Scott Got­tlieb aced his con­fir­ma­tion hear­ing, vow­ing to main­tain R&D gold stan­dard

Scott Got­tlieb – April 5, 2017 Zach Gib­son/Get­ty Im­ages


Sen­ate De­moc­rats didn’t waste any time in rais­ing their ob­jec­tions to Scott Got­tlieb as the next FDA com­mis­sion­er.

Sen­a­tor Pat­ty Mur­ray (D-Wash­ing­ton) start­ed her re­marks at Got­tlieb’s con­fir­ma­tion hear­ing this morn­ing voic­ing her con­cerns about “whether you can with­stand po­lit­i­cal pres­sure” in main­tain­ing an un­bi­ased, sci­ence-based ap­proach in main­tain­ing the gold stan­dard on safe­ty and ef­fi­ca­cy re­views of new drugs.

Mur­ray com­plained that sen­a­tors had on­ly days to re­view Got­tlieb’s “un­prece­dent­ed fi­nan­cial en­tan­gle­ments” in the phar­ma in­dus­try — with dozens of drugs from the com­pa­nies he’s worked with head­ed to the FDA — as well as more than 800 list­ed pub­li­ca­tions which “raise con­cerns about po­ten­tial con­flicts of in­ter­est.”

Scott Got­tlieb this morn­ing on Capi­tol Hill at his nom­i­na­tion hear­ing. (CRED­IT: C-SPAN/End­points News)

Got­tlieb, though, came pre­pared to re­but those sug­ges­tions, mak­ing promis­es to main­tain an in­de­pen­dent FDA com­mit­ted to find­ing new ef­fi­cien­cies at the agency while mak­ing sure that all the new prod­ucts the agency ap­proves are safe and ef­fec­tive, meet­ing the agency’s long-term gold stan­dard on R&D.

“I’ve seen the im­por­tance of the FDA’s work as both a doc­tor and a pa­tient,” Got­tlieb, a can­cer sur­vivor, said in his open­ing state­ment. He added that his in­vest­ments and en­deav­ors were aimed at im­prov­ing health­care in the US. Some have worked, oth­ers didn’t and many oth­ers are still in de­vel­op­ment.

Re­gard­less of those ties, he vowed to be an “im­par­tial and in­de­pen­dent ad­vo­cate for the pub­lic health” main­tain­ing a rig­or­ous ap­proach to re­view­ing drugs. “We’re at an in­flec­tion point in bio­med­ical sci­ence,” he added, and the chal­lenge at the FDA will be get­ting the most bang for its reg­u­la­to­ry bucks.

“We should re­ject the false di­choto­my that it all boils down to a choice be­tween speed and safe­ty.” Bet­ter ef­fi­cien­cy and safe­ty are both pos­si­ble, he said, while main­tain­ing the gold stan­dard. And he added that “it’s in­cum­bent on us to have a world class work force,” hir­ing the best for reg­u­la­to­ry work, which Sen­a­tor Lamar Alexan­der not­ed was Got­tlieb’s pick as his most im­por­tant chal­lenge.

Got­tlieb al­so helped soothe any in­dus­try con­cerns that he might want to come in as an abra­sive re­former, work­ing at odds with staff.

“I think every­thing is a bot­tom-up ap­proach in the agency,” he told the law­mak­ers. “The ideas re­al­ly need to come from the ca­reer staff in the cen­ters.” That’s go­ing to be a chal­lenge as the agency re­cruits re­place­ments for a line­up of se­nior agency of­fi­cials who are due for re­tire­ment.

Got­tlieb promised to bring best prac­tices to those ar­eas in the FDA that have been lag­ging. There’s been an “un­even adop­tion” of the break­through drug pro­gram, he added, which now re­quires broad adop­tion.  The prospec­tive com­mis­sion­er al­so said that while he was not crit­i­cal of Phase III, there is a pos­si­bil­i­ty with new tech­nol­o­gy to com­press Phase II and Phase III in­to an adap­tive de­sign.

And, as ex­pect­ed, he rel­ished the prospect of tack­ling a slew of com­plex gener­ics await­ing an ap­proval at the FDA. Many drugs are sold at high prices, he said, but should be sub­ject to com­pe­ti­tion.

A few days ago Got­tlieb care­ful­ly de­lin­eat­ed ties to some two dozen bio­phar­ma com­pa­nies he’s been work­ing with over the past eight years of the Oba­ma ad­min­is­tra­tion. He’s worked with Glax­o­SmithK­line, Ver­tex and Bris­tol-My­ers, so you can check the box on in­dus­try con­flicts of in­ter­est that De­moc­rats will con­demn.

Among Got­tlieb’s oth­er lines of work, the for­mer deputy FDA com­mis­sion­er penned a se­ries of ar­ti­cles on bio­phar­ma for Forbes, and the Dems have been giv­ing them a close look. Two pieces float­ed to the top of the read­ing list. One cen­tered on his pro­pos­al to move re­spon­si­bil­i­ty for iden­ti­fy­ing the opi­oid sup­pli­ers who were break­ing the law from the DEA to HHS. The oth­er dealt with his crit­i­cism of the REMS pro­grams, which set up post-mar­ket­ing safe­ty pro­grams to lim­it risks seen in cer­tain drugs.

Add it all up, says Sher­rod Brown (D-Ohio), and you find some­one who will “roll over for his Big Phar­ma friends,” as The Hill re­ports.

Re­pub­li­cans, though, were large­ly con­tent to lim­it their com­ments to praise for the nom­i­nee and his three young daugh­ters, who were seat­ed be­hind the can­di­date. And it’s the Re­pub­li­cans who have the votes to make this hap­pen.

The in­dus­try, mean­while, has made it clear that af­ter hear­ing some of the ideas backed by oth­er com­mis­sion­er can­di­dates like Jim O’Neill – who’s sug­gest­ed ap­prov­ing drugs as soon as they’re de­ter­mined safe — Got­tlieb is the best can­di­date by far. In a snap poll of qual­i­fied End­points News sub­scribers, 87% of the 580 who reg­is­tered an opin­ion backed Got­tlieb’s nom­i­na­tion.

So you can ex­pect most peo­ple in bio­phar­ma to keep their fin­gers crossed for Got­tlieb.

Bot­tom line: Got­tlieb came well pre­pared for every ques­tion, care­ful not to step be­yond the bound­aries of the com­mis­sion­er’s job when in­vit­ed to com­ment on Oba­macare. His an­swers were pol­ished and prac­ticed, leav­ing lit­tle room for un­ex­pect­ed at­tacks. And he de­flect­ed crit­i­cism eas­i­ly, promis­ing to avoid any po­ten­tial con­flicts of in­ter­est.

Bar­ring a last minute sur­prise, Got­tlieb’s con­fir­ma­tion as the next FDA com­mis­sion­er is all but guar­an­teed.

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

No­var­tis is ax­ing 150 ear­ly dis­cov­ery jobs as CNI­BR shifts fo­cus to the de­vel­op­ment side of R&D

Novartis is axing some 150 early discover jobs in Shanghai as it swells its staff on the drug development side of the equation in China. And the company is concurrently beefing up its investment in China’s fast-growing biotech sector with a plan to add to its investments in local VCs.

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Democratic presidential candidate, U.S. Sen. Elizabeth Warren (D-MA) speaks during the Nevada Democrats' "First in the West" event at Bellagio Resort & Casino on November 17, 2019 in Las Vegas, Nevada (Getty Images)

Eliz­a­beth War­ren pro­pos­es us­ing com­pul­so­ry li­cens­ing, an­titrust ac­tions to break bio­phar­ma’s con­trol of drug pric­ing — and here are the block­busters she’s tar­get­ing first

Nancy Pelosi’s drug pricing bill may have sparked some industrial strength headaches on the money side of biopharma, but Elizabeth Warren seems determined to become biopharma’s Nightmare on Pennsylvania Avenue.
Warren, one of the top-ranked candidates for the Democratic presidential nomination backing Medicare for all, is circulating a new plan that promises to break the industry’s grip on drug prices — and she has some very specific examples of how she would do it.
The Warren plan would rely on the federal government’s compulsory licensing powers to seize the IP of blockbuster drugs like Truvada and Harvoni to provide them at a fraction of what Gilead sells them for in the US. And she would throw some antitrust actions in as needed to rein in the price of Humira, AbbVie’s cash cow that continues to dominate the list of the most profitable therapeutics on the market.
Notably, she plans to rely on the powers already vested in the federal government, rather than suggest remedies that would require the assent of a deeply divided Congress.
In addition to the blockbusters on the list, Warren sends a clear signal that the same tactics would be used to beef up the supply of cheap antibiotics, as needed. And the same action could befall any other therapy patients can’t afford.

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Mer­ck’s $1B cash gam­ble pays off with a sur­pris­ing PhI­II car­dio suc­cess for Bay­er’s heart drug veri­ciguat

More than 3 years after Merck stepped up and paid $1 billion in cold, hard cash to gain the US commercial rights to Bayer’s high-risk heart drug vericiguat in a broad-ranging cardio alliance, the partners say their Phase III study has come through with promising data and a date with regulators.
We don’t have the data, and won’t until they put it out at an upcoming scientific session, but Merck touted the results, saying that their big Phase III VICTORIA study hit the primary endpoint  — with vericiguat combined with available therapies reducing “the risk of the composite endpoint of heart failure hospitalization or cardiovascular death in patients with worsening chronic heart failure with reduced ejection fraction (HFrEF) compared to placebo when given in combination with available heart failure therapies.”
Depending on the hard data, and how it breaks out with the combinations used, this drug could pose a threat to Novartis’ blockbuster drug Entresto, currently at $1.6 billion while analysts expect peak sales to hit $4 billion.
The drug is a soluble guanylate cyclase (sGC) stimulator, which Bayer and Merck have had high hopes for. Evidently, so did cardiologists. Cowen’s last analysis set potential sales at $400 million in 2024, but that number could go up significantly now.
Cowen’s Steve Scala noted this morning:
Vericiguat could be a lucrative product for Merck, and one with potentially under-appreciated value. At Cowen’s Therapeutics Conference in September 2019, 80% of specialists anticipated a positive result from VICTORIA whereas only 51% of investors shared this optimism.
Investigators recruited more than 5,000 patients at more than 600 centers in 42 countries for this study — one of the most expensive propositions in R&D. Millions of people in the US suffer from heart failure with reduced ejection fraction when the failing heart fails to contract properly to eject blood into the system. Bayer holds ex-US rights to the drug and also stands to earn cash from the $1.1 billion in milestones Merck agreed on for their collaboration.
Remarkably, the drug was pushed into Phase III despite failing the mid-stage trial — though investigators flagged a success at the high dose of 10 mg. In VICTORIA, researchers started patients at 2.5 mg and then titrated up to 5 and then 10 mg.

Alk­er­mes forges $950M biotech buy­out deal in a bold bet on an ear­ly-stage CNS drug plat­form

Alkermes $ALKS is investing $100 million cash and committing up to $850 million more in milestones in a big wager on a very early-stage CNS discovery platform. And the biotech is adding $20 million more to fund next year’s new research work on the platform it’s acquiring in today’s buyout with an eye to expanding the research work in oncology.

The biotech, helmed by Richard Pops, is buying Rodin Therapeutics, which had focused early on Alzheimer’s disease. Pops’ buyout, though, isn’t focused solely on the most troublesome sector in pharma R&D.

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Left to right: Arthur Pappas, Robert Nelsen, Peter Kolchinsky Doug Cole and David Beier

In rare po­lit­i­cal for­ay, top biotech in­vestors urge Con­gress to re­ject drug pric­ing bill

Thirteen of the top biotech venture capitalists in the country wrote a letter last week warning lawmakers that if Congress passes a drug pricing bill House Speaker Nancy Pelosi has put before lawmakers, they won’t be able to invest in biomedical research at their current rate, and patients will suffer.

“If policies such as those included within H.R. 3, the Lower Drug Costs Now Act, are passed, our ability to continue to invest in future biomedical innovation will be severely constrained, thus crushing the hopes of millions of patient waiting for the next breakthroughs to treat or cure their cancers, rare genetic diseases, Alzheimer’s, or other serious and life-threatening conditions,” they wrote in a letter addressed to the highest-ranking Democrats and Republicans in the House and Senate and acquired by Endpoints News. 

Dicer­na scores broad, 'rest of liv­er' deal with No­vo Nordisk, bag­ging $225M in cash to hit some 30 tar­gets with RNAi plat­form

Turns out Dicerna wasn’t done with deals yet after locking in $200 million upfront from Roche for a hepatitis B cocktail two weeks ago.

Novo Nordisk has signed on as the latest partner to its GalXC RNAi platform, handing over $175 million in cash to claim any and all targets of interest in liver-related cardio-metabolic diseases that are not already reserved in previous pacts. The Danish drugmaker — which has signaled its interest to expand considerably beyond its core diabetes franchise into areas like NASH — is also purchasing $50 million worth of Dicerna’s equity at a 25% premium of $21.93 per share. More research payments and milestones extending to the billions are on the line.

Gene ther­a­py wins the in­side track at EMA; PPD files for IPO

→ Gene therapy maker Orchard Therapeutics has been granted an accelerated assessment for OTL-200 by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The gene therapy — in development in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy — being used towards the treatment of metachromatic leukodystrophy.

→ Pharmaceutical Product Development has announced that its parent company, PPD, Inc has submitted a draft to the SEC relating to the proposal of an IPO of the parent company’s common stock. Number of shares and price range have not yet been determined.

Pfiz­er gets biosim­i­lar ap­proved for Hu­mi­ra, set­ting up com­pe­ti­tion — in 2023

In the story lawmakers and drug pricing reform advocates have told about the drug industry, there are perhaps few greater villains than Humira and its maker AbbVie.

Between 2012 and 2018, AbbVie upped the drug’s annual after-rebates cost from $19,000 to $38,000 in the US, with sticker prices now over $60,000 per year — increases that led to accusations of price gouging, most recently from Democratic presidential frontrunner Elizabeth Warren.