On January 10, 2025 Naveris, Inc., the leader in precision oncology diagnostics for viral-induced cancers, reported the expanded commercial availability of the NavDx test for Molecular Residual Disease (MRD) detection in anal squamous cell carcinoma (ASCC) patients (Press release, Naveris, JAN 10, 2025, View Source [SID1234649613]). NavDx, Naveris’ proprietary flagship blood test, is the first and only clinically validated circulating tumor TTMV-HPV DNA blood test that provides a non-invasive and precise method that can detect MRD before there is clinical or radiographic evidence of cancer recurrence.

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The commercial expansion coincides with the publication of clinical validity and utility data in the journal Cancers1 that demonstrates the NavDx test’s ability to accurately and reliably detect MRD in HPV-positive anal cancer patients in a multi-center, real-world cohort.

Dr. Barry M. Berger, Chief Medical Officer of Naveris, commented: "With an impressive per-test positive predictive value (PPV) of 98% and a negative predictive value (NPV) of 95%, the NavDx test demonstrates exceptional clinical value in detecting and ruling out disease recurrence in ASCC patients. This offers real utility for physicians, allowing them to accurately assess whether a patient has residual disease and implementing the most appropriate course of care without unnecessary delays. This expanded use of NavDx represents a critical step forward in advancing both precision medicine and the overall management of HPV-related anal cancer."

This new data further reinforces the clinical value of TTMV-HPV DNA testing in HPV-positive cancers, as supported by more than 30 peer reviewed publications validating use of the NavDx test in head and neck cancers.

The expansion of the NavDx test in HPV-positive anal cancer patients also reflects Naveris’ commitment to increasing access to high-quality care. As a blood test, the NavDx test offers a simpler, non-invasive, and more accessible compliment to traditional institution-based surveillance methods and is anticipated to increase healthcare access for those who face challenges in receiving timely, effective cancer care.

"Certain populations, notably those living with HIV and of lower socioeconomic status, are much more likely to develop anal cancer. These populations often face barriers to accessing care under current recurrence surveillance protocols," noted Alice L. Pomponio, Managing Director of the American Cancer Society’s BrightEdge impact investing arm and a Naveris Board Observer. "By bringing the NavDx test to clinic for anal cancer MRD testing, Naveris is addressing an urgent need for early cancer detection solutions and providing health equity for individuals that face challenges to accessing care. Our shared mission of impact investing, leveraging innovation to improve care for underserved populations, can be achieved through a test that will improve clinical care for all patients."

On January 10, 2025 Pfizer Inc. (NYSE: PFE) reported positive topline results from its pivotal Phase 3 CREST trial evaluating sasanlimab, an investigational anti-PD-1 monoclonal antibody (mAb), in combination with Bacillus Calmette-Guérin (BCG) as induction therapy with or without maintenance in patients with BCG-naïve, high-risk non-muscle invasive bladder cancer (NMIBC) (Press release, Seagen, JAN 10, 2025, View Source [SID1234649598]). The study met its primary endpoint of event-free survival (EFS) by investigator assessment, demonstrating a clinically meaningful and statistically significant improvement with sasanlimab in combination with BCG (induction and maintenance) as compared to BCG alone (induction and maintenance).

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"Patients with BCG-naïve high-risk non-muscle invasive bladder cancer have high rates of recurrence and progression," said Neal Shore, M.D., FACS, Medical Director for the Carolina Urologic Research Center, and lead investigator for the CREST trial. "These study results demonstrate the potential for sasanlimab in combination with BCG to redefine the treatment paradigm for patients living with BCG-naïve, high-risk non-muscle invasive bladder cancer, including patients with carcinoma in-situ (CIS), providing prolonged event-free survival which may delay or reduce the need for more aggressive treatment options. Administered subcutaneously every four weeks, sasanlimab, if approved, could also help lower the treatment burden on both patients and healthcare systems."

Each year, approximately 100,000 people globally are diagnosed with high-risk NMIBC.3 Induction therapy with BCG followed by maintenance has been the standard of care for patients with high-risk NMIBC for decades.4 40-50% of patients experience recurrent disease, often requiring radical cystectomy,5,6,7 which is associated with significant risks8,9,10 and bladder-sparing treatment options are still limited.11,12

"The initial therapy of high-risk, non-muscle invasive bladder cancer with BCG has not advanced in decades. Today’s pivotal Phase 3 CREST results are potentially practice-changing, representing the first advance in therapy for BCG-naïve, high-risk, non-muscle invasive cancer in over 30 years," said Roger Dansey, M.D., Chief Oncology Officer, Pfizer. "These results reinforce Pfizer’s leadership in genitourinary cancer research and development, demonstrating our ongoing commitment to deliver new treatment options for patients with bladder cancer."

The overall safety profile of sasanlimab in combination with BCG was generally consistent with the known profile of BCG and data reported from clinical trials with sasanlimab. The profile of sasanlimab was also generally consistent with the reported safety profile of PD-1 inhibitors.

Results will be submitted for presentation at an upcoming medical congress. Pfizer plans to discuss these data with global health authorities to support potential regulatory filings. Sasanlimab also continues to be investigated in combination with Pfizer’s antibody drug conjugate (ADC) portfolio in advanced solid tumors.

About CREST

The CREST trial is a Phase 3, multinational, randomized, open-label, three parallel-arm study of sasanlimab, an anti-PD-1 mAb, in combination with BCG (BCG induction with or without BCG maintenance) versus BCG (induction and maintenance) in participants with BCG-naïve, high-risk NMIBC. Patients were randomized to receive sasanlimab 300 mg by subcutaneous (SC) injection every four weeks up to cycle 25 (cycle = four weeks), in combination with BCG once weekly for six consecutive weeks (induction period) followed (Arm A) or not (Arm B) by maintenance with BCG, or BCG induction and maintenance up to cycle 25 (Arm C). The primary endpoint is EFS as assessed by the investigator, between Arm A and C, defined as the time from randomization to the earliest of recurrence of high-grade disease, progression of disease, persistence of CIS, or death. Key secondary endpoints include EFS as assessed by the investigator between Arm B and Arm C.

About Sasanlimab

Sasanlimab is a humanized immunoglobulin G4 mAb that binds to human programmed death-1 (PD-1) to block its interaction with PD-1 and PD-L1/PD-L2. PD-1 is a protein expressed on T cells, dendritic cells, natural killer cells, macrophages, and B cells, that functions as an immune checkpoint that negatively regulates T-cell activation and effector function when activated by its ligands and may play an important role in tumor evasion from host immunity. It can be administered through a once every four weeks subcutaneous injection by prefilled syringe (2mL).

In early-stage clinical studies, sasanlimab administered at 300 mg SC every four weeks showed clinical efficacy in advanced solid tumors and advanced urothelial cancer. In addition to NMIBC, sasanlimab is being evaluated in several ongoing clinical trials with other novel combinations.

On January 10, 2025 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel radioimmunotherapy and antibody-based therapeutic products for the treatment of cancer, reported strategic business updates and 2025 priorities in the Company’s mission to improve and extend people’s lives (Press release, Y-mAbs Therapeutics, JAN 10, 2025, View Source [SID1234649600]).

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Business Update

Y-mAbs is internally realigning operations with the establishment of two business units: Radiopharmaceuticals and DANYELZA.

● Radiopharmaceuticals
o Novel Self-Assembly DisAssembly Pre-targeted Radioimmunotherapy ("SADA PRIT") platform designed to improve upon traditional radioimmunotherapy by delivering high therapeutic dose while minimizing off-target exposure, increase physician participation and decrease manufacturing and infrastructure costs.
o SADA PRIT technology utilizes a pre-targeted payload delivery method where antibody constructs assemble in tetramers and bind to the tumor target.
o Platform can deliver a variety of payloads and could potentially be developed against multiple tumor targets, as well as for radiopharmaceutical purposes.
o Y-mAbs is currently evaluating its SADA PRIT technology in two clinical trials in the U.S.
● DANYELZA
o DANYELZA is a GD2 antibody and the only FDA-approved treatment for the treatment of patients one-year of age and older with high-risk relapsed/refractory neuroblastoma in the bone and bone marrow.
o Initially commercially launched in 2021, DANYELZA is commercialized across both U.S. and international markets.

The business realignment is designed to support the optimization of internal resources and provide flexibility and agility to advance the Company’s novel SADA PRIT platform programs through clinical development while simultaneously driving commercial growth of DANYELZA.

"Our mission at Y-mAbs has been clear from day one: bring important new cancer therapies to patients as quickly as possible. Since the successful commercial launch of DANYELZA, we are proud to have positively impacted the lives of children with high-risk relapsed/refractory neuroblastoma, giving the hope of a better future to families around the world," said Michael Rossi, President and Chief Executive Officer. "As we look ahead towards the potential of our novel Radiopharmaceutical platform and high value therapeutic areas, as well as the potential of DANYELZA, we believe now is the right time to focus our efforts into two business units. By doing so, we expect to expand our radiopharmaceutical capabilities, accelerate clinical execution, further improve capital efficiencies, and better align strategic priorities."

With these updates to our business strategy, the Company anticipates a reduction in its current workforce of up to approximately 13%, depending on whether a portion of impacted employees accept newly created positions. The Company also intends to move some roles from Denmark to the U.S. to more efficiently coordinate the advancement of its radiopharmaceutical platform, and implement a small adjustment to the DANYELZA commercial team to focus the team on potential growth opportunities within the anti-GD2 market.

Recent Pipeline Advancement

GD2-SADA (Trial 1001): Y-mAbs has dosed 21 patients at six sites to date in Part A of the GD2-SADA Phase 1 trial in adults with solid tumors. Tumor types include small cell lung cancer (SCLC), malignant melanoma, sarcomas and adult neuroblastoma. Preliminary data from the Part A GD2-SADA Phase 1 trial has demonstrated this novel pre-targeting approach to be well-tolerated with no dose-limiting toxicities (DLTs) and no treatment-related adverse events (AEs) reported. Part A remains ongoing as the Company aggregates final data on patients in open cohorts and continues to study further patients incorporating various elements to further optimize the platform aiming to maximize tumor uptake and retention. The Company expects to share data from Part A in the second quarter of 2025.

"The preliminary data from Part A of our GD2-SADA Phase 1 trial demonstrates the viability of the pre-targeted approach of the platform. We continue to gather learnings from the 21 patients dosed to date, which we anticipate will allow us to improve tumor uptake, determine the optimal therapeutic dose, and establish the ideal construct to further advance Trial 1001 in the clinic," said Norman LaFrance, M.D., Chief Development Officer. "We believe in the significant potential of our SADA PRIT platform, and we are excited to be at the forefront of this next-generation, pre-targeted radiotherapy technology."

CD38-SADA (Trial 1201): To date, six sites have been selected, and three sites have been activated. The Company expects to dose the first non-Hodgkin Lymphoma (NHL) patient in Study 1201 in the first quarter of 2025.

Unaudited Preliminary FY2024 Results

Y-mAbs reported preliminary estimated unaudited full year 2024 total net revenue of approximately $88 million, within the Company’s previously announced guidance range of between $87 million and $95 million, and preliminary estimated unaudited cash, cash equivalents and marketable securities as of December 31, 2024 of approximately $67 million, with preliminary estimated total cash investment for the full year 2024 of approximately $11 million, which is below the Company’s guidance range of between $15 million and $20 million.

Anticipated 2025 Milestones

● Part A data from GD2-SADA Phase 1 trial (Trial 1001) expected to be presented in the second quarter of 2025
● GD2-SADA optimization data expected to be presented in the second quarter of 2025
● Expect to present updates with respect to reprioritized SADA PRIT pipeline, including new high-value target indications and timelines, in the second quarter of 2025
● Expect to dose first patient in CD38-SADA Phase 1 trial (Trial 1201) in the first quarter of 2025
● Potential for marketing approval for DANYELZA in new ex-US market in 2025
● Plan to provide full year 2025 guidance in conjunction with full year 2024 earnings report in the first quarter of 2025

Upcoming Investor Presentation

The company previously announced that Mr. Rossi will present at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Wednesday, January 15, 2025 at 5:15 p.m. PT. A live webcast will be available under the Events section of the Company’s investor relations website at ir.ymabs.com. The webcast will be archived and available for replay for 30 days after the event.

On January 10, 2025 SN BioScience reported that its lead asset SNB-101 has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for a Phase 1b/2 clinical trial (Press release, SN BioScience, JAN 10, 2025, View Source [SID1234649601]). This achievement follows the designation of SNB-101 as an orphan drug for small cell lung cancer in 2023 and its Fast Track designation in 2024. With this latest clearance, SNB-101 is rapidly advancing toward early commercialization in the U.S. market. SN BioScience expects to initiate the clinical trials in Q2, 2025.

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Validated for ES-SCLC, SNB-101 Eyes 2028 U.S. Market Launch
SN BioScience has revealed details of its Phase 1b/2 clinical trial for SNB-101, targeting patients diagnosed with Extensive Stage Small Cell Lung Cancer (ES-SCLC). The trial will concentrate on dose escalation and optimization to improve both efficacy and safety. Approximately 55 patients will be enrolled, with a focus on reflecting the racial diversity of the U.S. population. Furthermore, the study, aimed at supporting Phase 2 clearance in Europe, will be conducted across South Korea, the U.S., and Europe. Efficacy, safety, and pharmacokinetics will serve as the primary endpoints. Following the dose optimization phase, the company plans to assess SNB-101’s efficacy and safety in approximately 100 patients through a single-arm, single-dose, open-label monotherapy study. If the results are positive, SN BioScience targets early U.S. commercialization, with market entry expected as early as 2028.

Small cell lung cancer (SCLC), representing 12-15% of all lung cancer cases, is an aggressive disease with a poor prognosis. Around 70% of patients are diagnosed at an extensive stage, where the cancer has already spread, resulting in a five-year survival rate of less than 7%. Despite these challenging statistics, the SCLC treatment market is expected to grow at a compound annual growth rate (CAGR) of 7-10% from 2023 to 2030, according to Evaluate Pharma, highlighting the urgent need for more effective therapies. In this context, SNB-101 emerges as a highly promising treatment option, offering the potential to significantly improve patient outcomes.

SN BioScience aims to establish its treatment as a second- or third-line option for patients resistant to existing therapies. Ultimately, the company seeks to position the therapy as a first- or second-line standard treatment, potentially in combination with immuno-oncology agents.

On January 10, 2025 Rein Therapeutics ("Rein") (NASDAQ: RNTX), formerly known as Aileron Therapeutics, Inc. ("Aileron") (NASDAQ: ALRN), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, reported that it has changed its name to Rein Therapeutics, Inc (Press release, Aileron Therapeutics, JAN 10, 2025, View Source [SID1234649585]). The new name, logo, website, and branding elements reflect the Company’s mission to develop first-in-class treatments to rein in diseases. The Company’s common stock will begin trading under the Nasdaq ticker symbol "RNTX" effective January 13, 2025. The CUSIP number for the Company’s common stock is not affected by the name change. The Company also highlighted its recent clinical and corporate achievements and provided its key strategic objectives for 2025.

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"Our rebrand to Rein Therapeutics reflects our unwavering commitment to address the critical needs of underserved patients with fibrotic diseases," said Brian Windsor, Ph.D., President and Chief Executive Officer. "This new chapter for the Company underscores our focus on reining in fibrosis and advancing our pipeline of novel candidates. We look forward to the initiation of a Phase 2 clinical trial for our lead candidate, LTI-03, in the first half of this year, aiming to bring hope to those affected by idiopathic pulmonary fibrosis."

Recent Clinical Achievements

LTI-03, a Caveolin-1 related peptide:
In November 2024, the Company announced positive topline data from Cohort 2 of the Phase 1b clinical trial evaluating the safety and tolerability of inhaled high dose LTI-03 (5 mg BID) and a set of exploratory biomarkers in patients with idiopathic pulmonary fibrosis (IPF). Four biomarkers showed statistical significance in the combined Cohort 1 and Cohort 2 dataset, and five demonstrated dose dependence with respect to low dose (2.5 mg BID) Cohort 1, indicative of active pharmacodynamics. High dose LTI-03 continued to exhibit a favorable safety profile.
2024 Corporate Highlights:
In March 2024, Brian Windsor, Ph.D., was appointed Chief Executive Officer of the Company, marking the Company’s sole focus on advancing a pipeline of first-in-class therapies for orphan pulmonary and fibrosis indications.

In May 2024, the Company completed an underwritten registered direct offering of its common stock and accompanying warrants raising net proceeds of approximately $17.7 million.
2025 Strategic Objectives and Anticipated Milestones

LTI-03: A Phase 2 trial of LTI-03 for the treatment of IPF is anticipated to be initiated in the first half of this year.

LTI-01: The Company’s Phase 2b-ready asset is a first-in-class therapy for the treatment of loculated pleural effusions (LPEs). It holds Orphan Drug Designation for the treatment of pleural empyema in the U.S and E.U. and Fast Track Designation in the U.S.