1stOncology™: Cancer Intelligence Service – Page 6044 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Gamida Cell Announces FDA Acceptance of Biologics License Application for Omidubicel with Priority Review

On August 1, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, reported that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s Biologics License Application (BLA) for omidubicel for the treatment of patients with blood cancers in need of an allogenic hematopoietic stem cell transplant (Press release, Gamida Cell, AUG 1, 2022, View Source [SID1234617212]). Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate with breakthrough and orphan drug designations.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

Schedule Your 30 min Free Demo!

The FDA granted Priority Review for the BLA and has set a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2023. The FDA grants Priority Review to product applications that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. At this time, the FDA has indicated that it is not planning an advisory committee meeting as part of the BLA review.

"The FDA’s acceptance of our BLA with Priority Review signifies a critical milestone in our mission to deliver a new stem cell therapy option for patients in need of a donor for an allogeneic stem cell transplant," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We are encouraged by the positive and sustained follow-up results from patients participating in the Phase 3 trial of omidubicel, including a positive overall survival trend one-year out from treatment. These results provide promising rationale that, if approved, omidubicel could become a treatment of choice for patients in need of an allo-HSCT transplant. We look forward to working with the FDA throughout the review process to bring omidubicel to patients as quickly as possible."

Upon FDA approval, omidubicel will be manufactured at the Gamida Cell owned manufacturing facility in Israel. This is a newly constructed, state-of-the-art, modular facility which allows for additional capacity to be added to address growing demand. Batches from this facility were used to support the BLA for omidubicel and the facility is currently manufacturing clinical batches.

The omidubicel BLA is supported by the statistically significant results from Gamida Cell’s pivotal Phase 3 study, the results of which were published in Blood, the official journal of the American Society of Hematology (ASH) (Free ASH Whitepaper). Results for the study’s primary endpoint, the median time to neutrophil engraftment in patients with hematologic malignancies undergoing allogeneic bone marrow transplant with omidubicel compared to standard umbilical cord blood (UCB), demonstrated a median time to neutrophil engraftment of 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p < 0.001). The secondary endpoints of this Phase 3 study were all achieved and were statistically significant. These secondary endpoints were platelet engraftment, the rate of infection, and days alive and out of hospital. Omidubicel was generally well tolerated in the Phase 3 study.

The full Blood manuscript is available here: View Source

In 2019, approximately 8,000 patients who were 12 years old and up with hematologic malignancies underwent an allogeneic stem cell transplant in the United States.1 Unfortunately, it is estimated that another 1,200 patients were eligible for transplant but could not find a donor source.2 If approved, omidubicel has the potential to improve outcomes for patients based on transplanter feedback and to potentially increase access for patients to get to transplant. If approved, omidubicel has the potential to treat approximately 2,000 – 2,500 patients each year in the U.S.

Conference Call Information

Gamida Cell will host a conference call today, August 1, 2022, at 8:00 a.m. ET to discuss this update. To access the conference call, please register here and please be advised to do so at least 10 minutes prior to joining the call. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.

About Omidubicel

Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.

Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit View Source

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

NanOlogy Publishes Results from a Phase 1/2 Clinical Trial of Large Surface Area Microparticle Docetaxel in High-Risk Non-Muscle Invasive Bladder Cancer

On August 1, 2022 NanOlogy LLC, a clinical-stage interventional oncology drug company, reported that results from a clinical trial of Large Surface Area Microparticle Docetaxel (LSAM-DTX) in High-Risk Non-Muscle Invasive Bladder Cancer (HR-NMIBC) have been published in The Journal of Urology (Press release, NanOlogy, AUG 1, 2022, View Source [SID1234617211]).

The research article entitled Phase 1/2 Trial Results of a Large Surface Area Microparticle Docetaxel for the Treatment of High-Risk Non-Muscle Invasive Bladder Cancer presents safety and response data from the multi-site study (NCT03636256). Clinical investigators included Max Kates, MD (Johns Hopkins Medical Institutions), Ahmed Mansour, MD (UT Health San Antonio), Donald Lamm, MD (BCG Oncology), and Neal Shore, MD (Carolina Urologic Research Institute).

The trial followed an open-label 3+3 dose-escalation design with enrollment expansion at the highest dose. After transurethral resection of the bladder tumor (TURBT), subjects received direct-injection LSAM-DTX into the resection site and intravesical LSAM-DTX, followed by six-week induction and three-week maintenance intravesical LSAM-DTX courses. The first-in-human trial was initially designed as a 7-month study with treatment limited to 4 months to establish safety but later amended to follow patients to 12 months despite the treatment limitation. Tumor recurrence was evaluated by cytology, cystoscopy, or biopsy. Pharmacokinetic analysis of blood and multiplex immunofluorescence analysis of tumor microenvironment were conducted pre/post treatment.

Nineteen subjects were enrolled, 14 with prior bacillus Calmette-Guérin (BCG) exposure, and 16 with one or more prior TURBTs. Direct-injection and intravesical LSAM-DTX were well-tolerated with minor recorded treatment-related local and systemic adverse events. Median recurrence free survival was 12.2 months in the high-dose and expansion cohorts and was significantly increased compared to the low dose cohorts at 5.4 months. Bladder biopsies show an increase in tumor microenvironment immunogenicity, including increases in adaptive (T cells) and innate (NK cells) effector cells. Notably, immune checkpoint receptor expression was increased across multiple cell types, suggesting LSAM-DTX in combination with immune checkpoint inhibitor therapy may provide additional benefit in treatment of HR-NMIBC.

Worldwide, an estimated 573,000 people were diagnosed with bladder cancer in 2020 and 81,000 people will be diagnosed with bladder cancer in the United States in 2022. About 40% of patients are at higher risk for disease progression at time of diagnosis (GLOBOCAN 2020; SEER). Recurrence rates are high in these patients with currently available treatments, potentially leading to disease progression and cystectomy. Bladder cancer has among the highest life-time treatment costs of all cancers and the negative impact to patient quality of life in patients facing cystectomy is severe. A significant need exists for better drug therapies to stave off disease progression.

"Bladder cancer at high risk for progression needs better treatment options," said Max Kates, MD, Director, Bladder Cancer Program and Associate Professor of Urology of the Brady Urological Institute at Johns Hopkins Medicine. "In this Phase 1/2 clinical trial, LSAM-DTX showed promising signs of preventing disease progression and interesting immunogenic effects with minimal adverse events. Further clinical research is warranted to confirm these findings."

NanOlogy is planning further clinical development of LSAM-DTX, which it believes has therapeutic potential in NMIBC and muscle invasive bladder cancer, which also showed promising results in a separate arm of the trial and will be reported once finalized.

In addition to LSAM-DTX, NanOlogy clinical programs have advanced tumor-directed investigational drugs in pancreas, lung, peritoneal, ovarian, prostate, and dermal cancers.

The NanOlogy therapeutic platform is based on a proprietary supercritical precipitation technology that converts active ingredients into stable large surface area microparticles (LSAMs) of pure drug optimized for tumor-directed therapy and continuous drug release to maximize drug delivered to the tumor and minimize systemic toxicity.

Taxane particles are covered by composition of matter patents issued in the US (US 9,814,685, US 10,507,195, US 10,993,927, and US 11,123,322), Canada, Europe, Japan, China, Hong Kong, South Korea, Australia, Indonesia, and Russia valid through June 2036. The composition patents form the foundation of an extensive intellectual property portfolio protecting NanOlogy investigational drugs, formulations, methods, and technology.

CohBar to Announce 2022 Second Quarter Financial Results and Provide Business Update on August 15, 2022

On August 1, 2022 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company leveraging the power of the mitochondria and the peptides encoded in its genome to develop potential breakthrough therapeutics targeting chronic and age-related diseases, reported that the company will release its 2022 second quarter financial results after the market closes on Monday, August 15, 2022 (Press release, CohBar, AUG 1, 2022, View Source [SID1234617210]). Management will host a conference call and webcast at 5:00 p.m. ET (2:00 p.m. PT) on the same day to provide an update on the company’s business.

A simultaneous webcast of the call will be accessible via the Investors section of the CohBar website at www.cohbar.com.
For individuals participating in the Investor Call or webcast, please call or login to the conference audio approximately 10 minutes prior to its start.

An audio replay of the call will be available beginning at 8:00 p.m. Eastern Time on August 15, 2022, through 11:59 p.m. Eastern Time on September 5, 2022. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID# 13730421. The audio recording will also be available at www.cohbar.com during the same period.

Agenus Announces First Patient Dosed in Phase 1 Study of AGEN1571 (anti-ILT2) in Advanced Solid Tumors

On August 1, 2022 Agenus Inc. (Nasdaq: AGEN), an immuno-oncology company with an extensive pipeline of therapeutics designed to activate the immune response to cancers and infections, reported that the first patient has been dosed in the Phase 1 study of AGEN1571 in advanced solid tumors (Press release, Agenus, AUG 1, 2022, View Source [SID1234617209]). The dose-escalation and expansion study will evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profiles of AGEN1571, a novel anti-ILT2 antibody designed to modulate tumor-associated macrophages, T, NK, and NKT cells. Participants will receive treatment with AGEN1571 as a single agent or in combination with botensilimab (Fc-enhanced anti-CTLA-4) and/or balstilimab (anti-PD-1).

"ILT2 is a major suppressor of anti-tumor immune responses and contributes to resistance to PD-1-directed therapies," said Steven O’Day, MD, Chief Medical Officer of Agenus. "We believe AGEN1571 has best-in-class potential to overcome this resistance and combining AGEN1571 with botensilimab and/or balstilimab may further enhance innate and adaptive anti-tumor immunity."

The initiation of the AGEN1571 study in patients with advanced solid tumors is based on preclinical data reported at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, demonstrating the ability of AGEN1571 to polarize macrophages to a pro-inflammatory phenotype and enhance the activation of CD8 T, NK, and NKT cells in the tumor microenvironment to overcome resistance to immune checkpoint blockade. These data demonstrate the superior potency and functional activity of AGEN1571 compared to the only known clinical-stage competitor and enhanced immune cell activation when combined with botensilimab or balstilimab.

The poster presentation on these data can be accessed in the investor section of the Agenus website at View Source

More information on the Phase I study (NCT05377528) is available at clinicaltrials.gov.

NantHealth to Report 2022 Second-Quarter Financial Results and Host Conference Call on Thursday, August 4

On August 1, 2022 NantHealth, Inc. (NASDAQ-GS: NH), a leading provider of enterprise solutions that help businesses transform complex data into actionable insights, reported that it will report financial results for its 2022 second quarter on Thursday, August 4, 2022, after market close (Press release, NantHealth, AUG 1, 2022, https://nanthealth.com/resources/press-releases/nanthealth-to-report-2022-second-quarter-financial-results-and-host-conference-call-on-thursday-august-4/ [SID1234617208]). NantHealth management will host a conference call that same day at 1:30 p.m. PT (4:30 p.m. ET) to review the company’s performance.

The conference call will be available to interested parties by dialing 800-771-6692 from the U.S. or Canada, or 212-231-2907 from international locations. The call will be broadcast via the Internet at www.nanthealth.com.