Pos­i­tive oc­u­lar im­plant da­ta bring Al­ler­gan one step clos­er to NDA; Vivek Ra­maswamy spawns an­oth­er 'Van­t'

→  In 2016, Brent Saun­der’s Al­ler­gan $AGN or­ches­trat­ed a string of deals af­ter its $160 bil­lion merg­er with Pfiz­er fell through thanks to Oba­ma-era tax rules, in­clud­ing For­Sight VI­SION5 and its glau­co­ma tech­nol­o­gy for $95 mil­lion up­front. But Al­ler­gan al­so has its own in­ter­nal­ly de­vel­oped oph­thal­mol­o­gy pro­gram, its oc­u­lar im­plant bi­mato­prost SR is de­signed to help low­er in­traoc­u­lar pres­sure for at least 4 months in pa­tients strug­gle with eye drops. On Mon­day, Al­ler­gan said a sec­ond Phase III study demon­strat­ed pos­i­tive da­ta adding to the re­sults of a late-stage study re­port­ed in June 2018. An NDA is slat­ed to be sub­mit­ted in the sec­ond half of 2019.

→ Vivek Ra­maswamy has an­nounced the launch of yet an­oth­er “Vant.” This one is called Aly­vant, and its tech is de­signed to help phar­ma­ceu­ti­cal com­pa­nies in­te­grate mar­ket re­search, sales force op­ti­miza­tion, and dig­i­tal en­gage­ment in­to a sin­gle plat­form to con­nect pa­tients and physi­cians with rel­e­vant med­i­cines cost-ef­fec­tive­ly. The com­pa­ny, led by in­dus­try vet­er­an Gillian Can­non, al­ready has an agree­ment with an un­named phar­ma­ceu­ti­cal part­ner to co-pro­mote three ap­proved brand­ed drugs. “This tech­nol­o­gy plat­form al­lows us to en­gage new mar­ket seg­ments that are un­der­served by tra­di­tion­al phar­ma com­mer­cial mod­els, while al­so re­duc­ing the need for ex­pen­sive and waste­ful mass-mar­ket DTC cam­paigns that in­flate the cost of de­liv­er­ing new med­i­cines to pa­tients,” said Ben­jamin Zim­mer, pres­i­dent of par­ent com­pa­ny Roivant Health, in a state­ment.

→ You may re­mem­ber Myr­i­ad Ge­net­ics from its le­gal fight with Tesaro over the ne­ces­si­ty of its di­ag­nos­tic as a com­pan­ion to the biotech’s PARP in­hibitor. But as in­ves­tiga­tive jour­nal­ist Rod­dy Boyd (of Valeant fame) writes in his lat­est ex­posé for South­ern In­ves­tiga­tive Re­port­ing Foun­da­tion, it was not the com­pa­ny’s on­ly ge­net­ic test that proved fu­tile.

Boyd’s sev­en-month in­ves­ti­ga­tion cen­tered around Gen­e­Sight, a di­ag­nos­tic that promis­es to guide a psy­chi­a­trist in se­lect­ing an an­ti­de­pres­sant by an­a­lyz­ing a pa­tient’s ge­net­ic pro­file. Put sim­ply: It didn’t work — failed the pri­ma­ry end­point and 23 out of 25 sec­ondary ones — but the ex­ecs spinned it as if it did, kick­ing stock prices up. It was, in Boyd’s words, a demon­stra­tion of the “com­pa­ny’s abil­i­ty to spin ever more fan­tas­ti­cal non­sense — and to bril­liant­ly nav­i­gate the opaque line be­tween re­quired dis­clo­sure and mis­di­rec­tion.”

But that’s not all. In a sep­a­rate sto­ry, Boyd re­ports that in 2013, Myr­i­ad Ge­net­ics avoid­ed a cut to the re­im­burse­ment lev­el by the Cen­ters for Medicare and Med­ic­aid Ser­vices thanks to an ag­gres­sive lob­by­ing cam­paign by a staffer work­ing for Sen. Or­rin Hatch. The Salt Lake City-based com­pa­ny’s close re­la­tion­ship with the long-serv­ing Utah sen­a­tor, he wrote, helped Myr­i­ad win in six months a reg­u­la­to­ry break that could have tak­en years. Add that to an ap­par­ent billing loop­hole that Myr­i­ad had seized for its breast can­cer-re­lat­ed gene test, and you get mil­lions in added rev­enue at a time in­creased com­pe­ti­tion is dri­ving prices down for ge­net­ic test­ing, ac­cord­ing to Boyd.

(Ed­i­tor’s note: Af­ter this item ap­peared Mon­day, a spokesper­son for Myr­i­ad Ge­net­ics con­tact­ed End­points News and of­fered this in re­sponse: “Myr­i­ad dis­putes (the) ac­cu­ra­cy of Mr. Boyd’s ar­ti­cles.”)

→ Fre­quen­cy Ther­a­peu­tics — which is fo­cused on cre­at­ing a new class of drugs based on small mol­e­cule stim­u­la­tion of dor­mant stem cells with­in the body to re­pair dis­eased or dam­aged tis­sue — closed $42 mil­lion in Se­ries B round, bring­ing funds raised in to­tal to $87 mil­lion. The cap­i­tal will be used to fi­nance the de­vel­op­ment of its ex­per­i­men­tal drug, FX-322, for hear­ing re­gen­er­a­tion. Top-line re­sults from an on­go­ing Phase 1/2 study are ex­pect­ed in the first half of 2019.

→ Blue­bird bio $BLUE has joined forces with In­hi­brx to co-de­vel­op CAR-T cell ther­a­pies us­ing In­hi­brx’s sin­gle do­main an­ti­body (sdAb) plat­form for mul­ti­ple can­cer tar­gets. In­hi­brx gets $7 mil­lion up­front, and is el­i­gi­ble to re­ceive un­spec­i­fied funds in mile­stones pay­ments.

→ Pfiz­er $PFE has tied up with Cy­toRe­a­son — a ma­chine learn­ing com­pa­ny for drug dis­cov­ery and de­vel­op­ment —for an undis­closed sum.Cy­toRe­a­son’s plat­form has the po­ten­tial to of­fer valu­able in­sights that may be ap­plied to our re­search in­to the hu­man im­mune sys­tem,” said Pfiz­er’s Michael Vin­cent, CSO for in­flam­ma­tion & im­munol­o­gy, in a state­ment.

→ Gala­pa­gos $GLPG is pair­ing up with pri­vate­ly held Fi­bro­cor to fo­cus on id­io­path­ic pul­monary fi­bro­sis and oth­er in­di­ca­tions. The two are work­ing to­geth­er on a small mol­e­cule in­hibitor pro­gram for an undis­closed tar­get, as part of the deal with undis­closed fi­nan­cial terms an­nounced on Fri­day.

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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UP­DAT­ED: The FDA sets a reg­u­la­to­ry speed record, pro­vid­ing a snap OK for Ver­tex's break­through triplet for cys­tic fi­bro­sis

The FDA has approved Vertex’s new triplet for cystic fibrosis at a record-setting speed.

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IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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That $335M JV Bay­er set up on CRISPR/Cas9? They’re let­ting the biotech part­ner car­ry on

Bayer committed $300 million to set up a joint venture on CRISPR/Cas9 tech with CRISPR Therapeutics $CRSP. But they’re handing off control now to the smaller biotech while retaining a couple of opt-ins for programs nearing an IND.

Bayer $BAY made much of the fact that they were going all-in on gene editing when they did their deal 3 years ago with CRISPR Therapeutics, which pitched $35 million in on their end. This was the cornerstone of their plan to set up new JVs that could make some serious leap forwards in hot new R&D spaces. Now CRISPR will have full management control of Casebia as they pursue programs in hemophilia, ophthalmology and autoimmune diseases.
Samarth Kulkarni, the CEO at CRISPR, made it sound like a natural progression.

J&J's block­buster Ste­lara wins US ap­proval for ul­cer­a­tive col­i­tis

J&J’s Stelara, which is set to be in the top ten list of blockbusters come 2025, is now cleared by the FDA for use in ulcerative colitis (UC), an inflammatory disease of the large intestine.

The biologic targets interleukin (IL)-12 and IL-23 cytokines, which are known to play a key role in inflammatory and immune responses. Stelara, which generated about $4.7 billion in the first nine months of 2019, is a key player in the crowded marketplace of drugs to treat autoimmune disorders such as psoriasis, rheumatoid arthritis and Crohn’s disease. AbbVie’s star therapy, Humira, continues to dominate, despite its looming patent cliff in the United States, while others including J&J’s $JNJ own anti-IL23 Tremfya, Lilly’s $LLY anti-IL-17 Taltz and AbbVie’s $ABBV recently approved anti-IL-23 antibody Skyrizi carve out a slice of market share.

Drug com­pa­nies reach $260M set­tle­ment just ahead of opi­oid tri­al; Oys­ter Point set terms for $85M IPO

→ Hours before the first federal opioid trial was set to begin, three drug distributors and an opioid manufacturer agreed to a $260 million agreement settlement, the Wall Street Journal was the first to report. The deal — which will see McKesson, Cardinal Health and AmerisourceBergen pay $215 million to Summit and Cuyahoga counties, and Teva deal out $35 million in cash and addiction treatments — does not resolve the pending, nationwide litigation that may result in a settlement worth upwards of $40 billion. Negotiators in that case, brought by 2,300 tribes, counties and cities nationwide and led by several states’ attorneys general, worked through much of Friday without success. Josh Stein, the attorney general for North Carolina, said they were trying to put together a $48 billion deal.

GSK of­floads two vac­cines in $1.1B deal as it works to re­vive the pipeline

GlaxoSmithKline is leaving the deep dark woods and its viruses behind.

GSK has agreed to divest its vaccines for rabies, RabAvert, and tick-born encephalitis vaccine, Encepur, to Bavarian Nordic, part of the company’s broader efforts to narrow its pipeline and focus on oncology and immunology.

The deal is worth up to nearly $1.1 billion, with a $336 million upfront payment. GSK acquired the vaccines from Novartis as part of an exchange for their late-stage oncology programs in 2015 under former chief Sir Andrew Witty.

Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrate their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of. The market leader here is Norditropin, a growth hormone from Novo Nordisk that uses the same basic ingredient as Genotropin, which the Danish company sells with a kid-friendly self-injectable pen. That would also present some big competition if the new therapy from Opko/Pfizer makes it to the market.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

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As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.