This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back (relapsed) after previous treatment, or does not respond to therapy (refractory). Langerhans cell histiocytosis is a type of disease that occurs when the body makes too many immature Langerhans cells (a type of white blood cell). When these cells build up, they can form tumors in certain tissues and organs including bones, skin, lungs and pituitary gland and can damage them. This tumor is more common in children and young adults. DAY101 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Using DAY101 may be effective in treating patients with relapsed or refractory Langerhans cell histiocytosis.

Start Date28 Mar 2024

Sponsor / Collaborator

National Cancer Institute

NCT06381570 / RecruitingEarly Phase 1IIT

VICTORY: A Pilot Study to Investigate Safety and Efficacy of Weekly Combination of Intravenous Vinblastine With Oral Type II RAF Inhibitor Tovorafenib in Pediatric Patients With Recurrent/Progressive RAF Altered Low Grade Gliomas

This is a Pilot, multicenter, open-label study of patients less than or equal to 25 years, with recurrent or progressive LGG harboring a CRAF or BRAF alteration, including BRAF V600 mutations and KIAA1549: BRAF fusions. Patients with BRAF or CRAF alterations will be identified through molecular assays as routinely performed at Clinical Laboratory Improvement Amendments (CLIA) of 1988 or other similarly certified laboratories.
The study will be conducted in two sequential phases:
Phase A: A Feasibility (combination dose finding) phase, followed by Phase B: An Efficacy phase. The maximum tolerated dose (MTD)/Recommended Phase 2 Dose (RP2D) of the combination as determined in Phase A would be the dose used in Phase B. The patients on Phase A who were below the MTD/RP2D would be eligible for intra-patient dose escalation to MTD/RP2D subject to criteria outlined later

Start Date21 Mar 2024

Sponsor / Collaborator

The Hospital for Sick Children

[+1]

NCT05566795 / RecruitingPhase 3

LOGGIC/FIREFLY-2: A Phase 3, Randomized, International Multicenter Trial of DAY101 Monotherapy Versus Standard of Care Chemotherapy in Patients With Pediatric Low-Grade Glioma Harboring an Activating RAF Alteration Requiring First-Line Systemic Therapy

This is a 2-arm, randomized, open-label, multicenter, global, Phase 3 trial to evaluate the efficacy, safety, and tolerability of tovorafenib monotherapy versus standard of care (SoC) chemotherapy in patients with pediatric low-grade glioma (LGG) harboring an activating rapidly accelerated fibrosarcoma (RAF) alteration requiring front-line systemic therapy.

Start Date27 Feb 2023

Sponsor / Collaborator

Day One Biopharmaceuticals, Inc.

100 Clinical Results associated with Tovorafenib

100 Translational Medicine associated with Tovorafenib

100 Patents (Medical) associated with Tovorafenib

Literatures (Medical) associated with Tovorafenib

01 Feb 2024·Nature reviews. Clinical oncology

Tovorafenib effective against low-grade gliomas harbouring BRAF fusions

Article

Author: Sidaway, Peter

01 Jan 2024·Nature medicineQ1 · MEDICINE

The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Q1 · MEDICINE

Article

Author: Cornelio, Izzy ; Hassall, Timothy E ; Baxter, Patricia A ; Manley, Peter ; Jabado, Nada ; Gerber, Nicolas U ; Larouche, Valérie ; Hargrave, Darren ; Kang, Hyoung Jin ; Bailey, Simon ; Kilburn, Lindsay B ; van der Lugt, Jasper ; Toledano, Helen ; Segal, Devorah ; Han, Jung Woo ; Oren, Liat ; Nysom, Karsten ; Whipple, Nicholas S ; Franson, Andrea T ; Abdelbaki, Mohamed S ; Perreault, Sébastien ; Kline, Cassie ; Ziegler, David S ; Mueller, Sabine ; McLeod, Lisa ; Da Costa, Daniel ; Walter, Ashley ; Zhao, Xin ; Yalon Oren, Michal ; Witt, Olaf ; Packer, Roger J ; Hansford, Jordan R ; McCowage, Geoffrey ; Blackman, Samuel C ; Gottardo, Nicholas G ; Khuong-Quang, Dong-Anh ; Chi, Susan N ; Driever, Pablo Hernáiz ; Waanders, Angela J ; Landi, Daniel ; Leary, Sarah E S ; Tan, Enrica E K

Abstract:

BRAF genomic alterations are the most common oncogenic drivers in pediatric low-grade glioma (pLGG). Arm 1 (n = 77) of the ongoing phase 2 FIREFLY-1 (PNOC026) trial investigated the efficacy of the oral, selective, central nervous system–penetrant, type II RAF inhibitor tovorafenib (420 mg m−2 once weekly; 600 mg maximum) in patients with BRAF-altered, relapsed/refractory pLGG. Arm 2 (n = 60) is an extension cohort, which provided treatment access for patients with RAF-altered pLGG after arm 1 closure. Based on independent review, according to Response Assessment in Neuro-Oncology High-Grade Glioma (RANO-HGG) criteria, the overall response rate (ORR) of 67% met the arm 1 prespecified primary endpoint; median duration of response (DOR) was 16.6 months; and median time to response (TTR) was 3.0 months (secondary endpoints). Other select arm 1 secondary endpoints included ORR, DOR and TTR as assessed by Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO) criteria and safety (assessed in all treated patients and the primary endpoint for arm 2, n = 137). The ORR according to RAPNO criteria (including minor responses) was 51%; median DOR was 13.8 months; and median TTR was 5.3 months. The most common treatment-related adverse events (TRAEs) were hair color changes (76%), elevated creatine phosphokinase (56%) and anemia (49%). Grade ≥3 TRAEs occurred in 42% of patients. Nine (7%) patients had TRAEs leading to discontinuation of tovorafenib. These data indicate that tovorafenib could be an effective therapy for BRAF-altered, relapsed/refractory pLGG. ClinicalTrials.gov registration: NCT04775485.

04 Aug 2023·Cancer discovery

Targeted Options for Glioma Looking Good.

Article

According to the phase III INDIGO trial, vorasidenib, an IDH1/2 inhibitor, significantly benefited adults with IDH1/2-mutant low-grade gliomas, reducing progression risk and delaying the need for chemoradiotherapy. Meanwhile, in a pediatric low-grade glioma cohort of FIREFLY-1, a phase II trial, robust responses to the type II pan-RAF inhibitor tovorafenib were seen.

News (Medical) associated with Tovorafenib

20 Jun 2024

·www.globenewswire.com

XOMA Declares Quarterly Preferred Stock Dividends

EMERYVILLE, Calif., June 20, 2024 (GLOBE NEWSWIRE) -- XOMA Corporation (Nasdaq: XOMA) (“XOMA” or the “Company”) today announced its Board of Directors has authorized the following cash dividends to holders of XOMA’s Series A and Series B Cumulative Preferred Stock: Holders of the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) shall receive a cash dividend equal to $0.53906 per share. Holders of depositary shares, each representing 1/1000 of a share of XOMA’s 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO), shall receive a cash dividend equal to $0.52344 per depositary share. The preferred dividends will be paid on or about July 15, 2024, to respective holders of record at the close of business on July 3, 2024. About XOMA CorporationXOMA is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA acquires the potential future economics associated with pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit www.xoma.com or follow the Company on LinkedIn. Forward-Looking Statements/Explanatory NotesCertain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify such forward-looking statements by terminology such as “will” or “shall”, the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA’s performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry, including those related to the fact that our product candidates subject to out-license agreements are still being developed, and our licensees may require substantial funds to continue development which may not be available; we do not know whether there will be, or will continue to be, a viable market for the products in which we have an ownership or royalty interest; and if the therapeutic product candidates to which we have a royalty interest do not receive regulatory approval, our third-party licensees will not be able to market them. Other potential risks to XOMA meeting these expectations are described in more detail in XOMA's most recent filing on Form 10-Q and in other SEC filings. Consider such risks carefully when considering XOMA's prospects. Any forward-looking statement in this press release represents XOMA's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA disclaims any obligation to update any forward-looking statement, except as required by applicable law. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. Investor contact:Juliane SnowdenXOMA+1-646-438-9754juliane.snowden@xoma.com Media contact:Kathy VincentKV Consulting & Management+1-310-403-8951kathy@kathyvincent.com

20 Jun 2024

·www.biospace.com

XOMA Declares Quarterly Preferred Stock Dividends - June 20, 2024

EMERYVILLE, Calif., June 20, 2024 (GLOBE NEWSWIRE) -- XOMA Corporation (Nasdaq: XOMA) (“XOMA” or the “Company”) today announced its Board of Directors has authorized the following cash dividends to holders of XOMA’s Series A and Series B Cumulative Preferred Stock: Holders of the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) shall receive a cash dividend equal to $0.53906 per share. Holders of depositary shares, each representing 1/1000 of a share of XOMA’s 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO), shall receive a cash dividend equal to $0.52344 per depositary share. The preferred dividends will be paid on or about July 15, 2024, to respective holders of record at the close of business on July 3, 2024. About XOMA Corporation XOMA is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA acquires the potential future economics associated with pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit or follow the Company on LinkedIn. Forward-Looking Statements/Explanatory Notes Certain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify such forward-looking statements by terminology such as “will” or “shall”, the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA’s performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry, including those related to the fact that our product candidates subject to out-license agreements are still being developed, and our licensees may require substantial funds to continue development which may not be available; we do not know whether there will be, or will continue to be, a viable market for the products in which we have an ownership or royalty interest; and if the therapeutic product candidates to which we have a royalty interest do not receive regulatory approval, our third-party licensees will not be able to market them. Other potential risks to XOMA meeting these expectations are described in more detail in XOMA's most recent filing on Form 10-Q and in other SEC filings. Consider such risks carefully when considering XOMA's prospects. Any forward-looking statement in this press release represents XOMA's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA disclaims any obligation to update any forward-looking statement, except as required by applicable law. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. Investor contact: Juliane Snowden XOMA +1-646-438-9754 juliane.snowden@xoma.com Media contact: Kathy Vincent KV Consulting & Management +1-310-403-8951 kathy@kathyvincent.com

20 Jun 2024

·www.pharmamanufacturing.com

Day One inks PTK7-targeting ADC licensing deal with MabCare

Day One Biopharmaceuticals has announced an exclusive licensing agreement with MabCare Therapeutics for MTX-13, a novel antibody-drug conjugate (ADC) targeting protein-tyrosine kinase 7 (PTK7). Under the terms of the agreement, Shanghai-based MabCare will receive $55 million upfront and up to $1.152 billion in additional milestones, along with royalties on net sales outside Greater China. Day One aims to dose the first patient in a phase 1 study by late 2024 or early 2025. The compound, now designated as DAY301, received IND clearance from the FDA in April 2024. It targets PTK7, a transmembrane protein highly expressed in various solid tumors, including esophageal, ovarian, lung, and endometrial cancers in adults, as well as pediatric cancers such as neuroblastoma and osteosarcoma. Preclinical studies have shown that DAY301 has significant antitumor activity, making it a promising candidate for treating both adult and pediatric cancers. Day One Biopharmaceuticals, headquartered in Brisbane, California, focuses on developing targeted cancer therapies. The company’s pipeline includes recently approved tovorafenib (branded Ojemda) and pimasertib, currently in phase 1/2 trials. ADCs work by combining the targeting ability of antibodies with the potent cell-killing power of chemotherapy drugs. This allows for the direct delivery of toxic agents to cancer cells, minimizing damage to healthy cells and potentially reducing side effects compared to traditional chemotherapy. The ADC market is rapidly growing , with several ADCs already approved by regulatory agencies worldwide and many more in clinical development.

License out/inADCDrug Approval

100 Deals associated with Tovorafenib

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15266

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
BRAF fusion or rearrangement postive low-grade glioma	US	Day One Biopharmaceuticals, Inc.	23 Apr 2024
BRAF V600 mutation low-grade glioma	US	Day One Biopharmaceuticals, Inc.	23 Apr 2024

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Low grade glioma	Phase 3	AU	Day One Biopharmaceuticals, Inc.	27 Feb 2023
Low grade glioma	Phase 3	AT	Day One Biopharmaceuticals, Inc.	27 Feb 2023
Low grade glioma	Phase 3	BE	Day One Biopharmaceuticals, Inc.	27 Feb 2023
Low grade glioma	Phase 3	CA	Day One Biopharmaceuticals, Inc.	27 Feb 2023
Low grade glioma	Phase 3	CZ	Day One Biopharmaceuticals, Inc.	27 Feb 2023
Low grade glioma	Phase 3	DK	Day One Biopharmaceuticals, Inc.	27 Feb 2023
Low grade glioma	Phase 3	FI	Day One Biopharmaceuticals, Inc.	27 Feb 2023
Low grade glioma	Phase 3	FR	Day One Biopharmaceuticals, Inc.	27 Feb 2023
Low grade glioma	Phase 3	DE	Day One Biopharmaceuticals, Inc.	27 Feb 2023
Low grade glioma	Phase 3	GR	Day One Biopharmaceuticals, Inc.	27 Feb 2023

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03429803 (NCT03429803, ASCO2024)	Phase 1	MAPK positive Neoplasms KIAA1549:BRAF \| BRAF V600E- \| FGFR1	35	Tovorafenib 530 mg/m2	cxjtwutrlr(tsuufwhbal) = decreased GV was observed in pre-pubertal patients on drug and warrants further investigation to understand the mechanism of action xtxryrgyer (zsumwsypnk ) View more	Positive	24 May 2024
NCT04775485 (FIREFLY-1, FDA_CDER) Manual	Phase 2	BRAF fusion or rearrangement postive low-grade glioma \| BRAF V600 mutation low-grade glioma BRAF V600E \| BRAF Rearrangement	137	OJEMDA	pbmfmlgfbg(vhiddigtxk) = vfuliwgziq cvnuipaizk (efzngvstbo, 40-63) View more	Positive	23 Apr 2024
NCT04775485 (FIREFLY-1, ASCO2023) Manual	Phase 2	Low grade glioma	77	Tovorafenib 420 mg/m2	tcxlmupzpf(swbeziyuke) = ttlxyyibms kbaogruesg (tsvxeppizm ) View more	Positive	31 May 2023
NCT04775485 (FIREFLY-1, Corporate Publications) Manual	Phase 2	Low grade glioma	22	Tovorafenib	dwlgzpvdnl(umziwepsfe) = zcaezxadbn bcczkkefxy (dizqkdmltd ) View more	Positive	12 Jun 2022
NCT02723006 (CTgov)	Phase 1	Melanoma	22	Nivolumab+TAK-580 (Arm 1: TAK-580 + Nivolumab)	bpmtmtfsqo(yptijsqeka) = fcyxfsfvxl nochjodfpf (miflvwosjv, hvkmtarwrq - xlselxhdtr) View more	-	01 Apr 2021
NCT02723006 (CTgov)	Phase 1	Melanoma	22	Nivolumab+TAK-202 (Arm 2: TAK-202 + Nivolumab)	bpmtmtfsqo(yptijsqeka) = aegwwrkerp nochjodfpf (miflvwosjv, wqpuetbibr - edlsqieamw) View more	-	01 Apr 2021

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