInvent International View the original press release on ACCESS Newswire Sign in to access your portfolio
Associated Press
14-04-2025
- Business
- Associated Press
Positive Outcome in 75% of CTCL Patients Treated with HyBryte™ for 18 Weeks
Interim Results from FDA-Funded Study Reinforces HyBryte's™ Rapid Response and Strong Safety Profile PRINCETON, N.J., April 14, 2025 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today interim results from the ongoing open-label, investigator-initiated study (IIS) evaluating extended HyBryte™ (synthetic hypericin) treatment for up to 54 weeks in patients with early-stage cutaneous T-cell lymphoma (CTCL). Following 18 weeks of treatment, 75% of patients achieved 'Treatment Success,' reinforcing HyBryte™ as a potentially safe and fast-acting therapy for this chronic and underserved cancer. The IIS is sponsored by Ellen Kim, MD, Director, Penn Cutaneous Lymphoma Program, Vice Chair of Clinical Operations, Dermatology Department, and Professor of Dermatology at the Hospital of the University of Pennsylvania who was a leading enroller in the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study and is the Principal Investigator for the confirmatory Phase 3 FLASH2 study for the treatment of early-stage CTCL. To date, nine patients have been enrolled and treated with HyBryte™ over a time period of up to 54 weeks in the IIS, with all data for the Week 18 timepoint now complete. Consistent with the Phase 3 trials, Treatment Success is predefined as a greater than or equal to 50% improvement in the cumulative mCAILS (modified Composite Assessment of Index Lesion Severity) score compared to Baseline. Of the eight patients who could be evaluated through Week 18, six (75%) had a Treatment Success. The 18-week treatment window is the same window that is being evaluated in the FLASH2 double-blind, placebo-controlled, randomized study that is currently enrolling patients. This rapid response is a distinct advantage of HyBryte™ therapy, with many other therapies used in CTCL taking up to six to 12 months to generate a clinically meaningful treatment response. Of these eight evaluable patients through Week 18, four have gone on to complete the 54-week treatment with an average maximum improvement in mCAILS score of 85%, three are still on treatment and one dropped out (due to logistical issues). HyBryte™ appears to be safe and well tolerated in all patients. 'The complete response rates observed, including three patients achieving a complete response on this study to date, as well as the consistent treatment response and safety profile across multiple HyBryte™ clinical studies, has been exciting to see,' noted Dr. Kim, Principal Investigator of the IIS. 'In the first Phase 3 FLASH study, HyBryte™ was shown to be efficacious with a benign safety profile compared to the current therapies of steroids, chemotherapeutics and ultraviolet light in this chronic orphan disease. With limited treatment options, especially in the early stages of their disease, CTCL patients are often searching for alternative treatments. In our study funded by the U.S. Food and Drug Administration (FDA), initial results evaluating the expanded use of HyBryte™ in a 'real world' treatment setting remain very promising, further supporting and extending results from the previous positive Phase 2 and 3 clinical trials. It also provides further confidence to the potential responses we can expect to see in the confirmatory Phase 3 placebo-controlled FLASH2 study. We look forward to continuing to work with the FDA to complete the IIS while we participate in the confirmatory 18-week FLASH2 study.' 'We are pleased with these recent study results, giving patients an opportunity to access the therapy in an open-label setting,' stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. 'CTCL is an incredibly difficult to treat orphan disease and remains an area of unmet medical need with a very limited number of safe and effective therapies. Following the initial Phase 3 FLASH study, which demonstrated the safety and efficacy of shorter courses of HyBryte™ therapy, we are pleased to see that continuing treatment for longer time periods is resulting in the anticipated improved outcomes for patients. The majority of patients show a strong treatment response by Week 18, a noticeable advantage over other therapies that may take six to 12 months to show improvement. As the body of compelling data continues to grow in support of this novel therapy, we look forward to continuing to work with Dr. Kim on this important study as well as advancing enrollment in the 80-patient confirmatory Phase 3 FLASH2 replication study. We will plan to provide additional updates on the IIS as data becomes available.' The clinical study RW-HPN-MF-01, 'Assessment of Treatment with Visible Light Activated Synthetic Hypericin Ointment in Mycosis Fungoides Patients' is designed as an open-label, multicenter clinical trial enrolling approximately 20 patients in the U.S. Patients have the potential to be treated for up to 54 weeks with twice a week dosing (visible light activation following ointment application by 24 ± 6 hours). The study also allows for potential transition to a 'real-world' setting with home-use. The primary endpoint for the study is evaluating the number of treatment successes defined as ≥50% reduction in the cumulative mCAILS score from Baseline to end of the treatment. Study RW-HPN-MF-01 is supported by an FDA Orphan Products Development Grant of up to $2.6 million. About HyBryte™ HyBryte™ (research name SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte™ is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by safe, visible light approximately 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte™ has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA). The published Phase 3 FLASH trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle (Cycle 1), 116 patients received HyBryte™ treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte™ achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte™ treatment in this cycle was safe and well tolerated. In the second open-label treatment cycle (Cycle 2), all patients received HyBryte™ treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte™ treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte™ treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment responses also revealed a statistically significant improvement (p<0.0001) between the two timepoints, indicating that continued treatment results in better outcomes. HyBryte™ continued to be safe and well tolerated. Additional analyses also indicated that HyBryte™ is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular. The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte™ treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte™ throughout all 3 cycles of treatment, 49% of them demonstrated a positive treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte™ is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte™ continued to be well tolerated despite extended and increased use of the product to treat multiple lesions. Overall safety of HyBryte™ is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte's™ mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant, and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte™ potentially represents the safest available efficacious treatment for CTCL. With very limited systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues. Following the first Phase 3 study of HyBryte™ for the treatment of CTCL, the FDA and the EMA indicated that they would require a second successful Phase 3 trial to support marketing approval. With agreement from the EMA on the key design components, the second, confirmatory study, called FLASH2, is expected to be initiated before the end of 2024. This study is a randomized, double-blind, placebo-controlled, multicenter study that will enroll approximately 80 subjects with early-stage CTCL. The FLASH2study replicates the double-blind, placebo-controlled design used in the first successful Phase 3 FLASH study that consisted of three 6-week treatment cycles (18 weeks total), with the primary efficacy assessment occurring at the end of the initial 6-week double-blind, placebo-controlled treatment cycle (Cycle 1). However, this second study extends the double-blind, placebo-controlled assessment to 18 weeks of continuous treatment (no 'between-Cycle' treatment breaks) with the primary endpoint assessment occurring at the end of the 18-week timepoint. In the first Phase 3 study, a treatment response of 49% (p<0.0001 vs patients receiving placebo in Cycle 1) was observed in patients completing 18 weeks (3 cycles) of therapy. In this second study, all important clinical study design components remain the same as in the first FLASH study, including the primary endpoint and key inclusion-exclusion criteria. The extended treatment for a continuous 18 weeks in a single cycle is expected to statistically demonstrate HyBryte's™ increased effect over a more prolonged, 'real world' treatment course. Given the extensive engagement with the CTCL community, the esteemed Medical Advisory Board and the previous trial experience with this disease, accelerated enrollment in support of this study is anticipated, including the potential to enroll previously identified and treated HyBryte™ patients from the FLASH study. Discussions with the FDA on an appropriate study design remain ongoing. While collaborative, the agency has expressed a preference for a longer duration comparative study over a placebo-controlled trial. Given the shorter time to potential commercial revenue and the similar trial design to the first FLASH study afforded by the EMA accepted protocol, this study is being initiated. At the same time, discussions with the FDA will continue on potential modifications to the development path to adequately address their feedback. Additional supportive studies have demonstrated the utility of longer treatment times (Study RW-HPN-MF-01, see above), the lack of significant systemic exposure to hypericin after topical application (Study HPN-CTCL-02) and its relative efficacy and tolerability compared to Valchlor® (Study HPN-CTCL-04). In addition, the FDA awarded an Orphan Products Development grant to support the investigator-initiated study evaluation of HyBryte™ for expanded treatment in patients with early-stage CTCL, including in the home use setting. The grant, totaling $2.6 million over 4 years, was awarded to the University of Pennsylvania that was a leading enroller in the Phase 3 FLASH study. About Cutaneous T-Cell Lymphoma (CTCL) CTCL is a class of non-Hodgkin's lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as patches and may progress to raised plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas. CTCL constitutes a rare group of NHLs, occurring in about 4% of the more than 1.7 million individuals living with the disease in the U.S. and Europe (European Union and United Kingdom). It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects approximately 31,000 individuals in the U.S. (based on SEER data, with approximately 3,200 new cases seen annually) and approximately 38,000 individuals in Europe (based on ECIS prevalence estimates, with approximately 3,800 new cases annually). About Soligenix, Inc. Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. Our Specialized BioTherapeutics business segment is developing and moving toward potential commercialization of HyBryte™ (SGX301 or synthetic hypericin sodium) as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma (CTCL). With successful completion of the second Phase 3 study, regulatory approvals will be sought to support potential commercialization worldwide. Development programs in this business segment also include expansion of synthetic hypericin (SGX302) into psoriasis, our first-in-class innate defense regulator (IDR) technology, dusquetide (SGX942) for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer, and (SGX945) in Behçet's Disease. Our Public Health Solutions business segment includes development programs for RiVax®, our ricin toxin vaccine candidate, as well as our vaccine programs targeting filoviruses (such as Marburg and Ebola) and CiVax™, our vaccine candidate for the prevention of COVID-19 (caused by SARS-CoV-2). The development of our vaccine programs incorporates the use of our proprietary heat stabilization platform technology, known as ThermoVax®. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID), the Defense Threat Reduction Agency (DTRA) and the Biomedical Advanced Research and Development Authority (BARDA). For further information regarding Soligenix, Inc., please visit the Company's website at and follow us on LinkedIn and Twitter at @Soligenix_Inc. This press release may contain forward-looking statements that reflect Soligenix's current expectations about its future results, performance, prospects and opportunities, including but not limited to, potential market sizes, patient populations, clinical trial enrollment, the expected timing for closing the offering described herein and the intended use of proceeds therefrom. Statements that are not historical facts, such as 'anticipates,' 'estimates,' 'believes,' 'hopes,' 'intends,' 'plans,' 'expects,' 'goal,' 'may,' 'suggest,' 'will,' 'potential,' or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements, and include the expected amount and use of proceeds from the offering and the expected closing date of the offering. Soligenix cannot assure you that it will be able to successfully develop, achieve regulatory approval for or commercialize products based on its technologies, particularly in light of the significant uncertainty inherent in developing therapeutics and vaccines against bioterror threats, conducting preclinical and clinical trials of therapeutics and vaccines, obtaining regulatory approvals and manufacturing therapeutics and vaccines, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that it will be able to successfully obtain any further funding to support product development and commercialization efforts, including grants and awards, maintain its existing grants which are subject to performance requirements, enter into any biodefense procurement contracts with the U.S. Government or other countries, that it will be able to compete with larger and better financed competitors in the biotechnology industry, that changes in health care practice, third party reimbursement limitations and Federal and/or state health care reform initiatives will not negatively affect its business, or that the U.S. Congress may not pass any legislation that would provide additional funding for the Project BioShield program. In addition, there can be no assurance as to the timing or success of any of its clinical/preclinical trials. Despite the statistically significant result achieved in the first HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma or any other studies (including the open-label, investigator-initiated study), there can be no assurance that the second HyBryte™ (SGX301) Phase 3 clinical trial will be successful or that a marketing authorization from the FDA or EMA will be granted. Additionally, although the EMA has agreed to the key design components of the second HyBryte™ (SGX301) Phase 3 clinical trial, no assurance can be given that the Company will be able to modify the development path to adequately address the FDA's concerns or that the FDA will not require a longer duration comparative study. Notwithstanding the result in the first HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma and the Phase 2a clinical trial of SGX302 for the treatment of psoriasis, there can be no assurance as to the timing or success of the clinical trials of SGX302 for the treatment of psoriasis. Additionally, despite the biologic activity observed in aphthous ulcers induced by chemotherapy and radiation, there can be no assurance as to the timing or success of the clinical trials of SGX945 for the treatment of Behçet's Disease. Further, there can be no assurance that RiVax® will qualify for a biodefense Priority Review Voucher (PRV) or that the prior sales of PRVs will be indicative of any potential sales price for a PRV for RiVax®. Also, no assurance can be provided that the Company will receive or continue to receive non-dilutive government funding from grants and contracts that have been or may be awarded or for which the Company will apply in the future. These and other risk factors are described from time to time in filings with the Securities and Exchange Commission (the 'SEC'), including, but not limited to, Soligenix's reports on Forms 10-Q and 10-K. Unless required by law, Soligenix assumes no obligation to update or revise any forward-looking statements as a result of new information or future events. View original content to download multimedia: SOURCE SOLIGENIX, INC.
