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
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.
BioGeneration Ventures (BGV), a leading early-stage VC in European biopharma, announces the closing of its fourth fund, BGV IV, at €105 million ($119 million). Strong market interest expediated the fund raise, with the commitments to date exceeding the fund’s original target.
The BGV IV fund has attracted notable institutions as investors such as, Bristol Myers Squibb, Schroder Adveq and the European Investment Fund. New investors to BGV IV include Industriens Pension and KfW Capital.
BGV is one of the largest VC funds in Europe focused on early investments in new bio-technology companies, with over € 220 million assets under management. Its team has broad experience in investment, life sciences, business development, and commercial operations. It also draws on experienced biotech entrepreneurs as venture partners and advisors, to the benefit of its portfolio companies.
The Company’s approach is to partner with scientists from major European institutions and entrepreneurs. The aim is to build new companies, around either single assets or technology platforms with the goal of creating transformational new medicines. This strategy is supported by the strong collaboration between BGV and Forbion, providing a platform across early- to late-stage companies.
The investment strategy for the BGV IV fund will follow the same successful path as BGV’s other funds, which have resulted in innovative new medicines reaching patients and yielded significant returns for investors.
Portfolio successes of earlier BGV funds include Acerta Pharma, which was acquired by AstraZeneca for up to $7bn and whose lead product Calquence® is now approved and marketed in the US, and Staten Biotechnology, which signed a €430 million exclusive option deal with Novo Nordisk. Other companies in the current BGV portfolio include NorthSea Therapeutics, Azafaros, Varmx, and Confo Therapeutics.
Edward van Wezel, BGV’s Managing Partner said:
“Over the last few years, BGV has demonstrated that investing in early-stage companies in Europe can bring innovative science to meet patients’ needs worldwide as well as financial value to investors. With this new fund, we are pleased to have the backing of some of our long-standing investors; we also welcome our new investors, Industriens Pension and KfW Capital. The early-stage science coming out of European Institutions is second to none and we are working with those at the forefront of their fields. We believe we can make a significant difference to companies in helping them translate promising science into transformational new medicines.”
Frederik Fischer Hjerl, Investment Manager at Industriens Pension, added:
“As a new investor to the BGV fund, we have had a lot of focus at the BGV team’s strong track record and their approach towards early innovation. There is an urgent demand for new medicines which might accelerate the level of innovation coming out of research institutions across Europe. Having an experienced team like BGV, which in multiple occasions has proven capable of identifying and nurturing the potential future stars, is crucial to bringing new ideas forward. We look forward to working with BGV to help bring new medicines through.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-06-24 14:29:002020-06-30 14:31:36BioGeneration Ventures closes BGV IV fund at €105 million← #nieuws
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.
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.
Two weeks ago, key
stakeholders from Massachusetts and the Netherlands came together in a digital
session to discuss how the Life Sciences & Health ecosystems are responding
to COVID-19, which lessons could be learned from the reaction to the pandemic,
and how to stimulate closer international collaboration.
The same organisations also
signed a MoU in 2019 to advance ongoing collaboration in life sciences and
included Mark Sullivan (Massachusetts Office of International Trade and
Investment, MOITI), Elizabeth Steele (MassBio), and Jennifer Griffin
(Massachusetts Life Sciences Center) from Massachusetts and Focco Vijselaar
(Ministry of Economic Affairs and Climate Policy), Clémence Ross-van Dorp
(Ministry of Economic Affairs and Climate Policy), Annemiek Verkamman
(HollandBIO) and Carmen van Vilsteren (Health~Holland) from the Netherlands. Akshay
Vaishnaw (Alnylam Pharmaceuticals) and Freddy Nguyen (MIT COVID-19 Challenge)
were invited as speakers to share their activities in light of COVID-19. The
session was moderated by Hans Schikan (Top Sector Life Sciences &
Health).
Biotech companies have proven
to be dynamic and are pivoting to focus their work on therapies and vaccines. Even
more so, participants observe an increased level of cooperation between
different companies, researchers and governments to find solutions. For
instance, the extent with which data has been shared across the sector has largely
increased. This doesn’t stop at the private sector, as public and semi-public
organizations have informed each other across borders to support each other in
handling the situation. Also when looking at formal procedures, agencies have
shown great efficiency in shortening approval procedures to speed up the
development of vaccines and therapies.
A paradox can be observed
regarding the supply of PPE and other vital medical equipment that companies
have sourced internationally. The pandemic has shown the limits of
international supply chains, therefore, will governments require that vital
manufacturing be maintained within their geographical reach? On the other hand,
participants in this virtual session agree that there will always be a need to
collaborate internationally and make use of each other’s strengths. According
to the Airbus model, if all strengths from different countries or ecosystems
could be brought together, this will create a stronger value chain where
collaboration between partners is needed and truly adds value.
When looking ahead, participants recognize that in a post-COVID world, we should be better prepared for a possible outbreak of a pandemic. That we need to be ready to think and act together, internationally, so we can organize the supply chain and share data and expertise when needed.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-06-23 15:40:312020-06-23 15:41:08Key stakeholders Massachusetts and the Netherlands meet again in virtual session← #nieuws
Een
prominente groep parlementsleden binnen de Duitse Groenen roept de partij op om
de huidige negatieve positie ten opzichte van de toepassing van moderne
veredelingsmethoden te herzien. Volgens de groep kunnen deze methoden een
belangrijke bijdrage leveren aan duurzaamheid. Ook roept de groep op tot
aanpassing van de verouderde en strenge Europese wetgeving voor genetische
modificatie. Deze belemmert met name innovatie door kleine bedrijven. HollandBIO
is blij met deze ontwikkelingen bij onze oosterburen en hoopt dat die navolging
krijgen in de rest van Europa!
https://www.hollandbio.nl/wp-content/uploads/2020/06/3921small-Tomaten.jpg13342000hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-06-23 15:35:032020-06-23 15:37:02Groep Duitse Groenen ziet toekomst voor moderne veredeling← #nieuws
EenVandaag stond afgelopen zondag stil bij de grote gevolgen van de coronacrisis op innovatieve start-ups, scale-ups en MKB. Remko van Leeuwen, CEO van Madam Therapeutics, vertelt in de uitzending over de financiële uitdagingen. De kleine bedrijven zijn de groeimotor van de Nederlandse economie. Ze hebben zwaar te lijden onder de coronacrisis, maar de steunmaatregelen sluiten veelal niet aan bij hun behoefte. Ook de Corona-Overbruggingslening (COL), die in eerste instantie een uitkomst leek, schiet tekort. Het budget is zwaar overvraagd, tijdslijnen worden overschreden en 43,5% van aanvragers kreeg al nul op het rekest.
Recent onderzoek van StartUp Genome laat zien
dat 86% van de innovatieve start-ups, scale-ups en MKB binnen drie maanden in
financiële problemen komt. Naast de COL, werd ook het Tijdelijke
Overbruggingskrediet innovatieve Start en Scale ups (TOPSS) in het leven
geroepen om deze belangrijke bedrijven door de crisis heen te slepen. Met een
totaal budget van €300 miljoen, zijn de regelingen echter een druppel op een gloeiende plaat. Al meer dan
1700 bedrijven hebben een aanvraag ingediend goed voor een bedrag van €760
miljoen. De nood is dus hoog. 25,8% van de aanvragen wacht nog op een oordeel
en 43,5% werd reeds afgewezen.
De COL-aanvraag van Madam Therapeutics was één
van die afgewezen voorstellen. In EenVandaag
vertelt CEO Remko van Leeuwen over de situatie. Hij geeft aan dat het normaal
al geen sinecure is om investeerders aan te trekken voor de ontwikkeling van
nieuwe antibiotica, laat staan in tijden van crisis. En dat terwijl de zorgen
rondom infecties en resistentie groter dan ooit zijn. De afwijzing vindt hij
dan ook onterecht.
Ook Luciel Burm, voorzitter van de Dutch
Startup Association (DSA) spreekt zijn zorg uit. Als er niet meer geld bijkomt,
dreigt een kaalslag van de sector. Landen om ons heen treden veel doortastender
op: Duitsland, Frankrijk en Engeland investeren momenteel miljarden in
start-ups, en daarmee in de economie van morgen.
De regionale ontwikkelingsmaatschappijen
(ROMs), die de COL aanvragen beoordelen en verstrekken, zien zelf ook dat de
nood hoog is. Zo spreekt Marc Janssen, manager Brabant Ventures bij de BOM de
hoop uit dat er uiteindelijk nog meer geld beschikbaar komt, vooral voor
bedrijven in hun vroege fase. HollandBIO kan zich daar alleen maar bij
aansluiten, net als bij de woorden van DSA’s Luciel Burm: ‘Minister Wiebes heeft gezegd dat we ons
uit de crisis moeten investeren. Ik nodig hem van harte uit dat te doen in
start-ups.’.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-06-23 15:22:262020-06-23 15:22:27EenVandaag: loods innovatieve start-ups door de coronacrisis← #nieuws
Gecultiveerd vlees. HollandBIO was al fan. De
Tweede Kamer ook, zo bleek uit het kweekvlees-debat en drie aangenomen moties.
En nu spreekt ook Minister Schouten (LNV) haar volle steun uit voor deze
interessante innovatie. In een Kamerbrief zet de minister uiteen hoe zij de
ontwikkeling van gecultiveerd vlees wil ondersteunen. Allereerst is en blijft
de minister in gesprek met de ontwikkelaars van gecultiveerd vlees in
Nederland, om zo mogelijke drempels of knelpunten op weg naar de markt tijdig
weg te nemen. HollandBIO is blij met de support en de erkenning voor de
bijdrage die gecultiveerd vlees kan leveren aan een duurzamere, gezondere en
diervriendelijke wereld.
Naast de dialoog geeft minister Schouten aan dat
zij, vanwege de grote potentie van de ontwikkelingen in gecultiveerd vlees, met
communicatie-uitingen het belang van deze innovatie gaat benadrukken en te gaan
investeren in vervolgonderzoek. Zo gaat zij in overleg met onder meer de groene
hogescholen om te bezien of zij komend jaar een onderzoekscall kunnen openen op
het gebied van gecultiveerd vlees. Tot slot krijgt gecultiveerd vlees een plek
krijgt in de Nationale Eiwitstrategie (NES) waar het ministerie van landbouw
momenteel aan werkt. Met de NES wil Nederland werk maken van een verschuiving
naar meer duurzame eiwitten. Gecultiveerd vlees past uitstekend binnen de
pijler alternatieve en innovatieve eiwitbronnen.
Benieuwd naar de hele Kamerbrief? Lees dan hier verder
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.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-06-19 18:03:002020-06-23 18:08:03uniQure Announces First Two Patients Treated in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
Access to COVID-19 Tools (ACT) Accelerator publishes consolidated plans
Gezondheid op maat, GezondheidNo-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
Hansa Biopharma receives positive CHMP opinion for Idefirix for kidney transplant in EU
GezondheidHansa 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)
BioGeneration Ventures closes BGV IV fund at €105 million
InnovatieklimaatBioGeneration Ventures (BGV), a leading early-stage VC in European biopharma, announces the closing of its fourth fund, BGV IV, at €105 million ($119 million). Strong market interest expediated the fund raise, with the commitments to date exceeding the fund’s original target.
The BGV IV fund has attracted notable institutions as investors such as, Bristol Myers Squibb, Schroder Adveq and the European Investment Fund. New investors to BGV IV include Industriens Pension and KfW Capital.
BGV is one of the largest VC funds in Europe focused on early investments in new bio-technology companies, with over € 220 million assets under management. Its team has broad experience in investment, life sciences, business development, and commercial operations. It also draws on experienced biotech entrepreneurs as venture partners and advisors, to the benefit of its portfolio companies.
The Company’s approach is to partner with scientists from major European institutions and entrepreneurs. The aim is to build new companies, around either single assets or technology platforms with the goal of creating transformational new medicines. This strategy is supported by the strong collaboration between BGV and Forbion, providing a platform across early- to late-stage companies.
The investment strategy for the BGV IV fund will follow the same successful path as BGV’s other funds, which have resulted in innovative new medicines reaching patients and yielded significant returns for investors.
Portfolio successes of earlier BGV funds include Acerta Pharma, which was acquired by AstraZeneca for up to $7bn and whose lead product Calquence® is now approved and marketed in the US, and Staten Biotechnology, which signed a €430 million exclusive option deal with Novo Nordisk. Other companies in the current BGV portfolio include NorthSea Therapeutics, Azafaros, Varmx, and Confo Therapeutics.
Edward van Wezel, BGV’s Managing Partner said:
“Over the last few years, BGV has demonstrated that investing in early-stage companies in Europe can bring innovative science to meet patients’ needs worldwide as well as financial value to investors. With this new fund, we are pleased to have the backing of some of our long-standing investors; we also welcome our new investors, Industriens Pension and KfW Capital. The early-stage science coming out of European Institutions is second to none and we are working with those at the forefront of their fields. We believe we can make a significant difference to companies in helping them translate promising science into transformational new medicines.”
Frederik Fischer Hjerl, Investment Manager at Industriens Pension, added:
“As a new investor to the BGV fund, we have had a lot of focus at the BGV team’s strong track record and their approach towards early innovation. There is an urgent demand for new medicines which might accelerate the level of innovation coming out of research institutions across Europe. Having an experienced team like BGV, which in multiple occasions has proven capable of identifying and nurturing the potential future stars, is crucial to bringing new ideas forward. We look forward to working with BGV to help bring new medicines through.”
Source: BGV (press release)
Scenic Biotech Awarded €3.1 million Innovation Credit
Gezondheid, InnovatieklimaatScenic 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)
Glasheldere procedures essentieel voor snelle toegang nieuwe geneesmiddelen
Gezondheid op maat, Hollandbio, GezondheidDe 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:
Key stakeholders Massachusetts and the Netherlands meet again in virtual session
IJzersterk innovatieklimaat, Hollandbio, InnovatieklimaatTwo weeks ago, key stakeholders from Massachusetts and the Netherlands came together in a digital session to discuss how the Life Sciences & Health ecosystems are responding to COVID-19, which lessons could be learned from the reaction to the pandemic, and how to stimulate closer international collaboration.
The same organisations also signed a MoU in 2019 to advance ongoing collaboration in life sciences and included Mark Sullivan (Massachusetts Office of International Trade and Investment, MOITI), Elizabeth Steele (MassBio), and Jennifer Griffin (Massachusetts Life Sciences Center) from Massachusetts and Focco Vijselaar (Ministry of Economic Affairs and Climate Policy), Clémence Ross-van Dorp (Ministry of Economic Affairs and Climate Policy), Annemiek Verkamman (HollandBIO) and Carmen van Vilsteren (Health~Holland) from the Netherlands. Akshay Vaishnaw (Alnylam Pharmaceuticals) and Freddy Nguyen (MIT COVID-19 Challenge) were invited as speakers to share their activities in light of COVID-19. The session was moderated by Hans Schikan (Top Sector Life Sciences & Health).
Biotech companies have proven to be dynamic and are pivoting to focus their work on therapies and vaccines. Even more so, participants observe an increased level of cooperation between different companies, researchers and governments to find solutions. For instance, the extent with which data has been shared across the sector has largely increased. This doesn’t stop at the private sector, as public and semi-public organizations have informed each other across borders to support each other in handling the situation. Also when looking at formal procedures, agencies have shown great efficiency in shortening approval procedures to speed up the development of vaccines and therapies.
A paradox can be observed regarding the supply of PPE and other vital medical equipment that companies have sourced internationally. The pandemic has shown the limits of international supply chains, therefore, will governments require that vital manufacturing be maintained within their geographical reach? On the other hand, participants in this virtual session agree that there will always be a need to collaborate internationally and make use of each other’s strengths. According to the Airbus model, if all strengths from different countries or ecosystems could be brought together, this will create a stronger value chain where collaboration between partners is needed and truly adds value.
When looking ahead, participants recognize that in a post-COVID world, we should be better prepared for a possible outbreak of a pandemic. That we need to be ready to think and act together, internationally, so we can organize the supply chain and share data and expertise when needed.
Groep Duitse Groenen ziet toekomst voor moderne veredeling
Wereld mét toekomst, Hollandbio, Voedsel en materialenEen prominente groep parlementsleden binnen de Duitse Groenen roept de partij op om de huidige negatieve positie ten opzichte van de toepassing van moderne veredelingsmethoden te herzien. Volgens de groep kunnen deze methoden een belangrijke bijdrage leveren aan duurzaamheid. Ook roept de groep op tot aanpassing van de verouderde en strenge Europese wetgeving voor genetische modificatie. Deze belemmert met name innovatie door kleine bedrijven. HollandBIO is blij met deze ontwikkelingen bij onze oosterburen en hoopt dat die navolging krijgen in de rest van Europa!
Bron: EurActiv
EenVandaag: loods innovatieve start-ups door de coronacrisis
IJzersterk innovatieklimaat, Hollandbio, Innovatieklimaat, UitgelichtEenVandaag stond afgelopen zondag stil bij de grote gevolgen van de coronacrisis op innovatieve start-ups, scale-ups en MKB. Remko van Leeuwen, CEO van Madam Therapeutics, vertelt in de uitzending over de financiële uitdagingen. De kleine bedrijven zijn de groeimotor van de Nederlandse economie. Ze hebben zwaar te lijden onder de coronacrisis, maar de steunmaatregelen sluiten veelal niet aan bij hun behoefte. Ook de Corona-Overbruggingslening (COL), die in eerste instantie een uitkomst leek, schiet tekort. Het budget is zwaar overvraagd, tijdslijnen worden overschreden en 43,5% van aanvragers kreeg al nul op het rekest.
Recent onderzoek van StartUp Genome laat zien dat 86% van de innovatieve start-ups, scale-ups en MKB binnen drie maanden in financiële problemen komt. Naast de COL, werd ook het Tijdelijke Overbruggingskrediet innovatieve Start en Scale ups (TOPSS) in het leven geroepen om deze belangrijke bedrijven door de crisis heen te slepen. Met een totaal budget van €300 miljoen, zijn de regelingen echter een druppel op een gloeiende plaat. Al meer dan 1700 bedrijven hebben een aanvraag ingediend goed voor een bedrag van €760 miljoen. De nood is dus hoog. 25,8% van de aanvragen wacht nog op een oordeel en 43,5% werd reeds afgewezen.
De COL-aanvraag van Madam Therapeutics was één van die afgewezen voorstellen. In EenVandaag vertelt CEO Remko van Leeuwen over de situatie. Hij geeft aan dat het normaal al geen sinecure is om investeerders aan te trekken voor de ontwikkeling van nieuwe antibiotica, laat staan in tijden van crisis. En dat terwijl de zorgen rondom infecties en resistentie groter dan ooit zijn. De afwijzing vindt hij dan ook onterecht.
Ook Luciel Burm, voorzitter van de Dutch Startup Association (DSA) spreekt zijn zorg uit. Als er niet meer geld bijkomt, dreigt een kaalslag van de sector. Landen om ons heen treden veel doortastender op: Duitsland, Frankrijk en Engeland investeren momenteel miljarden in start-ups, en daarmee in de economie van morgen.
De regionale ontwikkelingsmaatschappijen (ROMs), die de COL aanvragen beoordelen en verstrekken, zien zelf ook dat de nood hoog is. Zo spreekt Marc Janssen, manager Brabant Ventures bij de BOM de hoop uit dat er uiteindelijk nog meer geld beschikbaar komt, vooral voor bedrijven in hun vroege fase. HollandBIO kan zich daar alleen maar bij aansluiten, net als bij de woorden van DSA’s Luciel Burm: ‘Minister Wiebes heeft gezegd dat we ons uit de crisis moeten investeren. Ik nodig hem van harte uit dat te doen in start-ups.’.
En dat alles liever vandaag dan morgen.
Lees meer
Minister Schouten zet tanden in gecultiveerd vlees
Wereld mét toekomst, Hollandbio, Voedsel en materialen, UitgelichtGecultiveerd vlees. HollandBIO was al fan. De Tweede Kamer ook, zo bleek uit het kweekvlees-debat en drie aangenomen moties. En nu spreekt ook Minister Schouten (LNV) haar volle steun uit voor deze interessante innovatie. In een Kamerbrief zet de minister uiteen hoe zij de ontwikkeling van gecultiveerd vlees wil ondersteunen. Allereerst is en blijft de minister in gesprek met de ontwikkelaars van gecultiveerd vlees in Nederland, om zo mogelijke drempels of knelpunten op weg naar de markt tijdig weg te nemen. HollandBIO is blij met de support en de erkenning voor de bijdrage die gecultiveerd vlees kan leveren aan een duurzamere, gezondere en diervriendelijke wereld.
Naast de dialoog geeft minister Schouten aan dat zij, vanwege de grote potentie van de ontwikkelingen in gecultiveerd vlees, met communicatie-uitingen het belang van deze innovatie gaat benadrukken en te gaan investeren in vervolgonderzoek. Zo gaat zij in overleg met onder meer de groene hogescholen om te bezien of zij komend jaar een onderzoekscall kunnen openen op het gebied van gecultiveerd vlees. Tot slot krijgt gecultiveerd vlees een plek krijgt in de Nationale Eiwitstrategie (NES) waar het ministerie van landbouw momenteel aan werkt. Met de NES wil Nederland werk maken van een verschuiving naar meer duurzame eiwitten. Gecultiveerd vlees past uitstekend binnen de pijler alternatieve en innovatieve eiwitbronnen.
Benieuwd naar de hele Kamerbrief? Lees dan hier verder
uniQure Announces First Two Patients Treated in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
GezondheiduniQure, 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)