An­oth­er Ra­maswamy stunt plays well on Wall Street, Mar­tin Shkre­li on shame, and more

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

Ra­maswamy strikes biotech gold, again, with trade­mark IPO stunt

Last year’s big IPO for Ax­o­vant $AX­ON from Vivek Ra­maswamy nev­er made any sense in my opin­ion. If you reach in­to the fail­ure buck­et and pluck out an as­set for cheap, it didn’t make sense that in­vestors would buy in and sup­port it through a late-stage pro­gram. The risk/re­ward equa­tion was out of whack, and the whole thing reeked of an end-of-days play for the biotech bub­ble. Ra­maswamy, though, just pulled off an­oth­er record IPO af­ter whip­ping up a new com­pa­ny overnight, bring­ing in a cou­ple of high-pro­file play­ers to front the small team and tout­ing a trendy fo­cus on women’s health. The bub­ble, such as it was, is long popped. So I have to take my hat off to Ra­maswamy. He may know lit­tle about ac­tu­al drug de­vel­op­ment, but there’s no bet­ter fi­nan­cial en­gi­neer in biotech. Fi­nan­cial en­gi­neer­ing, though, goes just so far in biotech. In­evitably, da­ta will de­cide these com­pa­nies’ fate. And I’ll be wait­ing for that.

Shame isn’t di­lu­tive, when you’re Mar­tin Shkre­li

De­spite the fed­er­al fraud charges, de­spite his ex­po­sure for fi­nan­cial ru­in and charges of ly­ing to every­one around him, de­spite his tox­ic and well-earned rep­u­ta­tion as the most wide­ly hat­ed man on the plan­et, Mar­tin Shkre­li’s pub­lic per­sona re­mains un­changed. In his own mind, he’s still the smartest guy in the room, com­plete­ly un­re­pen­tant. Or so he presents him­self in the lat­est dis­as­trous at­tempt to im­press a jour­nal­ist, this time David Crow with the Fi­nan­cial Times. That de­ci­sion to go prof­i­teer­ing with an old drug? Sim­ple busi­ness acu­men, which he owed his in­vestors. The crim­i­nal case com­ing up next sum­mer? He’ll beat it, just like oth­er high-pro­file de­fen­dants like OJ. “My whole life has been one theme of self-sac­ri­fice for my in­vestors,” he replies, with­out bat­ting an eye­lid. “I did it for my share­hold­ers’ ben­e­fit be­cause that’s my job. The po­lit­i­cal risk is be­ing shamed — and shame isn’t di­lu­tive to earn­ings per share.” And so it goes.

Why the Sarep­ta con­tro­ver­sy won’t die

The Sarep­ta con­tro­ver­sy just won’t go away. Nor should it. This week we saw two promi­nent Har­vard pol­i­cy ex­perts weigh in with their own con­cerns about how the agency could have han­dled the Ex­ondys 51 sit­u­a­tion bet­ter. An ap­proval for what is clear­ly still an ex­per­i­men­tal ther­a­py rais­es all sorts of ques­tions over who should pay $300,000-plus a year for an un­proven drug des­per­ate­ly sought af­ter by pa­tients and their fam­i­lies.That part was un­der­scored by Hu­mana’s de­ci­sion to re­serve the drug for pa­tients who could stay am­bu­la­to­ry. Wheel­chair-bound pa­tients will be de­nied this drug. The Har­vard team sug­gest­ed an al­ter­na­tive path­way that would make drugs avail­able at cost, but the FDA’s Janet Wood­cock had cit­ed fi­nan­cial rea­sons for her un­prece­dent­ed in­sis­tence on an OK. It will take some time to clean up this mess. And the FDA would be ad­vised to do more than try to close the barn door af­ter the wild horse has es­caped.

As­traZeneca wait­ed way too long to ac­knowl­edge that dur­val­um­ab set­back

In phar­ma­land, big com­pa­nies rou­tine­ly pre­fer to keep bad news to a one-lin­er foot­note in a lengthy quar­ter­ly re­port — or a painful­ly brief state­ment. As­traZeneca failed to meet even this low stan­dard for trans­paren­cy on dur­val­um­ab. The com­pa­ny knew more than a month ago that it had a se­ri­ous safe­ty is­sue to ex­am­ine on its PD-L1 check­point pro­gram, which is es­sen­tial to its fu­ture. But it chose to keep an FDA par­tial hold qui­et un­til the safe­ty is­sue slow­ly leaked out on Twit­ter. To its cred­it, the com­pa­ny did im­me­di­ate­ly ex­plain what was hap­pen­ing rather than stonewall on a ma­te­r­i­al event.  But it was a day late and a dol­lar short on cor­po­rate re­spon­si­bil­i­ty, which played out with a painful drop in shares on Thurs­day. No­var­tis, Am­gen, Boehringer In­gel­heim and many oth­er big play­ers feel they have no debt to any­one to ex­plain set­backs. But in fact they do owe the pa­tients who vol­un­teer for their clin­i­cal tri­als, the in­vest­ment com­mu­ni­ty, and the pub­lic as a whole much bet­ter than what they have re­ceived. There should be no whin­ing from any­one in Big Phar­ma about their tat­tered pub­lic pro­file. These are all self-in­flict­ed wounds.

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Af­ter watch­ing its share price soar on a Bloomberg re­port and heat­ed ru­mors, Bio­haven stock takes a bil­lion-dol­lar bath

Back in April, Biohaven Pharmaceutical became one hot biotech stock $BHVN based on a report in Bloomberg that some “potential bidders” had been kicking the tires at the biotech, which has a lead drug for migraines. Then the rumor mill really started to smoke when execs canceled a presentation at an investor conference a little more than a week ago.

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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.