News brief­ing: FDA lifts a hold on Po­sei­da study; Deci­bel adopts 2-AAV ap­proach to hear­ing loss; As­traZeneca part­ners on com­bos, again

A lit­tle more than 2 months ago, shares of new­ly pub­lic Po­sei­da Ther­a­peu­tics $PSTX cratered af­ter the biotech re­port­ed that a pa­tient death had spurred the FDA to drop a full hold on its lead can­cer drug. This morn­ing we learned that reg­u­la­tors lift­ed the hold, but de­tails are sparse.

In a state­ment out ear­ly Mon­day, Po­sei­da said that reg­u­la­tors had lift­ed the hold on their Phase I study for their CAR-T P-PS­MA-101 in prostate can­cer. Here’s why:

The Com­pa­ny has agreed to im­ple­ment pro­to­col amend­ments in­tend­ed to in­crease pa­tient com­pli­ance and safe­ty that in­clude mod­i­fied in­clu­sion and ex­clu­sion cri­te­ria and fre­quen­cy of mon­i­tor­ing and lab­o­ra­to­ry test­ing.

So there’s no ex­pla­na­tion of what had caused the death or ex­act­ly what was be­ing done to cor­rect the tri­al de­sign.

Deci­bel snags IP for a 2-AAV ap­proach to fix­ing con­gen­i­tal hear­ing loss

Deci­bel Ther­a­peu­tics thinks it has found a strategy to tack­le a gene ther­a­py for con­gen­i­tal hear­ing loss.

Con­front­ed by the chal­lenge of de­liv­er­ing the large otofer­lin gene to fix the pro­found hear­ing loss trig­gered by mu­ta­tions of the gene, re­searchers at the Uni­ver­si­ty of Flori­da and UC San Fran­cis­co have come up with a 2 AAV ap­proach to the prob­lem. They are us­ing 2 vec­tors to de­liv­er the full gene.

And Deci­bel is in-li­cens­ing the tech from William Hauswirth and Omar Ak­il to try it in hu­mans.

Hauswirth notes:

We were able to cure deaf­ness in a mouse mod­el and look for­ward to the po­ten­tial of this tech­nol­o­gy in the de­vel­op­ment of a gene ther­a­py to re­store hear­ing in hu­man pa­tients.

Fu­sion part­ners up with As­traZeneca on a new com­bi­na­tion strat­e­gy for can­cer

A few days af­ter As­traZeneca $AZN tied up with Ar­cus $RCUS to test a com­bi­na­tion of their PD-L1 check­point Imfinzi with the biotech’s an­ti-TIG­IT ther­a­py, the phar­ma gi­ant is back with another al­liance aimed at ra­dio­phar­ma­ceu­ti­cals.

Fu­sion Phar­ma­ceu­ti­cals is part­ner­ing their lead drug off the Tar­get­ed Al­pha Ther­a­pies plat­form with drugs out of As­traZeneca. Their teams will de­vel­op nov­el TATs as well as an­ti­bod­ies from the glob­al com­pa­ny. And like the Ar­cus deal, both com­pa­nies will re­tain full own­er­ship of their prod­ucts.

Fu­sion CEO John Val­liant not­ed: We be­lieve (this deal) is ev­i­dence of the grow­ing in­ter­est in the use of mol­e­c­u­lar­ly-tar­get­ed ra­di­a­tion as a next-gen­er­a­tion can­cer ther­a­py.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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