Hunt­ing a cure, Spark re­ports steady progress on he­mo­phil­ia B gene ther­a­py as Pfiz­er preps for piv­otal hand­off

Af­ter some fine tun­ing on the man­u­fac­tur­ing side, Spark Ther­a­peu­tics $ONCE says it’s racked up a new round of promis­ing re­sults for its Phase I/II study of a ground­break­ing new gene ther­a­py that aims to cure he­mo­phil­ia B. And the biotech is prep­ping a hand­off of SPK-9001 to its Big Phar­ma al­lies at Pfiz­er $PFE this sum­mer, who are plan­ning to start a Phase III piv­otal study.

Kather­ine High

Re­searchers are now re­port­ing on 15 pa­tients in the ear­ly-stage study get­ting a re­place­ment fac­tor IX gene to cor­rect the mu­ta­tion that re­quires con­stant care and fac­tor re­place­ment ther­a­py to pre­vent bleeds. The an­nu­al­ized bleed rate for all of them was cut by 97% and re­searchers were care­ful to high­light three pa­tients who are do­ing fine — with fac­tor IX lev­els rang­ing from 38.1% to 54.5% be­yond the 12-week mark— af­ter be­ing treat­ed with an en­hanced ther­a­py pro­duced by a new man­u­fac­tur­ing ap­proach.

The an­nu­al in­fu­sion rate was slashed to 0.9 in­fu­sions, com­pared to 57.2 in­fu­sions be­fore the study. The da­ta were be­ing pre­sent­ed at the World Fed­er­a­tion of He­mo­phil­ia World Con­gress in Glas­gow to­day.

For the full group in the Spark study, the range on fac­tor IX was 14.3% to 76.8%. Six of the 15 had to get in­fu­sions; two for spon­ta­neous bleeds, two pri­or to surgery, and one each for a mi­nor trau­ma and at the end of the study pe­ri­od.

Elim­i­nat­ing these in­fu­sions safe­ly and re­li­ably will be key for Spark and Pfiz­er as they look to field the first he­mo­phil­ia gene ther­a­py, rac­ing against Bio­Marin $BM­RN and uniQure. Sang­amo $SG­MO, mean­while, is us­ing its gene edit­ing tech for he­mo­phil­ia B. 

Spark gained an his­toric FDA OK for Lux­tur­na, the first gene ther­a­py ap­proved in the US, which came out with a list price of $850,000. But an­a­lysts say that a he­mo­phil­ia gene ther­a­py could be the first to be mar­ket­ed for as much as $1.5 mil­lion, con­sid­er­ing the steep cost of in­fu­sions pa­tients un­der­go.

“We are pleased to see all 15 par­tic­i­pants, no­tably in­clud­ing the first four par­tic­i­pants who have been fol­lowed for more than two years, con­tin­ue to show that a sin­gle ad­min­is­tra­tion of SPK-9001 has re­sult­ed in dra­mat­ic re­duc­tions in bleed­ing and fac­tor IX in­fu­sions, with no se­ri­ous ad­verse events,” said Kather­ine High, the pres­i­dent and head of re­search & de­vel­op­ment at Spark Ther­a­peu­tics.

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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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

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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Fol­low­ing US, Chi­na hos­pi­tal ef­forts, Gilead plots its own PhI­II tri­als for close­ly watched Covid-19 drug

Gilead is launching its own Phase III trials of remdesivir, the repurposed antiviral that a WHO official called the “one drug right now we think may have real efficacy” against Covid-19 as the novel coronavirus originating from Wuhan, China ravages the world.

Announced just a day after the NIH and the University of Nebraska Medical Center registered their US-based trial online, Gilead’s program will comprise two studies enrolling around 1,000 patients beginning in March. They will recruit primarily in Asian countries but will also include patients from other locations with “high numbers of diagnosed cases,” the company said.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In at least one life-sci hub, gen­der and di­ver­si­ty ini­tia­tives haven’t made a dent

Gender and racial diversity at the top of UK life science companies has hardly budged over the last seven years despite repeated advocacy efforts, according to a new report.

The report, from the recruiting firm Liftstream, found that 14.8% of directors on life sciences boards were women and 21.1% of top executives were women. That’s a modest bump from the 9.8% of directors and 18.1% of executives Liftstream identified in their last report from 2014. The percentage of women CEOs moved from 8% to 9.8%.

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Will a 'risk-of­f' mind­set has­ten cell ther­a­py M&A? Io­vance surges on buy­out chat­ter

Is it time for some cell therapy M&A?

Investors of Iovance Biotherapeutics certainly thought so, sending its stock $IOVA up as much as 40% after Bloomberg reported that the cancer-focused biotech is talking to potential buyers.

While 2019 saw a number of high-profile gene therapy company takeovers — led by Roche’s $4.3 billion bid of Spark as Astellas went for Audentes, Biogen snapped up Nightstar and Vertex absorbed Exonics — large players appeared to prefer partnering on the cell therapy front, particularly when it comes to cancer. Hal Barron put his weight behind Rick Klausner’s startup as he rebuilt GlaxoSmithKline’s cancer pipeline. Takeda turned to MD Anderson to license their natural killer cell therapy.

One less ri­val for Im­muno­vant, as Alex­ion aban­dons FcRn in­hibitor

Less than one year after Alexion parted with $25 million upfront to secure access to a second anti-FcRn asset, it is abandoning the experimental drug. The discontinuation, disclosed at the SVB Leerink Global Healthcare Conference in New York during a fireside chat, bodes well for rival Immunovant.

The drug (ABY-039), partnered for development with Sweden’s Affibody, was forsaken on the basis of early-stage data that was not viewed favorably, Baird and SVB Leerink analysts noted.