If you dig into the small print on Roche’s Q1 report out Wednesday, you’ll find several high profile mid- and late-stage setbacks for its experimental drugs in the pipeline.

First up is RG7388 idasanutlin, where Roche noted that its 440-patient Phase III in relapsed and refractory AML — dubbed Mirros — failed the primary endpoint. The trial of the MDM2 inhibitor was axed from the pipeline tracker, along with a Phase II for polycythemia vera, which was termed a pipeline prioritization decision.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,200+ biopharma pros reading Endpoints daily — and it's free.

SUBSCRIBE SIGN IN

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,200+ biopharma pros reading Endpoints daily — and it's free.

SUBSCRIBE SIGN IN

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,200+ biopharma pros reading Endpoints daily — and it's free.

SUBSCRIBE SIGN IN

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,200+ biopharma pros reading Endpoints daily — and it's free.

SUBSCRIBE SIGN IN

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,200+ biopharma pros reading Endpoints daily — and it's free.

SUBSCRIBE SIGN IN

It’s only right that one of the industry’s top dealmakers just completed one of the biggest SPAC-related deals in the pipeline.

David Hung, of Medivation fame, has completed a back flip into the market, merging with EcoR1 Capital’s SPAC Panacea and landing neatly on Wall Street with an $NUVB stock ticker after filling out the blank check in his name. In addition to the $144 million held in the SPAC — provided none of the investors opt out — Hung is getting ahold of $500 million more being chipped in by a slate of institutional investors who feel that Hung could have the keys to another Medivation-style success.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,200+ biopharma pros reading Endpoints daily — and it's free.

SUBSCRIBE SIGN IN