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Vergunningverlening voor klinisch onderzoek cel- en gentherapie nog verder verbeterd

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De vergunningaanvraag voor klinisch onderzoek met cel- en gentherapie wordt nog sneller en beter. Het RIVM en Bureau GGO zetten in op een lerend systeem waarmee zij in samenwerking met veldpartijen tot zo veel mogelijk nieuwe generieke milieurisicobeoordelingen (MRB’s) komen. Hierdoor kan de tijd die nodig is om een vergunning te verlenen niet alleen binnen de wettelijke termijn van maximaal 56 dagen plaatsvinden, maar in de praktijk zelfs binnen 28 dagen. Dit blijkt uit de nieuwste Kamerbrief hierover van IenW-minister Van Nieuwenhuizen. De standaardisering is gereed zodra de eerder aangekondigde regelgeving gewijzigd is. HollandBIO is blij dat de minister in haar brief aangeeft dat Nederland een prachtige, innovatieve biotech sector heeft en dat zij met deze maatregelen nog een schep bovenop eerdere verbeteringen doet!

Daarnaast gaat de minister in op het voorstel van de Europese Commissie voor een spoedverordening die klinisch onderzoek in heel Europa naar coronavaccins- en behandelingen makkelijker moet maken. Nederland blijft voorstander van een milieurisicobeoordeling, maar zal het voorstel steunen als blijkt dat het voorstel de enige mogelijkheid is. Wel neemt de minister de mogelijkheid in beraad die de voorgestelde noodverordening biedt aan overheden om eventuele negatieve effecten te minimaliseren voor de veiligheid van mens en milieu. Ook benadrukt ze dat het afwijken van de geldende regels voor milieuveiligheid enkel gerechtvaardigd is voor deze uitzonderlijke situatie en enkel voor een beperkte periode.

HollandBIO zet zich ervoor in dat deze verbeteringen spoedig doorgang vinden, liever vandaag dan morgen. De ontwikkelingen rond het coronavirus tonen hoe urgent en belangrijk het is om zo snel mogelijk met klinisch onderzoek te kunnen beginnen, maar er zijn meer medische toepassingen waarvoor de administratieve lasten verlaagd zouden moeten worden. Gelukkig blijkt uit de eerste ervaringen dat het verbeterplan voor medische ggo’s een verkorting van de doorlooptijden en vermindering van de uitvoeringslasten oplevert. Bovenal hebben er sindsdien geen termijnoverschrijdingen meer plaatsgevonden. Hieruit proeft HollandBIO dat Nederland op het juiste spoor zit en hopelijk de smaak te pakken heeft!

Drastische versnelling vergunningaanvraag medische GGO’s, nu door voor de kopgroep
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Kiadis announces first patient enrolled with off-the-shelf K-NK cells

Kiadis Pharma, a clinical stage biopharmaceutical company, and The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital (OSUCCC-James), today announced that the first patient has been enrolled and treated in a phase I, first-in-human clinical trial in patients with relapsed/refractory acute myeloid leukemia (“R/R AML”) with off-the-shelf Natural Killer (“NK”) cells manufactured at the Cell Therapy laboratory at the OSUCCC-James using Kiadis’ FC21 and proprietary universal donor platforms. The trial is being conducted at the OSUCCC-James and is expected to provide valuable data to support Kiadis’ K-NK003 development program.

The phase I study, NCT04220684, will evaluate the NK cell product in up to 56 patients, ages 18 – 80 who have primary refractory AML, relapsed AML, or myelodysplastic syndromes (“MDS”). The goal of this study is to establish safety of the NK cell therapy for the induction of remission in patients with R/R AML or MDS and to determine the optimal dosing and overall response rate. Patients enrolled in the study will receive six doses of NK cells of 1 x 107 cells/kg to 1 x 108 cells/kg after receiving reinduction chemotherapy. The trial is expected to provide further clinical proof-of-concept of Kiadis’ K-NK003 product. Kiadis is supporting the Investigator-sponsored study through a collaborative research agreement with OSUCCC-James.

Sumithira Vasu, MBBS, the principal investigator of the clinical trial, hematologist and scientist, Medical Director of the Cell Therapy Lab at OSUCCC-James, and associate professor at the Ohio State College of Medicine says,

“This off-the-shelf universal donor NK cell therapy is an exciting new experimental treatment option for patients with R/R AML and MDS that allows us to infuse large numbers of hyperfunctional NK cells immediately when needed. Treating our first patient with this therapy is an important step in this ongoing clinical research.”

Andrew Sandler, MD, chief medical officer of Kiadis commented,

“This trial uses a novel off-the-shelf, readily available NK cell product to treat a very sick and difficult-to-treat group of patients. We are very enthusiastic with the initiation of this trial and to be one step closer to bringing K-NK-cell therapies to a broad patient population across a potentially wide range of cancers.”

Source: Kiadis Pharma (press release)

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Update wijziging Wgp en modernisering GVS

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De herijking van de maximumprijzen op grond van de Wet geneesmiddelenprijzen (Wgp) per 1 oktober 2020 gaat gepaard met twee mitigerende maatregelen, zo laat VWS-minister van Rijn de Kamer weten in een Kamerbrief. Met deze maatregelen beoogt de minister risico’s op beschikbaarheidsproblemen te verkleinen en de geneesmiddelenmarkt, gezien de COVID-19-crisis, niet extra te belasten.

De eerste maatregel houdt in dat de overheid de prijsdaling eenmalig maximeert op 10 procent, waarmee de grootste prijsdalingen worden gedempt. Als tweede maatregel bestaat voor producten met een omzet lager dan € 1 miljoen per jaar in Nederland de mogelijkheid om vrijstelling van de herijking aan te vragen. Daarnaast blijft het mogelijk om in uitzonderlijke gevallen, bijvoorbeeld bij acute beschikbaarheidsproblemen, voor een specifiek middel de maximumprijs aan te passen of zelfs los te laten als de prijs een belemmering vormt voor de inkoop.

In dezelfde brief informeert van Rijn de Kamer over de stand van zaken rondom de modernisering van het geneesmiddelenvergoedingssysteem (GVS), die op 1 januari 2022 in werking moet treden. Op dit moment werkt het ministerie van VWS aan de juridische verankering en de invulling van het begrip ‘medische noodzaak’ voor de GVS-bijbetaling. Aan het eind van dit kalenderjaar volgt meer informatie.

Benieuwd naar de hele Kamerbrief? Je vindt hem hier.

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Access to COVID-19 Tools (ACT) Accelerator publishes consolidated plans

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No-one is safe until everyone is safe. To end the ongoing COVID-pandemic, a groundbreaking global response is required. By drawing on the experience of leading global health organizations, governments, businesses, civil society and philanthropists the Access to COVID-19 Tools (ACT) Accelerator aims to accelerate the development, production, and equitable access to COVID-19 diagnostics, therapeutics, and vaccines. Set up in response to a call from G20 leaders in March, the ACT-Accelerator’s consolidated investment case was published last week. Read more

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Hansa Biopharma receives positive CHMP opinion for Idefirix for kidney transplant in EU

Hansa Biopharma, the leader in immunomodulatory enzyme technology for rare IgG mediated diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending conditional approval of IdefirixTM (imlifidase) for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor. Endorsement of the positive opinion by the European Commission is expected in the third quarter of 2020. 

“We are very excited to receive a positive opinion from the CHMP. This brings hope to the thousands of highly sensitized patients across Europe waiting for a life-saving kidney transplant and takes Hansa Biopharma one important step closer to becoming a commercial stage biopharmaceutical company” says Søren Tulstrup, President and CEO of Hansa Biopharma.

“Today’s decision by the CHMP further serves to validate the potential of Hansa Biopharma’s proprietary drug development engine to develop approvable immunomodulatory drug candidates for rare and serious diseases and comes at a time when we are significantly expanding our activities into autoimmune diseases, gene therapy and oncology”. 

The Marketing Authorization Application for imlifidase in kidney transplant was accepted for review by the European Medicines Agency on Feb. 28, 2019 based on data from four completed phase 2 studies across Sweden, France and the United States. Imlifidase met all primary and secondary endpoints in each study.

Imlifidase was supported through EMA’s PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients’ unmet medical needs. Imlifidase was granted eligibility to PRIME in May 2017.

In the US, following overall agreement with the FDA, Hansa Biopharma submitted a study protocol to the FDA on June 17, 2020. The randomized, controlled clinical study is planned to be initiated in Q4 this year and could support a future BLA submission in the US by 2023, as communicated earlier. The Company aims to recruit 45 highly sensitized patients at 10-15 centers in the US for this study. 

Clinical pipeline 
Enrollment in the investigator initiated Anti-GBM study was completed at the end of January 2020 and the first data read-out is expected in the third quarter of 2020 as previously guided. In the AMR and GBS phase 2 studies, 4 of the targeted 30 patients have been treated with imlifidase in the respective studies. Enrollment in the AMR and GBS studies is expected to be completed in H1 2021 and H2 2021, respectively, as communicated previously.

Source: Hansa Biopharma (press release)

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Scenic Biotech Awarded €3.1 million Innovation Credit

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Scenic Biotech, a pioneer in the discovery of genetic modifiers to enable the development of disease modifying therapeutics for rare genetic disorders and other devastating illnesses, today announced that it has been awarded a €3.1 million Innovation Credit from the Dutch Government. The funds will be used to advance the Company’s lead CD47/QPCTL immuno-oncology program through preclinical development towards human clinical studies.

The Innovation Credit is awarded by the Dutch government through its agency RVO of the Ministry of Economic Affairs and Climate Policy and is aimed at the development of promising and challenging innovations. It will support the preclinical development of novel QPCTL (Glutaminyl-peptide cyclotransferase-like) inhibitors, completing the two-year project with an IND filing.

Scenic’s CD47/QPCTL immuno-oncology program builds on seminal work by the Netherlands Cancer Institute (NKI) and Leiden University Medical Centre (LUMC). An enzyme present in cancer cells, QPTCL was first demonstrated as a promising target for immuno-oncology by using the Company’s proprietary high-resolution genetics platform called Cell-Seq. The results of this discovery were published in the journal Nature Medicine.

QPCTL was found to be a druggable modifier of the CD47 innate immune checkpoint, which is one of the major mechanisms by which cancer cells evade detection by the immune system. As a result of this activity, CD47 is also known as the ‘don’t eat me signal’. After being the first to discover and validate QPCTL as a promising target in immuno-oncology, NKI and LUMCs scientists also showed that small molecule inhibitors of QPCTL can prevent the expression of functional CD47 on cancer cells, thereby causing the cancer cells to be attacked by macrophages and destroyed.

Scenic has gone on to develop a series of chemical inhibitors with potent inhibition against QPCTL and has filed a patent application related to this chemical series.

Dr Sebastian Nijman, co-founder and CEO of Scenic Biotech said:
“We are delighted to be selected for this award from the Dutch Government, recognizing the potential of our program to develop a new cancer therapy. The funding will enable us to accelerate the optimization of our proprietary small molecule QPCTL inhibitors and then advance them towards filing an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA). This is the first step in the drug review process before clinical trials can begin and will represent an exciting milestone for our Company.”

An Innovation Credit can include the technical development of a new product or process or the clinical development of a medicine or device and requires matching funding from Scenic. After the IND approval phase, Scenic will seek a Partner to support human clinical trials.

Source: Scenic (press release)

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Glasheldere procedures essentieel voor snelle toegang nieuwe geneesmiddelen

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De herziene versie van de beoordelingsprocedure van de specialistische geneesmiddelen is gepubliceerd op de website van het Zorginstituut. De belangrijkste aanpassingen in de beoordelingsprocedure zijn de introductie van de sluisprocedure en de agendering van sluiskandidaten met behulp van de Horizonscan Geneesmiddelen. Voor een snelle toegankelijkheid van nieuwe geneesmiddelen zijn glasheldere procedures immers onmisbaar. HollandBIO is dan ook blij dat de beoordelingsprocedure weer helemaal up-to-date is en werkt graag mee om ook procedures voor open instroom te expliciteren.

Voor specialistische geneesmiddelen die op basis van de horizonscan niet in de sluis zijn geplaatst, zijn die glasheldere procedures nu helaas nog ver te zoeken. Die producten volgen de route van open instroom, een wat misleidende naam. Conform de Zorgverzekeringswet (Zvw) beoordelen Zorgverzekeraars of een geneesmiddel recht heeft op vergoeding, door de stand van wetenschap en praktijk (effectiviteit) te duiden. In de praktijk vraagt de zorgverzekeraar echter ook gegevens over kosteneffectiviteit en wordt de aanspraakstatus willekeurig op NEE gezet. Zonder formele procedures, tijdslijnen of bezwaarmogelijkheden, dreigt open instroom te verworden tot een vagevuur, met de patiënt als kind van de rekening. Hier is volop ruimte voor verbetering.

Hoewel open instroom niet binnen de scope van de herziening door het Zorginstituut viel, blijkt uit de consultatieronde dat HollandBIO niet de enige partij is die zich zorgen maakt over het open instroom proces. “Wij herkennen de wens om duidelijkheid te verkrijgen in de rollen voor betrokken partijen bij de verschillende routes voor een geneesmiddel om in het verzekerde pakket te worden opgenomen. In het Landelijk Overleg Dure Geneesmiddelen (LODG), waar veel van de geconsulteerde partijen zitting in hebben, zijn in de verschillende actielijnen deze partijen aan zet om hier gezamenlijk duidelijkheid in te geven, ieder vanuit de eigen verantwoordelijkheid”, aldus ZIN.

Helaas zit de industrie niet aan tafel bij het LODG, al staan we steevast op het menu. Het is hoogste tijd om écht met alle stakeholders die handschoen op te pakken, en werk te maken van een adaptief ecosysteem waarbij vergoeding naadloos aansluit op registratie. HollandBIO werkt daar maar wat graag aan mee!

Op de website van het Zorginstituut kan men de volgende documenten downloaden, klik hier:

  • Definitieve versie van de beoordelingsprocedure van de specialistische geneesmiddelen.
  • Reactiebrieven van de geconsulteerde partijen, waaronder die van HollandBIO.
  • Reactiebrief van het Zorginstituut, waarin zij ingaan op de binnengekomen reacties.
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uniQure Announces First Two Patients Treated in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease

uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, has announced that the first two patients in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease have been treated. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States, with now one patient treated with AMT-130, and one patient who received the imitation surgery.

“For years, uniQure has had an unwavering commitment to advance this first-in-human AAV gene therapy for Huntington’s disease into clinical testing, and this moment marks an important milestone for our company now that we have two AAV gene therapy candidates in clinical development,” said Matt Kapusta, chief executive officer of uniQure. “With the first two patients treated in this trial, we have taken a significant step forward in advancing AMT-130 closer to our goal of developing a therapy that inhibits the production of the mutant huntingtin protein. We are delighted to be working with leading experts in the field to evaluate this promising candidate.”

The Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease will explore the safety, tolerability, and efficacy signals in 26 patients with early manifest Huntington’s disease randomized to treatment with AMT-130 or an imitation (sham) surgery. The five-year, multi-center trial consists of a blinded 18-month core study period followed by unblinded long-term follow-up. Patients will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). Additional details are available on www.clinicaltrails.gov (NCT04120493).

The first two patients will be observed for an initial period of 90 days, followed by a meeting of the Data Safety Monitoring Board (DSMB). The DSMB will review the data on the first two patients and make a determination about continued dosing of the next patients.

AMT-130 is uniQure’s first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE™ platform.

“There is an urgent need for disease-modifying options to treat Huntington’s disease, and we’re excited to have an investigational gene therapy now available for HD patients,” stated George Yohrling, chief scientific officer and chief mission officer at Huntington’s Disease Society of America. “Based on the promising preclinical data presented on AMT-130 over the years, we are optimistic about its potential to alter the course of this devastating disease.”

About Huntington’s Disease
Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntington’s disease, there are no currently approved therapies to delay the onset or to slow the disease’s progression.

Source: uniQure (press release)

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ISA Pharmaceuticals Strengthens Strategic Immuno-Oncology Collaboration with Regeneron

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ISA Pharmaceuticals, a private clinical-stage immunotherapy company, plans to initiate a potentially pivotal clinical trial for the combination of ISA101b and Libtayo® (cemiplimab) in oropharyngeal cancer, a type of head-and-neck cancer, under its strategic immuno-oncology collaboration with Regeneron. In addition, Regeneron will increase its equity stake in ISA Pharmaceuticals.

The new oropharyngeal cancer trial is the third trial planned under the clinical collaboration between ISA Pharmaceuticals and Regeneron. ISA101b, an immunotherapy targeting human papillomavirus type 16 (HPV16), is currently in a Phase 2 clinical trial for first- and second-line HPV16-positive head-and-neck cancer in combination with Libtayo, a PD-1 inhibitor that is being jointly developed by Regeneron and Sanofi. A second Phase 2 trial investigating the same combination in cervical cancer is also planned. More than 60 percent of head-and-neck cancers and approximately 55 percent of cervical cancers are HPV16 induced.

Under the revised agreement, the clinical collaboration offers a potential shortened path to first approval in the HPV16-positive oncology indications being studied in the next few years. The agreement also streamlines ISA Pharmaceuticals’ access to license fees, milestone payments and royalties. Together with the recently secured 20 million Euro loan from the European Investment Bank, development of ISA101b is now fully funded through the data readout of ongoing and planned pivotal trials which will support regulatory filings.

“This agreement will accelerate the development and commercialization of our lead asset, ISA101b, to treat two important cancers with high unmet medical needs.” commented Gerben Moolhuizen, Chief Executive Officer of ISA Pharmaceuticals.“We are extremely pleased to be working with Regeneron and for their continued support of both ISA Pharmaceuticals and our clinical studies in both indications.”

“Thanks to this updated clinical collaboration, we are able to rapidly diversify our research into a broader array of HPV16-induced cancers,” said Israel Lowy, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Oncology, at Regeneron. “ISA101b offers a validated mechanism of action, and we look forward to working with ISA Pharmaceuticals to determine the synergistic potential of combining this novel immunotherapy with Libtayo to better address these difficult-to-treat cancers.”

Source: ISA Pharmaceuticals

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Johnson & Johnson Announces Acceleration of its COVID-19 Vaccine Candidate; Phase 1/2a Clinical Trial to Begin in Second Half of July

Johnson & Johnson today announced that through its Janssen Pharmaceutical Companies (Janssen) it has accelerated the initiation of the Phase 1/2a first-in-human clinical trial of its investigational SARS-CoV-2 vaccine, Ad26.COV2-S, recombinant. Initially scheduled to begin in September, the trial is now expected to commence in the second half of July.

Paul Stoffels, M.D., Vice Chairman of the Executive Committee and Chief Scientific Officer, Johnson & Johnson, said, “Based on the strength of the preclinical data we have seen so far and interactions with the regulatory authorities, we have been able to further accelerate the clinical development of our investigational SARS-CoV-2 vaccine, Ad26.COV2-S, recombinant. Simultaneously, we are continuing our efforts to build important global partnerships and invest in our vaccine production technology and manufacturing capabilities. Our goal is to ensure we can deliver a vaccine to the world and protect people everywhere from this pandemic.”

The randomized, double-blind, placebo-controlled Phase 1/2a study will evaluate the safety, reactogenicity (response to vaccination), and immunogenicity (immune response) of the investigational SARS-CoV-2 vaccine, Ad26.COV2-S, recombinant in 1045 healthy adults aged 18 to 55 years, as well as adults aged 65 years and older. The study will take place in the U.S. and Belgium.

The Company is in discussions with the National Institutes of Allergy and Infectious Diseases with the objective to start the Phase 3 SARS-CoV-2 vaccine, Ad26.COV2-S, recombinant, clinical trial ahead of its original schedule, pending outcome of Phase 1 studies and approval of regulators.

As the Company progresses the clinical development of its investigational SARS-CoV-2 vaccine, Ad26.COV2-S, recombinant, it continues to increase manufacturing capacity and is in active discussions with global partners to ensure worldwide access. The Company committed to the goal of supplying more than one billion doses globally through the course of 2021, provided the vaccine is safe and effective.

Johnson & Johnson’s efforts to expedite development and production of a SARS-CoV-2 vaccine are enhanced by a collaboration between Janssen and the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response (ASPR) at the U.S. Department of Health & Human Services.

COVID-19 is caused by SARS-CoV-2, which belongs to a group of viruses called coronaviruses that attack the respiratory system. There is currently no approved vaccine for COVID-19.

For more information on Johnson & Johnson’s multi-pronged approach to combatting the pandemic, visit: www.jnj.com/coronavirus.

Source: Johnson & Johnson (press release)

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