Pfizer gene therapy ups levels of dystrophin expression in Duchenne trial — but another safety setback mars results

May 15, 2020 08:02 AM EDTUpdated 09:47 AM

Pfizer gene therapy ups levels of dystrophin expression in Duchenne trial — but another safety setback mars results

John Carroll

Editor & Founder

Pfizer’s gene therapy group is back up at bat with a fresh update of data from its Phase Ib study for a gene therapy intended as a once-and-done treatment for Duchenne muscular dystrophy. And they had some good news, and some bad news, to report.

The good news was primarily in the dystrophin expression they tracked among the now 9 evaluable patients in their first clinical foray for PF-06939926.

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Targeting a Potential Vulnerability of Certain Cancers with DNA Damage Response

Andree Blaukat

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

February 25, 2021 08:31 AM ESTUpdated 09:46 AM

Editor's note

Follow biotechs going public with the Endpoints News IPO Tracker

Max Gelman

Associate Editor

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Tom Barnes (Orna)

February 24, 2021 07:00 AM ESTUpdated 07:20 AM

Startups

Cell/Gene Tx

The mRNA era is here. MPM believes the future belongs to oRNA — and Big Pharma wants a seat at the table

Amber Tong

Senior Editor

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

February 25, 2021 09:17 AM EST

R&D

Manufacturing

CMO Tal Zaks bids Moderna a surprise adieu as biotech projects $18.4B in revenue, plots post-Covid expansion

Jason Mast

Editor

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Steve Cutler, Icon CEO (Icon)

February 24, 2021 10:50 AM ESTUpdated 11:53 AM

Deals

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sciences to focus on decentralized clinical work

Max Gelman

Associate Editor

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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February 25, 2021 07:00 AM EST

R&D

Coronavirus

GlaxoSmithKline rethinks strategy for Covid-19 antibody — not the Vir ones — after trial flop. Is there hope in high-risk patients?

Kyle Blankenship

Managing Editor

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.

February 25, 2021 09:34 AM EST

Pharma

S&P expects steady erosion in Big Pharma's credit profile in 2021 as new M&A deals roll in — but don't underestimate their underlying strength

John Carroll

Editor & Founder

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

February 24, 2021 04:14 PM ESTUpdated February 24, 08:06 PM

R&D

Moderna's Stéphane Bancel plans to double down on vaccine production, new variants as mRNA rules in pandemic fight

Jason Mast

Editor

Stéphane Bancel thought he’d be sleeping more by now.

The 48-year-old Moderna CEO figured that by 2021 he’d have his vaccine through the clinic, authorized, and in mass production — that the hard part would be over. Instead, he’s still working Saturdays and Sundays, talking with his lab and manufacturing teams and fielding calls with two to three world leaders a day to answer their concerns about supply and emerging new variants.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

February 25, 2021 07:43 AM ESTUpdated 10:46 AM

Deals

Pharma

UPDATED: Merck takes a swing at the IL-2 puzzlebox with a $1.85B play for buzzy Pandion and its autoimmune hopefuls

Kyle Blankenship

Managing Editor

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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