Fimepinostat

Curis’ decision to advance emavusertib over others is based on data that was just received from the phase 1/2 TakeAim Leukemia study, which is due to be presented at the upcoming American Society of Hematology meeting in December. Curis had some tough decisions to make to ensure its leukemia drug could survive, but the data were clear: The company needed to rally behind the med, and, in today’s biotech landscape, that means layoffs and a slimming of other programs. The Lexington, Massachusetts-based company is cutting 30% of its workforce and reallocating resources to focus solely on emavusertib, which is currently in a phase 1/2 trial for acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS), and another phase 2 study called LUCAS for patients with lower-risk MDS. The cuts were announced in a third-quarter earnings report Wednesday afternoon.  The decision to advance emavusertib over the others is based on data that was just received from the phase 1/2 TakeAim Leukemia study, which is due to be presented at the upcoming American Society of Hematology meeting in December. Curis will present data on 11 additional evaluable patients treated with emavusertib alone and in combination with AbbVie and Genentech’s Venclexta. It’s the same trial that was stymied by an FDA hold earlier this year after a patient death spurred a safety review. The agency followed up the April hold with a second partial hold on the similar TakeAim Lymphoma trial. Both were lifted in August after the FDA conducted a safety review and collected additional information from Curis on the patient, who experienced rhabdomyolysis—a known dose-limiting toxicity of emavusertib that leads to the breakdown of skeletal muscle. The review also produced a rhabdomyolysis identification and management strategy for future patients. CEO Jim Dentzer acknowledged the tough decision to lay off workers but highlighted the unmet need in relapsed/refractory AML or MDS and said emavusertib could be a “game-changing therapy.” “I'd like to acknowledge that while we are excited about the heightened focus of emavusertib, we understand the impact of deprioritizing our other programs has on our valued colleagues and friends who worked on them,” Dentzer said during a third-quarter earnings call Wednesday afternoon. The cuts will allow Curis to extend its cash runway into 2025, Dentzer said. As of the end of September, Curis had $98.7 million in cash, cash equivalents and investments. Dentzer said the reduction will be across the company, in both R&D and administrative roles, and will focus on “everything that’s not directly related to emavusertib.” “Anything that wasn’t directly related was something that we could live without,” he said. “And if we could live without it and that extended our cash runway without having to do anything else—I mean this is the luxury of starting with such a strong balance sheet that we have this flexibility.” He again stressed that the data to come would make clear that all efforts need to shift behind emavusertib. “With these data in it becomes increasingly clear that this is a drop and this needs to get to NDA submission with haste,” Dentzer said of the efficacy results seen in the emavusertib data to come. “So we’re going to dedicate and redouble all of our efforts to make that happen and insulate ourselves from the need to raise money in the meantime.” The cuts will begin in the fourth quarter. Curis has four other therapies listed on its pipeline, including the early-stage diffuse large B-cell lymphoma treatment fimepinostat and CI-8993 in VISTA-expressing cancers. Dentzer said that the CI-8993 program will be “more of a pause” with no new patients being enrolled. The pending data on emavusertib were just too strong and puts the company on the precipice of a new drug filing. “If you can’t afford to do both, boy, the decision is clear, you go with the one that’s got the proven data,” Dentzer said. The CEO also gave some additional color on the nature of the FDA hold. While the patient death was a major part of the FDA’s review, he also said the agency had questions about dosing under its Project Optimus, which aims to find the lowest effective dose for newer cancer treatments. Curis tested three different doses ranging from 200 mg to 400 mg, which all have shown responses. The FDA wanted to be involved in the process to determine which dose moved forward in further testing: “That's the perfect place for FDA to want to sink their teeth in,” Dentzer said. In the end, Curis enrolled nine new patients at the lowest dose to help determine the correct amount to move forward with in phase 2. Dentzer said the hope is to have those new patients enrolled by the midpoint of next year, which will allow conversations about a new drug application to fire up. Curis' shares fell just over 7% as the market closed Wednesday to 72 cents compared with a previous open of 77 cents.

JStone/Shutterstock Disgraced pharma executive Martin Shkreli is pleading to be temporarily let out of prison in order to join in the fight against COVID-19, the disease that has swept across the globe infecting more than 1.3 million people across the globe. JStone / Shutterstock Disgraced pharma executive Martin Shkreli is pleading to be temporarily let out of prison in order to join in the fight against COVID-19, the disease that has swept across the globe infecting more than 1.3 million people across the globe. Shkreli is seeking a three-month furlough from federal prison to help develop a potential treatment for the disease. In a scientific paper published this week co-authored by Shkreli, he argued that the pharmaceutical industry has had an inadequate response to the threat of COVID-19. First reported by STAT News , Shkreli said the industry has “a large braintrust of talent” that is not working on the COVID-19 problem due to a deprioritization of infectious disease research across the pharmaceutical landscape. All biopharmaceutical companies should be responding with all resources to combat this health emergency, he stressed. “Medicinal chemists, structural biologists, enzymologists and assay development and research biology departments at EVERY pharmaceutical company should be put to work until COVID-19 is no more,” Shkreli wrote in the author’s note of the research paper “ In silico screening for potential COVID-19 beta-coronavirus non-nucleoside RdRp inhibitors .” The paper is credited to Prospero Pharma, a company established by Shkreli in 2015. Countless companies from across the pharmaceutical industry are taking aim at developing treatments and vaccines for COVID-19. Many companies are taking multiple shots on goal through their own internal R&D programs, as well as forging partnerships with other companies to tackle the issue from another angle. Not only are new drugs being developed, older pipeline drugs are being repurposed for the disease as well. BioSpace puts together a daily roundup of the industry’s response to COVID-19. Shkreli added that he was requesting three months and noted that someone with his credentials should not be excluded from helping develop a treatment for the disease. He said he is a “successful two-time biopharma entrepreneur” who has not only purchased multiple companies but has also “invented multiple new drug candidates,” and also filed Investigational New Drug Applications and applications for clinical trials. “I am one of the few executives experienced in ALL aspects of drug development from molecule creation and hypothesis generation, to preclinical assessments and clinical trial design/target engagement demonstration, and manufacturing/synthesis and global logistics and deployment of medicines,” he wrote. In a complete turnaround to his arguments in support of the 5,000% price hike for the toxoplasmosis drug Daraprim and his follow-up laments that he didn’t increase the price even higher, Shkreli said any treatment developed for COVID-19 should be made available to the public for next to nothing. He said a company that develops a treatment should be able to recoup its research and development costs “at most” and should be “willing to perform the work as a civil service at the least.” “If the government is willing to reward industry for their work on this catastrophic situation, it will be at each company’s discretion to accept, negotiate or deny such funding, including bulk purchases, cost reimbursement, tax credits and other benefits,” he added. The paper Shkreli co-authored claims to have identified multiple drugs already in existence that could be used against COVID-19, including clofazimine, an anti-leprotic and antibiotic medication, as well as hepatitis C nucleoside analogs paritaprevir and ledipasvir. Other candidates include bafetinib, apilimod, and fimepinostat. The paper was co-authored with Kevin Mulleady, a longtime associate of Shkreli and current chairman of the board of Phoenixus AG, the parent company of Vyera Pharmaceuticals, LLC, the company formerly known as Turing Pharmaceuticals that Shkreli founded. Shkreli was found guilty in 2017 on charges of securities fraud and one charge of conspiracy to commit conspiracy fraud. He is eligible for release in 2023. hbspt.forms.create({ portalId: "4413123", formId: "a9adb9af-0e12-4b29-a8c0-3da77fe7c020" });