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).

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.