Medicenna Strengthens U.S. Patent Estate with Newly Issued and Allowed Patents Across its IL-2, IL-4 and IL-13 Superkine Platforms

On June 11, 2026 Medicenna Therapeutics Corp. ("Medicenna" or the "Company") (TSX: MDNA, OTCQX: MDNAF), a clinical-stage immunotherapy company developing Superkines for targeting cancer and autoimmune disease, reported the issuance of two new U.S. patents and the allowance of an additional U.S. patent application covering its proprietary IL-4 and IL-13 Superkine platforms, including bizaxofusp (formerly MDNA55), the Company’s IL-4 Empowered Superkine in clinical development for recurrent glioblastoma (rGBM).

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The newly issued U.S. patents, together with parallel patents recently granted in Australia and Canada within the same patent families, further reinforce Medicenna’s intellectual property position across the cytokine biology underlying its lead clinical programs: bizaxofusp, MDNA11 (IL-2 Superkine) and MDNA113 (anti–PD-1 × IL-2 bifunctional Superkine).

"These newly issued and allowed U.S. patents underscore the depth and breadth of the Superkine science Medicenna has built and the strength of the IP estate now protecting the platforms behind bizaxofusp, MDNA11 and MDNA113," said Dr. Fahar Merchant, President and Chief Executive Officer of Medicenna. "Our cytokine engineering work spans three distinct receptor systems, including IL-2, IL-4 and IL-13 and these grants extend protection into important new applications, including cellular immunotherapy and combination treatment of CNS tumors. With more than 100 active granted patents and applications worldwide, Medicenna has assembled what we believe is one of the most comprehensive patent estates in Superkine-based immunotherapy."

U.S. Patents Recently Issued

U.S. Patent No. 12,503,496: "Interleukin-4 Receptor-Binding Fusion Proteins and Uses Thereof" (Medicenna and the U.S. National Institutes of Health, co-owners). The issued patent covers the application of Medicenna’s proprietary IL-4 Superkine fusion proteins to enhancing cellular immunotherapy. With this grant, the family now includes three issued U.S. patents, and additional granted patents in Europe and India.
U.S. Patent No. 12,590,133: "IL-13/IL-4 Superkines: Immune Cell Targeting Constructs and Methods of Use Thereof" (in-licensed by Medicenna from Stanford University). The issued patent is directed to IL-13 Superkine immune cell targeting constructs, vectors, and engineered cells. The family is now granted in the United States and China, with pending applications in Canada and Europe.
U.S. Patent Recently Allowed

U.S. Patent Application No. 18/248,601: "Combination Therapy of MDNA55 and a Vascular Endothelial Growth Factor a (VEGF-A)" (Medicenna-owned). Once issued, the patent will cover combinatorial treatment of CNS tumors with bizaxofusp, including with VEGF-A-directed agents. The family also has pending applications in Canada, China, Europe, India, Japan and Korea.
Additional Recent Patent Grants Outside the United States

In parallel with these U.S. milestones, Medicenna recently received patent grants in two additional jurisdictions that extend the geographic reach of its core Superkine families:

Australian Patent No. 2018347796, "IL-4 Fusion Formulations for Treatment of Central Nervous System (CNS) Tumors," directed to Medicenna’s proprietary bizaxofusp formulation and its application to the treatment of CNS tumors. This family is now granted in Australia, Europe and the United States, with pending applications in Canada, China and India.
Canadian Patent No. 3,067,909, "Uses and Methods for IL-2 Superagonists, Agonists, and Fusions Thereof," directed to the combination of Medicenna’s IL-2 Super Agonist and checkpoint inhibitors for the treatment of cancer. This family is now granted in Australia, Canada, Japan and the United States, with additional pending applications in China, Europe and India, as well as further applications in Australia, Japan and the United States.
Medicenna continues to develop a robust global patent portfolio across its R&D platforms and clinical programs, with over 100 active granted patents and applications.

(Press release, Medicenna Therapeutics, JUN 11, 2026, View Source [SID1234666599])

FDA grants Priority Review for Roche’s Tecentriq for a certain type of stage III colon cancer

On June 11, 2026 Roche (SIX: RO, ROP; OTCQX: RHHBY) reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) filing for adjuvant Tecentriq (atezolizumab) and Tecentriq Hybreza (atezolizumab and hyaluronidase-tqjs) in combination with chemotherapy in stage III deficient DNA mismatch repair (dMMR) or microsatellite instability-high (MSI-H) colon cancer, a type of tumour characterised by high mutation rates. The FDA has granted Priority Review and is expected to make a decision on the approval by 9 October 2026.

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"This filing acceptance brings us closer to establishing adjuvant Tecentriq plus chemotherapy as a new standard of care for certain types of early colon cancer," said Levi Garraway, MD, PhD, Roche’s Chief Medical Officer and Head of Global Product Development. "The ATOMIC results demonstrate that Tecentriq plus chemotherapy can substantially reduce the risk of disease recurrence or death, helping more patients remain cancer-free following surgery."

"One in three patients with stage III colon cancer will relapse within five years, underscoring the need for new adjuvant treatment options," said Michael Sapienza, CEO, Colorectal Cancer Alliance. "This milestone represents a critical step toward a reality where treatment is tailored to a patient’s specific tumor biology from the very beginning, giving them a better chance of preventing a recurrence."

The application is based on the landmark ATOMIC study, recently published in The New England Journal of Medicine. ATOMIC demonstrated that adding Tecentriq to standard FOLFOX6 chemotherapy reduced the risk of disease recurrence or death by 50%, compared to chemotherapy alone for people with stage III dMMR colon cancer, determined by an immunohistochemistry test, such as the VENTANA MMR RxDx Panel. The 36-month disease-free survival was 86% for Tecentriq combined with FOLFOX6 compared with 76% in the FOLFOX6 alone group. The safety profile was consistent with previous studies of Tecentriq and FOLFOX6.1

Colon cancer remains one of the world’s most common and deadliest tumours.4 Over one million people are diagnosed globally each year, and despite surgery and chemotherapy, approximately 30% of stage III patients relapse within five years.2-4 Approximately 15% of colon cancer patients present with dMMR/MSI-H tumours, which indicate a higher mutation rate and thus have the potential to respond to immunotherapy.5

Roche is pursuing further regulatory filings for Tecentriq, including with the European Medicines Agency, to bring this first immunotherapy-based adjuvant option to patients with dMMR/MSI-H colon cancer worldwide.

The ATOMIC study was sponsored by the National Cancer Institute (NCI) and conducted by the Alliance for Clinical Trials in Oncology in partnership with Roche and the Arbeitsgemeinschaft Internistische Onkologie (AIO) group in Germany. It highlights Roche’s commitment to working alongside leading academic groups to tackle some of the most challenging cancers.

About the ATOMIC study
ATOMIC (A021502, NCT02912559) is a phase III, randomised, open-label, multicentre study investigating the addition of Tecentriq (atezolizumab) to FOLFOX6 chemotherapy (a combination of folinic acid, fluorouracil, and oxaliplatin) in patients with stage III colon cancer who have a deficiency in DNA mismatch repair (dMMR). The trial enrolled 712 patients. Participants were randomised 1:1 to receive either FOLFOX6 plus Tecentriq for 12 cycles (six months) followed by Tecentriq monotherapy for 13 cycles (an additional six months), or FOLFOX6 alone for 12 cycles. The primary endpoint is disease-free survival (DFS).

About Tecentriq (atezolizumab)
Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1, which is expressed on tumour cells and tumour-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Tecentriq has been approved for some of the most aggressive and difficult-to-treat forms of cancer, and is the first PD-(L)1 cancer immunotherapy available in both subcutaneous and intravenous formulations.

(Press release, Hoffmann-La Roche, JUN 11, 2026, View Source [SID1234666598])

Cellectis Presents Final Phase 1 Results of Lasme-cel and Preliminary Results on Eti-cel at EHA 2026 Congress

On June 11, 2026 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, reported final Phase 1 data from the BALLI-01 clinical trial evaluating lasme-cel, a CD22 directed allogeneic CAR-T therapy, in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL), and preliminary data from the NATHALI-01 study evaluating eti-cel, a dual CD20 and CD22 directed CAR-T in relapsed/refractory B-cell non Hodgkin lymphoma (r/r B-NHL), at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Annual Congress.

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BALLI-01 clinical trial evaluating lasme-cel in r/r B-ALL – Oral Presentation

The BALLI-01 final Phase 1 data will be presented as an oral presentation by Nitin Jain, M.D., Professor of Medicine, Department of Leukemia at the University of Texas MD Anderson Cancer Center in Houston, TX.

45 patients in third line and beyond (3L+) were treated in the BALLI-01 study. 15 patients were treated at the recommended Phase 2 dose and 7 in the target Phase 2 population. Patients were heavily pretreated with those in the target Phase 2 population receiving a median of 5 prior lines of therapy (Range 2-11). Almost all patients were previously treated with blinatumumab (82%) and were also heavily exposed to CD19 CAR-T (53%), CD22-directed antibody-drug conjugate (ADC) (56%) and many had a prior hematopoietic stem cell transplantation (HSCT) (47%).

Final Phase 1 data

In the target Phase 2 population

An overall response rate (ORR) of 100% (7/7) was achieved with a complete remission/complete remission with incomplete count recovery (CR/CRi) rate of 57% (4/7). Of these, 75% achieved minimal residual disease negative (MRD-ve) status.

All patients subsequently proceeded to HSCT.

Lasme-cel demonstrated a manageable safety profile

Cytokine release syndrome (CRS) ≥ grade 3 occurred in 4% of patients.
Immune effector cell-associated neurotoxicity syndrome (ICANS) ≥ grade 3 occurred in 4% of patients.
Immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) ≥ grade 3 occurred in 2% of patients.
All CRS, ICANS, and IEC-HS resolved.

"These final Phase 1 results are particularly meaningful for a patient population that has very limited treatment options" said Nitin Jain, M.D., Professor of Medicine, Department of Leukemia at UT MD Anderson. "Being able to achieve deep remissions in these patients and allowing them to subsequently receive an HSCT is promising. We look forward to accelerating accrual into the ongoing Pivotal Phase 2 study and bringing this treatment to patients."

The Pivotal Phase 2 BALLI-01 trial is open for recruitment. Eligible patients and treating physicians are encouraged to visit BALLI-01 (NCT04150497) or contact Cellectis at [email protected] for information and participating sites. The first interim analysis is expected in Q4 2026.

Oral Presentation: Safety and efficacy of UCART22 in heavily pretreated patients with relapsed or refractory CD22+ B-cell acute lymphoblastic leukemia (B-ALL): results of the Phase 1 BALLI-01 trial

Date/Time: Saturday, June 13 at 5:15 – 6:30pm, local time
Session Title: Advances in the treatment of lymphoblastic leukemia
Session Room: K1
Abstract Number: 4689

Note: presentation slides will be uploaded to Cellectis’ website concurrently with the live presentation.

NATHALI-01 clinical trial evaluating eti-cel in r/r B-NHL – Poster Presentation

The NATHALI-01 preliminary data on the role of alemtuzumab in optimizing responses will be presented as a poster by Professor Emmanuel Bachy, M.D., Ph.D., Department of Hematology, Hospices Civils de Lyon, France.

Eti-cel is a highly differentiated product being the first allogeneic dual CAR-T targeting both CD20 and CD22, for patients with r/r B-NHL.

As of the February 2026 data cutoff, 14 patients with r/r B-NHL had been treated across three dose levels, in a heavily pre-treated population with a median of 3 prior lines of therapy, 93% of whom had received prior CD19-directed CAR-T therapy, and all of whom presented with stage IV disease at baseline.

In the optimal dose cohort, ORR and complete response (CR) were 88% and 63%, respectively. The analysis identified a positive correlation between alemtuzumab exposure and clinical outcomes: higher alemtuzumab exposure created a favorable lower inflammatory homeostatic milieu prior to eti-cel infusion and was associated with enhanced eti-cel expansion and higher response rates. Additionally, responders maintained sustained low-level interleukin 2 (IL-2) secretion when compared to non-responders.

These findings provide a scientific rationale for the implementation of a weight-based alemtuzumab dosing regimen, currently under investigation to optimize lymphodepletion. Additionally, subcutaneous low-dose IL-2 is being investigated to further enhance eti-cel expansion and treatment response.

"These encouraging data demonstrate that not only can eti-cel drive responses in a very difficult-to-treat population, but that by optimizing exposure to alemtuzumab we may be able to create a favorable environment for CAR-T expansion and persistence." said Professor Emmanuel Bachy, M.D., Ph.D., Department of Hematology, Hospices Civils de Lyon, France.

The NATHALI-01 study is open for recruitment with the full Phase 1 clinical data expected in Q4 2026.

Poster Presentation: Alemtuzumab exposure and sustained IL-2 drive UCART20x22 expansion and clinical response in adults with relapsed or refractory B-cell non-Hodgkin lymphoma: NATHALI-01 study

Date/Time: Saturday, June 13 at 6:45 – 7:45pm, local time
Session: Poster Session 2
Poster Number: 4758

(Press release, Cellectis, JUN 11, 2026, View Source [SID1234666597])

C4 Therapeutics Presents Phase 1 Data at European Hematology Association (EHA) 2026 Congress Highlighting Cemsidomide as a Potential Best-in-Class IKZF1/3 Degrader for Multiple Myeloma in Heavily Pretreated Relapsed/Refractory Population

On June 11, 2026 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation (TPD) science, reported it will present further analysis from its fully enrolled Phase 1 trial of cemsidomide, a next-generation oral IKZF1/3 degrader, in combination with dexamethasone for the treatment of relapsed/refractory multiple myeloma (RRMM) in a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress on Friday, June 12, 2026 at 6:45 pm CEST / 12:45 pm ET.

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The analysis is consistent with previous data disclosed from the Phase 1 clinical trial and highlights cemsidomide’s anti-myeloma activity and differentiated safety profile, further supporting its development as a potential best-in-class IKF1/3 degrader. The poster will be presented by Sagar Lonial, M.D., FACP, FASCO, chief medical officer at the Winship Cancer Institute at Emory University, and an investigator in the cemsidomide clinical trials.

"Despite advances in multiple myeloma therapies, IKZF1/3 degradation remains a foundational treatment strategy across lines of therapy because it is the only approach that addresses the underlying biology of the disease and has the built-in ability to stimulate the immune function, becoming a natural partner for immune therapies. Next-generation IKZF1/3 degraders are expected to help advance treatment regimens for this persistent disease, given data demonstrating their efficacy and tolerability," said Dr. Lonial. "The clinical activity and safety profile of cemsidomide are highly encouraging for patients with relapsed/refractory multiple myeloma as they continue to seek disease-altering treatment options. The data from the ongoing Phase 1 study support the continued development of cemsidomide for patients with relapsed/refractory multiple myeloma who may benefit from IKZF1/3 degradation."

"The totality of cemsidomide data, particularly data showing that patients have experienced a deepening response over time, continue to demonstrate its potential to deliver a tolerable therapy that can provide sustained benefit for patients who have progressed through other treatment options," said Len Reyno, M.D., chief medical officer of C4 Therapeutics. "We remain focused on advancing our clinical development strategy to capitalize upon cemsidomide’s differentiated profile in hopes patients at various stages of their treatment journey may be able to benefit from this important investigational therapeutic regimen."

The poster presentation includes data on 73 patients with a data cutoff of February 27, 2026. Patients were heavily pretreated, receiving a median of seven prior lines of therapy. Fifty-five patients (75%) received prior BCMA therapy, and 55 patients (75%) received prior CAR-T or T-cell engager therapy (TCE).

At the RP2D and maximum tolerated dose (100 µg,) cemsidomide achieved a 53% overall response rate (ORR). At the 75 µg dose level, cemsidomide achieved a 40% ORR. Across all doses evaluated, cemsidomide achieved a 36% ORR.

Key new data include:

Responses deepened over time across the cemsidomide 75 µg and 100 µg dose levels:
At 75 µg, one patient whose best response was previously a partial response (PR) deepened to a very good partial response (VGPR).
At 100 µg, several patients achieved a deeper response:
One patient whose best response was previously a PR deepened to a stringent complete response (sCR)
One patient whose best response was previously a PR deepened to a VGPR
Minimal residual disease (MRD) negativity was achieved in two patients who achieved a sCR and complete response (CR) at 100 µg.
ORR was consistent across key subgroups:

ORR % (95% confidence interval (CI))
All Doses
Prior CAR-T or TCE 37% (24, 51)
Prior BCMA 33% (21, 48)
Prior Lines of Therapy > 5 Lines 33% (20, 48)
100 µg (RP2D)
Prior CAR-T or TCE 53% (28, 77)
Prior BCMA 47% (21, 73)
Prior Lines of Therapy > 5 Lines 47% (21, 73)

Durable responses were observed across all dose levels:
Patients experienced a median duration of response of 7.9 months (95% CI, 3.0 – non-evaluable).
Seven patients remain on treatment currently.

Cemsidomide in combination with dexamethasone was generally well tolerated. Incidences of on-target neutropenia remained manageable; 42 patients (58%) experienced Grade 3/4 neutropenia. All treatment emergent adverse events were manageable with no discontinuations deemed related to cemsidomide and minimal dose reductions (five patients; 7%).

UPCOMING INVESTOR EVENTS

June 18, 2026 at 9 am ET: C4T will host an educational webinar with Nisha Joseph, M.D., associate professor at the Winship Cancer Institute at Emory University and investigator in the cemsidomide clinical trials to discuss the evolving multiple myeloma landscape, the role of IKZF1/3 degradation in treating multiple myeloma, and cemsidomide’s profile.

About Cemsidomide
Cemsidomide is an investigational, next-generation orally bioavailable MonoDAC degrader (molecular glue) of IKZF1/3, transcription factors foundational to multiple myeloma biology. Data from the fully enrolled Phase 1 trial show cemsidomide’s differentiated safety and tolerability profile and potentially class-leading anti-myeloma activity that support the potential for durable outcomes.

About Multiple Myeloma
Multiple myeloma is a blood cancer that affects plasma cells in the bone marrow. It is the second most common blood cancer, with approximately 36,000 people in the United States diagnosed each year. Multiple myeloma is characterized by cycles of remission and relapse, which leads to patients needing multiple lines of therapy to manage this persistent disease. More than 175,000 patients in the United States are estimated to be living with or in remission from myeloma. However, despite treatment advances, approximately 40% of patients do not survive beyond five years.

About IKZF1/3 Degradation
Targeted degradation of IKZF1 (Ikaros) and IKF3 (Aiolos) is a foundational therapeutic strategy to treat multiple myeloma, a blood cancer affecting plasma cells. IKZF1/3 degradation leads to downregulation of IRF4, which promotes myeloma cell death. IKZF1/3 degradation also activates T-cells, which contributes to broader anti-myeloma response. For decades, IKZF1/3 degradation has been used in approved therapies for multiple myeloma. Next-generation IKZF1/3 degraders are being developed to leverage advances in targeted protein degradation research while continuing to address the biology foundational to multiple myeloma.

About the MOMENTUM Trial
MOMENTUM (Multi-center trial Of cemsidoMidE iN relapsed/refracTory mUltiple Myeloma) is a Phase 2, open-label, single-arm study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of cemsidomide in combination with dexamethasone in patients with relapsed/refractory multiple myeloma. Data from the Phase 1 trial identified 100 µg as the recommended Phase 2 dose. The primary endpoint is overall response rate per International Myeloma Working Group response criteria, as assessed by an independent review committee. Approximately 100 patients who have received at least three prior anti-myeloma regimens that must have included an IKZF1/3 degrader, a proteasome inhibitor, an anti-CD38 antibody, and a T-cell engager or CAR-T therapy will be enrolled in the trial. More information is available at clinicaltrials.gov (NCT07284758).

About Cemsidomide in Combination With Elranatamab (ELREXFIO)
The Phase 1b trial is designed to evaluate the safety, tolerability and preliminary efficacy of cemsidomide and dexamethasone in combination with elranatamab, an FDA-approved B-cell maturation antigen CD3 targeted bispecific antibody. Data generated from the cemsidomide Phase 1 trial in relapsed/refractory multiple myeloma demonstrate robust T-cell activation and cytokine expression across multiple doses. By activating immune T-cells, cemsidomide, when combined with a BCMAxCD3 bispecific such as elranatamab, may amplify the anti-myeloma immune response and lead to deeper and more durable responses. The study will evaluate different cemsidomide dose levels (beginning with 75 µg, with the opportunity to simultaneously explore 50 µg and 100 µg) in patients who have received one to four prior lines of therapy, which must have consisted of at least one IKZF1/3 degrader. Exclusion criteria for patients include those who have received prior treatment with a BCMA-directed T-cell engager or BCMA-directed CAR-T therapy. More information is available at clinicaltrials.gov (NCT07280013).

(Press release, C4 Therapeutics, JUN 11, 2026, View Source [SID1234666596])

BlossomHill Therapeutics Presents Initial Clinical Data from Dose Escalation in Ongoing Phase 1/1b Trial of Novel Macrocyclic CLK Inhibitor, BH-30236, in Patients with R/R AML or HR-MDS at EHA 2026

On June 11, 2026 BlossomHill Therapeutics, Inc., a privately-held, clinical-stage biopharmaceutical company applying an intentional, chemistry-based approach to design and develop innovative small molecule medicines for the treatment of cancer, reported initial clinical data from its ongoing first-in-human Phase 1/1b trial of BH-30236 in relapsed or refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndrome (HR-MDS) at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress in Stockholm, Sweden. BH-30236 is an intentionally designed novel, orally bioavailable, macrocyclic CDC-like kinase (CLK) inhibitor that modulates aberrant alternative mRNA splicing, a defining feature implicated in cancer progression and therapeutic resistance across both hematologic malignancies and solid tumors.

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"The preliminary anti-leukemic activity observed with BH-30236 in R/R AML and HR-MDS, both as a monotherapy and in combination with venetoclax, is very encouraging," said Geoff Oxnard, M.D., Chief Medical Officer of BlossomHill Therapeutics. "In addition to the reassuring tolerability profile, we have seen provocative responses in patients after progression on prior venetoclax-based therapy, a population with few treatment options. In the ongoing study of BH-30236, we will further characterize the effect of this novel mechanism in myeloid malignancies, both as a monotherapy and in synergy with venetoclax to maximize the potential of this promising program."

"We intentionally designed BH-30236 to target CLK, which is upstream of multiple resistance pathways, not by addressing one resistance mechanism after it develops, but by suppressing the splicing machinery that enables resistance to begin," said Jean Cui, Ph.D., Founder and Chief Executive Officer of BlossomHill Therapeutics. "We believe these data further validate our approach of leveraging a deep understanding of disease and protein dynamics and applying our structure-based rational drug design expertise to identify specific structural liabilities that limit existing therapies or approaches and design novel chemical scaffolds to directly address these limitations."

Presentation Highlights:

As of the data cutoff date of April 10, 2026, 30 patients received BH-30236 monotherapy at doses ranging from 5 to 120 mg on a continuous daily administration schedule (QD), and 18 patients received BH-30236 at doses ranging from 20 to 90 mg QD in combination with venetoclax
BH-30236 was generally well tolerated as both a monotherapy and in combination with venetoclax, with most treatment-related adverse events being low-grade and manageable, with one dose limiting toxicity of Grade 3 diarrhea at the 120 mg QD monotherapy dose level
Dose escalation showed predictable pharmacokinetics without drug accumulation, and no significant drug-drug interactions were observed with venetoclax
With efficacy follow-up through May 20, 2026, preliminary anti-leukemic activity was observed in patients with R/R AML and HR-MDS when treated with BH-30236 as a monotherapy or in combination with venetoclax, including in patients previously relapsed or refractory to venetoclax-based therapy:
In the monotherapy cohort, 28.6% (n=6) of evaluable patients achieved at least a 50% reduction in bone marrow blast counts, including one HR-MDS patient treated at 60 mg with ongoing blast count reduction with treatment ongoing for more than a year
In the combination cohort, 87% (n=13) of patients had prior venetoclax exposure and 53% (n=8) of evaluable patients experienced at least a 50% blast reduction, including one patient refractory to all prior therapy including venetoclax, who achieved a minimal residual disease (MRD)-negative complete remission

About R/R AML and HR-MDS
AML is the most common acute leukemia in adults, with approximately 21,000 new diagnoses annually in the United States, with as many as 75% of those being patients resistant to or relapsing on first-line venetoclax therapy. Approximately 14,000 patients are diagnosed with HR-MDS annually in the United States. There is no currently approved targeted therapy for the venetoclax-resistant and refractory segment of AML patients without targetable genetic alterations. Patients with HR-MDS closely mirror AML in clinical course, frontline treatment, and unmet need in the relapsed and refractory setting.

About BH-30236
BH-30236 is an investigational orally bioavailable, macrocyclic inhibitor of the CDC-like kinase (CLK) family. BH-30236 is was intentionally designed to potently inhibit CLK, leading to modulation of aberrant alternative splicing in cancerous tissue, targeting the same aberrant splicing machinery that drives relapsed or refractory (R/R) acute myeloid leukemia (AML) and higher-risk myelodysplastic syndrome (HR-MDS) disease biology and that cancer cells exploit to develop resistance to venetoclax, FLT3 inhibitors and cytarabine.

BH-30236 is being evaluated in a Phase 1/1b multicenter, open-label, first-in-human dose escalation and expansion trial in adults with R/R AML and HR-MDS. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BH-30236 for the treatment of AML.

(Press release, BlossomHill Therapeutics, JUN 11, 2026, View Source [SID1234666595])