GSK’s flop Poti­ga is picked up and head­ed for a quick makeover for pe­di­atric epilep­sy. Any bets on the prospec­tive price?

Glax­o­SmithK­line took a con­sid­er­able amount of heat last year when the phar­ma gi­ant de­cid­ed to yank a drug called Poti­ga, which had been ap­proved as an an­ti-con­vul­sant for adults but of­ten sold off la­bel to treat an ex­treme­ly rare form of epilep­sy caused by an er­rant KC­NQ2 gene. The drug was con­sid­ered a life­saver by the small group of kids and their fam­i­lies that ben­e­fit­ed from it, but GSK didn’t see enough of a com­mer­cial re­turn to make it worth­while. So they dis­card­ed it, along with oth­er parts of the com­pa­ny that have been stripped away in pur­suit of mak­ing the com­pa­ny more prof­itable in the long run.

“If there was a non­prof­it phar­ma­ceu­ti­cal man­u­fac­tur­er that want­ed to jump in, this is the sort of place that could be a good pos­si­bil­i­ty,” Aaron Kessel­heim, an as­so­ciate pro­fes­sor of med­i­cine at Har­vard Med­ical School, told the Chica­go Tri­bune.

Now, though, a Cana­di­an biotech called Xenon Phar­ma­ceu­ti­cals $XENE wants to see if this old drug has some com­mer­cial po­ten­tial that GSK was blind to. And there’s no sign that they see this as a char­i­ta­ble ven­ture.

The drug was on­ly ap­proved for adults, nev­er among chil­dren, though there was a big de­mand for it from the small num­ber of chil­dren who re­lied on it. Ac­cord­ing to the Chica­go Tri­bune piece, re­searchers nev­er ex­plored the pe­di­atric use be­cause Poti­ga earned a black box warn­ing for some dan­ger­ous side ef­fects, such as blue tinged skin and vi­sion prob­lems, which the fam­i­lies and kids who re­lied on it ev­i­dent­ly learned to live with.

Si­mon Pim­stone

Xenon, though, says it has a green light from the FDA to run the ac­tive in­gre­di­ent in Poti­ga — ezo­gabine, now dubbed XEN496 — through a tiny, piv­otal 20-pa­tient study to prove what GSK proved in adults and what the fam­i­lies have been say­ing for years: The drug con­trols seizures among chil­dren with the ge­net­ic trig­ger. And they are work­ing on a spe­cif­ic pe­di­atric for­mu­la­tion which they say “may” ad­dress the safe­ty is­sues.

“We have done an im­mense amount of dili­gence lead­ing up to the ad­di­tion of XEN496 to our nov­el and ro­bust pipeline of ion-chan­nel, an­ti-epilep­tic drugs,” says Si­mon Pim­stone, the CEO at Xenon. “Based on feed­back from key opin­ion lead­ers, ad­vo­ca­cy groups, pre-ex­ist­ing lit­er­a­ture, and promis­ing da­ta gen­er­at­ed to date, we be­lieve there is tremen­dous sup­port for us to vig­or­ous­ly pur­sue the de­vel­op­ment and com­mer­cial­iza­tion of XEN496 in or­der to reach the pe­di­atric KC­NQ2-EE pa­tient pop­u­la­tion as rapid­ly as pos­si­ble.”

GSK, for its part, said they lost ex­clu­siv­i­ty on the drug in the US mar­ket in 2016. And they’re giv­ing Xenon ac­cess to its drug da­ta — to be used in their ap­pli­ca­tion — for free. The drug can be or­dered from a Cana­di­an phar­ma­cy at a cost of $189 for a bot­tle of 84 50 mg pills; that’s $2.25 per pill.

Typ­i­cal­ly, if an ap­proval is in the works for the US this would be fol­lowed by a high, 6-fig­ure com­mer­cial price fol­low­ing a small, once-off study. I’ve asked the com­pa­ny for some guid­ance on their ex­pec­ta­tions, but haven’t heard back.

Re­pur­pos­ing old drugs and ap­ply­ing sky-high prices on them has be­come a reg­u­lar, though of­ten con­tro­ver­sial, strat­e­gy in biotech. Marathon stud­ied an old steroid drug nev­er ap­proved in the US specif­i­cal­ly for Duchenne mus­cu­lar dy­s­tro­phy, and then stirred up a hor­nets nest of trou­ble as law­mak­ers caught wind of their price. That drug wound up at PTC. And of course Mar­tin Shkre­li made a sim­i­lar ap­proach glob­al­ly un­pop­u­lar as he took con­trol of an old gener­ic and sim­ply hiked the price 5000%.

One of the ironies here is that the drug may not be as ef­fec­tive as a ke­to­genic di­et, some­thing that Johns Hop­kins has been study­ing for decades as a first ap­proach to child­hood epilep­sy. There have al­so been stud­ies that in­di­cate that a ke­to di­et — low carbs, mod­er­ate pro­tein and high fat — al­so works in this par­tic­u­lar ge­net­ic sub­set, among oth­ers.

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.

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:

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Dave Barrett, Brian Chee, Amir Nashat, Amy Schulman. Polaris

Bob Langer's first port of call — Po­laris Part­ners — maps $400M for ninth fund

Health and tech ven­ture group Po­laris Part­ners, which counts Alec­tor, Al­ny­lam and Ed­i­tas Med­i­cine as part of its port­fo­lio, is set­ting up its ninth fund, rough­ly two years af­ter it closed Po­laris VI­II with $435 mil­lion in the bank, sur­pass­ing its tar­get by $35 mil­lion.

The Boston-based firm, in an SEC fil­ing, said it in­tends to raise $400 mil­lion for the fund. Po­laris — which rou­tine­ly backs com­pa­nies mold­ed out of the work done in the lab of pro­lif­ic sci­en­tist Bob Langer of MIT  — typ­i­cal­ly in­vests ear­ly, and sticks around till com­pa­nies are in the green. Like its peers at Flag­ship and Third Rock, Po­laris is all about cham­pi­oning the lo­cal biotech scene with a steady flow of start­up cash.

Partners Innovation Fund

David de Graaf now has his $28.5M launch round in place, build­ing a coen­zyme A plat­form in his lat­est start­up

Long­time biotech ex­ec David de Graaf has the cash he needs to set up the pre­clin­i­cal foun­da­tion for his coen­zyme A me­tab­o­lism com­pa­ny Comet. A few high-pro­file in­vestors joined the ven­ture syn­di­cate to sup­ply Comet with $28.5 mil­lion in launch mon­ey — enough to get it two years in­to the plat­form-build­ing game, with­in knock­ing dis­tance of the clin­ic.

Canaan jumped in along­side ex­ist­ing in­vestor Sofinno­va Part­ners to co-lead the round, with par­tic­i­pa­tion by ex­ist­ing in­vestor INKEF Cap­i­tal and new in­vestor BioIn­no­va­tion Cap­i­tal.

Step­ping on Roche's toes, Mer­ck cuts in­to SCLC niche with third-line Keytru­da OK

In the in­creas­ing­ly crowd­ed check­point race, small cell lung can­cer has been a rare area where Roche, a sec­ond run­ner-up, has a lead over the en­trenched lead­ers Mer­ck and Bris­tol-My­ers Squibb. But Mer­ck is fi­nal­ly mak­ing some head­way in that di­rec­tion with the lat­est ap­proval for its PD-1 star.

The lat­est green light en­dors­es Keytru­da in the third-line treat­ment of metasta­t­ic SCLC, where it would be giv­en to pa­tients whose dis­ease ei­ther don’t re­spond to or re­lapse af­ter chemother­a­py, which would have fol­lowed at least one pri­or line of ther­a­py.

Right back at you, Pfiz­er: BeiGene and a Pfiz­er spin­out launch a new­co to de­vel­op a MEK/BRAF in­hibitor that could ri­val $11.4B com­bo

A day af­ter Pfiz­er bought Ar­ray and its ap­proved can­cer com­bo, BeiGene and Pfiz­er spin­out Spring­Works have part­nered in launch­ing a new biotech that has an eye on the very same mar­ket the phar­ma gi­ant just paid bil­lions for. And they’re plan­ning on us­ing an ex-Pfiz­er drug to do it.

In a nut­shell, Chi­na’s BeiGene is toss­ing in a pre­clin­i­cal BRAF in­hibitor — BGB-3245, which cov­ers both V600 and non-V600 BRAF mu­ta­tions — for a big stake in a new, joint­ly con­trolled biotech called Map­Kure with Bain-backed Spring­Works.