Pfiz­er im­press­es car­dio crowd with mor­tal­i­ty and hos­pi­tal­iza­tion rates for tafamidis in AT­TR-CM -- but Al­ny­lam quick­ly blasts back

Pfiz­er has scored the kind of po­ten­tial­ly game-chang­ing piv­otal da­ta for tafamidis in rare cas­es of transthyretin amy­loid car­diomy­opa­thy that an­a­lysts have been on the look­out for. And the phar­ma gi­ant is rolling out an ex­pand­ed ac­cess pro­gram for AT­TR-CM pa­tients now — just as a ri­val ther­a­py from Al­ny­lam is hit­ting the mar­ket for the first time.

Re­searchers to­day fol­lowed up pos­i­tive top-line da­ta with the news that tafamidis spurred a 30% drop in mor­tal­i­ty risk along with a 32% re­duc­tion in the risk of car­dio-re­lat­ed hos­pi­tal­iza­tion.  That’s good enough to win over a key crowd of top an­a­lysts, but you can bet that there will be plen­ty of ques­tions to­day as every­one hunts for the dev­il in the de­tail. And Al­ny­lam wast­ed no time in rais­ing doubts about the da­ta, which is like­ly go­ing to re­lieve in­vestors fret­ting over the com­pe­ti­tion.

Cred­it Su­isse an­a­lysts ear­li­er not­ed that “even a mod­est im­prove­ment in mor­tal­i­ty (10% to 15%) would be fa­vor­able.” Any­thing 20% to 25% could prove to be a game-chang­er, they added, in a field where a land­mark RNAi ther­a­py from Al­ny­lam is about to hit the mar­ket and an­oth­er — less at­trac­tive — ther­a­py from Akcea and Io­n­is is like­ly right be­hind it.

The da­ta al­so hit a sec­ondary on an im­prove­ment in the re­duc­tion of per­for­mance in the 6-minute walk test along with an im­prove­ment in qual­i­ty of life scores.

But some ques­tions re­main. And at first blush Al­ny­lam got a quick thumbs up for re­main­ing com­pet­i­tive as a mar­ket show­down looms be­tween the lead­ers in the field.

Al­ny­lam fol­lowed up by point­ing out that Pfiz­er looks weak­est where it looks strongest, with­out a sta­tis­ti­cal­ly sig­nif­i­cant read­out for hered­i­tary AT­TR. The com­pa­ny al­so spot­light­ed the pooled dose re­sults, an un­usu­al de­ci­sion by Pfiz­er. They added:

  • In APOL­LO, over 50% of patisir­an pa­tients showed IM­PROVE­MENT rel­a­tive to base­line on both mNIS+7 and Nor­folk.
  • In APOL­LO car­diac sub­pop­u­la­tion of hAT­TR pa­tients*, patisir­an re­sult­ed in:
    • De­crease from base­line (i.e., IM­PROVE­MENT) in NT-proB­NP lev­els (55% re­duc­tion for pati rel­a­tive to pbo)
    • 31.6% of pati pa­tients had de­crease change from base­line of NT-proB­NP ≥30% and ≥300 pg/mL, a key mor­tal­i­ty prog­nos­ti­ca­tor, at Month 18, where­as no pbo pa­tients had de­creas­es of this mag­ni­tude.

Per­haps most sig­nif­i­cant­ly, Al­ny­lam al­so be­lieves that this field is pri­mar­i­ly a growth op­por­tu­ni­ty, as new and bet­ter di­ag­noses iden­ti­fy a grow­ing group of pa­tients for both. In that sce­nario, pa­tients and physi­cians can make their own choic­es as all the biotechs ben­e­fit.

You can get more de­tails in the study pub­lished in the New Eng­land Jour­nal of Med­i­cine.

In­vestors seem fair­ly hap­py ini­tial­ly with both sides. Al­nyam shares jumped 16% by the end of the day — a re­lief ral­ly — while Pfiz­er stock end­ed down a cou­ple of points.

Bren­da Coop­er­stone

Re­searchers of­fered pooled da­ta for two dos­es of the drug — an 80 mg and 20 mg reg­i­men — rather than break­ing the re­sults out in­to dosage groups, leav­ing it un­like­ly that they saw a clear dose re­sponse. An­a­lysts will be fol­low­ing up Pfiz­er’s state­ment to­day with more ques­tions on how the ther­a­py worked. But it’s clear that Pfiz­er will be claim­ing an ad­van­tage here, pro­vid­ing one rea­son for CEO Ian Read’s re­cent vote of con­fi­dence that the com­pa­ny’s late-stage pipeline can pro­vide the big drugs it needs to keep rev­enue on the up­swing.

The drug is al­ready armed with both a break­through ther­a­py des­ig­na­tion as well as a Saki­gake ti­tle from Japan­ese reg­u­la­tors. Look for some quick mar­ket­ing ap­pli­ca­tions and an ag­gres­sive roll­out if the da­ta hold up.

What we al­so didn’t get im­me­di­ate­ly was much de­tail on safe­ty da­ta. The drug arm and place­bo had a “com­pa­ra­ble” safe­ty pro­file, the com­pa­ny re­port­ed.

Cred­it Su­isse has es­ti­mat­ed peak sales at $600 mil­lion, with a shot at more un­der cer­tain cir­cum­stances. The big ques­tion now is how much Al­ny­lam — whose drug On­pat­tro was ap­proved for TTR polyneu­ropthay — might be af­fect­ed by the com­pe­ti­tion. Ac­cord­ing to the an­a­lysts:

Though the in­di­ca­tions may be dif­fer­ent, the prod­ucts will like­ly com­pete, as physi­cians we spoke with in­di­cat­ed that many pa­tients with car­diomy­opa­thy tend to al­so have polyneu­ropa­thy and vice ver­sa. A few physi­cians be­lieve Al­ny­lam’s prod­uct is the safer prod­uct and will use patisir­an to treat car­diomy­opa­thy while get­ting re­im­burse­ment for polyneu­ropa­thy. Oth­er physi­cians ex­pect to pri­mar­i­ly pre­scribe tafamidis if the out­come da­ta are clin­i­cal­ly mean­ing­ful.

Pfiz­er, though, al­so faces a chal­lenge in get­ting physi­cians to do a much bet­ter job at di­ag­nos­ing TTR-car­diomy­opa­thy. But their de­ci­sion to be­gin a wide-open ex­pand­ed ac­cess pro­gram is a clear shot over Al­ny­lam’s bow. The drug is al­ready ap­proved as Vyn­daqel and on the mar­ket to treat fa­mil­ial amy­loid polyneu­ropa­thy.

Their mar­ket ri­val­ry starts to­day.

Al­so af­fect­ed by to­day’s an­nounce­ment is Ei­dos Ther­a­peu­tics $EI­DX, which re­cent­ly went pub­lic as it pur­sued its own work in the field.

“We be­lieve the AT­TR-ACT study find­ings bring us a sig­nif­i­cant step clos­er to our goal of pro­vid­ing an ur­gent­ly need­ed ther­a­py for a se­ri­ous and of­ten fa­tal dis­ease,” said Bren­da Coop­er­stone, Pfiz­er’s chief de­vel­op­ment of­fi­cer for rare dis­ease. “We look for­ward to con­tin­u­ing dis­cus­sions with glob­al reg­u­la­to­ry au­thor­i­ties about the po­ten­tial of tafamidis as a treat­ment op­tion for peo­ple liv­ing with AT­TR-CM.” 

Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

Daniel O'Day [via AP Images]

UP­DAT­ED: Gilead un­leash­es a $5B late-stage cash al­liance with Gala­pa­gos — lay­ing out O'­Day's R&D strat­e­gy

Daniel O’Day is executing his first major development deal since taking over as CEO of Gilead $GILD. And he’s going in deep to ally himself with a longstanding partner.

O’Day announced today that he is spending $5 billion in cash to add new late-stage drugs to Gilead’s pipeline, picking up rights to Galapagos’ $GLPG Phase III IPF drug GLPG1690 alongside adoption of the biotech’s Phase IIb drug GLPG1972 for osteoarthritis. And Gilead is also putting billions more on the table for milestones, gaining options for everything else in Galapagos’ pipeline, with a shot at all rights outside of Europe.

Altogether, Gilead is gaining rights to 6 clinical-stage assets, 20 preclinical programs and everything else being hatched in translation.

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Alk­er­mes adds bipo­lar de­pres­sion to its FDA wish­list; Con­go con­firms first Ebo­la case in large city

→ An ever-ambitious Alkermes $ALKS team plans to add bipolar depression to its list of conditions for ALKS-3831, which it plans to pitch to the FDA in Q4. Alkermes says they were persuaded to add bipolar depression after a pre-NDA meeting with the agency, which came about 7 months after the biotech reported positive data for schizophrenia. The drug is a combo using olanzapine/samidorphan, which they hope will be shown to be as effective as olanzapine without the substantial increase in the risk of weight gain.

Pe­ter Kolchin­sky and Raj Shah raise a $300M fund de­vot­ed to biotech star­tups

Peter Kolchinsky and Raj Shah have another $300 million-plus to play with on the biotech venture side of their investment business. 

The two announced Monday morning that they’ve put together their first pure-play venture fund at RA Capital Management, which has been known to bet on just about every angle in healthcare investing — from rounds to follow-on investments at public companies. This new fund of theirs arrives well into a go-go era of new startup financing, with a particular focus on building new biotechs.

Boehringer buys Swiss biotech in its lat­est M&A deal, go­ing the next-gen can­cer vac­cine route

Boehringer Ingelheim has snapped up a Swiss biotech startup and added their group as a new platform for the oncology pipeline. 

The German biopharma company has bagged Geneva-based AMAL Therapeutics, paying out an unspecified upfront in a $358 million deal — cash, milestones and everything else, all in. Plus there’s 100 million euros on the line for commercial milestones.

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Ab­b­Vie beefs up the on­col­o­gy pipeline, bag­ging an up­start STING play­er with its own unique ap­proach

AbbVie isn’t letting its $63 billion buyout of Allergan stop its M&A/deals team from continuing their work.

Monday morning we learned that the pharma giant is snapping up tiny Mavupharma out of Seattle, a Frazier-backed startup that has its own unique take on STING — which is on the threshold of their first clinical trial.

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Hal Barron [File photo]

Hal Bar­ron's team at GSK scores a win with pos­i­tive Ze­ju­la PhI­II front­line study — now comes the hard part

Score one for Hal Barron and the new R&D team steering GlaxoSmithKline’s pipeline.

The pharma giant reported this morning that its recently acquired PARP, Zejula (niraparib), hit the primary endpoint on progression-free survival in a frontline maintenance setting for women suffering ovarian cancer — following chemo and regardless of their BRCA status.

GSK bet $5 billion on the Tesaro buyout primarily to get this drug, drawing the shaking heads of biopharma. Why pay a big premium for a drug like this when AstraZeneca was going from strength to strength with Lynparza, ran the argument, having won a hugely important accelerated approval to jump out ahead — way ahead — of the rest of the PARP players? Lynparza — now co-owned by a powerhouse cancer team at Merck — won the first approval in frontline maintenance in ovarian cancer.

Billing it­self as the first AI biotech to launch hu­man tri­als, Re­cur­sion adds $121M C round

Billing itself as the first AI biotech with programs in the clinic, Salt Lake City-based Recursion now has a $121 million bankroll to start gathering human data to see if it’s on the right track. 

“We’re trying to build this discovery engine,” Recursion CEO Chris Gibson tells me ahead of the C round news. “We now have the first two programs in the clinic.” And that, he adds, qualifies as a first for any AI establishment “that actually have something in the clinic.”

FDA bats back As­traZeneca's SGLT di­a­betes drug for Type 1 di­a­betes — block­ing a class on safe­ty fears

The FDA has just fired its latest salvo at the SGLT class of diabetes drugs, blowing up some commercial opportunity at AstraZeneca as part of the collateral damage.

The pharma giant reported early Monday that the FDA has rejected its blockbuster drug Farxiga for Type 1 diabetes that can’t be controlled by insulin. And while the pharma giant maintained its usual grim silence in the face of a setback, this one should be easy to interpret.