R&D, research

Traditionally the pharma industry has lent towards the view of a one-size fits all approach to the application of therapeutics. Now, eyes are set firmly on the notion of personalised medicine – therapeutics which are tailored to a patient’s specific biomarker information . The progression of next generation sequencing and genome technology is allowing for the first tangible personalised therapies to emerge.

However, there are still major strides to be made within molecular diagnostics and the sequencing of genomes. The high price tags attached to some precision medicines have been labelled as a blockade to their application.

Regina Au of BioMarketing Insight stated that the realisation of precision medicines can’t be attributed to a finite level of individuals because it exists as an industry wide culture shift. On this subject, she stated:

“The whole industry is striving towards personalized medicine, but getting there is not easy. If you define personalized medicine as "treating the right patient with the right medication," there have been a lot of companies … working on this with the existing medications, for example in the area of breast cancer.

“...If you are trying to develop a treatment for a disease that has no treatment or very few options, you have to understand the disease first to develop a treatment. This is difficult particularly for neurodegenerative disease[s] like Alzheimer's , dementia, Parkinson's disease where scientists have been working on this area for decades and have had many trial failures. The industry has realized that in order to accelerate this process for any disease, one has to use biomarkers. However, to find these biomarkers, it wasn't until we had the research tools like Next Generation Sequencing (NGS) for genome sequencing at an affordable price scientist[s] [were then] able to use this technology to move the science forward in trying to understand diseases and develop diagnostic tools.

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“In order to determine whether a drug will work on a patient, rather than trial and error, [we refer to] companion diagnostics with therapeutics. However, many companies that are developing a drug, don't want to [enter] the diagnostic business because it is not their area of expertise and it is a different business model... Therefore, some companies have partnered with diagnostic companies in developing companion diagnostics and therapeutics in parallel. The FDA has paved a regulatory path to review and approve submissions simultaneously rather than on separate tracks due to the fear of the drug being approved before the diagnostic test. - List of approved companion diagnostis1

“Some companies have been able to develop a therapeutic drug without a companion diagnostic test to determine whether the drug will work or not because the company has been able to get tissue samples to test it while developing the drug.

“According to Phrma.org, a recent industry survey revealed that 42% of new medicines in the pipeline have the potential to be personalized medicines.2

“If we focus on mutations, we have to credit all the people involved with HER2 and the development and approval of Herceptin by Genentech back in 1998. The people who may have discovered the mutation [might] not necessarily be the person in the company that develops the drug."

Regina notes that complications can be encountered with diseases that block the ability to obtain tissue samples like CF or cancer from biopsies. She adds that in these cases a blood test, an imaging test or both measures would have to be used.

Key players within personalized medicine include the following:

Adarza BioSystems, Inc., And Horizon Discovery Group

Back in 2014, these two firms entered an exclusive agreement which was labelled as a partnership that would boost the progress of personalised medicines. The deal entailed providing Horizon with the right to supply services on Adzarxa’s multiplex immunoassay technology to launch a portfolio of products able to identify between 100 and 400 analytes as well as custom assay development.

Genentech, Vertex Pharmaceuticals, Bristol Myers Squibb

In the regions of immuno-oncology - CAR-T and checkpoint inhibitors.

Blue Bird Bio

Combining gene therapy, gene editing and immuno-oncology for severe and rare disease and cancer.

Editas Medicine

With its CRISPR Cas9 focused efforts, Editas Medicine filed for an IPO this year and commenced in trading shares in February. In addition to this, the firm just reached a milestone valued at $2.5 million under its collaboration with Juno Therapeutics, Inc.

APPG and the UK Government:

The end of Q1 witnessed the launch of the APPG on Personalised Medicine in the UK. This group signalled the Government’s dedication to assisting the route of personalised medicine to end users. The forum evaluates the method’s potential, ways to counter challenges and the requirements that arise from the treatment's clinical implementation in the NHS. (1) Some have noted that personalised medicines can stand as an apt medium to prevent disease.

FDA

The US regulatory body has been praised for its support for precision medicines and personalised drug dosing. Between 2012 to 2015 the FDA’s Center for Drug Evaluation and Research (CDER) is said to have approved 30 targeted therapies. (4)

EU

Last October, policy makers, patients, industry representatives and the scientific allergy and asthma community came together at the European Parliament to discuss the need for precision medicine.

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When considering the hottest areas of research relating to personalized medicines, Regina Au highlighted the likes of:

· Next generation sequencing

· Big Data

· Gene therapy

· Gene Editing - CRISPR technology, although there are ethical debates surrounding this method

· Microbiomes - President Obama announced on May 13, 2016, to allocate $121 million for a National Microbiome Initiative to understand the complex interplay between humans and the trillions of organisms we host. There are a number of companies focusing on this area.

Resources

1.https://www.linkedin.com/pulse/personalised-medicine-all-party-parliamentary-group-launched-mcnamee?trkInfo=VSRPsearchId%3A1584559191463394044226%2CVSRPtargetId%3A7994141592194527895%2CVSRPcmpt%3Aprimary&trk=vsrp_influencer_content_res_name

2. http://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/pmc_the_case_for_personalized_medicine.pdf

3. http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/PersonalizedMedicine/UCM372421.pdf

4. http://blogs.fda.gov/fdavoice/index.php/tag/personalized-medicine/

5. http://www.pharmatimes.com/Article/15-11-18/NICE_says_no_to_personalised_bowel_cancer_drugs.aspx

6. https://www.horizondiscovery.com/about-us/news/horizon-discovery-group-plc-and-adarza-biosystems-enter-supply-licensing-agreement

7. http://www.herceptin.com/

8. https://www.whitehouse.gov/the-press-office/2016/05/12/fact-sheet-announcing-national-microbiome-initiative

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