On May 12, 2025 Curium reported that it has marketing authorization in Switzerland for the distribution of PYLCLARI (INN: Piflufolastat (18F) formerly known as (18F)-DCFPyL) (Press release, Curium Pharma, MAY 12, 2025, View Source [SID1234652875]). PYLCLARI is indicated for the detection of prostate-specific membrane antigen (PSMA) positive lesions with positron emission tomography (PET) in adults with prostate cancer in the following clinical settings:

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Primary staging of patients with high-risk prostate cancer prior to initial curative therapy
To localize recurrence of prostate cancer in patients with a suspected recurrence based on increasing serum prostate-specific antigen (PSA) levels after primary treatment with curative intent
Today’s announcement follows the decision in July 2023 by the European Commission granting marketing authorization for PYLCLARI in the European Union. b.e. Imaging currently distributes Curium’s entire suite of SPECT radiopharmaceuticals across Switzerland and following an exclusive distribution rights agreement in September 2023, will now also distribute PYLCLARI.

Dr. Michel Wuillemin, Managing Director at b.e. Imaging commented, "b.e. Imaging has proven to be a crucial supplier for the whole range of Curium’s SPECT radiopharmaceuticals for Switzerland. The extension of the partnership with Curium to PSMA PET imaging with PYLCLARI underscores b.e. Imaging’s focus in the field of prostate cancer. Curium’s PYLCLARI is in line with b.e. Imaging’s experience in the field of radioligand therapy for patients with prostate cancer."

Benoit Woessmer, PET Europe CEO at Curium commented, "We are pleased with the growing availability of PYLCLARI to reach more nuclear medicine physicians and their patients across Europe. With today’s announcement, we are extremely proud to be improving the choice of diagnostic radiopharmaceuticals available to our customers in Switzerland – ultimately for the benefit of patients with prostate cancer."

In Switzerland, prostate cancer is the most common cancer among men with around 7,800 new cases diagnosed nationwide every year. Today’s announcement is part of the continued roll-out of PYLCLARI across the European Union, which with the addition of Switzerland, is now available for patients with prostate cancer in 12 countries. In the U.S., Lantheus received approval for PYLARIFY (Piflufolastat (18F) Injection) from the Food and Drug Administration (FDA) in May 2021. It is the #1 utilized PSMA PET agent in the U.S. market. The European rights were licensed to Curium from Progenics, a Lantheus company, in 2018.

On May 12, 2025 Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, reported financial results for the first quarter ended March 31, 2025, and provided a review of recent accomplishments and anticipated upcoming developments (Press release, Biohaven Pharmaceutical, MAY 12, 2025, View Source [SID1234652891]).

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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "In spite of a challenging macroeconomic climate and economic uncertainty, our team has remained more focused than ever on strategic execution and creating a strong financial balance sheet to create value across our broad portfolio of innovative product candidates. Enthusiasm for our rare disease and degrader programs has grown, as we eagerly await the completion of the NDA review for troriluzole and continued advancement of our MoDE and TRAP degraders into the clinic. We expect to complete three separate Phase 1 studies in 1H 2025 with three innovative compounds, including BHV-1300 (IgG degrader targeting with first target indication in Graves’ disease), BHV-1400 (Gd-IgA1 degrader for IgA nephropathy), and BHV-1600 (β1AR AAb degrader targeting PPCM), and are advancing four additional degrader molecules in parallel. We also look forward to delivering key clinical readouts, including pivotal topline data with troriluzole in obsessive compulsive disorder in 1H 2025 and with our selective Kv7 activator BHV-7000 in major depressive disorder ("MDD") in 2H 2025. In oncology, interim Phase 1 data remain on track for 2025 with our lead clinical Trop-2 antibody drug conjugate ("ADC") program, BHV-1510, and initiation of a Phase 1 study with the field’s first FGFR3 ADC, BHV-1530, for patients with urothelial cancer & other tumors. With taldefgrobep alfa, our anti-myostatin agent, we continue to pursue a potential path forward in spinal muscular atrophy, while we hasten to initiate our Phase 2 study in obesity; we are also extremely excited about BHV-8000, our TYK2/JAK1 inhibitor, and eagerly anticipate initiating our Phase 2/3 study in Parkinson’s disease in 1H 2025."

Dr. Coric continued, "Importantly, we remain steadfastly committed to the SCA community as we continue unwaveringly pursuing an approval in this indication and are now past the mid-cycle review. In addition, our recent $600 million financing agreement with Oberland Capital will provide financial flexibility to advance our troriluzole commercialization plans, accelerate clinical development and operational execution across our five platforms, and be ready to execute on strategic opportunities as they arise. We look forward to sharing further progress from all of our innovative discovery and clinical programs and future development plans at our annual R&D Day at the Yale Innovation Summit on May 28, 2025 in New Haven, CT."

First Quarter 2025 and Recent Business Highlights

Announced up to $600 Million non-dilutive capital agreement with Oberland Capital — In April 2025, the Company entered into an agreement with Oberland Capital for an investment of up to $600 million in the Company, with the first tranche of $250 million of gross proceeds being funded at closing on April 30, 2025. The investment from Oberland Capital takes the form of a Note Purchase Agreement ("NPA") that is non-dilutive to current investors under which Oberland may purchase up to $600 million of Biohaven’s senior secured notes. The second tranche of up to $150 million can be funded at the Company’s option contingent upon FDA approval of troriluzole, and subject to the satisfaction of certain additional conditions, and the third tranche of up to $200 million can be funded upon the mutual agreement of the parties for permitted strategic acquisitions and related costs and expenses. The purchases of the senior notes are subject to other terms and conditions as set forth in the NPA. Under the terms of the NPA, Oberland Capital will have a right to receive a regulatory approval milestone payment of 35% of the amount funded, payable quarterly through December 31, 2030, and for the first tranche, single-digit royalty payments on global net sales of troriluzole for up to a maximum of 10 years from the closing date. These payments are capped at a multiple of amounts funded by Oberland Capital.
Troriluzole NDA mid-cycle review meeting with FDA completed — The Company completed a mid-cycle review meeting with the FDA of the NDA for troriluzole for the treatment of SCA. The FDA has not conveyed any intention of holding an Advisory Committee Meeting. The NDA had previously been accepted and granted priority review by the FDA with a Prescription Drug User Fee Act ("PDUFA") date expected in the third quarter of 2025.
Oral and poster presentations at AAN showcased breadth of development work across the platform — In April 2025, the Company delivered 3 oral presentations and 10 posters at the AAN Annual Meeting, showcasing development programs including Kv7 ion channel modulation, MoDEs, TRPM3 antagonism, TYK2/JAK1 inhibition, and glutamate modulation.
Biohaven’s selective, brain-penetrant TYK2/JAK1 inhibitor, BHV-8000, was selected for an oral presentation at AAN, highlighting the efficacy demonstrated in a human alpha-synuclein overexpressing Parkinson’s disease mouse model.
Other oral presentations covered the safety, tolerability, and pharmacokinetics of BHV-2100, a first-in-class TRPM3 antagonist for pain and migraine, as well as the rapid, robust, and selective IgG reduction observed in preclinical models of BHV-1310, Biohaven’s novel IgG degrader.
Expected Upcoming Milestones:

We believe Biohaven is well positioned to achieve significant milestones in 2025 and 2026 across numerous programs:

MoDE Platform

IgG MoDE Degraders (1300/1310): BHV-1300 Phase 1 with the optimized subcutaneous formulation completing in 1H 2025. Expect to initiate Phase 1b study in Graves’ disease in mid-2025, and additional programs in rheumatoid arthritis and myasthenia gravis continue to be pursued.
Phase 1 studies with BHV-1400 and BHV-1600 expected to be completed in 1H 2025.
Four additional degrader molecules advancing including: IgG4 degrader, PLA2R autoantibody degrader, pro-insulin autoantibody degrader, and TSH receptor autoantibody degrader.
Kv7 Activator (BHV-7000):

Pivotal major depressive disorder topline results expected in 2H 2025.
Focal epilepsy study pivotal topline results expected in 1H 2026.
Glutamate Modulator (Troriluzole):

Preparing for commercial launch in all-genotype SCA in 2025, following NDA acceptance with Priority Review and 3Q 2025 PDUFA date.
Pivotal topline data from two Phase 3 OCD trials expected in 1H 2025 and 2H 2025, respectively.
Myostatin (Taldefgrobep alfa):

FDA interaction to discuss Spinal Muscular Atrophy ("SMA") registrational path planned in 1H 2025.
Initiate taldefgrobep Phase 2 study in obesity in 1H 2025.
TRPM3 Antagonist (BHV-2100):

Expect data from proof-of-concept trial with BHV-2100 in acute migraine in 1H 2025.
TYK2/JAK1 Inhibitor (BHV-8000):

Initiate BHV-8000 Phase 2/3 study in Parkinson’s disease in 1H 2025.
Advance Alzheimer’s disease, multiple sclerosis ("MS") and amyloid-related imaging abnormalities ("ARIA") programs.
Next Generation ADC Platform:

Preliminary Phase 1 data with BHV-1510 and dose optimization as monotherapy and combination therapy with Libtayo in epithelial tumors in 2025.
Initiate Phase 1 trial of BHV-1530 in 1H 2025.
Advance additional preclinical ADCs, including Merus and GeneQuantum collaborations (undisclosed targets) in 2025.
Capital Position:

Cash, cash equivalents, marketable securities and restricted cash as of March 31, 2025 totaled approximately $327 million. In addition, the Company received $250 million in gross proceeds in April 2025 from Oberland Capital under the NPA discussed above.

First Quarter 2024 Financial Highlights:

Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $187.6 million for the three months ended March 31, 2025, compared to $156.0 million for the three months ended March 31, 2024. The increase of $31.6 million was due to increased non-cash share-based compensation expense in 2025, as well as increased direct program spend for advancing clinical trials and preclinical research programs in 2025, as compared to the same period in the prior year. Preclinical research expense for the three months ended March 31, 2025 included an upfront share payment valued at $4.9 million and an accrual for an upfront cash payment of $5.0 million related to agreements entered into during the three months ended March 31, 2025. These increases were partially offset by decreased program expense for BHV-1510, primarily related to the acquisition of Pyramid Biosciences, Inc., which resulted in a $10.9 million non-cash upfront payment and $7.2 million in milestones which became due during the three months ended March 31, 2024. Non-cash share-based compensation expense was $35.2 million for the three months ended March 31, 2025, an increase of $13.9 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

General and Administrative (G&A) Expenses: G&A expenses, including non-cash share-based compensation costs, were $34.0 million for the three months ended March 31, 2025, compared to $27.3 million for the three months ended March 31, 2024. The increase of $6.7 million was primarily due to increased non-cash share-based compensation expense and increased legal costs. Non-cash share-based compensation expense was $17.8 million for the three months ended March 31, 2025, an increase of $4.2 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

Other Income, Net: Other income, net was $0.5 million for the three months ended March 31, 2025, compared to other income, net of $4.3 million for the three months ended March 31, 2024. The decrease of $3.8 million was primarily due to non-cash changes in the fair value of our forward contract and derivative liability recorded in connection with the amendment to our Membership Interest Purchase Agreement with Knopp Biosciences LLC in May 2024 (the Knopp Amendment).

Net Loss: Biohaven reported a net loss for the three months ended March 31, 2025 of $221.7 million, or $2.17 per share, compared to $179.5 million, or $2.20 per share, for the same period in 2024. Non-GAAP adjusted net loss for the three months ended March 31, 2025 was $166.8 million, or $1.64 per share, compared to $144.6 million, or $1.77 per share for the same period in 2024. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

On May 12, 2025 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of masked, conditionally activated biologics, reported first quarter 2025 financial results and provided a business update (Press release, CytomX Therapeutics, MAY 12, 2025, View Source [SID1234652876]).

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"Our positive interim clinical results announced today for CX-2051 in advanced colorectal cancer are highly encouraging and provide a significant opportunity for CytomX. As an EpCAM-directed ADC, CX-2051 was intentionally designed to address the high unmet need in CRC. CX-2051 remains the Company’s top strategic priority and is positioned to rapidly advance towards later stage development. Just one year into the clinic, CX-2051 dose expansions are already in progress with a goal to initiate a Phase 2 study in advanced CRC in the first half of 2026. This excellent progress underscores the intense focus of the CytomX team on diligent execution for the benefit of the patients we serve," said Sean McCarthy, D.Phil., chief executive officer and chairman of CytomX.

Pipeline Program Updates:

CX-2051 (EpCAM PROBODY Topo-1 ADC)

Announced positive interim data from ongoing Phase 1 dose escalation study of EpCAM Antibody Drug Conjugate (CX-2051) candidate in patients with advanced colorectal cancer (CRC).
Initiated CX-2051 dose expansions at the 7.2 mg/kg, 8.6 mg/kg, and 10 mg/kg doses, administered every three weeks (Q3W).
Phase 1 data update in advanced CRC in at least 70 patients is expected to be presented by Q1 2026.
Planning Phase 2 study initiation in 1H 2026
CX-801 (PROBODY Interferon alpha-2b)

Phase 1 dose escalation continues with a focused early development strategy in metastatic melanoma and with the goal of initiating combination therapy with CX-801 and KEYTRUDA in 2025.
The Phase 1 study is currently in the fourth monotherapy dose escalation cohort where the dose of CX-801 exceeds the approved dose of the unmasked peginterferon alfa-2b (SYLATRON)1.
Initial Phase 1a translational and biomarker data in advanced melanoma is expected in the second half of 2025.
KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA

Corporate and Financial:

Financial:
Focused clinical development priorities and cost reductions implemented in Q1 2025 have extended the Company’s cash runway into the second quarter of 2026. CytomX ended the first quarter of 2025 with $79.9 million of cash, cash equivalents and investments.
Research collaborations:
Milestone achieved in Astellas T-cell engager collaboration: In February 2025, Astellas advanced the second program to GLP toxicology studies, triggering a $5.0 million milestone payment to CytomX.
Presented preclinical data for mRNA encoded masked IL-12 molecule in collaboration with Moderna at AACR (Free AACR Whitepaper) Annual Meeting showing potent anti-tumor activity with significantly enhanced tolerability vs. unmasked IL-12 molecule.
Multiple drug discovery programs continue across our research collaborations with a focus on T-cell engagers. CytomX has research collaborations with Bristol Myers Squibb, Amgen, Astellas, Regeneron, and Moderna.
First Quarter 2025 Financial Results:

Cash, cash equivalents and investments totaled $79.9 million as of March 31, 2025, compared to $100.6 million as of December 31, 2024.

Total revenue was $50.9 million for the quarter ended March 31, 2025, compared to $41.5 million for the quarter ended March 31, 2024. The increase in revenue was driven primarily by a higher percentage of completion for research programs in the Bristol Myers Squibb collaboration and the acceleration of revenue recognition in the Amgen collaboration due to the decision to not further develop the CX-904 program, partially offset by lower Astellas milestones and Moderna revenue.

Total operating expense in the first quarter of 2025 was $28.3 million compared to $29.8 million in the first quarter of 2024, a decrease of $1.5 million. Operating expenses in the first quarter of 2025 included $2.9 million of one-time expenses related to the Company’s January 2025 restructuring.

Research and development expenses were $18.9 million for the three months ended March 31, 2025, a decrease of $3.2 million compared to the corresponding period of 2024. Reduced research and development expenses were primarily due to reduced pre-clinical activities in wholly owned and partnered programs and decreased manufacturing activities for CX-801, partially offset by increased clinical trial activities related to CX-2051 and CX-801, and $1.8 million of restructuring expenses.

General and administrative expenses were $9.4 million for the three months ended March 31, 2025, an increase of $1.7 million compared to the corresponding period of 2024. The increase in general and administrative expenses was primarily driven by $1.1 million of restructuring expenses as well as other personnel-related expenses.

On May 12, 2025 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), the Breast International Group (BIG), Institut Jules Bordet Clinical Trials Support Unit and Frontier Science Foundation, reported statistically significant final overall survival (OS) results from the Phase III APHINITY study in people with human epidermal growth factor receptor 2 (HER2)-positive early-stage breast cancer (Press release, Genentech, MAY 12, 2025, View Source [SID1234652892]). After ten years, the risk of death was reduced by 17% for people treated with Perjeta (pertuzumab), Herceptin (trastuzumab) and chemotherapy (the Perjeta-based regimen) for a year as post-surgery (adjuvant) treatment, compared with individuals who received Herceptin, chemotherapy, and placebo.

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"Early treatment of breast cancer can provide substantial patient benefit and also increases the chance for cure. For people with early-stage HER2-positive disease, the APHINITY results validate the sustained benefits of the Perjeta-based regimen," said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. "These long-term data reinforce the regimen’s value as a well-established standard-of-care treatment in the curative setting."

"After ten years, the APHINITY trial clearly shows a statistically significant and clinically meaningful improvement of the overall survival," said Prof. Sibylle Loibl, APHINITY study chair, chair of the German Breast Group (GBG) and the chief executive officer of the GBG Forschungs GmbH. "Adding Perjeta to a standard adjuvant treatment is most beneficial for people with HER2-positive breast cancer with lymph node-positive disease who are at high risk of recurrence."

After ten years, results show:

91.6% of people treated with the Perjeta-based regimen were alive at ten years versus 89.8% of those treated with Herceptin, chemotherapy, and placebo (hazard ratio [HR]=0.83, 95% CI: 0.69-1.00, p-value=0.044).
A 21% reduction in the risk of death was seen in the prespecified subgroup of people with lymph node-positive disease (HR=0.79, 95% CI: 0.64-0.97).
The previously reported invasive disease-free survival (primary endpoint) benefit was maintained (HR=0.79, 95% CI: 0.68-0.92), strengthening results from earlier APHINITY analyses. No benefit was seen in the node negative subgroup.
The safety profile, including cardiac safety, was consistent with previous studies and no new or unexpected safety signals were identified.

Full results will be presented as a late-breaking abstract on Thursday, May 15 at the 2025 European Society for Medical Oncology Breast Cancer Congress.

"The international collaborations in APHINITY have facilitated important insights about HER2-positive breast cancer and are continuing to yield promising findings," said Liz Frank, independent research advocate. "Scientists and clinicians are working together with the broader goal of improving our understanding of HER2-positive breast cancer, improving the quality of life for people living with the disease and ultimately, helping them to live longer with no disease occurring."

The collaborative efforts of Genentech, BIG, and study partners enabled the initiation of pivotal trials such as APHINITY and HERA. These studies led to Herceptin and Perjeta becoming standards of care and helped improve outcomes for people with early-stage HER2-positive breast cancer.

About the APHINITY study

APHINITY (Adjuvant Pertuzumab and Herceptin IN Initial TherapY in Breast Cancer, NCT01358877/ BO25126/ BIG 4-11) is a global, Phase III, randomized, double-blind, placebo-controlled, two-arm study evaluating the efficacy and safety of Perjeta (pertuzumab) plus Herceptin (trastuzumab) and chemotherapy, compared with Herceptin and chemotherapy, as post-surgery (adjuvant) treatment in 4,804 people with operable human epidermal growth factor receptor 2-positive early-stage breast cancer.

The primary endpoint is invasive disease-free survival, which in this study is defined as the time a patient lives without recurrence of invasive breast cancer (when the cancer returns locally or spreads into the surrounding breast tissue and/or beyond) or death from any cause after post-surgery treatment. Secondary endpoints include cardiac and overall safety, overall survival and health-related quality of life.

What is Perjeta?

Perjeta (pertuzumab) is a prescription medicine approved for use in combination with Herceptin and chemotherapy for:

Use prior to surgery (neoadjuvant treatment) in adults with HER2-positive, locally advanced, inflammatory, or early stage breast cancer as part of a complete treatment regimen for early breast cancer
Use after surgery (adjuvant treatment) in adults with HER2-positive early breast cancer that has a high likelihood of coming back

Perjeta (pertuzumab) is a prescription medicine approved for use in combination with Herceptin and docetaxel in adults who have HER2-positive breast cancer that has spread to different parts of the body (metastatic) and who have not received prior anti-HER2 therapy or chemotherapy for metastatic breast cancer.

Important Safety Information

What are the possible side effects of Perjeta?

Perjeta may cause serious side effects, including:

Perjeta can cause heart problems, including those without symptoms (such as reduced heart function) and those with symptoms (such as congestive heart failure)
Your doctor will run tests to monitor your heart function before and during treatment
Based on these tests, your treatment may be interrupted or discontinued
Contact a health care professional immediately if you experience any of the following: new onset or worsening shortness of breath, cough, swelling of the ankles/legs, swelling of the face, palpitations, weight gain of more than 5 pounds in 24 hours, dizziness or loss of consciousness
Receiving Perjeta during pregnancy can result in the death of an unborn baby and birth defects.
Your doctor will verify your pregnancy status before treatment begins
Birth control should be used while receiving Perjeta and for 7 months after your last dose of Perjeta. If you are a mother who is breastfeeding, you should talk with your doctor about either stopping breastfeeding or stopping Perjeta
If you think you may be pregnant, you should contact your healthcare provider immediately
If you are exposed to Perjeta during pregnancy, or become pregnant while receiving Perjeta or within 7 months following the last dose of Perjeta with Herceptin, you are encouraged to report Perjeta exposure to Genentech at 1-888-835-2555

Who should not take Perjeta?

Perjeta should not be used in patients who are allergic to pertuzumab or to any of the ingredients in Perjeta.

What are other possible serious side effects of Perjeta?

Serious side effects of Perjeta may also include:

Infusion-related reactions: Perjeta is given as an infusion. Perjeta can cause serious infusion-related reactions, some fatal. When given alone, the most common infusion-related reactions were fever, chills, fatigue, headache, weakness, hypersensitivity, and vomiting. When given with Herceptin and docetaxel, the most common infusion-related reactions were fatigue, altered taste, hypersensitivity, muscle pain, and vomiting
Severe allergic reactions: Perjeta can cause hypersensitivity reactions, including anaphylaxis and fatal events. Contact a health care professional immediately if you experience any of the following symptoms: swelling of the face, lips or tongue, trouble breathing, or chest pains

The most common side effects of Perjeta include:

The most common side effects of Perjeta when given with Herceptin and chemotherapy prior to surgery for early breast cancer include:

Constipation
Damage to the nerves (numbness, tingling, pain in hands/feet)
Diarrhea
Fatigue
Hair loss
Headache
Decreased red blood cell counts, white blood cell counts, and platelet counts
Mouth sores or blisters
Nausea
Muscle pain
Vomiting
Weakness

The most common side effects of Perjeta when given with Herceptin and chemotherapy after surgery for early breast cancer include:

Diarrhea
Nausea
Hair loss
Fatigue
Damage to the nerves (numbness, tingling, pain in hands/feet)
Vomiting

The most common side effects of Perjeta when given with Herceptin and docetaxel for metastatic breast cancer include:

Diarrhea
Hair loss
Low levels of white blood cells with or without fever
Nausea
Fatigue
Rash
Damage to the nerves (numbness, tingling, pain in hands/feet)

Side effects may vary based on chemotherapy regimen. These are not all the possible side effects of Perjeta. Call your healthcare provider for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 or View Source You may also report side effects to Genentech at 1-877-436-3683.

Before you take Perjeta, tell your healthcare provider about all of your medical conditions, including if you:

Have a history of heart disease
Are pregnant or plan to become pregnant. Perjeta can harm your unborn baby
Are breastfeeding or plan to breastfeed. It is not known if Perjeta passes into your breastmilk
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Please see the full Prescribing Information for additional Important Safety Information, including most serious side effects.

What is Herceptin?

Herceptin is approved for the treatment of early stage breast cancer that is Human Epidermal growth factor Receptor 2-positive (HER2-positive) and has spread into the lymph nodes, or is HER2-positive and has not spread into the lymph nodes. If it has not spread into the lymph nodes, the cancer needs to be estrogen receptor/progesterone receptor (ER/PR)-negative or have one high-risk feature.* Herceptin can be used in several different ways:

As part of a treatment course including the chemotherapy drugs doxorubicin, cyclophosphamide, and either paclitaxel or docetaxel. This treatment course is known as "AC→ TH."
With the chemotherapy drugs docetaxel and carboplatin. This treatment course is known as "TCH."
Alone after treatment with multiple other therapies, including an anthracycline (doxorubicin)-based therapy (a type of chemotherapy).

Patients are selected for therapy based on an FDA-approved test for Herceptin.

*High risk is defined as ER/PR-positive with one of the following features: tumor size greater than 2 cm, age less than 35 years, or tumor grade 2 or 3.

Important Safety Information

Possible serious side effects with Herceptin

Not all people have serious side effects, but side effects with Herceptin therapy are common.

Although some people may have a life-threatening side effect, most do not.

A patient’s doctor will stop treatment if any serious side effects occur.

Herceptin is not for everyone. A patient should be sure to contact their doctor if they are experiencing any of the following:

HEART PROBLEMS

These include heart problems—such as congestive heart failure or reduced heart function—with or without symptoms. The risk for and seriousness of these heart problems were highest in people who received both Herceptin and a certain type of chemotherapy (anthracycline). In a study of adjuvant (early) breast cancer, one patient died of significantly weakened heart muscle. A patient’s doctor will check for signs of heart problems before, during, and after treatment with Herceptin.

INFUSION REACTIONS, including:

Fever and chills
Feeling sick to your stomach (nausea)
Throwing up (vomiting)
Pain (in some cases at tumor sites)
Headache
Dizziness
Shortness of breath

These signs usually happen within 24 hours after receiving Herceptin.

A patient should be sure to contact their doctor if they:

Are a woman who could become pregnant, or may be pregnant

Herceptin may result in the death of an unborn baby or birth defects. Contraception should be used while receiving Herceptin and for 7 months after your last dose of Herceptin. If you are or become pregnant while receiving Herceptin or within 7 months after your last dose of Herceptin, you should immediately report HERCEPTIN exposure to Genentech at 1-888-835-2555.

Have any signs of SEVERE LUNG PROBLEMS, including:

Severe shortness of breath
Fluid in or around the lungs
Weakening of the valve between the heart and the lungs
Not enough oxygen in the body
Swelling of the lungs
Scarring of the lungs

A patient’s doctor may check for signs of severe lung problems when he or she examines the patient.

Have LOW WHITE BLOOD CELL COUNTS

Low white blood cell counts can be life threatening. Low white blood cell counts were seen more often in patients receiving Herceptin plus chemotherapy than in patients receiving chemotherapy alone.

A patient’s doctor may check for signs of low white blood cell counts when he or she examines the patient.

Side effects seen most often with Herceptin

Some patients receiving Herceptin for breast cancer had the following side effects:

Fever
Feeling sick to your stomach (nausea)
Throwing up (vomiting)
Infusion reactions
Diarrhea
Infections
Increased cough
Headache
Feeling tired
Shortness of breath
Rash
Low white and red blood cell counts
Muscle pain

A patient should contact their doctor immediately if they have any of the side effects listed above.

Patients are encouraged to report side effects to Genentech and the FDA. You may report side effects to FDA at 1-800-FDA-1088 or View Source You may also report side effects to Genentech at 1-877-436-3683.

Please see the full Prescribing Information, including Boxed WARNINGS and additional Important Safety Information, at View Source

On May 12, 2025 Agenus Inc. ("Agenus" or the "Company") (NASDAQ: AGEN), a leader in immuno-oncology, reported financial and operational results for the first quarter of 2025, and shared key clinical and strategic milestones supporting the advancement of its botensilimab (BOT) and balstilimab (BAL) program (Press release, Agenus, MAY 12, 2025, View Source [SID1234652859]).

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"The growing strength of our BOT/BAL data across multiple hard-to-treat cancers reinforces our conviction in its transformative potential and fuels our unwavering commitment to delivering this combination to patients." said, Garo Armen, Ph.D., Chairman and CEO of Agenus. "With expanded datasets, key leadership appointments, and the FDA’s renewed focus on accelerating cures and meaningful treatments, Agenus is entering a pivotal phase—advancing toward regulatory engagement with financial discipline and a sharp focus on bringing innovative immunotherapies to individuals living with cancer."

Key Highlights from Q1 2025

New Data:

•
BOT/BAL continues to demonstrate robust and durable responses across microsatellite stable (MSS) "cold tumors" where current immuno-oncology treatments have historically failed. At American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Chicago, Illinois, new data highlighted the activity and safety profile in both multiple mismatch repair–proficient (pMMR/MSS) and mismatch repair–deficient (dMMR/MSI-H) solid tumors in neoadjuvant and later line treatment settings.
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Notably, new data from the investigator-sponsored pan-cancer NEOASIS study–a now the third clinical study evaluating BOT/BAL in the neoadjuvant setting–were presented. These initial results from the safety run-in portion indicate that BOT/BAL can induce pathological responses in patients with solid tumors beyond CRC, including TNBC and sarcomas. No dose-limiting toxicities were observed, and all patients proceeded to their scheduled surgery.

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100 percent of dMMR CRC patients given a higher dose of BOT/BAL achieved pCR.
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New data from the HCC cohort of the ongoing Phase 1 study were also presented. The HCC cohort comprised of patients with difficult-to-treat disease who had progressed following standard treatments, including approved immunotherapies. The durable responses and disease control in heavily pretreated HCC patients highlight the strength and differentiation of the BOT/BAL combination.
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data on late stage pan tumor activity to be presented at an upcoming kay cancer conference.
New Leadership:

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Dr. Richard Goldberg, an internationally recognized leader in GI cancer treatment and research, stepped out of early retirement to join Agenus as Chief Development Officer to support the advancement of BOT/BAL for patients. Dr. Goldberg will lead the company’s efforts as it prepares to re-engage global regulatory authorities with expanded data and longer-term follow-up in metastatic CRC.
New Efficiencies:

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Agenus is on track to reduce its annualized operating cash burn below $50 million starting in the second half of 2025, supported by recent cost optimization measures enabling the company to direct resources toward ensuring the potential of BOT/BAL is realized. The company is in final stages of an important collaboration which will result in substantial cash infusion.
Q1 2025 Financial Highlights

Agenus ended the first quarter 2025 with a consolidated cash balance of $18.5 million compared to $40.4 million on December 31, 2024. Cash used in operations for the first quarter ended March 31, 2025 was $25.6 million, reduced from $38.2 million for same period in 2024.

For the first quarter ended March 31, 2025, Agenus recognized revenue of $24.1 million and incurred a net loss of $26.4 million, or $1.03 per share. For the first quarter ended March 31, 2024, Agenus recognized revenue of $28.0 million and incurred a net loss of $63.5 million or $3.04 per share. Revenue primarily includes non-cash royalty revenue.

Financial Highlights

(in thousands, except per share data)

(unaudited)

Cash and cash equivalents

March 31, 2025

$ 18,488

December 31, 2024

$ 40,437

Key Financial Metrics

Q1 2025

Q1 2024

Cash used in operations

$ 25,618

$ 38,191

Revenue, including non-cash royalties

24,066

28,005

Net loss

26,370

63,454

Non-cash expenses included in net loss

19,388

38,255

Exhibit 99.1

Net loss per share attributable to Agenus Inc. common stockholders

1.03

3.04

Conference Call

Date: Monday, May 12th, at 8:30 a.m. ET
To access dial-in numbers, please register here.
Conference ID: 73242

Webcast

A live webcast and replay of the conference call will be accessible on the company’s website at View Source

About Botensilimab (BOT)

Botensilimab is a human Fc enhanced CTLA-4 blocking antibody designed to boost both innate and adaptive anti-tumor immune responses. Its novel design leverages mechanisms of action to extend immunotherapy benefits to "cold" tumors which generally respond poorly to standard of care or are refractory to conventional PD-1/CTLA-4 therapies and investigational therapies. Botensilimab augments immune responses across a wide range of tumor types by priming and activating T cells, downregulating intratumoral regulatory T cells, activating myeloid cells and inducing long-term memory responses.

Approximately 1,100 patients have been treated with botensilimab in phase 1 and phase 2 clinical trials. Botensilimab alone, or in combination with Agenus’ investigational PD-1 antibody, balstilimab, has shown clinical responses across nine metastatic, late-line cancers. For more information about botensilimab trials, visit www.clinicaltrials.gov with the identifiers NCT03860272, NCT05608044, NCT05630183, and NCT05529316.

About Balstilimab (BAL)

Balstilimab is a novel, fully human monoclonal immunoglobulin G4 (IgG4) designed to block PD-1 (programmed cell death protein 1) from interacting with its ligands PD-L1 and PD-L2. It has been evaluated in >900 patients to date and has demonstrated clinical activity and a favorable tolerability profile in several tumor types.