Sage’s quick flip in­to PhI­II crash­es as lead drug fails bad­ly for rare type of seizures

Af­ter build­ing in­tense in­ter­est in its lead drug for su­per-re­frac­to­ry sta­tus epilep­ti­cus with da­ta from tiny stud­ies, Sage Ther­a­peu­tics $SAGE says the drug failed bad­ly in Phase III.

Com­par­ing brex­anolone (SAGE-547) with a place­bo, the drug act­ed just like a sug­ar pill, with a 43.9% re­sponse for the drug com­pared to 42.4% in the place­bo arm — and a mis­er­able p-val­ue of 0.8775.

Sage’s shares dropped more than 20% on the news. But by the af­ter­noon the biotech had trimmed its loss­es, with the stock down about 14% — a mod­est re­treat giv­en the bad judg­ment this biotech has shown.

Jeff Jonas, Sage

Sage cre­at­ed both fierce be­liev­ers as well as even fiercer crit­ics of its drug for rare cas­es of pro­tract­ed seizures as CEO Jeff Jonas con­fi­dent­ly pre­dict­ed suc­cess. More than a year ago Sahm Ad­ran­gi’s Ker­ris­dale Cap­i­tal ex­e­cut­ed a short at­tack on Sage, caus­ti­cal­ly sum­ma­riz­ing its move from a small Phase I/II to a piv­otal tri­al as a strat­e­gy doomed to fail­ure.

Ker­ris­dale’s re­port picked apart da­ta from a sin­gle-arm study with no com­par­i­son group of pa­tients, which they be­lieve set the stage for the biotech to claim a big win with­out ac­tu­al­ly putting the ther­a­py to the test. And they add that there are ex­ist­ing ther­a­pies that go af­ter the ex­act same tar­get with sim­i­lar out­comes.

The fail­ure here will al­so cast a cloud over Sage’s oth­er R&D work, in­clud­ing a new drug for post­par­tum de­pres­sion which was al­so tout­ed with small stud­ies il­lus­trat­ing pos­i­tive re­sults.

But not every­one thinks the ug­ly flop here reads through to the rest of the pipeline. Notes Paul Mat­teis at Leerink:

The in­vest­ment de­bate this morn­ing cen­ters around the de­gree to which SRSE reads neg­a­tive­ly on­to the clin­i­cal po­ten­tial of SAGE-547 in PPD (and by ex­ten­sion oth­er mood dis­or­ders); while we un­der­stand that the lack of sig­nal in SRSE has ren­dered some in­vestors more cau­tious in their in­ter­pre­ta­tion of SAGE‘s many small CNS da­ta sets, for us, it’s hard to see a tan­gi­ble readthrough on­to PPD, which has (1) place­bo-con­trolled da­ta, (2) bet­ter un­der­stood nat­ur­al his­to­ry/place­bo ef­fects, and (3) break­through ther­a­py des­ig­na­tion.

As for Jonas, he still found rea­sons to ap­plaud the work.

“I’m proud of the Sage team for the sig­nif­i­cant progress they have made in im­prov­ing our un­der­stand­ing of how to best treat these crit­i­cal­ly ill pa­tients,” he said. “SRSE is a com­pli­cat­ed con­di­tion that is poor­ly un­der­stood, and I want to thank the pa­tients, their fam­i­lies, and the in­ves­ti­ga­tors who par­tic­i­pat­ed in the STA­TUS Tri­al. Al­though we did not meet the pri­ma­ry end­point, this first-ever tri­al in a high­ly vari­able and com­plex pa­tient pop­u­la­tion con­firms that re­search in a crit­i­cal care unit is pos­si­ble and deep­ens our un­der­stand­ing of GA­BA mech­a­nisms and their ef­fect on brain cir­cuit­ry. As we con­tin­ue ex­am­in­ing da­ta from the STA­TUS Tri­al in the com­ing weeks, I’m hope­ful this in­for­ma­tion will in­form cur­rent treat­ments, and aid in the de­vel­op­ment of fu­ture treat­ments for pa­tients with SRSE.”

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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UP­DAT­ED: Have a new drug that promis­es to fight Covid-19? The FDA promis­es fast ac­tion but some de­vel­op­ers aren't hap­py

After providing an emergency approval to use malaria drugs against coronavirus with little actual evidence of their efficacy or safety in that setting, the FDA has already proven that it has set aside the gold standard when it comes to the pandemic. And now regulators have spelled out a new approach to speeding development that promises immediate responses in no uncertain terms — promising a program offering the ultimate high-speed pathway to Covid-19 drug approvals.

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Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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The race to de­vel­op Covid-19 drugs and vac­cines is on — here’s what’s hap­pen­ing in the UK

Weeks away from the results of ongoing US and China trials testing its experimental antiviral remdesivir, Gilead is going to trial the failed Ebola drug in a small group of coronavirus patients in England and Scotland. The United Kingdom is also home to a range of other therapeutic efforts, as the pandemic rages on across the globe.

On Tuesday, Southampton, UK-based startup Synairgen kicked off a mid-stage placebo-controlled study testing its experimental drug, SNG001 — an inhaled formulation of interferon-beta-1a — that has previously shown to be safe and effective in improving lung function in asthma patients with a respiratory viral infection in a pair of Phase II trials.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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‘There was a grow­ing weari­ness’: Rush­ing against a pan­dem­ic clock, As­pen Neu­ro­sciences se­cures $70M Se­ries A

Just before Christmastime, Howard Federoff got a tip from Washington: There was a new virus in China. And this one could be bad.

News report of the virus had not yet appeared. Federoff, a neuroscientist, was briefed because years before, he was vetted as part of a group — he didn’t give a name for the group — to consult for the US government on emerging scientific issues. His day job, though, was CEO of Aspen Neurosciences, a Parkinson’s cell therapy startup that days before had come out of stealth mode and gave word to investors they were hoping to raise $70 million. That, Federoff realized, would be difficult if a pandemic shut down the global economy.

FDA puts pe­di­atric aGVHD drug on pri­or­i­ty re­view lane — will they go vir­tu­al with the ad­comm?

Despite worries about regulatory delays due to new work arrangements under Covid-19, the FDA appears intent to go full speed ahead with its everyday work, not only granting priority review to a stem cell therapy for acute graft versus host disease but also plotting an advisory committee meeting for it.

With a PDUFA date of September 30, the journey of the drug — remestemcel-L, or Ryoncil — could shed light on the agency’s capacity to facilitate drug development unrelated to Covid-19.

Covid-19 roundup: Trump push­es his new fa­vorite, untest­ed drug; CRISPR out­lines crip­pling im­pact of Covid-19

President Trump has a new favorite Covid-19 drug.

After a conversation with Japanese Prime Minister Shinzo Abe, Politico reports, the president is pressuring the FDA to issue emergency use authorization for favipiravir, a flu drug that showed glimpses of success in China but remains unproven and carries a list of worrying side effects. The push comes after a week-plus in which the White House touted a potentially effective but unproven malaria medication despite the concerns of scientific advisors such as NIAID director Anthony Fauci. And Trump ally Rudy Giuliani has been talking up unproven cell therapy efforts on Twitter.

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