Mit­subishi Tan­abe wins the race to snag the first new ALS drug OK in more than 20 years

Af­ter be­ing re­cruit­ed by the FDA, Mit­subishi Tan­abe has won the agency’s ap­proval to start sell­ing edar­avone as a new treat­ment for ALS — the first new drug for this lethal dis­ease in more than two decades and on­ly the sec­ond now avail­able to pa­tients.

Reg­u­la­tors asked the Japan­ese phar­ma com­pa­ny to file the drug for ALS af­ter its ap­proval in Ko­rea and Japan. The drug — which has al­so been sold for acute is­chemic stroke for years in Japan — will now hit the mar­ket as Rad­i­ca­va.

For a short time at least, a new sub­sidiary group set up to sell the drug will have a clear crack at a mar­ket that was slat­ed to shrink as the sole brand­ed ALS drug lost rev­enue to gener­ics. The last new ther­a­py, Ri­lutek (rilu­zole) from Sanofi, ar­rived in the US in the mid-90s, un­der­scor­ing just how dif­fi­cult this dis­ease has been to tack­le in the clin­ic.

Rad­i­ca­va is no cure. Pa­tients con­tin­ued to de­cline in the 167-pa­tient study re­viewed by the FDA, but they weren’t so quick to lose their abil­i­ty to func­tion. And in a twist, it ar­rives af­ter reg­u­la­tors asked the phar­ma com­pa­ny to file for an OK.

Er­ic Bast­ings, FDA CDER

“Af­ter learn­ing about the use of edar­avone to treat ALS in Japan, we rapid­ly en­gaged with the drug de­vel­op­er about fil­ing a mar­ket­ing ap­pli­ca­tion in the Unit­ed States,” said Er­ic Bast­ings, deputy di­rec­tor of the Di­vi­sion of Neu­rol­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “This is the first new treat­ment ap­proved by the FDA for ALS in many years, and we are pleased that peo­ple with ALS will now have an ad­di­tion­al op­tion.”

The com­pa­ny hasn’t for­mal­ly dis­trib­uted da­ta on the drug, that’s com­ing up at a sci­en­tif­ic con­fer­ence lat­er in the year, but re­searchers have not­ed that pa­tients tak­ing a 60 mg dose of the drug de­clined at a rate rough­ly a third slow­er than the place­bo arm, com­par­ing them to base­line on the re­vised ALS Func­tion­al Rat­ing Scale.

Neil A. Shnei­der, di­rec­tor of the Eleanor and Lou Gehrig ALS Cen­ter at Co­lum­bia Uni­ver­si­ty Med­ical Cen­ter, told The New York Times that the “ef­fect is mod­est but sig­nif­i­cant.”

Mit­subishi Tan­abe is plan­ning to go af­ter the mar­ket with a price of $1,086 per in­fu­sion and an an­nu­al list price of $145,524, which could pose some trou­ble for the phar­ma com­pa­ny. In ad­di­tion to the high cost and lim­it­ed ben­e­fits, Shnei­der and oth­ers in the field are none too hap­py about the risks posed to pa­tients who will need to keep an IV line left in place for their in­fu­sions, rais­ing the risk of in­fec­tion.

In the mean­time, there are at least two oth­er biotechs look­ing to make their mark in the field. Cy­to­ki­net­ics is one, with a late-stage drug called tirasem­tiv. AB Sci­ence’s AB-1010, an oral ty­ro­sine ki­nase in­hibitor, was filed for an ap­proval in Eu­rope last fall.

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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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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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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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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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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File photo (Endpoints News)

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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Bris­tol My­ers Squibb fi­nal­ly gets in the front­line NSCLC game dom­i­nat­ed by Mer­ck, adding a sec­ond Op­di­vo/Yer­voy-based op­tion

Bristol Myers Squibb may be trailing Merck and Roche in the checkpoint race to treat frontline cases of non-small cell lung cancer, but as it does, it makes sure to bring its best feet forward.

Just days after scoring a landmark NSCLC approval for Opdivo and Yervoy alone for PD-L1 positive patients, the company said the FDA has also OK’d using the two agents with a limited course of chemo regardless of the biomarker status.

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