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UP­DAT­ED: As ri­vals march on, No­var­tis se­cures pri­or­i­ty FDA re­view for sick­le cell dis­ease ther­a­py

About a month after rival Global Blood Therapeutics $GBT unveiled positive pivotal data on its sickle cell disease drug, Novartis’ $NVS therapy for the blood disorder has been granted a speedy review by the FDA.

Touted by Novartis chief Vas Narasimhan as a potential blockbuster, the Swiss drugmaker’s drug, crizanlizumab, in January secured the FDA’s breakthrough therapy designation for its ability to prevent debilitatingly painful vaso-occlusive crisis (VOCs) for patients affected by the group of inherited red blood cell disorders that typically afflict those of African ancestry.

Mi­cro­cap Capri­cor soars on in­ter­im PhII DMD da­ta show­ing func­tion­al ben­e­fit for old­er pa­tients

With the interim analysis for its Phase II Duchenne muscular dystrophy, little Capricor Therapeutics was merely looking for a guidepost, a signal, a trend that the effects seen in an earlier trial can be replicated with a new dosing regimen and delivery method. Instead, the biotech found itself blessed with a basket of data points that seem to buck the natural history trend and separate the drug from placebo in a statistically significant way, more than doubling its tiny stock $CAPR in pre-market trading.

Eton Phar­ma hand­ed CRL for re­for­mu­lat­ed con­junc­tivi­tis eye drop

Eton Pharma’s grand plan to launch an over-the-counter for an ocular itch that often afflicts children has hit a dead end at the FDA, which has issued a complete response letter for EM-100.

The CRL is also bad news for Bausch & Lomb — the eye care specialist subsidiary of the company formerly known as Valeant — which had bagged the US rights to the conjunctivitis drug back in February.

“If approved, EM-100 will be the first preservative-free allergy eye drop available in the United States and will allow us to better serve the millions of Americans that experience itchy eyes due to allergies,” Joseph Papa, CEO of Bausch Health, said back then.

FDA bats back As­traZeneca's SGLT di­a­betes drug for Type 1 di­a­betes — block­ing a class on safe­ty fears

The FDA has just fired its latest salvo at the SGLT class of diabetes drugs, blowing up some commercial opportunity at AstraZeneca as part of the collateral damage.

The pharma giant reported early Monday that the FDA has rejected its blockbuster drug Farxiga for Type 1 diabetes that can’t be controlled by insulin. And while the pharma giant maintained its usual grim silence in the face of a setback, this one should be easy to interpret.

Daniel O'Day [via AP Images]

Gilead un­leash­es a $5B late-stage cash al­liance with Gala­pa­gos — lay­ing out O'­Day's R&D strat­e­gy

Daniel O’Day is executing his first major development deal since taking over as CEO of Gilead $GILD. And he’s going in deep to ally himself with a longstanding partner.

O’Day announced today that he is spending $5 billion in cash to add new late-stage drugs to Gilead’s pipeline, picking up rights to Galapagos’ $GLPG Phase III IPF drug GLPG1690 alongside adoption of the biotech’s Phase IIb drug GLPG1972 for osteoarthritis. And Gilead is also putting billions more on the table for milestones, gaining options for everything else in Galapagos’ pipeline, with a shot at all rights outside of Europe.

Altogether, Gilead is gaining rights to 6 clinical-stage assets, 20 preclinical programs and everything else being hatched in translation.

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Feng Zhang, MIT

Broad star Feng Zhang un­veils a new CRISPR plat­form, edit­ing RNA and elim­i­nat­ing Alzheimer's threat — in cells

Broad Institute star scientist Feng Zhang is back in the spotlight, adapting CRISPR technology in a shift from permanently editing DNA to revising RNA — temporarily if needed. And he illustrated the promise of this approach by deactivating APOE4, which may be a ticking time bomb for people at risk of developing Alzheimer’s.

CRISPR/Cas9 gene editing tech has taken the lab by storm, in part because of the work Zhang and his one-time colleagues Jennifer Doudna and Emmanuelle Charpentier accomplished. They’re still scrapping over the patents to the original Cas9 work. But Zhang, who founded Beam Therapeutics with David Liu and Keith Joung, has moved on in search of better tech, and in a paper published in Science, says they have made real progress in switching from DNA to RNA editing.

Am­gen, No­var­tis scrap Alzheimer's stud­ies — is BACE fi­nal­ly dead or will Bio­gen and Ei­sai car­ry on?

The BACE theory of controlling Alzheimer’s died with failed pivotal projects at Merck, Eli Lilly and their partners at AstraZeneca. Now Amgen and Novartis have come along to bulldoze it under a mound of safety threats — leaving only Biogen and Eisai to carry on with a less than zero chance of success — with the notable addition that they may actually be doing harm to patients.

After the market closed Thursday, Amgen and Novartis announced that they were dumping two pivotal programs underway with the Banner Alzheimer’s Institute on their BACE drug CNP520 (umibecestat) after an independent review of the data indicated that patients’ cognitive abilities were actually worsening at a faster pace than the placebo arm.

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Scripps-led con­sor­tium scores $129M NIH grant to work on vac­cine for stub­born HIV

Scientific effort into developing a vaccine against the HIV has proved elusive, as the virus rapidly mutates and its outer spike protein operates as an invisibility cloak, allowing it to outwit the immune system’s defensive arsenal. On Wednesday, a consortium of scientists led by Scripps Research unveiled a $129 million grant from the National Institutes of Health (NIH) that will help shepherd vaccines — engineered to combat a plethora of HIV strains — into the clinic.

Faced with a PhI­II flop in NASH, Gilead is se­quenc­ing 15,000 pa­tients to find new drugs

Now well into its campaign to develop a new cocktail therapy for NASH — which has initially proved unsuccessful in its first pivotal study — Gilead is going back to the genetic drawing board in search of some added inspiration.

The big biotech has struck a deal with the Renown Institute for Health Innovation, offering to foot the bill for their work sequencing the genes of 15,000 people with NASH and nonalcoholic fatty liver disease and compare them against a backdrop of 40,000 healthy people in Nevada.

Analy­sis: In most of the Big Phar­ma world, R&D spend­ing tow­ers over rev­enue from new drugs. Guess who beat the odds

It’s always been the case that there’s no medicine less useful than one that doesn’t make it to patients (unless you regard the task of R&D to be perpetually learning about swimming, while under water…). Yet, launching new medicines that physicians want, that payers will cover, and that patients will take, is a discipline that is unevenly distributed among the big players.

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