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

Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $800 million.

En­her­tu picks up an­oth­er win for As­traZeneca and Dai­ichi Sankyo, join­ing the pri­or­i­ty re­view lane for gas­tric can­cer

Five months after Enhertu received twin breakthrough therapy designations, AstraZeneca and Daiichi Sankyo are one step closer to nabbing another approval for their potential blockbuster drug.

The companies announced Wednesday morning that their billion-dollar antibody-drug conjugate has received priority review for HER2 positive metastatic gastric cancer. Already approved in the US for third-line metastatic breast cancer patients that are HER2 positive, Enhertu’s gastric cancer PDUFA date is scheduled for the first quarter of 2021.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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Sci­en­tists warn Amer­i­cans are ex­pect­ing too much from a coro­n­avirus vac­cine

The White House and many Americans have pinned their hopes for defeating the Covid-19 pandemic on a vaccine being developed at “warp speed.” But some scientific experts warn they’re all expecting too much, too soon.

“Everyone thinks COVID-19 will go away with a vaccine,” said William Haseltine, chair and president of Access Health International, a foundation that advocates for affordable care.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early- and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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No­var­tis CEO Vas Narasimhan signs off on a $231M deal to try some­thing new in the R&D fight against SARS-CoV-2

Patrick Amstutz was baptized by pandemic fire early on.

He and colleagues attended the notorious Cowen conference in early March that included some of the top Biogen execs who helped trigger a superspreader event in Boston. Heading back to his post as CEO of Molecular Partners in Switzerland, the outbreak was sweeping through Italy, triggering near panic in some quarters and creeping into the voices of people he knew, including one friend on the Italian side of the country.

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