This year, the BIO International Convention will transition to virtual event format: BIO Digital. HollandBIO members can access this largest virtual gathering of the global biotech industry at a discounted rate of $950. Read more
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-04-21 14:24:182020-04-21 14:24:19BIO International Convention goes BIO digital← #nieuws
In the weekend of 3 till 5 April, 1500 selected
participants took part in the MIT Covid-19 Challenge – Beat the Pandemic
Hackathon. They were selected out of more than 4500 applications from over 100
countries. Dutch participants were also joining team sessions in 10 different
tracks.
The teams, under guidance of mentors, presented their
work at the end of the weekend to judge panels that picked 40 winning teams out
of 208 teams. The organization is now analyzing which teams are able to further
work on their solutions and what is needed to get these solutions into practice.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-04-21 14:19:252020-04-21 14:19:26First MIT Covid-19 Challenge; a great success← #nieuws
Synthon Biopharmaceuticals B.V., a biopharmaceutical research and development company creating innovative precision medicines for patients with relentless cancers and autoimmune diseases, today announced that it is relaunching as Byondis.
Byondis is creating safe and effective precision medicines based on its proprietary technologies, targeting intractable cancers and auto-immune diseases, which aim to improve the standard of care for patients.
The Company has a broad development pipeline, including advanced clinical programs, such as anti-HER2 ADC [vic-]trastuzumab duocarmazine (SYD985) for breast cancer. In the second half of 2020, Byondis is expecting results from its pivotal TULIP® Phase III trial comparing SYD985 to physician’s choice treatment in patients with HER2-positive unresectable locally advanced or metastatic breast cancer. Submission of a Biological License Application is scheduled before the end of 2020.
The Company anticipates entering the clinic with four additional programs in the course of 2020/2021. After progressing to clinical proof of concept, Byondis intends to enter into partnerships for late-stage development and commercialization.
Synthon Biopharmaceuticals was established in 2007 as part of Synthon B.V., and became a separate biopharmaceutical subsidiary in 2012, quickly building a promising pipeline of innovative R&D programs. In November last year, following the acquisition of Synthon International Holding B.V. by BC Partners, the founder, Dr Jacques Lemmens, decided to continue as an independent biopharmaceutical company, with its own management team and state-of-the-art facilities. This company relaunches today as Byondis.
“Our relaunch as Byondis is an exciting moment for the Company” said Dr Jacques Lemmens, Founder. “Building on our heritage, while preserving our company culture, we are further progressing in innovation, quality and scientific excellence.”
“Now is the time to step up as an independent biopharmaceutical company, with our exceptional people, unique technologies and a well-established development pipeline, including our lead ADC, [vic-]trastuzumab duocarmazine. I am very proud of how the Company has grown and evolved. The new name, Byondis, represents the next chapter and embodies our entrepreneurial spirit and ambition to create innovative precision medicines to outsmart cancers and auto-immune diseases.”
The Byondis management team has a wealth of scientific, clinical and operational experience, and is well networked within the pharmaceutical industry. Dr Marco Timmers, CEO, and others in the senior team have collaborated for many years as part of Synthon. During this time, they have created a company driven by science and innovation, with unique technologies and significant validation deals. These include the out-licensing of a biosimilar version of the monoclonal antibody trastuzumab (marketed as KANJINTI™) in a global partnership agreement with Amgen in 2012.
“As Byondis, we can now showcase our strong business and scientific proposition”, added Dr Marco Timmers, CEO. “We have generated a portfolio of preclinical and clinical programs with the potential to deliver completely novel treatments for cancer and auto-immune diseases. We have unique technologies with differentiating potential, state-of-the-art facilities including our own GMP manufacturing, and broad licensing expertise that makes us an attractive partner for out-licensing. This is a really exciting time for Byondis and our team.”
Byondis operates from its headquarters in Nijmegen in the Netherlands, with over 350 highly qualified employees working in state-of-the art research and development and GMP manufacturing facilities. The Company has strong links to academic institutions, global medical communities and the international pharma industry.
Galapagos and Ryvu Therapeutics have announced a collaboration focused on the discovery and development of novel small molecule drugs in inflammation.
Ryvu specializes in the discovery and development of first-in-class small molecules and drug candidates in diseases with high unmet medical needs. The collaboration announced today is based on a novel drug target identified by Ryvu, which will contribute its technology platform and related intellectual property (IP). Ryvu and Galapagos will both provide resources to support the collaboration and make use of their expertise in high-throughput screening, biology, medicinal chemistry, and toxicology.
This is a joint research collaboration in which Ryvu is responsible for early drug discovery. Under the terms of the agreement, Galapagos will have an exclusive option to license IP developed by Ryvu and to continue to develop this during the collaboration. Pending achievement of pre-agreed criteria and utilizing its option, Galapagos will be responsible for all further development of the program.
In exchange for global development and commercialization rights, Ryvu will receive an upfront payment and will be eligible for further option, milestone, and royalty payments.
“We believe that the collaboration with Ryvu is an excellent fit, as both companies are driven by the search for novel drugs to address unmet medical needs,” says Dr. Piet Wigerinck, Chief Scientific Officer at Galapagos. “We look forward to collaborating with the Ryvu team to push this program forward.”
Pawel Przewiezlikowski, Ryvu Chief Executive Officer, added: “We are thrilled to start working with Galapagos, a real role model for the European biotechnology sector. Throughout joint discussions on the collaboration, our teams have developed a strong rapport and built the foundations for a very promising start for the new project on an exciting novel target.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-04-16 22:19:002020-04-21 22:23:50Galapagos and Ryvu announce research collaboration← #nieuws
Khondrion, a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease, has published a scientific hypothesis that the inflammatory lipid modulator, prostaglandin E2 (PGE2), may execute a prominent role in COVID-19 pathophysiology and proposes that its lead drug candidate, sonlicromanol, currently in phase IIb development to treat a range of mitochondrial diseases, could be repurposed for the treatment of patients with severe COVID-19 disease.
The paper, published online by Preprints, the online multidisciplinary platform dedicated to making early versions of research outputs permanently available and citable, summarizes the potential role that elevated levels of PGE2, which is known to play an essential role in inflammation1 and defense against infectious agents2, may have in COVID-19 pathology. It calls for the measurement of PGE2 in affected patients and proposes selective inhibition of a key enzyme involved in PGE2 production – microsomal prostaglandin E synthase-1 (mPGES-1) – as a potential new treatment approach in protecting COVID-19 patients from severe disease progression and death.
Human mPGES-1 is already recognized as a promising target for the next generation of antiinflammatory drugs, without the side effects of those currently available, e.g. non-steroidal antiinflammatory drugs (NSAIDs) and COX-2 inhibitors3. The Khondrion research team’s hypothesis that selective mPGES-1 inhibition might reduce COVID-19 associated disease symptoms, e.g. acute respiratory distress syndrome (ARDS), is based on PGE2’s role as an inflammatory modulator in viral infections as well as evidence that targeted PGE2 inhibition enhances antiviral immunity4,5,6.
Sonlicromanol, Khondrion’s wholly-owned investigational lead asset currently in phase IIb development as a potentially disease-modifying treatment for mitochondrial disease, has a triple mode of action that includes the inhibition of mPGES-1 and may, in turn, result in an antiinflammatory effect. Khondrion research using human fibroblast cells and a mouse macrophage-like cell line shows that sonlicromanol is able to block mPGES-1 and decrease PGE2 production. Therefore, in addition to it being a novel therapeutic option for mitochondrial disease patients, the Company proposes that sonlicromanol may also have potential to be repurposed to treat PGE2- driven inflammatory consequences that might underly COVID-19 associated ARDS or, when administered early after diagnosis, might prevent progression to ARDS.
Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: “During the development of sonlicromanol for the treatment of mitochondrial disease we have generated a wealth of data on its mode of action, including its effects on the body’s inflammatory responses. What we have learned about this asset’s potential is striking when examined through the lens of COVID-19 and the scientific community’s search for new therapies as part of the global response to this pandemic. “While there remain limitations in our understanding of PGE2 and its role in the serious lung disease associated with COVID-19, we believe it warrants further investigation, particularly when new therapeutic options are so desperately needed. “We are inviting experts in the field of coronavirus to test our compound in-vitro and in-vivo. We are also actively searching for partners willing to assist us in evaluating our hypothesis and the potential of bringing sonlicromanol to COVID-19 patients.”
The paper “Hypothesis: mPGES-1-derived Prostaglandin E2, a so far missing link in COVID-19 pathophysiology?” is available on Preprints here.
A consortium of public and private R&D organisations from EU, India, and US has been awarded a 10 m€ grant for development of improved influenza virus vaccines for the globe. Later this year, this grant will be matched by the government of India. LiteVax BV (Oss, The Netherlands) and the Translational Health Science Technology Institute (Delhi, India) have initiated the project and the University of Amsterdam will be the coordinator. The INDIGO project builds on LiteVax’s patented, versatile adjuvant technology developed to improve prophylactic and therapeutic vaccines.
The grant will support the development of a next generation of influenza vaccines aiming at higher effectivity, lower costs and better accessibility. The INDIGO project combines innovations in antigen design, antigen delivery and immune-stimulation and plans to perform phase I/IIa clinical trials in EU and India. In addition to LiteVax, the INDIGO consortium consists of one Danish partner, two Belgian partners, three French partners, two Dutch partners, one US partner, and six Indian partners. An important goal is the collaboration between R&D organizations in these countries with complementary skills and know-how. See https://cordis.europa.eu/project/id/874653 for more information.
Pharvaris, a clinical-stage company focused on the discovery and development of novel oral B2-receptor antagonists for the treatment of hereditary angioedema (HAE) and other B2-receptor-mediated indications, announced data from a late-breaking poster planned for presentation at the recently canceled annual meeting of the American Academy of Allergy, Asthma & Immunology (AAAAI).
Pharvaris established a proof-of-concept model for HAE in non-human primates. In this model, PHA121 inhibited bradykinin-induced changes in blood pressure at all doses tested (0.1, 0.3, 1, 3, and 10 mg/kg given orally) with a faster onset of action than icatibant. The duration of the effect was dose dependent.
“These results provide confidence in PHA121’s clinical profile for the treatment of hereditary angioedema,” said Berndt Modig, Chief Executive Officer and co-founder of Pharvaris. “Combined with preliminary findings from our ongoing Phase 1 study, these data demonstrate that PHA121 is a potent, orally available bradykinin B2 receptor antagonist.”
The objective of the study was to investigate the ability of PHA121 to attenuate blood-pressure changes induced by bradykinin injection. Freely moving monkeys were challenged with bradykinin, resulting in a transient drop in blood pressure. Bradykinin is an endogenous peptide known to mediate signs and symptoms of HAE. The model was validated utilizing icatibant, a marketed injectable B2 receptor antagonist, providing back-translation from human clinical experience with icatibant.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-04-14 22:23:072020-04-14 22:23:08Pharvaris oral HAE drug shows positive results← #nieuws
Een aanvraag voor klinisch onderzoek met bepaalde vormen van cel- en
gentherapie duurt in Nederland straks nog maar 56 dagen. Daarnaast gaan de
informatievereisten omlaag. Dit blijkt uit het voorstel van minister Van
Nieuwenhuizen voor een wijziging van het besluit GGO. De wijziging geldt voor
een aantal toepassingen, waarvoor de aanvraagperiode tot voor kort vaak langer
dan een jaar in beslag nam. HollandBIO is blij dat de wensen van het veld ter
harte worden genomen, maar pleit ook voor verdere verbeteringen. Alleen dan
maken we Nederland ècht internationaal toonaangevend.
In het voorstel is een snellere route van maximaal 56 dagen met
verlaagde informatievereisten terug te vinden voor toepassingen die zijn
gebaseerd op AAV-vectoren en ex vivo getransduceerde
cellen vervaardigd met retrovirale of lentivirale vectoren. Ook biedt
het voorstel de ruimte voor kopievergunningen, waarbij aanvragers een eerdere
aanvraag als uitgangspunt kunnen nemen.
Hoewel dit een verbetering is, ziet HollandBIO mogelijkheden om Nederland
bij de internationale kopgroep aan te laten sluiten. We zetten ons daarom in
voor de volgende punten:
Het toevoegen van een categorie
voor kopievergunningen en toepassingen met een hoogstens verwaarloosbaar
milieurisico waarin vergunningverlening voor klinisch onderzoek maximaal 28
dagen duurt;
Het introduceren van een lerend
systeem waarmee de overheid niet telkens de regelgeving aan moet passen maar
eenvoudiger toepassingen aan een bepaalde categorie kan toewijzen.
Verbetering van de aansluiting bij
de Europese ‘Interplay’-afspraken. Nederland
heeft zich bijvoorbeeld niet gecommitteerd aan het automatisch afgeven van een
milieuvergunning voor onderzoek met een geregistreerde ggo-toepassing naar een
nieuwe indicatie. Terwijl deze afspraak al door 23 landen wordt onderschreven.
Minister Van Nieuwenhuizen heeft het voorstel ter wijziging van Besluit
GGO inmiddels naar de Tweede Kamer gestuurd, die tot 3 juni de tijd krijgt voor
inspraak voordat zij overgaat tot de inhoudelijke behandeling. Het
wijzigingsvoorstel is ook gepubliceerd in de Staatscourant, waarmee iedereen
die dat wil binnen 4 weken een reactie kan geven.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-04-14 22:09:172020-04-14 22:09:18Drastische versnelling vergunningaanvraag medische GGO’s, nu door voor de kopgroep← #nieuws
De economische
gevolgen van het coronavirus stellen biotech start-ups en scale-ups voor grote
uitdagingen. HollandBIO maakt zich hard voor ondersteuning toegesneden op de
specifieke behoeften van de biotech sector. Op 7 april presenteerde het
kabinet aanvullende
maatregelen om ondernemers te ondersteunen. HollandBIO
is blij met deze eerste stap, maar blijft benadrukken dat verdergaande en meer
duurzame ondersteuning nodig is voor de Nederlandse biotech sector. Niet alleen
is de sector cruciaal voor het toekomstig verdienvermogen van Nederland,
vandaag de dag blijkt de innovatiekracht van de sector op alle fronten harder
nodig dan ooit.
Overbruggingskrediet
Het kabinet maakt 100
miljoen euro vrij voor overbruggingskredieten voor non-bancair gefinancierde
bedrijven. Kredieten tot een maximum van 2 miljoen euro worden verstrekt door
regionale ontwikkelingsorganisaties (ROM’s), Invest-NL krijgt een rol voor
hogere bedragen. Ook werken het ministerie van Economische Zaken en Klimaat
(EZK), de ROM’s, Invest-NL en Techleap aan een gezamenlijk programma gericht op
het verstrekken van overbruggingskrediet in de vorm van (converteerbare)
leningen aan innovatieve start- en scale-ups. HollandBIO bepleit de snelle,
eenvoudige en brede beschikbaarheid van converteerbare kredieten om biotech
bedrijven in acute financiële nood te helpen.
Uitstel betaling Innovatiekrediet
In haar brief aan de
Tweede Kamer kondigt het kabinet uitstel van betaling voor Vroege Fase
Financiering (VVF) en het Innovatiekrediet aan, van maximaal 6 maanden.
HollandBIO vindt dit echter onvoldoende. Met deze maatregel wordt de rekening
slechts uitgesteld, terwijl die door rente op rente wel fors oploopt. Een
tijdelijke rentestop, tot twee jaar uitstel van afbetalingen en een generieke
en retrospectieve renteaanpassing geeft ondernemers meer lucht in deze
moeilijke tijd. Op verzoek van vele bedrijven in onze achterban, bespreekt
HollandBIO verdere mogelijkheden met RVO en EZK. Hierbij vragen we ook aandacht
voor soepele honorering van aanvragen voor nieuwe Innovatiekredieten.
WBSO
Biotech bedrijven
profiteren zonder uitzondering van de WBSO regeling, waarmee R&D intensieve
bedrijven korting krijgen op de loonheffing. HollandBIO bepleit dat bedrijven hun
volledige WBSO korting behouden, ook wanneer corona-beperkingen onderzoek en
ontwikkeling tijdelijk stilleggen. Liever nog wordt er tijdelijk volledig
afgezien van loonheffing voor innovatieve bedrijven.
HollandBIO roept
biotech bedrijven op om ons te laten weten hoe zij ervoor staan en in hoeverre
de huidige noodmaatregelen soelaas bieden. Aanspreekpunt is HollandBIO’s
Wieteke. Ook voor andere vragen, uitdagingen of problemen
waarbij HollandBIO’s inzet zou helpen: laat het ons weten!
Blackstone and Alnylam Pharmaceuticals have entered into a broad strategic collaboration under which Blackstone will provide up to $2 billion to support Alnylam’s advancement of innovative RNA interference (RNAi) medicines that have the potential to transform the lives of patients suffering from a range of debilitating diseases.
The deal is anchored by Blackstone’s purchase of 50 percent of the royalties owed to Alnylam on global sales of inclisiran, an investigational RNAi therapeutic for the treatment of hypercholesterolemia, currently under review by the U.S. Food and Drug Administration. Inclisiran is a twice-a-year, subcutaneously injected RNAi therapeutic that has been shown in a comprehensive Phase 3 program to reduce low-density lipoprotein (LDL) or “bad” cholesterol with an acceptable safety profile. If approved, this medicine is expected to help patients lower LDL cholesterol, a major risk factor for cardiovascular disease, the leading cause of mortality in the U.S. and globally.
The strategic financing collaboration, led by Blackstone Life Sciences and GSO Capital Partners (“GSO”), Blackstone’s credit platform, is expected to enable Alnylam’s achievement of a self-sustainable financial profile without need for future equity financing, accelerating the commercial potential of Alnylam’s rapidly advancing product portfolio. The investment by multiple Blackstone businesses will support the development and delivery of promising medicines to the patients who need them and is one of the largest ever private financings of a biotech company.
The transaction includes the inclisiran royalty monetization, corporate debt, purchase of Alnylam equity, and funding for certain R&D activities related to the clinical advancement of two Alnylam investigational RNAi therapeutic programs in cardiovascular disease.
Specifically, the transaction is comprised of the following components:
$1 billion in committed payments, led by Blackstone Life Sciences, to acquire 50 percent of Alnylam’s royalties and commercial milestones for inclisiran;
Up to $750 million in a first lien senior secured term loan led by GSO;
Up to $150 million from Blackstone Life Sciences for development of Alnylam’s cardiometabolic programs vutrisiran and ALN-AGT (to be established based upon a non-binding letter of intent);
$100 million purchase of Alnylam common stock.
“Alnylam is focused on building a top-tier biopharmaceutical company, advancing RNAi therapeutics as a whole new class of medicines with transformative potential for patients around the world. This exciting new relationship with Blackstone brings us much closer to that goal, securing our bridge towards a self-sustainable financial profile that we believe can now be achieved without any need for Alnylam to access the equity markets in the future,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “A central component of this strategic relationship is a partial monetization of our royalty for inclisiran. If approved, we believe this therapy holds enormous promise as a potential game-changer in hypercholesterolemia management. We are pleased to retain half of the royalties we receive from Novartis, allowing Alnylam to benefit from inclisiran’s anticipated future success. We couldn’t be more pleased to enter into this highly innovative arrangement with Blackstone, which has shown a significant commitment to Alnylam’s future and alignment with our long-term vision.”
“Blackstone is uniquely positioned to provide customized, one-stop-shop financing solutions at scale while establishing development collaborations with the world’s leading biotech companies. Alnylam’s RNAi technology represents one of the most promising and rapidly advancing frontiers in biology and drug development today, and aligns perfectly with our investment strategy,” said Nicholas Galakatos, Ph.D., Global Head of Blackstone Life Sciences. “Our collaboration with Alnylam provides non-dilutive access to capital to advance important new medicines in development across several disease indications including heart disease, the leading cause of death in the U.S. and globally.”
“We’re thrilled to be able to partner with Blackstone Life Sciences to provide a customized solution for Alnylam, whose approved therapies provide important new options for patients,” said Dwight Scott, Global Head of GSO. “GSO’s capital will support Alnylam during a period of global growth as it continues to launch its medicines and pursue innovative RNAi approaches to bring new therapies to market and ultimately to patients around the globe.”
Alnylam, an existing tenant of Blackstone Real Estate company BioMed Realty, is also in discussions with BioMed to expand its footprint in Cambridge, MA.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-04-14 17:03:092020-04-14 17:03:10Blackstone and Alnylam Enter Into $2 Billion Collaboration
BIO International Convention goes BIO digital
IJzersterk innovatieklimaat, Hollandbio, InnovatieklimaatThis year, the BIO International Convention will transition to virtual event format: BIO Digital. HollandBIO members can access this largest virtual gathering of the global biotech industry at a discounted rate of $950. Read more
First MIT Covid-19 Challenge; a great success
InnovatieklimaatIn the weekend of 3 till 5 April, 1500 selected participants took part in the MIT Covid-19 Challenge – Beat the Pandemic Hackathon. They were selected out of more than 4500 applications from over 100 countries. Dutch participants were also joining team sessions in 10 different tracks.
The teams, under guidance of mentors, presented their work at the end of the weekend to judge panels that picked 40 winning teams out of 208 teams. The organization is now analyzing which teams are able to further work on their solutions and what is needed to get these solutions into practice.
You can read more about the challenge at Mobi Health News
Or watch the Beat the Pandemic Kickoff at YouTube
Synthon Biopharmaceuticals relaunches as Byondis
InnovatieklimaatSynthon Biopharmaceuticals B.V., a biopharmaceutical research and development company creating innovative precision medicines for patients with relentless cancers and autoimmune diseases, today announced that it is relaunching as Byondis.
Byondis is creating safe and effective precision medicines based on its proprietary technologies, targeting intractable cancers and auto-immune diseases, which aim to improve the standard of care for patients.
The Company has a broad development pipeline, including advanced clinical programs, such as anti-HER2 ADC [vic-]trastuzumab duocarmazine (SYD985) for breast cancer. In the second half of 2020, Byondis is expecting results from its pivotal TULIP® Phase III trial comparing SYD985 to physician’s choice treatment in patients with HER2-positive unresectable locally advanced or metastatic breast cancer. Submission of a Biological License Application is scheduled before the end of 2020.
The Company anticipates entering the clinic with four additional programs in the course of 2020/2021. After progressing to clinical proof of concept, Byondis intends to enter into partnerships for late-stage development and commercialization.
Synthon Biopharmaceuticals was established in 2007 as part of Synthon B.V., and became a separate biopharmaceutical subsidiary in 2012, quickly building a promising pipeline of innovative R&D programs. In November last year, following the acquisition of Synthon International Holding B.V. by BC Partners, the founder, Dr Jacques Lemmens, decided to continue as an independent biopharmaceutical company, with its own management team and state-of-the-art facilities. This company relaunches today as Byondis.
“Our relaunch as Byondis is an exciting moment for the Company” said Dr Jacques Lemmens, Founder. “Building on our heritage, while preserving our company culture, we are further progressing in innovation, quality and scientific excellence.”
“Now is the time to step up as an independent biopharmaceutical company, with our exceptional people, unique technologies and a well-established development pipeline, including our lead ADC, [vic-]trastuzumab duocarmazine. I am very proud of how the Company has grown and evolved. The new name, Byondis, represents the next chapter and embodies our entrepreneurial spirit and ambition to create innovative precision medicines to outsmart cancers and auto-immune diseases.”
The Byondis management team has a wealth of scientific, clinical and operational experience, and is well networked within the pharmaceutical industry. Dr Marco Timmers, CEO, and others in the senior team have collaborated for many years as part of Synthon. During this time, they have created a company driven by science and innovation, with unique technologies and significant validation deals. These include the out-licensing of a biosimilar version of the monoclonal antibody trastuzumab (marketed as KANJINTI™) in a global partnership agreement with Amgen in 2012.
“As Byondis, we can now showcase our strong business and scientific proposition”, added Dr Marco Timmers, CEO. “We have generated a portfolio of preclinical and clinical programs with the potential to deliver completely novel treatments for cancer and auto-immune diseases. We have unique technologies with differentiating potential, state-of-the-art facilities including our own GMP manufacturing, and broad licensing expertise that makes us an attractive partner for out-licensing. This is a really exciting time for Byondis and our team.”
Byondis operates from its headquarters in Nijmegen in the Netherlands, with over 350 highly qualified employees working in state-of-the art research and development and GMP manufacturing facilities. The Company has strong links to academic institutions, global medical communities and the international pharma industry.
Source: Byondis (press release)
Galapagos and Ryvu announce research collaboration
InnovatieklimaatGalapagos and Ryvu Therapeutics have announced a collaboration focused on the discovery and development of novel small molecule drugs in inflammation.
Ryvu specializes in the discovery and development of first-in-class small molecules and drug candidates in diseases with high unmet medical needs. The collaboration announced today is based on a novel drug target identified by Ryvu, which will contribute its technology platform and related intellectual property (IP). Ryvu and Galapagos will both provide resources to support the collaboration and make use of their expertise in high-throughput screening, biology, medicinal chemistry, and toxicology.
This is a joint research collaboration in which Ryvu is responsible for early drug discovery. Under the terms of the agreement, Galapagos will have an exclusive option to license IP developed by Ryvu and to continue to develop this during the collaboration. Pending achievement of pre-agreed criteria and utilizing its option, Galapagos will be responsible for all further development of the program.
In exchange for global development and commercialization rights, Ryvu will receive an upfront payment and will be eligible for further option, milestone, and royalty payments.
“We believe that the collaboration with Ryvu is an excellent fit, as both companies are driven by the search for novel drugs to address unmet medical needs,” says Dr. Piet Wigerinck, Chief Scientific Officer at Galapagos. “We look forward to collaborating with the Ryvu team to push this program forward.”
Pawel Przewiezlikowski, Ryvu Chief Executive Officer, added: “We are thrilled to start working with Galapagos, a real role model for the European biotechnology sector. Throughout joint discussions on the collaboration, our teams have developed a strong rapport and built the foundations for a very promising start for the new project on an exciting novel target.”
Source: Galapagos (press release)
Khondrion proposes potential treatment pathway COVID-19 with sonlicromanol
GezondheidKhondrion, a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease, has published a scientific hypothesis that the inflammatory lipid modulator, prostaglandin E2 (PGE2), may execute a prominent role in COVID-19 pathophysiology and proposes that its lead drug candidate, sonlicromanol, currently in phase IIb development to treat a range of mitochondrial diseases, could be repurposed for the treatment of patients with severe COVID-19 disease.
The paper, published online by Preprints, the online multidisciplinary platform dedicated to making early versions of research outputs permanently available and citable, summarizes the potential role that elevated levels of PGE2, which is known to play an essential role in inflammation1 and defense against infectious agents2, may have in COVID-19 pathology. It calls for the measurement of PGE2 in affected patients and proposes selective inhibition of a key enzyme involved in PGE2 production – microsomal prostaglandin E synthase-1 (mPGES-1) – as a potential new treatment approach in protecting COVID-19 patients from severe disease progression and death.
Human mPGES-1 is already recognized as a promising target for the next generation of antiinflammatory drugs, without the side effects of those currently available, e.g. non-steroidal antiinflammatory drugs (NSAIDs) and COX-2 inhibitors3. The Khondrion research team’s hypothesis that selective mPGES-1 inhibition might reduce COVID-19 associated disease symptoms, e.g. acute respiratory distress syndrome (ARDS), is based on PGE2’s role as an inflammatory modulator in viral infections as well as evidence that targeted PGE2 inhibition enhances antiviral immunity4,5,6.
Sonlicromanol, Khondrion’s wholly-owned investigational lead asset currently in phase IIb development as a potentially disease-modifying treatment for mitochondrial disease, has a triple mode of action that includes the inhibition of mPGES-1 and may, in turn, result in an antiinflammatory effect. Khondrion research using human fibroblast cells and a mouse macrophage-like cell line shows that sonlicromanol is able to block mPGES-1 and decrease PGE2 production. Therefore, in addition to it being a novel therapeutic option for mitochondrial disease patients, the Company proposes that sonlicromanol may also have potential to be repurposed to treat PGE2- driven inflammatory consequences that might underly COVID-19 associated ARDS or, when administered early after diagnosis, might prevent progression to ARDS.
Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: “During the development of sonlicromanol for the treatment of mitochondrial disease we have generated a wealth of data on its mode of action, including its effects on the body’s inflammatory responses. What we have learned about this asset’s potential is striking when examined through the lens of COVID-19 and the scientific community’s search for new therapies as part of the global response to this pandemic. “While there remain limitations in our understanding of PGE2 and its role in the serious lung disease associated with COVID-19, we believe it warrants further investigation, particularly when new therapeutic options are so desperately needed. “We are inviting experts in the field of coronavirus to test our compound in-vitro and in-vivo. We are also actively searching for partners willing to assist us in evaluating our hypothesis and the potential of bringing sonlicromanol to COVID-19 patients.”
The paper “Hypothesis: mPGES-1-derived Prostaglandin E2, a so far missing link in COVID-19 pathophysiology?” is available on Preprints here.
Source: Khondrion (press release)
LiteVax receives EU Horizon 2020 grant
InnovatieklimaatA consortium of public and private R&D organisations from EU, India, and US has been awarded a 10 m€ grant for development of improved influenza virus vaccines for the globe. Later this year, this grant will be matched by the government of India. LiteVax BV (Oss, The Netherlands) and the Translational Health Science Technology Institute (Delhi, India) have initiated the project and the University of Amsterdam will be the coordinator. The INDIGO project builds on LiteVax’s patented, versatile adjuvant technology developed to improve prophylactic and therapeutic vaccines.
The grant will support the development of a next generation of influenza vaccines aiming at higher effectivity, lower costs and better accessibility. The INDIGO project combines innovations in antigen design, antigen delivery and immune-stimulation and plans to perform phase I/IIa clinical trials in EU and India. In addition to LiteVax, the INDIGO consortium consists of one Danish partner, two Belgian partners, three French partners, two Dutch partners, one US partner, and six Indian partners. An important goal is the collaboration between R&D organizations in these countries with complementary skills and know-how. See https://cordis.europa.eu/project/id/874653 for more information.
Source: LiteVax (press release)
Pharvaris oral HAE drug shows positive results
GezondheidPharvaris, a clinical-stage company focused on the discovery and development of novel oral B2-receptor antagonists for the treatment of hereditary angioedema (HAE) and other B2-receptor-mediated indications, announced data from a late-breaking poster planned for presentation at the recently canceled annual meeting of the American Academy of Allergy, Asthma & Immunology (AAAAI).
Pharvaris established a proof-of-concept model for HAE in non-human primates. In this model, PHA121 inhibited bradykinin-induced changes in blood pressure at all doses tested (0.1, 0.3, 1, 3, and 10 mg/kg given orally) with a faster onset of action than icatibant. The duration of the effect was dose dependent.
“These results provide confidence in PHA121’s clinical profile for the treatment of hereditary angioedema,” said Berndt Modig, Chief Executive Officer and co-founder of Pharvaris. “Combined with preliminary findings from our ongoing Phase 1 study, these data demonstrate that PHA121 is a potent, orally available bradykinin B2 receptor antagonist.”
The objective of the study was to investigate the ability of PHA121 to attenuate blood-pressure changes induced by bradykinin injection. Freely moving monkeys were challenged with bradykinin, resulting in a transient drop in blood pressure. Bradykinin is an endogenous peptide known to mediate signs and symptoms of HAE. The model was validated utilizing icatibant, a marketed injectable B2 receptor antagonist, providing back-translation from human clinical experience with icatibant.
The abstract of the poster was published in the Journal of Allergy and Clinical Immunology (https://doi.org/10.1016/j.jaci.2019.12.094). The poster is available on the AAAAI website (https://education.aaaai.org/sites/default/files/L37%20Lesage_1.pdf) and on the Pharvaris website.
Source: Pharvaris (press release)
Drastische versnelling vergunningaanvraag medische GGO’s, nu door voor de kopgroep
Gezondheid op maat, Wereld mét toekomst, Hollandbio, Gezondheid, UitgelichtEen aanvraag voor klinisch onderzoek met bepaalde vormen van cel- en gentherapie duurt in Nederland straks nog maar 56 dagen. Daarnaast gaan de informatievereisten omlaag. Dit blijkt uit het voorstel van minister Van Nieuwenhuizen voor een wijziging van het besluit GGO. De wijziging geldt voor een aantal toepassingen, waarvoor de aanvraagperiode tot voor kort vaak langer dan een jaar in beslag nam. HollandBIO is blij dat de wensen van het veld ter harte worden genomen, maar pleit ook voor verdere verbeteringen. Alleen dan maken we Nederland ècht internationaal toonaangevend.
In het voorstel is een snellere route van maximaal 56 dagen met verlaagde informatievereisten terug te vinden voor toepassingen die zijn gebaseerd op AAV-vectoren en ex vivo getransduceerde cellen vervaardigd met retrovirale of lentivirale vectoren. Ook biedt het voorstel de ruimte voor kopievergunningen, waarbij aanvragers een eerdere aanvraag als uitgangspunt kunnen nemen.
Hoewel dit een verbetering is, ziet HollandBIO mogelijkheden om Nederland bij de internationale kopgroep aan te laten sluiten. We zetten ons daarom in voor de volgende punten:
Minister Van Nieuwenhuizen heeft het voorstel ter wijziging van Besluit GGO inmiddels naar de Tweede Kamer gestuurd, die tot 3 juni de tijd krijgt voor inspraak voordat zij overgaat tot de inhoudelijke behandeling. Het wijzigingsvoorstel is ook gepubliceerd in de Staatscourant, waarmee iedereen die dat wil binnen 4 weken een reactie kan geven.
Benieuwd naar het voorstel tot wijziging van Besluit GGO? Lees hier de Kamerbrief en hier het voorstel.
Help biotech sector door coronacrisis
IJzersterk innovatieklimaat, Hollandbio, Innovatieklimaat, UitgelichtDe economische gevolgen van het coronavirus stellen biotech start-ups en scale-ups voor grote uitdagingen. HollandBIO maakt zich hard voor ondersteuning toegesneden op de specifieke behoeften van de biotech sector. Op 7 april presenteerde het kabinet aanvullende maatregelen om ondernemers te ondersteunen. HollandBIO is blij met deze eerste stap, maar blijft benadrukken dat verdergaande en meer duurzame ondersteuning nodig is voor de Nederlandse biotech sector. Niet alleen is de sector cruciaal voor het toekomstig verdienvermogen van Nederland, vandaag de dag blijkt de innovatiekracht van de sector op alle fronten harder nodig dan ooit.
Overbruggingskrediet
Het kabinet maakt 100 miljoen euro vrij voor overbruggingskredieten voor non-bancair gefinancierde bedrijven. Kredieten tot een maximum van 2 miljoen euro worden verstrekt door regionale ontwikkelingsorganisaties (ROM’s), Invest-NL krijgt een rol voor hogere bedragen. Ook werken het ministerie van Economische Zaken en Klimaat (EZK), de ROM’s, Invest-NL en Techleap aan een gezamenlijk programma gericht op het verstrekken van overbruggingskrediet in de vorm van (converteerbare) leningen aan innovatieve start- en scale-ups. HollandBIO bepleit de snelle, eenvoudige en brede beschikbaarheid van converteerbare kredieten om biotech bedrijven in acute financiële nood te helpen.
Uitstel betaling Innovatiekrediet
In haar brief aan de Tweede Kamer kondigt het kabinet uitstel van betaling voor Vroege Fase Financiering (VVF) en het Innovatiekrediet aan, van maximaal 6 maanden. HollandBIO vindt dit echter onvoldoende. Met deze maatregel wordt de rekening slechts uitgesteld, terwijl die door rente op rente wel fors oploopt. Een tijdelijke rentestop, tot twee jaar uitstel van afbetalingen en een generieke en retrospectieve renteaanpassing geeft ondernemers meer lucht in deze moeilijke tijd. Op verzoek van vele bedrijven in onze achterban, bespreekt HollandBIO verdere mogelijkheden met RVO en EZK. Hierbij vragen we ook aandacht voor soepele honorering van aanvragen voor nieuwe Innovatiekredieten.
WBSO
Biotech bedrijven profiteren zonder uitzondering van de WBSO regeling, waarmee R&D intensieve bedrijven korting krijgen op de loonheffing. HollandBIO bepleit dat bedrijven hun volledige WBSO korting behouden, ook wanneer corona-beperkingen onderzoek en ontwikkeling tijdelijk stilleggen. Liever nog wordt er tijdelijk volledig afgezien van loonheffing voor innovatieve bedrijven.
HollandBIO roept biotech bedrijven op om ons te laten weten hoe zij ervoor staan en in hoeverre de huidige noodmaatregelen soelaas bieden. Aanspreekpunt is HollandBIO’s Wieteke. Ook voor andere vragen, uitdagingen of problemen waarbij HollandBIO’s inzet zou helpen: laat het ons weten!
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Blackstone and Alnylam Enter Into $2 Billion Collaboration
Gezondheid, InnovatieklimaatBlackstone and Alnylam Pharmaceuticals have entered into a broad strategic collaboration under which Blackstone will provide up to $2 billion to support Alnylam’s advancement of innovative RNA interference (RNAi) medicines that have the potential to transform the lives of patients suffering from a range of debilitating diseases.
The deal is anchored by Blackstone’s purchase of 50 percent of the royalties owed to Alnylam on global sales of inclisiran, an investigational RNAi therapeutic for the treatment of hypercholesterolemia, currently under review by the U.S. Food and Drug Administration. Inclisiran is a twice-a-year, subcutaneously injected RNAi therapeutic that has been shown in a comprehensive Phase 3 program to reduce low-density lipoprotein (LDL) or “bad” cholesterol with an acceptable safety profile. If approved, this medicine is expected to help patients lower LDL cholesterol, a major risk factor for cardiovascular disease, the leading cause of mortality in the U.S. and globally.
The strategic financing collaboration, led by Blackstone Life Sciences and GSO Capital Partners (“GSO”), Blackstone’s credit platform, is expected to enable Alnylam’s achievement of a self-sustainable financial profile without need for future equity financing, accelerating the commercial potential of Alnylam’s rapidly advancing product portfolio. The investment by multiple Blackstone businesses will support the development and delivery of promising medicines to the patients who need them and is one of the largest ever private financings of a biotech company.
The transaction includes the inclisiran royalty monetization, corporate debt, purchase of Alnylam equity, and funding for certain R&D activities related to the clinical advancement of two Alnylam investigational RNAi therapeutic programs in cardiovascular disease.
Specifically, the transaction is comprised of the following components:
“Alnylam is focused on building a top-tier biopharmaceutical company, advancing RNAi therapeutics as a whole new class of medicines with transformative potential for patients around the world. This exciting new relationship with Blackstone brings us much closer to that goal, securing our bridge towards a self-sustainable financial profile that we believe can now be achieved without any need for Alnylam to access the equity markets in the future,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “A central component of this strategic relationship is a partial monetization of our royalty for inclisiran. If approved, we believe this therapy holds enormous promise as a potential game-changer in hypercholesterolemia management. We are pleased to retain half of the royalties we receive from Novartis, allowing Alnylam to benefit from inclisiran’s anticipated future success. We couldn’t be more pleased to enter into this highly innovative arrangement with Blackstone, which has shown a significant commitment to Alnylam’s future and alignment with our long-term vision.”
“Blackstone is uniquely positioned to provide customized, one-stop-shop financing solutions at scale while establishing development collaborations with the world’s leading biotech companies. Alnylam’s RNAi technology represents one of the most promising and rapidly advancing frontiers in biology and drug development today, and aligns perfectly with our investment strategy,” said Nicholas Galakatos, Ph.D., Global Head of Blackstone Life Sciences. “Our collaboration with Alnylam provides non-dilutive access to capital to advance important new medicines in development across several disease indications including heart disease, the leading cause of death in the U.S. and globally.”
“We’re thrilled to be able to partner with Blackstone Life Sciences to provide a customized solution for Alnylam, whose approved therapies provide important new options for patients,” said Dwight Scott, Global Head of GSO. “GSO’s capital will support Alnylam during a period of global growth as it continues to launch its medicines and pursue innovative RNAi approaches to bring new therapies to market and ultimately to patients around the globe.”
Alnylam, an existing tenant of Blackstone Real Estate company BioMed Realty, is also in discussions with BioMed to expand its footprint in Cambridge, MA.
Source: Alnylam