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

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Dan O'Day (AP Images)

A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisitions, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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Vlad Coric (Photo Credit: Andrew Venditti)

Bio­haven scores CGRP OK for acute mi­graine — can the com­mer­cial team catch up with Al­ler­gan on its de­but?

Seven years after spinning out of Yale, Biohaven has entered the ranks of commercial-stage biotechs.

The FDA handed down an OK for its CGRP drug, rimegepant, as an acute treatment. Dubbed Nurtec, the orally dissolving pill will join Allergan’s (soon to be AbbVie’s) Ubrelvy and Lilly’s Reyvow on the market amid a new wave of migraine therapies reshaping the disease space.

In a pivotal Phase III trial, Nurtec hit the co-primary endpoints on pain freedom and freedom from most bothersome symptoms at two hours post dose, proving superior to placebo.

Mi­cro­bio­me Q&A: New study maps the vagi­na's 'op­ti­mal mi­cro­bio­ta' — and its im­pli­ca­tions for bio­phar­ma

The widely-held notion that the “optimal” vaginal microbiota is dominated by one strain of lactic-acid producing bacteria has now been challenged in a new paper, published in Nature Communications on Wednesday, which used advanced gene sequencing methods to map out the most comprehensive gene catalog of the human vaginal microbiome.

Things have changed in the more than 50 years since the concept of vaginal microbiota transplants was proposed and subsequently tainted by a Texas-based gynecologist who transplanted the vaginal fluid of women who had bacterial vaginosis into healthy females, suspecting he had isolated the bacteria responsible for the condition.

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