On May 13, 2025 INOVIO (NASDAQ: INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, reported its financial results for the first quarter of 2025 and provided an update on recent company developments (Press release, Inovio, MAY 13, 2025, View Source [SID1234652969]).

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"I’m pleased to confirm that we remain on track to submit our BLA for INO-3107 this year. As previously stated, our goal is to begin rolling submission in mid-2025, complete the submission in the second half of 2025 and receive file acceptance from the FDA by year end. If we receive priority review, it could allow for a PDUFA date in mid-2026," said Dr. Jacqueline Shea, INOVIO’s President and Chief Executive Officer. "We continue to focus our efforts and resources toward bringing this important product candidate to patients eager for a non-surgical therapeutic option to treat this devastating disease. Based on market research, we believe INO-3107 could be the preferred product for patients and providers, if approved. Beyond INO-3107, we are excited about the recently announced promising data from a proof-of-concept Phase 1 clinical trial with our next-generation DMAb technology, as well as the potential of our entire DNA medicines pipeline."

Operational Highlights

INO-3107 – Recurrent Respiratory Papillomatosis (RRP)
The DV testing for INO-3107 has been initiated and is anticipated to be completed in the first half of 2025. INOVIO plans to begin rolling submission of the BLA in mid-2025 under FDA’s accelerated approval program, subject to FDA concurrence, with the goal of completing the submission in the second half of 2025 and receiving FDA acceptance of the submission by the end of the year. FDA has previously awarded breakthrough therapy designation for INO-3107 and INOVIO plans to request priority review of its BLA, which if granted would allow for an FDA approval decision (PDUFA date) in mid-2026.

INOVIO is conducting ongoing market research with physicians, patients and payors to support its commercial readiness plans. Additionally, clinical and immunology data from a Phase 1/2 trial was published in the peer-reviewed scientific journal Nature Communications in February 2025 and longer-term follow up data was submitted to another peer-reviewed journal for publication. INOVIO has presented key efficacy, safety, immunological and durability data from the Phase 1/2 trial at a number of scientific conferences, including the following:

National HPV Conference (April 15)
World Vaccine Congress (April 21)
Festival of Biologics (April 23)
European Laryngological Society (ELS) Annual Congress (May 9)
American Society of Gene and Cell Therapy (May 13)
On May 14th INOVIO will also be presenting at the American Broncho-Esophagological Association Combined Otolaryngology Spring Meeting (ABEA/COSM), the largest US national meeting for otolaryngologists, the specialist physicians who treat the majority of RRP patients.

Available abstracts are posted to INOVIO’s website following presentations.

DNA-Encoded Monoclonal Antibodies (DMAbs)
In the first quarter, INOVIO and its collaborators announced top-line interim results from an ongoing Phase 1 proof-of-concept trial evaluating its DMAb technology. Additional data from this trial will be presented at the annual meeting of the American Society of Gene and Cell Therapy in May. The data is also anticipated to be published in a peer-reviewed journal. As previously announced, the interim results are currently available in preprint form on Research Square. In the trial, which used monoclonal antibodies against COVID-19 as proof-of-concept targets, 100% (24/24) of participants who reached week 72 maintained biologically relevant levels of DMAbs, confirming the durability of in vivo antibody production. Notably, no participant developed anti-drug antibodies, a common challenge observed in other gene-based delivery platforms, such as adeno-associated virus-mediated antibody expression.

First Quarter 2025 Financial Results

Research and Development (R&D) Expenses: R&D expenses for the three months ended March 31, 2025, decreased to $16.1 million from $20.9 million for the same period in 2024. The decrease was primarily the result of lower drug manufacturing and immunology expenses related to INO-3107, lower contract labor expenses and lower expensed inventory, partially offset by higher engineering professional and outside services related to our device development, among other variances.

General and Administrative (G&A) Expenses: G&A expenses decreased to $9.0 million for the three months ended March 31, 2025 from $10.6 million for the same period in 2024. The decrease was primarily related to a decrease in legal expenses and employee and consultant stock-based compensation, among other variances.

Total Operating Expenses: Total operating expenses decreased to $25.1 million for the three months ended March 31, 2025 from $31.5 million for the same period in 2024.

Net Loss: Net loss for the three months ended March 31, 2025 decreased to $19.7 million, or $0.51 per basic and diluted share, from a net loss of $30.5 million, or $1.31 per basic and diluted share, for the three months ended March 31, 2024.

Shares Outstanding: As of March 31, 2025, INOVIO had 36.7 million common shares outstanding and 51.3 million common shares outstanding on a fully diluted basis, after giving effect to the exercise, vesting, and conversion, as applicable, of its outstanding common stock warrants, including pre-funded warrants and stock options, restricted stock units and convertible preferred stock.

Cash, Cash Equivalents and Short-term Investments: As of March 31, 2025, cash, cash equivalents and short-term investments were $68.4 million, compared to $94.1 million as of December 31, 2024.
INOVIO’s balance sheet and statement of operations are provided below. Additional information is included in INOVIO’s quarterly report on Form 10-Q for the quarter ended March 31, 2025, which can be accessed at: View Source

Cash Guidance
INOVIO estimates its current cash, cash equivalents and short-term investments balances to support the company’s operations into the first quarter of 2026. This projection includes an operational net cash burn estimate of approximately $22 million for the second quarter of 2025. These projections do not include any further capital-raising activities that INOVIO may undertake.

Conference Call / Webcast Information
INOVIO’s management will host a live conference call and webcast with slides at 4:30 p.m. ET today to discuss INOVIO’s financial results and provide a general business update. The live webcast and replay may be accessed by visiting INOVIO’s website at View Source

On May 13, 2025 Immatics N.V. (NASDAQ: IMTX, "Immatics" or the "Company"), a clinical-stage biopharmaceutical company active in the discovery and development of T cell-redirecting cancer immunotherapies, reported a business update and announced financial results for the quarter ended March 31, 2025 (Press release, Immatics, MAY 13, 2025, View Source [SID1234652968]).

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"Our focus in the first quarter of 2025 was led by the execution of our SUPRAME Phase 3 clinical trial in melanoma as well as our other clinical-stage PRAME product candidates," said Harpreet Singh, Ph.D., CEO and Co-Founder of Immatics. "At the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting, we will present another Phase 1b clinical update on our PRAME cell therapy, IMA203, in melanoma with substantially longer follow-up. We also look forward to providing clinical trial updates for our cell therapy and bispecific programs later this year, highlighting the potential of our therapies in and beyond melanoma. We maintain a strong cash position, enabling us to rapidly advance the development of all our clinical programs, with a specific focus on progressing IMA203 toward commercialization and delivering this highly differentiated PRAME therapy to cutaneous and uveal melanoma patients with unmet medical needs as quickly as possible."

First Quarter 2025 and Subsequent Company Progress

PRAME Programs

IMA203 PRAME Cell Therapy
IMA203 is Immatics’ lead PRAME cell therapy, currently being evaluated in a Phase 3 trial (SUPRAME) in patients with previously treated advanced melanoma. IMA203 has the potential to become the first PRAME therapy to enter the market. In parallel, Immatics is preparing its in-house, state-of-the-art cell therapy manufacturing facility to serve its planned commercial supply. As part of maximizing the PRAME cell therapy opportunity, Immatics plans to expand IMA203 into uveal melanoma through the ongoing Phase 1b clinical trial. The current addressable patient population of PRAME/HLA-A*02:01-positive 2L unresectable or metastatic cutaneous melanoma in the US and EU52 is ~7,300 plus ~1,300 uveal melanoma patients in the US and EU5.

Phase 3 trial, SUPRAME, for IMA203 in previously treated, advanced cutaneous melanoma

Based on the positive Phase 1b clinical data, Immatics has advanced its PRAME cell therapy, IMA203, into a randomized-controlled Phase 3 clinical trial, SUPRAME, evaluating the efficacy, safety and tolerability of IMA203 TCR T-cell therapy vs. investigator’s choice of treatment in patients with unresectable or metastatic cutaneous melanoma who have received prior treatment with a checkpoint inhibitor.
Primary endpoint for seeking full approval will be blinded independent central review ("BICR")-assessed (RECIST v1.1) progression-free survival (PFS). Secondary endpoints for the trial include objective response rate (ORR), safety, duration of response (DOR), overall survival (OS) and patient-reported outcomes.
The trial will be conducted internationally with approximately 50 sites in the US and Europe.
Patient enrollment and randomization for the trial was initiated in early 2025 and is expected to be completed in 2026. In April 2025, Immatics received regulatory approval from the German regulatory authority, Paul-Ehrlich-Institute (PEI), to commence the IMA203 SUPRAME Phase 3 trial in Germany.
A pre-specified interim data analysis will be triggered upon the occurrence of a defined number of events for PFS (progressive disease or death)3, anticipated to occur after approximately 200 patients. Immatics aims to submit a Biologics License Application (BLA) in 1Q 2027 for full approval.
IMA203 PRAME cell therapy development is supported by the FDA RMAT designation. Advantages of the RMAT designation (which includes all benefits of Breakthrough Therapy designation) include potential priority review of the BLA and frequent interactions with the US FDA as an opportunity to expedite development and review.
A trial-in-progress poster on SUPRAME will be presented in a poster presentation by the SUPRAME lead principal investigator, Jason Luke, MD, FACP, FASCO, at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting on June 2, 2025.

Phase 1b trial for IMA203 PRAME cell therapy in solid tumors with a focus on uveal melanoma

In addition to cutaneous melanoma, Immatics intends to expand the IMA203 opportunity to treat uveal melanoma patients and will continue to evaluate IMA203 in this patient population through the ongoing trial.
Updated data from the Phase 1b trial of IMA203 in metastatic melanoma with substantially longer follow-up compared to the last presentation in October 2024, and including data from additional uveal melanoma patients enrolled since then, will be highlighted by Martin Wermke, MD, in an oral presentation at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting on May 31, 2025.
In April 2025, Nature Medicine published a manuscript covering prior clinical results on IMA203. The publication includes data from 40 heavily pretreated patients with PRAME cancers, mostly treated during the Phase 1a dose escalation part of the trial.

Cell therapy manufacturing capabilities

The IMA203 PRAME cell therapy products are manufactured from a patient’s leukapheresis (with no surgery required) within 7-8 days, followed by 7-day QC release testing at >95% success rate4 to achieve the target dose (1-10×109 TCR T cells).
Immatics’ proprietary manufacturing process, timeline, capabilities and facility support late-stage clinical development and commercial cell therapy supply.
IMA203CD8 PRAME Cell Therapy (GEN2)
IMA203CD8 is the Company’s second-generation cell therapy product candidate targeting PRAME. Given its pharmacology profile, once the target dose is reached, the Company intends to pursue the clinical development of this product in multiple PRAME cancers, starting with gynecologic cancers.

Clinical data demonstrated enhanced pharmacology of IMA203CD8, which opens the possibility of addressing hard-to-treat solid tumor indications with both high- and medium-level PRAME copy numbers, such as ovarian cancer, uterine cancer, squamous non-small cell lung carcinoma, triple negative breast cancer and others.
Phase 1a dose escalation in solid tumors is ongoing to evaluate higher doses of IMA203CD8 with and without IL-2. As of today, patients are being treated with up to ~8 billion total GEN2 TCR T cells.
The next clinical trial update, which will report on the continued dose escalation in multiple PRAME cancers, including ovarian cancer patients treated at relevant doses, is planned in 2025.

IMA402 PRAME Bispecific

To expand the PRAME opportunity to additional solid cancer types and earlier lines of treatment, the Company is developing its half-life extended TCR Bispecific, IMA402. Upon delivering clinical proof-of-concept ("PoC") in last-line melanoma, Immatics plans to explore its potential in gynecologic cancers, NSCLC, breast cancer, and other solid tumor indications as well as earlier treatment lines of solid cancers, such as first-line (1L) cutaneous melanoma.

First clinical data from the early Phase 1a dose escalation trial demonstrated initial signs of dose-dependent and PRAME target expression-dependent clinical activity.
Phase 1a dose escalation at higher dose levels to determine the optimal therapeutic dose is advancing and currently ongoing at dose level 11 (12 mg).
The next Phase 1a clinical trial update with clinical data at relevant dose levels in second-line or later (2L) melanoma is planned in 2025.

Combination of IMA203 PRAME Cell Therapy and PRAME Adaptive Immune Modulating Therapy

In February 2025, the FDA granted IND clearance for a Phase 1 trial evaluating Immatics’ IMA203 PRAME cell therapy in combination with Moderna’s PRAME adaptive immune modulating therapy. The first-in-human, Phase 1a/1b trial is a multicenter, open-label, dose escalation/de-escalation (adaptive design) trial evaluating the safety, tolerability and efficacy of the combination therapy in an estimated 15 patients with advanced or recurrent cutaneous melanoma and synovial sarcoma. Immatics is responsible for conducting the Phase 1 trial. Each party retains full ownership of its investigational PRAME compound, and the parties will fund the clinical study on a cost sharing basis. In November 2024, Immatics presented preclinical proof-of-concept data at SITC (Free SITC Whitepaper) supporting this combination.

Other Programs

IMA401 MAGEA4/8 Bispecific
Immatics is further harnessing the potential of its proprietary bispecific platform to develop innovative therapeutics and unlock more cancer types. The Company’s half-life extended TCR Bispecific, IMA401 targeting MAGEA4/8, is progressing through a Phase 1 trial in patients with late-stage NSCLC, head & neck cancer, bladder cancer and other solid tumor indications, with the primary goal of developing this product candidate in earlier treatment lines.

Clinical proof-of-concept data from the Phase 1a dose escalation trial showed initial anti-tumor activity in multiple tumor types, including durable confirmed objective responses, a manageable tolerability profile and a half-life of 14+ days, which supported the switch to q2w dosing (once every two weeks).
The Phase 1a trial is ongoing and the Company continues to focus enrollment on indications with high MAGEA4/8 target expression, such as lung and head and neck cancer.
Dose refinement for IMA401 as monotherapy and in combination with a checkpoint inhibitor is ongoing. Through the combination, Immatics aims to generate relevant clinical data to position IMA401 as a combination therapy in earlier treatment lines.
The next update on IMA401 Phase 1a data, with a focus on head and neck cancer, is expected in 2025, and the Company plans to share data with a focus on non-small cell lung carcinoma in 2026.
Moderna Collaboration
Immatics generated regulatory support data for one of Moderna’s mRNA product candidates that leveraged Immatics’ XPRESIDENT and its bioinformatics and AI platform XCUBE. Pursuant to the Collaboration Agreement under the Database/Vaccine Program, Immatics received a milestone payment triggered by the initiation of the first Phase 1 clinical trial for the Moderna product candidate.

First Quarter 2025 Financial Results

Cash Position: Cash and cash equivalents as well as other financial assets total $588.1 million1 (€543.8 million) as of March 31, 2025, compared to $653.8 million1 (€604.5 million) as of December 31, 2024. The decrease is mainly due to ongoing research and development activities and includes unrealized foreign exchange translational losses of $14.2 million1 (€13.1 million).

Revenue: Total revenue, consisting of revenue from collaboration agreements, was $20.1 million1 (€18.6 million) for the three months ended March 31, 2025, compared to $32.8 million1 (€30.3 million) for the three months ended March 31, 2024. The decrease is mainly the result of the one-time revenue associated with the termination of the Genmab collaboration during the three months ended March 31, 2024.

Research and Development Expenses: R&D expenses were $45.3 million1 (€41.9 million) for the three months ended March 31, 2025, compared to $34.7 million1 (€32.1 million) for the three months ended March 31, 2024. The increase mainly resulted from costs associated with the advancement of the product candidates in clinical trials.

General and Administrative Expenses: G&A expenses were $13.1 million1 (€12.1 million) for the three months ended March 31, 2025, compared to $12.5 million1 (€11.6 million) for the three months ended March 31, 2024.

Net Profit and Loss: Net loss was $43.2 million1 (€39.9 million) for the three months ended March 31, 2025, compared to a net loss of $2.4 million1 (€2.2 million) for the three months ended March 31, 2024. The increase mainly resulted from lower revenue recognized and unrealized non-cash foreign exchange rate losses.

Full financial statements can be found in our Report on 6-K filed with the Securities and Exchange Commission (SEC) on May 13, 2025, and published on the SEC website under www.sec.gov.

Upcoming Investor Conferences

Bank of America Healthcare Conference, Las Vegas (NV) – May 13 – 15, 2025
Jefferies Global Healthcare Conference, New York (NY) – June 3 – 5, 2025
Cantor Global Healthcare Conference, New York (NY) – September 3 – 5, 2025

To see the full list of events and presentations, visit View Source

On May 13, 2025 Gate2Brain, a Barcelona-based biotech innovator in drug delivery across biological barriers, reported that its lead candidate, G2B-002, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) (Press release, Gate2Brain, MAY 13, 2025, View Source [SID1234652966]). This follows the European Medicines Agency’s ODD approval in late 2024 and highlights G2B-002’s potential in treating rare and aggressive pediatric and adult brain tumors.

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G2B-002 is designed to target diffuse intrinsic pontine glioma (DIPG) and pediatric glioblastoma (pGBM), two of the most lethal childhood brain cancers with very limited treatment options. By leveraging Gate2Brain’s proprietary peptide-based delivery technology, G2B-002 enhances drug penetration across the blood–brain barrier, aiming to improve efficacy while reducing systemic side effects.

Endowed with this FDA designation, G2B-002 benefits from enhanced regulatory support and strategic momentum, smoothing its transition from advanced preclinical development into clinical trial phase.

"Receiving ODD from both the EMA and US FDA reaffirms our conviction in G2B-002’s potential to address critical unmet needs in pediatric oncology," said Meritxell Teixidó, CEO & CSO of Gate2Brain. "This dual recognition brings us a step closer to clinical trials and, ultimately, to offering new hope for young patients and their families."

Founded in 2020 as a spin-off from IRB Barcelona, University of Barcelona, and Sant Joan de Déu Pediatric Hospital, Gate2Brain remains dedicated to transforming the treatment landscape for pediatric brain tumors through innovative delivery solutions.

The company has received support from institutions including Fundación Botín (Mind the Gap), Banco Sabadell (BStartup Health), CDTI (Neotec, Multipaís), the European Innovation Council (EIC Accelerator), Startup Capital d’ACCIÓ and CaixaResearch Consolidate de la "Fundació la Caixa".

On May 13, 2025 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, reported business highlights and financial results for the first quarter ended March 31, 2025 (Press release, Fate Therapeutics, MAY 13, 2025, View Source [SID1234652965]).

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"We continue to be pleased with the initial clinical profile of our FT819 off-the-shelf CAR T-cell program in patients with moderate-to-severe SLE, and we look forward to sharing new clinical data from our ongoing FT819 Phase 1 study at the EULAR Conference in June," said Bob Valamehr, Ph.D. MBA, President and Chief Executive Officer of Fate Therapeutics. "Our primary focus remains on driving patient enrollment and achieving therapeutic differentiation in SLE, including administration of FT819 with fludarabine-free conditioning and as an add-on to maintenance therapy without conditioning. We plan to work closely with the FDA under our FT819 RMAT designation to align on a registrational pathway for FT819 in SLE, and we have now initiated regulatory submissions in Europe to expand the geographic reach of the program for lupus patients. The expansion of patient population and reach is well supported by the ability to produce FT819 in large quantities from a master cell bank, ensuring consistent on-demand product supply for a large number of patients."

FT819 iPSC-derived 1XX CAR T-cell Program

Phase 1 Study Ongoing using Flu-free Conditioning Regimen for SLE. The Company’s ongoing multi-center, Phase 1 clinical trial of FT819 for moderate-to-severe systemic lupus erythematosus (SLE) (NCT06308978) is designed to evaluate the safety, pharmacokinetics, and efficacy of a single dose of FT819 following a fludarabine (flu)-free conditioning regimen, consisting of either bendamustine alone or cyclophosphamide alone. The Company is currently enrolling patients at two dose levels – a single dose of 360 million cells and a single dose of 900 million cells – with the intent of identifying a recommended dose for later-stage development. In addition, the Company is assessing the safety, pharmacokinetics, and anti-B cell activity of a single dose of FT819 at 360 million cells as an add-on to maintenance therapy without conditioning chemotherapy. The Company plans to present new clinical data from its FT819 Phase 1 study during an oral session at the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress in Barcelona, Spain on June 11.
RMAT Designation Received from the FDA for SLE. In April 2025, the Company was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for FT819 to treat moderate-to-severe SLE. The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases or conditions. The Company’s RMAT application included initial clinical safety and activity data from patients treated with FT819 in its ongoing multi-center, Phase 1 clinical trial. The Company plans to pursue differentiated treatment approaches, including treatment of patients in community centers without hospitalization, and novel registrational strategies with the FDA under its RMAT designation.
Expanded Phase 1 Study to Include Multiple Additional B Cell-mediated Autoimmune Diseases. In December 2024, the Company reached agreement with the FDA to allow for clinical investigation of multiple B cell-mediated autoimmune diseases under its current Phase 1 clinical trial of FT819. The Company has submitted an amended clinical protocol to the FDA that enables the conduct of independent dose-expansion cohorts for anti-neutrophilic cytoplasmic antibody-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). The Company plans to initiate dose-expansion cohorts in each of AAV, IIM, and SSc in 2025.
FT825 / ONO-8250 iPSC-derived CAR T-cell Program

Phase 1 Study Ongoing for Advanced Solid Tumors. Under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono), the Company is conducting a multi-center, Phase 1 study to assess the safety, pharmacokinetics, and activity of FT825 / ONO-8250, a multiplexed-engineered CAR T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2), in patients with advanced solid tumors (NCT06241456). Dose escalation is currently ongoing, with each patient administered conditioning chemotherapy and a single dose of FT825 / ONO-8250 either as monotherapy or in combination with epidermal growth factor receptor (EGFR)-targeted monoclonal antibody therapy. FT825 / ONO-8250 has demonstrated a favorable safety profile with no dose-limiting toxicities (DLTs) to date.
Next-generation iPSC-derived CAR T-cell Programs

FT836 MICA/B-targeted CAR T-cell Program. FT836 is the Company’s multiplexed-engineered CAR T-cell product candidate uniquely targeting major histocompatibility complex (MHC) proteins A (MICA) and B (MICB). The expression of MICA/B cell-surface proteins is induced by cellular stress or malignant transformation and is detectable across many types of cancer cells with limited expression on healthy tissue. At the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting being held in New Orleans on May 13-17, the Company plans to present preclinical data showing that FT836 exerted potent and durable anti-tumor activity in vivo across a broad array of solid tumors. FT836 is the Company’s first product candidate to incorporate its novel Sword & Shield technology that couples its novel Alloimmune Defense Receptor (ADR) technology with the complete knock-out of CD58 (CD58KO), which is uniquely designed to both target and evade host alloreactive immune cells and to reduce or eliminate the need for administration of conditioning chemotherapy to patients receiving cell therapies. In January 2025, the Company secured a $4 million award from the California Institute of Regenerative Medicine (CIRM) to support IND-enabling activities for FT836.
FT839 Dual CAR T-cell Program. FT839 is the Company’s dual CAR T-cell product candidate that incorporates its novel Sword & Shield technology and is designed to express two unique CARs: a first CAR targeting CD19+ B cells, and a second CAR targeting additional disease-causing cells. At the ASGCT (Free ASGCT Whitepaper) Annual Meeting in May, the Company plans to present preclinical data demonstrating iPSC-derived CAR T cells targeting CD19 and the cell-surface glycoprotein CD38 specifically eliminated a variety of malignant cell types, including CD19+ lymphoma and CD38+ multiple myeloma cell lines. In addition, using unmatched peripheral blood mononuclear cells sourced from a patient with SLE, dual CAR T cells showed robust eradication of aberrant CD19+ B cells, CD38+ plasma cells, and CD38+ activated T cells.
FT522 iPSC-derived CAR NK Cell Program

New Phase 1 Translational Data to be Presented at ASGCT (Free ASGCT Whitepaper). FT522 is the Company’s off-the-shelf CAR NK cell product candidate and its first to incorporate Alloimmune Defense Receptor (ADR) technology. The FDA has allowed the Company’s Investigational New Drug (IND) application to assess the safety, pharmacokinetics, and activity of FT522 across a basket of B cell-mediated autoimmune diseases. The Company is currently evaluating opportunities and timelines for the clinical development of FT522 in autoimmunity without administration of conditioning chemotherapy. At the ASGCT (Free ASGCT Whitepaper) Annual Meeting in May, the Company plans to present new clinical and translational data from its multi-center, Phase 1 clinical trial of FT522 in patients with relapsed / refractory B-cell lymphoma (BCL) (NCT05950334), where initial translational data demonstrated the potential of FT522 to persist and function in the presence of an unmatched, fully-intact immune system.
First Quarter 2025 Financial Results

Cash & Investment Position: Cash, cash equivalents, and investments as of March 31, 2025 were $272.7 million.
Total Revenue: Revenue was $1.6 million for the first quarter of 2025, which was derived from the conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under the Company’s collaboration with Ono Pharmaceutical.
Total Operating Expenses: Total operating expenses were $42.9 million for the first quarter of 2025, including research and development expenses of $29.1 million and general and administrative expenses of $13.8 million. Such amount included $7.4 million of non-cash stock-based compensation expense.
Shares Outstanding: As of March 31, 2025, common shares outstanding were 114.6 million, pre-funded warrants outstanding were 3.9 million, and preferred shares outstanding were 2.8 million. Each preferred share is convertible into five common shares.

On May 13, 2025 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the first quarter of 2025, announced an update on progress toward achieving key corporate objectives, and outlined its commercial, clinical and pipeline development milestones (Press release, Exelixis, MAY 13, 2025, View Source [SID1234652964]).

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"Exelixis delivered outstanding financial performance in the first quarter of 2025, driven by accelerating growth in CABOMETYX demand, new patient starts and revenues," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer, Exelixis. "Based on the strong first quarter dynamics of CABOMETYX, we’re increasing our 2025 full year financial guidance for net product revenues and total revenues by $100 million. The Exelixis commercial team rapidly mobilized for the launch of CABOMETYX in advanced neuroendocrine tumors (NET) within hours of receiving U.S. regulatory approval in late March. We are very pleased with the initial reception and plan to provide further updates to our 2025 financial guidance as we build momentum on the NET launch and gain further clarity on additional revenue opportunities for 2025."

Dr. Morrissey continued: "As we work toward our goal of becoming a multi-franchise oncology company, we look forward to important potential milestones for zanzalintinib in the second half of 2025, based on anticipated event rates. These include pivotal trial readouts from STELLAR-303 in colorectal cancer and STELLAR-304 in non-clear cell renal cell carcinoma, as well as data to drive a decision to move into the phase 3 portion of the STELLAR-305 trial in head and neck cancer. We also look forward to the initiation of the STELLAR-311 pivotal study in neuroendocrine tumors in the first half of the year, and Merck’s anticipated initiation of two pivotal studies evaluating zanzalintinib and belzutifan in renal cell carcinoma. Furthermore, we continue to provide insights on our earlier-stage pipeline, with preclinical data presented on multiple programs at the AACR (Free AACR Whitepaper) Annual Meeting in April. I’m proud of the entire Exelixis team for our progress so far in 2025 and look forward to sharing additional updates throughout the year."

First Quarter 2025 Financial Results

Total revenues for the quarter ended March 31, 2025 were $555.4 million, as compared to $425.2 million for the comparable period in 2024.

Total revenues for the quarter ended March 31, 2025 included net product revenues of $513.3 million, as compared to $378.5 million for the comparable period in 2024. The increase in net product revenues was primarily due to an increase in sales volume and an increase in average net selling price.

Collaboration revenues, composed of license revenues and collaboration services revenues, were $42.2 million for the quarter ended March 31, 2025, as compared to $46.7 million for the comparable period in 2024. The decrease in collaboration revenues was primarily related to lower royalty revenues for the sales of cabozantinib outside of the U.S. generated by Exelixis’ collaboration partners, Ipsen Pharma SAS (Ipsen) and Takeda Pharmaceutical Company Limited, and lower development cost reimbursements earned.

Research and development expenses for the quarter ended March 31, 2025 were $212.2 million, as compared to $227.7 million for the comparable period in 2024. The decrease in research and development expenses was primarily related to decreases in license and other collaboration costs and clinical trial costs, partially offset by increases in stock-based compensation and personnel expenses.

Selling, general and administrative expenses for the quarter ended March 31, 2025 were $137.2 million, as compared to $114.0 million for the comparable period in 2024. The increase in selling, general and administrative expenses was primarily related to increases in personnel expenses, corporate giving, and marketing expenses.

Provision for income taxes for the quarter ended March 31, 2025 was $46.1 million, as compared to $12.0 million for the comparable period in 2024.

GAAP net income for the quarter ended March 31, 2025 was $159.6 million, or $0.57 per share, basic and $0.55 per share, diluted, as compared to GAAP net income of $37.3 million, or $0.12 per share, basic and diluted, for the comparable period in 2024. GAAP net income per share for the quarter ended March 31, 2025 was favorably impacted by lower weighted-average common shares outstanding for the quarter ended March 31, 2025, as compared to the comparable period in 2024, as a result of the stock repurchase programs.

Non-GAAP net income for the quarter ended March 31, 2025 was $179.6 million, or $0.64 per share, basic and $0.62 per share, diluted, as compared to non-GAAP net income of $52.0 million, or $0.17 per share, basic and diluted, for the comparable period in 2024.

Non-GAAP Financial Measures

To supplement Exelixis’ financial results presented in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Exelixis presents non-GAAP net income (and the related per share measures), which excludes from GAAP net income (and the related per share measures) stock-based compensation, adjusted for the related income tax effect for all periods presented.

Exelixis believes that the presentation of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors. In particular, Exelixis believes that these non-GAAP financial measures, when considered together with its financial information prepared in accordance with GAAP, can enhance investors’ and analysts’ ability to meaningfully compare Exelixis’ results from period to period, and to identify operating trends in Exelixis’ business. Exelixis has excluded stock-based compensation, adjusted for the related income tax effect, because it is a non-cash item that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. Exelixis also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate its business and to make operating decisions.

These non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. Exelixis encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP financial information and the reconciliation between these presentations, to more fully understand Exelixis’ business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.

2025 Financial Guidance

Exelixis is providing the following updated financial guidance for fiscal year 2025:

Current Guidance

(provided on May 13, 2025)

Previous Guidance

(provided on January 12, 2025)

Total revenues

$2.25 billion – $2.35 billion

$2.15 billion – $2.25 billion

Net product revenues

$2.05 billion – $2.15 billion(1)

$1.95 billion – $2.05 billion(1)

Cost of goods sold

4% – 5% of net product revenues

4% – 5% of net product revenues

Research and development expenses

$925 million – $975 million(2)

$925 million – $975 million(4)

Selling, general and administrative expenses

$475 million – $525 million(3)

$475 million – $525 million(5)

Effective tax rate

21% – 23%

21% – 23%

____________________

(1)

Exelixis’ 2025 net product revenues guidance range includes the impact of a U.S. wholesale acquisition cost increase of 2.8% for CABOMETYX effective Jan. 1, 2025.

(2)

Includes $50.0 million of non-cash stock-based compensation.

(3)

Includes $80.0 million of non-cash stock-based compensation.

(4)

Includes $40.0 million of non-cash stock-based compensation.

(5)

Includes $60.0 million of non-cash stock-based compensation.

Cabozantinib and Pipeline Highlights

Cabozantinib Franchise Net Product Revenues and Royalties. Net product revenues generated by the cabozantinib franchise in the U.S. were $513.3 million during the first quarter of 2025, with net product revenues of $510.9 million from CABOMETYX (cabozantinib) and $2.4 million from COMETRIQ (cabozantinib). Based upon cabozantinib-related net product revenues generated by Exelixis’ collaboration partners during the quarter ended March 31, 2025, Exelixis earned $36.7 million in royalty revenues.

Received U.S. Food and Drug Administration (FDA) Approval of CABOMETYX for Patients with Previously Treated Advanced NET. In March, Exelixis announced that the U.S. FDA approved CABOMETYX for the treatment of 1) adult and pediatric patients 12 years of age and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic NET (pNET); and 2) adult and pediatric patients 12 years of age and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated extra-pancreatic NET (epNET). These latest FDA approvals — adding to five previous approvals for CABOMETYX — are based on results from CABINET, a phase 3 pivotal trial evaluating CABOMETYX compared with placebo in two cohorts of patients with previously treated NET: advanced pNET and advanced epNET. CABOMETYX is now the first and only systemic treatment that is FDA approved for previously treated NET regardless of primary tumor site, grade, somatostatin receptor expression and functional status. NET are heterogeneous tumors that arise from the neuroendocrine cells of the digestive tract and other organs, such as the lung and pancreas.

Announced Final Five-Year Follow-up Results from CheckMate -9ER Trial Evaluating CABOMETYX in Combination with Nivolumab (Opdivo) in Patients with Advanced Kidney Cancer at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 Genitourinary Cancers Symposium (ASCO GU 2025). In February, final results from the phase 3 CheckMate -9ER pivotal trial evaluating CABOMETYX in combination with nivolumab versus sunitinib for patients with previously untreated advanced renal cell carcinoma (RCC) were presented at ASCO (Free ASCO Whitepaper) GU 2025. After more than five years of follow-up, the findings demonstrated that efficacy benefits with CABOMETYX in combination with nivolumab were sustained long term. Safety and tolerability with long-term follow-up were manageable and consistent with previous analyses. No new safety signals were reported.

Detailed Results from Subgroup Analysis of Phase 3 CABINET Pivotal Study Evaluating Cabozantinib in Advanced Gastrointestinal (GI) NET Presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI 2025). In January, results from a subgroup analysis of the CABINET study of patients with epNET arising in the GI tract were featured in a poster session at ASCO (Free ASCO Whitepaper) GI 2025. The analysis showed cabozantinib was associated with an improvement in progression-free survival (PFS) compared with placebo in patients with advanced GI NET, which was a subgroup of the epNET cohort. Earlier in January, the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology for Neuroendocrine and Adrenal Tumors were updated to include cabozantinib as category 1 preferred regimen for the majority of well-differentiated advanced NET following specific treatments, and as a category 2A preferred regimen for other forms of advanced NET, depending on tumor grade and different requirements for prior therapy.

Encouraging Results from Phase 1b/2 STELLAR-001 Trial Evaluating Zanzalintinib Alone or in Combination with Atezolizumab (Tecentriq) in Metastatic Colorectal Cancer (CRC) Presented at ASCO (Free ASCO Whitepaper) GI 2025. In January, results from a randomized expansion cohort of the phase 1b/2 STELLAR-001 trial evaluating zanzalintinib versus the combination of zanzalintinib and atezolizumab in patients with previously treated metastatic CRC were presented during a poster session at ASCO (Free ASCO Whitepaper) GI 2025. Results from the study demonstrated that all efficacy parameters, including objective response rate, PFS and overall survival favored the combination of zanzalintinib plus atezolizumab over zanzalintinib monotherapy in the overall population, as well as in a subgroup of patients without liver metastases. These data provide insights into the contribution of components and support zanzalintinib’s ongoing pivotal development in metastatic CRC.

Presentation of Preclinical Data from Four Pipeline Programs in Advanced Cancers at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (AACR 2025). In April, Exelixis presented preclinical data from four pipeline molecules at AACR (Free AACR Whitepaper) 2025. Presentations included data for XL309 and XL495, small molecules that have demonstrated synthetic lethality in the context of certain genetic anomalies found in varying frequencies across a broad array of tumor types. The XL309 findings demonstrated activity of XL309 as monotherapy or in combination with PARP or topoisomerase inhibitors. Data analysis from the XL495 program demonstrated the potential for anti-tumor activity both as a monotherapy and in combination with DNA-damaging agents. However, based on early clinical data generated for XL495, Exelixis has discontinued further development of this program. Preclinical data were also presented for the PD-L1 + NKG2A-targeting bispecific antibody XB628 and for the tissue factor-targeting antibody-drug conjugate XB371. Data analysis from the XB628 program demonstrated the molecule’s tumor cell killing activity both in vitro and in vivo, supporting advancement of this molecule into clinical development. Earlier in March, the U.S. FDA cleared Exelixis’ Investigational New Drug (IND) application for XB628, and the company initiated the phase 1 study in April. Findings from XB371 non-clinical experiments demonstrated potent anti-tumor activity in vivo across a range of human tumor xenograft models including colorectal, lung, and pancreatic cancers, supporting the molecule’s advancement into phase 1 clinical development. Exelixis is on track to submit an IND application for XB371 to the FDA in 2025.

Cabozantinib and Zanzalintinib Data Presentations at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO 2025). Cabozantinib and zanzalintinib will be the subject of 17 presentations at ASCO (Free ASCO Whitepaper) 2025, which is being held from May 30 through June 3 in Chicago. Notably, presentations will include health-related quality of life findings from the phase 3 CABINET pivotal trial for cabozantinib, as well as results from two dose-escalation cohorts in advanced solid tumors and a clear cell RCC expansion cohort of the phase 1b/2 STELLAR-002 trial evaluating zanzalintinib in combination with immune checkpoint inhibitors in advanced solid tumors.

Corporate Highlights

Stock Repurchase Program. In August 2024, Exelixis’ Board of Directors authorized a stock repurchase program to acquire up to $500 million of the company’s common stock before December 31, 2025. In February 2025, the Board of Directors authorized the repurchase of up to an additional $500 million of the company’s common stock before December 31, 2025. Under these programs, as of March 31, 2025, Exelixis has repurchased $494.5 million of the company’s common stock, at an average price of $34.87 per share. Since the approval of the first stock repurchase program in March 2023, the weighted-average diluted common shares outstanding has decreased from 326.3 million shares to 288.2 million shares as of March 31, 2025. Stock repurchases under these programs may be made from time to time through a variety of methods, which may include open market purchases, in block trades, accelerated share repurchase transactions, exchange transactions, or any combination of such methods. The timing and amount of any stock repurchases under the stock repurchase programs will be based on a variety of factors, including ongoing assessments of the capital needs of the business, alternative investment opportunities, the market price of our common stock and general market conditions.

Basis of Presentation

Exelixis has adopted a 52- or 53-week fiscal year that generally ends on the Friday closest to December 31. For convenience, references in this press release as of and for the fiscal periods ended April 4, 2025 and March 29, 2024, are indicated as being as of and for the periods ended March 31, 2025 and March 31, 2024.

Conference Call and Webcast

Exelixis management will discuss the company’s financial results for the first quarter 2025 and provide a general business update during a conference call beginning at 5:00 p.m. ET / 2:00 p.m. PT today, Tuesday, May 13, 2025.

To access the conference call, please register using this link. Upon registration, a dial-in number and unique PIN will be provided to join the call. To access the live webcast link, log onto www.exelixis.com and proceed to the Event Calendar page under the Investors & News heading. A webcast replay of the conference call will also be archived on www.exelixis.com for one year.