Af­ter rocky hep C path, Achillion finds sur­er foot­ing with FDA break­through sta­tus

Achillion’s promis­ing proof-of-con­cept da­ta for its lat­est drug has won break­through sta­tus at the FDA.

The US reg­u­la­tor grant­ed its cov­et­ed des­ig­na­tion to dan­i­co­pan as a treat­ment for ane­mia in parox­ys­mal noc­tur­nal he­mo­glo­bin­uria pa­tients tak­ing the main­line C5 in­hibitor treat­ment Soliris. Up to 75% of PNH pa­tients suf­fer from ane­mia even af­ter treat­ment, ac­cord­ing to Achillion, with up to a third re­quir­ing blood trans­fu­sions in the past year.

Steven Ze­lenkofske

The news comes as a boon for Achillion $ACHN, still reel­ing from its de­ci­sion last year to shelve a drug they hoped could com­pete as a monother­a­py for PNH pa­tients. The com­pa­ny’s stock shot up 14%. In Q2 the biotech re­port­ed loss­es close to $20 mil­lion, leav­ing $241 mil­lion in cash and se­cu­ri­ties in re­serve.

The break­through des­ig­na­tion was based on in­ter­im da­ta from a Phase II tri­al, and the biotech said they hope to have full re­sults by the end of 2019 with a Phase III tri­al un­der­way by ear­ly 2020.

Achillion pre­sent­ed the Phase II da­ta from 11 pa­tients at a May con­fer­ence. They found pa­tients’ he­mo­glo­bin in­creased by 2 g/dl at week 4, with av­er­age in­creas­es of 2.6 g/dl for pa­tients who reached week 24. This meant a dra­mat­ic de­crease in blood trans­fu­sions for pa­tients – from 34 trans­fu­sions to­tal­ing 54 units to one trans­fu­sion to­tal­ing two units.

Pa­tients al­so im­proved on a func­tion­al as­sess­ment of chron­ic ill­ness ther­a­py fa­tigue ver­sus base­line, in­creas­ing on av­er­age from 4 t0 11. Achillion re­port­ed the drug was “gen­er­al­ly well tol­er­at­ed” when added to Soliris.

Achillion orig­i­nal­ly found the spot­light as a prime con­tender in the he­pati­tis C field, un­til Gilead came up with its pain­less cure and dra­mat­i­cal­ly shrunk the mar­ket.  That prompt­ed a piv­ot to ACH-4471 as their pri­ma­ry tar­get. Af­ter some rocky rid­ing, it seems to have found a foot­ing.

Alex­ion has raked bil­lions from Soliris, both for treat­ing PNH and oth­er blood dis­or­ders such as atyp­i­cal he­molyt­ic ure­mic syn­drome, but now faces po­ten­tial com­pe­ti­tion in the form of a biosim­i­lar from Am­gen.

So­cial im­age: Shut­ter­stock

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

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Covid-19 roundup: BAR­DA sup­ports Op­er­a­tion Warp Speed with big $628M con­tract to ser­vice Amer­i­ca's vac­cine pro­duc­tion needs

Another BARDA contract designed to service America’s Covid-19 vaccine needs has been deployed.

The White House-led initiative designed to bankroll development to bring a vaccine to the American public by this fall — Operation Warp Speed — has via BARDA handed a meaty contract to the maker of an FDA-licensed anthrax vaccine to open up its manufacturing apparatus to shore up production of Covid-19 vaccines.

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Adam Keeney (NodThera)

As NL­RP3 play­ers head for first clin­i­cal face off, No­vo, Sanofi fu­el trans-At­lantic con­tender with $55M

In the relative short history of inflammasome research, Adam Keeney sees two time points marking major breakthroughs: The early 2000s, when the role of inflammasomes as a major innate immunity node was elucidated; and 2015, when Pfizer found out a compound it thought was targeting IL-1 actually blocks NLRP3. Keeney’s biotech, NodThera, was founded the year after alongside several others to create its own superior small molecule drugs.

Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

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