As­traZeneca, Am­gen whip up some block­buster ex­cite­ment with a pos­si­ble game-chang­ing asth­ma drug

As­traZeneca and Am­gen rolled out Phase IIb da­ta on their late-stage drug teze­pelum­ab late on Wednes­day, spot­light­ing some dra­mat­ic re­sults for a ther­a­py they be­lieve could have megablock­buster po­ten­tial for the broad-based treat­ment of un­con­trolled asth­ma.

Bot­tom line: Treat­ment-re­sis­tant pa­tients in the drug arm tak­ing a med­ley of dos­es saw their an­nu­al ex­ac­er­ba­tion rate slashed any­where from 61% to 71%.

De­vel­oped at As­traZeneca and bound for a com­mer­cial part­ner­ship, re­searchers say the drug was ef­fec­tive be­cause it tar­gets TSLP, an ep­ithe­lial cy­tokine stoked by lung pathogens such as al­ler­gens and virus­es. TSLP in turn trig­gers a ram­page of in­flam­ma­to­ry cy­tokines such as IL-4, IL-5 and IL-13.

By mov­ing up­stream from those tar­gets, the phar­ma gi­ants be­lieve that they may be on to a break­through ap­proach that can leap the more sin­gu­lar­ly-tar­get­ed ri­vals com­ing in­to the mar­ket, seiz­ing bil­lions in new rev­enue.

Bahi­ja Jal­lal

“This broad bio­mark­er re­sponse is un­prece­dent­ed among res­pi­ra­to­ry bi­o­log­ics and re­flects our com­mit­ment to lead­ing res­pi­ra­to­ry sci­ence for un­met med­ical needs,” says Bahi­ja Jal­lal, who runs As­traZeneca’s big sub­sidiary Med­Im­mune, where the re­search work is be­ing done.

That broad-based ap­proach could have a big im­pact on oth­er in­jectable res­pi­ra­to­ry drugs such as GSK’s Nu­cala, Te­va’s Cinqair as well as As­traZeneca’s own ben­ral­izum­ab — which it doesn’t share with Am­gen — and Dupix­ent from Re­gen­eron and Sanofi. Ben­ral­izum­ab is cur­rent­ly un­der re­view and is giv­en a good shot at an ap­proval.

Leerink’s Sea­mus Fer­nan­dez was ex­cit­ed by the da­ta. So ex­cit­ed that he be­lieves these part­ners have a shot at grab­bing an ac­cel­er­at­ed ap­proval on the Phase IIb da­ta while launch­ing Phase III as a con­fir­ma­to­ry study — an ap­proach that could start de­liv­er­ing up to $4.5 bil­lion a year in rev­enue with­in 4-5 years.

His com­ment:

Based on the strength of these da­ta, we be­lieve this tri­al may qual­i­fy as a reg­is­tra­tional study (with the need for a con­fir­ma­to­ry Phase III tri­al for ap­proval), sim­i­lar to the ap­proach that SNY/REGN are tak­ing with Dupix­ent. How­ev­er, this may be de­pen­dent on whether the FDA will want to see ad­di­tion­al dose rang­ing stud­ies to ad­dress ques­tions re­gard­ing safe­ty and the lack of a clear ef­fi­ca­cy dose re­sponse.

Dupix­ent, of course, has al­ready been rack­ing up rev­enue with an es­tab­lished safe­ty pro­file, mak­ing a quick, added OK much eas­i­er for reg­u­la­tors. In this case, reg­u­la­tors are much more like­ly to raise some barbed ques­tions about con­cerns this drug has spurred about its im­pact on the im­mune sys­tem. That is­sue was touched on by Elis­a­beth Bel of Am­s­ter­dam’s Aca­d­e­m­ic Med­ical Cen­ter, who wrote a piece in the NE­JM not­ing the need for safe­ty stud­ies that could care­ful­ly ex­am­ine the in­ter­play be­tween drug and im­mune sys­tem.

Nev­er­the­less, As­traZeneca and Am­gen now have a late-stage drug they can tout as a po­ten­tial block­buster in an in­tense­ly com­pet­i­tive field — ex­act­ly what the biotech or­dered for these com­pa­nies as they work to build new rev­enue with game chang­ing ther­a­pies.

That’s a def­i­nite plus.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA buck­les un­der the strain of a pan­dem­ic work­load, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Pal­la­dio bags $20 mil­lion Se­ries B to top­ple a prob­lem­at­ic kid­ney dis­ease drug

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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