11 March 2021
Sofitel Legend Amsterdam The Grand
2020 Presenting Companies
Aelin Therapeutics (BEL)
Aelin Therapeutics will exploit its proprietary Pept-in™ protein knockdown platform as a novel modality in drug development. The company is based on a very comprehensive preclinical package illustrating different applications of the technology (in bacteria, fungi, viruses, cancer cells). The technology is furthermore protected by a strong and growing IP portfolio.
Alize Pharma 3 (FRA)
Alizé Pharma 3 develops innovative therapeutic peptides for rare endocrine and metabolic diseases. Our ambition is to become a leading company in rare diseases with operations in both Lyon (France) and Boston (MA) to support the global development of products. We are building an innovative and balanced portfolio of products targeting significant unmet medical needs. Our first two assets are AZP-3601, a PTH analog for hypoparathyroidism, and AZP-3404, a peptide leveraging the biology of IGFBP2 for treating lipodystrophies and other syndromes of severe insulin resistance.
AM-Pharma is a biopharmaceutical company, engaged in the development of novel human alkaline phosphatase therapeutics for unmet needs in severe inflammatory diseases. The Company’s lead programs for this therapeutic enzyme are in Acute Kidney Injury and Inflammatory Bowel Disease.
Arvelle Therapeutics (SWZ)
- Arvelle was formed with the mission of bringing innovative solutions to patients suffering from CNS disorders.
- Arvelle is responsible for the development and commercialization of cenobamate, an investigational antiepileptic drug, in the European market.
- Arvelle will be based in Switzerland
Atlantic Therapeutics (IRL)
Atlantic Therapeutics develops professional and consumer medical devices, related software, apps and connected technologies to treat incontinence and other associated pelvic health disorders by strengthening muscles and modulating nerves. Atlantic Therapeutics is a pioneer of consumer facing healthcare, recently launching a new, cutting-edge wearable technology called INNOVO® that delivers a therapy via a pair of bike shorts. INNOVO® received a DeNovo FDA clearance in 2018, followed by an OTC (direct to consumer) FDA clearance in January 2020, and was approved for reimbursement by CMS in February 2020.
The company vision is to improve the quality of life of millions of people each year by enabling them to restore their pelvic health, and regain the confidence and control to lead full and active lives.
Atlantic Therapeutics’ main offices are located on the Atlantic coast, in Galway and Boston.
BioArctic is a Swedish biopharma company that develops new drugs based on groundbreaking research for patients with central nervous system disorders. For a global market, the aim is to generate transformative medicines that can stop or slow down the progression of diseases, principally Alzheimer’s and Parkinson’s diseases.
The company’s most advanced projects are BAN2401, which is currently in a confirmatory Phase 3 study in early Alzheimer’s disease in partnership with Eisai and, ABBV-0805 aimed at Parkinson’s disease, which is currently in a Phase 1 study in partnership with AbbVie. BioArctic also works in research stages in Alzheimer’s disease, other neurodegenerative disorders, with a technology platform in blood-brain barrier transport, and in diagnostics and biomarkers.
BioArctic was founded in 2003 based on innovative research from Uppsala University, Sweden. BioArctic’s B-share is listed on Nasdaq Stockholm Mid Cap (ticker: BIOA B).
Cardior Pharmaceuticals (GER)
Cardior is a privately held biopharmaceutical company founded by Prof. Thomas Thum out of the Hannover Medical School. The company is dedicated to becoming world-leader in design, research and development of non-coding RNA-based therapeutics for cardiovascular diseases. Cardior aims to market its products in collaboration with a strategic pharma-partner after clinical validation.
Celyad is a clinical-stage biopharmaceutical company focused on the development of specialized CAR-T cell-based therapies. Celyad’s CAR-T cell platform has the potential to treat a broad range of solid and hematologic tumors. Its lead oncology candidate, CYAD-01, is currently being evaluated in a Phase I dose escalation clinical trial.
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Cergentis is a privately-held genomics company that develops and commercializes kits and services based on its proprietary Targeted Locus Amplification (TLA) technology. TLA is an ingenious well-established technology that enables cost-effective, targeted and complete sequencing of genes of interest, while uniquely allowing the detection of all single nucleotide and structural variants. The technology relies on DNA crosslinking and the physical proximity of DNA sequences as the basis of selection and confers essential advantages in FFPE tumour samples
Recent data generated from TLA sequencing of lymphoma cancer demonstrated a significant improvement over FISH (fluorescence in situ hybridization) and provided additional actionable information for clinicians. In addition, Cergentis presented preliminary data for lung, prostate and cervical cancers. TLA-based gene sequencing also enables the development of personalised circulating tumour DNA (ctDNA) tests to monitor disease progression and response to therapy.
Today the majority of pharma companies involved with the development and quality control of pharmaceutical cell lines and gene therapy products are using Cergentis’ TLA technology.
Eloxx Pharmaceuticals (ISR)
Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates that are designed to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have short half-life messenger RNA that produce truncated, nonfunctional proteins, accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 2,000 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and enabling protein synthesis to continue through the premature stop codons to restore protein functionality. Eloxx’s lead product candidate, ELX-02, is a small molecule drug candidate designed to restore full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis cystinosis. ELX-02 has not been approved by any global regulatory body. Eloxx is headquartered in Waltham, MA. with R&D operations in Rehovot, Israel.
eTheRNA is a clinical stage immunotherapy company developing mRNA based therapeutic products for the treatment of cancer. To date the Company has completed 4 clinical studies and has 2 studies underway currently. The company has built a new GMP facility which also provides contract manufacturing services.
Evotec is a drug discovery company focused on rapidly progressing innovative product approaches with its customers. It operates worldwide providing the highest quality stand-alone and integrated drug discovery solutions, covering all activities from target identification to IND-enabling studies and high-end CMC (Chemistry, manufacturing and controls). The Company focuses on key therapeutic areas including neuronal diseases, diabetes and diabetic complications, oncology, pain, anti-infectives, respiratory and fibrosis.
FoRx Therapeutics is a recently incorporated privately-held company based in Basel, Switzerland. It is backed by a syndicate of investors that include the Novartis Venture Fund, Pfizer Ventures, M Ventures, Omega Funds and LSP Venture Capital. FoRx Therapeutics focuses on drugging key molecular targets involved in DNA Replication Stress, as a new approach towards the development of targeted anticancer drugs.
Galapagos (Euronext & NASDAQ: GLPG) is a clinical-stage biotechnology company specialized in the discovery and development of small molecule medicines with novel modes of action. Galapagos’ pipeline comprises Phase 3 through to discovery programs in cystic fibrosis, inflammation, fibrosis, osteoarthritis and other indications. Our target discovery platform has delivered three novel mechanisms showing promising patient results in, respectively, inflammatory diseases, idiopathic fibrosis and atopic dermatitis. Galapagos is focused on the development and commercialization of novel medicines that will improve people’s lives. The Galapagos group, including fee-for-service subsidiary Fidelta, has approximately 578 employees, operating from its Mechelen, Belgium headquarters and facilities in the Netherlands, France, and Croatia. More information at www.glpg.com.
GTX medical (NL)
GTX Medical (Eindhoven, The Netherlands; Lausanne, Switzerland) is a clinical-stage medical device company developing a new therapy to help paralyzed people walk again, using a new form of Spinal Cord Stimulation: Targeted Epidural Spinal Stimulation (TESS), in combination with intensive rehabilitation training. GTX medical is a spinout of the group of Prof Courtine at the Center for Neuroprosthetics at the Swiss Federal Institute of Technology Lausanne (EPFL).
Heidelberg Pharma (DE)
Heidelberg Pharma is a publicly listed, biopharmaceutical company focused on oncology. It is the first company to develop the toxin Amanitin into cancer therapies using its proprietary Antibody Targeted Amanitin Conjugate (ATAC) technology and to advance the biological mode of action of the toxin as a novel therapeutic principle. This proprietary and innovative technology platform is being applied to develop the Company’s own therapeutic ATACs, as well as in third-party collaborations, to create a variety of ATAC candidates also for other indication fields. The proprietary lead candidate is HDP-101, a BCMA ATAC for multiple myeloma, which is currently being prepared for clinical development.
ImCheck Therapeutics is a Marseille-based, privately-held biotech company developing nenext-generationmmuno-modulatory antibodies acting on both adaptive and innate immunity for the treatment of cancer and other immune related diseases.
With a “precision medicine-based” translational approach and several established academic collaborations, ImCheck is uniquely positioned to develop the right therapy for each patient.
Imcyse develops active targeted immunotherapies to treat and prevent severe chronic diseases caused by disruptions of the immune system. Imcyse’s unique active immunotherapy technology platform, based on the administration of Imotopes™, allows it to locally target immune cells involved in the destruction of the diseased organ. Imcyse has completed its first clinical trial in type 1 diabetes in 7 European countries. Projects which address multiple sclerosis and rheumatoid arthritis, are at preclinical and proof-of-concept research stages, respectively.
ISA develops targeted immuno-activators for the treatment of cancer and infectious diseases. Its best-in-class SLP® technology stimulates the immune system in a highly specific fashion. ISA’s products aim to achieve more, deeper and longer lasting responses and delay or prevent disease recurrence. ISA collaborates with Regeneron around its lead asset, ISA101b targeting HPV16-induced cancers. ISA101b is currently in a randomized, controlled Phase 2 clinical trial for Head and Neck cancer, in combination with cemiplimab (Libtayo®), anti PD-1 antibody. ISA101b is fully funded to approval under the Regeneron partnership. ISA is currently raising funds to bring additional products to the clinic.
istar Medical (BE)
iSTAR Medical, founded in 2011 and headquartered in Wavre, Belgium, is a clinical-stage, medical technology company focused on the development of novel ophthalmic implants for patients with glaucoma. Glaucoma is the second leading cause of adult blindness globally, affecting more than 90 million people. Micro-invasive glaucoma surgery (MIGS) is the most promising and fastest-growing therapeutic option in the treatment of glaucoma.
iSTAR’s lead product, MINIject ™, has been designed to be a best-in-class MIGS device. Data from its first-in-human STAR-I trial confirms that it is safe and highly effective in achieving significant IOP reduction, as well as easing medication burden in glaucoma patients. Several additional studies are in progress to support iSTAR’s market entry plans into Europe and the United States.
iSTAR has raised €55 million to-date.
Kiadis Pharma (NL)
Kiadis is developing therapeutics based on Natural Killer cells, or NK-cells. The K-NK immunotherapy platform consists of off-the-shelf uniquely hyperfunctional NK cells, with high dose, low cost industrial production. Kiadis will have 3 products in clinical development in 2020/2021.
Lumeon Care Pathway Management platform enables healthcare organizations orchestrate and automate systems, processes and teams – uniting patients with care teams: improving patient experience; providing better, more consistent outcomes; ensuring care is efficiently coordinated in real-time. CPM enables digital control of care delivery models, maximizing resource utilization, reducing cost and minimizing variation.
Merus is a clinical-stage immuno-oncology company developing innovative bispecific antibody therapeutics with the goal of treating and potentially curing cancer patients. Our most advanced development programs use the Biclonics® format. Biclonics® are capable of simultaneously attacking tumors in multiple ways. For example by activating the immune system to kill tumor cells and directly inhibiting tumor cell growth and survival pathways
Neurent Medical (IRL)
Nouscom is an oncology company developing next-generation immunotherapies. Nouscom’s proprietary technology harnesses the full power of the immune response by combining viral vectored genetic vaccines based on neoantigens with other immunomodulators. Nouscom raised 54M EUR to date, and is backed by a strong syndicate of international investors: 5AM, Abingworth, LSP, and Versant Ventures.
Orphazyme’s current clinical programs investigate arimoclomol as a treatment for four protein-misfolding indications: The protein-aggregation diseases Amyotrophic Lateral Sclerosis (ALS) and sporadic Inclusion Body Myositis (sIBM), and the lysosomal storage diseases Niemann-Pick disease Type C (NPC) and Gaucher disease. The new molecular entity (NME) program focuses on the development of new molecules as a treatment for rele- vant protein-misfolding diseases.
OxThera is developing a novel treatment, Oxabact, for Primary hyperoxaluria (PH), a fatal ultra-orphan disease in children, and where there are currently no available therapies. The company has initiated a pivotal Phase III study in PH with Oxabact in order to stop and/or delay disease progression. Oxabact holds orphan drug designations in the EU and the US for the treatment of PH, and in the EU for treatment of Short Bowel Syndrome (SBS).
Pharming is a specialty pharmaceutical company developing innovative products for the safe, effective treatment of rare diseases and unmet medical needs. Pharming’s lead product, RUCONEST® (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of acute Hereditary Angioedema (“HAE”) attacks in patients in Europe, the US, Israel and South Korea. The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorisation. RUCONEST® is commercialised by Pharming in the USA and in Europe, and the Company holds all other commercialisation rights in other countries not specified below. In some of these other countries distribution is made in association with the HAEi Global Access Program (GAP). RUCONEST® is also being evaluated for various additional indications. Pharming’s technology platform includes a unique, GMP-compliant, validated process for the production of pure recombinant human proteins that has proven capable of producing industrial quantities of high quality recombinant human proteins in a more economical and less immunogenetic way compared with current cell-line based methods. In addition to RUCONEST® and variants of recombinant human C1 esterase inhibitor, Pharming has recently in-licensed leniolisib from Novartis, a small molecule which is in a registrational study for APDS, a form of Primary Immunodeficiency. Leads for enzyme replacement therapy (“ERT”) for Pompe and Fabry’s diseases are also being produced and optimised respectively at present, with additional programs not involving ERT also being explored at an early stage.
Sequana Medical (BEL)
Sequana Medical is a commercial stage medical device company developing the alfapump® platform for the management of fluid overload in liver disease, malignant ascites and heart failure. The company’s two pillars of growth are the commercialisation of the alfapump in North America, a large market driven by NASH-related cirrhosis, and the clinical development of alfapump DSR, a potential chronic therapy for patients suffering from heart failure-induced volume overload. Both markets leverage Sequana Medical’s alfapump, a unique, fully implanted wirelessly charged & controlled system that automatically pumps fluid from the abdomen into the bladder, where it is eliminated via urination.
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SafeHeal, based in Paris, France, is working to reshape digestive surgery by developing and commercializing anastomosis protection devices for improved post-operative patient recovery and reduced healthcare costs. SafeHeal develops Colovac™, an innovative endoluminal solution for the protection of colorectal anastomosis, designed to reduce anastomotic complications post-surgery and to obviate the need for diverting ostomies for patients undergoing colectomy. The Colovac device is meant to enable patients to resume their normal life after a colorectal surgery, without having to bear an artificial anus and wear an ostomy pouch.
SNIPR Biome (DEN)
SNIPR BIOME is engaged in the discovery and development of CRISPR/Cas-based drugs, using its proprietary and patent-protected CRISPR/Cas platform. SNIPR BIOME is pioneering a novel use of CRISPR/Cas technology to selectively and precisely eradicate target bacteria, while leaving the rest of the patient’s microbial community intact.
Vetex Medical (IRE)
Vetex Medical is a privately-held early stage company based in the strong Med Tech Cluster in Galway Ireland. Established in 2016, the company is focused on making venous clot removal predictable, quick and cost-effective. The company’s first thrombectomy product treats deep vein thrombus by removing large volumes of clot in a single session, without use of thrombolytic drugs.
“We exist to create innovative products that give people more time to enjoy life. We do things differently, but only when that difference is meaningful. Our goal is to be a company people know they can rely on, a friend that’s on their side.”
ViCentra is an innovative medical device company based in Utrecht, The Netherlands, with a ground-breaking product for the management of Type 1 diabetes. Kaleido, the world’s smallest and lightest insulin pump system is designed to help people with diabetes live life on their own terms
Vivasure Medical (IRE)
In percutaneous endovascular procedures such as heart valve replacement and aortic aneurysm repair, large arteriotomies are created. Existing percutaneous closure technologies are complex to use, are associated with significant complications and leave permanent implants in the vessel. Vivasure solves this problem with a safe, effective and easy-to-use device.
Vivoryon Therapeutics develops first-in-class drugs targeting post-translational modifying enzymes. Keeping the patient in mind we advance precision intervention medicines derived from our cutting-edge discovery engine into clinical development stages.
uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular technology platform to rapidly bring new disease-modifying therapies to patients with severe genetic diseases.
We are advancing a focused pipeline of innovative gene therapies and have established clinical proof-of-concept in our lead indication, hemophilia B, and preclinical proof-of-concept in Huntington’s disease. Our pipeline of adeno-associated virus (AAV)-based gene therapies has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities.
Xeltis ETR technology has been tested in various cardiovascular feasibility clinical trials (Fontan, Atrial patch and Pulmonary valve). Xeltis closed a Series B financing and an extension based on these first promising clinical results. In 2017 the company closed a round of 45 M to further expand applications in the cardiovascular space.
Mithra (Euronext: MITRA) is a Belgian biotech company dedicated to transforming Women’s Health by offering new choices through innovation, with a particular focus on contraception and menopause. Mithra’s goal is to develop products offering better efficacy, safety and convenience, meeting women’s needs throughout their life span. Its three lead development candidates are built on Mithra’s unique native estrogen platform, Estetrol (E4): Estelle®, a new era in oral contraception, PeriNesta®, the first complete oral treatment for perimenopause and Donesta®, the next-generation hormone therapy. Mithra also develops and manufactures complex therapeutics in the areas of contraception, menopause and hormone-dependent cancers. It offers partners a complete spectrum of research, development and specialist manufacturing at its technological platform Mithra CDMO. Active in more than 85 countries around the world, Mithra has an approximate headcount of 250 staff members and is headquartered in Liège, Belgium.
Microsure is a medical device company in Eindhoven (The Netherlands) founded by Eindhoven University of Technology and Maastricht University Medical Center in 2016, funded by Innovation Industries Fund, BOM and EIF. Microsure’s focus is to improve patients’ quality of life through developing robotic systems for microsurgery. Its current product – MUSA is the world’s first surgical robot for open microsurgery with a CE Mark. MUSA has been designed by microsurgeons and engineers, specifically for microsurgical applications. MUSA provides superhuman precision and dexterity for microsurgeons, enabling new interventions that are currently impossible to perform by hand.
Oryzon is a public clinical stage biopharmaceutical company developing epigenetics-based therapeutics. Oryzon is listed on the Spanish Stock Exchange (ORY, ISIN Code: ES0167733015). Oryzon has two compounds in Phase II in CNS and oncology.
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