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.

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.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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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.

As­traZeneca keeps the ball rolling on Dai­ichi-part­nered En­her­tu, pick­ing up 2nd in­di­ca­tion in gas­tric can­cer

AstraZeneca’s big gamble on Daiichi Sankyo’s antibody-drug conjugate Enhertu has already paid off with a big approval in breast cancer more than a year ago. But the partners have big plans for their blockbuster in the making, and a new nod in gastric cancer will raise their spirits even higher.

The FDA on Friday approved Enhertu to treat locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma in patients who have previously undergone at least one round of treatment with a Herceptin-based regimen, AstraZeneca said in a release.

Dan Skovronsky, Eli Lilly CSO (Lilly via Facebook)

Eli Lil­ly tees up dis­cov­ery pact worth more than $1.6B with Merus for T cell-fo­cused bis­pe­cif­ic an­ti­bod­ies

Under science chief Dan Skovronsky, Eli Lilly has taken some big swings at next-gen therapies, including trying to find the next big thing in oncology. Now, after one early failure in the field, Lilly is going back to the bispecific antibody well with a new deal with a Dutch biotech.

Lilly will pay $40 million upfront with an additional $20 million equity stake in Merus NV to identify and develop three bispecific antibodies looking to engage the CD3 antigen on T cells and redirect immune cells, the Indianapolis pharma giant said Tuesday.

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Andrew Allen (Gritstone)

As coro­n­avirus vari­ants trig­ger new alarms, the NIH is putting an un­der-the-radar ‘next-gen’ vac­cine in­to PhI

Over the past year, the world has been transfixed by the development of new vaccines to fight SARS-CoV-2. In a frenzy of activity, the new mRNA approach has delivered pioneering emergency approvals in record time. And with some setbacks, the more traditional big players are coming along with added jabs as the most affluent nations in the world begin to vaccinate large portions of their populations.

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The IPO queue adds 5 more biotechs hop­ing to ring in 2021 by blitz­ing Nas­daq

Following a record year for IPOs — in terms of both proceeds and count — there’s already a long lineup of biotechs ready to jump onto Nasdaq in the new year. The companies are likely looking for much higher raises than they initially projected on their S-1s. Now it’s time to see if investors are still hungry for another round of biotech stocks.

Sana helped set the pace early on, as its founders look to divvy up a fortune from their IPO. And late last week 5 more biotechs crowded in, looking to pick up the pace where 2020 left off. Here they are:

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News brief­ing: Beam bags a $260M pri­vate place­ment; mi­Ra­gen re­brands to Virid­i­an Ther­a­peu­tics

Agios vet John Evans has demonstrated how to raise big money for a little biotech.

The Beam Therapeutics CEO — and ARCH partner — has pieced together a $260 million private placement from a group of backers that includes Perceptive Advisors, Farallon Capital, Casdin Capital, Redmile Group and Cormorant Asset Management. And there are 3 main goals they’ll pursue with it: clinical development, strategic partnerships and general corporate purposes.

Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

Just a day after reports emerged that Janet Woodcock will serve as interim chief of the FDA, word has gotten out that she is also in the running for the permanent job.

The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

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