How Personal Genomics Companies Are Changing Drug Development

In recent years, companies offering direct-to-consumer genomics tests have started switching the business model and venturing into drug development. Amidst criticism on data protection and lack of transparency, personal genomics companies are leveraging their unique position to generate genomics data to strike lucrative deals with pharma companies.   

Thanks to cheaper data acquisition and convenient analysis, more and more companies are offering a broad range of genomics services directly to consumers. These can assess whether an individual is carrying a genetic mutation that will make them more susceptible to a certain disease, predict how they will respond to certain drugs, or provide information on their genetic ancestry.

These services are subject to controversy, especially regarding ethical and legal perspectives. For example, low transparency on how data can be reused and limited proof that data processing is compliant with European data protection law, since most of the stakeholders are based in the United States. In some countries, providing genomics services directly to individuals is simply forbidden. French law, for example, establishes that studying someone’s genetics can only be done for medical or research purposes. 

Surprisingly, personal genomics companies managed to set up large marketing initiatives in France and other countries with similar laws, including ‘influencers’ ordering tests and presenting their results on YouTube, despite the fact that selling such genomic services is forbidden. 

These companies also previously faced a huge setback in the US. In 2008, California health regulators asked to prove a physician was involved in the ordering of each test (which was obviously not the case) and requested these companies to fulfill clinical laboratory licensing requirements. 

In 2013, the US FDA forbade these companies to give patients health information based on genomics tests. Later, in 2017, the FDA approved some of these tests for clinical purposes, but only for a limited number of diseases and on a case by case basis. 

In order to address concerns regarding data safety, some companies have started using blockchain technology to facilitate and secure genomic data exchanges between individuals and researchers. But another concern is that the strategy of companies providing direct-to-consumer tests is to keep prices as low as possible. For example, 23andme offered health reports for $999 back in 2007, whereas the price went down to $99 in 2013. 

The pricing of these services is usually really low compared to the costs of carrying state of the art whole-genome analysis. In some cases, this may result from using less sophisticated techniques that only look at a series of specific genes. But in many cases, the prices are this low because of a business model focusing on reusing data.

genomics data

Raw data acquired from and analyzed with direct-to-customer tests is of high interest and value for the scientific community and the pharmaceutical industry. In particular in the field of personalized medicine, where it can be used to identify new drug targets or to screen patients for their eligibility into clinical trials. 

Personal genomics companies forecasted that genetic and genomic data would be of interest to companies developing drugs at an early stage of their development. They stored years of data from their customers after securing their consent to permit easy data reuse.

Taking it one step further, these companies started to prospectively generate data for specific purposes. For example, 23andme gave free kits to patients with neurodegenerative disease to generate a catalog of the genomic data of thousands of patients with Parkinson’s disease in partnership with Genentech. They achieved this in record time compared to usual prospective biobanking studies requiring regulatory approval.

Some companies are proposing a new business model where they put customers with a specific profile in a waiting list until a pharma or biotech company orders their genomic sequencing. Under this model, customers get their genome sequenced (almost) for free and the company that ordered the database is able to do research on this data without a complicated recruitment process. 

In Europe, personal genomics companies such as the Italian Dante Labs are offering additional genomics reports focused on specific conditions, sometimes offering discounts for a certain disease. Earlier this year, Dante Labs partnered with BC Platforms, a world leader in genomic data management and analysis, with the goal of building Europe’s largest private genomics laboratory.

Personal genomics

Companies offering direct-to-consumer genomics tests are in a good position to become a staple in pharmaceutical research and development. The alternative to their extensive databases is to run expensive studies that often recruit small numbers of people. Furthermore, if the study has been funded by an industrial partner, the reusability of the data would be often limited due to intellectual property limitations. Governments all over the world are proposing large scale plans to generate databases such as the French Plan for Genomic Medicine 2025, the UK’s 100,000 Genomes Project, or Finland’s FinnGen. However, it takes time to generate sufficient data from these projects to generate partnerships. 

A good example of their unique position to support pharmaceutical research is their involvement in the current Covid-19 pandemic. Both 23andme and Dante Labs have announced projects that aim to mine genomics data to identify genetic differences that may explain why some infected patients wind up on ventilators while others don’t even get a cough. This data will no doubt be valuable for developing new drugs with or without pharmaceutical partners.

As a result, companies that used to provide direct-to-consumer genetic tests are switching their business model towards that of a biotech company. Through partnerships and licensing deals, these companies are able to capture a much greater portion of the economic value its genetic databases could create over time. Indeed, they could at some point have licenses on marketed drugs that could be worth billions. 

One of the most known partnerships in the field is the deal between GSK and 23andMe in 2018 to leverage genetic insights to identify drug targets and speed up clinical studies, which was widely criticized in the media. A more recent example is the partnership between Spanish pharma company Almirall and 23andme in mid January, giving Almirall the license rights to develop an antibody treatment for dermatology conditions. This made it clear that the personal genomics company is moving forward on patents covering not just genome sequencing, but also therapeutic products. 

Thanks to this strategy, 23andme is leveraging data as a valuable asset. In the future, the company is likely expected to increase its leverage in the personalized medicine field using artificial intelligence and advanced analytics technologies.

Nonetheless, personal genomics companies should remain extremely vigilant in order to ensure the sustainability of their activities. They should have full transparency on how data is reused and set up partnerships that ensure the protection and awareness of patients whose data is being used. One of the issues in this regard is that the regulatory framework for direct-to-consumer genetic testing is not fully clear in many countries, and should be reviewed in order to protect the consumers.

AI Drug Discovery Firm Exscientia Closes €54M Series C

UK-based Exscientia has closed a Series C financing round of €54M ($60M) to develop its artificial intelligence (AI) technology to guide drug discovery. 

The Danish investor Novo Holdings led the round with its first investment in the company. Additional funding also came from existing investors including drug development company Evotec and Chinese VC firm GT Healthcare Capital.

With our new funding, we intend to explore opportunities where our AI techniques can uniquely bring new insights to the creation of new medicines,” Andrew Hopkins, CEO of Exscientia, told me.  “We plan to bring projects where there is an unmet need to the clinic faster than before.” 

This funding news follows one of the first-ever phase I entries of an AI-designed drug in January, developed by Exscientia and Japanese partner Sumitomo Dainippon Pharma. Using Exscientia’s technology,  the drug’s preclinical development lasted less than 12 months – a fraction of the 4.5 year industry average.

Another big event for Exscientia was starting a partnership with the academic research institutes Diamond Light Source and the Scripps Institute in April. The aim of the partnership was to screen thousands of clinical-stage drug candidates with its proprietary AI platform for use against Covid-19. 

The flexibility of Exscientia’s platform has also led to many corporate partnerships with a focus on rare disease and immuno-oncology both collaboratively and in house. One major partnership launched this year was a deal worth up to €240M with the German big pharma Bayer with the aim of discovering new candidates for treating cardiovascular disease and cancer. Another partnership launched with the US drug developer last year is focused on developing a treatment for an undisclosed rare metabolic disease. 

The opportunities the platform gives us are broad, hence such joint ventures enable us to scale the pipeline by combining our drug discovery platform with disease-focused drug developers,” said Hopkins.

This latest Series C will allow Exscientia to expand its portfolio and pipeline, as well as grow the company internationally. The key to this is developing its AI platform to incorporate knowledge from multiple scientific disciplines in the drug discovery process. 

We believe that AI has strengths across chemistry, biology and disease,” explained Hopkins. He added that the company is building up its technology with the aim of developing “innovations across all these three disciplines, to have all of the necessary skills from ideation to proof-of-concept in the creation of a new medicine.” 

Lead investor Novo Holdings is an active investor in the European biopharma space with several different investment arms. Novo Growth was the arm that invested in Exscientia’s Series C and it has invested in expanding companies such as UK-based Oxford Biomedica, which specializes in developing lentiviral vectors for cell and gene therapy. Another arm of Novo Holdings called Novo Seeds, focusing on companies seeking seed funding, also invested in French antimicrobial company Mutabilis and Danish immune-oncology company STipe Therapeutics in the last quarter of 2019.

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Argenx Follows up Phase III Success with Giant €785M Global Offering

The Dutch biotech argenx has cashed in on recent positive phase III results by launching an enormous €784.7M ($862.5M) global share offering.

Argenx’s fundraise swiftly follows the announcement of phase III trial results earlier this week, where its lead candidate antibody drug, efgartigimod, improved motor symptoms of patients with the chronic autoimmune disease myasthenia gravis. 

Argenx is listed on the Nasdaq Global Select Market and Euronext Brussels. With its stock price rising this week by more than 30% on these markets thanks to the trial results, argenx seems to have struck while the iron was hot.

The company will use part of the cash to fund the final push to commercialize efgartigimod, with argenx planning a commercial launch of the drug for the treatment of myasthenia gravis in 2021. 

Alongside myasthenia gravis, Joke Comijn, argenx’s Director of Corporate Communications & Investor Relations, told me that the company will run clinical trials of its lead candidate in other indications such as primary immune thrombocytopenia. It will also develop other drugs on its pipeline such as a phase II-stage antibody drug for cancer.

We have an ambitious business plan,” Comijn said. “By the end of the year, we will be running four phase III trials.”

This public fundraise dwarfs others in the European biotech scene this year so far. Some of the closest include a €378M rights issue by the Danish company Bavarian Nordic in March, and the €133M raised by the Swedish biotech Oncopeptides this month. Argenx’s latest winnings also tower over the €502M that it gathered last year in a similar offering. 

Four of the biggest public fundraises in European biotech in 2020 so far. Data taken from company press releases.

labiotech top four public raises biotech 2020

After record-breaking highs in biotech funding last year, the ongoing Covid-19 pandemic has done little to discourage big fundraises in established European biotechs on the public market. However, public markets will likely continue to be volatile, including for biotech companies, and we might expect an eventual downturn as the crisis continues to lock down countries worldwide.

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Spanish Neurology Biotech Launches with €7.6M Series A

Accure Therapeutics, a neurology biotech targeting central nervous system (CNS) conditions such as multiple sclerosis and Parkinson’s disease, has launched in Barcelona with a Series A of €7.6M. 

Alta Life Sciences, a life science venture capital firm based in the same city, and the Spanish governmental institution The Centre for Technological and Industrial Development funded the round.

The new company plans to target a number of different neurological conditions and has three small molecule candidate drugs it plans to develop. Its lead candidate, which is designed to target multiple sclerosis and optic neuritis, has already been tested at phase I and the company is ready to test it at phase II.

Accure Therapeutics also has two preclinical candidate drugs for Parkinson’s disease and epilepsy in development. All three of these candidate drugs have been licensed from the Spanish biotechs Bionure and Iproteos. Laurent Nguyen, Accure’s CEO, was previously CEO of Bionure.

“The concept of Accure Therapeutics is to power an R&D and regulatory engine dedicated only to the CNS in Europe with a critical mass able to build a diversified product portfolio by carefully selecting programs on their own merits, at least in advanced or late preclinical phase to early clinical development, to progress further,” Nguyen told me.

There is a large unmet demand in the sector, but neurological diseases have proved challenging for companies to target in the past. This is particularly true for diseases such as Alzheimer’s and Parkinson’s, with many trials failing at a late stage and many big pharma companies leaving the space

“Developing new drugs in CNS conditions is challenging but the reward will be high should you be successful,” said Nguyen. “We want to capitalize on the progress made in better understanding the neurobiology and in designing adequate clinical trials… to put our candidates in the best position to demonstrate a benefit.” 

Nguyen acknowledges that the current pandemic makes setting up a new company challenging, but believes that it is possible to continue with some restrictions.

“When it comes to R&D activities, we are outsourcing our preclinical experiments to contract research organizations,” he explained. “Initiating and running clinical trials may be more challenging and we are in constant contact with our investigator’s site and CRO to anticipate, monitor, and adjust for potential bottlenecks. The rest of the year will tell exactly what the impact will be on our ability to execute clinical trials, if any.”  

It’s been a good week for neurology biotechs in Europe in terms of financing. The Swiss company Arvelle recently confirmed the final closing of last year’s Series A, which has finally bagged a total of €188M to fund the commercialization of an anti-epileptic treatment. Another Swiss neurology startup called GliaPharm raised almost €2M earlier this week to fund the preclinical development of a treatment for an undisclosed orphan neurological disease.

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French Startup Secures €2.3M to Advance Cancer RNA Immunotherapy

Lyon-based biopharmaceutical company Tollys has closed a Series A funding round of €2.3M to bring its lead RNA immunotherapy candidate to clinical trials in people with bladder cancer.

The round was closed on the basis of the company reaching preclinical proof-of-concept for the drug. It brings the total amount raised by Tollys to €6.4M since the company was founded in 2015 by scientists at the European Cancer Research Center of Lyon. 

The funds will be used to complete the preclinical development of Tolly’s lead cancer immunotherapy drug. The round will also fund the manufacture of the drug for the start of clinical trials in people with bladder cancer by late 2021 or early 2022.

Tollys’ lead candidate drug is a double-stranded RNA molecule that activates a protein called toll-like receptor 3 (TLR3), which is found on the surface of tumor cells.

When activated, TLR3 kills cancer cells and puts innate immune cells such as dendritic cells on high alert. The immune system is then trained by antigens from dead cancer cells to kill the cancer cells that weren’t destroyed by the drug and prevent regrowth of the tumor via a vaccination-type mechanism.

Tollys isn’t the only company developing a double-stranded RNA molecule to hit TLR3 in cancer. For example, Hiltonol — currently developed by the US company Oncovir in clinical trials for a wide range of cancers — consists of a mix of double-stranded RNA and small molecules. However, Tollys’ CEO Vincent Charlon told me that his startup’s cancer immunotherapy stands out with its stability and reproducible manufacturing process. 

Charlon said the company is starting with bladder cancer because the tumors are known to be sensitive to cancer immunotherapy, and the treatment can be easily administered via a urethral catheter.

Like bladder cancer, other solid tumors such as lung, breast, and colon cancer can also express the TLR3 protein and could therefore be a target of Tollys’ drug or other TLR3 activators in future.

The European startup scene has been thriving in the field of immuno-oncology in the last several months. Examples of other recent Series A investments into European immuno-oncology startups include the €13M obtained in March by Austrian company OncoOne and the €10M secured in April by Montpellier-based Phost’in Therapeutics, which is developing a drug for oral cancer.

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MSD Enters Covid-19 Vaccine Race by Acquiring Themis Bioscience

The US-based big pharma MSD has entered the race to be the first to develop an effective vaccine for Covid-19 by acquiring Austrian biotech Themis Bioscience and its vaccine technology for an undisclosed amount.

MSD has been a bit slower than some of its contemporaries to announce its plans regarding a potential treatment or vaccine for Covid-19. But its silence on this front ended this week with the acquisition of Themis Bioscience, which comes with the Austrian biotech’s vaccine candidate for Covid-19. The vaccine, developed as part of a collaboration with the Institut Pasteur in Paris, consists of a measles viral vector programmed to produce antigens of the coronavirus responsible for Covid-19, called SARS-CoV-2.

In addition, MSD also announced this week that it will partner with the US-based non-profit research organization IAVI to develop a different Covid-19 vaccine candidate using the same technology as that used in MSD’s Ebola Zaire virus vaccine, which was approved by the FDA in late 2019. It will also work with US biotech Ridgeback Biotherapeutics to develop Ridgeback’s early-stage antiviral candidate to combat the coronavirus pandemic.

This acquisition follows on from a partnership launched between Themis and MSD to develop ‘undisclosed vaccine candidates’ in August 2019.  

“The team at MSD has known about our platform and its potential for some time now,” Themis CEO Erich Tauber, told me. “The acquisition combines the complementary strengths of Themis’ unique research expertise with the measles virus vector, originally developed at Institut Pasteur in France, and MSD’s extensive infectious disease and vaccine capabilities.”

In addition to its Covid-19 vaccine candidate, which should enter clinical trials later this year, Themis is developing vaccine candidates for a number of infectious diseases such as Lassa fever. It also has some immuno-oncology therapies in preclinical development.

Themis’ most advanced candidate is a vaccine for the mosquito-borne disease chikungunya. The company had originally planned to start a phase III trial of its vaccine candidate at the end of last year, but Covid-19 has meant a change of direction.

“In the pandemic crisis that we are facing, we are prioritizing our efforts on developing a vaccine against SARS-CoV-2,” explained Tauber. “The late-stage development of our Chikungunya vaccine candidate will be parked for now.”

Themis will become a wholly-owned subsidiary of MSD. According to Tauber, the specific integration plans for the two companies have yet to be decided.

There has been much debate about how soon a vaccine for Covid-19 will be approved and on the market. Traditionally vaccines take a long time to develop, but the current pandemic has seen many possible candidates being developed in record time. As one of the most advanced examples, the US biotech Moderna plans to begin phase III testing in summer of a Covid-19 vaccine that it first designed in January.

While Themis’ approach is not as novel, or as advanced, as some of the other options being developed, it may have the advantage that it uses a known and tested platform.

“Vaccine development is a very complex and expensive process and there are several steps to ensure safety as well as efficacy as we move towards regulatory approval of a vaccine,” commented Tauber.

“Only time will tell how fast we can be, but I believe the industry will surprise us in what is possible compared to prior vaccine development timelines. Being able to respond quickly in a crisis situation such as a pandemic is critical to save lives.”

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‘Just Add Water’ GM Mosquitoes Suppress Wild Population by 95%

Water-soluble capsules containing the eggs of genetically modified mosquitoes developed by the UK company Oxitec have reduced the mosquito population in a dengue-infected urban environment in Brazil by 95% after 13 weeks.

Aedes aegypti mosquitoes carry a number of infections including dengue, Zika, chikungunya, and yellow fever. Dengue has increased almost 15-fold over the last 20 years, and is a particular problem in Brazil.

In order to reduce the spread of dengue, Oxitec releases genetically modified male Aedes mosquitoes into the environment. Once in the wild, these insects breed with local female mosquitoes and pass on a gene that causes female offspring to die at an early age. This method was reported to suppress the Aedes aegypti population by up to 96% in Brazil last year.

Normally, Oxitec — recently sold by the US company Intrexon to the US VC firm Third Security — releases adult mosquitoes into the wild from airtight vehicles, which requires specialized equipment, infrastructure, and trained staff. However, with Brazil giving full approval of Oxitec’s genetically modified Aedes aegypti today, the company recently tested a way to scale up the release of the mosquitoes.

Oxitec’s new technology encapsulates eggs containing male larvae from its genetically modified Aedes aegypti mosquito. The capsule is designed to dissolve in water and provide a cheaper and easier release method for non-experts.

“It was definitely much more labor-intensive and therefore also more expensive to do this in the past,” Nathan Rose, Head of Regulatory Science at Oxitec, told me. “This makes it much more accessible and allows us to deploy in a much wider range of contexts as well.”

The trial took place in an area of the city of Indaiatuba, Brazil, with approximately 1,000 residents. Capsules were placed in water in residential properties in the area at least once per week over 13 weeks. At the end of this period, compared with other areas of the city that had not been treated, the population of mosquitoes had decreased by 95%.

A key advantage of the capsules is that they are economical and easy to order and store. “As long as you store them within the right temperature range and humidity range, you can store them for several weeks or even months,” said Rose. “We wanted to make sure that this is going to be accessible and affordable for places that need it.”

Oxitec obtained a permit from the US Environmental Protection Agency earlier this month to trial this technology in the US. The company is also hoping to use this technology to release other species of genetically modified insects in the future, such as two species of Anopheles mosquitoes that transmit malaria.

Rose was confident that the same technology will work in Anopheles as it has in a range of other insect species. “The biology of that particular mosquito is a little bit different, so it might be that we need to tweak it slightly, but this is definitely the way we want to move in the future,” he explained.

“It might not be eggs but it might be some other life stage that is easy to transport… it’s definitely something that could be applied to other insects too.”

In addition to mosquitoes, Oxitec is developing similarly modified strains of agricultural pests such as the diamondback moth, the fall armyworm, and the soybean looper moth.

Oxitec was also the focus of a media scare in September 2019 after a research paper in Scientific Reports suggested that the surviving mosquitoes from Oxitec’s control programs could lead to hybrid strains that are more resistant to insecticides. However, the paper — labeled “inaccurate and misleading” by Oxitec — received widespread criticism for its conclusions. Objections even came from some of the paper’s own authors, one of whom claimed that the final version of the article was different from the version that the authors all agreed on.

In response to the criticisms, Scientific Reports posted an editorial addendum listing the issues with the study in March this year. The paper’s authors remain divided over whether they agree with the criticisms or not.

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Why Listening to Patient Organizations is Key in Drug Development

As the biotech industry flourishes and therapies become more targeted and personalized, the need for drug developers to interact with patients is increasing as well. While patient organizations took a backseat in the past, they are continuously gaining importance in drug development today. Nowadays, talking with patient organizations has become paramount for successful drug development, especially in the rare disease space. 

When his newborn son was diagnosed with cystic fibrosis in 2012, Daniel de Boer, then an IT entrepreneur, decided to switch careers completely. He co-founded and became CEO of ProQR, a Dutch company that develops drugs for rare diseases. Working with patients and their carers has been a priority for him ever since. 

“Patients have always been the core of [our] mission,” said de Boer. “We’ve always, from the beginning, established deep relationships with the patient communities and their patient organization representatives.”

The importance of the patient voice

patient voice, patient organizations

The key point that everyone seems to agree on is the importance of the patient voice.  Understanding what patients and their carers need versus what biotechs think they need is crucial.

“These days, patient organizations are affecting drug development in ways they never have before or that weren’t standard in the industry before,” explained Bruce Bloom,

Argenx’s Rare Disease Treatment Strikes Gold in Phase III

The Dutch biotech argenx aims to apply for FDA approval of its lead candidate efgartigimod after the first-in-class drug improved motor symptoms of the autoimmune disease myasthenia gravis in a phase III trial.

The phase III trial tested the effect of efgartigimod in 167 patients with generalized myasthenia gravis. In this incurable chronic condition, the immune system attacks proteins vital for communication between nerves and muscles, weakening muscles in the eyes, limbs, jaw, and eventually muscles vital for breathing. 

The company tested the drug in two types of myasthenia gravis patients: those whose immune system produces antibodies attacking a signaling protein called the acetylcholine receptor — the most common type — and those lacking antibodies against the acetylcholine receptor.

According to the trial results, efgartigimod improved motor symptoms for at least four weeks in 68% of patients with the most common type of myasthenia gravis, compared to only 30% of patients in the placebo group. In addition, efgartigimod allowed 40% of patients expressing antibodies against the acetylcholine receptor to show minimal or no motor symptoms, compared to just 11% in the placebo group. 

In the second type of patients, the placebo group showed a high response, and the benefits of efgartigimod were less clear. 

Buoyed by these results, argenx expects to apply for FDA approval for the treatment of myasthenia gravis by the end of 2020. Current treatments for the condition are limited to treating the symptoms with drugs such as immunosuppressants.

Patients with this devastating disease can experience chronic and potentially life-threatening muscle weakness that has a major impact on their quality of life, and more treatment options are needed,” stated James Howard, Professor of Neurology at the University of North Carolina, USA, and principal investigator for the trial. 

These data show efgartigimod has potential to make a meaningful impact on daily living activities.

Efgartigimod is an antibody fragment designed to block a protein called the neonatal Fc receptor, which prevents the removal of disease-causing antibodies from the blood. The drug therefore reduces the number of antibodies attacking the nerve-to-muscle communication machinery, and could offer a new treatment option for patients with this chronic condition.

In addition to myasthenia gravis, argenx is developing efgartigimod for other indications such as the autoimmune condition primary immune thrombocytopenia. The drug succeeded in a phase II trial in patients with the condition in 2018, and is now undergoing three phase III trials to confirm its benefits.

The latest clinical success has pushed argenx’s stocks up by more than 30% on the Nasdaq Stock Exchange as of this morning. This could enhance argenx’s already sky-high ability to raise big rounds. For example, the company launched a global offering worth a hefty €500M last year.

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Could Cell Therapy Target the Symptoms of Severe Covid-19?

The excessive inflammatory response seen in the most serious cases of Covid-19, along with shortages of ventilators, has caused a healthcare crisis in many countries around the world. Could cell therapy provide an answer to this dilemma?

Most people who become infected with Covid-19 experience symptoms such as a dry cough and mild fever, and recover without medical care. But around 14% develop more severe symptoms including pneumonia and shortness of breath. 

Approximately 5% of patients with severe cases of Covid-19 have to be put on a ventilator due to acute respiratory distress syndrome (ARDS). The patients’ lungs become severely inflamed, causing fluid to seep into the tissue from nearby blood vessels, making breathing increasingly difficult. Some of these patients can go on to develop septic shock and multi-organ failure, with almost half dying after reaching this stage.

This respiratory failure in Covid-19 is linked to an excessive immune activation that causes a ‘cytokine storm’ where the levels of inflammatory immune proteins go dangerously high. This overreaction of the immune system was also seen in SARS and MERS patients in earlier epidemics — both of which are also coronaviruses. While ventilators can help to keep these patients alive, recent shortages of this life-saving equipment in some countries have made it crucial to minimize the time spent on the machines.

Cell therapy has shown promise for treating ARDS, which (even before Covid-19) affected 500,000 people a year across Europe, the US, and Japan. The US-based cell therapy biotech Athersys — presenting at a webinar held by the Alliance for Regenerative Medicine last week — has been developing a cell therapy called Multistem that it claims “decreases the adverse results of inflammation, while activating reparative immune cells.

Multistem is an ‘off the shelf’ cell therapy derived from bone marrow donations. Eric Jenkins, Senior Medical Director and Head of Clinical Operations at Athersys, explained that the cells are not mesenchymal stem cells — adult stem cells traditionally found in bone marrow and other connective tissue — but are a different type of adult bone marrow stem cell called multipotent adult progenitor cells, and have greater growth potential in the lab. 

Athersys’ cell therapy is being trialed for reducing the damaging inflammation seen in stroke patients. It was also being developed for the treatment of ARDS even before the Covid-19 pandemic.

The company recently reported results from a completed a phase I/II study testing Multistem in 30 patients (20 treated with cell therapy; 10 with placebo) with ARDS in the US and UK. The study showed that treatment with the cell therapy reduced mortality from 40% to 25% in these very sick patients. 

We believe that, in these vulnerable patients, Multistem can effectively interrupt this pathological cycle and promote an immunologic transition that leads to patient recovery,” emphasized Jenkins.

Based on these results, and with support from the Biomedical Advanced Research and Development Authority in the US, Multistem is now being tested in a phase II/III trial for the treatment of ARDS in Covid-19 patients. Athersys is additionally collaborating with Japanese biotech Healios, which is also currently testing Multistem in its own study in Covid-19 ARDS patients.

Another company diverting its expertise to the treatment of ARDS in Covid-19 patients is Israeli biotech Pluristem Therapeutics. Earlier this month, Pluristem announced it had secured €50M in non-dilutive funding from the European Investment Bank to fund its Covid-19 therapy development.

Pluristem develops cell therapies made from placental cells. Its most advanced candidates target critical limb ischemia, where reduced blood flow often leads to amputation or death, and muscle damage after hip fracture.

We have favorable safety profile and efficacy data from hundreds of patients treated worldwide in these two indications,” commented Racheli Ofir, VP Research & Intellectual Property at Pluristem, which participated in the same webinar as Athersys.

Similar to Athersys’ therapy, Pluristem’s cells are delivered ‘off the shelf’ and also have the ability to regulate the immune system to potentially calm the excessive immune activation seen in Covid-19 ARDS patients. The company announced in March that it planned to work with the Charité University of Medicine in Berlin to develop a cell therapy to treat these patients.

Since March, Pluristem has treated a number of patients with Covid-19-induced ARDS in Israel and in the US as part of a ‘compassionate use’ program. The company followed up  seven patients in Israel and one in the US for 28 days after treatment with the cell therapy. Pluristem found that 87.5% survived, 75% no longer needed ventilation and 62.5% had been discharged from hospital.

The company now plans to start a phase II trial of its therapy in the US, which has been cleared by the FDA, and is waiting for clearance to start a trial in Germany and Italy. 

These are just two examples of companies working to develop effective treatments for the severe symptoms seen in some Covid-19 patients. Another advanced example in clinical development is a cell therapy developed by the Australian biotech Mesoblast, which began a phase II/III trial this month. All in all, the Alliance for Regenerative Medicine is currently tracking 24 ongoing clinical trials and an additional 25 preclinical studies for cell therapies against Covid-19. 

Although these therapies seem to offer hope for the minority of patients who become seriously ill with Covid-19, it’s important to note that many of the studies that have been carried out so far are very small and may not represent efficacy on a larger scale. 

In the circumstances of the current pandemic, both Jenkins and Ofir think they will be able to recruit full cohorts for their respective clinical trials fairly quickly. But it is likely that we will have to wait for a few more months at least to find out whether these cell therapies can be effective on a larger scale for patients with ARDS. 

If they do work out, they could provide an extra line of defense in countries heavily affected by the Covid-19 pandemic, as well as any subsequent waves in the near future.

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Evonik and Beiersdorf Team Up to Turn CO₂ into Skin Care Products

The German companies Evonik and Beiersdorf will work together on a joint research project to investigate how bacteria can be used to turn carbon dioxide (CO₂) and water into raw materials for skincare products.

The process will be built on Evonik’s artificial photosynthesis technology – being developed independently of this project in collaboration with Siemens – and will be supported by €1M of funding from Germany’s Federal Ministry of Education and Research (BMBF).

Photosynthesis is the means by which plants use energy from light to convert CO₂ and water into sugars. Artificial photosynthesis mimics this process using bacteria rather than plants, to turn CO₂ and water into various different chemicals.

In this case, the chemicals produced will be raw materials for Beiersdorf to use in its skincare products, which include the well-known brands NIVEA and Eucerin. Evonik hopes that the first of the substances they develop will begin to undergo laboratory testing by the end of the year.

In 2016, the German government announced plans to make the country greenhouse gas-neutral by 2050 in fulfillment of the global Paris Agreement signed in 2015. To contribute to this goal, Evonik and Beiersdorf’s collaboration is part of BMBF’s P2X II Kopernicus, a project involving 42 academic and industrial partners with the aim of developing ways to produce high-quality end products such as fuels, plastics, and chemicals using renewable energy. 

Both Beiersdorf and Evonik themselves have ambitious environmental goals. A representative from Evonik told me that, by 2025, the company aims to reduce its absolute greenhouse gas emissions by 50% compared with 2008.

Using CO₂ as a raw material contributes to the reduction of CO₂ levels in the atmosphere,” the representative said. “We are convinced that industry is the key to driving forward sustainability, environmental protection, and the circular economy.”

The Berlin-based non-profit organization Climate Analytics reported in late 2019 that the EU’s efforts to reduce greenhouse gas emissions are currently “insufficient” for meeting the Paris Agreement objectives. While global emissions of greenhouse gases are likely to fall by around 11% in 2020 due to the Covid-19 pandemic lockdowns, Climate Analytics recently said that the pandemic is only delaying an increase.

If projects like Evonik and Beiersdorf’s collaboration gain traction, they could help the EU to keep down its greenhouse gas emissions and meet the ambitious Paris Agreement aim of limiting global temperature rise to less than two degrees centigrade above pre-industrial levels.

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Phase II Fail Ends Swiss-US Drug for Rare Kidney Disease

An initially promising drug has failed to show benefits in a phase II trial for the rare kidney disease focal segmental glomerulosclerosis (FSGS), prompting the US biotech ChemoCentryx and its Swiss collaborator Vifor Pharma to drop it for this indication.

Topline results from the phase II trial showed that 12 weeks of treatment with the drug candidate did not decrease urine protein levels significantly more than placebo in 46 patients with the disease. Normally, healthy kidneys let very little protein pass through; higher protein levels indicate there is kidney scarring and damage caused by this condition.

The company will now terminate development of the late-stage oral compound for FSGS. There are few approved treatments for the disease, including steroids and diuretics, and the condition can be resistant to steroids and prone to relapse.

In a 2014 phase II trial, the candidate drug had shown significant benefits for patients with diabetic nephropathy, a kidney condition common among type 2 diabetes patients. 

ChemoCentryx extended an existing collaboration with Swiss pharma company Vifor Pharma in 2016 to include the development and commercialization of this same drug. According to the terms of the deal, ChemoCentryx and Vifor Pharma would jointly develop the drug for the treatment of FSGS, while Vifor Pharma would have the sole rights to developing the candidate for more prevalent kidney diseases such as diabetic nephropathy. So far, Vifor Pharma doesn’t seem to have exercised its rights on the more common indications. 

While the failure of this phase II trial may be a setback, the partners have a more advanced drug in development: Chemocentryx’s lead drug candidate avacopan, for the treatment of inflammatory and autoimmune diseases. They expect to file for FDA approval by mid-2020 for a rare inflammatory condition, with results from two additional trials expected by the end of the year.

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