Thumbs Up/Thumbs Down: Ma­jor in­sur­er de­liv­ers a bad blow to Duchenne fam­i­lies

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.


  An­them’s rul­ing on Duchenne drug is a bad blow to fam­i­lies

The big US in­sur­er An­them has de­ter­mined that Sarep­ta’s con­tro­ver­sial Duchenne mus­cu­lar dy­s­tro­phy drug Ex­ondys 51 is “in­ves­ti­ga­tion­al and not med­ical­ly nec­es­sary,” a blow for fam­i­lies look­ing to gain cov­er­age for this drug. The de­ter­mi­na­tion cites a long and trou­bled his­to­ry for this drug, with failed stud­ies and a host of ques­tion­able moves Sarep­ta $SRPT made in ad­vanc­ing this drug on the back of a tiny study. “Ex­ondys 51 failed to show it im­proves health out­comes, and there­fore it is not a cov­ered ben­e­fit for our mem­bers,” An­them spokes­woman Leslie Por­ras told Reuters, con­firm­ing the ob­vi­ous. The dis­cus­sion over this drug turned in­to a bat­tle in­side the FDA, with CDER di­rec­tor Janet Wood­cock win­ning the ar­gu­ment in fa­vor of an ac­cel­er­at­ed ap­proval over the ve­he­ment ob­jec­tions of se­nior-lev­el of­fi­cials. As in­sur­ers refuse to cov­er this drug, which will be sold for $300,000, Wood­cock has set up a sce­nario where fam­i­lies will be ex­pect­ed to fork over huge sums to treat chil­dren with a drug they have vowed works just fine. That would be a trag­ic out­come. The drug is ex­per­i­men­tal, of course. But fam­i­lies shouldn’t be left on the hook for the cost of a rare dis­ease treat­ment like this, which may have no af­fect on the dis­ease at all. And there­in lies the tragedy be­hind this painful sit­u­a­tion. In­sur­ers can af­ford it and should step up and do the right thing, but we doubt they will. Af­ter all, in­sur­ers have in­vestors as well. Sarep­ta’s shares were down 8% to­day on the news.


 Thumbs up for the fastest, most de­ter­mined deal-mak­er in bio­phar­ma

Brent Saun­ders proved again that he’s the most in­tre­pid buy­er in the in­dus­try to­day. In To­bi­ra’s blow-by-blow ac­count of the auc­tion that took place for the com­pa­ny in SEC doc­u­ments, Saun­ders got the ball rolling last sum­mer at din­ner with To­bi­ra ex­ecs in NYC, and didn’t let up un­til he had bagged the com­pa­ny and its (yes, failed) NASH drug for a big pre­mi­um. Amid all the dis­cus­sion about biotech val­u­a­tions these days, Saun­ders’ Al­ler­gan has cash for new deals, and it’s not wait­ing around to see how the num­bers change. We’ll see even­tu­al­ly if the strat­e­gy on To­bi­ra pays off, but you have to ad­mire the do-it-now at­ti­tude at Al­ler­gan.


 Hubs win, again

Our most pop­u­lar sto­ry to ring the web traf­fic bell this week is def­i­nite­ly our scoop on No­var­tis‘ lat­est reshuf­fling of its glob­al R&D ops. The com­pa­ny has some clear ideas about bring­ing to­geth­er re­searchers in­to its main hubs, which once again un­der­scores the in­dus­try trend to­ward in­ter­min­gling with more biotechs and phar­ma R&D groups. Un­for­tu­nate­ly, the trend has a heavy im­pact on em­ploy­ees, as we saw with No­var­tis’ re­cent de­ci­sion to shut­ter its stand­alone cell ther­a­py op­er­a­tions and in­te­grate with the broad­er on­col­o­gy group. But the ir­re­sistible mo­men­tum has to be rec­og­nized as an over­all pos­i­tive for the in­dus­try, which will see the con­tin­ued ex­pan­sion of in­dus­try R&D hubs at the ex­pense of out­liers like Sin­ga­pore.


  RNAi suf­fers an­oth­er dam­ag­ing blow

Al­ny­lam $AL­NY has seen it all when it comes to RNAi de­vel­op­ment. They were there when en­thu­si­asm for gene si­lenc­ing hit a peak and Big Phar­ma stepped in. Then they watched Big Phar­ma step out. And they were ready when Sanofi de­cid­ed to part­ner up in a big way. With two drugs in late-stage de­vel­op­ment, they were al­so hop­ing to do some pi­o­neer­ing com­mer­cial work in the not-too-dis­tant fu­ture. Sad­ly, one of its two Phase III drugs im­plod­ed this week. Al­ny­lam pinned it on an im­bal­ance of deaths, mean­ing more pa­tients were dy­ing in the drug arm. But frankly, we don’t have a whole lot of hard in­sight on what went wrong. You can be sure that every bit of da­ta com­ing out in the fu­ture will be giv­en a hard look by an­a­lysts. This field still tends to ad­vance and re­treat as a group based on the work of the lead­ers. Right now, RNAi is back in the dog house. It’s up to Al­ny­lam to get back out.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

Can a mag­net­ic cell ther­a­py re­place corneal trans­plan­ta­tion? As eight-year jour­ney leads to the clin­ic, two broth­ers un­veil bold vi­sion

Jeff Goldberg was getting acquainted with a brand new way to do corneal transplants when an even newer, even bolder idea hit him.

It was almost 10 years ago, and Goldberg was in his first faculty position at Bascom Palmer Eye Institute at the University of Miami. Scientists had developed a new way to do cornea transplants where instead of sewing a whole donor cornea — a decades-old practice — they were just engrafting the inner layer of cells.

News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siolta Therapeutics, a microbiome company targeting allergic diseases, raked in a $30 million Series B to develop its lead candidate, STMC-103H. The drug, which has been FDA fast-tracked, is headed for proof-of-concept trials, according to the company. Its various indications include allergic asthma, food allergies, atopic dermatitis, allergic rhinitis, and allergy prevention.

The news comes just after the California-based biotech added a prominent biopharma veteran as an advisor: 20-year Gilead CEO John Martin. The biotech also gained Richard Shames as CMO, who came by way of Protagonist Therapeutics.

Embattled CDC director Robert Redfield (AP Images)

Covid-19 roundup: CDC ad­vi­so­ry com­mit­tee de­lays pri­or­i­ty dis­tri­b­u­tion vote; EU re­port­ed­ly in­dem­ni­fy­ing vac­cine mak­ers

A federal committee that advises the CDC was expected to hold a vote Tuesday on a plan regarding the distribution for initial doses of approved Covid-19 vaccines. But that vote has been scrapped.

The Advisory Committee on Immunization Practices, or ACIP, won’t be voting until the committee members learn more about which vaccines become available first, the Wall Street Journal reported. The vote could potentially wait until a specific vaccine is authorized before recommending how to dole out the first doses.

Anthony Coyle (Repertoire)

Flag­ship's merged biotech Reper­toire nets ex-Pfiz­er CSO An­tho­ny Coyle as R&D chief

Flagship is building a big-name C-suite at its new, $220 million merged biotech.

Repertoire Immune Medicines, which already boasts former Bioverativ chief John Cox as its CEO, announced yesterday that Anthony Coyle, the former Pfizer CSO and the founding CEO of Pandion, will join as their head of R&D.

“As we progress clinical trials for our multi-clonal T cell candidates in immuno-oncology, Tony’s deep expertise in cellular immunology and novel therapeutic development will help us achieve our vision of creating a new class of transformative medicines for patients,” Cox said in a statement.

Zai Lab hauls in $761M from Hong Kong IPO to push Ze­ju­la, more bud­ding can­di­dates in Chi­na — re­port

Zai Lab is set to net more than $761 million from its secondary listing in Hong Kong after pricing the IPO at $72.51 (HKD$562) — just a hair below its Nasdaq closing price on Monday, Bloomberg and Nikkei Asian Review reported.

A pioneer in bringing Western drugs to China, co-founder and CEO Samantha Du has more than tripled Zai Lab’s market cap in the three years it’s been public in the US. The HKEX listing is designed to fund R&D and commercialization for the current portfolio while fueling new in-licensing pacts, the biotech wrote in a filing.

Tae Han Kim, Samsung Biologics CEO (SeongJoon Cho/Bloomberg via Getty Images)

Sam­sung Bi­o­log­ics nets $330M+ deal from As­traZeneca ahead of 'Su­per Plan­t' con­struc­tion

Just a few weeks after announcing plans to construct a $2 billion “Super Plant,” Samsung Biologics is keeping its foot on the gas.

The Korean CDMO has inked a $330.8 million manufacturing contract with AstraZeneca, the companies announced Monday evening, providing the British drugmaker the ability to expand production capabilities in the Asia market. Per terms of the deal, the partnership could be increased to $545.6 million.