Bio­gen boasts PhII win in lu­pus — will any­one turn their head from Alzheimer's to no­tice?

As the gaze on ad­u­canum­ab in­ten­si­fies in the runup to Bio­gen’s hot­ly-an­tic­i­pat­ed da­ta pre­sen­ta­tion at an Alzheimer’s con­fer­ence this week, the biotech is of­fer­ing some re­lief in the form of pos­i­tive Phase II re­sults for a lu­pus drug.

Dubbed BI­IB059, the lit­tle known an­ti­body de­liv­ered clean hits on the pri­ma­ry end­points for both parts of the study, in­volv­ing pa­tients with cu­ta­neous lu­pus ery­the­mato­sus and sys­temic lu­pus ery­the­mato­sus, re­spec­tive­ly.

The re­sults are en­cour­ag­ing and add a third di­men­sion to the Bio­gen sto­ry out­side of Tec­fidera IP and the Alzheimer’s pro­gram, wrote Ever­core ISI an­a­lyst Umer Raf­fat in a note ti­tled “sleep­er hits”:

The MOA is broad­ly sim­i­lar to AZN an­i­frol­um­ab (AZN is an IFN block­er … BI­IB is NOT a di­rect IFN block­er – di­rect­ed against BD­CA2) – which re­cent­ly worked in Ph 3 TULIP-2 tri­al.

Un­like As­traZeneca’s TULIP-2, where ef­fi­ca­cy was re­port­ed in terms of re­spon­ders per­cent­age (per­cent­age of pa­tients who had a 50% re­duc­tion in the CLASI score), in the LILAC study Bio­gen mea­sured the re­duc­tions in CLASI on three dos­es of the drug.

Nathalie Franchi­mont

In the CLE por­tion, BI­IB059 in­duced re­duc­tions in dis­ease area and sever­i­ty by at least 40%: the 50mg group ex­pe­ri­enced re­duc­tions of 40.9% (p=0.008), the 150mg group 48% (p=0.001) and the 450mg group 40.9% (p=0.008). The place­bo arm saw an av­er­age 14.5% im­prove­ment on the score, which mea­sures skin dis­ease ac­tiv­i­ty.

As for SLE, the to­tal num­ber of ten­der or swollen joints, a com­mon symp­tom, was the proxy for ef­fi­ca­cy. All 132 SLE pa­tients en­rolled in the tri­al were giv­en ei­ther a 450mg dose or place­bo, and the treat­ment dif­fer­ence at week 24 was -3.4 (p-0.037).

Whether lim­it­ed to the skin (CLE) or sys­temic (SLE), lu­pus has no­to­ri­ous­ly de­feat­ed mul­ti­ple at­tempts at a vi­able treat­ment. Bio­gen has run in­to a wall with a late-stage an­ti-CD40L drug part­nered with UCB, and a few years ago scrapped sev­er­al oth­er R&D projects in the area in­clud­ing a ther­a­py tar­get­ing the TWEAK im­mune cell.

Back in 2016 Bio­gen was spot­ted sneak­ing a Bru­ton’s ty­ro­sine ki­nase in­hibitor in­to the clin­ic as a po­ten­tial treat­ment for SLE. That Phase I study has been com­plet­ed, but BI­IB068 is now nowhere to be seen in Bio­gen’s pipeline and its sta­tus is un­clear.

De­spite the con­fi­dence boost that the topline num­bers in­ject, though, Evan Seiger­man of Cred­it Su­isse said Bio­gen still has a lot to prove, from over­com­ing chal­lenges with di­ag­no­sis and vari­able clin­i­cal fea­tures to safe­ty.

“No sub­stan­tial in­for­ma­tion was pro­vid­ed re­gard­ing safe­ty (re­port­ed­ly will be pre­sent­ed at fu­ture med­ical meet­ing),” he not­ed. “We await more de­tails over safe­ty, giv­en that high­er dos­es demon­strat­ed bet­ter ef­fi­ca­cy while on­ly the 450mg dose was sta­tis­ti­cal­ly sig­nif­i­cant for SLE. Fur­ther­more, the large spread be­tween 150mg and 450mg could po­ten­tial­ly lead to in­creased safe­ty sig­nals.”

BI­IB059 is de­signed to tar­get blood den­drit­ic cell anti­gen 2, or BD­CA2, a re­cep­tor ex­pressed on a sub­set of im­mune cells known as plas­ma­cy­toid den­drit­ic cells. The sub­cu­ta­neous in­jec­tion, the the­o­ry goes, can block the cells’ pro­duc­tion of in­flam­ma­to­ry cy­tokines such as type-I IFN, there­by blunt­ing the au­toim­mune at­tack on pa­tients.

Nathalie Franchi­mont, VP in charge of the lu­pus and mul­ti­ple scle­ro­sis unit, hoped “these re­sults sup­port Bio­gen’s goal of con­tin­u­ing to build a mul­ti-fran­chise port­fo­lio.” But the tiny move­ment in Bio­gen’s stock price $BI­IB sug­gest­ed in­vestors are not quite ready to buy in.

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

As FDA’s work­load buck­les un­der the strain, 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.”

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

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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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.

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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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.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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