John Ford (Enterprise)

Roche joins Ab­b­Vie in step­ping on Ver­tex's turf, bet­ting $96M on an al­ter­na­tive CF ap­proach

James Sabry’s lat­est deal spree is tak­ing Roche back to a field where Genen­tech nabbed one of its first FDA ap­provals: cys­tic fi­bro­sis.

James Sabry

Turn­ing to En­ter­prise Ther­a­peu­tics out of Brighton, Eng­land, Roche is ac­quir­ing a port­fo­lio of TMEM16A po­ten­tia­tors, which promis­es to treat all CF pa­tients re­gard­less of CFTR geno­type as well as oth­er res­pi­ra­to­ry dis­eases. The up­front comes in at $96.76 mil­lion (£75 mil­lion).

The deal makes Roche a late en­trant to a field that Ver­tex has large­ly dom­i­nat­ed with a quar­tet of drugs, capped off with the lat­est, Trikaf­ta, which cov­ers up to 90% of the CF pop­u­la­tion.

In­stead of chas­ing CFTR pro­teins like Ver­tex, though, En­ter­prise has cho­sen to go af­ter TMEM16A, an al­ter­na­tive an­ion chan­nel in air­way ep­ithe­lial cells. By en­hanc­ing it, the biotech reck­oned that its drugs can in­crease flu­id flow in­to the air­ways, thin­ning the mu­cus and in­creas­ing its clear­ance — there­by restor­ing lung func­tion and re­duc­ing the fre­quen­cy of in­fec­tions.

Im­por­tant­ly, TMEM16A po­ten­ti­a­tion is in­de­pen­dent of the mu­ta­tion­al sta­tus of CFTR. That could spell po­ten­tial in oth­er mu­co-ob­struc­tive dis­or­ders out­side of CF, added Sabry, Roche’s glob­al head of phar­ma part­ner­ing.

A first-in-hu­man study of the lead drug, an in­haled com­pound dubbed ETD002, has re­cent­ly be­gun.

The treat­ment land­scape for CF, a rare dis­ease es­ti­mat­ed to af­fect 75,000 peo­ple glob­al­ly, has evolved dra­mat­i­cal­ly since the FDA OK’d Genen­tech’s Pul­mozyme — an en­zyme that breaks up and thin mu­cus.

Even with Ver­tex in the lead and well-po­si­tioned to ex­plore next-gen treat­ments such as gene edit­ing, though, com­peti­tors are see­ing room to bring bet­ter and per­haps cheap­er drugs from be­hind. Ab­b­Vie is an­oth­er Big Phar­ma play­er in the game, first scoop­ing up Gala­pa­gos’ CF suite in a $245 mil­lion deal and lat­er adding a new CFTR po­ten­tia­tor from the CF Foun­da­tion for a com­bo.

“TMEM16A po­ten­ti­a­tion has the po­ten­tial to sig­nif­i­cant­ly in­crease the qual­i­ty of life for peo­ple liv­ing with cys­tic fi­bro­sis, for many of whom ex­ist­ing ther­a­pies are not ef­fec­tive,” John Ford, En­ter­prise CEO, said in a state­ment.

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Retrophin beefs up the rare dis­ease drug pipeline with a $517M buy­out deal

A little more than a year after Retrophin conceded the complete failure of a drug co-invented by company founder Martin Shkreli, the biotech is beefing up its rare disease pipeline through a $517 million buyout deal — fronted with $90 million in cash.

After the bell sounded Thursday, Retrophin $RTRX put out word that it’s acquiring the low-profile biotech Orphan Technologies. The buyout gives them an enzyme replacement therapy called OT-58 for the treatment of classical homocystinuria, a rare disease that is triggered by insufficient levels of an enzyme called cystathionine beta synthase.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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Jean-Pierre Sommadossi, Atea president and CEO (file photo)

Roche wades deep­er in­to Covid-19 fight, ink­ing an­tivi­ral pact with $350M cash fol­low­ing Re­gen­eron deal

Roche is making its first bet on an antiviral against Covid-19 in style, shelling out $350 million in cash to grab ex-US rights.

The drug comes from Atea Pharmaceuticals, the 7-year-old biotech created by Pharmasset co-founder Jean-Pierre Sommadossi, which essentially rebranded itself as a Covid-19 fighter in May when it closed a whopping $215 million venture round. Over a dozen investors bought in, including marquee names like Bain Capital and RA Capital.