On the cusp of PhI, An­gel­man-fo­cused biotech scores po­ten­tial buy­out deal from Ul­tragenyx

Ul­tragenyx has found a new rare dis­ease to de­vote it­self to, rop­ing in a young up­start as it be­gins a long trek to a new treat­ment for An­gel­man syn­drome.

Their new part­ners at GeneTx is work­ing to­ward an IND for GTX-102, an an­ti­sense oligonu­cleotide de­signed to in­hib­it the UBE3A-AS DNA strand and there­by turn on an al­lele in neu­rons that pro­duces a key pro­tein miss­ing in pa­tients with An­gel­man.

Emil Kakkis

The col­lab­o­ra­tion — and po­ten­tial ac­qui­si­tion — would pit Ul­tragenyx against Je­re­my Levin’s team at Ovid, which has show­cased some con­tro­ver­sial Phase II da­ta last year for its ex­trasy­nap­tic GABAA re­cep­tor ag­o­nist. An­gel­man is al­so among the slate of neu­ro­log­i­cal tar­gets that Bio­gen and Io­n­is are tack­ling in an an­ti­sense deal.

Once the FDA ac­cepts the drug for re­view, Ul­tragenyx will have 30 days to ex­er­cise an ex­clu­sive op­tion to buy the com­pa­ny out — an op­tion worth $20 mil­lion in the agree­ment. The duo has pen­ciled in a po­ten­tial ex­ten­sion, which would call for an­oth­er $25 mil­lion pay­ment.

“An­gel­man syn­drome is a dev­as­tat­ing neu­ro­log­i­cal con­di­tion with no treat­ment op­tions that rep­re­sents a very sig­nif­i­cant un­met med­ical need,” said Ul­tragenyx CEO Emil Kakkis. “GTX-102 is a nov­el and promis­ing po­ten­tial ther­a­py spe­cif­ic to the dis­ease mech­a­nism in these pa­tients, and we are pleased that GeneTx chose to part­ner with us based on our track-record of rapid, in­no­v­a­tive de­vel­op­ment in rare dis­eases.”

In ad­di­tion, GeneTx has a unique con­nec­tion to the pa­tient com­mu­ni­ty that made it ap­peal­ing: The Foun­da­tion for An­gel­man Syn­drome Ther­a­peu­tics, the non­prof­it group that launched GeneTx last Feb­ru­ary, is the largest pa­tient or­ga­ni­za­tion for the rare neu­rode­vel­op­men­tal dis­or­der, Ul­tragenyx CBO Thomas Kass­berg told Bio­Cen­tu­ry.

Paula Evans

FAST is al­so the ma­jor­i­ty own­er of GeneTx while Texas A&M Uni­ver­si­ty Sys­tem owns a mi­nor­i­ty share. Paula Evans, FAST chair­per­son, GeneTx CEO and an An­gel­man mom, said it is fit­ting to shift from do­na­tions to a pact with an es­tab­lished in­dus­try play­er.

The two sides will col­lab­o­rate on both the IND and man­age­ment of the Phase I/II study. For now, GeneTx con­tin­ues to fund all de­vel­op­ment ac­tiv­i­ties and take charge of reg­u­la­to­ry and sci­en­tif­ic ac­tiv­i­ties, while Ul­tragenyx of­fers strate­gic and clin­i­cal guid­ance.

Tile im­age: The Evans fam­i­ly, FAST

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.

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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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.

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.