14 Studies Presented at AUA 2024 Show Decipher Tests’ Ability to Help Personalize Care for Prostate and Bladder Cancer Patients and Advance Disease Understanding

On May 6, 2024 Veracyte, Inc. (Nasdaq: VCYT), a leading cancer diagnostics company, reported that data from 14 presentations at AUA 2024, the annual meeting of the American Urological Association, show that the Decipher Prostate and Decipher Bladder Genomic Classifiers provide better prognostic information for patients with prostate and bladder cancer, compared to standard approaches (Press release, Veracyte, MAY 6, 2024, View Source [SID1234642688]). They also show that the research-use-only Decipher GRID (Genomic Resource for Intelligent Discovery) tool is helping to advance scientific understanding of these diseases. The findings were presented during the conference taking place May 3-6 in San Antonio.

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"The large amount of data presented at AUA 2024 reinforces Veracyte’s commitment to building rigorous evidence that demonstrates our tests’ performance and clinical utility," said Elai Davicioni, Ph.D., Veracyte’s medical director for Urology. "Further, our whole-transcriptome approach to testing provides an incredible amount of data that we are pleased to share with the research community through Decipher GRID to help advance understanding of urologic cancers and ultimately improve patient outcomes."

Studies showing the Decipher Prostate test’s ability to better inform prostate cancer treatment include:

Poster MP41-09: Genomic Signatures Associated with Adverse Pathologic Features at Radical Prostatectomy Among Active Surveillance Eligible Men. Presented by Eric Li, M.D., Northwestern University.

Summary: The Decipher Prostate Genomic Classifier is associated with adverse pathology in patients eligible for Active Surveillance (AS) who were treated with radical prostatectomy (RP). The findings suggest the Decipher test may be able to identify patients at increased risk of harboring higher grade and non-organ confined disease who may not be ideal candidates for AS.

"Despite having similar clinical features at diagnosis, our study suggests that AS-eligible prostate cancer patients have a spectrum of risk for occult adverse pathology that can be elucidated at the level of gene expression," said Ashley Ross, M.D., Ph.D., clinical director for the Polsky Urological Oncology Center at Northwestern University and principal investigator on the study. "Our results suggest that use of the Decipher Prostate test may help clinicians better stratify risk among patients eligible for AS, which may ultimately help reduce under- and over-treatment."

Poster MP41-14: High Decipher scores define the subgroup most at risk of metastatic progression among patients with lower-grade tumors classified as NCCN high-risk based on elevated prostate-specific antigen level alone. Presented by David Han, M.D., Columbia University Irving Medical Center.

Summary: Despite harboring favorable, lower grade (Grade Group 1 or 2) organ-confined disease, patients with a prostate-specific antigen (PSA) level of >20 ng/mL are currently classified by practice guidelines as "high risk". In a cohort of 453 patients with long-term outcomes, the Decipher Prostate test score better predicted the development of distant metastases than PSA. These results provide further evidence that a higher Decipher score is a more accurate risk factor than PSA in patients with otherwise favorable disease.

Poster MP49-09: Decipher Predicts Clinically Significant Upgrading on Final Radical Prostatectomy Pathology. Presented by John Sheng, M.D., Washington University School of Medicine in St. Louis.

Summary: A large registry (n=760) from prospective clinical use of Decipher Prostate and multiparametric prostate MRI (mpMRI) at diagnosis was examined to determine factors significantly associated with high-grade disease at radical prostatectomy (RP). In the subset with low- or intermediate-grade prostate cancer at initial biopsy only Decipher and grade group, but not mpMRI PIRADS or baseline PSA, predicted high-risk disease at final pathology after RP.

Podium Presentation PD42-03: Understanding Population-Wide Genomic Risk Distribution and Integrating Clinical-Genomic Risk for Prognostication in Prostate Cancer. Presented by Udit Singhal, M.D., University of Michigan.

Summary: The International Staging Collaboration for Prostate Cancer (STAR-CAP) is a highly validated prognostic clinical risk staging system. In an analysis of 52,565 patients from the state-wide Michigan Urological Surgery Improvement Collaborative (MUSIC) and the nation-wide Decipher GRID database, researchers found wide variation of Decipher Prostate Genomic Classifier scores within STAR-CAP risk groups. Overall, they found Decipher testing augmented by at least one STAR-CAP stage both upstaging for about 25% and down-staging for nearly 50% of cases, suggesting that integration of genomic with advanced clinicopathologic staging systems may lead to further improvements to risk stratification across the clinical spectrum of localized disease.
The following study demonstrates the Decipher Bladder test’s utility in informing treatment decisions for patients with bladder cancer:

Poster MP15-07: Molecular subtyping for predicting non-organ confined disease and survival outcomes after radical cystectomy in clinical high-grade T1 and T2 bladder cancer patients. Presented by Yair Lotan, M.D., UT Southwestern Medical Center.

Summary: Clinical staging in bladder cancer commonly underestimates the true disease stage as many patients are upstaged to non-organ confined (NOC) disease (pT3+ and/or N+) at radical cystectomy (RC). This multi-center study of 200 patients validates prior findings, further demonstrating the utility of the Decipher Bladder Genomic Subtyping Classifier (GSC) for predicting upstaging and outcomes in a cohort of patients with clinical T1 or T2 bladder cancer treated with radical cystectomy but without neoadjuvant therapy.
Additional studies used the RUO Decipher GRID tool to explore prostate and bladder cancer topics that include: which patients are likely to benefit from specific therapies, racial differences in disease biology, and molecular pathway alterations following treatment.

"The depth and breadth of Decipher-focused data at AUA 2024 underscores the value of our novel Veracyte Diagnostics Platform, which begins with delivering high-performing tests using a comprehensive, whole-transcriptome approach. This fosters additional research, which in turn supports further innovation to help more patients," said Phillip Febbo, M.D., Veracyte’s chief scientific officer and chief medical officer.

About Decipher Prostate

The Decipher Prostate Genomic Classifier is a 22-gene test, developed using RNA whole-transcriptome analysis and machine learning, that helps inform treatment decisions for patients with prostate cancer. The test is performed on biopsy or surgically resected samples and provides an accurate risk of developing metastasis with standard treatment. Armed with this information, the physician can better personalize their patients’ care and may recommend less-intensive options for those at lower risk or earlier, more-intensive treatment for those at higher risk of metastasis. The Decipher Prostate test has been validated in more than 80 published studies involving more than 100,000 patients. More information about the Decipher Prostate test can be found here.

About Decipher Bladder

The Decipher Bladder Genomic Classifier is a 219-gene test, developed using RNA whole-transcriptome analysis and machine learning, that is designed for use in patients following bladder cancer diagnosis who face questions regarding treatment intensity. The test classifies bladder tumors into five molecular subtypes, each having distinct tumor biology and potential clinical implications. This information can help physicians and their patients better understand the degree of benefit that would likely be gained from neoadjuvant chemotherapy and/or the likelihood of harboring non-organ-confined disease at time of surgery, respectively. More information about the Decipher Bladder test can be found here.

About Decipher GRID

The Decipher GRID database includes more than 200,000 whole-transcriptome profiles from patients with urologic cancers and is used by Veracyte and its partners to contribute to continued research and help advance understanding of prostate and other urologic cancers. GRID-derived information is available on a Research Use Only basis. More information about Decipher GRID can be found here.

TC BioPharm Announces Execution of Second Non-Binding Letter of Intent for Acquisition, Targeting Innovative CAR-T Therapies

On May 6, 2024 TC BioPharm (Holdings) PLC ("TC BioPharm" or the "Company") (NASDAQ: TCBP) a clinical stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer and other indications, reported the execution of a non-binding letter of intent to acquire a privately-held biotechnology company pursuing the development of innovative Chimeric Antigen Receptor T-cell (CAR-T) therapies for the treatment of refractory cancers and solid tumors (Press release, TC Biopharm, MAY 6, 2024, View Source [SID1234642687]).

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This agreement is the second such agreement in as many months shows TCBP’s commitment to its M&A strategy aimed at expanding its therapeutic platform.

CAR T-cell therapy modifies a patient’s own immune cells to provide a heightened ability to identify and eliminate cancer cells.

The target acquisition has developed a number of proprietary approaches to re-engineer CARs to better target solid tumors. The Company has generated pre-clinical proof of concept data that demonstrates the therapeutic potential of its lead CAR-T candidate for a range of solid tumors including colorectal, pancreatic, mesothelioma, ovarian and breast cancer. In addition, the target acquisition is also developing a novel, allogeneic CAR-T for the treatment of autoimmune diseases.

There can be no assurance that a definitive agreement will be executed or that the proposed transaction will be consummated on the terms or timeframe currently contemplated. Upon execution of the definitive agreements, the completion of the transaction will be subject to, among other matters, satisfaction of the conditions negotiated therein, the Company having secured adequate financing, and receipt of all third party (including governmental) approvals, licenses, consents, and clearances, as and when applicable.

"Management is extremely pleased to announce this second potential acquisiton which will provide strong advantages to our current therapeutic platform as well as expand our efforts into autoimmune disease," said Mr. Bryan Kobel, CEO of TC BioPharm. "There are substantial synergies between the two companies with extensive benefits to the business combination including CAR development expertise as well as gamma delta and alpha beta T-cells. Our existing expertise in gamma delta T-cells as well as transitioning processes and therapeutics from autologous to allogeneic will be impactful in advancing the solid tumor assets of the acquisition, the acquired company brings strong CAR engineering expertise to help us further develop our co-stimulatory CAR as well as a strong clinical team. We believe that the combination of these two companies provides a well balanced platform for investors and patients in our pursuit of creating next-generation cell therapies for the treatment of multiple indications. This represents not only multiple shots on goal, but an expanded asset base in several immune responder cells for the treatment of a wide range of disease with an extensive patent portfolio. As such, 2024 and 2025 could now have inflections points for data in several pipeline assets in a variety of indications. I am proud of our ability to navigate a difficult capital market environment and identify complimentary resources that will continue to enhance the overall value of our company and look forward to working with our expanded team."

Sana Biotechnology to Present at May and June 2024 Investor Conferences

On May 6, 2024 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported that it will webcast its presentations at three investor conferences in May and June. The presentations will feature a business overview and update.

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Sana will present at the Citizens JMP Securities Life Sciences Conference at 11:30 a.m. ET on Monday, May 13, 2024.
Sana will present at the BofA Securities 2024 Healthcare Conference at 3:00 p.m. PT on Tuesday, May 14, 2024.
Sana will present at the Goldman Sachs 45th Annual Global Healthcare Conference at 2:40 p.m. ET on Monday, June 10, 2024.

The webcasts will be accessible on the Investor Relations page of Sana’s website at View Source A replay of each presentation will be available at the same location for 30 days following the conference.

Rocket Pharmaceuticals Reports First Quarter 2024 Financial Results and Highlights Recent Progress

On May 6, 2024 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported financial and recent operational results for the quarter ending March 31, 2024 (Press release, Rocket Pharmaceuticals, MAY 6, 2024, View Source [SID1234642685]).

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"Rocket has had a strong start to 2024. This quarter has been marked by meaningful progress across all our clinical programs, most notably with the EMA’s review of RP-L102 for Fanconi Anemia, commercial preparations for the potential approval of KRESLADI for severe LAD-I, and ongoing enrollment in the Phase 2, pivotal study of RP-A501 for Danon Disease and the Phase 1 study of RP-A601 for PKP2-ACM," said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. "As we build on the advancements made this quarter, we remain focused on execution for patients with rare and devastating diseases with limited treatment options."

Recent Pipeline and Operational Updates


Advanced Fanconi Anemia (FA) program through key regulatory milestones.

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In April, Rocket announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102 for the treatment of FA. MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial.

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The Company remains on track to submit the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first half of 2024.


KRESLADI on track for June 30, 2024, PDUFA date.

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Rocket continues to ramp up enabling activities to support the launch of its lentiviral (LV) vector portfolio beginning with KRESLADI for severe Leukocyte Adhesion Deficiency-I (LAD-I). Qualified Treatment Center initiation, disease education, payer engagement, and field team build-out are all underway.


Bolstered finance, investor relations, and corporate communications expertise of company leadership to support evolution towards commercial stage.

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Rocket appointed Aaron Ondrey as Chief Financial Officer (CFO). Mr. Ondrey brings over 20 years of experience leading commercial-stage financial management, strategic planning, and capital allocation. Mr. Ondrey was previously the CFO at Mirati Therapeutics and has held multiple senior finance leadership positions at Arena Pharmaceuticals, Alexion Pharmaceuticals, and Regeneron Pharmaceuticals.

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In addition, Meg Dodge was appointed Vice President to lead Investor Relations & Corporate Communications. Ms. Dodge is experienced with engaging stakeholders across investors, media, and other communities in the biotech and financial sectors. Prior to joining Rocket, Ms. Dodge was Head of Investor Relations and Corporate Communications at Krystal Biotech.


Celebrated annual Rare Disease Day with multi-faceted awareness campaign.

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On February 29, 2024, Rocket hosted its annual Rare Disease Day recognition program highlighting the theme, "Leap into Action for Rare." Several hundred attendees gathered at the Liberty Science Center and virtually to hear inspirational stories from the community. Rocket also continued its Light Up for Rare initiative in collaboration with global partners to light up buildings and landmarks in Rare Disease Day colors, including the Empire State Building.

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Rocket remains highly committed to supporting the rare disease community through patient-focused events, education and advancing science to bring potential treatments to patients with unmet needs.


Milestones in 2024 are on track across its pipeline of gene therapies for rare and devastating diseases.

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Rocket continues to advance three disclosed programs from its adeno-associated virus (AAV) cardiovascular portfolio, including:


Phase 2 pivotal study of RP-A501 for Danon Disease,


Phase 1 study of RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM), and


IND-enabling studies for BAG3-associated dilated cardiomyopathy (DCM)

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In its late-stage LV portfolio, Rocket is working towards initiation of the Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD).


Data from Rocket’s LV hematology portfolio to be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting.

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Updated data across Rocket’s LV hematology programs will be highlighted as oral presentations at the ASGCT (Free ASGCT Whitepaper) 27th Annual Meeting taking place May 7-11, 2024, in Baltimore, MD

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Incremental updates include longer-term data demonstrating the safety and efficacy of Rocket’s Phase 1/2 pivotal studies of KRESLADI for severe LAD-I and RP-L102 for FA, in addition to the Phase 1 study of RP-L301 for PKD.

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Details for oral presentations are as follows:

Title: Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301
Session: Clinical Trials Spotlight Symposium
Presenter: Julián Sevilla, M.D., Ph.D., Clinical Investigator, Hematología y Hemoterapia, Hematología y Oncología Pediátricas, Hospital Infantil Universitario Niño Jesús
Presentation date and time: Wednesday, May 8, 2024, 8:00 a.m. – 8:15 a.m. ET
Location: Ballroom 1
Presentation number: 4

Title: Lentiviral-Mediated Gene Therapy (RP-L102) for Fanconi Anemia [Group A] is Associated with Polyclonal Integration Patterns in the Absence of Conditioning
Session: Cell Therapy and Cell-Based Gene Therapy Trials
Presenter: Agnieszka Czechowicz, M.D., Ph.D., Center for Definitive and Curative Medicine, Department of Pediatrics, Division of Hematology/ Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Lucile Packard Children’s Hospital Stanford
Presentation date and time: Friday, May 10, 2024, 2:45 p.m. – 3:00 p.m. ET
Location: Ballroom 1
Presentation number: 245

Title: Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Favorable Safety Profile
Session: Cell Therapy and Cell-Based Gene Therapy Trials
Presenter: Donald B. Kohn, M.D., Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy Program, University of California, Los Angeles
Presentation date and time: Friday, May 10, 2024, 3:00 p.m. – 3:15 p.m. ET
Location: Ballroom 1
Presentation number: 246

Upcoming Investor Conference


Bank of America Global Healthcare Conference 2024: May 16, 2024

First Quarter Financial Results


Cash position. Cash, cash equivalents and investments as of March 31, 2024, were $330.3 million.


R&D expenses. Research and development expenses were $45.2 million for the three months ended March 31, 2024, compared to $46.4 million for the three months ended March 31, 2023. The decrease in R&D expenses was primarily driven by decreases in manufacturing and development costs and direct materials of $5.8 million. Decreases were partially offset by increases in costs for compensation and benefits expense of $1.4 million due to increased R&D headcount, professional fees of $1.1 million, laboratory supplies of $0.9 million, non-cash stock compensation expense of $0.8 million, and clinical trial costs of $0.6 million.


G&A expenses. General and administrative expenses were $22.1 million for the three months ended March 31, 2024, compared to $15.8 million for the three months ended March 31, 2023. The increase in G&A expenses was primarily driven by increased commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $3.3 million, legal expenses of $1.5 million, and non-cash stock compensation expense of $0.5 million.


Net loss. Net loss was $62.1 million or $0.66 per share (basic and diluted) for the three months ended March 31, 2024, compared to $58.3 million or $0.73 (basic and diluted) for the three months ended March 31, 2023.


Shares outstanding. 90,646,590 shares of common stock were outstanding as of March 31, 2024.

Financial Guidance


Cash position. As of March 31, 2024, Rocket had cash, cash equivalents and investments of $330.3 million. Rocket expects such resources will be sufficient to fund its operations into 2026, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs.

Ratio Therapeutics Announces the Appointment of Marc Becker as Chief Financial Officer and Chief Operating Officer

On May 6, 2024 Ratio Therapeutics Inc. (Ratio), an emerging pharmaceutical company employing innovative technologies to develop best-in-class radiopharmaceuticals for cancer treatment and monitoring, reported the appointment of Marc Becker as the company’s Chief Financial Officer and Chief Operating Officer (Press release, Ratio Therapeutics, MAY 6, 2024, View Source [SID1234642684]). This appointment will reinforce Ratio’s financial and operational leadership by bringing significant experience in biotech and pharma to guide growth strategies and ensure continued success.

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"We are honored to welcome Marc to the Ratio team," commented Dr. Jack Hoppin, Chairman and Chief Executive Officer of Ratio. "Building on the recent expansion of our leadership team and board, the appointment of our new CFO/COO is essential as we navigate the dynamic and rapidly evolving radiopharmaceutical space, which is currently experiencing significant growth and activity. Marc’s experience in financial management and strategic planning makes him a valuable asset as we continue to grow and develop our pipeline of radiopharmaceuticals."

"It’s a privilege to become part of Ratio’s team during this critical juncture, especially with the escalating momentum surrounding the development of targeted radiopharmaceuticals within our industry," said Marc Becker, Chief Financial Officer and Chief Operating Officer of Ratio. "I look forward to applying my financial expertise and knowledge of the biopharmaceutical sector to help shape Ratio’s strategic direction and ensure our growth is robust and sustainable."

Mr. Becker brings 20+ years of experience in biotech and pharma to his role as CFO/COO at Ratio. With a proven track record of success in strategic planning, finance transactions, and operational scaling, he will play a crucial role in driving forward the company’s vision and business objectives. Prior to his most recent position as CFO, Growth Companies, at Flagship, Mr. Becker served as CFO for Concert Pharmaceuticals, Inc., where he raised several rounds of financing and led a successful M&A process culminating in a sale to Sun Pharma in March 2023. He also previously served as CFO at CRISPR Therapeutics, where he led a successful IPO. Additionally, he held the position of CFO and Senior Vice President at rEVO Biologics and served as Vice President at Genzyme Corporation. Mr. Becker holds a BS in Accounting from the University of Massachusetts, an MBA from Babson College and was licensed as a CPA.