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.

 

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Covid-19 roundup: Mod­er­na dos­es first pa­tients in PhII vac­cine tri­al; Eli Lil­ly touts clin­i­cal en­try of Ab­Cellera-dis­cov­ered an­ti­body

With an eye on kick-starting Phase III for its mRNA vaccine in July, Moderna said it’s dosed the first participants of each age cohort enrolled in the Phase II study.

Moderna is responsible for the whole trial, which is designed to recruit 600 healthy volunteers — 300 between the ages of 18 and 55, and another 300 older than 55. Two doses (50 μg and 100 μg) will be tested alongside placebo, and each participant receives two shots.

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Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.

Jean-Jacques Bienaime, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Ver­sant-backed, Bris­tol My­ers-stamped Re­pare Ther­a­peu­tics guns for $100M IPO

With a Bristol Myers Squibb endorsement in tow, Versant-backed cancer drug developer Repare Therapeutics has set its sights on a Nasdaq debut.

On Friday, the Montreal-based company with operations in Cambridge, Massachusetts that is yet to enter the clinic, unveiled plans for a $100 million IPO, banking on its “synthetic lethality” platform.

The basic idea is to target the genetic basis of tumors, a common idea across precision oncology medicines. But instead of targeting the perpetrator mutation directly, the compound is designed to go after the other gene in the gene pair. The rationale is based on the decades-old genetic principle that indicates two mutations are lethal only when combined together.

Lynn Seely, Myovant CEO

My­ovant’s re­l­u­golix wins a piv­otal prostate can­cer show­down with an old stan­dard — com­ing down to the wire on ap­provals

Myovant $MYOV has rounded the final turn in its development race to get relugolix down to the regulatory wire at the FDA. And the biotech joined the virtual crowd at ASCO with the kind of data needed to keep the investor crowd’s attention.

Much of the attention on the drug has been focused on uterine fibroids, where AbbVie just scored a regulatory win for their rival drug Oriahnn (elagolix) as the biotech posted results in prostate cancer at the ASCO meeting.

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In a sting­ing set­back, Pfiz­er’s can­cer block­buster Ibrance flops in key ad­ju­vant set­ting

One of Pfizer’s top, long-running R&D catalysts has gone up in smoke, and it took an $11 billion bite of their market cap in the process.

The monitoring committee determined that Pfizer’s adjuvant study using Ibrance combined with standard endocrine therapy in an adjuvant setting for early-stage breast cancer has officially failed to make the cut. The combo failed to beat the standard alone, tripping over the futility analysis. And the Pfizer team will now wrap the study early after pumping up hopes that their blockbuster cancer therapy could find billions more by proving its efficacy for disease-free survival in a major area — something AstraZeneca just accomplished with Tagrisso to great fanfare.

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