FDA mo­tions Al­ny­lam's lumasir­an in­to a PhI­II short track, set­ting up a shot at an OK in '20

Al­ny­lam $AL­NY may be go­ing so­lo on the de­vel­op­ment of rare dis­ease drug lumasir­an af­ter Sanofi took a pass on its op­tion, but the FDA has come through with a ma­jor as­sist. The com­pa­ny an­nounced to­day that reg­u­la­tors have agreed on a Phase III study de­sign that would ac­cel­er­ate — should all go well — its jour­ney to ap­proval.

For the tri­al de­signed to test lumasir­an in the ul­tra-rare pri­ma­ry hy­per­ox­aluria type 1 (PH1), the new­ly reached align­ment al­lows Al­ny­lam to adopt a pri­ma­ry end­point based on a bio­mark­er: the re­duc­tion of uri­nary ox­alate at six months. The FDA has al­so sanc­tioned a study size of around 25 pa­tients with PH1.

Pritesh Gand­hi

The use of a bio­mark­er spot­lights the FDA’s will­ing­ness to let drug­mak­ers shoot for faster OKs, rather than forc­ing them to wait for da­ta on dis­ease pathol­o­gy (Al­ny­lam notes the bio­mark­er is “di­rect­ly linked to the patho­phys­i­ol­o­gy of PH1 and known to be well cor­re­lat­ed with dis­ease pro­gres­sion”).

The tri­al de­sign pact with the agency al­so marks an ad­vance for the late-stage pipeline at Al­ny­lam as it waits for an ex­pect­ed ap­proval of patisir­an, which will launch their first com­mer­cial op­er­a­tions in RNAi. This biotech has been through all the ups and downs of­ten seen with any new ap­proach to drug de­vel­op­ment. And it’s poised for some ma­jor changes.

With the study de­sign ham­mered out, Al­ny­lam ex­pects to start the PhI­II study in mid-2018, re­port topline re­sults in 2019 and, if pos­i­tive, sub­mit an NDA in ear­ly 2020.

“We are very pleased with the FDA’s shared sense of ur­gency to eval­u­ate the ef­fi­ca­cy and safe­ty of lumasir­an as a po­ten­tial ther­a­peu­tic op­tion for pa­tients as rapid­ly as pos­si­ble,” said Pritesh Gand­hi, gen­er­al man­ag­er of the lumasir­an pro­gram, in a state­ment.

Al­ny­lam is cur­rent­ly con­duct­ing a Phase I/II Part B study in 20 pa­tients. Pre­lim­i­nary re­sults, an­nounced last year, sug­gest that lumasir­an led to a mean max­i­mal re­duc­tion in uri­nary ox­alate of 66% in an un­blind­ed group of four pa­tients, and all of them achieved uri­nary ox­alate lev­els at or near the nor­mal range. Lumasir­an, an RNAi ther­a­peu­tic, is de­signed to “re­duce the he­pat­ic lev­els of the [gly­co­late ox­i­dase] en­zyme, there­by de­plet­ing the sub­strate nec­es­sary for ox­alate pro­duc­tion.” It was grant­ed a break­through ther­a­py des­ig­na­tion in March.

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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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.

‘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.

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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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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ITeos nabs $125M as they prep Keytru­da com­bi­na­tion tri­al — if Covid-19 will let them

For iTeos, it turned out, $75 million could only last so long.

Two years after announcing their eye-catching Series B raise, the Belgian biotech is back with an even larger Series B-2: $125 million.

The now $175 million financing – $25 million of the first B round is considered part of the second – illustrates the vast capital available for those with promising new immuno-oncology compounds, particularly those that might be used in combination with existing therapies. In December, iTeos announced a collaboration with Merck to test its lead compound with Keytruda this year. The proceeds will push forward that trial and help fund the ongoing Phase I/II trials for that compound, EOS-850, and a second one, EOS-448.

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