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Cell Atlas; Creutzfeldt-Jakob disease; Profitable Biotechs; Artificial intelligence; HIV

What has caught my attention in Pharma, Biotech, Biomedicine and Science in the last two weeks? Here is a selection of the news, articles, papers, findings, risings, falls as I have read, heard, discussed, been involved in. Very subjective, not pretending to be comprehensive or representative. Just my selection.

My MedScan in cw49/50. As a gift were a few days spent in hot mineral waters just in middle of the hectic month. Let´s see whether in December announced positive results from the Biogen´s Phase Ib titration study with aducanumab will be translated into an Alzheimer´s drug, transforming our lives.
And, I wish you all very Merry Christmas spent with loved people and without stress.

Here we go:

Cell Atlas for all
Part of the Sweden-based Human Protein Atlas, the Cell Atlas, was launched in December. The Cell Atlas shows a location of more than 12 000 proteins in more than 30 cellular structures and opens the way for “spatial proteomics”. Basis for the Cell Atlas are 56 cell lines “selected to represent various cell populations in different organs of the human body”. Protein expression is accompanied by mRNA expression profile for all human genes.

What I find interesting is a possibility to submit antibodies and get recognized as an antibody provider fulfilling high Human Protein Atlas criteria as the antibodies are always carefully validated there. Furthermore, for proteins for which there are no other antibodies available, this validation is for free.

Early detection of Creutzfeldt-Jakob disease (CJD) from cerebrospinal fluid and nasal swabbing
Prion diseases as CJD can definitely be confirmed in brain tissue by CJD-specific abnormal prion protein (PrPCJD) presence. In between, CJD diagnosis employs a set of characteristics including presence of dementia, characteristic changes seen in EEG, presence of certain proteins in CSF and abnormalities in magnetic resonance imaging. Due to variable phenotypes of sporadic forms of CJD, sensitivity of diagnostic criteria for sporadic CJD based on the analysis of probable and definite cases has been 83%, with a specificity of 71%.

By introduction of the real-time quaking-induced conversion (RT-QuIC) assay which detects femtograms of PrPCJD from all subtypes of sporadic CJD, sensitivity and specificity has been increased. Italian scientists published recently a paper in JAMA introducing a diagnostic algorithm based on the RT-QuIC of PrPCJD from CSF and olfactory mucosa (OM) samples with introducing a gentle nasal swab procedure for OM. To date, for clinically suspected sporadic CJD their diagnostic flow provides a sensitivity and specificity of virtually 100%. A limitation of this study is that all patients were already symptomatic.

Most profitable Biotech companies
Three Biotech companies with the best operating margins, are according to the article in Biospace, United Therapeutics, Gilead Sciences and Biogen.

United Therapeutics as a leader in pulmonary arterial hypertension (PAH) with 5 drugs marketed has the most successful drug Remodulin, accounted for one third of the company revenue in 2016. Potential challenges to the company is patent expiration and entrance of generics in 2018.

Gilead Sciences is a leader in hepatitis C market, with Sovaldi and Harvoni “accounted for $ 20 billion in sales last year”. Gilead however also faces the biggest patent infringement issue in the U.S and after a jury decision on December 16th , it is ordered to pay to Merck about 10% of the hepatitis C drugs sales.

Biogen with its multiple sclerosis drugs Avonex and Tysabri enhanced by Tecfidera “maintain an industry-leading operating margins of 49,3% over the last 12 months”. As I already mentioned, a few days ago Biogen announced positive news from its Phase Ib study with an Alzheimer´s disease (AD) drug candidate aducanumab. A disease modifying or a preventive drug in AD will be, once found, worthy of tens billions of EUR.

Artificial intelligence helped discover new Amyotrophic Lateral Sclerosis genes (ALS)
Artificial intelligence (AI) represented by IBM Watson helped scientists from Barrow Neurological Institute identify new genes connected to ALS, a deadly neurodegenerative disease.

Top ranked genes from a gene set identified by the AI were further explored by the team of scientists and 8 genes proved to be associated with ALS. IBM Watson and a robust database behind accelerated gene identification which would normally took years, instead of a few months needed for the machine (and of course for people teaching a machine to learn).

And there is another work in biomedicine for artificial intelligence, as announced recently. The same system will help to identify basis behind cancer treatment resistance, for MIT´and Harvard´s Broad Institute in a 5-year, 5 million USD project.

HIV drugs linked to neurodegeneration
A paper from University of Pennsylvania published in the American Journal of Pathology brings evidences that certain HIV drugs known as protease inhibitors can have toxic effects on the central nervous system via increasing of BACE1 expression.

BACE is known to be involved in processing of the amyloid precursor protein APP into Aβ which is the main component of amyloid plaques, one of the culprits of Alzheimer´s disease pathology. BACE has been also a target of several AD drug candidates in pipeline. Another enzyme, PERK, has been found to be significantly involved in this process.