On November 6, 2025 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel allogeneic, or "off the shelf", cell therapies for serious medical conditions, reported its third quarter 2025 financial and operating results and will host a conference call today at 4:30 p.m. Eastern Time to discuss these results and provide a business update.

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"It has been a productive third quarter for the Lineage team," stated Brian M. Culley, Lineage CEO. "Last quarter, I outlined five areas of focus through the end of this year, and I am pleased to report that we delivered on several of these during the third quarter, as outlined below.

We entered into a research collaboration with William Demant Invest A/S, which is designed to fund all currently planned preclinical development of our ReSonance program, demonstrating the ability of our technology platform to produce a partnered program with limited investment and in a short amount of time.
We solidified our position as a leader in allogeneic cell process development by reporting cGMP production for each of OpRegen and OPC1, from a master and working cell bank system which, in its current form, can support a production capability of millions of doses of a single-administration product, all from our in-house facility.
We launched a new cell therapy initiative, focused on islet cell transplants for the treatment of Type 1 Diabetes, with an initial focus on addressing the unsolved issue of large-scale production of islet cells, which if successful could potentially solve a major hurdle to commercialization of islet cell therapy product candidates."
"Looking ahead, we will continue to work on the other strategic initiatives I outlined, including activities designed to drive milestone revenue from our alliance with Roche and Genentech and pursuing grant funding from the California Institute for Regenerative Medicine (CIRM) for our OPC1 program. We also continue to plan for a successful future by seeking to capitalize on our investments in our cell transplant platform and to utilize our manufacturing achievements as a foundation from which additional programs can be strategically advanced via funded collaborations or independently," added Mr. Culley.

Select Business Highlights

RG6501 (OpRegen Cell Therapy)
Positive RG6501 (OpRegen) Phase 1/2a Clinical Study 36 Month Results featured at Clinical Trials at the Summit (CTS) 2025, suggesting evidence of sustained gains in visual acuity and structural support of the retina.
Ongoing execution of Lineage’s contributions to its collaboration with Roche and Genentech, including support for the ongoing Phase 2a GAlette Study.
In addition to testing of other surgical parameters, Genentech currently plans to evaluate two proprietary surgical delivery devices that have potential advantages over available off-the-shelf devices in the Phase 2a GAlette Study.
Ongoing efforts to further support development of OpRegen cell therapy under a separate services agreement with Genentech, signed May 2024, including: (i) activities to support the ongoing Phase 1/2a study long term follow-up and the currently enrolling Phase 2a GAlette Study; and (ii) additional technical training and materials related to our cell therapy technology platform to support commercial manufacturing strategies.
ReSonance (ANP1)
Announced research collaboration with William Demant Invest A/S (WDI) to jointly advance development of ReSonance (ANP1) over a term of three years; up to $12 million of development costs to be contributed by WDI in a collaboration which covers preclinical development activities, including cell manufacturing, proof-of-concept studies, translational/functional models, delivery development, outcome measures, regulatory strategy, and market analysis.
Manufacturing Capability
Successfully completed a production run for each of OpRegen and OPC1, two of our product candidates, each produced from a customized, two-tiered current Good Manufacturing Practice ("cGMP") cell banking system, highlighting the application of the Lineage platform across multiple programs.
This production process utilizes a genetically-stable master cell bank created from a single, well-characterized pluripotent cell line, to generate a working cell bank, which then provides the source material for a final cell-based product candidate.
This demonstrated cGMP production process should enable the ability to produce millions of doses of a cost-effective, scalable and consistent supply of an allogeneic, cell-based product derived from a single initial cell line, that can be applied across multiple programs.
ILT1
Launched new cell therapy initiative, focused on islet cell transplants for the treatment of Type 1 Diabetes; plans are to deploy the company’s manufacturing capability to address the issue of large-scale production of islet cells, with the initial goal of establishing a production modality that can support the entire production process in a dynamic culturing system, which if successful could potentially solve a major hurdle to commercialization of islet cell therapy product candidates.
OPC1
First chronic spinal cord injury participant treated in the DOSED (Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device) clinical study.
First treated participant was a neurologically complete SCI injury (American Spinal Injury Association Impairment Scale [AIS] grade A), with a single neurological level of injury (NLI) from levels T1 to T10, and the novel delivery system successfully administered a one-time injection of OPC1.
No significant safety events have been reported sixty days following treatment in the first chronic SCI participant.
The California Institute for Regenerative Medicine (CIRM) continues to review Lineage’s application for a CLIN2 clinical grant to support our DOSED study of OPC1.
Balance Sheet Highlights

Cash, cash equivalents, and marketable securities of $40.5 million as of September 30, 2025, is expected to support planned operations into Q2 2027.

Third Quarter Operating Results

Revenues: Revenue is generated primarily from collaboration revenues, royalties, and other revenues. Total revenues for the three months ended September 30, 2025 were $3.7 million, a decrease of approximately $0.1 million as compared to $3.8 million for the same period in 2024. The decrease was primarily driven by lower royalty revenue and other service revenues recognized of $0.3 million, partially offset by more collaboration revenues of $0.2 million.

Operating Expenses: Operating expenses are primarily comprised of research and development ("R&D") expenses and general and administrative ("G&A") expenses. Total operating expenses for the three months ended September 30, 2025 were $7.5 million, a decrease of $0.1 million as compared to $7.6 million for the same period in 2024.

R&D Expenses: R&D expenses for the three months ended September 30, 2025 were $3.3 million, an increase of $0.1 million as compared to $3.2 million for the same period in 2024. The net increase was primarily driven by $0.2 million for our OPC1 program, $0.4 million for our preclinical programs and other undisclosed programs, partially offset by $0.5 million for our OpRegen program.

G&A Expenses: G&A expenses for the three months ended September 30, 2025 were $4.2 million, a decrease of $0.2 million as compared to $4.4 million for the same period in 2024. The decrease was primarily attributable stock-based compensation and services provided by third parties.

Loss from Operations: Loss from operations for the three months ended September 30, 2025 was $3.8 million, which was in-line with the comparative prior year period’s loss.

Other Income/(Expenses): Other income/(expenses) for the three months ended September 30, 2025 reflected other expense of $26.0 million, compared to other income of $0.8 million for the same period in 2024. The change was largely attributable to the non-cash quarterly fair value remeasurement of the warrant liabilities of $26.6 million primarily due to a change in our share price as compared to the prior year period, and $0.2 million for exchange rate fluctuations related to Lineage’s international subsidiaries.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended September 30, 2025 was $29.8 million, or $0.13 per share (basic and diluted), compared to a net loss of $3.0 million, or $0.02 per share (basic and diluted), for the same period in 2024. The change was primarily driven by the non-cash quarterly fair value remeasurement of the warrant liabilities due to a change in our share price as compared to the prior quarter.

Conference Call and Webcast

Interested parties may access the conference call on November 6, 2025, by dialing (888) 596-4144 from the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through November 14, 2025, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 3958367.

(Press release, Lineage Cell Therapeutics, NOV 6, 2025, View Source [SID1234659578])

On November 6, 2025 Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technologies to its Tumor Activated T Cell Engager (TRACTr), Tumor Activated Immunomodulator (TRACIr), and Adaptive Immune Response Modulator (ARM) platforms, reported financial results for the third quarter ended September 30, 2025, and provided a business update.

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"With enrollment ongoing in the JANX007 and JANX008 clinical trials and multiple new drug candidates expected to enter the clinic next year, we remain confident in our ability to advance these programs, supported by a strong balance sheet that enables continued execution," said David Campbell, Ph.D., President and CEO of Janux.

RECENT BUSINESS HIGHLIGHTS AND FUTURE MILESTONES:

•
JANX007 continues to enroll in the first-in-human Phase 1 clinical trial in mCRPC (NCT05519449).

•
JANX008 continues to enroll in the first-in-human Phase 1 clinical trial in advanced or metastatic solid tumors (NCT05783622).

Additional data from JANX007 and JANX008 will be presented at future Janux events in the fourth quarter of 2025.

THIRD QUARTER 2025 FINANCIAL RESULTS:

•
Cash and cash equivalents and short-term investments: As of September 30, 2025, Janux reported cash and cash equivalents and short-term investments of $989.0 million, compared to $1.03 billion at December 31, 2024.

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Research and development expenses: Research and development expenses for the quarter ended September 30, 2025 were $34.6 million, compared to $18.6 million for the comparable period in 2024.

•
General and administrative expenses: General and administrative expenses for the quarter ended September 30, 2025 were $10.6 million, compared to $17.7 million for the comparable period in 2024. With respect to the quarter ended September 30, 2024, $9.5 million of the general and administrative expense incurred was due to stock-based compensation expense associated with equity modifications.

•
Net loss: For the quarter ended September 30, 2025, Janux reported a net loss of $24.3 million, compared to a net loss of $28.1 million for the comparable period in 2024.

Janux’s TRACTr, TRACIr and ARM Pipeline

Janux’s first clinical candidate, JANX007, is a TRACTr that targets prostate-specific membrane antigen (PSMA) and is being investigated in a Phase 1 clinical trial in adult patients with mCRPC. Janux’s second clinical candidate, JANX008, is a TRACTr that targets epidermal growth factor receptor (EGFR) and is being studied in a Phase 1 clinical trial for the treatment of multiple solid cancers including colorectal carcinoma, squamous cell carcinoma of the head and neck, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma and triple-negative breast cancer. Janux is also advancing additional CD3-based TRACTr and CD28-based TRACIr programs for future clinical development, including a PSMA-TRACIr for use in combination with our PSMA-TRACTr JANX007, and a TROP2-TRACTr for the treatment of TROP2+ solid tumors. Janux is advancing its first ARM platform program candidate, a CD19-ARM for the potential treatment of autoimmune diseases toward clinical trials. Janux is also generating a number of additional TRACTr, TRACIr and ARM programs for potential future development.

(Press release, Janux Therapeutics, NOV 6, 2025, View Source [SID1234659577])

On November 6, 2025 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, reported third quarter and year-to-date 2025 financial results, business achievements, pipeline progress, and corporate updates.

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "We continued to see revenue growth with significant gross margin improvement in the third quarter of 2025. Amtagvi demand is increasing as we integrate our community treatment centers to drive earlier treatment and better outcomes for patients. We are building a successful commercial business, while advancing our high value development programs to address significant unmet medical needs in patients with solid tumor cancers."

Third Quarter Financial Highlights
Topline Growth, Significant Margin Improvement, and Initial Benefits of Cost Optimization

Total product revenue grew 13% over the prior quarter to ~$68 million, including U.S. Amtagvi revenue of ~$58 million and global Proleukin revenue of ~$10 million.
Gross margin of 43% from cost of sales of ~$39 million reflected improved execution and the initial benefits of cost optimization. Additional operational excellence initiatives are underway to drive further near- and long-term improvements.
Cash and cash equivalents, investments, and restricted cash totaled ~$307 million as of September 30, 2025. The current cash position, bolstered by expense reductions, is expected to fund operations into the second quarter of 2027.
Full-year 2025 revenue guidance is reaffirmed within the range of $250 to $300 million in the first full calendar year of Amtagvi sales.
Centralizing manufacturing at the Iovance Cell Therapy Center (iCTC) in early 2026 will reduce external manufacturing expenses and continue to improve gross margins.
Amtagvi U.S. Launch
Strong Commercial Execution Across Growing Academic and Community Treatment Networks

More than 80 U.S. authorized treatment centers (ATCs) have been activated across nearly 40 states, providing a broad network within a two-hour drive for ~95% of Amtagvi patients.
Community ATCs have treated their first Amtagvi patients, with growth acceleration expected in future quarters.
More community ATCs are being opened, which is expected to further drive demand.
Increased awareness of treatment benefits from real-world evidence data is driving earlier Amtagvi adoption and improved referral trends among medical oncologists.
A number of initiatives are underway to broaden patient access to Amtagvi, including a specialty pharmacy agreement with InspiroGene by McKesson.
Manufacturing turnaround time continues to improve with a current average of 32 days from inbound to return shipment to ATCs.
Amtagvi Global Expansion
Opportunity to Address up to 30,000 Patients Globally with Previously Treated Advanced Melanoma1

In August 2025, Health Canada granted the first Amtagvi approval outside the U.S. for patients with previously treated advanced melanoma.
Potential approvals of Amtagvi are anticipated in the United Kingdom and Australia in the first half of 2026 and Switzerland in 2027.
Iovance is finalizing a strategy with the European Medicines Agency (EMA) to support EU marketing authorization for Amtagvi.
Pipeline Progress and Anticipated Milestones by Program

Lifileucel in Solid Tumors

Positive interim data from the IOV-LUN-202 clinical trial demonstrated a potentially best-in-class clinical profile for lifileucel in previously treated advanced nonsquamous NSCLC patients.
The objective response rate (ORR) was 26% and the median duration of response (mDOR) was not reached at more than 25 months of follow up. Updated data will be presented at a medical meeting in 2026.
The U.S. Food and Drug Administration (FDA) previously provided positive regulatory feedback on the IOV-LUN-202 trial design and the proposed potency assay matrix to support registration.
Iovance expects the IOV-LUN-202 trial to complete enrollment in 2026 and support a supplemental Biologics License Application for lifileucel in nonsquamous NSCLC, with a potential launch in 2027.
Initial results from the IOV-END-201 clinical trial of lifileucel in previously treated advanced endometrial cancer are on track for early 2026.
The TILVANCE-301 clinical trial is active at more than 75 clinical sites and continues to accrue patients to investigate lifileucel in combination with pembrolizumab in frontline advanced melanoma. The trial is designed with FDA and EMA input to show contribution of components compared to pembrolizumab alone.
A new potentially registrational clinical trial, designated IOV-MEL 202, will investigate lifileucel in advanced melanoma patients previously treated with anti-PD-1 therapy, primarily outside the U.S. The trial includes outpatient use of Amtagvi in the community setting and will enroll a subgroup of true second-line BRAF mutation positive patients without prior BRAF inhibitor therapy.
Next Generation Programs

Clinical results for IOV-4001, a PD-1 inactivated TIL cell therapy, in previously treated advanced melanoma patients are anticipated in the first quarter of 2026. Other potential indications for IOV-4001 are also in development.
Dose escalation is continuing for IOV-3001, a second-generation, modified IL-2 analog for use in the TIL therapy treatment regimen. Preclinical studies of IOV-3001 demonstrated the potential for improved safety, convenience of less frequent dosing and strong effector T cell expansion. Advancement into Phase 2 development is expected in 2026.
An Investigational New Drug (IND) submission is planned in early 2026 for IOV-5001, a genetically engineered, inducible, and tethered interleukin-12 TIL therapy, in solid tumor cancers with large patient populations and urgent unmet medical.
Webcast and Conference Call

Management will host a conference call and live audio webcast to discuss these results and provide a corporate update today at 8:30 a.m. ET. To listen to the live or archived audio webcast, please register at View Source The live and archived webcast can be accessed in the Investors section of the Company’s website, IR.Iovance.com, for one year.

(Press release, Iovance Biotherapeutics, NOV 6, 2025, View Source [SID1234659576])

On November 6, 2025 Intensity Therapeutics, Inc. ("Intensity" or "the Company") (Nasdaq: INTS), a late-stage clinical biotechnology company focused on the discovery and development of novel intratumoral cancer therapies that are designed to kill tumors and increase immune system recognition of cancers using its proprietary non-covalent conjugation technology, reported third quarter 2025 financial results and provides a corporate update.

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Corporate Update

INVINCIBLE-4 Study: Phase 2 randomized open-label, multicenter study to analyze the clinical activity, safety, and tolerability of INT230-6 given before administration of the standard of care ("SOC") treatment in patients with early-stage, operable triple-negative breast cancer and SOC alone. The primary endpoint is the change in the pathological complete response rate for the combination compared to the SOC alone.

In October 2024, in collaboration with the Swiss Cancer Group, formerly the Swiss Cancer Group for Clinical Cancer Research (SAKK), the Company initiated and dosed our first patient in the INVINCIBLE-4 Study. In September 2025, the Company paused new patient enrollment to revise the dosing regimen for patients receiving INT230-6 in cohort A due to some patients in Cohort A experiencing localized skin irritation near the tumor site. The Company plans to file a protocol amendment for this revision in dosing in the first quarter of 2026, and expects to reinitiate enrollment for the 54-patient study in the first quarter of 2026. The Company is targeting to complete enrollment by the end of 2026 and will likely add resources to help sites enroll.

INVINCIBLE-3 Study: Phase 3 open-label, randomized study testing INT230-6 as monotherapy compared to the SOC drugs in second and third line treatment for specific soft tissue sarcoma subtypes. This study has been authorized by the US FDA, Health Canada, the European Medicines Agency (for France, Germany, Italy, Poland, and Spain), and Australia’s Therapeutic Goods Administration. The primary endpoint in the INVINCIBLE-3 Study is overall survival. In March 2025, the Company paused new site activations and patient enrollments due to funding constraints. Prior to this pause, the trial had enrolled 21 patients. The Company continues to treat patients enrolled in this study, maintain the database, conduct pharmacovigilance and other study related activities in cooperation with its third-party contract research organizations to reduce ongoing costs during this pause. Once sufficient funding is obtained, the Company plans to restart site activations and patient enrollment in the INVINCIBLE-3 Study.

IT-01 Study Manuscript Publication: On October 29, 2025, eBioMedicine, a Lancet Discovery Science journal, published the Company’s phase 1/2 IT-01 clinical study manuscript, "Safety and Efficacy of Intratumourally Administered INT230-6 in Adult Patients with Advanced Solid Tumours: Results from an Open-Label Phase 1/2 Dose Escalation Study," for the treatment of metastatic or refractory cancers. The manuscript included the following data results:
•In heavily pretreated patients with advanced disease having over 20 different types of cancer who had progressed following multiple prior lines of therapy, intratumoral INT230-6 achieved:
◦A disease control rate of 75% (48/64 patients) and median overall survival (mOS) of 11.9 months; these results compare favorably in phase 1/2 studies that historically reported an mOS of 4 to 7 months
◦In a metastatic sarcoma subset population receiving only INT230-6, the median overall survival was 21.3 months
•In an exploratory analysis comparing patients receiving INT230-6 at a total dose (in mL) that treated greater than 40% of the patient’s total tumor burden ("TTB") compared to those treated with less than 40% of their TTB, the:
◦Disease control rate was 83.3% (40/48) compared to 50% (8/16)
◦Median overall survival was 18.7 months (95% CI: 11.5–23.5) compared to 3.1 months (95% CI: 1.6–5.9) with a hazard ratio (HR) of 0.17 (95% CI: 0.081–0.342); P<0.0001
◦Improved survival was consistent across a range of low to high tumor burden and tumor sizes
•Approximately 20% of patients in the >40% group had uninjected tumors shrink, abscopal effects
•Fifteen of 64 patients survived for more than 21 months
•INT230-6 induced a qualitative decrease in proliferating cancer cells in injected tumors and a qualitative increase in activated T-cells infiltrating the tumor microenvironment
•No dose-limiting toxicities were reported among 64 monotherapy patients; seven patients had a grade 3 (10.9%) with no grade 4 or 5 treatment-related adverse events
•Pharmacokinetic results showed that greater than 95% of the active cytotoxic agents remained in the injected tumors

Capital Raises and Cash Runway: the Company has raised $13.6 million in gross proceeds since the beginning of the third quarter of 2025.
•$7.5 million raised in the third quarter of 2025 via the Company’s ATM (net proceeds of $7.2 million).
•$2.1 million raised in October 2025 via the Company’s ATM (net proceeds of $2.0 million).
•$4.0 million raised in October 2025 in a registered direct offering with an institutional investor, before deducting the placement agent’s fees and related offering expenses (net proceeds of $3.7 million).
With the capital raised to date, the Company has extended its cash runway until the end of the first quarter of 2027.

"In the past four months, we were able to substantially improve our balance sheet with multiple fundraising transactions. In particular, the October 2025 registered direct offering was significant, as this brought in a new long-term fundamental, healthcare-savvy investor. The new capital raised in 2025 enables us to execute on our business strategy until the end of the first quarter of 2027 without any additional funds. In addition, our peer-reviewed paper published in the Lancet Discovery Group’s journal, eBioMedicine, provides another level of validation of the potential of our lead drug INT230-6, injected directly into tumors, to treat multiple types of cancer in both the metastatic and local disease settings. We believe this is the first peer-review publication of a local therapy alone to report disease control rate, the potential for an overall survival benefit and a 20% patient abscopal rate in metastatic disease," stated Lewis H. Bender, Intensity Founder, President and CEO. "Lastly, working with our collaboration partners at the Swiss Cancer Institute, we analyzed the data from the patients in the Phase 2 randomized controlled INVINCIBLE-4 study, and have identified a path forward to restarting the study, which is expected to be in the first quarter of 2026. The endpoint will remain pathological complete response, which is accepted by FDA for accelerated approval."

Third Quarter 2025 Financial Results

Research and development expenses were $1.6 million for the three months ended September 30, 2025, compared to $2.2 million for the same period in 2024. Clinical trial expenses decreased $0.4 million primarily due to lower INVINCIBLE-3 Study costs. In March 2025, the Company paused new site activations and patient enrollments in the INVINCIBLE-3 Study, due to funding constraints. Prior to this pause, the trial had enrolled 21 patients. The Company will continue to treat all patients enrolled in this study in cooperation with our third-party contract research organizations during this pause, and once sufficient funding is obtained, the Company plans to restart site activations and patient enrollment.

General and administrative expenses were $1.2 million for the three months ended September 30, 2025, compared to $1.4 million for the same period in 2024. Consulting expense decreased due to less business development activity compared to the prior year period.

Overall, net loss was $2.7 million for the three months ended September 30, 2025, compared to a net loss of $3.5 million for the three months ended September 30, 2024.

As of September 30, 2025, cash and cash equivalents totaled $7.1 million.

About INT230-6
INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug consists of two proven, potent anti-cancer agents, cisplatin and vinblastine sulfate, and a diffusion and cell penetration enhancer molecule ("SHAO") that facilitates the dispersion of potent cytotoxic drugs throughout tumors, allowing the active agents to diffuse into cancer cells. These agents remain in the tumor, resulting in a favorable safety profile. In addition to local disease control and direct tumor killing, INT230-6 causes a release of a bolus of neoantigens specific to the malignancy, leading to immune system engagement and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression, which often occurs with systemic chemotherapy.

(Press release, Intensity Therapeutics, NOV 6, 2025, View Source [SID1234659575])

On November 6, 2025 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, reported an update on the MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials of nexiguran ziclumeran (nex-z) and announced other business updates and financial results for the third quarter ended September 30, 2025.

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"We were deeply saddened to learn that the patient who experienced Grade 4 liver transaminase elevations and increased total bilirubin following a dose of nex-z in the MAGNITUDE Phase 3 clinical trial, as reported on October 27, 2025, passed away last night," said Intellia President and Chief Executive Officer John Leonard, M.D. "We have been advised by the treating physician that this is a case with complicating comorbidities, and it is being further evaluated. As we await the FDA’s clinical hold letter, we are working with clinical investigators and external experts to better understand the liver-related events that have been observed within MAGNITUDE and to develop our risk mitigation plan."

"We continue to believe in nex-z’s potential to address important unmet needs for patients with ATTR amyloidosis," Dr. Leonard continued. "Regarding our other late-stage investigational product for the treatment of HAE, lonvo-z, we have made considerable progress over the course of 2025. Enrollment was completed in the HAELO Phase 3 clinical trial of lonvo-z in September, less than nine months after we dosed our first patient in the trial, putting us on track to share topline data by mid-2026. We look forward to presenting longer-term data from our Phase 1/2 clinical trial of lonvo-z on Saturday at ACAAI."

Recent Updates About Nexiguran Ziclumeran (nex-z) for Transthyretin (ATTR) Amyloidosis

Nex-z is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene in the liver, thereby preventing the production of transthyretin (TTR) protein. Nex-z offers the possibility of halting and reversing disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose. Intellia leads the development and commercialization of nex-z in collaboration with Regeneron Pharmaceuticals, Inc.

On October 29, 2025, the U.S. Food and Drug Administration (FDA) placed a clinical hold on the Investigational New Drug applications for the MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials for patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) and hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN), respectively.

More than 650 patients with ATTR-CM are currently enrolled in MAGNITUDE, and 47 patients with ATTRv-PN are enrolled in MAGNITUDE-2. To date, Grade 4 liver transaminase elevations have been reported in less than one percent of all patients enrolled in MAGNITUDE and no Grade 4 liver transaminase elevations have been reported in MAGNITUDE-2.

Intellia recently mandated that all MAGNITUDE and MAGNITUDE-2 clinical sites increase their monitoring of patient laboratory values in the weeks after dosing. The company continues to consult with clinical investigators and other experts to investigate the transaminase elevations and consider potential additional risk mitigation strategies while awaiting the FDA’s formal clinical hold letter. Given the clinical hold, the company has suspended its milestone guidance for nex-z pending regulatory alignment. Intellia plans to provide an update after it has finalized a plan with regulators on the path forward.

ATTR Amyloidosis with Cardiomyopathy (ATTR-CM):
On November 10, 2025 at the 2025 American Heart Association (AHA) Scientific Sessions, longer-term data will be presented in a late-breaker oral session from the company’s ongoing Phase 1 clinical trial in patients with ATTR-CM. Details about this session are as follows:
Title: Updated Phase 1 Clinical Trial Outcomes of CRISPR Gene Editing with Nexiguran Ziclumeran (Nex-z, NTLA-2001) in Patients with Transthyretin Amyloidosis with Cardiomyopathy
Session: Rewriting the Code for Cardiac Amyloid: Novel Identification, Treatment, and Cure
Date and Time: Monday, November 10, 2025, at 3:17 p.m. ET
Presenter: Julian Gillmore, M.D., Ph.D., Professor of Medicine, National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital, U.K.
Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN):
In September 2025, the company presented positive longer-term follow-up data from its Phase 1 clinical trial in an oral presentation at the 5th International ATTR Amyloidosis Meeting for Patients and Doctors in Baveno, Italy. The results were simultaneously published in the New England Journal of Medicine.

Recent Updates About Lonvoguran Ziclumeran (lonvo-z) for Hereditary Angioedema (HAE)

Lonvo-z is a wholly owned, investigational in vivo CRISPR-based therapy designed to inactivate the KLKB1 gene in the liver that offers the possibility of dramatically reducing or eliminating HAE attacks by driving consistent, deep and potentially lifelong reduction in kallikrein levels after a one-time treatment.

Dosing in the global Phase 3 HAELO clinical trial was initiated in January 2025 and enrollment was completed in September 2025. Patients in this trial are receiving a 50 milligram (mg) dose of lonvo-z. Intellia expects to:
Report HAELO topline data by mid-2026;
Submit a Biologics License Application (BLA) to the FDA in the second half of 2026; and
Continue preparing for an anticipated U.S. commercial launch in the first half of 2027.
On November 8, 2025 at the American College of Allergy, Asthma & Immunology Annual Scientific Meeting (ACAAI), longer-term clinical data will be presented in an oral session from all patients who received a 50 mg dose of lonvo-z in Intellia’s ongoing Phase 1/2 clinical trial. Details about this session are as follows:
Title: Two-Year Durability/Safety of One-time Lonvoguran Ziclumeran (Lonvo-z, NTLA-2002) 50 mg in Patients with Hereditary Angioedema
Session: Distinguished Industry & Late-Breaking Oral Abstracts – Session 1
Date and Time: Saturday, November 8, 2025, at 5:13 p.m. ET
Presenter: Danny Cohn, M.D., Ph.D., Internist, Department of Vascular Medicine, Amsterdam University Medical Center

Third Quarter 2025 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $669.9 million as of September 30, 2025, compared to $861.7 million as of December 31, 2024. In the third quarter of 2025, the company raised $114.5 million of net equity proceeds from its "At the Market" (ATM) program. The company’s cash, cash equivalents and marketable securities as of September 30, 2025, are now expected to fund operations into mid-2027 and through lonvo-z’s anticipated U.S. commercial launch for HAE.
Collaboration Revenue: Collaboration revenue was $13.8 million for the third quarter of 2025, compared to $9.1 million for the third quarter of 2024. The $4.7 million increase was mainly driven by cost reimbursements related to the company’s collaboration with Regeneron Pharmaceuticals, Inc.
R&D Expenses: Research and development (R&D) expenses were $94.7 million for the third quarter of 2025, compared to $123.4 million for the third quarter of 2024. The $28.7 million decrease was primarily driven by employee-related expenses, stock-based compensation, research materials and contracted services, partially offset by an increase in clinical trial expenses related to lonvo-z. Stock-based compensation expense included in R&D expenses was $12.2 million for the third quarter of 2025.
G&A Expenses: General and administrative (G&A) expenses were $30.5 million for the third quarter of 2025, compared to $30.5 million for the third quarter of 2024. Stock-based compensation expense included in G&A expenses was $7.4 million for the third quarter of 2025.
Net Loss: Net loss was $101.3 million for the third quarter of 2025, compared to $135.7 million for the third quarter of 2024.

Conference Call Information

The company will host a conference call and webcast today at 6:00 p.m. ET to discuss recent updates and the company’s third quarter 2025 financial results. To join the webcast, please visit the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com. To join by phone, U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726 approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call. A replay of the webcast will be available at intelliatx.com for approximately 90 days.

(Press release, Intellia, NOV 6, 2025, View Source [SID1234659574])