Khondrion, a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that the European Commission has granted Orphan Drug Designation (ODD) to its lead asset, KH176, for the treatment of patients with maternally inherited diabetes and deafness (MIDD). MIDD is a subtype of diabetes often accompanied by hearing loss that is caused by a mutation within mitochondrial DNA.
KH176, Khondrion’s wholly-owned lead asset is a potential first-in-class oral small molecule in phase IIb clinical development to treat a range of mitochondrial diseases. The compound, which belongs to a new class of drugs used to control oxidative and redox pathologies, has previously been granted ODDs for MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders and Leigh disease by the EMA, and for all inherited mitochondrial respiratory chain disorders by the US Food and Drug Administration (FDA).
An orphan designation allows a pharmaceutical company to benefit from incentives to develop a medicine for a rare disease, such as reduced fees in protocol assistance and a period of market exclusivity following regulatory approval. European applications for Orphan Designation are examined by the Committee for Medicinal Products (COMP), which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question.
Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: “MIDD is a rare and extremely debilitating form of diabetes with a critical need for new treatments. This designation is an important incentive for companies like Khondrion investing in rare disease research and brings us another step closer to advancing the science of mitochondrial disease for the benefit of patients.”
HollandBIO connects and supports the Dutch
biotech industry. We represent over 200 companies in the field of medical,
agrifood and industrial biotech. And, HollandBIO is growing steadily. Today, we
are proud to welcome AGILeBiotics to the HollandBIO community!
AGILeBiotics is a spin-off company of the
University of Groningen founded by the CEO Dr. Andreas A. Bastian in March
2017. The start-up is committed to contribute to the fight against bacterial
resistance by bringing new therapeutic solutions to hospitalized patients. The
company’s vision is to accelerate the development of novel antibiotics by
employing a proprietary technology named OxaSelect on existing complex natural
product-based antibiotics, which have lower risk for drug resistance. The
synthetic technology gives quick access to next generation drugs with
broad-spectrum activity and improved safety profile.
AGILeBiotics is about to enter the pre-clinical
stage. Their pre-clinical candidates overcome drug-class related bacterial
resistance and have high potency against multidrug-resistant clinical isolates
belonging to the top five pathogens of WHO’s priority list. AGILeBiotics’ goal
is to develop novel antibiotics for the treatment of infections with high death
rate, such as ventilator-associated pneumonia (VAP) and bloodstream infections
(BSI), and neonatal sepsis.
Since 2017, AGILeBiotics has successfully
enquired the pre-seed and seed investment round enabling the identification of
their pre-clinical candidates. Currently, AGILeBiotics is reaching out for
Series A investment round to develop the antibiotic candidate until clinical
Phase II.
For more information please visit AGILeBiotics website.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-07-09 14:07:372019-07-09 14:16:49HollandBIO welcomes AGILeBiotics as a new member← #nieuws
SMS-ONCOLOGY, a full-service clinical CRO solely dedicated to oncology, is pleased to announce the opening of a new office and the establishment or subsidiary company: SMS-oncology GmbH.
SMS-oncology, Europe’s leading oncology CRO, opened the doors of its new office in Frankfurt, Germany. SMS-oncology GmbH is ideally situated in one of the major oncology biotech hubs within Europe and in the vicinity of Frankfurt Airport. SMS-oncology operates in over 20 countries from its headquarters at Schiphol Airport in the Netherlands, and has established local presence in the Nordics, Spain and now Germany. The new office accommodates the growing team of oncology drug development specialists.
The new German entity SMS-oncology GmbH will be led by Managing Director Mr. Raymond Hoffmans, who has 15 years of experience in cancer research. After receiving his PhD from the University of Zurich (Switzerland), he joined the Dana Farber Cancer Institute in Boston (US) as a postdoctoral research fellow. In 2009, he returned to the Netherlands to join the Hubrecht Institute in Utrecht, before he moved to SMS oncology about 9 years ago. Since 2014, Raymond has been Chief Business Development Officer at SMS-oncology, a position he will combine with his new role of Managing Director of the GmbH.
Raymond: “We are very proud and pleased to announce the opening of our new office in Frankfurt. We experienced strong organic growth over the past years. The current opening of our GmbH office is the perfect next step in line with our business planning of international expansion.”
He continues: “The expansion will not only allow us to be closer to our clients and the medical sites we work with but will also allow us to provide more tailored solutions to the individual needs of our clients.”
Ms. Philine van den Tol, Chief Executive Officer of SMS oncology, said: “We are delighted to bring the company one step closer in achieving our ambitious goal of becoming the go-to oncology specialist CRO in the world. This is another step forward in our mission to excel in oncology trials for the benefit of our clients and cancer patients worldwide.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-07-08 15:13:322019-07-09 15:16:34SMS-oncology opens new office in Frankfurt, Germany← #nieuws
The Dutch
Biotech Event 2019 had it all: a great line-up, over 250 eager biotech
professionals, a lovely venue and perfect weather.
During her
opening speech, Annemiek Verkamman brought the festive news that HollandBIO
welcomed its 200th member! Our growing member base strengthens us in our mission
to maximize the contribution of biotech products to our society’s health and
sustainability. And it shows that HollandBIO’s impact is bigger than ever. Of
course, HollandBIO will continue what we do best: paving the way for biotech’s frontrunners
by challenging the status quo.
A three decade journey in cell therapy – Arie
Belldegrun
Arie
Belldegrun, our first keynote speaker, took us on a dazzling, three-decade
journey in cell therapy. Arie played an instrumental role in several of today’s
frontrunners in biotech, such as Kite Pharma, bringing ground-breaking science
from bench to bedside. And, as he is convinced that science drives innovation, Arie
is always looking for the next revolution. Today, he serves as Executive
Chairman & Co-Founder of Allogene Therapeutics. The company’s Allogenic
CAR-T therapy may be the next big breakthrough, since more patients can be
treated with engineered T-cells after a single manufacturing run. Although no
one can predict the future, Arie firmly believes cell therapy holds great
promise for cancer therapy. What is it that keeps him going after so many
successes? It is the great fun along the way. No better way to conclude a
fantastic story of a true biotech pioneer!
AmbAgon wins Venture Challenge Spring 2019
Chrétien Herben, Director at LifeSciences@Work, announced AmbAgon as the winner of the Venture Challenge Spring 2019 edition. The Venture Challenge is a 10-week programme to develop an initial idea – a breakthrough research result – into a solid business case. This edition’s winners AmbAgon aims to develop small-molecule therapeutics for breast cancer by augmenting the tumor-suppressing functions of the 14-3-3 protein. The prize includes a cheque worth €25.000.
Personal perspectives on a career in biotech – Tim
Knotnerus
“It’s not
about where you work, nor what you do, it’s who you work with.” And Tim
Knotnerus, our second keynote speaker, worked with the best. After graduation,
Tim joined Aescap, a VC fund established by Dinko Valerio. The experience to
work with one of the pioneers of the Dutch biotech sector, sparked Tim’s
ambition to pursue an entrepreneurial biotech career of his own. He continued
his career at AM-Pharma. Together with CEO Erik van den Berg, he secured
multiple funding rounds. An option to acquire deal with Pfizer was the cherry
on the cake. The deal also paved the way for Tim’s next big leap in biotech:
becoming CEO of AgomAb, a Belgium biotech start-up holding an exclusive license
agreement from argenx. With € 21 million secured in a series A earlier this
year, exciting times lie ahead for this ‘fourth-generation’ biotech star.
Lessons in leadership – John Haurum
Biotech owes its success to innovation. But how to create and sustain an innovative corporate culture? John Haurum’s keynote focused on the strong leadership it takes to pull that off. John’s former roles as CSO of Symphogen and CEO of F-Star not only made him an expert in the field of therapeutic antibodies, but also gave him hands-on experience in corporate cultures. As board member of a growing number of biotech companies, his knowledge is of great value to a new biotech generation. Guided by a Harvard Business Review article, John shared his vision on building an innovative biotech culture, stressing the apparent paradoxes involved: tolerance for failure but intolerance for incompetence, willingness to experiment combined with rigorous discipline and collaboration that involves individual accountability. All in all, John’s presentation provided plenty of food for thought for both established as well as up-and-coming biotech leaders.
Break-out session – Critical communication
As our
beautiful biotech sector is under bigger scrutiny than ever before, simply
being good is no longer good enough. The need to be good and tell it, to speak
up, to explain what you do and why you do it, is bigger than ever before. But
in a seemingly post-fact era, and a debate driven by emotion, that might be easier
said than done. Hugo van Rooijen (Janssen Pharmaceuticals) and Guus Scheefhals
(DegenRx) shared their communicative views, ideas and experiences. From
fact-checking as pharmacovigilance for politicians, to setting the right frame
for a constructive discussion, and managing expectations among patients and
other stakeholders. The session delivered a number of best practices to move
forward. HollandBIO hopes the attendees will also practice what these biotech
frontrunners preached!
Break-out session – Cell land gene therapy in
the spotlight
Kite Pharma
is one of the frontrunners in cell therapy, with the approval of Yescarta for treatment
of certain types of non-Hodgkin lymphoma. At the same time, gene therapy is also
booming! Guided by Kite’s Markwin Velders, two pioneering companies shared
their experiences in the gene therapy world. Rudolf van Olden (Avexis) highlighted
the development of Avexis’ Zolgensma, which got just approved by the FDA to
treat children with SMA. The results of the initial clinical trials were
spectacular, resulting in an ultra-short time to market. Thanks to the rapidly
built production site, patients in the US are already being treated. Tjarda
Kasteel from bluebird bio shared the fascinating story of Zynteglo, a one-time gene therapy for patients with ß-Thalassemia.
Based on the exceptional clinical outcomes, Zynteglo saw a ultra-fast approval by the EMA. Because both Zynteglo and Zolgensma are ‘one time’ treatments challenging the status quo,
both companies introduced correspondingly novel payment strategies. The impressive
results so far make it more than justified that cell and gene therapy are in
the spotlight right now.
Break-out session – Think different
During the
workshop Think Different, biotech entrepreneurs Wim de Laat (BioscienZ) and Jos
Seegers (MyMicroZoo) gave us a sneak peek of tomorrow’s food and how insights
in your microbiome can help you make better lifestyle choices. Wim aims to
serve a Christmas dinner with egg ingredients produced by fermentation of a
fungus, which converts sugar from sugar beet, cane or grain into ovalbumin. If
you are curious about the effect of a (G)Meal, or any other lifestyle
intervention, on your microbiome, the MyMicroZoo poo-it-yourself-test from Jos
will provide you with answers based on the most advanced DNA sequencing
techniques. HollandBIO is already looking forward to our next teambuilding
activity!
Break-out session – How to fund a biotech
A no now,
doesn’t mean a no later. That was one of the lesson’s learned by Synaffix’ CEO
Peter van de Sande. To secure funding to commercialize antibody-drug conjugates
and build a technology platform company, Peter had to knock on many doors. You
definitely need to be persistent, Xenikos CEO Ypke van Oosterhout added. Ypke’s
dedication did pay-off: Xenikos closed a €30 million series B financing round last
year. Edward van Weezel agreed as well. As a managing partner of venture fund BGV,
he sees hundreds of projects every year. The overall success rate for companies
is very low, only a few get an investment. It is worrisome that the Netherlands
is losing its strong start-up position to other countries around the globe.
Especially in the United States, huge amounts of risk capital is invested in
very young start-ups, paving the way for growth. To stay on track, the
Netherlands should invest more in innovative start-ups. Government and private
investors have to work together, to bridge the gaps in the funding landscape.
Until then, you had better take into account the panelists’ lessons learned: stay
flexible and creative along the way, and always keep looking for solutions.
Fueled by the
fascinating biotech stories and new insights, it was time to network while
enjoying a refreshing drink and tasty BBQ. A great day to celebrate what
biotech is all about: building a better world by combining top-notch science,
true entrepreneurship and a lot of fun.
Be sure to mark the last Friday of June next year in your agenda. See you next year!
Citryll announced today
the first closing of a targeted € 15 million investment round, funded by
ModiQuest B.V., BOM Brabant Ventures, BrightGene, and Curie Capital. The
proceeds will be used to progress the pre-clinical and clinical development of
Citryll’s tACPA antibodies, inhibitors of neutrophil extracellular traps (NETs)
and their formation (NETosis). tACPA interferes with the formation, function
and clearance of NETs, but downstream of the peptidylarginine deiminase (PAD)
dependant citrullination pathway. It does so by binding to N-terminal
citrullinated epitopes in histones that are essential for NET formation.
“We believe
that drugs that interfere with NETs and their production have the potential to
create game changing new treatments to prevent or treat human diseases. tACPA
targets autoimmune and other diseases such as lupus, vasculitis, pulmonary
fibrosis, rheumatoid arthritis and organ damage due to sepsis. Citryll’s
approach does not broadly target inflammation or acquired immunity, instead it
extinguishes the source of autoantigens and (NET)-derived toxic molecules in
the safest way currently possible”, says CEO Helmuth van Es.
“We welcome the investment by this investor syndicate
from the Netherlands and China, which will allow the development of Citryll’s
antibody therapeutic for patients with significant medical needs”, says
ModiQuest CEO, co-inventor of tACPA, Jos Raats.
“We are excited to be able to invest in Citryll as the company is an excellent example of a first in class therapeutic innovation that can have a strong impact on patients’ lives. An increasing amount of scientific data shows that NET formation plays a crucial role across various disease types, therefore we look forward to support the further development of tACPA”, says BOM Brabant Ventures Investment Manager Sjoerd van Gorp, speaking for the investor syndicate.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-07-03 15:03:162019-07-09 15:05:23Citryll Completes First Closing of a € 15M Series A to Develop its NETosis Inhibiting Antibody← #nieuws
HollandBIO connects and supports the Dutch biotech industry. We represent 200 companies in the field of medical, agrifood and industrial biotech. And, HollandBIO is growing steadily. Today, we are proud to announce that iClusion joined the HollandBIO community!
Clinical trials don’t just advance science; they also serve as in-the-moment treatment alternatives for patients. iClusion aims to make the clinical trial process faster, more effective, and more accessible for cancer patients and oncologists. “Our Trial Eye platform connects study sponsors, hospitals, physicians, and their patients in a closed-loop ecosystem,” says CEO Hanneke Janssen. Rather than each group remaining siloed, iClusion’s clinical study portal invites various stakeholders to connect. “This speeds up clinical trial accrual and therefore the entire drug development process.”
iClusion invites innovative Dutch
Biotech to start their clinical trials through iClusion’s Trial Eye, which is
already possible in the Netherlands and Belgium and keeps expanding. Instead of
relying on proven giants, which are slow no matter what they claim, they can
now rely on the fast and flexible Trial Eye services of iClusion for study
startup and patient recruitment.
iClusion believes its new model is nothing short of revolutionary. “This is the only initiative designed from the viewpoint of the 2 most important stakeholders within clinical trials; the patient and their treating physician. If they have easy access to clinical trials and can participate, drug development can speed up,” Janssen says. “The footprint footprint iClusion wants to leave on this planet is changing the lives of cancer patients.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-07-02 19:54:492019-07-02 20:12:28HollandBIO welcomes iClusion as a new member← #nieuws
HollandBIO connects and supports the Dutch biotech industry. We represent 200 companies in the field of medical, agrifood and industrial biotech. And, HollandBIO is growing steadily. Today, we are proud to announce that Oncode joined the HollandBIO community!
Oncode
wants to drive breakthrough innovations in the diagnosis and treatment of
cancer. The ultimate goal is to enable patients to survive cancer, improve the
quality of life for those affected by cancer and contribute to a more
affordable health care system. To put it simple: outsmarting cancer, impacting
lives.
To do so,
Oncode has joined the forces of over 800 molecular cancer researchers in 62 research groups across 12 universities and research
institutes in The Netherlands. Our scientists work under a common strategy, with six defined research themes.
We break with the competitive model of science and facilitate collaboration
between scientists and with the private sector.
Societal
and economic impact is a critical component of Oncode’s unique approach. We
work hard to make sure that new findings from the lab, find their way to
patients in the most optimal way. Academic research results manifest themselves
as intellectual assets in the form of people, scientific publications,
know-how, data, research tools, biological materials, software and inventions.
Oncode aims to accelerate the development of such discoveries into clinical and
economic benefits.
Their
dedicated team of business development
specialists
forms a single point of contact for both our research community and the life
science ecosystem in The Netherlands and abroad. We aim to create value for the
Dutch life sciences sector by investing in the development of new technologies
and products, building close interactions with industry to develop durable
partnerships, attract international investments and research funding and by
creating, growing and retaining oncology companies in the Netherlands.
Oncode is
funded by the Dutch Cancer Foundation, together with the Dutch ministry of
Economic Affairs & Climate Policy, the ministry of Education Culture &
Science, the ministry of Health, Welfare & Sport, Health~Holland, NWO and
ZonMw with a total of €120 million until 2022. This long term funding allows
our scientists to take steps into uncharted territory and start high risk –
high gain projects. We believe that this approach will lead to new paradigms
for treating and diagnosing cancer.
Do you want
to learn more about the possibilities to collaborate with Oncode, please reach
out to Yuva Oz
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-07-02 19:51:422019-07-02 20:01:07HollandBIO welcomes Oncode as a new member← #nieuws
Kiadis, a clinical-stage biopharmaceutical company, today announced that it has received feedback from the European Medicines Agency (EMA) related to the marketing authorization application (MAA) seeking approval of ATIR101 in hematopoietic stem cell transplant (HSCT). As previously announced, Kiadis submitted responses to the second Day 180 list of outstanding issues for ATIR101 to EMA in May 2019.
As part of the review process, EMA today informed Kiadis that it will convene a Scientific Advisory Group (SAG) in September, comprising of experts in hematology and HSCT to assist EMA in arriving at a determination.
“We appreciate the feedback from EMA and look forward to continuing our interaction with them during the review of our MAA for ATIR101,” said Arthur Lahr, CEO, of Kiadis Pharma. “With the SAG meeting now preceding the subsequent CAT and CHMP meetings as part of the EMA approval process, we are changing our guidance to potential EU conditional approval in 2020.”
While initial launch in the EU is delayed, Kiadis is on track to complete enrollment in the Phase 3 ATIR101 HSCT study in 2021 to support potential marketing approval in the US. Additionally, the company is also on track to start the Phase 1/2 studies of CSDT002 in HSCT and relapse and refractory acute myeloid leukemia in 2020. With a novel cell-based cancer immunotherapy platform consisting of both T-cell and NK-cell technologies, Kiadis has the opportunity to potentially revolutionize transplants and develop novel cancer cell therapies.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-07-02 19:44:262019-07-02 19:44:28Kiadis updates status of marketing authorization application for ATIR101← #nieuws
Gilead today announced that at a recent pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA), the company provided an update about the investigational oral, selective JAK1 inhibitor, filgotinib. The company discussed with the agency the Phase 3 FINCH studies, as well as the ongoing Phase 2 MANTA safety study assessing semen parameters with filgotinib treatment in men with moderately to severely active ulcerative colitis or Crohn’s disease. As a result of this discussion, a path forward has been established to submit the NDA for filgotinib as a treatment for rheumatoid arthritis in 2019.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-07-02 19:01:472019-07-02 19:01:50Gilead to submit new drug application for filgotinib to FDA← #nieuws
Staatssecretaris Blokhuis wil gezondheidswinst door vaccinaties maximaal verzilveren, zo liet hij de Tweede Kamer afgelopen week in een uitgebreide Kamerbrief weten. De Kamerbrief meldt dat de daling van de vaccinatiegraad tot staan is gebracht. Volgens Blokhuis geen reden om op zijn lauweren te rusten: “dit motiveert mij des te meer om onverminderd in te zetten op de maatregelen uit mijn brief Verder met vaccineren”. We roemden al eerder de doortastende aanpak van Blokhuis op het vaccinatiedossier. En ook nu weer is HollandBIO blij met de ambitie die uit de brief spreekt. We zetten de hoogtepunten uit de brief graag voor je op een rij.
Pneumokokkenvaccinatie voor ouderen
Vanaf het najaar van 2020 gaat de overheid ouderen wapenen tegen de pneumokokken. De Staatssecretaris neemt nu definitief het advies van de Gezondheidsraad over om 60, 65, 70 en 75 jarigen een vaccinatie aan te bieden. In eerste instantie mogen gegadigden zich bij de huisarts melden voor deze prik. Of dit de best passende aanpak is, zal na twee jaar uit een evaluatie blijken.
Werkdruk bedreigt voorspoedige
implementatie van nieuwe vaccins
De werkdruk bij de jeugdgezondheidszorg (JGZ) is hoog, zo
constateert Blokhuis. En dankzij de implementatie van diverse nieuwe vaccins,
waarvoor de JGZ verantwoordelijk is, zal die niet snel afnemen. “De praktijk
kan geen oneindige hoeveelheid wijzigingen tegelijkertijd aan”, concludeert Blokhuis.
En dus liggen vertragingen bij invoer op de loer. Volgens HollandBIO is er maar
één structurele oplossing: extra capaciteit en middelen. Op die manier kan de
werkdruk worden verlicht en vinden al die vaccins snel hun weg naar de juiste
arm.
Een verbeterde
communicatiestrategie
Blokhuis wil zich nog duidelijker uitspreken vóór het
belang van vaccinatie. Op verzoek van de Tweede Kamer gaat hierbij extra
aandacht uit naar ervaringsverhalen. Die krijgen dan ook een prominente plek op
een te ontwikkelen website, waar jong en oud terecht kunnen voor informatie en
actualiteiten over vaccineren. Het RIVM werkt intussen aan een vergrote
zichtbaarheid van de al bestaande website over vaccinatie. Tot slot zet de
staatssecretaris extra middelen in voor aanvullende gesprekstrainingen van
professionals.
Khondrion granted Orphan Drug Designation
GezondheidKhondrion, a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that the European Commission has granted Orphan Drug Designation (ODD) to its lead asset, KH176, for the treatment of patients with maternally inherited diabetes and deafness (MIDD). MIDD is a subtype of diabetes often accompanied by hearing loss that is caused by a mutation within mitochondrial DNA.
KH176, Khondrion’s wholly-owned lead asset is a potential first-in-class oral small molecule in phase IIb clinical development to treat a range of mitochondrial diseases. The compound, which belongs to a new class of drugs used to control oxidative and redox pathologies, has previously been granted ODDs for MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders and Leigh disease by the EMA, and for all inherited mitochondrial respiratory chain
disorders by the US Food and Drug Administration (FDA).
An orphan designation allows a pharmaceutical company to benefit from incentives to develop a medicine for a rare disease, such as reduced fees in protocol assistance and a period of market exclusivity following regulatory approval. European applications for Orphan Designation are examined by the Committee for Medicinal Products (COMP), which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question.
Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: “MIDD is a rare and extremely debilitating form of diabetes with a critical need for new treatments. This designation is an important incentive for companies like Khondrion investing in rare disease research and brings us another step closer to advancing the science of mitochondrial disease for the benefit of patients.”
Source: Khondrion
HollandBIO welcomes AGILeBiotics as a new member
HollandbioHollandBIO connects and supports the Dutch biotech industry. We represent over 200 companies in the field of medical, agrifood and industrial biotech. And, HollandBIO is growing steadily. Today, we are proud to welcome AGILeBiotics to the HollandBIO community!
AGILeBiotics is a spin-off company of the University of Groningen founded by the CEO Dr. Andreas A. Bastian in March 2017. The start-up is committed to contribute to the fight against bacterial resistance by bringing new therapeutic solutions to hospitalized patients. The company’s vision is to accelerate the development of novel antibiotics by employing a proprietary technology named OxaSelect on existing complex natural product-based antibiotics, which have lower risk for drug resistance. The synthetic technology gives quick access to next generation drugs with broad-spectrum activity and improved safety profile.
AGILeBiotics is about to enter the pre-clinical stage. Their pre-clinical candidates overcome drug-class related bacterial resistance and have high potency against multidrug-resistant clinical isolates belonging to the top five pathogens of WHO’s priority list. AGILeBiotics’ goal is to develop novel antibiotics for the treatment of infections with high death rate, such as ventilator-associated pneumonia (VAP) and bloodstream infections (BSI), and neonatal sepsis.
Since 2017, AGILeBiotics has successfully enquired the pre-seed and seed investment round enabling the identification of their pre-clinical candidates. Currently, AGILeBiotics is reaching out for Series A investment round to develop the antibiotic candidate until clinical Phase II.
For more information please visit AGILeBiotics website.
SMS-oncology opens new office in Frankfurt, Germany
InnovatieklimaatSMS-ONCOLOGY, a full-service clinical CRO solely dedicated to oncology, is pleased to announce the opening of a new office and the establishment or subsidiary company: SMS-oncology GmbH.
SMS-oncology, Europe’s leading oncology CRO, opened the doors of its new office in Frankfurt, Germany. SMS-oncology GmbH is ideally situated in one of the major oncology biotech hubs within Europe and in the vicinity of Frankfurt Airport. SMS-oncology operates in over 20 countries from its headquarters at Schiphol Airport in the Netherlands, and has established local presence in the Nordics, Spain and now Germany. The new office accommodates the growing team of oncology drug development specialists.
The new German entity SMS-oncology GmbH will be led by Managing Director Mr. Raymond Hoffmans, who has 15 years of experience in cancer research. After receiving his PhD from the University of Zurich (Switzerland), he joined the Dana Farber Cancer Institute in Boston (US) as a postdoctoral research fellow. In 2009, he returned to the Netherlands to join the Hubrecht Institute in Utrecht, before he moved to SMS oncology about 9 years ago. Since 2014, Raymond has been Chief Business Development Officer at SMS-oncology, a position he will combine with his new role of Managing Director of the GmbH.
Raymond: “We are very proud and pleased to announce the opening of our new office in Frankfurt. We experienced strong organic growth over the past years. The current opening of our GmbH office is the perfect next step in line with our business planning of international expansion.”
He continues: “The expansion will not only allow us to be closer to our clients and the medical sites we work with but will also allow us to provide more tailored solutions to the individual needs of our clients.”
Ms. Philine van den Tol, Chief Executive Officer of SMS oncology, said: “We are delighted to bring the company one step closer in achieving our ambitious goal of becoming the go-to oncology specialist CRO in the world. This is another step forward in our mission to excel in oncology trials for the benefit of our clients and cancer patients worldwide.”
SMS-oncology GmbH address:
De-Saint-Exupéry-Str. 10
60549 Frankfurt
Germany
Source: SMS-oncology
Dutch Biotech Event 2019 celebrates the beauty of biotech
Hollandbio, InnovatieklimaatThe Dutch Biotech Event 2019 had it all: a great line-up, over 250 eager biotech professionals, a lovely venue and perfect weather.
During her opening speech, Annemiek Verkamman brought the festive news that HollandBIO welcomed its 200th member! Our growing member base strengthens us in our mission to maximize the contribution of biotech products to our society’s health and sustainability. And it shows that HollandBIO’s impact is bigger than ever. Of course, HollandBIO will continue what we do best: paving the way for biotech’s frontrunners by challenging the status quo.
A three decade journey in cell therapy – Arie Belldegrun
Arie Belldegrun, our first keynote speaker, took us on a dazzling, three-decade journey in cell therapy. Arie played an instrumental role in several of today’s frontrunners in biotech, such as Kite Pharma, bringing ground-breaking science from bench to bedside. And, as he is convinced that science drives innovation, Arie is always looking for the next revolution. Today, he serves as Executive Chairman & Co-Founder of Allogene Therapeutics. The company’s Allogenic CAR-T therapy may be the next big breakthrough, since more patients can be treated with engineered T-cells after a single manufacturing run. Although no one can predict the future, Arie firmly believes cell therapy holds great promise for cancer therapy. What is it that keeps him going after so many successes? It is the great fun along the way. No better way to conclude a fantastic story of a true biotech pioneer!
AmbAgon wins Venture Challenge Spring 2019
Chrétien Herben, Director at LifeSciences@Work, announced AmbAgon as the winner of the Venture Challenge Spring 2019 edition. The Venture Challenge is a 10-week programme to develop an initial idea – a breakthrough research result – into a solid business case. This edition’s winners AmbAgon aims to develop small-molecule therapeutics for breast cancer by augmenting the tumor-suppressing functions of the 14-3-3 protein. The prize includes a cheque worth €25.000.
Personal perspectives on a career in biotech – Tim Knotnerus
“It’s not about where you work, nor what you do, it’s who you work with.” And Tim Knotnerus, our second keynote speaker, worked with the best. After graduation, Tim joined Aescap, a VC fund established by Dinko Valerio. The experience to work with one of the pioneers of the Dutch biotech sector, sparked Tim’s ambition to pursue an entrepreneurial biotech career of his own. He continued his career at AM-Pharma. Together with CEO Erik van den Berg, he secured multiple funding rounds. An option to acquire deal with Pfizer was the cherry on the cake. The deal also paved the way for Tim’s next big leap in biotech: becoming CEO of AgomAb, a Belgium biotech start-up holding an exclusive license agreement from argenx. With € 21 million secured in a series A earlier this year, exciting times lie ahead for this ‘fourth-generation’ biotech star.
Lessons in leadership – John Haurum
Biotech owes its success to innovation. But how to create and sustain an innovative corporate culture? John Haurum’s keynote focused on the strong leadership it takes to pull that off. John’s former roles as CSO of Symphogen and CEO of F-Star not only made him an expert in the field of therapeutic antibodies, but also gave him hands-on experience in corporate cultures. As board member of a growing number of biotech companies, his knowledge is of great value to a new biotech generation. Guided by a Harvard Business Review article, John shared his vision on building an innovative biotech culture, stressing the apparent paradoxes involved: tolerance for failure but intolerance for incompetence, willingness to experiment combined with rigorous discipline and collaboration that involves individual accountability. All in all, John’s presentation provided plenty of food for thought for both established as well as up-and-coming biotech leaders.
Break-out session – Critical communication
As our beautiful biotech sector is under bigger scrutiny than ever before, simply being good is no longer good enough. The need to be good and tell it, to speak up, to explain what you do and why you do it, is bigger than ever before. But in a seemingly post-fact era, and a debate driven by emotion, that might be easier said than done. Hugo van Rooijen (Janssen Pharmaceuticals) and Guus Scheefhals (DegenRx) shared their communicative views, ideas and experiences. From fact-checking as pharmacovigilance for politicians, to setting the right frame for a constructive discussion, and managing expectations among patients and other stakeholders. The session delivered a number of best practices to move forward. HollandBIO hopes the attendees will also practice what these biotech frontrunners preached!
Break-out session – Cell land gene therapy in the spotlight
Kite Pharma is one of the frontrunners in cell therapy, with the approval of Yescarta for treatment of certain types of non-Hodgkin lymphoma. At the same time, gene therapy is also booming! Guided by Kite’s Markwin Velders, two pioneering companies shared their experiences in the gene therapy world. Rudolf van Olden (Avexis) highlighted the development of Avexis’ Zolgensma, which got just approved by the FDA to treat children with SMA. The results of the initial clinical trials were spectacular, resulting in an ultra-short time to market. Thanks to the rapidly built production site, patients in the US are already being treated. Tjarda Kasteel from bluebird bio shared the fascinating story of Zynteglo, a one-time gene therapy for patients with ß-Thalassemia. Based on the exceptional clinical outcomes, Zynteglo saw a ultra-fast approval by the EMA. Because both Zynteglo and Zolgensma are ‘one time’ treatments challenging the status quo, both companies introduced correspondingly novel payment strategies. The impressive results so far make it more than justified that cell and gene therapy are in the spotlight right now.
Break-out session – Think different
During the workshop Think Different, biotech entrepreneurs Wim de Laat (BioscienZ) and Jos Seegers (MyMicroZoo) gave us a sneak peek of tomorrow’s food and how insights in your microbiome can help you make better lifestyle choices. Wim aims to serve a Christmas dinner with egg ingredients produced by fermentation of a fungus, which converts sugar from sugar beet, cane or grain into ovalbumin. If you are curious about the effect of a (G)Meal, or any other lifestyle intervention, on your microbiome, the MyMicroZoo poo-it-yourself-test from Jos will provide you with answers based on the most advanced DNA sequencing techniques. HollandBIO is already looking forward to our next teambuilding activity!
Break-out session – How to fund a biotech
A no now, doesn’t mean a no later. That was one of the lesson’s learned by Synaffix’ CEO Peter van de Sande. To secure funding to commercialize antibody-drug conjugates and build a technology platform company, Peter had to knock on many doors. You definitely need to be persistent, Xenikos CEO Ypke van Oosterhout added. Ypke’s dedication did pay-off: Xenikos closed a €30 million series B financing round last year. Edward van Weezel agreed as well. As a managing partner of venture fund BGV, he sees hundreds of projects every year. The overall success rate for companies is very low, only a few get an investment. It is worrisome that the Netherlands is losing its strong start-up position to other countries around the globe. Especially in the United States, huge amounts of risk capital is invested in very young start-ups, paving the way for growth. To stay on track, the Netherlands should invest more in innovative start-ups. Government and private investors have to work together, to bridge the gaps in the funding landscape. Until then, you had better take into account the panelists’ lessons learned: stay flexible and creative along the way, and always keep looking for solutions.
Fueled by the fascinating biotech stories and new insights, it was time to network while enjoying a refreshing drink and tasty BBQ. A great day to celebrate what biotech is all about: building a better world by combining top-notch science, true entrepreneurship and a lot of fun.
Be sure to mark the last Friday of June next year in your agenda. See you next year!
Photos by © Nils van Houts – 2019
Citryll Completes First Closing of a € 15M Series A to Develop its NETosis Inhibiting Antibody
InnovatieklimaatCitryll announced today the first closing of a targeted € 15 million investment round, funded by ModiQuest B.V., BOM Brabant Ventures, BrightGene, and Curie Capital. The proceeds will be used to progress the pre-clinical and clinical development of Citryll’s tACPA antibodies, inhibitors of neutrophil extracellular traps (NETs) and their formation (NETosis). tACPA interferes with the formation, function and clearance of NETs, but downstream of the peptidylarginine deiminase (PAD) dependant citrullination pathway. It does so by binding to N-terminal citrullinated epitopes in histones that are essential for NET formation.
“We believe that drugs that interfere with NETs and their production have the potential to create game changing new treatments to prevent or treat human diseases. tACPA targets autoimmune and other diseases such as lupus, vasculitis, pulmonary fibrosis, rheumatoid arthritis and organ damage due to sepsis. Citryll’s approach does not broadly target inflammation or acquired immunity, instead it extinguishes the source of autoantigens and (NET)-derived toxic molecules in the safest way currently possible”, says CEO Helmuth van Es.
“We welcome the investment by this investor syndicate from the Netherlands and China, which will allow the development of Citryll’s antibody therapeutic for patients with significant medical needs”, says ModiQuest CEO, co-inventor of tACPA, Jos Raats.
“We are excited to be able to invest in Citryll as the company is an excellent example of a first in class therapeutic innovation that can have a strong impact on patients’ lives. An increasing amount of scientific data shows that NET formation plays a crucial role across various disease types, therefore we look forward to support the further development of tACPA”, says BOM Brabant Ventures Investment Manager Sjoerd van Gorp, speaking for the investor syndicate.
Source: Citryll
HollandBIO welcomes iClusion as a new member
GezondheidHollandBIO connects and supports the Dutch biotech industry. We represent 200 companies in the field of medical, agrifood and industrial biotech. And, HollandBIO is growing steadily. Today, we are proud to announce that iClusion joined the HollandBIO community!
Clinical trials don’t just advance science; they also serve as in-the-moment treatment alternatives for patients. iClusion aims to make the clinical trial process faster, more effective, and more accessible for cancer patients and oncologists. “Our Trial Eye platform connects study sponsors, hospitals, physicians, and their patients in a closed-loop ecosystem,” says CEO Hanneke Janssen. Rather than each group remaining siloed, iClusion’s clinical study portal invites various stakeholders to connect. “This speeds up clinical trial accrual and therefore the entire drug development process.”
iClusion invites innovative Dutch Biotech to start their clinical trials through iClusion’s Trial Eye, which is already possible in the Netherlands and Belgium and keeps expanding. Instead of relying on proven giants, which are slow no matter what they claim, they can now rely on the fast and flexible Trial Eye services of iClusion for study startup and patient recruitment.
iClusion believes its new model is nothing short of revolutionary. “This is the only initiative designed from the viewpoint of the 2 most important stakeholders within clinical trials; the patient and their treating physician. If they have easy access to clinical trials and can participate, drug development can speed up,” Janssen says. “The footprint footprint iClusion wants to leave on this planet is changing the lives of cancer patients.
www.iclusion.com
HollandBIO welcomes Oncode as a new member
GezondheidHollandBIO connects and supports the Dutch biotech industry. We represent 200 companies in the field of medical, agrifood and industrial biotech. And, HollandBIO is growing steadily. Today, we are proud to announce that Oncode joined the HollandBIO community!
Oncode wants to drive breakthrough innovations in the diagnosis and treatment of cancer. The ultimate goal is to enable patients to survive cancer, improve the quality of life for those affected by cancer and contribute to a more affordable health care system. To put it simple: outsmarting cancer, impacting lives.
To do so, Oncode has joined the forces of over 800 molecular cancer researchers in 62 research groups across 12 universities and research institutes in The Netherlands. Our scientists work under a common strategy, with six defined research themes. We break with the competitive model of science and facilitate collaboration between scientists and with the private sector.
Societal and economic impact is a critical component of Oncode’s unique approach. We work hard to make sure that new findings from the lab, find their way to patients in the most optimal way. Academic research results manifest themselves as intellectual assets in the form of people, scientific publications, know-how, data, research tools, biological materials, software and inventions. Oncode aims to accelerate the development of such discoveries into clinical and economic benefits.
Their dedicated team of business development specialists forms a single point of contact for both our research community and the life science ecosystem in The Netherlands and abroad. We aim to create value for the Dutch life sciences sector by investing in the development of new technologies and products, building close interactions with industry to develop durable partnerships, attract international investments and research funding and by creating, growing and retaining oncology companies in the Netherlands.
Oncode is funded by the Dutch Cancer Foundation, together with the Dutch ministry of Economic Affairs & Climate Policy, the ministry of Education Culture & Science, the ministry of Health, Welfare & Sport, Health~Holland, NWO and ZonMw with a total of €120 million until 2022. This long term funding allows our scientists to take steps into uncharted territory and start high risk – high gain projects. We believe that this approach will lead to new paradigms for treating and diagnosing cancer.
Do you want to learn more about the possibilities to collaborate with Oncode, please reach out to Yuva Oz
Kiadis updates status of marketing authorization application for ATIR101
Gezondheid, InnovatieklimaatKiadis, a clinical-stage biopharmaceutical company, today announced that it has received feedback from the European Medicines Agency (EMA) related to the
marketing authorization application (MAA) seeking approval of ATIR101 in hematopoietic stem cell transplant (HSCT). As previously announced, Kiadis submitted responses to the second Day 180 list of outstanding issues for ATIR101 to EMA in May 2019.
As part of the review process, EMA today informed Kiadis that it will convene a Scientific Advisory Group (SAG) in September, comprising of experts in hematology and HSCT to assist EMA in arriving at a determination.
“We appreciate the feedback from EMA and look forward to continuing our interaction with them during the review of our MAA for ATIR101,” said Arthur Lahr, CEO, of Kiadis Pharma. “With the SAG meeting now preceding the subsequent CAT and CHMP meetings as part of the EMA approval process, we are changing our guidance to potential EU conditional approval in 2020.”
While initial launch in the EU is delayed, Kiadis is on track to complete enrollment in the Phase 3 ATIR101 HSCT study in 2021 to support potential marketing approval in the US. Additionally, the company is also on track to start the Phase 1/2 studies of CSDT002 in HSCT and relapse and refractory acute myeloid leukemia in 2020. With a novel cell-based cancer immunotherapy platform consisting of both T-cell and NK-cell technologies, Kiadis has the opportunity to potentially revolutionize transplants and develop novel cancer cell therapies.
Source: Kiadis Pharma
Gilead to submit new drug application for filgotinib to FDA
GezondheidGilead today announced that at a recent pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA), the company provided an update about the investigational oral, selective JAK1 inhibitor, filgotinib. The company discussed with the agency the Phase 3 FINCH studies, as well as the ongoing Phase 2 MANTA safety study assessing semen parameters with filgotinib treatment in men with moderately to severely active ulcerative colitis or Crohn’s disease. As a result of this discussion, a path forward has been established to submit the NDA for filgotinib as a treatment for rheumatoid arthritis in 2019.
Source: Galapagos
Staatssecretaris Blokhuis op stoom
Gezondheid op maat, Hollandbio, GezondheidStaatssecretaris Blokhuis wil gezondheidswinst door vaccinaties maximaal verzilveren, zo liet hij de Tweede Kamer afgelopen week in een uitgebreide Kamerbrief weten. De Kamerbrief meldt dat de daling van de vaccinatiegraad tot staan is gebracht. Volgens Blokhuis geen reden om op zijn lauweren te rusten: “dit motiveert mij des te meer om onverminderd in te zetten op de maatregelen uit mijn brief Verder met vaccineren”. We roemden al eerder de doortastende aanpak van Blokhuis op het vaccinatiedossier. En ook nu weer is HollandBIO blij met de ambitie die uit de brief spreekt. We zetten de hoogtepunten uit de brief graag voor je op een rij.
Pneumokokkenvaccinatie voor ouderen
Vanaf het najaar van 2020 gaat de overheid ouderen wapenen tegen de pneumokokken. De Staatssecretaris neemt nu definitief het advies van de Gezondheidsraad over om 60, 65, 70 en 75 jarigen een vaccinatie aan te bieden. In eerste instantie mogen gegadigden zich bij de huisarts melden voor deze prik. Of dit de best passende aanpak is, zal na twee jaar uit een evaluatie blijken.
Werkdruk bedreigt voorspoedige implementatie van nieuwe vaccins
De werkdruk bij de jeugdgezondheidszorg (JGZ) is hoog, zo constateert Blokhuis. En dankzij de implementatie van diverse nieuwe vaccins, waarvoor de JGZ verantwoordelijk is, zal die niet snel afnemen. “De praktijk kan geen oneindige hoeveelheid wijzigingen tegelijkertijd aan”, concludeert Blokhuis. En dus liggen vertragingen bij invoer op de loer. Volgens HollandBIO is er maar één structurele oplossing: extra capaciteit en middelen. Op die manier kan de werkdruk worden verlicht en vinden al die vaccins snel hun weg naar de juiste arm.
Een verbeterde communicatiestrategie
Blokhuis wil zich nog duidelijker uitspreken vóór het belang van vaccinatie. Op verzoek van de Tweede Kamer gaat hierbij extra aandacht uit naar ervaringsverhalen. Die krijgen dan ook een prominente plek op een te ontwikkelen website, waar jong en oud terecht kunnen voor informatie en actualiteiten over vaccineren. Het RIVM werkt intussen aan een vergrote zichtbaarheid van de al bestaande website over vaccinatie. Tot slot zet de staatssecretaris extra middelen in voor aanvullende gesprekstrainingen van professionals.
Benieuwd naar de hele Kamerbrief? Lees hem hier.