Opin­ion: US and EU re­views of blood clots and J&J’s Covid vac­cine show why a trans­par­ent process mat­ters

When the CDC’s top ad­vi­so­ry com­mit­tee met last week to re­view whether a hand­ful of very rare but po­ten­tial­ly fa­tal blood clots with low platelets might be linked to J&J’s Covid-19 vac­cine, the as­sump­tion was that the com­mit­tee would, at the very least, fol­low its own agen­da and vote on whether to wait for more da­ta, or make in­ter­im age- or risk-based rec­om­men­da­tions for the vac­cine.

In­stead, the com­mit­tee did nei­ther, punt­ing its vote un­til this Fri­day when it can hold a longer dis­cus­sion with the lat­est da­ta. None of the ACIP mem­bers raised ma­jor con­cerns with the de­ci­sion, or lack there­of, de­spite some calls to not pause the vac­cine in­def­i­nite­ly.

But the in­ac­tion al­so raised con­cerns about whether the CDC and FDA were shirk­ing their re­spon­si­bil­i­ties and un­nec­es­sar­i­ly de­lay­ing J&J vac­ci­na­tions as the race to slow the spread of the coro­n­avirus con­tin­ues.

In con­trast, the Eu­ro­pean Med­i­cines Agency’s safe­ty com­mit­tee quick­ly con­clud­ed its re­view of the blood clots and the J&J vac­cine on Tues­day, de­cid­ing the vac­cine’s ben­e­fits out­weigh the risks, while adding a warn­ing to the vac­cine’s la­bel, even as no one in Eu­rope has re­ceived the vac­cine yet. Fol­low­ing the com­mit­tee’s an­nounce­ment, J&J said it would re­sume ship­ments of the vac­cine.

At first blush, the EMA, which just went through a sim­i­lar re­view with As­traZeneca’s vac­cine and came to a sim­i­lar con­clu­sion, seems to have been the more proac­tive reg­u­la­tor. The EMA made a quick de­ci­sion on the avail­able da­ta and re-start­ed the process for get­ting more shots in­to arms.

But be­yond the head­line and fi­nal de­ci­sion lies a very dif­fer­ent sto­ry.

In the case of the CDC’s com­mit­tee, any mem­ber of the pub­lic (any­where in the world) could turn on a com­put­er and watch the pro­ceed­ings, lis­ten to the se­ri­ous­ness of the de­tails, hear the ar­gu­ments and see the slides that each of the com­mit­tee mem­bers re­view. The pub­lic even has an op­por­tu­ni­ty to weigh in at the CDC meet­ing, al­though it’s un­clear how those com­ments are ac­tu­al­ly used.

The ACIP de­ci­sion to de­lay ac­tion and wait for more da­ta on the blood clots may seem like an al­most neg­li­gent re­sponse, es­pe­cial­ly giv­en the EMA’s snap de­ci­sion, when time is of the essence. But the risk cal­cu­la­tions for the two re­gions are very dif­fer­ent. The US has more of the Pfiz­er and Mod­er­na Covid vac­cines than the EU to fill the gap left by the J&J pause. The US al­so has about 10 mil­lion dos­es of the J&J vac­cine wait­ing on shelves so if the FDA and CDC of­fer a green­light, those dos­es will im­me­di­ate­ly be ad­min­is­tered.

Al­so, the EMA’s safe­ty com­mit­tee com­plet­ed its analy­sis of the J&J and As­traZeneca vac­cine da­ta be­hind closed doors, on­ly of­fer­ing a peek in­to what oc­curred via lim­it­ed press re­leas­es and press con­fer­ences that usu­al­ly lead to as many new ques­tions as an­swers.

Pe­ter Ar­lett

For in­stance, at each of the EMA press con­fer­ences on the vac­cine la­bel up­dates, Pe­ter Ar­lett, the head of phar­ma­covig­i­lance at the EMA, has un­veiled new fig­ures on the cas­es of blood clots with low platelets for each of the vac­cines au­tho­rized in the EU.

But in do­ing so, Ar­lett fails to of­fer any fur­ther in­for­ma­tion on how many to­tal dos­es of the vac­cines have been ad­min­is­tered or where these new cas­es oc­curred. At least twice now, ei­ther Ar­lett or some­one else at the EMA has had to cor­rect or clar­i­fy what the num­bers mean, or where the cas­es did or did not oc­cur.

If the EMA is go­ing to re­lease these num­bers, why not put them in a press re­lease and ful­ly ex­plain them? In­stead, the agency leaves it to the pub­lic to de­cide on what they might mean, which is con­cern­ing as they are not ap­ples-to-ap­ples com­par­isons. Pfiz­er see­ing 25 cas­es of the blood clots and low platelets ver­sus J&J see­ing 8 cas­es are not com­pa­ra­ble con­sid­er­ing how many tens of mil­lions of more dos­es of the Pfiz­er vac­cine have been ad­min­is­tered.

In oth­er in­stances (e.g. re­leas­ing clin­i­cal tri­al re­sults), the EMA has tak­en a lead­ing role in show­ing the world how trans­paren­cy works. But in this case, even with the FDA and CDC tak­ing ex­tra days to make its de­ci­sion, and even if the de­ci­sion is to re­strict the use of the J&J vac­cine to a spe­cif­ic pop­u­la­tion, the im­por­tance of trans­paren­cy is clear.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).

BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

UP­DAT­ED: In­ter­nal Trump-era emails re­veal a pletho­ra of celebs, com­pa­nies vy­ing for FDA’s at­ten­tion

The FDA on Thursday publicly released a trove of about 500 pages of internal and heavily redacted emails, showing how celebrities like Dr. Oz and Laura Ingraham vied for the FDA’s attention on Covid-related issues that ultimately proved to be a waste of time.

Many of the conversations, obtained via the Freedom of Information Act, involve major Trump-era health officials who, in retrospect, made crucial and often ill-advised decisions on increasing access to the over-hyped hydroxychloroquine.

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