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.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Almirall is tapping artificial intelligence on behalf of its sales force for insights and efficiencies. (via Shutterstock)

Almi­rall rolls out sales rep ar­ti­fi­cial in­tel­li­gence sys­tem, cut­ting pre-call prep and 'wind­shield time'

Dermatology specialty pharma Almirall is making its sales reps smarter. Not with extra training or educational courses, but instead with artificial intelligence tools.

It began a soft launch of a sales rep AI and machine learning platform it calls Polaris last August in one of its 7 US coverage regions. The platform from Aktana gathers information from across Almirall internal sources and external ones – such as claims and prescribing data – to generate insights for reps. Now, instead of spending hours prepping for a sales call, Polaris can generate details about a physician’s preferences, past behaviors and prescription habits for reps in minutes, said Almirall head of commercial operations Vincent Cerio.

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Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Greg Mayes, Antios Therapeutics CEO

An­tios' HBV col­lab axed af­ter clin­i­cal hold, but biotech be­lieves safe­ty in­ci­dent is not treat­ment-re­lat­ed

The FDA has placed a clinical hold on a Phase IIa study of Antios Therapeutics’ investigational hepatitis B med, CEO Greg Mayes confirmed to Endpoints News in an emailed statement.

A safety report was delivered to the biotech on May 17 after a patient dosed in a triple combination cohort of the study had experienced bradycardia and hypotension. The triple combo included Antios’ ATI-2173, Assembly Biosciences’ vebicorvir and Viread, an approved antiviral for HIV and hepatitis B.

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Tim Schoen, BioMed Realty CEO

Life sci­ences de­vel­op­er Bio­Med Re­al­ty buys San Fran­cis­co ho­tel for $75M — re­port

In a somewhat unconventional deal, life sciences real estate developer BioMed Realty has bought a 169-room Hilton Garden Inn in South San Francisco for $75 million, the San Francisco Business Times reported.

BioMed Realty, an affiliate of Blackstone, has multiple life sciences and technology office projects in the Bay Area, including three sites within a five-minute drive of the hotel.

While the sale of the hotel property was announced earlier this month, the sellers, Summit and GIC, did not identify the buyer at the time.

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Pfizer CEO Albert Bourla at the World Economic Forum (Gian Ehrenzeller/Keystone via AP Images)

All about ac­cess: Pfiz­er moves to a non-prof­it mod­el for drug sales in 45 low­er-in­come coun­tries

Leading the way to increase access to cheaper drugs worldwide, Pfizer said Wednesday it will provide all current and future patent-protected medicines and vaccines available in the US or EU on a not-for-profit basis to about 1.2 billion people in 45 lower-income countries.

Rwanda, Ghana, Malawi, Senegal and Uganda are the first five countries to sign on to this accord, which will also seek to blaze new paths for quick and efficient regulatory and procurement processes to reduce the usual delays in making new medicines and vaccines available in these countries.