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

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

Jacob Van Naarden, Senior VP, CEO of Loxo Oncology at Lilly; President, Lilly Oncology

Eli Lil­ly bags FDA nod for Verzenio in ear­ly breast can­cer, but a con­tro­ver­sial di­ag­nos­tic could dog its roll­out

As Eli Lilly works to consolidate its internal and Loxo teams into an oncology powerhouse, the drug giant is putting high hopes on CDK 4/6 inhibitor Verzenio to help drive the portfolio into the future. Now, the drug has scored a paradigm-altering win in early breast cancer — but will a controversial companion diagnostic hamstring Lilly’s market plans?

The FDA on Wednesday approved CDK 4/6 inhibitor Verzenio in combination with physician’s-choice endocrine therapy to cut the risk of relapse in patients with high-risk HR-positive, HER2-negative breast cancer, Lilly said in a release.

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As TRIPS coun­cil meets, the IP waiv­er for vac­cines is on life sup­port ahead of a De­cem­ber dead­line

The WTO’s TRIPS Council is meeting today and tomorrow to discuss a Covid-19 vaccine IP waiver that remains divisive and unlikely to be adopted thanks to European opposition, but which proponents still think could unlock more vaccine doses for low and middle-income countries.

Following the meetings this week, it’s expected there will be a better sense if some kind of waiver can be agreed to by December, Tahir Amin, an IP lawyer and co-executive director of I-Mak, told Endpoints News.