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Barry Greene, Sage CEO

UP­DAT­ED: Sage's sec­ond chance at de­pres­sion hits the PhI­II pri­ma­ry, but ques­tions re­main over dura­bil­i­ty, side ef­fects

Looking to make a comeback after a big Phase III flop, Sage Therapeutics revealed data they believe could change the entire depression treatment landscape, given the vast array of failures in the field. But some results are spooking investors, sending Sage $SAGE shares down early Tuesday.

First, the primary: Sage and Biogen reported Phase III data for once-daily zuranolone Tuesday morning, saying the experimental drug hit its primary endpoint by spurring a statistically significant change from baseline in the 17-item Hamilton Rating Scale for Depression total score. After 15 days, patients in the drug arm saw an average change of -14.1 points, compared to -12.3 on placebo.

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Bio­gen sig­nals a big PhI­II fail­ure as the lead gene ther­a­py in their $800M Night­star buy­out goes down in flames

That $800 million buyout of Nightstar has turned into a bust for Biogen as the lead therapy in the deal failed a pivotal study, signaling a severe setback for the biotech’s ambitions in gene therapies.

The big biotech put out the word after the market closed on Monday that the gene therapy they picked up in the deal for a degenerative blindness called choroideremia failed the Phase III study, just a month after their #2 drug in the deal also flopped in a mid-stage study.

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Terns Phar­ma­ceu­ti­cals touts safe­ty da­ta from an ex-Eli Lil­ly can­di­date in the hunt for NASH treat­ment

While many others have tried — and failed — to get a NASH candidate across the finish line, Terns Pharmaceuticals thinks its FXR agonist will eventually earn its wings without the safety issues that have slowed others down. Now, a mid-stage safety readout could help add some validity to those hopes.

No patients in the Phase IIa LIFT study discontinued TERN-101 due to side effects, CMO Erin Quirk said during a call with investors on Monday morning. That includes pruritus, an uncomfortable itching sensation that frequently leads patients to drop out of other FXR agonist studies.

In next-gen BTK bat­tle, Ab­b­Vie and J&J tout piv­otal da­ta back­ing Im­bru­vi­ca as a com­bo treat­ment

Last week, AstraZeneca took a swing at J&J and AbbVie’s first-generation BTK inhibitor Imbruvica in the blood cancer chronic lymphocytic leukemia with long-term data from a head-to-head trial. But the partners aren’t going down without a fight.

AbbVie and Janssen unveiled pivotal data on Saturday showing that first-line CLL patients who received Imbruvica and AbbVie’s Roche-partnered Venclexta lived longer without disease progression than patients on Gazyva and the chemotherapy chlorambucil. The Phase III GLOW trial enrolled 211 elderly patients, or those with comorbidities or concurrent illnesses. Overall, the median age was 71 years old.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Months af­ter FDA re­jec­tion, Sanofi touts piv­otal win for rare dis­ease drug su­tim­limab as it preps to re­file

One of the pillar drugs of Sanofi’s $11.6 billion pickup of Bioverativ hit a big setback late last year when the FDA sent its application for approval back. Now, as Sanofi gears up to resubmit the drug for review, the drugmaker is touting pivotal data it hopes will help take it over the finish line.

Sanofi’s sutimlimab nailed all three of its primary endpoints in its Phase III CADENZA study for patients with cold agglutinin disease, a rare disorder that can cause severe anemia, without a recent history of blood transfusion, the French drugmaker said Friday. The topline results will be presented at this weekend’s virtual EHA meeting.

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Ver­tex and CRISPR Ther­a­peu­tics un­veil more pos­i­tive gene ther­a­py da­ta, but busul­fan again casts a shad­ow over the field

Less than 12 hours after revealing a flop on its second shot for alpha-1 antitrypsin deficiency, Vertex plowed ahead with another data drop from its partnership with CRISPR Therapeutics. And though the topline proved positive, concerns over conditioning agents continue to linger over the collaboration, as well as the entire gene therapy space.

Presenting data from two trials at the European Hematology Association annual meeting, the pair announced that follow-up data of at least three months for 22 patients with genetic blood disorders indicated a “consistent and sustained” response to the experimental drug CTX001. All 15 patients with transfusion-dependent beta thalassemia did not need further blood transfusions and all seven with severe sickle cell disease were pain free, the biotechs announced.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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Sadasivan Vidyasagar (Credit: University of Florida)

An API firm with a his­to­ry of J&J fund­ing scores a ma­jor loan to ad­vance its port­fo­lio

A Massachusetts-based company that manufactures APIs closed its Series A and B fundraising, with J&J among the list of investors. Now, the company is announcing another $49 million loan to help boost operations.

Entrinsic Bioscience — or EBS — expects to use the loan to fund programs for GI, airway and skin diseases. CEO Stephen Gatto called these programs a “blueprint for serial innovation” in a press release.

Michel Vounatsos (Credit: World Economic Forum/Ciaran McCrickard)

With its con­tro­ver­sial ad­u­canum­ab ap­proval in hand, Bio­gen gears up for a quick US roll­out

In what will prove to be a controversial decision for years to come, the FDA earlier this week approved Biogen’s aducanumab for Alzheimer’s disease despite some mixed — to say the least — data supporting its use. But Biogen has stayed confident it would secure that nod, and now all the manufacturing and logistics prework the drugmaker did to gear up for launch should spell a quick rollout.

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Bris­tol My­er­s' CAR-T Breyanzi busts out a win in ear­li­er-line lym­phoma, po­ten­tial­ly crack­ing open an ex­pand­ed mar­ket

Despite being third to the field in B cell lymphoma, Bristol Myers Squibb has repeatedly argued its CAR-T Breyanzi could have the juice to overtake its older competitors. Going into earlier lines of therapy may be the golden ticket on that front, and now Breyanzi has a late-stage win to back up that effort.

Bristol Myers’s Breyanzi beat out physicians’-choice salvage therapy followed by high-dose chemo and a stem cell transplant — what the drugmaker called a “gold standard treatment” — in second-line patients with relapsed or refractory large B cell lymphoma, according to topline data from the Phase III TRANSFORM study released Thursday.