On November 18, 2024 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that it has received CE Mark for its VENTANA FOLR1 (FOLR1-2.1) RxDx Assay. This is the first immunohistochemistry (IHC) companion diagnostic test to be made widely available in Europe to aid in identifying epithelial ovarian cancer (EOC) patients who may be eligible for targeted treatment with ELAHERE (mirvetuximab soravtansine) (Press release, Hoffmann-La Roche, NOV 18, 2024, View Source [SID1234648485]). ELAHERE is a first-in-class antibody-drug conjugate (ADC) therapy developed by AbbVie for the treatment of FRɑ-positive platinum-resistant ovarian cancer.

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This certification follows the news earlier this year that the VENTANA FOLR1 (FOLR1-2.1) test had received pre-authorisation in Germany and Austria. This exceptional decision allowed clinicians and patients in these countries to access the test ahead of the CE Mark certification.

"This certification will allow us to extend the reach of our innovative diagnostic solutions," said Jill German, Head of Pathology Lab at Roche Diagnostics. "The early exemption approval in Germany and Austria highlighted the urgent need for this test. Now, clinicians across Europe can access a critical tool to quickly identify ovarian cancer patients who may be eligible for targeted therapy. By enabling more precise and personalised treatment decisions, we hope this may help improve outcomes for the many women in Europe facing this devastating disease."

Folate receptor 1 protein (FOLR1), also known as folate receptor alpha (FRɑ), is expressed at some level in approximately 90 percent of ovarian carcinomas and serves as a predictive biomarker for FOLR1-targeted therapy for EOC patients.1,2 The VENTANA FOLR-1 (FOLR1-2.1) test informs clinicians about the likelihood of potential patient benefit from FOLR1 therapy,3,4 advancing Roche’s commitment to personalised healthcare through innovative solutions that help fit the treatment to the individual.

Ovarian cancer is the eighth overall cause for cancer death in women worldwide, representing 4.7% of all cancer deaths in women. It is also one of the deadliest gynaecological cancers worldwide. In 2022, 46,232 women in Europe and 209,596 women worldwide died from ovarian cancer.5

The launch of Roche’s first IHC companion test for ovarian cancer in CE countries highlights the company’s commitment, as the world’s leading provider of in vitro diagnostics, to continued innovation and evolution of its products in order to advance personalised healthcare and deliver novel, high medical value solutions that improve patients’ lives.

About the VENTANA FOLR1 (FOLR1-2.1) RxDx Assay
Roche has developed a leading, comprehensive and differentiated cancer immunohistochemical portfolio, with biomarkers that support multiple guidelines for the diagnosis and stratification of cancers. VENTANA FOLR1 (FOLR1-2.1) RxDx Assay is a qualitative immunohistochemical assay using mouse monoclonal anti-FOLR1 clone FOLR1-2.1 intended for use in the assessment of folate receptor alpha (FRɑ) in formalin-fixed, paraffin-embedded epithelial ovarian cancer (EOC), including primary peritoneal cancer and primary fallopian tube cancer, tissue specimens by light microscopy. The OptiView DAB IHC Detection Kit is used for staining on a BenchMark ULTRA instrument.

The approval is based on the results from the SORAYA6 and MIRASOL7 clinical studies. Both studies enrolled platinum-resistant epithelial ovarian cancer patients who were FRɑ-positive by the VENTANA FOLR1 (FOLR1-2.1) RxDx Assay. In the single-arm SORAYA trial, 32% of patients demonstrated a partial or complete response to ELAHERE therapy.5 In the MIRASOL trial, patients who received ELAHERE demonstrated a significant improvement in progression-free survival by investigator assessment compared with IC chemotherapy, which represented a 35% reduction in the risk of tumour progression or death (HR 0.65, 95% CI, 0.52-0.81). Patients who received ELAHERE also demonstrated a significant improvement in overall survival compared to chemotherapy, which represented a 33% reduction in the risk of death (HR 0.67, 95% CI, 0.50-.0.89).

On November 18, 2024 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported abstracts detailing selinexor data have been selected to be presented at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, being held December 7-10, 2024 in San Diego, CA (Press release, Karyopharm, NOV 18, 2024, View Source [SID1234648471]).

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"We are pleased to share presentations on selinexor at this year’s annual meeting," said Reshma Rangwala, MD, PhD, Chief Medical Officer and Head of Research at Karyopharm. "The data being presented demonstrate our commitment, as well as that of our partners to our ongoing work in multiple myeloma and myelofibrosis."

Details for the ASH (Free ASH Whitepaper) 2024 selected Karyopharm abstracts are as follows:

Abstract Title

Presentation Type

Abstract #

Session Date/Time

Multiple Myeloma

Efficacy and Safety of Selinexor, Pomalidomide, and Dexamethasone (SPd) for Treatment of Patients with Relapsed or Refractory Multiple Myeloma (RRMM)

Poster

1996

Saturday, December 7, 2024

5:30pm – 7:30pm PST/ 8:30pm – 10:30pm EST

Myelofibrosis

Selinexor Depletes Ruxolitinib Refractory Myelofibrosis Hematopoietic Stem Cells By Inducing Apoptosis and Blunting the Pro-Inflammatory Milieu

Poster

1377

Saturday, December 7, 2024

5:30pm – 7:30pm PST/ 8:30pm – 10:30pm EST

Details for a selected partner presentation on Antengene’s latest data are as follows:

Abstract Title

Presentation Type

Abstract #

Session Date/Time

Multiple Myeloma

Weekly Selinexor, Bortezomib and Dexamethasone (SVd)
Versus Twice Weekly Bortezomib and Dexamethasone (Vd) in Chinese Patients with Relapsed and Refractory Multiple Myeloma: Primary Analysis of Phase 3 Bench Study

Poster

4748

Monday, December 9, 2024

6:00pm – 8:00pm PST/

9:00pm – 11:00pm EST

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with VELCADE (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) under accelerated approval in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in a growing number of ex-U.S. territories and countries, including Europe, the United Kingdom, China, South Korea and Israel, and is marketed in those areas by Karyopharm’s global partners. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer and myelofibrosis.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at: Tel: +1 (888) 209-9326; Email: [email protected]

XPOVIO (selinexor) is a prescription medicine approved in the United States:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti‐CD38 monoclonal antibody (Xd).
For the treatment of adult patients with relapsed or refractory diffuse large B‐cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo-Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3-4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3-4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
Use In Specific Populations
Lactation: Advise not to breastfeed.

For additional product information, including full prescribing information, please visit www.XPOVIO.com.

To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1-888-209-9326 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

On November 18, 2024 Bexion Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing a novel class of biologic therapy to treat solid tumor cancers and chemotherapy-induced peripheral neuropathy (CIPN), reported completion of enrollment in the open label portion of Phase 1b/2 ASIST study of BXQ-350 in combination with Standard-of-Care (SOC) for the first line treatment of metastatic colorectal cancer (mCRC) (Press release, Bexion, NOV 18, 2024, View Source [SID1234648486]).

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"I am delighted that Bexion has completed enrollment for its Phase 1b/2 clinical trial and am especially pleased my clinical site has made significant contributions toward this effort," said Reema A. Patel, MD, Associate Professor of Medicine at the Markey Cancer Center of the University of Kentucky. "BXQ-350’s mechanism of action lends itself to potential benefit in metastatic colorectal patients and peripheral neuropathy. Severe neuropathy is a key reason that patients drop off chemotherapy for colorectal cancer, and BXQ-350 has been shown to possess potential anti-neuropathy properties. I am excited by the potential of BXQ-350 to reduce or even potentially reverse neuropathic pain in these patients."

The ASIST study will assess the safety and efficacy of BXQ-350 plus SOC, modified FOLFOX7 (mFOLFOX7) and bevacizumab, in patients with newly diagnosed mCRC. The study is also evaluating whether the administration of BXQ-350 with mFOLFOX7 and bevacizumab may diminish CIPN, enabling participants to receive the total and planned doses of mFOLFOX7.

"We extend our gratitude to all the patients, clinical investigators, and site research staff who continue to contribute to the advances we are making," said Jim Beach, Chief Executive Officer of Bexion. "We are excited about advancing to the next stage of clinical development, including further discussions with FDA on study design, and we look forward to providing updates as we reach upcoming milestones in 2025."

More information about the clinical trial is available at clinicaltrials.gov (ASIST study; NCT05322590).

About BXQ-350
Bexion’s lead drug candidate is BXQ-350, a first-in-class biologic containing the multifunctional sphingolipid activator protein, Saposin C, and a phospholipid. Multiple Phase 1 clinical trials in adult and pediatric patients have demonstrated a robust safety profile for BXQ-350 with evidence of single agent activity across multiple solid tumor types. Additionally, other clinical and non-clinical data suggest BXQ-350 has activity in chemotherapy-induced peripheral neuropathy, an area of high unmet medical need in patients treated with oxaliplatin and other chemotoxic agents.

On November 18, 2024 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported new findings supporting the ability of Annamycin to overcome resistance to Venetoclax in acute myeloid leukemia ("AML") (Press release, Moleculin, NOV 18, 2024, View Source [SID1234648472]). This includes data from preclinical in vitro studies recently accepted for online publication at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting, and correlates with efficacy demonstrated by recent preliminary clinical data in subjects who were relapsed from or refractory to first line Venetoclax regimens and were then treated with Annamycin in combination with Ara-C ("AnnAraC").

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Jorge Cortes, MD, Director of the Georgia Cancer Center at Augusta University and a member of the Company’s Scientific Advisory Board, commented, "Although Venetoclax has been an important improvement in first line therapy for AML patients who are unfit for intensive chemotherapy, the rate of relapse is high and overall survival post relapse is just a few months. This turns out to be a large percentage of AML patients in total and we clearly need a better treatment option."

Michael Andreeff, MD, PhD, Professor of Medicine, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center and a member of the Company’s Scientific Advisory Board, added, "Many patients get Ven-Aza, not because they are ‘unfit’ but because of the high initial response rates. When they relapse, however, our options are very limited. Annamycin combined with Ara-C could significantly advance the standard of care and provide better outcomes for these high-risk patients. I am excited to be a part of the next step in the development of this important asset."

A prior publication, Outcomes of relapsed or refractory acute myeloid leukemia after frontline hypomethylating agent and venetoclax regimens1, reported that the CR/CRi2 rate for salvage therapy using available standard of care in AML subjects who relapsed from or were refractory to Venetoclax regimens was 12.5% (n=24, 4% CR). The new preliminary clinical findings announced today in the MB-106 clinical trial indicate that relapsed or refractory ("R/R") AML subjects previously treated with Venetoclax regimens achieved a 60% CR/CRi rate (n=5, 40% CR), more than 4 times the rate that would be expected based on the above referenced paper. As previously disclosed in MB-106, there was an overall CR/CRi rate of 60% (50% CR) in 2nd line subjects (n=10) and 41% (36% CR) in all subjects, 1st-7th line (n=22).

An abstract titled, "Annamycin, a non-cardiotoxic anthracycline, demonstrates unique organotropism and activity against Ara-C and Venetoclax resistant AML," supporting the clinical activity of Annamycin was accepted for online publication at the ASH (Free ASH Whitepaper) Annual Meeting being held December 7-10, 2024, in San Diego, CA. The abstract will be published in a supplemental issue of Blood, expected in late November, and will become part of the permanent ASH (Free ASH Whitepaper) and Blood abstracts archive following the conclusion of the Annual Meeting.

Additionally, new preliminary data from the Company’s Phase 1B/2 clinical trial evaluating AnnAraC for the treatment of subjects with AML as both first line therapy and for subjects who were refractory to or relapsed after induction therapy (MB-106) demonstrated median overall survival ("OS") of 9.1 months for subjects with a wide range of (0-6) prior lines of therapy (n=22) and 11.6 months (n=10) for subjects with 1 prior line of therapy (second line therapy).

"Moleculin is focusing on development of Annamycin to address the significant unmet need in R/R AML. The growing body of preliminary data continue to bolster our confidence in the safety and efficacy of Annamycin, and its potential to provide patients and physicians with a promising new treatment option in AML," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "We believe the latest preliminary OS data we are seeing in our MB-106 trial can now be considered exceptional and we look forward to the initiation of our pivotal registration study, especially now that our recent protocol amendment allows for disclosing unblinded data for the first 45 subjects, which we expect within the next 12 months."

The Company is advancing the development of Annamycin in a Phase 3 pivotal trial evaluating AnnAraC for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (MB-108). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US. The Company remains on track to initiate patient treatment in the first quarter of 2025.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).

On November 18, 2024 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported the publication in Science Immunology of preclinical data demonstrating the potential of IPH6501, Innate’s proprietary NK cell engager including an IL-2v and targeting CD20 from the ANKET platform (Press release, Innate Pharma, NOV 18, 2024, View Source [SID1234648487]). IPH6501 is currently evaluated in a Phase 1/2 clinical trial in B-cell non-Hodgkin lymphoma (B-NHL) (NCT06088654).

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The data published shows that IPH6501, also called CD20-NKCE-IL2v in the publication, boosts NK cell proliferation and cytotoxicity, showing activity against a range of B-NHL cell lines, including those with low CD20 density. In vivo studies in nonhuman primates and tumor mouse models further validated its efficacy and revealed that IPH6501 induces peripheral NK cell migration at the tumor site.

"These new findings underscore the remarkable potential of IPH6501 to transform the treatment landscape for B-NHL. IPH6501 showed greater killing efficacy over a T cell engager targeting CD20 in in vitro preclinical models while presenting reduced toxicities compared with those commonly associated with T cell therapies. We are particularly encouraged by results that reveal IPH6501’s ability to drive NK cell migration directly to tumor sites, highlighting its potential as a game-changer in immuno-oncology. These findings are a testament to the promise of NK cell therapies to deliver safer and more targeted solutions for patients in need," commented Eric Vivier, DVM, PhD, Chief Scientific Officer of Innate Pharma.

The data published further support the current clinical development plan of IPH6501 in Relapsed/Refractory B-NHL.

About ANKET

ANKET (Antibody-based NK cell Engager Therapeutics) is Innate’s proprietary platform for developing next-generation, multi-specific natural killer (NK) cell engagers to treat certain types of cancer. This versatile, fit-for-purpose technology is creating an entirely new class of molecules to induce synthetic immunity against cancer.

About IPH6501

IPH6501 is the first Antibody-based NK cell Engager Therapeutic to co-engage activating receptors on NK cells (NKp46 and CD16), IL-2R (but not a subunit) through a variant of human IL-2, and a tumor antigen (CD20) via a single molecule, hence providing proliferation and activation signals targeted to NK cells and promoting their cytotoxic activity against CD20 expressing malignant cells.

IPH6501 has shown better anti-tumor efficacy than approved benchmark antibodies in preclinical tumor models (Demaria, EHA (Free EHA Whitepaper) 2023, Carrette, SITC (Free SITC Whitepaper) 2024, Demaria et al, Science Immunology 2024).

IPH6501 is currently being evaluated in a Phase 1/2 multicenter trial (NCT06088654), investigating the safety and tolerability of IPH6501 in patients with Relapsed and/or Refractory CD20-expressing B-cell Non-Hodgkin’s Lymphoma.