On May 6, 2025 Zetagen Therapeutics, Inc., a private, clinical stage, biopharmaceutical company focused on developing breakthrough therapies, via local administration, for metastatic and primary breast cancers, reported they have successfully completed enrollment in their phase 2a study, which will evaluate ZetaMet (Zeta-BC-003) in the treatment of spinal metastatic lytic breast cancer lesions (ClinicalTrials.gov #NCT05280067) (Press release, Zetagen Therapeutics, MAY 6, 2025, View Source [SID1234652599]).

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"We are excited to reach another critical milestone in the development of ZetaMet (Zeta-BC-003), " said Joe C. Loy, Zetagen’s Chief Executive Officer. "We launched this study with stage IV breast cancer patients suffering from spinal metastases, because we recognize their severe pain and how debilitating it is, and that current treatments remain largely palliative. Our objective with ZetaMet is to eliminate cancer cells responsible for bone destruction, alleviate pain, stimulate the regeneration of bone lost to lytic lesions, enhance overall quality of life, while improving survival rates."

The 26-week study, conducted at the University of British Columbia, (UBC) Vancouver, BC, Canada, will evaluate the safety and efficacy of ZetaMet (Zeta-BC-003) in treating vertebral bone defects created by lytic metastatic breast cancer. The study will measure the reduction of skeletal related events (SRE), pain, change in vertebral body defect size, and postoperative prescribed opioid use. The Company anticipates reporting top-line results early fourth quarter of 2025.

Zetagen expects to submit an Investigation New Drug (IND) application to the U.S. Food and Drug Administration ("FDA"), leveraging its FDA’s Breakthrough designations.

About ZetaMet (Zeta-BC-003)
ZetaMet’s (Zeta-BC-003) small molecule mechanism of action (MOA) via a novel molecular pathway initiates a circuit which results in tumor cell death.

ZetaMet (Zeta-BC-003) is the first-of-its-kind, synthetic, small-molecule delivered via a proprietary controlled-release carrier intended to resolve metastatic breast cancer bone lesions, administered locoregionally, to inhibit pain while regenerating new bone, with the potential to increase survival rates.

The US Food & Drug Administration (FDA) has recognized Zetagen’s discoveries with multiple Breakthrough Designations including ZetaMet (Zeta-BC-003).

Zetagen with FDA and Health Canada (HC) approval via their Expanded Access (Compassionate Use) programs has treated several patients with ZetaMet (Zeta-BC-003) with results published in multiple peer-reviewed journals.

Peer-reviewed 2-year follow up clinical data published in 2023 on ZetaMet (Zeta-BC-003) demonstrated resolution of 7 lytic lesions (radiated and non-radiated), reduction in pain, significant reduction in opioid pain medication (4-fold), prevention of vertebral fracture, and increased survival rate in a patient living with Stage 4 breast cancer.[i] To view this publication via open access, go to: View Source

On May 6, 2025 Mission Bio, a pioneer in single-cell multi-omics solutions, reported it has partnered with Integrated DNA Technologies (IDT), a global leader in genomics solutions, to introduce an advanced workflow for precise on- and off-target confirmation in gene editing applications (Press release, Mission Bio, MAY 6, 2025, View Source [SID1234652616]). This collaboration integrates IDT’s award-winning rhAmpSeq technology with Mission Bio’s Tapestri Platform to provide a comprehensive, high-resolution approach for assessing genome editing outcomes at the single-cell level.

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Genome editing is crucial for the development of the next wave of cell and gene therapies, enabling precise and multiple genetic modifications. However, ensuring the specificity and efficiency of these edits remains a critical challenge, particularly in cell and gene therapy (CGT) applications. Traditional bulk sequencing methods provide valuable insights into genome editing efficiency, but lack the resolution to capture single-cell heterogeneity, zygosity, and co-occurrence of edits within individual cells.

To address these challenges, Mission Bio and IDT have developed an integrated workflow combining IDT’s rhAmpSeq amplicon-based targeted resequencing chemistry with Mission Bio’s droplet-based single-cell DNA sequencing technology. This novel solution builds on published approaches that researchers have previously used to leverage the platforms. The new, integrated approach enhances the efficiency for obtaining both on-target efficacy assessment and off-target detection in a single-cell context, offering researchers an unprecedented level of accuracy and confidence in genome editing analysis.

In a proof-of-concept study, to be presented at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, the integrated workflow was interrogated using well-characterized cell lines and selected for their extensive orthogonal bulk sequencing data. By targeting loci with known indel variations ranging from single-base pair changes to large insertions and deletions (>2,000 bp), the study demonstrated the robustness of rhAmpSeq chemistry in single-cell applications. The resulting targeted panel proved compatible with both bulk and single-cell assays, ensuring seamless adoption across various research needs.

"Our single-cell multi-omics has been used in combination with bulk sequencing to add specificity that otherwise would not be possible," said Brian Kim, CEO of Mission Bio. "By integrating our capabilities with IDT’s trusted approach to bulk sequencing, we’re adding together multiple layers of genetic insights in a single drop."

The Tapestri Platform’s automated single-cell genome editing analytics pipeline provided a quantitative assessment of editing efficiency, off-target editing levels and zygosity, offering a critical layer of data resolution beyond bulk sequencing. By integrating rhAmpSeq’s advanced primer design with Mission Bio’s single-cell DNA resequencing technology, the new workflow establishes a cost-effective, scalable, and highly customizable solution for genome editing validation in CGT applications.

"Safety is key to unlocking the true promise of CRISPR, and this collaboration with Mission Bio reflects another step forward to advancing the delivery of safer and more efficient genome editing platforms," said Sandy Ottensmann, VP/GM, Gene Writing and Editing at IDT. "By pairing the precision of rhAmpSeq with the single-cell resolution of Tapestri, we aim to support cell and gene therapy developers in driving the future of safe, life-changing therapies to benefit more patients."

Researchers and industry professionals can learn more about this cutting-edge approach at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting this month. Mission Bio and IDT will be presenting a poster detailing the study’s findings, demonstrating the power of integrated single-cell sequencing for genome editing validation.

The poster "Comprehensive On- and Off-target Validation Using Integrated rhAmpSeq and Targeted DNA Resequencing Single-Cell Technology for Gene Editing Applications" will be presented on Thursday, May 15 at 5:30 pm CST.

An oral presentation delivered by Dr. Ayal Hendel of Bar-Ilan University, "Single-Cell Profiling of Genome-Editing Alterations and Functional Outcomes in CRISPR-Engineered Cells," will take place on Saturday, May 17 at 9:00 am CST.

Visit Mission Bio’s Booth #1749 at ASGCT (Free ASGCT Whitepaper) to see how this technology can accelerate your research.

For more information, visit missionbio.com or contact [email protected].

On May 6, 2025 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for patients with cancer, reported financial results for the first quarter ended March 31, 2025, and provided a pipeline update on its clinical-stage investigational molecules across multiple common cancers (Press release, Arcus Biosciences, MAY 6, 2025, View Source [SID1234652568]).

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"Beginning with an oral presentation at ASCO (Free ASCO Whitepaper) for casdatifan in ccRCC, we expect to report a steady stream of data from ARC-20 throughout the remainder of 2025 and into 2026. We believe these data will support our robust development plan for casdatifan across multiple settings. This includes our Phase 3 trial, PEAK-1 in the IO-experienced setting, our clinical trial with AstraZeneca, which will combine casdatifan with their anti-PD-1/CTLA-4 bispecific antibody in the IO-naive setting, and three new cohorts in ARC-20 evaluating casdatifan in first- and second-line ccRCC," said Terry Rosen, Ph.D., chief executive officer of Arcus. "We believe that casdatifan has the potential to change the treatment paradigm for ccRCC, including the displacement of TKIs in earlier line settings, and our development plan is designed to establish casdatifan as the HIF-2a inhibitor of choice. Our balance sheet remains strong, and our operational plan and priorities are focused on ensuring that casdatifan is funded through its first Phase 3 readout."
Pipeline Highlights:

Casdatifan (HIF-2a inhibitor)
Casdatifan Updates:
•New clinical data from three monotherapy expansion cohorts in ARC-20 were presented in an oral session at the 2025 ASCO (Free ASCO Whitepaper) Genitourinary (GU) Cancers Symposium in February. At the time of data cut-off (DCO, January 3, 2025), the efficacy-evaluable population included a total of 87 patients with ccRCC who had received at least two prior lines of therapy, including both anti-PD-1 and a vascular endothelial growth factor receptor 2 (VEGFR)-TKI therapy. These data support the potential for casdatifan to be a best-in-class HIF-2a inhibitor for the treatment of ccRCC:
◦Despite limited follow-up, two of the cohorts exceeded 30% confirmed overall response rate (inclusive of one partial response confirmed after the DCO)
◦A 9.7-month median progression-free survival (PFS) was reached for the 50mg twice-daily casdatifan monotherapy cohort; median PFS was not yet reached for other cohorts
◦No unexpected safety signals were observed at the time of DCO, and casdatifan had an acceptable and manageable safety profile across all doses.

Planned Data Readouts:
•June 2025: Safety and initial efficacy data for the ARC-20 cohort evaluating casdatifan plus cabozantinib in IO-experienced patients will be presented in an oral session at the ASCO (Free ASCO Whitepaper) Annual Meeting.
•Fall 2025: More mature data from the cohorts evaluating casdatifan monotherapy in patients who had progressed on both an anti-PD-1 and a TKI therapy.
•2026: More mature data from the casdatifan plus cabozantinib cohort and initial data from the new ARC-20 cohorts evaluating casdatifan in the first-line (1L) and IO-experienced settings.
Upcoming Study and Cohort Initiations:
•The PEAK-1 Phase 3 study evaluating casdatifan plus cabozantinib versus cabozantinib in IO-experienced ccRCC is expected to initiate in the second quarter of 2025.
•Shortly thereafter, Arcus and AstraZeneca expect to initiate a clinical study, part of AstraZeneca’s eVOLVE portfolio, evaluating casdatifan plus volrustomig, AstraZeneca’s investigational anti-PD-1/CTLA-4 bispecific antibody, in IO-naive ccRCC. This study will be operationalized by AstraZeneca.

Domvanalimab (Fc-silent anti-TIGIT antibody) plus Zimberelimab (anti-PD-1 antibody)
•Overall survival data from the Phase 2 EDGE-Gastric study, evaluating domvanalimab plus zimberelimab and chemotherapy in upper gastrointestinal (GI) adenocarcinomas, are expected to be presented in the fall of 2025.
•The first Phase 3 data readout for domvanalimab plus zimberelimab will be from the ongoing Phase 3 study STAR-221, evaluating domvanalimab plus zimberelimab and chemotherapy in PD-L1 all-comer 1L metastatic upper GI adenocarcinomas and is expected in 2026.

CD73-Adenosine Axis: Quemliclustat (small-molecule CD73 inhibitor) and Etrumadenant (A2a/A2b receptor antagonist)

Quemliclustat:

•In the fourth quarter of 2024, Arcus initiated PRISM-1, a Phase 3 trial of quemliclustat combined with gemcitabine/nab-paclitaxel versus gemcitabine/nab-paclitaxel in first-line treatment of metastatic pancreatic cancer. PRISM-1 is recruiting rapidly, with enrollment completion expected by the end of 2025.
Etrumadenant:
•In March 2025, we engaged with the U.S. Food and Drug Administration (FDA) regarding promising results from the ARC-9 study evaluating etrumadenant in third-line metastatic colorectal cancer (mCRC); although the FDA’s feedback confirmed the potential for a registrational path for this program in third-line mCRC, based on our strategic priorities, we are not pursuing a Phase 3 study at this time.

Financial Results for First Quarter 2025:

•Cash, Cash Equivalents and Marketable Securities were $1.0 billion as of March 31, 2025, compared to $992 million as of December 31, 2024. The increase during the period is primarily due to the net proceeds from our underwritten offering in February 2025, partially offset by the use of cash in our research and development activities. We believe our cash, cash equivalents, and marketable securities, together with available facilities, will be sufficient to fund operations through the initial pivotal readouts for domvanalimab, quemliclustat and casdatifan, which include PEAK-1.
•Revenues were $28 million for the first quarter 2025, compared to $145 million for the same period in 2024. In the first quarter 2025, Arcus recognized $20 million in license and development services revenue related to the advancement of programs, as well as $8 million in other collaboration revenue primarily related to Gilead’s ongoing rights to access Arcus’s research and development pipeline in accordance with the Gilead collaboration agreement. Arcus expects to recognize GAAP revenue of between $75 million and $90 million for the full year 2025.
•Research and Development (R&D) Expenses were $122 million for the first quarter 2025, compared to $109 million for the same period in 2024. The net increase of $13 million was primarily driven by higher costs in our early-stage development and preclinical program activities, driven by higher enrollment in our Phase 2 studies. The overall increase was partially offset by reduced spend in our late-stage development activities due to lower manufacturing costs driven by the timing of manufacturing activities. Non-cash stock-based compensation expense was $8 million for the first quarter 2025, compared to $10 million for the same period in 2024. For the first quarters 2025 and 2024, Arcus recognized gross reimbursements of $38 million and $37 million, respectively, for shared expenses from its collaborations, primarily the Gilead collaboration. R&D expenses by quarter may fluctuate due to the timing of clinical manufacturing and standard-of-care therapeutic purchases with a corresponding impact on reimbursements.
•General and Administrative (G&A) Expenses were $28 million for the first quarter 2025, compared to $32 million for the same period in 2024. The decrease was primarily driven by the costs incurred to obtain the Third Gilead Agreement Amendment in 2024. Non-cash stock-based compensation expense was $8 million for the first quarter 2025, compared to $10 million for the same period in 2024.
•Net Loss was $112 million for the first quarter 2025, compared to $4 million for the same period in 2024.

Conference Call Information:

Arcus will host a conference call and webcast today, May 6th, at 1:30 PM PT / 4:30 PM ET to discuss its first-quarter 2025 financial results and pipeline updates. To access the call, please dial +1 (404) 975-4839 (local) or +1 (833) 470-1428 (toll-free), using Access Code: 762544. Participants may also register for the call online using the following link: View Source To access the live webcast and accompanying slide presentation, please visit the "Investors & Media" section of the Arcus Biosciences website at www.arcusbio.com. A replay of the webcast will be available following the live event.

Arcus Ongoing and Announced Clinical Studies:
Trial Name
Arms
Setting
Status
NCT No.
Kidney Cancer
PEAK-1
cas + cabo vs. cabo
Post-IO ccRCC
Planned Phase 3
TBD
AstraZeneca Collaboration (part of eVOLVE portfolio)
cas + volru
2L+ IO-Naive ccRCC
Planned
TBD
ARC-20
cas, cas + cabo, cas +zim
1L, 2L, and 2L+ Cancer Patients/ccRCC
Ongoing Phase 1/1b
NCT05536141
Upper Gastrointestinal Cancers
STAR-221
dom + zim + chemo vs. nivo + chemo
1L Gastric, GEJ and EAC
Ongoing Registrational Phase 3
NCT05568095
EDGE-Gastric (ARC-21)
dom +/- zim +/- chemo
1L/2L Upper GI Malignancies
Ongoing
Randomized Phase 2
NCT05329766
Lung Cancer
STAR-121
dom + zim + chemo vs. pembro + chemo
1L NSCLC (PD-L1 all-comers)
Ongoing Registrational Phase 3
NCT05502237
PACIFIC-8
dom + durva vs. durva
Unresectable Stage 3 NSCLC
Ongoing Registrational Phase 3
NCT05211895
EDGE-Lung
dom +/- zim +/- quemli +/- chemo
1L/2L NSCLC (lung cancer platform study)
Ongoing Randomized Phase 2
NCT05676931
VELOCITY-Lung
dom +/- zim +/- sacituzumab govitecan-hziy or other combos
1L/2L NSCLC (lung cancer platform study)
Ongoing Randomized Phase 2
NCT05633667
Pancreatic Cancer
PRISM-1
quemli + gem/nab-pac vs. gem/nab-pac
1L PDAC
Ongoing Randomized Phase 3
NCT06608927
ARC-8
quemli + zim + gem/nab-pac vs. quemli + gem/nab-pac
1L PDAC
Ongoing Randomized Phase 1/1b
NCT04104672
Other
ARC-25
AB598
Gastric Cancer
Ongoing Phase 1
NCT05891171
ARC-27
AB801
NSCLC
Ongoing Phase 1
NCT06120075

cabo: cabozantinib; cas: casdatifan; ccRCC: clear cell renal cell carcinoma; dom: domvanalimab; durva: durvalumab; EAC: esophageal adenocarcinoma; GEJ: gastroesophageal junction; gem/nab-pac: gemcitabine/nab-paclitaxel; GI: gastrointestinal; IO: immunotherapy; nivo: nivolumab; NSCLC: non-small cell lung cancer; PDAC: pancreatic ductal adenocarcinoma; pembro: pembrolizumab; quemli: quemliclustat; volru: volrustomig; zim: zimberelimab.

On May 6, 2025 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it will participate at two upcoming investor conferences in May (Press release, Neurocrine Biosciences, MAY 6, 2025, View Source [SID1234652584]).

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Chief Executive Officer Kyle Gano and Vice-President of Investor Relations Todd Tushla will present at the BofA Securities 2025 Health Care Conference on Tuesday, May 13, 2025 at 11:20 AM Pacific Time in Las Vegas.
Chief Financial Officer Matt Abernethy and Chief Commercial Officer Eric Benevich will present at the RBCCM Global Healthcare Conference on Tuesday, May 20, 2025 at 11:30 AM Eastern Time in New York.

The live webcasts can be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcasts will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.

On May 6, 2025 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported a business update and announced financial results for the first quarter ended March 31, 2025 (Press release, Cogent Biosciences, MAY 6, 2025, View Source [SID1234652601]).

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"The first quarter of 2025 was very productive for Cogent as our team focused on executing across our portfolio in preparation for three transformative data readouts this year. We look forward to reporting top-line results from our registration-directed SUMMIT trial with bezuclastinib in patients with nonadvanced systemic mastocytosis in July, followed later in the year with top-line results from our APEX and PEAK trials," said Andrew Robbins, the Company’s President and Chief Executive Officer. "While we are preparing for a potential launch of bezuclastinib in 2026, we are also very proud of the progress we have made with our early-stage pipeline, including presentations from four distinct programs recently at the annual AACR (Free AACR Whitepaper) conference."

Recent Business Highlights

Announced expanded clinical results from the Open Label Extension (OLE) portion of the Company’s ongoing SUMMIT trial evaluating bezuclastinib in patients with nonadvanced systemic mastocytosis (NonAdvSM) at the 2025 American Academy of Allergy Asthma & Immunology Annual Meeting (AAAAI) meeting.
Bezuclastinib showed a 65% mean improvement in Total Symptom Score (TSS) at 48 weeks, including 88% of patients achieving at least a 50% reduction in TSS.
The safety profile for bezuclastinib remains favorable, with adverse events reported primarily as low-grade and reversible. No treatment-related bleeding or cognitive impairment was observed. The most common treatment-related adverse events were hair discoloration and transient elevations in liver transaminases. All cases of elevated transaminases were asymptomatic and fully reversible.
Presented updated preclinical data from the company’s KRAS, PI3Kα, FGFR2/3 and ErbB2 candidates at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting.
Upcoming Milestones

Announce top-line results from SUMMIT in July 2025. SUMMIT is a registration-directed, randomized, double-blind, placebo-controlled, global, multicenter, clinical trial of bezuclastinib in patients with NonAdvSM.
Announce top-line results from APEX in the second half of 2025. APEX is a registration-directed, global, open-label trial in patients with advanced systemic mastocytosis (AdvSM).
Announce top-line results from PEAK by the end of 2025. PEAK is a global, blinded, randomized Phase 3 clinical trial studying the combination of bezuclastinib and sunitinib versus sunitinib alone in patients with imatinib-resistant gastrointestinal stromal tumors (GIST).
First Quarter 2025 Financial Results

Cash and Cash Equivalents: As of March 31, 2025, cash, cash equivalents and marketable securities were $245.7 million. During the quarter, the Company sold shares under the Company’s at-the-market (ATM) stock offering for net proceeds of $24.3 million and incurred a non-recurring payment of $9.6 million related to annual performance-based bonus compensation. Based on its current plans, the Company expects its existing cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses and capital expenditure requirements into late 2026, including through clinical readouts from ongoing SUMMIT, PEAK, and APEX registration-directed trials.

R&D Expenses: Research and development expenses were $63.0 million for the first quarter of 2025 compared to $52.7 million for the first quarter of 2024. The increase was primarily due to costs incurred to support our on-going SUMMIT, PEAK and APEX clinical trials and to the continued progression of our early stage, preclinical and discovery programs. R&D expenses include non-cash stock compensation expense of $5.3 million for the first quarter of 2025 as compared to $4.4 million for the first quarter of 2024.

G&A Expenses: General and administrative expenses were $11.9 million for the first quarter of 2025 compared to $9.7 million for the first quarter of 2024. The increase was primarily due to the growth of the organization. G&A expenses include non-cash stock compensation expense of $4.8 million for the first quarter of 2025 as compared to $5.0 million for the first quarter of 2024.

Net Loss: Net loss was $72.0 million for the first quarter of 2025 compared to a net loss of $58.3 million for the first quarter of 2024.