On October 25, 2019 Imago BioSciences, Inc., a clinical-stage biotechnology company focused on the treatment of myeloproliferative neoplasms (MPN) and related bone marrow diseases, reported that preliminary data from its ongoing Phase 2 study of IMG-7289 (bomedemstat) in patients with myelofibrosis (MF) has been selected for an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, on December 9 in Orlando, Florida (Press release, Imago BioSciences, OCT 25, 2019, View Source [SID1234542532]). The abstract will be published November 6, and the presentation will include results updated from those in the abstract.

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Kristen Pettit, M.D., assistant professor at the University of Michigan and investigator in the study at the Rogel Cancer Center in Ann Arbor, will present both preliminary results from Phase 2a, as well as initial data from patients from the Phase 2b expansion. The objectives of the study are to evaluate the safety and efficacy of IMG-7289 (bomedemstat) in up to 75 patients at sites in Australia, the US, UK and Europe. In this study, bomedemstat is administered orally once-daily as monotherapy in adult patients with intermediate-2 or high-risk MF resistant to or intolerant of ruxolitinib.

"The FDA recently approved a second JAK2 inhibitor but the majority of patients with myelofibrosis will eventually lose the benefit of those treatments," said Dr. Pettit. "Patients have an urgent need for new treatments that manage their symptoms. We continue to be encouraged by the bomedemstat data we see in this clinical investigation."

Imago Presentation

Title: A Phase 2 Study of the LSD1 Inhibitor IMG-7289 (bomedemstat) for the Treatment of Myelofibrosis. Session: 634. Myeloproliferative Syndromes: Clinical: Emerging and Novel Targeted Therapies
Session Date: Monday, December 9, 2019
Session Time: 7:00 AM – 8:30 AM EST
Presentation Time: 7:45 AM EST
Room: Orange County Convention Center, W304EFGH

About IMG-7289

IMG-7289 (bomedemstat) is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A). LSD1 is an enzyme regulating both cytokine expression and myeloid differentiation and sustaining self-renewal in malignant hematopoietic stem/progenitor cells. In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent and in combination with other therapeutic agents across a range of myeloid malignancy models, including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat for the treatment of myelofibrosis. An international Phase 2b study of bomedemstat for the treatment of myelofibrosis remains ongoing (Clinicaltrials.gov NCT03136185). Additional clinical studies in hematologic disorders will begin in 2020.

On October 25, 2019 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, reported that it will report its financial results for the third quarter ended September 30, 2019 before market open on Thursday, November 7, 2019 (Press release, X4 Pharmaceuticals, OCT 25, 2019, View Source [SID1234542533]).

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The Company will host a conference call and webcast on the same day at 8:00 a.m. ET to discuss these financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 4081686. The live webcast can be accessed on the investor relations section of X4’s website at View Source Following the completion of the call, a webcast replay of the conference call will be available on the website.

On October 25, 2019 Luminex Corporation (NASDAQ: LMNX) reported that it plans to report results for the third quarter ended September 30, 2019 on Monday, November 4, 2019 after the market close (Press release, Luminex, OCT 25, 2019, View Source [SID1234542534]).

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The company will host a conference call that day at 5:00 p.m. Eastern Time to discuss operating highlights and financial results.

The conference call will be webcast live and may be accessed at Luminex’s Investor Relations website at View Source The presentation slides will be posted to our Investor Relations website after the market close on November 4, 2019. Analysts and institutional investors may participate on the conference call by dialing (877) 930-7053 (U.S.) or (253) 336-7290 (outside the U.S.). The access code is 1293259. The webcast will be archived for six months on our website using the ‘replay’ link.

On October 25, 2019 LIPAC Oncology LLC reported the successful completion of a Phase 1 clinical study and progress in a Phase 2A study to further investigate TBC-1002 for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC) (Press release, Lipac Oncology, OCT 25, 2019, View Source [SID1234542535]). Phase 1 study results showed escalating doses of TBC-1002 to be well tolerated with no dose limiting toxicity and no irritative voiding (cystitis) symptoms. No systemic exposure or toxicity was observed, even with high concentrations of TBC-1002 being delivered topically to the bladder wall. Phase 1 results also indicated preliminary efficacy with no evidence of bladder cancer recurrences observed up to 18 months after dosing. Adverse events were minor.

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TBC-1002 is a proliposomal intravesical paclitaxel formulation (PLIP), in development for intravesical administration in the treatment of non-muscle invasive bladder cancer (NMIBC). NMIBC is a common and highly recurrent disease that can often be difficult to treat. If approved, TBC-1002 would be the first chemotherapeutic agent approved by the U.S. Food and Drug Administration for this indication in more than two decades.

"We are excited about the potential of TBC-1002 to target bladder cancer directly," said Michael Oefelein, Chief Medical Officer of LIPAC. "Having treated bladder cancer for more than 23 years, it is very clear that new treatment options and more effective delivery systems could help improve both clinical outcomes and quality of life."

In August, LIPAC commenced a Phase 2A marker lesion clinical study to establish efficacy responder rates within 16 weeks, which predict long-term (two-year) recurrence free survival rates. Efficacy data from this open label study is expected as soon as November 2019 with the study results available in the first quarter of 2020.

"Having established the technology to deliver chemotherapeutic agents safely in human trials, we look forward to applying this technology in other investigational areas such as upper tract urothelial carcinoma (UTUC), ovarian cancer and peritoneal cancer," said TR Thirucote, Chairman and CEO of LIPAC.

Further results on the clinical development program for TBC-1002 will be presented at the China Biomedical Industry Cooperation Forum in Shenzhen in October and at the Society of Urologic Oncology in Washington, D.C. in December.

On October 25, 2019 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported that the European Commission (EC) has approved the oral once-daily therapy XOSPATA (gilteritinib) as a monotherapy for the treatment of adult patients with relapsed or refractory (resistant to treatment) acute myeloid leukemia (AML) with a FLT3 mutation (FLT3mut+) (Press release, Astellas, OCT 25, 2019, View Source [SID1234542517]). Gilteritinib has the potential to improve treatment outcomes for AML patients with two forms of the most common mutation—FLT3 internal tandem duplication (ITD) and FLT3 tyrosine kinase domain (TKD) mutation.1,2

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This approval is based on results from the Phase 3 ADMIRAL trial, which investigated gilteritinib versus salvage chemotherapy in patients with relapsed or refractory FLT3mut+ AML. Patients treated with gilteritinib had significantly longer overall survival (OS) than those who received salvage chemotherapy. Median OS for patients who received gilteritinib was 9.3 months, compared to 5.6 months for patients who received salvage chemotherapy (Hazard Ratio = 0.64 (95% CI 0.49, 0.83), P=0.0004). Rates of one-year survival were 37% for patients who received gilteritinib, compared to 17% for patients who received salvage chemotherapy.3,4

"AML is a rare cancer and patients with a FLT3 mutation have a particularly poor prognosis, with a median survival of less than six months following treatment with salvage chemotherapy," said Giovanni Martinelli, M.D., Institute of Hematology, S.Orsola-Malpighi University Hospital, Bologna, Italy, an investigator in the ADMIRAL trial. "Gilteritinib is a new and clinically meaningful treatment option that provides a welcome advance for patients and health care professionals across the European Union."

The EC marketing authorization for gilteritinib in relapsed or refractory FLT3mut+ AML is applicable to the European Union (EU) member countries, and is also valid in Iceland, Norway and Liechtenstein. Gilteritinib has been designated an orphan medicinal product and also received accelerated assessment from the European Medicines Agency earlier this year, which reduced the timeframe for approval.5,6,7

"Today’s approval marks a significant advance for patients living with relapsed or refractory, FLT3 mutation-positive acute myeloid leukemia," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development, Astellas. "We look forward to working with health authorities across the EU to bring gilteritinib to patients who need it the most, as soon as possible."

Patients’ FLT3mut+ status can change over the course of AML treatment, even after relapse. Due to the poor outcomes associated with FLT3mut+ AML, patients’ FLT3 mutation status may be confirmed to help inform the best treatment approach.8,9,10

Astellas reflected the impact from this approval in its financial forecast of the current fiscal year ending March 31, 2020.

About XOSPATA (gilteritinib)
Gilteritinib was discovered through a research collaboration with Kotobuki Pharmaceutical Co., Ltd., and Astellas has exclusive global rights to develop, manufacture and commercialize gilteritinib. Gilteritinib was approved in the U.S. and Japan in 2018 for the treatment of adult patients who have relapsed or refractory FLT3mut+ AML.11,12

Astellas is currently investigating gilteritinib in various FLT3 mutation-positive AML patient populations through several clinical trials. Visit View Source to learn more about ongoing gilteritinib clinical trials.

About the ADMIRAL Trial
The Phase 3 ADMIRAL trial (NCT02421939) was an open-label, multicenter, randomized study of gilteritinib versus salvage chemotherapy in adult patients with FLT3mut+ who are refractory to or have relapsed after first-line AML therapy. The co-primary endpoints of the trial were OS and CR/CRh rates; OS, the primary endpoint at the trial’s final analysis, was the basis of EC approval. The study enrolled 371 patients with relapsed or refractory AML and FLT3mut+ present in bone marrow or whole blood. Subjects were randomized in a 2:1 ratio to receive gilteritinib (120 mg) or salvage chemotherapy.13

The most common adverse events (AEs) across both treatment arms of the ADMIRAL trial were febrile neutropenia (43.7%), anemia (43.4%), and pyrexia (38.6%). Common grade ≥3 AEs related to gilteritinib were anemia (19.5%), febrile neutropenia (15.4%), thrombocytopenia (12.2%), and decreased platelet count (12.2%). Adjusted for exposure duration, serious treatment-emergent AEs per patient year were less common with gilteritinib (7.1%) than salvage chemotherapy (9.2%).3