On March 23, 2026 Xilio Therapeutics, Inc. (Nasdaq: XLO), a clinical-stage biotechnology company discovering and developing masked immuno-oncology therapies for people living with cancer, reported pipeline progress and business updates and reported financial results for the fourth quarter and full year ended December 31, 2025.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"As we enter 2026, we are well-positioned to leverage our clinically validated masking technology to continue advancing our next generation of multi-specific I-O therapies toward the clinic," said René Russo, Pharm.D., president and chief executive officer of Xilio. "We are encouraged by the progress the field has recently made with masked T cell engagers for prostate cancer, and we are excited to be advancing a potential first-in-class multi-specific masked T cell engager targeting both PSMA and STEAP1 with built-in co-stimulatory signaling designed to enhance potency and durability of response. We believe that targeting both PSMA and STEAP1, which are the most prevalent tumor-associated antigens expressed in prostate cancer, will minimize resistance due to antigen escape. In addition, this quarter we continued to make strong progress advancing XTX501, our potential best-in-class bispecific PD-1 / masked IL-2, toward a planned IND submission mid-year."

Pipeline Progress and Business Updates

XTX501: bispecific PD-1 / masked IL-2

XTX501 is a novel bispecific PD-1 / masked IL-2 designed to selectively stimulate PD-1 positive, antigen-experienced T cells and enhance their function. XTX501 incorporates masking and is designed to overcome IL-2 receptor-mediated clearance, peripheral activity and tolerability issues associated with non-masked IL-2 agents. In preclinical studies, XTX501 demonstrated robust monotherapy activity (including in settings insensitive to PD-1 therapy) and tumor-selective pharmacodynamics consistent with its intended mechanism of action.

•
Xilio is currently advancing XTX501 through investigational new drug (IND)-enabling studies and plans to submit an IND application for XTX501 in the middle of 2026.
•
Xilio plans to initiate a Phase 1 trial for XTX501 in the second half of 2026 and report initial Phase 1 data in the second half of 2027, subject to clearance of the IND by the U.S. Food and Drug Administration.
•
Xilio plans to initially evaluate XTX501 in patients with metastatic non-small cell lung cancer before expanding development to other solid tumor types, including tumors that are insensitive to PD-1 therapy. The company believes XTX501 also has the potential to be a foundational "backbone" therapy for combination treatment with other agents.

Masked T Cell Engager Programs

Xilio is leveraging its proprietary, clinically-validated masking technology to advance two wholly-owned programs for masked T cell engagers, as well as an additional program in collaboration with AbbVie Group Holdings Limited (AbbVie).

Xilio’s masked T cell engagers include molecules with one or more tumor-associated antigen (TAA) binding domains and a CD3 targeting domain, which are designed to release a potent, short half-life T cell engager upon tumor-selective activation (ATACR format), and molecules that include a co-stimulatory domain designed to further enhance potency and durability of the T cell response (SEECR format). Depending on the desired properties that Xilio is seeking to achieve for a particular molecule and TAA(s), Xilio’s modular architecture for its masked T cell engagers enables optionality to: include multiple TAA binding domains; add a co-stimulatory domain; and/or mask the CD3 targeting domain, TAA binding domain(s) and/or the co-stimulatory signaling domain.

•
Xilio is advancing a wholly-owned masked T cell engager program targeting CLDN18.2 (ATACR format). Xilio’s modular design architecture for T cell engagers also enables flexibility to evaluate designs that incorporate masking of the CLDN18.2 binding domain and/or add a co-stimulatory domain (SEECR format) in parallel with advancing the current molecule design. CLDN18.2 is expressed in gastrointestinal cancers (including gastric, pancreatic and esophageal) and lung cancer.
•
Xilio plans to present new preclinical data for its CLDN18.2 program in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting taking place from April 17-22, 2026 (abstract number: 1619).
•
Xilio is advancing a wholly-owned multi-specific, masked T cell engager program targeting PSMA and STEAP1 with built-in co-stimulatory signaling (SEECR format). Xilio anticipates nominating a development candidate in the second quarter of 2026. PSMA and STEAP1 are expressed in most prostate cancer tumors, and targeting both of these TAAs has the potential to address tumor heterogeneity while minimizing the potential for resistance due to antigen escape.
•
Xilio plans to advance these masked T cell engager programs into IND-enabling studies and submit IND applications for each of these programs in 2027.

Efarindodekin alfa: masked IL-12

•
Xilio is evaluating efarindodekin alfa as a monotherapy in an ongoing Phase 2 clinical trial in patients with advanced solid tumors and expects to deliver an option data package to Gilead Sciences, Inc. (Gilead) in the first half of 2027.

Recent Corporate Updates

•
In January 2026, Xilio announced the appointment of Sara Bonstein as chair of the board of directors.
•
In January 2026, Xilio announced the receipt of $35.8 million in gross proceeds from the exercise of Series B warrants, before deducting underwriting discounts and commissions and any offering expenses, including the full exercise of Series B warrants held by Coastlands Capital, Frazier Life Sciences and Gilead. The Series B warrants were issued in connection with a follow-on offering in June 2025.
•
In January 2026, Xilio announced the achievement of a development milestone related to the masked antibody-based immunotherapy program under the company’s collaboration, license and option agreement with AbbVie.

•
In February 2026, Xilio closed a follow-on offering of prefunded warrants for $40.0 million in gross proceeds, before deducting underwriting discounts and commissions and any offering expenses. The offering was led by existing investor Coastlands Capital and included participation from OrbiMed, Perceptive Advisors and Gilead, as well as other new and existing institutional investors.

Year-End and Fourth Quarter 2025 Financial Results

•
Cash Position: Cash and cash equivalents were $137.5 million as of December 31, 2025, compared to $55.3 million as of December 31, 2024. In the fourth quarter of 2025, Xilio received $35.8 million in gross proceeds from Series B warrant exercises and a $17.5 million development milestone payment under its license agreement with Gilead.
•
Collaboration and License Revenue: Collaboration and license revenue was $13.7 million for the quarter ended December 31, 2025, compared to $1.7 million for the quarter ended December 31, 2024. Collaboration and license revenue was $43.8 million for the year ended December 31, 2025, compared to $6.3 million for the year ended December 31, 2024. The year-over-year increase was primarily driven by collaboration and license revenue recognized under the collaboration, license and option agreement and stock purchase agreement that Xilio entered into in February 2025 with AbbVie and an increase in collaboration and license revenue recognized under the license agreement with Gilead due to the achievement of a $17.5 million development milestone during the third quarter of 2025.
•
Research & Development (R&D) Expenses: R&D expenses were $18.1 million for the quarter ended December 31, 2025, compared to $8.8 million for the quarter ended December 31, 2024. R&D expenses were $56.0 million for the year ended December 31, 2025, compared to $41.2 million for the year ended December 31, 2024. The year-over-year increase was primarily driven by manufacturing activities related to IND-enabling studies and preclinical development activities for XTX501, increased clinical development activities related to efarindodekin alfa, increased costs related to masked T cell engager programs and indirect research and development and increased personnel-related costs, which were partially offset by a decrease in costs related to vilastobart and XTX202.
•
General & Administrative (G&A) Expenses: G&A expenses were $7.4 million for the quarter ended December 31, 2025, compared to $6.5 million for the quarter ended December 31, 2024. G&A expenses were $29.7 million for the year ended December 31, 2025, compared to $24.8 million for the year ended December 31, 2024. The year-over-year increase was primarily driven by an increase in professional and consulting fees, including legal fees and other professional costs, and an increase in personnel-related costs, which were partially offset by a decrease in costs related to directors’ and officers’ liability insurance.
•
Net Income (Loss): Net income was $10.4 million for the quarter ended December 31, 2025, compared to a net loss of $13.1 million for the quarter ended December 31, 2024. Net loss was $35.0 million for the year ended December 31, 2025, compared to $58.2 million for the year ended December 31, 2024. The year-over-year decrease in net loss was primarily driven by increased collaboration and license revenue for the year ended December 31, 2025.

Cash Runway

Based on its current operating plans, Xilio anticipates that its existing cash and cash equivalents will be sufficient to enable it to fund its operating expenses and capital expenditure requirements through the end of 2027.

This estimate excludes any potential future milestone payments, option-related fees or other contingent payments under Xilio’s collaboration and partnership agreements with AbbVie and Gilead and excludes up to $36.2 million in additional gross proceeds in the second half of 2026 if all outstanding Series C warrants are exercised at their current exercise price.

(Press release, Xilio Therapeutics, MAR 23, 2026, View Source [SID1234663836])

On March 23, 2026 Tempest Therapeutics, Inc. (Nasdaq: TPST) (the "Company"), a clinical-stage biotechnology company developing a pipeline of advanced CAR-T cell therapy product candidates to treat cancer, reported that it has entered into definitive agreements for the purchase and sale of an aggregate of 925,927 shares of common stock (or pre-funded warrant in lieu thereof), series A warrants to purchase up to 925,927 shares of common stock and short-term series B warrants to purchase up to 925,927 shares of common stock, at a combined purchase price of $2.16 per share of common stock (or $2.159 per pre-funded warrant in lieu thereof) and accompanying warrants in a private placement. The series A warrants and the short-term series B warrants will have an exercise price of $2.16 per share and will be exercisable beginning on the effective date of stockholder approval of the issuance of the shares issuable upon exercise of the warrants (the "Stockholder Approval Date"). The series A warrants will expire five years from the later of the Stockholder Approval Date and the Effectiveness Date (as defined below) and the short-term series B warrants will expire twenty-four months from the later of the Stockholder Approval Date and the Effectiveness Date. The private placement is expected to close on or about March 23, 2026, subject to the satisfaction of customary closing conditions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The gross proceeds from the offering are expected to be approximately $2 million, prior to deducting placement agent fees and other offering expenses payable by the Company. The potential additional gross proceeds to the Company from the series A warrants and the short-term series B warrants, if fully exercised on a cash basis, will be approximately $4 million. No assurance can be given that any of the series warrants will be exercised, or that the Company will receive cash proceeds from the exercise of the series warrants. The Company intends to use the net proceeds from the offering for working capital and other general corporate purposes.

The securities described above are being offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Securities Act"), and/or Regulation D promulgated thereunder and, along with the shares of common stock underlying the warrants, have not been registered under the Securities Act, or applicable state securities laws. Accordingly, the securities issued in the private placement and shares of common stock underlying the warrants may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. Pursuant to a registration rights agreement with the investors, the Company has agreed to file a resale registration statement covering the securities described above (such date of effectiveness of the resale registration statement, the "Effectiveness Date").

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

(Press release, Tempest Therapeutics, MAR 23, 2026, View Source [SID1234663835])

On March 23, 2026 Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), reported its financial results for the full year 2025 and recent business highlights.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2026 is poised to be a pivotal year, propelled by disciplined clinical execution and initial data that we believe will showcase the vast potential of our proprietary ADC platform," said Jane Chung, Sutro’s Chief Executive Officer. "We recently completed dosing the third cohort in the Phase 1 trial of STRO-004, our potential best-in-class Tissue Factor ADC, and look forward to reporting initial data mid-year. In parallel, we are advancing STRO-006, our ITGB6 ADC, and accelerating STRO-227, our wholly owned dual-payload program targeting PTK7, toward IND submission this year. In addition, patient dosing has commenced under our collaboration with Astellas Pharma for our first partnered dual-payload iADC — marking the first dual-payload program from Sutro’s platform to enter the clinic. Supported by our recent financing, continued financial stewardship, and sharpened strategic focus, we believe we are well positioned to execute across all our programs and deliver differentiated ADCs with best-in-class potential that could redefine standards of care in oncology."

Wholly Owned Pipeline

STRO-004: The Company has completed dosing across three cohorts in the first-in-human Phase 1 trial evaluating STRO-004, a DAR8 Topo1 ADC targeting Tissue Factor (TF), with potential as best-in-class TF ADC. Initial clinical data are expected in mid-2026, including safety and tolerability. Sutro also expects to share pharmacokinetic exposure and potentially early signs of activity. In non-human primate studies, STRO-004 demonstrated a favorable preclinical safety profile, with a highest non-severely toxic dose (HNSTD) of 50 mg/kg — supporting a clinical starting dose of 1mg/kg.

STRO-006: A highly selective, DAR 8 Topo1 ADC targeting integrin β6 (ITGB6), STRO-006 is expected to enter clinical development in 2026 for the treatment of multiple solid tumors.

STRO-227: Sutro’s PTK7-targeting dual-payload ADC program, consisting of MMAE (DAR2) and Topo1 (DAR8), remains on track, with an IND submission targeted for 2026.

Next-Generation ADC Collaborations

Astellas: Two research and development programs are progressing under Sutro’s collaboration with Astellas focused on dual-payload immunostimulatory ADCs (iADCs).

•
The first program, targeting TROP2, has entered the clinic and is actively dosing patients, triggering a $10 million milestone payment with receipt expected in the second quarter of 2026.
•
The second program entered an IND-enabling toxicology study in the fourth quarter of 2025, triggering a $7.5 million milestone payment to Sutro.

Upcoming and Recent Industry/Medical Conferences

American Association for Cancer Research (AACR) (Free AACR Whitepaper), April 17-22, 2026, San Diego California

•
Sutro’s strategic partner, Astellas Pharma, will present preclinical results from its TROP2-targeted iADC program in an oral presentation on Sunday, April 19, 2026 at 4:35-4:50 PM PT. The presentation, titled "ASP2998, a TROP2-targeted immunostimulatory antibody-drug conjugate (iADC) with dual payloads, demonstrates potent efficacy and a favorable safety profile in nonclinical models," highlights progress in the development of next-generation iADCs leveraging Sutro’s cell-free protein synthesis platform.
•
Additionally, Sutro will present an oral presentation and multiple posters highlighting advances across its ADC pipeline and discovery platforms at AACR (Free AACR Whitepaper). The presentation details are as follows:
o
Presentation: "STRO-004, an exatecan-based next-generation tissue factor (TF)-targeted ADC, demonstrates superior efficacy across TF-expressing solid tumors in a comprehensive single-mouse PDX trial"
•
Presentation Date and Time: Sunday, April 19, 2026; 4:35-4:50 PM PT
o
Poster: "Phase 1 open-label study to evaluate safety, pharmacokinetics, and preliminary anti-tumor activity of STRO-004 in adults with refractory/recurrent metastatic solid tumors"
•
Session Date and Time: Monday, April 20, 2026; 9:00 AM-12:00 PM PT
o
Poster: "STRO-006: An Integrin beta-6–targeting ADC demonstrates favorable safety profile and potent antitumor activity in preclinical solid tumors"
•
Session Date and Time: Monday, April 20, 2026; 9:00 AM-12:00 PM PT
o
Poster: "Preclinical characterization of STRO-227: A PTK7-targeting dual-payload ADC with topoisomerase 1 and tubulin inhibitors"
•
Session Date and Time: Monday, April 20, 2026; 9:00 AM-12:00 PM PT
o
Poster: "The HER2-targeting dual-payload antibody-drug conjugate combining a topoisomerase I inhibitor and a microtubule inhibitor demonstrates superior efficacy and overcomes resistance to single-payload ADCs in xenograft models"
•
Session Date and Time: Monday, April 20, 2026; 9:00 AM-12:00 PM PT
o
Poster: "Sutro’s Site-Specific Dual-Payload ADCs Combining TOPO1i and DNA Damage Response Inhibitors to Enhance Efficacy, Overcome Resistance, and Improve Safety"
•
Session Date and Time: Tuesday, April 21, 2026; 9:00 AM-12:00 PM PT

16th World ADC London Summit, February 23-26, 2026, London, UK

•
Sutro participated in a plenary and two panel discussions at the conference, covering topics including dual-payload innovation, key drivers of ADC differentiation, and ADC collaborations. For more details, read the full press release here.

Corporate Updates

•
Sutro strengthened its cash position with an underwritten offering of 7,868,383 shares of its common stock at a price of $13.98 per share, resulting in gross proceeds of $110.0 million, before deducting underwriting discounts and commissions and other offering expenses payable by Sutro. The Company’s cash runway is now expected into at least the second quarter of 2028, excluding additional anticipated milestones from Sutro’s existing collaboration.
•
Sutro management, joined by KOL Anthony Tolcher, M.D., FRCPC, FACP, hosted a virtual R&D Day on Wednesday, November 12, highlighting the Company’s platform innovations and next-generation ADC pipeline. Key updates included the initiation of the Phase 1 study with STRO-004 and the selection of PTK7 as the target for its initial dual-payload candidate, STRO-227.

Full Year 2025 Financial Highlights

Cash, Cash Equivalents and Marketable Securities

As of December 31, 2025, Sutro had cash, cash equivalents and marketable securities of $141.4 million, as compared to $167.6 million as of September 30, 2025. With gross proceeds from the recent financing totaling $110.0 million, the Company’s cash runway is expected into at least the second quarter of 2028, excluding anticipated milestones from Sutro’s existing collaborations.

Revenue

Revenue was $102.5 million for the year ended December 31, 2025, as compared to $62.0 million for the year ended December 31, 2024, with the 2025 amount related principally to the Astellas and Ipsen collaborations. Future collaboration and license revenue under existing agreements, and from any additional collaboration and license partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones, and other agreement payments.

Research & Development (R&D) and General & Administrative (G&A) Expenses

Total R&D and G&A expenses for the year ended December 31, 2025 were $207.4 million, as compared to $300.5 million for the year ended December 31, 2024. The 2025 year includes non-cash expenses for stock-based compensation of $14.0 million and depreciation and amortization of $7.3 million, as compared to $24.7 million and $7.2 million, respectively, in the comparable 2024 period.

Restructuring Costs

The total cash payments and costs related to the further operational restructuring announced on September 29, 2025 are estimated to be approximately $4.1 million to $4.3 million, with a majority of these amounts paid in the fourth quarter of 2025. These estimates are subject to a number of assumptions and actual results may differ. The Company may also incur additional costs not currently contemplated due to events that may occur as a result of, or that are associated with, the corporate restructuring.

(Press release, Sutro Biopharma, MAR 23, 2026, View Source [SID1234663834])

On March 23, 2026 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, reported that findings from recent supportive trials with HyBryte (synthetic hypericin) in the treatment of cutaneous T-cell lymphoma (CTCL) are being presented at the United States Cutaneous Lymphoma Consortium (USCLC) Workshop (March 26, 2026), which precedes the American Academy of Dermatology (AAD) Annual Meeting. 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 the Principal Investigator for the first Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study as well as the ongoing Phase 3 FLASH2 study, will present at the USCLC. Dr. Kim will detail positive results from the recently completed investigator-initiated study using HyBryte as a long-term treatment of CTCL. A poster also will be presented at the conference sharing the positive results of a study evaluating HyBryte versus Valchlor (mechlorethamine) conducted by Brian Poligone, MD, PhD, Director of the Rochester Skin Lymphoma Medical Group and Principal Investigator for the comparability study. The official conference program can be found here.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Oral Presentation:

Title: Phase 2 Investigator-Initiated Real-World Study Evaluating Topical Hypericin Ointment Photodynamic Therapy for Early-Stage Mycosis Fungoides/CTCL (RW-HPN-MF-01) presented by Dr. Ellen Kim, Director, Penn Cutaneous Lymphoma Program, Vice Chair of Clinical Operations, Dermatology Department, and Professor of Dermatology at the Hospital of the University of Pennsylvania.

Poster Presentation:

Title: Results from a Pilot Study of HyBryte (topical synthetic hypericin) versus Valchlor (mechlorethamine) in the Treatment of CTCL attended by Dr. Christopher Pullion, Medical Director, Soligenix, Inc.

The poster and presentation review the Company’s findings in recent supportive studies, which have demonstrated the clinical benefits of longer treatment times (Study RW-HPN-MF-01; investigator-initiated study), as well as HyBryte’s relative efficacy and tolerability compared to Valchlor (Study HPN-CTCL-04).

About the USCLC Workshop

The United States Cutaneous Lymphoma Consortium is a multidisciplinary society of physicians which use collaborative research and education to improve the quality of life and prognosis of patients with cutaneous lymphoma. This workshop is held annually to facilitate collaboration. The meeting website is available here.

About the AAD Annual Meeting

The American Academy of Dermatology Association Annual Meeting is one of the largest dermatologic scientific meetings globally and is attended by both researchers and dermatologists. The meeting website is available here.

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 modified Composite Assessment of Index Lesion Severity [mCAILS] 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.

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, has successfully achieved its first safety review milestone with a pre-specified, blinded interim analysis expected to be completed in 2Q2026. This study is a randomized, double-blind, placebo-controlled, multicenter study that will enroll approximately 80 subjects with early-stage CTCL. The FLASH2 study 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 was 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), 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 [Surveillance, Epidemiology, and End Results] data, with approximately 3,200 new cases seen annually) and approximately 38,000 individuals in Europe (based on ECIS [European Cancer Information System] prevalence estimates, with approximately 3,800 new cases annually).

(Press release, Soligenix, MAR 23, 2026, View Source [SID1234663832])

On March 23, 2026 Rakovina Therapeutics Inc. (TSX-V: RKV)(FSE: 7JO0), a biopharmaceutical company advancing innovative cancer therapies through artificial intelligence (AI)-powered drug discovery, reported that two research abstracts have been accepted for presentation at the upcoming 2026 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place April 17-22 in San Diego, California.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The AACR (Free AACR Whitepaper) Annual Meeting is a global forum for cancer research, where the world’s leading scientists, clinicians, and biotech innovators gather to unveil next-generation oncology breakthroughs. Rakovina’s acceptance to present highlights growing recognition for its work at the intersection of AI technology and precision cancer therapy.

From AI Design to Delivery: Advancing Novel DDR Therapies for Hard-to-Treat Cancers

Rakovina will present two abstracts at the meeting. The first abstract: A Novel Brain-Penetrant Dual ATR-mTOR Inhibitor for PTEN-Deficient Cancers, presents the use of the Enki generative AI platform to design first-in-class CNS-penetrating molecules that simultaneously inhibit ATR and mTOR, two key drivers of survival in PTEN-deficient cancer cells. PTEN deficiency is found in up to 40% of gliomas and up to 63% of breast cancers, which frequently metastasize to the brain, yet no approved therapy directly addresses this dual vulnerability. Using the Enki latent diffusion model to simultaneously optimize potency, selectivity, CNS penetrance, and ADMET properties in collaboration with Variational AI (Vancouver, BC), the team identified and synthesized promising dual inhibitor candidates, with data on efficacy and pharmacokinetics to be presented at the meeting.

Title: A novel brain-penetrant dual ATR-mTOR inhibitor for PTEN-deficient cancers
Date/Time: April 20, 2026 / 9:00 AM – 12:00 PM
Session Category: Experimental and Molecular Therapeutics
Session: DNA Damage and Repair 2
Abstract Number: 5034

The second abstract: Development of a Lipid Nanoparticle Formulation of the Bifunctional PARP and HDAC Inhibitor Kt-3283, presents preclinical development work on kt-3283, Rakovina’s bifunctional compound that simultaneously inhibits PARP and HDAC enzymes, eliminating the need for combination drug regimens and their associated toxicity risks.

In vitro studies with kt-3283 have demonstrated highly potent anti-tumor activity across multiple tumor types, supporting its potential as a differentiated therapeutic candidate. This abstract presents a novel advancement: encapsulation of kt-3283 into patterned lipid nanoparticles (pLNPs) designed using the EnsaliX AI platform, developed in collaboration with NanoPalm (Riyadh, Saudi Arabia). The pLNP/kt-3283 formulation is designed to enhance encapsulation efficiency, improve cell uptake, and optimize pharmacological performance to support clinical translation.

Title: Development of a lipid nanoparticle formulation of the bifunctional PARP and HDAC inhibitor kt-3283
Date/Time: April 21, 2026 / 2:00 PM – 5:00 PM
Session Category: Chemistry
Session: Drug Delivery
Abstract Number: 4665

"We are thrilled that for the second year in a row, we are presenting two abstracts at the AACR (Free AACR Whitepaper) Annual Meeting, an important stage for cancer research," said Kim Oishi, Chief Executive Officer of Rakovina Therapeutics. "These programs reflect the breadth and ambition of what our team is building. From improving how we deliver our molecules into the body, to designing entirely new inhibitors that can reach tumors in the brain, we are using AI to solve problems that have held back cancer drug development for years. We look forward to sharing our findings with the global oncology community in San Diego."

The company’s integration of AI platforms allows the evaluation of billions of potential compounds at 100x the speed of traditional methods. These innovations are further supported by Rakovina’s access to the University of British Columbia’s state-of-the-art wet lab infrastructure, enabling rapid in-house testing and optimization.

"What makes these two programs particularly exciting is that they address cancer from two distinct but complementary angles, one focused on smarter delivery of a proven bifunctional compound, and one on designing entirely new molecules to reach tumors that current therapies simply cannot access," said Dr. Mads Daugaard, President and Chief Scientific Officer of Rakovina Therapeutics.

Rakovina is driving innovation in a space projected to reach $18 billion annually by 2030. The company’s preclinical pipeline is focused on therapies that target DNA-repair vulnerabilities present in up to 75% of solid tumors, with an emphasis on hard-to-treat cancers such as breast, ovarian, prostate, and brain cancers. With AACR (Free AACR Whitepaper) presentations underway in Q2, the Company is targeting several near-term milestones across its pipeline, including in vivo testing of its new LNP formulations under the kt-3000 program, advancement of a lead in the kt-5000AI program through iterative AI-driven compound refinement and initiation of pharma partnership discussions as pipeline data matures. Rakovina expects these catalysts to meaningfully de-risk its programs and support its path toward IND-enabling studies.

(Press release, Rakovina Therapeutics, MAR 23, 2026, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-to-present-new-data-at-the-aacr-annual-meeting-2026-worlds-premier-cancer-research-forum [SID1234663831])