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BunyaVax provides technology to develop a human Rift Valley Fever vaccine in a $12.5 million project financed by CEPI

BunyaVax is technology provider in a consortium led by Wageningen Bioveterinary Research and financed by CEPI to develop a human vaccine against Rift Valley Fever. With support from the European Union’s (EU’s) Horizon 2020 programme, CEPI will provide up to US$12.5 million for vaccine manufacturing, preclinical research, and a phase 1 study to assess the safety, tolerability, and immunogenicity of a single-dose vaccine candidate (RVFV-4s) against Rift Valley fever virus for use in humans.

To produce this vaccine candidate, researchers at WBVR have altered the genome of the Rift Valley fever virus in a way that significantly weakens it. BunyaVax as a spin-off company from WBVR owns the underlying technology and further develops the vaccine towards application. The “attenuated” Rift Valley fever virus will now be used as a vaccine to generate a lasting immune response against the disease. This investment is part of CEPI’s third call for proposals, launched earlier this year with support from the EU’s Horizon 2020 research and innovation programme under grant agreement No. 857934.[1]

Rift Valley fever

Rift Valley fever vaccines have already been used successfully to protect livestock, but currently none have been licensed for use in humans.

Rift Valley fever virus mainly affects people living in pastoral communities in low-income and middle-income countries.[2] While the virus mostly infects humans through contact with the blood or organs of infected animals, there is a concern that it could also be transmitted from human-to-human by the Aedes aegypti mosquito, which could change its epidemiology considerably.[3] In view of the epidemic threat posed by this disease, the WHO has classified it as a priority pathogen in need of urgent R&D investment.[4]

Rift Valley fever kills about one in every hundred people infected. In people who develop the haemorrhagic form of the disease, the fatality rate is as high as 50%.[5],[6]

The virus was first identified in 1931 during an investigation into an outbreak among sheep on a farm in the Rift Valley of Kenya. Multiple outbreaks have since been reported across the African continent and on the Arabian Peninsula.

Between May and June, 2018, concurrent cases of Rift Valley fever were reported in farmers in South Africa and Kenya, nearly 5000 km apart.[7],[8] There is also an ongoing outbreak on the island of Mayotte, a French overseas territory in the Indian Ocean. As of May 13, 2019, 129 human and 109 animal cases of Rift Valley fever have been confirmed on the island.[9]

Prof. Jeroen Kortekaas, project lead and molecular virologist at WBVR, said:

“With this strategic collaboration, CEPI acknowledges the concept of One Health, by bringing together veterinary and public health experts to fight an emerging virus that affects both animals and humans.”

Jochem Bossenbroek, CEO of BunyaVax, said: “BunyaVax forwards a new generation of vaccines against infectious diseases, based on breakthrough platform technology originating from Wageningen Bioveterinary Research. We are excited to be working with CEPI to advance our lead vaccine against Rift Valley Fever and take key steps towards protecting societies at risk.”

[1] https://cepi.net/news_cepi/cepi-launches-call-for-proposals-to-develop-vaccines-against-rift-valley-fever- and-chikungunya-viruses/

[2] https://www.sciencedirect.com/science/article/pii/S2352771418300363

[3] https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0210122

[4] https://www.who.int/blueprint/priority-diseases/en/

[5] https://www.who.int/news-room/fact-sheets/detail/rift-valley-fever

[6] https://www.who.int/emergencies/diseases/rift-valley-fever/en/

[7] https://www.liebertpub.com/doi/pdf/10.1089/vbz.2018.2357

[8] https://www.who.int/csr/don/18-june-2018-rift-valley-fever-kenya/en/

[9] https://www.who.int/csr/don/13-may-2019-rift-valley-fever-mayotte-france/en/

Source: BunyaVax

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Tweede Kamer: vergunningverlening voor cel- en gentherapie moet sneller en beter

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De Tweede Kamer roept Minister Van Nieuwenhuizen van Infrastructuur en Waterstaat (IenW) op haast te maken om de vergunningverlening voor klinisch onderzoek met, en de toepassing van cel- en gentherapie sneller en beter te laten verlopen. Arne Weverling (VVD) diende hiertoe een motie in die op brede steun van de Kamer kon rekenen. Die verbetering is urgent en noodzakelijk, want de huidige procedure voor vergunningverlening is onnodig lang en veeleisend door een zware milieutoets. Doordat Nederland klinisch onderzoek met, en de toepassing van cel- en gentherapie standaard indeelt in de risicoklasse Introductie in het Milieu (IM), dreigt de winst van deze nieuwe generatie therapieën aan ons land voorbij te gaan. In reactie op de motie gaf de Minister aan het gevoel van urgentie met de Tweede Kamer te delen. HollandBIO is blij met de belofte van de minister om met gezwinde spoed werk te maken van een oplossing.

HollandBIO gaf eerder aan teleurgesteld te zijn in de terughoudende Kamerbrief van de minister om over te gaan tot aanpassing van de nationale wet- en regelgeving om de lichtere risicoklasse van Ingeperkt Gebruik (IG) als uitgangspunt te bewerkstelligen. De motie van Arne Weverling constateert echter dat de Minister van mening is dat de huidige regelgeving op het gebied van klinisch onderzoek naar gentherapie al mogelijkheden biedt voor aanvragen op basis van IG. Als dat zo is, is de door HollandBIO aangedragen aanpassing van Besluit ggo overbodig en is verbetering sneller een feit. Het is nu aan het ministerie om deze nu in de praktijk onbegaanbare route vrij te maken. De Kamer is in ieder geval duidelijk: de regering moet haast maken om alle mogelijke verbeteringen van beleid door te voeren en hiervoor met spoed extra capaciteit vrijmaken.

Aan het eind van de zomer vindt een overleg plaats tussen de betrokken ministeries en veldpartijen, onder wie HollandBIO, om de mogelijkheden voor het wegnemen van de huidige belemmeringen met elkaar te bespreken. IenW lijkt daarmee dus de daad bij het woord te voegen. Natuurlijk is HollandBIO pas tevreden als een oplossing daadwerkelijk gerealiseerd is. Want alleen dan kan Nederland op alle fronten profiteren van deze baanbrekende nieuwe therapieën.

Leestip!

Marc Kaptein, medisch directeur bij Pfizer Nederland en bestuurslid van HollandBIO, klom in de pen en schreef een uitstekende blog over de kansen om gen- en celtherapie te verzilveren. Je leest zijn blog hier!

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Now online: English information about CBG-ZIN Parallel Procedures Pilot

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As previously highlighted in our newsletter, the Dutch Medicines Evaluation Board (CBG-MEB) and the National Health Care Institute (Zorginstituut Nederland: ZIN) joined forces in a pilot to accelerate access to innovative medicines in the Netherlands. To inform your international colleagues and/or international affiliates about this opportunity, the information about the “CBG-ZIN Parallel Procedures pilot” is now available online.

Recently, HollandBIO and the Association of Innovative Medicines organized together with CBG-MEB and ZIN a well-attended information session about the pilot, during which Kevin Liebrand (project lead on behalf of CBG-MEB) and Pauline Pasman (project lead on behalf of ZIN) elaborated on the aim of the pilot and the prerequisites for participation. Companies were very enthusiastic about the pilot, based on the amount of questions that were asked during the session. Frequently asked questions will be combined and published online in the near future.

The pilot is a seamless fit with the HollandBIO program Faster & better from bench to bedside, where we work towards solutions that boost both health, innovation and affordability. This pilot is a first step towards a more flexible health ecosystem, where the Netherlands can achieve the full potential of tomorrow’s medicines. We are therefore very curious who will be the frontrunner of this faster & better opportunity.

More information:

The most important information about the pilot can be found here, including a link to the Interest Form – CBG-ZIN Parallel Procedures pilot. Explanation in English of the interest form can be found here. If you have any questions, you can also always contact Marit.

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Khondrion granted Orphan Drug Designation

Khondrion, a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that the European Commission has granted Orphan Drug Designation (ODD) to its lead asset, KH176, for the treatment of patients with maternally inherited diabetes and deafness (MIDD). MIDD is a subtype of diabetes often accompanied by hearing loss that is caused by a mutation within mitochondrial DNA.

KH176, Khondrion’s wholly-owned lead asset is a potential first-in-class oral small molecule in phase IIb clinical development to treat a range of mitochondrial diseases. The compound, which belongs to a new class of drugs used to control oxidative and redox pathologies, has previously been granted ODDs for MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders and Leigh disease by the EMA, and for all inherited mitochondrial respiratory chain
disorders by the US Food and Drug Administration (FDA).

An orphan designation allows a pharmaceutical company to benefit from incentives to develop a medicine for a rare disease, such as reduced fees in protocol assistance and a period of market exclusivity following regulatory approval. European applications for Orphan Designation are examined by the Committee for Medicinal Products (COMP), which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question.

Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: “MIDD is a rare and extremely debilitating form of diabetes with a critical need for new treatments. This designation is an important incentive for companies like Khondrion investing in rare disease research and brings us another step closer to advancing the science of mitochondrial disease for the benefit of patients.”

Source: Khondrion

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HollandBIO welcomes iClusion as a new member

HollandBIO connects and supports the Dutch biotech industry. We represent 200 companies in the field of medical, agrifood and industrial biotech. And, HollandBIO is growing steadily. Today, we are proud to announce that iClusion joined the HollandBIO community!

Clinical trials don’t just advance science; they also serve as in-the-moment treatment alternatives for patients. iClusion aims to make the clinical trial process faster, more effective, and more accessible for cancer patients and oncologists. “Our Trial Eye platform connects study sponsors, hospitals, physicians, and their patients in a closed-loop ecosystem,” says CEO Hanneke Janssen. Rather than each group remaining siloed, iClusion’s clinical study portal invites various stakeholders to connect. “This speeds up clinical trial accrual and therefore the entire drug development process.”

iClusion invites innovative Dutch Biotech to start their clinical trials through iClusion’s Trial Eye, which is already possible in the Netherlands and Belgium and keeps expanding. Instead of relying on proven giants, which are slow no matter what they claim, they can now rely on the fast and flexible Trial Eye services of iClusion for study startup and patient recruitment.

iClusion believes its new model is nothing short of revolutionary. “This is the only initiative designed from the viewpoint of the 2 most important stakeholders within clinical trials; the patient and their treating physician. If they have easy access to clinical trials and can participate, drug development can speed up,” Janssen says. “The footprint footprint iClusion wants to leave on this planet is changing the lives of cancer patients.

www.iclusion.com

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HollandBIO welcomes Oncode as a new member

HollandBIO connects and supports the Dutch biotech industry. We represent 200 companies in the field of medical, agrifood and industrial biotech. And, HollandBIO is growing steadily. Today, we are proud to announce that Oncode joined the HollandBIO community!

Oncode wants to drive breakthrough innovations in the diagnosis and treatment of cancer. The ultimate goal is to enable patients to survive cancer, improve the quality of life for those affected by cancer and contribute to a more affordable health care system. To put it simple: outsmarting cancer, impacting lives.

To do so, Oncode has joined the forces of over 800 molecular cancer researchers in 62 research groups across 12 universities and research institutes in The Netherlands. Our scientists work under a common strategy, with six defined research themes. We break with the competitive model of science and facilitate collaboration between scientists and with the private sector.

Societal and economic impact is a critical component of Oncode’s unique approach. We work hard to make sure that new findings from the lab, find their way to patients in the most optimal way. Academic research results manifest themselves as intellectual assets in the form of people, scientific publications, know-how, data, research tools, biological materials, software and inventions. Oncode aims to accelerate the development of such discoveries into clinical and economic benefits.

Their dedicated team of business development specialists forms a single point of contact for both our research community and the life science ecosystem in The Netherlands and abroad. We aim to create value for the Dutch life sciences sector by investing in the development of new technologies and products, building close interactions with industry to develop durable partnerships, attract international investments and research funding and by creating, growing and retaining oncology companies in the Netherlands.

Oncode is funded by the Dutch Cancer Foundation, together with the Dutch ministry of Economic Affairs & Climate Policy, the ministry of Education Culture & Science, the ministry of Health, Welfare & Sport, Health~Holland, NWO and ZonMw with a total of €120 million until 2022. This long term funding allows our scientists to take steps into uncharted territory and start high risk – high gain projects. We believe that this approach will lead to new paradigms for treating and diagnosing cancer.

Do you want to learn more about the possibilities to collaborate with Oncode, please reach out to Yuva Oz

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Kiadis updates status of marketing authorization application for ATIR101

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Kiadis, a clinical-stage biopharmaceutical company, today announced that it has received feedback from the European Medicines Agency (EMA) related to the
marketing authorization application (MAA) seeking approval of ATIR101 in hematopoietic stem cell transplant (HSCT). As previously announced, Kiadis submitted responses to the second Day 180 list of outstanding issues for ATIR101 to EMA in May 2019.

As part of the review process, EMA today informed Kiadis that it will convene a Scientific Advisory Group (SAG) in September, comprising of experts in hematology and HSCT to assist EMA in arriving at a determination.

“We appreciate the feedback from EMA and look forward to continuing our interaction with them during the review of our MAA for ATIR101,” said Arthur Lahr, CEO, of Kiadis Pharma. “With the SAG meeting now preceding the subsequent CAT and CHMP meetings as part of the EMA approval process, we are changing our guidance to potential EU conditional approval in 2020.”

While initial launch in the EU is delayed, Kiadis is on track to complete enrollment in the Phase 3 ATIR101 HSCT study in 2021 to support potential marketing approval in the US. Additionally, the company is also on track to start the Phase 1/2 studies of CSDT002 in HSCT and relapse and refractory acute myeloid leukemia in 2020. With a novel cell-based cancer immunotherapy platform consisting of both T-cell and NK-cell technologies, Kiadis has the opportunity to potentially revolutionize transplants and develop novel cancer cell therapies.

Source: Kiadis Pharma

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Gilead to submit new drug application for filgotinib to FDA

Gilead today announced that at a recent pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA), the company provided an update about the investigational oral, selective JAK1 inhibitor, filgotinib. The company discussed with the agency the Phase 3 FINCH studies, as well as the ongoing Phase 2 MANTA safety study assessing semen parameters with filgotinib treatment in men with moderately to severely active ulcerative colitis or Crohn’s disease. As a result of this discussion, a path forward has been established to submit the NDA for filgotinib as a treatment for rheumatoid arthritis in 2019.

Source: Galapagos

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Staatssecretaris Blokhuis op stoom

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Staatssecretaris Blokhuis wil gezondheidswinst door vaccinaties maximaal verzilveren, zo liet hij de Tweede Kamer afgelopen week in een uitgebreide Kamerbrief weten. De Kamerbrief meldt dat de daling van de vaccinatiegraad tot staan is gebracht. Volgens Blokhuis geen reden om op zijn lauweren te rusten: “dit motiveert mij des te meer om onverminderd in te zetten op de maatregelen uit mijn brief Verder met vaccineren”. We roemden al eerder de doortastende aanpak van Blokhuis op het vaccinatiedossier. En ook nu weer is HollandBIO blij met de ambitie die uit de brief spreekt. We zetten de hoogtepunten uit de brief graag voor je op een rij.

Pneumokokkenvaccinatie voor ouderen

Vanaf het najaar van 2020 gaat de overheid ouderen wapenen tegen de pneumokokken. De Staatssecretaris neemt nu definitief het advies van de Gezondheidsraad over om 60, 65, 70 en 75 jarigen een vaccinatie aan te bieden. In eerste instantie mogen gegadigden zich bij de huisarts melden voor deze prik. Of dit de best passende aanpak is, zal na twee jaar uit een evaluatie blijken.

Werkdruk bedreigt voorspoedige implementatie van nieuwe vaccins

De werkdruk bij de jeugdgezondheidszorg (JGZ) is hoog, zo constateert Blokhuis. En dankzij de implementatie van diverse nieuwe vaccins, waarvoor de JGZ verantwoordelijk is, zal die niet snel afnemen. “De praktijk kan geen oneindige hoeveelheid wijzigingen tegelijkertijd aan”, concludeert Blokhuis. En dus liggen vertragingen bij invoer op de loer. Volgens HollandBIO is er maar één structurele oplossing: extra capaciteit en middelen. Op die manier kan de werkdruk worden verlicht en vinden al die vaccins snel hun weg naar de juiste arm.

Een verbeterde communicatiestrategie

Blokhuis wil zich nog duidelijker uitspreken vóór het belang van vaccinatie. Op verzoek van de Tweede Kamer gaat hierbij extra aandacht uit naar ervaringsverhalen. Die krijgen dan ook een prominente plek op een te ontwikkelen website, waar jong en oud terecht kunnen voor informatie en actualiteiten over vaccineren. Het RIVM werkt intussen aan een vergrote zichtbaarheid van de al bestaande website over vaccinatie. Tot slot zet de staatssecretaris extra middelen in voor aanvullende gesprekstrainingen van professionals.

Benieuwd naar de hele Kamerbrief? Lees hem hier.

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First volunteer dosed with microneedle patch of MyLife Technologies at CHDR

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MyLife Technologies started a first-in-human study with its ceramic nanoporous MicroNeedle Arrays (npMNA) technology at Centre for Human Drug Research (CHDR) in Leiden (NL) today. npMNA patches loaded with a pharmaceutical peptide formulation are being tested in healthy volunteers

In many cases pharmaceutical peptides are parenterally administered. Existing alternatives for peptide delivery are among others intranasal sprays or subcutaneous implants. npMNA patch administration could lead to reduced peak concentrations and modified drug release and should overcome several of the drawbacks of the existing delivery routes: npMNA patches are minimally invasive, pain-free and allow for self-administration. The drug formulation is stored in the nanopores of the npMNA devices and diffuses into the patient once the npMNA patches have been applied onto the skin. Drug release rates from the nanopores can be tuned. This makes MyLife Technologies’ npMNA patches a versatile drug delivery system.

“We have entered an exciting new phase in the development of our npMNA technology”, says Pieter Jan Vos, CEO of MyLife Technologies, “We have found a valuable partner in CHDR, an experienced clinical CRO in the field of novel concepts and dermatology”.

Source: MyLife Technologies

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