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EMA’s digital transformation: new Orphan Drug Designation application system

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As of June 15th, the European Medicines Agency (EMA) starts the voluntary use of its Scientific and Regulatory Evaluation Procedure Support (S-REPS) for Orphan Drug Designation applications. From September 15th onwards, S-REPS will become mandatory for these applications, replacing the current PDF and Word form-based submission.

Application through S-REPS

S-REPS is a cloud-based end-to-end submission system. It is important to timely arrange for submission through this platform. Companies applying through S-REPS need to establish a User Account and Access Management. They should pre-register themselves through the organisations management services (OMS) in the substance, product, organisation and referential  (SPOR) web-portal. In addition, their substance should be pre-registered via the EU Telematics Controlled Terms (EUTCT) list. Finally, the company needs to request a Research Product Identifier before they can draft and submit applications. To support applicants in this new process, EMA currently develops a guidebook and training videos.

S-REPS extension to PRIME and Business Pipeline Meetings

Although June 15th marks the launch of S-REPS for Orphan Drug Designations applications, EMA acknowledges that this project is the first step in the EMA’s wider digital transformation strategy. Therefore, over time, S-REPS use may be extended to other scientific regulatory processes. The next procedures in line are PRIME and Business Pipeline Meetings.

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Leestip(s) – Kinder- of naschoolse opvang: mag ik je vaccinatieboekje even zien?

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Een compleet ‘geel boekje’ als toegangsbewijs voor kinder- en naschoolse opvang, daar pleiten universitair docent rechtsfilosofie Roland Pierik en hoogleraar filosofie Marcel Verweij in de Volkskrant voor. Net als HollandBIO maken beide heren zich zorgen over de dalende vaccinatiegraad: “Zolang de trend van de langzaam maar zeker afnemende vaccinatiegraad niet wordt gekeerd, is het niet de vraag of mazelen ergens in Nederland gaat uitbreken, maar wanneer en waar.”

Elders in Europa zijn de eerste mazelenuitbraken al een feit. Italië, Griekenland, Frankrijk en Roemenië hebben het zelfs zwaar te verduren, zo bracht het NOS recent naar buiten. EU-wijd zijn in 2017 bijna 15 000 gevallen en 56 doden gemeld. En dat terwijl deze ziekte met een prikje te voorkomen is.

Het is daarom hoog tijd voor actie. Pierik en Verweij roepen op tot indirect overheidsingrijpen. Geen vaccinatie? Dan ook geen toegang tot kinder- en naschoolse opvang. Als de overheid deze maatregel invoert, neemt zij haar verantwoordelijkheid om kinderen, burgers en de samenleving tegen ernstige infectieziekten te beschermen. HollandBIO is erg benieuwd of de overheid gehoor geeft aan deze oproep, ook nu verschillende D66-Kamerleden hier vragen over stellen. Elke stap die bijdraagt aan een heldere visie op de bestrijding van infectieziekten is welkom.

Lees ook het opinieartikel in de Volkskrant en het achtergrondartikel van de NOS. De Kamervragen van D66 zijn hier in te zien.

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Janssen taking Legend CAR T therapy to clinic

Janssen said FDA cleared an IND for CAR T therapy JNJ-68284528 to treat multiple myeloma. Next half, the unit of Johnson & Johnson expects to start a U.S. and European Phase Ib/II study of Legend Biotech’s therapy targeting BCMA.

At last year’s American Society of Clinical Oncology (ASCO) meeting, Legend reported JNJ-68284528 led to a 100% objective response rate among 19 MM patients in the first phase of the Chinese Phase I/II LEGEND-2 study. No neurotoxicities, which can plague anti-CD19 CAR T therapies, were observed.

In December 2017, Janssen obtained a global license to the therapy from the Legend Biotech USA Inc. and Legend Biotech Ireland Ltd. subsidiaries of Genscript Biotech Corp. The partners are developing JNJ-68284528 jointly worldwide.

The therapy is Janssen’s first in the CAR T space. At the time of the deal, Janssen’s Global Therapeutic Area Head of Oncology Peter Lebowitz told BioCentury BCMA was the first target to meet the company’s safety and efficacy standards for the CAR T modality (see BioCentury, Jan. 5).

Janssen said the primary efficacy endpoint in the Phase II portion of its planned trial to treat relapsed or refractory MM will be overall response rate (ORR) as defined by the International Myeloma Working Group response criteria.

Source: Biocentury

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Treeway prepares phase 3 study for TW001 to treat ALS

Phase 1 study suggests that novel oral formulation improves bioavailability while remaining well tolerated.

Treeway, a biotech company developing therapies against amyotrophic lateral sclerosis (ALS), announced the completion of its Phase I trial of lead program TW001, an oral formulation of edaravone. The study compared the bioavailability of TW001 in its novel market formulation to Radicava, an FDA approved, intravenous formulation of edaravone, indicated for the treatment of ALS. The study indicates increased bioavailability of TW001 in a single-dose treatment whilst no safety concerns developed within the subject group, implying the potential of the novel formulation to improve treatment strategies for ALS patients. Based on these results, Treeway has initiated the upscaling program and preparations for a pivotal Phase 3 study. Edaravone is a neuroprotective agent that reduces oxidative stress, a key contributor to neuronal death in ALS.

“It is Treeway’s mission to identify novel treatment routes for ALS patients and we believe that this study indicates that an oral formulation of edavarone may offer patients a better quality-of-life through a simpler and more effective route of administration,” said Ronald van der Geest, Chief Development Officer of Treeway. “Given the nature of this program and based on the positive results we have seen in this comparative study, we can initiate a pivotal Phase 3 study and move rapidly toward bringing TW001 to patients.”

The randomized, cross-over comparative bioavailability trial tested a single oral dose of 140 mg TW001, compared to a 1-hour intravenous infusion of 60 mg edaravone in 18 healthy subjects. In this study, the bioavailability of the novel oral formulation exceeded the bioavailability of the intravenous infusion of Radicava. Within the subject group of the study the treatment was well-tolerated and no safety concerns arose during the conduct of the study. Furthermore, ongoing formulation studies also suggest that the formulation is stable.

Merit Cudkowicz, MD, Chief Neurology Service and Director of the ALS Clinic at the Massachusetts General Hospital, a key advisor to the company, commented:

“The results of this Phase I study are a promising sign for ALS patients, who are in high need of better and more patient-friendly treatments for their disease. I regard this study to be a key milestone for the development of TW001 in ALS and I look forward to supporting the company’s scientific and clinical progress.”

Source: BusinessWire

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Hartwig Medical Foundation and ttopstart announce collaboration

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Hartwig Medical Foundation and ttopstart are pleased to announce collaboration to accelerate the implementation of clinical decision support systems for personalised cancer treatment in the Netherlands.

Hartwig Medical Foundation is developing innovative clinical decision support systems to enable personalised cancer treatment in the Netherlands. As a part of this, Hartwig Medical Foundation provides access to a unique databank of Whole Genome Sequencing and clinical data of thousands of patients. This will enable a substantial improvement in the quality of diagnosis and treatment of cancer patients, stimulate scientific research into methods of treatment and cancer therapies, and reduce the cost of care and over-treatment. Furthermore, Hartwig Medical Foundation has developed a unique patient report that provides clinicians with an actionable assessment of the genetic characteristics of the tumour to guide clinical decision-making for personalised cancer treatment.

Hartwig Medical Foundation is making important progress in advancing the implementation of DNA sequencing technology and clinical-decision making for personalised cancer care. To accelerate this process, Hartwig Medical Foundation and ttopstart co-design smarter strategies and develop new business models to support patient access to precision medicine in a timely and responsible manner and the introduction of actionable patient reporting for clinical decision-making in personalised cancer treatment. In addition, ttopstart will provide ongoing project management support for the different key partnerships of Hartwig Medical Foundation.

Source: ttopstart

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Merus’ MCLA-158 Phase I clinical trial has started

Merus, a clinical-stage immuno-oncology company developing innovative bispecific antibody therapeutic, announced that the first patient has been dosed in a Phase 1, first-in human clinical trial of MCLA-158 in patients with solid tumors with an initial focus on metastatic colorectal cancer. The trial will be conducted in Europe, where several Clinical Trial Applications (CTAs) have been approved to date. The Company also announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for MCLA-158, which was accepted by the FDA in April 2018. With this acceptance, Merus plans to open additional sites for this trial in the United States.

MCLA-158 is designed to bind to cancer stem cells expressing leucine-rich repeat-containing G protein-coupled receptor 5 (Lgr5) and epidermal growth factor receptors (EGFR). MCLA-158 was identified from a large library of bispecific antibodies targeting molecules belonging to the Wnt and receptor tyrosine kinase signaling pathways as part of work performed by the suppresSTEM consortium, a project that was funded by the European Union. Functional evaluation of patient-derived colorectal tumors, including those harboring RAS and/or PI3K mutations, demonstrated that MCLA-158 was more effective at inhibiting tumor growth and promoting apoptosis than an approved targeted therapy comparator for metastatic colorectal cancer, cetuximab. In preclinical studies, Merus also observed that the growth inhibitory activity of MCLA-158 was greater for colon tumors compared to normal colon tissue, consistent with its good safety profile in non-human primates.

“The commencement of our Phase 1 clinical trial of MCLA-158 is an important milestone for the advancement of our pipeline of bispecific antibodies obtained from our Biclonics technology platform,” said Ton Logtenberg, Ph.D., Chief Executive Officer. “We believe MCLA-158 has the potential to address features that limit currently approved colorectal cancer-targeted therapies, including issues with off-target toxicity and inability to target tumor stem cells, and thus, potentially treat a broader population of patients more effectively.”

The Phase 1, open-label, multicenter clinical trial of MCLA-158 consists of two parts, a dose escalation and a dose expansion. The dose escalation part is intended to determine the appropriate dose of MCLA-158. The dose expansion part will evaluate the safety and tolerability of the defined dose of MCLA-158 in patients with solid tumors. The dose escalation and expansion parts of the trial will also examine the preliminary antitumor activity of single-agent MCLA-158.

Source: GlobeNewsWire

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Lava Therapeutics Raises EUR 16 Million

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Gilde Healthcare and Versant Ventures have driven Lava Therapeutics to a €16 million ($18.8 million) financing. The round equips Lava to advance bispecific engagers of gamma-delta (γδ) T cells, a small subgroup of lymphocytes involved in natural and induced immunity to cancer.

Like other bispecific T-cell engagers, Lava’s drugs are designed to activate the lymphocytes while also acting on tumor cells. The drugs could, for example, block the EGFR-signaling pathway and activate T cells, triggering the production of pro-inflammatory cytokines and killing of tumor cells that express EGFR.

Lava’s platform is differentiated by its focus on Vγ9Vδ2 T cells. Researchers have plugged away on γδ T cells over the past 15 years without garnering the sort of results seen by peers focused on alpha-beta lymphocytes, such as CD4+ helper and CD8+ cytotoxic T cells. But interest has grown as studies have shown intratumoral γδ T-cell signatures correlate to positive prognoses and linked the cells to a range of antitumor activities.

“The cells exhibit potent cytotoxicity and interferon-γ secretion and HLA-unrestricted tumor cell killing and have antigen-presenting capabilities for αβ-T cells promoting the development of adaptive immune responses,” a spokesperson for Lava said.

Gilde and Versant have bought into the idea, as has Paul Parren, Ph.D. Parren, the former head of preclinical R&D at Genmab, has joined Lava as head of R&D. With MRL Ventures Fund also stepping up and joining with founding backers Lupus Ventures and Biox Biosciences to support Lava financially, the Dutch biotech is set up to move programs toward clinical trials.

In keeping with other preclinical biotechs, Lava is yet to say much about targets, indications or other specifics but the activities of the academic group that originated the platform provide some clues. Hans van der Vliet, M.D., Ph.D., Lava’s CSO and CMO, and his team at VU University Medical Center have published a series of papers on Vγ9Vδ2 T cells in recent years.

The papers analyzed the activity of the T cells and described the use of llama-generated single domain antibodies—the modality in which Ablynx is specialized—to activate them. The series culminated in a paper linking an anti-EGFR bispecific to decreased tumor burden and increased overall survival in mice.

That mice study administered the T-cell engagers in combination with Vγ9Vδ2 T cells. However, Lava thinks its candidates will work without an accompanying infusion of T cells.

“These cells are very potent and we believe that sufficient cells are available in tumors and in circulation to obtain an effective therapy,” the spokesperson said. “The administration of therapeutic bispecific antibodies is a much more widely applicable and cost-effective treatment as it avoids cumbersome and costly ex vivo expansion of these cells.”

Lava is now progressing several T-cell engagers toward human testing but is yet to commit publicly to a timeline for getting into the clinic. To support the activity, the biotech is looking to add five to seven new hires to its existing nine-person team. Lava is outsourcing the bulk of its operational activities.

Source: FierceBiotech

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Thuja Capital invests in Salvia BioElectronics

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Salvia BioElectronics secured € 1.3 million in seed funding to develop a minimally invasive bioelectronics solution for people suffering from chronic neurological disease.

The funding is provided by a syndicate led by Thuja Capital Healthcare Seed Fund II and the Brabant Development Agency (BOM), and includes the Netherlands Enterprise Agency (RVO.nl) and founders and employees of Salvia. Salvia intends to use the funds to develop its product concept in preparation for a larger Series-A round that will support the realization of the device and the clinical studies towards CE marking and commercial launch.
Hubert Martens, CEO of Salvia BioElectronics, noted: “The treatment of chronic neurological disease with drugs is associated with undesired side effects that may be intolerable for patients. By gently influencing nerve activity, bioelectronics trigger the body’s natural mechanisms with the promise of being inherently free of side effects, and potentially more efficacious as a treatment.”
Florian Ludwig, investment manager at Thuja Capital: “Thuja Capital invests in early stage medical products for cure, care, diagnosis or prevention, that have a true impact on patients. We expect the neuromodulation field to develop rapidly in the coming years and believe that Salvia’s differentiated product concept will provide physicians with a powerful and patient centric tool in the treatment and management of their patients. The reason for our early contribution is the good track record of Salvia’s team. We recognise that there is a large unmet need for patients suffering from chronic neurological disease, to which Salvia’s innovative concepts can provide a solution. We are therefore delighted to contribute to the early development of this med-tech company.”

Source: Thuja Capital

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Novo Nordisk increases its focus on stem cell-based therapies

The Danish pharma company has announced an increased commitment to stem cell-based therapies, something made possible through a collaborations with the University of California San Francisco (UCSF) in which a first milestone in the development of human embryonic stem cell lines has been reached.

Under the terms of the agreement with UCSF, Novo Nordisk has licenced a technology to enable the generation of good manufacturing practice (GMP) compliant human embryonic stem cell (hESC) lines as well as the rights to further develop these into future regenerative medicine therapies. The milestone was reached in early May when a new GMP laboratory at UCSF was inaugurated. Employees from the university and Novo Nordisk will be working here on deriving the cell lines that are expected to define a new quality standard in production of stem cell-based therapies, reports the company.

Encapsulation device

Together with Cornell University, Novo Nordisk has also made progress in developing an encapsulation device that will protect the beta cells that are transplanted into patients from attack by the immune system. Novo Nordisk anticipates that the first clinical trial could be initiated within the next few years.

“Finding a cure for diabetes is part of Novo Nordisk’s vision and recent progress in our stem cell research and the access to robust and high-quality cell lines raises hopes for people with type 1 diabetes. Our collaboration with UCSF is also expected to accelerate current and future partnerships to develop stem cell-based therapies for treatment of other serious chronic diseases”, said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk.

Collaboration with BioLamina and University of Lund

The development of GMP grade stem cell lines in collaboration with UCSF has also enabled Novo Nordisk to expand the focus on serious chronic diseases beyond diabetes. For example, through partnerships with the Swedish biotech company Biolamina and Lund University, activities have been initiated to develop stem cell-based treatments for Parkinson’s disease. In another partnership with Biolamina and the DUKE National University Singapore Medical School, the research focus is on chronic heart failure and age-related macular degeneration. Novo Nordisk has an ambition to pursue further collaborations to develop stem cell-based therapies for other serious chronic diseases.

Source: Nordic Life Science News

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Voorwaardelijke toelating vormt kans voor sneller en beter van lab naar patiënt

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In een brief die Minister Bruins vandaag aan de Tweede Kamer stuurde, belooft hij in aanloop naar dit najaar een nieuwe voorwaardelijke toegangsregeling voor geneesmiddelen uit te werken. Deze regeling moet zorgen dat veelbelovende middelen sneller beschikbaar komen voor patiënten. Een naadloze aansluiting bij HollandBIO’s programma sneller en beter van lab naar patiënt!

De focus van de nieuwe subsidieregeling komt in het bijzonder te liggen op de vergoeding van weesgeneesmiddelen en geneesmiddelen die van het Europees Medicijn Agentschap (EMA) een toelating onder voorwaarden hebben gekregen. Het gaat om veelbelovende middelen die zich in de praktijk nog verder moeten bewijzen. Minister Bruins geeft in zijn brief aan dat het belangrijk is dat er krachtige prikkels blijven voor fabrikanten om hun geneesmiddel met goed en volledig onderzoek naar de markt te brengen. Ook moet de fabrikant zich binnen het traject van voorwaardelijke toelating committeren aan een verlaagde prijs.

De te publiceren regeling heeft als doel om veelbelovende innovaties te stimuleren, veelal ontwikkeld door startups en MKB’ers in de achterban van HollandBIO. De regeling kan een eerste stap zijn op weg naar een meer adaptief ecosysteem voor de toelating van geneesmiddelen, waarin het nationale vergoedingstraject veel  beter aansluit op de vaak Europese registratietrajecten van geneesmiddelen dan nu het geval is. De actualiteit laat zien hoe groot de urgentie is: vele geregistreerde (wees)geneesmiddelen liepen de afgelopen periode stuk op de nationale vergoedingsbeoordeling. Patiënten moeten hierdoor onacceptabel lang wachten op een bewezen effectieve behandeling, en in sommige gevallen blijven ze er zelfs van verstoken. HollandBIO maakt zich er hard voor dat het vernieuwde traject voor voorwaardelijke toelating hier verandering in zal brengen. We dragen dan ook graag bij aan de totstandkoming van een breed gedragen en praktisch toepasbare regeling!

Meer weten? Lees de hele Kamerbrief van Minister Bruins hier.

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