Watch out Al­ny­lam/Io­n­is: Pfiz­er’s pos­i­tive PhI­II tafamidis da­ta makes drug a new ri­val

Pfiz­er $PFE re­port­ed out some good news from a late-stage tri­al test­ing its drug tafamidis against a rare con­di­tion that leads to heart fail­ure. The pos­i­tive re­sults of the Phase III study have both Al­ny­lam $AL­NY and Io­n­is $IONS in­vestors squea­mish, as it could be com­pe­ti­tion for patisir­an and in­ot­ersen.

Bren­da Coop­er­stone

The Pfiz­er tri­al, called AT­TR-ACT, test­ed its drug tafamidis in pa­tients with transthyretin car­diomy­opa­thy, an in­her­it­ed con­di­tion in which pro­teins don’t fold in­to their nor­mal shapes cor­rect­ly. The mis­fold­ing makes them build up and cause dam­age to the nerves, heart, and oth­er or­gans. In the tri­al, the drug met its pri­ma­ry end­point, Pfiz­er said, and demon­strat­ed a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in the com­bi­na­tion of all-cause mor­tal­i­ty and fre­quen­cy of car­dio­vas­cu­lar-re­lat­ed hos­pi­tal­iza­tions com­pared to place­bo at 30 months.

Fol­low­ing news on the da­ta, Al­ny­lam stock fell 5%, po­ten­tial­ly on fear of an up­com­ing ri­val as the phar­ma com­pa­ny is al­so pur­su­ing a car­diomy­opa­thy ap­pli­ca­tion for patisir­an. And Io­n­is, which owns a ma­jor­i­ty stake in its spin­out Akcea (which is de­vel­op­ing in­ot­ersen for the same con­di­tion), al­so sank 5%.

Pfiz­er land­ed tafamidis when it ac­quired Fol­dRx in a buy­out back in 2010 as it pushed in­to the rare dis­ease field. Like oth­er phar­ma com­pa­nies, Pfiz­er was lured by the prospect of big re­turns for drugs that meet an ur­gent need in tiny pa­tient pop­u­la­tions.

Pfiz­er test­ed the drug in a rare neu­rode­gen­er­a­tive dis­ease called TTR-FAP, but got re­ject­ed by the FDA in 2012 af­ter try­ing to gain ap­proval with on­ly one tri­al as proof of the drug’s ef­fi­ca­cy. Tafamidis was ap­proved in 40 coun­tries — in­clud­ing the EU in 2011 — how­ev­er. It sells un­der the brand name Vyn­daquel.

In a state­ment ear­li­er this year, Pfiz­er said it’s still work­ing on a way for­ward in the US for tafamidis in TTR-FAP.

“As a leader in TTR amy­loi­do­sis, Pfiz­er Rare Dis­ease con­tin­ues to part­ner with the FDA re­gard­ing a po­ten­tial path to ap­proval for tafamidis for TTR-FAP, as we hope to achieve the ob­jec­tive of pro­vid­ing TTR-FAP pa­tients liv­ing in the Unit­ed States with the same treat­ment op­tion as those pa­tients liv­ing in many oth­er parts of the world.”

For now, tack­ling transthyretin car­diomy­opa­thy gives the drug a new av­enue for rev­enue gen­er­a­tion in the US. Bren­da Coop­er­stone, SVP and chief de­vel­op­ment of­fi­cer in Pfiz­er’s rare dis­ease unit, had this to say about the re­cent Phase III da­ta:

These topline re­sults are im­por­tant for peo­ple with transthyretin car­diomy­opa­thy and bring us one step clos­er to re­al­iz­ing the po­ten­tial for a new treat­ment for those in des­per­ate need. Pfiz­er Rare Dis­ease has been at the fore­front of im­prov­ing the un­der­stand­ing of transthyretin car­diomy­opa­thy, and we thank the pa­tients who par­tic­i­pat­ed in the tri­al and their fam­i­lies, as well as the physi­cians and in­ves­ti­ga­tion­al sites that con­tributed to this im­por­tant study. We look for­ward to shar­ing the de­tailed re­sults of the study with the car­dio­vas­cu­lar com­mu­ni­ty and dis­cussing these da­ta with health au­thor­i­ties to de­ter­mine an ap­pro­pri­ate reg­u­la­to­ry path for­ward.

 

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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#ES­MO20: Trodelvy da­ta show that Gilead­'s $21B buy­out may have been worth the big pre­mi­um

Gilead CEO Dan O’Day has been on a shopping spree. And while some analysts gawked at the biotech’s recent $21 billion Immunomedics buyout, new data released at virtual ESMO 2020 suggest the acquisition may have been worth the hefty price.

The deal, announced last weekend, will give California-based Gilead $GILD Trodelvy, which was recently approved for metastatic triple-negative breast cancer (mTNBC).

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#ES­MO20: As­traZeneca bur­nish­es Tagris­so's ad­ju­vant NSCLC pro­file with 'un­prece­dent­ed' re­duc­tion in brain mets. Can they win over skep­tics?

When AstraZeneca trumpeted “momentous” and “transformative” results for Tagrisso earlier this year at ASCO, some practitioners threw cold water on the ADAURA fervor. Sure, the disease-free survival data look good, but overall survival is the endpoint that matters when it comes to choosing adjuvant therapy for non-small cell lung cancer patients, the experts said.

The OS data still aren’t here, but AstraZeneca is back at ESMO to bolster their case with a look at brain metastasis data.

Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.

#ES­MO20: Alk­er­mes of­fers their first snap­shot of a ben­e­fit for their next-gen IL-2 drug. But why did 1 pa­tient starve to death?

Everyone in the cancer R&D arena is looking to build new franchises around better drugs and combos. And one busy pocket of that space is centered entirely on creating an IL-2 drug that can be as effective as the original without the toxicity that damned it to the sidelines.

Alkermes $ALKS formally tossed its hat into the ring of contenders at virtual ESMO today, highlighting the first glimpse of efficacy for their candidate, ALKS 4230, as both a monotherapy as well as in combination with Merck’s Keytruda.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

Pfiz­er match­es Mod­er­na with their full Covid-19 tri­al blue­print — As­traZeneca says it will un­veil its pro­to­col 'short­ly'

Yesterday, after sustained public pressure as Moderna released its Phase III Covid-19 trial blueprint, Pfizer released its own full trial design for their vaccine trials. The move was designed to boost transparency and shore up public trust in the vaccines, but it also revealed differences in how the two companies are approaching the much-watched studies while failing to satisfy the demands of the fiercest advocates for transparency.

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#ES­MO20: Re­gen­eron, Sanofi eye an­oth­er first for their PD-1 con­tender Lib­tayo with promis­ing da­ta for on­col­o­gy niche

Regeneron and Sanofi took another step forward in the long march towards a greatly expanded market for their late-bloomer PD-1 checkpoint Libtayo.

The two occasional allies posted an objective response rate of 31% for Libtayo among 84 patients suffering from advanced cases of basal cell carcinoma at virtual ESMO. That spotlights progress for 26 patients, 5 of whom had a complete response. The data also reflect a boost in the number of responses seen from the last cut of the numbers.

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Albert Bourla, Pfizer CEO (Steven Ferdman/Getty Images)

Pfiz­er ex­ecs con­fi­dent­ly tap their top 10 block­busters-to-be. But what are the chances of sur­viv­ing PhI­II, let alone hit­ting these big peak sales es­ti­mates?

Pfizer’s top executive team doesn’t lack for confidence.

Where many Big Pharmas would be reluctant to put a peak sales figure on their late-stage drugs, Pfizer CEO Albert Bourla has shrugged off the usual diffidence to outline where the pharma giant expects to get $15 billion-plus.

The list, outlined this week during their investor presentations, is topped by 3 drugs in the $3 billion-plus peak sales category. They are:

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