Veelbelovende en uitdagende innovaties met een uitstekend marktperspectief, die ondernemers niet helemaal zelf kunnen financieren: daarvoor kan het Innovatiekrediet uitkomst bieden. Ter gelegenheid van het 10 jarig bestaan licht RVO een paar mooie voorbeelden uit. Neem een kijkje op de website en laat je inspireren!
Genmab announced that worldwide net sales of DARZALEX (daratumumab), as reported by Johnson & Johnson, were USD 498 million in the third quarter of 2018 compared to USD 317 million in the third quarter of 2017, an increase of 57%. The 2018 third quarter net sales were USD 318 million in the U.S. and USD 180 million in the rest of the world.
Johnson & Johnson reported worldwide operational growth (excluding the impact of foreign currency movements) between the two third quarter periods of 60%.The growth was partially offset by a one-time adjustment outside the U.S. related to accruals for retroactive pricing adjustments which negatively impacted this worldwide operational growth by 16 percentage points.
Genmab will receive royalties on the worldwide net sales of DARZALEX under the exclusive worldwide license to Janssen Biotech, Inc. to develop, manufacture and commercialize DARZALEX.
Source: Genmab
Vorig jaar werd in het Regeerakkoord een pakket aan maatregelen aangekondigd om het Nederlandse vestigingsklimaat te versterken. Een goed vestigingsklimaat is voor Nederland van groot belang en geldt als een randvoorwaarde voor een ijzersterk innovatieklimaat. Afgelopen week maakte premier Rutte bekend af te zien van de meest veelbesproken maatregel: de afschaffing van de dividendbelasting. Deze week presenteerde staatssecretaris Snel van Financiën de alternatieve plannen. Het gaat onder andere om een verdere verlaging van de vennootschapsbelasting, een overgangsregeling voor het verkorten van de looptijd van de 30% regeling en een verhoging van de tweede schijf van de WBSO van 14% naar 16%. De maatregelen zijn een mooie impuls voor het innovatieve MKB en verstevigen de positie van Nederland als aantrekkelijk vestigingsland. Lees hier de hele kamerbrief met de aangekondigde maatregelen.
Intravacc and TherapyX are pleased to announce that they have entered into a strategic partnership for the co-development of NGoXIMTM, the world’s first prophylactic vaccine against infection with Neisseria gonorrhoeae. This new vaccine is intended to enhance specific adaptive immune responses that provide lasting, protective immunity against N. gonorrhoeae. NGoXIMTM is a combination of a sustained-release formulation of interleukin 12 (GneX12TM) and bacterial outer membrane vesicles (OMV) formulated for mucosal delivery.
No effective gonorrhea vaccine is currently available and the disease is known to be acquired repeatedly with apparently no development of protective immunity from previous infection. The American Centers for Disease Control and Prevention (CDC) has listed antibiotic-resistant N. gonorrhoeae as one of the top three pathogens presenting “an immediate public health threat that requires urgent and aggressive action.” A recent WHO technical consultation on vaccines against sexually transmitted infections (2017) called for renewed efforts to develop a vaccine against gonorrhea. This partnership effort is a direct response to that call.
Under the terms of the partnership agreement, TherapyX will focus efforts on IND-enabling efficacy and toxicology studies in primates while Intravacc will be responsible for the discovery and development of OMVs with improved immunogenic properties and reduced toxicity. Financial terms were not disclosed.
“We are thrilled to partner with Intravacc” said Dr. Dominick L. Auci, Vice President for Research and Development at TherapyX. “They bring over 100 years of expertise in vaccines including novel OMV technology to this important project. We couldn’t be working with a more experienced, innovative and dedicated team”.
“We are delighted to be working with TherapyX” said Mr. Nico Oudendijk, General Director of Intravacc. “The IL-12 technology they bring into the project is key to develop an efficacious vaccine against this disease.”
Source: Intravacc
The European biotech sector is on track to break its annual funding record, according to BioWorld. After a big third quarter, the total for the first nine months of the year stands at $6.3 billion, putting the region on course to clear the $8 billion bar.
European biotechs made a solid start to 2018, pulling in $3.9 billion over the first six months. But the fundraising haul really took off in the third quarter, when major follow-on offerings contributed to the sector pulling in $2.5 billion. The surge means the sector is set to ease past last year’s total of $6.9 billion and puts it a solid quarter away from breaking the $8 billion barrier.
The deals underpinning the sector’s move toward the new fundraising high says something about the state of biotech in the region today. Galapagos, a European biotech with the once-rare aspiration of becoming a midsized, standalone company, led the way with a $346 million follow-on offering that built on positive clinical trial data.
Argenx and ProQR Therapeutics made similar moves to Galapagos, pulling in $301 million and $104 million, respectively, after generating data on clinical-phase candidates.
The financing highlights the emergence of a clutch of growing European biotechs with aspirations to take drugs deep into the clinic. However, they also show the importance of the U.S. in the fulfillment of these aspirations. Galapagos, Argenx and ProQR are all listed on Nasdaq.
Yet, while Nasdaq is the default option for European biotechs, Idorsia is showing there is another way. Idorsia, which spun out of Actelion following the Johnson & Johnson takeover, used its listing on the Swiss stock exchange to raise $307 million to fund a slate of late-phase trials.
While the follow-on market thrived in the third quarter, the IPO pipeline dried up. The VC world was more active. As previously reported, British biotechs, led by Nasdaq-bound gene therapy player Orchard Therapeutics, raised serious sums of money in the third quarter. Orchard was one of six U.K. companies in the list of top 10 private European financings for the quarter.
The question now is whether European biotechs can maintain the momentum built up in the third quarter and hit a new funding high. Early signs are good. GW Pharmaceuticals got the quarter underway with a $300 million follow-on offering, moving the total for the year above $6.6 billion.
Source: FierceBiotech
Kite, a Gilead Company, and HiFiBiO Therapeutics, a biotechnology company focused on the discovery of therapeutic antibodies through single B cell screening and analysis, announced the companies have entered into a research collaboration and license agreement to develop technology supporting the discovery of neoantigen-reactive T cell receptors (TCRs) for the potential treatment of various cancers, including solid tumors.
Kite and HiFiBiO intend to adapt HiFiBiO’s proprietary single cell technology platform to create a high throughput approach that will potentially allow for the in-depth screening of TCR repertoires from patient samples to identify shared antigen and neoantigen TCRs for use in adoptive cellular therapies. Neoantigens arise from tumor-specific mutations that are unique to each patient’s cancer, offering the potential for more targeted antitumor activity.
Under the terms of the agreement, HiFiBiO will receive a $10 million upfront payment and will be eligible for additional payments based on the achievement of certain research milestones. Kite will have an exclusive option to license HiFiBiO’s platform to screen T cell repertoires and to identify TCRs for use in TCR engineered T cell therapies with a corresponding payment to HiFiBiO.
“Neoantigen-based cell therapy is a very exciting, yet complex, area of research that has the potential to transform the way we treat many solid tumors,” said Alessandro Riva, MD, Gilead’s Executive Vice President, Oncology Therapeutics & Head, Cell Therapy. “We are excited about this collaboration with HiFiBiO, which will build upon our existing capabilities focused on discovering cell therapies which target patient-specific tumor neoantigens.”
“Kite is a recognized leader in the cell therapy field,” said Liang Schweizer, PhD, President and Chief Executive Officer, HiFiBiO Therapeutics. “We are proud that our proprietary single cell technology platform, which is utilized to support the rapid progression of our novel antibody therapeutics pipeline, will now also be implemented by Kite for the development of novel cell therapies targeting neoantigens.”
Source: Gilead
Forbion, a leading European life science venture capital firm, announced the final close of its new flagship fund, Forbion IV. The fund closed above its original target at EUR 360 million and was significantly oversubscribed.
Forbion IV, like its predecessor fund, Forbion III, will primarily focus on opportunities in the EU including the UK, with the remainder of the fund targeting opportunities mainly in North America.
Forbion’s fourth fund will focus on biopharma investments, building on its track record of creating high-return, high-impact businesses, built around exciting new science, proven management teams or assets sourced from the pharmaceutical industry.
Forbion IV will target substantial initial stakes, looking to take lead positions and work alongside entrepreneurial management teams to deliver exceptional financial and societal returns. Forbion has already had considerable success with this strategy via its first three funds, with the EUR 183 million Forbion III already delivering 4 exits including the sale of Rigontec to MSD and the highly successful IPO of Replimune on NASDAQ in July 2018.
The fund, managed by one of Europe’s most experienced, specialised life sciences investment teams, will build a portfolio of approximately fifteen companies, of which five will be new companies (co)founded by Forbion, so-called “build” opportunities, and ten will be existing, “growth” companies.
Sander Slootweg, Managing Partner and co-founder of Forbion, commented:
“The successful final close for our fourth fund at EUR360m, effectively doubling the size of our third fund, unlocks the potential for higher deal sizes for Forbion IV, giving us the flexibility to choose between stand-alone and syndicated investments.
“We are seeing clear opportunities for superior returns in European development-stage life sciences companies, a non-cyclical sector marked by an undersupply of investment capital.
“Forbion IV attracted both new and existing blue-chip investors. New investors include UK based Pantheon, Dutch institutions ASR Insurances and the TNO Pension Fund, Nordic investors Formuesforvaltning and KLP, and some undisclosed North American institutional investors. Existing LPs European Investment Fund and development bank KfW also returned in the new fund. We look forward to working alongside all our Fund IV investors at what is an exceptionally exciting time for our business.”
Robbert van de Griendt, General Partner and Head of Investor Relations and Impact at Forbion, commented:
“The successful fundraise for Forbion IV is testament to the investor appetite for our specialist investment strategy and to our team’s unrivalled experience and long standing track record in European life sciences investing.
“We’re delighted to welcome back returning LPs and extend a warm welcome to new investors in Forbion IV. We are actively working on a number of Fund IV investments and look forward to putting capital to work across an exciting pipeline”
Source: Forbion
Netherlands Enterprise Agency (“Rijksdienst voor Ondernemend Nederland”), a part of the Ministry of Economic Affairs and Climate Policy, grants through a financial loan (“Innovatiekrediet”) €2.7 million for the development of a diagnostic skin cancer test. Based on genetic information from cancer cells, this skin cancer test predicts if a patient is at risk of having metastases in the lymph nodes. Melanoma is a malignant skin cancer and responsible for 75%1 of all skin cancer related deaths. The number of melanoma patients increases at a rate of 3 – 7%2 annually and amongst children and young adults the number of patients increased by more than 250% between 1973 and 20153. Because incidence levels are rising, there is a high need to select patients for surgery to determine the presence of metastases, and to avoid patients having surgery that have a proven low risk at metastases by a diagnostic test. The development of this reliable diagnostic test will therefore make an important and much needed contribution to the improvement of skin cancer care.
“It is great recognition that the Dutch government, as an independent party, expresses its confidence in our capacity to successfully develop this test and bring value to the doctor and their patients globally,” says Dharminder Chahal, CEO SkylineDx. SkylineDx, a high-tech company in (cancer) diagnostics, is developing the test in collaboration with the research group of Alexander Meves4, M.D., of the renowned Mayo Clinic, which this year has again been named the best hospital in the United States by the U.S. News and World Report. “Mayo Clinic is a great collaborator. They diagnose and treat skin cancer patients on a daily basis and translate the needs for improvement to help patients. Together we can develop a test that has a significant impact on the entire treatment process,” continues Dharminder Chahal. “We are close to initiating our first clinical validation trials.”
Melanoma is formed in the pigment cells of the skin. Long-term exposure to UV radiation from sunlight and tanning beds increases the risk of melanoma5 . If a doctor wants to detect melanoma metastases, the nearest lymph nodes are surgically removed to check if they are clean. “A striking 85%6-7 of these surgeries are unnecessary and 5%6-7 of patients that do not receive this surgery do have metastases. Our skin cancer test will save society a great deal of healthcare costs by enabling personalized treatment plans and – more importantly – no longer exposes the patient to unnecessary risk of complications from surgical interventions,” concludes Dharminder Chahal.
Footnotes 1. Wouters et al. Crit Rev Oncol Hematol. 2018 2. Cancer Research UK 3. ASCO Abstract 112435 4. Meves et al. Journal of Clinical Oncology. 2015 5. Karimkhari et al. B J Dermatol. 2015 6. Society AC. Cancer Facts & Figures. 2018 7. Gerschenwald et al. CA Cancer J Clin. 2017
Source: SkylineDx
European Biotech Week 2018 begins on Monday the 24th September, with more than 120 events taking place across 18 European countries. The events, hosted at national and local level by a variety of organisations, companies and other biotech enthusiasts, aim to raise awareness, to fuel curiosity and to engage communities in exploring the benefits of biotechnology and discussing its potential for the future.
Commenting on this, EuropaBio Secretary General, Joanna Dupont-Inglis noted: “Biotech already enables us to use precious resources more efficiently, improves nutrition, helps develop new crops to feed a growing population and develops treatments that respond to unmet medical needs. This week enables us to highlight the best and brightest examples of this, live and direct across Europe”
Initiatives throughout the week are as diverse as the organisers themselves. They include conferences, workshops, hands on laboratories, exhibitions and open-door days with companies, laboratories, research institutes and museums. In Brussels, events will include a week-long exhibition, highlighting the role of biotech in delivering on the UN Sustainable Development Goals, which takes on Place Luxembourg, in the heart of Europe’s capital. This will be followed by a film screening of the documentary Well Fed at the world famous BOZAR Centre For Fine Arts.
EuropaBio Chair, Tjerk de Ruiter concluded: “Biotechnology already improves lives in so many ways – but people are often unaware of it. It’s one of the most exciting and innovative technologies that we have and we’re part of a community that’s passionate about it. When you’re passionate about something, you want to share it and that’s what biotech week is really all about.”
To learn more about activities taking place near you visit www.biotechweek.org
Source: EuropaBIO
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.
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