Thumbs Up/Thumbs Down: Am­gen's si­lence is deaf­en­ing; The drug pric­ing mob is back

 

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

 

Am­gen bugs out, and they’re mak­ing every­one in bio­phar­ma look bad.

Just about every­one en­gaged in drug de­vel­op­ment loves to talk about pos­i­tive da­ta and how the most im­por­tant thing about suc­cess is al­ways the pa­tient who ben­e­fits. But when the light turns red on a set­back, the death star men­tal­i­ty dom­i­nates and ooz­ing com­pas­sion dis­ap­pears as they clamp down and hun­ker down in si­lence. I’ve seen it a thou­sand times. Am­gen, though, just took the bug-out shuf­fle to a whole new ex­treme. Their kid­ney dis­ease drug Parsabiv (etel­cal­ce­tide) was cheered through a big Phase III pro­gram, leav­ing every­one wait­ing for the in­evitable ap­proval. But this week we learned that it was re­ject­ed by the FDA. Why? Am­gen won’t say, or even hint at it. Those pa­tients they re­cruit­ed to test the drug for the good of all (and to pro­tect a key rev­enue source for Am­gen)? Well, they don’t need to know ei­ther. Frankly, the whole thing dis­gusts me. Want to know why Big Bio­phar­ma has such a seedy rep? It’s shady events like this that put it in­to per­spec­tive. To Am­gen: At least try to hold your­selves ac­count­able and ex­plain what hap­pened here hon­est­ly. Shame on Am­gen CEO Robert Brad­way and R&D di­rec­tor Sean Harp­er for al­low­ing it to hap­pen. You need to do bet­ter. Much, much bet­ter.  There are pub­lic re­spon­si­bil­i­ties in­volved in drug de­vel­op­ment, and you’re shirk­ing them.

The mob is back, will the in­dus­try ever ef­fec­tive­ly tack­le the drug pric­ing con­tro­ver­sy?

It’s quite clear now that if there’s one is­sue that is guar­an­teed to trig­ger a fu­ri­ous pub­lic out­cry, it’s price goug­ing on old prod­ucts. Mar­tin Shkre­li, Valeant, now My­lan have all proved that. Buy­ing old prod­ucts and then march­ing up the price for a wind­fall prof­it can in­cite a dig­i­tal lynch mob. And one day the pitch­forks won’t stay fo­cused on just one com­pa­ny. They’ll come for every­one, and it will not be ig­nored by the po­lit­i­cal elite. Be­cause the fact is that the ma­jors have gen­er­al­ly adopt­ed this strat­e­gy. The price in­creas­es may be a more mod­est 100% to 200%, but the con­tro­ver­sy they’re invit­ing is clear. I’ve said it be­fore, and I’ll say it again, high prices on new, sig­nif­i­cant ther­a­pies are jus­ti­fied and need­ed to sus­tain biotech and pro­vide the next wave of treat­ments. It’s time, as some­one I know al­lud­ed to re­cent­ly, for the lead­ers in the in­dus­try to stop fight­ing fires and fig­ure out how to get rid of the match­es. Or it may be your biotech house that burns next. And biotech isn’t the bad guy in this dra­ma.

 The $14B Medi­va­tion buy­out of­fers an in­dus­try-wide lift af­ter a tough H1.

It wasn’t As­traZeneca or Al­ler­gan, but Pfiz­er did fi­nal­ly get a buy­out done with the Medi­va­tion ac­qui­si­tion. Yes, they prob­a­bly paid too much. But Medi­va­tion is the kind of com­pa­ny that makes a good tar­get for a deal: Small pipeline, mar­ket­ed prod­uct, easy to bolt on with­out dis­rupt­ing big, es­tab­lished R&D groups. It was good to see that there are buy­ers will­ing to pay a pre­mi­um for buy­outs like this. Score one for biotech — and star deal­mak­er David Hung — af­ter an ug­ly first half of the year. Frankly, we all ben­e­fit by a big score like this.

Sanofi comes up short, again.

Sanofi knew it need­ed the Medi­va­tion buy­out when CEO Olivi­er Brandi­court de­cid­ed to go af­ter it as his first big growth move. It failed, which isn’t all that sur­pris­ing giv­en its track record of the past few years. Where Sanofi suc­ceeds is through its col­lab­o­ra­tions. In­ter­nal R&D re­mains a dis­ap­point­ment. As a top 10 glob­al R&D op­er­a­tion, Sanofi has to start prov­ing that it can ac­quire late-stage as­sets and get them over the goal line alone. Maybe next time they won’t be left at the al­tar. There’s a lot rid­ing on a turn­around.

What a great week for in­no­va­tion lovers in biotech.

I start­ed off the week with a piece on Ax­el Bou­chon, the point man at Bay­er for start­ing a whole port­fo­lio of am­bi­tious new biotech ven­tures. Case­bia will help de­fine that new group with its 5-year, $335 mil­lion re­search bud­get and a new home on Kendall Square. Then there were sto­ries on Kr­ish­na Yesh­want’s strat­e­gy at GV (Google Ven­tures) and a new ven­ture from some young en­tre­pre­neurs fresh out of Brown. To help wrap the week, De­nali’s ex­ec­u­tive crew took some time to re­view Chap­ter Two at the neu­rode­gen­er­a­tion start­up, which has now raised close to $350 mil­lion as it preps its first clin­i­cal tri­al. I love the for­ward-look­ing think­ing. A mix of big and lit­tle play­ers al­so of­fers some per­spec­tive on where we are now in biotech; a no-longer-young in­dus­try in con­stant need of new ideas and new ven­tures. And they keep com­ing.

Ike the mouse is ag­ing R&D’s new mas­cot.

Or, he would be if he wasn’t dead. Ike lived to the ripe old age of 1,400 days, 140 years in hu­man longevi­ty. Matt Kae­ber­lein and his team of in­ves­ti­ga­tors in Seat­tle ac­com­plished this with ra­pamycin, a dan­ger­ous drug that no one needs to think of as the spring of youth. But there are path­ways and mech­a­nisms in­volved here that will help in­spire the next round of re­search in­to hu­man longevi­ty and healthy liv­ing. I trig­gered quite a dust-up on Twit­ter with com­ments like that. The FDA has to get in line (no easy task!)  and there’s more to think about than ex­tend­ing our lives by hun­dreds of years. Get­ting 90 good years would be a great start. Make no mis­take, ag­ing R&D is on its way to be­com­ing a big field in biotech. Things take time in this world, though. So don’t look for any overnight rev­o­lu­tions.

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.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Boost­er bo­nan­za: FDA en­dors­es 'mix-and-match' scheme, and Mod­er­na and J&J too

The FDA late Wednesday signed off on authorizing the use of heterologous — or what FDA calls a “mix and match” of a primary vaccine series and different booster doses — for all currently available Covid-19 vaccines, in addition to separately authorizing Moderna and J&J boosters.

On the mix-and-match approach, which FDA officials insisted isn’t too confusing in a press conference, the agency offered the example of an 18-year-old who received the J&J shot at least two months ago and may now receive a single booster of the J&J, a half dose of the Moderna, or the Pfizer-BioNTech booster.

David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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