CRISPR Ther­a­peu­tics, Ver­tex set for first sick­le cell dis­ease tri­al af­ter FDA lifts clin­i­cal hold — just don't look for any ex­pla­na­tion

CRISPR Ther­a­peu­tics and Ver­tex have re­solved a clin­i­cal hold on a high-pro­file gene edit­ing pro­gram — but they’re still not ready to di­vulge much in the way of de­tails about the tem­po­rary de­rail­ment they got slapped with five months ago.

The part­ners say the FDA has now giv­en the green light to test their sick­le cell dis­ease treat­ment in hu­mans. That will like­ly ex­pand the Phase I/II study the com­pa­nies are on track to ini­ti­ate by the end of the year, adding to sites in the UK and Cana­da, where they have se­cured clin­i­cal tri­al ap­provals.

The an­nounce­ment was out just af­ter mar­ket close Wednes­day, car­ry­ing CRISPR shares $CR­SP up as much as 15% af­ter hours. It is how­ev­er down again in pre-mar­ket trad­ing, as is Ver­tex $VRTX, which saw a drop of al­most 5%.

If suc­cess­ful, the tri­al would mark the first clin­i­cal val­i­da­tion for CRISPR’s ex vi­vo gene edit­ing ap­proach, which Ver­tex paid $105 mil­lion to part­ner on back in 2015.

Sci­en­tists are still try­ing to un­der­stand the po­ten­tial harms of the tech. For Ver­tex — which has built its name large­ly on cys­tic fi­bro­sis — it’s al­so the first bud on its new he­mo­glo­binopa­thy branch.

CTX001, which in­volves en­gi­neer­ing a pa­tient’s hematopoi­et­ic stem cells such that they pro­duce high lev­els of fe­tal he­mo­glo­bin (or he­mo­glo­bin F), will al­so be in­ves­ti­gat­ed as a ther­a­py for be­ta-tha­lassemia. En­roll­ment has be­gun in Eu­rope.

In a brief state­ment, CRISPR and Ver­tex re­main tight-lipped about why reg­u­la­tors were con­cerned or what they did to re­as­sure them.

A Ver­tex spokesper­son has this to say in re­sponse to my query: “We can’t share more at this point be­yond the fact that we and CRISPR Ther­a­peu­tics had a pro­duc­tive di­a­logue with the FDA over re­cent months and an­swered the ques­tions raised in their ini­tial re­view of the IND.”

Jake Van Naarden, Josh Bilenker, Nisha Nanda (Credit: Loxo, Aisling Capital)

Josh Bilenker and his Loxo crew are tak­ing the reins on on­col­o­gy R&D at Eli Lil­ly, culling the weak and map­ping a new path

Josh Bilenker, Jake Van Naarden and Nisha Nanda came out of Eli Lilly’s $8 billion Loxo Oncology buyout with a bundle of cash and plenty of choices on what they could do next. Start a new company, go public. Live on the beach in 5-star luxury. Contemplate the stars — in their own observatory.

So what are they doing?

They formed a new executive team that is taking over the management of Eli Lilly’s hundreds-strong oncology R&D group — essentially using Loxo as a base for a bold new experiment in Big Pharma R&D in an attempt to create a true biotech environment with the deep pockets of a top-15 industry player. They’ve recruited David Hyman from Memorial Sloan Kettering to join the team as chief medical officer. And the mandate includes culling out the oncology pipeline, highlighting their star prospects and going after new programs wherever they can find the best prospects.

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One of Wall Street’s most high-pro­file hedge funds push­es Alex­ion's CEO to the auc­tion block — and he's not budg­ing

Fresh off buying Barnes & Noble and prodding AT&T with some heavy-handed criticism after picking up a $3.2 billion stake in the company, the activist — and supremely high profile — hedge fund Elliott Management has stepped up with some M&A advice for Alexion’s management team.
And the execs on the team $ALXN are giving them a polite — but very firm — stiff arm Friday morning.
In a release out early Friday, the big biotech said that the Elliott team had been in touch to encourage them to sell the company. But that’s not on the agenda.

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Un­lock the full End­points ex­pe­ri­ence for your com­pa­ny — and sup­port our mis­sion of in­de­pen­dent bio­phar­ma re­port­ing

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Samantha Budd Haeberlein. Biogen via YouTube

UP­DAT­ED: Skep­tics pounce as Bio­gen de­tails pos­i­tive sub­group analy­sis on ad­u­canum­ab — and both sides are dig­ging in

“Exhilarating.” “A major advance.” “A milestone achievement.” If one had just tuned into the panel comments on Biogen’s presentation at CTAD, it would seem that the biotech had an impressive, disease-modifying Alzheimer’s drug in aducanumab.

But off the stage, reactions to their admittedly complicated dataset and the biotech’s explanation for resurrecting a drug that failed its futility analysis were a lot more mixed, with analysts continuing to question whether the evidence is substantial enough to warrant an FDA approval and raising new doubts on the safety side.

In an investor call later in the day, execs noted that they are not planning another study and stood by their intention, publicized in October to much surprise, to submit regulatory filings based on what they have.

“We don’t file willy nilly,” said Al Sandrock, head of R&D. “We only go to filing when we believe that there is a benefit-risk argument based on science, based on data. And if you look at our history, we haven’t done filings right and left without good reason.”

Biogen had a theory going into the Clinical Trials on Alzheimer’s Disease meeting.

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No­var­tis CEO Vas Narasimhan's R&D up­date spot­lights next wave of drug stars as well as late-stage fa­vorites

As one of the biggest spenders in biopharma R&D, Novartis execs love to tout the scope of its late-stage pipeline, spotlighting the winners most likely to create blockbuster revenue streams in the near future.

Building on the 5 drug approvals the pharma giant expects to end the year with, Novartis CEO Vas Narasimhan — who’s done a slate of acquisitions topped by the recent $9.7 billion MedCo buyout — tapped the top emerging drugs as:

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Jeff Jonas, Sage

UP­DAT­ED: Sage's star ex­per­i­men­tal de­pres­sion drug fails the cru­cial MOUN­TAIN study — shares crash

Sage Therapeutics’ crucial MOUNTAIN study for Sage-217 has failed, setting the stage for a quick and ugly investor backlash.

Widely viewed by analysts as the critical clinical study $SAGE needed to win on major depression, researchers say the drug failed to beat out a placebo at day 15, falling well short of the mark for statistical significance on the primary endpoint. And investors reacted with alacrity, fleeing the stock and gutting the price with a 60% instantaneous drop — erasing about $4.6 billion in market cap in an instant.

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Jasper Ther­a­peu­tics launch­es out of Stan­ford with new ap­proach to stem cell treat­ment

The first girl in the trial came in with chronic diarrhea and the immune system of an untreated HIV patient. Born with a rare genetic disease that impeded her ability to make B and T cells, she had once been given a stem cell transplant but it didn’t take.  Back in the hospital, she was injected with a new experimental antibody and then given a new stem cell transplant. Soon, she gained weight. The diarrhea stopped.

Ex-Cel­gene ex­ec Ter­rie Cur­ran puts her Phath­om team in place; Car­away taps Mar­tin Williams as CEO

→ Gastrointestinal disease-focused Phathom Pharmaceuticals has shaken up its leadership team. The company has tapped former Celgene exec Terrie Curran as CEO, succeeding David Socks, who is transitioning to interim CFO. Curran was president of Celgene’s global inflammation and immunology franchise — helping with the sale of Otezla for $13.4 billion to Amgen — and has held a previous stint at Merck. In addition to Curran, the company also welcomed former Omeros CMO Eckhard Leifke as CMO, ex-Celgene exec Joseph Hand as chief administrative officer, and former general counsel for Cyclerion Therapeutics Larry Miller as general counsel. They also replaced Chris Slavinsky on the board with Takeda exec Asit Parikh.

UCB buffs up in block­buster pso­ri­a­sis race as bimek­izum­ab beats Hu­mi­ra in head-to-head

Just weeks after boasting head-to-head victories over first placebo and then J&J’s IL-23 contender Stelara in clearing psoriasis, the results are in for UCB’s last Phase III trial, in which bimekizumab went up against the world’s best-selling drug.

Only topline results are provided for today’s readout of the BE SURE study, so we won’t find out just how superior bimekizumab proved against Humira on the co-primary endpoints — standard scores known as PASI90 and IGA measuring the impact and severity of the disease — until a scientific conference in 2020.