On January 16, 2024 Myriad genetics presented its corporate presentation (Presentation, Myriad Genetics, JAN 16, 2024, View Source [SID1234639262]).

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On January 16, 2024 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported new data from Part A of the DeFianCe study, a Phase 2 study evaluating DKN-01, Leap’s anti-Dickkopf-1 (DKK1) antibody, in combination with standard of care bevacizumab and chemotherapy in second-line patients with advanced colorectal cancer (CRC), to be presented at the upcoming 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium taking place in San Francisco, CA and virtually on January 18-20, 2024 (Press release, Leap Therapeutics, JAN 16, 2024, View Source [SID1234639261]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Data from Part A of the DeFianCe study demonstrates that the addition of DKN-01 to bevacizumab and chemotherapy can generate clinically meaningful response rates and durable tumor reductions with a favorable safety profile in second-line CRC patients, particularly those with rectal or rectosigmoid tumors," said Meredith Pelster, MD, Assistant Director of Gastrointestinal Research at Sarah Cannon Research Institute and a study investigator. "We are very interested in utilizing DKN-01 to modulate the Wnt pathway, which is active in a high percentage of CRC patients, particularly left-sided tumors, to overcome resistance to chemotherapy, and to decrease angiogenesis in order to enhance the activity of the standard of care bevacizumab plus chemotherapy regimens. These results provide a strong foundation for the randomized controlled Part B of this study, which is enrolling extremely well and expected to complete enrollment mid-year."

"In this heterogenous second-line population with several unfavorable characteristics, the DKN-01 plus bevacizumab and chemotherapy Part A ORR of 30%, with a disease control rate of over 90%, and enhanced activity in left-sided tumors and rectal tumors, with a 46% ORR and 9.4 month preliminary PFS, represent an encouraging efficacy signal," said Zev Wainberg, MD, Professor of Medicine and Co-Director of the GI Oncology Program at UCLA. "CRC is a heterogenous disease where the selection of therapy and expected outcomes vary based on whether the tumor is on the left or right side, the presence or absence of genetic mutations, microsatellite instability, and prior therapy. Physicians want to be able to select a second-line therapy combination based on the patient’s personalized tumor characteristics, which is what we hope to achieve by adding DKN-01 and understanding the subgroups with the greatest clinical benefit."

Leap will host a conference call on January 23, 2024 at 8:30 a.m. Eastern Time in which Dr. Pelster and Dr. Wainberg will further discuss the new data from the DeFianCe study.

Key Findings:

· As of the December 6, 2023 data cutoff, 33 patients enrolled in Part A of the DeFianCe study
· Across all evaluable patients with second-line microsatellite stable CRC (n=27):
o Objective response rate (ORR) was 30% and disease control rate (DCR) was 93%, including 8 partial responses (PR) and 17 patients with a best response of stable disease (SD)
o Median progression-free survival (PFS) was 6.3 months
o 9 patients remain on therapy beyond 8.5 months
· Analysis revealed a breadth of clinical activity across additional subgroups, including patients with left-sided tumors (n=25)
o 33% ORR and 100% DCR in response-evaluable population (7 PRs, 14 SDs)
o Preliminary median PFS of 8.6 months (9 patients continuing on therapy within subgroup)
· Patients with rectal/rectosigmoid carcinomas (n=15) represent an important subpopulation:
o 46% ORR and 100% DCR in response-evaluable population (6 PRs, 7 SDs)
o Preliminary median PFS of 9.4 months (6 patients continuing on therapy within subgroup)
o Higher baseline plasma DKK1 levels correlated with improved responses
· DKN-01 plus bevacizumab and chemotherapy was well-tolerated, with a majority of DKN-01 related events being low grade (Grade 1/2)
· Randomized controlled Part B of the study is underway with 54 patients currently enrolled

Conference Call:

Leap’s management team, together with Dr. Pelster and Dr. Wainberg, will host a conference call on Tuesday, January 23, 2024 at 8:30 a.m. Eastern Time to further discuss the data. The conference call will be broadcast live in listen-only mode and can be accessed via the website URL: View Source A replay of the event will also be available for a limited time on the Investors page of the Company’s website at View Source

About the DeFianCe Study

The DeFianCe study (NCT05480306) is a Phase 2, open-label, global study of DKN-01 in combination with standard of care bevacizumab and chemotherapy in patients with advanced CRC who have received one prior systemic therapy for advanced disease. The Part A cohort enrolled 33 patients, including significant numbers of patients who had early progression on first-line therapy, previous exposure to bevacizumab, tumors with Ras mutations, or liver metastases. The study has expanded into a 130-patient Part B randomized controlled trial. The primary objective of the study is progression free survival. Secondary objectives include overall response rate, duration of response, and overall survival.

On January 16, 2024 Instil Bio, Inc. ("Instil" or the "Company") (NASDAQ: TIL), a clinical-stage biopharmaceutical company focused on developing tumor infiltrating lymphocyte, or TIL, therapies for the treatment of patients with cancer, reported a strategic update (Press release, Instil Bio, JAN 16, 2024, View Source [SID1234639260]).

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Instil has entered into an agreement with a collaborator that has a successful track record of manufacturing and dosing patients with cell therapies to conduct preclinical manufacturing feasibility studies in the ITIL-306 program. The feasibility studies have been initiated, and if the feasibility studies are successful, Instil’s collaborator may open an investigator-initiated clinical trial (IIT) to enroll patients with non-small cell lung cancer (NSCLC) in China. In the event the IIT generates compelling proof-of-concept clinical data in 2024, Instil may explore options for a potential transition of ITIL-306 to a US-based CDMO for manufacturing and clinical development primarily at US clinical trial sites.

With the objective of saving costs and improving time efficiency, the Company is announcing the closure of its UK manufacturing and clinical operations, thereby reducing its UK workforce which is expected to be substantially completed by the first half of 2024. Instil plans to retain key process development, research, and related personnel to advance early-stage pipeline development of CoStAR and other novel TIL technologies, and to support the company’s collaboration.

On January 16, 2024 iBio, Inc. (NYSEA:IBIO) ("iBio" or the "Company"), an AI-driven innovator of precision antibody immunotherapies, reported that Matthew P. Greving Ph.D., its Vice President and Head of Platform Technologies and Machine Learning, will give a podium presentation titled "Enhancing Bispecific T-Cell Engager Discovery, Potency, Safety, and Developability with Machine Learning and Mammalian Display" at the 23rd annual PepTalk Conference Jan. 16-19 in San Diego, California (Press release, iBioPharma, JAN 16, 2024, View Source [SID1234639259]). He will also moderate a BuzZ talk on the topic as part of the conference’s "Developability of Bispecifics" program.

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During the podium presentation, Dr. Greving will provide an overview of how iBio’s technology stack – including epitope engineering, human-diversity antibody libraries, EngageTx for bispecific optimization, and ShieldTx for antibody masking – potentially overcomes challenges in the discovery of bispecific T-Cell Engagers ("TCE"), a promising area of research in immunotherapies for cancer. He will present data demonstrating how iBio’s machine learning (ML)-driven epitope steering and mammalian-display antibody libraries efficiently discover diverse TCE arms tuned for potency, toxicity, developability, and cyno cross-reactivity. The presentation will take place Tuesday, Jan. 16 at 5:15 p.m. Pacific Time.

Before the presentation, Dr. Greving will moderate an informal, open discussion diving into topics including, but not limited to, improving discovery and productivity for bispecific TCE’s immune cell arm, advances in discovering difficult tumor-antigen arm targets and epitopes, enhancing T-cell engager safety with machine-learning derived mammalian display libraries, and large-scale bispecific activity and developability screening with mammalian display. The BuzZ session will occur on Tuesday, Jan. 16, at 3:15 p.m. Pacific Time.

iBio’s Drug Discovery Platform is a precision-driven and deeply integrated technology stack that aims to efficiently and consistently deliver antibody candidates against challenging targets and move them into the clinic faster. The Company uses its technology to advance candidates with partners and collaborators, and for its own proprietary pipeline.

On January 16, 2024 Ferring Pharmaceuticals reported that ADSTILADRIN (nadofaragene firadenovec-vncg) is now fully available across the U.S. for healthcare providers to prescribe for their adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (Press release, Ferring, JAN 16, 2024, View Source [SID1234639258]). Approved by the U.S. Food & Drug Administration (FDA) in December 2022, ADSTILADRIN is the first and only FDA-approved intravesical gene therapy for adults with NMIBC who no longer respond to standard therapy.

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"From the day we received FDA approval, Ferring has been committed to making
ADSTILADRIN available to every appropriate high-risk NMIBC patient quickly and responsibly, working collaboratively with the bladder cancer community to inform our approach," said Bipin Dalmia, Senior Vice President, Global Head, Uro-Oncology Franchise at Ferring Pharmaceuticals. "With our significant manufacturing investments, we have achieved full product supply ahead of schedule. We are therefore ending our ADSTILADRIN Early Experience Program and look forward to bringing this novel therapy to every patient who needs it"

In September 2023, Ferring initiated the ADSTILADRIN Early Experience Program to a mix of clinical trial sites that participated in the ADSTILADRIN Phase 3 study and community clinics with the highest number of appropriate patients with NMIBC. This temporary program represented Ferring’s commitment to treat as many patients as possible in the short term while ensuring every patient who started on ADSTILADRIN had the ability to continue therapy for the duration of their treatment. Now that full product supply is available ahead of schedule, Ferring can end the temporary ADSTILADRIN Early Experience Program and dramatically increase patient access.

"ADSTILADRIN represents an effective alternative therapy for patients with NMIBC who, historically, had very few options once they no longer responded to standard BCG therapy," said Andrea Maddox-Smith, CEO of the Bladder Cancer Advocacy Network, BCAN. "Increasing access to this innovative therapy offers the potential of what patients need most – safe, effective treatment options that bring hope."

Ferring also initiated a non-interventional study, known as the "ADSTILADRIN in BLadder
CancEr" (ABLE-41) U.S. Real World Evidence (RWE) Study (NCT06026332). This ongoing
study is exploring early utilization, experiences, and outcomes of ADSTILADRIN in the routine care setting.

About ADSTILADRIN
ADSTILADRIN (nadofaragene firadenovec-vncg) is the first and only FDA-approved
intravesical gene-therapy for the treatment of adult patients with high-risk Bacillus CalmetteGuérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high quantities of interferon alfa-2b protein, a naturally-occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer.

ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with highgrade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on
clinicaltrials.gov: NCT02773849).1

About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.2
Bladder cancer is the sixth most common cancer in the U.S., and it is estimated that there were approximately 81,180 new cases of bladder cancer in the U.S. in 2022,3
75% of which present as NMIBC.4 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard of care. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease.3 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder).5

INDICATION
ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the
treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive nonmuscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.

WARNINGS AND PRECAUTIONS:

Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.

Please click to see the full Prescribing Information.