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

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”

CDC’s Robert Redfield, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust of the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

Covid-19 roundup: Op­er­a­tion Warp Speed's 7th vac­cine is live at­ten­u­at­ed; Small biotech touts big suc­cess where gi­ants have failed

Operation Warp Speed is stacking its vaccine portfolio with a “TBD” new candidate: a live attenuated vaccine that can be administered in a single dose, potentially as an oral formulation rather than an injection.

Sound familiar?

That could be because the unannounced candidate appears to match the profile of an inoculation being developed by Merck, according to Bloomberg, which first reported the development based on a presentation by Moncef Slaoui.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.

On­ly five months af­ter a Se­ries A launch, Taysha goes pub­lic with $157M IPO

As has been the trend in 2020, Taysha Gene Therapies has become the latest biotech to make a quick ascent from a small, privately-funded company to enjoying its very own Nasdaq ticker.

The Dallas-based biotech raised $157 million for its IPO after pricing shares at $20 apiece Thursday, the high-point of its expected range. Initially pegging $100 million in financing, Taysha offered a little less than 8 million shares and will trade under the $TSHA symbol.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.

David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the company.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.