On December 4, 2024 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncological and inflammatory diseases, reported that a patient currently treated with Namodenoson in a compassionate use program in Can-Fite’s Phase II Liver Cancer Study has an overall survival time of 8 years with a complete response (Press release, Can-Fite BioPharma, DEC 4, 2024, View Source [SID1234648786]).

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The patient, who suffered from advanced liver cancer was enrolled in the former Can-Fite Phase II study, continue to be treated with Namodenoson, and has now an overall survival of 8 years, with disappearance of ascites, normal liver function, good quality of life and is defined as a long term complete response.

Can-Fite is currently enrolling patients in Israel, Europe and the US for a pivotal Phase III clinical study for patients with advanced HCC as a 2nd or 3rd line treatment and Namodenoson is administered twice daily orally. The study protocol has been agreed upon with U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA).

Namodenoson has Orphan Drug status with both the FDA and EMA, as well as Fast Track Status with the FDA for the treatment of HCC. A compassionate use program has been ongoing in Israel and Romania.

"With a very favorable safety profile and anti-cancer effect of Namodenoson, we are now enrolling patients for the pivotal Phase III clinical study where we expect to prolong patients’ overall survival, and see a response similar to that of the patient who has now been treated with Namodenoson for 8 years. The uniqueness of Namodenoson which specifically acts against the tumor cells and protects the normal liver cells, is the rationale for the conductance of the current trial," stated Prof. Salomon Stemmer, a leading key opinion leader, at the Institute of Oncology, Rabin Medical Center, Israel.

According to the American Cancer Society, liver cancer accounts for more than 700,000 deaths globally each year. HCC is commonly aggressive with poor survival rates. As new drugs that effectively and safely treat HCC are developed and approved, the market for HCC treatments is estimated by Delveinsight to reach $6.1 billion by 2027 for the G8 countries.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson was evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

On December 4, 2024 Phanes Therapeutics, Inc. (Phanes), a clinical stage biotech company focused on innovative drug discovery and development in oncology, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PT217 for the treatment of patients with metastatic de novo or treatment-emergent neuroendocrine prostate cancer (NEPC) (Press release, Phanes Therapeutics, DEC 4, 2024, View Source [SID1234648807]). This is the second Fast Track Designation granted to PT217 by the FDA. Earlier this year, PT217 was granted Fast Track designation for extensive-stage small cell lung cancer (ES-SCLC) with disease progression following platinum chemotherapy with or without a checkpoint inhibitor by the agency.

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PT217, a first-in-class native IgG-like bispecific antibody (bsAb) targeting DLL3 and CD47, is being developed for the treatment of patients with small cell lung cancer (SCLC) and neuroendocrine carcinoma, including neuroendocrine prostate cancer (NEPC). In addition to Fast Track designation, PT217 was also granted orphan drug designations for the treatment of small cell lung cancer and neuroendocrine carcinoma (NEC), respectively.

The multi-center Phase I/II clinical trial of PT217 (NCT05652686), known as the SKYBRIDGE study, is currently evaluating the safety, tolerability, pharmacokinetics and preliminary efficacy of PT217 in patients with advanced or refractory cancers expressing DLL3. A Phase I clinical trial of PT217 is also ongoing in China (CTR20242720). Earlier this year, Phanes entered into a clinical supply agreement with Roche to study PT217 in combination with Roche’s anti-PD-L1 therapy, atezolizumab.

On December 3, 2024 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that it is in discussions and has an upcoming meeting with the U.S. Food and Drug Administration (FDA) regarding the evolving treatment landscape in endometrial cancer and any implications this may have with respect to the Company’s Phase 3 XPORT-EC-042 trial (Press release, Karyopharm, DEC 3, 2024, View Source [SID1234648751]). In light of this, Karyopharm does not plan to discuss its endometrial cancer program during the Piper Sandler 36th Annual Healthcare Conference being held in New York, NY today. The Company intends to provide an update on its endometrial cancer program as soon as practical following the discussion with FDA.

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A live webcast of the fireside chat at the Piper Sandler 36th Annual Healthcare Conference at 11:30 a.m. ET today can be accessed under "Events & Presentations" in the Investor section of the Company’s website, View Source, and will be available for replay following the event.

About the EC-042 Study
EC-042 (XPORT-EC-042; NCT05611931) is a global, Phase 3, randomized, double-blind study evaluating selinexor as a maintenance therapy following systemic therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer. The EC-042 study was initiated in November 2022 and is expected to enroll up to 220 patients who will be randomized 1:1 to receive either a 60 mg, once-weekly, administration of oral selinexor or placebo until disease progression. The primary endpoint of the study is progression free survival, as assessed by an investigator, with overall survival as a key secondary endpoint. Further, in connection with the EC-042 Study, Karyopharm entered into a global collaboration with Foundation Medicine, Inc. to develop FoundationOneCDx, a tissue-based comprehensive genomic profiling test to identify and enroll patients whose tumors are TP53 wild-type.

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 various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, Mainland China, Taiwan, Hong Kong, Australia, South Korea, Singapore, Israel, and Canada. XPOVIO/NEXPOVIO is marketed in these respective ex-U.S. territories by Karyopharm’s partners: Antengene, Menarini, Neopharm, and FORUS. 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 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 December 3, 2024 SciTech Development, a clinical-stage pharmaceutical company focused on advancements in cancer treatment, reported promising updates from its clinical trial of ST-001 nanoFenretinide (ST-001) (Press release, SciTech Development, DEC 3, 2024, View Source [SID1234648770]).

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Preliminary Trial Findings
Preliminary data from the accelerated Phase 1a trial in patients with Cutaneous T-cell Lymphoma indicates favorable clinical improvements to ST-001 treatment, including earlier-than-anticipated stable disease and partial responses – one initial and one confirmed.

In addition, results from patients treated with ST-001 demonstrate that the advanced nanoparticle delivery system successfully achieves the desired pharmacokinetic profile. It enables rapid and targeted delivery of fenretinide directly to tissues while minimizing side effects and toxicity. These findings exceed expectations and mark a significant milestone in the continued development of ST-001.

ST-001 Clinical Trial Information
SciTech’s initial trial is targeting T-cell non-Hodgkin lymphoma (T-cell NHL) indications including all sub-types of Cutaneous T-cell lymphoma (CTCL), Mycosis Fungoides (MF), Sézary Syndrome (SS), Angioimmunoblastic T-cell lymphoma (AITL), and other types of systemic T-cell lymphomas. The trial is activated and enrolling at 8 prestigious cancer institutions across the U.S., supported by world-renowned scientific advisors and principal investigators specializing in T-cell NHL. View Source

"The early findings from the Phase 1a trial of ST-001 nanoFenretinide are incredibly promising. Achieving a partial response at this stage is a remarkable step forward and highlights the potential of this innovative treatment to address significant gaps in care for patients with Cutaneous T-cell Lymphoma (CTCL)," said Dr. Larisa Geskin, Director of the Comprehensive Cutaneous Oncology Center at Columbia University. "Fenretinide has shown an excellent safety profile so far, with minimal side effects, including no reported thyroid abnormalities or significant lipid issues. If responses continue to improve as doses are escalated, this trial could mark a turning point in CTCL therapy and bring renewed hope to patients."

"We are extremely encouraged by the preliminary data from the ST-001 trial," said Earle Holsapple, CEO of SciTech Development. "The findings to date strengthen our resolve to gather additional data and advance to the next phase of the trial, as we work toward bringing a new therapeutic option to oncology patients."

Trial Design and Advancements
The FDA approved the Phase 1a accelerated dose-escalation design based on extensive research, prior clinical testing, and the safety profile of fenretinide in more than 3,000 patients. This accelerated portion of the trial is designed to achieve early clinical development objectives including confirming safety, assessing pharmacokinetics, pharmacodynamics, and determine the optimal therapeutic dosing in patients with T-cell NHL.

Upcoming Trial Milestones
The accelerated Phase 1a portion is nearing completion. SciTech is preparing to progress to the next phase of the T-cell NHL study and plans to enroll approximately 45 additional patients. An upcoming clinical trial for ST-001 targeting small cell lung cancer (SCLC) is anticipated to commence in Q2 2025.

About ST-001
SciTech’s lead drug candidate, ST-001 nanoFenretinide, is a patented nanoparticle formulation of fenretinide and biocompatible phospholipids, allowing for the rapid infusion (IV) of high-dose fenretinide. ST-001 solves bioavailability challenges, avoids triglyceride toxicity of prior formulations, and optimizes therapeutic efficacy. ST-001 represents a novel therapeutic approach for targeting difficult-to-treat cancers, utilizing patented methods to improve drug safety and efficacy profiles while optimizing treatment outcomes.

On December 3, 2024 Keros Therapeutics, Inc. ("Keros" or the "Company") (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta ("TGF-ß") family of proteins, reported an exclusive global development and commercialization license agreement with Takeda (TSE:4502/NYSE:TAK) to advance elritercept (Press release, Keros Therapeutics, DEC 3, 2024, View Source [SID1234648752]). Elritercept is currently in two ongoing Phase 2 clinical trials; one in patients with very low-, low-, or intermediate-risk myelodysplastic syndrome ("MDS") and one in patients with myelofibrosis ("MF"). The Phase 3 RENEW clinical trial evaluating elritercept in adult patients with transfusion-dependent anemia with very low-, low-, or intermediate-risk MDS will begin enrollment soon.

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Under the terms of the agreement, Takeda will obtain an exclusive license to further develop, manufacture and commercialize elritercept worldwide outside of mainland China, Hong Kong and Macau. Takeda will be responsible for all development, manufacturing and commercialization as of the effective date of the agreement. Subject to the terms of the agreement, Keros will receive a $200 million upfront cash payment and is eligible to receive development, approval and commercial milestones with the potential to exceed $1.1 billion. Keros will also be eligible to receive tiered royalties on net sales.

"We are thrilled to announce this agreement with Takeda, a leader in the hematologic oncology treatment space," said Jasbir S. Seehra, Ph.D., Chair and Chief Executive Officer of Keros. "We believe this global license further validates Keros’ position as a leader in understanding the role of the TGF-ß family of proteins and the broad potential of this biological pathway."

"We believe Takeda is an ideal partner to maximize the potential of elritercept’s differentiated profile and continue to build on the great progress our team has accomplished with elritercept," said Chris Rovaldi, President and Chief Operating Officer of Keros. "We expect that the net proceeds from the upfront payment will enable us to extend our operational runway into the fourth quarter of 2028, facilitating the continued advancement of cibotercept (KER-012) and KER-065, both of which are wholly-owned assets with near term clinical updates."

"We are excited to partner with Keros, an accomplished team with exceptional expertise in TGF-ß biology," said P.K. Morrow, Head of the Oncology Therapeutic Area Unit at Takeda. "Building on the promising results elritercept has shown in the clinic to date, we look forward to continuing to explore its potential and to having the opportunity to potentially deliver it to patients with hematologic disorders. This agreement aligns with our goal of advancing therapies that may shift the treatment paradigm for underserved patient populations."

The effectiveness of the agreement is subject to clearance under the Hart-Scott-Rodino Antitrust Improvements Act ("HSR Act").

About Elritercept

Elritercept is an engineered ligand trap comprised of a modified ligand-binding domain of the TGF-ß receptor known as activin receptor type IIA that is fused to the portion of the human antibody known as the Fc domain. Elritercept is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia, in patients with MDS and in patients with MF.