Mikael Dolsten, Pfizer CSO

FDA re­leas­es sec­ond Pfiz­er gene ther­a­py from clin­i­cal hold, as he­mo­phil­ia pro­gram gets OK to restart

The FDA has re­leased a clin­i­cal hold on Pfiz­er and Sang­amo’s he­mo­phil­ia A gene ther­a­py af­ter or­der­ing the com­pa­nies to stop the tri­al last year due to con­cerns about blood clots.

Just a week af­ter Pfiz­er an­nounced the FDA had un­leashed its gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy from a clin­i­cal hold — which was paused as one pa­tient died and oth­ers ex­pe­ri­enced se­vere side ef­fects  — an­oth­er one of its gene ther­a­py pro­grams has been giv­en the green light to re­sume, Pfiz­er an­nounced dur­ing its Q1 earn­ings call yes­ter­day.

How­ev­er, the com­pa­ny an­tic­i­pates it won’t restart the Phase III tri­al un­til the third quar­ter of this year, Pfiz­er CSO Mikael Dol­sten not­ed dur­ing the meet­ing. In the mean­time, Pfiz­er needs to get ap­proval of their up­dat­ed study pro­to­cols from the FDA, ac­cord­ing to their Q1 earn­ings news re­lease.

Pfiz­er vol­un­tar­i­ly paused the study late last year af­ter pa­tients ex­pe­ri­enced ab­nor­mal­ly high lev­els of fac­tor VI­II, an es­sen­tial blood clot­ting fac­tor. In he­mo­phil­ia A, pa­tients lack a func­tion­al gene for mak­ing fac­tor VI­II, mean­ing that they have very low fac­tor VI­II lev­els at un­der 50%. In peo­ple with­out he­mo­phil­ia A, fac­tor VI­II lev­els usu­al­ly range be­tween 50% and 100%, but in the Pfiz­er tri­al, pa­tients had fac­tor VI­II of above 150%, putting them at risk for blood clots.

Soon af­ter Pfiz­er paused the tri­al, the FDA slapped on a clin­i­cal hold.

One pa­tient in the tri­al ex­pe­ri­enced deep vein throm­bo­sis be­low the knee, Pfiz­er not­ed in the news re­lease. That pa­tient had a his­to­ry of blood clots be­fore the tri­al be­gan, which is an ex­clu­sion cri­te­ria for par­tic­i­pat­ing in the study. How­ev­er, the pa­tient is now do­ing well, Pfiz­er said.

Now with the clin­i­cal hold gone, Pfiz­er and Sang­amo may have a chance to catch up to Bio­Marin, as the biotech plots to re­turn to the FDA with its gene ther­a­py this year. Bio­Marin’s gene ther­a­py was re­ject­ed by the FDA two years ago af­ter mul­ti­ple re­ports of fac­tor VI­II lev­els dwin­dling in pa­tients just years af­ter they re­ceived what is sup­posed to be a long-term fix.

Pfiz­er ex­pects a read­out from their he­mo­phil­ia A pro­gram some­time in the sec­ond half of 2023.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

BREAK­ING: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

FDA's ad­vanced ther­a­pies of­fice pro­vides more clar­i­ty on gene ther­a­py CMC con­sid­er­a­tions

As the Office of Tissue and Advanced Therapies (OTAT) transforms into the Office of Therapeutic Products (OTP), with new user fee funds and “super office” status, the department focused on cell and gene therapies also opened its doors to a town hall Thursday offering clarification on guidance and regulations for manufacturers.

Some of the major concerns from manufacturers were the CMC considerations between first-in-human studies and late-phase studies supporting a marketing approval.

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CMS spent more than $18B in four years on ac­cel­er­at­ed ap­provals with in­com­plete con­fir­ma­to­ry tri­als, in­spec­tor gen­er­al finds

The battle over whether and how to reform the FDA’s accelerated approval pathway is heating up again, just as the Senate punted any talks until the lame duck session just before the end of the year.

On Thursday, HHS’ inspector general released a new report reiterating concerns, also noted recently by the FDA’s Oncology Center of Excellence, about delayed or slowed confirmatory trials that are necessary to prove that the accelerated approvals were worth their salt in the first place.

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Cell and gene ther­a­pies from acad­e­mia: EMA to help 5 projects go­ing af­ter un­met clin­i­cal needs

The European Medicines Agency said Thursday that it’s launching a new pilot program to help academic and other nonprofit researchers developing advanced therapy medicinal products, which includes cell and gene therapies.

Academics have proven to be enormously useful in feeding new products, like chimeric antigen receptor (CAR)-T cell therapies first developed by Memorial Sloan Kettering, and ushered to the market by biopharma companies. Jean Bennett, formerly with the University of Pennsylvania, also saw her research lead to the approval of gene therapy Luxturna, which Roche now owns.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) (Olivier Douliery/Sipa USA (Sipa via AP Images)

Sen­ate pass­es bill to re­work an­i­mal test­ing re­quire­ments for drug de­vel­op­ers

The US Senate passed via unanimous consent on Thursday afternoon a bipartisan bill that would alter a federal mandate for animal testing on new drugs, but stops short of removing animal testing entirely.

Touted as a much-needed modernization of FDA’s rules, co-sponsor Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) have said the bill will stop lots of needless suffering of animals.