Vivace Therapeutics Announces Receipt of Orphan Drug Designation for VT3989 for Treatment of Mesothelioma

On July 30, 2025 Vivace Therapeutics, Inc., a small molecule discovery and development company developing first-in-class cancer therapies targeting the Hippo pathway, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to VT3989 for the treatment of mesothelioma in the United States (Press release, Vivace Therapeutics, JUL 30, 2025, View Source [SID1234654656]). VT3989, the company’s first-in-class and best-in-class transcriptional enhanced associate domain (TEAD) autopalmitoylation inhibitor, is a novel investigational small molecule cancer therapeutic that is designed to target the Hippo pathway by inhibiting palmitoylation of members of the TEAD protein family.

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"The granting of Orphan Drug Designation to VT3989 underscores the critical need for new, effective therapies for mesothelioma, an aggressive cancer with limited treatment options. The benefits provided by this important designation will support our continued advancement of VT3989, which has already generated compelling clinical trial data, a first for this promising therapeutic class," said Sofie Qiao, Ph.D., president and chief executive officer of Vivace Therapeutics. "We are committed to continuing clinical development of VT3989 and discussing a move into a registrational Phase 3 study in mesothelioma with FDA by the end of 2025."

VT3989 has been evaluated in more than 200 patients to date in an ongoing, open-label Phase 1 clinical study and, to the company’s knowledge, is the first and only member of the TEAD autopalmitoylation inhibitor class for which compelling clinical efficacy data have been publicly reported. In addition to the promising data to date, VT3989 has demonstrated a positive safety profile in the Phase 1 trial, which supports its best-in-class potential.

Clinical findings for VT3989 have been particularly notable in patients with mesothelioma who have failed chemotherapy and immuno-oncology combination regimens, which represent the only approved therapies in this indication. These results will be presented at a major medical conference in the second half of 2025.

Orphan Drug Designation is granted by FDA and is intended to support the development and evaluation of treatments for rare diseases affecting fewer than 200,000 people in the U.S. The designation provides drug developers with potential benefits including tax credits for qualified clinical trials, exemptions from certain FDA fees for clinical trials, and the potential for seven years of market exclusivity following drug approval.

About Phase 1 study of VT3989
The Phase 1 study of VT3989 (View Source) is a multi-center, open label trial designed to evaluate the safety, tolerability, pharmacokinetics (PK) and biological activity of VT3989 in patients with refractory metastatic solid tumors, including refractory pleural and non-pleural malignant mesothelioma.

Espervita’s EVT0185 Shows Dramatic Tumor Reduction and Immune Activation in Preclinical MASH-Driven Hepatocellular Carcinoma

On July 30, 2025 Espervita Therapeutics, a biotechnology company developing targeted metabolic reprogramming therapies, reported the publication in Nature featuring groundbreaking preclinical data for the treatment of hepatocellular carcinoma (HCC) with its lead drug candidate, EVT0185, a first-in-class liver and kidney targeted inhibitor of acetyl-CoA metabolic enzymes (ACLY, ACSS2, ACC) (Press release, Espervita Therapeutics, JUL 30, 2025, View Source [SID1234654654]). Over 80% of people with advanced HCC do not respond to immunotherapies due to a "cold" immune deficient tumor microenvironment. EVT0185 reverses this effect, making tumors "hot" and harnessing the body’s immune system to attack and kill tumors more effectively than current standards of care.

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Unmet Need

Liver cancer is the third leading cause of cancer death globally. HCC, the most common form of liver cancer, is increasingly caused by the global rise in metabolic dysfunction-associated steatohepatitis (MASH). With current treatment options, five-year survival for advanced HCC is < 5% and the five-year recurrence rate for earlier-stage HCC following surgical resection or ablation is > 75%, with HCC being the only common cancer in the world with no approved adjuvant therapy. HCC is thus one of the highest priority unmet needs in oncology.

Preclinical Efficacy in HCC

Espervita Therapeutics evaluated the therapeutic potential of acetyl-CoA metabolic enzyme inhibition in MASH-HCC using EVT0185 across three preclinical models.

Key findings from the studies:

EVT0185 significantly reduced tumor burden in multiple preclinical models of MASH-HCC prevention and treatment, both as a monotherapy and in enhancing the effects of existing treatments, including tyrosine kinase inhibitors and immunotherapies.

EVT0185 enhanced immune recognition of tumors by reprogramming the tumor microenvironment, increasing CXCL13 expression and B cell infiltration, and triggering strong anti-tumor responses.

Through tissue-selective activation, EVT0185 minimized off-target effects and avoided immune cell suppression.
"These findings illustrate how targeted metabolic reprogramming can counter the immunosuppressive tumor microenvironment and significantly reduce tumor burden in MASH-HCC," said Spencer Heaton M.D., CEO at Espervita Therapeutics. "They also highlight the potential of EVT0185 to redefine how we approach oncology immunotherapies."

Sumitomo Pharma America Announces that the European Medicines Agency Has Granted Orphan Drug Designation to Nuvisertib (TP-3654) for the Treatment of Myelofibrosis

On July 30, 2025 Sumitomo Pharma America, Inc. (SMPA) reported that the European Medicines Agency (EMA) granted Orphan Drug Designation to nuvisertib (TP-3654), an oral investigational highly selective inhibitor of PIM1 kinase, for the treatment of patients with myelofibrosis (MF) (Press release, Sumitomo Pharmaceuticals, JUL 30, 2025, View Source [SID1234654653]).

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Orphan Drug Designation is granted by the EMA to investigational therapies addressing rare diseases or conditions that affect not more than 5 in 10,000 people in the European Union, and for which there is no satisfactory method of diagnosis, prevention or treatment of the condition. This designation comes on the heels of the recent U.S. Food and Drug Administration (FDA) Fast Track Designation granted to nuvisertib, and following the oral presentation of updated preliminary Phase 1/2 data at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress in Milan, Italy.

"Following the recent FDA Fast Track Designation, being granted Orphan Drug Designation from European regulators emphasizes the potential of nuvisertib as a future option for patients living with myelofibrosis," said Tsutomu Nakagawa, Ph.D, President and Chief Executive Officer of SMPA. "As we pursue our mission of addressing unique and unmet patient needs, we look forward to working with the EMA to advance the clinical development of nuvisertib."

MF, a rare type of blood cancer, is characterized by the buildup of fibrous tissues in the bone marrow, which is caused by dysregulation in the Janus-associated kinase (JAK) signaling pathway. The clinical manifestations of MF include an enlarged spleen, debilitating symptoms and reduction in hemoglobin and/or platelets. MF affects 1 in 500,000 people worldwide.1

"Patients living with incurable blood cancers like myelofibrosis continue to face limited treatment options, particularly in the relapsed or refractory setting," said Professor Francesco Passamonti, M.D., Head of Department of Oncology and Hematology-Oncology, University of Milan, Italy. "The orphan drug designation for nuvisertib underscores its potential and the urgent need for new therapeutic approaches to a difficult to treat disease."

About Nuvisertib (TP-3654)
Nuvisertib (TP-3654) is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.2,3 Nuvisertib was observed to inhibit proliferation and increase apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2 V617F mutation.3 Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization, and also reduced spleen size and bone marrow fibrosis in JAK2 V617F and MPLW515L murine models of myelofibrosis.2 The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate and high-risk myelofibrosis (NCT04176198). The FDA granted Orphan Drug Designation to nuvisertib for the indication of myelofibrosis in May 2022. The Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in November 2024. The FDA granted Fast Track Designation to nuvisertib for the indication of myelofibrosis in June 2025.

Pancreatic Cancer Phase 2a Study with Can-Fite’s Namodenoson Achieved Over 50% Enrollment Milestone

On July 30, 2025 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a clinical-stage biotechnology company developing a pipeline of proprietary small molecule drugs for the treatment of cancer and inflammatory diseases, reported that it achieved the over 50% enrollment milestone in its Phase 2a trial of Namodenoson for pancreatic cancer (Press release, Can-Fite BioPharma, JUL 30, 2025, View Source [SID1234654651]).

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The Phase 2a study is a multicenter, open-label trial enrolling patients with advanced pancreatic adenocarcinoma whose disease has progressed following at least one line of prior therapy. The study is evaluating the safety (primary endpoint), clinical activity, and pharmacokinetics (PK) of Namodenoson in this patient population. Participants receive oral Namodenoson at a dose of 25 mg, administered twice daily in continuous 28-day cycles. Patients are regularly monitored for safety, and to date, Namodenoson has demonstrated a favorable safety profile. The study is led by Prof. Salomon Stemmer, a renowned oncologist and key opinion leader at the Davidoff Center, Rabin Medical Center, Israel.

"This milestone reflects the strong interest among both investigators and patients in exploring Namodenoson as a potential treatment for one of the deadliest and most aggressive cancers," stated Pnina Fishman, Ph.D., Chief Scientific Officer of Can-Fite BioPharma. "We are encouraged by the pace of enrollment and remain committed to advancing Namodenoson as a much-needed therapeutic option for patients with pancreatic cancer."

Namodenoson is a highly selective A3 adenosine receptor (A3AR) agonist, which has shown a compelling safety profile and demonstrated anti-tumor activity in preclinical pancreatic cancer models. The drug is also being evaluated in clinical trials for advanced liver cancer.

Namodenoson has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of pancreatic cancer.

Immutep Quarterly Activities Report Q4 FY25

On July 30, 2025 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a clinical-stage biotechnology company developing novel LAG-3 immunotherapies for cancer and autoimmune disease, reported an update on its activities for the quarter ended 30 June 2025 (Q4 FY25) (Press release, Immutep, JUL 30, 2025, View Source [SID1234654646]).

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EFTI DEVELOPMENT PROGRAM IN ONCOLOGY

LUNG CANCER

TACTI-004 (KEYNOTE-F91) – Ongoing Phase III Trial in 1L NSCLC
Immutep’s pivotal TACTI-004 Phase III trial is on track and continues to build momentum and is recruiting patients at a growing number of activated clinical sites and countries, with now 78 sites and 23 countries having received regulatory approval, following the successful dosing of the first patient at Calvary Mater Newcastle Hospital in Australia in March 2025.

The TACTI-004 trial evaluates eftilagimod alfa (efti), a first-in-class MHC Class II agonist, in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA and chemotherapy as first line treatment for patients with advanced or metastatic non-small cell lung cancer (1L NSCLC). The global Phase III trial with efti will randomize approximately 756 patients at more than 150 clinical sites and trial results will inform a potential marketing approval application in non-small cell lung cancer, one of the largest indications in oncology.

In late May, Immutep presented a Trial-in-Progress poster for TACTI-004 at the 2025 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in the United States. We have observed encouraging support from the investigators participating in the study in our meetings to date including those held at ASCO (Free ASCO Whitepaper) 2025, ELCC 2025, and an investigator meeting in Budapest, Hungary. Consistent feedback has been that the efficacy and the safety data collected thus far from the TACTI-002 and INSIGHT-003 trials are impressive and address the unmet medical needs seen by many key opinion leaders.

INSIGHT-003 – Phase I Trial in Non-Squamous 1L NSCLC
In May, Immutep announced a high 60.8% response rate and 90.2% disease control rate (N=51), according to RECIST1.1, had been achieved in the investigator-initiated INSIGHT-003 trial as of the data cut-off date of 6 May 2025. INSIGHT-003 is evaluating efti in combination with the anti-PD-1 therapy, KEYTRUDA and doublet chemotherapy as first-line treatment for patients with advanced or metastatic non-squamous non-small cell lung cancer (1L NSCLC).

In patients with TPS <50% (N=47), who represent a high unmet need and over two-thirds of the 1L NSCLC population, the triple combination with efti achieved a 59.6% response rate as compared to historical control of 40.8% from KEYTRUDA and chemotherapy.3 Safety continues to be favourable with no new safety signals. Data from this trial are expected to be presented at a medical conference later in CY2025.

HEAD AND NECK CANCER

TACTI-003 (KEYNOTE-C34) Cohort B – Phase IIb Trial in 1L HNSCC with CPS <1
In May, Immutep announced an excellent median Overall Survival (OS) of 17.6 months had been achieved in Cohort B of the TACTI-003 (KEYNOTE-C34) Phase IIb trial. This part of the Phase II study evaluates efti in combination with KEYTRUDA as first line therapy in recurrent/metastatic head and neck squamous cell carcinoma (1L HNSCC) patients with PD-L1 expression below one (Combined Positive Score [CPS] <1).

The mature 17.6-months median OS in evaluable patients (N=31) with a data cut-off of 31 March 2025 compares favourably to historical results from the two current standard-of-care approaches in the United States for 1L HNSCC patients with CPS <1 including 10.7-months from cetuximab + chemotherapy and 11.3-months from anti-PD-1 therapy + chemotherapy, as well as 7.9-months from anti-PD-1 monotherapy.1,2

Immutep requested a meeting with the U.S. Food and Drug Administration (FDA) to discuss next steps including potential paths to approval for 1L HNSCC with PD-L1 CPS <1.

SOFT TISSUE SARCOMA

EFTISARC-NEO – Phase II Trial in Soft Tissue Sarcoma
In May, Immutep announced the investigator-initiated EFTISARC-NEO Phase II trial evaluating efti with radiotherapy plus KEYTRUDA in the neoadjuvant setting for resectable soft tissue sarcoma (STS) has met its primary endpoint. The novel combination significantly exceeded the study’s prespecified median of 35% tumour hyalinization/fibrosis versus 15% for historical data from radiotherapy alone in patients with resectable STS.

The EFTISARC-NEO study is primarily funded with a grant from the Polish government awarded by the Polish Medical Research Agency program.

The trial’s investigators at the Maria Skłodowska-Curie National Research Institute of Oncology (MSCNRIO) in Warsaw, the national reference centre for STS in Poland, plan to present results from the study at a medical meeting later in CY2025.

BREAST CANCER

AIPAC-003 – Phase II/III Trial in Metastatic Breast Cancer
Immutep is continuing the AIPAC-003 trial, which enrolled 71 metastatic hormone receptor positive (HR+), HER2-negative/low or triple-negative breast cancer patients who exhausted endocrine therapy including cyclin-dependent kinase 4/6 (CDK4/6) inhibitors.

Immutep completed patient enrolment in the randomised Phase II portion of the AIPAC-003 trial in late 2024. Patients across 22 clinical sites in Europe and the United States have been randomised 1:1 to receive either 30mg or 90mg dosing of efti in combination with paclitaxel to determine the optimal biological dose consistent with the FDA’s Project Optimus initiative and prior regulatory interaction with FDA. Patient follow up, data cleaning and analysis is ongoing and an update is anticipated later in CY2025.

IMP761 DEVELOPMENT PROGRAM FOR AUTOIMMUNE DISEASE

IMP761 – Phase I Trial
Immutep is progressing with the ongoing Phase I trial of its autoimmune candidate IMP761. IMP761 is a first-in-class LAG-3 agonist antibody designed to restore balance to the immune system by enhancing the "brake" function of LAG-3 to silence dysregulated self-antigen-specific memory T cells that cause many autoimmune diseases.

In June, Immutep announced positive initial efficacy data and continued favourable safety data from the first-in-human Phase I study. Through the highest dosing level of IMP761 to date (0.9 mg/kg), there have been no treatment-related adverse events in healthy participants. Additionally, pharmacodynamic data at this dosing level show that the inhibition of T cell infiltration in the skin at day 10 following a neoantigen rechallenge has already reached 80%. The substantial reduction in T cell activity highlights the potential efficacy of IMP761 in treating autoimmune diseases.

Immutep is continuing with single ascending dose levels of 2.5, 7 and 14 mg/kg. Additional data from the Phase I is expected to follow later in CY 2025.

INTELLECTUAL PROPERTY
During the quarter, Immutep was granted four new patents. Immutep was granted two new patents for efti in combination with a PD-1 pathway inhibitor by the New Zealand Intellectual Property Office. In addition, two new patents were granted for IMP761, one by the Intellectual Property Office of the Philippines and the other by the Korean Intellectual Property Office.

CORPORATE & FINANCIAL SUMMARY

Senior Management Changes
Immutep’s Acting Chief Medical Officer, Stephan Winckels M.D, Ph.D., has been appointed to the permanent position of Chief Medical Officer. Stephan has over 15 years of experience in oncology drug development and has been working on efti trials as Medical Monitor or Data Monitoring Committee member for more than nine years.

Cash Flow Summary
During the quarter, Immutep continued to exercise prudent cash management as it advanced its clinical trial programs for efti and for IMP761.

The Company is well funded with a strong cash and cash equivalent, and term deposit balance as at 30 June 2025 of approximately A$129.69 million, which is greater than budgeted as at the beginning of FY2025, while progressing our clinical programs within announced timeframes. The total balance consists of: 1) a cash and cash equivalent balance of A$67.41 million and 2) bank term deposits totalling A$62.28 million, which have been recognised as short-term investments due to having maturities of more than 3 months and less than 12 months.

In Q4 FY25, cash receipts from customers were A$6k. The net cash used in G&A activities in the quarter was A$1.44 million, compared to A$704k in Q3 FY25. Payments to Related Parties comprises Non-Executive Directors’ fees and Executive Directors’ remuneration of A$307k.

The net cash used in R&D activities during the quarter was A$15.66 million, compared to A$13.6 million in Q3 FY25. The increase is in line with increased clinical trial activities.

Payment for staff costs was A$2.5 million in the quarter, the same as for Q3 FY25. Total net cash outflows used in operating activities in the quarter were A$18.92 million compared to A$16.26 million in Q3 FY25.

Total cash outflows used in investing activities for the quarter was A$8.16 million, mainly due to the net increase of short-term investments. The short-term investments are comprised of term deposits with maturities of greater than 3 months and less than 12 months. During the quarter, the company transferred back A$12.92 million from short-term investments that had matured to cash at bank and invested A$21.05 million in short-term investments.

In July, US-based Ridgeback Capital Investments L.P. exercised its last remaining convertible notes and warrants in the Company. The cashless exercise resulted in the issuance of 7,475,208 ordinary shares. The Company is now free of any convertible debt, warrants or options.