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Spread the word: HollandBIO zoekt communicatieadviseur!

Net als de biotech sector, groeit ook HollandBIO alsmaar verder door. En om al die bedrijvigheid rondom biotech innovaties nog steviger op de kaart te zetten, gaan we onze communicatie uitbreiden. Daarom zijn we ter versterking van ons team op zoek naar een communicatieadviseur die HollandBIO’s communicatie naar het volgende niveau helpt tillen. Ben of ken jij die nieuwe collega die samen met een superleuk team en onze leden wil bijdragen aan een meer duurzame, gezonde wereld? Spread the word!

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Ferrer and Treeway enter into licence agreement for the development and commercialisation of TW001/FNP122 for Amyotrophic Lateral Sclerosis

Ferrer, an international pharmaceutical company focused on pulmonary vascular and interstitial lung diseases and neurological disorders, and Treeway, a clinical-stage biotechnology company, announced they have entered into a licence agreement for the development and commercialisation of an oral formulation of edaravone (TW001/FNP122)[i] for Amyotrophic Lateral Sclerosis (ALS) in certain territories, including Europe and some Asian countries. Under the terms of the licence agreement, Ferrer is responsible for advancing the development and commercialisation of the product and will make royalty payments to Treeway at certain milestones.

The deal leverages Ferrer’s strong capabilities and expertise in neurology to underscore both companies’ commitment to expanding global access to TW001/FNP122 for the treatment of ALS. 

Inez de Greef, CEO at Treeway comments: “Guided by patients, we have developed TW001/FNP122 through the early stages of development. We are therefore delighted that Ferrer has committed to advance our lead product to the required pivotal phase III trial and make it available for ALS patients in large parts of the world.

Oscar Pérez, Ferrer’s Chief BD&L Officer, adds: “Driven by our purpose of bringing transformative products to patients over the world, we are pleased to enter the agreement with Treeway and look forward to patients benefiting from improved treatment options. TW001/FNP122 is the perfect fit for Ferrer’s strategy to bring new molecules to late-stage clinical development, registration and market entry in the field of neurological disorders and strengthen our pipeline for the coming years.” 

The agreement comes into play with immediate effect.

Source: Press release (Treeway)

Read more (In Dutch):

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Osteo-Pharma receives MREC approval for initiation of clinical trial

Osteo-Pharma today announced that it has successfully obtained MREC approval for testing their lead product, OsteoActivator-P, in patients. OsteoActivator-P is a novel coated porcine collagen barrier membrane that will be placed on a dental defect to improve early bone formation. The study is a randomized prospective clinical trial, comparing OsteoActivator-P coated membranes vs Collagen Membrane-P uncoated for accelerated localized alveolar ridge preservation and will investigate the efficacy and safety of OsteoActivator-P. The study is expected to enroll a total of 16 patients at 2 clinical centers in The Netherlands.

“OsteoActivator-P is a new and promising innovative product for the dental market for all applications requiring guided bone regeneration. Preclinical data have provided a strong rationale for this first clinical trial and a robust study protocol is now available to address the efficacy and safety profile of OsteoActivator-P” said Jan Gossen, CEO of Osteo-Pharma.

Source: Osteo-Pharma (Press release)

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Leestip: kosteneffectiviteitsanalyse van behandeling voor SMA

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Zeldzame aandoeningen worden gelukkig steeds beter behandelbaar. Elke dag werken onderzoekers keihard aan het ontwikkelen van nieuwe geneesmiddelen. Een van de nieuwste toevoegingen aan het arsenaal is een gentherapie voor de behandeling van spinale musculaire atrofie (SMA), een erfelijke neurologische aandoening. Op het moment buigt het Zorginstituut zich over de vraag of deze gentherapie ook in Nederland vergoed zou moeten worden. Toen HollandBIO’s Thomas zijn master Epidemiologie aan het afronden was heeft hij een kosteneffectiviteitsanalyse uitgevoerd over de behandeling van SMA met deze nieuwe gentherapie. Het wetenschappelijke artikel is vorige week gepubliceerd in Value in Health. Interesse? Lees hier het artikel

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Patented ‘molecular heaters’ to increase crop yield

Researchers at the University of Amsterdam have developed a novel class of molecules capable of raising the temperature of plants. Application of such ‘molecular heaters’ allows crops to grow at lower environmental temperatures. This can extend the growth season, increase the availability of arable land, and facilitate speed breeding programs for new crop varieties.

The molecular heaters were developed by Prof. Wybren Jan Buma of Molecular Photonics (Van ’t Hoff Institute for Molecular Sciences) and Dr Teun Munnik of Plant Cell Biology (Swammerdam Institute for Life Sciences) in a cooperation with researchers from the universities of Warwick and Bristol.

In experiments in the laboratory and greenhouses, the team has demonstrated that the molecular heating effect can indeed be used to warm plants and increase the temperature of leaves by more than one degree Celsius. Furthermore, it was established that application of these molecular heaters can lead to increased amounts of biomass. Currently, the molecular heaters are subject of further study in the European ‘Boostcrop’ project, funded with nearly 5 million euros from the EU’s ‘Future and Emerging Technologies’ programme. This research includes small field tests and assessment of safety issues.

When applied at large scale, molecular heaters could contribute to boosting crop yields worldwide. Allowing crops to grow at lower environmental temperatures can extend their cultivation at higher altitudes or latitudes than what is normally possible. Application of the molecular heaters can also prolong the growth season (to earlier and later time periods) and lead to faster development of plants.

After having recently been awarded a patent, the researchers are now looking for partners to further develop feasible applications of the molecular heaters. Preliminary contacts with key players have already shown a high level of interest in the field of crop protection and development. The researchers envision large-scale application of the molecular heaters in mixtures with surfactants and wetting agents or combinations thereof. This would optimise their application on plants, for instance through spraying techniques. Another line of development they pursue is to chemically adjust the molecular heaters to make them stick better to the surface of plants.

The way molecular heaters work is comparable to molecules used in sunscreen lotions. They absorb UV photons and transform this energy into molecular vibrations. This effectively converts harmful light into harmless heat, albeit on such a small scale that this escapes the sunbather’s notion. Having developed advanced laser spectroscopic techniques for elucidating the photodynamics of these sunscreen molecules, Wybren Jan Buma set out to broaden the scope of his research. He teamed up with plant biologist Teun Munnik to explore the idea of using the molecular heating phenomenon for heating plants. When it turned out this notion had not been reported in literature before, they knew they were on to something.

Buma and Munnik decided to focus on naturally occurring types of molecules. In fact, plants produce molecules such as sinapoyl malate to protect themselves against UV radiation from the sun in a sunscreen-like fashion. The researchers studied different variants of these type of molecules to optimize their heat-generating effect.

Source: UvA (Press release)

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Cergentis appoints new CEO and Board members

Cergentis, a genomics company that develops and commercializes kits and services based on its proprietary Targeted Locus Amplification (TLA) technology, today announced the appointment of Joris Schuurmans as Chief Executive Officer and Dirk Pollet and Maja Sanders as Supervisory Board members. Novalis Biotech Incubation will invest in Cergentis’ growth potential.

Incoming CEO Joris Schuurmans has extensive experience in the molecular diagnostics and biopharmaceutical industry. He held senior executive positions at Biocartis and MyCartis, and recently lead SkylineDx’ melanoma program prior to transitioning to his role at Cergentis.

Joris Schuurmans, newly appointed CEO of Cergentis, commented: “I am very excited by the opportunity to join Cergentis at this pivotal stage. The company has developed an impressive array of products and services in the field of genetic engineering, and is extremely well-positioned to enter the molecular diagnostics field with products that elucidate DNA variants in tumors that cannot be well-analyzed with current technologies. We are all excited to embark on our mission to improve the diagnosis and treatment of cancer patients.”

Dr. Jan Groen, Chairman of the Board: “A heartfelt gratitude to outgoing CEO Jan Dekker, who laid the foundation for Cergentis to take the next step to accelerate the development and commercialization of its unique TLA technology for cancer diagnostics. We are pleased to welcome Joris as new CEO. Joris has an excellent international track record in the biotech industry. We are also very pleased with the appointment of our two new Board members Maja and Dirk. Both are industry veterans and experienced Board members.”

Dr. Dirk Pollet is currently CEO of Enzyre, and heralded the acquisition of Multiplicom by Agilent as the CEO of Multiplicom. Maja Sanders currently holds supervisory roles at various non-profit and for-profit organizations, and has built up extensive managerial experience in the private equity industry.

Dr. Jan Groen, Chairman of the Board: “Dirk and Maja are valuable additions to the Board and we are pleased they join us in these exciting times. As Cergentis has already established a strong position it the support of drug development, we are looking forward to working together to support the company in further expansion in the cancer diagnostics field.”

Novalis Biotech Incubation to invest in Cergentis

Novalis Biotech Incubation (www.novalis.is), a Belgium venture capital investment fund, is investing in Cergentis.

Wim Van Criekinge, Co-founder of Novalis, and Board member of Cergentis added: “We are pleased to invest in Cergentis. Their proprietary Target Locus Amplification (TLA) technology is unique and allows the most advanced genome characterization to date”.

Joris Schuurmans, CEO of Cergentis: “We are delighted that Novalis has decided to invest in Cergentis. We appreciate their trust in our company and their support in our mission to improve the quality of genetic research and human healthcare worldwide.”

Source: Cergentis (press release)

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uniQure Announces License Agreement with CSL Behring to Commercialize Hemophilia B Gene Therapy

uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that uniQure and CSL Behring have entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene dezaparvovec, uniQure’s investigational gene therapy for patients with hemophilia B. Etranacogene dezaparvovec consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). Under the terms of the agreement, uniQure will receive a $450 million upfront cash payment and be eligible to receive up to $1.6 billion in payments based on regulatory and commercial milestones. uniQure will also be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percentage of net product sales arising from the collaboration.

The collaboration leverages CSL Behring’s strong global reach and commercial infrastructure in hematology to accelerate access of etranacogene dezaparvovec to hemophilia B patients around the world.

“We are thrilled to enter into this commercialization and license agreement with CSL Behring, an ideal commercial partner with global reach and decades of expertise in hemophilia,” stated Matt Kapusta, chief executive officer of uniQure. “We believe that through this arrangement, we are ideally positioned to deliver globally our innovative gene therapy to the largest number of hemophilia B patients as quickly as possible.” 

“The transaction represents a major milestone in the development of etranacogene dezaparvovec and, when closed, we expect that it will provide uniQure with significant financial resources to advance and expand our pipeline of gene therapy candidates, anchored by AMT-130 in Huntington’s disease, and to invest further in our leading gene therapy manufacturing and technology platform to support pipeline growth,” he added.

As a CSL Limited company, CSL Behring is a global biotherapeutics leader delivering lifesaving medicines to patients with rare and serious diseases. A global leader in treating bleeding disorders, CSL Behring has been delivering innovations for the hemophilia patient community for more than 30 years. The company reported more than $1 billion in sales of hemophilia-related medicines in 2019.

“Our vision with hemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease,” said CSL’s CEO and Managing Director Paul Perreault. “With more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximize the potential benefit of this therapy. Upon approval, we believe this next-generation therapy will be highly complementary to our existing best-in-class hemophilia B product portfolio with an alternate best-in-class treatment option.”

Under the terms of the agreement, uniQure will be responsible for the completion of the HOPE-B pivotal study, manufacturing process validation, and the manufacturing supply of etranacogene dezaparvovec until such time that these capabilities are transferred to CSL Behring. Clinical development and regulatory activities performed by uniQure under the agreement will be reimbursed by CSL Behring. CSL Behring will be responsible for regulatory submissions and commercialization of etranacogene dezaparvovec.

The closing of the transaction is contingent on completion of review under antitrust laws in the United States, Australia and the United Kingdom. 

Accelerate Build-out of Innovative Gene Therapy Pipeline and Platform

uniQure expects that the agreement will provide additional capital to significantly accelerate and expand its pipeline of innovative gene therapies, including advancing the Phase I/II study of AMT-130 in Huntington’s disease, initiating IND-enabling studies of AMT-150 in spinocerebellar ataxia type 3, selecting a lead candidate in Fabry disease and progressing other current and new candidates for central nervous system disorders and rare liver-directed diseases. Regarding AMT-130, uniQure recently announced the successful completion of the first two patient procedures in the Phase I/II study and anticipates announcing early safety data in the second half of 2020 and initial efficacy data in 2021.

uniQure plans to continue to leverage its leading gene therapy platform, including the Company’s deep expertise with AAV5, to develop potentially best-in-class gene therapies. AAV5-based gene therapies have been demonstrated to be safe and well tolerated in a multitude of clinical trials, including uniQure trials conducted in hemophilia B and other indications. No patient treated in clinical trials with uniQure’s AAV5 gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be viable treatments in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment.  uniQure also may seek to in-license or acquire additional product candidates that align with its research and development strategy. 

In addition, uniQure plans to further strengthen its proprietary gene therapy platform by expanding its manufacturing capacity to support a broad pipeline, including product candidates for diseases with larger prevalence, as well as investing further in new technologies to improve the efficacy, safety and applicability of its gene therapies to patients.

As part of uniQure’s effort to focus on those gene therapy programs that have the greatest potential to improve patients’ lives and generate long-term value for shareholders, uniQure plans to de-prioritize its research program of AMT-180 for patients with hemophilia A.   

Moelis & Company acted as a financial advisor to uniQure in this transaction.

Source: uniQure (press release)

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AveXis receives EC approval and activates “Day One” access program for Zolgensma

AveXis, a Novartis company, today announced the European Commission (EC) granted conditional approval for Zolgensma® (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene. The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.

In Europe each year, approximately 550–600 infants are born with SMA, a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.1,2,3 Zolgensma is a one-time gene therapy designed to address the genetic root cause of the disease by replacing the function of the missing or nonworking SMN1 gene. Administered during a single, intravenous (IV) infusion, Zolgensma delivers a new working copy of the SMN1 gene into a patient’s cells, halting disease progression. According to Pediatric Neuromuscular Clinical Research (PNCR) natural history study of SMA, almost all patients under the age of five years of age will be under 21kg with some patients at 6, 7 or 8 weighing below 21 kg.4 AveXis is planning a product presentation that allows for treatment of patients weighing up to 21 kg and is working with the European Medicines Agency (EMA) to finalize supply timelines. 

“The EC approval of Zolgensma is a significant milestone for the SMA community, and further underscores the substantial clinical value of the only gene therapy for SMA, bringing new hope to those impacted by this rare, but devastating disease.” said Dave Lennon, president of AveXis. “Even under the current pandemic conditions, the urgent need to treat SMA has resulted in access pathways in France and Germany for Zolgensma, a potentially life-saving medicine delivered in a single dose. Additionally, we have met with more than 100 stakeholder organizations across Europe to discuss our “Day One” access program to enable rapid access with customizable options designed to work within local pricing and reimbursement frameworks.”

SMA is a significant burden to the healthcare system in Europe with cumulative estimated healthcare costs per child ranging between €2.5 to €4 million within the first 10 years alone.Zolgensma is a transformative and highly innovative one-time gene therapy for a devastating and progressive genetic disease and is consistently priced worldwide under a value-based framework, however final pricing and reimbursement decisions are determined at the local level. Designed to work within existing, local pricing and reimbursement frameworks, the “Day One” access program offers ministries of health and reimbursement bodies a variety of flexible options that can be implemented immediately to support swift access and broad reimbursement.

The “Day One” access program ensures the cost of patients treated before national pricing and reimbursement agreements are in place align with the value-based prices negotiated following clinical and economic assessments. The program maintains the integrity of the local pricing and reimbursement frameworks with a variety of customizable options including:

  • Retroactive rebates ensuring early access costs are aligned with negotiated prices following local clinical and economic assessment processes
  • Deferred payments and installment options allowing reimbursement bodies to manage budget impact during the early access phase
  • Outcomes-based rebates negotiated following clinical and economic assessments can be applied to patients treated during the early access period
  • Robust training for treating institutions on administration and follow-up care
  • Access to RESTORE, a global registry of patients who have been diagnosed with SMA that draws upon existing country registries

Immediate access to Zolgensma, aligned to the label, is available in France through the ATU framework and expected shortly in Germany.

“Today’s approval brings tangible progress in harnessing the transformational power of gene therapy,” said Dr. Eugenio Mercuri, Professor, Pediatric Neurology, Catholic University, Rome, Italy. “The approval of Zolgensma represents an important new way for physicians to treat patients with SMA. The results we have seen for Zolgensma to date from the STR1VE clinical trial show an impressive survival rate at the conclusion of the study, with the majority of patients achieving functional milestones, like sitting without support, that wouldn’t have been reached in untreated infants.”

“SMA Europe receives with deep excitement the news on the approval by the European Commission, of a gene therapy for treating a part of our community,” said Mencía de Lemus, President of SMA Europe. “Many hopes have been put into this much awaited therapy. It will be now be up to all stakeholders involved to ensure that treating doctors, together with parents, can take the best therapeutic option based on the benefit that each of them can provide to each individual. Gathering more data on how Zolgensma impacts in the lives of patients will be extremely important to better understand the potential of this new therapy on improving lives of those living with SMA.”

The EC approval is based on the completed Phase 3 STR1VE-US and Phase 1 START trials that evaluated the efficacy and safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 patients <6 months of age at dosing, who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively. STR1VE-EU, a comparable Phase 3 study is ongoing. Zolgensma demonstrated prolonged event-free survival; rapid motor function improvement, often within one month of dosing; and, sustained milestone achievement, including the ability to sit without support, crawl and walk independently – milestones never achieved in untreated Type 1 patients.6

Additional supportive data included interim results from the ongoing SPR1NT trial, a Phase 3, open-label, single-arm study of a single, one-time IV infusion of Zolgensma in pre-symptomatic patients (<6 weeks at age of dosing) genetically defined by bi-allelic deletion of SMN1 with 2 or 3 copies of SMN2. These data demonstrate rapid, age appropriate major milestone gain, reinforcing the critical importance of early intervention in SMA patients.5

The most commonly observed side effects after treatment were elevated liver enzymes and vomiting. Acute serious liver injury and elevated aminotransferases can occur. Patients with pre-existing liver impairment may be at higher risk. Prior to infusion, physicians should assess liver function of all patients by clinical examination and laboratory testing. And, they should administer systemic corticosteroid to all patients before and after treatment, and then continue to monitor liver function for at least 3 months after infusion.There is limited experience in patients 2 years of age and older or with body weight above 13.5 kg. The safety and efficacy of Zolgensma in these patients have not been established.

AveXis has an exclusive, worldwide license with Nationwide Children’s Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Généthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

Source: Avexis (press release)

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Nieuw: biotech vraag & aanbod op de Member-to-Member Marketplace


Biotech ondernemers besteden in de regel veel werk uit bij hooggespecialiseerde dienstverleners. En dat is maar goed ook, want zo kunnen zij zich volledig toeleggen op de ontwikkeling van hun eigen bedrijf. Er is een breed aanbod van dienstverleners: van alle mogelijke vormen van contract research, juridisch advies, experts in intellectueel eigendom of personeelszaken en van financieel management tot communicatie. Met de Member-to-Member Marketplace biedt HollandBIO inzicht in het dienstenaanbod en profiteren onze leden van aantrekkelijke aanbiedingen. Bekijk het groeiende aanbod hier.

Ben je lid van HollandBIO en wil jij ook een aanbieding doen? Download dit formulier in en stuur het ingevuld retour naar bs@hollandbio.nl.

In de schijnwerper

HollandBIO leden hebben een streepje voor: alleen leden kunnen aanbiedingen doen op de Member-to-Member Marketplace. Door een handige zoekfilter komt jouw bedrijf snel naar voren. Op het platform krijg je niet alleen de kans om een aantrekkelijke aanbieding te doen, ook is er ruimte om meer te vertellen over je bedrijf. En, de beste aanbiedingen maken kans op een extra promotieplek in de HollandBIO Weekly.

De spelregels

  • Alleen leden van HollandBIO kunnen een aanbieding doen. Geen lid? Word hier lid!
  • De aanbieder is eigenaar van de aanbieding. Dit betekent dat je zelf verantwoordelijk bent voor het aanleveren van de teksten en voor het actueel houden van de aanbieding.
  • Hier kan je controleren of de aanvrager ook lid is van HollandBIO, of stuur ons een mailtje
  • Teksten kunnen worden aangeleverd in het Nederlands of in het Engels.
  • HollandBIO kan niet garanderen dat leden gebruik zullen maken van het aanbod.

Vragen? Neem contact op met HollandBIO’s Leonie

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Life Science industry insights from Aon’s 2019 Global Risk Management Survey

The Life Science industry is an extremely dynamic, R&D-focused, heavily regulated industry that is confronted with more risks than ever before. Considering this backdrop, it is troubling that many organizations participating in the 2019 Global Risk Management Survey stated that they may be less prepared to manage or mitigate their most critical risks.

Aon’s 2019 Global Risk Management Survey, the seventh of its kind since 2007, is designed to offer organizations the insights necessary to enable better management of risk related volatility and compete in an increasingly complex business environment. The survey gathered input from 60+ Life Science industry respondents across geographies, covering biotechnology, pharmaceutical, medical devices and other industry sub-sectors.

This Life Science industry specific report highlights key insights from the survey findings and provides an interpretation, validation and viewpoint from Aon’s industry experts. It is essential reading for any entity as they evolve their risk register and refine their risk management strategy.

Download the Life Science industry highlights from the 2019 Global Risk Management Survey here.

If you have any questions or comments about the Life Science insights or wish to discuss the results, please contact Perry Steenvoorden