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Orca Bio’s TREGZI™ Receives U.S. FDA Approval as First and Only Precision-Engineered Cell Therapy for Allogeneic Transplant in Adults with Hematological Malignancies

On June 30, 2026 Orca Bio, a commercial-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, reported the U.S. Food and Drug Administration (FDA) has approved TREGZI (allogeneic regulatory T cell immunotherapy with HSPC and T cells-vldq), clinically known as Orca-T, a precision-engineered cell therapy for use in matched-donor hematopoietic stem cell transplantation with myeloablative preparative regimen, for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host disease (GVHD)-free survival (cGFS), in the treatment of adults with hematological malignancies.

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"For transplant physicians, one of our greatest challenges has long been preserving the vital graft-versus-leukemia effect while minimizing the risk of GVHD and infection," said Miguel-Angel Perales, M.D., medical oncologist and chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center. "The FDA approval of TREGZI signals a new era in transplant medicine. This precision-engineered cell therapy is built on the foundational principles established by our early CD34 cell selection work and can now be delivered at scale, equipping providers with a new option to reduce serious toxicities and improve treatment outcomes."

"We founded Orca Bio on the audacious goal to engineer living cells into curative medicines, rooted in the belief that single-cell precision could fundamentally rewrite patient outcomes," said Nate Fernhoff, Ph.D., co-founder and chief executive officer of Orca Bio. "The FDA approval of TREGZI is a significant milestone that stands on the shoulders of decades of pioneering science. As we enter this next chapter, our focus turns to the immense responsibility of delivering TREGZI reliably, precisely and safely to the patients and families counting on us."

TREGZI is a personalized treatment manufactured for each individual patient using living cells from a matched donor. TREGZI uses hematopoietic stem and progenitor cells (HSPCs) to reconstitute the immune system, highly purified regulatory T cells (Tregs) to suppress GVHD and conventional T cells (Tcons) to accelerate immune reconstitution and produce graft-versus-leukemia (GVL) activity.

"Developing this concept from early foundational research in our labs based upon the fundamental biology of regulatory T cells, to it now receiving the first FDA approval for a therapy that utilizes highly purified Tregs, is a defining moment for the transplant community," said Robert Negrin, M.D., professor of medicine, blood and marrow transplantation at Stanford Medicine. "The peer-reviewed findings demonstrated this precision-engineered cell therapy delivered improved GVHD-free survival alongside less toxicity, including fewer serious infections and lower non-relapse mortality."

The FDA approval of TREGZI is based on results from the randomized, multi-center Precision-T Phase 3 study of patients (n=187) with a median age of 43.6 years (range 19-65 years) with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). Results with TREGZI plus single-agent tacrolimus (TAC) compared with a conventional allogeneic hematopoietic stem cell transplant (alloHSCT) plus TAC/methotrexate (TAC/MTX) found the following at 12 months:

The primary endpoint of chronic cGFS was 78% with TREGZI compared to 38% with alloHSCT (HR 0.26; p<0.00001), an improvement driven by a reduction in chronic GVHD and fewer patient deaths.
The rate of chronic GVHD was 13% and 44% with TREGZI and alloHSCT, respectively (HR 0.19; p<0.00002).
Overall survival (OS) was 94% with TREGZI and 83% with alloHSCT.
GVHD-free and relapse-free survival (GRFS) was 63% and 31% with TREGZI and alloHSCT, respectively. 
Non-relapse mortality (NRM) was 3% for TREGZI compared with 13% for alloHSCT. 

Additional safety findings were consistent with previous studies. The cumulative incidence for Grade 3 or 4 acute GVHD at day +180 with TREGZI was 6% versus 10% with alloHSCT (HR 0.37; p=0.044). Grade ³3 infections were less common with TREGZI, with a one year estimated incidence of 44% for TREGZI and 51% for alloHSCT. 

"Historically, surviving a blood cancer has often meant navigating serious, long-term effects that can shape patients’ lives well beyond treatment," said Gwen Nichols, M.D., executive vice president and chief medical officer at Blood Cancer United. "As a researcher, physician and a patient advocate, it’s exciting that patients will have a new option that may change what life after transplant can look like, including the potential to support recovery and quality of life."

Hematological malignancies such as AML, ALL and MDS, commonly referred to as blood cancers, are cancers that can originate in the bone marrow and disrupt normal cell production. Despite therapeutic advances, these diseases, particularly in adult and high-risk populations, remain associated with poor outcomes, high relapse rates and significant treatment-related toxicities.

IMPORTANT SAFETY INFORMATION AND INDICATION

WARNINGS AND PRECAUTIONS

Graft Failure: Graft failure has occurred after TREGZI administration. Screen TREGZI recipients for antidonor antibodies that may prevent engraftment. Monitor patients closely for laboratory evidence of hematopoietic recovery.

Graft-Versus-Host Disease: Acute and chronic Graft-Versus-Host disease (GVHD), including life-threatening and fatal cases, occurred following treatment with TREGZI. Acute GVHD manifests as maculopapular rash, gastrointestinal symptoms, and elevated bilirubin. Chronic GVHD may include skin rash, mouth sores, dry eyes, liver inflammation, and development of scar tissue in the skin and joints and damage to the lungs. Treat patients with a single agent calcineurin inhibitor as prophylaxis to decrease the risk of GVHD. Monitor for signs and symptoms of GVHD, and treat if GVHD develops.

Infusion Reactions: Infusion reactions (IRs) may occur during or following treatment with TREGZI. Serious hypersensitivity reactions including anaphylaxis may occur to DMSO, human serum albumin (HSA), Dextran or murine protein present in TREGZI. IRs may begin within minutes of the start of TREGZI infusion, although symptoms may continue to intensify and not peak for several hours after the completion of the infusion. Monitor patients for signs and symptoms of IRs during and after TREGZI administration. When a reaction occurs, pause the infusion and institute supportive care as needed. Premedicate patients with antipyretics and histamine antagonists prior to infusion to reduce the incidence and intensity of infusion reactions.

Secondary Malignancies and Malignancies of Donor Origin: Secondary malignancies and malignancies of donor origin may occur following treatment with TREGZI. Development of secondary malignancies, including posttransplantation lymphoproliferative disorder (PTLD) may occur many years after transplantation. PTLD manifests as a lymphoma-like disease favoring non-nodal sites. PTLD is usually fatal if not treated. Serial monitoring of blood for EBV DNA may be warranted in patients with persistent cytopenias. No patient treated with TREGZI has developed PTLD. Monitor for malignancies of donor origin and secondary malignancies. Contact Orca Bio at 1-877-411-6722 if any patient is diagnosed with a secondary malignancy or a malignancy of donor origin.

Transmission of Infectious Agents: Transmission of serious infectious or communicable disease or agents may occur with TREGZI treatment as it is derived from human donor blood and manufactured using animal-derived reagents. Risks of transmission of infectious agents may occur despite screening or testing of donors. Risks of transmission of serious infections include, but are not limited to, human immunodeficiency virus, human T cell lymphotropic virus (HTLV)-1 and -2, hepatitis B virus (HBV), hepatitis C virus (HCV), Treponema pallidum, Trypanosoma cruzi, West Nile virus (WNV), cytomegalovirus, transmissible spongiform encephalopathy agents and vaccinia. Monitor patients for signs and symptoms of infections, perform tests for infectious agents and treat as clinically indicated.

ADVERSE REACTIONS

The most common adverse reactions (incidence ≥ 20%) were mucositis, diarrhea, rash, viral infections, infections pathogen unspecified, abdominal pain, vomiting, nausea, bacterial infections, hemorrhage, aGVHD, edema, and fungal infections.

The most common Grade 3-4 laboratory abnormalities (≥ 20%) are lymphocyte count decreased, platelet count decreased, leukocyte count decreased, neutrophil count decreased and hemoglobin decreased.

INDICATIONS AND USAGE

TREGZI is indicated for use in matched donor hematopoietic stem cell transplantation with myeloablative preparative regimen, for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host-free survival, in the treatment of adults with hematological malignancies.

Please see accompanying full Prescribing Information.

About Precision-T
Precision-T (NCT05316701) is a randomized, open-label, multi-center study that evaluated the safety, efficacy and tolerability of TREGZI compared with conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Orca Bio received guidance from the U.S. Food and Drug Administration on the design of Precision-T, which evaluated TREGZI in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). There were 19 leading treatment centers participating in the trial, which enrolled 187 patients across the U.S. Results were published in Blood in December 2025.

(Press release, Orca Bio, JUN 30, 2026, View Source [SID1234669011])

Moleculin Reports Positive Phase 2/3 MIRACLE Interim Results, With Annamycin Complete Remission Rates 3-fold Greater than Control

On June 30, 2026 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported positive preliminary unblinded efficacy results from the first 45 patients enrolled in Part A of the Company’s pivotal Phase 2/3 MIRACLE trial, analyzed on a full intent-to-treat basis with no patient exclusions. Both Annamycin treatment arms demonstrated favorable efficacy trends compared with the control arm in patients with relapsed or refractory acute myeloid leukemia (R/R AML).

The interim analysis demonstrated a clear efficacy advantage for both Annamycin treatment arms, 190 mg/m² plus HiDAC and 230 mg/m² plus HiDAC, over the HiDAC control arm. CR reached 43% and 36% in the respective Annamycin cohorts, compared with 12% for control, while CRc reached 50% and 57%, respectively, versus 29% for the control arm. The n=45 population contained 75.6% over 60 years of age, 55.6% 7+3 and 31.1% venetoclax regimens for first line (1L) therapies.

Importantly, the remission rates for all three arms, including the control arm, reflect outcomes measured after only a single cycle of therapy, as specified by the MIRACLE protocol. The most commonly cited historical benchmarks in this setting, including the MIRROS and CLASSIC I studies, as well as Moleculin’s own MB-106 study, permitted multiple cycles of treatment. The Company therefore expected absolute remission rates for both the control and Annamycin arms in this single-cycle interim analysis to be lower than those reported in such multi-cycle datasets, and believes the most meaningful comparison is the performance of the Annamycin arms relative to the concurrent, randomized control arm evaluated on the same single-cycle basis.

"These interim Phase 2/3 results on such a challenging subject population represent a defining moment for Moleculin and, we believe, the strongest clinical validation of Annamycin we have seen to date," said Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "To see both Annamycin dose arms meaningfully outperform the control arm across both the primary endpoint of complete remission as well as composite complete remission in patients with relapsed or refractory AML is an exceptional outcome. Importantly, these responses were achieved after only a single treatment cycle, providing what we believe is compelling evidence of Annamycin’s anti-leukemic activity. We believe these results further validate Annamycin’s differentiated profile and strengthen our confidence as we advance the MIRACLE trial toward completion."

"From a clinical perspective, these interim results are highly encouraging," Mr. Klemp continued. "Historically, achieving meaningful remission rates in relapsed or refractory AML has been exceptionally difficult, especially with subjects pretreated with venetoclax, which is why seeing both Annamycin treatment arms outperform the control arm across both efficacy endpoints is so noteworthy. The remission rates observed to date are particularly compelling when viewed in the context of outcomes historically reported for currently available therapies in this setting, especially given that patients in MIRACLE were evaluated after only a single treatment cycle. While cross-trial comparisons should be made with caution, these findings suggest the potential for a level of clinical activity that could meaningfully advance the treatment landscape for patients with relapsed or refractory AML. If these results continue to be observed as the trial progresses, Annamycin could represent an important new option for patients facing a disease with substantial unmet medical need."

The interim review was conducted by the trial’s Independent Data Monitoring Committee (iDMC). The committee unanimously concluded that for the primary efficacy endpoint of CR rates, although there was no statistical significance, there was a strong numeric trend suggesting that the experimental treatment arms (L-Annamycin at one of two doses plus high dose cytarabine) were superior to the placebo plus high dose cytarabine control arm. To that end, there was sufficient evidence of efficacy to support continuing the trial. The committee also concluded that the data did not support dropping either of the two experimental treatment arms since the efficacy data were too similar between these two treatment arms. The Company decided to accept the recommendation of the iDMC and continue with the MIRACLE trial as planned.

The absence of formal statistical significance at this first interim analysis reflects the trial’s prespecified statistical design, not the strength of the data. As in most group-sequential studies, MIRACLE distributes its statistical "budget" across three planned analyses using a conservative O’Brien-Fleming spending function, which by design sets a very high bar for significance at an early interim look and reserves essentially the entire budget for the final analysis in the full population of approximately 282 subjects. Statistical significance was therefore neither expected nor required at this stage; the relevant question at an interim look is the direction and strength of the efficacy trend, which the iDMC found clearly favored both Annamycin arms over control. The trial remains fully powered, at 80%, to establish statistical significance at its planned final analysis (calculated to detect 20% vs. 35% CR for control vs. test, respectively).

The MIRACLE trial is a Pivotal Phase 2/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Adaptive Design Study of L-Annamycin for Injection in Combination with Cytarabine Injection Versus Placebo in Combination with Cytarabine Injection as Second Line Therapy for Remission Induction in Adult Subjects with Refractory/Relapsed Acute Myeloid Leukemia. Part A of the study is designed to identify the optimal Annamycin dose before advancing into the pivotal portion of the trial. Moleculin reported that 67 of the targeted 90 patients for Part A have been enrolled, representing approximately 74% of the planned enrollment. Following completion of Part A, the selected dose arm is expected to advance into Part B, where efficacy data from Part A will be carried forward into the pivotal analysis.

Acute myeloid leukemia remains an area of significant unmet medical need, particularly among patients who relapse after frontline therapy. Moleculin believes the preliminary MIRACLE results further support Annamycin’s potential to improve remission outcomes in this patient population and strengthen the rationale for the program’s continued advancement toward registration.

The interim results related to efficacy are final and other data remain subject to Part A final database review and readout.

(Press release, Moleculin, JUN 30, 2026, View Source [SID1234669010])

Aptose Biosciences Announces Completion of Acquisition by Hanmi Pharmaceutical

On June 30, 2026 Aptose Biosciences Inc. ("Aptose" or the "Company") (TSX: APS and OTC: APTOF), a clinical-stage precision oncology company developing a tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed acute myeloid leukemia (AML), reported the closing of the plan of arrangement (announced on November 19, 2025 and February 24, 2026) pursuant to which HS North America Ltd. (the "Purchaser"), a wholly owned subsidiary of Hanmi Pharmaceutical Co. Ltd. ("Hanmi", and together with the Purchaser, the "Hanmi Purchasers") acquired all of the issued and outstanding common shares of Aptose (the "Common Shares") that were not currently owned or controlled by the Hanmi Purchasers or their respective affiliates (the "Arrangement").

Under the terms of the amended and restated arrangement agreement among Aptose and the Hanmi Purchasers dated February 23, 2026, Aptose shareholders, other than the Hanmi Purchasers and their respective affiliates that hold any Common Shares, will receive C$2.41 in cash per Common Share, which represents a premium of 28% over Aptose’s 30-day VWAP of C$1.88 on the Toronto Stock Exchange ("TSX") for the period immediately preceding entering into the Arrangement Agreement.

The Arrangement received approval from the Company’s shareholders at a special meeting held on March 31, 2026, and the Arrangement received final court approval on the same date. The Arrangement has also received the necessary regulatory approvals in Korea.

As a result of the Arrangement, the Common Shares are expected to be delisted from the TSX at the close of trading on or about July 3, 2026. The Company has submitted an application to cease to be a reporting issuer under applicable Canadian securities laws and to otherwise terminate the Company’s public reporting requirements in the United States and Canada.

(Press release, Hanmi, JUN 30, 2026, View Source [SID1234669009])

Genomic Testing Cooperative Highlights Expanded Role of Comprehensive CSF-Based Molecular Profiling Following NCCN Guideline Update for Inoperable High-Grade Gliomas and Glioblastomas

On June 30, 2026 Genomic Testing Cooperative (GTC), a molecular diagnostics company advancing comprehensive DNA and RNA-based cancer testing, reported the growing clinical importance of its Liquid Trace cerebrospinal fluid (CSF)-based molecular profiling following the recent update to the National Comprehensive Cancer Network (NCCN) Guidelines for Central Nervous System Cancers.

"The NCCN update reinforces what many leading clinicians and researchers have recognized: CSF can be an essential source of molecular information for patients with central nervous system tumors," said Maher Albitar, MD, Chief Executive Officer and Chief Medical Officer of Genomic Testing Cooperative. "For these patients, partial information is not enough. A comprehensive CSF-based approach that includes both cell-free DNA and RNA analysis has the potential to provide a more complete view of the tumor and help physicians make more informed care decisions."

GTC’s Liquid Trace CSF testing is designed to address this clinical need with a comprehensive approach that evaluates both cell-free DNA and RNA. By integrating cfDNA and RNA analysis, GTC’s testing may help clinicians identify molecular abnormalities, support diagnosis, evaluate the primary tumor, assess disease biology and better inform treatment planning when tissue is unavailable or insufficient.

The NCCN update expands recommendations for next-generation sequencing to include CSF-based molecular tumor profiling for patients with inoperable high-grade gliomas and glioblastomas when tissue biopsy is not feasible due to tumor location, patient health, surgical risk or patient preference.

For patients with central nervous system cancers, this change represents an important step forward. Tissue biopsy is not always possible, and blood-based testing may not provide adequate molecular information for tumors confined to the brain, spine or cerebrospinal fluid. In those settings, CSF can provide a more informative liquid biopsy sample because it directly bathes the central nervous system and may contain tumor-derived molecular material not readily detected by cytopathology.

GTC’s Liquid Trace CSF testing is optimized for primary and metastatic central nervous system neoplasms and is designed to support diagnosis, genomic profiling, measurable residual disease monitoring, therapy selection and clinical trial matching. The assay is part of GTC’s broader cooperative model, which combines advanced next-generation sequencing, machine learning and collaboration with leading academic and clinical partners to advance precision oncology.

"We look forward to continuing to demonstrate the clinical utility of CSF testing in additional and expanded disease states, especially where CSF may be more informative than tissue or blood," Dr. Albitar said. "This is an important moment for the field because it recognizes that patients with inoperable CNS tumors still need access to high-quality molecular information that can help guide care."

The guideline update comes as GTC continues to work with academic collaborators and technology partners to demonstrate the clinical utility of CSF testing across an expanded range of disease states.

Utilized by top cancer programs and academic medical centers nationwide, GTC’s CSF-based testing stands as the sole commercially available option with Medicare coverage (for DNA analysis).

The NCCN update adds inoperable high-grade gliomas and glioblastomas to a growing list of central nervous system cancers for which CSF-based molecular testing is increasingly recognized as clinically important, including leptomeningeal and parenchymal metastases, primary CNS tumors and CNS lymphomas.

"Cancer is complex, and the central nervous system presents some of the most difficult diagnostic challenges in oncology," Albitar added. "When tissue is not feasible, physicians and patients still deserve access to comprehensive molecular answers. That is exactly the problem GTC is working to solve."

(Press release, Genomic Testing Cooperative, JUN 30, 2026, View Source [SID1234669008])

Imugene Reports First Complete Response in Concurrent BTKi Cohort of azer-cel Phase 1b Trial

On June 30, 2026 Imugene Limited (ASX: IMU), a clinical-stage immuno-oncology company, reported the first patient data from the concurrent BTK inhibitor (BTKi) cohort of its ongoing Phase 1b basket study of azer-cel (azercabtagene zapreleucel).

This Follicular Lymphoma (FL) patient, who had previously failed BTKi therapy, achieved a complete response at the Day 28 assessment.

FL is the most common indolent B-cell lymphoma and, while many patients respond to initial treatment, it remains a high unmet need with standard therapies and most patients will relapse over time.

All patients enrolled in the concurrent BTKi cohort have relapsed on or are refractory to BTKi therapy, a standard treatment across multiple B-cell malignancies. Despite the established efficacy of BTK inhibitors, a significant proportion of patients develop resistance over time and are left with limited remaining options. This cohort evaluates whether concurrent dosing of azer-cel with a BTKi may restore or enhance therapeutic activity in this setting. The global BTKi market reached approximately US$12.0 billion in 2025.

Leslie Chong, Managing Director and CEO of Imugene, said, "This initial data provides further confidence in the potential of azer-cel in patients who have exhausted standard treatment options, including BTK inhibitors. Given how widely BTKi therapies are used, we believe this combination approach could represent a meaningful development pathway for azer-cel."

To date, four patients have been dosed in the concurrent BTKi combination cohort, including the first patient with mantle cell lymphoma. Further updates will be provided as additional data becomes available, and the dataset matures.

Azer-cel is an off-the-shelf, allogeneic CAR T cell therapy which targets CD19 to treat blood cancers. Azer-cel is derived from healthy donor T cells and ready for administration within days, without the three-to-six-week manufacturing lead time required for autologous CAR T products.

About the Phase 1b azer-cel trial

The azer-cel allogeneic CAR T trial is an ongoing, open-label, multi-centre Phase 1b clinical trial in the U.S. and Australia, for CAR T relapsed patients and CAR T naïve patients diagnosed with a broad range of Non-Hodgkins lymphomas including follicular lymphoma (FL), chronic lymphocytic leukemia (CLL)/ small lymphocytic lymphoma (SLL), marginal zone lymphoma (MZL), Waldenstrom macroglobulinemia (WM), and mantle cell lymphoma (MCL). The trial has most recently expanded into a concurrent BTKi cohort, for patients with a range of B-cell malignancies who have previously failed BTKi therapy. Treatment with azer-cel, lymphodepletion and IL-2 has produced meaningful clinical responses across multiple indications, including a complete response in the first evaluable patient from the BTKi combination cohort. Additionally, the safety profile is manageable and generally well tolerated.

(Press release, Imugene, JUN 30, 2026, View Source [SID1234668996])