On February 20, 2025 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it has obtained regulatory approval today from the Ministry of Health, Labour and Welfare for the anti-cancer agent/humanized anti-PD-L1 monoclonal antibody Tecentriq Intravenous Infusion [generic name: atezolizumab (genetical recombination)] for an additional indication of unresectable alveolar soft part sarcoma (Press release, Chugai, FEB 20, 2025, View Source;category= [SID1234650398]). Tecentriq is the first immune checkpoint inhibitor in Japan for the treatment of this disease.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are very pleased that we can offer Tecentriq as a new treatment for unresectable alveolar soft part sarcoma in adults and children over 2 years. This very rare disease, which occurs most often in the adolescents and young adults (AYA) generation, is known to have a poor prognosis with no standard treatment if it becomes unresectable. We will continue our efforts to provide information on the proper use of Tecentriq in order to contribute to the patients with unresectable alveolar soft part sarcoma," said Chugai’s President and CEO, Dr. Osamu Okuda.

This approval is based on the results from a phase II ALBERT study initiated by investigators in Japan including National Cancer Center Hospital and an overseas phase II clinical study conducted by the National Cancer Institute (NCI), which evaluated the efficacy and safety of Tecentriq in patients with unresectable alveolar soft part sarcoma.

Chugai Pharmaceutical, a leading company in the oncology field, remains committed to addressing unmet medical needs in cancer treatment with innovative medicines, supporting patients and healthcare professionals.

Approval Information *Newly added description
Indications: unresectable alveolar soft part sarcoma
Dosage and administrations: The usual adult dosage is 1200 mg atezolizumab (genetical recombination) administered by intravenous infusion over 60 minutes once every 3 weeks. The usual dose for children over 2 years old is 15 mg/kg (weight) (max 1200 mg) atezolizumab (genetical recombination) administered by intravenous infusion over 60 minutes once every 3 weeks. If the initial infusion is well tolerated, subsequent infusions can be delivered over 30 minutes.

About the ALBERT study1
The ALBERT study is a domestic Phase II, multicenter, open-label, single-arm study led by physicians including National Cancer Center Hospital in Japan to evaluate the efficacy and safety of Tecentriq in patients aged 16 years and older with unresectable alveolar soft part sarcoma. The study enrolled 20 patients to investigate safety and efficacy.

The ALBERT study is being conducted as a substudy of the MASTER KEY project, which promotes the development of treatments for rare cancers through industry-academia collaboration with the National Cancer Center Hospital.

About alveolar soft part sarcoma
Alveolar soft part sarcoma is one of the ultra-rare cancers accounting for less than 1% of soft tissue sarcomas. It is estimated to occur in 15-40 Japanese people annually. It most commonly affects the limbs, mainly the thighs, and is more common among adolescents and young adults (15-35 years old, AYA (Adolescent and Young Adult) generation). Unresectable alveolar soft part sarcoma has a poor prognosis, and no standard treatment has been established.

About Tecentriq
Tecentriq is a cancer immune checkpoint inhibitor targeting PD-L1, which is a protein expressed on tumor and tumor-infiltrating immune cells. PD-L1 blocks T cell activity by binding with PD-1 and B7.1 receptors on T cell surface. By inhibiting PD-L1, Tecentriq may enable the activation of T cells and boost immune response against cancer cells. In Japan, Tecentriq was launched in April 2018 and has obtained approval for 4 indications (extensive-stage small cell lung cancer, non-small cell lung cancer, breast cancer, and hepatocellular carcinoma).

On February 20, 2025 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported financial results for the fourth quarter and full year periods ended December 31, 2024 and provided an update on recent company developments (Press release, SpringWorks Therapeutics, FEB 20, 2025, View Source [SID1234650420]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are very pleased with the strong execution of OGSIVEO in 2024 and believe that we are still in the early stages of realizing the full potential of our opportunity to serve the desmoid tumor community. With the recent FDA approval of GOMEKLI for adults and children with NF1-PN, we believe we are ready to deliver another strong launch and are delighted that the broad label enables us to help patients throughout their treatment journey," said Saqib Islam, Chief Executive Officer of SpringWorks. "In parallel with our U.S. launches, we are working with urgency to bring our medicines to patients globally and are advancing a diversified pipeline across a variety of indications that provide the potential for us to develop important therapeutic advances for patients who are currently underserved."

Recent Business Highlights and Upcoming Milestones

OGSIVEO (Nirogacestat)

Continued strong commercial execution of the OGSIVEO launch in the U.S. with fourth quarter and full-year 2024 U.S. net product revenue for OGSIVEO of $61.5 million and $172.0 million, respectively.
A Marketing Authorization Application (MAA) for nirogacestat for the treatment of adult patients with desmoid tumors is under review with the European Medicines Agency (EMA). If approved, SpringWorks expects to launch OGSIVEO following reimbursement authorization in individual EU countries, beginning with Germany in mid-2025.
Presented long-term follow-up data from the Phase 3 DeFi trial of nirogacestat in adults with progressing desmoid tumors at the 2024 Connective Tissue Oncology Society Annual Meeting, which highlighted further reductions in tumor size, increase in objective response rate, sustained improvement in desmoid tumor symptoms, and consistent safety profile. SpringWorks expects to publish these data in a peer-reviewed journal in 2025.
SpringWorks expects to report initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with ovarian granulosa cell tumors in the first half of 2025.
SpringWorks continues to support several industry and academic collaborator studies evaluating nirogacestat as part of B-cell maturation antigen (BCMA) combination therapy regimens across treatment lines in patients with multiple myeloma.
GOMEKLI (Mirdametinib)

On February 11, 2025, SpringWorks received U.S. Food and Drug Administration (FDA) approval for GOMEKLI, an oral MEK inhibitor, for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN. With the approval, SpringWorks was granted a rare pediatric disease priority review voucher (PRV) by the FDA.
GOMEKLI is now available through a specialty pharmacy and specialty distributor network in the United States.
An MAA for mirdametinib for the treatment of adults and children with NF1-PN is under review with the EMA. If approved, SpringWorks expects to begin its initial launch in the European Union in 2025.
A Phase 2 study evaluating mirdametinib in pediatric and young adult patients with low-grade gliomas (LGG) is ongoing and enrolling patients.
Emerging Pipeline

A Phase 1b trial of brimarafenib and Amgen’s EGFR inhibitor, panitumumab, in colorectal and pancreatic cancer patients with known MAPK pathway mutations is ongoing. Brimarafenib is an investigational, selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene, Ltd.
SpringWorks is continuing to enroll patients in a Phase 1 trial of SW-682, an investigational novel, oral, potent, and selective pan-TEAD inhibitor, in Hippo-mutant solid tumors.
SpringWorks obtained an exclusive, global license from Rappta Therapeutics Oy for a first-in-class molecular glue of specific Protein Phosphatase 2A (PP2A) complexes. PP2A mutations represent a class of targetable oncogenic drivers in molecularly defined subsets of uterine cancer patients with high unmet need. In preclinical models of PP2A mutant uterine cancer, SW-3431 (formerly RPT04402) showed rapid, deep and durable tumor regressions as a monotherapy. SpringWorks expects to file an Investigational New Drug (IND) application for SW-3431 by the end of 2025.
Fourth Quarter and Full Year 2024 Financial Results

Product Revenues: OGSIVEO net product revenues were $61.5 million and $172.0 million in the fourth quarter of 2024 and full year 2024, respectively.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $77.1 million and $256.7 million for the fourth quarter and full year 2024, respectively, compared to $59.8 million and $197.6 million for the comparable periods of 2023. The increase in SG&A expense for the fourth quarter and the full year 2024 were largely attributable to commercial readiness activities to support the U.S. launch of GOMEKLI as well as commercial activity supporting the U.S. launch of OGSIVEO.
Research and Development (R&D) Expenses: R&D expenses were $60.2 million and $200.5 million for the fourth quarter and full year 2024, respectively, compared to $43.7 million and $150.5 million for the comparable periods of 2023. The increase in R&D expense for the fourth quarter and year ended 2024 was primarily attributable to an increase in external costs related to licensing fees, drug manufacturing, clinical trials, other research, consulting and professional services.
Net Loss Attributable to Common Stockholders: SpringWorks reported a net loss of $77.3 million, or $1.04 per share, for the fourth quarter of 2024 and a net loss of $258.1 million, or $3.48 loss per share, for the year ended December 31, 2024. This compares to a net loss of $94.3 million, or $1.44 per share, for the fourth quarter of 2023 and a net loss of $325.1 million, or $5.15 per share for the year ended December 31, 2023.
Cash Position: Cash, cash equivalents and marketable securities were $461.9 million as of December 31, 2024.
Additional Information

Additional information on the Company’s results can be found on the Investors and Media section of the SpringWorks website at View Source The previously scheduled conference call and webcast has been cancelled.

On February 20, 2025 ReCerise Therapeutics Inc. ("ReCerise"), a company committed to research and development of first-in-class therapeutics in oncology, reported to have entered into a research partnership with the National Cancer Centre Singapore ("NCCS") to develop innovative treatments utilizing multi-omics data analyses in hepatocellular carcinoma (HCC) (Press release, ReCerise Therapeutics, FEB 20, 2025, View Source [SID1234650438]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This collaboration will be conducted under the PLANet programme (Precision Medicine in Liver Cancer across an Asia-Pacific Network), which was conceptualized and led by NCCS based on the intra-tumoral heterogeneity of liver tumors and its highly dynamic tumor microenvironment (TME). The longitudinal study performs comprehensive multi-omics profiling of tumor and blood samples from liver cancer patients. This collaboration will leverage the processed multi-omics data collected under the PLANet programme, and will be led by Prof. Pierce Chow Kah Hoe, a prominent global expert in the field of HCC. Prof Chow is a Senior Consultant Surgeon in the Division of Surgery and Surgical Oncology at Singapore General Hospital (SGH) and NCCS, and principal investigator of the PLANet programme.

HCC is a highly prevalent cancer with a poor prognosis due to late diagnosis and low response to existing immunotherapeutic treatment options. ReCerise was established with the purpose of investigating and developing new therapeutic modalities to address such unmet medical needs. So far, basic research and proof-of-concept studies on a liver specific protein have demonstrated promise as a potential solution. ReCerise hopes to expand upon this background knowledge by utilizing real-life evidence in collaboration with NCCS to investigate an HCC patient cohort and confirm the target’s expression and its effect on the liver microenvironment.

"We are excited to collaborate with NCCS to accelerate the development of an innovative new drug for the treatment of liver cancer that ReCerise has been working toward," said Yong-Bae Kim, CEO of ReCerise Therapeutics. "Through this joint research, we plan to conduct multi-faceted studies on the development potential of RCT1213, a hepatocellular carcinoma treatment candidate currently under development, by utilizing the various omics data and research database of Asian HCC patients at NCCS."

The research collaboration between these two parties is a research and development project funded by the Korean government and is subject to the management and supervision of the Korea Evaluation Institute of Industrial Technology (KEIT).

On February 20, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported expansion of its oncology pipeline portfolio through the acquisition of assets relating to a next-generation oncolytic virus ICVB-1042 from IconOVir Bio, Inc. (IconOVir) (Press release, UroGen Pharma, FEB 20, 2025, View Source [SID1234650421]). In addition, UroGen also announced that it has entered into multiple strategic research collaborations to explore the potential of its proprietary RTGel technology to enhance clinical effectiveness of multiple immunotherapies, including optimizing dwell time to improve treatment outcomes.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"UroGen’s long-term growth strategy is built on advancing our uro-oncology pipeline, expanding our portfolio, and driving innovation in cancer treatment," said Liz Barrett, President and Chief Executive Officer of UroGen. "The acquisition of ICVB-1042, a next-generation investigational oncolytic virus, marks a significant milestone in our plan to develop novel, locally administered therapies for bladder cancer and other specialty cancers. This strategic investment underscores our commitment to identifying and advancing high-impact therapies that address critical unmet needs. Through targeted acquisitions and research collaborations, we are strengthening UroGen’s leadership in oncology and laying the foundation for sustained innovation and growth."

The use of biological agents to treat bladder cancer has its roots in the development of Bacillus Calmette-Guérin (BCG) therapy. Much like BCG therapy, ICVB-1042 is being developed to activate the immune system within the tumor microenvironment, but unlike BCG therapy, ICVB-1042 has the potential to selectively destroy cancer cells while retaining potency and trigger a robust immune response. A fusion of potency and cancer cell destruction would mark a groundbreaking step forward, unlocking new possibilities in the fight against cancer.

"The treatment of bladder cancer has long been shaped by immunological approaches such as BCG therapy. UroGen now seeks to advance bladder cancer treatment with highly targeted, next-generation viral immunotherapies like ICVB-1042," said Mark Schoenberg, M.D., Chief Medical Officer of UroGen. "ICVB-1042 may represent an exciting leap forward, with several attributes we believe differentiate this asset from other oncolytic viruses. Supported by a robust non-clinical data package, we are eager to develop this investigational therapy as a potential new treatment in localized cancer."

Asset Purchase

On February 14, 2025, UroGen acquired certain assets of IconOVir, including product candidate ICVB-1042, and assumed certain liabilities and obligations of IconOVir arising under certain acquired contracts. As consideration for the assets, UroGen issued IconOVir 374,843 of its ordinary shares (representing a value of approximately $4.0 million based on 30-day VWAP), agreed to pay IconOVir a one-time payment of $15.0 million in cash upon the achievement of a cumulative aggregate worldwide net sales milestone for all products, including combination products, that incorporate or comprise ICVB-1042 (ICVB Products), and agreed to pay IconOVir a certain low, single-digit percentage royalty on a ICVB Product-by-ICVB Product basis on annual worldwide net sales of such ICVB Product.

Conference Call & Webcast Information

Members of UroGen’s management team will host a live conference call and webcast today at 12:00 PM ET to discuss the Company’s long-term growth strategy. The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast.

On February 20, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported multiple oral and poster presentations, including an oral presentation of additional analyses from the ongoing Phase 2 RAMP 201 (ENGOT-ov60/GOG-3052) trial evaluating the investigational combination of avutometinib plus defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC), at the Society of Gynecologic Oncology (SGO) 2025 Annual Meeting on Women’s Cancer, to be held on March 14-17 in Seattle, Washington (Press release, Verastem, FEB 20, 2025, View Source [SID1234650424]). Verastem will also have an exhibition booth (#622) at the meeting where it will be available to discuss its ongoing cancer research.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The presentation of the RAMP 201 primary analysis, which served as the basis of the acceptance of our NDA that is under Priority Review with the FDA, includes additional subgroup analysis by KRAS mutational status," said Dan Paterson, president, and chief executive officer of Verastem Oncology. "We look forward to sharing these learnings with many of the world’s leading gynecologic oncologists at SGO as part of our continued commitment to people living with recurrent low-grade serous ovarian cancer. We also recognize the importance of these findings to the broader cancer community as part of our growing pool of data reinforcing the potential to change expectations in managing RAS/MAPK pathway-driven cancers."

Oral Presentation:

Abstract Title: Avutometinib + Defactinib in Recurrent Low-Grade Serous Ovarian Cancer (ENGOT-ov60/GOG-3052/RAMP 201): Dose Intensity and Subgroup Analysis
Presenter: Rachel Grisham, M.D.
Session: Focused Forum XV: Ongoing IMPACT
Date/Time: Monday, March 17, 2025, 9:15 am PST
Oral Presentation​ – Investigator-Sponsored Trial:

Abstract Title: A Phase II Study of Avutometinib and Defactinib in Advanced or Recurrent Gynecologic Mesonephric Cancer: Interim Results
Presenter: Rachel Grisham, M.D.
Session: Focused Forum IV: Finding IMPACT: The Needle in the Haystack​
Date and Time: Saturday, March 15, 2025, 4:15 pm PST
Preclinical Virtual Poster​:

Abstract Title: Preclinical Efficacy of the Estrogen Receptor Degrader Fulvestrant in Combination with RAF/MEK clamp Avutometinib and FAK Inhibitor in Low-Grade Serous Ovarian Cancer with Acquired Resistance to Chemotherapy and Aromatase Inhibitor
Study Author: Cem Demirkiran, M.D.
About the Avutometinib and Defactinib ​​Combination

Avutometinib is an oral RAF/MEK clamp that potently inhibits MEK1/2 kinase activities and induces inactive complexes of MEK with ARAF, BRAF, and CRAF, potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. In contrast to currently available MEK-only inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of the MEK-only inhibitors.

Defactinib is an oral, selective inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2), the two members of the focal adhesion kinase family of non-receptor protein tyrosine kinases. FAK and Pyk2 integrate signals from integrin and growth factor receptors to regulate cell proliferation, survival, migration, and invasion. FAK activation has been shown to mediate resistance to multiple anti-cancer agents, including RAF and MEK inhibitors.

Verastem Oncology is currently conducting clinical trials with avutometinib with and without defactinib in RAS/MAPK-driven tumors as part of its Raf And Mek Program or RAMP. Verastem is currently enrolling patients and activating sites for RAMP 301 (GOG-3097/ENGOT-ov81/NCRI) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC).

Verastem was granted Priority Review and a Prescription Drug User Fee Act (PDUFA) date of June 30, 2025, for its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), for the investigational combination of avutometinib and defactinib in adults with recurrent KRAS mutant LGSOC who received at least one prior systemic therapy. Verastem initiated a rolling NDA in May 2024 to the FDA and completed its NDA submission in October 2024. The FDA granted Breakthrough Therapy Designation for the treatment of patients with recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy, in May 2021. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC.

Verastem Oncology has established a clinical collaboration with Amgen to evaluate LUMAKRAS (sotorasib) in combination with avutometinib and defactinib in both treatment-naïve patients and in patients whose KRAS G12C mutant non-small cell lung cancer progressed on a G12C inhibitor as part of the RAMP 203 trial (NCT05074810). Verastem has received Fast Track Designation from the FDA for the triplet combination in April 2024. RAMP 205 (NCT05669482), a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer, is supported by the PanCAN Therapeutic Accelerator Award. FDA granted Orphan Drug Designation to the avutometinib and defactinib combination for the treatment of pancreatic cancer.