A Phase 2, Open-label, Multicenter Study to Evaluate the Safety and Efficacy of Belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander Participants With Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic Therapy

The purpose of this study is to measure safety and efficacy of oral belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander male and female participants with cGVHD who have previously been treated with at least 2 prior lines of systemic therapy aged 12 years and above.
The duration of participants participation will be up to 4 weeks for screening, treatment until clinically significant progression of disease, and 4 weeks of safety follow-up, and then long-term follow-up every 12 weeks.1 Cycle = 28 days.

Start Date02 Jul 2024

Sponsor / Collaborator

Kadmon Holdings, Inc.

NCT05922761 / RecruitingPhase 2IIT

An Open-Label, Phase 2 Study to Evaluate the Activity of Belumosudil in Subjects With New Onset and Incipient Bronchiolitis Obliterans Syndrome Following Allogeneic Hematopoietic Cell Transplantation

The goal of this research study is to test the efficacy of a novel immunosuppressive agent, belumosudil, in allogeneic hematopoietic stem cell transplant (HSCT) recipients who have been newly diagnosed or have developing (early stage) bronchiolitis obliterans syndrome (BOS).
The name of the study drugs involved in this study are:
Belumosudil (an immunotherapy)
Fluticasone (an intranasal corticosteroid)
Azithromycin (an antibiotic)
Montelukast (a leukotriene receptor antagonist)
Prednisone (a corticosteroid)

Start Date31 May 2024

Sponsor / Collaborator

Dana-Farber Cancer Institute, Inc.

[+2]

NCT06105554 / RecruitingPhase 1/2IIT

Phase I/II Open Label Study of Belumosudil Mesylate Alone, and in Combination With Dexamethasone, in Patients With Relapsed/Refractory Multiple Myeloma

Phase 1 is to find the recommended dose of belumosudil mesylate that can be given to patients with relapsed/refractory MM.
Phase 2 is to learn if the dose of belumosudil mesylate found in Phase 1 can help to control the disease.

Start Date30 Jan 2024

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

[+1]

100 Clinical Results associated with Belumosudil

100 Translational Medicine associated with Belumosudil

100 Patents (Medical) associated with Belumosudil

Literatures (Medical) associated with Belumosudil

01 Jan 2024·Pharmacological research

Cost in the United States of FDA-approved small molecule protein kinase inhibitors used in the treatment of neoplastic and non-neoplastic diseases

Review

Author: Roskoski, Robert

Because genetic alterations including mutations, overexpression, translocations, and dysregulation of protein kinases are involved in the pathogenesis of many illnesses, this enzyme family is the target of many drug discovery programs worldwide. The FDA has approved 80 small molecule protein kinase inhibitors with 77 drugs orally bioavailable. The data indicate that 69 of these medicinals are approved for the management of neoplasms including solid tumors such as breast and lung cancer as well as non-solid tumors such as leukemia. Moreover, the remaining 11 drugs target non-neoplastic diseases including psoriasis, rheumatoid arthritis, and ulcerative colitis. The cost of drugs was obtained from www.pharmacychecker.com using the FDA label to determine the dosage and number of tablets required per day. This methodology excludes any private or governmental insurance coverage, which would cover the entire cost or more likely a fraction of the stated price. The average monthly cost for the treatment of neoplastic diseases was $17,900 with a price of $44,000 for futibatinib (used to treat cholangiocarcinomas with FGFR2 fusions) and minimum of $5100 for binimetinib (melanoma). The average monthly cost for the treatment of non-neoplastic diseases was $6800 with a maximum of $17,000 for belumosudil (graft vs. host disease) and a minimum of $200 for netarsudil eye drops (glaucoma). There is a negative correlation of the cost of the drugs and the incidence of the targeted disease. Many of these agents are or were designated as orphan drugs meaning that there are fewer than 200,000 potential patients in the United States.

06 Dec 2023·Advances in hematology

Novel Pharmacological Treatment Options of Steroid-Refractory Graft-versus-Host Disease

Review

Author: Chattaraj, Asmi ; Laek, Babray ; Khan, Rafiullah ; Wahab, Ahsan ; Proskuriakova, Ekaterina ; Ali, Rimsha ; Shah, Mitali ; Garg, Chandi ; Kovalenko, Iuliia ; Seyedroudbari, Sara ; Golubykh, Konstantin ; Saleem, Tabinda ; Mirza, Taaha

Background. Graft-versus-host disease (GVHD) is a potentially fatal complication of allogeneic hematopoietic stem cell transplant. The mainstay of treatment is corticosteroids, which are ineffective in 30–50% of cases. Steroid-refractory GVHD (SR-GVHD) confers a poor prognosis, with high mortality rates despite appropriate therapy. While there is no reliable treatment for SR-GVHD, a variety of novel therapeutic options are slowly emerging and have yet to be examined simultaneously. Objectives. This review evaluates the potential of novel therapeutic options, as well as their efficacy and safety, for the treatment of SR-GVHD. Study Design. The literature search was conducted in PubMed, Cochrane, and Embase, employing MeSH terms and keywords. The studies had to be prospective phases 1, 2, or 3. We excluded retrospective and nonoriginal studies. Results. While the only approved drug for acute GVHD is ruxolitinib with an impressive overall response rate of 73.2% and a complete response of 56.3%, several monoclonal antibodies and other agents are currently under investigation, offering promising results. These include anti-CD2, anti-CD147, IL-2 antagonist, a mixture of anti-CD3 and anti-CD7 antibodies, anti-CD25, monoclonal antibody to a4b7 on T-cells, anti-CD26, pentostatin, sirolimus, denileukin diftitox, infliximab, itacitinib, and alpha-1 antitripsin. However, the toxicities associated with these novel drugs need further investigation. For chronic GVHD, approved options include ruxolitinib with an ORR of up to 62%, ibrutinib with an ORR of up to 77%, and belumosudil with an ORR of up to 77%. Meanwhile, emerging treatments include tyrosine kinase inhibitors such as nilotinib, rituximab, and low-dose IL-2, as well as axatilimab and pomalidomide. Conclusion. While their efficacy needs to be better evaluated through large-scale, multicenter, randomized clinical trials, these novel agents show potential and could provide a better alternative for SR-GVHD treatment in the future.

01 Dec 2023·Molecular therapy : the journal of the American Society of Gene Therapy

FUNDC1/PFKP-mediated mitophagy induced by KD025 ameliorates cartilage degeneration in osteoarthritis

Article

Author: Huang, Guiwu ; Liao, Weiming ; Zhang, Zhiqi ; Liu, Ruonan ; Zhang, Chengyun ; Wen, Xingzhao ; Fang, Guibin ; Jiang, Zongrui ; Du, Xue

Osteoarthritis (OA) is the most common joint disease, but no disease-modifying drugs have been approved for OA treatment. Mitophagy participates in mitochondrial homeostasis regulation by selectively clearing dysfunctional mitochondria, which might contribute to cartilage degeneration in OA. Here, we provide evidence of impaired mitophagy in OA chondrocytes, which exacerbates chondrocyte degeneration. Among the several classic mitophagy-regulating pathways and receptors, we found that FUNDC1 plays a key role in preserving chondrocyte homeostasis by inducing mitophagy. FUNDC1 knockdown in vitro and knockout in vivo decreased mitophagy and exacerbated mitochondrial dysfunction, exacerbating chondrocyte degeneration and OA progression. FUNDC1 overexpression via intra-articular injection of adeno-associated virus alleviated cartilage degeneration in OA. Mechanistically, our study demonstrated that PFKP interacts with and dephosphorylates FUNDC1 to induce mitophagy in chondrocytes. Further analysis identified KD025 as a candidate drug for restoring chondrocyte mitophagy by increasing the FUNDC1-PFKP interaction and thus alleviating cartilage degeneration in mice with DMM-induced OA. Our study highlights the role of the FUNDC1-PFKP interaction in chondrocyte homeostasis via mitophagy induction and identifies KD025 as a promising agent for treating OA by increasing chondrocyte mitophagy.

News (Medical) associated with Belumosudil

29 Feb 2024

·www.pharmaceutical-technology.com

NHS expedites availability of Belumosudil for chronic GVHD

The once-daily tablet hinders proteins that trigger the severe inflammatory response linked to chronic GVHD. Credit: fizkes / Shutterstock.com. The National Health Service (NHS) in the UK has expedited the availability of the new drug, Belumosudil (Rezurock), for cancer patients with chronic graft versus host disease (GVHD). The decision provides a treatment that can be administered in the at-home setting, reducing hospital visits. Administered as a once-daily tablet, Belumosudil hinders proteins that trigger the severe inflammatory response linked to chronic GVHD. Clinical trials have demonstrated that up to three-quarters of the subjects responded to the therapy, with significant effectiveness in treating symptoms affecting the mouth, intestine or joints. The treatment is accessible through the NHS England’s Innovative Medicines Fund, which fast-tracks the delivery of the most promising non-cancer drugs to patients. See Also: AbbVie licenses OSE’s chronic inflammation therapy for $48m upfront DNTH-103 by Dianthus Therapeutics for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): Likelihood of Approval Chronic GVHD is a rare but serious condition that can occur after a life-saving stem cell transplant. White blood cells in the donated tissue attack the patient’s organs and weaken their immune system. Approximately one-third of patients who receive stem cell or bone marrow transplants for blood cancers such as leukaemia and lymphoma can develop chronic GVHD. Around 1,200 patients in England undergo such transplants each year. Around 400 go on to develop the condition, which is sometimes mild but for one in ten cases is fatal. For half of patients with chronic GVHD, existing therapies such as pentostatin, rituximab and imatinib are ineffective. Belumosudil will now be made available for 200 severely affected patients who have not responded to two previous therapies. This development comes after the NHS decided to fast-track the funding for the drug based on clinical guidance from the UK National Institute of Health and Care Excellence ( Nice ). Belumosudil is the fourth therapy offered to patients through the Innovative Medicines Fund. In September 2023, the NHS used the fund to roll out Sebelipase alfa, a therapy for treating Wolman’s disease, a rare genetic ailment. NHS England specialised services medical director James Palmer stated: “This rollout is a real breakthrough for patients with graft versus host disease and it will help people living with this debilitating condition effectively manage their symptoms and enhance their quality of life and daily functioning. “It’s great news that the NHS has been able to fast-track funding for a treatment which will have a significant impact for hundreds of patients over the coming years.”

Clinical Result

28 Feb 2024

·www.pharmaceutical-technology.com

AbbVie licenses OSE’s chronic inflammation therapy for $48m upfront

OSE-230 is a ChemR23, a G-Protein coupled receptor (GPCR) targeting monoclonal antibody. Image Credit: Poetra.RH / Shutterstock. AbbVie has signed a partnership and licensing agreement with OSE Immunotherapeutics for the latter’s monoclonal antibody OSE-230 in a deal worth up to $713m. OSE-230 is a monoclonal antibody that targets ChemR23, a G-Protein coupled receptor (GPCR). The therapy can potentially resolve chronic inflammation and modulate the function of macrophages and neutrophils. It is currently in preclinical development. Following the news, OSE’s stock was up by more than 55% in trading today. The French pharmaceutical company’s market cap stands at $111.2m. Under the collaboration agreement, AbbVie will pay $48m in upfront payment for the exclusive global licence of the therapy. OSE will also be in line to receive up to $665m in milestone-based payments along with tiered royalties on net sales. OSE has a pipeline of multiple monoclonal antibody candidates that it is developing in collaboration with other pharmaceutical companies. The company is developing two signal regulatory protein α (SIRPα) targeting monoclonal antibodies—BI 765063/ OSE-172 and BI 770371—in collaboration with Boehringer Ingelheim. See Also: NHS expedites availability of Belumosudil for chronic GVHD Beacon Therapeutics unveils promising interim results for gene therapy AGTC-501 BI 765063 is being evaluated as a combination therapy with different cancer therapies such as Boehringer’s ezabenlimab, BI 836880, chemotherapy, and Eli Lilly’s Erbitux (cetuximab) in a Phase I trial (NCT05249426). BI 770371 is being evaluated as a monotherapy and in combination with a PD1 inhibitor, BI 754091, in an international Phase I dose escalation and expansion trial (NCT05327946) in patients with solid tumours. OSE is also developing an anti-CD28 selective monoclonal antibody FR104/VEL-101 in partnership with Denmark-based Veloxis Pharmaceuticals. It is being researched as a treatment to help prevent organ rejection in people who have had a kidney transplant. In February 2022, the therapy received a fast track designation by the FDA for prophylaxis against transplant rejection. Veloxis is planning Phase II trials for the subcutaneous therapy. AbbVie has also made recent investments related to antibody therapies. In December 2023, it partnered with BigHat Biosciences to identify and develop antibody therapies in oncology and neuroscience indications. On a larger scale, in November 2023, AbbVie announced a $10.1bn ImmunoGen acquisition deal . The antibody-drug conjugate (ADC) Elahere (mirvetuximab soravtansine-gynx) was a central part of the deal. The therapy was granted priority review by the US Food and Drug Administration with a Prescription Drug User Fee Act (PDUFA) action date of 5 April 2024.

Phase 1License out/inPriority ReviewFast TrackADC

01 Feb 2024

·www.globenewswire.com

Press Release: Sanofi Delivers Solid Sales and Business EPS Growth at CER Amidst Successful Product Launches and Advancements in Immunology Pipeline

Sanofi Delivers Solid Sales and Business EPS Growth at CER Amidst Successful Product Launches and Advancements in Immunology Pipeline Paris, February 1, 2024 Q4 2023 sales growth of 9.3% at CER and business EPS(1) increase of 8.2% at CER Specialty Care grew 13.7% driven by Dupixent and ALTUVIIIO launch performance, more than offsetting competition from generics of Aubagio in all key marketsVaccines sales increased strongly (up 21.1%) mainly as a result of the unprecedented uptake of Beyfortus, reaching €410 million in the second quarter of its launch General Medicines decline moderated (down 2.4%), reflecting accelerated growth of core assets (up 6.3%), offset by lower sales of Lantus and non-core asset divestmentCHC rose 8.5% due to Digestive Wellness and Physical and Mental Wellness which benefited from Qunol acquisition Full-year 2023 delivered 5.3% sales growth and 5.4% business EPS growth at CER Sales reached €43,070 million driven by Dupixent (€10,715 million, +34.0%, adding €2.8bn at CER), Vaccines (up 8.3%) which benefited from strong launch performance of Beyfortus (€547 millions) and CHC (+6.3%)Business EPS(1) of €8.11 down 1.8% on a reported basis and up 5.4% at CER IFRS EPS of €4.31 (down 35.6%) mainly reflecting an impairment loss of technology assets resulting from de-prioritization of R&D programs and a charge related to the liability remeasurement of expected future royalty payments on U.S. Beyfortus sales, which mainly occurred in Q4Board held on January 31, proposes annual dividend of €3.76 an increase of 5.6% R&D transformation and Key milestones in Q4 Strong pipeline including record 12 blockbuster opportunities under clinical evaluation detailed at recent R&D DayDupixent submitted for COPD (Chronic Obstructive Pulmonary Disease) in the U.S., EU, and ChinaSarclisa delivered positive phase 3 results in 1L transplant-ineligible Multiple Myeloma (IMROZ) Progress on Corporate Social Responsibility strategy in Q4 Sanofi Global Health Unit announced three new investments through its Impact Fund to support healthcare start-ups in Sub-Saharan AfricaSanofi at COP28: supporting the decarbonization of healthcare systems Full-year 2024 business EPS guidance Sanofi expects 2024 business EPS(1) to remain roughly stable excluding the impact of an expected effective tax rate increase to 21% and decrease low single-digit(2) at CER including the higher expected tax rate, barring unforeseen major adverse events. Applying average January 2024 exchange rates, the currency impact on 2024 business EPS is estimated between -3.5% to -4.5%. Paul Hudson, Sanofi Chief Executive Officer, commented: “2023 marked a critical year on our journey to become a development-driven, tech-powered biopharma company committed to serving patients and accelerating growth. We have delivered another year of strong underlying performance of our core drivers in Specialty Care and Vaccines supported by the outstanding launch execution of Beyfortus, Altuviiio and Tzield. With scientific news flow at an all-time high, pipeline advances and 12 potential blockbusters in late-stage development including amlitelimab, frexalimab and tolebrutinib, our R&D transformation has reached an inflection point on the road to industry leadership in immunology. Looking forward, we remain committed to investing in R&D to fully unlock the value of our pipeline, powered by AI at scale, and continue to focus on our expected launch opportunities such as Dupixent in COPD. At the same time, we are taking steps to become a pure-play biopharma company with more than €10bn sales contribution from Pharma launches by 2030(4).” Q4 2023 Change Change at CER 2023 Change Change at CER IFRS net sales reported €10,919m +1.8% +9.3% €43,070m +0.2% +5.3% IFRS net income reported -€555m -117.8% _ €5,400m -35.5% — IFRS EPS reported -€0.44 -117.7% _ €4.31 -35.6% — Free cash flow(3) €3,496m +37.3% _ €8,478m -0.1% — Business operating income €2,583m -5.2% +5.3% €12,670m -2.8% +4.3% Business net income(1) €2,083m -2.7% +8.2% €10,155m -1.8% +5.5% Business EPS(1) €1.66 -2.9% +8.2% €8.11 -1.8% +5.4% Changes in net sales are expressed at constant exchange rates (CER) unless otherwise indicated (definition in Appendix 9). (1) In order to facilitate an understanding of operational performance, Sanofi comments on the business net income statement. Business net income is a non-IFRS financial measure (definition in Appendix 9). The consolidated income statement for Q4 2023 is provided in Appendix 3 and a reconciliation of reported IFRS net income to business net income is set forth in Appendix 4; (2) 2023 business EPS was €8.11; (3) Free cash flow is a non-IFRS financial measure (definition in Appendix 9). (4) Risk-adjusted net sales at CER . Already launched: ALTUVIIIO, TZIELD, Sarclisa, Nexviazyme, Rezurock, Xenpozyme, Enjaymo, Cablivi; Potential launches tolebrutinib, itepekimab, amlitelimab, frexalimab, rilzabrutinib, lunsekimig, Oral TNFR1si Attachment Press_Release

AcquisitionClinical ResultPhase 3Financial StatementBiosimilar

100 Deals associated with Belumosudil

External Link

KEGG	Wiki	ATC	Drug Bank
D11815	-	-	-

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Chronic graft-versus-host disease	US	Kadmon Pharmaceuticals LLC	16 Jul 2021

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Allograft Rejection	Phase 3	US	Sanofi	10 Oct 2023
Allograft Rejection	Phase 3	BE	Sanofi	10 Oct 2023
Allograft Rejection	Phase 3	CZ	Sanofi	10 Oct 2023
Allograft Rejection	Phase 3	FI	Sanofi	10 Oct 2023
Allograft Rejection	Phase 3	FR	Sanofi	10 Oct 2023
Allograft Rejection	Phase 3	HU	Sanofi	10 Oct 2023
Allograft Rejection	Phase 3	IL	Sanofi	10 Oct 2023
Allograft Rejection	Phase 3	IT	Sanofi	10 Oct 2023
Allograft Rejection	Phase 3	NL	Sanofi	10 Oct 2023
Allograft Rejection	Phase 3	ES	Sanofi	10 Oct 2023

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EHA2024	Not Applicable	Graft vs Host Disease	35	Belumosudil	jqhcpwkanh(oxnekqvidc) = 2 patients where additional treatment for cGvHD had to be initiated avvwentpgw (amsttqakjc ) View more	Positive	14 May 2024
Phase 3 Japanese Study of Belumosudil (EHA2024)	Phase 3	Chronic graft-versus-host disease	21	Belumosudil tablets 200 mg	edfkpejuks(awtbyavxjw) = ucnggopxyl yenhpkynhz (oxejhmuajf ) View more	Positive	14 May 2024
NCT04930562 (phase II study of belumosudil, Pubmed)	Phase 2	Graft vs Host Disease Second line	30	Belumosudil 200 mg	lcbxlibrvr(avtajnppjg) = rxxjsifaop xchrifqjoc (zapkyoxyty, 54.1 - 87.7)	Positive	26 Mar 2024
NCT04930562 (CTgov)	Phase 2	Chronic graft-versus-host disease	30	BN101	xlcsnefnkx(sxknaqmcka) = bkmgjpbxmu accnocyxzi (uptvchcxmv, wkyuamenrz - bbjhachkwb) View more	-	01 Nov 2023
NCT03919799 (CTgov)	Phase 2	Scleroderma, Diffuse	36	Placebo (DB Period: Placebo)	hwcxpidjep(sffoumlnoi) = jyjnuyjchc hsdygndkks (fuojwnoniv, lajownlasw - qvqqeansft) View more	-	30 Aug 2023
				Belumosudil (KD025) (OLE Period: Placebo/Belumosudil QD)	nebwyvtxly(tvcikqwxae) = whsayfaqzb iurxsnuofu (errzxeldhh, vlseccpzhz - ejhhdchlch) View more
NCT02841995 (KD025-208, CTgov)	Phase 2	Chronic graft-versus-host disease	54	Belumosudil (KD025) (Cohort 1: Belumosudil 200 mg QD)	dcbasnzlin(pqstcmkwvz) = knqtktmrmw ortbnhnaoh (evelcutnvz, xopwqjcaex - ejvsogblsh) View more	-	28 Jun 2023
				Belumosudil (KD025) (Cohort 2: Belumosudil 200 mg BID)	dcbasnzlin(pqstcmkwvz) = ggotftcvwp ortbnhnaoh (evelcutnvz, xqinkwasvw - cuybrxmrva) View more
NCT04680975 (CTgov)	Phase 2	Scleroderma, Diffuse	10	Belumosudil	bqjujojmhc(pbgvwjhwcl) = yldjxmikgd qvwsglxrox (amiemkojnp, elwonioold - cwaxtjjtsr) View more	-	05 Jun 2023
NCT02688647 (KD025-207, CTgov)	Phase 2	Idiopathic Pulmonary Fibrosis	76	Belumosudil (Belumosudil-R)	szbxsqulrc(efgohoeycw) = tupkipvqrk pzziiudvhl (ibeymmykeh, mdcduwjpvu - cqtcidzvwn) View more	-	08 Sep 2022
				BSC (BSC-R)	szbxsqulrc(efgohoeycw) = hexiiiopyw pzziiudvhl (ibeymmykeh, zozuqhquvy - alixhxdzko) View more
NCT03640481 (ROCKstar study, EBMT2022)	Phase 2	Graft vs Host Disease	-	Belumosudil 200 mg QD	dzkmqohdpa(apixcrhrvh) = 4% ixanesjzki (mejdmlbmnb ) View more	Positive	19 Mar 2022
				Belumosudil 200 mg BID
NCT03640481 (ROCKstar, Pubmed \| Circulation) Manual	Phase 2	Graft vs Host Disease Third line	132	belumosudil (200 mg daily)	kohsnuleew(nmxkwixslf) = yvmgbhznvb xhdtobvkcq (szltcmahiu, 62 ~ 84)	Positive	02 Dec 2021
				belumosudil (200 mg twice daily)	kohsnuleew(nmxkwixslf) = rrqxpjwmjv xhdtobvkcq (szltcmahiu, 65 ~ 87)

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