Should Sarep­ta’s patents be seized by the gov­ern­ment? Pa­tient ad­vo­cates pitch con­tro­ver­sial drug pric­ing pro­pos­al

Six ad­vo­ca­cy groups are send­ing a big ask to the fed­er­al gov­ern­ment to low­er the price of one rather ex­pen­sive drug for Duchenne mus­cu­lar dy­s­tro­phy, pe­ti­tion­ing health reg­u­la­tors to flex pow­er it’s nev­er ex­er­cised be­fore.

Amidst a years-long de­bate over drug pric­ing, Sarep­ta has hit a sen­si­tive chord with the high price tag of its DMD drug Ex­ondys 51 (eteplirsen), which goes for a around $300,000 per year.

The or­ga­ni­za­tions draft­ed a let­ter to the De­part­ment of Health and Hu­man Ser­vices (HHS), plead­ing that Sec­re­tary Alex Azar act to low­er the price of the drug. Specif­i­cal­ly, the group wants HHS to lever­age a piece of leg­is­la­tion called the Bayh-Dole Act — along with con­trac­tu­al agree­ments with fund­ing agen­cies — to take over own­er­ship of five patents on Ex­ondys 51. They can do that, the group in­sists, be­cause the in­tel­lec­tu­al prop­er­ty was backed by fed­er­al re­search dol­lars. Grant re­cip­i­ents are re­quired to dis­close fed­er­al fund­ing that con­tributes to a patent­ed in­ven­tion on their patent ap­pli­ca­tion — a step that both Sarep­ta and the Uni­ver­si­ty of West­ern Aus­tralia failed to do.

By tak­ing ti­tle to the patents, the HHS could lever­age their po­si­tion to low­er the price of Ex­ondys, the or­ga­ni­za­tions said.

An­a­lysts at Leerink, who cov­er Sarep­ta’s stock $SRPT, sent a note to in­vestors this morn­ing not­ing the un­like­li­hood that such ac­tion would be tak­en by the gov­ern­ment.

“Bot­tom Line: To­day’s let­ter from sev­er­al groups de­liv­ered to HHS Sec­re­tary (Alex) Azar high­lights the lengths that some are will­ing to go in or­der to force drug prices low­er; how­ev­er we be­lieve these groups have an up­hill bat­tle, and even if they were to pre­vail there would be lim­it­ed read through to oth­er rare dis­ease com­pa­nies whose busi­ness mod­els re­ly on pre­mi­um pric­ing.”

Leerink re­minds in­vestors that a sim­i­lar strat­e­gy was used against Gilead, Ver­tex, and No­var­tis, among oth­ers. Those ef­forts failed, an­a­lysts said in a note.

But KEI, one of the or­ga­ni­za­tions that wrote the let­ter, said Leerink’s note is mis­lead­ing.

“If they are re­fer­ring to re­search we’ve sub­mit­ted to HHS on drugs mar­ket­ed by Gilead and oth­er firms, that state­ment isn’t quite ac­cu­rate,” KEI spokesper­son Kim Tre­anor wrote in an email. “Our re­search has been sub­mit­ted very re­cent­ly and is still be­ing re­viewed by HHS, so no res­o­lu­tion to these cas­es has oc­curred as of yet.”

The let­ter writ­ers do ac­knowl­edge, how­ev­er, that the ac­tion they’re re­quest­ing is un­prece­dent­ed:

In the past, the fed­er­al gov­ern­ment has, on sev­er­al oc­ca­sions, asked re­cip­i­ents of fed­er­al grants and con­tracts to cor­rect fail­ures to dis­close fed­er­al fund­ing of the in­ven­tions, but has not ex­er­cised its rights to take the ti­tle of such patents for pur­pos­es of in­flu­enc­ing drug prices. In this re­spect, we rec­og­nize that we are ask­ing HHS to do some­thing new.

And lat­er, the let­ter re­it­er­ates what the or­ga­ni­za­tions are up against:

We re­spect­ful­ly ask for a meet­ing with your staff to fur­ther dis­cuss this is­sue, not­ing that as a prac­ti­cal mat­ter, if the de­ci­sions are del­e­gat­ed sole­ly to the NIH (Of­fice of Tech­nol­o­gy Trans­fer) staff it is high­ly un­like­ly any ac­tion will be tak­en to mod­er­ate the price of this drug.

Read the full let­ter here, writ­ten and sub­mit­ted by KEI, Health GAP, Pa­tients for Af­ford­able Drugs, Peo­ple of Faith for Ac­cess to Med­i­cines, So­cial Se­cu­ri­ty Works and Uni­ver­si­ties Al­lied for Es­sen­tial Med­i­cines.


Im­age: Sarep­ta Ther­a­peu­tics. AP IM­AGES

The FDA will hus­tle up an ex­pe­dit­ed re­view for As­traZeneca’s next shot at a block­buster can­cer drug fran­chise

AstraZeneca paid a hefty price to partner with Daiichi Sankyo on their experimental antibody drug conjugate for HER2 positive breast cancer. And they’ve been rewarded with a fast ride through the FDA, with a straight shot at creating another blockbuster oncology franchise.

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Sean Parker, AP

Sean Park­er helps cre­ate a CRISPRed cell ther­a­py 2.0 play — and he’s got a high-pro­file set of lead­ers on the team

You can rack up one more high-profile debut effort in the wave of activity forming around cell therapy 2.0. It’s another appealing Bay Area group that’s attracted some of the top hands in the business to a multi-year effort to create a breakthrough. And they have $85 million in hand to make that first big step to the clinic.

Today it’s Ken Drazan and the team at South San Francisco-based ArsenalBio that are coming from behind the curtain for a public bow, backed by billionaire Sean Parker and a collection of investors that includes Beth Seidenberg’s new venture investment operation based in LA.
Drazan — a J&J Innovation vet with a long record of entrepreneurial endeavors — exited the stage in 2018 when his last mission ended as he stepped aside as president of Grail. It wasn’t long, though, before he was helping out with a business plan for ArsenalBio that revolved around the work of a large group of interconnected scientists supported by the Parker Institute for Cancer Immunology.
The biotech started by putting together an “arsenal” of technologies aimed at making cell therapies for cancer much, much better than the rather crude first-generation drugs that hit the market from Novartis and Kite.
Their drugs have become the baseline against which all others are being measured.
“The technology set we’re developing is independent of the chassis,” Drazan tells me. “It doesn’t have to be autologous (extracted from the patient) or allogeneic (off the shelf). It doesn’t have to be a T cell, it could be a B cell.” But they are starting out on the autologous side, where they have the most knowledge and insight into manufacturing techniques.
It also doesn’t have to be close to the clinic.
Drazan expects the biotech will be working its way through preclinical operations for “a few years,” with enough money from the $85 million launch round to get into humans.
By today’s superheated fundraising standards, that’s not a huge amount of cash. Lyell, another cell therapy 2.0 startup we featured last week, raised $600 million in a year, including a big chunk of cash from GlaxoSmithKline. Drazan is interested in dealmaking as well, but he also knows he has the cash necessary to support the company for a good run — a key part of what it takes to bring together a stellar team of top players.

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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.

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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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?

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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