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.

 

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Pearl Huang, former Cygnal Therapeutics president and CEO

UP­DAT­ED: Flag­ship builds a new start­up out of pieces from 2 of its biotechs. And a Roche vet leaves to do some­thing new

Flagship has crafted a new startup out of pieces from a pair of fledglings in the VC’s nest. And a prominent Roche veteran who ran one of the biotechs won’t be making the next leg of the journey.

The new company is called Sonata Therapeutics, which is picking up the work that Inzen was doing related to the cellular microenvironment and combining with Flagship’s Cygnal Therapeutics, which came out of stealth more than 3 years ago and put Pearl Huang — the BeiGene founder and former Roche SVP — at the helm.

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Amidst R&D reshuf­fle, Ver­tex ex­pands its pres­ence in Boston, aim­ing to be­come num­ber one

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

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Martin Shkreli (Dennis Van Tine/MediaPunch/IPX)

In­fa­mous biotech ex­ec Mar­tin Shkre­li gets out of prison, hits the street

Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after 4 years inside a federal penitentiary.

Shkreli’s attorney put out a statement Wednesday afternoon saying that the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14. Attorney Benjamin Brafman acknowledged the release and vowed that he and Shkreli are keeping quiet.

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De­spite fed­er­al ef­forts to di­ver­si­fy clin­i­cal tri­als, progress re­mains 'stag­nan­t' — re­port

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

Paul Chaplin, Bavarian Nordic president and CEO

Bavar­i­an Nordic se­cures BAR­DA con­tract for small­pox vac­cine

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.