ASH 2023 | TRANSFORM-1: navitoclax + ruxolitinib vs placebo + ruxolitinib in patients with untreated MF
Naveen Pemmaraju, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, presents initial findings from TRANSFORM-1 (NCT04472598), a Phase III trial investigating navitoclax in combination with ruxolitinib in patients with previously untreated myelofibrosis (MF). The trial successfully met its primary endpoint, demonstrating an improvement in spleen volume reduction (SVR) in patients treated with navitoclax plus ruxolitinib, and the combination was well-tolerated. This interview took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript (edited for clarity)
The TRANSFORM-1 study was a large Phase III randomized global study, and I’m really proud that we were able to present that here at ASH for the very first time. It featured a combination for the first time of ruxolitinib with a novel agent, Bcl/xL inhibitor known as navitoclax, frontline upfront treated patients, they’ve never seen a JAK inhibitor, versus ruxolitinib, which is the standard-of-care in our field JAK inhibitor, plus placebo...
The TRANSFORM-1 study was a large Phase III randomized global study, and I’m really proud that we were able to present that here at ASH for the very first time. It featured a combination for the first time of ruxolitinib with a novel agent, Bcl/xL inhibitor known as navitoclax, frontline upfront treated patients, they’ve never seen a JAK inhibitor, versus ruxolitinib, which is the standard-of-care in our field JAK inhibitor, plus placebo.
252 patients were enrolled, and it was adults with ECOG performance status of two or less. These patients had to have three characteristics: no JAK inhibitor before, so untreated, number two splenomegaly, and number three, a symptom burden that could be measured. We randomized those patients 1 to 1 and we found that in the combination arm compared to the control arm, that the spleen size reduction was doubled compared to the control arm. So 60 plus percent in the combination navitoclax/rux versus only 31.5% in the control arm. What does that mean? That means the primary endpoint of this large Phase III randomized, double blind, controlled study was met. But there are also key secondary endpoints and that included symptom burden and durability of spleen response. Some of those measures are still ongoing at this time.
The second part of this study was the fact that we were able to conduct it. A randomized study, global international sites largely done during the COVID-19 pandemic, and so we were very happy about that.
One last point is it’s a very well tolerated clinical regimen. The side effects were expected. They included cytopenia, so thrombocytopenia, anemia, neutropenia. And the most non-hematologic common side effect was that of diarrhea. We found these to be manageable with dose interruptions reductions all specified by the protocol.
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Disclosures
Consultancy: ClearView Healthcare Partners, Imedex, Protagonist Therapeutics Inc., EUSA Pharma, Curio Science, CareDx, Harborside Press, Dava Oncology, CancerNet, Aplastic Anemia & MDS International Foundation, Pacylex, Patient Power, Menarini Group, Astellas, BMS, PeerView Institute for Medical Education, Stemline, Medscape, OncLive, Physician Education Resource (PER), Celgene, PharmaEssentia, Novartis Pharmaceuticals:, Magdalen Medical Publishing, Neopharm, Intellisphere, Blueprint, Cimeio Therapeutics AG, ImmunoGen, CTI BioPharma, Incyte, Aptitude Health, AbbVie, Bristol Myers Squibb Pharmaceuticals
Membership on Board of Directors/Advisory Committees: ClearView Healthcare Partners, Imedex, Protagonist Therapeutics Inc., EUSA Pharma, Curio Science, Dan’s House of Hope, CareDx, Harborside Press, Dava Oncology, CancerNet, Aplastic Anemia & MDS International Foundation, Pacylex, Patient Power, Menarini Group, Astellas, BMS, PeerView Institute for Medical Education, Stemline, Medscape, OncLive, Physician Education Resource (PER), Celgene, PharmaEssentia, Novartis Pharmaceuticals:, Magdalen Medical Publishing, Neopharm, Intellisphere, Blueprint, Cimeio Therapeutics AG, ImmunoGen, CTI BioPharma, Incyte, Aptitude Health, AbbVie, Bristol Myers Squibb Pharmaceuticals
Speakers Bureau: ClearView Healthcare Partners, Imedex, EUSA Pharma, Curio Science, CareDx, Harborside Press, Dava Oncology, CancerNet, Aplastic Anemia & MDS International Foundation, Pacylex, Patient Power, Menarini Group, Astellas, BMS, PeerView Institute for Medical Education, Medscape, OncLive, Physician Education Resource (PER), Celgene, PharmaEssentia, Novartis Pharmaceuticals:, Magdalen Medical Publishing, Neopharm, Intellisphere, Blueprint, Cimeio Therapeutics AG, ImmunoGen, CTI BioPharma, Incyte, Aptitude Health, AbbVie, Bristol Myers Squibb Pharmaceuticals
Research Funding: United States Department of Defense (DOD), National Institute of Health/National Cancer Institute (NIH/NCI)
Licenses: Karger Publishers
Leadership role on ASH Committee on Communications and ASCO Cancer.Net Editorial Board
