Alex­ion preps an FDA pitch on Soliris suc­ces­sor while sig­nal­ing more deals ahead for rare dis­ease drugs

Alex­ion used its Q1 up­date Thurs­day to un­veil an­oth­er set of Phase III non-in­fe­ri­or stats for its Soliris suc­ces­sor, adding to its reg­u­la­to­ry pack­age for new ap­provals that should ship lat­er this year.

Fol­low­ing ear­li­er proof of non-in­fe­ri­or­i­ty for treat­ment-naive pa­tients, the new Phase III high­light­ed their suc­cess­ful switch­ing of Soliris pa­tients to ALXN1210, a shift from dos­ing every two weeks to eight weeks.

Lud­wig Hantson

To be sure, the in­ves­ti­ga­tors un­der­scored the same kind of im­prove­ments they found over Soliris on key mea­sures, but there was noth­ing sta­tis­ti­cal­ly sig­nif­i­cant about it. That will be good for shoring up their de­fense of the fran­chise drug for years to come — com­pa­ny ex­ecs talked of patents stretch­ing out to 2035 — as ri­vals press along with new drugs they hope can beat the stan­dard ther­a­py for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH).

Now the biotech is prep­ping reg­u­la­to­ry pitch­es that should ar­rive in a mat­ter of months.

Ear­li­er this month Alex­ion an­nounced a deal to buy a Stock­holm-based biotech for $855 mil­lion in cash, bag­ging a new drug for rare cas­es of Wil­son dis­ease. The com­pa­ny is us­ing that as a mod­el for more deals just like it.

Paul Clan­cy

CFO Paul Clan­cy not­ed:

I would char­ac­ter­ize the bias to­wards prod­ucts, not to­wards plat­forms. Nev­er say nev­er, but this is rare dis­ease. Wil­son rep­re­sents a good ex­am­ple of our bias. Rare dis­ease, dev­as­tat­ing dis­eases, a po­ten­tial prod­uct to trans­form the dis­ease, and we’re build­ing up our abil­i­ty to do that in­side the com­pa­ny.

CEO Lud­wig Hantson sought to re­as­sure an­a­lysts that they were sat­is­fied that they could con­vert a large num­ber of pa­tients to the new drug, which some an­a­lysts be­lieve will have to come with a dis­count for pay­ers. Dur­ing the call with an­a­lysts, the CEO said:

We have a dif­fer­en­ti­at­ed pro­file with 1210. I don’t think any­body will dis­pute that. And, for sure, we’re go­ing to try to get the best la­bel. And we be­lieve that the da­ta that we have will be re­flect­ed in the clin­i­cal tri­al sec­tion. The way I look at it, we don’t need the su­pe­ri­or­i­ty claim to be suc­cess­ful for a fast con­ver­sion. We have a strong, dif­fer­en­ti­at­ed clin­i­cal pro­file, a ro­bust da­ta pack­age.

It’s not ide­al, but an­a­lysts like Ge­of­frey Porges saw rea­sons for ap­plause.

Alex­ion not­ed that ‘1210 was nu­mer­i­cal­ly su­pe­ri­or on both the pri­ma­ry and all key sec­ondary end­points. We are al­so en­cour­aged by the dis­clo­sure that ze­ro sub­jects treat­ed with ‘1210 ex­pe­ri­enced break­through he­mol­y­sis ver­sus 5 sub­jects treat­ed with Soliris. In terms of safe­ty, there were no dis­con­tin­u­a­tions due to ad­verse events, neu­tral­iz­ing an­ti­bod­ies, or cas­es of meningo­coc­cal in­fec­tion.

Eli Lil­ly’s first PhI­II show­down for their $1.6B can­cer drug just flopped — what now?

When Eli Lilly plunked down $1.6 billion in cash to acquire Armo Biosciences a little more than a year ago, the stars seemed aligned in its favor. The jewel in the crown they were buying was pegilodecakin, which had cleared the proof-of-concept stage and was already in a Phase III trial for pancreatic cancer.

And that study just failed.

Lilly reported this morning that their cancer drug flopped on overall survival when added to FOLFOX (folinic acid, 5-FU, oxaliplatin), compared to FOLFOX alone among patients suffering from advanced pancreatic cancer.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

CSL ac­cus­es ri­val Pharm­ing of par­tic­i­pat­ing in a scheme to rip off IP on HAE while re­cruit­ing se­nior R&D staffer

Pharming has landed in the middle of a legal donnybrook after recruiting a senior executive from a rival R&D team at CSL. The Australian pharma giant slapped Pharming with a lawsuit alleging that the Dutch biotech’s new employee, Joseph Chiao, looted a large cache of proprietary documents as he hit the exit. And they want it all back.
Federal Judge Juan Sanchez in the Eastern District Pennsylvania court issued an injunction on Tuesday prohibiting Chiao from doing any work on HAE or primary immune deficiency in his new job and demanding that he return any material from CSL that he may have in his possession. And he wants Pharming to tell its employees not to ask for any information on the forbidden topics.
For its part, Pharming fired off an indignant response this morning denying any involvement in extracting any kind of IP from CSL, adding that it’s cooperating in the internal probe that CSL has underway.

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UP­DAT­ED: Alex­ion pays $930M to buy out Achillion and its promis­ing com­pan­ion drug to Soliris

After a series of stock-crunching setbacks over the years, Achillion enjoyed a turn in the sun a few weeks ago as the FDA blessed their lead drug danicopan (ACH-4471) — a complementary therapy for PNH patients taking Alexion’s Soliris — with a breakthrough drug designation after taking a look at some solid supporting Phase II data.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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From left to right: Lilian Kim, Associate Director Business Development; John Moller, CEO; Yooni Kim, Executive Director, Asia Operations; Michelle Park, Director South Korea Operations.

Novotech CRO sees 26% growth in Asia tri­al ac­tiv­i­ty from biotechs, but still plen­ty of ca­pac­i­ty

As the Asia-Pacific clinical trials sector continues to grow rapidly, Novotech the Asia-Pacific-based CRO is seeing biotech clinical activity up by 26%. But says there is still plenty of capacity in the region that features advanced medical facilities, supportive regulatory environments, and more than 2.3 billion people, largely treatment naïve, living in urban areas.

China, South Korea and Australia have the most studies registered as recruiting or about to recruit according to ClinicalTrials.Gov.

The $102B club: The top 15 R&D spenders in the glob­al bio­phar­ma busi­ness — 2019 edi­tion

Over the past few years, the deluge of capital into biotech has helped lead to a dramatic shift in focus on new drug approvals, as startups are now able to raise enough cash to get through a pivotal and onto the market. But the top 15 players still account for $102 billion in spending, and their successes and failures continue to determine just how productive the industry is.

Recently we’ve seen a number of new R&D chiefs take their places at the Big 15, either setting the stage for a more focused R&D strategy — often playing more heavily in oncology. That’s true for AstraZeneca, which has had some landmark successes, and GSK, which is in search of its own turnaround in pharma R&D. HIV and vaccines are separate from that group, now led by Hal Barron.

I’ve made a point of watching their track record every year for more than a decade now. What follows is intended as a broad gauge of their activity. You don’t have to have a lot of major successes to score a winning record here, but it’s virtually impossible without a blockbuster or three in the pipeline.

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Andrew Dickinson, Gilead

Gilead­'s chief strat­e­gy ex­ec gets a big pro­mo­tion af­ter or­ches­trat­ing multi­bil­lion-dol­lar deals

After gaining credit as the architect of Gilead’s $12 billion Kite buyout as well as the recent $5 billion partnership with Galapagos, chief strategy officer Andrew Dickinson is being promoted to the prestigious CFO post at the big biotech. And new CEO Daniel O’Day says the latest move completes his makeover of the top team.
Dickinson will remain in charge of strategy in his new post.
A 3-year veteran at Gilead, Dickinson joined the bellwether biotech after a lengthy stint at Lazard Frères & Co, where he was global co-head of healthcare investing. Before that, ironically enough, he had been at Myogen, which was bought out by Gilead in 2006. Now he’ll be primarily responsible for building confidence in the numbers at a company that has a strong foundation in HIV, a disappearing franchise in hep C and a CAR-T subsidiary in Kite that has a long way to go in establishing a new business.

That big neu­ro­sciences R&D group Eli Lil­ly built is be­ing dis­man­tled, with lay­offs and parts shipped home

Seven years after Eli Lilly bulked up its neurosciences research group in Surrey and heralded the move as an indication of its commitment to the field, the pharma giant is shutting down and locking up labs.

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