Alex­ion's cash cow Soliris just got fat­ter as FDA green­lights an­oth­er block­buster in­di­ca­tion

Alex­ion has scored an ad­di­tion­al FDA OK for its key­stone rare ther­a­py Soliris, un­lock­ing what a key an­a­lyst calls a sig­nif­i­cant part of its C5 fran­chise and el­e­vat­ing that port­fo­lio fur­ther up the block­buster ranks.

The drug is now ap­proved to treat neu­romyelitis op­ti­ca spec­trum dis­or­der, a rare au­toim­mune con­di­tion char­ac­ter­ized by sud­den at­tacks on the cen­tral ner­vous sys­tem. The dis­ease of­ten strikes women in their 30s and 40s, and could be con­fused with mul­ti­ple scle­ro­sis. While off-la­bel Rit­ux­an helps most pa­tients — ap­prox­i­mate­ly 70% — keep the dis­ease at bay, re­laps­es still threat­en to do ir­re­versible harm and cause se­vere dis­abil­i­ty to those for whom the stan­dard of care doesn’t work.

Ge­of­frey Porges

In a Phase III test­ing the drug in pa­tients who had mul­ti­ple re­laps­es de­spite pri­or im­muno-sup­pres­sive ther­a­pies, Soliris re­duced the risk of NMOSD re­lapse by 94.2% com­pared to place­bo (p<0.0001) — a stel­lar re­sult. And in the lat­est da­ta cut at 144 weeks, 96% of the Soliris group didn’t ex­pe­ri­ence an at­tack, com­pared to 45% of pa­tients in the place­bo arm who re­mained re­lapse-free.

No­tably, reg­u­la­tors did not lim­it Soliris’ use to non-re­spon­ders, of­fer­ing a blan­ket la­bel that cov­ers any pa­tient with an­ti-aqua­por­in-4 an­ti­bod­ies — which is be­lieved to ac­ti­vate the com­ple­ment sys­tem that Soliris in­hibits.

That sur­prised SVB Leerink’s George Porges, a big cheer­leader of the drug, but he is care­ful to note that a broad­er la­bel does not guar­an­tee a broad­er pa­tient pop­u­la­tion.

De­spite the la­bel, we don’t think Soliris will cap­ture sig­nif­i­cant use in front line treat­ment giv­en Rit­ux­an’s fa­vor­able cost-ben­e­fit pro­file (~70% dis­ease con­trol un­der Rit­ux­an and ~$60K first year cost com­pared ~$700K first year cost for Soliris). We ex­pect most in­sur­ance pay­ers to re­quire ev­i­dence of Rit­ux­an fail­ure be­fore re­im­burs­ing for Soliris in this dis­ease, but do ex­pect rel­a­tive­ly open ac­cess to Soliris for re­lapsed pa­tients in late line treat­ment.

He notes that Alex­ion is soon start­ing a Phase III tri­al in the same in­di­ca­tion for Ul­tomiris, its fol­lowup to Soliris that’s eas­i­er to use. By his es­ti­mates, be­tween the two drugs the NMOSD sales alone will bring in around $1 bil­lion in 2028, rep­re­sent­ing one-fifth of the to­tal C5 fran­chise that year.

As far as com­pe­ti­tion goes, Roche and its Chugai sub­sidiary has some Phase III da­ta to show for its IL-6 in­hibitor satral­izum­ab. As­traZeneca spin­out Viela Bio is fast on its heels with the CD-19 in­hibitor inebi­lizum­ab. But with no di­rect ri­val in the C5 class, Porges sees Soliris cap­tur­ing the dom­i­nant share in re­lapse cas­es.

For Alex­ion’s part, R&D chief John Orloff is hap­py to high­light the fact that Soliris is the first drug ap­proved to re­duce the risk of NMOSD re­lapse. Re­views in Eu­rope and Japan are on­go­ing.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

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Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Charlie Fuchs, Roche and Genentech global head of product development for oncology and hematology (Yale Cancer Center)

Yale can­cer spe­cial­ist Char­lie Fuchs tapped as new glob­al de­vel­op­ment chief for Roche/Genen­tech

Roche and their big sub Genentech have just recruited a top cancer specialist at Yale to head up global product development in oncology and hematology.

I just got word that the pharma giant, which leads one of the most active cancer research operations in the world, recruited Charlie Fuchs, director of the Yale Cancer Center and physician-in-chief of Smilow Cancer Hospital. He’ll join the global operation March 1 and will be based in South San Francisco, where Genentech is based.

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Jonathan Weissman (MIT)

Can a new CRISPR tech­nique un­lock the se­crets of how can­cer spreads?

Jonathan Weissman’s team watched the cancer cells spread across the doomed mouse. Engineered with a bioluminescent enzyme, they appeared in scans first as a small navy blue diamond lodged near the heart; a week later, as a triangle splayed across the mouse’s upper body, with streaks of green and two distinct bright red hubs of activity. By day 54, the mouse resembled a lava lamp.

The images would have been familiar to any cancer biologist, but they didn’t actually tell you much about what was going on: why the cancer was metastasizing or which cells were responsible. For that, Weissman’s team had designed a new tool. Inside the original navy blue diamond, they had engineered the microbiological equivalent of an airplane’s black box — a “molecular recorder” that, after the mouse’s death, could allow them to extract the cells and wind back intimate footage of a single cancer’s ascent.

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Michelle McMurry-Heath, BIO CEO (BIO via YouTube)

BIO looks to re­struc­ture, lay­ing off staff amid chal­lenge to the trade org's nor­mal face-to-face style

The biopharma industry, on the whole, had a red-letter year in 2020 amid Covid-19, with fundraising at an all-time high and major players speeding vaccines ahead to approval. But for BIO, the industry’s leading trade organization, the pandemic has prompted a reconsideration of the game plan.

BIO will pivot to digital as the Covid-19 pandemic continues to rage, making “some staff reductions” as it looks to bring its roughly 37,000 in-person meetings each year to the web, the organization said Thursday.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.