Buzz: This lit­tle biotech has so much go­ing against it, some­body is go­ing to want to buy it. Right?

It’s the week af­ter JP­Mor­gan was dom­i­nat­ed by M&A news, so let’s get start­ed on the rest of the year by talk­ing about which biotechs are be­ing groomed for an ac­qui­si­tion.

Com­pa­ny num­ber 1 fits the bill per­fect­ly.

Alder $AL­DR has a lot go­ing against it. The biotech has se­cured a last-place fin­ish for it­self among the CGRP mi­graine play­ers af­ter stum­bling over its de­vel­op­ment sched­ule for eptinezum­ab and ex­e­cut­ing a man­age­ment shake­up. It’s es­sen­tial­ly a one-drug com­pa­ny, with a tiny staff go­ing up against mar­ket­ing gi­ants like Am­gen and No­var­tis and Eli Lil­ly and Te­va. Even if it wins an ap­proval for its late-stage drug and goes on to launch in 2020, how does it ex­pect to sell the ther­a­py?

Leerink an­a­lyst Ge­of­frey Porges an­swers each neg­a­tive with a pos­i­tive, and then ham­mers down the “for sale” sign on the vir­tu­al front lawn.

Ge­of­frey Porges

That last place fin­ish? Hey, those big com­pa­nies can give away drugs and es­tab­lish what he be­lieves will be a mega block­buster mar­ket. Alder has the on­ly IV ther­a­py in the bunch, has the po­ten­tial to ex­e­cute a great part­ner­ship or would make a great bolt-on — af­ter the ap­pro­pri­ate mark-up to a bat­tered share price. The mar­ket cap is at $742 mil­lion this morn­ing, mak­ing this a small por­tion of an M&A meal for some like­ly buy­er.

Porges all but so­lic­its a bid from Bio­gen, which needs to do some deals, not­ing how ep­ti fits in well to a neu­ro­science port­fo­lio. He notes:

Alder is now en­gaged in ac­tive part­ner­ship dis­cus­sions with much larg­er com­pa­nies about es­tab­lish­ing a com­mer­cial part­ner­ship for ep­ti in Eu­rope and oth­er ter­ri­to­ries. The com­pa­ny ac­knowl­edged re­cent­ly that they would pre­fer for such a part­ner­ship to be in place be­fore the MAA is filed in Eu­rope, which would mean mid to sec­ond half of 2019. Such part­ner­ship dis­cus­sions al­ways have the risk of blos­som­ing in­to ac­qui­si­tion dis­cus­sions, and we be­lieve this is par­tic­u­lar­ly like­ly in the case of Alder.

Risk? Well, that’s one way to put it.

Eli Lil­ly’s de­ci­sion to buy out Loxo’s or­gan-ag­nos­tic pipeline of can­cer drugs helped set the tone for the be­gin­ning of this year, fol­low­ing GSK’s Tesaro buy­out and the stun­ning an­nounce­ment that Bris­tol-My­ers is buy­ing Cel­gene for $74 bil­lion. The shop­ping spree has put M&A front and cen­ter at biotech. It’s no co­in­ci­dence that these moves are be­ing made af­ter the sec­tor got mauled in a bear mar­ket. Prices have come down on Nas­daq and no one can over­look the in­creas­ing pres­sure on phar­ma to bulk up on new prod­ucts if it can’t raise prices as it has in the past.

As a re­sult, the buzz fac­tor on M&A is go­ing to be in­tense. That was ev­i­dent af­ter ru­mors float­ed around that Pfiz­er was in­ter­est­ed in buy­ing Amarin, an­oth­er one-drug won­der that has spurred lots of spec­u­la­tion about the po­ten­tial of its drug Vas­cepa.

This con­ver­sa­tion is just get­ting start­ed. Look for lots more chat­ter ahead.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.


ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology


ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development


CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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SEC calls out lit­tle Ther­a­peu­tic­sMD for its in­sid­er con­tacts with an­a­lysts to boost share price, then halt rout

Back in May 2017, following an FDA rejection, TherapeuticsMD saw its share price plummet to the lowest levels in two years. The little Florida biotech eventually found its way back to the good side of regulators, scoring a curious OK a year later for its therapy preventing vaginal pain during sex. But the SEC is now accusing it of selectively disclosing nonpublic information in attempts to manipulate its stock.

In two instances in June and July of 2017, TherapeuticsMD allegedly violated the Regulation Fair Disclosure rule by sharing material information with certain sell-side analysts and not the public, resulting in a more favorable stock move than otherwise would be expected.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Therapists Marcela Ot'alora and Bruce Poulter are trained to conduct MDMA-assisted psychotherapy. In this reenactment, they demonstrate how they help guide and watch over a patient who is revisiting traumatic memories while under the influence of MDMA. (Photo: Multidisciplinary Association for Psychedelic Studies)

MD­MA, now in Phase III, shows promise as a PTSD treat­ment

The first time Lori Tipton tried MDMA, she was skeptical it would make a difference.

“I really was, at the beginning, very nervous,” Tipton said.

MDMA is the main ingredient in the club drug known as ecstasy or molly. But Tipton wasn’t taking pills sold on the street to get high. She was trying to treat her post-traumatic stress disorder by participating in a clinical trial.

After taking a dose of pure MDMA, Tipton lay in a quiet room with two specially trained psychotherapists. They sat next to her as she recalled some of her deepest traumas, such as discovering her mother’s body after Tipton’s mother killed two people and then herself in a murder-suicide.

Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin and UBS in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.