Don't let a slump in bio­phar­ma deals get you down — yet; The con­tro­ver­sy on drug prices is tak­ing a toll

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

Hey, what about that big up­swing pre­dict­ed for bio­phar­ma M&A?

Deal­mak­ers in bio­phar­ma are end­ing April with a con­sid­er­able amount of grum­bling. This was the year that M&A was sup­posed to take off, with a new pres­i­dent bull­ish­ly as­sert­ing plans to re­form tax­es in a way that would free up bil­lions in Big Phar­ma cash held over­seas.

Didn’t hap­pen. Don­ald Trump is so far bogged down on Oba­macare re­form and not able to de­liv­er a much bal­ly­hooed tax re­form pledge — for now.

“Most peo­ple would say they are dis­ap­point­ed with the pace of M&A,” Ge­of­frey Hsu, a part­ner at the deal-ori­ent­ed Or­biMed Ad­vi­sors, told Bloomberg. “Frankly, we were ex­pect­ing more to hap­pen by this time.”

The newswire ser­vice con­clud­ed that Q1 deals were down 13% cash-wise, com­pared to the same pe­ri­od a year ago.

True, deal­mak­ers are hard to please. It’s a feast or famine world in M&A, and lean pick­ings cause a lot of dis­sat­is­fac­tion.

The year, though, is a long way from over. Don’t let a Q1 re­port make you too un­hap­py. Con­cerned, yes. Un­hap­py, no.

Drug­mak­ers face a turn­ing point on pric­ing — putting the fo­cus where it be­longs

An­oth­er big trend that is draw­ing con­sid­er­able at­ten­tion these days is the im­mense pres­sure be­ing put on drug mak­ers to rein in prices. Pay­ers in the US are get­ting bet­ter at ap­ply­ing pres­sure right on the most sen­si­tive parts of the in­dus­try, and Pres­i­dent Trump start­ed his ad­min­is­tra­tion stomp­ing on the in­dus­try’s rep for stick­er shock.

Cred­it Su­isse put its thumb right on the pulse, though, with a new re­port this week show­ing just how im­por­tant an­nu­al price hikes are to the big play­ers. Their an­a­lysts con­clude:

Ar­guably, this is the most im­por­tant is­sue for a Phar­ma in­vestor to­day. De­spite pub­lic scruti­ny, we es­ti­mate US net price ris­es con­tributed c$8.7bn in 2016 to net in­come, 100% of sec­tor EPS growth. US net price growth was >100% of Bio­gen, Lil­ly, and Ab­b­Vie’s to­tal net in­come growth. Bio­Marin, Gilead, No­vo and Re­gen­eron were the least re­liant on US net price ris­es.

In this brave new world, on­ly the in­no­v­a­tive can thrive. And that means more part­ner­ing with biotech — the true source of in­no­va­tion in the drug world. I sus­pect we’ll see a grow­ing scram­ble for the best ex­per­i­men­tal drugs lat­er in the year. And that means val­u­a­tions should con­tin­ue to point north.

A con­tro­ver­sial Marathon is mar­gin­al­ized

We just learned that Marathon has been es­sen­tial­ly forced out at PhRMA, fol­low­ing a res­ig­na­tion by Mallinck­rodt — an­oth­er de­vel­op­er that has been dogged by a drug pric­ing scan­dal.

Ku­dos to PhRMA, which has been mak­ing life more un­com­fort­able for the com­pa­nies that set the pace for Mar­tin Shkre­li. Far too many com­pa­nies have found it easy to get cheap drugs and jack up the price by as­tro­nom­i­cal amounts.

In this new bio­phar­ma world we’re liv­ing in, that kind of con­duct won’t fly. You can’t be in the club now un­less you’re dead se­ri­ous about in­vest­ing in im­por­tant new ther­a­pies. That’s not enough, but it is a healthy start. And PTC Ther­a­peu­tics may want to think over their strat­e­gy one more time.

You have a pho­to op com­ing up Sat­ur­day morn­ing. Don’t miss it.

To­mor­row Mass­Bio and some of the top lead­ers of the Boston area biotech sec­tor want to cre­ate a mo­ment for every­one to reg­is­ter their op­po­si­tion to some of the re­cent moves from Pres­i­dent Trump on im­mi­gra­tion, H1B visas and NIH fund­ing.

It has come to no sur­prise to me that the vast ma­jor­i­ty of the in­dus­try hates what it’s been see­ing over the last few months. Biotech thrives when it can re­cruit the best and the bright­est from all over the world, and the US is home to the most dy­nam­ic drug de­vel­op­ment sec­tor in the world be­cause of it.

NIH fund­ing, mean­while, cre­ates the sci­en­tif­ic foun­da­tion that bio­phar­ma rests on. Cut­ting fund­ing sig­nif­i­cant­ly now would mean lay­ing waste to a gen­er­a­tion of young sci­en­tists look­ing to make their mark.

So at 10am on Sat­ur­day every­one is be­ing asked to give up some of their free time and join the crowd on Kendall Square for an aer­i­al shot to cap­ture a pic­ture of the in­dus­try’s sol­i­dar­i­ty on this is­sue. Too bad it’s not a week­day, when most peo­ple would on­ly have to walk over from their of­fices. But that might have stopped traf­fic.

A gen­er­al view of the bronze and glass pub­lic art sculp­ture “Nerve Cen­ter” by artist Chris Williams in Kendall Square on Ju­ly 1, 2016 in Cam­bridge, Mass­a­chu­setts. Pho­to by Paul Marot­ta/Get­ty Im­ages


De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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Large advertisements for the drug Vivitrol decorate the walls of Grand Central Station on June 15, 2017 in New York City. (Photo: Andrew Lichtenstein via Getty)

FDA slaps down Alk­er­mes for mis­lead­ing Viv­it­rol ads — don't for­get vul­ner­a­bil­i­ty to opi­oid over­dose

The ads piqued interest as soon as they started appearing in 2016: at Grand Central Station, on the Red Line in Cambridge, and on a billboard off the New Jersey Turnpike. All showed a young person, generally with his or her arms crossed, and the question, “what is Vivitrol?”

Vivitrol’s maker, Alkermes, was in the midst of a marketing and lobbying campaign to promote the anti-opioid addiction drug — a campaign that would face significant backlash for tarnishing competitors despite little evidence for Vivitrol’s superiority.

FDA in-house re­view spot­lights an is­sue with one of Hori­zon's end­points but notes ef­fi­ca­cy for lead drug

The FDA in-house review highlights a disagreement of investigators’ use of a key endpoint by Horizon Pharma in the late-stage trial for the top drug in its pipeline, but largely agreed that the antibody was effective.

Horizon submitted a BLA for thyroid eye disease (TED) drug teprotumumab in March, less than two years after they bought the drug (and the rest of a division) from Narrow River for $145 million upfront. With breakthrough status, priority review, orphan designation and in-house sales projections of up to $750 million, the one-time Roche reject became the marquee pipeline asset for a company that’s developed some of the world’s most expensive drugs.

Seat­tle Ge­net­ics de­tails pos­i­tive OS and PFS da­ta for tu­ca­tinib in breast can­cer

Seattle Genetics $SGEN is showing off more positive data around tucatinib, its pivotal-stage drug for HER2 positive breast cancer.

A month after hearing about solidly upbeat hazard ratios, we learned today that the estimated progression-free survival rate at one year was 33% in the tucatinib arm compared to 12% for patients taking trastuzumab and capecitabine alone.

Median PFS was 7.8 months (95% CI: 7.5, 9.6) in the tucatinib arm, compared to 5.6 months (95% CI: 4.2, 7.1) in the control arm.

Bat­tered, cash hun­gry In­tec feels the burn of No­var­tis re­jec­tion

It’s a case of some bad timing for Intec.

Just when a key trial testing the company’s Accordion drug delivery tech imploded in Parkinson’s disease, they handed Novartis data from a successful PK study of a custom Accordion pill engineered to deliver a Novartis compound to entice the Swiss drugmaker into signing a licensing agreement.

Novartis said thanks, but no thanks.

For the cash-strapped Israeli drug developer, the failure to clinch the deal marks a big blow. As of the third quarter, the company has $15.7 million in cash and equivalents, which HC Wainwright analysts estimate will keep the lights on into mid-2020.

Bris­tol-My­ers shows off a low-pro­file AML con­tender it gained from Cel­gene buy­out — and they’re tak­ing it straight to the FDA

Bristol-Myers Squibb reaped an enormous pipeline with its much-criticized $64 billion megadeal to buy Celgene. And it got a few hidden gems in the deal.

One of those gems was brought out for display on Tuesday, with a late-breaker at ASH on CC-486, which is now being prepped for regulatory filings at the FDA and elsewhere.

Celgene top-lined the positive results in a maintenance setting for acute myeloid leukemia a few months ago, but at ASH investigators pulled back the curtains on the all-important data they believe will give them an advantage in the commercial wars to come.

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De­sert­ed by Astel­las and Mer­ck, lit­tle Cor­re­vio still can't win over FDA pan­el con­cerned with its AFib drug's safe­ty

When the FDA spurned Astellas’ pitch for atrial fibrillation drug vernakalant in 2008, regulators made it abundantly clear that it wasn’t the efficacy they had a problem with — two Phase III trials had shown the drug successfully restored 52% of patients’ heartbeat from irregular to normal — but the cardio safety issues for a drug that was to compete with well established, low-risk options. One licensing deal, one clinical hold and several studies later, the chances of approval aren’t looking any better.