Amicus Therapeutics said it has acquired Nationwide Children’s Hospital spinout Celenex for up to $452 million, in a deal that expands the rare disease drug developer into gene therapy with a portfolio of 10 clinical and preclinical programs in neurologic lysosomal storage disorders (LSDs).
The 10 adeno-associated virus 9 (AAV9) gene therapy programs—developed at the hospital’s Center for Gene Therapy and The Ohio State University, both based in Columbus, OH—include lead programs in CLN6, CLN3, and CLN8 Batten disease that are potential first-to-market curative treatments, Amicus said.
The other programs include efforts to develop gene therapies for Niemann Pick C, Wolman disease, Tay Sachs disease, and LSDs for other unspecified CNS disorders, according to a presentation for investors by Amicus.
“The groundbreaking work of [principal investigators] Brian Kaspar, Ph.D. and Kathrin Meyer, Ph.D., at Nationwide Children’s Hospital, along with collaborator, Arthur Burghes, Ph.D., professor at The Ohio State University, on these programs has led to remarkably strong and consistent preclinical results and now, in CLN6 Batten disease, encouraging early results in children. This is science and biotechnology at its best,” Amicus Chairman and CEO John F. Crowley said in a statement.
Recruiting of patients has begun for a Phase I/II clinical trial of the CLN6 therapy (NCT02725580), while the first patient is expected to be dosed in a Phase I/II trial of the CLN3 program “in coming months,” Amicus stated in its presentation.
According to Amicus, a single administration of the CLN6 gene therapy generated “encouraging” preliminary efficacy data in the first two patients treated: Two years post-treatment, Hamburg motor and language scores indicated no disease progression in the younger sibling, while disease progression in older sibling showed evidence of stabilization. Additional data for CLN6 will be presented in 2019.
The CLN6, CLN3, and CLN8 programs gave all generated promising preclinical data in mouse models: “The preclinical proof-of-concept we have seen to date in CLN6, CLN3, and CLN8 further support the applicability of the AAV vector we developed at Nationwide Children’s in genetic disease of the CNS,” Dr. Meyer adds.
Billion-Dollar Vision
Amicus said the deal would more than achieve its vision of growing into a rare disease developer whose therapies treat more than 5,000 patients, and generate more than $1 billion in worldwide sales by 2023. Amicus generated $38.005 million in the first half of this year and $36.93 million for all of 2017 in net product sales, all from its sole marketed drug, the Fabry disease treatment Galafold™ (migalastat), authorized in Europe since 2016 and only approved in the U.S. last month.
Of the Celenex programs, Amicus said in the presentation, the largest estimated addressable patient population is the 5,000 projected for the CLN3 treatment, followed by the 3,500 for the Niemann Pick C, Wolman disease, Tay Sachs disease, and other CNS LSD programs. Another 1,000 patients are estimated for the CLN6 treatment, and 750 for the CLN8 therapy.
Amicus has agreed to pay Celenex shareholders $100 million cash upfront, $15 million in payments tied to achieving development milestones, and $262 million in regulatory submission and approval milestones across multiple programs. Amicus said it expected to shell out no more than $75 million over the next four years in milestone payments, and would not pay out any royalties.
Celenex shareholders are also eligible for up to $75 million in payments tied to tiered sales milestones (tiers of $500 million/$750 million). The acquisition and several years of related development costs for all of the gene therapy programs will be financed through a new $150 million debt facility to be provided by BioPharma Credit, an investment fund managed by Pharmakon Advisors.
The transaction was closed immediately following approval by the boards of Amicus and Celenex.
Columbus-based Celenex was established by Gordon Gray, producer of movies that include Secretariat, and his wife, Kristen Gray, whose daughters Charlotte and Gwenyth were both diagnosed with Batten’s disease in 2015, at ages 4 and 2, respectively. The Grays established the Charlotte and Gwenyth Gray Foundation, created to fund research and clinical studies toward Batten disease treatments.
The Celenex acquisition comes after Amicus on August 10 won FDA accelerated approval for Galafold 123 mg capsules, an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) taken every other day. Galafold was approved based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. As a condition of accelerated approval, Amicus agreed to continue studying Galafold in a confirmatory Phase IV program.
Amicus is also conducting a global Phase I/II study (NCT02675465) for another clinical candidate, AT-GAA for Pompe disease. AT-GAA consists of ATB200, a recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate, co-administered with AT2221, a pharmacological chaperone. Clinical results are expected to be shared at the 23rdInternational Congress of the World Muscle Society, set for October 2-6 in Mendoza, Argentina.
The European Commission needs to quickly propose new rules for crops created by modern targeted plant breeding techniques such as Crispr –Cas9 genome editing, or face a withering of Europe’s agricultural research base.
There was a similar message from 33 research institution, universities and plant breeders in the U.K last week in an open letter to agriculture minister and arch-Brexiteer Michael Gove, asking for a round-table meeting involving all stakeholder to agree a clear way forward on research and future use of new plant breeding technologies. Gove has previously indicated he would be prepared to lift EU restrictions on genetically modified crops and farm animals, as part of a “green Brexit.”
The moves follow a surprise ruling by the European Court of Justice (ECJ) in July, which said that new products created by Crispr and similar techniques that offer a precision tool for editing a plant’s genetic code, must go through the same time-consuming approval process prescribed under 2001 EU legislation for older genetic modification techniques.
“In its current form, EU genetic engineering legislation cannot do justice to the opportunities and challenges of [Crispr] technologies,” BEC said.
Plant breeders working with Crispr say the technique can speed development of a new generation of hardier, more productive, more nutritious food crops, improving traits such as pest, salinity and drought resistance, or boosting nutritional content.
They warn that the ECJ ruling could paralyse funding and force an exodus of talent from Europe to places where there are not the same onerous restrictions. The US announced in March it has no plans to regulate genome editing when used to produce new plant varieties that are indistinguishable from those bred through traditional breeding methods. China has made a similar announcement.
Small companies locked out
The ruling is “devastating” to smaller companies, which will need to invest big money to meet the regulatory requirements for bringing a GMO crop on the market in Europe, said Holger Puchta, plant biochemist at the Karlsruhe Institute of Technology in Germany. “Only global international players will be able to do so,” he said.
If the ruling had been different, “Big funders and companies would of course have invested a lot in developing new crop varieties. Now, they are unlikely to do it,” said Stefan Jansson, plant biochemist at Umeå University in Sweden. “European taxpayers will [also] be hesitant to fund research that only will strengthen agriculture in other parts of the world.”
Scientists say making Crispr techniques subject to laws developed for older genetic modification techniques which involve introducing genes from other species, imposes expensive and risky hurdles. Even when crops pass strict regulatory criteria, EU countries can ban them.
Growing a GMO plant outside of the lab would cost about €5,000, “and take one week of my time to get the bureaucracy right,” Jansson said. “If we would get the permit, the costs associated with the experiment would also increase very much.” All this is in Sweden, “which is the most liberal of all EU countries in this respect,” he added.
Subjecting gene editing research to GMO regulations will threaten funding too, scientists said.
Calls in the forthcoming EU research programme Horizon Europe are likely to “become even more restricted for the use of this technology,” said Johnathan Napier, a crop geneticist at the Rothamsted agricultural research centre in the UK.
Napier’s institute was one of 33 signatories of last week’s letter to the UK government calling for clarity on genome editing following the ECJ ruling. “We feel there are significant questions that must be addressed urgently by government if the UK is to retain its strength in plant genetics,” the letter said.
PhD students, meanwhile, are finding that industry positions in the field in Europe are in short supply, said Puchta. “This is a fatal development for the academic labs in Europe too which are in urgent need of these students,” he said.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-09-20 18:22:282018-10-02 18:24:32Scientists urge new EU rules on gene editing crops← #nieuws
A new pack of anti-aging companies like Unity Biotech, Cleara Biotech, and Oisín Biotechnologies think there’s a connection between so-called senescent cells—those that no longer divide—and the effects of growing old.
Clear out cells that have gone into this sleeplike state, the thinking goes, and youthful vigor will return. But not much has been known about how these cells affect the brain.
Now scientists who have taken a careful look at the brains of mice think senescent cells could have a role in neurodegeneration and memory loss.
In the new study, senior author Darren Baker of the Mayo Clinic in Minnesota found that if researchers removed senescent cells or prevented them from building up, they could prevent brain degeneration in mice prone to it.
The results, published in Nature, are among the first to take a careful look at senescence and its possible connection to conditions like Alzheimer’s and Parkinson’s disease. “I think this is a very interesting new avenue, a new way to look at aging,” says Li-Huei Tsai, director of the Picower Institute for Learning and Memory at the Massachusetts Institute of Technology and the author of an editorial accompanying the study. “I am kind of optimistic that this new idea is going to stimulate a lot of people to think about this.”
Baker, a molecular biologist, says the concept of clearing out detrimental cells from the brain is nowhere near ready to try on people. Early tests of this anti-aging concept, like one under way at Unity (which holds patents naming Baker as a co-inventor), are so far targeting specific diseases, like arthritis in the knees, rather than aging as a whole.
Baker got interested in senescence about 15 years ago after genetically altering a mouse to make it more cancer-prone. Instead, the changes accidentally sped up its aging process, he says, and there was evidence senescent cells were involved.
Targeting these old cells has become a major quest among a few anti-aging companies. Baker says he’s unsure whether targeting senescent cells in the brain will be helpful or have side effects like triggering cancer. “We really have no idea right now,” he says.
Because mature neurons naturally don’t divide anyway, it hasn’t been clear how they are affected by senescence. The new study suggests it is the “helper” cells—the microglia and astrocytes surrounding the neurons—that are at the center of the effect.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-09-19 17:48:532018-09-25 18:08:42Clearing out old cells might help the brain← #nieuws
aescuvest en EIT Health kondigen vandaag de lancering aan van het eerste pan-Europese platform voor durfkapitaal crowdfunding voor Europese bedrijven in de zorg: www.aescuvest.eu. aescuvest is het eerste platform dat zich volledig richt op het stimuleren en financieren van innovaties van Europese start-ups en MKB-bedrijven in de zorg. Met behulp van een volledig digitaal proces stelt het bedrijven in staat geld op te halen en geeft het EU-burgers de kans om, over landsgrenzen heen, te investeren. Het platform biedt zowel, particuliere als institutionele beleggers toegang tot de beste bedrijven in de kern zorgsectoren; biotechnologie, medische technologie en digitale zorg. Net als bij beleggingen op basis van een prospectus hebben deze investeringsvoorstellen een grondig goedkeuringsproces doorlopen. De start van de eerste campagne wordt verwacht in het vierde kwartaal van 2018.
“Wij zijn erg trots dat EIT Health ons als partner heeft uitgekozen voor dit baanbrekende project dat ook binnen de financiële en crowdfunding sectoren zelf innovatief is”, reageert Dr. Patrick Pfeffer, CEO & oprichter van aescuvest. “De activiteiten van EIT Health zijn behoefte gedreven en tonen de betrokkenheid van toekomstige gebruikers en patiënten in een co-creatie omgeving bij het ontwikkelen, testen en uitbouwen van toepassingen. Het is het doel van aescuvest.eu een brug te bouwen tussen Europese burgers en zorg- professionals en innovatieve entrepreneurs.”
Jan-Philipp Beck, CEO van EIT Health, voegt daaraan toe: “Crowdfunding stopt vandaag de dag nog aan de landsgrenzen. De samenwerking met aescuvest.eu stelt EIT Health in staat een unieke dienst aan te bieden die vele start-ups zal aantrekken en ons duidelijk onderscheidt van elke andere accelerator. aescuvest.eu geeft een sterke zichtbaarheid, helpt bij het scheppen van een grotere klantenbasis en hoger financieringsvolume en geeft zo een grotere kans op succes en het creëren van impact. aescuvest’s specialisatie, expert due diligence en excellent digitaal bereik van professionals in de sector, in combinatie met EIT Health’s unieke toegang tot honderden innovatieve bedrijven, biedt investeerders een boeiende investeringsmogelijkheid.”
Eén uniek hulpmiddel voor financiering, marketing en verkoop EIT Health is een netwerk van de beste zorg innovators gesteund door de EU. Door over grenzen heen samen te werken draagt EIT Health oplossingen aan die Europese burgers in staat stelt langer en gezonder te leven. Om dit te realiseren verbindt EIT Health alle relevante zorg spelers in Europa – waarbij zij de betrokkenheid van alle kanten van de ‘kennis driehoek’ borgt – zodat innovatie op het snijpunt van onderzoek, onderwijs en bedrijfsleven kan ontstaan. EIT Health brengt zorg professionals, ondernemers en burgers samen om ze nieuwe kansen en middelen te bieden. Het crowdfunding platform aescuvest.eu geeft dit aanbod verdere armslag door middel van een uniek hulpmiddel dat zich richt op financiering, marketing en verkoop om zo de meest veelbelovende zorg start-ups de gelegenheid te bieden hun activiteiten naar Europees niveau op te schalen.
Dr. Pfeffer vult aan: “Crowdfunding geeft ondernemers de kans aandacht te krijgen van investeerders en klanten en te profiteren van hun bereidheid geld, kennis en een breed scala aan contacten ter beschikking te stellen. Op aescuvest.eu wordt dit allemaal aangeboden inclusief vermogen over een vaste contractperiode. Ondernemingen kunnen op deze manier EUR 1 tot 10 miljoen ophalen via een gestandaardiseerde prospectus die grens overstijgende financiering onder het Europese paspoortregime mogelijk maakt. Zonder uitgave van een prospectus is het mogelijk om bedragen van EUR 500.000 tot 1 miljoen op te halen.”
aescuvest.eu stelt investeerders in staat deel te nemen in speciaal geselecteerde projecten die door een onafhankelijke investeringscommissie zijn beoordeeld. Het geeft daarmee het voordeel een vroege investeerder te zijn in grootste Europese economische sector die een stabiel en aanhoudende groei laat zien, niet in het minst door een vergrijzende bevolking. Het co-eigenaar model is opgezet om financiering door particuliere investeerders, samen met individuele of geïnstitutionaliseerde durfkapitalisten mogelijk te maken. Financiering is mogelijk voor ondernemingen vanuit de hele EU, maar de initiële focus zal liggen op bedrijven uit België, Denemarken, Duitsland, Nederland, Zweden en het Verenigd Koninkrijk.
Professor Bart Haex, Business Creation Manager van EIT Health België/Nederland voegt toe: “Ik ben zeer verheugd met deze ontwikkeling. Het eerste initiatief dat via aescuvest.eu gelanceerd wordt is van Nederlandse bodem en toont duidelijk hoe aescuvest.eu innovatieve bedrijven in België en Nederland extra mogelijkheden geeft om groei en ontwikkeling te financieren.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-09-18 23:22:422018-09-18 23:26:54aescuvest en EIT Health lanceren eerste pan-Europees durfkapitaal crowdfunding platform gericht op zorg← #nieuws
Vaccineren helpt, constateert kersverse doctor Maarten van Wijhe. Met succes verdedigde hij vorige week aan de Rijksuniversiteit Groningen zijn proefschrift over de impact van vaccinatieprogramma’s in Nederland. Zijn conclusie liegt er niet om. Door tegen zeven aandoeningen te vaccineren, zijn tussen 1953 en 1992 ruim zesduizend tot twaalfduizend gevallen van kindersterfte voorkomen. Het proefschrift is een stevige bevestiging van belang van het Rijksvaccinatieprogramma. HollandBIO is van mening dat verdere uitbreiding van dit programma, dat inmiddels tegen twaalf ziekten beschermt, Nederland in staat stelt nóg meer gezondheidswinst te verzilveren, zowel op collectief als individueel niveau. HollandBIO roept daarom op tot een breed gedragen, proactieve overheidsvisie op de preventie van infectieziekten. Wij zijn immers pas écht tevreden als alle vaccineerbare aandoeningen de wereld uit zijn.
Geen griepprik, maar grieppleisters: daar werken de onderzoekers van het Disease Research Institute in Seattle hard aan. Een prachtige innovatie, vindt ook HollandBIO. Pleisters zijn immers minder invasief, vereisen geen gekoeld transport en zijn makkelijker te verspreiden tijdens een epidemie. Gewoon, via de post.
Toch zijn er, zoals dat gaat met innovatie, nog wel wat hordes te overwinnen voordat de eerste grieppleisters op je mat vallen. Hoe houd je toezicht op correcte toediening, bijvoorbeeld. Of hoe vaak moet je de pleister plakken. Zo zijn de eerste proefpersonen vooralsnog niet na één, maar pas na twee pleisters beschermd tegen de griep. Tot slot is nog niet duidelijk of het productieproces op te schalen is, bijvoorbeeld in het geval van een pandemie. Aan PostNL zal het in elk geval niet liggen – zij zijn er al helemaal klaar voor.
Meer weten over de grieppleister? Lees dan dit artikel uit de Volkskrant.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-09-18 22:22:192023-09-27 16:32:22Van prikken naar plakken: de grieppleister← #nieuws
In een open brief aan de Britse overheid vragen 33 ondertekenaars om een proactieve en ondersteunende houding en inzet van de Britse overheid rond de toepassing van gene editing. Deze wetenschapsinstellingen, universiteiten, veredelaars, biotech bedrijven en landbouworganisaties doen dit naar aanleiding van de recente uitspraak van het Europese Hof over mutagenese in de context van de Brexit. Net als Nederland kent Groot-Brittannië een sterke agrofood sector waarin veel innovaties en aanzienlijke investeringen plaatsvinden. De ondertekenaars vrezen dat deze positie en het vermogen om sneller en beter tot duurzame en gezonde gewassen en voeding te komen, in gevaar komt als het Verenigd Koninkrijk zich tijdens de Brexit op dit vlak aanpast aan de Europese Unie en de uitspraak van het Hof. De auteurs sluiten af met een pleidooi voor een Britse koers die uitgaat van science-based wet- en regelgeving voor innovatieve plantveredeling en stellen voor dit in een rondetafelgesprek verder te bespreken. HollandBIO kan zich hier helemaal vinden en vindt dat de Nederlandse overheid een zelfde voortrekkersrol moet vervullen vanuit dit streven, maar dan binnen Europa.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-09-18 22:13:172018-09-18 22:13:17Open brief aan Britse overheid met oproep tot steun voor gene editing← #nieuws
Het ministerie van Economische Zaken en Klimaat (EZK) heeft de plannen voor de WBSO in 2019 bekend gemaakt. Met het oog op een stabiel investerings- en vestigingsklimaat voor R&D-bedrijven blijven de voordeelpercentages en schijflengtes gelijk aan 2018. Ook de voorwaarden en het aanvraagprogramma blijven ongewijzigd. Het kabinet stelt € 1.205 miljoen beschikbaar voor de WBSO in 2019.
Dit staat in de op Prinsjesdag gepubliceerde Kamerbrief.
Voordeelpercentages en -bedragen
Bedrijven kunnen het voordeel van de WBSO (Wet Bevordering Speur- en Ontwikkelingswerk) verrekenen via de belastingaangifte. Ook in 2019 gelden er 2 schijven met een verschillend voordeelpercentage voor ontwikkelings- of onderzoekkosten. Voor startende bedrijven geldt een hoger percentage in de eerste schijf.
Voor zelfstandig ondernemers (ZZP-ers) die minimaal 500 onderzoeksuren per jaar maken, is er een vaste aftrek. Startende zelfstandig ondernemers ontvangen daarnaast een extra aftrek. De exacte bedragen voor zelfstandigen volgen later dit jaar.
WBSO evaluatie
Het onderzoeks- en adviesbureau Dialogic evalueert op dit moment de WBSO, op verzoek van het ministerie van EZK. Het bureau rondt de evaluatie naar verwachting in november af. De uitkomsten van de evaluatie kunnen leiden tot aanpassingen van de budgetsystematiek, de parameters en/of de voorwaarden van de WBSO per 2020.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-09-18 18:10:532018-09-25 18:14:52WBSO in 2019: budget en voorwaarden stabiel← #nieuws
New York University School of Medicine researchers described in Nature Medicine a machine learning-based program that can distinguish lung cancer subtypes and at least six driving mutations using only histological slide images, in less time than could a pathologist and standard profiling techniques. The available online code, which the authors think can be applied across cancers, could help clinicians diagnose cancer and make treatment decisions faster.
The deep-learning program was trained to distinguish between lung adenocarcinoma, lung squamous cell carcinoma and non-cancerous lung tissue using open-source code from Google by analyzing hundreds of pixels within images of patient samples from The Cancer Genome Atlas (TCGA).
Among 340 additional patient images, the program then correctly distinguished between the two tumor types with area under the curve (AUC) for true positives vs. false positives ranging from 0.861-0.977.
The researcher’s machine learning technique also correctly classified more images in the TCGA data set than pathologists. The program took an average of 20 seconds to classify a slide; a pathologist took at least one minute per slide.
The program was also trained to identify common, clinically relevant lung cancer mutations from the slide images in about 20 seconds, a process that otherwise requires immuno-staining or tumor sequencing that can take a week or more.
Six frequently mutated genes including serine/threonine kinase 11 (STK11; LKB1), K-Ras (KRAS), p53 and multiple mutations of EGFR could be reliably identified by the program, suggesting it could help identify mutations that guide treatment decisions. The paper’s authors suggested the algorithm could detect morphologic changes in tissue caused by certain mutations.
Study author Aristotelis Tsirigos told BioCentury the group plans to improve the algorithm’s accuracy by using more images to train the algorithm, including images with more detailed annotations from pathologists. Tsirigos is an assistant professor of pathology and director of the applied bioinformatics laboratory at NYU.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-09-18 16:42:502018-09-25 16:18:24AI CODE COULD SPEED CANCER DIAGNOSIS and TREATMENT DECISIONS← #nieuws
argenx a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, announced the pricing of an underwritten public offering in the United States and an offering in Europe only to qualified investors (within the meaning of Directive 2003/71/EC, as amended) (the Offering) with anticipated gross proceeds totalling approximately $300.6 million from the sale of 3,475,000 American Depositary Shares (ADSs) at a price to the public of $86.50 per ADS.
Each of the ADSs offered represents the right to receive one ordinary share, nominal value of €0.10 per share. All of the ADSs in the Offering are being sold by argenx.
argenx has granted the underwriters an option to purchase up to an additional 521,250 ADSs, representing 15% of the ADSs sold in the Offering. This option can be exercised during the 30-day period commencing September 18, 2018.
argenx’s ADSs are currently listed on the Nasdaq Global Select Market under the symbol “ARGX” and argenx’s ordinary shares are currently listed on Euronext Brussels under the symbol “ARGX.”
Morgan Stanley, Cowen and Evercore ISI are acting as joint bookrunning managers for the Offering, and Kempen and Nomura are acting as co-managers. Piper Jaffray is acting as a financial advisor in connection with the Offering. The Offering is expected to close on September 21, 2018, subject to customary closing conditions.
The securities are being offered pursuant to an automatically effective shelf registration statement that was previously filed with the Securities and Exchange Commission (SEC). A preliminary prospectus supplement relating to and describing the terms of the Offering has been filed with the SEC and is available on the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus relating to the Offering may be obtained for free from Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, New York 10014, United States, Attention: Prospectus Department; from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by telephone at (631) 274-2806 or by fax at (631) 254-7140; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, NY 10055, or by telephone at (888) 474-0200.
A request for the admission to listing and trading of ordinary shares underlying the ADSs (the New Ordinary Shares) on the regulated market of Euronext Brussels will be made. For such admission to listing and trading of the New Ordinary Shares on the regulated market of Euronext Brussels, a listing prospectus for the purpose of Article 3, §3 of the Directive 2003/71/EC of the European Parliament and of the Council of November 4, 2003, as amended, including by Directive 2010/73/EU, is prepared in accordance with Chapter 5.1 of the Dutch Financial Supervision Act (Wet op het financieel toezicht) for the admission to listing and trading of the New Ordinary Shares, will be filed with the Dutch Authority for the Financial Markets (Stichting Autoriteit Financiële Markten) (the AFM). After approval, the listing prospectus will be notified by the AFM to the Belgian Financial Services and Markets Authority (Autoriteit voor Financiële Diensten en Markten), for passporting in accordance with article 18 of the Prospectus Directive.
This press release is for information purposes only and does not constitute, and should not be construed as, an offer to sell or the solicitation of an offer to buy or subscribe to any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale is not permitted or to any person or entity to whom it is unlawful to make such offer, solicitation or sale. Reference is also made to the restrictions set out in “Important information” below. This press release is not for publication or distribution, directly or indirectly, in or into any state or jurisdiction into which doing so would be unlawful or where a prior registration or approval is required for such purpose.
Amicus Expands into Gene Therapy with Celenex Acquisition
Gezondheid, InnovatieklimaatAmicus Therapeutics said it has acquired Nationwide Children’s Hospital spinout Celenex for up to $452 million, in a deal that expands the rare disease drug developer into gene therapy with a portfolio of 10 clinical and preclinical programs in neurologic lysosomal storage disorders (LSDs).
The 10 adeno-associated virus 9 (AAV9) gene therapy programs—developed at the hospital’s Center for Gene Therapy and The Ohio State University, both based in Columbus, OH—include lead programs in CLN6, CLN3, and CLN8 Batten disease that are potential first-to-market curative treatments, Amicus said.
The other programs include efforts to develop gene therapies for Niemann Pick C, Wolman disease, Tay Sachs disease, and LSDs for other unspecified CNS disorders, according to a presentation for investors by Amicus.
“The groundbreaking work of [principal investigators] Brian Kaspar, Ph.D. and Kathrin Meyer, Ph.D., at Nationwide Children’s Hospital, along with collaborator, Arthur Burghes, Ph.D., professor at The Ohio State University, on these programs has led to remarkably strong and consistent preclinical results and now, in CLN6 Batten disease, encouraging early results in children. This is science and biotechnology at its best,” Amicus Chairman and CEO John F. Crowley said in a statement.
Recruiting of patients has begun for a Phase I/II clinical trial of the CLN6 therapy (NCT02725580), while the first patient is expected to be dosed in a Phase I/II trial of the CLN3 program “in coming months,” Amicus stated in its presentation.
According to Amicus, a single administration of the CLN6 gene therapy generated “encouraging” preliminary efficacy data in the first two patients treated: Two years post-treatment, Hamburg motor and language scores indicated no disease progression in the younger sibling, while disease progression in older sibling showed evidence of stabilization. Additional data for CLN6 will be presented in 2019.
The CLN6, CLN3, and CLN8 programs gave all generated promising preclinical data in mouse models: “The preclinical proof-of-concept we have seen to date in CLN6, CLN3, and CLN8 further support the applicability of the AAV vector we developed at Nationwide Children’s in genetic disease of the CNS,” Dr. Meyer adds.
Billion-Dollar Vision
Amicus said the deal would more than achieve its vision of growing into a rare disease developer whose therapies treat more than 5,000 patients, and generate more than $1 billion in worldwide sales by 2023. Amicus generated $38.005 million in the first half of this year and $36.93 million for all of 2017 in net product sales, all from its sole marketed drug, the Fabry disease treatment Galafold™ (migalastat), authorized in Europe since 2016 and only approved in the U.S. last month.
Of the Celenex programs, Amicus said in the presentation, the largest estimated addressable patient population is the 5,000 projected for the CLN3 treatment, followed by the 3,500 for the Niemann Pick C, Wolman disease, Tay Sachs disease, and other CNS LSD programs. Another 1,000 patients are estimated for the CLN6 treatment, and 750 for the CLN8 therapy.
Amicus has agreed to pay Celenex shareholders $100 million cash upfront, $15 million in payments tied to achieving development milestones, and $262 million in regulatory submission and approval milestones across multiple programs. Amicus said it expected to shell out no more than $75 million over the next four years in milestone payments, and would not pay out any royalties.
Celenex shareholders are also eligible for up to $75 million in payments tied to tiered sales milestones (tiers of $500 million/$750 million). The acquisition and several years of related development costs for all of the gene therapy programs will be financed through a new $150 million debt facility to be provided by BioPharma Credit, an investment fund managed by Pharmakon Advisors.
The transaction was closed immediately following approval by the boards of Amicus and Celenex.
Columbus-based Celenex was established by Gordon Gray, producer of movies that include Secretariat, and his wife, Kristen Gray, whose daughters Charlotte and Gwenyth were both diagnosed with Batten’s disease in 2015, at ages 4 and 2, respectively. The Grays established the Charlotte and Gwenyth Gray Foundation, created to fund research and clinical studies toward Batten disease treatments.
The Celenex acquisition comes after Amicus on August 10 won FDA accelerated approval for Galafold 123 mg capsules, an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) taken every other day. Galafold was approved based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. As a condition of accelerated approval, Amicus agreed to continue studying Galafold in a confirmatory Phase IV program.
Amicus is also conducting a global Phase I/II study (NCT02675465) for another clinical candidate, AT-GAA for Pompe disease. AT-GAA consists of ATB200, a recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate, co-administered with AT2221, a pharmacological chaperone. Clinical results are expected to be shared at the 23rdInternational Congress of the World Muscle Society, set for October 2-6 in Mendoza, Argentina.
Source: GEN
Scientists urge new EU rules on gene editing crops
Voedsel en materialenThe European Commission needs to quickly propose new rules for crops created by modern targeted plant breeding techniques such as Crispr –Cas9 genome editing, or face a withering of Europe’s agricultural research base.
The warning comes from the German Bioeconomy Council (BEC), a panel of 17 researchers who advise the German government, and is widely echoed by plant researchers around the continent.
There was a similar message from 33 research institution, universities and plant breeders in the U.K last week in an open letter to agriculture minister and arch-Brexiteer Michael Gove, asking for a round-table meeting involving all stakeholder to agree a clear way forward on research and future use of new plant breeding technologies. Gove has previously indicated he would be prepared to lift EU restrictions on genetically modified crops and farm animals, as part of a “green Brexit.”
The moves follow a surprise ruling by the European Court of Justice (ECJ) in July, which said that new products created by Crispr and similar techniques that offer a precision tool for editing a plant’s genetic code, must go through the same time-consuming approval process prescribed under 2001 EU legislation for older genetic modification techniques.
“In its current form, EU genetic engineering legislation cannot do justice to the opportunities and challenges of [Crispr] technologies,” BEC said.
Plant breeders working with Crispr say the technique can speed development of a new generation of hardier, more productive, more nutritious food crops, improving traits such as pest, salinity and drought resistance, or boosting nutritional content.
They warn that the ECJ ruling could paralyse funding and force an exodus of talent from Europe to places where there are not the same onerous restrictions. The US announced in March it has no plans to regulate genome editing when used to produce new plant varieties that are indistinguishable from those bred through traditional breeding methods. China has made a similar announcement.
Small companies locked out
The ruling is “devastating” to smaller companies, which will need to invest big money to meet the regulatory requirements for bringing a GMO crop on the market in Europe, said Holger Puchta, plant biochemist at the Karlsruhe Institute of Technology in Germany. “Only global international players will be able to do so,” he said.
If the ruling had been different, “Big funders and companies would of course have invested a lot in developing new crop varieties. Now, they are unlikely to do it,” said Stefan Jansson, plant biochemist at Umeå University in Sweden. “European taxpayers will [also] be hesitant to fund research that only will strengthen agriculture in other parts of the world.”
Scientists say making Crispr techniques subject to laws developed for older genetic modification techniques which involve introducing genes from other species, imposes expensive and risky hurdles. Even when crops pass strict regulatory criteria, EU countries can ban them.
Growing a GMO plant outside of the lab would cost about €5,000, “and take one week of my time to get the bureaucracy right,” Jansson said. “If we would get the permit, the costs associated with the experiment would also increase very much.” All this is in Sweden, “which is the most liberal of all EU countries in this respect,” he added.
Subjecting gene editing research to GMO regulations will threaten funding too, scientists said.
Calls in the forthcoming EU research programme Horizon Europe are likely to “become even more restricted for the use of this technology,” said Johnathan Napier, a crop geneticist at the Rothamsted agricultural research centre in the UK.
Napier’s institute was one of 33 signatories of last week’s letter to the UK government calling for clarity on genome editing following the ECJ ruling. “We feel there are significant questions that must be addressed urgently by government if the UK is to retain its strength in plant genetics,” the letter said.
PhD students, meanwhile, are finding that industry positions in the field in Europe are in short supply, said Puchta. “This is a fatal development for the academic labs in Europe too which are in urgent need of these students,” he said.
Source: Science Business
Clearing out old cells might help the brain
GezondheidA new pack of anti-aging companies like Unity Biotech, Cleara Biotech, and Oisín Biotechnologies think there’s a connection between so-called senescent cells—those that no longer divide—and the effects of growing old.
Clear out cells that have gone into this sleeplike state, the thinking goes, and youthful vigor will return. But not much has been known about how these cells affect the brain.
Now scientists who have taken a careful look at the brains of mice think senescent cells could have a role in neurodegeneration and memory loss.
In the new study, senior author Darren Baker of the Mayo Clinic in Minnesota found that if researchers removed senescent cells or prevented them from building up, they could prevent brain degeneration in mice prone to it.
The results, published in Nature, are among the first to take a careful look at senescence and its possible connection to conditions like Alzheimer’s and Parkinson’s disease. “I think this is a very interesting new avenue, a new way to look at aging,” says Li-Huei Tsai, director of the Picower Institute for Learning and Memory at the Massachusetts Institute of Technology and the author of an editorial accompanying the study. “I am kind of optimistic that this new idea is going to stimulate a lot of people to think about this.”
Baker, a molecular biologist, says the concept of clearing out detrimental cells from the brain is nowhere near ready to try on people. Early tests of this anti-aging concept, like one under way at Unity (which holds patents naming Baker as a co-inventor), are so far targeting specific diseases, like arthritis in the knees, rather than aging as a whole.
Baker got interested in senescence about 15 years ago after genetically altering a mouse to make it more cancer-prone. Instead, the changes accidentally sped up its aging process, he says, and there was evidence senescent cells were involved.
Targeting these old cells has become a major quest among a few anti-aging companies. Baker says he’s unsure whether targeting senescent cells in the brain will be helpful or have side effects like triggering cancer. “We really have no idea right now,” he says.
Because mature neurons naturally don’t divide anyway, it hasn’t been clear how they are affected by senescence. The new study suggests it is the “helper” cells—the microglia and astrocytes surrounding the neurons—that are at the center of the effect.
Source: MIT technology review
aescuvest en EIT Health lanceren eerste pan-Europees durfkapitaal crowdfunding platform gericht op zorg
Innovatieklimaataescuvest en EIT Health kondigen vandaag de lancering aan van het eerste pan-Europese platform voor durfkapitaal crowdfunding voor Europese bedrijven in de zorg: www.aescuvest.eu. aescuvest is het eerste platform dat zich volledig richt op het stimuleren en financieren van innovaties van Europese start-ups en MKB-bedrijven in de zorg. Met behulp van een volledig digitaal proces stelt het bedrijven in staat geld op te halen en geeft het EU-burgers de kans om, over landsgrenzen heen, te investeren. Het platform biedt zowel, particuliere als institutionele beleggers toegang tot de beste bedrijven in de kern zorgsectoren; biotechnologie, medische technologie en digitale zorg. Net als bij beleggingen op basis van een prospectus hebben deze investeringsvoorstellen een grondig goedkeuringsproces doorlopen. De start van de eerste campagne wordt verwacht in het vierde kwartaal van 2018.
“Wij zijn erg trots dat EIT Health ons als partner heeft uitgekozen voor dit baanbrekende project dat ook binnen de financiële en crowdfunding sectoren zelf innovatief is”, reageert Dr. Patrick Pfeffer, CEO & oprichter van aescuvest. “De activiteiten van EIT Health zijn behoefte gedreven en tonen de betrokkenheid van toekomstige gebruikers en patiënten in een co-creatie omgeving bij het ontwikkelen, testen en uitbouwen van toepassingen. Het is het doel van aescuvest.eu een brug te bouwen tussen Europese burgers en zorg- professionals en innovatieve entrepreneurs.”
Jan-Philipp Beck, CEO van EIT Health, voegt daaraan toe: “Crowdfunding stopt vandaag de dag nog aan de landsgrenzen. De samenwerking met aescuvest.eu stelt EIT Health in staat een unieke dienst aan te bieden die vele start-ups zal aantrekken en ons duidelijk onderscheidt van elke andere accelerator. aescuvest.eu geeft een sterke zichtbaarheid, helpt bij het scheppen van een grotere klantenbasis en hoger financieringsvolume en geeft zo een grotere kans op succes en het creëren van impact. aescuvest’s specialisatie, expert due diligence en excellent digitaal bereik van professionals in de sector, in combinatie met EIT Health’s unieke toegang tot honderden innovatieve bedrijven, biedt investeerders een boeiende investeringsmogelijkheid.”
Eén uniek hulpmiddel voor financiering, marketing en verkoop EIT Health is een netwerk van de beste zorg innovators gesteund door de EU. Door over grenzen heen samen te werken draagt EIT Health oplossingen aan die Europese burgers in staat stelt langer en gezonder te leven. Om dit te realiseren verbindt EIT Health alle relevante zorg spelers in Europa – waarbij zij de betrokkenheid van alle kanten van de ‘kennis driehoek’ borgt – zodat innovatie op het snijpunt van onderzoek, onderwijs en bedrijfsleven kan ontstaan. EIT Health brengt zorg professionals, ondernemers en burgers samen om ze nieuwe kansen en middelen te bieden. Het crowdfunding platform aescuvest.eu geeft dit aanbod verdere armslag door middel van een uniek hulpmiddel dat zich richt op financiering, marketing en verkoop om zo de meest veelbelovende zorg start-ups de gelegenheid te bieden hun activiteiten naar Europees niveau op te schalen.
Dr. Pfeffer vult aan: “Crowdfunding geeft ondernemers de kans aandacht te krijgen van investeerders en klanten en te profiteren van hun bereidheid geld, kennis en een breed scala aan contacten ter beschikking te stellen. Op aescuvest.eu wordt dit allemaal aangeboden inclusief vermogen over een vaste contractperiode. Ondernemingen kunnen op deze manier EUR 1 tot 10 miljoen ophalen via een gestandaardiseerde prospectus die grens overstijgende financiering onder het Europese paspoortregime mogelijk maakt. Zonder uitgave van een prospectus is het mogelijk om bedragen van EUR 500.000 tot 1 miljoen op te halen.”
aescuvest.eu stelt investeerders in staat deel te nemen in speciaal geselecteerde projecten die door een onafhankelijke investeringscommissie zijn beoordeeld. Het geeft daarmee het voordeel een vroege investeerder te zijn in grootste Europese economische sector die een stabiel en aanhoudende groei laat zien, niet in het minst door een vergrijzende bevolking. Het co-eigenaar model is opgezet om financiering door particuliere investeerders, samen met individuele of geïnstitutionaliseerde durfkapitalisten mogelijk te maken. Financiering is mogelijk voor ondernemingen vanuit de hele EU, maar de initiële focus zal liggen op bedrijven uit België, Denemarken, Duitsland, Nederland, Zweden en het Verenigd Koninkrijk.
Professor Bart Haex, Business Creation Manager van EIT Health België/Nederland voegt toe: “Ik ben zeer verheugd met deze ontwikkeling. Het eerste initiatief dat via aescuvest.eu gelanceerd wordt is van Nederlandse bodem en toont duidelijk hoe aescuvest.eu innovatieve bedrijven in België en Nederland extra mogelijkheden geeft om groei en ontwikkeling te financieren.”
Bron: aescuvest
Proefschrift: vaccinaties redden duizenden levens in Nederland
Gezondheid op maat, GezondheidVaccineren helpt, constateert kersverse doctor Maarten van Wijhe. Met succes verdedigde hij vorige week aan de Rijksuniversiteit Groningen zijn proefschrift over de impact van vaccinatieprogramma’s in Nederland. Zijn conclusie liegt er niet om. Door tegen zeven aandoeningen te vaccineren, zijn tussen 1953 en 1992 ruim zesduizend tot twaalfduizend gevallen van kindersterfte voorkomen. Het proefschrift is een stevige bevestiging van belang van het Rijksvaccinatieprogramma. HollandBIO is van mening dat verdere uitbreiding van dit programma, dat inmiddels tegen twaalf ziekten beschermt, Nederland in staat stelt nóg meer gezondheidswinst te verzilveren, zowel op collectief als individueel niveau. HollandBIO roept daarom op tot een breed gedragen, proactieve overheidsvisie op de preventie van infectieziekten. Wij zijn immers pas écht tevreden als alle vaccineerbare aandoeningen de wereld uit zijn.
Benieuwd naar het proefschrift van Maarten? Lees (delen van) ‘The public health impact of vaccination programmes in the Netherlands – A historical analysis of mortality, morbidity, and costs’ hier: https://www.rug.nl/research/portal/en/publications/the-public-health-impact-of-vaccination-programmes-in-the-netherlands(29a46600-ad10-447d-a38c-df8f1bdc7ed1).html
Van prikken naar plakken: de grieppleister
Gezondheid op maat, GezondheidGeen griepprik, maar grieppleisters: daar werken de onderzoekers van het Disease Research Institute in Seattle hard aan. Een prachtige innovatie, vindt ook HollandBIO. Pleisters zijn immers minder invasief, vereisen geen gekoeld transport en zijn makkelijker te verspreiden tijdens een epidemie. Gewoon, via de post.
Toch zijn er, zoals dat gaat met innovatie, nog wel wat hordes te overwinnen voordat de eerste grieppleisters op je mat vallen. Hoe houd je toezicht op correcte toediening, bijvoorbeeld. Of hoe vaak moet je de pleister plakken. Zo zijn de eerste proefpersonen vooralsnog niet na één, maar pas na twee pleisters beschermd tegen de griep. Tot slot is nog niet duidelijk of het productieproces op te schalen is, bijvoorbeeld in het geval van een pandemie. Aan PostNL zal het in elk geval niet liggen – zij zijn er al helemaal klaar voor.
Meer weten over de grieppleister? Lees dan dit artikel uit de Volkskrant.
Open brief aan Britse overheid met oproep tot steun voor gene editing
Voedsel en materialenIn een open brief aan de Britse overheid vragen 33 ondertekenaars om een proactieve en ondersteunende houding en inzet van de Britse overheid rond de toepassing van gene editing. Deze wetenschapsinstellingen, universiteiten, veredelaars, biotech bedrijven en landbouworganisaties doen dit naar aanleiding van de recente uitspraak van het Europese Hof over mutagenese in de context van de Brexit. Net als Nederland kent Groot-Brittannië een sterke agrofood sector waarin veel innovaties en aanzienlijke investeringen plaatsvinden. De ondertekenaars vrezen dat deze positie en het vermogen om sneller en beter tot duurzame en gezonde gewassen en voeding te komen, in gevaar komt als het Verenigd Koninkrijk zich tijdens de Brexit op dit vlak aanpast aan de Europese Unie en de uitspraak van het Hof. De auteurs sluiten af met een pleidooi voor een Britse koers die uitgaat van science-based wet- en regelgeving voor innovatieve plantveredeling en stellen voor dit in een rondetafelgesprek verder te bespreken. HollandBIO kan zich hier helemaal vinden en vindt dat de Nederlandse overheid een zelfde voortrekkersrol moet vervullen vanuit dit streven, maar dan binnen Europa.
Lees meer:
WBSO in 2019: budget en voorwaarden stabiel
InnovatieklimaatHet ministerie van Economische Zaken en Klimaat (EZK) heeft de plannen voor de WBSO in 2019 bekend gemaakt. Met het oog op een stabiel investerings- en vestigingsklimaat voor R&D-bedrijven blijven de voordeelpercentages en schijflengtes gelijk aan 2018. Ook de voorwaarden en het aanvraagprogramma blijven ongewijzigd. Het kabinet stelt € 1.205 miljoen beschikbaar voor de WBSO in 2019.
Dit staat in de op Prinsjesdag gepubliceerde Kamerbrief.
Voordeelpercentages en -bedragen
Bedrijven kunnen het voordeel van de WBSO (Wet Bevordering Speur- en Ontwikkelingswerk) verrekenen via de belastingaangifte. Ook in 2019 gelden er 2 schijven met een verschillend voordeelpercentage voor ontwikkelings- of onderzoekkosten. Voor startende bedrijven geldt een hoger percentage in de eerste schijf.
Voor zelfstandig ondernemers (ZZP-ers) die minimaal 500 onderzoeksuren per jaar maken, is er een vaste aftrek. Startende zelfstandig ondernemers ontvangen daarnaast een extra aftrek. De exacte bedragen voor zelfstandigen volgen later dit jaar.
WBSO evaluatie
Het onderzoeks- en adviesbureau Dialogic evalueert op dit moment de WBSO, op verzoek van het ministerie van EZK. Het bureau rondt de evaluatie naar verwachting in november af. De uitkomsten van de evaluatie kunnen leiden tot aanpassingen van de budgetsystematiek, de parameters en/of de voorwaarden van de WBSO per 2020.
Bron: RVO
AI CODE COULD SPEED CANCER DIAGNOSIS and TREATMENT DECISIONS
GezondheidNew York University School of Medicine researchers described in Nature Medicine a machine learning-based program that can distinguish lung cancer subtypes and at least six driving mutations using only histological slide images, in less time than could a pathologist and standard profiling techniques. The available online code, which the authors think can be applied across cancers, could help clinicians diagnose cancer and make treatment decisions faster.
The deep-learning program was trained to distinguish between lung adenocarcinoma, lung squamous cell carcinoma and non-cancerous lung tissue using open-source code from Google by analyzing hundreds of pixels within images of patient samples from The Cancer Genome Atlas (TCGA).
Among 340 additional patient images, the program then correctly distinguished between the two tumor types with area under the curve (AUC) for true positives vs. false positives ranging from 0.861-0.977.
The researcher’s machine learning technique also correctly classified more images in the TCGA data set than pathologists. The program took an average of 20 seconds to classify a slide; a pathologist took at least one minute per slide.
The program was also trained to identify common, clinically relevant lung cancer mutations from the slide images in about 20 seconds, a process that otherwise requires immuno-staining or tumor sequencing that can take a week or more.
Six frequently mutated genes including serine/threonine kinase 11 (STK11; LKB1), K-Ras (KRAS), p53 and multiple mutations of EGFR could be reliably identified by the program, suggesting it could help identify mutations that guide treatment decisions. The paper’s authors suggested the algorithm could detect morphologic changes in tissue caused by certain mutations.
Study author Aristotelis Tsirigos told BioCentury the group plans to improve the algorithm’s accuracy by using more images to train the algorithm, including images with more detailed annotations from pathologists. Tsirigos is an assistant professor of pathology and director of the applied bioinformatics laboratory at NYU.
Source: BIOCENTURY
argenx raises $301M
Innovatieklimaatargenx a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, announced the pricing of an underwritten public offering in the United States and an offering in Europe only to qualified investors (within the meaning of Directive 2003/71/EC, as amended) (the Offering) with anticipated gross proceeds totalling approximately $300.6 million from the sale of 3,475,000 American Depositary Shares (ADSs) at a price to the public of $86.50 per ADS.
Each of the ADSs offered represents the right to receive one ordinary share, nominal value of €0.10 per share. All of the ADSs in the Offering are being sold by argenx.
argenx has granted the underwriters an option to purchase up to an additional 521,250 ADSs, representing 15% of the ADSs sold in the Offering. This option can be exercised during the 30-day period commencing September 18, 2018.
argenx’s ADSs are currently listed on the Nasdaq Global Select Market under the symbol “ARGX” and argenx’s ordinary shares are currently listed on Euronext Brussels under the symbol “ARGX.”
Morgan Stanley, Cowen and Evercore ISI are acting as joint bookrunning managers for the Offering, and Kempen and Nomura are acting as co-managers. Piper Jaffray is acting as a financial advisor in connection with the Offering. The Offering is expected to close on September 21, 2018, subject to customary closing conditions.
The securities are being offered pursuant to an automatically effective shelf registration statement that was previously filed with the Securities and Exchange Commission (SEC). A preliminary prospectus supplement relating to and describing the terms of the Offering has been filed with the SEC and is available on the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus relating to the Offering may be obtained for free from Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, New York 10014, United States, Attention: Prospectus Department; from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by telephone at (631) 274-2806 or by fax at (631) 254-7140; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, NY 10055, or by telephone at (888) 474-0200.
A request for the admission to listing and trading of ordinary shares underlying the ADSs (the New Ordinary Shares) on the regulated market of Euronext Brussels will be made. For such admission to listing and trading of the New Ordinary Shares on the regulated market of Euronext Brussels, a listing prospectus for the purpose of Article 3, §3 of the Directive 2003/71/EC of the European Parliament and of the Council of November 4, 2003, as amended, including by Directive 2010/73/EU, is prepared in accordance with Chapter 5.1 of the Dutch Financial Supervision Act (Wet op het financieel toezicht) for the admission to listing and trading of the New Ordinary Shares, will be filed with the Dutch Authority for the Financial Markets (Stichting Autoriteit Financiële Markten) (the AFM). After approval, the listing prospectus will be notified by the AFM to the Belgian Financial Services and Markets Authority (Autoriteit voor Financiële Diensten en Markten), for passporting in accordance with article 18 of the Prospectus Directive.
This press release is for information purposes only and does not constitute, and should not be construed as, an offer to sell or the solicitation of an offer to buy or subscribe to any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale is not permitted or to any person or entity to whom it is unlawful to make such offer, solicitation or sale. Reference is also made to the restrictions set out in “Important information” below. This press release is not for publication or distribution, directly or indirectly, in or into any state or jurisdiction into which doing so would be unlawful or where a prior registration or approval is required for such purpose.
Source: argenx