On May 7, 2025 Nuvation Bio Inc. (NYSE: NUVB), a global oncology company tackling some of the toughest challenges in cancer treatment, reported financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, Nuvation Bio, MAY 7, 2025, View Source [SID1234652674]).

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"Nuvation Bio continued to execute with focus in the first quarter as we prepare for the potential U.S. approval and launch of taletrectinib for advanced ROS1-positive NSCLC," said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. "Data recently published in the Journal of Clinical Oncology and presented at the European Lung Cancer Congress and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting further reinforce taletrectinib’s potential to deliver a differentiated profile with strong efficacy, high central nervous system activity, and favorable tolerability. We also continue to advance our broader pipeline, including safusidenib and NUV-1511, for which we plan to provide program updates later this year. With a team that has successfully brought numerous oncology therapies to market and a strong balance sheet, we are well-positioned to enter our next chapter as a commercial-stage company."

Recent Pipeline Updates:

Taletrectinib, ROS1 inhibitor: Advanced ROS1+ NSCLC

The Priority Review of the Company’s NDA for taletrectinib for advanced ROS1+ NSCLC (line agnostic, full approval) is progressing on time with all planned inspections now completed. The PDUFA goal date of June 23, 2025, positions Nuvation Bio to commercialize taletrectinib in the U.S., if approved, in mid-2025.
In January 2025, China’s National Medical Products Administration (NMPA) approved taletrectinib for adult patients with locally advanced or metastatic ROS1+ NSCLC. As part of an exclusive license agreement, Innovent Biologics is commercializing taletrectinib in Greater China.
In February 2025, Nuvation Bio launched an Expanded Access Program in the U.S., enabling eligible patients with advanced ROS1+ NSCLC to access taletrectinib outside of the ongoing pivotal TRUST-II study.
In March 2025, Nippon Kayaku completed submission of a Marketing Authorization Application for taletrectinib for advanced ROS1+ NSCLC to Japan’s Pharmaceuticals and Medical Devices Agency. As part of an exclusive license agreement, Nippon Kayaku will commercialize taletrectinib in Japan.
In March 2025, new data from a matching-adjusted indirect comparison study evaluating taletrectinib versus crizotinib in advanced ROS1+ NSCLC were presented at the European Lung Cancer Congress.
In April 2025, results from the pivotal Phase 2 TRUST-I and TRUST-II studies were published in Journal of Clinical Oncology.
In April 2025, new nonclinical data for taletrectinib in ROS1+ NSCLC were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
Safusidenib, mIDH1 inhibitor: Diffuse IDH1-mutant glioma

The Company expects to provide an update on the pivotal study design for the safusidenib program in the second half of 2025.
NUV-1511, drug-drug conjugate (DDC): Advanced solid tumors

The Company expects to provide an update from the Phase 1/2 dose escalation study of NUV-1511 in the second half of 2025.
NUV-868, BD2-selective BET inhibitor: Advanced solid tumors

As previously announced, the Company is evaluating next steps for the NUV-868 program, including external partnership opportunities or further development in combination with approved products for indications in which BD2-selective BET inhibitors may improve outcomes for patients.
Corporate Updates:

In March 2025, Nuvation Bio secured up to $250 million in non-dilutive financings from Sagard Healthcare Partners. The Company will receive $150 million in royalty interest financing and $50 million in debt upon U.S. FDA approval of taletrectinib by September 30, 2025, with access to an additional $50 million in debt at the Company’s option until June 30, 2026, as long as the Company has achieved first U.S. commercial sale. The royalty interest financing is expected to fully fund the U.S. commercial launch of taletrectinib; the pro forma cash balance is expected to fully fund development of the Company’s clinical-stage pipeline and create a path to potential profitability without a need for additional fundraising.
In March 2025, Nuvation Bio appointed Stephen Dang, Ph.D., as Senior Vice President, General Counsel. Dr. Dang brings over 17 years of experience in the biopharmaceutical industry across all stages of the drug product life cycle.
First Quarter 2025 Financial Results

As of March 31, 2025, Nuvation Bio had cash, cash equivalents, and marketable securities of $461.7 million.

For the three months ended March 31, 2025, research and development expenses were $24.6 million, compared to $12.8 million for the three months ended March 31, 2024. The increase was primarily due to a $6.2 million increase in personnel-related costs driven by the acquisition of AnHeart as well as stock-based compensation and other benefits and $0.2 million increase in amortization of assembled workforce and $5.4 million increase in third-party costs related to clinical trial expense for taletrectinib.

For the three months ended March 31, 2025, selling, general, and administrative expenses were $35.4 million, compared to $7.4 million for the three months ended March 31, 2024. The increase was due to a $10.7 million increase in personnel-related costs as a result of the acquisition of AnHeart, $12.2 million increase in sales and marketing expenses, $1.4 million increase in professional fees, $0.5 million increase in occupancy expenses, $1.1 million increase in legal fees, $0.2 million increase in foreign currency impact and $2.0 million increase in other expenses as a result of the integration of AnHeart offset by $0.1 million decrease in taxes.

For the three months ended March 31, 2025, Nuvation Bio reported a net loss of $53.2 million, or $(0.16) per share. This compares to a net loss of $14.8 million, or $(0.07) per share, for the comparable period in 2024.

Conference Call and Webcast

Nuvation Bio will host a conference call on Wednesday, May 7, 2025, at 4:30 p.m. ET, during which Company executives will provide an overview of its programs, summarize the commercial strategy for taletrectinib, and review financial results for the first quarter of 2025.

Investors and the general public are invited to listen to the live webcast and can register on the Nuvation Bio website at View Source Those unable to register can access the live conference call by dialing +1 833-470-1428 (U.S. toll-free) and entering access code 341248.

A replay of the webcast will be available on the Company’s website shortly after the conference call concludes.

About Taletrectinib

Taletrectinib is an oral, potent, central nervous system-active, selective, next-generation ROS1 inhibitor specifically designed for the treatment of patients with advanced ROS1+ NSCLC. Taletrectinib is being evaluated for the treatment of patients with advanced ROS1+ NSCLC in two Phase 2 single-arm pivotal studies: TRUST-I (NCT04395677) in China, and TRUST-II (NCT04919811), a global study.

Based on pooled results of the TRUST-I and TRUST-II clinical studies, the U.S. FDA has accepted and granted Priority Review to Nuvation Bio’s NDA for taletrectinib for advanced ROS1+ NSCLC (line agnostic, full approval) and assigned a PDUFA goal date of June 23, 2025. The U.S. FDA previously granted taletrectinib Breakthrough Therapy Designation for the treatment of patients with locally advanced or metastatic ROS1+ NSCLC who either have or have not previously been treated with ROS1 TKIs, and Orphan Drug Designation for the treatment of patients with ROS1+ NSCLC and other NSCLC indications. In January 2025, China’s NMPA approved taletrectinib for the treatment of adult patients with locally advanced or metastatic ROS1+ NSCLC.

On May 7, 2025 Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, reported financial results for the first quarter of 2025 and recent business highlights (Press release, Geron, MAY 7, 2025, View Source [SID1234652640]).

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"We are confident in the long-term potential of RYTELO as an important therapeutic for eligible patients with lower-risk-MDS and are sharply focused on maximizing the U.S. commercial opportunity," said Dawn Carter Bir, Interim President and Chief Executive Officer of Geron. "We have received positive feedback from clinicians who have utilized RYTELO, supporting its strong therapeutic profile. We’ve identified specific opportunities and are making focused investments that we believe will strengthen the U.S. commercial trajectory. We expect our increased commercial investments to bolster uptake across a broader group of prescribers and drive long-term demand. We are also expanding our medical affairs efforts to support increased awareness and education. Looking ahead, our Phase 3 IMpactMF trial evaluating overall survival with imetelstat in patients with JAKi relapsed/refractory myelofibrosis (R/R MF), which we believe represents a tremendous expansion opportunity, is progressing well and the event-driven interim analysis is still expected in the second half of 2026."

Recent Business Highlights

Continued first year of U.S. commercialization of RYTELO, with net product revenue of $39.4 million in the first quarter of 2025. Demand for RYTELO in the 13-week period through the week ending March 28 increased 1% compared to the prior 13 weeks.
Received marketing authorization for RYTELO from the European Commission (EC) as a monotherapy for the treatment of adult patients with transfusion-dependent (TD) anemia due to very low, low, or intermediate risk myelodysplastic syndromes (lower-risk MDS or LR-MDS) without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy (ESAs).
Launch planning is underway and Geron expects to commercialize RYTELO in select EU countries commencing in 2026.
Reached 85% enrollment in Phase 3 IMpactMF clinical trial evaluating imetelstat in patients with JAKi R/R MF. We continue to expect an interim analysis readout for overall survival in the second half of 2026 (when approximately 35% of patient events have occurred), and final analysis in the second half of 2028 (when approximately 50% of patient events have occurred).
The Company believes there is a significant market opportunity for imetelstat to treat JAKi R/R MF patients based on its unique mechanism of action, strong clinical data to date, and the substantial size of the addressable patient population, if the trial is positive and imetelstat is approved in this indication.
Clinical data from the Phase 2 study showed a strong signal regarding prolonged survival and resolution of bone marrow fibrosis in patients treated with imetelstat, suggesting the potential for disease modification.
First Quarter 2025 Financial Results

As of March 31, 2025, Geron had approximately $457.5 million in cash, cash equivalents, restricted cash and marketable securities, compared to $502.9 million as of December 31, 2024.

Net Loss

For the three months ended March 31, 2025, the Company reported a net loss of $19.8 million, or $0.03 per share, compared to $55.4 million, or $0.09 per share, for the three months ended March 31, 2024.

Revenues

Total product revenue, net for the three months ended March 31, 2025, was $39.4 million. There was no product revenue in the prior year period, as RYTELO was approved by the FDA in June 2024. The decline in net product revenue compared to the three months ended December 31, 2024, which was $47.5 million, was primarily due to inventory drawdown among RYTELO distributors from the fourth quarter of 2024 into the first quarter of 2025.

Total net revenue for the three months ended March 31, 2025, was $39.6 million, compared to $0.3 million for the same period in 2024. Total net revenue includes license fees and royalties in addition to any product revenue, net. The increase in revenue is due to product revenue from U.S. sales of RYTELO, which was approved by the FDA in June 2024.

Operating Expenses

Total operating expenses for the three months ended March 31, 2025, were $56.3 million, compared to $56.4 million for the same period in 2024.

Cost of goods sold was approximately $1.2 million for the three months ended March 31, 2025, which consisted of costs to manufacture and distribute RYTELO, compared to nil in the prior year period.

Research and development expenses for the three months ended March 31, 2025, were $15.1 million, compared to $29.4 million for the same period in 2024. The decrease was primarily due to the wind down of clinical trial costs associated with a decrease of activity in our Phase 3 IMerge MDS study after FDA approval of RYTELO in 2024, as well as manufacturing and quality costs that were capitalized in the current period now that RYTELO is approved, versus being expensed in the prior year period.

Selling, general and administrative expenses for the three months ended March 31, 2025, were $40.0 million, compared to $27.1 million for the same period in 2024. The increase in general and administrative expenses in 2025 as compared to 2024 primarily reflects higher personnel expenses related to increased headcount to support commercialization of RYTELO in the U.S.

Interest income was $5.2 million for the three months ended March 31, 2025, compared to $4.2 million for the same period in 2024. The increase in interest income in 2025 compared to 2024 primarily reflects a larger marketable securities portfolio, with the receipt of net cash proceeds from synthetic royalty and debt financings in November 2024. Interest earned in future periods will depend on the size of our marketable securities portfolio and prevailing interest rates.

Interest expense was $8.2 million for the three months ended March 31, 2025, compared to $3.4 million for the same period in 2024. The increase in interest expense primarily reflects $4.8 million in non-cash interest expense related to our synthetic royalty agreement and an increased principal debt balance under our loan agreement and a prior loan agreement, which was repaid in the fourth quarter of 2024. Interest expense reflects interest expense recognized under the synthetic royalty agreement, interest owed under the loan agreements, as well as amortization of associated debt issuance costs and debt discounts using the effective interest method and accrual for an end of term charge.

2025 Financial Guidance

For fiscal year 2025, the Company maintains its previously announced expectations of total operating expenses to be in the range of approximately $270 million to $285 million, which includes non-cash items such as stock-based compensation expense, amortization of debt discounts and issuance costs, and depreciation and amortization.

Based on the current operating plans and assumptions, the Company believes that existing cash, cash equivalents, and marketable securities, together with anticipated net revenues from U.S. sales of RYTELO, will be sufficient to fund projected operating requirements for the foreseeable future.

Conference Call

Geron will host a conference call at 8:00 a.m. ET on Wednesday, May 7, 2025, to discuss business updates and first quarter 2025 financial results.

A live webcast of the conference call and related presentation will be available on the Company’s website at www.geron.com/investors/events. An archive of the webcast will be available on the Company’s website for 30 days.

Participants may access the webcast by registering online using the following link, View Source

About RYTELO (imetelstat)

RYTELO is an oligonucleotide telomerase inhibitor approved in the U.S. for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.

In addition, RYTELO is approved in the European Union as a monotherapy for the treatment of adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration and the European Commission.

On May 7, 2025 UroGen (NASDAQ: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that the U.S. Food and Drug Administration (FDA) has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting on May 21, 2025 to review the new drug application (NDA) for UGN-102 (mitomycin) for intravesical solution, an investigational treatment for recurrent LG-IR-NMIBC (Press release, UroGen Pharma, MAY 7, 2025, View Source [SID1234652658]).

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The ODAC meeting will provide an opportunity for independent clinicians and other experts to evaluate the UGN-102 data and make a recommendation to the FDA as to whether the NDA should be approved and under what conditions. The FDA carefully considers the advice of the Advisory Committee, but is not bound by its recommendations. The FDA has stated in its correspondence to the Company that they intend to complete their review in time to meet the Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025.

"We are excited to discuss our data with the Advisory Committee and broader medical community as we continue our mission to bring innovative solutions to patients suffering from bladder cancer," said Liz Barrett, President and Chief Executive Officer of UroGen. "We believe UGN-102 represents a meaningful advancement for patients facing the recurrent and challenging nature of LG-IR-NMIBC, and we look forward to the opportunity to discuss its potential."

UGN-102 is in development as a therapeutic option for patients with recurrent LG-IR-NMIBC, a condition for which there are no FDA-approved drugs. The NDA is supported by results from the pivotal Phase 3 ENVISION trial which demonstrated a 79.6% complete response (CR) rate at 3 months after first instillation of UGN-102, and duration of response of 82.5% at 12 months after 3-month CR by Kaplan-Meier analysis.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102, scheduled an ODAC meeting for May 21, 2025, and assigned a PDUFA target action date of June 13, 2025.

About Low-Grade Non-Muscle Invasive Bladder Cancer (NMIBC)

In the U.S., bladder cancer is the second most common urologic cancer in men and primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. More specifically, LG-IR-NMIBC represents approximately 23,000 newly diagnosed bladder cancer patients each year and an estimated 59,000 recurrences annually among patients diagnosed in previous years. Guideline recommendations for the management of NMIBC include trans-urethral resection of bladder tumor (TURBT) as the standard of care. Up to 70% of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures.

On May 7, 2025 Experts from City of Hope, one of the largest and most advanced cancer research and treatment organizations in the United States with its National Medical Center named a Top 5 "Best Hospital" in the nation for cancer care by U.S. News & World Report, reported that it will be among the specialists sharing the biggest oncology research data of the year at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place May 30-June 3 in Chicago and online (Press release, City of Hope, MAY 7, 2025, View Source [SID1234652675]).

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This year’s ASCO (Free ASCO Whitepaper) meeting — themed "Driving Knowledge to Action. Building a Better Future" — unites nearly 45,000 oncology professionals to discuss leading-edge scientific data and attend educational sessions, empowering health care teams to deliver more effective, lifesaving cancer care to patients.

"Every year City of Hope researchers share potentially practice-changing oncology data at the ASCO (Free ASCO Whitepaper) meeting to build a better future for cancer patients and their families. We are excited to continue that tradition this year — to collaborate with and inform the best oncology experts in the world for the benefit of cancer patients everywhere," said Marcel van den Brink, M.D., Ph.D., president of City of Hope Los Angeles and National Medical Center, and chief physician executive.

Highlights of City of Hope’s ASCO (Free ASCO Whitepaper) data and educational sessions include those listed below, which focus on cancers of the breast, pancreas, colon and rectum, prostate, kidney and blood as well as supportive care, including palliative care and integrative oncology. City of Hope experts also will lead discussions on precision medicine and cancer survivorship.

ORAL ABSTRACT AND CLINICAL SCIENCE SYMPOSIUM SESSIONS

4510: Genomic characterization of baseline and post-progression tumors in IMmotion010, a randomized, Phase 3 study of adjuvant (adj) atezolizumab (atezo) vs placebo (pbo) in patients (pts) with high-risk localized renal cell carcinoma (RCC)
Session time: Saturday, May 31, from 4:54 to 5:06 p.m. CT
Session title: Biomarkers in Kidney Cancer: Are We There Yet?
Presenter: Sumanta Kumar Pal, M.D., F.A.S.C.O., City of Hope professor, Department of Medical Oncology & Therapeutics Research

Symptom Science and Palliative Care
Session time: Sunday, June 1, from 9:45 a.m. to 12:45 p.m. CT
Chair and panelist: Tanyanika Phillips, M.D., M.P.H., City of Hope assistant clinical professor, Department of Medical Oncology & Therapeutics Research

Diving Deeper to Inform Hereditary Cancer Risk and Outcomes
Session time: Monday, June 2, from 8 to 9:30 a.m. CT
Chair and panelist: Heather Hampel, M.S., C.G.C., City of Hope professor, Department of Medical Oncology & Therapeutics Research
RAPID ORAL ABSTRACT SESSIONS

1015: Treatment rechallenge after trastuzumab-deruxtecan–related interstitial lung disease: A multi-institution cohort study
Session time: Friday, May 30, from 2:57 to 3:03 p.m. CT
Session title: Breast Cancer – Metastatic
Presenter: Hope S. Rugo, M.D., F.A.S.C.O., City of Hope Women’s Cancers Program director; professor, Department of Medical Oncology & Therapeutics Research
Chair and Moderator: Jose Bazan, M.D., M.S., City of Hope associate professor, Department of Radiation Oncology

6510: MRD negativity after end of induction in the Phase 3 PhALLCON trial: A post hoc analysis
Session time: Friday, May 30, from 1:06 to 1:12 p.m. CT
Session title: Hematologic Malignancies — Leukemia, Myelodysplastic Syndromes, and Allotransplant
Presenter: Ibrahim T. Aldoss, M.D., City of Hope associate professor, Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation
DISCUSSANT FOR ORAL ABSTRACT SESSION

7504, 7505, 7506: New Roads in Myeloma
Session time: Tuesday, June 3, from 11:33 to 11:45 a.m. CT
Session title: Hematologic Malignancies — Plasma Cell Dyscrasia
Discussant: Amrita Krishnan, M.D., City of Hope professor, Department of Hematology & Hematopoietic Cell Transplantation
EDUCATION SESSIONS

An Oncologist’s Approach to Managing Endocrine Toxicities
Session time: Sunday, June 1, from 11:30 to 11:42 a.m. CT
Session title: New Drugs, New Toxicities: Side Effects of New and Emerging Breast Cancer Therapies
Chair and Presenter: Hope S. Rugo, M.D., F.A.S.C.O., City of Hope Women’s Cancers Program director; professor, Department of Medical Oncology & Therapeutics Research

Role of Personalized Interventions Among High-Risk Cancer Survivors to Reduce Morbidity
Session time: Friday, May 30, from 2:57 to 3:09 p.m. CT
Session title: Redefining Success and Survivorship in Pediatric Oncology
Chair and Presenter: Saro H. Armenian, D.O., M.P.H., City of Hope Children’s Cancer Center chair and professor

Integrative Oncology: Defining the Evidence
Session time: Sunday, June 1, from 4:30 to 4:42 p.m. CT
Session title: Real-world Implementation of ASCO (Free ASCO Whitepaper) SIO Guidelines: Pain, Fatigue, Depression
Presenter: Krisstina Gowin, D.O., City of Hope associate professor, Medical Oncology and Therapeutics Research, Department of Supportive Care Medicine
POSTER SESSIONS

1070 / Poster Board 49: Elacestrant (Ela) combinations with ribociclib (Ribo) and everolimus (Eve) in patients (pts) with ER+/HER2- locally advanced or metastatic breast cancer (mBC): Update from ELEVATE, a Phase (Ph) 1b/2, open-label, umbrella study
Session time: Monday, June 2, from 9 a.m. to noon CT
Session title: Breast Cancer — Metastatic
Presenter: Hope S. Rugo, M.D., F.A.S.C.O., City of Hope Women’s Cancers Program director; professor, Department of Medical Oncology & Therapeutics Research

LBA 4175 / Poster Board 465: NAPOLI 3, a Phase 3 study of NALIRIFOX in patients with metastatic pancreatic ductal adenocarcinoma (mPDAC): Final overall survival (OS) analysis and characteristics of the long-term survivors
Session time: Saturday, May 31, from 9 a.m. to noon CT
Session title: Gastrointestinal Cancer — Gastroesophageal, Pancreatic, and Hepatobiliary
Presenter: Vincent Chung, M.D., City of Hope professor, Department of Medical Oncology & Therapeutics Research

3553 / Poster Board 222: Vilastobart (XTX101), a tumor-activated, Fc-enhanced anti–CTLA-4 monoclonal antibody, in combination with atezolizumab in patients with MSS CRC
Session time: Saturday, May 31, from 9 a.m. to noon CT
Session title: Gastrointestinal Cancer — Colorectal and Anal
Presenter: Marwan Fakih, M.D., City of Hope professor, Department of Medical Oncology & Therapeutics Research

5041 / Poster Board 240: Cardiovascular (CV) event risk in patients (pts) with metastatic castration-resistant prostate cancer (mCRPC) treated with enzalutamide (ENZA) or abiraterone acetate (AA) in the United States (US)
Session time: Monday, June 2, from 9 a.m. to noon CT
Session title: Genitourinary Cancer — Prostate, Testicular, and Penile
Presenter: Alan H. Bryce, M.D., City of Hope professor, Department of Medical Oncology & Therapeutics Research

4540 / Poster Board 340: Efficacy of second line (2L) treatment with tivozanib (Tivo) as monotherapy or with nivolumab (Nivo) in patients (pts) with metastatic renal cell carcinoma (mRCC) previously treated with an immune checkpoint inhibitor (ICI) combination of ipilimumab (Ipi)/Nivo or vascular endothelial growth factor receptor-tyrosine kinase inhibitor (VEGFR-TKI)/ICI in the Phase 3 TiNivo-2 study
Session time: Monday, June 2, from 9 a.m. to noon CT
Session title: Genitourinary Cancer — Kidney and Bladder
Presenter: Alexander Chehrazi-Raffle, M.D., City of Hope assistant professor, Department of Medical Oncology & Therapeutics Research

4550 / Poster Board 350: Long-term clinical outcomes with nivolumab/ipilimumab with or without Clostridium butyricum MIYAIRI588 in metastatic renal cell carcinoma (mRCC): A randomized Phase Ib clinical trial
Session time: Monday, June 2, from 9 a.m. to noon CT
Session title: Genitourinary Cancer — Kidney and Bladder
Presenter: Miguel Zugman, M.D., City of Hope postdoctoral medical oncology research fellow
RECOGNITION

Both Lorna Rodriguez-Rodriguez, M.D., Ph.D., City of Hope professor, Division of Gynecologic Oncology, Department of Surgery, and Stacy W. Gray, M.D., A.M., City of Hope professor, Medical Oncology & Therapeutics Research, Population Sciences, were inducted as fellows of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (FASCO), a distinction that honors people who have encouraged others to conquer cancer through research, education and promotion of the highest-quality patient care.

On May 7, 2025 Immunocore Holdings plc (Nasdaq: IMCR) ("Immunocore" or the "Company"), a commercial-stage biotechnology company pioneering and delivering transformative immunomodulating medicines to radically improve outcomes for patients with cancer, infectious diseases and autoimmune diseases, reported its financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, Immunocore, MAY 7, 2025, View Source [SID1234652641]).

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"We are thrilled to have achieved strong revenue performance in Q1, marked by year-over-year growth of 33%. This reflects our unwavering dedication to making KIMMTRAK accessible to patients who need it," said Bahija Jallal, Chief Executive Officer of Immunocore. "In R&D, we continue to be laser-focused on execution in our oncology franchise with three ongoing Phase 3 trials and a promising early pipeline. We are also excited to have presented the initial MAD data at CROI from our ongoing HIV trial, which, together with our progress in autoimmune, underscores the breadth of our platform."

First Quarter 2025 Highlights (including post-period)

Financial Results
Total net product revenue (or "net sales") arising from the sales of KIMMTRAK (tebentafusp) was $93.9 million in the first quarter of 2025, an increase of 33% over the first quarter of 2024, of which $56.6 million was generated in the United States, $32.8 million in Europe and $4.5 million in international regions.

Research & development expenses for the three months ended March 31, 2025, were $56.5 million, compared to $57.5 million for the same period in 2024. Selling, general and administrative (SG&A) expenses for the three months ended March 31, 2025, were $40.2 million, compared to $39.3 million in the same period in 2024.

Net income for the first quarter of 2025 was $5.0 million compared to a net loss of $24.4 million in the same period in 2024. The first quarter basic and diluted income/(loss) per share was $0.10, compared to $(0.49) for the first quarter of 2024.

Cash, cash equivalents and marketable securities at March 31, 2025, were $837.0 million.

KIMMTRAK
The Company’s lead product, KIMMTRAK (tebentafusp), is approved in 39 countries and has been launched in 26 countries globally to date for HLA-A*02:01 positive people with metastatic uveal melanoma (mUM). KIMMTRAK continues to be the standard of care in most markets where it is launched.

The Company sees three key growth areas for KIMMTRAK including continued global expansion in mUM, the potential expansion into 2L+ advanced cutaneous melanoma (CM), and the potential expansion into adjuvant uveal melanoma.

Metastatic uveal melanoma

In the first quarter of 2025, KIMMTRAK net product sales were $93.9 million.
Now launched in two additional countries for a total of 26 globally.
13% year-over-year growth in the United States, with demand continuing to grow as outreach extends into the community setting.
Growth in Europe and in international regions driven by increased demand, new country launches, and completion of price negotiations in France and Germany.
2L+ advanced cutaneous melanoma

The Company is currently enrolling the TEBE-AM registrational Phase 3 trial and expects to complete enrollment in the first half of 2026.
The Phase 3 trial is enrolling three arms: tebentafusp monotherapy, tebentafusp in combination with pembrolizumab, and a control (investigator’s choice of therapy including options such as investigator’s choice of clinical trials, chemotherapy, or retreatment with anti-PD1 or BRAF therapy).
There is great unmet need in second- and later-line cutaneous melanoma, with no therapy having shown an Overall Survival (OS) improvement post checkpoint inhibitors in a randomized clinical trial. The Company estimates that there is a potential to address up to 4,000 previously treated advanced CM patients.

Adjuvant uveal (or ocular) melanoma

The European Organisation for Research and Treatment of Cancer (EORTC) is enrolling patients in the Phase 3 Adjuvant Trial in Ocular Melanoma (ATOM).
The Company estimates that the HLA-A*02:01 high-risk adjuvant uveal melanoma patient population could be up to 1,200 patients.
PRAME portfolio
Brenetafusp is the Company’s lead PRAME-A02 ImmTAC bispecific candidate. Brenetafusp is being evaluated in combination with nivolumab in a Phase 3 registrational trial (PRISM-MEL-301) in patients with first-line, advanced cutaneous melanoma, and in a Phase 1/2 clinical trial as monotherapy and in combination across multiple tumor types, including ovarian cancer and non-small cell lung cancer (NSCLC).

PRISM-MEL-301 – First PRAME Phase 3 clinical trial with brenetafusp in first-line advanced cutaneous melanoma

The Company is enrolling patients in the registrational Phase 3 clinical trial evaluating brenetafusp + nivolumab versus a control arm of either nivolumab or nivolumab + relatlimab for HLA-A*02:01 positive patients with first-line, advanced or metastatic cutaneous melanoma.
The trial is currently randomizing to three arms: two brenetafusp dose regimens (40 mcg and 160 mcg) and a control arm. The Company is on track for selection of the go-forward brenetafusp dose in the second half of 2025; this analysis will be conducted by an IDMC.
Despite approved therapies, there remains a need for improved progression-free survival and overall survival, and there is the potential to address an estimated 10,000 patients.

Phase 1/2 clinical trial of brenetafusp in multiple solid tumors

The Company continues to evaluate brenetafusp in a Phase 1/2 trial in combination with non-platinum chemotherapies in platinum-resistant ovarian cancer (PROC) and with bevacizumab or with platinum chemotherapy in earlier lines of platinum-sensitive ovarian cancer (PSOC). In the same trial, the Company continues signal detection in metastatic non-small cell lung cancer (NSCLC) cohorts, including brenetafusp in combination with docetaxel and with osimertinib in earlier-line NSCLC.
The Company estimates that, across all solid tumors, the annual number of patients worldwide who test positive for HLA-A*02:01 and can potentially benefit from this program is up to 150,000.
IMC-P115C (PRAME-A02 Half-Life Extended) & IMC-T119C (PRAME-A24)

The Company is enrolling patients in the Phase 1 dose escalation trial, in multiple solid tumors, with IMC-P115C.
IMC-P115C is the Company’s first half-life extended ImmTAC therapy – targeting the same PRAME peptide and with the same CD3 effector and TCR specificity as brenetafusp. It is designed to improve patient convenience by reducing the frequency of treatment administration.
IMC-R117C (PIWIL1) for colorectal and other gastrointestinal cancers

The Company is enrolling patients in the Phase 1/2 dose escalation trial evaluating IMC-R117C in HLA-A*02:01 positive patients with advanced solid tumors, including colorectal cancer, as a single agent and in combination with standards of care.
PIWIL1 is believed to play a role in tumor progression and is expressed across a range of tumors, including colorectal cancer.
ImmTAV candidates for a functional cure in infectious diseases
The Company’s bispecific TCR technology platform has the potential to offer a new approach for the treatment of certain chronic infections and aims to eliminate evidence of remaining virus in circulation after the patient stops taking medication – known as a "functional cure." Two investigational candidates are in Phase 1 or Phase 1/2 trials for people living with human immunodeficiency virus (HIV) and people with chronic hepatitis B infection (HBV).

Phase 1/2 trial of IMC-M113V (Gag-A02) for people living with HIV

The Company presented data from the initial multiple ascending dose (MAD) portion of the Phase 1/2 dose escalation trial, including 16 people living with HIV (PLWH), at the 2025 Conference on Retroviruses and Opportunistic Infections (CROI).
All doses were well tolerated and no serious adverse events or dose limiting toxicities were observed.
In the 15 evaluable PLWH, delayed viral rebound and/or viremia control at any point during the analytical treatment interruption (ATI) was observed in 0 of 5 PLWH at 60 mcg, 1 of 5 at 120 mcg, and 2 of 5 at 300 mcg.
The 3 PLWH with evidence of viral control had a viral load of approximately 200 copies/mL at week 8. The historical rate for this observation is 5%1. Furthermore, 2 of these 3 PLWH remained off ART for the entire 12-week ATI period that was pre-specified in the protocol.
Patient enrollment continues at higher doses in the multiple ascending dose part of the Phase 1/2 clinical trial to identify a safe and tolerable dose.

Phase 1 trial of IMC-I109V (Envelope-A02) for people living with HBV or HBV-positive hepatocellular carcinoma

The Company plans to report data from the single ascending dose portion of the trial in the second half of 2025.

Tissue-specific down modulation of the immune system for autoimmune diseases
The key differentiator of the ImmTAAI platform is tissue-specific, down modulation of the immune system, as the candidates suppress pathogenic T cells via PD1 receptor agonism only when tethered to the target tissue.

IMC-S118AI (PPI-A02) for type 1 diabetes

The Company is on track to file a CTA or investigational new drug application (IND) for IMC-S118AI (PPI x PD1) in the second half of 2025.
IMC-S118AI is targeted specifically to the pancreatic beta-cell and intended as a disease-modifying treatment in type 1 diabetes. IMC-S118AI recognizes a peptide from pre-pro-insulin protein that is presented by HLA-A02 on beta cells and has a PD1 agonist effector arm.
IMC-U120AI (CD1a) for atopic dermatitis as the initial indication – first universal program

The Company plans to file a CTA/IND for IMC-U120AI (CD1a x PD1) in 2026.
IMC-U120AI is a non-HLA-restricted (i.e. universal for all populations) CD1a-tethered PD1 agonist ImmTAAI therapy. IMC-U120AI has a dual mechanism of action: blocking CD1a (which presents lipids) from activating CD1a-specific T cells and preventing HLA Class I/II (which presents peptides) from activating T cells via PD1 agonism on the T cell.
Financial Results
Basic and diluted income per share was $0.10 for the quarter ended March 31, 2025, as compared to a basic and diluted loss per share of ($0.49) for the same period in 2024. Net income for the quarter ended March 31, 2025, was $5.0 million, as compared to a net loss of $(24.4) million for the same period in 2024.

For the first quarter ended March 31, 2025, the Company generated net product sales of $93.9 million compared to $70.3 million for the same period in 2024, due to sales of KIMMTRAK, of which $56.6 million was in the United States, $32.8 million (including one-time favorable revenue adjustments recorded upon completion of price negotiations in France and Germany of $6.0M) was in Europe, and $4.5 million was in the international regions. The increase in net product sales was due to global country expansion and increased sales volume in the United States, as we continued our commercialization efforts.

For the first quarter ended March 31, 2025, research and development (R&D) expenses were $56.5 million compared to $57.5 million for the same period in 2024.

For the quarter ended March 31, 2025, SG&A expenses were $40.2 million compared to $39.3 million for the same period in 2024.

Cash, cash equivalents and marketable securities were $837.0 million as of March 31, 2025, as compared to $820.4 million as of December 31, 2024.