On November 12, 2025 Enliven Therapeutics, Inc. (Enliven or the Company) (Nasdaq: ELVN), a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapeutics, reported financial results for the third quarter ended September 30, 2025, and provided a business update, including highlights of pipeline progress.

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"We continue to make great progress advancing the ENABLE Phase 1 trial of ELVN-001 in people living with CML. I’m pleased to report that during the quarter, we completed enrollment of the randomized Phase 1b portion of the ENABLE trial and achieved key readiness milestones across multiple geographies, positioning us to rapidly launch our Phase 3 trial globally next year," said Sam Kintz, Co-Founder and Chief Executive Officer of Enliven. "Since we announced our positive, updated ENABLE data in June, we have had oral presentations at multiple medical meetings, which is a testament to the strength of the data and the potential for ELVN-001 as a selective active-site inhibitor. Additionally, we are pleased that the CML community continues to validate our work and enthusiastically supports ELVN-001 as we move through clinical trials. We remain focused on clinical execution as we look to initiate our Phase 3 pivotal trial in 2026."

Pipeline Updates

ELVN-001 is a potent, highly selective, small molecule kinase inhibitor designed to specifically target the BCR::ABL gene fusion, the oncogenic driver for patients with chronic myeloid leukemia (CML).

Completed enrollment of the randomized Phase 1b cohorts of the ongoing ENABLE trial of ELVN-001 in CML (NCT05304377).
Presented encore data from the ENABLE Phase 1a/1b clinical trial of ELVN-001 at several medical meetings, including:
An oral and poster presentation at the Society of Hematologic Oncology (SOHO) 2025 Annual Meeting in Houston, Texas, on September 3, 2025.
An oral presentation at the European Society of Hematology International Chronic Myeloid Leukemia Foundation (ESH-iCMLf) 27th Annual John Goldman Conference on Chronic Myeloid Leukemia: Biology and Therapy in Estoril, Portugal, on October 10, 2025.
An oral presentation at the German, Austrian, and Swiss Societies for Hematology and Medical Oncology (DGHO) in Cologne, Germany, on October 25, 2025.
Featured in an oral presentation titled, ELVN-001 for the treatment of CML with and without T315I mutation: a Phase 1 trial in Japan, at the 87th Annual Meeting of the Japanese Society of Hematology (JSH) in Kobe, Japan, on October 10, 2025.
Remains on track to initiate a Phase 3 pivotal trial of ELVN-001 in 2026.
Upcoming Medical Meeting Presentations

The Company recently announced that data from the ENABLE Phase 1a/1b clinical trial of ELVN-001 in a subset of CML patients with atypical fusion transcripts will be presented at the 67th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) 2025 Annual Meeting and Exposition, taking place December 6-9, 2025, in Orlando, Florida. Andreas Hochhaus, M.D., will present the data in a poster presentation on December 7, 2025.

Upcoming Investor Conference Participation

Management will participate in a fireside chat at the Jefferies Global Healthcare Conference in London on Tuesday, November 18, 2025, at 2:00 p.m. GMT. The fireside chat will be webcast live and can be accessed by visiting the investor relations section of the Company’s website at View Source The webcast will be archived for a period of 90 days following the conclusion of the live event.

Third Quarter 2025 Financial Results

Cash Position: As of September 30, 2025, the Company had cash, cash equivalents and marketable securities totaling $477.6 million, which is expected to provide cash runway into the first half of 2029.
Research and development (R&D) expenses: R&D expenses were $18.2 million for the third quarter of 2025, compared to $21.3 million for the third quarter of 2024.
General and administrative (G&A) expenses: G&A expenses were $6.9 million for the third quarter of 2025, compared to $5.8 million for the third quarter of 2024.
Net Loss: Enliven reported a net loss of $20.1 million for the third quarter of 2025, compared to a net loss of $23.2 million for the third quarter of 2024.
About ELVN-001

ELVN-001 is a potent, highly selective, potentially best-in-class small molecule kinase inhibitor designed to specifically target the BCR::ABL gene fusion, the oncogenic driver for patients with chronic myeloid leukemia. As a highly selective active site inhibitor, ELVN-001 has a mechanism of action that is complementary to allosteric BCR::ABL1 inhibitors, which may play an increasingly important role in the standard of care. ELVN-001 was also designed to have activity against the T315I mutation, the most common BCR::ABL1 mutation, which confers resistance to nearly all approved TKIs, as well as activity against mutations known to confer resistance to allosteric BCR::ABL1 inhibitors.

About the ENABLE Trial

The ENABLE study (NCT05304377) is a Phase 1 study of ELVN-001 in patients with previously treated CML. The trial is currently in Phase 1a/1b development and is a dose escalation and expansion trial designed to evaluate safety and tolerability and to determine the recommended dose for further clinical evaluation of ELVN-001 in patients with CML with and without T315I mutations that is relapsed, refractory or intolerant to TKIs. Secondary endpoints include pharmacokinetics, MMR by central quantitative reverse transcriptase polymerase chain reaction, duration of MMR, BCR::ABL1 transcript levels and complete hematologic response. Enliven is preparing for the potential start of a pivotal trial for ELVN-001 in 2026.

(Press release, Enliven Therapeutics, NOV 12, 2025, View Source [SID1234659846])

On November 12, 2025 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported financial results for the third quarter ended September 30, 2025, and provided a corporate update.

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"Recent presentations of clinical data for VCN-01 and preclinical data for VCN-12 at medical congresses have highlighted the progress made across our oncology pipeline," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "Having successully completed the VIRAGE Phase 2b clinical trial, we are now pursuing interactions with the European Medicines Agency and the US FDA to seek alignment on a protocol for a proposed Phase 3 study of VCN-01 plus gemcitabine/nab-paclitaxel standard-of-care chemotherapy as first-line treatment for metastatic PDAC. In addition, we have made significant progress on the design of a potential Phase 2/3 clinical trial in the rare pediatric disease retinoblastoma for potential discussion with regulators in the first half of 2026. With capital raised from our recent warrant inducement and sales made pursuant to our at-the-market (ATM) facility, we are well positioned to advance our regulatory and clinical strategies and explore potential partnerships for the innovative assets in our pipeline."

Recent Highlights and Anticipated Milestones

VCN-01

Metastatic Pancreatic Ductal Adenocarcinoma (PDAC):

Expanded data from the VIRAGE randomized, controlled clinical trial investigating up to two doses of VCN-01 (zabilugene almadenorepvec) in combination with gemcitabine/nab-paclitaxel standard-of-care (SoC) chemotherapy in 112 metastatic PDAC patients were presented at ESMO (Free ESMO Whitepaper) 2025:
The ESMO (Free ESMO Whitepaper) presentation included data for patients randomized into the modified intent to treat (mITT) population, who received at least 1 dose of SoC chemotherapy (Arm I) or at least 1 dose of VCN-01 (Arm II) regardless of whether they received SoC chemotherapy. These were compared to data for the full analysis set (FAS), where patients received at least 1 dose of gemcitabine/nab-paclitaxel SoC chemotherapy (Arm I) or VCN-01 followed by at least 1 dose of SoC chemotherapy (Arm II).
As previously reported, the two doses of VCN-01 were well tolerated and the study met its primary endpoints, such that metastatic PDAC patients receiving VCN-01 with SoC chemotherapy had improved overall survival (OS), progression free survival (PFS) and Duration of Response (DoR) compared to SoC chemotherapy alone. Improvements in these parameters were observed in both the mITT and FAS populations.
Patients who received 2 doses of VCN-01 and started cycle 4 of SoC chemotherapy (Arm II) showed greater improvement in OS and PFS compared to patients who started cycle 4 of SoC chemotherapy alone (Arm I), indicating a potential benefit of additional VCN-01 doses.
Expanded data presented at ESMO (Free ESMO Whitepaper) 2025 showed that peaks of VCN-01 viral genomes (vg) in blood were similar after both VCN-01 doses and circulating vgs were measurable for at least 3 months after each dose despite the presence of neutralizing antibodies. These data highlight the persistent bioactivity of intravenously administered VCN-01 upon repeated dosing.
Following the successful VIRAGE Phase 2b trial, a study protocol for a potential Phase 3 clinical trial investigating VCN-01 plus gemcitabine/nab-paclitaxel SoC chemotherapy as first-line treatment for metastatic PDAC is planned to be discussed with regulators in Europe and the United States.
VCN-12

VCN-12 is a next generation oncolytic adenovirus developed as part of Theriva’s VCN-X discovery program
VCN-12 uses the same virus capsid as the Company’s lead clinical candidate VCN-01 (zabilugene almadenorepvec), but includes modifications intended to (i) increase stroma degradation by replacing human hyaluronidase PH20 with the more active bee hyaluronidase; and (ii) increase tumor cell lysis by expressing the pore forming protein parasporin-2 to enable both cytotoxic and immunogenic cell death.
Preclinical data for VCN-12 was presented at the ESGCT 32nd Annual Congress:
Data presented at ESGCT showed that VCN-12 provided increased cell killing compared to VCN-01 in a variety of cancer cell models in vitro and displayed higher levels of hyaluronidase activity. In animal studies, intravenous VCN-12 had a similar toxicity profile to VCN-01 in immunodeficient mice bearing human tumor xenografts. Intratumoral VCN-12 significantly reduced tumor growth compared to VCN-01 in immunocompetent hamsters bearing HP-1 pancreatic tumors and appeared to stimulate a persistent anti-tumor immune response that prevented tumor establishment in some VCN-12 treated animals. Additional preclinical studies to confirm these findings are ongoing.
Third Quarter Ended September 30, 2025 Financial Results

General and administrative expenses decreased to $1.9 million for the three months ended September 30, 2025, from $2.3 million for the three months ended September 30, 2024. This decrease of 18% is primarily comprised of the decrease in compensation costs offset by the increase in the fair value of the contingent consideration and increased investor relations expense. The charge related to stock-based compensation expense was the same for the three months ended September 30, 2025 and September 30, 2024. The Company expects general and administrative expenses to decrease due to the workforce reduction implemented on September 30, 2025.

Research and development expenses decreased to $2.6 million for the three months ended September 30, 2025, from approximately $2.7 million for the three months ended September 30, 2024. This decrease of 7% is primarily the result of lower clinical trial expenses related to the completion of the Company’s VIRAGE Phase 2b clinical trial of VCN-01 in PDAC, lower indirect cost related to decreased compensation and lower clinical trial expenses related to its Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, offset by higher patent expenses related to SYN-020. The Company anticipates research and development expenses to decrease due to the completion of its VIRAGE Phase 2b clinical trial of VCN-01 as it focuses on regulatory interactions around potential pivotal clinical trials of VCN-01 in PDAC and retinoblastoma, continues exploratory VCN-01 manufacturing scale-up activities, and continues supporting the Company’s other preclinical and discovery initiatives. The charge related to stock-based compensation expense was $110,000 for the three months ended September 30, 2025, compared to $59,000 related to stock-based compensation expense for the three months ended September 30, 2024.

Other income was $79,000 for the three months ended September 30, 2025 compared to other income of $161,000 for the three months ended September 30, 2024. Other income for the three months ended September 30, 2025 is primarily comprised of interest income of $65,000 and an exchange gain of $14,000. Other income for the three months ended September 30, 2024 is primarily comprised of interest income of $158,000 and exchange gain of $3,000.

Cash and cash equivalents totaled $7.5 million as of September 30, 2025, compared to $11.6 million as of December 31, 2024. Following a capital raise subsequent to September 30, 2025, the Company’s cash and cash equivalents totaled $15.5 millon. The Company’s current cash of approximately $15.5 million at early November 2025 will allow it to fund operations into the first quarter of 2027, including overhead costs, close out of the VIRAGE Phase 2b clinical trial, exploratory VCN-01 manufacturing scale-up activities, regulatory interactions regarding proposed VCN-01 clinical trials in PDAC and retinoblastoma, and preclinical studies supporting VCN-01 and VCN-12, the first candidate from the Company’s VCN-X discovery program. Additional funding will be required to initiate new VCN-01 clinical trials.

About VCN-01

VCN-01 (zabilugene almadenorepvec) is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to 142 patients to date in clinical trials of different cancers, including pancreatic ductal adenocarcinoma (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.

About Pancreatic Ductal Adenocarcinoma

Cancer of the pancreas consists of two main histological types: cancer that arises from the ductal (exocrine) cells of the pancreas or, much less often, cancers may arise from the endocrine compartment of the pancreas. Pancreatic ductal adenocarcinoma ("PDAC") accounts for more than 90% of all pancreatic tumors. It can be located either in the head of the pancreas or in the body/tail. Pancreatic cancer usually metastasizes to the liver and peritoneum. Other less common metastatic sites are the lungs, brain, kidney and bone. In its early stages, pancreatic cancer does not typically result in any characteristic symptoms, so in most cases it is diagnosed in its late stages (locally advanced non-metastatic or metastatic disease) when surgical resection and possibly curative treatment is not possible. It is generally assumed that only 10% of cases are resectable at presentation, whereas 30-40% of patients are diagnosed at local advanced/unresectable stage and 50-60% present with distant metastases.

About VIRAGE

VIRAGE was a two-arm, Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. Patients were enrolled at 5 sites in the U.S. and 9 sites in Spain. In both the control and VCN-01 (zabilugene almadenorepvec) treatment arms, patients received gemcitabine/nab-paclitaxel standard-of-care chemotherapy in repeated 28-day cycles until disease progression. In the VCN-01 treatment arm only, patients were also administered intravenous VCN-01 seven-days prior to starting the first and fourth cycles of gemcitabine/nab-paclitaxel treatment (study days 1 and ~92, respectively). Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, duration of response, and measures of VCN-01 biodistribution, replication, and immune response. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).

(Press release, Theriva Biologics, NOV 12, 2025, View Source [SID1234659845])

On November 12, 2025 Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), reported to host a virtual R&D Day highlighting the details of its platform innovation and next-generation ADC pipeline. The presentation will include an overview of Sutro’s near- and long-term priorities, beginning with STRO-004, its potential best-in-class Tissue Factor ADC, which has now entered clinical development. The Company will also provide details on its dual-payload ADC programs, uniquely enabled by its proprietary cell-free platform, and reveal STRO-227, its first dual-payload candidate designed to target PTK7.

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"The programs we are advancing showcase our platform’s unique potential to meaningfully expand the therapeutic window and address some of the most persistent challenges with conventional ADCs," said Jane Chung, Sutro’s Chief Executive Officer. "With STRO-004 entering the clinic and our dual-payload programs accelerating, we are positioned to deliver ADCs that combine optimized target engagement, differentiated design, and industry-leading pharmacokinetics to enable deeper and more durable responses to redefine what is possible for cancer care."

Ms. Chung continued: "The selection of PTK7 for our initial dual-payload program underscores our strategy to address difficult-to-treat solid tumors where standard approaches have been insufficient. Additionally, through strategic collaborations including our partnership with Astellas, we continue to broaden the impact of our ADC technology and advance novel payload combinations to deliver therapies with the potential to transform the standard of care to patients."

The event will feature presentations by key members of Sutro’s senior management team and Anthony Tolcher, M.D., FRCPC, FACP, a medical oncologist and founder and head of NEXT Oncology. Sutro management will participate in a Q&A session at the end of the presentation.

Next-Generation ADC Innovation
Sutro’s platform is designed to optimize every component of an ADC—the antibody, linker, and payload—rather than focusing on one or two elements in isolation. This holistic control is enabled by the Company’s cell-free technology, which allows unparalleled flexibility in designing ADCs and the potential to dose higher due to optimized safety and efficacy, even in complex biological settings. In addition, the platform enables sophisticated dual-payload combinations, enabling therapies that could redefine the standard-of-care.

Program Highlights and Near-Term Pipeline Milestones

Single-payload ADC Programs
Single-payload programs will establish Sutro’s foundation by tackling hard-to-reach, complex targets.

STRO-004: A potential best-in-class Tissue Factor (TF) targeting ADC, STRO-004, has entered clinical trials following recent IND clearance from the U.S. Food and Drug Administration (FDA). In preclinical TF-expressing models of head and neck squamous cell carcinoma, non-small cell lung cancer, colorectal cancer, and pancreatic cancer, STRO-004 showed promising anti-tumor activity. Combined with a highest non-severely toxic dose of 50 mg/kg in non-human primate studies, STRO-004 is anticipated to meaningfully widen the therapeutic window versus conventional ADCs to address a wide range of tumors of unmet need.

STRO-006: A potential best-in-class, highly selective integrin β6 (ITGB6) targeting ADC, STRO-006, has demonstrated a superior pharmacokinetic profile compared to the current ITGB6-targeting ADC in Phase 3 development. Additionally, STRO-006 demonstrated encouraging anti-tumor activity and duration of response in preclinical models. STRO-006 is expected to enter clinical development in 2026 for the treatment of multiple solid tumors.

Dual-Payload ADC Program
Sutro’s dual-payload ADCs are designed to overcome resistance, delay progression, and potentially set a new standard-of-care by unlocking durable efficacy.

STRO-227: Sutro has selected tyrosine-protein kinase-like 7 (PTK7) as the target for its initial dual-payload candidate. PTK7 is overexpressed in many different cancers, including breast, lung, ovarian and colorectal cancer. The Company is working to accelerate its dual-payload ADC program, with an IND submission now targeted for 2026/2027.

Summary of Pipeline Milestones

STRO-004: Phase 1 study ongoing; initial data expected mid-2026
STRO-006: IND submission targeted for 2026
STRO-227: IND submission targeted for 2026/2027

Next-Generation ADC Collaborations
Research and development programs are progressing under Sutro’s collaboration, with Astellas focused on dual-payload immunostimulatory ADCs (iADCs). The first iADC program is expected to enter the clinic in early 2026.

Webcast Information:
To access the live audio webcast beginning at 7:00AM PT / 10:00AM ET, please go to View Source An archived replay of the webcast will be available on the Company’s website following the event.

(Press release, Sutro Biopharma, NOV 12, 2025, View Source [SID1234659844])

On November 12, 2025 Lyell Immunopharma, Inc. (Nasdaq: LYEL), a late-stage clinical company advancing next-generation chimeric antigen receptor (CAR) T-cell therapies for patients with cancer, reported financial results and business highlights for the third quarter ended September 30, 2025.

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Lyell’s lead clinical program, rondecabtagene autoleucel (ronde-cel, or LYL314), is an autologous dual-targeting CD19/CD20 chimeric antigen receptor (CAR) T-cell product candidate under evaluation in PiNACLE, a single-arm pivotal trial enrolling patients with relapsed and/or refractory (R/R) large B-cell lymphoma (LBCL) in the third- or later-line (3L+) setting and in a Phase 1/2 trial in the 2L setting. A second pivotal trial, PiNACLE – H2H, which is a Phase 3 head-to-head CAR T-cell therapy randomized controlled trial of ronde-cel for LBCL in the 2L, is expected to begin by early 2026. The U.S. Food and Drug Administration (FDA) granted ronde-cel Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of patients with R/R LBCL receiving treatment in the 2L setting in November 2025 to complement the RMAT designation received for ronde-cel in the 3L+ setting in April 2025.

Lyell recently acquired exclusive global rights (outside of mainland China, Hong Kong, Macau and Taiwan) to LYL273, a novel autologous guanylyl cyclase-C (GCC)-targeted CAR T-cell product candidate in development for patients with refractory metastatic colorectal cancer (mCRC) and other GCC-expressing cancers from Innovative Cellular Therapeutics. At the highest dose level studied to date, patients with mCRC treated with LYL273 in a Phase 1 clinical trial conducted in the United States achieved a 67% confirmed overall response rate (ORR) and an 83% disease control rate per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 based on local site review, with a manageable safety profile. LYL273 is a GCC-targeted CAR T-cell product candidate enhanced with CD19 CAR expression and controlled cytokine release designed to improve cell expansion, immune cell infiltration and cell killing in the hostile tumor microenvironment.

"Lyell is well positioned with two next-generation CAR T-cell product candidates, each with promising clinical data. We look forward to effectively executing on both clinical programs taking full advantage of our expertise in CAR T-cell clinical development and our wholly-owned manufacturing facility capable of commercial launch," said Lynn Seely, MD, President and CEO of Lyell. "We will be presenting new ronde-cel clinical and translational data at the upcoming ASH (Free ASH Whitepaper) meeting and exposition and are focused on rapidly advancing the pivotal development of ronde-cel through our two pivotal programs: PiNACLE – H2H, which is expected to begin enrolling patients receiving treatment in the 2L setting by early 2026, and PiNACLE, our ongoing single-arm pivotal trial for patients receiving treatment in the 3L+ setting."

Third Quarter Updates and Recent Business Highlights

Lyell is advancing a pipeline of next-generation CAR T-cell product candidates targeting cancers with large unmet need and substantial patient populations. Ronde-cel is in pivotal development for patients with R/R LBCL and LYL273 is in Phase 1 clinical development for refractory mCRC. Lyell’s pipeline also includes additional preclinical programs targeting undisclosed solid tumor indications.

Ronde-cel: A next-generation dual-targeting CD19/CD20 CAR T-cell product candidate designed to increase complete response rates and prolong the duration of response as compared to approved CD19‑targeted CAR T-cell therapies for the treatment of LBCL

Ronde-cel is an autologous CAR T-cell product candidate with a true ‘OR’ logic gate to target B cells that express either CD19 or CD20 with full potency. It is manufactured with a process that enriches for CD62L-positive cells to generate more naïve and central memory CAR T cells with enhanced stemlike features and antitumor activity. Following successful End-of-Phase 1 meetings with the U.S. Food and Drug Administration (FDA), Lyell announced the initiation of two pivotal trials – PiNACLE and PiNACLE – H2H. PiNACLE is a single-arm pivotal trial that is currently underway. It is a seamless expansion of the 3L+ cohort of the Phase 1/2 trial of patients with R/R LBCL. PiNACLE – H2H is a Phase 3 head-to-head CAR T-cell therapy randomized controlled trial for LBCL in the 2L. Enrollment in PiNACLE – H2H is expected to begin by early 2026 and until then the Phase 1/2 trial continues to enroll patients with R/R LBCL receiving treatment in the 2L setting.

The FDA has granted ronde-cel Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for the treatment of R/R diffuse LBCL in the 3L+ setting. RMAT provides all the benefits of the Fast Track and Breakthrough Therapy designation programs and enables increased frequency of communications with the FDA on the development of ronde-cel.

PiNACLE is a single-arm pivotal trial evaluating ronde-cel at a dose of 100 x 106 CAR T cells in patients with LBCL receiving treatment in the 3L+ setting. The trial is expected to enroll approximately 120 patients with R/R LBCL. Patients may be treated with ronde-cel in either the inpatient or outpatient setting, and there is no upper age limit for eligibility. The primary endpoint of the trial is the overall response rate, including an evaluation of duration of response.
Two abstracts highlighting new clinical and translational data from the Phase 1/2 trial of ronde-cel for the treatment of aggressive LBCL have been accepted for oral presentation at the ASH (Free ASH Whitepaper) 67th Annual Meeting and Exposition in December 2025.
Updated data from the PiNACLE trial will be presented at ASH (Free ASH Whitepaper). Data from this trial are expected to form the basis of a Biologics License Application submission to the FDA in 2027 for patients with R/R LBCL receiving treatment in the 3L+ setting.
The FDA has granted ronde-cel RMAT designation for the treatment of patients with relapsed or refractory LBCL receiving treatment in the 2L setting. RMAT designation recognizes the potential of ronde-cel to address significant unmet needs of patients with LBCL and enables an increased frequency of communications with the FDA on the development of ronde-cel.
PiNACLE – H2H is a Phase 3 head-to-head CAR T-cell therapy trial, which will evaluate ronde-cel versus an investigator’s choice of approved CD19 CAR T-cell therapies (lisocabtagene maraleucel or axicabtagene ciloleucel) in patients with aggressive LBCL receiving treatment in the 2L setting. Patients randomized to ronde-cel will be treated with a dose of 100 x 106 CAR T cells. The primary endpoint of the trial is event-free survival. The trial is expected to enroll approximately 400 patients with R/R LBCL (200 per arm). Patients may be treated with ronde-cel in either the inpatient or outpatient setting.
Clinical site initiation is underway for PiNACLE – H2H in the United States, Canada and Australia, and the first patient is expected to be enrolled by early 2026.
Lyell recently announced the formation of an expert steering committee comprised of a distinguished group of lymphoma and cell therapy experts who will collaborate with the Company on the design and conduct of PiNACLE – H2H.

LYL273: A next-generation GCC-targeted CAR T-cell product candidate enhanced with CD19 CAR expression and controlled cytokine release designed to improve outcomes for patients with mCRC

LYL273 is a GCC-targeted CAR T-cell therapy enhanced with CD19 CAR expression and controlled cytokine release to increase cell expansion, immune cell infiltration and cancer cell killing in the hostile tumor microenvironment. LYL273 was granted Fast Track designation for the treatment of mCRC by the FDA.

Dose-dependent clinical activity was observed in patients with refractory mCRC in a U.S. Phase 1 clinical trial.
Across both dose levels, the overall response rate was 50% (6 of 12 patients), and the disease control rate was 83%. At Dose Level 2, the highest dose tested to-date, the overall response rate was 67%, including one patient with a pathological complete response, one patient with complete reduction in tumor volume of the target lesions (100% partial response) and two additional patients with confirmed partial responses. For patients treated at Dose Level 2, the disease control rate was 83%, and the median progression-free survival was 7.8 months.
The incidence and severity of treatment-related adverse events were highest at Dose Level 2, where the most common treatment-related adverse events were cytokine release syndrome in 83% (5/6) of patients (Grade 1, 67%; Grade 2, 17%) and diarrhea in 83% (5/6) of patients (Grade 1, 33%; Grade 2, 33%; Grade 3, 17%). The median duration of diarrhea was 11 days. Immune effector cell-associated neurotoxicity syndrome occurred in 33% (2/6) of patients (Grade 2, 17%; Grade 3, 17%) and resolved rapidly with treatment. One patient experienced a dose-limiting toxicity at Dose Level 2, including Grade 3 diarrhea, Grade 4 enterocolitis and death from fungal sepsis 48 days post-infusion. No Grade 3 or higher diarrhea occurred in the last three patients treated since establishing an optimized management protocol for diarrhea, including prophylaxis.
The U.S. Phase 1 clinical trial is continuing to enroll patients to determine the recommended Phase 2 dose.
The next data update from this Phase 1 clinical trial is expected in the first half of 2026.

Preclinical Pipeline, Technologies and Manufacturing Protocols

Lyell is advancing next-generation fully-armed CAR T-cell product candidates, each including multiple technologies, designed to overcome T-cell exhaustion and the lack of durable stemness, as well as immune suppression within the hostile tumor microenvironment.

The first IND for a fully-armed CAR T-cell product candidate with an undisclosed target for solid tumors is expected in 2026.

Third Quarter 2025 Financial Results

Lyell reported a net loss of $38.8 million for the third quarter ended September 30, 2025, compared to a net loss of $44.6 million for the same period in 2024. The $5.7 million decrease in net loss was primarily due to decreased personnel expenses, including a $2.4 million reduction in stock-based compensation expense attributable to lower headcount and the reduced value of new equity awards granted. The decrease was partially offset by a $4.0 million loss related to the put/call asset issued to certain institutional and other accredited investors as part of our July 2025 securities purchase agreement. Non‑GAAP net loss, which excludes stock-based compensation, non-cash expenses related to the change in the estimated fair value of success payment liabilities and certain non-cash investment gains and charges, decreased to $29.1 million for the third quarter ended September 30, 2025, compared to $37.1 million for the same period in 2024, primarily due to decreased personnel expenses resulting from lower headcount and lower interest income primarily driven by decreased interest rates in 2025 coupled with lower cash equivalent and marketable securities balances.

(Press release, Lyell Immunopharma, NOV 12, 2025, View Source [SID1234659840])

On November 12, 2025 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the third quarter ended September 30, 2025, and provided a business update.

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"With our achievements in the third quarter and year to date, Ascendis is making great progress toward achieving Vision 2030. The ongoing strong global launch of YORVIPATH is transforming our financial profile and, based on positive feedback from physicians and patients, we expect to continue to build on this momentum," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "With TransCon CNP now under FDA and EMA review, we are on the verge of bringing our third high-value medicine to patients, and we expect our engine for future innovation to drive sustainable growth for years to come."

Select Highlights & Anticipated 2025 Milestones

TransCon PTH:
(palopegteriparatide, marketed as YORVIPATH)
YORVIPATH revenue for the third quarter of 2025 totaled €143.1 million, including a negative foreign currency impact of €3.6 million compared to the previous quarter.
Continued uptake from YORVIPATH in the U.S., with more than 4,250 unique patient enrollments and more than 2,000 prescribing health care providers as of September 30, 2025.
Outside the U.S., YORVIPATH is available commercially or through named patient programs in more than 30 countries.
Our strategic partner Teijin announced that YORVIPATH is now commercially available for prescription in Japan, expanding global access. In the third quarter, we recognized €12.9 million in milestone revenue related to the approval.
PaTHway60 trial, is ongoing as a single-arm safety and efficacy trial in adults with hypoparathyroidism to enable dose titration up to 60 mcg/day and is intended to support U.S. label expansion of YORVIPATH.
PaTHway Adolescent trial is planned to begin during the fourth quarter and is intended to support U.S. label expansion of YORVIPATH for the treatment of children with hypoparathyroidism, 12 to less than 18 years of age.
Presented pooled analysis of 3-year data from PaTHway and PaTH Forward trials at the American Society of Nephrology (ASN) Kidney Week 2025, reinforcing that treatment with TransCon PTH led to rapid and sustained improvements in kidney function in adults with hypoparathyroidism.

TransCon hGH:
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA revenue for the third quarter of 2025 totaled €50.7 million, including a negative foreign currency impact of €1.6 million compared to the previous quarter.
The FDA approved our first label expansion for SKYTROFA in adult growth hormone deficiency, which represents the first of multiple additional label expansions we expect to pursue.
We initiated a basket trial for several established growth-hormone indications including idiopathic short stature (ISS), short stature homeobox-containing gene deficiency (SHOX deficiency), Turner syndrome, and small for gestational age (SGA).

TransCon CNP:
(navepegritide, FDA NDA and EMA MAA filed)
We recently completed our late-cycle meeting with the FDA, who accepted for priority review the New Drug Application (NDA) for the treatment of children with achondroplasia; Prescription Drug User Fee Act (PDUFA) goal date is November 30, 2025.
In Europe, our Marketing Authorisation Application (MAA) has been submitted and validated by the Committee for Medicinal Products for Human Use (CHMP) and is under review.
At the annual meeting of the American Society for Bone and Mineral Research (ASBMR), we presented new analyses from the pivotal ApproaCH Trial showing that children treated with TransCon CNP had improvements in the Physical Functioning domain of the Achondroplasia Child Experience Measure (ACEM-PF), with greatest benefits in younger children who had more severe genu varum (≥5°) at baseline, supporting benefits beyond linear growth.

TransCon CNP + TransCon hGH Combination Therapy
(navepegritide plus lonapegsomatropin)

Following a successful end-of-Phase 2 meeting with FDA, we plan to initiate a Phase 3 trial of TransCon CNP in combination with TransCon hGH this quarter.
Anticipate presenting 52-week data from the COACH Phase 2 trial of TransCon CNP + TransCon hGH in early 2026.
During the fourth quarter of 2025, we plan to submit an Investigational New Drug (IND) or similar to investigate TransCon CNP alone and in combination with TransCon hGH for the treatment of hypochondroplasia.

TransCon IL-2 β/γ
(onvapegleukin alfa)

In our IL-Believe trial, 70 patients with late-stage platinum-resistant ovarian cancer (PROC) (median 4 prior lines of therapy; 67% previously treated with at least 2 lines of taxane-containing therapy) have been enrolled. Data continued to suggest that TransCon IL-2 β/γ dosed every three weeks in combination with weekly paclitaxel is generally well-tolerated, with the majority of TransCon IL-2 β/γ-related treatment-emergent adverse events (TEAEs) being Grade 1 or 2 in severity.
We expect to provide median overall survival (OS) data for this cohort of 70 patients in the first quarter of next year as the dataset continues to mature.

Financial Update
Based on the continued growth of YORVIPATH, we achieved operating profitability. In the third quarter we recorded an operating profit of €11.0 million.
During the third quarter, our cash balance increased €45 million from €494 as of June 30, 2025, to €539 million as of September 30, 2025.

Third Quarter 2025 Financial Results
Total revenue for the third quarter of 2025 was €213.6 million, compared to €57.8 million during the same period in 2024. The year-over-year increase in revenue was primarily attributable to an increase in product revenue, which reflected a contribution of €134.6 million from YORVIPATH.

Total Revenue

(In EUR’000s) Three Months Ended
September 30,
Nine Months Ended
September 30,

2025
2024
2025
2024

Revenue
Commercial products 193,790 55,710 443,480 153,598
Rendering of services and clinical supply 6,134 1,272 13,228 9,637
Licenses 788 851 3,002 26,490
Milestones 12,922 — 12,922 —
Total revenue 213,634 57,833 472,632 189,725

Commercial Products Revenue

(In EUR’000s) Three Months Ended
September 30,
Nine Months Ended
September 30,

2025
2024
2025
2024

Revenue from commercial products
SKYTROFA 50,701 47,249 152,745 138,455
YORVIPATH 143,089 8,461 290,735 15,143
Total revenue from commercial products 193,790 55,710 443,480 153,598

Research and development costs for the third quarter of 2025 were €66.9 million, compared to €73.5 million during the same period in 2024. The decrease was driven by the completion of clinical trials and development activities within our Endocrinology Rare Disease pipeline.

Selling, general, and administrative expenses for the third quarter of 2025 were €113.4 million, compared to €69.8 million during the same period in 2024. The increase was primarily due to the continued impact from global commercial expansion, including launch activities for YORVIPATH.

Total operating expenses for the third quarter of 2025 were €180.3 million, compared to €143.4 million during the same period in 2024.

Operating profit for the third quarter of 2025 was €11.0 million primarily attributable to higher revenue from the launch of YORVIPATH in the U.S., partly offset by higher operating expenses.

Net finance expense for the third quarter of 2025 was €60.9 million, including non-cash remeasurement loss of financial liabilities of €47.2 million, compared to €2.9 million net finance income during the same period in 2024.

For the third quarter of 2025, Ascendis Pharma reported a net loss of €61.0 million, or €1.00 per share basic and diluted compared to a net loss of €99.2 million, or €1.72 per share basic and diluted for the same period in 2024.

As of September 30, 2025, Ascendis Pharma had cash and cash equivalents totaling €539 million compared to €560 million as of December 31, 2024. As of September 30, 2025, Ascendis Pharma had 61,695,211 ordinary shares outstanding, including 597,055 ordinary shares represented by ADSs held by the company.

Conference Call and Webcast Information
Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its third quarter 2025 financial results.

Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at View Source A replay of the webcast will be available in this section of the Ascendis Pharma website shortly after the conclusion of the event for 30 days.

(Press release, Ascendis Pharma, NOV 12, 2025, View Source [SID1234659839])