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Pharming koopt rechten medicijn zeldzame aandoening van Novartis

Leiden, 13 augustus 2019: Pharming Group NV (Euronext Amsterdam: PHARM) heeft een ontwikkelingssamenwerkings- en licentieovereenkomst gesloten met Novartis voor het ontwikkelen en commercialiseren van CDZ173 (leniolisib), een klein molecuul fosfoïnositide 3-kinase delta (PI3Kẟ)-remmer die door Novartis wordt ontwikkeld voor de behandeling van patiënten met het Geactiveerd PI3K-delta-syndroom (“APDS”).

APDS is een primaire immuundeficiëntie veroorzaakt door een mutatie in het PIK3CD-gen dat de activiteit van PI3Kẟ verhoogt, een aandrijver van activiteit in het immuunsysteem. Als gevolg van deze overactiviteit kunnen de cellen die betrokken zijn bij de immuunrespons niet goed worden uitontwikkeld, wat betekent dat patiënten niet goed kunnen reageren op infecties en vroege celdood kunnen ondervinden. Patiënten hebben vaak een functioneel onvermogen om infecties te bestrijden, maar lopen ook het risico op de ontwikkeling van onder andere beschadiging van de luchtwegen en van bepaalde soorten kanker. Het betreft een uiterst zeldzame aandoening met incidentiepercentages wereldwijd van ongeveer 1 à 2 per miljoen. Belangrijk is dat er een genetische test verkrijgbaar is die patiënten kan identificeren die baat zullen hebben bij CDZ173, waarmee dit programma voor ‘gepersonaliseerde therapie’ zorgt voor APDS-patiënten en hun familieleden die ook de mutatie hebben.

Novartis heeft alle preklinische en klinische werkzaamheden tot op heden voltooid en zal doorgaan met het lopende registratieonderzoek, alsmede met de lopende open-label extensiestudie. Pharming zal met Novartis samenwerken bij de voltooiing van de rekrutering van patiënten voor de lopende studie. Na goedkeuring zal Pharming CDZ173 gaan commercialiseren via zijn bestaande verkoopinfrastructuur in de VS en Europa en onderzoeken hoe het medicijn in andere mondiale markten beschikbaar kan worden gemaakt.

Sijmen de Vries, Chief Executive Officer van Pharming, zegt in toelichting op de overeenkomst:

“Deze transactie betekent een geweldige mijlpaal voor Pharming. De licentie voor CDZ173 is onze eerste stap waarmee we voortbouwen op het commerciële succes van Ruconest in erfelijk angio-oedeem en waarmee we ons portfolio verder kunnen laten groeien en diversificeren. We zijn bijzonder enthousiast over het profiel van dit medicijn en over de match tussen het werkingsmechanisme en de pathologie van de ziekte. Het medicijn is naadloos in te passen in onze bestaande medische en commerciële infrastructuur en vormt een geweldig voorbeeld van waar solide wetenschap en een fundamenteel begrip van aandoeningen samenkomen voor het creëren van een gepersonaliseerde therapie-optie voor patiënten zonder uitzicht op behandeling. We zijn verheugd ons te kunnen voegen in Novartis’ commitment naar deze patiënten en zien uit naar de gezamenlijke afronding van de ontwikkeling van dit medicijn.”

Conform de voorwaarden van de overeenkomst doet Pharming een vooruitbetaling van € 17,9 miljoen. Novartis heeft recht op wettelijke en commerciële mijlpaalbetalingen en op een dubbelcijferig percentage royalties over de netto-omzet.

Bron: https://www.pharming.com/news/pharming-acquires-exclusive-license-cdz173-late-stage-drug-treatment-apds

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ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP)

LEIDEN, Netherlands and CAMBRIDGE, Mass., Aug. 12, 2019 (GLOBE NEWSWIRE) — ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for QR-1123. ProQR plans to start enrolling patients in a Phase 1/2 trial for QR-1123 in 2019.

QR-1123 is a first-in-class investigational oligonucleotide designed to address the underlying cause of the vision loss associated with autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO) gene.

P23H is the most prevalent mutation associated with adRP in the U.S. This disease causes progressive vision loss in approximately 2,500 patients in the United States, leading to blindness in mid-adulthood. There are no approved therapies for adRP and QR-1123 is the first investigational medicine to be developed for patients that suffer from this disease.

“We are pleased to have an open IND for QR-1123, based on which we will be advancing our next inherited retinal disease program into the clinic this year,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “This represents our fifth IND in less than five years and our third clinical program for severe genetic eye diseases. With a strong in vitro and in vivo proof-of-concept, we are excited about the potential of this medicine to make a positive impact on the lives of patients with adRP.”

Source: https://ir.proqr.com/news-releases/news-release-details/proqr-announces-clearance-ind-start-clinical-trial-qr-1123

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HALIX: NEW CGMP FACILITY STARTING OPERATIONAL PRODUCTION IN Q4-2019

Leiden, the Netherlands, August 9th 2019 – HALIX, a contract development and manufacturing organization of clinical and commercial proteins and viral products, based in the Netherlands, announced today that they have met their major milestone with the interim delivery of their new 6,700 m2 cGMP facility in Leiden Bio Science Park.

Following one year of construction, HALIX has completed its new state-of-the-art cGMP facility for the development and production of biopharmaceutical drug substances.

The new five-level production facility contains a state-of-the-art manufacturing line for viral vaccines and viral vectors and a separate protein manufacturing area with a capacity up to 1,000 Liter single-use bioreactors. In addition, lab space is available for process development, analytical development and quality control. All cleanroom areas have a unidirectional process flow and are designed to allow commercial manufacturing of biopharmaceuticals products.

“This new facility offers our current and future clients capacity and flexibility for future expansion and will allow for cGMP manufacturing solutions for viral products, proteins, gene therapy and client-specific new technologies.” said Roland Hecht, HALIX Chief Customer Officer.

Alex Huybens, HALIX Chief Operations Officer, further added “We are very pleased to have met this major milestone for our new manufacturing facility. We have made great progress under very aggressive timelines. After a construction time of just 12 months we are now able to start production of viral products and protein production will follow soon. This milestone aligns with our growth and expansion strategy that will enable us to accommodate the growing market demand as well as our clients’ demands”.

Source: https://www.halix.nl/2019/08/09/new-cgmp-facility-starting-operational-production-q4-2019/

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NEW IMI PROJECT “GNA NOW” KICKS OFF ITS BATTLE AGAINST ANTIMICROBIAL RESISTANCE

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Evotec and Lygature announced their cooperation in a new initiative for the development of novel antibacterial agents today: Gram-Negative Antibacterials NOW (“GNA NOW”). The new GNA NOW project, led by Evotec, managed by Lygature and funded by the Innovative Medicines Initiative (IMI), will work on the development of novel antibacterial agents to battle antimicrobial resistance in gram-negative bacteria. The multi-stakeholder consortium includes nine other partners from academia, industry and SMEs: Nosopharm, BIOASTER, Helmholtz Centre for Infection Research, North Bristol National Health Service Trust, University of Liverpool, Inserm, Erasmus Medical Center, Medical University of Vienna, and Fraunhofer IME.

Collectively, the GNA NOW members will progress three programmes in parallel with the goal of bringing one through completion of Phase I studies and one reaching Investigational New Drug (IND) stage and/or up to two programmes reaching clinical development candidate stage, by 2024.

GNA NOW is supported by the IMI, a joint initiative between the European Commission and the European Federation of Pharmaceutical Industries and Associations (EFPIA), of which Evotec is a member. The IMI, the world’s largest public-private partnership (PPP) in life sciences, will match Evotec’s in-kind contribution with a € 12 m grant over the next six years, to fund the activities of the consortium. This award will allow the eleven partners of this consortium to build European platforms of excellence around each step of the critical path for drug discovery and development. European experts will join forces to contribute to “mechanism of action elucidation”, “medicinal chemistry and design”, “in vitro profiling”, “pre-candidate efficacy studies”, “candidate PK/PD studies”, “safety and ADME”, “CMC”, as well as “clinical studies and modelling”.

Dr Cord Dohrmann, Chief Scientific Officer of Evotec, commented: “We are extremely glad to initiate GNA NOW with the backing of the European Commission and the EFPIA. Bacterial infections are a growing threat around the globe and are driving the need for innovative therapeutics with new mechanisms of action. GNA NOW gives us the opportunity to join forces with leading institutions of both the public and the private sector across Europe to develop new gram-negative antibacterial agents as quickly and as efficiently as possible.”

Dr Kristina Orrling, program manager at Lygature and GNA NOW project coordinator, commented: “By joining forces we can fend off a grim future where a simple urinary infection can be fatal. Together, we’ll strengthen the antibacterial arsenal.”

About antibiotic-resistant bacteria and GNA NOW Antibiotic-resistant bacteria were estimated to be responsible for 670,000 infections and 33,110 attributable deaths in the EU and the European Economic Area (EEA) in 2015. From a global perspective, antimicrobial resistance could kill up to ten million people every year by 2050, which could cost up to € 94 trillion ($ 100 tn). In February 2017, the WHO published a list of priority pathogens for the development of new antibiotics. Carbapenem-resistant gram-negative bacteria (Enterobacteriaceae, Pseudomonas aeruginosa, Acinetobacter baumannii) were at the top of that list, with critical priority.

GNA NOW is an Evotec-led joint initiative of eleven partners, project managed by Lygature, with the goal of developing novel antibacterial agents and bringing one of the three simultaneously developed compounds through completion of Phase I studies plus one compound reaching Investigational New Drug (IND) stage and/or up to two compounds reaching clinical development candidate stage, by 2024.

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement No 853979. The JU receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.

Source: Lygature


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Enhanced Bone Regeneration Properties of Osteo-Pharma’s OsteoActivator coated collagen membranes

Osteo-Pharma, a Dutch Life Sciences company developing novel pharmaceuticals and medical devices to improve the local healing of bone fractures and defects, today has announced that it has successfully completed a large preclinical study in mini-pigs, which shows its OsteoActivator coated collagen membranes to effectively promote new bone formation. The data obtained indicated a 24% increase of new bone within 6 weeks in defects covered by an OsteoActivator membranes as compared to uncoated collagen membranes. Importantly, OsteoActivator membranes were shown to be well tolerated and there were no findings of inflammation.

“We are very encouraged by the results of this clinically relevant mini-pig study that was performed in collaboration with the group of Prof. John Jansen from the Radboud university medical center (Nijmegen, The Netherlands) and are a good basis to advance into clinical studies in 2020” said Jan Gossen, chief executive officer of Osteo-Pharma.

Source: Osteo-Pharma

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Johnson & Johnson Establishes International Consortium to End Tuberculosis

Multi-partner collaboration aims to advance the discovery and development of critically-needed innovative TB antibiotics

As part of the Company’s 10-year initiative to drive progress against tuberculosis (TB), Johnson & Johnson today announced that it has launched an international research consortium to discover and develop new TB antibiotics in collaboration with eight European academic and biotechnology partners. Co-funded by Europe’s Innovative Medicines Initiative (IMI) and Janssen Pharmaceutica N.V., part of the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen), the initiative—called RESPIRI-TB—is the first in a series of new collaborations that Johnson & Johnson is undertaking to advance TB research and development.

TB is the world’s deadliest infectious disease, killing an estimated 1.6 million people in 2017 alone.[i] Current TB treatment regimens have suboptimal cure rates because they are lengthy, complex and difficult for patients to adhere to, and growing resistance to first-line treatments is compounding this challenge. Drug-resistant TB (DR-TB) is the single biggest contributor to the rising global health challenge of antimicrobial resistance (AMR).

“Over the past half century, just two new TB medicines have been developed. To achieve the ambitious global goal of ending TB, we urgently need many more innovative therapies,” said Paul Stoffels, M.D., Vice Chairman of the Executive Committee and Chief Scientific Officer, Johnson & Johnson. “No single company or institution holds all the answers.  As we did in the case of HIV, we need to work together to advance the very best science as quickly as possible. We are proud to be a founding partner in the RESPIRI-TB consortium, and we look forward to working with our global partners to develop the necessary TB treatments of the future.”

The ultimate goal of the RESPIRI-TB collaboration is to help enable the development of a new, more efficient combination drug regimen to cure multidrug-resistant TB (MDR-TB), with a focus on shortening treatment duration and minimizing the likelihood of resistance. To this end, the partners will work to advance the discovery and early-stage development of new compounds from different drug classes that could be used – in combination with each other or with existing therapies, such as Janssen’s bedaquiline. Bedaquiline was the first novel TB medicine to be approved in more than 40 years and, today, is one of the last lines of defense against MDR-TB.

The new consortium includes nine research teams spanning five European countries with expertise in mycobacterial diseases and drug target investigation, as well as project management. In addition to Janssen, members include: Leiden University Medical Center and the University of Leiden (Netherlands, which will serve as the project coordinator); University of Antwerp, Belgium; Sorbonne University, France; Medical University of Vienna, Austria; University of Copenhagen, Denmark; Mitologics, France; and FFUND B.V., Netherlands.

In addition to RESPIRI-TB, the research consortium will also work to build a pipeline of drug candidates for the non-TB mycobacterial (NTM) species M. avium and M. abscessus through a second project, RESPIRI-NTM. Although NTM infections are less prevalent than TB, currently available treatments for NTM infections are suboptimal, underscoring the urgent need for new therapeutic options.

“Tuberculosis and related diseases represent a major threat to public health worldwide, and a collaborative approach is essential if we are to develop new, safe and effective treatments,” said Pierre Meulien, Executive Director, IMI. “I am delighted that the Janssen Pharmaceutical Companies of Johnson & Johnson and its partners in RESPIRI-TB and RESPIRI-NTM have come together to form the type of strategic, multi-sector alliances we need to boost the world’s response to the threat of these serious infectious diseases.”

IMI is supporting the development of a number of multi-partner research consortia under its AMR Accelerator program that aim to speed TB drug development across the R&D spectrum – from discovery/early-stage development (i.e., RESPIRI-TB) to later-stage translational development – with the overarching goal of dramatically improving TB treatment regimens.

Together, these and other company-specific and collaborative R&D efforts will help drive critical and necessary TB innovation. This is in line with the current draft of the World Health Organization’s Global Strategy for TB Research and Innovation, which is currently undergoing review and is expected to be finalized for adoption by the World Health Assembly in May 2020.

[i] World Health Organization. Global Health Observatory (GHO) data. Tuberculosis. Last accessed June 2019. Available at: https://www.who.int/gho/tb/en/

Source: J&J pressrelease

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TPI Bidbook: sneller en beter met proefdiervrije innovaties

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TPI is op zoek naar Nederlandse bedrijven die hun proefdiervrije innovaties willen publiceren in een bidbook: een mooie kans om je bedrijf in de kijker te spelen! TPI staat voor Transitie Proefdiervrije Innovatie. Deze transitie is gaande: wetenschap, gezondheidszorg, beoordeling van veiligheid en acceptatie van dierproeven veranderen. TPI staat ook voor de groep die deze ontwikkelingen wil versnellen: RIVM, NFU, VSNU, ZonMw, Samenwerkende Gezondheidsfondsen, Stichting Proefdiervrij en de ministeries van LNV, VWS, EZK, OCW en IenM. Het TPI-netwerk signaleert dat er behoefte is meer te zien van wat start en scale ups allemaal ontwikkelen zonder proefdiergebruik.

TPI nodigt leveranciers van proefdiervrije innovaties daarom uit om zich in het TPI bidbook te profileren. Het doel is om met zo’n bidbook overzichtelijk te laten zien wat en waarom Nederlandse start en scale ups proefdiervrij innoveren. Het bidbook wordt gepresenteerd tijdens de International pioneer2policymaker conference ‘Accelerating animalfree innovations’ van 27-29 november 2019.

Wil je dat TPI jouw bedrijfsprofiel in het bidbook opneemt? Laat het HollandBIO dan uiterlijk 23 juli weten via info@hollandbio.nl.  

Meer informatie:

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Drempels cel- en gentherapie in de media

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De beslissing van het Nederlandse bedrijf Orca Therapeutics om uit te wijken naar Canada onderstreept de urgentie om de nationale drempels rondom klinisch onderzoek naar cel- en gentherapie weg te nemen. Afgelopen week besteedden het Financieele Dagblad en BNR Nieuwsradio aandacht aan het probleem en de oproep van de Tweede Kamer aan het kabinet om met spoed knelpunten weg te nemen. Eerder deelden we al onze blijdschap over de politieke steun.

In de krant en op de radio vertelt Orca Therapeutics CEO Kees Groen dat het vergunningsverleningstraject voor klinisch onderzoek naar gentherapie tegen prostaatkanker in Canada slechts een maand duurde. Dit in schril contrast met het jaar dat Nederland gemiddeld uittrekt voor een dergelijk traject. Naast Kees geven ook VVD-Kamerlid Arne Weverling en HollandBIO’s Annemiek een toelichting op de problematiek en de hopelijk spoedige oplossing.

Het volledige FD-artikel lees je hier (inlog nodig).

De bijdrage op BNR van Kees Groen luister je hier terug, de bijdrage van Annemiek Verkamman hier.

Ons bericht van vorige week vind je hier.

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Ncardia Expands iPSC Manufacturing and Phenotypic Drug Discovery Services

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Ncardia expands its iPSC manufacturing site in Belgium, R&D and drug discovery center in the Netherlands, and commercial operations in the USA. The accelerated investment followed the retraction of the Cor.4U product line from the market and resulting insolvency of its former legal entity in Germany(operating under the name Ncardia AG / former Axiogenesis).

Ncardia shared today that all active investors participated in a capital increase, signaling confidence in its expanded offerings in iPSC drug screening services, contract cell manufacturing and iPSC-based cardiovascular and neurological cell models. Under the restructuring following the closing of Ncardia AG operations in Germany, all manufacturing services have been concentrated at its ISO 9001-certified contract and product manufacturing facility in Gosselies, Belgium and activities in drug discovery / safety services continue to operate from its Leiden, Netherlands headquarters.

“We continue our dedication to the advancement of iPSC technologies in the drug discovery space and are excited that with the support of our investors, we can continue providing quality iPSC-derived products and accelerate our capabilities in contract cell manufacturing and drug discovery assay development,” said Stefan Braam, CEO of Ncardia.

Source: Ncardia

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Subsidieoproep Create2Solve

Create2Solve is een oproep binnen het ZonMw programma Meer Kennis met Minder Dieren. Met Create2Solve ondersteunt ZonMw de ontwikkeling van impactvolle, proefdiervrije innovaties die moeten leiden tot verkoopbare methoden, modellen en/of diensten. Deze proefdiervrije innovaties verbeteren de gezondheid van de mens, de kwaliteit en relevantie van onderzoek en bedrijfsprocessen.

Lees meer

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