Theolytics presents data on THEO-260 and THEO-310 at ASCO and EHA

On June 9, 2023 Theolytics, a biotechnology company harnessing viruses to combat disease, reported data presentations at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, which was held 2 to 6 June, 2023 in Chicago and at the European Hematology Association (EHA) (Free EHA Whitepaper) 2023 Congress, which is being held 8 to 11 June in Frankfurt (Press release, Theolytics, JUN 9, 2023, View Source [SID1234635716]).

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ASCO Abstract

Title: Evaluation of THEO-260 as a novel oncolytic virus therapy for treating stromal rich tumours

LINK to abstract

Stromal rich cancers pose a challenge to many cancer therapies due to the abundance of cancer associated fibroblasts (CAFs). This compartment of the tumour microenvironment physically blocks the penetration of drugs, immune cells and promotes resistance to many therapies. THEO-260 is a new oncolytic virus in development that is intrinsically capable of targeted killing of both cancer cells and CAFs.

The efficacy and safety of THEO-260 was assessed in multicellular spheroids, in patient-derived ex vivo fresh ovarian samples and in mouse models of human stromal tumours. Unpassaged ascites and tumour samples collected from cytoreductive surgeries for advanced ovarian cancer were treated with a clinically-relevant dose of THEO-260. THEO-260 effectively killed the cancer cell and CAF populations, being efficacious in treatment-naive and platinum-resistant patient samples. Importantly, the T cell population in these patients, in addition to primary normal fibroblasts from healthy donors, were not damaged. Ex vivo studies show THEO-260 stimulates effector T cell responses upon tumour lysis and demonstrates suitability as an IV

delivered agent. THEO-260 excels in low nutrient, low proliferative environments, typical of human solid tumours and has been developed with a view to persist in the tumour microenvironment without the need for repeated cycles of delivery. In ovarian cancer murine models containing human CAFs and cancer cells, THEO-260 shows antitumor efficacy, with a complete reduction in tumour volume in subcutaneous and intraperitoneal systems, and superiority to standard of care. Virus activity is not restricted to ovarian cancer, with cell line data demonstrating killing in additional solid tumour types, including pancreatic, colorectal, lung, and breast cancer. THEO-260 exhibits genetic and temperature based stability, and GMP manufacturing to high yields is underway.

"The positive THEO-260 data presented at ASCO (Free ASCO Whitepaper) come at an exciting time for Theolytics as we progress this lead product towards the clinic in a first-in-human study in ovarian cancer," said Charlotte Casebourne Stock, Chief Executive Officer and Co-Founder of Theolytics. "THEO-260 is a virotherapy that potentially could demonstrate something that neither chemotherapy, nor immunotherapeutic approaches have been capable of to date and has the potential to deliver a step-change for the oncolytic virus field."

EHA Poster Presentation

Title: Development of a Novel Oncolytic Virus – THEO-310 – Specialised for Targeting the Bone Marrow Tumour Microenvironment of Multiple Myeloma Patients

LINK to abstract

Session Title: 24. Gene therapy, cellular immunotherapy and vaccination – Biology & Translational Research

Poster ID: P1362

Session date and time: Friday, 9 June, 18:00 – 19:00 CEST

Session room: Poster area

Presenting Author: Dr Margaret Duffy

THEO-310 is an oncolytic virus developed for tackling the bone marrow tumour microenvironment (TME) of advanced late-stage myeloma patients. THEO-310 demonstrates efficacy in >80% of ex vivo bone marrow aspirates, across newly diagnosed, relapsed and refractory myeloma patients. In these experiments, TME immune modulation and superiority to ‘best-in-class’ oncolytic virus strains for myeloma, including the measles virus, was observed.

"These exciting set of results differentiates Theolytics’ approach to other oncolytic virus candidates. In addition to showing increased efficacy in patients ex vivo, the candidate demonstrates systemic safety as shown by cytokines levels post IV injection in vivo, whilst exhibiting >40-fold better circulation compared to a clinically validated oncolytic virus strain, thus supporting effective intravenous delivery," said Margaret Duffy, Chief Scientific Officer and Co-Founder of Theolytics. "Taken together, THEO-310 represents a new therapeutic option, with a highly differentiated mechanism independent of BCMA, for myeloma patients with relapsed refractory disease.

NovalGen announces two oral presentations at upcoming conferences

On June 9, 2023 NovalGen Ltd ("NovalGen"), a biopharmaceutical company developing disruptive therapies, based on a proprietary autoregulation (AR) platform, reported the presentation of preclinical data on this novel technology in two separate indications at the International Society on Thrombosis and Haemostasis Congress (ISTH), 24-28 June, 2023 in Toronto and the Immuno-Oncology Summit Boston 2023, 7-9 August, 2023 in Boston (Press release, NovalGen, JUN 9, 2023, View Source [SID1234633763]).

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"We are delighted to be showcasing our proprietary platform, which has the potential to improve the safety profile and therapeutic index of a range of immunotherapies. Preclinical data with NVG-444 shows a reduction in thrombotic complications with a bispecific Factor VIII mimetic. These data will be presented in an oral presentation at ISTH. The AR platform is target agnostic as illustrated by its application to an extended half-life T cell engager (TCE) targeting ROR1, resulting in a dramatic reduction in toxicity, thus addressing the "Achilles heel" of TCEs allowing more patients to benefit from this off the shelf, T cell activating technology. The AR technology can be applied to a wide range of immunotherapies and heralds a new and exciting class of self-regulating drugs that respond to biological cues to avoid life-threatening toxicities," said Professor Amit Nathwani, Founder and CEO of NovalGen.

Both abstracts will be delivered as oral presentations at the conferences.

Abstracts to be presented:

Conference: ISTH 2023 Congress
Abstract Title: Thrombin-mediated autoregulation restores sensitivity to the coagulation feedback loop and reduces the risk of prothrombotic events associated with FVIII mimetic antibodies.

Abstract Presentation Number: OC 69.1

Session Title: Hemophilia – Novel therapies II

Session Date: Wednesday, 28 June, 2023
Presentation Time: 10:15 am – 10:30 am

Presenting Author: Vincent Muczynski

Conference: Immuno-Oncology Summit Boston 2023
Abstract Title: NVG-222: A ROR1-Targeting T Cell Engager with Integral Autoregulating Capability Designed to Reduce the Risk of Serious Adverse Events Related to T Cell Activation

Session Title: Bispecific Antibodies for Cancer Immunotherapy: Overcoming Obstacles to Efficacy

Session Date: Tuesday, 8 August, 2023

Presentation Time: 8:05am – 8:35am

Presenting Author: David Granger

AbbVie’s VENCLYXTO®/VENCLEXTA® (venetoclax) Continues to Show Sustained Progression-Free Survival (PFS) in Chronic Lymphocytic Leukemia (CLL) Patients

On June 9, 2023 AbbVie (NYSE: ABBV) reported new findings demonstrating sustained long-term safety and efficacy of VENCLYXTO/ VENCLEXTA (venetoclax)-based combination therapies in patients with previously untreated CLL with co-existing conditions, as well as R/R CLL (Press release, AbbVie, JUN 9, 2023, View Source [SID1234632624]). The results are being presented during oral sessions at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in Frankfurt, Germany.

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"Results from the CLL14 and MURANO studies demonstrate the long-term benefits of fixed-duration venetoclax combinations for patients living with CLL," said Mariana Cota Stirner, M.D., Ph.D., vice president, hematology, AbbVie. "These results underscore our commitment to transform how blood cancers are treated today and show that venetoclax can give patients lasting results with time off treatment."

CLL14 Long-Term Analysis

New six-year follow-up results from the Phase 3 CLL14 study showcase updated outcomes in previously untreated patients with CLL and co-existing conditions. Patients treated with fixed-duration venetoclax plus obinutuzumab continued to experience improved PFS (95% Confidence Interval (CI) 0.31-0.52; Hazard Ratio (HR) 0.40) and higher rates of undetectable minimal residual disease (uMRD) when treated with fixed-duration venetoclax plus obinutuzumab compared to those who received chlorambucil plus obinutuzumab (53.1% vs 21.7%, respectively).

The data also showed significantly improved rates of time to next treatment (TTNT) with venetoclax plus obinutuzumab at 65.2 percent (95% CI 0.33-0.58; HR 0.44) compared to chlorambucil plus obinutuzumab at 37.1 percent.1 The observed differences in PFS and TTNT benefits between venetoclax-based treatment and chemoimmunotherapy were maintained across all risk groups, including patients with high-risk molecular features of CLL.

No new safety signals were observed in this six-year analysis. The most frequently occurring Grade 3 (≥2%) adverse events (AEs) in patients receiving the venetoclax-based combination were neutropenia, thrombocytopenia, infusion-related reaction (during treatment), anemia, febrile neutropenia, pneumonia and leukopenia.1

"The latest findings show that patients can experience long-term disease control, five years after stopping treatment," said Othman Al-Sawaf, M.D., investigator in the CLL14 study, hematologist-oncologist at the University Hospital Cologne in Germany, and study physician at the German CLL Study Group. "These results confirm the treatment benefits of fixed-duration venetoclax and obinutuzumab for previously untreated CLL patients with co-existing conditions."

Results will also be featured at EHA (Free EHA Whitepaper)’s Press Briefing.

MURANO Long-Term Analysis

Final data from the Phase 3 MURANO trial showcase that R/R CLL patients treated with two-year fixed-duration venetoclax plus rituximab sustained significantly longer median PFS at 54.7 months (95% CI 52.3, 59.9), the study’s primary endpoint, compared to 17.0 months (95% CI 15.5, 21.7; HR 0.23) with bendamustine plus rituximab after 7 years of median follow-up.2

Seven-year OS rates were 69.6 percent (95% CI 62.8, 76.5) for patients treated with the venetoclax-based combination versus 51 percent (95% CI 43.3, 58.7) for study participants who received bendamustine-based combination (HR 0.53).2 Furthermore, most of the patients treated with the full two-year venetoclax-based combination achieved uMRD (70.3%) at the end of their treatment course, and those patients were shown to have improved PFS and OS compared to patients with detectable MRD (29.7%).

The safety profile of the venetoclax-rituximab combination is consistent with the known safety profile of each individual therapy alone. No new serious safety issues were observed in the MURANO updated analysis. The most common adverse reactions (ARs) (≥20%) of any grade were neutropenia, diarrhea, upper respiratory tract infection, fatigue, and nausea.

"We are pleased to find that uMRD was associated with prolonged PFS in R/R CLL patients after seven years," said study investigator Prof. John Seymour, Director of the Integrated Haematology Department of the Peter MacCallum Cancer Center and the Royal Melbourne Hospital in Melbourne. "Overall, these findings continue to support the use of treatment with venetoclax plus rituximab in this patient population."

VENCLYXTO/VENCLEXTA is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.

About the CLL14 Phase 3 Trial1,3
The prospective, multicenter, open-label, randomized Phase 3 CLL14 trial, which was conducted in close collaboration with the German CLL Study Group (GCLLSG), evaluated the efficacy and safety of a combined regimen of VENCLYXTO/VENCLEXTA and obinutuzumab (n=216) versus obinutuzumab and chlorambucil (n=216) in previously untreated patients with CLL and co-existing medical conditions (total Cumulative Illness Rating Scale [CIRS] score >6 or creatinine clearance <70 mL/min). The therapies were administered for a fixed-duration of 12 months for VENCLYXTO/VENCLEXTA in combination with six cycles of obinutuzumab. The trial enrolled 432 patients, all of whom were previously untreated, according to the International Workshop on Chronic Lymphocytic Leukemia (iwCLL) criteria. Efficacy was based on PFS, as assessed by an independent review committee.

Key secondary endpoints were rates of MRD in peripheral blood and bone marrow, overall and complete response rates, MRD in complete response in peripheral blood and bone marrow, and OS.

In patients with CLL receiving venetoclax combination therapy with obinutuzumab, the most frequently occurring ≥ Grade 3 AEs (≥2%) were neutropenia (51.9%), thrombocytopenia (14.2%), infusion-related reaction (9.0%), anemia (7.5%), febrile neutropenia (4.2%), pneumonia (3.8%) and leukopenia (2.4%). Serious ARs were most often due to febrile neutropenia and pneumonia (5% each). The most common ARs (≥20%) of any grade were neutropenia (60%), diarrhea (28%), and fatigue (21%). Fatal ARs that occurred in the absence of disease progression and with onset within 28 days of the last study treatment were reported in 2 percent (4/212) of patients, most often from infection.

About the MURANO Phase 3 Trial2,4
A total of 389 patients with R/R CLL who had received at least one prior therapy were enrolled in the international, multicenter, open-label, randomized Phase 3 MURANO trial. The trial was designed to evaluate the efficacy and safety of VENCLYXTO/VENCLEXTA and rituximab (n=194) compared with bendamustine and rituximab (n=195). The median age of patients in the trial was 65 years (range: 22 to 85).

The trial met its primary efficacy endpoint of investigator (INV)-assessed PFS. At the time of the primary analysis, median PFS with VENCLYXTO/VENCLEXTA and rituximab was not reached compared with 17.0 months for bendamustine and rituximab (HR: 0.17; 95% CI: 0.11- 0.25; p<0.0001). In the primary efficacy analysis, the median follow-up for PFS was 23.8 months (range: 0 to 37.4). Additional efficacy endpoints included independent review committee (IRC)-assessed PFS, INV- and IRC-assessed overall response rate (defined as complete response + complete response with incomplete marrow recovery + partial response + nodular partial response), OS and rates of MRD-negativity.

In patients with CLL receiving combination therapy with rituximab, the most frequent serious adverse reaction (AR; ≥5%) was pneumonia (9%). The most common ARs (≥20%) of any grade were neutropenia (65%), diarrhea (40%), upper respiratory tract infection (39%), fatigue (22%), and nausea (21%). Fatal ARs that occurred in the absence of disease progression and within 30 days of the last venetoclax treatment and/or 90 days of the last rituximab were reported in 2% (4/194) of patients.

About VENCLYXTO (venetoclax)

VENCLYXTO (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLYXTO targets the BCL-2 protein and works to help restore the process of apoptosis.

VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood and other cancers. Venetoclax is approved in more than 80 countries, including the U.S.

Indication and Important VENCLYXTO (venetoclax) EU Safety Information5

Indications

Venclyxto in combination with obinutuzumab is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).

Venclyxto in combination with rituximab is indicated for the treatment of adult patients with CLL who have received at least one prior therapy.

Venclyxto monotherapy is indicated for the treatment of CLL:

In the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor, or
In the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor.
Venclyxto in combination with a hypomethylating agent is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy.

Contraindications

Hypersensitivity to the active substance or to any of the excipients is contraindicated. Concomitant use of strong CYP3A inhibitors at initiation and during the dose-titration phase due to increased risk for tumour lysis syndrome (TLS). Concomitant use of preparations containing St. John’s wort as Venclyxto efficacy may be reduced.

Special Warnings & Precautions for Use

Tumour Lysis syndrome, including fatal events, has occurred in patients when treated with Venclyxto. For CLL and AML, please refer to the indication-specific recommendations for prevention of TLS in the Venclyxto summary of product characteristic (SmPC).

Patients should be assessed for risk and should receive appropriate prophylaxis, monitoring, and management for TLS. The risk of TLS is a continuum based on multiple factors, including comorbidities. Venclyxto poses a risk for TLS at initiation and during the dose-titration phase. Changes in electrolytes consistent with TLS that require prompt management can occur as early as 6 to 8 hours following the first dose of Venclyxto and at each dose increase.

Neutropenia (grade 3 or 4) has been reported. Complete blood counts should be monitored throughout the treatment period.

In patients with AML, neutropenia (grade 3 or 4) is common before starting treatment. The neutrophil counts can worsen with Venetoclax in combination with a hypomethylating agent. Neutropenia can recur with subsequent cycles of therapy. Dose modification and interruptions for cytopenias are dependent on remission status.

For CLL and AML, please refer to the indication-specific recommendations for dose modifications for toxicities in the Venclyxto SmPC.

Serious infections including sepsis with fatal outcome have been reported. Monitoring of any signs and symptoms of infection is required. Suspected infections should receive prompt treatment including antimicrobials and dose interruption or reduction as appropriate.

Live vaccines should not be administered during treatment or thereafter until B-cell recovery.

Drug Interactions

In CLL and AML CYP3A inhibitors may increase Venclyxto plasma concentrations.

In CLL, at initiation and dose-titration phase, Strong CYP3A inhibitors are contraindicated due to increased risk for TLS and moderate CYP3A inhibitors should be avoided. If moderate CYP3A inhibitors must be used, please refer to the recommendations for dose modifications in the Venclyxto SmPC.

In AML, please refer to the AML-specific recommendation for dose modifications for potential interactions with CYP3A inhibitors, in the VENCLYXTO SmPC.

Avoid concomitant use of P-gp and BCRP inhibitors at initiation and during the dose titration phase.

CYP3A4 inducers may decrease Venclyxto plasma concentrations. Avoid coadministration with strong or moderate CYP3A inducers. These agents may decrease venetoclax plasma concentrations.

Co-administration of bile acid sequestrants with VENCLYXTO is not recommended as this may reduce the absorption of VENCLYXTO.

Adverse Reactions

CLL

The most commonly occurring adverse reactions (>=20%) of any grade in patients receiving venetoclax in the combination studies with obinutuzumab or rituximab were neutropenia, diarrhoea, and upper respiratory tract infection. In the monotherapy studies, the most common adverse reactions were neutropenia/neutrophil count decreased, diarrhoea, nausea, anaemia, fatigue, and upper respiratory tract infection.

The most frequently occurring serious adverse reactions (>=2%) in patients receiving venetoclax in combination with obinutuzumab or rituximab were pneumonia, sepsis, febrile neutropenia, and TLS. In the monotherapy studies, the most frequently reported serious adverse reactions (>=2%) were pneumonia and febrile neutropenia.

Discontinuations due to adverse reactions occurred in 16% of patients treated with venetoclax in combination with obinutuzumab or rituximab in the CLL14 and Murano studies, respectively. In the monotherapy studies with venetoclax, 11% of patients discontinued due to adverse reactions.

Dosage reductions due to adverse reactions occurred in 21% of patients treated with the combination of venetoclax and obinutuzumab in CLL14, in 15% of patients treated with the combination of venetoclax and rituximab in Murano, and in 14% of patients treated with venetoclax in the monotherapy studies. The most common adverse reaction that led to dose interruptions was neutropenia.

AML

The most commonly occurring adverse reactions (>=20%) of any grade in patients receiving venetoclax in combination with azacitidine or decitabine in the VIALE-A and M14-358, respectively, were thrombocytopenia, neutropenia, febrile neutropenia, nausea, diarrhoea, vomiting, anaemia, fatigue, pneumonia, hypokalaemia, and decreased appetite, haemorrhage, dizziness/syncope, hypotension, headache, abdominal pain, and anaemia.

The most frequently reported serious adverse reactions (≥5%) in patients receiving venetoclax in combination with azacitidine were febrile neutropenia, pneumonia, sepsis and haemorrhage. In M14-358, the most frequently reported serious adverse reactions (≥5%) were febrile neutropenia, pneumonia, bacteraemia and sepsis.

Discontinuations due to adverse reactions occurred in 24% of patients treated with venetoclax in combination with azacitidine in the VIALE-A study, and 26% of patients treated with venetoclax in combination with decitabine in the M14-358 study, respectively.

Dosage reductions due to adverse reactions occurred in 2% of patients in VIALE-A, and in 6% of patients in M14-358. Venetoclax dose interruptions due to adverse reactions occurred in 72% and 65% of patients, respectively. The most common adverse reaction that led to dose interruption (>10%) of Venetoclax in VIALE-A, were febrile neutropenia, neutropenia, pneumonia, and thrombocytopenia. The most common adverse reactions that led to dose interruption (≥5%) of venetoclax in M14-358 were febrile neutropenia, neutropenia/neutrophil count decreased, pneumonia, platelet count decreased, and white blood cell count decreased.

Special Populations

Patients with reduced renal function (CrCl <80 mL/min) may require more intensive prophylaxis and monitoring to reduce the risk of TLS at initiation and during the dose-titration phase. Safety in patients with severe renal impairment (CrCl <30 mL/min) or on dialysis has not been established, and a recommended dose for these patients has not been determined.

For patients with severe (Child-Pugh C) hepatic impairment, a dose reduction of at least 50% throughout treatment is recommended.

Venclyxto may cause embryo-fetal harm when administered to a pregnant woman. Advise nursing women to discontinue breastfeeding during treatment.

This is not a complete summary of all safety information. See Venclyxto (venetoclax) SmPC at www.ema.europa.eu. Globally, prescribing information varies; refer to the individual country product label for complete information.

Servier Presents Updated Results for TIBSOVO® (ivosidenib tablets) in IDH1-mutated Relapsed/Refractory Myelodysplastic Syndromes at the 2023 European Hematology Association (EHA) Congress

On June 9, 2023 Servier, a leader in oncology committed to bringing the promise of tomorrow to the patients we serve, reported updated data from the Phase 1 trial of TIBSOVO (ivosidenib tablets) as monotherapy for patients with isocitrate dehydrogenase 1 (IDH1)-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS) (Press release, Servier, JUN 9, 2023, View Source [SID1234632623]). The results presented today at the European Hematology Association (EHA) (Free EHA Whitepaper) 2023 Annual Congress in Frankfurt, Germany demonstrate that the efficacy and safety profile of TIBSOVO may provide an important new treatment option for MDS patients within this molecularly defined subset.

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The Phase 1, open-label study included an evaluation of the safety, tolerability, and clinical activity of TIBSOVO in patients with IDH1-mutated R/R MDS. The primary endpoint was complete remission (CR) plus partial remission (PR) rate and key secondary endpoints included duration of CR plus PR, duration of transfusion independence, and time to transfusion independence.

In the efficacy analysis set (n=18), a complete remission (CR) rate of 38.9% and overall response rate (ORR) of 83.3% were documented in patients treated with TIBSOVO. In addition, the median time to CR was 1.87 months (range: 1.0, 5.6). At the time of data cutoff, the median duration of CR had not been reached (range: 1.9, 80.8*) and the median overall survival was 35.7 months (range: 3.7*, 88.7*).

"We are pleased to share these updated efficacy results that demonstrate durable remissions and an acceptable safety profile in patients with IDH1-mutated relapsed or refractory MDS at this year’s EHA (Free EHA Whitepaper) Congress," said Susan Pandya, M.D., Vice President Clinical Development and Head of Cancer Metabolism Global Development Oncology & Immuno-Oncology, Servier. "These data add to the growing body of evidence behind targeted IDH inhibition, and we look forward to taking the next steps with regulatory authorities to potentially expand the use of TIBSOVO in the United States to include the treatment of IDH1-mutated relapsed or refractory MDS."

Additionally, of the nine patients who were transfusion dependent with red blood cells or platelets at baseline, 66.7% (n=6) became independent of transfusions during any ≥56-day post-baseline period. Further, of the nine patients who were transfusion independent with red blood cells or platelets at baseline, 77.8% (n=7) maintained transfusion independence during any ≥56-day post-baseline period.

"Patients with IDH1-mutated relapsed or refractory MDS currently have no targeted therapy options, and outcomes are generally poor for those who experience disease progression after treatment with standard care," said Courtney D. DiNardo, M.D., MSCE, Associate Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, and investigator on the study. "This updated analysis demonstrates that TIBSOVO has the potential to improve outcomes in the treatment of IDH1-mutated relapsed or refractory MDS and reinforces the importance of molecular analysis to ensure we’re harnessing the potential benefit of targeted therapies."

Overall, treatment-related adverse events (TRAEs) were consistent with the known safety profile of TIBSOVO. Among 19 patients included in the safety analysis set, TRAEs occurred in eight (42.1%) patients, including a grade 1 QTc interval increase in one (5.3%) patient, grade 3 fatigue in one patient, and grade 3 hyponatremia in one patient, none of which led to discontinuation with treatment.

In 2019, the U.S. FDA granted Breakthrough Therapy designation for TIBSOVO (ivosidenib) for the treatment of adult patients with relapsed or refractory myelodysplastic syndromes with a susceptible isocitrate dehydrogenase 1 (IDH1) mutation as detected by an FDA-approved test. Servier plans to submit a supplemental New Drug Application (sNDA) based on these results to the U.S. FDA for TIBSOVO in adult patients with R/R IDH1-mutated myelodysplastic syndromes.

TIBSOVO is currently approved in the U.S. as monotherapy for the treatment of adults with IDH1-mutant relapsed or refractory AML and in monotherapy or in combination with azacitidine for adults with newly diagnosed IDH1-mutant AML who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy. TIBSOVO was recently approved by the European Commission as a targeted therapy in two indications: in combination with azacitidine for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy; as well as in monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy. TIBSOVO has also been approved in the U.S. and Australia for patients with previously treated IDH1-mutated cholangiocarcinoma. TIBSOVO is also approved in China[i] for the treatment of adult patients with relapsed or refractory AML who have a susceptible IDH1 mutation. Servier has granted CStone a co-exclusive license for the development and an exclusive license agreement for the commercialization of TIBSOVO in Mainland China, Taiwan, Hong Kong, Macao and Singapore.

* Denotes a censored observation.

About the Study
In the Phase 1 dose-escalation and expansion study evaluating TIBSOVO in adults with advanced hematologic malignancies with IDH1 mutations, the clinical activity, safety, tolerability, pharmacokinetics and pharmacodynamics of TIBSOVO in adult patients with relapsed or refractory MDS with a susceptible IDH1 mutation is being assessed. (NCT03503409)

About Myelodysplastic Syndromes (MDS)
MDS comprises a diverse group of bone marrow disorders in which immature blood cells in the bone marrow do not mature or become healthy blood cells. The Leukemia and Lymphoma Society estimates that more than 20,000 people are diagnosed with MDS in the U.S. each year. Failure of the bone marrow to produce mature healthy cells is a gradual process, and reduced blood cell and/or reduced platelet counts may be accompanied by the loss of the body’s ability to fight infections and control bleeding. For roughly 30 percent of the patients diagnosed with MDS, this bone marrow failure will progress to AML1. Chemotherapy, supportive therapy, stem cell transplant, growth factors, and similar approaches are used to treat MDS.

MEDSIR Study Finds No Progression-Free Survival Benefit with Palbociclib Maintenance After First-Line Regimen in HR+/HER2- Advanced Breast Cancer Patients

On June 9, 2023 MEDSIR reported the results of the PALMIRA trial (Press release, MedSIR, JUN 9, 2023, View Source;advanced-breast-cancer-patients-301847256.html [SID1234632622]). This randomized phase 2 study evaluated the safety and efficacy of palbociclib maintenance in combination with second-line endocrine therapy in patients with hormone receptor-positive/HER2-negative (HR+/HER2-) advanced breast cancer who had showed a confirmed progressive disease on first-linepalbociclib plus endocrine therapy-based treatment after having achieved clinical benefit to this regimen. The results of this trial demonstrate that palbociclib maintenance does not significantly improve progression-free survival, the primary endpoint of the study, compared with second-line endocrine therapy alone in this patient population.

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The PALMIRA study is sponsored by MEDSIR, a company dedicated to advancing clinical research in oncology, and was led by Dr. Antonio Llombart-Cussac along with Dr. Javier Cortés and Dr. José Pérez as Scientific Coordinators. The trial involved 198 patients with HR+/HER2- advanced breast cancer from 41 centers in 6 European countries.

Endocrine therapy is the mainstay of treatment for patients with HR+/HER2- advanced breast cancer. Several studies have demonstrated that the addition of cyclin-dependent kinases 4 and 6 (CDK4/6) to first- and second-line endocrine therapy leads to an improved progression-free survival and overall survival. However, the optimal treatment after progression on a CDK4/6 inhibitor in patients with HR+/HER2- advanced breast cancer remains undetermined.

One of the therapeutic options that have been evaluated in this scenario is the maintenance of CDK4/6 inhibition beyond progression switching the endocrine therapy and maintaining or shifting the CDK4/6 inhibitor or continuing the same endocrine therapy and changing to a different CDK4/6 inhibitor. Some of these studies had already suggested a continued benefit from this clinical approach but they must be interpreted with caution due to potential biases, mainly the use of a different CDK4/6 inhibitor.

The PALMIRA study aimed to determine if palbociclib maintenance could improve the antitumor activity of second-line endocrine therapy in HR+/HER2- advanced breast cancer patients who had showed a confirmed progressive disease on first-linepalbociclib plus endocrine therapy-based treatment after having achieved clinical benefit to this regimen.

"Results of the PALMIRA trial, while negative, will guide further research on the optimal treatment upon progression to CDK4/6 inhibitors and our clinical practice," said Dr. Llombart-Cussac, principal investigator of the study and Head of Service at Arnau de Vilanova Hospital in Valencia, Spain. Findings from the PALMIRA study indicate that "re-treatment with the same CDK4/6 inhibitor and switching the endocrine therapy does not improve patient outcomes."

Dr. Cortés, director of the International Breast Cancer Center, stated, "based on the results of the PALMIRA study, we suspect that there is a subgroup of patients that could benefit from maintaining palbociclib after prior progression on a palbociclib-based regimen. Biomarkers are the way to identify these patients and optimize the best future treatment options for them."