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Saturday, December 15, 2018

FDA OKs Amgen Med For Pediatric Immune Thrombocytopenia


Application Granted Priority Review DesignationApproval Based on Results That Demonstrated Nplate Successfully Increased and Sustained Platelet Counts in Children Affected by Rare Blood Disorder
Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for Nplate®(romiplostim) for the treatment of pediatric patients one year of age and older with immune thrombocytopenia (ITP) for at least six months who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.
“Today’s approval underscores our long-standing commitment to making a positive impact on the lives of patients with rare and difficult-to-treat hematological disorders,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “In the 10 years since the FDA approved Nplate as the first platelet booster for adult patients with chronic ITP, it has made a difference in the lives of thousands of adults, and we’re proud to bring this treatment option to children who need it most.”
The approval was based on two placebo-controlled studies – Phase 3 and Phase 1/2 – evaluating the safety and efficacy of Nplate in pediatric patients. In the Phase 3 study, published in The Lancet, rates of overall platelet response were increased with the Nplate group (71 percent) compared with placebo (20 percent), p<0.05. Additionally, durable platelet response occurred more frequently with Nplate (52 percent) compared with placebo (10 percent), p<0.05. In the two placebo-controlled trials, adverse reactions with an incidence of > 25 percent in the Nplate arm were contusion, upper respiratory tract infection and oropharyngeal pain.
“Children with ITP are at risk for serious bleeding events and spontaneous bruising due to low platelet counts, which can be worrying for these young patients and their parents. Currently, these patients have a limited number of treatment options, especially for those with refractory disease,” said Michael D. Tarantino, M.D., president of the Bleeding and Clotting Disorders Institute and professor of Pediatrics and Medicine, University of Illinois College of Medicine-Peoria, Peoria, Ill. “Today’s approval of Nplate offers new hope to the pediatric ITP community as it provides children with a new treatment option that may help to maintain safe platelet counts.”
ITP is a rare, serious autoimmune disease characterized by low platelet counts in the blood (a condition known as thrombocytopenia) and impaired platelet production.1 In the U.S., the estimated prevalence of ITP in children is 5.3 per 100,000 children annually.1 The treatment goal for children with ITP is to achieve and maintain a platelet count that reduces the risk of bleeding.2
About Nplate® (romiplostim)Nplate is a thrombopoietin (TPO) receptor agonist that mimics the body’s natural TPO and is designed to increase platelet counts in patients with chronic immune thrombocytopenia (ITP).3 In the U.S. and European Union, Nplate is approved for the treatment of chronic ITP in adults and in children age one year and older with ITP for at least six months, who have had an insufficient response to other medicines or had surgery to remove the spleen.
Nplate is also approved in 67 countries, including Canada, Australia and Japan.
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BOEHRINGER Vargatef plus docetaxel may be option in lung cancer: ESMO


  • Results from a real-world study1 support a recent update to the ESMO guidelines2 recommending nintedanib (Vargatef®) plus docetaxel following first-line chemotherapy (+/- immunotherapy) in advanced NSCLC of adenocarcinoma histology

Boehringer Ingelheim announced the first interim results of VARGADO, an ongoing non-interventional study in routine clinical practice in Germany evaluating the efficacy and safety of Vargatef® (nintedanib) and docetaxel in patients with stage III/IV locally advanced or metastatic non-small cell lung cancer (NSCLC) of adenocarcinoma histology. The study consists of three cohorts, two of which allow for prior treatment with immune checkpoint inhibitors (ICIs) either in combination with chemotherapy in the first-line or as monotherapy in the second-line of treatment. The interim results from Cohort B (first-line chemotherapy, second-line immune checkpoint inhibitors, third-line Vargatef® plus docetaxel) were presented at ESMO Immuno-Oncology Congress 2018 in Geneva.1 Despite a limited sample size, the VARGADO study adds to the body of evidence for nintedanib in lung adenocarcinoma following pre-treatment with chemotherapy and ICIs.1,3

The addition of nintedanib to docetaxel has a proven survival benefit as a second-line treatment following chemotherapy in a broad population of lung adenocarcinoma patients.4 However, data are scarce regarding the efficacy and safety of nintedanib in adenocarcinoma patients who have been pre-treated with chemotherapy as well as ICIs.

The results support a recent update to the ESMO guidelines around the treatment of NSCLC, which now recommend nintedanib in combination with chemotherapy as a second-line treatment following prior chemotherapy +/- ICIs.2 A recent report by Jesus Corral et al (WCLC, 2017) which evaluated nintedanib in combination with docetaxel in a similar setting showed similar efficacy and safety information.3

Professor Christian Grohé, Department of Respiratory Diseases, ELK Berlin, said: “As immunotherapy becomes the first-line treatment of choice in advanced lung adenocarcinoma, it’s important to have effective and safe treatment options following immunotherapy. The results from VARGADO provide important evidence supporting the use of nintedanib following initial treatment with immunotherapy and are in line with the recent update to the ESMO guidelines.”

Dr Victoria Zazulina, Global Head of Solid Tumour Oncology, Medicine at Boehringer Ingelheim, said: “With a devastating disease like lung cancer, every option matters. Today’s results provide evidence that nintedanib, when added to docetaxel, is a good therapeutic option for patients that progress following treatment with immunotherapy.”
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Whole-Body Cyrotherapy Caused Cold Burn Injury


Another injury related to whole body cryotherapy (WBC) has been reported by practitioners in Philadelphia, serving as yet another warning of WBC’s potential to cause serious adverse effects.
As detailed by Jordan Wang, MD, of Thomas Jefferson University and colleagues in the Journal of the American Academy of Dermatology, a 71-year old patient presented to the center with a cold burn injury that he had sustained while undergoing WBC at a cryotherapy facility the day before. The man had opted to use WBC for the treatment of his back pain and arthritis.
The researchers said they suspected that a nozzle through which liquid nitrogen is sprayed into the chamber had malfunctioned and that the liquid nitrogen was instead sprayed directly on the patient’s back for a brief period of time.
“This is an unfortunate case of a burn secondary to cold, [and] it can be compared to a frost bite,” Cameron Rokhsar, MD, of New York Cosmetic, Skin & Laser Surgery Center in New York, told MedPage Today via email when asked for his perspective. “The cooling treatment protocol is not Food and Drug Administration-approved, and there is no proven scientific benefit to this treatment, but there is a real risk of burn as is highlighted by this case,” he said.
The authors agreed, and noted in their case report that “WBC is a new and trendy treatment of which practitioners should be aware because of potential adverse events.”
The burn patient was subsequently treated with systemic steroids, topical corticosteroids, ibuprofen, and silver sulfadiazine cream to protect his denuded skin.
Wang and colleagues explained that spas, wellness centers, and cryotherapy facilities have opened up to the general public in recent years and have begun offering WBC to anyone willing to pay for it. “The cryotherapy chamber is appropriately the size of a phototherapy light box,” Wang explained. “And while standing in the chamber, the head of the patient is above the top and outside of the chamber,” with the rest of the body is “bombarded” with a liquid nitrogen mist that cools the chamber from -100° to -140° C.
Each session lasts for 2-5 minutes, and proponents of WBC think that people can engage in several sessions a week. Treatment is thought to hasten muscle recovery and alleviate back pain and muscle stiffness. WBC is also claimed to improve energy and sleep patterns as well as skin health. However, the FDA has never approved any type of WBC chamber and in fact, warns against their use because of the potential harmful effects including frostbite, burns, eye injury, and even asphyxiation.
A Cochrane review published in 2015 found insufficient evidence to support any claims that WBC can prevent or treat muscle soreness after exercise, and several documented adverse events related to WBC have been reported in the literature.
For example, one case report documented an abdominal aortic dissection in a middle-aged male following multiple WBC sessions, and another report described a patient who developed an episode of cold panniculitis after eight WBC treatments.
As Rokhsar explained, these cooling machines use liquid nitrogen. “If the liquid nitrogen or its vapors come in contact with the skin, it can induce a burn,” he added. The degree of the burn will depend on the severity and duration of exposure to the liquid nitrogen, but even short bursts of exposure can cause blisters and pain just like a burn from sources of heat.
Dermatologists often use liquid nitrogen to destroy unwanted growths on the skin such as warts and precancerous lesions by spraying nitrogen on the lesion in a controlled manner, but that is quite different to WBC.
“Cold burns are much less common than their thermal counterparts,” Wang and co-authors pointed out. Nevertheless, freezing temperatures to which individuals are exposed during WBC can cause intracellular water crystallization, damaging proteins and membranes, and the resulting vasoconstriction induced by cold temperatures can also lead to hypoperfusion; endothelial injury may decrease vascular integrity.
Wang and co-authors reported no conflicts of interest.
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Meridian Gets FDA Clearance for New Neonatal Saliva CMV Test


Meridian Bioscience, Inc. (VIVO) announced that it has received FDA clearance for its new Alethia™ CMV Molecular Amplification Test (formerly, the illumigene brand). This assay is designed to specifically detect congenital Cytomegalovirus (cCMV) infection in newborns from an easy-to-collect saliva sample. Alethia CMV is the first qualitative test in a molecular amplification format, FDA-cleared for cCMV testing in newborns.
Congenital CMV is the most common congenital infection and a leading cause of childhood hearing loss, cognitive deficits, and visual impairment. According to the CDC about 1 out of every 200 babies are born with cCMV infection; and approximately 10% to 25% of all childhood sensorial hearing loss (SNHL) can be attributed to cCMV. Babies are at risk of infection during pregnancy if the virus in the mother’s blood crosses through the placenta. Early detection is critical in establishing appropriate treatment. Diagnosis can be attained by detecting the virus in a baby’s saliva or urine within 2 to 3 weeks from birth. CMV is a public health issue and legislation has been passed or is under consideration in numerous states regarding CMV education and testing recommendations in neonates.
Lawrence Mertz, Senior Vice-President of Research and Development, stated, “We are excited to be the first to develop and bring to market a very important test for newborns. It is an advancement in testing for a viral infection that can lead to such complicated and long-lasting conditions in children.”
Jack Kenny, Chief Executive Officer, said, “Unfortunately cCMV infection is more common than other newborn related illnesses, like Group B Strep for example, yet the level of awareness is considerably lower. With Alethia CMV, we not only look to increase awareness, but also provide laboratories with an FDA-cleared test that they can use with confidence when diagnosing newborns with cCMV. Alethia CMV helps meet a critical need with a simple-to-collect saliva sample in combination with a procedurally simple, rapid, and sensitive test. Alethia CMV marks the launch of the new branding for our current molecular platform.”
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Atara Positive Results for Epstein-Barr Virus-Associated Leiomyosarcoma: ESMO


— Second EBV‑associated solid tumor with encouraging responses to tab-cel® — Tab-cel® safety appears consistent with a favorable risk profile and previous observations — Results were presented today in an oral session at the European Society for Medical Oncology Immuno-Oncology Congress 2018
Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, today presented results indicating that tab-cel® (tabelecleucel) was generally well tolerated with responses for patients with Epstein-Barr virus-associated leiomyosarcoma (EBV+ LMS). EBV+ LMS is a rare soft tissue sarcoma that occurs in transplant and immunosuppressed patients and is typically an aggressive radiation- and chemotherapy-resistant disease with poor patient outcomes. The results were presented in an oral session at the European Society for Medical Oncology Immuno-Oncology (ESMO I‑O) Congress 2018 taking place in Geneva, Switzerland.
“The EBV+ LMS results presented at ESMO I-O are the second example, along with nasopharyngeal carcinoma (NPC), of a difficult-to-treat, EBV-associated solid tumor with encouraging responses to tab‑cel®,” said Dietmar Berger, M.D., Ph.D., Global Head of Research and Development of Atara Biotherapeutics. “Observations of responses based on standard-CT and metabolic PET-CT imaging, in the context of prolonged survival, further highlight the opportunity for tab‑cel® and off-the-shelf, allogeneic T-cell immunotherapy in EBV-associated cancers beyond our ongoing studies for patients with post‑transplant lymphoproliferative disease (PTLD) and NPC.”
The oral presentation summarized the evaluation of tab-cel® in an analysis of EBV+ LMS patients from three clinical studies, 2 single-center, open-label studies (NCT00002663, NCT01498484) and the multi‑center expanded access protocol (EAP) study (NCT02822495). Twelve patients with EBV+ LMS received one or more doses of tab-cel®, of whom 10 were assessed for responses with two patients not evaluable. Two of the 10 patients achieved a partial response via CT-based RECIST 1.1 criteria and eight patients achieved stable disease. In the two single-center studies with longer follow-up, six of eight patients survived more than 27 months and the estimated median survival was 77.4 months. At the time of this analysis, responses assessed by PET-CT imaging were available from the multi-center EAP study where 3 of the 4 patients achieved a metabolic response. Tab-cel® was generally well tolerated and the safety appeared consistent with a favorable risk profile and previous clinical studies.
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Noxxon latest data from NOX-A12 / KEYTRUDA combo trial at ESMO


NOXXON Pharma N.V. (Euronext Growth Paris: ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), announced today the details of its participation at the ESMO Immuno-Oncology Congress on December 13-16, 2018 at the Palexpo Exhibition Center, 30 Route François-Peyrot, 1218 Le Grand-Saconnex, in Geneva, Switzerland.
Poster Title: Safety and clinical outcome in patients with microsatellite-stable, metastatic colorectal or pancreatic cancer treated with the CXCL12 inhibitor NOX-A12 in combination with PD-1 checkpoint inhibitor pembrolizumab
The poster will be presented on Friday, December 14 from 12.30-01.00 p.m. CET in Room B and the authors will be available for questions. The poster will also be uploaded to the NOXXON website.
The abstract which is based on an earlier interim dataset will be published on Monday, December 10 at 12.00 p.m. CET on the ESMO website.
Lead investigator of the trial, Dr. Niels Halama, will discuss some of the results from the poster in a presentation entitled “A goal for immunotherapy in pancreatic cancer” on Sunday, December 16, from 09.50-10.10 a.m. CET in Room A.
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IMV Positive Data From Phase 1b/2 Ovarian Cancer Combo at 2018 ESMO


IMV Inc. (Nasdaq: IMV; TSX: IMV), a clinical stage immuno-oncology corporation, today announced that investigators shared new positive data from the company’s ongoing DeCidE1 (DPX-Survivac with low dose CyclophosphamIDe and Epacadostat) clinical trial at the 2018 ESMO Immuno-Oncology Congress. The phase 1b/2 study is evaluating the safety and efficacy of the combination of IMV’s lead candidate DPX-Survivac, low dose cyclophosphamide, and 100 mg or 300 mg of Incyte’s IDO1 enzyme inhibitor epacadostat in patients with advanced recurrent ovarian cancer.
In a poster presentation, Oliver Dorigo, M.D., Ph.D., Associate Professor of Obstetrics and Gynecology (Oncology), Stanford University Medical Center, who served as the trial’s lead investigator and author on the poster, shared topline safety results from 53 enrolled patients and efficacy data from the 32 participants evaluable for immune-related and clinical responses, as well as blood sample and tumor biopsy analyses.
Key findings include:
  • Evidence of a clinical marker based on Baseline Tumor Burden (BTB), a measure of tumor size predictive of patient response to DPX-Survivac.
    • 37.5% (12/32) of evaluable study subjects began treatment with a non-bulky disease defined as BTB < 5 cm.
    • 73% (8/11) of tumor regressions and 80% of clinical responses (4/5) observed in subset of patients with BTB < 5 cm.
  • Responders thus far showing prolonged duration of clinical benefits reaching up to more than two years, surpassing the progression-free interval from their previous chemotherapy treatment.
  • Robust systemic survivin-specific T cell responses and evidence of survivin-specific T cells tumor infiltration correlated with clinical benefits.
    • 100% of durable clinical responses correlated with T cell infiltration.
  • Epacadostat triggered inhibition of the conversion of tryptophan into kynurenine that was dose dependent.
  • Cohort demographics were balanced and the combination yielded a tolerable safety profile.
“This data set provided meaningful information on how the potential benefits of DPX-Survivac may best be translated to patients, including the connection between tumor regressions and T cell infiltration in the tumor microenvironment,” said Frederic Ors, Chief Executive Officer at IMV. “We believe that DPX-Survivac is the first targeted T cell therapy to induce significant tumor regressions in challenging tumors such as those seen in ovarian cancer. We remain committed to developing DPX-Survivac for patients with significant unmet medical needs, and look forward to our upcoming discussions with regulatory authorities in the USA, Canada and Europe.”
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