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.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Gene Wang, Immetas co-founder and CEO (file photo)

Im­metas Ther­a­peu­tics nabs $11M Se­ries A to nar­row their bis­pe­cif­ic work tar­get­ing in­flam­ma­tion in age-re­lat­ed dis­eases

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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