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Gezondheidsraad adviseert over meningokokken en maternale kinkhoest

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Kort voor kerst publiceerde de Gezondheidsraad (GR) haar adviezen rond meningokokken- en kinkhoestvaccinatie. 2018 was een markant jaar voor het vaccinatiedossier. Een jaar waarin de vaccinatiegraad opnieuw daalde, de meningokokken en de griep om zich heen sloegen en de maatschappelijke discussie over het belang van vaccineren vol oplaaide. HollandBIO is blij met de grote aandacht voor vaccinatie. We maakten ons dag in, dag uit hard om de preventieve kracht van vaccinatie voor onze samenleving optimaal te benutten. Ook in 2019 zetten wij ons vol in om de met vaccins te boeken gezondheidswinst te verzilveren.

Meningokokken C en W vaccinatie in het rijksvaccinatieprogramma

In haar advies pleit de GR voor een structurele verankering van het meningokokken C en W vaccin in ons rijksvaccinatieprogramma (RVP). Zij bevestigt hiermee de besluiten van het outbreak management team en het bestuurlijk afstemmingsoverleg in reactie op de meningokokken W uitbraak eerder in 2017 en 2018. Voor collectieve invoer van het meningokokken B vaccin vindt de GR de bewijslast rond de vaccineffectiviteit nog te summier. Zij raadt aan dit dossier over drie jaar opnieuw te beoordelen.

Eén prik minder voor baby’s van gevaccineerde moeders

Voor kinkhoest adviseert de GR een wijziging in het prikkenschema. Bij een succesvolle in- en uitvoer van de kinkhoestvaccinatie voor zwangeren, kunnen de meeste zuigelingen met een prik minder af, aldus de GR.

HollandBIO op de bres voor de beoordeling en inzet van nieuwe vaccins

HollandBIO kijkt uit naar de beleidsreactie op de adviezen van de GR. Staatssecretaris Blokhuis is aan zet. Wat HollandBIO betreft is het hele systeem van registratie tot beoordeling en inzet van nieuwe vaccins aan een daadkrachtige update toe. Ook in 2019 zetten wij ons daarom vol in om het momentum rond vaccinatie te verzilveren. Een breed gedragen position paper die oproept tot een gezamenlijke ambitie op het vlak van vaccins is een mooie eerste stap.

Ben je benieuwd naar de adviezen van de GR? Lees ze hier:

https://www.gezondheidsraad.nl/documenten/adviezen/2018/12/19/vaccinatie-tegen-meningokokken

https://www.gezondheidsraad.nl/documenten/adviezen/2018/12/18/maternale-kinkhoestvaccinatie

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Galapagos initiates NOVESA Phase 2a trial in patients with systemic sclerosis

Galapagos expands its clinical study program with GLPG1690 in systemic sclerosis, following the recent start of the ISABELA Phase 3 program with ‘1690 in IPF.

NOVESA is a double-blind, placebo-controlled Phase 2a trial evaluating the efficacy, safety and PK/PD of ‘1690 in patients with systemic sclerosis (SSc, or scleroderma). NOVESA is planned to recruit 30 patients with diffuse cutaneous SSc, an autoimmune disease involving multiorgan fibrosis, which has one of the highest mortality rates among rheumatic diseases1 . One of the most visible manifestions is hardening of the skin. In diffuse cutaneous SSc, skin thickening affects several body areas, and patients have a higher risk of developing fibrosis of various internal organs, such as the lung. Currently, there are no approved drugs for this disease. SSc affects approximately 90,000 patients in the US and Europe, with a predominance of female patients (75%).

The primary endpoint of NOVESA is the modified Rodnan skin score (mRSS) at 24 weeks. mRRS measures the skin thickness as a surrogate measure of disease severity and mortality, with an increase in thickness associated with involvement of internal organs and increased mortality2 . Secondary objectives and exploratory endpoints include FVC3 , HRCT4 , quality of life as measured by QoL-Q (SHAQ)5 , and CRISS6 , a SSc disease composite score.

“In addition to our Phase 3 program in IPF7 , we are excited to broaden our development program with ‘1690 to a second indication,” said Dr. Walid Abi-Saab, Chief Medical Officer at Galapagos. “Moreover, SSc is particularly interesting, as this disease straddles our expertise in autoimmune diseases as well as in fibrosis. Thanks to the broad MoA of ‘1690, which is both anti-inflammatory and anti-fibrotic, this compound has the potential to address the important unmet medical need in SSc.”

Source: Galapagos

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Fibrocor and Galapagos sign partnership in fibrosis

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Fibrocor Therapeutics, a privately held Canadian company, and Galapagos announced a global partnership focused on a novel target for idiopathic pulmonary fibrosis (IPF) and other indications.

Fibrocor specializes in the development of tissue-specific therapeutics to treat the underlying cause of fibrotic diseases of the kidney and other organs. The collaboration announced today concerns a small molecule inhibitor program, currently in the lead optimization stage of development for the treatment of fibrotic diseases of the lung and other organs, the target of which is undisclosed. In exchange for global commercialization rights to Galapagos, Fibrocor will receive an upfront payment, and potentially is eligible for further milestone and royalty payments. Galapagos will be responsible for all further development of the program.

“This collaboration validates the fibrosis drug development expertise of Fibrocor,” says Mark A. Steedman, President and CEO of Fibrocor. “I take my hat off to Dr. Richard Gilbert and the scientific team, including Evotec GmbH, our CRO1 partner, for establishing a compelling data package that ultimately attracted Galapagos, a world-renowned biotech company with a key franchise in fibrosis. We feel this is the beginning of a great relationship and look forward to working with Galapagos to the benefit of fibrosis sufferers everywhere.”

“The collaboration with Fibrocor announced today is an excellent strategic fit for Galapagos, as we continue to expand our franchise in IPF, and more broadly, in fibrosis,” says Dr. Piet Wigerinck, Chief Scientific Officer at Galapagos. “We are enthusiastic about the drug discovery approach at Fibrocor, and look forward to collaborating with the team to address the large unmet medical need in fibrosis.”

Source: Galapagos

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Amicus Therapeutics Announces Phase 1/2 Study of Gene Therapy for CLN3 Batten Disease

Amicus Therapeutics, a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, has initiated a Phase 1/2 clinical study to evaluate the safety and efficacy of a single intrathecal administration of adeno-associated virus serotype 9 AAV9-CLN3 (AAV9-CLN3) gene therapy in children with CLN3 Batten disease. Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. The initial patient completed a one-month observation period following dosing with no serious adverse events reported to date.

This first in human study of an investigational gene therapy in CLN3 Batten disease, a life-threatening genetic neurologic disorder that typically begins in early childhood and results in premature death, is currently being conducted at Nationwide Children’s Hospital (Columbus, Ohio). CLN3 is the most prevalent of the Batten’s disorders affecting an estimated 5,000+ patients.

“We are pleased to announce that the first child has been dosed in the Phase 1/2 study for CLN3 Batten disease,” said Jay Barth, MD, Chief Medical Officer at Amicus Therapeutics, Inc.“With this pioneering study in CLN3 Batten disease, a severe and devastating neurodegenerative disorder with no approved treatments, we hope that a single intrathecal administration of our gene therapy has the potential to treat the underlying cause of disease and provide a durable and meaningful treatment benefit. This initial participant in our CLN3 Batten disease study, together with the participants in our ongoing clinical study in CLN6 Batten disease, further supports the encouraging safety profile for the intrathecal AAV delivery platform. Advancing this program is another important step forward to potentially improve the lives of thousands of children living with Batten disease and other neurologic lysosomal storage disorders.”

Emily C. De Los Reyes, MD, Principal Investigator at Nationwide Children’s stated, “The initiation of this clinical study in CLN3 Batten disease is an important initial milestone. The proof-of-concept demonstrated in preclinical studies and validation of the intrathecal AAV platform across multiple indications here at Nationwide Children’s Hospital provides promise for the delivery approach in this study. We look forward to enrolling additional participants in the study and comparing the findings to the existing natural history data in CLN3 Batten disease.”

The Phase 1/2 study is enrolling children aged 3 to 10 years with a confirmed diagnosis of CLN3 Batten disease in two sequential intrathecal dose groups: a one-time low-dose of AAV9-CLN3 (Group 1) and a one-time high-dose of AAV9-CLN3 (Group 2) after evaluation of Group 1 participants. Both groups will participate in the current study for a period of three years. Amicus plans to present clinical data from this study, including interim data, at future scientific congresses and other relevant venues. The primary outcome measures are safety and efficacy as determined using the physical disability subscale of the Unified Batten Disease Rating Scale (UBDRS) in CLN3 Batten disease. More information is available at www.clinicaltrials.gov: NCT03770572.

CLN3 Batten disease, the most common form of NCL results from a mutation in the CLN3 gene which primarily affects the nervous system. Children with this condition develop vision impairment, intellectual disability, progressive loss of motor function, speech difficulties, and seizures which worsen over time.

Source: Amicus Therapeutics

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ProQR Receives Fast Track Designation from FDA for QR-421a for Usher Syndrome Type 2

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ProQR has received a Fast Track designation from the Food and Drug Administration (FDA) for QR-421a. QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of the vision loss associated with Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.

Fast Track designation is granted by FDA to drugs that are under development for serious conditions and have the potential to fulfill an unmet medical need. It was established with the intention to bring promising drugs to patients sooner by facilitating the development with more frequent FDA interactions and expediting the review process.

“We are very pleased with the Fast Track designation the FDA granted us for QR-421a. Patients with Usher syndrome, the leading cause of combined deafness and blindness, currently have no available therapies for their vision loss and this designation emphasizes the high unmet need in this disease,” said Daniel de Boer, Chief Executive Officer of ProQR. “We are also looking forward to begin enrollment in the Phase 1/2 STELLAR clinical trial in the coming months with preliminary data expected in mid-2019.”

Source: ProQR

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Xenikos will conduct Phase 3 trial to test T-Guard(R) in acute graft-versus-host disease

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Xenikos has partnered with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) to conduct a U.S.-based Phase 3 registration trial to test the efficacy of T-Guard in treating steroid-refractory acute graft-versus-host disease (SR-aGVHD) in patients following an allogeneic stem cell transplant. Funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute, both part of the National Institues of Health, the BMT CTN includes leading transplant centers in the United States (U.S.) and will provide a total of USD 1.37 million in funding for the T-Guard trial.

“At Xenikos, we see BMT CTN’s access to major transplant centers in the U.S., as well as their established track record for successfully recruiting patients with acute GVHD, and vast knowledge and expertise in this field, as aiding in the development of innovative new immunotherapies for improving patient outcomes” said Dr. Ypke van Oosterhout, CEO of Xenikos. “We are therefore confident that our partnership with BMT CTN will help bring T-Guard to transplant patients as quickly as possible.”

“Our network often conducts large, multi-institutional clinical trials in order to test new treatments designed to improve outcomes associated with hematopoietic stem cell transplantation or HSCT,” said Dr. Mehdi Hamadani of the BMT CTN. “Acute GVHD is a life-threatening complication of HSCT and better treatments are needed for our patients. The preliminary data obtained with T-Guard is quite promising, and we believe that the Phase 3 trial is the next logical step.”

The U.S.-based Phase 3 trial will be a multi-center study involving patients who have received an allogeneic stem cell transplant for a myeloid or lymphoid malignancy and subsequently developed SR-aGVHD. Importantly, the Phase 3 trial design is based on input received from the U.S. Food and Drug Administration at the End-of-Phase 2 meeting. Xenikos plans to file an Investigational New Drug application in 2019.

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Enpicom sets a new standard for B cell receptor repertoire analysis

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The Dutch bioinformatics software engineering company ENPICOM announced that they have finalized development and released an expanded version of their ImmunoGenomiX (IGX) platform to support discovery and development of novel immunotherapies, patient stratification for, and treatment monitoring of immunotherapies. IGX now also accepts B cell receptor (BCR) in addition to T cell receptor (TCR) sequencing data. Head-to-head benchmarking studies versus world’s most popular tool, pRESTO, show superiority on several levels.

The improvements in high-throughput sequencing (HTS) technology provide unprecedented opportunities to explore the enormous diversity of the immune repertoire by deep sequencing T cell receptors and B cell receptors. However, an efficient, integrated, easy-to-use and accurate analytical tool required to process the huge amounts of immunosequencing data has so far been lacking.

With the IGX platform, ENPICOM is on a mission to provide the immunotherapy community with a best-in-class solution to analyze T cell and B cell receptor (TCR/BCR) immunosequencing data. The base module of the IGX platform to perform T cell receptor repertoire analysis, IGX Explore, was launched in August this year. The current functional expansion of IGX Explore adds support for the identification and quantification of B cell receptors (BCRs) from HTS data. Dr. Nicola Bonzanni, ENPICOM’s Chief Scientific Officer, commented: “As BCR analysis harbors additional complexities, such as immunoglobulin (Ig) (i.e. antibody) gene somatic hypermutations, the development of a state-of-the-art solution for Ig/BCR sequence analysis offered more extensive challenges for our bioinformatics and software engineering teams. It is with great pleasure that we announce the timely release of the next version of IGX Explore and that our benchmarking studies showed even better performance than expected.”

Dr. Bonzanni continues: “We benchmarked the performance of IGX Explore for BCRs using both synthetic and experimental datasets. IGX Explore shows superior performance for critical metrics such as correctly identified CDR3 sequences and V/J gene identification. Specifically, we compared IGX Explore with pRESTO (Vander Heiden, et al. 2014), the world’s most popular BCR sequencing analysis tool. On synthetic data, we show that IGX Explore achieves consistent and highly precise performance across all commonly used read lengths, and outperforms pRESTO at short reads lengths in particular, as visualized in the graphs below.

Furthermore, using an independently published experimental dataset (Meng W, et al. 2017), we showed that IGX Explore is able to recapitulate the reference immune repertoire composition with high accuracy. Finally, IGX Explore outperforms pRESTO with regards to speed and scalability”.

As announced before, the ImmunoGenomiX (IGX) platform will be modularly expanded to become a comprehensive end-to-end immunosequencing data analysis platform designed to analyze, monitor, and compare immune repertoires in the context of immunotherapy development and at all stages of treatment and disease over time. Starting from high-throughput sequencing data, it will deliver an easy-to-read report appropriate for the specific application, be it research, diagnosis, patient stratification, or treatment monitoring. The IGX platform will allow customers to select their own sample preparation protocols and the HTS technology of choice. It requires no programming skills as the interface is intuitive and flexible.

The complete base module, IGX Explore, is now available and can perform clonality analysis of both TCR and BCR repertoires starting from raw sequencing data, report back the number of individual TCR/BCR clones and interactively visualize them in a user-friendly way. With IGX Explore, immunotherapy companies can boost their target discovery, research and drug development processes. Tailored software extensions to answer specific research or clinical questions can be custom-built in collaboration with ENPICOM’s team of immunologists and bioinformaticians.

Using a recently published and independent benchmark, ENPICOM showed in August that IGX Explore offered superior accuracy on TCRs and the current benchmark studies complement this picture for BCRs. More information about these benchmark studies can be found at the company’s website and will be published in a white paper shortly.

Source: ENPICOM

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Staten Biotechnology and Novo Nordisk collaboration on novel treatment for dyslipidaemia

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Staten Biotechnology and Novo Nordisk announced that they have entered into a collaboration and exclusive option agreement to develop novel therapeutics for the treatment of hypertriglyceridaemia.

Under the collaboration agreement, Novo Nordisk will provide research and development (R&D) funding and support for Staten to develop its lead asset STT-5058 for treatment of dyslipidaemia.

“Novo Nordisk is joining forces with Staten Biotechnology at an exciting time for us, with the company’s lead compound moving towards its first clinical trial, aiming to address the residual cardiovascular risk in patients with hypertriglyceridaemia,” said Dr Hilde Steineger, CEO of Staten Biotechnology. “This partnership provides the company not only with a knowledgeable development partner but also with a funding structure that allows founders, management and the founding investors to accelerate the development of company”.

“Our partnership with Staten Biotechnology is a key step in executing our strategic priority to expand into cardiovascular diseases. Hypertriglyceridemia is a serious risk factor for cardiovascular disease, in particular often present in people with diabetes and obesity. Staten Biotechnology has developed STT-5058, a new promising concept validated by human genetics, for the treatment of hypertriglyceridemia. Combining Staten’s know-how on STT-5058, the scientific excellence of the company’s executive team and founders, with Novo Nordisk experience in drug development and commercialization, the project holds potential to make a real difference for people suffering from cardiovascular disease,” said Marcus Schindler, Senior Vice President, Global Drug Discovery in Novo Nordisk.

Under the exclusive option agreement, Novo Nordisk has the right to acquire Staten Biotechnology and gain worldwide rights to STT-5058. Staten Biotechnology and its shareholders will potentially receive signing and exercise fees, R&D funding, and milestone payments of up to 430 million euros.

Source: Staten Biotechnology

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HollandBIO Leestip: 3x gentherapie in het FD

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Waar gentherapie in één behandeling genoeg heeft om een patiënt te genezen, krijgt de revolutionaire biotech ontwikkeling maar liefst driemaal het podium in het Financieele Dagblad. En niet zonder reden: het Nederlandse zorgsysteem is nog lang niet klaar om de potentie van de technologieën benutten. Van overheid tot bedrijfsleven, van ziekenhuis tot Zorginstituut en van patiënt tot verzekeraar: als het aan HollandBIO ligt, slaan we in 2019 de handen ineen om ervoor te zorgen dat Nederland dé internationale koploper wordt in de ontwikkeling, toepassing én financiering van gen- en celtherapie.

Lees meer (log in verplicht):

 

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ProQR’s QR-110 Data for Leber’s Congenital Amaurosis 10 published in Nature

ProQR, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, announced the publication of a peer-reviewed manuscript describing the previously announced interim results of a clinical trial of QR-110 for the treatment of Leber’s congenital amaurosis 10 (LCA10) in the journal Nature Medicine.

“It was enormously gratifying to see robust improvements in visual acuity and significant augmentations in the patient’s ability to detect lights, and impressive to observe these effects within the first three months following a single injection,” said Professor Artur V. Cideciyan, Ph.D., who was one of the co-investigators at the Scheie Eye Institute of the University of Pennsylvania. “LCA10 is a severe form of childhood blindness and this is a major step forward in the treatment of these previously incurable conditions,” said Professor Samuel G. Jacobson, M.D., Ph.D, who is the ophthalmologist caring for four of the patients enrolled in the study and is also a co-investigator at the Scheie Eye Institute.

Published results detail a planned interim analysis of the ongoing PQ-110-001 first-in-human clinical study, evaluating QR-110 in patients with LCA10. The publication focused on a landmark analysis of eight subjects who reached three months of single dose treatment experience. Results demonstrate a substantive overall improvement in best corrected visual acuity (BCVA) with a mean improvement of -0.67 LogMAR (SEM 0.32) in the treated eye versus 0.02 LogMAR (SEM 0.05) in the contralateral (control) eye (p=0.011). The majority of patients showed an increase of at least -0.3 LogMAR in the treated eye, which is generally considered clinically meaningful, and equivalent to 15 letters, or three lines of improvement for individuals able to read a standard eye chart. Visual acuity improvements were also associated with concordant improvements in full-field stimulus thresholds to both blue and red light, improved mobility course navigation and ocular instability measurement.

LCA10 is a severe inherited retinal dystrophy associated with mutations in the CEP290 gene. QR-110 is an RNA-based drug candidate that has the potential to restore sight or slow down the process of vision loss in patients with LCA10 by correcting the most common mutation causing LCA10, p.Cys998X.

The ongoing Phase 1/2 PQ-110-001 study of QR-110 will be completed and in parallel a Phase 2/3 “ILLUMINATE” study is expected to be initiated in the first half of 2019.

Source: ProQR

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