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.” 

Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Nick Galakatos, Blackstone global head of life sciences

Nick Galakatos and the Black­stone team now have a record $4.6B to in­vest in bio­phar­ma, with a big fo­cus on push­ing com­pa­nies over the top

Nick Galakatos and his team at Blackstone Life Sciences have seen their biggest opportunities swell up in mostly established players who don’t have all the money they need to accomplish everything on the to-do list. And right now, with the industry booming, that’s a long list with some hefty needs.

The Blackstone team has neatly tied up the largest private fund ever raised in life sciences for making big dreams come true in biopharma. Late Thursday, Blackstone put out word that they had closed their highly anticipated fund with the projected $4.6 billion all in.

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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Gilead boasts of pos­i­tive remde­sivir da­ta on mor­tal­i­ty — but their analy­sis pro­vokes the skep­tics

Gilead is surging again off data that suggest its antiviral remdesivir might improve survival.

The new data come from an analysis Gilead conducted comparing the death rate and recovery time of patients in one of its remdesivir trials to a group of 800 patients “with similar baseline characteristics and disease severity” who received only standard-of-care around the same time. The result, they said, suggested that patients who received remdesivir had a 62% better chance at surviving than those who did not.

Andrew Kruegel, Kures president and co-founder (Columbia Tech Ventures via Vimeo)

Af­ter psilo­cy­bin and ke­t­a­mine, a new biotech comes along de­vel­op­ing a drug Scott Got­tlieb fought

Andrew Kruegel was six years into his chemistry work at Columbia University, when, one day in August 2016, he learned he might have only 30 days before the government made him destroy his research.

Kruegel had been studying kratom, a leaf long used in Southeast Asia as a stimulant or for pain. It had opioid-like properties, he found, but seemed to offer pain relief without the addictive potential or respiratory side effects of traditional opioids — a riddle that might help illuminate how human opioid receptors work.

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The home run count: The $100M+ mega-round boom in biotech in­spired a $7.3B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMed)

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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Hal Barron, GSK

Win or lose on the mar­ket­ing OK, the FDA just gunned down GSK’s bright hopes for their BC­MA ther­a­py

The FDA’s ODAC — the Oncologic Drugs Advisory Committee — has a well-known bias in favor of adding new cancer drugs to the market, even if efficacy is at best marginal and serious safety issues demand careful management.

Doctors want as many arrows in their quiver as they can get. And when patients are dying after failing multiple drugs, why not give it a go one more time?

GlaxoSmithKline, though, is about to test out how their new BCMA antibody drug conjugate belantamab mafodotin can do after being mauled in an in-house FDA review, ahead of the Tuesday expert panel discussion. Even if the agency goes ahead with an expected green light, this drug will likely be constrained to a small niche — icing any plans they may have for making waves in oncology anytime soon.

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