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Clinical trials. Two years of PRIME

Caterina Lucchini
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Clinical trials. Two years of PRIME

It was 2016 when EMA launched the PRIority Medicines scheme (PRIME), a program to drive innovation and improve the efficiency of the development process in therapeutic areas with the most pressing unmet medical needs.

Two years later the Agency published a report to present the first results of PRIME. It explains how the criteria for eligibility to the scheme have been applied and what type of support applicants have received so far. “Our analysis of the first two years shows we have established a platform that promotes the development of promising medicines for unmet medical needs and at the same time addresses the complexity of medicine development,” said Guido Rasi, EMA’s Executive Director. “Through PRIME, we offer early and enhanced dialogue to enable the generation of better data and more robust evidence on a medicine’s benefits and risks”.

Since the launch of PRIME in March 2016, the Agency has received and assessed a total of 177 requests for eligibility to PRIME. Overall, the quality of applications received is good. Only a few requests (8) were out of scope and not deemed suitable for review. The main reasons were that either the product was too early in its development (e.g. too early for proof of principle for SME or for proof of concept for non-SME) or that it was unclear whether the product could fall under the definition of a medicinal product and thus be within the remit of EMA. Out of 169 requests received and assessed, 36 were granted eligibility to PRIME. Twenty-one percent of requests have therefore been accepted in the scheme. This indicator has remained stable since launch. Since its launch, PRIME has generated a high interest from SMEs which have constantly represented more than half of the requests received. On the other hand, only three requests have been received from applicants in the academic sector. As set out in the framework for collaboration between EMA and academia and supporting action plan published in April 2017, EMA will raise awareness on the support the Agency can offer with the aim to promote and further develop regulatory support to translate academic research into novel methodologies and medicines. Oncology and haematology represents the principal therapeutic area (Figure 1), and in 39% of cases the request was for advanced therapy medicinal products (ATMPs) (Figure 2).

Overview of PRIME eligibility requests granted and denied by therapeutic area
Figure 1. Overview of PRIME eligibility requests granted and denied by therapeutic area

 

PRIME eligibility requests by type of product
Figure 2. PRIME eligibility requests by type of product

While PRIME is open to all companies on the basis of preliminary clinical evidence (proof of concept), applicants from the academic sector and SMEs can apply earlier on the basis of compelling non-clinical data and tolerability data from initial clinical trials (proof of principle). Over this two year period, only eight requests were submitted by SMEs at the proof of principle stage. Out of these, three had been granted eligibility to PRIME at this early stage of development. Notably, the sponsor for one of these products has subsequently provided the Agency with exploratory data on its progress to proof of concept, enabling confirmation of PRIME eligibility. Most eligibility requests received (161 out of 169) are at the proof of concept stage supported by exploratory data. The source or phase of clinical trial of the supportive data is illustrated in the Figure 3. Overall, the majority of applications base their request on phase 1 or a combination of phase 1 and phase 2 data.

Source of exploratory clinical data of eligibility requests at the proof of concept stage
Figure 3. Source of exploratory clinical data of eligibility requests at the proof of concept stage

Multidisciplinary support

A key feature of the PRIME scheme are ‘kick-off’ meetings – a unique type of meeting for medicines that are eligible for PRIME. These multidisciplinary meetings bring together the rapporteur for the medicine as well as the chairs and experts of relevant EMA committees to ensure that all aspects of a medicine’s life cycle are discussed early, including risk-management issues. The first kick-off meetings were organised in July 2016 and, to date, 31 meetings have taken place. The aim of the meetings is to agree on next steps on how best to address any identified issues and/or to identify issues to be discussed normally in the context of scientific advice.

Scientific advice

After the kick-off meeting, the enhanced scientific support to optimise the development programme is channelled through scientific advice by the SAWP. This procedure allows the applicant to discuss the details of the development plan, the design of pivotal studies and post-authorisation activities. Since launch of the scheme, a total of 37 scientific advice requests have been received concerning 22 products accepted into PRIME and for which a kick-off meeting has taken place. The Agency could also prioritise Scientific Advice of PRIME products. 12 procedures (32%) were finalised within the shorter 40-day timeframe (compared to the standard 70 days). Scientific Advice have been requested not only on clinical but on all aspects of the development, i.e. quality, nonclinical and clinical, as well as questions related to the post-authorisation follow-up studies and registries.

Conclusion

Overall, after a successful launch in 2016, the PRIME scheme continues to meet expectations in terms of performance. Experience and feedback from stakeholders has been implemented in the key supporting documents and guidance. Kickoff meetings and scientific advice procedures are considered key features of the scheme, enabling support that is not only provided by EMA and Rapporteurs but is coordinated across all of the Agency’s Committees. So far the Agency has received three marketing authorisation applications for products eligible to PRIME and these are still under evaluation. It is too early to draw conclusions on whether PRIME is meeting its objectives and thus facilitating accelerated assessment and, ultimately, timely access to patients. This will be the subject of future analyses and reporting once more applications have been received and assessed.

Industry embraces the virtual trial platforms

Caterina Lucchini
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Industry embraces the virtual trial platforms

The idea of ​​conducting “site-less” or “virtual” clinical trials has been around for some time, and like many new insights, has collected discrete scepticism. However, the industry has recently begun to see the possibility of realizing this approach and the benefits it can bring to patients and sponsors. The columns of Nature Drug Discovery of the month of May have dedicated two pages to the topic of virtual trials, ie clinical trials conducted limiting or eliminating the need to make patients recruited at research Centres. The attention is also high from a Regulatory point of view: companies must be sure to completely adhere to laws and guidelines requests; in this sense, both the GCPs in the second revision (validation of computerized systems, risk analysis) and FDA guidelines were implemented. The American agency seems to be ahead of EMA on this point to date.

Virtual trial

Virtual clinical trials are a relatively new method of conducting a clinical trial to collect data on the safety and efficacy of a molecule or medical device. These trials take full advantage of technology (apps, monitoring devices, etc.) and inclusion platforms (recruitment, informed consent, counselling, measurement of endpoints and any adverse reactions) to allow the patient to be homebased at every stage of clinical trial. By relying on the new possibilities offered by technology, many argue that virtually conducted clinical trials offer an opportunity for a more patient-centered approach.

Pioneeristic virtual studies

The first partially virtual study dates back to 2001, when Eli Lilly tested his drug tadalafil to combat erectile dysfunction with asking men, in addition to participating in visits to the centers of reference, to fill out an online questionnaire. After 10 years it is Pfizer’s time, which paved the way for the first totally virtual clinical trial model with the REMOTE (Research On Electronic Monitoring of Overactive Bladder Treatment Experience) hyperactive bladder study. The study was the first randomized clinical trial with recruitment and collection of patient data included via web and smartphones. One of the main objectives was to compare the virtual approach with a conventional Phase IV clinical study to determine if the virtual experimentation project could be a useful model for future studies. Unfortunately, the REMOTE study by Pfizer has faced a number of challenges, not least the problem of recruiting patients (most of the members of the target group were elderly, so with limited skill in the use of technology).

Probably, as long as the use of technology and this new way of conducting clinical trials will not capture the trust of patients, the right approach is a step by step one, in which virtual study is not the only possibility offered, but starts to be presented among the possible options. A so-called hybrid approach, as Genentech has carried out with its head to head trial on rituximab and mycophenolate mofetil in patients with pemphigus vulgaris, a rare autoimmune skin disease. Recruitment through the “virtual” channel (especially through Facebook and Google AdWords) was faster than the standard one that included 21 research centers involved. This speed was again on display last year when Science 37 completed a 372-person, placebo-controlled trial of a topical probiotic spray for mild-to-moderate acne on behalf of AOBiome. Todd Krueger, AOBiome’s CEO, says that the study lasted less than 12 months, much faster than anticipated. In March of this year, Novartis announced that, with an agreement with Science 37, it will launch up to 10 virtual clinical trials in the next three years.

Benefits

The number of recruited patients increases considerably if the barriers linked to travel distances are broken down to reach the research centers. Furthermore, by reducing the discomfort associated with travel and simplifying the collection of information, it is hypothesized that virtual trials can reduce the current high drop-out rate of patients involved in phase III studies (equal to 40% today). «This is a very important and interesting point – confirms Lorenzo Cottini, vice-coordinator of the AFI (Associazione Farmaceutici industriali) Clinical Trials study group, and President of High Research CRO. There is already enough technology to avoid as much as possible the patients’ movement and therefore increase the compliance in the study. You could make “remote” visitis for those patients who are not able to move but have a minimum of technology (sometimes even just a tablet) or caregivers who can help them. In order not to select patients and enlist only the most “technological” ones, this type of view could be added to the traditional ones – as said the hybrid approach». About the use of technology, electronic medical records can help to identify subjects with specific targeted characteristics and to increase awareness of experimentations and recruit subjects directly. From an economic point of view, although this new approach requires strong technological support and the movement of health personnel, it is believed that savings can be achieved both because time is reduced and because the traditional set-up of multiple study sites is eliminated. Virtual testing also offers the possibility of reducing risks in the drug development process. The “live” collection of data thanks to the monitoring devices allows the investigators to calibrate, modify, and possibly even interrupt the study more easily. This is to the benefit of patient safety, allowing a more specific study design and limiting costs in case of study failure. Finally, the virtual experimental design could allow groups with an interest (investors, doctors, government agencies, patient advocacy groups and even patients themselves) to have more opportunities to play an active role in the study.

Scepticisms

The virtual trials scare, as we read in Nature Drug Discovery, from a legal point of view. On the one hand we must guarantee the privacy of the recruited patients, on the other we must be sure of the truthfulness of the collected data, otherwise the results of the study would be compromised. This is perhaps the main reticence of the industries in the sector. Another perplexity concerns the small number of clinicians interfacing with patients: virtual studies must guarantee the same quality standards as standard ones. To increase enrolment and limit the abandonment of virtual trials, as had happened in the REMOTE study, the idea of ​​rewarding the participants was carried forward. The so-called virtual gaming, tested for example by EmpiraMed, in which, in the specific case proposed, the patient received points for each goal achieved (registration, recruitment, etc) to be used for gift cards or to make a donation to an association. «This approach – said Cottini – could introduce ethical evaluations, but it is a point to be taken into consideration: our legislation, in Italy, would not allow it at the moment». According to Jason Bobe, who studies patient engagement at the Icahn School of Medicine at Mount Sinai, the main problem is that «industry still tends to see individual patients as case studies and not as active participants».

FDA point of view

Though the FDA has stated that they see benefits in the appropriate use of technology in clinical trials, they are still in the process of learning about virtual clinical trials, the bring-your-own-device (BYOD) model of provisioning and other aspects of today’s tech-enabled research environment. In particular, the agency is looking for input on four specific issues: how the FDA could encourage the adoption of technological tools in clinical trials; what are the barriers; how the new research models will influence patients and the eventual limiting action of regulatory requirements. Not surprisingly, the FDA is looking for input, as supporting and encouraging innovation is the agency’s job. To this end, the guidance document issued by the FDA “Use of electronic informed consent in clinical investigations” explains how federal regulators allow companies to use electronic media (such as interactive websites) to facilitate the informed consent process. This will certainly help companies to conduct virtual clinical trials. The FDA has been in favor of virtual testing Transparency Life Sciences, a Boston-based biotechnology start-up, for example, recently received FDA approval for a fully tele-monitored test protocol in just 30 days, and was encouraged to make more innovation in the direction of virtual experimentation.

Future

Looking to the future, different scenarios seem plausible. Perhaps virtual studies will increase instead of replacing traditional study practices and workflows. The study’s virtualization aspects can be exploited when circumstances require it – similar to how remote monitoring streams are being adopted by the study supervision teams today. On-site monitoring is still a pillar of the study, for example, but much of the data is monitored remotely, depending on the case «CRO, emphasizes Cottini, and companies are already equipped to reduce (maybe not to zero) the monitoring at the experimental centers for a “physical” check of the folders, reports and consents (the so-called “source data” for the GCPs)». Maybe virtual clinical trials will be used in areas where traditional models have failed (eg for geographically dispersed groups or rare disease populations). Interestingly, the Sanofi VERKKO study does not test a drug but instead focuses on a wireless glucometer. Perhaps virtual studios will lend themselves well to the evaluation of medical devices such as sensors and diagnostics, which will continue to increase in importance as technology evolves. As Pfizer’s experience has shown us, it is not an easy road, but it is likely to offer significant benefits for some studies and selected patient populations.

Amazon’s first steps into the business of life sciences

Caterina Lucchini
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Amazon’s first steps into the business of life sciences

The giant of retail e-commerce Amazon, an investment holding under the control of multi-billionaire Warren Buffet, Berkshire Hathaway, and one of the main world-wide investment banks, JPMorgan Chase & Co., closed a new partnership at the end of January 2018 to create a new, no profit company to jointly provide healthcare services to their US employees (approx. 750,000 people, according to the estimates published by Forbes, equal to some $ 7,5 billion/year combined healthcare expenditure). A deal that sounded “strange” to Glenn Luk, founder of the shared knowledge platform Quora, commenting the operation from Forbes’ pages. «Capitalism is not altruistic and one should always be suspicious of capitalists masquerading as altruists», he wrote.

According to the announcement made by the three partners, the declared goal of the operation is to achieve a better satisfaction of their respective workforces. Furthermore, the new, no profit company will provide the founding partners with the needed healthcare technological solutions and services, thus reducing their costs on the long term thanks to the complementarity of the expertise the three partners can provide to the newco. «The ballooning costs of healthcare act as a hungry tapeworm on the American economy. Our group does not come to this problem with answers. But we also do not accept it as inevitable» said Berkshire Hathaway Chairman and CEO, Warren Buffett, commenting the deal. A declaration of intent that let suppose the final cut of healthcare costs might be the real objective of the three partners.

The real target might be the pharmaceutical market

According to the CNBC, quoting a report from the financial research firm Leerink, Amazon ’s exploratory team working on the pharmaceutical and healthcare sector should have reached 30 to 40 people. Another clue in favour of the hypothesis of an interest of the e-commerce giant to enter the online pharmacy channel sooner or later. Another tip towards a new role for Amazon in the prescription drugs’ market is the approval that the company obtained during Fall 2017, according to the St. Louis Post-Dispatch, for wholesale pharmacy licenses in at least twelve US states (Nevada, Arizona, North Dakota, Louisiana, Alabama, New Jersey, Michigan, Connecticut, Idaho, New Hampshire, Oregon and Tennessee). These pharmacy licenses should not be used to sell prescriptions medicines, reported Christina Farr for CNBC, «but (Amazon, ndr) will use them to sell medical devices and supplies instead, the company has told regulators».

These first steps made by Amazon into the healthcare business might represent just a starting point from which to experiment completely new models for the medicinal and medtech’s supply chain based on the company’s advanced knowledge in the field of efficient and low cost goods distribution. How this will impact on current models of pharmaceutical distribution is too early to say, but observers agree on the fact that the initiative might put pressure at the regulatory and legislative level to keep prices low and under control.

Observers also hypothesised that Amazon itself might become in future a pharmacy benefit manager (Pbm) in the US, thus directly negotiating the prices of healthcare products and services with regulators, pharmaceutical companies and insurances. Something that might disrupt the current model and significantly reduce prices for prescription drugs, as Amazon might directly act to negotiate costs and reimbursement plans with the pharma and biotech industry.

Competitors are not waiting

Home distribution has already become a realty for prescription medicines, at least in the US, as many new initiatives have been started by traditional players in order to protect their markets from the arrival of Amazon.

Cvs Health announced in December 2017 the intention to start next-day delivery of prescription drugs in New York (and same-day service to follow in some big cities during 2018). Cvs is one of the leading pharmacy benefit manager companies (Pbm) in the US; it also controls Caremark, among the main US’s online pharmacies, and acquired Aetna in 2017, an insurance company active in the private healthcare market.

Another big player, Walgreens Boots Alliance, closed a partnership with FedEx for package pickup and drop-off services at more than 7,500 Walgreens locations in all 50 U.S. states. Walgreens also started a collaboration with Prime Therapeutics, an healthcare insurance covering more than 20 million people in the US.

The discount company Target, counting more than 1,600 internal pharmacies to its shops, also closed an agreement for the acquisition of the leading Shipt’s online same-day delivery platform, a $550 million deal in cash. According to Pharmaforum, Target might represent the final object for an acquisition by Amazon, with the aim to directly access its prescription medicines selling structure on the internet. Another possibility might see the opening of Amazon’s pharmacies into the grocery Whole Foods retail network Jeff Bezos’ company acquired in June 2017 for $13.7 billion.

What is gong on in Europe

The new partnership between Amazon, Berkshire and JPMorgan is focused on the US market, but it might pave the way to similar initiatives to be pursued also in Europe in coming years. Here too, the trend indicates a tendency towards the consolidation of a new model of pharmaceutical distribution more centred on the online channel and the creation of pharmacies’ networks.

In Germany, for example, 2017 saw the launch of the digital branch “Linda 24/7”, a modern multi-channel ordering portal open 24 hours a day, seven days a week, offering Click & Collect services, expert advice on location and use of Payback points. The project is there result of the cooperation between approx. 1,100 owner-managed, independent German pharmacies adhering to the Linda umbrella network. An initial pilot phase of the project should provide feedback from customers upon which to optimise the digital branch before the official presentation.

In other countries the online market is still far behind, in favour of an approach up to now more focused on the creation of a wider concentration of retail pharmacies. One of the main wholesale and retail company pharmacy operators of the European market, Celesio Ag, underwent a renaming in September 2017 to become McKesson Europe. Its network counts more than 2,100 own pharmacies and about 300 managed ones, serving every day more than 2 millions European citizens. McKesson Europe also supplies approx. 55,000 pharmacies and hospitals in ten European countries via its 110 own and seven managed wholesale branches.

Another example comes from Central and Eastern Europe, where the Dr. Max brand represents the largest pharmacy network with more than 1,300 pharmacies and total sales of € 1.25 billion. The brand is owned by the investment group Penta, it is currently present in the Czech Republic, Slovakia, Poland, Romenia, Serbia and is undergoing expansion also towards the Italian market, where 2017 saw the opening to capital-owned pharmacy networks.

Negative topline results of intepirdine phase 3 MINDSET trial in Alzheimer’s Disease

Caterina Lucchini
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Axovant Sciences announced that the Phase 3 MINDSET clinical trial of its investigational drug intepirdine in patients with mild to moderate Alzheimer’s disease (AD) who were receiving background donepezil therapy did not meet its co-primary efficacy endpoints. At 24 weeks, patients treated with 35 mg of intepirdine did not experience improvement in cognition or in measures of activities of daily living as measured by the Alzheimer’s Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) and by the Alzheimer’s Disease Cooperative Study-Activities of Daily Living scale (ADCS-ADL), respectively, compared to patients treated with placebo. In the study, intepirdine was generally well tolerated. After 24 weeks of treatment, change from baseline in cognition was non-significantly improved in the intepirdine arm versus the placebo arm (0.36 ADAS-Cog points; p-value = 0.22). In addition, there was essentially no difference between the intepirdine and placebo arms in change from baseline in activities of daily living (0.09 ADCS-ADL points; p-value = 0.83). Of the endpoints analyzed to date, the only endpoint in which any significant improvement was seen in the intepirdine arm versus the placebo arm was in the first key secondary endpoint, the Clinician Interview-Based Impression of Change plus caregiver interview, or CIBIC+ (0.12 CIBIC+ points; p-value = 0.02). The Company will work with investigators to conclude the MINDSET open-label extension study.

Alzheimer’s disease: trigriluzole phase 2 trial in collaboration with ADCS

Caterina Lucchini
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Biohaven Pharmaceutical Holding Company Ltd., a clinical-stage biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases, has announced its clinical trial collaboration with the Alzheimer’s Disease Cooperative Study (ADCS). The ADCS is a leading Alzheimer’s disease (AD) clinical trials research consortium that receives major support from the U.S. National Institute on Aging (NIA), a part of the US National Institutes of Health. The ADCS was developed to promote the discovery, development, and testing of new drugs for the treatment of AD. The ADCS includes a coordinating center housed at the University of California San Diego School of Medicine and a robust network of clinical research sites located at major academic medical centers and private clinics specializing in AD care and research throughout the United States and Canada. The ADCS has successfully completed the largest number of clinical trials in the AD field while also developing some of the important instruments for use within clinical trials. The ADCS has been undertaking the development efforts for this study over the last six months in partnership with Biohaven. The company will work with the ADCS to evaluate trigriluzole in patients with AD. Trigriluzole is a third-generation prodrug and new chemical entity that modulates glutamate, the most abundant excitatory neurotransmitter in the human body. Agents that modulate glutamate neurotransmission may have therapeutic potential in multiple diseases that involve glutamate dysfunction. As part of this collaboration, Biohaven will fund a randomized, double-blind, placebo-controlled, Phase 2 clinical trial to evaluate the efficacy and safety of trigriluzole in patients with mild-to-moderate AD.

NBTXR3 will be tested in combination with immune checkpoints inhibitors

Caterina Lucchini
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Nanobiotix, a late clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, has recently announced its intention to start a new trial in the company’s immuno-oncology (IO) program. The trial is aimed at expanding the potential of NBTXR3 to recurrent and metastatic disease. The trial would target recurrent head and neck, and metastatic lung cancer patients. Studies indicate that the vast majority of oncology patients do not respond to checkpoint inhibitors. Nanobiotix will evaluate its lead product, NBTXR3, in combination with immune checkpoint inhibitors, with the aim of unlocking their vast potential to convert refractory patients into responders. For the past decade, there has been excitement around immuno-oncology agents’ capacity to boost the immune system’s response, priming it for an active attack against tumor cells. The response to checkpoint inhibitors in so-called “hot” tumors, infiltrated by T-cells and characterized by an inflammatory profile, has been striking with long-lasting clinical benefits in many cancer patients. However, many tumors exhibit little or no response to therapies targeting the immune system and are considered “cold”, due to a lack of immunogenicity. According to published data, only 15 to 20% of non-small-cell lung cancer patients (NSCLC), and 13 to 22% of head and neck squamous cell carcinoma patients (NHSCC) respond to immunotherapy treatments. Moreover, treatment using checkpoint inhibitors is generally not effective against all tumor types (“cold” tumors devoid of T-cell saturation, PD-1/PDL-1 blockage cannot drive an anti-cancer response). The physical mode of action and subsequent cell death generated by NBTXR3 induce a different immunogenicity compared to radiotherapy and chemotherapy. This could be the key to significantly increasing the number of cancer patients who can benefit from immuno-oncology strategies. As Nanobiotix reported earlier this year at ASCO 2017, NBTXR3 activated by radiotherapy was shown to induce a specific adaptive immune pattern that could potentially convert a non-responder into an immune-responsive patient receptive to treatment with checkpoint inhibitors. On top of NBTXR3’s core developments as a single agent across seven oncology indications, Nanobiotix’s Immuno-Oncology combination program opens the door to new developments, potential new indications, and important value creation opportunities.

Good results for a phase I/II head and neck cancer trial

Caterina Lucchini
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NANOBIOTIX a late clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, presented the results of the Phase I/II head and neck cancer trial with its lead product candidate, NBTXR3, at the American Society of Clinical Oncology (ASCO), Chicago. Head and neck cancers represent a group of aggressive cancers that appear in the mouth, nose, sinuses and at the top of the aerodigestive tract. This disease is a major public health concern in USA, Europe and Asia. Nanobiotix’s Phase I/II head and neck trial targets frail and elderly patients (more than 70 years) who have advanced stage III/IV cancer with very limited therapeutic options.

The only available treatment for these patients is radiotherapy, as their condition does not allow them to receive a combination of radiotherapy and chemotherapy, which offers a better survival outcome. These patients with radiotherapy treatment alone have a poorer outcome with lower Response Rate, and shorter Overall Survival.

Uncontrolled tumor growth in such population will significantly decrease patients’ Quality of Life because basic functions such as swallowing, breathing, speaking and eating are impaired. NBTXR3 has demonstrated an excellent safety profile, with no Adverse Events (serious or not) related to the product.The radiotherapy safety observed in the trial has been strictly the same to the IMRT well-known toxicity. This is an important finding, given the elderly and frail population treated in this trial. Additionally, the injection was demonstrated to be feasible and appropriate as the product remained in the tumor from the first day until the last day of radiotherapy. Marginal passage in the blood circulation has been observed during injection time. No leakage in the surrounding tissues have been observed. The highest dose (22%) continues to be evaluated. The Overall Response Rate (Partial Response plus Complete Response) was evaluated as per RECIST 1.1. The first data showed promising signs of antitumor activity.

The Overall Response Rate is 91% (10 out of 11 patients evaluable) and 7 out 9 patients (78%) had Complete Reponse at 10% dose level or more. In addition, the tumor response suggests a dose dependent effect (see figure below: waterfall plot). So far, all of the patients treated at higher dose levels (15% and 22%) have shown a prolongated Response with no loco-regional or distant relapse, with a medium follow up of 12 months. Looking at the future, Nanobiotix is filing a protocol amendment of this study to include 44 additional patients in an expansion to demonstrate the efficacy of NBTXR3. Nanobiotix is opening 12-15 additional sites in Europe to expand the development of this indication. The company plans to expand this study in the US.

 

Results of the antibiotic cadazolid

Caterina Lucchini
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Swiss biotechnology company Actelion presented mixed trial results for its antibiotic hopeful cadazolid, saying it met the main goal of one late-stage study but failed to hit the target in a second, identical study. Cadazolid, being tested against tough-to-treat Clostridium difficile-associated diarrhoea, is among assets being purchased by Johnson & Johnson as part of the U.S. healthcare giant’s $30 billion takeover of Actelion.

Actelion said the investigational drug met its primary endpoint – resolution of diarrhoea and no further need for therapy on study treatment, maintained for two days after treatment – in its pivotal IMPACT 1 study, but fell short of the same endpoint in its IMPACT 2 study. The company was silent on the future of the drug.

The Swiss company, which said cadazolid demonstrated an acceptable tolerability and safety profile, now plans to analyze the full study results and make them available at upcoming congresses and in peer-reviewed publications.

Chinese cell therapy effective in small multiple myeloma trial

Caterina Lucchini
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A small trial conducted in China found that an experimental therapy using altered cells to recruit the body’s immune system to attack cancer can induce remission in most patients with advanced multiple myeloma, a blood plasma cancer.

The study of 35 patients tested a chimeric antigen receptor T-cell (CAR-T) therapy developed by China’s Nanjing Legend Biotech Co. The drug candidate, known as LCAR-B38M, targets a protein called BCMA found on cancerous blood plasma cells – the same target being pursued by Bluebird Bio Inc and Celgene Corp with their CAR-T called bb2121. The American Society of Clinical Oncology, which featured the data here at its annual meeting, said that out of 19 trial patients followed for more than four months, 14 reached complete remission.

One patient had a partial response and four patients reached “very good partial remission,” but the cancer did get worse in one of those patients. Eighty five percent of trial patients experienced cytokine release syndrome (CRS), a potentially life-threatening inflammatory condition, but researchers said the side effect was temporary and manageable in most patients. Two people had severe CRS, but recovered after treatment with Actemra, an anti-inflammatory drug.

The study is being conducted at Second Affiliated Hospital of Xi’an Jiaotong University in Xi’an, China.

 

New vaccine to protect adolescent and adult from Men B

Caterina Lucchini
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0

The European Commission granted a marketing authorisation valid throughout the European Union for Trumenba on 24 May 2017. Trumenba is a Pfizer vaccine used to protect individuals from 10 years old against invasive meningococcal disease caused by Neisseria meningitidis group B.

Invasive disease occurs when these bacteria spread through the body causing serious infections such as meningitis and septicaemia. Trumenba contains two components, proteins that are found in the outer coats of Neisseria meningitidis group B bacteria. These proteins are fixed onto a compound containing aluminium, which helps to stabilise them, allowing the immune system to respond to them.

Trumenba has been shown to trigger the production of protective levels of antibodies against Neisseria meningitidis group B in two main studies. The first study involved around 3,600 participants aged 10 to 18 years, and the second study involved around 3,300 young adults between 18 and 25 years of age; none of the participants had previously been vaccinated against N. meningitidis group B. Those taking part were given 3 doses of the vaccine and antibody response against 4 main test strains of the bacteria was measured one month after the last injection. The studies also looked at response to 10 other, secondary strains of N. meningitidis group B.

Antibodies were produced in sufficient quantities to provide protection against the 4 main test strains in between 80 and 90% of those in the first study, depending on the strain; 84% of those given the vaccine had protective antibodies against all 4 strains when tested. In the second study sufficient amounts of antibodies were produced in 79 to 90% of cases, and protective levels of antibodies against all 4 strains were seen in 85% of participants. Antibody responses were also seen against the 10 secondary strains and confirmed the responses seen with the 4 main strains. Supportive studies were also carried out, which showed that 2 doses of the vaccine achieved a broadly similar antibody response to 3 doses, and that even though protective antibody levels declined over time they could be improved by an additional booster dose after both 2- and 3-dose treatments.

The most common side effects with Trumenba are pain, redness or swelling at the site of the injection, headache, tiredness, chills, diarrhoea, nausea, and muscle or joint pain. The available data indicated that Trumenba should provide broad protection against the strains of Neisseria meningitidis group B that are currently found in Europe, whether given in a 3-dose or a 2- dose schedule. As the protection provided seemed to decline over time, a booster dose should be considered in recipients who were thought to be at continued risk of invasive meningococcal disease.

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