FDA's con­tro­ver­sial OK for eteplirsen spurs sec­ond thoughts for a re­ject­ed ri­val at Bio­Marin

Jean-Jacques Bi­en­aimé, Bio­Marin CEO

Sarep­ta’s reg­u­la­to­ry win for Ex­ondys51 (eteplirsen) left one group of an­a­lysts and FDA in­sid­ers more than a lit­tle flab­ber­gast­ed.

There was on­ly a mi­nus­cule amount of da­ta to look at. And af­ter all, PTC nev­er even got in the doors at the FDA, while the agency had no trou­ble slap­ping down Bio­Marin’s ap­pli­ca­tion for dris­apersen, which had more da­ta — in­clud­ing its fail­ure in clin­i­cal stud­ies and trou­bling safe­ty signs.

In­spired by the eteplirsen de­ci­sion, Bio­Marin $BM­RN is re­think­ing its de­ci­sion to heave dris­apersen off to the scrap yard. The rare dis­ease spe­cial­ist is mulling the no­tion of ap­peal­ing its FDA re­jec­tion for its ri­val Duchenne mus­cu­lar dy­s­tro­phy drug, ac­quired in the Pros­en­sa buy­out for $680 mil­lion. In a state­ment to End­points News, the com­pa­ny said:

The de­ci­sion by the FDA to is­sue a Com­plete Re­sponse Let­ter for dris­apersen is one that can be ap­pealed.  Bio­Marin is ex­plor­ing its op­tions.  The com­pa­ny has not de­ter­mined if it will pur­sue an ap­peal.

The FDA’s ap­proval for Ex­ondys51 is shap­ing up as one of the most con­tro­ver­sial on record. Janet Wood­cock’s stead­fast sup­port for Sarep­ta $SRPT and its drug trig­gered a war in­side the agency, as se­nior-lev­el of­fi­cials ridiculed the lack of ev­i­dence on ef­fi­ca­cy and ques­tioned the safe­ty pro­file that Sarep­ta had pre­sent­ed. The FDA com­mis­sion­er even chid­ed Sarep­ta for us­ing a clear­ly dodgy study to back its ap­pli­ca­tion, but de­ferred to Wood­cock’s ex­pe­ri­ence in the field in al­low­ing the ap­proval to go through.

Even the sug­ges­tion of a new shot at an ap­proval for dris­apersen, though, trig­gered with­er­ing re­marks on Twit­ter over the tox­i­c­i­ty is­sues tied to the drug. Sarep­ta’s share price soared on the FDA’s ap­proval, leav­ing it in sole com­mand of the mar­ket for a drug that costs $300,000 a year. Any per­ceived threat to that, how­ev­er slight, by bring­ing back this trou­bled ap­pli­ca­tion was in line for a back­lash.

It may be a long shot, but Bio­Marin al­so has noth­ing much to lose at this point if it de­cides to move ahead.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: 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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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

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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News brief­ing: UK biotech 4D phar­ma heads for Nas­daq via SPAC; Dr. Red­dy's shuts down man­u­fac­tur­ing af­ter cy­ber­at­tack

Another pharma company is intending to use a SPAC to join the Nasdaq.

4D pharma, a UK-based biotech, is reverse-merging with a blank check company in a deal worth up to $37.6 million. The move will give 4D pharma a new Nasdaq ticker, which will be $LBPS, using the American Depositary Share program.

As a result of the move, 4D pharma will gain $14.6 million in cash held by the blank check company, dubbed Longevity. The merger is expected to be completed in early 2021, after which shares will be immediately tradeable.

Can B cells break the bound­aries of cell ther­a­py? Long­wood start­up has $52M to prove a new en­gi­neer­ing tech

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Children’s Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

Jason Kelly, Gingko Bioworks CEO (Mike Blake/Reuters via Adobe)

Ex­clu­sive: Eye­ing big Covid-19 test­ing ex­pan­sion, Gink­go rolls out 50M rapid anti­gen di­ag­nos­tics

In what they hope will be a key part of an extensive effort to boost Covid-19 testing in the US, Ginkgo Bioworks is acquiring and distributing 50 million rapid antigen tests that can potentially be used for virus surveillance in schools and communities and for quick, on the ground diagnoses.

The tests, developed by SD Biosensor, are in line with proposals from the Rockefeller Foundation and Harvard epidemiologists, among others, to blanket the country with fast, low-cost tests. Although not yet authorized in the US, they are a key part of testing efforts from the Bill and Melinda Gates Foundation, who announced plans last month to distribute 120 million of them in low and middle income countries. Roche has commercialized the diagnostic in Europe.

Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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HHS secretary Alex Azar (at the podium) and FDA commissioner Stephen Hahn (Pete Marovich/Getty Images)

Covid-19 roundup: Azar open­ly plan­ning Hahn ouster — re­port; Vul­ner­a­ble pop­u­la­tions like­ly to get vac­cines by Jan­u­ary

The relationship between HHS secretary Alex Azar and FDA commissioner Stephen Hahn has deteriorated to the point where Azar has suggested replacing Hahn, according to a Politico report.

Azar was angered by the FDA’s pushback of the Trump administration’s proposals for authorizing Covid-19 vaccines, so much so that he began openly floating potential replacements for Hahn. The report cited six unnamed sources that said Azar discussed bringing up Hahn’s removal to the White House.

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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.