Neu­rotrope bat­tered in lat­est Alzheimer's flop; Platelet Bio­Gen­e­sis rais­es $26M-plus

Neu­rotrope be­came the lat­est ca­su­al­ty in the high-risk Alzheimer’s R&D field. The biotech re­port­ed that their Phase II study of Bryo­statin-1 failed to show any im­prove­ment over place­bo us­ing the Se­vere Im­pair­ment Bat­tery (SIB) to­tal score at week 13. In fact, the drug arm did a lit­tle worse than a sug­ar pill, with an av­er­age in­crease of 1.3 points for the drug and 2.1 points for place­bo. Their shares $NTRP cratered in the rout that fol­lowed, plung­ing 78%. It’s cur­rent­ly a pen­ny stock.

The Alzheimer’s R&D field hasn’t record­ed a suc­cess in chang­ing the tra­jec­to­ry of the dis­ease, with re­cent piv­otal fail­ures forc­ing all the play­ers to re­con­sid­er their strate­gies.

Platelet Bio­Gen­e­sis (PBG) — a com­pa­ny fo­cused on stem cell-de­rived, on-de­mand hu­man platelets and platelet-based ther­a­peu­tics — has raised more than $26 mil­lion in Se­ries A-1 fi­nanc­ing. In com­bi­na­tion to this round, as well as Se­ries A fi­nanc­ing and grants, the com­pa­ny’s to­tal fund­ing is brought to about $45 mil­lion. The fi­nanc­ing was co-led by Ziff Cap­i­tal Part­ners and Qim­ing Ven­ture Part­ners USA in ad­di­tion to ex­ist­ing in­ves­tures Nest.Bio Ven­tures and eCoast An­gels.

The com­pa­ny said that it “will use the funds from the Se­ries A-1 round to ac­cel­er­ate the de­vel­op­ment of its donor-in­de­pen­dent platelet man­u­fac­tur­ing ca­pa­bil­i­ties and ad­vance its proof of con­cept work around platelet-based ther­a­peu­tics.” PBG plans to dou­ble its staff to ap­prox­i­mate­ly 50 em­ploy­ees in 2020.

Stu­art Levy, co-founder of Paratek Phar­ma­ceu­ti­cals, passed away last week.

“Stu­art was a dis­tin­guished physi­cian who was the fore­front of an­tibi­ot­ic de­vel­op­ment and a ded­i­cat­ed cham­pi­on for the pru­dent use of an­tibi­otics,” said Evan Loh, Paratek CEO. “More im­por­tant­ly, he was a dear friend and men­tor to so many and his pas­sion lives on in each of us as we work to con­tin­ue his mis­sion of com­bat­ing the dai­ly threat of life-threat­en­ing in­fec­tions. We will great­ly miss him.”

Levy co-found­ed Paratek more than 20 years ago along with Wal­ter Gilbert. He was in­stru­men­tal in the de­vel­op­ment of the com­pa­ny’s lead an­tibi­otics, Nuzyra and Seysara. Levy dis­cov­ered the ef­flux mech­a­nism for drug (tetra­cy­cline) re­sis­tance and was among the first sci­en­tists to doc­u­ment the trans­fer of re­sis­tant bac­te­ria from an­i­mals to farm­work­ers. His 1992 book, The An­tibi­ot­ic Para­dox: How Mir­a­cle Drugs Are De­stroy­ing the Mir­a­cle, has been cit­ed wide­ly and trans­lat­ed in­to four lan­guages.

The news comes a few weeks af­ter the com­pa­ny pub­lished pos­i­tive Phase III da­ta for Nuzyra for pa­tients with AB­SS­SI.

→ In the midst of a sur­prise in­ter partes re­view by the US Patent Tri­als Ap­peal Board (PT­AB) for Alex­ion’s Soliris, the com­pa­ny and Bridge­Bio’s sub­sidiary Ei­dos Ther­a­peu­tics have inked an agree­ment grant­i­ng Alex­ion an ex­clu­sive li­cense to de­vel­op and com­mer­cial­ize their oral­ly-ad­min­is­tered small mol­e­cule AG10 — de­signed to treat transthyretin amy­loi­do­sis (AT­TR) by bind­ing and sta­bi­liz­ing transthyretin (TTR) in the blood— in Japan.

“Ei­dos is cur­rent­ly eval­u­at­ing AG10 in a Phase III study in the US and Eu­rope for AT­TR car­diomy­opa­thy (AT­TR-CM) – a pro­gres­sive, fa­tal dis­ease caused by the ac­cu­mu­la­tion of mis­fold­ed TTR amy­loid in the heart – and plans to be­gin a Phase III study in AT­TR polyneu­ropa­thy (AT­TR-PN) – a pro­gres­sive, fa­tal dis­ease caused by the ac­cu­mu­la­tion of mis­fold­ed TTR amy­loid in the pe­riph­er­al ner­vous sys­tem.”

→ A few months af­ter Ac­celeron and its part­ner at Cel­gene se­cured pri­or­i­ty re­view for their red blood cell boost­ing drug, lus­pa­ter­cept — to man­age ane­mia re­sult­ing from myelodys­plas­tic syn­drome (MDS) as well as be­ta tha­lassemia — the com­pa­ny has an­nounced an­oth­er win. This time they’ve gained an or­phan drug des­ig­na­tion by the FDA for its in­ves­ti­ga­tion­al ther­a­py so­tater­cept for the treat­ment of pa­tients with pul­monary ar­te­r­i­al hy­per­ten­sion (PAH).

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Jean-Jacques Bienaime, 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:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Lynn Seely, Myovant CEO

My­ovant’s re­l­u­golix wins a piv­otal prostate can­cer show­down with an old stan­dard — com­ing down to the wire on ap­provals

Myovant $MYOV has rounded the final turn in its development race to get relugolix down to the regulatory wire at the FDA. And the biotech joined the virtual crowd at ASCO with the kind of data needed to keep the investor crowd’s attention.

Much of the attention on the drug has been focused on uterine fibroids, where AbbVie just scored a regulatory win for their rival drug Oriahnn (elagolix) as the biotech posted results in prostate cancer at the ASCO meeting.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

File photo (Endpoints News)

In a sting­ing set­back, Pfiz­er’s can­cer block­buster Ibrance flops in key ad­ju­vant set­ting

One of Pfizer’s top, long-running R&D catalysts has gone up in smoke, and it took an $11 billion bite of their market cap in the process.

The monitoring committee determined that Pfizer’s adjuvant study using Ibrance combined with standard endocrine therapy in an adjuvant setting for early-stage breast cancer has officially failed to make the cut. The combo failed to beat the standard alone, tripping over the futility analysis. And the Pfizer team will now wrap the study early after pumping up hopes that their blockbuster cancer therapy could find billions more by proving its efficacy for disease-free survival in a major area — something AstraZeneca just accomplished with Tagrisso to great fanfare.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.