Oc­cu­pied with im­munol­o­gy and can­cer, Mer­ck KGaA hands off os­teoarthri­tis drug to No­var­tis for €50M cash

By the time Mer­ck KGaA pub­lished promis­ing Phase II da­ta for sprifer­min — once tapped as a big prospect for the ail­ing R&D group — late last year, the Ger­man drug­mak­er made clear it was look­ing for a part­ner to take over its whole os­teoarthri­tis pipeline. While sprifer­min is still sit­ting in the port­fo­lio, it’s man­aged to find a buy­er for an­oth­er drug.

No­var­tis is pay­ing €50 mil­lion up­front to li­cense M6495, a Phase II-ready com­pound that tar­gets an en­zyme known as ADAMTS5, with promis­es of €400 mil­lion in mile­stones.

Lu­ciano Ros­set­ti

The deal comes more than three years af­ter Mer­ck KGaA grabbed the Nanobody from Abl­ynx — since ac­quired by Sanofi — with a €15 mil­lion pay­ment to re­ward the pre­clin­i­cal proof-of-con­cept pack­age. Then known as ALX-1141, the can­di­date was one of two to emerge from a col­lab­o­ra­tion dat­ing back to 2011 to tack­le os­teoarthri­tis.

Since then Lu­ciano Ros­set­ti’s team has com­plet­ed two Phase I stud­ies for M6495, one in healthy vol­un­teers and an­oth­er in OA pa­tients. With signs that the drug could cut down ARGS (a neoepi­tope from cleaved ag­gre­can, found in the syn­ovial flu­id and serum of OA pa­tients) lev­els, the drug had po­ten­tial in many dif­fer­ent types of OA, he said.

The ul­ti­mate goal, of course, is to show it can pro­tect against car­ti­lage dam­age and re­duce joint pain. But it’s now No­var­tis’ job to de­vise the tri­als need­ed to prove it.

“With this deal we have found the right so­lu­tion for this as­set de­signed to present an in­no­v­a­tive mech­a­nism of ac­tion for the po­ten­tial treat­ment of os­teoarthri­tis, as we pri­or­i­tize our pipeline to de­liv­er the great­est im­pact for pa­tients across our in­ter­nal ar­eas of ex­per­tise,” Ros­set­ti, head of glob­al bio­phar­ma R&D, said in a state­ment.

Un­der Belén Gar­i­jo, who’s step­ping up as Mer­ck KGaA’s CEO, the pri­or­i­ties will like­ly re­volve heav­i­ly around on­col­o­gy, im­munol­o­gy and the in­ter­sec­tion of the two.

For No­var­tis, OA falls un­der its fo­cus on im­munol­o­gy, he­pa­tol­ogy and der­ma­tol­ogy. Its web­site lists two ear­ly-stage pro­grams ded­i­cat­ed to the dis­ease, one be­ing an ANGPTL3 ag­o­nist de­signed to help re­gen­er­ate car­ti­lage. The Phase I study was com­plet­ed in 2018 and a sec­ond tri­al is slate to wrap in 2022.

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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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.

Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.