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.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology

ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development

CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.

An MIT spin­out kills one of its ‘liv­ing ther­a­peu­tics’ af­ter flunk­ing an ear­ly-stage study — shares rout­ed

Just a few weeks after bagging $80 million in a deal to collaborate with Gingko Bioworks on its special blend of engineered bacteria used for “living therapeutics,” little Synlogic in Boston $SYBX is tossing one of its two clinical programs after watching an early-stage study go down in defeat.

Their Phase Ib/IIa study for SYNB1020 to counter the accumulation of ammonia in the body, a condition called hyperammonemia or urea cycle disorder, floundered at the interim readout, forcing the biotech to kill it and reserve its cash for pipeline therapies with greater potential.

Elan­co to buy Bay­er's an­i­mal health busi­ness for $7.6B, as deal­mak­ing gath­ers steam in the sec­tor

Last week, Elanco explicitly dodged answering questions about its rumored interest in Bayer’s animal health business in its post-earnings call. On Tuesday, the Eli Lilly spinoff disclosed it was purchasing the German drug maker’s veterinary unit in a cash-and-stock deal worth $7.6 billion. 

Elanco $ELAN has been busy on the deal-making front. In April, it laid out plans to swallow its partner, Kansas-based pet therapeutics company Aratana $PETX. A July report by Reuters suggested a potential Bayer deal was being explored, and Bloomberg last week said the deal was imminent, citing sources. 

As­traZeneca's di­a­betes drug Farx­i­ga helps pa­tients with heart dis­ease and with­out di­a­betes in land­mark tri­al

Months ago, data on J&J’s $JNJ Invokana indicated the diabetes drug conferred cardiovascular (CV) benefit in patients who do and do not have preexisting CV disease. On Tuesday, AstraZeneca’s $AZN rival treatment, Farxiga, was shown to cut the risk of CV death or the worsening of heart failure in patients with heart disease, in a landmark trial.

The treatments, in addition to Jardiance from Eli Lilly $LLY, belong to a class of diabetes drugs called sodium-glucose co-transporter 2 (SGLT2) inhibitors, which work by curbing the absorption of glucose via the kidneys so that surplus glucose is excreted through urination.

Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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