On April 22, 2026 TuHURA Biosciences, Inc. (NASDAQ:HURA) ("TuHURA" or the "Company"), a Phase 3 immuno-oncology company developing novel therapeutics to overcome resistance to cancer immunotherapy, reported that it has entered into a loan agreement providing a credit facility of up to $50 million in funding to support the Company’s pipeline, ongoing clinical trials, and general corporate expenses. The lender is an affiliate of the Company’s largest stockholder, K&V Investment One LLC.

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Under the terms of the loan agreement, TuHURA will have the ability to draw down monthly on the facility on an as-needed basis to fund monthly expenses for ongoing clinical development and operations. The facility bears a 12% annual interest rate on outstanding funds drawn, with interest paid monthly and principal repayment due at a 5-year maturity date of April 21, 2031. The loan facility is secured by the assets of the Company and its subsidiaries. In connection with the credit facility, the Company granted the lender a low to mid-single digit percentage royalty on annual commercial sales by the Company or its sublicensees of products based on IFx-2.0.

"We are gratified to have established this non-equity based source of operating capital on what we believe are attractive terms for a company such as TuHURA. This agreement allows us to fund operations through anticipated key milestones this year and beyond through anticipated top-line Phase 3 results of our lead IFx-2.0 program. Importantly, we control the timing and amount of funds drawn under this facility while preserving the ability to be opportunistic in securing other potential sources of capital, including corporate partnerships or equity financings," said Dr. James Bianco, President and Chief Executive Officer of TuHURA Biosciences. "We believe that it is unusual to access such an attractive source of capital in advance of a BLA submission or pending FDA approval. This funding is a testament to the conviction our largest shareholder has in our strategy and in the potential for the clinical and commercial success of IFx-2.0."

Additional information regarding the credit facility and royalty agreement, including the terms and provisions of the loan agreement, can be found in the Company’s Current Report on Form 8-K filed today with the Securities and Exchange Commission.

(Press release, TuHURA Biosciences, APR 22, 2026, View Source [SID1234664692])

On April 22, 2026 Tempest Therapeutics, Inc. (Nasdaq: TPST) ("Tempest"), a clinical-stage biotechnology company developing a pipeline of advanced CAR-T cell therapy product candidates to treat cancer, reported that it has achieved a key milestone in the development of TPST-2003, Tempest’s dual-targeting CD19/BCMA CAR-T therapy under development for the treatment of relapsed/refractory multiple myeloma ("rrMM"). Earlier this month, Tempest’s manufacturing partner, Cincinnati Children’s Applied Gene and Cell Therapy Center ("AGCTC"), took delivery of the TPST-2003 lentiviral vector, a critical component used in the manufacturing of TPST-2003. This milestone supports Tempest’s plans to initiate the first potentially registrational study to evaluate a dual-targeting CAR-T therapy in patients with rrMM, including patients who are experiencing extramedullary disease ("EMD"), later this year.

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Tempest recently announced that, as of a January 31, 2026 data cutoff, a total of 36 patients with rrMM had received one infusion of TPST-2003, including 24 patients in a prior Phase 1/2 investigator-initiated trial ("IIT") and 12 patients in the ongoing REDEEM-1 trial, representing one of the largest datasets evaluating a CD19/BCMA dual-targeting CAR-T therapy. As of the data cutoff, all six efficacy evaluable patients enrolled in the REDEEM-1 trial had achieved a complete response according to the International Myeloma Working Group uniform response criteria. Among 25 evaluable patients with measurable disease at baseline across both studies, the overall response rate was 100% (25/25). The IIT also demonstrated durable disease control, with median progression-free survival ("PFS") of 23.1 months across all patients and median PFS of 23.1 months in patients with EMD. Tempest plans to present the results of the REDEEM-1 trial and updated results from the IIT at a scientific meeting later this year.

"We are pleased by the rapid progress we have been making in partnership with AGCTC," said Dr. Matt Angel, President and Chief Executive Officer of Tempest. "The delivery of lentiviral vector, which is a critical component in the manufacturing of autologous CAR-T products, has enabled us to proceed with the manufacturing activities required for the pivotal development of TPST-2003. We are grateful for our partnership with AGCTC, and we are looking forward to continued rapid progress toward the initiation of a potentially registrational study for TPST-2003 later this year."

AGCTC is a research, development, and manufacturing hub advancing future cell and gene therapy (CGT) treatments for patients with unmet needs. Established in 2001, the center has evolved into a nationally recognized leader in CTG CDMO services with a proven track record that reflects Cincinnati Children’s commitment to solving unmet medical needs through translational science. AGCTC is part of the Cincinnati Children’s Cancer and Blood Diseases Institute, which is ranked #1 in the nation by U.S. News & World Report for pediatric cancer care.

"We are excited to have achieved this important milestone in the development of TPST-2003," said Dr. Chaozhong Zou, Executive Director and General Manager of AGCTC, Cancer and Blood Diseases Institute, Cincinnati Children’s Hospital Medical Center. "The clinical data generated so far support the idea that the parallel-structure dual-targeting CAR architecture of TPST-2003 could offer patients with rrMM a meaningful new treatment option, and we are grateful to be in position to support the development of TPST-2003 by leveraging our extensive experience making novel CAR-T programs IND-ready. We look forward to generating the information needed to support the pivotal development of TPST-2003."

About TPST-2003

TPST-2003 is an autologous CD19/BCMA dual-targeting CAR-T therapy designed to improve response depth and durability in patients with relapsed/refractory multiple myeloma ("rrMM") through a parallel dual-targeting CAR structure designed to address tumor heterogeneity and antigen escape. TPST-2003 is being developed in China by Tempest’s partner, Novatim Immune Therapeutics ("Novatim"). Under its agreement with Novatim, Tempest has the exclusive right to develop TPST-2003 outside of China, India, Turkey, and Russia.

About REDEEM-1

REDEEM-1 (Study nos. CTR20233309/NCT06223646) is a Phase 1/2a clinical trial evaluating TPST-2003 in patients with relapsed/refractory multiple myeloma, including patients with high-risk cytogenetics and patients with extramedullary disease. The REDEEM-1 trial has a targeted full enrollment of 29 patients. The REDEEM-1 trial is sponsored and being conducted by Tempest’s partner, Novatim Immune Therapeutics, with a total of eight clinical sites registered in China: Peking Union Medical College Hospital (Dr. Jian Li; lead site), The First Affiliated Hospital of Nanchang University (Dr. Fei Li), Peking University First Hospital (Dr. Yujin Dong), Henan Cancer Hospital (Dr. Baijun Fang), Shanxi Provincial Cancer Hospital (Dr. Liping Su), The Second Xiangya Hospital of Central South University (Dr. Hongling Peng), The First Affiliated Hospital of China Medical University (Dr. Xiaojing Yan), and The Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College (Dr. Dehui Zou).

Additional clinical trials evaluating TPST-2003

A Phase 1/2 IIT (Study no. NCT04714827) is evaluating TPST-2003 in patients with relapsed/refractory multiple myeloma, including patients with high-risk cytogenetics and patients with extramedullary disease. The IIT is sponsored and being conducted by Tempest’s partner, Novatim, with a total of two clinical sites registered in China: Shanghai Fourth People’s Hospital (Dr. Weijun Fu; lead site) and Shanxi Provincial Cancer Hospital (Dr. Liping Su).

A Phase 1 trial (Study nos. CTR20242409/NCT06518876) is evaluating TPST-2003 in patients with POEMS, a rare blood disorder caused by abnormal plasma cells. The Phase 1 trial is sponsored and being conducted by Tempest’s partner, Novatim, with a total of three clinical sites registered in China: Peking Union Medical College Hospital (Dr. Jian Li; lead site), Xuanwu Hospital Capital Medical University (Dr. Wanling Sun), and West China Hospital, Sichuan University (Dr. Yu Wu).

(Press release, Tempest Therapeutics, APR 22, 2026, View Source [SID1234664691])

On April 22, 2026 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported the independent presentation of new preclinical data at the 2026 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, showing positive effects of opaganib as potential add-on therapy in models of neuroblastoma (NB) and triple-negative breast cancer (TNBC).

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The positive NB data, from studies undertaken by Penn State University’s Jeremy Hengst, PhD, and Apogee Biotechnology funded by the Beat Childhood Cancer Foundation and Four Diamonds, indicate that opaganib may enhance the therapeutic efficacy of the oxaliplatin + doxorubicin (OXDOX) chemotherapy combination in high-risk NB. The data showed that opaganib directly destabilized n-Myc, a key oncogenic driver of neuroblastoma and other solid tumors, regulating cell proliferation, differentiation, and apoptosis during embryonic development, a critical factor driving poor outcomes.

A second poster from the University of Kansas’ Colette Worcester describes in vitro model data showing that pre-treatment with opaganib, followed by low-dose diABZI treatment, potentiated the downstream STING-mediated effects and may augment anti-tumor immunity in TNBC, which has the poorest prognosis of the breast cancer subtypes.

Dr. Mark Levitt, Chief Scientific Officer at RedHill said: "These data represent exciting findings that could hold promise for improving outcomes in treating pediatric NB and TNBC, providing additional encouragement for further exploration. Opaganib has previously shown potential as add-on therapy in several preclinical oncology models in combination with chemotherapy. Moreover, the ongoing Phase 2 clinical study of opaganib in combination with darolutamide in advanced prostate cancer could potentially provide paradigm-shifting clinical data in support of the additive use of opaganib in a cancer setting."

Neuroblastoma is the most common infancy cancer with ~5,500 global pediatric cases per year in children aged 0–14. It accounts for 10% of childhood cancers and 15% of pediatric cancer-related deaths in the U.S.5,6 Opaganib received FDA Orphan Drug and Rare Pediatric Disease designations for the treatment of neuroblastoma, a rare pediatric cancer, with potential for a Rare Pediatric Disease Priority Review Voucher ("PRV"). Development discussions for this indication are ongoing with Penn State University and the Beat Childhood Cancer consortium.

About Opaganib (ABC294640)

Opaganib is a proprietary first-in-class investigational, orally administered sphingosine kinase-2 (SPHK2) selective inhibitor drug. Potentially broad-acting, it is in development for multiple oncology, viral, inflammatory, metabolic (diabetes and obesity) and additional indications.

Peer-reviewed data, published in the journal Diabetes, Metabolic Syndrome and Obesity7, provides evidence that opaganib uniquely works through the inhibition of multiple pathways implicated in insulin resistance, β-cell disruption, adipocyte function, inflammation / immune regulation, vascular complications, energy metabolism, induction of autophagy and apoptosis, and disruption of viral replication, through simultaneous inhibition of three sphingolipid-metabolizing enzymes in human cells (SPHK2, DES1 and GCS).

Opaganib has received Orphan Drug designation from the FDA for the treatment of neuroblastoma and cholangiocarcinoma. A Bayer-supported 80-patient placebo-controlled randomized Phase 2 study is ongoing to evaluate the efficacy of opaganib in combination with Bayer’s darolutamide in men with metastatic castrate-resistant prostate cancer (mCRPC), testing the potentially enhancing effect of opaganib in patients with a poor prognosis8. Opaganib also has a Phase 1 chemoradiotherapy study protocol ready for FDA-IND submission.

Opaganib has demonstrated its safety and tolerability profile in more than 470 people in multiple clinical studies and expanded access use, including a large global Phase 2/3 study in hospitalized patients with moderate to severe COVID-19, published in Microorganisms.

(Press release, RedHill Biopharma, APR 22, 2026, View Source [SID1234664690])

On April 22, 2026 Rakovina Therapeutics Inc. (TSX-V: RKV; FSE: 7JO0), a biopharmaceutical company advancing innovative cancer therapies through artificial intelligence (AI)-powered drug discovery, reported the presentation of new preclinical data from two of its lead programs at the 2026 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, held April 17–22 in San Diego, California.

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The data, presented across two poster sessions at the world’s premier cancer research forum, advance Rakovina’s AI-driven pipeline targeting DNA damage response (DDR) vulnerabilities in hard-to-treat solid tumors. Both programs leverage generative AI platforms to address longstanding limitations of existing cancer therapies, including poor central nervous system (CNS) penetrance and the toxicity burden of drug combination regimens.

Novel Brain-Penetrant Dual ATR-mTOR Inhibitor Demonstrates In Vivo Efficacy in PTEN-Deficient Cancers

The first poster, titled A Novel Brain-Penetrant Dual ATR-mTOR Inhibitor for PTEN-Deficient Cancers (Presentation #1743, DNA Damage and Repair 2 session, April 20), presented preclinical data from Rakovina’s program to develop first-in-class CNS-penetrating molecules that simultaneously inhibit ATR and mTOR, two key drivers of survival in PTEN-deficient cancer cells. The program was developed in collaboration with Variational AI (Vancouver, BC) using the Enki generative AI platform.

PTEN deficiency is found in up to 40% of gliomas and 63% of breast cancers, which frequently metastasize to the brain. Simultaneous inhibition of ATR and mTOR is a rational therapeutic strategy in PTEN-deficient tumors, as PTEN loss activates both ATR-dependent DNA damage signaling and mTOR-driven cell survival pathways. However, no approved therapy directly addresses this dual vulnerability with effective CNS penetrance.

Using the Enki latent diffusion model to simultaneously optimize potency, selectivity, CNS penetrance, and ADMET properties, Rakovina generated and synthesized a curated set of novel small-molecule dual ATR-mTOR inhibitor candidates. Key findings presented at AACR (Free AACR Whitepaper) 2026 include:

Enzymatic potency: Candidate compounds demonstrated equal or greater inhibition of recombinant ATR and mTOR enzymes compared to reference compounds ceralasertib and tuvusertib.
Selectivity: Candidates are equally or more selective against PIKK family enzymes than the reference compounds ceralasertib and tuvusertib.
Cell viability inhibition: Candidates inhibit cell viability of D283 medulloblastoma cells equally or more than reference compounds. A prototype lead candidate inhibited cell viability of both PTEN wild-type and PTEN-deficient cancer cell lines.
Metabolic stability: After 45 minutes of incubation with human liver microsomes, candidate compounds demonstrated strong metabolic stability.
CNS penetrance: Pharmacokinetic profiling following intraperitoneal administration in mice confirmed varying but measurable levels of CNS penetrance across candidates, with brain-to-plasma ratios broadly consistent with Enki AI predictions.
In vivo efficacy: In a subcutaneous LNCaP prostate tumor model, a prototype lead candidate significantly prolonged tumor doubling time compared to vehicle control, with equal potency to reference compound ceralasertib. Critically, the Rakovina candidate was better tolerated than ceralasertib, demonstrating less weight loss with daily dosing and no signs of hematological toxicity at terminal complete blood count analysis.
Optimization of candidate inhibitors is ongoing.

Novel AI-Designed Lipid Nanoparticle Formulation of kt-3283 Successfully Characterized

The second poster, titled Development of a Lipid Nanoparticle Formulation of the Bifunctional PARP and HDAC Inhibitor Kt-3283 (Presentation #6373, Drug Delivery session, April 21), presented preclinical formulation data on pLNP/kt-3283, developed in collaboration with NanoPalm (Riyadh, Saudi Arabia) using the EnsaliX AI platform.

kt-3283 integrates PARP inhibition and HDAC-mediated chromatin remodeling into a single compound, thereby improving the PARP efficacy, and eliminating the need for combination drug regimens and their associated toxicity risks. While kt-3283 has demonstrated potent anti-tumor activity across multiple tumor types in prior in vitro studies, its clinical viability has been limited by bioavailability and metabolic stability challenges. The pLNP formulation has been specifically designed to address these limitations.

Data presented confirm the successful assembly of the EnsaliX-designed patterned lipid nanoparticles. Physicochemical characterization confirmed uniform particle size, stable colloidal behavior, and a structured surface texture predicted to enhance cellular uptake. The pLNP/kt-3283 formulation demonstrated structure and particle size consistency supporting further biological evaluation.

Next steps include in vitro and in vivo characterization to confirm activity against PARP and HDAC enzymes, determine ADME properties, and evaluate efficacy in tumor models.

"Presenting at AACR (Free AACR Whitepaper) is a meaningful milestone for our team, and these results represent a genuine step forward for both programs," said Kim Oishi, Chief Executive Officer of Rakovina Therapeutics. "The in vivo efficacy data for our ATR-mTOR inhibitor are particularly encouraging. The compound demonstrated potency comparable to an established reference compound while exhibiting a meaningfully improved tolerability profile. That is exactly the differentiation we are building toward. Combined with the initial characterization of our LNP formulation for kt-3283, we believe these results reinforce the potential of our AI-driven pipeline and support a path toward IND-enabling studies."

Rakovina’s AI-powered discovery approach leverages generative AI platforms to evaluate billions of potential drug candidates at a pace not achievable through traditional methods. These capabilities are supported by the company’s access to the University of British Columbia’s lab infrastructure, enabling rapid in-house testing of lead compounds.

"These results demonstrate that our strategy of integrating AI-guided design with biological validation, is working as intended," said Dr. Mads Daugaard, President and Chief Scientific Officer of Rakovina Therapeutics. "For the ATR-mTOR program, our candidate inhibitors are tracking closely with the AI predictions for potency, selectivity, and CNS penetrance and our in vivo results give confidence in the direction of this program. For kt-3283, we have demonstrated that the EnsaliX-designed LNP formulation produces a well-characterized nanoparticle. The structured surface and organized phospholipid assembly we observed are precisely the properties expected to enhance nanoparticle stability and cellular uptake of kt-3283. Both programs have clear next steps, and we are moving forward with purpose."

The data presented at AACR (Free AACR Whitepaper) 2026 reinforce the progress of Rakovina’s AI-enabled DDR inhibitor pipeline and inform the next phase of preclinical development for both programs. For the ATR-mTOR program, further optimization of candidate inhibitors is ongoing. For the kt-3283 LNP program, the company will advance in vitro and in vivo studies to further characterize biological activity prior to evaluating efficacy in tumor models.

Rakovina intends to use these findings to advance best-in-class lead candidates toward IND-enabling studies in collaboration with pharmaceutical partners.

(Press release, Rakovina Therapeutics, APR 22, 2026, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-presents-new-preclinical-data-at-aacr-2026-annual-meeting [SID1234664689])

On April 22, 2026 PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value, reported positive topline data from the completed Phase 1b clinical trial of LYT-200, a first-in-class, fully human anti-galectin-9 monoclonal antibody, in heavily pretreated patients with relapsed/refractory (R/R) high-risk (HR) myelodysplastic syndrome (MDS) and R/R acute myeloid leukemia (AML). Based on the results, PureTech’s Founded Entity, Gallop Oncology, has selected a recommended Phase 2 dose (RP2D) and intends to engage with the U.S. Food and Drug Administration (FDA) to discuss the design of a subsequent trial that could potentially support registration of LYT-200 in R/R HR-MDS.

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"The data from the completed Phase 1b trial highlight the potential for LYT-200 to offer a differentiated treatment approach across a range of myeloid hematological malignancies," said Aleksandra Filipovic, M.D., Ph.D., Head of Oncology at PureTech and Chief Medical Officer of Gallop Oncology. "Across patients with R/R HR-MDS and R/R AML, treatment with LYT-200 resulted in deep responses with an exceptionally favorable safety profile. Importantly, the data in R/R HR-MDS were particularly compelling and support prioritizing this indication, especially given the significant unmet need and lack of successful innovation to help these patients. We intend to engage with the FDA to discuss the design of a subsequent trial in R/R HR-MDS, as our goal is to accelerate delivery of this promising first-in-class therapy to patients while also laying the foundation for broader clinical development, including in AML."

The completed Phase 1b trial (NCT05829226), conducted across nine U.S. sites, evaluated LYT-200 both as a monotherapy and in combination regimens in two heavily pretreated patient populations.

The study included dose escalation of monotherapy LYT-200, followed by dose escalation of LYT-200 in combination with a hypomethylating agent (HMA; azacitidine or decitabine) in patients with R/R HR-MDS and with venetoclax (VEN) and an HMA in R/R AML.

"The safety profile, combinatorial potential, and level of clinical activity observed with LYT-200 in this Phase 1b study across both R/R HR-MDS and R/R AML is very encouraging, particularly given the number of prior lines of treatment and the risk profile in the populations studied," said Amir T. Fathi, M.D., Program Director of the Center for Leukemia at the Mass General Brigham Cancer Institute and Professor of Medicine at Harvard Medical School. "In R/R high-risk MDS, where treatment options are extremely limited and outcomes are poor, the findings are particularly notable. In this context, the potential to achieve clinical responses without added toxicity would represent a meaningful advance in the MDS treatment landscape and warrants continued clinical development."

"The results from this Phase 1b trial provide a strong foundation for the next stage of development of LYT-200," said Eric Elenko, Ph.D., President and Co-founder of PureTech and Acting Chief Executive Officer of Gallop Oncology. "Our decision to prioritize relapsed/refractory high-risk MDS reflects a focused and disciplined approach, grounded in both the data generated to date and the potential to address a tremendous patient need. We intend to engage with the FDA to discuss a subsequent trial design with the potential to support registration, while continuing to evaluate the broader potential of LYT-200."

TOPLINE SAFETY DATA

LYT-200 demonstrated a favorable and consistent safety profile across all cohorts and dose levels studied (N=101), with no dose-limiting toxicities, infusion-related reactions, LYT-200 dose reductions, or LYT-200-related serious adverse events (AEs), discontinuations, or deaths. Importantly, no overlapping or additive toxicities were observed when LYT-200 was combined with an HMA or VEN/HMA.

Six patients at one study site reported experiencing hematology/chemistry-related Grade 3 or 4 AEs attributed as possibly related or related to LYT-200 in the combination arm at the RP2D dose. The reported AEs consisted of decreased levels of platelets, white blood cells, and neutrophils that were below the lower limit of normal physiological levels. The blood count deficits for some of the relevant patients were present at baseline prior to the administration of LYT-200 and are common occurrences in patients due to the underlying advanced MDS/AML, as well as in those receiving VEN/HMA treatment. No other sites reported Grade 3 or greater AEs related to LYT-200 treatment.

TOPLINE EFFICACY DATA

Treatment with LYT-200 in combination with an HMA in R/R HR-MDS patients and VEN/HMA in R/R AML patients demonstrated robust antileukemic activity, including complete responses, bridging to transplant, and durable clinical benefit. The data also provided important insights into the contribution of LYT-200 within combination regimens.

R/R HR-MDS

Across all efficacy-evaluable[1] patients (n=11), the recommended Phase 2 dose (LYT-200 12mg/kg in combination with an HMA) demonstrated:

· 27.3% complete response rate

· 9.1% partial response rate

· 9.1% marrow complete response rate

· 45.5% overall response rate

· 18% conversion to transplant rate

Due to the number of patients alive at the time of study completion (>50%), the upper bound of overall survival could not be calculated; therefore, the median overall survival for this cohort of 6.4 months is not considered fully mature.

Efficacy-evaluable patients had a median of 3 prior lines of therapy (range: 1-5), and all (100%) had previously been treated with an HMA. Additionally, all patients had high-risk cytogenetics, which – coupled with prior exposure to treatment – suggests biologically aggressive, treatment-refractory disease with elevated risk of progression and poor clinical outcomes. Taken together, these attributes underscore the potential mutation-agnostic mechanism of LYT-200 and its potential for broad clinical use.

R/R AML

Across all efficacy-evaluable1 patients (n=26), LYT-200 12mg/kg in combination with VEN/HMA demonstrated:

· 30.8% composite complete response rate[2]; responders included patients with mutations associated with VEN resistance

· 7.7% partial response rate

· 42.3% overall response rate

· 19.2% conversion to transplant rate

Due to the number of patients alive at the time of study completion (50%), the upper bound of overall survival could not be calculated; therefore, the median overall survival for this cohort of 8.2 months is not considered fully mature.

Efficacy-evaluable patients had a median of 2 prior lines of therapy (range: 1-9), and 84.6% had previously been treated with VEN/HMA.

INITIAL PHARMACODYNAMIC FINDINGS

The systemic effects of LYT-200 were evaluated through pharmacodynamic analyses of peripheral blood mononuclear cells, a population of immune cells in the bloodstream that provides insight into how a treatment affects both the immune system and leukemic blast cells. These analyses suggest that LYT-200 engages complementary and potentially synergistic pathways directed at cancer cell killing and anti-cancer immune responses when combined with VEN and HMA-based therapy, which may contribute to the clinical activity observed in patients with relapsed/refractory disease following HMA and VEN/HMA treatment, in MDS and AML, respectively.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are a group of serious blood cancers characterized by ineffective blood cell production in the bone marrow, leading to anemia, infections, and bleeding complications. [3], [4] MDS affects approximately 60,000-170,000 people in the United States, with an estimated 30-40% of patients diagnosed with the more aggressive form of the disease known as high-risk (HR) MDS.3, [5] HR-MDS is associated with poor outcomes, with median survival typically less than two years following diagnosis, and approximately 30% of patients progressing to acute myeloid leukemia (AML).

The current standard frontline treatment for HR-MDS are hypomethylating agents (HMAs), such as azacitidine and decitabine; however, most patients do not respond to these therapies or eventually stop benefiting from them.[7] Once the disease becomes relapsed or refractory (R/R), outcomes are especially poor, with survival often limited to only a few months.

Treatment options for patients with R/R HR-MDS remain very limited. Only one therapy has been approved specifically for this setting in the past two decades, and it targets only a small subset of patients (~3-5%) with a specific genetic mutation.7 As a result, there remains a significant need for new treatment approaches for patients with HR-MDS.

About Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is an aggressive blood cancer characterized by the rapid growth of abnormal myeloid blast cells in the bone marrow and blood. It is the most common form of acute leukemia in adults, with a five-year survival rate of less than 30%.[9] Despite available therapies, many patients relapse or fail to respond, and outcomes are especially poor in the relapsed/refractory setting. Around 450,000 people globally are living with AML.9

AML is an area of urgent medical need where new therapies with improved safety, efficacy, and durability or responses are critical. Importantly, the incidence of AML is increasing, and the market is expected to grow to $6 billion annually by 2030,[10] underscoring the scale of the opportunity to bring forward therapies that are not only more effective but also applicable across a broader segment of patients.

About LYT-200

LYT-200 is a fully human IgG4 monoclonal antibody in development for the treatment of hematological malignancies. LYT-200 targets galectin-9, which is an important oncogenic driver and potent immunosuppressor in cancer, positioning it as a novel target for cancer therapy.[11] LYT-200 has been granted Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia.

(Press release, PureTech Health, APR 22, 2026, View Source [SID1234664688])