Myeloproliferative Disorders

Company to host a conference call tomorrow at 8:00 a.m. ET NEWTON, Mass., Oct. 30, 2024 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced it will host a conference call and audio webcast at 8:00 a.m. ET on Thursday, October 31, 2024 to provide a favorable study design update on the Company's pivotal Phase 3 SENTRY study in JAKi naive myelofibrosis. The call will feature leading myelofibrosis key opinion leaders Dr. Raajit Rampal, Director of the Center for Hematologic Malignancies and Director of the Myeloproliferative Neoplasms Program at Memorial Sloan Kettering Cancer Center and Dr. John Mascarenhas, principal investigator of the Phase 3 SENTRY trial, Professor of Medicine at the Icahn School of Medicine at Mount Sinai and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders. To access the conference call, please dial (800) 836-8184 (local) or (646) 357-8785 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company's website, . An archived webcast will be available on the Company's website approximately two hours after the event. About Karyopharm Therapeutics Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company whose dedication to pioneering novel cancer therapies is fueled by a belief in the extraordinary strength and courage of patients with cancer. Since its founding, Karyopharm has been an industry leader in oral compounds that address nuclear export dysregulation, a fundamental mechanism of oncogenesis. Karyopharm's lead compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and marketed by the Company in three oncology indications. It has also received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®) and China. Karyopharm has a focused pipeline targeting indications in multiple high unmet need cancers, including in multiple myeloma, endometrial cancer, myelofibrosis, and diffuse large B-cell lymphoma (DLBCL). For more information about our people, science and pipeline, please visit , and follow us on LinkedIn and on X at @Karyopharm. XPOVIO® and NEXPOVIO® are registered trademarks of Karyopharm Therapeutics Inc. SOURCE Karyopharm Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

In the ASC4FIRST trial, Scemblix showed superior MMR rates at week 48 compared to standard-of-care TKIs and imatinib alone. Credit: Elle Aon/Shutterstock. Novartis has received US Food and Drug Administration (FDA) accelerated approval for Scemblix (asciminib) to treat adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukaemia (CML) in its chronic phase (Ph+ CML-CP). Scemblix, a “specifically targeting the ABL myristoyl pocket” (STAMP) inhibitor, is the first CML therapy to demonstrate efficacy along with a favourable tolerability and safety profile in comparison with imatinib and second-generation tyrosine kinase inhibitors (TKIs). The FDA’s accelerated approval is based on the major molecular response rate (MMR) at week 48 from the Phase III, head-to-head, multi-centre, open-label, randomised ASC4FIRST trial. The study compared once-daily 80mg Scemblix to all other investigator-selected standard-of-care TKIs in 405 adults with Ph+ CML-CP. Novartis US president Victor Bulto stated: “Despite many advances in the field, patients still need treatment options that are highly effective with a favourable tolerability profile to help enable them to achieve meaningful outcomes as they manage chronic conditions.” See Also: GSK to acquire Chimagen’s CMG1A46 for autoimmune disease Arrivo targets major depressive disorder in females after early efficacy data The approval was also backed by preliminary data from the Phase II ASC2ESCALATE study, which includes patients previously treated with one prior TKI. In the ASC4FIRST trial, Scemblix showed superior MMR rates at week 48 compared to the standard of care TKIs and imatinib alone. 20% more subjects who received the therapy achieved MMR compared to investigator-selected standard-of-care TKIs, and 30% achieved MMR compared to imatinib alone at week 48. Patients treated with Scemblix also achieved deeper rates of molecular responses, including MR4, against investigator-selected TKIs and imatinib alone by week 48. Full approval for this new indication may depend on the verification as well as a description of clinical benefit from confirmatory evidence. The expanded indication increases the eligible population for Scemblix by nearly four times, including newly diagnosed and previously treated adult patients. The ASC4FIRST trial is ongoing and the next scheduled analysis will be at week 96 for evaluating the key secondary endpoint along with additional secondary endpoints. In July 2024, the FDA granted priority review for the therapy in the given indication.

iStock, TBE Scemblix was granted accelerated approval by the FDA for the treatment of certain patients newly diagnosed with chronic myeloid leukemia. The expanded indication increases the eligible patient population by approximately four times, according to Novartis. The FDA on Tuesday approved the expansion of Novartis ’ Scemblix for the treatment of adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase. Granted under the regulator’s accelerated approval pathway, Tuesday’s label expansion pushes Scemblix into the first-line setting in chronic myeloid leukemia (CML), allowing its use in patient with chronic phase disease positive for the Philadelphia chromosome. To retain this approval, Novartis will need to verify the clinical benefit of Scemblix in this indication through a confirmatory study. “Despite many advances in the field, patients still need treatment options that are highly effective with a favorable tolerability pro help enable them to achieve meaningful outcomes,” Victor Bulto, U.S. president of Novartis, said in a statement. Scemblix fulfills both criteria and possesses the “potential to change the trajectory of many more people living with CML,” Bulto said. Novartis said the first-line approval broadens the patient population for Scemblix by approximately four times, which will help the pharma achieve its sales target for its oral tyrosine kinase blocker. During the company’s second-quarter earnings call in July, CEO Vas Narasimhan projected “$3 billion-plus peak sales” for Scemblix, adding that it is likely to be exempt from the Inflation Reduction Act’s drug price negotiations because it targets a rare disease. “A really great profile, great medicine, very excited about its future,” Narasimhan said of Scemblix during the investor call. In Q3, Scemblix was one of Novartis’ main growth drivers, surging 72% year-over-year at constant currencies, bringing in $182 million. Scemblix’s sales in the quarter beat the analyst consensus by 3%, according to Jefferies analyst Peter Welford. Scemblix is an orally available tyrosine kinase inhibitor that works by blocking the BCR-ABL1 fusion protein, a known hallmark of certain leukemias. The drug was first approved for CML three years ago , winning two indications. The first is for the treatment of Philadelphia chromosome-positive, chronic phase CML, for which the drug was given accelerated approval. The second is a full approval for the same patient population, with the additional specification that they carry the T315I mutation. Tuesday’s label expansion was backed by data from the Phase III ASC4FIRST study, which showed that 68% of patients on Scemblix achieved major molecular response, compared with 49% of those treated with standard-of-care tyrosine kinase inhibitors such as imatinib and nilotinib. The difference of nearly 20 percentage points was statistically significant with a p-value of less than 0.001, according to Novartis. Molecular responses were likewise deeper with Scemblix, while the drug’s safety pro consistent with what had been established in prior studies.