On July 18, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported that the European Medicines Agency (EMA) has validated the company’s Marketing Authorization Application (MAA) for avapritinib for the treatment of adult patients with PDGFRα D842V mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST (Press release, Blueprint Medicines, JUL 18, 2019, View Source [SID1234537608]). Validation of the MAA confirms that the application is sufficiently complete to begin the formal review process. Avapritinib is an investigational, potent and highly selective KIT and PDGFRA inhibitor for patients with advanced GIST. The European Commission has granted orphan medicinal product designation to avapritinib for the treatment of GIST.

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"Prognoses are traditionally poor for patients with PDGFRα D842V mutant GIST and fourth-line GIST, and currently no effective therapy exists for either population," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "Avapritinib is specifically designed to inhibit the disease drivers of GIST, including mutant kinases associated with treatment resistance, representing a promising therapeutic approach. We look forward to working expeditiously with the EMA to bring this potential new treatment option to patients."

In June 2019, Blueprint Medicines announced the submission of a New Drug Application to the U.S. Food and Drug Administration (FDA) for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant GIST, regardless of prior therapy, and fourth-line GIST.

About GIST

GIST is a sarcoma, or tumor of bone or connective tissue, of the gastrointestinal (GI) tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging and, in rare cases, tumor rupture or GI obstruction.

Most GIST cases are caused by a spectrum of clinically relevant mutations that force the KIT or PDGFRA protein kinases into an increasingly active state. Because currently available therapies primarily bind to the inactive protein conformations, certain primary and secondary mutations typically lead to treatment resistance and disease progression.

In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type. Mutational testing is critical to tailor therapy to the underlying disease driver and is recommended in expert guidelines. Currently, there are no approved therapies for patients with KIT-driven GIST whose disease progresses beyond imatinib, sunitinib and regorafenib. In patients with metastatic PDGFRα D842V-driven GIST, progression occurs in a median of approximately three to four months with available therapy.

About Avapritinib

Avapritinib is an investigational, oral precision therapy that selectively and potently inhibits KIT and PDGFRA mutant kinases. It is a type 1 inhibitor designed to target the active kinase conformation; all oncogenic kinases signal via this conformation. Avapritinib has demonstrated broad inhibition of KIT and PDGFRA mutations associated with GIST, including potent activity against activation loop mutations that are associated with resistance to currently approved therapies.

Blueprint Medicines is initially developing avapritinib for the treatment of advanced GIST, advanced systemic mastocytosis (SM), and indolent and smoldering SM. The FDA has granted Breakthrough Therapy Designation to avapritinib for two indications: one for the treatment of unresectable or metastatic GIST harboring the PDGFRα D842V mutation and one for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia. The European Commission has granted orphan medicinal product designation to avapritinib for the treatment of GIST and mastocytosis.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.

On July 18, 2019 CTI BioPharma Corp. (Nasdaq: CTIC) ("CTI" or "the Company") reported the outcome of a Type B, End-of-Phase-2a meeting with the U.S. Food and Drug Administration ("FDA" or "the Agency") for the continued development of its investigational myelofibrosis treatment candidate, pacritinib (Press release, CTI BioPharma, JUL 18, 2019, View Source;p=RssLanding&cat=news&id=2404063 [SID1234537593]). Following this meeting, CTI plans to evaluate 200 mg of pacritinib administered twice daily (BID) in 180 patients with myelofibrosis and severe thrombocytopenia. The Company plans to initiate the Phase 3 PACIFICA study in the third quarter of 2019.

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"We are pleased to be able to move the pacritinib program forward and are now in the process of finalizing an amendment to the PAC203 protocol, which the FDA will review, to allow a transition to the new PACIFICA Phase 3 study, in which we plan to compare the 200 mg BID dose of pacritinib to Physician’s Choice in myelofibrosis patients with severe thrombocytopenia, an important unmet medical need," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. "We anticipate initiating the trial in the third quarter, which would put us on track for topline Phase 3 data in mid-2021."

The randomized, open-label Phase 2 PAC203 dose-finding study was designed to evaluate the safety and efficacy of three dosing regimens of oral pacritinib in 150 patients with myelofibrosis. The Company expects topline safety and efficacy results from the Phase 2 portion of the PAC203 study in the third quarter of 2019 and is targeting presentation of the Phase 2 results at a scientific conference before the end of 2019.

The previous Phase 3 PERSIST program consisted of the PERSIST-1 trial, which was conducted in a broad set of patients without limitations on platelet counts, and the PERSIST-2 trial, which was conducted in patients with low platelet counts. An ad-hoc analysis of pooled data from PERSIST-1 and PERSIST-2 evaluated results from patients with platelet counts of less than 50,000 per microliter and showed that 23% (n=104) of patients administered pacritinib had a ≥35% spleen volume reduction (SVR), compared to 2% (n=48) (p=0.0007) given the best available therapy, which in the PERSIST-1 trial excluded JAK2 inhibitors and in the PERSIST-2 trial included the approved JAK2 inhibitor, ruxolitinib. The most common treatment-emergent adverse events of any grade occurring in 20% or more of patients treated with pacritinib within 24 weeks during the PERSIST-1 and PERSIST-2 trials were gastrointestinal (generally manageable diarrhea, nausea and vomiting) and hematologic (anemia and thrombocytopenia).

The Company is currently amending the protocol for the ongoing Phase 2 PAC203 study to include the new PACIFICA Phase 3 portion, in which CTI intends to compare the safety and efficacy of 200 mg of pacritinib administered twice daily to Physician’s Choice in 180 myelofibrosis patients with severe thrombocytopenia (platelet counts of less than 50,000 per microliter) at approximately 120 sites worldwide. Patients will be randomized in a ratio of 2:1 between pacritinib and Physician’s Choice. The primary endpoint of the trial is the percentage of patients who achieve at least 35% reduction in spleen volume at week 24. Dr. Srdam Verstovsek, Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, and Dr. John Mascarenhas, Associate Professor of Medicine Myeloproliferative Disorders Program, Tisch Cancer Institute, Mount Sinai School of Medicine, will be co-principal investigators in the PACIFICA study. In addition, Professor Claire Harrison, Professor of Medicine, Guy’s and St Thomas’ NHS Foundation Trust, London, will chair the study’s Steering Committee.

About Myelofibrosis and Severe Thrombocytopenia
Myelofibrosis is a type of bone marrow cancer that results in formation of fibrous scar tissue and can lead to severe anemia, weakness, fatigue and an enlarged spleen and liver. Patients with severe thrombocytopenia are estimated to make up more than one-third of patients treated for myelofibrosis, or approximately 18,000 people.1 Severe thrombocytopenia, defined as blood platelet counts of less than 50,000 per microliter, has been shown to result in overall survival rates of just 15 months.2 Thrombocytopenia in patients with myelofibrosis is associated with the underlying disease but has also been shown to correlate with treatment with ruxolitinib, currently the only approved treatment for myelofibrosis, which can lead to dose reductions and as a result may potentially reduce clinical benefit. Survival in patients who have discontinued ruxolitinib therapy is further compromised, with an average overall survival of seven to 14 months.3,4 There are currently no approved therapies available to treat myelofibrosis patients with severe thrombocytopenia, or patients who have failed ruxolitinib treatment, thereby making this a significant unmet medical need.

About Pacritinib
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.

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On July 18, 2019 Diplomat Pharmacy, Inc. (NYSE: DPLO), reported that it will release its second-quarter 2019 operating results before market open Friday, August 9. A conference call and live webcast will be held at 8:30 a.m. ET (Press release, Diplomat Speciality Pharmacy, JUL 18, 2019, View Source [SID1234537594]).

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Shareholders and interested participants can listen to a live broadcast by calling 833.286.5805 (647.689.4450 for international callers) and entering participation code 7394702, starting about 15 minutes before the call. A live webcast of the conference call will be available on the investor relations section of Diplomat’s website at ir.diplomat.is. The site will host an audio recording and supplemental investor information for 90 days.

On July 18, 2019 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported that has executed a contract under the previously announced Memorandum of Understanding, with Shanghai Fosun Long March Medical Science Co., Ltd. ("Fosun Long March") to help introduce the Nu.Q(TM) platform to China (Press release, VolitionRX, JUL 18, 2019, View Source [SID1234537610]). Based on recent promising proof-of-concept data using Volition’s product-grade Nu.Q assays, the parties have commenced a clinical study in China in lung cancer.

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Dr. Jasmine Kway, Chief Executive Officer of Singapore Volition, commented: "We are happy to have executed this agreement with Fosun Long March and are delighted that the clinical study is already underway and expected to be completed by the end of this year."

Dr. Yuejian Zhang, Chairman of Fosun Long March, remarked: "We were impressed with Volition’s preliminary data, particularly in lung cancer, and are happy to have started work on these initial clinical studies. Whilst China represents a little under 20% of the world’s population, it accounted for almost 24% of cancer diagnoses and 30% of cancer-related deaths worldwide in 2018, almost a third of which were due to lung cancer and colorectal cancer. We look forward to completing this important study and working together with Volition to launch Nu.Q in China."

"The need for a simple, easy to use, cost effective test for cancer is truly universal and we hope very much that our Nu.Q tests can help revolutionize the way cancer is diagnosed worldwide," commented Cameron Reynolds, Chief Executive Officer of Volition. "We see Asia, and particularly lung cancer in China, as a key market for Volition. We are excited about the commencement of our work in China and look forward to a successful collaboration with Fosun Long March."

About Fosun Long March

Established in 1989, Fosun Long March is the investment platform of in-vitro diagnostics division of Fosun Pharma Group and has since accelerated merger and acquisition of new products introduction into China. With its wide range of in-vitro diagnostics products, strength in manufacturing and strong nation-wide sales network, Fosun Long March is one of the most influential brands in in-vitro diagnostics in China.

Fosun Long March’s network and expertise will assist Volition with the facilitation and coordination of the collection, provision and testing of blood samples using Volition’s proprietary Nu.Q(TM) assays for the clinical studies, with Fosun’s hospitals, research institutions and clinics, as well as other third-party hospitals, universities, research institutions and clinics in China.