12 IG Living | August-September 2018 | IGLiving.com

IN THE NEWS

Prometic Life Sciences’ Phase IIIclinical study of its intravenousimmune globulin (IVIG) 10% producthas met its primary and secondaryendpoints in adult patients with pri-mary immunodeficiencies (PIs). Theprimary endpoint was less than oneserious bacterial infection (SBI) perperson per year. An SBI is defined asbacterial pneumonia, bacteremia andsepticemia, osteomyelitis/septic arthritis,bacterial meningitis or visceralabscess. Secondary endpoints includedepisodes of fever (100.4 degrees

Fahrenheit or less), number of misseddays from work, number of days ofhospitalization due to infection, num-ber of days on antibiotics, number ofinfections other than SBI and troughIgG levels comparable to other com-mercial products. With Prometic’sIVIG, only 4.94 days per subject peryear were lost from work, which wassignificantly less than the rate observedwhile on another commercial product.In addition, the proportion of infu-sions for which at least one treatment-emergent adverse event was well within

the U.S. Food and Drug Admini-stration (FDA) guidance thresholdacross all time points within 72 hourspost-infusion.“The results with Prometic IGIV10% met the FDA guideline require-ments for both safety and efficacy. Thisis the second plasma-derived therapeuticclinical program to generate pivotalPhase III results,” said Pierre Laurin,president and CEO of Prometic. Prometic Reports Positive Clinical Data from Its IntravenousImmunoglobulin (IVIG) Pivotal Phase 3 Trial. Prometic pressrelease, April 30, 2018. Accessed at www.prnewswire.com/news-releases/prometic-reports-positive-clinical-data-from-its-intravenous-immunoglobulin-ivig-pivotal-phase-3-trial-300638716.html.

Research

Prometic’s Phase III IVIG Trial Shows Positive Results

Evolve Biologics has dosed the firstpediatric patient in its Phase III multi-center clinical trial of PlasmaCap IG(intravenous immune globulin [IVIG]),in addition to having administered halfof the infusions required for the adultportion of the study, which completedenrollment in February. The studyreceived approval to proceed from theU.S. Food and Drug Administrationand Health Canada last summer.PlasmaCap IG is an investigationalIVIG replacement therapy being

studied in both adult and pediatricpatients with primary immunodefi-ciency diseases (PI). It has beendeveloped utilizing Evolve’s PlasmaCapEBA technology, which allows for theefficient capture of plasma proteinsand offers the potential of higher yieldsand purities. Existing plasma proteinproducts available in the market andderived from human plasma are stillprimarily produced using the tradi-tional Cohn manufacturing processdeveloped in the 1940s.The clinical trial has 13 study centersin the U.S. and Canada. Titled “AProspective, Open-Label, MulticenterStudy of the Efficacy, Safety,Tolerability, and Pharmacokinetics ofEvolve PlasmaCap IG in Adults andChildren with Primary ImmuneDeficiency Diseases,” the study willdetermine, based on historical controldata, how PlasmaCap IG compareswith other 10% IVIG products currentlylicensed in the U.S. and Canada for

the treatment of PI patients.“We are pleased to announce wehave now dosed the first pediatricpatient in this important study ofPlasmaCap IG. In addition, we havesuccessfully reached the mid-point forthe adult portion of our study, havingadministered half of the total infu-sions,” said Blaine Forshage, Evolve’sCEO. “These events mark importantprogress in this study. PlasmaCap IG isthe first product in our portfolio to bestudied in a clinical setting anddemonstrates our commitment tobringing modern and innovativetechnologies to the growing globalmarket for plasma-derived therapeutics,as well as developing new therapeuticoptions for both adult and pediatricpatients with a number of rare chronicdiseases, including PI.”

Evolve Biologics Reaches Major Milestones in Phase III Trial forPlasmaCap IG (Intravenous Immunoglobulin), Including Dosing ofFirst Pediatric Patient. Evolve Biologics press release, May 4, 2018.Accessed at www.businesswire.com/news/home/20180504005168/en/Evolve-Biologics™-Reaches-Major-Milestones-Phase-III.

Research

New 10% IVIG Being Tested for Pediatric and Adult PI Patients

IN THE NEWS

A first-ever prospective economicanalysis by Canadian investigatorsfound that, from both hospital- andhealth system-based perspectives,home-based subcutaneous immuneglobulin (SCIG) therapy was associatedwith significantly lower average totalnondrug costs than hospital-basedintravenous immune globulin (IVIG)therapy for patients with primaryimmunodeficiency (PI) disorders.The analysis included 30 adultpatients in the IVIG group and 27patients in the SCIG group. Theaverage age and baseline weight werenot significantly different betweenthe two groups. Patients on IVIG

therapy typically came to the hospitalevery three to four weeks where anurse inserted an intravenous line forinfusions that generally requiredabout two to three hours. Initiationof SCIG treatment required trainingby a qualified nurse, generally in asingle one-on-one visit. Once patientshad been trained, they infused theproduct on their own at home, gener-ally in small volumes ranging fromone to seven times per week. Forpatients transitioning from IVIG toSCIG at the beginning of the study,treatment was initiated at a doseequivalent to the previous IVIG dose,given once a week.

Over the 12-month study period, allnondrug hospital costs (includinghospital nurses and technicians) andphysician visit costs were respectively$1,836 and $84 for the SCIG group, and $4,187 and $744 for the IVIG group. “SCIG has significantlydecreased costs for the Canadianhealth care system compared withIVIG,” the investigators concluded. “Itshould be considered in patients whoare currently on IVIG and in thosewho are to start immunoglobulinreplacement therapy.”

Fu, LW, Song, C, Isarunuwatchai, W, et al. Home-Based SubcutaneousImmunoglobulin Therapy vs Hospital-Based IntravenousImmunoglobulin Therapy: A Prospective Economic Analysis. Annalsof Allergy, Asthma and Immunology, 2018 Feb;120(2):195-9.

Research

SCIG Therapy Is Cost-Saving Versus IVIG for PI Patients

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14 IG Living | August-September 2018 | IGLiving.com

The U.S. Food and Drug Admini-stration (FDA) has approved Tavalisse(fostamatinib) to treat patients withchronic immune thrombocytopenia(ITP) who have had insufficientresponse to another therapy. Approvalis based on findings from three trialsthat enrolled 150 patients who hadreceived prior ITP treatment thatconsisted of corticosteroids (84 per-cent), immune globulin (53 percent),thrombopoietin receptor agonists (48percent) or splenectomy (35 percent),nearly half of which were on stableconcurrent ITP therapy (4 percent). In the first randomized study knownas FIT-1, 18 percent of patients treatedwith Tavalisse experienced a plateletresponse compared with none in the

placebo arm. In the second studyknown as FIT-2, a stable plateletresponse was seen in 16 percent ofpatients in the Tavalisse group com-pared with 4 percent treated withplacebo. Patients from these trials werealso included in an open-label expan-sion cohort known as FIT-3 in which23 percent of those who received priorplacebo in FIT-1 or FIT-2 had aplatelet response to Tavalisse. In allstudies, Tavalisse was started at 100mg twice daily with a dose escalationto 150 mg twice daily based on plateletcounts and tolerability. Most patients(88 percent) were dose-escalated atweek four or later. The primary end-point was stable platelet response,which was defined as at least 50 x 109

platelets per liter of blood on at leastfour of six visits between weeks 14 and24 of the study.“Chronic ITP is challenging to treatbecause the heterogeneity of the diseasemakes it difficult to predict how anindividual patient will respond to avail-able treatments, and not all patients canfind a treatment that works well forthem,” said lead investigator of thetrials James Bussel, MD, professoremeritus of pediatrics at Weill CornellMedicine. “The FDA approval offostamatinib arms physicians with anew treatment option, which works viaa novel mechanism.”

Inman, S. FDA Approves Drug for Chronic ImmuneThrombocytopenia. Cancer Updates, Research and Education, April18, 2018. Accessed at www.curetoday.com/articles/fda-approves-drug-for-chronic-immune-thrombocytopenia.

Medicines

FDA Approves Tavalisse to Treat Chronic Immune Thrombocytopenia

Significant improvements in cognitiveand behavioral function were observed in14 children with autism spectrumdisorder and evidence of immune dys-function, who were administered high-dose intravenous immune globulin(IVIG) treatment over a period of 30weeks, according to a pilot study con-ducted by U.S. investigators.A select group of autistic children witha diagnosis of autistic disorder, Asperger’sdisorder or pervasive developmentaldisorder and evidence of a dysregulatedimmune system received 1 g/kg of 5%IVIG (Gammaplex, Bio ProductsLaboratory) for 10 21-day treatmentcycles. The primary endpoint was pre-and posttreatment disease improvementassessed using standardized cognitive

and behavioral tests (e.g., Children’sCommunication Checklist [CCC-2],Social Responsiveness Scale [SRS],Aberrant Behavior Checklist [ABC],Clinical Global Impressions-Severity[CGI-S] and Improvement [CGI-I], andAutism Diagnostic Observation Schedule[ADOS]). A number of experimentalbiomarkers associated with neuroin-flammation were also captured.

Significant improvements from base-line to study endpoint were observed inseveral sub-scales of the CCC-2, SRS,CGI-I, CGI-S and ADOS, includingassociated maladaptive behavior, recip-rocal social interaction, communicationand stereotyped behaviors and repeti-tive interests. Statistically significantreductions were also seen in numerousimmunological biomarkers indicative ofneuroinflammation. IVIG treatmentswere well-tolerated. These findings sug-gest inflammatory etiologies may playa role in some cases of autism, and IVIGtreatment may, through an anti-inflam-matory effect, exert a positive impact onits behavioral manifestations.Melamed, IR, Heffron, M, Testori, A, et al. A Pilot Study of High-Dose IntravenousImmunoglobulin 5% for Autism: Impact on Autism Spectrum andMarkers of Neuroinflammation. Autism Research, 2018 Mar;11(3):421-33.

Research

Autistic Children Treated with IVIG Show Improvement

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European researchers have discoveredthe mechanism behind the antibodyimmunoglobulin E (IgE) that canpotentially inactivate the body’sallergic processes. It is hoped thebreakthrough will help to develop auniversal treatment to prevent allergicreactions.When an allergic response to acompound occurs, the body’s immunesystem produces IgE molecules thatbind to receptors on mast cells, causingthe release of histamines that result inallergic symptoms. But, this newresearch shows anti-IgE antibodiescan prevent the allergen-induced IgEmolecules from binding with the

histamine-producing mast cells toeffectively stop all allergic symptomsregardless of the volume of IgE mole-cules generated by the external aller-gen. “Once the IgE on immune cellscan be eliminated, it doesn’t matterthat the body produces millions ofallergen-specific IgE molecules,” saidEdward Spillner from the Departmentof Engineering at Aarhus Universityin Denmark. “When we can removethe trigger, the allergic reaction andsymptoms will not occur.”However, the antibody has onlybeen shown to be effective in ex vivo laboratory models. While theresearchers believe the method should

be effective in almost all allergic reactionsin humans, it is still years away frombeing deployed in general medicine. Haridy, R. Antibody Breakthrough Brings Universal Allergy TreatmentOne Step Closer. News Atlas, Jan. 28, 2018. Accessed atnewatlas.com/universal-allergy-antibody-treatment/53153.

Research

Antibody Discovery Could Prevent Allergies