Does an im­proved safe­ty pro­file strength­en Al­ler­gan's case for a wet AMD chal­lenge to Lu­cen­tis?

Al­ler­gan has chalked up some flat­ter­ing safe­ty da­ta for abic­i­par as it re­fines the eye drug’s man­u­fac­tur­ing process­es in prepa­ra­tion of an im­pend­ing BLA fil­ing. But whether or how much that mat­ters is still up for de­bate.

In an open-la­bel, sin­gle-arm study, in­ves­ti­ga­tors re­port­ed that the in­ci­dence of in­traoc­u­lar in­flam­ma­tion was 8.9% — low­er than the rate ob­served in pri­or Phase III stud­ies, which hov­ered around 15%. The MAPLE tri­al re­cruit­ed 123 age-re­lat­ed neo­vas­cu­lar mac­u­lar de­gen­er­a­tion (or wet AMD) pa­tients and ad­min­is­tered the in­jec­tion five times over 28 weeks.

David Nichol­son, Al­ler­gan

“The re­sults of this open-la­bel study en­abled us to as­sess im­prove­ments to the man­u­fac­tur­ing process for abic­i­par. The safe­ty pro­file demon­strat­ed in MAPLE gives us con­fi­dence to pro­ceed and scale up man­u­fac­tur­ing,” said David Nichol­son, chief re­search and de­vel­op­ment of­fi­cer. “We plan to sub­mit the abic­i­par BLA and con­tin­ue to pur­sue man­u­fac­tur­ing process im­prove­ments as we de­vel­op larg­er scale stud­ies in ad­di­tion­al dis­ease states, such as di­a­bet­ic mac­u­lar ede­ma.”

Any lit­tle im­prove­ment could be cru­cial as Al­ler­gan and its al­lies at Mol­e­c­u­lar Part­ners go up against the en­trenched mar­ket lead­ers, Re­gen­eron’s Eylea and No­var­tis’ Lu­cen­tis, both ap­proved for mul­ti­ple in­di­ca­tions oth­er than wet AMD, which is far less com­mon but much more se­vere than dry AMD. And the two gi­ants are each ad­vanc­ing fol­low-on ef­forts of their own.

The new in­flam­ma­tion rates, though, might still do lit­tle to change in­vestors’ skep­ti­cal views on the com­mer­cial prospect, said Umer Raf­fat of Ever­core ISI.

Raf­fat notes that se­vere in­flam­ma­tion reg­is­tered at 1.6% in the MAPLE tri­al, com­pared to 3.2% to 3.7% ob­served in pre­vi­ous Phase III stud­ies.

AGN feels strong­ly that it’s re­al­ly just the se­vere in­flam­ma­tion that mat­ters … and that it’s now at a well con­trolled rate (<2%) … and that mild in­flam­ma­tion is asymp­to­matic.

If we agree with AGN’s log­ic, there is an impt piece of da­ta that hasn’t been dis­closed about MAPLE: the rate of mod­er­ate in­flam­ma­tion. (we know mild+mod+se­vere = 8.9% … and se­vere is 1.6% … but what is mod­er­ate? if we go by Ph 3 re­sults, mod­er­ate could be half of this 8.9% … but un­clear if that ac­tu­al­ly hap­pened in MAPLE al­so).

To put it blunt­ly: “It will be a ‘show-me’ sto­ry at launch.”

An­a­lysts for SVB Leerink was even more doubt­ful, not­ing:

Re­port­ed in­ci­dence of in­traoc­u­lar in­flam­ma­tion (IOI) was 8.9% in the MAPLE study, which was low­er than pri­or Phase 3 stud­ies (>15% af­ter 1 year of treat­ment), but still sig­nif­i­cant­ly high­er than the rate ob­served in No­var­tis’ (NVS, NR) Lu­cen­tis and Re­gen­eron’s (REGN, OP) Eylea, which we think would lim­it its suc­cess to be a re­al play­er in the space.

In­vestors seem to be sit­ting this one out for now. Al­ler­gan $AGN shares are slight­ly in the red this morn­ing, dip­ping 0.33%, or $0.5, pre-mar­ket.

The duo has pre­vi­ous­ly tout­ed Phase III da­ta sug­gest­ing abic­i­par is non-in­fe­ri­or to Lu­cen­tis on both test­ed sched­ules, though the in­ci­dence of in­traoc­u­lar in­flam­ma­tion re­mained a sore point giv­en that less than 1% of the pa­tients on the ri­val drug ex­pe­ri­enced it.

Where Al­ler­gan is hop­ing to stand out is the dos­ing reg­i­men, a fixed 12-week reg­i­men that could “great­ly re­duce the treat­ment bur­den” ac­cord­ing to Cleve­land Clin­ic Cole Eye In­sti­tute’s Pe­ter Kaiser. That’s thanks to Mol­e­c­u­lar Part­ners’ DARPin tech­nol­o­gy, which it says has high­er sta­bil­i­ty and po­ten­cy than mon­o­clon­al an­ti­bod­ies.

A BLA is still slat­ed for the next two months, Al­ler­gan and Mol­e­c­u­lar Part­ners say, as pre­vi­ous­ly re­port­ed.

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

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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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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Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist partnered with Novartis, the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Novus Ther­a­peu­tics plunges deep in­to pen­ny stock ter­ri­to­ry af­ter failed ear tri­al

After a more than 15-year run, a California-based biotech is exploring options, including a sale, after its lead experimental therapy failed an exploratory mid-stage study in patients with middle ear infections characterized by a build-up of fluid behind the eardrum.

The company, initially called Tokai Pharmaceuticals but which subsequently changed its name to Novus Therapeutics in 2017, saw its shares more than halve on Monday after the drug — OP0201— did not pass muster as an adjunct therapy to oral antibiotics in infants and children aged 6 to 24 months with acute otitis media (OM).

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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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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Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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