Better than CAR-T? Already prepping for a US launch, MorphoSys posts stellar DLBCL data to field against Novartis, Gilead

May 17, 2019 07:27 AM EDTUpdated May 21, 10:40 AM

Better than CAR-T? Already prepping for a US launch, MorphoSys posts stellar DLBCL data to field against Novartis, Gilead

John Carroll

Editor & Founder

Already beavering away at building a US commercial operation, MorphoSys $MOR is now bearing down on Novartis and Gilead with a potential CAR-T killer soon to be put into the hands of US regulators.

The German biotech says its final cut of the data on their CD19-targeting antibody tafasitamab — or MOR208 — combined with lenalidomide crossed the finish line in a group of patients suffering from treatment-resistant DLBCL, a common form of non-Hodgkin lymphoma.

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Michel Vounatsos, Biogen CEO (via YouTube)

October 21, 2020 08:11 AM EDTUpdated 10:22 AM

R&D

UPDATED: Biogen spotlights a pair of painful pipeline setbacks as aducanumab showdown looms at the FDA

John Carroll

Editor & Founder

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.

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October 22, 2020 10:19 PM EDTUpdated 06:34 AM

Regulatory

Coronavirus

In historic Covid-19 adcomm, vaccine experts debate a sea of questions — but offer no clear answers

Jason Mast

Associate Editor

Nicole DeFeudis

Associate Editor

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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October 23, 2020 10:30 AM EDT

R&D

A top drug program at Bayer clears a high bar for CKD — opening the door to an FDA pitch

John Carroll

Editor & Founder

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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ENDPOINTS CAREERS

Clinical Operations Lead

Vigeo Therapeutics

Cambridge, MA

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October 23, 2020 04:25 PM EDT

R&D

BiondVax stock implodes after a big PhIII gamble for its universal flu vaccine fails

Nicole DeFeudis

Associate Editor

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

October 23, 2020 03:17 PM EDTUpdated 04:55 PM

Regulatory

Coronavirus

UPDATED: FDA gives AstraZeneca the thumbs-up to restart PhIII Covid-19 vaccine trials, and J&J is preparing to resume its study

Max Gelman

Associate Editor

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

October 23, 2020 11:19 AM EDTUpdated 12:53 PM

Deals

Cell/Gene Tx

Ultragenyx injects $40M to grab Solid's microdystrophin transgene — while sidestepping the AAV9 vector that stirred up safety fears

Amber Tong

Editor

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Adam Koppel and Jeffrey Schwartz, Bain

October 23, 2020 08:05 AM EDT

IPOs

Bain execs Adam Koppel and Jeffrey Schwartz line up $125M for their first blank check deal as Wall Street continues to embrace biotech

John Carroll

Editor & Founder

Adam Koppel and Jeffrey Schwartz have jumped into the blank check game, raising $125 million for a stock listing in search of a company.

Their SPAC, BCLS Acquisition Corp, raised $125 million this week, with a line on $25 million more as it scouts for a biotech in search of money and a place on Wall Street.

The two principals at Bain Life Sciences have been on a romp since they set up the Bain operation 4 years ago. Their S-1 spells out a track record of 22 deals totaling $650 million for the life sciences group, which led to 9 IPOs.

ENDPOINTS CAREERS

Director of Regulatory Affairs

Recursion Pharmaceuticals

Salt Lake City, UT

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October 23, 2020 07:22 AM EDTUpdated 10:29 AM

Coronavirus

Covid-19 roundup: An mRNA player gets a boost out of the latest round of animal data; PhaseBio pulls the plug on treatment trial

Nicole DeFeudis

Associate Editor

Max Gelman

Associate Editor

John Carroll

Editor & Founder

The big tell for CureVac $CVAC is coming up with a looming early-stage readout on their mRNA Covid-19 vaccine in the clinic. But for now they’ll make do with an upbeat assessment on the preclinical animal data they used to get into the clinic.

Researchers for the German biotech say they got the high antibody titers and T cell activation they were looking for, lining up a hamster challenge to demonstrate — in a simple model — that the vaccine could protect the furry creatures. Like the other mRNA vaccines, the drug sends instructions to spur cells to decorate themselves with the distinctive spike on the virus to elicit an immune response.

Stephen Hahn, FDA commissioner (AP Images)

October 20, 2020 10:21 AM EDTUpdated 10:48 AM

Regulatory

Coronavirus

As FDA sets the stage for the first Covid-19 vaccine EUAs, some big players are asking for a tweak of the guidelines

John Carroll

Editor & Founder

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.

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