Op uitnodiging van het ministerie van VWS reageerde HollandBIO vorige week op de voorgenomen wijziging van de Wet Geneesmiddelenprijzen, de WGP. Het vervangen van referentieland Duitsland door Noorwegen realiseert jaarlijks een besparing van 239 miljoen euro, zo becijfert de Stichting Farmaceutische Kengetallen. De maatregel is het zoveelste lapmiddel om de zorgkosten te beheersen door minimalisatie van de geneesmiddelenuitgaven. Geneesmiddelen fungeren zo als kiloknallers van de zorg, in plaats van als hofleveranciers voor volksgezondheid. In onze reactie breken we een lans voor duurzame oplossingen waarbij gzondheidswinst, innovatie en betaalbaarheid hand in hand gaan. Je leest onze brief hier.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-08-28 14:13:482018-08-28 14:16:45Geneesmiddelen: van kiloknaller naar hofleverancier← #nieuws
Madam Therapeutics closed its first financing round of 1,1M EUR. The biotech company develops a novel class of antibiotics inspired by the defense mechanisms of the human body. The new funding will be used to continue and accelerate the clinical development of Madam Therapeutics’ lead candidate SAAP-148, by executing a first-in-men proof of concept study starting early next year and preparation of the following clinical phase II trial. The investment comes from a network of European business angels facilitated by Ekoy Investment Partners, De Investeerders Club and Investormatch.
Lead candidate: SAAP-148
When a small subset of bacteria survives antibiotic treatment e.g. by mutations, an infection can get out of control fast. As these resilient microbes thrive, they can group together on a surface—like a wound or a medical device—and encase themselves in a slimy protective layer known as a biofilm. Such biofilm infections “are the really nasty things for patients,” says immunologist Peter Nibbering at Leiden University Medical Center in the Netherlands with whom Madam Therapeutics closely collaborates.
Madam Therapeutics and a team of esteemed Dutch scientists from o.a. Leiden University Medical Center (LUMC) and Amsterdam Medical Center(AMC) have developed a new antibiotic with improved characteristics to fight a broad spectrum of infections. In a recent editorial comment on the new compound on the website of the scientific journal Science, Dr Kim Lewis, a microbiologist at Northeastern University in Boston says that the candidate adds “an important piece … to the puzzle of creating a perfect antibiotic”.
Dr. Remko van Leeuwen, CEO of Madam Therapeutics, comments on this first financing round: “Most of the antibiotic drugs currently in the worldwide clinical pipeline are modifications of existing classes of antibiotics and are only short-term solutions. Antimicrobial resistance is a global health emergency. There is an urgent need for more research and development for antibiotic-resistant infections, otherwise people will die from very simple infections”. Remko van Leeuwen is thrilled to welcome the group of investors to Madam Therapeutics. “The recent investment clearly represents a growing commitment and understanding from the investment community to the development of new antimicrobial agents and reflects the potential of our drug programs.”
Jaap Wieling, a spokesmen of the investor group says: “This funding will allow Madam Therapeutics to take important steps forward with its lead candidate SAAP-148. Where most traditional antibiotics have a slow acting mechanism of action, SAAP-148 kills the bacteria very fast by attacking them directly from the outside, puncturing the cell membrane and make it deflate. Now with the backing of this solid investor base, the seasoned team will be able to explore the full potential of its technology”.
Ron Byron, Partner at Ekoy invest that helped to successfully put together the investment group says: “From my experience in marketing of antibiotics, I am very much aware of growing need for new antibiotics. Unless antibacterial development is re-energised, there is a serious risk that a growing proportion of infections, especially in hospitals, will become effectively untreatable leading to many unnecessary casualties.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-08-27 14:38:102018-08-28 14:44:29Madam Therapeutics closes first financing round to develop novel antibiotics against drug resistant bacteria← #nieuws
Premier Mark Rutte roept “alle ouders nog eens nadrukkelijk op” hun kinderen te laten inenten. De minister-president mengt zich in de discussie over vaccinatie nu steeds meer kinderen niet worden ingeënt.
Rutte “snapt” dat ouders zich zorgen maken om het groeiende aantal niet-ingeënte kinderen. De bescherming die de prik biedt is “niet alleen voor je eigen kinderen, maar dat doe je ook omdat je omgeving kwetsbaar is als dat onvoldoende gebeurt”.
Het kabinet gaat onderzoeken of het mogelijk is kinderdagverblijven de mogelijkheid te bieden niet-ingeënte kinderen te weigeren. Rutte waarschuwt dat “daar wel veel haken en ogen aan zitten”. Maar omdat het kabinet volgens hem de zorgen van ouders deelt, wil het toch onderzoeken of dat verstandig en haalbaar is.
Een nationale vaccinatieplicht gaat Rutte nog te ver.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-08-24 16:36:312018-08-28 16:48:07Kabinet gaat onderzoeken of kinderdagverblijven niet gevaccineerde kinderen mogen weigeren← #nieuws
uniQure has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. AMT-061 has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.
“The initiation of the AMT-061 dose-confirmation study is an important step toward our goal of advancing a potentially life-changing treatment for patients with hemophilia B,” said Robert Gut, M.D., Ph.D., chief medical officer of uniQure. “I am extremely proud of the efforts made by the uniQure team to initiate this study, the objective of which is to demonstrate meaningful increases in FIX activity using the Padua variant and confirm dosing for the HOPE-B pivotal trial initiated this past June. We look forward to completing patient enrollment shortly and providing top-line data before the end of the year.”
“As a one-time administered therapy, AMT-061 has the potential to transform the treatment paradigm for hemophilia B patients,” said Annette von Drygalski, M.D., associate clinical professor at the University of California San Diego and director of its hemophilia and thrombosis treatment center. “By incorporating both AAV5 and the FIX-Padua variant, AMT-061 has the potential to deliver clinically relevant increases in FIX activity with low risk of cellular immune responses, which could expand patient eligibility for treatment with gene therapy. I greatly appreciate the opportunity to participate in the AMT-061 clinical program and view the initiation of the Phase IIb and Phase III studies as important milestones in the development of this potentially important therapy for patients with hemophilia B.”
The Phase IIb dose-confirmation study is an open-label, single-arm, single-dose trial being conducted in the United States. Approximately three patients are expected to receive a single intravenous (IV) infusion of 2×1013 vc/kg and be evaluated for a period of approximately six to eight weeks to assess Factor IX (FIX) activity.
Patient enrollment is also underway in the global Phase III HOPE-B clinical trial to evaluate the safety and efficacy of AMT-061. Approximately 50 adult hemophilia B patients classified as severe and moderately-severe will be enrolled in a six-month observational period during which time they will continue to use their current standard of care to establish a baseline control. After the six-month lead-in period, patients will go onto receive a single intravenous administration of AMT-061. Dosing of patients in the HOPE-B pivotal trial is expected to start early in the first quarter of 2019.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-08-23 16:32:322018-08-28 16:35:54First Patient Treated in uniQure’s in Dose-Confirmation Study of AMT-061← #nieuws
Novo Nordisk has acquired all of the shares of Ziylo, a University of Bristol spin-out company based at Unit DX science incubator in Bristol, UK.
The acquisition gives Novo Nordisk full rights to Ziylo’s glucose binding molecule platform to develop glucose responsive insulins. Ziylo’s glucose binding molecules are synthetic molecules that were designed by Professor Anthony Davis at the University of Bristol. These stable, synthetic molecules exhibit an unprecedented selectivity to glucose in complex environments such as blood.
“We believe the glucose binding molecules discovered by the Ziylo team together with Novo Nordisk world-class insulin capabilities have the potential to lead to the development of glucose responsive insulins which we hope can remove the risk of hypoglycaemia and ensure optimal glucose control for people with diabetes,” said Marcus Schindler, senior vice president, Global Drug Discovery, Novo Nordisk.
Novo Nordisk acquires all shares in Ziylo for an upfront payment and earn-outs with contingent milestone payments. Total payments under the agreement could ultimately exceed 800 million dollars upon the achievement of certain development, regulatory and sales milestones by Novo Nordisk.
A key strategic area
The development of glucose responsive insulins is a key strategic area for Novo Nordisk in its effort to develop this next generation of insulin which would lead to a safer and more effective insulin therapy, it reports. A glucose responsive insulin would help eliminate the risk of hypoglycaemia, which is the main risk associated with insulin therapy and one of the main barriers for achieving optimal glucose control. Thus, a glucose responsive insulin could also lead to better metabolic control and thus overall reduce the burden of diabetes for people living with the disease.
Carbometrics
Prior to closing of the acquisition, certain research activities have been spun out of Ziylo to a new company, Carbometrics. Carbometrics has entered into a research collaboration with Novo Nordisk to assist with ongoing optimisation of glucose binding molecules for use in glucose responsive insulins. Carbometrics has licenced rights to develop non-therapeutic applications of glucose binding molecules, with a focus on developing continuous glucose monitoring applications.
Aduro Biotech announced that the United States Patent and Trademark Office has issued a new composition of matter patent related to altered APRIL-binding antibodies, further enhancing the company’s B-select intellectual property portfolio. Specifically, the granted claims cover BION-1301, Aduro’s first-in-class anti-APRIL antibody being evaluated in a Phase 1/2 dose escalation trial for the treatment of multiple myeloma.
APRIL
APRIL (A PRoliferation-Inducing Ligand) is a member of the tumor necrosis factor (TNF) superfamily and is primarily secreted by bone marrow and/or myeloid cells. APRIL is overproduced in patients with multiple myeloma and binds to BCMA (B cell maturation antigen) and TACI (Transmembrane Activator and CAML Interactor) to stimulate a wide variety of responses that promote multiple myeloma growth and survival and suppress the immune system so that the tumor cells are protected and sustained in the bone marrow.
BION-1301
Aduro is currently evaluating BION-1301, its most advanced proprietary B-select monoclonal antibody, as a novel therapy for multiple myeloma. Despite new treatments recently approved in multiple myeloma, this disease remains incurable as patients relapse, or become resistant to, currently-available therapies. In preclinical studies, Aduro has established that A PRoliferation-Inducing Ligand (APRIL) plays a crucial part in the protective bone marrow tumor microenvironment. In these studies, APRIL, through the B cell maturation antigen (BCMA), was shown to be critically involved in the survival, proliferation and chemoresistance of multiple myeloma, and upregulates mechanisms of immunoresistance, including PD-L1 upregulation. BION-1301, a humanized antibody that blocks APRIL from binding to its receptors, has been shown in preclinical studies to halt tumor growth and overcome drug resistance. In addition, BION-1301 also demonstrated the ability to inhibit immune suppressive effects of regulatory T cells via TACI but not BCMA in multiple myeloma blood and bone marrow. BION-1301 is currently being evaluated in a Phase 1/2 clinical study.
“Blocking APRIL represents a unique approach to treating patients with multiple myeloma and we believe BION-1301 has potential to treat a myriad of oncology indications as well as other autoimmune and inflammatory diseases,” commented Stephen Isaacs, chairman and chief executive officer of Aduro Biotech. “Ensuring a robust intellectual property position around BION-1301 is inherent to advancing the program and the claims granted in this particular patent exemplify the novel science behind this exciting program.”
U.S. patent 9,969,808 adds to previously issued U.S. and international counterpart patents and patent applications that form Aduro’s APRIL patent portfolio. Claims were granted on the basis that BION-1301 enables full blockade of APRIL binding to both its receptors BCMA and TACI. Preclinical studies have demonstrated that blocking APRIL with BION-1301 not only inhibited proliferation and survival of multiple myeloma cells but also alleviated drug resistance and immune suppression, leading to enhanced myeloma cell killing.1 Further preclinical research by Aduro and its collaborators indicate that blocking APRIL further enhances anti-BMCA cytotoxic cell killing and that prevention of APRIL binding to TACI may also be a potentially important mechanism for BION-1301 to inhibit the function of regulatory T cells.2
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-08-22 13:48:162018-08-28 13:57:56Aduro Biotech Granted Composition of Matter Patent for Novel Human APRIL Binding Antibodies← #nieuws
Goed nieuws: Minister Bruins van het ministerie van VWS heeft besloten om combinatiebehandeling met Darzalex® (daratumumab) van patiënten met multipel myeloom, oftewel de ziekte van Kahler, per 1 september 2018 op te nemen in het basispakket van de zorgverzekering. Een besluit met grote waarde voor patiënten die lijden aan deze ernstige ziekte.
Geneesmiddelenontwikkelaar Janssen en VWS kwamen middels een sluisprocedure tot een akkoord. Michel van Agthoven, directielid Janssen, licht toe: “Wij waarderen het zorgvuldige en constructieve proces waarin de onderhandelingen met het ministerie van VWS hebben plaatsgevonden. We zetten ons in voor een effectieve toepassing in de praktijk om de meeste gezondheidswinst te behalen, op een kosteneffectieve manier. Alleen dan kunnen we de zorg betaalbaar houden.”
Het feit dat Darzalex® Nederlandse roots heeft – het geneesmiddel komt oorspronkelijk uit de pijplijn van Genmab – geeft het mooie nieuws extra glans. Een mooi succes van eigen bodem!
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-08-21 17:16:402018-08-21 17:19:46Geneesmiddel tegen de ziekte van Kahler in het basispakket← #nieuws
Tijdens het internationale congres voor moleculaire plantenbiologie IPMB afgelopen maand in Montpellier, lanceerde een groep wetenschappers een petitie die oproept tot een herziening van de uitspraak van het Europees Hof van Justitie over plantenveredeling. De initiatiefnemers vinden dat de uitspraak volledig voorbij gaat aan wetenschappelijke inzichten en aantoonbare feitelijke onjuistheden bevat. De gevolgen zijn groot, want de uitspraak leidt tot een hoge drempel voor het gebruik van moderne plantenveredelingsmethoden. Dat staat het ontwikkelen van gewassen en producten die bijdragen aan duurzaam en gezondheid voedsel in de weg. Bijna 4.000 wetenschappers en burgers ondertekenden de petitie tot nu toe. En hoewel de kans klein is dat hij leidt tot een herziening, geeft de petitie een kans om de grote onvrede over de uitspraak zichtbaar te maken. Ook je steun geven aan dit initiatief? Bekijk en onderteken de petitie hier!
“De code van het brood”, zo kopte de NRC afgelopen vrijdag ter ere van het compleet in kaart gebrachte tarwe genoom. Een groot internationaal consortium van wetenschappers, waaronder Keygene, heeft dertien jaar aan de ontrafeling van het complexe tarwecomplex gewerkt. Met de opgedane kennis kunnen plantenveredelaars veel sneller en preciezer nieuwe tarwegewassen ontwikkelen. Dit biedt hen de mogelijkheid om snel in te spelen op het veranderende klimaat en tegemoet te komen aan de wensen van de consument. Er zijn bijvoorbeeld genen aan het licht gekomen die verband houden met gluten-intolerantie. De moderne veredelingsmethoden bieden de plantenveredelaars de mogelijkheid om deze genen aan te passen of te vervangen. In Europa zullen we helaas nog wel even op zo’n glutenvrij tarwebrood moeten wachten. Het Europese Hof van justitie heeft namelijk recent beslist dat de moderne veredelingsmethoden onder de strenge wetgeving voor genetische modificatie vallen. Voordat de boeren de glutenvrije tarwevarianten op de Europese akkers mogen telen, moeten ze dus eerst door een streng, langdurig, kostbaar en onzeker toelatingstraject. Een gemiste kans, volgens HollandBIO.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-08-21 17:05:212018-08-21 17:05:21De code van ons brood← #nieuws
Het Nederlandse biotechbedrijf ProQR Therapeutics geeft geen prioriteit meer aan het werken aan een middel voor taaislijmziekte en investeert nu in medicijnen tegen zeldzame oog- en huidziektes. Dat heeft topman Daniel de Boer gezegd in een interview met het Financieele Dagblad.
,,Ongeveer een jaar geleden hebben we ons afgevraagd waar we ons geld aan moesten besteden”, verklaart De Boer. ,,Dat was niet langer taaislijmziekte. Op dat gebied hebben andere bedrijven grote vooruitgang geboekt.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-08-19 17:19:552018-08-21 18:12:18ProQR geeft geen prioriteit meer aan middel voor taaislijmziekte
Geneesmiddelen: van kiloknaller naar hofleverancier
Hollandbio, InnovatieklimaatOp uitnodiging van het ministerie van VWS reageerde HollandBIO vorige week op de voorgenomen wijziging van de Wet Geneesmiddelenprijzen, de WGP. Het vervangen van referentieland Duitsland door Noorwegen realiseert jaarlijks een besparing van 239 miljoen euro, zo becijfert de Stichting Farmaceutische Kengetallen. De maatregel is het zoveelste lapmiddel om de zorgkosten te beheersen door minimalisatie van de geneesmiddelenuitgaven. Geneesmiddelen fungeren zo als kiloknallers van de zorg, in plaats van als hofleveranciers voor volksgezondheid. In onze reactie breken we een lans voor duurzame oplossingen waarbij gzondheidswinst, innovatie en betaalbaarheid hand in hand gaan. Je leest onze brief hier.
Madam Therapeutics closes first financing round to develop novel antibiotics against drug resistant bacteria
InnovatieklimaatMadam Therapeutics closed its first financing round of 1,1M EUR. The biotech company develops a novel class of antibiotics inspired by the defense mechanisms of the human body. The new funding will be used to continue and accelerate the clinical development of Madam Therapeutics’ lead candidate SAAP-148, by executing a first-in-men proof of concept study starting early next year and preparation of the following clinical phase II trial. The investment comes from a network of European business angels facilitated by Ekoy Investment Partners, De Investeerders Club and Investormatch.
Lead candidate: SAAP-148
When a small subset of bacteria survives antibiotic treatment e.g. by mutations, an infection can get out of control fast. As these resilient microbes thrive, they can group together on a surface—like a wound or a medical device—and encase themselves in a slimy protective layer known as a biofilm. Such biofilm infections “are the really nasty things for patients,” says immunologist Peter Nibbering at Leiden University Medical Center in the Netherlands with whom Madam Therapeutics closely collaborates.
Madam Therapeutics and a team of esteemed Dutch scientists from o.a. Leiden University Medical Center (LUMC) and Amsterdam Medical Center(AMC) have developed a new antibiotic with improved characteristics to fight a broad spectrum of infections. In a recent editorial comment on the new compound on the website of the scientific journal Science, Dr Kim Lewis, a microbiologist at Northeastern University in Boston says that the candidate adds “an important piece … to the puzzle of creating a perfect antibiotic”.
Dr. Remko van Leeuwen, CEO of Madam Therapeutics, comments on this first financing round: “Most of the antibiotic drugs currently in the worldwide clinical pipeline are modifications of existing classes of antibiotics and are only short-term solutions. Antimicrobial resistance is a global health emergency. There is an urgent need for more research and development for antibiotic-resistant infections, otherwise people will die from very simple infections”. Remko van Leeuwen is thrilled to welcome the group of investors to Madam Therapeutics. “The recent investment clearly represents a growing commitment and understanding from the investment community to the development of new antimicrobial agents and reflects the potential of our drug programs.”
Jaap Wieling, a spokesmen of the investor group says: “This funding will allow Madam Therapeutics to take important steps forward with its lead candidate SAAP-148. Where most traditional antibiotics have a slow acting mechanism of action, SAAP-148 kills the bacteria very fast by attacking them directly from the outside, puncturing the cell membrane and make it deflate. Now with the backing of this solid investor base, the seasoned team will be able to explore the full potential of its technology”.
Ron Byron, Partner at Ekoy invest that helped to successfully put together the investment group says: “From my experience in marketing of antibiotics, I am very much aware of growing need for new antibiotics. Unless antibacterial development is re-energised, there is a serious risk that a growing proportion of infections, especially in hospitals, will become effectively untreatable leading to many unnecessary casualties.”
Source: Madam Therapeutics
Kabinet gaat onderzoeken of kinderdagverblijven niet gevaccineerde kinderen mogen weigeren
GezondheidPremier Mark Rutte roept “alle ouders nog eens nadrukkelijk op” hun kinderen te laten inenten. De minister-president mengt zich in de discussie over vaccinatie nu steeds meer kinderen niet worden ingeënt.
Rutte “snapt” dat ouders zich zorgen maken om het groeiende aantal niet-ingeënte kinderen. De bescherming die de prik biedt is “niet alleen voor je eigen kinderen, maar dat doe je ook omdat je omgeving kwetsbaar is als dat onvoldoende gebeurt”.
Het kabinet gaat onderzoeken of het mogelijk is kinderdagverblijven de mogelijkheid te bieden niet-ingeënte kinderen te weigeren. Rutte waarschuwt dat “daar wel veel haken en ogen aan zitten”. Maar omdat het kabinet volgens hem de zorgen van ouders deelt, wil het toch onderzoeken of dat verstandig en haalbaar is.
Een nationale vaccinatieplicht gaat Rutte nog te ver.
Bron: Skipr
First Patient Treated in uniQure’s in Dose-Confirmation Study of AMT-061
GezondheiduniQure has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. AMT-061 has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.
“The initiation of the AMT-061 dose-confirmation study is an important step toward our goal of advancing a potentially life-changing treatment for patients with hemophilia B,” said Robert Gut, M.D., Ph.D., chief medical officer of uniQure. “I am extremely proud of the efforts made by the uniQure team to initiate this study, the objective of which is to demonstrate meaningful increases in FIX activity using the Padua variant and confirm dosing for the HOPE-B pivotal trial initiated this past June. We look forward to completing patient enrollment shortly and providing top-line data before the end of the year.”
“As a one-time administered therapy, AMT-061 has the potential to transform the treatment paradigm for hemophilia B patients,” said Annette von Drygalski, M.D., associate clinical professor at the University of California San Diego and director of its hemophilia and thrombosis treatment center. “By incorporating both AAV5 and the FIX-Padua variant, AMT-061 has the potential to deliver clinically relevant increases in FIX activity with low risk of cellular immune responses, which could expand patient eligibility for treatment with gene therapy. I greatly appreciate the opportunity to participate in the AMT-061 clinical program and view the initiation of the Phase IIb and Phase III studies as important milestones in the development of this potentially important therapy for patients with hemophilia B.”
The Phase IIb dose-confirmation study is an open-label, single-arm, single-dose trial being conducted in the United States. Approximately three patients are expected to receive a single intravenous (IV) infusion of 2×1013 vc/kg and be evaluated for a period of approximately six to eight weeks to assess Factor IX (FIX) activity.
Patient enrollment is also underway in the global Phase III HOPE-B clinical trial to evaluate the safety and efficacy of AMT-061. Approximately 50 adult hemophilia B patients classified as severe and moderately-severe will be enrolled in a six-month observational period during which time they will continue to use their current standard of care to establish a baseline control. After the six-month lead-in period, patients will go onto receive a single intravenous administration of AMT-061. Dosing of patients in the HOPE-B pivotal trial is expected to start early in the first quarter of 2019.
Source: uniQure
Novo Nordisk acquires Ziylo for 800 million USD
Gezondheid, InnovatieklimaatNovo Nordisk has acquired all of the shares of Ziylo, a University of Bristol spin-out company based at Unit DX science incubator in Bristol, UK.
The acquisition gives Novo Nordisk full rights to Ziylo’s glucose binding molecule platform to develop glucose responsive insulins. Ziylo’s glucose binding molecules are synthetic molecules that were designed by Professor Anthony Davis at the University of Bristol. These stable, synthetic molecules exhibit an unprecedented selectivity to glucose in complex environments such as blood.
“We believe the glucose binding molecules discovered by the Ziylo team together with Novo Nordisk world-class insulin capabilities have the potential to lead to the development of glucose responsive insulins which we hope can remove the risk of hypoglycaemia and ensure optimal glucose control for people with diabetes,” said Marcus Schindler, senior vice president, Global Drug Discovery, Novo Nordisk.
Novo Nordisk acquires all shares in Ziylo for an upfront payment and earn-outs with contingent milestone payments. Total payments under the agreement could ultimately exceed 800 million dollars upon the achievement of certain development, regulatory and sales milestones by Novo Nordisk.
A key strategic area
The development of glucose responsive insulins is a key strategic area for Novo Nordisk in its effort to develop this next generation of insulin which would lead to a safer and more effective insulin therapy, it reports. A glucose responsive insulin would help eliminate the risk of hypoglycaemia, which is the main risk associated with insulin therapy and one of the main barriers for achieving optimal glucose control. Thus, a glucose responsive insulin could also lead to better metabolic control and thus overall reduce the burden of diabetes for people living with the disease.
Carbometrics
Prior to closing of the acquisition, certain research activities have been spun out of Ziylo to a new company, Carbometrics. Carbometrics has entered into a research collaboration with Novo Nordisk to assist with ongoing optimisation of glucose binding molecules for use in glucose responsive insulins. Carbometrics has licenced rights to develop non-therapeutic applications of glucose binding molecules, with a focus on developing continuous glucose monitoring applications.
Source: Nordic Life Science News
Aduro Biotech Granted Composition of Matter Patent for Novel Human APRIL Binding Antibodies
InnovatieklimaatAduro Biotech announced that the United States Patent and Trademark Office has issued a new composition of matter patent related to altered APRIL-binding antibodies, further enhancing the company’s B-select intellectual property portfolio. Specifically, the granted claims cover BION-1301, Aduro’s first-in-class anti-APRIL antibody being evaluated in a Phase 1/2 dose escalation trial for the treatment of multiple myeloma.
APRIL
APRIL (A PRoliferation-Inducing Ligand) is a member of the tumor necrosis factor (TNF) superfamily and is primarily secreted by bone marrow and/or myeloid cells. APRIL is overproduced in patients with multiple myeloma and binds to BCMA (B cell maturation antigen) and TACI (Transmembrane Activator and CAML Interactor) to stimulate a wide variety of responses that promote multiple myeloma growth and survival and suppress the immune system so that the tumor cells are protected and sustained in the bone marrow.
BION-1301
Aduro is currently evaluating BION-1301, its most advanced proprietary B-select monoclonal antibody, as a novel therapy for multiple myeloma. Despite new treatments recently approved in multiple myeloma, this disease remains incurable as patients relapse, or become resistant to, currently-available therapies. In preclinical studies, Aduro has established that A PRoliferation-Inducing Ligand (APRIL) plays a crucial part in the protective bone marrow tumor microenvironment. In these studies, APRIL, through the B cell maturation antigen (BCMA), was shown to be critically involved in the survival, proliferation and chemoresistance of multiple myeloma, and upregulates mechanisms of immunoresistance, including PD-L1 upregulation. BION-1301, a humanized antibody that blocks APRIL from binding to its receptors, has been shown in preclinical studies to halt tumor growth and overcome drug resistance. In addition, BION-1301 also demonstrated the ability to inhibit immune suppressive effects of regulatory T cells via TACI but not BCMA in multiple myeloma blood and bone marrow. BION-1301 is currently being evaluated in a Phase 1/2 clinical study.
“Blocking APRIL represents a unique approach to treating patients with multiple myeloma and we believe BION-1301 has potential to treat a myriad of oncology indications as well as other autoimmune and inflammatory diseases,” commented Stephen Isaacs, chairman and chief executive officer of Aduro Biotech. “Ensuring a robust intellectual property position around BION-1301 is inherent to advancing the program and the claims granted in this particular patent exemplify the novel science behind this exciting program.”
U.S. patent 9,969,808 adds to previously issued U.S. and international counterpart patents and patent applications that form Aduro’s APRIL patent portfolio. Claims were granted on the basis that BION-1301 enables full blockade of APRIL binding to both its receptors BCMA and TACI. Preclinical studies have demonstrated that blocking APRIL with BION-1301 not only inhibited proliferation and survival of multiple myeloma cells but also alleviated drug resistance and immune suppression, leading to enhanced myeloma cell killing.1 Further preclinical research by Aduro and its collaborators indicate that blocking APRIL further enhances anti-BMCA cytotoxic cell killing and that prevention of APRIL binding to TACI may also be a potentially important mechanism for BION-1301 to inhibit the function of regulatory T cells.2
Geneesmiddel tegen de ziekte van Kahler in het basispakket
GezondheidGoed nieuws: Minister Bruins van het ministerie van VWS heeft besloten om combinatiebehandeling met Darzalex® (daratumumab) van patiënten met multipel myeloom, oftewel de ziekte van Kahler, per 1 september 2018 op te nemen in het basispakket van de zorgverzekering. Een besluit met grote waarde voor patiënten die lijden aan deze ernstige ziekte.
Geneesmiddelenontwikkelaar Janssen en VWS kwamen middels een sluisprocedure tot een akkoord. Michel van Agthoven, directielid Janssen, licht toe: “Wij waarderen het zorgvuldige en constructieve proces waarin de onderhandelingen met het ministerie van VWS hebben plaatsgevonden. We zetten ons in voor een effectieve toepassing in de praktijk om de meeste gezondheidswinst te behalen, op een kosteneffectieve manier. Alleen dan kunnen we de zorg betaalbaar houden.”
Het feit dat Darzalex® Nederlandse roots heeft – het geneesmiddel komt oorspronkelijk uit de pijplijn van Genmab – geeft het mooie nieuws extra glans. Een mooi succes van eigen bodem!
Lees meer:
– Persbericht Janssen
– Persbericht VWS
Tekenen! Petitie voor herziening uitspraak Europees Hof over plantenveredeling
Hollandbio, Voedsel en materialen, UitgelichtTijdens het internationale congres voor moleculaire plantenbiologie IPMB afgelopen maand in Montpellier, lanceerde een groep wetenschappers een petitie die oproept tot een herziening van de uitspraak van het Europees Hof van Justitie over plantenveredeling. De initiatiefnemers vinden dat de uitspraak volledig voorbij gaat aan wetenschappelijke inzichten en aantoonbare feitelijke onjuistheden bevat. De gevolgen zijn groot, want de uitspraak leidt tot een hoge drempel voor het gebruik van moderne plantenveredelingsmethoden. Dat staat het ontwikkelen van gewassen en producten die bijdragen aan duurzaam en gezondheid voedsel in de weg. Bijna 4.000 wetenschappers en burgers ondertekenden de petitie tot nu toe. En hoewel de kans klein is dat hij leidt tot een herziening, geeft de petitie een kans om de grote onvrede over de uitspraak zichtbaar te maken. Ook je steun geven aan dit initiatief? Bekijk en onderteken de petitie hier!
De code van ons brood
Hollandbio, Voedsel en materialen“De code van het brood”, zo kopte de NRC afgelopen vrijdag ter ere van het compleet in kaart gebrachte tarwe genoom. Een groot internationaal consortium van wetenschappers, waaronder Keygene, heeft dertien jaar aan de ontrafeling van het complexe tarwecomplex gewerkt. Met de opgedane kennis kunnen plantenveredelaars veel sneller en preciezer nieuwe tarwegewassen ontwikkelen. Dit biedt hen de mogelijkheid om snel in te spelen op het veranderende klimaat en tegemoet te komen aan de wensen van de consument. Er zijn bijvoorbeeld genen aan het licht gekomen die verband houden met gluten-intolerantie. De moderne veredelingsmethoden bieden de plantenveredelaars de mogelijkheid om deze genen aan te passen of te vervangen. In Europa zullen we helaas nog wel even op zo’n glutenvrij tarwebrood moeten wachten. Het Europese Hof van justitie heeft namelijk recent beslist dat de moderne veredelingsmethoden onder de strenge wetgeving voor genetische modificatie vallen. Voordat de boeren de glutenvrije tarwevarianten op de Europese akkers mogen telen, moeten ze dus eerst door een streng, langdurig, kostbaar en onzeker toelatingstraject. Een gemiste kans, volgens HollandBIO.
Lees ook het artikel van het NRC.
ProQR geeft geen prioriteit meer aan middel voor taaislijmziekte
GezondheidHet Nederlandse biotechbedrijf ProQR Therapeutics geeft geen prioriteit meer aan het werken aan een middel voor taaislijmziekte en investeert nu in medicijnen tegen zeldzame oog- en huidziektes. Dat heeft topman Daniel de Boer gezegd in een interview met het Financieele Dagblad.
,,Ongeveer een jaar geleden hebben we ons afgevraagd waar we ons geld aan moesten besteden”, verklaart De Boer. ,,Dat was niet langer taaislijmziekte. Op dat gebied hebben andere bedrijven grote vooruitgang geboekt.”
Bronnen: iex en FD