Het Zorginstituut beantwoordt de veel gestelde vragen rondom het beleid voorwaardelijke toelating weesgeneesmiddelen, conditionals en exceptionals. De voorwaardelijke toelating biedt de mogelijkheid om geregistreerde, veelbelovende geneesmiddelen voor ernstige, vaak zeldzame ziekten, waarvan de effectiviteit (nog) onvoldoende is aangetoond eerder beschikbaar te maken. Lees meer
Mymetics, a pioneer and leader in the research and development of virosome-based vaccines against life threatening and life disabling diseases, has signed a Research Collaboration Agreement with Baylor College of Medicine and Texas Children’s Center for Vaccine Development for evaluating their rationally designed recombinant protein-based SARS-CoV and SARS-CoV-2 antigens and combining them with Mymetics’ virosomes for the development of a safe and effective Covid-19 vaccine.
Since the end of April, Mymetics has started a project for the rapid development of a Covid-19 virosome-based vaccine and is thereby partnering with leading academic institutions to explore and select the best SARS-CoV antigens, which could not only be efficacious in protecting against Covid-19 but also safe, affordable and accessible for use in the global population.
Baylor College of Medicine in Texas is a leading institution with a track record for developing low cost, safe and effective vaccines against emerging and neglected tropical diseases through collaboration with the Texas Children’s Center for Vaccine Development. The vaccine center already has been successful in developing potential vaccine against SARS-CoV and with this expertise has already initiated development of a SARS-CoV-2 recombinant protein-based vaccine.
“Our vaccine center has traditionally focused on advancing recombinant protein-based vaccines, but we recognize that for them to be effective, appropriate delivery platforms need to be considered, especially to ensure long-term immunity. Through the collaboration with Mymetics we will evaluate the coupling of our COVID-19 vaccine candidates to their flexible carrier to bring a new dimension towards accelerating vaccines for global access,” said Dr. Maria Elena Bottazzi, Professor, Pediatrics & Molecular Virology & Microbiology at Baylor College of Medicine and co-director of Texas Children’s Hospital Center for Vaccine Development.
“There is no question that if we want to get back to a life as before and a normal functioning economy, the world needs the rapid development of a safe and efficacious vaccine against Covid-19. The only serious way to get there fast is to have global collaborations to bring cross functional expertise together, establish result focused projects to explore and select the best vaccine candidates coupled with safe platform technologies and move them forward to clinical trials,” said Ronald Kempers, CEO of Mymetics Corporation. “Mymetics virosomes have proven to be a safe, efficacious and flexible vaccine carrier with other infectious diseases and we believe, with the right antigens and the right collaborations, we can have a serious attempt at developing a virosome-based Covid-19 vaccine. We are very much looking forward to this collaboration with the team at Baylor College of Medicine.”
This collaboration is supported through the combined resources of Mymetics, Baylor and Texas’s Children’s Center for Vaccine Development and covers the initial preclinical development. To speed-up the development of a potential Covid-19 vaccine, Mymetics and Baylor are seeking additional funding opportunities to start in parallel the GMP manufacturing and preparations for clinical trials. The aim is to have a virosome-based Covid-19 vaccine candidate evaluated in humans within 12 months.
Source: Mymetics (press release)
Lava Therapeutics has entered into a research and license agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to discover and develop novel bispecific antibodies to gamma-delta T cells for the treatment of cancer. The collaboration was facilitated by Johnson & Johnson Innovation.
“We are excited to enter into this collaboration with Janssen, a global innovator and leader in the development of new medicines,” said Stephen Hurly, chief executive officer of Lava Therapeutics. “We strongly believe in the strength of our bispecific gamma-delta T cell engager platform and are committed to creating highly potent, target-specific therapeutics with increased durability and safety over current T cell-based approaches.”
Under the terms of the agreement, Lava Therapeutics will perform discovery and product development activities, and is eligible to receive an undisclosed financial package consisting of an upfront payment and potential development and commercial milestones, and future tiered royalties.
Intravacc, one of the leading translational research and development vaccine institutes with an extensive track record in developing viral and bacterial vaccines, today announced the development of three COVID-19 vaccine concepts. These concepts are all based on Intravacc’s platform technologies, that have been proven safe in humans.
“This global COVID19 pandemic requires an international effort to understand the complexity of the disease and to develop innovative COVID-19 emergency and conventional vaccines”, said Dr. Jan Groen CEO of Intravacc. “We also need to establish partnerships with pharma for large scale manufacturing and global distribution of these vaccines”.
To develop safe and effective COVID-19 vaccines, Intravacc is focusing on emergency and conventional vaccines. Intravacc’s emergency vaccine is designed to activate cytotoxic T-cell responses. This emergency vaccine would reduce mortality and morbidity and as a result reduces the pressure on the public health system. For this vaccine Intravacc uses its outer membrane vesicle (OMV) technology.
For its conventional COVID-19 vaccine Intravacc will use OMV’s as a carrier for spike proteins of the virus to induce a protective antibody response. For the third concept Intravacc’s approved Vero cell platform will be used to develop an intranasal vector vaccine based on Newcastle disease virus, that can replicate in humans but does not cause disease.
The expected development timelines for these COVID-19 vaccines to enter into phase 1 range from 9 to 21 months.
Source: Intravacc (press release)
Gisteren verscheen de jaarlijkse monitor dure geneesmiddelen medisch-specialistische zorg 2020. Goed nieuws, want dankzij de introductie van nieuwe geneesmiddelen kunnen steeds meer patiënten rekenen op een behandeling. De keerzijde is dat hierdoor de uitgaven aan geneesmiddelen ook groeien. En helaas groeit onze zorgbudgetboom niet tot in de hemel. Daarom moet er nu gezamenlijk werk gemaakt wordt van oplossingen die bijdragen aan het beheersen van de stijgende uitgaven, zodat de medisch-specialistische zorg ook in de toekomst ook voor iedereen toegankelijk blijft. Lees het persbericht van de NZa hier.
MyoKardia has announced positive topline data from the company’s Phase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM) (clinicaltrials.gov NCT03470545). Mavacamten demonstrated a robust treatment effect: the primary and all secondary endpoints of the EXPLORER trial were met with statistical significance (p≤0.0006 for all endpoints). Mavacamten was well tolerated, and meaningful improvements in symptoms, functional status and quality of life, as well as reduction or elimination in obstruction of the left ventricle, were observed among patients on treatment versus placebo.
“The extraordinary data from the EXPLORER pivotal trial confirm mavacamten’s ability to relieve dynamic outflow obstruction, control symptoms and improve quality of life in patients with hypertrophic cardiomyopathy,” said Iacopo Olivotto, M.D., Careggi University Hospital and lead clinical investigator for the EXPLORER-HCM clinical trial. “HCM is the most common inherited cardiovascular disease, and patients face an uncertain journey that all too frequently includes debilitating symptoms, as well as serious complications, such as heart failure, stroke and cardiac arrest. Mavacamten is the first drug developed to target the specific molecular defect of the disease. EXPLORER represents a major achievement toward a precision-medicine approach in cardiomyopathies and should provide great hope to a community painfully aware of the lack of disease-specific treatment options.”
The 30-week treatment with mavacamten resulted in a highly statistically significant outcome relative to placebo (p=0.0005) for the primary endpoint in the EXPLORER-HCM trial, a composite functional analysis designed to capture the treatment effect of mavacamten relative to placebo on both symptoms and cardiac function.
All secondary endpoints also demonstrated statistically significant and clinically meaningful improvements for mavacamten as compared to placebo. Secondary endpoints in the EXPLORER-HCM trial evaluated improvements in post-exercise left ventricular outflow tract (LVOT) peak gradient (p<0.0001), New York Heart Association (NYHA) functional classification (p<0.0001), peak VO2 (p=0.0006), the Kansas City Cardiomyopathy Clinical Summary Score (KCCQ-CSS) (p<0.0001), and the HCM Symptom Questionnaire Shortness of Breath Domain Score (p<0.0001).
Mavacamten was well tolerated and demonstrated safety results comparable to placebo, with no new findings observed. Ninety-eight percent of patients enrolled completed the study. Of the two percent who dropped out, none were due to reduced ejection fraction or symptoms of heart failure. Overall rates of adverse events, serious adverse events, and cardiac adverse events, including atrial fibrillation, were comparable for patients treated with mavacamten and placebo.
“The resoundingly positive data from EXPLORER bring us a significant step closer to improving the lives of people with serious cardiovascular conditions, starting with HCM, a debilitating disease estimated to affect one in every 500 people,” said Tassos Gianakakos, Chief Executive Officer of MyoKardia. “The activity and tolerability profile observed for mavacamten in this pivotal study underscores the profound impact and potential for therapeutics that target the underlying biology of disease. We look forward to the submission of MyoKardia’s first New Drug Application and, importantly, to serving the many patients that stand to benefit from mavacamten.”
The EXPLORER-HCM clinical trial is part of MyoKardia’s pivotal program studying mavacamten as a treatment for symptomatic, obstructive hypertrophic cardiomyopathy. MyoKardia plans to submit a New Drug Application to the U.S. Food and Drug Administration (FDA) in the first quarter of 2021. Results from the Phase 3 EXPLORER-HCM clinical trial will be submitted to a future professional meeting in 2020.
Source: Myocardia (press release)
Het moet even slikken zijn voor GroenLinks: het lot van de wereld ligt in handen van hun favoriete boeman. GroenLinks vangt al jarenlang bot in haar windmolengevecht tegen de “macht van de farmaceut”. En nu zijn we voor gezondheid, sociaal contact, zelfs voor een fysieke meetup of partijcongres totaal afhankelijk van de farmaceutische industrie. Een crisis is een kans, moeten partijprominenten Klaver en Eickhout gedacht hebben toen ze hun anti-farma retoriek weer eens recycleden in een NRC opiniestuk. Ditmaal door deze met veel kunst- en (v)liegwerk te koppelen aan de uitbraak van het coronavirus.
Het coronavirus stelt de wereld voor een uitdaging die zijn weerga niet kent. We hebben nieuwe diagnostica nodig, geneesmiddelen, vaccins, medische hulpmiddelen en apparatuur. Liever vandaag dan morgen, genoeg en toegankelijk voor miljarden mensen. Onze hoop is gevestigd op biotech bedrijven, farmaceuten en medtech bedrijven. Ze kunnen wereldwijd op steun rekenen van overheden, kennisinstellingen, WHO, de Gates Foundation, noem maar op. Binnen een mum van tijd hebben bedrijven wereldwijd al talloze potentiële vaccins, geneesmiddelen en tests in ontwikkeling. En daarmee nemen ze ongekend grote investeringsrisico’s, want veel ontwikkelingen zullen mislukken en de investeringen verdampen. Gelukkig zijn er partijen zo gek om dit risico te nemen. Bedrijven starten bijvoorbeeld grootschalige productie op voordat er ook maar enige zekerheid is of het product ooit de eindstreep haalt . Alles om maar zo snel mogelijk, zo veel mogelijk levens te redden en om mensen overal ter wereld uit dit ellendige isolement te verlossen.
Tot nu toe zijn alleen bedrijven succesvol in het ontwikkelen van innovatieve, nieuwe geneesmiddelen en vaccins. Want vergeet niet: het staat iedereen vrij om medische innovaties te ontdekken, ontwikkelen en te leveren. Er is geen wet die zegt dat alleen bedrijven dit mogen. Waar deze tijd om vereende krachten, samenwerking en misschien zelfs verzoening vraagt, polariseert GroenLinks liever. Het opiniestuk bevat geen enkele oplossing, en heeft enkel ten doel om de sector die ons uit de penarie kan helpen, een stok in de wielen te gooien. Dat getuigt van onkunde en ongekend opportunisme.
Klaarblijkelijk slijten Klaver en Eickhout hun leven liever in quarantaine, dan met hulp van de farmaceutische industrie in gezondheid en sociale vrijheid.
Zo. En dan gaan we nu weer werken aan oplossingen.
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Inmiddels zijn, na de eerste proefpersonen in Duitsland, ook de eerste Amerikaanse proefpersonen geïnjecteerd met een dosis van de kandidaat-vaccins uit het BNT162 vaccinprogramma dat Pfizer en BioNtech ontwikkelen tegen het coronavirus. Om geen tijd te verliezen schalen de bedrijven hun productie nu al op. Als het onderzoek een goedgekeurd mRNA-vaccin oplevert, kunnen nog dit jaar miljoenen vaccins worden geproduceerd voor mensen die het meest kwetsbaar zijn voor het coronavirus COVID-19.
Vorige week werden al twaalf Duitse proefpersonen ingeënt met een van de kandidaat-vaccins die onderdeel uitmaken van het BNT162-vaccinprogramma. In het eerste stadium van het BNT162-onderzoek worden in totaal 200 gezonde proefpersonen van tussen de 18 en 55 jaar oud gevaccineerd met vier verschillende mRNA-vaccins, in verschillende doseringen. Tijdens de klinische onderzoeksfase produceert BioNTech het vaccin in haar GMP-gecertificeerde fabrieken in Europa
Het doel van deze eerste onderzoeksfase is om te bepalen welk vaccin het lichaam het beste activeert om antilichamen tegen het coronavirus te maken. Ook wordt beoordeeld wat de optimale dosering van het vaccin is en of het vaccin veilig is. Als de onderzoekers dat weten, wordt ook een groep van 160 proefpersonen van tussen de 65 en 85 jaar gevaccineerd.
De Amerikaanse proefpersonen worden aangeleverd door vier grote universiteitsziekenhuizen. Dat zijn de NYU Grossman School of Medicine en de University of Maryland School of Medicine en binnenkort ook de University of Rochester Medical Center/Rochester Regional Health and Cincinnati Children’s Hospital Medical Center.
Ondertussen zijn Pfizer en BioNTech bezig met het opschalen van hun productiefaciliteiten om een goedgekeurd vaccin wereldwijd te kunnen leveren. Het doel is om in de loop van 2020 miljoenen doses vaccin te produceren voor mensen die het meest kwetsbaar zijn voor het coronavirus. Het aantal doses moet vervolgens oplopen tot honderden miljoenen in 2021.
De vaccinproductiefaciliteiten van Pfizer in het Belgische Puurs en drie Amerikaanse fabrieken in Massachusetts, Michigan en Missouri zullen als eerste het COVID-19 vaccin gaan maken. Indien nodig zal Pfizer nog meer locaties beschikbaar maken in haar wereldwijde netwerk voor de productie van een goedgekeurd COVID-19 vaccin.
“We zijn ontzettend trots dat Pfizer Puurs een belangrijke rol kan spelen in de wereldwijde productie van een potentieel COVID-19 vaccin als antwoord op deze pandemie. Dit is een erkenning voor het wereldniveau in Puurs, en voor de kennis en ervaring van onze bijna 3000 werknemers,” zegt Site Lead Pfizer Puurs, Luc Van Steenwinkel.
BioNTech breidt de productiecapaciteit van haar al bestaande mRNA-fabrieken in Mainz en Idar-Oberstein verder uit, zodat een wereldwijde levering van het vaccin mogelijk wordt. BioNTech werkt in China samen met Fosun Pharma en wil het vaccin ook daar gaan testen en op de markt brengen.
‘We zetten onze beste middelen in de strijd tegen het coronavirus’
’’Met ons unieke en sterke klinische onderzoeksprogramma dat nu ook in Amerika start, hopen we in samenwerking met BioNTech en de regelgevende autoriteiten snel een veilig en effectief vaccin te kunnen bieden aan mensen die dat het hardste nodig hebben,’’ zegt Albert Bourla, CEO en voorzitter van de raad van bestuur van Pfizer. ’’In slechts vier maanden tijd is het ons gelukt om van preklinisch onderzoek de stap te maken naar onderzoek bij proefpersonen. Dat is een buitengewone prestatie; in de strijd tegen het coronavirus COVID-19 zetten we onze beste middelen in, van de voorzieningen in het laboratorium tot aan het productieproces.’’
’’Het is bemoedigend dat we meer dan tien jaar ervaring met het ontwikkelen van het mRNA-platform kunnen gebruiken om in een hele korte periode een wereldwijd onderzoeksprogramma op te zetten voor de kandidaat-vaccins die we ontwikkelen,’’ zegt Ugur Sahin, CEO en medeoprichter van BioNTech. ’’We zijn optimistisch dat het testen van vier verschillende kandidaat-vaccins bij proefpersonen ertoe leidt dat we ontdekken wat de veiligste, meest effectieve vaccinatieopties tegen COVID-19 zijn.’’
Bron: Pfizer (persbericht)
Kiadis Pharma has received approval from the U.S. Food and Drug Administration (FDA) to start its NK-REALM phase 2 clinical trial, which the Company submitted through an investigational new drug (IND) application in April 2020. Kiadis is developing K-NK002 as an adjunctive therapy to the haploidentical HSCT standard of care with the goal of reducing relapse rates.
Kiadis’ Phase 2 study, called NK-REALM (haploidentical NK-cells to prevent post-transplant RElapse in AML and MDS), will evaluate the use of K-NK002 as an adjunctive therapy for blood cancer patients undergoing a haploidentical HSCT with the current standard of care, the post-transplant cyclophosphamide (PTCy) protocol. The study, which will be conducted with the NIH-supported Blood and Marrow Transplant Clinical Trials Network (BMT CTN), funded by the National Heart, Lung and Blood Institute and the National Cancer Institute, both part of the National Institutes of Health (NIH), will enroll 63 patients at leading transplant centers in the U.S. The first 6 patients in the NK-REALM study will be evaluated as a safety lead-in. The study is designed to confirm earlier proof-of-concept data in 24 patients, which showed that adjunctive treatment with K-NK002 has the potential to substantially improve outcomes for patients in need of a HSCT.
The Company’s phase 2 study will evaluate K-NK002 produced using Kiadis’ proprietary PM21 technology platform that enables high dose, low cost, scalable and industrial production of NK-cell therapy without the risk of residual tumor cells in the final product. This is the first human trial using drug produced with Kiadis’ PM21 technology. Kiadis is able to proceed directly with a phase 2 trial based on bridging from K-NK cells produced with FC21 used in earlier clinical studies to K-NK cells now produced with PM21.
Arthur Lahr, CEO of Kiadis Pharma, commented, “The rapid approval of the IND that allows us to initiate this phase 2 clinical trial is an important step forward for Kiadis in bringing K-NK cell therapy to patients in need. This study is the first with K-NK cells produced with our PM21 particle production platform. Our ability to proceed directly with a Phase 2 leverages existing clinical data and further validates similarity between K-NK cells produced with FC21 and PM21. We will now work with BMT CTN to start the study and expect to enroll the first patients in the safety lead in this year.”
Kiadis Pharma’s K-NK-Cell Therapies
Kiadis Pharma’s K-NK platform is designed to deliver potent NK cells to help patients, without the need for genetic engineering. Kiadis Pharma’s programs consist of off-the-shelf and haploidentical donor NK-cell therapy products for the treatment of liquid and solid tumors as adjunctive and stand-alone therapies.
The Company’s PM21 particle technology enables improved ex vivo expansion and activation of cytotoxic NK cells supporting multiple high-dose infusions. Kiadis Pharma’s proprietary off-the-shelf NK-cell platform is based on NK cells from unique universal donors and can make NK-cell therapy product rapidly and economically available for a broad patient population across a wide range of indications. Kiadis Pharma is developing K-NK002 as an adjunctive immunotherapeutic on top of HSCT, and K-NK003 for the treatment of relapse/refractory acute myeloid leukemia. In addition, Kiadis Pharma has pre-clinical programs evaluating NK-cell therapy for the treatment of solid tumors.
Source: Kiadis Pharma (press release)
InteRNA Technologies announced today regulatory approvals in the Netherlands and Belgium for the initiation of the first-in-human (Phase I) clinical trial testing its lead microRNA candidate, INT-1B3, in patients with advanced solid tumors. INT1B3 is a lipid nanoparticle (LNP) formulated, chemically modified miR-193a-3p mimic that can be delivered by systemic administration to cancer cells. Due to the ongoing COVID-19 pandemic, clinical trial initiation has been delayed and is now anticipated to start during H2 2020. The Company will provide an update once the trial has officially launched.
The study is a multicentric, open-label and multiple ascending dose clinical trial that will investigate the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of INT-1B3 in patients with advanced solid tumors. The study is expected to enroll a total of up to 80 patients at 12 clinical centers in the United States and Europe. The first part of the trial is a Phase Ia dose escalation study, conducted in the Netherlands and Belgium, which will enroll approximately 30 patients with advanced solid tumors. These patients will receive INT-1B3 via infusions twice weekly in 21-day cycles. Subsequently, approximately 50 patients with either hepatocellular carcinoma or triple negative breast cancer will be enrolled in the Phase Ib dose expansion part of the trial. Initial data readout from the Phase Ia study is expected in the end of 2021.
“The multi-targeted nature of microRNAs represents a novel approach to treating a broad range of cancer indications by effectively targeting several biochemical pathways simultaneously,” said Dr. Roel Schaapveld, CEO of InteRNA Technologies. “Driven by the promise of microRNAs as a treatment modality in cancer, our understanding of the underlying mechanism of action of INT1B3 and the identification of a delivery vehicle for optimized performance, our primary focus has been on preparing our lead candidate for first-in-human clinical trials. As such, we are truly excited to bring INT-1B3 into the clinic and to gain deeper insights on how it reacts against solid tumors.”
“INT-1B3 is a promising and novel anti-cancer approach with anti-tumor potential involving direct effects on tumor cells and modulation of the immunosuppressive tumor microenvironment leading to immune system activation,” added Prof. Dr. Emile Voest, Chairman of InteRNA’s Scientific Advisory Board and Medical Director of the Netherlands Cancer Institute in Amsterdam. “The safety profile and significant anti-tumor efficacy demonstrated in preclinical studies provide a strong basis for first-in-human studies with INT-1B3.”
Source: InteRNA (press release)