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.

Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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John McHutchison in 2012. Getty Images

The $1.1M good­bye: Gilead CSO John McHutchi­son is out as Daniel O’Day shakes up the se­nior team

Just a little more than a year after John McHutchison grabbed a promotion to become CSO at Gilead in the wake of Norbert Bischofberger’s exit, he’s out amid a shakeup of the senior team that is also triggering the departure of two other top execs.

Gilead stated that McHutchison “has decided to step down” from the job as of August 2nd. And their SEC filing notes that he’ll be getting a $1.1 million check to settle up on his contract.

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Thomas Gajewski, David Steinberg. (CRI, Pyxis)

Bay­er, Long­wood back star re­searcher's deep dive in­to the tu­mor mi­croen­vi­ron­ment for new I/O tar­gets

From PD-1 targeting to the RAS pathway to the STING complex, Thomas Gajewski has spent the past two decades of his career decoding the various ways the immune system can be unleashed to defend against cancer. So when the University of Chicago professor comes around to putting all his findings into a new platform for finding new targets, VCs and pharma groups alike pay attention.

“He’s been studying T cells for 20 years, plus he’s one of the world’s leaders if not the world leader in the space,” David Steinberg, partner at Longwood Fund, said. “Furthermore, let me add he did a lot of the foundational research and also some of the seminal clinical trials in the existing set of I/O agents. He understands the space really well, he understands the current strengths, and I think he understood really well what was missing, so he knew where to look.”

Kamala Harris speaking yesterday at the Des Moines Register Iowa Presidential Candidate Forum [via Getty]

Who’s the tough­est on drug prices? A game of po­lit­i­cal one-up­man­ship is dri­ving the pol­i­cy de­bate in Wash­ing­ton

Earlier this week we got a look at Senator Kamala Harris’ position on drug prices. She’s proposing that HHS take an average price from single-payer systems like the UK, Germany and Canada — which leverage market access for lower prices — and use that to set the US price. Anything drug companies collect above that would be taxed at a rate of 100%.

And the rhetoric is scathing:
While families struggle to make it to the end of the month, pharmaceutical companies are turning record profits. They’re spending nearly as much on advertising as R&D. They’re manipulating their market power to hike prices on lifesaving generic drugs. They’re making twice the profit of the average industry in America and still increased drug prices by 10.5% over the past six months alone. Meanwhile, they are charging dramatically higher prices to American consumers.
That’s an escalation on Joe Biden’s plan, which includes drug importation from those cheaper markets as well as allowing Medicare to negotiate prices — something that virtually all Dems agree on now.

SJ Lee [File photo]

Go­ing in­side cells, Sung Joo Lee has sketched some big goals for his small — but glob­al — team of drug hunters

For a small biotech based in South Korea with a research arm in Cambridge, MA, Orum Therapeutics has sketched out some big goals aimed at developing antibodies for intracellular targets. And now they have a new $30 million round to push the work forward, aiming at a slate of currently undruggable quests.

Orum has been working on a platform tech out of Ajou University that relies on endocytosis to smuggle antibodies and their cargo inside a cell. They’ve published work in Nature that illustrates its preclinical potential in RAS mutations, and KRAS is on their list of targets. 

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Astel­las buys in­to Fre­quen­cy's re­gen­er­a­tive med strat­e­gy with a $625M al­liance on hear­ing loss

The executive team at Frequency Therapeutics never oversold the results of their maiden Phase I/II study for a new drug to rectify hearing loss. It was, they said back in April, primarily about safety and tolerability, where their drug FX-322 performed as they had hoped. 

That early glimpse of efficacy everyone searches for in their first try on humans? 

(I)mprovements in hearing function, including audiometry and word scores, were observed in multiple FX-322 treated patients.

We don’t know exactly what that means. But whatever the details, Astellas found enough in the data to jump in with a sizable collaboration deal.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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