An­a­lysts track a pay­er re­volt against Sarep­ta’s con­tro­ver­sial Duchenne drug

Janet Wood­cock

When the FDA’s Janet Wood­cock pushed through a con­tro­ver­sial ap­proval for Sarep­ta’s Ex­ondys 51 for Duchenne mus­cu­lar dy­s­tro­phy in Sep­tem­ber, she cre­at­ed a test case for pay­ers who were es­sen­tial­ly be­ing asked to re­im­burse for an ex­per­i­men­tal drug.

But in­stead of rolling with the sit­u­a­tion and pay­ing a price that starts at $300,000 or so a year, many of the pay­ers are kick­ing back hard, ac­cord­ing to a sur­vey of pay­ers con­duct­ed by Jef­feries.

Gena Wang, Jef­fries

“We con­duct­ed in depth due dili­gence on Ex­ondys 51 launch with 4 pri­vate pay­ers, 3 Med­ic­aid ex­perts, and 2 DMD KOLs with ex­pe­ri­ence in ad­min­is­ter­ing Ex­ondys 51,” not­ed Jef­feries Gena Wang, who had been in the large con­tin­gent of an­a­lysts who had pre­dict­ed a re­jec­tion for Sarep­ta. In ad­di­tion we com­piled a com­pre­hen­sive list of pub­lished med­ical poli­cies from 21 pri­vate pay­ers (5 na­tion­al & 15 re­gion­al MCOs; 1 PBM). Based on avail­able da­ta, 3/5 na­tion­al and 8/15 re­gion­al man­aged care or­ga­ni­za­tions (MCOs) have de­nied/re­strict­ed cov­er­age for Ex­ondys 51, in line with our ex­pec­ta­tion of push­back from pri­vate pay­ers.”

That push­back has cost Sarep­ta $SRPT plen­ty. The biotech has seen its share price fade back by about half since the ap­proval more than two months ago as ques­tions have con­tin­ued to cir­cu­late about pay­ers’ po­si­tion on the drug.

An­them got the back­lash rolling with its po­si­tion that it will not cov­er an ex­per­i­men­tal drug, even though it’s been giv­en an ac­cel­er­at­ed ap­proval. And Hu­mana fol­lowed up with re­stric­tive rules that re­quires pa­tients be­ing able to walk so long as they are re­im­burs­ing for the drug. It’s clear from the Jef­feries sur­vey that a slight ma­jor­i­ty of pay­ers don’t want to han­dle this like any oth­er ap­proved ther­a­py for a rare and dead­ly dis­ease.

The Ex­ondys 51 ap­proval trig­gered an in­ter­nal civ­il war at the FDA, pit­ting top reg­u­la­tors against Wood­cock, who ex­pressed sup­port for the drug and the com­pa­ny, even though it had on­ly been stud­ied in a tiny tri­al and the da­ta had been harsh­ly re­viewed by in­ter­nal reg­u­la­tors. That view helped set the stage for its re­jec­tion by a pan­el of out­side ex­perts, which al­so didn’t help the drug’s chances once it was hand­ed over to pay­ers to de­cide.

The kick­back from pay­ers is go­ing to make it much hard­er for some of these fam­i­lies to get this drug, just when they felt that the biggest hur­dle had al­ready been cleared.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

Biden ad­min­is­tra­tion backs a po­lar­iz­ing pro­pos­al to waive IP for all Covid-19 vac­cines

In a surprise U-turn, the Biden administration said Wednesday that it will support a proposal at the World Trade Organization to temporarily waive intellectual property protections on Covid-19 vaccines.

The proposal, backed by South Africa and India at the WTO, seeks to help developing countries with limited vaccine supplies. The US and Europe historically opposed the proposal, saying IP should be protected because it incentivizes new drug and vaccine development.

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FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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