New epilep­sy com­pa­ny on the block wins FDA ap­proval for made-in-Ko­rea drug to treat fo­cal seizures

The FDA has ush­ered the fifth brand­ed drug for par­tial-on­set seizures to the mar­ket — in what its mak­er calls a first for the Ko­re­an bio­phar­ma in­dus­try.

Marc Kamin

Al­so known as fo­cal seizures, par­tial-on­set seizures are de­fined by ab­nor­mal elec­tri­cal ac­tiv­i­ty in one part of the brain (ver­sus the whole brain as in gen­er­al­ized seizures). While the 3 mil­lion epilep­sy pa­tients have had a swath of both pre­scrip­tion and gener­ic drugs to choose from, as much as 60% con­tin­ue to re­port hav­ing seizures. The new pill, Xco­pri, promis­es to cut the num­ber of episodes down to ze­ro for some.

“The find­ing from our clin­i­cal tri­als that up­wards of 1 in 5 pa­tients stopped hav­ing seizures, had 100% re­duc­tion in seizures, is what we think is unique about this drug,” Marc Kamin, CMO of SK Life Sci­ence, told End­points News ahead of the ap­proval.

SK Life Sci­ence is the US sub­sidiary of SK Bio­phar­ma­ceu­ti­cals, which is, in turn, the drug dis­cov­ery and de­vel­op­ment arm of a South Ko­re­an con­glom­er­ate that ranks 73 on For­tune’s Glob­al 500. Hav­ing start­ed out as a tex­tile com­pa­ny, SK Hold­ings now de­fines it­self as a “mak­er of greater hap­pi­ness.”

Their ef­forts with Xco­pri, then cenoba­mate, be­gan in earnest around 10 years ago. Af­ter re­view­ing phar­ma­co­ki­net­ic da­ta on the in-house project, they de­cid­ed to go it alone and pro­ceed in­to two Phase II stud­ies that ul­ti­mate­ly formed the ba­sis of the US ap­proval.

Seb­by Bor­riel­lo

Be­tween the two stud­ies, 655 adults who had par­tial-on­set seizures with or with­out sec­ondary gen­er­al­iza­tion were giv­en dif­fer­ent dos­es of the drug or place­bo. These were pa­tients who had ex­pe­ri­enced seizures for an av­er­age of 24 years and whose me­di­an seizure fre­quen­cy was 8.5 every 28 days.

In one of the stud­ies that’s since been pub­lished, me­di­an per­cent­age changes in seizure fre­quen­cy were -24·0% for the place­bo group com­pared with -35·5% for the 100 mg dose group (p=0·0071), -55·0% for 200 mg (p<0·0001), and -55·0% for 400 mg (p<0·0001), re­spec­tive­ly. SK Life Sci­ence said the oth­er tri­al showed a sta­tis­ti­cal­ly sig­nif­i­cant 56% re­duc­tion in me­di­an seizure fre­quen­cy with the 113 pa­tients on 200 mg, ver­sus a 22% re­duc­tion with place­bo (n=108).

FDA reg­u­la­tors end­ed up rec­om­mend­ing the 200 mg dose for stan­dard main­te­nance but al­lowed that some may need 400 mg dai­ly.

As far as they can de­ci­pher, Kamin said, the drug has a dual mech­a­nism of ac­tion: It blocks a sodi­um chan­nel while revving up GA­BA re­cep­tors.

SK Life Sci­ence’s com­mer­cial team has had close to three years to in­tro­duce them­selves to pa­tient ad­vo­ca­cy groups, physi­cians and pay­ers, chief com­mer­cial of­fi­cer Seb­by Bor­riel­lo said.

“In the most re­cent years a lot of prod­ucts have come out for these or­phan in­di­ca­tions like Lennox Gas­taut, which has 50 to 60,000 pa­tients in the Unit­ed States,” he not­ed, but there is still room for new op­tions on a much broad­er scale.

Xco­pri will be priced “com­pet­i­tive­ly” with oth­er brand­ed an­ti-epilep­tic drugs, he added, such as UCB Phar­ma’s Briv­i­act and Ei­sai’s Fy­compa. They tend to be used af­ter pa­tients have tried gener­ics.

The par­ent com­pa­ny is ful­ly on board and sup­port­ive for a launch in the sec­ond quar­ter of 2020 pend­ing DEA sched­ul­ing, ac­cord­ing to Bor­riel­lo. The cur­rent plan is to even­tu­al­ly mar­ket the drug in Ko­rea, Japan and Chi­na as well, while part­ner (and Ax­o­vant spin­out) Arvelle Ther­a­peu­tics han­dles Eu­rope.

“It’s al­ways good to add the mus­cle and the com­mit­ment be­hind our ef­forts from a very large or­ga­ni­za­tion,” he said.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,700+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,700+ biopharma pros reading Endpoints daily — and it's free.

Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,700+ biopharma pros reading Endpoints daily — and it's free.

Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.