New PhI­II gives Es­pe­ri­on's CEO a chance to push back against a le­gion of crit­ics — but can he win back in­vestors?

Es­pe­ri­on $ES­PR would like to have back its rep for own­ing a cho­les­terol drug de­signed to hit a sweet spot in the mar­ket — with­out any un­sa­vory safe­ty is­sues that could per­suade reg­u­la­tors to re­quire an ex­pen­sive and lengthy car­dio out­comes study ahead of a mar­ket­ing ap­proval.

To that end, CEO Tim Mayleben is rolling out a fresh bat­tery of da­ta from their third piv­otal study of be­mpe­doic acid. And they want you to be­lieve as they do: That the con­sis­tent abil­i­ty to cut LDL — in this case by demon­strat­ing a fa­mil­iar 23% drop, with a 25% low­er­ing in high-sen­si­tiv­i­ty C-re­ac­tive pro­tein — tied in to no new fa­tal­i­ties (whew) and what they in­sist is a clean safe­ty pro­file should be wide­ly ap­plaud­ed.

Whether they can achieve their goal in a mar­ket that has sharp­ened its knives for biotech stocks and kept on the look­out for any weak­ness­es, though, is still open to ques­tion. But they’re go­ing to try.

“We’re try­ing to pro­vide more con­text than we did ear­li­er this month,” CEO Tim Mayleben told me in a pre­view of to­day’s num­bers. Mayleben has seen his stock price gy­rate be­fore on the way an in­di­vid­ual da­ta point is in­ter­pret­ed, but now he has re­port­ed da­ta on 4,000 pa­tients all to­geth­er — 1,500 who have been on the drug for more than a year.

“That’s what reg­u­la­tors will look at, the pooled da­ta,” says the CEO. “When you look at it over­all, you see a very bal­anced safe­ty and tol­er­a­bil­i­ty pro­file.” And he still isn’t wor­ried about the prospect of head-to-head com­pe­ti­tion with the PC­SK9s. 

Part of that pooled da­ta that will get close scruti­ny to­day is a fa­tal­i­ty rate of 0.5 per­cent in the drug arm and 0.2 per­cent for the place­bo group.

“I would say that with re­spect to pric­ing, one of the things we have seen con­stant­ly is con­fu­sion, mis­un­der­stand­ings about the net price,” the CEO adds. Once you fac­tor in the usu­al dis­counts ex­pect­ed in this mar­ket, he says, you can ex­pect a net price of less than half the $5,000 (or so) that the PC­SK9s will go for. And for mil­lions of pa­tients who are not con­trolled on statins and don’t need a dra­mat­ic cut in LDL, he be­lieves he’ll have a clear­ly de­fined mar­ket of­fer­ing a big ad­van­tage to pay­ers.

Ul­rich Laufs

The team at Es­pe­ri­on has good rea­son to push back af­ter their stock was pum­meled ear­li­er this month in the af­ter­math of its last Phase III read­out, when an­a­lysts and in­vestors re­coiled from an im­bal­ance of deaths in the study and asked some sharply point­ed ques­tions about the fu­ture of a drug that has a pro­ject­ed price just un­der what Re­gen­eron and Sanofi re­cent­ly set as their low mark for an ap­proved PC­SK9 ri­val that every­one agrees is much, much bet­ter at low­er­ing LDL, with ri­vals on the mar­ket and in the pipeline that could do just as well — or bet­ter.

That prospect of low­er­ing prices on the PC­SK9 class could crush a play­er like Es­pe­ri­on, a sen­ti­ment that helped trig­ger a 30% land­slide in the share price.

De­spite the ab­sence of deaths, the be­mpe­doic acid arm did see a 6% rate of se­ri­ous ad­verse events, com­pared to 3.6% for the place­bo group. Dis­con­tin­u­a­tions were 18.4% for the drug arm ver­sus 11.7% for the place­bo — but the study in­ves­ti­ga­tor ruled out any con­nec­tion be­tween the drug and the SAEs.

Ul­rich Laufs, a mem­ber of Es­pe­ri­on’s Phase III ex­ec­u­tive com­mit­tee and di­rec­tor of the de­part­ment of car­di­ol­o­gy at Leipzig Uni­ver­si­ty, called the da­ta “amaz­ing­ly con­sis­tent” and added that “the med­ical com­mu­ni­ty is in need of a new oral ther­a­py which is ef­fec­tive, well tol­er­at­ed and con­ve­nient for this com­plex pa­tient pop­u­la­tion who may have run out of oth­er op­tions.”  

Es­pe­ri­on has two more Phase III read­outs, this sum­mer and then in Sep­tem­ber. No lat­er than Q1, says Mayleben, they’ll have the NDA com­plete. And then reg­u­la­tors can have the last say on ef­fi­ca­cy and safe­ty.

In the mean­time, the in­vestors are still see­ing more red than green in the up­dates from Es­pe­ri­on. The stock was down 3% by mid-day Wednes­day.

Im­age: Tim Mayleben

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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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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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.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.

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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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.