Aer­pio shares tum­ble as lead eye drug trips up in mid-stage di­a­bet­ic retinopa­thy study

Rough­ly two years af­ter rais­ing $40 mil­lion in a pri­vate place­ment, Aer­pio Phar­ma­ceu­ti­cals’ lead ex­per­i­men­tal eye drug has hit a road­block af­ter fail­ing a mid-stage study in pa­tients with non-pro­lif­er­a­tive di­a­bet­ic retinopa­thy (NPDR) — the ear­li­est stage of di­a­bet­ic eye dis­ease, which oc­curs when high blood sug­ar lev­els cause dam­age to blood ves­sels in the reti­na.

Stephen Hoff­man

These blood ves­sels can swell and leak, or close re­strict­ing blood flow and even­tu­al­ly cul­mi­nate in blind­ness. In the 167-pa­tient tri­al, called TIME-2b, pa­tients were giv­en the drug — AKB-9778 —  (once or twice dai­ly) or place­bo (once or twice dai­ly) for 48 weeks. The main goal was to im­prove the the study eye di­a­bet­ic retinopa­thy sever­i­ty score (DRSS) by two or more steps com­pared to the place­bo.

Ad­min­is­tra­tion of AKB-9778 twice dai­ly missed the study’s pri­ma­ry end­point, the com­pa­ny said on Mon­day. Shares of the Cincin­nati-based drug de­vel­op­er $AR­PO cratered more than 68% to $1.36 in pre-mar­ket trad­ing.

About 9.6% of pa­tients giv­en AKB-9779 twice dai­ly ex­pe­ri­enced such an im­prove­ment, ver­sus 3.8% on the place­bo — draw­ing a less than rosy p val­ue (p=0.270). The rates of pro­gres­sion to sight-threat­en­ing com­pli­ca­tions, in­clud­ing di­a­bet­ic mac­u­lar ede­ma (DME) and/or pro­lif­er­a­tive di­a­bet­ic retinopa­thy (PDR), dur­ing the treat­ment pe­ri­od were sim­i­lar be­tween treat­ment groups, Aer­pio not­ed, adding that the use of the ex­per­i­men­tal drug had an “en­cour­ag­ing” im­pact on key sec­ondary goals, in­clud­ing a mea­sure of kid­ney func­tion and in­traoc­u­lar pres­sure.

Un­like pa­tients with di­a­bet­ic eye dis­ease who are typ­i­cal­ly giv­en an­ti-VEGF in­jec­tions in­to the eye, AKB-9778 is self-ad­min­is­tered sub­cu­ta­neous­ly by the pa­tient, akin to in­sulin. The ex­per­i­men­tal drug is a small mol­e­cule in­hibitor of VE-PTP, the most crit­i­cal neg­a­tive reg­u­la­tor of Tie2 — a path­way be­lieved to sta­bi­lize vas­cu­la­ture — in dis­eased blood ves­sels. In a sep­a­rate study, called TIME-2, AKB-9778 im­proved un­der­ly­ing symp­toms by two or more steps on a di­a­bet­ic retinopa­thy sever­i­ty scale in both eyes.

Un­de­terred by the TIME-2b fail­ure, Aer­pio chief Stephen Hoff­man ex­pressed en­thu­si­asm for the to­tal­i­ty of da­ta on the drug. “(C)ol­lec­tive­ly these da­ta sup­port a po­ten­tial­ly im­por­tant role of the Tie2 path­way for the treat­ment of di­a­bet­ic com­pli­ca­tions, as well as for open an­gle glau­co­ma,” he said in a state­ment, adding that a Phase Ib study of a top­i­cal drop for­mu­la­tion of AKB-9778 is ex­pect­ed to com­mence in the sec­ond quar­ter.

Aer­pio al­so has an­oth­er ex­per­i­men­tal eye drug in de­vel­op­ment, in ad­di­tion to an in­ves­ti­ga­tion­al ther­a­py for in­flam­ma­to­ry bow­el dis­ease.

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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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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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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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 $200 million financing 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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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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Daniel O'Day (AP Images)

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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