Sci­en­tists un­furl­ing the hu­man genome at the turn of the cen­tu­ry came across many sur­pris­es but per­haps noth­ing as shock­ing as the per­cent­age of the genome that didn’t ap­pear to code for any­thing. Around 2% wrote pro­teins and the rest ap­peared to be “junk,” “dark,” or, as the New York Times then put it, “the ap­par­ent prod­uct of a typ­ing pool of drunk­en ba­boons. ”

Armed with the sup­po­si­tion ba­boons hadn’t cre­at­ed hu­man DNA, many sci­en­tists have the spent the pre­vail­ing two decades fig­ur­ing out what the so-called junk DNA is for or how it got there. One start­up out of the UK is now by­pass­ing part of that ques­tion and at­tempt­ing to lever­age seg­ments of “dark” DNA for can­cer vac­cines and im­munother­a­pies.

Kevin Po­jasek

Er­vaxx launched out of Lon­don with $17.5 mil­lion in seed and Se­ries A fund­ing on the promise that some non-cod­ing DNA in fact codes pro­teins in can­cer cells and that those pro­teins could be tar­gets for can­cer vac­cines and off-the-shelf CAR–T. The fund­ing is from SV Health In­vestors and an undis­closed glob­al phar­ma­ceu­ti­cal com­pa­ny. They’ve dubbed the tech “Dark Anti­gens” — af­ter the dark genome and a play on cos­mo­log­i­cal dark mat­ter, the vast amount of mat­ter in the uni­verse we’re pret­ty sure is there but re­mains in­vis­i­ble and large­ly in­scrutable.

“What we found is a set of se­quences in the genome that are se­lec­tive­ly tran­scribed and trans­lat­ed in can­cer and not in nor­mal cells,” CEO Kevin Po­jasek told End­points News.  “I think it’s sci­en­tif­i­cal­ly fas­ci­nat­ing that we’re sit­ting here in 2019 and find­ing new pro­teins in can­cer cells and from a ther­a­peu­tic per­spec­tive, it could make great anti­gens or neo-anti­gens for cell ther­a­py.”

Er­vaxx’s plat­form emerged out of a rel­a­tive­ly well-un­der­stood part of the dark genome, en­doge­nous retro­virus­es (ERV). Over mil­len­nia or eons of in­fect­ing hu­mans and our mam­malian an­ces­tors, these an­cient virus­es in­cor­po­rat­ed them­selves in­to our DNA and left a foot­print that ac­counts for about 8% of hu­man ge­net­ic code. Er­vaxx’s pitch is that the rapid dam­age can­cer caus­es to tu­mor DNA can lead pro­teins in these nor­mal­ly dead re­gions to be cod­ed.

It’s not clear what – if any­thing – these pro­teins do, but Po­jasek said they can func­tion as an anti­gen tar­get for tu­mor cells. He said their re­search showed that they are al­ready de­tectable to naive tu­mor cells, in the­o­ry al­low­ing Er­vaxx to use a vac­cine or a CAR-T process to then amp-up the im­mune re­sponse.

Be­cause the same anti­gens ap­pear to be present in dif­fer­ent peo­ple, they might al­low for an off-the-shelf CAR-T ap­proach, al­though Er­vaxx is al­so pur­su­ing a vac­cine and clas­sic CAR-T strat­e­gy. Its lead pro­gram is a vac­cine for melanoma, with oth­er ther­a­pies in the works for non-small cell lung can­cer, ovar­i­an can­cer and breast can­cer, among oth­ers. They hope to ward off re­sis­tance to any po­ten­tial ther­a­py by trig­ger­ing an im­mune re­sponse against mul­ti­ple dark anti­gens.

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Two and a half years after launch, Merck KGaA spinout iOnctura is getting its first major round of funding.

The oncology startup raised €15 million ($16.6 million) to put its lead drug into the clinic and get its second drug past IND-enabling tests. INKEF Capital and VI Partners co-led the round and were joined by the biotech’s longtime backer M Ventures, an arm of Merck KGaA, and Schroder Adveq.

California-based Amgen, which does the bulk of its business in the United States, made its ambition to reinvigorate its growth prospects by expanding its presence in Asia clear at the sidelines of the JP Morgan healthcare conference in San Francisco earlier this month.

The Thousand Oaks-based company on Thursday executed its plan to dissolve the joint venture with Astellas — created in 2013 — to operate the unit independently in Japan. With its rapidly aging population, the region represents an appealing market for Amgen’s osteoporosis treatments Prolia and Evenity as well as a cholesterol-lowering injection Repatha.