Is bio­phar­ma’s first $1.5M ther­a­py now in Phase III?

Key­ing off of Spark Ther­a­peu­tics’ roll­out of Lux­tur­na as the first ap­proved gene ther­a­py in the US, the an­a­lysts at Leerink have been crunch­ing the num­bers on what the first he­mo­phil­ia gene ther­a­pies could fetch. And by rais­ing their es­ti­mate to $1.5 mil­lion, they may well be tempt­ing fate at a time the de­bate over drug pric­ing is reach­ing the boil­ing point in the US.

Spark sat­is­fied most an­a­lysts with its $425,000 price per eye for Lux­tur­na. And Leerink says that the pos­i­tive re­sponse by Har­vard Pil­grim to a 30-month dura­bil­i­ty start­ing point for a gene ther­a­py pric­ing agree­ment set the stage for many of the rest of the field to fol­low suit. And that will jus­ti­fy a $1.5 mil­lion-plus price for a ther­a­py that could re­place cur­rent stan­dards in he­mo­phil­ia with a sim­i­lar track record on dura­bil­i­ty.

Af­ter check­ing with some KOLs in the field, Leerink of­fered:

As a re­sult of this work, we are rais­ing our as­sumed price for he­mo­phil­ia gene ther­a­pies in our cov­er­age uni­verse to $1.5M per pa­tient in the US ($1M in the EU), an in­crease from pri­or as­sump­tions of $350,000-$500,000 (he­mo­phil­ia A) and $500,000 (he­mo­phil­ia B) per pa­tient. Our checks in­di­cate that there may be some head­room above this price, po­ten­tial­ly up to $2M per pa­tient on a gross ba­sis, but giv­en the un­cer­tain­ty about re­spon­sive­ness, re­bates and oth­er fac­tors, we be­lieve $1.5M is a jus­ti­fi­able es­ti­mate for net price per pa­tient at this junc­ture….

We be­lieve the man­u­fac­tur­ers are test­ing the wa­ters cur­rent­ly as they em­bark on Phase 3 tri­als in or­der to de­ter­mine where pay­ors push back sig­nif­i­cant­ly. Based on our checks, a price some­where in the range of $1-2M per pa­tient seems most like­ly.

The big rea­son for that 7-fig­ure price tag is based on pay­ers’ will­ing­ness to cov­er the $580,000 to $800,000 price per pa­tient for fac­tor re­place­ment ther­a­py, giv­ing them some up­side even with a $1 mil­lion-plus stick­er for a once-and-done gene ther­a­py. And that could have a pro­found ef­fect on Spark $ONCE, Bio­Marin $BM­RN and uniQure $QURE, which are all en­gaged in de­vel­op­ment ef­forts.

As long as the av­er­age pa­tient can re­ly on fac­tor re­place­ment ther­a­py, Leerink adds, the low hang­ing fruit for the de­vel­op­ers ear­ly on will be among the most se­vere pa­tients, and stick­ing with that small pa­tient pop­u­la­tion al­so helps jus­ti­fy the big num­ber.

At this stage of the game, the biotechs lead­ing this field will like­ly hedge their bets, steer­ing clear of of­fer­ing any num­bers they may have in mind as they test the wa­ters on the fi­nal price tag. But Leerink just gave them a big op­por­tu­ni­ty to con­tin­ue their pri­vate pay­er talks with some big num­bers in mind.

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”

Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Emily Leproust, Twist Bioscience CEO

Twist Bio­science’s 'fac­to­ry of the fu­ture' in Ore­gon could de­liv­er with com­pet­i­tive pric­ing, SVB Se­cu­ri­ties says

The synthetic DNA manufacturer Twist Bioscience has given a peek behind the curtain to several analysts into its “factory of the future” as well as insight into the cost structure, workflow and technology at the site.

The 110,000-square-foot manufacturing site in the city of Wilsonville, OR, just south of Portland, which was announced back in 2020, will double Twist’s production capacity and bring around 400 jobs to the area.

Lil­ly's Covid-19 mAb no longer au­tho­rized due to Omi­cron sub­vari­ants, FDA says

The FDA on Wednesday announced that Eli Lilly’s Covid-19 drug bebtelovimab is no longer authorized to treat Covid-19 because of the rising numbers of two new subvariants that the drug does not work against.

The Centers for Disease Control and Prevention last week published new estimates that the combined proportion of Covid-19 cases caused by the Omicron subvariants BQ.1 and BQ.1.1 are greater than 57% nationally, and already above 50% in all individual regions but one.

Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

Digital render of CPI's Medicines Manufacturing Innovation Centre in Glasgow, Scotland (Image: uk-cpi.com)

CPI opens the doors to a new $100M+ man­u­fac­tur­ing fa­cil­i­ty in Scot­land

A manufacturing site that has received interest and investments from large pharma companies and the UK government is opening its doors in Scotland.

The manufacturer CPI (Centre for Process Innovation) has opened a new £88 million ($105 million) “Medicines Manufacturing Innovation Centre” in Glasgow, Scotland, to accelerate the development of manufacturing tech and solve longstanding challenges in medicine development and manufacturing.