FDA re­quests ad­di­tion­al time for Flex­ion's sN­DA; Genen­tech re­leas­es da­ta for PhI­II study of Rit­ux­an

In 2017, the FDA green­light­ed Flex­ion Ther­a­peu­tics‘ in­tra-ar­tic­u­lar ther­a­py, Zil­ret­ta, de­signed as a treat­ment for os­teoarthri­tis-re­lat­ed knee pain. Now, two years lat­er, the com­pa­ny has an­nounced that the FDA needs ad­di­tion­al time to com­plete the re­view of its sup­ple­men­tal NDA for Zil­ret­ta. The an­tic­i­pat­ed PDU­FA date was set for to­day. Al­though the FDA has not pro­vid­ed a new ac­tion date, Flex­ion says they were in­formed that the re­view should be com­plet­ed in the com­ing weeks. No ad­di­tion­al clin­i­cal da­ta has been re­quest­ed by the FDA.

Genen­tech has re­leased da­ta from its Phase III PEM­PHIX study eval­u­at­ing the ef­fi­ca­cy and safe­ty of Rit­ux­an (rit­ux­imab) com­pared to my­cophe­no­late mofetil (MMF), in adults with mod­er­ate to se­vere pem­phi­gus vul­garis (PV). The da­ta showed su­pe­ri­or re­sults in pa­tients giv­en Rit­ux­an, with 40.3% of pa­tients treat­ed with Rit­ux­an achiev­ing sus­tained com­plete re­mis­sion with­out the use of steroids for 16 con­sec­u­tive weeks or more, com­pared to 9.5% in the MMF arm (p<0.0001). The stud­ies pri­ma­ry end­point was reached at week 52.

John­son & John­son‘s drug Xarel­to (ri­varox­a­ban) has been ap­proved by the FDA for the pre­ven­tion of ve­nous throm­boem­bolism (VTE), or blood clots, in hos­pi­tal­ized, acute­ly ill med­ical pa­tients at risk for throm­boem­bol­ic com­pli­ca­tions who are not at high risk of bleed­ing. The ap­proval comes a few months af­ter J&J and Bay­er paid out $775 mil­lion to thou­sands of Xarel­to pa­tients af­ter a 5-year long le­gal war — the com­pa­nies were ac­cused of fail­ing to warn pa­tients prop­er­ly of the bleed­ing risk as­so­ci­at­ed with the blood thin­ner.

Ul­tragenyx Phar­ma­ceu­ti­cal has an­nounced that the FDA has ac­cept­ed their NDA for UX007 (tri­hep­tanoin) for the treat­ment of long-chain fat­ty acid ox­i­da­tion dis­or­ders. The PDU­FA is set for Ju­ly 31, 2020.

As­traZeneca, Pfiz­er and Take­da have come to­geth­er for a col­lab­o­ra­tion deal with Imag­inAb, agree­ing to guide a clin­i­cal tri­al for the imag­ing play­er in ex­change for ear­ly ac­cess to the da­ta. The study is aimed at as­sess­ing the util­i­ty and val­ue of Imag­inAb’s Im­munoPET tech­nol­o­gy, which us­es mi­ni an­ti­bod­ies and tags CD8+ T cells with ra­dioiso­topes to ren­der im­mune ac­tiv­i­ty vis­i­ble in a PET scan.

“One of our key ob­jec­tives is to stream­line the clin­i­cal de­vel­op­ment of next-gen­er­a­tion can­cer im­munother­a­pies so that ul­ti­mate­ly can­cer pa­tients have ac­cess to the best pos­si­ble treat­ments,” CEO Ian Wil­son said in a state­ment. “We be­lieve that work­ing with glob­al lead­ers in im­muno-on­col­o­gy will help us fur­ther de­vel­op CD8 Im­munoPET as a phar­ma­co­dy­nam­ic mark­er for use in drug de­vel­op­ment and, in the fu­ture, as a di­ag­nos­tic and pre­dic­tive test for use in hos­pi­tals.”

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

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Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Charlie Fuchs, Roche and Genentech global head of product development for oncology and hematology (Yale Cancer Center)

Yale can­cer spe­cial­ist Char­lie Fuchs tapped as new glob­al de­vel­op­ment chief for Roche/Genen­tech

Roche and their big sub Genentech have just recruited a top cancer specialist at Yale to head up global product development in oncology and hematology.

I just got word that the pharma giant, which leads one of the most active cancer research operations in the world, recruited Charlie Fuchs, director of the Yale Cancer Center and physician-in-chief of Smilow Cancer Hospital. He’ll join the global operation March 1 and will be based in South San Francisco, where Genentech is based.

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News brief­ing: Eli Lil­ly com­pletes $1B+ Pre­vail buy­out; El­zon­ris ap­proved in Eu­rope for adults

Eli Lilly’s $1.04 billion takeover of Prevail Therapeutics is officially complete, the company announced Friday.

The sides had entered into the buyout agreement last month with Lilly focusing on Prevail’s pipeline of gene therapies, highlighting two AAV9 programs in Parkinson’s disease and frontotemporal dementia as potential winners. Lilly paid $22.50 per share, which amounted to an 82% premium over the previous day’s closing price and a 117% premium over Prevail’s 60-day average.

Jonathan Weissman (MIT)

Can a new CRISPR tech­nique un­lock the se­crets of how can­cer spreads?

Jonathan Weissman’s team watched the cancer cells spread across the doomed mouse. Engineered with a bioluminescent enzyme, they appeared in scans first as a small navy blue diamond lodged near the heart; a week later, as a triangle splayed across the mouse’s upper body, with streaks of green and two distinct bright red hubs of activity. By day 54, the mouse resembled a lava lamp.

The images would have been familiar to any cancer biologist, but they didn’t actually tell you much about what was going on: why the cancer was metastasizing or which cells were responsible. For that, Weissman’s team had designed a new tool. Inside the original navy blue diamond, they had engineered the microbiological equivalent of an airplane’s black box — a “molecular recorder” that, after the mouse’s death, could allow them to extract the cells and wind back intimate footage of a single cancer’s ascent.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.