On January 3, 2018 Syntimmune, Inc., a clinical-stage biotechnology company focused on FcRn biology, reported that the company has appointed Jean-Paul Kress, M.D., as President and Chief Executive Officer (Press release, Syntimmune, JAN 3, 2018, View Source [SID1234522867]). Dr. Kress, who will also serve as a director of the company, brings senior leadership experience in pharmaceutical and biotechnology firms, with a focus on operations and commercialization of innovative products addressing unmet medical needs across diverse disease indications. David de Graaf, Ph.D., has stepped down from his position as CEO and resigned from the company’s Board of Directors. Syntimmune co-founder Laurence Blumberg, M.D., will continue to serve as Syntimmune’s Chief Operating Officer and as a member of the Board.

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"Jean-Paul possesses decades of experience bringing to market therapies based on breakthrough science," stated Burt Adelman, M.D., Syntimmune’s lead independent director and advisor to the company. "We are very pleased to welcome this industry veteran at the helm of Syntimmune. We believe Jean-Paul’s leadership brings vision and expertise that will be transformational as the company matures toward becoming a late-stage development organization and continues to rapidly advance therapies targeting FcRn biology to patients."

Dr. Blumberg added, "We are grateful for David de Graaf’s many contributions during a period of substantial progress at Syntimmune, during which we initiated two mid-stage clinical trials of our lead candidate, SYNT001, and completed a successful Series B financing that has positioned us well for further advancement."

Prior to joining Syntimmune, Dr. Kress served as Executive Vice President and President, International, and Head of Global Therapeutic Operations at Biogen Inc., overseeing the company’s rare and specialty disease teams. Previously, Dr. Kress was Senior Vice President, Head of North America at Sanofi Genzyme, where he was instrumental in launching several therapeutic products, including dupilumab, the first biologic agent approved in atopic dermatitis. Prior to this, he was President and Chief Executive Officer of Sanofi Pasteur MSD, a vaccines joint venture of Sanofi and Merck & Co. Dr. Kress also held leadership roles at Gilead, serving as Vice President, US Sales and Marketing, and Vice President, General Manager for France. He began his industry career with Eli Lilly in France and held commercial and business development roles in the US and Europe at Abbott (now AbbVie). Dr. Kress received an M.D. degree from Faculté Necker-Enfants Malades in Paris, and graduate and post-graduate degrees in pharmacology and immunology from École Normale Supérieure in Paris.

"I am excited to join the accomplished team at Syntimmune, which possesses world-class expertise in immunology and FcRn biology applicable to a broad range of autoimmune diseases," said Dr. Kress. "Syntimmune has made important strides in advancing its clinical development pipeline, and I look forward to the progress of the company’s ongoing Phase 1b/2a clinical trials of SYNT001 in pemphigus and warm autoimmune hemolytic anemia, as well as the anticipated commencement of clinical studies in additional indications."

On January 3, 2018 Foundation Medicine reported that Troy Cox, chief executive officer, is scheduled to present at the 36th Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2018 at 3:30 p.m. PST in San Francisco (Press release, Foundation Medicine, JAN 3, 2018, View Source [SID1234522851]). Additionally, Troy Cox will participate in a panel discussion focused on the Food & Drug Administration and Centers for Medicare and Medicaid Services Parallel Review process on Monday, January 8, 2018 at 5:15 p.m. PST.

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Live, listen-only webcasts of these presentations can be accessed by visiting the investors section of the Foundation Medicine website at investors.foundationmedicine.com. A replay of the webcasts will be available shortly after the conclusion of the presentation and archived on the company’s website for two weeks following the presentations.

On January 3, 2018 Rasna Therapeutics, Inc. (OTCQX: RASP), a clinical stage biotechnology company focused on the development of disease-modifying drugs for hematological malignancies, reported follow up Phase II clinical data showing that 4 out of 9 (44%) evaluable AML patients carrying the NPM1 gene mutation treated with actinomycin D ("Act D"), achieved complete remission (CR) (Press release, Rasna Therapeutics, JAN 3, 2018, View Source [SID1234522873]). Treatment with Act D was well tolerated, except that patients experienced oral mucositis as the major toxicity. Rasna Therapeutics has developed a proprietary nanoparticle based formulation of Act D (RASP-101), which is anticipated to maximize efficacy while minimizing oral mucositis. RASP-101 could potentially be a first-in-class modality for treatment of NPM1-mutated acute myeloid leukemia (AML).

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NPM1-mutated AML is a specific genetic leukemia entity that accounts for approximately one-third cases of AML in adults. As we reported previously, intravenous treatment of refractory or relapsed NPM1-mutated AML patients with Act D (12.5 µg or 15 µg/day) for 5 consecutive days produced hematological complete response in some of them (Falini et al., N Eng J Med. 373: 12, 2015).

In a follow-up phase II clinical study (ActD-AML-PG01, EudraCT 2014-000693-18) in refractory/relapse (R/R) AML patients carrying NPM1 gene mutation, treatment with Act D at 15 µg/kg/day for 5 days every 28 days induced CR in 4 out of the 9 evaluable patients (44.4%) with only 1 or 2 cycles of therapy. Three out of the 4 (75%) patients who obtained CR relapsed after 3, 5 and 7 months, respectively. One patient underwent haploidentical allogeneic PBSC transplantation at 3 months after CR achievement and is alive in molecular CR (MRD-negative) after 24 months.

"The precise mechanism of action of Act D in NPM1-mutated AML is still not clearly understood. Follow up data confirms our earlier findings and further support the use of Act D to induce complete hematological remissions with possible long-term molecular responses in NPM1-mutated AML patients. To note, our first previously reported NPM1-mutated AML patient (resistant to hypomethylating therapy) treated with Act D remains in molecular remission at over 3 years since CR achievement," said Dr. Brunangelo Falini, a member of the scientific advisory board of Rasna Therapeutics, Inc.

"We have developed a proprietary formulated Act D (RASP-101), which we anticipate will produce a superior clinical outcome with improved efficacy and safety profile. Emerging data from the multicenter clinical studies will allow us to develop a biomarker strategy to select responsive patients and improve clinical outcome for this unmet clinical need" commented Alessandro Padova, Chairman of Rasna.

About Actinomycin D (Dactinomycin)

Actinomycin D, also known as dactinomycin, a cytotoxic antibiotic produced by Streptomyces parvullus, was approved for medical use in the United States in 1964. It is on the World Health Organization’s List of Essential Medicines, the most effective and safe medicines needed in a health system. It is believed to work by blocking RNA synthesis. The drug has been used to treat a number of types of cancers, including Wilms tumor, rhabdomyosarcoma, Ewing’s sarcoma, trophoblastic neoplasm, testicular cancer, and certain types of ovarian cancer.

About Dr. Brunangelo Falini, M.D.

Brunangelo Falini is the head of the Institute of Hematology and Hemopoietic Stem Cell Transplantation at the University of Perugia, Perugia, Italy. His research activity has mainly focused on the genetic characterization of lymphomas and leukemias using monoclonal antibodies and, more recently, NGS technologies. He led the research group who discovered NPM1 mutations in AML in 2005 and the BRAF-V600E mutation in hairy cell leukemia in 2011. Both these seminal discoveries have already translated into a better diagnosis and therapy of patients affected by these hematological malignancies. Dr. Falini is the recipient of numerous prestigious prizes, including the "Josè Carreras Award" from EHA (Free EHA Whitepaper) (Barcelona, 2010), the "Leopold Griffuel Prize" from ARC (Paris, 2015) and the "Prize for Excellence in Medicine" from the American-Italian Cancer Foundation (New York, 2017).

On January 2, 2018 CymaBay Therapeutics, Inc. (NASDAQ:CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that Sujal Shah, President and Chief Executive Officer, will provide a corporate overview at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Thursday, January 11, 2018 at 9:30am PT (Press release, CymaBay Therapeutics, JAN 2, 2018, View Source [SID1234522816]).

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A live audio webcast of the presentation can be accessed through the Investors section of the CymaBay Therapeutics corporate website at View Source The webcast will be archived on the corporate website for 90 days.

On January 2, 2018 Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a biotechnology company discovering and developing innovative immuno-oncology protein therapeutics, reported that on December 27, 2017, the company initiated dosing in the Phase 1 portion of the FIGHT Phase 1/3 clinical trial (NCT03343301 ) of FPA144, an isoform-selective anti-FGF receptor 2b antibody, in combination with chemotherapy in patients with previously untreated, advanced gastric or gastroesophageal cancer (Press release, Five Prime Therapeutics, JAN 2, 2018, View Source [SID1234522818]).

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"Patients with advanced gastric cancer need new treatment options. Progression free survival with mFOLFOX6, a standard front-line therapy for gastric cancer, is typically less than seven months," said Charles Fuchs, M.D., Director of the Yale Cancer Center. "For patients whose tumors overexpress FGFR2b or have FGFR2 gene amplification, prognosis has been found to be particularly poor, so we are hopeful that a targeted therapy like FPA144 may provide a clinical benefit in this setting."

The open label Phase 1 portion of the trial will evaluate ascending doses of FPA144 in combination with the modified FOLFOX6 regimen (mFOLFOX6) to identify a recommended dose for Phase 3. Endpoints include safety, tolerability, and pharmacokinetic and pharmacodynamics parameters. Approximately 21 patients with unresectable, locally advanced, or metastatic gastrointestinal cancer will be enrolled during the Phase 1 portion of the FIGHT trial. FGFR2 status will be tested retrospectively but is not a requirement for enrollment.

This safety lead-in portion of the study is designed to support the Phase 3 portion of the trial, which Five Prime expects to transition to in mid-2018. Five Prime designed the randomized, controlled Phase 3 portion of the trial to serve as a global registrational study. The FIGHT trial will evaluate FPA144 plus mFOLFOX6 versus placebo plus mFOLFOX6 in approximately 550 patients with advanced gastric or gastroesophageal cancer whose tumors overexpress FGFR2b or have FGFR2 gene amplification. Five Prime will use immunohistochemistry and circulating tumor DNA tests to identify patients who would be eligible for inclusion in the trial. The primary Phase 3 endpoint is overall survival with progression free survival, objective response rate, and safety as secondary endpoints. The Phase 3 portion of the trial is expected to include sites in the U.S., Europe and Asia, including China and Japan, where the incidence of gastric cancer is high.

"We are very pleased to have the initial run-in to the FIGHT trial underway and we anticipate transitioning to the global, registrational portion of the trial this year," said Helen Collins, M.D., Senior Vice President and Chief Medical Officer of Five Prime. "We have seen encouraging monotherapy activity with FPA144 as a late-line treatment for gastric cancer and we believe that combining with chemotherapy in the front-line setting will provide the greatest patient benefit, as has been seen with other targeted therapies. Similarly, our pre-clinical data suggest that FPA144 should be additive when combined with chemotherapy."

Data from the Phase 1 clinical trial of single-agent FPA144 were presented at the 2017 ASCO (Free ASCO Whitepaper) Annual Meeting. FPA144 demonstrated single-agent activity and an acceptable safety profile in heavily pretreated patients with metastatic gastric cancer whose tumors overexpress FGFR2b.

Efficacy Results:

Objective Response Rate (ORR): 19.0%
Disease control rate at 6 weeks: 57.1%
Median duration of response of 15.4 weeks
Median number of prior therapies: 3
About FPA144
FPA144 is an isoform-selective, humanized monoclonal antibody in clinical development as a targeted immuno-therapy for tumors that overexpress FGFR2b, a splice variant of a receptor for some members of the fibroblast growth factor (FGF) family. Clinical results to date suggest that the specificity of FPA144 avoids toxicities that have been seen with less selective FGFR2 small molecule therapeutics. FPA144 has also been engineered for enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) to increase direct tumor cell killing by recruiting natural killer (NK) cells.

FPA144 is being evaluated in clinical trials as a potential treatment for gastric cancer and bladder cancer. An estimated 10% of patients with gastric cancer have tumors that overexpress FGFR2b or have FGFR2 gene amplification, which is associated with poor prognosis.