On May 13, 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 it held a conference for 75 oncologists and coordinators who are conducting the pivotal Phase 3 advanced liver cancer study, to accelerate patient enrolment (Press release, Can-Fite BioPharma, MAY 13, 2024, View Source [SID1234643136]).

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The participants have been presented with the drug chemical and biological profile and the study protocol as well as study procedures. Dr. Lencioni, a key opinion leader in radiology of liver tumors, Department of Radiology, Pisa University School of Medicine Italy, educated the oncologists on the way to measure tumor lesion size prior and post namodenoson treatment. The difference in tumor growth or regression defines progression free survival (PFS) and the objective response rate (ORR) to the drug, serving as part of the study objectives. US experts presented to the participants the electronic data entry of the patients’ data during the trial.

"The conference was very successful and we are very pleased with the enthusiasm of the participants with respect to the drug characteristics. They are looking forward to complete patient enrolment and conclude the data of this very promising anti-cancer drug," said Dr. Pnina Fishman, Can-Fite CSO and Executive Chairperson.

Can-Fite has received agreement from both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) on a pivotal Phase 3 clinical study which is now enrolling patients is Israel, Europe and the US. The study is designed to perform an interim analysis by an Independent Data Monitoring Committee (IDMC) upon enrollment of 50% patients. Namodenoson will be evaluated as a 2nd or 3rd line treatment for CPB7 patients in whom other approved therapies have not been effective.

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.

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 May 13, 2024 Boundless Bio (Nasdaq: BOLD), a clinical-stage oncology company interrogating extrachromosomal DNA (ecDNA) biology to deliver transformative therapies to patients with previously intractable oncogene amplified cancers, reported financial results for the first quarter of 2024 and highlighted recent progress (Press release, Boundless Bio, MAY 13, 2024, View Source [SID1234643135]).

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"It has been an exciting quarter at Boundless Bio. The Phase 1/2 POTENTIATE trial of BBI-355, our potentially best-in-class, oral, selective CHK1 inhibitor, advanced into initial combination therapy modules evaluating BBI-355 together with an EGFR inhibitor or an FGFR inhibitor in patients with tumors harboring EGFR or FGFR oncogene amplifications, respectively. We also dosed the first patient with our second ecDNA-directed therapy (ecDTx), BBI-825, a first-in-class, oral, selective RNR inhibitor, which marks the company’s rapid growth and transition into a multi-asset, clinical-stage oncology company" said Zachary Hornby, President and Chief Executive Officer of Boundless Bio. "With the completion of our recent IPO, we have the capital to take the next steps toward delivering on the promise of our ecDTx, a potential new vertical in cancer therapeutics."

Recent Highlights

BBI-355, a novel CHK1 inhibitor and the first ecDTx in development

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Patient enrollment is ongoing in Part 1 of the Phase 1/2 POTENTIATE (Precision Oncology Trial Evaluating Novel Therapeutic Interrupting Amplifications Tied to ecDNA) trial, which evaluates BBI-355 as a single agent in patients with locally advanced or metastatic solid tumors with oncogene amplifications.

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Initiated dose escalation in Part 2 of the Phase 1/2 POTENTIATE trial, which evaluates BBI-355 in combination with the EGFR inhibitor erlotinib and BBI-355 in combination with the FGFR inhibitor futibatinib in patients with tumors harboring EGFR or FGFR oncogene amplifications,

respectively, to evaluate the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of each combination regimen.

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Presented preliminary preclinical and clinical pharmacodynamic data on BBI-355 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024. Findings further support the development of BBI-355 as a differentiated single agent and combination treatment approach for oncogene amplified cancers.

BBI-825, a novel, selective RNR inhibitor targeting ecDNA assembly and repair

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Dosed the first patient in the STARMAP (Study Targeting Acquired Resistance: MAPK Amplifications) trial, a first-in-human, Phase 1/2 study of BBI-825 as a single agent and in combination with select targeted cancer therapies, for patients with locally advanced or metastatic cancer with resistance gene amplifications.

First Quarter 2024 Financial Highlights

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Cash Position: Cash, cash equivalents, and short-term investments totaled $104.9 million as of March 31, 2024. In addition, Boundless Bio completed its IPO in early April 2024 in which it sold 6,250,000 shares of its common stock for gross proceeds of $100.0 million. Boundless Bio expects its current cash position to fund operations into the second half of 2026 and through key clinical data milestones.

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R&D Expenses: Research and development (R&D) expenses were $13.1 million for the first quarter of 2024, compared to $9.5 million for the same period in 2023. The increase in R&D expenses was primarily due to a $1.8 million increase in the direct program costs for BBI-355, BBI-825, and other development programs, a $0.5 million increase in personnel-related costs resulting from an increase in headcount and salary increases, $0.3 million of additional stock-based compensation, and a $1.0 million increase in third-party services and other miscellaneous R&D costs.

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G&A Expenses: General and administrative (G&A) expenses were $3.8 million for the first quarter of 2024, compared to $2.6 million for the same period in 2023. The increase in G&A expenses was primarily due to a $0.3 million increase in personnel-related costs due to an increase in headcount and salary increases, $0.5 million of additional stock-based compensation, an increase in professional service fees of $0.2 million, and a $0.2 million increase in other G&A costs.

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Net Loss: Net loss totaled $15.4 million and $11.7 million for the first quarters of 2024 and 2023, respectively, with non-cash stock-based compensation expense of $1.3 million and $0.6 million for the first quarters of 2024 and 2023, respectively.

About BBI-355

Boundless Bio’s lead ecDNA-directed therapy (ecDTx), BBI-355, is a novel, oral, selective small molecule inhibitor of checkpoint kinase 1 (CHK1) being studied in the ongoing, first-in-human, Phase 1/2 POTENTIATE clinical trial (NCT05827614) in patients with oncogene amplified cancers. CHK1 is a master regulator of cells’ response to replication stress (RS). RS is elevated in ecDNA-enabled oncogene amplified cancer cells and, because of this, represents a key vulnerability of those cells. BBI-355 was designed to exploit the elevated RS in ecDNA-enabled oncogene amplified cancer cells by disrupting proper CHK1 function in regulating RS and thereby facilitating catastrophic RS to preferentially kill cancer cells relative to healthy cells.

About BBI-825

Boundless Bio’s second ecDTx, BBI-825, is a novel, oral, selective small molecule inhibitor of ribonucleotide reductase (RNR) being studied in the ongoing, first-in-human, Phase 1/2 STARMAP clinical trial (NCT06299761) in cancer patients with resistance gene amplifications. In preclinical studies, BBI-825 demonstrated low double digit nanomolar RNR inhibition and tumor growth inhibition, including regressions, in both the prevention and treatment of amplification-mediated resistance in mitogen-activated protein kinase (MAPK) pathway-activated tumors. RNR is the rate-limiting enzyme responsible for cellular de novo synthesis of deoxynucleotide triphosphates (dNTPs), the building blocks of DNA, and is essential to the assembly and repair of ecDNA. BBI-825 was shown to dysregulate ecDNA-reliant cancer cell dNTP pools, deplete ecDNA, and was synthetic lethal in multiple oncogene amplified preclinical cancer models.

On May 13, 2024 Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") reported that it will share new research from across its innovative portfolio of approved and investigational cancer therapies during the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting from May 31 – June 4 (Press release, Astellas, MAY 13, 2024, View Source [SID1234643133]). A total of 16 abstracts will be presented, including new data from pivotal trials supporting ongoing regulatory reviews. The volume of data being presented by Astellas reinforces its commitment to changing the course of cancer treatment through targeted therapies for hard-to-treat cancers like prostate, urothelial, and gastric/gastroesophageal junction (GEJ) cancers.

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Tadaaki Taniguchi, MD, PhD, Chief Medical Officer, Astellas
"The data at ASCO (Free ASCO Whitepaper) demonstrate the strength and breadth of our growing oncology portfolio and provide new insights into our transformative therapies for patients living with some of the most devastating cancers. Recent regulatory achievements mean our oncology medicines are reaching more patients than ever worldwide, and we are continuing to pursue novel targets and invest in research to improve overall survival and raise quality of life."

Highlights at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting include:

Further data from the Phase 3 EV-302 trial evaluating enfortumab vedotin in combination with pembrolizumab versus chemotherapy in previously untreated locally advanced or metastatic urothelial carcinoma (la/mUC), including data in cisplatin-eligible and cisplatin-ineligible populations. These results support the combination as a landmark advancement in the care of patients with la/mUC, regardless of cisplatin eligibility, and serve as the basis of ongoing regulatory reviews by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), Japan’s Ministry of Health, Labour and Welfare (MHLW), and the China National Medical Products Administration (NMPA).
Final overall survival (OS) results from the Phase 3 SPOTLIGHT study, evaluating the efficacy and safety of zolbetuximab – a first-in-class claudin (CLDN) 18.2-targeted monoclonal antibody approved by Japan’s MLHW and currently in review by multiple regulatory authorities for approval worldwide. In this abstract, zolbetuximab is evaluated in combination with mFOLFOX6 (a combination chemotherapy regimen that includes oxaliplatin, leucovorin, and fluorouracil) for the first-line treatment of patients with CLDN18.2 positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced unresectable or metastatic gastric or GEJ adenocarcinoma. These results underpin the strength of the zolbetuximab clinical data supporting the pursuit of regulatory approvals worldwide.
Two new post-hoc analyses of the Phase 3 EMBARK trial, which evaluated enzalutamide plus leuprolide, placebo plus leuprolide, and enzalutamide (single agent) in patients with nonmetastatic hormone- (or castration-) sensitive prostate cancer (nmHSPC or nmCSPC) with high-risk biochemical recurrence (BCR), including an oral presentation on the impact of treatment suspension on health-related quality of life and a poster presentation on sexual activity patient-reported outcomes.
Astellas Presentations at 2024 ASCO (Free ASCO Whitepaper) Annual Meeting

Enfortumab Vedotin

Presentation Title

Lead Author

Presentation Details

Impact of exposure on outcomes with enfortumab vedotin in patients with locally

advanced or metastatic urothelial cancer

D. Petrylak

Type: Oral

Presentation

Abstract

Number: 4503

Date: June 3, 2024

8:00-11:00 AM

CDT

Patient-reported outcomes (PROs) from a randomized, phase 3 trial of enfortumab

vedotin plus pembrolizumab (EV+P) versus platinum-based chemotherapy (PBC) in

previously untreated locally advanced or metastatic urothelial cancer (la/mUC)

S. Gupta

Type: Oral

Presentation

Abstract

Number: 4502

Date: June 3, 2024

8:00-11:00 AM

CDT

Enfortumab vedotin (EV) in triple-negative breast cancer (TNBC) and HR+/HER2-

breast cancer (BC) cohorts of EV-202

A. Giordano

Type: Oral

Presentation

Abstract

Number: 1005

Date: June 1, 2024

3:00-6:00 PM

CDT

Enfortumab vedotin (EV) with pembrolizumab (P) versus chemotherapy (chemo) in

previously untreated locally advanced or metastatic urothelial carcinoma (la/mUC): Analysis of

cisplatin (cis)-eligible population from EV-302/KEYNOTE-A39

J. Bedke

Type: Poster

Presentation

Abstract

Number: 4562

Date: June 2, 2024
9:00 AM-12:00

PM CDT

Enfortumab vedotin (EV) with pembrolizumab (P) versus chemotherapy (chemo) in

previously untreated locally advanced or metastatic urothelial carcinoma (la/mUC): Analysis of

the cisplatin (cis)-ineligible population from EV-302/KEYNOTE-A39

M. Van Der Heijden

Type: Poster

Presentation

Abstract

Number: 4563

Date: June 2, 2024

9:00 AM-12:00

PM CDT

Enfortumab vedotin (EV) in non-squamous and squamous non–small cell lung cancer

(NSCLC) cohorts of EV-202

K. Muro

Type: Poster

Presentation

Abstract

Number: 8585

Date: June 3,2024

1:30-4:30 PM

CDT

Enfortumab vedotin (EV) in previously treated gastric/esophageal cancers cohorts of

EV-202

K. Muro

Type: Poster

Presentation

Abstract

Number: 4046

Date: June 1, 2024

1:30-4:30 PM

CDT

Study EV-103: Neoadjuvant treatment with enfortumab vedotin monotherapy in

cisplatin-ineligible patients with muscle invasive bladder cancer (MIBC): 2-year event-

free survival and safety data for Cohort H

P. O’Donnell

Type: Poster

Presentation

Abstract

Number: 4564

Date: June 2, 2024

9:00 AM-12:00

PM CDT

Enfortumab vedotin and pembrolizumab as first-line treatment in recurrent or

metastatic head and neck squamous cell carcinoma: a cohort of the EV-202 trial

P. Swiecicki

Type: Poster

Presentation

Abstract

Number: TPS6116

Date: June 2, 2024

9:00 AM-12:00

PM CDT

Systematic Literature Review and Network Meta-Analysis of First-Line Therapies for

Locally Advanced/Metastatic Urothelial Carcinoma

L. Bloudek

Type: Online

publication

Abstract

Number: e16547

Real-world first-line treatment patterns and outcomes in patients with locally advanced

or metastatic urothelial carcinoma in the United States

R. Chen

Type: Online

Publication

Abstract

Number: e23287

Enzalutamide

Presentation Title

Lead Author

Presentation Details

EMBARK post-hoc analysis of impact of treatment suspension (TxS) on health-

related quality of life (HRQoL)

S. Freedland

Type: Oral Presentation

Abstract Number: 5005

Date: June 1, 2024

3:00-6:00PM CDT

EMBARK post hoc analysis of sexual activity (SA) patient-reported outcomes

(PROs) in patients (pts) who were sexually active or interested in sex at baseline (BL)

S. Freedland

Type: Poster Presentation

Abstract Number: 5084

Date: June 2, 2024

9:00 AM-12:00 PM CDT

Physicians use of first-line treatment intensification in metastatic castration-

sensitive prostate cancer (mCSPC): A discrete choice experiment

S. Loeb

Type: Poster Presentation

Abstract Number: 5087

Date: June 2, 2024

9:00 AM-12:00 PM CDT

Characteristics and treatment (Tx) patterns (TxP) of high-risk biochemically

recurrent (HR-BCR) non-metastatic castration-sensitive prostate cancer in the

real-world by race, age, and prostate-specific antigen (PSA) doubling time

(PSADT)

A. Morgans

Type: Online-Only Abstract

Abstract Number: e17071

Zolbetuximab

Presentation Title

Lead Author

Presentation Details

Final overall survival results from phase 3 SPOTLIGHT study evaluating

zolbetuximab + mFOLFOX6 as first-line (1L) treatment for patients (pts) with

claudin 18 isoform 2 (CLDN18.2)+, HER2−, locally advanced (LA) unresectable or

metastatic gastric or gastroesophageal junction (mG/GEJ) adenocarcinoma

K. Shitara

Type: Poster Presentation

Abstract Number: 4036

Date: June 1, 2024

1:30-4:30 PM CDT

About PADCEV and the Astellas, Pfizer and Merck Collaboration
Astellas and Pfizer have a clinical collaboration agreement with Merck to evaluate the combination of Astellas’ and Pfizer’s PADCEV (enfortumab vedotin-ejfv) and Merck’s KEYTRUDA (pembrolizumab) in patients with previously untreated metastatic urothelial cancer. KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Rahway, NJ, USA (known as MSD outside of the United States and Canada).

About XTANDI and the Pfizer/Astellas Collaboration
In October 2009, Medivation, Inc., which is now part of Pfizer (NYSE:PFE), and Astellas (TSE: 4503) entered into a commercial agreement to jointly develop and commercialize XTANDI (enzalutamide) in the United States, while Astellas has responsibility for manufacturing and all additional regulatory filings globally, as well as commercializing the product outside the United States. Pfizer receives alliance revenues as a share of U.S. profits and receives royalties on sales outside the U.S.

On May 13, 2024 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea" or the "Company"), a clinical stage biopharmaceutical company focused on precision oncology through synthetic lethality, reported that management will attend the 2024 RBC Capital Markets Global Healthcare Conference that will take place May 14-15, 2024 in New York, NY. Details are as follows (Press release, Aprea, MAY 13, 2024, View Source [SID1234643132]):

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2024 RBC Capital Markets Global Healthcare Conference
Date: May 14-15, 2024
Location: New York, NY

Management will be available for one-on-one meetings. To request a meeting, please contact your conference representative.

On May 13, 2024 Applied DNA Sciences, Inc. (NASDAQ: APDN) (Applied DNA), a leader in PCR-based DNA technologies, and the Institute of Hematology and Blood Transfusion (ÚHKT/IHBT) reported that an abstract relating to the development of a fully enzymatic, non-viral manufacturing workflow to enable the rapid and cost-effective production of clinical-grade (GMP) CAR T-cell therapies has been accepted for presentation at the prestigious European Hematology Association (EHA) (Free EHA Whitepaper) 2024 Hybrid Congress to be held in Madrid, Spain from June 13 – 16, 2024 (Press release, Applied DNA Sciences, MAY 13, 2024, View Source [SID1234643131]). Accepted abstracts will be available on the Congress’ website starting on May 14, 2024. This current study extends the successful results previously published on the virus-free preparation of CD19-specific CAR T-cells against refractory B cell malignancies utilizing Applied DNA’s Linea DNA.

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The current study was designed to address the high cost of conventional CAR T-cell therapies, which is primarily due to conventional manufacturing processes that utilize difficult-to-manufacture viral vectors. By generating CD123-specific CAR T-cells in a non-viral workflow via piggyBac transposons made from Linea DNA at substantial and cost-efficient yields with no risk of antibiotic-resistance gene transfer, we believe the study could offer a promising solution for the rapid manufacture of CAR T-cell therapies. IHBT is currently seeking SÚKL-EMA approval to initiate clinical trials using its Linea DNA-enabled CD123-specific CAR T-cells in patients with refractory acute myeloid leukemia.

Details of the oral presentation:
Abstract Title: "Linear DNA Platform For The Non-Viral Point-Of-Care Production Of Car-T Cells"
Abstract Number: P1722
Presenting Author: Dr. Pavel Otáhal

About the Linea DNA and Linea IVT Platforms
The Linea DNA platform is a completely cell-free DNA production platform founded on Applied DNA’s long-standing expertise in the large-scale enzymatic production of DNA. Capable of producing DNA in quantities ranging from milligrams to grams, the Linea DNA platform can produce high-fidelity DNA constructs ranging from 100bp to 20kb in size. The DNA produced via the Linea DNA platform is free of the adventitious DNA sequences found in other sources of DNA, is rapidly scalable, and provides for simple chemical modification of DNA constructs.

The Linea IVT platform combines DNA IVT templates manufacturing via the Linea DNA platform with a proprietary Linea RNAP to enable mRNA and sa-mRNA manufacturers to produce what Applied DNA believes to be better mRNA faster, with advantages over conventional mRNA production, including: 1) the elimination of plasmid DNA as a starting material; 2) the prevention or reduction of double-stranded DNA (dsRNA) contamination; and 3) simplified mRNA production workflows.

Learn more about the Linea DNA and Linea IVT platforms: LineaRx, an Applied DNA Sciences company