Alex­ion's suc­ces­sor to Soliris hits co-pri­ma­ry end­points in a piv­otal, but can't sur­pass the big block­buster

With a group of ri­vals press­ing in from every side, look­ing to cap­ture a piece of Alex­ion’s $ALXN block­buster mar­ket for the rare dis­ease trend­set­ter Soliris, re­searchers for the biotech say the first of two piv­otal stud­ies for PNH came through with non-in­fe­ri­or da­ta on their next-gen drug dubbed ALXN1210. That should leave the com­pa­ny on track for a bad­ly need­ed reg­u­la­to­ry fil­ing lat­er this year.

John Orloff

But, the parox­ys­mal noc­tur­nal he­mo­glo­bin­uria drug al­so failed a key mea­sure of su­pe­ri­or­i­ty, leav­ing Alex­ion tout­ing the sim­i­lar­i­ties of the two ther­a­pies and an eas­i­er dos­ing sched­ule that may leave quite a few sup­port­ers a wee bit dis­sat­is­fied.

The co-pri­ma­ry end­points in the study were trans­fu­sion avoid­ance and lac­tate de­hy­dro­ge­nase (LDH) nor­mal­iza­tion, stacked along­side four key sec­ondary end­points. The analy­sis on su­pe­ri­or­i­ty quick­ly broke down on the ex­am­i­na­tion of break­through he­mo­lo­y­sis: 4% of pa­tients giv­en and 10.7% for pa­tients treat­ed with Soliris. That de­liv­ered a failed p val­ue of 0.074, halt­ing the su­pe­ri­or­i­ty re­view and leav­ing some an­a­lysts a bit frus­trat­ed.

Un­der new man­age­ment af­ter CEO Lud­wig Hantson swept the old crew out, the biotech has con­cen­trat­ed heav­i­ly on ALXN1210 to save the com­pa­ny’s on­ly big fran­chise. At $542,640 a year, Soliris re­mains one of the 10 most ex­pen­sive ther­a­pies on the plan­et and Alex­ion’s cash cow.

The stock spiked on the news, pick­ing up a 9% gain mid-morn­ing as in­vestors ab­sorbed the da­ta.

Ge­of­frey Porges, Leerink

Leerink’s Ge­of­frey Porges led the cheer­ing sec­tion this morn­ing, but al­so not­ed that the tri­al fell well short of a com­plete suc­cess.

This con­sis­tent pic­ture of pos­i­tive trends in fa­vor of ALXN1210 should re­as­sure in­vestors that Alex­ion’s 2nd gen­er­a­tion prod­uct is in­deed bet­ter than Soliris; we ex­pect the stock to re­act pos­i­tive­ly to this dis­clo­sure, al­though con­tro­ver­sy will still ex­ist about whether 1210 could have shown su­pe­ri­or­i­ty in a larg­er tri­al, and whether the up­com­ing switch study could still meet the high­er hur­dle.

R&D chief John Orloff said:

Soliris has es­tab­lished a high bar for ef­fi­ca­cy. Achiev­ing non-in­fe­ri­or­i­ty on both co-pri­ma­ry and all key sec­ondary end­points, as well as see­ing nu­mer­ic re­sults in fa­vor of ALXN1210, in such a rig­or­ous study met a very high hur­dle. We look for­ward to reg­u­la­to­ry sub­mis­sions of ALXN1210 in PNH in the U.S., EU, and Japan in the sec­ond half of 2018.

Porges and al­lies be­lieve the da­ta sets up 1210 for a near cer­tain ap­proval, guard­ing the com­pa­ny’s fran­chise for many more years. But Alex­ion isn’t op­er­at­ing alone here any­more. Sev­er­al ri­vals are well along with new PNH drugs that they be­lieve can out­per­form Soliris, and now 1210.

One of those ri­vals is Apel­lis $APLS, which com­plet­ed a $150 mil­lion IPO last No­vem­ber. In 3 pa­tients nev­er treat­ed with Soliris, in­ves­ti­ga­tors re­port­ed that all of them ex­pe­ri­enced a quick cor­rec­tion on a key bio­mark­er for lac­tate de­hy­dro­ge­nase, or LDH, af­ter be­ing treat­ed with the biotech’s drug. In 6 pa­tients not re­spond­ing well to Soliris, the av­er­age he­mo­glo­bin lev­el was brought up an av­er­age of 36%, LDH was cor­rect­ed and trans­fu­sions dropped from 3.4/month on eculizum­ab monother­a­py to 0.3/month when APL-2 was added to eculizum­ab. And the biotech raised no un­usu­al red flags on the safe­ty side. Now it’s aim­ing at a piv­otal of its own.

The big ques­tion at Alex­ion re­mains open. What will Hantson do with the pipeline now? He has sought to stream­line de­vel­op­ment ef­forts and quite a few an­a­lysts are press­ing hard to make them di­ver­si­fy be­yond PNH.

UP­DAT­ED: A small, ob­scure biotech just won big with their IPO. In this mar­ket. Are you kid­ding me?

How could a small, largely unknown biotech that emerged from stealth mode just months ago with early-stage cancer programs jump onto Wall Street in the middle of a Category 6 financial hurricane and sail through with a $165 million IPO?

And what does that mean for the rest of the industry waiting to see just how much damage global lockdowns will wreak on clinical development?

The biotech is a company called Zentalis. The crew there nabbed an $85 million crossover round late last year — notably waiting 5 years before waving the numbers around to attract attention, according to my read of a FierceBiotech story. Perceptive joined in, but the syndicate was not in general the kind of marquee affair that gets tongues wagging.

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Gilead CEO Daniel O'Day attends a meeting with the President and other biopharma leaders at the White House on March 2, 2020 (AP Photo)

Ramp­ing up glob­al pro­duc­tion of remde­sivir, Gilead CEO Dan O’Day de­tails plans to dis­trib­ute 1.5M dos­es to fight Covid-19 — for free

Gilead is still some days away from turning the card on its first round of data on remdesivir’s ability to fight severe cases of Covid-19, but the big biotech is ramping up an emergency supply of a million courses of therapy as it starts free distribution of the drug to tens of thousands of patients under their compassionate use and expanded access program as well as clinical trials.

In his latest open letter posted over the weekend, Gilead CEO Dan O’Day outlined how the company has been successful in cutting production time on remdesivir while repurposing some of their own facilities and turning to contract manufacturers to build a near-term supply of 1.5 million doses. They are still working on efficacy and dosing, but that supply could cover 140,000 courses of treatment. That supply, he added, would be more widely available following a potential approval.

Bob Nelsen at the Milken Institute Global Conference on April 29, 2019 in Beverly Hills, California. (Photo by Michael Kovac/Getty Images)

ARCH chief Bob Nelsen has $1.5B to prove 2 sim­ple points: ‘We’re in the most in­no­v­a­tive time ever’ and in­vestors are stay­ing

ARCH co-founder and managing director Bob Nelsen has a well known yen for the home run swing, betting big on potentially transformative meds and tech and the biotech teams he helps bring together. He thrives and bleeds on the cutting edge. And now Nelsen and the ARCH group have debuted 2 big funds to prove that this is the time for the best of biotech to shine — deadly pandemic be damned.

Two new funds, ARCH Venture Fund X and ARCH Venture Fund X Overage, gathered a combined $1.46 billion. And that’s a record. ARCH Venture Fund IX and ARCH Venture Fund IX Overage closed in 2016 with a combined $1.1 billion. ARCH Venture Fund VIII and ARCH Venture Fund VIII Overage closed in 2014 with a combined $560 million.

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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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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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Aaron Royston, venBio

In­vest­ing in the time of coro­n­avirus: the good, the bad and the hope­ful, as biotech VC firms close funds worth $3B

Apart from disrupting biopharma R&D and regulatory timelines, the coronavirus pandemic has inevitably ravaged financial markets and eroded investor risk appetite. Investing in the time of coronavirus feels reckless, but if biotech venture funds are any indication, the time is ripe.

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Drug dis­cov­ery in the age of coro­n­avirus

Developing new drugs is incredibly hard. That’s why, despite superhuman efforts from the industry, we’re still looking at 12-18 months minimum before we can realistically hope for a vaccine for Covid-19, and probably months before there’s a proven viable drug treatment.

But our increasing ability to begin to industrialize the drug discovery and development process through an engineering approach means that we have more hope for speeding up this process than ever before — and not just to defeat coronavirus, but to benefit the development of all new medicines in the future.