FDA Orphan Drug Designation Awarded to a Myelodysplastic Syndromes Investigative Therapy

Karyopharm Therapeutics has just announced that Eltanexor, their investigative therapy for myelodysplastic syndromes (MDS), has been given Orphan Drug Designation by the FDA. This designation means that the FDA believes…

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Recent Advances in Acute Myeloid Leukemia and Myelodysplastic Syndromes
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Recent Advances in Acute Myeloid Leukemia and Myelodysplastic Syndromes

An article in PubMed dated October 8, 2021 outlines immune therapies treating myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) patients. Both cancers are hematologic malignancies that begin in the…

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Phase 3 Data Available on Pevonedistat for AML, MDS, CMML
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Phase 3 Data Available on Pevonedistat for AML, MDS, CMML

In the Phase 3 PANTHER clinical trial, also known as the Pevonedistat-3001 study, researchers were evaluating pevonedistat in conjunction with azacitidine for patients with acute myeloid leukemia (AML), myelodysplastic syndromes…

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FDA Halted Phase 3 Trial for Cerebral Adrenoleukodystrophy Due to Participant Developing MDS
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FDA Halted Phase 3 Trial for Cerebral Adrenoleukodystrophy Due to Participant Developing MDS

      Science Magazine recently joined other news sources in reporting that Bluebird Bio’s Phase 3 clinical trial investigating a treatment for a neurological disease was put on hold by…

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Venclexta with Azacitidine Granted Breakthrough Therapy Designation for MDS
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Venclexta with Azacitidine Granted Breakthrough Therapy Designation for MDS

In a news release from July 21, 2021, biotechnology company Genentech shared that its therapy Venclexta (venetoclax), in conjunction with azacitidine, received Breakthrough Therapy designation from the FDA. Overall, the…

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First Patient Dosed in CA-4948 Trial for Hematologic Malignancies
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First Patient Dosed in CA-4948 Trial for Hematologic Malignancies

The first patient has been dosed in a Phase 1 clinical trial, which is exploring Curis Inc.'s CA-4948 in conjunction with ibrutinib for hematologic malignancies. Examples of hematologic malignancies include…

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Risk of Myelodysplastic Syndrome and Acute Myeloid Leukemia May Increase with PARP Inhibitors
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Risk of Myelodysplastic Syndrome and Acute Myeloid Leukemia May Increase with PARP Inhibitors

Poly (ADP-Ribose) polymerase inhibitors (PARP inhibitors) are used to treat neoplasms. They are frequently used as a therapy for ovarian cancer. A recent study has attempted to detect the possibility…

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Sabatolimab Can Be Added Safely to Hypomethylating Agents in the Treatment of MDS and AML

Dr. Andrew Brunner, an oncologist at Massachusetts Cancer Center was recently interviewed by Targeted Oncology. The discussion centered around a phase 1b clinical trial (NCT03066648) investigating sabatolimab together with hypomethylating…

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Experimental Treatment Could be Useful in Numerous Rare and Deadly Cancers
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Experimental Treatment Could be Useful in Numerous Rare and Deadly Cancers

While novel treatments have been rapidly emerging onto the drug scene, new treatment options for pancreatic cancer have fallen behind. But now, according to OncLive’s interview with Dr. P.A. Philip,…

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50% Enrollment Reached in Imetelstat Trial for MDS
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50% Enrollment Reached in Imetelstat Trial for MDS

Since its conception, biopharmaceutical company Geron Corporation ("Geron") has worked to develop first-in-class therapies for hematologic myeloid malignancies. More recently, Geron began evaluating imetelstat for patients with lower risk myelodysplastic syndromes…

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