Ver­tex's rapid-fire PhI­II pro­gram hits a road­block at the FDA. Will reg­u­la­tors force ri­val Gala­pa­gos to slow down too?

Ver­tex’s R&D team doesn’t make many mis­takes. So dur­ing yes­ter­day’s Q1 call, a group of an­a­lysts were quick to pick up on a rare stum­ble that just might make it more vul­ner­a­ble to a ri­val like Gala­pa­gos.

Ge­of­frey Porges, Leerink

While not­ing plans for launch­ing a new Phase III ef­fort with a com­bi­na­tion drug us­ing VX-445 with Symdeko, fol­low­ing up as planned, in­ves­ti­ga­tors $VRTX al­so con­ced­ed that the FDA sees its deuter­at­ed ver­sion of Ka­ly­de­co — VX-561 — as a nov­el agent that needs some ad­di­tion­al dose-rang­ing tri­al work be­fore reg­u­la­tors will al­low a move in­to Phase III.

Ver­tex bought ‘561 from Con­cert a lit­tle more than a year ago for $160 mil­lion in cash and an­oth­er $90 mil­lion in mile­stones, tak­ing over the Phase II tri­al.

Ge­of­frey Porges at Leerink sees a de­lay on VX-561 of at least a year, forc­ing the shift of fo­cus to the VX-445 com­bo now as a kind of Plan B.

This means that the com­pa­ny is now run­ning two rough­ly equiv­a­lent hors­es in the same ex­pen­sive race, and while the risk mit­i­ga­tion from this strat­e­gy is laud­able, it does seem more ex­pen­sive, in terms of tri­al costs, time and rev­enue fore­gone, than is nec­es­sary. Ver­tex may still de­vel­op a Vx561 con­tain­ing once-dai­ly triple com­bi­na­tion, but such a prod­uct might not come to mar­ket now un­til 2022 or 2023. The FDA’s stance does pro­vide some re­as­sur­ance about Ver­tex’s com­pet­i­tive po­si­tion – the FDA is clear­ly sig­nal­ing that there are no short-cuts (at least in the US) to tra­di­tion­al good drug de­vel­op­ment prac­tices in this in­di­ca­tion.

That “com­pet­i­tive po­si­tion” he’s re­fer­ring to deals with Gala­pa­gos $GLPG, which launched an ex­plorato­ry study called FAL­CON for its triple ear­li­er this week. And what the FDA finds good for Ver­tex, they’ll like­ly find good for Gala­pa­gos, where the dose-rang­ing stan­dards may al­so like­ly to slow things down. Notes Michael Yee from Jef­feries:

Thus since GLPG has all new NCE’s they will need to do lots of dose-find­ing work re­quir­ing lots more Phase IIs than the “quick” stud­ies they’re do­ing now…so while GLPG might have “da­ta” for FAL­CON study H2-18 and more da­ta in 2019 — these are sim­ple stud­ies and will need thor­ough work per FDA read-through.

Bri­an Sko­r­ney

Baird’s Bri­an Sko­r­ney al­so took a shot at Gala­pa­gos.

(W)e be­lieve it shows clear reg­u­la­to­ry prece­dent for po­ten­tial com­peti­tors who think they can move a com­bi­na­tion of three new chem­i­cal en­ti­ties in­to late stage de­vel­op­ment with­out demon­strat­ing the clin­i­cal at­trib­ut­es and li­a­bil­i­ties of each com­po­nent (we’re look­ing at you Gala­pa­gos).

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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Swamy Vijayan. Plexium

San Diego up­start de­buts dis­cov­ery en­gine that puts a twist to pro­tein degra­da­tion

For years, the idea of protein degradation — utilizing the cell’s natural garbage disposal system to mark problematic proteins for destruction — remained an elegant but technically difficult concept. But now established as a promising clinical strategy, with major biopharma players such as Bayer, Gilead and Vertex trying to grab a foothold via partnership deals, a San Diego startup is looking to exploit it and push its limits.

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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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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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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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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