On June 30,2026 Vect-Horus, a privately held biotechnology company that designs and develops molecular vectors facilitating the targeted delivery of therapeutic molecules and imaging agents, reported a research evaluation and exclusive license option agreement with Servier, an independent international pharmaceutical group governed by a foundation. This collaboration aims to develop targeted oligonucleotide therapeutics for the treatment of central nervous system (CNS) diseases.

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Under the agreement, Vect-Horus will leverage its proprietary VECTrans technology platform to facilitate the transport of Servier’s oligonucleotide therapeutics across biological barriers to the brain, addressing one of the key challenges in the treatment of neurological disorders.

"We are very pleased to enter into this collaboration with Servier, a global pharmaceutical company with a strong commitment to innovation and patient-focused research," said Alexandre Tokay, co-founder and CEO of Vect-Horus. "This agreement further validates the potential of our VECTrans platform to overcome delivery challenges in the CNS and expand therapeutic opportunities for patients suffering from serious neurological diseases."

Vect-Horus and Servier will conduct the research evaluation activities jointly during an initial option period. Following this evaluation phase, Servier will have the exclusive option to advance selected compounds into clinical development and commercialization.

"Progress in rare neurological disorders depends on bringing together complementary expertise and a shared sense of purpose," said Nitza Thomasson, Global Head of Neurology at Servier. "We are excited to work alongside Vect-Horus to unlock the potential of this technology and help advance transformative oligonucleotide therapies for patients."

Vect-Horus will receive research and exclusivity fees during the option period. Upon exercise of the option, Vect-Horus will be eligible to receive an upfront payment, as well as development, regulatory and commercial milestone payments. In addition, the company will be entitled to single-digit royalties on annual net product sales of any resulting commercialized products.

(Press release, Vect-Horus, JUN 30, 2026, View Source [SID1234669014])

On June 30, 2026 Ratio Therapeutics Inc. (Ratio), a clinical-stage pharmaceutical company employing innovative technologies to develop best-in-class radiopharmaceuticals for cancer treatment, reported that it has entered into a strategic supply agreement with PanTera, an innovative producer scaling the supply of vital medical radionuclides, supporting Ratio’s ongoing efforts to build a robust and scalable supply infrastructure for its radiopharmaceutical development programs.

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The agreement provides Ratio with access to dedicated Ac-225 supply and establishes a framework for future expansion as the company’s development needs evolve. The agreement will support Ratio’s clinical-stage radiotherapeutic programs while further strengthening Ratio’s manufacturing and supply chain foundation required to advance targeted alpha therapies.

"Building a resilient and diversified supply network is an important component of our long-term strategy as we advance our radiopharmaceutical pipeline," said Matthias Friebe, Chief Development Officerof Ratio Therapeutics. "PanTera has emerged as an important contributor to the global Actinium-225 ecosystem, and this agreement reflects our commitment to establishing the infrastructure needed to support ongoing clinical development of our radiotherapies."

"We are honored to partner with Ratio Therapeutics to support the advancement of their innovative radiopharmaceutical pipeline," said Christophe Malice, CBO of PanTera. "Ratio’s targeted approach to treating cancer exemplifies the kind of clinical progress enabled by reliable Ac-225 supply. By providing dependable Ac-225, produced in compliance with cGMP, we are proud to help ensure that programs such as Ratio’s can advance without the supply constraints that have long challenged the field. This collaboration reflects PanTera’s commitment to supporting the pharmaceutical industry as it works to bring next-generation targeted therapeutics to patients in need."

As demand for targeted alpha therapies continues to increase across the industry, reliable access to critical radioisotopes such as Ac-225 has become increasingly important. Through strategic collaborations with leading suppliers and manufacturing partners, Ratio continues to strengthen the supply chain necessary to support the development of its therapeutic candidates.

(Press release, Ratio Therapeutics, JUN 30, 2026, View Source [SID1234669013])

On June 30, 2026 Pasithea Therapeutics Corp. (Nasdaq: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic oral MEK inhibitor, for the long-term treatment of chronic diseases including the neurocutaneous manifestations of neurofibromatosis type 1 (NF1), reported updated interim safety and clinical activity data from its ongoing first-in-human trial evaluating PAS-004 in patients with MAPK pathway-driven advanced solid tumors with a documented RAS, NF1 or RAF mutation, including patients who have failed prior BRAF/MEK inhibitor treatment (NCT06299839). The Company also announced details of its protocol amendment to extend dose escalation and initiate a pilot food effect assessment.

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Dr. Kartik Krishnan, Chief Medical Officer of Pasithea, said, "The literature reports that patients with BRAF mutated cancer who progress on BRAF/MEK combination therapy have a median progression-free survival of approximately 5 months with rechallenge with BRAF/MEK combination therapy. We have observed that a number of these patients who previously progressed on prior BRAF/MEK inhibitor treatment have demonstrated stable disease on PAS-004 for greater than six months, including two patients for over one year. We believe that this demonstrates that PAS-004 is an active agent. Importantly, we continue to be encouraged by the observed adverse event (AE) profile and the ability to maintain dosing in patients over a long period of time without discontinuations. Given that we have not reached the maximum tolerated dose (MTD) and the tolerability of the doses we have evaluated to date, we have decided to continue dose escalation in the study with the introduction of our tablet formulation, while also exploring the effect of food on the PK of PAS-004 to enhance our understanding about how best to maximize the benefit of PAS-004 in long-term chronic dosing."

Interim Phase 1 Results for PAS-004 through May 22, 2026:

34 patients have been enrolled and dosed
Heavily pre-treated population with a median of 3 prior lines of therapy (range 1 – 8)
Difficult to treat diagnoses (e.g., refractory pancreatic cancer and ovarian cancer), including 12 patients with BRAF V600E mutations, 10 of whom previously progressed on BRAFi and/or BRAFi/MEKi combinations

Well-tolerated safety profile with long-term, once daily dosing

No dose-limiting toxicities (DLTs), and no discontinuations due to TRAEs with 10 patients on study for >90 days, of which 6 patients >150 days, 4 patients >300 days, and 2 patients >365 days
All TRAEs were Grade 1 or Grade 2; Low cumulative rates of rash (12%) and diarrhea (6%) across all patients at all doses throughout the study, including no (0%) rash or diarrhea in the 4 patients on study >300 days and 2 patients >365 days
No events of retinal or cardiac toxicity
Disease stabilization in the refractory population (N=12)
Multiple patients demonstrating durable clinical benefit and tumor shrinkage, including several MEK/BRAF-pretreated patients who have remained on study for greater than six months and two patients on study for over one year – exceeding the approximately 5-month median PFS reported in the literature for BRAF/MEK rechallenge in this setting. The final PFS data will not be known until the end of the trial.
Pharmacokinetics support daily dosing and exploration of higher dose levels
Long half-life (approximately 60 hours)
Linear, dose proportional PK
Cmax/Cmin ratio <2, with both Cmax and Cmin above the IC50 (half-maximal inhibitory concentration) from our cellular assay at all dose cohorts
The Company plans to submit and present a more robust data set at a future scientific conference.

(Press release, Pasithea Therapeutics, JUN 30, 2026, View Source [SID1234669012])

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])

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).

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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])