Startup Impact Biomedicines Raises $22M to Bring Fedratinib to MF Patients

SAN DIEGO–(Business Wire)–Today, Impact Biomedicines (“Impact”) announced its launch to pioneer the development of life-changing treatments for patients with myeloproliferative neoplasms and other cancers. The Company’s pipeline is centered around fedratinib, a potent and highly selective oral small molecule JAK2 kinase inhibitor that is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV). In conjunction with this launch, Impact is pleased to share that the U.S. Food and Drug Administration (FDA) has removed the clinical hold placed on fedratinib and that the company has received $22.5 million from Medicxi through a Series A financing.

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Secondary Myelofibrosis Recruiting Phase 1 Trials for DB00493 (Cefotaxime)

This pilot clinical trial studies the side effects of combination chemotherapy, total body irradiation, and donor blood stem cell transplant in treating patients with secondary myelofibrosis. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving combination chemotherapy and total body irradiation before a donor blood stem cell transplant helps to stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient’s bone marrow make stem cells, red blood cells, white blood cells, and platelets.

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MPN Advocacy and Education International Attends a Workshop Hosted by the FDA

By Ann Brazeau, CEO MPN Advocacy & Education International

MPN Advocacy & Education International was invited to attend a very important and timely meeting with the FDA last month. The invitation was extended to us and a few other MPN focused organizations. This meeting launched a unique beginning for those of us representing the MPN Community.

The FDA’s newly formed Oncology Center of Excellence and the Office of Patient Outcomes are committed to engaging MPN patients and advocates to better respond to the need for quality treatment options.

Among the many topics presented by their hematologists, researchers and cancer patient liaison, was an in depth explanation on drug approval processes and how valuable and critical the patient’s voice is to their mission.

After a drug is approved, companies are required to send frequent updates on any and all reported adverse side effects from their drug. Physicians also report on these events and patients can do so by contacting the FDA directly through Med Watch Consumer Voluntary Reporting. Go to www.fda.gov/reportinghelp. Patient Reported Outcomes are extremely important and valuable to the Drug Approval Process and after a drug is approved.

We also learned the complexities surrounding drug “holds” and what happens and why when that hold begins and when it is released. They stressed the importance of ongoing communication between BioPharma and the FDA and encourage regular conversations and frequent updates with comprehensive data.

As the MPN Community pursues the use of Interferon and hopes for accessibility to Interferon as a viable option for treatment, we learned that company(ies) producing Interferon would have to apply for approval specifically for MPNs. It could be a daunting process but there may be avenues we can utilize through patient advocacy efforts.

Although we did not share all of our concerns at this meeting, we look forward to subsequent meetings where we hope to tackle the issues of endpoints, pricing, fast-tracking, and other challenges surrounding MPN drug approval. We were very pleased to have had this opportunity.

 

 

 

 

Stemline Therapeutics to Present SL-801 Phase 1 Data at Upcoming ESMO Congress

NEW YORK, Aug. 30, 2017 (GLOBE NEWSWIRE) — Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, announced today that data from SL-801’s ongoing Phase 1 trial in patients with advanced solid tumors has been selected by the European Society of Medical Oncology (ESMO) for poster presentation at their Annual Congress 2017, to be held September 8-12, 2017 in Madrid, Spain. SL-801 is a novel, potent and reversible Exportin-1 (XPO1) inhibitor.

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Galena Announces Presentation of Encouraging GALE-401 Safety Data

GALE-401 clinical trials were presented at the European Hematology Association 21st Congress.

Galena Biopharma, Inc. (Nasdaq: GALE) announced that combined safety data from the Company’s GALE-401 clinical trials were presented at the European Hematology Association 21st Congress. GALE-401 is Galena’s controlled release (CR) version of anagrelide for the treatment of patients with thrombocythemia, secondary to myeloproliferative disorders, to reduce elevated platelet counts in patients suffering from myeloproliferative neoplasms (MPNs).

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Stemline Therapeutics Clears First Cohort of Patients in Ongoing SL-801 Phase 1 Trial

NEW YORK, May 03, 2016 (GLOBE NEWSWIRE) — Stemline Therapeutics, Inc. (Nasdaq:STML) announced today that it has completed the initial dosing cohort in its SL-801 Phase 1 advanced solid tumor trial. The second cohort is currently open. SL-801 is a novel, oral, small molecule that reversibly inhibits the XPO1 (Exportin-1) nuclear transport protein, a clinically validated target active in a wide variety of cancer types.

This Phase 1 dose escalation trial will enroll up to 50 patients with advanced solid tumors at multiple centers in the U.S. The trial is designed to evaluate safety, identify an optimal dosing regimen, and detect potential signals of efficacy in one or more cancer types.

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CTI BioPharma Updates on Pacritinib Full Clinical Hold; Dose Exploration Studies Suggested by FDA

On March 14, 2016, CTI BioPharma Corp. (the “Company”) provided an update regarding the availability of pacritinib to certain patients with myelofibrosis. Following the issuance of the Company’s February 9, 2016, press release describing the full clinical hold issued by the U.S. Food and Drug Administration (“FDA”) regarding pacritinib Phase 3 clinical studies, the FDA has been in communication with the Company regarding pacritinib. The FDA recently expressed an interest in allowing patients who were receiving benefit from pacritinib treatment at the time the clinical hold was imposed to submit requests to the FDA to resume pacritinib treatment under a Single Patient IND (“SPI”) program on a case by case basis. The Company is working with the FDA on certain regulatory and logistical items necessary to assist investigators in submitting SPIs for their patients to the FDA for consideration. The Company has informed clinical investigators worldwide of the FDA’s interest in allowing SPIs, and plans to continue to provide updates as meaningful details become available.

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Optimal Therapy for PV and ET

Preferred use of interferon therapy based on phase 2 trials

To determine the value of recombinant interferon-alfa (rIFNα) in the treatment of polycythemia vera (PV) and essential thrombocythemia (ET) based on its biological activities and phase 2 clinical studies, pending completion of phase 3 trials; to determine importance of the Internet in patient decision-making in treatment selection.

Results

The value of rIFNα in PV and ET is based upon its biological effects on PV stem cells and megakaryocyte proliferation. Single-arm trials are useful for life-threatening diseases when there are relatively few patients to evaluate endpoints, such as rIFNα treatment of PV and ET. Proper diagnostic criteria are mandatory; for PV, the current World Health Organization criteria emphasizing increased hemoglobin values exclude approximately one-third of eligible patients.

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