Treatment of Germline BRCA-Mutated Metastatic Pancreatic Cancer

AstraZeneca and Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has approved LYNPARZA for the maintenance treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm) metastatic pancreatic adenocarcinoma whose disease has not progressed on at least 16 weeks of a first-line platinum-based chemotherapy regimen. Patients will be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA.The approval follows the recommendation by the U.S. FDA Oncologic Drugs Advisory Committee on Dec. 17 for LYNPARZA in this indication and was based on results from the pivotal Phase 3 POLO trial published in the New England Journal of Medicine and presented at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting.Results showed a statistically significant and clinically meaningful improvement in progression-free survival (PFS) where LYNPARZA nearly doubled the time patients with gBRCAm metastatic pancreatic cancer lived without disease progression or death to a median of 7.4 months vs. 3.8 months on placebo (HR 0.53 [95% CI 0.35-0.81] p=0.0035).
Dave Fredrickson, executive vice president, head of the oncology business unit, AstraZeneca, said, “Patients with advanced pancreatic cancer historically have faced poor outcomes due to the aggressive nature of the disease and limited treatment advances over the last few decades. LYNPARZA is now the only approved targeted medicine in biomarker-selected patients with advanced pancreatic cancer.”
Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories, said, “LYNPARZA embodies Merck’s and AstraZeneca’s commitment to advance the treatment of challenging types of cancer, including metastatic pancreatic cancer. The expanded approval of LYNPARZA represents a significant milestone for patients and supports the value of germline BRCA testing in patients with this disease.” Dr. Hedy L. Kindler, co-principal investigator of the POLO trial and professor of medicine, University of Chicago Medicine, said, “Today’s approval of olaparib based on the POLO results gives clinicians an important first-line maintenance treatment option which nearly doubled the progression-free survival benefit in patients with germline BRCA-mutated metastatic pancreatic cancer.”
The Pancreatic Cancer Action Network (PanCAN) is a U.S.-based organization that supports and advocates on behalf of the patients, caregivers and communities affected by pancreatic cancer.LYNPARZA is currently approved in 65 countries for the maintenance treatment of platinum-sensitive relapsed ovarian cancer, regardless of BRCA status. It is approved in the U.S., the EU, Japan and several other countries as first-line maintenance treatment of BRCA-mutated advanced ovarian cancer following response to platinum-based chemotherapy. It is also approved in 44 countries, including the U.S. and Japan, for gBRCAm, HER2-negative metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer.

New Treatments for Gastrointestinal Disorders

Cerevance, a clinical stage biopharmaceutical company advancing new medicines for brain diseases, has formed a multi-year research alliance with Takeda Pharmaceutical Company Limited (“Takeda”) to identify novel target proteins expressed in the central nervous system and to develop new therapies against them for certain GI disorders. Scientists from the companies will select, confirm and validate targets from rich gene expression data sets generated by Cerevance’s NETSseq technology, which sorts and profiles specific neuronal and glial cell types in post-mortem human central nervous system tissue at unparalleled depth.Under the terms of the agreement, which includes an upfront technology access fee and research support to Cerevance, Takeda will pay preclinical and clinical milestone payments and royalties to Cerevance as targets are validated and compounds are advanced through development and onto the market worldwide. Cerevance is eligible to receive development, commercialization and net sales-based milestone payments that may exceed $170 million on a per target basis.

Chemotherapy-free Venclexta/Venclyxto-based treatments in chronic lymphocytic leukaemia

Basel. Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced updated data from two pivotal phase III Venclexta®/Venclyxto® (venetoclax) studies (MURANO and CLL14) that highlight Venclexta/Venclyxto combination treatments as chemotherapy-free, fixed-duration options that achieve minimal residual disease (MRD)-negativity, in people with chronic lymphocytic leukaemia (CLL). These data and others from the Venclexta/Venclyxto clinical development programme will be featured in more than 50 abstracts at the 61st American Society of Hematology (ASH) Annual Meeting.“Venclexta/Venclyxto plus anti-CD20 monoclonal antibody-based regimens continue to demonstrate improved long-term outcomes for people with chronic lymphocytic leukaemia,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “These results reinforce the sustained clinical benefits observed in patients with this common type of blood cancer, after completing this fixed-duration, chemotherapy-free treatment.”
The pivotal phase III CLL14 study evaluated the combination of Venclexta/Venclyxto plus Gazyva®/Gazyvaro® (obinutuzumab) in people with previously untreated CLL, who had co-existing medical conditions. At a median follow-up of more than three years (39.57 months), when all patients had been off therapy for a minimum of two years, Venclexta/Venclyxto plus Gazyva/Gazyvaro showed high response rates, including MRD-negativity. Specifically:Higher rates of MRD-negativity in peripheral blood (76% vs. 35%; p<0.001) and bone marrow (57% vs. 17; p<0.001%) were observed at the end of treatment in people treated with Venclexta/Venclyxto plus Gazyva/Gazyvaro versus Gazyva/Gazyvaro plus chlorambucil, respectively. MRD-negativity indicates that no cancer can be detected using a specific, highly sensitive test, and was defined as less than one CLL cell in 10,000 white blood cells.

Treatment of Venous Thromboembolism in Patients with Active Cancer

The Bristol-Myers Squibb-Pfizer Alliance today announced results from retrospective real-world data analyses reporting outcomes on the safety and effectiveness of Eliquis® (apixaban) compared to low molecular weight heparin (LMWH) or warfarin for the treatment of venous thromboembolism (VTE) in patients with active cancer (n=14,086). The real-world data analyses were highlighted during oral presentations at the American Society of Hematology (ASH) Annual Meeting in Orlando, Florida.
In a second oral presentation, results from a sub-group analysis of the primary study were highlighted based on different levels of risk for developing recurrent VTE, a blood clot most often found in the legs or lungs. Study findings were generally consistent with the primary analysis. It is important to note that, anticoagulants, including Eliquis, increase the risk of bleeding and can cause serious, potentially fatal bleeding. VTE is the third most common cause of vascular death after heart attack and stroke.i VTE can be a major health problem among patients with cancer, with studies showing that patients with cancer are at a significantly increased risk for VTE compared to those without cancer.ii VTE is also a leading cause of death in cancer patients, and a significant predictor for all-cause mortality.iii,iv
In these real-world analyses, four U.S. commercial insurance claims databases were used to identify VTE patients with active cancer (defined as cancer diagnosis or cancer treatment [chemotherapy, radiation and/or cancer-related surgery] within six months before or 30 days after VTE diagnosis) who initiated apixaban, LMWH, or warfarin within 30 days following the first VTE event. The risk of events was evaluated using a Cox proportional hazard model. Inverse probability treatment weighting (IPTW) was used to balance patient characteristics between apixaban, LMWH, and warfarin cohorts. Patients were followed to the earliest of: health plan disenrollment, death, index therapy discontinuation, switch to another anticoagulant, study end, or a maximum of 6 months. This was done to evaluate the rates of MB, CRNMB, and recurrent VTE (fatal or non-fatal) among VTE patients with active cancer prescribed apixaban, LMWH, or warfarin in routine clinical practice.

Lifesaving Addiction Treatment Opens in Memphis

Spero Health, a CARF-accredited, community-based organization and national leader specializing in outpatient addiction treatment of substance use disorders, has announced the opening of an additional clinic in West Tennessee. The Memphis Spero Health Clinic, located at 920 Estate Drive, Suite 8, will open its doors to welcome new patients on Friday, November 15th. Individuals who need addiction treatment services are encouraged to call 901-302-9532 today to schedule an appointment or walk-into treatment on November 15th.Spero Health is enrolled with TennCare and accepting Medicaid as well as select commercial insurance plans to ensure access to care is not an issue.Dr. Dustin Inman, who is board-certified in Family Medicine and board-eligible in Addiction Medicine, will be the lead physician at this clinic. A graduate of UT Memphis Medical School, Dr. Inman completed his residency in Jackson at the University of Tennessee Family Medicine Center where he was the Chief resident in the 2004 class.In Memphis there were 854 opioid related emergency department visits in 2018 and over 600 deaths involving opioids between 2013-2017, according to the Tennessee Department of Health. Tennessee remains in the top of all states in drug overdose deaths. Recently, there’s been an increase in polysubstance use as drugs like methamphetamine are on the rise. Across the country, more and more opioid users say they now use meth as well, up from 19% in 2011 to 34% in 2017, according to a study published last year in the journal Drug and Alcohol Dependence.

PolarityTE Announces Positive Pilot Study Data of SkinTE as a Treatment for Venous Stasis Leg Ulcers

PolarityTE, Inc. (Nasdaq: PTE), a biotechnology company developing and commercializing regenerative tissue products and biomaterials, today announced findings from an open-label, single-arm pilot study, which examined the impact of SkinTE™, a novel human cellular and tissue-based product derived from a patient’s own skin, in closing venous stasis leg ulcers (VLUs) following failure of conventional treatments. The clinical outcomes were reported in a poster presentation, entitled Pilot Study Assessing Novel Autologous Homologous Skin Construct Treatment of Venous Stasis Leg Ulcers, at the Symposium on Advanced Wound Care (SAWC) Fall Meeting, held in Las Vegas, October 12-14, 2019. In addition, the study poster abstract received the highest scores from reviewers in the Case Series/Study Category, the largest category at the conference.
The pilot study included 10 patients with VLUs that remained open after at least one month of conventional treatments. The patients were treated with SkinTE, a first-of-its-kind autologous, homologous human cellular and tissue-based product designed to regenerate full-thickness, functional skin for the repair, reconstruction, and replacement of a patient’s own skin.The study found an 80 percent closure rate of the VLUs within 12 weeks after treatment with SkinTE. In addition, all of the patients’ wounds demonstrated graft take and initial signs of closure, including granulation and progressive epithelialization, shortly after a single treatment with SkinTE. One treated VLU, which had previously been deemed closed, had reopened prior to the 2-week durability visit as a result of external factors unrelated to the SkinTE procedure. Further, another VLU did not close within 12 weeks, which was the largest in the study (12.2cm2) and had remained open for several months prior to treatment with SkinTE after previously failed treatment with a split-thickness skin graft; this VLU closed within 13.5 weeks post application of SkinTE.

Treatment of Adults With Rheumatoid Arthritis in Japan

Gilead Sciences, Inc. (NASDAQ: GILD) today announced that the New Drug Application (NDA) for filgotinib, an investigational, oral, selective JAK1 inhibitor for the treatment of adults with rheumatoid arthritis (RA) has been submitted to the Japanese Ministry of Health, Labor and Welfare (MHLW). The NDA for filgotinib is supported by data from the Phase 3 FINCH clinical trial program in which once-daily treatment with filgotinib demonstrated the potential to improve clinical signs and symptoms, to achieve low disease activity and remission, and to inhibit structural damage for patients living with RA. Safety data across the FINCH clinical trial program was consistent with previously reported results.“Despite multiple medications currently available to treat rheumatoid arthritis, there are still far too many people who do not experience adequate relief from their symptoms,” said John Sundy, MD, PhD, Senior Vice President, Inflammation and Respiratory Diseases, Gilead Sciences. “The FINCH clinical trial program demonstrated that filgotinib may offer a wide range of people living with RA, including those in the early treatment and those who have tried standard therapies without success, an important new treatment option.” “This new drug application is an important milestone as we continue to expand Gilead’s presence in Japan to now also include inflammation,” said Luc Hermans, MD, President and Representative Director, Gilead Sciences, K.K. “We are committed to bringing innovative products to patients and healthcare providers in Japan, expanding beyond antivirals into new areas where our medicines can make a meaningful difference to patients.”

Treatment of People With Rheumatoid Arthritis

Celltrion Healthcare today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for CT-P13 SC for marketing authorisation in the European Union (EU) in people with rheumatoid arthritis (RA). CT-P13 SC is the subcutaneous version of Remsima® (biosimilar infliximab, CT-P13). This recommendation will now be reviewed by the European Commission, which has the authority to approve medicines in the EU.A subcutaneous formulation has the potential to enhance treatment options for the use of biosimilar infliximab by providing high consistency in drug exposure and a convenient method of administration.1,2. This positive opinion is based on the phase I/III study to evaluate pharmacokinetics, efficacy and safety between CT-P13 SC and the intravenous (IV) version in people with active RA. Part one of the study demonstrated that CT-P13 SC showed comparable efficacy to CT-P13 IV up to week 54 with comparable DAS28 (CRP) / DAS28 (ESR) and ACR20 scores (measures of disease activity) demonstrated for both routes of administration. The safety profile of CT-P13 SC was also comparable to CT-P13 IV.1 The study was followed up by a phase I/III randomised controlled trial (part two) which demonstrated non-inferiority in efficacy of CT-P13 SC to CT-P13 IV in people with RA over 30 weeks with similar DAS28 and ACR20, ACR50, ACR70 scores and EULAR-CRP response. The safety profile of CT-13 SC was comparable to CT-13 IV up to week 30.2. Global Principal Investigator Professor Rene Westhovens, Rheumatologist at the University Hospitals KU Leuven, Belgium said, “This announcement is very encouraging as CT-P13 SC has demonstrated a comparable safety and efficacy profile to the well-established intravenous version of infliximab. This new SC formulation of infliximab could give patients the opportunity to administer the treatment themselves as an injection, giving them more control over their own treatment. Having two formulations of CT-P13 could also benefit patients by offering a more personalised treatment option whilst also reducing the time spent in hospital having intravenous treatment alone.”
A phase III study of CT-P13 SC for people with inflammatory bowel disease (IBD) is underway. Celltrion hope to seek expanded indications following the results of this trial. Celltrion Healthcare will adopt a different business strategy for CT-P13SC, compared to strategies for previous products in the portfolio and is considering both direct and indirect sales based on the optimal model in each local country. Celltrion has applied for a patent for CT-P13 SC in approximately 130 countries throughout the US, Europe and Asia.

Treatment of Men with Metastatic Hormone-Sensitive Prostate Cancer

Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) and Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the filing of a supplemental New Drug Application (sNDA) for XTANDI® (enzalutamide) to add an indication for the treatment of men with metastatic hormone-sensitive prostate cancer (mHSPC). The application has also been granted Priority Review, a designation given to those applications for drugs that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications. XTANDI is currently indicated in the U.S. for the treatment of patients with castration-resistant prostate cancer (CRPC).The submission is based on results from the Phase 3 ARCHES trial presented at the 2019 Genitourinary Cancers Symposium (ASCO GU) in February and published in The Journal of Clinical Oncology in July 2019. The study evaluated the efficacy and safety of XTANDI plus androgen deprivation therapy (ADT) versus ADT plus placebo in men with mHSPC. The primary endpoint of radiographic progression-free survival (rPFS) was met in the study.
Additionally, the submission is supported by data from ENZAMET, an Astellas-supported, investigator-sponsored Phase 3 research study led by the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP) and sponsored by the University of Sydney. The ENZAMET trial evaluated XTANDI plus ADT versus ADT plus a standard nonsteroidal antiandrogen therapy (bicalutamide, nilutamide or flutamide) in men with mHSPC to provide an active control. The results were presented during the Plenary Session at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting in June and simultaneously published in The New England Journal of Medicine. The primary endpoint of overall survival (OS) was met in the ENZAMET trial. The safety analyses of the ARCHES and ENZAMET trials appear consistent with the safety profile of enzalutamide in previous clinical trials in CRPC.

First FDA-approved treatment designed to target both ROS1 and NTRK that also shows response in cancer that has spread to the brain

Basel. Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the US Food and Drug Administration (FDA) has approved Rozlytrek™ (entrectinib) for the treatment of adults with ROS1-positive, metastatic non-small cell lung cancer (NSCLC). The FDA has also granted accelerated approval to Rozlytrek for the treatment of adult and paediatric patients 12 years of age and older with solid tumours that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. These approvals are based on results from the integrated analysis of the pivotal Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA-372-001 trials, and data from the Phase I/II STARTRK-NG study. In the integrated analysis, Rozlytrek was studied in several solid tumour types, including breast, cholangiocarcinoma, colorectal, gynaecological, neuroendocrine, non-small cell lung, salivary gland, pancreatic, sarcoma and thyroid cancers. In ROS1-positive, metastatic NSCLC, Rozlytrek shrank tumours in 78% of people with the disease (overall response rate [ORR]; N=51) and the duration of response (DoR) ranged from 1.8 to 36.8+ months (N=40 out of 51).1 Rozlytrek also shrank tumours in more than half of people with NTRK gene fusion-positive, locally advanced or metastatic solid tumours (ORR=57%; N=54), and objective responses were observed across 10 tumour types (DoR ranged from 2.8 to 26.0+ months; N=31 out of 54).1 Objective responses to Rozlytrek were seen in people with central nervous system (CNS) metastases at baseline.
The most common adverse reactions (≥20 percent) with Rozlytrek were fatigue, constipation, altered sense of taste (dysgeusia), swelling (oedema), dizziness, diarrhoea, nausea, nervous system disorders (dysaesthesia), shortness of breath (dyspnoea), muscle pain (myalgia), cognitive impairment, increased weight, cough, vomiting, fever (pyrexia), joint pain (arthralgia) and vision disorders. The FDA’s Accelerated Approval Program allows conditional approval of a medicine that fills an unmet medical need for a serious or life-threatening disease or condition.2 The accelerated approval for NTRK gene fusion-positive solid tumors is based on tumour response rate and durability of response, and continued approval may be contingent upon verification and description of clinical benefit in the confirmatory trials.Biomarker testing for ROS1 in NSCLC and NTRK gene fusions across all solid tumours is the only way to identify people who are eligible for treatment with Rozlytrek. Roche is leveraging its expertise in developing personalised medicines and advanced diagnostics, in conjunction with Foundation Medicine, to help identify people with ROS1 and NTRK gene fusions. Foundation Medicine will submit FoundationOne®CDx to the FDA for approval as a companion diagnostic for Rozlytrek. An FDA-approved companion diagnostic for Rozlytrek is not available at this time.

Treatment of Irritable Bowel Syndrome with Constipation

Alfasigma USA, Inc. today announced that ZELNORM™ (tegaserod) is now available in the United States as a prescription pharmaceutical treatment for irritable bowel syndrome with constipation (IBS-C) in adult women less than 65 years of age. Alfasigma USA acquired ZELNORM from Sloan Pharma S.à r.l., a subsidiary of US WorldMeds Holdings, LLC, in July 2019.“We are pleased to inform U.S. physicians and pharmacists about the reintroduction of ZELNORM and how Alfasigma USA is supporting the availability, access to, and appropriate use of this important medicine for the many patients who cope daily with an urgent need for new treatment options,” said Bryan Downey, president and chief executive officer at Alfasigma USA. “Our growing U.S. organization and national sales force is excited to lead the return of ZELNORM and committed to making a positive difference in the lives of patients.”
Originally approved in 2002 as the first prescription medication for IBS-C, ZELNORM was voluntarily withdrawn from the market in 2007 due to concerns regarding possible cardiovascular risk. In March 2019, ZELNORM was approved for reintroduction in the U.S. for use in adult women under 65 years of age with IBS-C, following a thorough safety review by the FDA and an FDA-assembled Gastrointestinal Drugs Advisory Committee (GIDAC). The evaluation consisted of a review of clinical data from 29 placebo-controlled trials and post-marketing treatment outcome data.
ZELNORM is the only serotonin-4 (5-HT4) receptor agonist approved to treat IBS-C. It targets the 5-HT4 receptor at multiple neurons (sensory, motor, secretory motor) and smooth muscle cells in the gastrointestinal tract to induce contraction and relaxation, and decrease pain signaling.

Strategic Alliance in FDC for Cancer Treatment

Biotechnology company Antikor Biopharma Ltd is pleased to announce that it has entered into an Investment Agreement for up to US$3,100,000 with Essex Bio-Investment, a wholly-owned subsidiary of Essex Bio-Technology Ltd (”EssexBio”), which will enable Antikor to consolidate and expand its position as a leading innovator in smaller-format conjugate therapies for solid tumours.Mahendra Deonarain, Antikor’s CEO and visiting Reader in Antibody Technology at Imperial College London where Antikor has its roots, commented: “We believe we have a platform that is tailored to make an impact in an area of major unmet medical need, and with EssexBio’s considerable commercial and clinical expertise, we now have the opportunity for translating the promised advantages of Antikor’s proprietary products into clinical benefit.”“We are excited to have established a strong alliance with Antikor”, said Malcolm Ngiam, President of Essex Bio-Investment, “Fragment-Drug Conjugate is an innovative approach with the potential to overcome many of the challenges faced by current treatment methods. The research and commercial partnership with Antikor is an important step towards developing first-in-class treatment for cancer.” Antikor’s novel technology platform will enrich Essex’s research pipeline and is aligned with EssexBio’s long-term research and commercial strategy.

First Non-Invasive, Adaptive Neuromodulation Digital Treatment for Migraine

Neurolief, a developer of next-generation digital therapeutics brain neuromodulation technology, today announced that it has received the CE mark for its Relivion™ non-invasive, adaptive digital treatment for migraine. The CE mark allows Neurolief to market, sell and distribute the Relivion device as an over-the-counter therapy within the European Union and countries that participate with Agreements on Mutual Recognition of Conformity Assessment. The Relivion is the first non-invasive, adaptive multi-channel brain neuromodulation technology that offers a highly effective therapy, without the risks and costs associated with invasive procedures and without the side effects related to medications. This type of therapy was previously possible only with implanted devices. The Relivion system is simple and safe for patients to self-administer at home at a fraction of the cost of surgical implants. Neurolief’s Relivion is comprised of a comfortable and adjustable headset that provides precise modulated pulses simultaneously to six branches of the occipital and trigeminal nerves via several adaptive output channels around the patient’s head. The sophisticated cloud-enabled device connects to a proprietary mobile phone app and learns over time to deliver personalized treatment for each patient.
The CE mark was granted based on data from a randomized, double-blind, placebo-controlled clinical trial which showed very high efficacy and safety of the Relivion in treatment of migraine headache. Seventy-six percent of the participants achieved headache relief after only one treatment while no serious adverse effect were observed. Full results of the trial will be presented at the American Headache Society’s 61st Annual Scientific Meeting on Saturday, July 13, 2019 in Philadelphia.
Migraine is a prevalent and debilitating primary headache disorder that affects approximately 12 percent of the population worldwide. The World Health Organization (WHO) estimates that 324 million people worldwide suffer from migraines. It is the most prevalent pain condition, prompting loss of productivity with an estimated economic annual burden of over €27 billion in European Union countries.

New Vaccine in the Treatment of Rheumatoid Arthritis

CEL-SCI Corporation (NYSE American: CVM) announced that one of its key collaborators from Rush University Medical center, Dr. Adrienn Markovitz an Assistant professor in the department of Orthopedic Surgery, presented new LEAPS data at i-Chem2019, International Conference on Immunity and Immunochemistry in San Francisco, California on Wednesday July 3, 2019. The title of her presentation was “Vaccination with DerG LEAPS peptide conjugates incorporating distinct PG (aggrecan) epitopes protects by different immune mechanisms in the PG G1 domain induced mouse model of rheumatoid arthritis”. Dr. Markovitz also presented a poster on other aspects of her work on modulation of autoimmune arthritis by the tyrosine phosphatase enzyme SHP-1, and identification of immunogenic citrullinated proteoglycan epitopes that may play a role in this disease in mice and humans.The LEAPS work was performed in conjunction with researchers at CEL-SCI on CEL-4000 and a newly discovered LEAPS conjugate, DerG-PG275Cit. Both vaccines were evaluated alone and in combination in the model of proteoglycan [PG] induced arthritis (PGIA) called recombinant PG G1 domain-induced arthritis (GIA), an autoimmune mouse model of rheumatoid arthritis (RA).The conclusion presented by Dr. Markovitz’s was that vaccination with DerG LEAPS conjugates of distinct PG epitopes alone or together were effective at modulating the inflammatory response and arresting the progression of arthritis. As observed previously, the DerGPG70 conjugate modulated disease through up-regulation of Th2, and down-regulation of Th1 and Th17 responses. The results suggest that the two vaccines act mechanistically on different immune responses. The combination of the two vaccines provided not only broader epitope coverage, but also a greater therapeutic effect than either vaccine alone.
The LEAPS platform technology is currently being utilized to develop a therapeutic antigen-specific treatment for RA under a $1.5 million grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the National Institutes of Health (NIH). Upon completion of preclinical and Investigational New Drug (IND) enabling studies for the antigen-specific LEAPS vaccine treatment of RA, CEL-SCI intends to file an IND application with the U.S. Food and Drug Administration.

Treatment of Patients with Acute Graft-Versus-Host Disease

Incyte Corporation (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has approved Jakafi® (ruxolitinib) for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older. Jakafi is the first and only FDA-approved treatment for this indication.The approval was based on data from REACH1, an open-label, single-arm, multicenter study of Jakafi in combination with corticosteroids in patients with steroid-refractory grade II-IV acute GVHD. Of the 71 patients recruited into REACH1, 49 patients were refractory to steroids alone, 12 patients had received two or more prior anti-GVHD therapies and 10 patients did not otherwise meet the FDA definition of steroid-refractory. Jakafi was administered at 5 mg twice daily, and the dose could be increased to 10 mg twice daily after three days in the absence of toxicity.The efficacy of Jakafi was evaluated based upon Day 28 overall response rate (ORR), defined as a complete response (CR), very good partial response or partial response based on the Center for International Blood and Marrow Transplant Research (CIBMTR) criteria. The Day 28 ORR in the 49 patients refractory to steroids alone was 57 percent with a CR rate of 31 percent. The most frequently reported adverse reactions among all 71 study participants were infections (55 percent) and edema (51 percent), and the most common laboratory abnormalities were anemia (75 percent), thrombocytopenia (75 percent) and neutropenia (58 percent).GVHD is a condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) where the donated cells initiate an immune response and attack the transplant recipient’s organs, leading to significant morbidity and mortality. There are two major forms of GVHD, acute and chronic, that can affect multiple organ systems including the skin, gastrointestinal (digestive) tract and liver. Patients who develop steroid-refractory acute GVHD can progress to severe disease, with one-year mortality rates of approximately 70 percent. Previously, the FDA granted Jakafi Breakthrough Therapy Designation and Orphan Drug Designation for the treatment of patients with steroid-refractory acute GVHD, and the supplemental New Drug Application (sNDA) was reviewed under the FDA’s Priority Review program.Jakafi will be made available to appropriate patients with steroid-refractory acute GVHD immediately. Incyte is committed to supporting patients and removing barriers to access medicines. Eligible patients in the U.S. who are prescribed Jakafi have access to IncyteCARES (Connecting to Access, Reimbursement, Education and Support), a comprehensive program offering patient support, including financial assistance and ongoing education and resources to eligible patients.

Immunotherapies for the treatment of solid tumors

Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of solid tumors, today announces that it has received Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA) to proceed with a Phase 1 clinical trial of its lead myvac™ candidate TG4050 as a potential treatment for ovarian cancer patients after first-line surgery and chemotherapy.
G4050 is an individualized MVA-based immunotherapy derived from the myvac™ platform. It has been designed to stimulate and educate the immune system of patients to recognize and destroy tumor cells. Tumor cells accumulate mutations and each patient has a set of mutations that are unique to his tumor. TG4050 is designed to target a panel of patient specific mutations selected using a NEC’s Neoantigen Prediction System“We are very pleased to have been granted an IND for TG4050 by the FDA allowing us to commence the first trial with our lead myvac™ candidate in ovarian cancer patients who have already received first-line treatment” said Maud Brandely, Chief Medical Officer of Transgene. “We believe individualized vaccination is a promising solution with significant potential to transform treatment outcomes for a wide range of solid tumors. With TG4050, we are confident that we can show that this therapeutic modality will improve patient outcome. We look forward to updating you on the progress of this clinical trial, which is expected to start later this year.”The Phase 1 clinical trial will evaluate the safety and the tolerability of TG4050 in patients with ovarian, fallopian or peritoneal serous cell carcinoma. Antitumor activity will also be measured. This multi-center, one-arm trial will recruit patients in the United States and Europe.The study, sponsored by Transgene, will be co-financed by Transgene and its partner NEC, which will also support the trial by contributing to the therapeutic vaccine design and the selection of target neoantigens.

“Transcatheter Treatment: Procedures and Heart Valve Market”

This report is an analytical business tool with the primary purpose of providing a thorough evaluation of the global market for transcatheter treatment for heart valve diseases.
Regionally, the U.S. is expected to maintain dominance in the global market of transcatheter treatment devices. Those wishing to invest in the transcatheter treatment devices market ought to strongly consider high-growth areas such as transcatheter aortic valve repair (TAVR), transcatheter mitral valve implantation (TMVI), and transcatheter mitral valve repair (TMVR) devices and technologies.The market is growing primarily due to the growing geriatric population, increasing the prevalence of chronic diseases, continuous technological advancements, increasing adoption of transcatheter techniques, and positive reimbursement and other healthcare reforms.

High-Level Dialogue on Diagnosis and Treatment of Paediatric HIV

Cardinal Peter Turkson, Prefect of the Dicastery for the Promotion of Integral Human Development, will convene a dialogue on Diagnosis and Treatment of Paediatric HIV at the Pontifical Academy of Sciences in the Vatican on 6-7 December 2018. He will address CEOs of pharmaceutical and diagnostic companies as part of the Holy See’s drive to assure optimal drugs formulations and testing tools for infants, children and adolescents living with HIV.
Building on consultations in April and May 2016 and November 2017, the meeting, fully titled High-Level Dialogue to Assess Progress on and Intensify Commitment to Scaling Up Diagnosis and Treatment of Paediatric HIV, will be organised by the co-chairs of the AIDS Free Working Group of the Start Free, Stay Free and AIDS Free Framework, PEPFAR, UNAIDS, in collaboration with partners of the AIDS Free Working Group, including Caritas Internationalis and the World Council of Churches-Ecumenical Advocacy Alliance.As well as leaders of major diagnostic and pharmaceutical companies, multilateral organisations, governments, regulators, faith-based and other organizations directly engaged in services to children living with HIV, and other key stakeholders, will participate in the consultation.The main aim of the meeting is to address bottlenecks that limit access to Early Infant Diagnostic products and programs and to scale up strategies that can help quickly identify HIV-exposed children and link them to testing and treatment services. In addition, the Dialogue will be an opportunity to assess progress on achieving the commitments made by participants in 2017.As the global community continues to make significant progress on scaling up access to adult HIV and AIDS testing and treatment services, more than 120,000 children continue to die each year from AIDS-related causes, and over 13,000 children are newly infected each month.
Participants in the Dialogue will agree to a plan of action to introduce and scale-up optimized diagnostics and case-finding strategies for HIV. Also, to intensify commitments in key challenging areas, including formulations development, and regulatory approval of optimal formulations and diagnostic products.Caritas Internationalis has taken strong responsibilities and commitments over mobilizing our network. We work with communities to help build treatment literacy and expand access to antitretroviral drugs among children. We also raise awareness in global fora about the unmet diagnostic and treatment needs of children with HIV.
Caritas members confirmed recently the need to continue to work at a global and national level for the wellbeing of children living with HIV. At the Dialogue, we will reaffirm our commitment and pledge to expand our efforts to help diagnose and treat those children.

Women About Symptoms, Hormones and Latest Treatments for Menopause

As approximately one third of a woman’s life is spent post-menopause, doctors from Yale University hosted Yale’s “One-Day Medical School: Everything You Always Wanted to Know About Menopause but Were Too Afraid to Ask” event on October 25, 2018 to provide essential health education for women 45+.“Public health and medicine improvements over the past 150 years have increased the life expectancy of women into their late 80s. While the age of menopause onset has not changed, the transition to menopause has larger implications as it relates to sexuality and overall heath, both physiological and psychological,” says Mary Jane Minkin, MD, Clinical Professor of Obstetrics & Gynecology at Yale University. “It’s important for women to recognize the early signs of menopause and learn how to cope with hot flashes, changes in sleep quality, joint and muscle health, sexual functioning, and hindrances to comfort in everyday life.” Live stream available at: https://www.faboverfifty.com/events/day-medical-school/

Pre-Clinical Evidence Demonstrates PBT434 as a Potential Treatment for MSA

Prana Biotechnology Ltd (ASX: PBT) (NASDAQ: PRAN) today announced further pre-clinical evidence for PBT434 will be presented in a poster at the International Congress of Parkinson’s Disease and Movement Disorders® in Hong Kong on Sunday, October 7th, 2018 at 1:45pm.The poster is entitled ‘PBT434 prevents the accumulation of glial cell inclusions and insoluble alpha-synuclein in a mouse model of Multiple System Atrophy’. Multiple System Atrophy (MSA) is a fatal neurodegenerative disease and an important form of atypical Parkinsonism. The data demonstrate in the most widely accepted animal model of the disease that PBT434 prevents α-synuclein aggregation, preserves neurons, decreases the number of glial cell inclusions and slows motor dysfunction. Glial cell inclusions are the key pathological finding in MSA and contain abundant aggregated α-synuclein that is associated with neurodegeneration. Alpha-synuclein is of great interest to scientists and clinicians because aggregated forms of the protein are toxic and underlie the pathology of neurological diseases such as MSA and Parkinson’s disease.“We are excited to share these data with the movement disorder community because Multiple System Atrophy is a terrible disease and has no approved treatments. The data indicate that PBT434 targets the key pathology in MSA and has excellent potential to treat this devastating condition. Our Phase 1 study in healthy volunteers is ongoing and we look forward to bringing PBT434 to patient studies in the future”, said David Stamler, Chief Medical Officer and Senior Vice President of Clinical Development.PBT434 is the first of a new generation of small molecules designed to inhibit the aggregation of α-synuclein and tau, vital intracellular proteins that are implicated in neurodegenerative diseases such as Parkinson’s disease and atypical Parkinsonism. http://www.pranabio.com.