Eye­ing Duchenne MD cure, The Col­umn Group gam­bles $40M on CRISPR/Cas9 start­up Ex­on­ics

The Col­umn Group thinks that gene edit­ing may pro­vide just the right kind of tech­nol­o­gy need­ed to cure Duchenne mus­cu­lar dy­s­tro­phy. And the in­vest­ment group is wa­ger­ing $40 mil­lion that the pre­clin­i­cal mouse da­ta used to back biotech start­up Ex­on­ics is point­ing in the right di­rec­tion.

John Rip­ple

This in­fu­sion of cash is a big boost for Ex­on­ics, a Cam­bridge, MA-based com­pa­ny which start­ed out with $5 mil­lion in seed mon­ey from Cure­Duchenne. But their back­ers in San Fran­cis­co are not just hand­ing the funds off, Ex­on­ics CEO John Rip­ple told End­points News. In ad­di­tion to bankrolling the pre­clin­i­cal re­search (much of which will be op­ti­miz­ing the cur­rent tech­nol­o­gy), it will al­so be piv­otal in ex­pan­sion ef­forts as Ex­on­ics looks to start a lab in Cam­bridge to sup­ple­ment founder Er­ic Ol­son’s re­search at UT South­west­ern.

“With Dr Ol­son and his team’s ex­per­tise there at UT South­west­ern, and with the team that we’ve been able to build here at Ex­on­ics, and now the fund­ing that we have, we think we’re very well po­si­tioned to at least be a leader in this field,” Rip­ple said, “and to hope­ful­ly be the first, or one of the first, to bring promis­ing treat­ments that will ad­dress the cause of Duchenne mus­cu­lar dy­s­tro­phy for these boys.”

The goal is for the new lab to be op­er­a­tional ear­ly next year. One undis­closed sci­en­tist from Ol­son’s team will head the fa­cil­i­ty, and Ex­on­ics is in the process of scout­ing oth­er tal­ents, with the help of TCG’s re­cruit­ment staff.

David Goed­del

Ad­di­tion­al­ly, two TCG mem­bers will be join­ing Ex­on­ics’ board of di­rec­tors. One of them is man­ag­ing part­ner David Goed­del, the first sci­en­tist hired by Genen­tech, who co-found­ed Tu­larik —a gene reg­u­la­tion com­pa­ny lat­er ac­quired by Am­gen. He is al­so serv­ing on mul­ti­ple boards in­clud­ing NGM Bio and Tenaya.

“The com­pa­ny has gen­er­at­ed com­pelling ear­ly da­ta, and we are pleased to sup­port Ex­on­ics as it ad­vances its pre­clin­i­cal de­vel­op­ment pro­gram in Duchenne clos­er to the clin­ic,” Goed­del said in a state­ment.

An ear­ly adopter of CRISPR/Cas-9 in treat­ing DMD, Ex­on­ics was found­ed on the promise to iden­ti­fy and re­pair a range of ex­on mu­ta­tions — not just ex­on 51, which FDA-ap­proved Ex­ondys51 tar­gets — that pre­vent the pro­duc­tion of dy­s­trophin, the mus­cle fiber pro­tect­ing pro­tein miss­ing in chil­dren with DMD.

Since Ex­on­ics launched in Feb­ru­ary, and even be­fore, oth­er com­pa­nies have been de­vel­op­ing their own gene ther­a­py for DMD. Sarep­ta — which sells con­tro­ver­sial Ex­ondys51  — an­nounced a cou­ple weeks back that it’s part­ner­ing with Duke to ex­plore its own CRISPR tech­nol­o­gy. This high­lights the ur­gent un­met need for im­proved DMD treat­ment, and while Rip­ple ac­knowl­edges that, he said Ex­on­ics want to en­sure they have it right.

“We are try­ing to de­vel­op an op­ti­mal treat­ment for these boys to make sure that we have the best pos­si­ble treat­ment when we do en­ter the clin­ic and give that first ad­min­is­tra­tion to these boys,” he said.

Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.

Ronald Herb­st fol­lows Med­Im­mune ex­o­dus to Pyx­is CSO post; Jeff God­dard to suc­ceed CEO of AIT Bio­science

→ The outflow of top execs from MedImmune continues to fill the leadership ranks of smaller biotechs. The latest to take off is Ronald Herbst, the head of oncology research, who’s assuming the CSO post at Pyxis Oncology.  

Herbst was part of the old MedImmune organization AstraZeneca CEO Pascal Soriot restructured earlier this year, reorganizing the company and eliminating the storied subsidiary as a separate organization.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

UP­DAT­ED: J&J's Xarel­to, Amar­in's Vas­cepa are cost-ef­fec­tive, not bud­get friend­ly — ICER

ICER, an increasingly influential cost-effectiveness watchdog in the United States, has concluded in its review of treatments for cardiovascular disease that while the cost of J&J’s Xarelto and Amarin’s Vascepa meet its benchmark for value pricing — the two treatments will not likely treat as many patients as hoped without surpassing the annual budget threshold calculated by ICER for each therapy.