An emboldened AstraZeneca splurges $95M on a priority review voucher. Where do they need the FDA to hustle up?

August 22, 2019 08:47 AM EDTUpdated 01:25 PM

An emboldened AstraZeneca splurges $95M on a priority review voucher. Where do they need the FDA to hustle up?

John Carroll

Editor & Founder

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Cell and Gene Contract Manufacturers Must Embrace Digitization

Rachit Jain

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Inside FDA HQ (File photo)

August 12, 2020 06:30 PM EDTUpdated 4 hours ago

Pharma

Regulatory

The FDA just approved the third Duchenne MD drug. And regulators still don’t know if any of them work

Max Gelman

Associate Editor

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

August 11, 2020 09:29 AM EDTUpdated 9 hours ago

Coronavirus

In Focus

UPDATED: Endpoints News ranks all 28 players in the Covid-19 vaccine race. Here's how it stacks up today

Nicole DeFeudis

Associate Editor

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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ENDPOINTS CAREERS

Director of Regulatory Affairs

Recursion Pharmaceuticals

Salt Lake City, UT

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August 13, 2020 10:49 AM EDT

Deals

R&D

Calliditas bets up to $102M on a biotech buyout, snagging a once-failed PBC drug

Nicole DeFeudis

Associate Editor

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to €55 million in contingent rights.

James Wilson, WuXi Global Forum at JPM20

August 13, 2020 09:13 AM EDTUpdated 10:44 AM

R&D

Regulatory

FDA puts up a red light for Passage Bio’s first gene therapy program, delaying a program from James Wilson's group at Penn

John Carroll

Editor & Founder

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

August 12, 2020 06:48 AM EDTUpdated 07:02 AM

Coronavirus

UPDATED: Moderna offers steep discount in US supply deal — but still takes the crown with close to $2.5B in vaccine contracts

Amber Tong

Editor

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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August 13, 2020 05:57 PM EDT

Regulatory

FDA's ODAC shrugs off objections to Mesoblast's GVHD drug for children, voting 8-2 in favor and improving the odds of an approval

Max Gelman

Associate Editor

The FDA’s Oncologic Drugs Advisory Committee once again waved through an investigational drug, clearing the potential final hurdle before the agency’s decision.

Thursday’s winner was Mesoblast $MESO, an Australian stem-cell company that submitted its Ryoncil drug for the treatment of steroid refractory acute graft-versus-host disease in children younger than 12. ODAC gave Ryoncil the thumbs up by an 8-2 vote, shrugging off concerns about trial design and pushing aside the need for an additional study.

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ENDPOINTS CAREERS

Director of Clinical Operations

Recursion Pharmaceuticals

Salt Lake City, UT

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August 10, 2020 07:15 AM EDTUpdated 09:52 AM

R&D

Phase III readouts spell disaster for Genentech’s lead IBD drug

Jason Mast

Associate Editor

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

August 13, 2020 10:20 AM EDTUpdated 10:44 AM

Deals

Qiagen investors spurn Thermo Fisher’s takeover offer, derailing a $12B+ deal

Max Gelman

Associate Editor

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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