Split the pot? Ra­dius shows its os­teo­poro­sis cards in fi­nal block­buster match with Am­gen

Ra­dius Health $RDUS has tak­en an­oth­er big leap down the fi­nal stretch of its Phase III race with Am­gen $AMGN, post­ing Phase III re­sults that show abaloparatide’s clear clin­i­cal ben­e­fit over a place­bo in treat­ing os­teo­poro­sis. But while the da­ta pro­vide a fresh ar­gu­ment in fa­vor of its near-term ap­proval at the FDA and EMA, there’s still a big ques­tion left unan­swered: Just how com­pet­i­tive will this drug be against Am­gen’s Phase III ri­val, as well as Eli Lil­ly’s ag­ing For­teo? And mean­while, the roll up to the FDA is al­so light­ing a fresh fire un­der the heat­ed de­bate over the cost of drugs, both old and new.

First, the num­bers, pub­lished in the Jour­nal of the Amer­i­can Med­ical As­so­ci­a­tion. In a large Phase III tri­al, just four of 824 pa­tients tak­ing Ra­dius’s abaloparatide suf­fered a spinal frac­ture af­ter 18 months. In a place­bo arm, that po­ten­tial­ly dev­as­tat­ing event was ex­pe­ri­enced by 30 pa­tients—giv­ing the ex­per­i­men­tal drug a clear ad­van­tage. But in the For­teo arm with 818 pa­tients, there were 6 spinal frac­tures. That’s slight­ly worse, but the study wasn’t large enough to show a clear dif­fer­ence be­tween the stan­dard of care and the new drug.

Pa­tients on the Ra­dius Health drug al­so ex­pe­ri­enced im­prove­ments in bone min­er­al den­si­ty. In­ves­ti­ga­tors in the study con­cen­trat­ed on old­er women with a his­to­ry of frac­tures, the at-risk pop­u­la­tion that the drug is pri­mar­i­ly aimed at, though it is be­ing stud­ied in oth­er pop­u­la­tions as well.

Not in­clud­ed in this matchup is Am­gen’s ro­mosozum­ab, it’s own bone-build­ing drug that was filed for an ap­proval on Ju­ly 21. Am­gen is part­nered with UCB on the drug. Back in Feb­ru­ary, in­ves­ti­ga­tors not­ed that their Phase III study came out with a 73% re­duc­tion in the risk of frac­tures, which paled a bit in re­la­tion to Ra­dius’s 86% risk re­duc­tion. That sparked a de­bate among an­a­lysts whether Am­gen’s drug could dis­tin­guish it­self with a much eas­i­er dos­ing reg­i­men — once month­ly ver­sus once dai­ly.

An­a­lysts’ peak sales pro­jec­tions have been all over the map. Deutsche Bank last year pegged aba­lo’s peak at $1.1 bil­lion, though they be­lieved that ro­mo would come out on top as the bet­ter drug with a big­ger mar­ket share, de­pend­ing on how the gener­ics shake out.

While both Am­gen and Ra­dius stand a good chance of win­ning an ap­proval, com­mer­cial suc­cess is a com­plete­ly dif­fer­ent is­sue. As The New York Times re­ports, pa­tients are gen­er­al­ly start­ed on bis­pho­s­phanates like Fos­amax, which are old and cheap. But they’re al­so lim­it­ed, un­able to build bone the way For­teo and the two new drugs are de­signed to do.

Lil­ly, mean­while, has been rapid­ly jack­ing up the price of For­teo ahead of its loss of patent pro­tec­tion. The Times re­ports that the whole­sale price has soared to $3,100 a month, more than three times its price in 2010. Lil­ly has been in­creas­ing the price twice a year, for 6 years.

Now the ques­tion is whether the new drugs will cost as much or more than For­teo, which is gen­er­al­ly lim­it­ed to two years of ther­a­py due to fears about po­ten­tial can­cer risks. And in­sur­ers – who are like­ly to re­strict ac­cess to these drugs to pa­tients with the high­est risk fac­tors – have grown leery about cov­er­ing the Lil­ly drug, of­ten re­quir­ing big co-pays from pa­tients.

Dr. Do­lores Shoback

Dr. Do­lores Shoback, a pro­fes­sor of med­i­cine at the Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co, told the Times:

“The cost in the pri­vate sec­tor is get­ting pro­hib­i­tive. You have to hunt for a rea­son, find a very strong rea­son that is iron­clad,” to get an in­sur­er to ap­prove For­teo, she added. “We find it can be ex­treme­ly dif­fi­cult to get it cov­ered.”

Ra­dius may have just high­light­ed a clear path to an ap­proval. But po­si­tion­ing this drug to win the four-way com­mer­cial pok­er match ahead is far more prob­lem­at­ic.

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 costs in com­pa­ny-wide re­org

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy reveal tomorrow 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, 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.

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Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

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Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

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Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

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KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.